[{"id":"i131-mibg","kind":"drug","name":"131I-MIBG (iobenguane I-131) therapy","aka":["Iobenguane I-131 (therapeutic)","131I-MIBG","Ultratrace iobenguane","iobenguane-i-131"],"tldr":"High-dose radioactive MIBG delivers radiation from inside neuroblastoma cells that take up noradrenaline; used for relapsed disease and tested in upfront therapy. A branded high-specific-activity form, Azedra, was approved for phaeochromocytoma and paraganglioma in 2018 and discontinued in 2024.","summary":"Response rate ~30-40% in relapsed/refractory MIBG-avid neuroblastoma (NANT, COG studies), with myelosuppression requiring stem-cell support at ≥12 mCi/kg. COG ANBL1531 randomised 131I-MIBG added to induction (primary results pending 2026).\n\nHigh-specific-activity iobenguane I-131 (Azedra, Progenics then Lantheus) was approved by the FDA in July 2018 for adults and children aged 12 and over with iobenguane scan-positive, unresectable, locally advanced or metastatic phaeochromocytoma or paraganglioma requiring systemic therapy, on a single-arm phase 2 study in which a quarter of patients halved their antihypertensive medication for at least six months and most had tumour control; it was the first approved therapy for these tumours. Lantheus discontinued manufacture in 2024, so patients rely on compounded low-specific-activity 131I-MIBG or lutetium-177 dotatate. Myelosuppression, secondary MDS and leukaemia, hypothyroidism and renal toxicity are the main risks; the diagnostic-dose iobenguane (AdreView) is an imaging agent covered under MIBG theranostics.","status":"established","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Iobenguane","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Iobenguane"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=iobenguane"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/iobenguanei131"}],"tags":["nci-list"],"related":["lutathera"],"cancers":["neuroblastoma","neuroblastoma-high-risk","metastatic-ppgl","neuroendocrine"],"sections":[],"technologies":["mibg-theranostics","radioligand-therapy"],"targets":[],"drugs":[],"companies":["lantheus"],"institutions":[],"pathways":[],"terms":["radioiodine-term"],"trials":["anbl1531"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Azedra (the approved high-specific-activity product) and compounded 131I-MIBG are one INN, iobenguane I-131, so both are recorded here; the duplicate record iobenguane-i-131 redirects to this page."],"brand":"Azedra","modality":"Radioligand therapy (beta, norepinephrine transporter)","mechanism":"Norepinephrine transporter uptake of radio-iodinated benzylguanidine; 131I beta emission (2 mm range).","approvals":[{"region":"US","year":2018,"indication":"Iobenguane scan-positive unresectable, locally advanced or metastatic phaeochromocytoma or paraganglioma in patients 12 and older (Azedra, high-specific-activity form; discontinued 2024)"}],"mechanismSteps":[],"dosing":{"route":"IV","schedule":"12-18 mCi/kg single or tandem doses with autologous stem-cell support; thyroid blockade with potassium iodide","monitoring":"Radiation isolation, counts, thyroid function, secondary malignancy"},"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"itm-11","kind":"drug","name":"177Lu-edotreotide","aka":[],"tldr":"A second lutetium radioligand for neuroendocrine tumours that beat the standard pill everolimus in a head-to-head trial and is awaiting an FDA decision.","summary":"COMPETE (Lancet 2025; 309 patients, grade 1-2 GEP-NETs): PFS 23.9 vs 14.1 months versus everolimus (HR 0.67); response rate higher; interim OS 63.4 vs 58.7 months, a difference that did not reach statistical significance (HR 0.78). Uses non-carrier-added 177Lu from ITM's own supply. NDA accepted with PDUFA 28 August 2026. COMPOSE (grade 2-3, versus CAPTEM/everolimus/FOLFOX) ongoing.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT03049189 (COMPETE)","url":"https://clinicaltrials.gov/study/NCT03049189"}],"tags":[],"related":[],"cancers":["neuroendocrine","grade-3-net"],"sections":[],"technologies":["prrt","radioligand-therapy"],"targets":["sstr2"],"drugs":[],"companies":["itm"],"institutions":[],"pathways":[],"terms":[],"trials":["compete","nct04919226"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ITM-11, n.c.a. 177Lu-DOTATOC","modality":"Radioligand therapy (beta)","mechanism":"DOTATOC peptide (SSTR2 agonist) chelating 177Lu; four cycles of 7.5 GBq every 3 months.","approvals":[],"mechanismSteps":[],"dosing":{"route":"Intravenous","schedule":"7.5 GBq every 3 months × 4 with amino-acid renal protection"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2025-03","type":"designation","region":"US","note":"COMPETE topline at ENETS 2025","source":"https://www.itm-radiopharma.com/news/press-releases/press-releases-detail/itm-presents-positive-topline-phase-3-compete-trial-data-with-nca-177lu-edotreotide-itm-11-a-targeted-radiopharmaceutical-therapy-in-patients-with-grade-1-or-2-gastroenteropancreatic-neuroendocrine-tumors-at-the-enets-2025-conference-688/"},{"date":"2026-08-28","type":"pdufa","region":"US","note":"PDUFA goal date for GEP-NET indication","source":"https://www.cancernetwork.com/view/fda-accepts-new-drug-application-for-177lu-edotreotide-in-gep-nets"}]},{"id":"lu177-psma-it","kind":"drug","name":"177Lu-PSMA-I&T","aka":[],"tldr":"177Lu-PSMA-I&T is a second PSMA radioligand, chemically different from Pluvicto, that has passed two phase 3 trials on progression but has not yet shown a survival benefit.","summary":"SPLASH (Lantheus PNT2002, ESMO 2024): rPFS 9.5 vs 6.0 months (HR 0.71) vs ARPI switch in taxane-naive mCRPC; interim OS HR 1.11 (immature, crossover). ECLIPSE (Curium, 7.4 GBq × 6): met rPFS endpoint vs ARPI in the same setting; OS maturing; FDA submission plan pending. Curium's Japanese registrational trial with PeptiDream started February 2026. Widely used off-protocol in Germany and Australia.","status":"phase-3","asOf":"2026-09-06","links":[{"label":"SPLASH primary analysis (Lantheus, ESMO 2024)","url":"https://lantheusholdings.gcs-web.com/news-releases/news-release-details/lantheus-presents-results-primary-analysis-phase-3-pivotal"},{"label":"ECLIPSE meets rPFS (OncLive)","url":"https://www.onclive.com/view/177lu-psma-i-t-meets-rpfs-end-point-in-psma-mcrpc"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["radioligand-therapy","psma-pet"],"targets":["psma"],"drugs":[],"companies":["lantheus","curium"],"institutions":[],"pathways":[],"terms":[],"trials":["splash","eclipse-psma"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"PNT2002 (Lantheus); Curium 177Lu-PSMA-I&T","modality":"Radioligand therapy (beta)","mechanism":"Urea-based PSMA ligand with DOTAGA chelator carrying 177Lu; beta emission.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"alphamedix","kind":"drug","name":"212Pb-DOTAMTATE","aka":[],"tldr":"An alpha-particle version of neuroendocrine radioligand therapy that produced responses in over half of patients who had never had PRRT, with FDA Breakthrough designation.","summary":"ALPHAMEDIX-02 (Sanofi/Orano Med/RadioMedix, October 2025): met all primary endpoints; ORR 54.3% in PRRT-naive patients with ~70-75% progression-free at about 3 years, and durable disease control in PRRT-exposed patients (~83% progression-free at 18 months). Breakthrough Therapy designation 2024. Sanofi acquired global rights (2024); registrational strategy under discussion. Supply relies on Orano Med's 212Pb generators.","status":"phase-2","asOf":"2026-09-07","links":[{"label":"Sanofi press release Oct 2025","url":"https://www.sanofi.com/en/media-room/press-releases/2025/2025-10-08-05-00-00-3163053"}],"tags":[],"related":[],"cancers":["neuroendocrine"],"sections":[],"technologies":["prrt","targeted-alpha-therapy"],"targets":["sstr2"],"drugs":[],"companies":["orano-med","radiomedix","sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["alphamedix-02"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"AlphaMedix","modality":"Targeted alpha therapy (lead-212)","mechanism":"DOTAMTATE (SSTR2 agonist) chelating 212Pb, which decays via 212Bi to emit an alpha particle at the tumour cell.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aminolevulinic-acid-gleolan","kind":"drug","name":"5-Aminolevulinic acid (oral, for glioma surgery)","aka":[],"tldr":"A drink taken before brain surgery that makes high-grade glioma glow under blue light, letting surgeons remove more tumour safely.","summary":"Oral 5-aminolevulinic acid is taken up by glioma cells and converted to protoporphyrin IX, which fluoresces pink-red under blue light at 400 to 410 nm through the surgical microscope, so the surgeon can distinguish tumour from brain and resect more of it while sparing normal tissue. Stummer's randomised trial (Lancet Oncology 2006) showed complete resection in 65% versus 36% of patients and better six-month progression-free survival. The EMA approved it in 2007 for WHO grade III and IV glioma surgery and the FDA in 2017, the first optical imaging agent for brain surgery. The trial was not powered for overall survival. Related aminolevulinate compounds are used topically for actinic keratosis photodynamic therapy (Levulan, Ameluz) and in the bladder (hexaminolevulinate). It is a drink that makes brain tumours glow.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Aminolevulinic_acid","links":[{"label":"Stummer 2006 (Lancet Oncol)","url":"https://doi.org/10.1016/S1470-2045(06)70665-9"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Gleolan"}],"tags":["gap-fill","diagnostic"],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":["fluorescence-guided-surgery","optical-imaging"],"targets":[],"drugs":[],"companies":["photonamic"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05804370"],"people":[],"bottlenecks":[],"keyPapers":["paper-stummer-lancet-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Gleolan / Gliolan","modality":"Fluorescence-guided surgery agent (protoporphyrin IX precursor)","mechanism":"Oral 5-ALA is taken up by glioma cells and converted to protoporphyrin IX, which fluoresces pink-red under blue light (400-410 nm) through the surgical microscope.","approvals":[{"region":"EU","year":2007,"indication":"Visualisation of malignant tissue during surgery for WHO grade III-IV glioma"},{"region":"US","year":2017,"indication":"Imaging agent for visualisation of malignant tissue during glioma surgery (suspected high-grade)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"a166","kind":"drug","name":"A166","aka":[],"tldr":"A166 is an experimental antibody-drug conjugate from Sichuan Kelun-Biotech Biopharmaceutical in phase 3 trials for HER2-positive breast cancer, aimed at HER2.","summary":"A166 is an antibody-drug conjugate developed by Sichuan Kelun-Biotech Biopharmaceutical. Its target is HER2 (the sponsor names HER2). The sponsor states: A166 is a HER2-targeted antibody-drug conjugate with a microtubule inhibitor payload, administered intravenously at 4.8 mg/kg every three weeks (Q3W) to HER2-positive breast cancer patients. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06968585 (A Study of A166 Versus Trastuzumab Emtansine (T-DM1) in Patients With HER2-Positive Unresectable or Metastatic Breast Cancer Previously Treated With Trastuzumab and Taxane Therapy), in HER2-positive breast cancer. The largest, NCT06968585, plans to enrol 365 participants (actual) with primary completion expected 2026-11-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of A166","url":"https://clinicaltrials.gov/search?intr=A166"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["kelun-biotech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06968585","nct07299825"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"A166 is a HER2-targeted antibody-drug conjugate with a microtubule inhibitor payload, administered intravenously at 4.8 mg/kg every three weeks (Q3W) to HER2-positive breast cancer patients.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aaa817","kind":"drug","name":"AAA817","aka":["[225Ac]Ac-PSMA-617"],"tldr":"AAA817 is an experimental radioligand therapy from Novartis Pharmaceuticals in phase 3 trials for prostate cancer, aimed at PSMA.","summary":"AAA817 is a radioligand therapy developed by Novartis Pharmaceuticals. Its target is PSMA (the sponsor names PSMA). The sponsor states: Inside the body, AAA817 ([225Ac]Ac-PSMA-617) attaches to PSMA on the surface of prostate cancer cells and emits radiation to kill them; this treatment is also called a radioligand therapy. ClinicalTrials.gov describes the intervention as: AAA817 is being studied for treating PSMA positive mCRPC. Inside the body, it attaches itself to PSMA on the cell surface of the prostate cancer cells and emits radiation to kill them. This treatment is also called a radioligand therapy. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06855277 (Study Comparing AAA817+ARPI Versus Standard of Care in Adult Participants With PSMA-positive mCRPC) and NCT06780670 (Open-label Study Comparing AAA817 Versus Standard of Care in the Treatment of Previously Treated PSMA-positive mCRPC Adults Who Have Disease Progressed on or After [177Lu]Lu-PSMA Targeted Therapy), in prostate cancer. The largest, NCT06855277, plans to enrol 940 participants with primary completion expected 2028-09-29. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AAA817","url":"https://clinicaltrials.gov/search?intr=AAA817"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["psma"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06855277","nct06780670"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"radioligand","mechanism":"Inside the body, AAA817 ([225Ac]Ac-PSMA-617) attaches to PSMA on the surface of prostate cancer cells and emits radiation to kill them; this treatment is also called a radioligand therapy.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"abarelix","kind":"drug","name":"Abarelix","aka":[],"tldr":"Abarelix was the first GnRH antagonist for prostate cancer, approved in the United States in 2003 for men who could not take agonists, but allergic reactions restricted it and it was withdrawn from the US market in 2005; it remained available in Germany.","summary":"Abarelix lowers testosterone to castrate levels within about a week without the flare seen with leuprolide or goserelin, which made it useful for men with impending spinal cord compression or severe bone pain. The FDA approved it in 2003 under a restricted programme because of immediate-onset systemic allergic reactions, and the manufacturer withdrew it from the US market in 2005. It was later approved in Germany. Degarelix, approved in 2008, took over the GnRH antagonist role in most countries.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Abarelix","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Abarelix"},{"label":"ChEMBL CHEMBL1201180","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201180"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["endocrine-therapy"],"targets":["gnrhr"],"drugs":["degarelix","leuprolide"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct00841113","nct00100243"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Plenaxis","modality":"GnRH antagonist peptide, intramuscular depot","mechanism":"Blocks the pituitary GnRH receptor directly, so luteinising hormone and testosterone fall within days without the initial testosterone surge that GnRH agonists cause.","approvals":[{"region":"US","year":2003,"indication":"Advanced symptomatic prostate cancer in men who cannot take GnRH agonists (restricted programme)","note":"Withdrawn from the US market in 2005"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"abbv-706","kind":"drug","name":"ABBV-706","aka":[],"tldr":"ABBV-706 is an experimental antibody-drug conjugate from AbbVie in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"ABBV-706 is an antibody-drug conjugate developed by AbbVie. The sponsor describes its target as SEZ6 (seizure-related homolog 6), which OnCo does not yet have a target page for. The sponsor states: ABBV-706 is described as a targeted antibody-drug conjugate; a trial exclusion criterion barring prior SEZ6-targeted ADC use indicates it targets SEZ6, given intravenously in small cell lung cancer, alone or combined with atezolizumab. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07365241 (A Study to Evaluate Adverse Events and Change in Disease Activity of Intravenous ABBV-706 Versus Standard of Care in Adult Participants With Relapsed/Refractory Small Cell Lung Cancer), in non-small-cell lung cancer. The largest, NCT07365241, plans to enrol 531 participants with primary completion expected 2030-09. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ABBV-706","url":"https://clinicaltrials.gov/search?intr=ABBV-706"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07365241","nct07155174"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"ABBV-706 is described as a targeted antibody-drug conjugate; a trial exclusion criterion barring prior SEZ6-targeted ADC use indicates it targets SEZ6, given intravenously in small cell lung cancer, alone or combined with atezolizumab.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"abemaciclib","kind":"drug","name":"Abemaciclib","aka":[],"tldr":"Abemaciclib was the first CDK4/6 inhibitor approved after surgery for high-risk hormone-positive breast cancer.","summary":"Abemaciclib is a CDK4-biased CDK4/6 inhibitor potent enough for continuous dosing, at 150 mg twice daily with endocrine therapy or 200 mg twice daily alone. MONARCH 2 and 3 established it in HR-positive, HER2-negative advanced breast cancer (overall survival benefit in MONARCH 2), and monarchE made it the first CDK4/6 inhibitor approved after surgery for high-risk node-positive disease (2021, label broadened 2023): 2 years of treatment gave an invasive disease-free survival HR of 0.68 at 5 years (83.6% versus 76.0%), with a favourable but not yet significant overall survival trend. Eli Lilly markets it. Diarrhoea is the defining toxicity (85%, 8% grade 3 or higher). Whether the recurrence benefit becomes a survival benefit is open. For a newcomer: the CDK4/6 pill that first proved it could stop early breast cancer coming back.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Abemaciclib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Abemaciclib"}],"tags":[],"related":[],"cancers":["breast-hr-positive","hr-positive-early-high-risk","hr-positive-metastatic-post-cdk46"],"sections":[],"technologies":["cdk46-inhibitor"],"targets":["cdk4-6"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["monarche","nct02152631","nct03706365","nct05735080","nct06810544","nct05867251","nct05768139","nct07100106","nct06413706","nct06065748","nct05440786","nct02763566","nct05999994","nct05696626","nct07002177","nct02107703","nct05548127","nct02747004","nct05288166","nct04862663","nct06380751","nct06760637","nct05386108","nct05573126","nct07605728"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Verzenio","modality":"Small-molecule CDK4/6 inhibitor","mechanism":"CDK4-biased inhibitor with continuous dosing.","approvals":[{"region":"US","year":2017,"indication":"HR+/HER2- advanced breast cancer"},{"region":"US","year":2021,"indication":"Adjuvant high-risk node-positive HR+/HER2- early breast cancer"}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of CDK4 (and CDK6)","Phosphorylation of downstream substrates stops","Continuous dosing keeps RB unphosphorylated; less neutropenia, more diarrhoea than the 3-weeks-on drugs","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral, continuous","schedule":"150 mg twice daily with endocrine therapy (2 years adjuvant); 200 mg twice daily as monotherapy","modifications":"Reduce to 100 then 50 mg BID for grade 3 diarrhoea, neutropenia, hepatotoxicity; loperamide at first loose stool","monitoring":"Blood counts and LFTs every 2 weeks for 2 months then monthly; VTE and ILD symptoms","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06"},"toxicity":[{"event":"Diarrhoea","anyGradePct":85,"grade3PlusPct":8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"81-90% any grade; 8-20% grade 3 across trials"},{"event":"Neutropenia","anyGradePct":42,"grade3PlusPct":19,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"37-46% any grade; 19-32% grade 3-4"},{"event":"Infections","grade3PlusPct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"monarchE / MONARCH 2"},{"event":"Venous thromboembolism","grade3PlusPct":2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"2-5% across trials"},{"event":"Interstitial lung disease","grade3PlusPct":0.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"monarchE / MONARCH 2"},{"event":"Fatigue","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"monarchE / MONARCH 2"},{"event":"Nausea","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"monarchE / MONARCH 2"},{"event":"Anaemia","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"monarchE / MONARCH 2"},{"event":"ALT increased","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=be4bc0de-0fdc-4d46-8d25-be43c79e6a06","note":"monarchE / MONARCH 2"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.lillycares.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with fulvestrant (TA579), aromatase inhibitor (TA563), and adjuvant (TA810)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-09-28","type":"approval","region":"US","note":"With fulvestrant after endocrine therapy, and as monotherapy after chemotherapy (MONARCH 2/1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-09-18","type":"approval","region":"US","note":"With imlunestrant, for ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-imlunestrant-combination-abemaciclib-er-positive-her2-negative-esr1-mutated-advanced-or"},{"date":"2018-02-26","type":"approval","region":"US","note":"With aromatase inhibitor first line (MONARCH 3)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-10-12","type":"approval","region":"US","note":"Adjuvant high-risk node-positive HR+/HER2- early breast cancer, Ki-67 ≥20% (monarchE)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-03-03","type":"label-change","region":"US","note":"Adjuvant indication broadened to remove Ki-67 requirement","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"abexinostat","kind":"drug","name":"Abexinostat","aka":[],"tldr":"Abexinostat is an experimental small-molecule drug from Xynomic Pharmaceuticals in phase 3 trials for renal cell carcinoma, diffuse large B-cell lymphoma and follicular lymphoma, with its target not yet stated publicly.","summary":"Abexinostat (PCI-24781) is a small-molecule drug developed by Xynomic Pharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Abexinostat tosylate salt is formulated into an oral tablet formulation and is available in 20 mg strength. It is the investigational product in 5 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT03592472 (A Study of Pazopanib With or Without Abexinostat in Patients With Locally Advanced or Metastatic Renal Cell Carcinoma (RENAVIV)), in renal cell carcinoma, diffuse large B-cell lymphoma, follicular lymphoma and hodgkin lymphoma. The largest, NCT03592472, plans to enrol 413 participants with primary completion expected 2026-12-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Abexinostat","url":"https://clinicaltrials.gov/search?intr=PCI-24781"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["rcc","dlbcl","follicular-lymphoma","hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["xynomic-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["renaviv","nct03600441","nct03936153","nct04024696","nct03934567"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PCI-24781","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"abiraterone","kind":"drug","name":"Abiraterone acetate","aka":["Abiraterone"],"tldr":"Abiraterone is a pill that shuts down testosterone production everywhere, including inside the tumour. Discovered at the Institute of Cancer Research, now generic and used from the first metastatic diagnosis.","summary":"Abiraterone acetate irreversibly inhibits CYP17A1 (17-alpha-hydroxylase and 17,20-lyase), shutting down androgen synthesis in the adrenal glands and inside the tumour, not just the testes; it is given with prednisone to offset mineralocorticoid excess. Its evidence spans advanced prostate cancer: COU-AA-301 and 302 (mCRPC post- and pre-docetaxel), LATITUDE and STAMPEDE (mHSPC, overall survival benefit; LATITUDE OS 53.3 versus 36.5 months, HR 0.66) and PEACE-1 (triplet with docetaxel). Generic since 2018 to 2019, it is the backbone partner for PARP inhibitors (PROpel, MAGNITUDE), capivasertib (CAPItello-281) and Pluvicto (PSMAddition). Open questions are how to choose between abiraterone and the AR antagonists, and which men need a third drug. Discovered at the Institute of Cancer Research, it is the affordable hormone pill most men with metastatic prostate cancer now start at diagnosis.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Abiraterone_acetate","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Abiraterone%20acetate"},{"label":"NICE TA1110: abiraterone (originator and generics) for newly diagnosed high-risk hormone-sensitive metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta1110"},{"label":"NICE TA387: abiraterone for treating metastatic hormone-relapsed prostate cancer before chemotherapy is indicated","url":"https://www.nice.org.uk/guidance/ta387"},{"label":"NICE TA259: abiraterone for castration-resistant metastatic prostate cancer previously treated with a docetaxel-containing regimen","url":"https://www.nice.org.uk/guidance/ta259"}],"tags":[],"related":[],"cancers":["prostate","prostate-mhspc","prostate-mcrpc","prostate-high-risk"],"sections":[],"technologies":["androgen-deprivation"],"targets":["androgen-receptor"],"drugs":[],"companies":["johnson-johnson"],"institutions":["icr-london","royal-marsden"],"pathways":["ar-signaling"],"terms":["prostate-uk-drug-approvals"],"trials":["latitude","stampede","peace-1","propel","magnitude","capitello-281","nct03348670","nct06520345","nct03706365","nct07213674","nct04446117","nct05171816","nct07611110","nct04691804","nct05422911","nct07287150","nct07230106","nct02960022","nct05367440","nct06991556","nct07005154","nct02861573","nct05067140","nct07198633","nct07190300","nct07553988","nct02257736","nct03431350","nct04497844"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In England, four NICE appraisals cover abiraterone in prostate cancer and the newest replaced the oldest. TA1110 recommends abiraterone (originator and generics) plus androgen deprivation with prednisolone or prednisone within its marketing authorisation for newly diagnosed high-risk hormone-sensitive metastatic prostate cancer; it is a review of TA721, which did not recommend abiraterone in this indication at all, so TA1110 reverses a refusal rather than lifting a restriction. TA387 recommends abiraterone with prednisone or prednisolone for metastatic hormone-relapsed prostate cancer in men with no or mild symptoms after androgen deprivation has failed and before chemotherapy is indicated. TA259 recommends it after one docetaxel-containing regimen. TA951 recommends olaparib with abiraterone for untreated hormone-relapsed metastatic prostate cancer in adults who cannot have or do not want chemotherapy."],"brand":"Zytiga (generic)","modality":"Small-molecule CYP17A1 inhibitor","mechanism":"Irreversible CYP17A1 (17α-hydroxylase/17,20-lyase) inhibition blocks adrenal and intratumoural androgen synthesis.","approvals":[{"region":"US","year":2011,"indication":"mCRPC after docetaxel"},{"region":"US","year":2012,"indication":"mCRPC pre-chemotherapy"},{"region":"US","year":2018,"indication":"High-risk mHSPC (LATITUDE)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-06-12","type":"approval","region":"US","note":"Capivasertib approved for use with abiraterone and prednisone in PTEN-deficient metastatic prostate cancer (CAPItello-281); abiraterone itself was already approved","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-capivasertib-abiraterone-and-prednisone-pten-deficient-androgen-pathway-modulation"}]},{"id":"absk061","kind":"drug","name":"ABSK061","aka":[],"tldr":"ABSK061 is an experimental small-molecule drug from Abbisko Therapeutics in phase 2 trials for gastric & gastro-oesophageal junction cancer, bladder & urothelial cancer and non-small-cell lung cancer, aimed at HER2 and FGFR2.","summary":"ABSK061 is a small-molecule drug developed by Abbisko Therapeutics. Its targets are HER2 and FGFR2. ClinicalTrials.gov describes the intervention as: Participants with HER2-Gastric/Gastroesophageal Junction Cancer and fibroblast growth factor receptor 2( FGFR2 ) amplification and overexpression will receive a dose of ABSK061 + ABSK043 oral capsule in combination with CAPOX until disease. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in gastric & gastro-oesophageal junction cancer, bladder & urothelial cancer and non-small-cell lung cancer. The largest, NCT06632262, plans to enrol 202 participants with primary completion expected 2029-11-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ABSK061","url":"https://clinicaltrials.gov/search?intr=ABSK061"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric","urothelial","nsclc"],"sections":[],"technologies":[],"targets":["her2","fgfr2"],"drugs":[],"companies":["abbisko"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06632262"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Small-molecule drug directed at HER2 and FGFR2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"abt-301","kind":"drug","name":"ABT-301","aka":["MPT0E028","Imofinostat"],"tldr":"ABT-301 is an experimental small-molecule drug from Anbogen Therapeutics in phase 2 trials for colorectal cancer, aimed at Histone deacetylases (HDAC).","summary":"ABT-301 is a small-molecule drug developed by Anbogen Therapeutics. Its target is Histone deacetylases (HDAC). ClinicalTrials.gov describes the intervention as: ABT-301 is an oral histone deacetylase inhibitor (HDACi) administered in capsule form once daily (QD ±3 hours) or every 12 hours (Q12H ±3 hours, with at least 9 hours between doses) with water in 21-day treatment cycles. In Part 1 (dose-esc. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in colorectal cancer. The largest, NCT07244705, plans to enrol 66 participants with primary completion expected 2028-04. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ABT-301","url":"https://clinicaltrials.gov/search?intr=ABT-301"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["hdac"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07244705"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Small-molecule drug directed at Histone deacetylases (HDAC), as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"acalabrutinib","kind":"drug","name":"Acalabrutinib","aka":[],"tldr":"Acalabrutinib is a cleaner BTK blocker with fewer heart and bleeding problems than ibrutinib. In February 2026 it became half of the first all-oral, fixed-duration CLL regimen.","summary":"ELEVATE-TN (frontline, 6-year median PFS not reached vs 27.8 months for chlorambucil-obinutuzumab; OS benefit for acalabrutinib-obinutuzumab), ELEVATE-RR (non-inferior PFS to ibrutinib with less atrial fibrillation), and AMPLIFY (acalabrutinib + venetoclax ± obinutuzumab, fixed duration: 3-year PFS 76.5%/83.1% vs 66.5% for chemoimmunotherapy) led to the 19 February 2026 approval of acalabrutinib-venetoclax for previously untreated CLL without del(17p)/TP53. Also approved in MCL (first-line 2025, ECHO). Tablet formulation removed the acid-suppressant interaction.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Acalabrutinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Acalabrutinib"}],"tags":[],"related":[],"cancers":["cll","dlbcl","cll-treatment-naive"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["btk"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["elevate-tn","amplify","nct04529772","nct05952024","nct02213926","nct05951959","nct02180711","nct03932331","nct04502394","nct07029737","nct04075292","nct07277231","nct06428019","nct02477696","nct02970318","nct07024706","nct02180724","nct05057494","nct04662255","nct02717611","nct02029443","nct02972840"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Calquence","modality":"Small-molecule covalent BTK inhibitor (second generation)","mechanism":"Highly selective covalent BTK inhibitor (C481) with minimal EGFR/TEC/ITK activity.","approvals":[{"region":"US","year":2017,"indication":"Relapsed MCL (accelerated)"},{"region":"US","year":2019,"indication":"CLL/SLL, first-line and relapsed (ELEVATE-TN, ASCEND)"},{"region":"US","year":2026,"indication":"Fixed-duration acalabrutinib + venetoclax, previously untreated CLL/SLL without del(17p)/TP53 (AMPLIFY)"}],"mechanismSteps":["Binds and inactivates BTK at C481 with high selectivity","BCR-driven survival and adhesion signals stop","Far less inhibition of EGFR and TEC kinases, so less rash, diarrhoea, atrial fibrillation","With venetoclax, mobilised cells are killed rather than merely suppressed, allowing a 14-cycle fixed course"],"dosing":{"route":"Oral","schedule":"100 mg twice daily continuously (monotherapy), or 100 mg twice daily for 14 cycles with venetoclax added from cycle 3 (AMPLIFY fixed-duration)","monitoring":"Headache (early), infections, bleeding, atrial fibrillation (lower than ibrutinib), TLS with venetoclax","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Calquence"},"toxicity":[{"event":"Headache","anyGradePct":39,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Calquence","note":"Mostly first weeks; responds to caffeine/paracetamol"},{"event":"Atrial fibrillation","anyGradePct":9,"note":"ELEVATE-RR vs 16% ibrutinib"},{"event":"Neutropenia","grade3PlusPct":23,"note":"With venetoclax (AMPLIFY)"},{"event":"Diarrhoea","anyGradePct":35}],"access":[],"regulatoryEvents":[{"date":"2017-10-31","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy"},{"date":"2019-11-21","type":"approval","region":"US","note":"CLL/SLL"},{"date":"2022-08-03","type":"label-change","region":"US","note":"Tablet formulation co-administrable with acid-reducing agents"},{"date":"2025-01-16","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 7.2 years after it was granted.","indication":"Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy"},{"date":"2026-02-19","type":"approval","region":"US","note":"Acalabrutinib + venetoclax fixed duration (AMPLIFY)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-acalabrutinib-venetoclax-chronic-lymphocytic-leukemia-or-small-lymphocytic-lymphoma"}]},{"id":"acasunlimab","kind":"drug","name":"Acasunlimab","aka":["DuoBody®-PD-L1×4-1BB"],"tldr":"Acasunlimab is an experimental bispecific antibody from Genmab in phase 3 trials for non-small-cell lung cancer, aimed at PD-L1.","summary":"Acasunlimab (GEN1046) is a bispecific antibody developed by Genmab. Its target is PD-L1 (the sponsor names PD-L1 x 4-1BB (CD137)). The sponsor states: Acasunlimab (GEN1046, DuoBody-PD-L1x4-1BB) is a bispecific antibody that co-targets PD-L1 and the costimulatory receptor 4-1BB, given intravenously in combination with pembrolizumab versus docetaxel in PD-L1-positive metastatic NSCLC that progressed after prior anti-PD-(L)1 therapy and chemotherapy. ClinicalTrials.gov describes the intervention as: Acasunlimab will be administered intravenously (IV). It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06635824 (Trial to Evaluate Acasunlimab and Pembrolizumab Combination Superiority Over Standard of Care Docetaxel in Non-Small Cell Lung Cancer (ABBIL1TY NSCLC-06)), in non-small-cell lung cancer. The largest, NCT06635824, plans to enrol 191 participants (actual) with primary completion expected 2027-07-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Acasunlimab","url":"https://clinicaltrials.gov/search?intr=GEN1046"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["pdl1","cd137"],"drugs":[],"companies":["genmab"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06635824","nct05117242"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"GEN1046","modality":"bispecific antibody","mechanism":"Acasunlimab (GEN1046, DuoBody-PD-L1x4-1BB) is a bispecific antibody that co-targets PD-L1 and the costimulatory receptor 4-1BB, given intravenously in combination with pembrolizumab versus docetaxel in PD-L1-positive metastatic NSCLC that progressed after prior anti-PD-(L)1 therapy and chemotherapy.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"acitretin","kind":"drug","name":"Acitretin","aka":["Neotigason"],"tldr":"Acitretin is a vitamin A tablet licensed for severe psoriasis. In people who have had an organ transplant and keep developing skin cancers, it reduces the number of new squamous cell carcinomas while it is taken.","summary":"Acitretin is a second-generation oral retinoid, the active metabolite of etretinate, taken once daily. The FDA approved it in 1996 for severe psoriasis. In oncology it is used off-label as chemoprevention for cutaneous squamous cell carcinoma in organ-transplant recipients and other patients with many keratinocyte cancers: a randomised trial in renal-transplant recipients in 1995 showed fewer new squamous cell carcinomas during treatment, and the effect is lost when the drug is stopped.\n\nIt causes dry skin and lips, raised lipids and liver enzyme changes, and is teratogenic for years after the last dose, so its use is restricted to men and to women who will not become pregnant. Nicotinamide is the other, better tolerated, chemopreventive option named alongside it.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Acitretin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Acitretin"}],"tags":["subtype-drugs-wave"],"related":["nicotinamide"],"cancers":["cutaneous-scc","skin-cancer"],"sections":[],"technologies":["chemoprevention"],"targets":["rara","rxr"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["skin-cancer-after-organ-transplant"],"trials":["nct02050321"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Soriatane","modality":"Oral systemic retinoid","mechanism":"A vitamin A derivative that binds retinoic acid and retinoid X receptors in keratinocytes, normalising their growth and differentiation and slowing the progression of pre-cancerous sun-damaged skin.","approvals":[{"region":"US","year":1996,"indication":"Severe psoriasis (oncology use as chemoprevention of squamous cell carcinoma is off-label)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ryz101","kind":"drug","name":"Actinium-225 DOTATATE","aka":[],"tldr":"An alpha-particle version of Lutathera for neuroendocrine tumours that have stopped responding to the beta version.","summary":"RYZ101 is actinium-225 DOTATATE, a targeted alpha therapy: the same SSTR2-binding peptide as Lutathera but chelating 225Ac, whose alpha decay chain delivers dense, short-range double-strand DNA breaks that beta particles cannot match. Bristol Myers Squibb acquired it with RayzeBio in 2024 for $4.1B. The ACTION-1 phase 3 trial tests it in SSTR-positive GEP-NETs progressing after 177Lu-SSTR therapy, at 10.5 MBq/kg every 8 weeks for 4 cycles, and it is also being combined with chemo-immunotherapy in extensive-stage small-cell lung cancer. Nausea, fatigue and lymphopenia are expected, and renal function is monitored because the kidneys clear the peptide. Whether alpha emission overcomes resistance to beta therapy, and whether 225Ac supply can meet demand, are the open questions. For a newcomer: an alpha-particle upgrade of an approved neuroendocrine radioligand.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Actinium-225 DOTATATE","url":"https://clinicaltrials.gov/search?intr=RYZ101"}],"tags":[],"related":[],"cancers":["neuroendocrine","sclc"],"sections":[],"technologies":["targeted-alpha-therapy"],"targets":["sstr2"],"drugs":[],"companies":["bms","rayzebio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06590857"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"RYZ101","modality":"Targeted alpha therapy","mechanism":"DOTATATE chelating 225Ac; alpha decay chain.","approvals":[],"mechanismSteps":["Radioligand circulates and binds Somatostatin receptor 2 on tumour cells","Ligand is internalised or retained at the membrane","Alpha (225Ac) emissions deposit energy within a short range","Clustered DNA double-strand breaks form in the tumour cell and its neighbours (crossfire)","Cells die; unbound ligand is cleared via the kidneys"],"dosing":{"route":"IV infusion","schedule":"10.5 MBq/kg every 8 weeks for 4 cycles (ACTION-1)","monitoring":"Renal function, blood counts, amino acid co-infusion","source":"https://clinicaltrials.gov/study/NCT05477576"},"toxicity":[{"event":"Nausea"},{"event":"Fatigue"},{"event":"Lymphopenia"},{"event":"Renal toxicity (monitor)"}],"access":[{"country":"US","reimbursement":"Investigational","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-02-26","type":"filing","region":"US","note":"BMS completes RayzeBio acquisition ($4.1B)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"ac225-psma","kind":"drug","name":"Actinium-225 PSMA agents","aka":[],"tldr":"Alpha-emitting PSMA drugs that produce responses even after Pluvicto fails, held back mainly by isotope supply.","summary":"Novartis (225Ac-PSMA-617, phase 3 AcTION), Bayer (225Ac-PSMA-Trillium with albumin binder, PAnTHA phase 1 at ASCO GU 2026), Fusion/AstraZeneca (FPI-2265, 225Ac-PSMA-I&T, phase 3 AlphaBreak), and academic 225Ac-PSMA data from South Africa and Germany. Xerostomia from salivary uptake is dose-limiting.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Actinium-225 PSMA agents","url":"https://clinicaltrials.gov/search?intr=225Ac-PSMA-617"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["targeted-alpha-therapy"],"targets":["psma"],"drugs":[],"companies":["novartis","bayer","fusion-pharma","astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07590934"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"225Ac-PSMA-617, 225Ac-PSMA-I&T, BAY 3563254 (Trillium)","modality":"Targeted alpha therapy","mechanism":"PSMA ligand with 225Ac; four alpha emissions per decay chain.","approvals":[],"mechanismSteps":["Radioligand circulates and binds PSMA on tumour cells","Ligand is internalised or retained at the membrane","Alpha (225Ac) emissions deposit energy within a short range","Clustered DNA double-strand breaks form in the tumour cell and its neighbours (crossfire)","Cells die; unbound ligand is cleared via the kidneys"],"dosing":{"route":"IV infusion","schedule":"Trial regimens ~100 kBq/kg every 6-8 weeks","monitoring":"Salivary gland toxicity (xerostomia), renal function, blood counts","source":"https://clinicaltrials.gov"},"toxicity":[{"event":"Xerostomia","note":"Dose-limiting in academic series"},{"event":"Anaemia"},{"event":"Thrombocytopenia"},{"event":"Renal toxicity"}],"access":[{"country":"US","reimbursement":"Investigational (phase 3)","asOf":"2026-09-06"},{"country":"DE","reimbursement":"Compassionate use at selected centres (Heidelberg and others)","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-06","type":"filing","region":"US","note":"AstraZeneca completes Fusion Pharmaceuticals acquisition; FPI-2265 phase 3 planned","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"adagloxad-simolenin","kind":"drug","name":"Adagloxad simolenin","aka":[],"tldr":"Adagloxad simolenin is OBI Pharma's vaccine against the tumour sugar Globo H, in the phase 3 GLORIA trial as adjuvant treatment for triple-negative breast cancer that expresses the antigen.","summary":"OBI Pharma of Taiwan developed adagloxad simolenin (OBI-822) from Samuel Danishefsky's Globo H synthesis. After a phase 2 trial in metastatic breast cancer showed benefit only in patients who mounted an antibody response, the phase 3 GLORIA trial tests it with the adjuvant OBI-821 in the adjuvant setting for Globo H-positive triple-negative breast cancer at high risk of recurrence.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Adagloxad simolenin","url":"https://clinicaltrials.gov/search?intr=OBI-822"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["tnbc"],"sections":[],"technologies":["dendritic-cell-vaccines"],"targets":[],"drugs":[],"companies":["obi-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03562637"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"OBI-822","modality":"Carbohydrate antigen vaccine (Globo H conjugate) with the adjuvant OBI-821","mechanism":"A Globo H glycan conjugated to a carrier protein, given with a saponin adjuvant, to raise antibodies against Globo H, a sugar antigen on breast and other epithelial cancer cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"adagrasib","kind":"drug","name":"Adagrasib","aka":[],"tldr":"Adagrasib was the second KRAS G12C inhibitor, with a long half-life and brain penetration, and is approved in lung and colorectal cancer.","summary":"Adagrasib is a covalent KRAS G12C inhibitor with a 24-hour half-life and brain penetration, dosed at 600 mg twice daily to keep the target continuously occupied. It was approved in 2022 (accelerated) for previously treated KRAS G12C NSCLC on KRYSTAL-1, with KRYSTAL-12 as the randomised follow-on, and in 2024 with cetuximab for KRAS G12C colorectal cancer on the KRYSTAL-1 combination cohort. Mirati developed it and was acquired by Bristol Myers Squibb in 2024. Gastrointestinal toxicity is frequent (diarrhoea 70%, nausea 69%), with hepatotoxicity (37%) and QT prolongation (20%) also needing monitoring. Activity in brain metastases is the main argument for choosing it over sotorasib, but resistance still develops within months. For a newcomer: the second KRAS G12C pill, distinguished by its long action and brain reach.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Adagrasib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Adagrasib"},{"label":"Bekaii-Saab et al., adagrasib in KRAS G12C solid tumours, KRYSTAL-1 (JCO 2023)","url":"https://doi.org/10.1200/JCO.23.00434"},{"label":"Krazati label (openFDA): indications","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22KRAZATI%22"},{"label":"FDA oncology approval notifications","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications"}],"tags":[],"related":["kras-g12c"],"cancers":["nsclc","colorectal","pancreatic","kras-g12c-colorectal","kras-g12c-nsclc","kras-g12c-pdac"],"sections":[],"technologies":["kras-inhibitors"],"targets":["kras"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04613596","nct05853575","nct05609578","nct07415031","nct06875310","nct03785249"],"people":[],"bottlenecks":[],"keyPapers":["paper-krystal-12-plain-language-summary-future-oncol-2026","paper-krystal-1-adagrasib-kras-g12c-solid-tumours-jco-2023","paper-schirripa-kras-g12c-metastatic-colorectal-clin-colorectal-cancer-2020"],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: the Krazati label (effective 25 March 2026) covers KRAS G12C lung and colorectal cancer only; the KRYSTAL-1 phase 2 cohort of other solid tumours reported responses in 7 of 21 pancreatic cancers (33.3 percent), a median duration of response of 5.3 months and progression-free survival of 7.4 months across the cohort (JCO 2023), which is the basis of the NCCN listing. No NICE or EMA pancreatic indication.","Pancreatic ductal adenocarcinoma: KRYSTAL-1's solid-tumour cohort gave 7 responses among 21 KRAS G12C pancreatic patients (33.3%), with a median duration of response of 5.3 months across tumour types (Bekaii-Saab 2023).","Colorectal cancer biomarkers: KRAS G12C, with cetuximab rather than alone, for the same adaptive EGFR reactivation reason (KRYSTAL-1)."],"brand":"Krazati","modality":"Small-molecule inhibitor (KRAS G12C)","mechanism":"Covalent KRAS G12C inhibitor, 24-hour half-life.","approvals":[{"region":"US","year":2022,"indication":"KRAS G12C NSCLC (accelerated)"},{"region":"US","year":2024,"indication":"KRAS G12C colorectal cancer with cetuximab"},{"region":"EU","year":2024,"indication":"KRAS G12C NSCLC after ≥1 line; 5 Jan 2024 (conditional)","note":"Conditional marketing authorisation"},{"region":"US","year":2024,"indication":"KRAS G12C-mutated locally advanced or metastatic colorectal cancer after fluoropyrimidine, oxaliplatin and irinotecan chemotherapy, with cetuximab","note":"Accelerated approval on 21 June 2024 on the KRYSTAL-1 colorectal cohort; KRYSTAL-10 is the confirmatory randomised trial. No NICE recommendation for colorectal cancer at September 2026."}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of KRAS G12C","Phosphorylation of downstream substrates stops","Covalent inhibitor with a 24-hour half-life and CNS penetration","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"600 mg twice daily; with cetuximab in colorectal cancer","modifications":"Reduce to 400 mg BID then 600 mg once daily for GI, hepatic, or QTc toxicity","monitoring":"LFTs monthly for 3 months; ECG and electrolytes; renal function","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01"},"toxicity":[{"event":"Diarrhoea","anyGradePct":70,"grade3PlusPct":0.9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"Nausea","anyGradePct":69,"grade3PlusPct":4.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"Fatigue","anyGradePct":59,"grade3PlusPct":7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"Vomiting","anyGradePct":56,"grade3PlusPct":0.9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"Musculoskeletal pain","anyGradePct":41,"grade3PlusPct":7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"Hepatotoxicity","anyGradePct":37,"grade3PlusPct":10,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"Renal impairment","anyGradePct":36,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"Dyspnoea","anyGradePct":35,"grade3PlusPct":10,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"Oedema","anyGradePct":32,"grade3PlusPct":0,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"},{"event":"QT prolongation","anyGradePct":20,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0b8bf078-34c2-4f45-9012-38a8ac082b01","note":"KRYSTAL-1 NSCLC"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.bmsaccesssupport.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: appraisal for KRAS G12C NSCLC (2025)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-06","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-12-12","type":"accelerated-approval","region":"US","note":"Accelerated approval, KRAS G12C NSCLC after ≥1 therapy The confirmatory requirement was still open 3.8 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy."},{"date":"2024-06-01","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-adagrasib-cetuximab-kras-g12c-mutated-colorectal-cancer","indication":"In combination with cetuximab for the treatment of adult patients with KRAS G12C mutated locally advanced or metastatic colorectal cancer who have been previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy."},{"date":"2024-06-21","type":"approval","region":"US","note":"KRAS G12C colorectal cancer with cetuximab (accelerated)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-06","type":"approval","region":"US","note":"Full approval in NSCLC (KRYSTAL-12)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-09-01","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 2.3 years after its accelerated approval.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-adagrasib-cetuximab-kras-g12c-mutated-colorectal-cancer","indication":"In combination with cetuximab for the treatment of adult patients with KRAS G12C mutated locally advanced or metastatic colorectal cancer who have been previously treated with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy."}]},{"id":"adavosertib","kind":"drug","name":"Adavosertib","aka":[],"tldr":"Adavosertib is an oral serine/threonine kinase inhibitor from AstraZeneca, in registered phase 2 trials for triple-negative breast cancer.","summary":"Adavosertib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by AstraZeneca, in triple-negative breast cancer. A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Adavosertib","url":"https://clinicaltrials.gov/search?intr=Adavosertib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["tnbc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03330847"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral serine/threonine kinase inhibitor","mechanism":"A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"adebrelimab","kind":"drug","name":"Adebrelimab","aka":[],"tldr":"Adebrelimab is Hengrui's PD-L1 antibody, approved in China for first-line extensive-stage small cell lung cancer on the CAPSTONE-1 trial.","summary":"CAPSTONE-1 randomised 462 patients with extensive-stage small cell lung cancer to adebrelimab or placebo with carboplatin and etoposide: median overall survival 15.3 versus 12.8 months (hazard ratio 0.72), among the largest survival gains reported for a PD-L1 antibody in this disease. The NMPA approved the combination in March 2023. Hengrui is testing adebrelimab in limited-stage disease after chemoradiotherapy and with its own ADCs.","status":"approved","asOf":"2026-09-10","links":[{"label":"Jiangsu Hengrui Pharmaceuticals","url":"https://www.hengrui.com/en/"},{"label":"CAPSTONE-1 (Lancet Oncol 2022)","url":"https://doi.org/10.1016/S1470-2045(22)00224-8"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["sclc","extensive-stage-sclc"],"sections":[],"technologies":["checkpoint-inhibitor","monoclonal-antibody"],"targets":["pdl1"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":["pd1-checkpoint"],"terms":["limited-extensive-stage"],"trials":["capstone-1","nct07168200","nct06589778","nct07059221","nct06512051","nct07071714","nct07229729","nct06754930","nct06840002","nct06222879","nct06465563","nct06474455","nct07229586","nct06247956","nct07111832","nct06778031","nct05482568","nct07268040","nct06639347","nct07110571","nct06385678","nct07679360","nct06915142","nct07028281","nct07393542","nct06417554","nct07522151","nct07241767","nct06879145","nct06474468","nct06703177","nct05671822","nct07296341","nct07073534"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Arelili","code":"SHR-1316","modality":"Monoclonal antibody (anti-PD-L1, humanised IgG4)","mechanism":"Humanised anti-PD-L1 IgG4 antibody blocking PD-L1 binding to PD-1 and B7-1.","approvals":[{"region":"China","year":2023,"indication":"First-line extensive-stage small cell lung cancer with carboplatin and etoposide (CAPSTONE-1)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"adg126","kind":"drug","name":"ADG126","aka":[],"tldr":"ADG126 is an experimental monoclonal antibody from Adagene in phase 2 trials for hepatocellular carcinoma, aimed at CTLA-4.","summary":"ADG126 is a monoclonal antibody developed by Adagene and Sanofi. Its target is CTLA-4 (the sponsor names CTLA-4). The sponsor states: A fully human anti-CTLA-4 monoclonal antibody that specifically binds to human CTLA-4. ClinicalTrials.gov describes the intervention as: ADG126 is an anti-CTLA-4 fully human monoclonal antibody that specifically binds to human CTLA-4. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in hepatocellular carcinoma. The largest, NCT05584670, plans to enrol 542 participants with primary completion expected 2027-01-19. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ADG126","url":"https://clinicaltrials.gov/search?intr=ADG126"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hcc"],"sections":[],"technologies":[],"targets":["ctla4"],"drugs":[],"companies":["sanofi","roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04524871","nct05405595","nct05584670"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"A fully human anti-CTLA-4 monoclonal antibody that specifically binds to human CTLA-4.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"adi-270","kind":"drug","name":"ADI-270","aka":[],"tldr":"ADI-270 is an experimental CAR-T cell therapy from Adicet Therapeutics in phase 2 trials for renal cell carcinoma, aimed at CD70.","summary":"ADI-270 is a CAR-T cell therapy developed by Adicet Therapeutics. Its target is CD70 (the sponsor names CD70). The sponsor states: An armoured, allogeneic 'off-the-shelf' CAR gamma-delta (CAR-γδ) T-cell therapy directed against CD70-expressing cells, with reported CAR-directed and CAR-independent anti-tumour activity against solid and haematologic malignancies. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in renal cell carcinoma. The largest, NCT06480565, plans to enrol 60 participants with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ADI-270","url":"https://clinicaltrials.gov/search?intr=ADI-270"},{"label":"Sponsor pipeline page","url":"https://adicetbio.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["rcc"],"sections":[],"technologies":[],"targets":["cd70"],"drugs":[],"companies":["adicet-bio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06480565"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"An armoured, allogeneic 'off-the-shelf' CAR gamma-delta (CAR-γδ) T-cell therapy directed against CD70-expressing cells, with reported CAR-directed and CAR-independent anti-tumour activity against solid and haematologic malignancies.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"adi-peg20","kind":"drug","name":"ADI-PEG20","aka":["pegargiminase"],"tldr":"ADI-PEG20 is an experimental investigational agent whose form is not stated in the registry from Polaris in phase 3 trials for hepatocellular carcinoma, with its target not yet stated publicly.","summary":"ADI-PEG20 (PEG20) is an investigational agent whose form is not stated in the registry developed by Polaris. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05317819 (Study of ADI-PEG 20 Versus Placebo in Subjects With High Arginine Level and Unresectable Hepatocellular Carcinoma), in hepatocellular carcinoma. The largest, NCT05317819, plans to enrol 300 participants with primary completion expected 2028-09. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ADI-PEG20","url":"https://clinicaltrials.gov/search?intr=PEG20"},{"label":"Sponsor page","url":"https://www.polarispharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hcc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["polaris-group"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05317819"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PEG20","modality":"PEGylated enzyme (pegargiminase, arginine deiminase; INN stem -ase; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"adu-s100","kind":"drug","name":"ADU-S100 (MIW815)","aka":[],"tldr":"ADU-S100 was the first STING agonist in the clinic. Injected directly into tumours, it produced almost no responses, alone or with checkpoint blockade.","summary":"A cyclic dinucleotide STING agonist from Aduro (partnered with Novartis). Phase 1 monotherapy and combinations with spartalizumab or ipilimumab produced single-digit response rates; Novartis returned rights in 2019 and Aduro discontinued the programme in 2020. Merck's MK-1454 followed the same path. The pathway remains important (it mediates immune effects of radiation and ADCs), and systemic and antibody-conjugated STING agonists continue.\n\nLesson: intratumoural delivery to one lesion rarely produces systemic immunity in humans as it does in mice; pharmacology (rapid clearance, dosing) matters as much as the target.","status":"withdrawn","asOf":"2026-09-06","links":[{"label":"ADU-S100 phase 1 (Cancer Discov 2023)","url":"https://aacrjournals.org/cancerdiscovery/article/13/5/1117/725648"}],"tags":["failure","lesson:wrong-drug"],"related":[],"cancers":["melanoma","head-and-neck"],"sections":[],"technologies":["sting-agonist"],"targets":[],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":["cgas-sting"],"terms":[],"trials":["nct03937141"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ADU-S100","modality":"Small molecule (STING agonist, intratumoural)","mechanism":"Synthetic cyclic dinucleotide activating STING → TBK1 → IRF3 → type I interferon.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"afamitresgene-autoleucel","kind":"drug","name":"Afamitresgene autoleucel","aka":[],"tldr":"Afamitresgene autoleucel was the first engineered T-cell receptor therapy approved for a solid tumour, synovial sarcoma.","summary":"Afamitresgene autoleucel (afami-cel) is a TCR-T therapy: patient T cells are engineered with an affinity-enhanced T-cell receptor that recognises the MAGE-A4 230-239 peptide on HLA-A*02, so eligibility requires both HLA typing and tumour MAGE-A4 testing. It received accelerated approval in August 2024 for advanced MAGE-A4-positive, HLA-A*02-positive synovial sarcoma on SPEARHEAD-1 (ORR about 39%, median duration of response about 12 months), and in Q2 2026 the label was extended to patients aged 12 and over. Treatment is a single infusion of 2.68 to 10 x 10^9 TCR-positive cells after fludarabine and cyclophosphamide; cytokine release syndrome and prolonged cytopenias are the main risks. Whether responses are durable enough to change survival is open. For a newcomer: the first engineered T-cell receptor therapy approved for a solid tumour.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Afamitresgene_autoleucel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Afamitresgene%20autoleucel"}],"tags":[],"related":["hla-a-02-01"],"cancers":["sarcoma","synovial-sarcoma"],"sections":[],"technologies":["tcr-t"],"targets":["mage-a4","hla-a"],"drugs":[],"companies":["adaptimmune"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05642455","nct04044768"],"people":[],"bottlenecks":[],"keyPapers":["paper-spearhead-1-lancet-2024"],"journals":[],"dependsOn":[],"notes":[],"brand":"Tecelra","code":"afami-cel","modality":"TCR-T (MAGE-A4)","mechanism":"Affinity-enhanced TCR against MAGE-A4 230-239/HLA-A*02.","approvals":[{"region":"US","year":2024,"indication":"Advanced MAGE-A4+ synovial sarcoma, HLA-A*02+ (age ≥12 from 2026)"}],"mechanismSteps":["Patient's T cells are engineered to express an affinity-enhanced TCR recognising MAGE-A4 peptide on HLA-A*02","Cells are expanded and infused after lymphodepletion","TCR-T cells recognise MAGE-A4-derived peptide displayed by tumour cells","Cytotoxic granules kill the tumour cell","Persistence provides ongoing surveillance"],"dosing":{"route":"Single IV infusion after lymphodepletion","schedule":"2.68 × 10⁹ to 10 × 10⁹ MAGE-A4 TCR+ T cells; fludarabine 30 mg/m² ×4 and cyclophosphamide 600 mg/m² ×3","modifications":"Tocilizumab/steroids for CRS","monitoring":"Daily monitoring for 7 days; cytopenias; infections","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Cytokine release syndrome"},{"event":"Nausea"},{"event":"Vomiting"},{"event":"Fatigue"},{"event":"Infections"},{"event":"Prolonged cytopenias"}],"access":[{"country":"US","listPrice":"$727,000 per infusion (list price at launch, 2024)","reimbursement":"Medicare Part B (inpatient/outpatient); commercial per plan","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-08-01","type":"approval","region":"US","note":"Accelerated approval, MAGE-A4+ HLA-A*02+ advanced synovial sarcoma (SPEARHEAD-1): first TCR-T for a solid tumour","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-08-02","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 2.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-afamitresgene-autoleucel-unresectable-or-metastatic-synovial-sarcoma","indication":"Treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen as determined by FDA-approved or cleared companion diagnostic devices."},{"date":"2025-08","type":"filing","region":"US","note":"US rights sold by Adaptimmune to US WorldMeds","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q2","type":"label-change","region":"US","note":"Label extended to patients aged 12 and older","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"afatinib","kind":"drug","name":"Afatinib","aka":[],"tldr":"Afatinib (Gilotrif) is a second-generation pill that binds EGFR, HER2 and HER4 irreversibly, approved in 2013 for EGFR-mutant lung cancer. Its lasting value is activity against the uncommon EGFR mutations G719X, L861Q and S768I, approved in 2018, because osimertinib has displaced it for common mutations and its wild-type EGFR binding causes more rash and diarrhoea.","summary":"Afatinib is a second-generation, irreversible pan-ErbB tyrosine kinase inhibitor that covalently binds EGFR, HER2 and HER4. LUX-Lung 3 and LUX-Lung 6 (2013) showed superiority over chemotherapy in EGFR-mutant NSCLC, leading to US approval in 2013 for first-line exon 19 deletion or L858R disease, and LUX-Lung 7 compared it with gefitinib. Its distinctive value is activity against uncommon EGFR mutations, G719X, L861Q and S768I, for which it was approved in 2018, and LUX-Lung 8 also supported approval in 2016 for squamous NSCLC after platinum. Because it also hits wild-type EGFR irreversibly, it causes more rash, diarrhoea and paronychia than first-generation agents, and dose reduction is common. Osimertinib has displaced it for common mutations. For a newcomer, afatinib is the EGFR pill to remember for the rarer mutations.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Afatinib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=afatinib"}],"tags":["gap-fill"],"related":[],"cancers":["nsclc","egfr-mutant-nsclc","her2-mutant-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr","her2"],"drugs":[],"companies":["boehringer-ingelheim"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03785249","lux-lung-3"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Afatinib holds a distinct FDA indication, granted in January 2018, for non-resistant uncommon EGFR mutations (S768I, L861Q and G719X), based on a pooled analysis of LUX-Lung 2, 3 and 6. It is the reason a patient with one of those mutations may be offered afatinib rather than osimertinib. There is no NICE appraisal of afatinib for the uncommon mutations."],"brand":"Gilotrif / Giotrif","modality":"Small-molecule pan-ErbB TKI (second generation, irreversible)","mechanism":"Covalent inhibitor of EGFR, HER2 and HER4 kinases.","approvals":[{"region":"US","year":2013,"indication":"First-line metastatic NSCLC with EGFR exon 19 del or L858R"},{"region":"US","year":2016,"indication":"Squamous NSCLC after platinum"},{"region":"US","year":2018,"indication":"Uncommon EGFR mutations (S768I, L861Q, G719X)"},{"region":"EU","year":2013,"indication":"EU brand Giotrif"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"afirma","kind":"drug","name":"Afirma Genomic Sequencing Classifier","aka":[],"tldr":"A gene test on the needle sample from a thyroid lump that can safely call an 'uncertain' result benign and spare an operation.","summary":"Afirma (Veracyte) was the first molecular test to change thyroid practice: the original Gene Expression Classifier (2011) and the Genomic Sequencing Classifier that replaced it in 2017 are used on Bethesda III and IV nodules, where cytology is indeterminate and 20-30% of nodules prove malignant at surgery. In the blinded multicentre validation of the GSC (JAMA Surgery 2018) sensitivity was 91% and specificity 68%, giving a negative predictive value around 96%, so about half of indeterminate nodules can be observed rather than removed. It is a laboratory-developed test covered by Medicare and most US insurers; ThyroSeq v3 is the main alternative.","status":"established","asOf":"2026-09-10","links":[{"label":"Afirma GSC validation (JAMA Surg 2018)","url":"https://doi.org/10.1001/jamasurg.2018.1153"},{"label":"Veracyte: Afirma","url":"https://www.afirma.com"}],"tags":["test"],"related":[],"cancers":["thyroid"],"sections":[],"technologies":["thyroid-fna-molecular","rna-seq"],"targets":[],"drugs":[],"companies":["veracyte"],"institutions":[],"pathways":[],"terms":["sensitivity-specificity"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-patel-jama-surg"],"journals":[],"dependsOn":[],"notes":["What a result means: 'benign' by Afirma carries about the same cancer risk as a benign cytology result and can be followed with ultrasound; 'suspicious' means surgery is usually advised."],"brand":"Afirma GSC","modality":"RNA-sequencing rule-out classifier test (indeterminate thyroid nodules)","mechanism":"Whole-transcriptome RNA sequencing of a fine-needle aspirate, with a machine-learned classifier trained to call cytologically indeterminate nodules benign or suspicious, plus reporting of BRAF, RET and other variants.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aglatimagene-besadenovec","kind":"drug","name":"Aglatimagene besadenovec","aka":["AdV-tk"],"tldr":"Aglatimagene besadenovec is an experimental gene therapy from Candel Therapeutics in phase 3 trials for non-small-cell lung cancer and prostate cancer, with its target not yet stated publicly.","summary":"Aglatimagene besadenovec (CAN-2409) is a gene therapy developed by Candel Therapeutics. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. The sponsor states: An adenovirus-based immunotherapy (AdV-tk/CAN-2409) that blocks DNA synthesis in dividing tumour cells, causing tumour cell death, while adenovirus capsid proteins attract immune cells to the tumour microenvironment so activated immune cells can then attack distant metastases. ClinicalTrials.gov describes the intervention as: Aglatimagene besadenovec will be delivered to the prostate via trans-rectal ultrasound guided injection followed by 14 days of oral prodrug, valacyclovir. The second aglatimagene besadenovec injection will be 2-3 weeks after the first follo. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07660094 (Aglatimagene Besadenovec + Prodrug and Pembrolizumab vs Docetaxel for Stage IV Non-Squamous NSCLC Progressing on Pembrolizumab (AURORA)), in non-small-cell lung cancer and prostate cancer. The largest, NCT07660094, plans to enrol 500 participants with primary completion expected 2031-10-22. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Aglatimagene besadenovec","url":"https://clinicaltrials.gov/search?intr=CAN-2409"},{"label":"Sponsor pipeline page","url":"https://www.candeltx.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","prostate"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["candel-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["aurora","nct01436968","nct02768363","nct07332000","nct04495153"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CAN-2409","modality":"gene therapy","mechanism":"An adenovirus-based immunotherapy (AdV-tk/CAN-2409) that blocks DNA synthesis in dividing tumour cells, causing tumour cell death, while adenovirus capsid proteins attract immune cells to the tumour microenvironment so activated immune cells can then attack distant metastases.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ai-081","kind":"drug","name":"AI-081","aka":["Bispecific anti-PD-1/VEGF"],"tldr":"AI-081 is an experimental bispecific antibody from OncoC4 in phase 2 trials, aimed at PD-1 and VEGF / VEGFR.","summary":"AI-081 is a bispecific antibody developed by OncoC4. Its targets are PD-1 and VEGF / VEGFR (the sponsor names PD-1/VEGF). The sponsor states: dual checkpoint and angiogenesis inhibition. ClinicalTrials.gov describes the intervention as: AI-081 is a humanized monoclonal antibody targeting PD-1 and VEGF. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT06635785, plans to enrol 387 participants with primary completion expected 2027-06-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AI-081","url":"https://clinicaltrials.gov/search?intr=AI-081"},{"label":"Sponsor page","url":"https://www.oncoc4.com/en/pipeline/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["pd1","vegf"],"drugs":[],"companies":["oncoc4"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06635785"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"dual checkpoint and angiogenesis inhibition.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ak117","kind":"drug","name":"AK117","aka":[],"tldr":"AK117 is an experimental investigational agent whose form is not stated in the registry from Akeso in phase 3 trials for head and neck squamous cell carcinoma, colorectal cancer and non-small-cell lung cancer, with its target not yet stated publicly.","summary":"AK117 is an investigational agent whose form is not stated in the registry developed by Akeso. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: AK117 via intravenous (IV) infusion until disease progression or unacceptable toxicity. It is the investigational product in 6 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06601335 (A Phase 3 Study of AK112 Plus AK117 Versus Pembrolizumab in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)), in head and neck squamous cell carcinoma, colorectal cancer, non-small-cell lung cancer, acute myeloid leukaemia and triple-negative breast cancer. The largest, NCT06601335, plans to enrol 510 participants with primary completion expected 2027-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AK117","url":"https://clinicaltrials.gov/search?intr=AK117"},{"label":"NCI Drug Dictionary: ligufalimab","url":"https://www.cancer.gov/publications/dictionaries/cancer-drug/def/ligufalimab"},{"label":"ClinicalTrials.gov NCT04980885","url":"https://clinicaltrials.gov/study/NCT04980885"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["head-and-neck","colorectal","nsclc","aml","tnbc","sclc"],"sections":[],"technologies":[],"targets":["cd47"],"drugs":[],"companies":["akeso"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06601335","nct07245446","nct05382442","nct05229497","nct05227664","nct04980885"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["AK117 is ligufalimab, a humanised IgG4 monoclonal antibody against CD47 that blocks the CD47 to SIRP-alpha signal tumour cells use to avoid being eaten by macrophages (NCI Drug Dictionary; ClinicalTrials.gov NCT04980885 is titled \"A Trial of AK117 (Anti-CD47 Antibody) in Patients With Acute Myeloid Leukemia\")."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody (AK117 is ligufalimab; INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ak119","kind":"drug","name":"AK119","aka":[],"tldr":"AK119 is an experimental monoclonal antibody from Akeso in phase 2 trials for colorectal cancer, aimed at CD73 / adenosine axis.","summary":"AK119 is a monoclonal antibody developed by Akeso. Its target is CD73 / adenosine axis (the sponsor names CD73). The sponsor states: AK119 is an anti-CD73 monoclonal antibody, studied in combination with AK112 for advanced solid tumours and colorectal cancer. ClinicalTrials.gov describes the intervention as: AK119 is an anti-CD73 monoclonal antibody. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in colorectal cancer. The largest, NCT05846867, plans to enrol 170 participants with primary completion was scheduled for 2026-05-08 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AK119","url":"https://clinicaltrials.gov/search?intr=AK119"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["cd73-adenosine"],"drugs":[],"companies":["akeso"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05846867","nct05689853"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"AK119 is an anti-CD73 monoclonal antibody, studied in combination with AK112 for advanced solid tumours and colorectal cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ak146d1","kind":"drug","name":"AK146D1","aka":[],"tldr":"AK146D1 is Akeso's Nectin-4 × TROP2 bispecific ADC, combining the two most validated ADC addresses in one molecule.","summary":"AK146D1 is Akeso's bispecific antibody-drug conjugate that binds both Nectin-4 and TROP2, the two most clinically validated ADC targets, and delivers a topoisomerase I inhibitor through a cleavable linker. The rationale is complementary expression across urothelial, breast and lung cancers and potentially higher internalisation when two receptors are engaged on the same cell. It entered the clinic in 2025 in a first-in-human phase 1 dose-escalation study after IND clearance in China, alongside Avenzo's AVZO-103 in the same target pair. No efficacy or toxicity data have yet been reported, so the programme is very early. Whether dual targeting improves on single-target ADCs such as enfortumab vedotin and sacituzumab govitecan, or simply adds toxicity, is the central question. For a newcomer: it combines two proven ADC addresses in one molecule, and the clinic has yet to show if that helps.","status":"phase-1","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov NCT06929663: AK146D1 in advanced solid tumours (phase 1)","url":"https://clinicaltrials.gov/study/NCT06929663"},{"label":"Akeso","url":"https://www.akesobio.com/en/"}],"tags":[],"related":[],"cancers":["urothelial","tnbc","nsclc"],"sections":[],"technologies":["bispecific-adc"],"targets":["nectin4","trop2"],"drugs":[],"companies":["akeso"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07669779","nct07591090"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Bispecific ADC","payload":"TOP1 inhibitor","linker":"Cleavable","mechanism":"Bispecific Nectin-4/TROP2 antibody with TOP1 payload.","approvals":[],"mechanismSteps":["Antibody binds Nectin-4 and TROP2 (bispecific) on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","TOP1 inhibitor is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"Phase 1 dose escalation","source":"https://clinicaltrials.gov"},"toxicity":[],"access":[{"country":"CN","reimbursement":"Investigational","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2025-03","type":"filing","region":"China","note":"IND cleared; first-in-human phase 1 started","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"al2846","kind":"drug","name":"AL2846","aka":[],"tldr":"AL2846 is an experimental small-molecule drug from Chia Tai Tianqing Pharmaceutical in phase 3 trials for thyroid cancer, aimed at MET and RET.","summary":"AL2846 is a small-molecule drug developed by Chia Tai Tianqing Pharmaceutical. Its targets are MET, RET and KIT (the sponsor names c-MET, c-KIT, VEGFR1, RET). The sponsor states: A multi-target tyrosine kinase inhibitor capsule with significant inhibitory effects on c-MET, c-KIT, VEGFR1 and RET. ClinicalTrials.gov describes the intervention as: AL2846 Capsule is a multi - target tyrosine kinase inhibitor, which has significant inhibitory effects on c-Mesenchymal-epithelial transition factor (c - MET), stem cell factor receptor (c - KIT), VEGFR1 and Ret Proto-Oncogene (RET). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06860971 (A Study of AL2846 Capsule Versus Placebo in the Treatment of Advanced Radioiodine-Refractory Differentiated Thyroid Carcinoma), in thyroid cancer. The largest, NCT06860971, plans to enrol 144 participants with primary completion expected 2027-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AL2846","url":"https://clinicaltrials.gov/search?intr=AL2846"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["thyroid"],"sections":[],"technologies":[],"targets":["met","ret","kit"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06860971"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"A multi-target tyrosine kinase inhibitor capsule with significant inhibitory effects on c-MET, c-KIT, VEGFR1 and RET.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"al8326","kind":"drug","name":"AL8326","aka":[],"tldr":"AL8326 is an experimental small-molecule drug from Advenchen Pharmaceuticals in phase 3 trials for small-cell lung cancer, non-small-cell lung cancer and renal cell carcinoma, with its target not yet stated publicly.","summary":"AL8326 is a small-molecule drug developed by Advenchen Pharmaceuticals and Advenchen Laboratories Nanjing. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: 10mg/tablet, Oral administration, once daily. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06247605 (A Phase IIII Study of AL8326 in Small Cell Lung Cancer), in small-cell lung cancer, non-small-cell lung cancer and renal cell carcinoma. The largest, NCT06247605, plans to enrol 243 participants with primary completion expected 2028-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AL8326","url":"https://clinicaltrials.gov/search?intr=AL8326"},{"label":"Sponsor page","url":"https://www.advenchen.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["sclc","nsclc","rcc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["advenchen-laboratories"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06247605"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aldesleukin","kind":"drug","name":"Aldesleukin (high-dose IL-2)","aka":["Interleukin-2","IL-2","Recombinant interleukin-2"],"tldr":"High-dose interleukin-2 was the first immunotherapy to cure a small fraction of patients with metastatic melanoma and kidney cancer, at the cost of ICU-level toxicity; today it mainly supports TIL therapy.","summary":"Approved 1992 (RCC) and 1998 (melanoma) on ~15% response with ~5-7% durable complete responses (Rosenberg). Capillary leak, hypotension and multi-organ effects confine it to specialised centres. Now used as a short course after lifileucel (TIL) infusion; engineered IL-2 variants (bempegaldesleukin) failed in phase 3.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Aldesleukin","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=aldesleukin"}],"tags":["gap-fill","historic"],"related":[],"cancers":["rcc","melanoma"],"sections":[],"technologies":["cytokine-therapy","til-therapy"],"targets":[],"drugs":[],"companies":["iovance"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06119685","nct07681596"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Proleukin","modality":"Recombinant interleukin-2 (cytokine)","mechanism":"Activates IL-2 receptors on T and NK cells, driving proliferation and cytotoxicity; high doses cause capillary leak.","approvals":[{"region":"US","year":1992,"indication":"Metastatic renal cell carcinoma"},{"region":"US","year":1998,"indication":"Metastatic melanoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aldoxorubicin","kind":"drug","name":"Aldoxorubicin","aka":["INNO-206","DOXO-EMCH","Aldox"],"tldr":"Aldoxorubicin is doxorubicin, the workhorse chemotherapy nicknamed the red devil, attached to a linker that hitches it to the body's own albumin so more reaches the tumour and less reaches the heart; it beat doxorubicin in a randomised phase 2 sarcoma trial but missed the primary endpoint of its phase 3, and a new company is now trying to finish its development.","summary":"Aldoxorubicin was invented by Felix Kratz at the Tumor Biology Center in Freiburg and developed by CytRx. In a randomised phase 2b trial of 140 patients with previously untreated advanced soft tissue sarcoma, it improved progression-free survival over doxorubicin (median 5.6 versus 2.7 months) and allowed cumulative doxorubicin-equivalent doses far above the usual cardiac ceiling. The 433-patient phase 3 trial in relapsed soft tissue sarcoma compared it with investigator's choice of dacarbazine, pazopanib, gemcitabine with docetaxel, doxorubicin or ifosfamide and did not meet its primary progression-free survival endpoint, and CytRx licensed the drug to ImmunityBio in 2017, which has tested it with its IL-15 agonist in pancreatic cancer. Gemini Therapeutics, formed after the GitLab co-founder Sid Sijbrandij was treated with doxorubicin for osteosarcoma, took up the programme and presented integrated analyses at ASCO 2026 reporting higher tumour exposure than doxorubicin and better preserved cardiac function across the randomised sarcoma studies. It is not approved anywhere.","status":"phase-3","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Aldoxorubicin","links":[{"label":"Chawla et al., first-line aldoxorubicin vs doxorubicin in advanced soft tissue sarcoma, phase 2b (JAMA Oncology 2015)","url":"https://doi.org/10.1001/jamaoncol.2015.3101"},{"label":"Phase 3 trial NCT02049905 on ClinicalTrials.gov","url":"https://clinicaltrials.gov/study/NCT02049905"},{"label":"Gemini Therapeutics: aldoxorubicin","url":"https://www.geminithera.com/aldoxorubicin.html"},{"label":"Cardiac safety of aldoxorubicin vs doxorubicin, integrated analysis (ASCO 2026 abstract 11565)","url":"https://doi.org/10.1200/JCO.2026.44.16_suppl.11565"}],"tags":["gap-fill","owner-request"],"related":[],"cancers":["sarcoma","osteosarcoma","pancreatic"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["top2a"],"drugs":["doxorubicin"],"companies":["gemini-therapeutics","immunitybio"],"institutions":[],"pathways":[],"terms":[],"trials":["aldoxorubicin-phase-3-sts","nct04390399"],"people":[],"bottlenecks":[],"keyPapers":["paper-chawla-jama-oncol"],"journals":[],"dependsOn":[],"notes":[],"modality":"Albumin-binding small-molecule prodrug of doxorubicin, intravenous","mechanism":"Doxorubicin joined through an acid-sensitive hydrazone linker to a maleimide that bonds covalently to circulating albumin within minutes of injection; albumin carries the drug into tumours, where the acidic microenvironment and lysosomes cleave the linker and release doxorubicin, keeping free doxorubicin in the blood and heart low.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"alectinib","kind":"drug","name":"Alectinib","aka":[],"tldr":"Alectinib is a well-tolerated ALK pill, standard first line for years and, since 2024, the first targeted therapy given after surgery for ALK-positive lung cancer.","summary":"Alectinib is an ATP-competitive second-generation ALK inhibitor that penetrates the central nervous system and spares ROS1. It is the long-standing first-line standard for ALK-positive non-small-cell lung cancer and, since 2024, the first targeted therapy given after surgery for resected ALK-positive disease. ALEX (2017) showed PFS of 34.8 versus 10.9 months against crizotinib with strong CNS control, and ALINA (2024) showed adjuvant alectinib cut recurrence by 76% versus chemotherapy (DFS HR 0.24 in stage II to IIIA). Its first-line position is being challenged by lorlatinib (CROWN) and, in the ALKAZAR trial, by neladalkib, so the open question is which ALK inhibitor to use first. For a newcomer, alectinib is the well-tolerated ALK pill that most patients start on and that now also protects against relapse after an operation.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Alectinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Alectinib"},{"label":"NICE TA536: alectinib for untreated ALK-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta536"}],"tags":[],"related":["alk-fusion"],"cancers":["nsclc","alk-positive-nsclc","resectable-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["alk"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["alina","nct05170204","nct06765109","nct03178552","nct04302025","nct04774718","nct05987956","alex","alesia","profile-1014"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Alecensa","modality":"Small-molecule kinase inhibitor (ALK)","mechanism":"ATP-competitive second-generation ALK inhibitor, CNS-penetrant, spares ROS1.","approvals":[{"region":"US","year":2015,"indication":"ALK+ NSCLC after crizotinib"},{"region":"US","year":2017,"indication":"First-line ALK+ NSCLC"},{"region":"US","year":2024,"indication":"Adjuvant ALK+ NSCLC after resection (stage IB ≥4 cm to IIIA)"},{"region":"England (NICE)","year":2018,"indication":"Untreated ALK-positive advanced non-small-cell lung cancer","note":"TA536, published 8 August 2018, within its marketing authorisation subject to the commercial arrangement (ALEX)."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2015-12-11","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"ALK-positive metastatic NSCLC that progressed on or is intolerant to crizotinib"},{"date":"2017-11-06","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2015 converted to traditional approval 1.9 years after it was granted.","indication":"ALK-positive metastatic NSCLC that progressed on or is intolerant to crizotinib"}]},{"id":"alemtuzumab","kind":"drug","name":"Alemtuzumab","aka":["MabCampath","Lemtrada (multiple sclerosis formulation)"],"tldr":"Alemtuzumab (Campath) is an antibody that wipes out lymphocytes carrying the CD52 marker. It is approved for chronic lymphocytic leukaemia and is supplied in the US through a restricted distribution programme.","summary":"Alemtuzumab is a CD52-directed cytolytic antibody approved by the FDA in 2001 (accelerated) for fludarabine-refractory B-cell CLL, with full approval as a single agent for B-CLL after the CAM307 randomised trial against chlorambucil in previously untreated patients (297 randomised; longer progression-free survival on alemtuzumab). Profound lymphopenia brings cytomegalovirus reactivation and opportunistic infection, so prophylaxis and monitoring are mandatory. In the EU, MabCampath was authorised in 2001 and withdrawn in 2012 when the sponsor repositioned the molecule as Lemtrada for multiple sclerosis; Campath remains available for CLL through the US Campath Distribution Program. It is also used off label as lymphodepleting conditioning in stem cell transplantation.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Alemtuzumab","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=alemtuzumab"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/alemtuzumab"}],"tags":["nci-list"],"related":[],"cancers":["cll","peripheral-t-cell-lymphoma","non-hodgkin-lymphoma"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd52"],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05607420"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: CD52 is a 12-amino-acid glycopeptide on a lipid anchor, present at very high surface density, which makes it an efficient complement-fixing target; it is also on normal B cells, T cells, monocytes and dendritic cells, which is why this antibody causes the deepest and longest lymphopenia of any used in lymphoma, with CD4 counts that can stay low for a year and with cytomegalovirus, Pneumocystis and fungal infection to match. Loss of the lipid anchor removes the antigen without touching the gene, which is the described escape route."],"brand":"Campath","modality":"Monoclonal antibody (anti-CD52)","mechanism":"Humanised IgG1 that binds CD52 on B and T lymphocytes, monocytes and NK cells; kills by antibody-dependent cellular cytotoxicity and complement-dependent lysis.","approvals":[{"region":"US","year":2001,"indication":"B-cell chronic lymphocytic leukaemia (single agent)"},{"region":"EU","year":2001,"indication":"B-CLL after fludarabine (MabCampath; authorisation withdrawn 2012)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2001-05-07","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"B-cell CLL treated with alkylating agents and failed fludarabine therapy"},{"date":"2007-09-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2001 converted to traditional approval 6.4 years after it was granted.","indication":"B-cell CLL treated with alkylating agents and failed fludarabine therapy"}]},{"id":"algenpantucel-l","kind":"drug","name":"Algenpantucel-L","aka":["HyperAcute Pancreas"],"tldr":"Algenpantucel-L was a whole-cell pancreatic cancer vaccine given after surgery; the phase 3 IMPRESS trial showed no survival benefit.","summary":"NewLink Genetics designed algenpantucel-L to exploit hyperacute rejection, the reaction that destroys pig organs in humans. A phase 2 study after pancreatic cancer surgery reported encouraging survival, which led to the largest adjuvant vaccine trial in the disease.\n\nIMPRESS randomised 722 patients with resected pancreatic cancer to standard adjuvant gemcitabine with or without chemoradiation, with or without algenpantucel-L. Overall survival was not improved, and a second phase 3 in borderline resectable and locally advanced disease (PILLAR) was also negative.","status":"negative","asOf":"2026-09-17","links":[{"label":"ClinicalTrials.gov NCT01072981","url":"https://clinicaltrials.gov/study/NCT01072981"}],"tags":[],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":["shared-antigen-vaccine"],"targets":[],"drugs":[],"companies":["newlink-genetics"],"institutions":[],"pathways":[],"terms":["vaccines-and-oncolytic-viruses"],"trials":["impress-trial"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"HyperAcute Pancreas","modality":"Allogeneic whole-cell vaccine expressing alpha-gal","mechanism":"Irradiated pancreatic cancer cell lines engineered to express alpha-1,3-galactosyl, a sugar humans lack, so pre-existing anti-alpha-gal antibodies attack the vaccine cells and, in theory, train immunity against shared tumour antigens.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"alisertib","kind":"drug","name":"Alisertib","aka":["PB-8237","MLN8237"],"tldr":"Alisertib is an oral serine/threonine kinase inhibitor from Puma Biotechnology, Inc., in registered phase 2 trials for small-cell lung cancer.","summary":"Alisertib is listed on ClinicalTrials.gov as an intervention in 5 registered phase 2 trials sponsored by Puma Biotechnology, Inc., in small-cell lung cancer. A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Alisertib","url":"https://clinicaltrials.gov/search?intr=Alisertib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["puma-biotechnology"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07465757","nct07465757","nct07465757","nct07465757","nct07465757","nct06095505","nct06369285"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral serine/threonine kinase inhibitor","mechanism":"A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"alitretinoin","kind":"drug","name":"Alitretinoin","aka":["9-cis-retinoic acid"],"tldr":"Alitretinoin gel is a vitamin A derivative applied directly to Kaposi sarcoma skin lesions in people with AIDS, approved in the United States in 1999 as the first topical treatment for the disease.","summary":"Alitretinoin (9-cis-retinoic acid) activates every known retinoid receptor. As Panretin gel it was approved by the FDA in February 1999 for cutaneous lesions of AIDS-related Kaposi sarcoma in patients who do not need systemic therapy, on the basis of two randomised trials in which about a third of lesions responded. Skin irritation at the application site is the main side effect. The oral form is approved in Europe for severe chronic hand eczema, not for cancer.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Alitretinoin","links":[{"label":"Drugs@FDA NDA020886","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=020886"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=alitretinoin"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Alitretinoin"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["kaposi-sarcoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["eisai"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05016349"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Panretin","modality":"Topical retinoid gel","mechanism":"A natural retinoid that binds both retinoic acid receptors and retinoid X receptors, switching on genes that slow proliferation and induce differentiation in Kaposi sarcoma cells.","approvals":[{"region":"US","year":1999,"indication":"Cutaneous lesions of AIDS-related Kaposi sarcoma (topical gel)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"allo-647","kind":"drug","name":"ALLO-647","aka":[],"tldr":"ALLO-647 is an experimental monoclonal antibody from Allogene Therapeutics in phase 2 trials for diffuse large B-cell lymphoma, aimed at CD52.","summary":"ALLO-647 is a monoclonal antibody developed by Allogene Therapeutics. Its target is CD52 (the sponsor names CD52). The sponsor states: Monoclonal antibody that recognises the CD52 antigen; used as part of a lymphodepletion regimen (with fludarabine and cyclophosphamide) ahead of allogeneic CAR T-cell dosing. ClinicalTrials.gov describes the intervention as: ALLO-647 is a monoclonal antibody that recognizes a CD52 antigen. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in diffuse large B-cell lymphoma. The largest, NCT04416984, plans to enrol 160 participants with primary completion expected 2029-02. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ALLO-647","url":"https://clinicaltrials.gov/search?intr=ALLO-647"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":[],"targets":["cd52"],"drugs":[],"companies":["allogene"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04416984"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Monoclonal antibody that recognises the CD52 antigen; used as part of a lymphodepletion regimen (with fludarabine and cyclophosphamide) ahead of allogeneic CAR T-cell dosing.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tregzi","kind":"drug","name":"Allogeneic regulatory T cell immunotherapy with HSPC and T cells-vldq","aka":["Orca-T","Tregzi"],"tldr":"Tregzi, formerly Orca-T, is a donor stem cell transplant sorted with high precision and given with regulatory T cells, so that patients with blood cancers keep the graft's anti-cancer effect while being protected from chronic graft-versus-host disease. The FDA approved it on 30 June 2026 for adults having a matched-donor transplant.","summary":"Tregzi is an allogeneic graft made by sorting a matched donor's mobilised blood into haematopoietic stem and progenitor cells and regulatory T cells, infusing them first, and giving a measured dose of conventional T cells two days later, so that the regulatory cells are in place before the effector cells arrive. In the phase 3 Precision-T trial, 187 adults with acute leukaemia or myelodysplastic syndrome undergoing myeloablative matched-donor transplantation were randomised to Tregzi with tacrolimus alone or a standard graft with tacrolimus and methotrexate; chronic graft-versus-host disease-free survival was not reached against 7.3 months (hazard ratio 0.26), and moderate to severe chronic graft-versus-host disease at one year fell from 44 percent to 13 percent, with every treated patient achieving neutrophil recovery within 28 days. The FDA approved it on 30 June 2026 for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease-free survival in adults with haematological malignancies having matched-donor transplantation with a myeloablative preparative regimen. The label warns of graft failure, graft-versus-host disease, infusion reactions and secondary malignancies. Whether the approach extends to mismatched and reduced-intensity transplants is being tested.","status":"approved","asOf":"2026-09-17","links":[{"label":"FDA approval notice (30 June 2026)","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-allogeneic-regulatory-t-cell-based-immunotherapy-hspc-and-t-cells-vldq-use-matched"},{"label":"ClinicalTrials.gov NCT05316701 (Precision-T)","url":"https://clinicaltrials.gov/study/NCT05316701"}],"tags":[],"related":[],"cancers":["aml","all-leukemia","mds"],"sections":["cell-therapy"],"technologies":["allogeneic-hsct","allogeneic-cell-therapy"],"targets":[],"drugs":[],"companies":["orca-bio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05316701"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tregzi","code":"Orca-T","modality":"Precision-sorted allogeneic stem cell graft with regulatory T cells","mechanism":"High-precision cell sorting separates the donor graft into stem and progenitor cells, regulatory T cells and conventional T cells; regulatory T cells infused ahead of a controlled conventional T cell dose suppress alloreactivity while preserving graft-versus-leukaemia and immune reconstitution.","approvals":[{"region":"US","year":2026,"indication":"Adults with haematological malignancies undergoing matched-donor haematopoietic stem cell transplantation with a myeloablative preparative regimen, for hematopoietic and immunologic reconstitution and to improve chronic graft-versus-host disease-free survival"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-06-30","type":"approval","region":"US","note":"FDA approves allogeneic regulatory T cell-based immunotherapy with HSPC and T cells-vldq (Tregzi, Orca Bio) on the Precision-T trial","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-allogeneic-regulatory-t-cell-based-immunotherapy-hspc-and-t-cells-vldq-use-matched"}]},{"id":"alpelisib","kind":"drug","name":"Alpelisib","aka":[],"tldr":"Alpelisib was the first PI3K drug for PIK3CA-mutant breast cancer (2019). It is effective, but high blood sugar and rash limited its use, and newer drugs are displacing it.","summary":"SOLAR-1: PFS 11.0 vs 5.7 months with fulvestrant in PIK3CA-mutant disease. Hyperglycaemia (grade 3-4 ~37%), rash, and diarrhoea are frequent; requires metformin prophylaxis strategies. Superseded in first-line endocrine-resistant disease by inavolisib and in the unselected AKT-pathway-altered population by capivasertib.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Alpelisib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Alpelisib"}],"tags":[],"related":["er-status","pik3ca-hotspot-mutation"],"cancers":["breast-hr-positive","hr-positive-metastatic-post-cdk46"],"sections":[],"technologies":["kinase-inhibitors","pi3k-akt-mtor-inhibitors"],"targets":["pik3ca"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":["pi3k-akt-mtor"],"terms":["hyperglycaemia"],"trials":["solar-1","nct05501886","nct04208178","nct05948943","nct05563220","nct05646862","nct05230810","nct05038735","nct04524000","nct04544189"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Piqray","modality":"Small-molecule PI3Kα inhibitor","mechanism":"ATP-competitive PI3Kα-selective inhibitor.","approvals":[{"region":"US","year":2019,"indication":"PIK3CA-mutant HR+/HER2- advanced breast cancer with fulvestrant after endocrine therapy"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"300 mg once daily with fulvestrant","modifications":"Reduce to 250 then 200 mg for hyperglycaemia/rash","monitoring":"Fasting glucose and HbA1c before and during; antihistamine prophylaxis for rash"},"toxicity":[{"event":"Hyperglycaemia","anyGradePct":64,"grade3PlusPct":37},{"event":"Diarrhoea","anyGradePct":58,"grade3PlusPct":7},{"event":"Rash","anyGradePct":36,"grade3PlusPct":10}],"access":[],"regulatoryEvents":[{"date":"2022-04-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 4.5 years later, when the FDA's table was read.","indication":"Treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy"},{"date":"2024-04-24","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 2.4 years later, when the FDA's table was read.","indication":"Formulation: Treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy."}]},{"id":"altretamine","kind":"drug","name":"Altretamine","aka":["Hexamethylmelamine","HMM"],"tldr":"Altretamine is an oral chemotherapy tablet approved in 1990 as a single agent for ovarian cancer that has persisted or come back after platinum-based treatment, now rarely used.","summary":"Hexamethylmelamine was studied through the 1970s in ovarian, lung and cervical cancers. The FDA approved it in December 1990 for palliative treatment of persistent or recurrent ovarian cancer after first-line cisplatin or alkylating agent combinations, on response rates of roughly 15 to 20 percent in platinum-refractory disease. Nausea, peripheral neuropathy and mood changes limit dosing, and it is taken in 14- or 21-day cycles. With the arrival of paclitaxel, liposomal doxorubicin, gemcitabine and PARP inhibitors it fell to the end of the ovarian cancer sequence and has been discontinued in several countries.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Altretamine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Altretamine"},{"label":"ChEMBL CHEMBL1200740","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200740"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Hexalen","modality":"Oral alkylating-like cytotoxic","mechanism":"Metabolised by liver cytochromes to reactive methylol intermediates that form DNA adducts; its exact alkylating chemistry differs from classic nitrogen mustards.","approvals":[{"region":"US","year":1990,"indication":"Persistent or recurrent ovarian cancer after first-line cisplatin or alkylating agent combinations"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"amifostine","kind":"drug","name":"Amifostine","aka":["WR-2721"],"tldr":"Amifostine (Ethyol) is an infusion given just before chemotherapy or radiotherapy to shield the kidneys from cisplatin and the salivary glands from radiation, reducing dry mouth after head and neck treatment.","summary":"Amifostine was approved in December 1995 to reduce cumulative renal toxicity from repeated cisplatin in advanced ovarian cancer and in 1999 to reduce moderate to severe xerostomia during post-operative radiotherapy for head and neck cancer when the field includes much of the parotid glands, on randomised trials in each setting. Concern that a radioprotector might also shield tumours, together with nausea, hypotension during infusion and skin reactions, has confined it to selected patients, and intensity-modulated radiotherapy has largely replaced it for salivary sparing. Guidelines (ASCO, MASCC) list it as an option rather than a standard.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Amifostine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=amifostine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/amifostine"}],"tags":["nci-list","supportive"],"related":[],"cancers":["ovarian","head-and-neck"],"sections":[],"technologies":["imrt-igrt"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07157033"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ethyol","modality":"Cytoprotective thiol prodrug","supportive":true,"mechanism":"Dephosphorylated by alkaline phosphatase, which is more abundant in normal tissue, to the free thiol WR-1065 that scavenges reactive oxygen species and binds cisplatin metabolites, protecting kidney and salivary tissue.","approvals":[{"region":"US","year":1995,"indication":"Reduction of cumulative cisplatin renal toxicity in advanced ovarian cancer"},{"region":"US","year":1999,"indication":"Reduction of moderate to severe xerostomia from post-operative radiotherapy for head and neck cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aminoglutethimide","kind":"drug","name":"Aminoglutethimide","aka":["Orimeten"],"tldr":"Aminoglutethimide produced a medical adrenalectomy for advanced breast and prostate cancer in the 1970s and 1980s, the ancestor of today's aromatase inhibitors, and was withdrawn once selective drugs arrived.","summary":"Originally an anticonvulsant, aminoglutethimide was found to block adrenal steroid synthesis and was approved in the United States in 1980 for Cushing's syndrome. Oncologists used it with hydrocortisone as a medical adrenalectomy in postmenopausal women with advanced breast cancer and in men with prostate cancer, with response rates similar to surgical adrenalectomy. Drowsiness, rash and the need for steroid replacement limited it, and the selective aromatase inhibitors anastrozole, letrozole and exemestane replaced it in the 1990s. It has been discontinued in most markets.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Aminoglutethimide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Aminoglutethimide"},{"label":"ChEMBL CHEMBL1200538","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200538"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["breast-hr-positive","prostate"],"sections":[],"technologies":["endocrine-therapy"],"targets":["aromatase","cyp11a1"],"drugs":["letrozole","exemestane"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct00006371"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cytadren","modality":"Oral steroidogenesis inhibitor","mechanism":"Inhibits CYP11A1, the first step of steroid synthesis from cholesterol, and aromatase, so adrenal steroids and oestrogens both fall; cortisol replacement is needed.","approvals":[{"region":"US","year":1980,"indication":"Cushing's syndrome (used off label as medical adrenalectomy in breast and prostate cancer)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aminolevulinic-acid","kind":"drug","name":"Aminolevulinic acid (topical, for photodynamic therapy)","aka":["5-ALA","ALA HCl"],"tldr":"Aminolevulinic acid is painted on sun-damaged skin and then activated with a lamp, destroying actinic keratoses before they can become skin cancer; in Europe the gel is also approved for superficial basal cell carcinoma.","summary":"Levulan Kerastick (aminolevulinic acid 20% solution) with BLU-U blue light was approved in December 1999 for actinic keratoses of the face and scalp; Ameluz (10% nanoemulsion gel) with the BF-RhodoLED red lamp followed in May 2016 for lesion- and field-directed treatment of mild to moderate actinic keratosis, on three randomised trials with complete clearance in the majority of patients. The EU authorised Ameluz in 2011 and extended it in 2017 to superficial and nodular basal cell carcinoma unsuitable for surgery. Photodynamic therapy is a field treatment for precancerous skin; pain during illumination and local erythema are expected. Oral aminolevulinic acid (Gleolan) for fluorescence-guided glioma surgery is an imaging agent covered elsewhere.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Aminolevulinic_acid","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=aminolevulinic%20acid"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/aminolevulinicacid"},{"label":"EPAR (Ameluz)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/ameluz"},{"label":"Jansen et al., five-year results of a randomised controlled trial comparing photodynamic therapy, topical imiquimod and topical 5-fluorouracil in superficial basal cell carcinoma (J Invest Dermatol 2018)","url":"https://doi.org/10.1016/j.jid.2017.09.033"},{"label":"Cancer Research UK: photodynamic therapy for non-melanoma skin cancer","url":"https://www.cancerresearchuk.org/about-cancer/skin-cancer/treatment/photodynamic-therapy"}],"tags":["nci-list","prevention"],"related":[],"cancers":["cutaneous-scc","basal-cell-carcinoma","skin-cancer"],"sections":[],"technologies":["precancer-ablation"],"targets":[],"drugs":[],"companies":["biofrontera","sun-pharma","photonamic"],"institutions":[],"pathways":[],"terms":["topical-and-destructive-treatment-bcc"],"trials":["nct07225621","nct05736406"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Which photosensitiser the basal cell carcinoma trials used. The randomised comparisons quoted on the basal cell carcinoma page (the Dutch three-arm trial and the Cochrane review) all used methyl aminolevulinate, not 5-aminolevulinic acid, so the five-year tumour-free figure of 62.7 percent and the cosmetic outcomes belong to that cream and are recorded there. Aminolevulinic acid gel is separately approved in the European Union for superficial and nodular basal cell carcinoma as well as for actinic keratosis; the two are not interchangeable in the evidence."],"brand":"Levulan Kerastick / Ameluz","modality":"Topical porphyrin precursor for photodynamic therapy","mechanism":"Taken up by dysplastic keratinocytes and converted to protoporphyrin IX, which on illumination with blue (Levulan) or red (Ameluz) light generates singlet oxygen and destroys the lesion.","approvals":[{"region":"US","year":1999,"indication":"Actinic keratoses of the face and scalp with blue-light photodynamic therapy (Levulan Kerastick)"},{"region":"US","year":2016,"indication":"Mild to moderate actinic keratosis of face and scalp with red-light PDT (Ameluz)"},{"region":"EU","year":2011,"indication":"Actinic keratosis; superficial and nodular basal cell carcinoma added 2017 (Ameluz)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aminopterin","kind":"drug","name":"Aminopterin","aka":["4-aminopteroylglutamic acid"],"tldr":"Aminopterin was the first drug to send childhood leukaemia into remission. In 1948 Sidney Farber reported temporary remissions in children with acute leukaemia, the birth of cancer chemotherapy; its safer cousin methotrexate replaced it within a decade.","summary":"Aminopterin was synthesised at Lederle Laboratories after Sidney Farber observed that folic acid seemed to accelerate leukaemia in children and reasoned that a folate antagonist might do the opposite. In June 1948 Farber and colleagues reported in the New England Journal of Medicine that 10 of 16 children with acute leukaemia had temporary remissions on aminopterin, the first demonstration that a drug could reverse a cancer.\n\nIts narrow margin between benefit and toxicity led to its replacement by methotrexate (amethopterin) in the 1950s, and it is no longer marketed. It is named on the acute lymphoblastic leukaemia pages as the founding drug of chemotherapy.","status":"historic","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Aminopterin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Aminopterin"}],"tags":["subtype-drugs-wave"],"related":["methotrexate"],"cancers":["all-leukemia","all-paediatric-standard-risk","aml"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["dhfr"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Antifolate (historic)","mechanism":"A folic acid analogue that blocks dihydrofolate reductase, starving dividing leukaemia cells of the reduced folate they need to make DNA; methotrexate, a methylated derivative, replaced it.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"amivantamab","kind":"drug","name":"Amivantamab","aka":[],"tldr":"A two-armed antibody that blocks EGFR and its escape partner MET, now first-line for EGFR-mutant lung cancer with lazertinib.","summary":"Amivantamab is a low-fucose bispecific IgG1 antibody that binds EGFR and MET at once, with enhanced ADCC and trogocytosis. It is approved for EGFR exon 20 insertion NSCLC (2021, with chemotherapy 2024, PAPILLON), first-line common EGFR mutations with lazertinib (MARIPOSA, 2024; OS benefit shown 2025), and post-osimertinib with chemotherapy (MARIPOSA-2). In MARIPOSA the combination extended progression-free survival to 23.7 versus 16.6 months against osimertinib (HR 0.70) and improved overall survival (HR 0.75). The cost is rash, nail toxicity and venous thromboembolism needing prophylactic anticoagulation; a subcutaneous formulation (2025) reduces infusion reactions. Whether the extra benefit justifies the extra burden is still debated. For a newcomer: the first regimen to beat the standard EGFR pill, at the price of more side effects.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Amivantamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Amivantamab"},{"label":"NICE TA850: amivantamab for EGFR exon 20 insertion mutation-positive advanced non-small-cell lung cancer after platinum-based chemotherapy (not recommended)","url":"https://www.nice.org.uk/guidance/ta850"},{"label":"NICE TA1122: amivantamab with lazertinib for untreated EGFR mutation-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1122"},{"label":"NICE TA1158: amivantamab with carboplatin and pemetrexed for untreated EGFR exon 20 insertion mutation-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1158"}],"tags":[],"related":["egfr-exon-19-deletion","egfr-exon-20-insertion"],"cancers":["nsclc","egfr-mutant-nsclc","met-altered-nsclc","lung-cancer"],"sections":[],"technologies":["bispecific-antibody"],"targets":["egfr","met"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":["cancer-drugs-fund","egfr-exon20-insertion"],"trials":["mariposa","nct05801029","nct05379595","nct05498428","nct05663866","nct06667076","nct07276399","nct04538664","nct07586202","nct06120140","nct06662786","nct06385080","nct06750094","nct05908734","nct05488314","nct07227025","nct04988295","chrysalis"],"people":[],"bottlenecks":[],"keyPapers":["paper-mariposa-nejm-2024"],"journals":[],"dependsOn":[],"notes":[],"brand":"Rybrevant","modality":"Bispecific antibody (EGFR×MET)","mechanism":"Low-fucose bispecific IgG1 blocking EGFR and MET with enhanced ADCC/trogocytosis.","approvals":[{"region":"US","year":2021,"indication":"EGFR exon 20 insertion NSCLC"},{"region":"US","year":2024,"indication":"First-line EGFR-mutant NSCLC with lazertinib"},{"region":"EU","year":2021,"indication":"Exon 20; 1L with lazertinib Jan 2025"},{"region":"England (NICE)","year":2022,"indication":"Locally advanced or metastatic non-small-cell lung cancer with an EGFR exon 20 insertion after platinum-based chemotherapy: not recommended","note":"TA850, published 14 December 2022, does not recommend amivantamab, so the second-line indication is not funded in England."},{"region":"England (NICE)","year":2026,"indication":"Untreated advanced EGFR mutation-positive non-small-cell lung cancer (exon 19 deletion or L858R), with lazertinib","note":"TA1122, published 21 January 2026 (MARIPOSA); must be funded in England within 90 days of publication."},{"region":"England (NICE)","year":2026,"indication":"Untreated advanced non-small-cell lung cancer with an activating EGFR exon 20 insertion, with carboplatin and pemetrexed","note":"TA1158, published 28 May 2026, available during a managed access period under a managed access agreement (PAPILLON)."}],"mechanismSteps":["Bispecific antibody binds EGFR and MET on the tumour cell","Receptor signalling is blocked and receptors are downregulated","Low-fucose Fc recruits macrophages and NK cells (trogocytosis, ADCC)","Tumour cells lose growth signals and are cleared by immune effectors","Lazertinib blocks intracellular EGFR kinase in parallel"],"dosing":{"route":"IV infusion (subcutaneous Rybrevant Faspro available)","schedule":"1,050 mg (<80 kg) or 1,400 mg (≥80 kg) weekly for 5 weeks (split first dose over 2 days), then every 2 weeks; with lazertinib 240 mg daily","modifications":"Hold for grade 3 rash or ILD; dermatologic prophylaxis (doxycycline, moisturisers) for 12 weeks; prophylactic anticoagulation for 4 months with lazertinib","monitoring":"Infusion reactions during first dose, skin and nails, VTE symptoms, ILD","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8"},"toxicity":[{"event":"Rash","anyGradePct":86,"grade3PlusPct":26,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8","note":"MARIPOSA, with lazertinib"},{"event":"Nail toxicity","anyGradePct":71,"grade3PlusPct":11,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8","note":"MARIPOSA, with lazertinib"},{"event":"Infusion-related reaction","anyGradePct":63,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8","note":"MARIPOSA, with lazertinib"},{"event":"Musculoskeletal pain","anyGradePct":47,"grade3PlusPct":2.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8","note":"MARIPOSA, with lazertinib"},{"event":"Oedema","anyGradePct":43,"grade3PlusPct":2.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8","note":"MARIPOSA, with lazertinib"},{"event":"Stomatitis","anyGradePct":43,"grade3PlusPct":2.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8","note":"MARIPOSA, with lazertinib"},{"event":"Venous thromboembolism","anyGradePct":36,"grade3PlusPct":11,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8","note":"MARIPOSA, with lazertinib"},{"event":"Paraesthesia","anyGradePct":35,"grade3PlusPct":1.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1466c070-9f97-4fa4-a955-6a6b59981fb8","note":"MARIPOSA, with lazertinib"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.janssencarepath.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with lazertinib for untreated EGFR-mutant NSCLC (2025) and exon 20 insertion after platinum","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-05-21","type":"accelerated-approval","region":"US","note":"Accelerated approval, EGFR exon 20 insertion NSCLC after platinum","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with locally advanced or metastatic non small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA approved test, whose disease has progressed on or after platinum based chemotherapy"},{"date":"2024-03-01","type":"conversion","region":"US","note":"First-line exon 20 insertion with chemotherapy (PAPILLON)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with locally advanced or metastatic non small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA approved test, whose disease has progressed on or after platinum based chemotherapy"},{"date":"2024-08-19","type":"approval","region":"US","note":"First-line EGFR-mutant NSCLC with lazertinib (MARIPOSA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-09-19","type":"approval","region":"US","note":"After osimertinib, with chemotherapy (MARIPOSA-2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-09","type":"approval","region":"US","note":"Subcutaneous formulation (PALOMA-3)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"amo959","kind":"drug","name":"AMO959","aka":["BY1298","BY101298"],"tldr":"AMO959 is a small-molecule inhibitor from Novartis Pharmaceuticals, in registered phase 2 trials for prostate cancer.","summary":"AMO959 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Novartis Pharmaceuticals, in prostate cancer. Described in the registry record as a by1298 by101298 dna damage response inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of AMO959","url":"https://clinicaltrials.gov/search?intr=AMO959"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07226986"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"AMO959","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a by1298 by101298 dna damage response inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"amrubicin","kind":"drug","name":"Amrubicin","aka":["Calsed","SM-5887"],"tldr":"Amrubicin is a Japanese anthracycline approved there in 2002 for small cell and non-small cell lung cancer, a standard second-line option for small cell lung cancer in Japan that failed to beat topotecan in a Western phase 3 trial and now serves as the comparator in global trials.","summary":"Sumitomo Dainippon's amrubicin was approved in Japan in 2002 for small cell and non-small cell lung cancer. Japanese trials established it as second-line treatment for relapsed small cell lung cancer, where response rates exceeded topotecan's, but the international ACT-1 phase 3 trial showed no overall survival advantage over topotecan and it was never approved in the United States or Europe. It remains in Japanese guidelines, and current phase 3 trials of new agents such as tarlatamab and ifinatamab deruxtecan use it as one of the standard comparator arms.","status":"established","asOf":"2026-09-16","wikipedia":"https://en.wikipedia.org/wiki/Amrubicin","links":[{"label":"ClinicalTrials.gov: trials of Amrubicin","url":"https://clinicaltrials.gov/search?intr=Amrubicin"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["sclc","nsclc"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["top2a"],"drugs":[],"companies":["sumitomo-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07365241","dellphi-304","ideate-lung02","nct06801834"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"modality":"Synthetic anthracycline (topoisomerase II inhibitor), intravenous","mechanism":"A fully synthetic anthracycline whose active metabolite amrubicinol poisons topoisomerase II; it causes less cardiotoxicity than doxorubicin in preclinical models.","approvals":[{"region":"JP","year":2002,"indication":"Small cell lung cancer and non-small cell lung cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"amsacrine","kind":"drug","name":"Amsacrine","aka":["m-AMSA"],"tldr":"Amsacrine is an intravenous leukaemia drug, approved in Europe, Canada and Australia in the 1980s, used mainly for acute myeloid leukaemia that has relapsed or resisted anthracycline-based treatment.","summary":"Amsacrine was synthesised in New Zealand by Bruce Cain's group and was the first synthetic DNA-intercalating agent to reach the clinic. It has been approved since the 1980s in several European countries, Canada and Australia for induction and consolidation of relapsed or refractory acute myeloid leukaemia, often with cytarabine and etoposide, and appears in some acute lymphoblastic leukaemia salvage protocols. It was never approved in the United States, where it remained investigational. Cardiac arrhythmias in patients with low potassium and the usual marrow suppression are the main hazards.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Amsacrine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Amsacrine"},{"label":"ChEMBL CHEMBL1200856","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200856"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["aml","all-leukemia"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["top2a"],"drugs":["cytarabine","etoposide"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct00002658","nct00003436","nct00002719"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Amsidine / Amsidyl","modality":"Acridine topoisomerase II inhibitor, intravenous","mechanism":"An intercalating acridine that traps topoisomerase II on DNA, producing double-strand breaks in dividing cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"amt-116","kind":"drug","name":"AMT-116","aka":[],"tldr":"AMT-116 is an antibody-drug conjugate from Multitude Therapeutics Inc., in registered phase 2 trials for non-small-cell lung cancer, small-cell lung cancer.","summary":"AMT-116 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Multitude Therapeutics Inc., in non-small-cell lung cancer, small-cell lung cancer. An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of AMT-116","url":"https://clinicaltrials.gov/search?intr=AMT-116"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc","sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["multitude-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07590531","nct06782334"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"AMT-116","modality":"Antibody-drug conjugate","mechanism":"An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"anagrelide","kind":"drug","name":"Anagrelide","aka":[],"tldr":"Anagrelide (Agrylin) is a capsule that lowers dangerously high platelet counts in essential thrombocythaemia and other myeloproliferative neoplasms, reducing the risk of clots and bleeding.","summary":"Anagrelide was approved by the FDA in March 1997 for thrombocythaemia secondary to myeloproliferative neoplasms, to reduce elevated platelet counts and the risk of thrombosis and to ameliorate thrombo-haemorrhagic symptoms, on open-label studies in which the majority of patients achieved platelet responses; the EU authorised Xagrid in November 2004 as second-line therapy for at-risk essential thrombocythaemia patients intolerant of or not adequately controlled by their current therapy. The UK PT-1 trial found hydroxyurea plus aspirin superior to anagrelide plus aspirin for arterial thrombosis and myelofibrotic transformation, so hydroxycarbamide remains first line and anagrelide is used in younger patients or when hydroxycarbamide fails. Palpitations, headache, fluid retention and, rarely, cardiomyopathy are the main adverse effects.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Anagrelide","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/xagrid"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=anagrelide"}],"tags":["ema-list"],"related":["hydroxyurea"],"cancers":["myeloproliferative-neoplasms","essential-thrombocythaemia"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04285086"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Agrylin / Xagrid","modality":"Oral platelet-lowering agent (imidazoquinazoline)","mechanism":"Inhibits megakaryocyte maturation and platelet production, probably through phosphodiesterase 3 inhibition and effects on megakaryocyte post-mitotic development; does not affect white cells.","approvals":[{"region":"US","year":1997,"indication":"Thrombocythaemia secondary to myeloproliferative neoplasms"},{"region":"EU","year":2004,"indication":"Elevated platelet counts in at-risk essential thrombocythaemia intolerant of or inadequately controlled by current therapy (Xagrid)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"anastrozole","kind":"drug","name":"Anastrozole","aka":["ZD1033"],"tldr":"Anastrozole is a daily tablet that stops the body making oestrogen after the menopause. It treats and prevents hormone-receptor-positive breast cancer and is an alternative to tamoxifen after surgery for ductal carcinoma in situ.","summary":"Anastrozole is a non-steroidal aromatase inhibitor developed by Zeneca (now AstraZeneca). It is taken as a 1 mg tablet once a day and is only effective after the menopause, or in premenopausal women whose ovaries have been suppressed, because it does not stop ovarian oestrogen production.\n\nThe FDA approved it in 1995 for advanced breast cancer in postmenopausal women after tamoxifen and in 2002 for adjuvant treatment of early hormone-receptor-positive disease, after the ATAC trial showed fewer recurrences than tamoxifen. In prevention, IBIS-II halved the incidence of breast cancer in high-risk postmenopausal women. After breast-conserving surgery for ductal carcinoma in situ, NSABP B-35 and IBIS-II DCIS found it at least as effective as tamoxifen, with a different side-effect profile: joint pain, bone loss and fractures rather than hot flushes, clots and endometrial cancer. Letrozole and exemestane are the other aromatase inhibitors in the class; OnCo's letrozole page covers the class as a whole.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Anastrozole","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Anastrozole"}],"tags":["subtype-drugs-wave"],"related":["letrozole","exemestane","tamoxifen"],"cancers":["breast-hr-positive","ductal-carcinoma-in-situ"],"sections":[],"technologies":["endocrine-therapy"],"targets":["aromatase"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Arimidex","modality":"Oral non-steroidal aromatase inhibitor","mechanism":"Reversibly blocks aromatase (CYP19A1), the enzyme that makes oestrogen from androgens in fat, muscle and the tumour itself, cutting circulating oestrogen in postmenopausal women to near zero.","approvals":[{"region":"US","year":1995,"indication":"Advanced breast cancer in postmenopausal women after tamoxifen"},{"region":"US","year":2002,"indication":"Adjuvant treatment of postmenopausal women with hormone receptor-positive early breast cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2002-09-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adjuvant treatment of postmenopausal women with hormone receptor positive early breast cancer"},{"date":"2005-09-16","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2002 converted to traditional approval 3.0 years after it was granted.","indication":"Adjuvant treatment of postmenopausal women with hormone receptor positive early breast cancer"}]},{"id":"anetumab-ravtansine","kind":"drug","name":"Anetumab ravtansine","aka":["BAY 94-9343"],"tldr":"Anetumab ravtansine was the first antibody-drug conjugate tested in a randomised trial in mesothelioma, aimed at the mesothelin protein that nearly all mesotheliomas carry; it was no better than vinorelbine chemotherapy as second-line treatment, and is now being tried with pembrolizumab.","summary":"Bayer built anetumab ravtansine around mesothelin, a surface protein over-expressed in mesothelioma, ovarian, pancreatic and some lung cancers but scarce in normal tissue. In the 248-patient randomised phase 2 trial in relapsed, mesothelin-positive pleural mesothelioma after platinum and pemetrexed, median progression-free survival was 4.3 months with anetumab ravtansine against 4.5 months with vinorelbine (hazard ratio 1.22), with far less neutropenia but corneal and other toxicities, so development for mesothelioma as a single agent stopped. A National Cancer Institute phase 1/2 trial is testing it with pembrolizumab, and the drug remains the reference case for why mesothelin-directed conjugates have struggled: shed mesothelin in the blood soaks up antibody, expression is heterogeneous, and a tubulin payload adds little to a slow-growing tumour that already resists chemotherapy.","status":"negative","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Anetumab_ravtansine","links":[{"label":"Kindler et al., anetumab ravtansine versus vinorelbine in relapsed mesothelin-positive mesothelioma, randomised phase 2 (Lancet Oncology 2022)","url":"https://doi.org/10.1016/S1470-2045(22)00061-4"},{"label":"Hassan et al., first-in-human phase 1 of anetumab ravtansine (JCO 2020)","url":"https://doi.org/10.1200/JCO.19.02085"},{"label":"Golfier et al., preclinical characterisation of BAY 94-9343 (Mol Cancer Ther 2014)","url":"https://doi.org/10.1158/1535-7163.MCT-13-0926"}],"tags":["gap-fill","owner-request"],"related":[],"cancers":["mesothelioma","ovarian","pancreatic","pleural-mesothelioma"],"sections":[],"technologies":["adc"],"targets":["mesothelin"],"drugs":["vinorelbine","pembrolizumab"],"companies":["bayer"],"institutions":[],"pathways":[],"terms":[],"trials":["anetumab-vs-vinorelbine-mpm","nci-anetumab-pembrolizumab-mpm"],"people":[],"bottlenecks":[],"keyPapers":["paper-kindler-lancet-oncol","paper-golfier-mol-cancer-ther","paper-hassan-j-clin-oncol"],"journals":[],"dependsOn":[],"notes":[],"modality":"Antibody-drug conjugate (anti-mesothelin antibody, DM4 payload), intravenous","mechanism":"A fully human anti-mesothelin antibody linked through a disulfide linker to the maytansinoid tubulin inhibitor DM4; binding to mesothelin on the tumour cell surface internalises the conjugate and releases the payload.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"anitocabtagene-autoleucel","kind":"drug","name":"Anitocabtagene autoleucel","aka":[],"tldr":"Anitocabtagene autoleucel is a BCMA CAR-T whose binder is a small synthetic D-domain protein rather than an antibody fragment, a design chosen for low immunogenicity. In heavily pretreated myeloma it produced responses in 97% of patients with mostly low-grade cytokine release syndrome and no delayed parkinsonism reported, and an FDA decision is due on 27 December 2026.","summary":"Anitocabtagene autoleucel is a BCMA CAR-T that uses a small synthetic D-domain binder instead of an antibody-derived scFv, with 4-1BB costimulation; the design gives low immunogenicity and avoids framework-related aggregation. In the pivotal phase 2 iMMagine-1 study of patients with at least 4 prior lines, it produced ORR 97% and high MRD negativity, with mostly low-grade cytokine release syndrome and no delayed parkinsonism reported to date. The BLA was accepted in February 2026 with a PDUFA date of 27 December 2026. Gilead's Kite acquired developer Arcellx in a deal that closed in April 2026. The phase 3 iMMagine-3 (second line and beyond) and iMMagine-4 (newly diagnosed) trials will show whether the safety profile holds in earlier populations. It is a next-generation myeloma CAR-T promising comparable efficacy with fewer rare neurological complications.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT05396885 (iMMagine-1)","url":"https://clinicaltrials.gov/study/NCT05396885"}],"tags":[],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["car-t"],"targets":["bcma"],"drugs":[],"companies":["gilead"],"institutions":[],"pathways":[],"terms":[],"trials":["immagine-1","nct06413498"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"anito-cel, CART-ddBCMA","modality":"CAR-T (BCMA, D-domain binder)","mechanism":"D-domain (non-scFv) BCMA binder CAR with 4-1BB costimulation; low immunogenicity, no framework-related aggregation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-02","type":"filing","region":"US","note":"BLA accepted; PDUFA 27 December 2026","source":"https://allsci.com/news/earnings-call-insights/gileads-q126-earnings-call-anito-cel-poised-for-december-pdufa-decision-three-acquisitions-near-inflection-points/"}]},{"id":"anlotinib","kind":"drug","name":"Anlotinib","aka":["Catequentinib","Qezzaqar"],"tldr":"Anlotinib is one of the most used cancer pills in China, first approved for lung cancer after other treatments have failed and then for several rarer tumours.","summary":"Developed by Chia Tai Tianqing (Sino Biopharm); approved by the NMPA in May 2018 for advanced NSCLC after at least two lines on ALTER 0303 (439 patients: median overall survival 9.6 versus 6.3 months with placebo, hazard ratio 0.68), then for soft tissue sarcoma (2019), small cell lung cancer after two lines (2019) and medullary thyroid cancer (2020), with a string of combination trials alongside domestic PD-1 antibodies. Its wide use reflects NRDL listing from 2018 and a large domestic sales force as much as its effect size.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Anlotinib","links":[{"label":"Sino Biopharmaceutical / Chia Tai Tianqing (archived copy)","url":"https://web.archive.org/web/20251122085435/https://www.sinobiopharm.com/en/"},{"label":"ALTER 0303 (JAMA Oncol 2018)","url":"https://doi.org/10.1001/jamaoncol.2018.3039"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["nsclc","sclc","sarcoma","alveolar-soft-part-sarcoma","thyroid"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf","fgfr2","kit"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":["vegf-angiogenesis"],"terms":[],"trials":["alter-0303","nct04854668","nct05862337","nct03016819","nct05913089","nct07548177","nct07562581"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Focus V","code":"AL3818","modality":"Small-molecule multi-kinase inhibitor (VEGFR, FGFR, PDGFR, KIT)","mechanism":"Oral inhibitor of VEGFR1-3, FGFR1-4, PDGFR alpha and beta, and KIT, blocking angiogenesis and tumour cell proliferation.","approvals":[{"region":"China","year":2018,"indication":"Advanced NSCLC after at least two lines of systemic therapy (ALTER 0303)"},{"region":"China","year":2019,"indication":"Advanced soft tissue sarcoma after chemotherapy; small cell lung cancer after at least two lines"},{"region":"China","year":2020,"indication":"Advanced medullary thyroid cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"anvumetostat","kind":"drug","name":"Anvumetostat","aka":["MTA Cooperative PRMT5 inhibitor","AMG 193"],"tldr":"Anvumetostat is an experimental small-molecule drug from Amgen in phase 2 trials for non-small-cell lung cancer, aimed at PRMT5 (MTAP-deleted cancers).","summary":"Anvumetostat is a small-molecule drug developed by Amgen. Its target is PRMT5 (MTAP-deleted cancers). The sponsor states: Anvumetostat (AMG 193) is an MTA-cooperative PRMT5 inhibitor, formulated as an oral film-coated tablet, studied in MTAP-deleted/null advanced NSCLC and other solid tumours. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in non-small-cell lung cancer. The largest, NCT05094336, plans to enrol 329 participants (actual) with primary completion expected 2026-11-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Anvumetostat","url":"https://clinicaltrials.gov/search?intr=Anvumetostat"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["prmt5-mtap"],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05094336","nct06593522"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Anvumetostat (AMG 193) is an MTA-cooperative PRMT5 inhibitor, formulated as an oral film-coated tablet, studied in MTAP-deleted/null advanced NSCLC and other solid tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"apalutamide","kind":"drug","name":"Apalutamide","aka":[],"tldr":"An AR blocker approved for prostate cancer that has spread and for high-risk disease before it shows on scans.","summary":"Apalutamide is a second-generation androgen receptor antagonist with lower CNS penetration than enzalutamide, intended to reduce seizures and cognitive effects while blocking the receptor as completely. It is given with androgen deprivation for non-metastatic castration-resistant prostate cancer and for metastatic hormone-sensitive disease. SPARTAN (nmCRPC) showed an overall survival benefit and TITAN (mHSPC) reduced deaths by about a third (OS HR 0.65), supporting the 2018 and 2019 approvals. Rash and hypothyroidism are its distinctive side effects and distinguish it from enzalutamide and darolutamide. There is no head-to-head trial among the three AR antagonists, so choice rests on side-effect profile and trial populations. In plain terms, it is an AR-blocking pill used both for cancer that has spread and for high-risk disease that has not yet shown on scans.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Apalutamide","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Apalutamide"},{"label":"NICE TA741: apalutamide with androgen deprivation therapy for treating hormone-sensitive metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta741"},{"label":"NICE TA740: apalutamide with androgen deprivation therapy for treating high-risk hormone-relapsed non-metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta740"}],"tags":[],"related":[],"cancers":["prostate","prostate-nmcrpc","prostate-mhspc","salivary-duct-carcinoma"],"sections":[],"technologies":["androgen-deprivation"],"targets":["androgen-receptor"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":["ar-signaling"],"terms":["mcrpc-mhspc","prostate-uk-drug-approvals"],"trials":["nct05884398","nct07611110","nct02489318","nct05422911","nct05367440","nct07745361","nct04557059","nct02257736","nct03767244","nct02531516","nct01946204","nct04325828"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NICE TA741 recommends apalutamide with androgen deprivation for hormone-sensitive metastatic prostate cancer only if docetaxel is not suitable: the committee found it not cost effective against docetaxel but within the acceptable range against androgen deprivation alone. NICE TA740 recommends apalutamide with androgen deprivation for high-risk hormone-relapsed non-metastatic prostate cancer, high risk being a PSA that has doubled in 10 months or less on continuous androgen deprivation."],"brand":"Erleada","modality":"Small-molecule AR antagonist","mechanism":"Second-generation AR antagonist with lower CNS penetration than enzalutamide.","approvals":[{"region":"US","year":2018,"indication":"Non-metastatic CRPC (SPARTAN)"},{"region":"US","year":2019,"indication":"mHSPC (TITAN)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aprepitant","kind":"drug","name":"Aprepitant (and fosaprepitant)","aka":["Fosaprepitant","Ivemend","Emend IV","Aponvie"],"tldr":"Aprepitant (Emend) was the first of a new class of anti-sickness drugs. Taken with a 5-HT3 blocker and dexamethasone, it prevents the delayed nausea and vomiting that follow strongly emetogenic chemotherapy such as cisplatin.","summary":"Aprepitant capsules were approved by the FDA in March 2003 for prevention of acute and delayed nausea and vomiting with highly emetogenic chemotherapy, extended to moderately emetogenic regimens in 2005, with the intravenous prodrug fosaprepitant (Emend for injection) approved in 2008 and a paediatric oral suspension from 6 months of age in 2015; Cinvanti (2017) is an intravenous aprepitant emulsion. In the pivotal cisplatin trials, adding aprepitant to ondansetron and dexamethasone raised complete response rates by roughly 20 percentage points, establishing triple therapy as the standard that every guideline (ASCO, MASCC/ESMO, NCCN) now recommends for highly emetogenic chemotherapy. The EU authorised Emend in 2003 and Ivemend in 2008. It is a moderate CYP3A4 inhibitor, so dexamethasone doses are halved and interactions with some chemotherapy and warfarin need attention.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Aprepitant","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=aprepitant"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/aprepitant"},{"label":"EPAR (Emend)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/emend"}],"tags":["nci-list","supportive"],"related":["ondansetron","palonosetron","dexamethasone"],"cancers":[],"sections":[],"technologies":["antiemetic-therapy"],"targets":[],"drugs":[],"companies":["merck","heron-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06904235"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Emend / Cinvanti","modality":"Neurokinin-1 receptor antagonist (antiemetic)","supportive":true,"mechanism":"Selective high-affinity antagonist of substance P at NK1 receptors in the brainstem vomiting centre; fosaprepitant is an intravenous prodrug converted to aprepitant within minutes.","approvals":[{"region":"US","year":2003,"indication":"Prevention of acute and delayed nausea and vomiting with highly emetogenic chemotherapy (moderately emetogenic added 2005; paediatric suspension 2015)"},{"region":"US","year":2008,"indication":"Fosaprepitant intravenous prodrug for the same indications"},{"region":"EU","year":2003,"indication":"Prevention of chemotherapy-induced nausea and vomiting (Emend); Ivemend 2008"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ard103","kind":"drug","name":"ARD103","aka":[],"tldr":"ARD103 is a car-t cell therapy from ARCE Therapeutics, Inc., in registered phase 2 trials for acute myeloid leukaemia, myelodysplastic syndromes / neoplasms.","summary":"ARD103 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by ARCE Therapeutics, Inc., in acute myeloid leukaemia, myelodysplastic syndromes / neoplasms. A chimeric antigen receptor T-cell therapy, as described in the registry record: the patient's T cells are engineered to recognise a tumour antigen and infused back. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of ARD103","url":"https://clinicaltrials.gov/search?intr=ARD103"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["aml","mds"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06680752"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"ARD103","modality":"CAR-T cell therapy","mechanism":"A chimeric antigen receptor T-cell therapy, as described in the registry record: the patient's T cells are engineered to recognise a tumour antigen and infused back.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"arlocabtagene-autoleucel","kind":"drug","name":"Arlocabtagene Autoleucel","aka":["Arlo-cel","GPRC5D CAR T cells"],"tldr":"Arlocabtagene Autoleucel is an experimental CAR-T cell therapy from Juno Therapeutics, a Bristol-Myers Squibb in phase 3 trials for multiple myeloma, aimed at GPRC5D.","summary":"Arlocabtagene Autoleucel (CC-95266, BMS-986393) is a CAR-T cell therapy developed by Juno Therapeutics, a Bristol-Myers Squibb. Its target is GPRC5D (the sponsor names GPRC5D). The sponsor states: Arlocabtagene autoleucel (BMS-986393, CC-95266) is a GPRC5D-directed CAR T cell therapy, engineered to target GPRC5D-expressing myeloma cells, given as an infusion to heavily pretreated relapsed/refractory multiple myeloma patients. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06615479 (A Study to Compare the Efficacy and Safety of BMS-986393 Versus Standard Regimens in Adult Participants With Relapsed or Refractory and Lenalidomide-exposed Multiple Myeloma (QUINTESSENTIAL-2)), in multiple myeloma. The largest, NCT06615479, plans to enrol 440 participants with primary completion expected 2027-12-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Arlocabtagene Autoleucel","url":"https://clinicaltrials.gov/search?intr=CC-95266"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":["gprc5d"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["quintessential-2","nct06297226"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CC-95266, BMS-986393","modality":"CAR-T cell therapy","mechanism":"Arlocabtagene autoleucel (BMS-986393, CC-95266) is a GPRC5D-directed CAR T cell therapy, engineered to target GPRC5D-expressing myeloma cells, given as an infusion to heavily pretreated relapsed/refractory multiple myeloma patients.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"arsenic-trioxide","kind":"drug","name":"Arsenic trioxide","aka":[],"tldr":"An ancient poison turned cure: with retinoic acid it cures more than 95% of acute promyelocytic leukaemia without conventional chemotherapy.","summary":"Developed from traditional Chinese medicine in Harbin (1990s); approved 2000 for relapsed APL and 2018 first line with tretinoin after APL0406 (Lo-Coco, NEJM 2013) showed ATRA-ATO superior to ATRA-chemotherapy in standard-risk APL. Toxicities: differentiation syndrome, QT prolongation, hepatotoxicity. Oral arsenic (Realgar-Indigo naturalis) is used in China.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Arsenic_trioxide","links":[{"label":"APL0406 (NEJM 2013)","url":"https://doi.org/10.1056/NEJMoa1300874"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=arsenic%20trioxide"}],"tags":["gap-fill"],"related":["traditional-chinese-herbal-medicine"],"cancers":["aml","apl"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":[],"companies":["teva"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Trisenox","modality":"Inorganic arsenical (differentiation agent)","mechanism":"Binds PML moiety of PML-RARA, inducing SUMOylation and degradation of the fusion oncoprotein; triggers differentiation and apoptosis of APL blasts.","approvals":[{"region":"US","year":2000,"indication":"Relapsed/refractory APL"},{"region":"US","year":2018,"indication":"Newly diagnosed low-risk APL with tretinoin"},{"region":"EU","year":2002,"indication":"APL"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"artera-ai-breast","kind":"drug","name":"ArteraAI Breast","aka":[],"tldr":"An FDA-cleared AI test (May 2026) that reads breast cancer slides to estimate recurrence risk in early hormone-positive disease.","summary":"ArteraAI Breast is an AI digital pathology test that extracts foundation-model-derived features from H&E whole-slide images to estimate recurrence risk in early-stage HR-positive, HER2-negative invasive breast cancer. FDA-cleared in May 2026, it is the first digital-pathology-based risk stratification tool for this setting and is positioned as a faster and cheaper alternative to gene-expression assays such as Oncotype DX, because it needs only the slide already made for diagnosis rather than a separate laboratory send-out. It is cleared for prognosis; whether it also predicts chemotherapy benefit, as TAILORx established for the 21-gene score, has not been shown. Performance across scanners, laboratories and populations will decide how widely it is adopted. For a newcomer: software that reads the routine pathology slide to judge how likely a hormone-positive breast cancer is to return.","status":"approved","asOf":"2026-09-04","links":[{"label":"Artera: ArteraAI tests","url":"https://artera.ai/"}],"tags":[],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["digital-pathology-ai","pathology-foundation-model"],"targets":[],"drugs":[],"companies":["artera"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"AI digital pathology risk test","mechanism":"Foundation-model-derived features from H&E whole-slide images.","approvals":[{"region":"US","year":2026,"indication":"Risk stratification in early-stage HR+/HER2- invasive breast cancer"}],"mechanismSteps":["H&E whole-slide images are scanned","Foundation-model features are extracted","Recurrence risk is predicted for early HR+/HER2- disease","Result complements or replaces gene-expression assays"],"toxicity":[],"access":[{"country":"US","reimbursement":"Launched May 2026; coverage determinations pending","source":"https://artera.ai","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2026-05","type":"approval","region":"US","note":"FDA clearance: first digital-pathology-based risk stratification tool for early HR+/HER2- breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"artera-ai-prostate","kind":"drug","name":"ArteraAI Prostate","aka":[],"tldr":"The first AI tool cleared by the FDA to predict both prognosis and treatment benefit from a routine biopsy slide, in prostate cancer.","summary":"ArteraAI Prostate is a multimodal artificial intelligence test that combines a deep-learning image encoder applied to routine H&E biopsy slides with clinical variables to predict the risk of distant metastasis and the benefit of short-term androgen deprivation therapy added to radiotherapy in localised prostate cancer. It was validated retrospectively on NRG/RTOG randomised trial cohorts, received FDA de novo authorisation in August 2025 as the first AI tool cleared to predict both prognosis and treatment benefit from a slide, and is listed in NCCN guidelines. Its practical use is helping men with intermediate-risk disease decide whether hormone therapy is worth its side effects. Prospective validation is the open question. For a newcomer: software that reads a biopsy slide to say how aggressive the cancer is and whether hormone therapy will help.","status":"approved","asOf":"2026-09-04","links":[{"label":"Artera: ArteraAI tests","url":"https://artera.ai/"}],"tags":[],"related":[],"cancers":["prostate"],"sections":[],"technologies":["digital-pathology-ai","pathology-foundation-model"],"targets":[],"drugs":[],"companies":["artera"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"AI digital pathology prognostic/predictive test","mechanism":"Deep-learning image encoder plus clinical data fusion.","approvals":[{"region":"US","year":2025,"indication":"Localised prostate cancer risk stratification and ADT benefit prediction"}],"mechanismSteps":["Biopsy slides are scanned to digital images","A deep-learning model extracts morphology features","Features are fused with clinical variables (PSA, Gleason, T stage)","The model outputs distant-metastasis risk and predicted benefit from short-term ADT","The report supports the radiation-plus-hormone decision"],"toxicity":[],"access":[{"country":"US","listPrice":"~$1,500 (Medicare CLFS rate established 2025, approximate)","reimbursement":"Medicare coverage via MolDX; NCCN-listed","source":"https://artera.ai","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2025-08-14","type":"approval","region":"US","note":"De novo authorisation: first AI pathology test cleared for prognostic and predictive use in localised prostate cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"arx788","kind":"drug","name":"ARX788","aka":[],"tldr":"ARX788 is a HER2 ADC with a precisely placed, non-cleavable payload that beat lapatinib-capecitabine in China and showed activity in brain metastases.","summary":"ARX788 is a HER2 antibody-drug conjugate in which the payload is attached at a precisely engineered non-natural amino acid (pAF), giving a homogeneous drug-to-antibody ratio of about 2; its non-cleavable linker limits the bystander effect but improves stability in circulation. Developed by Ambrx (acquired by Johnson & Johnson in 2024) and NovoCodex, it is aimed at HER2-positive metastatic breast cancer. The ACE-Breast-02 phase 3 trial showed PFS of 11.3 versus 8.2 months (HR 0.64) against lapatinib plus capecitabine, and ACE-Breast-06 showed intracranial activity in active brain metastases. Ocular and interstitial lung toxicities are the main concerns. It holds FDA Fast Track designation and a Chinese marketing application is planned; how it will be positioned against T-DXd is unclear. For a newcomer, it is a HER2 ADC with a precisely placed payload that showed activity in the brain.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT04829604 (ACE-Breast-02)","url":"https://clinicaltrials.gov/study/NCT04829604"}],"tags":[],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":["adc","site-specific-conjugation"],"targets":["her2"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["ace-breast-02","nct04829604"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"ADC","payload":"AS269 (MMAF-like tubulin inhibitor), DAR ~1.9","linker":"Non-natural amino acid, non-cleavable","mechanism":"Site-specific pAF conjugation gives homogeneous DAR ~2; non-cleavable linker limits bystander effect but improves stability.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"asc40","kind":"drug","name":"ASC40","aka":[],"tldr":"ASC40 is an experimental small-molecule drug from Ascletis Pharmaceuticals in phase 3 trials for glioma & glioblastoma, with its target not yet stated publicly.","summary":"ASC40 (TVB-2640) is a small-molecule drug developed by Ascletis Pharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: ASC40 tablets administered orally once daily. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05118776 (Study to Evaluate the Safety and Efficacy of ASC40 Tablets in Combination With Bevacizumab in Subjects With rGBM), in glioma & glioblastoma. The largest, NCT05118776, plans to enrol 136 participants (actual) with primary completion was scheduled for 2025-06 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ASC40","url":"https://clinicaltrials.gov/search?intr=TVB-2640"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["ascletis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05118776"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"TVB-2640","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"asciminib","kind":"drug","name":"Asciminib","aka":[],"tldr":"Asciminib is a BCR::ABL1 blocker that binds a different pocket from every other TKI, approved for all newly diagnosed chronic myeloid leukaemia in 2024 and now being tested in Ph-positive ALL.","summary":"Binds the myristoyl pocket (STAMP), so it works against ATP-site resistance mutations and combines with ATP-competitive TKIs. ASCEMBL (third line) and ASC4FIRST (frontline CML: MMR at 48 weeks 67.7% vs 49.0% for investigator-selected TKIs) led to approvals in 2021 and October 2024. Trials combine asciminib with dasatinib or ponatinib and with blinatumomab in Ph+ ALL to pre-empt resistance. Included here because Ph+ ALL shares the target.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Asciminib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Asciminib"}],"tags":[],"related":["bcr-abl1-t315i"],"cancers":["all-leukemia","cml-chronic-phase"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["bcr-abl","abl1"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":["ph-positive-all"],"trials":["nct07354074","nct06514534","nct04971226","nct04925479","nct05384587","nct05456191","nct07387926"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Approved indications are in chronic myeloid leukaemia; Ph+ ALL use is investigational."],"brand":"Scemblix","modality":"Small-molecule allosteric ABL1 inhibitor (STAMP)","mechanism":"Allosteric inhibitor locking ABL1 in an inactive conformation via the myristoyl pocket; active against T315I at higher dose.","approvals":[{"region":"US","year":2021,"indication":"CML after ≥2 prior TKIs; T315I CML"},{"region":"US","year":2024,"indication":"Newly diagnosed Ph+ CML in chronic phase (ASC4FIRST)"}],"mechanismSteps":["Asciminib occupies the myristoyl pocket, mimicking the natural auto-inhibitory switch","ABL1 is locked inactive regardless of ATP-site mutations","Combination with an ATP-site TKI blocks two independent escape routes"],"dosing":{"route":"Oral","schedule":"CML: 80 mg once daily or 40 mg twice daily; 200 mg twice daily for T315I","monitoring":"Pancreatic enzymes, blood pressure, myelosuppression","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Scemblix"},"toxicity":[{"event":"Thrombocytopenia","grade3PlusPct":17,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Scemblix"},{"event":"Hypertension","anyGradePct":13},{"event":"Pancreatic enzyme elevation","anyGradePct":13}],"access":[],"regulatoryEvents":[{"date":"2021-10-29","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitors (TKIs)"},{"date":"2022-10-12","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2021 converted to traditional approval 1.0 year after it was granted.","indication":"Adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with 2 or more tyrosine kinase inhibitors (TKIs)"},{"date":"2024-10-29","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.9 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-asciminib-newly-diagnosed-chronic-myeloid-leukemia","indication":"Treatment of adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP)."}]},{"id":"asp2138","kind":"drug","name":"ASP2138","aka":[],"tldr":"ASP2138 is an experimental bispecific antibody from Astellas Pharma Global Development in phase 3 trials for gastric & gastro-oesophageal junction cancer, aimed at Claudin 18.2 and CD3.","summary":"ASP2138 is a bispecific antibody developed by Astellas Pharma Global Development. Its targets are Claudin 18.2 and CD3 (the sponsor names CLDN18.2 x CD3). The sponsor states: Binds CLDN18.2 (claudin 18.2) on tumour cells and CD3 on T cells, bringing T cells into proximity with CLDN18.2-expressing tumour cells to activate an anti-tumour T-cell response; administered subcutaneously. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07673887 (A Study of ASP2138 Together With Chemotherapy and Pembrolizumab in Adults With Gastric Cancer), in gastric & gastro-oesophageal junction cancer. The largest, NCT07673887, plans to enrol 570 participants with primary completion expected 2031-02-28. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ASP2138","url":"https://clinicaltrials.gov/search?intr=ASP2138"},{"label":"Sponsor pipeline page","url":"https://newsroom.astellas.com/2026-08-25-astellas-doses-first-patient-in-phase-3-study-of-asp2138-in-cldn18-2-postive-and-her2-negative-locally-advanced-unresectable-or-metastatic-gastric-or-gastroesophageal-junction-gej-adenocarcinoma"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric"],"sections":[],"technologies":[],"targets":["cldn18-2","cd3"],"drugs":[],"companies":["astellas"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07673887"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"Binds CLDN18.2 (claudin 18.2) on tumour cells and CD3 on T cells, bringing T cells into proximity with CLDN18.2-expressing tumour cells to activate an anti-tumour T-cell response; administered subcutaneously.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"asparaginase","kind":"drug","name":"Asparaginase (pegaspargase, calaspargase pegol, Erwinia asparaginase)","aka":[],"tldr":"An enzyme that starves leukaemia cells of an amino acid they cannot make; a mainstay of childhood ALL therapy for 50 years, with new versions solving allergy and supply problems.","summary":"E. coli asparaginase (1978), pegaspargase (1994; first-line 2006), calaspargase pegol (2018, longer interval) and recombinant Erwinia asparaginase (Rylaze, 2021) after Erwinaze shortages. Asparaginase intensity underlies paediatric-inspired adult ALL regimens. Toxicities: hypersensitivity, pancreatitis, thrombosis, hepatotoxicity, hyperglycaemia; silent inactivation is monitored by asparaginase activity levels.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Asparaginase","links":[{"label":"Label: Rylaze (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rylaze"}],"tags":["gap-fill"],"related":[],"cancers":["all-leukemia","peripheral-t-cell-lymphoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["servier","jazz"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Oncaspar / Asparlas / Rylaze / Erwinaze","modality":"Enzyme therapy","mechanism":"Depletes plasma asparagine (and glutamine), starving lymphoblasts that lack asparagine synthetase; pegylation extends half-life, Erwinia-derived enzyme avoids cross-reactive hypersensitivity.","approvals":[{"region":"US","year":1994,"indication":"ALL (pegaspargase, hypersensitivity to native); first line 2006"},{"region":"US","year":2018,"indication":"ALL in patients 1 month-21 years (calaspargase pegol)"},{"region":"US","year":2021,"indication":"ALL/LBL with hypersensitivity to E. coli asparaginase (Rylaze)"},{"region":"EU","year":2016,"indication":"Oncaspar centralised 2016; Erwinase and native asparaginases national"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aspirin","kind":"drug","name":"Aspirin","aka":["Acetylsalicylic acid","Low-dose aspirin"],"tldr":"Aspirin is not a cancer drug but sits in cancer care in two places: low doses prevent clots in polycythaemia vera and essential thrombocythaemia, and long-term use lowers colorectal cancer in people with Lynch syndrome, while a trial in the healthy elderly found no benefit and possible harm.","summary":"In the myeloproliferative neoplasms, low-dose aspirin is given to almost everyone with polycythaemia vera and to low-risk and high-risk essential thrombocythaemia, because activated platelets cause both the microvascular symptoms (erythromelalgia, headache) and the arterial clots that drive mortality; the ECLAP trial in polycythaemia vera showed fewer combined cardiovascular events with aspirin. In prevention, the CAPP2 trial in Lynch syndrome found that 600 mg of aspirin a day for at least two years roughly halved colorectal cancers over the following decade, and long-term follow-up of cardiovascular trials suggested fewer colorectal cancer deaths. Against that, ASPREE, a trial in healthy adults over 70, found more cancer deaths on aspirin than placebo, so aspirin is not recommended for cancer prevention in older people without a specific indication. The Add-Aspirin trial is testing aspirin after treatment for breast, colorectal, gastro-oesophageal and prostate cancer. Its harms are bleeding, especially in the gut, and it must be avoided in essential thrombocythaemia with acquired von Willebrand deficiency.","status":"established","asOf":"2026-09-16","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Aspirin"},{"label":"CAPP2 long-term follow-up, Lancet 2020","url":"https://doi.org/10.1016/S0140-6736(20)30366-4"},{"label":"ASPREE cancer outcomes, JNCI 2021","url":"https://doi.org/10.1093/jnci/djaa114"},{"label":"Add-Aspirin trial","url":"https://www.addaspirintrial.org/"}],"tags":["mpn","prevention"],"related":[],"cancers":["polycythaemia-vera","essential-thrombocythaemia","colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-mcneil-j-natl-cancer-inst"],"journals":[],"dependsOn":[],"notes":[],"modality":"Antiplatelet and anti-inflammatory small molecule (cyclo-oxygenase inhibitor), used in cancer for thrombosis prevention and studied for chemoprevention","mechanism":"Irreversibly blocks cyclo-oxygenase 1 in platelets, stopping thromboxane production and platelet clumping; at higher doses also blocks cyclo-oxygenase 2 and prostaglandin-driven inflammation, the proposed route to its effect on colorectal cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"at101","kind":"drug","name":"AT101","aka":["AT101(Anti-CD19 Chimeric Antigen Receptor T cell)"],"tldr":"AT101 is an experimental CAR-T cell therapy from AbClon in phase 2 trials for hodgkin lymphoma, aimed at CD19.","summary":"AT101 is a CAR-T cell therapy developed by AbClon. Its target is CD19 (the sponsor names CD19). The sponsor states: Autologous chimeric antigen receptor (CAR) T cells engineered to recognise CD19 on B-cell non-Hodgkin lymphoma cells. ClinicalTrials.gov describes the intervention as: Anti-CD19 Chimeric Antigen Receptor T cell. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in hodgkin lymphoma. The largest, NCT05338931, plans to enrol 82 participants with primary completion expected 2030-03-15. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AT101","url":"https://clinicaltrials.gov/search?intr=AT101"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":["cd19"],"drugs":[],"companies":["abclon"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05338931"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"Autologous chimeric antigen receptor (CAR) T cells engineered to recognise CD19 on B-cell non-Hodgkin lymphoma cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"atebimetinib","kind":"drug","name":"Atebimetinib","aka":[],"tldr":"Atebimetinib is an experimental small-molecule drug from Immuneering in phase 3 trials for pancreatic ductal adenocarcinoma, aimed at MEK1/2.","summary":"Atebimetinib is a small-molecule drug developed by Immuneering. Its target is MEK1/2 (the sponsor names MEK). The sponsor states: Oral, once-daily 'Deep Cyclic Inhibitor' of MEK that pulses target inhibition rather than suppressing it continuously. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07562152 (Atebimetinib + GnP as a First Line Treatment in Patients With Metastatic Pancreatic Adenocarcinoma), in pancreatic ductal adenocarcinoma. The largest, NCT07562152, plans to enrol 510 participants with primary completion expected 2028-06. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Atebimetinib","url":"https://clinicaltrials.gov/search?intr=Atebimetinib"},{"label":"Sponsor page","url":"https://www.immuneering.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":["mek"],"drugs":[],"companies":["immuneering"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07562152"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Oral, once-daily 'Deep Cyclic Inhibitor' of MEK that pulses target inhibition rather than suppressing it continuously.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"atezolizumab","kind":"drug","name":"Atezolizumab","aka":[],"tldr":"A PD-L1 blocker used in lung, liver, and bladder cancer. In 2026 it became the first drug approved based on a blood test showing leftover cancer after bladder surgery.","summary":"Approvals in NSCLC (including adjuvant, IMpower010), SCLC (first-line with chemotherapy, IMpower133), HCC (with bevacizumab, IMbrave150), melanoma (with cobimetinib/vemurafenib), alveolar soft-part sarcoma, and Q2 2026 adjuvant ctDNA-positive muscle-invasive bladder cancer (IMvigor011), the first ctDNA-guided approval. Its TNBC indication (IMpassion130) was withdrawn in the US in 2021.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Atezolizumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Atezolizumab"},{"label":"NICE TA639: atezolizumab with nab-paclitaxel for untreated PD-L1-positive advanced triple-negative breast cancer (1 July 2020)","url":"https://www.nice.org.uk/guidance/ta639"},{"label":"Tecentriq label (openFDA): indications list, no breast cancer indication after the 2021 withdrawal","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22TECENTRIQ%22"},{"label":"EMA Tecentriq product page: EU TNBC indication retained","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/tecentriq"},{"label":"NICE TA520: atezolizumab for locally advanced or metastatic non-small-cell lung cancer after chemotherapy","url":"https://www.nice.org.uk/guidance/ta520"},{"label":"NICE TA584: atezolizumab in combination for treating metastatic non-squamous non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta584"},{"label":"NICE TA638: atezolizumab with carboplatin and etoposide for untreated extensive-stage small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta638"},{"label":"NICE TA705: atezolizumab monotherapy for untreated advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta705"},{"label":"NICE TA1071: atezolizumab for adjuvant treatment of resected non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1071"}],"tags":[],"related":["pd-l1-ic-score","pd-l1-tc-score","ctdna-mrd-positive"],"cancers":["nsclc","sclc","hcc","hcc-advanced","urothelial","tnbc","alveolar-soft-part-sarcoma","tnbc-metastatic","pdl1-high-nsclc","resectable-nsclc","extensive-stage-sclc","pancreatic","resectable-pdac","lung-cancer"],"sections":[],"technologies":["checkpoint-inhibitor","mrd-testing"],"targets":["pdl1"],"drugs":[],"companies":["roche-genentech","chugai"],"institutions":[],"pathways":[],"terms":[],"trials":["imvigor011","impassion130","nct06712355","nct07625644","nct03755791","nct05425940","nct04446117","nct05317858","nct06921785","nct07472517","nct05468489","nct02486718","nct05047250","nct05904886","nct03178552","nct07195695","nct04712643","nct05652686","nct03228667","nct07654400","nct05009069","nct04440735","nct06463665","nct07235293","nct04302025","nct04471727","nct05440708","nct06096779","nct07155174","nct05733598","nct06362252","nct05703971","nct07227597","nct04931342","nct05142696","nct04524871","nct05224141","nct05645692","nct07280377","nct03148418","nct05434234","nct04665856","nct04873362","nct05112965","nct00781612","nct03533283","nct07407933","nct05968326","neotrip","impassion030","gepardouze","barbican","autogene-cevumeran-phase-1","oak","impower110","impower150","impower133","imforte"],"people":[],"bottlenecks":[],"keyPapers":["paper-rugo-pd-l1-assay-comparison-impassion130-jnci-2021","paper-eng-imblaze370-atezolizumab-cobimetinib-colorectal-lancet-oncol-2019"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: the IMpassion130 threshold was SP142 IC 1% or more (46.4% of samples), not transferable to 22C3 or SP263 (Rugo 2021); the US indication was withdrawn in 2021 and the European one remains.","Triple-negative breast cancer: the US indication was withdrawn in 2021 but the EU licence and the NICE recommendation (TA639) stand, so in England atezolizumab with nab-paclitaxel remains a commissioned first-line option for PD-L1 immune-cell-positive disease, and NICE TA801 restricts pembrolizumab to the complementary group (CPS 10 or more with immune-cell staining below 1 percent). In early disease three neoadjuvant or adjuvant phase 3 trials were negative or null (NeoTRIP pathological complete response, GeparDouze event-free survival, ALEXANDRA/IMpassion030 invasive disease-free survival) and the NICE appraisals of atezolizumab for early triple-negative disease (GID-TA10531, GID-TA11165) and with paclitaxel for advanced disease (GID-TA10570) were discontinued.","Colorectal cancer biomarkers: IMblaze370 capped microsatellite instability-high enrolment at 5%, so it is the clean test of checkpoint blockade in microsatellite-stable disease, and it failed: median overall survival 8.87 months with cobimetinib and 7.10 alone against 8.51 with regorafenib (Eng 2019)."],"brand":"Tecentriq / Tecentriq Hybreza (SC)","modality":"Monoclonal antibody (anti-PD-L1)","mechanism":"Fc-engineered humanised IgG1 anti-PD-L1.","approvals":[{"region":"US","year":2016,"indication":"Urothelial carcinoma (later withdrawn); NSCLC"},{"region":"US","year":2026,"indication":"Adjuvant muscle-invasive bladder cancer, ctDNA-positive after cystectomy"},{"region":"EU","year":2019,"indication":"Unresectable locally advanced or metastatic TNBC with PD-L1 expression of 1 percent or more (SP142 immune cells), with nab-paclitaxel, no prior chemotherapy for metastatic disease (IMpassion130); the indication remains in the EU product information on 24 September 2026","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/tecentriq"},{"region":"UK","year":2020,"indication":"Triple-negative unresectable locally advanced or metastatic breast cancer with PD-L1 of 1 percent or more, with nab-paclitaxel, untreated for metastatic disease; NICE TA639 (1 July 2020) recommends within the marketing authorisation with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta639"},{"region":"England (NICE)","year":2018,"indication":"Locally advanced or metastatic non-small-cell lung cancer after chemotherapy","note":"TA520, published 16 May 2018, stopped at 2 years of uninterrupted treatment or earlier on progression (OAK)."},{"region":"England (NICE)","year":2019,"indication":"Metastatic non-squamous non-small-cell lung cancer, with bevacizumab, carboplatin and paclitaxel","note":"TA584, published 5 June 2019, for untreated disease with a PD-L1 tumour proportion score of 0 to 49 percent, or after targeted therapy for EGFR- or ALK-positive disease (IMpower150)."},{"region":"England (NICE)","year":2020,"indication":"Untreated extensive-stage small-cell lung cancer, with carboplatin and etoposide","note":"TA638, published 1 July 2020, only at ECOG performance status 0 or 1 (IMpower133)."},{"region":"England (NICE)","year":2021,"indication":"Untreated metastatic non-small-cell lung cancer with PD-L1 on at least 50 percent of tumour cells or 10 percent of tumour-infiltrating immune cells and no EGFR or ALK alteration","note":"TA705, published 2 June 2021 (IMpower110)."},{"region":"England (NICE)","year":2025,"indication":"Adjuvant treatment of resected non-small-cell lung cancer after platinum chemotherapy, where PD-L1 is on 50 percent or more of tumour cells and the tumour is not EGFR-mutant or ALK-positive","note":"TA1071, published 19 June 2025 (IMpower010); NICE asks that the least expensive suitable option is used."}],"mechanismSteps":["Antibody binds PD-L1 on tumour and immune cells","PD-1/PD-L1 engagement between T cell and tumour is blocked","Exhausted tumour-reactive T cells regain effector function","Interferon-γ and cytotoxic granules are released at the tumour","Tumour cells are killed; memory T cells persist"],"dosing":{"route":"IV infusion (subcutaneous Tecentriq Hybreza available)","schedule":"840 mg every 2 weeks, 1200 mg every 3 weeks, or 1680 mg every 4 weeks","modifications":"Hold for grade 2 immune-mediated events; discontinue for grade 4","monitoring":"Thyroid, LFTs, creatinine, glucose","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee"},"toxicity":[{"event":"Fatigue/asthenia","anyGradePct":48,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"},{"event":"Decreased appetite","anyGradePct":25,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"},{"event":"Nausea","anyGradePct":24,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"},{"event":"Cough","anyGradePct":22,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"},{"event":"Dyspnoea","anyGradePct":22,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"},{"event":"Hypothyroidism (immune-mediated)","anyGradePct":4.9,"grade3PlusPct":0.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"},{"event":"Pneumonitis (immune-mediated)","anyGradePct":3,"grade3PlusPct":0.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"},{"event":"Hepatitis (immune-mediated)","anyGradePct":1.8,"grade3PlusPct":0.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"},{"event":"Colitis (immune-mediated)","anyGradePct":1,"grade3PlusPct":0.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fa682c9-a312-4932-9831-f286908660ee","note":"Monotherapy pooled"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.genentech-access.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended in NSCLC (several TAs), SCLC with chemotherapy, HCC with bevacizumab, adjuvant NSCLC","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2016-05-18","type":"accelerated-approval","region":"US","note":"Urothelial carcinoma after platinum (accelerated; later withdrawn)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Locally advanced or metastatic urothelial carcinoma that progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy"},{"date":"2016-10-18","type":"approval","region":"US","note":"NSCLC after platinum","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2017-04-17","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 as determined by an FDA approved test or who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status"},{"date":"2019-03-08","type":"accelerated-approval","region":"US","note":"PD-L1+ metastatic TNBC with nab-paclitaxel (accelerated; IMpassion130)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with paclitaxel protein-bound for unresectable locally advanced or metastatic triple-negative breast cancer whose tumors express PD-L1 (PD-L1 stained tumor-infiltrating immune cells of any intensity covering = 1% of the tumor area), as determined by an FDA-approved test."},{"date":"2019-03","type":"approval","region":"US","note":"Accelerated approval with nab-paclitaxel for PD-L1-positive (SP142 IC 1 percent or more) unresectable locally advanced or metastatic TNBC on IMpassion130 progression-free survival","source":"https://doi.org/10.1056/NEJMoa1809615"},{"date":"2019-03-18","type":"approval","region":"US","note":"Extensive-stage SCLC with chemotherapy (IMpower133)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-05-29","type":"approval","region":"US","note":"Unresectable HCC with bevacizumab (IMbrave150)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-04-13","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 4.9 years after its accelerated approval.","indication":"Locally advanced or metastatic urothelial carcinoma that progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy"},{"date":"2021-08","type":"withdrawal","region":"US","note":"Genentech withdrew the US TNBC indication after the confirmatory IMpassion131 trial (paclitaxel partner) showed no progression-free or overall survival benefit and the FDA's accelerated-approval review; the current Tecentriq label carries no breast cancer indication","source":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22TECENTRIQ%22"},{"date":"2021-08-27","type":"withdrawal","region":"US","note":"TNBC indication withdrawn after IMpassion131","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-10-06","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 2.6 years after its accelerated approval.","source":"https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-atezolizumab-pd-l1-positive-unresectable-locally-advanced-or-metastatic-triple-negative","indication":"In combination with paclitaxel protein-bound for unresectable locally advanced or metastatic triple-negative breast cancer whose tumors express PD-L1 (PD-L1 stained tumor-infiltrating immune cells of any intensity covering = 1% of the tumor area), as determined by an FDA-approved test."},{"date":"2021-10-15","type":"approval","region":"US","note":"Adjuvant NSCLC, PD-L1 ≥1% (IMpower010)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-12-02","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 5.6 years after its accelerated approval.","indication":"Patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 as determined by an FDA approved test or who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status"},{"date":"2024-09-12","type":"approval","region":"US","note":"Subcutaneous atezolizumab (Tecentriq Hybreza)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q2","type":"approval","region":"US","note":"Adjuvant ctDNA-positive muscle-invasive bladder cancer (IMvigor011): first ctDNA-guided indication","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"atigotatug","kind":"drug","name":"Atigotatug","aka":["BMS-986489 (Atigotatug"],"tldr":"Atigotatug is an experimental monoclonal antibody from Bristol-Myers Squibb in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"Atigotatug (BMS-986489) is a monoclonal antibody developed by Bristol-Myers Squibb. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. The sponsor states: Atigotatug is an anti-fucosyl-GM1 antibody supplied as a fixed-dose combination with the PD-1 inhibitor nivolumab. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06646276 (A Study to Compare the Efficacy and Safety of BMS-986489 (Atigotatug+ Nivolumab Fixed Dose Combination) in Combination With Carboplatin Plus Etoposide to That of Atezolizumab With Carboplatin Plus Etoposide as First-Line Therapy in Participants With Extensive-Stage Small Cell Lung Cancer (TIGOS)), in non-small-cell lung cancer. The largest, NCT06646276, plans to enrol 530 participants with primary completion expected 2028-04-06. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Atigotatug","url":"https://clinicaltrials.gov/search?intr=Atigotatug"},{"label":"Sponsor pipeline page","url":"https://www.bms.com/researchers-and-partners/in-the-pipeline.html"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06646276"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"BMS-986489","modality":"monoclonal antibody","mechanism":"Atigotatug is an anti-fucosyl-GM1 antibody supplied as a fixed-dose combination with the PD-1 inhibitor nivolumab.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"atirmociclib","kind":"drug","name":"Atirmociclib","aka":[],"tldr":"A next-generation pill that blocks only CDK4, not CDK6, to keep the benefit of today's drugs without the low blood counts.","summary":"Pfizer's CDK4-selective inhibitor. Phase 2 FOURLIGHT-1 (second line, with fulvestrant) positive in early 2026 with lower neutropenia than dual inhibitors; phase 3 FOURLIGHT-3 compares atirmociclib + letrozole with a CDK4/6 inhibitor + letrozole in first line. Rationale: CDK6 inhibition drives neutropenia while CDK4 carries most of the antitumour effect in luminal breast cancer.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT06105632 (FOURLIGHT-1)","url":"https://clinicaltrials.gov/study/NCT06105632"}],"tags":[],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["cdk46-inhibitor"],"targets":["cdk4-6"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["fourlight-1","nct04557449","nct04606446","nct06206837","nct07427394","nct05262400","nct06760637"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"PF-07220060","modality":"Small-molecule CDK4-selective inhibitor","mechanism":"ATP-competitive inhibitor selective for CDK4 over CDK6 (~70-fold).","approvals":[],"mechanismSteps":["Cyclin D1-CDK4 phosphorylates RB in luminal breast cancer cells","Atirmociclib blocks CDK4 but largely spares CDK6","RB stays bound to E2F; cells arrest in G1","Haematopoietic progenitors, which depend on CDK6, keep dividing","Less neutropenia allows continuous dosing"],"dosing":{"route":"Oral","schedule":"Twice daily (phase 2/3 dosing under evaluation)"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-02","type":"designation","region":"Global","note":"Positive topline phase 2 FOURLIGHT-1","source":"https://www.pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-topline-phase-2-results-next"}]},{"id":"atrasentan","kind":"drug","name":"Atrasentan","aka":["ABT-627","Xinlay"],"tldr":"The other endothelin blocker. Adding it to chemotherapy for advanced prostate cancer changed nothing, and the trial was stopped for futility.","summary":"Atrasentan is a selective endothelin A receptor antagonist developed by Abbott. It reached phase 3 in prostate cancer twice: alone in men with non-metastatic and metastatic disease, where the results did not support approval and the FDA declined it in 2005, and then in combination with docetaxel.\n\nSWOG S0421 randomised 994 men with metastatic castration-resistant prostate cancer and bone metastases 1:1 to docetaxel 75 mg/m2 every 21 days with atrasentan 10 mg daily or placebo, for up to 12 cycles, stratified by progression type, baseline pain, extraskeletal metastases and bisphosphonate use. The trial was halted early for futility in April 2011.\n\nMedian progression-free survival was 9.2 months (95 percent confidence interval 8.5 to 9.9) with atrasentan and 9.1 months (8.4 to 10.2) with placebo, hazard ratio 1.02 (0.89 to 1.16, p=0.81). Median overall survival was 17.8 months (16.4 to 19.8) against 17.6 (16.4 to 20.1), hazard ratio 1.04 (0.90 to 1.19, p=0.64). Grade 3 or worse toxicity occurred in 57 percent against 60 percent. The separately published patient-reported outcomes found no clinically meaningful difference in pain palliation (41.7 against 44.0 percent) or functional status.\n\nThe endothelin axis is the largest completely negative target hypothesis in prostate cancer: two drugs, five phase 3 trials, no effect.","status":"withdrawn","asOf":"2026-09-25","links":[{"label":"SWOG S0421 (Lancet Oncology 2013)","url":"https://doi.org/10.1016/S1470-2045(13)70294-8"},{"label":"SWOG S0421 patient-reported outcomes (Journal of Patient-Reported Outcomes 2017)","url":"https://doi.org/10.1186/s41687-018-0054-5"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":[],"targets":["ednra"],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":["bone-metastases","castration-resistance","qol-pro"],"trials":["swog-s0421-atrasentan","enthuse-m1-zibotentan"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ABT-627","modality":"small molecule","mechanism":"Selective endothelin A receptor antagonist.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"au-007","kind":"drug","name":"AU-007","aka":[],"tldr":"AU-007 is a monoclonal antibody from Aulos Bioscience, Inc., in registered phase 2 trials for metastatic cancer, melanoma, non-small-cell lung cancer.","summary":"AU-007 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Aulos Bioscience, Inc., in metastatic cancer, melanoma, non-small-cell lung cancer, small-cell lung cancer. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of AU-007","url":"https://clinicaltrials.gov/search?intr=AU-007"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer","melanoma","nsclc","sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["aulos-bioscience"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05267626"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"AU-007","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"aumolertinib","kind":"drug","name":"Aumolertinib","aka":["Aumseqa","Almonertinib"],"tldr":"Aumolertinib is Hansoh's third-generation lung cancer pill, the first China-developed drug of its class, approved for first-line EGFR-mutant lung cancer on the AENEAS trial.","summary":"Approved by the NMPA in March 2020 for EGFR T790M-positive NSCLC after prior EGFR inhibitors and in December 2021 for first-line EGFR-mutant NSCLC on AENEAS (429 patients: median progression-free survival 19.3 versus 9.9 months with gefitinib, hazard ratio 0.46). It has become one of the most prescribed targeted drugs in China after NRDL listing and is under evaluation in Europe. Hansoh is testing it after chemoradiotherapy in stage III disease and in combination regimens.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Almonertinib","links":[{"label":"Hansoh Pharma (page moved; nearest live section)","url":"https://www.hspharm.com/"},{"label":"AENEAS (J Clin Oncol 2022)","url":"https://doi.org/10.1200/JCO.21.02641"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["hansoh-pharma"],"institutions":[],"pathways":["ras-mapk"],"terms":["egfr-mutation-subtypes"],"trials":["aeneas","nct07183189","nct04687241","nct04951635","nct04923906","nct06474455"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ameile","code":"HS-10296, almonertinib","modality":"Small-molecule third-generation EGFR TKI","mechanism":"Irreversible third-generation EGFR inhibitor selective for sensitising and T790M mutations over wild-type EGFR; a cyclopropyl modification of the osimertinib scaffold.","approvals":[{"region":"China","year":2020,"indication":"EGFR T790M-positive locally advanced or metastatic NSCLC after EGFR TKI therapy"},{"region":"China","year":2021,"indication":"First-line EGFR exon 19 deletion or L858R NSCLC (AENEAS)"},{"region":"EU","year":2026,"indication":"Aumseqa: first-line advanced NSCLC with EGFR exon 19 deletion or L858R; advanced EGFR T790M-positive NSCLC"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"autogene-cevumeran","kind":"drug","name":"Autogene cevumeran","aka":[],"tldr":"Autogene cevumeran is BioNTech and Genentech's personalised mRNA vaccine encoding each patient's own tumour neoantigens. In a small phase 1 in resected pancreatic cancer, half of patients mounted T-cell responses and stayed free of recurrence far longer; phase 2 IMCODE003 tests whether that holds.","summary":"Phase 1 (Rojas/Balachandran, Nature 2023; 3-year follow-up 2025): half of resected PDAC patients mounted T-cell responses and had markedly longer recurrence-free survival than non-responders. Phase 2 IMCODE003 in adjuvant PDAC and trials in colorectal (ctDNA+ after surgery) and melanoma. BioNTech/Genentech.","status":"phase-2","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Autogene cevumeran","url":"https://clinicaltrials.gov/search?intr=BNT122"},{"label":"Sethna et al., Nature 2025","url":"https://doi.org/10.1038/s41586-024-08508-4"}],"tags":[],"related":[],"cancers":["pancreatic","resectable-pdac","colorectal","melanoma"],"sections":[],"technologies":["neoantigen-mrna-vaccine"],"targets":[],"drugs":[],"companies":["biontech","roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05968326","autogene-cevumeran-phase-1"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: the phase 1 (16 vaccinated patients) induced T cells in 8, with recurrence-free survival not reached in responders against 13.4 months in non-responders at 3.2 years and vaccine-induced clones estimated to live 7.7 years (Nature 2023 and 2025); IMCODE003 (NCT05968326, 260 patients, disease-free survival, eight UK sites) is active but closed to recruitment."],"code":"BNT122, RO7198457","modality":"Personalised mRNA neoantigen vaccine","mechanism":"Up to 20 neoantigens in uridine mRNA-lipoplex, intravenous, with atezolizumab.","approvals":[],"mechanismSteps":["Up to 20 neoantigens are selected from the resected tumour's mutations","Uridine mRNA-lipoplex is infused intravenously and taken up by splenic dendritic cells","Neoantigen-specific CD8 T cells are induced (in ~half of patients)","Atezolizumab prevents PD-L1-mediated suppression","Responders show delayed recurrence; non-responders do not"],"dosing":{"route":"Intravenous infusion (lipoplex)","schedule":"Priming: 8 weekly doses; boosters at 6-week intervals; with atezolizumab and mFOLFIRINOX in adjuvant PDAC","monitoring":"Infusion reactions, cytokine-related fever","source":"https://clinicaltrials.gov/study/NCT05968326"},"toxicity":[{"event":"Pyrexia"},{"event":"Chills"},{"event":"Fatigue"},{"event":"Infusion-related reactions"}],"access":[{"country":"US","reimbursement":"Investigational (phase 2 IMCODE003)","asOf":"2026-09-06"}],"regulatoryEvents":[]},{"id":"avapritinib","kind":"drug","name":"Avapritinib","aka":[],"tldr":"Avapritinib is the first drug for GIST driven by the PDGFRA D842V mutation, which resists every other kinase inhibitor; it is also approved for systemic mastocytosis.","summary":"Avapritinib is a type I kinase inhibitor that binds the active conformation of KIT and PDGFRA, making it potent against the PDGFRA D842V and KIT exon 17 mutations that resist imatinib and every other approved GIST kinase inhibitor. In NAVIGATOR, patients with PDGFRA exon 18 (D842V) GIST had an ORR of 91%, and the FDA approved it in January 2020 for this group. VOYAGER, against regorafenib in unselected third-line GIST, was negative with PFS 4.2 versus 5.6 months (HR 1.25), so the GIST label remains limited to PDGFRA exon 18 mutations. It is now mainly a mastocytosis drug (PATHFINDER, PIONEER), approved for advanced systemic mastocytosis in 2021. Cognitive effects and a small risk of intracranial haemorrhage mean it is avoided in thrombocytopenia; Blueprint Medicines, acquired by Sanofi in 2025, developed it. It is the one drug that works for a specific GIST mutation nothing else touches.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Avapritinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Avapritinib"}],"tags":[],"related":["pdgfra-exon-18-d842v"],"cancers":["sarcoma","gist-pdgfra-d842v","systemic-mastocytosis","indolent-systemic-mastocytosis","advanced-systemic-mastocytosis"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["kit"],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["voyager","nct03731260"],"people":[],"bottlenecks":[],"keyPapers":["paper-navigator-avapritinib-heinrich-lancet-oncol-2020"],"journals":[],"dependsOn":[],"notes":[],"brand":"Ayvakit","modality":"Small-molecule kinase inhibitor (PDGFRA D842V / KIT)","mechanism":"Type I inhibitor binding the active conformation of KIT/PDGFRA, potent against D842V and exon 17 mutations.","approvals":[{"region":"US","year":2020,"indication":"Unresectable/metastatic GIST with PDGFRA exon 18 mutation including D842V"},{"region":"US","year":2021,"indication":"Advanced systemic mastocytosis"},{"region":"EU","year":2020,"indication":"EU brand Ayvakyt"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"300 mg once daily (GIST); 200 mg (advanced SM); 25 mg (indolent SM)","monitoring":"Cognitive effects, intracranial haemorrhage risk (avoid with thrombocytopenia), oedema"},"toxicity":[{"event":"Cognitive effects","anyGradePct":40,"note":"NAVIGATOR 300-400 mg"},{"event":"Intracranial haemorrhage","anyGradePct":1,"note":"in GIST cohorts"},{"event":"Periorbital oedema","anyGradePct":45}],"access":[],"regulatoryEvents":[]},{"id":"avasopasem-manganese","kind":"drug","name":"Avasopasem manganese","aka":[],"tldr":"Avasopasem is Galera Therapeutics' radioprotector for the severe mouth ulcers of head and neck chemoradiation; its phase 3 trial met its main goal but the FDA asked for another study in 2023.","summary":"Avasopasem manganese is given by infusion before each radiotherapy fraction. The ROMAN phase 3 trial in head and neck cancer patients receiving cisplatin chemoradiation reduced the incidence of severe oral mucositis compared with placebo. The FDA issued a complete response letter in August 2023 asking for an additional trial before approval. A related compound, rucosopasem, is being tested to increase the effect of stereotactic radiotherapy on tumours rather than protect normal tissue.","status":"phase-3","asOf":"2026-09-24","links":[{"label":"ROMAN (eClinicalMedicine 2025)","url":"https://doi.org/10.1016/j.eclinm.2025.103539"},{"label":"ClinicalTrials.gov NCT03689712","url":"https://clinicaltrials.gov/study/NCT03689712"},{"label":"ClinicalTrials.gov: trials of avasopasem","url":"https://clinicaltrials.gov/search?intr=avasopasem"}],"tags":["radiation-wave3"],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":["radioprotectors"],"targets":[],"drugs":[],"companies":["galera-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["roman"],"people":[],"bottlenecks":[],"keyPapers":["paper-roman-anderson-eclinicalmedicine-2025"],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo radiation expansion","editedOn":"2026-09-16"},"code":"GC4419","modality":"Superoxide dismutase mimetic small molecule","supportive":true,"mechanism":"A manganese complex that mimics the enzyme superoxide dismutase, converting the superoxide radicals that radiation creates in normal tissue into hydrogen peroxide, which healthy cells clear more easily than tumour cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"avelumab","kind":"drug","name":"Avelumab","aka":[],"tldr":"Avelumab is a PD-L1 blocker given as maintenance after chemotherapy for advanced bladder cancer, which lengthened survival by about seven months.","summary":"Avelumab is a fully human IgG1 antibody against PD-L1 with an intact Fc region, so it can trigger antibody-dependent cellular cytotoxicity as well as releasing the checkpoint brake. Its main role is maintenance after first-line platinum in advanced urothelial cancer: JAVELIN Bladder 100 showed OS 21.4 versus 14.3 months (HR 0.69), which made avelumab maintenance the standard after platinum in 2020. It is also approved in Merkel cell carcinoma and, with axitinib, in renal cell carcinoma. The maintenance role has been largely displaced in first-line urothelial cancer by EV plus pembrolizumab, but remains relevant where that combination is unavailable or contraindicated. Grade 3 or higher immune-related events occurred in 7%; Merck KGaA and Pfizer developed it. Starting immunotherapy straight after chemotherapy, rather than waiting for relapse, lengthened survival by about seven months.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Avelumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Avelumab"}],"tags":[],"related":[],"cancers":["urothelial","rcc","merkel-cell-carcinoma","tnbc","tnbc-early"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pdl1"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["javelin-bladder-100","nct03228667","nct03260023","nct03547973","nct03815643","nct05327530","a-brave"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Bavencio","modality":"Monoclonal antibody (anti-PD-L1)","mechanism":"Fully human IgG1 anti-PD-L1 with intact Fc (ADCC-capable).","approvals":[{"region":"US","year":2017,"indication":"Merkel cell carcinoma; urothelial cancer after platinum (accelerated)"},{"region":"US","year":2020,"indication":"Maintenance after first-line platinum in advanced urothelial cancer"}],"mechanismSteps":["Binds PD-L1 on tumour and myeloid cells","Restores T-cell activity against residual disease after chemotherapy","Intact Fc may add antibody-dependent cytotoxicity"],"dosing":{"route":"Intravenous","schedule":"800 mg every 2 weeks until progression, started within 10 weeks of last chemotherapy"},"toxicity":[{"event":"Fatigue","anyGradePct":18},{"event":"Pruritus","anyGradePct":17},{"event":"Hypothyroidism","anyGradePct":12},{"event":"Immune-related grade 3+","grade3PlusPct":7}],"access":[],"regulatoryEvents":[{"date":"2017-03-23","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adults and pediatrics patients 12 years and older with metastatic merkel cell carcinoma (MCC)"},{"date":"2017-05-09","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Locally advanced or metastatic urothelial carcinoma following diease progression on platinum-containing chemotherapy or disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy"},{"date":"2020-06-30","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 3.1 years after it was granted.","indication":"Locally advanced or metastatic urothelial carcinoma following diease progression on platinum-containing chemotherapy or disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy"},{"date":"2023-09-06","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 6.5 years after it was granted.","indication":"Treatment of adults and pediatrics patients 12 years and older with metastatic merkel cell carcinoma (MCC)"}]},{"id":"avutometinib-defactinib","kind":"drug","name":"Avutometinib + defactinib","aka":["Avutometinib","Defactinib","Avutometinib and defactinib"],"tldr":"Avutometinib plus defactinib is the first treatment approved specifically for low-grade serous ovarian cancer, a slow-growing type driven by the RAS pathway.","summary":"Accelerated approval 8 May 2025 for adults with KRAS-mutated recurrent low-grade serous ovarian cancer after prior systemic therapy, based on RAMP 201 (ORR 44%, median DOR 31.1 months in KRAS-mutant disease). Avutometinib inhibits MEK and blocks RAF-driven MEK reactivation; defactinib blocks FAK, a resistance node. RAMP 301 (phase 3) is confirmatory; combinations in KRAS-mutant lung and pancreatic cancer are being explored.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Avutometinib"}],"tags":[],"related":[],"cancers":["ovarian","low-grade-serous-ovarian-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["kras","fak"],"drugs":[],"companies":["verastem"],"institutions":[],"pathways":["ras-mapk"],"terms":["lgsoc"],"trials":["ramp-201"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Avmapki Fakzynja Co-Pack","code":"VS-6766 + VS-6063","modality":"Small-molecule RAF/MEK clamp + FAK inhibitor","mechanism":"Avutometinib is a dual RAF/MEK 'clamp' that prevents compensatory MEK phosphorylation; defactinib inhibits focal adhesion kinase, a feedback survival signal.","approvals":[{"region":"US","year":2025,"indication":"KRAS-mutated recurrent low-grade serous ovarian cancer after prior systemic therapy (accelerated)"}],"mechanismSteps":["Avutometinib binds MEK and locks RAF-MEK in an inactive complex","ERK signalling from mutant KRAS drops without feedback rebound","Defactinib blocks FAK, removing an adhesion-driven escape route","LGSOC cells arrest and regress over months"],"dosing":{"route":"Oral","schedule":"Avutometinib 3.2 mg twice weekly + defactinib 200 mg twice daily, 3 weeks on / 1 week off","monitoring":"Creatine kinase, liver enzymes, eye exams, skin","source":"https://www.onclive.com/view/accelerated-fda-approval-establishes-role-of-avutometinib-defactinib-in-kras-mutant-recurrent-low-grade-serous-ovarian-cancer"},"toxicity":[{"event":"Nausea","anyGradePct":67},{"event":"Diarrhoea","anyGradePct":58},{"event":"Rash / dermatitis acneiform","anyGradePct":50},{"event":"Creatine kinase increase","anyGradePct":61,"note":"RAMP 201 pooled, per FDA label summary"}],"access":[],"regulatoryEvents":[{"date":"2025-05-08","type":"accelerated-approval","region":"US","note":"Accelerated approval on RAMP 201; RAMP 301 confirmatory The confirmatory requirement was still open 1.4 years later, when the FDA's table was read.","source":"https://www.targetedonc.com/view/avutometinib-plus-defactinib-gains-fda-approval-in-kras-ovarian-cancer","indication":"Treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy"}]},{"id":"avzo-021","kind":"drug","name":"AVZO-021","aka":[],"tldr":"AVZO-021 is an experimental small-molecule drug from Avenzo Therapeutics in phase 2 trials for HR-positive / HER2-negative breast cancer, ovarian cancer and endometrial cancer, aimed at HER2.","summary":"AVZO-021 is a small-molecule drug developed by Avenzo Therapeutics. Its target is HER2 (the sponsor names CDK2). The sponsor states: AVZO-021 is a highly potent and selective oral CDK2 inhibitor targeting the cyclin E-CDK2 pathway, which becomes hyperactivated as a resistance mechanism to CDK4/6 inhibitors in HR+/HER2- breast cancer and other CCNE1-amplified solid tumours. ClinicalTrials.gov describes the intervention as: AVZO-021 is an oral selective CDK2 inhibitor. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in HR-positive / HER2-negative breast cancer, ovarian cancer, endometrial cancer and triple-negative breast cancer. The largest, NCT05867251, plans to enrol 430 participants with primary completion expected 2028-01-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AVZO-021","url":"https://clinicaltrials.gov/search?intr=AVZO-021"},{"label":"Sponsor pipeline page","url":"https://avenzotx.com/pipeline-and-science/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","ovarian","endometrial","tnbc"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["avenzo"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05867251"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"AVZO-021 is a highly potent and selective oral CDK2 inhibitor targeting the cyclin E-CDK2 pathway, which becomes hyperactivated as a resistance mechanism to CDK4/6 inhibitors in HR+/HER2- breast cancer and other CCNE1-amplified solid tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"avzo-023","kind":"drug","name":"AVZO-023","aka":[],"tldr":"AVZO-023 is a small-molecule inhibitor from Avenzo Therapeutics, Inc., in registered phase 2 trials for metastatic cancer.","summary":"AVZO-023 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Avenzo Therapeutics, Inc., in metastatic cancer. Described in the registry record as a avzo-023 is an oral selective cdk4 inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of AVZO-023","url":"https://clinicaltrials.gov/search?intr=AVZO-023"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":["cdk4-6"],"drugs":[],"companies":["avenzo"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06998407"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"AVZO-023","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a avzo-023 is an oral selective cdk4 inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"axicabtagene-ciloleucel","kind":"drug","name":"Axicabtagene ciloleucel","aka":[],"tldr":"A CD19 CAR-T that cures about 40% of patients with large B-cell lymphoma who had failed everything, and beat transplant in second line.","summary":"Axicabtagene ciloleucel (axi-cel) is an autologous CAR-T: a patient's T cells are engineered with a retroviral vector to express a CD19-binding scFv with a CD28 costimulatory domain and reinfused after cyclophosphamide and fludarabine lymphodepletion. It was approved in 2017 for large B-cell lymphoma after two or more lines (ZUMA-1; 5-year OS about 43%), in 2022 as second-line therapy for early relapse (ZUMA-7, OS benefit), and for follicular lymphoma. Kite/Gilead manufactures it. CD28 costimulation gives fast expansion and more neurotoxicity than 4-1BB products; cytokine release syndrome, prolonged cytopenias and infections are the other main risks. How to choose between CAR-T and CD20xCD3 bispecifics, and how to widen access, remain open. For a newcomer: a one-time living drug that cures a meaningful share of lymphomas with no standard treatment left to try.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Axicabtagene_ciloleucel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Axicabtagene%20ciloleucel"},{"label":"Deng et al., Nat Med 2020: single-cell features of the axicabtagene ciloleucel infusion product that track efficacy and toxicity in 24 patients","url":"https://doi.org/10.1038/s41591-020-1061-7"},{"label":"Plaks et al., Blood 2021: CD19 target evasion as a mechanism of relapse after axicabtagene ciloleucel in large B-cell lymphoma","url":"https://doi.org/10.1182/blood.2021010930"},{"label":"Sotillo et al., Cancer Discov 2015: acquired mutations and alternative splicing of CD19 enable resistance to CART-19","url":"https://doi.org/10.1158/2159-8290.CD-15-1020"}],"tags":[],"related":[],"cancers":["dlbcl","primary-mediastinal-b-cell-lymphoma"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":[],"companies":["gilead"],"institutions":[],"pathways":[],"terms":["lymphoma-bio-antigen-escape"],"trials":["nct05371093"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: half the medicine is the patient's own T cells. Single-cell sequencing of 24 infusion products found three times the frequency of memory-signature CD8 T cells in patients who were in complete response at three months, and an exhaustion signature associated with a poor molecular response in cell-free DNA at day 7; a rare monocyte-like population in the product was associated with high-grade neurotoxicity (Deng 2020). The other failure mode is CD19 target evasion (Plaks 2021, Sotillo 2015)."],"brand":"Yescarta","code":"axi-cel","modality":"CAR-T (CD19)","mechanism":"CD19 scFv, CD28 costimulation, retroviral.","approvals":[{"region":"US","year":2017,"indication":"Relapsed/refractory large B-cell lymphoma ≥2 lines"},{"region":"US","year":2022,"indication":"Second-line LBCL refractory or relapsed within 12 months"}],"mechanismSteps":["Patient's T cells are collected by leukapheresis","Cells are engineered to express a CAR against CD19 and expanded","Patient receives lymphodepleting chemotherapy","CAR-T cells are infused, home to tumour, and expand","CAR binds CD19; T cell kills the tumour cell and proliferates","Memory CAR-T cells persist and patrol"],"dosing":{"route":"Single IV infusion after lymphodepletion","schedule":"2 × 10⁶ CAR+ T cells/kg (max 2 × 10⁸); cyclophosphamide 500 mg/m² + fludarabine 30 mg/m² days −5 to −3","modifications":"Tocilizumab ± steroids for CRS; steroids for ICANS; prophylactic steroids permitted","monitoring":"Daily monitoring for 7 days (outpatient permitted with rapid access), then twice weekly for 4 weeks; cytopenias, infections, B-cell aplasia","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Cytokine release syndrome","note":"ZUMA-7: ~92% any grade, 6% grade ≥3"},{"event":"Neurologic toxicity","note":"ZUMA-7: ~60% any grade, 21% grade ≥3"},{"event":"Prolonged cytopenias"},{"event":"Infections"},{"event":"Hypogammaglobulinaemia"}],"access":[{"country":"US","listPrice":"$373,000 per infusion (list price at launch 2017; higher today)","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.kitekonnect.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for relapsed/refractory DLBCL after ≥2 therapies (TA872) and second-line early relapse (TA895)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-10-18","type":"approval","region":"US","note":"Relapsed/refractory large B-cell lymphoma after ≥2 lines (ZUMA-1): second CAR-T ever approved","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-03-05","type":"accelerated-approval","region":"US","note":"Relapsed/refractory follicular lymphoma (ZUMA-5) The confirmatory requirement was still open 5.5 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy"},{"date":"2022-04-01","type":"approval","region":"US","note":"Second-line LBCL refractory or relapsed within 12 months (ZUMA-7)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-01-19","type":"label-change","region":"US","note":"Class boxed warning for T-cell malignancies","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"axitinib","kind":"drug","name":"Axitinib","aka":[],"tldr":"Axitinib is a selective VEGF-receptor pill, now given mainly with pembrolizumab or avelumab as first-line kidney cancer treatment.","summary":"Axitinib is a potent, selective inhibitor of VEGFR1-3 that blocks the angiogenic signalling clear-cell kidney cancers depend on. In AXIS (2011) it gave PFS 6.7 versus 4.7 months against sorafenib in second line, earning approval in 2012. Its main use now is first line in combination: it was the partner in KEYNOTE-426 with pembrolizumab, which improved OS against sunitinib (final medians 47.2 versus 40.8 months; PFS 15.7 versus 11.1 months), and in JAVELIN Renal 101 with avelumab, which improved PFS. Dosing starts at 5 mg twice daily and is titrated to 7 then 10 mg if blood pressure allows; its short half-life permits titration and rapid washout, which helps distinguish immune hepatitis from TKI hepatitis. Diarrhoea (55%) and hypertension (40%) are the common effects. Axitinib is the anti-angiogenic pill most often paired with immunotherapy at the start of kidney cancer treatment.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Axitinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Axitinib"}],"tags":[],"related":[],"cancers":["rcc","clear-cell-rcc","adenoid-cystic-carcinoma"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["keynote-426","nct07227415","nct05805501","nct06962787"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Inlyta","modality":"Small-molecule kinase inhibitor (VEGFR)","mechanism":"Potent selective VEGFR1-3 inhibitor.","approvals":[{"region":"US","year":2012,"indication":"Advanced RCC after one prior systemic therapy"},{"region":"US","year":2019,"indication":"First-line advanced RCC with pembrolizumab or avelumab"}],"mechanismSteps":["Oral, twice-daily dosing with short half-life","Blocks VEGFR2 on endothelial cells","Anti-angiogenesis normalises tumour vasculature, aiding T-cell entry when combined with PD-1 blockade"],"dosing":{"route":"Oral","schedule":"5 mg twice daily; titrate to 7 then 10 mg if tolerated (no hypertension)","modifications":"Hold for hypertension crisis, proteinuria, or to distinguish immune from TKI hepatitis","monitoring":"Blood pressure, thyroid, urine protein, liver enzymes"},"toxicity":[{"event":"Hypertension","anyGradePct":40},{"event":"Diarrhoea","anyGradePct":55},{"event":"Fatigue","anyGradePct":40},{"event":"Dysphonia","anyGradePct":30},{"event":"Hand-foot syndrome","anyGradePct":28}],"access":[],"regulatoryEvents":[]},{"id":"azacitidine","kind":"drug","name":"Azacitidine","aka":[],"tldr":"A gentle chemotherapy that switches silenced genes back on. With venetoclax it became the standard for older people with AML who cannot take intensive treatment.","summary":"Approved for MDS (2004) and, in combination with venetoclax, for newly diagnosed AML in patients unfit for intensive chemotherapy (VIALE-A, 2020). Oral azacitidine (Onureg, CC-486) is approved as maintenance after intensive induction (QUAZAR AML-001: OS 24.7 vs 14.8 months). Backbone partner for IDH inhibitors (AGILE), menin inhibitors, and FLT3 inhibitors in trials.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Azacitidine","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Azacitidine"}],"tags":[],"related":[],"cancers":["aml","aml-older-unfit","mds-higher-risk"],"sections":[],"technologies":["epigenetic-drugs","cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":["apoptosis-bcl2"],"terms":[],"trials":["viale-a","agile","nct04501120","nct04068597","nct04988555","nct04730258","nct05673057","nct07007312","nct04588922","nct02677922","nct05883956","nct04771130","nct04229979","nct04256317","nct07743112","nct04980885","nct06858618","nct05907057","nct04581512","nct05520567","nct07581002","nct04086264","nct04023526","nct07255872","nct06389292","nct06456463","nct05197426","nct07761533"],"people":[],"bottlenecks":[],"keyPapers":["paper-aza-001-fenaux-lancet-oncol-2009"],"journals":[],"dependsOn":[],"notes":[],"brand":"Vidaza / Onureg (oral)","modality":"Small-molecule hypomethylating agent (cytotoxic)","mechanism":"Cytidine analogue incorporated into RNA and DNA; traps and depletes DNA methyltransferases, reactivating silenced tumour-suppressor genes and inducing differentiation and apoptosis.","approvals":[{"region":"US","year":2004,"indication":"Myelodysplastic syndromes"},{"region":"US","year":2020,"indication":"Newly diagnosed AML unfit for intensive chemotherapy, with venetoclax (VIALE-A)"},{"region":"US","year":2020,"indication":"Oral azacitidine (Onureg) maintenance after intensive induction (QUAZAR AML-001)"}],"mechanismSteps":["Azacitidine enters the cell and is phosphorylated","Incorporated mostly into RNA (protein synthesis disrupted) and partly into DNA","DNMT1 binds the incorporated base and is trapped and degraded","Daughter cells lose methylation at silenced promoters","Tumour-suppressor and differentiation genes are re-expressed; blasts die or mature"],"dosing":{"route":"Subcutaneous or IV (Vidaza); oral (Onureg)","schedule":"75 mg/m² daily for 7 days of each 28-day cycle (with venetoclax in AML); Onureg 300 mg daily for 14 of 28 days as maintenance","monitoring":"Blood counts each cycle; renal function","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=azacitidine"},"toxicity":[{"event":"Neutropenia","grade3PlusPct":42,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=azacitidine","note":"VIALE-A combination arm"},{"event":"Febrile neutropenia","grade3PlusPct":42,"note":"VIALE-A combination arm"},{"event":"Nausea","anyGradePct":44},{"event":"Injection-site reactions","anyGradePct":30,"note":"subcutaneous route"}],"access":[],"regulatoryEvents":[{"date":"2004-05-19","type":"approval","region":"US","note":"First hypomethylating agent approved (MDS)"},{"date":"2020-09-01","type":"approval","region":"US","note":"Onureg maintenance in AML"},{"date":"2020-10-16","type":"approval","region":"US","note":"Venetoclax + azacitidine full approval in unfit AML (VIALE-A)"}]},{"id":"azd0120","kind":"drug","name":"AZD0120","aka":[],"tldr":"AZD0120 is a car-t cell therapy from AstraZeneca, in registered phase 3 trials for multiple myeloma.","summary":"AZD0120 is listed on ClinicalTrials.gov as an intervention in 2 registered phase 3 trials sponsored by AstraZeneca and Alexion Pharmaceuticals, Inc., in multiple myeloma. A chimeric antigen receptor T-cell therapy, as described in the registry record: the patient's T cells are engineered to recognise a tumour antigen and infused back. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of AZD0120","url":"https://clinicaltrials.gov/search?intr=AZD0120"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":["cd19","bcma"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07391657","nct07764978","nct05850234"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"AZD0120","modality":"CAR-T cell therapy","mechanism":"A chimeric antigen receptor T-cell therapy, as described in the registry record: the patient's T cells are engineered to recognise a tumour antigen and infused back.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd0486","kind":"drug","name":"AZD0486","aka":[],"tldr":"AZD0486 is an experimental bispecific antibody from AstraZeneca in phase 3 trials for diffuse large B-cell lymphoma, aimed at CD19 and CD3.","summary":"AZD0486 (TNB-486) is a bispecific antibody developed by AstraZeneca. Its targets are CD19 and CD3 (the sponsor names CD19 x CD3). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07215585 (AZD0486 1L Therapy for Elderly or Unfit Participants With LBCL), in diffuse large B-cell lymphoma. The largest, NCT07215585, plans to enrol 420 participants with primary completion expected 2030-06-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD0486","url":"https://clinicaltrials.gov/search?intr=TNB-486"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":[],"targets":["cd19","cd3"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07215585","nct06549595","nct07509151","nct06526793","nct06564038"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"TNB-486","modality":"bispecific antibody","mechanism":"Bispecific antibody directed at CD19 and CD3, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd3470","kind":"drug","name":"AZD3470","aka":[],"tldr":"AZD3470 is an experimental small-molecule drug from AstraZeneca in phase 2 trials for hodgkin lymphoma and peripheral T-cell lymphomas, aimed at PRMT5 (MTAP-deleted cancers).","summary":"AZD3470 is a small-molecule drug developed by AstraZeneca. Its target is PRMT5 (MTAP-deleted cancers) (the sponsor names PRMT5). The sponsor states: AZD3470 is a second-generation, MTAP-selective, MTA-cooperative inhibitor of PRMT5, designed for tumours with MTAP deletion/deficiency. ClinicalTrials.gov describes the intervention as: AZD3470 is a novel, potent and selective, second-generation, MTAP-selective, inhibitor of PRMT5. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in hodgkin lymphoma and peripheral T-cell lymphomas. The largest, NCT06130553, plans to enrol 334 participants with primary completion expected 2028-12-04. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD3470","url":"https://clinicaltrials.gov/search?intr=AZD3470"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma","peripheral-t-cell-lymphoma"],"sections":[],"technologies":[],"targets":["prmt5-mtap"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06137144","nct06130553"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"AZD3470 is a second-generation, MTAP-selective, MTA-cooperative inhibitor of PRMT5, designed for tumours with MTAP deletion/deficiency.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd4045","kind":"drug","name":"AZD4045","aka":[],"tldr":"AZD4045 is an experimental CAR-T cell therapy from AstraZeneca in phase 2 trials for multiple myeloma, aimed at BCMA.","summary":"AZD4045 is a CAR-T cell therapy developed by AstraZeneca. Its target is BCMA (the sponsor names BCMA). The sponsor states: An allogeneic chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen (BCMA). ClinicalTrials.gov describes the intervention as: Allogeneic Chimeric Antigen Receptor T cell (CAR-T) Therapy Targeting B Cell Maturation Antigen (BCMA). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in multiple myeloma. The largest, NCT07681596, plans to enrol 101 participants with primary completion expected 2031-03-06. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD4045","url":"https://clinicaltrials.gov/search?intr=AZD4045"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":["bcma"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07681596"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"An allogeneic chimeric antigen receptor (CAR) T-cell therapy targeting B-cell maturation antigen (BCMA).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd4360","kind":"drug","name":"AZD4360","aka":[],"tldr":"AZD4360 is an experimental antibody-drug conjugate from AstraZeneca in phase 2 trials for gastric & gastro-oesophageal junction cancer, biliary tract cancer and pancreatic ductal adenocarcinoma, aimed at Claudin 18.2.","summary":"AZD4360 is an antibody-drug conjugate developed by AstraZeneca. Its target is Claudin 18.2 (the sponsor names Claudin 18.2 (CLDN18.2)). The sponsor states: AZD4360 is an antibody-drug conjugate targeting Claudin 18.2, being studied in advanced solid tumours including gastric, gastro-oesophageal junction, biliary tract and pancreatic cancers. ClinicalTrials.gov describes the intervention as: Antibody-Drug Conjugate targeting Claudin 18.2 (CLDN18.2). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in gastric & gastro-oesophageal junction cancer, biliary tract cancer and pancreatic ductal adenocarcinoma. The largest, NCT06921928, plans to enrol 31 participants (actual) with primary completion expected 2027-03-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD4360","url":"https://clinicaltrials.gov/search?intr=AZD4360"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric","cholangiocarcinoma","pancreatic"],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06921928"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["23 Sept 2026: the payload is not stated on any record OnCo reads, so the open drug engine shows AZD4360 under Claudin 18.2 with the payload class not recorded rather than guessing."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"AZD4360 is an antibody-drug conjugate targeting Claudin 18.2, being studied in advanced solid tumours including gastric, gastro-oesophageal junction, biliary tract and pancreatic cancers.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd4512","kind":"drug","name":"AZD4512","aka":[],"tldr":"AZD4512 is an experimental antibody-drug conjugate from AstraZeneca in phase 2 trials for hodgkin lymphoma, aimed at CD22.","summary":"AZD4512 is an antibody-drug conjugate developed by AstraZeneca. Its target is CD22 (the sponsor names CD22). The sponsor states: An antibody-drug conjugate targeting CD22, administered by intravenous infusion; specific payload/mechanism detail beyond CD22 targeting was not stated on the fetched page. ClinicalTrials.gov describes the intervention as: AZD4512 is an antibody-drug conjugate targeting cluster of differentiation 22 (CD22) that will be administered via IV infusion. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in hodgkin lymphoma. The largest, NCT07123454, plans to enrol 91 participants with primary completion expected 2028-03-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD4512","url":"https://clinicaltrials.gov/search?intr=AZD4512"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":["cd22"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07123454","nct07109219"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"An antibody-drug conjugate targeting CD22, administered by intravenous infusion; specific payload/mechanism detail beyond CD22 targeting was not stated on the fetched page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd5335","kind":"drug","name":"AZD5335","aka":[],"tldr":"AZD5335 is an experimental antibody-drug conjugate from AstraZeneca in phase 3 trials for ovarian cancer, with its target not yet stated publicly.","summary":"AZD5335 is an antibody-drug conjugate developed by AstraZeneca. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07218809 (AZD5335 vs. Mirvetuximab Soravtansine in FRα-high and AZD5335 vs. Chemotherapy in FRα-low Platinum-resistant Ovarian Cancer), in ovarian cancer. The largest, NCT07218809, plans to enrol 1100 participants with primary completion expected 2028-11-17. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD5335","url":"https://clinicaltrials.gov/search?intr=AZD5335"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07218809","nct05797168"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd5492","kind":"drug","name":"AZD5492","aka":[],"tldr":"AZD5492 is an experimental T-cell engager from AstraZeneca in phase 2 trials, aimed at CD20.","summary":"AZD5492 is a T-cell engager developed by AstraZeneca. Its target is CD20 (the sponsor names CD20). The sponsor states: A CD8/TCR-based T-cell engaging antibody targeting CD20, administered subcutaneously to redirect T cells against CD20-positive B-cell malignancies. ClinicalTrials.gov describes the intervention as: CD8/TCR based T-cell engaging antibody targeting CD20, which is administered subcutaneously. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT06542250, plans to enrol 174 participants with primary completion expected 2029-12-18. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD5492","url":"https://clinicaltrials.gov/search?intr=AZD5492"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["cd20"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06542250"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"T-cell engager","mechanism":"A CD8/TCR-based T-cell engaging antibody targeting CD20, administered subcutaneously to redirect T cells against CD20-positive B-cell malignancies.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd5863","kind":"drug","name":"AZD5863","aka":[],"tldr":"AZD5863 is an experimental bispecific antibody from AstraZeneca in phase 2 trials for gastric & gastro-oesophageal junction cancer, pancreatic ductal adenocarcinoma and oesophageal cancer, aimed at Claudin 18.2 and CD3.","summary":"AZD5863 is a bispecific antibody developed by AstraZeneca. Its targets are Claudin 18.2 and CD3 (the sponsor names CLDN18.2 x CD3). The sponsor states: T cell-engaging bispecific antibody that binds Claudin 18.2 on tumour cells and CD3 on T cells, bridging them to drive immune-mediated tumour cell killing. ClinicalTrials.gov describes the intervention as: T cell-engaging bi-specific antibody that targets CLDN18.2 (Claudin18.2) on tumour cells and CD3 on T cells. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in gastric & gastro-oesophageal junction cancer, pancreatic ductal adenocarcinoma and oesophageal cancer. The largest, NCT06005493, plans to enrol 280 participants with primary completion expected 2027-07-16. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD5863","url":"https://clinicaltrials.gov/search?intr=AZD5863"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric","pancreatic","esophageal"],"sections":[],"technologies":[],"targets":["cldn18-2","cd3"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06005493"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"T cell-engaging bispecific antibody that binds Claudin 18.2 on tumour cells and CD3 on T cells, bridging them to drive immune-mediated tumour cell killing.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd6621","kind":"drug","name":"AZD6621","aka":[],"tldr":"AZD6621 is an experimental T-cell engager from AstraZeneca in phase 2 trials for prostate cancer, aimed at CD3.","summary":"AZD6621 is a T-cell engager developed by AstraZeneca. Its target is CD3 (the sponsor names STEAP2 x CD3 x CD8). The sponsor states: AZD6621 is a T-cell-engaging antibody that targets STEAP2, CD3 and CD8, designed to engage T cells against STEAP2-expressing prostate cancer cells (ACTIVATED-4-PC study). ClinicalTrials.gov describes the intervention as: A T Cell-engaging Antibody that targets STEAP2, CD3, and CD8. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in prostate cancer. The largest, NCT07192614, plans to enrol 52 participants with primary completion expected 2029-03-29. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD6621","url":"https://clinicaltrials.gov/search?intr=AZD6621"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["cd3"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07192614"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"T-cell engager","mechanism":"AZD6621 is a T-cell-engaging antibody that targets STEAP2, CD3 and CD8, designed to engage T cells against STEAP2-expressing prostate cancer cells (ACTIVATED-4-PC study).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd7789","kind":"drug","name":"AZD7789","aka":[],"tldr":"AZD7789 is an experimental bispecific antibody from AstraZeneca in phase 2 trials for non-small-cell lung cancer and gastric & gastro-oesophageal junction cancer, aimed at PD-1 and TIM-3.","summary":"AZD7789 is a bispecific antibody developed by AstraZeneca. Its targets are PD-1 and TIM-3 (the sponsor names PD-1 x TIM-3). The sponsor states: An anti-PD-1 and anti-TIM-3 bispecific antibody that simultaneously engages both immune checkpoints to enhance anti-tumour immunity. ClinicalTrials.gov describes the intervention as: anti-PD-1 and anti-TIM-3 bispecific antibody. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer and gastric & gastro-oesophageal junction cancer. The largest, NCT04931654, plans to enrol 136 participants (actual) with primary completion was scheduled for 2024-12-04 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD7789","url":"https://clinicaltrials.gov/search?intr=AZD7789"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","gastric"],"sections":[],"technologies":[],"targets":["pd1","tim3"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04931654"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"An anti-PD-1 and anti-TIM-3 bispecific antibody that simultaneously engages both immune checkpoints to enhance anti-tumour immunity.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd8421","kind":"drug","name":"AZD8421","aka":[],"tldr":"AZD8421 is a small-molecule inhibitor from AstraZeneca, in registered phase 2 trials for ovarian cancer.","summary":"AZD8421 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by AstraZeneca, in ovarian cancer. Described in the registry record as a cdk2 inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of AZD8421","url":"https://clinicaltrials.gov/search?intr=AZD8421"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06188520"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"AZD8421","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a cdk2 inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd9750","kind":"drug","name":"AZD9750","aka":[],"tldr":"AZD9750 is an experimental protein degrader from AstraZeneca in phase 2 trials for prostate cancer, aimed at Androgen receptor.","summary":"AZD9750 is a protein degrader developed by AstraZeneca. Its target is Androgen receptor (the sponsor names androgen receptor (AR)). The sponsor states: An AR-PROTAC that binds the androgen receptor and recruits it for proteasomal degradation, rather than simply inhibiting its function. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in prostate cancer. The largest, NCT07336446, plans to enrol 300 participants with primary completion expected 2029-01-26. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD9750","url":"https://clinicaltrials.gov/search?intr=AZD9750"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["androgen-receptor"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07336446"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"protein degrader","mechanism":"An AR-PROTAC that binds the androgen receptor and recruits it for proteasomal degradation, rather than simply inhibiting its function.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azd9793","kind":"drug","name":"AZD9793","aka":[],"tldr":"AZD9793 is an experimental T-cell engager from AstraZeneca in phase 2 trials for hepatocellular carcinoma, aimed at Glypican-3.","summary":"AZD9793 is a T-cell engager developed by AstraZeneca. Its target is Glypican-3 (the sponsor names GPC3). The sponsor states: AZD9793 is a T-cell-engaging antibody that targets GPC3 on tumour cells, dosed intravenously and subcutaneously in the first-in-human RHEA-1 study, with a focus on hepatocellular carcinoma. ClinicalTrials.gov describes the intervention as: T cell-engaging antibody that targets GPC3 on tumour cells. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in hepatocellular carcinoma. The largest, NCT06795022, plans to enrol 304 participants with primary completion expected 2027-06-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of AZD9793","url":"https://clinicaltrials.gov/search?intr=AZD9793"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hcc"],"sections":[],"technologies":[],"targets":["gpc3"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06795022"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"T-cell engager","mechanism":"AZD9793 is a T-cell-engaging antibody that targets GPC3 on tumour cells, dosed intravenously and subcutaneously in the first-in-human RHEA-1 study, with a focus on hepatocellular carcinoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"azenosertib","kind":"drug","name":"Azenosertib","aka":["ZN-c3"],"tldr":"Azenosertib is an experimental small-molecule drug from K-, Beta in phase 3 trials for ovarian cancer, aimed at WEE1.","summary":"Azenosertib is a small-molecule drug developed by K-, Beta. Its target is WEE1 (the sponsor names WEE1). The sponsor states: Azenosertib (ZN-c3) is a selective, orally bioavailable WEE1 inhibitor that promotes premature cell-cycle progression and DNA damage/mitotic catastrophe in tumours with high cyclin E1 expression; tested against investigator's choice chemotherapy in platinum-resistant, cyclin E1-positive ovarian, primary peritoneal, or fallopian tube cancer. ClinicalTrials.gov describes the intervention as: Azenosertib 400 mg will be administered orally. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07546500 (A Study of Azenosertib (ZN-c3) Versus Investigator's Choice Chemotherapy in Subjects With Platinum-Resistant High-Grade Serous Ovarian, Primary Peritoneal, or Fallopian Tube Cancers Positive for Cyclin E1 Protein Expression), in ovarian cancer. The largest, NCT07546500, plans to enrol 420 participants with primary completion expected 2028-05-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Azenosertib","url":"https://clinicaltrials.gov/search?intr=Azenosertib"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":["wee1"],"drugs":[],"companies":["zentalis-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07546500","nct05128825"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Azenosertib (ZN-c3) is a selective, orally bioavailable WEE1 inhibitor that promotes premature cell-cycle progression and DNA damage/mitotic catastrophe in tumours with high cyclin E1 expression; tested against investigator's choice chemotherapy in platinum-resistant, cyclin E1-positive ovarian, primary peritoneal, or fallopian tube cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ba3071","kind":"drug","name":"BA3071","aka":[],"tldr":"BA3071 is an experimental monoclonal antibody from BioAtla in phase 2 trials for non-small-cell lung cancer and melanoma, aimed at CTLA-4.","summary":"BA3071 is a monoclonal antibody developed by BioAtla. Its target is CTLA-4 (the sponsor names CTLA-4). The sponsor states: A Conditionally Active Biologic (CAB-C) anti-CTLA-4 antibody engineered to bind and block CTLA-4 preferentially within the tumour microenvironment (rather than systemically), activating T cells locally to reduce systemic toxicity. ClinicalTrials.gov describes the intervention as: Conditionally active biologic (CAB) antibody that binds to CTLA-4. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer and melanoma. The largest, NCT05180799, plans to enrol 320 participants with primary completion was scheduled for 2025-03-19 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BA3071","url":"https://clinicaltrials.gov/search?intr=BA3071"},{"label":"Sponsor pipeline page","url":"https://www.bioatla.com/cab-portfolio/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","melanoma"],"sections":[],"technologies":[],"targets":["ctla4"],"drugs":[],"companies":["bioatla"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05180799"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"A Conditionally Active Biologic (CAB-C) anti-CTLA-4 antibody engineered to bind and block CTLA-4 preferentially within the tumour microenvironment (rather than systemically), activating T cells locally to reduce systemic toxicity.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"balstilimab","kind":"drug","name":"Balstilimab","aka":["AGEN2034"],"tldr":"Balstilimab is a monoclonal antibody from Agenus Inc., in registered phase 2 trials for colorectal cancer.","summary":"Balstilimab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Agenus Inc. and ImmunoGenesis, in colorectal cancer. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Balstilimab","url":"https://clinicaltrials.gov/search?intr=Balstilimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["pd1"],"drugs":[],"companies":["agenus"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05608044","nct05205330","nct06782555"],"people":[],"bottlenecks":[],"keyPapers":["paper-bullock-botensilimab-balstilimab-mss-colorectal-nat-med-2024"],"journals":[],"dependsOn":[],"notes":["Colorectal cancer: the anti-PD-1 half of the botensilimab pairing tested in 148 heavily pre-treated microsatellite-stable patients, where the combination gave a 17% objective response rate and 61% disease control (Bullock 2024)."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bcb-276","kind":"drug","name":"BCB-276","aka":[],"tldr":"BCB-276 is an experimental CAR-T cell therapy from BrainChild Bio in phase 2 trials for glioma & glioblastoma, aimed at B7-H3.","summary":"BCB-276 is a CAR-T cell therapy developed by BrainChild Bio. Its target is B7-H3 (the sponsor names B7-H3). The sponsor states: An autologous CAR T-cell therapy targeting B7-H3, an antigen described as universally expressed in diffuse intrinsic pontine glioma (DIPG), manufactured from the patient's own leukapheresed immune cells after standard radiation therapy. ClinicalTrials.gov describes the intervention as: Following completion of standard radiation therapy, eligible participants with DIPG will undergo leukapheresis, a procedure to collect white blood cells used to manufacture BCB-276, an autologous CAR T cell therapy made from the participant. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in glioma & glioblastoma. The largest, NCT07680439, plans to enrol 75 participants with primary completion expected 2028-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BCB-276","url":"https://clinicaltrials.gov/search?intr=BCB-276"},{"label":"Sponsor pipeline page","url":"https://brainchildbio.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":[],"targets":["b7h3"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07680439"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"An autologous CAR T-cell therapy targeting B7-H3, an antigen described as universally expressed in diffuse intrinsic pontine glioma (DIPG), manufactured from the patient's own leukapheresed immune cells after standard radiation therapy.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bcd-100","kind":"drug","name":"BCD-100","aka":["prolgolimab","Forteca"],"tldr":"BCD-100 is an experimental investigational agent whose form is not stated in the registry from Biocad in phase 3 trials for melanoma, with its target not yet stated publicly.","summary":"BCD-100 is an investigational agent whose form is not stated in the registry developed by Biocad. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Subject recieves prolgolimab 3 mg/kg as an intravenous infusion once every 3 weeks (Q3W) simultaneously with placebo, a total of 4 intravenous infusions. Beginning with the 5th infusion, subjects are switched to prolgolimab 1 mg/kg monother. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05732805 (A Clinical Study of BCD-217 (Nurulimab + Prolgolimab) Followed by Anti-PD-1 Compared to Anti-PD-1 Monotherapy as First-Line Treatment in Subjects With Unresectable/Metastatic Melanoma), in melanoma. The largest, NCT05732805, plans to enrol 270 participants with primary completion was scheduled for 2024-06-20 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BCD-100","url":"https://clinicaltrials.gov/search?intr=BCD-100"},{"label":"Sponsor page","url":"https://biocad.ru"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["biocad"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05732805"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody (prolgolimab; INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bcd-217","kind":"drug","name":"BCD-217","aka":["nurulimab+prolgolimab"],"tldr":"BCD-217 is an experimental investigational agent whose form is not stated in the registry from Biocad in phase 3 trials for melanoma, aimed at PD-1 and CTLA-4.","summary":"BCD-217 is an investigational agent whose form is not stated in the registry developed by Biocad. Its targets are PD-1 and CTLA-4. ClinicalTrials.gov describes the intervention as: Subject recieves BCD-217 0.2 mL/kg, which is equivalent to 1 mg/kg nurulimab + 3 mg/kg prolgolimab, as an intravenous infusion once every 3 weeks (Q3W) simultaneously with placebo, a total of 4 intravenous infusions. Beginning with the 5th. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05732805 (A Clinical Study of BCD-217 (Nurulimab + Prolgolimab) Followed by Anti-PD-1 Compared to Anti-PD-1 Monotherapy as First-Line Treatment in Subjects With Unresectable/Metastatic Melanoma) and NCT05751928 (A Study of Neoadjuvant Therapy With BCD-217 (Nurulimab + Prolgolimab) in Patients With Resectable Stage III Skin Melanoma), in melanoma. The largest, NCT05751928, plans to enrol 411 participants (actual) with primary completion expected 2027-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BCD-217","url":"https://clinicaltrials.gov/search?intr=BCD-217"},{"label":"Sponsor page","url":"https://biocad.ru/products"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":[],"targets":["pd1","ctla4"],"drugs":[],"companies":["biocad"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05732805","nct05751928"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody combination (nurulimab and prolgolimab; INN stem -mab; form not stated in the registry record)","mechanism":"Investigational agent whose form is not stated in the registry directed at PD-1 and CTLA-4, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bdc-4182","kind":"drug","name":"BDC-4182","aka":[],"tldr":"BDC-4182 is an experimental monoclonal antibody from Bolt Biotherapeutics in phase 2 trials for gastric & gastro-oesophageal junction cancer, aimed at Claudin 18.2.","summary":"BDC-4182 is a monoclonal antibody developed by Bolt Biotherapeutics. Its target is Claudin 18.2 (the sponsor names Claudin 18.2). The sponsor states: A 'Boltbody' immune-stimulating antibody conjugate (ISAC) combining an anti-Claudin 18.2 monoclonal antibody with a TLR7/8 dual agonist, working through immune activation (inducing immunological memory) rather than direct cytotoxicity. ClinicalTrials.gov describes the intervention as: Immune stimulating antibody conjugate (ISAC), consisting of an anti-claudin 18.2 monoclonal antibody conjugated to a TLR 7/8 dual agonist. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in gastric & gastro-oesophageal junction cancer. The largest, NCT06921837, plans to enrol 122 participants with primary completion expected 2027-05. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BDC-4182","url":"https://clinicaltrials.gov/search?intr=BDC-4182"},{"label":"Sponsor pipeline page","url":"https://boltbio.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric"],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":["bolt-biotherapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06921837"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"A 'Boltbody' immune-stimulating antibody conjugate (ISAC) combining an anti-Claudin 18.2 monoclonal antibody with a TLR7/8 dual agonist, working through immune activation (inducing immunological memory) rather than direct cytotoxicity.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bebt-209","kind":"drug","name":"BEBT-209","aka":[],"tldr":"BEBT-209 is an experimental small-molecule drug from BeBetter Med in phase 3 trials for triple-negative breast cancer and HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"BEBT-209 (KCBI-0191) is a small-molecule drug developed by BeBetter Med. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Dosage: 150 mg orally per dose. Schedule: Administered on Day 1 (D1; before dinner), Day 2 (D2; before breakfast), Day 8 (D8; before dinner), and Day 9 (D9; before breakfast) of each 21-day cycle. Timing: On chemotherapy days (D2 and D9), B. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07544056 (A Study of BEBT-209 Plus Chemotherapy in Patients With Locally Advanced or Metastatic Triple-Negative Breast Cancer) and NCT06998108 (Study of BEBT-209 in Combination With Fulvestrant Versus Placebo in Combination With Fulvestrant in Patients With HR+/HER2- Locally Advanced or Metastatic Breast Cancer Who Have Progressed After Prior Endocrine Therapy), in triple-negative breast cancer and HR-positive / HER2-negative breast cancer. The largest, NCT07544056, plans to enrol 446 participants with primary completion expected 2030-06. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BEBT-209","url":"https://clinicaltrials.gov/search?intr=KCBI-0191"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["tnbc","breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["bebetter-med"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07544056","nct06998108"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"KCBI-0191","modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"befotertinib","kind":"drug","name":"Befotertinib","aka":[],"tldr":"Befotertinib is an oral kinase inhibitor from Betta Pharmaceuticals Co., Ltd., in registered phase 2 trials for non-small-cell lung cancer.","summary":"Befotertinib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Betta Pharmaceuticals Co., Ltd., in non-small-cell lung cancer. A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Befotertinib","url":"https://clinicaltrials.gov/search?intr=Befotertinib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["betta"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07181499"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral kinase inhibitor","mechanism":"A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"belantamab-mafodotin","kind":"drug","name":"Belantamab mafodotin","aka":[],"tldr":"A myeloma ADC that was withdrawn in 2022 then came back in 2025 after strong trials in earlier lines.","summary":"Belantamab mafodotin is an afucosylated anti-BCMA IgG1 antibody linked through a non-cleavable linker to MMAF, a non-permeable tubulin inhibitor; it kills through the payload and through enhanced antibody-dependent cellular cytotoxicity. It received accelerated approval in 2020 for myeloma after four or more lines (DREAMM-2), was withdrawn in 2022 when DREAMM-3 failed to confirm benefit, and returned in 2025 in the US and EU for relapsed or refractory myeloma after at least one prior line, after DREAMM-7 (with bortezomib and dexamethasone) and DREAMM-8 (with pomalidomide and dexamethasone) showed progression-free and overall survival benefits. Dosing is 2.5 mg/kg every 3 weeks in DREAMM-7 or 2.5 then 1.9 mg/kg every 4 weeks in DREAMM-8. Keratopathy occurs in most patients and carries a boxed warning, so an ophthalmology-based REMS is required.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Belantamab_mafodotin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Belantamab%20mafodotin"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-relapsed-refractory"],"sections":[],"technologies":["adc"],"targets":["bcma"],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":["mc-non-cleavable"],"trials":["nct06868654","nct06956170","nct04162210","nct06679101","nct06868667","nct07084896","nct07150091","nct07227311","nct07217184","nct05714839","nct07150104"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Blenrep","modality":"ADC","payload":"MMAF (tubulin inhibitor, non-permeable)","linker":"mc, non-cleavable","mechanism":"Afucosylated anti-BCMA IgG1 with MMAF; ADCC plus payload.","approvals":[{"region":"US","year":2020,"indication":"Relapsed myeloma ≥4 lines (accelerated; withdrawn 2022)"},{"region":"US","year":2025,"indication":"Relapsed/refractory myeloma after ≥1 prior line, in combination"},{"region":"EU","year":2025,"indication":"R/R myeloma with BorDex or PomDex; 23 Jul 2025"}],"mechanismSteps":["Antibody binds BCMA on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","MMAF (non-permeable) is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis"],"dosing":{"route":"IV infusion","schedule":"2.5 mg/kg every 3 weeks with bortezomib/dexamethasone (DREAMM-7) or 2.5 mg/kg then 1.9 mg/kg every 4 weeks with pomalidomide/dexamethasone (DREAMM-8)","modifications":"Hold and reduce for keratopathy or visual acuity decline per REMS","monitoring":"Ophthalmic exam before each dose; preservative-free lubricants","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Keratopathy","note":"Ocular events in most patients; boxed warning"},{"event":"Thrombocytopenia"},{"event":"Blurred vision"},{"event":"Dry eye"},{"event":"Infections"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended in combination regimens for relapsed myeloma (2025)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2020-08-05","type":"accelerated-approval","region":"US","note":"Accelerated approval, relapsed/refractory myeloma after ≥4 lines (DREAMM-2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adults patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior therapies including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent"},{"date":"2022-11-22","type":"withdrawal","region":"US","note":"Withdrawn after DREAMM-3 failed to confirm benefit","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-02-06","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 2.5 years after its accelerated approval.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-granted-accelerated-approval-belantamab-mafodotin-blmf-multiple-myeloma","indication":"Adults patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior therapies including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an immunomodulatory agent"},{"date":"2025-07-23","type":"crl","region":"US","note":"Complete response letter on combination filing; resubmitted","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-10","type":"approval","region":"US","note":"Approval in combination regimens for relapsed/refractory myeloma after ≥1 line (DREAMM-7/8)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"belinostat","kind":"drug","name":"Belinostat","aka":[],"tldr":"Belinostat is an HDAC inhibitor for relapsed peripheral T-cell lymphoma; about a quarter of patients respond, and it can be used in patients with low platelets.","summary":"Belinostat is a hydroxamate pan-HDAC inhibitor that blocks class I, II and IV histone deacetylases, increasing acetylation of histones and non-histone proteins with antiproliferative effects in T-cell lymphoma. It is used intravenously in relapsed or refractory peripheral T-cell lymphoma and can be given to patients with low platelet counts, which sets it apart from some alternatives. The BELIEF study (2014) reported a 26% response rate with a median response duration of 10.8 months, supporting accelerated US approval in 2014. The dose is reduced in patients homozygous for UGT1A1*28 because they clear the drug more slowly, and combination with CHOP (Bel-CHOP) has been studied in the front line. Randomised survival evidence is lacking, as for other single-agent PTCL approvals. In short, belinostat helps about a quarter of relapsed PTCL patients and is tolerable when blood counts are poor.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Belinostat","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=belinostat"}],"tags":["gap-fill"],"related":[],"cancers":["peripheral-t-cell-lymphoma"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":[],"companies":["acrotech-biopharma","spectrum-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06072131"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Beleodaq","modality":"Pan-HDAC inhibitor (hydroxamate)","mechanism":"Inhibits class I, II and IV histone deacetylases, increasing acetylation of histone and non-histone proteins with antiproliferative effects in T-cell lymphoma.","approvals":[{"region":"US","year":2014,"indication":"Relapsed/refractory peripheral T-cell lymphoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2014-07-03","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 12.2 years later, when the FDA's table was read.","indication":"Treatment of relapsed or refractory peripheral T-Cell Lymphoma (PTCL)"}]},{"id":"belumosudil","kind":"drug","name":"Belumosudil","aka":[],"tldr":"Belumosudil is a pill for chronic graft-versus-host disease, the long-term immune complication of donor stem-cell transplants used to cure blood cancers.","summary":"Belumosudil is an oral selective inhibitor of Rho-associated coiled-coil kinase 2 (ROCK2). By rebalancing Th17 and regulatory T-cell responses and reducing fibrotic signalling, it addresses both the inflammatory and the sclerotic components of chronic graft-versus-host disease, the long-term immune complication of donor stem-cell transplants used to cure leukaemias and MDS. In ROCKstar (2021) the overall response rate was 74% in patients with cGVHD after at least two prior lines, including responses in fibrotic organ involvement. It was approved in the US in 2021 for patients aged 12 and over, alongside ruxolitinib (REACH3) and ibrutinib, and axatilimab (anti-CSF1R) followed in 2024. Sequencing among them is unsettled. Belumosudil is part of the transplant survivorship toolkit: a pill that helps donor immune attack on the patient's own tissues without broad immunosuppression.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Belumosudil","links":[{"label":"ROCKstar (Blood 2021)","url":"https://doi.org/10.1182/blood.2021012021"},{"label":"FDA approval summary (Clinical Cancer Research 2022)","url":"https://doi.org/10.1158/1078-0432.CCR-21-4176"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=belumosudil"}],"tags":["gap-fill","supportive"],"related":[],"cancers":["aml","all-leukemia","mds"],"sections":["rejuvenation"],"technologies":["allogeneic-hsct","kinase-inhibitors","gvhd-belumosudil-axatilimab-ibrutinib"],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["rockstar"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Rezurock","modality":"Oral ROCK2 inhibitor","supportive":true,"mechanism":"Selective inhibition of Rho-associated coiled-coil kinase 2 rebalances Th17/Treg responses and reduces fibrosis in chronic graft-versus-host disease.","approvals":[{"region":"US","year":2021,"indication":"Chronic GVHD after failure of ≥2 lines of systemic therapy (age ≥12)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"belzutifan","kind":"drug","name":"Belzutifan","aka":[],"tldr":"Belzutifan is the first HIF-2α inhibitor, born from Nobel-winning biology, and is now approved after kidney cancer surgery with pembrolizumab.","summary":"Belzutifan is a small-molecule HIF-2-alpha inhibitor that blocks HIF-2-alpha from dimerising with ARNT, silencing the hypoxia programme that drives clear-cell kidney cancer when VHL is lost; it is taken as 120 mg once daily. It was approved in 2021 for VHL disease-associated renal cell carcinoma, CNS haemangioblastoma and pancreatic neuroendocrine tumours, in 2023 for advanced RCC after PD-1/PD-L1 and VEGF-TKI therapy (LITESPARK-005), in 2025 for pheochromocytoma and paraganglioma, and in Q2 2026 as adjuvant therapy for clear-cell RCC with pembrolizumab (LITESPARK-022). Merck markets it. Anaemia and hypoxia are on-mechanism effects because HIF-2-alpha regulates erythropoietin. Whether the adjuvant benefit translates into survival, and which patients respond, remain open. For a newcomer: the first drug from the Nobel-winning biology of how cells sense oxygen.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Belzutifan","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Belzutifan"}],"tags":[],"related":[],"cancers":["rcc","clear-cell-rcc","spinal-cord-tumours","pancreatic-net","hereditary-ppgl","metastatic-ppgl"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["hif2a"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07049926","nct03634540","nct04924075","nct05468697","nct04626479","nct04586231","nct05899049","nct04976634","nct04626518","nct06428396","nct07405164","nct04489771"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Welireg","modality":"Small-molecule HIF-2α inhibitor","mechanism":"Blocks HIF-2α/ARNT dimerisation.","approvals":[{"region":"US","year":2021,"indication":"VHL-associated RCC, CNS haemangioblastoma, pNET"},{"region":"US","year":2023,"indication":"Advanced RCC after PD-1/PD-L1 and VEGF-TKI"},{"region":"US","year":2026,"indication":"Adjuvant clear-cell RCC with pembrolizumab"},{"region":"EU","year":2025,"indication":"ccRCC after ≥2 lines; VHL-associated tumours; 12 Feb 2025 (conditional)","note":"Conditional marketing authorisation"}],"mechanismSteps":["Belzutifan binds the PAS-B pocket of HIF-2α","HIF-2α cannot dimerise with HIF-1β (ARNT)","Transcription of VEGF, GLUT1, cyclin D1 and other hypoxia genes stops","VHL-deficient tumour cells lose their growth programme; erythropoietin falls (anaemia)"],"dosing":{"route":"Oral","schedule":"120 mg once daily","modifications":"Hold for haemoglobin <8 g/dL or hypoxia; permanently discontinue for life-threatening hypoxia","monitoring":"Haemoglobin and oxygen saturation before and during treatment; embryo-fetal toxicity","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Anaemia","note":"LITESPARK-005: ~83% any grade, ~33% grade 3"},{"event":"Hypoxia"},{"event":"Fatigue"},{"event":"Nausea"},{"event":"Oedema"},{"event":"Dyspnoea"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.merckaccessprogram-welireg.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: appraisal for RCC after IO and VEGF-TKI (2025-26)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-08-13","type":"approval","region":"US","note":"VHL disease-associated RCC, CNS haemangioblastoma, pNET: first HIF-2α inhibitor","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-12-14","type":"approval","region":"US","note":"Advanced RCC after PD-1/PD-L1 and VEGF-TKI (LITESPARK-005)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-05","type":"approval","region":"US","note":"Pheochromocytoma/paraganglioma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-06-12","type":"approval","region":"US","note":"Adjuvant clear-cell RCC with pembrolizumab (LITESPARK-022)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belzutifan-pembrolizumab-adjuvant-treatment-renal-cell-carcinoma"},{"date":"2026-09-24","type":"approval","region":"US","note":"With lenvatinib, advanced renal cell carcinoma with a clear cell component","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belzutifan-combination-lenvatinib-advanced-renal-cell-carcinoma-clear-cell-component"}]},{"id":"bemarituzumab","kind":"drug","name":"Bemarituzumab","aka":[],"tldr":"Bemarituzumab is an antibody against FGFR2b, a growth receptor overproduced in about a third of stomach cancers. It improved survival early in its phase 3 trial, but the gain faded with longer follow-up.","summary":"Afucosylated IgG1 blocking FGFR2b ligand binding and enhancing ADCC. FORTITUDE-101: interim OS 17.9 vs 12.5 months (HR 0.61) in FGFR2b ≥10% 2+/3+ tumours, attenuated at updated analysis (ESMO 2025). Corneal adverse events are frequent. FORTITUDE-102 adds nivolumab. The regulatory path was still uncertain in September 2026.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"Amgen topline announcement","url":"https://www.amgen.com/newsroom/press-releases/2025/06/amgen-announces-positive-topline-phase-3-results-for-bemarituzumab-in-fibroblast-growth-factor-receptor-2b-fgfr2b-positive-firstline-gastric-cancer"}],"tags":[],"related":[],"cancers":["gastric"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["fgfr2"],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["fortitude-101"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"FPA144","modality":"Monoclonal antibody (anti-FGFR2b)","mechanism":"Blocks FGF ligand binding to FGFR2b and recruits NK-mediated ADCC.","approvals":[],"mechanismSteps":["Bemarituzumab binds the FGFR2b receptor on tumour cells","Growth-factor ligands can no longer activate it","Downstream RAS-MAPK signalling drops","The afucosylated antibody tail recruits NK cells to kill the coated cells"],"toxicity":[{"event":"Corneal adverse events (keratitis, dry eye)","note":"Frequent; dose-limiting"},{"event":"Stomatitis"}],"access":[],"regulatoryEvents":[]},{"id":"bempegaldesleukin","kind":"drug","name":"Bempegaldesleukin","aka":[],"tldr":"Bempegaldesleukin was a re-engineered interleukin-2 meant to be a safer version of a famous old immunotherapy. It added nothing to nivolumab in three phase 3 trials.","summary":"Bempegaldesleukin is a PEGylated IL-2 prodrug biased toward the CD122 receptor to expand CD8 T cells over regulatory T cells. Phase 1/2 PIVOT-02 with nivolumab reported high response rates in melanoma. In 2022, PIVOT IO-001 (melanoma), PIVOT-09 (RCC), and PIVOT-10 (urothelial) all failed to improve response, PFS, or OS over nivolumab alone; BMS and Nektar ended the collaboration (BMS had paid $1.85B upfront in 2018).\n\nLesson: single-arm combination response rates in melanoma are unreliable because nivolumab alone already produces them; biological rationale (Treg sparing) did not translate.","status":"negative","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Bempegaldesleukin","links":[{"label":"PIVOT IO-001 (J Clin Oncol 2023)","url":"https://ascopubs.org/doi/10.1200/JCO.23.00172"}],"tags":["failure","lesson:phase-2-mirage"],"related":[],"cancers":["melanoma","rcc","urothelial"],"sections":[],"technologies":["cytokine-therapy"],"targets":[],"drugs":["nivolumab"],"companies":["bms","nektar-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03635983","nct04410445","nct03729245"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"NKTR-214, bempeg","modality":"Engineered cytokine (PEGylated IL-2)","mechanism":"IL-2 with releasable PEG chains; preferential CD122 (IL-2Rβγ) engagement.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bendamustine","kind":"drug","name":"Bendamustine","aka":[],"tldr":"An East German chemotherapy rediscovered in the 2000s that became the backbone partner for rituximab in follicular, mantle cell and Waldenström lymphomas.","summary":"Approved 2008 for CLL and rituximab-refractory indolent NHL. StiL and BRIGHT trials showed bendamustine-rituximab at least as effective as R-CHOP with less alopecia and neuropathy, making BR the most common first-line indolent lymphoma regimen. Prolonged lymphopenia and infections (PJP, CMV) are the main concerns; combined with obinutuzumab (GALLIUM) or acalabrutinib (ECHO).","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Bendamustine","links":[{"label":"StiL NHL1 (Lancet 2013)","url":"https://doi.org/10.1016/S0140-6736(12)61763-2"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=bendamustine"}],"tags":["gap-fill","generic"],"related":[],"cancers":["follicular-lymphoma","mantle-cell-lymphoma","waldenstrom","cll"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["symbio-pharmaceuticals","mundipharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04002297","nct06363994","nct04792489","nct07189065","nct04844866","nct04268706","nct05624554","nct06084936","nct06846671","nct05023980","nct02970318","nct06522737","nct06970743","nct02972840"],"people":[],"bottlenecks":[],"keyPapers":["paper-rummel-lancet"],"journals":[],"dependsOn":[],"notes":[],"brand":"Treanda / Bendeka / Belrapzo","modality":"Alkylating agent (nitrogen mustard-purine hybrid)","mechanism":"Bifunctional alkylator causing DNA cross-links with a benzimidazole ring conferring purine-analogue-like properties; less cross-resistance with other alkylators.","approvals":[{"region":"US","year":2008,"indication":"CLL; indolent B-cell NHL progressed within 6 months of rituximab"},{"region":"EU","year":2010,"indication":"CLL, indolent NHL, multiple myeloma (national approvals)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"benmelstobart","kind":"drug","name":"Benmelstobart","aka":[],"tldr":"Benmelstobart is Chia Tai Tianqing's PD-L1 antibody, approved in China in 2024 for extensive-stage small-cell lung cancer in combination with anlotinib and chemotherapy.","summary":"Benmelstobart (TQB2450) is a humanised anti-PD-L1 antibody developed by Chia Tai Tianqing, part of Sino Biopharmaceutical. The NMPA approved it in 2024 for first-line treatment of extensive-stage small-cell lung cancer together with the multi-kinase inhibitor anlotinib and platinum-etoposide chemotherapy, on the ETER701 phase 3 trial, which reported longer overall survival than chemotherapy alone. It is the first checkpoint inhibitor approved in a four-drug first-line regimen for this cancer.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of benmelstobart","url":"https://clinicaltrials.gov/search?intr=TQB2450"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["sclc","extensive-stage-sclc"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pdl1"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"TQB2450","modality":"Anti-PD-L1 monoclonal antibody","mechanism":"Blocks PD-L1 on tumour and immune cells so that T cells are no longer switched off at the tumour.","approvals":[{"region":"CN","year":2024,"indication":"Extensive-stage small-cell lung cancer, first line, with anlotinib and platinum-etoposide chemotherapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bevacizumab","kind":"drug","name":"Bevacizumab","aka":[],"tldr":"Bevacizumab is a humanised antibody that soaks up VEGF-A, the signal tumours use to grow new blood vessels. Approved in 2004, it partners chemotherapy in colorectal, ovarian, cervical, lung, kidney and liver cancer and glioblastoma, and adds to trifluridine/tipiracil in late-line bowel cancer; hypertension, protein in the urine and bleeding are its characteristic side effects.","summary":"Humanised anti-VEGF-A IgG1. Standard with first- and second-line chemotherapy in mCRC (all RAS statuses, right-sided tumours), with trifluridine/tipiracil in refractory disease (SUNLIGHT), and in ovarian, cervical, NSCLC, RCC, HCC (with atezolizumab), and glioblastoma. Biosimilars since 2017 have cut its cost sharply. Hypertension, proteinuria, bleeding, wound-healing delay, and rare GI perforation.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Bevacizumab","links":[{"label":"FDA label","url":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/125085s340lbl.pdf"},{"label":"NICE TA1136: bevacizumab (originator and biosimilars) with fluoropyrimidine-based chemotherapy for metastatic colorectal cancer","url":"https://www.nice.org.uk/guidance/ta1136"},{"label":"NICE TA212 (2010, not recommended)","url":"https://www.nice.org.uk/guidance/ta212"},{"label":"EMA Avastin","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/avastin"}],"tags":[],"related":["bevacizumab-glioma"],"cancers":["colorectal","ovarian","nsclc","rcc","hcc","cervical","glioblastoma"],"sections":[],"technologies":["monoclonal-antibody","antiangiogenic","angiogenesis-models"],"targets":["vegf"],"drugs":[],"companies":["roche-genentech","hetero"],"institutions":[],"pathways":["vegf-angiogenesis"],"terms":[],"trials":["sunlight","paradigm","nct04854668","nct05445778","nct07221357","nct07775287","nct07216703","nct07318558","nct06824467","nct03390686","nct05654454","nct06921785","nct04547166","nct06819007","nct03847428","nct06123884","nct07722494","nct06497985","nct05904886","nct07225270","nct07564141","nct04712643","nct07676162","nct07527858","nct06445062","nct05775159","nct06512051","nct07024784","nct07446322","nct06107413","nct07054567","nct06792695","nct07805551","nct07479485","nct07079631","nct06820463","nct06948448","nct06679985","nct06873763","nct07071714","nct04068610","nct05739981","nct05919264","nct05797168","nct07714668","nct05440708","nct07042802","nct07244705","nct04938583","nct06427941","nct06096779","nct07474727","nct06607458","nct06890338","nct06840002","nct06859775","nct05733598","nct06843447","nct06040099","nct06493760","nct05824975","nct06558227","nct05489211","nct04931342","nct02519348","nct04524871","nct07059845","nct06496971","nct06465563","nct03899155","nct06276283","nct06061809","nct05405595","nct06783647","nct06951503","nct06771622","nct04262466","nct06895928","nct06730750","nct03148418","nct05867420","nct04121455","nct07297212","nct02671435","nct05584670","nct05112965","nct02734004","nct07448922","nct05101070","nct06750094","nct06906341","nct07111546","nct06271837","nct06016062","mountaineer","nct06697197","nct07283367","nct04626635","nct05239741","nct06703177","nct07286266","nct06796907","gog-0218-icon7","paola-1","keynote-b96","avf2107g","cairo3","cairo5","tribe","tribe2"],"people":["judah-folkman"],"bottlenecks":[],"keyPapers":["paper-hurwitz-bevacizumab-crc-nejm-2004"],"journals":[],"dependsOn":[],"notes":["In gynaecological oncology bevacizumab is used in first-line and recurrent ovarian cancer (GOG-0218, ICON7, OCEANS, AURELIA), as the partner for olaparib maintenance in HRD-positive disease (PAOLA-1), and in first-line cervical cancer regimens with pembrolizumab (KEYNOTE-826) or atezolizumab (BEATcc); it is given at 15 mg/kg every 3 weeks with chemotherapy and continued as maintenance. In the GOG-0218 concurrent and maintenance arm hypertension affected 42 percent (17 percent grade 3 or higher) and bowel perforation 2.6 percent."],"brand":"Avastin (and biosimilars)","modality":"Monoclonal antibody (anti-VEGF)","mechanism":"Neutralises circulating VEGF-A, reducing angiogenesis and normalising tumour vasculature.","approvals":[{"region":"US","year":2004,"indication":"First-line metastatic colorectal cancer with 5-FU chemotherapy"},{"region":"US","year":2006,"indication":"NSCLC; later ovarian, cervical, RCC, glioblastoma, HCC"},{"region":"US","year":2023,"indication":"Refractory mCRC with trifluridine/tipiracil (SUNLIGHT)"},{"region":"US","year":2014,"indication":"Platinum-resistant ovarian cancer with chemotherapy; recurrent/metastatic cervical cancer with chemotherapy"},{"region":"US","year":2018,"indication":"First-line stage III-IV ovarian cancer after surgery"},{"region":"US","year":2020,"indication":"First-line maintenance with olaparib in HRD-positive ovarian cancer"},{"region":"EU","year":2005,"indication":"Metastatic colorectal cancer with fluoropyrimidine-based chemotherapy","note":"Avastin marketing authorisation issued 12 January 2005."},{"region":"England (NICE)","year":2026,"indication":"First- and second-line metastatic colorectal cancer with fluoropyrimidine-based chemotherapy, only when targeted treatment or immunotherapy is unsuitable","note":"TA1136, published 25 February 2026, covers the originator and biosimilars and requires an agreed price within the Medicines Procurement and Supply Chain. The earlier first-line appraisals TA212 (2010) and TA118 (2007) did not recommend it."}],"mechanismSteps":["Bevacizumab binds VEGF-A in the blood and tumour","VEGF-A can no longer reach VEGFR2 on blood-vessel cells","New vessel growth slows and existing tumour vessels become less leaky","Chemotherapy penetrates better and the tumour is starved"],"dosing":{"route":"Intravenous","schedule":"5-10 mg/kg every 2 weeks (mCRC); 15 mg/kg every 3 weeks in other tumours","modifications":"Hold 28 days around surgery; stop for GI perforation or severe haemorrhage","monitoring":"Blood pressure, urine protein","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/125085s340lbl.pdf"},"toxicity":[{"event":"Hypertension","note":"Common; manageable"},{"event":"Proteinuria"},{"event":"GI perforation","note":"Rare, boxed warning"},{"event":"Bleeding","note":"Epistaxis common; serious haemorrhage rare"},{"event":"Impaired wound healing","note":"Boxed warning"}],"access":[],"regulatoryEvents":[{"date":"2004-02","type":"approval","region":"US","note":"First anti-angiogenic approval (AVF2107)"},{"date":"2008-02-22","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with paclitaxel for patients who have not received chemotherapy for metastatic HER2 negative breast cancer"},{"date":"2009-05-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Progressive glioblastoma following prior therapy"},{"date":"2011-11-18","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.7 years after its accelerated approval.","indication":"In combination with paclitaxel for patients who have not received chemotherapy for metastatic HER2 negative breast cancer"},{"date":"2014-08-14","type":"approval","region":"US","note":"Cervical cancer with chemotherapy (GOG-0240)"},{"date":"2017-09","type":"approval","region":"US","note":"First biosimilar (bevacizumab-awwb) approved"},{"date":"2017-12-05","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2009 converted to traditional approval 8.6 years after it was granted.","indication":"Progressive glioblastoma following prior therapy"},{"date":"2018-06-13","type":"approval","region":"US","note":"First-line ovarian cancer (GOG-0218)"},{"date":"2020-05-08","type":"approval","region":"US","note":"With olaparib as HRD-positive maintenance (PAOLA-1)"},{"date":"2023-08","type":"label-change","region":"US","note":"With trifluridine/tipiracil in refractory mCRC"}]},{"id":"bevacizumab-glioma","kind":"drug","name":"Bevacizumab (glioblastoma use)","aka":[],"tldr":"A blood-vessel-blocking antibody that shrinks glioblastoma on scans and reduces swelling, but has never been shown to help patients live longer.","summary":"Accelerated approval for recurrent glioblastoma (2009, full 2017) on radiographic response. AVAglio and RTOG 0825 (2014): PFS gain in newly diagnosed disease without OS benefit; EORTC 26101 at recurrence: PFS but no OS benefit with lomustine. Valuable for steroid-sparing control of oedema and radiation necrosis. A case study in pseudo-response and the limits of imaging endpoints in glioma.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Bevacizumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Bevacizumab"}],"tags":[],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":["antiangiogenic","monoclonal-antibody"],"targets":["vegf"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":["vegf-angiogenesis"],"terms":["recist"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Avastin","modality":"Monoclonal antibody (anti-VEGF)","mechanism":"Neutralises VEGF-A; normalises vasculature and reduces contrast enhancement and oedema.","approvals":[{"region":"US","year":2009,"indication":"Recurrent glioblastoma (accelerated; full approval 2017)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bexarotene","kind":"drug","name":"Bexarotene","aka":[],"tldr":"Bexarotene (Targretin) is a vitamin A-like capsule and gel for the skin disease of cutaneous T-cell lymphoma when other treatments have stopped working.","summary":"Bexarotene is a synthetic retinoid selective for the retinoid X receptor. The FDA approved the capsules in December 1999 and the 1% gel in 2000 for cutaneous manifestations of cutaneous T-cell lymphoma (mycosis fungoides and Sezary syndrome) refractory to at least one prior systemic therapy, on two open-label historically controlled trials in 152 patients. The EU authorised Targretin capsules in 2001 for the same population. Central hypothyroidism and marked hypertriglyceridaemia are expected effects of RXR activation and need thyroid replacement and lipid-lowering therapy alongside treatment; it is teratogenic.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Bexarotene","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=bexarotene"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/bexarotene"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/targretin"}],"tags":["nci-list"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["rxr"],"drugs":[],"companies":["bausch-health"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Cutaneous T-cell lymphoma has no cancer record yet; link when one exists."],"brand":"Targretin","modality":"Small-molecule retinoid X receptor agonist","mechanism":"Selectively binds and activates RXR alpha, beta and gamma; RXR heterodimers regulate transcription of genes controlling differentiation and apoptosis in malignant T cells.","approvals":[{"region":"US","year":1999,"indication":"Cutaneous manifestations of cutaneous T-cell lymphoma refractory to at least one systemic therapy"},{"region":"EU","year":2001,"indication":"Skin manifestations of advanced-stage CTCL refractory to at least one systemic treatment"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bexmarilimab","kind":"drug","name":"Bexmarilimab","aka":["FP-1305"],"tldr":"Bexmarilimab is a monoclonal antibody from Faron Pharmaceuticals Ltd, in registered phase 2 trials for acute myeloid leukaemia, chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms, myelodysplastic syndromes / neoplasms.","summary":"Bexmarilimab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Faron Pharmaceuticals Ltd, in acute myeloid leukaemia, chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms, myelodysplastic syndromes / neoplasms. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Bexmarilimab","url":"https://clinicaltrials.gov/search?intr=Bexmarilimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["aml","cmml","mds"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["faron-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05428969"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bexobrutideg","kind":"drug","name":"Bexobrutideg","aka":[],"tldr":"Bexobrutideg is an experimental protein degrader from Nurix Therapeutics in phase 3 trials for chronic lymphocytic leukaemia, aimed at BTK (Bruton tyrosine kinase).","summary":"Bexobrutideg (NX-5948) is a protein degrader developed by Nurix Therapeutics. Its target is BTK (Bruton tyrosine kinase) (the sponsor names BTK (Bruton's tyrosine kinase)). The sponsor states: NX-5948 is an oral, CNS-penetrant BTK degrader that removes the BTK protein rather than only inhibiting its activity, which the sponsor states may help overcome kinase-domain and scaffolding-mediated resistance mutations. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07516093 (Study of NX-5948 Versus Pirtobrutinib in R/R CLL/SLL), in chronic lymphocytic leukaemia. The largest, NCT07516093, plans to enrol 620 participants with primary completion expected 2029-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Bexobrutideg","url":"https://clinicaltrials.gov/search?intr=Bexobrutideg"},{"label":"Sponsor pipeline page","url":"https://www.nurixtx.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cll"],"sections":[],"technologies":[],"targets":["btk"],"drugs":[],"companies":["nurix"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07516093","nct07520006","nct07221500"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"NX-5948","modality":"protein degrader","mechanism":"NX-5948 is an oral, CNS-penetrant BTK degrader that removes the BTK protein rather than only inhibiting its activity, which the sponsor states may help overcome kinase-domain and scaffolding-mediated resistance mutations.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bezuclastinib","kind":"drug","name":"Bezuclastinib","aka":[],"tldr":"Bezuclastinib is an experimental small-molecule drug from Cogent Biosciences in phase 3 trials for gastrointestinal stromal tumour, aimed at KIT and MET.","summary":"Bezuclastinib (CGT9486) is a small-molecule drug developed by Cogent Biosciences. Its targets are KIT and MET (the sponsor names KIT (including KIT D816V)). The sponsor states: Bezuclastinib (CGT9486/PLX9486) is a selective tyrosine kinase inhibitor designed to potently inhibit KIT driver mutations, including the KIT D816V mutation that drives systemic mastocytosis, and is combined with sunitinib versus sunitinib alone in gastrointestinal stromal tumours. ClinicalTrials.gov describes the intervention as: Participants will receive CGT9486 orally until study stopping rules are met. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05208047 ((Peak) A Phase 3 Randomised Trial of CGT9486+Sunitinib vs. Sunitinib in Subjects With Gastrointestinal Stromal Tumours), in gastrointestinal stromal tumour. The largest, NCT05208047, plans to enrol 482 participants with primary completion was scheduled for 2025-09-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Bezuclastinib","url":"https://clinicaltrials.gov/search?intr=Bezuclastinib"},{"label":"Sponsor pipeline page","url":"https://www.cogentbio.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gist","indolent-systemic-mastocytosis","advanced-systemic-mastocytosis"],"sections":[],"technologies":[],"targets":["kit","met"],"drugs":[],"companies":["cogent-biosciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05208047","nct05186753","nct04996875"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CGT9486","modality":"small molecule","mechanism":"Bezuclastinib (CGT9486/PLX9486) is a selective tyrosine kinase inhibitor designed to potently inhibit KIT driver mutations, including the KIT D816V mutation that drives systemic mastocytosis, and is combined with sunitinib versus sunitinib alone in gastrointestinal stromal tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bgb-16673","kind":"drug","name":"BGB-16673","aka":[],"tldr":"Instead of blocking BTK, this pill destroys it, so it works even when the enzyme has mutated to escape every inhibitor.","summary":"Chimeric degradation-activating compound recruiting an E3 ligase to BTK. CaDAnCe-101 (phase 1, relapsed CLL/SLL after covalent BTKi, many with BTK mutations): ORR 86% in 66 patients with a manageable profile (fatigue, bruising, diarrhoea, neutropenia), updated at EHA 2026. Phase 3 CaDAnCe-304 (~500 patients) compares BGB-16673 with pirtobrutinib after covalent BTKi. The first BTK degrader in phase 3.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"CaDAnCe-304 design (Blood 2025)","url":"https://ashpublications.org/blood/article/146/Supplement%201/5691/550156/CaDAnCe-304-a-phase-3-open-label-randomized-study"}],"tags":[],"related":[],"cancers":["cll","dlbcl"],"sections":[],"technologies":["protac-degrader"],"targets":["btk"],"drugs":[],"companies":["beone"],"institutions":[],"pathways":[],"terms":[],"trials":["cadance-304","nct05006716","nct05294731","nct06970743"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Small-molecule BTK degrader (CDAC)","mechanism":"Heterobifunctional degrader: one end binds BTK (wild-type or C481/T474/L528 mutant), the other recruits an E3 ligase, tagging BTK for proteasomal destruction.","approvals":[],"mechanismSteps":["BGB-16673 bridges BTK and an E3 ubiquitin ligase","BTK is ubiquitinated and destroyed by the proteasome","Both kinase and scaffolding functions of BTK are removed","Resistance mutations that block inhibitors do not prevent degradation"],"dosing":{"route":"Oral","schedule":"200 mg once daily (recommended phase 2 dose), continuous","monitoring":"Neutropenia, infections, bleeding"},"toxicity":[{"event":"Fatigue","anyGradePct":25,"source":"https://cllsociety.org/2026/03/btk-degrader-shows-promise-for-relapsed-cll/","note":"≥25% in CaDAnCe-101"},{"event":"Contusion/bruising","anyGradePct":25},{"event":"Neutropenia","anyGradePct":25}],"access":[],"regulatoryEvents":[]},{"id":"bgb-43395","kind":"drug","name":"BGB-43395","aka":[],"tldr":"BGB-43395 is an experimental small-molecule drug from BeOne Medicines in phase 3 trials for HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"BGB-43395 is a small-molecule drug developed by BeOne Medicines. The sponsor describes its target as CDK4, which OnCo does not yet have a target page for. The sponsor states: A CDK4-selective inhibitor designed to spare CDK6, intended to drive down cancer cell growth signalling while reducing the blood-cell toxicities associated with CDK6 inhibition. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07492641 (BGB-43395 Plus Letrozole Versus CDK4/6i Plus Letrozole for Patients With Advanced or Metastatic HR+/HER2- Breast Cancer Who Have Not Received Prior Treatment for Advanced or Metastatic Disease), in HR-positive / HER2-negative breast cancer. The largest, NCT07492641, plans to enrol 1056 participants with primary completion expected 2029-04. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BGB-43395","url":"https://clinicaltrials.gov/search?intr=BGB-43395"},{"label":"Sponsor pipeline page","url":"https://www.beonemedicines.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["beone"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07492641"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"A CDK4-selective inhibitor designed to spare CDK6, intended to drive down cancer cell growth signalling while reducing the blood-cell toxicities associated with CDK6 inhibition.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bicalutamide","kind":"drug","name":"Bicalutamide","aka":[],"tldr":"The old antiandrogen pill used to block the testosterone flare from GnRH agonists and in combined androgen blockade; superseded by enzalutamide-class drugs.","summary":"Bicalutamide is an oral first-generation non-steroidal antiandrogen that competes with testosterone and dihydrotestosterone for the androgen receptor. It has partial agonist activity in tumours with AR amplification or the W741L mutation, which explains the antiandrogen withdrawal response seen when it is stopped. It was approved in the US and EU in 1995 for metastatic prostate cancer in combination with an LHRH agonist, and in the EU a 150 mg monotherapy dose is licensed for locally advanced disease. It is still used to block the testosterone flare at the start of agonist therapy and remains a mainstay in low-resource settings because it is cheap and well tolerated. Enzalutamide-class drugs, which bind the receptor without agonism, have superseded it in most guidelines. Bicalutamide is the old antiandrogen pill whose limits taught the field how to build better ones.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Bicalutamide","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=bicalutamide"}],"tags":["gap-fill","generic"],"related":[],"cancers":["prostate","salivary-duct-carcinoma","tnbc"],"sections":[],"technologies":["androgen-deprivation"],"targets":["androgen-receptor"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":["ar-signaling"],"terms":[],"trials":["rtog-9601","nct02531516"],"people":[],"bottlenecks":[],"keyPapers":["paper-gucalp-bicalutamide-ar-positive-tbcrc011-ccr-2013"],"journals":[],"dependsOn":[],"notes":[],"brand":"Casodex","modality":"Oral first-generation non-steroidal antiandrogen","mechanism":"Competitive AR antagonist with partial agonist activity in AR-amplified or mutated (W741L) disease; combined with GnRH agonists.","approvals":[{"region":"US","year":1995,"indication":"Metastatic prostate cancer with LHRH analogue"},{"region":"EU","year":1995,"indication":"Advanced prostate cancer; 150 mg for locally advanced"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1995-10-04","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with an LHRH analogue for advanced prostate cancer"},{"date":"1997-12-12","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1995 converted to traditional approval 2.2 years after it was granted.","indication":"In combination with an LHRH analogue for advanced prostate cancer"}]},{"id":"binimetinib","kind":"drug","name":"Binimetinib","aka":[],"tldr":"Binimetinib is the MEK inhibitor partnered with encorafenib; blocking the next step in the same relay stops the tumour rerouting around the BRAF block.","summary":"Binimetinib is an allosteric, ATP-non-competitive inhibitor of MEK1 and MEK2, the kinases immediately downstream of BRAF; blocking the next step in the relay stops the tumour rerouting around a BRAF inhibitor. It is given at 45 mg twice daily with encorafenib 450 mg once daily for BRAF V600E/K melanoma (approved June 2018 on COLUMBUS) and BRAF V600E metastatic NSCLC (2023). COLUMBUS showed median PFS of 14.9 versus 7.3 months for vemurafenib (HR 0.54) and median OS of 33.6 months, with less pyrexia than dabrafenib-trametinib. Class effects are retinopathy (serous retinal detachment), CK elevation, left-ventricular dysfunction, rash and diarrhoea, so eye examinations, echocardiograms and CK monitoring are recommended. Whether BRAF/MEK doublets or immunotherapy should come first in BRAF-mutant melanoma remains debated. For a newcomer, binimetinib is the MEK half of the encorafenib pair.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Binimetinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Binimetinib"}],"tags":[],"related":[],"cancers":["melanoma","nsclc","braf-v600e-nsclc","braf-v600-melanoma","advanced-melanoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf"],"drugs":["encorafenib"],"companies":["pfizer"],"institutions":[],"pathways":["ras-mapk"],"terms":["ocular-toxicity"],"trials":["columbus","nct05270044","nct04657991","nct03947385","nct05195632"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mektovi","modality":"Small-molecule kinase inhibitor (MEK1/2)","mechanism":"Allosteric, ATP-non-competitive MEK1/2 inhibitor.","approvals":[{"region":"US","year":2018,"indication":"BRAF V600E/K melanoma with encorafenib"},{"region":"US","year":2023,"indication":"BRAF V600E metastatic NSCLC with encorafenib"}],"mechanismSteps":["Binds MEK1/2 outside the ATP site and locks them in an inactive conformation","ERK phosphorylation falls even if RAF activity rebounds","Combined with a BRAF inhibitor, blocks the paradoxical activation that causes squamous skin lesions and delays resistance"],"dosing":{"route":"Oral","schedule":"45 mg twice daily with encorafenib 450 mg once daily","modifications":"Hold for retinopathy, LVEF drop, CK rise","monitoring":"Ophthalmology at baseline and for symptoms; echocardiogram; CK","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/210498s009lbl.pdf"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2018-06-27","type":"approval","region":"US","note":"Encorafenib + binimetinib approved for BRAF V600 melanoma"}]},{"id":"bio-106","kind":"drug","name":"BIO-106","aka":[],"tldr":"BIO-106 is an experimental antibody-drug conjugate from BiOneCure Therapeutics in phase 2 trials, aimed at TROP2.","summary":"BIO-106 is an antibody-drug conjugate developed by BiOneCure Therapeutics. Its target is TROP2 (the sponsor names Trop-2). The sponsor states: BIO-106 is a novel antibody-drug conjugate targeting the human trophoblast cell surface antigen 2 (Trop-2). ClinicalTrials.gov describes the intervention as: BIO-106 is a novel antibody-drug conjugate (ADC) that targets the human trophoblast cell surface antigen 2 (Trop-2). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT05320588, plans to enrol 332 participants with primary completion was scheduled for 2024-04 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BIO-106","url":"https://clinicaltrials.gov/search?intr=BIO-106"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":["adc"],"targets":["trop2"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct05320588"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"BIO-106 is a novel antibody-drug conjugate targeting the human trophoblast cell surface antigen 2 (Trop-2).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bleomycin","kind":"drug","name":"Bleomycin","aka":[],"tldr":"Bleomycin is the 'B' in BEP for testicular cancer and ABVD for Hodgkin lymphoma; its unique danger is scarring of the lungs, so lung function is monitored and it is often dropped when safe.","summary":"Approved 1973. BEP (testicular; omitted in EP for good-risk patients with lung risk), ABVD (Hodgkin; omitted after negative interim PET in RATHL), Kaposi sarcoma (bleomycin-vincristine in Africa), sclerotherapy of lymphatic malformations and pleurodesis, electrochemotherapy for cutaneous tumours. Cumulative dose limit ~400 units; pulmonary toxicity 10%, fatal in 1-2%.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Bleomycin","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=bleomycin"}],"tags":["gap-fill","generic","who-essential"],"related":[],"cancers":["testicular","hodgkin-lymphoma","kaposi-sarcoma","seminoma","non-seminoma"],"sections":[],"technologies":["cytotoxic-chemotherapy","electrochemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["ild"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Blenoxane","modality":"Glycopeptide antibiotic (DNA-cleaving)","mechanism":"Chelates iron and generates free radicals that cause single- and double-strand DNA breaks; inactivated by bleomycin hydrolase, which is scarce in lung and skin (fibrosis, hyperpigmentation).","approvals":[{"region":"US","year":1973,"indication":"Squamous cell carcinomas, Hodgkin and non-Hodgkin lymphoma, testicular carcinoma, malignant pleural effusion"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bleximenib","kind":"drug","name":"Bleximenib","aka":[],"tldr":"Bleximenib is an experimental small-molecule drug from Janssen Research & Development in phase 3 trials for acute myeloid leukaemia, with its target not yet stated publicly.","summary":"Bleximenib (JNJ-75276617) is a small-molecule drug developed by Janssen Research & Development. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06852222 (A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukaemia (AML)), in acute myeloid leukaemia. The largest, NCT06852222, plans to enrol 600 participants with primary completion expected 2029-06-22. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Bleximenib","url":"https://clinicaltrials.gov/search?intr=JNJ-75276617"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["aml","aml-npm1-kmt2a"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06852222"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"JNJ-75276617","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"blinatumomab","kind":"drug","name":"Blinatumomab","aka":[],"tldr":"Blinatumomab was the first T-cell engager (2014), and is now given to children and adults with leukaemia even when in remission, because it improves survival.","summary":"Blinatumomab is a bispecific T-cell engager (BiTE) built from two tandem single-chain antibody fragments, one binding CD19 on B-lineage leukaemia cells and the other binding CD3 on T cells, so any T cell can be redirected to kill the leukaemia cell without MHC presentation. Its half-life is about 2 hours, so it is given by continuous intravenous infusion in 42-day cycles; a subcutaneous formulation is in development. Approved in 2014 as the first BiTE for relapsed or refractory B-ALL, it became the first drug approved on a minimal residual disease endpoint in 2018, and in 2024 was approved as consolidation for CD19-positive B-ALL regardless of MRD after E1910 showed an overall survival benefit in adults and AALL1731 in children. Neurological toxicity occurred in 65 percent of relapsed adults and cytokine release syndrome in 14 percent, so early cycle days are spent in hospital.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Blinatumomab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Blinatumomab"}],"tags":[],"related":["cd19-expression"],"cancers":["all-leukemia","all-paediatric-standard-risk","all-paediatric-relapsed","all-infant"],"sections":[],"technologies":["t-cell-engager"],"targets":["cd19","cd3"],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04521231","nct07134088","nct07387926"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Blincyto","modality":"Bispecific T-cell engager (CD19×CD3)","mechanism":"Tandem scFv BiTE.","approvals":[{"region":"US","year":2014,"indication":"Relapsed/refractory B-ALL"},{"region":"US","year":2024,"indication":"Consolidation in CD19+ B-ALL regardless of MRD"}],"mechanismSteps":["One arm binds CD19 on the tumour cell","The other arm binds CD3 on any passing T cell","An artificial immune synapse forms, independent of MHC","The T cell releases perforin and granzymes into the tumour cell","Tumour cell dies; cytokines are released (source of CRS)"],"dosing":{"route":"Continuous IV infusion","schedule":"Adults: 9 µg/day days 1-7 then 28 µg/day to day 28, 14-day break; 42-day cycles; weight-based in children","modifications":"Interrupt for grade 3 CRS or neurotoxicity; dexamethasone premedication","monitoring":"Hospitalisation for first 9 days of cycle 1 and first 2 days of cycle 2; neurological exams; infection surveillance","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=38b482a8-960b-4591-9857-5031ecb830aa"},"toxicity":[{"event":"Neurological toxicities","anyGradePct":65,"grade3PlusPct":13,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=38b482a8-960b-4591-9857-5031ecb830aa","note":"Relapsed/refractory adult ALL"},{"event":"Pyrexia","anyGradePct":55,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=38b482a8-960b-4591-9857-5031ecb830aa","note":"Relapsed/refractory adult ALL"},{"event":"Febrile neutropenia","anyGradePct":31,"grade3PlusPct":28,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=38b482a8-960b-4591-9857-5031ecb830aa","note":"Relapsed/refractory adult ALL"},{"event":"Infections","anyGradePct":28,"grade3PlusPct":15,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=38b482a8-960b-4591-9857-5031ecb830aa","note":"Relapsed/refractory adult ALL"},{"event":"Cytokine release syndrome","anyGradePct":14,"grade3PlusPct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=38b482a8-960b-4591-9857-5031ecb830aa","note":"Relapsed/refractory adult ALL"},{"event":"Infusion-related reactions","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=38b482a8-960b-4591-9857-5031ecb830aa","note":"Relapsed/refractory adult ALL"},{"event":"Headache","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=38b482a8-960b-4591-9857-5031ecb830aa","note":"Relapsed/refractory adult ALL"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.amgensupportplus.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended in Ph-negative relapsed/refractory B-ALL (TA450) and MRD-positive ALL (TA589)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2014-12-03","type":"accelerated-approval","region":"US","note":"Accelerated approval, Ph-negative relapsed/refractory B-ALL: first BiTE","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Philadelphia chromosome negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia"},{"date":"2017-07-11","type":"conversion","region":"US","note":"Full approval including Ph+ ALL","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Philadelphia chromosome negative relapsed or refractory B-cell precursor acute lymphoblastic leukemia"},{"date":"2018-03-29","type":"accelerated-approval","region":"US","note":"MRD-positive B-ALL: first approval based on MRD","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL) in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1% in adults and children 1"},{"date":"2023-06-20","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 5.2 years after it was granted.","source":"https://www.fda.gov#footnote1_815g9a4","indication":"Treatment of CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL) in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1% in adults and children 1"},{"date":"2024-06-14","type":"approval","region":"US","note":"Consolidation in CD19+ Ph-negative B-ALL regardless of MRD (E1910)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"bms-986365","kind":"drug","name":"BMS-986365","aka":[],"tldr":"BMS-986365 is an experimental protein degrader from Celgene in phase 3 trials for prostate cancer, aimed at Androgen receptor.","summary":"BMS-986365 is a protein degrader developed by Celgene. Its target is Androgen receptor (the sponsor names androgen receptor (AR)). The sponsor states: A dual androgen receptor ligand-directed degrader and antagonist, i.e. it both degrades the AR protein and blocks its activity. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06764485 (A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer), in prostate cancer. The largest, NCT06764485, plans to enrol 960 participants with primary completion expected 2027-09-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BMS-986365","url":"https://clinicaltrials.gov/search?intr=BMS-986365"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["androgen-receptor"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06764485"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"protein degrader","mechanism":"A dual androgen receptor ligand-directed degrader and antagonist, i.e. it both degrades the AR protein and blocks its activity.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bnt113","kind":"drug","name":"BNT113","aka":[],"tldr":"BNT113 is an experimental investigational agent whose form is not stated in the registry from BioNTech SE in phase 3 trials for head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"BNT113 is an investigational agent whose form is not stated in the registry developed by BioNTech SE. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04534205 (A Clinical Trial Investigating the Safety, Tolerability, and Therapeutic Effects of BNT113 in Combination With Pembrolizumab Versus Pembrolizumab Alone for Patients With a Form of Head and Neck Cancer Positive for Human Papilloma Virus 16 and Expressing the Protein PD-L1), in head and neck squamous cell carcinoma. The largest, NCT04534205, plans to enrol 358 participants (actual) with primary completion expected 2028-08. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BNT113","url":"https://clinicaltrials.gov/search?intr=BNT113"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["biontech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04534205"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"mRNA vaccine (per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bnt324","kind":"drug","name":"BNT324","aka":[],"tldr":"BNT324 is an experimental antibody-drug conjugate from BioNTech SE in phase 3 trials for prostate cancer and non-small-cell lung cancer, aimed at B7-H3.","summary":"BNT324 (DB-1311) is an antibody-drug conjugate developed by BioNTech SE. Its target is B7-H3 (the sponsor names B7-H3). The sponsor states: BNT324 (DB-1311) is an antibody-drug conjugate targeting B7-H3, given as an intravenous infusion in 21-day cycles; a phase 3 trial compares it with docetaxel in metastatic castration-resistant prostate cancer. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07365995 (A Phase III Trial of BNT324 Versus Docetaxel in Metastatic Castration-resistant Prostate Cancer), in prostate cancer and non-small-cell lung cancer. The largest, NCT07365995, plans to enrol 736 participants with primary completion expected 2031-02. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of BNT324","url":"https://clinicaltrials.gov/search?intr=DB-1311"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate","nsclc"],"sections":[],"technologies":[],"targets":["b7h3"],"drugs":[],"companies":["biontech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07365995","nct05914116","nct06892548","nct06953089"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"DB-1311","modality":"ADC","mechanism":"BNT324 (DB-1311) is an antibody-drug conjugate targeting B7-H3, given as an intravenous infusion in 21-day cycles; a phase 3 trial compares it with docetaxel in metastatic castration-resistant prostate cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bomedemstat","kind":"drug","name":"Bomedemstat","aka":[],"tldr":"Bomedemstat is an experimental small-molecule drug from Merck Sharp & Dohme in phase 3 trials for myeloproliferative neoplasms, with its target not yet stated publicly.","summary":"Bomedemstat (MK-3543, IMG-7289) is a small-molecule drug developed by Merck Sharp & Dohme. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06351631 (A Study to Evaluate Safety and Efficacy of Bomedemstat (MK-3543-017)), in myeloproliferative neoplasms. The largest, NCT06351631, plans to enrol 400 participants with primary completion expected 2034-12-04. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Bomedemstat","url":"https://clinicaltrials.gov/search?intr=MK-3543"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["myeloproliferative-neoplasms","polycythaemia-vera","essential-thrombocythaemia"],"sections":[],"technologies":["lsd1-inhibitors"],"targets":[],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06351631"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"MK-3543, IMG-7289","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bortezomib","kind":"drug","name":"Bortezomib","aka":[],"tldr":"Bortezomib was the first proteasome inhibitor: it jams the cell's protein-recycling machine, which antibody-factory plasma cells cannot tolerate.","summary":"Bortezomib is a reversible inhibitor of the chymotrypsin-like site of the 26S proteasome; the resulting unfolded-protein stress and NF-kappaB inhibition trigger apoptosis in plasma cells, which produce huge amounts of antibody and cannot tolerate a jammed protein-recycling system. It was the first proteasome inhibitor, approved in 2003 for relapsed myeloma and in 2008 for newly diagnosed disease. Subcutaneous weekly dosing cut peripheral neuropathy from around 50% to around 30% and is now preferred over intravenous twice-weekly schedules. It is part of VRd and Dara-VRd induction and of bortezomib-based regimens in mantle cell lymphoma and amyloidosis, and it is now generic. Thrombocytopenia and herpes zoster reactivation, which needs prophylaxis, are the other main issues. Bortezomib made proteasome inhibition a pillar of myeloma treatment and remains in most frontline regimens.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Bortezomib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Bortezomib"}],"tags":[],"related":["green-tea-egcg"],"cancers":["multiple-myeloma","myeloma-transplant-eligible","plasma-cell-leukaemia"],"sections":[],"technologies":["kinase-inhibitors"],"targets":[],"drugs":[],"companies":["takeda","johnson-johnson"],"institutions":[],"pathways":["ubiquitin-proteasome-system"],"terms":[],"trials":["nct06868654","nct06464991","nct06956170","nct05572515","nct05623020","nct06152575","nct05519085","nct04975997","nct06413498","nct06158841","nct06208150","nct06868667","nct02343042","nct03314181","nct04414475","nct04133636","nct03989414","nct04068597","nct06577025","nct07227311","nct07018050","nct02773030"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Velcade","modality":"Proteasome inhibitor","mechanism":"Reversible inhibitor of the 26S proteasome chymotrypsin-like site; unfolded-protein stress and NF-κB inhibition trigger plasma-cell apoptosis.","approvals":[{"region":"US","year":2003,"indication":"Relapsed myeloma (accelerated)"},{"region":"US","year":2008,"indication":"Newly diagnosed myeloma"}],"mechanismSteps":[],"dosing":{"route":"Subcutaneous (preferred) or IV","schedule":"1.3 mg/m2 days 1, 4, 8, 11 (21-day) or weekly","monitoring":"Peripheral neuropathy, herpes zoster prophylaxis, thrombocytopenia"},"toxicity":[{"event":"Peripheral neuropathy","anyGradePct":38,"grade3PlusPct":6,"note":"subcutaneous (MMY-3021)"},{"event":"Thrombocytopenia","grade3PlusPct":30}],"access":[],"regulatoryEvents":[{"date":"2003-05-13","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Multiple myeloma patients who received at least two prior therapies and demontrated disease progression on the last therapy"},{"date":"2005-03-25","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2003 converted to traditional approval 1.9 years after it was granted.","indication":"Multiple myeloma patients who received at least two prior therapies and demontrated disease progression on the last therapy"}]},{"id":"boserolimab","kind":"drug","name":"Boserolimab","aka":["MK-5890"],"tldr":"Boserolimab is a monoclonal antibody from Merck Sharp & Dohme LLC, in registered phase 2 trials for non-small-cell lung cancer.","summary":"Boserolimab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Merck Sharp & Dohme LLC, in non-small-cell lung cancer. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Boserolimab","url":"https://clinicaltrials.gov/search?intr=Boserolimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04165070"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bosutinib","kind":"drug","name":"Bosutinib","aka":[],"tldr":"Bosutinib is a CML pill with less cardiovascular and pleural toxicity than its rivals; its main side effect is diarrhoea.","summary":"Bosutinib is a dual SRC/ABL tyrosine kinase inhibitor for Philadelphia-chromosome-positive chronic myeloid leukaemia, active against most imatinib-resistant BCR-ABL1 mutations except T315I and V299L. It was approved in 2012 for CML after prior TKI therapy (EU 2013) and in 2017 for newly diagnosed chronic-phase disease after the BFORE trial. Its niche is defined by what it avoids: it is preferred for patients with cardiovascular risk, where nilotinib and ponatinib carry arterial occlusive risk, and for those at risk of pleural effusion with dasatinib. Diarrhoea is its main side effect, usually early and manageable, along with liver enzyme rises. It served as the comparator arm in ASCEMBL, the trial that established asciminib in later-line CML, and where it sits against asciminib is now the open question. For a newcomer: a CML pill chosen for the side effects it does not cause.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Bosutinib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=bosutinib"}],"tags":["gap-fill"],"related":[],"cancers":["cml","cml-chronic-phase"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["bcr-abl"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04971226"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Bosulif","modality":"Small-molecule BCR-ABL1/SRC TKI","mechanism":"Dual SRC/ABL inhibitor active against most imatinib-resistant BCR-ABL1 mutations except T315I and V299L.","approvals":[{"region":"US","year":2012,"indication":"Ph+ CML after prior TKI"},{"region":"US","year":2017,"indication":"Newly diagnosed Ph+ CML chronic phase"},{"region":"EU","year":2013,"indication":"Ph+ CML"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2017-12-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adults with newly diagnosed chronic phase Philadelphia chromosome positive CML"},{"date":"2021-05-14","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 3.4 years after it was granted.","indication":"Adults with newly diagnosed chronic phase Philadelphia chromosome positive CML"}]},{"id":"botensilimab","kind":"drug","name":"Botensilimab","aka":[],"tldr":"Botensilimab is an experimental monoclonal antibody from Agenus in phase 2 trials for colorectal cancer, aimed at CTLA-4.","summary":"Botensilimab (AGEN1181) is a monoclonal antibody developed by Agenus. Its target is CTLA-4 (the sponsor names CTLA-4). The sponsor states: Botensilimab is an Fc-enhanced anti-CTLA-4 monoclonal antibody designed to prime a broader anti-tumour immune response, typically studied in combination with balstilimab (anti-PD-1). ClinicalTrials.gov describes the intervention as: An anti-cytotoxic T-lymphocyte antigen 4 (CTLA-4) monoclonal antibody. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in colorectal cancer. The largest, NCT05608044, plans to enrol 234 participants (actual) with primary completion expected 2027-09. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Botensilimab","url":"https://clinicaltrials.gov/search?intr=AGEN1181"},{"label":"Sponsor pipeline page","url":"https://agenusbio.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["ctla4"],"drugs":[],"companies":["agenus"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06589440"],"people":[],"bottlenecks":[],"keyPapers":["paper-bullock-botensilimab-balstilimab-mss-colorectal-nat-med-2024"],"journals":[],"dependsOn":[],"notes":["Colorectal cancer biomarkers: this is the one agent aimed at the microsatellite-stable majority rather than the deficient minority. With balstilimab it gave an objective response rate of 17% and disease control of 61% in 101 response-evaluable microsatellite-stable patients out of 148 treated, with no biomarker threshold beyond microsatellite stability and prior treatment (Bullock 2024)."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AGEN1181","modality":"monoclonal antibody","mechanism":"Botensilimab is an Fc-enhanced anti-CTLA-4 monoclonal antibody designed to prime a broader anti-tumour immune response, typically studied in combination with balstilimab (anti-PD-1).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bracanalysis-cdx","kind":"drug","name":"BRACAnalysis CDx","aka":[],"tldr":"The inherited BRCA test that decides who can have PARP inhibitor pills for ovarian, breast, pancreatic and prostate cancer.","summary":"Approved 19 December 2014 (PMA P140020) as the companion diagnostic for olaparib in germline BRCA-mutated advanced ovarian cancer, the first FDA-approved companion diagnostic based on an inherited variant. Claims followed for olaparib and talazoparib in HER2-negative metastatic breast cancer (2018), olaparib maintenance in first-line ovarian cancer (SOLO-1, 2018), pancreatic cancer (POLO, 2019), metastatic castration-resistant prostate cancer (PROfound, 2020) and early breast cancer (OlympiA, 2022). Myriad's monopoly on BRCA testing ended with the 2013 US Supreme Court ruling that isolated genes cannot be patented, and multi-gene hereditary panels have since become the norm, but BRACAnalysis CDx remains the labelled test.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P140020","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P140020"}],"tags":["test"],"related":[],"cancers":["ovarian","breast-hr-positive","tnbc","pancreatic","prostate"],"sections":[],"technologies":["germline-testing","hrd-testing","companion-diagnostic"],"targets":["brca","parp"],"drugs":["olaparib","talazoparib"],"companies":["myriad-genetics"],"institutions":[],"pathways":[],"terms":["gbrca-mutation","germline-vs-somatic"],"trials":["solo-1"],"people":[],"bottlenecks":[],"keyPapers":["paper-couch-tnbc-germline-17-genes-jco-2015","paper-polo-olaparib-maintenance-gbrca-pancreatic-nejm-2019"],"journals":[],"dependsOn":[],"notes":["What a result means: a pathogenic BRCA1/2 variant makes PARP inhibitors an option, raises the case for risk-reducing surgery or surveillance, and means blood relatives can be tested.","Pancreatic ductal adenocarcinoma: POLO selected patients on a germline BRCA1/2 result from this assay, the companion diagnostic for olaparib maintenance (Golan 2019)."],"brand":"BRACAnalysis CDx","modality":"Germline BRCA1/2 sequencing companion diagnostic test","mechanism":"Sanger and NGS sequencing plus large-rearrangement analysis of germline BRCA1 and BRCA2 from blood, classifying variants as deleterious or suspected deleterious.","approvals":[{"region":"US","year":2014,"indication":"Companion diagnostic for olaparib in germline BRCA-mutated advanced ovarian cancer"},{"region":"US","year":2018,"indication":"Olaparib and talazoparib in germline BRCA-mutated HER2-negative metastatic breast cancer"},{"region":"US","year":2019,"indication":"Olaparib maintenance in germline BRCA-mutated metastatic pancreatic cancer"},{"region":"US","year":2020,"indication":"Olaparib in germline BRCA-mutated metastatic castration-resistant prostate cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"breast-cancer-index","kind":"drug","name":"Breast Cancer Index","aka":[],"tldr":"The only test designed to tell a woman who has finished five years of hormone therapy whether another five years is worth the side effects.","summary":"Breast Cancer Index (Biotheranostics, acquired by Hologic in 2021) is a laboratory-developed test for hormone-receptor-positive early breast cancer. Its HOXB13:IL17BR component was shown to predict benefit from extending endocrine therapy to 10 years in the MA.17, Trans-aTTom and IDEAL trial cohorts, and the ASCO and NCCN guidelines list it as the test for that decision; a prespecified NSABP B-42 analysis was less clear-cut. It also gives a late-recurrence risk (years 5 to 10). Medicare covers it. The test does not address the initial chemotherapy decision, which is the province of Oncotype DX, MammaPrint, Prosigna and EndoPredict.","status":"established","asOf":"2026-09-10","links":[{"label":"Breast Cancer Index","url":"https://www.breastcancerindex.com"},{"label":"ASCO biomarker guideline update (2022)","url":"https://doi.org/10.1200/JCO.22.00069"}],"tags":["test"],"related":["oncotype-dx","endopredict"],"cancers":["breast-hr-positive"],"sections":[],"technologies":["rna-seq","endocrine-therapy"],"targets":[],"drugs":[],"companies":["hologic"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-andre-j-clin-oncol"],"journals":[],"dependsOn":[],"notes":["What a result means: 'BCI (H/I) high' predicts that continuing hormone therapy past five years reduces recurrence; 'low' means the extra years add side effects without measurable benefit."],"brand":"Breast Cancer Index (BCI)","modality":"Gene-expression prognostic and predictive assay (extended endocrine therapy benefit)","mechanism":"RT-PCR combining the HOXB13:IL17BR ratio (predicts benefit from extended endocrine therapy) with the Molecular Grade Index (prognosis for late recurrence).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"brenetafusp","kind":"drug","name":"Brenetafusp","aka":[],"tldr":"Brenetafusp is a soluble T-cell receptor bispecific, tebentafusp's successor, that recognises a PRAME peptide on HLA-A*02:01 and drags T cells onto the tumour. A phase 3 with nivolumab in first-line melanoma is enrolling, but only patients carrying HLA-A*02:01, about half of those of European ancestry, are eligible.","summary":"Phase 1/2 in heavily pretreated cutaneous melanoma: median OS 14.3 months on monotherapy (ASCO 2026 update). PRISM-MEL-301 phase 3 with nivolumab in first-line HLA-A*02:01-positive melanoma is enrolling; further trials in ovarian, lung, and endometrial cancer. Requires HLA-A*02:01 (roughly half of European-ancestry patients).","status":"phase-3","asOf":"2026-09-07","links":[{"label":"Immunocore ASCO 2026 data","url":"https://www.globenewswire.com/news-release/2026/05/31/3303917/0/en/immunocore-presents-updated-phase-1-data-of-brenetafusp-in-patients-with-heavily-pretreated-advanced-melanoma.html"}],"tags":[],"related":[],"cancers":["melanoma","ovarian","nsclc"],"sections":[],"technologies":["t-cell-engager"],"targets":["prame","cd3","hla-a"],"drugs":["tebentafusp"],"companies":["immunocore"],"institutions":[],"pathways":[],"terms":[],"trials":["prism-mel-301","nct04262466"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"IMC-F106C","modality":"ImmTAC (PRAME TCR × CD3 bispecific)","mechanism":"Affinity-enhanced TCR binding PRAME peptide-HLA-A*02:01 fused to an anti-CD3 scFv; redirects polyclonal T cells.","approvals":[],"mechanismSteps":["The TCR arm binds PRAME peptide displayed on HLA-A*02:01 on the tumour cell","The CD3 arm engages any nearby T cell","An artificial immune synapse forms and the T cell releases perforin and granzymes","Interferon-gamma release upregulates PD-L1, the rationale for pairing with nivolumab"],"dosing":{"route":"Intravenous","schedule":"Weekly with step-up dosing to 160 µg","monitoring":"Cytokine release syndrome during the first doses; rash","source":"https://clinicaltrials.gov/study/NCT06112314"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2024","type":"designation","region":"US","note":"Phase 3 PRISM-MEL-301 initiated after FDA alignment on design"}]},{"id":"brentuximab-vedotin","kind":"drug","name":"Brentuximab vedotin","aka":[],"tldr":"The ADC that made the modern field credible (2011), for Hodgkin lymphoma and CD30+ lymphomas.","summary":"Brentuximab vedotin is a chimeric anti-CD30 IgG1 antibody carrying the microtubule poison MMAE (drug-to-antibody ratio about 4) through a cleavable mc-vc-PABC linker; inside a CD30-positive cell MMAE blocks mitosis and diffuses to kill neighbouring cells. Approved in 2011 for relapsed Hodgkin lymphoma and systemic anaplastic large-cell lymphoma, it made the modern ADC field credible and later moved to first line with AVD (ECHELON-1, overall survival benefit), CD30-positive peripheral T-cell lymphoma with CHP (ECHELON-2), post-transplant consolidation (AETHERA) and paediatric Hodgkin lymphoma. Peripheral neuropathy affected 65 percent of the A+AVD arm in ECHELON-1 and neutropenia 91 percent, so G-CSF prophylaxis is mandatory and bleomycin is contraindicated. Its vc-MMAE linker-payload was reused in enfortumab, polatuzumab, tisotumab and disitamab vedotin.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Brentuximab_vedotin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Brentuximab%20vedotin"},{"label":"Kuppers, Nat Rev Cancer 2009: the biology of Hodgkin's lymphoma","url":"https://doi.org/10.1038/nrc2542"}],"tags":[],"related":["cd30-expression"],"cancers":["hodgkin-lymphoma","cutaneous-t-cell-lymphoma","advanced-stage-classical-hodgkin-lymphoma","relapsed-refractory-hodgkin-lymphoma"],"sections":[],"technologies":["adc"],"targets":[],"drugs":[],"companies":["pfizer","takeda"],"institutions":[],"pathways":[],"terms":["mc-vc-pabc"],"trials":["nct04404283","nct02979522","nct05673785"],"people":[],"bottlenecks":[],"keyPapers":["paper-alcanza-brentuximab-vedotin-lancet-2017"],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: CD30 is as close as lymphoma gets to an antigen restricted to the tumour in an adult, because resting lymphocytes do not carry it. That is why this conjugate does not produce the compartment-emptying effects of the anti-B-cell agents and why its limiting toxicity is the auristatin payload. In classical Hodgkin lymphoma the antigen is on essentially every Reed-Sternberg cell even though those cells are a small minority of the tissue (Kuppers 2009)."],"brand":"Adcetris","modality":"ADC","payload":"MMAE, DAR ~4","linker":"mc-vc-PABC, cleavable","mechanism":"Chimeric anti-CD30 IgG1 with MMAE.","approvals":[{"region":"US","year":2011,"indication":"Relapsed Hodgkin lymphoma and systemic ALCL"},{"region":"US","year":2018,"indication":"First-line stage III/IV Hodgkin lymphoma with AVD"}],"mechanismSteps":["Antibody binds CD30 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","MMAE is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"1.8 mg/kg (max 180 mg) every 3 weeks; 1.2 mg/kg (max 120 mg) every 2 weeks with AVD","modifications":"G-CSF prophylaxis mandatory with AVD; hold for grade 2 neuropathy; contraindicated with bleomycin","monitoring":"Neuropathy, blood counts, PML symptoms, LFTs","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=3904f8dd-1aef-3490-e48f-bd55f32ed67f"},"toxicity":[{"event":"Neutropenia","anyGradePct":91,"grade3PlusPct":82,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=3904f8dd-1aef-3490-e48f-bd55f32ed67f","note":"ECHELON-1, A+AVD arm"},{"event":"Peripheral sensory neuropathy","anyGradePct":65,"grade3PlusPct":10,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=3904f8dd-1aef-3490-e48f-bd55f32ed67f","note":"ECHELON-1, A+AVD arm"},{"event":"Constipation","anyGradePct":42,"grade3PlusPct":2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=3904f8dd-1aef-3490-e48f-bd55f32ed67f","note":"ECHELON-1, A+AVD arm"},{"event":"Nausea","anyGradePct":40,"grade3PlusPct":2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=3904f8dd-1aef-3490-e48f-bd55f32ed67f","note":"ECHELON-1, A+AVD arm"},{"event":"Vomiting","anyGradePct":33,"grade3PlusPct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=3904f8dd-1aef-3490-e48f-bd55f32ed67f","note":"ECHELON-1, A+AVD arm"},{"event":"Febrile neutropenia","anyGradePct":19,"grade3PlusPct":19,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=3904f8dd-1aef-3490-e48f-bd55f32ed67f","note":"ECHELON-1, A+AVD arm"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended in CD30+ Hodgkin lymphoma settings (TA446, TA524) and first-line stage IV with AVD","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2011-08-19","type":"accelerated-approval","region":"US","note":"Accelerated approval, relapsed Hodgkin lymphoma and systemic ALCL","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Hodgkin lymphoma after failure of autologous stem cell transplant (ASCT), or after failure of at least 2 multi-agent chemotherapy regimens in patients who are not ASCT candidates"},{"date":"2011-08-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Systemic anaplastic large cell lymphoma after failure of at least one multi-agent chemotherapy regimen"},{"date":"2015-08-17","type":"conversion","region":"US","note":"Post-transplant consolidation in Hodgkin lymphoma (AETHERA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Hodgkin lymphoma after failure of autologous stem cell transplant (ASCT), or after failure of at least 2 multi-agent chemotherapy regimens in patients who are not ASCT candidates"},{"date":"2018-03-20","type":"conversion","region":"US","note":"First-line stage III/IV Hodgkin lymphoma with AVD (ECHELON-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Systemic anaplastic large cell lymphoma after failure of at least one multi-agent chemotherapy regimen"},{"date":"2018-11-16","type":"approval","region":"US","note":"First-line CD30+ peripheral T-cell lymphoma with CHP (ECHELON-2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-11-10","type":"approval","region":"US","note":"Paediatric high-risk Hodgkin lymphoma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"brexucabtagene-autoleucel","kind":"drug","name":"Brexucabtagene autoleucel","aka":[],"tldr":"Brexucabtagene autoleucel is the CAR-T therapy for mantle cell lymphoma and adult acute lymphoblastic leukaemia, giving long remissions after BTK inhibitors fail.","summary":"Brexucabtagene autoleucel is an autologous CD19 CAR-T with a CD28 costimulatory domain, the same construct as axicabtagene, manufactured with a T-cell enrichment step that removes circulating tumour cells from the leukapheresis product. It is used for relapsed or refractory mantle cell lymphoma after BTK inhibitor failure and for adults with relapsed or refractory B-cell acute lymphoblastic leukaemia. ZUMA-2 (NEJM 2020) reported a 93% response rate and 67% complete remissions in BTKi-exposed MCL, with a median progression-free survival of about 30 months in updates. ZUMA-3 showed 71% CR or CRi in adult B-ALL, leading to the 2021 approval. Cytokine release syndrome and neurotoxicity are frequent, with grade 3 or higher neurological events in roughly 30%. In short, one infusion can produce long remissions in two diseases that were hard to control after relapse.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Brexucabtagene_autoleucel","links":[{"label":"ZUMA-2 (NEJM 2020)","url":"https://doi.org/10.1056/NEJMoa1914347"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Tecartus"}],"tags":["gap-fill"],"related":[],"cancers":["mantle-cell-lymphoma","all-leukemia"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":[],"companies":["gilead"],"institutions":[],"pathways":[],"terms":["crs","icans"],"trials":["nct06253663"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tecartus","modality":"Autologous CD19 CAR-T (CD28 costimulatory domain)","mechanism":"Anti-CD19 CAR T cells manufactured with T-cell enrichment to remove circulating tumour cells; same construct as axicabtagene.","approvals":[{"region":"US","year":2020,"indication":"Relapsed/refractory mantle cell lymphoma"},{"region":"US","year":2021,"indication":"Relapsed/refractory B-ALL in adults"},{"region":"EU","year":2020,"indication":"MCL after ≥2 lines including a BTK inhibitor"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2020-07-24","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL)."},{"date":"2026-04-01","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 5.7 years after it was granted.","indication":"Treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL)."}]},{"id":"brigatinib","kind":"drug","name":"Brigatinib","aka":[],"tldr":"Brigatinib is an ALK pill with strong brain activity, approved first after crizotinib and then first line after beating it in ALTA-1L.","summary":"Brigatinib is a second-generation ALK inhibitor with broad activity against resistance mutations including G1202R, and at higher doses it also inhibits wild-type EGFR; it penetrates the brain well. The ALTA trial (2017) established activity after crizotinib, and ALTA-1L (2018 to 2020) showed first-line superiority over crizotinib with median progression-free survival of 24 versus 11 months and a 78% intracranial response rate. It was approved in the US in 2017 after crizotinib and in 2020 first line, and in the EU in 2018. Early pulmonary events in the first week are its distinctive toxicity, which is why treatment starts with a 90 mg lead-in dose before escalation. It competes with alectinib and lorlatinib in the first line, and no trial has directly compared them. Brigatinib is an ALK pill whose main strengths are brain activity and coverage of resistance mutations.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Brigatinib","links":[{"label":"ALTA (JCO 2017)","url":"https://doi.org/10.1200/JCO.2016.71.5904"},{"label":"ALTA-1L (NEJM 2018)","url":"https://doi.org/10.1056/NEJMoa1810171"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=brigatinib"},{"label":"NICE TA571: brigatinib for treating ALK-positive advanced non-small-cell lung cancer after crizotinib","url":"https://www.nice.org.uk/guidance/ta571"},{"label":"NICE TA670: brigatinib for ALK-positive advanced non-small-cell lung cancer not previously treated with an ALK inhibitor","url":"https://www.nice.org.uk/guidance/ta670"}],"tags":["gap-fill"],"related":["alk-fusion"],"cancers":["nsclc","alk-positive-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["alk"],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["alta","alta-1l"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Alunbrig","modality":"Small-molecule ALK/EGFR TKI (second generation)","mechanism":"Broad-spectrum ALK inhibitor with activity against G1202R and other resistance mutations plus wild-type EGFR at higher doses.","approvals":[{"region":"US","year":2017,"indication":"ALK-positive metastatic NSCLC after crizotinib"},{"region":"US","year":2020,"indication":"First-line ALK-positive metastatic NSCLC"},{"region":"EU","year":2018,"indication":"ALK-positive NSCLC"},{"region":"England (NICE)","year":2019,"indication":"ALK-positive advanced non-small-cell lung cancer after crizotinib","note":"TA571, published 20 March 2019, subject to the commercial arrangement."},{"region":"England (NICE)","year":2021,"indication":"ALK-positive advanced non-small-cell lung cancer not previously treated with an ALK inhibitor","note":"TA670, published 27 January 2021 (ALTA-1L), subject to the commercial arrangement."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2017-04-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Patients with ALK-positive metastatic NSCLC that have progressed or are intolerant to crizotinib"},{"date":"2020-05-22","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 3.1 years after it was granted.","indication":"Patients with ALK-positive metastatic NSCLC that have progressed or are intolerant to crizotinib"}]},{"id":"brigimadlin","kind":"drug","name":"Brigimadlin","aka":["BI 907828","BI-907828"],"tldr":"Brigimadlin is a tablet that frees the p53 'guardian' protein from MDM2, the protein that liposarcomas make in excess to keep p53 switched off. It is being compared with doxorubicin as first treatment for dedifferentiated liposarcoma in a phase 2/3 trial.","summary":"Brigimadlin (BI 907828) is Boehringer Ingelheim's oral MDM2-p53 antagonist, dosed once every three weeks to limit the thrombocytopenia that troubled earlier MDM2 inhibitors. Dedifferentiated liposarcoma almost always carries MDM2 amplification with wild-type TP53, so it is the model disease for the class. In the phase 1a/1b trial, responses and long disease control were seen in MDM2-amplified liposarcoma.\n\nThe Brightline-1 phase 2/3 trial compares brigimadlin with doxorubicin as first-line treatment for advanced dedifferentiated liposarcoma, and Brightline-2 tests it in other MDM2-amplified, TP53 wild-type cancers such as biliary tract and pancreatic cancer. The liposarcoma page names it with CDK4/6 inhibitors among the targeted drugs in late trials for MDM2-amplified disease.","status":"phase-3","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["doxorubicin"],"cancers":["liposarcoma"],"sections":[],"technologies":[],"targets":["mdm2"],"drugs":[],"companies":["boehringer-ingelheim"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"BI 907828","modality":"Oral MDM2-p53 antagonist","mechanism":"Binds MDM2 in the pocket that normally holds p53, releasing p53 from degradation; in tumours with MDM2 amplification and intact TP53, such as dedifferentiated liposarcoma, restored p53 stops the cycle and triggers cell death.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"bromocriptine","kind":"drug","name":"Bromocriptine","aka":["Bromocriptine mesylate"],"tldr":"Bromocriptine was the first pill that could shrink a pituitary tumour. From the 1970s it turned prolactinomas from a surgical disease into one usually controlled with tablets, and it is still used where cabergoline is unavailable or unsuitable.","summary":"Bromocriptine is an ergot-derived dopamine agonist developed by Sandoz (now Novartis). The FDA approved it in 1978 for hyperprolactinaemia-associated disorders, acromegaly and Parkinson's disease. Taken two or three times daily, it normalises prolactin, restores fertility and shrinks most prolactinomas, establishing medical therapy as the first-line treatment for these tumours.\n\nIt is now largely superseded by cabergoline, which is more potent, better tolerated and taken once or twice a week, but it remains the preferred agent when pregnancy is planned because of its longer safety record. The pituitary tumours page records it as the drug that established medical therapy for prolactinoma.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Bromocriptine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Bromocriptine"}],"tags":["subtype-drugs-wave"],"related":["cabergoline"],"cancers":["pituitary-tumours"],"sections":[],"technologies":[],"targets":["drd2"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Parlodel","modality":"Oral ergot dopamine D2 receptor agonist","mechanism":"Stimulates dopamine D2 receptors on pituitary lactotroph cells, which switches off prolactin secretion and shrinks prolactin-secreting adenomas; it also lowers growth hormone in some acromegaly.","approvals":[{"region":"US","year":1978,"indication":"Hyperprolactinaemia-associated dysfunction including prolactin-secreting adenomas; acromegaly; Parkinson's disease"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"budigalimab","kind":"drug","name":"Budigalimab","aka":[],"tldr":"Budigalimab is an experimental investigational agent whose form is not stated in the registry from AbbVie in phase 3 trials for hepatocellular carcinoma, non-small-cell lung cancer and bladder & urothelial cancer, with its target not yet stated publicly.","summary":"Budigalimab (ABBV-181) is an investigational agent whose form is not stated in the registry developed by AbbVie. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 5 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06109272 (A Study to Assess the Dose, Adverse Events, and Change in Disease Activity of Livmoniplimab as an Intravenous (IV) Solution in Combination With Budigalimab as an IV Solution in Adult Participants With Hepatocellular Carcinoma (HCC)) and NCT06236438 (Study to Evaluate Adverse Events, Optimal Dose, and Change in Disease Activity, With Livmoniplimab in Combination With Budigalimab Plus Chemotherapy Versus IV Infused Pembrolizumab Plus Chemotherapy in Adult Participants With Untreated Metastatic Non-Squamous Non-Small Cell Lung Cancer (NSCLC)), in hepatocellular carcinoma, non-small-cell lung cancer and bladder & urothelial cancer. The largest, NCT06236438, plans to enrol 840 participants with primary completion expected 2031-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Budigalimab","url":"https://clinicaltrials.gov/search?intr=ABBV-181"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hcc","nsclc","urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06109272","nsclc-2","nct05822752","nct06772623","nct06632951"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ABBV-181","modality":"antibody (INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"burosumab","kind":"drug","name":"Burosumab","aka":["burosumab","Crysvita","KRN23"],"tldr":"Crysvita is an antibody for a rare bone-softening condition in which the body leaks phosphate. Some small tumours cause it by making too much of a hormone called FGF23; when the tumour cannot be found or removed, Crysvita blocks the hormone.","summary":"Burosumab (Crysvita) has been authorised in the EU since 19 February 2018 (CHMP opinion 14 December 2017; marketing authorisation holder Kyowa Kirin Holdings B.V.; orphan designation) for X-linked hypophosphataemia in children and adolescents aged 1 to 17 with radiographic bone disease and in adults, and for FGF23-related hypophosphataemia in tumour-induced osteomalacia associated with phosphaturic mesenchymal tumours that cannot be curatively resected or localised. The cancer-care indication is the second: it treats the metabolic effect of a tumour rather than the tumour itself.","status":"approved","asOf":"2026-09-24","links":[{"label":"UX023T-CL201 (J Bone Miner Res 2021)","url":"https://doi.org/10.1002/jbmr.4233"},{"label":"EMA: Crysvita (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/crysvita"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=102f96a0-6e3a-4fc1-b204-34d604683af6"}],"tags":["ema-register","supportive"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["kyowa-kirin"],"institutions":[],"pathways":[],"terms":[],"trials":["ux023t-cl201"],"people":[],"bottlenecks":[],"keyPapers":["paper-ux023t-cl201-jan-de-beur-jbmr-2021"],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo content wave 5 (EMA medicines register and EPAR pages)","editedOn":"2026-09-22"},"brand":"Crysvita","modality":"Monoclonal antibody (anti-FGF23)","supportive":true,"mechanism":"Antibody to fibroblast growth factor 23; used for FGF23-related hypophosphataemia, including the form caused by phosphate-wasting tumours (tumour-induced osteomalacia), where the tumour cannot be found or removed (indication wording, EMA).","approvals":[{"region":"EU","year":2018,"indication":"X-linked hypophosphataemia; FGF23-related hypophosphataemia in tumour-induced osteomalacia (phosphaturic mesenchymal tumours not curatively resectable or localisable)","note":"Authorised 19 Feb 2018; Kyowa Kirin"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"buserelin","kind":"drug","name":"Buserelin","aka":[],"tldr":"Buserelin is one of the original GnRH agonists, approved in Europe and Canada in the 1980s for advanced prostate cancer and also used in endometriosis and fertility treatment; it is not sold in the United States.","summary":"Buserelin, developed by Hoechst, was among the first GnRH agonists to reach the market and has been used since the mid-1980s in Europe, Canada and elsewhere for hormone-dependent advanced prostate cancer, given as a depot injection or nasal spray, with the same testosterone suppression and tumour flare as its class. Its wider use has been in endometriosis and in vitro fertilisation cycles. Goserelin, leuprolide and triptorelin displaced it in most oncology settings because of their longer depot formulations.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Buserelin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Buserelin"},{"label":"ChEMBL CHEMBL1201248","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201248"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["endocrine-therapy"],"targets":["gnrhr"],"drugs":["leuprolide","goserelin","triptorelin"],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04513717","nct00567580","nct00936390"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Suprefact","modality":"GnRH agonist, injection or nasal spray","mechanism":"A GnRH analogue that after an initial stimulation desensitises pituitary GnRH receptors, shutting down gonadotrophin release and so testosterone or oestrogen production.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"busulfan","kind":"drug","name":"Busulfan","aka":["Busilvex"],"tldr":"Busulfan is one of the oldest cancer drugs. As a tablet it controlled chronic myeloid leukaemia before imatinib; as an infusion it is used to clear the bone marrow before a stem cell transplant.","summary":"Oral busulfan (Myleran) was approved in 1954 for palliative treatment of chronic myelogenous leukaemia and was the standard until hydroxyurea, interferon and then imatinib displaced it. Intravenous busulfan (Busulfex, approved 1999) is indicated with cyclophosphamide as a conditioning regimen before allogeneic haematopoietic progenitor cell transplantation for CML, and pharmacokinetically targeted dosing is routine in transplant units. The EU authorised Busilvex in 2003 for conditioning before transplantation in adults and children. Hepatic sinusoidal obstruction syndrome, seizures (anticonvulsant prophylaxis is given) and prolonged myelosuppression are the main risks; pulmonary fibrosis (busulfan lung) follows chronic oral use.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Busulfan","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=busulfan"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/busulfan"},{"label":"EPAR (Busilvex)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/busilvex"}],"tags":["nci-list"],"related":[],"cancers":["cml"],"sections":[],"technologies":["cytotoxic-chemotherapy","allogeneic-hsct"],"targets":[],"drugs":[],"companies":["otsuka"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Myleran / Busulfex","modality":"Alkylating agent (alkyl sulfonate)","mechanism":"Bifunctional alkylating agent that cross-links DNA; highly lipophilic and myeloablative at transplant doses.","approvals":[{"region":"US","year":1954,"indication":"Palliative treatment of chronic myelogenous leukaemia (Myleran tablets)"},{"region":"US","year":1999,"indication":"Conditioning with cyclophosphamide before allogeneic HPC transplantation for CML (Busulfex)"},{"region":"EU","year":2003,"indication":"Conditioning before haematopoietic progenitor cell transplantation (Busilvex)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cabazitaxel","kind":"drug","name":"Cabazitaxel","aka":[],"tldr":"A second taxane that works after docetaxel and beat a second hormone pill head-to-head (CARD).","summary":"Cabazitaxel is a taxane with low affinity for the P-glycoprotein efflux pump, so it remains active in docetaxel-resistant disease. It is used in metastatic castration-resistant prostate cancer after docetaxel, with neutropenia as its main toxicity. TROPIC (2010) showed an overall survival benefit after docetaxel, and CARD (2019) showed it was superior to switching to a second androgen receptor pathway inhibitor in men who had already had one ARPI and docetaxel. It served as the comparator in TheraP, where it lost to 177Lu-PSMA-617 on PSA50 response (66% versus 37%) although overall survival was similar (HR 0.97), and in XALute against the T-cell engager xaluritamig. Its place relative to radioligands and T-cell engagers is therefore still being worked out. For a newcomer, it is the second taxane that works after docetaxel and beat a second hormone pill head-to-head.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Cabazitaxel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Cabazitaxel"},{"label":"NICE TA391: cabazitaxel for hormone-relapsed metastatic prostate cancer treated with docetaxel","url":"https://www.nice.org.uk/guidance/ta391"},{"label":"PROSELICA (Journal of Clinical Oncology 2017)","url":"https://doi.org/10.1200/JCO.2016.72.1076"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":["efflux-pump","prostate-crpc-sequencing","prostate-uk-drug-approvals"],"trials":["therap","xalute","nct07213674","nct07611110","nct06353386"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NICE TA391 recommends cabazitaxel with prednisone or prednisolone for metastatic hormone-relapsed prostate cancer that has progressed during or after docetaxel, only if the person has an ECOG performance status of 0 or 1, has had at least 225 mg/m2 of docetaxel, and stops treatment at progression or after a maximum of 10 cycles, with the commercial arrangement in place. PROSELICA showed that 20 mg/m2 is non-inferior to 25 mg/m2 for overall survival (13.4 against 14.5 months) with grade 3 or 4 adverse events in 39.7 against 54.5 percent, which is the basis for starting an older or frailer man on the lower dose."],"brand":"Jevtana","modality":"Cytotoxic chemotherapy (taxane)","mechanism":"Taxane with low P-glycoprotein affinity, active in docetaxel-resistant disease.","approvals":[{"region":"US","year":2010,"indication":"mCRPC after docetaxel"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cabergoline","kind":"drug","name":"Cabergoline","aka":["Cabaser"],"tldr":"Cabergoline is a tablet taken once or twice a week that shrinks prolactin-secreting pituitary tumours and normalises hormone levels in most patients, so surgery is reserved for the few whose tumours resist or who cannot tolerate it.","summary":"Cabergoline is an ergoline dopamine agonist developed by Farmitalia Carlo Erba, later Pharmacia and now Pfizer. The FDA approved it in 1996 for hyperprolactinaemic disorders. Its long half-life allows dosing once or twice weekly, and it normalises prolactin in roughly 80 to 90 percent of patients and shrinks macroprolactinomas in most, with less nausea than bromocriptine.\n\nIt is the first-line treatment for prolactinoma of any size; transsphenoidal surgery is reserved for resistance, intolerance or cystic tumours compressing the optic chiasm. High doses used in Parkinson's disease were linked to cardiac valve fibrosis, so echocardiographic monitoring is advised on long-term higher doses. The pituitary tumours page names it as the standard treatment for prolactinomas.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Cabergoline","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Cabergoline"}],"tags":["subtype-drugs-wave"],"related":["bromocriptine"],"cancers":["pituitary-tumours"],"sections":[],"technologies":[],"targets":["drd2"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07463235","nct03271918","nct00889525"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Dostinex","modality":"Oral long-acting dopamine D2 receptor agonist","mechanism":"A potent, long-acting dopamine D2 agonist that suppresses prolactin release from pituitary lactotrophs and shrinks prolactinomas, taken only once or twice a week.","approvals":[{"region":"US","year":1996,"indication":"Hyperprolactinaemic disorders, idiopathic or due to pituitary adenomas"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cabozantinib","kind":"drug","name":"Cabozantinib","aka":[],"tldr":"A pill that blocks both blood-vessel growth and the MET escape pathway, used in kidney, liver, thyroid and, since 2025, neuroendocrine cancers.","summary":"CELESTIAL: OS 10.2 vs 8.0 months after sorafenib in HCC (HR 0.76). CheckMate 9ER: with nivolumab, first-line RCC. CABINET (2024-25): PFS 13.8 vs 4.4 months in pancreatic NETs and 8.4 vs 3.9 months in extra-pancreatic NETs after prior therapy, leading to FDA approval in March 2025; ESMO 2025 subgroup showed an 81% reduction in progression risk in lung and thymic NETs. COSMIC-312 (with atezolizumab, first-line HCC) improved PFS but not OS.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Cabozantinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Cabozantinib"}],"tags":[],"related":[],"cancers":["hcc","hcc-advanced","rcc","neuroendocrine","thyroid","medullary-thyroid-cancer","clear-cell-rcc","papillary-rcc"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["met","vegf","ret"],"drugs":[],"companies":["exelixis","ipsen"],"institutions":[],"pathways":[],"terms":[],"trials":["celestial","cabinet","nct03755791","nct04446117","nct03690388","nct07011719","nct03634540","nct07293351","nct03899155","nct04586231","nct07405164","checkmate-9er","cosmic-313","contact-03"],"people":[],"bottlenecks":[],"keyPapers":["paper-exam-cabozantinib-mtc-elisei-jco-2013","paper-papmet-pal-lancet-2021"],"journals":[],"dependsOn":[],"notes":["In kidney cancer: CABOSUN (first line versus sunitinib, PFS benefit), METEOR (second line versus everolimus, OS 21.4 versus 16.5 months), CheckMate 9ER (with nivolumab, OS benefit), COSMIC-313 (triplet, no OS gain) and CONTACT-03 (no benefit from adding atezolizumab). The dose is 40 mg daily with nivolumab, and about 65 percent of patients have a grade 3 or higher event; the long half-life of about 99 hours matters when toxicity appears."],"brand":"Cabometyx","modality":"Small-molecule multi-kinase inhibitor (MET, VEGFR2, AXL, RET)","mechanism":"Oral inhibitor of MET, VEGFR2, AXL, RET, KIT and FLT3; MET/AXL inhibition counters anti-VEGF resistance.","approvals":[{"region":"US","year":2012,"indication":"Medullary thyroid cancer (Cometriq)"},{"region":"US","year":2016,"indication":"Advanced RCC"},{"region":"US","year":2019,"indication":"HCC previously treated with sorafenib"},{"region":"US","year":2025,"indication":"Advanced pancreatic and extra-pancreatic neuroendocrine tumours after prior therapy (CABINET)"},{"region":"US","year":2017,"indication":"First-line advanced RCC (CABOSUN)"},{"region":"US","year":2021,"indication":"First-line advanced RCC with nivolumab (CheckMate 9ER)"},{"region":"EU","year":2014,"indication":"Cometriq for medullary thyroid cancer; 21 Mar 2014. Cabometyx (RCC) Sep 2016"}],"mechanismSteps":["Blocks VEGFR2 on endothelium","Blocks MET and AXL, which tumours upregulate under hypoxia to escape anti-VEGF therapy","Reduces invasion and metastasis signalling"],"dosing":{"route":"Oral","schedule":"60 mg daily (Cabometyx tablets)","modifications":"40 mg then 20 mg for intolerance","monitoring":"Blood pressure, liver enzymes, wound healing"},"toxicity":[{"event":"Hand-foot skin reaction","anyGradePct":46,"grade3PlusPct":17,"note":"CELESTIAL"},{"event":"Hypertension","anyGradePct":29,"grade3PlusPct":16,"note":"CELESTIAL"},{"event":"Diarrhoea","anyGradePct":54,"grade3PlusPct":10,"note":"CELESTIAL"}],"access":[],"regulatoryEvents":[{"date":"2025-03-26","type":"approval","region":"US","note":"NET indication on CABINET","source":"https://www.ncf.net/post/2025-highlights-in-neuroendocrine-cancer"}]},{"id":"cadonilimab","kind":"drug","name":"Cadonilimab","aka":[],"tldr":"A Chinese two-armed antibody that blocks PD-1 and CTLA-4 together, approved in China for cervical cancer and extending survival even in PD-L1-negative tumours.","summary":"Cadonilimab is Akeso's tetravalent PD-1/CTLA-4 bispecific. NMPA approval June 2022 (second-line cervical cancer, ORR ~33%) and 2024 for first-line combination with chemotherapy ± bevacizumab (COMPASSION-16, OS HR 0.64). Also approved in China for first-line gastric cancer (COMPASSION-15, 2024). Designed for tumour-enriched dual blockade with lower immune toxicity than ipilimumab + nivolumab. It had not been approved in the US or EU by 2026.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Cadonilimab"}],"tags":[],"related":[],"cancers":["cervical","gastric"],"sections":[],"technologies":["bispecific-antibody","checkpoint-inhibitor"],"targets":["pd1","ctla4"],"drugs":[],"companies":["akeso"],"institutions":[],"pathways":["pd1-checkpoint"],"terms":[],"trials":["compassion-16","nct05489289","nct07412613","nct06617416","nct06371157","nct05932212","nct06646055","nct05904379","nct07052253","nct07245446","nct06560112","nct06530251","nct05235516","nct05944224","nct06221748","nct07023315","nct05859750"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Kaitanni","code":"AK104","modality":"Bispecific antibody (PD-1×CTLA-4)","mechanism":"Binds PD-1 and CTLA-4 simultaneously; higher avidity for cells co-expressing both (tumour-infiltrating T cells) concentrates activity in the tumour.","approvals":[{"region":"China","year":2022,"indication":"Recurrent/metastatic cervical cancer after platinum"},{"region":"China","year":2024,"indication":"First-line persistent/recurrent/metastatic cervical cancer with chemotherapy ± bevacizumab; first-line gastric cancer with chemotherapy"}],"mechanismSteps":["Two arms bind PD-1 and CTLA-4 on the same exhausted T cell","Avidity concentrates the drug where both receptors are present","Both brakes are released at once","T cells kill tumour cells even when PD-L1 is low"],"dosing":{"route":"Intravenous","schedule":"10 mg/kg every 3 weeks with platinum-paclitaxel ± bevacizumab","monitoring":"Thyroid, liver, glucose, skin; immune-related adverse events"},"toxicity":[{"event":"Grade ≥3 treatment-related adverse events","grade3PlusPct":82,"note":"COMPASSION-16 cadonilimab arm (mostly chemotherapy-related haematologic)"},{"event":"Immune-related adverse events, any grade","anyGradePct":52}],"access":[],"regulatoryEvents":[{"date":"2022-06-29","type":"approval","region":"China","note":"First PD-1×CTLA-4 bispecific approved anywhere"},{"date":"2024-09","type":"approval","region":"China","note":"First-line cervical cancer (COMPASSION-16)","source":"https://www.onclive.com/view/china-s-nmpa-approves-cadonilimab-plus-chemo-with-without-bevacizumab-in-cervical-cancer"}]},{"id":"calderasib","kind":"drug","name":"Calderasib","aka":[],"tldr":"Calderasib is an experimental small-molecule drug from Merck Sharp & Dohme in phase 3 trials for non-small-cell lung cancer and colorectal cancer, aimed at KRAS.","summary":"Calderasib (MK-1084) is a small-molecule drug developed by Merck Sharp & Dohme. Its target is KRAS (the sponsor names KRAS G12C). The sponsor states: MK-1084 is described as an investigational, oral, selective KRAS G12C inhibitor. It is the investigational product in 6 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07554339 (A Clinical Trial of Calderasib (MK-1084) and Durvalumab in People With Non-Small Cell Lung Cancer (MK-1084-015/KANDLELIT-015)), NCT06345729 (A Study of Calderasib (MK-1084) Plus Pembrolizumab (MK-3475) in Participants With KRAS G12C Mutant Non-small Cell Lung Cancer (NSCLC) With Programmed Cell Death Ligand 1 (PD-L1) Tumour Proportion Score (TPS) ≥50% (MK-1084-004/KANDLELIT-004)) and NCT07431827 (MK-3475A±Calderasib (MK-1084) in Completely Resected Stage IIA-IIIB (N2) KRAS G12Cm NSCLC (MK-1084-013)), in non-small-cell lung cancer and colorectal cancer. The largest, NCT07190248, plans to enrol 675 participants with primary completion expected 2029-12-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Calderasib","url":"https://clinicaltrials.gov/search?intr=MK-1084"},{"label":"Sponsor pipeline page","url":"https://www.merck.com/research/product-pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","colorectal"],"sections":[],"technologies":[],"targets":["kras"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07554339","nct06345729","nct07431827","nct06997497","nct07190248","nct07252739","nct07286149","nct07209111"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"MK-1084","modality":"small molecule","mechanism":"MK-1084 is described as an investigational, oral, selective KRAS G12C inhibitor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cam2029","kind":"drug","name":"CAM2029 (octreotide subcutaneous depot)","aka":[],"tldr":"CAM2029 is Camurus's monthly octreotide depot that patients can inject themselves, positive in the phase 3 SORENTO trial for gastroenteropancreatic neuroendocrine tumours against the standard depot formulations.","summary":"Camurus of Lund built CAM2029 on its FluidCrystal lipid depot so that octreotide can be given as a small subcutaneous self-injection rather than the intramuscular Sandostatin LAR. The phase 3 SORENTO trial in advanced gastroenteropancreatic neuroendocrine tumours met its primary endpoint of progression-free survival against octreotide LAR or lanreotide, and the company has filed in Europe and the United States; it is approved for acromegaly as Oczyesa in some markets.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of CAM2029 (octreotide subcutaneous depot)","url":"https://clinicaltrials.gov/search?intr=CAM2029%20(octreotide%20subcutaneous%20depot)"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["neuroendocrine"],"sections":[],"technologies":["endocrine-therapy"],"targets":["sstr2"],"drugs":[],"companies":["camurus"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05050942"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"modality":"Peptide somatostatin analogue (octreotide) in a long-acting subcutaneous depot","mechanism":"Octreotide, a somatostatin analogue, released for a month from a lipid depot that forms under the skin after a small ready-to-use injection.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"camizestrant","kind":"drug","name":"Camizestrant","aka":[],"tldr":"Camizestrant is an oral oestrogen-receptor degrader approved in September 2026 for a new kind of decision: switching treatment when a blood test shows resistance developing, before the cancer visibly grows.","summary":"Camizestrant is AstraZeneca's next-generation oral SERD. SERENA-6 randomised patients whose ctDNA acquired an ESR1 mutation during first-line AI + CDK4/6 to switch to camizestrant + CDK4/6: PFS 16.0 vs 9.2 months (HR 0.44). After a 6-3 negative ODAC vote (April 2026) over the clinical meaning of acting on ctDNA, FDA granted accelerated approval on 4 September 2026, the first indication triggered by a molecular rather than radiographic event. SERENA-4 (first-line, unselected) and the adjuvant CAMBRIA trials will define its wider role.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Camizestrant"}],"tags":[],"related":["oral-serd-plus-cdk46-after-esr1"],"cancers":["breast-hr-positive","hr-positive-metastatic-post-cdk46"],"sections":[],"technologies":["endocrine-therapy","liquid-biopsy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":["esr1-mutation","oral-serd"],"trials":["serena-6","cambria","nct04644068","nct05774951","nct07427394","nct04711252","nct04214288","nct06380751","nct07647328"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Etcamah","code":"AZD9833","modality":"Oral SERD","mechanism":"Oral SERD: ER antagonist and degrader active against wild-type and ESR1-mutant receptors.","approvals":[{"region":"US","year":2026,"indication":"HR+/HER2- advanced breast cancer with an emergent ESR1 mutation on AI + CDK4/6, with a CDK4/6 inhibitor (accelerated, 4 Sep 2026)"},{"region":"EU","year":2026,"indication":"ER+/HER2- advanced breast cancer with emergent ESR1 mutation, with a CDK4/6 inhibitor; 20 Jul 2026"}],"mechanismSteps":["Serial ctDNA detects an ESR1 mutation while scans still show control","The mutant receptor no longer needs oestrogen, so the aromatase inhibitor stops working","Camizestrant binds and degrades both mutant and wild-type ER","CDK4/6 inhibition is continued","Progression is delayed by about seven months"],"dosing":{"route":"Oral","schedule":"75 mg once daily with a CDK4/6 inhibitor (per SERENA-6)","monitoring":"Bradycardia and visual effects (photopsia) reported with the class"},"toxicity":[{"event":"Photopsia (visual flashes)","anyGradePct":20,"note":"class effect, reversible"},{"event":"Bradycardia","anyGradePct":15},{"event":"Neutropenia (with CDK4/6)","grade3PlusPct":45,"note":"largely from the CDK4/6 partner"}],"access":[],"regulatoryEvents":[{"date":"2025-06-01","type":"filing","region":"US","note":"NDA based on SERENA-6"},{"date":"2026-04-30","type":"advisory-committee","region":"US","note":"ODAC voted 6-3 against (clinical relevance of ctDNA-triggered switch)","source":"https://www.cancernetwork.com/view/fda-odac-no-camizestrant-for-hr-her2-esr1-advanced-breast-cancer"},{"date":"2026-09-04","type":"accelerated-approval","region":"US","note":"Accelerated approval (Etcamah) The confirmatory requirement was still open 0.0 years later, when the FDA's table was read.","source":"https://www.fda.gov/news-events/press-announcements/fda-grants-accelerated-approval-new-breast-cancer-treatment","indication":"In combination with a CDK4/6 inhibitor (abemaciclib, palbociclib, or ribociclib) for the treatment of adult patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer upon detection of ESR1 mutation during aromatase inhibitor and CDK4/6 inhibitor therapy, based on an FDA-authorized test"}]},{"id":"camonsertib","kind":"drug","name":"Camonsertib","aka":[],"tldr":"Camonsertib is an oral serine/threonine kinase inhibitor from Hoffmann-La Roche, in registered phase 2 trials for metastatic cancer.","summary":"Camonsertib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Hoffmann-La Roche, in metastatic cancer. A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Camonsertib","url":"https://clinicaltrials.gov/search?intr=Camonsertib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["flatiron-health"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04589845"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral serine/threonine kinase inhibitor","mechanism":"A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"camrelizumab","kind":"drug","name":"Camrelizumab","aka":[],"tldr":"Camrelizumab is Jiangsu Hengrui's humanised PD-1 antibody, approved in China for oesophageal, liver and lung cancer but not in the US, where its liver cancer combination with rivoceranib drew complete response letters in 2024 and 2025 over manufacturing and inspection issues. Its signature side effect is reactive cutaneous capillary endothelial proliferation, a skin reaction seen in most patients.","summary":"Humanised IgG4 anti-PD-1 (Jiangsu Hengrui). ESCORT (second-line ESCC) and ESCORT-1st (first-line with chemotherapy, OS 15.3 vs 12.0 months) in China; ESCORT-NEO neoadjuvant; CARES-310 with rivoceranib in HCC (OS 22.1 vs 15.2 months). A US BLA for the HCC combination received complete response letters (2024, 2025) over manufacturing and travel-inspection issues. Distinctive toxicity: reactive cutaneous capillary endothelial proliferation (RCCEP) in most patients.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Camrelizumab","links":[{"label":"ESCORT-1st final analysis","url":"https://pubmed.ncbi.nlm.nih.gov/38870932/"}],"tags":[],"related":[],"cancers":["esophageal","hcc","nsclc","nasopharyngeal","recurrent-metastatic-nasopharyngeal-carcinoma"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":["irae"],"trials":["escort-1st","captain-1st","nct04639180","nct05320692","nct05841472","nct06361888","nct04906993","nct04342910"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"AiRuiKa","code":"SHR-1210","modality":"Monoclonal antibody (anti-PD-1)","mechanism":"PD-1 blockade.","approvals":[{"region":"China","year":2019,"indication":"Classical Hodgkin lymphoma; subsequently HCC, NSCLC, ESCC (second line 2020, first line with chemotherapy 2021), nasopharyngeal"}],"mechanismSteps":["Camrelizumab binds PD-1 on T cells","Blocks the PD-L1 'stop' signal from the tumour","T cells regain the ability to kill","A quirk of its binding causes small skin blood-vessel growths (RCCEP) in most patients"],"dosing":{"route":"Intravenous","schedule":"200 mg every 2-3 weeks"},"toxicity":[{"event":"Reactive cutaneous capillary endothelial proliferation (RCCEP)","note":"Very common; benign, resolves after stopping"},{"event":"Immune-related adverse events"}],"access":[],"regulatoryEvents":[{"date":"2024-05","type":"crl","region":"US","note":"Complete response letter for camrelizumab + rivoceranib in HCC (manufacturing/inspection)"},{"date":"2025-03","type":"crl","region":"US","note":"Second complete response letter"}]},{"id":"camrelizumab-rivoceranib","kind":"drug","name":"Camrelizumab + rivoceranib","aka":[],"tldr":"A Chinese immunotherapy-plus-anti-angiogenic pill combination that clearly beat sorafenib in liver cancer, yet remains unapproved in the US after three manufacturing-related rejections.","summary":"CARES-310: median OS 23.8 vs 15.2 months (HR 0.62) and PFS 5.6 vs 3.7 months versus sorafenib in first-line unresectable HCC; approved in China (2023). FDA complete response letters in May 2024, March 2025 and July 2026, each on cGMP inspection deficiencies at the rivoceranib manufacturing site, not on clinical data. Rivoceranib (apatinib) is a selective VEGFR2 TKI; camrelizumab is a PD-1 antibody notable for reactive cutaneous capillary endothelial proliferation, which the TKI suppresses.","status":"approved","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT03764293 (CARES-310)","url":"https://clinicaltrials.gov/study/NCT03764293"}],"tags":[],"related":[],"cancers":["hcc"],"sections":[],"technologies":["checkpoint-inhibitor","antiangiogenic"],"targets":["pd1","vegf"],"drugs":[],"companies":["hengrui","elevar-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["cares-310"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"SHR-1210 + apatinib","modality":"PD-1 antibody + oral VEGFR2 inhibitor","mechanism":"PD-1 blockade plus VEGFR2 inhibition (vascular normalisation and reduced immunosuppression).","approvals":[{"region":"China","year":2023,"indication":"First-line unresectable HCC"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2024-05","type":"crl","region":"US","note":"First CRL: manufacturing inspection findings"},{"date":"2025-03","type":"crl","region":"US","note":"Second CRL"},{"date":"2026-01-30","type":"filing","region":"US","note":"Resubmission accepted; PDUFA 23 July 2026","source":"https://www.globenewswire.com/news-release/2026/01/30/3229623/0/en/elevar-therapeutics-announces-fda-acceptance-of-new-drug-application-resubmission-for-rivoceranib-in-combination-with-camrelizumab-as-a-first-line-systemic-treatment-for-unresectab.html"},{"date":"2026-07-23","type":"crl","region":"US","note":"Third CRL, again cGMP","source":"https://www.targetedonc.com/view/fda-passes-again-on-approving-rivoceranib-plus-camrelizumab-in-hcc"}]},{"id":"cancerguard","kind":"drug","name":"Cancerguard","aka":[],"tldr":"Exact Sciences' multi-cancer blood test, sold since 2025 as a lab test that has not been through FDA approval.","summary":"Cancerguard launched in September 2025 as a laboratory-developed test priced for self-pay, built on the multi-biomarker approach validated in the Falcon Registry and DETECT-A studies (the latter, with Johns Hopkins, was the first prospective interventional study of a multi-cancer blood test). Unlike Galleri it does not predict where a cancer is, so a positive result triggers whole-body imaging. As an LDT it is regulated through CLIA rather than the FDA, a status confirmed when the FDA's 2024 LDT rule was vacated in 2025. No randomised trial has yet shown that a multi-cancer blood test reduces deaths.","status":"emerging","asOf":"2026-09-10","links":[{"label":"Exact Sciences: Cancerguard","url":"https://www.exactsciences.com/"}],"tags":["test"],"related":["galleri","shield"],"cancers":[],"sections":[],"technologies":["mced","liquid-biopsy","methylation-profiling"],"targets":[],"drugs":[],"companies":["exact-sciences"],"institutions":[],"pathways":[],"terms":["ppv","screening","fda-ldt-rule"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: a positive signal starts a diagnostic work-up with scans; most people with a positive result will not turn out to have cancer, and a negative result does not replace mammograms, colonoscopy or other proven screening."],"brand":"Cancerguard","modality":"Multi-cancer early detection blood test (laboratory-developed)","mechanism":"Plasma assay combining cell-free DNA methylation and protein biomarkers across multiple classes, reporting a cancer signal without a tissue-of-origin call, followed by imaging work-up.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"capecitabine","kind":"drug","name":"Capecitabine","aka":[],"tldr":"Capecitabine (Xeloda) is a tablet form of the chemotherapy fluorouracil. It is a backbone of treatment for bowel cancer and a standard option in advanced breast cancer.","summary":"Capecitabine was approved by the FDA in 1998 for anthracycline- and taxane-resistant metastatic breast cancer and later for adjuvant stage III colon cancer (X-ACT: disease-free survival at least equivalent to bolus fluorouracil and leucovorin), metastatic colorectal cancer, perioperative chemoradiotherapy for rectal cancer and, with docetaxel, for anthracycline-pretreated breast cancer. The EU authorised Xeloda in 2001; it is also approved there for gastric cancer. It is the oral partner in CAPOX and CAPTEM and in the CREATE-X post-neoadjuvant breast regimen. Hand-foot syndrome, diarrhoea and cardiotoxicity are the key toxicities, and DPD deficiency (DPYD variants) causes severe or fatal toxicity, so genotype-guided dosing is increasingly used. Warfarin interaction carries a boxed warning.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Capecitabine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=capecitabine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/capecitabine"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/xeloda"},{"label":"Palmer et al., ESPAC4 long-term outcomes (JCO 2025)","url":"https://doi.org/10.1200/JCO.24.01118"},{"label":"NICE NG85 recommendations 1.8.6 and 1.9.3","url":"https://www.nice.org.uk/guidance/ng85/chapter/Recommendations"}],"tags":["nci-list","generic"],"related":["fluorouracil","capox","capecitabine-temozolomide","uridine-triacetate"],"cancers":["colorectal","breast-hr-positive","tnbc","breast-her2-positive","gastric","pancreatic","her2-positive-breast-brain-metastases","tnbc-early"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["rapido","nct04854668","nct06435429","nct07412613","nct06118333","nct04430738","nct07805551","nct05009069","nct07432295","nct05126719","nct06324357","nct05382442","nct02955940","nct05093907","nct06493760","nct05489211","nct06081959","nct06393374","nct05300269","nct07554521","nct05980481","nct06202261","nct03899155","nct05702229","nct06469944","nct06155383","nct05814354","nct06901531","nct07331155","nct06942234","nct05785741","nct06247956","nct06312176","nct06764875","nct05840211","nct06343948","nct06771622","nct06568692","nct05700084","nct07060807","nct07056777","nct05934331","nct06109779","nct05144854","nct07007559","nct06764940","nct07582315","nct07606599","nct07431281","nct04895358","nct06957886","nct07315750","nct04379596","nct07069712","nct06247995","nct07679360","nct06279364","spotlight-glow","nct04721977","nct07390383","nct06313983","nct06825494","nct07283367","nct07140393","nct06703177","nct04550260","nct07648914","nct07071337","nct06741644","nct04639986","nct05671822","nct06256328","nct04919226","nct04725994","create-x","sysucc-001","geicam-ciboma","ea1131","bre12-158","ascent-05","tropion-breast03","keynote-119","espac-4","espac-5","scalop","scalop-2","lap07"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: capecitabine is the post-neoadjuvant drug for residual disease without a germline BRCA mutation on CREATE-X (triple-negative subgroup: five-year disease-free survival 69.8 versus 56.1 percent, hazard ratio 0.58; overall survival 78.8 versus 70.3 percent, hazard ratio 0.52), and one year of low-dose metronomic capecitabine after standard adjuvant chemotherapy improved disease-free survival in SYSUCC-001 (five-year 82.8 versus 73.0 percent; ten-year 78.1 versus 66.6 percent) without a significant overall survival gain, while eight cycles of extended adjuvant capecitabine did not reach significance in the Spanish GEICAM/CIBOMA trial (hazard ratio 0.82). In EA1131 platinum was not better than capecitabine for residual basal-like disease. It is the physician's-choice comparator or control in ASCENT, ASCENT-05, TROPION-Breast03, KEYNOTE-119 and OlympiAD. NICE has never appraised it for this use; it is a generic prescribed on the CREATE-X evidence.","Pancreatic cancer: with gemcitabine, capecitabine is the adjuvant regimen NICE NG85 recommends in England (1.8.6, an off-label use) on ESPAC-4 (median overall survival 28.0 against 25.5 months, hazard ratio 0.82; long-term 31.6 against 28.4 months and 49.9 against 32.2 months after R0 resection, JCO 2025), the standard for people not eligible for modified FOLFIRINOX. It is the preferred radiosensitiser for chemoradiotherapy (SCALOP: median survival 15.2 against 13.4 months with gemcitabine-based chemoradiotherapy; NG85 1.9.3) and the chemoradiotherapy partner in LAP07 and ESPAC-5."],"brand":"Xeloda","modality":"Oral fluoropyrimidine prodrug (antimetabolite)","mechanism":"Converted in three steps, the last by thymidine phosphorylase enriched in tumours, to fluorouracil; FdUMP blocks thymidylate synthase and FUTP is incorporated into RNA.","approvals":[{"region":"US","year":1998,"indication":"Metastatic breast cancer after anthracycline and taxane; later adjuvant and metastatic colorectal cancer, rectal chemoradiotherapy"},{"region":"EU","year":2001,"indication":"Colorectal, gastric and breast cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1998-04-30","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Metastatic breast cancer resistant to paclitaxel and an anthracycline-containing chemotherapy regimen or resistant to paclitaxel and further anthracycline therapy is contraindicated"},{"date":"2001-09-07","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1998 converted to traditional approval 3.4 years after it was granted.","indication":"Metastatic breast cancer resistant to paclitaxel and an anthracycline-containing chemotherapy regimen or resistant to paclitaxel and further anthracycline therapy is contraindicated"}]},{"id":"capecitabine-temozolomide","kind":"drug","name":"Capecitabine + temozolomide (CAPTEM)","aka":[],"tldr":"CAPTEM (capecitabine plus temozolomide) is an all-oral chemotherapy pair that shrinks pancreatic neuroendocrine tumours in about a third of patients.","summary":"ECOG-ACRIN E2211 (2018-2023): PFS 22.7 vs 14.4 months and higher response rate for CAPTEM versus temozolomide alone in pancreatic NETs; OS did not differ at final analysis. Used when tumour shrinkage is needed (bulky or symptomatic disease) and in grade 3 NETs. MGMT deficiency may predict response.","status":"established","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT01824875: ECOG-ACRIN E2211, temozolomide with or without capecitabine in advanced pancreatic neuroendocrine tumours (phase 2)","url":"https://clinicaltrials.gov/study/NCT01824875"}],"tags":[],"related":[],"cancers":["neuroendocrine","pancreatic-net","grade-3-net"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":["temozolomide"],"companies":["ecog-acrin"],"institutions":[],"pathways":[],"terms":["mgmt"],"trials":["nct04919226"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Oral cytotoxic regimen","mechanism":"Fluoropyrimidine plus alkylating agent; capecitabine depletes MGMT, sensitising to temozolomide.","approvals":[],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"Capecitabine 750 mg/m² twice daily days 1-14, temozolomide 200 mg/m² days 10-14, every 28 days","monitoring":"Blood counts, hand-foot syndrome"},"toxicity":[{"event":"Neutropenia (grade 3-4)","grade3PlusPct":13,"note":"E2211"},{"event":"Thrombocytopenia (grade 3-4)","grade3PlusPct":15},{"event":"Fatigue","anyGradePct":50}],"access":[],"regulatoryEvents":[]},{"id":"capivasertib","kind":"drug","name":"Capivasertib","aka":[],"tldr":"Capivasertib (Truqap) is the first AKT inhibitor, for breast cancer with PI3K-pathway mutations and, since 2026, for prostate cancer with PTEN loss.","summary":"Capivasertib is an ATP-competitive pan-AKT inhibitor given intermittently at 400 mg twice daily for 4 days on and 3 days off, a schedule chosen to limit hyperglycaemia and rash. It was approved in 2023 with fulvestrant for HR-positive, HER2-negative advanced breast cancer carrying PIK3CA, AKT1 or PTEN alterations after CAPItello-291, and in Q2 2026 with abiraterone for PTEN-deficient metastatic hormone-sensitive prostate cancer after CAPItello-281, the first AKT inhibitor in either disease. AstraZeneca markets it. Diarrhoea (77%, 12% grade 3 or higher) and cutaneous reactions (56%, 15%) are the main toxicities. The breast label is limited to tumours with a pathway alteration, and sequencing against PI3K-alpha inhibitors such as inavolisib is unresolved. For a newcomer: a pill that blocks the AKT node of the PI3K pathway in tumours that have switched it on.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Capivasertib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Capivasertib"},{"label":"CAPItello-290 (Annals of Oncology 2026)","url":"https://doi.org/10.1016/j.annonc.2025.12.012"}],"tags":[],"related":["er-status","pik3ca-hotspot-mutation","akt1-e17k","pten-alteration"],"cancers":["breast-hr-positive","prostate","prostate-mhspc","hr-positive-metastatic-post-cdk46","tnbc","tnbc-metastatic"],"sections":[],"technologies":["kinase-inhibitors","pi3k-akt-mtor-inhibitors"],"targets":["akt","pik3ca"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05348577","nct07287917","nct06635447","nct05563220","nct04862663","nct03742102","nct06764186","nct03997123"],"people":[],"bottlenecks":[],"keyPapers":["paper-schmid-pakt-capivasertib-tnbc-jco-2020"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: CAPItello-290 (812 patients, first-line paclitaxel with or without capivasertib) missed both overall survival endpoints (17.7 versus 18.0 months overall, hazard ratio 0.92; 20.4 months in both arms in PIK3CA, AKT1 or PTEN-altered tumours, hazard ratio 1.05) despite a progression-free survival signal (5.6 versus 5.1 months, hazard ratio 0.72), so the AKT inhibitor has no triple-negative indication and the NICE appraisal for untreated metastatic triple-negative disease (GID-TA11411) was discontinued. With IPATunity130 (ipatasertib) this closes the first-line AKT-inhibitor programme in triple-negative disease that the phase 2 PAKT and LOTUS trials had opened."],"brand":"Truqap","modality":"Small-molecule AKT inhibitor","mechanism":"ATP-competitive pan-AKT inhibitor, intermittent 4-days-on/3-off dosing.","approvals":[{"region":"US","year":2023,"indication":"HR+/HER2- breast cancer with PIK3CA/AKT1/PTEN alteration, with fulvestrant"},{"region":"US","year":2026,"indication":"PTEN-deficient metastatic prostate cancer with abiraterone"},{"region":"EU","year":2024,"indication":"ER+/HER2- mBC with PIK3CA/AKT1/PTEN alteration; 17 Jun 2024"}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of AKT1/2/3","Phosphorylation of downstream substrates stops","Intermittent dosing limits hyperglycaemia while PI3K-pathway signalling is suppressed","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"400 mg twice daily for 4 days on, 3 days off, with fulvestrant (breast) or abiraterone plus prednisone (prostate)","modifications":"Reduce to 320 then 240 mg for grade 3 diarrhoea, rash, or hyperglycaemia; hold for fasting glucose >250 mg/dL","monitoring":"Fasting glucose and HbA1c at baseline and periodically; skin; loperamide guidance","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d698c106-2322-401e-b738-cbd83c843ecf"},"toxicity":[{"event":"Diarrhoea","anyGradePct":77,"grade3PlusPct":12,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d698c106-2322-401e-b738-cbd83c843ecf","note":"CAPItello-291"},{"event":"Cutaneous adverse reactions","anyGradePct":56,"grade3PlusPct":15,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d698c106-2322-401e-b738-cbd83c843ecf","note":"CAPItello-291"},{"event":"Fatigue","anyGradePct":38,"grade3PlusPct":1.9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d698c106-2322-401e-b738-cbd83c843ecf","note":"CAPItello-291"},{"event":"Nausea","anyGradePct":35,"grade3PlusPct":1.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d698c106-2322-401e-b738-cbd83c843ecf","note":"CAPItello-291"},{"event":"Stomatitis","anyGradePct":25,"grade3PlusPct":1.9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d698c106-2322-401e-b738-cbd83c843ecf","note":"CAPItello-291"},{"event":"Vomiting","anyGradePct":21,"grade3PlusPct":1.9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d698c106-2322-401e-b738-cbd83c843ecf","note":"CAPItello-291"},{"event":"Hyperglycaemia","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d698c106-2322-401e-b738-cbd83c843ecf","note":"Warning: DKA reported"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.astrazeneca-us.com/medicines/access-360","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with fulvestrant for PIK3CA/AKT1/PTEN-altered HR+ breast cancer (TA998)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-11-16","type":"approval","region":"US","note":"HR+/HER2- breast cancer with PIK3CA/AKT1/PTEN alteration, with fulvestrant (CAPItello-291): first AKT inhibitor","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-06-12","type":"approval","region":"US","note":"PTEN-deficient metastatic prostate cancer with abiraterone (CAPItello-281)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-capivasertib-abiraterone-and-prednisone-pten-deficient-androgen-pathway-modulation"}]},{"id":"capmatinib","kind":"drug","name":"Capmatinib","aka":["INC280"],"tldr":"Capmatinib is a targeted pill for the small group of lung cancers driven by a MET exon 14 skipping mutation, found by tumour sequencing.","summary":"Capmatinib is an oral MET inhibitor for metastatic non-small cell lung cancer whose tumours carry a MET exon 14 skipping mutation, present in roughly 3 to 4 percent of lung adenocarcinomas. In the GEOMETRY mono-1 trial it produced responses in most untreated patients and in a smaller share of previously treated ones. The FDA granted accelerated approval in 2020. NICE did not complete its appraisal, so it is not routinely funded in England. Peripheral oedema and nausea are the common side effects.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Capmatinib","links":[{"label":"GEOMETRY mono-1 (NEJM 2020)","url":"https://doi.org/10.1056/NEJMoa2002787"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=capmatinib"}],"tags":["gap-fill"],"related":["met-ex14"],"cancers":["nsclc","met-altered-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["met"],"drugs":["tepotinib","crizotinib"],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["geometry-mono-1","nct03040973","nct05488314"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tabrecta","modality":"Small-molecule MET tyrosine kinase inhibitor","mechanism":"A selective, ATP-competitive inhibitor of the MET receptor kinase; tumours with MET exon 14 skipping mutations keep the receptor switched on, and blocking it shuts down the growth signal.","approvals":[{"region":"US","year":2020,"indication":"Metastatic NSCLC with a MET exon 14 skipping mutation (accelerated approval)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2020-05-06","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adult patients with mNSCLC whose tumors have a mutation that leads to MET exon 14 skipping as detected by an FDA-approved test"},{"date":"2022-08-10","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 2.3 years after it was granted.","indication":"Adult patients with mNSCLC whose tumors have a mutation that leads to MET exon 14 skipping as detected by an FDA-approved test"}]},{"id":"capmatinib-tepotinib","kind":"drug","name":"Capmatinib & tepotinib","aka":[],"tldr":"Capmatinib and tepotinib are two pills for the roughly 3% of lung cancers with a MET exon 14 skipping mutation.","summary":"Capmatinib and tepotinib are type Ib MET inhibitors that bind only the active conformation of the MET kinase, sparing other kinases. They treat the roughly 3% of non-small-cell lung cancers driven by a MET exon 14 skipping mutation, in both untreated and previously treated patients. Capmatinib (GEOMETRY mono-1: ORR 68% treatment-naive) was approved in 2020 with full approval in 2022; tepotinib (VISION: ORR ~57%) was approved in 2021 with full approval in 2024. Peripheral oedema is the class toxicity and often needs dose adjustment. MET amplification as an EGFR-TKI escape route is a different problem and is instead addressed by amivantamab and MET ADCs. The simple version: test lung adenocarcinomas for MET exon 14 skipping, because two pills work well when it is present.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Capmatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Capmatinib%20%26%20tepotinib"}],"tags":[],"related":[],"cancers":["nsclc","met-altered-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["met"],"drugs":[],"companies":["novartis","merck"],"institutions":[],"pathways":[],"terms":[],"trials":["geometry-mono-1","nct02864992"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tabrecta / Tepmetko","modality":"Small-molecule kinase inhibitors (MET)","mechanism":"Highly selective type Ib MET inhibitors.","approvals":[{"region":"US","year":2020,"indication":"MET exon 14 skipping NSCLC (capmatinib; tepotinib 2021)"},{"region":"EU","year":2022,"indication":"Tepmetko 2022; Tabrecta 2022"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"capox","kind":"drug","name":"CAPOX (capecitabine, oxaliplatin)","aka":[],"tldr":"CAPOX pairs the oral fluoropyrimidine capecitabine with intravenous oxaliplatin as a pill-based alternative to FOLFOX. For low-risk stage III colon cancer three months after surgery works as well as six and halves nerve damage; it is also standard in gastric cancer, with hand-foot syndrome as its price.","summary":"IDEA collaboration (2018): 3 months of CAPOX is non-inferior to 6 for low-risk stage III (T1-3 N1) colon cancer, halving neuropathy. Standard perioperative and first-line option in gastric cancer (with nivolumab in CheckMate 649; with zolbetuximab in GLOW). Hand-foot syndrome from capecitabine replaces some 5-FU toxicity.","status":"standard-of-care","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/XELOX","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/XELOX"}],"tags":[],"related":["folfox"],"cancers":["colorectal","gastric","appendiceal-adenocarcinoma","goblet-cell-adenocarcinoma","localised-small-bowel-adenocarcinoma","advanced-small-bowel-adenocarcinoma"],"sections":[],"technologies":["cytotoxic-chemotherapy","platinum"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["circulate-japan","nct04854668","nct04384484","nct07221357","nct07228832","nct07412613","nct06508658","nct07621718","nct07462143","nct07676162","nct07790510","nct05482893","nct04430738","nct07805551","nct05846867","nct06820463","nct06948448","nct07124936","nct05919264","nct04421820","nct07432295","nct04844866","nct07474727","nct05632939","nct06840002","nct05382442","nct05379595","nct06632262","nct06493760","nct05702229","nct04725994","idea-collaboration","scot","tosca","stellar-rectal","cairo3"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"XELOX","modality":"Cytotoxic regimen","mechanism":"Capecitabine is an oral prodrug of 5-FU activated preferentially in tumour; oxaliplatin crosslinks DNA.","approvals":[],"mechanismSteps":[],"dosing":{"route":"Oral + intravenous","schedule":"Capecitabine 1,000 mg/m² twice daily days 1-14 + oxaliplatin 130 mg/m² day 1, every 21 days","modifications":"Reduce capecitabine for hand-foot syndrome or renal impairment","monitoring":"Blood counts, creatinine, neuropathy"},"toxicity":[{"event":"Hand-foot syndrome"},{"event":"Peripheral neuropathy"},{"event":"Diarrhoea"}],"access":[],"regulatoryEvents":[]},{"id":"carboplatin","kind":"drug","name":"Carboplatin","aka":[],"tldr":"Carboplatin is a platinum chemotherapy that crosslinks DNA; it is part of the standard pre-surgery regimen for triple-negative breast cancer.","summary":"Carboplatin is a platinum chemotherapy that forms intrastrand DNA crosslinks, blocking replication and triggering apoptosis in dividing cells. First approved in 1989 for ovarian cancer and generic since 2004, it is dosed by the Calvert formula to a target AUC of 5 to 6 every 3 weeks or AUC 1.5 to 2 weekly with paclitaxel. It causes less kidney damage than cisplatin but more myelosuppression, with thrombocytopenia dose-limiting and hypersensitivity after 6 or more cycles. In triple-negative breast cancer, adding carboplatin to neoadjuvant chemotherapy raised pathological complete response rates in BrighTNess and GeparSixto, and it is part of the KEYNOTE-522 backbone, where pembrolizumab plus chemotherapy achieved pCR of 64.8% versus 51.2%. For a newcomer: a workhorse chemotherapy that damages DNA and underpins many curative-intent regimens.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Carboplatin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Carboplatin"}],"tags":[],"related":[],"cancers":["tnbc","ovarian","nsclc","sclc","seminoma","cns-germ-cell-tumours","cup-favourable-subsets","metastatic-anal-cancer"],"sections":[],"technologies":["platinum","cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["keynote-522","gefitinib-chemo-tmh","nct06212752","nct04351555","nct06712355","nct07625644","nct07502300","nct06305754","nct06692738","nct04379635","nct07216703","nct05353257","nct06699212","nct05800015","nct06956001","nct06564844","nct06417814","nct07168200","nct07005128","nct06452277","nct03390686","nct05654454","nct05116462","nct06989112","nct03800134","nct06422143","nct06230224","nct06396065","nct04129502","nct06712316","nct06312137","nct06952504","nct05555732","nct05870319","nct03164616","nct07472517","nct06561386","nct05722015","nct06123884","nct07642024","nct06793215","nct06687369","nct05048797","nct06627647","nct04956692","nct07196774","nct05984277","nct06915025","nct04974398","nct03178552","nct07777822","nct07178795","nct06097728","nct07100080","nct05668988","nct07564141","nct06891833","nct04316364","nct05840016","nct05652686","nct07059221","nct06535607","nct06514027","nct01081951","nct06512051","nct07024784","nct06448754","nct04083599","nct06946797","nct05633667","nct04745689","nct07397338","nct06030258","nct07680764","nct06731907","nct05789082","nct05605535","nct05904379","nct05739981","nct06522828","nct03775486","nct06943820","nct07296809","nct05797168","nct07714668","nct05351788","nct07245446","nct05533775","nct07221474","nct07229729","nct05456685","nct05635708","nct07622186","nct06996782","nct06706076","nct06161441","nct05098132","nct03393884","nct06162221","nct04736173","nct06754930","nct06727565","nct04938583","nct05816252","nct06890338","nct04644068","nct05061550","nct07310784","nct06840002","nct07623356","nct06859775","nct07155174","nct07122687","nct07070440","nct07102381","actuate-1801","nct07020221","nct06449209","nct04665206","nct06362252","nct05841472","nct06843447","nct04675333","nct05247684","nct07174583","nct07322094","nct07227597","nct06758557","nct06047379","nct05186974","nct05403385","nct05489211","nct03944772","nct05742607","nct07059845","nct04956640","nct07486817","nct05609578","nct05229497","nct06623422","nct05498428","nct06246110","nct06667076","nct05302284","nct06474455","nct07276399","nct07108270","nct06227117","nct06317311","nct05224141","nct05771480","nct03547973","nct07129993","nct03682068","nct06783647","nct06008093","nct04538664","nct05687266","nct06151574","nct07586202","nct04025879","nct05261399","nct06119581","nct06966700","nct06449222","nct04665856","nct03036098","nct07492680","nct07268040","nct06797635","nct07171606","nct03485209","nct02516241","nct05902169","nct06385080","nct06667908","nct02453282","nct06385678","nct06194448","nct07109531","nct05456256","nct06772623","nct07173751","nct06592326","nct07448922","nct02542293","nct06112379","nct07544654","nct06682780","nct03003962","nct05173987","nct06875310","nct07466160","nct03967977","nct07256782","nct06279364","nct07028281","nct05911295","nct05410145","nct06225596","nct04274426","nct07393542","nct04988295","nct06970639","nct07730021","nct05566041","nct07241767","nct07019675","nct07287995","nct07106762","nct03473743","nct06741644","nct00248287","nct04380636","nct06841354","nct04634877","brightness","geparsixto","calgb-40603","tnt","neotrip","partner","ea1131","brocade3","neopact"],"people":[],"bottlenecks":[],"keyPapers":["paper-keynote-189-nejm-2018"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: carboplatin entered neoadjuvant regimens on GeparSixto (pathological complete response 53.2 versus 36.9 percent in the triple-negative cohort; disease-free survival hazard ratio 0.56), CALGB 40603 (higher pathological complete response, no long-term survival gain in a trial not powered for it) and BrighTNess (58 versus 31 percent; event-free survival hazard ratio 0.57 at 4.5 years), and is the platinum in the KEYNOTE-522 backbone. In metastatic disease the UK TNT trial found carboplatin no better than docetaxel overall (response 31.4 versus 34.0 percent) but twice as active in germline BRCA carriers (68 versus 33 percent)."],"modality":"Cytotoxic chemotherapy (platinum)","mechanism":"DNA intrastrand crosslinks.","approvals":[{"region":"US","year":1989,"indication":"Ovarian cancer (now broad use)"}],"mechanismSteps":["Carboplatin is aquated inside the cell to a reactive platinum species","Platinum binds guanine N7, forming intrastrand DNA crosslinks","Replication and transcription are blocked","HR-deficient tumour cells cannot repair the lesions","Apoptosis follows; marrow cells are the main collateral target"],"dosing":{"route":"IV infusion","schedule":"AUC 5-6 mg/mL·min every 3 weeks (Calvert formula) or AUC 1.5-2 weekly with paclitaxel (KEYNOTE-522 backbone)","modifications":"Reduce for thrombocytopenia; renal-function-based dosing","monitoring":"Blood counts, creatinine, hypersensitivity after multiple cycles","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Thrombocytopenia"},{"event":"Anaemia"},{"event":"Neutropenia"},{"event":"Nausea and vomiting"},{"event":"Hypersensitivity (after ≥6 cycles)"},{"event":"Peripheral neuropathy (with paclitaxel)"}],"access":[{"country":"US","reimbursement":"Medicare Part B; generic","generic":true,"source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NHS standard; generic","generic":true,"asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"1989-03-03","type":"approval","region":"US","note":"Ovarian cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2004","type":"approval","region":"US","note":"First generics","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"carfilzomib","kind":"drug","name":"Carfilzomib","aka":[],"tldr":"Carfilzomib is a second-generation proteasome inhibitor with less nerve damage but more heart and blood-pressure effects.","summary":"Carfilzomib is an epoxyketone that irreversibly binds the beta5 subunit of the proteasome, giving deeper inhibition than bortezomib with much less peripheral neuropathy. In ENDEAVOR, carfilzomib-dexamethasone (Kd) beat bortezomib-dexamethasone (Vd) with PFS 18.7 versus 9.4 months and an OS benefit, underpinning its 2015 approvals after the 2012 accelerated approval in relapsed disease. It is used in KRd and in Isa-Kd and Dara-Kd combinations, mainly in relapsed disease, because ENDURANCE showed no frontline advantage of KRd over VRd in standard-risk patients. The trade-off is cardiovascular: hypertension in 25%, cardiac failure in 5% and dyspnoea in 28% in ENDEAVOR, so blood pressure and cardiac function are monitored. Amgen develops it. It is a stronger proteasome inhibitor that spares the nerves but asks more of the heart.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Carfilzomib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Carfilzomib"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-relapsed-refractory"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":["ubiquitin-proteasome-system"],"terms":[],"trials":["nct05438043","nct05572515","nct06152575","nct06158841","nct05552976","nct03989414","nct06892522","nct05704049","nct07227311","nct02899052","nct04973605","nct02773030"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Kyprolis","modality":"Proteasome inhibitor (irreversible)","mechanism":"Epoxyketone that irreversibly binds the β5 proteasome subunit.","approvals":[{"region":"US","year":2012,"indication":"Relapsed myeloma (accelerated)"},{"region":"US","year":2015,"indication":"With lenalidomide-dexamethasone; with dexamethasone (ENDEAVOR)"}],"mechanismSteps":[],"dosing":{"route":"IV","schedule":"20/56 mg/m2 twice weekly or 20/70 mg/m2 weekly","monitoring":"Blood pressure, cardiac function, renal function, dyspnoea"},"toxicity":[{"event":"Hypertension","anyGradePct":25,"grade3PlusPct":9,"note":"ENDEAVOR"},{"event":"Cardiac failure","grade3PlusPct":5},{"event":"Dyspnoea","anyGradePct":28}],"access":[],"regulatoryEvents":[{"date":"2012-07-20","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Multiple myeloma after at least 2 prior therapies including bortezomib and an immunomodulatory agent and disease progression on or within 60 days of completion of the last therapy"},{"date":"2016-01-21","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2012 converted to traditional approval 3.5 years after it was granted.","indication":"Multiple myeloma after at least 2 prior therapies including bortezomib and an immunomodulatory agent and disease progression on or within 60 days of completion of the last therapy"}]},{"id":"carmustine","kind":"drug","name":"Carmustine","aka":["BCNU","Carmustine implant"],"tldr":"Carmustine is a chemotherapy that reaches the brain. It is given by infusion for brain tumours, lymphoma and myeloma, and as a slow-release wafer left in the cavity after a brain tumour is removed.","summary":"Intravenous carmustine (BiCNU, approved 1977) is indicated as palliative therapy, alone or in combination, for brain tumours (glioblastoma, brainstem glioma, medulloblastoma, astrocytoma, ependymoma and metastatic brain tumours), multiple myeloma with prednisone, and relapsed or refractory Hodgkin and non-Hodgkin lymphoma; it is also part of the BEAM conditioning regimen before autologous transplant in lymphoma. The Gliadel wafer (approved 1996 for recurrent glioblastoma, 2003 for newly diagnosed high-grade glioma) is a biodegradable polifeprosan 20 implant placed in the resection cavity; the pivotal 240-patient placebo-controlled trial in newly diagnosed high-grade glioma showed a modest survival benefit. Delayed cumulative myelosuppression and dose-related pulmonary fibrosis limit lifetime dosing.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Carmustine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=carmustine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/carmustine"},{"label":"NCI page (carmustine implant)","url":"https://www.cancer.gov/about-cancer/treatment/drugs/carmustineimplant"}],"tags":["nci-list","generic"],"related":[],"cancers":["glioblastoma","medulloblastoma","multiple-myeloma","hodgkin-lymphoma","dlbcl"],"sections":[],"technologies":["cytotoxic-chemotherapy","autologous-stem-cell-transplant"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06915246"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"BiCNU / Gliadel Wafer","modality":"Alkylating agent (nitrosourea); intravenous and implantable wafer","mechanism":"Alkylates and cross-links DNA and RNA and carbamoylates proteins; lipophilic, so it crosses the blood-brain barrier. The Gliadel wafer releases carmustine slowly into the resection cavity.","approvals":[{"region":"US","year":1977,"indication":"Brain tumours, multiple myeloma, relapsed Hodgkin and non-Hodgkin lymphoma (BiCNU)"},{"region":"US","year":1996,"indication":"Recurrent glioblastoma as an adjunct to surgery (Gliadel Wafer); newly diagnosed high-grade glioma with surgery and radiation added 2003"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"carotuximab","kind":"drug","name":"Carotuximab","aka":["TRC105"],"tldr":"Carotuximab was an antibody against endoglin, a protein on growing blood vessels and on angiosarcoma cells. Added to pazopanib in the phase 3 TAPPAS trial it did not help, and the trial was stopped in 2019.","summary":"Carotuximab (TRC105) was Tracon Pharmaceuticals' antibody against endoglin, a marker of angiogenic endothelium and of angiosarcoma, a cancer that arises from blood vessel lining. Endoglin signalling was thought to be one way tumours escape VEGF blockade, so the antibody was combined with VEGFR inhibitors. Early-phase results in angiosarcoma with pazopanib were encouraging.\n\nThe randomised phase 3 TAPPAS trial of pazopanib with or without carotuximab in advanced angiosarcoma was stopped in 2019 after an interim analysis showed no improvement in progression-free survival, and the programme ended. The angiosarcoma page lists it with bevacizumab added to paclitaxel (ANGIOTAX-PLUS) among the antiangiogenic additions that failed.","status":"negative","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["pazopanib"],"cancers":["angiosarcoma"],"sections":[],"technologies":["monoclonal-antibody"],"targets":[],"drugs":[],"companies":["tracon"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"TRC105","modality":"Chimeric anti-endoglin (CD105) monoclonal antibody","mechanism":"Binds endoglin (CD105), a TGF-beta co-receptor highly expressed on proliferating tumour endothelium and on angiosarcoma cells, to block a vessel-growth pathway that escapes VEGF inhibition.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cart84","kind":"drug","name":"CART84","aka":[],"tldr":"CART84 is an experimental CAR-T cell therapy from Gyala Therapeutics in phase 2 trials for acute myeloid leukaemia, aimed at CD3.","summary":"CART84 is a CAR-T cell therapy developed by Gyala Therapeutics. Its target is CD3 (the sponsor names CD84). The sponsor states: Autologous T cells transduced with a lentiviral vector expressing an anti-CD84 chimeric antigen receptor, to recognise and kill CD84-expressing leukaemia cells. ClinicalTrials.gov describes the intervention as: GYA01 (CART84): autologous cell-based product containing CD3+ T cells transduced with a lentiviral vector expressing an anti-CD84 chimeric antigen receptor (CAR). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in acute myeloid leukaemia. The largest, NCT07471789, plans to enrol 33 participants with primary completion expected 2029-05-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of CART84","url":"https://clinicaltrials.gov/search?intr=CART84"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["aml"],"sections":[],"technologies":[],"targets":["cd3"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07471789"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"Autologous T cells transduced with a lentiviral vector expressing an anti-CD84 chimeric antigen receptor, to recognise and kill CD84-expressing leukaemia cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"catumaxomab","kind":"drug","name":"Catumaxomab","aka":["Removab (2009 to 2017)"],"tldr":"Catumaxomab (Korjuny) is an antibody infused into the abdominal cavity to control malignant ascites, the build-up of fluid caused by cancers such as ovarian and stomach cancer, in people whose other treatments have stopped working.","summary":"Catumaxomab was the first bispecific antibody approved anywhere, authorised in the EU in 2009 as Removab for malignant ascites in EpCAM-positive carcinomas on a randomised trial against paracentesis alone in which puncture-free survival was quadrupled. Its marketing authorisation was withdrawn in 2017 for commercial reasons after the original company failed, and a new authorisation was granted to Korjuny in February 2025 for intraperitoneal treatment of malignant ascites in adults with EpCAM-positive carcinomas not eligible for further systemic anticancer therapy. It is given as four intraperitoneal infusions over about ten days. Cytokine release-type reactions (fever, nausea, vomiting, abdominal pain) are almost universal and hepatic enzyme rises are common; it has never been approved in the US.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Catumaxomab","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/korjuny"}],"tags":["ema-list","supportive"],"related":[],"cancers":["ovarian","gastric","pancreatic","colorectal"],"sections":[],"technologies":["bispecific-antibody","t-cell-engager"],"targets":["epcam","cd3"],"drugs":[],"companies":["lindis-biotech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00377429","nct00189345","nct00563836"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Korjuny","modality":"Trifunctional bispecific antibody (EpCAM x CD3, rat-mouse hybrid)","mechanism":"Binds EpCAM on tumour cells and CD3 on T cells while its Fc region engages Fc-gamma receptor-positive accessory cells, killing EpCAM-positive cells in the peritoneal cavity by T-cell cytotoxicity, phagocytosis and antibody-dependent mechanisms.","approvals":[{"region":"EU","year":2009,"indication":"Malignant ascites in EpCAM-positive carcinomas when standard therapy is unavailable (Removab; withdrawn 2017)"},{"region":"EU","year":2025,"indication":"Intraperitoneal treatment of malignant ascites in adults with EpCAM-positive carcinomas not eligible for further systemic therapy (Korjuny)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cd19-t-hank","kind":"drug","name":"CD19 t-haNK","aka":[],"tldr":"CD19 t-haNK is an experimental cell therapy whose type is not stated from ImmunityBio in phase 2 trials for hodgkin lymphoma, aimed at CD19.","summary":"CD19 t-haNK is a cell therapy whose type is not stated developed by ImmunityBio. Its target is CD19 (the sponsor names CD19). The sponsor states: CD19-targeted natural killer (haNK) cell therapy intended to bind CD19 on malignant B cells and drive immune-mediated cell death; studied in combination with the IL-15 superagonist N-803 (nogapendekin alfa inbakicept) and rituximab. ClinicalTrials.gov describes the intervention as: N-803 Subcutaneous (SQ): N-803 is a novel IL-15 superagonist immunotherapy administered subcutaneously. It is designed to enhance the proliferation and activation of natural killer (NK) cells and CD8+ T cells without stimulating regulatory. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in hodgkin lymphoma. The largest, NCT07125872, plans to enrol 20 participants with primary completion expected 2027-05-25. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of CD19 t-haNK","url":"https://clinicaltrials.gov/search?intr=CD19%20t-haNK"},{"label":"Sponsor pipeline page","url":"https://www.immunitybio.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":["cd19"],"drugs":[],"companies":["immunitybio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07125872"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"cell therapy (type not stated)","mechanism":"CD19-targeted natural killer (haNK) cell therapy intended to bind CD19 on malignant B cells and drive immune-mediated cell death; studied in combination with the IL-15 superagonist N-803 (nogapendekin alfa inbakicept) and rituximab.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cediranib","kind":"drug","name":"Cediranib","aka":["AZD2171","Recentin","Cediranib maleate","Zemfirza"],"tldr":"Cediranib is a tablet that blocks the blood-vessel growth signal VEGF. In alveolar soft part sarcoma, a rare very vascular sarcoma that ignores chemotherapy, a randomised trial showed it shrinks tumours and delays progression, although it was never licensed.","summary":"Cediranib (AZD2171) is AstraZeneca's oral inhibitor of VEGFR-1, -2 and -3. It was tested widely, including the ICON6 trial in relapsed ovarian cancer, but never gained approval; AstraZeneca withdrew its EU application (Zemfirza, relapsed ovarian cancer) on 19 September 2016 before a Commission decision. Its clearest activity is in alveolar soft part sarcoma: a National Cancer Institute phase 2 showed responses in a third of patients, and the randomised, placebo-controlled CASPS trial (Lancet Oncology, 2019) found a greater reduction in tumour size at 24 weeks and longer progression-free survival than placebo.\n\nCediranib remains an investigational drug available in some countries through trials or special access; other VEGFR inhibitors such as sunitinib, pazopanib and anlotinib, and checkpoint inhibitors such as atezolizumab, are used in practice. The alveolar soft part sarcoma page names it among the VEGFR-directed drugs that are active in the disease.","status":"phase-2","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Cediranib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Cediranib"}],"tags":["subtype-drugs-wave"],"related":["sunitinib","pazopanib","anlotinib","atezolizumab"],"cancers":["alveolar-soft-part-sarcoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["vegf"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00399035","nct00777153","nct00532194"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"AZD2171","modality":"Oral VEGFR 1-3 tyrosine kinase inhibitor","mechanism":"A potent inhibitor of all three VEGF receptors, with activity against PDGFR and KIT, that blocks tumour angiogenesis; alveolar soft part sarcoma is highly vascular and driven by the ASPSCR1-TFE3 fusion, which upregulates angiogenic genes.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"celecoxib","kind":"drug","name":"Celecoxib","aka":["SC-58635"],"tldr":"Celecoxib is a common anti-inflammatory painkiller. In oncology it is paired with low-dose oral methotrexate as a cheap 'metronomic' treatment for advanced head and neck cancer, a regimen from Tata Memorial in Mumbai that beat intravenous cisplatin in a randomised trial.","summary":"Celecoxib is a selective COX-2 inhibitor that Pfizer (then Searle and Pharmacia) launched for arthritis pain; the FDA approved it in 1998. It had an accelerated approval in 1999 for reducing polyps in familial adenomatous polyposis, which Pfizer withdrew in 2011 after the confirmatory study was not completed.\n\nIts oncology use today is as part of low-cost oral metronomic chemotherapy. At Tata Memorial Hospital, Patil and colleagues randomised patients with recurrent or metastatic head and neck cancer who could not afford or tolerate standard drugs to oral methotrexate 15 mg/m2 weekly with celecoxib 200 mg twice daily or to intravenous cisplatin every three weeks; the oral pair gave longer survival with fewer serious side effects, and adding one-twentieth of the usual dose of nivolumab improved survival further in a later trial. The regimen is named on the oral cavity, tongue, buccal mucosa and recurrent or metastatic head and neck cancer pages as the option where resources are limited.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Celecoxib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Celecoxib"}],"tags":["subtype-drugs-wave"],"related":["methotrexate","nivolumab"],"cancers":["oral-cavity-cancer","oral-tongue-cancer","buccal-mucosa-cancer","recurrent-metastatic-hnscc","hpv-negative-head-and-neck-cancer"],"sections":[],"technologies":[],"targets":["cox2"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Celebrex","modality":"Oral COX-2 inhibitor","mechanism":"Selectively inhibits cyclo-oxygenase-2, the inducible prostaglandin enzyme that tumours and their stroma use to drive inflammation, angiogenesis and immune suppression, while sparing COX-1 in the stomach and platelets.","approvals":[{"region":"US","year":1998,"indication":"Osteoarthritis and rheumatoid arthritis pain (oncology use in metronomic regimens is off-label)"},{"region":"US","year":1999,"indication":"Reduction of adenomatous colorectal polyps in familial adenomatous polyposis (accelerated approval; indication withdrawn 2011)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1999-12-23","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"To reduce the number of adenomatous colorectal polyps in familial adenomatous polyposis patients, as an adjunct to usual care"},{"date":"2012-06-08","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 12.5 years after its accelerated approval.","indication":"To reduce the number of adenomatous colorectal polyps in familial adenomatous polyposis patients, as an adjunct to usual care"}]},{"id":"cemiplimab","kind":"drug","name":"Cemiplimab","aka":[],"tldr":"A PD-1 blocker that is the standard for advanced skin squamous cell carcinoma, and in 2025 became the first adjuvant immunotherapy for it.","summary":"Cemiplimab is a fully human IgG4 antibody against PD-1 that restores the activity of exhausted tumour-reactive T cells, given as a flat 350 mg infusion every 3 weeks. It was the first systemic therapy approved for advanced cutaneous squamous cell carcinoma (2018) and remains the standard there, and in 2025 it became the first adjuvant immunotherapy for high-risk cutaneous squamous cell carcinoma after surgery and radiation, based on C-POST. It is also approved for advanced basal cell carcinoma after a hedgehog inhibitor (2021) and for first-line NSCLC with PD-L1 of 50 percent or more (EMPOWER-Lung 1, 2021). Developed by Regeneron with Sanofi, it shares the class profile of fatigue, musculoskeletal pain, rash, diarrhoea and hypothyroidism. Selecting skin cancer patients for adjuvant therapy remains open. It gave advanced skin squamous cell carcinoma its first effective drug.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Cemiplimab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Cemiplimab"},{"label":"NICE TA1165: cemiplimab with platinum-based chemotherapy for untreated advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1165"}],"tags":[],"related":["pd-l1-tps"],"cancers":["nsclc","melanoma","pdl1-high-nsclc","cutaneous-scc","advanced-cutaneous-scc","lip-cancer","basal-cell-carcinoma","locally-advanced-bcc","cervical","recurrent-metastatic-cervical-cancer","lung-cancer"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["regeneron"],"institutions":[],"pathways":[],"terms":["skin-cancer-after-organ-transplant"],"trials":["nct06246916","nct05800015","nct06585410","nct05317858","nct05608291","nct05785767","nct03972657","nct04050436","nct06463665","nct05557591","nct04291105","nct06162572","nct06161441","nct03916627","nct06190951","nct07594106","nct06413680","nct06769698","nct04305795","nct06908304","nct06384807","nct04626635","nct05208944","nct03491683","empower-lung-1","c-post","neoadjuvant-cemiplimab-cscc","cemiplimab-advanced-bcc","cemiplimab-kidney-transplant-cscc"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Libtayo","modality":"Monoclonal antibody (anti-PD-1)","mechanism":"Human IgG4 anti-PD-1.","approvals":[{"region":"US","year":2018,"indication":"Advanced cutaneous squamous cell carcinoma"},{"region":"US","year":2025,"indication":"Adjuvant high-risk CSCC after surgery and radiation"},{"region":"England (NICE)","year":2026,"indication":"Untreated locally advanced non-small-cell lung cancer unsuitable for definitive chemoradiation, or metastatic disease, with PD-L1 in 1 percent or more of tumour cells and no EGFR or ALK alteration, with platinum-based chemotherapy","note":"TA1165, published 16 June 2026, as an option where pembrolizumab with platinum-based chemotherapy would otherwise be offered."}],"mechanismSteps":["Antibody binds PD-1","PD-1/PD-L1 engagement between T cell and tumour is blocked","Exhausted tumour-reactive T cells regain effector function","Interferon-γ and cytotoxic granules are released at the tumour","Tumour cells are killed; memory T cells persist"],"dosing":{"route":"IV infusion","schedule":"350 mg every 3 weeks","modifications":"Standard immune-mediated event algorithm","monitoring":"Thyroid, LFTs, creatinine, skin","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Fatigue"},{"event":"Musculoskeletal pain"},{"event":"Rash"},{"event":"Diarrhoea"},{"event":"Anaemia"},{"event":"Hypothyroidism"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.libtayosurroundcare.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for advanced cutaneous squamous cell carcinoma (TA592) and PD-L1 ≥50% NSCLC","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2018-09-28","type":"approval","region":"US","note":"Advanced cutaneous squamous cell carcinoma: first systemic therapy approved for CSCC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-02-09","type":"accelerated-approval","region":"US","note":"Advanced basal cell carcinoma after hedgehog inhibitor","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Patients with metastatic basal cell carcinoma (mBCC) previously treated with a hedgehog pathway inhibitor or for whom a hedgehog pathway inhibitor is not appropriate"},{"date":"2021-02-22","type":"approval","region":"US","note":"First-line PD-L1 ≥50% NSCLC (EMPOWER-Lung 1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-04-28","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2021 converted to traditional approval 2.2 years after it was granted.","indication":"Patients with metastatic basal cell carcinoma (mBCC) previously treated with a hedgehog pathway inhibitor or for whom a hedgehog pathway inhibitor is not appropriate"},{"date":"2025-10","type":"approval","region":"US","note":"Adjuvant high-risk CSCC after surgery and radiation (C-POST)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"ceralasertib","kind":"drug","name":"Ceralasertib","aka":[],"tldr":"Ceralasertib is an experimental small-molecule drug from AstraZeneca in phase 3 trials for non-small-cell lung cancer, melanoma and triple-negative breast cancer, aimed at ATR.","summary":"Ceralasertib (AZD6738) is a small-molecule drug developed by AstraZeneca. Its target is ATR (the sponsor names ATR (Ataxia Telangiectasia and Rad3-related) kinase). The sponsor states: Ceralasertib is an oral small-molecule ATR kinase inhibitor, tested here in combination with the PD-L1 inhibitor durvalumab; the CT.gov record's exclusion criterion barring prior ATR inhibitor use is consistent with this. ClinicalTrials.gov describes the intervention as: Participants will receive oral tablet of ceralasertib as stated in arm description. It is the investigational product in 5 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05450692 (A Phase III Study of Ceralasertib Plus Durvalumab Versus Docetaxel in Patients With Non Small Cell Lung Cancer (NSCLC) Whose Disease Progressed On or After Prior Anti PD (L)1 Therapy And Platinum Based Chemotherapy), in non-small-cell lung cancer, melanoma and triple-negative breast cancer. The largest, NCT05450692, plans to enrol 594 participants (actual) with primary completion was scheduled for 2025-10-06 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Ceralasertib","url":"https://clinicaltrials.gov/search?intr=AZD6738"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","melanoma","tnbc"],"sections":[],"technologies":[],"targets":["atr"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05450692","nct05061134","nct05941897","phoenix"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AZD6738","modality":"small molecule","mechanism":"Ceralasertib is an oral small-molecule ATR kinase inhibitor, tested here in combination with the PD-L1 inhibitor durvalumab; the CT.gov record's exclusion criterion barring prior ATR inhibitor use is consistent with this.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ceritinib","kind":"drug","name":"Ceritinib","aka":[],"tldr":"Ceritinib is a second-generation ALK pill for lung cancer, effective after crizotinib but with gastrointestinal toxicity that limited uptake.","summary":"Ceritinib is a second-generation ALK tyrosine kinase inhibitor active against several crizotinib-resistance mutations including L1196M, G1269A, I1171T and S1206Y. The ASCEND-1, 2 and 5 studies established activity after crizotinib, and ASCEND-4 showed first-line superiority over chemotherapy with median progression-free survival of 16.6 versus 8.1 months. It received accelerated US approval in 2014 after crizotinib and full first-line approval in 2017. Gastrointestinal toxicity at the original 750 mg fasting dose limited uptake; the 450 mg dose taken with food improved tolerability with equivalent exposure. It is rarely used now because alectinib, brigatinib and lorlatinib offer better tolerability and CNS control. Ceritinib was proof that resistance to the first ALK drug could be overcome by a more potent one.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Ceritinib","links":[{"label":"ASCEND-4 (Lancet 2017)","url":"https://doi.org/10.1016/S0140-6736(17)30123-X"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ceritinib"},{"label":"NICE TA500: ceritinib for untreated ALK-positive non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta500"}],"tags":["gap-fill"],"related":[],"cancers":["nsclc","alk-positive-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["alk"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01828099","nct01828112","nct03784014"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zykadia","modality":"Small-molecule ALK TKI (second generation)","mechanism":"Potent ALK inhibitor active against several crizotinib-resistance mutations (L1196M, G1269A, I1171T, S1206Y).","approvals":[{"region":"US","year":2014,"indication":"ALK-positive metastatic NSCLC after crizotinib"},{"region":"US","year":2017,"indication":"First-line ALK-positive metastatic NSCLC"},{"region":"EU","year":2015,"indication":"Conditional May 2015; full approval 2017"},{"region":"England (NICE)","year":2018,"indication":"Untreated ALK-positive advanced non-small-cell lung cancer","note":"TA500, published 24 January 2018, with the discount agreed in the patient access scheme (ASCEND-4)."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2014-04-29","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"ALK-positive metastatic NSCLC that progressed on or is intolerant to crizotinib"},{"date":"2017-05-26","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2014 converted to traditional approval 3.1 years after it was granted.","indication":"ALK-positive metastatic NSCLC that progressed on or is intolerant to crizotinib"}]},{"id":"cervavac","kind":"drug","name":"CERVAVAC (quadrivalent HPV vaccine, India)","aka":["qHPV vaccine (Serum Institute)","Cervavac"],"tldr":"India's first home-made HPV vaccine, protecting against the two virus types that cause most cervical cancers and the two that cause genital warts, at a price meant for a national programme.","summary":"CERVAVAC is a quadrivalent HPV vaccine (types 6, 11, 16 and 18) developed by the Serum Institute of India with Department of Biotechnology support and produced in Hansenula polymorpha yeast. It is indicated for girls and women aged 9 to 26 against cervical, vulvar, vaginal and anal cancers and genital warts, and for boys and men aged 9 to 26 against anal cancer and genital warts; the schedule is two doses at 0 and 6 months for ages 9 to 14 and three doses (0, 2 and 6 months) for ages 15 to 26. It received Indian market authorisation in 2022 and was introduced in 2023, and published comparisons report immunogenicity non-inferior to established vaccines.\n\nIts importance is supply and price: India records about 127,500 cervical cancer cases and 79,900 deaths a year (GLOBOCAN 2022), and vaccination depended on two imported products. A domestic vaccine, together with the IARC India evidence that a single dose protects as well as two or three, is what makes a national programme for girls aged 9 to 14 affordable; its proposed inclusion in the Universal Immunisation Programme is described in the 2026 literature as a milestone still being rolled out.","status":"approved","asOf":"2026-09-10","links":[{"label":"Serum Institute of India: CERVAVAC","url":"https://www.seruminstitute.com/product_ind_cervavac.php"},{"label":"Cervical cancer elimination in India (Cancer 2026)","url":"https://doi.org/10.1002/cncr.70292"},{"label":"New HPV vaccines on the market (Vaccines 2026)","url":"https://doi.org/10.3390/vaccines14020140"}],"tags":[],"related":["gardasil-9","idea-single-dose-hpv-self-sampling-elimination"],"cancers":["cervical","head-and-neck"],"sections":[],"technologies":["hpv-vaccine"],"targets":[],"drugs":[],"companies":["serum-institute-of-india"],"institutions":["dbt-india","iarc"],"pathways":[],"terms":[],"trials":["iarc-india-hpv-dose-study"],"people":[],"bottlenecks":["b-prevention-adoption","b-global-access"],"keyPapers":["paper-basu-single-dose-hpv-lancet-oncol-2021","paper-tamrakar-cancer","paper-akgor-vaccines-basel"],"journals":[],"dependsOn":[],"notes":[],"brand":"CERVAVAC","modality":"Prophylactic vaccine (virus-like particles)","mechanism":"Recombinant L1 capsid proteins of HPV 6, 11, 16 and 18 self-assemble into virus-like particles that induce neutralising antibodies preventing persistent infection.","approvals":[{"region":"IN","year":2022,"indication":"Prevention of HPV 6/11/16/18-related cancers and genital warts, ages 9-26","note":"Market authorisation from the DCGI in 2022; introduced 2023"}],"mechanismSteps":["L1 protein particles mimic the virus shell without DNA","The immune system makes neutralising antibodies","Antibodies at the cervix block HPV from infecting basal cells","No persistent infection, so no precancer or cancer"],"dosing":{"route":"Intramuscular","schedule":"Ages 9-14: two doses at 0 and 6 months; ages 15-26: three doses at 0, 2 and 6 months","monitoring":"None routine","source":"https://www.seruminstitute.com/product_ind_cervavac.php"},"toxicity":[],"access":[{"country":"IN","reimbursement":"Planned for the Universal Immunisation Programme; state programmes and private market meanwhile","generic":false,"source":"https://doi.org/10.1002/cncr.70292","asOf":"2026-09-10"}],"regulatoryEvents":[]},{"id":"cetrelimab","kind":"drug","name":"Cetrelimab","aka":[],"tldr":"Cetrelimab is an experimental investigational agent whose form is not stated in the registry from Janssen Research & Development in phase 3 trials for bladder & urothelial cancer, prostate cancer and non-small-cell lung cancer, with its target not yet stated publicly.","summary":"Cetrelimab (JNJ-63723283) is an investigational agent whose form is not stated in the registry developed by Janssen Research & Development. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Participants will receive intravenous Cetrelimab. It is the investigational product in 6 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT04658862 (A Study of TAR-200 in Combination With Cetrelimab Versus Concurrent Chemoradiotherapy in Participants With Muscle-invasive Bladder Cancer (MIBC) of the Bladder) and NCT05714202 (A Study of TAR-200 in Combination With Cetrelimab or TAR-200 Alone Versus Intravesical Bacillus Calmette-Guérin (BCG) in Participants With BCG-naïve High-risk Non-muscle Invasive Bladder Cancer (HR-NMIBC)), in bladder & urothelial cancer, prostate cancer and non-small-cell lung cancer. The largest, NCT05714202, plans to enrol 1135 participants (actual) with primary completion expected 2029-09-18. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Cetrelimab","url":"https://clinicaltrials.gov/search?intr=JNJ-63723283"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial","prostate","nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04658862","hr-nmibc","nct02908906","nct03431350","nct05908734","nct03473743"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"JNJ-63723283","modality":"antibody (INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cetuximab","kind":"drug","name":"Cetuximab","aka":[],"tldr":"Cetuximab is a chimeric antibody that blocks the EGFR growth receptor. It is used with FOLFIRI or FOLFOX in RAS wild-type, left-sided bowel cancer, with encorafenib in BRAF V600E disease, with KRAS G12C inhibitors, and with radiation or chemotherapy in head and neck cancer; RAS-mutant tumours gain nothing and may be harmed, so RAS testing comes first.","summary":"Chimeric IgG1 anti-EGFR. First-line with FOLFIRI/FOLFOX in RAS/BRAF wild-type, left-sided mCRC; with encorafenib (± chemotherapy) in BRAF V600E; with KRAS G12C inhibitors (adagrasib); in head and neck cancer with radiation or chemotherapy. Requires RAS testing (KRAS and NRAS exons 2-4) because RAS-mutant tumours do not benefit and may be harmed. Acneiform rash correlates with response; infusion reactions and hypomagnesaemia are characteristic. Biosimilars are emerging.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Cetuximab","links":[{"label":"FDA label","url":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/125084s279lbl.pdf"},{"label":"NICE TA439: cetuximab and panitumumab for previously untreated metastatic colorectal cancer","url":"https://www.nice.org.uk/guidance/ta439"},{"label":"EMA Erbitux","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/erbitux"}],"tags":[],"related":["ras-wild-type","kras-g12d"],"cancers":["colorectal","head-and-neck","recurrent-metastatic-hnscc","hpv-negative-head-and-neck-cancer"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["egfr"],"drugs":[],"companies":["eli-lilly","merck"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["crystal-fire3","breakwater","nct07775287","nct07702032","nct04793958","nct06445062","nct07059221","nct07790510","nct06194656","nct07659795","nct06943820","nct03785249","nct05194995","nct07474727","nct07644559","nct07020221","nct07259590","nct05910827","nct05217446","nct05358249","nct07223047","nct04956640","nct07415031","nct07252739","nct04585035","nct07286149","nct02671435","nct06662786","nct05584670","nct06385678","nct06750094","nct07209111","nct06915142","nct05410145","mountaineer","nct05239741","nct00248287","nct05226871","nct06332092","prime","opus","calgb-80405","swog-s1406","new-epoc","n0147","petacc-8"],"people":[],"bottlenecks":[],"keyPapers":["paper-karapetis-kras-cetuximab-colorectal-nejm-2008","paper-arnold-primary-tumour-side-ras-wild-type-ann-oncol-2017","paper-tejpar-tumour-location-crystal-fire3-jama-oncol-2017"],"journals":[],"dependsOn":[],"notes":["Colorectal cancer biomarkers: eligibility is extended RAS wild-type (KRAS and NRAS exons 2, 3 and 4) plus, in practice, a left-sided primary. RAS mutation predicts no benefit at all, with overall survival hazard ratios of 0.55 in wild-type and 0.98 in mutant tumours against supportive care (Karapetis 2008), and side is predictive as well as prognostic: overall survival hazard ratio 0.75 on the left and 1.12 on the right (Arnold 2017, Tejpar 2017). Acquired resistance appears as RAS-mutant clones in plasma months before progression (Misale 2012, Diaz 2012)."],"brand":"Erbitux","modality":"Monoclonal antibody (anti-EGFR)","mechanism":"Binds EGFR domain III, blocking ligand binding and dimerisation; also ADCC via IgG1 Fc.","approvals":[{"region":"US","year":2004,"indication":"EGFR-expressing metastatic colorectal cancer (later restricted to RAS wild-type)"},{"region":"US","year":2006,"indication":"Head and neck squamous cell carcinoma with radiation"},{"region":"US","year":2020,"indication":"BRAF V600E mCRC with encorafenib"},{"region":"US","year":2024,"indication":"KRAS G12C mCRC with adagrasib"},{"region":"EU","year":2004,"indication":"EGFR-expressing metastatic colorectal cancer; later restricted to RAS wild-type","note":"Erbitux marketing authorisation issued 29 June 2004."},{"region":"England (NICE)","year":2017,"indication":"Previously untreated EGFR-expressing RAS wild-type metastatic colorectal cancer with FOLFOX or FOLFIRI","note":"TA439, published 29 March 2017 and last updated 25 September 2017, recommends cetuximab within its marketing authorisation subject to the commercial access agreement. TA242 (2012) covers use after first-line chemotherapy and NICE does not recommend cetuximab beyond first line."}],"mechanismSteps":["Cetuximab binds the extracellular domain of EGFR on the tumour cell","Ligands (EGF, TGF-α) can no longer bind; the receptor cannot pair up and signal","RAS-MAPK and PI3K signalling downstream falls, slowing growth; requires RAS to be wild-type","The IgG1 tail recruits NK cells for antibody-dependent killing"],"dosing":{"route":"Intravenous","schedule":"400 mg/m² loading then 250 mg/m² weekly, or 500 mg/m² every 2 weeks","modifications":"Hold or reduce for grade 3 rash; premedicate for infusion reactions","monitoring":"Magnesium, potassium, calcium; skin","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/125084s279lbl.pdf"},"toxicity":[{"event":"Acneiform rash","note":"Very common; severity correlates with response"},{"event":"Hypomagnesaemia","note":"Common, can be severe; monitor electrolytes"},{"event":"Infusion reactions","note":"Including rare severe reactions; higher in the southeastern US (alpha-gal sensitisation)"},{"event":"Diarrhoea","note":"Additive with FOLFIRI"}],"access":[],"regulatoryEvents":[{"date":"2004-02-12","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with irinotecan for EGFR-expressing metastatic colorectal carcinoma refractory to irinotecan-based chemotherapy"},{"date":"2004-02-12","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"As a single agent for EGFR-expressing metastatic colorectal carcinoma intolerant to irinotecan-based chemotherapy"},{"date":"2004-02","type":"approval","region":"US","note":"Initial approval in EGFR-expressing mCRC (BOND trial)"},{"date":"2007-10-02","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2004 converted to traditional approval 3.6 years after it was granted.","indication":"As a single agent for EGFR-expressing metastatic colorectal carcinoma intolerant to irinotecan-based chemotherapy"},{"date":"2012-07-06","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2004 converted to traditional approval 8.4 years after it was granted.","indication":"In combination with irinotecan for EGFR-expressing metastatic colorectal carcinoma refractory to irinotecan-based chemotherapy"},{"date":"2012-07","type":"label-change","region":"US","note":"Label restricted to KRAS wild-type; first-line FOLFIRI indication (CRYSTAL)"},{"date":"2020-04","type":"approval","region":"US","note":"With encorafenib for BRAF V600E mCRC (BEACON)"}]},{"id":"cetuximab-sarotalocan","kind":"drug","name":"Cetuximab sarotalocan","aka":[],"tldr":"Cetuximab sarotalocan is an EGFR antibody carrying a light-activated dye: after infusion, a red laser is shone on the tumour and the cells burst. It has been approved in Japan since 2020.","summary":"Cetuximab sarotalocan is Rakuten Medical's photoimmunotherapy. It was approved in Japan (September 2020) for unresectable locally advanced or recurrent head and neck cancer with conditional approval; two global phase 3 trials in locally recurrent HNSCC continue without US approval to date. Also being studied in cutaneous squamous cell carcinoma.","status":"approved","asOf":"2026-09-07","links":[{"label":"Development status","url":"https://www.pharmaceutical-technology.com/data-insights/cetuximab-sarotalocan-rakuten-medical-recurrent-head-and-neck-squamous-cell-carcinoma-likelihood-of-approval/"}],"tags":[],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":["photoimmunotherapy"],"targets":["egfr"],"drugs":[],"companies":["rakuten-medical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06699212"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Akalux","code":"ASP-1929, RM-1929","modality":"Photoimmunotherapy conjugate (anti-EGFR antibody-IR700 dye)","mechanism":"Cetuximab conjugated to IRDye700DX; 690 nm light triggers photochemical membrane damage in EGFR-bound cells and immunogenic cell death.","approvals":[{"region":"Japan","year":2020,"indication":"Unresectable locally advanced or recurrent head and neck cancer (conditional)"}],"mechanismSteps":["Antibody-dye conjugate binds EGFR on tumour cells within 24 hours","Near-infrared light (690 nm) is delivered by surface or interstitial fibres","Photochemical reaction ruptures the membrane of bound cells only","Released antigens and damage signals recruit an immune response"],"dosing":{"route":"Intravenous infusion, then light at 24 hours","schedule":"640 mg/m² followed by 50 J/cm² (surface) or 100 J/cm (interstitial) illumination; repeatable","source":"https://www.pmda.go.jp/english/"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2020-09-25","type":"approval","region":"Japan","note":"World-first approval of a photoimmunotherapy drug"}]},{"id":"cevostamab","kind":"drug","name":"Cevostamab","aka":[],"tldr":"Cevostamab is an experimental investigational agent whose form is not stated in the registry from Hoffmann-La Roche in phase 3 trials for multiple myeloma, with its target not yet stated publicly.","summary":"Cevostamab is an investigational agent whose form is not stated in the registry developed by Hoffmann-La Roche. The sponsor describes its target as FcRH5, which OnCo does not yet have a target page for. ClinicalTrials.gov describes the intervention as: Participants will receive cevostamab IV as per the schedule given in the protocol. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07555938 (Cevostamab in Combination With Pomalidomide and Dexamethasone Versus Standard of Care in Participants With Previously Treated Multiple Myeloma), in multiple myeloma. The largest, NCT07555938, plans to enrol 380 participants with primary completion expected 2028-10-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Cevostamab","url":"https://clinicaltrials.gov/search?intr=Cevostamab"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07555938","nct05583617","nct05535244"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody (INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cgt4859","kind":"drug","name":"CGT4859","aka":[],"tldr":"CGT4859 is an experimental small-molecule drug from Cogent Biosciences in phase 2 trials for biliary tract cancer, aimed at FGFR2.","summary":"CGT4859 is a small-molecule drug developed by Cogent Biosciences. Its target is FGFR2 (the sponsor names FGFR2, FGFR3). The sponsor states: A selective FGFR2/3 inhibitor designed to target tumours with genetic alterations in these fibroblast growth factor receptors. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in biliary tract cancer. The largest, NCT06777316, plans to enrol 110 participants with primary completion expected 2027-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of CGT4859","url":"https://clinicaltrials.gov/search?intr=CGT4859"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cholangiocarcinoma"],"sections":[],"technologies":[],"targets":["fgfr2"],"drugs":[],"companies":["cogent-biosciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06777316"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"A selective FGFR2/3 inhibitor designed to target tumours with genetic alterations in these fibroblast growth factor receptors.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"chiauranib","kind":"drug","name":"Chiauranib","aka":[],"tldr":"Chiauranib is an experimental small-molecule drug from Chipscreen Biosciences in phase 3 trials for ovarian cancer, aimed at VEGF / VEGFR and KIT.","summary":"Chiauranib (CS2164) is a small-molecule drug developed by Chipscreen Biosciences. Its targets are VEGF / VEGFR, KIT, PDGFRA, CSF1R and Folate receptor alpha (the sponsor names VEGFR1, VEGFR2, VEGFR3, PDGFRa, c-Kit, Aurora B, CSF-1R). The sponsor states: A novel orally active multi-target inhibitor that simultaneously inhibits angiogenesis-related kinases (VEGFR1/2/3, PDGFRa, c-Kit), the mitosis-related kinase Aurora B, and the chronic-inflammation-related kinase CSF-1R at single-digit nanomolar potency. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04921527 (Chiauranib Plus Weekly Paclitaxel in Patients with Platinum-refractory or Platinum-resistant Recurrent Ovarian Cancer), in ovarian cancer. The largest, NCT04921527, plans to enrol 454 participants with primary completion was scheduled for 2024-12-31 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Chiauranib","url":"https://clinicaltrials.gov/search?intr=CS2164"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":["vegf","kit","pdgfra","csf1r","folr1"],"drugs":[],"companies":["chipscreen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04921527"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CS2164","modality":"small molecule","mechanism":"A novel orally active multi-target inhibitor that simultaneously inhibits angiogenesis-related kinases (VEGFR1/2/3, PDGFRa, c-Kit), the mitosis-related kinase Aurora B, and the chronic-inflammation-related kinase CSF-1R at single-digit nanomolar potency.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"chlorambucil","kind":"drug","name":"Chlorambucil","aka":[],"tldr":"Chlorambucil (Leukeran) is a gentle oral chemotherapy tablet used for decades in chronic lymphocytic leukaemia and low-grade lymphomas, mainly in older patients.","summary":"Approved in 1957, chlorambucil is indicated for chronic lymphocytic leukaemia and malignant lymphomas including lymphosarcoma, giant follicular lymphoma and Hodgkin disease; the label states it is palliative rather than curative. It was the comparator arm in the trials that established fludarabine, alemtuzumab and, with obinutuzumab (CLL11), the chemoimmunotherapy era in unfit CLL patients, and it is still used with an anti-CD20 antibody in frail patients where BTK inhibitors or venetoclax are unavailable. Myelosuppression is dose-limiting; long-term use increases the risk of secondary leukaemia and it is teratogenic and impairs fertility.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Chlorambucil","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=chlorambucil"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/chlorambucil"}],"tags":["nci-list","generic"],"related":[],"cancers":["cll","hodgkin-lymphoma","follicular-lymphoma","cll-treatment-naive"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04075292","nct06970743"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Leukeran","modality":"Alkylating agent (nitrogen mustard)","mechanism":"Aromatic nitrogen mustard that alkylates DNA, interfering with replication and inducing p53- and Bax-mediated apoptosis.","approvals":[{"region":"US","year":1957,"indication":"Chronic lymphocytic leukaemia; Hodgkin and non-Hodgkin lymphomas (palliative)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"choline-c11","kind":"drug","name":"Choline C-11","aka":["[11C]choline"],"tldr":"Choline C-11 was the first PET tracer approved in the United States, in 2012, to find where prostate cancer has come back when PSA rises after treatment; PSMA tracers have now largely replaced it.","summary":"The FDA approved Choline C 11 Injection in September 2012, produced at the Mayo Clinic, for PET imaging of patients with suspected prostate cancer recurrence and non-informative bone scan, CT or MRI, the first approval of a PET agent for prostate cancer. Its 20-minute half-life means it must be made on site with a cyclotron, which confined it to a few centres. From 2020 the PSMA-targeted tracers gallium-68 PSMA-11 and piflufolastat F-18, with higher sensitivity at low PSA and longer half-lives, took over most recurrence imaging, and fluciclovine F-18 offered a distributable amino-acid alternative.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Choline_C-11","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Choline_C-11"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=choline%20c%2011"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["pet","psma-pet"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct02260817","nct01377220"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Choline C 11 Injection","modality":"PET radiotracer, intravenous","mechanism":"Carbon-11-labelled choline is taken up by cells with high phosphatidylcholine synthesis, including prostate cancer, and imaged by PET within minutes because of the isotope's 20-minute half-life.","approvals":[{"region":"US","year":2012,"indication":"PET imaging of suspected prostate cancer recurrence with non-informative conventional imaging"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ciltacabtagene-autoleucel","kind":"drug","name":"Ciltacabtagene autoleucel","aka":[],"tldr":"Ciltacabtagene autoleucel is a one-time BCMA CAR-T for myeloma that, in CARTITUDE-4, cut the risk of death by about 45% compared with standard regimens.","summary":"Approved 2022 (≥4 lines) and 2024 (≥1 prior line, lenalidomide-refractory; CARTITUDE-4 with OS benefit HR 0.55). A third of patients in CARTITUDE-1 remain progression-free at 5 years without maintenance. Delayed neurotoxicity (parkinsonism) and secondary malignancies are rare but notable. Legend Biotech/Johnson & Johnson.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Ciltacabtagene_autoleucel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ciltacabtagene%20autoleucel"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-relapsed-refractory"],"sections":[],"technologies":["car-t"],"targets":["bcma"],"drugs":[],"companies":["legend-biotech","johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06550895","nct04133636","nct06577025","nct07149857"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Carvykti","code":"cilta-cel","modality":"CAR-T (BCMA)","mechanism":"Two BCMA-binding VHH domains, 4-1BB costimulation; lentiviral.","approvals":[{"region":"US","year":2022,"indication":"Relapsed/refractory myeloma ≥4 lines"},{"region":"US","year":2024,"indication":"Relapsed myeloma after ≥1 line, lenalidomide-refractory"}],"mechanismSteps":["Patient's T cells are collected by leukapheresis","Cells are engineered to express a CAR against BCMA (two VHH binders) and expanded","Patient receives lymphodepleting chemotherapy","CAR-T cells are infused, home to tumour, and expand","CAR binds BCMA (two VHH binders); T cell kills the tumour cell and proliferates","Memory CAR-T cells persist and patrol"],"dosing":{"route":"Single IV infusion after lymphodepletion","schedule":"0.5 to 1.0 × 10⁶ CAR+ viable T cells/kg (max 1 × 10⁸); cyclophosphamide 300 mg/m² + fludarabine 30 mg/m² days −5 to −3","modifications":"Delay infusion for active infection; tocilizumab and steroids for CRS; anakinra for refractory","monitoring":"Daily monitoring for 10 days for CRS/ICANS; delayed neurotoxicity (parkinsonism) for months; blood counts and IgG; secondary malignancy surveillance for 15 years","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7d040b91-3fb8-41db-ba7f-60a36f06e2c2"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":78,"grade3PlusPct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7d040b91-3fb8-41db-ba7f-60a36f06e2c2","note":"CARTITUDE-4, n=188"},{"event":"Neutropenia (grade 3+)","grade3PlusPct":95,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7d040b91-3fb8-41db-ba7f-60a36f06e2c2","note":"CARTITUDE-4, n=188"},{"event":"Hypogammaglobulinaemia","anyGradePct":94,"grade3PlusPct":9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7d040b91-3fb8-41db-ba7f-60a36f06e2c2","note":"CARTITUDE-4, n=188"},{"event":"Infections (grade 3+)","grade3PlusPct":24,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7d040b91-3fb8-41db-ba7f-60a36f06e2c2","note":"CARTITUDE-4, n=188"},{"event":"ICANS","anyGradePct":7,"grade3PlusPct":0.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7d040b91-3fb8-41db-ba7f-60a36f06e2c2","note":"CARTITUDE-4, n=188"},{"event":"Parkinsonism / movement disorders","anyGradePct":1,"grade3PlusPct":0,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7d040b91-3fb8-41db-ba7f-60a36f06e2c2","note":"CARTITUDE-4, n=188"},{"event":"Secondary haematological malignancies","anyGradePct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7d040b91-3fb8-41db-ba7f-60a36f06e2c2","note":"CARTITUDE-4, n=188"}],"access":[{"country":"US","listPrice":"$465,000 per infusion (list price at launch, 2022; 2026 list higher)","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.janssencarepath.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for relapsed myeloma after ≥1 line, lenalidomide-refractory (2025)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2019-12","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-02-28","type":"approval","region":"US","note":"Relapsed/refractory myeloma after ≥4 lines (CARTITUDE-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-04-05","type":"approval","region":"US","note":"Relapsed myeloma after ≥1 prior line, lenalidomide-refractory (CARTITUDE-4)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-01-19","type":"label-change","region":"US","note":"Class boxed warning for T-cell malignancies","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-06","type":"label-change","region":"US","note":"REMS requirements removed for CAR-T class","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"cinacalcet","kind":"drug","name":"Cinacalcet","aka":[],"tldr":"Cinacalcet is a tablet that quiets overactive parathyroid tissue, approved in 2004 and labelled for the dangerous high calcium levels of parathyroid carcinoma, a rare cancer where surgery often cannot remove all the hormone-producing tissue.","summary":"Cinacalcet, developed by NPS and Amgen, was approved by the FDA in March 2004 for secondary hyperparathyroidism in dialysis patients and for hypercalcaemia in patients with parathyroid carcinoma, the only drug with that oncology indication. In an open-label study of 29 patients with inoperable parathyroid carcinoma, it lowered serum calcium by at least 1 mg/dL in about two-thirds, controlling the hypercalcaemia that is the main cause of death in the disease. Nausea and vomiting are the common side effects. It is also approved for primary hyperparathyroidism in patients who cannot have surgery.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Cinacalcet","links":[{"label":"Drugs@FDA NDA021688","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=021688"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=cinacalcet"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Cinacalcet"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["parathyroid-carcinoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01054079"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Sensipar / Mimpara","modality":"Oral small-molecule calcimimetic","supportive":true,"mechanism":"Increases the sensitivity of the calcium-sensing receptor on parathyroid cells so they release less parathyroid hormone at any given blood calcium.","approvals":[{"region":"US","year":2004,"indication":"Hypercalcaemia in patients with parathyroid carcinoma; secondary hyperparathyroidism on dialysis; primary hyperparathyroidism when surgery is not possible"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cinrebafusp-alfa","kind":"drug","name":"Cinrebafusp alfa","aka":["Cinrebafusp alfa (PRS-343)"],"tldr":"Cinrebafusp alfa is an experimental fusion protein from Pieris Pharmaceuticals in phase 2 trials for gastric & gastro-oesophageal junction cancer, aimed at HER2.","summary":"Cinrebafusp alfa (PRS-343) is a fusion protein developed by Pieris Pharmaceuticals. Its target is HER2 (the sponsor names HER2 x 4-1BB). The sponsor states: Cinrebafusp alfa (PRS-343) is a HER2/4-1BB bispecific anticalin-antibody fusion protein that binds HER2 on tumour cells while engaging 4-1BB (CD137) to costimulate T cells. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in gastric & gastro-oesophageal junction cancer. The largest, NCT05190445, plans to enrol 80 participants with primary completion was scheduled for 2023-02-01 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Cinrebafusp alfa","url":"https://clinicaltrials.gov/search?intr=PRS-343"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric"],"sections":[],"technologies":[],"targets":["her2","cd137"],"drugs":[],"companies":["pieris-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05190445"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PRS-343","modality":"fusion protein","mechanism":"Cinrebafusp alfa (PRS-343) is a HER2/4-1BB bispecific anticalin-antibody fusion protein that binds HER2 on tumour cells while engaging 4-1BB (CD137) to costimulate T cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cisplatin","kind":"drug","name":"Cisplatin","aka":[],"tldr":"Cisplatin is the original platinum chemotherapy, discovered by accident in 1965; it cures testicular cancer and makes radiation work better in cervical and head and neck cancer.","summary":"Approved 1978. Weekly cisplatin with radiation has been the backbone of curative cervical cancer treatment since five 1999 trials (the NCI alert), and remains so within KEYNOTE-A18 and INTERLACE. Nephrotoxicity, ototoxicity, neuropathy, and emesis limit it; carboplatin substitutes when kidneys or hearing are at risk.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Cisplatin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Cisplatin"}],"tags":[],"related":[],"cancers":["cervical","head-and-neck","urothelial","nsclc","ovarian","pancreatic","pancreatoblastoma","locally-advanced-cervical-cancer","vaginal-adenocarcinoma","placental-site-trophoblastic-tumour","esthesioneuroblastoma"],"sections":[],"technologies":["platinum","cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["keynote-a18","interlace","euramos-1","nct06212752","nct04351555","nct04442022","nct04379635","nct07216703","nct06699212","nct05800015","nct06956001","nct06564844","nct06417814","nct07168200","nct06452277","nct06430437","nct05116462","nct03800134","nct04129502","nct06312137","nct06952504","nct05555732","nct05870319","nct03164616","nct06561386","nct02486718","nct05722015","nct06123884","nct07642024","nct06793215","nct06687369","nct05048797","nct06627647","nct04956692","nct04974398","nct03178552","nct07777822","nct07178795","nct06097728","nct07100080","nct07258979","nct07059221","nct06535607","nct06514027","nct05775159","nct06512051","nct04083599","nct06946797","nct05633667","nct04745689","nct07397338","nct07680764","nct05789082","nct05351788","nct05635708","nct07622186","nct06996782","nct06161441","nct06162221","nct05061550","nct07310784","nct06859775","nct07122687","nct05020457","nct04675333","nct05247684","nct05186974","nct06428409","nct04914598","nct04956640","nct07486817","nct05235516","nct05609578","nct07700667","nct05229497","nct06623422","nct06022861","nct05302284","nct06465563","nct07276399","nct07331155","nct07710885","nct05224141","nct05771480","nct03547973","nct06247956","nct06764875","nct07129993","nct03682068","nct06008093","nct05687266","nct06151574","nct04025879","nct05945823","nct05261399","nct06119581","nct06467357","nct07566156","nct03036098","nct07268040","nct03485209","nct02516241","nct06959082","nct02453282","nct06960577","nct06385678","nct06194448","nct07109531","nct06772623","nct04241185","nct06592326","nct02542293","nct03003962","nct05173987","nct06875310","nct07466160","nct03967977","nct07256782","nct07111546","nct07028281","nct05911295","nct05410145","nct05966194","nct06225596","nct07554456","nct05893888","nct07393542","nct07398339","nct06788912","nct06970639","nct06591520","nct05566041","nct07241767","nct05543330","nct06474468","nct06493552","nct07106762","nct03473743","nct04550260","nct04380636","nct04634877","nct06896890","ialt","jbr-10","anita","lace-pooled-analysis","turrisi-intergroup-0096"],"people":["lance-armstrong"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Platinol (generic)","modality":"Cytotoxic chemotherapy (platinum)","mechanism":"Forms intrastrand DNA crosslinks (1,2-GpG) that block replication and transcription; radiosensitises by inhibiting repair of radiation-induced damage.","approvals":[{"region":"US","year":1978,"indication":"Testicular and ovarian cancer; bladder cancer 1993"}],"mechanismSteps":["Enters the cell and loses chloride ligands to become reactive","Binds adjacent guanines, kinking DNA","Repair fails in dividing or irradiated cells","Apoptosis follows; radiation damage is fixed rather than repaired"],"dosing":{"route":"Intravenous","schedule":"40 mg/m² weekly × 5-6 with radiation (cervical); 75-100 mg/m² every 3 weeks (other tumours)","modifications":"Hold for creatinine clearance <50 mL/min, grade ≥2 neuropathy or hearing loss","monitoring":"Creatinine, magnesium, audiometry, hydration protocol"},"toxicity":[{"event":"Nausea and vomiting","anyGradePct":90,"note":"Without modern antiemetics; ~30% with NK1/5-HT3 prophylaxis"},{"event":"Nephrotoxicity (any grade)","anyGradePct":30},{"event":"Ototoxicity","anyGradePct":40,"note":"Dose-dependent, higher in children"}],"access":[],"regulatoryEvents":[]},{"id":"cladribine","kind":"drug","name":"Cladribine","aka":[],"tldr":"A one-week infusion that puts most people with hairy cell leukaemia into remission for years; also used in multiple sclerosis in tablet form.","summary":"Piro et al. (NEJM 1990) reported durable complete remissions in hairy cell leukaemia from a single 7-day course; approved 1993. Also used in mastocytosis, Langerhans cell histiocytosis, Waldenström and AML regimens (CLAG-M). Prolonged CD4 lymphopenia and infection risk; adding rituximab increases MRD-negative remission.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Cladribine","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=cladribine%20injection"}],"tags":["gap-fill","generic"],"related":[],"cancers":["hairy-cell-leukemia","waldenstrom","rosai-dorfman-disease","advanced-systemic-mastocytosis","lch-multisystem"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00136084","nct00718549","nct02072811"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Leustatin / Litak","modality":"Purine nucleoside analogue","mechanism":"2-chlorodeoxyadenosine resists adenosine deaminase, accumulates as triphosphate in lymphocytes with high deoxycytidine kinase, and induces apoptosis in dividing and resting cells.","approvals":[{"region":"US","year":1993,"indication":"Active hairy cell leukaemia"},{"region":"EU","year":2004,"indication":"Hairy cell leukaemia (Litak, subcutaneous)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"clifutinib","kind":"drug","name":"Clifutinib","aka":[],"tldr":"Clifutinib is an experimental small-molecule drug from Sunshine Lake Pharma in phase 3 trials for acute myeloid leukaemia, with its target not yet stated publicly.","summary":"Clifutinib (HEC73543) is a small-molecule drug developed by Sunshine Lake Pharma. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05586074 (HEC73543 Versus Salvage Chemotherapy in R/R FLT3-ITD AML), in acute myeloid leukaemia. The largest, NCT05586074, plans to enrol 324 participants with primary completion expected 2027-11-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Clifutinib","url":"https://clinicaltrials.gov/search?intr=HEC73543"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["aml"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct05586074","nct05133882"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"HEC73543","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"clofarabine","kind":"drug","name":"Clofarabine","aka":["Evoltra"],"tldr":"Clofarabine (Clolar) is an infusion for children and young adults whose acute lymphoblastic leukaemia has come back after at least two other treatments.","summary":"Clofarabine is a second-generation purine nucleoside analogue combining features of fludarabine and cladribine. The FDA granted accelerated approval in December 2004 for patients aged 1 to 21 with relapsed or refractory acute lymphoblastic leukaemia after at least two prior regimens, based on a single-arm study in which about 20% of heavily pretreated children achieved complete remission; the EU authorised Evoltra in 2006 for the same paediatric population. It is now used mainly as a bridge to transplant and in combination regimens for relapsed ALL, and off label in adult AML. Capillary leak and systemic inflammatory response syndrome, hepatotoxicity and prolonged cytopenias are the key risks.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Clofarabine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=clofarabine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/clofarabine"},{"label":"EPAR (Evoltra)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/evoltra"}],"tags":["nci-list"],"related":[],"cancers":["all-leukemia"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00703820","nct02883049","nct05917405"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Clolar","modality":"Purine nucleoside analogue (antimetabolite)","mechanism":"Phosphorylated by deoxycytidine kinase to clofarabine triphosphate, which inhibits ribonucleotide reductase and DNA polymerase and disrupts mitochondrial membranes, triggering apoptosis.","approvals":[{"region":"US","year":2004,"indication":"Relapsed or refractory ALL in patients aged 1 to 21 after at least two prior regimens (accelerated)"},{"region":"EU","year":2006,"indication":"Relapsed or refractory ALL in children after at least two prior regimens (Evoltra)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2004-12-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of pediatric patients 1 to 21 yrs old with relapsed or refractory acute lymphocytic leukemia (ALL) after at least 2 prior regimens"},{"date":"2022-07-18","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2004 converted to traditional approval 17.6 years after it was granted.","indication":"Treatment of pediatric patients 1 to 21 yrs old with relapsed or refractory acute lymphocytic leukemia (ALL) after at least 2 prior regimens"}]},{"id":"clonoseq","kind":"drug","name":"clonoSEQ","aka":[],"tldr":"A DNA test that counts leftover leukaemia, myeloma or lymphoma cells down to one in a million, used to decide whether treatment has really worked.","summary":"Adaptive Biotechnologies' clonoSEQ was granted FDA De Novo authorisation on 28 September 2018 (DEN170080) for minimal residual disease assessment in bone marrow from patients with acute lymphoblastic leukaemia and multiple myeloma, with chronic lymphocytic leukaemia added in 2020 and blood-based testing for some indications since. It is the reference MRD method in myeloma trials (the International Myeloma Working Group threshold of 10^-5 and 10^-6) and is covered by Medicare. MRD negativity by clonoSEQ has been accepted by the FDA as an endpoint supporting accelerated approval in myeloma (2024 advisory committee), which ties the test directly to how new drugs are approved.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA De Novo DEN170080","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpmn/denovo.cfm?id=DEN170080"},{"label":"clonoSEQ","url":"https://www.clonoseq.com"}],"tags":["test"],"related":[],"cancers":["all-leukemia","multiple-myeloma","cll","dlbcl","mantle-cell-lymphoma"],"sections":[],"technologies":["ngs-mrd-clonoseq","mrd-testing"],"targets":[],"drugs":[],"companies":["adaptive-biotechnologies"],"institutions":[],"pathways":[],"terms":["mrd","mrd-negativity-myeloma","umrd","mrd-negative-cr"],"trials":["nct06483100","nct06828991","nct06252675"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: 'MRD negative' means no cancer cells were detected at the assay's sensitivity, which predicts longer remission; 'MRD positive' may prompt continued or changed therapy depending on the disease."],"brand":"clonoSEQ","modality":"NGS minimal residual disease test (immunoglobulin and T-cell receptor clonotypes)","mechanism":"Multiplex PCR and sequencing of rearranged IGH, IGK, IGL, TRB and TRG loci identify the dominant malignant clonotypes at diagnosis, then track them in bone marrow or blood to a sensitivity of one cell in a million.","approvals":[{"region":"US","year":2018,"indication":"MRD in bone marrow, B-cell acute lymphoblastic leukaemia and multiple myeloma (De Novo)"},{"region":"US","year":2020,"indication":"MRD in chronic lymphocytic leukaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cobas-egfr-mutation-test","kind":"drug","name":"cobas EGFR Mutation Test v2","aka":[],"tldr":"The lung cancer gene test that became the first blood-based companion diagnostic the FDA ever approved.","summary":"Roche's cobas EGFR Mutation Test was approved in 2013 with erlotinib for EGFR-mutant non-small-cell lung cancer. Version 2 (PMA P150044, decision 28 September 2016 for the tissue claim; plasma claim granted in June 2016) added the T790M resistance mutation as the companion for osimertinib and, critically, allowed testing of plasma when tissue is unavailable, the first FDA-approved liquid biopsy companion diagnostic. Plasma sensitivity is lower than tissue, so a negative plasma result must be followed by tissue testing. The test is CE-marked and remains widely used where sequencing is not available.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P150044","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P150044"}],"tags":["test"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":["companion-diagnostic","liquid-biopsy"],"targets":["egfr"],"drugs":["erlotinib","osimertinib"],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["companion-diagnostic-term","ctdna"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: an EGFR mutation makes an EGFR pill the first treatment; T790M after a first-generation pill points to osimertinib."],"brand":"cobas EGFR Mutation Test","modality":"Real-time PCR companion diagnostic test (tissue and plasma)","mechanism":"Allele-specific real-time PCR for 42 EGFR mutations in exons 18 to 21, including L858R, exon 19 deletions and T790M, from tumour tissue or plasma cell-free DNA.","approvals":[{"region":"US","year":2013,"indication":"EGFR exon 19 deletion / L858R companion diagnostic for erlotinib (v1)"},{"region":"US","year":2016,"indication":"v2: T790M for osimertinib; plasma testing (first liquid biopsy companion diagnostic)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cobimetinib","kind":"drug","name":"Cobimetinib","aka":[],"tldr":"Cobimetinib is the MEK partner for vemurafenib, and the first drug approved for histiocytic neoplasms.","summary":"Cobimetinib is an allosteric MEK1/2 inhibitor given 60 mg once daily for 21 days of each 28-day cycle with the BRAF inhibitor vemurafenib, blocking two consecutive steps of the MAPK pathway. coBRIM (2015) showed PFS of 12.3 versus 7.2 months when it was added to vemurafenib in BRAF V600 melanoma. It is also approved with atezolizumab and vemurafenib (IMspire150, 2020), the first checkpoint plus targeted triplet in melanoma, and alone for histiocytic neoplasms (2022), the first drug approved for that group of rare diseases. Retinopathy, photosensitivity and creatine kinase elevation are the characteristic toxicities. Its melanoma use is limited by competition from dabrafenib-trametinib and encorafenib-binimetinib, and the value of the IMspire150 triplet remains debated. For a newcomer, cobimetinib is the MEK partner for vemurafenib and the go-to drug for histiocytosis.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Cobimetinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Cobimetinib"}],"tags":[],"related":[],"cancers":["melanoma","braf-v600-melanoma","advanced-melanoma","erdheim-chester-disease","rosai-dorfman-disease","lch-single-system","colorectal"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf"],"drugs":["vemurafenib","atezolizumab"],"companies":["roche-genentech"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct04302025","nct07449754","nct04931342","imblaze370"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["IMblaze370 tested cobimetinib with atezolizumab against regorafenib in previously treated, mostly microsatellite-stable metastatic colorectal cancer and found no difference in overall survival (8.87 against 8.51 months, hazard ratio 1.00), ending the hypothesis that MEK inhibition could make these tumours immunotherapy-sensitive."],"brand":"Cotellic","modality":"Small-molecule kinase inhibitor (MEK1/2)","mechanism":"Allosteric MEK1/2 inhibitor.","approvals":[{"region":"US","year":2015,"indication":"BRAF V600 melanoma with vemurafenib"},{"region":"US","year":2020,"indication":"With atezolizumab and vemurafenib in BRAF V600 melanoma"},{"region":"US","year":2022,"indication":"Histiocytic neoplasms"}],"mechanismSteps":["Binds MEK1/2 allosterically","Suppresses ERK reactivation that limits BRAF inhibitor monotherapy","With atezolizumab, MAPK inhibition may increase antigen presentation and T-cell infiltration"],"dosing":{"route":"Oral","schedule":"60 mg once daily for 21 days of each 28-day cycle, with vemurafenib","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/206192s006lbl.pdf"},"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cobolimab","kind":"drug","name":"Cobolimab","aka":[],"tldr":"Cobolimab is an experimental investigational agent whose form is not stated in the registry from GlaxoSmithKline in phase 3 trials for non-small-cell lung cancer, melanoma and hodgkin lymphoma, with its target not yet stated publicly.","summary":"Cobolimab is an investigational agent whose form is not stated in the registry developed by GlaxoSmithKline. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT04655976 (Efficacy Comparison of Cobolimab + Dostarlimab + Docetaxel to Dostarlimab + Docetaxel to Docetaxel Alone in Participants With Advanced Non-small Cell Lung Cancer Who Have Progressed on Prior Anti-PD-(L)1 Therapy and Chemotherapy), in non-small-cell lung cancer, melanoma, hodgkin lymphoma, glioma & glioblastoma and osteosarcoma. The largest, NCT04655976, plans to enrol 758 participants (actual) with primary completion was scheduled for 2025-06-05 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Cobolimab","url":"https://clinicaltrials.gov/search?intr=Cobolimab"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","melanoma","hodgkin-lymphoma","glioblastoma","osteosarcoma","hcc","rhabdomyosarcoma"],"sections":[],"technologies":["tim3-blockade"],"targets":[],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04655976","nct06521567"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody (INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cologuard","kind":"drug","name":"Cologuard","aka":[],"tldr":"A home stool test done every three years that looks for cancer DNA and hidden blood; a positive result means you need a colonoscopy.","summary":"Cologuard (Exact Sciences) was approved by the FDA on 11 August 2014 (PMA P130017) for average-risk adults, the first stool DNA screening test. In the pivotal DeeP-C study (NEJM 2014, 9,989 participants) sensitivity for colorectal cancer was 92.3% versus 73.8% for FIT, sensitivity for advanced precancerous lesions 42.4% versus 23.8%, and specificity 86.6% versus 94.9%. It is a USPSTF-endorsed option at a three-year interval, covered by Medicare, and by 2024 more than 16 million tests had been performed. Its weakness is the false-positive rate, which sends about one in eight negative-for-cancer people to colonoscopy, and its limited detection of polyps compared with colonoscopy.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Cologuard","links":[{"label":"FDA PMA P130017","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P130017"},{"label":"DeeP-C (NEJM 2014)","url":"https://doi.org/10.1056/NEJMoa1311194"},{"label":"USPSTF colorectal cancer screening (2021)","url":"https://www.uspreventiveservicestaskforce.org/uspstf/recommendation/colorectal-cancer-screening"}],"tags":["test"],"related":["cologuard-plus","shield"],"cancers":["colorectal"],"sections":[],"technologies":["colorectal-screening","methylation-profiling"],"targets":[],"drugs":[],"companies":["exact-sciences"],"institutions":[],"pathways":[],"terms":["fit-test","screening","ppv"],"trials":["deep-c"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: negative means repeat in three years; positive means a diagnostic colonoscopy, and most positives turn out not to be cancer."],"brand":"Cologuard","modality":"Multitarget stool DNA colorectal cancer screening test","mechanism":"Stool sample analysed for methylated NDRG4 and BMP3, mutant KRAS, and faecal haemoglobin, combined by an algorithm into a positive or negative result.","approvals":[{"region":"US","year":2014,"indication":"Colorectal cancer screening in average-risk adults aged 50 and older (later 45 and older)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cologuard-plus","kind":"drug","name":"Cologuard Plus","aka":[],"tldr":"The updated Cologuard, approved in 2024, which keeps the high cancer detection rate while causing fewer false alarms.","summary":"Cologuard Plus was approved by the FDA in October 2024 and launched in 2025. In the BLUE-C study (NEJM 2024, more than 20,000 participants) sensitivity for colorectal cancer was 93.9% versus 67.3% for FIT, sensitivity for advanced precancerous lesions was 43.4% versus 23.3%, and specificity for advanced neoplasia was 90.6% versus 94.8%, a roughly 30% reduction in false positives compared with the original Cologuard. The test is used every three years in average-risk adults aged 45 and older. Exact Sciences, now being acquired by Abbott, positions it against blood tests such as Shield, whose sensitivity for precancerous polyps is much lower.","status":"approved","asOf":"2026-09-10","links":[{"label":"BLUE-C (NEJM 2024)","url":"https://doi.org/10.1056/NEJMoa2310336"},{"label":"Exact Sciences: Cologuard Plus","url":"https://www.exactsciences.com/"}],"tags":["test"],"related":["cologuard","shield"],"cancers":["colorectal"],"sections":[],"technologies":["colorectal-screening","methylation-profiling"],"targets":[],"drugs":[],"companies":["exact-sciences"],"institutions":[],"pathways":[],"terms":["fit-test","screening","sensitivity-specificity"],"trials":["blue-c"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: the same as Cologuard, with fewer people sent for a colonoscopy they did not need."],"brand":"Cologuard Plus","modality":"Next-generation multitarget stool DNA colorectal cancer screening test","mechanism":"Redesigned stool test measuring methylated DNA markers (LASS4, LRRC4, PPP2R5C, ZDHHC1) and faecal haemoglobin with a new algorithm, replacing the mutation markers of the original test.","approvals":[{"region":"US","year":2024,"indication":"Colorectal cancer screening in average-risk adults aged 45 and older"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"copanlisib","kind":"drug","name":"Copanlisib","aka":[],"tldr":"Copanlisib is an intravenous PI3K inhibitor for relapsed follicular lymphoma, approved in 2017 and withdrawn in 2023 when its confirmatory trial failed.","summary":"Copanlisib is an intravenous pan-class I PI3K inhibitor with predominant PI3K-alpha and PI3K-delta activity, reducing AKT signalling in B-cell lymphoma. Weekly intravenous dosing gives intermittent exposure, and hyperglycaemia and hypertension are on-target alpha effects that appear during infusion. CHRONOS-1 showed a 59% response rate in relapsed indolent lymphoma, supporting accelerated US approval in 2017 for follicular lymphoma after at least two therapies. CHRONOS-3, which added copanlisib to rituximab, was positive for progression-free survival, but CHRONOS-4 failed. After the FDA class review of PI3K inhibitors in 2022, Bayer withdrew the US indication in November 2023. Copanlisib is a case study in how a drug with a real response rate can still be withdrawn when confirmatory trials do not show a clear benefit that outweighs the class toxicity.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Copanlisib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Copanlisib"}],"tags":["gap-fill"],"related":[],"cancers":["follicular-lymphoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["pik3ca"],"drugs":[],"companies":["bayer"],"institutions":[],"pathways":["pi3k-akt-mtor"],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aliqopa","modality":"Intravenous pan-class I PI3K inhibitor (α/δ predominant)","mechanism":"Weekly IV PI3K inhibition with PI3Kα and δ potency, reducing AKT signalling in B-cell lymphoma; hyperglycaemia and hypertension are on-target α effects.","approvals":[{"region":"US","year":2017,"indication":"Relapsed follicular lymphoma after ≥2 therapies","note":"Withdrawn 2023"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2017-09-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies"},{"date":"2024-03-18","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 6.5 years after its accelerated approval.","indication":"Treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies"}]},{"id":"cosibelimab","kind":"drug","name":"Cosibelimab","aka":[],"tldr":"Cosibelimab is a PD-L1 antibody approved in December 2024 for advanced cutaneous squamous cell carcinoma, offering a third immunotherapy choice.","summary":"Cosibelimab is an IgG1 monoclonal antibody against PD-L1 that keeps a functional Fc region, so in addition to blocking the PD-1/PD-L1 checkpoint it can trigger antibody-dependent cellular cytotoxicity against PD-L1-expressing cells. It is used for metastatic or locally advanced cutaneous squamous cell carcinoma that cannot be cured by surgery or radiation. In the pivotal phase 1 expansion cohorts the response rate was 47% in metastatic disease and 48% in locally advanced disease, with durable responses. A December 2023 complete response letter citing third-party manufacturing inspection findings delayed approval, and the FDA approved it in December 2024 as a third immunotherapy option after cemiplimab and pembrolizumab. Sun Pharma acquired Checkpoint Therapeutics in 2025. Whether the ADCC-competent Fc adds clinical benefit over standard PD-1 antibodies has not been tested head to head.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Cosibelimab","links":[{"label":"CK-301-101 metastatic cohort (JITC 2023)","url":"https://doi.org/10.1136/jitc-2023-007637"},{"label":"Unloxcyt label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=06bdadd5-d2db-406f-a3f8-de47f48a52e3"}],"tags":["gap-fill"],"related":[],"cancers":["cutaneous-scc","advanced-cutaneous-scc"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pdl1"],"drugs":[],"companies":["checkpoint-therapeutics"],"institutions":[],"pathways":["pd1-checkpoint"],"terms":[],"trials":["ck-301-101","nct04786964"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Unloxcyt","modality":"Anti-PD-L1 monoclonal antibody (IgG1, ADCC-competent)","mechanism":"Blocks PD-L1 while retaining an active Fc for antibody-dependent cellular cytotoxicity against PD-L1-expressing cells.","approvals":[{"region":"US","year":2024,"indication":"Metastatic or locally advanced cutaneous SCC not curable by surgery or radiation"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2023-12","type":"crl","region":"US","note":"Complete response letter citing third-party manufacturing inspection findings"},{"date":"2024-12-13","type":"approval","region":"US","note":"Approved for cSCC"}]},{"id":"cpx-351","kind":"drug","name":"CPX-351 (liposomal daunorubicin-cytarabine)","aka":[],"tldr":"The two old chemotherapy drugs packed together into tiny fat bubbles at a fixed ratio, which doubled five-year survival in older adults with high-risk AML.","summary":"Fixed 5:1 molar ratio of cytarabine to daunorubicin in liposomes that persist in marrow. Study 301 (n=309, age 60-75, therapy-related or MDS-related AML): median OS 9.56 vs 5.95 months (HR 0.69), 5-year OS 18% vs 8%; benefit concentrated in patients bridged to transplant. Approved 2017 (adults) and 2021 (paediatric). Being tested in younger high-risk patients and MDS.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Daunorubicin/cytarabine","links":[{"label":"5-year results (Lancet Haematology 2021)","url":"https://www.thelancet.com/journals/lanhae/article/PIIS2352-3026(21)00134-4/abstract"}],"tags":[],"related":[],"cancers":["aml","aml-secondary"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["jazz"],"institutions":[],"pathways":[],"terms":[],"trials":["cpx-351-301"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vyxeos","modality":"Liposomal cytotoxic formulation","mechanism":"Liposomal co-delivery maintains the synergistic drug ratio in leukaemic blasts and prolongs exposure.","approvals":[{"region":"US","year":2017,"indication":"Newly diagnosed therapy-related AML or AML with myelodysplasia-related changes, adults"},{"region":"US","year":2021,"indication":"Paediatric patients ≥1 year"}],"mechanismSteps":["Liposomes circulate for days and accumulate in bone marrow","Blasts take up intact liposomes preferentially over normal progenitors","Cytarabine and daunorubicin are released together at the synergistic 5:1 ratio","DNA chain termination plus topoisomerase II poisoning kill blasts","Deeper remissions enable more patients to reach transplant"],"dosing":{"route":"IV over 90 minutes","schedule":"Induction days 1, 3, 5 (daunorubicin 44 mg/m² + cytarabine 100 mg/m² per dose); second induction days 1, 3; consolidation at reduced dose","monitoring":"Counts, cardiac function; do not substitute for conventional 7+3 dosing","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Vyxeos"},"toxicity":[{"event":"Prolonged cytopenias","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Vyxeos","note":"Longer neutrophil and platelet recovery than 7+3"},{"event":"Haemorrhage","anyGradePct":74,"note":"Study 301, any grade"},{"event":"Febrile neutropenia","anyGradePct":68},{"event":"Cardiotoxicity","note":"Cumulative anthracycline exposure counts"}],"access":[],"regulatoryEvents":[{"date":"2017-08-03","type":"approval","region":"US","note":"Adults"},{"date":"2021-03-30","type":"label-change","region":"US","note":"Paediatric expansion"}]},{"id":"crb-701","kind":"drug","name":"CRB-701","aka":[],"tldr":"CRB-701 is an experimental antibody-drug conjugate from Corbus Pharmaceuticals in phase 2 trials, aimed at Nectin-4.","summary":"CRB-701 is an antibody-drug conjugate developed by Corbus Pharmaceuticals. Its target is Nectin-4 (the sponsor names Nectin-4). ClinicalTrials.gov describes the intervention as: Nectin-4 targeted Antibody Drug Conjugate (ADC). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT06265727, plans to enrol 348 participants with primary completion expected 2027-01-16. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of CRB-701","url":"https://clinicaltrials.gov/search?intr=CRB-701"},{"label":"Sponsor page","url":"https://www.corbuspharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["nectin4"],"drugs":[],"companies":["corbus-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06265727"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Antibody-drug conjugate directed at Nectin-4, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"crc01","kind":"drug","name":"CRC01","aka":[],"tldr":"CRC01 is an experimental CAR-T cell therapy from Curocell in phase 2 trials for diffuse large B-cell lymphoma, follicular lymphoma and acute lymphoblastic leukaemia, aimed at CD19.","summary":"CRC01 is a CAR-T cell therapy developed by Curocell. Its target is CD19 (the sponsor names CD19). The sponsor states: A single intravenous infusion of chimeric antigen receptor (CAR)-transduced autologous T cells directed against CD19, engineered with PD-1 and TIGIT knock-down to enhance T-cell effector function, given after fludarabine/cyclophosphamide conditioning. ClinicalTrials.gov describes the intervention as: Cohort A :A single infusion of chimeric antigen receptor (CAR)-transduced autologous T cells administered intravenously at a target dose of 2 x 10\\^6 anti-CD19 CAR T cells/kg. Cohort B :A single infusion of chimeric antigen receptor (CAR)-t. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in diffuse large B-cell lymphoma, follicular lymphoma and acute lymphoblastic leukaemia. The largest, NCT04836507, plans to enrol 91 participants with primary completion expected 2030-08-15. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of CRC01","url":"https://clinicaltrials.gov/search?intr=CRC01"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl","follicular-lymphoma","all-leukemia"],"sections":[],"technologies":[],"targets":["cd19"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct04836507"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"A single intravenous infusion of chimeric antigen receptor (CAR)-transduced autologous T cells directed against CD19, engineered with PD-1 and TIGIT knock-down to enhance T-cell effector function, given after fludarabine/cyclophosphamide conditioning.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cretostimogene","kind":"drug","name":"Cretostimogene grenadenorepvec","aka":[],"tldr":"Cretostimogene is a virus engineered to multiply only in bladder cancer cells with a broken RB pathway, instilled into the bladder. It cleared carcinoma in situ in 75% of BCG-unresponsive patients.","summary":"Replication restricted by the E2F-1 promoter to RB-deficient cells; expresses GM-CSF. BOND-003 cohort C: CR 75.2%, 24-month CR 41.8%, no grade 3+ treatment-related events. Rolling BLA under way; full submission expected Q4 2026. Also tested in BCG-naive (CORE-008) and intermediate-risk (PIVOT-006) NMIBC. CG Oncology.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT04452591 (BOND-003)","url":"https://clinicaltrials.gov/study/NCT04452591"}],"tags":[],"related":[],"cancers":["urothelial","non-muscle-invasive-bladder-cancer"],"sections":[],"technologies":["oncolytic-virus","bcg-and-intravesical-therapy"],"targets":[],"drugs":[],"companies":["cg-oncology"],"institutions":[],"pathways":[],"terms":["bcg-unresponsive"],"trials":["bond-003","nct07283835","nct06111235","nct06567743"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"CG0070","modality":"Oncolytic adenovirus (intravesical)","mechanism":"Conditionally replicating adenovirus lyses RB-pathway-defective tumour cells and secretes GM-CSF to recruit antigen-presenting cells.","approvals":[],"mechanismSteps":["Intravesical instillation after a surfactant (DDM) to loosen the urothelial barrier","Adenovirus enters cells; E2F-1 promoter allows replication only where RB is inactivated","Lysis releases tumour antigens; encoded GM-CSF recruits dendritic cells","Systemic anti-tumour immunity contributes to durable complete responses"],"dosing":{"route":"Intravesical","schedule":"Weekly ×6 induction, re-induction if needed, then maintenance every 3 months up to 3 years"},"toxicity":[{"event":"Bladder spasm","anyGradePct":20},{"event":"Urinary urgency","anyGradePct":18},{"event":"Grade 3+ treatment-related","grade3PlusPct":0}],"access":[],"regulatoryEvents":[{"date":"2026","type":"filing","region":"US","note":"Rolling BLA initiated; completion expected Q4 2026","source":"https://cgoncology.com/cg-oncology-reports-first-quarter-2026-financial-results-and-provides-business-updates-2/"}]},{"id":"crisantaspase","kind":"drug","name":"Crisantaspase (recombinant Erwinia asparaginase)","aka":["crisantaspase","Enrylaze","recombinant Erwinia chrysanthemi asparaginase","JZP-458"],"tldr":"Enrylaze is a replacement asparaginase for children and adults with acute lymphoblastic leukaemia or lymphoblastic lymphoma whose bodies have reacted against, or quietly neutralised, the usual E. coli asparaginase. It keeps the asparagine-starving part of their chemotherapy going.","summary":"Crisantaspase (Enrylaze) is authorised in the EU since 15 September 2023 (CHMP opinion 20 July 2023; marketing authorisation holder Jazz Pharmaceuticals Ireland Limited) as a component of a multi-agent chemotherapeutic regimen for acute lymphoblastic leukaemia and lymphoblastic lymphoma in adults and children from one month of age who have developed hypersensitivity or silent inactivation to E. coli-derived asparaginase. It is under additional monitoring in the EU. The class record `asparaginase` covers pegaspargase, calaspargase pegol and the native Erwinia enzyme; this record is the recombinant Erwinia product.","status":"approved","asOf":"2026-09-22","links":[{"label":"EMA: Enrylaze (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/enrylaze"}],"tags":["ema-register"],"related":["asparaginase","eryaspase"],"cancers":["all-leukemia","all-paediatric-standard-risk","all-paediatric-high-risk"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["jazz"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02521493","aall1731","nct04293562"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo content wave 5 (EMA medicines register and EPAR pages)","editedOn":"2026-09-22"},"brand":"Enrylaze","modality":"Enzyme therapy (recombinant asparaginase)","mechanism":"Asparaginase enzyme that depletes circulating asparagine, on which lymphoblasts depend; the recombinant Erwinia enzyme is given to patients who have reacted to or silently inactivated E. coli-derived asparaginase (indication wording, EMA).","approvals":[{"region":"EU","year":2023,"indication":"ALL and lymphoblastic lymphoma, in a multi-agent regimen, after hypersensitivity or silent inactivation to E. coli-derived asparaginase (adults and children from 1 month)","note":"Authorised 15 Sep 2023; Jazz Pharmaceuticals Ireland"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"crizotinib","kind":"drug","name":"Crizotinib","aka":[],"tldr":"Crizotinib was the first ALK inhibitor, approved four years after ALK fusions were found in lung cancer; it was also the first drug for ROS1 lung cancer and for ALK-positive lymphoma and inflammatory myofibroblastic tumour in children.","summary":"PROFILE 1007/1014 established it over chemotherapy in ALK+ NSCLC (2011 accelerated, 2013 full); ROS1 approval 2016 (PROFILE 1001, 72% response). Superseded first line by alectinib, brigatinib and lorlatinib (poor CNS penetration). Paediatric approvals for ALK+ ALCL (2021) and IMT (2022). Visual disturbance, oedema, hepatotoxicity.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Crizotinib","links":[{"label":"PROFILE 1014 (NEJM 2014)","url":"https://doi.org/10.1056/NEJMoa1408440"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=crizotinib"},{"label":"NICE TA1021: crizotinib for treating ROS1-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1021"}],"tags":["gap-fill"],"related":["alk-fusion","ros1-fusion"],"cancers":["nsclc","peripheral-t-cell-lymphoma","sarcoma","alk-positive-nsclc","ros1-positive-nsclc","met-altered-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["alk","ros1","met"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":["gene-fusion","oncogene-addiction"],"trials":["nct02767804","nct06564324","nct05987332","nct03947385","nct04603807","nct06140836","profile-1014","profile-1001-ros1"],"people":[],"bottlenecks":[],"keyPapers":["paper-solomon-n-engl-j-med"],"journals":[],"dependsOn":[],"notes":[],"brand":"Xalkori","modality":"Small-molecule ALK/ROS1/MET TKI (first generation)","mechanism":"ATP-competitive inhibitor of ALK, ROS1 and MET kinases.","approvals":[{"region":"US","year":2011,"indication":"ALK-positive metastatic NSCLC"},{"region":"US","year":2016,"indication":"ROS1-positive metastatic NSCLC"},{"region":"US","year":2021,"indication":"Relapsed ALK-positive ALCL in patients 1-21 years"},{"region":"US","year":2022,"indication":"ALK-positive inflammatory myofibroblastic tumour"},{"region":"EU","year":2012,"indication":"Conditional Oct 2012; full approval 2015"},{"region":"England (NICE)","year":2024,"indication":"ROS1-positive advanced non-small-cell lung cancer in patients who have not had a ROS1 inhibitor","note":"TA1021, published 4 December 2024; NICE asks that the least expensive of crizotinib and entrectinib is used."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2011-08-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Locally advanced or metastatic NSCLC that is ALK-positive as detected by an FDA-approved test"},{"date":"2013-11-20","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2011 converted to traditional approval 2.2 years after it was granted.","indication":"Locally advanced or metastatic NSCLC that is ALK-positive as detected by an FDA-approved test"}]},{"id":"ct3001","kind":"drug","name":"CT3001","aka":[],"tldr":"CT3001 is a small-molecule inhibitor from Crossignal Therapeutics, Inc., in registered phase 2 trials for colorectal cancer, pancreatic ductal adenocarcinoma.","summary":"CT3001 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Crossignal Therapeutics, Inc., in colorectal cancer, pancreatic ductal adenocarcinoma. Described in the registry record as a a small molecule inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of CT3001","url":"https://clinicaltrials.gov/search?intr=CT3001"},{"label":"ClinicalTrials.gov NCT06598007","url":"https://clinicaltrials.gov/study/NCT06598007"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["colorectal","pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06598007"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["CT3001 is described in its registry record as an oral solution of a small-molecule inhibitor of GPR35, a G-protein-coupled receptor. GPR35 has no target record in the corpus yet."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"CT3001","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a a small molecule inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ctx112","kind":"drug","name":"CTX112","aka":[],"tldr":"CTX112 is an experimental CAR-T cell therapy from CRISPR Therapeutics in phase 2 trials for hodgkin lymphoma, chronic lymphocytic leukaemia and follicular lymphoma, aimed at CD19.","summary":"CTX112 is a CAR-T cell therapy developed by CRISPR Therapeutics. Its target is CD19 (the sponsor names CD19). The sponsor states: An allogeneic CD19-directed CAR T-cell immunotherapy made from donor T cells genetically modified ex vivo using CRISPR-Cas9 gene editing (single guide RNA and Cas9 nuclease) to attack CD19-expressing B-cell malignancies. ClinicalTrials.gov describes the intervention as: CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in hodgkin lymphoma, chronic lymphocytic leukaemia, follicular lymphoma, mantle cell lymphoma and diffuse large B-cell lymphoma. The largest, NCT05643742, plans to enrol 120 participants with primary completion expected 2030-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of CTX112","url":"https://clinicaltrials.gov/search?intr=CTX112"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma","cll","follicular-lymphoma","mantle-cell-lymphoma","dlbcl"],"sections":[],"technologies":[],"targets":["cd19"],"drugs":[],"companies":["crispr-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05643742"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"An allogeneic CD19-directed CAR T-cell immunotherapy made from donor T cells genetically modified ex vivo using CRISPR-Cas9 gene editing (single guide RNA and Cas9 nuclease) to attack CD19-expressing B-cell malignancies.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cusatuzumab","kind":"drug","name":"Cusatuzumab","aka":[],"tldr":"Cusatuzumab is an experimental monoclonal antibody from OncoVerity in phase 2 trials for acute myeloid leukaemia, aimed at CD70.","summary":"Cusatuzumab (OV-1001, JNJ-74494550, ARGX-110) is a monoclonal antibody developed by OncoVerity. Its target is CD70. ClinicalTrials.gov describes the intervention as: Cusatuzumab IV will be administered as 10 mg/kg or 20 mg/kg on days 3 and 17 of each cycle. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in acute myeloid leukaemia. The largest, NCT06384261, plans to enrol 140 participants with primary completion expected 2027-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Cusatuzumab","url":"https://clinicaltrials.gov/search?intr=OV-1001"},{"label":"Sponsor page","url":"https://www.oncoverity.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["aml"],"sections":[],"technologies":[],"targets":["cd70"],"drugs":[],"companies":["oncoverity"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04023526"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"OV-1001, JNJ-74494550, ARGX-110","modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at CD70, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"custirsen","kind":"drug","name":"Custirsen","aka":["OGX-011","TV-1011"],"tldr":"A drug designed to switch off a protein that helps cancer cells survive chemotherapy. Two large trials found it added nothing.","summary":"Clusterin is a chaperone protein that rises after apoptotic stress, including chemotherapy, and is associated with treatment resistance. Custirsen is a second-generation antisense oligonucleotide that blocks its production, and the hypothesis was that it would re-sensitise prostate cancer to taxanes.\n\nA randomised phase 2 trial (CUOG P-06c, 42 men) reported encouraging pain responses and a correlation between low serum clusterin during treatment and survival, which was enough to justify two phase 3 trials. Both failed. SYNERGY added custirsen to first-line docetaxel in 1,022 men and found median survival 23.4 against 22.0 months, hazard ratio 0.93 (95 percent confidence interval 0.79 to 1.10, p=0.415). AFFINITY added it to cabazitaxel after docetaxel in 635 men and found 14.1 against 13.4 months, hazard ratio 0.95 (0.80 to 1.12, p=0.53), with no benefit in the prespecified poor-prognosis subgroup either.\n\nOncoGenex, which developed it, is now Achieve Life Sciences and no longer works on cancer. The drug is a clean example of a biomarker association, serum clusterin and survival, that did not survive as a therapeutic target.","status":"withdrawn","asOf":"2026-09-25","links":[{"label":"SYNERGY (Lancet Oncology 2017)","url":"https://doi.org/10.1016/S1470-2045(17)30168-7"},{"label":"AFFINITY (Lancet Oncology 2017)","url":"https://doi.org/10.1016/S1470-2045(17)30605-8"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["antisense-sirna"],"targets":[],"drugs":[],"companies":["achieve-life-sciences"],"institutions":[],"pathways":[],"terms":["castration-resistance"],"trials":["synergy-custirsen","affinity-custirsen"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"OGX-011","modality":"antisense oligonucleotide","mechanism":"Second-generation antisense oligonucleotide that inhibits production of the chaperone protein clusterin.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cxbladder","kind":"drug","name":"Cxbladder","aka":[],"tldr":"A urine test that can rule out bladder cancer in people with blood in their urine, so fewer need a camera examination of the bladder.","summary":"Cxbladder (Pacific Edge, New Zealand) is a laboratory-developed urine test used in the work-up of haematuria (Detect and Triage) and in surveillance after treated non-muscle-invasive bladder cancer (Monitor), where its high negative predictive value lets low-risk patients skip some cystoscopies. It is included in US urology guidance for haematuria as an adjunct and has been used in New Zealand's public system and in US Veterans Affairs and Kaiser pathways. US Medicare coverage has been contested through the MolDX programme in 2024-25, which illustrates how reimbursement, more than regulation, decides which tests survive.","status":"established","asOf":"2026-09-10","links":[{"label":"Pacific Edge: Cxbladder","url":"https://www.cxbladder.com"}],"tags":["test"],"related":["urovysion"],"cancers":["urothelial"],"sections":[],"technologies":["cystoscopy-turbt","rna-seq"],"targets":[],"drugs":[],"companies":["pacific-edge"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: a negative Cxbladder in someone with low-risk haematuria makes bladder cancer very unlikely; a positive result leads to cystoscopy."],"brand":"Cxbladder Detect / Triage / Monitor","modality":"Urine mRNA biomarker test (bladder cancer detection and surveillance)","mechanism":"RT-qPCR of five mRNA markers (IGFBP5, HOXA13, MDK, CDK1, CXCR2) in voided urine, combined with clinical variables in the Triage version, to rule out urothelial carcinoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cyclophosphamide","kind":"drug","name":"Cyclophosphamide","aka":[],"tldr":"Cyclophosphamide is an alkylating chemotherapy that damages DNA in dividing cells. It is part of CHOP for lymphoma, AC for breast cancer and VAC for childhood sarcomas, clears lymphocytes before CAR-T and prevents graft-versus-host disease after transplant; bladder bleeding, infertility and secondary leukaemia are its harms.","summary":"Approved 1959. Backbone of CHOP/R-CHOP, AC/TC (breast), VDC/IE (Ewing), VAC (rhabdomyosarcoma), CVP, hyper-CVAD, EMA-CO; lymphodepletion before CAR-T; post-transplant cyclophosphamide for GVHD prophylaxis; low-dose metronomic use. Toxicities: myelosuppression, haemorrhagic cystitis (mesna), infertility, secondary leukaemia, cardiotoxicity at high dose.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Cyclophosphamide","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=cyclophosphamide"}],"tags":["gap-fill","generic","who-essential"],"related":["mesna"],"cancers":["dlbcl","breast-hr-positive","tnbc","ewing-sarcoma","rhabdomyosarcoma","neuroblastoma","all-leukemia","multiple-myeloma","pancreatic"],"sections":[],"technologies":["cytotoxic-chemotherapy","car-t","allogeneic-hsct"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["dbcg-82bc","nct06097364","nct04529772","nct06717347","nct06911502","nct06413498","nct06091865","nct06091254","nct06045806","nct05888493","nct05371093","nct03407144","nct04663347","nct04542824","nct06480565","nct04792489","nct06253663","nct07124936","nct03709680","nct05826535","nct05201248","nct07589543","nct04920617","nct07730515","nct07510815","nct07617753","nct03907852","nct07149857","nct07617805","nct06119685","nct07480928","nct05283720","nct04245839","nct03836352","nct06093841","nct07627711","nct04836507","nct06564038","nct07500220","nct07627698","nct07641036","nct04416984","nct03725059","nct07523529","nct07502287","nct05624554","nct05999994","nct06389006","nct06966700","nct06797635","nct06112379","nct05406401","nct07598955","nct04623541","nct06890884","nct05673785","nct04390399","nct06500273","nct06947967","nct06425302","nct07551362","nct06072612","eclipse"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cytoxan / Endoxan","modality":"Alkylating agent (oxazaphosphorine prodrug)","mechanism":"Activated by hepatic CYP2B6 to 4-hydroxycyclophosphamide/phosphoramide mustard, which cross-links DNA; acrolein by-product causes bladder toxicity.","approvals":[{"region":"US","year":1959,"indication":"Lymphomas, leukaemias, multiple myeloma, breast, ovarian, neuroblastoma, retinoblastoma and other cancers"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cyh33","kind":"drug","name":"CYH33","aka":[],"tldr":"CYH33 is a small-molecule inhibitor from Haihe Biopharma Co., Ltd., in registered phase 2 trials for ovarian cancer.","summary":"CYH33 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Haihe Biopharma Co., Ltd., in ovarian cancer. Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of CYH33","url":"https://clinicaltrials.gov/search?intr=CYH33"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["haihe-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05043922"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"CYH33","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"cytarabine","kind":"drug","name":"Cytarabine","aka":["Ara-C","Cytosine arabinoside","DepoCyt (liposomal, discontinued)"],"tldr":"Cytarabine is the core chemotherapy for acute myeloid leukaemia, given with an anthracycline as the classic 7+3 induction and at high doses for consolidation; it is also injected into the spinal fluid to treat or prevent leukaemia in the brain.","summary":"Cytarabine was approved in 1969 and remains, with an anthracycline, the remission-induction standard in acute non-lymphocytic leukaemia of adults and children; the label also covers acute lymphocytic leukaemia and blast-phase CML, and intrathecal use for prophylaxis and treatment of meningeal leukaemia. High-dose cytarabine consolidation (CALGB 8525) defined post-remission therapy for favourable-risk AML, and it partners fludarabine (FLAG), cladribine and midostaurin, gemtuzumab or venetoclax in modern regimens. A liposomal co-formulation with daunorubicin (CPX-351, Vyxeos) has its own record. Cerebellar toxicity at high doses, conjunctivitis (steroid eye drops) and profound myelosuppression are characteristic.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Cytarabine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=cytarabine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/cytarabine"}],"tags":["nci-list","generic"],"related":["cytarabine-7-3","cpx-351"],"cancers":["aml","all-leukemia","cml"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06788756","nct07007312","nct03591510","nct04229979","nct07255872","nct07387926","nct03182244","nct03793478"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cytosar-U (historic)","modality":"Pyrimidine nucleoside analogue (antimetabolite)","mechanism":"Converted to ara-CTP, which competitively inhibits DNA polymerase and is incorporated into DNA, halting chain elongation during S phase.","approvals":[{"region":"US","year":1969,"indication":"Remission induction in acute non-lymphocytic leukaemia; ALL; blast-phase CML; intrathecal therapy of meningeal leukaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1999-04-01","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Intrathecal treatment of lymphomatous meningitis"},{"date":"2007-04-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1999 converted to traditional approval 8.0 years after it was granted.","indication":"Intrathecal treatment of lymphomatous meningitis"}]},{"id":"cytarabine-7-3","kind":"drug","name":"Cytarabine + anthracycline ('7+3')","aka":[],"tldr":"The intensive chemotherapy that has induced remission in fit AML patients since 1973: seven days of one drug, three of another. Still the backbone that targeted drugs are added to.","summary":"Cytarabine 100-200 mg/m² continuous infusion for 7 days with daunorubicin 60-90 mg/m² or idarubicin 12 mg/m² for 3 days. Induces CR in 60-80% of younger adults. Modern practice adds midostaurin or quizartinib (FLT3), gemtuzumab (CD33+ favourable/intermediate risk), or uses CPX-351 (secondary AML). High-dose cytarabine consolidation follows. Five decades of incremental optimisation rather than replacement.","status":"standard-of-care","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Cytarabine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Cytarabine"}],"tags":[],"related":[],"cancers":["aml","all-leukemia"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["ratify","quantum-first","alfa-0701","aaml0531"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Cytotoxic regimen","mechanism":"Cytarabine is a pyrimidine analogue that terminates DNA chain elongation; anthracyclines intercalate DNA and poison topoisomerase II.","approvals":[{"region":"US","year":1969,"indication":"Cytarabine approved for acute leukaemia; 7+3 established 1973"}],"mechanismSteps":["Cytarabine is converted to ara-CTP inside blasts","ara-CTP competes with dCTP and is incorporated into DNA, halting replication","Daunorubicin intercalates DNA and traps topoisomerase II","Double-strand breaks trigger apoptosis in rapidly dividing blasts","Marrow empties (aplasia) then recovers with normal haematopoiesis if leukaemia is cleared"],"dosing":{"route":"IV","schedule":"Cytarabine 100-200 mg/m²/day continuous infusion days 1-7; daunorubicin 60-90 mg/m² or idarubicin 12 mg/m² days 1-3","monitoring":"Daily counts, infection prophylaxis, cardiac function (anthracycline cumulative dose)","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=cytarabine"},"toxicity":[{"event":"Profound myelosuppression","anyGradePct":100,"note":"Expected; 2-4 weeks aplasia"},{"event":"Febrile neutropenia / infection","anyGradePct":80},{"event":"Induction mortality","note":"~5% in younger fit adults, higher over 60"},{"event":"Anthracycline cardiotoxicity","note":"Cumulative-dose dependent"}],"access":[],"regulatoryEvents":[]},{"id":"d-0502","kind":"drug","name":"D-0502","aka":[],"tldr":"D-0502 is an experimental small-molecule drug from InventisBio in phase 3 trials for HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"D-0502 is a small-molecule drug developed by InventisBio. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. The sponsor states: An oral selective estrogen receptor degrader (SERD) being developed for ER-positive, HER2-negative locally advanced or metastatic breast cancer, currently in a Phase III registrational trial. ClinicalTrials.gov describes the intervention as: * Dosage form: Tablet * Administration route: Oral, once a day. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06954961 (A Study to Evaluate D-0502 in Subjects With ER+ Her2- Locally Advanced or Metastatic Breast Cancer), in HR-positive / HER2-negative breast cancer. The largest, NCT06954961, plans to enrol 640 participants with primary completion expected 2026-11-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of D-0502","url":"https://clinicaltrials.gov/search?intr=D-0502"},{"label":"Sponsor pipeline page","url":"https://www.inventisbio.com/en/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["inventisbio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06954961"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"An oral selective estrogen receptor degrader (SERD) being developed for ER-positive, HER2-negative locally advanced or metastatic breast cancer, currently in a Phase III registrational trial.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"d07001","kind":"drug","name":"D07001","aka":["D07001-softgel"],"tldr":"D07001 is an experimental small-molecule drug from InnoPharmax in phase 3 trials for biliary tract cancer, with its target not yet stated publicly.","summary":"D07001 is a small-molecule drug developed by InnoPharmax. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: D07001-softgel capsules: 3 times per week (on Days 1, 3, 5, 8, 10, 12, 15, 17, and 19 of a 21-day cycle, 9 doses per cycle). Xeloda (or TS-1): twice daily for 14 consecutive days followed by 7 days rest (1 treatment cycle). It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05065957 (Study of Combination Therapy of D07001-Softgel Capsules and Xeloda/TS-1 in Subjects With Advanced Biliary Tract Cancer) and NCT06622057 (D07001 Softgel-Capsules and Capecitabine Combination Therapy in Patients With Advanced Biliary Tract Cancer), in biliary tract cancer. The largest, NCT06622057, plans to enrol 240 participants with primary completion expected 2028-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of D07001","url":"https://clinicaltrials.gov/search?intr=D07001"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cholangiocarcinoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["innopharmax"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05065957","nct06622057"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dabrafenib","kind":"drug","name":"Dabrafenib","aka":[],"tldr":"Dabrafenib (Tafinlar) is a capsule that switches off the faulty BRAF protein driving some melanomas, lung, thyroid and other cancers. It is almost always paired with trametinib.","summary":"Dabrafenib was approved by the FDA in May 2013 as a single agent for unresectable or metastatic BRAF V600E melanoma (BREAK-3: progression-free survival superior to dacarbazine) and in 2014 in combination with trametinib, which became the standard after COMBI-d and COMBI-v showed longer overall survival than single-agent BRAF inhibition. Later indications, all with trametinib: adjuvant stage III BRAF-mutant melanoma (COMBI-AD, 2018), BRAF V600E metastatic NSCLC (2017), anaplastic thyroid cancer (2018), tumour-agnostic BRAF V600E solid tumours (2022) and paediatric low-grade glioma with a dispersible tablet formulation (2023). The EU authorised Tafinlar in 2013. Pyrexia is the signature toxicity of the combination; cutaneous squamous cell carcinomas and hyperglycaemia occur with monotherapy.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Dabrafenib","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=dabrafenib"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/dabrafenib"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/tafinlar"}],"tags":["nci-list"],"related":["dabrafenib-trametinib","braf-v600e"],"cancers":["melanoma","nsclc","thyroid","anaplastic-thyroid-cancer","papillary-thyroid-cancer","paediatric-low-grade-glioma","paediatric-high-grade-glioma","braf-v600e-nsclc","braf-v600-melanoma","stage-iii-melanoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["combi-ad","nct04940052","nct05585320"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tafinlar","modality":"Small-molecule BRAF kinase inhibitor","mechanism":"ATP-competitive inhibitor of BRAF V600E, V600K and V600D kinases (and wild-type BRAF and CRAF at higher concentrations); blocks MAPK signalling in BRAF-mutant tumours.","approvals":[{"region":"US","year":2013,"indication":"BRAF V600E unresectable or metastatic melanoma (single agent)"},{"region":"US","year":2014,"indication":"With trametinib: BRAF V600E/K metastatic melanoma; later adjuvant melanoma, BRAF V600E NSCLC, anaplastic thyroid cancer, tumour-agnostic solid tumours and paediatric low-grade glioma"},{"region":"EU","year":2013,"indication":"BRAF V600 melanoma; later NSCLC and adjuvant melanoma with trametinib"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2014-01-09","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with trametinib for unresectable or metastatic melanoma with BRAF V600E or V600K mutations, as detected by an FDA-approved test"},{"date":"2015-11-20","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2014 converted to traditional approval 1.9 years after it was granted.","indication":"In combination with trametinib for unresectable or metastatic melanoma with BRAF V600E or V600K mutations, as detected by an FDA-approved test"},{"date":"2022-06-22","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 4.2 years later, when the FDA's table was read.","source":"https://www.fda.gov#endnote2","indication":"In combination with trametinib for adult and pediatric patients 6 years of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options. *"}]},{"id":"dabrafenib-trametinib","kind":"drug","name":"Dabrafenib + trametinib","aka":[],"tldr":"Dabrafenib plus trametinib is the BRAF-plus-MEK pill combination, approved for BRAF V600E lung cancer and, since 2022, for any solid tumour with that mutation.","summary":"Dabrafenib blocks BRAF V600 and trametinib is an allosteric MEK1/2 inhibitor; blocking two consecutive steps of the MAPK pathway (vertical blockade) prevents the paradoxical reactivation seen with BRAF inhibitors alone. In BRAF V600E non-small-cell lung cancer (about 2% of adenocarcinomas) the combination gave an ORR of 64% first line with median PFS of about 10.8 months (BRF113928), and it is an established doublet in BRAF-mutant melanoma and thyroid cancer. A tumour-agnostic approval followed in June 2022 for BRAF V600E solid tumours (excluding colorectal) in patients aged 6 and over. Pyrexia is the characteristic toxicity and the usual reason for dose interruptions. Encorafenib plus binimetinib (PHAROS, 2023) is the alternative doublet in lung cancer. In short, if a tumour carries BRAF V600E, this pair of pills is a treatment option in most tissue types.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Dabrafenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Dabrafenib"},{"label":"NICE TA898: dabrafenib plus trametinib for BRAF V600 mutation-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta898"}],"tags":[],"related":["braf-plus-mek","braf-v600e"],"cancers":["nsclc","melanoma","thyroid","paediatric-low-grade-glioma","paediatric-high-grade-glioma","secondary-brain-tumours","braf-v600e-nsclc","braf-v600-melanoma","stage-iii-melanoma","advanced-melanoma","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":["ras-mapk"],"terms":["tumour-agnostic"],"trials":["brf113928","pharos"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tafinlar + Mekinist","modality":"Small-molecule kinase inhibitors (BRAF + MEK)","mechanism":"BRAF V600 inhibitor plus allosteric MEK1/2 inhibitor; vertical blockade prevents paradoxical MAPK reactivation.","approvals":[{"region":"US","year":2014,"indication":"BRAF V600E/K unresectable or metastatic melanoma, combination (accelerated; each drug alone approved 2013)"},{"region":"US","year":2017,"indication":"BRAF V600E metastatic NSCLC"},{"region":"US","year":2022,"indication":"BRAF V600E solid tumours, tumour-agnostic (age ≥6)"},{"region":"England (NICE)","year":2023,"indication":"BRAF V600 mutation-positive advanced non-small-cell lung cancer, first line only","note":"TA898, published 14 June 2023, recommends the combination only as first-line treatment of advanced disease, subject to the commercial arrangement. A patient who has already had chemotherapy or immunotherapy is not funded for it."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dacarbazine","kind":"drug","name":"Dacarbazine","aka":["DTIC"],"tldr":"Dacarbazine is an older chemotherapy infusion. It was the standard treatment for advanced melanoma for decades and is the D in the ABVD regimen that cures most Hodgkin lymphoma.","summary":"Dacarbazine was approved in 1975 for metastatic malignant melanoma and as second-line therapy with other agents for Hodgkin disease. In melanoma it produced responses in roughly one patient in ten and was the control arm in the trials that established ipilimumab, vemurafenib and dabrafenib, after which it left routine use. In Hodgkin lymphoma it remains essential as part of ABVD (doxorubicin, bleomycin, vinblastine, dacarbazine) and its brentuximab vedotin variant A+AVD. It is strongly emetogenic, causes a flu-like syndrome and photosensitivity, and is hepatotoxic, with rare hepatic vein thrombosis.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Dacarbazine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=dacarbazine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/dacarbazine"}],"tags":["nci-list","generic"],"related":["temozolomide"],"cancers":["melanoma","hodgkin-lymphoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct03016819","nct05987332","nct06703398","nct03407144","nct04733183","nct07310784","nct02979522"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"DTIC-Dome (historic)","modality":"Alkylating agent (triazene)","mechanism":"Hepatically activated to the methylating species MTIC, which methylates DNA at O6- and N7-guanine; the same active metabolite as temozolomide.","approvals":[{"region":"US","year":1975,"indication":"Metastatic malignant melanoma; Hodgkin disease (second line, combination)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dacomitinib","kind":"drug","name":"Dacomitinib","aka":[],"tldr":"A second-generation EGFR pill that beat gefitinib on survival in EGFR-mutant lung cancer but was quickly overshadowed by osimertinib.","summary":"Dacomitinib is a second-generation, irreversible pan-ErbB tyrosine kinase inhibitor that blocks EGFR, HER2 and HER4. In the ARCHER 1050 trial (2017 to 2018) it beat gefitinib in first-line EGFR-mutant NSCLC, with median progression-free survival of 14.7 versus 9.2 months and overall survival of 34.1 versus 26.8 months, although patients with brain metastases were excluded. It was approved in the US in 2018 and the EU in 2019 for first-line metastatic NSCLC with exon 19 deletion or L858R. Higher rates of rash, diarrhoea and paronychia mean most patients need dose reductions, which appear not to compromise efficacy. It was quickly overshadowed by osimertinib, which has CNS activity and better tolerability, so it is now seldom used. Dacomitinib remains one of the few EGFR inhibitors to have shown an overall survival gain over another EGFR inhibitor.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Dacomitinib","links":[{"label":"ARCHER 1050 (Lancet Oncol 2017)","url":"https://doi.org/10.1016/S1470-2045(17)30608-3"},{"label":"ARCHER 1050 overall survival (JCO 2018)","url":"https://doi.org/10.1200/JCO.2018.78.7994"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=dacomitinib"},{"label":"NICE TA595: dacomitinib for untreated EGFR mutation-positive non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta595"}],"tags":["gap-fill"],"related":["egfr-exon-19-deletion"],"cancers":["nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01774721","nct01360554","nct06486142"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vizimpro","modality":"Small-molecule pan-ErbB TKI (second generation, irreversible)","mechanism":"Irreversible inhibitor of EGFR, HER2 and HER4.","approvals":[{"region":"US","year":2018,"indication":"First-line metastatic NSCLC with EGFR exon 19 del or L858R"},{"region":"EU","year":2019,"indication":"Same"},{"region":"England (NICE)","year":2019,"indication":"Untreated locally advanced or metastatic EGFR mutation-positive non-small-cell lung cancer","note":"TA595, published 14 August 2019, subject to the commercial arrangement (ARCHER 1050)."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dactinomycin","kind":"drug","name":"Dactinomycin (actinomycin D)","aka":["Actinomycin D","Actinomycin"],"tldr":"The first antibiotic used as an anticancer drug (1954) and still the core of chemotherapy for Wilms tumour, rhabdomyosarcoma and gestational trophoblastic disease.","summary":"Dactinomycin (actinomycin D) intercalates into GC-rich DNA and blocks RNA polymerase elongation, halting transcription; it also sensitises tissue to radiation. It was the first antibiotic used as an anticancer drug in 1954, and Sidney Farber's Wilms tumour work in the 1950s established it before US approval in 1964. It remains central to paediatric oncology: in VA and DD-4A for Wilms tumour, in VAC and IVA for rhabdomyosarcoma, historically in Ewing sarcoma regimens, and in EMA-CO and pulsed single-agent therapy for gestational trophoblastic neoplasia, one of the most curable cancers. It is a vesicant, can cause sinusoidal obstruction syndrome of the liver in young children, and can provoke radiation recall in previously irradiated skin. Dactinomycin is a seventy-year-old drug that still cures children.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Dactinomycin","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=dactinomycin"}],"tags":["gap-fill","generic","who-essential"],"related":[],"cancers":["wilms-tumor","rhabdomyosarcoma","gestational-trophoblastic","ewing-sarcoma","testicular","low-risk-gtn","high-risk-gtn"],"sections":[],"technologies":["cytotoxic-chemotherapy","isolated-limb-perfusion"],"targets":[],"drugs":[],"companies":["recordati","nobelpharma"],"institutions":[],"pathways":[],"terms":["re-irradiation"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cosmegen","modality":"Actinomycin antibiotic (transcription inhibitor)","mechanism":"Intercalates GC-rich DNA and blocks RNA polymerase elongation; potent radiosensitiser.","approvals":[{"region":"US","year":1964,"indication":"Wilms tumour, rhabdomyosarcoma, Ewing sarcoma, gestational trophoblastic neoplasia, metastatic testicular cancer, regional perfusion for sarcoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dalpiciclib","kind":"drug","name":"Dalpiciclib","aka":[],"tldr":"Dalpiciclib is Hengrui's CDK4/6 inhibitor, the first China-developed drug of the class, approved for hormone-driven advanced breast cancer on the DAWNA trials.","summary":"DAWNA-1 (361 patients, Nature Medicine 2021) showed median progression-free survival of 15.7 versus 7.2 months when dalpiciclib was added to fulvestrant after endocrine therapy (hazard ratio 0.42), the basis of NMPA approval in December 2021; DAWNA-2 with an aromatase inhibitor in the first line followed in 2023. Neutropenia is frequent, as with palbociclib. It gives Chinese patients a reimbursed domestic alternative to imported CDK4/6 inhibitors.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Dalpiciclib","links":[{"label":"Jiangsu Hengrui Pharmaceuticals","url":"https://www.hengrui.com/en/"},{"label":"DAWNA-1 (Nat Med 2021)","url":"https://doi.org/10.1038/s41591-021-01562-9"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["cdk46-inhibitor","kinase-inhibitors"],"targets":["cdk4-6"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":["p53-cell-cycle"],"terms":[],"trials":["dawna-1","nct04842617","nct06167694"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Airuikang","code":"SHR6390","modality":"Small-molecule CDK4/6 inhibitor","mechanism":"Selective inhibitor of cyclin-dependent kinases 4 and 6, preventing RB phosphorylation and G1 to S progression in hormone receptor-positive breast cancer.","approvals":[{"region":"China","year":2021,"indication":"HR-positive, HER2-negative advanced breast cancer with fulvestrant after endocrine therapy (DAWNA-1)"},{"region":"China","year":2023,"indication":"First-line HR-positive, HER2-negative advanced breast cancer with an aromatase inhibitor (DAWNA-2)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"danazol","kind":"drug","name":"Danazol","aka":["Danol"],"tldr":"Danazol is an old hormone tablet, licensed for endometriosis, that can raise haemoglobin and platelets in myelofibrosis and bone marrow failure. It was the comparison treatment that momelotinib beat in the MOMENTUM trial.","summary":"Danazol is a synthetic androgen licensed since the 1970s for endometriosis, fibrocystic breast disease and hereditary angioedema. In haematology it is used off-label for the anaemia of myelofibrosis and myelodysplastic syndromes and for immune and aplastic cytopenias, where about a third of patients improve; it also lengthens telomeres in telomere-biology disorders.\n\nIn the phase 3 MOMENTUM trial in symptomatic, anaemic myelofibrosis previously treated with a JAK inhibitor, danazol was the active comparator: 25 percent of patients on momelotinib had their symptom score halve against 9 percent on danazol, and more became transfusion independent. Liver toxicity, virilisation and thrombosis limit its use. The primary myelofibrosis page names it among the treatments for anaemia.","status":"established","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Danazol","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Danazol"}],"tags":["subtype-drugs-wave"],"related":["momelotinib"],"cancers":["primary-myelofibrosis"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Danocrine","modality":"Oral synthetic androgen (hormonal agent)","mechanism":"A weak androgen derived from ethisterone that stimulates erythropoiesis and thrombopoiesis, in part by raising telomerase activity in haematopoietic cells, and suppresses gonadotropins.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"daratumumab","kind":"drug","name":"Daratumumab","aka":[],"tldr":"The CD38 antibody that turned triplets into quadruplets: adding it to standard induction roughly halves the risk of myeloma progressing.","summary":"First approved 2015 (monotherapy after ≥3 lines); now in frontline quadruplets: Dara-VRd (PERSEUS, transplant-eligible; CEPHEUS, transplant-ineligible), Dara-Rd (MAIA, OS benefit), Dara-VMP (ALCYONE). Subcutaneous Faspro (2020) gives a 5-minute injection. Approved in high-risk smouldering myeloma (AQUILA, 2025-26) and with teclistamab (MajesTEC-3, March 2026). Also used in AL amyloidosis. J&J/Genmab.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Daratumumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Daratumumab"}],"tags":[],"related":[],"cancers":["multiple-myeloma","smouldering-myeloma","myeloma-transplant-eligible","myeloma-transplant-ineligible","plasma-cell-leukaemia"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd38"],"drugs":[],"companies":["johnson-johnson","genmab","halozyme"],"institutions":[],"pathways":[],"terms":["adcc"],"trials":["perseus","cepheus","majestec-3","nct06868654","nct06952478","nct06464991","nct05438043","nct05623020","nct04975997","nct05020236","nct07095452","nct06679101","nct06868667","nct05552222","nct05455320","nct07393282","nct02343042","nct03314181","nct04133636","nct03989414","nct06577025","nct04782687","nct06892522","nct06119685","nct06953960","nct07681596","nct04973605","nct02773030","nct07742215"],"people":[],"bottlenecks":[],"keyPapers":["paper-aquila-daratumumab-smouldering-nejm-2025","paper-cassiopeia-lancet-2019","paper-maia-daratumumab-rd-nejm-2019"],"journals":[],"dependsOn":[],"notes":[],"brand":"Darzalex / Darzalex Faspro (SC)","modality":"Monoclonal antibody (anti-CD38)","mechanism":"Human IgG1κ anti-CD38: CDC, ADCC, ADCP, apoptosis via Fc crosslinking, and depletion of CD38+ regulatory T and B cells.","approvals":[{"region":"US","year":2015,"indication":"Relapsed myeloma after ≥3 lines"},{"region":"US","year":2019,"indication":"Newly diagnosed, transplant-ineligible with Rd (MAIA) and VMP"},{"region":"US","year":2024,"indication":"Newly diagnosed transplant-eligible with VRd (PERSEUS)"},{"region":"US","year":2026,"indication":"With teclistamab after ≥1 line (MajesTEC-3); high-risk smouldering myeloma (AQUILA)"}],"mechanismSteps":["Binds CD38 on the plasma cell surface","Recruits complement (CDC) and Fc-receptor effector cells (ADCC, ADCP)","Crosslinking triggers direct apoptosis","Depletes CD38-high immunosuppressive cells, freeing T cells"],"dosing":{"route":"Subcutaneous 1,800 mg (or IV 16 mg/kg)","schedule":"Weekly × 8, every 2 weeks × 8, then every 4 weeks","monitoring":"Injection/infusion reactions (first dose), infections, interference with blood typing and M-protein assays, HBV reactivation"},"toxicity":[{"event":"Infusion/injection reaction","anyGradePct":13,"grade3PlusPct":1,"note":"subcutaneous (COLUMBA)"},{"event":"Neutropenia (with Rd)","grade3PlusPct":50,"note":"MAIA"},{"event":"Pneumonia","grade3PlusPct":14,"note":"MAIA"}],"access":[],"regulatoryEvents":[{"date":"2015-11-16","type":"accelerated-approval","region":"US","note":"First CD38 antibody","indication":"Multiple myeloma after at least 3 prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or double refractory to a proteasome inhibitor and an immunomodulatory agent"},{"date":"2016-11-21","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2015 converted to traditional approval 1.0 year after it was granted.","indication":"Multiple myeloma after at least 3 prior lines of therapy including a proteasome inhibitor and an immunomodulatory agent or double refractory to a proteasome inhibitor and an immunomodulatory agent"},{"date":"2020-05-01","type":"approval","region":"US","note":"Subcutaneous Darzalex Faspro"},{"date":"2021-01-15","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-darzalex-faspro-newly-diagnosed-light-chain-amyloidosis","indication":"Treatment of patients with light chain (AL) amyloidosis in combination with bortezomib, cyclophosphamide and dexamethasone in newly diagnosed patients"},{"date":"2024-07-30","type":"approval","region":"US","note":"Dara-VRd induction/consolidation (PERSEUS)"},{"date":"2025-11-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2021 converted to traditional approval 4.8 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-darzalex-faspro-newly-diagnosed-light-chain-amyloidosis","indication":"Treatment of patients with light chain (AL) amyloidosis in combination with bortezomib, cyclophosphamide and dexamethasone in newly diagnosed patients"},{"date":"2026-03-05","type":"approval","region":"US","note":"Teclistamab + daratumumab after ≥1 prior line","source":"https://www.jnj.com/media-center/press-releases/johnson-johnson-announces-u-s-fda-approval-of-tecvayli-plus-darzalex-faspro-for-relapsed-refractory-multiple-myeloma-offering-a-potential-new-standard-of-care-as-early-as-second-line"},{"date":"2026-08-13","type":"approval","region":"US","note":"FDA grants accelerated approval to iberdomide with daratumumab and hyaluronidase-fihj and dexamethasone for multiple myeloma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-iberdomide-daratumumab-and-hyaluronidase-fihj-and-dexamethasone"}]},{"id":"daraxonrasib","kind":"drug","name":"Daraxonrasib","aka":[],"tldr":"The first drug that blocks all active RAS variants, approved by the FDA in August 2026 for metastatic pancreatic cancer, where KRAS drives 90% of tumours.","summary":"Daraxonrasib is Revolution Medicines' tri-complex inhibitor. Phase 1/2: median OS ~14.5 months in second-line pancreatic cancer versus historical ~6 months. RASolute 302 (second-line PDAC) supported FDA approval on 26 August 2026 as Rasonque for metastatic pancreatic cancer; RASolute 303 (first-line) and the NSCLC phase 3 (RASolve 301) continue. Rash and stomatitis are the main toxicities.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA approval notice","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-daraxonrasib-metastatic-pancreatic-adenocarcinoma"},{"label":"FDA approval letter, NDA 220910","url":"https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2026/220910Orig1s000ltr.pdf"},{"label":"FDA prescribing information (label)","url":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/220910Orig1s000lbl.pdf"},{"label":"FDA novel drug approvals 2026","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2026"},{"label":"Rasonque label (openFDA): indication, RASolute 302 results and adverse reactions","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22RASONQUE%22"},{"label":"FDA notice: daraxonrasib for metastatic pancreatic adenocarcinoma (26 August 2026)","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"label":"O'Reilly et al., daraxonrasib or chemotherapy in previously treated metastatic pancreatic cancer, RASolute 302 (NEJM 2026)","url":"https://doi.org/10.1056/NEJMoa2605555"},{"label":"Wolpin et al., daraxonrasib in previously treated advanced RAS-mutated pancreatic cancer, phase 1 (NEJM 2026)","url":"https://doi.org/10.1056/NEJMoa2505783"}],"tags":[],"related":[],"cancers":["pancreatic","metastatic-pdac","kras-g12c-pdac","nsclc","colorectal"],"sections":[],"technologies":["kras-inhibitors"],"targets":["kras"],"drugs":[],"companies":["revolution-medicines"],"institutions":[],"pathways":[],"terms":[],"trials":["rasolute-302","nct07491445","nct07805954","nct07252232","nct06881784","nct06445062","nct07397338","nct06922591","nct05379985","nct06162221","nct06128551","nct07492680","codebreak-100"],"people":[],"bottlenecks":[],"keyPapers":["paper-daraxonrasib-pancreatic-n-engl-j-med-2026"],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer, regulators: the Rasonque label (effective 27 August 2026) indicates daraxonrasib 300 mg once daily for metastatic pancreatic adenocarcinoma after at least one prior systemic therapy or in adults who are not candidates for multi-agent chemotherapy, on RASolute 302 (overall population: overall survival 13.2 against 6.7 months, hazard ratio 0.40; progression-free survival by blinded review 7.2 against 3.6 months, hazard ratio 0.49; response 30 against 11 percent; RAS G12 population 13.2 against 6.6 months). Label safety (241 treated): serious adverse reactions 30 percent, discontinuation 2.9 percent, dose interruption 69 percent (rash 27, stomatitis 20 percent), dose reduction 37 percent. No EMA authorisation and no NICE appraisal were listed on 24 September 2026; RASolute 303 (first line) recruits at six UK hospitals and RASolute 304 (adjuvant) at six.","Pancreatic ductal adenocarcinoma biomarkers: RASolute 302 enrolled on RAS G12, G13 or Q61 mutation or no identified RAS mutation rather than on one allele; 91.8% of the 500 patients carried a G12 allele and the hazard ratio for death was 0.40 in both the G12 and the overall population, so the practical threshold is a RAS-mutant or RAS-unknown tumour rather than a specific variant (O'Reilly 2026)."],"brand":"Rasonque","code":"RMC-6236","modality":"Small-molecule pan-RAS(ON) inhibitor","mechanism":"Binds cyclophilin A to form a tri-complex that sterically blocks RAS(ON) from engaging effectors, across G12X/G13X/Q61X and wild-type RAS.","approvals":[{"region":"US","year":2026,"indication":"Metastatic pancreatic adenocarcinoma after at least one prior systemic therapy, or in adults who are not candidates for multiagent systemic therapy","note":"Rasonque, NDA 220910, 26 August 2026"}],"mechanismSteps":["Drug binds cyclophilin A inside the cell","The drug-cyclophilin complex binds the active (GTP-bound) RAS protein, forming a tri-complex","RAS(ON) is sterically blocked from engaging RAF and other effectors, regardless of which mutation it carries","MAPK signalling collapses in RAS-addicted cells","Pancreatic and other RAS-driven tumour cells arrest and die"],"dosing":{"route":"Oral","schedule":"300 mg once daily (phase 3 dose; 200 mg in some cohorts)","modifications":"Reduce for grade 3 rash or stomatitis","monitoring":"Skin, oral mucosa, LFTs","source":"https://clinicaltrials.gov/study/NCT06625320"},"toxicity":[{"event":"Rash","note":"Most common; mostly grade 1-2 per phase 1/2 reports"},{"event":"Stomatitis"},{"event":"Nausea"},{"event":"Diarrhoea"},{"event":"Fatigue"}],"access":[{"country":"US","reimbursement":"FDA-approved 26 August 2026 (Rasonque, NDA 220910), oral tablets; coverage decisions and list price not yet recorded","source":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=220910","asOf":"2026-09-18"}],"regulatoryEvents":[{"date":"2024-06","type":"designation","region":"US","note":"Breakthrough Therapy designation, previously treated metastatic PDAC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-07-17","type":"filing","region":"US","note":"NDA 220910 received by the FDA on the RASolute 302 result","source":"https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2026/220910Orig1s000ltr.pdf"},{"date":"2026-08-26","type":"approval","region":"US","note":"NDA 220910 approved as Rasonque: adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy or who are not candidates for multiagent systemic therapy","source":"https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2026/220910Orig1s000ltr.pdf"}]},{"id":"darbepoetin-alfa","kind":"drug","name":"Darbepoetin alfa","aka":[],"tldr":"Darbepoetin alfa (Aranesp) is a longer-acting version of erythropoietin given every one to three weeks to treat anaemia caused by chemotherapy, reducing transfusions but with the same warnings about clots and tumour growth as epoetin.","summary":"Darbepoetin alfa was approved by the FDA in September 2001 for anaemia of chronic kidney disease and in July 2002 for anaemia due to concomitant myelosuppressive chemotherapy in non-myeloid malignancies with at least two further months of planned chemotherapy; the EU authorised Aranesp in June 2001. Weekly or three-weekly dosing suited chemotherapy schedules and it displaced much epoetin use in oncology before the survival and thrombosis findings of the 2000s (including the darbepoetin trial in anaemic cancer patients not on chemotherapy, which showed excess mortality) restricted all erythropoiesis-stimulating agents to the lowest dose needed to avoid transfusion in the palliative setting. The label states it has not been shown to improve quality of life or fatigue. Hypertension, thromboembolism and rare pure red cell aplasia are the main risks.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Darbepoetin_alfa","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=darbepoetin%20alfa"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/darbepoetinalfa"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/aranesp"}],"tags":["nci-list","supportive"],"related":["epoetin-alfa"],"cancers":[],"sections":[],"technologies":["transfusion-support"],"targets":[],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00261313","nct00077311","nct02890602"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aranesp","modality":"Hyperglycosylated recombinant erythropoietin (erythropoiesis-stimulating agent)","supportive":true,"mechanism":"Erythropoietin analogue with two additional N-linked carbohydrate chains that triple its half-life; stimulates the erythropoietin receptor to increase red cell production.","approvals":[{"region":"US","year":2001,"indication":"Anaemia of chronic kidney disease"},{"region":"US","year":2002,"indication":"Anaemia due to concomitant myelosuppressive chemotherapy in non-myeloid malignancies"},{"region":"EU","year":2001,"indication":"Anaemia of chronic renal failure; symptomatic anaemia in adults with non-myeloid malignancies receiving chemotherapy (Aranesp)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"darolutamide","kind":"drug","name":"Darolutamide","aka":[],"tldr":"Darolutamide is an AR blocker that barely enters the brain, so it causes fewer falls and cognitive side effects; it is approved with and without chemotherapy.","summary":"Darolutamide is a structurally distinct androgen receptor antagonist with low blood-brain-barrier penetration and activity against the AR F877L resistance mutation. Because little drug reaches the brain it causes fewer falls, fatigue and cognitive side effects than enzalutamide, which matters for older men and those on many medicines. ARAMIS (nmCRPC) showed an overall survival benefit; ARASENS proved triplet therapy with ADT plus docetaxel in mHSPC (OS HR 0.68, 4-year OS 62.7% versus 50.4%); ARANOTE supported the 2025 approval with ADT alone in mHSPC. Whether all men with metastatic hormone-sensitive disease need the chemotherapy component, or only those with high-volume disease, is still argued. For a newcomer, darolutamide is the AR blocker that barely enters the brain, approved with and without chemotherapy.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Darolutamide","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Darolutamide"},{"label":"NICE TA660: darolutamide with androgen deprivation therapy for treating hormone-relapsed non-metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta660"},{"label":"NICE TA903: darolutamide with androgen deprivation therapy and docetaxel for treating hormone-sensitive metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta903"},{"label":"NICE TA1109: darolutamide with androgen deprivation therapy for treating hormone-sensitive metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta1109"}],"tags":[],"related":[],"cancers":["prostate","prostate-nmcrpc","prostate-mhspc"],"sections":[],"technologies":["androgen-deprivation"],"targets":["androgen-receptor"],"drugs":[],"companies":["bayer"],"institutions":[],"pathways":["ar-signaling"],"terms":["mcrpc-mhspc","prostate-uk-drug-approvals"],"trials":["arasens","nct07611110","nct05794906","nct06785636","nct05367440","nct07450599","nct06568094","nct06190899","nct07190300","nct04464226"],"people":[],"bottlenecks":[],"keyPapers":["paper-aramis-nejm-2019"],"journals":[],"dependsOn":[],"notes":["Three NICE appraisals cover darolutamide in England. TA660 recommends it with androgen deprivation for hormone-relapsed prostate cancer at high risk of metastasis. TA903 recommends it with androgen deprivation and docetaxel for hormone-sensitive metastatic prostate cancer. TA1109 recommends it with androgen deprivation alone for hormone-sensitive metastatic prostate cancer only if docetaxel is not suitable, and adds an explicit instruction to use the least expensive of the suitable options, including apalutamide with androgen deprivation."],"brand":"Nubeqa","modality":"Small-molecule AR antagonist","mechanism":"Structurally distinct AR antagonist, low blood-brain-barrier penetration, active against AR F877L.","approvals":[{"region":"US","year":2019,"indication":"Non-metastatic CRPC"},{"region":"US","year":2022,"indication":"mHSPC with docetaxel (ARASENS)"},{"region":"US","year":2025,"indication":"mHSPC with ADT alone (ARANOTE)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"daromun","kind":"drug","name":"Daromun","aka":["L19IL2 + L19TNF","Onfekafusp alfa and bifikafusp alfa","Darleukin + Fibromun"],"tldr":"Daromun is a pair of antibody-linked immune messengers injected straight into melanoma deposits before surgery. In a phase 3 trial it lowered the chance of the melanoma coming back after the operation compared with surgery alone.","summary":"Daromun is Philogen's combination of two immunocytokines, L19IL2 (onfekafusp alfa) and L19TNF (bifikafusp alfa). Both use the human antibody L19, which targets the extra-domain B of fibronectin found in new tumour blood vessels, to concentrate interleukin-2 and tumour necrosis factor in the tumour after direct injection into skin and lymph node deposits. Local delivery gives the immune stimulation of these cytokines without the toxicity of giving them systemically.\n\nIn the randomised phase 3 PIVOTAL trial in resectable stage III melanoma with injectable deposits, four weekly intralesional treatments before surgery improved relapse-free survival compared with surgery alone. Philogen has filed for European approval on this result. The stage III melanoma page names it beside talimogene laherparepvec as an intralesional option before excision.","status":"phase-3","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["talimogene-laherparepvec"],"cancers":["stage-iii-melanoma"],"sections":[],"technologies":["immunocytokines"],"targets":[],"drugs":[],"companies":["philogen-s-p-a"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Intralesional immunocytokine combination","mechanism":"Two antibody-cytokine fusion proteins that use the L19 antibody, which binds the EDB domain of fibronectin in tumour blood vessels, to hold interleukin-2 and tumour necrosis factor inside the injected melanoma deposit, where they recruit and activate T cells and destroy tumour vasculature.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"darovasertib","kind":"drug","name":"Darovasertib","aka":["IDE 196"],"tldr":"Darovasertib is an experimental small-molecule drug from IDEAYA Biosciences in phase 3 trials for uveal melanoma and melanoma, with its target not yet stated publicly.","summary":"Darovasertib (IDE196, LXS196) is a small-molecule drug developed by IDEAYA Biosciences. The sponsor describes its target as PKC (protein kinase C), which OnCo does not yet have a target page for. The sponsor states: Darovasertib is a small-molecule PKC inhibitor targeting the oncogenic driver of uveal melanoma associated with GNAQ/GNA11 mutations or PRKC fusions. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07804186 (Study of Adjuvant Darovasertib and Crizotinib in Participants With Primary Non-metastatic Uveal Melanoma) and NCT07015190 (Neoadjuvant Darovasertib in Primary Uveal Melanoma), in uveal melanoma and melanoma. The largest, NCT07015190, plans to enrol 520 participants with primary completion expected 2030-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Darovasertib","url":"https://clinicaltrials.gov/search?intr=IDE196"},{"label":"Sponsor pipeline page","url":"https://ideayabio.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["uveal-melanoma","melanoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["ideaya-biosciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07804186","nct07015190","nct05987332","nct03947385","nct05907954"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"IDE196, LXS196","modality":"small molecule","mechanism":"Darovasertib is a small-molecule PKC inhibitor targeting the oncogenic driver of uveal melanoma associated with GNAQ/GNA11 mutations or PRKC fusions.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dasatinib","kind":"drug","name":"Dasatinib","aka":[],"tldr":"Dasatinib is a second-generation BCR::ABL1 pill that, combined with the immunotherapy blinatumomab, can put Ph-positive ALL into deep remission with no chemotherapy at all.","summary":"Approved in CML (2006) and Ph+ ALL (resistant, 2006; paediatric frontline with chemotherapy 2018). The GIMEMA D-ALBA trial (dasatinib induction then blinatumomab) achieved 18-month OS 95% and DFS 88% in adults, launching the chemotherapy-free paradigm; long-term follow-up shows ~75% DFS at 4 years, with IKZF1-plus and T315I as the relapse drivers. Generic since 2024.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Dasatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Dasatinib"}],"tags":[],"related":[],"cancers":["all-leukemia","all-paediatric-ph-positive","all-ph-like","cml-chronic-phase"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["bcr-abl","ddr2","abl1"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":["ph-positive-all"],"trials":["d-alba","nct04971226"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Sprycel","modality":"Small-molecule kinase inhibitor (BCR::ABL1, SRC)","mechanism":"Dual SRC/ABL inhibitor binding active and inactive ABL1 conformations; inactive against T315I.","approvals":[{"region":"US","year":2006,"indication":"CML and Ph+ ALL resistant or intolerant to imatinib"},{"region":"US","year":2018,"indication":"Paediatric Ph+ ALL with chemotherapy"}],"mechanismSteps":["Dasatinib blocks BCR::ABL1 and SRC-family kinases in lymphoblasts","Proliferation and survival signalling stop; blasts clear within weeks","Blinatumomab then recruits T cells to kill residual MRD-level CD19+ cells","Transplant reserved for MRD persistence or high-risk genetics"],"dosing":{"route":"Oral","schedule":"Ph+ ALL: 140 mg once daily (adult); paediatric weight-based; continue through consolidation and maintenance","monitoring":"Pleural effusion, myelosuppression, pulmonary arterial hypertension, QT","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Sprycel"},"toxicity":[{"event":"Pleural effusion","anyGradePct":28,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Sprycel","note":"CML long-term data"},{"event":"Myelosuppression","grade3PlusPct":50,"note":"Higher in ALL with chemotherapy"},{"event":"Pulmonary arterial hypertension","note":"Rare, reversible on stopping"}],"access":[],"regulatoryEvents":[{"date":"2006-06-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Chronic myeloid leukemia with resistance or intolerance to prior therapy including imatinib"},{"date":"2009-05-21","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2006 converted to traditional approval 2.9 years after it was granted.","indication":"Chronic myeloid leukemia with resistance or intolerance to prior therapy including imatinib"},{"date":"2010-10-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Newly diagnosed adults with Philadelphia chromosome-positive CML in chronic phase"},{"date":"2015-08-12","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2010 converted to traditional approval 4.8 years after it was granted.","indication":"Newly diagnosed adults with Philadelphia chromosome-positive CML in chronic phase"}]},{"id":"datopotamab-deruxtecan","kind":"drug","name":"Datopotamab deruxtecan","aka":[],"tldr":"Datopotamab deruxtecan (Datroway) is the second TROP2 ADC and shares Enhertu's payload. In 2026 it became a first-line option for triple-negative breast cancer patients who cannot receive immunotherapy.","summary":"Approved January 2025 for HR+/HER2- metastatic breast cancer after endocrine and chemotherapy (TROPION-Breast01) and June 2025 for EGFR-mutant NSCLC after TKI and chemotherapy (TROPION-Lung05). In Q2 2026 approved for first-line unresectable/metastatic TNBC in patients ineligible for PD-1/PD-L1 inhibitors based on TROPION-Breast02 (OS 23.7 vs 18.7 months per patient-facing summaries). Lower DAR than T-DXd; stomatitis and ocular surface events are characteristic; ILD occurs. TROPION-Breast05 tests it with durvalumab in PD-L1+ TNBC.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Datopotamab_deruxtecan","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Datopotamab%20deruxtecan"},{"label":"Datroway label (openFDA): TNBC indication and TROPION-Breast02 study section","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22DATROWAY%22"},{"label":"EMA Datroway product page (HR-positive indication only)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/datroway"}],"tags":[],"related":[],"cancers":["tnbc","breast-hr-positive","nsclc","tnbc-metastatic","egfr-mutant-nsclc","pdl1-high-nsclc"],"sections":[],"technologies":["adc","topoisomerase-inhibitors"],"targets":["trop2"],"drugs":[],"companies":["daiichi-sankyo","astrazeneca"],"institutions":[],"pathways":[],"terms":["ggfg"],"trials":["tropion-breast01","tropion-breast02","tropion-breast05","tropion-lung01","nct06350097","nct06564844","nct06417814","nct05215340","nct05555732","nct06357533","nct07291037","nct07098338","nct04644068","nct05061550","nct05489211","nct05687266","nct06130553","nct05417594","nct05460273","nct03742102","diamond","nct06112379"],"people":[],"bottlenecks":[],"keyPapers":["paper-tropion-breast01-china-cohort-esmo-open-2026"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer biomarkers: TROPION-Breast02 enrolled first-line patients not candidates for PD-1/PD-L1 inhibitors, in practice PD-L1-negative by CPS or IC score or otherwise ineligible, with no TROP2 requirement; TROPION-Breast05 pairs it with durvalumab in PD-L1-positive disease.","Triple-negative breast cancer, UK and EU status: the US indication (first-line, PD-1 or PD-L1 inhibitor ineligible; Datroway label effective 27 May 2026) has no EU counterpart yet, and in England the NICE appraisal for untreated triple-negative disease (GID-TA11535) and the appraisal for residual disease after neoadjuvant treatment (GID-TA11835, TROPION-Breast03) are both awaiting development. TROPION-Breast02 recruited at nine UK sites (Bristol, Cardiff, Edinburgh, three in London, Northampton, Nottingham and Warwick)."],"brand":"Datroway","code":"Dato-DXd, DS-1062","modality":"ADC","payload":"DXd (TOP1 inhibitor), DAR ~4","linker":"Tetrapeptide GGFG, cleavable","mechanism":"Humanised anti-TROP2 IgG1 with DXd; internalisation, lysosomal release, TOP1 inhibition, bystander effect.","approvals":[{"region":"US","year":2025,"indication":"HR+/HER2- metastatic breast cancer after endocrine therapy and chemotherapy"},{"region":"US","year":2025,"indication":"EGFR-mutant NSCLC after EGFR TKI and platinum chemotherapy"},{"region":"US","year":2026,"indication":"First-line unresectable/metastatic TNBC, PD-1/PD-L1 inhibitor ineligible"},{"region":"EU","year":2025,"indication":"HR+/HER2- mBC after endocrine + chemo; 4 Apr 2025"},{"region":"EU","year":2025,"indication":"HR-positive, HER2-negative unresectable or metastatic breast cancer after endocrine therapy and at least one line of chemotherapy; marketing authorisation issued 4 April 2025. No triple-negative indication in the EU on 24 September 2026","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/datroway"}],"mechanismSteps":["Antibody binds TROP2 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","DXd is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"6 mg/kg (maximum 540 mg for ≥90 kg) every 3 weeks","modifications":"Hold for grade ≥2 stomatitis or ocular events; permanently discontinue for grade ≥2 ILD","monitoring":"Oral care with steroid mouthwash prophylaxis; baseline and periodic eye examination; respiratory symptoms","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0"},"toxicity":[{"event":"Stomatitis","anyGradePct":59,"grade3PlusPct":7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0","note":"TROPION-Breast01, n=360"},{"event":"Nausea","anyGradePct":56,"grade3PlusPct":1.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0","note":"TROPION-Breast01, n=360"},{"event":"Fatigue","anyGradePct":44,"grade3PlusPct":4.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0","note":"TROPION-Breast01, n=360"},{"event":"Alopecia","anyGradePct":38,"grade3PlusPct":0,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0","note":"TROPION-Breast01, n=360"},{"event":"Constipation","anyGradePct":34,"grade3PlusPct":0.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0","note":"TROPION-Breast01, n=360"},{"event":"Dry eye","anyGradePct":27,"grade3PlusPct":0.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0","note":"TROPION-Breast01, n=360"},{"event":"Keratitis","anyGradePct":24,"grade3PlusPct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0","note":"TROPION-Breast01, n=360"},{"event":"Vomiting","anyGradePct":24,"grade3PlusPct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2950227c-6230-4ca4-a135-46e44d9424a0","note":"TROPION-Breast01, n=360"},{"event":"Stomatitis (any grade)","anyGradePct":63,"source":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22DATROWAY%22","note":"Datroway label, pooled safety population of 1,365 patients"},{"event":"Nausea (any grade)","anyGradePct":51,"source":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22DATROWAY%22","note":"Datroway label, pooled safety population"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.datroway.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: appraisal in progress for HR+/HER2- breast cancer; not yet recommended (2026)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2025-01-17","type":"approval","region":"US","note":"HR+/HER2- metastatic breast cancer after endocrine therapy and chemotherapy (TROPION-Breast01)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-03","type":"approval","region":"JP","note":"First approval worldwide for HR+/HER2- breast cancer (December 2024, Japan MHLW)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-06-23","type":"accelerated-approval","region":"US","note":"EGFR-mutant NSCLC after TKI and platinum chemotherapy (accelerated) The confirmatory requirement was still open 1.2 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with locally advanced or metastatic epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC) who have received prior EGFR-directed therapy and platinum-based chemotherapy."},{"date":"2026-05-22","type":"approval","region":"US","note":"First-line unresectable/metastatic TNBC, PD-1/PD-L1 inhibitor ineligible (TROPION-Breast02)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-datopotamab-deruxtecan-dlnk-unresectable-or-metastatic-triple-negative-breast-cancer"}]},{"id":"daunorubicin","kind":"drug","name":"Daunorubicin","aka":["Daunomycin","Rubidomycin"],"tldr":"Daunorubicin is the anthracycline most often combined with cytarabine to bring acute myeloid leukaemia into remission; it is also part of induction for acute lymphoblastic leukaemia.","summary":"Daunorubicin, isolated from Streptomyces in the 1960s, was approved in 1979 for remission induction in acute non-lymphocytic leukaemia in adults and in acute lymphocytic leukaemia in adults and children, in combination with other agents. With cytarabine it forms the 7+3 regimen that has anchored AML induction for fifty years; the label reports complete remission rates of 53 to 65% for the combination. A liposomal fixed-ratio co-formulation with cytarabine (CPX-351, Vyxeos, 2017) improved survival in secondary AML and has its own record. Cumulative dose-dependent cardiomyopathy limits lifetime exposure, and extravasation causes severe tissue necrosis.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Daunorubicin","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=daunorubicin"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/daunorubicinhydrochloride"}],"tags":["nci-list","generic"],"related":["cytarabine-7-3","cpx-351","doxorubicin"],"cancers":["aml","all-leukemia"],"sections":[],"technologies":["cytotoxic-chemotherapy","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07007312","nct03591510","nct07255872"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cerubidine (historic)","modality":"Anthracycline (topoisomerase II inhibitor)","mechanism":"Intercalates DNA and stabilises the topoisomerase II cleavage complex, causing double-strand breaks; generates free radicals responsible for cardiotoxicity.","approvals":[{"region":"US","year":1979,"indication":"Remission induction in acute non-lymphocytic leukaemia (adults) and acute lymphocytic leukaemia (adults and children), in combination"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dcvax-l","kind":"drug","name":"DCVax-L","aka":[],"tldr":"A personalised vaccine made from the patient's own immune cells and tumour. Its 20-year-old phase 3 trial reported longer survival, but the way the result was analysed has divided the field.","summary":"DCVax-L is made by Northwest Biotherapeutics. Its phase 3 (NCT00045968, enrolled 2007-2015, n=331) published in JAMA Oncology 2023 using an external control comparison after a protocol change from PFS to OS: median OS 19.3 vs 16.5 months (newly diagnosed) and 13.2 vs 7.8 months (recurrent) versus matched external controls. Because almost all placebo patients crossed over and the primary endpoint was changed post hoc, most neuro-oncologists regard the evidence as inconclusive. UK MHRA marketing application submitted December 2023; it was still not FDA-approved in September 2026.","status":"phase-3","asOf":"2026-09-06","links":[{"label":"ClinicalTrials.gov NCT00045968","url":"https://clinicaltrials.gov/study/NCT00045968"},{"label":"NICE appraisal (in development)","url":"https://www.nice.org.uk/guidance/indevelopment/gid-ta10143"}],"tags":[],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":["shared-antigen-vaccine","neoantigen-mrna-vaccine"],"targets":[],"drugs":[],"companies":["northwest-biotherapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00045968"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Listed at phase 3 rather than approved; the external-control design and endpoint change are the reasons this remains contested."],"modality":"Autologous dendritic cell vaccine (tumour lysate)","mechanism":"Autologous dendritic cells pulsed with autologous tumour lysate, injected intradermally to prime anti-tumour T cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"decipher-prostate","kind":"drug","name":"Decipher Prostate","aka":[],"tldr":"A 22-gene test on the biopsy or surgical specimen that predicts spread and death, used to decide on surveillance or adding hormone therapy.","summary":"Decipher Prostate is a genomic classifier run on a whole-transcriptome array of biopsy or surgical tissue; a 22-gene random-forest model gives a score from 0 to 1 that predicts metastasis and prostate cancer death. It is used to choose between surveillance and treatment in localised disease and whether to add hormone therapy to radiation. It was validated on the NRG/RTOG 9601, 0126 and 9902 cohorts, carries NCCN level-1 evidence, and predicts benefit from ADT with radiation in some analyses. It is the basis of the Decipher-stratified trials NRG GU009 and GU010, which test whether intensifying or de-escalating treatment by score improves outcomes. It is complemented and challenged by ArteraAI Prostate, a digital pathology model that works from slide images alone. A newcomer can think of it as a gene test that estimates how aggressive a prostate tumour really is.","status":"established","asOf":"2026-09-06","links":[{"label":"Veracyte: Decipher Prostate genomic classifier","url":"https://www.veracyte.com/tests/decipher-prostate/"}],"tags":[],"related":[],"cancers":["prostate"],"sections":[],"technologies":["rna-seq","active-surveillance"],"targets":[],"drugs":["artera-ai-prostate"],"companies":["veracyte"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Gene-expression genomic classifier","mechanism":"Whole-transcriptome array; 22-gene random-forest genomic classifier score 0-1.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"decitabine","kind":"drug","name":"Decitabine","aka":["5-aza-2'-deoxycytidine"],"tldr":"Decitabine (Dacogen) is an infusion that loosens the chemical silencing of genes in myelodysplastic syndromes and acute myeloid leukaemia, helping the bone marrow work again. An oral version combined with cedazuridine has its own record.","summary":"Decitabine was approved by the FDA in 2006 for myelodysplastic syndromes of all FAB subtypes and IPSS intermediate-1, intermediate-2 and high-risk groups, on a randomised trial of 170 patients in which decitabine plus supportive care produced responses and delayed progression to AML compared with supportive care alone. The EU authorised Dacogen in 2012 for newly diagnosed AML in adults aged 65 and over not eligible for standard induction (DACO-016). It is the hypomethylating partner for venetoclax in unfit AML alongside azacitidine, and the oral fixed-dose combination decitabine-cedazuridine (Inqovi, 2020) delivers equivalent exposure. Myelosuppression is universal early in treatment and responses typically take four to six cycles.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Decitabine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=decitabine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/decitabine"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/dacogen"}],"tags":["nci-list"],"related":["decitabine-cedazuridine","azacitidine"],"cancers":["mds","aml","mds-higher-risk"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":[],"companies":["otsuka"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05883956","nct04229979","nct02781883"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Dacogen","modality":"Hypomethylating agent (DNA methyltransferase inhibitor)","mechanism":"Incorporated into DNA after phosphorylation and traps DNA methyltransferase, causing genome-wide hypomethylation that re-expresses silenced genes and drives differentiation or apoptosis.","approvals":[{"region":"US","year":2006,"indication":"Myelodysplastic syndromes, all FAB subtypes and IPSS intermediate-1 to high risk"},{"region":"EU","year":2012,"indication":"Newly diagnosed de novo or secondary AML in adults 65 and over not candidates for standard induction"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"decitabine-cedazuridine","kind":"drug","name":"Decitabine + cedazuridine (oral)","aka":["Decitabine-cedazuridine","Oral decitabine","Cedazuridine"],"tldr":"Oral decitabine-cedazuridine is a pill version of the hypomethylating chemotherapy that, since May 2026, lets older AML patients take their whole venetoclax regimen at home.","summary":"Cedazuridine inhibits cytidine deaminase in the gut and liver so oral decitabine achieves IV-equivalent exposure. Approved for MDS/CMML (2020). On 13 May 2026 the FDA approved Inqovi with venetoclax for newly diagnosed AML in adults ≥75 or with comorbidities precluding intensive induction, based on ASCERTAIN-V (CR 41.6%, median time to CR 2 months), the first all-oral hypomethylating regimen for AML.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Decitabine/cedazuridine","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Decitabine"}],"tags":[],"related":[],"cancers":["aml","mds-higher-risk"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":["venetoclax"],"companies":["taiho-oncology"],"institutions":[],"pathways":[],"terms":[],"trials":["ascertain-v"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Inqovi","modality":"Oral hypomethylating agent","mechanism":"Decitabine incorporates into DNA and traps DNMT1; cedazuridine protects it from first-pass deamination.","approvals":[{"region":"US","year":2020,"indication":"MDS and CMML"},{"region":"US","year":2026,"indication":"Newly diagnosed AML, unfit for intensive induction, with venetoclax (ASCERTAIN-V)"},{"region":"EU","year":2023,"indication":"EU brand Inaqovi"}],"mechanismSteps":["Cedazuridine blocks cytidine deaminase in the gut and liver","Oral decitabine survives first pass and reaches the marrow","Decitabine is incorporated into DNA and traps DNMT1","Methylation is lost at silenced genes; blasts differentiate or die","Venetoclax removes BCL-2 protection so primed blasts undergo apoptosis"],"dosing":{"route":"Oral","schedule":"One tablet (35 mg decitabine / 100 mg cedazuridine) daily on days 1-5 of each 28-day cycle, with venetoclax 400 mg daily after ramp-up","monitoring":"Blood counts; TLS precautions during venetoclax ramp","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-oral-combination-decitabine-and-cedazuridine-tablets-venetoclax-newly-diagnosed-acute"},"toxicity":[{"event":"Neutropenia and febrile neutropenia","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Inqovi","note":"Class effect with venetoclax; counts monitored each cycle"},{"event":"Nausea, fatigue","note":"Common, mostly grade 1-2"}],"access":[],"regulatoryEvents":[{"date":"2020-07-07","type":"approval","region":"US","note":"MDS/CMML"},{"date":"2026-05-13","type":"approval","region":"US","note":"With venetoclax in newly diagnosed unfit AML","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-oral-combination-decitabine-and-cedazuridine-tablets-venetoclax-newly-diagnosed-acute"}]},{"id":"defibrotide","kind":"drug","name":"Defibrotide","aka":[],"tldr":"Defibrotide (Defitelio) is the only approved treatment for hepatic veno-occlusive disease, a life-threatening liver complication of high-dose chemotherapy before stem cell transplant, in patients whose kidneys or lungs are also failing.","summary":"Defibrotide was approved by the FDA in March 2016 for adults and children with hepatic veno-occlusive disease (sinusoidal obstruction syndrome) with renal or pulmonary dysfunction after haematopoietic stem cell transplantation, on a phase 3 trial against historical controls in which survival at day 100 was 38% versus 25%, supported by an expanded-access programme of several hundred patients; the EU authorised Defitelio in 2013 for severe VOD after HSCT in patients over one month old. A subsequent phase 3 prophylaxis trial in high-risk patients (HARMONY) did not meet its primary endpoint, so use remains therapeutic. Bleeding is the main risk and it is contraindicated with systemic anticoagulants or thrombolytics. Marketed by Jazz Pharmaceuticals.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Defibrotide","links":[{"label":"Study 2005-01 (Blood 2016)","url":"https://doi.org/10.1182/blood-2015-10-676924"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2c3db989-d7ad-41ed-9ebf-698dcf6c24ec"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/defibrotide-sodium"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/defitelio"}],"tags":["nci-list","supportive"],"related":[],"cancers":[],"sections":[],"technologies":["allogeneic-hsct","autologous-stem-cell-transplant"],"targets":[],"drugs":[],"companies":["jazz"],"institutions":[],"pathways":[],"terms":[],"trials":["study-2005-01"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Defitelio","modality":"Polydisperse oligonucleotide mixture (endothelial protectant)","supportive":true,"mechanism":"Mixture of single-stranded oligonucleotides from porcine intestinal mucosa that enhances plasmin-mediated fibrinolysis, reduces endothelial activation and protects sinusoidal endothelial cells; the mechanism is not fully elucidated.","approvals":[{"region":"US","year":2016,"indication":"Hepatic veno-occlusive disease with renal or pulmonary dysfunction after HSCT, adults and children"},{"region":"EU","year":2013,"indication":"Severe hepatic veno-occlusive disease after HSCT in patients over one month (Defitelio)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"degarelix","kind":"drug","name":"Degarelix","aka":[],"tldr":"An injectable hormone blocker for prostate cancer that lowers testosterone within days without the initial surge caused by agonists.","summary":"Degarelix is a competitive GnRH receptor antagonist given as a monthly subcutaneous depot for advanced prostate cancer. Because it blocks rather than stimulates the receptor, it lowers testosterone within days without the initial surge, or flare, caused by GnRH agonists, so no antiandrogen cover is needed. The CS21 trial (2008) showed testosterone suppression non-inferior to leuprolide with faster onset and lower PSA rebound, supporting US approval in 2008 and EU approval in 2009. A possible cardiovascular advantage over agonists in men with pre-existing heart disease was suggested by observational data, but the PRONOUNCE trial was inconclusive. Injection-site reactions are common and the monthly schedule is less convenient than three- or six-monthly agonist depots. For a newcomer, degarelix switches testosterone off quickly and cleanly.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Degarelix","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=degarelix"},{"label":"NICE TA404: degarelix for treating advanced hormone-dependent prostate cancer","url":"https://www.nice.org.uk/guidance/ta404"}],"tags":["gap-fill"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["androgen-deprivation"],"targets":["androgen-receptor"],"drugs":[],"companies":["ferring"],"institutions":[],"pathways":[],"terms":["prostate-adt-burden","prostate-uk-drug-approvals"],"trials":["nct02663908","nct00833248","nct00884273"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NICE TA404 recommends degarelix for advanced hormone-dependent prostate cancer only in people with spinal metastases, and only if the commissioner can achieve at least the same discounted drug cost as that available to the NHS in June 2016. The restriction to spinal metastases reflects the avoidance of the testosterone flare that a gonadotrophin-releasing hormone agonist can cause, which matters most where a flare could precipitate cord compression."],"brand":"Firmagon","modality":"GnRH antagonist depot","mechanism":"Competitive GnRH receptor antagonist producing rapid testosterone suppression without flare.","approvals":[{"region":"US","year":2008,"indication":"Advanced prostate cancer"},{"region":"EU","year":2009,"indication":"Same"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"denileukin-diftitox","kind":"drug","name":"Denileukin diftitox","aka":["Ontak (1999 to 2014)","E7777"],"tldr":"Denileukin diftitox (Lymphir) is an infusion that uses the interleukin-2 signal as a homing device to deliver a bacterial toxin into cutaneous T-cell lymphoma cells. It was withdrawn in 2014 and returned in an improved form in 2024.","summary":"The original denileukin diftitox (Ontak) was approved in 1999 for persistent or recurrent CD25-positive cutaneous T-cell lymphoma and withdrawn from the US market in 2014 because of manufacturing problems. A reformulated, higher-purity product was approved by the FDA in August 2024 as Lymphir for adults with relapsed or refractory stage I to III CTCL after at least one systemic therapy, on Study 302, an open-label single-arm trial in which 36% of patients responded (with CD25 expression on at least 20% of malignant cells required for entry). Capillary leak syndrome, infusion reactions and visual changes carry boxed warnings and require premedication and close monitoring.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Denileukin_diftitox","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=denileukin%20diftitox"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/denileukindiftitox-cxdl"}],"tags":["nci-list"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["cd25"],"drugs":[],"companies":["citius-pharmaceuticals","eisai"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01127451","nct00299689","nct00880360"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Cutaneous T-cell lymphoma has no cancer record yet."],"brand":"Lymphir","modality":"IL-2 receptor-directed cytotoxin (fusion protein)","mechanism":"Fusion of interleukin-2 with diphtheria toxin fragments A and B; binds CD25-expressing cells, is internalised, and the toxin blocks protein synthesis and kills the cell.","approvals":[{"region":"US","year":1999,"indication":"Persistent or recurrent CD25-positive CTCL (Ontak; withdrawn 2014)"},{"region":"US","year":2024,"indication":"Relapsed or refractory stage I to III CTCL after at least one systemic therapy (Lymphir)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1999-02-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Persistent or recurrent cutaneous T-cell lymphoma expressing the CD25 component of IL-2 receptor"},{"date":"2008-10-15","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1999 converted to traditional approval 9.7 years after it was granted.","indication":"Persistent or recurrent cutaneous T-cell lymphoma expressing the CD25 component of IL-2 receptor"}]},{"id":"denosumab","kind":"drug","name":"Denosumab","aka":["Wyost","Jubbonti","Osenvelt","Stoboclo"],"tldr":"Denosumab is an injection under the skin that blocks the signal driving bone breakdown. As Xgeva it prevents fractures and other bone complications in people whose cancer has spread to bone or who have myeloma; as Prolia it treats osteoporosis, including bone loss caused by hormone therapy for breast and prostate cancer.","summary":"Denosumab (Xgeva, 120 mg monthly) was approved by the FDA in November 2010 for prevention of skeletal-related events in bone metastases from solid tumours, on three phase 3 trials against zoledronic acid in breast cancer, prostate cancer and other solid tumours in which it delayed first skeletal events at least as well as, and in breast and prostate cancer better than, the bisphosphonate. Later Xgeva indications: giant cell tumour of bone (2013), hypercalcaemia of malignancy refractory to bisphosphonates (2014) and multiple myeloma (2018). Prolia (60 mg six-monthly, 2010) treats osteoporosis and bone loss from aromatase inhibitors or androgen deprivation. The EU authorised Prolia in 2010 and Xgeva in 2011; biosimilars (Wyost/Jubbonti, Osenvelt/Stoboclo) reached the market from 2024. Osteonecrosis of the jaw, atypical femoral fractures, hypocalcaemia (boxed warning in advanced kidney disease) and rebound vertebral fractures after stopping are the key risks; unlike bisphosphonates it needs no renal dose adjustment.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Denosumab","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=denosumab"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/denosumab"},{"label":"EPAR (Xgeva)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/xgeva"}],"tags":["nci-list","supportive"],"related":["zoledronic-acid"],"cancers":["breast-hr-positive","prostate","multiple-myeloma","nsclc","osteosarcoma"],"sections":[],"technologies":["bone-modifying-agents","monoclonal-antibody"],"targets":["rankl"],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":["bone-metastases"],"trials":["nct07028268"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Xgeva / Prolia","modality":"Monoclonal antibody (anti-RANKL, fully human IgG2)","supportive":true,"mechanism":"Binds RANK ligand and prevents it activating RANK on osteoclasts and their precursors, suppressing bone resorption and the tumour-bone vicious cycle.","approvals":[{"region":"US","year":2010,"indication":"Prevention of skeletal-related events in bone metastases from solid tumours (Xgeva); postmenopausal osteoporosis and treatment-induced bone loss (Prolia)"},{"region":"US","year":2013,"indication":"Giant cell tumour of bone; hypercalcaemia of malignancy (2014); multiple myeloma (2018)"},{"region":"EU","year":2011,"indication":"Prevention of skeletal-related events in bone metastases and, later, multiple myeloma; giant cell tumour of bone (Xgeva)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"depatuxizumab-mafodotin","kind":"drug","name":"Depatuxizumab mafodotin","aka":["ABT-414","Depatux-M"],"tldr":"Depatuxizumab mafodotin carried a cell-killing payload to glioblastomas with extra copies of the EGFR gene. It caused serious eye problems and, in the phase 3 INTELLANCE-1 trial, did not help patients live longer, so development stopped in 2019.","summary":"Depatuxizumab mafodotin (ABT-414) was AbbVie's antibody-drug conjugate for EGFR-amplified glioblastoma, present in roughly 40 to 50 percent of cases. The antibody binds EGFR only where it is overexpressed or mutated, sparing normal tissue, and delivers monomethyl auristatin F. It was given intravenously every two weeks with radiotherapy and temozolomide.\n\nINTELLANCE-1 (RTOG 3508), a phase 3 trial in newly diagnosed EGFR-amplified glioblastoma, found no improvement in overall survival at its 2019 interim analysis and enrolment stopped; INTELLANCE-2, in recurrent disease, showed only a late trend. Corneal toxicity with blurred vision affected most patients. The glioblastoma page lists it among the major systemic trials that failed after temozolomide.","status":"negative","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Depatuxizumab_mafodotin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Depatuxizumab_mafodotin"}],"tags":["subtype-drugs-wave"],"related":["temozolomide"],"cancers":["glioblastoma"],"sections":[],"technologies":["adc"],"targets":["egfr","tubulin"],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ABT-414","modality":"EGFR-targeted antibody-drug conjugate (MMAF payload)","mechanism":"An antibody that binds a conformational EGFR epitope exposed on tumours with EGFR amplification or the EGFRvIII mutant, linked to the microtubule toxin monomethyl auristatin F; the conjugate is internalised and the payload stops cell division.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"devimistat","kind":"drug","name":"Devimistat","aka":["CPI-613"],"tldr":"Devimistat tried to starve pancreatic cancer cells of their energy supply alongside chemotherapy; the phase 3 AVENGER 500 trial found no survival gain.","summary":"Devimistat (CPI-613) was developed by Rafael Pharmaceuticals, later renamed Cornerstone Pharmaceuticals, as a first-in-class drug against cancer cell metabolism. Early trials combined it with modified FOLFIRINOX in metastatic pancreatic cancer with encouraging response rates.\n\nThe phase 3 AVENGER 500 trial randomised patients with untreated metastatic pancreatic adenocarcinoma to modified FOLFIRINOX with or without devimistat. It did not improve overall survival, and a parallel phase 3 in acute myeloid leukaemia (ARMADA 2000) also missed. Development in pancreatic cancer stopped.","status":"negative","asOf":"2026-09-17","links":[{"label":"ClinicalTrials.gov NCT03504423","url":"https://clinicaltrials.gov/study/NCT03504423"}],"tags":[],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["cornerstone-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["avenger-500"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"CPI-613","modality":"Small-molecule inhibitor of mitochondrial metabolism (PDH and KGDH)","mechanism":"A lipoate analogue that disrupts the tumour's mitochondrial energy metabolism by inhibiting pyruvate dehydrogenase and alpha-ketoglutarate dehydrogenase.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dexamethasone","kind":"drug","name":"Dexamethasone","aka":[],"tldr":"Dexamethasone is a long-acting glucocorticoid steroid that kills lymphoid cancer cells directly, which is why it sits in nearly every myeloma regimen and in childhood leukaemia and lymphoma protocols. It is also the standard drug for preventing chemotherapy sickness and for brain swelling and spinal cord compression; high blood sugar, insomnia, muscle wasting and infection follow prolonged use.","summary":"Dexamethasone was approved in 1958 and is labelled for palliative management of leukaemias and lymphomas among dozens of inflammatory indications; in oncology it is the steroid of virtually every multiple myeloma regimen (with bortezomib, lenalidomide, daratumumab and the newer antibodies), of paediatric ALL induction where it replaced prednisone in several protocols, and of the R-CHOP-like and hyper-CVAD regimens. Hemady (20 mg tablets, 2019) is a US product labelled specifically for multiple myeloma, and Neofordex (40 mg tablets) was authorised in the EU in 2016 for the same disease. It is also first-line antiemetic prophylaxis, the standard for tumour-associated brain oedema and spinal cord compression, and a premedication for taxanes and pemetrexed. Hyperglycaemia, insomnia, myopathy, infection and adrenal suppression follow prolonged use.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Dexamethasone","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=dexamethasone"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/dexamethasone"},{"label":"EPAR (Neofordex)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/neofordex"}],"tags":["nci-list","generic","supportive"],"related":[],"cancers":["multiple-myeloma","all-leukemia","dlbcl","hodgkin-lymphoma"],"sections":[],"technologies":["antiemetic-therapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["bone-metastases"],"trials":["nct04442022","nct06868654","nct05730036","nct06464991","nct06305754","nct06956170","nct05438043","nct05572515","nct05623020","nct06152575","nct03539744","nct05519085","nct06824467","nct04975997","nct06413498","nct06158841","nct06230224","nct05020236","nct07095452","nct06208150","nct06679101","nct06868667","nct05552976","nct05552222","nct05455320","nct02343042","nct03314181","nct05372354","nct04414475","nct07284758","nct04133636","nct05583617","nct03989414","nct04068597","nct06988488","nct06087653","nct07150091","nct06892522","nct06953960","nct05704049","nct07227311","nct07018050","nct06106945","nct02899052","nct05714839","nct04973605","nct02773030","nct07150104","nct05663866","nct06979596","nct04270409","nct07742215","nct05319730","nct06353386","nct07387926","nct06788912"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Decadron (historic) / Hemady / Neofordex","modality":"Glucocorticoid","mechanism":"Synthetic glucocorticoid receptor agonist; lympholytic in leukaemia, lymphoma and myeloma, anti-inflammatory and antiemetic through genomic and non-genomic actions.","approvals":[{"region":"US","year":1958,"indication":"Palliative management of leukaemias and lymphomas; inflammatory and allergic conditions"},{"region":"US","year":2019,"indication":"Multiple myeloma in combination with other antimyeloma products (Hemady)"},{"region":"EU","year":2016,"indication":"Multiple myeloma in combination (Neofordex)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-08-13","type":"approval","region":"US","note":"FDA grants accelerated approval to iberdomide with daratumumab and hyaluronidase-fihj and dexamethasone for multiple myeloma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-iberdomide-daratumumab-and-hyaluronidase-fihj-and-dexamethasone"}]},{"id":"dexrazoxane","kind":"drug","name":"Dexrazoxane","aka":["Cardioxane","Savene"],"tldr":"Dexrazoxane protects the heart from the cumulative damage of doxorubicin in women with metastatic breast cancer who need to keep receiving it, and, as Totect, limits tissue destruction when an anthracycline leaks out of a vein.","summary":"Dexrazoxane (Zinecard) was approved in May 1995 to reduce the incidence and severity of doxorubicin cardiomyopathy in women with metastatic breast cancer who have received a cumulative 300 mg/m2 and will continue doxorubicin, on randomised trials that markedly reduced cardiac events; it is not used from the start of treatment because of an early signal of reduced response, later not confirmed. Totect was approved in 2007 for anthracycline extravasation, given within six hours on three consecutive days, on two single-arm European studies in which surgery was avoided in the great majority. Dexrazoxane is also used in paediatric leukaemia and lymphoma protocols to protect against late cardiotoxicity, an area where long-term Children's Oncology Group follow-up has been reassuring about secondary malignancy. Myelosuppression adds to that of chemotherapy.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Dexrazoxane","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=dexrazoxane"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/dexrazoxanehydrochloride"}],"tags":["nci-list","supportive"],"related":["doxorubicin"],"cancers":["breast-hr-positive","all-leukemia"],"sections":[],"technologies":["cardio-oncology"],"targets":[],"drugs":[],"companies":["pfizer","kissei"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06220032","nct00016276","nct04293562"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zinecard / Totect","modality":"Cardioprotectant and extravasation antidote (bisdioxopiperazine)","supportive":true,"mechanism":"Cyclic EDTA derivative hydrolysed intracellularly to a metal chelator that removes iron from anthracycline-iron complexes, limiting free-radical cardiac injury; also a catalytic topoisomerase II inhibitor, which underlies its action against extravasation injury.","approvals":[{"region":"US","year":1995,"indication":"Reduction of doxorubicin cardiomyopathy in women with metastatic breast cancer after 300 mg/m2 cumulative dose (Zinecard)"},{"region":"US","year":2007,"indication":"Anthracycline extravasation (Totect)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1995-05-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Supportive Care: Prevention of cardiomyopathy associated with doxorubicin administration"},{"date":"2002-10-31","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1995 converted to traditional approval 7.4 years after it was granted.","indication":"Supportive Care: Prevention of cardiomyopathy associated with doxorubicin administration"}]},{"id":"diazoxide","kind":"drug","name":"Diazoxide","aka":["Diazoxide oral suspension"],"tldr":"Diazoxide is a medicine that stops insulin-producing tumours releasing insulin. It is used to control dangerous low blood sugar from an insulinoma while the patient is prepared for surgery or when the tumour cannot be removed.","summary":"Diazoxide is a non-diuretic benzothiadiazine that inhibits insulin secretion by opening potassium channels in beta cells; it is licensed as an oral suspension for hypoglycaemia due to hyperinsulinism, including insulinoma and islet cell hyperplasia. Taken two or three times daily, it raises blood glucose within hours and is the first-line medical treatment for insulinoma symptoms before surgery or when surgery is not possible.\n\nFluid retention, hirsutism and nausea limit its use, and a thiazide diuretic is often added. Everolimus is the alternative when diazoxide fails, because mTOR inhibition also suppresses insulin release in malignant insulinoma. The pancreatic neuroendocrine tumour page names it for controlling hypoglycaemia before insulinoma surgery.","status":"established","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Diazoxide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Diazoxide"}],"tags":["subtype-drugs-wave"],"related":["everolimus"],"cancers":["pancreatic-net"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Proglycem","modality":"Oral potassium-channel opener that suppresses insulin hormone release","mechanism":"Opens ATP-sensitive potassium channels in pancreatic beta cells and insulinoma cells, preventing the depolarisation that triggers insulin release, so blood glucose rises.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"diethylstilbestrol","kind":"drug","name":"Diethylstilbestrol","aka":["DES","Stilboestrol","Honvan (fosfestrol)"],"tldr":"Diethylstilbestrol was the first hormonal treatment for prostate cancer, standard from the 1940s, and was also given for breast cancer, before its cardiovascular harms and the cancers it caused in the daughters of women who took it in pregnancy ended its use.","summary":"Charles Huggins showed in 1941 that oestrogen or castration made prostate cancer regress, work that earned the 1966 Nobel Prize, and diethylstilbestrol became the standard hormonal therapy for advanced prostate cancer for four decades. It was also a palliative treatment for postmenopausal breast cancer. The Veterans Administration trials of the 1960s showed that the standard dose caused excess cardiovascular deaths, and its use in pregnancy was linked in 1971 to clear cell adenocarcinoma of the vagina in exposed daughters, the first transplacental carcinogen identified in humans. GnRH agonists and antiandrogens replaced it; low-dose or transdermal oestrogen is still studied in castration-resistant disease.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Diethylstilbestrol","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Diethylstilbestrol"},{"label":"ChEMBL CHEMBL411","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL411"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["prostate","breast-hr-positive"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct00316927"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Stilbestrol","modality":"Oral small-molecule synthetic oestrogen","mechanism":"A non-steroidal oestrogen receptor agonist; in men it suppresses pituitary gonadotrophins and so testosterone, a chemical castration.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dinutuximab","kind":"drug","name":"Dinutuximab (ch14.18) / dinutuximab beta","aka":[],"tldr":"The antibody that raised cure rates in high-risk childhood neuroblastoma by about 20 points when given after transplant with immune boosters and retinoid.","summary":"ANBL0032: ch14.18 + GM-CSF + IL-2 + isotretinoin vs isotretinoin: 2-year EFS 66% vs 46%, OS 86% vs 75%; approved March 2015 (United Therapeutics). Dinutuximab beta (SIOPEN/EUSA/Recordati) approved in EU 2017; HR-NBL1 showed adding IL-2 to dinutuximab beta added toxicity without benefit, so IL-2 is omitted in Europe. Now also given with chemotherapy for relapse (ANBL1221: irinotecan-temozolomide-dinutuximab ORR ~50%) and during induction (ANBL17P1).","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Dinutuximab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Dinutuximab/%20dinutuximab%20beta"}],"tags":[],"related":[],"cancers":["neuroblastoma","neuroblastoma-high-risk"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["gd2"],"drugs":[],"companies":["united-therapeutics"],"institutions":[],"pathways":[],"terms":["adcc"],"trials":["anbl0032","hr-nbl1"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Unituxin / Qarziba (EU)","modality":"Monoclonal antibody (anti-GD2, chimeric)","mechanism":"Chimeric IgG1 anti-GD2; ADCC (NK cells, granulocytes with GM-CSF) and CDC against GD2+ cells; neuropathic pain from binding to peripheral nerve GD2.","approvals":[{"region":"US","year":2015,"indication":"High-risk neuroblastoma after at least partial response to induction, with GM-CSF, IL-2 and isotretinoin"},{"region":"EU","year":2017,"indication":"Dinutuximab beta: high-risk neuroblastoma after induction/transplant; relapsed/refractory disease"}],"mechanismSteps":["Antibody binds GD2 on the neuroblastoma cell surface","Fc region recruits NK cells and neutrophils (ADCC) and complement (CDC)","GM-CSF (and formerly IL-2) expands effector cells","Tumour cells lyse; peripheral nerves also bind antibody, causing pain"],"dosing":{"route":"IV","schedule":"17.5 mg/m2/day × 4 days per cycle, 5 cycles, with GM-CSF (cycles 1, 3, 5) and isotretinoin; IL-2 omitted in European practice","modifications":"Hold for grade 3-4 infusion reactions, capillary leak, neuropathy","monitoring":"Pain management (opioids, gabapentin), hypotension, capillary leak, infusion reactions, neuropathy, electrolytes"},"toxicity":[{"event":"Neuropathic pain","anyGradePct":85,"grade3PlusPct":52,"note":"ANBL0032 immunotherapy arm"},{"event":"Infusion reactions","grade3PlusPct":25},{"event":"Capillary leak syndrome","grade3PlusPct":23},{"event":"Hypokalaemia","grade3PlusPct":35}],"access":[],"regulatoryEvents":[{"date":"2015-03-10","type":"approval","region":"US","note":"Unituxin (ANBL0032)"},{"date":"2017-05","type":"approval","region":"EU","note":"Dinutuximab beta (Qarziba)"}]},{"id":"disitamab-vedotin","kind":"drug","name":"Disitamab vedotin","aka":[],"tldr":"Disitamab vedotin is a Chinese HER2 ADC approved for gastric and bladder cancer, now in global trials with Pfizer.","summary":"Disitamab vedotin is an anti-HER2 antibody (hertuzumab) linked through a cleavable mc-vc-PABC linker to the microtubule inhibitor MMAE, whose membrane permeability lets it kill neighbouring cells with lower HER2 expression. Developed by RemeGen, it was approved in China in 2021 for HER2-positive gastric cancer after two or more lines and for HER2-expressing urothelial cancer after platinum, and is given at 2.0 mg/kg every 2 weeks. A phase 3 trial with toripalimab in first-line HER2-expressing urothelial cancer was positive in 2025, and the FDA granted Breakthrough Therapy designation in HER2-positive urothelial cancer in 2020. Pfizer, via Seagen, holds ex-China rights and is running global trials. Peripheral neuropathy, neutropenia, alopecia and raised transaminases are the main toxicities. It is a Chinese HER2 ADC now heading into global development.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Disitamab%20vedotin"}],"tags":[],"related":[],"cancers":["gastric","urothelial"],"sections":[],"technologies":["adc"],"targets":["her2"],"drugs":[],"companies":["remegen","pfizer"],"institutions":[],"pathways":[],"terms":["mc-vc-pabc"],"trials":["nct06155396","nct06155383","nct06227117","nct06389006","nct06003231","nct06221748","nct06178159","nct06378242","nct06157892","nct07315750","nct05911295","nct06417554","nct06966453","nct06642545","nct04879329"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"RC48","modality":"ADC","payload":"MMAE","linker":"mc-vc-PABC","mechanism":"Anti-HER2 (hertuzumab) with MMAE.","approvals":[{"region":"China","year":2021,"indication":"HER2+ gastric cancer; HER2-expressing urothelial cancer"}],"mechanismSteps":["Antibody binds HER2 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","MMAE is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"2.0 mg/kg every 2 weeks (China label)","monitoring":"Peripheral neuropathy, neutropenia, LFTs","source":"https://www.remegen.com"},"toxicity":[{"event":"Peripheral neuropathy"},{"event":"Neutropenia"},{"event":"Alopecia"},{"event":"AST/ALT increased"}],"access":[{"country":"CN","reimbursement":"NMPA approved 2021; on the National Reimbursement Drug List","asOf":"2026-09-06"},{"country":"US","reimbursement":"Investigational (Pfizer; phase 3 in urothelial cancer)","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-06","type":"approval","region":"China","note":"Conditional approval, HER2+ gastric cancer after ≥2 lines","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-12-31","type":"approval","region":"China","note":"HER2-expressing urothelial cancer after platinum","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-09","type":"designation","region":"US","note":"Breakthrough Therapy designation, HER2+ urothelial cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-12","type":"filing","region":"China","note":"First-line urothelial cancer with toripalimab, positive phase 3","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"divarasib","kind":"drug","name":"Divarasib","aka":[],"tldr":"Divarasib is Roche's KRAS G12C pill, the first to beat the two approved KRAS drugs head-to-head (July 2026).","summary":"Covalent G12C inhibitor with ~5-20-fold greater potency than sotorasib/adagrasib in preclinical models; phase 1 ORR 53% in NSCLC. Krascendo 1 (vs sotorasib or adagrasib, previously treated NSCLC) met PFS and OS. Krascendo 2 tests divarasib + pembrolizumab ± chemotherapy first line. FDA orphan designation 2026; filing expected.","status":"phase-3","asOf":"2026-09-06","links":[{"label":"Roche Krascendo 1 release","url":"https://www.roche.com/media/releases/med-cor-2026-07-02"}],"tags":[],"related":[],"cancers":["nsclc","colorectal","kras-g12c-nsclc"],"sections":[],"technologies":["kras-inhibitors"],"targets":["kras"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["krascendo-1","nct07541170","nct06793215","nct05789082"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"GDC-6036","modality":"Small-molecule inhibitor (KRAS G12C)","mechanism":"Covalent KRAS G12C (OFF-state) inhibitor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dji136","kind":"drug","name":"DJI136","aka":[],"tldr":"DJI136 is an experimental CAR-T cell therapy from Novartis Pharmaceuticals in phase 2 trials for non-small-cell lung cancer, aimed at DLL3.","summary":"DJI136 is a CAR-T cell therapy developed by Novartis Pharmaceuticals. Its target is DLL3 (the sponsor names DLL3). The sponsor states: DLL3-targeted chimeric antigen receptor (CAR) T-cell therapy administered by intravenous infusion. ClinicalTrials.gov describes the intervention as: DLL3 targeted CAR-T therapy administered by intravenous (i.v.) infusion. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT07564401, plans to enrol 80 participants with primary completion expected 2031-03-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of DJI136","url":"https://clinicaltrials.gov/search?intr=DJI136"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["dll3"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07564401"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"DLL3-targeted chimeric antigen receptor (CAR) T-cell therapy administered by intravenous infusion.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"doc1021","kind":"drug","name":"DOC1021","aka":[],"tldr":"DOC1021 is a cancer vaccine from Diakonos Oncology Corporation, in registered phase 2 trials for glioma & glioblastoma, melanoma.","summary":"DOC1021 is listed on ClinicalTrials.gov as an intervention in 2 registered phase 2 trials sponsored by Diakonos Oncology Corporation, in glioma & glioblastoma, melanoma. A cancer vaccine, as described in the registry record: it trains the immune system against tumour antigens. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of DOC1021","url":"https://clinicaltrials.gov/search?intr=DOC1021"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["glioblastoma","melanoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["diakonos-oncology"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06805305","nct07288112"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"DOC1021","modality":"Cancer vaccine","mechanism":"A cancer vaccine, as described in the registry record: it trains the immune system against tumour antigens.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"docetaxel","kind":"drug","name":"Docetaxel","aka":[],"tldr":"The first chemotherapy to extend life in prostate cancer (2004), now part of triplet therapy at first metastatic diagnosis.","summary":"Docetaxel is a taxane that stabilises microtubules and arrests dividing cells; in prostate cancer it also disrupts androgen receptor nuclear trafficking. It was the first chemotherapy to extend life in metastatic castration-resistant prostate cancer (TAX 327, 2004) and later moved to first metastatic diagnosis: CHAARTED (OS 57.6 versus 44.0 months, HR 0.61) and STAMPEDE showed an overall survival benefit in high-volume hormone-sensitive disease, and PEACE-1 and ARASENS built triplets by adding abiraterone or darolutamide. It is also widely used in breast, lung, gastric and head and neck cancers. Neutropenia, neuropathy and fluid retention limit its use in frail patients, and whether men with low-volume metastatic disease gain anything from it remains contested. For a newcomer, it is the workhorse chemotherapy given every three weeks across many cancers.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Docetaxel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Docetaxel"},{"label":"NICE TA101: docetaxel for the treatment of hormone-refractory metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta101"},{"label":"TAX 327 (New England Journal of Medicine 2004)","url":"https://doi.org/10.1056/NEJMoa040720"}],"tags":[],"related":["acupuncture-chemotherapy-neuropathy"],"cancers":["prostate","nsclc","breast-hr-positive","gastric","head-and-neck","prostate-mhspc","prostate-mcrpc","tnbc","tnbc-metastatic","tnbc-early","lung-cancer"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":["prostate-crpc-sequencing"],"trials":["chaarted","stampede","peace-1","arasens","nct05089734","nct06340568","nct06382129","nct05348577","nct06520345","nct07518173","nct06699212","nct06356311","nct06928389","nct06989112","nct06300177","nct07213674","nct06976190","nct06952504","nct06118333","nct01933932","nct06925737","nct02486718","nct06074588","nct07291037","nct07196774","nct06881784","nct06891833","nct07287150","nct04140526","nct07306624","nct05676931","nct04895709","nct06463665","nct05904379","nct06943820","nct05431270","nct06568094","nct06445400","nct07590934","nct07246863","nct02861573","nct05126719","nct06228326","nct07070440","nct05020457","nct07102381","nct06841055","nct05910827","nct05592626","nct05054439","nct05427383","nct06908304","nct05296798","nct07415031","nct06057610","nct05999994","nct07419295","nct05094336","nct06783647","nct03671044","nct04025879","nct05867420","nct07518147","nct06857175","nct06863272","nct06745908","nct06545955","nct03505320","nct00781612","nct06947291","nct06682780","nct06780098","nct06644781","nct07225946","nct06353386","nct05173987","nct06016062","nct06632951","nct05631262","nct07669415","nct06012435","nct06123494","nct06296706","nct06741644","nct04634877","nct06663137","tnt","neopact","checkmate-017","checkmate-057","oak"],"people":[],"bottlenecks":[],"keyPapers":["paper-tpextreme-lancet-oncol-2021"],"journals":[],"dependsOn":[],"notes":["In prostate cancer, NICE TA101 recommends docetaxel within its licensed indications for hormone-refractory metastatic disease only if the Karnofsky performance-status score is 60 percent or more, and requires treatment to stop after up to 10 planned cycles, on severe adverse events, or on clinical, laboratory or imaging progression; repeat cycles are not recommended if the disease recurs after a completed course. TAX 327 is the trial behind it: median survival 18.9 months with three-weekly docetaxel and prednisone against 16.5 with mitoxantrone and prednisone, with pain response in 35 against 22 percent."],"brand":"Taxotere (generic)","modality":"Cytotoxic chemotherapy (taxane)","mechanism":"Microtubule stabilisation; also disrupts AR nuclear trafficking.","approvals":[{"region":"US","year":1996,"indication":"Locally advanced or metastatic breast cancer after chemotherapy (Taxotere, NDA 020449)"},{"region":"US","year":2004,"indication":"Metastatic CRPC (with prednisone)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1996-05-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Locally advanced or metastatic breast carcinoma that progressed during anthracycline-based therapy or relapsed during anthracycline-based adjuvant therapy"},{"date":"1998-06-22","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1996 converted to traditional approval 2.1 years after it was granted.","indication":"Locally advanced or metastatic breast carcinoma that progressed during anthracycline-based therapy or relapsed during anthracycline-based adjuvant therapy"}]},{"id":"dolasetron","kind":"drug","name":"Dolasetron","aka":["Dolasetron mesylate"],"tldr":"Dolasetron is one of the serotonin-blocking antiemetics that transformed chemotherapy in the 1990s, approved in 1997 to prevent nausea and vomiting; its intravenous form lost its chemotherapy indication in 2010 because of heart rhythm effects.","summary":"Dolasetron, approved by the FDA in September 1997, joined ondansetron and granisetron as a first-generation 5-HT3 antagonist for prevention of nausea and vomiting from moderately emetogenic chemotherapy and after surgery. It is a prodrug converted to hydrodolasetron. In 2010 the FDA contraindicated the intravenous form for chemotherapy-induced nausea because of dose-dependent QT prolongation and arrhythmias, leaving the tablets. Guidelines treat the first-generation agents as interchangeable for acute emesis, with palonosetron preferred for delayed emesis and NK1 antagonists and olanzapine added for highly emetogenic regimens.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Dolasetron","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=dolasetron"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Dolasetron"},{"label":"ChEMBL CHEMBL1200880","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200880"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["htr3a"],"drugs":["ondansetron","granisetron","palonosetron"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Anzemet","modality":"Oral 5-HT3 receptor antagonist antiemetic","supportive":true,"mechanism":"Blocks 5-HT3 serotonin receptors on vagal nerve endings in the gut and in the brainstem, cutting the signal that chemotherapy-released serotonin sends to the vomiting centre.","approvals":[{"region":"US","year":1997,"indication":"Prevention of nausea and vomiting from moderately emetogenic chemotherapy (oral); intravenous chemotherapy indication removed in 2010"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"domvanalimab","kind":"drug","name":"Domvanalimab","aka":[],"tldr":"Domvanalimab is an experimental monoclonal antibody from Gilead Sciences in phase 3 trials for non-small-cell lung cancer, bladder & urothelial cancer and head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"Domvanalimab (AB154, GS-0154) is a monoclonal antibody developed by Gilead Sciences and Arcus Biosciences. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 7 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05211895 (A Global Study to Assess the Effects of Durvalumab + Domvanalimab Following Concurrent Chemoradiation in Participants With Stage III Unresectable NSCLC) and NCT05502237 (Zimberelimab and Domvanalimab in Combination With Chemotherapy Versus Pembrolizumab With Chemotherapy in Patients With Untreated Metastatic Non-Small Cell Lung Cancer), in non-small-cell lung cancer, bladder & urothelial cancer and head and neck squamous cell carcinoma. The largest, NCT05502237, plans to enrol 1021 participants (actual) with primary completion expected 2028-06. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Domvanalimab","url":"https://clinicaltrials.gov/search?intr=AB154"},{"label":"Sponsor page","url":"https://www.arcusbio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","urothelial","head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["gilead","arcus-biosciences","astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05211895","nct05502237","nct05633667","nct05676931","nct04736173","nct06727565","nct05329766"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AB154, GS-0154","modality":"monoclonal antibody","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"donafenib","kind":"drug","name":"Donafenib","aka":[],"tldr":"Donafenib is a Chinese redesign of sorafenib that lived longer than the original in a head-to-head liver cancer trial and is approved in China as a first-line option.","summary":"Zelgen's donafenib was approved by the NMPA in June 2021 for first-line unresectable hepatocellular carcinoma on the ZGDH3 trial (668 patients: median overall survival 12.1 versus 10.3 months with sorafenib, hazard ratio 0.83; Journal of Clinical Oncology 2021), the first drug to beat sorafenib on survival in this setting. Radioiodine-refractory differentiated thyroid cancer followed in 2022. A modest gain, but a first for a Chinese company and a model of incremental chemistry (deuteration) turned into an approval.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Donafenib","links":[{"label":"Suzhou Zelgen Biopharmaceuticals","url":"https://www.zelgen.com/en/"},{"label":"ZGDH3 (J Clin Oncol 2021)","url":"https://doi.org/10.1200/JCO.20.02672"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["hcc","thyroid"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf","braf"],"drugs":[],"companies":["zelgen"],"institutions":[],"pathways":["vegf-angiogenesis","ras-mapk"],"terms":[],"trials":["zgdh3"],"people":[],"bottlenecks":[],"keyPapers":["paper-gordan-j-clin-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Zepsun","code":"CM4307","modality":"Small-molecule multi-kinase inhibitor (deuterated sorafenib)","mechanism":"Deuterium-substituted analogue of sorafenib inhibiting RAF kinases and VEGFR/PDGFR; deuteration slows metabolism and raises exposure.","approvals":[{"region":"China","year":2021,"indication":"First-line unresectable hepatocellular carcinoma (ZGDH3)"},{"region":"China","year":2022,"indication":"Radioiodine-refractory differentiated thyroid cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dordaviprone","kind":"drug","name":"Dordaviprone","aka":[],"tldr":"Dordaviprone is the first drug ever approved for a childhood and young-adult brain tumour that had none, diffuse midline glioma with the H3 K27M mutation (August 2025).","summary":"FDA accelerated approval 6 August 2025 for patients ≥1 year with H3 K27M-mutant diffuse midline glioma progressing after prior therapy, based on an integrated analysis of 50 patients across five trials: ORR 22%, median duration of response 10.3 months. Brain-penetrant oral agent from Oncoceutics → Chimerix → Jazz Pharmaceuticals (2025). Confirmatory phase 3 ACTION trial (newly diagnosed, after radiotherapy) ongoing. Debate continues on the strength of single-arm evidence.","status":"approved","asOf":"2026-09-06","links":[{"label":"FDA approval notice","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-dordaviprone-diffuse-midline-glioma"},{"label":"OncLive","url":"https://www.onclive.com/view/fda-approves-dordaviprone-for-diffuse-midline-glioma"},{"label":"Arrillaga-Romany et al., ONC201 (dordaviprone) in recurrent H3 K27M-mutant diffuse midline glioma: pooled analysis behind the accelerated approval (Journal of Clinical Oncology 2024)","url":"https://doi.org/10.1200/JCO.23.01134"}],"tags":[],"related":["h3-k27m"],"cancers":["glioblastoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["jazz"],"institutions":[],"pathways":[],"terms":["h3k27m","blood-brain-barrier","accelerated-approval"],"trials":["action-dmg"],"people":[],"bottlenecks":[],"keyPapers":["paper-arrillaga-romany-j-clin-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Modeyso","code":"ONC201","modality":"Small-molecule imipridone (ClpP agonist / DRD2 antagonist)","mechanism":"Hyperactivates the mitochondrial protease ClpP and antagonises dopamine receptor D2, triggering integrated stress response and apoptosis in H3 K27M-altered cells.","approvals":[{"region":"US","year":2025,"indication":"Recurrent H3 K27M-mutant diffuse midline glioma, age ≥1 (accelerated)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2025-08-06","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-dordaviprone-diffuse-midline-glioma","indication":"Treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy."}]},{"id":"dorocubicel","kind":"drug","name":"Dorocubicel (UM171-expanded cord blood)","aka":["UM171 cell therapy","ECT-001"],"tldr":"Zemcelpro is a laboratory-expanded umbilical cord blood transplant for adults with blood cancers who need a donor stem cell transplant but have no suitable matched donor; expanding the cells lets a single small cord blood unit be enough.","summary":"Dorocubicel was authorised by the European Commission in August 2025 as an advanced therapy medicinal product for adults with haematological malignancies requiring allogeneic haematopoietic stem cell transplantation after myeloablative conditioning for whom no other suitable donor cells are available. It consists of the CD34-positive fraction of a single cord blood unit expanded with UM171, a pyrimidoindole agonist of stem cell self-renewal discovered at Universite de Montreal, together with the unexpanded CD34-negative cells. Phase 2 studies showed faster neutrophil engraftment than historical unmanipulated cord blood and low rates of severe chronic graft-versus-host disease, addressing the slow engraftment that limited cord blood transplants in adults. It is not approved in the US. Risks are those of allogeneic transplantation: infection, graft failure and graft-versus-host disease. Developed by ExCellThera.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Cord_blood","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/zemcelpro"}],"tags":["ema-list"],"related":[],"cancers":["aml","all-leukemia","mds"],"sections":[],"technologies":["allogeneic-hsct","allogeneic-cell-therapy"],"targets":[],"drugs":[],"companies":["excellthera"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zemcelpro","modality":"Allogeneic expanded cord blood cell therapy (ATMP)","mechanism":"CD34-positive cord blood cells expanded ex vivo for seven days with the small molecule UM171, which preserves haematopoietic stem cell self-renewal, co-infused with the non-expanded CD34-negative fraction to reconstitute haematopoiesis after myeloablative conditioning.","approvals":[{"region":"EU","year":2025,"indication":"Adults with haematological malignancies requiring allogeneic HSCT after myeloablative conditioning with no other suitable donor cells"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dostarlimab","kind":"drug","name":"Dostarlimab","aka":[],"tldr":"Dostarlimab is a PD-1 blocker famous for making rectal cancer disappear without surgery in every patient with a mismatch-repair-deficient tumour.","summary":"Dostarlimab is a humanised IgG4 antibody against PD-1 that releases exhausted tumour-reactive T cells, given at 500 mg every 3 weeks for four doses and then 1,000 mg every 6 weeks. It is approved in dMMR endometrial cancer with carboplatin and paclitaxel (RUBY, with an overall survival benefit), in all-comer primary advanced or recurrent endometrial cancer with chemotherapy (2024), and for dMMR solid tumours. Its fame comes from the MSK rectal cancer study (Cercek, NEJM 2022), in which every patient with dMMR rectal cancer achieved a clinical complete response, sustained in more than 40 patients by 2025, without surgery, chemotherapy or radiation. That result led to an organ-preservation paradigm, a Breakthrough Therapy designation in 2025 and the AZUR-1 registrational trial. The durability of organ preservation is the open question.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Dostarlimab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Dostarlimab"},{"label":"Jemperli label (openFDA): dMMR solid tumour indication","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22JEMPERLI%22"},{"label":"EMA Jemperli product page","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/jemperli"}],"tags":[],"related":["dmmr-ihc"],"cancers":["msi-high-pdac","endometrial","colorectal","msi-high-colorectal","rectal-cancer","endometrial-mmr-deficient","advanced-recurrent-endometrial-cancer","uterine-carcinosarcoma","pancreatic"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06521567","nct05565378","nct06640049","nct06062420","nct06256588","nct07108270","nct06567782","nct06317311","nct03602859","nct05855200","nct06472076","nct06796907"],"people":[],"bottlenecks":[],"keyPapers":["paper-cercek-dostarlimab-rectal-nejm-2022"],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: the Jemperli label (effective 2 September 2025) indicates dostarlimab as a single agent for mismatch repair deficient recurrent or advanced solid tumours that have progressed on prior treatment with no satisfactory alternative, an accelerated approval that applies to the roughly 1 percent of pancreatic cancers with mismatch repair deficiency; the EU indication (Jemperli, marketing authorisation 21 April 2021) is endometrial cancer only."],"brand":"Jemperli","modality":"Monoclonal antibody (anti-PD-1)","mechanism":"Humanised IgG4 anti-PD-1.","approvals":[{"region":"US","year":2021,"indication":"dMMR recurrent/advanced endometrial cancer; dMMR solid tumours"},{"region":"US","year":2024,"indication":"Primary advanced/recurrent endometrial cancer with chemotherapy (all-comers)"}],"mechanismSteps":["Antibody binds PD-1","PD-1/PD-L1 engagement between T cell and tumour is blocked","Exhausted tumour-reactive T cells regain effector function","Interferon-γ and cytotoxic granules are released at the tumour","Tumour cells are killed; memory T cells persist"],"dosing":{"route":"IV infusion","schedule":"500 mg every 3 weeks for 4 doses, then 1,000 mg every 6 weeks; with carboplatin-paclitaxel in endometrial cancer","modifications":"Standard immune-mediated event algorithm","monitoring":"Thyroid, LFTs, creatinine","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Fatigue"},{"event":"Anaemia"},{"event":"Rash"},{"event":"Nausea"},{"event":"Diarrhoea"},{"event":"Hypothyroidism"},{"event":"Transaminase increase"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with chemotherapy for dMMR/MSI-H advanced endometrial cancer (TA1068)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-04-22","type":"accelerated-approval","region":"US","note":"Accelerated approval, dMMR recurrent/advanced endometrial cancer after platinum","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with mismatch repair deficient (dMMR) recurrent or advanced endometrial cancer (EC), as determined by an FDA-approved test, that has progressed on or following prior treatment with a platinum-containing regimen."},{"date":"2021-08-17","type":"accelerated-approval","region":"US","note":"dMMR solid tumours (tumour-agnostic, accelerated) The confirmatory requirement was still open 5.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment for adult patients with mismatch repair deficient (dMMR) recurrent or advanced solid tumors, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options"},{"date":"2023-02-09","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2021 converted to traditional approval 1.8 years after it was granted.","indication":"Adult patients with mismatch repair deficient (dMMR) recurrent or advanced endometrial cancer (EC), as determined by an FDA-approved test, that has progressed on or following prior treatment with a platinum-containing regimen."},{"date":"2023-07-31","type":"approval","region":"US","note":"dMMR primary advanced/recurrent endometrial cancer with chemotherapy (RUBY)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-08-01","type":"approval","region":"US","note":"All-comer primary advanced/recurrent endometrial cancer with chemotherapy","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-06","type":"designation","region":"US","note":"Breakthrough Therapy designation, dMMR locally advanced rectal cancer (organ preservation)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"doxifluridine","kind":"drug","name":"Doxifluridine","aka":["5'-Deoxy-5-fluorouridine","5'-DFUR"],"tldr":"Doxifluridine is an oral fluorouracil prodrug approved in Japan in 1987 and used in China and Korea for stomach, bowel and breast cancers; capecitabine, which the body turns into doxifluridine, later carried the same idea to the rest of the world.","summary":"Doxifluridine was developed by Roche and approved in Japan in 1987 for gastric, colorectal, breast, cervical and bladder cancers, taken as daily capsules. Because it depends on thymidine phosphorylase for activation, it achieves higher fluorouracil concentrations in tumours than in normal gut, though diarrhoea remains its main toxicity. Capecitabine adds two further enzymatic steps so that doxifluridine is generated inside the body, and it displaced doxifluridine in Western development. Doxifluridine remains in use in Japan, China and South Korea, mostly in adjuvant gastric cancer regimens.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Doxifluridine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Doxifluridine"},{"label":"ChEMBL CHEMBL1200953","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200953"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["gastric","colorectal","breast-hr-positive"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["tyms"],"drugs":["capecitabine","fluorouracil"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct00858429"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Furtulon","modality":"Oral fluoropyrimidine prodrug","mechanism":"Converted to fluorouracil by thymidine phosphorylase, an enzyme more abundant in many tumours than in normal tissue; it is the final intermediate in capecitabine's activation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"doxorubicin","kind":"drug","name":"Doxorubicin","aka":[],"tldr":"The red chemotherapy drug from a soil bacterium that is still the backbone of treatment for sarcoma, lymphoma and breast cancer, limited by cumulative heart damage.","summary":"Doxorubicin is an anthracycline from a soil bacterium that poisons topoisomerase II, intercalates into DNA and generates free radicals; its cardiotoxicity arises through topoisomerase II beta in cardiomyocytes. Approved in 1974, it is the backbone of AIM and doxorubicin-ifosfamide (sarcoma), CHOP and R-CHOP (lymphoma), ABVD and AVD (Hodgkin lymphoma), AC and EC (breast) and MAP (osteosarcoma). Cumulative cardiotoxicity above about 400 to 450 mg/m2 limits use, with cardiomyopathy in around 5% at 400 mg/m2 and rising steeply above 550 mg/m2, so serial echocardiography is required; dexrazoxane and liposomal formulations mitigate the risk. Neutropenia, alopecia and nausea are expected with every cycle. It was also the payload of early ADC attempts. This is the red chemotherapy that still anchors many curative regimens, rationed by what the heart can take over a lifetime.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Doxorubicin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Doxorubicin"}],"tags":[],"related":[],"cancers":["pancreatoblastoma","sarcoma","extremity-soft-tissue-sarcoma","undifferentiated-pleomorphic-sarcoma","angiosarcoma","retroperitoneal-sarcoma","dlbcl","hodgkin-lymphoma","tnbc","breast-hr-positive","nodular-lymphocyte-predominant-hodgkin-lymphoma","thymoma"],"sections":[],"technologies":["cytotoxic-chemotherapy","cardio-oncology"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["euramos-1","nct06097364","nct04529772","nct07044336","nct06717347","nct06340568","nct06584032","nct06191744","nct06911502","nct06486441","nct06132958","nct06091865","nct06091254","nct05888493","nct05371093","nct03407144","nct04663347","nct04542824","nct07124936","nct05201248","nct07730515","actuate-1801","nct06277154","nct06564038","nct05824975","nct05615623","nct04008797","nct03725059","nct02979522","nct06312176","nct07286331","nct06966700","nct07060807","nct06797635","nct06960577","nct04895358","nct06112379","nct05406401","nct04623541","nct06890884","nct05673785","nct04980222","nct04274426","nct06947967","nct05630209","nct04928222","nct06425302","nct06088290","nct07286266"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Adriamycin","modality":"Cytotoxic chemotherapy (anthracycline)","mechanism":"Topoisomerase II poisoning and DNA intercalation with free-radical generation; cardiotoxicity via topoisomerase IIβ in cardiomyocytes.","approvals":[{"region":"US","year":1974,"indication":"Broad: sarcomas, lymphomas, leukaemias, breast, and other solid tumours"}],"mechanismSteps":["Intercalates between DNA base pairs","Traps topoisomerase II on DNA, causing double-strand breaks","Generates reactive oxygen species","Dividing cells arrest and die; cardiomyocytes accumulate damage"],"dosing":{"route":"IV","schedule":"75 mg/m2 every 3 weeks (sarcoma, single agent or with ifosfamide); 50 mg/m2 in CHOP; 25 mg/m2 days 1, 15 in ABVD","modifications":"Lifetime cumulative limit ~450 mg/m2 (lower with chest RT); hepatic dose reduction","monitoring":"Baseline and serial echocardiography or MUGA; counts; extravasation precautions"},"toxicity":[{"event":"Neutropenia","grade3PlusPct":60,"note":"single-agent 75 mg/m2 (ANNOUNCE control arm)"},{"event":"Cardiomyopathy (cumulative)","anyGradePct":5,"note":"at 400 mg/m2; rises steeply above 550 mg/m2"},{"event":"Alopecia","anyGradePct":90},{"event":"Nausea/vomiting","anyGradePct":60}],"access":[{"country":"US","listPrice":"Generic; low cost","generic":true,"asOf":"2026-09-07"}],"regulatoryEvents":[]},{"id":"dr-01","kind":"drug","name":"DR-01","aka":[],"tldr":"DR-01 is a monoclonal antibody from Dren Bio, in registered phase 2 trials for peripheral T-cell lymphomas.","summary":"DR-01 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Dren Bio, in peripheral T-cell lymphomas. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of DR-01","url":"https://clinicaltrials.gov/search?intr=DR-01"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["peripheral-t-cell-lymphoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["dren-bio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05475925"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"DR-01","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dronabinol","kind":"drug","name":"Dronabinol","aka":["Synthetic THC","Delta-9-tetrahydrocannabinol"],"tldr":"Dronabinol is synthetic THC in a capsule, approved in 1985 for chemotherapy nausea and vomiting that other antiemetics have failed to control, and later for appetite loss in AIDS; it sits well behind the 5-HT3 and NK1 antagonists in today's guidelines.","summary":"Dronabinol, the principal psychoactive component of cannabis made synthetically, was approved by the FDA in May 1985 for nausea and vomiting from cancer chemotherapy in patients who had not responded adequately to conventional antiemetics, and in 1992 for anorexia with weight loss in AIDS. Trials in the 1970s and 1980s showed it beat placebo and matched prochlorperazine, but the 5-HT3 antagonists from 1991 and the NK1 antagonists from 2003, with dexamethasone and olanzapine, are far more effective and better tolerated, so cannabinoids are a later-line option in ASCO and NCCN guidance. Dizziness, euphoria or dysphoria and sedation limit its use, especially in older patients. Syndros is an oral solution approved in 2016.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Dronabinol","links":[{"label":"Drugs@FDA NDA018651","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=018651"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=dronabinol"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Dronabinol"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["cnr1"],"drugs":[],"companies":["abbvie","insys-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06731894"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Marinol / Syndros","modality":"Oral small-molecule synthetic cannabinoid","supportive":true,"mechanism":"Activates CB1 cannabinoid receptors in the brainstem's emetic circuitry and in appetite centres.","approvals":[{"region":"US","year":1985,"indication":"Nausea and vomiting from cancer chemotherapy in patients who have failed conventional antiemetics"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"durvalumab","kind":"drug","name":"Durvalumab","aka":[],"tldr":"A PD-L1 blocker that became standard after chemoradiation for stage III lung cancer, and now in bladder, biliary, and gastric cancers.","summary":"PACIFIC (unresectable stage III NSCLC), ADRIATIC (limited-stage SCLC, 2024), TOPAZ-1 (biliary tract), HIMALAYA (HCC with tremelimumab), NIAGARA (perioperative muscle-invasive bladder cancer, 2025), MATTERHORN (perioperative gastric, 2025), and Q2 2026 high-risk non-muscle-invasive bladder cancer with BCG (POTOMAC). Partner of Dato-DXd in TROPION-Breast05.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Durvalumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Durvalumab"},{"label":"NICE TA798: durvalumab for maintenance treatment of unresectable non-small-cell lung cancer after platinum-based chemoradiation","url":"https://www.nice.org.uk/guidance/ta798"},{"label":"NICE TA1030: durvalumab with chemotherapy before surgery then alone after surgery for resectable non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1030"},{"label":"NICE TA1041: durvalumab with etoposide and either carboplatin or cisplatin for untreated extensive-stage small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1041"},{"label":"NICE TA1099: durvalumab for treating limited-stage small-cell lung cancer after platinum-based chemoradiotherapy","url":"https://www.nice.org.uk/guidance/ta1099"}],"tags":[],"related":[],"cancers":["nsclc","sclc","cholangiocarcinoma","intrahepatic-cholangiocarcinoma","extrahepatic-cholangiocarcinoma","hcc","hcc-advanced","hcc-intermediate","urothelial","gastric","tnbc","resectable-nsclc","stage-iii-unresectable-nsclc","limited-stage-sclc","extensive-stage-sclc","advanced-recurrent-endometrial-cancer","tnbc-early","tnbc-metastatic","lung-cancer"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pdl1"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["tropion-breast05","nct05170204","nct07005128","nct03800134","nct05043090","nct07361497","nct06746116","nct03164616","nct05883644","nct03847428","nct05557838","nct07195695","nct07081633","nct06780137","nct03228667","nct05925530","nct04745689","nct06606847","nct04644289","nct06463665","nct04068610","nct03775486","nct05061550","nct05063565","nct05733598","nct07459634","nct06040099","nct07174583","nct05061134","nct03944772","nct02519348","nct05742607","nct07720284","nct03706690","nct07710885","nct05771480","nct03682068","nct05498155","nct06008093","nct05687266","nct04538742","nct06467357","nct02671435","nct02516241","nct05943106","nct06667908","nct02453282","nct06960577","nct03837899","nct02542293","nct06112379","nct03833154","nct04999969","nct04379596","nct03003962","nct05374603","nct06992609","nct03459846","nct03742102","nct07221253","nct06591520","nct04960709","nct05941897","nct04550260","nct06890598","phoenix","diamond","tropion-breast03","pacific","pacific-2","caspian","adriatic","gemstone-301"],"people":["lajos-pusztai"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Imfinzi","modality":"Monoclonal antibody (anti-PD-L1)","mechanism":"Human IgG1 anti-PD-L1 with reduced Fc effector function.","approvals":[{"region":"US","year":2017,"indication":"Locally advanced or metastatic urothelial carcinoma after platinum (accelerated; indication withdrawn 2021)"},{"region":"US","year":2018,"indication":"Unresectable stage III NSCLC after chemoradiation"},{"region":"US","year":2022,"indication":"Locally advanced or metastatic biliary tract cancer (gallbladder cancer included) with gemcitabine and cisplatin (TOPAZ-1)","note":"Imfinzi label section 1.3 (openFDA): https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22IMFINZI%22"},{"region":"UK","year":2024,"indication":"Locally advanced, unresectable or metastatic biliary tract cancer with gemcitabine and cisplatin; NICE TA944 recommended 10 January 2024 with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta944"},{"region":"US","year":2026,"indication":"High-risk NMIBC with BCG"},{"region":"England (NICE)","year":2022,"indication":"Locally advanced unresectable non-small-cell lung cancer with PD-L1 on 1 percent or more of cells that has not progressed after concurrent platinum-based chemoradiation","note":"TA798, published 22 June 2022 (PACIFIC). The PD-L1 restriction and the requirement for concurrent, not sequential, chemoradiation are NICE conditions the licence does not carry."},{"region":"England (NICE)","year":2025,"indication":"Untreated extensive-stage small-cell lung cancer, with etoposide and either carboplatin or cisplatin","note":"TA1041, published 19 February 2025, only at ECOG performance status 0 or 1 (CASPIAN)."},{"region":"England (NICE)","year":2025,"indication":"Resectable non-small-cell lung cancer (4 cm or more, or node positive) without an EGFR mutation or ALK rearrangement, neoadjuvant with platinum chemotherapy then adjuvant alone","note":"TA1030, published 15 January 2025 (AEGEAN)."},{"region":"England (NICE)","year":2025,"indication":"Limited-stage small-cell lung cancer that has not progressed after platinum-based chemoradiotherapy","note":"TA1099, published 1 October 2025 (ADRIATIC); must be funded in England within 90 days of publication."}],"mechanismSteps":["Antibody binds PD-L1","PD-1/PD-L1 engagement between T cell and tumour is blocked","Exhausted tumour-reactive T cells regain effector function","Interferon-γ and cytotoxic granules are released at the tumour","Tumour cells are killed; memory T cells persist"],"dosing":{"route":"IV infusion","schedule":"10 mg/kg every 2 weeks or 1,500 mg every 4 weeks (≥30 kg); up to 12 months after chemoradiation in stage III NSCLC","modifications":"Hold for grade 2 pneumonitis; discontinue for grade 3-4","monitoring":"Respiratory symptoms after chemoradiation, thyroid, LFTs, creatinine","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8baba4ea-2855-42fa-9bd9-5a7548d4cec3"},"toxicity":[{"event":"Pneumonitis (any cause, PACIFIC)","anyGradePct":18.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8baba4ea-2855-42fa-9bd9-5a7548d4cec3","note":"vs 12.8% placebo; 1.1% fatal"},{"event":"Cough","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8baba4ea-2855-42fa-9bd9-5a7548d4cec3","note":"PACIFIC; all-grade ≥20%"},{"event":"Fatigue","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8baba4ea-2855-42fa-9bd9-5a7548d4cec3","note":"PACIFIC; all-grade ≥20%"},{"event":"Radiation pneumonitis","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8baba4ea-2855-42fa-9bd9-5a7548d4cec3","note":"PACIFIC; all-grade ≥20%"},{"event":"Upper respiratory infection","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8baba4ea-2855-42fa-9bd9-5a7548d4cec3","note":"PACIFIC; all-grade ≥20%"},{"event":"Dyspnoea","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8baba4ea-2855-42fa-9bd9-5a7548d4cec3","note":"PACIFIC; all-grade ≥20%"},{"event":"Rash","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8baba4ea-2855-42fa-9bd9-5a7548d4cec3","note":"PACIFIC; all-grade ≥20%"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.astrazeneca-us.com/medicines/access-360","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended after chemoradiation in stage III NSCLC (TA798), biliary tract cancer, limited-stage SCLC, perioperative bladder and gastric cancer","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-05-01","type":"accelerated-approval","region":"US","note":"Urothelial carcinoma (accelerated; later withdrawn 2021)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Locally advanced or metastatic urothelial carcinoma that progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy"},{"date":"2018-02-16","type":"approval","region":"US","note":"Unresectable stage III NSCLC after chemoradiation (PACIFIC)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-03-27","type":"approval","region":"US","note":"Extensive-stage SCLC with chemotherapy (CASPIAN)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-02-19","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.8 years after its accelerated approval.","indication":"Locally advanced or metastatic urothelial carcinoma that progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy"},{"date":"2022-09-02","type":"approval","region":"US","note":"Biliary tract cancer with chemotherapy (TOPAZ-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-10-21","type":"approval","region":"US","note":"HCC with tremelimumab (HIMALAYA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-12-04","type":"approval","region":"US","note":"Limited-stage SCLC after chemoradiation (ADRIATIC)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-03-28","type":"approval","region":"US","note":"Perioperative muscle-invasive bladder cancer (NIAGARA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-11","type":"approval","region":"US","note":"Perioperative gastric/GEJ cancer with FLOT (MATTERHORN)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-05-28","type":"approval","region":"US","note":"High-risk non-muscle-invasive bladder cancer with BCG (POTOMAC)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-durvalumab-combination-bacillus-calmette-guerin-high-risk-non-muscle-invasive-bladder"}]},{"id":"duvelisib","kind":"drug","name":"Duvelisib","aka":[],"tldr":"A dual PI3K inhibitor for relapsed CLL whose survival data raised FDA concern; use is now limited to late lines.","summary":"DUO (2018): PFS 13.3 vs 9.9 months versus ofatumumab in relapsed CLL. Approved 2018 (CLL/SLL after 2 lines; FL accelerated, withdrawn 2021). A 2022 FDA review found a possible increase in deaths; boxed warnings for infections, diarrhoea/colitis, cutaneous reactions and pneumonitis. Studied in PTCL (PRIMO).","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Duvelisib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=duvelisib"}],"tags":["gap-fill"],"related":[],"cancers":["cll","peripheral-t-cell-lymphoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["pik3ca"],"drugs":[],"companies":["infinity-pharmaceuticals"],"institutions":[],"pathways":["pi3k-akt-mtor"],"terms":[],"trials":["nct06522737"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Copiktra","modality":"Small-molecule PI3Kδ/γ inhibitor","mechanism":"Dual inhibition of PI3K delta (malignant B cells) and gamma (T cells, macrophages in the microenvironment).","approvals":[{"region":"US","year":2018,"indication":"Relapsed/refractory CLL/SLL after ≥2 prior therapies; FL (withdrawn 2021)"},{"region":"EU","year":2021,"indication":"CLL after ≥2 therapies; FL after ≥2 therapies"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2018-09-24","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after at least 2 prior systemic therapies"},{"date":"2021-12-17","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.2 years after its accelerated approval.","indication":"Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after at least 2 prior systemic therapies"}]},{"id":"dzd8586","kind":"drug","name":"DZD8586","aka":["Birelentinib"],"tldr":"DZD8586 is an experimental small-molecule drug from Dizal Pharmaceuticals in phase 3 trials for chronic lymphocytic leukaemia and diffuse large B-cell lymphoma, with its target not yet stated publicly.","summary":"DZD8586 is a small-molecule drug developed by Dizal Pharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07139873 (A Study of DZD8586 Versus Investigator's Choice in r/r CLL/SLL (TAI-SHAN6)), in chronic lymphocytic leukaemia and diffuse large B-cell lymphoma. The largest, NCT07139873, plans to enrol 250 participants with primary completion expected 2029-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of DZD8586","url":"https://clinicaltrials.gov/search?intr=DZD8586"},{"label":"Sponsor page","url":"https://www.dizalpharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cll","dlbcl"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["dizal"],"institutions":[],"pathways":[],"terms":[],"trials":["tai-shan6","nct07059650","nct07154264","nct06539182"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"e-edv-d682","kind":"drug","name":"E-EDV-D682","aka":[],"tldr":"E-EDV-D682 is an experimental EDV nanocell (bacterial minicell) drug conjugate from Engeneic Pty in phase 2 trials for pancreatic ductal adenocarcinoma, aimed at EGFR.","summary":"E-EDV-D682 (EDV-D682) is an EDV nanocell (bacterial minicell) drug conjugate developed by Engeneic Pty. Its target is EGFR (the sponsor names EGFR). The sponsor states: E-EDV-D682 packages the chemotherapeutic payload PNU-159682 inside a 400 nm, bacterially derived EnGeneIC EDV nanocell that is targeted to EGFR-expressing cancer cells via a bispecific antibody, delivering the payload directly into tumour cells. ClinicalTrials.gov describes the intervention as: E-EDV-D682 is a product based on the EnGeneIC EDV™ technology. EDVs are bacterially derived nanocells 400 nm in diameter that can be packaged with a range of different chemotherapeutic drugs and specifically targeted to cancer cell receptor. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in pancreatic ductal adenocarcinoma. The largest, NCT07049055, plans to enrol 144 participants with primary completion expected 2028-09. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of E-EDV-D682","url":"https://clinicaltrials.gov/search?intr=EDV-D682"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":["egfr"],"drugs":[],"companies":["engeneic"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07049055"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"EDV-D682","modality":"EDV nanocell (bacterial minicell) drug conjugate","mechanism":"E-EDV-D682 packages the chemotherapeutic payload PNU-159682 inside a 400 nm, bacterially derived EnGeneIC EDV nanocell that is targeted to EGFR-expressing cancer cells via a bispecific antibody, delivering the payload directly into tumour cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eb-car30-nk","kind":"drug","name":"EB-CAR30-NK","aka":[],"tldr":"EB-CAR30-NK is an experimental CAR-NK cell therapy from Beijing Biotech in phase 2 trials for thymoma and thymic carcinoma, aimed at CD30.","summary":"EB-CAR30-NK is a CAR-NK cell therapy developed by Beijing Biotech. Its target is CD30. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in thymoma and thymic carcinoma. The largest, NCT07598955, plans to enrol 36 participants with primary completion expected 2027-06-14. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of EB-CAR30-NK","url":"https://clinicaltrials.gov/search?intr=EB-CAR30-NK"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["thymic-epithelial"],"sections":[],"technologies":[],"targets":["cd30"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07598955"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-NK cell therapy","mechanism":"CAR-NK cell therapy directed at CD30, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eb-mf-car-nk-01","kind":"drug","name":"EB-MF-CAR-NK-01","aka":["Mesothelin/FAP Dual-Target CAR-NK Cells"],"tldr":"EB-MF-CAR-NK-01 is an experimental bispecific antibody from Beijing Biotech in phase 2 trials for mesothelioma, aimed at Mesothelin and FAP.","summary":"EB-MF-CAR-NK-01 is a bispecific antibody developed by Beijing Biotech. Its targets are Mesothelin and FAP. ClinicalTrials.gov describes the intervention as: Example investigational product: donor-derived allogeneic NK cells engineered to recognize both MSLN-positive tumour cells and FAPpositive stromal elements, manufactured under GMP. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in mesothelioma. The largest, NCT07510815, plans to enrol 36 participants with primary completion expected 2027-03-14. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of EB-MF-CAR-NK-01","url":"https://clinicaltrials.gov/search?intr=EB-MF-CAR-NK-01"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["mesothelioma"],"sections":[],"technologies":[],"targets":["mesothelin","fap"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07510815"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"Bispecific antibody directed at Mesothelin and FAP, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eb-nk-301","kind":"drug","name":"EB-NK-301","aka":["TROP2-CAR NK"],"tldr":"EB-NK-301 is an experimental CAR-NK cell therapy from Beijing Biotech in phase 2 trials, aimed at TROP2.","summary":"EB-NK-301 is a CAR-NK cell therapy developed by Beijing Biotech. Its target is TROP2. ClinicalTrials.gov describes the intervention as: Investigational allogeneic CAR-NK cell product targeting TROP2, administered by intravenous infusion. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT07589530, plans to enrol 60 participants with primary completion expected 2027-02-14. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of EB-NK-301","url":"https://clinicaltrials.gov/search?intr=EB-NK-301"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":["car-nk-macrophage"],"targets":["trop2"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07589530"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-NK cell therapy","mechanism":"CAR-NK cell therapy directed at TROP2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"efbemalenograstim-alfa","kind":"drug","name":"Efbemalenograstim alfa","aka":["F-627"],"tldr":"Efbemalenograstim alfa (Ryzneuta) is a once-per-cycle white cell booster, an alternative to pegfilgrastim, that reduces the risk of febrile infections after chemotherapy.","summary":"Efbemalenograstim alfa was approved by the FDA in November 2023 to decrease the incidence of infection manifested by febrile neutropenia in adults with non-myeloid malignancies receiving myelosuppressive chemotherapy, and by the European Commission in March 2024 for reduction of the duration of neutropenia and incidence of febrile neutropenia after cytotoxic chemotherapy (excluding CML and MDS). Two phase 3 trials in breast cancer patients receiving docetaxel-cyclophosphamide showed a duration of severe neutropenia comparable to pegfilgrastim (GC-627-04) and superior to placebo (GC-627-05). Unlike pegylated G-CSF it uses Fc fusion rather than PEG for half-life extension. Bone pain, nausea and thrombocytopenia are the common adverse effects; it is not indicated for stem cell mobilisation.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Efbemalenograstim_alfa","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/ryzneuta"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=efbemalenograstim"}],"tags":["ema-list","supportive"],"related":["pegfilgrastim","filgrastim"],"cancers":[],"sections":[],"technologies":["g-csf-growth-factors"],"targets":[],"drugs":[],"companies":["evive-biotech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03252431","nct04174599","nct02872103"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ryzneuta","modality":"Long-acting recombinant G-CSF dimer (Fc fusion)","supportive":true,"mechanism":"Two G-CSF molecules fused to a human IgG2 Fc fragment; Fc-mediated recycling prolongs the half-life so that a single dose per chemotherapy cycle stimulates neutrophil production and release.","approvals":[{"region":"US","year":2023,"indication":"Decrease in febrile neutropenia in adults with non-myeloid malignancies on myelosuppressive chemotherapy"},{"region":"EU","year":2024,"indication":"Reduction in duration of neutropenia and febrile neutropenia after cytotoxic chemotherapy in adults (excluding CML and MDS)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eflapegrastim","kind":"drug","name":"Eflapegrastim","aka":["Eflapegrastim-xnst","SPI-2012"],"tldr":"Eflapegrastim is a once-per-cycle white cell growth factor approved in the United States in 2022 to prevent infections when chemotherapy for solid tumours knocks down neutrophils, an alternative to pegfilgrastim.","summary":"Eflapegrastim uses Hanmi's long-acting carrier technology to extend the life of G-CSF. The FDA approved it in September 2022 to decrease the incidence of febrile neutropenia in adults with non-myeloid malignancies receiving myelosuppressive chemotherapy, after the ADVANCE and RECOVER trials showed it was non-inferior to pegfilgrastim for the duration of severe neutropenia in breast cancer patients on docetaxel and cyclophosphamide. It is given about 24 hours after chemotherapy; bone pain and nausea are the common side effects. It was developed by Spectrum Pharmaceuticals and is now marketed by Assertio.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Eflapegrastim","links":[{"label":"Drugs@FDA BLA761148","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761148"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=eflapegrastim"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Eflapegrastim"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["cytokine-therapy"],"targets":["csf3r"],"drugs":["pegfilgrastim"],"companies":["spectrum-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04570423"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Rolvedon","modality":"Long-acting G-CSF fused to an antibody Fc fragment, once per chemotherapy cycle","supportive":true,"mechanism":"G-CSF joined to an IgG4 Fc fragment through a polyethylene glycol linker, so it is cleared slowly and keeps stimulating neutrophil production for the whole cycle.","approvals":[{"region":"US","year":2022,"indication":"To decrease the incidence of febrile neutropenia in adults with non-myeloid malignancies receiving myelosuppressive chemotherapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eflornithine","kind":"drug","name":"Eflornithine (DFMO)","aka":[],"tldr":"Eflornithine (DFMO) is an old sleeping-sickness drug repurposed as the first oral maintenance therapy for high-risk neuroblastoma, approved in December 2023 to reduce relapse after immunotherapy.","summary":"Approved 13 December 2023 (US WorldMeds) for adults and children with high-risk neuroblastoma who achieved at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy. Evidence: single-arm NMTRC003/003B (n=105) compared with an external control from ANBL0032 (EFS HR 0.48, OS HR 0.32), a controversial externally controlled approval. EU applications filed 2025. Two years of twice-daily tablets.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Eflornithine","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Eflornithine"}],"tags":[],"related":[],"cancers":["neuroblastoma","neuroblastoma-high-risk"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["us-worldmeds"],"institutions":[],"pathways":[],"terms":["mycn-amplification"],"trials":["nmtrc003","nct07287917"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Iwilfin","modality":"Small-molecule ornithine decarboxylase inhibitor","mechanism":"Irreversible inhibitor of ornithine decarboxylase (ODC1), a direct MYCN target, depleting polyamines required for neuroblastoma proliferation.","approvals":[{"region":"US","year":2023,"indication":"Maintenance to reduce relapse risk in high-risk neuroblastoma after ≥PR to prior therapy including anti-GD2"}],"mechanismSteps":["DFMO irreversibly inactivates ornithine decarboxylase","Putrescine, spermidine and spermine fall","MYCN-driven translation and proliferation slow","Residual cells are held in check during two years of maintenance"],"dosing":{"route":"Oral","schedule":"Body-surface-area-based dose twice daily for 2 years (e.g. 768 mg twice daily for BSA >1.5 m2)","monitoring":"Hearing (ototoxicity), liver enzymes, counts, growth"},"toxicity":[{"event":"Hearing loss","anyGradePct":27,"note":"NMTRC003/003B; largely pre-existing from platinum"},{"event":"Otitis media","anyGradePct":27},{"event":"Neutropenia","anyGradePct":24},{"event":"Elevated ALT","anyGradePct":33}],"access":[],"regulatoryEvents":[{"date":"2023-12-13","type":"approval","region":"US","note":"First oral maintenance therapy; externally controlled evidence","source":"https://ascopubs.org/doi/10.1200/JCO.24.00546"},{"date":"2025","type":"filing","region":"EU","note":"MAA submitted to EMA, UK, Australia, Switzerland","source":"https://www.onclive.com/view/eu-approval-is-sought-for-eflornithine-in-high-risk-neuroblastoma"}]},{"id":"eik1001","kind":"drug","name":"EIK1001","aka":[],"tldr":"EIK1001 is an experimental investigational agent whose form is not stated in the registry from Eikon Therapeutics in phase 3 trials for melanoma and non-small-cell lung cancer, with its target not yet stated publicly.","summary":"EIK1001 is an investigational agent whose form is not stated in the registry developed by Eikon Therapeutics. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: EIK1001 is a Toll-like receptor 7/8 (TLR 7/8) dual agonist. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06697301 (Safety and Efficacy of EIK1001 in Combo With Pembro Versus Placebo and Pembro as First-Line Therapy in Patients With Advanced Melanoma) and NCT07365319 (A Safety and Efficacy Study of EIK1001 in Combination With Pembrolizumab and Chemotherapy in Participants With Stage 4 Non-Small Cell Lung Cancer), in melanoma and non-small-cell lung cancer. The largest, NCT07365319, plans to enrol 750 participants with primary completion expected 2035-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of EIK1001","url":"https://clinicaltrials.gov/search?intr=EIK1001"},{"label":"Sponsor page","url":"https://www.eikontx.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma","nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["eikon-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06697301","nct07365319","nct06246110"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eik1005","kind":"drug","name":"EIK1005","aka":[],"tldr":"EIK1005 is a small-molecule inhibitor from Eikon Therapeutics, in registered phase 2 trials for colorectal cancer, endometrial cancer.","summary":"EIK1005 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Eikon Therapeutics, in colorectal cancer, endometrial cancer. Described in the registry record as a eik1005 is a selective inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of EIK1005","url":"https://clinicaltrials.gov/search?intr=EIK1005"},{"label":"ClinicalTrials.gov NCT07262619","url":"https://clinicaltrials.gov/study/NCT07262619"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["colorectal","endometrial"],"sections":[],"technologies":[],"targets":["wrn"],"drugs":[],"companies":["eikon-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07262619"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["EIK1005 is described in its registry record as a selective inhibitor of the Werner helicase, the synthetic-lethal dependency of microsatellite-unstable tumours, and is being tested alone and with pembrolizumab."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"EIK1005","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a eik1005 is a selective inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"elacestrant","kind":"drug","name":"Elacestrant","aka":[],"tldr":"Elacestrant was the first oral oestrogen-receptor degrader (2023), for ESR1-mutant breast cancer detected by blood test.","summary":"Elacestrant is an oral selective oestrogen receptor degrader (SERD) that binds ER-alpha and promotes its degradation, retaining activity against ESR1 mutations. Taken as 345 mg once daily with food, it was approved in 2023, the first oral SERD, for ESR1-mutant ER-positive, HER2-negative advanced breast cancer after at least one endocrine therapy. In EMERALD the progression-free survival benefit was confined to ESR1-mutant disease, and the Guardant360 CDx blood test is the companion diagnostic, so eligibility is decided by liquid biopsy. Stemline/Menarini markets it. Imlunestrant (Lilly, Inluriyo, 2025) and camizestrant (AstraZeneca, SERENA-6 ctDNA-guided switch) followed, and whether to switch when an ESR1 mutation first appears in blood is the live question. For a newcomer: an oestrogen-blocking pill chosen by a blood test.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Elacestrant","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Elacestrant"}],"tags":[],"related":["er-status","esr1-mutation-ctdna"],"cancers":["breast-hr-positive","hr-positive-metastatic-post-cdk46"],"sections":[],"technologies":["endocrine-therapy","liquid-biopsy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["menarini"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07766018","nct05596409","nct05563220","nct06492616","nct05386108","nct05573126"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Orserdu","modality":"Oral SERD","mechanism":"Oral selective ER degrader.","approvals":[{"region":"US","year":2023,"indication":"ESR1-mutant ER+/HER2- advanced breast cancer after ≥1 endocrine therapy"}],"mechanismSteps":["Oral SERD binds ERα","Receptor conformation changes and ER is degraded","ESR1-mutant, ligand-independent receptors are removed","ER-dependent transcription stops"],"dosing":{"route":"Oral","schedule":"345 mg once daily with food","modifications":"Reduce to 258 then 172 mg for grade 3 toxicity","monitoring":"Lipids at baseline and periodically","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Nausea","note":"EMERALD: 35% any grade"},{"event":"Musculoskeletal pain"},{"event":"Vomiting"},{"event":"Fatigue"},{"event":"Dyspepsia"},{"event":"Hypercholesterolaemia"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for ESR1-mutated ER+/HER2- advanced breast cancer (2024)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-01-27","type":"approval","region":"US","note":"ESR1-mutated ER+/HER2- advanced breast cancer after ≥1 endocrine therapy (EMERALD): first oral SERD","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-09","type":"approval","region":"EU","note":"EMA approval","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"elenestinib","kind":"drug","name":"Elenestinib","aka":[],"tldr":"Elenestinib is an experimental small-molecule drug from Blueprint Medicines in phase 3 trials for systemic mastocytosis, with its target not yet stated publicly.","summary":"Elenestinib (BLU-263) is a small-molecule drug developed by Blueprint Medicines. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04910685 ((HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis), in systemic mastocytosis. The largest, NCT04910685, plans to enrol 534 participants with primary completion expected 2032-09-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Elenestinib","url":"https://clinicaltrials.gov/search?intr=BLU-263"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["systemic-mastocytosis","indolent-systemic-mastocytosis"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04910685"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"BLU-263","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eli-002-7p","kind":"drug","name":"ELI-002 7P","aka":[],"tldr":"A ready-made vaccine against the seven commonest KRAS mutations, given after pancreatic cancer surgery. Its phase 2 missed the main goal in 2026 but showed signs of activity.","summary":"Elicio's amphiphile peptides (7 mKRAS antigens) plus CpG adjuvant, engineered to drain to lymph nodes. Phase 1 AMPLIFY-201 induced mKRAS-specific T cells in most patients with ctDNA reductions. Randomised phase 2 AMPLIFY-7P (adjuvant PDAC, results June 2026) did not meet its primary DFS endpoint in the intent-to-treat population; post-hoc landmark analyses reported ~14% absolute DFS benefit during active treatment. Elicio is refining a phase 3 strategy around R0-resected patients.","status":"phase-2","asOf":"2026-09-06","links":[{"label":"Elicio AMPLIFY-7P results (June 2026)","url":"https://elicio.com/press_releases/elicio-therapeutics-reports-results-from-phase-2-amplify-7p-study-and-outlines-refined-phase-3-development-strategy-for-eli-002-7p-in-adjuvant-pancreatic-cancer/"}],"tags":[],"related":[],"cancers":["pancreatic","resectable-pdac","colorectal"],"sections":[],"technologies":["shared-antigen-vaccine","mrd-testing"],"targets":["kras"],"drugs":[],"companies":["elicio-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["amplify-7p"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Off-the-shelf lymph-node-targeted KRAS peptide vaccine","mechanism":"Albumin-binding lipid tails carry mutant-KRAS peptides to lymph nodes for dendritic cell presentation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"elironrasib","kind":"drug","name":"Elironrasib","aka":[],"tldr":"A next-generation KRAS G12C drug that hits the active form of the protein, from the same company as daraxonrasib.","summary":"Elironrasib is a covalent tri-complex inhibitor that binds KRAS G12C in its active, GTP-bound (ON) state, unlike the first-generation drugs sotorasib and adagrasib, which trap the inactive OFF state. Hitting the active protein is intended to produce deeper, more durable responses and to overcome the adaptive RAS reactivation that limits OFF-state inhibitors. It is being tested alone and with daraxonrasib, the pan-RAS(ON) inhibitor from the same company, in non-small-cell lung cancer, colorectal cancer and the roughly 1 to 2% of pancreatic cancers that carry G12C. It is in phase 2 with no approval yet, and whether ON-state inhibition delivers a clinically meaningful advantage over the approved G12C drugs is the open question. For a newcomer, it is a next-generation KRAS G12C drug that hits the switched-on form of the protein.","status":"phase-2","asOf":"2026-09-06","links":[{"label":"ClinicalTrials.gov: trials of Elironrasib","url":"https://clinicaltrials.gov/search?intr=RMC-6291"}],"tags":[],"related":[],"cancers":["nsclc","colorectal","pancreatic","kras-g12c-pdac"],"sections":[],"technologies":["kras-inhibitors"],"targets":["kras"],"drugs":[],"companies":["revolution-medicines"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct07397338","nct06162221","nct06128551"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"RMC-6291","modality":"Small-molecule RAS(ON) G12C-selective inhibitor","mechanism":"Covalent tri-complex inhibitor of KRAS G12C in the GTP-bound state.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"elotuzumab","kind":"drug","name":"Elotuzumab","aka":[],"tldr":"An immune-stimulating antibody for myeloma that works only in combination, adding benefit to lenalidomide or pomalidomide.","summary":"Elotuzumab is a monoclonal antibody against SLAMF7 (CS1), a surface protein on both myeloma cells and natural killer cells. It marks myeloma cells for NK-mediated antibody-dependent cellular cytotoxicity and simultaneously activates the NK cells, but it has no single-agent activity and is always combined with an immunomodulatory drug and dexamethasone. ELOQUENT-2 (2015) showed median progression-free survival of 19.4 versus 14.9 months when added to lenalidomide-dexamethasone in patients after 1 to 3 prior therapies, leading to US approval in 2015. ELOQUENT-3 (2018) supported a second approval with pomalidomide-dexamethasone after at least two therapies. CD38 antibodies have largely displaced it, but it remains an option in daratumumab-refractory disease and is generally well tolerated. The key idea is an antibody that works only by recruiting the patient's own immune cells.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Elotuzumab","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=elotuzumab"}],"tags":["gap-fill"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["monoclonal-antibody"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":["adcc"],"trials":["nct05730036","nct06152575","nct06158841","nct06208150","nct03989414"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Empliciti","modality":"Anti-SLAMF7 monoclonal antibody","mechanism":"Binds SLAMF7 (CS1) on myeloma cells and NK cells, enhancing NK-mediated ADCC; no single-agent activity.","approvals":[{"region":"US","year":2015,"indication":"Multiple myeloma after 1-3 prior therapies, with lenalidomide-dexamethasone"},{"region":"US","year":2018,"indication":"With pomalidomide-dexamethasone after ≥2 therapies"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"elraglusib","kind":"drug","name":"Elraglusib","aka":["9-ING-41"],"tldr":"Elraglusib blocks a kinase that helps pancreatic cancer resist chemotherapy; in a randomised phase 2 it lengthened survival when added to gemcitabine and nab-paclitaxel, and a phase 3 is planned.","summary":"Actuate Therapeutics developed elraglusib from a series of GSK-3 beta inhibitors. In the Actuate 1801 study, part 3B randomised patients with untreated metastatic pancreatic adenocarcinoma to gemcitabine and nab-paclitaxel with or without elraglusib.\n\nThe company reported that adding elraglusib improved overall survival and one-year survival, with manageable added toxicity, mainly visual disturbance and fatigue. The result is from a phase 2 trial and awaits confirmation in a phase 3, which Actuate has said it is planning with regulators.","status":"phase-2","asOf":"2026-09-17","links":[{"label":"ClinicalTrials.gov NCT03678883","url":"https://clinicaltrials.gov/study/NCT03678883"},{"label":"Actuate Therapeutics: elraglusib programme","url":"https://www.actuatetherapeutics.com"},{"label":"Mahalingam et al., elraglusib and chemotherapy in metastatic pancreatic ductal adenocarcinoma, randomised phase 2 (Nature Medicine 2026)","url":"https://doi.org/10.1038/s41591-026-04327-4"},{"label":"Mahalingam et al., single-arm phase 2 (ESMO Open 2025)","url":"https://doi.org/10.1016/j.esmoop.2025.105122"}],"tags":[],"related":[],"cancers":["pancreatic","metastatic-pdac"],"sections":[],"technologies":["kinase-inhibitors"],"targets":[],"drugs":[],"companies":["actuate-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["actuate-1801"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Randomised phase 2 (Nature Medicine 2026, 155 against 78 patients, untreated metastatic disease): median overall survival 10.1 against 7.2 months with gemcitabine plus nab-paclitaxel alone (hazard ratio 0.62, 95 percent confidence interval 0.46 to 0.84; p 0.01); one-year survival 44.1 against 22.3 percent; grade 3 or worse neutropenia 52.3 against 30.8 percent, fatigue 16.8 against 5.1 percent; the elraglusib dose was reduced to 9.3 mg/kg during the earlier single-arm phase because it exacerbated chemotherapy toxicity (ESMO Open 2025). A phase 3 is planned."],"code":"9-ING-41","modality":"Small-molecule GSK-3 beta inhibitor","mechanism":"Inhibits glycogen synthase kinase 3 beta, a kinase that drives NF-kB survival signalling and chemoresistance in pancreatic cancer and dampens anti-tumour immunity.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"elranatamab","kind":"drug","name":"Elranatamab","aka":[],"tldr":"Elranatamab is Pfizer's BCMA bispecific, given under the skin every week then every two weeks, for myeloma after four prior lines.","summary":"Elranatamab is a humanised IgG2a BCMAxCD3 bispecific antibody that links T cells to BCMA-expressing plasma cells, given subcutaneously with step-up doses of 12 mg and 32 mg, then 76 mg weekly and every 2 weeks after 24 weeks in responders. In MagnetisMM-3, BCMA-naive patients had ORR 61% and CR 35% with median PFS of about 17 months, leading to accelerated approval in August 2023 after at least 4 prior lines. Cytokine release syndrome was common but mild; infections affected 70% (40% grade 3 or higher), so IVIG for hypogammaglobulinaemia and infection vigilance are central to its use. MagnetisMM-5 (with daratumumab, versus Dara-Pd) and MagnetisMM-7 (post-transplant maintenance) are the confirmatory phase 3 trials. Pfizer develops it. It is an off-the-shelf injection that recruits the patient's own T cells against myeloma without the manufacturing wait of CAR-T.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Elranatamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Elranatamab"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-relapsed-refractory"],"sections":[],"technologies":["t-cell-engager"],"targets":["bcma","cd3"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":["crs","step-up-dosing"],"trials":["magnetismm-3","nct05623020","nct06152575","nct05020236","nct05317416","nct06988488","nct06106945"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Elrexfio","modality":"Bispecific T-cell engager (BCMA×CD3)","mechanism":"Humanised IgG2a BCMA×CD3 bispecific; step-up dosing 12 mg, 32 mg, then 76 mg weekly.","approvals":[{"region":"US","year":2023,"indication":"Relapsed/refractory myeloma after ≥4 lines (accelerated)"}],"mechanismSteps":[],"dosing":{"route":"Subcutaneous","schedule":"12 mg day 1, 32 mg day 4, 76 mg weekly from day 8; every 2 weeks after 24 weeks if responding","monitoring":"CRS/ICANS (48-hour hospitalisation after first two step-up doses), infections, IVIG for hypogammaglobulinaemia"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":58,"grade3PlusPct":0,"note":"MagnetisMM-3 cohort A"},{"event":"Infections","anyGradePct":70,"grade3PlusPct":40},{"event":"Neutropenia","grade3PlusPct":49}],"access":[],"regulatoryEvents":[{"date":"2023-08-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 3.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-elranatamab-bcmm-multiple-myeloma","indication":"Adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody."}]},{"id":"elritercept","kind":"drug","name":"Elritercept","aka":["TAK-226"],"tldr":"Elritercept is Keros and Takeda's ligand trap for the anaemia of myelodysplastic syndromes, a relative of luspatercept, in phase 3 trials.","summary":"Keros Therapeutics designed elritercept (KER-050) to release the brake that activin-class ligands put on erythropoiesis and thrombopoiesis; Takeda licensed it in 2024 as TAK-226. Phase 2 data showed transfusion independence in lower-risk myelodysplastic syndromes including patients with ring sideroblasts, and phase 3 trials in transfusion-dependent lower-risk MDS are registered. Luspatercept is the approved comparator in the class.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Elritercept","url":"https://clinicaltrials.gov/search?intr=KER-050"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["mds"],"sections":[],"technologies":["cytokine-therapy"],"targets":[],"drugs":[],"companies":["keros-therapeutics","takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06499285"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"KER-050","modality":"Activin receptor type IIA ligand trap (fusion protein), subcutaneous","mechanism":"Binds TGF-beta family ligands such as activin A and GDF11 that hold back red cell and platelet precursors, freeing late-stage blood cell production in ineffective haematopoiesis.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eltrombopag","kind":"drug","name":"Eltrombopag","aka":["Eltrombopag Olamine","SB-497115"],"tldr":"Eltrombopag is a daily tablet that raises platelet counts by stimulating the bone marrow; it is approved for immune thrombocytopenia and severe aplastic anaemia, and haematologists also use it when low platelets complicate blood cancers and their treatment.","summary":"Eltrombopag is an oral thrombopoietin receptor agonist first approved in the United States in 2008 for chronic immune thrombocytopenia (ITP) that has not responded to steroids, immunoglobulins or splenectomy. Its label also covers thrombocytopenia in hepatitis C to allow interferon treatment and severe aplastic anaemia, a bone marrow failure that sits alongside myelodysplastic syndromes in haematology clinics. It is not approved for chemotherapy-induced thrombocytopenia or for low platelets in leukaemia or myelodysplasia, though it has been studied in both and is used off label. Liver toxicity and a possible increase in marrow fibrosis or blast counts are the main cautions.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Eltrombopag","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=eltrombopag"},{"label":"Drugs@FDA NDA 022291","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=022291"}],"tags":["gap-fill","supportive-care"],"related":[],"cancers":["aml","mds"],"sections":[],"technologies":["supportive-care"],"targets":["mpl"],"drugs":["romiplostim"],"companies":["novartis"],"institutions":[],"pathways":[],"terms":["thrombocytopenia"],"trials":["nct03603795","nct01890746","nct01772420"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Promacta / Revolade","modality":"Oral thrombopoietin receptor agonist","supportive":true,"mechanism":"A small molecule that binds the transmembrane part of the thrombopoietin receptor (MPL) on megakaryocytes and their precursors, driving them to make platelets.","approvals":[{"region":"US","year":2008,"indication":"Chronic immune thrombocytopenia with insufficient response to corticosteroids, immunoglobulins or splenectomy","note":"Later extended to thrombocytopenia in chronic hepatitis C and to severe aplastic anaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"emactuzumab","kind":"drug","name":"Emactuzumab","aka":[],"tldr":"Emactuzumab is an experimental monoclonal antibody from SynOx Therapeutics in phase 3 trials for tenosynovial giant cell tumour, aimed at CSF1R.","summary":"Emactuzumab is a monoclonal antibody developed by SynOx Therapeutics and Hoffmann-La Roche. Its target is CSF1R (the sponsor names CSF-1R). The sponsor states: Emactuzumab is given as five intravenous infusions once every two weeks; a trial exclusion criterion (prior therapy targeting CSF-1 or CSF-1R) indicates it is a CSF-1R-directed antibody used to treat tenosynovial giant cell tumour by disrupting CSF-1/CSF-1R signalling. ClinicalTrials.gov describes the intervention as: Emactuzumab administered once every 2 weeks (q2w). It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05417789 (Study of Emactuzumab for Tenosynovial Giant Cell Tumour (TGCT)) and NCT03768063 (A Study in Patients Previously Enrolled in a Genentech and/or F. Hoffmann-La Roche Ltd Sponsored Atezolizumab Study), in tenosynovial giant cell tumour. The largest, NCT03768063, plans to enrol 1000 participants with primary completion expected 2028-07-05. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Emactuzumab","url":"https://clinicaltrials.gov/search?intr=Emactuzumab"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["tenosynovial-giant-cell-tumour"],"sections":[],"technologies":[],"targets":["csf1r"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05417789","nct03768063"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Emactuzumab is given as five intravenous infusions once every two weeks; a trial exclusion criterion (prior therapy targeting CSF-1 or CSF-1R) indicates it is a CSF-1R-directed antibody used to treat tenosynovial giant cell tumour by disrupting CSF-1/CSF-1R signalling.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"emapalumab","kind":"drug","name":"Emapalumab","aka":["Emapalumab-lzsg","NI-0501"],"tldr":"Emapalumab switches off interferon gamma, the signal behind the runaway immune activation of haemophagocytic lymphohistiocytosis (HLH), a condition that can also be triggered by lymphoma and by CAR-T therapy.","summary":"Emapalumab is a monoclonal antibody against interferon gamma, approved in the United States in 2018 for primary haemophagocytic lymphohistiocytosis (HLH) that is refractory, recurrent or progressive, or where conventional HLH therapy is not tolerated; the label was later extended to HLH and macrophage activation syndrome in Still's disease. HLH matters in oncology because lymphomas and CAR-T cell therapy can set it off, and treatment protocols borrow the etoposide and dexamethasone used for the primary form. Use of emapalumab in these secondary, cancer-linked forms is off label and reported in case series rather than trials.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Emapalumab","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=emapalumab"},{"label":"Drugs@FDA BLA 761107","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761107"}],"tags":["gap-fill","supportive-care"],"related":[],"cancers":["histiocytoses","dlbcl"],"sections":[],"technologies":["supportive-care","car-t"],"targets":[],"drugs":[],"companies":["sobi"],"institutions":[],"pathways":[],"terms":["crs"],"trials":["nct06550141","nct07567014"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Gamifant","modality":"Anti-interferon-gamma monoclonal antibody","mechanism":"Binds free and receptor-bound interferon gamma and neutralises it, damping the macrophage and T-cell activation loop that drives haemophagocytic lymphohistiocytosis.","approvals":[{"region":"US","year":2018,"indication":"Primary haemophagocytic lymphohistiocytosis, refractory, recurrent or progressive, or intolerant of conventional therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"emavusertib","kind":"drug","name":"Emavusertib","aka":["CA-4948"],"tldr":"Emavusertib is an oral serine/threonine kinase inhibitor from Curis, Inc., in registered phase 2 trials for chronic lymphocytic leukaemia, brain and spinal cord tumours.","summary":"Emavusertib is listed on ClinicalTrials.gov as an intervention in 2 registered phase 2 trials sponsored by Curis, Inc., in chronic lymphocytic leukaemia, brain and spinal cord tumours. A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Emavusertib","url":"https://clinicaltrials.gov/search?intr=Emavusertib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["cll","brain-tumours"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["curis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07271667","nct03328078"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral serine/threonine kinase inhibitor","mechanism":"A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"enasidenib","kind":"drug","name":"Enasidenib","aka":[],"tldr":"Enasidenib is the IDH2 counterpart of ivosidenib, approved in the US for relapsed disease but never approved in Europe after it failed to extend survival in a confirmatory trial.","summary":"Approved 2017 (US) for relapsed/refractory IDH2-mutated AML on a single-arm study (CR+CRh ~23%). The phase 3 IDHENTIFY trial did not improve OS versus conventional care; the EMA application was withdrawn (2019), and BMS later announced market withdrawal in some territories while maintaining US availability. Illustrates the gap between response rate and survival endpoints in relapsed AML.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Enasidenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Enasidenib"}],"tags":["confirmatory-trial-negative"],"related":["idh2-mutation"],"cancers":["aml","aml-idh","sinonasal-undifferentiated-carcinoma"],"sections":[],"technologies":["epigenetic-drugs","idh-inhibitors"],"targets":["idh"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Idhifa","modality":"Small-molecule IDH2 inhibitor","mechanism":"Allosteric inhibitor of mutant IDH2 (R140Q and R172K), lowering 2-HG and inducing differentiation.","approvals":[{"region":"US","year":2017,"indication":"Relapsed/refractory IDH2-mutated AML"}],"mechanismSteps":["Mutant IDH2 produces 2-HG in mitochondria","Enasidenib binds the mutant dimer and blocks 2-HG","Differentiation block lifts; blasts mature over weeks","Response is often gradual, without marrow aplasia"],"dosing":{"route":"Oral","schedule":"100 mg once daily until progression (minimum 6 months)","monitoring":"Differentiation syndrome (boxed warning), bilirubin (indirect hyperbilirubinaemia from UGT1A1 inhibition)","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Idhifa"},"toxicity":[{"event":"Differentiation syndrome","anyGradePct":14,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Idhifa"},{"event":"Indirect hyperbilirubinaemia","anyGradePct":81},{"event":"Nausea","anyGradePct":50}],"access":[],"regulatoryEvents":[{"date":"2017-08-01","type":"approval","region":"US","note":"Single-arm AG221-C-001"},{"date":"2019-12-01","type":"withdrawal","region":"EU","note":"Marketing application withdrawn after EMA objections on efficacy","source":"https://www.ema.europa.eu/en/documents/medicine-qa/questions-and-answers-withdrawal-application-marketing-authorisation-idhifa-enasidenib_en.pdf"}]},{"id":"enb003","kind":"drug","name":"ENB003","aka":[],"tldr":"ENB003 is a small-molecule antagonist from ENB Therapeutics, Inc, in registered phase 2 trials for melanoma.","summary":"ENB003 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by ENB Therapeutics, Inc, in melanoma. Described in the registry record as a nb003 is selective endothelin b receptor antagonist; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of ENB003","url":"https://clinicaltrials.gov/search?intr=ENB003"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["enb-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04205227"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"ENB003","modality":"Small-molecule antagonist","mechanism":"Described in the registry record as a nb003 is selective endothelin b receptor antagonist; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"encorafenib","kind":"drug","name":"Encorafenib","aka":[],"tldr":"Encorafenib is a BRAF inhibitor that, with cetuximab and chemotherapy, became first-line standard for BRAF-mutant colorectal cancer in 2026.","summary":"Encorafenib is an ATP-competitive BRAF V600 inhibitor with a long target residence time, giving sustained pathway suppression. It is used with binimetinib in BRAF V600 melanoma (COLUMBUS, approved 2018), with cetuximab in previously treated BRAF V600E colorectal cancer (BEACON, 2020), and with cetuximab plus FOLFOX first line for BRAF V600E metastatic colorectal cancer after BREAKWATER (accelerated approval December 2024, full approval 2026 with an overall survival benefit); it is also used with binimetinib in BRAF V600E NSCLC. In colorectal cancer the EGFR antibody is essential because BRAF blockade alone triggers feedback reactivation through EGFR. Dosing is 450 mg daily with binimetinib or 300 mg daily with cetuximab; new primary skin cancers are a class risk. For a newcomer: the BRAF drug that finally worked in bowel cancer once paired with the right partners.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Encorafenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Encorafenib"},{"label":"NICE TA668: encorafenib plus cetuximab for previously treated BRAF V600E metastatic colorectal cancer","url":"https://www.nice.org.uk/guidance/ta668"},{"label":"EMA Braftovi","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/braftovi"},{"label":"FDA oncology approval notifications","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications"}],"tags":[],"related":["braf-v600e"],"cancers":["colorectal","melanoma","nsclc","braf-v600e-colorectal","braf-v600e-nsclc","braf-v600-melanoma","advanced-melanoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf","egfr"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05270044","nct04657991","nct05217446","nct05195632","swog-s1406"],"people":[],"bottlenecks":[],"keyPapers":["paper-venderbosch-mmr-braf-metastatic-colorectal-pooled-ccr-2014","paper-jones-non-v600-braf-colorectal-jco-2017"],"journals":[],"dependsOn":[],"notes":["Colorectal cancer biomarkers: the threshold is BRAF V600E specifically, present in 6 to 18% of tumours by cohort and 8.2% of first-line metastatic patients. Non-V600 BRAF mutations, 22% of all BRAF mutations found on sequencing, are not covered: they signal differently and carry a median overall survival of 60.7 months rather than 11.4 (Jones 2017)."],"brand":"Braftovi","modality":"Small-molecule kinase inhibitor (BRAF)","mechanism":"ATP-competitive BRAF V600 inhibitor with long target residence time.","approvals":[{"region":"US","year":2018,"indication":"BRAF V600 melanoma with binimetinib"},{"region":"US","year":2020,"indication":"BRAF V600E mCRC with cetuximab, previously treated"},{"region":"US","year":2026,"indication":"First-line BRAF V600E mCRC with cetuximab and chemotherapy"},{"region":"US","year":2024,"indication":"Metastatic colorectal cancer with a BRAF V600E mutation, with cetuximab and mFOLFOX6, previously untreated","note":"Accelerated approval on 20 December 2024 on BREAKWATER."},{"region":"US","year":2026,"indication":"Metastatic colorectal cancer with a BRAF V600E mutation","note":"Traditional approval granted 24 February 2026, converting the accelerated approval."},{"region":"EU","year":2018,"indication":"BRAF V600E-mutant metastatic colorectal cancer, with cetuximab","note":"Braftovi marketing authorisation issued 19 September 2018; the EMA product page lists metastatic colorectal cancer with the BRAF V600E mutation among its uses alongside melanoma and non-small-cell lung cancer."},{"region":"England (NICE)","year":2021,"indication":"BRAF V600E mutation-positive metastatic colorectal cancer after previous systemic treatment, with cetuximab","note":"TA668, published 6 January 2021, subject to the commercial arrangements. There is no NICE recommendation for the first-line triplet with chemotherapy."}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of BRAF V600E","Phosphorylation of downstream substrates stops","Long target residence time; paired with MEK or EGFR blockade to prevent pathway reactivation","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"450 mg once daily with binimetinib (melanoma); 300 mg once daily with cetuximab ± FOLFOX (colorectal)","modifications":"Reduce for uveitis, QTc, hepatotoxicity","monitoring":"Dermatologic exams (new primary skin cancers), ECG, LFTs, eye exams","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Fatigue"},{"event":"Nausea"},{"event":"Diarrhoea"},{"event":"Vomiting"},{"event":"Arthralgia"},{"event":"Rash"},{"event":"Dermatologic reactions"},{"event":"New primary cutaneous malignancies"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.pfizeroncologytogether.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with binimetinib in BRAF V600 melanoma (TA562) and with cetuximab in BRAF V600E CRC (TA668)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2018-06-27","type":"approval","region":"US","note":"BRAF V600 melanoma with binimetinib (COLUMBUS)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-04-08","type":"approval","region":"US","note":"BRAF V600E metastatic CRC with cetuximab after prior therapy (BEACON)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-10-11","type":"approval","region":"US","note":"BRAF V600E NSCLC with binimetinib","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-12-20","type":"accelerated-approval","region":"US","note":"First-line BRAF V600E mCRC with cetuximab and mFOLFOX6 (BREAKWATER, accelerated)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with cetuximab and mFOLFOX6, for the treatment of patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-approved test"},{"date":"2026-Q1","type":"approval","region":"US","note":"Full approval in first-line BRAF V600E mCRC with OS benefit","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-02-24","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 1.2 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-encorafenib-cetuximab-and-mfolfox6-metastatic-colorectal-cancer-braf","indication":"In combination with cetuximab and mFOLFOX6, for the treatment of patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation, as detected by an FDA-approved test"}]},{"id":"endopredict","kind":"drug","name":"EndoPredict","aka":[],"tldr":"A 12-gene test that combines gene activity with tumour size and lymph nodes to say whether hormone therapy alone is enough, and whether to continue it beyond five years.","summary":"EndoPredict (Sividon, acquired by Myriad Genetics in 2016) is CE-marked and validated in the ABCSG-6 and ABCSG-8 trial cohorts and in TransATAC, where EPclin identified a low-risk group with about 4% distant recurrence at 10 years on endocrine therapy alone and predicted late recurrence between years five and ten. NICE DG34 recommends it for guiding chemotherapy decisions in the NHS; it is offered in the US as a laboratory-developed test. Because it includes clinical variables, EPclin outperformed purely molecular scores in head-to-head comparisons on the TransATAC cohort.","status":"established","asOf":"2026-09-10","links":[{"label":"NICE DG34","url":"https://www.nice.org.uk/guidance/dg34"},{"label":"Myriad: EndoPredict","url":"https://myriad.com/oncology/endopredict/"}],"tags":["test"],"related":["oncotype-dx","prosigna"],"cancers":["breast-hr-positive"],"sections":[],"technologies":["rna-seq"],"targets":[],"drugs":[],"companies":["myriad-genetics"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: EPclin low means chemotherapy can safely be omitted and the case for extended hormone therapy is weak; EPclin high means the opposite."],"brand":"EndoPredict","modality":"Gene-expression prognostic assay (12-gene EPclin score)","mechanism":"RT-PCR of eight cancer genes and three reference genes gives a molecular score, combined with tumour size and nodal status into the EPclin score predicting 10-year distant recurrence.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"enfortumab-vedotin","kind":"drug","name":"Enfortumab vedotin","aka":[],"tldr":"Enfortumab vedotin is an ADC against Nectin-4 that, combined with pembrolizumab, nearly doubled survival in advanced bladder cancer.","summary":"EV-302 (2023): enfortumab vedotin plus pembrolizumab versus platinum chemotherapy in first-line advanced urothelial cancer, OS 31.5 vs 16.1 months (HR 0.47). Now the global standard. Being tested in muscle-invasive bladder cancer (EV-303/304, positive in 2025). Rash, neuropathy, and hyperglycaemia are class effects.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Enfortumab_vedotin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Enfortumab%20vedotin"}],"tags":[],"related":[],"cancers":["urothelial","muscle-invasive-bladder-cancer"],"sections":[],"technologies":["adc"],"targets":["nectin4"],"drugs":[],"companies":["astellas","pfizer"],"institutions":[],"pathways":[],"terms":["mc-vc-pabc"],"trials":["ev-302","nct06483334","nct07815665","nct07720284","nct07475806","nct06305767","nct07421700","nct07566156","nct04225117","nct04960709","nct07287995","nct06493552"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Padcev","modality":"ADC","payload":"MMAE (tubulin inhibitor), DAR ~3.8","linker":"mc-vc-PABC, protease-cleavable","mechanism":"Fully human anti-Nectin-4 IgG1; MMAE released by cathepsin B; bystander killing.","approvals":[{"region":"US","year":2019,"indication":"Advanced urothelial cancer after platinum and PD-1/PD-L1 (accelerated)"},{"region":"US","year":2023,"indication":"First-line advanced urothelial cancer with pembrolizumab"},{"region":"EU","year":2022,"indication":"mUC after platinum + PD-1; 1L with pembrolizumab 2024"}],"mechanismSteps":["Antibody binds Nectin-4 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","MMAE is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion over 30 min","schedule":"1.25 mg/kg (max 125 mg) days 1, 8, 15 of 28-day cycle (monotherapy) or days 1 and 8 of 21-day cycle with pembrolizumab","modifications":"Hold for grade 3 rash or hyperglycaemia; discontinue for SJS/TEN or grade ≥3 neuropathy","monitoring":"Skin, glucose (HbA1c/BMI risk), neuropathy, eyes","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed"},"toxicity":[{"event":"Rash","anyGradePct":68,"grade3PlusPct":15,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Peripheral neuropathy","anyGradePct":67,"grade3PlusPct":8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Fatigue","anyGradePct":51,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Pruritus","anyGradePct":41,"grade3PlusPct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Diarrhoea","anyGradePct":38,"grade3PlusPct":4.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Alopecia","anyGradePct":35,"grade3PlusPct":0.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Decreased appetite","anyGradePct":33,"grade3PlusPct":1.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Weight decrease","anyGradePct":33,"grade3PlusPct":3.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Dry eye","anyGradePct":24,"grade3PlusPct":0,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"},{"event":"Hyperglycaemia (grade 3-4 glucose elevation)","grade3PlusPct":14,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b5631d3e-4604-4363-8f20-11dfc5a4a8ed","note":"EV-302 with pembrolizumab, n=440"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.padcev.com/support","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with pembrolizumab for untreated advanced urothelial cancer (2024)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"},{"country":"EU","reimbursement":"EMA approved 2022 (monotherapy), 2024 (with pembrolizumab)","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2018-03","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2019-12-18","type":"accelerated-approval","region":"US","note":"Accelerated approval, advanced urothelial cancer after platinum and PD-1/PD-L1 (EV-201)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adults with locally advanced or metastatic urothelial cancer who received a PD-1 or PD-L1 inhibitor and a platinum-containing chemotherapy in the neoadjuvant, locally advanced, or metastatic setting"},{"date":"2021-07-09","type":"conversion","region":"US","note":"Full approval and cisplatin-ineligible expansion (EV-301)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adults with locally advanced or metastatic urothelial cancer who received a PD-1 or PD-L1 inhibitor and a platinum-containing chemotherapy in the neoadjuvant, locally advanced, or metastatic setting"},{"date":"2023-04-03","type":"accelerated-approval","region":"US","note":"Accelerated approval with pembrolizumab, cisplatin-ineligible first line","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with pembrolizumab for the treatment of adult patients with locally advanced or metastatic urothelial cancer who are not eligible for cisplatin-containing chemotherapy"},{"date":"2023-12-15","type":"conversion","region":"US","note":"Full approval with pembrolizumab, all first-line advanced urothelial cancer (EV-302)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with pembrolizumab for the treatment of adult patients with locally advanced or metastatic urothelial cancer who are not eligible for cisplatin-containing chemotherapy"},{"date":"2025-Q4","type":"filing","region":"US","note":"Muscle-invasive bladder cancer (EV-303/304) submissions following positive results","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-07-10","type":"approval","region":"US","note":"Muscle-invasive bladder cancer with pembrolizumab, with or without berahyaluronidase alfa","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-or-pembrolizumab-and-berahyaluronidase-alfa-pmph-each-enfortumab-vedotin"}]},{"id":"englumafusp-alfa","kind":"drug","name":"Englumafusp alfa","aka":["A CD19 Targeted 4-1BB Ligand; RO7227166"],"tldr":"Englumafusp alfa is an experimental fusion protein from Hoffmann-La Roche in phase 2 trials for hodgkin lymphoma, aimed at CD19.","summary":"Englumafusp alfa (RO7227166) is a fusion protein developed by Hoffmann-La Roche. Its target is CD19 (the sponsor names CD19 x 4-1BBL). The sponsor states: Englumafusp alfa (RO7227166) is a CD19-targeted 4-1BB ligand fusion protein that costimulates T cells, given by intravenous infusion three-weekly in combination with obinutuzumab and/or glofitamab in relapsed/refractory B-cell non-Hodgkin lymphoma. ClinicalTrials.gov describes the intervention as: Englumafusp alfa will be administered by intravenous (IV) infusion three-weekly (Q3W) in combination with a fixed dose of obinutuzumab (Part I) and in combination with a fixed dose of glofitamab (Part II and Part III). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in hodgkin lymphoma. The largest, NCT04077723, plans to enrol 498 participants with primary completion expected 2027-03-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Englumafusp alfa","url":"https://clinicaltrials.gov/search?intr=RO7227166"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":["cd19"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04077723"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"RO7227166","modality":"fusion protein","mechanism":"Englumafusp alfa (RO7227166) is a CD19-targeted 4-1BB ligand fusion protein that costimulates T cells, given by intravenous infusion three-weekly in combination with obinutuzumab and/or glofitamab in relapsed/refractory B-cell non-Hodgkin lymphoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ensartinib","kind":"drug","name":"Ensartinib","aka":[],"tldr":"A Chinese-developed ALK pill approved in the US in December 2024 for first-line ALK-positive lung cancer.","summary":"Ensartinib is a potent second-generation ALK tyrosine kinase inhibitor with activity against crizotinib-resistant mutations and penetration into the central nervous system. In the eXalt3 trial (JAMA Oncology 2021) it beat crizotinib in ALK-TKI-naive NSCLC, with median progression-free survival of 25.8 versus 12.7 months. It was approved in China in 2020 for use after crizotinib and in 2022 for first-line treatment, and the FDA approved it in December 2024 for ALK-inhibitor-naive locally advanced or metastatic disease, developed in the US by Xcovery. Rash is its characteristic toxicity, in contrast to the pulmonary events of brigatinib or the metabolic effects of lorlatinib. It has not been compared directly with alectinib or lorlatinib, so its place among first-line ALK drugs is still being worked out. Ensartinib adds a further option to an already crowded class.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Ensartinib","links":[{"label":"eXalt3 (JAMA Oncol 2021)","url":"https://doi.org/10.1001/jamaoncol.2021.3523"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ensartinib"},{"label":"FDA novel approvals 2024","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2024"}],"tags":["gap-fill"],"related":["alk-fusion"],"cancers":["nsclc","alk-positive-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["alk"],"drugs":[],"companies":["xcovery","betta"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02767804","nct07448116"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ensacove","modality":"Small-molecule ALK TKI (second generation)","mechanism":"Potent ALK inhibitor with activity against crizotinib-resistant mutations and CNS penetration.","approvals":[{"region":"CN","year":2020,"indication":"ALK-positive NSCLC after crizotinib"},{"region":"US","year":2024,"indication":"ALK-positive locally advanced or metastatic NSCLC, ALK-inhibitor-naive"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"entrectinib","kind":"drug","name":"Entrectinib","aka":[],"tldr":"Entrectinib (Rozlytrek) is a pill for NTRK-fusion cancers and ROS1 lung cancer that reaches brain metastases.","summary":"Entrectinib is a multikinase inhibitor of TRKA, TRKB, TRKC, ROS1 and ALK that was designed to cross the blood-brain barrier. It is a tumour-agnostic drug for solid tumours with NTRK gene fusions, which occur across salivary gland tumours, sarcomas, colorectal cancer and many other types, and for ROS1-positive metastatic NSCLC. An integrated analysis of STARTRK-2, STARTRK-1 and ALKA showed a 57% response rate in NTRK-fusion tumours and 77% in ROS1-positive NSCLC, with activity against brain metastases. Japan approved it in June 2019 for NTRK-fusion tumours, the US in August 2019 for both indications and the EU in 2020; the paediatric label was extended in 2023 to infants from one month of age. Weight gain, dizziness, cognitive effects and cardiac failure are class-related TRK toxicities. Entrectinib treats cancers by their fusion gene rather than their organ of origin.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Entrectinib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=entrectinib"},{"label":"NICE TA1118: entrectinib for NTRK fusion-positive solid tumours, terminated appraisal (7 January 2026)","url":"https://www.nice.org.uk/guidance/ta1118"},{"label":"NICE TA644 (12 August 2020), replaced","url":"https://www.nice.org.uk/guidance/ta644"},{"label":"EMA Rozlytrek product page","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/rozlytrek"},{"label":"NICE TA643: entrectinib for treating ROS1-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta643"}],"tags":["gap-fill"],"related":["ros1-fusion","ntrk-fusion"],"cancers":["nsclc","salivary-gland","sarcoma","colorectal","ros1-positive-nsclc","ntrk-fusion-nsclc","pancreatic","kras-wild-type-pdac","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["ntrk","ros1","alk"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["tumour-agnostic","gene-fusion","blood-brain-barrier"],"trials":["nct05170204","nct02568267","nct04302025","nct04603807","nct02650401","profile-1001-ros1"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: entrectinib's tumour-agnostic NTRK indication (FDA accelerated approval; EU marketing authorisation 31 July 2020) applies to the rare NTRK fusion-positive pancreatic tumours, but in England the Cancer Drugs Fund route closed with the terminated TA1118 in January 2026, leaving larotrectinib (TA630) as the funded option."],"brand":"Rozlytrek","modality":"Small-molecule TRK/ROS1/ALK TKI (CNS-penetrant)","mechanism":"Multikinase inhibitor of TRKA/B/C, ROS1 and ALK designed to cross the blood-brain barrier.","approvals":[{"region":"JP","year":2019,"indication":"NTRK fusion solid tumours"},{"region":"US","year":2019,"indication":"ROS1-positive metastatic NSCLC; NTRK fusion solid tumours (≥12 years, extended to ≥1 month 2023)"},{"region":"EU","year":2020,"indication":"Same"},{"region":"UK","year":2020,"indication":"NTRK fusion-positive solid tumours: NICE TA644 (12 August 2020) recommended entrectinib within the Cancer Drugs Fund; it was replaced by TA1118 (terminated appraisal, 7 January 2026) because the company did not make a submission, and only people already on treatment continue","note":"https://www.nice.org.uk/guidance/terminated/ta1118"},{"region":"England (NICE)","year":2020,"indication":"NTRK fusion-positive solid tumours, including colorectal cancer","note":"TA644, published 12 August 2020, withdrawn: replaced by TA1118, which NICE terminated on 7 January 2026 with no complete evidence submission. People already treated through the Cancer Drugs Fund may continue."},{"region":"England (NICE)","year":2020,"indication":"ROS1-positive advanced non-small-cell lung cancer in patients who have not had a ROS1 inhibitor","note":"TA643, published 12 August 2020, subject to the commercial arrangement. This is a separate appraisal from the tumour-agnostic NTRK guidance."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2019-08-15","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 7.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-expands-pediatric-indication-entrectinib-and-approves-new-pellet-formulation","indication":"Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy . *"},{"date":"2023-10-20","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 2.9 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-expands-pediatric-indication-entrectinib-and-approves-new-pellet-formulation","indication":"Formulation: Adult and pediatric patients older than 1 month of age with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation as detected by an FDA-approved test, are metastatic or where surgical resection is likely to result in severe morbidity and have progressed following treatment or have no satisfactory alternative therapy ."}]},{"id":"env-501","kind":"drug","name":"ENV-501","aka":[],"tldr":"ENV-501 is an experimental antibody-drug conjugate from Hummingbird Bioscience in phase 2 trials for melanoma, non-small-cell lung cancer and HR-positive / HER2-negative breast cancer, aimed at HER3.","summary":"ENV-501 is an antibody-drug conjugate developed by Hummingbird Bioscience. Its target is HER3. ClinicalTrials.gov describes the intervention as: ENV-501 is a HER3-targeted antibody-drug conjugate (ADC) with a humanized monoclonal antibody (mAb) conjugated with a chemotherapeutic payload via a linker. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in melanoma, non-small-cell lung cancer and HR-positive / HER2-negative breast cancer. The largest, NCT06956690, plans to enrol 180 participants with primary completion expected 2027-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ENV-501","url":"https://clinicaltrials.gov/search?intr=ENV-501"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma","nsclc","breast-hr-positive"],"sections":[],"technologies":[],"targets":["her3"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06956690"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Antibody-drug conjugate directed at HER3, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"envafolimab","kind":"drug","name":"Envafolimab","aka":[],"tldr":"Envafolimab is the first PD-L1 antibody given as a quick injection under the skin rather than an infusion, approved in China for advanced tumours with mismatch-repair deficiency.","summary":"Approved by the NMPA in November 2021 for previously treated MSI-H or dMMR advanced solid tumours, on a single-arm study across colorectal and other cancers, the first subcutaneous PD-(L)1 antibody approved anywhere. Developed by Alphamab Oncology with 3D Medicines in China; TRACON Pharmaceuticals held North American rights for a sarcoma programme that did not succeed. Its convenience rather than potency is the point: a 30-second injection in a country where infusion chairs are scarce outside big cities.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Envafolimab","links":[{"label":"Alphamab Oncology","url":"https://www.alphamabonc.com/en/"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["colorectal","cancer-of-unknown-primary"],"sections":[],"technologies":["checkpoint-inhibitor","monoclonal-antibody"],"targets":["pdl1"],"drugs":[],"companies":["alphamab","3d-medicines","tracon"],"institutions":[],"pathways":["pd1-checkpoint"],"terms":["msi"],"trials":["nct05112991","nct05024214","nct04891198","nct03478488","nct03667170","nct06123754"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Enweida","code":"KN035","modality":"Single-domain antibody (anti-PD-L1, subcutaneous)","mechanism":"Camelid-derived humanised single-domain anti-PD-L1 antibody fused to an IgG1 Fc, small enough for a rapid subcutaneous injection.","approvals":[{"region":"China","year":2021,"indication":"Previously treated MSI-H or dMMR advanced solid tumours"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"enzalutamide","kind":"drug","name":"Enzalutamide","aka":[],"tldr":"Enzalutamide is a second-generation androgen-receptor blocker that stops the receptor binding testosterone, entering the nucleus and switching on genes. It is approved at every stage of advanced prostate cancer, from rising PSA after surgery to castration-resistant disease, and fatigue, falls and memory problems are its main drawbacks.","summary":"Enzalutamide is a second-generation androgen receptor antagonist that blocks ligand binding, nuclear translocation and DNA binding, giving more complete AR blockade than older antiandrogens. It is approved across every stage of advanced prostate cancer: AFFIRM and PREVAIL (mCRPC), PROSPER (nmCRPC), ARCHES and ENZAMET (mHSPC, overall survival benefit; ARCHES OS HR 0.66) and EMBARK (high-risk biochemical recurrence, 2023, metastasis-free survival HR 0.42 with leuprolide). It is the partner in TALAPRO-2 (talazoparib) and the MEVPRO trials (mevrometostat), so the next generation of combinations is built on it. Fatigue, falls and cognitive effects are the main toxicities, which is why darolutamide or apalutamide is sometimes preferred in frail men. For a newcomer, enzalutamide is the most widely used androgen-receptor blocker, from rising PSA after surgery to late-stage disease.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Enzalutamide","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Enzalutamide"},{"label":"NICE TA712: enzalutamide for treating hormone-sensitive metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta712"},{"label":"NICE TA377: enzalutamide for treating metastatic hormone-relapsed prostate cancer before chemotherapy is indicated","url":"https://www.nice.org.uk/guidance/ta377"},{"label":"NICE TA316: enzalutamide for metastatic hormone-relapsed prostate cancer previously treated with a docetaxel-containing regimen","url":"https://www.nice.org.uk/guidance/ta316"},{"label":"NICE TA580: enzalutamide for hormone-relapsed non-metastatic prostate cancer, not recommended","url":"https://www.nice.org.uk/guidance/ta580"}],"tags":[],"related":[],"cancers":["prostate","salivary-duct-carcinoma","prostate-mhspc","prostate-nmcrpc","prostate-mcrpc","prostate-bcr","tnbc"],"sections":[],"technologies":["androgen-deprivation"],"targets":["androgen-receptor"],"drugs":[],"companies":["astellas","pfizer","zydus"],"institutions":[],"pathways":["ar-signaling"],"terms":["mcrpc-mhspc","prostate-uk-drug-approvals"],"trials":["arches","embark","talapro-2","mevpro-1","nct06629779","nct06520345","nct07028853","nct04076059","nct04446117","nct07611110","nct05422911","nct04986423","nct07287150","nct06228053","nct07230106","nct02960022","nct05367440","nct06991556","nct02861573","nct04557449","nct07198633","nct04104776","nct06702995","nct05914116","nct06863272","nct05112965","nct04821622"],"people":[],"bottlenecks":[],"keyPapers":["paper-prosper-nejm-2018","paper-traina-enzalutamide-ar-tnbc-jco-2018"],"journals":[],"dependsOn":[],"notes":["Four NICE appraisals cover enzalutamide in prostate cancer in England and one of them is a refusal. TA712 recommends enzalutamide with androgen deprivation for hormone-sensitive metastatic prostate cancer. TA377 recommends it for metastatic hormone-relapsed disease before chemotherapy is indicated. TA316 recommends it after docetaxel, and states that use after abiraterone is not covered by that guidance. TA580 does not recommend it for high-risk hormone-relapsed non-metastatic prostate cancer, although apalutamide (TA740) and darolutamide (TA660) are recommended in that population. TA1130 recommends talazoparib with enzalutamide for untreated hormone-relapsed metastatic disease under tight conditions."],"brand":"Xtandi","modality":"Small-molecule AR antagonist","mechanism":"Second-generation AR antagonist blocking ligand binding, nuclear translocation, and DNA binding.","approvals":[{"region":"US","year":2012,"indication":"mCRPC after docetaxel (pre-chemo 2014)"},{"region":"US","year":2018,"indication":"Non-metastatic CRPC"},{"region":"US","year":2019,"indication":"mHSPC (ARCHES)"},{"region":"US","year":2023,"indication":"High-risk biochemical recurrence (EMBARK)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ep0031","kind":"drug","name":"EP0031","aka":[],"tldr":"EP0031 is an experimental small-molecule drug from Ellipses Pharma in phase 2 trials for non-small-cell lung cancer, aimed at RET.","summary":"EP0031 is a small-molecule drug developed by Ellipses Pharma. Its target is RET (the sponsor names RET). ClinicalTrials.gov describes the intervention as: EP0031 is a potent next-generation selective RET-inhibitor (SRI). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT05443126, plans to enrol 265 participants with primary completion expected 2027-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of EP0031","url":"https://clinicaltrials.gov/search?intr=EP0031"},{"label":"Sponsor page","url":"https://www.ellipses.life"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["ret"],"drugs":[],"companies":["ellipses-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05443126"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Small-molecule drug directed at RET, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ep0062","kind":"drug","name":"EP0062","aka":[],"tldr":"EP0062 is an experimental small-molecule drug from Ellipses Pharma in phase 2 trials for HR-positive / HER2-negative breast cancer, aimed at Androgen receptor.","summary":"EP0062 is a small-molecule drug developed by Ellipses Pharma. Its target is Androgen receptor. ClinicalTrials.gov describes the intervention as: EP0062 is an orally administered investigational selective androgen receptor modulator (SARM). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in HR-positive / HER2-negative breast cancer. The largest, NCT05573126, plans to enrol 95 participants with primary completion expected 2028-02. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of EP0062","url":"https://clinicaltrials.gov/search?intr=EP0062"},{"label":"Sponsor page","url":"https://www.ellipses.life"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":["androgen-receptor"],"drugs":[],"companies":["ellipses-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05573126"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Small-molecule drug directed at Androgen receptor, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"epacadostat","kind":"drug","name":"Epacadostat","aka":[],"tldr":"An enzyme blocker meant to stop tumours starving T cells of tryptophan. Its 2018 phase 3 failure ended an entire class overnight.","summary":"IDO1 degrades tryptophan to kynurenine, suppressing T cells. Epacadostat plus pembrolizumab produced ~55% response rates in a phase 1/2 melanoma cohort. ECHO-301/KEYNOTE-252 (n=706, first-line melanoma) showed no PFS or OS difference (2018). Incyte, Merck, BMS, and others halted more than a dozen IDO1 trials within weeks; the drug may never have achieved adequate pathway inhibition in tumours.\n\nLesson: pharmacodynamic proof of target engagement in the tumour should precede phase 3; a large uncontrolled response rate on a pembrolizumab backbone in melanoma is not evidence of added benefit.","status":"negative","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Epacadostat","links":[{"label":"ECHO-301/KEYNOTE-252 (Lancet Oncol 2019)","url":"https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(19)30274-8/fulltext"}],"tags":["failure","lesson:phase-2-mirage"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["ido1"],"drugs":["pembrolizumab"],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03260894","nct03358472","nct03348904"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"INCB024360","modality":"Small molecule (IDO1 inhibitor)","mechanism":"Selective, reversible IDO1 enzyme inhibitor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"epcoritamab","kind":"drug","name":"Epcoritamab","aka":[],"tldr":"Epcoritamab is an under-the-skin injection that pulls T cells onto lymphoma cells; it is approved for relapsed large B-cell and follicular lymphoma, and moving toward first line.","summary":"EPCORE NHL-1: ORR 63%, CR 39% in DLBCL after ≥2 lines; 3-year data show ~53% of complete responders in durable remission. Accelerated approval May 2023 (DLBCL) and 2024 (follicular lymphoma). Phase 3 EPCORE DLBCL-1 (R/R, vs investigator's choice, topline January 2026): PFS HR 0.74 but OS HR 0.96, not significant, so the US primary endpoint was missed. EPCORE DLBCL-2 (frontline, with R-CHOP) topline 2026 and EHA 2026 presentation; EPCORE FL-1 positive in follicular lymphoma. Genmab/AbbVie; 2025 label change allowed outpatient step-up dosing.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Epcoritamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Epcoritamab"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["t-cell-engager"],"targets":["cd20","cd3"],"drugs":[],"companies":["genmab","abbvie"],"institutions":[],"pathways":[],"terms":["crs","icans","step-up-dosing"],"trials":["epcore-nhl-1","epcore-dlbcl-1","epcore-dlbcl-2","nct07226752","nct06508658","nct06191744","nct05409066","nct06090539","nct04542824","nct05201248","nct05451810","nct05283720","nct04623541"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Epkinly","modality":"Bispecific T-cell engager (CD20×CD3)","mechanism":"DuoBody IgG1 binding CD20 on B cells and CD3 on T cells, forming a cytolytic synapse; subcutaneous step-up dosing to limit CRS.","approvals":[{"region":"US","year":2023,"indication":"Relapsed/refractory DLBCL or high-grade B-cell lymphoma after ≥2 lines (accelerated)"},{"region":"US","year":2024,"indication":"Relapsed/refractory follicular lymphoma after ≥2 lines (accelerated; full 2025 with R2)"},{"region":"EU","year":2023,"indication":"EU brand Tepkinly","note":"Conditional marketing authorisation"}],"mechanismSteps":["Subcutaneous injection; slow absorption blunts cytokine peaks","One arm binds CD20 on the lymphoma cell","Other arm engages CD3 on any nearby T cell","T cell activates, releases perforin and granzymes","Serial killing of CD20+ cells; CRS mainly in cycle 1"],"dosing":{"route":"Subcutaneous","schedule":"Step-up 0.16 mg (day 1), 0.8 mg (day 8), then 48 mg weekly cycles 1-3, every 2 weeks cycles 4-9, then every 4 weeks until progression","monitoring":"CRS and ICANS after the first full dose; infections; CMV","source":"https://www.epkinly.com"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":49.7,"grade3PlusPct":2.5,"note":"EPCORE NHL-1 DLBCL cohort"},{"event":"ICANS","anyGradePct":6.4,"grade3PlusPct":0.6},{"event":"Neutropenia","grade3PlusPct":14.6},{"event":"Infections","grade3PlusPct":14.6}],"access":[],"regulatoryEvents":[{"date":"2023-05-19","type":"accelerated-approval","region":"US","note":"Accelerated approval in DLBCL (EPCORE NHL-1) The confirmatory requirement was still open 3.3 years later, when the FDA's table was read.","indication":"Adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma after two or more lines of systemic therapy."},{"date":"2024-06-24","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-epcoritamab-bysp-relapsed-or-refractory-follicular-lymphoma","indication":"Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy"},{"date":"2025-11-18","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 1.4 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-epcoritamab-bysp-relapsed-or-refractory-follicular-lymphoma","indication":"Treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy"},{"date":"2026-01-16","type":"filing","region":"US","note":"EPCORE DLBCL-1 topline: PFS met, OS (US primary endpoint) not met","source":"https://news.abbvie.com/2026-01-16-AbbVie-Announces-Topline-Results-for-Epcoritamab-DuoBody-R-CD3xCD20-from-Phase-3-EPCORE-R-DLBCL-1-Trial-in-Patients-with-Relapsed-Refractory-Diffuse-Large-B-cell-Lymphoma-DLBCL"}]},{"id":"epirubicin","kind":"drug","name":"Epirubicin","aka":["Pharmorubicin"],"tldr":"Epirubicin (Ellence) is a close relative of doxorubicin used mainly after breast cancer surgery when lymph nodes are involved, and in stomach cancer regimens; it is a little kinder to the heart.","summary":"Epirubicin was approved by the FDA in 1999 as a component of adjuvant therapy for axillary node-positive breast cancer, based on the Canadian MA.5 (CEF versus CMF) and French FASG trials in which epirubicin-containing regimens improved relapse-free and overall survival. Outside the US it has been used far more widely since the 1980s, including in the ECF/EOX regimens for gastro-oesophageal cancer (REAL-2) and in FEC-docetaxel sequences for breast cancer. Cardiotoxicity is cumulative-dose dependent (the label caps lifetime exposure at 900 mg/m2), secondary acute myeloid leukaemia is a recognised late effect, and it is a vesicant.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Epirubicin","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=epirubicin"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/epirubicinhydrochloride"}],"tags":["nci-list","generic"],"related":["doxorubicin"],"cancers":["breast-hr-positive","breast-her2-positive","tnbc","gastric"],"sections":[],"technologies":["cytotoxic-chemotherapy","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03725059","nct06227117","nct06389006","nct06966700","nct06797635","nct06112379"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ellence","modality":"Anthracycline (topoisomerase II inhibitor)","mechanism":"4'-epimer of doxorubicin; intercalates DNA and inhibits topoisomerase II, with less cardiotoxicity per milligram than doxorubicin.","approvals":[{"region":"US","year":1999,"indication":"Adjuvant therapy for axillary node-positive breast cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"epoetin-alfa","kind":"drug","name":"Epoetin alfa","aka":["Retacrit (epoetin alfa-epbx)","Eprex","Binocrit","Abseamed","Erythropoietin","Erythropoiesis-stimulating agent (ESA)"],"tldr":"Epoetin alfa is a manufactured version of the kidney hormone that tells the bone marrow to make red blood cells. In cancer it treats anaemia caused by chemotherapy and reduces the need for transfusions, but it is used cautiously because it can shorten survival and cause clots.","summary":"Epoetin alfa was approved in June 1989 for anaemia of chronic kidney disease and in 1993 for anaemia due to concomitant myelosuppressive chemotherapy in non-myeloid malignancies when at least two further months of chemotherapy are planned; the biosimilar Retacrit followed in 2018 and the EU authorised the first epoetin biosimilars in 2007. Trials in the 2000s that targeted higher haemoglobin found worse survival and tumour progression in several cancers, leading to boxed warnings, the ESA APPRISE risk programme (2010 to 2017) and the current rule to use the lowest dose that avoids transfusion, never in patients treated with curative intent. Thromboembolism, hypertension and rare pure red cell aplasia are the safety concerns. Darbepoetin alfa is the long-acting alternative.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Epoetin_alfa","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=epoetin%20alfa"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/epoetinalfa"},{"label":"EPAR (Binocrit)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/binocrit"}],"tags":["nci-list","supportive"],"related":["darbepoetin-alfa"],"cancers":["mds-lower-risk"],"sections":[],"technologies":["transfusion-support"],"targets":[],"drugs":[],"companies":["amgen","johnson-johnson","pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Epogen / Procrit","modality":"Recombinant erythropoietin (erythropoiesis-stimulating agent)","supportive":true,"mechanism":"Recombinant human erythropoietin that binds the erythropoietin receptor on erythroid progenitors, stimulating red cell production by the same mechanism as the endogenous hormone.","approvals":[{"region":"US","year":1989,"indication":"Anaemia of chronic kidney disease"},{"region":"US","year":1993,"indication":"Anaemia due to concomitant myelosuppressive chemotherapy in non-myeloid malignancies"},{"region":"EU","year":2007,"indication":"First epoetin alfa biosimilars (Binocrit, Abseamed, Epoetin alfa Hexal) including chemotherapy-induced anaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"epoetin-theta","kind":"drug","name":"Epoetin theta","aka":["epoetin theta","Eporatio","Biopoin"],"tldr":"Epoetin theta is a made-to-order version of erythropoietin, the hormone that tells the marrow to make red blood cells. It is used to treat the anaemia of chemotherapy in people with non-myeloid cancers, sparing some transfusions.","summary":"Epoetin theta is authorised in the EU under two names: Biopoin (authorised 23 October 2009; marketing authorisation holder Teva GmbH) and Eporatio (authorised 29 October 2009; marketing authorisation holder Ratiopharm GmbH, a Teva company per Wikidata Q265318). Both are indicated for symptomatic anaemia associated with chronic renal failure in adults and for symptomatic anaemia in adult cancer patients with non-myeloid malignancies receiving chemotherapy. The corpus's other erythropoiesis-stimulating agents are epoetin alfa and darbepoetin alfa.","status":"approved","asOf":"2026-09-24","links":[{"label":"XM01-22 (Archives of Drug Information 2011)","url":"https://doi.org/10.1111/j.1753-5174.2011.00035.x"},{"label":"EMA: Eporatio (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/eporatio"},{"label":"EMA: Biopoin (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/biopoin"}],"tags":["ema-register","supportive"],"related":["epoetin-alfa","darbepoetin-alfa"],"cancers":[],"sections":[],"technologies":["transfusion-support"],"targets":["epor"],"drugs":[],"companies":["teva"],"institutions":[],"pathways":[],"terms":[],"trials":["xm01-22"],"people":[],"bottlenecks":[],"keyPapers":["paper-xm01-22-tjulandin-arch-drug-inf-2011"],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo content wave 5 (EMA medicines register and EPAR pages)","editedOn":"2026-09-22"},"brand":"Eporatio / Biopoin","modality":"Recombinant erythropoietin (erythropoiesis-stimulating agent)","supportive":true,"mechanism":"Recombinant human erythropoietin that acts on the erythropoietin receptor to stimulate red-cell production; used for symptomatic anaemia in adults with non-myeloid cancers receiving chemotherapy and in chronic renal failure (indication wording, EMA).","approvals":[{"region":"EU","year":2009,"indication":"Symptomatic anaemia in adult cancer patients with non-myeloid malignancies receiving chemotherapy; anaemia of chronic renal failure","note":"Biopoin authorised 23 Oct 2009 (Teva); Eporatio 29 Oct 2009 (Ratiopharm)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eprenetapopt","kind":"drug","name":"Eprenetapopt","aka":[],"tldr":"Eprenetapopt (APR-246) was a drug meant to refold mutant p53, the most common broken protein in cancer. Its phase 3 in blood cancer failed in 2020.","summary":"Eprenetapopt (APR-246) is a prodrug of methylene quinuclidinone, proposed to covalently modify mutant p53 and restore wild-type conformation. Phase 2 with azacitidine in TP53-mutant MDS reported ~50% complete remission. The phase 3 (n=154) missed its primary endpoint of complete remission rate in 2020 (33% vs 22%, not significant). Aprea pivoted away. Subsequent studies suggest much of the activity reflected glutathione depletion and oxidative stress rather than p53 refolding.\n\nLesson: TP53 remains undrugged by direct reactivation; the field moved to mutation-specific correctors (rezatapopt for Y220C) and to exploiting p53-loss dependencies (WEE1, ATR).","status":"negative","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Eprenetapopt","links":[{"label":"Aprea phase 3 topline (Dec 2020)","url":"https://www.globenewswire.com/news-release/2020/12/28/2150865/0/en/Aprea-Therapeutics-Announces-Results-of-Primary-Endpoint-from-Phase-3-Trial-of-Eprenetapopt-in-TP53-Mutant-Myelodysplastic-Syndromes-MDS.html"}],"tags":["failure","lesson:wrong-target"],"related":[],"cancers":["aml"],"sections":[],"technologies":[],"targets":["tp53"],"drugs":[],"companies":["aprea-therapeutics"],"institutions":[],"pathways":["p53-cell-cycle"],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"APR-246","modality":"Small molecule (p53 reactivator)","mechanism":"Converted to MQ, which alkylates cysteines in mutant p53 (claimed) and depletes glutathione.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"equecabtagene-autoleucel","kind":"drug","name":"Equecabtagene autoleucel","aka":[],"tldr":"Equecabtagene autoleucel is a BCMA CAR-T from IASO Bio and Innovent, approved in China in June 2023 for multiple myeloma that has returned after at least three lines of treatment. In the FUMANBA-1 trial about 96% of infused patients responded, most with deep minimal-residual-disease-negative remissions and few severe cytokine release reactions; it competes with cilta-cel and zevor-cel in China.","summary":"Developed by IASO Bio with Innovent from work at Tongji Hospital in Wuhan, and approved by the NMPA in June 2023 for relapsed or refractory multiple myeloma after three or more lines including a proteasome inhibitor and an immunomodulatory drug. The FUMANBA-1 trial (JAMA Oncology 2024) reported objective responses in about 96% of infused patients with deep, minimal residual disease-negative remissions and a low rate of severe cytokine release syndrome. It competes in China with ciltacabtagene autoleucel and zevorcabtagene autoleucel; it also holds FDA regenerative medicine advanced therapy designation.","status":"approved","asOf":"2026-09-10","links":[{"label":"IASO Biotherapeutics","url":"https://www.iasobio.com/"},{"label":"FUMANBA-1 (JAMA Oncol 2024)","url":"https://doi.org/10.1001/jamaoncol.2024.4879"},{"label":"Equecabtagene autoleucel: first approval (Mol Diagn Ther 2023)","url":"https://doi.org/10.1007/s40291-023-00673-y"}],"tags":["china"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["car-t"],"targets":["bcma"],"drugs":[],"companies":["iaso-bio","innovent"],"institutions":["tongji-hospital-wuhan"],"pathways":[],"terms":[],"trials":["fumanba-1","nct06464991"],"people":[],"bottlenecks":[],"keyPapers":["paper-li-jama-oncol","paper-keam-mol-diagn-ther"],"journals":[],"dependsOn":[],"notes":[],"brand":"Fucaso","code":"eque-cel, CT103A","modality":"Cell therapy (autologous BCMA CAR-T, fully human binder)","mechanism":"Autologous T cells expressing a fully human BCMA-directed single-chain variable fragment with 4-1BB co-stimulation, lentivirally transduced; the fully human binder is designed to reduce anti-CAR immunity and allow persistence.","approvals":[{"region":"China","year":2023,"indication":"Relapsed or refractory multiple myeloma after three or more lines including a proteasome inhibitor and an immunomodulatory agent"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"erdafitinib","kind":"drug","name":"Erdafitinib","aka":[],"tldr":"The first targeted pill for bladder cancer, for the roughly 20% of tumours with FGFR3 alterations, used after immunotherapy.","summary":"Erdafitinib is an oral pan-FGFR (1 to 4) tyrosine kinase inhibitor for the roughly 20% of urothelial cancers with FGFR3 or FGFR2 alterations, so molecular testing is required before use. It received accelerated approval in 2019 on BLC2001 (ORR 40%) and full approval in January 2024 on THOR cohort 1, where it gave OS 12.1 versus 7.8 months (HR 0.64) against chemotherapy after platinum and immunotherapy. THOR cohort 2, against pembrolizumab in immunotherapy-naive patients, showed no benefit, so the drug is positioned after a PD-1 or PD-L1 inhibitor. Hyperphosphataemia (79%), stomatitis (58%) and central serous retinopathy (17%) need monitoring, including regular eye examinations. Johnson & Johnson develops it. It is the first targeted pill in bladder cancer, and it only works when the tumour carries the matching alteration.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Erdafitinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Erdafitinib"}],"tags":[],"related":["fgfr3-alteration"],"cancers":["urothelial","muscle-invasive-bladder-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["fgfr2"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":["fgfr-signalling"],"terms":["fgfr3","ocular-toxicity"],"trials":["thor","nct05316155","nct02365597","nct03473743"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Balversa","modality":"Small-molecule kinase inhibitor (pan-FGFR)","mechanism":"Pan-FGFR (1-4) tyrosine kinase inhibitor.","approvals":[{"region":"US","year":2019,"indication":"FGFR3/2-altered advanced urothelial cancer after platinum (accelerated)"},{"region":"US","year":2024,"indication":"FGFR3-altered advanced urothelial cancer after ≥1 systemic therapy incl. PD-1/PD-L1 (full)"}],"mechanismSteps":["Oral absorption; dose titrated to serum phosphate","Blocks FGFR3 (mutated or fused) autophosphorylation","Shuts down MAPK and PI3K signalling in FGFR-driven luminal-papillary tumours","Resistance via gatekeeper mutations and pathway bypass"],"dosing":{"route":"Oral","schedule":"8 mg daily, up-titrated to 9 mg if serum phosphate <5.5 mg/dL at day 14","modifications":"Hold for phosphate >7 mg/dL or ocular toxicity","monitoring":"Phosphate monthly; ophthalmology exam monthly for 4 months"},"toxicity":[{"event":"Hyperphosphataemia","anyGradePct":79},{"event":"Stomatitis","anyGradePct":58},{"event":"Diarrhoea","anyGradePct":55},{"event":"Central serous retinopathy","anyGradePct":17},{"event":"Onycholysis","anyGradePct":23}],"access":[],"regulatoryEvents":[{"date":"2019-04-12","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC), that has: susceptible FGFR3 or FGFR2 genetic alterations, and progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA."},{"date":"2024-01-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2019 converted to traditional approval 4.8 years after it was granted.","indication":"Treatment of adult patients with locally advanced or metastatic urothelial carcinoma (mUC), that has: susceptible FGFR3 or FGFR2 genetic alterations, and progressed during or following at least one line of prior platinum-containing chemotherapy, including within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for BALVERSA."}]},{"id":"eribulin","kind":"drug","name":"Eribulin","aka":[],"tldr":"A sea-sponge-derived chemotherapy that extended survival in heavily pretreated breast cancer and in liposarcoma, where almost nothing else had.","summary":"EMBRACE (Lancet 2011): OS 13.1 vs 10.6 months versus treatment of physician's choice in pretreated metastatic breast cancer. Liposarcoma approval 2016 (OS 15.6 vs 8.4 months versus dacarbazine in the liposarcoma subgroup). Neuropathy and neutropenia; the payload of the ADC MORAb-202 (farletuzumab ecteribulin).","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Eribulin","links":[{"label":"EMBRACE (Lancet 2011)","url":"https://doi.org/10.1016/S0140-6736(11)60070-6"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=eribulin"}],"tags":["gap-fill"],"related":[],"cancers":["breast-hr-positive","tnbc","sarcoma","liposarcoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["eisai"],"institutions":[],"pathways":[],"terms":["payload"],"trials":["nct06435429","nct05824975","nct06081959","nct05458674","nct06202261","nct05814354","nct06942234","nct06343948","nct06449222","nct07173751","nct07007559","nct06957886","nct06279364","nct04639986","keynote-119","ascent","destiny-breast04"],"people":[],"bottlenecks":[],"keyPapers":["paper-cortes-lancet"],"journals":[],"dependsOn":[],"notes":[],"brand":"Halaven","modality":"Synthetic halichondrin B analogue (microtubule dynamics inhibitor)","mechanism":"Binds the plus ends of microtubules, suppressing growth without affecting shortening; also reverses EMT and remodels tumour vasculature.","approvals":[{"region":"US","year":2010,"indication":"Metastatic breast cancer after ≥2 regimens including an anthracycline and a taxane"},{"region":"US","year":2016,"indication":"Unresectable or metastatic liposarcoma after anthracycline"},{"region":"EU","year":2011,"indication":"Metastatic breast cancer; liposarcoma (2016)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"erlotinib","kind":"drug","name":"Erlotinib","aka":[],"tldr":"Erlotinib was one of the first EGFR pills for lung cancer; it was approved before anyone knew EGFR mutations predicted who would respond, then redefined by them.","summary":"Erlotinib is a reversible, ATP-competitive first-generation EGFR tyrosine kinase inhibitor that is most active against exon 19 deletions and the L858R mutation. It was first approved in the US in 2004 for advanced NSCLC after chemotherapy on the strength of BR.21 (2005), which showed a survival benefit in unselected pretreated patients before EGFR mutations were known to predict response. EURTAC (2012) and OPTIMAL then established first-line use in EGFR-mutant disease, leading to the 2013 US approval for exon 19 deletion or L858R tumours, and osimertinib later displaced it in the FLAURA trial. It is also approved with gemcitabine in pancreatic cancer since 2005, where the benefit is marginal. Rash and diarrhoea are the characteristic toxicities. Erlotinib is the drug that taught oncology to select patients by mutation rather than by histology alone.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Erlotinib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=erlotinib"},{"label":"EMA Tarceva product page: pancreatic indication","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/tarceva"},{"label":"Sinn et al., CONKO-005 (JCO 2017)","url":"https://doi.org/10.1200/JCO.2017.72.6463"},{"label":"Abrams et al., RTOG 0848 step 1 (Am J Clin Oncol 2020)","url":"https://doi.org/10.1097/COC.0000000000000633"}],"tags":["gap-fill","generic"],"related":[],"cancers":["nsclc","pancreatic","egfr-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["roche-genentech","astellas"],"institutions":[],"pathways":[],"terms":["egfr-exon19-l858r","histology"],"trials":["nct02152631","nct02411448","nct05442060","conko-005","lap07"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: erlotinib with gemcitabine is licensed for metastatic disease in the United States (2005) and the EU on a trial whose survival gain was under two weeks, and it added nothing in the adjuvant CONKO-005 (disease-free survival 11.4 months in both arms), in RTOG 0848 step 1 (median survival 29.9 against 28.1 months) or in the first randomisation of LAP07 (13.6 months with gemcitabine against 11.9 with the combination). NICE NG85 does not recommend it and NCCN no longer lists it as a preferred option."],"brand":"Tarceva","modality":"Small-molecule EGFR TKI (first generation, reversible)","mechanism":"Reversible ATP-competitive inhibitor of EGFR kinase; most active against exon 19 deletion and L858R mutations.","approvals":[{"region":"US","year":2004,"indication":"Locally advanced/metastatic NSCLC after chemotherapy"},{"region":"US","year":2005,"indication":"Pancreatic cancer with gemcitabine"},{"region":"US","year":2013,"indication":"First-line NSCLC with EGFR exon 19 del or L858R"},{"region":"EU","year":2007,"indication":"Metastatic pancreatic cancer with gemcitabine (Tarceva product information: factors associated with prolonged survival should be taken into account when prescribing; 100 mg daily); marketing authorisation for the product 19 September 2005","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/tarceva"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"eryaspase","kind":"drug","name":"Eryaspase","aka":["GRASPA","L-asparaginase encapsulated in red blood cells"],"tldr":"Eryaspase packed a leukaemia enzyme into red blood cells to starve pancreatic cancer of an amino acid; the phase 3 TRYbeCA-1 trial missed its survival goal.","summary":"Erytech Pharma, now part of Phaxiam, built eryaspase for tumours that cannot make their own asparagine. A randomised phase 2 in second-line pancreatic cancer had shown longer survival, which led to the phase 3 TRYbeCA-1 trial of eryaspase with chemotherapy against chemotherapy alone in the second line.\n\nTRYbeCA-1 reported in 2021: overall survival was not significantly improved, and the pancreatic programme ended.","status":"negative","asOf":"2026-09-17","links":[{"label":"ClinicalTrials.gov NCT03665441","url":"https://clinicaltrials.gov/study/NCT03665441"}],"tags":[],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["phaxiam"],"institutions":[],"pathways":[],"terms":[],"trials":["trybeca-1"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"GRASPA","modality":"L-asparaginase encapsulated in donor red blood cells","mechanism":"Depletes circulating asparagine, an amino acid that pancreatic tumours with low asparagine synthetase cannot make, while the red-cell shell protects the enzyme and reduces toxicity.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"esg401","kind":"drug","name":"ESG401","aka":[],"tldr":"ESG401 is an experimental investigational agent whose form is not stated in the registry from Qilu Pharmaceutical in phase 3 trials for triple-negative breast cancer and HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"ESG401 is an investigational agent whose form is not stated in the registry developed by Qilu Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06732323 (A Phase III Study of ESG401 for Unresectable Recurrent or Metastatic Triple-Negative Breast Cancer) and NCT06383767 (A Phase III Study of ESG401 for Locally Advanced or Metastatic HR+/HER2- Breast Cancer), in triple-negative breast cancer and HR-positive / HER2-negative breast cancer. The largest, NCT06732323, plans to enrol 504 participants with primary completion expected 2027-06. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ESG401","url":"https://clinicaltrials.gov/search?intr=ESG401"},{"label":"Sponsor page","url":"https://www.qilu-pharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["tnbc","breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06732323","nct06383767"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"estramustine","kind":"drug","name":"Estramustine","aka":["Estramustine phosphate"],"tldr":"Estramustine is an oral prostate cancer drug that combines an oestrogen with an alkylating agent, approved in the United States in 1981 for metastatic disease and later combined with taxanes, though its clotting risk has pushed it out of routine use.","summary":"Estramustine phosphate was designed to deliver a nitrogen mustard to oestrogen-receptor-bearing prostate cancer cells, but it turned out to work mostly by disrupting microtubules. The FDA approved it in 1981 for palliative treatment of metastatic or progressive prostate cancer. In the 1990s and 2000s it was combined with docetaxel and other taxanes with higher response rates than the taxane alone, but venous thromboembolism and the equal survival of docetaxel alone in the TAX 327 era left it out of standard regimens. It remains licensed in the United States and Europe.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Estramustine","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=estramustine"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Estramustine"},{"label":"ChEMBL CHEMBL1201078","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201078"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["tubulin"],"drugs":["docetaxel"],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00055731","nct00004054","nct00024167"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Emcyt","modality":"Oral oestradiol and nitrogen mustard conjugate","mechanism":"Carries a nornitrogen mustard on an oestradiol backbone; in the body it acts mainly as an antimicrotubule agent that binds microtubule-associated proteins, with oestrogenic effects that also lower testosterone.","approvals":[{"region":"US","year":1981,"indication":"Palliative treatment of metastatic or progressive prostate cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ethiodized-oil","kind":"drug","name":"Ethiodized oil","aka":["Ethiodol","Iodised poppy-seed oil"],"tldr":"Lipiodol is an iodine-rich oil injected into the liver's artery that lodges in liver tumours; it lets radiologists see them on CT and is the vehicle that carries chemotherapy into the tumour in conventional chemoembolisation for liver cancer.","summary":"Ethiodized oil has been approved in the United States since 1954, originally for lymphangiography and hysterosalpingography. In 2014 the FDA added selective hepatic intra-arterial use for imaging tumours in adults with known hepatocellular carcinoma, formalising its decades-old role in conventional transarterial chemoembolisation, where it is emulsified with doxorubicin or cisplatin and injected before the feeding vessels are blocked. The oil is also the carrier for iodine-131 lipiodol and for imaging hepatocellular carcinoma nodules on follow-up CT. Guerbet manufactures it.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Ethiodized_oil","links":[{"label":"Drugs@FDA NDA009190","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=009190"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Ethiodized_oil"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["hcc"],"sections":[],"technologies":["tace"],"targets":[],"drugs":["doxorubicin"],"companies":["guerbet"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02762266","nct02971345","nct02755311"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lipiodol","modality":"Oily iodinated contrast agent for hepatic arterial injection","mechanism":"An iodinated poppy-seed oil that is retained selectively by hepatocellular carcinoma after hepatic arterial injection, both showing the tumour on CT and carrying chemotherapy into it during chemoembolisation.","approvals":[{"region":"US","year":1954,"indication":"Lymphangiography and hysterosalpingography; selective hepatic intra-arterial imaging of hepatocellular carcinoma added in 2014"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"etoposide","kind":"drug","name":"Etoposide","aka":[],"tldr":"Etoposide is a chemotherapy from the mayapple plant, essential to curing testicular cancer (BEP), treating small-cell lung cancer, lymphomas, childhood sarcomas and leukaemias, and used in transplant conditioning.","summary":"Approved 1983. BEP/EP (germ cell), platinum-etoposide (SCLC, neuroendocrine carcinoma), VDC/IE (Ewing), ICE/DHAP salvage (lymphoma), EPOCH, HLH and Wilms/neuroblastoma regimens, EMA-CO (GTN), oral etoposide palliation, high-dose conditioning (BEAM). Secondary AML with 11q23 (KMT2A) rearrangement is a recognised late effect.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Etoposide","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=etoposide"}],"tags":["gap-fill","generic","who-essential"],"related":[],"cancers":["testicular","sclc","ewing-sarcoma","dlbcl","neuroendocrine","wilms-tumor","retinoblastoma","gestational-trophoblastic","adrenocortical","aml","cns-germ-cell-tumours","high-risk-gtn","placental-site-trophoblastic-tumour","advanced-adrenocortical-carcinoma","esthesioneuroblastoma","sinonasal-undifferentiated-carcinoma"],"sections":[],"technologies":["topoisomerase-inhibitors","cytotoxic-chemotherapy","autologous-stem-cell-transplant"],"targets":["kmt2a"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["euramos-1","nct06712355","nct07625644","nct07502300","nct06564844","nct07005128","nct06230224","nct07472517","nct05652686","nct04745689","nct06030258","nct07296809","nct07245446","nct05533775","nct07730515","nct07155174","nct06449209","nct07227597","nct05224141","nct05844150","nct04665856","nct07268040","nct07544654","nct05566041","nct03182244","nct01064466","nct06741644","nct03793478","nct04380636"],"people":["lance-armstrong"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"VePesid / Etopophos / Toposar","modality":"Topoisomerase II inhibitor (podophyllotoxin derivative)","mechanism":"Stabilises topoisomerase II-DNA cleavage complexes, producing double-strand breaks in S/G2; schedule-dependent (multi-day dosing).","approvals":[{"region":"US","year":1983,"indication":"Refractory testicular tumours; small-cell lung cancer (1986)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"everolimus","kind":"drug","name":"Everolimus","aka":[],"tldr":"An mTOR-blocking pill that doubled progression-free time with exemestane in 2012 and is now paired with the oral SERD giredestrant.","summary":"Everolimus is a rapalog that binds FKBP12 and allosterically inhibits mTORC1, a growth-signalling hub downstream of PI3K and AKT. It is approved in HR-positive breast cancer with exemestane after non-steroidal aromatase inhibitor failure, in progressive pancreatic, lung and GI neuroendocrine tumours, and in advanced RCC after sunitinib or sorafenib. BOLERO-2 showed PFS of 7.8 versus 3.2 months for everolimus plus exemestane versus exemestane alone, with no OS benefit; RADIANT-3 and RADIANT-4 showed PFS of 11.0 versus 4.6 and 3.9 months in NETs. Stomatitis (mitigated by dexamethasone mouthwash, SWISH), pneumonitis and hyperglycaemia are the main toxicities. evERA (2025) revived it as the partner of giredestrant after CDK4/6 inhibitors; it is the comparator beaten in LITESPARK-005 and COMPETE. For a newcomer, it is a growth-pathway pill with several niches rather than one dominant use.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Everolimus","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Everolimus"}],"tags":[],"related":[],"cancers":["breast-hr-positive","rcc","neuroendocrine","hr-positive-metastatic-post-cdk46","chromophobe-rcc"],"sections":[],"technologies":["kinase-inhibitors","pi3k-akt-mtor-inhibitors"],"targets":["pik3ca","akt"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":["pi3k-akt-mtor"],"terms":["hyperglycaemia"],"trials":["evera","nct07165886","nct07002177","nct05563220","nct06428396","nct04919226","nct05573126","radiant-3-4","compete","litespark-005","clear"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In neuroendocrine tumours: RADIANT-3 (2011) gave PFS 11.0 versus 4.6 months in pancreatic NETs and RADIANT-4 (2016) PFS 11.0 versus 3.9 months in lung and GI NETs; it is now the comparator that 177Lu-edotreotide beat in COMPETE. In RADIANT-3 stomatitis affected 64 percent (7 percent grade 3 or higher), rash 49 percent and pneumonitis 17 percent; dexamethasone mouthwash (SWISH) prevents most stomatitis.","In kidney cancer: RECORD-1 (2008) gave PFS 4.9 versus 1.9 months against placebo after a VEGF-TKI, making everolimus the second-line standard from 2009; it has since been beaten by nivolumab (CheckMate 025), cabozantinib (METEOR), lenvatinib plus everolimus (Study 205) and belzutifan (LITESPARK-005), and is now mostly a comparator arm and a partner for lenvatinib at 5 mg daily. Feedback activation of AKT limits its cytostatic effect."],"brand":"Afinitor","modality":"Small-molecule mTOR inhibitor","mechanism":"Allosteric mTORC1 inhibitor (rapalog) via FKBP12.","approvals":[{"region":"US","year":2012,"indication":"HR+/HER2- advanced breast cancer with exemestane after NSAI failure"},{"region":"US","year":2011,"indication":"Progressive pancreatic NETs"},{"region":"US","year":2016,"indication":"Progressive non-functional lung and GI NETs"},{"region":"US","year":2009,"indication":"Advanced RCC after sunitinib or sorafenib"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"10 mg once daily","modifications":"5 mg for stomatitis or pneumonitis","monitoring":"Glucose, lipids, pneumonitis symptoms; steroid mouthwash prophylaxis"},"toxicity":[{"event":"Stomatitis","anyGradePct":59,"grade3PlusPct":8},{"event":"Pneumonitis","anyGradePct":16,"grade3PlusPct":3},{"event":"Hyperglycaemia","anyGradePct":14,"grade3PlusPct":5}],"access":[],"regulatoryEvents":[{"date":"2010-10-29","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Patients with subependymal giant cell astrocytoma associated with tuberous sclerosis who require therapeutic intervention but are not candidates for curative surgical resection"},{"date":"2012-04-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adults with renal angiomyolipma and tuberous sclerosis complex not requiring immediate surgery"},{"date":"2012-08-29","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Formulation: Pediatric and adult patients with tuberous sclerosis complex for treatment of subependymal giant cell astrocytoma that requires therapeutic intervention but cannot be curatively resected"},{"date":"2016-01-29","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2010 converted to traditional approval 5.3 years after it was granted.","indication":"Patients with subependymal giant cell astrocytoma associated with tuberous sclerosis who require therapeutic intervention but are not candidates for curative surgical resection"},{"date":"2016-01-29","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2012 converted to traditional approval 3.4 years after it was granted.","indication":"Formulation: Pediatric and adult patients with tuberous sclerosis complex for treatment of subependymal giant cell astrocytoma that requires therapeutic intervention but cannot be curatively resected"},{"date":"2016-02-18","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2012 converted to traditional approval 3.8 years after it was granted.","indication":"Adults with renal angiomyolipma and tuberous sclerosis complex not requiring immediate surgery"}]},{"id":"evexomostat","kind":"drug","name":"Evexomostat","aka":["SDX-7320"],"tldr":"Evexomostat is a small-molecule inhibitor from SynDevRx, Inc., in registered phase 2 trials for metastatic cancer.","summary":"Evexomostat is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by SynDevRx, Inc., in metastatic cancer. Described in the registry record as a polymer-drug conjugate of a novel metap2 inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Evexomostat","url":"https://clinicaltrials.gov/search?intr=Evexomostat"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["syndevrx"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05455619"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a polymer-drug conjugate of a novel metap2 inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"evm14","kind":"drug","name":"EVM14","aka":[],"tldr":"EVM14 is a cancer vaccine from Everest Medicines (Beijing) Co., Ltd., in registered phase 2 trials for metastatic cancer.","summary":"EVM14 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Everest Medicines (Beijing) Co., Ltd., in metastatic cancer. A cancer vaccine, as described in the registry record: it trains the immune system against tumour antigens. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of EVM14","url":"https://clinicaltrials.gov/search?intr=EVM14"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["everest-medicines"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07095868"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"EVM14","modality":"Cancer vaccine","mechanism":"A cancer vaccine, as described in the registry record: it trains the immune system against tumour antigens.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"evofosfamide","kind":"drug","name":"Evofosfamide","aka":[],"tldr":"Evofosfamide was the most advanced hypoxia-activated drug, designed to kill the oxygen-starved tumour cells radiotherapy and chemotherapy miss, but it failed both of its phase 3 trials in 2015.","summary":"Evofosfamide (TH-302), developed by Threshold Pharmaceuticals with Merck KGaA, showed promising responses in early trials. In 2015 both phase 3 trials missed their overall survival endpoints: MAESTRO with gemcitabine in pancreatic cancer and TH CR-406 with doxorubicin in soft-tissue sarcoma. Development largely stopped, though small studies with radiotherapy and immunotherapy continue, and the programme remains the reference case for why hypoxia targeting is hard: patients were not selected for hypoxia, and the drug's activation depends on oxygen levels that vary from hour to hour.","status":"negative","asOf":"2026-09-16","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Evofosfamide"}],"tags":["radiation-wave3"],"related":[],"cancers":["pancreatic","sarcoma"],"sections":[],"technologies":["tumour-hypoxia-modification"],"targets":[],"drugs":[],"companies":["threshold-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo radiation expansion","editedOn":"2026-09-16"},"code":"TH-302","modality":"Hypoxia-activated prodrug (small molecule alkylating agent)","mechanism":"A nitroimidazole-linked prodrug that is inert in oxygenated tissue and releases the DNA-crosslinking agent bromo-isophosphoramide mustard only in the low-oxygen regions of tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"evorpacept","kind":"drug","name":"Evorpacept","aka":["Evorpacept (ALX148)"],"tldr":"Evorpacept is an experimental immune checkpoint modulator from ALX Oncology in phase 3 trials for gastric & gastro-oesophageal junction cancer, HR-positive / HER2-negative breast cancer and HER2-positive breast cancer, with its target not yet stated publicly.","summary":"Evorpacept (ALX148) is an immune checkpoint modulator developed by ALX Oncology. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05002127 (A Study of Evorpacept (ALX148) in Patients With Advanced HER2+ Gastric Cancer (ASPEN-06)), in gastric & gastro-oesophageal junction cancer, HR-positive / HER2-negative breast cancer and HER2-positive breast cancer. The largest, NCT05002127, plans to enrol 127 participants (actual) with primary completion was scheduled for 2024-05-24 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Evorpacept","url":"https://clinicaltrials.gov/search?intr=ALX148"},{"label":"Sponsor page","url":"https://www.alxoncology.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric","breast-hr-positive","breast-her2-positive"],"sections":[],"technologies":["cd47-blockade"],"targets":[],"drugs":[],"companies":["alx-oncology"],"institutions":[],"pathways":[],"terms":[],"trials":["aspen-06","nct04675294","nct04675333","nct07007559"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ALX148","modality":"immune checkpoint modulator","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"exemestane","kind":"drug","name":"Exemestane","aka":[],"tldr":"Exemestane is a steroidal aromatase inhibitor, the partner of everolimus and the agent tested with ovarian suppression in young women.","summary":"Exemestane is a steroidal aromatase inactivator: it binds aromatase irreversibly as a 'suicide' substrate, permanently disabling the enzyme that makes oestrogen from androgens in postmenopausal women. It is used in hormone receptor-positive breast cancer after failure of a non-steroidal aromatase inhibitor, as the partner of everolimus (BOLERO-2), and with ovarian function suppression in premenopausal women (TEXT). In the combined SOFT and TEXT analysis, exemestane plus OFS improved 12-year disease-free survival over tamoxifen plus OFS (80.5% versus 75.9%, HR 0.79) without an overall survival difference. It is taken as 25 mg once daily after a meal and is generic. Whether its steroidal structure gives a real advantage after non-steroidal AI failure is not settled. For a newcomer, exemestane is the aromatase inhibitor most often chosen when another one has stopped working.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Exemestane","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Exemestane"}],"tags":[],"related":[],"cancers":["breast-hr-positive","hr-positive-early-high-risk"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["soft-text","evera","nct05768139","nct06979596","nct05774951","nct04906395","nct06016738","nct06957379","nct06492616","nct06428396","nct05514054","nct05827081","nct05573126"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aromasin","modality":"Small-molecule steroidal aromatase inactivator","mechanism":"Irreversible 'suicide' inactivation of aromatase.","approvals":[{"region":"US","year":1999,"indication":"Advanced breast cancer after tamoxifen"},{"region":"US","year":2005,"indication":"Adjuvant after 2-3 years of tamoxifen"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"25 mg once daily after a meal"},"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"f182112","kind":"drug","name":"F182112","aka":["F182112 single-agent"],"tldr":"F182112 is an experimental investigational agent whose form is not stated in the registry from Shandong New Time Pharmaceutical in phase 3 trials for multiple myeloma, with its target not yet stated publicly.","summary":"F182112 is an investigational agent whose form is not stated in the registry developed by Shandong New Time Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07579234 (A Multicenter, Randomised, Open-label, Parallel-group, Controlled, Superiority Phase III Clinical Study Comparing the Efficacy and Safety of F182112 Versus Standard of Care in Patients With Relapsed or Refractory Multiple Myeloma), in multiple myeloma. The largest, NCT07579234, plans to enrol 261 participants with primary completion expected 2029-01-25. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of F182112","url":"https://clinicaltrials.gov/search?intr=F182112"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07579234"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fap-2286","kind":"drug","name":"FAP-2286 (177Lu / 68Ga)","aka":[],"tldr":"A FAP-targeted theranostic pair: one version images almost any solid tumour, the other treats it with radiation.","summary":"FAP-2286 is a theranostic pair: a cyclic peptide that binds fibroblast activation protein (FAP), linked to a DOTA chelator carrying 68Ga for PET imaging or 177Lu for beta therapy. FAP sits on cancer-associated fibroblasts in the stroma of most solid tumours rather than on cancer cells, so one agent can image and irradiate many tumour types, and the cyclic peptide gives longer tumour retention than the FAPI small molecules used for imaging. Clovis Oncology developed it until its 2022 bankruptcy; Novartis then acquired it via 3B Pharmaceuticals. The 177Lu-FAP-2286 LuMIERE phase 1/2 trial is dose-escalating in cycles every 6 weeks with pancreatic, sarcoma and breast cohorts. For a newcomer: a radioligand aimed at the scaffolding around tumours rather than the tumour cells.","status":"phase-2","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of FAP-2286 (177Lu / 68Ga)","url":"https://clinicaltrials.gov/search?intr=FAP-2286"}],"tags":[],"related":[],"cancers":["pancreatic","sarcoma","tnbc"],"sections":[],"technologies":["fapi-pet","radioligand-therapy"],"targets":["fap"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05939414","nct06520345","nct07219238","nct05413850","nct06894511","nct05142696","nct04939610"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"FAP-2286","modality":"Theranostic pair (peptide radioligand)","mechanism":"Cyclic peptide FAP binder with DOTA chelator for 68Ga (imaging) or 177Lu (therapy).","approvals":[],"mechanismSteps":["Radioligand circulates and binds FAP on cancer-associated fibroblasts on tumour cells","Ligand is internalised or retained at the membrane","Beta emissions deposit energy within a short range","Clustered DNA double-strand breaks form in the tumour cell and its neighbours (crossfire)","Cells die; unbound ligand is cleared via the kidneys"],"dosing":{"route":"IV infusion (177Lu) or injection (68Ga)","schedule":"LuMIERE phase 1/2 dose escalation, cycles every 6 weeks","monitoring":"Blood counts, renal function","source":"https://clinicaltrials.gov/study/NCT04939610"},"toxicity":[{"event":"Anaemia"},{"event":"Thrombocytopenia"},{"event":"Fatigue"},{"event":"Nausea"}],"access":[{"country":"US","reimbursement":"Investigational","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-01","type":"filing","region":"US","note":"Novartis acquires rights after Clovis bankruptcy","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"farletuzumab-ecteribulin","kind":"drug","name":"Farletuzumab ecteribulin","aka":["MORAb-202"],"tldr":"Farletuzumab ecteribulin is an antibody-drug conjugate from Eisai Inc., in registered phase 2 trials for metastatic cancer.","summary":"Farletuzumab ecteribulin is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Eisai Inc., in metastatic cancer. An antibody-drug conjugate: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. The registry record does not state the target or payload; the trial entries below carry the sponsor's description. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Farletuzumab ecteribulin","url":"https://clinicaltrials.gov/search?intr=Farletuzumab%20ecteribulin"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["eisai"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04300556"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Antibody-drug conjugate","mechanism":"An antibody-drug conjugate: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. The registry record does not state the target or payload; the trial entries below carry the sponsor's description.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"farletuzumab-exatecan","kind":"drug","name":"Farletuzumab-exatecan","aka":[],"tldr":"Farletuzumab-exatecan is an experimental antibody-drug conjugate from CSPC Megalith Biopharmaceutical in phase 2 trials, aimed at Folate receptor alpha.","summary":"Farletuzumab-exatecan (SYS6041) is an antibody-drug conjugate developed by CSPC Megalith Biopharmaceutical. Its target is Folate receptor alpha (the sponsor names Folate receptor alpha (FRα)). The sponsor states: SYS6041 (farletuzumab-exatecan) is an antibody-drug conjugate targeting human folate receptor alpha with a topoisomerase I inhibitor payload, given by intravenous injection on day 1 of each three-week cycle. ClinicalTrials.gov describes the intervention as: Farletuzumab-exatecan antibody-drug conjugate targeting human FRα. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT07744763, plans to enrol 260 participants with primary completion expected 2027-03-28. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Farletuzumab-exatecan","url":"https://clinicaltrials.gov/search?intr=Farletuzumab-exatecan"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["folr1"],"drugs":[],"companies":["cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07714668","nct07744763"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"SYS6041","modality":"ADC","mechanism":"SYS6041 (farletuzumab-exatecan) is an antibody-drug conjugate targeting human folate receptor alpha with a topoisomerase I inhibitor payload, given by intravenous injection on day 1 of each three-week cycle.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"favezelimab","kind":"drug","name":"Favezelimab","aka":[],"tldr":"Favezelimab is Merck's LAG-3 antibody, tested with pembrolizumab in phase 3 trials in colorectal cancer and classical Hodgkin lymphoma; the colorectal trial did not succeed.","summary":"Merck's favezelimab (MK-4280) blocks LAG-3, the second checkpoint validated by relatlimab in melanoma. Coformulated with pembrolizumab as MK-4280A, it entered phase 3 trials in previously treated microsatellite-stable colorectal cancer, where the trial did not meet its overall survival endpoint, and in relapsed classical Hodgkin lymphoma after PD-1 therapy. Registered phase 3 trials remain on ClinicalTrials.gov.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Favezelimab","url":"https://clinicaltrials.gov/search?intr=MK-4280"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal","hodgkin-lymphoma"],"sections":[],"technologies":["checkpoint-inhibitor","lag3-blockade"],"targets":["lag3"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03486873","nct04895722","nct04626479","nct04938817","nct04626518","nct05845814"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"MK-4280","modality":"Anti-LAG-3 monoclonal antibody, intravenous, also coformulated with pembrolizumab (MK-4280A)","mechanism":"Blocks the LAG-3 checkpoint on exhausted T cells; given with PD-1 blockade to overcome resistance to pembrolizumab alone.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fda018-adc","kind":"drug","name":"FDA018-ADC","aka":["FDA018-Antibody-drug Conjugate"],"tldr":"FDA018-ADC is an experimental antibody-drug conjugate from Shanghai Fudan-Zhangjiang Bio-Pharmaceutical in phase 3 trials for triple-negative breast cancer, with its target not yet stated publicly.","summary":"FDA018-ADC (FDA018) is an antibody-drug conjugate developed by Shanghai Fudan-Zhangjiang Bio-Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Subjects will receive FDA018-ADC 10 mg/kg of body weight via intravenous(IV) infusion on Day1 and 8 of a 21-day cycle in follow-up period until disease progression, unacceptable toxicity or death. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06519370 (FDA018-ADC vs Investigator's Choice Chemotherapy to Treat Locally Advanced, Recurrent or Metastatic Triple-negative Breast Cancer), in triple-negative breast cancer. The largest, NCT06519370, plans to enrol 350 participants with primary completion was scheduled for 2026-08-18 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of FDA018-ADC","url":"https://clinicaltrials.gov/search?intr=FDA018"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["tnbc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":["fuscc"],"pathways":[],"terms":[],"trials":["nct06519370"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"FDA018","modality":"ADC","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fedratinib","kind":"drug","name":"Fedratinib","aka":[],"tldr":"A second JAK inhibitor for myelofibrosis that works after ruxolitinib fails; it carries a boxed warning for a rare brain toxicity (Wernicke encephalopathy) so thiamine is checked.","summary":"Fedratinib is a selective JAK2 inhibitor, with additional FLT3 and BRD4 activity, that reduces JAK-STAT signalling to shrink the spleen and ease symptoms in myelofibrosis. In JAKARTA (first line) and JAKARTA-2 (after ruxolitinib) about 35 to 45% of patients achieved a spleen response, giving it evidence in ruxolitinib failure. Development was halted in 2013 after cases of encephalopathy and resumed after review; it was approved in the US in 2019 for intermediate-2 or high-risk primary or secondary myelofibrosis and in the EU in 2021, with a boxed warning for Wernicke encephalopathy, so thiamine is checked before and during treatment. Anaemia and thrombocytopenia occur as with other JAK inhibitors. For a newcomer: a myelofibrosis pill that works after ruxolitinib, with a rare brain toxicity that vitamin monitoring prevents.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Fedratinib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=fedratinib"}],"tags":["gap-fill"],"related":[],"cancers":["myeloproliferative-neoplasms","primary-myelofibrosis"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["jak2","flt3","brd4"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04817007"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Inrebic","modality":"Small-molecule JAK2/FLT3 inhibitor","mechanism":"Selective JAK2 inhibitor (also FLT3, BRD4) reducing JAK-STAT signalling and splenomegaly.","approvals":[{"region":"US","year":2019,"indication":"Intermediate-2/high-risk primary or secondary myelofibrosis"},{"region":"EU","year":2021,"indication":"Myelofibrosis, JAK-inhibitor-naive or after ruxolitinib"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"feladilimab","kind":"drug","name":"Feladilimab","aka":["GSK3359609"],"tldr":"Feladilimab is a monoclonal antibody from GlaxoSmithKline, in registered phase 2 trials for multiple myeloma.","summary":"Feladilimab is listed on ClinicalTrials.gov as an intervention in 2 registered phase 2 trials sponsored by GlaxoSmithKline, in multiple myeloma. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Feladilimab","url":"https://clinicaltrials.gov/search?intr=Feladilimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07217119","nct04126200"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fenbendazole","kind":"drug","name":"Fenbendazole (veterinary anthelmintic)","aka":["FBZ"],"tldr":"Fenbendazole is a dog and livestock dewormer that is sold online as a cancer cure. It has never been approved for people, no trial has tested it in cancer, and doctors have reported severe liver injury in patients who took it.","summary":"Fenbendazole is an anthelmintic approved only for animals, and its human use has grown because social media promotes it as an anticancer drug (Thakurdesai case report 2024). The first case of biopsy-confirmed severe liver injury from self-administered fenbendazole was a 67-year-old woman whose jaundice cleared three months after she stopped (Thakurdesai case report 2024). A 65-year-old man with prostate cancer who alternated veterinary fenbendazole and ivermectin daily for three months developed hepatocellular liver injury with a bilirubin of 12.9 mg/dL, which normalised within six weeks of stopping (Powderly case report 2026). A three-patient case series claiming remissions was retracted by the journal in January 2026 (Retraction notice 2026). After a January 2025 podcast promoted fenbendazole with ivermectin for cancer, combined prescribing to US cancer patients rose 2.6-fold (Rockwell JAMA Network Open 2026). The FDA's guidance on products promoted as cancer cures without trial evidence applies here (FDA on products claiming to cure cancer).","status":"preclinical","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Fenbendazole","links":[{"label":"Thakurdesai case report 2024","url":"https://doi.org/10.14309/crj.0000000000001354"},{"label":"Powderly case report 2026","url":"https://doi.org/10.7759/cureus.108896"},{"label":"Retraction notice 2026","url":"https://doi.org/10.1159/000549387"},{"label":"Rockwell JAMA Network Open 2026","url":"https://doi.org/10.1001/jamanetworkopen.2026.16780"},{"label":"FDA on products claiming to cure cancer","url":"https://www.fda.gov/consumers/consumer-updates/products-claiming-cure-cancer-are-cruel-deception"}],"tags":[],"related":[],"cancers":[],"sections":[],"technologies":["fenbendazole-ivermectin-repurposing-claims"],"targets":[],"drugs":["ivermectin","mebendazole"],"companies":[],"institutions":[],"pathways":[],"terms":["hepatotoxicity","off-label","preclinical"],"trials":[],"people":[],"bottlenecks":["b-misinformation"],"keyPapers":["paper-thakurdesai-fenbendazole-liver-injury-2024","paper-powderly-fenbendazole-ivermectin-liver-injury-2026","paper-makis-fenbendazole-case-series-retracted-2025","paper-rockwell-ivermectin-benzimidazole-prescribing-jama-netw-open-2026"],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo agent (Claude Fable 5.1)","editedOn":"2026-09-24","note":"Written from cached ClinicalTrials.gov v2, Europe PMC, DailyMed, Drugs@FDA and WHO eEML responses; no figure appears without its source link."},"modality":"Veterinary benzimidazole anthelmintic; not approved for human use in any country","mechanism":"Binds parasite tubulin and stops microtubule assembly, which is how it kills worms in dogs and livestock. Cancer cell lines respond to the same microtubule effect in a dish; that is laboratory evidence with no human trial behind it.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fentanyl","kind":"drug","name":"Fentanyl (transmucosal, for breakthrough cancer pain)","aka":["fentanyl","fentanyl citrate","Effentora","Instanyl","PecFent"],"tldr":"These are fast-acting forms of the opioid fentanyl, taken as a tablet against the cheek or as a nasal spray, for sudden flares of pain in people whose cancer pain is otherwise controlled by a regular opioid. They are only for patients already tolerant to opioids.","summary":"Three centrally authorised fentanyl products are indicated for breakthrough pain in adults who are already receiving maintenance opioid therapy for chronic cancer pain, breakthrough pain being a transitory exacerbation on a background of otherwise controlled persistent pain: Effentora buccal tablets (authorised 4 April 2008; marketing authorisation holder Phoenix Labs Unlimited Company), Instanyl nasal spray (authorised 20 July 2009; Istituto Gentili S.r.l.) and PecFent nasal spray (authorised 31 August 2010; Gruenenthal GmbH). The EMA lists the therapeutic area as pain. This record covers the cancer-pain formulations only.","status":"approved","asOf":"2026-09-22","links":[{"label":"EMA: Effentora (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/effentora"},{"label":"EMA: Instanyl (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/instanyl"},{"label":"EMA: PecFent (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/pecfent"}],"tags":["ema-register","supportive"],"related":["methylnaltrexone"],"cancers":[],"sections":[],"technologies":["pain-management"],"targets":["oprm1"],"drugs":[],"companies":["gruenenthal"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00542542"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo content wave 5 (EMA medicines register and EPAR pages)","editedOn":"2026-09-22"},"brand":"Effentora / Instanyl / PecFent","modality":"Small-molecule opioid analgesic (buccal tablet and nasal sprays)","supportive":true,"mechanism":"Mu-opioid receptor agonist formulated for fast absorption through the mouth or nose, for breakthrough pain in adults already on maintenance opioid therapy for chronic cancer pain (indication wording, EMA).","approvals":[{"region":"EU","year":2008,"indication":"Breakthrough pain in adults receiving maintenance opioid therapy for chronic cancer pain","note":"Effentora authorised 4 Apr 2008 (Phoenix Labs); Instanyl 20 Jul 2009 (Istituto Gentili); PecFent 31 Aug 2010 (Gruenenthal)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fianlimab","kind":"drug","name":"Fianlimab","aka":[],"tldr":"Fianlimab is Regeneron's LAG-3 blocking antibody, paired with the PD-1 blocker cemiplimab. A 60% phase 1 response rate in untreated melanoma prompted phase 3 trials, but the metastatic trial against pembrolizumab missed its progression endpoint in 2026.","summary":"Phase 1 with cemiplimab reported ORR ~60% in first-line melanoma, prompting phase 3 trials versus pembrolizumab in metastatic disease (missed PFS, 2026) and in the adjuvant setting (ongoing). Also being tested in NSCLC. The result narrows the LAG-3 story to the specific nivolumab-relatlimab pairing so far.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"Regeneron update","url":"https://investor.regeneron.com/news-releases/news-release-details/regeneron-provides-update-phase-3-trial-fianlimab-lag-3"}],"tags":["failed-so-far"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["lag3"],"drugs":["cemiplimab"],"companies":["regeneron"],"institutions":[],"pathways":[],"terms":[],"trials":["fianlimab-phase3-melanoma","nct06246916","nct05800015","nct05608291","nct05785767","nct06161441","nct03916627","nct06190951","nct06769698"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"REGN3767","modality":"Monoclonal antibody (anti-LAG-3)","mechanism":"Fully human IgG4 blocking LAG-3 binding to MHC class II and FGL1.","approvals":[],"mechanismSteps":["Blocks LAG-3 on exhausted T cells","With PD-1 blockade, aims to restore effector function in doubly inhibited T cells"],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026","type":"designation","region":"US","note":"First-line metastatic phase 3 missed its primary PFS endpoint (Regeneron release)"}]},{"id":"ficerafusp-alfa","kind":"drug","name":"Ficerafusp alfa","aka":[],"tldr":"Ficerafusp alfa is an EGFR antibody fused to a TGF-beta sponge, designed to remove the immune-suppressing signal that keeps HPV-negative throat cancers cold.","summary":"Ficerafusp alfa (Bicara Therapeutics) is a bifunctional antibody: an anti-EGFR IgG1 with the extracellular domain of TGF-beta receptor II fused to its heavy chain, so it blocks EGFR on tumour cells and neutralises TGF-beta locally, removing an immune-suppressing signal that keeps HPV-negative head and neck tumours cold. It is given at 1500 mg every 2 weeks with pembrolizumab in first-line HPV-negative head and neck squamous cell carcinoma. A phase 1/1b study with pembrolizumab showed durable responses at two years (JCO 2025). The FORTIFI-HN01 phase 2/3 trial fixed the dose at 1500 mg, excludes HPV-positive oropharyngeal cancer, and expects an interim analysis in mid-2027. Whether trapping TGF-beta adds meaningfully to EGFR and PD-1 blockade in a randomised setting is the open question. For a newcomer, it is an EGFR antibody fused to a TGF-beta sponge.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"JCO 2025 two-year results","url":"https://ascopubs.org/doi/10.1200/JCO-25-02027"}],"tags":[],"related":[],"cancers":["head-and-neck","recurrent-metastatic-hnscc","hpv-negative-head-and-neck-cancer"],"sections":[],"technologies":["bispecific-antibody"],"targets":["egfr"],"drugs":["pembrolizumab"],"companies":["bicara-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["fortifi-hn01","nct07807163"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"BCA101","modality":"Bifunctional antibody (EGFR × TGF-β trap)","mechanism":"Anti-EGFR IgG1 with a TGF-βRII extracellular domain fused to the heavy chain, neutralising TGF-β locally.","approvals":[],"mechanismSteps":["Binds EGFR on tumour cells, localising the molecule to the tumour","The TGF-βRII trap sequesters TGF-β in the microenvironment","Fibroblast activation and T-cell exclusion decrease; PD-1 blockade then acts on infiltrating T cells"],"dosing":{"route":"Intravenous","schedule":"1500 mg every 2 weeks with pembrolizumab","source":"https://clinicaltrials.gov/study/NCT06788990"},"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ficlatuzumab","kind":"drug","name":"Ficlatuzumab","aka":[],"tldr":"Ficlatuzumab is an experimental monoclonal antibody from AVEO Pharmaceuticals in phase 3 trials for head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"Ficlatuzumab (AV-299) is a monoclonal antibody developed by AVEO Pharmaceuticals. The sponsor describes its target as HGF (hepatocyte growth factor), which OnCo does not yet have a target page for. The sponsor states: A humanized hepatocyte growth factor (HGF) inhibitory IgG1 monoclonal antibody that blocks HGF signalling, distinct from EGFR-targeted agents such as cetuximab. ClinicalTrials.gov describes the intervention as: Ficlatuzumab (AV-299) is a humanized hepatocyte growth factor (HGF) inhibitory immunoglobulin G1 (IgG1) monoclonal antibody (mAb). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06064877 (A Study of Ficlatuzumab in Combination With Cetuximab in Participants With Recurrent or Metastatic (R/M) HPV Negative Head and Neck Squamous Cell Carcinoma), in head and neck squamous cell carcinoma. The largest, NCT06064877, plans to enrol 410 participants with primary completion expected 2027-08. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Ficlatuzumab","url":"https://clinicaltrials.gov/search?intr=AV-299"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["aveo"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06064877"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AV-299","modality":"monoclonal antibody","mechanism":"A humanized hepatocyte growth factor (HGF) inhibitory IgG1 monoclonal antibody that blocks HGF signalling, distinct from EGFR-targeted agents such as cetuximab.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"filgrastim","kind":"drug","name":"Filgrastim (G-CSF)","aka":["Zarxio (filgrastim-sndz)","Nivestym (filgrastim-aafi)","Releuko (filgrastim-ayow)","Granix (tbo-filgrastim)","Nypozi","Zarzio","Tevagrastim","Ratiograstim","Accofil","Grastofil"],"tldr":"Filgrastim (Neupogen and its biosimilars) is a daily injection that speeds the recovery of infection-fighting white cells after chemotherapy, lets doctors give chemotherapy on schedule and mobilises stem cells for transplant.","summary":"Filgrastim was approved by the FDA in February 1991 to reduce the incidence of infection from febrile neutropenia in non-myeloid malignancies receiving myelosuppressive chemotherapy, and later for AML induction, bone marrow transplantation, peripheral blood progenitor cell mobilisation, severe chronic neutropenia and acute radiation syndrome. It was the first Amgen blockbuster and the model for supportive-care biologics; the first US biosimilar of any kind was filgrastim-sndz (Zarxio, 2015), and the EU authorised the first filgrastim biosimilars in 2008. Guidelines recommend primary prophylaxis when the febrile neutropenia risk of a regimen exceeds about 20%. Bone pain is the main adverse effect; splenic rupture, acute respiratory distress syndrome and sickle cell crises are rare but serious. Pegfilgrastim gives a once-per-cycle alternative.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Filgrastim","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=filgrastim"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/filgrastim"},{"label":"EPAR (Zarzio)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/zarzio"}],"tags":["nci-list","supportive"],"related":["pegfilgrastim","plerixafor"],"cancers":[],"sections":[],"technologies":["g-csf-growth-factors"],"targets":[],"drugs":[],"companies":["amgen","sandoz","teva","eurofarma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06212752","nct06514508","nct03246529","nct05722015"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Neupogen","modality":"Recombinant granulocyte colony-stimulating factor","supportive":true,"mechanism":"Recombinant methionyl human G-CSF that binds the G-CSF receptor on myeloid progenitors, driving neutrophil proliferation, differentiation and release from marrow.","approvals":[{"region":"US","year":1991,"indication":"Reduction of febrile neutropenia in non-myeloid malignancies on myelosuppressive chemotherapy; later AML induction, transplantation, stem cell mobilisation, severe chronic neutropenia"},{"region":"US","year":2015,"indication":"First US biosimilar (Zarxio, filgrastim-sndz); further biosimilars 2018 onwards"},{"region":"EU","year":2008,"indication":"First biosimilar filgrastims (Tevagrastim, Ratiograstim, Biograstim); Zarzio 2009"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"flonoltinib","kind":"drug","name":"Flonoltinib","aka":[],"tldr":"Flonoltinib is an oral kinase inhibitor from Chengdu Zenitar Biomedical Technology Co., Ltd, in registered phase 2 trials for myeloproliferative neoplasms.","summary":"Flonoltinib is listed on ClinicalTrials.gov as an intervention in 3 registered phase 2 trials sponsored by Chengdu Zenitar Biomedical Technology Co., Ltd, in myeloproliferative neoplasms. A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Flonoltinib","url":"https://clinicaltrials.gov/search?intr=Flonoltinib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["myeloproliferative-neoplasms"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07232290","nct07232290","nct07232290"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral kinase inhibitor","mechanism":"A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"florastamin-f18","kind":"drug","name":"Florastamin F-18","aka":[],"tldr":"Florastamin is FutureChem's fluorine-18 PSMA PET tracer from South Korea, in phase 3 trials for staging high-risk and recurrent prostate cancer alongside the established PSMA agents.","summary":"FutureChem, a Seoul radiopharmaceutical company, developed florastamin (FC303) as a fluorine-18 PSMA ligand for PET imaging of prostate cancer. Registered phase 3 trials assess it for initial staging of high-risk prostate cancer and for biochemical recurrence, the settings in which gallium-68 PSMA-11 and piflufolastat are already approved in the United States and Europe.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Florastamin F-18","url":"https://clinicaltrials.gov/search?intr=FC303"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["psma-pet","pet"],"targets":["psma"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct05936658","nct05004285","nct06754085"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"FC303","modality":"PSMA-targeted PET radiotracer, fluorine-18","mechanism":"A fluorine-18-labelled ligand of prostate-specific membrane antigen that lights up prostate cancer on PET wherever it has spread.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"flot","kind":"drug","name":"FLOT (5-FU, leucovorin, oxaliplatin, docetaxel)","aka":[],"tldr":"FLOT is the four-drug chemotherapy given before and after surgery for stomach cancer in the West; since 2025 immunotherapy is added to it.","summary":"FLOT4-AIO (2019): perioperative FLOT beat ECF/ECX (OS 50 vs 35 months). MATTERHORN added durvalumab (OS HR 0.78; FDA approval November 2025). Four cycles before and four after gastrectomy. Neutropenia, neuropathy, and diarrhoea; tolerability limits use in frail patients, where FOLFOX or CAPOX substitute.","status":"standard-of-care","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/FLOT","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/FLOT"}],"tags":[],"related":["folfox"],"cancers":["gastric","esophageal","oesophageal-adenocarcinoma"],"sections":[],"technologies":["cytotoxic-chemotherapy","platinum"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["flot-term"],"trials":["matterhorn","nct07775287","nct07228832","nct07621718","nct06535607","nct07790510","nct04430738","nct04083599","nct06792695","nct07805551","nct06820463","nct06948448","nct06219941","nct06943820","nct05919264","nct04421820","nct07432295","nct07474727","nct05382442","nct05379595","nct06428409"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Cytotoxic regimen","mechanism":"Docetaxel stabilises microtubules; oxaliplatin crosslinks DNA; 5-FU/leucovorin inhibit thymidylate synthase.","approvals":[],"mechanismSteps":["Docetaxel freezes the microtubule scaffold so cells cannot divide","Oxaliplatin crosslinks DNA","5-FU with leucovorin blocks thymidine synthesis","Three mechanisms at once shrink the tumour before surgery and mop up microscopic disease after"],"dosing":{"route":"Intravenous","schedule":"Docetaxel 50 mg/m², oxaliplatin 85 mg/m², leucovorin 200 mg/m², 5-FU 2,600 mg/m² over 24 h, every 14 days; 4 cycles pre- and 4 post-operative","modifications":"G-CSF support commonly used; reduce for neuropathy or neutropenic fever","monitoring":"Blood counts, neuropathy, nutrition"},"toxicity":[{"event":"Neutropenia","note":"Common, including febrile neutropenia"},{"event":"Peripheral neuropathy"},{"event":"Diarrhoea, mucositis"},{"event":"Fatigue"}],"access":[],"regulatoryEvents":[]},{"id":"flotufolastat","kind":"drug","name":"Flotufolastat F-18","aka":[],"tldr":"Flotufolastat is a third PSMA PET tracer, with a radiohybrid chemistry designed to be reused for therapy.","summary":"Flotufolastat F-18 (Posluma) is a radiohybrid PSMA ligand: a silicon-fluoride acceptor allows 18F labelling for PET while the same scaffold can carry 177Lu for therapy, so imaging and treatment agents share one chemistry. It is used for PSMA PET in men with prostate cancer at initial staging and at biochemical recurrence. The LIGHTHOUSE (initial staging) and SPOTLIGHT (biochemical recurrence) trials supported approval in May 2023. Low urinary excretion aids reading of the pelvis, where bladder activity can obscure local recurrence with other tracers. It is the third PSMA PET tracer in the US after gallium-68 PSMA-11 and Pylarify, and head-to-head comparisons are limited. For a newcomer, it is a PSMA scan agent designed from the start to be reused for therapy.","status":"approved","asOf":"2026-09-06","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Flotufolastat%20F-18"}],"tags":[],"related":[],"cancers":["prostate"],"sections":[],"technologies":["psma-pet"],"targets":["psma"],"drugs":[],"companies":["blue-earth-diagnostics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07285057","nct07455903","nct06617481"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Posluma","code":"18F-rhPSMA-7.3","modality":"PET imaging agent","mechanism":"Radiohybrid PSMA ligand; silicon-fluoride acceptor allows 18F imaging and 177Lu therapy on the same scaffold.","approvals":[{"region":"US","year":2023,"indication":"PSMA PET in prostate cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"floxuridine","kind":"drug","name":"Floxuridine","aka":["5-fluoro-2'-deoxyuridine","FdUrd"],"tldr":"Floxuridine is a cousin of fluorouracil pumped directly into the liver's artery to treat bowel cancer that has spread to the liver, delivering a very high local dose with little reaching the rest of the body.","summary":"Floxuridine was approved in the United States in 1970 for palliative treatment of gastrointestinal adenocarcinoma metastatic to the liver by continuous regional arterial infusion. Because the liver removes most of the drug on first pass, hepatic arterial infusion through an implanted pump gives the liver metastases many times the exposure that systemic therapy achieves. Randomised trials, mostly from Memorial Sloan Kettering, showed higher hepatic response rates and, combined with systemic chemotherapy after liver resection, better hepatic disease-free survival. Biliary sclerosis is the characteristic toxicity, and the technique is confined to specialist centres.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Floxuridine","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=floxuridine"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Floxuridine"},{"label":"ChEMBL CHEMBL1199","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1199"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["colorectal","hcc"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["tyms"],"drugs":["fluorouracil"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct00268463","nct03678428","nct05863195"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"FUDR","modality":"Fluoropyrimidine for hepatic arterial infusion","mechanism":"Converted in cells to fluorodeoxyuridine monophosphate, which locks up thymidylate synthase; given straight into the hepatic artery most of the dose is extracted by the liver on first pass.","approvals":[{"region":"US","year":1970,"indication":"Gastrointestinal adenocarcinoma metastatic to the liver, by continuous regional intra-arterial infusion"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fluciclovine-f18","kind":"drug","name":"Fluciclovine F-18","aka":[],"tldr":"Fluciclovine F-18 was the first PET tracer approved for finding recurrent prostate cancer after treatment (2016), and has been largely superseded since 2020 by PSMA PET.","summary":"Approved May 2016 for suspected recurrence with rising PSA; detection ~68% overall, lower at low PSA. EMSPOT/EMPIRE-1 (Lancet 2021) showed fluciclovine-guided salvage radiotherapy improved failure-free survival. PSMA PET (Ga-68 PSMA-11, piflufolastat, flotufolastat) has higher detection and displaced it.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Fluciclovine_(18F)","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Axumin"}],"tags":["gap-fill","diagnostic"],"related":[],"cancers":["prostate","glioblastoma"],"sections":[],"technologies":["pet","pet-ct"],"targets":[],"drugs":[],"companies":["blue-earth-diagnostics"],"institutions":[],"pathways":[],"terms":["biochemical-recurrence"],"trials":["nct04423211","nct04410133","nct05479136"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Axumin","modality":"PET radiotracer (synthetic amino acid)","mechanism":"18F-labelled leucine analogue taken up via LAT1/ASCT2 amino-acid transporters upregulated in prostate cancer; low urinary excretion improves pelvic imaging.","approvals":[{"region":"US","year":2016,"indication":"PET imaging in men with suspected prostate cancer recurrence based on elevated PSA"},{"region":"EU","year":2017,"indication":"Same"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fludarabine","kind":"drug","name":"Fludarabine","aka":[],"tldr":"Fludarabine is a chemotherapy infusion for chronic lymphocytic leukaemia. It anchored the FCR regimen that was the first to give long remissions, and today it is used above all to prepare patients for CAR-T cells and transplants.","summary":"Fludarabine phosphate was approved in 1991 for B-cell CLL that has not responded to, or has progressed during, at least one alkylating-agent regimen, on single-arm studies from MD Anderson and SWOG. Its lasting importance is in combinations: fludarabine, cyclophosphamide and rituximab (FCR; CLL8) was the first regimen to improve overall survival in CLL and remains an option for fit patients with mutated IGHV, while fludarabine plus cyclophosphamide is the standard lymphodepletion before every approved CAR-T product and part of reduced-intensity conditioning for allogeneic transplant. Prolonged T-cell depletion brings opportunistic infection, and autoimmune haemolysis is a class hazard; neurotoxicity occurs at high doses.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Fludarabine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=fludarabine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/fludarabinephosphate"}],"tags":["nci-list","generic"],"related":[],"cancers":["cll"],"sections":[],"technologies":["cytotoxic-chemotherapy","car-t","allogeneic-hsct"],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06413498","nct06045806","nct05371093","nct07486089","nct06480565","nct04792489","nct06253663","nct05826535","nct07589543","nct07510815","nct03907852","nct07149857","nct07617805","nct06119685","nct07480928","nct04245839","nct06093841","nct04268706","nct07627711","nct04836507","nct07500220","nct07627698","nct07641036","nct07551349","nct04416984","nct07523529","nct07502287","nct07589530","nct05624554","nct03591510","nct07598955","nct07015242","nct06500273","nct03182244","nct03793478","nct07551362"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Fludara (historic)","modality":"Purine nucleotide analogue (antimetabolite)","mechanism":"Dephosphorylated to 2-fluoro-ara-A and rephosphorylated to the triphosphate, which inhibits DNA polymerase alpha, ribonucleotide reductase and DNA primase and is incorporated into DNA.","approvals":[{"region":"US","year":1991,"indication":"B-cell chronic lymphocytic leukaemia after at least one alkylating-agent regimen"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2008-12-18","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"For adults with B-cell CLL that has not responded to or progressed during or after treatment with at least one standard alkylating agent containing regimen"},{"date":"2011-12-31","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.0 years after its accelerated approval.","indication":"For adults with B-cell CLL that has not responded to or progressed during or after treatment with at least one standard alkylating agent containing regimen"}]},{"id":"fludeoxyglucose-f18","kind":"drug","name":"Fludeoxyglucose F-18 (FDG)","aka":[],"tldr":"The radioactive sugar used in almost every cancer PET scan, approved in the 1990s and made fresh each day by regional cyclotrons.","summary":"Fludeoxyglucose F-18 was the first PET radiopharmaceutical approved by the FDA (1994, initially for epilepsy foci and cardiac viability) and gained its oncology indication in 2000 for assessing abnormal glucose metabolism to assist in evaluating malignancy in patients with known or suspected cancer. It is produced under multiple abbreviated NDAs by commercial and academic cyclotron networks (PETNET, Cardinal Health, SOFIE, hospital sites) and is the tracer behind staging, response assessment (Deauville in lymphoma, PERCIST) and surveillance across most solid tumours. Its weaknesses, uptake in inflammation and poor sensitivity for indolent or low-glucose tumours, drive the development of target-specific tracers.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Fludeoxyglucose_(18F)","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Fludeoxyglucose%20F%2018"}],"tags":["test"],"related":[],"cancers":[],"sections":[],"technologies":["fdg-pet","pet-ct","pet"],"targets":[],"drugs":[],"companies":["petnet-solutions","cardinal-health","sofie-biosciences"],"institutions":[],"pathways":[],"terms":["suv","deauville"],"trials":["ahod2131","nct04333537","nct04692103"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: 'hot spots' show tissue burning glucose fast; in a person with cancer they usually mark tumour, but infection, healing and brown fat can light up too, so findings are interpreted with CT and biopsy."],"brand":"FDG (multiple manufacturers)","modality":"PET imaging agent (glucose analogue)","mechanism":"18F-labelled glucose analogue taken up through GLUT transporters and trapped after phosphorylation by hexokinase, so metabolically active tumours accumulate signal.","approvals":[{"region":"US","year":1994,"indication":"First FDG NDA (neurology and cardiology indications)"},{"region":"US","year":2000,"indication":"Oncology: assessment of abnormal glucose metabolism to assist in evaluating malignancy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"flumatinib","kind":"drug","name":"Flumatinib","aka":["Xinfu"],"tldr":"Flumatinib is Hansoh Pharma's second-generation BCR-ABL inhibitor, approved in China in 2019 for newly diagnosed chronic-phase chronic myeloid leukaemia.","summary":"Flumatinib is an imatinib derivative developed by Hansoh Pharma. The NMPA approved it in November 2019 for adults with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase, after the FESTnd phase 3 trial reported deeper and faster molecular responses than imatinib. It is the first BCR-ABL inhibitor discovered and developed in China.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of flumatinib","url":"https://clinicaltrials.gov/search?intr=flumatinib"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["cml"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["bcr-abl"],"drugs":[],"companies":["hansoh-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04375683","nct01503502","nct05433532"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"HH-GV-678","modality":"Oral second-generation BCR-ABL tyrosine kinase inhibitor","mechanism":"Blocks the BCR-ABL fusion kinase that drives chronic myeloid leukaemia, with higher potency than imatinib.","approvals":[{"region":"CN","year":2019,"indication":"Newly diagnosed Philadelphia chromosome-positive chronic myeloid leukaemia, chronic phase"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fluoroestradiol-f18","kind":"drug","name":"Fluoroestradiol F-18 (FES PET)","aka":[],"tldr":"A PET scan that shows which breast cancer deposits still have oestrogen receptors, helping decide whether hormone therapy will work when biopsy is impractical.","summary":"Fluoroestradiol F-18 is a PET radiotracer in which 18F-labelled oestradiol binds oestrogen receptor alpha, imaging ER expression across every lesion in the body in a single non-invasive scan. It was approved in the US in May 2020 as an adjunct to biopsy for detecting ER-positive lesions in recurrent or metastatic breast cancer, and entered NCCN guidelines in 2024. It is used when biopsy is impractical or when lesions may have lost ER, because it predicts endocrine responsiveness and reveals heterogeneous receptor loss that a single biopsy would miss. It also serves as a pharmacodynamic tool in trials of ER degraders, measuring receptor occupancy, and is under study in ER-positive ovarian and endometrial cancers. Uptake in the liver limits assessment of hepatic metastases. FES PET tells the oncologist whether hormone therapy still has a target.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Fluoroestradiol_F-18","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Cerianna"}],"tags":["gap-fill","diagnostic"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["pet","pet-ct"],"targets":["estrogen-receptor"],"drugs":[],"companies":["ge-healthcare"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04692103","nct02398773","nct06260033"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cerianna","modality":"PET radiotracer (oestrogen receptor ligand)","mechanism":"18F-labelled oestradiol binds oestrogen receptor alpha, imaging ER expression across all lesions non-invasively.","approvals":[{"region":"US","year":2020,"indication":"Detection of ER-positive lesions as an adjunct to biopsy in patients with recurrent or metastatic breast cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fluorouracil","kind":"drug","name":"Fluorouracil (5-FU)","aka":[],"tldr":"The 1957 chemotherapy that remains the backbone of treatment for bowel, stomach, pancreatic, anal, head and neck and breast cancers, and as a cream for skin precancers.","summary":"Heidelberger (1957). Infusional 5-FU with leucovorin is the core of FOLFOX/FOLFIRI/FOLFIRINOX, chemoradiation (rectal, anal, oesophageal, pancreatic, head and neck), and adjuvant colon therapy; capecitabine is its oral prodrug. DPYD genotyping before use is now recommended (EMA 2020; FDA label 2024). Topical 5-FU treats actinic keratosis and superficial BCC.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Fluorouracil","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=fluorouracil%20injection"},{"label":"Jansen et al., five-year results of a randomised controlled trial comparing photodynamic therapy, topical imiquimod and topical 5-fluorouracil in superficial basal cell carcinoma (J Invest Dermatol 2018)","url":"https://doi.org/10.1016/j.jid.2017.09.033"},{"label":"Cancer Research UK: chemotherapy cream for non-melanoma skin cancer","url":"https://www.cancerresearchuk.org/about-cancer/skin-cancer/treatment/chemotherapy-cream"},{"label":"British Association of Dermatologists: basal cell carcinoma, patient information leaflet (updated July 2025)","url":"https://www.skinhealthinfo.org.uk/condition/basal-cell-carcinoma/"}],"tags":["gap-fill","generic","who-essential"],"related":["uridine-triacetate"],"cancers":["colorectal","gastric","pancreatic","anal","esophageal","head-and-neck","basal-cell-carcinoma","hepatoblastoma","anal-hsil-precursor","localised-anal-cancer","localised-penile-cancer","skin-cancer"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["mast-therapeutics"],"institutions":[],"pathways":[],"terms":["topical-and-destructive-treatment-bcc"],"trials":["int-0116","cao-aro-aio-94","dbcg-82bc","nct07775287","nct07676162","nct04430738","nct06107413","nct06792695","nct07805551","nct05009069","nct05846867","nct06820463","nct04421820","nct05379595","nct04675333","nct05489211","nct06428409","nct07554521","nct06469944","nct06901531","nct04241185","nct04379596","nct07069712","nct07256782","nct06780111","nct06922383","nct01954992","mal-pdt-imiquimod-fluorouracil-superficial-bcc"],"people":[],"bottlenecks":[],"keyPapers":["paper-sauer-preoperative-chemoradiotherapy-rectal-nejm-2004"],"journals":[],"dependsOn":[],"notes":["As a cream for superficial basal cell carcinoma. In the Dutch randomised trial of 601 patients, fluorouracil cream was applied twice daily for four weeks, and the probability of tumour-free survival five years later was 70.0 percent, against 80.5 percent for imiquimod and 62.7 percent for photodynamic therapy; fluorouracil was inferior to imiquimod (hazard ratio 0.65 in favour of imiquimod, 95 percent confidence interval 0.43 to 0.98) and not significantly different from photodynamic therapy at that timepoint, having been found non-inferior to it at one and three years. The NHS says a chemotherapy cream is used once or twice a day for three to four weeks for cancers affecting only the top layer of skin."],"brand":"Adrucil / Efudex","modality":"Fluoropyrimidine antimetabolite","mechanism":"Converted to FdUMP (thymidylate synthase inhibition) and FUTP (RNA incorporation); potentiated by leucovorin; DPD deficiency causes severe toxicity.","approvals":[{"region":"US","year":1962,"indication":"Colorectal, breast, gastric and pancreatic adenocarcinoma"},{"region":"US","year":1970,"indication":"Topical: actinic keratosis and superficial basal cell carcinoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fluoxymesterone","kind":"drug","name":"Fluoxymesterone","aka":["Androxy"],"tldr":"Fluoxymesterone is an oral male hormone approved in 1956 and once used to palliate advanced breast cancer in women, an approach abandoned when better tolerated antioestrogens and aromatase inhibitors arrived.","summary":"Androgens were among the first hormonal treatments for breast cancer, and fluoxymesterone, a potent oral androgen approved in the United States in 1956, carried a labelled indication for palliation of androgen-responsive recurrent breast cancer in women one to five years past the menopause. Virilisation, liver toxicity and fluid retention limited it, and by the 1980s tamoxifen and then the aromatase inhibitors had replaced androgen therapy. The drug has been discontinued in most markets, but androgen receptor biology in breast cancer is again under study with newer agents such as enobosarm.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Fluoxymesterone","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Fluoxymesterone"},{"label":"ChEMBL CHEMBL1445","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1445"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["endocrine-therapy"],"targets":["androgen-receptor"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Halotestin","modality":"Oral small-molecule synthetic androgen","mechanism":"A 17-alpha-alkylated testosterone derivative that activates the androgen receptor; in breast cancer it opposes oestrogen-driven growth and suppresses gonadotrophins.","approvals":[{"region":"US","year":1956,"indication":"Palliation of androgen-responsive advanced breast cancer in postmenopausal women (historic label)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"flutamide","kind":"drug","name":"Flutamide","aka":[],"tldr":"Flutamide was the first tablet to block testosterone at the prostate cancer cell. It is taken with an injection that stops testosterone production, but newer drugs have largely replaced it.","summary":"Flutamide was approved in 1989 for use with LHRH agonists in stage B2 to C prostate cancer (with radiotherapy) and in stage D2 metastatic disease, introducing combined androgen blockade. Trials in the 1980s and 1990s showed at best a small survival gain over castration alone, and bicalutamide, then enzalutamide, apalutamide and darolutamide superseded it because of three-times-daily dosing, diarrhoea and a boxed warning for hepatotoxicity including fatal liver failure. It is still listed for antiandrogen flare cover at the start of LHRH agonist therapy.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Flutamide","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=flutamide"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/flutamide"}],"tags":["nci-list","generic"],"related":["bicalutamide"],"cancers":["prostate"],"sections":[],"technologies":["androgen-deprivation"],"targets":["androgen-receptor"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00104741","nct00002881","nct03678025"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Eulexin (historic)","modality":"Non-steroidal antiandrogen (first generation)","mechanism":"Competitive antagonist of the androgen receptor; its active metabolite hydroxyflutamide blocks androgen binding and nuclear translocation in prostate cells.","approvals":[{"region":"US","year":1989,"indication":"Locally advanced (B2 to C) and metastatic (D2) prostate cancer in combination with an LHRH agonist"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fluzoparib","kind":"drug","name":"Fluzoparib","aka":[],"tldr":"Fluzoparib is an oral parp inhibitor from Risen (Suzhou) Pharma Tech Co., Ltd., in registered phase 2 trials for metastatic cancer.","summary":"Fluzoparib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Risen (Suzhou) Pharma Tech Co., Ltd., in metastatic cancer. A PARP inhibitor: the name stem -parib marks a small molecule that blocks the PARP DNA-repair enzyme, lethal to tumour cells that already lack a second repair pathway. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Fluzoparib","url":"https://clinicaltrials.gov/search?intr=Fluzoparib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["junshi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06208410"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral PARP inhibitor","mechanism":"A PARP inhibitor: the name stem -parib marks a small molecule that blocks the PARP DNA-repair enzyme, lethal to tumour cells that already lack a second repair pathway.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"folfiri","kind":"drug","name":"FOLFIRI (5-FU, leucovorin, irinotecan)","aka":[],"tldr":"FOLFIRI is the other backbone bowel-cancer chemotherapy, swapping oxaliplatin for irinotecan. Used first or second line and, since 2026, with the BRAF combination.","summary":"FOLFIRI pairs irinotecan, whose active metabolite SN-38 inhibits topoisomerase I, with 5-FU and leucovorin, which inhibit thymidylate synthase, given as a 46-hour infusion every 14 days. It is one of the two backbone chemotherapies for metastatic colorectal cancer, interchangeable with FOLFOX in first-line efficacy (Tournigand 2004) and the standard second-line switch. It is the partner for cetuximab (CRYSTAL, FIRE-3), bevacizumab, aflibercept, ramucirumab and, since 2026, encorafenib plus cetuximab in BRAF V600E disease (BREAKWATER cohort 3, PFS HR 0.44). Diarrhoea, neutropenia and alopecia are the main toxicities, and UGT1A1*28 homozygotes need irinotecan dose reduction. Whether to start with FOLFIRI or FOLFOX is decided by side-effect preference rather than efficacy. For a newcomer, FOLFIRI is bowel cancer chemotherapy with irinotecan instead of oxaliplatin.","status":"standard-of-care","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/FOLFIRI","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/FOLFIRI"}],"tags":[],"related":["folfox"],"cancers":["colorectal"],"sections":[],"technologies":["cytotoxic-chemotherapy","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["crystal-fire3","breakwater","nct07221357","nct06356311","nct07621718","nct06496048","nct07702032","nct07790510","nct07446322","nct06107413","nct06792695","nct05846867","nct06219941","nct03709680","nct03526835","nct04657068","nct07474727","nct05382442","nct05379595","nct05592626","nct07498725","tribe","tribe2","calgb-80405","velour","raise"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Cytotoxic regimen","mechanism":"Irinotecan (via SN-38) inhibits topoisomerase I; 5-FU/leucovorin inhibit thymidylate synthase.","approvals":[],"mechanismSteps":["Irinotecan is converted in the body to SN-38","SN-38 traps topoisomerase I on DNA, causing breaks during copying","5-FU with leucovorin starves the cell of thymidine","Dividing cancer cells cannot complete replication"],"dosing":{"route":"Intravenous","schedule":"Irinotecan 180 mg/m² + leucovorin 400 mg/m² day 1, 5-FU 400 mg/m² bolus then 2,400 mg/m² over 46 h, every 14 days","modifications":"Reduce irinotecan for UGT1A1*28/*28; hold for grade 3 diarrhoea","monitoring":"Blood counts; early and late diarrhoea"},"toxicity":[{"event":"Diarrhoea","note":"Early (cholinergic) and late; can be severe"},{"event":"Neutropenia"},{"event":"Alopecia"}],"access":[],"regulatoryEvents":[]},{"id":"folfirinox","kind":"drug","name":"FOLFIRINOX / mFOLFIRINOX","aka":[],"tldr":"FOLFIRINOX is a four-drug chemotherapy combination that, in 2011, became the first treatment to meaningfully extend life in metastatic pancreatic cancer; it is now the standard before and after surgery.","summary":"Oxaliplatin, irinotecan, leucovorin, and 5-fluorouracil. PRODIGE 4/ACCORD 11 (2011): OS 11.1 vs 6.8 months versus gemcitabine in fit metastatic patients. Modified FOLFIRINOX (PRODIGE 24, 2018) became the adjuvant standard after resection with median OS ~54 months, and is the most used neoadjuvant regimen for borderline-resectable disease. Toxicity (neutropenia, diarrhoea, neuropathy) restricts it to ECOG 0-1 patients. NALIRIFOX (liposomal irinotecan) is a 2024 variant for metastatic disease.","status":"standard-of-care","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/FOLFIRINOX","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/FOLFIRINOX"},{"label":"NICE NG85 recommendation 1.9.4","url":"https://www.nice.org.uk/guidance/ng85/chapter/Recommendations"},{"label":"Conroy et al., PRODIGE 4/ACCORD 11 (NEJM 2011)","url":"https://doi.org/10.1056/NEJMoa1011923"},{"label":"Conroy et al., PRODIGE 24 five-year outcomes (JAMA Oncology 2022)","url":"https://doi.org/10.1001/jamaoncol.2022.3829"}],"tags":[],"related":[],"cancers":["pancreatic","resectable-pdac","borderline-resectable-pdac","metastatic-pdac"],"sections":[],"technologies":["cytotoxic-chemotherapy","platinum","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["prodige-24","napoli-3","nct06922591","nct07255404","prodige-4-accord-11","preopanc-2","preopanc-3","alliance-a021806","alliance-a021501","swog-s1505","neolap","conko-007","panoptimox-prodige-35","crossfire","avenger-500","norpact-1","espac-5"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer, UK status: NICE NG85 1.9.4 offers FOLFIRINOX to people with metastatic disease and ECOG performance status 0 to 1 (recorded as an off-label use in February 2018), and modified FOLFIRINOX is given as adjuvant treatment on PRODIGE 24 although NG85 (2018) predates that trial and names gemcitabine plus capecitabine. Regulator status: a combination of individually licensed generic medicines with no single marketing authorisation. Four months of FOLFIRINOX followed by fluorouracil and leucovorin maintenance is an accepted alternative to six months (PANOPTIMOX-PRODIGE 35)."],"modality":"Cytotoxic regimen","mechanism":"DNA crosslinking (oxaliplatin), topoisomerase-I inhibition (irinotecan), antimetabolite (5-FU with leucovorin modulation).","approvals":[{"region":"Global","year":2011,"indication":"Metastatic PDAC (PRODIGE 4 / ACCORD 11; component drugs generic)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"folfox","kind":"drug","name":"FOLFOX (5-FU, leucovorin, oxaliplatin)","aka":[],"tldr":"FOLFOX is the workhorse chemotherapy combination for bowel cancer, used after surgery to cure and in advanced disease as the backbone that targeted drugs are added to.","summary":"Infusional 5-fluorouracil with leucovorin plus oxaliplatin every 2 weeks. MOSAIC (2004) established it as adjuvant therapy for stage III colon cancer; IDEA (2018) showed 3 months suffices for low-risk stage III with CAPOX. In mCRC it pairs with bevacizumab, cetuximab/panitumumab, encorafenib + cetuximab (BREAKWATER), or tucatinib + trastuzumab (MOUNTAINEER-03). Cumulative oxaliplatin neuropathy is the limiting toxicity.","status":"standard-of-care","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/FOLFOX","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/FOLFOX"},{"label":"Oettle et al., CONKO-003 (JCO 2014)","url":"https://doi.org/10.1200/JCO.2013.53.6995"},{"label":"Gill et al., PANCREOX (JCO 2016)","url":"https://doi.org/10.1200/JCO.2016.68.5776"}],"tags":[],"related":["folfiri","capox","folfirinox"],"cancers":["colorectal","gastric","pancreatic","pancreatic-acinar-cell-carcinoma","appendiceal-adenocarcinoma","goblet-cell-adenocarcinoma","localised-small-bowel-adenocarcinoma","advanced-small-bowel-adenocarcinoma","gallbladder","cholangiocarcinoma","metastatic-pdac"],"sections":[],"technologies":["cytotoxic-chemotherapy","platinum"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["breakwater","paradigm","atomic","mountaineer","nct07221357","nct07702032","nct05482893","nct07659795","nct04068610","nct03526835","nct06324357","nct02671435","nct07498725","abc-06","sevilla","combomatch-binimetinib-folfox","conko-003","sequoia","alliance-a021501","primus-001","mosaic","idea-collaboration","scot","foxtrot","eortc-40983","prospect"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Gallbladder cancer: ABC-06, the UK phase 3 that made FOLFOX the second-line standard for biliary tract cancer (overall survival 6.2 versus 5.3 months, hazard ratio 0.69), enrolled gallbladder and ampullary carcinoma alongside cholangiocarcinoma. FOLFOX is the comparator in the UK-French SEVILLA trial of ivonescimab and the backbone of the ComboMATCH binimetinib trial.","Pancreatic cancer: oxaliplatin with fluorouracil and folinic acid is a second-line option after gemcitabine on CONKO-003 (OFF schedule: overall survival 5.9 against 3.3 months), but PANCREOX found modified FOLFOX6 worse than fluorouracil and leucovorin alone (6.1 against 9.9 months); FOLFOX was the control arm of SEQUOIA (6.3 months) and the post-operative regimen in Alliance A021501."],"code":"mFOLFOX6","modality":"Cytotoxic regimen","mechanism":"5-FU inhibits thymidylate synthase (potentiated by leucovorin); oxaliplatin forms platinum-DNA adducts.","approvals":[{"region":"US","year":2004,"indication":"Adjuvant stage III colon cancer (oxaliplatin, MOSAIC)"}],"mechanismSteps":["Oxaliplatin crosslinks DNA so it cannot be copied","5-FU blocks the enzyme that makes the building block thymidine","Leucovorin locks 5-FU onto that enzyme for longer","Rapidly dividing cancer cells cannot replicate and die"],"dosing":{"route":"Intravenous","schedule":"Oxaliplatin 85 mg/m² + leucovorin 400 mg/m² day 1, 5-FU 400 mg/m² bolus then 2,400 mg/m² over 46 h, every 14 days","modifications":"Stop or reduce oxaliplatin for persistent grade 2 neuropathy; DPD deficiency testing before 5-FU is recommended in Europe","monitoring":"Blood counts, neuropathy, liver function"},"toxicity":[{"event":"Peripheral sensory neuropathy","note":"Cumulative, often persistent; oxaliplatin"},{"event":"Neutropenia"},{"event":"Diarrhoea, mucositis"},{"event":"Cold-induced dysaesthesia","note":"Acute, oxaliplatin"}],"access":[],"regulatoryEvents":[]},{"id":"folfoxiri","kind":"drug","name":"FOLFOXIRI (5-FU, leucovorin, oxaliplatin, irinotecan)","aka":[],"tldr":"All three of the active bowel cancer chemotherapy drugs given together instead of two. It shrinks more tumours than a doublet and is chosen when shrinking the tumour is what matters, usually with bevacizumab.","summary":"FOLFOXIRI combines infusional fluorouracil and leucovorin with both oxaliplatin and irinotecan every fourteen days, most often with bevacizumab. TRIBE showed that FOLFOXIRI plus bevacizumab gives a longer progression-free survival (12.1 against 9.7 months) and a higher response rate (65 against 53 percent) than FOLFIRI plus bevacizumab in untreated metastatic disease, and TRIBE2 showed the advantage survives as a strategy, with progression-free survival 2 of 19.2 against 16.4 months against a planned sequence of doublets. CAIRO5 found it the best induction for right-sided or RAS/BRAF-mutant liver-limited disease. The price is toxicity: grade 3 or 4 neutropenia in about half of patients, and more diarrhoea, stomatitis and neuropathy, so it is reserved for fit patients under about 75 with good performance status. In practice it is used when deep response matters most, to convert unresectable liver metastases, in BRAF V600E disease before the targeted triplet was available, and in young patients with heavy disease burden. Induction is usually limited to eight to twelve cycles followed by fluoropyrimidine and bevacizumab maintenance.","status":"established","asOf":"2026-09-24","links":[{"label":"TRIBE: initial therapy with FOLFOXIRI and bevacizumab for metastatic colorectal cancer (New England Journal of Medicine 2014)","url":"https://doi.org/10.1056/NEJMoa1403108"},{"label":"TRIBE2 (Lancet Oncology 2020)","url":"https://doi.org/10.1016/S1470-2045(19)30862-9"},{"label":"NCCN Colon Cancer guideline","url":"https://www.nccn.org/guidelines/guidelines-detail?category=1&id=1428"}],"tags":[],"related":[],"cancers":["colorectal"],"sections":[],"technologies":["cytotoxic-chemotherapy","platinum","topoisomerase-inhibitors"],"targets":["dpyd","ugt1a1"],"drugs":["fluorouracil","leucovorin","oxaliplatin","irinotecan","bevacizumab"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["tribe","tribe2","cairo5"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"mFOLFOXIRI","modality":"Cytotoxic regimen","mechanism":"Oxaliplatin crosslinks DNA, irinotecan (through SN-38) traps topoisomerase I, and fluorouracil with leucovorin inhibits thymidylate synthase: three separate attacks on DNA replication at once.","approvals":[],"mechanismSteps":["Oxaliplatin forms platinum adducts that crosslink the two strands of DNA","Irinotecan is converted to SN-38, which traps topoisomerase I on DNA and causes strand breaks","Fluorouracil, held on the enzyme by leucovorin, starves the cell of thymidine","A dividing cell facing all three cannot repair or replicate and dies"],"dosing":{"route":"Intravenous","schedule":"Irinotecan 165 mg/m2 and oxaliplatin 85 mg/m2 with leucovorin 200 mg/m2 on day 1, then fluorouracil 3,200 mg/m2 as a 48-hour infusion, every 14 days (modified schedules reduce irinotecan to 150 mg/m2 and fluorouracil to 2,400 mg/m2)","modifications":"Dose-reduce for UGT1A1*28 homozygotes and for age over 70; test DPYD before any fluoropyrimidine","monitoring":"Full blood count before each cycle, neuropathy score, early and late diarrhoea"},"toxicity":[{"event":"Neutropenia (grade 3-4)","grade3PlusPct":50,"source":"https://doi.org/10.1016/S1470-2045(19)30862-9","note":"50 percent in the TRIBE2 experimental arm against 21 percent with a doublet"},{"event":"Diarrhoea (grade 3-4)","grade3PlusPct":17,"source":"https://doi.org/10.1016/S1470-2045(19)30862-9"},{"event":"Peripheral sensory neuropathy","note":"Cumulative, from oxaliplatin"},{"event":"Stomatitis"}],"access":[],"regulatoryEvents":[]},{"id":"formestane","kind":"drug","name":"Formestane","aka":["4-Hydroxyandrostenedione","4-OHA"],"tldr":"Formestane, launched in Europe in 1993, was the first selective aromatase inhibitor for advanced breast cancer, given by injection every two weeks; oral exemestane and the non-steroidal inhibitors made it redundant within a few years.","summary":"Formestane was developed by Angela Brodie's laboratory in the 1970s as the first aromatase inhibitor designed to be selective, in contrast to aminoglutethimide, and Ciba-Geigy brought it to market in the United Kingdom and other European countries in 1993 for advanced postmenopausal breast cancer after tamoxifen. It lowered oestrogen levels effectively but had to be injected because of poor oral bioavailability. Anastrozole, letrozole and the oral steroidal inactivator exemestane followed between 1995 and 1999 and displaced it; it was withdrawn from most markets in the mid-2000s. It was never approved in the United States.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Formestane","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Formestane"},{"label":"ChEMBL CHEMBL1201310","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201310"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["endocrine-therapy"],"targets":["aromatase"],"drugs":["exemestane"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lentaron","modality":"Steroidal aromatase inactivator, fortnightly intramuscular injection","mechanism":"An androstenedione analogue that aromatase binds and converts to a reactive intermediate, permanently inactivating the enzyme that makes oestrogen.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fostamatinib","kind":"drug","name":"Fostamatinib","aka":["Fostamatinib Disodium","R788","R935788"],"tldr":"Fostamatinib is a tablet that blocks the enzyme SYK so the spleen stops destroying antibody-coated platelets; it is approved for chronic immune thrombocytopenia and was first trialled as a lymphoma drug.","summary":"Fostamatinib is an oral SYK inhibitor approved in the United States in 2018 for adults with chronic immune thrombocytopenia (ITP) who have not responded to a previous treatment. SYK sits downstream of the B-cell receptor and of Fc receptors, and fostamatinib was originally studied in B-cell lymphomas and chronic lymphocytic leukaemia, where responses were modest and the programme moved to autoimmune disease. Its place in cancer care is therefore supportive: managing immune-mediated low platelets, including in patients whose ITP accompanies lymphoid cancers. Hypertension, diarrhoea and raised liver enzymes are the main side effects.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Fostamatinib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=fostamatinib"},{"label":"Drugs@FDA NDA 209299","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=209299"}],"tags":["gap-fill","supportive-care"],"related":[],"cancers":["cll","dlbcl"],"sections":[],"technologies":["supportive-care","kinase-inhibitors"],"targets":[],"drugs":[],"companies":["rigel-pharmaceuticals"],"institutions":[],"pathways":[],"terms":["thrombocytopenia"],"trials":["nct01499303","nct00923481","nct04543279"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tavalisse","modality":"Oral spleen tyrosine kinase (SYK) inhibitor","supportive":true,"mechanism":"A prodrug converted to R406, which inhibits SYK and so blocks the antibody-triggered destruction of platelets by macrophages in the spleen.","approvals":[{"region":"US","year":2018,"indication":"Chronic immune thrombocytopenia in adults with insufficient response to a previous treatment"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"foundationone-cdx","kind":"drug","name":"FoundationOne CDx / Liquid CDx","aka":[],"tldr":"The FDA-approved tissue (324 genes) and blood genomic tests that serve as companion diagnostics for dozens of drugs.","summary":"FoundationOne CDx is Foundation Medicine's (Roche) tissue-based comprehensive genomic profiling test, using hybrid-capture next-generation sequencing of 324 genes to report mutations, copy-number changes, fusions, tumour mutational burden, microsatellite instability and homologous recombination deficiency by loss of heterozygosity; FoundationOne Liquid CDx applies the approach to circulating tumour DNA in blood. The tissue test was FDA-approved in 2017 and the liquid test in 2020. They are companion diagnostics for more than 30 therapies, including olaparib, capivasertib, trastuzumab deruxtecan for HER2-mutant NSCLC and selpercatinib, so one report can open several treatment doors at once. Tissue-blood concordance and unequal access remain issues. For a newcomer: a single sequencing test that tells an oncologist which targeted drugs a tumour might respond to.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA premarket approval P170019: FoundationOne CDx","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P170019"},{"label":"Foundation Medicine: FoundationOne CDx","url":"https://www.foundationmedicine.com/test/foundationone-cdx"}],"tags":[],"related":["tmb-testing"],"cancers":["tnbc","pancreatic","colorectal"],"sections":[],"technologies":["cgp","companion-diagnostic","liquid-biopsy"],"targets":[],"drugs":[],"companies":["foundation-medicine"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-radovich-ctdna-ctc-bre12-158-jama-oncol-2020","paper-barroso-sousa-breast-tmb-prevalence-ann-oncol-2020","paper-singhi-targeted-genome-profiling-3594-pdac-gastroenterology-2019","paper-pishvaian-lancet-oncol","paper-sepulveda-molecular-biomarkers-colorectal-guideline-jco-2017"],"journals":[],"dependsOn":[],"notes":["Pancreatic ductal adenocarcinoma: the 3,594-tumour profiling series that fixed the 88% KRAS rate, the 14% repair-gene rate and the 0.5% MSI or TMB-high rate was run on this panel (Singhi 2019); comprehensive profiling found actionable alterations in 26% of 1,082 Know Your Tumor patients (Pishvaian 2020).","Colorectal cancer: a comprehensive panel covers extended RAS, BRAF, MSI, tumour mutational burden and HER2 amplification in one test, which is what the guideline asks for, but panel content decides what is seen: RSPO2, ACVR2A and BCL9L are absent from several widely used panels, and fusion detection depends on intron baiting or RNA (Sepulveda 2017)."],"modality":"Comprehensive genomic profiling test","mechanism":"Hybrid-capture NGS of 324 genes.","approvals":[{"region":"US","year":2017,"indication":"Tissue CGP companion diagnostic (Liquid CDx 2020)"},{"region":"EU","year":2019,"indication":"CE-IVD"}],"mechanismSteps":["FFPE tumour DNA is extracted","Hybrid-capture enrichment of 324 genes","Deep sequencing detects mutations, copy-number changes, fusions, TMB, MSI","Variants are matched to FDA-approved therapies and trials in a report"],"toxicity":[],"access":[{"country":"US","listPrice":"~$3,500 (Medicare CLFS rate for FoundationOne CDx)","reimbursement":"Medicare national coverage for FDA-approved CGP in advanced cancer (NCD 90.2); commercial coverage broad","source":"https://www.foundationmedicine.com","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-11-30","type":"approval","region":"US","note":"FDA approval and parallel Medicare NCD: first FDA-approved broad CGP companion diagnostic","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-08-26","type":"approval","region":"US","note":"FoundationOne Liquid CDx approved","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-06","type":"label-change","region":"US","note":"TMB ≥10 mut/Mb companion claim for pembrolizumab","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"fpi-2265","kind":"drug","name":"FPI-2265","aka":["Ac225-PSMA I&T"],"tldr":"FPI-2265 is an experimental radioligand therapy from Fusion Pharmaceuticals in phase 2 trials for prostate cancer, aimed at PSMA.","summary":"FPI-2265 is a radioligand therapy developed by Fusion Pharmaceuticals. Its target is PSMA (the sponsor names PSMA). The sponsor states: A PSMA-targeting ligand (PSMA-I&T) radiolabeled with the alpha-emitter actinium-225, delivering targeted alpha radiotherapy to PSMA-positive prostate cancer cells. ClinicalTrials.gov describes the intervention as: Small molecule capable of binding to the domain of PSMA radiolabeled with Ac225. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in prostate cancer. The largest, NCT05219500, plans to enrol 115 participants with primary completion was scheduled for 2025-05-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of FPI-2265","url":"https://clinicaltrials.gov/search?intr=FPI-2265"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["psma"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07611110","nct05219500","nct07590934","nct06909825"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"radioligand","mechanism":"A PSMA-targeting ligand (PSMA-I&T) radiolabeled with the alpha-emitter actinium-225, delivering targeted alpha radiotherapy to PSMA-positive prostate cancer cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"fruquintinib","kind":"drug","name":"Fruquintinib","aka":[],"tldr":"A Chinese-discovered pill that blocks the blood-vessel receptors, approved in 2023 for bowel cancer after all standard treatments.","summary":"Fruquintinib is an ATP-competitive inhibitor of VEGFR1, 2 and 3 with little activity on other kinases, discovered in China, that starves tumours of new blood vessels with fewer off-target effects than regorafenib. It is used for refractory metastatic colorectal cancer after fluoropyrimidine, oxaliplatin, irinotecan, anti-VEGF and, where appropriate, anti-EGFR therapy, taken 5 mg once daily for 21 days of a 28-day cycle. FRESCO (China, 2018) and FRESCO-2 (global, 2023) showed an overall survival benefit, 7.4 versus 4.8 months in FRESCO-2 (HR 0.66); FDA approval followed in November 2023 and EU approval in 2024. It is better tolerated than regorafenib; hypertension, hand-foot syndrome, proteinuria and hypothyroidism are the main toxicities. There is no head-to-head trial against regorafenib. For a newcomer, it is a late-line bowel cancer pill that adds a few months with manageable side effects.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Fruquintinib","links":[{"label":"FDA approval summary, Clinical Cancer Research","url":"https://aacrjournals.org/clincancerres/article/30/15/3100/746597/FDA-Approval-Summary-Fruquintinib-for-the"},{"label":"NICE TA1079: fruquintinib for previously treated metastatic colorectal cancer","url":"https://www.nice.org.uk/guidance/ta1079"},{"label":"EMA Fruzaqla","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/fruzaqla"}],"tags":[],"related":[],"cancers":["colorectal"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf"],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["fresco-2","nct06199973","nct06584032","nct06497985","nct07235293","nct05565417","nct06992258","nct05405595","nct05867420","fresco"],"people":["su-weiguo"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Fruzaqla","modality":"Small-molecule kinase inhibitor (VEGFR1-3)","mechanism":"ATP-competitive inhibition of VEGFR1, 2, and 3 tyrosine kinases.","approvals":[{"region":"China","year":2018,"indication":"Refractory mCRC (FRESCO)"},{"region":"US","year":2023,"indication":"Refractory mCRC after fluoropyrimidine, oxaliplatin, irinotecan, anti-VEGF, and anti-EGFR if appropriate"},{"region":"EU","year":2024,"indication":"Previously treated metastatic colorectal cancer","note":"Fruzaqla marketing authorisation issued 20 June 2024."},{"region":"England (NICE)","year":2025,"indication":"Metastatic colorectal cancer at third line or later, only when trifluridine-tipiracil with bevacizumab is unsuitable","note":"TA1079, published 23 July 2025, with a commercial arrangement; it must be funded in England within 90 days of publication."}],"mechanismSteps":["Fruquintinib enters blood-vessel cells and binds VEGFR kinases","VEGF signals can no longer be transmitted","Tumour vessel growth halts","Tumour growth slows; survival is extended modestly"],"dosing":{"route":"Oral","schedule":"5 mg once daily, days 1-21 of a 28-day cycle","modifications":"Reduce to 4 mg then 3 mg for toxicity","monitoring":"Blood pressure, urine protein, liver tests, thyroid","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217564s000lbl.pdf"},"toxicity":[{"event":"Hypertension","note":"Most common grade 3 event"},{"event":"Hand-foot syndrome"},{"event":"Proteinuria"},{"event":"Hypothyroidism"}],"access":[],"regulatoryEvents":[{"date":"2023-11-08","type":"approval","region":"US","note":"FRESCO-2"}]},{"id":"fulvestrant","kind":"drug","name":"Fulvestrant","aka":[],"tldr":"Fulvestrant is a monthly intramuscular injection that degrades the oestrogen receptor rather than blocking it, the endocrine backbone paired with CDK4/6, PI3K and AKT inhibitors once aromatase inhibitors fail. Slow uptake and incomplete receptor degradation drove the search for oral degraders.","summary":"First selective oestrogen receptor degrader (2002). Backbone of PALOMA-3, MONARCH 2, MONALEESA-3, SOLAR-1, CAPItello-291, INAVO120, and the control arm of every oral SERD trial. Limitations: intramuscular 500 mg injections, slow steady state, and incomplete ER degradation, which motivated oral SERDs and the PROTAC vepdegestrant.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Fulvestrant","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Fulvestrant"}],"tags":[],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["capitello-291","inavo120","solar-1","postmonarch","veritac-2","nct06506955","nct06726148","nct05735080","nct04557449","nct07287917","nct04553133","nct04606446","nct07288359","nct05867251","nct07109726","nct04567420","nct05501886","nct05768139","nct06979596","nct06202261","nct06065748","nct06736704","nct02763566","nct07558733","nct06016738","nct05696626","nct07498478","nct06555068","nct06702618","nct02107703","nct06635447","nct06957379","nct05646862","nct00099437","nct04214288","nct07389733","nct04115306","nct05262400","nct05230810","nct06428396","nct06757634","nct07368998","nct04862663","nct04576455","nct05038735","nct07235176","nct04579380","nct05251714","nct04539496","nct06380751","nct06764186","nct04524000","nct06993844","nct07405801","nct04544189","nct05573126","nct05226871","nct04851613"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Faslodex","modality":"Injectable SERD","mechanism":"Binds ER, blocks dimerisation and nuclear localisation, accelerates proteasomal degradation.","approvals":[{"region":"US","year":2002,"indication":"HR+ advanced breast cancer after antioestrogen therapy"},{"region":"US","year":2017,"indication":"First-line HR+ advanced breast cancer (FALCON)"}],"mechanismSteps":[],"dosing":{"route":"Intramuscular","schedule":"500 mg on days 1, 15, 29, then monthly","monitoring":"Injection-site reactions"},"toxicity":[{"event":"Injection-site pain","anyGradePct":14},{"event":"Hot flushes","anyGradePct":13},{"event":"Arthralgia","anyGradePct":17}],"access":[],"regulatoryEvents":[{"date":"2026-07-14","type":"approval","region":"US","note":"FDA approves gedatolisib with fulvestrant, with or without palbociclib, for HR-positive, HER2-negative locally advanced or metastatic breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-gedatolisib-fulvestrant-or-without-palbociclib-hr-positive-her2-negative-locally"}]},{"id":"fulzerasib","kind":"drug","name":"Fulzerasib","aka":[],"tldr":"Fulzerasib was the first KRAS G12C-blocking pill approved in China, for lung cancers carrying that mutation after earlier treatment.","summary":"Discovered by GenFleet Therapeutics and developed in China with Innovent, fulzerasib was approved by the NMPA in August 2024 for KRAS G12C-mutant advanced NSCLC after at least one systemic therapy, on a pivotal single-arm phase 2 study (Journal of Thoracic Oncology 2024) with a confirmed response rate near half of patients. It arrived three years after sotorasib in the US but within weeks of garsorasib, giving China two domestic KRAS G12C inhibitors before either Western drug was approved there. First-line combinations with cetuximab are in trials.","status":"approved","asOf":"2026-09-10","links":[{"label":"GenFleet Therapeutics","url":"https://www.genfleet.com/en/"},{"label":"IBI351 pivotal phase 2 (J Thorac Oncol 2024)","url":"https://doi.org/10.1016/j.jtho.2024.08.005"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":["kras-inhibitors","kinase-inhibitors"],"targets":["kras"],"drugs":[],"companies":["genfleet","innovent"],"institutions":[],"pathways":["ras-mapk"],"terms":["kras-mutation-subtypes"],"trials":["nct07601048","nct07581912","nct06936644"],"people":["yu-michael"],"bottlenecks":[],"keyPapers":["paper-zhou-j-thorac-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Dupert","code":"GFH925, IBI351","modality":"Small-molecule covalent KRAS G12C inhibitor","mechanism":"Covalent inhibitor locking KRAS G12C in its inactive GDP-bound state, blocking downstream RAF-MEK-ERK signalling.","approvals":[{"region":"China","year":2024,"indication":"KRAS G12C-mutant advanced NSCLC after at least one systemic therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"furmonertinib","kind":"drug","name":"Furmonertinib","aka":[],"tldr":"Furmonertinib is a Chinese lung cancer pill of the same class as osimertinib, which more than doubled the time before cancer grew compared with gefitinib in the FURLONG trial.","summary":"Developed by Shanghai Allist Pharmaceuticals; approved by the NMPA in March 2021 for EGFR T790M-positive NSCLC after earlier EGFR inhibitors and in June 2022 for first-line EGFR-mutant NSCLC on FURLONG (358 patients: median progression-free survival 20.8 versus 11.1 months with gefitinib, hazard ratio 0.44). A higher dose is being developed for EGFR exon 20 insertions, where ArriVent BioPharma holds ex-China rights and is running the global FURVENT phase 3 trial. Adjuvant and central nervous system studies continue.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Furmonertinib","links":[{"label":"Shanghai Allist Pharmaceuticals","url":"https://www.allist.com.cn/"},{"label":"FURLONG (Lancet Respir Med 2022)","url":"https://doi.org/10.1016/S2213-2600(22)00168-0"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["nsclc","egfr-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["allist"],"institutions":[],"pathways":["ras-mapk"],"terms":["egfr-mutation-subtypes"],"trials":["furlong","nct05607550","nct07185997","nct06962865","nct07622186","nct05994131","nct04853342","nct05466149","nct06970639"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ivesa","code":"AST2818, alflutinib","modality":"Small-molecule third-generation EGFR TKI","mechanism":"Irreversible third-generation EGFR inhibitor active against sensitising mutations and T790M with a trifluoroethoxy pyridine that improves brain penetration; its main metabolite is also active.","approvals":[{"region":"China","year":2021,"indication":"EGFR T790M-positive locally advanced or metastatic NSCLC after EGFR TKI therapy"},{"region":"China","year":2022,"indication":"First-line EGFR exon 19 deletion or L858R NSCLC (FURLONG)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"futibatinib","kind":"drug","name":"Futibatinib","aka":[],"tldr":"Futibatinib is a covalent FGFR inhibitor for FGFR2-fusion bile duct cancer, with the highest response rate of the first-generation drugs.","summary":"Futibatinib is an irreversible inhibitor of FGFR1-4 that binds covalently to a cysteine in the kinase P-loop, a mechanism that retains activity against some resistance mutations that defeat reversible FGFR inhibitors. It is used in previously treated intrahepatic cholangiocarcinoma with an FGFR2 fusion or rearrangement. FOENIX-CCA2 (NEJM 2023) showed an ORR of 42%, median PFS of 9.0 months and median OS of 21.7 months, the highest response rate of the first-generation FGFR drugs. Accelerated approval followed in September 2022 (EMA 2023) at 20 mg daily continuously; hyperphosphataemia is near universal (85%, 30% grade 3 or higher). Whether it should be preferred over pemigatinib, and how to treat acquired FGFR2 kinase-domain mutations, are open questions now addressed by agents such as tinengotinib. For a newcomer, futibatinib is the covalent FGFR pill for bile duct cancer.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Futibatinib","links":[{"label":"NEJM 2023","url":"https://www.nejm.org/doi/full/10.1056/NEJMoa2206834"},{"label":"NICE TA1005: futibatinib for previously treated FGFR2 fusion or rearrangement cholangiocarcinoma (11 September 2024)","url":"https://www.nice.org.uk/guidance/ta1005"}],"tags":[],"related":["fgfr2-fusion-rearrangement"],"cancers":["cholangiocarcinoma","intrahepatic-cholangiocarcinoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["fgfr2"],"drugs":[],"companies":["taiho"],"institutions":[],"pathways":[],"terms":["fgfr2-fusion"],"trials":["foenix-cca2","nct06506955","nct07710885","nct05945823"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Gallbladder cancer: the FDA indication is intrahepatic cholangiocarcinoma with an FGFR2 fusion or rearrangement (Lytgobi label), so futibatinib has no licensed or trial role in gallbladder cancer."],"brand":"Lytgobi","modality":"Small-molecule irreversible kinase inhibitor (FGFR1-4)","mechanism":"Irreversible covalent binder of the FGFR kinase P-loop cysteine.","approvals":[{"region":"US","year":2022,"indication":"Previously treated FGFR2-fusion/rearranged intrahepatic cholangiocarcinoma (accelerated)"},{"region":"EU","year":2023,"indication":"Same"},{"region":"UK","year":2024,"indication":"Locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement after at least one systemic treatment; NICE TA1005 recommended 11 September 2024 as an alternative to pemigatinib","note":"https://www.nice.org.uk/guidance/ta1005"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"20 mg daily continuously","monitoring":"Phosphate, eyes"},"toxicity":[{"event":"Hyperphosphataemia","anyGradePct":85,"grade3PlusPct":30,"note":"FOENIX-CCA2"},{"event":"Alopecia","anyGradePct":33},{"event":"Dry mouth","anyGradePct":30}],"access":[],"regulatoryEvents":[{"date":"2022-09-30","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 4.0 years later, when the FDA's table was read.","indication":"Adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements"}]},{"id":"galeterone","kind":"drug","name":"Galeterone","aka":["TOK-001","VN/124-1"],"tldr":"An experimental prostate cancer tablet meant to work in men whose cancer makes a broken form of the androgen receptor. The trial designed to prove it collapsed.","summary":"Galeterone is a steroidal compound with three claimed activities against the androgen axis at once: inhibition of CYP17 lyase, direct androgen receptor antagonism, and degradation of androgen receptor protein including the constitutively active splice variant AR-V7, which lacks the ligand-binding domain and so cannot be blocked by abiraterone or enzalutamide.\n\nThat last property was the whole commercial thesis. AR-V7 detected in circulating tumour cells predicts a poor response to abiraterone and enzalutamide, so a drug that degrades the receptor rather than blocking its ligand pocket should work where they do not. Tokai Pharmaceuticals tested the thesis in ARMOR3-SV, the only phase 3 trial in prostate cancer to select men by AR-V7 status.\n\nThe trial prescreened 953 men to find 73 who were AR-V7 positive, a prevalence of 8 percent (95 percent confidence interval 6 to 10). Of those, 38 were randomised before the data monitoring committee recommended early closure: too many men progressed and left the trial before the required radiographs. Tokai stopped developing galeterone. No approval was ever sought.","status":"withdrawn","asOf":"2026-09-25","links":[{"label":"ARMOR3-SV (European Urology 2019)","url":"https://doi.org/10.1016/j.eururo.2019.08.034"},{"label":"ClinicalTrials.gov NCT02438007","url":"https://clinicaltrials.gov/study/NCT02438007"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":[],"targets":["androgen-receptor","cyp17a1"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["ar-v7","arpi","castration-resistance"],"trials":["armor3-sv"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"TOK-001","modality":"small molecule","mechanism":"CYP17 lyase inhibition, androgen receptor antagonism and androgen receptor protein degradation, including the AR-V7 splice variant.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"galleri","kind":"drug","name":"Galleri","aka":[],"tldr":"A blood test screening for more than 50 cancers at once, before the FDA in September 2026.","summary":"Galleri is GRAIL's methylation-based MCED. Available as an LDT since 2021 (~$949). PATHFINDER 2 (25,000+ participants) and NHS-Galleri (140,000 randomised) underpin the PMA submitted January 2026; FDA advisory committee 23 September 2026. Cancer signal detected in ~1% of screened adults with PPV ~40-60% in PATHFINDER 2; sensitivity for stage I disease is low.\n\nIn the NHS-Galleri randomised trial, the peer-reviewed test-performance paper (Nature Medicine, 22 September 2026) reports that about 1 in 100 intervention-arm participants had a positive result in each of three annual rounds (1.03, 0.80 and 0.90 percent), that 58.0, 50.4 and 45.8 percent of those positives were cancer, that specificity was 99.50 to 99.60 percent, and that the test found 26.7 to 37.2 percent of all cancers and 47.6 to 63.4 percent of the 12 prespecified types in a screening round; the same paper states the trial's primary endpoint, a reduction in stage III/IV cancer incidence, was not met.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"GRAIL PMA announcement","url":"https://grail.com/press-releases/grail-submits-fda-premarket-approval-application-for-the-galleri-multi-cancer-early-detection-test/"},{"label":"NHS-Galleri test performance (Nature Medicine 2026)","url":"https://doi.org/10.1038/s41591-026-04652-8"}],"tags":[],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":["mced","liquid-biopsy","methylation-profiling","cfdna-methylation-testing"],"targets":[],"drugs":[],"companies":["grail"],"institutions":[],"pathways":[],"terms":[],"trials":["nhs-galleri","pathfinder-2"],"people":[],"bottlenecks":[],"keyPapers":["paper-nhs-galleri-performance-nat-med-2026","paper-pathfinder-lancet-2023","paper-nhs-galleri-design-cancers-2022","paper-klein-ccga3-mced-validation-ann-oncol-2021"],"journals":[],"dependsOn":[],"notes":["Pancreatic ductal adenocarcinoma: the CCGA3 validation read 16.8% sensitivity at stage I across all cancers at 99.5% specificity, which is the level at which a screening test would have to find pancreatic cancer to change its outcome; no pancreatic screening indication exists (Klein 2021)."],"modality":"Multi-cancer early detection blood test","mechanism":"Targeted cfDNA methylation sequencing with machine-learned cancer signal and tissue-of-origin classifier.","approvals":[],"mechanismSteps":["Blood is drawn and cell-free DNA extracted","Targeted methylation sequencing of ~100,000 regions","Machine-learning classifier detects a shared cancer methylation signal","A second classifier predicts the tissue of origin","A positive result directs diagnostic imaging and biopsy"],"toxicity":[],"access":[{"country":"US","listPrice":"$949 self-pay (GRAIL list price)","reimbursement":"Not covered by Medicare (pending FDA decision and the MCED Screening Coverage Act); some employer and Medicare Advantage programmes cover","source":"https://www.galleri.com/","asOf":"2026-09-06"},{"country":"UK","reimbursement":"Not available on the NHS; trial access via NHS-Galleri","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2018-05","type":"designation","region":"US","note":"FDA Breakthrough Device designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-06","type":"filing","region":"US","note":"Launched as a laboratory-developed test","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-01-29","type":"filing","region":"US","note":"Final PMA module submitted","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-09-23","type":"advisory-committee","region":"US","note":"FDA Molecular and Clinical Genetics Panel meeting","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"ga68-dotatate","kind":"drug","name":"Gallium-68 DOTATATE (and Cu-64 DOTATATE)","aka":[],"tldr":"The PET scan for neuroendocrine tumours that finds far more disease than older scans and confirms eligibility for lutetium radioligand therapy (the theranostic pair).","summary":"Ga-68 DOTATATE (Netspot, kit) approved June 2016; Cu-64 DOTATATE (Detectnet, 12.7-hour half-life allows central production) approved September 2020; Ga-68 DOTATOC (2019, academic). Changes management in about a third of patients; required before Lu-177 DOTATATE (Lutathera). Also images meningioma and paraganglioma.","status":"approved","asOf":"2026-09-08","links":[{"label":"Label: Netspot (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Netspot"},{"label":"Label: Detectnet (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Detectnet"}],"tags":["gap-fill","diagnostic"],"related":[],"cancers":["neuroendocrine","small-intestinal-net","pancreatic-net","lung-net"],"sections":[],"technologies":["sstr-pet","pet-ct","prrt","radioligand-therapy"],"targets":["sstr2"],"drugs":[],"companies":["novartis","curium"],"institutions":[],"pathways":[],"terms":["theranostics","prrt-term"],"trials":["nct04847505","nct05880394"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Netspot / Detectnet","modality":"PET radiotracer (somatostatin receptor ligand)","mechanism":"Radiolabelled somatostatin analogue binding SSTR2 on neuroendocrine tumours; PET replaces the older In-111 octreotide SPECT with higher sensitivity and same-day imaging.","approvals":[{"region":"US","year":2016,"indication":"PET localisation of SSTR-positive neuroendocrine tumours (Ga-68 DOTATATE)"},{"region":"US","year":2020,"indication":"Same (Cu-64 DOTATATE)"},{"region":"EU","year":2017,"indication":"SomaKit TOC (Ga-68 DOTATOC)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ga68-psma-11","kind":"drug","name":"Gallium-68 gozetotide (PSMA-11)","aka":[],"tldr":"Gallium-68 PSMA-11 was the first PSMA PET tracer approved in the US (2020), and is made on site from a generator or cyclotron.","summary":"Gallium-68 gozetotide (PSMA-11) is a 68Ga-labelled urea-based ligand that binds prostate-specific membrane antigen, lighting up prostate cancer on PET wherever it has spread. It is made on site from a germanium-68 generator or cyclotron and used for initial staging of higher-risk disease, for biochemical recurrence, and to confirm PSMA expression before 177Lu-PSMA-617 therapy. The UCLA/UCSF academic NDA (2020) established PSMA PET in the US, followed by Telix's Illuccix kit (2021) and Gozellix (2025, longer shelf-life) and Novartis' Locametz (2022, companion for Pluvicto). It competes with 18F agents (Pylarify, Posluma) that offer central distribution and a longer half-life. Whether findings on PSMA PET should change treatment in the absence of outcome trials remains debated. In plain terms, it was the first PSMA PET tracer approved in the US.","status":"approved","asOf":"2026-09-06","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Gallium-68%20gozetotide"}],"tags":[],"related":[],"cancers":["prostate"],"sections":[],"technologies":["psma-pet"],"targets":["psma"],"drugs":[],"companies":["telix","novartis"],"institutions":["ucla-jonsson","ucsf"],"pathways":[],"terms":[],"trials":["psma-prerp","nct07052214"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Illuccix / Gozellix / Locametz","modality":"PET imaging agent","mechanism":"68Ga-labelled urea-based PSMA ligand.","approvals":[{"region":"US","year":2020,"indication":"PSMA PET (UCLA/UCSF NDA); kits 2021-2022"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"garsorasib","kind":"drug","name":"Garsorasib","aka":[],"tldr":"Garsorasib is InventisBio's KRAS G12C-blocking pill, approved in China in 2024 for previously treated lung cancer with that mutation.","summary":"Approved by the NMPA in August 2024 for KRAS G12C-mutant advanced NSCLC after prior systemic therapy, on a single-arm phase 2 trial that followed the phase 1 study published in the Journal of Thoracic Oncology in 2023. With fulzerasib it made China the second market with domestic KRAS G12C inhibitors; combination studies with EGFR antibodies and SHP2 inhibitors are under way.","status":"approved","asOf":"2026-09-10","links":[{"label":"InventisBio","url":"https://www.inventisbio.com/"},{"label":"D-1553 phase 1 (J Thorac Oncol 2023)","url":"https://doi.org/10.1016/j.jtho.2023.03.015"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["nsclc","kras-g12c-nsclc"],"sections":[],"technologies":["kras-inhibitors","kinase-inhibitors"],"targets":["kras"],"drugs":[],"companies":["inventisbio"],"institutions":[],"pathways":["ras-mapk"],"terms":["kras-mutation-subtypes"],"trials":["nct06300177","nct04585035","nct05379946"],"people":[],"bottlenecks":[],"keyPapers":["paper-li-j-thorac-oncol"],"journals":[],"dependsOn":[],"notes":[],"code":"D-1553","modality":"Small-molecule covalent KRAS G12C inhibitor","mechanism":"Covalent KRAS G12C inhibitor binding the switch II pocket of the GDP-bound mutant protein.","approvals":[{"region":"China","year":2024,"indication":"KRAS G12C-mutant advanced NSCLC after at least one systemic therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gc012f","kind":"drug","name":"GC012F","aka":[],"tldr":"GC012F is an experimental CAR-T cell therapy from Gracell Biotechnologies (Shanghai) in phase 2 trials for multiple myeloma, aimed at CD19 and BCMA.","summary":"GC012F is a CAR-T cell therapy developed by Gracell Biotechnologies (Shanghai). Its targets are CD19 and BCMA (the sponsor names BCMA x CD19). The sponsor states: A dual chimeric antigen receptor (CAR) T-cell product engineered to recognise and attack multiple myeloma cells expressing either BCMA or CD19. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in multiple myeloma. The largest, NCT06235229, plans to enrol 110 participants with primary completion was scheduled for 2026-03-02 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of GC012F","url":"https://clinicaltrials.gov/search?intr=GC012F"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":["cd19","bcma"],"drugs":[],"companies":["gracell-biotechnologies","astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06235229"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"A dual chimeric antigen receptor (CAR) T-cell product engineered to recognise and attack multiple myeloma cells expressing either BCMA or CD19.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gc101-til","kind":"drug","name":"GC101 TIL","aka":[],"tldr":"GC101 TIL is an experimental tumour-infiltrating lymphocyte therapy from Shanghai Juncell Therapeutics in phase 2 trials for melanoma, aimed at PD-1 and BRAF.","summary":"GC101 TIL (GC101) is a tumour-infiltrating lymphocyte therapy developed by Shanghai Juncell Therapeutics. Its targets are PD-1, BRAF and MEK1/2 (the sponsor names melanoma and non-small cell lung cancer (solid tumours)). The sponsor states: An autologous natural TIL cell therapy administered without high-intensity lymphodepleting chemotherapy and without IL-2 administration. ClinicalTrials.gov describes the intervention as: Patients with advanced melanoma (excluding uveal melanoma) who have failed to PD-1 antibodies (if BRAF mutation is present, BRAF and MEK inhibitors have failed; if NRAS mutation is present, tunlametinib have failed) and are ineligible for r. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in melanoma. The largest, NCT06703398, plans to enrol 98 participants with primary completion was scheduled for 2026-05-12 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of GC101 TIL","url":"https://clinicaltrials.gov/search?intr=GC101"},{"label":"Sponsor page","url":"https://www.juncell.com/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":[],"targets":["pd1","braf","mek"],"drugs":[],"companies":["shanghai-juncell-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06703398","nct07560111"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"GC101","modality":"TIL therapy","mechanism":"An autologous natural TIL cell therapy administered without high-intensity lymphodepleting chemotherapy and without IL-2 administration.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gecacitinib","kind":"drug","name":"Gecacitinib","aka":[],"tldr":"Gecacitinib is an oral kinase inhibitor from Suzhou Zelgen Biopharmaceuticals Co.,Ltd, in registered phase 2 trials for cervical cancer.","summary":"Gecacitinib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Suzhou Zelgen Biopharmaceuticals Co.,Ltd, in cervical cancer. A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Gecacitinib","url":"https://clinicaltrials.gov/search?intr=Gecacitinib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["cervical"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["zelgen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07080216","nct06903377","nct06883526"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral kinase inhibitor","mechanism":"A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gedatolisib","kind":"drug","name":"Gedatolisib","aka":[],"tldr":"An intravenous drug that blocks the whole PI3K/mTOR pathway, approved in July 2026 for hormone-positive breast cancer.","summary":"Gedatolisib is an intravenous pan-class I PI3K and mTORC1/2 inhibitor, given at 180 mg weekly for 3 weeks of every 4 alongside palbociclib and fulvestrant, so it shuts down the whole PI3K/AKT/mTOR axis rather than one isoform. Celcuity's VIKTORIA-1 trial tested it after progression on a CDK4/6 inhibitor in PIK3CA-wild-type HR-positive, HER2-negative advanced breast cancer and supported FDA approval in July 2026, listed among that month's oncology approvals. Its notable feature is activity in PIK3CA-wild-type disease, a group without a targeted PI3K-pathway option until now. Stomatitis, neutropenia, hyperglycaemia and rash are the main adverse events. Results in the PIK3CA-mutant cohort, and whether weekly infusions are acceptable against oral alternatives, are open. For a newcomer: a pathway-wide PI3K/mTOR blocker for hormone-driven breast cancer after CDK4/6 therapy stops working.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Gedatolisib"}],"tags":[],"related":[],"cancers":["breast-hr-positive","hr-positive-metastatic-post-cdk46"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["pik3ca","akt"],"drugs":[],"companies":["celcuity"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06190899","nct05501886","nct06757634"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Revtorpyk","modality":"Small-molecule pan-PI3K/mTOR inhibitor","mechanism":"Pan-class I PI3K and mTORC1/2 inhibitor, intravenous weekly.","approvals":[{"region":"US","year":2026,"indication":"HR+/HER2- advanced breast cancer after CDK4/6 inhibitor (July 2026)"}],"mechanismSteps":["Intravenous drug distributes to tumour","Binds all class I PI3K isoforms and mTORC1/2","Pathway output is suppressed regardless of PIK3CA mutation","With CDK4/6 and ER blockade, resistant tumours regress"],"dosing":{"route":"IV infusion","schedule":"180 mg weekly, 3 weeks on 1 week off, with palbociclib and fulvestrant","modifications":"Hold for grade 3 stomatitis or hyperglycaemia","monitoring":"Glucose, oral mucosa, blood counts","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Stomatitis"},{"event":"Neutropenia"},{"event":"Hyperglycaemia"},{"event":"Fatigue"},{"event":"Rash"},{"event":"Nausea"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2025-05","type":"designation","region":"US","note":"Breakthrough Therapy designation, PIK3CA-wild-type HR+/HER2- breast cancer after CDK4/6","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-07-14","type":"approval","region":"US","note":"HR+/HER2- advanced breast cancer after CDK4/6 inhibitor with palbociclib and fulvestrant (VIKTORIA-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-gedatolisib-fulvestrant-or-without-palbociclib-hr-positive-her2-negative-locally"}]},{"id":"gefitinib","kind":"drug","name":"Gefitinib","aka":[],"tldr":"The lung-cancer pill whose dramatic responses in a few patients led to the discovery of EGFR mutations in 2004.","summary":"Gefitinib is a reversible first-generation EGFR tyrosine kinase inhibitor selective for sensitising mutations. It received accelerated US approval in 2003 in unselected NSCLC after chemotherapy, but the ISEL trial (2005) failed to show a survival benefit in that population and US use was restricted. The dramatic responses seen in a minority of patients led Lynch and Paez in 2004 to discover activating EGFR mutations in responders, one of the founding discoveries of precision oncology. IPASS (NEJM 2009) then proved that mutation-selected first-line gefitinib was superior to chemotherapy, and the EU approved it for EGFR-mutant NSCLC in 2009; the US re-approved it in 2015 for first-line exon 19 deletion or L858R disease. Rash, diarrhoea and rare interstitial lung disease are the main toxicities. Gefitinib's story is the reason EGFR testing is now routine at diagnosis.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Gefitinib","links":[{"label":"IPASS (NEJM 2009)","url":"https://doi.org/10.1056/NEJMoa0810699"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=gefitinib"}],"tags":["gap-fill","generic"],"related":["egfr-exon-19-deletion"],"cancers":["nsclc","egfr-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":["egfr-exon19-l858r","oncogene-addiction"],"trials":["gefitinib-chemo-tmh","nct02411448","nct03944772","nct03040973"],"people":[],"bottlenecks":[],"keyPapers":["paper-mok-ipass-gefitinib-pulmonary-adenocarcinoma-nejm-2009"],"journals":[],"dependsOn":[],"notes":[],"brand":"Iressa","modality":"Small-molecule EGFR TKI (first generation, reversible)","mechanism":"Reversible EGFR kinase inhibitor selective for sensitising mutations.","approvals":[{"region":"US","year":2003,"indication":"NSCLC after chemotherapy (accelerated; restricted 2005)"},{"region":"EU","year":2009,"indication":"EGFR-mutant NSCLC"},{"region":"US","year":2015,"indication":"First-line NSCLC with EGFR exon 19 del or L858R"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2003-05-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"As monotherapy for locally advanced or metastatic NSCLC after failure of platinum-based and docetaxel chemotherapy"},{"date":"2012-04-25","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 9.0 years after its accelerated approval.","indication":"As monotherapy for locally advanced or metastatic NSCLC after failure of platinum-based and docetaxel chemotherapy"}]},{"id":"gemcitabine","kind":"drug","name":"Gemcitabine","aka":[],"tldr":"A versatile chemotherapy used in pancreatic, bladder, lung, ovarian, breast and biliary cancers, in nasopharyngeal cancer, and as a bladder instillation.","summary":"Approved 1996 for pancreatic cancer (clinical benefit response versus 5-FU); combinations: gemcitabine-cisplatin (biliary ABC-02, bladder, NPC induction), gemcitabine-nab-paclitaxel (pancreatic), gemcitabine-carboplatin (ovarian, breast), gemcitabine-docetaxel (sarcoma), GemOx (lymphoma). Intravesical gemcitabine for NMIBC. Flu-like symptoms, myelosuppression, rare TMA/pneumonitis.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Gemcitabine","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=gemcitabine"},{"label":"NICE TA25: gemcitabine for pancreatic cancer (2001, replaced by NG85)","url":"https://www.nice.org.uk/guidance/ta25"},{"label":"NICE NG85 recommendations 1.8.7 and 1.9.6","url":"https://www.nice.org.uk/guidance/ng85/chapter/Recommendations"}],"tags":["gap-fill","generic","who-essential"],"related":[],"cancers":["pancreatic","urothelial","nsclc","ovarian","cholangiocarcinoma","nasopharyngeal","sarcoma"],"sections":[],"technologies":["cytotoxic-chemotherapy","bcg-and-intravesical-therapy"],"targets":[],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":["dna-replication-licensing"],"terms":[],"trials":["notable-trial","nct06828354","nct04442022","nct04384484","nct06435429","nct07491445","nct07805954","nct06508658","nct06459180","nct03800134","nct07621718","nct06976190","nct04969731","nct06312137","nct07604766","nct06118333","nct03164616","nct06619236","nct02486718","nct04974398","nct03178552","nct07564141","nct06445062","nct06710288","nct06601504","nct05482893","nct05775159","nct04083599","nct06646055","nct07049055","nct06219941","nct06922591","nct07124936","nct03775486","nct07255404","nct05431270","nct07483554","nct05669482","nct04657068","nct04844866","nct07563738","actuate-1801","nct07020221","nct07259590","nct06843447","nct04198766","nct07223047","nct06081959","nct05229497","nct07701486","nct06623422","nct06361888","nct05302284","nct06908304","nct06465563","nct03257033","nct06953999","nct05814354","nct07229586","nct03816163","nct03579836","nct07710885","nct05771480","nct05999994","nct06343948","nct07129993","nct03682068","nct07280377","nct04589234","nct07575191","nct06319820","nct06211764","nct06467357","nct06449222","nct07566156","nct05827796","nct03036098","nct02516241","nct05934331","nct07259317","nct05548296","nct02453282","nct06960577","nct06545955","nct05653453","nct07173751","nct06821503","nct07007559","nct04241185","nct06592326","nct02542293","nct03478488","nct04999969","nct06957886","nct06806033","nct06752811","nct03003962","nct03967977","nct07111546","nct05911295","nct06854445","nct06225596","nct04274426","nct04390399","nct07393542","nct06632951","nct07398339","nct06788912","nct06522737","nct07238283","nct06591520","nct06241781","nct02340117","nct06493552","nct07106762","nct07444541","nct04639986","nct05859750","nct07286266","nct06841354","nct07296341","nct06663137","nct06460298","nct01954992","nct05355298","prodige-4-accord-11","mpact","jaspac-01","apact","conko-005","conko-007","preopanc-2","scalop","panoptimox-prodige-35"],"people":[],"bottlenecks":[],"keyPapers":["paper-burris-gemcitabine-pancreatic-jco-1997"],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: gemcitabine was the comparator beaten by FOLFIRINOX (PRODIGE 4), nab-paclitaxel plus gemcitabine (MPACT), adjuvant modified FOLFIRINOX (PRODIGE 24), gemcitabine plus capecitabine (ESPAC-4) and S-1 (JASPAC 01), and the drug proven against observation after surgery in CONKO-001. It remains the backbone for less fit patients, the radiosensitiser alternative to capecitabine (SCALOP) and part of the PREOPANC chemoradiotherapy regimen."],"brand":"Gemzar / Infugem","modality":"Nucleoside analogue (deoxycytidine)","mechanism":"Difluorodeoxycytidine triphosphate incorporated into DNA causes masked chain termination; also inhibits ribonucleotide reductase (self-potentiation).","approvals":[{"region":"US","year":1996,"indication":"Pancreatic cancer"},{"region":"US","year":1998,"indication":"NSCLC with cisplatin"},{"region":"US","year":2004,"indication":"Metastatic breast cancer with paclitaxel"},{"region":"US","year":2006,"indication":"Ovarian cancer with carboplatin"},{"region":"UK","year":2001,"indication":"Pancreatic cancer: NICE TA25 (8 May 2001), since replaced by the NG85 guideline, which recommends gemcitabine alone for people not well enough for combination chemotherapy (1.9.6) and as adjuvant treatment for those who cannot tolerate gemcitabine plus capecitabine (1.8.7)","note":"https://www.nice.org.uk/guidance/ta25"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gemcitabine-cisplatin","kind":"drug","name":"Gemcitabine + cisplatin","aka":[],"tldr":"Gemcitabine plus cisplatin has been the chemotherapy backbone for bile duct cancer since 2010 and is now given with immunotherapy.","summary":"Gemcitabine, a nucleoside analogue that terminates DNA chains, is combined with cisplatin, which crosslinks DNA, giving two complementary attacks on dividing cells. The doublet has been the chemotherapy backbone for advanced biliary tract cancer since ABC-02 (2010) showed overall survival of 11.7 versus 8.1 months against gemcitabine alone (HR 0.64), and it is given on days 1 and 8 of a 3-week cycle for up to 8 cycles. It is now the backbone of TOPAZ-1 with durvalumab (OS HR 0.76, 24-month OS 23.6% versus 11.5%) and KEYNOTE-966 with pembrolizumab (OS 12.7 versus 10.9 months), so most patients receive it with immunotherapy. Adding nab-paclitaxel (SWOG 1815) did not improve survival. Grade 3 to 4 neutropenia (25%) and fatigue are the main toxicities, and renal function and hearing need monitoring. For a newcomer, this is the standard two-drug chemotherapy for bile duct cancer.","status":"standard-of-care","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Gemcitabine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Gemcitabine"}],"tags":[],"related":[],"cancers":["cholangiocarcinoma","cup-unfavourable","locoregionally-advanced-nasopharyngeal-carcinoma","recurrent-metastatic-nasopharyngeal-carcinoma","gallbladder","biliary-tract-cancer"],"sections":[],"technologies":["cytotoxic-chemotherapy","platinum"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["abc-02","topaz-1","keynote-966","nct05771480","nct06109779","nct02453282","nct02542293","nct03003962","nct06225596","nct07221253","nct06591520","acticca-1","swog-s1815","opt-in","polcagb","gain-igbc","rugb","debate","eortc-1607-pembro-cisgem","abc-07","abc-12","ea2197-ivonescimab","trap-btc","gap-phase2-mdacc","nife"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Gallbladder cancer: the doublet is the backbone for gallbladder cancer as for cholangiocarcinoma. In TOPAZ-1 25 percent of patients had gallbladder cancer (Imfinzi label), in SWOG S1815 16 percent, and in the 60-patient phase 2 that preceded S1815 22 percent; the adjuvant ACTICCA-1 trial randomised a separate muscle-invasive gallbladder cohort to 24 weeks of the doublet, and the perioperative OPT-IN, GAIN and POLCAGB trials use it before radical surgery."],"modality":"Cytotoxic regimen","mechanism":"Nucleoside analogue (gemcitabine) plus DNA crosslinker (cisplatin).","approvals":[],"mechanismSteps":[],"dosing":{"route":"Intravenous","schedule":"Gemcitabine 1000 mg/m² + cisplatin 25 mg/m² days 1 and 8 every 3 weeks, up to 8 cycles","monitoring":"Renal function, hearing, blood counts"},"toxicity":[{"event":"Neutropenia (grade 3-4)","grade3PlusPct":25,"note":"ABC-02"},{"event":"Fatigue","grade3PlusPct":19,"note":"ABC-02"}],"access":[],"regulatoryEvents":[]},{"id":"gemcitabine-nab-paclitaxel","kind":"drug","name":"Gemcitabine + nab-paclitaxel","aka":[],"tldr":"Gemcitabine plus nab-paclitaxel is the gentler of the two standard chemotherapy backbones for pancreatic cancer, and the base on which most new drugs are being tested.","summary":"MPACT (2013): OS 8.5 vs 6.7 months versus gemcitabine alone. Preferred for less fit patients; the control or backbone arm in PANOVA-3 (TTFields), zoldonrasib first-line combinations, and CLDN18.2 and other add-on trials. Gemcitabine monotherapy (1997) was the standard for 14 years before FOLFIRINOX.","status":"standard-of-care","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Gemcitabine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Gemcitabine"},{"label":"NICE TA476 (6 September 2017)","url":"https://www.nice.org.uk/guidance/ta476"}],"tags":[],"related":[],"cancers":["pancreatic","locally-advanced-pdac","metastatic-pdac"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":["paclitaxel"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["panova-3","nct05257993","nct04589234","mpact","apact","canstem111p","halo-301","resolve","neolap","neonax","swog-s1505","scalop-2","starpac2","primus-001"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer, UK status: NICE TA476 (6 September 2017) recommends the combination for untreated metastatic disease only if other combination chemotherapies are unsuitable and the person would otherwise have gemcitabine alone; NICE NG85 1.9.5 repeats this. Adjuvant use missed its primary endpoint in APACT and is not recommended by NICE or NCCN. It is the control arm of PANOVA-3, NAPOLI 3, CanStem111P (median 11.7 months), HALO-301, RESOLVE and RASolute 303."],"brand":"Gemzar + Abraxane","modality":"Cytotoxic regimen","mechanism":"Nucleoside analogue (gemcitabine) plus albumin-bound taxane that may deplete stroma and increase gemcitabine delivery.","approvals":[{"region":"US","year":2013,"indication":"First-line metastatic pancreatic adenocarcinoma (nab-paclitaxel label)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tar-200","kind":"drug","name":"Gemcitabine intravesical system (TAR-200)","aka":[],"tldr":"TAR-200 is a small pretzel-shaped device placed in the bladder that releases gemcitabine for three weeks at a time. It was approved in 2025 for early bladder cancer that no longer responds to BCG.","summary":"TAR-200 is a small pretzel-shaped osmotic mini-tablet system placed in the bladder via a urinary catheter in the clinic; it releases gemcitabine continuously into urine, giving sustained local exposure without systemic levels. The FDA approved it on 9 September 2025 for BCG-unresponsive NMIBC with carcinoma in situ on the basis of SunRISe-1, where monotherapy produced a complete response in 82% of patients and 51% remained in CR at 1 year. Urinary tract infection and dysuria were the common side effects, with grade 3 or higher treatment-related events in 12%. Phase 3 trials cover BCG-naive NMIBC (SunRISe-3) and muscle-invasive disease (SunRISe-4 with cetrelimab, SunRISe-2 versus chemoradiation); Johnson & Johnson develops it after acquiring Taris. It offers slow-release chemotherapy from inside the bladder as an alternative to bladder removal when BCG fails.","status":"approved","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT04640623 (SunRISe-1)","url":"https://clinicaltrials.gov/study/NCT04640623"}],"tags":[],"related":[],"cancers":["urothelial","non-muscle-invasive-bladder-cancer"],"sections":[],"technologies":["bcg-and-intravesical-therapy"],"targets":[],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":["bcg-unresponsive"],"trials":["sunrise-1","nct06211764"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Inlexzo","code":"TAR-200","modality":"Intravesical drug-eluting device","mechanism":"Osmotic mini-tablet system continuously releases gemcitabine into urine, achieving sustained local exposure without systemic levels.","approvals":[{"region":"US","year":2025,"indication":"BCG-unresponsive high-risk NMIBC with CIS, with or without papillary tumours"}],"mechanismSteps":["Device inserted through a urinary placement catheter and coils in the bladder","Osmotic pump releases gemcitabine continuously for ~3 weeks","Sustained urothelial drug exposure kills CIS and papillary cells","Device removed cystoscopically; repeated every 3 weeks then every 12 weeks"],"dosing":{"route":"Intravesical (indwelling device)","schedule":"225 mg gemcitabine device every 3 weeks for 24 weeks, then every 12 weeks up to 3 years","monitoring":"Cystoscopy and cytology at 3-month intervals"},"toxicity":[{"event":"Urinary tract infection","anyGradePct":24},{"event":"Dysuria","anyGradePct":22},{"event":"Pollakiuria","anyGradePct":20},{"event":"Grade 3+ treatment-related","grade3PlusPct":12}],"access":[],"regulatoryEvents":[{"date":"2025-09-09","type":"approval","region":"US","note":"FDA approval of Inlexzo","source":"https://www.onclive.com/view/fda-approves-tar-200-in-bcg-unresponsive-nmibc-with-cis"}]},{"id":"gemtuzumab-ozogamicin","kind":"drug","name":"Gemtuzumab ozogamicin","aka":[],"tldr":"Gemtuzumab ozogamicin (Mylotarg) is an anti-CD33 antibody linked to the DNA-cutting payload calicheamicin, the first ADC approved, in 2000 for relapsed acute myeloid leukaemia. An unstable linker and no benefit in a confirmatory trial led to withdrawal in 2010; it returned in 2017 at a lower fractionated dose, with liver toxicity, including veno-occlusive disease, its defining risk.","summary":"Gemtuzumab ozogamicin (Mylotarg) is a humanised anti-CD33 IgG4 antibody linked through an acid-labile hydrazone to calicheamicin, a DNA cleaver that causes double-strand breaks, at a drug-to-antibody ratio of about 2 to 3. It was the very first ADC, approved in 2000 for relapsed CD33-positive AML, but this first-generation design had an unstable linker and heterogeneous conjugation, and it was withdrawn in 2010 after a confirmatory trial showed no benefit and excess deaths. Re-approval in 2017 was based on ALFA-0701, in which fractionated dosing (3 mg/m² on days 1, 4 and 7 with induction chemotherapy) improved event-free survival in CD33-positive AML. Hepatotoxicity including veno-occlusive disease, haemorrhage and infection are the key risks, so liver tests are checked before each dose. Its history shows that schedule and linker chemistry can decide the fate of a sound target.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Gemtuzumab_ozogamicin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Gemtuzumab%20ozogamicin"}],"tags":[],"related":["cd33-expression"],"cancers":["aml","aml-paediatric"],"sections":[],"technologies":["adc"],"targets":["cd33"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":["hydrazone"],"trials":["aaml0531"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mylotarg","modality":"ADC","payload":"Calicheamicin (DNA cleaver), DAR ~2-3","linker":"Acid-labile hydrazone","mechanism":"Anti-CD33 humanised IgG4 with calicheamicin; DNA double-strand cleavage.","approvals":[{"region":"US","year":2000,"indication":"Relapsed CD33+ AML (withdrawn 2010)"},{"region":"US","year":2017,"indication":"Newly diagnosed and relapsed CD33+ AML"}],"mechanismSteps":["Antibody binds CD33 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","Calicheamicin is released inside the cell","DNA is damaged (crosslinks or double-strand breaks)","Tumour cell dies by apoptosis"],"dosing":{"route":"IV infusion","schedule":"Newly diagnosed AML: 3 mg/m² (max 4.5 mg) on days 1, 4, 7 of induction with daunorubicin/cytarabine; relapsed: 3 mg/m² days 1, 4, 7 as single agent","modifications":"Hold for hepatotoxicity; veno-occlusive disease risk, especially around transplant","monitoring":"LFTs before each dose, signs of VOD, blood counts","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Haemorrhage"},{"event":"Infection"},{"event":"Hepatotoxicity including veno-occlusive disease"},{"event":"Thrombocytopenia"},{"event":"Infusion reactions"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2000-05-17","type":"accelerated-approval","region":"US","note":"Accelerated approval, relapsed CD33+ AML in older patients; first ADC ever approved","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"For patients with CD33+ AML in first relapse 60 years of age or older and not candidates for cytotoxic chemotherapy"},{"date":"2010-06-21","type":"withdrawal","region":"US","note":"Voluntary withdrawal after confirmatory trial showed no benefit and excess deaths","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2011-11-28","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 11.5 years after its accelerated approval.","indication":"For patients with CD33+ AML in first relapse 60 years of age or older and not candidates for cytotoxic chemotherapy"},{"date":"2017-09-01","type":"approval","region":"US","note":"Re-approval at lower fractionated dose for newly diagnosed and relapsed CD33+ AML (ALFA-0701)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"gfh375","kind":"drug","name":"GFH375","aka":[],"tldr":"GFH375 is an experimental small-molecule drug from Genfleet Therapeutics (Shanghai) in phase 3 trials for pancreatic ductal adenocarcinoma and non-small-cell lung cancer, aimed at KRAS.","summary":"GFH375 is a small-molecule drug developed by Genfleet Therapeutics (Shanghai). Its target is KRAS (the sponsor names KRAS G12D). The sponsor states: GFH375 is an oral, highly selective, non-covalent small-molecule inhibitor targeting the KRAS G12D mutation, blocking KRAS-driven signalling and cancer cell growth. ClinicalTrials.gov describes the intervention as: The experimental group receives GFH375 monotherapy, QD, orally. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07262567 (Phase III Study to Compare GFH375 and Chemotherapy in Patients With KRAS G12D-Mutant Metastatic Pancreatic Cancer) and NCT07668752 (A Study to Evaluate GFH375 Versus Docetaxel in Participants With Non-Small Cell Lung Cancer With KRAS G12D Mutation), in pancreatic ductal adenocarcinoma and non-small-cell lung cancer. The largest, NCT06500676, plans to enrol 407 participants with primary completion expected 2028-07-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of GFH375","url":"https://clinicaltrials.gov/search?intr=GFH375"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic","nsclc"],"sections":[],"technologies":[],"targets":["kras"],"drugs":[],"companies":["genfleet"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07262567","nct07668752","nct07259590","nct06500676"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"GFH375 is an oral, highly selective, non-covalent small-molecule inhibitor targeting the KRAS G12D mutation, blocking KRAS-driven signalling and cancer cell growth.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gi-6301","kind":"drug","name":"GI-6301","aka":["Yeast-brachyury vaccine"],"tldr":"GI-6301 was a vaccine made from yeast carrying brachyury, the protein that chordomas depend on. Given with radiotherapy in a randomised trial it did not improve tumour responses.","summary":"GI-6301 was GlobeImmune's Tarmogen vaccine against brachyury, the notochord transcription factor whose expression defines chordoma and which also drives epithelial-to-mesenchymal transition in other cancers. Phase 1 testing at the National Cancer Institute showed brachyury-specific T-cell responses and some disease stabilisation in chordoma. A randomised, placebo-controlled phase 2 trial combined the vaccine with radiotherapy in locally advanced chordoma; it found no difference in objective response between the arms, although the vaccine was safe.\n\nThe chordoma page names it with adenoviral brachyury vaccines among the attempts to immunise against brachyury, alongside newer brachyury degraders and CDK7/9 inhibitors that lower its expression.","status":"negative","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":[],"cancers":["chordoma"],"sections":[],"technologies":["shared-antigen-vaccine"],"targets":[],"drugs":[],"companies":["globeimmune"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"GI-6301","modality":"Heat-killed recombinant yeast vaccine expressing brachyury","mechanism":"Saccharomyces cerevisiae engineered to express the transcription factor brachyury (TBXT), the driver of chordoma, is injected to raise brachyury-specific T cells; the yeast itself acts as the adjuvant.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gilteritinib","kind":"drug","name":"Gilteritinib","aka":[],"tldr":"A single pill that beats salvage chemotherapy for relapsed FLT3-mutated AML, and the backbone of the triplets now being tested in frontline disease.","summary":"ADMIRAL (n=371): gilteritinib vs salvage chemotherapy in relapsed/refractory FLT3-mutated AML, OS 9.3 vs 5.6 months (HR 0.64), CR/CRh 34%. Approved 2018. Post-transplant maintenance (MORPHO) reduced relapse in MRD-positive patients. Frontline triplets (gilteritinib + azacitidine + venetoclax) show high CR rates in unfit patients; the phase 3 vs midostaurin (HOVON 156 AML) reported non-superiority for EFS in 2025-26 data, keeping midostaurin/quizartinib as intensive standards.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Gilteritinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Gilteritinib"}],"tags":[],"related":["flt3-itd","flt3-tkd"],"cancers":["aml","aml-flt3"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["flt3"],"drugs":[],"companies":["astellas"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome"],"trials":["admiral","nct04988555","nct05520567","nct03182244"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Xospata","modality":"Small-molecule kinase inhibitor (FLT3, type I)","mechanism":"Type I FLT3/AXL inhibitor active against ITD and TKD (D835) mutations.","approvals":[{"region":"US","year":2018,"indication":"Relapsed or refractory FLT3-mutated AML"}],"mechanismSteps":["Gilteritinib binds active FLT3 regardless of ITD or TKD mutation","AXL, a bypass kinase, is co-inhibited","STAT5/MAPK/PI3K signalling collapses in blasts","Oral daily dosing continues until progression or as post-transplant maintenance"],"dosing":{"route":"Oral","schedule":"120 mg once daily until progression or unacceptable toxicity (minimum 6 months to allow response)","monitoring":"Differentiation syndrome (boxed warning), QT, LFTs, PRES, pancreatitis","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Xospata"},"toxicity":[{"event":"Differentiation syndrome","anyGradePct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Xospata","note":"Boxed warning"},{"event":"Transaminase elevation","anyGradePct":44},{"event":"Myalgia/arthralgia","anyGradePct":42},{"event":"Febrile neutropenia","grade3PlusPct":46}],"access":[],"regulatoryEvents":[{"date":"2018-11-28","type":"approval","region":"US","note":"ADMIRAL interim; full OS data 2019"}]},{"id":"giredestrant","kind":"drug","name":"Giredestrant","aka":[],"tldr":"Giredestrant is Roche's oral SERD: it failed to beat an aromatase inhibitor in first-line metastatic disease but succeeded after surgery and after CDK4/6 failure.","summary":"evERA (with everolimus after CDK4/6): PFS HR 0.56, HR 0.38 in ESR1-mutant; NDA accepted February 2026. lidERA (adjuvant): ~30% fewer invasive recurrences than standard endocrine therapy (SABCS 2025). persevERA (first-line with palbociclib): missed its primary endpoint (March 2026). Together these define where oral SERDs add value: resistance and adjuvant settings rather than upfront in unselected metastatic disease.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT05306340 (evERA)","url":"https://clinicaltrials.gov/study/NCT05306340"}],"tags":[],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["oral-serd"],"trials":["evera","lidera","persevera","nct07100106","nct06065748","nct05296798","nct07541079","nct04576455","nct07054190"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"GDC-9545, RG6171","modality":"Oral SERD","mechanism":"Oral SERD with full ER antagonism and degradation.","approvals":[],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"30 mg once daily (trial dose)"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-02-19","type":"filing","region":"US","note":"NDA accepted (evERA, ESR1-mutant)","source":"https://www.gene.com/media/press-releases/15100/2026-02-19/fda-accepts-new-drug-application-for-gen"},{"date":"2026-03-09","type":"designation","region":"Global","note":"persevERA primary endpoint not met","source":"https://www.roche.com/media/releases/med-cor-2026-03-09"}]},{"id":"givinostat","kind":"drug","name":"Givinostat","aka":["givinostat (ITF2357)"],"tldr":"Givinostat is an experimental small-molecule drug from Italfarmaco in phase 3 trials for myeloproliferative neoplasms, with its target not yet stated publicly.","summary":"Givinostat (ITF2357) is a small-molecule drug developed by Italfarmaco. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Patients will continue at their last tolerable dose and treatment schedule of givinostat monotherapy. Givinostat is a histone-deacetylases inhibitor. The product will be supplied as hard gelatine capsules for oral administration at the stre. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06093672 (Study on Efficacy and Safety of Givinostat Versus Hydroxyurea in Patients With Polycythemia Vera), in myeloproliferative neoplasms. The largest, NCT06093672, plans to enrol 220 participants with primary completion was scheduled for 2026-07 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Givinostat","url":"https://clinicaltrials.gov/search?intr=ITF2357"},{"label":"Sponsor page","url":"https://www.italfarmaco.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["myeloproliferative-neoplasms","polycythaemia-vera"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["italfarmaco"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06093672","nct01761968"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ITF2357","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"glasdegib","kind":"drug","name":"Glasdegib","aka":[],"tldr":"Glasdegib is a hedgehog-pathway pill that, with low-dose chemotherapy, extends survival in older AML patients who cannot have intensive treatment; it has largely been displaced by venetoclax combinations.","summary":"Glasdegib inhibits smoothened (SMO), reducing hedgehog signalling in leukaemic stem cells, and is combined with low-dose cytarabine for newly diagnosed AML in adults aged 75 or over or unfit for intensive chemotherapy. In BRIGHT AML 1003 the combination gave a median overall survival of 8.8 versus 4.9 months with low-dose cytarabine alone, leading to US approval in 2018 and EU approval in 2020. The BRIGHT AML 1019 trials, which added glasdegib to intensive chemotherapy or azacitidine backbones, were negative. In the same period venetoclax with azacitidine became the preferred regimen for unfit AML, so glasdegib is now rarely chosen. Muscle spasms, dysgeusia and alopecia are class hedgehog-inhibitor effects. For a newcomer, glasdegib proved that a hedgehog pill could extend survival in older AML but was overtaken by a better combination.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Glasdegib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=glasdegib"}],"tags":["gap-fill"],"related":[],"cancers":["aml","aml-older-unfit"],"sections":[],"technologies":["kinase-inhibitors","hedgehog-inhibitors"],"targets":["smoothened"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03416179","nct04842604","nct04093505"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Daurismo","modality":"Small-molecule smoothened (hedgehog) inhibitor","mechanism":"Inhibits SMO, reducing hedgehog signalling in leukaemic stem cells; combined with low-dose cytarabine.","approvals":[{"region":"US","year":2018,"indication":"Newly diagnosed AML in adults ≥75 or unfit for intensive chemotherapy, with low-dose cytarabine"},{"region":"EU","year":2020,"indication":"Same"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"glecirasib","kind":"drug","name":"Glecirasib","aka":[],"tldr":"Glecirasib is Jacobio's KRAS G12C inhibitor, approved in China in 2024 for previously treated non-small cell lung cancer with that mutation, and in a phase 3 trial against docetaxel.","summary":"Jacobio Pharma developed glecirasib (JAB-21822) and partnered it with Allist Pharmaceuticals for China. The NMPA approved it in 2024 for KRAS G12C-mutant non-small cell lung cancer after at least one prior systemic therapy, on a single-arm phase 2 trial with a response rate in the range of the approved Western inhibitors and a low rate of gastrointestinal side effects. A randomised phase 3 trial against docetaxel and combination studies with a SHP2 inhibitor are registered.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Glecirasib","url":"https://clinicaltrials.gov/search?intr=JAB-21822"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","kras-g12c-nsclc"],"sections":[],"technologies":["kras-inhibitors","kinase-inhibitors"],"targets":["kras"],"drugs":[],"companies":["jacobio-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06416410","nct05194995","nct05288205","nct07164170","nct05276726","nct05009329","nct06008288"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"JAB-21822","modality":"Oral covalent KRAS G12C inhibitor","mechanism":"Binds the mutant cysteine of KRAS G12C and locks the protein in its inactive state, the same mechanism as sotorasib and adagrasib.","approvals":[{"region":"CN","year":2024,"indication":"KRAS G12C-mutant advanced non-small cell lung cancer after prior systemic therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"glofitamab","kind":"drug","name":"Glofitamab","aka":[],"tldr":"Glofitamab is a fixed-duration bispecific for large B-cell lymphoma, with OS benefit when combined with chemotherapy.","summary":"Glofitamab is a CD20×CD3 bispecific T-cell engager in a 2:1 format, with two CD20-binding arms and one CD3 arm, that recruits any passing T cell to kill a B cell. A single 1,000 mg dose of obinutuzumab is given first to blunt cytokine release, followed by step-up doses of 2.5 mg and 10 mg, then 30 mg every 3 weeks for up to 12 cycles, so treatment is fixed in duration. Accelerated US approval in 2023 and conditional EU authorisation followed a response rate of 56 percent with complete responses in 43 percent in relapsed or refractory DLBCL after two or more lines. STARGLO then showed an overall survival benefit when glofitamab was added to GemOx in transplant-ineligible relapsed DLBCL; the combination is approved in the EU and elsewhere, but the FDA declined in 2025 citing regional heterogeneity. Cytokine release syndrome, neutropenia and infections are the main toxicities.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Glofitamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Glofitamab"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["t-cell-engager"],"targets":["cd20","cd3"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04077723","nct06090539","nct05533775","nct06534437","nct03075696","nct06084936","nct06634589","nct03533283","nct06806033","nct04980222"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Columvi","modality":"Bispecific T-cell engager (CD20×CD3, 2:1)","mechanism":"2:1 CD20:CD3 format with obinutuzumab pre-treatment to mitigate CRS.","approvals":[{"region":"US","year":2023,"indication":"Relapsed/refractory DLBCL after ≥2 lines (accelerated)"}],"mechanismSteps":["One arm binds CD20 (bivalent, 2:1 format) on the tumour cell","The other arm binds CD3 on any passing T cell","An artificial immune synapse forms, independent of MHC","The T cell releases perforin and granzymes into the tumour cell","Tumour cell dies; cytokines are released (source of CRS)"],"dosing":{"route":"IV infusion","schedule":"Obinutuzumab 1,000 mg on cycle 1 day 1, then glofitamab 2.5 mg day 8, 10 mg day 15, then 30 mg every 3 weeks for up to 12 cycles (fixed duration)","modifications":"Hold for grade ≥2 CRS; dexamethasone premedication","monitoring":"Hospitalisation for the first 2.5 mg dose; CRS, neurology, infections","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Cytokine release syndrome"},{"event":"Neutropenia"},{"event":"Musculoskeletal pain"},{"event":"Rash"},{"event":"Fatigue"},{"event":"Infections"},{"event":"Tumour flare"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.genentech-access.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for relapsed/refractory DLBCL after ≥2 therapies (TA927)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-06-15","type":"accelerated-approval","region":"US","note":"Accelerated approval, relapsed/refractory DLBCL after ≥2 lines The confirmatory requirement was still open 3.3 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Relapsed or refractory diffuse large B-cell lymphoma, not otherwise specified or large B-cell lymphoma arising from follicular lymphoma, after two or more lines of systemic therapy"},{"date":"2023-07","type":"approval","region":"EU","note":"Conditional marketing authorisation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-07-02","type":"crl","region":"US","note":"Complete response letter for STARGLO combination (regional heterogeneity); approved in EU","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"glsi-100","kind":"drug","name":"GLSI-100","aka":[],"tldr":"GLSI-100 is an experimental cancer vaccine from Greenwich LifeSciences in phase 3 trials for HR-positive / HER2-negative breast cancer, aimed at HER2.","summary":"GLSI-100 is a cancer vaccine developed by Greenwich LifeSciences. Its target is HER2 (the sponsor names HER2/neu). The sponsor states: Combines GP2, a nine-amino-acid HER2/neu-derived transmembrane peptide, with the immunoadjuvant GM-CSF to prime and expand HER2/neu-specific CD8+ cytotoxic T lymphocytes that recognise and destroy HER2/neu-expressing cancer cells, aiming to prevent breast cancer recurrence. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05232916 (Phase 3 Study to Evaluate the Efficacy and Safety of HER2/Neu Peptide GLSI-100 (GP2 + GM-CSF) in HER2/Neu Positive Subjects), in HR-positive / HER2-negative breast cancer. The largest, NCT05232916, plans to enrol 2250 participants with primary completion expected 2031-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of GLSI-100","url":"https://clinicaltrials.gov/search?intr=GLSI-100"},{"label":"Sponsor pipeline page","url":"https://greenwichlifesciences.com/gp2/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["greenwich-lifesciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05232916"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"vaccine","mechanism":"Combines GP2, a nine-amino-acid HER2/neu-derived transmembrane peptide, with the immunoadjuvant GM-CSF to prime and expand HER2/neu-specific CD8+ cytotoxic T lymphocytes that recognise and destroy HER2/neu-expressing cancer cells, aiming to prevent breast cancer recurrence.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"glucarpidase","kind":"drug","name":"Glucarpidase","aka":["Carboxypeptidase G2"],"tldr":"Glucarpidase (Voraxaze) is a single emergency injection that chops up methotrexate in the blood when a patient's kidneys have failed to clear a high dose, preventing potentially fatal toxicity.","summary":"Glucarpidase was approved by the FDA in January 2012 to reduce toxic plasma methotrexate concentrations (above 1 micromole per litre) in adults and children with delayed methotrexate clearance due to impaired renal function, on pooled single-arm studies in which a single 50 units/kg dose lowered methotrexate by more than 95% within 15 minutes in almost all patients. It does not replace leucovorin, which must continue (given at least two hours apart because glucarpidase also cleaves leucovorin), and it is not for patients with expected clearance. In the EU it is available through named-patient supply rather than a central authorisation. Marketed by BTG, now SERB.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Glucarpidase","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=glucarpidase"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/glucarpidase"}],"tags":["nci-list","supportive"],"related":["methotrexate"],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["serb-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01305655","nct00634322","nct02022358"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Voraxaze","modality":"Recombinant bacterial enzyme (rescue agent)","supportive":true,"mechanism":"Carboxypeptidase G2 from Pseudomonas that cleaves the terminal glutamate from methotrexate, converting it within minutes to inactive DAMPA and glutamate, independent of renal function.","approvals":[{"region":"US","year":2012,"indication":"Toxic plasma methotrexate concentrations with delayed clearance due to impaired renal function"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"glumetinib","kind":"drug","name":"Glumetinib","aka":["Gumarontinib","Haiyitan"],"tldr":"Glumetinib is a Chinese MET inhibitor approved in 2023 for lung cancers with MET exon 14 skipping, one of three such drugs approved in China and now in a phase 3 trial in gastric cancer.","summary":"Haihe Biopharma's glumetinib (SCC244) was approved by the NMPA in 2023 for locally advanced or metastatic non-small cell lung cancer with MET exon 14 skipping, on the GLORY phase 2 trial. It joins savolitinib and vebreltinib as China's approved MET inhibitors, alongside capmatinib and tepotinib elsewhere. A phase 3 trial in MET-amplified gastric cancer with chemotherapy is registered.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Glumetinib","url":"https://clinicaltrials.gov/search?intr=SCC244"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","gastric","sclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["met"],"drugs":[],"companies":["haihe-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06829459","nct06947291","nct04270591"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"SCC244","modality":"Oral selective MET tyrosine kinase inhibitor","mechanism":"A highly selective type I MET kinase inhibitor for tumours driven by MET exon 14 skipping or amplification.","approvals":[{"region":"CN","year":2023,"indication":"Locally advanced or metastatic non-small cell lung cancer with MET exon 14 skipping"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"golcadomide","kind":"drug","name":"Golcadomide","aka":[],"tldr":"Golcadomide is a next-generation lenalidomide-like pill that degrades two lymphoma transcription factors far more potently, now in phase 3 with R-CHOP.","summary":"Golcadomide is a cereblon E3 ligase modulator (CELMoD): it binds cereblon and recruits the transcription factors Ikaros (IKZF1) and Aiolos (IKZF3) for ubiquitination and proteasomal degradation, which downregulates IRF4 and MYC in lymphoma cells and stimulates T cells, far more potently than lenalidomide. In phase 1/2, golcadomide with rituximab gave an ORR of about 50% in relapsed or refractory DLBCL, and with R-CHOP in untreated high-risk DLBCL it produced high complete response rates (ASH 2024). The phase 3 GOLSEEK-1 trial is enrolling patients with IPI 3 to 5 disease to compare golcadomide plus R-CHOP with R-CHOP alone, with readout expected in 2027 to 2028. BMS develops it. It is a next-generation lenalidomide-like pill designed to be added to standard first-line chemotherapy.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT06356129 (GOLSEEK-1)","url":"https://clinicaltrials.gov/study/NCT06356129"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["protac-degrader"],"targets":["ikzf1","ikzf3"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["golseek-1","nct06911502","nct06090539","nct03930953","nct06425302"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"CC-99282, BMS-986369","modality":"Molecular glue degrader (CELMoD, IKZF1/3)","mechanism":"Binds cereblon and recruits IKZF1/IKZF3 for ubiquitination and proteasomal degradation; downregulates IRF4/MYC and stimulates T cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"golidocitinib","kind":"drug","name":"Golidocitinib","aka":[],"tldr":"Golidocitinib is Dizal's JAK1 inhibitor, approved in China in 2024 as the first JAK inhibitor for relapsed peripheral T-cell lymphoma, a cancer with few options after first-line chemotherapy.","summary":"Dizal Pharmaceutical's golidocitinib (DZD4205) was approved by the NMPA in June 2024 for relapsed or refractory peripheral T-cell lymphoma, on the JACKPOT8 trial in which about 44 percent of patients responded. It is the first JAK1-selective inhibitor approved for a cancer. A phase 3 trial testing it as maintenance after first-line therapy is registered.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Golidocitinib","url":"https://clinicaltrials.gov/search?intr=DZD4205"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["peripheral-t-cell-lymphoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["jak1"],"drugs":[],"companies":["dizal"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07234162","nct07496229"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"DZD4205","modality":"Oral selective JAK1 inhibitor","mechanism":"Selectively inhibits Janus kinase 1, the signalling node that peripheral T-cell lymphoma cells use through the JAK-STAT pathway.","approvals":[{"region":"CN","year":2024,"indication":"Relapsed or refractory peripheral T-cell lymphoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"goserelin","kind":"drug","name":"Goserelin / leuprolide (ovarian function suppression)","aka":[],"tldr":"Monthly or 3-monthly injections that switch off the ovaries, letting premenopausal women use aromatase inhibitors and lowering recurrence in higher-risk cases.","summary":"Goserelin and leuprolide are GnRH agonists: continuous stimulation of the GnRH receptor desensitises the pituitary, suppressing LH and FSH and therefore ovarian oestradiol, producing a reversible medical menopause. In premenopausal women with HR-positive breast cancer this ovarian function suppression (OFS) lets an aromatase inhibitor be used and lowers recurrence in higher-risk cases. SOFT and TEXT showed OFS plus exemestane or tamoxifen reduces recurrence versus tamoxifen alone, with absolute gains largest in women who needed chemotherapy (12-year DFS 80.5% versus 75.9% for exemestane plus OFS versus tamoxifen plus OFS). They are also used for fertility preservation during chemotherapy (POEMS). Hot flushes, vaginal dryness and bone loss are common, so who gains enough to justify five years of induced menopause is a judgement call. In short, these injections switch the ovaries off.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Goserelin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Goserelin"}],"tags":[],"related":[],"cancers":["breast-hr-positive","hr-positive-early-high-risk"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["eurofarma"],"institutions":[],"pathways":[],"terms":["ovarian-function-suppression"],"trials":["soft-text","nct02960022"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zoladex; Lupron","modality":"GnRH agonist","mechanism":"Continuous GnRH receptor stimulation desensitises the pituitary, suppressing LH/FSH and ovarian oestradiol.","approvals":[{"region":"US","year":1989,"indication":"Advanced breast cancer (goserelin); prostate cancer"}],"mechanismSteps":[],"dosing":{"route":"Subcutaneous implant / intramuscular depot","schedule":"Goserelin 3.6 mg monthly or 10.8 mg 3-monthly; leuprolide 3.75 mg monthly or 11.25 mg 3-monthly, for 5 years","monitoring":"Oestradiol to confirm suppression; bone density"},"toxicity":[{"event":"Hot flushes","anyGradePct":93},{"event":"Vaginal dryness / sexual dysfunction","anyGradePct":50},{"event":"Bone loss","anyGradePct":30}],"access":[],"regulatoryEvents":[]},{"id":"gotistobart","kind":"drug","name":"Gotistobart","aka":["A humanized anti-CTLA4 IgG1 monoclonal antibody"],"tldr":"Gotistobart is an experimental monoclonal antibody from OncoC4 in phase 3 trials for non-small-cell lung cancer, melanoma and head and neck squamous cell carcinoma, aimed at CTLA-4.","summary":"Gotistobart (ONC-392, BNT316) is a monoclonal antibody developed by OncoC4. Its target is CTLA-4 (the sponsor names CTLA-4). The sponsor states: CTLA-4 checkpoint inhibition, in some regimens combined with pembrolizumab or Pluvicto. ClinicalTrials.gov describes the intervention as: Gotistobart will be administrated through IV infusion over 60 minutes, once every 21 days in assigned dose. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05671510 (ONC-392 Versus Docetaxel in Metastatic NSCLC That Progressed on PD-1/PD-L1 Inhibitors), in non-small-cell lung cancer, melanoma, head and neck squamous cell carcinoma, renal cell carcinoma and colorectal cancer. The largest, NCT04140526, plans to enrol 733 participants with primary completion was scheduled for 2026-06-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Gotistobart","url":"https://clinicaltrials.gov/search?intr=ONC-392"},{"label":"Sponsor page","url":"https://www.oncoc4.com/en/pipeline/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","melanoma","head-and-neck","rcc","colorectal","sarcoma","prostate","ovarian","breast-hr-positive","pancreatic","gastric","esophageal","cervical","salivary-gland","mucoepidermoid-carcinoma","urothelial"],"sections":[],"technologies":[],"targets":["ctla4"],"drugs":[],"companies":["oncoc4"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05671510","nct05446298","nct04140526","nct05682443"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ONC-392, BNT316","modality":"monoclonal antibody","mechanism":"CTLA-4 checkpoint inhibition, in some regimens combined with pembrolizumab or Pluvicto.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gq1005","kind":"drug","name":"GQ1005","aka":[],"tldr":"GQ1005 is an experimental antibody-drug conjugate from GeneQuantum Healthcare (Suzhou) in phase 3 trials for HER2-positive breast cancer, aimed at HER2.","summary":"GQ1005 is an antibody-drug conjugate developed by GeneQuantum Healthcare (Suzhou). Its target is HER2 (the sponsor names HER2). The sponsor states: A HER2-targeting antibody-drug conjugate using a cleavable open-ring linker and a Topoisomerase I inhibitor payload. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07673029 (A Trial Comparing GQ1005 and T-DM1 in Patients With HER2-Positive Unresectable or Metastatic Breast Cancer), in HER2-positive breast cancer. The largest, NCT07673029, plans to enrol 228 participants with primary completion expected 2027-01-09. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of GQ1005","url":"https://clinicaltrials.gov/search?intr=GQ1005"},{"label":"Sponsor page","url":"https://www.genequantum.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["genequantum-healthcare-suzhou"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07673029"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"A HER2-targeting antibody-drug conjugate using a cleavable open-ring linker and a Topoisomerase I inhibitor payload.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gr1803","kind":"drug","name":"GR1803","aka":[],"tldr":"GR1803 is an experimental investigational agent whose form is not stated in the registry from Genrix (Shanghai) Biopharmaceutical in phase 3 trials for multiple myeloma, with its target not yet stated publicly.","summary":"GR1803 is an investigational agent whose form is not stated in the registry developed by Genrix (Shanghai) Biopharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: D1 given at a dose of 30ug/kg, D4 given at a dose of 90ug/kg, D8 given at a dose of 180ug/kg, followed by weekly dosing up to cycle 9, and cycle 10 and onwards, every 2 weeks, with a dosing cycle of every 4 weeks. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07452198 (A Study of GR1803 Injection Versus Daratumumab, Pomalidomide, and Dexamethasone (DPd) in Participants With Relapsed or Refractory Multiple Myeloma), in multiple myeloma. The largest, NCT07452198, plans to enrol 358 participants with primary completion expected 2029-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of GR1803","url":"https://clinicaltrials.gov/search?intr=GR1803"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["genrix-bio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07452198","nct06566547"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody (BCMA x CD3, per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"granisetron","kind":"drug","name":"Granisetron","aka":["Granisetron hydrochloride","Granisetron transdermal system","Granisetron extended-release injection"],"tldr":"Granisetron is an anti-sickness medicine for chemotherapy given as a tablet, an injection, a week-long skin patch (Sancuso) or a slow-release injection that lasts several days (Sustol).","summary":"Granisetron (Kytril) was approved in December 1993 for prevention of nausea and vomiting from emetogenic chemotherapy, with tablets and a radiotherapy indication following; oral and intravenous granisetron are now generic. Two formulations extended its reach: the Sancuso transdermal patch (2008) applied 24 to 48 hours before chemotherapy and worn for up to seven days for regimens of up to five consecutive days, and Sustol (2016), a polymer-based extended-release subcutaneous injection that maintains levels for at least five days and is approved with other antiemetics for acute and delayed nausea and vomiting after moderately emetogenic or anthracycline-cyclophosphamide chemotherapy. The EU authorised Sancuso in 2012. Headache and constipation are the class effects; QT prolongation is less than with dolasetron.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Granisetron","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=granisetron"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/granisetron"},{"label":"NCI page (granisetron hydrochloride)","url":"https://www.cancer.gov/about-cancer/treatment/drugs/granisetronhydrochloride"},{"label":"EPAR (Sancuso)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/sancuso"}],"tags":["nci-list","supportive","generic"],"related":["ondansetron","palonosetron"],"cancers":[],"sections":[],"technologies":["antiemetic-therapy"],"targets":[],"drugs":[],"companies":["heron-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00186628"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Kytril (historic) / Sancuso / Sustol","modality":"5-HT3 receptor antagonist (antiemetic)","supportive":true,"mechanism":"Selective serotonin 5-HT3 receptor antagonist acting on vagal afferents in the gut and the chemoreceptor trigger zone.","approvals":[{"region":"US","year":1993,"indication":"Prevention of nausea and vomiting from emetogenic chemotherapy (Kytril; radiotherapy later)"},{"region":"US","year":2008,"indication":"Transdermal system for chemotherapy of up to five consecutive days (Sancuso)"},{"region":"US","year":2016,"indication":"Extended-release injection for acute and delayed nausea and vomiting with moderately emetogenic or anthracycline-cyclophosphamide chemotherapy (Sustol)"},{"region":"EU","year":2012,"indication":"Transdermal granisetron for moderately or highly emetogenic chemotherapy (Sancuso)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"grt-c901","kind":"drug","name":"GRT-C901","aka":[],"tldr":"GRT-C901 is an experimental cancer vaccine from Seattle Project in phase 3 trials for colorectal cancer, with its target not yet stated publicly.","summary":"GRT-C901 is a cancer vaccine developed by Seattle Project. The sponsor describes its target as patient-specific neoantigens, which OnCo does not yet have a target page for. The sponsor states: A patient-specific neoantigen cancer vaccine given as the prime dose (1x10^12 viral particles, intramuscular) in a heterologous prime/boost regimen with GRT-R902, combined with checkpoint inhibition, intended to generate T-cell responses specific to each patient's tumour mutations. ClinicalTrials.gov describes the intervention as: A patient-specific neoantigen cancer vaccine administered via intramuscular (IM) injection as prime and single boost at a dose of 1x10\\^12 viral particles 2 times over the course of the first year. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05141721 (A Study of a Patient-Specific Neoantigen Vaccine in Combination With Immune Checkpoint Blockade for Patients With Metastatic Colorectal Cancer), in colorectal cancer. The largest, NCT05141721, plans to enrol 700 participants with primary completion expected 2027-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of GRT-C901","url":"https://clinicaltrials.gov/search?intr=GRT-C901"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["gritstone"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05141721"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"vaccine","mechanism":"A patient-specific neoantigen cancer vaccine given as the prime dose (1x10^12 viral particles, intramuscular) in a heterologous prime/boost regimen with GRT-R902, combined with checkpoint inhibition, intended to generate T-cell responses specific to each patient's tumour mutations.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"grt-r902","kind":"drug","name":"GRT-R902","aka":[],"tldr":"GRT-R902 is an experimental cancer vaccine from Seattle Project in phase 3 trials for colorectal cancer, with its target not yet stated publicly.","summary":"GRT-R902 is a cancer vaccine developed by Seattle Project. The sponsor describes its target as patient-specific neoantigens, which OnCo does not yet have a target page for. The sponsor states: A patient-specific neoantigen cancer vaccine boost (30 micrograms, intramuscular, given four times over the first year) used after the GRT-C901 prime, as part of a heterologous prime/boost regimen with checkpoint inhibition to generate T-cell responses against patient-specific tumour mutations. ClinicalTrials.gov describes the intervention as: A patient-specific neoantigen cancer vaccine boost, administered via IM injection at a dose of 30ug 4 times over the course of the first year. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05141721 (A Study of a Patient-Specific Neoantigen Vaccine in Combination With Immune Checkpoint Blockade for Patients With Metastatic Colorectal Cancer), in colorectal cancer. The largest, NCT05141721, plans to enrol 700 participants with primary completion expected 2027-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of GRT-R902","url":"https://clinicaltrials.gov/search?intr=GRT-R902"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["gritstone"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05141721"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"vaccine","mechanism":"A patient-specific neoantigen cancer vaccine boost (30 micrograms, intramuscular, given four times over the first year) used after the GRT-C901 prime, as part of a heterologous prime/boost regimen with checkpoint inhibition to generate T-cell responses against patient-specific tumour mutations.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"gsk2256098","kind":"drug","name":"GSK2256098","aka":["GSK-2256098"],"tldr":"GSK2256098 is a tablet that blocks an enzyme called FAK, which meningiomas missing the NF2 gene rely on. In the Alliance A071401 trial it slowed progression in recurrent NF2-mutant meningiomas, one of the first positive results for a targeted drug in this tumour.","summary":"GSK2256098 is a small-molecule inhibitor of focal adhesion kinase developed by GSK. Loss of NF2 (merlin), the commonest driver in meningioma, makes cells dependent on FAK signalling for growth on and adhesion to their matrix, which preclinical work showed could be exploited.\n\nIn Alliance A071401, a genomically guided phase 2 trial that assigns progressive meningiomas to drugs by mutation, the FAK-inhibitor arm for NF2-mutant tumours met its progression-free survival endpoint: six-month progression-free survival was 83 percent for grade 1 and 33 percent for grade 2 to 3 tumours, with good tolerability (Journal of Clinical Oncology, 2023). The meningioma page names it as the first mutation-matched treatment to show activity in the disease.","status":"phase-2","asOf":"2026-09-24","links":[{"label":"Alliance A071401 (J Clin Oncol 2023)","url":"https://doi.org/10.1200/JCO.21.02371"},{"label":"ClinicalTrials.gov NCT02523014","url":"https://clinicaltrials.gov/study/NCT02523014"}],"tags":["subtype-drugs-wave"],"related":[],"cancers":["meningioma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["fak"],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["a071401"],"people":[],"bottlenecks":[],"keyPapers":["paper-a071401-brastianos-jco-2023"],"journals":[],"dependsOn":[],"notes":[],"code":"GSK2256098","modality":"Oral focal adhesion kinase (FAK) inhibitor","mechanism":"An ATP-competitive inhibitor of focal adhesion kinase; meningiomas that have lost the NF2 tumour suppressor merlin depend on FAK signalling, so blocking it is a synthetic-lethal approach to NF2-mutant tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"guardant-reveal","kind":"drug","name":"Guardant Reveal","aka":[],"tldr":"A blood test for leftover cancer after surgery that needs no tumour sample, so results come faster than tumour-informed tests.","summary":"Guardant Reveal launched in 2021 as the first tissue-free MRD test, initially for stage II-III colorectal cancer, with Medicare coverage for colorectal cancer surveillance and later breast and lung indications. Tumour-naive design gives a result within about a week of surgery and suits patients whose tissue is unavailable, at the cost of somewhat lower sensitivity than bespoke tumour-informed panels such as Signatera. Published colorectal data show ctDNA positivity after surgery carries a high risk of recurrence, and serial negative results are strongly reassuring. It is a laboratory-developed test under CLIA.","status":"established","asOf":"2026-09-10","links":[{"label":"Guardant Reveal (page moved; nearest live section)","url":"https://guardanthealth.com/"}],"tags":["test"],"related":["signatera","radar-mrd"],"cancers":["colorectal","breast-hr-positive","nsclc"],"sections":[],"technologies":["mrd-testing","liquid-biopsy","methylation-profiling"],"targets":[],"drugs":[],"companies":["guardant-health"],"institutions":[],"pathways":[],"terms":["mrd","ctdna"],"trials":["nct07802626"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: ctDNA detected after surgery means a high chance of recurrence and a case for closer follow-up or adjuvant therapy; not detected is reassuring but not a guarantee.","Colorectal cancer: a tumour-naive residual disease assay combining methylation and mutation signal, used in the same postoperative setting as the tumour-informed tests but without needing tumour tissue; the randomised evidence in colorectal cancer is for tumour-informed assays (Tie 2022), so the equivalence is inferred rather than shown."],"brand":"Guardant Reveal","modality":"Tumour-naive ctDNA minimal residual disease test (methylation and genomic)","mechanism":"Blood-only assay combining somatic variant detection with cancer-specific methylation signals, so no tumour tissue is needed to design the test.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"guardant360-cdx","kind":"drug","name":"Guardant360 CDx","aka":[],"tldr":"A blood test that reads a tumour's mutations without a tissue biopsy and is the FDA-approved gateway to several targeted drugs.","summary":"Guardant360 CDx was the first blood-based comprehensive genomic profiling test approved by the FDA (7 August 2020, PMA P200010), initially as a companion diagnostic for osimertinib in EGFR-mutant non-small-cell lung cancer. Companion claims were added for sotorasib (KRAS G12C, 2021), amivantamab (EGFR exon 20 insertions, 2021) and elacestrant (ESR1 mutations in ER-positive breast cancer, 2023). A negative blood result does not exclude an alteration, because some tumours shed little DNA; labels direct reflex tissue testing when plasma is negative. Guardant360 also reports tumour profiling results beyond the companion claims as professional information.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P200010","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P200010"}],"tags":["test"],"related":["foundationone-cdx","cobas-egfr-mutation-test"],"cancers":["nsclc","breast-hr-positive","pancreatic","colorectal"],"sections":[],"technologies":["liquid-biopsy","companion-diagnostic","cgp"],"targets":["egfr","kras","estrogen-receptor","met","alk","ret","braf"],"drugs":["osimertinib","sotorasib","amivantamab","elacestrant"],"companies":["guardant-health"],"institutions":[],"pathways":[],"terms":["ctdna","companion-diagnostic-term","esr1-mutation"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-diaz-molecular-evolution-egfr-resistance-colorectal-nature-2012","paper-nakamura-triumph-ctdna-pertuzumab-trastuzumab-her2-colorectal-nat-med-2021"],"journals":[],"dependsOn":[],"notes":["What a result means: a detected alteration with a companion claim makes you eligible for the matched drug; a negative blood test should be followed by tissue testing before ruling a target out.","Pancreatic ductal adenocarcinoma: plasma panels detect KRAS in about half of advanced patients, with detection and allele fraction both prognostic, but a negative result does not exclude disease and no pancreatic label uses a plasma threshold (Pietrasz 2017, Bernard 2019).","Colorectal cancer: plasma panels genotype RAS and BRAF when tissue is exhausted, track the RAS-mutant clones that cause acquired resistance to EGFR antibodies months before imaging (Diaz 2012), and can select HER2-amplified patients for treatment as accurately as tissue when tumour fraction is adequate (Nakamura 2021). A negative plasma result in low-volume disease does not exclude an alteration."],"brand":"Guardant360 CDx","modality":"Liquid biopsy companion diagnostic test (NGS, cfDNA)","mechanism":"Hybrid-capture next-generation sequencing of cell-free DNA from a blood draw, reporting mutations, amplifications and fusions across more than 50 genes with companion-diagnostic claims for specific drugs.","approvals":[{"region":"US","year":2020,"indication":"Companion diagnostic for osimertinib (EGFR) in NSCLC; first liquid comprehensive genomic profiling test"},{"region":"US","year":2021,"indication":"Companion claims for sotorasib (KRAS G12C) and amivantamab (EGFR exon 20 insertion)"},{"region":"US","year":2023,"indication":"Companion diagnostic for elacestrant (ESR1 mutation) in ER+/HER2- advanced breast cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"h101-oncolytic-adenovirus","kind":"drug","name":"H101 oncolytic adenovirus","aka":["Oncorine"],"tldr":"Oncorine is Shanghai Sunway Biotech's oncolytic adenovirus, approved in China in 2005 for nasopharyngeal carcinoma with chemotherapy, the first oncolytic virus approved anywhere in the world.","summary":"H101 (Oncorine) is an E1B-55K-deleted adenovirus derived from the same design as ONYX-015, developed by Shanghai Sunway Biotech. The NMPA (then the SFDA) approved it in November 2005 for late-stage nasopharyngeal carcinoma in combination with chemotherapy, ten years before talimogene laherparepvec became the first oncolytic virus approved in the United States. It is injected into the tumour.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of H101","url":"https://clinicaltrials.gov/search?intr=H101"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["nasopharyngeal"],"sections":[],"technologies":["oncolytic-virus"],"targets":[],"drugs":[],"companies":["shanghai-sunway-biotech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03780049","nct02579564","nct07398664"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"H101","modality":"Oncolytic virus (E1B-deleted adenovirus)","mechanism":"An adenovirus lacking the E1B-55K gene replicates only in cells with a broken p53 pathway, bursting tumour cells and alerting the immune system.","approvals":[{"region":"CN","year":2005,"indication":"Late-stage nasopharyngeal carcinoma, with chemotherapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hb-200","kind":"drug","name":"HB-200","aka":["HB-201","HB-202","Eseba-vec"],"tldr":"HB-200 is a vaccine built from two harmless engineered viruses that teach the immune system to attack the HPV16 proteins inside cervical and throat cancers. It has been tested with pembrolizumab in HPV16-positive head and neck cancer.","summary":"HB-200 is Hookipa Pharma's therapeutic vaccine for HPV16-positive cancers, made of two arenavirus vectors, HB-201 (based on lymphocytic choriomeningitis virus) and HB-202 (based on Pichinde virus), that express the same HPV16 E6/E7 fusion antigen. Alternating the two vectors avoids immunity against the vector itself and produced some of the largest antigen-specific T-cell expansions reported for a cancer vaccine.\n\nIt was given intravenously with pembrolizumab in phase 1/2 trials in recurrent or metastatic HPV16-positive head and neck squamous cell carcinoma, with response rates in the first-line setting above those expected from pembrolizumab alone. It is named on the recurrent or metastatic head and neck cancer page among the newer immunotherapy approaches for HPV-positive disease.","status":"phase-2","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["pembrolizumab"],"cancers":["recurrent-metastatic-hnscc"],"sections":[],"technologies":["hpv-vaccine"],"targets":[],"drugs":[],"companies":["hookipa-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"HB-200","modality":"Arenavirus-vector therapeutic vaccine (HPV16 E6/E7)","mechanism":"Two replicating arenavirus vectors (lymphocytic choriomeningitis and Pichinde virus) each carrying a non-oncogenic HPV16 E6/E7 fusion protein, given alternately to raise very high frequencies of E6- and E7-specific killer T cells against HPV16-driven tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hb0036","kind":"drug","name":"HB0036","aka":["Anti-PD-L1 and anti-TIGIT bifunctional molecule"],"tldr":"HB0036 is an experimental investigational agent whose form is not stated in the registry from Shanghai Huaota Biopharmaceutical in phase 2 trials for non-small-cell lung cancer, aimed at PD-L1 and TIGIT.","summary":"HB0036 is an investigational agent whose form is not stated in the registry developed by Shanghai Huaota Biopharmaceutical. Its targets are PD-L1 and TIGIT (the sponsor names PD-L1 x TIGIT). The sponsor states: HB0036 is an anti-PD-L1/anti-TIGIT bifunctional antibody molecule, given by intravenous infusion every three weeks, simultaneously engaging both checkpoint pathways. ClinicalTrials.gov describes the intervention as: Patients will be assigned to dose regimens in the order of enrollment, and they will receive their assigned fixed dose of HB0036 via intravenous infusion. HB0036 IV every 3 weeks (q3w). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT05417321, plans to enrol 80 participants with primary completion was scheduled for 2025-08-01 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HB0036","url":"https://clinicaltrials.gov/search?intr=HB0036"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["pdl1","tigit"],"drugs":[],"companies":["huabo-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05417321"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bifunctional antibody (anti-PD-L1/anti-TIGIT, per the sponsor's description; form not stated in the registry record)","mechanism":"HB0036 is an anti-PD-L1/anti-TIGIT bifunctional antibody molecule, given by intravenous infusion every three weeks, simultaneously engaging both checkpoint pathways.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hb1801","kind":"drug","name":"HB1801","aka":[],"tldr":"HB1801 is an experimental investigational agent whose form is not stated in the registry from Shanghai JMT-Bio in phase 3 trials for HER2-positive breast cancer, with its target not yet stated publicly.","summary":"HB1801 is an investigational agent whose form is not stated in the registry developed by Shanghai JMT-Bio. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05838066 (Efficacy and Safety of KN026 in Combination With HB1801 in the First-line Treatment of Subjects With HER2-positive Recurrent or Metastatic Breast Cancer), NCT07441460 (A Phase III Study of KN026 in Combination With HB1801 as Adjuvant Therapy for Resectable HER2-Positive Breast Cancer) and NCT06747338 (A Phase III Study of KN026 in Combination With HB1801 ± Carboplatin as Neoadjuvant Treatment for Early or Locally Advanced HER2-Positive Breast Cancer), in HER2-positive breast cancer. The largest, NCT07441460, plans to enrol 1800 participants with primary completion expected 2034-08-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HB1801","url":"https://clinicaltrials.gov/search?intr=HB1801"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05838066","nct07441460","nct06747338"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hdm2017","kind":"drug","name":"HDM2017","aka":[],"tldr":"HDM2017 is an experimental investigational agent whose form is not stated in the registry from Hangzhou Zhongmei Huadong Pharmaceutical in phase 2 trials for colorectal cancer, aimed at MET.","summary":"HDM2017 is an investigational agent whose form is not stated in the registry developed by Hangzhou Zhongmei Huadong Pharmaceutical. Its target is MET. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in colorectal cancer. The largest, NCT07805551, plans to enrol 220 participants with primary completion expected 2030-08. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HDM2017","url":"https://clinicaltrials.gov/search?intr=HDM2017"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["met"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07805551"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Investigational agent whose form is not stated in the registry directed at MET, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"her2-testing-assays","kind":"drug","name":"HER2 IHC and ISH companion assays (HercepTest, PATHWAY 4B5, HER2 Dual ISH)","aka":[],"tldr":"The tests that grade a breast or stomach cancer's HER2 level, from the original trastuzumab test in 1998 to the new 'HER2-low' and 'ultralow' cut-offs.","summary":"HercepTest (Dako, PMA P980018, 25 September 1998) was the first companion diagnostic ever approved, launched with trastuzumab. Roche's PATHWAY anti-HER2/neu (4B5) and INFORM HER2 Dual ISH are the other FDA-approved workhorses, extended to gastric cancer with the ToGA trial. The HER2-low era changed what a score means: in October 2022 the FDA approved PATHWAY 4B5 as the companion diagnostic for trastuzumab deruxtecan in HER2-low (IHC 1+ or 2+/ISH-negative) metastatic breast cancer after DESTINY-Breast04, and in January 2025 extended it to HER2-ultralow (IHC 0 with membrane staining) after DESTINY-Breast06. A score of 0 versus 1+ that once meant nothing now decides eligibility for an antibody-drug conjugate, which has forced pathologists to re-standardise the low end of the scale.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P980018","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P980018"},{"label":"ASCO/CAP HER2 testing guideline update (2023)","url":"https://doi.org/10.1200/JCO.22.02864"}],"tags":["test"],"related":[],"cancers":["breast-her2-positive","breast-hr-positive","gastric","tnbc"],"sections":[],"technologies":["companion-diagnostic","histopathology-ihc","cytogenetics-fish"],"targets":["her2"],"drugs":["trastuzumab","trastuzumab-deruxtecan"],"companies":["agilent","roche-genentech"],"institutions":[],"pathways":[],"terms":["her2-positive","her2-low","ihc","fish"],"trials":["destiny-breast04","destiny-breast06"],"people":[],"bottlenecks":[],"keyPapers":["paper-schettini-her2-low-features-npj-breast-cancer-2021","paper-boissiere-michot-her2-ultralow-tnbc-virchows-arch-2026","paper-wolff-j-clin-oncol"],"journals":[],"dependsOn":[],"notes":["What a result means: 3+ (or 2+ with amplification) is HER2-positive and unlocks trastuzumab-based therapy; 1+ or 2+ without amplification is HER2-low and, in metastatic disease, unlocks trastuzumab deruxtecan."],"brand":"HercepTest / PATHWAY anti-HER2 (4B5) / INFORM HER2 Dual ISH","modality":"HER2 immunohistochemistry and in situ hybridisation companion diagnostic assays","mechanism":"Immunohistochemistry for HER2 protein scored 0, 1+, 2+ or 3+ under ASCO/CAP guidelines, with in situ hybridisation (FISH or dual-colour chromogenic ISH) to resolve 2+ cases by measuring ERBB2 gene amplification.","approvals":[{"region":"US","year":1998,"indication":"HercepTest: HER2 overexpression to select trastuzumab in breast cancer (first companion diagnostic)"},{"region":"US","year":2010,"indication":"HER2 testing extended to gastric and GEJ adenocarcinoma with trastuzumab (ToGA)"},{"region":"US","year":2022,"indication":"PATHWAY 4B5 as companion diagnostic for trastuzumab deruxtecan in HER2-low breast cancer"},{"region":"US","year":2025,"indication":"PATHWAY 4B5 extended to HER2-ultralow (IHC 0 with membrane staining)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hexaminolevulinate","kind":"drug","name":"Hexaminolevulinate","aka":["HAL","Hexyl aminolevulinate"],"tldr":"Hexaminolevulinate makes bladder tumours fluoresce red under blue light so the urologist can see and remove flat and tiny cancers that white-light cystoscopy misses.","summary":"Instilled into the bladder an hour before cystoscopy, hexaminolevulinate is taken up preferentially by neoplastic urothelium and converted to protoporphyrin IX. The FDA approved it in May 2010 (Cysview) as an adjunct to white-light cystoscopy for detecting non-muscle-invasive papillary bladder cancer, after trials showed it found additional Ta and T1 tumours and carcinoma in situ in a meaningful share of patients; it has been marketed in Europe as Hexvix since 2005. Blue-light cystoscopy with hexaminolevulinate reduces early recurrence after resection in meta-analyses and is recommended in European and American guidelines for suspected carcinoma in situ.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Hexaminolevulinate","links":[{"label":"Drugs@FDA NDA022555","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=022555"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Hexaminolevulinate"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":["sentinel-node"],"targets":[],"drugs":[],"companies":["photocure"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cysview / Hexvix","modality":"Optical imaging agent instilled into the bladder","mechanism":"A lipophilic ester of aminolevulinic acid that tumour cells convert to fluorescent protoporphyrin IX; under blue light at cystoscopy, bladder tumours glow red against normal urothelium.","approvals":[{"region":"US","year":2010,"indication":"Cystoscopic detection of non-muscle-invasive papillary bladder cancer, with blue-light cystoscopy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"histamine-dihydrochloride","kind":"drug","name":"Histamine dihydrochloride","aka":[],"tldr":"Ceplene is a twice-daily injection of histamine given alongside low-dose interleukin-2 as maintenance treatment for adults with acute myeloid leukaemia in first remission, to help immune cells prevent relapse. It is authorised in Europe only.","summary":"Histamine dihydrochloride was authorised in the EU in October 2008 under exceptional circumstances, with low-dose interleukin-2, as maintenance therapy for adults with acute myeloid leukaemia in first complete remission, on a phase 3 trial of 320 patients in which the combination improved leukaemia-free survival compared with no maintenance; the label notes efficacy is not fully established over age 60 and an FDA application was not approvable in 2000. It is one of the very few approved maintenance immunotherapies in AML, though oral azacitidine (Onureg) and FLT3 inhibitors now fill that space, and it is little used. Flushing, headache, hypotension and injection-site reactions occur with each histamine dose.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Histamine_dihydrochloride","links":[{"label":"Study 0201 (Blood 2006)","url":"https://doi.org/10.1182/blood-2005-10-4073"},{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/ceplene"}],"tags":["ema-list"],"related":["aldesleukin"],"cancers":["aml"],"sections":[],"technologies":["cytokine-therapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["study-0201"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ceplene","modality":"Immunomodulator (H2 receptor agonist) given with interleukin-2","mechanism":"Inhibits NADPH oxidase in monocytes and macrophages via H2 receptors, reducing the reactive oxygen species that inactivate NK and T cells and thereby protecting IL-2-activated lymphocytes in the leukaemic microenvironment.","approvals":[{"region":"EU","year":2008,"indication":"Maintenance with interleukin-2 for adults with AML in first remission (exceptional circumstances)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"histrelin","kind":"drug","name":"Histrelin","aka":["Histrelin acetate"],"tldr":"Histrelin is a GnRH agonist delivered by a small implant under the skin of the arm that lasts a whole year, approved in the United States as Vantas for palliative treatment of advanced prostate cancer and as Supprelin for early puberty in children.","summary":"Histrelin acetate was first approved in the United States in 1991 as a daily injection for central precocious puberty. The Vantas hydrogel implant, approved in 2004, releases the drug for twelve months and was labelled for palliative treatment of advanced prostate cancer, suppressing testosterone as effectively as monthly or three-monthly leuprolide with one minor procedure a year. Vantas was discontinued in 2021 after manufacturing problems, while the Supprelin LA implant for children continues. Hot flushes, fatigue and the class effects of androgen deprivation apply.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Histrelin","links":[{"label":"Drugs@FDA NDA022058","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=022058"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Histrelin"},{"label":"ChEMBL CHEMBL1201303","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201303"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["endocrine-therapy"],"targets":["gnrhr"],"drugs":["leuprolide","goserelin"],"companies":["endo"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01697384","nct01394263","nct04513717"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vantas / Supprelin LA","modality":"GnRH agonist, twelve-month subcutaneous implant","mechanism":"A potent GnRH analogue that, given continuously, desensitises the pituitary so luteinising hormone and testosterone fall to castrate levels after an initial surge.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hlx11","kind":"drug","name":"HLX11","aka":[],"tldr":"HLX11 is an experimental monoclonal antibody from Shanghai Henlius Biotech in phase 3 trials for HR-positive / HER2-negative breast cancer and HER2-positive breast cancer, aimed at HER2.","summary":"HLX11 is a monoclonal antibody developed by Shanghai Henlius Biotech. Its target is HER2 (the sponsor names HER2). The sponsor states: A recombinant anti-HER2 domain II humanised monoclonal antibody developed as a biosimilar to pertuzumab (EU-Perjeta), given with trastuzumab and docetaxel in neoadjuvant HER2-positive breast cancer. ClinicalTrials.gov describes the intervention as: Neoadjuvant(q3w/cycle，total 4cycle): HLX11(loading dose of 840 mg IV, followed by 420 mg IV q3w)+trastuzumab(loading dose of 8 mg/kg IV, followed by 6mg/kg IV q3w)+docetaxel(75mg/m2 IV q3w) Adjuvant: doxorubicin( 60 mg/m2 IV q3w)+cyclophos. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05346224 (A Study to Evaluate the Efficacy and Safety of HLX11 vs. EU-Perjeta® in the Neoadjuvant Therapy of HER2-Positive and HR-Negative Early-stage or Locally Advanced Breast Cancer), in HR-positive / HER2-negative breast cancer and HER2-positive breast cancer. The largest, NCT05346224, plans to enrol 900 participants with primary completion was scheduled for 2024-05-15 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HLX11","url":"https://clinicaltrials.gov/search?intr=HLX11"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","breast-her2-positive"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["henlius"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05346224"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"A recombinant anti-HER2 domain II humanised monoclonal antibody developed as a biosimilar to pertuzumab (EU-Perjeta), given with trastuzumab and docetaxel in neoadjuvant HER2-positive breast cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hlx22","kind":"drug","name":"HLX22","aka":[],"tldr":"HLX22 is an experimental monoclonal antibody from Shanghai Henlius Biotech in phase 3 trials for gastric & gastro-oesophageal junction cancer and HR-positive / HER2-negative breast cancer, aimed at HER2.","summary":"HLX22 is a monoclonal antibody developed by Shanghai Henlius Biotech. Its target is HER2 (the sponsor names HER2). The sponsor states: HLX22 is a recombinant humanised anti-HER2 monoclonal antibody injection, given at 15 mg/kg every three weeks in combination with trastuzumab and chemotherapy (XELOX) in HER2-positive gastric or gastroesophageal junction adenocarcinoma. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06532006 (A Phase Ⅲ Clinical Study of HLX22 in Combination With Trastuzumab and Chemotherapy for the Treatment of Gastroesophageal Junction and Gastric Cancer), in gastric & gastro-oesophageal junction cancer and HR-positive / HER2-negative breast cancer. The largest, NCT06532006, plans to enrol 550 participants with primary completion expected 2027-06-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HLX22","url":"https://clinicaltrials.gov/search?intr=HLX22"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric","breast-hr-positive"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["henlius"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06532006","nct06832202"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"HLX22 is a recombinant humanised anti-HER2 monoclonal antibody injection, given at 15 mg/kg every three weeks in combination with trastuzumab and chemotherapy (XELOX) in HER2-positive gastric or gastroesophageal junction adenocarcinoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hlx26","kind":"drug","name":"HLX26","aka":["Anti-LAG-3 monoclonal Antibody Injection"],"tldr":"HLX26 is an experimental monoclonal antibody from Shanghai Henlius Biotech in phase 2 trials for non-small-cell lung cancer, aimed at LAG-3.","summary":"HLX26 is a monoclonal antibody developed by Shanghai Henlius Biotech. Its target is LAG-3 (the sponsor names LAG-3). The sponsor states: An anti-LAG-3 monoclonal antibody injection that blocks LAG-3 signalling to enhance T-cell mediated anti-tumour immunity, studied in combination with PD-1 inhibition and chemotherapy. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT05787613, plans to enrol 132 participants with primary completion was scheduled for 2026-02 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HLX26","url":"https://clinicaltrials.gov/search?intr=HLX26"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["lag3"],"drugs":[],"companies":["henlius"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05787613"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"An anti-LAG-3 monoclonal antibody injection that blocks LAG-3 signalling to enhance T-cell mediated anti-tumour immunity, studied in combination with PD-1 inhibition and chemotherapy.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hlx43","kind":"drug","name":"HLX43","aka":[],"tldr":"HLX43 is an experimental antibody-drug conjugate from Shanghai Henlius Biotech in phase 3 trials for non-small-cell lung cancer, cervical cancer and ovarian cancer, aimed at PD-L1 and EGFR.","summary":"HLX43 is an antibody-drug conjugate developed by Shanghai Henlius Biotech. Its targets are PD-L1 and EGFR (the sponsor names PD-L1). The sponsor states: HLX43 is an anti-PD-L1 antibody-drug conjugate (ADC), studied as monotherapy or combined with the anti-EGFR antibody HLX07. ClinicalTrials.gov describes the intervention as: HLX43 is an anti-PD-L1 monoclonal antibody conjugated with a novel high potency DNA topoisomerase I (topo I) inhibitor, with a drug-antibody-ratio (DAR) of 8. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07459751 (A Global Phase II/III Clinical Study to Evaluate the Efficacy and Safety of HLX43 Monotherapy or HLX43 in Combination With HLX07 Versus Docetaxel in Advanced or Metastatic Squamous Non-Small Cell Lung Cancer), in non-small-cell lung cancer, cervical cancer, ovarian cancer and oesophageal cancer. The largest, NCT07459751, plans to enrol 706 participants with primary completion expected 2030-01-15. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HLX43","url":"https://clinicaltrials.gov/search?intr=HLX43"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","cervical","ovarian","esophageal"],"sections":[],"technologies":[],"targets":["pdl1","egfr"],"drugs":[],"companies":["henlius"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07459751","nct06769152","nct06769113"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"HLX43 is an anti-PD-L1 antibody-drug conjugate (ADC), studied as monotherapy or combined with the anti-EGFR antibody HLX07.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hmbd-001","kind":"drug","name":"HMBD-001","aka":[],"tldr":"HMBD-001 is an experimental monoclonal antibody from Hummingbird Bioscience in phase 2 trials for non-small-cell lung cancer, head and neck squamous cell carcinoma and oesophageal cancer, aimed at HER3 and EGFR.","summary":"HMBD-001 is a monoclonal antibody developed by Hummingbird Bioscience. Its targets are HER3 and EGFR. ClinicalTrials.gov describes the intervention as: HMBD-001 is a humanized Immunoglobulin G1 (IgG1) anti-Human Epidermal Growth Factor Receptor 3(HER3) monoclonal antibody (mAb). It is administered intravenously (IV) weekly. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer, head and neck squamous cell carcinoma, oesophageal cancer, cervical cancer and cutaneous squamous cell carcinoma. The largest, NCT05910827, plans to enrol 398 participants with primary completion expected 2027-12-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HMBD-001","url":"https://clinicaltrials.gov/search?intr=HMBD-001"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","head-and-neck","esophageal","cervical","cutaneous-scc","nasopharyngeal"],"sections":[],"technologies":[],"targets":["her3","egfr"],"drugs":[],"companies":["hummingbird-bioscience"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05910827"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at HER3 and EGFR, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hmpl-306","kind":"drug","name":"HMPL-306","aka":["HMPL-306 Regimen"],"tldr":"HMPL-306 is an experimental small-molecule drug from Hutchmed in phase 3 trials for acute myeloid leukaemia, aimed at IDH1 / IDH2.","summary":"HMPL-306 is a small-molecule drug developed by Hutchmed. Its target is IDH1 / IDH2 (the sponsor names IDH1/IDH2 (mutant)). ClinicalTrials.gov describes the intervention as: Patients will receive HMPL-306 monotherapy: HMPL-306 PO at 250 mg QD (C1) + 150 mg QD (starting from C2), 28 days as a cycle. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06387069 (A Study to Evaluate HMPL-306 in Patients With IDH1or IDH2-mutated Acute Myeloid Leukaemia), in acute myeloid leukaemia. The largest, NCT06387069, plans to enrol 316 participants with primary completion expected 2028-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HMPL-306","url":"https://clinicaltrials.gov/search?intr=HMPL-306"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["aml"],"sections":[],"technologies":[],"targets":["idh"],"drugs":[],"companies":["hutchmed"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06387069"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Small-molecule drug directed at IDH1 / IDH2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hmpl-453","kind":"drug","name":"HMPL-453","aka":[],"tldr":"HMPL-453 is an experimental small-molecule drug from Hutchmed in phase 3 trials for biliary tract cancer, aimed at FGFR2.","summary":"HMPL-453 is a small-molecule drug developed by Hutchmed. Its target is FGFR2 (the sponsor names FGFR2). ClinicalTrials.gov describes the intervention as: Cohort\\_1:HMPL-453 150mg QD continuously in 21-day cycles; Cohort\\_2, Cohort\\_3 and Cohort\\_4:HMPL-453 tartrate 300 mg QD orally (for 14 consecutive days \\[Day 1 to 14\\], followed by 7 days off \\[Day 15 to 21\\], 21 days as a treatment cycle. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04353375 (HMPL-453 Tartrate in Advanced Intrahepatic Cholangiocarcinoma), in biliary tract cancer. The largest, NCT04353375, plans to enrol 235 participants with primary completion expected 2028-06-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HMPL-453","url":"https://clinicaltrials.gov/search?intr=HMPL-453"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cholangiocarcinoma"],"sections":[],"technologies":[],"targets":["fgfr2"],"drugs":[],"companies":["hutchmed"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04353375"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Small-molecule drug directed at FGFR2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hmpl-760","kind":"drug","name":"HMPL-760","aka":[],"tldr":"HMPL-760 is an experimental small-molecule drug from Hutchmed in phase 3 trials for diffuse large B-cell lymphoma, with its target not yet stated publicly.","summary":"HMPL-760 is a small-molecule drug developed by Hutchmed. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Patients will receive HMPL-760 once daily (QD) orally. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07409428 (A Phase III Study of HMPL-760 Plus R-GemOx VS Placebo Plus R-GemOx in Relapsed/Refractory DLBCL), in diffuse large B-cell lymphoma. The largest, NCT07409428, plans to enrol 240 participants with primary completion expected 2028-04-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HMPL-760","url":"https://clinicaltrials.gov/search?intr=HMPL-760"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hutchmed"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07409428"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hpv-bivalent-vaccine","kind":"drug","name":"HPV bivalent vaccine (types 16 and 18)","aka":[],"tldr":"Cervarix is a vaccine against HPV types 16 and 18, the two types that cause most cervical cancers, authorised in the EU in 2007 and the US in 2009 after the 18,000-woman PATRICIA trial showed near-complete protection against type-specific precancer. GSK withdrew it from the US in 2016, but it remains WHO-prequalified and used in national programmes, including single-dose schedules.","summary":"The bivalent HPV vaccine was authorised in the EU in September 2007 and approved by the FDA in October 2009 for prevention of cervical cancer, cervical intraepithelial neoplasia and adenocarcinoma in situ caused by HPV 16 and 18 in females aged 9 to 25 (10 to 25 in the US), on the PATRICIA phase 3 trial of more than 18,000 women, which showed near-complete protection against type-specific CIN2+ in HPV-naive women and cross-protection against types 31, 33 and 45. GSK withdrew it from the US market in 2016 for commercial reasons; it remains WHO-prequalified and is used in national programmes, including single-dose schedules supported by the KEN SHE trial and the Costa Rica Vaccine Trial follow-up. Injection-site pain and fatigue are the main adverse effects.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Cervarix","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Cervarix"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/recombinant-hpv-bivalent-vaccine"},{"label":"PATRICIA (Lancet 2009)","url":"https://doi.org/10.1016/S0140-6736(09)61248-4"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/cervarix"}],"tags":["nci-list","prevention"],"related":["gardasil-9","hpv-quadrivalent-vaccine"],"cancers":["cervical"],"sections":[],"technologies":["hpv-vaccine","interception-vaccination"],"targets":[],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["patricia"],"people":[],"bottlenecks":[],"keyPapers":["paper-patricia-paavonen-lancet-2009"],"journals":[],"dependsOn":[],"notes":[],"brand":"Cervarix","modality":"Recombinant virus-like particle vaccine (adjuvanted)","mechanism":"L1 capsid virus-like particles of HPV types 16 and 18 with the AS04 adjuvant induce neutralising antibodies that prevent persistent infection with the two types responsible for about 70% of cervical cancers.","approvals":[{"region":"EU","year":2007,"indication":"Prevention of premalignant cervical lesions and cervical cancer caused by HPV 16 and 18 from age 9 (Cervarix); anal lesions added later"},{"region":"US","year":2009,"indication":"Prevention of cervical cancer and precursor lesions caused by HPV 16 and 18 in females 10 to 25 (discontinued in the US 2016)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hpv-quadrivalent-vaccine","kind":"drug","name":"HPV quadrivalent vaccine (types 6, 11, 16 and 18)","aka":["Silgard"],"tldr":"Gardasil was the first HPV vaccine, licensed in 2006 to prevent cervical and other genital cancers and genital warts. It has been replaced in most countries by the nine-type version, Gardasil 9.","summary":"The quadrivalent HPV vaccine was approved by the FDA in June 2006 for females aged 9 to 26 to prevent cervical, vulvar and vaginal cancers and precancers and genital warts caused by HPV 6, 11, 16 and 18, on the FUTURE I and II trials, with males (2009) and anal cancer prevention (2010) added; the EU authorised Gardasil in September 2006. It was the first vaccine indicated to prevent a cancer, and population data from Australia, Scandinavia and the UK have since shown large falls in cervical precancer and invasive cancer in vaccinated cohorts. Gardasil 9, covering five further oncogenic types, replaced it in the US in 2017 and in most programmes since. Syncope after injection and injection-site reactions are the main adverse effects; extensive surveillance has found no link to autoimmune disease.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Gardasil","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Gardasil"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/recombinant-hpv-quadrivalent-vaccine"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/gardasil"}],"tags":["nci-list","prevention"],"related":["gardasil-9","hpv-bivalent-vaccine"],"cancers":["cervical","vulvar","anal"],"sections":[],"technologies":["hpv-vaccine","interception-vaccination"],"targets":[],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04422366","nct06465914"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Gardasil","modality":"Recombinant virus-like particle vaccine","mechanism":"L1 virus-like particles of HPV types 6, 11, 16 and 18 on an aluminium adjuvant induce neutralising antibodies that prevent the infections behind most cervical, anal, vulvar and vaginal cancers and genital warts.","approvals":[{"region":"US","year":2006,"indication":"Prevention of cervical, vulvar and vaginal cancers and precancers and genital warts caused by HPV 6, 11, 16 and 18 in females 9 to 26 (males 2009; anal cancer 2010); superseded by Gardasil 9"},{"region":"EU","year":2006,"indication":"Prevention of premalignant genital lesions, cervical cancer and genital warts from age 9 (Gardasil); anal lesions and cancer added later"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hr070803","kind":"drug","name":"HR070803","aka":[],"tldr":"HR070803 is an experimental small-molecule drug from Jiangsu HengRui Medicine in phase 3 trials for colorectal cancer, with its target not yet stated publicly.","summary":"HR070803 is a small-molecule drug developed by Jiangsu HengRui Medicine. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. The sponsor states: Identified on the trial page as an irinotecan liposome formulation (pharmacokinetics tracked via the active metabolite SN-38 and parent compound CPT-11), given intravenously in combination with oxaliplatin, 5-FU/leucovorin and bevacizumab as first-line therapy for metastatic colorectal cancer. ClinicalTrials.gov describes the intervention as: HR070803 plus oxaliplatin, 5-FU/LV, bevacizumab Patients will receive the study drug after randomization, and those with effective efficacy evaluation (CR, PR or SD) will receive intravenous chemotherapy for up to 8-12 cycles, and then ente. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05945901 (A Phase II/III Study of HR070803 in Combination With Oxaliplatin, 5-fluorouracil, Calcium Folinate and Bevacizumab Versus FOLFOX in Combination With Bevacizumab for First-line Treatment of Advanced Colorectal Cancer), in colorectal cancer. The largest, NCT05945901, plans to enrol 669 participants (actual) with primary completion was scheduled for 2025-12-01 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HR070803","url":"https://clinicaltrials.gov/search?intr=HR070803"},{"label":"Liposomal irinotecan (HR070803) with fluorouracil and leucovorin in advanced solid tumours: phase 1b (Investigational New Drugs 2024)","url":"https://doi.org/10.1007/s10637-024-01442-2"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["top1"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05945901"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["HR070803 is a novel nanoliposomal formulation of irinotecan, so its target is topoisomerase I through the active metabolite SN-38 (phase 1b dose-escalation report, Investigational New Drugs 2024)."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Identified on the trial page as an irinotecan liposome formulation (pharmacokinetics tracked via the active metabolite SN-38 and parent compound CPT-11), given intravenously in combination with oxaliplatin, 5-FU/leucovorin and bevacizumab as first-line therapy for metastatic colorectal cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hrs-4357","kind":"drug","name":"HRS-4357","aka":[],"tldr":"HRS-4357 is an experimental investigational agent whose form is not stated in the registry from Jiangsu HengRui Medicine in phase 3 trials for prostate cancer, aimed at PSMA.","summary":"HRS-4357 is an investigational agent whose form is not stated in the registry developed by Jiangsu HengRui Medicine. Its target is PSMA (the sponsor names PSMA (inferred from trial population)). ClinicalTrials.gov describes the intervention as: HRS-4357 injection are administered each time, with dosing for 4 to 6 cycles. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07311694 (A Phase III Study Comparing HRS-4357 With Novel Androgen Receptor Pathway Inhibitors in Patients With Progressive, PSMA-Positive Metastatic Castration-Resistant Prostate Cancer), in prostate cancer. The largest, NCT07311694, plans to enrol 370 participants with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HRS-4357","url":"https://clinicaltrials.gov/search?intr=HRS-4357"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["psma"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07311694"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Investigational agent whose form is not stated in the registry directed at PSMA, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hrs-8080","kind":"drug","name":"HRS-8080","aka":[],"tldr":"HRS-8080 is an experimental small-molecule drug from Shandong Suncadia Medicine in phase 3 trials for HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"HRS-8080 is a small-molecule drug developed by Shandong Suncadia Medicine. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: * HRS-8080 Tablet * Dalpiciclib Isethionate. It is the investigational product in 5 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07354022 (A Study to Evluate Efficacy and Safety of HRS-8080 Combined With Dalpiciclib in Patients With Advanced or Metastatic Breast Cancer Resistant to Adjuvant Endocrine Therapy), NCT07024173 (A Phase 3 Study of HRS-8080 Versus Treatment Chosen by Physicians in Locally Advanced and Metastatic Breast Cancer) and NCT07349069 (A Phase III Trial Comparing HRS-8080 With Standard Endocrine Therapy in Patients With Intermediate or High Risk Early Breast Cancer), in HR-positive / HER2-negative breast cancer. The largest, NCT07349069, plans to enrol 5500 participants with primary completion expected 2030-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HRS-8080","url":"https://clinicaltrials.gov/search?intr=HRS-8080"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07354022","nct07024173","nct07349069","nct06222879","nct06679036","nct06555068","nct07389733","nct06167694"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hs-20089","kind":"drug","name":"HS-20089","aka":[],"tldr":"HS-20089 is an experimental antibody-drug conjugate from Hansoh BioMedical R&D in phase 3 trials for ovarian cancer and endometrial cancer, with its target not yet stated publicly.","summary":"HS-20089 is an antibody-drug conjugate developed by Hansoh BioMedical R&D. The sponsor describes its target as B7-H4, which OnCo does not yet have a target page for. The sponsor states: HS-20089 is a humanised IgG1 anti-B7-H4 monoclonal antibody conjugated to a topoisomerase I inhibitor payload via a protease-cleavable linker (average drug-to-antibody ratio of about 6), delivering the payload to B7-H4-expressing tumour cells, given intravenously every three weeks in ovarian and endometrial cancer. ClinicalTrials.gov describes the intervention as: All patients will receive intravenous HS-20089 once every three weeks (Q3W) until experiencing objective disease progression (except for study drug treatment beyond progression) or meeting other protocol-specified criteria of study treatmen. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06855069 (HS-20089 for Injection in Patients With Platinum-Resistant Recurrent Epithelial Ovarian Cancer), in ovarian cancer and endometrial cancer. The largest, NCT06855069, plans to enrol 468 participants with primary completion expected 2027-03-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HS-20089","url":"https://clinicaltrials.gov/search?intr=HS-20089"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian","endometrial"],"sections":[],"technologies":[],"targets":["b7h4"],"drugs":[],"companies":["hansoh-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06855069","nct06014190"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"HS-20089 is a humanised IgG1 anti-B7-H4 monoclonal antibody conjugated to a topoisomerase I inhibitor payload via a protease-cleavable linker (average drug-to-antibody ratio of about 6), delivering the payload to B7-H4-expressing tumour cells, given intravenously every three weeks in ovarian and endometrial cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hs-20093","kind":"drug","name":"HS-20093","aka":[],"tldr":"HS-20093 is an experimental investigational agent whose form is not stated in the registry from Hansoh BioMedical R&D in phase 3 trials for osteosarcoma, non-small-cell lung cancer and sarcomas, with its target not yet stated publicly.","summary":"HS-20093 is an investigational agent whose form is not stated in the registry developed by Hansoh BioMedical R&D and Jiangsu HengRui Medicine. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Participants in all subjucts will receive HS-20093 at 10mg/kg. It is the investigational product in 6 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06935409 (Study of HS-20093 Versus Gemcitabine in Combination With Docetaxel in Treatment of Osteosarcoma After Previous Second-line Treatment Failure) and NCT06498479 (ARTEMIS-008：HS-20093 Compared With Topotecan in Subjects With Relapsed Small Cell Lung Cancer), in osteosarcoma, non-small-cell lung cancer, sarcomas, prostate cancer and head and neck squamous cell carcinoma. The largest, NCT06498479, plans to enrol 460 participants with primary completion expected 2026-09-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HS-20093","url":"https://clinicaltrials.gov/search?intr=HS-20093"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["osteosarcoma","nsclc","sarcoma","prostate","head-and-neck","esophageal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hansoh-pharma","hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06935409","nct06498479","nct05830123","nct07230106","nct06007729","nct06112704"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hs-20117","kind":"drug","name":"HS-20117","aka":[],"tldr":"HS-20117 is an experimental bispecific antibody from Hansoh BioMedical R&D in phase 3 trials for non-small-cell lung cancer, aimed at EGFR and MET.","summary":"HS-20117 (PM1080) is a bispecific antibody developed by Hansoh BioMedical R&D. Its targets are EGFR and MET (the sponsor names EGFR x MET). The sponsor states: A fully-human EGFR-MET IgG1-like bispecific antibody. ClinicalTrials.gov describes the intervention as: Participants will receive HS-20117 once during cycle 1 and once every 2 weeks during subsequent cycles (The duration of each treatment cycle is 28 days). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06417008 (A Study of HS-20117 Combined With Aumolertinib in Participants With Advanced Non-Squamous Non-Small Cell Lung Cancer), in non-small-cell lung cancer. The largest, NCT06417008, plans to enrol 1080 participants with primary completion was scheduled for 2026-06-01 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of HS-20117","url":"https://clinicaltrials.gov/search?intr=PM1080"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["egfr","met"],"drugs":[],"companies":["hansoh-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06417008"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PM1080","modality":"bispecific antibody","mechanism":"A fully-human EGFR-MET IgG1-like bispecific antibody.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hst-1011","kind":"drug","name":"HST-1011","aka":["CBL-B inhibitor"],"tldr":"HST-1011 is a small-molecule inhibitor from HotSpot Therapeutics, Inc, in registered phase 2 trials for metastatic cancer.","summary":"HST-1011 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by HotSpot Therapeutics, Inc, in metastatic cancer. Described in the registry record as a cbl-b inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of HST-1011","url":"https://clinicaltrials.gov/search?intr=HST-1011"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hotspot-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05662397"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"HST-1011","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a cbl-b inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"human-normal-immunoglobulin","kind":"drug","name":"Human normal immunoglobulin (IVIg)","aka":["human normal immunoglobulin (IVIg)","human normal immunoglobulin","IVIg","intravenous immunoglobulin","Privigen","Kiovig"],"tldr":"Intravenous immunoglobulin is pooled antibody from many blood donors. People with chronic lymphocytic leukaemia or myeloma whose own antibody levels have collapsed, and who keep getting bacterial infections despite antibiotics, receive it as replacement.","summary":"Human normal immunoglobulin for intravenous use is authorised in the EU as Kiovig (authorised 19 January 2006; marketing authorisation holder Takeda Manufacturing Austria AG) and Privigen (authorised 24 April 2008; CHMP opinion 21 February 2008; marketing authorisation holder CSL Behring GmbH). The replacement-therapy indications include primary immunodeficiency syndromes and, in cancer care, hypogammaglobulinaemia with recurrent bacterial infections in patients with chronic lymphocytic leukaemia in whom prophylactic antibiotics have failed, and in plateau-phase multiple myeloma patients who have failed to respond to pneumococcal immunisation; the products also carry immunomodulation indications outside oncology.","status":"approved","asOf":"2026-09-22","links":[{"label":"EMA: Privigen (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/privigen"},{"label":"EMA: Kiovig (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/kiovig"}],"tags":["ema-register","supportive"],"related":[],"cancers":["cll","multiple-myeloma"],"sections":[],"technologies":["rejuv-tx-b-cell-aplasia-and-immunoglobulin"],"targets":[],"drugs":[],"companies":["csl","takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02205762"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo content wave 5 (EMA medicines register and EPAR pages)","editedOn":"2026-09-22"},"brand":"Privigen / Kiovig","modality":"Pooled human polyclonal IgG antibody preparation (intravenous immunoglobulin)","supportive":true,"mechanism":"Replacement of antibodies in patients whose own production has failed; in cancer care, for hypogammaglobulinaemia with recurrent bacterial infections in chronic lymphocytic leukaemia after prophylactic antibiotics have failed and in plateau-phase multiple myeloma after failure to respond to pneumococcal immunisation (indication wording, EMA).","approvals":[{"region":"EU","year":2006,"indication":"Replacement therapy in hypogammaglobulinaemia with recurrent bacterial infections in CLL (after failed antibiotic prophylaxis) and plateau-phase multiple myeloma (after failed pneumococcal immunisation); primary immunodeficiency","note":"Kiovig authorised 19 Jan 2006 (Takeda); Privigen 24 Apr 2008 (CSL Behring)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hydroxyurea","kind":"drug","name":"Hydroxyurea (hydroxycarbamide)","aka":[],"tldr":"Hydroxyurea is a cheap, decades-old pill that lowers high blood counts in polycythaemia vera and essential thrombocythaemia and is also the main drug for sickle cell disease.","summary":"First-line cytoreduction in high-risk PV and ET (reduces thrombosis; PT-1 trial superior to anagrelide in ET). Historic role in CML before imatinib. Also used in sickle cell disease and, less often, for leukocytosis in acute leukaemia. Concerns: leg ulcers, skin cancers, questionable leukaemogenicity.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Hydroxycarbamide","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=hydroxyurea"}],"tags":["gap-fill","generic"],"related":[],"cancers":["myeloproliferative-neoplasms","cml","polycythaemia-vera","essential-thrombocythaemia"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Hydrea / Droxia / Siklos","modality":"Oral ribonucleotide reductase inhibitor (cytoreductive)","mechanism":"Inhibits ribonucleotide reductase, depleting deoxyribonucleotides and arresting S-phase; lowers blood counts in myeloproliferative disease.","approvals":[{"region":"US","year":1967,"indication":"CML, head and neck cancer with radiotherapy, melanoma, ovarian cancer (historic labelling)"},{"region":"US","year":1998,"indication":"Sickle cell anaemia (Droxia)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"iah0968","kind":"drug","name":"IAH0968","aka":[],"tldr":"IAH0968 is an experimental small-molecule drug from SUNHO（China）BioPharmaceutical CO. in phase 3 trials for gastric & gastro-oesophageal junction cancer, aimed at HER2.","summary":"IAH0968 is a small-molecule drug developed by SUNHO（China）BioPharmaceutical CO.. Its target is HER2 (the sponsor names HER2). ClinicalTrials.gov describes the intervention as: Experimental：IAH0968 15 mg/kg on Day 1 of each 3-week cycle as an IV infusion. Drug: Oxaliplatin 130 mg/m\\^2 on Day 1 of each 3-week cycle over 2 hours as an IV infusion, administered as part of CAPOX chemotherapy regimen. Drug: Capecitabin. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06504732 (To Evaluate IAH0968 in Combination With CAPEOX in HER2-positive Gastric Cancer), in gastric & gastro-oesophageal junction cancer. The largest, NCT06504732, plans to enrol 574 participants with primary completion expected 2028-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IAH0968","url":"https://clinicaltrials.gov/search?intr=IAH0968"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["hutchmed"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06504732"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody (anti-HER2, per the sponsor's pipeline page; registered as a drug on ClinicalTrials.gov)","mechanism":"Small-molecule drug directed at HER2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ibandronic-acid","kind":"drug","name":"Ibandronic acid","aka":["Ibandronate","Boniva (osteoporosis formulation)","Bonviva"],"tldr":"Ibandronic acid (Bondronat) is a bisphosphonate available in Europe as a monthly infusion or daily tablet to prevent fractures and other bone complications in women with breast cancer that has spread to bone, and to treat cancer-related high calcium.","summary":"Ibandronic acid was authorised by the European Commission in June 1996 for tumour-induced hypercalcaemia and later for prevention of skeletal events (pathological fractures, bone complications requiring radiotherapy or surgery) in patients with breast cancer and bone metastases, on placebo-controlled trials of intravenous 6 mg every three to four weeks and oral 50 mg daily that reduced skeletal morbidity. It is the only bisphosphonate with an oral formulation licensed for bone metastases; in the US ibandronate (Boniva) is approved for osteoporosis only. The ZICE trial found oral ibandronate slightly less effective than zoledronic acid for skeletal events, so guidelines list it as an alternative. Renal toxicity is lower than with zoledronic acid; osteonecrosis of the jaw and oesophageal irritation with tablets are the recognised risks. Originated by Roche.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Ibandronic_acid","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/bondronat"}],"tags":["ema-list","supportive"],"related":["zoledronic-acid","denosumab"],"cancers":["breast-hr-positive"],"sections":[],"technologies":["bone-modifying-agents"],"targets":[],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02739594","nct00326820","nct00082927"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Bondronat","modality":"Nitrogen-containing bisphosphonate (intravenous and oral)","supportive":true,"mechanism":"Binds bone mineral and inhibits farnesyl pyrophosphate synthase in osteoclasts, suppressing bone resorption and lowering tumour-induced calcium release.","approvals":[{"region":"EU","year":1996,"indication":"Tumour-induced hypercalcaemia; prevention of skeletal events in breast cancer with bone metastases (Bondronat)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"iberdomide","kind":"drug","name":"Iberdomide","aka":[],"tldr":"Iberdomide is a far more potent successor to lenalidomide, now in phase 3 as post-transplant maintenance and in relapsed disease.","summary":"Iberdomide is a cereblon E3 ligase modulator (CELMoD) that binds cereblon with around 20-fold higher affinity than lenalidomide, degrading Ikaros and Aiolos more completely and so killing myeloma cells and stimulating T cells more potently. Because it acts through the same pathway with greater force, it is active in lenalidomide-refractory disease: in CC-220-MM-001 the doublet with dexamethasone gave an ORR of about 26%, higher in combinations. Its main development is as post-transplant maintenance, where the phase 3 EXCALIBER-Maintenance trial against lenalidomide met its primary endpoint according to BMS reports in 2026, and in relapsed disease, where EXCALIBER-RRMM (Iber-Dd versus Dara-Vd) is ongoing. It is a more powerful successor to lenalidomide aiming to keep myeloma in remission longer after transplant.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Iberdomide"}],"tags":[],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["protac-degrader","celmods"],"targets":[],"drugs":["lenalidomide"],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05827016","nct04975997","nct05583617","nct06892522","nct02773030"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zenbexus","code":"CC-220","modality":"CELMoD (cereblon E3 ligase modulator)","mechanism":"Binds cereblon with ~20-fold higher affinity than lenalidomide, degrading Ikaros/Aiolos more completely.","approvals":[{"region":"US","year":2026,"indication":"Multiple myeloma after at least one prior line including a proteasome inhibitor and an immunomodulatory agent, with subcutaneous daratumumab and dexamethasone (Zenbexus, label effective 13 August 2026)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-08-13","type":"accelerated-approval","region":"US","note":"FDA grants accelerated approval to iberdomide with daratumumab and hyaluronidase-fihj and dexamethasone for multiple myeloma The confirmatory requirement was still open 0.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-iberdomide-daratumumab-and-hyaluronidase-fihj-and-dexamethasone","indication":"In combination with daratumumab and hyaluronidase-fihj and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least 1 prior line of therapy including a proteasome inhibitor and an immunomodulatory agent"}]},{"id":"ibi310","kind":"drug","name":"IBI310","aka":[],"tldr":"IBI310 is an experimental investigational agent whose form is not stated in the registry from Innovent Biopharmaceutical Technology (Hangzhou) in phase 3 trials for hepatocellular carcinoma, with its target not yet stated publicly.","summary":"IBI310 is an investigational agent whose form is not stated in the registry developed by Innovent Biopharmaceutical Technology (Hangzhou). Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: 1 mg/kg intravenous infusion, administered on Day 1 of each 6-week treatment cycle. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07490262 (A Study to Evaluate the Efficacy and Safety of IBI310 and Sintilimab Combination Therapy in Patients With Hepatocellular Carcinoma as First-line Treatment), in hepatocellular carcinoma. The largest, NCT07490262, plans to enrol 680 participants with primary completion expected 2027-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IBI310","url":"https://clinicaltrials.gov/search?intr=IBI310"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hcc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["innovent"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07490262"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody (anti-CTLA-4, per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ibi343","kind":"drug","name":"IBI343","aka":[],"tldr":"IBI343 is an experimental investigational agent whose form is not stated in the registry from Innovent Biologics (Suzhou) in phase 3 trials for pancreatic ductal adenocarcinoma, aimed at Claudin 18.2.","summary":"IBI343 is an investigational agent whose form is not stated in the registry developed by Innovent Biologics (Suzhou). Its target is Claudin 18.2 (the sponsor names Claudin 18.2 (CLDN18.2)). ClinicalTrials.gov describes the intervention as: Subjects in the experimental arm will receive IBI343 6mg/kg intravenous infusion (IV) D1, Q3W in 3-week cycle. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07066098 (A Multicenter Study of IBI343 Monotherapy Versus Placebo in Subjects With Previously Treated, Claudin (CLDN) 18.2-positive, Pancreatic Cancer(G-HOPE-002)), in pancreatic ductal adenocarcinoma. The largest, NCT07066098, plans to enrol 201 participants with primary completion expected 2027-06-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IBI343","url":"https://clinicaltrials.gov/search?intr=IBI343"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":["innovent"],"institutions":[],"pathways":[],"terms":[],"trials":["g-hope-002","nct07483554"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["23 Sept 2026: the payload is not stated on any record OnCo reads (registry record, sponsor page as recorded here), so the open drug engine shows IBI343 under Claudin 18.2 with the payload class not recorded rather than guessing. IBI343 (Innovent), AZD4360 (AstraZeneca) and XNW27011 are three separate records."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Investigational agent whose form is not stated in the registry directed at Claudin 18.2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ibi354","kind":"drug","name":"IBI354","aka":[],"tldr":"IBI354 is an experimental antibody-drug conjugate from Innovent Biologics (Suzhou) in phase 3 trials for HER2-positive breast cancer and ovarian cancer, aimed at HER2.","summary":"IBI354 is an antibody-drug conjugate developed by Innovent Biologics (Suzhou) and Innovent Biopharmaceutical Technology (Hangzhou). Its target is HER2 (the sponsor names HER2). The sponsor states: IBI354 is a HER2-targeting antibody-drug conjugate (ADC), given as a 12 mg/kg IV infusion every three weeks, studied versus investigator's choice of chemotherapy in HER2-expressing tumours. ClinicalTrials.gov describes the intervention as: Recombinant Anti-HER2 monoclonal Antibody-Camptothecin derivative conjugate for injection. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07377643 (IBI354 With or Without Pertuzumab Versus Taxane, Trastuzumab and Pertuzumab in HER2-positive Metastatic Breast Cancer) and NCT06834672 (Study of IBI354 Versus Investigator's Choice of Chemotherapy in Patients With Platinum-resistant Ovarian, Primary Peritoneal, or Fallopian Tube Cancer), in HER2-positive breast cancer and ovarian cancer. The largest, NCT07377643, plans to enrol 540 participants with primary completion expected 2028-04-28. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IBI354","url":"https://clinicaltrials.gov/search?intr=IBI354"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-her2-positive","ovarian"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["innovent"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07377643","nct06834672","nct05636215"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"IBI354 is a HER2-targeting antibody-drug conjugate (ADC), given as a 12 mg/kg IV infusion every three weeks, studied versus investigator's choice of chemotherapy in HER2-expressing tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ibi363","kind":"drug","name":"IBI363","aka":[],"tldr":"IBI363 is an experimental fusion protein from Innovent Biologics (Suzhou) in phase 2 trials for melanoma, non-small-cell lung cancer and colorectal cancer, aimed at PD-1.","summary":"IBI363 is a fusion protein developed by Innovent Biologics (Suzhou). Its target is PD-1 (the sponsor names IL-2 pathway x PD-1). The sponsor states: A mutated IL-2 cytokine fused to an anti-PD-1 antibody, combining IL-2 pathway stimulation with PD-1 checkpoint blockade in a single molecule. ClinicalTrials.gov describes the intervention as: a mutated IL-2 cytokine fused to an anti-PD-1 antibody to combine IL-2 pathway stimulation with checkpoint blockade. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in melanoma, non-small-cell lung cancer, colorectal cancer and renal cell carcinoma. The largest, NCT06797297, plans to enrol 180 participants with primary completion was scheduled for 2026-03-31 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IBI363","url":"https://clinicaltrials.gov/search?intr=IBI363"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma","nsclc","colorectal","rcc"],"sections":[],"technologies":[],"targets":["pd1"],"drugs":[],"companies":["innovent"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07722494","nct06797297","nct06081920","nct06281678","nct07122687"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"fusion protein","mechanism":"A mutated IL-2 cytokine fused to an anti-PD-1 antibody, combining IL-2 pathway stimulation with PD-1 checkpoint blockade in a single molecule.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ibritumomab-tiuxetan","kind":"drug","name":"Ibritumomab tiuxetan","aka":["Y-90 ibritumomab","Zevalin therapeutic regimen"],"tldr":"Zevalin is an antibody carrying a radioactive isotope that seeks out CD20 on lymphoma cells. It treats follicular lymphoma that has relapsed and is given as a one-off consolidation after chemotherapy.","summary":"Ibritumomab tiuxetan was the first radioimmunotherapy approved by the FDA, in February 2002, for relapsed or refractory low-grade, follicular or transformed B-cell non-Hodgkin lymphoma including rituximab-refractory disease (Study 106-06: response in a majority of 54 rituximab-refractory patients), and in 2009 for consolidation after first-line chemotherapy in follicular lymphoma with a response (FIT trial: prolonged progression-free survival). The EU authorised Zevalin in 2004. Despite efficacy and a single-day treatment, use collapsed because of logistics between nuclear medicine and haematology, prolonged cytopenias and competition from rituximab maintenance and later agents; tositumomab (Bexxar) was discontinued in 2014. Severe cytopenias and infusion reactions carry boxed warnings.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Ibritumomab_tiuxetan","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ibritumomab%20tiuxetan"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/ibritumomabtiuxetan"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/zevalin"}],"tags":["nci-list"],"related":["rituximab"],"cancers":["follicular-lymphoma"],"sections":[],"technologies":["radioimmunotherapy","monoclonal-antibody"],"targets":["cd20"],"drugs":[],"companies":["acrotech-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01827605","nct00582166","nct01497275"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zevalin","modality":"Radioimmunotherapy (yttrium-90 labelled anti-CD20 antibody)","mechanism":"Murine anti-CD20 IgG1 (the parent of rituximab) chelated via tiuxetan to yttrium-90; beta emission delivers radiation to CD20-positive B cells and neighbouring tumour cells (crossfire), given after rituximab to clear circulating B cells.","approvals":[{"region":"US","year":2002,"indication":"Relapsed or refractory low-grade, follicular or transformed B-cell NHL including rituximab-refractory follicular lymphoma"},{"region":"US","year":2009,"indication":"Consolidation after first-line chemotherapy in previously untreated follicular NHL"},{"region":"EU","year":2004,"indication":"Rituximab-relapsed or refractory CD20-positive follicular lymphoma; first-line consolidation added 2008"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2002-02-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Relapsed or refractory low-grade follicular or transformed B-cell NHL other than rituximab-refractory follicular NHL"},{"date":"2009-09-03","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2002 converted to traditional approval 7.5 years after it was granted.","indication":"Relapsed or refractory low-grade follicular or transformed B-cell NHL other than rituximab-refractory follicular NHL"}]},{"id":"ibrutinib","kind":"drug","name":"Ibrutinib","aka":[],"tldr":"The pill that ended chemotherapy for most CLL. It blocks the survival signal B cells depend on, and it was the first drug to beat chemoimmunotherapy in nearly every CLL setting.","summary":"Approved 2014 (relapsed CLL, RESONATE), 2016 (frontline, RESONATE-2), and with venetoclax as fixed-duration therapy (GLOW, CAPTIVATE; EU 2022). Also mantle cell lymphoma (withdrawn in the US 2023), Waldenström, marginal zone (withdrawn), and chronic GVHD. Off-target inhibition of EGFR, TEC, and CSK causes atrial fibrillation (~10-16%), hypertension, bleeding, and arthralgia; head-to-head trials (ELEVATE-RR, ALPINE) showed acalabrutinib and zanubrutinib are better tolerated, and zanubrutinib more effective, so ibrutinib is now second choice where alternatives exist.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Ibrutinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ibrutinib"},{"label":"Tempero et al., RESOLVE (Annals of Oncology 2021)","url":"https://doi.org/10.1016/j.annonc.2021.01.070"},{"label":"Davis et al., Nature 2010: chronic active B-cell receptor signalling in diffuse large B-cell lymphoma","url":"https://doi.org/10.1038/nature08638"},{"label":"Woyach et al., N Engl J Med 2014: BTK C481S and PLCG2 resistance mutations under ibrutinib","url":"https://doi.org/10.1056/NEJMoa1400029"}],"tags":[],"related":[],"cancers":["cll","dlbcl","pancreatic","metastatic-pdac"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["btk"],"drugs":[],"companies":["abbvie","johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["resonate","captivate","glow","alpine","nct01804686","nct03960840","nct05283720","nct03379428","nct02477696","nct04509700","nct05254743","nct05963074","nct04494503","nct04662255","resolve"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: RESOLVE (424 randomised) found no overall survival benefit (9.7 against 10.8 months) and shorter progression-free survival (5.3 against 6.0 months) when ibrutinib was added to nab-paclitaxel and gemcitabine.","Lymphoma biology: the drug exists because knocking down BTK killed activated B-cell-like lymphoma cells with wild-type CARD11 and left other lymphomas alone, which is what identified chronic active B-cell receptor signalling as a pathogenetic mechanism (Davis 2010). Resistance is on-target in most patients, through BTK C481S, and bypass in a minority, through gain-of-function PLCG2 changes below the kinase that no BTK inhibitor can reach (Woyach 2014)."],"brand":"Imbruvica","modality":"Small-molecule covalent BTK inhibitor (first generation)","mechanism":"Covalent binding to cysteine 481 in the BTK active site irreversibly blocks BCR signalling; also inhibits ITK, TEC, EGFR (off-target).","approvals":[{"region":"US","year":2013,"indication":"Relapsed mantle cell lymphoma (accelerated); first BTK inhibitor"},{"region":"US","year":2014,"indication":"Relapsed CLL (RESONATE); del(17p) CLL"},{"region":"US","year":2016,"indication":"First-line CLL (RESONATE-2)"},{"region":"EU","year":2014,"indication":"Relapsed mantle cell lymphoma; CLL after one prior therapy or with del(17p)/TP53 mutation"},{"region":"EU","year":2022,"indication":"Fixed-duration ibrutinib + venetoclax, first-line CLL (GLOW, CAPTIVATE)"}],"mechanismSteps":["B-cell receptor engagement activates BTK in the CLL cell","Ibrutinib binds C481 covalently and permanently inactivates that BTK molecule","PLCγ2, NF-κB, and chemokine signalling stop; cells lose adhesion","CLL cells are flushed from lymph nodes into blood (transient lymphocytosis) and die over months","Continuous daily dosing is required because new BTK is synthesised"],"dosing":{"route":"Oral","schedule":"420 mg once daily continuously until progression (CLL); 560 mg in MCL","monitoring":"Atrial fibrillation, hypertension, bleeding (hold around surgery), infections, cytopenias","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Imbruvica"},"toxicity":[{"event":"Atrial fibrillation","anyGradePct":16,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Imbruvica","note":"Pooled long-term CLL data; 5% grade ≥3"},{"event":"Hypertension","anyGradePct":42,"note":"Cumulative over years"},{"event":"Major haemorrhage","anyGradePct":4},{"event":"Diarrhoea","anyGradePct":50},{"event":"Arthralgia","anyGradePct":30}],"access":[],"regulatoryEvents":[{"date":"2013-11-13","type":"accelerated-approval","region":"US","note":"Mantle cell lymphoma (accelerated); first BTK inhibitor","indication":"Adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy"},{"date":"2014-02-12","type":"accelerated-approval","region":"US","note":"Relapsed CLL","indication":"Chronic lymphocytic leukemia after at least one prior therapy"},{"date":"2014-07-28","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2014 converted to traditional approval 0.5 years after it was granted.","indication":"Chronic lymphocytic leukemia after at least one prior therapy"},{"date":"2016-03-04","type":"approval","region":"US","note":"First-line CLL"},{"date":"2017-01-18","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adult patients with marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy"},{"date":"2023-04-06","type":"withdrawal","region":"US","note":"MCL and MZL indications voluntarily withdrawn after confirmatory trials missed"},{"date":"2023-05-18","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 9.5 years after its accelerated approval.","indication":"Adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy"},{"date":"2023-05-18","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 6.3 years after its accelerated approval.","indication":"Adult patients with marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy"}]},{"id":"icotinib","kind":"drug","name":"Icotinib","aka":[],"tldr":"Icotinib, approved in 2011, was the first cancer drug invented and developed in China, and it showed that a domestic lung cancer pill could match a Western one head to head.","summary":"Betta Pharmaceuticals' icotinib was approved by the then SFDA in June 2011 for advanced NSCLC after chemotherapy, on the ICOGEN trial that showed non-inferiority to gefitinib (Lancet Oncology 2013). First-line EGFR-mutant approval followed in 2014, and the EVIDENCE trial (Lancet Respiratory Medicine 2021) supported adjuvant use after resection of stage II to IIIA EGFR-mutant disease. It was the first drug in the 2016 pilot of national price negotiation, taking a large cut to enter reimbursement, the template for the later NRDL rounds.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Icotinib","links":[{"label":"Betta Pharmaceuticals","url":"https://www.bettapharma.com/"},{"label":"ICOGEN (Lancet Oncol 2013)","url":"https://doi.org/10.1016/S1470-2045(13)70355-3"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["betta"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct06041776"],"people":[],"bottlenecks":[],"keyPapers":["paper-shi-lancet-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Conmana","code":"BPI-2009H","modality":"Small-molecule first-generation EGFR TKI","mechanism":"Reversible ATP-competitive inhibitor of EGFR tyrosine kinase, structurally related to erlotinib.","approvals":[{"region":"China","year":2011,"indication":"Advanced NSCLC after failure of at least one chemotherapy regimen (ICOGEN)"},{"region":"China","year":2014,"indication":"First-line EGFR-mutant NSCLC"},{"region":"China","year":2021,"indication":"Adjuvant treatment of resected stage II to IIIA EGFR-mutant NSCLC (EVIDENCE)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"icp-248","kind":"drug","name":"ICP-248","aka":[],"tldr":"ICP-248 is an experimental small-molecule drug from Beijing InnoCare Pharma Tech in phase 3 trials for mantle cell lymphoma and chronic lymphocytic leukaemia, with its target not yet stated publicly.","summary":"ICP-248 is a small-molecule drug developed by Beijing InnoCare Pharma Tech. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Eligible patients will receive ICP-248 orally as per the protocol，once daily for every 28 days as one treatment cycle. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06378138 (ICP-248 in Combination With Orelabrutinib in Treatment-naïve Chronic Lymphocytic Leukaemia/Small Lymphocytic Lymphoma (APEX-03)), in mantle cell lymphoma and chronic lymphocytic leukaemia. The largest, NCT06378138, plans to enrol 226 participants with primary completion expected 2030-11-25. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ICP-248","url":"https://clinicaltrials.gov/search?intr=ICP-248"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["mantle-cell-lymphoma","cll"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["innocare"],"institutions":[],"pathways":[],"terms":[],"trials":["apex-03","nct07082686"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"idarubicin","kind":"drug","name":"Idarubicin","aka":[],"tldr":"Idarubicin is an anthracycline chemotherapy approved in 1990 for adult acute myeloid leukaemia, given with cytarabine in 3+7 induction and FLAG-Ida salvage and in acute promyelocytic leukaemia protocols. Randomised trials showed more complete remissions than with daunorubicin, which is why centres prefer it; heart damage, marrow suppression and extravasation injury are the class risks.","summary":"Idarubicin was approved in 1990 for adult acute myeloid leukaemia as part of combination induction. Four randomised trials totalling 823 newly diagnosed patients (Memorial Sloan Kettering, Southeastern Cancer Study Group, US multicentre and GIMEMA) compared idarubicin with daunorubicin, each with cytarabine, and showed higher complete remission rates and, in some, longer survival with idarubicin; it is a standard induction anthracycline (for example in the 3+7 and the FLAG-Ida salvage regimens) and in acute promyelocytic leukaemia protocols with all-trans retinoic acid. Cardiotoxicity, severe myelosuppression and extravasation injury are the class risks.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Idarubicin","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=idarubicin"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/idarubicinhydrochloride"}],"tags":["nci-list","generic"],"related":["daunorubicin","cytarabine"],"cancers":["aml"],"sections":[],"technologies":["cytotoxic-chemotherapy","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03591510"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Idamycin PFS","modality":"Anthracycline (topoisomerase II inhibitor)","mechanism":"4-demethoxy analogue of daunorubicin; more lipophilic, with an active metabolite (idarubicinol); intercalates DNA and inhibits topoisomerase II.","approvals":[{"region":"US","year":1990,"indication":"Adult acute myeloid leukaemia, as a component of combination induction chemotherapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"idecabtagene-vicleucel","kind":"drug","name":"Idecabtagene vicleucel","aka":[],"tldr":"Idecabtagene vicleucel was the first myeloma CAR-T (2021) and is now approved after two prior lines based on KarMMa-3.","summary":"Idecabtagene vicleucel is an autologous CAR-T in which a lentiviral vector adds a murine scFv anti-BCMA receptor with 4-1BB costimulation to the patient's T cells, infused once after lymphodepletion. It was the first myeloma CAR-T, approved in 2021 after at least 4 lines on KarMMa (ORR 73%, CR 33%). KarMMa-3 showed PFS 13.3 versus 4.4 months (HR 0.49) against standard regimens in triple-class-exposed patients, leading to approval after at least 2 lines in April 2024; OS was not significantly different unadjusted, and whether crossover-adjusted analyses favouring ide-cel should count remains debated. Cytokine release syndrome occurred in 88% (5% grade 3 or higher) and neurotoxicity in 15%; the REMS was removed in 2025. BMS and 2seventy bio developed it. It is a one-time infusion of engineered immune cells that tripled the time without progression in heavily pretreated myeloma.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Idecabtagene_vicleucel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Idecabtagene%20vicleucel"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-relapsed-refractory"],"sections":[],"technologies":["car-t"],"targets":["bcma"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":["crs","icans","crossover"],"trials":["karmma-3","nct06045806"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Abecma","code":"ide-cel","modality":"CAR-T (BCMA)","mechanism":"Murine scFv anti-BCMA CAR with 4-1BB costimulation; lentiviral.","approvals":[{"region":"US","year":2021,"indication":"Relapsed/refractory myeloma after ≥4 lines"},{"region":"US","year":2024,"indication":"After ≥2 lines including IMiD, PI, and anti-CD38"}],"mechanismSteps":[],"dosing":{"route":"IV single infusion","schedule":"300-510 × 10⁶ CAR+ T cells after fludarabine/cyclophosphamide lymphodepletion","monitoring":"CRS, ICANS, prolonged cytopenias, infections; REMS removed 2025"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":88,"grade3PlusPct":5,"note":"KarMMa-3"},{"event":"Neurotoxicity","anyGradePct":15,"grade3PlusPct":3},{"event":"Neutropenia","grade3PlusPct":76}],"access":[],"regulatoryEvents":[]},{"id":"idelalisib","kind":"drug","name":"Idelalisib","aka":[],"tldr":"Idelalisib was the first PI3K inhibitor for blood cancers; it is effective in CLL with rituximab but so toxic (colitis, hepatitis, pneumonitis, infections) that the class has largely been abandoned.","summary":"Approved 2014 (CLL with rituximab; FL and SLL accelerated). Trials in earlier lines were halted in 2016 over fatal infections and toxicity; the FL/SLL indications were withdrawn in 2022. Remains an option in relapsed CLL after BTK and BCL2 inhibitors. Its history shaped FDA scepticism of PI3K inhibitors (2022 ODAC).","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Idelalisib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=idelalisib"}],"tags":["gap-fill"],"related":[],"cancers":["cll","follicular-lymphoma","cll-relapsed"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["pik3ca"],"drugs":[],"companies":["gilead"],"institutions":[],"pathways":["pi3k-akt-mtor"],"terms":[],"trials":["nct06846671","nct02970318"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zydelig","modality":"Small-molecule PI3Kδ inhibitor","mechanism":"Selective inhibition of the delta isoform of PI3K, blocking B-cell receptor-driven survival signalling in malignant B cells.","approvals":[{"region":"US","year":2014,"indication":"Relapsed CLL with rituximab; relapsed FL and SLL (accelerated, withdrawn 2022)"},{"region":"EU","year":2014,"indication":"CLL, FL"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2014-07-23","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"For the treatment of relapsed follicular B-cell Non-Hodgkin Lymphoma (FL) in patients who have received at least 2 prior systemic therapies and relapsed small lymphocytic lymphoma (SLL) in patients who have received at least 2 prior systemic therapies"},{"date":"2022-02-18","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 7.6 years after its accelerated approval.","indication":"For the treatment of relapsed follicular B-cell Non-Hodgkin Lymphoma (FL) in patients who have received at least 2 prior systemic therapies and relapsed small lymphocytic lymphoma (SLL) in patients who have received at least 2 prior systemic therapies"}]},{"id":"idrx-42","kind":"drug","name":"IDRX-42","aka":["velzatinib - subject to USAN approval"],"tldr":"IDRX-42 is an experimental small-molecule drug from GlaxoSmithKline in phase 3 trials for gastrointestinal stromal tumour, with its target not yet stated publicly.","summary":"IDRX-42 is a small-molecule drug developed by GlaxoSmithKline. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07218926 (A Study of IDRX-42 (GSK6042981) Versus (vs) Sunitinib in Participants With Gastrointestinal Stromal Tumours After Imatinib Therapy), in gastrointestinal stromal tumour. The largest, NCT07218926, plans to enrol 450 participants with primary completion expected 2028-05-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IDRX-42","url":"https://clinicaltrials.gov/search?intr=IDRX-42"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gist"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07218926"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ifinatamab-deruxtecan","kind":"drug","name":"Ifinatamab deruxtecan","aka":[],"tldr":"Ifinatamab deruxtecan is a B7-H3 ADC showing some of the best response rates ever seen in relapsed small-cell lung cancer.","summary":"Ifinatamab deruxtecan is an anti-B7-H3 IgG1 antibody carrying the topoisomerase I inhibitor DXd at a drug-to-antibody ratio of about 8 through a cleavable GGFG linker; B7-H3 is present on most small-cell lung cancers and the permeable payload kills neighbouring cells too. Daiichi Sankyo and Merck are developing it for extensive-stage SCLC after platinum, where the phase 2 IDeate-Lung01 trial reported an objective response rate around 55 percent at the selected 12 mg/kg every-3-weeks dose, among the best seen in relapsed SCLC. The phase 3 IDeate-Lung02 trial versus topotecan is ongoing, and the FDA granted Breakthrough Therapy designation in 2024. Nausea, anaemia, decreased appetite and neutropenia are common, and interstitial lung disease is monitored. It is also being studied in prostate and oesophageal cancer, and offers a lung cancer with few new options a genuinely new mechanism.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Ifinatamab deruxtecan","url":"https://clinicaltrials.gov/search?intr=I-DXd"}],"tags":[],"related":[],"cancers":["sclc","prostate","extensive-stage-sclc"],"sections":[],"technologies":["adc"],"targets":["b7h3"],"drugs":[],"companies":["daiichi-sankyo","merck"],"institutions":[],"pathways":[],"terms":["ggfg"],"trials":["nct06925737","nct06780137","nct05280470","nct04471727","nct06362252","nct07227597","nct04145622","nct06330064","nct07630974","nct06863272","nct06644781","nct06780085"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"I-DXd, DS-7300","modality":"ADC","payload":"DXd, DAR ~8","linker":"GGFG cleavable","mechanism":"Anti-B7-H3 IgG1 with DXd.","approvals":[],"mechanismSteps":["Antibody binds B7-H3 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","DXd is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"12 mg/kg every 3 weeks (IDeate-Lung01 selected dose)","monitoring":"ILD, blood counts, infusion reactions","source":"https://clinicaltrials.gov/study/NCT05280470"},"toxicity":[{"event":"Nausea"},{"event":"Anaemia"},{"event":"Decreased appetite"},{"event":"Neutropenia"},{"event":"ILD/pneumonitis"}],"access":[{"country":"US","reimbursement":"Investigational (phase 3 IDeate-Lung02)","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-08","type":"designation","region":"US","note":"Breakthrough Therapy designation, extensive-stage SCLC after platinum","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"ifosfamide","kind":"drug","name":"Ifosfamide","aka":[],"tldr":"Ifosfamide is an alkylating chemotherapy partnered with doxorubicin in sarcoma and with etoposide in Ewing sarcoma, given with a bladder-protecting drug.","summary":"Ifosfamide is an alkylating prodrug activated by CYP2B6 and CYP3A4 to a mustard that crosslinks DNA; its chloroacetaldehyde metabolite causes the characteristic encephalopathy and nephrotoxicity, and mesna uroprotection is mandatory to prevent haemorrhagic cystitis. Approved in 1988 for germ cell testicular cancer, it is standard off-label as the partner of doxorubicin in AIM for soft-tissue sarcoma, in MAP-IE for osteosarcoma, and with etoposide in VDC/IE for Ewing sarcoma, the regimen established by INT-0091. Febrile neutropenia occurred in 46% of patients on the AIM arm of EORTC 62012, encephalopathy in about 10% (treated with methylene blue) and haemorrhagic cystitis in about 5% even with mesna. It is a powerful alkylator whose use depends on protecting the bladder, kidneys and brain from its by-products.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Ifosfamide","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ifosfamide"}],"tags":[],"related":["mesna"],"cancers":["sarcoma","extremity-soft-tissue-sarcoma","undifferentiated-pleomorphic-sarcoma","malignant-peripheral-nerve-sheath-tumour","cns-germ-cell-tumours","node-positive-penile-cancer"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["euramos-1","nct06230224","nct05533775","nct06277154"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ifex","modality":"Cytotoxic chemotherapy (alkylating agent)","mechanism":"Prodrug activated by CYP2B6/3A4 to an alkylating mustard causing DNA crosslinks; chloroacetaldehyde metabolite causes neuro- and nephrotoxicity.","approvals":[{"region":"US","year":1988,"indication":"Germ cell testicular cancer; widely used off-label in sarcoma"}],"mechanismSteps":[],"dosing":{"route":"IV","schedule":"9-10 g/m2 per cycle over 3-5 days with mesna (sarcoma); 1.8 g/m2 × 5 days with etoposide (Ewing)","monitoring":"Urinalysis for haematuria, renal function, encephalopathy (methylene blue), counts"},"toxicity":[{"event":"Febrile neutropenia (with doxorubicin)","grade3PlusPct":46,"note":"EORTC 62012 AIM arm"},{"event":"Encephalopathy","anyGradePct":10},{"event":"Haemorrhagic cystitis","anyGradePct":5,"note":"with mesna"}],"access":[],"regulatoryEvents":[]},{"id":"ifupinostat","kind":"drug","name":"Ifupinostat","aka":["Ifupinostat Hydrochloride","BEBT-908 for Injection"],"tldr":"Ifupinostat is an experimental investigational agent whose form is not stated in the registry from BeBetter Med in phase 3 trials for diffuse large B-cell lymphoma, with its target not yet stated publicly.","summary":"Ifupinostat (BEBT-908, CUDC-908) is an investigational agent whose form is not stated in the registry developed by BeBetter Med. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Ifupinostat Hydrochloride for Injection,dosage of administration:18.5mg/m\\^2,frequency and duration of administration:Medication is administered on days 1, 3, 5, 8, 10, and 12 of each cycle,with a 21-day or 42-day cycle duration. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06792253 (Study Comparing BEBT-908 Combined With R to SOC for the Treatment of Relapsed/Refractory Diffuse Large B-Cell Lymphoma), in diffuse large B-cell lymphoma. The largest, NCT06792253, plans to enrol 416 participants with primary completion expected 2028-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Ifupinostat","url":"https://clinicaltrials.gov/search?intr=BEBT-908"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["bebetter-med"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06792253"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"BEBT-908, CUDC-908","modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"illuccix","kind":"drug","name":"Illuccix (kit for Ga-68 gozetotide)","aka":[],"tldr":"Telix's ready-to-label kit that lets any nuclear medicine department make the gallium PSMA scan for prostate cancer.","summary":"Illuccix was approved by the FDA in December 2021 for PET imaging of PSMA-positive lesions in men with suspected metastasis who are candidates for definitive therapy and in men with suspected recurrence based on rising PSA, and in 2025 the label was extended to selecting patients for PSMA radioligand therapy. Gozellix, a second-generation kit with longer post-labelling shelf life, was approved in 2025. Illuccix is also registered in Australia and several other markets. The kit format made PSMA PET available to centres without access to the cyclotron-produced 18F tracers.","status":"approved","asOf":"2026-09-24","links":[{"label":"PSMA-PreRP (JAMA Oncology 2021)","url":"https://doi.org/10.1001/jamaoncol.2021.3771"},{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d4643b31-9b4f-673f-e053-2a95a90a559d"}],"tags":["test"],"related":["ga68-psma-11","locametz","pylarify"],"cancers":["prostate"],"sections":[],"technologies":["psma-pet","pet-ct"],"targets":["psma"],"drugs":["pluvicto"],"companies":["telix"],"institutions":[],"pathways":[],"terms":["biochemical-recurrence","theranostics"],"trials":["psma-prerp"],"people":[],"bottlenecks":[],"keyPapers":["paper-psma-prerp-hope-jama-oncol-2021"],"journals":[],"dependsOn":[],"notes":["What a result means: PSMA-avid spots on the scan indicate prostate cancer deposits and decide between local treatment, systemic therapy or radioligand therapy."],"brand":"Illuccix / Gozellix","modality":"PET imaging agent kit (PSMA, gallium-68)","mechanism":"Kit for on-site labelling of PSMA-11 (gozetotide) with gallium-68 from a generator or cyclotron; the tracer binds PSMA on prostate cancer cells for PET imaging.","approvals":[{"region":"US","year":2021,"indication":"PSMA PET imaging in prostate cancer (initial staging with suspected metastasis; suspected recurrence)"},{"region":"Australia","year":2021,"indication":"PSMA PET imaging in prostate cancer"},{"region":"US","year":2025,"indication":"Gozellix kit; selection of patients for PSMA-targeted radioligand therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ima203","kind":"drug","name":"IMA203","aka":["anzutresgene autoleucel","anzu-cel"],"tldr":"IMA203 is an experimental TCR-T cell therapy from Immatics US in phase 3 trials for melanoma, aimed at PRAME.","summary":"IMA203 is a TCR-T cell therapy developed by Immatics US. Its target is PRAME (the sponsor names PRAME). The sponsor states: An autologous, genetically engineered T-cell receptor (TCR)-T cell therapy directed against PRAME-expressing tumour cells, given as a one-time infusion followed by adjunctive low-dose IL-2 therapy for up to 10 days. ClinicalTrials.gov describes the intervention as: one-time administration of IMA203, and adjunctive therapy with low dose interleukin (IL)-2 for up to 10 days, starting approximately 24 h after IMA203 infusion, optional bridging therapy. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06743126 (SUPRAME-ACTengine® IMA203 vs. Investigator's Choice of Treatment in Previously Treated, Unresectable or Metastatic Cutaneous Melanoma), in melanoma. The largest, NCT06743126, plans to enrol 360 participants with primary completion expected 2028-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IMA203","url":"https://clinicaltrials.gov/search?intr=IMA203"},{"label":"Sponsor pipeline page","url":"https://www.immatics.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":[],"targets":["prame"],"drugs":[],"companies":["immatics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06743126"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"TCR-T cell therapy","mechanism":"An autologous, genetically engineered T-cell receptor (TCR)-T cell therapy directed against PRAME-expressing tumour cells, given as a one-time infusion followed by adjunctive low-dose IL-2 therapy for up to 10 days.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"imatinib","kind":"drug","name":"Imatinib","aka":["STI571","STI-571","Glivec"],"tldr":"The drug that started the targeted therapy era in 2001, turning chronic myeloid leukaemia into a manageable condition with near-normal life expectancy.","summary":"Imatinib is an ATP-competitive inhibitor of the ABL, KIT and PDGFR kinases, taken as 400 mg once daily in chronic-phase CML and as adjuvant therapy for GIST, with 600-800 mg for advanced disease. Approved in 2001 for chronic myeloid leukaemia and 2002 for KIT-positive gastrointestinal stromal tumours, it started the targeted therapy era: in IRIS, 10-year overall survival was about 83%, turning CML into a manageable condition with near-normal life expectancy. Three years of adjuvant imatinib is standard after high-risk GIST resection, and it is also used in rare kinase-driven tumours. It is now generic. Resistance mutations such as T315I led to later ABL inhibitors, and whether patients in deep remission can stop safely is an active question. For a newcomer: the proof that a cancer could be switched off by blocking one enzyme.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Imatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Imatinib"}],"tags":[],"related":["american-ginseng-fatigue","st-johns-wort-interaction","kit-d816v","bcr-abl1-transcript"],"cancers":["sarcoma","cll","cml-chronic-phase","gist-kit-exon-11","all-paediatric-ph-positive","dermatofibrosarcoma-protuberans","mucosal-melanoma","acral-melanoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["kit"],"drugs":[],"companies":["novartis","natco"],"institutions":[],"pathways":["bcr-abl1-signalling"],"terms":[],"trials":["nct07406633","nct04971226","nct03459534"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Gleevec","modality":"Small-molecule kinase inhibitor (BCR-ABL, KIT, PDGFRA)","mechanism":"ATP-competitive inhibitor of ABL, KIT, PDGFR.","approvals":[{"region":"US","year":2001,"indication":"Chronic myeloid leukaemia"},{"region":"US","year":2002,"indication":"KIT+ GIST"}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of BCR-ABL, KIT, and PDGFRA","Phosphorylation of downstream substrates stops","Binds the inactive kinase conformation; CML cells lose their sole driver","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"400 mg once daily (chronic-phase CML, GIST adjuvant 3 years); 600-800 mg for advanced disease","modifications":"Hold for grade 3 neutropenia or hepatotoxicity","monitoring":"Blood counts, LFTs, BCR-ABL PCR every 3 months in CML, fluid retention","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Oedema"},{"event":"Nausea"},{"event":"Muscle cramps"},{"event":"Diarrhoea"},{"event":"Rash"},{"event":"Fatigue"},{"event":"Neutropenia"}],"access":[{"country":"US","reimbursement":"Medicare Part D; generic since 2016 (list price fell >90%)","generic":true,"source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NHS standard; generic","generic":true,"asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2001-05-10","type":"accelerated-approval","region":"US","note":"Chronic myeloid leukaemia: first targeted kinase inhibitor, approved in under 3 months","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"CML in blast crisis, accelerated phase, or in chronic phase after failure of of interferon-alpha"},{"date":"2002-02-01","type":"accelerated-approval","region":"US","note":"KIT+ gastrointestinal stromal tumour","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"KIT (CD117) positive unresectable and/or metastatic GIST"},{"date":"2002-12-20","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Newly diagnosed adults with Philadelphia chromosome positive CML"},{"date":"2003-05-20","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Pediatric patients with recurrent PH+ chronic phase CML after stem cell transplant or who are resistant to interferon alpha therapy"},{"date":"2003-12-08","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2001 converted to traditional approval 2.6 years after it was granted.","indication":"CML in blast crisis, accelerated phase, or in chronic phase after failure of of interferon-alpha"},{"date":"2006-09-27","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Newly diagnosed Philadelphia positive CML in pediatric patients"},{"date":"2006-09-27","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2003 converted to traditional approval 3.4 years after it was granted.","indication":"Pediatric patients with recurrent PH+ chronic phase CML after stem cell transplant or who are resistant to interferon alpha therapy"},{"date":"2008-09-26","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2002 converted to traditional approval 6.7 years after it was granted.","indication":"KIT (CD117) positive unresectable and/or metastatic GIST"},{"date":"2008-12-19","type":"accelerated-approval","region":"US","note":"Adjuvant GIST","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adjuvant treatment of adults following complete gross resection of KIT (CD117) positive gastrointestinal stromal tumors (GIST)"},{"date":"2009-05-27","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2002 converted to traditional approval 6.4 years after it was granted.","indication":"Newly diagnosed adults with Philadelphia chromosome positive CML"},{"date":"2011-04-01","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2006 converted to traditional approval 4.5 years after it was granted.","indication":"Newly diagnosed Philadelphia positive CML in pediatric patients"},{"date":"2012-01-31","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2008 converted to traditional approval 3.1 years after it was granted.","indication":"Adjuvant treatment of adults following complete gross resection of KIT (CD117) positive gastrointestinal stromal tumors (GIST)"},{"date":"2016-02-01","type":"approval","region":"US","note":"First US generic imatinib launched","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"imetelstat","kind":"drug","name":"Imetelstat","aka":[],"tldr":"Imetelstat is a lipid-linked oligonucleotide that binds the RNA template of telomerase, the enzyme cancer cells use to stay immortal, and it is the first telomerase inhibitor approved for any cancer. In lower-risk myelodysplastic syndromes it freed 40% of transfusion-dependent patients from transfusions for at least eight weeks versus 15% on placebo, once growth factors have failed.","summary":"Imetelstat is a 13-mer lipid-conjugated oligonucleotide that binds the RNA template of telomerase (hTR), inhibiting the enzyme in malignant clones that depend on high telomerase activity; it is the first telomerase inhibitor approved for any cancer. In the IMerge phase 3 trial (2024), 40% of patients with lower-risk transfusion-dependent myelodysplastic syndromes achieved 8-week transfusion independence versus 15% with placebo and reductions in mutant allele burden suggested disease modification. It was approved in the US in 2024 for low- to intermediate-1-risk MDS with transfusion-dependent anaemia after erythropoiesis-stimulating agents fail, and in the EU in 2025. The IMpactMF phase 3 trial in JAK-inhibitor-refractory myelofibrosis tests whether it extends overall survival. For a newcomer: a drug that attacks the enzyme cancer cells use to stay immortal.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Imetelstat","links":[{"label":"IMerge (Lancet 2024)","url":"https://doi.org/10.1016/S0140-6736(23)01724-5"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=imetelstat"}],"tags":["gap-fill"],"related":[],"cancers":["mds","myeloproliferative-neoplasms","mds-lower-risk"],"sections":[],"technologies":["antisense-sirna","transfusion-support"],"targets":[],"drugs":[],"companies":["geron"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05583552","impactmf"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Rytelo","modality":"Telomerase inhibitor (oligonucleotide)","mechanism":"13-mer lipid-conjugated oligonucleotide that binds the RNA template of telomerase (hTR), inhibiting telomerase in malignant clones with high telomerase activity.","approvals":[{"region":"US","year":2024,"indication":"Low- to intermediate-1-risk MDS with transfusion-dependent anaemia after ESA failure"},{"region":"EU","year":2025,"indication":"Lower-risk MDS transfusion-dependent anaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"imiquimod","kind":"drug","name":"Imiquimod","aka":[],"tldr":"Imiquimod (Aldara) is a cream that provokes the skin's own immune system to clear superficial basal cell carcinomas and the sun-damage patches (actinic keratoses) that can turn into skin cancer.","summary":"Imiquimod 5% cream was approved in 1997 for external genital warts and in 2004 for actinic keratosis and for superficial basal cell carcinoma up to 2 cm in immunocompetent adults when surgery is less appropriate, on vehicle-controlled trials with histological clearance in about three-quarters of treated sBCC lesions. Zyclara 2.5% and 3.75% (2010) offers a shorter field regimen for actinic keratosis. The EU authorised Aldara in 1998 with the same three indications. It is applied five times a week for six weeks for sBCC; local inflammation, erosion and flu-like symptoms are expected and predict response. Long-term recurrence is higher than after excision, so it is reserved for low-risk lesions.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Imiquimod","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=imiquimod"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/imiquimod"},{"label":"EPAR (Aldara)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/aldara"},{"label":"Bath-Hextall et al., surgical excision versus imiquimod 5% cream for nodular and superficial basal-cell carcinoma, SINS (Lancet Oncology 2014)","url":"https://doi.org/10.1016/S1470-2045(13)70530-8"},{"label":"Williams et al., surgery versus 5% imiquimod for nodular and superficial basal cell carcinoma, 5-year results of the SINS randomised controlled trial (J Invest Dermatol 2017)","url":"https://doi.org/10.1016/j.jid.2016.10.019"},{"label":"Jansen et al., five-year results of a randomised controlled trial comparing photodynamic therapy, topical imiquimod and topical 5-fluorouracil in superficial basal cell carcinoma (J Invest Dermatol 2018)","url":"https://doi.org/10.1016/j.jid.2017.09.033"},{"label":"Thomson et al., interventions for basal cell carcinoma of the skin: Cochrane review of 52 randomised trials and 6,690 participants (2020)","url":"https://doi.org/10.1002/14651858.CD003412.pub3"}],"tags":["nci-list"],"related":[],"cancers":["basal-cell-carcinoma","cutaneous-scc","skin-cancer"],"sections":[],"technologies":["chemoprevention"],"targets":["tlr7"],"drugs":[],"companies":["bausch-health"],"institutions":[],"pathways":[],"terms":["histology","topical-and-destructive-treatment-bcc"],"trials":["sins-trial","mal-pdt-imiquimod-fluorouracil-superficial-bcc","scin-trial"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Low-risk basal cell carcinoma, what the randomised trials show. In the UK SINS trial, 501 participants with primary nodular or superficial basal cell carcinoma at low-risk sites were randomised to imiquimod once daily (six weeks for superficial, twelve weeks for nodular) or to excision with a 4 mm margin. Success was 178 of 213 (83.6 percent) against 185 of 188 (98.4 percent) at three years and 170 of 206 (82.5 percent) against 173 of 177 (97.7 percent) at five years, and most imiquimod failures occurred in year one. The authors' conclusion was that imiquimod was inferior to surgery by their predefined criterion, and that excisional surgery remains the best treatment for low-risk basal cell carcinoma.","Among the non-surgical options, imiquimod is the one with the best evidence. In a Dutch trial of 601 patients with superficial basal cell carcinoma, five-year tumour-free survival was 80.5 percent for imiquimod, 70.0 percent for fluorouracil cream and 62.7 percent for methyl-aminolevulinate photodynamic therapy. The Cochrane review reached the same conclusion, that of the non-surgical treatments imiquimod has the best evidence to support its efficacy.","What it does to the skin while it works: in SINS the commonest adverse events were itching (211 in the imiquimod group against 129 in the surgery group) and weeping (160 against 81), and 12 of 249 participants in the imiquimod group withdrew because of adverse events against four of 229 in the surgery group."],"brand":"Aldara / Zyclara","modality":"Topical immune response modifier (TLR7 agonist)","mechanism":"Toll-like receptor 7 agonist that induces interferon-alpha, TNF and other cytokines from dendritic cells and macrophages; the label states the mechanism in basal cell carcinoma and actinic keratosis is not fully established.","approvals":[{"region":"US","year":1997,"indication":"External genital and perianal warts"},{"region":"US","year":2004,"indication":"Actinic keratosis; superficial basal cell carcinoma in immunocompetent adults"},{"region":"EU","year":1998,"indication":"Genital warts, superficial basal cell carcinoma and actinic keratosis (Aldara)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"imlunestrant","kind":"drug","name":"Imlunestrant","aka":[],"tldr":"Imlunestrant is Lilly's oral oestrogen-receptor degrader, approved in 2025 for ESR1-mutant breast cancer and shown to work with abemaciclib regardless of mutation.","summary":"EMBER-3: monotherapy PFS benefit confined to ESR1-mutant tumours (HR 0.62; OS 34.5 vs 23.1 months in update); imlunestrant + abemaciclib beat imlunestrant alone in all patients (PFS 10.9 vs 5.5 months, HR 0.59). FDA approved 25 September 2025 for ESR1-mutant disease after ≥1 endocrine line. EMBER-4 tests it as adjuvant therapy.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Imlunestrant"}],"tags":[],"related":["esr1-mutation-ctdna"],"cancers":["breast-hr-positive","hr-positive-metastatic-post-cdk46"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":["esr1-mutation","oral-serd"],"trials":["ember-3","nct07287098","nct05514054"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Inluriyo","code":"LY3484356","modality":"Oral SERD","mechanism":"Brain-penetrant oral SERD; pure antagonist that degrades ER.","approvals":[{"region":"US","year":2025,"indication":"ER+/HER2- ESR1-mutated advanced breast cancer after ≥1 line of endocrine therapy"},{"region":"EU","year":2026,"indication":"ESR1-mutant ER+/HER2- mBC; 9 Jan 2026"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"400 mg once daily (approved as monotherapy); combination with abemaciclib studied","monitoring":"Liver enzymes; diarrhoea with abemaciclib"},"toxicity":[{"event":"Diarrhoea (with abemaciclib)","anyGradePct":86,"grade3PlusPct":8},{"event":"Nausea (monotherapy)","anyGradePct":17},{"event":"Fatigue","anyGradePct":23}],"access":[],"regulatoryEvents":[{"date":"2025-09-25","type":"approval","region":"US","note":"Approved as Inluriyo","source":"https://investor.lilly.com/news-releases/news-release-details/us-fda-approves-inluriyo-imlunestrant-adults-er-her2-esr1"},{"date":"2026-09-18","type":"approval","region":"US","note":"With abemaciclib, for ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-imlunestrant-combination-abemaciclib-er-positive-her2-negative-esr1-mutated-advanced-or"}]},{"id":"imp1734","kind":"drug","name":"IMP1734","aka":[],"tldr":"IMP1734 is a small-molecule inhibitor from Eikon Therapeutics, in registered phase 2 trials for metastatic cancer.","summary":"IMP1734 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Eikon Therapeutics, in metastatic cancer. Described in the registry record as a parp1 selective inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of IMP1734","url":"https://clinicaltrials.gov/search?intr=IMP1734"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["eikon-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06253130"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"IMP1734","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a parp1 selective inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"in10018","kind":"drug","name":"IN10018","aka":["IN10018 add on to PLD treatment","BI 853520"],"tldr":"IN10018 is an experimental small-molecule drug from InxMed (Shanghai) in phase 3 trials for non-small-cell lung cancer, pancreatic ductal adenocarcinoma and ovarian cancer, with its target not yet stated publicly.","summary":"IN10018 (BI853520) is a small-molecule drug developed by InxMed (Shanghai) and InventisBio. The sponsor describes its target as FAK, which OnCo does not yet have a target page for. The sponsor states: FAK (focal adhesion kinase) inhibitor. ClinicalTrials.gov describes the intervention as: Based on existing clinical data, the RP2D of IN10018 as monotherapy and in combination with chemotherapy, targeted therapy, and immunotherapy is 100 mg QD. For RNK08954 as monotherapy, the Maximum Tolerated Dose (MTD) was not reached during. It is the investigational product in 8 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07174908 (A Phase 3 Study of IN10018 in Combination With D-1553 Versus Standard Therapy for First Line Non-squamous Non-small Cell Lung Cancer With KRAS G12C Mutation), in non-small-cell lung cancer, pancreatic ductal adenocarcinoma, ovarian cancer and small-cell lung cancer. The largest, NCT07174908, plans to enrol 400 participants with primary completion expected 2028-09. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IN10018","url":"https://clinicaltrials.gov/search?intr=BI853520"},{"label":"Sponsor page","url":"https://www.inxmed.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","pancreatic","ovarian","sclc"],"sections":[],"technologies":[],"targets":["fak"],"drugs":[],"companies":["inventisbio","inxmed"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07174908","nct06014528","nct06030258","nct05551507","nct06708663","nct05994131","nct07441174","nct06166836","nct05827796","nct05379946"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"BI853520","modality":"small molecule","mechanism":"FAK (focal adhesion kinase) inhibitor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"inaticabtagene-autoleucel","kind":"drug","name":"Inaticabtagene autoleucel","aka":[],"tldr":"Inati-cel is a Chinese CAR-T for adults with acute lymphoblastic leukaemia that has come back or stopped responding, approved in 2023.","summary":"Developed by Juventas Cell Therapy with the Institute of Hematology and Blood Diseases Hospital of the Chinese Academy of Medical Sciences, and approved by the NMPA in 2023 for adult relapsed or refractory CD19-positive B-cell acute lymphoblastic leukaemia, the first CAR-T approved in China for leukaemia. It illustrates how China's academic haematology centres have become the origin of approved cell therapies rather than only trial sites.","status":"approved","asOf":"2026-09-10","links":[{"label":"Juventas Cell Therapy","url":"https://www.juventas.cn/"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["all-leukemia"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":[],"companies":["juventas-cell-therapy"],"institutions":["cams-hematology"],"pathways":[],"terms":[],"trials":["nct05667506","nct04684147","nct04586478"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Yuanruida","code":"inati-cel, CNCT19","modality":"Cell therapy (autologous CD19 CAR-T)","mechanism":"Autologous CD19-directed CAR-T cells using a novel CD19 binder (HI19a) developed at the Institute of Hematology, Chinese Academy of Medical Sciences, in Tianjin.","approvals":[{"region":"China","year":2023,"indication":"Adult relapsed or refractory CD19-positive B-cell acute lymphoblastic leukaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"inavolisib","kind":"drug","name":"Inavolisib","aka":[],"tldr":"Inavolisib is a PI3K drug that also destroys the mutant protein, approved in 2024 with palbociclib and fulvestrant for PIK3CA-mutant breast cancer.","summary":"Inavolisib is a PI3K-alpha-selective inhibitor that also promotes degradation of the mutant p110-alpha protein, combining enzyme blockade with removal of the target. It is taken as 9 mg once daily with palbociclib and fulvestrant, and was approved in 2024 for PIK3CA-mutant HR-positive, HER2-negative advanced breast cancer on INAVO120, where the triplet improved progression-free survival to 15.0 versus 7.3 months in endocrine-resistant disease, with an overall survival benefit reported in 2025. Roche/Genentech developed it. Hyperglycaemia, stomatitis, neutropenia and rash are the main adverse events. INAVO120 enrolled patients relapsing during or soon after adjuvant endocrine therapy, so its value in other PIK3CA-mutant settings is still being defined. For a newcomer: a PI3K drug that both blocks and destroys the mutant protein.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Inavolisib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Inavolisib"}],"tags":[],"related":["pi3k-pathway-plus-endocrine","pik3ca-hotspot-mutation"],"cancers":["breast-hr-positive","hr-positive-metastatic-post-cdk46"],"sections":[],"technologies":["kinase-inhibitors","pi3k-akt-mtor-inhibitors"],"targets":["pik3ca"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["hyperglycaemia"],"trials":["nct07287150","nct05646862","nct07368998","nct07405801","nct05894239","nct06790693","nct07054190"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Itovebi","modality":"Small-molecule PI3Kα inhibitor and degrader","mechanism":"PI3Kα-selective inhibitor that promotes degradation of mutant p110α.","approvals":[{"region":"US","year":2024,"indication":"PIK3CA-mutant HR+/HER2- advanced breast cancer with palbociclib and fulvestrant"},{"region":"EU","year":2025,"indication":"PIK3CA-mutant ER+/HER2- mBC with palbociclib + fulvestrant; 18 Jul 2025"}],"mechanismSteps":["Inavolisib binds mutant and wild-type PI3Kα","It also triggers degradation of the mutant p110α protein","AKT and mTOR signalling downstream collapse","With CDK4/6 and ER blockade, all three growth axes are shut","Endocrine-resistant tumour cells arrest and die"],"dosing":{"route":"Oral","schedule":"9 mg once daily with palbociclib and fulvestrant","modifications":"Reduce to 6 then 3 mg for hyperglycaemia, stomatitis, diarrhoea","monitoring":"Fasting glucose weekly for 4 weeks then every 4 weeks; HbA1c; oral care","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Neutropenia","note":"INAVO120 triplet: most common grade 3-4 (from palbociclib)"},{"event":"Hyperglycaemia"},{"event":"Stomatitis"},{"event":"Diarrhoea"},{"event":"Rash"},{"event":"Fatigue"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.genentech-access.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: appraisal for PIK3CA-mutant HR+ breast cancer (2025-26)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-10-10","type":"approval","region":"US","note":"PIK3CA-mutant HR+/HER2- endocrine-resistant advanced breast cancer with palbociclib and fulvestrant (INAVO120)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-06","type":"label-change","region":"US","note":"OS benefit added to label","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"inbrx-106","kind":"drug","name":"INBRX-106","aka":["Hexavalent OX40 agonist antibody"],"tldr":"INBRX-106 is an experimental monoclonal antibody from Inhibrx Biosciences in phase 3 trials for head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"INBRX-106 is a monoclonal antibody developed by Inhibrx Biosciences. The sponsor describes its target as OX40, which OnCo does not yet have a target page for. The sponsor states: A hexavalent OX40 agonist antibody designed to promote hyperclustering of OX40, giving more potent activation of the OX40 costimulatory pathway than traditional Fc-crosslinking-dependent bivalent OX40 antibodies. ClinicalTrials.gov describes the intervention as: INBRX-106 by intravenous (IV) infusion, given every 3 weeks (QW3). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06295731 (INBRX-106 in Combination With Pembrolizumab in First-line PD-L1 CPS≥20 HNSCC), in head and neck squamous cell carcinoma. The largest, NCT06295731, plans to enrol 410 participants with primary completion expected 2029-05. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of INBRX-106","url":"https://clinicaltrials.gov/search?intr=INBRX-106"},{"label":"Sponsor pipeline page","url":"https://inhibrx.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["inhibrx"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06295731"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"A hexavalent OX40 agonist antibody designed to promote hyperclustering of OX40, giving more potent activation of the OX40 costimulatory pathway than traditional Fc-crosslinking-dependent bivalent OX40 antibodies.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"inca033989","kind":"drug","name":"INCA033989","aka":["INCA-033989","Mutant-CALR antibody"],"tldr":"INCA033989 is an antibody that recognises only the mutant form of calreticulin found in about a quarter of essential thrombocythaemia and myelofibrosis patients. Unlike existing drugs, which control counts, it targets the diseased clone itself and is in first-in-human trials.","summary":"INCA033989 is Incyte's antibody against mutant calreticulin (CALR). CALR frameshift mutations are the second commonest driver of essential thrombocythaemia and primary myelofibrosis after JAK2 V617F; the mutant protein gains a positively charged tail that lets it bind and activate the thrombopoietin receptor MPL from outside the cell, an accessible target absent from normal cells. In preclinical work the antibody blocked mutant-CALR signalling and selectively suppressed the clone.\n\nA phase 1 trial in essential thrombocythaemia and myelofibrosis began in 2024, with early reports of platelet normalisation and falls in mutant-allele burden in essential thrombocythaemia. The essential thrombocythaemia page names it among the first treatments that target the clone rather than the counts.","status":"phase-1","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["ruxolitinib"],"cancers":["essential-thrombocythaemia"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["jak2"],"drugs":[],"companies":["incyte"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"INCA033989","modality":"Monoclonal antibody against mutant calreticulin","mechanism":"Binds the novel C-terminal tail that frameshift mutations give calreticulin, which the mutant protein uses to dimerise and activate the thrombopoietin receptor MPL on the cell surface; blocking it switches off JAK-STAT signalling only in the mutant clone.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"inca33890","kind":"drug","name":"INCA33890","aka":[],"tldr":"INCA33890 is an experimental investigational agent whose form is not stated in the registry from Incyte in phase 3 trials for colorectal cancer, with its target not yet stated publicly.","summary":"INCA33890 is an investigational agent whose form is not stated in the registry developed by Incyte. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: INCA33890 will be administered at protocol defined dose. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07284849 (A Study to Evaluate the Efficacy and Safety of Standard-of-Care Chemotherapy and Bevacizumab With or Without INCA33890 in the First-Line Treatment of Metastatic Microsatellite Stable Colorectal Cancer), in colorectal cancer. The largest, NCT07284849, plans to enrol 700 participants with primary completion expected 2028-12-29. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of INCA33890","url":"https://clinicaltrials.gov/search?intr=INCA33890"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["incyte"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07284849"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody (TGF-beta receptor II x PD-1, per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"incb123667","kind":"drug","name":"INCB123667","aka":[],"tldr":"INCB123667 is an experimental small-molecule drug from Incyte in phase 3 trials for ovarian cancer, with its target not yet stated publicly.","summary":"INCB123667 is a small-molecule drug developed by Incyte. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07214779 (Study to Evaluate INCB123667 Versus Investigator's Choice of Chemotherapy in Participants With Platinum-Resistant Ovarian Cancer With Cyclin E1 Overexpression), in ovarian cancer. The largest, NCT07214779, plans to enrol 466 participants with primary completion expected 2028-11-15. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of INCB123667","url":"https://clinicaltrials.gov/search?intr=INCB123667"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["incyte"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07214779","nct07023627"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"incb161734","kind":"drug","name":"INCB161734","aka":[],"tldr":"INCB161734 is an experimental small-molecule drug from Incyte in phase 3 trials for pancreatic ductal adenocarcinoma, aimed at KRAS.","summary":"INCB161734 is a small-molecule drug developed by Incyte. Its target is KRAS (the sponsor names KRAS G12D). The sponsor states: An oral tablet identified on the trial page as a KRAS G12D inhibitor, evaluated in combination with standard chemotherapy (mFOLFIRINOX or GemNabP) versus chemotherapy alone in previously untreated, KRAS G12D-mutated metastatic pancreatic ductal adenocarcinoma. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07522073 (A Study to Evaluate Chemotherapy With or Without INCB161734 in Previously Untreated, KRAS G12D-Mutated Metastatic Pancreatic Ductal Adenocarcinoma), in pancreatic ductal adenocarcinoma. The largest, NCT07522073, plans to enrol 588 participants with primary completion expected 2028-09-15. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of INCB161734","url":"https://clinicaltrials.gov/search?intr=INCB161734"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":["kras"],"drugs":[],"companies":["incyte"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07522073"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"An oral tablet identified on the trial page as a KRAS G12D inhibitor, evaluated in combination with standard chemotherapy (mFOLFIRINOX or GemNabP) versus chemotherapy alone in previously untreated, KRAS G12D-mutated metastatic pancreatic ductal adenocarcinoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"inetetamab","kind":"drug","name":"Inetetamab","aka":["Cipterbin"],"tldr":"Inetetamab is 3SBio's HER2 antibody, approved in China in 2020 with vinorelbine for HER2-positive metastatic breast cancer.","summary":"Inetetamab (Cipterbin) is a HER2 antibody developed by 3SBio in Shenyang with an engineered Fc region for stronger antibody-dependent cellular cytotoxicity. The NMPA approved it in June 2020 in combination with vinorelbine for HER2-positive metastatic breast cancer previously treated with chemotherapy, making it the first HER2 antibody developed in China.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of inetetamab","url":"https://clinicaltrials.gov/search?intr=inetetamab"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["her2"],"drugs":[],"companies":["3sbio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04736589","nct07366840","nct04963595"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"modality":"Anti-HER2 monoclonal antibody, Fc-engineered","mechanism":"Binds HER2 on tumour cells, blocking growth signals and recruiting immune cells; its modified Fc region strengthens antibody-dependent cell killing compared with trastuzumab.","approvals":[{"region":"CN","year":2020,"indication":"HER2-positive metastatic breast cancer after chemotherapy, with vinorelbine"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"infigratinib","kind":"drug","name":"Infigratinib","aka":["BGJ398"],"tldr":"Infigratinib is an FGFR inhibitor pill given accelerated approval in the United States in 2021 for bile duct cancer with an FGFR2 fusion, then withdrawn in 2024 when its confirmatory trial could not enrol; it is now being developed for achondroplasia instead.","summary":"Infigratinib, discovered at Novartis and developed by BridgeBio's QED Therapeutics with Helsinn, received FDA accelerated approval in May 2021 for previously treated, unresectable or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement, on a response rate of about 23 percent in a single-arm phase 2 trial. The first-line confirmatory trial PROOF 301 struggled to recruit against pemigatinib and futibatinib, and the sponsors withdrew the cholangiocarcinoma indication in 2024. Hyperphosphataemia, eye and nail changes are the FGFR class effects. BridgeBio continues the molecule at low dose for children with achondroplasia.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Infigratinib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Infigratinib"},{"label":"ChEMBL CHEMBL1852688","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1852688"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["cholangiocarcinoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["fgfr2"],"drugs":["pemigatinib","futibatinib"],"companies":["bridgebio-oncology-therapeutics","helsinn"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02159066","nct01975701","nct02706691"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Truseltiq","modality":"Oral FGFR1 to 3 kinase inhibitor","mechanism":"An ATP-competitive inhibitor of FGFR1, FGFR2 and FGFR3 kinases; tumours driven by FGFR2 fusions depend on that signalling and shrink when it is blocked.","approvals":[{"region":"US","year":2021,"indication":"Previously treated unresectable or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement (accelerated approval)","note":"Indication withdrawn in 2024"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2021-05-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/withdrawn-fda-grants-accelerated-approval-infigratinib-metastatic-cholangiocarcinoma","indication":"Treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangement as detected by an FDA approved test"},{"date":"2024-05-16","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.0 years after its accelerated approval.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/withdrawn-fda-grants-accelerated-approval-infigratinib-metastatic-cholangiocarcinoma","indication":"Treatment of adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangement as detected by an FDA approved test"}]},{"id":"iniparib","kind":"drug","name":"Iniparib","aka":[],"tldr":"Billed as the first PARP inhibitor for triple-negative breast cancer, it failed its phase 3 in 2011. It turned out not to inhibit PARP at all.","summary":"A randomised phase 2 (O'Shaughnessy, NEJM 2011) in metastatic TNBC showed improved response, PFS, and OS when iniparib was added to gemcitabine-carboplatin. The phase 3 (n=519) missed both co-primary endpoints. Subsequent biochemistry showed iniparib does not trap or inhibit PARP1/2 at clinically relevant concentrations; it acts as a non-specific thiol-reactive compound. Sanofi (which had bought BiPar for up to $500M) discontinued it after a negative phase 3 in squamous NSCLC (2013).\n\nLesson: mechanism claims should be verified before a class label is attached; the true PARP inhibitors (olaparib, talazoparib) later succeeded in BRCA-mutant breast cancer.","status":"negative","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Iniparib","links":[{"label":"Iniparib is not a PARP inhibitor (Clin Cancer Res 2012)","url":"https://aacrjournals.org/clincancerres/article/18/2/510/76892/A-Deep-Look-at-the-PARP-Inhibitor-Iniparib"}],"tags":["failure","lesson:wrong-drug"],"related":[],"cancers":["tnbc","nsclc"],"sections":[],"technologies":["parp-inhibitor"],"targets":["parp"],"drugs":["olaparib","talazoparib"],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00938652","nct01045304","nct01204125"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"BSI-201, SAR240550","modality":"Small molecule (claimed PARP inhibitor)","mechanism":"Originally described as a PARP1 inhibitor; later shown to be a non-selective cysteine-modifying agent without PARP inhibition.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"inlexisertib","kind":"drug","name":"Inlexisertib","aka":["DCC-3116"],"tldr":"Inlexisertib is an oral serine/threonine kinase inhibitor from Deciphera Pharmaceuticals, LLC, in registered phase 2 trials for metastatic cancer.","summary":"Inlexisertib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Deciphera Pharmaceuticals, LLC, in metastatic cancer. A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Inlexisertib","url":"https://clinicaltrials.gov/search?intr=Inlexisertib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["deciphera"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05957367"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral serine/threonine kinase inhibitor","mechanism":"A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ino-5401","kind":"drug","name":"INO-5401","aka":[],"tldr":"INO-5401 is an experimental cancer vaccine from Inovio Pharmaceuticals in phase 2 trials for glioma & glioblastoma, aimed at PSMA.","summary":"INO-5401 is a cancer vaccine developed by Inovio Pharmaceuticals. Its target is PSMA (the sponsor names WT1, PSMA, hTERT). The sponsor states: A combination of three separate DNA plasmids targeting the Wilms tumour gene-1 (WT1) antigen, prostate-specific membrane antigen (PSMA) and human telomerase reverse transcriptase (hTERT), delivered intramuscularly followed by electroporation with the CELLECTRA device to stimulate an anti-tumour immune response. ClinicalTrials.gov describes the intervention as: INO-5401 is a combination of 3 separate DNA plasmids targeting Wilms tumour gene-1 (WT1) antigen, prostate-specific membrane antigen (PSMA) and human telomerase reverse transcriptase (hTERT) genes. Starting on Day 0 three milligrams (mg) of. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in glioma & glioblastoma. The largest, NCT03491683, plans to enrol 52 participants (actual) with primary completion expected 2026-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of INO-5401","url":"https://clinicaltrials.gov/search?intr=INO-5401"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":[],"targets":["psma"],"drugs":[],"companies":["inovio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03491683"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"vaccine","mechanism":"A combination of three separate DNA plasmids targeting the Wilms tumour gene-1 (WT1) antigen, prostate-specific membrane antigen (PSMA) and human telomerase reverse transcriptase (hTERT), delivered intramuscularly followed by electroporation with the CELLECTRA device to stimulate an anti-tumour immune response.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"inotuzumab-ozogamicin","kind":"drug","name":"Inotuzumab ozogamicin","aka":[],"tldr":"Inotuzumab ozogamicin is an antibody carrying a DNA-cutting toxin to CD22 on leukaemia cells. It gets far more relapsed ALL patients into remission than chemotherapy and bridges them to transplant.","summary":"INO-VATE (n=326): CR/CRi 80.7% vs 29.4% vs standard chemotherapy, more MRD-negative remissions and transplants; OS 7.7 vs 6.7 months (HR 0.77), statistically significant on the 2-year landmark. Approved 2017 (adults); paediatric approval 2024 (ITCC-059). Hepatic veno-occlusive disease after transplant (boxed warning) is mitigated by limiting cycles and avoiding dual-alkylator conditioning. Moving frontline for older adults (ALLIANCE A041501; Mini-hyper-CVD + InO) and children (COG AALL1732).","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Inotuzumab_ozogamicin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Inotuzumab%20ozogamicin"}],"tags":[],"related":["cd22-expression"],"cancers":["all-leukemia","all-paediatric-relapsed","all-paediatric-high-risk"],"sections":[],"technologies":["adc"],"targets":["cd22"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":["payload","linker"],"trials":["ino-vate","nct05748171"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Besponsa","modality":"ADC","payload":"Calicheamicin (DNA-cleaving enediyne), DAR ~6","linker":"Acid-labile hydrazone (AcBut)","mechanism":"Humanised anti-CD22 IgG4 internalises; acid-cleaved calicheamicin binds the DNA minor groove and causes double-strand breaks.","approvals":[{"region":"US","year":2017,"indication":"Relapsed/refractory CD22+ B-ALL, adults"},{"region":"US","year":2024,"indication":"Paediatric patients ≥1 year with relapsed/refractory CD22+ B-ALL"}],"mechanismSteps":["Antibody binds CD22 on the B-lymphoblast","The complex is rapidly internalised into acidic endosomes","The hydrazone linker hydrolyses and releases calicheamicin","Calicheamicin binds the DNA minor groove and cleaves both strands","The blast dies; bystander effect is limited because free calicheamicin is short-lived"],"dosing":{"route":"IV over 1 hour","schedule":"Cycle 1: 0.8 mg/m² day 1, 0.5 mg/m² days 8 and 15 (21-day cycle); subsequent cycles 0.5 mg/m² days 1, 8, 15; limit to 2 cycles if proceeding to transplant, maximum 6","monitoring":"Liver enzymes and bilirubin (VOD boxed warning), counts, QT","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Besponsa"},"toxicity":[{"event":"Hepatic veno-occlusive disease","anyGradePct":14,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Besponsa","note":"Boxed warning; 22% of those who went on to transplant in INO-VATE"},{"event":"Thrombocytopenia","grade3PlusPct":41},{"event":"Febrile neutropenia","grade3PlusPct":27},{"event":"Transaminase elevation","anyGradePct":27}],"access":[],"regulatoryEvents":[{"date":"2017-08-17","type":"approval","region":"US","note":"INO-VATE"},{"date":"2024-03-06","type":"label-change","region":"US","note":"Paediatric expansion"}]},{"id":"int230-6","kind":"drug","name":"INT230-6","aka":[],"tldr":"INT230-6 is an experimental investigational agent whose form is not stated in the registry from Intensity Therapeutics in phase 3 trials for sarcomas, with its target not yet stated publicly.","summary":"INT230-6 is an investigational agent whose form is not stated in the registry developed by Intensity Therapeutics. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: INT230-6 is a fixed combination of cisplatin, vinblastine and SHAO. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06263231 (A Study to Investigate Efficacy & Safety of Intratumoral INT230-6 Compared to US Standard of Care in Adults With Soft Tissue Sarcomas (INVINCIBLE-3)), in sarcomas. The largest, NCT06263231, plans to enrol 333 participants with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of INT230-6","url":"https://clinicaltrials.gov/search?intr=INT230-6"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["sarcoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["intensity-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["invincible-3"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"interferon-alfa","kind":"drug","name":"Interferon alfa-2a/2b","aka":[],"tldr":"Interferon alfa was the first biologic cancer drug (1986). It treated hairy cell leukaemia, CML, melanoma, kidney cancer and Kaposi sarcoma, and has been replaced almost everywhere by better-tolerated agents.","summary":"Approved 1986 for hairy cell leukaemia, then AIDS-KS (1988), CML (1990s), adjuvant melanoma (1995, high-dose; pegylated 2011) and follicular lymphoma. Flu-like symptoms, depression, fatigue and autoimmunity limited use; imatinib, checkpoint inhibitors and TKIs replaced it, and the non-pegylated products were discontinued (Intron A 2021, Roferon-A 2007). Interferon survives in MPN as ropeginterferon.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Interferon_type_I","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Interferon_type_I"}],"tags":["gap-fill","historic"],"related":[],"cancers":["hairy-cell-leukemia","cml","melanoma","kaposi-sarcoma","rcc","polycythaemia-vera","erdheim-chester-disease"],"sections":[],"technologies":["cytokine-therapy"],"targets":[],"drugs":[],"companies":["roche-genentech","merck"],"institutions":[],"pathways":[],"terms":[],"trials":["euramos-1"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Roferon-A / Intron A / Sylatron","modality":"Recombinant type I interferon (cytokine)","mechanism":"Signals through IFNAR to induce antiproliferative, pro-apoptotic and immunostimulatory interferon-stimulated genes.","approvals":[{"region":"US","year":1986,"indication":"Hairy cell leukaemia"},{"region":"US","year":1988,"indication":"AIDS-related Kaposi sarcoma"},{"region":"US","year":1995,"indication":"Adjuvant high-risk melanoma (high-dose)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"intismeran-autogene","kind":"drug","name":"Intismeran autogene","aka":["mRNA-4157","V940","personalised cancer vaccine (PCV)","individualised neoantigen therapy (INT)"],"tldr":"A custom mRNA vaccine encoding up to 34 of a patient's own tumour mutations. In August 2026 it became the first personalised cancer vaccine to win a phase 3 trial.","summary":"Intismeran is developed by Moderna and Merck. Phase 2b KEYNOTE-942 showed 49% reduction in recurrence or death with pembrolizumab in resected melanoma. Phase 3 INTerpath-001 (1,137 patients, stage IIB-IV resected melanoma) met RFS and DMFS endpoints (19 August 2026); regulatory filings expected. Phase 3 trials in adjuvant NSCLC (INTerpath-002), RCC, bladder, and cutaneous SCC ongoing. Manufacturing ~6 weeks per patient.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"Merck/Moderna announcement","url":"https://www.merck.com/news/merck-and-moderna-announce-phase-3-interpath-001-trial-of-intismeran-autogene-plus-keytruda-met-endpoints-of-recurrence-free-survival-rfs-and-distant-metastasis-free-survival-dmfs-in-patient/"}],"tags":[],"related":[],"cancers":["melanoma","nsclc","rcc","urothelial","stage-iii-melanoma","stage-ii-melanoma"],"sections":[],"technologies":["neoantigen-mrna-vaccine"],"targets":[],"drugs":["pembrolizumab"],"companies":["moderna","merck"],"institutions":[],"pathways":[],"terms":[],"trials":["interpath-001","nct06077760","nct07513376","nct03897881","nct07221474","nct06307431","nct06961006","nct06623422","nct06305767","nct06833073"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"V940, mRNA-4157","modality":"Personalised mRNA neoantigen vaccine","mechanism":"Tumour/normal WES → neoantigen prediction → single mRNA encoding up to 34 neoepitopes in LNP → intramuscular dosing with pembrolizumab.","approvals":[],"mechanismSteps":["Tumour and normal DNA are sequenced; up to 34 patient-specific neoantigens are predicted","A single mRNA encoding those neoantigens is manufactured (~6 weeks) and packaged in lipid nanoparticles","Injected mRNA is translated by dendritic cells, which present the neoantigens","Neoantigen-specific T cells are primed and expanded","Pembrolizumab keeps those T cells active against residual tumour cells","Recurrence is prevented or delayed"],"dosing":{"route":"Intramuscular injection","schedule":"1 mg every 3 weeks for up to 9 doses, with pembrolizumab 200 mg every 3 weeks for up to 18 cycles","monitoring":"Injection-site and systemic reactions; immune-related events from pembrolizumab","source":"https://clinicaltrials.gov/study/NCT05933577"},"toxicity":[{"event":"Fatigue"},{"event":"Injection-site pain"},{"event":"Chills"},{"event":"Pyrexia"},{"event":"Myalgia"},{"event":"Headache"}],"access":[{"country":"US","reimbursement":"Investigational; BLA expected 2026-27 after INTerpath-001","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-02-22","type":"designation","region":"US","note":"Breakthrough Therapy designation, adjuvant melanoma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-04","type":"designation","region":"EU","note":"PRIME designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-08-19","type":"filing","region":"US","note":"Phase 3 INTerpath-001 met RFS and DMFS endpoints; filings to follow","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"bcg-intravesical","kind":"drug","name":"Intravesical BCG","aka":[],"tldr":"A weakened tuberculosis vaccine put directly into the bladder. Since 1976 it has been the most effective treatment for early bladder cancer, and it remains in chronic short supply.","summary":"Induction (6 weekly instillations) plus 1-3 years of maintenance reduces recurrence and progression in high-risk NMIBC. Global shortages since 2012 (single US manufacturer, Merck) have forced dose-splitting and priority rules. New combinations: durvalumab + BCG (POTOMAC, approved 2026), N-803 + BCG (Anktiva, 2024), and recombinant BCG (VPM1002) in trials. About 30-40% of patients become BCG-unresponsive.","status":"standard-of-care","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/BCG_vaccine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/BCG_vaccine"}],"tags":[],"related":[],"cancers":["urothelial"],"sections":[],"technologies":["bcg-and-intravesical-therapy"],"targets":[],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":["nmibc-vs-mibc","bcg-unresponsive"],"trials":["potomac","quilt-3-032"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"TICE BCG","modality":"Live bacterial immunotherapy (intravesical)","mechanism":"Attaches to urothelium via fibronectin, is internalised, and triggers a Th1 innate and adaptive immune response with trained immunity.","approvals":[{"region":"US","year":1990,"indication":"Carcinoma in situ of the bladder (TICE BCG)"}],"mechanismSteps":["BCG binds fibronectin on disrupted urothelium and is internalised by tumour cells","Cytokine release (IL-2, IFN-γ, TNF) recruits neutrophils, macrophages, and T cells","Trained innate immunity and BCG-specific T cells eliminate residual tumour","Repeated maintenance instillations sustain the response"],"dosing":{"route":"Intravesical","schedule":"Weekly ×6 induction, then 3 weekly instillations at months 3, 6, 12, 18, 24, 30, 36 (SWOG maintenance) for high-risk disease","modifications":"Reduced (one-third) dose during shortages; hold for haematuria, UTI, or systemic symptoms","monitoring":"Cystoscopy and cytology every 3 months for 2 years"},"toxicity":[{"event":"Cystitis symptoms","anyGradePct":70},{"event":"Haematuria","anyGradePct":25},{"event":"Fever","anyGradePct":20},{"event":"Systemic BCG infection","anyGradePct":1,"note":"Rare; requires anti-tuberculous therapy"}],"access":[],"regulatoryEvents":[]},{"id":"io102-io103","kind":"drug","name":"IO102-IO103","aka":[],"tldr":"IO102-IO103 is an experimental peptide (immunotherapeutic vaccine) from IO Biotech in phase 3 trials for melanoma and head and neck squamous cell carcinoma, aimed at PD-L1.","summary":"IO102-IO103 (IO102, IO103) is a peptide (immunotherapeutic vaccine) developed by IO Biotech. Its target is PD-L1 (the sponsor names IDO1 and PD-L1). The sponsor states: IO102-IO103 comprises an IDO peptide antigen (IO102) and a PD-L1 peptide antigen (IO103) emulsified with adjuvant (Montanide ISA 51 VG) and given subcutaneously to stimulate an immune response against IDO- and PD-L1-expressing cells, dosed alongside pembrolizumab in advanced melanoma. ClinicalTrials.gov describes the intervention as: IO102-IO103 comprises IDO peptide antigen (IO102) and PD-L1 peptide antigen (IO103) emulsified with adjuvant (Montanide ISA 51 VG) administered subcutaneously. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05155254 (IO102-IO103 in Combination With Pembrolizumab Versus Pembrolizumab Alone in Advanced Melanoma (IOB-013 / KN-D18)), in melanoma and head and neck squamous cell carcinoma. The largest, NCT05155254, plans to enrol 407 participants (actual) with primary completion was scheduled for 2025-05-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IO102-IO103","url":"https://clinicaltrials.gov/search?intr=IO102"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma","head-and-neck"],"sections":[],"technologies":[],"targets":["pdl1","ido1"],"drugs":[],"companies":["io-biotech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05155254","nct05280314"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"IO102, IO103","modality":"peptide (immunotherapeutic vaccine)","mechanism":"IO102-IO103 comprises an IDO peptide antigen (IO102) and a PD-L1 peptide antigen (IO103) emulsified with adjuvant (Montanide ISA 51 VG) and given subcutaneously to stimulate an immune response against IDO- and PD-L1-expressing cells, dosed alongside pembrolizumab in advanced melanoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"iomab-b","kind":"drug","name":"Iomab-B","aka":["Apamistamab-I131","131I-apamistamab"],"tldr":"Iomab-B is Actinium's radioactive antibody that clears the bone marrow before a transplant for older patients with active relapsed acute myeloid leukaemia; the phase 3 SIERRA trial met its primary endpoint but the FDA asked for another study.","summary":"Iomab-B delivers iodine-131 to CD45-positive cells, allowing patients with relapsed or refractory acute myeloid leukaemia who would not tolerate conventional myeloablative conditioning to proceed to allogeneic transplant. In the phase 3 SIERRA trial, Iomab-B followed by transplant produced durable complete remission at six months in a substantially higher share of patients than conventional care. Actinium reported in 2024 that the FDA requested an additional randomised trial before filing, and it is seeking partners; the trial is recorded here.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Iomab-B","url":"https://clinicaltrials.gov/search?intr=Iomab-B"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["aml"],"sections":[],"technologies":["radioimmunotherapy","radioligand-therapy"],"targets":[],"drugs":[],"companies":["actinium-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02665065"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"modality":"Radioimmunotherapy conditioning agent (anti-CD45 antibody labelled with iodine-131)","mechanism":"An antibody against CD45, found on all white blood cells, carries iodine-131 to the marrow and leukaemia cells and ablates them in place of high-dose chemotherapy before a stem cell transplant.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"iparomlimab-tuvonralimab","kind":"drug","name":"Iparomlimab and tuvonralimab","aka":["PSB205"],"tldr":"QL1706 is Qilu Pharmaceutical's PD-1 plus CTLA-4 antibody mixture, approved in China in 2024 for recurrent or metastatic cervical cancer after platinum chemotherapy.","summary":"Iparomlimab and tuvonralimab (QL1706) uses Qilu's MabPair platform to produce an anti-PD-1 and a short-half-life anti-CTLA-4 antibody in a single manufacturing run. The NMPA approved it in September 2024 for recurrent or metastatic cervical cancer that had failed platinum-based chemotherapy, the first dual checkpoint product approved for this cancer. Phase 3 trials in lung and liver cancers continue.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of QL1706","url":"https://clinicaltrials.gov/search?intr=QL1706"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["cervical"],"sections":[],"technologies":["checkpoint-inhibitor","bispecific-antibody"],"targets":["pdl1"],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06789796","nct07447570","nct07446387"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"QL1706","modality":"Bifunctional antibody mixture: anti-PD-1 and anti-CTLA-4","mechanism":"Two antibodies made in one cell line and given as a fixed mixture: a PD-1 blocker with a normal half-life and a CTLA-4 blocker engineered to clear quickly, so the double checkpoint blockade comes with less of the toxicity seen with ipilimumab combinations.","approvals":[{"region":"CN","year":2024,"indication":"Recurrent or metastatic cervical cancer after platinum chemotherapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ipatasertib","kind":"drug","name":"Ipatasertib","aka":[],"tldr":"Ipatasertib is an experimental small-molecule drug from Hoffmann-La Roche in phase 3 trials for HR-positive / HER2-negative breast cancer and ovarian cancer, aimed at AKT.","summary":"Ipatasertib (GDC-0068, RO5532961, RG7440) is a small-molecule drug developed by Hoffmann-La Roche. Its target is AKT (the sponsor names AKT). ClinicalTrials.gov describes the intervention as: For participants 12-17 years of age, ipatasertib will be administered at the starting dose of 200 mg for participants \\< 35 kg, 300 mg for participants ≥35 and \\< 45 kg, 400 mg for those ≥ 45 kg orally QD, beginning of Cycle 1, on Days 1-21. It is the investigational product in 5 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05862285 (A Rollover Study for Participants Previously Enrolled in a Genentech and/or F. Hoffman-La Roche Sponsored Study), in HR-positive / HER2-negative breast cancer and ovarian cancer. The largest, NCT03424005, plans to enrol 1132 participants with primary completion expected 2030-09-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Ipatasertib","url":"https://clinicaltrials.gov/search?intr=GDC-0068"},{"label":"IPATunity130 cohort A (Clinical Cancer Research 2024)","url":"https://doi.org/10.1158/1078-0432.CCR-24-0465"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","ovarian","tnbc","tnbc-metastatic","tnbc-early"],"sections":[],"technologies":[],"targets":["akt"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05862285","nct04931342","ipatunity130","barbican"],"people":[],"bottlenecks":[],"keyPapers":["paper-kim-lotus-ipatasertib-tnbc-lancet-oncol-2017"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: IPATunity130 cohort A (255 patients with PIK3CA, AKT1 or PTEN-altered advanced disease, first-line paclitaxel with or without ipatasertib) showed no progression-free survival difference (7.4 versus 6.1 months, hazard ratio 1.02) and no overall survival difference (24.4 versus 24.9 months), failing to confirm the phase 2 LOTUS signal; grade 3 or worse diarrhoea was 9 versus 2 percent. The UK BARBICAN phase 2 (146 patients, Barts) tests its contribution to neoadjuvant chemotherapy with atezolizumab."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"GDC-0068, RO5532961, RG7440","modality":"small molecule","mechanism":"Small-molecule drug directed at AKT, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ipg1094","kind":"drug","name":"IPG1094","aka":[],"tldr":"IPG1094 is a small-molecule inhibitor from Nanjing Immunophage Biotech Co., Ltd, in registered phase 2 trials for metastatic cancer.","summary":"IPG1094 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Nanjing Immunophage Biotech Co., Ltd, in metastatic cancer. Described in the registry record as a an inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of IPG1094","url":"https://clinicaltrials.gov/search?intr=IPG1094"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06212076"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"IPG1094","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a an inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ipilimumab","kind":"drug","name":"Ipilimumab","aka":[],"tldr":"Ipilimumab was the first checkpoint inhibitor (2011), and proved the immune system could be unleashed against cancer.","summary":"Ipilimumab is a fully human IgG1 antibody against CTLA-4 that enhances T-cell priming by stopping CTLA-4 from outcompeting CD28 for B7 ligands, and depletes intratumoural regulatory T cells through Fc effector function. It was the first checkpoint inhibitor, approved in 2011 for metastatic melanoma after monotherapy improved overall survival (2010 NEJM), and proved the immune system could be unleashed against cancer. It is now mostly given with nivolumab in melanoma, RCC, MSI-high colorectal cancer, HCC, mesothelioma and NSCLC, at 3 mg/kg in melanoma and 1 mg/kg elsewhere. In CheckMate 067 the combination produced 10-year overall survival of 43 percent versus 37 percent for nivolumab alone and 19 percent for ipilimumab alone. Immune-related adverse events are frequent, with colitis in 25 percent and hepatitis in 15 percent alongside nivolumab, and severe cases end treatment.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Ipilimumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ipilimumab"},{"label":"NICE TA1065","url":"https://www.nice.org.uk/guidance/ta1065"},{"label":"NICE TA724: nivolumab with ipilimumab and chemotherapy for untreated metastatic non-small-cell lung cancer (not recommended)","url":"https://www.nice.org.uk/guidance/ta724"}],"tags":[],"related":[],"cancers":["melanoma","rcc","colorectal","hcc","mesothelioma","msi-high-colorectal","advanced-melanoma","stage-iii-melanoma","mucosal-melanoma","acral-melanoma","lung-cancer"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["ctla4"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["checkmate-067","nct03873402","nct06581406","nct06097728","nct05987332","nct06946797","nct07227012","nct06784648","nct07291076","nct07227415","nct06362369","nct07293351","nct03907852","nct03647163","nct07563738","nct06047379","nct03899155","nct03036098","nct05584670","nct05144854","nct03865082","nct05533697","checkmate-227","checkmate-9la"],"people":["inge-marie-svane"],"bottlenecks":[],"keyPapers":["paper-niche-2-nejm-2024"],"journals":[],"dependsOn":[],"notes":[],"brand":"Yervoy","modality":"Monoclonal antibody (anti-CTLA-4)","mechanism":"Fully human IgG1 anti-CTLA-4; enhances T-cell priming and depletes intratumoural Tregs.","approvals":[{"region":"US","year":2011,"indication":"Metastatic melanoma"},{"region":"England (NICE)","year":2025,"indication":"Untreated unresectable or metastatic MSI-high or dMMR colorectal cancer, with nivolumab","note":"TA1065, published 28 May 2025."},{"region":"England (NICE)","year":2021,"indication":"Untreated metastatic non-small-cell lung cancer, with nivolumab and two cycles of platinum doublet chemotherapy: not recommended","note":"TA724, published 8 September 2021."}],"mechanismSteps":["Antibody binds CTLA-4 on T cells in lymph nodes and tumour","CTLA-4 can no longer outcompete CD28 for B7 during priming","More T cells are activated and proliferate; regulatory T cells in the tumour are depleted via Fc effector function","Broadened T-cell repertoire infiltrates the tumour","Durable tumour control in a subset; autoimmunity is the trade-off"],"dosing":{"route":"IV infusion","schedule":"Melanoma monotherapy 3 mg/kg every 3 weeks ×4; with nivolumab 3 mg/kg (melanoma) or 1 mg/kg (RCC, NSCLC, HCC, MSI-H CRC) every 3 or 6 weeks","modifications":"Permanently discontinue for grade 3-4 colitis, hepatitis, hypophysitis with severe symptoms","monitoring":"LFTs, thyroid, cortisol/ACTH; colitis education; early steroids","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394"},"toxicity":[{"event":"Colitis (with nivolumab 1+3)","anyGradePct":25,"grade3PlusPct":14.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Nivolumab 1 mg/kg + ipilimumab 3 mg/kg, melanoma"},{"event":"Hepatitis (with nivolumab)","anyGradePct":15,"grade3PlusPct":13.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Nivolumab 1 mg/kg + ipilimumab 3 mg/kg, melanoma"},{"event":"Hypothyroidism (with nivolumab)","anyGradePct":20,"grade3PlusPct":0.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Nivolumab 1 mg/kg + ipilimumab 3 mg/kg, melanoma"},{"event":"Rash (with nivolumab)","anyGradePct":28,"grade3PlusPct":4.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Nivolumab 1 mg/kg + ipilimumab 3 mg/kg, melanoma"},{"event":"Adrenal insufficiency (with nivolumab)","anyGradePct":8,"grade3PlusPct":2.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Nivolumab 1 mg/kg + ipilimumab 3 mg/kg, melanoma"},{"event":"Hyperthyroidism (with nivolumab)","anyGradePct":9,"grade3PlusPct":0.9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Nivolumab 1 mg/kg + ipilimumab 3 mg/kg, melanoma"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.bmsaccesssupport.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with nivolumab in melanoma, RCC, MSI-H CRC, mesothelioma, HCC","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2011-03-25","type":"approval","region":"US","note":"Metastatic melanoma: first checkpoint inhibitor approved anywhere","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2015-10-28","type":"approval","region":"US","note":"Adjuvant stage III melanoma (10 mg/kg; later superseded)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2018-04-16","type":"approval","region":"US","note":"With nivolumab in RCC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2018-07-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with nivolumab, is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan"},{"date":"2020-03-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with nivolumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib"},{"date":"2020-05-15","type":"approval","region":"US","note":"With nivolumab in first-line NSCLC (CheckMate 227) and HCC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-10-02","type":"approval","region":"US","note":"With nivolumab in mesothelioma (CheckMate 743)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-04-08","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 6.7 years after it was granted.","indication":"In combination with nivolumab, is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan"},{"date":"2025-04-11","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 5.1 years after it was granted.","indication":"In combination with nivolumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib"},{"date":"2025-04","type":"approval","region":"US","note":"With nivolumab first-line MSI-H/dMMR colorectal cancer (CheckMate 8HW)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"irinotecan","kind":"drug","name":"Irinotecan (and liposomal irinotecan)","aka":[],"tldr":"Irinotecan is a topoisomerase-blocking chemotherapy central to bowel and pancreatic cancer regimens (FOLFIRI, FOLFIRINOX, NALIRIFOX) and to salvage therapy in childhood sarcomas; it carries the same warhead as the deruxtecan ADC payloads.","summary":"Approved 1996 (colorectal after 5-FU); FOLFIRI first line with bevacizumab/cetuximab; FOLFIRINOX (2011) and liposomal irinotecan (NAPOLI-1 2015, NAPOLI-3 NALIRIFOX 2024) in pancreatic cancer; irinotecan-temozolomide in Ewing, rhabdomyosarcoma, neuroblastoma; vincristine-irinotecan windows in RMS. Diarrhoea (cholinergic and delayed) and neutropenia; UGT1A1 genotyping is label-recommended.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Irinotecan","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=irinotecan"}],"tags":["gap-fill","generic","who-essential"],"related":["st-johns-wort-interaction"],"cancers":["colorectal","pancreatic","ewing-sarcoma","rhabdomyosarcoma","neuroblastoma","sclc","medulloblastoma"],"sections":[],"technologies":["topoisomerase-inhibitors","cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["pfizer","ipsen","merrimack-pharmaceuticals"],"institutions":[],"pathways":[],"terms":["payload"],"trials":["nct06219941","nct04657068","nct05592626","nct07356973","nct04725994","swog-s1406","avf2107g"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Camptosar / Onivyde","modality":"Topoisomerase I inhibitor (camptothecin prodrug)","mechanism":"Converted by carboxylesterases to SN-38, which traps topoisomerase I-DNA cleavage complexes; UGT1A1*28 slows SN-38 clearance and raises toxicity.","approvals":[{"region":"US","year":1996,"indication":"Metastatic colorectal cancer after 5-FU; first line with 5-FU/LV 2000"},{"region":"US","year":2015,"indication":"Liposomal irinotecan with 5-FU/LV in metastatic pancreatic cancer after gemcitabine"},{"region":"US","year":2024,"indication":"Liposomal irinotecan in NALIRIFOX first-line metastatic pancreatic cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1996-06-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Metastatic colon or rectal carcinoma that progressed following 5-FU therapy"},{"date":"1998-10-22","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1996 converted to traditional approval 2.4 years after it was granted.","indication":"Metastatic colon or rectal carcinoma that progressed following 5-FU therapy"}]},{"id":"iruplinalkib","kind":"drug","name":"Iruplinalkib","aka":["Qixinke"],"tldr":"Iruplinalkib is Qilu Pharmaceutical's second-generation ALK inhibitor, approved in China in 2023 for ALK-positive non-small-cell lung cancer after crizotinib and in 2024 as first-line treatment.","summary":"Iruplinalkib (WX-0593) was developed by Qilu Pharmaceutical in Jinan. The NMPA approved it in June 2023 for locally advanced or metastatic ALK-positive non-small-cell lung cancer that had progressed on or was intolerant to crizotinib, and in 2024 extended the approval to first-line use after the INSPIRE phase 3 trial showed longer progression-free survival than crizotinib.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of iruplinalkib","url":"https://clinicaltrials.gov/search?intr=WX-0593"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["nsclc","alk-positive-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["alk","ros1"],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04632758","nct05765877","nct04641754"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"WX-0593","modality":"Oral ALK and ROS1 tyrosine kinase inhibitor","mechanism":"Blocks the ALK fusion kinase that drives about five percent of non-small-cell lung cancers, including resistance mutations that stop first-generation inhibitors.","approvals":[{"region":"CN","year":2023,"indication":"ALK-positive non-small-cell lung cancer after crizotinib; first line from 2024"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"isatuximab","kind":"drug","name":"Isatuximab","aka":[],"tldr":"Isatuximab is Sanofi's CD38 antibody, approved in frontline transplant-ineligible myeloma (IMROZ) and, from July 2026, as an under-the-skin injection.","summary":"Isatuximab is a chimeric IgG1 antibody against CD38 that binds a distinct epitope from daratumumab and kills plasma cells through direct apoptosis without crosslinking as well as ADCC and ADCP. It was approved in 2020 with pomalidomide-dexamethasone (ICARIA-MM) and in 2021 with carfilzomib-dexamethasone (IKEMA) for relapsed myeloma. In IMROZ, adding isatuximab to VRd in transplant-ineligible newly diagnosed patients cut progression with a PFS HR of 0.60 and 60-month PFS of 63.2% versus 45.2%, leading to approval in September 2024. A subcutaneous on-body delivery system (Sarclisa Escena) was approved in July 2026, removing long intravenous infusions. Sanofi develops it; the main competitive question is how it compares with daratumumab, which reached the frontline quadruplet setting first. It is the second CD38 antibody, now usable from diagnosis in older patients not having a transplant.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Isatuximab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Isatuximab"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-transplant-ineligible"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd38"],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["imroz","nct05704049","nct07217184","nct04270409"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Sarclisa / Sarclisa Escena (SC)","modality":"Monoclonal antibody (anti-CD38)","mechanism":"Chimeric IgG1 anti-CD38 binding a distinct epitope; direct apoptosis without crosslinking plus ADCC/ADCP.","approvals":[{"region":"US","year":2020,"indication":"Relapsed myeloma with pomalidomide-dexamethasone; 2021 with carfilzomib-dexamethasone"},{"region":"US","year":2024,"indication":"Newly diagnosed transplant-ineligible with VRd (IMROZ)"},{"region":"US","year":2026,"indication":"Subcutaneous formulation (Sarclisa Escena)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-07-09","type":"approval","region":"US","note":"FDA approves isatuximab-irfc for subcutaneous injection for multiple myeloma indications","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-isatuximab-irfc-subcutaneous-injection-multiple-myeloma-indications"},{"date":"2024-09-20","type":"approval","region":"US","note":"IMROZ frontline"},{"date":"2026-07","type":"approval","region":"US","note":"Subcutaneous on-body injector"}]},{"id":"isb-2001","kind":"drug","name":"ISB 2001","aka":[],"tldr":"An Indian-discovered antibody that grips two targets on myeloma cells at once while pulling in T cells to kill them, licensed to AbbVie in 2025 in the biggest deal yet for an Indian cancer drug.","summary":"ISB 2001 is a trispecific antibody from Ichnos Glenmark Innovation that binds BCMA and CD38 on multiple myeloma cells and CD3 on T cells, with the aim of engaging tumours that express either antigen at low levels and reducing escape through single-antigen loss. It has been in phase 1 for relapsed or refractory multiple myeloma with orphan drug designation in the US. In July 2025 AbbVie licensed it for North America, Europe, Japan and Greater China for 700 million dollars upfront and up to 1.225 billion dollars in milestones plus royalties, with Glenmark retaining India and emerging markets. It is the clearest sign that Indian discovery, not only manufacturing, is entering global oncology.","status":"phase-1","asOf":"2026-09-10","links":[{"label":"Glenmark Pharmaceuticals","url":"https://www.glenmarkpharma.com"}],"tags":[],"related":["teclistamab","talquetamab"],"cancers":["multiple-myeloma"],"sections":[],"technologies":["t-cell-engager","bispecific-antibody"],"targets":["bcma","cd38"],"drugs":[],"companies":["glenmark","abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05862012"],"people":[],"bottlenecks":["b-translational-valley"],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ISB 2001","modality":"Trispecific T-cell engager (BCMA x CD38 x CD3)","mechanism":"Multispecific antibody with BCMA and CD38 binding arms and a CD3 arm that redirects T cells to lyse myeloma cells expressing either or both antigens.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"isotretinoin","kind":"drug","name":"Isotretinoin","aka":["13-cis-retinoic acid","13-cis-RA","cis-retinoic acid"],"tldr":"Isotretinoin, the acne drug, is given to children with high-risk neuroblastoma for six months after transplant to nudge any surviving cancer cells into growing up into harmless nerve cells. A 1999 trial showed it improved survival and it has been part of standard care since.","summary":"Isotretinoin (13-cis-retinoic acid) was approved by the FDA in 1982 for severe acne. Its use in neuroblastoma follows laboratory work showing retinoids induce differentiation of neuroblastoma cells. In the Children's Cancer Group trial CCG-3891 (New England Journal of Medicine, 1999), six months of high-dose pulsed isotretinoin after myeloablative therapy with autologous marrow rescue improved event-free survival, and the drug became the post-consolidation backbone onto which anti-GD2 immunotherapy was later added in ANBL0032.\n\nIt is taken by mouth in two-week courses; dry skin and lips, raised triglycerides and hypercalcaemia are the usual effects, and it is highly teratogenic. In medulloblastoma, the SJMB03 trial tested isotretinoin maintenance and found no benefit. The neuroblastoma and medulloblastoma pages name it.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Isotretinoin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Isotretinoin"}],"tags":["subtype-drugs-wave"],"related":["dinutuximab","tretinoin-atra"],"cancers":["neuroblastoma","neuroblastoma-high-risk","medulloblastoma-group-3-4"],"sections":[],"technologies":[],"targets":["rara","rxr"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Accutane","modality":"Oral retinoid (differentiating agent)","mechanism":"A vitamin A derivative that, through retinoic acid and retinoid X receptors, pushes residual neuroblastoma cells to stop dividing and differentiate towards mature neural cells, given after high-dose therapy when tumour burden is minimal.","approvals":[{"region":"US","year":1982,"indication":"Severe recalcitrant nodular acne (use in high-risk neuroblastoma is off-label but standard)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ivermectin","kind":"drug","name":"Ivermectin","aka":["Stromectol","IVM","MK-0933"],"tldr":"Ivermectin is a worm and parasite medicine that is being tested as an add-on to immunotherapy in two small early trials. No trial has shown that it treats any cancer, and people who have dosed themselves outside a trial have ended up in hospital with seizures or liver damage.","summary":"Ivermectin is approved in the United States as Stromectol for intestinal strongyloidiasis and onchocerciasis, two parasitic worm infections, and for nothing else (Stromectol label on DailyMed). The approval dates from 22 November 1996 under NDA 050742 (Drugs@FDA NDA 050742). The World Health Organization lists it as an essential medicine for onchocerciasis, strongyloidiasis, other soil-transmitted worm infections and scabies (WHO Model List of Essential Medicines).\n\nReviews collect cell-culture and mouse experiments in which ivermectin slowed cancer cells or shrank tumours in animals through several pathways at once, and note that no large randomised trial has confirmed any benefit in people (Patel review 2025). The most cited animal experiment reported that ivermectin plus an anti-PD-1 antibody limited tumour growth in mouse models of breast cancer where neither agent alone worked (Draganov mouse study 2021). That paper now carries an editorial expression of concern because several images in one figure were found to be duplicates and the authors said the original data were no longer available (Expression of concern 2026).\n\nAll 239 ivermectin studies registered on ClinicalTrials.gov were screened for this page, and two are cancer treatment trials: a phase 1/2 study at Cedars-Sinai giving ivermectin with balstilimab or pembrolizumab to about 34 patients with metastatic triple-negative breast cancer, recruiting since October 2023 with no results posted (ClinicalTrials.gov NCT05318469), and ICONIC, a University of Florida phase 2 comparing two ivermectin doses alongside a standard checkpoint inhibitor in 80 patients with solid tumours, not yet recruiting and measuring a change in immune cells rather than tumour response (ClinicalTrials.gov NCT07487805). The ICONIC registry entry says that among the first nine patients treated in the Cedars-Sinai study no treatment-related serious adverse events were observed, which is the only human safety information from either trial so far (ClinicalTrials.gov NCT07487805). Two other registered studies are not tests of ivermectin against cancer: a São Paulo phase 2 of ivermectin plus losartan for COVID-19 in cancer patients, stopped for futility after 77 of a planned 176 patients (ClinicalTrials.gov NCT04447235), and a Mexican clinic's 'atavistic chemotherapy' protocol that lists ivermectin among eight antiprotozoal drugs, last updated in 2022 with no results (ClinicalTrials.gov NCT02366884). No phase 3 trial exists, and neither cancer trial has reported whether ivermectin shrinks tumours or lengthens life (ClinicalTrials.gov NCT05318469).\n\nOutside trials the human evidence is survey and anecdote: 19 percent of cancer patients interviewed in Loja, Ecuador said they took ivermectin alongside chemotherapy, radiotherapy or immunotherapy (Loja survey 2023). A telemedicine cohort of 197 patients prescribed ivermectin with mebendazole reported self-assessed benefit in 84 percent of the 122 who answered a six-month survey (Hulscher cohort 2026). The journal has placed an expression of concern on that paper while it audits ethical approval and whether the diagnoses and reported regressions can be verified (Anticancer Research expression of concern 2026). A review for gynaecological cancers finds the clinical data limited to cell lines and 'strongly cautions' against use (Mujumdar review 2025), and the accompanying editorial notes that no major oncology organisation, including ASCO, SGO or NCCN, endorses ivermectin for cancer (Straughn editorial 2025).\n\nHarm from self-dosing is documented: a 73-year-old woman with metastatic breast cancer who took high doses on the strength of online information developed seizures and respiratory failure and needed a ventilator, recovering within 48 hours (Saperstein case report 2026). A 65-year-old man with prostate cancer who alternated veterinary fenbendazole and ivermectin for three months developed severe liver injury that resolved within six weeks of stopping (Powderly case report 2026). An Oregon poison-centre series of 37 people who took ivermectin against COVID-19, most men over 60, recorded neurotoxicity in 30, hospital admission in 21 and one death (Hoang poison centre series 2022). The label itself records drowsiness, stupor, coma, confusion and death at recommended doses and in overdose, most often after veterinary products (Stromectol label on DailyMed). After a January 2025 podcast, combined ivermectin and benzimidazole prescribing to US cancer patients ran 2.6 times higher than in the same months a year earlier (Rockwell JAMA Network Open 2026).\n\nThe bottom line for a patient who has read about this drug online: it is being studied in two early trials, no trial has shown that it treats any cancer, and dosing outside a trial has caused documented harm (Stromectol label on DailyMed). The one trial open to patients is the Cedars-Sinai study in metastatic triple-negative breast cancer (ClinicalTrials.gov NCT05318469). The FDA's guidance on products promoted as cancer cures without trial evidence applies here (FDA on products claiming to cure cancer).","status":"phase-2","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Ivermectin","links":[{"label":"Stromectol label on DailyMed","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd"},{"label":"Drugs@FDA NDA 050742","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=050742"},{"label":"WHO Model List of Essential Medicines","url":"https://list.essentialmeds.org/medicines/58"},{"label":"ClinicalTrials.gov NCT05318469","url":"https://clinicaltrials.gov/study/NCT05318469"},{"label":"ClinicalTrials.gov NCT07487805","url":"https://clinicaltrials.gov/study/NCT07487805"},{"label":"ClinicalTrials.gov NCT04447235","url":"https://clinicaltrials.gov/study/NCT04447235"},{"label":"ClinicalTrials.gov NCT02366884","url":"https://clinicaltrials.gov/study/NCT02366884"},{"label":"FDA on products claiming to cure cancer","url":"https://www.fda.gov/consumers/consumer-updates/products-claiming-cure-cancer-are-cruel-deception"},{"label":"Patel review 2025","url":"https://doi.org/10.1007/s11912-025-01704-z"},{"label":"Draganov mouse study 2021","url":"https://doi.org/10.1038/s41523-021-00229-5"},{"label":"Expression of concern 2026","url":"https://doi.org/10.1038/s41523-026-00988-z"},{"label":"Loja survey 2023","url":"https://doi.org/10.3390/nursrep13010030"},{"label":"Hulscher cohort 2026","url":"https://doi.org/10.21873/anticanres.18194"},{"label":"Anticancer Research expression of concern 2026","url":"https://doi.org/10.21873/anticanres.18276"},{"label":"Mujumdar review 2025","url":"https://doi.org/10.1016/j.gore.2025.101803"},{"label":"Straughn editorial 2025","url":"https://doi.org/10.1016/j.gore.2025.101920"},{"label":"Saperstein case report 2026","url":"https://doi.org/10.1080/00325481.2026.2672183"},{"label":"Powderly case report 2026","url":"https://doi.org/10.7759/cureus.108896"},{"label":"Hoang poison centre series 2022","url":"https://doi.org/10.1080/15563650.2022.2134788"},{"label":"Rockwell JAMA Network Open 2026","url":"https://doi.org/10.1001/jamanetworkopen.2026.16780"},{"label":"Yilmaz toxicity review 2026","url":"https://doi.org/10.1002/jat.70166"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Ivermectin"}],"tags":[],"related":[],"cancers":["tnbc"],"sections":[],"technologies":["fenbendazole-ivermectin-repurposing-claims","drug-repurposing","checkpoint-inhibitor"],"targets":[],"drugs":["balstilimab","pembrolizumab","mebendazole","fenbendazole"],"companies":["merck","agenus"],"institutions":["cedars-sinai-cancer","uf-health-cancer-center"],"pathways":[],"terms":["off-label","preclinical","in-vitro-in-vivo","cell-line","rp2d","orr","hepatotoxicity","trial-phases","pharmacokinetics","single-arm"],"trials":["nct05318469","nct07487805","nct04447235","nct02366884"],"people":[],"bottlenecks":["b-generic-repurposing","b-misinformation"],"keyPapers":["paper-patel-ivermectin-cancer-curr-oncol-rep-2025","paper-mujumdar-ivermectin-gynaecological-cancer-2025","paper-straughn-ivermectin-hope-versus-hype-2025","paper-yilmaz-ivermectin-toxicity-j-appl-toxicol-2026","paper-draganov-ivermectin-cold-tumours-npj-breast-cancer-2021","paper-hulscher-ivermectin-mebendazole-cohort-anticancer-res-2026","paper-rockwell-ivermectin-benzimidazole-prescribing-jama-netw-open-2026","paper-saperstein-ivermectin-neurotoxicity-breast-cancer-2026","paper-powderly-fenbendazole-ivermectin-liver-injury-2026","paper-hoang-ivermectin-toxicity-clin-toxicol-2022","paper-gilene-ivermectin-toxicity-paediatric-oncology-2025","paper-ghai-california-poison-control-ivermectin-2024","paper-jimenez-gaona-ivermectin-loja-ecuador-2023","paper-juarez-ivermectin-repositioned-cancer-drug-2018","paper-tang-ivermectin-potential-anticancer-pharmacol-res-2021","paper-cheng-melanoma-ctdna-antiparasitic-front-oncol-2026","paper-ishiguro-dichloroacetate-ivermectin-cureus-2022","paper-guilford-antiparasitic-acupuncture-meridian-case-series-2026"],"journals":[],"dependsOn":[],"notes":["Being studied: two early trials, one recruiting in metastatic triple-negative breast cancer (ClinicalTrials.gov NCT05318469) and one not yet open, ICONIC, which measures an immune-cell change rather than tumour shrinkage (ClinicalTrials.gov NCT07487805).","Not shown to treat any cancer: neither trial has reported a result and no phase 3 exists (ClinicalTrials.gov NCT05318469). The only published human data are a survey (Loja survey 2023) and a self-reported cohort under an expression of concern (Anticancer Research expression of concern 2026).","Harm outside trials: seizures and mechanical ventilation after high-dose self-use (Saperstein case report 2026), severe liver injury with veterinary fenbendazole (Powderly case report 2026), a poison-centre series with one death (Hoang poison centre series 2022), and the label's own warning of coma and death with overdose (Stromectol label on DailyMed).","Tell your oncologist if you are taking it: the toxicity review names P-glycoprotein, the pump that keeps ivermectin out of the brain, as the central factor in neurotoxicity, so anything that blocks it raises the risk (Yilmaz toxicity review 2026). A paediatric case of severe neurotoxicity in a patient on regorafenib was attributed to a CYP3A4 interaction (ClinicalTrials.gov NCT07487805)."],"provenance":{"editedBy":"OnCo agent (Claude Fable 5.1)","editedOn":"2026-09-24","note":"Written from cached ClinicalTrials.gov v2, Europe PMC, DailyMed, Drugs@FDA and WHO eEML responses; no figure appears without its source link."},"brand":"Stromectol","modality":"Oral antiparasitic small molecule (macrocyclic lactone), studied in cancer as an add-on to checkpoint antibodies","mechanism":"In parasites ivermectin opens glutamate-gated chloride channels and paralyses the worm. In cancer cell lines and mice it has been reported to act on several things at once, including the ATP-gated P2X4 and P2X7 receptors, WNT-TCF and Akt/mTOR signalling and PAK1, and the trials pair it with an anti-PD-1 antibody on the mouse finding that the pair drew T cells into tumours where neither drug alone worked. All of this is laboratory evidence; none of it has been shown in a patient.","approvals":[],"mechanismSteps":["Laboratory: in breast cancer cell lines, ivermectin triggers a form of cell death that releases ATP and other signals which attract immune cells (Draganov mouse study 2021).","Laboratory: as a modulator of the ATP-gated P2X4 and P2X7 receptors it was reported to reduce regulatory T cells and suppressive myeloid cells in mouse tumours (Draganov mouse study 2021).","Laboratory: on its own it did nothing to tumour growth in mice; with an anti-PD-1 antibody the combination limited growth, and the images behind part of that figure are now under an expression of concern (Expression of concern 2026).","Untested: whether any of this happens in people at the doses the two trials use is exactly the question the trials are asking (ClinicalTrials.gov NCT05318469)."],"dosing":{"route":"Oral tablet, 3 mg (Stromectol)","schedule":"Label: a single dose of about 200 micrograms per kg for strongyloidiasis and about 150 micrograms per kg for onchocerciasis, taken on an empty stomach with water; no cancer dose exists. Trials: the Cedars-Sinai study gives an assigned dose on days 1 to 3 of each week of a 21-day cycle with the antibody, and ICONIC compares 200 and 400 micrograms per kg on days 1 to 3 weekly for four weeks.","monitoring":"The label's warnings and overdosage sections describe drowsiness, stupor, coma, confusion, seizures and death with recommended doses and with overdose, most often after veterinary formulations; poisoning is managed with supportive care.","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd"},"toxicity":[{"event":"Pruritus (itching)","anyGradePct":27.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults, 100 to 200 micrograms per kg; Mazzotti reactions to dying parasites, not expected outside onchocerciasis"},{"event":"Skin oedema, papular, pustular or urticarial rash","anyGradePct":22.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults, 100 to 200 micrograms per kg; Mazzotti reactions to dying parasites, not expected outside onchocerciasis"},{"event":"Fever","anyGradePct":22.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults, 100 to 200 micrograms per kg; Mazzotti reactions to dying parasites, not expected outside onchocerciasis"},{"event":"Headache (regardless of cause)","anyGradePct":22.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults; the label judged 0.2 percent drug-related"},{"event":"Myalgia (regardless of cause)","anyGradePct":19.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults; the label judged 0.4 percent drug-related"},{"event":"Arthralgia or synovitis","anyGradePct":9.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults, 100 to 200 micrograms per kg; Mazzotti reactions to dying parasites, not expected outside onchocerciasis"},{"event":"Tachycardia (arrhythmia: fast heart rate)","anyGradePct":3.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults; judged possibly, probably or definitely drug-related"},{"event":"Peripheral oedema","anyGradePct":3.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults; judged possibly, probably or definitely drug-related"},{"event":"Facial oedema","anyGradePct":1.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults; judged possibly, probably or definitely drug-related"},{"event":"Orthostatic hypotension","anyGradePct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Onchocerciasis trials, 963 adults; judged possibly, probably or definitely drug-related"},{"event":"Decrease in white cell count","anyGradePct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Strongyloidiasis trials, 109 patients, one or two doses of 170 to 200 micrograms per kg; laboratory finding regardless of drug relationship"},{"event":"Dizziness (neurological, per label)","anyGradePct":2.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Strongyloidiasis trials, 109 patients, one or two doses of 170 to 200 micrograms per kg; judged drug-related"},{"event":"ALT or AST elevation","anyGradePct":2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Strongyloidiasis trials, 109 patients, one or two doses of 170 to 200 micrograms per kg; laboratory finding regardless of drug relationship"},{"event":"Diarrhoea","anyGradePct":1.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Strongyloidiasis trials, 109 patients, one or two doses of 170 to 200 micrograms per kg; judged drug-related"},{"event":"Nausea","anyGradePct":1.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Strongyloidiasis trials, 109 patients, one or two doses of 170 to 200 micrograms per kg; judged drug-related"},{"event":"Somnolence, vertigo or tremor (neurological, per label)","anyGradePct":0.9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Strongyloidiasis trials, 109 patients, one or two doses of 170 to 200 micrograms per kg; 0.9 percent each, judged drug-related"},{"event":"Neurotoxicity: altered consciousness, stupor, coma, confusion, seizures, death","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=681888c9-af79-4b7d-ae80-c3f4f6f1effd","note":"Warnings and overdosage sections; reported at recommended doses and in overdose, most often after veterinary formulations; no rate is given"}],"access":[],"regulatoryEvents":[]},{"id":"ivonescimab","kind":"drug","name":"Ivonescimab","aka":[],"tldr":"A Chinese bispecific that beat Keytruda head-to-head on progression-free survival in lung cancer, the first drug ever to do so.","summary":"HARMONi-2 (China): PFS 11.1 vs 5.8 months versus pembrolizumab in PD-L1+ first-line NSCLC. Approved in China (2024) for EGFR-mutant NSCLC after TKI (HARMONi-A) and first-line PD-L1+ NSCLC. Summit Therapeutics holds Western rights; HARMONi (global, EGFR-mutant post-TKI) met PFS but OS was not statistically significant in 2025; HARMONi-3 (squamous and non-squamous first-line) ongoing. Spawned a wave of PD-(L)1×VEGF bispecifics (Pfizer/3SBio, BMS/BioNTech, Merck/LaNova).","status":"approved","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Ivonescimab","url":"https://clinicaltrials.gov/search?intr=AK112"}],"tags":[],"related":[],"cancers":["nsclc","colorectal","tnbc","pdl1-high-nsclc"],"sections":[],"technologies":["bispecific-antibody","checkpoint-inhibitor"],"targets":["pd1","vegf"],"drugs":[],"companies":["akeso","summit-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07010263","nct06767527","nct06928389","nct06396065","nct06767514","nct05840016","nct06686030","nct05846867","nct06646055","nct07397338","nct07208773","nct05904379","nct07052253","nct07245446","nct06560112","nct07623356","nct05382442","nct05247684","nct07669779","nct06530251","nct07815665","nct06953999","nct06877650"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"AK112, SMT112","modality":"Bispecific antibody (PD-1×VEGF)","mechanism":"Tetravalent PD-1×VEGF-A bispecific with cooperative binding: VEGF binding increases PD-1 affinity, concentrating activity in tumour.","approvals":[{"region":"China","year":2024,"indication":"EGFR-mutant NSCLC after TKI (with chemotherapy); first-line PD-L1+ NSCLC"}],"mechanismSteps":["Tetravalent antibody binds VEGF-A in the tumour microenvironment","VEGF binding increases the antibody's affinity for PD-1 (cooperative binding), concentrating it in tumour","PD-1 blockade releases T cells; VEGF neutralisation normalises vessels and reduces immunosuppressive myeloid cells","Improved T-cell infiltration and killing"],"dosing":{"route":"IV infusion","schedule":"20 mg/kg every 3 weeks (China label; HARMONi-2)","modifications":"Standard immune-mediated event algorithm plus VEGF-class precautions (bleeding, hypertension, proteinuria)","monitoring":"Blood pressure, urine protein, thyroid, LFTs","source":"https://www.akesobio.com"},"toxicity":[{"event":"Proteinuria"},{"event":"Hypertension"},{"event":"Anaemia"},{"event":"AST increased"},{"event":"Hypothyroidism"},{"event":"Haemorrhage (low grade)"}],"access":[{"country":"CN","reimbursement":"NMPA approved 2024; NRDL inclusion 2025","asOf":"2026-09-06"},{"country":"US","reimbursement":"Investigational; BLA under review (PDUFA 14 November 2026)","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-05-24","type":"approval","region":"China","note":"EGFR-mutant NSCLC after TKI, with chemotherapy (HARMONi-A)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-04","type":"approval","region":"China","note":"First-line PD-L1+ NSCLC (HARMONi-2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-01","type":"filing","region":"US","note":"BLA submitted by Summit for EGFR-mutant NSCLC after TKI (HARMONi)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-11-14","type":"pdufa","region":"US","note":"PDUFA target date","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"ivosidenib","kind":"drug","name":"Ivosidenib","aka":[],"tldr":"The first drug to block a mutant metabolic enzyme in cancer. With azacitidine it tripled survival in IDH1-mutated AML that could not take intensive chemotherapy.","summary":"Approved 2018 (relapsed/refractory IDH1 AML), 2019 (newly diagnosed unfit, monotherapy), 2022 (with azacitidine, AGILE: EFS HR 0.33, OS 24.0 vs 7.9 months, HR 0.44), 2021 (IDH1 cholangiocarcinoma), and 2023 (IDH1 MDS). Servier acquired the Agios oncology portfolio in 2021. Differentiation syndrome is the class toxicity; isocitrate-dehydrogenase-2 isoform switching and second-site IDH1 mutations cause resistance.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Ivosidenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ivosidenib"},{"label":"NICE TA948: ivosidenib for IDH1 R132 cholangiocarcinoma (31 January 2024)","url":"https://www.nice.org.uk/guidance/ta948"}],"tags":[],"related":["idh1-r132"],"cancers":["aml","cholangiocarcinoma","aml-idh","intrahepatic-cholangiocarcinoma"],"sections":[],"technologies":["epigenetic-drugs","kinase-inhibitors","idh-inhibitors"],"targets":["idh"],"drugs":[],"companies":["servier"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome","crossover"],"trials":["agile","nct05876754","nct05907057","nct06127407","claridhy"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In cholangiocarcinoma: ClarIDHy showed PFS of 2.7 versus 1.4 months (HR 0.37) and a crossover-adjusted OS benefit in previously treated IDH1-mutant disease, approved in August 2021; the response rate was 2 percent, so disease stabilisation is the benefit. In ClarIDHy nausea affected 41 percent, diarrhoea 35 percent, fatigue 31 percent and grade 3 or higher ascites 9 percent.","Gallbladder cancer: 91 percent of ClarIDHy patients had intrahepatic cholangiocarcinoma (Tibsovo label), and IDH1 mutations are an intrahepatic feature, so ivosidenib has minimal evidence in gallbladder cancer; the licence wording is cholangiocarcinoma."],"brand":"Tibsovo","modality":"Small-molecule IDH1 inhibitor","mechanism":"Allosteric inhibitor of mutant IDH1; blocks 2-hydroxyglutarate production, restoring TET2-dependent demethylation and differentiation.","approvals":[{"region":"US","year":2018,"indication":"Relapsed/refractory IDH1-mutated AML"},{"region":"US","year":2019,"indication":"Newly diagnosed IDH1-mutated AML unfit for intensive chemotherapy"},{"region":"US","year":2021,"indication":"Previously treated IDH1-mutated cholangiocarcinoma"},{"region":"US","year":2022,"indication":"Newly diagnosed IDH1-mutated AML with azacitidine (AGILE)"},{"region":"US","year":2023,"indication":"Relapsed/refractory IDH1-mutated MDS"},{"region":"UK","year":2024,"indication":"Locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation after one or more systemic treatments; NICE TA948 recommended 31 January 2024 with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta948"}],"mechanismSteps":["Mutant IDH1 converts α-ketoglutarate to the oncometabolite 2-HG","Ivosidenib binds the mutant dimer interface and shuts off 2-HG production","TET2 and histone demethylases regain activity","Blasts differentiate into mature neutrophils (differentiation syndrome risk)","Combination with azacitidine deepens and prolongs remission"],"dosing":{"route":"Oral","schedule":"500 mg once daily with or without food (avoid high-fat meals), until progression; with azacitidine 75 mg/m² days 1-7 of 28","monitoring":"Differentiation syndrome (boxed warning), QT prolongation, leukocytosis, Guillain-Barré (rare)","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Tibsovo"},"toxicity":[{"event":"Differentiation syndrome","anyGradePct":14,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Tibsovo","note":"AGILE combination arm"},{"event":"QT prolongation","anyGradePct":20},{"event":"Neutropenia","grade3PlusPct":27,"note":"with azacitidine"},{"event":"Leukocytosis","anyGradePct":12}],"access":[],"regulatoryEvents":[{"date":"2018-07-20","type":"approval","region":"US","note":"First IDH1 inhibitor"},{"date":"2022-05-25","type":"approval","region":"US","note":"Combination with azacitidine (AGILE)"}]},{"id":"ivospemin","kind":"drug","name":"Ivospemin","aka":[],"tldr":"Ivospemin is an experimental small-molecule drug from Panbela Therapeutics in phase 3 trials for pancreatic ductal adenocarcinoma, with its target not yet stated publicly.","summary":"Ivospemin (SBP-101) is a small-molecule drug developed by Panbela Therapeutics. The sponsor describes its target as polyamine metabolism, which OnCo does not yet have a target page for. The sponsor states: A small-molecule polyamine metabolic inhibitor administered by subcutaneous injection. ClinicalTrials.gov describes the intervention as: small molecule polyamine metabolic inhibitor for subcutaneous injection. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05254171 (Study of Nab-Paclitaxel and Gemcitabine With or Without SBP-101 in Pancreatic Cancer), in pancreatic ductal adenocarcinoma. The largest, NCT05254171, plans to enrol 600 participants with primary completion was scheduled for 2026-08-29 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Ivospemin","url":"https://clinicaltrials.gov/search?intr=Ivospemin"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["panbela-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05254171"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"SBP-101","modality":"small molecule","mechanism":"A small-molecule polyamine metabolic inhibitor administered by subcutaneous injection.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ixabepilone","kind":"drug","name":"Ixabepilone","aka":[],"tldr":"Ixabepilone (Ixempra) is a chemotherapy infusion for breast cancer that has stopped responding to anthracyclines, taxanes and capecitabine.","summary":"Ixabepilone, a semi-synthetic epothilone, was approved by the FDA in October 2007 with capecitabine for metastatic or locally advanced breast cancer resistant to an anthracycline and a taxane (a 752-patient randomised trial showed longer progression-free survival than capecitabine alone) and as monotherapy after anthracycline, taxane and capecitabine failure. The EMA refused a marketing authorisation in 2008 on the balance of benefit and toxicity. Peripheral neuropathy is dose-limiting; the combination is contraindicated with hepatic impairment because of excess toxic deaths, and the Cremophor-based diluent requires premedication for hypersensitivity.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Ixabepilone","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ixabepilone"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/ixabepilone"}],"tags":["nci-list"],"related":[],"cancers":["breast-hr-positive","tnbc"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00630032","nct00883116","nct00998738"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ixempra","modality":"Epothilone B analogue (microtubule stabiliser)","mechanism":"Binds beta-tubulin and suppresses microtubule dynamics like a taxane but retains activity in cells with taxane-resistance mechanisms such as P-glycoprotein overexpression and beta-III tubulin.","approvals":[{"region":"US","year":2007,"indication":"Metastatic or locally advanced breast cancer after anthracycline and taxane (with capecitabine) or after anthracycline, taxane and capecitabine (monotherapy)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ixazomib","kind":"drug","name":"Ixazomib","aka":[],"tldr":"Ixazomib (Ninlaro) is the first oral proteasome inhibitor for multiple myeloma, enabling an all-oral triplet with lenalidomide and dexamethasone.","summary":"Ixazomib is an oral boronate proteasome inhibitor that reversibly blocks the beta-5 chymotrypsin-like site of the 20S proteasome, causing misfolded-protein stress and apoptosis in myeloma cells. Taken once weekly, it allows an all-oral triplet with lenalidomide and dexamethasone for patients with multiple myeloma after at least one prior therapy. TOURMALINE-MM1 (2016) showed median progression-free survival of 20.6 versus 14.7 months when ixazomib was added to lenalidomide-dexamethasone in relapsed disease, and approval followed in the US in 2015 and the EU in 2016. The maintenance trials TOURMALINE-MM3 and MM4 showed modest progression-free survival gains. It causes less neuropathy than bortezomib but is less potent than carfilzomib, so it is often chosen for convenience or frailty rather than depth of response. For a newcomer, ixazomib is the proteasome inhibitor you can take at home.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Ixazomib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ixazomib"}],"tags":["gap-fill"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":[],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04068597"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ninlaro","modality":"Oral proteasome inhibitor (boronate)","mechanism":"Reversible inhibitor of the β5 chymotrypsin-like site of the 20S proteasome; weekly oral dosing.","approvals":[{"region":"US","year":2015,"indication":"Multiple myeloma after ≥1 prior therapy, with lenalidomide and dexamethasone"},{"region":"EU","year":2016,"indication":"Same"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"izalontamab-brengitecan","kind":"drug","name":"Izalontamab brengitecan","aka":[],"tldr":"Izalontamab brengitecan is the first bispecific ADC to succeed in a phase 3 trial, hitting two growth receptors at once in triple-negative breast cancer.","summary":"EGFR×HER3 bispecific ADC from SystImmune (Sichuan Biokin), partnered with Bristol Myers Squibb (2023, up to $8.4B). Phase 3 BL-B01D1-307 in previously treated locally advanced/metastatic TNBC met PFS and OS at interim analysis (announced 26 February 2026; presented ASCO 2026). Also positive in oesophageal squamous cell carcinoma. IZABRIGHT-Breast01 (first-line TNBC, PD-1 ineligible) and trials in NSCLC (EGFR-mutant, post-TKI), urothelial, nasopharyngeal, and SCLC are ongoing. Haematologic toxicity is the main limitation.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"OncLive report","url":"https://www.onclive.com/view/iza-bren-yields-pfs-and-os-benefits-vs-chemo-in-previously-treated-advanced-tnbc"}],"tags":[],"related":[],"cancers":["tnbc","esophageal","nsclc","urothelial"],"sections":[],"technologies":["bispecific-adc","adc"],"targets":["egfr","her3"],"drugs":[],"companies":["systimmune","bms"],"institutions":[],"pathways":[],"terms":["hydrophilic-next-gen"],"trials":["bl-b01d1-307","izabright-breast01","nct06838273","nct07502300","nct06382129","nct07640789","nct06994195","nct06118333","nct07642024","nct07100080","nct06500026","nct06382116","nct06475300","nct06498986","nct06437522","nct07054567","nct05880706","nct05956587","nct05990803","nct06006169","nct05924841","nct06008054","nct06437509","nct06787664"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"iza-bren, BL-B01D1","modality":"Bispecific ADC","payload":"Ed-04 (camptothecin-derived TOP1 inhibitor), DAR ~8","linker":"Cleavable","mechanism":"Dual EGFR/HER3 binding drives internalisation across heterogeneous tumours; TOP1 payload with bystander effect.","approvals":[],"mechanismSteps":["Antibody binds EGFR and HER3 (bispecific) on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","Ed-04 (camptothecin-derived TOP1 inhibitor) is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"2.5 mg/kg on days 1 and 8 of each 21-day cycle (phase 3 TNBC regimen)","monitoring":"Blood counts (neutropenia, anaemia, thrombocytopenia), stomatitis, ILD symptoms","source":"https://clinicaltrials.gov/study/NCT06382142"},"toxicity":[{"event":"Neutropenia","note":"Haematologic toxicity is the main limitation in phase 1-3 reports; rates per ASCO 2026 presentation"},{"event":"Anaemia"},{"event":"Thrombocytopenia"},{"event":"Stomatitis"},{"event":"Nausea"}],"access":[{"country":"US","reimbursement":"Investigational; BMS filing expected after BL-B01D1-307","asOf":"2026-09-06"},{"country":"CN","reimbursement":"NMPA filings under review (2026)","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-12-11","type":"filing","region":"US","note":"BMS licenses global rights ex-China from SystImmune (up to $8.4B)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-02-26","type":"filing","region":"China","note":"Positive interim phase 3 in pretreated TNBC (BL-B01D1-307) announced; regulatory submissions follow","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"jbi-802","kind":"drug","name":"JBI-802","aka":[],"tldr":"JBI-802 is a small-molecule inhibitor from Jubilant Therapeutics Inc., in registered phase 2 trials for myeloproliferative neoplasms, chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms.","summary":"JBI-802 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Jubilant Therapeutics Inc., in myeloproliferative neoplasms, chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms. Described in the registry record as a corest inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of JBI-802","url":"https://clinicaltrials.gov/search?intr=JBI-802"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["myeloproliferative-neoplasms","cmml"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["jubilant-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07612280"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"JBI-802","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a corest inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jdq443","kind":"drug","name":"JDQ443","aka":[],"tldr":"JDQ443 is an experimental small-molecule drug from Novartis Pharmaceuticals in phase 3 trials for non-small-cell lung cancer and colorectal cancer, aimed at KRAS.","summary":"JDQ443 is a small-molecule drug developed by Novartis Pharmaceuticals. Its target is KRAS (the sponsor names KRAS G12C). ClinicalTrials.gov describes the intervention as: JDQ443 per os (PO) 200 mg twice a day continuously. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05132075 (Study of JDQ443 in Comparison With Docetaxel in Participants With Locally Advanced or Metastatic KRAS G12C Mutant Non-small Cell Lung Cancer), in non-small-cell lung cancer and colorectal cancer. The largest, NCT04699188, plans to enrol 344 participants (actual) with primary completion expected 2027-01-25. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JDQ443","url":"https://clinicaltrials.gov/search?intr=JDQ443"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","colorectal"],"sections":[],"technologies":[],"targets":["kras"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05132075","nct05358249","nct05445843","nct04699188"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Small-molecule drug directed at KRAS, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jk08","kind":"drug","name":"JK08","aka":[],"tldr":"JK08 is an experimental fusion protein or cytokine from Salubris Biotherapeutics in phase 2 trials for melanoma, colorectal cancer and non-small-cell lung cancer, aimed at CTLA-4.","summary":"JK08 is a fusion protein or cytokine developed by Salubris Biotherapeutics. Its target is CTLA-4. ClinicalTrials.gov describes the intervention as: Recombinant fusion protein consisting of two functional elements, which are a fully human monoclonal antibody, directed against CTLA-4 and a protein complex formed by the human IL-15 and the Sushi domain of human IL-15Rα. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in melanoma, colorectal cancer, non-small-cell lung cancer, bladder & urothelial cancer and head and neck squamous cell carcinoma. The largest, NCT05620134, plans to enrol 263 participants with primary completion was scheduled for 2025-10-17 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JK08","url":"https://clinicaltrials.gov/search?intr=JK08"},{"label":"ClinicalTrials.gov NCT05620134","url":"https://clinicaltrials.gov/study/NCT05620134"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma","colorectal","nsclc","urothelial","head-and-neck","breast-hr-positive","tnbc","rcc","gastric","pancreatic","hcc","ovarian","thyroid"],"sections":[],"technologies":[],"targets":["ctla4"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct05620134"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["JK08 is a recombinant fusion protein of a fully human anti-CTLA-4 antibody with a complex of human IL-15 and the sushi domain of IL-15 receptor alpha, so it blocks a checkpoint and delivers a T-cell growth signal from the same molecule (registry intervention description, NCT05620134). The IL-15 half has no target record in the corpus."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"fusion protein or cytokine","mechanism":"Fusion protein or cytokine directed at CTLA-4, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jmt101","kind":"drug","name":"JMT101","aka":[],"tldr":"JMT101 is an experimental monoclonal antibody from Shanghai JMT-Bio in phase 3 trials for non-small-cell lung cancer, aimed at EGFR.","summary":"JMT101 is a monoclonal antibody developed by Shanghai JMT-Bio. Its target is EGFR (the sponsor names EGFR). The sponsor states: JMT101 is a recombinant humanised anti-EGFR monoclonal antibody, given intravenously (6 mg/kg every two weeks) in combination with osimertinib versus osimertinib alone in EGFR-mutant non-small cell lung cancer. ClinicalTrials.gov describes the intervention as: JMT101 is a recombinant humanized anti-EGFR monoclonal antibody. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06735391 (A Clinical Study of JMT101 in Combination With Osimertinib Versus Osimertinib Alone as First-Line Treatment for Patients With Locally Advanced or Metastatic Non-Squamous Non-Small Cell Lung Cancer (NSCLC) Harboring Epidermal Growth Factor Receptor (EGFR) Sensitive Mutations), in non-small-cell lung cancer. The largest, NCT06735391, plans to enrol 516 participants with primary completion expected 2026-09-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JMT101","url":"https://clinicaltrials.gov/search?intr=JMT101"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["egfr"],"drugs":[],"companies":["cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06735391"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"JMT101 is a recombinant humanised anti-EGFR monoclonal antibody, given intravenously (6 mg/kg every two weeks) in combination with osimertinib versus osimertinib alone in EGFR-mutant non-small cell lung cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jmt203","kind":"drug","name":"JMT203","aka":[],"tldr":"JMT203 is a monoclonal antibody from Shanghai JMT-Bio Inc., in registered phase 2 trials for non-small-cell lung cancer, pancreatic ductal adenocarcinoma, colorectal cancer.","summary":"JMT203 is listed on ClinicalTrials.gov as an intervention in 2 registered phase 2 trials sponsored by Shanghai JMT-Bio Inc., in non-small-cell lung cancer, pancreatic ductal adenocarcinoma, colorectal cancer. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of JMT203","url":"https://clinicaltrials.gov/search?intr=JMT203"},{"label":"ClinicalTrials.gov NCT06868849","url":"https://clinicaltrials.gov/study/NCT06868849"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc","pancreatic","colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06868849","nct06868849"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["JMT203 is described in its registry record as an anti-GFRAL monoclonal antibody given subcutaneously for cancer cachexia; GFRAL is the brainstem receptor for GDF15, the signal that drives appetite loss and weight loss in advanced cancer. GFRAL has no target record in the corpus yet."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"JMT203","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jnj-79635322","kind":"drug","name":"JNJ-79635322","aka":[],"tldr":"JNJ-79635322 is an experimental investigational agent whose form is not stated in the registry from Janssen Research & Development in phase 3 trials for multiple myeloma, with its target not yet stated publicly.","summary":"JNJ-79635322 is an investigational agent whose form is not stated in the registry developed by Janssen Research & Development. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: JNJ-79635322 will be administered as SC injection. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07258511 (A Study Comparing JNJ-79635322 and an Anti-B-cell Maturation Antigen (BCMA)xCD3 Bispecific Antibody in Participants With Relapsed or Refractory Multiple Myeloma) and NCT07518186 (A Study Comparing JNJ-79635322 and Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma), in multiple myeloma. The largest, NCT07518186, plans to enrol 700 participants with primary completion expected 2029-04-29. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JNJ-79635322","url":"https://clinicaltrials.gov/search?intr=JNJ-79635322"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07258511","nct07518186","nct07266441","nct07589634"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"trispecific antibody (BCMA x GPRC5D x CD3, per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jnj-87189401","kind":"drug","name":"JNJ-87189401","aka":[],"tldr":"JNJ-87189401 is an experimental costimulatory agent from Janssen Research & Development in phase 3 trials for prostate cancer, aimed at PSMA.","summary":"JNJ-87189401 is a costimulatory agent developed by Janssen Research & Development. Its target is PSMA (the sponsor names PSMA / CD28). The sponsor states: A PSMA-CD28 costimulatory agent intended to enhance T-cell activation via PSMA engagement plus CD28 costimulation. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07164443 (A Study of Pasritamig With or Without JNJ-87189401 Versus Placebo for Late Line Metastatic Castration-resistant Prostate Cancer (mCRPC)), in prostate cancer. The largest, NCT07164443, plans to enrol 1203 participants with primary completion expected 2028-08-18. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JNJ-87189401","url":"https://clinicaltrials.gov/search?intr=JNJ-87189401"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["psma","cd28"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["mcrpc"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"costimulatory bispecific antibody (PSMA x CD28, per the sponsor's pipeline page)","mechanism":"A PSMA-CD28 costimulatory agent intended to enhance T-cell activation via PSMA engagement plus CD28 costimulation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jnj-90014496","kind":"drug","name":"JNJ-90014496","aka":[],"tldr":"JNJ-90014496 is an experimental CAR-T cell therapy from Janssen Research & Development in phase 2 trials for hodgkin lymphoma and diffuse large B-cell lymphoma, aimed at CD19 and CD20.","summary":"JNJ-90014496 is a CAR-T cell therapy developed by Janssen Research & Development. Its targets are CD19 and CD20 (the sponsor names CD20 x CD19). The sponsor states: An autologous dual-targeting chimeric antigen receptor (CAR) T-cell therapy directed against both CD20 and CD19, enabling the engineered T cells to attack lymphoma cells expressing either or both antigens. ClinicalTrials.gov describes the intervention as: JNJ-90014496, an autologous dual targeting chimeric antigen receptor (CAR) - T cell therapy targeting Cluster of differentiation (CD)20 and CD19. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in hodgkin lymphoma and diffuse large B-cell lymphoma. The largest, NCT05421663, plans to enrol 166 participants (actual) with primary completion expected 2041-07-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JNJ-90014496","url":"https://clinicaltrials.gov/search?intr=JNJ-90014496"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma","dlbcl"],"sections":[],"technologies":[],"targets":["cd19","cd20"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05421663"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"An autologous dual-targeting chimeric antigen receptor (CAR) T-cell therapy directed against both CD20 and CD19, enabling the engineered T cells to attack lymphoma cells expressing either or both antigens.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jnj-90301900","kind":"drug","name":"JNJ-90301900","aka":["JNJ-90301900 (NBTXR3)","NBTXR3"],"tldr":"JNJ-90301900 is an experimental radioenhancer nanoparticle from Johnson & Johnson Enterprise Innovation in phase 3 trials for head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"JNJ-90301900 is a radioenhancer nanoparticle developed by Johnson & Johnson Enterprise Innovation. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. The sponsor states: A suspension of inert, crystalline hafnium oxide nanoparticles designed to generate oxygen free radicals that destroy cancer cells once activated by ionising radiation. ClinicalTrials.gov describes the intervention as: Suspension of inert, crystalline hafnium oxide particles, designed to generate oxygen free radicals to destroy cancer cells after activation by ionizing radiation. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04892173 (JNJ-90301900 (NBTXR3) Activated by Radiotherapy With or Without Cetuximab in LA-HNSCC), in head and neck squamous cell carcinoma. The largest, NCT04892173, plans to enrol 500 participants with primary completion expected 2028-06-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JNJ-90301900","url":"https://clinicaltrials.gov/search?intr=JNJ-90301900"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04892173","nct06667908"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"radioenhancer nanoparticle","mechanism":"A suspension of inert, crystalline hafnium oxide nanoparticles designed to generate oxygen free radicals that destroy cancer cells once activated by ionising radiation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"js105","kind":"drug","name":"JS105","aka":[],"tldr":"JS105 is an experimental small-molecule drug from Risen (Suzhou) Pharma Tech in phase 3 trials for HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"JS105 is a small-molecule drug developed by Risen (Suzhou) Pharma Tech. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Patients will receive oral JS105 on Days 1-28 of each 4-week cycle. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07207070 (A Randomised, Open-label, Multicentre Phase III Clinical Study to Evaluate the Efficacy and Safety of JS105 Combined With Dalpiciclib and Fulvestrant Compared With Dalpiciclib and Fulvestrant in Patients With PIK3CA-mutated, HR-positive, HER2-negative Recurrent or Metastatic Breast Cancer), in HR-positive / HER2-negative breast cancer. The largest, NCT07207070, plans to enrol 312 participants with primary completion expected 2029-05-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JS105","url":"https://clinicaltrials.gov/search?intr=JS105"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["junshi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07207070"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"js111","kind":"drug","name":"JS111","aka":[],"tldr":"JS111 is an experimental small-molecule drug from Suzhou Junjing BioSciences in phase 2 trials for non-small-cell lung cancer, aimed at EGFR and MET.","summary":"JS111 is a small-molecule drug developed by Suzhou Junjing BioSciences. Its targets are EGFR and MET (the sponsor names EGFR). The sponsor states: Oral EGFR tyrosine kinase inhibitor (capsule) targeting EGFR exon 19 deletion and L858R sensitising mutations in NSCLC, dosed once daily; an active metabolite (M546b) is also tracked. ClinicalTrials.gov describes the intervention as: In Phase I, approximately 3-12 subjects will be enrolled in each dose group (160 mg QD or 240 mg QD) and receive oral JS111 capsules once daily until any treatment discontinuation criteria are met. After all subjects have completed at least. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT06940401, plans to enrol 44 participants (actual) with primary completion expected 2028-07-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JS111","url":"https://clinicaltrials.gov/search?intr=JS111"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["egfr","met"],"drugs":[],"companies":["junshi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06940401"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Oral EGFR tyrosine kinase inhibitor (capsule) targeting EGFR exon 19 deletion and L858R sensitising mutations in NSCLC, dosed once daily; an active metabolite (M546b) is also tracked.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jskn003","kind":"drug","name":"JSKN003","aka":[],"tldr":"JSKN003 is an experimental investigational agent whose form is not stated in the registry from Jiangsu Alphamab Biopharmaceuticals in phase 3 trials for HR-positive / HER2-negative breast cancer and HER2-positive breast cancer, with its target not yet stated publicly.","summary":"JSKN003 is an investigational agent whose form is not stated in the registry developed by Jiangsu Alphamab Biopharmaceuticals and Shanghai JMT-Bio. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: JSKN003 should be administered intravenously on the first day of each 3-week cycle. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06079983 (JSKN003 Versus Treatment Of Physician'S Choice For HER2-low, Unresectable and/or Metastatic Breast Cancer Subjects) and NCT06846437 (JSKN003 Versus Trastuzumab Emtansine (T-DM1) for HER2-Positive, Advanced Breast Cancer), in HR-positive / HER2-negative breast cancer and HER2-positive breast cancer. The largest, NCT05744427, plans to enrol 725 participants with primary completion was scheduled for 2026-07-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JSKN003","url":"https://clinicaltrials.gov/search?intr=JSKN003"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","breast-her2-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["alphamab","cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06079983","nct06846437","nct05744427"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jskn016","kind":"drug","name":"JSKN016","aka":[],"tldr":"JSKN016 is an experimental investigational agent whose form is not stated in the registry from Jiangsu Alphamab Biopharmaceuticals in phase 3 trials for triple-negative breast cancer and HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"JSKN016 is an investigational agent whose form is not stated in the registry developed by Jiangsu Alphamab Biopharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: JSKN016 is administered via intravenous infusion at doses of 5mg/kg or 6mg/kg every 3 weeks, starting on Day 1 of each cycle. If the 5mg/kg dose is well tolerated during the safety lead-in phase, the dose may be increased to 6mg/kg for subs. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07533123 (A Phase III Study of JSKN016 Versus Treatment of Physician's Choice in Patients With Triple-Negative Breast Cancer Who Have Failed Standard of Care), in triple-negative breast cancer and HR-positive / HER2-negative breast cancer. The largest, NCT07533123, plans to enrol 364 participants with primary completion expected 2029-03-17. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JSKN016","url":"https://clinicaltrials.gov/search?intr=JSKN016"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["tnbc","breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["alphamab"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07533123","nct06942234"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"jyp0015","kind":"drug","name":"JYP0015","aka":[],"tldr":"JYP0015 is an experimental small-molecule drug from Guangzhou JOYO Pharma in phase 2 trials for pancreatic ductal adenocarcinoma, non-small-cell lung cancer and colorectal cancer, aimed at KRAS.","summary":"JYP0015 is a small-molecule drug developed by Guangzhou JOYO Pharma. Its target is KRAS (the sponsor names KRAS, NRAS, HRAS (pan-RAS)). The sponsor states: An orally bioavailable pan-RAS inhibitor designed to selectively target the active (ON) form of wild-type and mutant RAS across KRAS, NRAS and HRAS isoforms, including mutations at codons 12, 13, 61, 117 and 146. ClinicalTrials.gov describes the intervention as: JYP0015 is an orally bioavailable pan-RAS inhibitor designed to target the active (ON) form of wild-type and mutant RAS across KRAS, NRAS, and HRAS isoforms. The drug will be administered orally, with dosing determined by the study protocol. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in pancreatic ductal adenocarcinoma, non-small-cell lung cancer and colorectal cancer. The largest, NCT06895031, plans to enrol 210 participants with primary completion was scheduled for 2026-03-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of JYP0015","url":"https://clinicaltrials.gov/search?intr=JYP0015"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic","nsclc","colorectal"],"sections":[],"technologies":[],"targets":["kras","nras"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06895031"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"An orally bioavailable pan-RAS inhibitor designed to selectively target the active (ON) form of wild-type and mutant RAS across KRAS, NRAS and HRAS isoforms, including mutations at codons 12, 13, 61, 117 and 146.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"kc1036","kind":"drug","name":"KC1036","aka":[],"tldr":"KC1036 is an experimental small-molecule drug from Beijing Konruns Pharmaceutical in phase 3 trials for oesophageal cancer, ewing sarcoma and thymoma and thymic carcinoma, with its target not yet stated publicly.","summary":"KC1036 is a small-molecule drug developed by Beijing Konruns Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: For patients weighing between 30 kg and less than 50 kg, administer 40 mg QD of KC1036. For patients weighing between 50 kg and less than 70 kg, administer 50 mg QD of KC1036. For patients weighing 70 kg or more, administer 60 mg QD of KC10. It is the investigational product in 5 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06194734 (A Study of KC1036 Versus Investigator's Choice of Chemotherapy in Patients With Advanced Oesophageal Cancer), in oesophageal cancer, ewing sarcoma and thymoma and thymic carcinoma. The largest, NCT06194734, plans to enrol 490 participants with primary completion expected 2026-12-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of KC1036","url":"https://clinicaltrials.gov/search?intr=KC1036"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["esophageal","ewing-sarcoma","thymic-epithelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["shanghai-kechow-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06194734"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"kl-a167","kind":"drug","name":"KL-A167","aka":[],"tldr":"KL-A167 is an experimental investigational agent whose form is not stated in the registry from Sichuan Kelun-Biotech Biopharmaceutical in phase 3 trials for nasopharyngeal carcinoma and non-small-cell lung cancer, with its target not yet stated publicly.","summary":"KL-A167 is an investigational agent whose form is not stated in the registry developed by Sichuan Kelun-Biotech Biopharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: KL-A167 4-6 cycles for combined therapy.KL-A167 maintenance. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05294172 (KL-A167 Injection Combined With Cisplatin and Gemcitabine vs Placebo Combined With Cisplatin and Gemcitabine in the Treatment of Recurrent or Metastatic Nasopharyngeal Carcinoma), in nasopharyngeal carcinoma and non-small-cell lung cancer. The largest, NCT05294172, plans to enrol 295 participants (actual) with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of KL-A167","url":"https://clinicaltrials.gov/search?intr=KL-A167"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nasopharyngeal","nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["kelun-biotech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05294172","nct07296809","nct05351788"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody (KL-A167 is tagitanlimab; INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"kn026","kind":"drug","name":"KN026","aka":[],"tldr":"KN026 is an experimental monoclonal antibody from Shanghai JMT-Bio in phase 3 trials for HER2-positive breast cancer and HR-positive / HER2-negative breast cancer, aimed at HER2.","summary":"KN026 is a monoclonal antibody developed by Shanghai JMT-Bio and CSPC Megalith Biopharmaceutical. Its target is HER2. ClinicalTrials.gov describes the intervention as: KN026 is an antibody targeting HER2, administered via intravenous infusion. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07441460 (A Phase III Study of KN026 in Combination With HB1801 as Adjuvant Therapy for Resectable HER2-Positive Breast Cancer) and NCT06747338 (A Phase III Study of KN026 in Combination With HB1801 ± Carboplatin as Neoadjuvant Treatment for Early or Locally Advanced HER2-Positive Breast Cancer), in HER2-positive breast cancer and HR-positive / HER2-negative breast cancer. The largest, NCT07441460, plans to enrol 1800 participants with primary completion expected 2034-08-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of KN026","url":"https://clinicaltrials.gov/search?intr=KN026"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-her2-positive","breast-hr-positive"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07441460","nct06747338","nct07597629"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at HER2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"krt-232","kind":"drug","name":"KRT-232","aka":[],"tldr":"KRT-232 is an experimental small-molecule drug from Kartos Therapeutics in phase 2 trials for myeloproliferative neoplasms, diffuse large B-cell lymphoma and chronic lymphocytic leukaemia, aimed at MDM2 and TP53.","summary":"KRT-232 is a small-molecule drug developed by Kartos Therapeutics. Its targets are MDM2 and TP53 (the sponsor names MDM2-p53 interaction). The sponsor states: A novel, potent and selective oral MDM2 inhibitor, referred to on the current site as navtemadlin. ClinicalTrials.gov describes the intervention as: KRT-232 is an experimental MDM2 inhibitor anticancer drug taken by mouth. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in myeloproliferative neoplasms, diffuse large B-cell lymphoma, chronic lymphocytic leukaemia and hodgkin lymphoma. The largest, NCT04502394, plans to enrol 84 participants with primary completion was scheduled for 2022-10 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of KRT-232","url":"https://clinicaltrials.gov/search?intr=KRT-232"},{"label":"Sponsor page","url":"https://www.kartosthera.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["myeloproliferative-neoplasms","dlbcl","cll","hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":["mdm2","tp53"],"drugs":[],"companies":["kartos-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05797831","nct04502394","nct03669965"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"A novel, potent and selective oral MDM2 inhibitor, referred to on the current site as navtemadlin.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lam561","kind":"drug","name":"LAM561","aka":[],"tldr":"LAM561 is an experimental small-molecule drug from Laminar Pharmaceuticals in phase 3 trials for glioma & glioblastoma, aimed at MET.","summary":"LAM561 is a small-molecule drug developed by Laminar Pharmaceuticals. Its target is MET (the sponsor names membrane lipids). The sponsor states: A hydroxylated lipid derivative that targets membrane lipids and induces cytocidal autophagy in cancer models; in Phase 2b/3 testing for newly diagnosed, MGMT-methylated glioblastoma. ClinicalTrials.gov describes the intervention as: Once a patient is allocated a dose of LAM561, they will receive the same dose on a daily basis in treatment cycles of 21 days (3 weeks), which may be repeated without therapy interruption until a criterion for discontinuation (clinical or r. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT04250922 (LAM561 With RT and TMZ for Adults With Glioblastoma), in glioma & glioblastoma. The largest, NCT04250922, plans to enrol 144 participants (actual) with primary completion was scheduled for 2026-01-15 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of LAM561","url":"https://clinicaltrials.gov/search?intr=LAM561"},{"label":"Sponsor page","url":"https://www.laminarpharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":[],"targets":["met"],"drugs":[],"companies":["laminar-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04250922","nct04299191"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"A hydroxylated lipid derivative that targets membrane lipids and induces cytocidal autophagy in cancer models; in Phase 2b/3 testing for newly diagnosed, MGMT-methylated glioblastoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lapatinib","kind":"drug","name":"Lapatinib","aka":[],"tldr":"Lapatinib was the first HER2-blocking pill (2007) and is now mostly a comparator arm and a late-line option, displaced by tucatinib and ADCs.","summary":"Lapatinib is a reversible, ATP-competitive dual inhibitor of the EGFR and HER2 kinases, and was the first HER2-blocking pill (2007). It is approved with capecitabine for HER2-positive advanced breast cancer after trastuzumab (EGF100151: time to progression 8.4 versus 4.4 months) and with letrozole in hormone receptor-positive, HER2-positive disease. The adjuvant ALTTO trial was negative and neoadjuvant NeoALTTO raised pathological complete response but not survival, so it never established a role in early disease. Diarrhoea and rash are the main toxicities, with a hepatotoxicity warning. It has been displaced by tucatinib and by antibody-drug conjugates and now serves mainly as the control arm in ACE-Breast-02 and other Chinese ADC trials. For a newcomer, lapatinib is the original HER2 pill, now mostly a comparator and a late-line option.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Lapatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lapatinib"}],"tags":[],"related":[],"cancers":["breast-her2-positive","her2-positive-breast-brain-metastases","colorectal"],"sections":[],"technologies":["kinase-inhibitors","her2-tyrosine-kinase-inhibitors"],"targets":["her2","egfr"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["ace-breast-02","nct06313983"],"people":[],"bottlenecks":[],"keyPapers":["paper-sartore-bianchi-heracles-trastuzumab-lapatinib-lancet-oncol-2016"],"journals":[],"dependsOn":[],"notes":[],"brand":"Tykerb","modality":"Small-molecule reversible HER2/EGFR kinase inhibitor","mechanism":"Reversible dual EGFR/HER2 ATP-competitive inhibitor.","approvals":[{"region":"US","year":2007,"indication":"HER2+ advanced breast cancer with capecitabine after trastuzumab"},{"region":"US","year":2010,"indication":"HR+/HER2+ metastatic breast cancer with letrozole"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"1250 mg once daily with capecitabine; 1500 mg with letrozole","monitoring":"Liver function, LVEF"},"toxicity":[{"event":"Diarrhoea","anyGradePct":65,"grade3PlusPct":13},{"event":"Palmar-plantar erythrodysaesthesia (with capecitabine)","anyGradePct":53},{"event":"Rash","anyGradePct":28}],"access":[],"regulatoryEvents":[{"date":"2010-01-29","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with letrozole for post-menopausal women with HR-positive metastatic breast cancer that overexpresses the HER2 receptor for whom hormonal therapy is indicated"},{"date":"2018-12-06","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2010 converted to traditional approval 8.9 years after it was granted.","indication":"In combination with letrozole for post-menopausal women with HR-positive metastatic breast cancer that overexpresses the HER2 receptor for whom hormonal therapy is indicated"}]},{"id":"larotrectinib","kind":"drug","name":"Larotrectinib","aka":[],"tldr":"The first drug approved for a gene fusion regardless of where the cancer started; it works in about 75% of NTRK-fusion cancers, from infant fibrosarcoma to salivary and thyroid cancers.","summary":"Drilon (NEJM 2018): 75% response across 17 tumour types; approved November 2018 as the second tumour-agnostic approval (after pembrolizumab MSI-H). Long durability (median DOR >40 months in updates); resistance via NTRK solvent-front mutations (G595R) addressed by repotrectinib and next-generation TRK inhibitors. Paediatric formulation.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Larotrectinib","links":[{"label":"Drilon 2018 (NEJM)","url":"https://doi.org/10.1056/NEJMoa1714448"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=larotrectinib"},{"label":"NICE TA630: larotrectinib for NTRK fusion-positive solid tumours, Cancer Drugs Fund (27 May 2020)","url":"https://www.nice.org.uk/guidance/ta630"},{"label":"EMA Vitrakvi product page","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/vitrakvi"}],"tags":["gap-fill"],"related":["ntrk-fusion"],"cancers":["salivary-gland","thyroid","colorectal","nsclc","sarcoma","pancreatic","kras-wild-type-pdac","ntrk-fusion-nsclc"],"sections":[],"technologies":["kinase-inhibitors","cgp"],"targets":["ntrk"],"drugs":[],"companies":["bayer"],"institutions":[],"pathways":[],"terms":["tumour-agnostic","gene-fusion"],"trials":["nct02637687"],"people":[],"bottlenecks":[],"keyPapers":["paper-oreilly-hechtman-ntrk-fusion-pancreatic-larotrectinib-ann-oncol-2019"],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: NTRK fusions are rare drivers of KRAS wild-type pancreatic cancer; NICE NG85 (1.9.11) points to TA630, which makes larotrectinib available through the Cancer Drugs Fund while more data are collected. EU marketing authorisation 19 September 2019 (Vitrakvi)."],"brand":"Vitrakvi","modality":"Small-molecule pan-TRK inhibitor (first generation)","mechanism":"Highly selective ATP-competitive inhibitor of TRKA/B/C (NTRK1/2/3) fusion kinases.","approvals":[{"region":"US","year":2018,"indication":"Solid tumours with NTRK gene fusion without known acquired resistance mutation, metastatic or unresectable, adults and children"},{"region":"EU","year":2019,"indication":"Same"},{"region":"UK","year":2020,"indication":"NTRK fusion-positive solid tumours in adults and children, locally advanced or metastatic or where surgery would cause severe morbidity, with no satisfactory treatment options: NICE TA630 (27 May 2020) recommends it within the Cancer Drugs Fund under a managed access agreement","note":"https://www.nice.org.uk/guidance/ta630"},{"region":"England (NICE)","year":2020,"indication":"NTRK fusion-positive solid tumours, including colorectal cancer","note":"TA630, published 27 May 2020: recommended for use within the Cancer Drugs Fund only, under a managed access agreement, where there is no satisfactory treatment option."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2018-11-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/fda-approves-larotrectinib-solid-tumors-ntrk-gene-fusions-0","indication":"Treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation; are metastatic or where surgical resection is likely to result in severe morbidity; and have no satisfactory alternative treatments or that have progressed following treatment"},{"date":"2018-11-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/fda-approves-larotrectinib-solid-tumors-ntrk-gene-fusions-0","indication":"Formation: Treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation; are metastatic or where surgical resection is likely to result in severe morbidity; and have no satisfactory alternative treatments or that have progressed following treatment"},{"date":"2025-04-09","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 6.4 years after it was granted.","source":"https://www.fda.gov/drugs/fda-approves-larotrectinib-solid-tumors-ntrk-gene-fusions-0","indication":"Treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation; are metastatic or where surgical resection is likely to result in severe morbidity; and have no satisfactory alternative treatments or that have progressed following treatment"},{"date":"2025-04-09","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 6.4 years after it was granted.","source":"https://www.fda.gov/drugs/fda-approves-larotrectinib-solid-tumors-ntrk-gene-fusions-0","indication":"Formation: Treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation; are metastatic or where surgical resection is likely to result in severe morbidity; and have no satisfactory alternative treatments or that have progressed following treatment"}]},{"id":"lazertinib","kind":"drug","name":"Lazertinib","aka":[],"tldr":"A third-generation EGFR pill used together with amivantamab as the first regimen to beat osimertinib in EGFR-mutant lung cancer.","summary":"Developed by Yuhan, licensed to Janssen. Approved August 2024 only in combination with amivantamab (MARIPOSA: PFS HR 0.70; OS HR 0.75 with median >12 months longer than osimertinib, 2025). Rash, paronychia, and venous thromboembolism (prophylactic anticoagulation for 4 months) are the combination's main burdens.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Lazertinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lazertinib"},{"label":"NICE TA1122: amivantamab with lazertinib for untreated EGFR mutation-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1122"}],"tags":[],"related":["egfr-l858r"],"cancers":["nsclc","egfr-mutant-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":["amivantamab"],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["mariposa","nct05388669","nct05498428","nct05663866","nct06667076","nct07586202","nct06120140","nct04988295","nct05541822"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lazcluze","modality":"Small-molecule kinase inhibitor (EGFR)","mechanism":"Irreversible mutant-selective EGFR TKI (exon 19 del, L858R, T790M), CNS-penetrant.","approvals":[{"region":"US","year":2024,"indication":"First-line EGFR exon 19 del / L858R NSCLC with amivantamab"},{"region":"EU","year":2025,"indication":"1L EGFR-mutant NSCLC with amivantamab; 20 Jan 2025"},{"region":"England (NICE)","year":2026,"indication":"Untreated advanced EGFR mutation-positive non-small-cell lung cancer (exon 19 deletion or L858R), with amivantamab","note":"TA1122, published 21 January 2026, subject to the commercial arrangements for both drugs."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lb1410","kind":"drug","name":"LB1410","aka":[],"tldr":"LB1410 is an experimental bispecific antibody from L & L Bio, Ningbo, China in phase 2 trials, aimed at PD-1 and TIM-3.","summary":"LB1410 is a bispecific antibody developed by L & L Bio, Ningbo, China. Its targets are PD-1 and TIM-3 (the sponsor names PD-1 x TIM3). The sponsor states: Anti-PD-1/TIM3 bispecific antibody intended to block dual immune checkpoint pathways, given in combination with LB4330. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT06468358, plans to enrol 194 participants with primary completion expected 2026-12-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of LB1410","url":"https://clinicaltrials.gov/search?intr=LB1410"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["pd1","tim3"],"drugs":[],"companies":["hutchmed"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06468358"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"Anti-PD-1/TIM3 bispecific antibody intended to block dual immune checkpoint pathways, given in combination with LB4330.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lb4330","kind":"drug","name":"LB4330","aka":[],"tldr":"LB4330 is an experimental fusion protein from L & L Bio, Ningbo, China in phase 2 trials, aimed at Claudin 18.2.","summary":"LB4330 is a fusion protein developed by L & L Bio, Ningbo, China. Its target is Claudin 18.2 (the sponsor names Claudin18.2 x IL-10). The sponsor states: Anti-Claudin18.2/IL-10 fusion protein combining tumour-antigen targeting with an immunomodulatory cytokine, given in combination with LB1410. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT06468358, plans to enrol 194 participants with primary completion expected 2026-12-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of LB4330","url":"https://clinicaltrials.gov/search?intr=LB4330"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":["hutchmed"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06468358"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"fusion protein","mechanism":"Anti-Claudin18.2/IL-10 fusion protein combining tumour-antigen targeting with an immunomodulatory cytokine, given in combination with LB1410.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lcb02a","kind":"drug","name":"LCB02A","aka":[],"tldr":"LCB02A is an experimental monoclonal antibody from LigaChem Biosciences in phase 2 trials, aimed at Claudin 18.2.","summary":"LCB02A is a monoclonal antibody developed by LigaChem Biosciences. Its target is Claudin 18.2. ClinicalTrials.gov describes the intervention as: CLDN18.2-directed human monoclonal antibody (Ab) linked to a topoisomerase I inhibiting payload. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT07460375, plans to enrol 191 participants with primary completion expected 2030-02. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of LCB02A","url":"https://clinicaltrials.gov/search?intr=LCB02A"},{"label":"Sponsor page","url":"https://www.ligachembio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":["ligachem-biosciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07460375"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at Claudin 18.2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lcb84","kind":"drug","name":"LCB84","aka":[],"tldr":"LCB84 is an experimental monoclonal antibody from LigaChem Biosciences in phase 2 trials, aimed at TROP2.","summary":"LCB84 is a monoclonal antibody developed by LigaChem Biosciences. Its target is TROP2. ClinicalTrials.gov describes the intervention as: TROP2-directed human monoclonal antibody (Ab) linked to a monomethyl auristatin E (MMAE) prodrug. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT05941507, plans to enrol 300 participants with primary completion expected 2027-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of LCB84","url":"https://clinicaltrials.gov/search?intr=LCB84"},{"label":"Sponsor page","url":"https://www.ligachembio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["trop2"],"drugs":[],"companies":["ligachem-biosciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05941507"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at TROP2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lenalidomide","kind":"drug","name":"Lenalidomide","aka":[],"tldr":"Lenalidomide is a thalidomide descendant that glues the proteins IKZF1 and IKZF3 to cereblon so the cell destroys them, killing plasma cells and rousing T cells. It is the backbone of myeloma treatment and maintenance, also used in mantle cell and follicular lymphoma, and generic since 2022.","summary":"Approved 2006 (myeloma with dexamethasone), 2017 (post-transplant maintenance), plus MDS del(5q), mantle cell and follicular lymphoma (R2). Generic since 2022. Degrades IKZF1/IKZF3 via cereblon, killing plasma cells and co-stimulating T cells; the basis for CELMoDs and molecular-glue degraders generally.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Lenalidomide","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lenalidomide"}],"tags":[],"related":[],"cancers":["multiple-myeloma","dlbcl","myeloma-transplant-ineligible"],"sections":[],"technologies":["protac-degrader"],"targets":["ikzf1","ikzf3"],"drugs":[],"companies":["bms","natco"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05438043","nct04712097","nct05429268","nct06149286","nct05623020","nct05827016","nct06508658","nct06191744","nct06911502","nct04224493","nct07095452","nct06679101","nct04680052","nct05317416","nct05552222","nct06045806","nct04404283","nct05100862","nct05888493","nct05409066","nct05371093","nct05222555","nct02343042","nct04663347","nct04133636","nct05583617","nct07189065","nct06577025","nct05950165","nct06087653","nct02180711","nct07150091","nct04782687","nct04246086","nct06892522","nct05283720","nct04669171","nct07018050","nct04270409","nct06084936","nct06082102"],"people":[],"bottlenecks":[],"keyPapers":["paper-augment-lenalidomide-rituximab-leonard-jco-2019","paper-ifm-2009-attal-nejm-2017"],"journals":[],"dependsOn":[],"notes":[],"brand":"Revlimid","modality":"Immunomodulatory drug (cereblon E3 ligase modulator)","mechanism":"Binds cereblon (CRBN) in the CRL4 E3 ligase, recruiting Ikaros and Aiolos for ubiquitination and degradation; downregulates IRF4/MYC; enhances IL-2 and T/NK activity.","approvals":[{"region":"US","year":2005,"indication":"Transfusion-dependent anaemia in low-risk MDS with del(5q)"},{"region":"US","year":2006,"indication":"Relapsed myeloma with dexamethasone"},{"region":"US","year":2015,"indication":"Newly diagnosed myeloma with dexamethasone"},{"region":"US","year":2017,"indication":"Maintenance after autologous transplant"}],"mechanismSteps":["Lenalidomide binds cereblon in the CRL4 ubiquitin ligase","The complex gains affinity for Ikaros and Aiolos","Both transcription factors are ubiquitinated and destroyed","IRF4 and MYC fall; myeloma cell dies","T and NK cells lose an inhibitory brake and activate"],"dosing":{"route":"Oral","schedule":"25 mg days 1-21 of 28 (induction); 10-15 mg continuous (maintenance); renal dose adjustment","modifications":"Hold for neutrophils <1.0 or platelets <30; thromboprophylaxis with aspirin or anticoagulant","monitoring":"Counts, venous thromboembolism, second primary malignancies, rash; REMS for teratogenicity"},"toxicity":[{"event":"Neutropenia","grade3PlusPct":35,"note":"MM-009/010 with dexamethasone"},{"event":"Venous thromboembolism","anyGradePct":12,"note":"without prophylaxis"},{"event":"Second primary malignancy","anyGradePct":7,"note":"maintenance after melphalan (CALGB 100104, 7-8% vs 3%)"}],"access":[{"country":"US","listPrice":"~$20,000 per month (brand, 2022); generics volume-limited until 2026","generic":true,"asOf":"2026-09-07"}],"regulatoryEvents":[{"date":"2005-12-27","type":"approval","region":"US","note":"MDS del(5q)"},{"date":"2006-06-29","type":"approval","region":"US","note":"Myeloma with dexamethasone"},{"date":"2022-03","type":"label-change","region":"US","note":"First generics launch (volume-limited)"}]},{"id":"lenograstim","kind":"drug","name":"Lenograstim","aka":["rHuG-CSF (glycosylated)"],"tldr":"Lenograstim is Europe's and Japan's glycosylated form of G-CSF, given after chemotherapy to shorten the period of dangerously low neutrophils and to mobilise stem cells for transplant; it does the same job as filgrastim.","summary":"Lenograstim is granulocyte colony-stimulating factor made in Chinese hamster ovary cells, so it carries the sugar chain of the natural protein, unlike filgrastim made in E. coli. It has been approved in Europe and Japan since the early 1990s to reduce the duration of neutropenia after cytotoxic chemotherapy and bone marrow transplantation and to mobilise peripheral blood stem cells in patients and healthy donors. Trials comparing it with filgrastim show equivalent efficacy at comparable doses. It was never approved in the United States.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Lenograstim","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Lenograstim"},{"label":"ChEMBL CHEMBL1201567","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201567"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":[],"sections":[],"technologies":["cytokine-therapy"],"targets":["csf3r"],"drugs":["filgrastim","pegfilgrastim"],"companies":["chugai"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01985724","nct00615602","nct00431080"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Granocyte","modality":"Recombinant glycosylated G-CSF, injection","supportive":true,"mechanism":"Activates the G-CSF receptor on neutrophil precursors to drive their proliferation, maturation and release from the marrow.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lenvatinib","kind":"drug","name":"Lenvatinib","aka":[],"tldr":"An oral anti-angiogenic pill that matched sorafenib in liver cancer with higher response rates, and partners with pembrolizumab in kidney and endometrial cancer.","summary":"REFLECT (2018): non-inferior OS (13.6 vs 12.3 months) with better PFS and ORR than sorafenib in first-line HCC. LEAP-002 (with pembrolizumab) missed its OS endpoint versus lenvatinib alone; LEAP-012 (with pembrolizumab plus TACE) improved PFS but not OS. Also approved in thyroid cancer, RCC (with everolimus or pembrolizumab) and endometrial cancer (with pembrolizumab). Hypertension, proteinuria and weight loss are dose-limiting.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Lenvatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lenvatinib"}],"tags":[],"related":["msi-high"],"cancers":["hcc","hcc-advanced","hcc-intermediate","thyroid","rcc","endometrial","papillary-thyroid-cancer","follicular-thyroid-cancer","anaplastic-thyroid-cancer"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf","fgfr2","ret","kit"],"drugs":[],"companies":["eisai","merck"],"institutions":[],"pathways":["vegf-angiogenesis"],"terms":["tace-term"],"trials":["reflect","leap-002","leap-012","nct04194775","nct06371157","nct05822752","nct05775159","nct07219459","nct07081633","nct05112991","nct05024214","nct02861573","nct05903833","nct04977453","nct04008797","nct04626479","nct05319730","nct04586231","nct05899049","nct04938817","nct04976634","nct04626518","nct06682780","nct07405164","nct06962787","clear","litespark-012","keynote-775"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In kidney cancer: CLEAR (with pembrolizumab) produced the longest first-line PFS reported, 23.9 months, with OS HR 0.79; lenvatinib plus everolimus is used in later lines (Study 205). The dose is 20 mg daily with pembrolizumab or 18 mg with everolimus 5 mg, about 70 percent of CLEAR patients needed a reduction, and 82 percent had a grade 3 or higher event.","In endometrial cancer: approved with pembrolizumab for pMMR advanced disease after platinum (KEYNOTE-775, OS 18.3 versus 11.4 months), after accelerated approval on KEYNOTE-146 in 2019; LEAP-001 in first line against chemotherapy was negative. The dose is 20 mg daily with pembrolizumab 200 mg every 3 weeks; hypertension affected 64 percent (38 percent grade 3 or higher), hypothyroidism 57 percent, diarrhoea 54 percent, and about 67 percent of KEYNOTE-775 patients needed a dose reduction. Blood pressure is checked weekly for two cycles and TSH every 6 weeks."],"brand":"Lenvima","modality":"Small-molecule multi-kinase inhibitor (VEGFR, FGFR, PDGFR, RET, KIT)","mechanism":"Oral inhibitor of VEGFR1-3, FGFR1-4, PDGFR-α, RET and KIT.","approvals":[{"region":"US","year":2015,"indication":"Radioiodine-refractory thyroid cancer"},{"region":"US","year":2018,"indication":"First-line unresectable HCC"},{"region":"US","year":2021,"indication":"Advanced RCC with pembrolizumab; endometrial cancer with pembrolizumab"},{"region":"US","year":2016,"indication":"Advanced RCC with everolimus after anti-angiogenic therapy"}],"mechanismSteps":["Blocks VEGFR and FGFR on tumour vessels","Vascular pruning and reduced perfusion","FGFR blockade counters a resistance pathway to anti-VEGF therapy"],"dosing":{"route":"Oral","schedule":"12 mg daily (≥60 kg) or 8 mg daily (<60 kg) in HCC","modifications":"Interrupt then reduce for grade 3 toxicity","monitoring":"Blood pressure, urine protein, thyroid function"},"toxicity":[{"event":"Hypertension","anyGradePct":42,"grade3PlusPct":23,"note":"REFLECT"},{"event":"Diarrhoea","anyGradePct":39,"grade3PlusPct":4,"note":"REFLECT"},{"event":"Decreased appetite/weight loss","anyGradePct":34,"grade3PlusPct":5,"note":"REFLECT"},{"event":"Proteinuria","anyGradePct":25,"grade3PlusPct":6,"note":"REFLECT"}],"access":[],"regulatoryEvents":[{"date":"2019-09-17","type":"accelerated-approval","region":"US","note":"Accelerated approval with pembrolizumab in endometrial cancer (KEYNOTE-146)","indication":"In combination with pembrolizumab for advanced endometrial carcinoma not MSI-H or dMMR, with progression following systemic therapy and not candidates for curative surgery or radiation"},{"date":"2021-07-21","type":"conversion","region":"US","note":"Full approval, pMMR endometrial cancer (KEYNOTE-775)","indication":"In combination with pembrolizumab for advanced endometrial carcinoma not MSI-H or dMMR, with progression following systemic therapy and not candidates for curative surgery or radiation"},{"date":"2026-09-24","type":"approval","region":"US","note":"With belzutifan, advanced renal cell carcinoma with a clear cell component","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belzutifan-combination-lenvatinib-advanced-renal-cell-carcinoma-clear-cell-component"}]},{"id":"lenzilumab","kind":"drug","name":"Lenzilumab","aka":["KB003"],"tldr":"Lenzilumab is an antibody that soaks up GM-CSF, a growth signal that chronic myelomonocytic leukaemia cells with RAS mutations are unusually sensitive to. It is being tested with azacitidine in this leukaemia.","summary":"Lenzilumab was developed by Humanigen and is best known for its trials in COVID-19 pneumonia. Its oncology rationale comes from the observation that CMML progenitors, especially those with NRAS, KRAS or CBL mutations, proliferate in response to very low levels of GM-CSF. The investigator-led PREACH-M trial in Australia is testing lenzilumab with azacitidine in CMML with RAS-pathway mutations, given intravenously with each azacitidine cycle, and has reported responses in early analyses.\n\nHumanigen itself entered bankruptcy in 2024, so the drug's future depends on the academic programme and any new sponsor. The CMML page names it among the targeted approaches, with MEK inhibitors and ruxolitinib, being tested in the disease.","status":"phase-2","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Lenzilumab","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Lenzilumab"}],"tags":["subtype-drugs-wave"],"related":["azacitidine"],"cancers":["cmml"],"sections":[],"technologies":["monoclonal-antibody"],"targets":[],"drugs":[],"companies":["humanigen"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"KB003","modality":"Humanised anti-GM-CSF monoclonal antibody","mechanism":"Neutralises granulocyte-macrophage colony-stimulating factor, the cytokine to which chronic myelomonocytic leukaemia cells with RAS-pathway mutations are hypersensitive, cutting the signal that drives their proliferation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lerociclib","kind":"drug","name":"Lerociclib","aka":["G1T38"],"tldr":"Lerociclib is a CDK4/6 inhibitor from G1 Therapeutics developed in China by Genor Biopharma, in a phase 3 trial with letrozole for hormone receptor-positive breast cancer.","summary":"G1 Therapeutics designed lerociclib (G1T38) as a CDK4/6 inhibitor that could be taken continuously without a break; it licensed Chinese rights to Genor Biopharma, which runs the phase 3 LEONARDA trials with letrozole or fulvestrant in HR-positive, HER2-negative advanced breast cancer. EQRx briefly held Western rights before returning them.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Lerociclib","url":"https://clinicaltrials.gov/search?intr=GB491"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["cdk4-6"],"drugs":[],"companies":["g1-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05851014"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"GB491","modality":"Oral CDK4/6 inhibitor","mechanism":"Inhibits cyclin-dependent kinases 4 and 6, arresting hormone receptor-positive breast cancer cells in G1, the class of palbociclib, ribociclib and abemaciclib.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lerotinib","kind":"drug","name":"Lerotinib","aka":["Z650"],"tldr":"Lerotinib is an experimental small-molecule drug from Sunshine Lake Pharma in phase 3 trials for oesophageal cancer, with its target not yet stated publicly.","summary":"Lerotinib is a small-molecule drug developed by Sunshine Lake Pharma. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Specification: 50 mg/capsule and 150 mg/capsule. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04415853 (Study of Larotinib in Unresectable Advanced or Recurrent Oesophageal Cancer), in oesophageal cancer. The largest, NCT04415853, plans to enrol 416 participants with primary completion was scheduled for 2026-08 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Lerotinib","url":"https://clinicaltrials.gov/search?intr=Lerotinib"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["esophageal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct04415853"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"letetresgene-autoleucel","kind":"drug","name":"Letetresgene autoleucel","aka":[],"tldr":"Letetresgene autoleucel is a second engineered T-cell receptor therapy for sarcoma, targeting NY-ESO-1 in synovial sarcoma and myxoid/round cell liposarcoma; a BLA is expected by the end of 2026.","summary":"Letetresgene autoleucel is an engineered T-cell receptor therapy: the patient's own T cells receive an affinity-enhanced TCR that recognises the NY-ESO-1/LAGE-1a peptide presented on HLA-A*02, then are returned after lymphodepletion. Its targets are synovial sarcoma and myxoid/round cell liposarcoma (MRCLS), two rare sarcomas that usually express NY-ESO-1. In the pivotal phase 2 IGNYTE-ESO trial, the ORR by independent review was 42% (27 of 64), with 41% in synovial sarcoma and 43% in MRCLS, and a median duration of response of 12.2 months overall and 18.3 months in synovial sarcoma. It holds Breakthrough Therapy designation for MRCLS. Rights passed from GSK to Adaptimmune in 2023 and to US WorldMeds in August 2025, which committed to a BLA by 31 December 2026. It is a one-time infusion of T cells re-engineered to see an antigen these two sarcomas display.","status":"phase-2","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT03967223 (IGNYTE-ESO)","url":"https://clinicaltrials.gov/study/NCT03967223"}],"tags":[],"related":[],"cancers":["sarcoma","synovial-sarcoma"],"sections":[],"technologies":["tcr-t"],"targets":["hla-a"],"drugs":[],"companies":["us-worldmeds"],"institutions":[],"pathways":[],"terms":["crs"],"trials":["ignyte-eso"],"people":[],"bottlenecks":[],"keyPapers":["paper-spearhead-1-lancet-2024"],"journals":[],"dependsOn":[],"notes":[],"code":"lete-cel, GSK3377794","modality":"TCR-T (NY-ESO-1)","mechanism":"Autologous T cells with an affinity-enhanced TCR against NY-ESO-1/LAGE-1a peptide on HLA-A*02; lymphodepletion then infusion.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2024-06","type":"designation","region":"US","note":"Breakthrough Therapy for MRCLS"},{"date":"2025-08-04","type":"filing","region":"US","note":"Asset transferred to US WorldMeds; BLA committed by end-2026"}]},{"id":"letrozole","kind":"drug","name":"Letrozole (and other aromatase inhibitors)","aka":[],"tldr":"Daily pills that stop the body making oestrogen after menopause, the backbone of hormone therapy for most breast cancers.","summary":"Non-steroidal (letrozole, anastrozole) and steroidal (exemestane) aromatase inhibitors block conversion of androgens to oestrogens in fat and tumour tissue. Superior to tamoxifen for postmenopausal women in adjuvant (ATAC, BIG 1-98) and metastatic settings; the standard partner for CDK4/6 inhibitors in first-line metastatic disease. Used with ovarian suppression in premenopausal women (SOFT/TEXT). Arthralgia, bone loss, and vaginal dryness drive non-adherence.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Letrozole","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Letrozole"}],"tags":[],"related":[],"cancers":["breast-hr-positive","ovarian","endometrial","granulosa-cell-tumour","low-grade-serous-ovarian-cancer"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":["er-signaling"],"terms":["lgsoc"],"trials":["soft-text","monaleesa-2","paloma-2","monarch-3","nct06072781","nct04557449","nct04553133","nct04606446","nct07288359","nct05867251"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In low-grade serous ovarian cancer, retrospective series and the NRG-GY019 trial support endocrine maintenance, and letrozole is now widely used after surgery or chemotherapy in LGSOC and in ER-positive endometrial cancer, often with palbociclib or abemaciclib; it is cheap and well tolerated, with bone loss and arthralgia the main issues."],"brand":"Femara; anastrozole (Arimidex); exemestane (Aromasin)","modality":"Small-molecule aromatase inhibitor","mechanism":"Competitive (letrozole, anastrozole) or irreversible (exemestane) inhibition of CYP19A1 aromatase.","approvals":[{"region":"US","year":1997,"indication":"Advanced breast cancer after tamoxifen (letrozole)"},{"region":"US","year":2005,"indication":"Adjuvant early breast cancer (letrozole)"}],"mechanismSteps":["Adrenal androgens circulate to fat, muscle, and tumour","Aromatase (CYP19A1) would convert them to oestradiol","The inhibitor occupies the enzyme's active site","Oestradiol falls by >95%","ER-dependent transcription and proliferation stop"],"dosing":{"route":"Oral","schedule":"Letrozole 2.5 mg, anastrozole 1 mg, or exemestane 25 mg once daily; 5-10 years adjuvant; until progression metastatic","modifications":"Rarely needed; switch agent for intolerable arthralgia","monitoring":"Bone density every 1-2 years; lipids","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2014/020726s027lbl.pdf"},"toxicity":[{"event":"Arthralgia/myalgia","anyGradePct":35},{"event":"Hot flushes","anyGradePct":33},{"event":"Bone fracture (5-year)","anyGradePct":10},{"event":"Fatigue","anyGradePct":20}],"access":[],"regulatoryEvents":[{"date":"1997-07-25","type":"approval","region":"US","note":"Letrozole approved for advanced breast cancer"},{"date":"2004-10-29","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Extended adjuvant treatment of early breast cancer in postmenopausal women who received 5 years of adjuvant tamoxifen"},{"date":"2005-12-28","type":"accelerated-approval","region":"US","note":"Letrozole adjuvant indication","indication":"Postmenopausal women with HR positive early breast cancer"},{"date":"2010-04-30","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2004 converted to traditional approval 5.5 years after it was granted.","indication":"Extended adjuvant treatment of early breast cancer in postmenopausal women who received 5 years of adjuvant tamoxifen"},{"date":"2010-04-30","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2005 converted to traditional approval 4.3 years after it was granted.","indication":"Postmenopausal women with HR positive early breast cancer"}]},{"id":"leucovorin","kind":"drug","name":"Leucovorin (folinic acid)","aka":["Leucovorin Calcium","Folinic acid","Calcium folinate","Levoleucovorin (Fusilev, Khapzory)"],"tldr":"Leucovorin is not a cancer drug in itself: it rescues normal tissue after high-dose methotrexate and, given with fluorouracil, makes that chemotherapy work better, which is why it appears in FOLFOX, FOLFIRI and FOLFIRINOX.","summary":"Leucovorin (folinic acid, calcium folinate) is a form of folate that cells can use without the enzyme methotrexate blocks. Given after high-dose methotrexate it lets normal bone marrow and gut recover while the drug has already acted on the tumour; given with fluorouracil it increases the drug's binding to its target enzyme thymidylate synthase, an effect that underlies every fluorouracil-based bowel, stomach and pancreatic cancer regimen. Levoleucovorin is the active isomer alone at half the dose. It is a generic medicine on the WHO essential medicines list.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Folinic_acid","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=leucovorin%20calcium"}],"tags":["gap-fill","generic"],"related":[],"cancers":["colorectal","pancreatic","gastric","all-leukemia"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["dhfr","tyms"],"drugs":["methotrexate","fluorouracil"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["int-0116","nct07775287","nct07412613","nct07621718","nct06107413","nct05846867","nct04421820","nct06493760","nct06901531","nct07056777","nct06662786","nct03505320","nct06750094","nct07238283","nct06703177","nct05859750","nct04919226"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Wellcovorin / generic","modality":"Reduced folate (rescue agent and chemotherapy modulator)","supportive":true,"mechanism":"A reduced folate that bypasses dihydrofolate reductase, rescuing normal cells from methotrexate, and that stabilises the fluorouracil-thymidylate synthase complex to make fluorouracil more effective.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"leuprolide","kind":"drug","name":"Leuprolide (leuprorelin) and GnRH agonists","aka":["Leuprorelin","Leuprolide acetate","GnRH agonist"],"tldr":"Leuprolide is the injectable that shuts off testosterone production, the foundation of hormone therapy for prostate cancer since the 1980s; it is also used for ovarian suppression in premenopausal breast cancer.","summary":"Approved 1985 as an alternative to orchiectomy or oestrogens; depot formulations (1-6 months) followed. Backbone of ADT combinations with abiraterone, enzalutamide, apalutamide, darolutamide and docetaxel. Testosterone flare (antiandrogen cover), cardiovascular and bone effects; oral relugolix (2020) and degarelix are antagonist alternatives without flare.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Leuprorelin","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=leuprolide"}],"tags":["gap-fill","generic"],"related":[],"cancers":["prostate","breast-hr-positive","salivary-duct-carcinoma"],"sections":[],"technologies":["androgen-deprivation","endocrine-therapy"],"targets":["androgen-receptor"],"drugs":[],"companies":["abbvie","takeda","foresee-pharmaceuticals","oakwood-laboratories"],"institutions":[],"pathways":[],"terms":["castration-resistance","ovarian-function-suppression"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lupron / Eligard / Camcevi","modality":"GnRH agonist depot","mechanism":"Continuous GnRH receptor stimulation desensitises the pituitary, suppressing LH/FSH and hence testosterone (or oestrogen) to castrate levels after an initial flare.","approvals":[{"region":"US","year":1985,"indication":"Palliative treatment of advanced prostate cancer"},{"region":"US","year":1989,"indication":"Depot formulation (Lupron Depot)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lifileucel","kind":"drug","name":"Lifileucel","aka":[],"tldr":"Lifileucel was the first approved TIL therapy: the patient's own tumour-fighting immune cells are expanded to billions and given back.","summary":"Accelerated approval February 2024 for anti-PD-1-refractory advanced melanoma (C-144-01: ORR 31%; 5-year data show median DOR 36.5 months and ~30% of responders in ongoing response). Confirmatory TILVANCE-301 with pembrolizumab in first line. EU approval 2025. Trials in NSCLC, cervical, and endometrial cancer.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Lifileucel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lifileucel"}],"tags":[],"related":[],"cancers":["melanoma","nsclc","advanced-melanoma"],"sections":[],"technologies":["til-therapy"],"targets":[],"drugs":[],"companies":["iovance"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07288203","nct06481592"],"people":["inge-marie-svane"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Amtagvi","modality":"TIL cell therapy","mechanism":"Autologous TILs expanded ex vivo with IL-2; lymphodepletion; high-dose IL-2 after infusion.","approvals":[{"region":"US","year":2024,"indication":"Unresectable/metastatic melanoma after anti-PD-1 (and BRAF/MEK if applicable)"}],"mechanismSteps":["Tumour is surgically resected and shipped to the manufacturing site","Tumour-infiltrating lymphocytes are expanded ex vivo with IL-2 over ~3 weeks","Patient receives lymphodepleting chemotherapy","Billions of polyclonal tumour-reactive T cells are infused","IL-2 supports engraftment and expansion","TILs recognise neoantigens and kill tumour cells"],"dosing":{"route":"Single IV infusion after lymphodepletion","schedule":"7.5 × 10⁹ to 72 × 10⁹ viable cells; cyclophosphamide 60 mg/kg ×2 and fludarabine 25 mg/m² ×5 before; IL-2 600,000 IU/kg every 8-12 h for up to 6 doses after","modifications":"IL-2 held for hypotension, arrhythmia, or organ dysfunction","monitoring":"Inpatient management; cardiac, pulmonary, renal function; cytopenias; infections","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Thrombocytopenia"},{"event":"Chills"},{"event":"Pyrexia"},{"event":"Anaemia"},{"event":"Febrile neutropenia"},{"event":"Hypotension"},{"event":"Treatment-related mortality","note":"Boxed warning; deaths related to lymphodepletion/IL-2 reported"}],"access":[{"country":"US","listPrice":"$515,000 per treatment (list price at launch, 2024)","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.iovancecares.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"EU","reimbursement":"EMA approved 2025; national pricing pending","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-05","type":"filing","region":"US","note":"BLA submitted","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-02-16","type":"accelerated-approval","region":"US","note":"Accelerated approval, unresectable/metastatic melanoma after anti-PD-1 (C-144-01): first TIL therapy The confirmatory requirement was still open 2.6 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with unresectable or metastatic melanoma previously treated with a PD-1 blocking antibody, and if BRAF V600 mutation positive, a BRAF inhibitor with or without a MEK inhibitor."},{"date":"2025-06","type":"approval","region":"EU","note":"Conditional marketing authorisation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"limertinib","kind":"drug","name":"Limertinib","aka":[],"tldr":"Limertinib is Aosaikang's third-generation EGFR inhibitor, approved in China in 2024 for lung cancer with the T790M resistance mutation after earlier EGFR drugs, and in phase 3 trials for first-line use.","summary":"Jiangsu Aosaikang's limertinib (ASK120067) was approved by the NMPA in 2024 for locally advanced or metastatic EGFR T790M-positive non-small cell lung cancer after progression on earlier EGFR inhibitors, joining osimertinib, furmonertinib and befotertinib among China's third-generation options. A phase 3 first-line trial against gefitinib is registered.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Limertinib","url":"https://clinicaltrials.gov/search?intr=ASK120067"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["aosaikang-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04143607","nct07109531"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"ASK120067","modality":"Oral third-generation EGFR tyrosine kinase inhibitor","mechanism":"A covalent inhibitor of EGFR sensitising and T790M resistance mutations that spares wild-type EGFR, the osimertinib class.","approvals":[{"region":"CN","year":2024,"indication":"Locally advanced or metastatic EGFR T790M-mutant non-small cell lung cancer after prior EGFR inhibitor therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"linperlisib","kind":"drug","name":"Linperlisib","aka":["Yinlijia"],"tldr":"Linperlisib is Shanghai Yingli's PI3K-delta inhibitor, approved in China in 2022 for relapsed or refractory follicular lymphoma after two or more lines.","summary":"Linperlisib (YY-20394) was developed by Shanghai Yingli Pharmaceutical. The NMPA gave it conditional approval in November 2022 for adults with relapsed or refractory follicular lymphoma after at least two prior systemic therapies, on a single-arm phase 2 trial with an overall response rate near 80 percent. It is the first PI3K inhibitor developed in China to reach approval.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of linperlisib","url":"https://clinicaltrials.gov/search?intr=YY-20394"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["follicular-lymphoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":[],"drugs":[],"companies":["shanghai-yingli"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06224257","nct04379167"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"YY-20394","modality":"Oral PI3K-delta inhibitor","mechanism":"Blocks the delta form of PI3K, a signalling enzyme B-cell lymphomas depend on for survival.","approvals":[{"region":"CN","year":2022,"indication":"Relapsed or refractory follicular lymphoma after two or more lines, conditional"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"linvoseltamab","kind":"drug","name":"Linvoseltamab","aka":[],"tldr":"Linvoseltamab is Regeneron's BCMA bispecific, approved in July 2025 with the highest complete-response rate of the class in its pivotal study.","summary":"Linvoseltamab is a fully human IgG4 BCMAxCD3 bispecific antibody built on Regeneron's Veloci-Bi platform, given intravenously with step-up dosing and moved to every-4-week dosing in patients who respond. In LINKER-MM1, patients with at least 3 prior lines including an anti-CD38 antibody had ORR 70% and CR or better 45%, the highest complete response rate of the BCMA bispecific class in a pivotal study. The FDA granted accelerated approval on 2 July 2025 after at least 4 prior lines, and the EU approved it in 2025. LINKER-MM3, comparing it with elotuzumab-pomalidomide-dexamethasone, is the confirmatory phase 3. Its place alongside teclistamab and elranatamab, which reached the market earlier, depends on whether the deeper responses and less frequent dosing hold up. It is the third BCMA bispecific for myeloma, notable for how many patients reach complete remission.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Linvoseltamab"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-relapsed-refractory"],"sections":[],"technologies":["t-cell-engager"],"targets":["bcma","cd3"],"drugs":[],"companies":["regeneron"],"institutions":[],"pathways":[],"terms":["pivotal-trial"],"trials":["linker-mm1","nct05730036","nct07393282","nct06140524","nct05955508","nct06669247","nct05828511","nct07455851"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lynozyfic","modality":"Bispecific T-cell engager (BCMA×CD3)","mechanism":"Fully human IgG4 BCMA×CD3 bispecific (Veloci-Bi), IV with step-up.","approvals":[{"region":"US","year":2025,"indication":"Relapsed/refractory myeloma after ≥4 lines (accelerated)"},{"region":"EU","year":2025,"indication":"R/R myeloma ≥3 lines; 23 Apr 2025 (conditional)","note":"Conditional marketing authorisation"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2025-07-02","type":"accelerated-approval","region":"US","note":"Accelerated approval (LINKER-MM1) The confirmatory requirement was still open 1.2 years later, when the FDA's table was read.","source":"https://investor.regeneron.com/news-releases/news-release-details/lynozyfictm-linvoseltamab-gcpt-receives-fda-accelerated-approval","indication":"Treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody."}]},{"id":"lipegfilgrastim","kind":"drug","name":"Lipegfilgrastim","aka":[],"tldr":"Lipegfilgrastim (Lonquex) is a once-per-chemotherapy-cycle white cell booster used in Europe, similar to pegfilgrastim, for adults and children from two years old.","summary":"Lipegfilgrastim was authorised by the European Commission in July 2013 for reduction in the duration of neutropenia and the incidence of febrile neutropenia in adults treated with cytotoxic chemotherapy for malignancy (excluding CML and MDS), extended to children aged 2 and over in 2021, on phase 3 trials in breast cancer (non-inferior duration of severe neutropenia versus pegfilgrastim) and NSCLC (superior to placebo). It differs from pegfilgrastim in the site-specific enzymatic glycopegylation used to attach the polyethylene glycol chain. It is not approved in the US. Bone pain and musculoskeletal pain are the common adverse effects; splenic rupture and pulmonary adverse reactions are rare class risks. Marketed by Teva.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Lipegfilgrastim","links":[{"label":"XM22-03 (BMC Cancer 2013)","url":"https://doi.org/10.1186/1471-2407-13-386"},{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/lonquex"}],"tags":["ema-list","supportive"],"related":["pegfilgrastim"],"cancers":[],"sections":[],"technologies":["g-csf-growth-factors"],"targets":[],"drugs":[],"companies":["teva"],"institutions":[],"pathways":[],"terms":[],"trials":["xm22-03"],"people":[],"bottlenecks":[],"keyPapers":["paper-xm22-03-bondarenko-bmc-cancer-2013"],"journals":[],"dependsOn":[],"notes":[],"brand":"Lonquex","modality":"Glycopegylated recombinant G-CSF","supportive":true,"mechanism":"Filgrastim site-specifically glycopegylated at threonine 134; reduced renal clearance gives once-per-cycle dosing with neutrophil-mediated self-regulating elimination.","approvals":[{"region":"EU","year":2013,"indication":"Reduction in duration of neutropenia and febrile neutropenia after cytotoxic chemotherapy in adults (children from 2 years added 2021)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"liposomal-irinotecan","kind":"drug","name":"Liposomal irinotecan","aka":[],"tldr":"Irinotecan wrapped in a fat bubble so it circulates longer. It is approved for pancreatic cancer, and in bile duct and gallbladder cancer one Korean trial found it helped as second-line treatment while a German trial did not.","summary":"Liposomal irinotecan is a nanoliposomal formulation of irinotecan that prolongs circulation of the active metabolite SN-38 and increases tumour exposure. It is approved for metastatic pancreatic adenocarcinoma after gemcitabine-based therapy (NAPOLI-1; United States 2015, European Union 2016) and, with oxaliplatin, fluorouracil and leucovorin (NALIRIFOX), as first-line treatment of metastatic pancreatic adenocarcinoma (NAPOLI 3; United States 2024). In biliary tract cancer it has no licence. The Korean NIFTY phase 2b trial randomised 174 patients who had progressed on gemcitabine and cisplatin to fluorouracil and leucovorin with or without liposomal irinotecan: blinded central review progression-free survival was 7.1 versus 1.4 months (hazard ratio 0.56) at the first analysis and 4.2 versus 1.7 months (hazard ratio 0.61) when the scans were re-read with extended follow-up, with grade 3 to 4 neutropenia in 24 percent versus 1 percent. The German NALIRICC trial (100 patients) found no benefit, and the earlier German NIFE trial compared it non-comparatively with gemcitabine and cisplatin in first line. NCCN lists it as an option after gemcitabine and cisplatin; in the UK it is not commissioned for biliary cancer, where FOLFOX (ABC-06) is the second-line standard. UGT1A1 poor metabolisers need dose reduction.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Irinotecan","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c8b58efa-1820-48a4-b70d-62918fc4abfc"},{"label":"NIFTY, Lancet Oncology 2021","url":"https://doi.org/10.1016/S1470-2045(21)00486-1"},{"label":"NIFTY updated analysis, JAMA Oncology 2023","url":"https://doi.org/10.1001/jamaoncol.2023.0016"},{"label":"NICE TA440: pegylated liposomal irinotecan after gemcitabine, not recommended (26 April 2017)","url":"https://www.nice.org.uk/guidance/ta440"},{"label":"Onivyde label (openFDA): NAPOLI 3 and NAPOLI-1 study sections","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22ONIVYDE%22"},{"label":"EMA Onivyde pegylated liposomal product page","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/onivyde-pegylated-liposomal"},{"label":"FDA notice: irinotecan liposome for first-line metastatic pancreatic adenocarcinoma (13 February 2024)","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}],"tags":[],"related":[],"cancers":["gallbladder","cholangiocarcinoma","pancreatic"],"sections":[],"technologies":["cytotoxic-chemotherapy","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":["ipsen","servier"],"institutions":[],"pathways":[],"terms":[],"trials":["nifty","nife","naliricc","napoli-1","napoli-3","rasolute-302"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer, regulators: the Onivyde label (effective 3 March 2025) carries two indications, NALIRIFOX first line (NAPOLI 3: overall survival 11.1 against 9.2 months, hazard ratio 0.84; progression-free survival 7.4 against 5.6 months, hazard ratio 0.70; FDA notice 13 February 2024) and with fluorouracil and leucovorin after gemcitabine (NAPOLI-1; FDA 2015), and states it is not indicated as a single agent. In England neither use is commissioned: TA440 does not recommend the second-line combination and the NALIRIFOX appraisal (TA1052) was terminated on 2 April 2025 because the company made no submission. Label safety for NALIRIFOX (383 patients): serious adverse reactions 54 percent, fatal 6 percent, discontinuation of Onivyde 17 percent."],"brand":"Onivyde","code":"nal-IRI, MM-398, PEP02","modality":"Liposome-encapsulated topoisomerase I inhibitor (cytotoxic)","mechanism":"Liposomal delivery of irinotecan; SN-38 traps topoisomerase I and DNA in cleavage complexes.","approvals":[{"region":"US","year":2015,"indication":"Metastatic pancreatic adenocarcinoma after gemcitabine-based therapy, with fluorouracil and leucovorin (NAPOLI-1)","note":"FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c8b58efa-1820-48a4-b70d-62918fc4abfc"},{"region":"EU","year":2016,"indication":"Metastatic pancreatic adenocarcinoma after gemcitabine, with fluorouracil and leucovorin","note":"EMA EPAR: https://www.ema.europa.eu/en/medicines/human/EPAR/onivyde-pegylated-liposomal"},{"region":"US","year":2024,"indication":"First-line metastatic pancreatic adenocarcinoma in NALIRIFOX (NAPOLI 3)","note":"FDA label (DailyMed)"},{"region":"UK","year":2017,"indication":"Metastatic adenocarcinoma of the pancreas after gemcitabine-based therapy, with fluorouracil and leucovorin: NICE TA440 (26 April 2017) does not recommend it within its marketing authorisation; NICE NG85 1.9.9 restates the position","note":"https://www.nice.org.uk/guidance/ta440"},{"region":"EU","year":2016,"indication":"Metastatic adenocarcinoma of the pancreas after gemcitabine-based therapy, with fluorouracil and leucovorin (marketing authorisation 14 October 2016, orphan designation 2011); the EMA product information now also lists first-line use with oxaliplatin, fluorouracil and leucovorin (NALIRIFOX)","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/onivyde-pegylated-liposomal"}],"mechanismSteps":[],"dosing":{"route":"Intravenous","schedule":"70 mg/m² over 90 minutes every 2 weeks with leucovorin 400 mg/m² and fluorouracil 2,400 mg/m² over 46 hours (NIFTY schedule)","modifications":"Reduced dose for UGT1A1*28 homozygotes","monitoring":"Neutrophil count, diarrhoea"},"toxicity":[{"event":"Neutropenia (grade 3-4)","grade3PlusPct":24,"source":"https://doi.org/10.1016/S1470-2045(21)00486-1","note":"NIFTY, liposomal irinotecan arm"},{"event":"Fatigue or asthenia (grade 3-4)","grade3PlusPct":13,"source":"https://doi.org/10.1016/S1470-2045(21)00486-1","note":"NIFTY"}],"access":[],"regulatoryEvents":[]},{"id":"lirafugratinib","kind":"drug","name":"Lirafugratinib","aka":[],"tldr":"An oral FGFR2 inhibitor approved in September 2026 for bile duct cancer whose tumour carries an FGFR2 fusion or rearrangement, after earlier treatment.","summary":"Lirafugratinib (RLY-4008, Lyrfigtu) is an FGFR2-selective inhibitor from Elevar Therapeutics. The US Food and Drug Administration approved it on 23 September 2026 for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma whose tumour harbours an FGFR2 gene fusion or other rearrangement, on the REFOCUS study. Selectivity for FGFR2 is the design claim: the earlier pan-FGFR inhibitors are dose-limited by the raised phosphate that follows FGFR1 inhibition.","status":"approved","asOf":"2026-09-11","links":[{"label":"FDA: approval of lirafugratinib","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lirafugratinib-previously-treated-unresectable-locally-advanced-or-metastatic"},{"label":"ClinicalTrials.gov: trials of Lirafugratinib","url":"https://clinicaltrials.gov/search?intr=RLY-4008"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cholangiocarcinoma"],"sections":[],"technologies":[],"targets":["fgfr2"],"drugs":[],"companies":["elevar-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07359820"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"brand":"Lyrfigtu","code":"RLY-4008","modality":"Small-molecule FGFR2 inhibitor","mechanism":"Oral inhibitor selective for FGFR2 over the other FGFR family members, which is the point: pan-FGFR inhibitors are limited by the hyperphosphataemia that comes from hitting FGFR1.","approvals":[{"region":"US","year":2026,"indication":"Previously treated unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-09-23","type":"approval","region":"US","note":"Previously treated unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or other rearrangement (REFOCUS)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lirafugratinib-previously-treated-unresectable-locally-advanced-or-metastatic"}]},{"id":"lisocabtagene-maraleucel","kind":"drug","name":"Lisocabtagene maraleucel","aka":[],"tldr":"Lisocabtagene maraleucel is the only CAR-T approved for chronic lymphocytic leukaemia, for patients whose disease has outrun both BTK and BCL-2 inhibitors.","summary":"TRANSCEND CLL 004: in CLL/SLL after BTK and venetoclax failure, CR/CRi 18-20%, ORR 47%, uMRD in blood 64%, durable in complete responders; accelerated approval March 2024. Also approved in LBCL (second line, TRANSFORM), follicular lymphoma, mantle cell lymphoma, and marginal zone lymphoma (2025). Defined 1:1 CD4:CD8 composition; 4-1BB costimulation. Lower CRS/ICANS than CD28-based products.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Lisocabtagene_maraleucel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lisocabtagene%20maraleucel"}],"tags":[],"related":[],"cancers":["cll","dlbcl","cll-relapsed","primary-mediastinal-b-cell-lymphoma"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":["crs","icans"],"trials":["transcend-cll-004","nct07015242"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Breyanzi","code":"liso-cel","modality":"CAR-T (CD19, defined CD4:CD8)","mechanism":"Autologous CD19 CAR-T (4-1BB) manufactured as separate CD4 and CD8 components and infused at a fixed ratio.","approvals":[{"region":"US","year":2021,"indication":"Relapsed/refractory LBCL after ≥2 lines"},{"region":"US","year":2022,"indication":"Second-line LBCL (TRANSFORM)"},{"region":"US","year":2024,"indication":"Relapsed/refractory CLL/SLL after BTK inhibitor and BCL-2 inhibitor (accelerated)"}],"mechanismSteps":["Leukapheresis; CD4 and CD8 T cells transduced and expanded separately","Lymphodepletion with fludarabine/cyclophosphamide","Fixed 1:1 CD4:CD8 infusion engages CD19 on CLL cells","Expansion is slower than CD28 CARs, moderating CRS; complete responders show long-term uMRD"],"dosing":{"route":"Single IV infusion (two components)","schedule":"90-110 × 10^6 CAR+ T cells (CLL)","monitoring":"CRS/ICANS (boxed warning), prolonged cytopenias, hypogammaglobulinaemia, secondary T-cell malignancy warning","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Breyanzi"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":83,"grade3PlusPct":9,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Breyanzi","note":"TRANSCEND CLL 004"},{"event":"Neurotoxicity","anyGradePct":46,"grade3PlusPct":20},{"event":"Prolonged cytopenias","grade3PlusPct":54}],"access":[],"regulatoryEvents":[{"date":"2024-03-14","type":"approval","region":"US","note":"First CAR-T in CLL (TRANSCEND CLL 004), accelerated"},{"date":"2024-03-21","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 2.5 years later, when the FDA's table was read.","indication":"Adult patients with relapsed/ refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in patients who have received at least two prior lines of therapy including a Bruton’s tyrosine kinase (BTK) inhibitor and a B-cell lymphoma-2 (BCL-2) inhibitor"},{"date":"2024-05-15","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-lisocabtagene-maraleucel-follicular-lymphoma","indication":"Adult patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy"},{"date":"2026-02-20","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 1.8 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-lisocabtagene-maraleucel-follicular-lymphoma","indication":"Adult patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy"}]},{"id":"livmoniplimab","kind":"drug","name":"Livmoniplimab","aka":[],"tldr":"Livmoniplimab is an experimental investigational agent whose form is not stated in the registry from AbbVie in phase 3 trials for hepatocellular carcinoma, non-small-cell lung cancer and bladder & urothelial cancer, with its target not yet stated publicly.","summary":"Livmoniplimab (ABBV-151) is an investigational agent whose form is not stated in the registry developed by AbbVie. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06109272 (A Study to Assess the Dose, Adverse Events, and Change in Disease Activity of Livmoniplimab as an Intravenous (IV) Solution in Combination With Budigalimab as an IV Solution in Adult Participants With Hepatocellular Carcinoma (HCC)) and NCT06236438 (Study to Evaluate Adverse Events, Optimal Dose, and Change in Disease Activity, With Livmoniplimab in Combination With Budigalimab Plus Chemotherapy Versus IV Infused Pembrolizumab Plus Chemotherapy in Adult Participants With Untreated Metastatic Non-Squamous Non-Small Cell Lung Cancer (NSCLC)), in hepatocellular carcinoma, non-small-cell lung cancer and bladder & urothelial cancer. The largest, NCT06236438, plans to enrol 840 participants with primary completion expected 2031-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Livmoniplimab","url":"https://clinicaltrials.gov/search?intr=ABBV-151"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hcc","nsclc","urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06109272","nsclc-2","nct05822752","nct06632951"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ABBV-151","modality":"antibody (INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lm-108","kind":"drug","name":"LM-108","aka":[],"tldr":"LM-108 is an experimental monoclonal antibody from Chia Tai Tianqing Pharmaceutical Nanjing Shunxin Pharmaceutical in phase 3 trials for pancreatic ductal adenocarcinoma and gastric & gastro-oesophageal junction cancer, with its target not yet stated publicly.","summary":"LM-108 is a monoclonal antibody developed by Chia Tai Tianqing Pharmaceutical Nanjing Shunxin Pharmaceutical and LaNova Medicines. The sponsor describes its target as CCR8, which OnCo does not yet have a target page for. ClinicalTrials.gov describes the intervention as: LM-108 injection is a monoclonal antibody that selectively clears regulatory T cells that infiltrate tumour sites. Penpulimab is a novel and differentiated programmed cell death protein 1 (PD-1) monoclonal antibody. Oxaliplatin is a third-ge. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07362186 (LM-108 in Combination With Toripalimab Versus Paclitaxel Injection for the Treatment of Subjects With CCR8-Positive Gastric and Gastroesophageal Junction Adenocarcinoma), in pancreatic ductal adenocarcinoma and gastric & gastro-oesophageal junction cancer. The largest, NCT07362186, plans to enrol 400 participants with primary completion expected 2028-08-17. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of LM-108","url":"https://clinicaltrials.gov/search?intr=LM-108"},{"label":"Sponsor page","url":"https://www.lanovamedicines.com/en"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic","gastric"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sino-biopharm","lanova-medicines"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07362186","nct05518045"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lm-302","kind":"drug","name":"LM-302","aka":[],"tldr":"LM-302 is an experimental antibody-drug conjugate from LaNova Medicines Zhejiang in phase 3 trials for gastric & gastro-oesophageal junction cancer, aimed at Claudin 18.2.","summary":"LM-302 is an antibody-drug conjugate developed by LaNova Medicines Zhejiang and Shanghai Chia Tai Tianqing Pharmaceutical Technology Development. Its target is Claudin 18.2 (the sponsor names CLDN18.2 (Claudin 18.2)). The sponsor states: LM-302 is an antibody-drug conjugate targeting CLDN18.2, evaluated in combination with tislelizumab versus tislelizumab plus chemotherapy in previously untreated, CLDN18.2-positive, HER2-negative gastric or gastroesophageal junction adenocarcinoma. ClinicalTrials.gov describes the intervention as: The LM-302 injection is an ADC drug targeting CLDN18.2. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07385703 (Clinical Study on the Efficacy and Safety of LM-302 Injection Combined With Tislelizumab and Tislelizumab Combined Chemotherapy for the Treatment of Gastric or Gastroesophageal Junction Adenocarcinoma), in gastric & gastro-oesophageal junction cancer. The largest, NCT07385703, plans to enrol 752 participants with primary completion expected 2028-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of LM-302","url":"https://clinicaltrials.gov/search?intr=LM-302"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric"],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07385703","nct05934331"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"LM-302 is an antibody-drug conjugate targeting CLDN18.2, evaluated in combination with tislelizumab versus tislelizumab plus chemotherapy in previously untreated, CLDN18.2-positive, HER2-negative gastric or gastroesophageal junction adenocarcinoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lns8801","kind":"drug","name":"LNS8801","aka":[],"tldr":"LNS8801 is an experimental investigational agent whose form is not stated in the registry from Linnaeus Therapeutics in phase 3 trials for melanoma, aimed at Estrogen receptor (ERα).","summary":"LNS8801 is an investigational agent whose form is not stated in the registry developed by Linnaeus Therapeutics. Its target is Estrogen receptor (ERα). ClinicalTrials.gov describes the intervention as: G protein-coupled estrogen receptor (GPER) agonist. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06624644 (A Trial of LNS8801 With or Without Pembrolizumab in Patients With Refractory Melanoma), in melanoma. The largest, NCT06624644, plans to enrol 135 participants with primary completion expected 2029-02. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of LNS8801","url":"https://clinicaltrials.gov/search?intr=LNS8801"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":[],"targets":["estrogen-receptor"],"drugs":[],"companies":["linnaeus-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06624644","nct04130516"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Investigational agent whose form is not stated in the registry directed at Estrogen receptor (ERα), as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"locametz","kind":"drug","name":"Locametz (kit for Ga-68 gozetotide)","aka":[],"tldr":"Novartis' gallium PSMA scan kit, approved the same day as Pluvicto as the test that qualifies men for that radioactive drug.","summary":"Locametz was approved by the FDA on 23 March 2022, alongside Pluvicto (lutetium-177 vipivotide tetraxetan), with an explicit indication for selecting patients with metastatic castration-resistant prostate cancer for PSMA-directed therapy, as well as for initial staging and biochemical recurrence. The European Commission approved it in December 2022. It is manufactured by Advanced Accelerator Applications, the Novartis radiopharmaceutical unit, and was the first PSMA PET agent approved in the EU. The scan-then-treat pairing is the template for theranostics.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Locametz"}],"tags":["test"],"related":["ga68-psma-11","illuccix"],"cancers":["prostate"],"sections":[],"technologies":["psma-pet","pet-ct","radioligand-therapy"],"targets":["psma"],"drugs":["pluvicto"],"companies":["novartis","advanced-accelerator-applications"],"institutions":[],"pathways":[],"terms":["theranostics"],"trials":["vision"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: enough PSMA uptake on the scan is required before Pluvicto can be given; a scan without uptake means the drug is unlikely to work."],"brand":"Locametz","modality":"PET imaging agent kit (PSMA, gallium-68)","mechanism":"Kit for on-site labelling of gozetotide (PSMA-11) with gallium-68; the resulting tracer images PSMA-expressing prostate cancer and identifies candidates for lutetium-177 PSMA therapy.","approvals":[{"region":"US","year":2022,"indication":"PSMA PET in prostate cancer, including selection of patients for PSMA radioligand therapy"},{"region":"EU","year":2022,"indication":"PSMA PET in prostate cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lomustine","kind":"drug","name":"Lomustine (CCNU)","aka":[],"tldr":"An old chemotherapy pill used when glioblastoma comes back, and the control arm most new glioblastoma drugs must beat.","summary":"Standard second-line agent in Europe and the control in EORTC 26101 (lomustine ± bevacizumab), REGOMA, and most recurrent-glioblastoma trials; median OS ~8-9 months at recurrence. CeTeG/NOA-09 suggested lomustine-temozolomide improves OS in MGMT-methylated newly diagnosed disease. Delayed, cumulative myelosuppression limits cycles.","status":"standard-of-care","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Lomustine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Lomustine"}],"tags":[],"related":[],"cancers":["glioblastoma","idh-mutant-astrocytoma","oligodendroglioma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["nextsource-biotechnology"],"institutions":[],"pathways":[],"terms":["blood-brain-barrier"],"trials":["rtog-9402","eortc-26951","actuate-1801","nct04762069"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Gleostine","modality":"Oral nitrosourea chemotherapy","mechanism":"Lipophilic nitrosourea; DNA alkylation and crosslinking; crosses the blood-brain barrier.","approvals":[{"region":"US","year":1976,"indication":"Brain tumours after surgery/radiation; Hodgkin lymphoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zynlonta","kind":"drug","name":"Loncastuximab tesirine","aka":[],"tldr":"Loncastuximab tesirine (Zynlonta) is a CD19 ADC with a DNA-crosslinking payload for relapsed large B-cell lymphoma.","summary":"Loncastuximab tesirine (Zynlonta) is a humanised anti-CD19 antibody carrying SG3199, a pyrrolobenzodiazepine (PBD) dimer that forms interstrand DNA crosslinks, through a cleavable valine-alanine linker at a drug-to-antibody ratio of about 2.3. It is given at 0.15 mg/kg every 3 weeks for two cycles, then 0.075 mg/kg, with dexamethasone premedication, for relapsed or refractory DLBCL after two or more lines. Accelerated approval in 2021 rested on LOTIS-2 (objective response rate 48 percent), and the EU granted conditional authorisation in 2022. PBD payloads are extremely potent but bring oedema and effusions, photosensitivity, thrombocytopenia and raised GGT, toxicities that have limited the class in solid tumours. Its place alongside CD19 CAR-T and CD20 bispecifics remains to be settled. It is a CD19-directed ADC with a DNA-crosslinking payload for lymphoma that has come back.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Loncastuximab_tesirine","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Loncastuximab%20tesirine"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["adc"],"targets":["cd19"],"drugs":[],"companies":["adc-therapeutics"],"institutions":[],"pathways":[],"terms":["val-ala"],"trials":["nct04384484","nct05658562"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zynlonta","modality":"ADC","payload":"SG3199 (PBD dimer, DNA crosslinker), DAR ~2.3","linker":"Val-Ala, cleavable","mechanism":"Humanised anti-CD19 with PBD dimer; interstrand DNA crosslinks.","approvals":[{"region":"US","year":2021,"indication":"Relapsed/refractory DLBCL after ≥2 systemic therapies"},{"region":"EU","year":2022,"indication":"R/R DLBCL ≥2 lines (conditional)"}],"mechanismSteps":["Antibody binds CD19 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","SG3199 (PBD dimer) is released inside the cell","DNA is damaged (crosslinks or double-strand breaks)","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"0.15 mg/kg every 3 weeks for 2 cycles, then 0.075 mg/kg every 3 weeks; dexamethasone premedication","modifications":"Hold for grade ≥3 oedema/effusion or cytopenias","monitoring":"Weight and fluid status, blood counts, LFTs, photosensitivity precautions","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Thrombocytopenia"},{"event":"Increased GGT"},{"event":"Neutropenia"},{"event":"Anaemia"},{"event":"Fatigue"},{"event":"Oedema and effusions"},{"event":"Photosensitivity/rash"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-04-23","type":"accelerated-approval","region":"US","note":"Accelerated approval, relapsed/refractory DLBCL after ≥2 lines (LOTIS-2) The confirmatory requirement was still open 5.4 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with relapsed or refractory (R/R) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, DLBCL arising from low grade lymphoma, and high-grade B-cell lymphoma."},{"date":"2022-12","type":"approval","region":"EU","note":"Conditional marketing authorisation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"lorigerlimab","kind":"drug","name":"Lorigerlimab","aka":[],"tldr":"Lorigerlimab is an experimental bispecific antibody from MacroGenics in phase 2 trials for ovarian cancer, vulvar cancer and cervical cancer, aimed at PD-1 and CTLA-4.","summary":"Lorigerlimab (MGD019) is a bispecific antibody developed by MacroGenics. Its targets are PD-1 and CTLA-4 (the sponsor names PD-1 x CTLA-4). The sponsor states: A DART (Dual Affinity Re-Targeting) bispecific protein that simultaneously engages the PD-1 and CTLA-4 checkpoint pathways to enhance anti-tumour immune responses. ClinicalTrials.gov describes the intervention as: Bispecific DART protein binding PD-1 and CTLA-4. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in ovarian cancer, vulvar cancer, cervical cancer and endometrial cancer. The largest, NCT06730347, plans to enrol 80 participants with primary completion expected 2027-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Lorigerlimab","url":"https://clinicaltrials.gov/search?intr=MGD019"},{"label":"Sponsor pipeline page","url":"https://macrogenics.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian","vulvar","cervical","endometrial"],"sections":[],"technologies":[],"targets":["pd1","ctla4"],"drugs":[],"companies":["macrogenics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06730347"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"MGD019","modality":"bispecific antibody","mechanism":"A DART (Dual Affinity Re-Targeting) bispecific protein that simultaneously engages the PD-1 and CTLA-4 checkpoint pathways to enhance anti-tumour immune responses.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lorlatinib","kind":"drug","name":"Lorlatinib","aka":[],"tldr":"An ALK inhibitor with the longest disease control ever recorded for a targeted lung cancer pill: 60% progression-free at five years.","summary":"Lorlatinib is a macrocyclic third-generation ALK and ROS1 inhibitor designed to cover resistance mutations including G1202R and to cross the blood-brain barrier, taken as 100 mg once daily. In CROWN it achieved a 5-year progression-free survival of 60% versus 8% for crizotinib, with near-complete protection against brain progression. It was approved in 2018 after prior ALK inhibitors and in 2021 for first-line ALK-positive NSCLC. Its distinctive toxicities are metabolic and neurological: raised cholesterol and triglycerides, weight gain, oedema, neuropathy and cognitive or mood effects, which need active management. Its position against alectinib and newer agents such as neladalkib remains open. For a newcomer: the ALK pill with the most durable results, balanced against side effects that touch thinking and mood.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Lorlatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lorlatinib"},{"label":"NICE TA628: lorlatinib for previously treated ALK-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta628"},{"label":"NICE TA1103: lorlatinib for ALK-positive advanced non-small-cell lung cancer not treated with an ALK inhibitor","url":"https://www.nice.org.uk/guidance/ta1103"}],"tags":[],"related":["alk-fusion"],"cancers":["nsclc","secondary-brain-tumours","neuroblastoma-high-risk","alk-positive-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["alk"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05297890","crown"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lorbrena","modality":"Small-molecule kinase inhibitor (ALK/ROS1)","mechanism":"Macrocyclic third-generation ALK/ROS1 TKI covering G1202R.","approvals":[{"region":"US","year":2018,"indication":"ALK+ NSCLC after prior ALK TKI"},{"region":"US","year":2021,"indication":"First-line ALK+ NSCLC"},{"region":"England (NICE)","year":2020,"indication":"ALK-positive advanced non-small-cell lung cancer progressing after alectinib or ceritinib as the first ALK inhibitor, or after crizotinib and at least one other ALK inhibitor","note":"TA628, published 13 May 2020, subject to the commercial arrangement."},{"region":"England (NICE)","year":2025,"indication":"ALK-positive advanced non-small-cell lung cancer not previously treated with an ALK inhibitor","note":"TA1103, published 21 October 2025 (CROWN); must be funded in England within 90 days of publication."}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of ALK (and ROS1), including G1202R resistance mutations","Phosphorylation of downstream substrates stops","Macrocycle enters the brain and blocks ALK fusion signalling","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"100 mg once daily","modifications":"Reduce to 75 then 50 mg for CNS effects, hyperlipidaemia, AV block","monitoring":"Lipids at baseline and monthly; ECG; mood and cognition; LFTs","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Hypercholesterolaemia","note":"CROWN: ~70% any grade, ~20% grade 3-4"},{"event":"Hypertriglyceridaemia"},{"event":"Oedema"},{"event":"Weight gain"},{"event":"Peripheral neuropathy"},{"event":"Cognitive and mood effects"},{"event":"Hypertension"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.pfizeroncologytogether.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended first-line for ALK+ NSCLC (TA909)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2018-11-02","type":"accelerated-approval","region":"US","note":"ALK+ NSCLC after prior ALK TKIs (accelerated)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"ALK-positive metastatic NSCLC that has progressed on: • Crizotinib and at least one other ALK inhibitor for metastatic disease; or • Alectinib as the first ALK inhibitor therapy for metastatic disease; or • Ceritinib as the first ALK inhibitor therapy for metastatic disease"},{"date":"2021-03-03","type":"conversion","region":"US","note":"First-line ALK+ NSCLC (CROWN); full approval","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"ALK-positive metastatic NSCLC that has progressed on: • Crizotinib and at least one other ALK inhibitor for metastatic disease; or • Alectinib as the first ALK inhibitor therapy for metastatic disease; or • Ceritinib as the first ALK inhibitor therapy for metastatic disease"}]},{"id":"lp-184","kind":"drug","name":"LP-184","aka":[],"tldr":"LP-184 is a small-molecule inhibitor from Lantern Pharma Inc., in registered phase 2 trials for triple-negative breast cancer, non-small-cell lung cancer, pancreatic ductal adenocarcinoma.","summary":"LP-184 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Lantern Pharma Inc., in triple-negative breast cancer, non-small-cell lung cancer, pancreatic ductal adenocarcinoma. Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of LP-184","url":"https://clinicaltrials.gov/search?intr=LP-184"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["tnbc","nsclc","pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["lantern-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05933265"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"LP-184","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lunit-insight-mmg","kind":"drug","name":"Lunit INSIGHT MMG","aka":[],"tldr":"A Korean AI mammography reader used in screening programmes in Sweden and Australia and cleared in the US and Europe.","summary":"Lunit INSIGHT MMG is CE-marked and received FDA 510(k) clearance in 2021. It has been evaluated as a stand-alone and second reader in Swedish population screening (the Capital Region of Denmark and Sweden's Capio S:t Göran reported detection and workload outcomes) and in the Australian BreastScreen system, and Lunit acquired Volpara in 2024 to add breast density and risk assessment. Prospective randomised evidence for Lunit's software is less mature than for Transpara, but the two products are the most widely deployed AI readers in European screening.","status":"approved","asOf":"2026-09-24","links":[{"label":"ScreenTrustCAD (Lancet Digital Health 2023)","url":"https://doi.org/10.1016/S2589-7500(23)00153-X"},{"label":"Lunit INSIGHT MMG","url":"https://www.lunit.io/en/products/mmg"}],"tags":["test"],"related":[],"cancers":["breast-hr-positive","tnbc"],"sections":[],"technologies":["radiology-ai-screening","mammography"],"targets":[],"drugs":[],"companies":["lunit"],"institutions":[],"pathways":[],"terms":[],"trials":["screentrustcad"],"people":[],"bottlenecks":[],"keyPapers":["paper-screentrustcad-dembrower-lancet-digit-health-2023"],"journals":[],"dependsOn":[],"notes":["What a result means: as with other AI readers, the software flags areas for a radiologist; it can also estimate breast density, which affects how well mammograms work for you."],"brand":"Lunit INSIGHT MMG","modality":"AI mammography detection software","mechanism":"Convolutional neural network trained on large mammography datasets marks suspicious lesions and gives an abnormality score per breast.","approvals":[{"region":"EU","year":2019,"indication":"CE mark"},{"region":"US","year":2021,"indication":"510(k) clearance, mammography lesion detection"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lurbinectedin","kind":"drug","name":"Lurbinectedin","aka":[],"tldr":"A marine-derived chemotherapy that jams cancer's gene-reading machinery, approved for relapsed small-cell lung cancer and, since 2025, as first-line maintenance with atezolizumab.","summary":"Accelerated approval June 2020 for metastatic SCLC after platinum (ORR 35%, DOR 5.3 months, basket phase 2). ATLANTIS (with doxorubicin) missed OS; IMforte (with atezolizumab as first-line maintenance) improved OS and PFS, leading to full approval on 2 October 2025. Also studied in the LAGOON second-line trial. Jazz Pharmaceuticals (US) and PharmaMar.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Lurbinectedin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lurbinectedin"}],"tags":[],"related":[],"cancers":["sclc","extensive-stage-sclc","lung-cancer"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["jazz","pharmamar"],"institutions":[],"pathways":[],"terms":[],"trials":["imforte","atlantis","nct07625644","nct06801834","nct06496048","nct05652686","nct05734066","nct07155174","nct07459634","nct06088290","lurbinectedin-basket-sclc"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lurbinectedin has no United Kingdom marketing authorisation and no NICE appraisal, so it is not available on the NHS in England, although it holds FDA approval (accelerated, 15 June 2020, converted on the IMforte maintenance data) and an EU authorisation for maintenance with atezolizumab."],"brand":"Zepzelca","code":"PM01183","modality":"Cytotoxic (transcription inhibitor)","mechanism":"Binds the DNA minor groove at CG-rich promoters, stalls RNA polymerase II and triggers its degradation; also depletes tumour-associated macrophages.","approvals":[{"region":"US","year":2020,"indication":"Metastatic SCLC after platinum chemotherapy (accelerated)"},{"region":"US","year":2025,"indication":"First-line maintenance with atezolizumab in ES-SCLC (full approval)"},{"region":"EU","year":2026,"indication":"Maintenance with atezolizumab in ES-SCLC after first-line induction; 29 May 2026"}],"mechanismSteps":["Binds guanine in the DNA minor groove at active promoters","Elongating RNA polymerase II stalls and is degraded","Transcription-dependent double-strand breaks accumulate","Cells with high transcriptional addiction (SCLC, TP53-mutant) die; macrophage depletion reduces immunosuppression"],"dosing":{"route":"Intravenous","schedule":"3.2 mg/m² over 60 minutes every 21 days (monotherapy); with atezolizumab 1200 mg in maintenance","modifications":"Delay and reduce for grade 3-4 neutropenia or hepatotoxicity","monitoring":"Blood counts and liver enzymes before each cycle","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/213702s004lbl.pdf"},"toxicity":[{"event":"Neutropenia","anyGradePct":71,"grade3PlusPct":46},{"event":"Fatigue","anyGradePct":77},{"event":"Nausea","anyGradePct":37},{"event":"Transaminase increase","anyGradePct":66}],"access":[],"regulatoryEvents":[{"date":"2020-06-15","type":"accelerated-approval","region":"US","note":"Accelerated approval, second-line SCLC The confirmatory requirement was still open 6.3 years later, when the FDA's table was read.","indication":"Treatment of adult patients with metastatic small cell lung cancer (SCLC) with disease progression on or after prior platinum-based chemotherapy ."},{"date":"2025-10-02","type":"approval","region":"US","note":"Full approval with atezolizumab as first-line maintenance (IMforte)","source":"https://www.roche.com/media/releases/med-cor-2025-10-03b"}]},{"id":"luspatercept","kind":"drug","name":"Luspatercept","aka":[],"tldr":"An injection that helps the marrow finish making red cells, reducing or removing the need for transfusions in lower-risk MDS and beta-thalassaemia.","summary":"MEDALIST (2020) established transfusion independence in ring-sideroblast MDS after ESA failure; COMMANDS (2023) showed superiority to epoetin alfa in ESA-naive lower-risk MDS, making it a first-line option. Also approved in beta-thalassaemia (BELIEVE) and studied in myelofibrosis anaemia (INDEPENDENCE).","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Luspatercept","links":[{"label":"COMMANDS (Lancet 2023)","url":"https://doi.org/10.1016/S0140-6736(23)00874-7"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=luspatercept"}],"tags":["gap-fill"],"related":[],"cancers":["mds","myeloproliferative-neoplasms","mds-lower-risk"],"sections":[],"technologies":["transfusion-support"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04717414","nct04064060"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Reblozyl","modality":"Erythroid maturation agent (activin receptor IIB ligand trap, Fc fusion)","mechanism":"Binds TGF-β superfamily ligands (GDF11, activin B) to relieve SMAD2/3 suppression of late-stage erythropoiesis.","approvals":[{"region":"US","year":2019,"indication":"Beta-thalassaemia transfusion-dependent anaemia"},{"region":"US","year":2020,"indication":"Very low- to intermediate-risk MDS with ring sideroblasts after ESA failure"},{"region":"US","year":2023,"indication":"First-line anaemia in ESA-naive lower-risk MDS"},{"region":"EU","year":2020,"indication":"MDS-RS and beta-thalassaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lutathera","kind":"drug","name":"Lutetium-177 dotatate","aka":[],"tldr":"Lutetium-177 dotatate was the first modern radioligand therapy (2018), for neuroendocrine tumours, and is now used in first line.","summary":"Lutetium-177 dotatate is a radioligand therapy: the somatostatin analogue DOTATATE binds SSTR2 on neuroendocrine tumour cells and carries the beta emitter 177Lu into them, irradiating over a few millimetres. NETTER-1 established it in midgut NETs progressing on octreotide, and NETTER-2 (2024) moved it into first line for higher grade-2 and grade-3 GEP-NETs with PFS 22.8 versus 8.5 months. It was approved in 2018 for SSTR-positive GEP-NETs, with paediatric use from age 12 added in 2024. Dosing is 7.4 GBq every 8 weeks for 4 doses with an amino acid infusion to protect the kidneys; lymphopenia is common, and myelodysplastic syndrome (2.3%) and acute leukaemia (0.5%) are rare late risks. Alpha-emitting successors RYZ101 and AlphaMedix are in phase 3. For a newcomer: the first modern radioligand therapy and the template for a whole class.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Lutetium_(177Lu)_oxodotreotide","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lutetium-177%20dotatate"}],"tags":[],"related":["sstr-pet-expression"],"cancers":["neuroendocrine","small-intestinal-net","pancreatic-net","grade-3-net","metastatic-ppgl","esthesioneuroblastoma"],"sections":[],"technologies":["radioligand-therapy"],"targets":["sstr2"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":["prrt-term"],"trials":["netter-1","nct04711135","nct05142696","nct03972488"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lutathera","code":"177Lu-DOTATATE","modality":"Radioligand therapy (beta)","mechanism":"SSTR2 agonist peptide with 177Lu.","approvals":[{"region":"US","year":2018,"indication":"SSTR+ GEP-NETs"},{"region":"US","year":2024,"indication":"Paediatric ≥12 years; first-line (NETTER-2 label)"},{"region":"EU","year":2017,"indication":"First approval globally (Sep 2017)"}],"mechanismSteps":["Radioligand circulates and binds Somatostatin receptor 2 on tumour cells","Ligand is internalised or retained at the membrane","Beta emissions deposit energy within a short range","Clustered DNA double-strand breaks form in the tumour cell and its neighbours (crossfire)","Cells die; unbound ligand is cleared via the kidneys"],"dosing":{"route":"IV infusion","schedule":"7.4 GBq (200 mCi) every 8 weeks for 4 doses, with amino acid infusion starting 30 minutes before and continuing ≥3 hours","modifications":"Hold for grade ≥3 haematologic toxicity or renal toxicity; discontinue after 16-week delay","monitoring":"Blood counts, creatinine, LFTs before each dose; long-term MDS/AML surveillance","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55"},"toxicity":[{"event":"Lymphopenia","grade3PlusPct":44,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"},{"event":"GGT increased","grade3PlusPct":20,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"},{"event":"Vomiting","grade3PlusPct":7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"},{"event":"Nausea","grade3PlusPct":5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"},{"event":"AST increased","grade3PlusPct":5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"},{"event":"ALT increased","grade3PlusPct":4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"},{"event":"Hyperglycaemia","grade3PlusPct":4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"},{"event":"Hypokalaemia","grade3PlusPct":4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"},{"event":"Myelodysplastic syndrome","anyGradePct":2.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"Median onset 29 months"},{"event":"Acute leukaemia","anyGradePct":0.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=72d1a024-00b7-418a-b36e-b2cb48f2ab55","note":"NETTER-1; grade 3-4 rates"}],"access":[{"country":"US","listPrice":"~$47,500 per dose (launch price 2018)","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for unresectable/metastatic GEP-NETs (TA539)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-09-26","type":"approval","region":"EU","note":"EMA approval: first modern PRRT","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2018-01-26","type":"approval","region":"US","note":"SSTR+ gastroenteropancreatic NETs (NETTER-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-04-23","type":"approval","region":"US","note":"Paediatric patients ≥12 years","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-06","type":"label-change","region":"US","note":"First-line label based on NETTER-2","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"pluvicto","kind":"drug","name":"Lutetium-177 vipivotide tetraxetan","aka":[],"tldr":"A radioactive drug that seeks out PSMA on prostate cancer cells; the best-selling radiopharmaceutical ever.","summary":"VISION (2021): OS 15.3 vs 11.3 months in post-chemotherapy mCRPC. PSMAfore (2023) led to a 2025 label before chemotherapy. July 2026 FDA action further expanded the label (per AACR/FDA roundups). PSMAddition tests it in hormone-sensitive disease. Requires PSMA PET positivity. Six cycles every 6 weeks; xerostomia, cytopenias, renal monitoring.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Lutetium_(177Lu)_vipivotide_tetraxetan","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Lutetium-177%20vipivotide%20tetraxetan"},{"label":"NICE TA930: lutetium-177 vipivotide tetraxetan for treating PSMA-positive hormone-relapsed metastatic prostate cancer after 2 or more treatments, not recommended","url":"https://www.nice.org.uk/guidance/ta930"}],"tags":[],"related":["psma-pet-expression"],"cancers":["prostate","prostate-mcrpc","prostate-mhspc"],"sections":[],"technologies":["radioligand-therapy","psma-pet"],"targets":["psma"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":["prostate-crpc-sequencing","prostate-uk-drug-approvals"],"trials":["vision","psmafore","nct05682443","nct06894511","nct06004661"],"people":["louise-emmett"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NICE TA930 does not recommend lutetium-177 vipivotide tetraxetan in England for PSMA-positive hormone-relapsed metastatic prostate cancer after taxane-based chemotherapy and an anti-androgen, or where taxanes are medically unsuitable, despite accepting that it extends both progression-free and overall survival against best supportive care. The committee could not consider the comparison with radium-223 because no such evidence was submitted. Men whose treatment started before publication are unaffected."],"brand":"Pluvicto","code":"177Lu-PSMA-617","modality":"Radioligand therapy (beta)","mechanism":"Small-molecule PSMA ligand chelated to 177Lu; beta emission with 2 mm range.","approvals":[{"region":"US","year":2022,"indication":"PSMA+ mCRPC after ARPI and taxane"},{"region":"US","year":2025,"indication":"PSMA+ mCRPC after ARPI, before chemotherapy"},{"region":"US","year":2026,"indication":"Label expansion (July 2026)"},{"region":"EU","year":2022,"indication":"mCRPC post-ARPI and taxane; pre-chemo 2025"}],"mechanismSteps":["Radioligand circulates and binds PSMA on tumour cells","Ligand is internalised or retained at the membrane","Beta emissions deposit energy within a short range","Clustered DNA double-strand breaks form in the tumour cell and its neighbours (crossfire)","Cells die; unbound ligand is cleared via the kidneys"],"dosing":{"route":"IV injection","schedule":"7.4 GBq (200 mCi) every 6 weeks for up to 6 doses","modifications":"Hold for grade ≥3 myelosuppression or renal impairment; permanently discontinue for grade 4 haematologic toxicity","monitoring":"Blood counts and renal function before each dose; hydration; radiation-protection instructions for 7 days","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=14908037-2892-4d98-a053-253ce35afb1a"},"toxicity":[{"event":"Lymphocytes decreased","anyGradePct":85,"grade3PlusPct":47,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=14908037-2892-4d98-a053-253ce35afb1a","note":"VISION"},{"event":"Haemoglobin decreased","anyGradePct":64,"grade3PlusPct":15,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=14908037-2892-4d98-a053-253ce35afb1a","note":"VISION"},{"event":"Fatigue","anyGradePct":48,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=14908037-2892-4d98-a053-253ce35afb1a","note":"VISION"},{"event":"Dry mouth","anyGradePct":39,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=14908037-2892-4d98-a053-253ce35afb1a","note":"VISION"},{"event":"Nausea","anyGradePct":36,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=14908037-2892-4d98-a053-253ce35afb1a","note":"VISION"},{"event":"Decreased appetite","anyGradePct":21,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=14908037-2892-4d98-a053-253ce35afb1a","note":"VISION"},{"event":"Platelets decreased","grade3PlusPct":9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=14908037-2892-4d98-a053-253ce35afb1a","note":"VISION"}],"access":[{"country":"US","listPrice":"~$42,500 per dose (Novartis, 2022 launch price)","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.novartis.com/us-en/patient-support","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for PSMA+ mCRPC after ARPI and taxane (2025); pre-chemotherapy appraisal in progress","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-06","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-03-23","type":"approval","region":"US","note":"PSMA+ mCRPC after ARPI and taxane (VISION)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-12","type":"approval","region":"EU","note":"EMA approval","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-03-28","type":"approval","region":"US","note":"PSMA+ mCRPC after ARPI, before chemotherapy (PSMAfore)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-07-31","type":"approval","region":"US","note":"PSMA+ metastatic hormone-sensitive prostate cancer with ARPI (PSMAddition)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"luveltamab-tazevibulin","kind":"drug","name":"Luveltamab tazevibulin","aka":[],"tldr":"A folate-receptor ADC designed to work across low and high receptor levels, in a pivotal ovarian cancer trial.","summary":"Sutro Biopharma's site-specifically conjugated FRα ADC. Phase 1 showed responses in FRα-low as well as FRα-high tumours; the phase 2/3 REFRαME-O1 trial in platinum-resistant ovarian cancer (any FRα expression >25%) is ongoing, with an interim analysis expected in 2026. Neutropenia and arthralgia are characteristic.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"REFRαME-O1","url":"https://clinicaltrials.gov/study/NCT05870748"}],"tags":[],"related":[],"cancers":["ovarian","endometrial"],"sections":[],"technologies":["adc","site-specific-conjugation"],"targets":["folr1"],"drugs":[],"companies":["sutro-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05870748"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"STRO-002","modality":"ADC","payload":"SC209 (hemiasterlin, tubulin inhibitor), DAR ~4","linker":"Cleavable, site-specific (Sutro XpressCF)","mechanism":"Anti-FRα antibody with a hemiasterlin microtubule inhibitor conjugated at defined non-natural amino acids.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"lvgn6051","kind":"drug","name":"LVGN6051","aka":[],"tldr":"LVGN6051 is a monoclonal antibody from Lyvgen Biopharma Holdings Limited, in registered phase 2 trials for head and neck squamous cell carcinoma.","summary":"LVGN6051 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Lyvgen Biopharma Holdings Limited, in head and neck squamous cell carcinoma. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of LVGN6051","url":"https://clinicaltrials.gov/search?intr=LVGN6051"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["lyvgen-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06378177"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"LVGN6051","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"m108","kind":"drug","name":"M108","aka":["M108 monoclonal antibody"],"tldr":"M108 is an experimental monoclonal antibody from FutureGen Biopharmaceutical (Beijing) in phase 3 trials for gastric & gastro-oesophageal junction cancer, aimed at Claudin 18.2.","summary":"M108 is a monoclonal antibody developed by FutureGen Biopharmaceutical (Beijing). Its target is Claudin 18.2 (the sponsor names Claudin 18.2 (CLDN18.2)). ClinicalTrials.gov describes the intervention as: M108 monoclonal antibody will be administered as a minimum 2-hour IV infusion. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06177041 (M108 Plus CAPOX Versus Placebo Plus CAPOX as First-line Treatment for Claudin (CLDN) 18.2-Positive, HER2-Negative, PD-L1 CPS<5, Locally Advanced Unresectable or Metastatic Gastric or Gastroesophageal Junction (GEJ) Adenocarcinoma), in gastric & gastro-oesophageal junction cancer. The largest, NCT06177041, plans to enrol 486 participants with primary completion expected 2027-01-11. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of M108","url":"https://clinicaltrials.gov/search?intr=M108"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric"],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06177041"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at Claudin 18.2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"m701","kind":"drug","name":"M701","aka":[],"tldr":"M701 is Wuhan YZY Biopharma's EpCAM-CD3 bispecific antibody infused into the abdomen to control malignant ascites, in a phase 3 trial in China.","summary":"Malignant ascites, fluid that builds up in the abdomen from cancer spread to the peritoneum, is drained repeatedly for symptom relief. Catumaxomab, an EpCAM-CD3 bispecific, was approved in Europe in 2009 for this use and later withdrawn. M701 revives the approach: YZY Biopharma's phase 3 trial in China compares intraperitoneal M701 with paracentesis alone for the time until fluid must be drained again.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of M701","url":"https://clinicaltrials.gov/search?intr=M701"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian","gastric"],"sections":[],"technologies":["bispecific-antibody","t-cell-engager"],"targets":["epcam","cd3"],"drugs":[],"companies":["wuhan-yzy-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06432296"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"modality":"Bispecific antibody (EpCAM x CD3), intraperitoneal","mechanism":"One arm binds EpCAM on tumour cells in the ascites fluid and the other engages CD3 on T cells, bringing T cells to kill the cancer cells that cause malignant ascites, the mechanism of the earlier drug catumaxomab.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"magrolimab","kind":"drug","name":"Magrolimab","aka":[],"tldr":"The first 'don't eat me' signal blocker. Gilead paid $4.9B for it; it was stopped in 2024 after trials showed more deaths, not fewer.","summary":"Magrolimab blocks CD47 so macrophages can phagocytose tumour cells. Early single-arm data with azacitidine in TP53-mutant AML and MDS were striking. ENHANCE (higher-risk MDS) was stopped for futility in 2023; ENHANCE-2 (TP53-mutant AML) and ENHANCE-3 (unfit AML) were halted in 2024 after an increased risk of death in the magrolimab arms; the FDA placed a full clinical hold. Gilead had acquired Forty Seven for $4.9B in 2020.\n\nLesson: a ubiquitous target (CD47 is on every red cell) plus an immunosuppressed population is a narrow window; single-arm response rates in TP53-mutant disease were not predictive of survival.","status":"withdrawn","asOf":"2026-09-06","links":[{"label":"Gilead discontinues magrolimab in AML (Feb 2024) (page moved; nearest live section)","url":"https://www.gilead.com/news-and-press/press-room/press-releases/"}],"tags":["failure","lesson:toxicity"],"related":[],"cancers":["aml"],"sections":[],"technologies":["monoclonal-antibody","cd47-blockade"],"targets":["cd47"],"drugs":[],"companies":["gilead"],"institutions":[],"pathways":["cd47-sirpa"],"terms":[],"trials":["nct04313881","nct05079230","nct04778397"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"Hu5F9-G4, GS-4721","modality":"Monoclonal antibody (anti-CD47)","mechanism":"Humanised IgG4 anti-CD47; blocks the CD47-SIRPα 'don't eat me' signal, enabling macrophage phagocytosis.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mammaprint","kind":"drug","name":"MammaPrint (70-gene signature)","aka":[],"tldr":"A 70-gene test that tells whether an early breast cancer is genomically low or high risk, used to decide who can skip chemotherapy.","summary":"MammaPrint is Agendia's 70-gene signature. MINDACT (n=6,693): women with high clinical but low genomic risk had 5-year distant metastasis-free survival of 94.7% without chemotherapy; 8-year update showed a small benefit of chemotherapy in women under 50. FDA-cleared (2007), NCCN-listed alongside Oncotype DX; BluePrint adds molecular subtyping. Also used to identify 'ultralow' risk tumours with indolent behaviour.","status":"established","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/MammaPrint","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/MammaPrint"}],"tags":[],"related":[],"cancers":["breast-hr-positive","hr-positive-early-high-risk"],"sections":[],"technologies":["rna-seq","companion-diagnostic","gene-expression-prognostic-assays"],"targets":[],"drugs":[],"companies":["agendia"],"institutions":[],"pathways":[],"terms":[],"trials":["mindact","tailorx"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Gene-expression prognostic assay","mechanism":"Microarray or NGS expression of 70 genes → dichotomous low/high risk.","approvals":[{"region":"US","year":2007,"indication":"FDA 510(k) clearance for recurrence risk in early breast cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"margetuximab","kind":"drug","name":"Margetuximab","aka":[],"tldr":"A trastuzumab look-alike with an engineered tail that binds immune cells more tightly; approved in 2020 but rarely used after ADCs arrived.","summary":"Margetuximab is an anti-HER2 IgG1 antibody whose Fc region is engineered to bind the activating receptor FcγRIIIa (CD16A) more tightly and the inhibitory FcγRIIb less, enhancing antibody-dependent cellular cytotoxicity; its HER2-binding arm is otherwise trastuzumab-like. It is approved for HER2-positive metastatic breast cancer after two or more anti-HER2 regimens, given with chemotherapy at 15 mg/kg every 3 weeks. SOPHIA (versus trastuzumab, both with chemotherapy) showed PFS of 5.8 versus 4.9 months (HR 0.76); overall survival was not significantly improved, with a suggestion of benefit in CD16A-158F carriers. It was approved in December 2020 but is rarely used since antibody-drug conjugates arrived. It illustrates the ceiling of Fc engineering alone versus payload delivery. For a newcomer, it is a trastuzumab look-alike with a sharper immune tail that did not change practice.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Margetuximab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Margetuximab"}],"tags":[],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["her2"],"drugs":[],"companies":["macrogenics"],"institutions":[],"pathways":[],"terms":["adcc","fc-effector"],"trials":["nct02492711","nct04082364","nct04425018"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Margenza","modality":"Fc-engineered monoclonal antibody (anti-HER2)","mechanism":"Anti-HER2 IgG1 with Fc mutations increasing FcγRIIIa (CD16A) affinity and decreasing FcγRIIb binding, enhancing ADCC.","approvals":[{"region":"US","year":2020,"indication":"HER2+ metastatic breast cancer after ≥2 anti-HER2 regimens, with chemotherapy"}],"mechanismSteps":[],"dosing":{"route":"Intravenous","schedule":"15 mg/kg every 3 weeks with chemotherapy"},"toxicity":[{"event":"Infusion-related reactions","anyGradePct":13},{"event":"Left ventricular dysfunction","anyGradePct":2}],"access":[],"regulatoryEvents":[]},{"id":"masofaniten","kind":"drug","name":"Masofaniten","aka":[],"tldr":"A drug designed to block the part of the androgen receptor that resistant variants keep; its phase 2 was stopped for futility and development ended.","summary":"ESSA Pharma terminated the phase 2 of masofaniten + enzalutamide in mCRPC (2024) after a futility analysis on PSA90; the company later discontinued development (2025). The EXTRA-PC investigator trial in mHSPC closed early. AR-NTD inhibition remains unproven clinically despite compelling biology (AR-V7).","status":"withdrawn","asOf":"2026-09-06","links":[{"label":"ESSA terminates phase 2 (OncLive)","url":"https://www.onclive.com/view/essa-discontinues-phase-2-study-of-masofaniten-plus-enzalutamide-in-mcrpc"}],"tags":["failed-so-far"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["androgen-receptor"],"drugs":[],"companies":["essa-pharma"],"institutions":[],"pathways":[],"terms":["ar-v7"],"trials":["nct06312670"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"EPI-7386","modality":"Small-molecule AR N-terminal domain inhibitor","mechanism":"Binds the disordered N-terminal domain of AR, intended to inhibit full-length and splice-variant AR.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mavorixafor","kind":"drug","name":"Mavorixafor","aka":["X4P-001","AMD-070"],"tldr":"Mavorixafor is a once-daily tablet that blocks CXCR4, a receptor that traps white cells in the bone marrow. It is approved for the rare immunodeficiency WHIM syndrome and is being tested in Waldenström macroglobulinaemia, where CXCR4 mutations blunt the response to ibrutinib.","summary":"Mavorixafor is X4 Pharmaceuticals' oral CXCR4 antagonist. The FDA approved it in April 2024 for WHIM syndrome, a rare primary immunodeficiency caused by gain-of-function CXCR4 mutations, where it raises neutrophil and lymphocyte counts and reduces infections. The same mutations occur somatically in 30 to 40 percent of Waldenström macroglobulinaemia, where they lower and slow responses to BTK inhibitors.\n\nA phase 1b trial combined mavorixafor with ibrutinib in CXCR4-mutant Waldenström macroglobulinaemia and reported deeper responses than expected from ibrutinib alone in this subgroup. The Waldenström page names the trials as one answer to the open problem of CXCR4-mutant disease.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Mavorixafor","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Mavorixafor"}],"tags":["subtype-drugs-wave"],"related":["ibrutinib"],"cancers":["waldenstrom"],"sections":[],"technologies":[],"targets":["cxcr4"],"drugs":[],"companies":["x4-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02823405","nct04274738"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Xolremdi","code":"X4P-001","modality":"Oral CXCR4 antagonist","mechanism":"Blocks the chemokine receptor CXCR4, releasing neutrophils and lymphocytes from the marrow; in Waldenström macroglobulinaemia, activating CXCR4 mutations in about a third of patients cause resistance to BTK inhibitors, which the antagonist is meant to overcome.","approvals":[{"region":"US","year":2024,"indication":"WHIM syndrome (warts, hypogammaglobulinaemia, infections and myelokathexis) in patients aged 12 and over"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mavrostobart","kind":"drug","name":"Mavrostobart","aka":["Mavrostobart (PT199)"],"tldr":"Mavrostobart is an experimental monoclonal antibody from Phanes Therapeutics in phase 2 trials for non-small-cell lung cancer and pancreatic ductal adenocarcinoma, aimed at CD73 / adenosine axis.","summary":"Mavrostobart (PT199) is a monoclonal antibody developed by Phanes Therapeutics. Its target is CD73 / adenosine axis. ClinicalTrials.gov describes the intervention as: Mavrostobart (PT199) is an anti-CD73 mAb with a differentiated mechanism of action. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer and pancreatic ductal adenocarcinoma. The largest, NCT05431270, plans to enrol 40 participants with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Mavrostobart","url":"https://clinicaltrials.gov/search?intr=PT199"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","pancreatic"],"sections":[],"technologies":[],"targets":["cd73-adenosine"],"drugs":[],"companies":["phanes-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05431270"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PT199","modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at CD73 / adenosine axis, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mcla-129","kind":"drug","name":"MCLA-129","aka":["bispecific"],"tldr":"MCLA-129 is an experimental bispecific antibody from Betta Pharmaceuticals in phase 2 trials for non-small-cell lung cancer, head and neck squamous cell carcinoma and colorectal cancer, aimed at EGFR and MET.","summary":"MCLA-129 is a bispecific antibody developed by Betta Pharmaceuticals and Merus. Its targets are EGFR and MET (the sponsor names EGFR x c-Met). The sponsor states: A human bispecific antibody targeting both EGFR and c-Met, simultaneously blocking the signalling pathways of both receptors to inhibit tumour growth and survival. ClinicalTrials.gov describes the intervention as: MCLA-129 is a bispecific antibody that targets both EGFR and c-Met, simultaneously blocking the signaling pathways of both EGFR and c-Met, thereby inhibiting tumour growth and survival. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in non-small-cell lung cancer, head and neck squamous cell carcinoma, colorectal cancer, gastric & gastro-oesophageal junction cancer and oesophageal cancer. The largest, NCT04930432, plans to enrol 400 participants with primary completion expected 2028-04-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of MCLA-129","url":"https://clinicaltrials.gov/search?intr=MCLA-129"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","head-and-neck","colorectal","gastric","esophageal"],"sections":[],"technologies":[],"targets":["egfr","met"],"drugs":[],"companies":["betta","genmab"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04930432","nct04868877","nct07448116","nct07208149"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"A human bispecific antibody targeting both EGFR and c-Met, simultaneously blocking the signalling pathways of both receptors to inhibit tumour growth and survival.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mebendazole","kind":"drug","name":"Mebendazole","aka":["Vermox","MBZ"],"tldr":"Mebendazole is a human worm medicine that has been through two very small cancer trials: a dose-finding study in brain tumours found a tolerable dose, and a study in advanced bowel and stomach cancers saw every patient's cancer keep growing.","summary":"Reverse swing-M, a phase 1 study of 11 patients with recurrent glioblastoma, added mebendazole to re-irradiation with temozolomide, to lomustine or to temozolomide alone, and set a recommended phase 2 dose of 1600 mg three times a day with temozolomide and 800 mg three times a day with lomustine, with anaemia in 82 percent and nausea in 64 percent the most common adverse events (Reverse swing-M phase 1 2020). A phase 2a study in treatment-refractory gastrointestinal cancer treated 10 of 11 enrolled patients with individually dosed mebendazole up to 4 g a day, saw no severe adverse effects, and stopped every patient for progressive disease by the eight-week scan, four of them meeting suggested criteria for hyperprogression (Mebendazole phase 2a 2021). A telemedicine cohort of 197 patients prescribed compounded ivermectin with mebendazole reported self-assessed benefit, and the journal has placed an expression of concern on the paper while it audits the data (Anticancer Research expression of concern 2026). Combined ivermectin and benzimidazole prescribing to US cancer patients rose 2.6-fold after a January 2025 podcast (Rockwell JAMA Network Open 2026).","status":"phase-2","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Mebendazole","links":[{"label":"Reverse swing-M phase 1 2020","url":"https://doi.org/10.1002/cam4.3094"},{"label":"Mebendazole phase 2a 2021","url":"https://doi.org/10.1038/s41598-021-88433-y"},{"label":"Anticancer Research expression of concern 2026","url":"https://doi.org/10.21873/anticanres.18276"},{"label":"Rockwell JAMA Network Open 2026","url":"https://doi.org/10.1001/jamanetworkopen.2026.16780"}],"tags":[],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":["fenbendazole-ivermectin-repurposing-claims","drug-repurposing"],"targets":[],"drugs":["ivermectin","fenbendazole","temozolomide","lomustine"],"companies":[],"institutions":[],"pathways":[],"terms":["rp2d","off-label","preclinical"],"trials":[],"people":[],"bottlenecks":["b-generic-repurposing","b-misinformation"],"keyPapers":["paper-patil-mebendazole-glioma-phase-1-cancer-med-2020","paper-mansoori-mebendazole-gi-cancer-phase-2a-sci-rep-2021","paper-hulscher-ivermectin-mebendazole-cohort-anticancer-res-2026"],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo agent (Claude Fable 5.1)","editedOn":"2026-09-24","note":"Written from cached ClinicalTrials.gov v2, Europe PMC, DailyMed, Drugs@FDA and WHO eEML responses; no figure appears without its source link."},"modality":"Oral benzimidazole anthelmintic for human worm infections, tested in two small cancer trials","mechanism":"Binds tubulin and blocks microtubule assembly in worms. In glioma and other cancer cell lines the same effect stops cell division; that is laboratory evidence, and the two human studies below are the clinical record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mecapegfilgrastim","kind":"drug","name":"Mecapegfilgrastim","aka":["Aiduo","19K"],"tldr":"Mecapegfilgrastim is Hengrui's long-acting G-CSF, approved in China in 2018 to prevent chemotherapy-induced neutropenia.","summary":"Mecapegfilgrastim (Aiduo) is a long-acting pegylated G-CSF developed by Jiangsu Hengrui. The NMPA approved it in May 2018 to reduce the incidence and duration of neutropenia in patients receiving myelosuppressive chemotherapy for non-myeloid cancers. It is one of the most widely used supportive-care biologics in China.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of mecapegfilgrastim","url":"https://clinicaltrials.gov/search?intr=mecapegfilgrastim"}],"tags":["china","nmpa-approved"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hengrui"],"institutions":["nmpa-cde"],"pathways":[],"terms":[],"trials":["nct05463601","nct05076682"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"HHPG-19K","modality":"Long-acting granulocyte colony-stimulating factor (growth factor support)","supportive":true,"mechanism":"A pegylated form of G-CSF that tells the bone marrow to make neutrophils, shortening the low-neutrophil period after chemotherapy.","approvals":[{"region":"CN","year":2018,"indication":"Prevention of chemotherapy-induced neutropenia in non-myeloid cancers"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mechlorethamine","kind":"drug","name":"Mechlorethamine (chlormethine)","aka":["Nitrogen mustard","Chlormethine","Ledaga"],"tldr":"Mechlorethamine, or nitrogen mustard, was the first chemotherapy ever used, in 1940s Hodgkin lymphoma. Today its main use is as a skin gel (Valchlor in the US, Ledaga in Europe) for early cutaneous T-cell lymphoma.","summary":"Intravenous mechlorethamine (Mustargen, approved 1949) was the M in MOPP, the first curative combination chemotherapy for Hodgkin disease, and was labelled for Hodgkin and non-Hodgkin lymphomas, leukaemias, mycosis fungoides, bronchogenic carcinoma and malignant effusions; the intravenous product has been discontinued in the US. The 0.016% gel (Valchlor) was approved in August 2013 for stage IA and IB mycosis fungoides-type cutaneous T-cell lymphoma after prior skin-directed therapy, on a randomised observer-blinded trial (Study 201) against a compounded ointment; the EU authorised Ledaga in 2017 for the same indication. Contact dermatitis is the main adverse effect of the gel; the intravenous form was a potent vesicant and emetogen.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Chlormethine","links":[{"label":"Study 201 (JAMA Dermatol 2013)","url":"https://doi.org/10.1001/2013.jamadermatol.541"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6a52e4c2-6a9f-4ebb-bc77-046b4f8bcd57"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/mechlorethaminehydrochloride"},{"label":"EPAR (Ledaga)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/ledaga"}],"tags":["nci-list","historic"],"related":[],"cancers":["hodgkin-lymphoma","cutaneous-t-cell-lymphoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["helsinn","recordati"],"institutions":[],"pathways":[],"terms":[],"trials":["study-201"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mustargen (historic) / Valchlor gel","modality":"Alkylating agent (nitrogen mustard); intravenous and topical gel","mechanism":"Bifunctional alkylating agent that forms interstrand DNA cross-links; the topical gel acts locally on malignant T cells in the skin.","approvals":[{"region":"US","year":1949,"indication":"Hodgkin disease and other lymphomas, leukaemias, mycosis fungoides (intravenous; discontinued)"},{"region":"US","year":2013,"indication":"Topical treatment of stage IA and IB mycosis fungoides-type CTCL after skin-directed therapy (Valchlor)"},{"region":"EU","year":2017,"indication":"Topical treatment of mycosis fungoides-type CTCL (Ledaga)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"megestrol","kind":"drug","name":"Megestrol acetate","aka":[],"tldr":"Megestrol acetate is a hormone tablet used to ease advanced breast and womb cancer, and as a liquid to improve appetite and weight in people with cancer or AIDS-related wasting.","summary":"Megestrol acetate tablets were approved in 1971 for palliative treatment of advanced carcinoma of the breast or endometrium (recurrent, inoperable or metastatic disease); the oral suspension (1993) is indicated for anorexia, cachexia or unexplained weight loss in AIDS and is used off label for cancer-associated anorexia, where trials show weight gain mostly as fat and fluid. In breast cancer it was a standard second-line endocrine agent before aromatase inhibitors, which proved superior in the pivotal letrozole and anastrozole trials; in endometrial cancer it remains a guideline option for low-grade hormone-receptor-positive disease. Thromboembolism, adrenal suppression and hyperglycaemia are the notable risks.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Megestrol_acetate","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=megestrol"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/megestrolacetate"}],"tags":["nci-list","generic","supportive"],"related":[],"cancers":["breast-hr-positive","endometrial"],"sections":[],"technologies":["endocrine-therapy","cachexia-appetite-pharmacotherapy"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06979596","nct07150663"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Megace","modality":"Synthetic progestin (hormonal)","mechanism":"Progestational agent; antitumour effect in hormone-responsive breast and endometrial cancer is thought to act through progesterone receptor signalling and suppression of the pituitary-gonadal axis; appetite stimulation is separate and not fully understood.","approvals":[{"region":"US","year":1971,"indication":"Palliative treatment of advanced carcinoma of the breast or endometrium"},{"region":"US","year":1993,"indication":"Anorexia, cachexia or unexplained weight loss in AIDS (oral suspension)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"melphalan","kind":"drug","name":"Melphalan (including hepatic delivery system)","aka":[],"tldr":"The myeloma chemotherapy that is also the standard high-dose conditioning before autologous transplant, and, delivered directly into the liver's blood supply or the eye, treats uveal melanoma metastases and retinoblastoma.","summary":"Approved 1964 (oral, myeloma; melphalan-prednisone was the standard for decades). High-dose melphalan 200 mg/m² with autologous stem-cell rescue remains standard consolidation in myeloma (IFM 2009, DETERMINATION). Hepzato Kit (2023): percutaneous hepatic perfusion for uveal melanoma liver metastases (FOCUS trial, 36% response). Intra-arterial and intravitreal melphalan are standard in retinoblastoma. Melflufen is a peptide-conjugated prodrug.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Melphalan","links":[{"label":"Label: Hepzato Kit (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Hepzato"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=melphalan"}],"tags":["gap-fill","generic"],"related":[],"cancers":["multiple-myeloma","uveal-melanoma","retinoblastoma","ovarian","myeloma-transplant-eligible"],"sections":[],"technologies":["cytotoxic-chemotherapy","autologous-stem-cell-transplant","percutaneous-hepatic-perfusion","isolated-limb-perfusion"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct05022901","nct06607458"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Alkeran / Evomela / Hepzato Kit","modality":"Alkylating agent (nitrogen mustard)","mechanism":"Bifunctional alkylator producing DNA interstrand cross-links; active in myeloma at conventional dose and as high-dose conditioning; regional delivery for eye melanoma liver metastases and retinoblastoma.","approvals":[{"region":"US","year":1964,"indication":"Multiple myeloma; non-resectable epithelial ovarian carcinoma"},{"region":"US","year":2016,"indication":"High-dose conditioning before autologous HSCT in myeloma (Evomela)"},{"region":"US","year":2023,"indication":"Uveal melanoma with unresectable hepatic metastases via hepatic delivery system (Hepzato Kit)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"melflufen","kind":"drug","name":"Melphalan flufenamide","aka":[],"tldr":"A myeloma drug given accelerated approval in 2021 and withdrawn in the US months later when its confirmatory trial suggested it shortened survival.","summary":"Melflufen is a lipophilic peptide-conjugated alkylator activated by aminopeptidases enriched in myeloma cells. Accelerated approval (February 2021) rested on the single-arm HORIZON response rate. The confirmatory OCEAN trial met its PFS endpoint but showed an overall survival detriment in a subgroup (patients with prior autologous transplant), leading to a partial clinical hold, an ODAC vote against, and US withdrawal (2021; formal FDA withdrawal 2024). It remains approved in the EU with restrictions.\n\nLesson: accelerated approval on response rate carries real risk when the confirmatory trial shows harm; the same subgroup could be a true signal or noise, and regulators in the US and EU read it differently.","status":"withdrawn","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Melphalan_flufenamide","links":[{"label":"FDA withdrawal of Pepaxto approval (Feb 2024) (page moved; nearest live section)","url":"https://www.fda.gov/drugs/drug-safety-and-availability/"}],"tags":["failure","lesson:regulatory"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["peptide-drug-conjugate"],"targets":[],"drugs":[],"companies":["oncopeptides"],"institutions":[],"pathways":[],"terms":["accelerated-approval"],"trials":["nct03151811","nct04649060","nct02963493"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Pepaxto","code":"melflufen","modality":"Peptide-drug conjugate","mechanism":"Peptidase-activated alkylating prodrug of melphalan.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2021-02-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-melphalan-flufenamide-relapsed-or-refractory-multiple-myeloma","indication":"In combination with dexamethasone for the treatment of adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody"},{"date":"2024-02-23","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.0 years after its accelerated approval.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-melphalan-flufenamide-relapsed-or-refractory-multiple-myeloma","indication":"In combination with dexamethasone for the treatment of adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD-38 directed monoclonal antibody"}]},{"id":"hepzato","kind":"drug","name":"Melphalan hepatic delivery system","aka":[],"tldr":"High-dose melphalan pumped through the liver's own blood supply while the blood leaving the liver is filtered, the first approved treatment for uveal melanoma that has spread to the liver.","summary":"The Hepzato Kit combines melphalan with a percutaneous hepatic perfusion system: catheters isolate the liver's circulation, deliver a high dose of melphalan into the hepatic artery, and filter the drug out of the blood leaving the liver before it returns to the body. In the FOCUS trial in uveal melanoma with liver metastases, a cancer with few effective options, about a third of patients responded. The FDA approved it in August 2023 for adults with uveal melanoma whose unresectable liver metastases affect less than half the liver and who have no disease outside the liver, or only limited disease amenable to surgery or radiation. Bone marrow suppression, bleeding and the risks of a major procedure under general anaesthesia are the main hazards.","status":"approved","asOf":"2026-09-24","links":[{"label":"FOCUS (Ann Surg Oncol 2024)","url":"https://doi.org/10.1245/s10434-024-15293-x"},{"label":"Hepzato Kit label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=4f83c8f7-4cc0-4219-88d5-7cfddce91198"},{"label":"FDA approval (August 2023): melphalan as a liver-directed treatment for uveal melanoma","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-melphalan-liver-directed-treatment-uveal-melanoma"}],"tags":[],"related":[],"cancers":["uveal-melanoma"],"sections":["surgery","chemotherapy"],"technologies":[],"targets":[],"drugs":[],"companies":["delcath"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02678572"],"people":[],"bottlenecks":[],"keyPapers":["paper-zager-ann-surg-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Hepzato Kit","modality":"Liver-directed chemotherapy with percutaneous hepatic perfusion","mechanism":"Regional perfusion exposes liver metastases to melphalan concentrations far above what systemic dosing allows, while extracorporeal filtration limits marrow toxicity.","approvals":[{"region":"US","year":2023,"indication":"Uveal melanoma with unresectable hepatic metastases affecting less than half the liver and no or limited extrahepatic disease"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"memantine","kind":"drug","name":"Memantine","aka":["Ebixa"],"tldr":"Memantine is an Alzheimer's drug taken during whole-brain radiotherapy to protect memory. With hippocampal-sparing radiotherapy it has become the standard way to limit the thinking problems the treatment causes.","summary":"Memantine is an NMDA-receptor antagonist approved by the FDA in 2003 for moderate to severe Alzheimer's disease. In neuro-oncology it is given orally for 24 weeks starting with whole-brain radiotherapy. RTOG 0614 showed it delayed cognitive decline, and NRG CC001 (Journal of Clinical Oncology, 2020) showed that memantine plus hippocampal-avoidance intensity-modulated radiotherapy preserved cognition better than memantine with conventional whole-brain radiotherapy, without loss of intracranial control.\n\nIt does not treat the tumour itself. The secondary brain tumours page names it as the standard neuroprotective companion whenever whole-brain radiotherapy is still used rather than stereotactic radiosurgery.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Memantine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Memantine"}],"tags":["subtype-drugs-wave"],"related":["wbrt"],"cancers":["secondary-brain-tumours"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Namenda","modality":"Oral NMDA receptor antagonist (neuroprotectant)","supportive":true,"mechanism":"A low-affinity blocker of the NMDA glutamate receptor that limits the excitotoxic damage radiation causes to hippocampal neurons, given during and after whole-brain radiotherapy.","approvals":[{"region":"US","year":2003,"indication":"Moderate to severe Alzheimer's disease (use as a neuroprotectant during whole-brain radiotherapy is off-label)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"men1703","kind":"drug","name":"MEN1703","aka":["Dapolsertib hydrochloride"],"tldr":"MEN1703 is an experimental small molecule (kinase inhibitor) from Ryvu Therapeutics in phase 2 trials for hodgkin lymphoma, aimed at FLT3.","summary":"MEN1703 (SEL24) is a small molecule (kinase inhibitor) developed by Ryvu Therapeutics. Its target is FLT3 (the sponsor names PIM/FLT3 kinase). ClinicalTrials.gov describes the intervention as: MEN1703 (Dapolsertib hydrochloride) is a potent dual inhibitor of proviral integration site for Moloney murine leukaemia virus (PIM) kinases and Fms-like tyrosine kinase 3 (FLT3). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in hodgkin lymphoma. The largest, NCT06534437, plans to enrol 178 participants with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of MEN1703","url":"https://clinicaltrials.gov/search?intr=SEL24"},{"label":"Sponsor page","url":"https://www.ryvu.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":["flt3"],"drugs":[],"companies":["ryvu-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06534437"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"SEL24","modality":"small molecule (kinase inhibitor)","mechanism":"Small molecule (kinase inhibitor) directed at FLT3, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mercaptopurine","kind":"drug","name":"Mercaptopurine","aka":["6-MP","Xaluprine"],"tldr":"Mercaptopurine is a daily tablet, or a liquid for children, that keeps acute lymphoblastic leukaemia in remission during the long maintenance phase of treatment.","summary":"Mercaptopurine, developed by Elion and Hitchings and approved in 1953, is indicated for acute lymphoblastic leukaemia as part of a combination maintenance regimen; daily oral mercaptopurine with weekly methotrexate for two to three years is the backbone of maintenance in every paediatric and adult ALL protocol. Purixan (2014) is an oral suspension that made accurate paediatric dosing possible; the EU authorised the equivalent Xaluprine in 2012. TPMT and NUDT15 genotyping is recommended before starting because poor metabolisers suffer life-threatening myelosuppression on standard doses; hepatotoxicity and a small excess of later malignancy are recognised. Thioguanine is the related agent used in AML.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Mercaptopurine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=mercaptopurine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/mercaptopurine"},{"label":"EPAR (Xaluprine)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/xaluprine"}],"tags":["nci-list","generic"],"related":["methotrexate","thioguanine"],"cancers":["all-leukemia"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct03150693","nct02883049","nct00557193"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Purinethol / Purixan","modality":"Thiopurine antimetabolite","mechanism":"Purine analogue converted to thioguanine nucleotides that are incorporated into DNA and RNA and inhibit de novo purine synthesis; inactivated by TPMT and NUDT15, whose variants dictate dosing.","approvals":[{"region":"US","year":1953,"indication":"Acute lymphoblastic leukaemia, combination maintenance therapy"},{"region":"US","year":2014,"indication":"Oral suspension for ALL maintenance (Purixan)"},{"region":"EU","year":2012,"indication":"ALL in adults, adolescents and children (Xaluprine oral suspension)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"merestinib","kind":"drug","name":"Merestinib","aka":["LY2801653"],"tldr":"Merestinib is an oral kinase inhibitor from Eli Lilly and Company, in registered phase 2 trials for metastatic cancer.","summary":"Merestinib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Eli Lilly and Company, in metastatic cancer. A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Merestinib","url":"https://clinicaltrials.gov/search?intr=Merestinib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02711553"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral kinase inhibitor","mechanism":"A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mesna","kind":"drug","name":"Mesna","aka":["Uromitexan","Sodium 2-mercaptoethanesulfonate"],"tldr":"Mesna is given with the chemotherapy ifosfamide (and high-dose cyclophosphamide) to stop its breakdown products from burning the bladder lining and causing bleeding.","summary":"Mesna was approved in December 1988 as a prophylactic agent to reduce the incidence of ifosfamide-induced haemorrhagic cystitis, with oral tablets approved in 2002 allowing intravenous-then-oral schedules; it is given with every ifosfamide dose (sarcoma, germ cell, lymphoma regimens such as ICE and VeIP) and with high-dose cyclophosphamide in transplant conditioning. The label notes it does not prevent haematuria from other causes such as thrombocytopenia. Hypersensitivity reactions, including rare severe skin reactions, and false-positive urine ketone tests are the practical issues; nausea and headache are common.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Mesna","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=mesna"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/mesna"}],"tags":["nci-list","supportive","generic"],"related":["ifosfamide","cyclophosphamide"],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["baxter"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07255664","nct06119685"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mesnex","modality":"Uroprotective thiol","supportive":true,"mechanism":"Free thiol excreted in urine that binds and detoxifies acrolein and 4-hydroxy-ifosfamide, the urotoxic metabolites of ifosfamide and cyclophosphamide, in the bladder.","approvals":[{"region":"US","year":1988,"indication":"Prophylaxis of ifosfamide-induced haemorrhagic cystitis (oral tablets 2002)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"methotrexate","kind":"drug","name":"Methotrexate","aka":[],"tldr":"The 1948 antifolate that produced the first chemotherapy remissions in childhood leukaemia and the first cure of a solid tumour; still essential in ALL, lymphoma, osteosarcoma and CNS lymphoma.","summary":"Farber (1948) with aminopterin; methotrexate cured choriocarcinoma (1956). Roles: ALL maintenance and CNS prophylaxis (intrathecal), high-dose regimens in osteosarcoma (MAP), PCNSL and Burkitt/DLBCL CNS prophylaxis, GTN, head and neck and bladder (MVAC). Toxicities: mucositis, nephrotoxicity (high dose; glucarpidase rescue), hepatotoxicity, pneumonitis. Also the standard DMARD in rheumatoid arthritis and used in ectopic pregnancy.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Methotrexate","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=methotrexate%20injection"}],"tags":["gap-fill","generic","who-essential"],"related":["glucarpidase"],"cancers":["all-leukemia","osteosarcoma","primary-cns-lymphoma","gestational-trophoblastic","dlbcl","urothelial","head-and-neck","low-risk-gtn","high-risk-gtn","placental-site-trophoblastic-tumour"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["blood-brain-barrier"],"trials":["euramos-1","dbcg-82bc","nct05663866","nct06960577","nct07015242","nct07387926"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Trexall / Otrexup / Xatmep","modality":"Antifolate (DHFR inhibitor)","mechanism":"Inhibits dihydrofolate reductase, depleting tetrahydrofolate needed for thymidylate and purine synthesis; high doses with leucovorin rescue cross the blood-brain barrier.","approvals":[{"region":"US","year":1953,"indication":"ALL, choriocarcinoma, and later osteosarcoma, breast, head and neck, lung, lymphoma, mycosis fungoides"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"methoxsalen-ecp","kind":"drug","name":"Methoxsalen (extracorporeal photopheresis)","aka":["8-Methoxypsoralen","8-MOP"],"tldr":"Uvadex is the drug used in photopheresis, where a patient's white cells are drawn off, treated with methoxsalen and ultraviolet light, and returned; it is approved for the skin symptoms of cutaneous T-cell lymphoma and widely used for graft-versus-host disease.","summary":"Extracorporeal photopheresis was developed by Richard Edelson at Yale in the 1980s. The sterile methoxsalen solution Uvadex was approved by the FDA in 1999 for palliative treatment of the skin manifestations of cutaneous T-cell lymphoma unresponsive to other therapy, used with the THERAKOS photopheresis system. Responses occur in about half of patients with erythrodermic disease and Sézary syndrome, and the treatment is unusually well tolerated. Its largest use today is off label in steroid-refractory chronic graft-versus-host disease after stem cell transplantation and in transplant rejection. Oral methoxsalen has been used with UVA (PUVA) for psoriasis since the 1950s.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Extracorporeal_photopheresis","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=uvadex"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Extracorporeal_photopheresis"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["peripheral-t-cell-lymphoma","cutaneous-t-cell-lymphoma"],"sections":[],"technologies":["gvhd-photopheresis"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["gvhd"],"trials":["nct01686594","nct04930653","nct07619898"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Uvadex","modality":"Psoralen photosensitiser used with ultraviolet A on white cells outside the body","mechanism":"A psoralen that, when activated by UVA light, cross-links DNA in treated lymphocytes; reinfusing the damaged malignant T cells provokes an immune response against the clone.","approvals":[{"region":"US","year":1999,"indication":"Palliative treatment of skin manifestations of cutaneous T-cell lymphoma unresponsive to other therapy, by extracorporeal photopheresis"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"methyl-aminolevulinate","kind":"drug","name":"Methyl aminolevulinate","aka":["MAL"],"tldr":"Methyl aminolevulinate is a cream applied to sun-damaged skin or a superficial basal cell carcinoma and then activated with red light, clearing precancerous and early skin cancers with better cosmetic results than surgery or freezing.","summary":"Methyl aminolevulinate photodynamic therapy has been approved in Europe since 2001 for actinic keratosis, superficial and nodular basal cell carcinoma and Bowen's disease, and in the United States since 2004 (Metvixia) for thin actinic keratoses of the face and scalp. Clearance rates for superficial basal cell carcinoma approach those of surgery in randomised trials with fewer scars, though recurrence over five years is somewhat higher, so guidelines reserve it for low-risk lesions and for patients with many lesions or poor healing. Pain during illumination is the main drawback. Galderma markets it.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Methyl_aminolevulinate","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Methyl_aminolevulinate"},{"label":"ChEMBL CHEMBL1201306","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201306"},{"label":"Arits et al., photodynamic therapy versus topical imiquimod versus topical fluorouracil for superficial basal-cell carcinoma, randomised trial of 601 patients (Lancet Oncology 2013)","url":"https://doi.org/10.1016/S1470-2045(13)70143-8"},{"label":"Jansen et al., five-year results of a randomised controlled trial comparing photodynamic therapy, topical imiquimod and topical 5-fluorouracil in superficial basal cell carcinoma (J Invest Dermatol 2018)","url":"https://doi.org/10.1016/j.jid.2017.09.033"},{"label":"Thomson et al., interventions for basal cell carcinoma of the skin: Cochrane review of 52 randomised trials and 6,690 participants (2020)","url":"https://doi.org/10.1002/14651858.CD003412.pub3"},{"label":"Cancer Research UK: photodynamic therapy for non-melanoma skin cancer","url":"https://www.cancerresearchuk.org/about-cancer/skin-cancer/treatment/photodynamic-therapy"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["basal-cell-carcinoma","skin-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["galderma"],"institutions":[],"pathways":[],"terms":["topical-and-destructive-treatment-bcc"],"trials":["mal-pdt-imiquimod-fluorouracil-superficial-bcc","mal-pdt-versus-surgery-nodular-bcc","mal-pdt-versus-cryotherapy-superficial-bcc","nct02144077"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Photodynamic therapy for superficial basal cell carcinoma, read as a patient would need it. In the Dutch three-arm trial of 601 patients, the proportion tumour-free at both three and twelve months was 72.8 percent after methyl-aminolevulinate photodynamic therapy, 83.4 percent after imiquimod and 80.1 percent after fluorouracil; by five years the probabilities of tumour-free survival were 62.7, 80.5 and 70.0 percent. Moderate to severe pain and a burning sensation during the treatment itself were what patients reported most often.","The case for it is cosmetic and it is a strong one. Against surgical excision for low-risk nodular and superficial basal cell carcinoma, the Cochrane review found good or excellent cosmetic outcomes at one year in 97.3 percent of patients' own ratings against 82.5 percent for surgery, and 87.1 percent against 46.6 percent on observer ratings, both moderate-certainty. The same review found recurrence of 36.4 percent against 0 percent at three years in a single 68-patient study of nodular disease in the head and neck, which is why it is offered for superficial rather than nodular lesions."],"brand":"Metvix / Metvixia","modality":"Topical photosensitiser cream for photodynamic therapy","mechanism":"A lipophilic ester of aminolevulinic acid that abnormal keratinocytes convert to protoporphyrin IX; red light three hours later generates singlet oxygen that destroys the lesion.","approvals":[{"region":"US","year":2004,"indication":"Thin non-hyperkeratotic actinic keratoses of the face and scalp, with red light photodynamic therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"methylnaltrexone","kind":"drug","name":"Methylnaltrexone","aka":[],"tldr":"Methylnaltrexone (Relistor) is an injection or tablet that relieves the severe constipation caused by strong painkillers in people with advanced cancer, without blocking the pain relief itself.","summary":"Methylnaltrexone was approved by the FDA in April 2008 for opioid-induced constipation in adults with advanced illness receiving palliative care when laxatives have failed, on two randomised placebo-controlled trials in hospice and palliative populations in which about half of patients had a bowel movement within four hours of the first subcutaneous dose versus around 15% on placebo; indications for chronic non-cancer pain and oral tablets followed in 2014 to 2016. The EU authorised Relistor in 2008. It is a standard second-line option in palliative care guidelines after conventional laxatives; naloxegol and naldemedine are oral alternatives. Abdominal pain, flatulence and nausea are common, and gastrointestinal perforation has been reported in patients with structural bowel disease. Marketed by Salix (Bausch Health).","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Methylnaltrexone","links":[{"label":"MNTX 302 (NEJM 2008)","url":"https://doi.org/10.1056/NEJMoa0707377"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c488fb7c-0a5b-487c-b452-996809d1cb99"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/methylnaltrexonebromide"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/relistor"}],"tags":["nci-list","supportive"],"related":[],"cancers":[],"sections":[],"technologies":["palliative-care","pain-management"],"targets":["oprm1"],"drugs":[],"companies":["bausch-health"],"institutions":[],"pathways":[],"terms":[],"trials":["mntx-302"],"people":[],"bottlenecks":[],"keyPapers":["paper-mntx-302-thomas-nejm-2008"],"journals":[],"dependsOn":[],"notes":[],"brand":"Relistor","modality":"Peripherally acting mu-opioid receptor antagonist","supportive":true,"mechanism":"Quaternary derivative of naltrexone that does not cross the blood-brain barrier; blocks mu-opioid receptors in the gut to reverse opioid-induced constipation without reducing analgesia or precipitating withdrawal.","approvals":[{"region":"US","year":2008,"indication":"Opioid-induced constipation in advanced illness receiving palliative care (chronic non-cancer pain and oral tablets added 2014 to 2016)"},{"region":"EU","year":2008,"indication":"Opioid-induced constipation in advanced illness receiving palliative care when laxatives are insufficient (Relistor)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"metyrapone","kind":"drug","name":"Metyrapone","aka":["Metyrapone capsules"],"tldr":"Metyrapone is an older tablet that blocks cortisol production in the adrenal glands. It is used to bring cortisol down quickly in Cushing's disease before or after pituitary surgery, and in the United States mainly as a diagnostic test.","summary":"Metyrapone has been used since the 1960s. In Europe it is licensed for the management of Cushing's syndrome, including Cushing's disease caused by a pituitary corticotroph adenoma, and is a mainstay for rapid pre-operative control of hypercortisolism or when surgery has failed. In the United States it is approved as a diagnostic agent for testing hypothalamic-pituitary ACTH function, and its therapeutic use is off-label.\n\nIt is taken several times a day and titrated against cortisol levels; adverse effects include nausea, dizziness, hirsutism and acne from androgen precursors, and hypertension and hypokalaemia from mineralocorticoid precursors. Osilodrostat is the newer, more potent inhibitor of the same enzyme. The pituitary tumours page names both.","status":"established","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Metyrapone","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Metyrapone"}],"tags":["subtype-drugs-wave"],"related":["osilodrostat"],"cancers":["pituitary-tumours"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Metopirone","modality":"Oral 11-beta-hydroxylase inhibitor","mechanism":"Inhibits adrenal 11-beta-hydroxylase, reducing cortisol synthesis; ACTH rises in response, so 11-deoxycortisol accumulates, which is the basis of its use as a test of pituitary reserve as well as a treatment.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mevrometostat","kind":"drug","name":"Mevrometostat","aka":[],"tldr":"An epigenetic drug that may re-sensitise prostate cancer to hormone therapy, in three phase 3 trials with enzalutamide.","summary":"Mevrometostat is an oral EZH2 inhibitor; blocking the methyltransferase reverses PRC2-mediated gene silencing and the AR-independent lineage programmes that let prostate cancer escape hormone therapy, which is intended to re-sensitise tumours to enzalutamide. In a phase 1/2 study after abiraterone, mevrometostat with enzalutamide gave rPFS of about 14 months versus 6 months with enzalutamide alone. Three phase 3 trials with enzalutamide are running: MEVPRO-1 (post-abiraterone mCRPC, open-label, about 600 men, started October 2024, completion around 2028), MEVPRO-2 (double-blind) and MEVPRO-3 (mCSPC, ASCO GU 2026 trial-in-progress). There is no approval yet, and whether the randomised trials confirm the early rPFS signal is the key question. For a newcomer, it is an epigenetic drug that may make hormone therapy work again.","status":"phase-3","asOf":"2026-09-06","links":[{"label":"MEVPRO-1/2 protocols (Future Oncology 2026)","url":"https://www.tandfonline.com/doi/full/10.1080/14796694.2026.2667441"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["ezh2","androgen-receptor"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["mevpro-1","nct06629779","nct07028853"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"PF-06821497","modality":"Small-molecule EZH2 inhibitor","mechanism":"EZH2 inhibition reverses PRC2-mediated silencing and AR-independent lineage programmes.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mezigdomide","kind":"drug","name":"Mezigdomide","aka":[],"tldr":"Mezigdomide is the most potent oral cereblon modulator, producing responses in about 40% of triple-class-refractory myeloma with dexamethasone alone.","summary":"Mezigdomide is the most potent oral cereblon E3 ligase modulator (CELMoD) in development, producing rapid and deep degradation of Ikaros and Aiolos with strong T-cell co-stimulation. In CC-92480-MM-001, mezigdomide with dexamethasone alone produced an ORR of 41% in heavily pretreated, triple-class-refractory myeloma, including responses in 30% of patients with extramedullary disease, a group that responds poorly to most therapies. Two phase 3 trials are ongoing: SUCCESSOR-1 compares Mezi-Vd with Pom-Vd, and SUCCESSOR-2 compares Mezi-Kd with Kd. The open questions are durability of response and whether cytopenias and infections limit its use in combination. BMS develops it alongside the related CELMoD iberdomide. It is a pill that acts on the same target as lenalidomide but strongly enough to work when that drug and its successors have failed.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov: trials of Mezigdomide","url":"https://clinicaltrials.gov/search?intr=CC-92480"}],"tags":[],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["protac-degrader","celmods"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05519085","nct05552976","nct05372354","nct03989414","nct06988488","nct06892522"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"CC-92480","modality":"CELMoD (cereblon E3 ligase modulator)","mechanism":"High-affinity cereblon modulator; rapid, deep Ikaros/Aiolos degradation with strong T-cell co-stimulation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mhb036c","kind":"drug","name":"MHB036C","aka":[],"tldr":"MHB036C is an antibody-drug conjugate from Minghui Pharmaceutical (Hangzhou) Ltd, in registered phase 2 trials for metastatic cancer.","summary":"MHB036C is listed on ClinicalTrials.gov as an intervention in 3 registered phase 2 trials sponsored by Minghui Pharmaceutical (Hangzhou) Ltd, in metastatic cancer. An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of MHB036C","url":"https://clinicaltrials.gov/search?intr=MHB036C"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["minghui-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06373406","nct07130383","nct07229599"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"MHB036C","modality":"Antibody-drug conjugate","mechanism":"An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mhb039a","kind":"drug","name":"MHB039A","aka":[],"tldr":"MHB039A is a bispecific antibody from Minghui Pharmaceutical (Hangzhou) Ltd, in registered phase 2 trials for metastatic cancer.","summary":"MHB039A is listed on ClinicalTrials.gov as an intervention in 4 registered phase 2 trials sponsored by Minghui Pharmaceutical (Hangzhou) Ltd, in metastatic cancer. A bispecific antibody, as described in the registry record: one molecule binds two different targets, most often a tumour antigen and an immune cell or a second tumour pathway. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of MHB039A","url":"https://clinicaltrials.gov/search?intr=MHB039A"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["minghui-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07130383","nct07126665","nct07229599","nct06345482"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"MHB039A","modality":"Bispecific antibody","mechanism":"A bispecific antibody, as described in the registry record: one molecule binds two different targets, most often a tumour antigen and an immune cell or a second tumour pathway.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"caris-mi-cancer-seek","kind":"drug","name":"MI Cancer Seek","aka":[],"tldr":"The first FDA-approved test that sequences all of a tumour's genes and gene activity at once, used to match patients to several targeted drugs.","summary":"Caris Life Sciences' MI Cancer Seek was approved by the FDA in November 2024 as the first companion diagnostic combining whole-exome and whole-transcriptome sequencing, with claims covering several targeted and immune therapies across breast, colorectal, melanoma and lung cancers alongside tumour-profiling claims. The broad readout underpins Caris's real-world database, and the company's argument is that exome-plus-transcriptome captures fusions, expression and signatures that panels miss. Turnaround and tissue requirements are larger than for targeted panels.","status":"approved","asOf":"2026-09-10","links":[{"label":"Caris: MI Cancer Seek (page moved; nearest live section)","url":"https://www.carislifesciences.com/products-and-services/molecular-profiling/"}],"tags":["test"],"related":[],"cancers":["colorectal","nsclc","melanoma","breast-hr-positive","pancreatic"],"sections":[],"technologies":["cgp","wes-wgs","rna-seq","companion-diagnostic"],"targets":[],"drugs":[],"companies":["caris"],"institutions":[],"pathways":[],"terms":["ngs"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-philip-kras-wild-type-pancreatic-ccr-2022","paper-sepulveda-molecular-biomarkers-colorectal-guideline-jco-2017"],"journals":[],"dependsOn":[],"notes":["What a result means: one sample yields the full genomic picture; the companion claims matter for approved drugs, and the rest supports trial matching.","Pancreatic ductal adenocarcinoma: the DNA and RNA profiling of 2,483 tumours that produced the KRAS wild-type fusion and immune-marker table was run on this laboratory's platform (Philip 2022)."],"brand":"MI Cancer Seek","modality":"Whole-exome and whole-transcriptome companion diagnostic test","mechanism":"Simultaneous whole-exome DNA and whole-transcriptome RNA sequencing from a single tumour sample, with companion diagnostic claims layered on top of the broad profile.","approvals":[{"region":"US","year":2024,"indication":"Companion diagnostic claims across several solid tumours, with tumour-profiling claims"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"kheiron-mia","kind":"drug","name":"Mia (Mammography Intelligent Assessment)","aka":[],"tldr":"A London-built breast screening AI trialled across NHS sites as a second reader, now folded into RadNet's DeepHealth after a 2024 acquisition.","summary":"Kheiron Medical Technologies (founded in London in 2016) received the CE mark for Mia in 2019 and won an NHS AI in Health and Care Award in 2020, which funded prospective evaluation of Mia as an independent second reader in NHS breast screening units, including a service evaluation in NHS Grampian. Retrospective multi-site studies in the UK and Hungary reported non-inferior performance to human readers. In 2024 Kheiron became part of DeepHealth, RadNet's AI subsidiary, and Mia's technology was integrated into DeepHealth's SmartMammo. The UK's national evaluation of AI in breast screening (the EDITH trial, announced 2025) will test several products at scale.","status":"established","asOf":"2026-09-10","links":[{"label":"DeepHealth: Kheiron Medical Technologies","url":"https://deephealth.com/kheiron-medical-technologies/"}],"tags":["test"],"related":[],"cancers":["breast-hr-positive","tnbc"],"sections":[],"technologies":["radiology-ai-screening","mammography"],"targets":[],"drugs":[],"companies":["kheiron-medical-technologies"],"institutions":["anchor-centre-aberdeen"],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: in the NHS the AI would replace one of two human readers; discordant cases go to arbitration by a radiologist, as now."],"brand":"Mia","modality":"AI mammography detection software (independent or second reader)","mechanism":"Deep-learning reader trained on multi-vendor mammograms, designed to act as an independent second reader in double-reading programmes such as the NHS Breast Screening Programme.","approvals":[{"region":"EU","year":2019,"indication":"CE mark, mammography decision support"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"midostaurin","kind":"drug","name":"Midostaurin","aka":[],"tldr":"The first drug to improve survival in FLT3-mutated AML, added to standard chemotherapy: median survival went from about two years to more than six.","summary":"RATIFY (n=717, FLT3-ITD or TKD, age 18-59): midostaurin with 7+3 and as maintenance improved OS (median 74.7 vs 25.6 months, HR 0.78). Approved 2017 for newly diagnosed FLT3-mutated AML and for systemic mastocytosis. Now being displaced in FLT3-ITD by quizartinib (QuANTUM-First) and challenged by gilteritinib in frontline trials; still the only option labelled for FLT3-TKD.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Midostaurin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Midostaurin"}],"tags":[],"related":["flt3-itd","flt3-tkd"],"cancers":["aml","systemic-mastocytosis","aml-flt3","advanced-systemic-mastocytosis"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["flt3","kit"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["ratify","nct03591510"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Rydapt","modality":"Small-molecule multikinase inhibitor (FLT3)","mechanism":"Type I multikinase inhibitor of FLT3 (ITD and TKD), KIT, PDGFR, VEGFR2, and PKC.","approvals":[{"region":"US","year":2017,"indication":"Newly diagnosed FLT3-mutated AML with 7+3 and consolidation; advanced systemic mastocytosis"}],"mechanismSteps":["Midostaurin binds the active conformation of FLT3","Constitutive FLT3-ITD/TKD signalling through STAT5, RAS/MAPK and PI3K stops","Blast proliferation halts and apoptosis resumes","Given with 7+3 during induction and consolidation, then 12 months of maintenance"],"dosing":{"route":"Oral","schedule":"50 mg twice daily on days 8-21 of each induction and consolidation cycle, then 50 mg twice daily continuously for up to 12 cycles of maintenance","monitoring":"QT interval, pulmonary symptoms, counts","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rydapt"},"toxicity":[{"event":"Febrile neutropenia","grade3PlusPct":84,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rydapt","note":"Both arms of RATIFY; not drug-specific"},{"event":"Nausea","anyGradePct":83},{"event":"Rash/desquamation","grade3PlusPct":14},{"event":"QT prolongation","note":"Monitor; avoid strong CYP3A4 inhibitors where possible"}],"access":[],"regulatoryEvents":[{"date":"2017-04-28","type":"approval","region":"US","note":"First FLT3 inhibitor approved"}]},{"id":"mifamurtide","kind":"drug","name":"Mifamurtide","aka":[],"tldr":"An immune-activating drug approved in Europe for osteosarcoma after a trial suggested it improved survival; the FDA never approved it, and it remains one of oncology's transatlantic disagreements.","summary":"Mifamurtide is a synthetic muramyl tripeptide, a NOD2 ligand, packaged in liposomes so it is taken up by monocytes and macrophages and switches them to a tumour-killing state. It is given with chemotherapy after complete resection of high-grade non-metastatic osteosarcoma in patients aged 2 to 30. In the INT-0133 trial (Meyers, JCO 2008) six-year overall survival was 78% versus 70% when mifamurtide was added to MAP chemotherapy, but the factorial design, confounded by a parallel ifosfamide question, made interpretation contentious. The EMA approved it in 2009 while the FDA issued a not-approvable letter in 2007, and the transatlantic disagreement persists. Chills, fever and fatigue are the typical infusion effects. Mifamurtide is an immune-activating drug whose benefit in bone cancer is accepted in Europe and doubted in the US on the same data.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Mifamurtide","links":[{"label":"EMA product information","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/mepact"}],"tags":["gap-fill"],"related":[],"cancers":["osteosarcoma"],"sections":[],"technologies":["cytokine-therapy"],"targets":[],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03643133","nct07787429","nct02441309"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mepact","modality":"Liposomal muramyl tripeptide (macrophage activator)","mechanism":"Synthetic NOD2 ligand delivered in liposomes to monocytes and macrophages, activating them to a tumoricidal state.","approvals":[{"region":"EU","year":2009,"indication":"High-grade resectable non-metastatic osteosarcoma after complete resection, with chemotherapy, ages 2-30"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mifepristone","kind":"drug","name":"Mifepristone","aka":["RU-486","RU486"],"tldr":"Mifepristone, best known as an abortion pill and a treatment for Cushing's syndrome, blocks progesterone receptors that most meningiomas carry. A large phase 3 trial found it did not slow inoperable meningiomas.","summary":"Mifepristone is a synthetic steroid that antagonises the progesterone receptor and, at high doses, the glucocorticoid receptor; it is licensed for medical termination of pregnancy and for hyperglycaemia in Cushing's syndrome. Because around 70 percent of meningiomas express progesterone receptors and grow faster in pregnancy, small series in the 1990s suggested antiprogestin therapy might control unresectable tumours.\n\nThe SWOG S9005 phase 3 trial randomised 164 patients with unresectable meningioma to mifepristone 200 mg daily or placebo for up to two years; there was no difference in failure-free or overall survival (Journal of Clinical Oncology, 2015). The meningioma page lists it with hydroxyurea, somatostatin analogues and interferon among the systemic treatments that failed.","status":"negative","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Mifepristone","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Mifepristone"}],"tags":["subtype-drugs-wave"],"related":[],"cancers":["meningioma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct05016349","nct02788981","nct06099769"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Oral progesterone and glucocorticoid receptor antagonist","mechanism":"Blocks the progesterone receptor, which most meningiomas express, and at higher doses the glucocorticoid receptor; the hope was that removing progesterone drive would slow the tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mirdametinib","kind":"drug","name":"Mirdametinib","aka":["PD0325901","PD 0325901","PD-0325901"],"tldr":"Mirdametinib (Gomekli in the US, Ezmekly in Europe) is a MEK-blocking capsule or dispersible tablet for adults and children with neurofibromatosis type 1 whose plexiform neurofibromas cannot be removed surgically; it is the first such drug approved for adults.","summary":"Mirdametinib was approved by the FDA in February 2025 for adults and children aged 2 and over with NF1 and symptomatic plexiform neurofibromas not amenable to complete resection, on the ReNeu phase 2b trial in which 41% of adults and 52% of children had a confirmed objective response by central review, with deep and durable responses and improvement in pain. The European Commission granted a conditional marketing authorisation for Ezmekly in July 2025 for the same population. It is the first MEK inhibitor approved for adults with this benign but disabling tumour (selumetinib covered children first). Rash, diarrhoea, nausea, musculoskeletal pain, reduced ejection fraction and ocular toxicity are class effects. Developed by SpringWorks Therapeutics, acquired by Merck KGaA in 2025.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Mirdametinib","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/ezmekly"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=mirdametinib"}],"tags":["ema-list"],"related":["selumetinib"],"cancers":[],"sections":[],"technologies":["kinase-inhibitors"],"targets":["mek"],"drugs":[],"companies":["springworks"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct06153173","nct07061951","nct07237100"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NF1 plexiform neurofibroma is a benign tumour with no cancer record; not linked to a cancer id."],"brand":"Gomekli / Ezmekly","modality":"Small-molecule allosteric MEK1/2 inhibitor","mechanism":"Non-ATP-competitive MEK1/2 inhibitor that penetrates the CNS; lowers ERK signalling in NF1-deficient Schwann-lineage cells driving plexiform neurofibromas.","approvals":[{"region":"US","year":2025,"indication":"NF1 with symptomatic plexiform neurofibromas not amenable to complete resection, adults and children 2 and over (Gomekli)"},{"region":"EU","year":2025,"indication":"Symptomatic, inoperable plexiform neurofibromas in NF1, age 2 and over (Ezmekly, conditional)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mirvetuximab-soravtansine","kind":"drug","name":"Mirvetuximab soravtansine","aka":[],"tldr":"Mirvetuximab soravtansine (Elahere) is the first ADC for ovarian cancer, for tumours with high folate receptor alpha.","summary":"Mirvetuximab soravtansine (Elahere) is an anti-folate-receptor-alpha antibody carrying the maytansinoid DM4 (drug-to-antibody ratio about 3.5) through a cleavable sulfo-SPDB disulfide linker, and the released payload crosses membranes to kill neighbouring cells. It is the first ADC for ovarian cancer, given at 6 mg/kg every 3 weeks for FRα-high platinum-resistant disease. Accelerated approval in 2022 rested on SORAYA, and full approval in 2024 followed MIRASOL, which showed overall survival of 16.5 versus 12.7 months against chemotherapy; EMA approval came in late 2024. Ocular toxicity defines the side-effect profile, with blurred vision in 45 percent and keratopathy in 37 percent of MIRASOL patients, so eye examinations and steroid and lubricating drops are built into treatment. Combination and earlier-line trials are ongoing. It works in ovarian cancer at the price of careful eye care.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Mirvetuximab_soravtansine","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Mirvetuximab%20soravtansine"}],"tags":[],"related":["folr1-expression"],"cancers":["ovarian","high-grade-serous-ovarian-cancer","platinum-resistant-ovarian-cancer"],"sections":[],"technologies":["adc"],"targets":["folr1"],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":["sulfo-spdb","ocular-toxicity"],"trials":["nct06682988","nct06365853","nct05456685","nct06890338","nct06390995","nct07059845","nct04274426"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Elahere","modality":"ADC","payload":"DM4 (maytansinoid), DAR ~3.5","linker":"Sulfo-SPDB, cleavable disulfide","mechanism":"Anti-FRα antibody with DM4; bystander-capable after disulfide cleavage.","approvals":[{"region":"US","year":2022,"indication":"FRα-high platinum-resistant ovarian cancer (full approval 2024)"},{"region":"EU","year":2024,"indication":"FRα+ platinum-resistant ovarian; 14 Nov 2024"}],"mechanismSteps":["Antibody binds Folate receptor alpha on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","DM4 is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"6 mg/kg adjusted ideal body weight every 3 weeks","modifications":"Hold for grade 2 keratopathy or blurred vision until grade ≤1; discontinue for grade 4 ocular events","monitoring":"Ophthalmic exam at baseline and every other cycle for 8 cycles; prophylactic steroid and lubricating eye drops","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2"},"toxicity":[{"event":"AST increased","anyGradePct":57,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"Fatigue","anyGradePct":47,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"Blurred vision","anyGradePct":45,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"ALT increased","anyGradePct":38,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"Keratopathy","anyGradePct":37,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"Peripheral neuropathy","anyGradePct":37,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"Abdominal pain","anyGradePct":34,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"Dry eye","anyGradePct":29,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"Diarrhoea","anyGradePct":29,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"},{"event":"Nausea","anyGradePct":27,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=00c424b5-6ccd-48ab-9e88-1986451120e2","note":"MIRASOL; all-grade rates"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.elahere.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for FRα-high platinum-resistant ovarian cancer (2025)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2022-11-14","type":"accelerated-approval","region":"US","note":"Accelerated approval, FRα-high platinum-resistant ovarian cancer (SORAYA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with folate receptor-alpha (FRα) positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Select patients for therapy based on an FDA-approved test"},{"date":"2024-03-22","type":"conversion","region":"US","note":"Full approval with OS benefit (MIRASOL)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with folate receptor-alpha (FRα) positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer, who have received one to three prior systemic treatment regimens. Select patients for therapy based on an FDA-approved test"},{"date":"2024-11","type":"approval","region":"EU","note":"EMA approval","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"mitazalimab","kind":"drug","name":"Mitazalimab","aka":["ADC-1013, JNJ-64457107"],"tldr":"Mitazalimab is an antibody-drug conjugate from Alligator Bioscience AB, in registered phase 2 trials for pancreatic ductal adenocarcinoma.","summary":"Mitazalimab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Alligator Bioscience AB, in pancreatic ductal adenocarcinoma. An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Mitazalimab","url":"https://clinicaltrials.gov/search?intr=Mitazalimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["alligator-bioscience"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04888312"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Antibody-drug conjugate","mechanism":"An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mitobronitol","kind":"drug","name":"Mitobronitol","aka":["Dibromomannitol","DBM"],"tldr":"Mitobronitol was a Hungarian-developed oral tablet used in Europe from the 1960s to control chronic myeloid leukaemia and polycythaemia, an alternative to busulfan that disappeared once hydroxycarbamide, interferon and imatinib took over.","summary":"Mitobronitol was developed at the Hungarian company Chinoin and marketed in several European countries for chronic myeloid leukaemia in its chronic phase and for polycythaemia vera and essential thrombocythaemia, achieving haematological control comparable to busulfan with less pulmonary fibrosis. Like other alkylating agents it carried a risk of secondary leukaemia, and it was never approved in the United States. Hydroxycarbamide and interferon alfa displaced it in the 1980s and 1990s, and imatinib made chemotherapy for chronic phase disease obsolete after 2001.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Mitobronitol","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Mitobronitol"},{"label":"ChEMBL CHEMBL1200645","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200645"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["cml","myeloproliferative-neoplasms"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":["busulfan","hydroxyurea"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Myelobromol","modality":"Oral alkylating sugar alcohol","mechanism":"A brominated mannitol that alkylates DNA after conversion to an epoxide, suppressing proliferating myeloid cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mitomycin","kind":"drug","name":"Mitomycin C","aka":[],"tldr":"The chemotherapy given with radiation to cure anal cancer without surgery, used as a bladder instillation after tumour resection, and since 2020-25 in gel form for upper-tract and recurrent bladder cancers.","summary":"Approved 1974 (gastric, pancreatic). Anal SCC chemoradiation (Nigro, ACT II), intravesical single instillation after TURBT for NMIBC, hyperthermic intravesical and HIPEC use (appendiceal/PMP), ophthalmic antifibrotic. Mitomycin gel (Jelmyto, 2020) for low-grade upper-tract urothelial carcinoma and intravesical gel (Zusduri, 2025) for recurrent low-grade intermediate-risk NMIBC. Haemolytic uraemic syndrome and pulmonary toxicity at cumulative doses.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Mitomycin_C","links":[{"label":"Label: Jelmyto (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Jelmyto"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=mitomycin"}],"tags":["gap-fill","generic"],"related":[],"cancers":["anal","urothelial","appendiceal","gastric","localised-anal-cancer","low-grade-appendiceal-mucinous-neoplasm","appendiceal-adenocarcinoma"],"sections":[],"technologies":["cytotoxic-chemotherapy","bcg-and-intravesical-therapy","hipec","imrt-igrt"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06211764","nct07566156","nct04241185"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mutamycin / Jelmyto / Zusduri","modality":"Alkylating antibiotic (bioreductive)","mechanism":"Reduced intracellularly to a bifunctional alkylator that cross-links DNA at CpG sites; preferentially activated in hypoxic cells, making it a radiosensitiser.","approvals":[{"region":"US","year":1974,"indication":"Disseminated gastric or pancreatic adenocarcinoma (with other agents)"},{"region":"US","year":2020,"indication":"Low-grade upper tract urothelial carcinoma (mitomycin pyelocalyceal gel)"},{"region":"US","year":2025,"indication":"Recurrent low-grade intermediate-risk NMIBC (intravesical gel)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mitotane","kind":"drug","name":"Mitotane","aka":[],"tldr":"A relative of the insecticide DDT that is the only drug specific to adrenal cortex cancer; it needs blood-level monitoring and permanent steroid replacement.","summary":"Approved 1970. Adjuvant use for high-risk resected ACC (Terzolo 2007; ADIUVO 2023 negative for low risk), monotherapy for indolent advanced disease, and combined with EDP chemotherapy (FIRM-ACT). Therapeutic range 14-20 mg/L; toxicities include neurologic effects, GI intolerance, adrenal insufficiency (hydrocortisone replacement at higher doses), CYP3A4 induction affecting other drugs.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Mitotane","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=mitotane"}],"tags":["gap-fill"],"related":[],"cancers":["adrenocortical","localised-adrenocortical-carcinoma","advanced-adrenocortical-carcinoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lysodren","modality":"Adrenolytic agent (DDD derivative)","mechanism":"Metabolised in adrenocortical cells to an acyl chloride that binds mitochondrial proteins and inhibits steroidogenesis (SOAT1 inhibition causes ER stress); cytotoxic to adrenocortical tissue.","approvals":[{"region":"US","year":1970,"indication":"Inoperable adrenocortical carcinoma (functional and non-functional)"},{"region":"EU","year":2004,"indication":"Advanced adrenocortical carcinoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mitoxantrone","kind":"drug","name":"Mitoxantrone","aka":[],"tldr":"Mitoxantrone is a blue chemotherapy infusion used with cytarabine for acute myeloid leukaemia and, historically, to relieve pain in advanced prostate cancer; it is also licensed for multiple sclerosis.","summary":"Mitoxantrone was approved in 1987 for acute non-lymphocytic leukaemia in adults in combination with other agents and later for pain relief in advanced hormone-refractory prostate cancer with corticosteroids, where it improved palliative endpoints without extending survival and was then displaced by docetaxel (TAX 327). It remains in AML salvage regimens such as MEC and FLAG-Ida alternatives and in some lymphoma protocols, and carries a separate indication for progressive multiple sclerosis. Cardiotoxicity (lifetime dose limits and ejection fraction monitoring), secondary acute leukaemia and blue-green discolouration of urine and sclerae are characteristic.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Mitoxantrone","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=mitoxantrone"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/mitoxantronehydrochloride"}],"tags":["nci-list","generic"],"related":[],"cancers":["aml","prostate"],"sections":[],"technologies":["cytotoxic-chemotherapy","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct03591510","nct04668690","nct03182244"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Novantrone (historic)","modality":"Anthracenedione (topoisomerase II inhibitor)","mechanism":"Synthetic anthracenedione that intercalates DNA, causes cross-links and strand breaks and inhibits topoisomerase II; less cardiotoxic per dose than anthracyclines but cumulative limits still apply.","approvals":[{"region":"US","year":1987,"indication":"Acute non-lymphocytic leukaemia in adults (combination); later hormone-refractory prostate cancer pain (with corticosteroids) and multiple sclerosis"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mk-1045","kind":"drug","name":"MK-1045","aka":[],"tldr":"MK-1045 is an experimental investigational agent whose form is not stated in the registry from Merck Sharp & Dohme in phase 3 trials for acute lymphoblastic leukaemia, follicular lymphoma and hodgkin lymphoma, aimed at CD19.","summary":"MK-1045 (CN-201, CN201) is an investigational agent whose form is not stated in the registry developed by Merck Sharp & Dohme and MSD R&D (China). Its target is CD19 (the sponsor names CD19). The sponsor states: MK-1045 is described as a novel immunotherapy, given intravenously, being compared with blinatumomab in relapsed/refractory CD19-positive B-cell acute lymphoblastic leukaemia. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07570173 (A Clinical Trial of MK-1045 in People With B-cell Acute Lymphoblastic Leukaemia (MK-1045-005)) and NCT07634471 (A Clinical Trial of MK-1045 and Rituximab in People With Follicular Lymphoma (MK-1045-007)), in acute lymphoblastic leukaemia, follicular lymphoma, hodgkin lymphoma and diffuse large B-cell lymphoma. The largest, NCT07634471, plans to enrol 960 participants with primary completion expected 2032-07-23. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of MK-1045","url":"https://clinicaltrials.gov/search?intr=CN-201"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["all-leukemia","follicular-lymphoma","hodgkin-lymphoma","dlbcl"],"sections":[],"technologies":[],"targets":["cd19"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07570173","nct07634471","nct07519772","nct05579132"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CN-201, CN201","modality":"bispecific antibody (CD3 x CD19, per the sponsor's description; form not stated in the registry record)","mechanism":"MK-1045 is described as a novel immunotherapy, given intravenously, being compared with blinatumomab in relapsed/refractory CD19-positive B-cell acute lymphoblastic leukaemia.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mobocertinib","kind":"drug","name":"Mobocertinib","aka":[],"tldr":"Mobocertinib was the first oral drug for EGFR exon 20 insertion lung cancer, approved in 2021 and withdrawn in 2023-24 after its confirmatory trial failed.","summary":"Mobocertinib is a covalent EGFR inhibitor designed for exon 20 insertion mutations, which resist earlier EGFR TKIs, while sparing wild-type EGFR relative to those agents. It received accelerated US approval in 2021 on a 28% response rate in patients who had progressed after platinum chemotherapy, the first oral drug for this subgroup. The confirmatory EXCLAIM-2 trial (2023) failed to beat platinum chemotherapy in the first-line setting, and Takeda withdrew the drug globally, with the US withdrawal completed in 2024. Diarrhoea and QT prolongation were prominent toxicities. Amivantamab and sunvozertinib now cover the exon 20 insertion indication. Mobocertinib shows how an accelerated approval can be reversed when the confirmatory trial does not deliver.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Mobocertinib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Mobocertinib"}],"tags":["gap-fill"],"related":[],"cancers":["nsclc","egfr-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":["egfr-exon20-insertion"],"trials":["nct04129502"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Exkivity","modality":"Small-molecule EGFR exon 20 insertion TKI (irreversible)","mechanism":"Covalent EGFR inhibitor designed for exon 20 insertion mutations, sparing wild-type EGFR relative to earlier TKIs.","approvals":[{"region":"US","year":2021,"indication":"EGFR exon 20 insertion NSCLC after platinum","note":"Withdrawn 2023-24"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2021-09-15","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy"},{"date":"2024-07-15","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 2.8 years after its accelerated approval.","indication":"Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy"}]},{"id":"mogamulizumab","kind":"drug","name":"Mogamulizumab","aka":[],"tldr":"Mogamulizumab is an antibody that clears cancerous T cells from the blood in mycosis fungoides and Sézary syndrome, the leukaemic form of skin lymphoma.","summary":"MAVORIC (2018): PFS 7.7 vs 3.1 months versus vorinostat, with the best responses in blood compartment and Sézary syndrome. Approved in Japan (2012) for ATLL and CCR4+ PTCL/CTCL; US/EU 2018 for MF/SS after one systemic therapy. Rash (mogamulizumab-associated rash) and increased GVHD risk after subsequent allogeneic transplant.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Mogamulizumab","links":[{"label":"MAVORIC (Lancet Oncol 2018)","url":"https://doi.org/10.1016/S1470-2045(18)30379-6"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=mogamulizumab"},{"label":"Kim et al., Lancet Oncol 2018: MAVORIC, mogamulizumab against vorinostat in previously treated cutaneous T-cell lymphoma","url":"https://doi.org/10.1016/S1470-2045(18)30379-6"},{"label":"Kataoka et al., Nat Genet 2015: integrated molecular analysis of 426 adult T-cell leukaemia/lymphoma cases","url":"https://doi.org/10.1038/ng.3415"}],"tags":["gap-fill"],"related":[],"cancers":["peripheral-t-cell-lymphoma","cutaneous-t-cell-lymphoma"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["ccr4"],"drugs":[],"companies":["kyowa-kirin"],"institutions":[],"pathways":[],"terms":["adcc","fc-effector"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-mavoric-mogamulizumab-lancet-oncol-2018"],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: CCR4 is on the tumour in more than 80% of mycosis fungoides and Sezary syndrome and about 90% of adult T-cell leukaemia/lymphoma, and it is also on regulatory T cells, so the antibody depletes those too. That explains both the rash and the association between giving it shortly before an allogeneic transplant and severe graft-versus-host disease. MAVORIC randomised 372 previously treated patients to mogamulizumab or vorinostat (Kim 2018). In adult T-cell leukaemia/lymphoma, CCR4 is not only expressed but mutated, with gain-of-function truncations of the cytoplasmic tail in about a quarter of cases (Kataoka 2015)."],"brand":"Poteligeo","modality":"Defucosylated anti-CCR4 monoclonal antibody","mechanism":"Binds CCR4 on malignant T cells (and regulatory T cells) and kills them through enhanced ADCC from the afucosylated Fc.","approvals":[{"region":"JP","year":2012,"indication":"Relapsed CCR4+ adult T-cell leukaemia/lymphoma; later PTCL/CTCL"},{"region":"US","year":2018,"indication":"Relapsed/refractory mycosis fungoides or Sézary syndrome after ≥1 systemic therapy"},{"region":"EU","year":2018,"indication":"MF/SS after ≥1 systemic therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"momelotinib","kind":"drug","name":"Momelotinib","aka":[],"tldr":"Momelotinib is the JAK inhibitor designed for anaemic myelofibrosis patients: it can improve haemoglobin while shrinking the spleen.","summary":"Momelotinib inhibits JAK1 and JAK2 to shrink the spleen and relieve symptoms, and additionally blocks ACVR1 (ALK2), which lowers hepcidin and frees iron for red-cell production. That second action is why it was developed for myelofibrosis patients whose anaemia rules out or limits other JAK inhibitors. MOMENTUM (2023) compared it with danazol in symptomatic anaemic patients after a prior JAK inhibitor: symptom response was superior, transfusion independence non-inferior, and spleen response better. The earlier SIMPLIFY-1 and SIMPLIFY-2 trials against ruxolitinib and best available therapy built the anaemia signal. It was approved in the US in 2023 and the EU in 2024 for myelofibrosis with anaemia. For a newcomer, momelotinib is the JAK inhibitor chosen when low haemoglobin is the dominant problem.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Momelotinib","links":[{"label":"MOMENTUM (Lancet 2023)","url":"https://doi.org/10.1016/S0140-6736(22)02036-0"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=momelotinib"}],"tags":["gap-fill"],"related":[],"cancers":["myeloproliferative-neoplasms","primary-myelofibrosis"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["jak2","jak1","acvr1"],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ojjaara / Omjjara","modality":"Small-molecule JAK1/JAK2/ACVR1 inhibitor","mechanism":"Inhibits JAK1/2 for spleen and symptoms and ACVR1 (ALK2) to lower hepcidin and improve anaemia.","approvals":[{"region":"US","year":2023,"indication":"Intermediate/high-risk myelofibrosis with anaemia"},{"region":"EU","year":2024,"indication":"Myelofibrosis with moderate-to-severe anaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"monalizumab","kind":"drug","name":"Monalizumab","aka":[],"tldr":"Monalizumab is an experimental investigational agent whose form is not stated in the registry from AstraZeneca in phase 3 trials for non-small-cell lung cancer and head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"Monalizumab (IPH2201) is an investigational agent whose form is not stated in the registry developed by AstraZeneca and MedImmune. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Participants will receive IV infusion of monalizumab as stated in arm description. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05221840 (A Global Study to Assess the Effects of Durvalumab With Oleclumab or Durvalumab With Monalizumab Following Concurrent Chemoradiation in Patients With Stage III Unresectable Non-Small Cell Lung Cancer) and NCT04590963 (Assessment of Efficacy and Safety of Monalizumab Plus Cetuximab Compared to Placebo Plus Cetuximab in Recurrent or Metastatic Head and Neck Cancer), in non-small-cell lung cancer and head and neck squamous cell carcinoma. The largest, NCT05221840, plans to enrol 1051 participants (actual) with primary completion expected 2026-09-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Monalizumab","url":"https://clinicaltrials.gov/search?intr=IPH2201"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05221840","nct04590963","nct05061550","nct02671435"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"IPH2201","modality":"antibody (INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mosunetuzumab","kind":"drug","name":"Mosunetuzumab","aka":[],"tldr":"Mosunetuzumab is a fixed-duration CD20 bispecific approved for follicular lymphoma and studied with polatuzumab in large B-cell lymphoma.","summary":"Mosunetuzumab is a full-length humanised IgG1 CD20xCD3 bispecific antibody that brings T cells to CD20-positive B cells, given with step-up dosing for a fixed 8 to 17 cycles depending on response. It was approved in December 2022 for relapsed or refractory follicular lymphoma after at least 2 lines, where it produced ORR 80% and CR 60%. In DLBCL, mosunetuzumab plus polatuzumab vedotin met its PFS endpoint in the phase 3 SUNMO trial against R-GemOx in transplant-ineligible relapsed disease in 2025, positioning the chemotherapy-free, outpatient doublet for patients not fit for CAR-T. A subcutaneous formulation was approved in 2025. Cytokine release syndrome occurred in 44% of the follicular lymphoma cohort but was grade 3 or higher in only 2%; Roche develops it. Its distinguishing point is fixed-duration treatment, so patients stop rather than continuing indefinitely.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Mosunetuzumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Mosunetuzumab"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["t-cell-engager"],"targets":["cd20","cd3"],"drugs":["polatuzumab-vedotin"],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["sunmo","nct04712097","nct06090539","nct04246086","nct05207670","nct03671018","nct06634589"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lunsumio","modality":"Bispecific T-cell engager (CD20×CD3)","mechanism":"Full-length humanised IgG1 CD20×CD3 bispecific with step-up dosing; fixed 8-17 cycles.","approvals":[{"region":"US","year":2022,"indication":"R/R follicular lymphoma after ≥2 lines (accelerated)"}],"mechanismSteps":[],"dosing":{"route":"IV or subcutaneous","schedule":"Step-up 1 mg, 2 mg, 60 mg in cycle 1, 60 mg cycle 2, 30 mg thereafter; 8 cycles if CR, up to 17 if PR/SD","monitoring":"CRS cycle 1; tumour flare; infections"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":44,"grade3PlusPct":2,"note":"GO29781 FL cohort"},{"event":"Neutropenia","grade3PlusPct":27}],"access":[],"regulatoryEvents":[{"date":"2022-12-22","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 3.7 years later, when the FDA's table was read.","indication":"Adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy."},{"date":"2025-12-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 0.7 years later, when the FDA's table was read.","indication":"Formulation: Treatment of adult patients with relapsed or refractory follicular lymphoma after two or more lines of systemic therapy."}]},{"id":"motixafortide","kind":"drug","name":"Motixafortide","aka":["BL-8040","BKT140"],"tldr":"Motixafortide is an injection given with G-CSF before stem cell collection in people with multiple myeloma, approved in the United States in 2023, so that enough stem cells for an autologous transplant can be harvested in one or two sessions.","summary":"BioLineRx's motixafortide blocks CXCR4 for far longer than plerixafor, the earlier drug of the class. In the GENESIS trial, a single dose with filgrastim let about 90 percent of multiple myeloma patients collect the target of six million CD34-positive cells per kilogram in up to two apheresis sessions, against about ten percent with filgrastim alone. The FDA approved it in September 2023, in combination with filgrastim, to mobilise haematopoietic stem cells for collection and autologous transplantation in patients with multiple myeloma. Injection site reactions and flushing are the common side effects. Ayrmid Pharma took over US commercial rights in 2024, and trials are exploring CXCR4 blockade with checkpoint inhibitors in pancreatic cancer.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Motixafortide","links":[{"label":"Drugs@FDA NDA217159","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=217159"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=motixafortide"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Motixafortide"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":["filgrastim","plerixafor"],"companies":["biolinerx"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06514508"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aphexda","modality":"CXCR4 antagonist peptide, subcutaneous injection","supportive":true,"mechanism":"A cyclic peptide that blocks the CXCR4 receptor, breaking the CXCL12 tether that holds stem cells in the marrow so they flood into the blood for collection.","approvals":[{"region":"US","year":2023,"indication":"With filgrastim, to mobilise haematopoietic stem cells for collection and autologous transplantation in multiple myeloma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"moxetumomab-pasudotox","kind":"drug","name":"Moxetumomab pasudotox","aka":[],"tldr":"An immunotoxin for hairy cell leukaemia that produced lasting remissions in relapsed patients but was withdrawn from sale in 2023 for commercial reasons.","summary":"Moxetumomab pasudotox is a recombinant immunotoxin: an anti-CD22 Fv fragment fused to a truncated Pseudomonas exotoxin A (PE38). After binding CD22 it is internalised and inactivates elongation factor 2 by ADP-ribosylation, shutting down protein synthesis in the leukaemic cell. It was approved in the US in 2018 for relapsed or refractory hairy cell leukaemia after at least two systemic therapies including a purine analogue. The pivotal study (Kreitman, Leukemia 2018) achieved a 41% complete remission rate, 30% of them durable, in multiply relapsed patients; capillary leak and haemolytic uraemic syndrome were the key toxicities. AstraZeneca withdrew it from the US market in 2023 because of low use, with compassionate supply continuing through the NIH. It remains an example of an effective rare-disease drug that failed commercially rather than clinically.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Moxetumomab_pasudotox","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Moxetumomab_pasudotox"}],"tags":["gap-fill"],"related":[],"cancers":["hairy-cell-leukemia"],"sections":[],"technologies":["adc"],"targets":["cd22"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":["pivotal-trial"],"trials":["nct01829711","nct02338050"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lumoxiti","modality":"Anti-CD22 immunotoxin (Pseudomonas exotoxin A fragment)","mechanism":"Recombinant anti-CD22 Fv fused to truncated Pseudomonas exotoxin PE38; internalised and inhibits protein synthesis via ADP-ribosylation of EF-2.","approvals":[{"region":"US","year":2018,"indication":"Relapsed/refractory hairy cell leukaemia after ≥2 systemic therapies including a purine analogue","note":"Withdrawn from market 2023"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mrg002","kind":"drug","name":"MRG002","aka":[],"tldr":"MRG002 is an experimental investigational agent whose form is not stated in the registry from Shanghai Miracogen in phase 3 trials for gastric & gastro-oesophageal junction cancer and bladder & urothelial cancer, with its target not yet stated publicly.","summary":"MRG002 is an investigational agent whose form is not stated in the registry developed by Shanghai Miracogen. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05754853 (A Study of MRG002 Versus Investigator's Choice of Chemotherapy in the Treatment of Patients With HER2-positive Unresectable Advanced or Metastatic Urothelial Cancer), in gastric & gastro-oesophageal junction cancer and bladder & urothelial cancer. The largest, NCT05754853, plans to enrol 290 participants with primary completion was scheduled for 2025-10 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of MRG002","url":"https://clinicaltrials.gov/search?intr=MRG002"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric","urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["shanghai-miracogen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05754853","nct04492488"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mrg003","kind":"drug","name":"MRG003","aka":[],"tldr":"MRG003 is an experimental antibody-drug conjugate from Lepu Biopharma in phase 3 trials for head and neck squamous cell carcinoma and nasopharyngeal carcinoma, with its target not yet stated publicly.","summary":"MRG003 is an antibody-drug conjugate developed by Lepu Biopharma. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05751512 (A Study to Evaluate MRG003 vs Cetuximab/Methotrexate in in the Treatment of Patients With RM-SCCHN), in head and neck squamous cell carcinoma and nasopharyngeal carcinoma. The largest, NCT05126719, plans to enrol 238 participants with primary completion was scheduled for 2024-12-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of MRG003","url":"https://clinicaltrials.gov/search?intr=MRG003"},{"label":"Sponsor page","url":"https://www.lepubiopharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["head-and-neck","nasopharyngeal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["lepu-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05751512","nct06976190","nct05126719"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mrtx1133","kind":"drug","name":"MRTX1133","aka":[],"tldr":"MRTX1133 was the first potent chemical tool against KRAS G12D, and proved the mutation could be drugged even though it lacks the reactive handle G12C has.","summary":"MRTX1133 is a non-covalent small molecule that binds the switch-II pocket of KRAS G12D and inhibits both the ON and OFF states; because G12D lacks the reactive cysteine that G12C drugs exploit, it needed a different, high-affinity chemistry. It was the first potent chemical tool against KRAS G12D, a common driver in pancreatic cancer, and it produced striking regressions in pancreatic patient-derived xenograft models (Nature 2023). Poor oral bioavailability forced intravenous dosing in the phase 1/2 trial, and development slowed after Bristol Myers Squibb acquired Mirati, while covalent RAS(ON) competitors advanced. Its lasting importance is as proof that G12D can be drugged, opening the door to oral and pan-RAS successors. For a newcomer, it showed that a KRAS mutation once thought undruggable can be hit.","status":"phase-1","asOf":"2026-09-06","links":[{"label":"ClinicalTrials.gov NCT05737706: MRTX1133 in KRAS G12D-mutant advanced solid tumours (phase 1)","url":"https://clinicaltrials.gov/study/NCT05737706"}],"tags":[],"related":[],"cancers":["pancreatic","metastatic-pdac","colorectal"],"sections":[],"technologies":["kras-inhibitors"],"targets":["kras"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct05737706"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Small-molecule non-covalent KRAS G12D inhibitor","mechanism":"Non-covalent binder to the switch-II pocket of KRAS G12D, inhibiting both ON and OFF states.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"mychoice-cdx","kind":"drug","name":"myChoice CDx","aka":[],"tldr":"A tumour test that measures DNA-repair scarring so that women with ovarian cancer beyond BRCA carriers can benefit from PARP inhibitors.","summary":"Approved 23 October 2019 (PMA P190014) with niraparib for homologous-recombination-deficient ovarian cancer after several lines of therapy, and in May 2020 as the companion diagnostic for olaparib plus bevacizumab first-line maintenance in HRD-positive ovarian cancer (PAOLA-1). Roughly half of high-grade serous ovarian cancers are HRD-positive by this definition, about twice the BRCA-mutated fraction. The scar is permanent, so a tumour can remain HRD-positive after it has developed resistance, one reason the test predicts benefit better in first-line than in later lines.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P190014","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P190014"}],"tags":["test"],"related":[],"cancers":["ovarian","tnbc"],"sections":[],"technologies":["hrd-testing","companion-diagnostic"],"targets":["brca","parp"],"drugs":["niraparib","olaparib"],"companies":["myriad-genetics"],"institutions":[],"pathways":[],"terms":["hrd"],"trials":["paola-1","prima"],"people":[],"bottlenecks":[],"keyPapers":["paper-telli-hrd-score-platinum-tnbc-ccr-2016","paper-loibl-geparsixto-survival-hrd-ann-oncol-2018","paper-tutt-nat-med"],"journals":[],"dependsOn":[],"notes":["What a result means: HRD-positive (by genomic instability score or BRCA mutation) means adding a PARP inhibitor after chemotherapy is likely to delay relapse; HRD-negative means the benefit is much smaller.","Triple-negative breast cancer: the 42 genomic instability threshold was derived in TNBC platinum trials (Telli 2016) and HR deficiency by this definition was 70.5% in GeparSixto (Loibl 2018), but the score did not predict carboplatin benefit in TNT (Tutt 2018) and the assay has no breast indication."],"brand":"myChoice CDx","modality":"Tumour HRD genomic instability companion diagnostic test","mechanism":"Tumour BRCA1/2 sequencing combined with a genomic instability score (GIS) built from loss of heterozygosity, telomeric allelic imbalance and large-scale state transitions; GIS ≥42 or a BRCA mutation defines HRD-positive.","approvals":[{"region":"US","year":2019,"indication":"Companion diagnostic for niraparib in HRD-positive advanced ovarian cancer"},{"region":"US","year":2020,"indication":"Companion diagnostic for olaparib plus bevacizumab first-line maintenance in HRD-positive ovarian cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nab-paclitaxel","kind":"drug","name":"Nab-paclitaxel","aka":[],"tldr":"Paclitaxel bound to albumin so it needs no solvent and no steroid premedication. Adding it to gemcitabine and cisplatin looked promising in a small study of bile duct and gallbladder cancer but did not lengthen life in the 452-patient SWOG S1815 trial.","summary":"Nab-paclitaxel is a solvent-free, albumin-bound formulation of paclitaxel that stabilises microtubules and arrests mitosis; the albumin carrier uses gp60 and SPARC-mediated transport and allows higher doses without the hypersensitivity of Cremophor. It is approved for metastatic breast cancer (United States 2005, European Union 2008), non-small-cell lung cancer (2012) and, with gemcitabine, metastatic pancreatic adenocarcinoma (MPACT, 2013). In biliary tract cancer, a 60-patient MD Anderson and Mayo phase 2 of gemcitabine, cisplatin and nab-paclitaxel (22 percent gallbladder cancer) reported a partial response rate of 45 percent, median progression-free survival of 11.8 months and median overall survival of 19.2 months, which set up SWOG S1815. That randomised phase 3 (441 analysable patients, 16 percent gallbladder cancer) found no overall survival difference between the triplet and gemcitabine plus cisplatin (14.0 versus 13.6 months, hazard ratio 0.91, p 0.41) or progression-free survival (7.5 versus 6.3 months, hazard ratio 0.89); exploratory analyses suggested greater progression-free survival benefit in gallbladder cancer (interaction p 0.01) and in locally advanced disease, but not in overall survival. It therefore has no place in routine biliary treatment and is not licensed or commissioned for it in the UK; several Chinese phase 2 trials continue to test gemcitabine plus nab-paclitaxel backbones with PD-1 antibodies in gallbladder cancer.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Protein-bound_paclitaxel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=24d10449-2936-4cd3-b7db-a7683db721e4"},{"label":"SWOG S1815, JCO 2025","url":"https://doi.org/10.1200/JCO-24-01383"},{"label":"Shroff et al., JAMA Oncology 2019: phase 2 of gemcitabine, cisplatin and nab-paclitaxel","url":"https://doi.org/10.1001/jamaoncol.2019.0270"},{"label":"NICE TA476: nab-paclitaxel with gemcitabine for untreated metastatic pancreatic cancer (6 September 2017)","url":"https://www.nice.org.uk/guidance/ta476"},{"label":"Abraxane label (openFDA): pancreatic indication and MPACT study section","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22ABRAXANE%22"},{"label":"EMA Abraxane product page: EU indications","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/abraxane"}],"tags":[],"related":[],"cancers":["gallbladder","cholangiocarcinoma","pancreatic","breast-cancer","nsclc","tnbc","tnbc-metastatic"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["swog-s1815","gap-phase2-mdacc","rugb","impassion130","neotrip","mpact","apact","canstem111p","halo-301","resolve","neolap","neonax","swog-s1505","primus-001","starpac2"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: the chemotherapy partner of atezolizumab in IMpassion130 (progression-free survival 7.5 versus 5.0 months in PD-L1 immune-cell-positive disease), one of three partners for pembrolizumab in KEYNOTE-355, and with carboplatin the neoadjuvant backbone of NeoTRIP; it needs no steroid premedication, which is one explanation offered for why IMpassion131 with conventional paclitaxel failed.","Pancreatic cancer, regulators: the Abraxane label lists first-line metastatic adenocarcinoma of the pancreas with gemcitabine on the MPACT trial (median overall survival 8.5 against 6.7 months, hazard ratio 0.72), and the EMA product information carries the same indication (marketing authorisation 11 January 2008, pancreatic indication added later). In England NICE TA476 restricts the combination to people for whom other combination chemotherapies are unsuitable; NG85 (1.9.5) repeats this, so FOLFIRINOX is the first choice for fit patients and gemcitabine plus nab-paclitaxel is the fallback."],"brand":"Abraxane","code":"ABI-007","modality":"Albumin-bound taxane (cytotoxic)","mechanism":"Microtubule stabiliser delivered as 130 nm albumin nanoparticles.","approvals":[{"region":"US","year":2005,"indication":"Metastatic breast cancer after combination chemotherapy","note":"FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=24d10449-2936-4cd3-b7db-a7683db721e4"},{"region":"EU","year":2008,"indication":"Metastatic breast cancer; later pancreatic (2013) and non-small-cell lung cancer (2015)","note":"EMA EPAR: https://www.ema.europa.eu/en/medicines/human/EPAR/abraxane"},{"region":"US","year":2013,"indication":"First-line metastatic pancreatic adenocarcinoma with gemcitabine (MPACT)","note":"FDA label (DailyMed)"},{"region":"UK","year":2017,"indication":"Untreated metastatic adenocarcinoma of the pancreas with gemcitabine; NICE TA476 (6 September 2017, replacing TA360) recommends it only if other combination chemotherapies are unsuitable and the person would otherwise have gemcitabine alone, with the patient access scheme discount","note":"https://www.nice.org.uk/guidance/ta476"}],"mechanismSteps":[],"dosing":{"route":"Intravenous","schedule":"100 mg/m² days 1 and 8 every 21 days with gemcitabine 800 mg/m² and cisplatin 25 mg/m² (S1815 schedule); 125 mg/m² weekly 3 of 4 with gemcitabine in pancreatic cancer","monitoring":"Neutrophils, peripheral neuropathy"},"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nabilone","kind":"drug","name":"Nabilone","aka":[],"tldr":"Nabilone is a synthetic cannabinoid capsule approved in 1985 for chemotherapy-induced nausea and vomiting that standard drugs cannot control; like dronabinol it is a later-line option because of its effects on mood and balance.","summary":"Nabilone was developed by Eli Lilly and approved by the FDA in December 1985 for nausea and vomiting associated with cancer chemotherapy in patients who have failed to respond adequately to conventional antiemetic treatments; it is also approved in Canada and the United Kingdom. Randomised trials showed it superior to prochlorperazine and placebo, but the serotonin and neurokinin antagonists that followed are more effective. Drowsiness, vertigo, dry mouth and euphoria are common, and it is a controlled substance. It has been studied off label for chronic pain and appetite.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Nabilone","links":[{"label":"Drugs@FDA NDA018677","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=018677"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=nabilone"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Nabilone"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["cnr1"],"drugs":["dronabinol"],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02802540"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cesamet","modality":"Oral small-molecule synthetic cannabinoid","supportive":true,"mechanism":"A synthetic THC analogue that activates CB1 receptors in the brain's vomiting centres.","approvals":[{"region":"US","year":1985,"indication":"Nausea and vomiting from cancer chemotherapy in patients who have failed conventional antiemetics"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nadofaragene-firadenovec","kind":"drug","name":"Nadofaragene firadenovec","aka":[],"tldr":"Nadofaragene firadenovec is the first gene therapy for bladder cancer: a virus that makes bladder cells produce interferon for weeks, given once every three months.","summary":"Nadofaragene firadenovec is a non-replicating adenoviral gene therapy: the vector delivers the IFN-alpha2b gene to urothelial cells, which then secrete interferon locally for weeks. It is given as a bladder instillation once every 3 months. In the phase 3 study (Boorjian 2021), 53% of patients with BCG-unresponsive carcinoma in situ had a complete response at 3 months, and 46% of responders maintained it at 12 months. The FDA approved it in December 2022, and Ferring launched commercially in 2023 to 2024 after manufacturing scale-up. Fatigue and bladder spasm were the main side effects, with grade 3 or higher treatment-related events in 4%, and it competes with nogapendekin alfa and TAR-200 in the same setting. It is the first gene therapy for bladder cancer, turning the bladder lining into its own interferon factory.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Nadofaragene_firadenovec","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Nadofaragene%20firadenovec"}],"tags":[],"related":[],"cancers":["urothelial","non-muscle-invasive-bladder-cancer"],"sections":[],"technologies":["bcg-and-intravesical-therapy"],"targets":[],"drugs":[],"companies":["ferring"],"institutions":[],"pathways":[],"terms":["bcg-unresponsive"],"trials":["nct06510374","nct06545955"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Adstiladrin","modality":"Non-replicating adenoviral gene therapy (intravesical)","mechanism":"Adenovirus vector delivers the IFN-α2b gene to urothelial cells, which secrete interferon locally.","approvals":[{"region":"US","year":2022,"indication":"BCG-unresponsive high-risk NMIBC with CIS ± papillary tumours"},{"region":"EU","year":2026,"indication":"BCG-unresponsive NMIBC with CIS; 28 May 2026 (conditional)","note":"Conditional marketing authorisation"}],"mechanismSteps":["Instilled with Syn3 excipient to enhance transduction","Urothelial cells express and secrete IFN-α2b for weeks","Interferon induces apoptosis, anti-angiogenesis, and immune activation","Repeated every 3 months in responders"],"dosing":{"route":"Intravesical","schedule":"75 mL once every 3 months"},"toxicity":[{"event":"Fatigue","anyGradePct":24},{"event":"Bladder spasm","anyGradePct":20},{"event":"Micturition urgency","anyGradePct":19},{"event":"Grade 3+ treatment-related","grade3PlusPct":4}],"access":[],"regulatoryEvents":[]},{"id":"nalirifox","kind":"drug","name":"NALIRIFOX (liposomal irinotecan + oxaliplatin + 5-FU/LV)","aka":[],"tldr":"NALIRIFOX is a version of FOLFIRINOX using a liposome-wrapped irinotecan, approved in 2024 as a first-line option for metastatic pancreatic cancer.","summary":"NALIRIFOX combines liposomal irinotecan, which gives prolonged exposure to the active metabolite SN-38, with oxaliplatin and 5-FU/leucovorin; it is a version of FOLFIRINOX built around the liposome-wrapped drug. It is a first-line option for metastatic pancreatic adenocarcinoma in patients fit enough for a multi-drug regimen. NAPOLI 3 (n=770) showed overall survival of 11.1 versus 9.2 months (HR 0.84) and PFS of 7.4 versus 5.6 months against gemcitabine plus nab-paclitaxel, and the FDA approved it in February 2024 (Ipsen). It was the first phase 3 head-to-head win between the two chemotherapy backbones, though the absolute gain is modest and critics note the absence of a modified FOLFIRINOX arm, so whether it beats conventional FOLFIRINOX is unknown. For a newcomer, it is four-drug pancreatic chemotherapy with a longer-acting irinotecan.","status":"approved","asOf":"2026-09-06","links":[{"label":"OncLive: FDA approves NALIRIFOX","url":"https://www.onclive.com/view/fda-approves-frontline-nalirifox-for-metastatic-pancreatic-adenocarcinoma"},{"label":"Onivyde label (openFDA): NALIRIFOX indication and NAPOLI 3 results table","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22ONIVYDE%22"},{"label":"NICE TA1052: pegylated liposomal irinotecan in combination for untreated metastatic pancreatic cancer, terminated appraisal (2 April 2025)","url":"https://www.nice.org.uk/guidance/terminated/ta1052"}],"tags":[],"related":[],"cancers":["pancreatic","metastatic-pdac"],"sections":[],"technologies":["cytotoxic-chemotherapy","topoisomerase-inhibitors","platinum"],"targets":[],"drugs":[],"companies":["ipsen"],"institutions":[],"pathways":[],"terms":["prodrug"],"trials":["napoli-3","rasolute-302"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["UK status: NICE's appraisal of NALIRIFOX (TA1052) was terminated on 2 April 2025 because the company made no evidence submission, and NG85 does not name it, so it is not routinely commissioned in England; NICE's other position on liposomal irinotecan is TA440 (second line, not recommended). NAPOLI 3 recruited at six UK sites (Guy's and St Thomas', the Royal Marsden, Imperial, the Christie, Nottingham City)."],"brand":"Onivyde regimen","modality":"Cytotoxic regimen","mechanism":"Liposomal irinotecan (prolonged SN-38 exposure) with oxaliplatin and 5-FU/leucovorin.","approvals":[{"region":"US","year":2024,"indication":"First-line metastatic pancreatic adenocarcinoma"},{"region":"EU","year":2016,"indication":"Onivyde 2L after gemcitabine (2016); NALIRIFOX label 2025"},{"region":"EU","year":2025,"indication":"First-line treatment of metastatic adenocarcinoma of the pancreas (Onivyde pegylated liposomal with oxaliplatin, fluorouracil and leucovorin), listed in the EMA therapeutic indications read on 24 September 2026; the year is the corpus's regional row for the label extension","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/onivyde-pegylated-liposomal"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"napabucasin","kind":"drug","name":"Napabucasin","aka":["BBI-608","BBI608"],"tldr":"Napabucasin, an oral drug marketed as a cancer stemness inhibitor, was tested in 1,134 people with untreated metastatic pancreatic cancer on top of nab-paclitaxel and gemcitabine; survival was identical and the trial was stopped for futility.","summary":"Napabucasin is bioactivated by NQO1 to generate reactive oxygen species and was developed as an inhibitor of STAT3-driven stemness. CanStem111P randomised 1,134 patients across 165 sites in 20 countries to napabucasin with nab-paclitaxel and gemcitabine or chemotherapy alone; median overall survival was 11.4 against 11.7 months (hazard ratio 1.07, 95 percent confidence interval 0.93 to 1.23), with no difference in the 382 patients whose tumours were positive for phosphorylated STAT3, and the trial was terminated for futility. Grade 3 or worse diarrhoea (11.6 against 4.9 percent) and abdominal pain (10.0 against 4.8 percent) were commoner with napabucasin. The trial remains the largest randomised dataset of nab-paclitaxel and gemcitabine and a reference control arm for first-line metastatic disease.","status":"negative","asOf":"2026-09-24","links":[{"label":"Bekaii-Saab et al., CanStem111P (eClinicalMedicine 2023)","url":"https://doi.org/10.1016/j.eclinm.2023.101897"},{"label":"ClinicalTrials.gov NCT02993731","url":"https://clinicaltrials.gov/study/NCT02993731"}],"tags":[],"related":[],"cancers":["pancreatic","metastatic-pdac","colorectal"],"sections":[],"technologies":[],"targets":["stat3"],"drugs":[],"companies":["sumitomo-pharma"],"institutions":[],"pathways":[],"terms":["pancreatic-failed-programmes"],"trials":["canstem111p"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"BBI-608","modality":"Oral small-molecule NQO1-bioactivated reactive oxygen species generator (STAT3 pathway inhibitor)","mechanism":"NQO1-dependent redox cycling producing reactive oxygen species; reduces STAT3 phosphorylation and stemness gene expression in preclinical models.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"naporafenib","kind":"drug","name":"Naporafenib","aka":[],"tldr":"Naporafenib is an experimental small-molecule drug from Erasca in phase 3 trials for melanoma, with its target not yet stated publicly.","summary":"Naporafenib (ERAS-254, LXH254) is a small-molecule drug developed by Erasca. The sponsor describes its target as RAF (pan-RAF), which OnCo does not yet have a target page for. The sponsor states: An experimental pan-RAF inhibitor blocking RAF kinase signalling in the MAPK pathway, studied in combination with the MEK inhibitor trametinib in NRAS-mutant melanoma. ClinicalTrials.gov describes the intervention as: Naporafenib (ERAS-254) is an experimental Pan-Raf inhibitor. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06346067 (A Study to Assess Naporafenib (ERAS-254) Administered With Trametinib in Patients With NRAS-mutant Melanoma (SEACRAFT-2)), in melanoma. The largest, NCT06346067, plans to enrol 78 participants (actual) with primary completion expected 2028-04. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Naporafenib","url":"https://clinicaltrials.gov/search?intr=ERAS-254"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["erasca"],"institutions":[],"pathways":[],"terms":[],"trials":["seacraft-2","nct04417621"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ERAS-254, LXH254","modality":"small molecule","mechanism":"An experimental pan-RAF inhibitor blocking RAF kinase signalling in the MAPK pathway, studied in combination with the MEK inhibitor trametinib in NRAS-mutant melanoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"navitoclax","kind":"drug","name":"Navitoclax","aka":[],"tldr":"Navitoclax is an experimental small-molecule drug from AbbVie in phase 3 trials for myeloproliferative neoplasms, with its target not yet stated publicly.","summary":"Navitoclax is a small-molecule drug developed by AbbVie. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04468984 (Study of Oral Navitoclax Tablet in Combination With Oral Ruxolitinib Tablet Versus Best Available Therapy to Assess Change in Spleen Volume in Adult Participants With Relapsed/Refractory Myelofibrosis), in myeloproliferative neoplasms. The largest, NCT04468984, plans to enrol 330 participants with primary completion was scheduled for 2025-01-29 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Navitoclax","url":"https://clinicaltrials.gov/search?intr=Navitoclax"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["myeloproliferative-neoplasms","primary-myelofibrosis"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04468984"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"navlimetostat","kind":"drug","name":"Navlimetostat","aka":[],"tldr":"Navlimetostat is an experimental small-molecule drug from Bristol-Myers Squibb in phase 3 trials for non-small-cell lung cancer and pancreatic ductal adenocarcinoma, aimed at PRMT5 (MTAP-deleted cancers).","summary":"Navlimetostat (BMS-986504, MRTX1719) is a small-molecule drug developed by Bristol-Myers Squibb. Its target is PRMT5 (MTAP-deleted cancers) (the sponsor names PRMT5 (protein arginine methyltransferase 5)). The sponsor states: Navlimetostat (BMS-986504) is a selective MTA-cooperative inhibitor of PRMT5, studied in participants with homozygous MTAP deletion. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07063745 (A Study to Compare the Combination of Navlimetostat (BMS-986504) With Pembrolizumab and Chemotherapy Versus Placebo Plus Pembrolizumab and Chemotherapy in First-line Metastatic Non-small Cell Lung Cancer Participants With Homozygous MTAP Deletion) and NCT07076121 (A Study Comparing Navlimetostat (BMS-986504) in Combination With Nab-paclitaxel and Gemcitabine Versus Placebo in Combination With Nab-paclitaxel and Gemcitabine in Participants With Untreated Metastatic Pancreatic Ductal Adenocarcinoma With Homozygous MTAP Deletion (MountainTAP-30)), in non-small-cell lung cancer and pancreatic ductal adenocarcinoma. The largest, NCT07063745, plans to enrol 590 participants with primary completion expected 2031-08-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Navlimetostat","url":"https://clinicaltrials.gov/search?intr=BMS-986504"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","pancreatic"],"sections":[],"technologies":[],"targets":["prmt5-mtap"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07063745","mountaintap-30"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"BMS-986504, MRTX1719","modality":"small molecule","mechanism":"Navlimetostat (BMS-986504) is a selective MTA-cooperative inhibitor of PRMT5, studied in participants with homozygous MTAP deletion.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"naxitamab","kind":"drug","name":"Naxitamab","aka":[],"tldr":"Naxitamab is a humanised anti-GD2 antibody from Memorial Sloan Kettering, given as an outpatient with GM-CSF for relapsed neuroblastoma in bone or marrow.","summary":"Accelerated approval November 2020 for relapsed/refractory high-risk neuroblastoma in bone or bone marrow (Study 201: ORR 50%; MSK 12-230: ORR 45%). Phase 2 in primary refractory disease with stepped-up GM-CSF: 75% CR in a 32-patient series (2025). Outpatient 30-minute infusions but severe pain and hypertension. Y-mAbs.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Naxitamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Naxitamab"}],"tags":[],"related":[],"cancers":["neuroblastoma","neuroblastoma-high-risk"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["gd2"],"drugs":[],"companies":["y-mabs"],"institutions":[],"pathways":[],"terms":[],"trials":["naxitamab-201"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Danyelza","modality":"Monoclonal antibody (anti-GD2, humanised)","mechanism":"Humanised 3F8 IgG1 anti-GD2 with higher affinity; ADCC/CDC enhanced by GM-CSF-primed granulocytes.","approvals":[{"region":"US","year":2020,"indication":"Relapsed/refractory high-risk neuroblastoma in bone or bone marrow with partial response, minor response or stable disease, with GM-CSF (accelerated)"}],"mechanismSteps":[],"dosing":{"route":"IV over 30-60 minutes (outpatient)","schedule":"3 mg/kg days 1, 3, 5 of each 4-week cycle with GM-CSF days -4 to 5; premedication with antihistamine, antipyretic, gabapentin, opioids","monitoring":"Pain, hypertension (including delayed), infusion reactions, neurotoxicity (transverse myelitis, RPLS), hypotension"},"toxicity":[{"event":"Pain","anyGradePct":100,"grade3PlusPct":65,"note":"Study 201 / 12-230 pooled"},{"event":"Hypertension","anyGradePct":42,"grade3PlusPct":28},{"event":"Infusion-related reaction","grade3PlusPct":32},{"event":"Neurotoxicity","anyGradePct":20}],"access":[],"regulatoryEvents":[{"date":"2020-11-25","type":"accelerated-approval","region":"US","note":"Accelerated approval (Study 201, 12-230) The confirmatory requirement was still open 5.8 years later, when the FDA's table was read.","indication":"In combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy."}]},{"id":"nb003","kind":"drug","name":"NB003","aka":[],"tldr":"NB003 is an experimental small-molecule drug from Ningbo Newbay Technology Development in phase 3 trials for gastrointestinal stromal tumour, with its target not yet stated publicly.","summary":"NB003 is a small-molecule drug developed by Ningbo Newbay Technology Development. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: 15 mg BID on a continuous schedule in 28-day cycles. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07379047 (Efficacy and Safety of NB003 in Patients With Advanced Gastrointestinal Stromal Tumours (GIST)), in gastrointestinal stromal tumour. The largest, NCT07379047, plans to enrol 255 participants with primary completion expected 2028-08-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of NB003","url":"https://clinicaltrials.gov/search?intr=NB003"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gist"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07379047"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nbtxr3","kind":"drug","name":"NBTXR3 (hafnium oxide nanoparticles)","aka":["Hensify","JNJ-90301900"],"tldr":"NBTXR3 is Nanobiotix's radioenhancer: a single injection of hafnium oxide nanoparticles that makes ordinary radiotherapy hit harder inside the tumour. It carries a European CE mark for soft-tissue sarcoma and is in a phase 3 trial in head and neck cancer with Johnson and Johnson.","summary":"NBTXR3 was developed by Nanobiotix in Paris. In the Act.In.Sarc phase 2/3 trial, adding the nanoparticles to preoperative radiotherapy for locally advanced soft-tissue sarcoma doubled the rate of pathological complete response, and the product received a CE mark (as Hensify) in 2019. The NANORAY-312 phase 3 trial tests it with radiotherapy, with or without cetuximab, in elderly patients with head and neck cancer who cannot have cisplatin, under a 2023 licence to Johnson and Johnson; trials with immunotherapy in lung and other cancers continue.","status":"phase-3","asOf":"2026-09-24","links":[{"label":"Act.In.Sarc (Lancet Oncology 2019)","url":"https://doi.org/10.1016/S1470-2045(19)30326-2"},{"label":"ClinicalTrials.gov: trials of NBTXR3","url":"https://clinicaltrials.gov/search?intr=NBTXR3"}],"tags":["radiation-wave3"],"related":[],"cancers":["sarcoma","head-and-neck"],"sections":[],"technologies":["radiosensitisers","imrt-igrt"],"targets":[],"drugs":[],"companies":["nanobiotix"],"institutions":[],"pathways":[],"terms":[],"trials":["act-in-sarc"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo radiation expansion","editedOn":"2026-09-16"},"code":"NBTXR3","modality":"Hafnium oxide nanoparticle radioenhancer, injected into the tumour","mechanism":"Crystalline hafnium oxide nanoparticles injected once into the tumour absorb X-rays far more strongly than tissue and release showers of electrons, multiplying the energy deposited inside the tumour without changing the dose to surrounding organs.","approvals":[{"region":"EU","year":2019,"indication":"CE mark: locally advanced soft-tissue sarcoma of the limb or trunk wall, with preoperative radiotherapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ndi-219216","kind":"drug","name":"NDI-219216","aka":[],"tldr":"NDI-219216 is an experimental small-molecule drug from Nimbus Wadjet in phase 2 trials, aimed at WRN helicase (MSI-high cancers).","summary":"NDI-219216 is a small-molecule drug developed by Nimbus Wadjet. Its target is WRN helicase (MSI-high cancers) (the sponsor names WRN helicase). The sponsor states: NDI-219216 is a highly selective, non-covalent small-molecule inhibitor of WRN helicase activity that triggers a DNA damage response and suppresses viability selectively in microsatellite instability-high (MSI-H) tumour cells, a synthetic-lethal approach. ClinicalTrials.gov describes the intervention as: NDI-219216 is a highly selective small molecule inhibitor of WRN helicase activity. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT06898450, plans to enrol 134 participants with primary completion expected 2031-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of NDI-219216","url":"https://clinicaltrials.gov/search?intr=NDI-219216"},{"label":"Sponsor pipeline page","url":"https://nimbustx.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["wrn"],"drugs":[],"companies":["nimbus-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06898450"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"NDI-219216 is a highly selective, non-covalent small-molecule inhibitor of WRN helicase activity that triggers a DNA damage response and suppresses viability selectively in microsatellite instability-high (MSI-H) tumour cells, a synthetic-lethal approach.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"necitumumab","kind":"drug","name":"Necitumumab","aka":[],"tldr":"Necitumumab (Portrazza) is an antibody added to gemcitabine and cisplatin as first treatment for squamous non-small cell lung cancer that has spread; the benefit is modest and it is little used.","summary":"Necitumumab was approved by the FDA in November 2015 in combination with gemcitabine and cisplatin for first-line metastatic squamous NSCLC, on the SQUIRE trial (1,093 patients) in which adding the antibody produced a small but statistically significant gain in overall survival, while the INSPIRE trial in non-squamous disease was stopped for lack of benefit and excess thromboembolic deaths. The EU authorised Portrazza in 2016 for EGFR-expressing squamous NSCLC. Cardiopulmonary arrest and hypomagnesaemia carry boxed warnings; rash is universal. Immunotherapy-chemotherapy combinations (KEYNOTE-407) have since made it largely obsolete, and it is listed by the NCCN only as an option in selected patients.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Necitumumab","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=necitumumab"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/necitumumab"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/portrazza"}],"tags":["nci-list"],"related":["cetuximab","gemcitabine-cisplatin"],"cancers":["nsclc"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["egfr"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03944772"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Portrazza","modality":"Monoclonal antibody (anti-EGFR, IgG1)","mechanism":"Fully human IgG1 that binds the EGFR ligand-binding domain, blocking ligand-induced activation and downstream MAPK and PI3K signalling.","approvals":[{"region":"US","year":2015,"indication":"First-line metastatic squamous NSCLC with gemcitabine and cisplatin"},{"region":"EU","year":2016,"indication":"EGFR-expressing locally advanced or metastatic squamous NSCLC with gemcitabine and cisplatin"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nedaplatin","kind":"drug","name":"Nedaplatin","aka":["254-S"],"tldr":"Nedaplatin is a Japanese platinum drug, approved there in 1995, used for head and neck, oesophageal, lung, cervical and other squamous cancers as a gentler alternative to cisplatin on the kidneys; it has never been approved in the West.","summary":"Shionogi developed nedaplatin to keep cisplatin's activity while reducing kidney damage and vomiting. Japan approved it in 1995 for head and neck, testicular, lung, oesophageal, ovarian and cervical cancers, and China followed. Randomised trials in Japan and China have shown it comparable to cisplatin with radiotherapy in nasopharyngeal and cervical cancer and in squamous non-small cell lung cancer with docetaxel, with less nephrotoxicity but more thrombocytopenia. It is not approved in the United States or Europe, where carboplatin fills the role of the better tolerated platinum.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Nedaplatin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Nedaplatin"},{"label":"ChEMBL CHEMBL2107854","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL2107854"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["head-and-neck","esophageal","nsclc","cervical"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":["cisplatin","carboplatin"],"companies":["shionogi"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aqupla","modality":"Second-generation platinum cytotoxic, intravenous","mechanism":"A platinum complex that forms DNA cross-links like cisplatin but with a glycolate leaving group that makes it less nephrotoxic and emetogenic.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"neladalkib","kind":"drug","name":"Neladalkib","aka":[],"tldr":"A fourth-generation ALK pill that works after lorlatinib and avoids the TRK-related brain side effects; under FDA priority review with a decision due 27 November 2026.","summary":"Nuvalent's ALK-selective, TRK-sparing, brain-penetrant inhibitor covering G1202R and compound mutations. ALKOVE-1: ORR 31% in heavily TKI-pretreated patients (n=253) with intracranial activity. NDA submitted April 2026 (priority review, PDUFA 27 Nov 2026). ALKAZAR phase 3 vs alectinib first line recruiting.","status":"phase-3","asOf":"2026-09-06","links":[{"label":"OncLive: priority review","url":"https://www.onclive.com/view/fda-grants-priority-review-to-neladalkib-for-tki-pretreated-alk-nsclc"}],"tags":[],"related":[],"cancers":["nsclc","alk-positive-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["alk"],"drugs":["lorlatinib","alectinib"],"companies":["nuvalent"],"institutions":[],"pathways":[],"terms":[],"trials":["alkove-1","nct06765109"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"NVL-655","modality":"Small-molecule kinase inhibitor (ALK)","mechanism":"ALK-selective TKI designed to avoid TRK inhibition (dizziness, weight gain) and cover solvent-front and compound mutations.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nelarabine","kind":"drug","name":"Nelarabine","aka":["Atriance"],"tldr":"Nelarabine (Arranon) is an infusion for T-cell acute lymphoblastic leukaemia and lymphoma that has come back after at least two other treatments; it is now also added to first-line therapy for children with T-cell leukaemia.","summary":"Nelarabine received accelerated FDA approval in October 2005 for T-cell acute lymphoblastic leukaemia and T-cell lymphoblastic lymphoma that has not responded to or has relapsed after at least two chemotherapy regimens, on phase 2 studies in adults (complete remission in about a fifth) and children; the EU authorised Atriance in 2007. The Children's Oncology Group AALL0434 trial later showed that adding nelarabine to frontline therapy improved disease-free survival in newly diagnosed T-ALL, and it is now standard in paediatric protocols. Neurotoxicity, including somnolence, peripheral neuropathy and Guillain-Barre-like demyelination, carries a boxed warning.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Nelarabine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=nelarabine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/nelarabine"},{"label":"EPAR (Atriance)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/atriance"}],"tags":["nci-list"],"related":[],"cancers":["all-leukemia","all-paediatric-high-risk","all-paediatric-relapsed"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["novartis","sandoz"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-aall0434-nelarabine-t-all-dunsmore-jco-2020"],"journals":[],"dependsOn":[],"notes":[],"brand":"Arranon","modality":"Purine nucleoside prodrug (antimetabolite)","mechanism":"Demethylated by adenosine deaminase to ara-G, which accumulates as ara-GTP preferentially in T lymphoblasts and is incorporated into DNA, blocking synthesis.","approvals":[{"region":"US","year":2005,"indication":"Relapsed or refractory T-ALL and T-LBL after at least two regimens (accelerated)"},{"region":"EU","year":2007,"indication":"T-ALL and T-LBL after at least two regimens (Atriance)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2005-10-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic leukemia following at least 2 chemotherapy regimens"},{"date":"2019-07-31","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2005 converted to traditional approval 13.8 years after it was granted.","indication":"Relapsed/refractory T-cell acute lymphoblastic leukemia and T-cell lymphoblastic leukemia following at least 2 chemotherapy regimens"}]},{"id":"nemtabrutinib","kind":"drug","name":"Nemtabrutinib","aka":[],"tldr":"Nemtabrutinib is Merck's reversible BTK blocker, active against the C481S escape mutation, being tested against ibrutinib and acalabrutinib in first-line CLL.","summary":"BELLWAVE-001 (phase 1/2): ORR 53% (including partial response with lymphocytosis) at the 65 mg dose in relapsed CLL/SLL. Phase 3 BELLWAVE-011 (vs ibrutinib or acalabrutinib, untreated CLL) and BELLWAVE-008 (vs chemoimmunotherapy) are recruiting. Also inhibits other kinases (ERK pathway), which may add activity in Richter transformation.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT06136559 (BELLWAVE-011)","url":"https://clinicaltrials.gov/study/NCT06136559"}],"tags":[],"related":[],"cancers":["cll"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["btk"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["bellwave-011","nct03162536","nct05458297","nct04728893","nct05947851","nct05624554"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"MK-1026, ARQ-531","modality":"Small-molecule non-covalent BTK inhibitor","mechanism":"Reversible ATP-competitive inhibitor of wild-type and C481S BTK, with off-target activity on ERK-pathway kinases.","approvals":[],"mechanismSteps":["Binds the BTK ATP pocket reversibly, unaffected by C481S","BCR signalling and downstream ERK signalling are blocked","Once-daily 65 mg dosing"],"dosing":{"route":"Oral","schedule":"65 mg once daily (RP2D)","monitoring":"Dysgeusia, neutropenia, hypertension, infections"},"toxicity":[{"event":"Dysgeusia","anyGradePct":21,"source":"https://www.onclive.com/view/nemtabrutinib-generates-responses-displays-manageable-safety-in-cll-sll"},{"event":"Neutropenia","grade3PlusPct":17},{"event":"Hypertension","anyGradePct":15}],"access":[],"regulatoryEvents":[]},{"id":"neovax","kind":"drug","name":"NeoVax","aka":["Personalised neoantigen peptide vaccine (Dana-Farber)"],"tldr":"NeoVax is a made-to-order vaccine: a patient's tumour is sequenced, the mutations most likely to be seen by the immune system are turned into peptides, and these are injected to train T cells against that tumour alone. Early trials in melanoma and glioblastoma showed it raises the intended immune response.","summary":"NeoVax is the personalised neoantigen vaccine platform developed at the Dana-Farber Cancer Institute and the Broad Institute. Whole-exome and RNA sequencing of the tumour identify somatic mutations, algorithms predict which mutant peptides bind the patient's HLA molecules, and up to 20 long peptides are synthesised and given subcutaneously with poly-ICLC over several weeks.\n\nThe first phase 1 trial in high-risk melanoma (Nature, 2017) showed strong CD4 and CD8 responses against the chosen neoantigens and durable freedom from recurrence in most patients, with epitope spreading after later PD-1 blockade. A phase 1 study in newly diagnosed glioblastoma (Nature, 2019) showed neoantigen-specific T cells could be generated and found in the tumour, but only in patients not taking dexamethasone, which blunted the response. The glioblastoma page names it among the personalised vaccine approaches under study.","status":"phase-1","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":["dana-farber"],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Personalised neoantigen long-peptide vaccine with poly-ICLC adjuvant","mechanism":"Up to 20 synthetic long peptides encoding mutations found only in the patient's own tumour, chosen by sequencing and HLA-binding prediction, are injected with the TLR3 agonist poly-ICLC to raise T cells against the tumour's private antigens.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"neratinib","kind":"drug","name":"Neratinib","aka":[],"tldr":"A pill taken for a year after trastuzumab to further reduce recurrence in HER2-positive, hormone-positive breast cancer, limited by severe diarrhoea.","summary":"ExteNET: extended adjuvant neratinib for 1 year after trastuzumab improved 5-year iDFS (90.2% vs 87.7%, HR 0.73), with benefit concentrated in HR+ disease that started within a year of trastuzumab. NALA (with capecitabine, metastatic): PFS HR 0.76 and fewer CNS interventions. Grade 3 diarrhoea ~40% without prophylaxis; loperamide prophylaxis or dose escalation (CONTROL) mitigates it.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Neratinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Neratinib"}],"tags":[],"related":[],"cancers":["breast-her2-positive","her2-positive-breast-brain-metastases"],"sections":[],"technologies":["kinase-inhibitors","her2-tyrosine-kinase-inhibitors"],"targets":["her2","egfr"],"drugs":[],"companies":["puma-biotechnology"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Nerlynx","modality":"Small-molecule irreversible pan-HER kinase inhibitor","mechanism":"Irreversible covalent inhibitor of HER1/HER2/HER4 kinases.","approvals":[{"region":"US","year":2017,"indication":"Extended adjuvant HER2+ early breast cancer after trastuzumab"},{"region":"US","year":2020,"indication":"HER2+ metastatic breast cancer with capecitabine after ≥2 anti-HER2 regimens"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"240 mg once daily with food for 1 year (adjuvant); with capecitabine in metastatic","modifications":"Dose escalation (120→160→240 mg) over 2 weeks; loperamide prophylaxis","monitoring":"Diarrhoea, liver enzymes"},"toxicity":[{"event":"Diarrhoea","anyGradePct":95,"grade3PlusPct":40,"note":"without prophylaxis; ~15% grade 3 with escalation"},{"event":"Nausea","anyGradePct":43},{"event":"Fatigue","anyGradePct":27}],"access":[],"regulatoryEvents":[]},{"id":"netupitant-palonosetron","kind":"drug","name":"Netupitant and palonosetron","aka":["NEPA","Fosnetupitant and palonosetron (intravenous)"],"tldr":"Akynzeo combines two anti-sickness drugs in one capsule or infusion taken once before chemotherapy, so a single dose plus dexamethasone protects against both immediate and delayed vomiting.","summary":"Netupitant/palonosetron was approved by the FDA in October 2014 for prevention of acute and delayed nausea and vomiting with initial and repeat courses of chemotherapy including highly emetogenic regimens, in combination with dexamethasone, on phase 3 trials against palonosetron alone in cisplatin and anthracycline-cyclophosphamide regimens in which complete response over five days was higher, a statistically significant difference; the intravenous fosnetupitant/palonosetron formulation followed in 2018. The EU authorised Akynzeo in 2015. It simplifies the guideline-recommended triple regimen to one antiemetic product plus a steroid. Headache, asthenia and constipation are the main adverse effects; netupitant is a moderate CYP3A4 inhibitor.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Netupitant/palonosetron","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=netupitant"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/netupitant-palonosetronhydrochloride"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/akynzeo"}],"tags":["nci-list","supportive"],"related":["palonosetron","aprepitant"],"cancers":[],"sections":[],"technologies":["antiemetic-therapy"],"targets":[],"drugs":[],"companies":["helsinn"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03367572","nct03668639","nct03386617"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Akynzeo","modality":"Fixed combination NK1 antagonist plus 5-HT3 antagonist (antiemetic)","supportive":true,"mechanism":"Netupitant, a long-acting substance P/NK1 receptor antagonist, blocks delayed emesis while palonosetron, a 5-HT3 antagonist, covers the acute phase; a single capsule or infusion with dexamethasone.","approvals":[{"region":"US","year":2014,"indication":"Acute and delayed chemotherapy-induced nausea and vomiting including highly emetogenic regimens, with dexamethasone (intravenous form 2018)"},{"region":"EU","year":2015,"indication":"Acute and delayed nausea and vomiting with highly and moderately emetogenic chemotherapy (Akynzeo)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nicotinamide","kind":"drug","name":"Nicotinamide","aka":["Niacinamide","Vitamin B3 (amide)"],"tldr":"Nicotinamide is a cheap over-the-counter form of vitamin B3. Taken twice a day it cuts the number of new skin cancers in people who have already had several, by helping skin cells repair sun damage.","summary":"Nicotinamide (niacinamide) is the amide form of vitamin B3, available without prescription. Unlike niacin it does not cause flushing. In the Australian ONTRAC trial (New England Journal of Medicine, 2015), 386 people who had had at least two non-melanoma skin cancers in the previous five years took 500 mg twice daily or placebo for a year; new basal and squamous cell carcinomas fell by about a quarter, and actinic keratoses also fell. The benefit disappears when it is stopped.\n\nIt is now recommended in guidelines as chemoprevention for high-risk patients, including organ-transplant recipients, alongside sun protection and regular skin checks. Its proposed mechanism is replenishing cellular NAD+ so that ultraviolet-damaged DNA is repaired and the local immune suppression caused by sunlight is reduced.","status":"established","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Nicotinamide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Nicotinamide"}],"tags":["subtype-drugs-wave"],"related":["acitretin"],"cancers":["basal-cell-carcinoma","cutaneous-scc"],"sections":[],"technologies":["chemoprevention"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["skin-cancer-after-organ-transplant"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Oral vitamin B3 (over the counter; cytoprotective NAD+ precursor)","mechanism":"A precursor of NAD+ that supplies energy for DNA repair after ultraviolet damage and blunts the immunosuppression sunlight causes in skin.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nilotinib","kind":"drug","name":"Nilotinib","aka":[],"tldr":"Nilotinib is a second-generation CML pill that produces deeper responses faster than imatinib, at the cost of cardiovascular and metabolic side effects.","summary":"Approved in 2007 for imatinib-resistant CML and in 2010 first line (ENESTnd: higher major molecular response than imatinib without an overall survival difference). Requires fasting dosing, QT monitoring and attention to arterial occlusive events, hyperglycaemia and lipids. Widely used as the bridge to treatment-free remission because of deep responses; generic since 2024 in some markets.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Nilotinib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=nilotinib"}],"tags":["gap-fill"],"related":["bcr-abl1-transcript"],"cancers":["cml","cml-chronic-phase"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["bcr-abl","kit"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04971226","nct05456191"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tasigna","modality":"Small-molecule BCR-ABL1 TKI (second generation)","mechanism":"ATP-competitive inhibitor of BCR-ABL1 with ~30-fold greater potency than imatinib; also KIT, PDGFR.","approvals":[{"region":"US","year":2007,"indication":"Ph+ CML, chronic or accelerated phase, resistant/intolerant to imatinib"},{"region":"US","year":2010,"indication":"Newly diagnosed Ph+ CML chronic phase"},{"region":"EU","year":2007,"indication":"Ph+ CML"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2007-10-29","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Chronic phase and accelerated phase Philadelphia chromosome positive CML in adults resistant or intolerant to prior therapy that included Gleevec (imatinib)"},{"date":"2010-06-17","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Newly diagnosed adults with with Philadelphia chromosome-positive CML in chronic phase"},{"date":"2011-01-14","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2007 converted to traditional approval 3.2 years after it was granted.","indication":"Chronic phase and accelerated phase Philadelphia chromosome positive CML in adults resistant or intolerant to prior therapy that included Gleevec (imatinib)"},{"date":"2015-01-27","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2010 converted to traditional approval 4.6 years after it was granted.","indication":"Newly diagnosed adults with with Philadelphia chromosome-positive CML in chronic phase"}]},{"id":"nilutamide","kind":"drug","name":"Nilutamide","aka":["Anandron"],"tldr":"Nilutamide (Nilandron) is an older antiandrogen tablet started on the day of surgical castration for metastatic prostate cancer; it is rarely used now because of lung and eye side effects.","summary":"Nilutamide was approved in 1996 for metastatic (stage D2) prostate cancer in combination with surgical castration, with treatment beginning on the day of or the day after orchiectomy, on a randomised trial in which the combination modestly delayed progression compared with castration alone. It has been superseded by bicalutamide and the second-generation antiandrogens. Interstitial pneumonitis (about 2% of patients, more in Japanese men) carries a boxed warning; delayed dark adaptation, alcohol intolerance and hepatitis are also characteristic.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Nilutamide","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=nilutamide"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/nilutamide"}],"tags":["nci-list","generic"],"related":["flutamide","bicalutamide"],"cancers":["prostate"],"sections":[],"technologies":["androgen-deprivation"],"targets":["androgen-receptor"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct03678025","nct00003653","nct00002633"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Nilandron","modality":"Non-steroidal antiandrogen (first generation)","mechanism":"Competitive androgen receptor antagonist without oestrogenic, progestational or glucocorticoid activity.","approvals":[{"region":"US","year":1996,"indication":"Metastatic (stage D2) prostate cancer in combination with surgical castration"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nimorazole","kind":"drug","name":"Nimorazole","aka":["Naxogin"],"tldr":"Nimorazole is the one hypoxic radiosensitiser in routine use: given with radiotherapy for head and neck cancer in Denmark since the DAHANCA 5 trial improved control, and tested across Europe in patients whose tumours carry a hypoxia gene signature.","summary":"Nimorazole was developed as an anti-infective and repurposed as a hypoxic cell radiosensitiser because it is far better tolerated than the earlier nitroimidazoles misonidazole and etanidazole. DAHANCA 5 showed higher locoregional control with nimorazole than placebo during radiotherapy for supraglottic and pharyngeal cancer, and Danish practice has included it ever since; a later analysis found the benefit confined to tumours with a 15-gene hypoxia signature. The EORTC 1219 and DAHANCA 29 trials tested it with accelerated chemoradiation in the wider European population and in hypoxia-selected patients. It has no oncology marketing authorisation outside Denmark and is used under national guidance.","status":"established","asOf":"2026-09-16","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Nimorazole"},{"label":"ClinicalTrials.gov: trials of nimorazole","url":"https://clinicaltrials.gov/search?intr=nimorazole"}],"tags":["radiation-wave3"],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":["radiosensitisers","tumour-hypoxia-modification"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["dahanca-5"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo radiation expansion","editedOn":"2026-09-16"},"modality":"Hypoxic cell radiosensitiser (nitroimidazole small molecule)","mechanism":"A nitroimidazole that mimics oxygen in hypoxic tumour cells, fixing radiation-induced DNA damage that would otherwise be repaired, so the oxygen-starved cells that normally survive radiotherapy are killed.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nimotuzumab","kind":"drug","name":"Nimotuzumab","aka":["h-R3"],"tldr":"Nimotuzumab is a Cuban-developed antibody against EGFR approved in India, China, Cuba and other countries for head and neck cancer, nasopharyngeal cancer and brain tumours, usually with radiotherapy; it causes far less rash than cetuximab but has never been approved in the United States or Europe.","summary":"Nimotuzumab was developed at Cuba's Center of Molecular Immunology and is marketed through partners in India (BIOMAb EGFR), China (Taixinsheng), Cuba and more than 25 other countries. Approvals cover squamous cell carcinoma of the head and neck with radiotherapy or chemoradiotherapy, nasopharyngeal carcinoma, high-grade glioma in children and adults, and in some countries oesophageal and pancreatic cancer. Its intermediate affinity means bivalent binding only on cells with dense EGFR, which gives it a milder toxicity profile than cetuximab. Randomised trials from India and China show improved locoregional control and survival with chemoradiotherapy in head and neck cancer, but the drug has not been submitted to the FDA or EMA.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Nimotuzumab","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Nimotuzumab"},{"label":"ChEMBL CHEMBL2108574","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL2108574"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["head-and-neck","glioblastoma","esophageal","pancreatic","kras-wild-type-pdac"],"sections":[],"technologies":[],"targets":["egfr"],"drugs":["cetuximab"],"companies":["cimab","biocon","eurofarma"],"institutions":[],"pathways":[],"terms":[],"trials":["notable-trial"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"BIOMAb EGFR / TheraCIM / CIMAher","modality":"Humanised anti-EGFR monoclonal antibody, intravenous","mechanism":"Binds the extracellular domain of EGFR with moderate affinity, so it accumulates mainly on cells with high receptor density and spares normal skin, which explains its mild rash.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nintedanib","kind":"drug","name":"Nintedanib","aka":[],"tldr":"Nintedanib is a triple angiokinase inhibitor pill (VEGFR, FGFR, PDGFR) approved for pulmonary fibrosis and, in the EU, for second-line lung adenocarcinoma with docetaxel. In mesothelioma the phase 2 part of LUME-Meso suggested slower progression in epithelioid disease, but the phase 3 part showed none, a much-cited warning about small randomised phase 2 signals.","summary":"Nintedanib is a triple angiokinase inhibitor of VEGFR1-3, FGFR1-3 and PDGFR alpha and beta, designed to cut off tumour blood supply and stromal signalling. It is approved for idiopathic pulmonary fibrosis and, in the EU, for second-line lung adenocarcinoma with docetaxel. In mesothelioma, the LUME-Meso phase 2 suggested a PFS benefit in epithelioid disease, but the phase 3 part of the same trial showed none, with PFS 6.8 versus 7.0 months (HR 1.01) and no OS benefit. The programme is a frequently cited lesson in how unreliable small randomised phase 2 PFS signals can be in this disease. Boehringer Ingelheim developed it, and its oncology role is now limited to the EU lung indication. A plausible mechanism and an encouraging early trial are not enough until a large trial confirms them.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Nintedanib","links":[{"label":"EMA: Vargatef (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/vargatef"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Nintedanib"},{"label":"NICE TA347: nintedanib for previously treated locally advanced, metastatic or locally recurrent non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta347"}],"tags":["failure","lesson:phase-2-pfs-signal"],"related":[],"cancers":["mesothelioma","lung-cancer","lung-adenocarcinoma"],"sections":[],"technologies":["antiangiogenic","kinase-inhibitors"],"targets":["vegf","fgfr2"],"drugs":[],"companies":["boehringer-ingelheim"],"institutions":[],"pathways":[],"terms":[],"trials":["lume-meso"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ofev (fibrosis); Vargatef (NSCLC, EU)","modality":"Small-molecule kinase inhibitor (VEGFR/FGFR/PDGFR)","mechanism":"Triple angiokinase inhibitor of VEGFR1-3, FGFR1-3, PDGFRα/β.","approvals":[{"region":"EU","year":2014,"indication":"Adenocarcinoma NSCLC after first-line chemotherapy, with docetaxel"},{"region":"US","year":2014,"indication":"Idiopathic pulmonary fibrosis (non-oncology)"},{"region":"England (NICE)","year":2015,"indication":"Locally advanced, metastatic or locally recurrent non-small-cell lung cancer of adenocarcinoma histology progressing after first-line chemotherapy, with docetaxel","note":"TA347, published 22 July 2015, with the discount agreed in the patient access scheme."}],"mechanismSteps":["Blocks VEGFR, FGFR, and PDGFR signalling in endothelial and stromal cells","Reduces angiogenesis and fibroblast activation","In mesothelioma, no meaningful effect on tumour progression in phase 3"],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"niraparib","kind":"drug","name":"Niraparib","aka":["Niraparib Tosylate Monohydrate and Abiraterone Acetate","Akeega"],"tldr":"Niraparib is a PARP inhibitor approved as maintenance for ovarian cancer regardless of BRCA status, and in prostate cancer with abiraterone.","summary":"Niraparib is a PARP1/2 inhibitor that traps PARP on damaged DNA, killing tumour cells that cannot repair double-strand breaks through homologous recombination. It was approved in 2017 for maintenance in recurrent ovarian cancer (NOVA) and in 2020 for first-line maintenance regardless of BRCA status (PRIMA) (label later restricted to HRD-positive in some settings). Combined with abiraterone as Akeega, it is approved for BRCA-mutant metastatic castration-resistant prostate cancer (MAGNITUDE, 2023). Dosing is individualised at 200 or 300 mg daily by weight and platelet count, for up to 3 years in first-line maintenance; thrombocytopenia (66%, 38% grade 3 or higher) and anaemia are the main toxicities, and MDS or AML occurs in 3.3%. Whether HRD-negative patients gain survival remains contested. For a newcomer: a PARP pill that stretched maintenance therapy beyond BRCA carriers.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Niraparib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Niraparib"},{"label":"NICE TA1032: niraparib with abiraterone acetate and prednisone for untreated hormone-relapsed metastatic prostate cancer, terminated appraisal","url":"https://www.nice.org.uk/guidance/terminated/ta1032"}],"tags":[],"related":["brca-germline","hrd-positive"],"cancers":["ovarian","prostate","high-grade-serous-ovarian-cancer","prostate-mcrpc","platinum-sensitive-ovarian-cancer"],"sections":[],"technologies":["parp-inhibitor"],"targets":["parp"],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":["prostate-hrr-eligibility","prostate-uk-drug-approvals"],"trials":["nct06915025","nct05335993","nct04915755","nct03431350","nct06682780","nct04497844","nct03602859","nct04641247","nct06077877"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Niraparib with abiraterone acetate and prednisone has no NICE recommendation for prostate cancer in England. TA1032, terminated on 22 January 2025, records that NICE was unable to make a recommendation because Johnson and Johnson Innovative Medicine did not provide an evidence submission; the decision will be reviewed if the company submits."],"brand":"Zejula","modality":"Small-molecule PARP inhibitor","mechanism":"PARP1/2 inhibitor.","approvals":[{"region":"US","year":2017,"indication":"Recurrent ovarian cancer maintenance"},{"region":"US","year":2020,"indication":"First-line ovarian maintenance"}],"mechanismSteps":["Niraparib binds and traps PARP1/2 on damaged DNA","Single-strand break repair fails; forks collapse","HR-deficient cells accumulate lethal double-strand breaks","Tumour cells die; marrow suppression is the main collateral effect"],"dosing":{"route":"Oral","schedule":"200 mg once daily if <77 kg or platelets <150,000/µL; otherwise 300 mg once daily; 3 years first-line maintenance","modifications":"Reduce for thrombocytopenia; hold for hypertension crisis","monitoring":"Blood counts weekly for the first month, monthly for 11 months; blood pressure; MDS/AML awareness","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b7f675e2-159c-490c-b6f4-3f16d9492b7d"},"toxicity":[{"event":"Thrombocytopenia","anyGradePct":66,"grade3PlusPct":38,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b7f675e2-159c-490c-b6f4-3f16d9492b7d","note":"PRIMA"},{"event":"Anaemia","anyGradePct":65,"grade3PlusPct":31,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b7f675e2-159c-490c-b6f4-3f16d9492b7d","note":"PRIMA"},{"event":"Nausea","anyGradePct":62,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b7f675e2-159c-490c-b6f4-3f16d9492b7d","note":"PRIMA"},{"event":"Fatigue","anyGradePct":52,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b7f675e2-159c-490c-b6f4-3f16d9492b7d","note":"PRIMA"},{"event":"Neutropenia","grade3PlusPct":17,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b7f675e2-159c-490c-b6f4-3f16d9492b7d","note":"PRIMA"},{"event":"MDS/AML","anyGradePct":3.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=b7f675e2-159c-490c-b6f4-3f16d9492b7d","note":"vs 2.4% placebo, PRIMA long-term"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for ovarian maintenance (TA528, TA673) and with abiraterone in BRCA-mutant prostate cancer (Akeega)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-03-27","type":"approval","region":"US","note":"Maintenance in recurrent ovarian cancer regardless of BRCA (NOVA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-04-29","type":"approval","region":"US","note":"First-line ovarian maintenance, all-comers (PRIMA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-11","type":"label-change","region":"US","note":"Second-line maintenance restricted to gBRCA after OS data","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-08-11","type":"approval","region":"US","note":"Niraparib + abiraterone (Akeega) for BRCA-mutated mCRPC (MAGNITUDE)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"nirogacestat","kind":"drug","name":"Nirogacestat","aka":[],"tldr":"Nirogacestat is the first approved medicine for desmoid tumours, locally invasive growths that do not spread but can be crippling; it works by blocking Notch signalling.","summary":"Nirogacestat is an oral gamma-secretase inhibitor that prevents cleavage of Notch receptors and release of the Notch intracellular domain; desmoid tumours, driven by CTNNB1 or APC mutations, depend on crosstalk between Notch and Wnt signalling. In the phase 3 DeFi trial it reduced progression with a PFS HR of 0.29 versus placebo, produced objective responses in 41% versus 8%, and improved patient-reported pain and physical function (NEJM 2023). The FDA approved it on 27 November 2023 as the first systemic therapy for progressing desmoid tumours, and the EU followed in 2025. Diarrhoea is common, and ovarian toxicity affected 75% of women of childbearing potential, largely reversing on stopping. SpringWorks developed it and was acquired by Merck KGaA. Desmoid tumours never spread but can be crippling, and this is the first medicine proven to shrink them.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Nirogacestat","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Nirogacestat"}],"tags":[],"related":[],"cancers":["sarcoma","desmoid-tumour"],"sections":[],"technologies":["kinase-inhibitors","gamma-secretase-inhibitors"],"targets":[],"drugs":[],"companies":["springworks"],"institutions":[],"pathways":["wnt"],"terms":[],"trials":["defi","nct07084896","nct07170644","nct07150091","nct07150104"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ogsiveo","modality":"Small-molecule gamma-secretase inhibitor","mechanism":"Inhibits gamma-secretase, preventing Notch receptor cleavage and NICD release; desmoid tumours (CTNNB1 or APC mutant) depend on Notch/Wnt crosstalk.","approvals":[{"region":"US","year":2023,"indication":"Progressing desmoid tumours requiring systemic treatment"},{"region":"EU","year":2025,"indication":"Progressing desmoid tumours"}],"mechanismSteps":["Nirogacestat binds the gamma-secretase complex","Notch receptors are not cleaved after ligand binding","No Notch intracellular domain reaches the nucleus","Desmoid fibroblast proliferation and matrix production fall","Tumours shrink slowly over months"],"dosing":{"route":"Oral","schedule":"150 mg twice daily continuously","monitoring":"Diarrhoea, ovarian function/hormones, phosphate, liver enzymes, skin, non-melanoma skin cancer"},"toxicity":[{"event":"Diarrhoea","anyGradePct":84,"grade3PlusPct":16,"note":"DeFi"},{"event":"Ovarian toxicity","anyGradePct":75,"note":"women of childbearing potential; mostly resolved on stopping"},{"event":"Rash","anyGradePct":68},{"event":"Hypophosphataemia","anyGradePct":65}],"access":[],"regulatoryEvents":[{"date":"2023-11-27","type":"approval","region":"US","note":"First systemic therapy for desmoid tumours (DeFi)","source":"https://www.onclive.com/view/fda-approves-nirogacestat-for-desmoid-tumors"},{"date":"2025","type":"approval","region":"EU","note":"European Commission approval","source":"https://www.onclive.com/view/nirogacestat-wins-eu-approval-for-progressing-desmoid-tumors"}]},{"id":"nivolumab","kind":"drug","name":"Nivolumab","aka":[],"tldr":"Nivolumab was the second PD-1 blocker and is often combined with ipilimumab. Long-term data show about half of advanced melanoma patients alive at 10 years on the combination.","summary":"Approved across melanoma (CheckMate 067 combination), NSCLC, RCC, Hodgkin, head and neck, urothelial, MSI-H CRC (CheckMate 8HW first-line), gastric/oesophageal, HCC, mesothelioma, and perioperative NSCLC. March 2026: first-line advanced classical Hodgkin lymphoma with AVD (SWOG S1826) for ages 12+. Partner of relatlimab (Opdualag) and of the oncolytic virus Tudriqev.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Nivolumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Nivolumab"},{"label":"NICE TA1065: nivolumab plus ipilimumab for untreated MSI-high or dMMR colorectal cancer","url":"https://www.nice.org.uk/guidance/ta1065"},{"label":"NICE TA716: nivolumab with ipilimumab for previously treated MSI-high or dMMR colorectal cancer","url":"https://www.nice.org.uk/guidance/ta716"},{"label":"FDA oncology approval notifications","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications"},{"label":"NICE TA655: nivolumab for advanced squamous non-small-cell lung cancer after chemotherapy","url":"https://www.nice.org.uk/guidance/ta655"},{"label":"NICE TA713: nivolumab for advanced non-squamous non-small-cell lung cancer after chemotherapy","url":"https://www.nice.org.uk/guidance/ta713"},{"label":"NICE TA724: nivolumab with ipilimumab and chemotherapy for untreated metastatic non-small-cell lung cancer (not recommended)","url":"https://www.nice.org.uk/guidance/ta724"},{"label":"NICE TA876: nivolumab with chemotherapy for neoadjuvant treatment of resectable non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta876"},{"label":"NICE TA1127: nivolumab with chemotherapy neoadjuvant then alone adjuvant for resectable non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1127"},{"label":"Ansell et al., N Engl J Med 2015: nivolumab in relapsed or refractory Hodgkin lymphoma (23 patients)","url":"https://doi.org/10.1056/NEJMoa1411087"},{"label":"Green et al., Blood 2010: selective 9p24.1 amplification and PD-1 ligand induction through JAK2 in Hodgkin lymphoma and mediastinal large B-cell lymphoma","url":"https://doi.org/10.1182/blood-2010-05-282780"},{"label":"Roemer et al., J Clin Oncol 2018: MHC class II and PD-L1 expression predict outcome after PD-1 blockade in classical Hodgkin lymphoma (CheckMate 205)","url":"https://doi.org/10.1200/JCO.2017.77.3994"}],"tags":[],"related":["pd-l1-cps","pd-l1-tc-score","dmmr-ihc","msi-high"],"cancers":["melanoma","nsclc","rcc","hodgkin-lymphoma","colorectal","gastric","gastric-pdl1-high","hcc","hcc-advanced","mesothelioma","urothelial","head-and-neck","recurrent-metastatic-hnscc","msi-high-colorectal","pdl1-high-nsclc","resectable-nsclc","advanced-melanoma","stage-iii-melanoma","stage-ii-melanoma","mucosal-melanoma","acral-melanoma","lung-cancer"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["checkmate-067","nct03873402","nct06054555","nct06581406","nct04810078","nct06246916","nct07541170","nct04674683","nct06640530","nct05317858","nct05625399","nct06561386","nct04099251","nct03383458","nct06097728","nct07195695","nct05987332","nct07527858","nct05180799","nct07219459","nct06946797","nct05423262","nct03228667","nct06101134","nct04895709","nct05601752","nct06948448","nct06463665","nct05919264","nct07432295","nct06362369","nct03907852","nct07246863","nct04180371","nct03647163","nct03767348","nct07563738","nct03259867","nct05655312","nct06047379","nct05257590","nct05888831","nct06022861","nct07720284","nct03899155","nct04725474","nct04025879","nct03686124","nct03036098","nct07492680","nct04078295","nct05934331","nct05584670","nct07221149","nct03505320","nct07431281","nct05163041","nct03865082","nct05533697","nct07405164","nct06697197","nct06618287","nct07221734","nct07276373","nct06256328","nct03897543","checkmate-017","checkmate-057","checkmate-026","checkmate-227","checkmate-9la","checkmate-816"],"people":["tasuku-honjo"],"bottlenecks":[],"keyPapers":["paper-niche-2-nejm-2024","paper-checkmate-017-nejm-2015","paper-overman-checkmate-142-nivolumab-dmmr-colorectal-lancet-oncol-2017"],"journals":[],"dependsOn":[],"notes":["Colorectal cancer biomarkers: mismatch repair deficiency or MSI-high. CheckMate 142 gave 23 objective responses in 74 previously treated patients (31.1%) with disease control for 12 weeks or longer in 69% (Overman 2017); the ipilimumab combination that followed is now first-line under CheckMate 8HW.","Lymphoma biology: Hodgkin lymphoma is the clearest genetically explained checkpoint indication in oncology. An objective response was reported in 20 of 23 heavily pre-treated patients, 87%, most of whom had already failed autologous transplant and brentuximab vedotin (Ansell 2015), and the reason is the 9p24.1 amplicon, which raises both PD-1 ligands by gene dose and by JAK2-driven transcription (Green 2010). Within the responsive group, MHC class II rather than class I expression predicted complete remission (Roemer 2018)."],"brand":"Opdivo / Opdivo Qvantig (SC)","modality":"Monoclonal antibody (anti-PD-1)","mechanism":"Fully human IgG4 anti-PD-1.","approvals":[{"region":"US","year":2014,"indication":"Melanoma"},{"region":"US","year":2026,"indication":"Untreated advanced classical Hodgkin lymphoma with AVD, age ≥12"},{"region":"US","year":2025,"indication":"Unresectable or metastatic MSI-high or dMMR colorectal cancer, with ipilimumab","note":"Approved 8 April 2025 on CheckMate 8HW."},{"region":"England (NICE)","year":2025,"indication":"Untreated unresectable or metastatic MSI-high or dMMR colorectal cancer, with ipilimumab","note":"TA1065, published 28 May 2025, within the marketing authorisation and subject to the commercial arrangements; TA716 (July 2021) covers previously treated disease."},{"region":"England (NICE)","year":2020,"indication":"Locally advanced or metastatic squamous non-small-cell lung cancer after chemotherapy","note":"TA655, published 21 October 2020, stopped at 2 years of uninterrupted treatment and only for patients who have not had a PD-1 or PD-L1 inhibitor before (CheckMate 017)."},{"region":"England (NICE)","year":2021,"indication":"Locally advanced or metastatic PD-L1-positive non-squamous non-small-cell lung cancer after chemotherapy","note":"TA713, published 7 July 2021, stopped at 2 years and only for patients who have not had a PD-1 or PD-L1 inhibitor before (CheckMate 057)."},{"region":"England (NICE)","year":2021,"indication":"Untreated metastatic non-small-cell lung cancer without an EGFR or ALK alteration, with ipilimumab and two cycles of platinum doublet chemotherapy: not recommended","note":"TA724, published 8 September 2021, does not recommend the CheckMate 9LA regimen, so it is not routinely funded in England."},{"region":"England (NICE)","year":2023,"indication":"Neoadjuvant treatment of resectable non-small-cell lung cancer (4 cm or more, or node positive), with chemotherapy","note":"TA876, published 22 March 2023 (CheckMate 816)."},{"region":"England (NICE)","year":2026,"indication":"Resectable non-small-cell lung cancer at high risk of recurrence without an EGFR mutation or ALK rearrangement: neoadjuvant with platinum chemotherapy then adjuvant alone","note":"TA1127, published 4 February 2026 (CheckMate 77T); NICE asks that the least expensive of nivolumab, pembrolizumab and durvalumab is used."}],"mechanismSteps":["Antibody binds PD-1 on T cells","PD-1/PD-L1 engagement between T cell and tumour is blocked","Exhausted tumour-reactive T cells regain effector function","Interferon-γ and cytotoxic granules are released at the tumour","Tumour cells are killed; memory T cells persist"],"dosing":{"route":"IV infusion over 30 min (subcutaneous Opdivo Qvantig available)","schedule":"240 mg every 2 weeks or 480 mg every 4 weeks; with ipilimumab in melanoma: 1 mg/kg + ipilimumab 3 mg/kg every 3 weeks ×4 then maintenance","modifications":"Hold for grade 2 immune-mediated events; permanently discontinue for grade 4","monitoring":"Thyroid, liver, renal function, glucose; symptoms of colitis and pneumonitis","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394"},"toxicity":[{"event":"Immune-mediated rash","anyGradePct":9,"grade3PlusPct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"},{"event":"Hypothyroidism","anyGradePct":8,"grade3PlusPct":0.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"},{"event":"Pneumonitis","anyGradePct":3.1,"grade3PlusPct":1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"},{"event":"Colitis","anyGradePct":2.9,"grade3PlusPct":1.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"},{"event":"Hepatitis","anyGradePct":1.8,"grade3PlusPct":1.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"},{"event":"Nephritis","anyGradePct":1.2,"grade3PlusPct":0.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"},{"event":"Adrenal insufficiency","anyGradePct":1,"grade3PlusPct":0.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"},{"event":"Colitis with ipilimumab (1+3 mg/kg)","anyGradePct":25,"grade3PlusPct":14.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"},{"event":"Hepatitis with ipilimumab","anyGradePct":15,"grade3PlusPct":13.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f570b9c4-6846-4de2-abfa-4d0a4ae4e394","note":"Monotherapy pooled unless stated"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.bmsaccesssupport.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended across melanoma, NSCLC, RCC, Hodgkin, HNSCC, gastric/oesophageal, urothelial, MSI-H CRC indications","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2014-07-04","type":"approval","region":"JP","note":"First PD-1 inhibitor approved worldwide (melanoma, Japan)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2014-12-22","type":"accelerated-approval","region":"US","note":"Advanced melanoma after ipilimumab (accelerated)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Unresectable or metastatic melanoma and progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor"},{"date":"2015-03-04","type":"approval","region":"US","note":"Squamous NSCLC after platinum: first PD-1 in lung cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2015-09-30","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with ipilimumab for BRAF V600 wild-type unresectable or metastatic melanoma"},{"date":"2015-10-01","type":"approval","region":"US","note":"With ipilimumab, BRAF wild-type melanoma (CheckMate 067)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2016-01-23","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"1) In combination with ipilimumab for unresectable or metastatic melanoma to remove the restriction for treatment of only patients with BRAF wild-type melanoma; 2) As a single agent for BRAF V600 mutation positive unresectable or metastatic melanoma to remove the restriction that such patients should have disease progression following ipilimumab and a BRAF inhibitor"},{"date":"2016-05-17","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"For the treatment of classical Hodgkin Lymphoma that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and post-transplantation brentuximab vedotin"},{"date":"2017-02-02","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Locally advanced or metastatic urothelial carcinoma that: • progressed during or following platinum-containing chemotherapy • progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy"},{"date":"2017-04-25","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with classical Hodgkin lymphoma that has relapsed or progressed after: Autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin, Or 3 or more lines of systemic therapy that includes autologous HSCT"},{"date":"2017-07-31","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"For the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan"},{"date":"2017-09-22","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Hepatocellular carcinoma previously treated with sorafenib"},{"date":"2018-04-16","type":"approval","region":"US","note":"With ipilimumab, intermediate/poor-risk RCC (CheckMate 214)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2018-07-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with ipilimumab, is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan"},{"date":"2018-08-16","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Metastatic SCLC with progression after platinum-based chemotherapy and at least one other line of therapy"},{"date":"2019-03-07","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2014 converted to traditional approval 4.2 years after it was granted.","indication":"Unresectable or metastatic melanoma and progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor"},{"date":"2019-03-07","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2015 converted to traditional approval 3.4 years after it was granted.","indication":"In combination with ipilimumab for BRAF V600 wild-type unresectable or metastatic melanoma"},{"date":"2019-03-07","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2016 converted to traditional approval 3.1 years after it was granted.","indication":"1) In combination with ipilimumab for unresectable or metastatic melanoma to remove the restriction for treatment of only patients with BRAF wild-type melanoma; 2) As a single agent for BRAF V600 mutation positive unresectable or metastatic melanoma to remove the restriction that such patients should have disease progression following ipilimumab and a BRAF inhibitor"},{"date":"2020-03-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with ipilimumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib"},{"date":"2020-12-29","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 2.4 years after its accelerated approval.","indication":"Metastatic SCLC with progression after platinum-based chemotherapy and at least one other line of therapy"},{"date":"2021-07-23","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.8 years after its accelerated approval.","indication":"Hepatocellular carcinoma previously treated with sorafenib"},{"date":"2021-08-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 4.5 years after it was granted.","indication":"Locally advanced or metastatic urothelial carcinoma that: • progressed during or following platinum-containing chemotherapy • progressed within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy"},{"date":"2022-03-04","type":"approval","region":"US","note":"Neoadjuvant NSCLC with chemotherapy (CheckMate 816)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-12-27","type":"accelerated-approval","region":"US","note":"Subcutaneous nivolumab (Opdivo Qvantig)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Formulation: As monotherapy or as monotherapy following treatment with intravenous nivolumab and ipilimumab combination therapy for the treatment of adult patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan ."},{"date":"2024-12-27","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nivolumab-and-hyaluronidase-nvhy-subcutaneous-injection","indication":"Formulation: Treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib and following treatment with intravenous nivolumab and ipilimumab ."},{"date":"2025-04-08","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 7.7 years after it was granted.","indication":"For the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan"},{"date":"2025-04-08","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 6.7 years after it was granted.","indication":"In combination with ipilimumab, is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or DNA mismatch repair deficient (dMMR), metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan"},{"date":"2025-04-11","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 5.1 years after it was granted.","indication":"In combination with ipilimumab, for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib"},{"date":"2025-10-27","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 0.8 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nivolumab-and-hyaluronidase-nvhy-subcutaneous-injection","indication":"Formulation: As monotherapy or as monotherapy following treatment with intravenous nivolumab and ipilimumab combination therapy for the treatment of adult patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan ."},{"date":"2025-10-27","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 0.8 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-nivolumab-and-hyaluronidase-nvhy-subcutaneous-injection","indication":"Formulation: Treatment of adult patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib and following treatment with intravenous nivolumab and ipilimumab ."},{"date":"2026-03","type":"approval","region":"US","note":"First-line advanced classical Hodgkin lymphoma with AVD, age ≥12 (SWOG S1826)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-03-20","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2016 converted to traditional approval 9.8 years after it was granted.","indication":"For the treatment of classical Hodgkin Lymphoma that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and post-transplantation brentuximab vedotin"},{"date":"2026-03-20","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 8.9 years after it was granted.","indication":"Treatment of adult patients with classical Hodgkin lymphoma that has relapsed or progressed after: Autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin, Or 3 or more lines of systemic therapy that includes autologous HSCT"},{"date":"2026-08-06","type":"approval","region":"US","note":"Accelerated approval of vusolimogene oderparepvec in combination with nivolumab, unresectable advanced cutaneous melanoma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-vusolimogene-oderparepvec-wtpg-combination-nivolumab-melanoma"}]},{"id":"nogapendekin-alfa","kind":"drug","name":"Nogapendekin alfa inbakicept","aka":["ALT-803","NAI","nogapendekin-alfa-inbakicept"],"tldr":"Nogapendekin alfa inbakicept is an engineered version of the immune-boosting protein IL-15, given with BCG into the bladder, approved in 2024 for early bladder cancer that BCG alone no longer controls.","summary":"Nogapendekin alfa inbakicept is an IL-15 superagonist, an IL-15/IL-15R alpha-Fc fusion that expands natural killer cells and memory CD8 T cells without preferentially stimulating regulatory T cells. It is instilled into the bladder together with BCG, weekly for 6 weeks and then as maintenance for up to 37 months. In QUILT 3.032 the combination produced a complete response in 71% of patients with BCG-unresponsive carcinoma in situ, with long bladder preservation in responders. The FDA approved it on 22 April 2024 after a 2023 complete response letter over manufacturing; side effects are mostly local, with grade 3 or higher treatment-related events in only 3%. ImmunityBio is expanding into BCG-naive disease and papillary-only cohorts. The idea is to make BCG work again by giving the immune cells it recruits a stronger signal.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Nogapendekin_alfa_inbakicept","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Nogapendekin%20alfa%20inbakicept"},{"label":"QUILT-3.032 (NEJM Evidence 2023)","url":"https://doi.org/10.1056/EVIDoa2200167"}],"tags":[],"related":[],"cancers":["urothelial","non-muscle-invasive-bladder-cancer"],"sections":[],"technologies":["cytokine-therapy","bcg-and-intravesical-therapy"],"targets":[],"drugs":[],"companies":["immunitybio"],"institutions":[],"pathways":[],"terms":["bcg-unresponsive"],"trials":["quilt-3-032","nct06710288","nct03228667","nct06061809","nct06745908","nct04390399"],"people":[],"bottlenecks":[],"keyPapers":["paper-chamie-nejm-evid"],"journals":[],"dependsOn":[],"notes":[],"brand":"Anktiva","code":"N-803","modality":"IL-15 superagonist (intravesical, with BCG)","mechanism":"IL-15/IL-15Rα-Fc fusion that expands NK and memory CD8 T cells without preferentially stimulating regulatory T cells.","approvals":[{"region":"US","year":2024,"indication":"BCG-unresponsive NMIBC with CIS ± papillary tumours, with BCG"},{"region":"EU","year":2026,"indication":"BCG-unresponsive NMIBC with CIS, with BCG; 16 Feb 2026 (conditional)","note":"Conditional marketing authorisation"}],"mechanismSteps":["Co-instilled with BCG","IL-15 receptor signalling expands NK and CD8 T cells in the bladder wall","BCG provides antigen and danger signals; N-803 amplifies the effector response","Durable clearance of CIS with bladder preservation"],"dosing":{"route":"Intravesical with BCG","schedule":"400 µg with BCG weekly ×6, re-induction if needed, then maintenance at months 4, 7, 10, 13, 19 (and up to 37)"},"toxicity":[{"event":"Dysuria","anyGradePct":22},{"event":"Haematuria","anyGradePct":17},{"event":"Urinary frequency","anyGradePct":15},{"event":"Grade 3+ treatment-related","grade3PlusPct":3}],"access":[],"regulatoryEvents":[]},{"id":"gardasil-9","kind":"drug","name":"Nonavalent HPV vaccine","aka":[],"tldr":"A vaccine against nine HPV types that prevents about 90% of cervical cancers, and now works with a single dose.","summary":"Approved 2014 (US), covering HPV 6, 11, 16, 18, 31, 33, 45, 52, 58. Population data from Sweden (NEJM 2020) and Scotland (JNCI 2024) show ~90% and near-100% reductions in invasive cervical cancer in women vaccinated at 12-13. WHO recommends one- or two-dose schedules since 2022 (KEN SHE, IARC India); FDA label remains two doses under 15 and three doses at 15-45. Also prevents anal, oropharyngeal, vulvar, vaginal, and penile cancers and genital warts. Global coverage of girls with at least one dose was ~27% in 2023, the binding constraint on elimination.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Gardasil","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Nonavalent%20HPV%20vaccine"}],"tags":[],"related":[],"cancers":["cervical","head-and-neck","anal-hsil-precursor","vaginal-squamous-cell-carcinoma"],"sections":[],"technologies":["hpv-vaccine"],"targets":[],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["ken-she"],"people":[],"bottlenecks":[],"keyPapers":["paper-basu-single-dose-hpv-lancet-oncol-2021"],"journals":[],"dependsOn":[],"notes":[],"brand":"Gardasil 9","modality":"Prophylactic vaccine (virus-like particles)","mechanism":"Recombinant L1 capsid proteins self-assemble into virus-like particles that induce high-titre neutralising antibodies preventing persistent oncogenic HPV infection.","approvals":[{"region":"US","year":2014,"indication":"Prevention of HPV-related cancers and precancers, ages 9-26; extended to 45 in 2018"},{"region":"WHO","year":2022,"indication":"Single-dose schedule recommended for girls 9-20"}],"mechanismSteps":["L1 protein particles mimic the virus shell without DNA","The immune system makes neutralising antibodies","Antibodies at the cervix block HPV from entering basal cells","No persistent infection, so no precancer or cancer"],"dosing":{"route":"Intramuscular","schedule":"1 dose (WHO, age 9-20) or 2 doses 6-12 months apart (age 9-14); 3 doses at 15-45 (US label)","monitoring":"None routine"},"toxicity":[{"event":"Injection-site pain","anyGradePct":90},{"event":"Syncope (adolescents)","note":"Observe 15 minutes after injection"}],"access":[],"regulatoryEvents":[{"date":"2006-06-08","type":"approval","region":"US","note":"Original quadrivalent Gardasil approved"},{"date":"2014-12-10","type":"approval","region":"US","note":"Gardasil 9 approved"},{"date":"2022-12","type":"label-change","region":"WHO","note":"One-dose schedule endorsed (SAGE)"}]},{"id":"np137","kind":"drug","name":"NP137","aka":[],"tldr":"NP137 is a monoclonal antibody from NETRIS Pharma, in registered phase 2 trials for endometrial cancer, cervical cancer.","summary":"NP137 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by NETRIS Pharma, in endometrial cancer, cervical cancer. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of NP137","url":"https://clinicaltrials.gov/search?intr=NP137"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["endometrial","cervical"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["netris-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04652076"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"NP137","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nuv-1511","kind":"drug","name":"NUV-1511","aka":[],"tldr":"NUV-1511 is a small-molecule inhibitor from Nuvation Bio Inc., in registered phase 2 trials for prostate cancer, pancreatic ductal adenocarcinoma, ovarian cancer.","summary":"NUV-1511 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Nuvation Bio Inc., in prostate cancer, pancreatic ductal adenocarcinoma, ovarian cancer. Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of NUV-1511","url":"https://clinicaltrials.gov/search?intr=NUV-1511"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["prostate","pancreatic","ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["nuvation-bio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06334432"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"NUV-1511","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"nxc-201-car-t","kind":"drug","name":"NXC-201 CAR-T","aka":["HBI0101 CAR-T"],"tldr":"NXC-201 CAR-T is an experimental CAR-T cell therapy from Nexcella in phase 2 trials, aimed at BCMA.","summary":"NXC-201 CAR-T (NXC-201, HBI0101) is a CAR-T cell therapy developed by Nexcella. Its target is BCMA. The sponsor states: Autologous T cells transduced ex vivo with an anti-BCMA CAR retroviral vector, provided fresh without cryopreservation. ClinicalTrials.gov describes the intervention as: NXC-201 (formerly HBI0101) CAR-T is defined as autologous T cells transduced ex-vivo with anti-BCMA CAR retroviral vector encoding the chimeric antigen receptor (CAR) targeted to human BCMA. The NXC-201 CAR-T is provided fresh without cryop. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT06097832, plans to enrol 45 participants with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov. The published efficacy data come from the Hadassah phase 1 in relapsed or refractory multiple myeloma (NCT04720313; Haematologica 2023): overall response 75 percent and complete response 50 percent in the first 20 patients, with only grade 1 to 2 cytokine release syndrome.","status":"phase-2","asOf":"2026-09-24","links":[{"label":"HBI0101 phase 1 (Haematologica 2023)","url":"https://doi.org/10.3324/haematol.2022.281628"},{"label":"ClinicalTrials.gov NCT04720313","url":"https://clinicaltrials.gov/study/NCT04720313"},{"label":"ClinicalTrials.gov: trials of NXC-201 CAR-T","url":"https://clinicaltrials.gov/search?intr=NXC-201"},{"label":"Sponsor pipeline page","url":"https://www.nexcella.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["car-t"],"targets":["bcma"],"drugs":[],"companies":["immix-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nxc-201-mm"],"people":[],"bottlenecks":[],"keyPapers":["paper-nxc-201-hbi0101-asherie-haematologica-2023"],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"NXC-201, HBI0101","modality":"CAR-T cell therapy","mechanism":"Autologous T cells transduced ex vivo with an anti-BCMA CAR retroviral vector, provided fresh without cryopreservation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"obecabtagene-autoleucel","kind":"drug","name":"Obecabtagene autoleucel","aka":[],"tldr":"A CD19 CAR-T built to grip and release quickly, which cut severe side effects and gave adults with relapsed ALL a real chance at durable remission.","summary":"FELIX (n=127 infused): ORR 77%, CR 55%, median EFS 11.9 months, with grade ≥3 CRS 2.4% and grade ≥3 ICANS 7%, far lower than first-generation CD19 CARs; longer follow-up shows ~40% event-free at 2 years, most without transplant. Approved 8 November 2024 (US) for adults with relapsed/refractory B-ALL, without a REMS. The low-affinity, fast-off-rate binder (CAT) reduces exhaustion and cytokine release.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Obecabtagene%20autoleucel"}],"tags":[],"related":[],"cancers":["all-leukemia"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":[],"companies":["autolus"],"institutions":[],"pathways":[],"terms":["crs","icans"],"trials":["felix"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aucatzyl","code":"obe-cel, AUTO1","modality":"CAR-T (CD19, fast off-rate)","mechanism":"Autologous T cells with a CD19 CAR using the CAT scFv (fast target dissociation), 4-1BB costimulation; split-dose infusion by marrow blast burden.","approvals":[{"region":"US","year":2024,"indication":"Relapsed or refractory B-cell precursor ALL, adults"},{"region":"EU","year":2025,"indication":"Relapsed or refractory B-ALL, adults ≥26 years"}],"mechanismSteps":["Leukapheresis and lentiviral transduction with the CAT-CD19 CAR","Lymphodepletion with fludarabine/cyclophosphamide","Split dose on day 1 and day 10 sized to marrow blast percentage","Fast off-rate lets each CAR-T cell serially engage many blasts without over-activation","Persistence and B-cell aplasia continue for months in most responders"],"dosing":{"route":"IV, split infusion","schedule":"Total 410 × 10^6 CAR+ cells given on day 1 and day 10; day-1 fraction depends on marrow blasts (≤20% vs >20%)","monitoring":"CRS/ICANS (boxed warning) but no REMS; cytopenias; infections","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Aucatzyl"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":69,"grade3PlusPct":2.4,"source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-obecabtagene-autoleucel-adults-relapsed-or-refractory-b-cell-precursor-acute"},{"event":"ICANS","anyGradePct":23,"grade3PlusPct":7},{"event":"Prolonged cytopenias","note":"Common; infection prophylaxis"}],"access":[],"regulatoryEvents":[{"date":"2024-11-08","type":"approval","region":"US","note":"FELIX; approved without REMS","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-obecabtagene-autoleucel-adults-relapsed-or-refractory-b-cell-precursor-acute"}]},{"id":"obi-902","kind":"drug","name":"OBI-902","aka":[],"tldr":"OBI-902 is an antibody-drug conjugate from OBI Pharma, Inc, in registered phase 2 trials for metastatic cancer.","summary":"OBI-902 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by OBI Pharma, Inc, in metastatic cancer. An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of OBI-902","url":"https://clinicaltrials.gov/search?intr=OBI-902"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["obi-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07124117"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"OBI-902","modality":"Antibody-drug conjugate","mechanism":"An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"obi-992","kind":"drug","name":"OBI-992","aka":[],"tldr":"OBI-992 is an antibody-drug conjugate from OBI Pharma, Inc, in registered phase 2 trials for metastatic cancer.","summary":"OBI-992 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by OBI Pharma, Inc, in metastatic cancer. An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of OBI-992","url":"https://clinicaltrials.gov/search?intr=OBI-992"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["obi-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06480240"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"OBI-992","modality":"Antibody-drug conjugate","mechanism":"An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"obinutuzumab","kind":"drug","name":"Obinutuzumab","aka":[],"tldr":"Obinutuzumab is a glycoengineered CD20 antibody that recruits immune cells and kills B cells more directly than rituximab. Paired with venetoclax for a fixed 12 months in chronic lymphocytic leukaemia, it keeps over half of patients treatment-free six years later, and it is also used in follicular lymphoma; first-dose infusion reactions are common and managed by splitting the dose.","summary":"Glycoengineered for enhanced ADCC and direct cell death. CLL11 (with chlorambucil, OS benefit vs rituximab-chlorambucil), CLL14 (venetoclax-obinutuzumab fixed 12 months: 6-year PFS 53% vs 22%), CLL13 (GIV triplet 5-year PFS 81%), ELEVATE-TN (with acalabrutinib, OS benefit). Also follicular lymphoma and (2024-25) lupus nephritis. Infusion reactions in the first dose are common and managed by split dosing.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Obinutuzumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Obinutuzumab"}],"tags":[],"related":[],"cancers":["cll","dlbcl","cll-treatment-naive","follicular-lymphoma","non-hodgkin-lymphoma"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd20"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":["complement-in-cancer"],"terms":["adcc"],"trials":["cll14","cll13-gaia","elevate-tn","nct05100862","nct04077723","nct06090539","nct05533775","nct04787042","nct03075696","nct03930953","nct05673057","nct06943872","nct06084936","nct06428019","nct06634589","nct03533283","nct06806033","nct05057494","nct04895436","nct07520006","nct06970743"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: a glycoengineered type II anti-CD20 antibody with higher Fc receptor affinity and more direct cell death, designed around the observation that rituximab depends on effector cells that become refractory with repeated dosing. It does not solve antigen loss, which is a different problem."],"brand":"Gazyva","modality":"Monoclonal antibody (anti-CD20, glycoengineered type II)","mechanism":"Type II anti-CD20 with afucosylated Fc: strong ADCC and direct, caspase-independent cell death; less CDC than rituximab.","approvals":[{"region":"US","year":2013,"indication":"Previously untreated CLL with chlorambucil (CLL11)"},{"region":"US","year":2019,"indication":"Previously untreated CLL with venetoclax, fixed duration (CLL14); with acalabrutinib (ELEVATE-TN)"}],"mechanismSteps":["Obinutuzumab binds CD20 in a type II orientation","Direct actin-dependent lysosomal cell death is triggered","Afucosylated Fc recruits NK cells and macrophages (ADCC, ADCP)","Debulking in cycle 1 lowers TLS risk before venetoclax ramp-up"],"dosing":{"route":"IV","schedule":"Cycle 1: 100 mg day 1, 900 mg day 2, 1000 mg days 8 and 15; then 1000 mg day 1 of cycles 2-6 (with venetoclax from cycle 1 day 22 for 12 cycles total)","monitoring":"Infusion reactions (premedicate), HBV reactivation (boxed), PML (boxed), TLS, neutropenia","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Gazyva"},"toxicity":[{"event":"Infusion-related reactions","anyGradePct":45,"grade3PlusPct":9,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Gazyva","note":"CLL14 combination arm"},{"event":"Neutropenia","grade3PlusPct":53,"note":"with venetoclax"},{"event":"Infections","grade3PlusPct":18}],"access":[],"regulatoryEvents":[]},{"id":"obrixtamig","kind":"drug","name":"Obrixtamig","aka":["BI 764532","BI-764532"],"tldr":"Obrixtamig is an antibody with two arms: one grabs DLL3, a protein on the surface of most neuroendocrine carcinomas, and the other grabs a T cell, pulling the killer cell onto the cancer. It is in a phase 3 trial for neuroendocrine carcinomas outside the lung.","summary":"Obrixtamig (BI 764532) is Boehringer Ingelheim's DLL3-directed T-cell engager, a class established by tarlatamab in small-cell lung cancer. DLL3 is expressed on the surface of most small-cell lung cancers and extrapulmonary neuroendocrine carcinomas but almost no normal adult tissue. It is given intravenously with step-up dosing to limit cytokine release syndrome.\n\nThe phase 2 DAREON-5 trial reported responses in DLL3-positive small-cell lung cancer and extrapulmonary neuroendocrine carcinoma, and the phase 3 DAREON-NEC-1 trial is testing obrixtamig with carboplatin and etoposide first line in DLL3-positive extrapulmonary neuroendocrine carcinoma, the first phase 3 dedicated to that disease. The extrapulmonary neuroendocrine carcinoma page names it with peluntamig and ZG006 among the DLL3 engagers in trials.","status":"phase-3","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["tarlatamab","peluntamig","zg006"],"cancers":["extrapulmonary-nec"],"sections":[],"technologies":["t-cell-engager","bispecific-antibody"],"targets":["dll3","cd3"],"drugs":[],"companies":["boehringer-ingelheim"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"BI 764532","modality":"DLL3 x CD3 bispecific T-cell engager","mechanism":"One arm binds delta-like ligand 3 on neuroendocrine carcinoma cells and the other binds CD3 on T cells, forcing an immune synapse that makes the T cell kill the tumour cell.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"odronextamab","kind":"drug","name":"Odronextamab","aka":[],"tldr":"Odronextamab is Regeneron's CD20 bispecific, approved in Europe for lymphoma and in the US for follicular lymphoma after earlier FDA rejections over confirmatory-trial enrolment.","summary":"Odronextamab is a fully human IgG4 CD20xCD3 bispecific antibody given intravenously with step-up dosing to limit cytokine release, redirecting T cells against CD20-positive lymphoma cells. In ELM-2 it produced ORR 52% and CR 31% in relapsed or refractory DLBCL after at least 2 lines, and ORR 80% with CR 73% in follicular lymphoma. The EU granted conditional approval for both indications in August 2024. In the US, the FDA issued complete response letters in March 2024 because confirmatory-trial enrolment was insufficient, then granted accelerated approval in follicular lymphoma in July 2025; the DLBCL indication remains unapproved there. Regeneron's OLYMPIA phase 3 programme is under way to confirm the results and move into earlier lines. It is one of several CD20 bispecifics, and its story shows that regulators now want confirmatory trials enrolling before granting accelerated approval.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Odronextamab"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["t-cell-engager"],"targets":["cd20","cd3"],"drugs":[],"companies":["regeneron"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06097364","nct06149286","nct06230224","nct06091865","nct06091254","nct03888105"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lynozyfic (EU: Ordspono)","modality":"Bispecific T-cell engager (CD20×CD3)","mechanism":"Fully human IgG4 CD20×CD3 bispecific; IV step-up.","approvals":[{"region":"EU","year":2024,"indication":"R/R DLBCL and follicular lymphoma after ≥2 lines (conditional)"},{"region":"US","year":2025,"indication":"R/R follicular lymphoma after ≥2 lines (accelerated)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2024-03-25","type":"crl","region":"US","note":"CRLs for DLBCL and FL, confirmatory trial enrolment status"},{"date":"2024-08","type":"approval","region":"EU","note":"Conditional approval DLBCL and FL"},{"date":"2025-07-30","type":"approval","region":"US","note":"Accelerated approval in follicular lymphoma"}]},{"id":"ofatumumab","kind":"drug","name":"Ofatumumab","aka":["Kesimpta (multiple sclerosis formulation)"],"tldr":"Ofatumumab (Arzerra) is a fully human anti-CD20 antibody for chronic lymphocytic leukaemia, used with chlorambucil in untreated patients or alone after other drugs have failed; the same molecule is sold as Kesimpta for multiple sclerosis.","summary":"Ofatumumab received accelerated FDA approval in October 2009 for CLL refractory to fludarabine and alemtuzumab, then full approvals with chlorambucil in previously untreated CLL where fludarabine is inappropriate (COMPLEMENT-1, 2014: longer progression-free survival than chlorambucil alone), as extended treatment in recurrent or progressive CLL after response (PROLONG, 2016) and with fludarabine and cyclophosphamide in relapsed CLL (COMPLEMENT-2, 2016). The EU granted conditional authorisation in 2010; the marketing authorisation was later withdrawn for commercial reasons and Arzerra is now supplied in many countries through a compassionate programme, while Kesimpta (subcutaneous ofatumumab) is marketed for multiple sclerosis. Hepatitis B reactivation and progressive multifocal leukoencephalopathy carry boxed warnings.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Ofatumumab","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ofatumumab"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/ofatumumab"}],"tags":["nci-list"],"related":["rituximab","obinutuzumab"],"cancers":["cll"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd20"],"drugs":[],"companies":["novartis","genmab"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Arzerra","modality":"Monoclonal antibody (anti-CD20, fully human IgG1)","mechanism":"Binds a distinct small- and large-loop epitope of CD20 close to the membrane, giving potent complement-dependent cytotoxicity as well as antibody-dependent cellular cytotoxicity against B cells.","approvals":[{"region":"US","year":2009,"indication":"CLL refractory to fludarabine and alemtuzumab (accelerated)"},{"region":"US","year":2014,"indication":"Previously untreated CLL with chlorambucil; later extended treatment and relapsed CLL with fludarabine and cyclophosphamide"},{"region":"EU","year":2010,"indication":"CLL refractory to fludarabine and alemtuzumab (conditional; authorisation since withdrawn)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2009-10-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Chronic lymphocytic leukemia refractory to fludarabine and alemtuzumab"},{"date":"2014-04-17","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2009 converted to traditional approval 4.5 years after it was granted.","indication":"Chronic lymphocytic leukemia refractory to fludarabine and alemtuzumab"}]},{"id":"olaparib","kind":"drug","name":"Olaparib","aka":[],"tldr":"Olaparib was the first PARP inhibitor, and turned an inherited BRCA mutation from a risk factor into a drug target, including after surgery in breast cancer.","summary":"Approved 2014 (ovarian), then maintenance first-line (SOLO-1, 7-year OS benefit), with bevacizumab (PAOLA-1, HRD+), metastatic BRCA breast (OlympiAD), adjuvant germline-BRCA HER2-negative early breast cancer (OlympiA, OS HR 0.72), pancreatic maintenance (POLO), and prostate (PROfound; PROpel with abiraterone). AstraZeneca/Merck.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Olaparib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Olaparib"},{"label":"NICE TA886: olaparib for adjuvant treatment of BRCA mutation-positive HER2-negative high-risk early breast cancer (10 May 2023)","url":"https://www.nice.org.uk/guidance/ta886"},{"label":"NICE TA1040: olaparib for BRCA mutation-positive HER2-negative advanced breast cancer after chemotherapy (12 February 2025)","url":"https://www.nice.org.uk/guidance/ta1040"},{"label":"Lynparza label (openFDA): OlympiA and OlympiAD study sections","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22LYNPARZA%22"},{"label":"Kindler et al., POLO overall survival (JCO 2022)","url":"https://doi.org/10.1200/JCO.21.01604"},{"label":"NICE TA887: olaparib for previously treated BRCA mutation-positive hormone-relapsed metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta887"},{"label":"NICE TA951: olaparib with abiraterone for untreated hormone-relapsed metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta951"}],"tags":[],"related":["brca-germline","brca-somatic","hrd-positive"],"cancers":["ovarian","tnbc","breast-hr-positive","prostate","pancreatic","high-grade-serous-ovarian-cancer","prostate-mcrpc","tnbc-early","tnbc-metastatic","platinum-sensitive-ovarian-cancer","brca-palb2-pdac"],"sections":[],"technologies":["parp-inhibitor"],"targets":["parp","brca"],"drugs":[],"companies":["astrazeneca","merck"],"institutions":[],"pathways":[],"terms":["prostate-hrr-eligibility","prostate-uk-drug-approvals"],"trials":["olympia","nct04884360","nct04421963","nct04624204","nct05171816","nct06915025","nct01081951","nct06686030","nct07024784","nct04644289","nct03775486","nct02861573","nct06909825","nct05898399","nct06525298","nct05498155","nct01874353","nct02734004","nct03742895","nct06353386","nct03459846","nct04380636","partner","phoenix","nct06112379","polo"],"people":[],"bottlenecks":[],"keyPapers":["paper-olympia-nejm-2021","paper-couch-tnbc-germline-17-genes-jco-2015","paper-bedrosian-asco-sso-germline-testing-breast-jco-2024","paper-polo-olaparib-maintenance-gbrca-pancreatic-nejm-2019","paper-polo-overall-survival-olaparib-gbrca-pancreatic-jco-2022","paper-hu-germline-mutations-pancreatic-cancer-risk-jama-2018"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: eligibility is germline BRCA1/2 by blood test, present in about 11 to 17% of TNBC (Couch 2015, Hahnen 2017); somatic-only or HRD-positive results do not qualify under the breast labels.","Triple-negative breast cancer: 82 percent of OlympiA patients had triple-negative disease (Lynparza label), so the adjuvant indication is in practice a triple-negative one; about half of OlympiAD patients were triple-negative, and in the OlympiAD final overall survival analysis the triple-negative subgroup hazard ratio was 0.93 (0.62 to 1.43). OlympiA recruited at 22 UK sites. The UK-led PARTNER trial (30 NHS hospitals) tests olaparib added to neoadjuvant carboplatin and paclitaxel in triple-negative or germline BRCA breast cancer.","Pancreatic cancer, UK status: NICE's appraisal of olaparib for pancreatic cancer (TA750) was terminated on 8 December 2021 because AstraZeneca made no evidence submission (its published olaparib appraisals cover ovarian, breast, prostate and endometrial cancer), so maintenance olaparib for germline BRCA carriers is not routinely commissioned in England although the drug is licensed for the use in the EU and UK. POLO label figures: progression-free survival 7.4 against 3.8 months (hazard ratio 0.53; p 0.0035), overall survival 19.0 against 19.2 months (hazard ratio 0.83; p 0.3487), response 23 against 12 percent with a median duration of 25 against 4 months; 75 percent of patients had received FOLFIRINOX; nine UK sites (Edinburgh, Glasgow, Liverpool, London, Manchester, Northwood, Nottingham, Surrey).","Pancreatic ductal adenocarcinoma biomarkers: eligibility is a germline BRCA1 or BRCA2 variant, present in about 2 to 3% of patients, plus at least 16 weeks of first-line platinum without progression (POLO). Somatic BRCA, PALB2 and genomic instability scores do not qualify under the label, although rucaparib showed activity in germline PALB2 and somatic BRCA2 (Reiss 2021).","Two NICE appraisals cover olaparib in prostate cancer in England. TA887 recommends it as monotherapy for hormone-relapsed metastatic prostate cancer with BRCA1 or BRCA2 mutations that has progressed after a newer hormonal treatment; the recommendation is for BRCA specifically, not the wider homologous recombination repair panel. TA951 recommends it with abiraterone and prednisone or prednisolone for untreated hormone-relapsed metastatic prostate cancer in adults who cannot have or do not want chemotherapy, which is an unselected indication."],"brand":"Lynparza","modality":"Small-molecule PARP inhibitor","mechanism":"PARP1/2 inhibitor and trapper; synthetic lethality with HRD.","approvals":[{"region":"US","year":2014,"indication":"gBRCA ovarian cancer ≥3 lines"},{"region":"US","year":2018,"indication":"gBRCA HER2- metastatic breast cancer"},{"region":"US","year":2022,"indication":"Adjuvant gBRCA high-risk HER2- early breast cancer"},{"region":"EU","year":2019,"indication":"Germline BRCA1/2-mutated HER2-negative locally advanced or metastatic breast cancer after an anthracycline and a taxane (OlympiAD)","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/lynparza"},{"region":"EU","year":2022,"indication":"Adjuvant treatment, alone or with endocrine therapy, of germline BRCA1/2-mutated HER2-negative high-risk early breast cancer after neoadjuvant or adjuvant chemotherapy (OlympiA)","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/lynparza"},{"region":"UK","year":2023,"indication":"Adjuvant treatment of BRCA mutation-positive HER2-negative high-risk early breast cancer after chemotherapy; NICE TA886 (10 May 2023) recommends within the marketing authorisation with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta886"},{"region":"UK","year":2025,"indication":"BRCA mutation-positive HER2-negative locally advanced or metastatic breast cancer after an anthracycline and a taxane; NICE TA1040 (12 February 2025) recommends with a commercial arrangement, the lowest-cost option to be used where talazoparib is also suitable","note":"https://www.nice.org.uk/guidance/ta1040"},{"region":"US","year":2019,"indication":"Maintenance treatment of deleterious or suspected deleterious germline BRCA-mutated metastatic pancreatic adenocarcinoma whose disease has not progressed on at least 16 weeks of first-line platinum-based chemotherapy (POLO), with an FDA-approved companion diagnostic","note":"Lynparza label (openFDA), indication 1.6 and POLO study section: https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22LYNPARZA%22"},{"region":"EU","year":2020,"indication":"Maintenance treatment of germline BRCA1/2-mutated metastatic pancreatic adenocarcinoma that has not progressed after at least 16 weeks of platinum-based first-line chemotherapy; listed in the EMA Lynparza product information read on 24 September 2026 (extension of indication in 2020)","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/lynparza"}],"mechanismSteps":["Olaparib binds PARP1/2 at sites of single-strand DNA breaks","PARP is trapped on DNA and repair of single-strand breaks stops","Replication forks collide with trapped PARP and collapse into double-strand breaks","BRCA-deficient cells cannot repair the breaks by homologous recombination","Genomic catastrophe and apoptosis in tumour cells; normal cells with intact BRCA survive"],"dosing":{"route":"Oral","schedule":"300 mg twice daily; 1 year adjuvant (OlympiA); 2 years first-line ovarian maintenance; until progression otherwise","modifications":"200 mg BID for moderate renal impairment; hold for grade ≥3 anaemia; discontinue for MDS/AML or pneumonitis","monitoring":"Blood counts monthly; respiratory symptoms; embryo-fetal counselling","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa"},"toxicity":[{"event":"Nausea","anyGradePct":57,"grade3PlusPct":0.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"},{"event":"Fatigue","anyGradePct":42,"grade3PlusPct":1.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"},{"event":"Anaemia","anyGradePct":24,"grade3PlusPct":9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"},{"event":"Vomiting","anyGradePct":23,"grade3PlusPct":0.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"},{"event":"Diarrhoea","anyGradePct":18,"grade3PlusPct":0.3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"},{"event":"Leukopenia","anyGradePct":17,"grade3PlusPct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"},{"event":"Neutropenia","anyGradePct":16,"grade3PlusPct":5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"},{"event":"MDS/AML","anyGradePct":1.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"Cumulative across trials; 54% fatal"},{"event":"Pneumonitis","anyGradePct":1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"},{"event":"Venous thromboembolism (prostate)","anyGradePct":8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=741ff3e3-dc1a-45a6-84e5-2481b27131aa","note":"OlympiA adjuvant, n=911"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.astrazeneca-us.com/medicines/access-360","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended in ovarian maintenance (TA598, TA620), adjuvant gBRCA breast (TA886), metastatic breast (TA1100), prostate (TA887)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"},{"country":"EU","reimbursement":"EMA approved across indications","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2014-12-19","type":"accelerated-approval","region":"US","note":"gBRCA advanced ovarian cancer after ≥3 lines: first PARP inhibitor","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Deleterious or suspected deleterious germline BRCA-mutated advanced ovarian cancer after treatment with 3 or more lines of chemotherapy 2"},{"date":"2017-08-17","type":"conversion","region":"US","note":"Maintenance in platinum-sensitive recurrent ovarian cancer; tablet formulation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Deleterious or suspected deleterious germline BRCA-mutated advanced ovarian cancer after treatment with 3 or more lines of chemotherapy 2"},{"date":"2018-01-12","type":"approval","region":"US","note":"gBRCA HER2-negative metastatic breast cancer (OlympiAD)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2018-12-19","type":"approval","region":"US","note":"First-line maintenance in BRCA-mutated ovarian cancer (SOLO-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2019-12-27","type":"approval","region":"US","note":"gBRCA metastatic pancreatic cancer maintenance (POLO)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-05-19","type":"approval","region":"US","note":"HRR-mutant mCRPC (PROfound)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-03-11","type":"approval","region":"US","note":"Adjuvant gBRCA high-risk HER2-negative early breast cancer (OlympiA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-05-31","type":"approval","region":"US","note":"With abiraterone in BRCA-mutated mCRPC (PROpel)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"olaratumab","kind":"drug","name":"Olaratumab","aka":["IMC-3G3","LY3012207"],"tldr":"Olaratumab was approved in 2016 with doxorubicin for soft tissue sarcoma after a small trial suggested it added almost a year of life, but the large confirmatory trial found no benefit and it was withdrawn in 2019, a warning case for accelerated approvals.","summary":"Eli Lilly's olaratumab received FDA accelerated approval in October 2016, and EU conditional approval the same year, for adults with soft tissue sarcoma not curable by surgery or radiotherapy, in combination with doxorubicin, after a randomised phase 2 trial reported median survival of 26.5 months versus 14.7 months with doxorubicin alone. The phase 3 ANNOUNCE trial in 509 patients then showed no difference in overall survival, and Lilly withdrew the drug worldwide in 2019. The episode is cited whenever the reliability of small randomised phase 2 survival signals is debated.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Olaratumab","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Olaratumab"},{"label":"ChEMBL CHEMBL2109625","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL2109625"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["sarcoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":[],"drugs":["doxorubicin"],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lartruvo","modality":"Anti-PDGFR alpha monoclonal antibody, intravenous","mechanism":"A human IgG1 antibody that binds platelet-derived growth factor receptor alpha and stops its ligands activating it on tumour and stromal cells.","approvals":[{"region":"US","year":2016,"indication":"Soft tissue sarcoma not curable by surgery or radiotherapy, with doxorubicin (accelerated approval)","note":"Withdrawn in 2019 after the ANNOUNCE trial"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2016-10-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with doxorubicin for adults with soft tissue sarcoma with a histologic subtype for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery"},{"date":"2020-02-25","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.4 years after its accelerated approval.","indication":"In combination with doxorubicin for adults with soft tissue sarcoma with a histologic subtype for which an anthracycline-containing regimen is appropriate and which is not amenable to curative treatment with radiotherapy or surgery"}]},{"id":"oleclumab","kind":"drug","name":"Oleclumab","aka":[],"tldr":"Oleclumab is an experimental investigational agent whose form is not stated in the registry from AstraZeneca in phase 3 trials for non-small-cell lung cancer, triple-negative breast cancer and colorectal cancer, with its target not yet stated publicly.","summary":"Oleclumab (MEDI9447) is an investigational agent whose form is not stated in the registry developed by AstraZeneca and MedImmune. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Participants will receive oleclumab in combination with osimertinib or AZD4635 as stated in the arms' description. It is the investigational product in 7 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05221840 (A Global Study to Assess the Effects of Durvalumab With Oleclumab or Durvalumab With Monalizumab Following Concurrent Chemoradiation in Patients With Stage III Unresectable Non-Small Cell Lung Cancer), in non-small-cell lung cancer, triple-negative breast cancer and colorectal cancer. The largest, NCT05221840, plans to enrol 1051 participants (actual) with primary completion expected 2026-09-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Oleclumab","url":"https://clinicaltrials.gov/search?intr=MEDI9447"},{"label":"NCI Drug Dictionary: oleclumab","url":"https://www.cancer.gov/publications/dictionaries/cancer-drug/def/oleclumab"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","tnbc","colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05221840","nct06606847","nct04068610","nct05061550","nct03381274","nct03742102"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Oleclumab (MEDI9447) is a monoclonal antibody against the ectoenzyme CD73, also called ecto-5'-nucleotidase, which converts AMP to adenosine; blocking it lowers free adenosine and lifts adenosine-mediated suppression of CD8 T cells (NCI Drug Dictionary). CD73 has no target record in the corpus yet."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"MEDI9447","modality":"antibody (INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"olmutinib","kind":"drug","name":"Olmutinib","aka":["HM61713","BI 1482694"],"tldr":"Olmutinib was a Korean-developed EGFR pill approved in South Korea in 2016 for lung cancers that had developed the T790M resistance mutation, the same niche as osimertinib; severe skin reactions and osimertinib's success ended its development.","summary":"Hanmi Pharmaceutical's olmutinib became the first third-generation EGFR inhibitor to reach any market when South Korea's Ministry of Food and Drug Safety approved it in May 2016 for T790M-positive non-small cell lung cancer after first-line EGFR therapy. Boehringer Ingelheim licensed it worldwide but returned the rights within months after reports of fatal toxic epidermal necrolysis and Stevens-Johnson syndrome and after osimertinib's phase 3 results. Hanmi ended development in 2018 and the drug is no longer marketed. It is remembered as a case study in how a first-to-market approval on early data can be overtaken.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Olmutinib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Olmutinib"},{"label":"ChEMBL CHEMBL3545110","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL3545110"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":["osimertinib"],"companies":["hanmi-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02485652","nct02444819","nct03228277"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Olita","modality":"Oral third-generation EGFR kinase inhibitor","mechanism":"A covalent inhibitor that binds cysteine 797 of EGFR and blocks the T790M resistance mutation while sparing the normal receptor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"olomorasib","kind":"drug","name":"Olomorasib","aka":[],"tldr":"Olomorasib is Lilly's KRAS G12C pill, designed to combine safely with immunotherapy in first-line lung cancer.","summary":"Second-generation covalent G12C inhibitor with a cleaner liver profile than sotorasib in combination with pembrolizumab (ORR 74% in first-line PD-L1 ≥50%, JTO 2025). Breakthrough Therapy designation with pembrolizumab in untreated KRAS G12C NSCLC and in KRAS G12C pancreatic cancer. Phase 3 SUNRAY-01 (first line ± chemotherapy) and SUNRAY-02 (early-stage) ongoing.","status":"phase-3","asOf":"2026-09-06","links":[{"label":"OncLive: Breakthrough designation","url":"https://www.onclive.com/view/olomorasib-plus-pembrolizumab-earns-breakthrough-therapy-designation-in-untreated-kras-g12c-mutated-nsclc"}],"tags":[],"related":[],"cancers":["nsclc","pancreatic","kras-g12c-pdac","kras-g12c-nsclc"],"sections":[],"technologies":["kras-inhibitors"],"targets":["kras"],"drugs":["pembrolizumab"],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04956640","nct06119581","nct07227025","nct06890598"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"LY3537982","modality":"Small-molecule inhibitor (KRAS G12C)","mechanism":"Covalent KRAS G12C inhibitor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"olutasidenib","kind":"drug","name":"Olutasidenib","aka":[],"tldr":"Olutasidenib is a second IDH1 inhibitor for relapsed AML, with a higher response rate in its pivotal cohort than ivosidenib had in its own.","summary":"Olutasidenib is a selective, allosteric inhibitor of mutant IDH1 that lowers the oncometabolite 2-hydroxyglutarate and lets blocked myeloid cells differentiate. It was approved on 1 December 2022 for relapsed or refractory IDH1-mutated AML, taken twice daily for a minimum of 6 months. In the pivotal cohort (n=147) the CR plus CRh rate was 35% with a median duration of 25.9 months, higher than ivosidenib achieved in its own pivotal cohort, although the two were not compared directly. Differentiation syndrome (16%) carries a boxed warning, and liver enzymes and QT are monitored. Forma Therapeutics developed it and licensed it to Rigel, and combination trials with azacitidine and venetoclax, and a frontline study, are ongoing. It is a second pill for the same genetic subtype of AML, working by switching differentiation back on rather than killing cells.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Olutasidenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Olutasidenib"}],"tags":[],"related":["idh1-r132"],"cancers":["aml","aml-idh"],"sections":[],"technologies":["epigenetic-drugs","idh-inhibitors"],"targets":["idh"],"drugs":[],"companies":["rigel-pharmaceuticals"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome","pivotal-trial"],"trials":["nct06161974"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Rezlidhia","modality":"Small-molecule IDH1 inhibitor","mechanism":"Selective, allosteric mutant-IDH1 inhibitor lowering 2-HG.","approvals":[{"region":"US","year":2022,"indication":"Relapsed/refractory IDH1-mutated AML"}],"mechanismSteps":["Binds mutant IDH1 and blocks 2-HG production","Epigenetic demethylation restores differentiation programmes","Blasts mature; responses can take several months"],"dosing":{"route":"Oral","schedule":"150 mg twice daily on an empty stomach, until progression (minimum 6 months)","monitoring":"Differentiation syndrome (boxed warning), hepatotoxicity, QT","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rezlidhia"},"toxicity":[{"event":"Differentiation syndrome","anyGradePct":16,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rezlidhia"},{"event":"Transaminase elevation","anyGradePct":23},{"event":"Nausea","anyGradePct":38}],"access":[],"regulatoryEvents":[{"date":"2022-12-01","type":"approval","region":"US","note":"Study 2102-HEM-101 pivotal cohort"}]},{"id":"olverembatinib","kind":"drug","name":"Olverembatinib","aka":["GZD824"],"tldr":"Olverembatinib is Ascentage Pharma's third-generation BCR-ABL inhibitor, approved in China in 2021 for chronic myeloid leukaemia carrying the T315I mutation, and now in global phase 3 trials against the established drugs; it was the one China-only approval OnCo found missing from the headline list.","summary":"Olverembatinib (HQP1351) was developed by Ascentage Pharma in Suzhou and approved by China's NMPA in November 2021 for chronic-phase and accelerated-phase chronic myeloid leukaemia with the T315I mutation, with a later extension to patients resistant or intolerant to first and second-generation inhibitors. Its global programme, partnered with Takeda from 2024, includes the phase 3 POLARIS trials in previously treated chronic-phase CML and in Philadelphia-positive acute lymphoblastic leukaemia. It competes with ponatinib and asciminib, the two approved options for T315I disease outside China.","status":"approved","asOf":"2026-09-16","wikipedia":"https://en.wikipedia.org/wiki/Olverembatinib","links":[{"label":"ClinicalTrials.gov: trials of Olverembatinib","url":"https://clinicaltrials.gov/search?intr=HQP1351"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cml","all-leukemia","cml-advanced-phase"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["bcr-abl"],"drugs":[],"companies":["ascentage-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06423911","nct06640361","nct06051409","nct04501120"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"HQP1351","modality":"Oral third-generation BCR-ABL tyrosine kinase inhibitor","mechanism":"Inhibits BCR-ABL including the T315I gatekeeper mutation that defeats imatinib, dasatinib and nilotinib, and several other kinases; taken every other day.","approvals":[{"region":"CN","year":2021,"indication":"Chronic myeloid leukaemia (chronic or accelerated phase) with the T315I mutation after prior tyrosine kinase inhibitors"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"olvimulogene-nanivacirepvec","kind":"drug","name":"Olvimulogene nanivacirepvec","aka":["GL-ONC1 and GLV-1h68","Olvi-Vec","GL-ONC","GLV-1h68"],"tldr":"Olvimulogene nanivacirepvec is an experimental oncolytic virus from Genelux in phase 3 trials for ovarian cancer and non-small-cell lung cancer, with its target not yet stated publicly.","summary":"Olvimulogene nanivacirepvec is an oncolytic virus developed by Genelux. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Olvi-Vec is an engineered oncolytic vaccinia virus. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05281471 (Efficacy & Safety of Olvi-Vec and Platinum-doublet + Bevacizumab Compared to Physician's Choice of Chemotherapy and Bevacizumab in Platinum-Resistant/Refractory Ovarian Cancer (PRROC) (OnPrime, GOG-3076)), in ovarian cancer and non-small-cell lung cancer. The largest, NCT05281471, plans to enrol 186 participants with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Olvimulogene nanivacirepvec","url":"https://clinicaltrials.gov/search?intr=Olvimulogene%20nanivacirepvec"},{"label":"Sponsor page","url":"https://www.genelux.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian","nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["genelux"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05281471","nct06463665"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"oncolytic virus","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"omacetaxine","kind":"drug","name":"Omacetaxine mepesuccinate","aka":["Homoharringtonine"],"tldr":"Omacetaxine (Synribo) is a twice-daily injection under the skin for chronic myeloid leukaemia that has stopped responding to at least two tyrosine kinase inhibitor pills.","summary":"Omacetaxine mepesuccinate, a semi-synthetic form of the plant alkaloid homoharringtonine, was granted accelerated approval by the FDA in October 2012, converted to full approval in 2014, for chronic or accelerated phase CML with resistance or intolerance to two or more tyrosine kinase inhibitors, on pooled phase 2 studies in which a minority of heavily pretreated patients achieved major cytogenetic responses, including those with the T315I mutation. Because it does not act on the kinase, it retains activity against any BCR-ABL mutation, but asciminib and ponatinib have narrowed its role. Myelosuppression is the main toxicity, and it has not been authorised in the EU.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Omacetaxine_mepesuccinate","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=omacetaxine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/omacetaxinemepesuccinate"}],"tags":["nci-list"],"related":[],"cancers":["cml"],"sections":[],"technologies":[],"targets":["bcr-abl"],"drugs":[],"companies":["teva"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Synribo","modality":"Protein synthesis inhibitor (cephalotaxine ester)","mechanism":"Binds the A-site cleft of the ribosome and blocks the elongation step of protein synthesis; reduces short-lived oncoproteins such as BCR-ABL and MCL1 independently of kinase mutations.","approvals":[{"region":"US","year":2012,"indication":"Chronic or accelerated phase CML resistant or intolerant to two or more TKIs (accelerated; full approval 2014)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2012-10-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adults with chronic or accelerated phase chronic myeloid leukemia with resistance or intolerance to 2 or more TKIs"},{"date":"2014-02-10","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2012 converted to traditional approval 1.3 years after it was granted.","indication":"Adults with chronic or accelerated phase chronic myeloid leukemia with resistance or intolerance to 2 or more TKIs"}]},{"id":"omidubicel","kind":"drug","name":"Omidubicel","aka":[],"tldr":"An expanded umbilical cord blood graft that shortens the dangerous wait for neutrophils to return after a transplant for blood cancer, the first cell therapy approved to speed engraftment.","summary":"Omidubicel takes a single umbilical cord blood unit and expands its stem and progenitor cells for three weeks with nicotinamide, so that patients with blood cancers who need a cord blood transplant but lack a matched donor receive a larger graft. In its phase 3 trial against standard cord blood transplantation, neutrophil recovery came sooner and serious bacterial and fungal infections were fewer. The FDA approved it in April 2023 for adults and children aged 12 and over with haematologic malignancies planned for cord blood transplantation after myeloablative conditioning. It is manufactured for each patient from a matched cord blood unit.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA approval announcement (April 2023)","url":"https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell"}],"tags":[],"related":[],"cancers":["aml","all-leukemia","mds"],"sections":["cell-therapy"],"technologies":["allogeneic-hsct"],"targets":[],"drugs":[],"companies":["gamida-cell"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02730299"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Omisirge","modality":"Nicotinamide-expanded allogeneic cord blood cell therapy","mechanism":"Ex vivo nicotinamide-based expansion of CD34-positive cord blood cells preserves stemness while multiplying the graft, so engraftment after myeloablative conditioning is faster.","approvals":[{"region":"US","year":2023,"indication":"Adults and children aged 12 and over with haematologic malignancies undergoing umbilical cord blood transplantation after myeloablative conditioning, to reduce time to neutrophil recovery and infection"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"oncodetect","kind":"drug","name":"Oncodetect","aka":[],"tldr":"Exact Sciences' entry into the leftover-cancer blood test market, launched in 2025 for bowel cancer follow-up.","summary":"Oncodetect launched in 2025 with initial validation in stage II-IV colorectal cancer, where the company reported that ctDNA positivity after surgery and during surveillance was strongly associated with recurrence, and Medicare coverage for colorectal surveillance followed. Exact Sciences has Cologuard's primary-care channel and the Oncotype DX oncology sales force behind it, which makes the test a commercial rather than technical challenge to Signatera. Broader indications and interventional trials are pending.","status":"emerging","asOf":"2026-09-10","links":[{"label":"Exact Sciences: Oncodetect","url":"https://www.exactsciences.com/"}],"tags":["test"],"related":["signatera"],"cancers":["colorectal"],"sections":[],"technologies":["mrd-testing","liquid-biopsy"],"targets":[],"drugs":[],"companies":["exact-sciences"],"institutions":[],"pathways":[],"terms":["mrd","ctdna"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-reinert-ctdna-ultradeep-sequencing-colorectal-jama-oncol-2019"],"journals":[],"dependsOn":[],"notes":["What a result means: the same as other tumour-informed MRD tests; ask whether a positive result would change your treatment before having it."],"brand":"Oncodetect","modality":"Tumour-informed ctDNA minimal residual disease test","mechanism":"Whole-exome sequencing of tumour tissue designs a patient-specific panel of up to 200 variants tracked in plasma by targeted sequencing.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"oncomine-dx-target-test","kind":"drug","name":"Oncomine Dx Target Test","aka":[],"tldr":"The first FDA-approved gene panel that could match one biopsy to several different lung cancer drugs at once.","summary":"Approved 22 June 2017 (PMA P160045, Life Technologies, a Thermo Fisher business) as the first next-generation sequencing companion diagnostic covering multiple markers: BRAF V600E (dabrafenib plus trametinib), ROS1 fusions (crizotinib) and EGFR mutations (gefitinib) in non-small-cell lung cancer. Claims were later added for RET fusions (pralsetinib, 2020) and for IDH1 mutations in cholangiocarcinoma (ivosidenib, 2021). It was also approved in Japan (2019) as a companion diagnostic system reimbursed for lung cancer. Its short turnaround and modest tissue requirement made it a workhorse for in-hospital laboratories, in contrast to send-out tests such as FoundationOne CDx.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P160045","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P160045"},{"label":"Thermo Fisher: Oncomine Dx Target Test","url":"https://www.thermofisher.com/uk/en/home/clinical/diagnostic-testing/condition-disease-diagnostics/oncology-diagnostics/oncomine-dx-target-test.html"}],"tags":["test"],"related":[],"cancers":["nsclc","cholangiocarcinoma"],"sections":[],"technologies":["companion-diagnostic","cgp"],"targets":["egfr","braf","ros1","ret","idh"],"drugs":["pralsetinib","ivosidenib"],"companies":["thermo-fisher"],"institutions":[],"pathways":[],"terms":["ngs","companion-diagnostic-term"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: a reported driver alteration points to a specific approved pill; panels need enough tumour in the biopsy, and a failed sample is not a negative result."],"brand":"Oncomine Dx Target Test","modality":"Tissue NGS companion diagnostic test (multi-gene panel)","mechanism":"Amplicon-based DNA and RNA sequencing on the Ion Torrent platform, detecting variants in 23 genes from formalin-fixed tumour tissue in a few days, with companion claims for several lung and bile-duct cancer drugs.","approvals":[{"region":"US","year":2017,"indication":"Companion diagnostic for BRAF, ROS1 and EGFR-directed therapies in NSCLC"},{"region":"Japan","year":2019,"indication":"Multi-gene companion diagnostic system for NSCLC"},{"region":"US","year":2020,"indication":"RET fusion companion claim (pralsetinib)"},{"region":"US","year":2021,"indication":"IDH1 companion claim in cholangiocarcinoma (ivosidenib)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"oncotype-dx","kind":"drug","name":"Oncotype DX","aka":[],"tldr":"A 21-gene test that tells most women with early hormone-positive breast cancer whether they can safely skip chemotherapy.","summary":"Oncotype DX is a gene-expression assay that measures 16 cancer-related and 5 reference genes by RT-PCR in tumour tissue to give a recurrence score estimating both the risk of distant recurrence and the likely benefit from chemotherapy in early hormone-positive, HER2-negative breast cancer. TAILORx (2018) showed that women with a score of 25 or below, if over 50, derive no benefit from adding chemotherapy to endocrine therapy, and RxPONDER extended that finding to postmenopausal women with 1 to 3 positive nodes. Younger women with mid-range scores did show some chemotherapy benefit in TAILORx, which may reflect ovarian suppression rather than cytotoxic effect, and this remains debated. Exact Sciences markets it; MammaPrint (Agendia) and Prosigna/PAM50 are alternatives. For a newcomer: a tumour test that lets most women with early hormone-positive breast cancer safely skip chemotherapy.","status":"established","asOf":"2026-09-04","links":[{"label":"TAILORx (NEJM 2018)","url":"https://doi.org/10.1056/NEJMoa1804710"},{"label":"NICE DG34: tumour profiling tests for breast cancer","url":"https://www.nice.org.uk/guidance/dg34"}],"tags":[],"related":["mammaprint","prosigna","endopredict","genomic-assay-to-chemo-omission"],"cancers":["breast-hr-positive","hr-positive-early-high-risk"],"sections":[],"technologies":["rna-seq","companion-diagnostic","gene-expression-prognostic-assays"],"targets":[],"drugs":[],"companies":["exact-sciences"],"institutions":[],"pathways":[],"terms":[],"trials":["tailorx","rxponder"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Gene-expression prognostic/predictive assay","mechanism":"RT-PCR of 16 cancer and 5 reference genes into a recurrence score.","approvals":[],"mechanismSteps":["RNA is extracted from the tumour block","Expression of 16 cancer genes and 5 reference genes is measured by RT-PCR","A recurrence score from 0 to 100 is computed","Score and menopausal status predict chemotherapy benefit"],"toxicity":[],"access":[{"country":"US","listPrice":"~$4,500 list price","reimbursement":"Medicare and most commercial plans for node-negative and node-positive (1-3) HR+/HER2- early breast cancer","source":"https://www.oncotypeiq.com","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for ER+/HER2- node-negative and some node-positive early breast cancer (DG58)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2004-01","type":"filing","region":"US","note":"Launched as a laboratory-developed test","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2018-06-03","type":"label-change","region":"US","note":"TAILORx results define recurrence score ≤25 as chemotherapy-sparing","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-12","type":"label-change","region":"US","note":"RxPONDER extends to 1-3 positive nodes (postmenopausal)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"oncotype-dx-gps","kind":"drug","name":"Oncotype DX Genomic Prostate Score","aka":[],"tldr":"A 17-gene biopsy test that helps men with newly diagnosed low-risk prostate cancer decide whether to watch or treat.","summary":"Developed by Genomic Health and sold by Exact Sciences until MDxHealth acquired the test in 2022, the Genomic Prostate Score predicts adverse pathology at prostatectomy and long-term metastasis and death, validated in surgical cohorts and in the Kaiser Permanente and Veterans Affairs populations. NCCN lists it for very-low to favourable-intermediate risk disease. In practice it is ordered when a man and his urologist are on the fence about active surveillance. As with Prolaris and Decipher, evidence that the test changes long-term outcomes rather than decisions is lacking.","status":"established","asOf":"2026-09-10","links":[{"label":"MDxHealth: Oncotype DX GPS (page moved; nearest live section)","url":"https://mdxhealth.com/"}],"tags":["test"],"related":["decipher-prostate","prolaris"],"cancers":["prostate"],"sections":[],"technologies":["rna-seq","active-surveillance"],"targets":[],"drugs":[],"companies":["mdxhealth"],"institutions":[],"pathways":[],"terms":["gleason-grade-group","active-surveillance-term"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: a low GPS supports active surveillance; higher scores predict more aggressive disease at surgery."],"brand":"Oncotype DX GPS","modality":"Gene-expression prognostic assay (17-gene Genomic Prostate Score)","mechanism":"RT-PCR of 12 cancer genes across four pathways (androgen signalling, proliferation, cellular organisation, stromal response) and five reference genes on biopsy tissue, giving a 0-100 Genomic Prostate Score.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ondansetron","kind":"drug","name":"Ondansetron","aka":["Zuplenz (oral film)"],"tldr":"Ondansetron (Zofran) was the drug that made cisplatin-type chemotherapy bearable. It blocks the serotonin signal that triggers vomiting and is now a cheap generic tablet, dissolving wafer or injection used across cancer care.","summary":"Ondansetron was approved in January 1991 for prevention of nausea and vomiting from highly emetogenic chemotherapy including cisplatin, with moderately emetogenic chemotherapy, radiotherapy (total body irradiation and abdominal fields) and post-operative nausea added later, and oral, orally disintegrating, film and injectable forms available. Its introduction, the first selective 5-HT3 antagonist, transformed supportive care and it remains the backbone of antiemetic prophylaxis worldwide and on the WHO essential medicines list. QT prolongation led the FDA to withdraw the 32 mg single intravenous dose in 2012; headache and constipation are common and serotonin syndrome is a rare interaction. Palonosetron and granisetron are the longer-acting alternatives.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Ondansetron","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ondansetron"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/ondansetronhydrochloride"}],"tags":["nci-list","supportive","generic"],"related":["granisetron","palonosetron","aprepitant"],"cancers":[],"sections":[],"technologies":["antiemetic-therapy"],"targets":[],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06904235"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zofran","modality":"5-HT3 receptor antagonist (antiemetic)","supportive":true,"mechanism":"Selective antagonist of serotonin 5-HT3 receptors on vagal afferent nerves and in the chemoreceptor trigger zone; blocks the serotonin surge released from enterochromaffin cells by chemotherapy and radiation.","approvals":[{"region":"US","year":1991,"indication":"Prevention of nausea and vomiting from highly emetogenic chemotherapy; later moderately emetogenic chemotherapy, radiotherapy and post-operative nausea"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"onvansertib","kind":"drug","name":"Onvansertib","aka":[],"tldr":"Onvansertib is an oral serine/threonine kinase inhibitor from Cardiff Oncology, in registered phase 2 trials for colorectal cancer.","summary":"Onvansertib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Cardiff Oncology, in colorectal cancer. A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Onvansertib","url":"https://clinicaltrials.gov/search?intr=Onvansertib"},{"label":"NCI Drug Dictionary: onvansertib","url":"https://www.cancer.gov/publications/dictionaries/cancer-drug/def/onvansertib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["plk1"],"drugs":[],"companies":["cardiff-oncology"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06106308"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Onvansertib is an orally bioavailable, ATP-competitive inhibitor of polo-like kinase 1, which disrupts mitosis and causes G2/M arrest in PLK1-overexpressing cells (NCI Drug Dictionary)."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral serine/threonine kinase inhibitor","mechanism":"A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"opevesostat","kind":"drug","name":"Opevesostat","aka":[],"tldr":"Opevesostat is an experimental small-molecule drug from Merck Sharp & Dohme in phase 3 trials for prostate cancer, with its target not yet stated publicly.","summary":"Opevesostat (MK-5684, ODM-208) is a small-molecule drug developed by Merck Sharp & Dohme. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 5 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06136624 (Study of Opevesostat (MK-5684) Versus Alternative NHA in mCRPC (MK-5684-003)) and NCT06136650 (A Study of Opevesostat (MK-5684) Versus Alternative Next-generation Hormonal Agent (NHA) in Metastatic Castration-resistant Prostate Cancer (mCRPC) Post One NHA (MK-5684-004)), in prostate cancer. The largest, NCT06136650, plans to enrol 1314 participants with primary completion expected 2028-05-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Opevesostat","url":"https://clinicaltrials.gov/search?intr=MK-5684"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06136624","nct06136650","nct03436485","nct06979596","nct06863272","nct06353386"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"MK-5684, ODM-208","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"oprelvekin","kind":"drug","name":"Oprelvekin","aka":["Recombinant interleukin-11","rhIL-11"],"tldr":"Oprelvekin was the first drug approved, in 1997, to prevent the severe platelet falls that chemotherapy causes, but fluid retention and heart rhythm problems limited it and it was withdrawn in 2011; thrombopoietin agonists took over the problem.","summary":"Oprelvekin, recombinant human interleukin-11 made by Genetics Institute and later Wyeth, was approved by the FDA in November 1997 to prevent severe thrombocytopenia and reduce the need for platelet transfusions after myelosuppressive chemotherapy in adults with non-myeloid malignancies, after a randomised trial in which fewer treated patients needed transfusions. Fluid retention, dilutional anaemia, atrial arrhythmias and, rarely, anaphylaxis limited its use, and Pfizer withdrew it from the market in 2011 for commercial reasons. Romiplostim and eltrombopag now cover the thrombopoietic role, though neither is approved for chemotherapy-induced thrombocytopenia.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Oprelvekin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Oprelvekin"},{"label":"ChEMBL CHEMBL1201564","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201564"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":[],"sections":[],"technologies":["cytokine-therapy"],"targets":["il11ra"],"drugs":["romiplostim","eltrombopag"],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00493181","nct00004157"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Neumega","modality":"Recombinant interleukin-11, subcutaneous injection","supportive":true,"mechanism":"Activates the IL-11 receptor on megakaryocyte progenitors to increase platelet production after chemotherapy.","approvals":[{"region":"US","year":1997,"indication":"Prevention of severe thrombocytopenia and reduction of platelet transfusions after myelosuppressive chemotherapy in non-myeloid malignancies","note":"Withdrawn in 2011"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"optellum-virtual-nodule-clinic","kind":"drug","name":"Optellum Virtual Nodule Clinic","aka":[],"tldr":"The first AI cleared to estimate how likely a lung nodule on a CT scan is to be cancer, helping doctors decide who needs a biopsy and who can wait.","summary":"Optellum (Oxford, UK) received FDA 510(k) clearance in March 2021 for the Virtual Nodule Clinic, the first AI decision-support software for lung cancer diagnosis, and is CE-marked. The LCP score was validated on the NLST and other cohorts and, in reader studies, improved clinicians' discrimination of benign from malignant nodules; the product is used in NHS trusts and US health systems to run nodule clinics and to prioritise early biopsy of high-risk nodules. As lung screening programmes expand, most nodules found are benign, and the value of such tools lies in reducing unnecessary procedures without delaying cancer diagnoses.","status":"approved","asOf":"2026-09-10","links":[{"label":"Optellum","url":"https://optellum.com"}],"tags":["test"],"related":["sybil"],"cancers":["nsclc"],"sections":[],"technologies":["radiology-ai-screening","low-dose-ct-screening","ct"],"targets":[],"drugs":[],"companies":["optellum"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: a low malignancy score supports watchful CT follow-up of a nodule; a high score speeds up PET scan or biopsy."],"brand":"Virtual Nodule Clinic","modality":"AI lung nodule malignancy prediction software (CT)","mechanism":"Deep-learning Lung Cancer Prediction (LCP) score on CT images of indeterminate pulmonary nodules, combined with nodule tracking software to support clinic decision-making.","approvals":[{"region":"US","year":2021,"indication":"510(k) clearance: AI decision support for indeterminate pulmonary nodules"},{"region":"EU","year":2020,"indication":"CE mark"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"optune","kind":"drug","name":"Optune / Optune Pax (TTFields)","aka":[],"tldr":"Optune is a wearable device delivering electric fields that disrupt cell division. It is approved for glioblastoma and, in 2026, pancreatic cancer.","summary":"Optune delivers tumour treating fields (TTFields): low-intensity alternating electric fields at 200 kHz for glioma or 150 kHz for pancreatic and lung cancer, applied through transducer arrays worn for at least 18 hours a day, which disrupt mitotic spindle assembly. Optune Gio is approved for recurrent (2011) and newly diagnosed (2015, EF-14) glioblastoma and for mesothelioma; Optune Lua for metastatic NSCLC after platinum (LUNAR, 2024); and Optune Pax, approved in Q1 2026, for unresectable locally advanced pancreatic cancer with gemcitabine and nab-paclitaxel after PANOVA-3 showed overall survival of 16.2 versus 14.2 months. Novocure makes the devices. The pivotal trials were open-label without a sham device, and the mechanism and effect size remain contested among neuro-oncologists. For a newcomer: a wearable that uses electric fields, not drugs, to slow cell division.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Tumor_treating_fields","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Tumor_treating_fields"},{"label":"Babiker et al., PANOVA-3 (JCO 2025)","url":"https://doi.org/10.1200/JCO-25-00746"}],"tags":[],"related":[],"cancers":["glioblastoma","pancreatic","locally-advanced-pdac","nsclc","mesothelioma"],"sections":[],"technologies":["ttfields"],"targets":[],"drugs":[],"companies":["novocure"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer, UK status: no NICE technology appraisal or medical technologies guidance for tumour treating fields in pancreatic cancer was listed on 24 September 2026. PANOVA-3 (JCO 2025): overall survival 16.2 against 14.2 months (hazard ratio 0.82; p 0.039), pain-free survival 15.2 against 9.1 months (hazard ratio 0.74), distant progression-free survival 13.9 against 11.5 months; progression-free survival, local control and response not improved; device-related skin events in 76.3 percent (grade 3 in 7.7 percent); no UK sites."],"brand":"Optune","modality":"Device (tumour treating fields)","mechanism":"200 kHz (glioma) or 150 kHz (pancreas, lung) alternating fields via transducer arrays.","approvals":[{"region":"US","year":2011,"indication":"Recurrent glioblastoma (newly diagnosed 2015)"},{"region":"US","year":2024,"indication":"Metastatic NSCLC after platinum with PD-1 or docetaxel"},{"region":"US","year":2026,"indication":"Locally advanced pancreatic cancer with chemotherapy"},{"region":"EU","year":2015,"indication":"CE mark (GBM); pancreatic and NSCLC CE marks 2024-26"}],"mechanismSteps":["Arrays on the skin deliver alternating electric fields at 150-200 kHz","Fields penetrate tissue and act on polar molecules in dividing cells","Tubulin and septin alignment during mitosis is disrupted","Cytokinesis fails; daughter cells die or undergo immunogenic stress","Non-dividing normal cells are largely unaffected"],"dosing":{"route":"Wearable transducer arrays on the skin","schedule":"Continuous, at least 18 hours per day; arrays changed 2-3 times weekly","modifications":"Reduce wear time for skin toxicity; topical steroids","monitoring":"Skin under arrays; compliance (usage reports)","source":"https://www.optune.com"},"toxicity":[{"event":"Skin irritation under arrays","note":"EF-14: ~52% any grade, 2% grade 3"},{"event":"Headache"},{"event":"Malaise"},{"event":"Muscle twitching"}],"access":[{"country":"US","listPrice":"~$21,000 per month rental (Novocure disclosed pricing, US)","reimbursement":"Medicare covers Optune Gio for newly diagnosed glioblastoma (LCD); Optune Lua and Pax coverage evolving","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: not recommended for glioblastoma (2018 assessment); reassessment pending","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2011-04-08","type":"approval","region":"US","note":"PMA approval, recurrent glioblastoma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2015-10-05","type":"approval","region":"US","note":"Newly diagnosed glioblastoma with temozolomide (EF-14)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2019-05-23","type":"approval","region":"US","note":"Humanitarian device exemption, malignant pleural mesothelioma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-10-15","type":"approval","region":"US","note":"Optune Lua, metastatic NSCLC after platinum (LUNAR)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q1","type":"approval","region":"US","note":"Optune Pax, locally advanced pancreatic cancer with chemotherapy (PANOVA-3)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"or502","kind":"drug","name":"OR502","aka":[],"tldr":"OR502 is a monoclonal antibody from OncoResponse, Inc., in registered phase 2 trials for metastatic cancer, melanoma, non-small-cell lung cancer.","summary":"OR502 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by OncoResponse, Inc., in metastatic cancer, melanoma, non-small-cell lung cancer, small-cell lung cancer. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of OR502","url":"https://clinicaltrials.gov/search?intr=OR502"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer","melanoma","nsclc","sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["oncoresponse"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06090266"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"OR502","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"oregovomab","kind":"drug","name":"Oregovomab","aka":["MAb-B43.13","Monoclonal antibody B43.13"],"tldr":"Oregovomab is an experimental monoclonal antibody from CanariaBio in phase 3 trials for ovarian cancer, with its target not yet stated publicly.","summary":"Oregovomab is a monoclonal antibody developed by CanariaBio. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: 2 mg, dissolved in 2 mL of 0.9% Sodium Chloride Injection USP, then added to 50 mL of Sodium Chloride Injection USP infused over 20 ± 5 minutes. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT04498117 (Oregovomab Plus Chemo in Newly Diagnosed Patients With Advanced Epithelial Ovarian Cancer Following Optimal Debulking Surgery), in ovarian cancer. The largest, NCT04498117, plans to enrol 615 participants (actual) with primary completion expected 2026-12-26. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Oregovomab","url":"https://clinicaltrials.gov/search?intr=Oregovomab"},{"label":"Sponsor page","url":"https://www.canariabio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["canariabio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04498117","nct05605535","nct04938583","nct05335993"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"orelabrutinib","kind":"drug","name":"Orelabrutinib","aka":[],"tldr":"Orelabrutinib is InnoCare's BTK-blocking pill for chronic lymphocytic leukaemia and mantle cell lymphoma, approved in China in 2020.","summary":"Approved by the NMPA in December 2020 for relapsed or refractory chronic lymphocytic leukaemia or small lymphocytic lymphoma and for relapsed or refractory mantle cell lymphoma, with relapsed or refractory marginal zone lymphoma added in 2023, the first approval for any BTK inhibitor in that disease. It competes with zanubrutinib and ibrutinib in China's NRDL and is being tested in primary central nervous system lymphoma and, outside oncology, in multiple sclerosis.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Orelabrutinib","links":[{"label":"InnoCare Pharma","url":"https://www.innocarepharma.com/en"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["cll","mantle-cell-lymphoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["btk"],"drugs":[],"companies":["innocare"],"institutions":[],"pathways":["bcr-signalling"],"terms":[],"trials":["nct06363994","nct06082102","nct04014205"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Yinuokai","code":"ICP-022","modality":"Small-molecule covalent BTK inhibitor (second generation)","mechanism":"Irreversible, highly selective covalent inhibitor of BTK with minimal off-target kinase activity, designed for once-daily dosing and central nervous system penetration.","approvals":[{"region":"China","year":2020,"indication":"Relapsed or refractory CLL/SLL; relapsed or refractory mantle cell lymphoma"},{"region":"China","year":2023,"indication":"Relapsed or refractory marginal zone lymphoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"orteronel","kind":"drug","name":"Orteronel","aka":["TAK-700"],"tldr":"A more selective version of abiraterone that was meant to avoid the need for steroids. It delayed the cancer on scans but did not help men live longer, and was abandoned.","summary":"Orteronel is a non-steroidal, partially selective inhibitor of CYP 17,20-lyase. The selectivity was the point: abiraterone inhibits both the 17-alpha-hydroxylase and the 17,20-lyase activities of CYP17, which is why it raises mineralocorticoids and has to be given with a corticosteroid. A lyase-selective inhibitor should avoid that.\n\nELM-PC 4 randomised 1,560 chemotherapy-naive men with metastatic castration-resistant prostate cancer at 324 centres in 43 countries to orteronel 400 mg with prednisone twice daily or placebo with prednisone. Median radiographic progression-free survival was 13.8 months against 8.7, hazard ratio 0.71 (95 percent confidence interval 0.63 to 0.80, p<0.0001), a large and unambiguous effect. Median overall survival was 31.4 against 29.5 months, hazard ratio 0.92 (0.79 to 1.08, p=0.31), which is nothing. The companion trial in men after docetaxel, ELM-PC 5, also missed overall survival.\n\nGrade 3 or worse increases in lipase (17 against 2 percent) and amylase (10 against 1 percent) were the characteristic toxicities, along with fatigue and pulmonary embolism. Millennium, a Takeda subsidiary, stopped developing the drug. Orteronel is the reason the field treats radiographic progression-free survival in prostate cancer with suspicion as a surrogate for survival.","status":"withdrawn","asOf":"2026-09-25","links":[{"label":"ELM-PC 4 (Lancet Oncology 2015)","url":"https://doi.org/10.1016/S1470-2045(15)70027-6"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":[],"targets":["cyp17a1"],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":["arpi","castration-resistance"],"trials":["elm-pc-4-orteronel"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"TAK-700","modality":"small molecule","mechanism":"Non-steroidal, partially selective inhibitor of CYP 17,20-lyase, reducing extragonadal androgen synthesis.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ose2101","kind":"drug","name":"OSE2101","aka":[],"tldr":"OSE2101 is an experimental cancer vaccine from OSE Immunotherapeutics in phase 3 trials for non-small-cell lung cancer, aimed at HER2 and CEACAM5.","summary":"OSE2101 is a cancer vaccine developed by OSE Immunotherapeutics. Its targets are HER2, CEACAM5 and TP53 (the sponsor names P53, HER-2, CEA, MAGE-2, MAGE-3 (HLA-A2-restricted epitopes) plus PADRE pan-HLA-DR epitope). The sponsor states: A peptide vaccine composed of nine HLA-A2-restricted tumour-associated antigen epitopes plus one pan-HLA-DR binding epitope, emulsified in Montanide ISA 51 adjuvant, that activates tumour-specific T lymphocytes. ClinicalTrials.gov describes the intervention as: OSE2101 is a peptidic cancer vaccine composed of nine epitopes restricted to HLA-A2 phenotype targeting the tumour associated antigens of P53, HER-2, CEA, MAGE-2 and MAGE-3, and one pan-HLA DR binding epitope (PADRE), all epitopes emulsified. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06472245 (Trial of Therapeutic Cancer Vaccine OSE2101 in Patients With Non-Small Cell Lung Cancer and Secondary Resistance to Immune Checkpoint Inhibitor), in non-small-cell lung cancer. The largest, NCT06472245, plans to enrol 363 participants with primary completion expected 2027-12-15. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of OSE2101","url":"https://clinicaltrials.gov/search?intr=OSE2101"},{"label":"Sponsor pipeline page","url":"https://www.ose-immuno.com/en/pipeline/tedopi/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["her2","ceacam5","tp53"],"drugs":[],"companies":["ose-immunotherapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06472245","nct05751798"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"vaccine","mechanism":"A peptide vaccine composed of nine HLA-A2-restricted tumour-associated antigen epitopes plus one pan-HLA-DR binding epitope, emulsified in Montanide ISA 51 adjuvant, that activates tumour-specific T lymphocytes.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"osilodrostat","kind":"drug","name":"Osilodrostat","aka":["LCI699"],"tldr":"Osilodrostat is a twice-daily tablet that stops the adrenal glands making cortisol. It controls Cushing's disease caused by a pituitary tumour when surgery has failed or is not possible, normalising cortisol in most patients.","summary":"Osilodrostat (LCI699) is a potent oral steroidogenesis inhibitor developed by Novartis and sold by Recordati. In the phase 3 LINC 3 trial in Cushing's disease, most patients achieved normal urinary free cortisol, and withdrawal to placebo showed the effect was drug-dependent. The European Commission approved it in January 2020 for endogenous Cushing's syndrome and the FDA in March 2020 for Cushing's disease in adults for whom pituitary surgery is not an option or has not been curative.\n\nIt is given twice daily with dose titration against cortisol; adrenal insufficiency, precursor accumulation with hypokalaemia and hypertension, and androgen excess in women are the main effects to watch. Metyrapone is the older drug of the same class. The pituitary tumours page names both among the steroidogenesis inhibitors used after surgery.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Osilodrostat","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Osilodrostat"}],"tags":["subtype-drugs-wave"],"related":["metyrapone"],"cancers":["pituitary-tumours"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["recordati"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02697734","nct01331239"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Isturisa","modality":"Oral 11-beta-hydroxylase (CYP11B1) inhibitor","mechanism":"Blocks 11-beta-hydroxylase, the final enzyme in cortisol synthesis in the adrenal cortex, so cortisol falls whatever the pituitary tumour's ACTH output; it also inhibits aldosterone synthase.","approvals":[{"region":"EU","year":2020,"indication":"Endogenous Cushing's syndrome in adults"},{"region":"US","year":2020,"indication":"Cushing's disease in adults for whom pituitary surgery is not an option or has not been curative"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"osimertinib","kind":"drug","name":"Osimertinib","aka":[],"tldr":"Osimertinib (Tagrisso) is the standard pill for EGFR-mutant lung cancer, now also given after surgery and with chemotherapy or after chemoradiation.","summary":"Osimertinib is an irreversible third-generation EGFR tyrosine kinase inhibitor that spares wild-type EGFR, covers the T790M resistance mutation and penetrates the brain, taken as 80 mg once daily. It is the standard first-line pill for EGFR-mutant NSCLC after FLAURA showed an overall survival benefit, and its reach has widened: ADAURA (adjuvant, up to 3 years, OS HR 0.49 with 5-year OS 88% versus 78%), FLAURA2 (with chemotherapy, PFS 25.5 versus 16.7 months and OS HR 0.77 in 2025) and LAURA (after chemoradiation in stage III). Diarrhoea and rash affect over half of patients but are rarely severe; interstitial lung disease (4%) needs vigilance. It is now challenged first line by amivantamab plus lazertinib (MARIPOSA), and whether every patient should receive added chemotherapy is unsettled. For a newcomer: the drug that made EGFR-mutant lung cancer a long-term treatable disease.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Osimertinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Osimertinib"},{"label":"NICE TA654: osimertinib for untreated EGFR mutation-positive non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta654"},{"label":"NICE TA1043: osimertinib for adjuvant treatment of EGFR mutation-positive non-small-cell lung cancer after complete tumour resection","url":"https://www.nice.org.uk/guidance/ta1043"},{"label":"NICE TA1060: osimertinib with pemetrexed and platinum-based chemotherapy for untreated EGFR mutation-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1060"},{"label":"NICE TA1156: osimertinib for EGFR mutation-positive unresectable locally advanced non-small-cell lung cancer after platinum-based chemoradiotherapy","url":"https://www.nice.org.uk/guidance/ta1156"}],"tags":[],"related":["egfr-exon-19-deletion","egfr-l858r","egfr-t790m"],"cancers":["nsclc","egfr-mutant-nsclc","resectable-nsclc","stage-iii-unresectable-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["adaura","flaura2","nct06350097","nct04351555","nct06838273","nct05120349","nct06670196","nct02411448","nct07640789","nct06417814","nct07642024","nct04765059","nct04606771","nct06498986","nct05801029","nct05880706","nct07229729","nct07622186","nct07079475","nct04868877","nct05816252","nct07329322","nct05546866","nct07206498","nct04665206","nct07669779","nct03944772","nct06574347","nct04486833","nct05785741","nct03778229","nct06895928","nct05261399","nct03381274","nct06194448","nct03040973","nct03833154","nct07295821","nct05948813","nct05629234","nct06970639","nct06618287","nct06391944","nct05526755","nct06868485","flaura","aura3","laura"],"people":[],"bottlenecks":[],"keyPapers":["paper-flaura-nejm-2018","paper-flaura2-long-term-safety-lung-cancer-2026","paper-mariposa-nejm-2024"],"journals":[],"dependsOn":[],"notes":[],"brand":"Tagrisso","modality":"Small-molecule kinase inhibitor (EGFR)","mechanism":"Irreversible third-generation EGFR TKI sparing wild-type EGFR; active against T790M; CNS penetrant.","approvals":[{"region":"US","year":2015,"indication":"EGFR T790M NSCLC"},{"region":"US","year":2018,"indication":"First-line EGFR-mutant NSCLC"},{"region":"US","year":2020,"indication":"Adjuvant EGFR-mutant NSCLC"},{"region":"England (NICE)","year":2020,"indication":"Untreated locally advanced or metastatic EGFR mutation-positive non-small-cell lung cancer","note":"TA654, published 14 October 2020, recommends osimertinib within its marketing authorisation subject to the commercial arrangement."},{"region":"England (NICE)","year":2020,"indication":"EGFR T790M mutation-positive locally advanced or metastatic non-small-cell lung cancer after a first-line EGFR inhibitor","note":"TA653, published 14 October 2020, subject to the commercial arrangement."},{"region":"England (NICE)","year":2025,"indication":"Adjuvant treatment of stage IB to IIIA EGFR exon 19 deletion or L858R non-small-cell lung cancer after complete resection","note":"TA1043, published 26 February 2025, recommends it only if osimertinib is stopped at 3 years, or earlier on recurrence or unacceptable toxicity. It replaced a managed access agreement whose data, from the ADAURA trial and from NHS use in England, were reviewed in the appraisal."},{"region":"England (NICE)","year":2025,"indication":"Untreated advanced EGFR mutation-positive non-small-cell lung cancer, with pemetrexed and platinum-based chemotherapy","note":"TA1060, published 8 May 2025, subject to the commercial arrangement (FLAURA2)."},{"region":"England (NICE)","year":2026,"indication":"Unresectable stage III EGFR mutation-positive non-small-cell lung cancer that has not progressed after platinum-based chemoradiotherapy","note":"TA1156, published 21 May 2026 (LAURA); must be funded in England within 90 days of publication."}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of mutant EGFR (including T790M)","Phosphorylation of downstream substrates stops","Covalent bond to Cys797 blocks ATP binding; wild-type EGFR is spared","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"80 mg once daily with or without food; adjuvant up to 3 years","modifications":"Reduce to 40 mg for QTc >500 ms or grade 3 toxicity; permanently discontinue for ILD","monitoring":"ECG and electrolytes in at-risk patients; LVEF; respiratory symptoms","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7"},"toxicity":[{"event":"Diarrhoea","anyGradePct":58,"grade3PlusPct":2.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"},{"event":"Rash","anyGradePct":58,"grade3PlusPct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"},{"event":"Dry skin","anyGradePct":36,"grade3PlusPct":0.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"},{"event":"Nail toxicity","anyGradePct":35,"grade3PlusPct":0.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"},{"event":"Stomatitis","anyGradePct":32,"grade3PlusPct":0.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"},{"event":"Fatigue","anyGradePct":21,"grade3PlusPct":1.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"},{"event":"Decreased appetite","anyGradePct":20,"grade3PlusPct":2.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"},{"event":"Interstitial lung disease","anyGradePct":4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"0.4% fatal; higher after chemoradiation"},{"event":"Cardiomyopathy","anyGradePct":3.8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"},{"event":"QTc >500 ms","anyGradePct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5e81b4a7-b971-45e1-9c31-29cea8c87ce7","note":"FLAURA"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.astrazeneca-us.com/medicines/access-360","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended first-line (TA654), adjuvant (TA761), and after chemoradiation","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"},{"country":"CN","reimbursement":"NRDL listed since 2019 with major price cut","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2015-11-13","type":"accelerated-approval","region":"US","note":"Accelerated approval, EGFR T790M NSCLC after TKI","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Metastatic EGFR T790M mutation-positive NSCLC, as detected by an FDA-approved test, that progressed on or after EGFR TKI therapy"},{"date":"2017-03-30","type":"conversion","region":"US","note":"Full approval (AURA3)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Metastatic EGFR T790M mutation-positive NSCLC, as detected by an FDA-approved test, that progressed on or after EGFR TKI therapy"},{"date":"2018-04-18","type":"approval","region":"US","note":"First-line EGFR-mutant NSCLC (FLAURA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-12-18","type":"approval","region":"US","note":"Adjuvant EGFR-mutant NSCLC after resection (ADAURA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-02-16","type":"approval","region":"US","note":"First-line with chemotherapy (FLAURA2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-09-25","type":"approval","region":"US","note":"Unresectable stage III after chemoradiation (LAURA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"oxaliplatin","kind":"drug","name":"Oxaliplatin","aka":[],"tldr":"The platinum drug that works in bowel cancer where cisplatin does not, the 'OX' in FOLFOX and CAPOX; its cost is nerve damage in hands and feet.","summary":"Approved EU 1996, US 2002 (second line, then adjuvant colon after MOSAIC 2004). FOLFOX/CAPOX backbone in colorectal, gastro-oesophageal (FLOT), pancreatic (FOLFIRINOX), biliary (second line), appendiceal and neuroendocrine (CAPTEM alternatives). IDEA (2018) showed 3 months of CAPOX suffices for low-risk stage III colon cancer, limiting neuropathy.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Oxaliplatin","links":[{"label":"MOSAIC (NEJM 2004)","url":"https://doi.org/10.1056/NEJMoa032709"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=oxaliplatin"}],"tags":["gap-fill","generic","who-essential"],"related":[],"cancers":["colorectal","gastric","esophageal","pancreatic","appendiceal","mucinous-ovarian-cancer"],"sections":[],"technologies":["platinum","cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["rapido","nct05300269","nct07554521","nct05980481","nct06469944","nct06155383","nct07415031","nct06901531","nct05771480","nct06247956","nct06951503","nct06771622","nct05945823","nct07056777","nct06662786","nct03505320","nct05329766","nct05144854","nct07582315","nct07606599","nct07431281","nct03478488","nct07315750","nct04379596","nct06806033","nct06780111","spotlight-glow","nct06854445","mountaineer","nct07390383","nct06825494","nct07238283","nct07522151","nct07730021","nct07283367","nct05239741","nct06741644","nct05671822","nct05859750","nct06256328","nct04919226","nct04725994","mosaic","idea-collaboration","prodige-7","colopec","prophylochip"],"people":[],"bottlenecks":[],"keyPapers":["paper-mosaic-oxaliplatin-adjuvant-colon-nejm-2004"],"journals":[],"dependsOn":[],"notes":[],"brand":"Eloxatin","modality":"Third-generation platinum (DACH-platinum)","mechanism":"Forms bulky DACH-platinum DNA adducts poorly recognised by mismatch repair, giving activity in cisplatin-resistant colorectal cancer; causes acute cold-triggered and cumulative sensory neuropathy.","approvals":[{"region":"EU","year":1996,"indication":"Metastatic colorectal cancer"},{"region":"US","year":2002,"indication":"Metastatic colorectal cancer with 5-FU/LV"},{"region":"US","year":2004,"indication":"Adjuvant stage III colon cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2002-08-09","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with infusional 5-FU/LV for metastatic color or rectal carcinoma that progressed during or within 6 months of completion of first-line therapy with bolus 5 -FU/LV and irinotectan"},{"date":"2004-01-09","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2002 converted to traditional approval 1.4 years after it was granted.","indication":"In combination with infusional 5-FU/LV for metastatic color or rectal carcinoma that progressed during or within 6 months of completion of first-line therapy with bolus 5 -FU/LV and irinotectan"}]},{"id":"paclitaxel","kind":"drug","name":"Paclitaxel / nab-paclitaxel","aka":[],"tldr":"A microtubule poison discovered in the Pacific yew tree, among the most used chemotherapies in breast, lung, and ovarian cancer.","summary":"Paclitaxel is a taxane that binds and stabilises microtubules, freezing the mitotic spindle so dividing cells cannot complete mitosis. Discovered in the bark of the Pacific yew, it was approved in 1992 for ovarian cancer and is now among the most used chemotherapies in breast, lung, ovarian and pancreatic cancer, at 80 mg/m2 weekly or 175 mg/m2 every 3 weeks. Weekly paclitaxel is a backbone of neoadjuvant TNBC therapy. Nab-paclitaxel binds the drug to albumin, removing the Cremophor solvent that causes hypersensitivity, so it needs no steroid premedication and is given at 100 to 125 mg/m2 on days 1, 8 and 15; it partners with immunotherapy (IMpassion130) and, in 2026, with relacorilant in ovarian cancer and TTFields in pancreatic cancer. Neuropathy and neutropenia are the main adverse events. For a newcomer: a plant-derived drug that jams the machinery of cell division.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Paclitaxel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Paclitaxel"}],"tags":[],"related":["acupuncture-chemotherapy-neuropathy"],"cancers":["tnbc","breast-hr-positive","nsclc","ovarian","pancreatic","angiosarcoma","cup-favourable-subsets","metastatic-anal-cancer"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06212752","nct06828354","nct06767527","nct07044336","nct06340568","nct06692738","nct06584032","nct04379635","nct07216703","nct07491445","nct06699212","nct05800015","nct07805954","nct06356311","nct07168200","nct06072781","nct03390686","nct05654454","nct05116462","nct06989112","nct03800134","nct06422143","nct06712316","nct07604766","nct06486441","nct06952504","nct06132958","nct03164616","nct06619236","nct05722015","nct06123884","nct04956692","nct05984277","nct06915025","nct07564141","nct06703398","nct06445062","nct05652686","nct06535607","nct06463028","nct01081951","nct05482893","nct05283226","nct04843098","nct06448754","nct04083599","nct06946797","nct05932212","nct06646055","nct06693336","nct07049055","nct07680764","nct06731907","nct05605535","nct06922591","nct05904379","nct05739981","nct06522828","nct03775486","nct06943820","nct07255404","nct07714668","nct07221474","nct05585320","nct05431270","nct07229729","nct05635708","nct06434610","nct06996782","nct06161441","nct03393884","nct05669482","nct04736173","nct07042802","nct06727565","nct04938583","nct04644068","nct07310784","nct07563738","nct06840002","nct07102381","actuate-1801","nct06449209","nct07259590","nct06843447","nct05247684","nct07322094","nct06758557","nct05824975","nct06047379","nct04931342","nct07223047","nct05742607","nct05054439","nct05814354"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Taxol / Abraxane","modality":"Cytotoxic chemotherapy (taxane)","mechanism":"Stabilises microtubules, blocking mitosis.","approvals":[{"region":"US","year":1992,"indication":"Ovarian cancer (now broad)"}],"mechanismSteps":["Paclitaxel binds β-tubulin in microtubules","Microtubules are stabilised and cannot disassemble","Mitotic spindle dynamics fail; cells arrest in G2/M","Prolonged arrest triggers apoptosis","Rapidly dividing tumour cells (and hair follicles, marrow) are most affected"],"dosing":{"route":"IV infusion","schedule":"80 mg/m² weekly (breast, with carboplatin in TNBC) or 175 mg/m² every 3 weeks; nab-paclitaxel 100-125 mg/m² days 1, 8, 15 without premedication","modifications":"Hold for grade ≥3 neuropathy or neutropenia <1,500/µL","monitoring":"Blood counts, neuropathy, hypersensitivity (solvent-based formulation requires steroid/antihistamine premedication)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Neutropenia"},{"event":"Peripheral neuropathy"},{"event":"Alopecia"},{"event":"Myalgia/arthralgia"},{"event":"Hypersensitivity reactions (Cremophor)"},{"event":"Nausea"}],"access":[{"country":"US","reimbursement":"Medicare Part B; generic paclitaxel and nab-paclitaxel available","generic":true,"source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NHS standard; generic","generic":true,"asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"1992-12-29","type":"approval","region":"US","note":"Refractory ovarian cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"1994-04-13","type":"approval","region":"US","note":"Metastatic breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2005-01-07","type":"approval","region":"US","note":"nab-Paclitaxel (Abraxane) for metastatic breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2013-09-06","type":"approval","region":"US","note":"nab-Paclitaxel with gemcitabine for metastatic pancreatic cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-04","type":"approval","region":"US","note":"First nab-paclitaxel generics","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"pacritinib","kind":"drug","name":"Pacritinib","aka":[],"tldr":"The JAK inhibitor for myelofibrosis patients whose platelet counts are too low for ruxolitinib.","summary":"Pacritinib inhibits JAK2 and FLT3 while sparing JAK1, and also blocks IRAK1 and ACVR1, the receptor that drives hepcidin production; because it spares JAK1 it causes less myelosuppression and can be used when platelet counts are too low for ruxolitinib. In PERSIST-2 it produced spleen and symptom responses in myelofibrosis patients with platelets below 100 x 10^9/L, including those below 50, and it received accelerated approval in 2022 for intermediate or high-risk myelofibrosis with platelets below 50 x 10^9/L. ACVR1 inhibition lowers hepcidin and may improve anaemia. Diarrhoea is the main adverse event. PACIFICA is the confirmatory trial needed to convert accelerated approval into full approval, and its role in patients with higher platelet counts is unsettled. For a newcomer: the myelofibrosis JAK inhibitor for people whose platelets are dangerously low.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pacritinib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pacritinib"}],"tags":["gap-fill"],"related":[],"cancers":["myeloproliferative-neoplasms","primary-myelofibrosis"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["jak2","flt3","acvr1"],"drugs":[],"companies":["sobi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07033598"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vonjo","modality":"Small-molecule JAK2/IRAK1/ACVR1 inhibitor","mechanism":"Inhibits JAK2 and FLT3 while sparing JAK1, plus IRAK1 and ACVR1 (hepcidin pathway), allowing use with very low platelets.","approvals":[{"region":"US","year":2022,"indication":"Intermediate/high-risk myelofibrosis with platelets <50×10⁹/L"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"padeliporfin","kind":"drug","name":"Padeliporfin","aka":["WST11","Padeliporfin di-potassium","Vascular-targeted photodynamic therapy (VTP)"],"tldr":"Tookad is a light-activated drug for men with low-risk prostate cancer in one side of the prostate. It is infused and then switched on with laser fibres placed in the gland, destroying the cancer-bearing tissue as an alternative to watchful waiting.","summary":"Padeliporfin was authorised in the EU in November 2017 as monotherapy for previously untreated, unilateral, low-risk prostate adenocarcinoma (stage T1c or T2a, Gleason 6, PSA 10 ng/mL or below) in men with a life expectancy of at least 10 years, on the PCM301 phase 3 trial in which vascular-targeted photodynamic therapy roughly halved progression and the need for radical therapy compared with active surveillance, at the cost of transient urinary and erectile symptoms. It is the only focal drug-device therapy for prostate cancer with a marketing authorisation; an FDA advisory committee questioned its benefit over surveillance and it is not approved in the US. Trials in intermediate-risk disease and in upper urinary tract urothelial cancer are ongoing. Dysuria, haematuria and erectile dysfunction are the main adverse effects and patients must avoid light exposure for 48 hours.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Padeliporfin","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/tookad"},{"label":"PCM301 (Lancet Oncology 2017)","url":"https://doi.org/10.1016/S1470-2045(16)30661-1"},{"label":"NICE TA546: padeliporfin for untreated localised prostate cancer, not recommended","url":"https://www.nice.org.uk/guidance/ta546"}],"tags":["ema-list"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["active-surveillance"],"targets":[],"drugs":[],"companies":["steba-biotech"],"institutions":[],"pathways":[],"terms":["active-surveillance-term","prostate-focal-therapy","prostate-uk-drug-approvals"],"trials":["nct01310894","nct01875393","nct03315754"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["PCM301 randomised 413 men with low-risk prostate cancer to padeliporfin vascular-targeted photodynamic therapy or active surveillance and found disease progression at 24 months in 28 against 58 percent (adjusted hazard ratio 0.34) with a negative biopsy at 24 months in 49 against 14 percent. NICE TA546 nonetheless does not recommend it for untreated, unilateral, low-risk prostate cancer, on the reasoning that men with low-risk disease live as long under active surveillance and that radical and focal therapies carry long-term side effects."],"brand":"Tookad","modality":"Intravenous photosensitiser for vascular-targeted photodynamic therapy","mechanism":"Water-soluble bacteriochlorophyll derivative that stays in the circulation; activation by 753 nm laser light delivered through transperineal fibres generates oxygen radicals that occlude tumour vasculature and cause focal necrosis of the treated prostate lobe.","approvals":[{"region":"EU","year":2017,"indication":"Unilateral, low-risk, previously untreated prostate adenocarcinoma in men with life expectancy of at least 10 years (Tookad VTP)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pafolacianine","kind":"drug","name":"Pafolacianine","aka":[],"tldr":"An injected dye that makes ovarian and lung cancer deposits glow during surgery so surgeons can find lesions they would otherwise miss.","summary":"Pafolacianine is a folate analogue conjugated to an indocyanine near-infrared dye. Injected before surgery, it binds folate receptor alpha, which is overexpressed on ovarian and lung adenocarcinoma cells, and fluoresces under NIR light so surgeons can see tumour deposits they would otherwise miss. In the phase 3 ovarian trial (2021) additional lesions were found in 27 to 33% of patients, and in ELUCIDATE in lung cancer (2022) 24% of patients had otherwise-undetected lesions or positive margins revealed. The FDA approved it in November 2021 for intraoperative imaging of ovarian cancer and in December 2022 for lung lesions; it requires NIR imaging systems in theatre, and folate-containing supplements are withheld beforehand. Whether finding more lesions improves survival is unproven. In short, pafolacianine makes cancer glow so the surgeon can find it.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pafolacianine","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pafolacianine"}],"tags":["gap-fill","diagnostic"],"related":[],"cancers":["ovarian","nsclc"],"sections":[],"technologies":["fluorescence-guided-surgery","optical-imaging"],"targets":["folr1"],"drugs":[],"companies":["on-target-laboratories"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04241315","nct07039526","nct06434909"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cytalux","modality":"Folate receptor-targeted near-infrared fluorescent imaging agent","mechanism":"Folate analogue conjugated to an indocyanine dye; binds folate receptor alpha on ovarian and lung adenocarcinoma cells and fluoresces under NIR light during surgery.","approvals":[{"region":"US","year":2021,"indication":"Intraoperative imaging of ovarian cancer lesions in adults"},{"region":"US","year":2022,"indication":"Intraoperative identification of lung lesions"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"paige-prostate","kind":"drug","name":"Paige Prostate Detect","aka":[],"tldr":"The first AI for reading biopsy slides authorised by the FDA, which points pathologists to prostate cancer they might otherwise miss.","summary":"Paige Prostate received FDA De Novo authorisation on 21 September 2021 (DEN200080), the first artificial-intelligence software for pathology. In the FDA reader study, pathologists using the software improved cancer detection on prostate biopsy slides, with the agency reporting a 7.3% improvement in detection and a reduction in false negatives. It runs on scanned whole-slide images and highlights areas of concern rather than making the diagnosis. Paige, a Memorial Sloan Kettering spin-out, went on to build the Virchow foundation models with Microsoft, and the software is CE-marked in Europe.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA De Novo DEN200080","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpmn/denovo.cfm?id=DEN200080"},{"label":"FDA press release (2021) (archived copy)","url":"https://web.archive.org/web/20250427051345/https://www.fda.gov/news-events/press-announcements/fda-authorizes-software-can-help-identify-prostate-cancer"}],"tags":["test"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["digital-pathology-ai","whole-slide-scanners","pathology-foundation-model"],"targets":[],"drugs":[],"companies":["paige"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: nothing changes for the patient directly; the pathologist still makes the diagnosis, with a second pair of algorithmic eyes reducing missed small cancers."],"brand":"Paige Prostate","modality":"AI digital pathology detection software (prostate biopsy)","mechanism":"Deep-learning model trained on tens of thousands of whole-slide images flags biopsy slides and regions suspicious for cancer for pathologist review.","approvals":[{"region":"US","year":2021,"indication":"Aid to pathologists in detecting areas suspicious for cancer on prostate needle biopsy slides (De Novo)"},{"region":"EU","year":2019,"indication":"CE mark"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"palacaparib","kind":"drug","name":"Palacaparib","aka":[],"tldr":"Palacaparib is an experimental small-molecule drug from AstraZeneca in phase 2 trials for ovarian cancer, non-small-cell lung cancer and endometrial cancer, aimed at PARP.","summary":"Palacaparib (AZD9574) is a small-molecule drug developed by AstraZeneca. Its target is PARP (the sponsor names PARP). The sponsor states: An oral PARP inhibitor that blocks PARP enzyme activity, preventing DNA repair in cancer cells, particularly tumours with homologous recombination deficiency. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in ovarian cancer, non-small-cell lung cancer and endometrial cancer. The largest, NCT05797168, plans to enrol 602 participants with primary completion expected 2028-08-04. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Palacaparib","url":"https://clinicaltrials.gov/search?intr=AZD9574"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian","nsclc","endometrial"],"sections":[],"technologies":[],"targets":["parp"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07181161","nct05797168","nct05123482","nct07590934","nct05417594"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AZD9574","modality":"small molecule","mechanism":"An oral PARP inhibitor that blocks PARP enzyme activity, preventing DNA repair in cancer cells, particularly tumours with homologous recombination deficiency.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"palbociclib","kind":"drug","name":"Palbociclib","aka":[],"tldr":"Palbociclib was the first CDK4/6 inhibitor (2015), and in 2026 became the first approved as maintenance in HER2-positive, hormone-positive breast cancer.","summary":"Palbociclib is a reversible CDK4/6 inhibitor that halts the cell cycle at G1, given as 125 mg daily for 21 days of a 28-day cycle with an aromatase inhibitor or fulvestrant. It was the first CDK4/6 inhibitor, approved in 2015 for HR-positive, HER2-negative advanced breast cancer on PALOMA-2 and PALOMA-3, which improved progression-free but not overall survival; the adjuvant trials PALLAS and PENELOPE-B were negative. In PATINA (SABCS 2024; approved Q2 2026) adding palbociclib to anti-HER2 plus endocrine maintenance in HR-positive, HER2-positive metastatic disease extended progression-free survival by about 15 months. Pfizer markets it; neutropenia (80%) is frequent but rarely febrile. Why it lacks the survival benefit seen with ribociclib and abemaciclib is debated. For a newcomer: the drug that opened the CDK4/6 class, now finding a new home in HER2-positive disease.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Palbociclib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Palbociclib"}],"tags":[],"related":["pr-status"],"cancers":["breast-hr-positive","breast-her2-positive"],"sections":[],"technologies":["cdk46-inhibitor"],"targets":["cdk4-6"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03709680","nct05468697","nct04553133","nct04606446","nct05867251","nct04567420","nct05501886","nct05768139","nct06065748","nct06736704","nct07002177","nct05563220","nct04711252","nct06757634","nct04862663","nct05909397","nct06380751","nct06760637","nct05226871"],"people":["massimo-cristofanilli"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ibrance","modality":"Small-molecule CDK4/6 inhibitor","mechanism":"Reversible CDK4/6 inhibitor.","approvals":[{"region":"US","year":2015,"indication":"HR+/HER2- advanced breast cancer with endocrine therapy"},{"region":"US","year":2026,"indication":"HR+/HER2+ advanced breast cancer maintenance with anti-HER2 and endocrine therapy"}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of CDK4 and CDK6","Phosphorylation of downstream substrates stops","RB stays unphosphorylated and holds E2F; cells arrest in G1","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"125 mg once daily for 21 days, then 7 days off (28-day cycle), with aromatase inhibitor or fulvestrant","modifications":"Reduce to 100 then 75 mg for grade 3 neutropenia with fever or grade 4","monitoring":"Complete blood count before each cycle and on day 15 of the first two cycles; ILD symptoms","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce"},"toxicity":[{"event":"Neutropenia","anyGradePct":80,"grade3PlusPct":66,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Infections","anyGradePct":60,"grade3PlusPct":7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Leukopenia","anyGradePct":39,"grade3PlusPct":25,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Fatigue","anyGradePct":37,"grade3PlusPct":2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Nausea","anyGradePct":35,"grade3PlusPct":1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Alopecia","anyGradePct":33,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Stomatitis","anyGradePct":30,"grade3PlusPct":1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Diarrhoea","anyGradePct":26,"grade3PlusPct":1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Anaemia","anyGradePct":24,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"},{"event":"Rash","anyGradePct":18,"grade3PlusPct":1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=fecbdd7d-b729-41b5-9872-231b8fe104ce","note":"PALOMA-2 with letrozole"}],"access":[{"country":"US","reimbursement":"Medicare Part D; generic entry expected 2027 in the US","assistance":"https://www.pfizeroncologytogether.com","generic":false,"source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with aromatase inhibitor (TA495) and fulvestrant (TA619)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2015-02-03","type":"accelerated-approval","region":"US","note":"Accelerated approval with letrozole: first CDK4/6 inhibitor (PALOMA-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with letrozole for postmenopausal women with ER-positive, HER2-negative advanced breast cancer as initial endocrine-based therapy for metastatic disease"},{"date":"2016-02-19","type":"approval","region":"US","note":"With fulvestrant after endocrine therapy (PALOMA-3)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2017-03-31","type":"conversion","region":"US","note":"Full approval (PALOMA-2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with letrozole for postmenopausal women with ER-positive, HER2-negative advanced breast cancer as initial endocrine-based therapy for metastatic disease"},{"date":"2019-04-04","type":"approval","region":"US","note":"Men with HR+/HER2- breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-06-24","type":"approval","region":"US","note":"Maintenance in HR+/HER2+ advanced breast cancer with anti-HER2 and endocrine therapy (PATINA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-palbociclib-trastuzumab-or-without-pertuzumab-and-endocrine-therapy-maintenance"},{"date":"2026-07-14","type":"approval","region":"US","note":"Gedatolisib with fulvestrant, with or without palbociclib, HR+/HER2- advanced breast cancer (VIKTORIA-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-gedatolisib-fulvestrant-or-without-palbociclib-hr-positive-her2-negative-locally"}]},{"id":"palifermin","kind":"drug","name":"Palifermin","aka":["Recombinant keratinocyte growth factor","rHuKGF"],"tldr":"Palifermin (Kepivance) is a growth factor injected for three days before and after high-dose chemotherapy and stem cell rescue to reduce the severe mouth ulcers (mucositis) that this treatment causes in people with blood cancers.","summary":"Palifermin was approved by the FDA in December 2004 to decrease the incidence and duration of severe oral mucositis in patients with haematological malignancies receiving myelotoxic therapy with autologous stem cell support, when the regimen is expected to cause WHO grade 3 or worse mucositis in most patients; in the pivotal randomised trial of 212 patients receiving total body irradiation-based conditioning, grade 3 to 4 mucositis fell from 98% to 63% and its duration from 9 to 3 days. The EU authorised Kepivance in 2005. Its safety in non-haematological malignancies is not established because KGF receptors are present on many epithelial tumours, and it is not given within 24 hours of chemotherapy. Rash, tongue thickening and taste change are the usual effects. Marketed by Sobi.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Palifermin","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=palifermin"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/palifermin"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/kepivance"}],"tags":["nci-list","supportive"],"related":[],"cancers":["multiple-myeloma","dlbcl","hodgkin-lymphoma"],"sections":[],"technologies":["autologous-stem-cell-transplant"],"targets":[],"drugs":[],"companies":["sobi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00434161","nct00101582","nct00109031"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Kepivance","modality":"Recombinant human keratinocyte growth factor","supportive":true,"mechanism":"Truncated recombinant KGF (FGF7) that binds the KGF receptor (FGFR2b) on epithelial cells of the mouth and gut, stimulating proliferation, differentiation and cytoprotection of the mucosa.","approvals":[{"region":"US","year":2004,"indication":"Reduction of severe oral mucositis in haematological malignancies with myelotoxic therapy and autologous stem cell support"},{"region":"EU","year":2005,"indication":"Same (Kepivance)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"palonosetron","kind":"drug","name":"Palonosetron","aka":["Posfrea"],"tldr":"Palonosetron (Aloxi) is a long-acting anti-sickness injection given once before chemotherapy; a single dose covers both the first day and the delayed nausea that follows.","summary":"Palonosetron was approved by the FDA in July 2003 for prevention of acute and delayed nausea and vomiting with moderately emetogenic chemotherapy and acute nausea and vomiting with highly emetogenic chemotherapy, on randomised trials against ondansetron and dolasetron in which a single 0.25 mg dose was non-inferior in the acute phase and superior over five days; post-operative nausea (2008) and a paediatric indication from one month of age (2014) followed. The EU authorised Aloxi in 2005. It is the 5-HT3 partner in the Akynzeo fixed combination with netupitant and is preferred by MASCC/ESMO guidelines for moderately emetogenic regimens when an NK1 antagonist is not used. Headache and constipation are the main adverse effects, with less QT effect than first-generation agents.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Palonosetron","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=palonosetron"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/palonosetronhydrochloride"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/aloxi"}],"tags":["nci-list","supportive"],"related":["netupitant-palonosetron","ondansetron"],"cancers":[],"sections":[],"technologies":["antiemetic-therapy"],"targets":[],"drugs":[],"companies":["helsinn","eisai"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06904235"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aloxi","modality":"Second-generation 5-HT3 receptor antagonist (antiemetic)","supportive":true,"mechanism":"High-affinity 5-HT3 antagonist with a 40-hour half-life that also triggers receptor internalisation and inhibits crosstalk with NK1 signalling, which is thought to explain its effect on delayed emesis.","approvals":[{"region":"US","year":2003,"indication":"Acute and delayed nausea and vomiting with moderately emetogenic chemotherapy; acute with highly emetogenic chemotherapy (paediatric from 1 month added 2014)"},{"region":"EU","year":2005,"indication":"Prevention of chemotherapy-induced nausea and vomiting (Aloxi)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"paltusotine","kind":"drug","name":"Paltusotine","aka":["CRN00808"],"tldr":"Paltusotine is the first somatostatin-type drug that works as a once-daily pill instead of a monthly injection. It was approved in 2025 for acromegaly and is being tested for the flushing and diarrhoea of carcinoid syndrome from small-bowel neuroendocrine tumours.","summary":"Paltusotine is Crinetics Pharmaceuticals' oral, non-peptide agonist of somatostatin receptor 2. In the phase 3 PATHFNDR trials in acromegaly it maintained IGF-1 control in patients switched from injected somatostatin analogues and achieved control in untreated patients, and the FDA approved it in September 2025 for the treatment of acromegaly in adults.\n\nCarcinoid syndrome depends on the same receptor: the phase 3 CAREFNDR trial is testing paltusotine against placebo for flushing and bowel frequency in patients with carcinoid syndrome from neuroendocrine tumours. The small intestinal neuroendocrine tumour page names it, with a subcutaneous octreotide depot, among the symptom-control options in development after the injected analogues.","status":"approved","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["octreotide-lanreotide"],"cancers":["small-intestinal-net"],"sections":[],"technologies":[],"targets":["sstr2"],"drugs":[],"companies":["crinetics-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Palsonify","modality":"Oral non-peptide somatostatin receptor 2 agonist","mechanism":"A small molecule that selectively activates somatostatin receptor 2, the receptor through which octreotide and lanreotide suppress growth hormone and the hormones that cause carcinoid syndrome, but taken as a once-daily tablet rather than an injection.","approvals":[{"region":"US","year":2025,"indication":"Acromegaly in adults"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pamidronate","kind":"drug","name":"Pamidronate","aka":[],"tldr":"Pamidronate (Aredia) is a bisphosphonate infusion approved in 1991 for dangerously high blood calcium in cancer and later for bone damage from myeloma and breast cancer, where it roughly halved skeletal complications. Its two-hour infusion, against fifteen minutes for zoledronic acid, pushed most centres to switch, though it is cheaper and may cause less jaw osteonecrosis.","summary":"Pamidronate disodium was approved in October 1991 for moderate or severe hypercalcaemia of malignancy with or without bone metastases and later for osteolytic bone metastases of breast cancer and osteolytic lesions of multiple myeloma in conjunction with standard antineoplastic therapy, on placebo-controlled trials (Hortobagyi 1996, Berenson 1996) that roughly halved skeletal complications. The two-hour infusion, compared with fifteen minutes for zoledronic acid, and the pivotal head-to-head hypercalcaemia trials led most centres to switch, although pamidronate remains a lower-cost generic option and is preferred by some in myeloma because of a lower rate of osteonecrosis of the jaw in observational series. Renal toxicity with rapid infusion, acute-phase reactions and jaw osteonecrosis are the key risks.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Pamidronic_acid","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pamidronate"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/pamidronatedisodium"}],"tags":["nci-list","generic","supportive"],"related":["zoledronic-acid","denosumab"],"cancers":["multiple-myeloma","breast-hr-positive"],"sections":[],"technologies":["bone-modifying-agents"],"targets":[],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00376883","nct00033332","nct00083382"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aredia (historic)","modality":"Nitrogen-containing bisphosphonate (intravenous)","supportive":true,"mechanism":"Adsorbs to hydroxyapatite and inhibits osteoclast-mediated bone resorption, lowering serum calcium and reducing osteolytic lesion progression.","approvals":[{"region":"US","year":1991,"indication":"Hypercalcaemia of malignancy"},{"region":"US","year":1995,"indication":"Osteolytic bone metastases of breast cancer and osteolytic lesions of multiple myeloma, with antineoplastic therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pamiparib","kind":"drug","name":"Pamiparib","aka":["BGB-290"],"tldr":"Pamiparib is an oral parp inhibitor from BeiGene, in registered phase 3 trials for ovarian cancer.","summary":"Pamiparib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 3 trial sponsored by BeiGene, in ovarian cancer. A PARP inhibitor: the name stem -parib marks a small molecule that blocks the PARP DNA-repair enzyme, lethal to tumour cells that already lack a second repair pathway. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Pamiparib","url":"https://clinicaltrials.gov/search?intr=Pamiparib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["beone"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03519230"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral PARP inhibitor","mechanism":"A PARP inhibitor: the name stem -parib marks a small molecule that blocks the PARP DNA-repair enzyme, lethal to tumour cells that already lack a second repair pathway.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pamrevlumab","kind":"drug","name":"Pamrevlumab","aka":["FG-3019"],"tldr":"Pamrevlumab targeted the scar-like tissue around pancreatic tumours; its phase 3 LAPIS trial in locally advanced disease did not lengthen survival.","summary":"FibroGen developed pamrevlumab for fibrotic diseases and for pancreatic cancer, where connective tissue growth factor helps build the stroma that shields tumour cells. A phase 1/2 study suggested more patients could reach surgery.\n\nThe phase 3 LAPIS trial in locally advanced unresectable pancreatic cancer, combined with gemcitabine and nab-paclitaxel or FOLFIRINOX, did not meet its overall survival endpoint, reported in 2024. A metastatic arm within the Precision Promise platform trial was also stopped for futility, ending the programme.","status":"negative","asOf":"2026-09-17","links":[{"label":"ClinicalTrials.gov NCT03941093","url":"https://clinicaltrials.gov/study/NCT03941093"},{"label":"FibroGen announcement, LAPIS topline results (2024)","url":"https://investor.fibrogen.com/news-releases"}],"tags":[],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":["monoclonal-antibody"],"targets":[],"drugs":[],"companies":["fibrogen"],"institutions":[],"pathways":[],"terms":[],"trials":["lapis-trial"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"FG-3019","modality":"Monoclonal antibody against connective tissue growth factor (CTGF)","mechanism":"Binds connective tissue growth factor, a driver of the dense fibrous stroma that surrounds pancreatic tumours, aiming to make them more resectable and more permeable to chemotherapy.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pancrelipase","kind":"drug","name":"Pancrelipase (pancreatic enzyme replacement therapy)","aka":["PERT","Pancreatin","Pancreatic enzyme replacement therapy","Enteric-coated pancreatin"],"tldr":"Enzyme capsules taken with every meal replace the digestive enzymes a pancreas blocked or removed by cancer no longer delivers; NICE says to offer them to everyone with unresectable pancreatic cancer and to consider them around surgery.","summary":"Pancreatic exocrine insufficiency, with fat malabsorption, steatorrhoea and weight loss, affects about 72 percent of people with advanced pancreatic cancer and is commoner with tumours of the head (relative risk 3.36) (Iglesia 2020, seven cohorts). NICE NG85 recommends enteric-coated pancreatin for people with unresectable pancreatic cancer (1.6.1) and says to consider it before and after resection (1.6.2); it notes the September 2024 supply disruption and points to the Specialist Pharmacy Service tool for equivalent products. Evidence of benefit is mixed: observational studies pooled by Iglesia and colleagues associate replacement with 3.8 months longer survival and better quality of life, but the one multicentre randomised trial (88 patients on chemotherapy for unresectable disease, 48,000 lipase units a meal) found no difference in body mass index at eight weeks (Saito 2018). Dosing is titrated to symptoms, typically 40,000 to 50,000 units of lipase with meals and half that with snacks, taken with the first mouthfuls; acid suppression is added when response is poor.","status":"established","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Pancrelipase","links":[{"label":"NICE NG85 recommendations 1.6.1 to 1.6.3: nutritional management","url":"https://www.nice.org.uk/guidance/ng85/chapter/Recommendations"},{"label":"Saito et al., randomised trial of pancreatic enzyme replacement in unresectable pancreatic cancer (Pancreas 2018)","url":"https://doi.org/10.1097/MPA.0000000000001079"},{"label":"Iglesia et al., meta-analysis (UEG Journal 2020)","url":"https://doi.org/10.1177/2050640620938987"}],"tags":[],"related":[],"cancers":["pancreatic","locally-advanced-pdac","metastatic-pdac","resectable-pdac"],"sections":[],"technologies":["oncology-nutrition"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["cachexia","malnutrition-screening","pancreatic-palliation-obstruction-pain-nutrition"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Creon, Nutrizym, Pancrease","modality":"Oral porcine pancreatic enzymes (lipase, protease, amylase) in enteric-coated capsules","supportive":true,"mechanism":"Replaces lipase, protease and amylase in the duodenum so that fat, protein and starch are digested; the enteric coating releases the enzymes at the alkaline pH of the small bowel.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"panitumumab","kind":"drug","name":"Panitumumab","aka":[],"tldr":"Panitumumab is a fully human EGFR antibody, the preferred first-line partner for chemotherapy in left-sided, RAS-normal bowel cancer after the PARADIGM trial.","summary":"Fully human IgG2 anti-EGFR; fewer infusion reactions than cetuximab and no ADCC. PARADIGM (2022) established superiority over bevacizumab in left-sided RAS wild-type disease; PRIME established it with FOLFOX. Partner of sotorasib in KRAS G12C mCRC (CodeBreaK 300, approved 2025). Same RAS-testing requirement and skin toxicity as cetuximab.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Panitumumab","links":[{"label":"FDA label","url":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/125147s210lbl.pdf"},{"label":"NICE TA439","url":"https://www.nice.org.uk/guidance/ta439"},{"label":"EMA Vectibix","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/vectibix"}],"tags":[],"related":["ras-wild-type","kras-g12d"],"cancers":["colorectal"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["egfr"],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["paradigm","codebreak-300","nct06252649","nct06820463","nct07659795","nct07172919","prime","cairo5","foxtrot"],"people":[],"bottlenecks":[],"keyPapers":["paper-douillard-prime-panitumumab-ras-nejm-2013","paper-arnold-primary-tumour-side-ras-wild-type-ann-oncol-2017","paper-diaz-molecular-evolution-egfr-resistance-colorectal-nature-2012"],"journals":[],"dependsOn":[],"notes":["Colorectal cancer biomarkers: the PRIME extended RAS analysis moved the label from KRAS exon 2 to all RAS. Among 639 KRAS exon 2 wild-type patients, 108 (17%) had another RAS mutation and did worse on panitumumab-FOLFOX4, while the 512 with no RAS mutation gained 5.8 months of overall survival (26.0 against 20.2, hazard ratio 0.78) (Douillard 2013). Left-sided primary is the second requirement (Arnold 2017)."],"brand":"Vectibix","modality":"Monoclonal antibody (anti-EGFR)","mechanism":"High-affinity EGFR blockade without effector function.","approvals":[{"region":"US","year":2006,"indication":"EGFR-expressing mCRC after chemotherapy (later RAS wild-type)"},{"region":"US","year":2014,"indication":"First-line RAS wild-type mCRC with FOLFOX"},{"region":"US","year":2025,"indication":"KRAS G12C mCRC with sotorasib"},{"region":"EU","year":2007,"indication":"Metastatic colorectal cancer; RAS wild-type from 2013","note":"Vectibix marketing authorisation issued 3 December 2007."},{"region":"England (NICE)","year":2017,"indication":"Previously untreated RAS wild-type metastatic colorectal cancer with FOLFOX or FOLFIRI","note":"TA439 recommends panitumumab within its marketing authorisation subject to the patient access scheme."}],"mechanismSteps":["Panitumumab binds EGFR on the tumour cell surface","Blocks ligand-driven activation of the receptor","Downstream RAS-MAPK signalling falls in RAS wild-type cells","Cells stop proliferating; in RAS-mutant cells the pathway stays on and the drug does nothing"],"dosing":{"route":"Intravenous","schedule":"6 mg/kg every 2 weeks","modifications":"Withhold for grade 3-4 dermatologic toxicity","monitoring":"Electrolytes; skin","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/125147s210lbl.pdf"},"toxicity":[{"event":"Dermatologic toxicity (rash, paronychia)","note":"Very common; boxed warning"},{"event":"Hypomagnesaemia"},{"event":"Diarrhoea"}],"access":[],"regulatoryEvents":[{"date":"2006-09","type":"approval","region":"US","note":"Accelerated approval, refractory mCRC"},{"date":"2006-09-27","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"EGFR-expressing metastatic colorectal carcinoma with progression on or following fluoropyrimidine-, oxaliplatin-, and irinotecan-containing chemotherapy regimens"},{"date":"2014-05","type":"label-change","region":"US","note":"First-line with FOLFOX in RAS wild-type (PRIME)"},{"date":"2014-05-23","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2006 converted to traditional approval 7.7 years after it was granted.","indication":"EGFR-expressing metastatic colorectal carcinoma with progression on or following fluoropyrimidine-, oxaliplatin-, and irinotecan-containing chemotherapy regimens"},{"date":"2025-01","type":"approval","region":"US","note":"With sotorasib for KRAS G12C mCRC (CodeBreaK 300)"}]},{"id":"panobinostat","kind":"drug","name":"Panobinostat","aka":[],"tldr":"An HDAC inhibitor for relapsed myeloma approved in 2015 and withdrawn in 2021 after its confirmatory trial was never completed.","summary":"Panobinostat is a pan-HDAC inhibitor that blocks class I, II and IV histone deacetylases including HDAC6. Inhibiting HDAC6 disrupts aggresome-mediated disposal of misfolded proteins, which synergises with proteasome inhibition in myeloma. PANORAMA-1 showed median progression-free survival of 12 versus 8.1 months when panobinostat was added to bortezomib-dexamethasone, at the cost of severe diarrhoea and cardiac events. The FDA granted accelerated approval in 2015 restricted to patients after at least two regimens including bortezomib and an immunomodulatory drug, and the EU approved it the same year. The confirmatory trial was never completed, and Secura Bio withdrew the US approval in 2021. Panobinostat showed that HDAC inhibition adds real activity in myeloma, but its toxicity meant the trade-off never earned a permanent place.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Panobinostat","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Panobinostat"}],"tags":["gap-fill"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":[],"companies":["novartis","securabio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00742027","nct02506959","nct00880269"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Farydak","modality":"Pan-HDAC inhibitor","mechanism":"Inhibits class I, II and IV HDACs including HDAC6, disrupting aggresome-mediated protein disposal and synergising with proteasome inhibition in myeloma.","approvals":[{"region":"US","year":2015,"indication":"Multiple myeloma after ≥2 regimens including bortezomib and an IMiD","note":"Withdrawn 2021"},{"region":"EU","year":2015,"indication":"Relapsed/refractory multiple myeloma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2015-02-23","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with bortezomib (BTZ) and dexamethasone (DEX) for the treatment of patients with multiple myeloma (MM) who have received at least 2 prior regimens, including BTZ and an immunomodulatory agent."},{"date":"2022-03-24","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 7.1 years after its accelerated approval.","indication":"In combination with bortezomib (BTZ) and dexamethasone (DEX) for the treatment of patients with multiple myeloma (MM) who have received at least 2 prior regimens, including BTZ and an immunomodulatory agent."}]},{"id":"parsaclisib","kind":"drug","name":"Parsaclisib","aka":["INCB050465"],"tldr":"Parsaclisib is an oral pi3k inhibitor from Incyte Corporation, in registered phase 2 trials for diffuse large B-cell lymphoma.","summary":"Parsaclisib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Incyte Corporation and Incyte Biosciences Japan GK, in diffuse large B-cell lymphoma. A phosphoinositide 3-kinase inhibitor: the name stem -lisib marks a small molecule that blocks PI3K signalling. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Parsaclisib","url":"https://clinicaltrials.gov/search?intr=Parsaclisib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["incyte"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04661007","nct04509700"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral PI3K inhibitor","mechanism":"A phosphoinositide 3-kinase inhibitor: the name stem -lisib marks a small molecule that blocks PI3K signalling.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pasireotide","kind":"drug","name":"Pasireotide","aka":["SOM230"],"tldr":"Pasireotide is a second-generation somatostatin analogue that hits more receptor types than octreotide. It is approved for Cushing's disease and acromegaly caused by pituitary tumours when surgery has not cured them, at the cost of frequent high blood sugar.","summary":"Pasireotide (SOM230) was developed by Novartis and is now marketed by Recordati. The FDA approved the twice-daily subcutaneous form in 2012 for Cushing's disease when surgery is not an option or has failed, the first drug approved for that indication, and the monthly long-acting depot in 2014 for acromegaly inadequately controlled by surgery. It normalises cortisol in a minority and growth hormone and IGF-1 in a larger fraction of patients, and shrinks tumours in many.\n\nHyperglycaemia, from suppression of insulin and incretins, is the main side effect and often needs treatment. Beyond the pituitary, the phase 2 LUNA trial tested it alone and with everolimus in lung and thymic neuroendocrine tumours and found activity in each arm, without a placebo comparison. The pituitary tumours and lung neuroendocrine tumour pages name it.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Pasireotide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Pasireotide"}],"tags":["subtype-drugs-wave"],"related":["octreotide-lanreotide","everolimus"],"cancers":["pituitary-tumours","lung-net"],"sections":[],"technologies":[],"targets":["sstr2"],"drugs":[],"companies":["recordati"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00600886","nct01137682","nct00434148"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Signifor / Signifor LAR","modality":"Multireceptor somatostatin analogue (subcutaneous or monthly depot)","mechanism":"Binds somatostatin receptors 1, 2, 3 and 5, with far higher affinity for SSTR5 than octreotide; SSTR5 dominates on corticotroph adenomas, so it suppresses ACTH in Cushing's disease and growth hormone in acromegaly resistant to first-generation analogues.","approvals":[{"region":"US","year":2012,"indication":"Cushing's disease when pituitary surgery is not an option or has not been curative"},{"region":"US","year":2014,"indication":"Acromegaly inadequately controlled by surgery or for whom surgery is not an option (long-acting depot)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pasritamig","kind":"drug","name":"Pasritamig","aka":[],"tldr":"Pasritamig is Johnson & Johnson's bispecific T-cell engager that binds KLK2 on prostate cancer cells and CD3 on T cells, with a deliberately low-affinity CD3 arm. In phase 1 about 40% of patients had a PSA50 response with far less cytokine release syndrome than other engagers, and a phase 3 trial began in 2025.","summary":"Pasritamig (JNJ-78278343) is a bispecific T-cell engager that binds KLK2 on prostate cancer cells and CD3 on T cells; a deliberately low-affinity CD3 arm limits cytokine release so that it can be given in the outpatient setting. It is being developed for metastatic castration-resistant prostate cancer after an androgen receptor pathway inhibitor, with or without a prior taxane. In phase 1, about 40% of patients achieved a PSA50 response at the recommended dose, with cytokine release syndrome rates far below other engagers, and the phase 3 KLK2-P3-01 trial began in 2025. Whether PSA responses will translate into radiographic and survival benefit, and how durable responses are, remain open. For a newcomer, it is Johnson & Johnson's prostate-specific T-cell engager, notable for how little cytokine storm it caused in early trials.","status":"phase-3","asOf":"2026-09-06","links":[{"label":"ClinicalTrials.gov: trials of Pasritamig","url":"https://clinicaltrials.gov/search?intr=JNJ-78278343"}],"tags":[],"related":[],"cancers":["prostate"],"sections":[],"technologies":["t-cell-engager"],"targets":["klk2","cd3"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":["crs"],"trials":["nct07225946"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"JNJ-78278343","modality":"Bispecific T-cell engager (KLK2×CD3)","mechanism":"Low-affinity CD3 arm and KLK2 targeting; designed for outpatient dosing.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"patidegib","kind":"drug","name":"Patidegib","aka":["Saridegib (topical)","IPI-926"],"tldr":"Patidegib is a hedgehog-pathway blocker made into a skin gel. It is being tested in people with Gorlin syndrome, who grow dozens of basal cell carcinomas, to prevent new tumours without the hair loss, muscle cramps and taste loss that the oral drugs cause.","summary":"Patidegib is the topical formulation of saridegib (IPI-926), a Smoothened inhibitor first developed by Infinity Pharmaceuticals as an oral drug. PellePharm reformulated it as a 2 percent gel applied twice daily to the face. Because Gorlin (basal cell nevus) syndrome is caused by germline PTCH1 loss, every basal cell carcinoma depends on hedgehog signalling, and oral vismodegib prevents them but is poorly tolerated for long-term use.\n\nA phase 2 study in Gorlin syndrome reported fewer new surgically eligible basal cell carcinomas with the gel than with vehicle, and a phase 3 trial followed. It is named on the locally advanced basal cell carcinoma page as the prevention approach for patients with the syndrome.","status":"phase-3","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["vismodegib","sonidegib"],"cancers":["locally-advanced-bcc","skin-cancer"],"sections":[],"technologies":[],"targets":["smoothened"],"drugs":[],"companies":["pellepharm"],"institutions":[],"pathways":[],"terms":["hedgehog-inhibitor-tolerability","inherited-skin-cancer-syndromes"],"trials":["patidegib-gel-gorlin-phase-2"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Topical hedgehog pathway (SMO) inhibitor gel","mechanism":"A cyclopamine derivative that binds Smoothened and blocks hedgehog signalling in the skin; applied as a gel it reaches the basal cells where basal cell carcinomas start without the systemic side effects of oral hedgehog inhibitors.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"patritumab-deruxtecan","kind":"drug","name":"Patritumab deruxtecan","aka":[],"tldr":"A HER3-directed ADC with Enhertu's payload, active in lung and all subtypes of breast cancer, but with a bumpy regulatory road.","summary":"HERTHENA-Lung01 in EGFR-mutant NSCLC after TKI (ORR ~30%) led to a BLA that received a complete response letter in 2024 over manufacturing; HERTHENA-Lung02 phase 3 was positive for PFS but not OS. In breast cancer, HERTHENA-BC dose-expansion data at ESMO Breast 2026 showed activity across HR+/HER2-, HER2+, and TNBC. Daiichi Sankyo and Merck partnership.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Patritumab deruxtecan","url":"https://clinicaltrials.gov/search?intr=HER3-DXd"}],"tags":[],"related":[],"cancers":["nsclc","tnbc","breast-hr-positive","egfr-mutant-nsclc"],"sections":[],"technologies":["adc"],"targets":["her3"],"drugs":[],"companies":["daiichi-sankyo","merck"],"institutions":[],"pathways":[],"terms":["ggfg"],"trials":["nct04619004","nct06172478","nct06731907","nct06469944","nct07286149","nct07060807","nct06797635","nct06596694","nct06686394","nct06941272"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"HER3-DXd, U3-1402","modality":"ADC","payload":"DXd","linker":"GGFG cleavable","mechanism":"Anti-HER3 IgG1 with DXd.","approvals":[],"mechanismSteps":["Antibody binds HER3 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","DXd is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"5.6 mg/kg every 3 weeks","monitoring":"ILD, blood counts","source":"https://clinicaltrials.gov/study/NCT04619004"},"toxicity":[{"event":"Neutropenia"},{"event":"Thrombocytopenia"},{"event":"Nausea"},{"event":"Fatigue"},{"event":"ILD/pneumonitis"}],"access":[{"country":"US","reimbursement":"Investigational; BLA withdrawn May 2025 after HERTHENA-Lung02 OS result","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-12-23","type":"designation","region":"US","note":"Breakthrough Therapy designation, EGFR-mutant NSCLC after TKI and platinum","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-12","type":"filing","region":"US","note":"BLA submitted (HERTHENA-Lung01)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-06-26","type":"crl","region":"US","note":"Complete response letter citing third-party manufacturing inspection","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-05","type":"withdrawal","region":"US","note":"BLA withdrawn after HERTHENA-Lung02 met PFS but not OS","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"pazopanib","kind":"drug","name":"Pazopanib","aka":[],"tldr":"Pazopanib is the only multi-kinase inhibitor approved for soft-tissue sarcoma (excluding fat-derived tumours), used after chemotherapy fails.","summary":"Pazopanib is an ATP-competitive inhibitor of VEGFR1-3, PDGFR alpha and beta, KIT and FGFR that starves tumours of new blood vessels. It is the only multi-kinase inhibitor approved for soft-tissue sarcoma and is used after chemotherapy fails. In PALETTE, patients with non-adipocytic soft-tissue sarcoma after anthracycline had PFS 4.6 versus 1.6 months on placebo (HR 0.31) but no OS benefit, and the FDA approved it in April 2012; liposarcoma was excluded after poor phase 2 results. It was first approved in 2009 for advanced renal cell carcinoma, where newer combinations have largely displaced it. Liver enzyme elevation carries a boxed warning, hypertension affected 41%, fatigue 65%, and pneumothorax in about 3% is a sarcoma-specific concern; Novartis markets it. Pazopanib delays progression in sarcoma without clearly extending life, a trade-off weighed against its side effects.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Pazopanib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pazopanib"}],"tags":[],"related":[],"cancers":["sarcoma","rcc","angiosarcoma","alveolar-soft-part-sarcoma"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf","kit"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07087158"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Votrient","modality":"Small-molecule kinase inhibitor (VEGFR/PDGFR/KIT)","mechanism":"ATP-competitive inhibitor of VEGFR1-3, PDGFRα/β, KIT, FGFR.","approvals":[{"region":"US","year":2009,"indication":"Advanced renal cell carcinoma"},{"region":"US","year":2012,"indication":"Advanced non-adipocytic soft-tissue sarcoma after chemotherapy"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"800 mg once daily on an empty stomach","monitoring":"Liver enzymes (boxed warning), blood pressure, thyroid, QT, pneumothorax in sarcoma"},"toxicity":[{"event":"Hypertension","anyGradePct":41,"grade3PlusPct":7,"note":"PALETTE"},{"event":"Fatigue","anyGradePct":65,"grade3PlusPct":13},{"event":"ALT elevation","grade3PlusPct":8},{"event":"Pneumothorax","anyGradePct":3}],"access":[],"regulatoryEvents":[]},{"id":"pbp1510","kind":"drug","name":"PBP1510","aka":[],"tldr":"PBP1510 is a monoclonal antibody from Prestige Biopharma Limited, in registered phase 2 trials for pancreatic ductal adenocarcinoma.","summary":"PBP1510 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Prestige Biopharma Limited, in pancreatic ductal adenocarcinoma. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of PBP1510","url":"https://clinicaltrials.gov/search?intr=PBP1510"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["prestige-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05141149"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"PBP1510","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"dako-pd-l1-22c3-pharmdx","kind":"drug","name":"PD-L1 IHC 22C3 pharmDx","aka":[],"tldr":"The 22C3 pharmDx assay is the PD-L1 stain tied to pembrolizumab since 2015 and the source of the combined positive score (CPS). The cut-off differs by cancer: 1% of tumour cells in lung cancer, CPS 1 in gastric, cervical and head and neck cancer, CPS 10 in oesophageal and triple-negative breast cancer, so one stain is read differently per disease.","summary":"Approved 2 October 2015 (PMA P150013, Dako, now Agilent) as the companion diagnostic for pembrolizumab in non-small-cell lung cancer, initially at TPS ≥50% and from 2016 at TPS ≥1%. It has since been approved for gastric and gastro-oesophageal junction cancer (CPS ≥1, 2017), cervical cancer (CPS ≥1, 2018), head and neck squamous cell carcinoma (CPS ≥1, 2019), oesophageal squamous cell carcinoma (CPS ≥10, 2019) and triple-negative breast cancer (CPS ≥10, 2020, KEYNOTE-355). The many cut-offs are a source of confusion for patients: the same stain is read differently depending on the cancer and the trial that set the threshold.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P150013","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P150013"},{"label":"Agilent: PD-L1 IHC 22C3 pharmDx","url":"https://www.agilent.com/en/product/pharmdx/pd-l1-ihc-22c3-pharmdx"}],"tags":["test"],"related":[],"cancers":["nsclc","gastric","cervical","head-and-neck","esophageal","tnbc","tnbc-metastatic"],"sections":[],"technologies":["companion-diagnostic","histopathology-ihc"],"targets":["pdl1"],"drugs":["pembrolizumab"],"companies":["agilent"],"institutions":[],"pathways":[],"terms":["pd-l1-testing","cps","ihc"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-rugo-pd-l1-assay-comparison-impassion130-jnci-2021","paper-sigurjonsdottir-sp142-22c3-tnbc-bcr-2023"],"journals":[],"dependsOn":[],"notes":["What a result means: the report gives a TPS or CPS; whether it counts as 'positive' depends on the drug label for your cancer, so the same number can be positive in one cancer and negative in another."],"brand":"PD-L1 IHC 22C3 pharmDx","modality":"PD-L1 immunohistochemistry companion diagnostic assay","mechanism":"Mouse monoclonal antibody 22C3 on the Dako Autostainer Link 48, reported as tumour proportion score (TPS) in lung cancer or combined positive score (CPS, tumour plus immune cells relative to tumour cells) in other cancers.","approvals":[{"region":"US","year":2015,"indication":"Companion diagnostic for pembrolizumab in NSCLC (TPS)"},{"region":"US","year":2017,"indication":"Gastric / GEJ adenocarcinoma, CPS ≥1"},{"region":"US","year":2019,"indication":"Head and neck squamous cell carcinoma (CPS ≥1) and oesophageal squamous cell carcinoma (CPS ≥10)"},{"region":"US","year":2020,"indication":"Triple-negative breast cancer, CPS ≥10 (pembrolizumab plus chemotherapy)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pegfilgrastim","kind":"drug","name":"Pegfilgrastim","aka":["Fulphila (pegfilgrastim-jmdb)","Udenyca (pegfilgrastim-cbqv)","Ziextenzo (pegfilgrastim-bmez)","Nyvepria (pegfilgrastim-apgf)","Fylnetra","Stimufend","Undencyca","Neulasta Onpro on-body injector"],"tldr":"Pegfilgrastim (Neulasta) is a long-acting form of the growth factor G-CSF, given once per chemotherapy cycle to speed white cell recovery and prevent febrile neutropenia; one dose replaces about eleven daily filgrastim injections. Biosimilars have been sold since 2018 and the Onpro on-body injector delivers it automatically the day after chemotherapy; bone pain is the main side effect.","summary":"Pegfilgrastim was approved by the FDA in January 2002 to decrease the incidence of infection manifested by febrile neutropenia in non-myeloid malignancies receiving myelosuppressive chemotherapy, on randomised trials showing a single 6 mg dose per cycle equivalent to about 11 daily filgrastim injections; the EU authorised Neulasta in August 2002 and an acute radiation syndrome indication was added in the US in 2015. It became one of the highest-selling cancer drugs before biosimilars arrived (Fulphila and Udenyca in 2018, Ziextenzo, Nyvepria, Fylnetra, Stimufend and others since), and the Onpro on-body injector avoids a next-day clinic visit. It is not indicated for stem cell mobilisation. Bone pain is the main adverse effect; splenic rupture, capillary leak, aortitis and glomerulonephritis are rare.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Pegfilgrastim","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pegfilgrastim"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/pegfilgrastim"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/neulasta"}],"tags":["nci-list","supportive"],"related":["filgrastim"],"cancers":[],"sections":[],"technologies":["g-csf-growth-factors"],"targets":[],"drugs":[],"companies":["amgen","sandoz","eurofarma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06690775"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Neulasta","modality":"Pegylated recombinant granulocyte colony-stimulating factor","supportive":true,"mechanism":"Filgrastim with a 20 kDa polyethylene glycol chain that abolishes renal clearance so that elimination depends on neutrophil-mediated uptake; levels fall as neutrophils recover, giving self-regulating once-per-cycle dosing.","approvals":[{"region":"US","year":2002,"indication":"Decrease in febrile neutropenia in non-myeloid malignancies on myelosuppressive chemotherapy (acute radiation syndrome added 2015)"},{"region":"US","year":2018,"indication":"First biosimilars (Fulphila, Udenyca); further biosimilars 2019 onwards"},{"region":"EU","year":2002,"indication":"Reduction in duration of neutropenia and febrile neutropenia after cytotoxic chemotherapy (Neulasta); biosimilars from 2018"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pegilodecakin","kind":"drug","name":"Pegilodecakin","aka":["AM0010","Pegylated interleukin-10"],"tldr":"Pegilodecakin, a long-acting interleukin-10 meant to expand tumour-killing T cells, added nothing to FOLFOX in the phase 3 SEQUOIA trial of second-line pancreatic cancer, and Eli Lilly dropped it.","summary":"Pegylated interleukin-10 raised CD8 T cell activity in early studies, and ARMO BioSciences (bought by Eli Lilly in 2018) took it into SEQUOIA: 567 patients whose metastatic pancreatic cancer had progressed on gemcitabine-based therapy (94.7 percent after gemcitabine and nab-paclitaxel) randomised to FOLFOX with or without pegilodecakin. Median overall survival was 5.8 against 6.3 months (hazard ratio 1.045), progression-free survival 2.1 months in both arms and response 4.6 against 5.6 percent; thrombocytopenia (55 against 20 percent) and anaemia (40 against 16 percent) were commoner with the cytokine while immune markers moved as predicted. Five UK sites took part (Cambridge, Hammersmith, Velindre Cardiff, University College London Hospitals, Guy's and St Thomas').","status":"negative","asOf":"2026-09-24","links":[{"label":"Hecht et al., SEQUOIA (JCO 2021)","url":"https://doi.org/10.1200/JCO.20.02232"},{"label":"ClinicalTrials.gov NCT02923921","url":"https://clinicaltrials.gov/study/NCT02923921"}],"tags":[],"related":[],"cancers":["pancreatic","metastatic-pdac"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":["cold-vs-hot","pancreatic-failed-programmes"],"trials":["sequoia"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"AM0010, LY3500518","modality":"PEGylated recombinant human interleukin-10 (cytokine)","mechanism":"Interleukin-10 receptor agonism, expanding and activating tumour-infiltrating CD8 T cells while suppressing inflammatory myeloid signalling.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"peginterferon-alfa-2b","kind":"drug","name":"Peginterferon alfa-2b","aka":[],"tldr":"Peginterferon alfa-2b, a long-acting interferon, was approved in 2011 as Sylatron for melanoma that had spread to lymph nodes and been removed, to delay recurrence; checkpoint inhibitors have since replaced it in that role.","summary":"Peginterferon alfa-2b was first approved in 2000 for chronic hepatitis C. The Sylatron formulation gained FDA approval in 2011 for adjuvant treatment of melanoma with microscopic or gross nodal involvement within 84 days of complete resection, based on the EORTC 18991 trial, in which five years of weekly injections improved relapse-free but not overall survival. Fatigue, depression and liver enzyme rises made most patients stop early. Adjuvant pembrolizumab, nivolumab and dabrafenib with trametinib have superseded interferon in resected stage III melanoma, and Sylatron was discontinued.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Peginterferon_alfa-2b","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=peginterferon%20alfa-2b"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Peginterferon_alfa-2b"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["melanoma","polycythaemia-vera","essential-thrombocythaemia"],"sections":[],"technologies":["cytokine-therapy"],"targets":["ifnar1"],"drugs":["interferon-alfa","pembrolizumab"],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01387763","nct00539591"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Sylatron / PegIntron","modality":"Pegylated type I interferon, weekly subcutaneous injection","mechanism":"Interferon alfa-2b with a polyethylene glycol chain that slows clearance; it signals through IFNAR to stimulate immune cells and slow tumour growth.","approvals":[{"region":"US","year":2011,"indication":"Adjuvant treatment of melanoma with nodal involvement after complete resection (Sylatron)","note":"Discontinued; superseded by adjuvant checkpoint and targeted therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pegulicianine","kind":"drug","name":"Pegulicianine","aka":[],"tldr":"A cavity-imaging dye used during breast lumpectomy to spot leftover tumour before the operation ends, reducing second surgeries.","summary":"Pegulicianine is a PEGylated, quenched fluorescent dye that is cleaved by cathepsins and matrix metalloproteinases abundant in tumour and tumour-associated cells. When the lumpectomy cavity is scanned with the Lumicell direct visualisation system after standard excision, activated dye reveals residual cancer so the surgeon can remove it before closing. In the INSITE trial (NEJM Evidence 2023) residual tumour was found in 7.6% of patients after standard lumpectomy, with a sensitivity of 49% and specificity of 86%, and some re-excisions were avoided. The FDA approved it in April 2024 as an adjunct for intraoperative detection of residual breast cancer; anaphylaxis is a recognised risk. Its modest sensitivity means it complements rather than replaces margin pathology. Pegulicianine aims to reduce the second operations that follow many breast-conserving surgeries.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Pegulicianine","links":[{"label":"INSITE (NEJM Evidence 2023)","url":"https://doi.org/10.1056/EVIDoa2200333"},{"label":"ClinicalTrials.gov NCT03686215","url":"https://clinicaltrials.gov/study/NCT03686215"}],"tags":["gap-fill","diagnostic"],"related":[],"cancers":["breast-hr-positive","tnbc","breast-her2-positive"],"sections":[],"technologies":["fluorescence-guided-surgery","optical-imaging"],"targets":[],"drugs":[],"companies":["lumicell"],"institutions":[],"pathways":[],"terms":[],"trials":["insite"],"people":[],"bottlenecks":[],"keyPapers":["paper-smith-nejm-evid"],"journals":[],"dependsOn":[],"notes":[],"brand":"Lumisight","modality":"Protease-activated fluorescent imaging agent","mechanism":"PEGylated quenched dye cleaved by cathepsins and MMPs abundant in tumour and tumour-associated cells, fluorescing in the lumpectomy cavity to reveal residual cancer.","approvals":[{"region":"US","year":2024,"indication":"Fluorescence imaging in adults with breast cancer as an adjunct for intraoperative detection of residual cancer after lumpectomy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pegvisomant","kind":"drug","name":"Pegvisomant","aka":["B2036-PEG"],"tldr":"Pegvisomant is a daily injection that blocks growth hormone at its receptor. It normalises the downstream hormone IGF-1 in most people with acromegaly whose tumours were not cured by surgery, although it does not shrink the tumour itself.","summary":"Pegvisomant is a growth hormone receptor antagonist developed by Sensus and Pharmacia, now marketed by Pfizer. The FDA approved it in 2003 for acromegaly in patients with an inadequate response to surgery or radiotherapy and other medical treatments. Given by daily subcutaneous injection, it normalises IGF-1 in the great majority of patients, the highest biochemical control rate of any acromegaly drug, and improves symptoms and glucose metabolism.\n\nBecause it acts at the receptor rather than on the tumour, growth hormone levels rise and the adenoma is not shrunk, so tumour volume is monitored by MRI; liver enzymes are also checked. It is used alone or with a somatostatin analogue. The pituitary tumours page names it among the medical treatments for acromegaly after surgery.","status":"approved","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Pegvisomant","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Pegvisomant"}],"tags":["subtype-drugs-wave"],"related":["pasireotide","octreotide-lanreotide"],"cancers":["pituitary-tumours"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00383708","nct00143416","nct02952885"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Somavert","modality":"Pegylated growth hormone receptor antagonist (daily subcutaneous injection)","mechanism":"A mutated, pegylated growth hormone that binds the growth hormone receptor but cannot activate it, blocking IGF-1 production in the liver regardless of how much growth hormone the pituitary tumour secretes.","approvals":[{"region":"US","year":2003,"indication":"Acromegaly with inadequate response to surgery or radiotherapy and other medical therapies"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pegvorhyaluronidase-alfa","kind":"drug","name":"Pegvorhyaluronidase alfa","aka":["PEGPH20","PEGylated recombinant human hyaluronidase"],"tldr":"PEGPH20 was designed to break down the hyaluronan that stiffens pancreatic tumours and let chemotherapy in; in the phase 3 HALO-301 trial it raised response rates but not survival, and Halozyme stopped developing it in 2019.","summary":"Hyaluronan accumulates in the desmoplastic stroma of pancreatic ductal adenocarcinoma and raises interstitial pressure; pegvorhyaluronidase alfa degrades it. HALO 109-301 randomised 494 patients with untreated, hyaluronan-high metastatic disease 2 to 1 to PEGPH20 or placebo with nab-paclitaxel and gemcitabine. Median overall survival was 11.2 against 11.5 months (hazard ratio 1.00, 95 percent confidence interval 0.80 to 1.27), progression-free survival 7.1 months in both arms and objective response 47 against 36 percent; fatigue, muscle spasms and hyponatraemia were commoner with PEGPH20. The registry entry (NCT02715804) records termination after the interim analysis and 15 UK sites including Cambridge, Glasgow, Edinburgh, Birmingham, the Christie, Hammersmith and the Royal Marsden. The authors concluded the results did not support further development in metastatic pancreatic cancer.","status":"negative","asOf":"2026-09-24","links":[{"label":"Van Cutsem et al., HALO 109-301 (JCO 2020)","url":"https://doi.org/10.1200/JCO.20.00590"},{"label":"ClinicalTrials.gov NCT02715804","url":"https://clinicaltrials.gov/study/NCT02715804"}],"tags":[],"related":[],"cancers":["pancreatic","metastatic-pdac"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["halozyme"],"institutions":[],"pathways":[],"terms":["desmoplasia","pancreatic-failed-programmes"],"trials":["halo-301"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"PEGPH20","modality":"PEGylated recombinant hyaluronidase (stroma-degrading enzyme)","mechanism":"Enzymatic degradation of hyaluronan in the tumour stroma, lowering interstitial pressure and re-expanding vessels.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pegylated-liposomal-doxorubicin","kind":"drug","name":"Pegylated liposomal doxorubicin","aka":[],"tldr":"Doxorubicin wrapped in a fatty bubble so it reaches tumours with less heart damage; a workhorse of relapsed ovarian cancer.","summary":"Approved 1995 (Kaposi sarcoma) and 1999 for platinum-refractory ovarian cancer. Standard single agent or partner (with carboplatin in CALYPSO, with trabectedin in OVA-301, with bevacizumab in AURELIA) in relapsed disease; the comparator arm in MIRASOL and KEYNOTE-B96. Hand-foot syndrome and mucositis replace the alopecia and cardiotoxicity of free doxorubicin.\n\nIt was invented in Jerusalem. Yechezkel Barenholz's Laboratory of Membrane and Liposome Research at the Hebrew University, with the clinician Alberto Gabizon, combined three unrelated ideas: a polyethylene-glycol coat that keeps the liposome circulating and away from the reticuloendothelial system, remote loading of doxorubicin down a transmembrane ammonium sulfate gradient that holds the drug in and releases it at the tumour, and a bilayer kept in a liquid-ordered phase by a high-melting phosphatidylcholine with cholesterol. Licensed through the university's Yissum company, it became in 1995 the first nano-drug approved by any regulator; Barenholz's own retrospective sets out what the first twenty years taught.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Liposomal_doxorubicin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pegylated%20liposomal%20doxorubicin"},{"label":"Barenholz, Doxil, the first FDA-approved nano-drug: lessons learned (J Control Release 2012)","url":"https://doi.org/10.1016/j.jconrel.2012.03.020"}],"tags":[],"related":[],"cancers":["ovarian","multiple-myeloma","sarcoma"],"sections":[],"technologies":["cytotoxic-chemotherapy","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":["cspc"],"institutions":["hebrew-university-of-jerusalem"],"pathways":[],"terms":[],"trials":["nct06072781","nct07604766","nct06619236","nct06014528","actuate-1801","nct04274426","nct07286266","nct07109414"],"people":["yechezkel-barenholz","alberto-gabizon"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Doxil / Caelyx","code":"PLD","modality":"Cytotoxic (liposomal anthracycline)","mechanism":"Topoisomerase II poisoning and DNA intercalation by doxorubicin, delivered by long-circulating PEGylated liposomes that extravasate in tumours.","approvals":[{"region":"US","year":1995,"indication":"AIDS-related Kaposi sarcoma after prior chemotherapy (Doxil, NDA 050718)"},{"region":"US","year":1999,"indication":"Ovarian cancer refractory to platinum and paclitaxel"}],"mechanismSteps":[],"dosing":{"route":"Intravenous","schedule":"40-50 mg/m² every 4 weeks (ovarian)","monitoring":"LVEF at cumulative doses; skin"},"toxicity":[{"event":"Palmar-plantar erythrodysaesthesia","anyGradePct":51,"grade3PlusPct":24,"note":"Doxil label, ovarian"},{"event":"Stomatitis","anyGradePct":41,"grade3PlusPct":8}],"access":[],"regulatoryEvents":[{"date":"1995-11-17","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Kaposi's sarcoma in AIDS patients with disease progression on prior combination chemotherapy or who are intolerant to such therapy"},{"date":"1999-06-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Metastatic ovarian carcinoma refractory to paclitaxel and platinum-based chemotherapy"},{"date":"2005-01-28","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1999 converted to traditional approval 5.6 years after it was granted.","indication":"Metastatic ovarian carcinoma refractory to paclitaxel and platinum-based chemotherapy"},{"date":"2008-06-10","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1995 converted to traditional approval 12.6 years after it was granted.","indication":"Kaposi's sarcoma in AIDS patients with disease progression on prior combination chemotherapy or who are intolerant to such therapy"}]},{"id":"pelabresib","kind":"drug","name":"Pelabresib","aka":[],"tldr":"Pelabresib is an experimental small-molecule drug from Novartis Pharmaceuticals in phase 3 trials, with its target not yet stated publicly.","summary":"Pelabresib (CPI-0610) is a small-molecule drug developed by Novartis Pharmaceuticals. The sponsor describes its target as BET (bromodomain and extraterminal protein), which OnCo does not yet have a target page for. The sponsor states: A small-molecule BET protein inhibitor. ClinicalTrials.gov describes the intervention as: Small molecule inhibitor of bromodomain and extraterminal (BET) protein. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06401356 (An Extension Study for Patients Previously Enrolled in Studies With Pelabresib). The largest, NCT06401356, plans to enrol 50 participants with primary completion expected 2027-06-02. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Pelabresib","url":"https://clinicaltrials.gov/search?intr=CPI-0610"},{"label":"Sponsor pipeline page","url":"https://www.novartis.com/research-development/novartis-pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["primary-myelofibrosis"],"sections":[],"technologies":[],"targets":["brd4"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06401356"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CPI-0610","modality":"small molecule","mechanism":"A small-molecule BET protein inhibitor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"peluntamig","kind":"drug","name":"Peluntamig","aka":["Peluntamig (PT217)"],"tldr":"Peluntamig is an experimental bispecific antibody from Phanes Therapeutics in phase 2 trials for non-small-cell lung cancer and neuroendocrine tumours, aimed at CD47 and DLL3.","summary":"Peluntamig (PT217) is a bispecific antibody developed by Phanes Therapeutics. Its targets are CD47 and DLL3. ClinicalTrials.gov describes the intervention as: A bispecific antibody (bsAb) against DLL3 and CD47. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer and neuroendocrine tumours. The largest, NCT05652686, plans to enrol 203 participants with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Peluntamig","url":"https://clinicaltrials.gov/search?intr=PT217"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","neuroendocrine","extrapulmonary-nec"],"sections":[],"technologies":[],"targets":["cd47","dll3"],"drugs":[],"companies":["phanes-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05652686"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PT217","modality":"bispecific antibody","mechanism":"Bispecific antibody directed at CD47 and DLL3, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pembrolizumab","kind":"drug","name":"Pembrolizumab","aka":[],"tldr":"Pembrolizumab is a PD-1 blocking antibody approved in more than 40 settings, from melanoma and lung cancer to the first tumour-agnostic approval for mismatch-repair-deficient tumours in 2017, and before and after surgery in triple-negative breast cancer. A subcutaneous form arrived in 2025, and it is the backbone partner for ADCs and personalised neoantigen vaccines.","summary":"Approvals span melanoma, NSCLC, head and neck, Hodgkin, urothelial, MSI-H/dMMR tumours (first tumour-agnostic approval, 2017), gastric, oesophageal, cervical, HCC, RCC, endometrial, TNBC (KEYNOTE-355 metastatic CPS ≥10; KEYNOTE-522 neoadjuvant/adjuvant with 7-year OS benefit), TMB-high, and more. 2026 additions include platinum-resistant PD-L1+ ovarian cancer, adjuvant RCC with belzutifan, and combination labels with sacituzumab govitecan (ASCENT-04) and enfortumab vedotin. Subcutaneous Keytruda Qlex approved 2025. Backbone for neoantigen vaccines (intismeran).","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Pembrolizumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pembrolizumab"},{"label":"NICE TA851: pembrolizumab for neoadjuvant and adjuvant treatment of triple-negative early or locally advanced breast cancer (14 December 2022)","url":"https://www.nice.org.uk/guidance/ta851"},{"label":"NICE TA801: pembrolizumab plus chemotherapy for untreated triple-negative metastatic breast cancer (29 June 2022)","url":"https://www.nice.org.uk/guidance/ta801"},{"label":"Keytruda label (openFDA): TNBC indications and KEYNOTE-522, KEYNOTE-355 and KEYNOTE-D19 study sections","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22KEYTRUDA%22"},{"label":"NICE TA914 (20 September 2023)","url":"https://www.nice.org.uk/guidance/ta914"},{"label":"NICE TA709: pembrolizumab for untreated MSI-high or dMMR metastatic colorectal cancer","url":"https://www.nice.org.uk/guidance/ta709"},{"label":"NICE TA531: pembrolizumab for untreated PD-L1-positive metastatic non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta531"},{"label":"NICE TA683: pembrolizumab with pemetrexed and platinum chemotherapy for untreated metastatic non-squamous non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta683"},{"label":"NICE TA770: pembrolizumab with carboplatin and paclitaxel for untreated metastatic squamous non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta770"},{"label":"NICE TA1017: pembrolizumab neoadjuvant then adjuvant for resectable non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1017"},{"label":"NICE TA1037: pembrolizumab for adjuvant treatment of resected non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1037"}],"tags":[],"related":["tmb-testing","pd-l1-cps","pd-l1-tps","dmmr-ihc","msi-high","tmb-high"],"cancers":["msi-high-pdac","tnbc","nsclc","melanoma","head-and-neck","recurrent-metastatic-hnscc","hpv-negative-head-and-neck-cancer","urothelial","colorectal","gastric","gastric-pdl1-high","gastric-msi-high","cervical","hcc","rcc","endometrial","hodgkin-lymphoma","ovarian","pancreatic","msi-high-colorectal","primary-mediastinal-b-cell-lymphoma","tnbc-early","tnbc-metastatic","pdl1-high-nsclc","resectable-nsclc","advanced-melanoma","stage-iii-melanoma","stage-ii-melanoma","advanced-cutaneous-scc","merkel-cell-carcinoma","endometrial-mmr-deficient","advanced-recurrent-endometrial-cancer","locally-advanced-cervical-cancer","recurrent-metastatic-cervical-cancer","platinum-resistant-ovarian-cancer","vaginal-adenocarcinoma","advanced-small-bowel-adenocarcinoma","lung-cancer"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["keynote-522","keynote-355","ascent-04","interpath-001","nct06212752","nct06348199","nct06758401","nct06216301","nct05920356","nct06430866","nct06692738","nct06448312","nct06899126","nct06698042","nct07216703","nct06699212","nct05727904","nct04613596","nct07541170","nct07361510","nct06170788","nct06452277","nct06311721","nct06989112","nct06422143","nct05215340","nct05317858","nct06711900","nct06712316","nct06312137","nct05608291","nct06947928","nct06952504","nct05555732","nct06077760","nct06726265","nct02362594","nct06561386","nct04624204","nct06767514","nct05722015","nct06793215","nct06357533","nct05048797","nct06627647","nct05549297","nct04657991","nct04956692","nct05984277","nct07777822","nct07178795","nct07195695","nct05987332","nct03556228","nct05180799","nct05446298","nct05784688","nct03970746","nct02332668","nct05482893","nct04140526","nct04083599","nct07054567","nct03228667","nct03407144","nct06099782","nct07098988","nct06797297","nct05970497","keynote-158","nct03611868","nct04752826","nct04895722","nct05081609","nct06731907","nct06784648","nct05789082","nct06699836","nct06284590","nct05320588","nct07255664","nct05086692","nct04701476","nct07807163","nct03897881","nct05797168","nct04585750","nct06644768","nct03785249","nct07452224","nct06265025","nct07221474","nct05585320","nct04787042","nct04920617","nct05620134","nct07622186","nct04675294","nct05440708","nct05877430","nct06162221","nct03526835","nct07264816","nct04736173","nct05144698","nct02861573","nct05394103","nct03647163","nct05805501","nct05318573","nct05816252","nct07287917","nct06307431","nct06961006","nct06228326","nct06205706","nct07122687","nct07020221","nct06137144","nct03836352","nct05128487","nct05217446","nct06843447","nct04675333","nct05438420","nct05824975","nct06047379","nct05186974","nct05903833","nct04198766","nct04977453","nct03476681","nct05309421","nct06428409","nct05117242","nct07223047","nct04130516","nct06483334","nct05120596","nct04956640","nct06393374","nct07486817","nct07158918","nct05565378","nct05609578","nct06623422","nct07815665","nct06293651","nct04305795","nct03725059","nct06795412","nct06731478","nct06246110","nct07197827","nct07720284","nct07276399","nct06469944","nct07232602","nct07415031","nct06901531","nct06249048","nct06942234","nct07475806","nct07710885","nct05224141","nct04626479","nct07252739","nct06157151","nct03547973","nct06312176","nct06764875","nct05405595","nct07302347","nct06008093","nct04585035","nct07469774","nct05687266","nct06151574","nct04262466","nct04521413","nct05945823","nct05329532","nct06119581","nct06966700","nct04121455","nct06305767","nct06116136","nct05562830","nct07566156","nct06797635","nct06877650","nct03485209","nct05642780","nct05609968","nct06385080","nct06549946","nct07644039","nct06545955","nct03505320","nct05319730","nct06772623","nct06041802","nct04225117","nct05732831","nct05669430","nct04241185","nct05899049","nct04938817","nct05101070","nct04976634","nct04895358","nct04626518","nct06112379","nct07315750","nct05761223","nct07679360","nct07639450","nct05173987","nct04041310","nct06875310","nct06016920","nct06780111","nct05911295","nct05410145","nct06225596","nct07751042","nct05533697","nct06868277","nct05438329","nct06978114","nct06788912","nct03711032","nct05852691","nct07730021","nct06962787","nct06618287","nct07811193","nct06472076","nct07287995","nct04561362","nct05239741","nct06493552","nct04879329","nct05053880","nct06556563","nct06939595","nct05845814","nct04380636","nct07286266","nct06841354","nct07749586","nct04592653","nct04634877","nct06890598","nct06634875","keynote-119","swog-s1418","neopact","c-trak-tn","impassion030","nct05093231","keynote-091","keynote-671","keynote-024-189","keynote-042","keynote-407"],"people":["tasuku-honjo","lajos-pusztai"],"bottlenecks":[],"keyPapers":["paper-keynote-189-nejm-2018","paper-keynote-564-nejm-2021","paper-keynote-355-nejm-2022","paper-sigurjonsdottir-sp142-22c3-tnbc-bcr-2023","paper-keynote-158-pembrolizumab-msi-high-noncolorectal-jco-2020","paper-hu-mismatch-repair-deficiency-pancreatic-adenocarcinoma-ccr-2018","paper-le-mmr-deficiency-pd1-nejm-2015","paper-le-mmr-deficiency-science-2017","paper-moreira-lynch-syndrome-identification-jama-2012"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: metastatic use requires 22C3 CPS 10 or more, about 27% of unselected early tumours and 38% of KEYNOTE-355 screening (Cortes 2022, Sigurjonsdottir 2023); early-stage use in KEYNOTE-522 needs no PD-L1 test.","Triple-negative breast cancer, UK status: two NICE appraisals are in force (TA851 early disease, TA801 metastatic CPS 10 or more with immune-cell staining below 1 percent). A third, pembrolizumab as adjuvant treatment after neoadjuvant chemotherapy alone (GID-TA11724, the SWOG S1418 question), is awaiting development, and the appraisal of pembrolizumab for previously treated metastatic disease (GID-TA10295, KEYNOTE-119) was discontinued after that trial missed its primary endpoint.","Pancreatic cancer: the Keytruda label's tumour-agnostic MSI-H or dMMR indication rests on KEYNOTE-158, whose pancreatic cohort had 4 responses in 22 patients (18 percent; 95 percent confidence interval 5 to 40; duration 8.1 to 24.3 or more months). In the EU the MSI-H indication names colorectal, endometrial, gastric, small intestine and biliary cancer and in England NICE TA914 mirrors that list, so mismatch repair deficient pancreatic cancer (about 1 percent of cases) has no funded checkpoint inhibitor route in Europe outside trials such as the Cambridge-led pembrolizumab and olaparib study (NCT05093231, 15 UK sites).","Pancreatic ductal adenocarcinoma: the threshold is MSI-high or mismatch repair deficiency, about 1% of cases; KEYNOTE-158's pancreatic cohort gave 4 responses among 22 patients against 34.3% across all non-colorectal MSI-high tumours (Marabelle 2020). PD-L1 expression is not a threshold in this disease and unselected checkpoint blockade has failed.","Colorectal cancer biomarkers: MSI-high or mismatch repair deficiency, about 5% of metastatic and 10 to 15% of resected disease. The first study gave 4 responses in 10 deficient colorectal cancers and 0 in 18 proficient ones (Le 2015). PD-L1 expression and tumour mutational burden are not colorectal thresholds; universal mismatch repair testing identifies the eligible patients as a by-product of Lynch syndrome screening (Moreira 2012)."],"brand":"Keytruda / Keytruda Qlex (SC)","modality":"Monoclonal antibody (anti-PD-1)","mechanism":"Humanised IgG4 blocking PD-1; restores T-cell effector function.","approvals":[{"region":"US","year":2014,"indication":"Melanoma (first of >40 indications)"},{"region":"US","year":2017,"indication":"MSI-H/dMMR solid tumours (tumour-agnostic)"},{"region":"US","year":2020,"indication":"Metastatic TNBC, PD-L1 CPS ≥10, with chemotherapy"},{"region":"US","year":2021,"indication":"High-risk early TNBC, neoadjuvant + adjuvant (KEYNOTE-522)"},{"region":"US","year":2023,"indication":"Locally advanced unresectable or metastatic biliary tract cancer (gallbladder cancer included) with gemcitabine and cisplatin (KEYNOTE-966)","note":"Keytruda label (openFDA), KEYNOTE-966 section: https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22KEYTRUDA%22; no NICE appraisal for biliary tract cancer found in September 2026"},{"region":"US","year":2026,"indication":"Platinum-resistant PD-L1+ ovarian cancer; adjuvant RCC with belzutifan; with sacituzumab govitecan in 1L TNBC"},{"region":"EU","year":2021,"indication":"Locally recurrent unresectable or metastatic TNBC, PD-L1 CPS 10 or more, with chemotherapy, no prior chemotherapy for metastatic disease (KEYNOTE-355)","note":"EMA Keytruda product page, therapeutic indications: https://www.ema.europa.eu/en/medicines/human/EPAR/keytruda"},{"region":"EU","year":2022,"indication":"Locally advanced or early-stage TNBC at high risk of recurrence: neoadjuvant with chemotherapy, then adjuvant monotherapy (KEYNOTE-522)","note":"EMA Keytruda product page: https://www.ema.europa.eu/en/medicines/human/EPAR/keytruda"},{"region":"UK","year":2022,"indication":"Untreated triple-negative locally recurrent unresectable or metastatic breast cancer with paclitaxel or nab-paclitaxel; NICE TA801 (29 June 2022) recommends it only for tumours with CPS 10 or more and immune-cell staining below 1 percent (the atezolizumab population is carved out), under a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta801"},{"region":"UK","year":2022,"indication":"Neoadjuvant with chemotherapy then adjuvant alone for triple-negative early breast cancer at high risk of recurrence or locally advanced disease; NICE TA851 (14 December 2022) recommends within the marketing authorisation, routine commissioning with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta851"},{"region":"UK","year":2023,"indication":"Mismatch repair deficient or MSI-high tumours: NICE TA914 (20 September 2023) covers previously treated endometrial, gastric, small intestine, biliary and colorectal cancer and does not include pancreatic cancer","note":"https://www.nice.org.uk/guidance/ta914"},{"region":"England (NICE)","year":2021,"indication":"Untreated metastatic colorectal cancer with high microsatellite instability or mismatch repair deficiency","note":"TA709, published 23 June 2021: recommended only if pembrolizumab is stopped after two years without documented progression and the company provides it under the commercial arrangement."},{"region":"England (NICE)","year":2023,"indication":"Previously treated MSI-high or dMMR endometrial, biliary, colorectal, gastric or small intestine cancer","note":"TA914, published 20 September 2023: in colorectal cancer after fluoropyrimidine combination therapy, only if nivolumab with ipilimumab is unsuitable, and stopped at two years."},{"region":"England (NICE)","year":2018,"indication":"Untreated PD-L1-positive metastatic non-small-cell lung cancer with a tumour proportion score of 50 percent or more and no EGFR or ALK alteration","note":"TA531, published 18 July 2018, only if pembrolizumab is stopped at 2 years of uninterrupted treatment or earlier on progression."},{"region":"England (NICE)","year":2021,"indication":"Untreated metastatic non-squamous non-small-cell lung cancer without an EGFR or ALK alteration, with pemetrexed and platinum chemotherapy","note":"TA683, published 10 March 2021, only if stopped at 2 years of uninterrupted treatment or earlier on progression."},{"region":"England (NICE)","year":2022,"indication":"Untreated metastatic squamous non-small-cell lung cancer, with carboplatin and paclitaxel","note":"TA770, published 9 February 2022, for a tumour proportion score of 0 to 49 percent, or 50 percent or more where urgent clinical intervention is needed, stopped at 2 years."},{"region":"England (NICE)","year":2017,"indication":"Locally advanced or metastatic PD-L1-positive non-small-cell lung cancer after at least one chemotherapy","note":"TA428, published 11 January 2017 and last updated 12 September 2017, stopped at 2 years of uninterrupted treatment."},{"region":"England (NICE)","year":2024,"indication":"Resectable non-small-cell lung cancer at high risk of recurrence, neoadjuvant with platinum chemotherapy then adjuvant alone","note":"TA1017, published 20 November 2024 (KEYNOTE-671), subject to the commercial arrangement."},{"region":"England (NICE)","year":2025,"indication":"Adjuvant treatment of non-small-cell lung cancer at high risk of recurrence after complete resection and platinum chemotherapy","note":"TA1037, published 5 February 2025 (KEYNOTE-091), subject to the commercial arrangement."}],"mechanismSteps":["Antibody binds PD-1 on T cells","PD-1/PD-L1 engagement between T cell and tumour is blocked","Exhausted tumour-reactive T cells regain effector function","Interferon-γ and cytotoxic granules are released at the tumour","Tumour cells are killed; memory T cells persist"],"dosing":{"route":"IV infusion over 30 min (subcutaneous Keytruda Qlex available)","schedule":"200 mg every 3 weeks or 400 mg every 6 weeks; paediatric 2 mg/kg (max 200 mg) every 3 weeks; up to 24 months in most metastatic settings","modifications":"Hold for grade 2 immune-mediated events; permanently discontinue for grade 4 or recurrent grade 3","monitoring":"Thyroid function, LFTs, creatinine, glucose at baseline and periodically; patient education on immune-related symptoms","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287"},"toxicity":[{"event":"Hypothyroidism (immune-mediated)","anyGradePct":8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"},{"event":"Pneumonitis (immune-mediated)","anyGradePct":3.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"},{"event":"Colitis (immune-mediated)","anyGradePct":1.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"},{"event":"Hepatitis (immune-mediated)","anyGradePct":0.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"},{"event":"Fatigue","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"},{"event":"Musculoskeletal pain","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"},{"event":"Rash","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"},{"event":"Diarrhoea","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"},{"event":"Pyrexia","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=9333c79b-d487-4538-a9f0-71b91a02b287","note":"Pooled monotherapy data, >2,800 patients"}],"access":[{"country":"US","listPrice":"$11,564 per 200 mg dose (WAC, Merck price disclosure 2024)","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.merckaccessprogram-keytruda.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended across many indications (melanoma, NSCLC, TNBC KEYNOTE-522/355, RCC, HNSCC, cervical, oesophageal and others)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"},{"country":"EU","reimbursement":"EMA approved; reimbursed in all member states for core indications","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2014-09-04","type":"accelerated-approval","region":"US","note":"Accelerated approval, advanced melanoma after ipilimumab; first PD-1 inhibitor in the US","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor"},{"date":"2015-10-02","type":"accelerated-approval","region":"US","note":"PD-L1+ NSCLC after platinum","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Metastatic PD-L1 positive NSCLC, as determined by an FDA-approved test, with progression on or after platinum-containing chemotherapy"},{"date":"2015-12-18","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2014 converted to traditional approval 1.3 years after it was granted.","indication":"Unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor"},{"date":"2016-08-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Recurrent or metastatic head and neck squamous cell carcinoma that progressed on or after platinum-containing chemotherapy"},{"date":"2016-10-24","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2015 converted to traditional approval 1.1 years after it was granted.","indication":"Metastatic PD-L1 positive NSCLC, as determined by an FDA-approved test, with progression on or after platinum-containing chemotherapy"},{"date":"2017-03-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adult and pediatric patients with refractory classical Hodgkin Lymphoma or who have relapsed after 3 or more prior lines of therapy"},{"date":"2017-05-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with pemetrexed and carboplatin for first-line treatment of metastatic non-squamous NSCLC"},{"date":"2017-05-18","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov#footnote1_815g9a4","indication":"Locally advanced or metastatic urothelial carcinoma ineligible for cisplatin-containing chemotherapy 1"},{"date":"2017-05-23","type":"accelerated-approval","region":"US","note":"MSI-H/dMMR solid tumours: first tumour-agnostic approval","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult and pediatric patients with unresectable or metastatic, microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or metastatic MSI-H or dMMR colorectal cancer that have progressed following treatment with a fluoropyrimidine, oxaliplatin and irinotecan"},{"date":"2017-09-22","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"For patients with recurrent or locally advanced or metastatic gastric or GEJ adenocarcinoma whose tumors express PD-L1 [CPS ≥1] as determined by an FDA-approved test, with disease progression on/after two or more prior lines of therapy including fluoropyrimidine and platinum containing chemotherapy and if appropriate, HER2/NEU targeted therapy"},{"date":"2018-06-12","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS >/=1) as determined by an FDA approved test"},{"date":"2018-06-13","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma, or who have relapsed after 2 or more prior lines of therapy"},{"date":"2018-08-20","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 1.3 years after it was granted.","indication":"In combination with pemetrexed and carboplatin for first-line treatment of metastatic non-squamous NSCLC"},{"date":"2018-11-09","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/fda-grants-accelerated-approval-pembrolizumab-hepatocellular-carcinoma","indication":"Treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib"},{"date":"2018-12-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/fda-approves-pembrolizumab-merkel-cell-carcinoma","indication":"Treatment of adults and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC)"},{"date":"2019-06-10","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2016 converted to traditional approval 2.8 years after it was granted.","indication":"Recurrent or metastatic head and neck squamous cell carcinoma that progressed on or after platinum-containing chemotherapy"},{"date":"2019-06-17","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Metastatic SCLC with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy"},{"date":"2019-09-17","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with lenvatinib for advanced endometrial carcinoma not MSI-H or dMMR, with progression following systemic therapy and not candidates for curative surgery or radiation"},{"date":"2020-04-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Dosing Regimen: Alternative dose/schedule of 400mg every 6 weeks for adult patients with melanoma, hepatocellular carcinoma, Merkel cell carcinoma, gastric cancer, non-small cell lung cancer, renal cell carcinoma, endometrial carcinoma, cervical carcinoma, urothelial carcinoma, head and neck squamous cell carcinoma, small cell lung cancer, esophageal cancer, microsatellite instability-high or mismatch repair deficient solid tumors"},{"date":"2020-04-28","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 6.4 years later, when the FDA's table was read.","indication":"Dosing Regimen: Alternative dose/schedule of 400 mg every 6 weeks for adult patients with classical Hodgkin lymphoma and primary mediastinal b-cell lymphoma"},{"date":"2020-06-16","type":"accelerated-approval","region":"US","note":"TMB-high solid tumours (tumour-agnostic) The confirmatory requirement was still open 6.3 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden high (TMB H) [=10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options ."},{"date":"2020-06-16","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Dosing Regimen: Alternate Dose/Schedule of 400 mg every 6 weeks for adult patients with unresectable or metastatic tumor mutational burden high [≥10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options"},{"date":"2020-06-24","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Dosing Regimen: Alternative dose/schedule of 400mg every 6 weeks for adult patients with cutaneous squamous cell carcinoma"},{"date":"2020-10-14","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 3.6 years after it was granted.","indication":"Adult and pediatric patients with refractory classical Hodgkin Lymphoma or who have relapsed after 3 or more prior lines of therapy"},{"date":"2020-10-14","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 2.3 years after it was granted.","indication":"Adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma, or who have relapsed after 2 or more prior lines of therapy"},{"date":"2020-11-13","type":"accelerated-approval","region":"US","note":"Metastatic TNBC, PD-L1 CPS ≥10, with chemotherapy (KEYNOTE-355)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with chemotherapy for locally recurrent unresectable or metastatic TNBC expressing PD L1 [CPS =10] as determined by an FDA-approved test"},{"date":"2021-03-30","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 1.8 years after its accelerated approval.","indication":"Metastatic SCLC with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy"},{"date":"2021-05-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-pembrolizumab-her2-positive-gastric-cancer","indication":"In combination with trastuzumab and fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of patients with locally advanced unresectable or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma 1"},{"date":"2021-07-21","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2019 converted to traditional approval 1.8 years after it was granted.","indication":"In combination with lenvatinib for advanced endometrial carcinoma not MSI-H or dMMR, with progression following systemic therapy and not candidates for curative surgery or radiation"},{"date":"2021-07-26","type":"conversion","region":"US","note":"High-risk early TNBC, neoadjuvant and adjuvant (KEYNOTE-522)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with chemotherapy for locally recurrent unresectable or metastatic TNBC expressing PD L1 [CPS =10] as determined by an FDA-approved test"},{"date":"2021-08-31","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 4.3 years after it was granted.","source":"https://www.fda.gov#footnote1_815g9a4","indication":"Locally advanced or metastatic urothelial carcinoma ineligible for cisplatin-containing chemotherapy 1"},{"date":"2021-10-13","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 3.3 years after it was granted.","indication":"Treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS >/=1) as determined by an FDA approved test"},{"date":"2022-02-04","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 4.4 years after its accelerated approval.","indication":"For patients with recurrent or locally advanced or metastatic gastric or GEJ adenocarcinoma whose tumors express PD-L1 [CPS ≥1] as determined by an FDA-approved test, with disease progression on/after two or more prior lines of therapy including fluoropyrimidine and platinum containing chemotherapy and if appropriate, HER2/NEU targeted therapy"},{"date":"2022-12-16","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 2.6 years after it was granted.","indication":"Dosing Regimen: Alternative dose/schedule of 400mg every 6 weeks for adult patients with melanoma, hepatocellular carcinoma, Merkel cell carcinoma, gastric cancer, non-small cell lung cancer, renal cell carcinoma, endometrial carcinoma, cervical carcinoma, urothelial carcinoma, head and neck squamous cell carcinoma, small cell lung cancer, esophageal cancer, microsatellite instability-high or mismatch repair deficient solid tumors"},{"date":"2022-12-16","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 2.5 years after it was granted.","indication":"Dosing Regimen: Alternate Dose/Schedule of 400 mg every 6 weeks for adult patients with unresectable or metastatic tumor mutational burden high [≥10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options"},{"date":"2022-12-16","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 2.5 years after it was granted.","indication":"Dosing Regimen: Alternative dose/schedule of 400mg every 6 weeks for adult patients with cutaneous squamous cell carcinoma"},{"date":"2023-03-28","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2017 converted to traditional approval 5.8 years after it was granted.","indication":"Treatment of adult and pediatric patients with unresectable or metastatic, microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options, or metastatic MSI-H or dMMR colorectal cancer that have progressed following treatment with a fluoropyrimidine, oxaliplatin and irinotecan"},{"date":"2023-04-03","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-enfortumab-vedotin-ejfv-pembrolizumab-locally-advanced-or-metastatic","indication":"In combination with enfortumab vedotin-ejfv for the treatment of adult patients with locally advanced or metastatic urothelial cancer who are not eligible for cisplatin-containing chemotherapy"},{"date":"2023-10-12","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 4.8 years after it was granted.","source":"https://www.fda.gov/drugs/fda-approves-pembrolizumab-merkel-cell-carcinoma","indication":"Treatment of adults and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC)"},{"date":"2023-10-16","type":"approval","region":"US","note":"Perioperative NSCLC (KEYNOTE-671)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-12-15","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2023 converted to traditional approval 0.7 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-enfortumab-vedotin-ejfv-pembrolizumab-locally-advanced-or-metastatic","indication":"In combination with enfortumab vedotin-ejfv for the treatment of adult patients with locally advanced or metastatic urothelial cancer who are not eligible for cisplatin-containing chemotherapy"},{"date":"2024-01-25","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 5.2 years after it was granted.","source":"https://www.fda.gov/drugs/fda-grants-accelerated-approval-pembrolizumab-hepatocellular-carcinoma","indication":"Treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib"},{"date":"2025-03-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2021 converted to traditional approval 3.9 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-pembrolizumab-her2-positive-gastric-cancer","indication":"In combination with trastuzumab and fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of patients with locally advanced unresectable or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma 1"},{"date":"2025-09","type":"approval","region":"US","note":"Subcutaneous pembrolizumab (Keytruda Qlex)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-09-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.0 year later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-and-berahyaluronidase-alfa-pmph-subcutaneous-injection","indication":"Formulation: Treatment of adult and pediatric patients 12 years and older with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options ."},{"date":"2026-Q1","type":"approval","region":"US","note":"Platinum-resistant PD-L1+ ovarian cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-06-12","type":"approval","region":"US","note":"Adjuvant RCC with belzutifan (LITESPARK-022)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-belzutifan-pembrolizumab-adjuvant-treatment-renal-cell-carcinoma"},{"date":"2026-06-24","type":"approval","region":"US","note":"First-line PD-L1+ TNBC with sacituzumab govitecan (ASCENT-04)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-sacituzumab-govitecan-hziy-monotherapy-and-combination-pembrolizumab-first-line"},{"date":"2026-07-10","type":"approval","region":"US","note":"Muscle-invasive bladder cancer with enfortumab vedotin, as pembrolizumab or pembrolizumab with berahyaluronidase alfa","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-or-pembrolizumab-and-berahyaluronidase-alfa-pmph-each-enfortumab-vedotin"}]},{"id":"pemetrexed","kind":"drug","name":"Pemetrexed","aka":[],"tldr":"Pemetrexed is the chemotherapy that, with a platinum drug, became the first approved treatment for mesothelioma in 2004, and is still the backbone today.","summary":"Pemetrexed is a multitargeted antifolate that inhibits thymidylate synthase, DHFR and GARFT, starving cells of the nucleotides needed for DNA synthesis. In EMPHACIS (2003), cisplatin-pemetrexed gave OS 12.1 versus 9.3 months compared with cisplatin alone, the first randomised survival gain in mesothelioma, and it became the first approved treatment for the disease in 2004. It is now given with pembrolizumab (KEYNOTE-483) or bevacizumab (MAPS), and it was the chemotherapy comparator that nivolumab-ipilimumab beat in CheckMate 743. It is also standard in non-squamous NSCLC, dosed at 500 mg/m2 every 21 days with a platinum for 4 to 6 cycles, then optional maintenance. Folic acid and vitamin B12 supplementation and dexamethasone premedication are required. It remains the chemotherapy backbone in mesothelioma that newer agents are added to rather than replacing.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Pemetrexed","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pemetrexed"}],"tags":[],"related":[],"cancers":["mesothelioma","nsclc"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["keynote-483","maps","checkmate-743","gefitinib-chemo-tmh","nct06212752","nct04351555","nct06305754","nct06899126","nct04379635","nct05800015","nct06956001","nct06564844","nct06417814","nct06459180","nct06452277","nct05116462","nct03800134","nct06711900","nct06396065","nct04129502","nct06712316","nct06312137","nct05555732","nct05870319","nct06726265","nct06561386","nct02486718","nct05722015","nct07642024","nct06793215","nct06687369","nct06074588","nct05048797","nct06627647","nct04956692","nct05984277","nct03178552","nct07777822","nct07178795","nct06097728","nct07100080","nct05668988","nct06382116","nct05180799","nct06475300","nct03970746","nct06514027","nct06448754","nct06946797","nct05633667","nct07680764","nct06731907","nct05789082","nct05904379","nct03775486","nct06943820","nct05431270","nct05635708","nct06996782","nct06706076","nct06161441","nct05098132","nct06162221","nct04736173","nct07122687","nct07070440","nct07020221","nct04665206","nct05841472","nct06758557","nct05403385","nct04198766","nct05742607","nct04956640","nct07486817","nct05609578","nct06623422","nct05498428","nct06246110","nct06667076","nct06474455","nct07415031","nct06783647","nct06008093","nct04538664","nct05687266","nct06151574","nct07586202","nct04025879","nct05261399","nct06119581","nct07492680","nct07171606","nct05112965","nct06385678","nct06194448","nct07109531","nct05456256","nct06772623","nct03003962","nct06875310","nct05410145","nct04988295","nct06788912","nct05443126","nct06970639","nct06741644","nct04380636"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Alimta (and generics)","modality":"Cytotoxic (antifolate)","mechanism":"Multitargeted antifolate inhibiting thymidylate synthase, DHFR, and GARFT.","approvals":[{"region":"US","year":2004,"indication":"Unresectable pleural mesothelioma with cisplatin"},{"region":"US","year":2008,"indication":"Non-squamous NSCLC first line"}],"mechanismSteps":["Enters cells via the reduced folate carrier","Polyglutamated and retained intracellularly","Inhibits thymidylate synthase, blocking DNA synthesis","Preferential toxicity to rapidly dividing cells; vitamin supplementation protects normal tissue"],"dosing":{"route":"Intravenous","schedule":"500 mg/m² day 1 every 21 days with cisplatin 75 mg/m² or carboplatin AUC 5; 4-6 cycles, then optional maintenance","modifications":"Hold for creatinine clearance <45 mL/min; dexamethasone premedication for rash","monitoring":"Renal function and blood counts; folic acid and B12 supplementation"},"toxicity":[{"event":"Neutropenia (grade 3+)","grade3PlusPct":23},{"event":"Fatigue","anyGradePct":48},{"event":"Nausea","anyGradePct":82},{"event":"Rash","anyGradePct":16}],"access":[],"regulatoryEvents":[{"date":"2004-08-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"As a single agent for locally advanced or metastatic NSCLC after prior chemotherapy"},{"date":"2008-09-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Non-squamous NSCLC: 1) as initial treatment with cisplatin; 2) as a single agent after prior chemotherapy"},{"date":"2009-07-02","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2004 converted to traditional approval 4.9 years after it was granted.","indication":"As a single agent for locally advanced or metastatic NSCLC after prior chemotherapy"},{"date":"2009-07-02","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2008 converted to traditional approval 0.8 years after it was granted.","indication":"Non-squamous NSCLC: 1) as initial treatment with cisplatin; 2) as a single agent after prior chemotherapy"},{"date":"2018-06-04","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with pembrolizumab and carboplatin for first-line treatment of metastatic non-squamous NSCLC"},{"date":"2019-01-30","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2018 converted to traditional approval 0.7 years after it was granted.","indication":"In combination with pembrolizumab and carboplatin for first-line treatment of metastatic non-squamous NSCLC"}]},{"id":"pemigatinib","kind":"drug","name":"Pemigatinib","aka":[],"tldr":"Pemigatinib was the first targeted therapy for bile duct cancer, for tumours with an FGFR2 gene fusion.","summary":"Pemigatinib is a selective oral inhibitor of FGFR1, 2 and 3, blocking the constitutively active receptor produced when FGFR2 is fused to another gene. It is used in previously treated cholangiocarcinoma with an FGFR2 fusion or rearrangement and was the first targeted therapy approved for bile duct cancer. FIGHT-202 showed an ORR of 37%, median PFS of 7.0 months and median OS of 17.5 months, with no responses in other FGF/FGFR alterations; accelerated approval followed in April 2020 (EU 2021), and it is also approved for FGFR1-rearranged myeloid and lymphoid neoplasms. Hyperphosphataemia (60%), alopecia, diarrhoea, nail toxicity and serous retinal detachment are on-target effects needing phosphate binders and eye checks. First-line FIGHT-302 was discontinued, so its place remains second line. For a newcomer, it is the first pill that targets a specific gene change in bile duct cancer.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Pemigatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pemigatinib"},{"label":"NICE TA722: pemigatinib for FGFR2 fusion or rearrangement cholangiocarcinoma (25 August 2021)","url":"https://www.nice.org.uk/guidance/ta722"}],"tags":[],"related":["fgfr2-fusion-rearrangement"],"cancers":["cholangiocarcinoma","intrahepatic-cholangiocarcinoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["fgfr2"],"drugs":[],"companies":["incyte"],"institutions":[],"pathways":[],"terms":["fgfr2-fusion"],"trials":["fight-202"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Gallbladder cancer: the FDA indication is cholangiocarcinoma with an FGFR2 fusion or rearrangement (Pemazyre label), and FIGHT-202 enrolled cholangiocarcinoma only; FGFR2 fusions are a feature of intrahepatic disease and gallbladder cancer was not part of the approval population, so there is no gallbladder evidence for pemigatinib beyond case reports."],"brand":"Pemazyre","modality":"Small-molecule kinase inhibitor (FGFR1-3)","mechanism":"Selective oral FGFR1-3 inhibitor.","approvals":[{"region":"US","year":2020,"indication":"Previously treated FGFR2-fusion/rearranged cholangiocarcinoma (accelerated)"},{"region":"EU","year":2021,"indication":"Same"},{"region":"UK","year":2021,"indication":"Locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement after systemic therapy; NICE TA722 recommended 25 August 2021 with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta722"}],"mechanismSteps":["Enters tumour cell","Occupies ATP pocket of fusion FGFR2 kinase","Blocks RAS-MAPK and PI3K signalling downstream","Cell-cycle arrest; hyperphosphataemia from FGFR1 blockade in kidney"],"dosing":{"route":"Oral","schedule":"13.5 mg daily for 14 days of each 21-day cycle","modifications":"Phosphate binders and dose holds for hyperphosphataemia","monitoring":"Serum phosphate, eye exams (retinal pigment epithelial detachment)"},"toxicity":[{"event":"Hyperphosphataemia","anyGradePct":60,"note":"FIGHT-202"},{"event":"Alopecia","anyGradePct":49},{"event":"Diarrhoea","anyGradePct":47},{"event":"Nail toxicity","anyGradePct":43},{"event":"Serous retinal detachment","anyGradePct":4}],"access":[],"regulatoryEvents":[{"date":"2020-04-17","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 6.4 years later, when the FDA's table was read.","indication":"Treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test."}]},{"id":"penpulimab","kind":"drug","name":"Penpulimab","aka":[],"tldr":"Penpulimab is a Chinese PD-1 antibody approved in the US in 2025 for nasopharyngeal carcinoma, the second after toripalimab.","summary":"Penpulimab is an IgG1 anti-PD-1 monoclonal antibody engineered so its Fc region does not bind Fc-gamma receptors, avoiding antibody-dependent killing or phagocytosis of the PD-1-positive T cells it is meant to activate. Developed by Akeso (also ivonescimab and cadonilimab), it was first approved in China in 2021 for relapsed or refractory classical Hodgkin lymphoma, later for NPC and NSCLC. The AK105-304 phase 3 trial showed that adding penpulimab to gemcitabine-cisplatin improved progression-free survival in recurrent or metastatic nasopharyngeal carcinoma, and single-agent activity was shown after platinum. The FDA approved it in April 2025 for the first-line combination and for later-line monotherapy, making it the second PD-1 antibody for this cancer in the US after toripalimab. Penpulimab is a checkpoint inhibitor designed to protect the T cells it switches on.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Penpulimab","links":[{"label":"FDA novel approvals 2025","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025"}],"tags":["gap-fill"],"related":[],"cancers":["nasopharyngeal","hodgkin-lymphoma","recurrent-metastatic-nasopharyngeal-carcinoma","relapsed-refractory-hodgkin-lymphoma"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["akeso"],"institutions":[],"pathways":["pd1-checkpoint"],"terms":[],"trials":["nct05862337","nct04974398","nct06821503","nct06825494"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Penpulimab-kcqx","modality":"Anti-PD-1 monoclonal antibody (IgG1, Fc-engineered null)","mechanism":"PD-1 blockade with Fc engineered to eliminate FcγR binding, avoiding ADCC/ADCP of PD-1+ T cells.","approvals":[{"region":"CN","year":2021,"indication":"Relapsed/refractory classical Hodgkin lymphoma; later NPC, NSCLC"},{"region":"US","year":2025,"indication":"Recurrent or metastatic non-keratinising NPC with cisplatin-gemcitabine; monotherapy after platinum and ≥1 other line"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pentostatin","kind":"drug","name":"Pentostatin","aka":["Deoxycoformycin","2'-deoxycoformycin"],"tldr":"An intravenous drug given every other week that produces long remissions in hairy cell leukaemia by poisoning the leukaemic lymphocytes' purine metabolism; cladribine is the one-week alternative.","summary":"Pentostatin was approved in the United States in 1991 for hairy cell leukaemia, initially after interferon alfa had failed and then as first-line treatment, on the strength of a randomised comparison in which it produced far more complete remissions than interferon (Grever, JCO 1995). It is given as a short intravenous infusion of 4 mg/m² every other week until maximal response. It is also used in T-cell leukaemias and lymphomas and, off label, in graft-versus-host disease and reduced-intensity transplant conditioning. Prolonged lymphopenia with infection risk is the main toxicity, and renal impairment requires dose reduction.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pentostatin","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pentostatin"},{"label":"Grever 1995, JCO: pentostatin versus interferon alfa in hairy cell leukaemia","url":"https://doi.org/10.1200/JCO.1995.13.4.974"}],"tags":["gap-fill","generic"],"related":["cladribine"],"cancers":["hairy-cell-leukemia"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["ada"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct00254163","nct01188798","nct01059786"],"people":[],"bottlenecks":[],"keyPapers":["paper-grever-j-clin-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Nipent","modality":"Purine analogue, adenosine deaminase inhibitor","mechanism":"Binds adenosine deaminase almost irreversibly, so deoxyadenosine triphosphate accumulates in lymphocytes, blocks DNA synthesis and triggers apoptosis.","approvals":[{"region":"US","year":1991,"indication":"Hairy cell leukaemia (initially alpha-interferon-refractory; later untreated disease)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pertuzumab","kind":"drug","name":"Pertuzumab","aka":[],"tldr":"A second HER2 antibody that binds a different spot from trastuzumab, blocking HER2 from pairing with HER3; together they extended survival by 16 months in CLEOPATRA.","summary":"Binds HER2 extracellular domain II, preventing HER2-HER3 heterodimerisation. Approved with trastuzumab and docetaxel in first-line metastatic disease (2012), neoadjuvant (2013, first pCR-based accelerated approval), and adjuvant node-positive disease (2017, APHINITY). Phesgo (2020) delivers both antibodies subcutaneously in minutes. Being partnered with T-DXd in first line (DESTINY-Breast09) and tucatinib in maintenance (HER2CLIMB-05).","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Pertuzumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pertuzumab"}],"tags":[],"related":["her2-ish-amplified"],"cancers":["breast-her2-positive","her2-positive-early-breast-cancer","gallbladder","cholangiocarcinoma","colorectal","biliary-tract-cancer"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["her2","her3"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["cleopatra","aphinity","destiny-breast09","her2climb-05","phergain","nct07518173","nct07196774","nct06891833","nct05593094","nct06445400","nct07102381","nct05802225","nct07294508","nct06057610","nct04208178","nct06771622","nct04538742","nct06178159","nct00781612","nct06686394","nct02320435","nct05150691","nct07140393","nct04514419","mypathway","determine-arm04"],"people":[],"bottlenecks":[],"keyPapers":["paper-nakamura-triumph-ctdna-pertuzumab-trastuzumab-her2-colorectal-nat-med-2021","paper-javle-mypathway-her2-biliary-lancet-oncol-2021"],"journals":[],"dependsOn":[],"notes":["Biliary tract cancer: with trastuzumab in the MyPathway basket, 9 of 39 patients with HER2-amplified or overexpressing metastatic biliary tract cancer responded (23 percent, 95 percent confidence interval 11 to 39); the UK DETERMINE platform (Cancer Research UK) offers the same pair to HER2-amplified rare cancers including gallbladder cancer at 27 NHS sites.","Colorectal cancer biomarkers: TRIUMPH enrolled on HER2 amplification confirmed in tissue or in circulating tumour DNA and gave confirmed responses in 30% of 27 tissue-positive and 28% of 25 ctDNA-positive patients, against 0% in a matched real-world salvage population; concurrent oncogenic RAS alterations in plasma predicted poor response (Nakamura 2021)."],"brand":"Perjeta; Phesgo (with trastuzumab, subcutaneous)","modality":"Monoclonal antibody (anti-HER2, dimerisation domain)","mechanism":"Anti-HER2 IgG1 against subdomain II; blocks ligand-dependent HER2-HER3 signalling; ADCC.","approvals":[{"region":"US","year":2012,"indication":"First-line HER2+ metastatic breast cancer with trastuzumab and docetaxel"},{"region":"US","year":2013,"indication":"Neoadjuvant HER2+ early breast cancer (accelerated; first pCR-based approval)"},{"region":"US","year":2017,"indication":"Adjuvant HER2+ early breast cancer at high risk of recurrence"},{"region":"US","year":2020,"indication":"Phesgo subcutaneous fixed-dose combination with trastuzumab"}],"mechanismSteps":["HER3, activated by neuregulin, seeks HER2 as a dimer partner","Pertuzumab occupies HER2 domain II, the dimerisation arm","HER2-HER3 pairs cannot form; PI3K signalling drops","Trastuzumab meanwhile blocks domain IV and recruits NK cells","Complementary blockade deepens response"],"dosing":{"route":"Intravenous (or subcutaneous as Phesgo)","schedule":"840 mg loading then 420 mg every 3 weeks; Phesgo 1200/600 mg loading then 600/600 mg SC every 3 weeks","monitoring":"LVEF at baseline and every 3 months","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/125409s128lbl.pdf"},"toxicity":[{"event":"Diarrhoea","anyGradePct":67,"grade3PlusPct":8},{"event":"Rash","anyGradePct":34},{"event":"Left ventricular dysfunction","anyGradePct":5,"grade3PlusPct":1.2},{"event":"Febrile neutropenia (with docetaxel)","grade3PlusPct":14}],"access":[],"regulatoryEvents":[{"date":"2012-06-08","type":"approval","region":"US","note":"First-line metastatic (CLEOPATRA)"},{"date":"2013-09-30","type":"accelerated-approval","region":"US","note":"Neoadjuvant accelerated approval on pCR","indication":"In combination with trastuzumab and docetaxel for neoadjuvant treatment of HER2-positive locally advanced inflammatory or early-stage breast cancer (either greater than 2 cm in diameter or node-positive) as part of a complete treatment regimen for early early breast cancer"},{"date":"2017-12-20","type":"conversion","region":"US","note":"Adjuvant (APHINITY)","indication":"In combination with trastuzumab and docetaxel for neoadjuvant treatment of HER2-positive locally advanced inflammatory or early-stage breast cancer (either greater than 2 cm in diameter or node-positive) as part of a complete treatment regimen for early early breast cancer"},{"date":"2020-06-29","type":"approval","region":"US","note":"Phesgo SC"},{"date":"2026-06-24","type":"approval","region":"US","note":"FDA approves palbociclib with trastuzumab, with or without pertuzumab, and endocrine therapy for the maintenance treatment of HR-positive, HER2-positive metastatic breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-palbociclib-trastuzumab-or-without-pertuzumab-and-endocrine-therapy-maintenance"}]},{"id":"petosemtamab","kind":"drug","name":"Petosemtamab","aka":[],"tldr":"A two-armed antibody that blocks EGFR while gripping LGR5, a marker of cancer stem cells, so it hits the cells that regrow tumours.","summary":"Petosemtamab is a Merus Biclonics bispecific antibody that blocks EGFR with one arm while gripping LGR5, a marker of cancer stem cells, with the other; LGR5 binding drives internalisation and degradation of EGFR, and an ADCC-enhanced Fc recruits immune killing. It is aimed at head and neck squamous cell carcinoma and colorectal cancer, given at 1500 mg every 2 weeks. Phase 2 with pembrolizumab in first-line PD-L1-positive HNSCC gave an ORR of about 63%, and monotherapy about 37% in second line, earning Breakthrough Therapy designation. The registrational LiGeR-HN1 (first line) and LiGeR-HN2 (second line) trials are enrolling, with interim analyses expected 2026 to 2027; Merus was acquired by Genmab (2025). Whether the single-arm response rates hold up in randomised comparison is the key uncertainty. For a newcomer, it is a two-armed antibody designed to hit the cells that regrow tumours.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT06525220","url":"https://clinicaltrials.gov/study/NCT06525220"}],"tags":[],"related":[],"cancers":["head-and-neck","colorectal","recurrent-metastatic-hnscc"],"sections":[],"technologies":["bispecific-antibody"],"targets":["egfr","lgr5"],"drugs":["pembrolizumab"],"companies":["merus","genmab"],"institutions":[],"pathways":[],"terms":[],"trials":["liger-hn1","nct07775287","nct06496178","nct07702032","nct03526835"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"MCLA-158","modality":"Bispecific antibody (EGFR × LGR5)","mechanism":"Simultaneous EGFR blockade and LGR5-mediated internalisation and degradation of EGFR; ADCC-enhanced Fc.","approvals":[],"mechanismSteps":["The LGR5 arm binds cancer stem-like cells","The EGFR arm blocks ligand binding and drives receptor internalisation","EGFR is degraded rather than merely blocked, silencing MAPK/PI3K signalling","Enhanced Fc recruits NK cells for ADCC; pembrolizumab sustains T-cell activity"],"dosing":{"route":"Intravenous","schedule":"1500 mg every 2 weeks (phase 3 dose)","monitoring":"Infusion reactions (premedication), rash, hypomagnesaemia","source":"https://clinicaltrials.gov/study/NCT06525220"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2024","type":"designation","region":"US","note":"Breakthrough Therapy designation for HNSCC"}]},{"id":"pevonedistat","kind":"drug","name":"Pevonedistat","aka":["TAK-924","MLN4924"],"tldr":"Pevonedistat was a first-in-class drug that jammed part of the cell's protein-disposal system. Promising with azacitidine in a mid-stage trial for higher-risk myelodysplastic syndromes, it failed to beat azacitidine alone in the phase 3 PANTHER trial in 2021.","summary":"Pevonedistat (TAK-924, MLN4924) was Takeda's inhibitor of the NEDD8-activating enzyme, the first drug in its class to reach late-stage trials. In the randomised phase 2 Pevonedistat-2001 study, adding it to azacitidine lengthened event-free survival and improved responses in higher-risk myelodysplastic syndromes, which prompted a phase 3 programme.\n\nThe phase 3 PANTHER trial in higher-risk myelodysplastic syndromes, chronic myelomonocytic leukaemia and low-blast acute myeloid leukaemia did not meet its primary endpoint of event-free survival when it reported in 2021, and Takeda ended development in these diseases. The higher-risk MDS page lists it with sabatolimab, magrolimab and eprenetapopt among the azacitidine partners that failed.","status":"negative","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Pevonedistat","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Pevonedistat"}],"tags":["subtype-drugs-wave"],"related":["azacitidine"],"cancers":["mds-higher-risk"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04090736","nct03268954","beat-aml-master-trial"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"TAK-924","modality":"Intravenous NEDD8-activating enzyme (NAE) inhibitor","mechanism":"Blocks the NEDD8-activating enzyme, which switches on cullin-RING ubiquitin ligases; without neddylation, proteins these ligases normally degrade accumulate, causing DNA re-replication and cell death in myeloid blasts.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pexidartinib","kind":"drug","name":"Pexidartinib","aka":[],"tldr":"Pexidartinib is the first drug for tenosynovial giant cell tumour, a benign but destructive joint tumour; it is effective but has liver toxicity that requires a restricted programme.","summary":"Pexidartinib is a small-molecule inhibitor of CSF1R, KIT and FLT3. In tenosynovial giant cell tumour, a benign but locally destructive joint tumour driven by CSF1 overproduction, blocking CSF1R depletes the macrophage-rich tumour mass. In ENLIVEN (Lancet 2019), 39% of patients responded at 25 weeks versus none on placebo, with improvements in joint function. The FDA approved it in 2019 for symptomatic disease not amenable to surgery, under a REMS programme because of mixed or cholestatic hepatotoxicity that requires liver monitoring, while the EMA refused approval in 2020 on the balance of benefit and liver risk. Vimseltinib, approved in 2025, offers a safer alternative in the same class. Pexidartinib was the first drug ever approved for this tumour and showed that a macrophage-driven tumour can be treated by starving it of macrophages.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pexidartinib","links":[{"label":"ENLIVEN (Lancet 2019)","url":"https://doi.org/10.1016/S0140-6736(19)30764-0"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pexidartinib"}],"tags":["gap-fill"],"related":[],"cancers":["sarcoma","tenosynovial-giant-cell-tumour"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["csf1r","kit"],"drugs":[],"companies":["daiichi-sankyo"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04488822"],"people":[],"bottlenecks":[],"keyPapers":["paper-tap-lancet"],"journals":[],"dependsOn":[],"notes":[],"brand":"Turalio","modality":"Small-molecule CSF1R/KIT/FLT3 inhibitor","mechanism":"Inhibits colony-stimulating factor 1 receptor, depleting the CSF1-driven macrophage-rich tumour of tenosynovial giant cell tumour.","approvals":[{"region":"US","year":2019,"indication":"Symptomatic tenosynovial giant cell tumour not amenable to surgery"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pf-07248144","kind":"drug","name":"PF-07248144","aka":[],"tldr":"PF-07248144 is an experimental small-molecule drug from Pfizer in phase 3 trials for HR-positive / HER2-negative breast cancer, prostate cancer and non-small-cell lung cancer, with its target not yet stated publicly.","summary":"PF-07248144 is a small-molecule drug developed by Pfizer. The sponsor describes its target as KAT6 (lysine acetyltransferase, e.g. KAT6A), which OnCo does not yet have a target page for. The sponsor states: PF-07248144 is a KAT6 inhibitor being studied as a single agent and in combination with fulvestrant, letrozole plus palbociclib, PF-07220060 (a CDK4 inhibitor) plus fulvestrant, or vepdegestrant, in HR-positive/HER2-negative breast cancer and other solid tumours. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07062965 (A Study to Learn About the Study Medicine Called PF-07248144 in Combination With Fulvestrant in People With HR-positive, HER2-negative Advanced or Metastatic Breast Cancer Who Progressed After a Prior Line of Treatment), in HR-positive / HER2-negative breast cancer, prostate cancer and non-small-cell lung cancer. The largest, NCT07062965, plans to enrol 400 participants with primary completion expected 2027-07-22. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of PF-07248144","url":"https://clinicaltrials.gov/search?intr=PF-07248144"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","prostate","nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07062965","nct04606446"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"PF-07248144 is a KAT6 inhibitor being studied as a single agent and in combination with fulvestrant, letrozole plus palbociclib, PF-07220060 (a CDK4 inhibitor) plus fulvestrant, or vepdegestrant, in HR-positive/HER2-negative breast cancer and other solid tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pf-08046054","kind":"drug","name":"PF-08046054","aka":["SGN-PDL1V"],"tldr":"PF-08046054 is an experimental antibody-drug conjugate from Pfizer in phase 3 trials for non-small-cell lung cancer, aimed at PD-L1.","summary":"PF-08046054 is an antibody-drug conjugate developed by Pfizer. Its target is PD-L1 (the sponsor names PD-L1). ClinicalTrials.gov describes the intervention as: Antibody Drug Conjugate Participants will receive PF-08046054, administered as an IV infusion. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07144280 (A Study to Learn About the Study Medicine Called PF-08046054/SGN-PDL1V Versus Docetaxel in Adult Participants With Previously-Treated Programmed Cell Death Ligand 1 (PD-L1) Positive Non-Small-Cell Lung Cancer (NSCLC)), in non-small-cell lung cancer. The largest, NCT07144280, plans to enrol 680 participants with primary completion expected 2028-03-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of PF-08046054","url":"https://clinicaltrials.gov/search?intr=PF-08046054"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["pdl1"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nsclc-4","nct07227298"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Antibody-drug conjugate directed at PD-L1, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pf-08634404","kind":"drug","name":"PF-08634404","aka":[],"tldr":"PF-08634404 is an experimental investigational agent whose form is not stated in the registry from Pfizer in phase 3 trials for colorectal cancer, gastric & gastro-oesophageal junction cancer and oesophageal cancer, with its target not yet stated publicly.","summary":"PF-08634404 (SSGJ-707) is an investigational agent whose form is not stated in the registry developed by Pfizer and Sunshine Guojian Pharmaceutical (Shanghai). Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 13 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07222800 (Symbiotic-GI-03: A Study to Learn About the Study Medicine Called PF-08634404 in Combination With Chemotherapy in Adult Participants With Metastatic Colorectal Cancer), NCT07392892 (Symbiotic-GI-16: A Study to Learn About the Study Medicine Called PF-08634404 in Combination With Chemotherapy in Gastroesophageal Cancer) and NCT07226999 (Symbiotic-Lung-04: A Study to Learn About the Study Medicine Called PF-08634404 in Combination With Chemotherapy in Adult Participants With Extensive-Stage Small Cell Lung Cancer), in colorectal cancer, gastric & gastro-oesophageal junction cancer, oesophageal cancer, non-small-cell lung cancer and bladder & urothelial cancer. The largest, NCT07222566, plans to enrol 1410 participants with primary completion expected 2029-02-27. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of PF-08634404","url":"https://clinicaltrials.gov/search?intr=SSGJ-707"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal","gastric","esophageal","nsclc","urothelial","hcc","rcc","sclc","ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["symbiotic-gi-03","symbiotic-gi-16","symbiotic-lung-04","symbiotic-lung-01","nct06980272","nct07227012","nct06522828","nct07476287","nct07227415","nct07227298","nct07489066","nct06493760","nct07421700"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"SSGJ-707","modality":"bispecific antibody (PD-1 x VEGF, per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"phosphorus-32","kind":"drug","name":"Phosphorus-32 (radiophosphorus)","aka":["Radiophosphorus","Sodium phosphate P-32","32P"],"tldr":"Radiophosphorus was one of the first treatments for polycythaemia vera, from the 1940s: a radioactive phosphate injection that quietened the overactive bone marrow for a year or more. It was abandoned when trials showed it raised the risk of leukaemia.","summary":"Phosphorus-32 was introduced by John Lawrence at Berkeley in the late 1930s as the first therapeutic use of an artificial radionuclide, and became a standard treatment for polycythaemia vera and essential thrombocythaemia. A single intravenous dose controlled blood counts for one to two years and was convenient for older patients.\n\nThe Polycythemia Vera Study Group's first randomised trial, launched in 1967, compared phlebotomy alone, phlebotomy plus radiophosphorus and phlebotomy plus chlorambucil, and showed that both radiophosphorus and chlorambucil raised the risk of acute leukaemia several-fold. Radiophosphorus was thereafter restricted to the very elderly and is now rarely used, replaced by hydroxyurea, interferon and JAK inhibitors. The polycythaemia vera page records it in the history of the disease.","status":"historic","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Phosphorus-32","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Phosphorus-32"}],"tags":["subtype-drugs-wave"],"related":["hydroxyurea","chlorambucil"],"cancers":["polycythaemia-vera"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Intravenous radiopharmaceutical (beta emitter; historic)","mechanism":"Phosphate labelled with the beta emitter phosphorus-32 is taken up by rapidly dividing marrow cells and incorporated into their DNA, where the radiation suppresses the overactive marrow of polycythaemia vera.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pylarify","kind":"drug","name":"Piflufolastat F-18 / Pylarify TruVu","aka":["Piflufolastat (18F)","Pylclari"],"tldr":"Piflufolastat F-18 (Pylarify) is the leading PSMA PET tracer for prostate cancer, with a new formulation approved in March 2026.","summary":"Piflufolastat F-18 is a PET tracer: an 18F-labelled urea that binds prostate-specific membrane antigen (PSMA), lighting up prostate cancer deposits that conventional CT and bone scan miss. It was approved in 2021 for PSMA PET in initial staging and biochemical recurrence, and Lantheus' Pylarify TruVu formulation, approved on 9 March 2026, offers an improved formulation and distribution profile. A single 333 MBq dose is injected and imaging follows 60 to 120 minutes later; adverse effects are minor. The 18F label allows central manufacture and shipping, unlike generator-produced 68Ga agents, and it competes with Illuccix/Gozellix (68Ga, Telix), Locametz (Novartis) and Posluma. The open question is whether the management changes PSMA PET triggers translate into longer survival. For a newcomer: the leading tracer for seeing where prostate cancer actually is.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Piflufolastat_F-18","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Piflufolastat%20F-18"}],"tags":[],"related":[],"cancers":["prostate"],"sections":[],"technologies":["psma-pet"],"targets":["psma"],"drugs":[],"companies":["lantheus"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03739684","nct04457245","nct02981368"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Pylarify","code":"18F-DCFPyL","modality":"PET imaging agent","mechanism":"18F-labelled PSMA-binding urea.","approvals":[{"region":"US","year":2021,"indication":"PSMA PET imaging in prostate cancer"},{"region":"US","year":2026,"indication":"Pylarify TruVu formulation"},{"region":"EU","year":2023,"indication":"EU brand Pylclari (Curium)"}],"mechanismSteps":["18F-labelled small molecule binds the PSMA active site","Tracer is internalised and retained in prostate cancer cells","Positron emissions are detected by the PET scanner","Whole-body map of PSMA-expressing disease is produced","Result selects patients for PSMA radioligand therapy"],"dosing":{"route":"IV injection (diagnostic)","schedule":"333 MBq (9 mCi) single dose; imaging 60-120 minutes later","monitoring":"Hydration and voiding to reduce bladder dose","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Headache","note":"Rare; diagnostic dose"},{"event":"Dysgeusia"},{"event":"Fatigue"}],"access":[{"country":"US","reimbursement":"Medicare pass-through/payment for PSMA PET; broad commercial coverage for staging and biochemical recurrence","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-05-26","type":"approval","region":"US","note":"PSMA PET imaging in prostate cancer: first 18F PSMA agent","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-03-09","type":"approval","region":"US","note":"Pylarify TruVu formulation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"pilocarpine","kind":"drug","name":"Pilocarpine","aka":["Salagen"],"tldr":"Pilocarpine tablets are approved to treat the dry mouth that follows head and neck radiotherapy; they help when some gland tissue is left, at the cost of sweating and other cholinergic side effects.","summary":"Pilocarpine, a plant alkaloid long used as eye drops for glaucoma, was approved by the FDA in 1994 (Salagen) for radiation-induced xerostomia in head and neck cancer, and later for Sjögren's syndrome. It works only where functioning salivary tissue remains, so it is most useful after modern parotid-sparing radiotherapy; sweating, flushing and urinary frequency limit the dose. Trials of pilocarpine during radiotherapy to protect the glands have been inconsistent.","status":"approved","asOf":"2026-09-16","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Pilocarpine"}],"tags":["radiation-wave3"],"related":[],"cancers":["head-and-neck","nasopharyngeal"],"sections":[],"technologies":["radioprotectors"],"targets":[],"drugs":[],"companies":["eisai"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00343382","nct00003139","nct00168181"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo radiation expansion","editedOn":"2026-09-16"},"modality":"Cholinergic agonist (small molecule) for radiation-induced dry mouth","supportive":true,"mechanism":"Stimulates muscarinic receptors on whatever salivary gland tissue survives radiotherapy, increasing saliva flow.","approvals":[{"region":"US","year":1994,"indication":"Dry mouth caused by radiotherapy for head and neck cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pimicotinib","kind":"drug","name":"Pimicotinib","aka":[],"tldr":"Pimicotinib is an oral kinase inhibitor from Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany, in registered phase 2 trials for tenosynovial giant cell tumour.","summary":"Pimicotinib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany, in tenosynovial giant cell tumour. A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Pimicotinib","url":"https://clinicaltrials.gov/search?intr=Pimicotinib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["tenosynovial-giant-cell-tumour"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["abbisko"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07499362"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral kinase inhibitor","mechanism":"A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pipobroman","kind":"drug","name":"Pipobroman","aka":[],"tldr":"Pipobroman was an oral tablet approved in 1966 for polycythaemia vera, the blood disorder that makes too many red cells, and for chronic myeloid leukaemia; it was discontinued after studies showed it raised the risk of leukaemia compared with hydroxycarbamide.","summary":"Pipobroman controlled red cell counts in polycythaemia vera for decades, especially in France and Italy, and was approved in the United States in 1966 for polycythaemia vera and for chronic myeloid leukaemia after busulfan failure. The French Polycythemia Study Group's randomised comparison with hydroxycarbamide, followed for a median of 16 years, found more transformations to acute leukaemia and shorter survival with pipobroman, which ended its use. Hydroxycarbamide, interferon and now ruxolitinib are the cytoreductive options in polycythaemia vera.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pipobroman","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Pipobroman"},{"label":"ChEMBL CHEMBL1201205","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201205"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["myeloproliferative-neoplasms","cml"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":["hydroxyurea","ruxolitinib"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["response"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vercyte","modality":"Oral alkylating-type cytotoxic","mechanism":"A piperazine derivative with two bromopropionyl groups that acts as an alkylating agent on proliferating marrow cells; its precise mechanism was never fully defined.","approvals":[{"region":"US","year":1966,"indication":"Polycythaemia vera; chronic myeloid leukaemia refractory to busulfan (historic label)","note":"Discontinued"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pirarubicin","kind":"drug","name":"Pirarubicin","aka":["Pinorubin","THP-adriamycin"],"tldr":"Pirarubicin is a doxorubicin relative developed in Japan and used there and in China for breast cancer, lymphoma, bladder cancer and other tumours, including instillation into the bladder after tumour resection.","summary":"Pirarubicin (THP) was developed in Japan in the 1980s as a less cardiotoxic anthracycline and is approved in Japan and China for a range of cancers including breast cancer, lymphomas, head and neck, ovarian and bladder cancer, where it is given intravesically after transurethral resection. It has never been approved in the United States or Europe. A current Chinese phase 3 trial tests it as the intravesical comparator in non-muscle-invasive bladder cancer.","status":"established","asOf":"2026-09-16","wikipedia":"https://en.wikipedia.org/wiki/Pirarubicin","links":[{"label":"ClinicalTrials.gov: trials of Pirarubicin","url":"https://clinicaltrials.gov/search?intr=Pirarubicin"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial","breast-hr-positive"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["top2a"],"drugs":[],"companies":["nippon-kayaku"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07424287"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"modality":"Anthracycline (topoisomerase II inhibitor), intravenous or intravesical","mechanism":"A doxorubicin analogue with a tetrahydropyranyl sugar that enters cells faster and causes less cardiotoxicity in animal models.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pirtobrutinib","kind":"drug","name":"Pirtobrutinib","aka":[],"tldr":"A BTK blocker that works after the older ones stop, because it grips a different part of the enzyme. Fully approved for CLL in December 2025.","summary":"Binds BTK reversibly and is active against C481 mutations. BRUIN CLL-321 (n=238, post-covalent BTKi): PFS 11.2 vs 8.7 months vs idelalisib-rituximab or bendamustine-rituximab (HR 0.58), leading to traditional approval on 3 December 2025 after the December 2023 accelerated approval. Also approved in MCL (2023). Frontline trials (BRUIN CLL-313 vs bendamustine-rituximab, CLL-314 vs ibrutinib) and combination with venetoclax (CLL-322) are reading out 2026-27. Resistance via T474I and L528W confers cross-resistance to some covalent inhibitors.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Pirtobrutinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pirtobrutinib"},{"label":"Woyach et al., N Engl J Med 2014: BTK C481S and PLCG2 resistance mutations under ibrutinib","url":"https://doi.org/10.1056/NEJMoa1400029"},{"label":"Mato et al., N Engl J Med 2023: pirtobrutinib after a covalent BTK inhibitor (BRUIN, 317 patients)","url":"https://doi.org/10.1056/NEJMoa2300696"},{"label":"Blombery et al., Blood Adv 2022: enrichment of BTK Leu528Trp on zanubrutinib and cross-resistance to pirtobrutinib","url":"https://doi.org/10.1182/bloodadvances.2022008325"}],"tags":[],"related":[],"cancers":["cll","dlbcl","cll-relapsed","richter-transformation-cll"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["btk"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":["richter-transformation"],"trials":["bruin-cll-321","nct07162181","nct06973187","nct06588478","nct05023980","nct05254743","nct04965493","nct04662255"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: a non-covalent inhibitor exists because of one amino acid. Covalent BTK inhibitors bind cysteine 481, and the C481S substitution leaves the kinase working while making the inhibition reversible (Woyach 2014); pirtobrutinib does not need that cysteine and gave an overall response of 73.3% in 247 patients who had already had a covalent inhibitor (Mato 2023). It is not a universal answer: the kinase-dead L528W substitution, enriched after zanubrutinib at 7 of 13 progressing patients against 1 of 24 after ibrutinib, confers cross-resistance and was enriched further under pirtobrutinib (Blombery 2022)."],"brand":"Jaypirca","modality":"Small-molecule non-covalent (reversible) BTK inhibitor","mechanism":"Non-covalent, highly selective BTK inhibitor occupying the ATP site independent of C481, with a long half-life for continuous occupancy.","approvals":[{"region":"US","year":2023,"indication":"Relapsed MCL after BTK inhibitor (accelerated, January); CLL/SLL after BTK and BCL-2 inhibitors (accelerated, December)"},{"region":"US","year":2025,"indication":"Relapsed/refractory CLL/SLL after a covalent BTK inhibitor (traditional approval, BRUIN CLL-321)"}],"mechanismSteps":["Pirtobrutinib binds the BTK ATP pocket reversibly and tightly","Works whether or not C481 is mutated","BCR signalling is blocked; CLL cells lose survival cues","Once-daily dosing maintains >90% occupancy through the dosing interval"],"dosing":{"route":"Oral","schedule":"200 mg once daily continuously","monitoring":"Infections, bleeding, cytopenias, atrial fibrillation (low), second primary malignancies","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Jaypirca"},"toxicity":[{"event":"Fatigue","anyGradePct":29,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Jaypirca"},{"event":"Neutropenia","grade3PlusPct":16},{"event":"Atrial fibrillation","anyGradePct":3.7},{"event":"Bruising","anyGradePct":24}],"access":[],"regulatoryEvents":[{"date":"2023-01-27","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 3.6 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-pirtobrutinib-relapsed-or-refractory-mantle-cell-lymphoma","indication":"Adult patients with relapsed or refractory mantle cel lymphoma (MCL) after at least two lines of systemic therapy, including a BTK inhibitor."},{"date":"2023-12-01","type":"accelerated-approval","region":"US","note":"CLL/SLL accelerated approval","indication":"Adult patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor"},{"date":"2025-12-02","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2023 converted to traditional approval 2.0 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-pirtobrutinib-chronic-lymphocytic-leukemia-and-small-lymphocytic","indication":"Adult patients with chronic lymphocytic leukemia or small lymphocytic lymphoma (CLL/SLL) who have received at least two prior lines of therapy, including a BTK inhibitor and a BCL-2 inhibitor"},{"date":"2025-12-03","type":"approval","region":"US","note":"Traditional approval; label broadened to any prior covalent BTKi","source":"https://ascopost.com/news/december-2025/fda-grants-traditional-approval-to-pirtobrutinib-for-cllsll/"}]},{"id":"pivekimab-sunirine","kind":"drug","name":"Pivekimab sunirine","aka":[],"tldr":"Pivekimab sunirine is an antibody-drug conjugate against CD123, approved in 2026 for blastic plasmacytoid dendritic cell neoplasm as the second targeted drug for this rare cancer.","summary":"Pivekimab sunirine is an antibody-drug conjugate: an anti-CD123 antibody joined by a cleavable linker to an indolinobenzodiazepine pseudodimer (IGN) payload that alkylates DNA without cross-linking. CD123 is highly expressed on blastic plasmacytoid dendritic cell neoplasm, a rare and aggressive haematological cancer. The CADENZA pivotal study in untreated BPDCN showed high complete response rates with fewer capillary-leak events than tagraxofusp, the first CD123-directed drug, and the FDA approved it in May 2026, making it the second targeted therapy for this disease. It is also being studied in CD123-positive AML in combination with azacitidine and venetoclax. Whether it improves long-term survival and how it should be sequenced with transplant remain open. For a newcomer, it delivers a DNA-damaging payload directly to cells that carry the CD123 marker.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pivekimab_sunirine","links":[{"label":"FDA novel approvals 2026","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2026"}],"tags":["gap-fill"],"related":[],"cancers":["bpdcn","aml"],"sections":[],"technologies":["adc"],"targets":["cd123"],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03386513","nct07581002","nct04086264"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Decnupaz","code":"IMGN632","modality":"ADC (anti-CD123 antibody, IGN payload)","mechanism":"Anti-CD123 antibody conjugated via a cleavable linker to an indolinobenzodiazepine pseudodimer (IGN) that alkylates DNA without cross-linking.","approvals":[{"region":"US","year":2026,"indication":"Blastic plasmacytoid dendritic cell neoplasm"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-05-27","type":"approval","region":"US","note":"FDA approves pivekimab sunirine-pvzy for blastic plasmacytoid dendritic cell neoplasm, an ultra-rare hematologic malignancy","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pivekimab-sunirine-pvzy-blastic-plasmacytoid-dendritic-cell-neoplasm-ultra-rare"}]},{"id":"pixantrone","kind":"drug","name":"Pixantrone","aka":["BBR 2778","Pixantrone dimaleate"],"tldr":"Pixantrone is an anthracycline-like drug engineered to spare the heart. The European Union authorised it in 2012 as a single agent for adults with aggressive B-cell lymphoma that had relapsed several times; the authorisation expired in June 2024 when the company did not renew it.","summary":"Pixantrone was granted conditional marketing authorisation in the European Union in 2012 for adults with multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphoma, based on the PIX301 trial in which it produced more complete responses than other single agents. Because it lacks the anthracycline quinone, it causes less cardiotoxicity than doxorubicin in animal models, which allows treatment of patients who have already had a full lifetime dose of anthracyclines. The FDA did not approve it. The EU authorisation stayed conditional and expired on 12 June 2024 after Les Laboratoires Servier chose not to apply for renewal, citing lack of demand, so pixantrone is no longer authorised anywhere.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pixantrone","links":[{"label":"EMA product page","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/pixuvri"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Pixantrone"},{"label":"ChEMBL CHEMBL1201855","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201855"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["top2a"],"drugs":["doxorubicin"],"companies":["servier"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01086605","nct03458260"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Pixuvri","modality":"Aza-anthracenedione cytotoxic, intravenous","mechanism":"A DNA intercalator and topoisomerase II poison designed to keep the antitumour activity of the anthracyclines while removing the iron-binding quinone that drives their cardiotoxicity.","approvals":[{"region":"EU","year":2012,"indication":"Multiply relapsed or refractory aggressive non-Hodgkin B-cell lymphoma, single agent (conditional)","note":"Authorisation expired 12 June 2024; the holder did not apply for renewal"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"platinum-etoposide","kind":"drug","name":"Platinum + etoposide (EP / CE)","aka":[],"tldr":"Platinum plus etoposide has been the chemotherapy backbone of small-cell lung cancer for over 40 years, and is now given with immunotherapy.","summary":"Cisplatin or carboplatin with etoposide for 4 (extensive-stage) to 4-6 (limited-stage, with concurrent radiotherapy) cycles. Response rates 60-80% but relapse is near-universal in extensive-stage disease. Carboplatin is preferred with immunotherapy and in frailer patients; cisplatin with concurrent thoracic radiotherapy in fit limited-stage patients.","status":"standard-of-care","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Etoposide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Etoposide"}],"tags":[],"related":[],"cancers":["sclc","limited-stage-sclc","extensive-stage-sclc","extrapulmonary-nec"],"sections":[],"technologies":["cytotoxic-chemotherapy","platinum","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["limited-extensive-stage"],"trials":["impower133","caspian","astrum-005"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Cytotoxic regimen","mechanism":"Platinum DNA crosslinks plus topoisomerase-II inhibition by etoposide.","approvals":[{"region":"US","year":1983,"indication":"Etoposide approved for SCLC; platinum-etoposide became standard in the 1980s"}],"mechanismSteps":["Platinum forms intrastrand DNA crosslinks","Etoposide traps topoisomerase II on DNA, causing double-strand breaks","Rapidly dividing SCLC cells (near-universal RB1 and TP53 loss) cannot arrest and undergo apoptosis","Surviving clones re-emerge within months, often with lineage plasticity"],"dosing":{"route":"Intravenous","schedule":"Carboplatin AUC 5 day 1 (or cisplatin 75 mg/m² day 1) + etoposide 100 mg/m² days 1-3, every 21 days, 4 cycles","modifications":"Dose reductions for neutropenia; G-CSF support with concurrent radiotherapy is avoided","monitoring":"Blood counts each cycle; renal function and hearing with cisplatin"},"toxicity":[{"event":"Neutropenia (grade 3+)","grade3PlusPct":40,"note":"Range across trials 23-45%"},{"event":"Anaemia","anyGradePct":40},{"event":"Nausea","anyGradePct":50},{"event":"Alopecia","anyGradePct":60}],"access":[],"regulatoryEvents":[]},{"id":"plerixafor","kind":"drug","name":"Plerixafor","aka":["AMD3100"],"tldr":"Plerixafor (Mozobil) is an injection given with G-CSF to flush stem cells out of the bone marrow into the blood so that enough can be collected for an autologous transplant in myeloma or lymphoma.","summary":"Plerixafor was approved by the FDA in December 2008 in combination with filgrastim to mobilise haematopoietic stem cells for collection and autologous transplantation in non-Hodgkin lymphoma and multiple myeloma, on two placebo-controlled phase 3 trials in which the proportion of patients reaching the target cell dose in few apheresis sessions roughly doubled; the EU authorised Mozobil in 2009 and a paediatric extension followed. It is used routinely for poor mobilisers and increasingly pre-emptively, and the same CXCR4 blockade underlies its investigation to mobilise stem cells for gene therapy and to sensitise leukaemia to chemotherapy. Motixafortide (Aphexda, 2023) is a longer-acting CXCR4 antagonist for the same purpose. Diarrhoea, nausea and injection-site reactions are common.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Plerixafor","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=plerixafor"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/plerixafor"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/mozobil"}],"tags":["nci-list","supportive"],"related":["filgrastim"],"cancers":["multiple-myeloma","dlbcl","follicular-lymphoma","mantle-cell-lymphoma"],"sections":[],"technologies":["autologous-stem-cell-transplant","apheresis-starting-material"],"targets":["cxcr4"],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00103662","nct01301963","nct01767714"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mozobil","modality":"Small-molecule CXCR4 antagonist (stem cell mobiliser)","supportive":true,"mechanism":"Bicyclam that reversibly blocks CXCR4 binding of stromal-derived factor-1 (CXCL12), releasing haematopoietic stem cells from bone marrow niches into the blood within hours.","approvals":[{"region":"US","year":2008,"indication":"Stem cell mobilisation with filgrastim for autologous transplantation in NHL and multiple myeloma"},{"region":"EU","year":2009,"indication":"Stem cell mobilisation with G-CSF in lymphoma and multiple myeloma with poor mobilisation (Mozobil)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"plicamycin","kind":"drug","name":"Plicamycin","aka":["Mithramycin","Aureolic acid"],"tldr":"Plicamycin, approved in 1970, was a chemotherapy for testicular cancer that fell out of use as cisplatin arrived, and was kept for treating dangerously high calcium levels caused by cancer until it was discontinued in 2000.","summary":"Mithramycin came from Streptomyces plicatus and was approved by the FDA in 1970 for disseminated testicular tumours not amenable to surgery or radiotherapy, and for hypercalcaemia and hypercalciuria of malignancy, where a single low dose lowered calcium within a day or two by suppressing osteoclasts. Bleeding from thrombocytopenia and platelet dysfunction and liver and kidney toxicity limited it, cisplatin-based combinations made it obsolete in germ cell tumours, and bisphosphonates replaced it for hypercalcaemia. Pfizer discontinued it in 2000. It has returned to research as a selective Sp1 inhibitor in Ewing sarcoma and other cancers.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Plicamycin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Plicamycin"},{"label":"ChEMBL CHEMBL1200937","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200937"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["testicular","ewing-sarcoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct01624090"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mithracin","modality":"Aureolic acid antibiotic cytotoxic, intravenous","mechanism":"Binds GC-rich DNA and displaces the transcription factor Sp1, silencing genes including those that drive osteoclast activity and cancer cell growth.","approvals":[{"region":"US","year":1970,"indication":"Disseminated testicular tumours; hypercalcaemia and hypercalciuria of malignancy","note":"Discontinued in 2000"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"plitidepsin","kind":"drug","name":"Plitidepsin","aka":["Dehydrodidemnin B"],"tldr":"Plitidepsin is a chemotherapy derived from a Mediterranean sea squirt, approved in Australia in 2018 with dexamethasone for multiple myeloma after several other treatments; the European regulator refused it, and it drew attention in 2021 as a possible COVID-19 antiviral.","summary":"PharmaMar isolated plitidepsin from the tunicate Aplidium albicans. In the phase 3 ADMYRE trial in relapsed and refractory multiple myeloma, plitidepsin with dexamethasone improved progression-free survival over dexamethasone alone by about a month, and the Therapeutic Goods Administration approved it in Australia in December 2018 for patients who had received at least three prior regimens including a proteasome inhibitor and an immunomodulator. The European Medicines Agency refused marketing authorisation in 2018, judging the benefit too small against the toxicity, and the FDA never approved it. Its inhibition of eEF1A2 also blocks SARS-CoV-2 replication in cells, which led to COVID-19 trials.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Plitidepsin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Plitidepsin"},{"label":"ChEMBL CHEMBL505124","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL505124"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["eef2"],"drugs":["dexamethasone"],"companies":["pharmamar"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01102426","nct00229203","nct01876043"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Aplidin","modality":"Marine-derived cyclic depsipeptide (small molecule), intravenous","mechanism":"Binds the translation elongation factor eEF1A2, triggering oxidative stress and rapid apoptosis in myeloma cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pm8002","kind":"drug","name":"PM8002","aka":["Pumitamig"],"tldr":"PM8002 is an experimental bispecific antibody from Biotheus in phase 3 trials for small-cell lung cancer, triple-negative breast cancer and neuroendocrine tumours, aimed at PD-L1 and VEGF / VEGFR.","summary":"PM8002 (BNT327) is a bispecific antibody developed by Biotheus. Its targets are PD-L1 and VEGF / VEGFR (the sponsor names PD-L1 x VEGF). The sponsor states: Study brief summary states PM8002 is a bispecific antibody targeting PD-L1 and VEGF; no further mechanistic detail given. ClinicalTrials.gov describes the intervention as: PM8002 20 mg/kg via intravenous (IV) infusion on Days 1 and 15 of each 28-day cycle. It is the investigational product in 8 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06616532 (PM8002 in Combination With Paclitaxel Compared With Chemotherapy as Second-line Treatment in Small Cell Lung Cancer) and NCT06419621 (PM8002 or Placebo Plus Nab-Paclitaxel as First-line Treatment in Inoperable Locally Advanced/Metastatic TNBC), in small-cell lung cancer, triple-negative breast cancer, neuroendocrine tumours, colorectal cancer and non-small-cell lung cancer. The largest, NCT06616532, plans to enrol 404 participants with primary completion expected 2027-01-25. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of PM8002","url":"https://clinicaltrials.gov/search?intr=BNT327"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["sclc","tnbc","neuroendocrine","colorectal","nsclc"],"sections":[],"technologies":[],"targets":["pdl1","vegf"],"drugs":[],"companies":["biontech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06616532","nct06419621","nct06712355","nct07221357","nct07361510","nct06712316","nct07361497","nct07111520","nct07079631","nct04895709","nct07325136","nct07680764","nct07291076","nct07133750","nct07255404","nct07293351","nct06841055","nct07070232","nct07160725","nct05844150","nct07147348","nct07297212","nct07492680","nct07221149","nct07173751","nct06827236","nct05438329","nct06892548","nct06618287","nct05918133","nct05879055","nct05879068","nct05918445","nct06953089"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"BNT327","modality":"bispecific antibody","mechanism":"Study brief summary states PM8002 is a bispecific antibody targeting PD-L1 and VEGF; no further mechanistic detail given.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"polatuzumab-vedotin","kind":"drug","name":"Polatuzumab vedotin","aka":[],"tldr":"Polatuzumab vedotin is an ADC against CD79b that, swapped into the classic R-CHOP regimen, became the first improvement on frontline lymphoma therapy in twenty years.","summary":"POLARIX (2022): Pola-R-CHP versus R-CHOP in untreated DLBCL, IPI 2-5. Two-year PFS 76.7% vs 70.2% (HR 0.73); five-year PFS 64.9% vs 59.1% (HR 0.77) with OS HR 0.85, not significant. Approved US April 2023 for frontline DLBCL (IPI ≥2) and since 2019 with bendamustine-rituximab for relapsed disease. Peripheral neuropathy is the class toxicity.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Polatuzumab_vedotin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Polatuzumab%20vedotin"},{"label":"Davis et al., Nature 2010: chronic active B-cell receptor signalling in diffuse large B-cell lymphoma","url":"https://doi.org/10.1038/nature08638"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["adc"],"targets":["cd79b"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["payload","bystander-effect"],"trials":["polarix","nct04844866","nct03671018","nct03533283","nct06890884"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: CD79b is chosen not because it is more specific than CD20 but because it internalises. It is part of the B-cell receptor complex, which is continuously taken into the cell, so it can carry monomethyl auristatin E inside; CD20 cannot. Expression is above 95% in diffuse large B-cell lymphoma, so nothing is tested before the first dose, and the dose-limiting toxicity is the payload, as cumulative peripheral neuropathy, rather than the antigen (Davis 2010)."],"brand":"Polivy","modality":"ADC","payload":"MMAE (tubulin inhibitor), DAR ~3.5","linker":"mc-vc-PABC, protease-cleavable","mechanism":"Humanised anti-CD79b IgG1 internalised via the B-cell receptor complex; MMAE released by cathepsin B; bystander killing.","approvals":[{"region":"US","year":2019,"indication":"Relapsed/refractory DLBCL with bendamustine-rituximab after ≥2 lines (accelerated)"},{"region":"US","year":2023,"indication":"Untreated DLBCL, IPI ≥2, with R-CHP (Pola-R-CHP)"}],"mechanismSteps":["Antibody binds CD79b on the B-cell receptor complex","Complex internalised and trafficked to lysosome","Cathepsin cleaves the vc linker, freeing MMAE","MMAE disrupts microtubules; mitotic arrest and apoptosis","Permeable MMAE diffuses to neighbouring cells"],"dosing":{"route":"IV","schedule":"1.8 mg/kg every 21 days for 6 cycles with R-CHP (frontline) or with BR (relapsed)","modifications":"Reduce to 1.4 mg/kg for grade 2-3 neuropathy; discontinue for grade 4","monitoring":"Neuropathy, neutropenia, infections, infusion reactions","source":"https://www.gene.com/download/pdf/polivy_prescribing.pdf"},"toxicity":[{"event":"Peripheral neuropathy","anyGradePct":52.9,"grade3PlusPct":1.6,"note":"POLARIX Pola-R-CHP arm"},{"event":"Neutropenia","grade3PlusPct":28.3},{"event":"Febrile neutropenia","grade3PlusPct":13.8},{"event":"Diarrhoea","anyGradePct":30.8}],"access":[],"regulatoryEvents":[{"date":"2019-06-10","type":"accelerated-approval","region":"US","note":"Accelerated approval with BR in R/R DLBCL","indication":"In combination with bendamustine and a rituximab product for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, after at least two prior therapies."},{"date":"2023-04-19","type":"conversion","region":"US","note":"Frontline Pola-R-CHP (POLARIX)","indication":"In combination with bendamustine and a rituximab product for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, after at least two prior therapies."}]},{"id":"pomalidomide","kind":"drug","name":"Pomalidomide","aka":[],"tldr":"The third-generation thalidomide analogue for myeloma that has failed lenalidomide, and since 2020 the first new drug for Kaposi sarcoma in two decades.","summary":"Pomalidomide, the third-generation thalidomide analogue, binds cereblon to trigger degradation of IKZF1 and IKZF3, giving direct anti-myeloma, immunostimulatory and anti-angiogenic effects that persist after lenalidomide failure. MM-003 (2013) showed an overall survival benefit versus high-dose dexamethasone in refractory myeloma, and US and EU approval followed in 2013 after at least two therapies including lenalidomide and a proteasome inhibitor. It anchors triplets such as isatuximab-Pd (ICARIA), elotuzumab-Pd and daratumumab-Pd (APOLLO). In 2020 it was approved for Kaposi sarcoma, both AIDS-related after antiretroviral failure and HIV-negative, on a 71% response rate, the first new Kaposi drug in two decades. Neutropenia and thrombosis risk need monitoring. Pomalidomide is the immunomodulator that keeps working after lenalidomide stops.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pomalidomide","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pomalidomide"}],"tags":["gap-fill"],"related":[],"cancers":["multiple-myeloma","kaposi-sarcoma","myeloma-relapsed-refractory"],"sections":[],"technologies":["protac-degrader"],"targets":["ikzf1","ikzf3"],"drugs":[],"companies":["bms","natco"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05730036","nct06464991","nct06956170","nct05438043","nct05572515","nct06152575","nct03539744","nct05519085","nct06413498","nct06158841","nct05020236","nct06208150","nct05455320","nct02343042","nct04068597","nct06953960","nct07227311","nct07018050","nct06106945","nct05714839","nct04973605","nct07150104","nct07742215"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Pomalyst / Imnovid","modality":"Oral cereblon E3 ligase modulator (IMiD)","mechanism":"Binds cereblon to induce degradation of IKZF1/3, with direct anti-myeloma, immunomodulatory and anti-angiogenic effects; active after lenalidomide failure.","approvals":[{"region":"US","year":2013,"indication":"Multiple myeloma after ≥2 therapies including lenalidomide and a proteasome inhibitor"},{"region":"US","year":2020,"indication":"Kaposi sarcoma (AIDS-related after ART failure; HIV-negative)"},{"region":"EU","year":2013,"indication":"Relapsed/refractory multiple myeloma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2013-02-08","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Multiple myeloma after at least 2 prior therapies including lenalidomide and bortezomib and disease progression on or within 60 days of completion of the last therapy"},{"date":"2015-04-23","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2013 converted to traditional approval 2.2 years after it was granted.","indication":"Multiple myeloma after at least 2 prior therapies including lenalidomide and bortezomib and disease progression on or within 60 days of completion of the last therapy"},{"date":"2020-05-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 6.3 years later, when the FDA's table was read.","indication":"Treatment of patients with AIDS-related Kaposi’s Sarcoma after failure of highly active antiretroviral therapy (HAART)."},{"date":"2020-05-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 6.3 years later, when the FDA's table was read.","indication":"Treatment of Kaposi’s Sarcoma in patients who are HIV-negative"}]},{"id":"ponatinib","kind":"drug","name":"Ponatinib","aka":[],"tldr":"Ponatinib is the only BCR::ABL1 inhibitor that covers the T315I resistance mutation. In 2024 it became the preferred pill for newly diagnosed Ph-positive ALL.","summary":"PhALLCON (n=245): ponatinib vs imatinib with reduced-intensity chemotherapy in newly diagnosed Ph+ ALL, MRD-negative CR at end of induction 34.4% vs 16.7%; accelerated approval March 2024. Also approved in CML (T315I and resistant disease) since 2012, with a 2013 temporary market suspension for arterial occlusive events that led to dose-reduction strategies (OPTIC). Ponatinib + blinatumomab chemotherapy-free regimens (MD Anderson) report near-universal molecular remission without transplant.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Ponatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ponatinib"}],"tags":[],"related":["bcr-abl1-t315i"],"cancers":["all-leukemia","cml-advanced-phase"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["bcr-abl","abl1"],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":["ph-positive-all"],"trials":["phallcon","nct04233346","nct03934372"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Iclusig","modality":"Small-molecule kinase inhibitor (pan-BCR::ABL1 incl. T315I)","mechanism":"Type II TKI binding inactive ABL1 with a carbon-carbon triple bond that accommodates the T315I gatekeeper; also inhibits FGFR, VEGFR, SRC.","approvals":[{"region":"US","year":2012,"indication":"CML and Ph+ ALL resistant to prior TKIs or with T315I"},{"region":"US","year":2024,"indication":"Newly diagnosed Ph+ ALL with chemotherapy (accelerated)"}],"mechanismSteps":["Ponatinib binds the ATP pocket of BCR::ABL1, including T315I mutants","Constitutive kinase signalling (STAT5, RAS, PI3K) stops","Ph+ lymphoblasts undergo apoptosis","Dose is reduced once response is achieved to limit vascular events"],"dosing":{"route":"Oral","schedule":"Ph+ ALL: 30 mg daily with chemotherapy, reduced to 15 mg once MRD-negative CR is achieved; CML: 45 mg daily reducing to 15 mg on response (OPTIC)","monitoring":"Arterial occlusive events, venous thromboembolism, heart failure, hepatotoxicity (boxed warnings); blood pressure; lipase","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Iclusig"},"toxicity":[{"event":"Arterial occlusive events","anyGradePct":26,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Iclusig","note":"CML long-term data at 45 mg; lower with response-based dose reduction"},{"event":"Hypertension","anyGradePct":68},{"event":"Pancreatitis / lipase elevation","anyGradePct":40},{"event":"Hepatotoxicity","note":"Boxed warning"}],"access":[],"regulatoryEvents":[{"date":"2012-12-14","type":"accelerated-approval","region":"US","note":"Resistant CML/Ph+ ALL","indication":"Adults with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia resistant or intolerant to TKI therapy or or Philadelphia chromosome positive acute lymphoblastic leukemia resistant or intolerant to prior TKI therapy"},{"date":"2013-10-31","type":"withdrawal","region":"US","note":"Temporary marketing suspension for arterial occlusion; returned with narrowed label December 2013"},{"date":"2016-11-28","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2012 converted to traditional approval 4.0 years after it was granted.","indication":"Adults with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia resistant or intolerant to TKI therapy or or Philadelphia chromosome positive acute lymphoblastic leukemia resistant or intolerant to prior TKI therapy"},{"date":"2024-03-19","type":"accelerated-approval","region":"US","note":"Frontline Ph+ ALL (PhALLCON), accelerated The confirmatory requirement was still open 2.5 years later, when the FDA's table was read.","indication":"Adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy."}]},{"id":"ponsegromab","kind":"drug","name":"Ponsegromab","aka":[],"tldr":"Ponsegromab is a monoclonal antibody from Pfizer, in registered phase 3 trials for pancreatic ductal adenocarcinoma.","summary":"Ponsegromab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 3 trial sponsored by Pfizer, in pancreatic ductal adenocarcinoma. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Ponsegromab","url":"https://clinicaltrials.gov/search?intr=Ponsegromab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06989437"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"porfimer-sodium","kind":"drug","name":"Porfimer sodium","aka":[],"tldr":"Porfimer sodium is the drug half of photodynamic therapy: injected, it gathers in tumours, and a laser passed through an endoscope two days later activates it to burn away oesophageal and lung tumours that block the airway or gullet.","summary":"Porfimer sodium was the first photodynamic therapy agent approved in the United States, in December 1995, for palliation of obstructing oesophageal cancer, followed by early and obstructing endobronchial non-small cell lung cancer in 1998 and high-grade dysplasia in Barrett's oesophagus in 2003. Treatment needs an endoscopic laser session about 40 to 50 hours after injection, and patients must avoid bright light for weeks because the drug also sits in skin. It is approved in Canada, Japan and parts of Europe for similar uses. Stent placement and thermal ablation have taken much of its role, but it remains an option where they fail.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Porfimer_sodium","links":[{"label":"Drugs@FDA NDA020451","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=020451"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=porfimer"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Porfimer_sodium"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["esophageal","nsclc"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["pinnacle-biologics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00003788","nct00513539","nct02628665"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Photofrin","modality":"Photosensitiser for photodynamic therapy, intravenous then laser light","mechanism":"A haematoporphyrin derivative that concentrates in tumour tissue; red laser light at 630 nm two days later excites it to generate singlet oxygen that destroys the cells and their blood supply.","approvals":[{"region":"US","year":1995,"indication":"Palliation of completely or partially obstructing oesophageal cancer; later endobronchial NSCLC and Barrett's high-grade dysplasia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"poziotinib","kind":"drug","name":"Poziotinib","aka":["HM781-36B","NOV120101"],"tldr":"Poziotinib was a tablet designed to fit the awkward shape of HER2 and EGFR exon 20 mutations in lung cancer. It shrank tumours in some patients but caused severe rash and diarrhoea, and the FDA declined to approve it in 2022.","summary":"Poziotinib was discovered by Hanmi Pharmaceutical and developed in the West by Spectrum Pharmaceuticals. Its selling point was activity against exon 20 insertion mutations, which resist earlier EGFR and HER2 kinase inhibitors. In the ZENITH20 trials in HER2 exon 20-mutant non-small-cell lung cancer, response rates were around 28 to 35 percent, but grade 3 rash, diarrhoea and stomatitis were common and most patients needed dose reductions.\n\nAn FDA advisory committee voted against approval in September 2022, citing modest benefit, toxicity and the availability of trastuzumab deruxtecan, and the agency issued a complete response letter in November 2022; Spectrum then stopped development. The HER2-mutant lung cancer page lists it with pyrotinib among the HER2 kinase inhibitors limited by EGFR-driven side effects.","status":"negative","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Poziotinib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Poziotinib"}],"tags":["subtype-drugs-wave"],"related":["pyrotinib","trastuzumab-deruxtecan"],"cancers":["her2-mutant-nsclc"],"sections":[],"technologies":["her2-tyrosine-kinase-inhibitors"],"targets":["her2","egfr"],"drugs":[],"companies":["spectrum-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02659514","nct03744715","nct04172597"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"HM781-36B","modality":"Oral irreversible pan-HER tyrosine kinase inhibitor","mechanism":"An irreversible inhibitor of EGFR, HER2 and HER4 whose small, flexible structure was designed to fit the narrowed drug-binding pocket created by exon 20 insertion mutations in HER2 and EGFR.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pralatrexate","kind":"drug","name":"Pralatrexate","aka":[],"tldr":"Pralatrexate was the first drug approved specifically for relapsed peripheral T-cell lymphoma; about a third of patients respond.","summary":"Pralatrexate is a folate analogue with high affinity for the reduced folate carrier RFC-1 and efficient polyglutamylation, so it accumulates selectively in tumour cells and inhibits dihydrofolate reductase. It is given intravenously to patients with relapsed or refractory peripheral T-cell lymphoma, a group with few options. The single-arm PROPEL study (2009) showed a 29% response rate in heavily pretreated PTCL and supported accelerated US approval in 2009, the first drug licensed specifically for this indication; it is also approved in Japan and China. Mucositis is the dose-limiting toxicity and is mitigated by folate and vitamin B12 supplementation. It offers durable responses in a minority rather than a cure, without randomised survival data. For a newcomer, pralatrexate is a targeted antifolate that helps about a third of relapsed T-cell lymphoma patients.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Pralatrexate","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=pralatrexate"}],"tags":["gap-fill"],"related":[],"cancers":["peripheral-t-cell-lymphoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["acrotech-biopharma","spectrum-pharmaceuticals","mundipharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06072131"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Folotyn","modality":"Antifolate (DHFR inhibitor)","mechanism":"Folate analogue with high affinity for the reduced folate carrier (RFC-1) and efficient polyglutamylation, selectively accumulating in tumour cells and inhibiting DHFR.","approvals":[{"region":"US","year":2009,"indication":"Relapsed/refractory peripheral T-cell lymphoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2009-09-24","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 17.0 years later, when the FDA's table was read.","indication":"Treatment of relapsed or refractory peripheral T-Cell Lymphoma (PTCL)"}]},{"id":"pralsetinib","kind":"drug","name":"Pralsetinib","aka":[],"tldr":"Pralsetinib is the second selective RET pill for lung cancer, less used than selpercatinib after a change of owner and label.","summary":"Pralsetinib is a selective RET inhibitor that blocks the RET kinase in tumours driven by RET fusions or mutations while sparing most other kinases. It is used for RET-fusion metastatic non-small-cell lung cancer, where ARROW reported a response rate of about 72% in treatment-naive patients. It was approved in 2020, with full approval in 2023; the thyroid indications were withdrawn in the US in 2023 after Roche returned rights to Blueprint (now Rigel). It remains an option in RET-fusion NSCLC but is less used than selpercatinib, which has randomised phase 3 support and a broader label. For a newcomer, pralsetinib is the second of two RET-selective pills, effective but overshadowed by commercial and label changes rather than by weak efficacy.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Pralsetinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pralsetinib"},{"label":"NICE TA812: pralsetinib for treating RET fusion-positive advanced non-small-cell lung cancer (not recommended)","url":"https://www.nice.org.uk/guidance/ta812"}],"tags":[],"related":["ret-fusion"],"cancers":["nsclc","medullary-thyroid-cancer","ret-fusion-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["ret"],"drugs":["selpercatinib"],"companies":["blueprint-medicines","rigel-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04302025","arrow-thyroid"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Gavreto","modality":"Small-molecule kinase inhibitor (RET)","mechanism":"Selective RET inhibitor.","approvals":[{"region":"US","year":2020,"indication":"RET-fusion metastatic NSCLC (full approval 2023)"},{"region":"England (NICE)","year":2022,"indication":"RET fusion-positive advanced non-small-cell lung cancer in patients who have not had a RET inhibitor: not recommended","note":"TA812, published 3 August 2022, does not recommend pralsetinib within its marketing authorisation, so it is not routinely funded in England."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2020-09-04","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test"},{"date":"2020-12-01","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy"},{"date":"2020-12-01","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 5.8 years later, when the FDA's table was read.","indication":"Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)"},{"date":"2023-07-20","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 2.6 years after its accelerated approval.","indication":"Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy"},{"date":"2023-08-09","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 2.9 years after it was granted.","indication":"Treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test"}]},{"id":"precemtabart-tocentecan","kind":"drug","name":"Precemtabart tocentecan","aka":["Precem-TcT"],"tldr":"Precemtabart tocentecan is an experimental antibody-drug conjugate from EMD Serono Research & Development Institute in phase 3 trials for colorectal cancer, aimed at CEACAM5.","summary":"Precemtabart tocentecan is an antibody-drug conjugate developed by EMD Serono Research & Development Institute. Its target is CEACAM5 (the sponsor names CEACAM5). The sponsor states: An antibody-drug conjugate against CEACAM5 carrying an exatecan (topoisomerase inhibitor) payload, given intravenously once every three weeks. ClinicalTrials.gov describes the intervention as: Precem-TcT, administered, once every 3 weeks intravenously, on Day 1 of each 21-day cycle. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07549412 (A Study of Precemtabart Tocentecan With or Without Bevacizumab Compared to Trifluridine/Tipiracil Plus Bevacizumab in Participants With Previously Treated Metastatic Colorectal Cancer (PROCEADE-CRC-03)), in colorectal cancer. The largest, NCT07549412, plans to enrol 1020 participants with primary completion expected 2029-10-16. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Precemtabart tocentecan","url":"https://clinicaltrials.gov/search?intr=Precemtabart%20tocentecan"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["ceacam5"],"drugs":[],"companies":["merck-kgaa"],"institutions":[],"pathways":[],"terms":[],"trials":["proceade-crc-03","nct06710132"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"An antibody-drug conjugate against CEACAM5 carrying an exatecan (topoisomerase inhibitor) payload, given intravenously once every three weeks.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"prednisone","kind":"drug","name":"Prednisone","aka":["Prednisolone (active metabolite, used in the UK and Europe)"],"tldr":"Prednisone is the everyday steroid tablet in cancer care. It is the P in CHOP and MOPP for lymphoma, part of childhood leukaemia treatment, and taken with abiraterone in prostate cancer.","summary":"Prednisone was approved in 1955 and its label lists leukaemias and lymphomas for palliative management and hypercalcaemia of malignancy alongside a long list of inflammatory conditions. In oncology it is a component of R-CHOP (diffuse large B-cell lymphoma), MOPP and BEACOPP (Hodgkin lymphoma), childhood and adult ALL induction and CLL regimens, and it is co-prescribed with abiraterone to prevent mineralocorticoid excess and with docetaxel in metastatic castration-resistant prostate cancer. It also treats immune-related adverse events of checkpoint inhibitors and is used in palliative care for appetite and fatigue. Prednisolone is the equivalent used in the UK and much of Europe. Long-term use brings hyperglycaemia, osteoporosis, infection risk, mood change and adrenal suppression.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Prednisone","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=prednisone"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/prednisone"}],"tags":["nci-list","generic","supportive"],"related":["abiraterone","docetaxel"],"cancers":["dlbcl","hodgkin-lymphoma","all-leukemia","cll","prostate"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["prodrug"],"trials":["nct06097364","nct04529772","nct06717347","nct06191744","nct03706365","nct04446117","nct06091865","nct06091254","nct06925737","nct04691804","nct04663347","nct06689163","nct04542824","nct02960022","nct07124936","nct05201248","nct06568094","nct07730515","nct07005154","nct05067140","nct05283720","nct06564038","nct07553988","nct02257736","nct05288166","nct03431350","nct05406401","nct07225946","nct06353386","nct04623541","nct06890884","nct04497844","nct05673785","nct04980222","nct06947967","nct06425302"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Deltasone (historic) / Rayos","modality":"Glucocorticoid","mechanism":"Prodrug converted in the liver to prednisolone, a glucocorticoid receptor agonist that is lympholytic and anti-inflammatory.","approvals":[{"region":"US","year":1955,"indication":"Palliative management of leukaemias and lymphomas; hypercalcaemia of malignancy; inflammatory conditions"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-06-12","type":"approval","region":"US","note":"FDA approves capivasertib with abiraterone and prednisone for PTEN-deficient androgen pathway modulation-naïve or -sensitive prostate cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-capivasertib-abiraterone-and-prednisone-pten-deficient-androgen-pathway-modulation"}]},{"id":"procarbazine","kind":"drug","name":"Procarbazine","aka":["Natulan"],"tldr":"Procarbazine (Matulane) is an old chemotherapy capsule from the MOPP regimen for Hodgkin lymphoma; it is now used mainly in the PCV combination for certain brain tumours.","summary":"Procarbazine was approved in 1969 for stage III and IV Hodgkin disease as part of MOPP (nitrogen mustard, vincristine, procarbazine, prednisone) and later BEACOPP; ABVD replaced MOPP because procarbazine causes permanent infertility in men and secondary leukaemia. Its major contemporary role is off label in PCV (procarbazine, lomustine, vincristine), which with radiotherapy roughly doubled survival in 1p/19q-codeleted oligodendroglioma (RTOG 9402, EORTC 26951). Patients must avoid tyramine-rich foods, alcohol and sympathomimetics because of monoamine oxidase inhibition; nausea and myelosuppression are common.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Procarbazine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=procarbazine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/procarbazinehydrochloride"}],"tags":["nci-list"],"related":["lomustine"],"cancers":["hodgkin-lymphoma","glioblastoma","oligodendroglioma","idh-mutant-astrocytoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["leadiant"],"institutions":[],"pathways":[],"terms":[],"trials":["rtog-9402","eortc-26951","nct07015242"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Matulane","modality":"Alkylating-like methylhydrazine derivative","mechanism":"Metabolically activated to methylating and free-radical species that damage DNA and inhibit DNA, RNA and protein synthesis; a weak monoamine oxidase inhibitor.","approvals":[{"region":"US","year":1969,"indication":"Stage III and IV Hodgkin disease in combination (MOPP)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"icad-profound-ai","kind":"drug","name":"ProFound AI (iCAD)","aka":[],"tldr":"The first AI cleared for 3D mammography in the US, now part of RadNet's imaging network.","summary":"iCAD's ProFound AI was cleared by the FDA in December 2018 as the first artificial-intelligence software for digital breast tomosynthesis, and later versions cover 2D mammography, breast density and risk. The company's reader study reported faster reading and improved sensitivity for radiologists using the software. iCAD, based in Nashua, New Hampshire, agreed in 2025 to be acquired by RadNet, which also owns DeepHealth (and, through it, Kheiron), consolidating three of the leading mammography AI products under one imaging chain. ProFound is installed at thousands of sites in the US and Europe.","status":"approved","asOf":"2026-09-10","links":[{"label":"iCAD: ProFound","url":"https://www.icadmed.com"}],"tags":["test"],"related":[],"cancers":["breast-hr-positive","tnbc"],"sections":[],"technologies":["radiology-ai-screening","mammography"],"targets":[],"drugs":[],"companies":["icad"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: the software supports the radiologist reading your 3D mammogram; you are unlikely to be told it was used."],"brand":"ProFound Detection","modality":"AI mammography and tomosynthesis detection software","mechanism":"Deep-learning detection across the slices of a tomosynthesis exam or a 2D mammogram, with case and lesion scores for radiologists.","approvals":[{"region":"US","year":2018,"indication":"510(k) clearance: AI detection for digital breast tomosynthesis (first for 3D mammography)"},{"region":"EU","year":2019,"indication":"CE mark"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"megestrol-progestins","kind":"drug","name":"Progestins (megestrol acetate, medroxyprogesterone, levonorgestrel IUD)","aka":["Levonorgestrel","Levonorgestrel intrauterine device","Megestrol acetate","Medroxyprogesterone","Oral progestins"],"tldr":"Progesterone-like hormones that can reverse early endometrial cancer in women who want to keep their uterus, and control advanced hormone-sensitive disease.","summary":"Fertility-sparing therapy for grade 1, stage IA endometrioid cancer or atypical hyperplasia (oral progestin or levonorgestrel IUD; complete response ~70-80% within 12 months, relapse ~30%). In advanced ER/PR-positive low-grade disease, progestins, alternating with tamoxifen (GOG-119), or aromatase inhibitors with CDK4/6 inhibitors (PALEO, NRG-GY019) give durable control with minimal toxicity.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Megestrol_acetate","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Progestins"}],"tags":[],"related":[],"cancers":["endometrial"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":[],"institutions":[],"pathways":["er-signaling"],"terms":[],"trials":["nct03463252","nct02990728","nct01594879"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Megace; Mirena (IUD)","modality":"Hormonal therapy (progestin)","mechanism":"Progesterone receptor agonism suppresses ER-driven proliferation and induces glandular differentiation of endometrial epithelium.","approvals":[{"region":"US","year":1971,"indication":"Palliative treatment of advanced endometrial and breast cancer (megestrol)"}],"mechanismSteps":[],"dosing":{"route":"Oral or intrauterine","schedule":"Megestrol 160 mg daily; levonorgestrel 52 mg IUD; re-biopsy every 3-6 months in fertility-sparing use","monitoring":"Endometrial sampling, weight, thrombosis risk"},"toxicity":[{"event":"Weight gain","anyGradePct":30},{"event":"Venous thromboembolism","note":"Increased risk with high-dose oral progestins"}],"access":[],"regulatoryEvents":[]},{"id":"prolaris","kind":"drug","name":"Prolaris","aka":[],"tldr":"A gene test on a prostate biopsy that estimates how aggressive the cancer is, to help decide between active surveillance and treatment.","summary":"Prolaris (Myriad Genetics) measures how fast tumour cells are dividing and reports a 10-year risk of dying from prostate cancer under conservative management and of metastasis after treatment. It is listed in NCCN guidelines as an option for men with low or favourable-intermediate risk disease considering active surveillance, is covered by Medicare, and validated in conservatively managed UK cohorts and US surgical series. Like Decipher and Oncotype DX GPS it refines, rather than replaces, PSA, Gleason grade and MRI; head-to-head comparisons are few and none of the three has randomised evidence that its use improves outcomes.","status":"established","asOf":"2026-09-10","links":[{"label":"Myriad: Prolaris (page moved; nearest live section)","url":"https://myriad.com/oncology/"}],"tags":["test"],"related":["decipher-prostate","oncotype-dx-gps"],"cancers":["prostate"],"sections":[],"technologies":["rna-seq","active-surveillance","gene-expression-prognostic-assays"],"targets":[],"drugs":[],"companies":["myriad-genetics"],"institutions":[],"pathways":[],"terms":["gleason-grade-group","psa","active-surveillance-term"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: a low score supports active surveillance; a high score in an otherwise low-risk cancer argues for treatment."],"brand":"Prolaris","modality":"Gene-expression prognostic assay (cell-cycle progression score, prostate)","mechanism":"RT-PCR of 31 cell-cycle progression genes and 15 housekeeping genes on biopsy tissue, combined with clinical risk (CAPRA) to estimate 10-year prostate cancer mortality and metastasis risk.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"propranolol-hemangeol","kind":"drug","name":"Propranolol (Hemangeol)","aka":["Propranolol Hydrochloride","Propranolol oral solution"],"tldr":"Hemangeol is a liquid form of the blood-pressure drug propranolol approved to shrink infantile haemangiomas, the common benign blood-vessel tumours of babies, replacing steroids and surgery for most children who need treatment.","summary":"Propranolol oral solution (Hemangeol) was approved in the United States in 2014 for proliferating infantile haemangioma requiring systemic therapy, after a chance observation in 2008 that the beta-blocker shrank these benign vascular tumours. Infantile haemangiomas are the commonest tumours of infancy and most need no treatment, but those threatening vision, breathing or feeding, or ulcerating, respond within weeks. Propranolol has since been studied in angiosarcoma and other vascular cancers on the same rationale, without approval. Low blood sugar, slow heart rate and low blood pressure are the main risks in infants.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Propranolol","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=propranolol%20hydrochloride%20oral%20solution"},{"label":"Drugs@FDA NDA 205410","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=205410"}],"tags":["gap-fill","supportive-care"],"related":[],"cancers":["sarcoma"],"sections":[],"technologies":["supportive-care"],"targets":["vegf"],"drugs":[],"companies":["pierre-fabre"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07125391","nct05741164"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Hemangeol","modality":"Small-molecule non-selective beta-adrenergic blocker, oral solution","mechanism":"Blocks beta-adrenergic receptors on the endothelial cells of a proliferating haemangioma, causing vasoconstriction, lower VEGF signalling and apoptosis of the abnormal vessels.","approvals":[{"region":"US","year":2014,"indication":"Proliferating infantile haemangioma requiring systemic therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"prosigna","kind":"drug","name":"Prosigna (PAM50)","aka":[],"tldr":"A 50-gene test run in local hospital laboratories that estimates the ten-year risk of a hormone-positive breast cancer coming back.","summary":"Prosigna was cleared by the FDA on 6 September 2013 (510(k) K130010, NanoString; now marketed by Veracyte) for postmenopausal women with hormone-receptor-positive, node-negative or one-to-three-node-positive early breast cancer treated with endocrine therapy. Validation came from the ABCSG-8 and TransATAC cohorts, where the ROR score predicted distant recurrence at 10 years, including late recurrence after five years. NICE diagnostics guidance DG34 (2018) recommends Prosigna, Oncotype DX and EndoPredict for guiding adjuvant chemotherapy decisions in the NHS. Because it runs on a decentralised instrument, it is common in Europe where send-out testing is less used.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA 510(k) K130010","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpmn/pmn.cfm?ID=K130010"},{"label":"NICE DG34: tumour profiling tests for breast cancer","url":"https://www.nice.org.uk/guidance/dg34"}],"tags":["test"],"related":["oncotype-dx","mammaprint","endopredict"],"cancers":["breast-hr-positive"],"sections":[],"technologies":["rna-seq"],"targets":[],"drugs":[],"companies":["veracyte"],"institutions":[],"pathways":[],"terms":["biomarker"],"trials":["nct02653755"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: a low ROR score means chemotherapy is unlikely to help on top of hormone therapy; a high score means it is likely to be recommended."],"brand":"Prosigna","modality":"Gene-expression prognostic assay (50-gene PAM50 risk of recurrence)","mechanism":"nCounter digital expression of 50 classifier genes assigns an intrinsic subtype (luminal A, luminal B, HER2-enriched, basal-like) and a risk-of-recurrence (ROR) score combined with tumour size and nodal status.","approvals":[{"region":"US","year":2013,"indication":"Prognosis of distant recurrence in postmenopausal HR+ early breast cancer (510(k))"},{"region":"EU","year":2012,"indication":"CE-IVD"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"prostvac","kind":"drug","name":"PROSTVAC","aka":["PROSTVAC-VF","PROSTVAC-V/F","rilimogene galvacirepvec"],"tldr":"A vaccine made from two viruses carrying the PSA gene, meant to teach the immune system to attack prostate cancer. The big trial found it did nothing at all.","summary":"PROSTVAC is a heterologous prime-boost vaccine: a vaccinia vector primes and a fowlpox vector boosts, both carrying the gene for prostate-specific antigen together with three co-stimulatory molecules (B7.1, ICAM-1 and LFA-3, known as TRICOM). It came from the National Cancer Institute and was licensed to Bavarian Nordic.\n\nA randomised phase 2 trial reported a median overall survival 8.5 months longer than placebo, which is the kind of result that makes a phase 3 inevitable. PROSPECT randomised 1,297 men with asymptomatic or minimally symptomatic metastatic castration-resistant prostate cancer to PROSTVAC alone (432), PROSTVAC with granulocyte-macrophage colony-stimulating factor (432) or placebo (433).\n\nAt the third interim analysis the trial met its futility criteria and stopped. Median overall survival was 34.4 months with PROSTVAC alone (hazard ratio 1.01, 95 percent confidence interval 0.84 to 1.20, p=0.47), 33.2 months with the granulocyte-macrophage colony-stimulating factor combination (1.02, 0.86 to 1.22, p=0.59) and 34.3 months with placebo. Patients alive without radiographic progression, pain progression, chemotherapy or death at 6 months were 29.4, 28.0 and 30.3 percent respectively.\n\nThe result is important beyond the drug. The phase 2 signal was an artefact of a placebo arm that did worse than the phase 3 placebo arm, which lived 34.3 months. Comparing a single-arm or small randomised result against a historical control in a disease where survival is long and improving is how this class of error happens.","status":"withdrawn","asOf":"2026-09-25","links":[{"label":"ClinicalTrials.gov NCT01322490","url":"https://clinicaltrials.gov/study/NCT01322490"},{"label":"PROSPECT (Journal of Clinical Oncology 2019)","url":"https://doi.org/10.1200/JCO.18.02031"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["shared-antigen-vaccine"],"targets":[],"drugs":[],"companies":["bavarian-nordic"],"institutions":[],"pathways":[],"terms":["psa","castration-resistance"],"trials":["prospect-prostvac","impact-sipuleucel-t"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"vaccine","mechanism":"Poxviral prime-boost vaccine (vaccinia then fowlpox) encoding prostate-specific antigen with the TRICOM co-stimulatory triad B7.1, ICAM-1 and LFA-3.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"prtx007","kind":"drug","name":"PRTX007","aka":[],"tldr":"PRTX007 is a small-molecule inhibitor from Primmune Therapeutics, Inc., in registered phase 2 trials for melanoma.","summary":"PRTX007 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Primmune Therapeutics, Inc., in melanoma. Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of PRTX007","url":"https://clinicaltrials.gov/search?intr=PRTX007"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["melanoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["primmune-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07565285"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"PRTX007","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"psma-1007-f18","kind":"drug","name":"PSMA-1007 F-18","aka":[],"tldr":"PSMA-1007 is a fluorine-18 PSMA PET tracer from ABX in Germany, widely used in Europe for prostate cancer staging because little of it reaches the bladder, and in a phase 3 trial registered by its maker.","summary":"PSMA-1007, developed at Heidelberg and manufactured by ABX advanced biochemical compounds in Radeberg, is a fluorine-18 PSMA ligand whose low urinary excretion improves views of the prostate bed and pelvic nodes. It is authorised in several European countries and Canada and used in many centres alongside gallium-68 PSMA-11 and piflufolastat; a phase 3 trial by ABX is registered on ClinicalTrials.gov.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of PSMA-1007 F-18","url":"https://clinicaltrials.gov/search?intr=PSMA-1007%20F-18"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["psma-pet","pet"],"targets":["psma"],"drugs":[],"companies":["abx-advanced-biochemical-compounds"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06122584"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"modality":"PSMA-targeted PET radiotracer, fluorine-18","mechanism":"A fluorine-18 PSMA ligand excreted mainly through the liver rather than the kidneys, so the bladder does not obscure the prostate bed on PET.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"pucotenlimab","kind":"drug","name":"Pucotenlimab","aka":[],"tldr":"Pucotenlimab is Lepu Biopharma's PD-1 antibody, approved in China in 2022 for mismatch-repair-deficient solid tumours and for melanoma, and in a phase 3 trial in colorectal cancer.","summary":"Taizhou Hanzhong (Lepu Biopharma) developed pucotenlimab (HX008), approved by the NMPA in July 2022 for previously treated MSI-high or mismatch-repair-deficient advanced solid tumours, China's first tissue-agnostic PD-1 approval, and for unresectable or metastatic melanoma after prior therapy. A phase 3 trial in metastatic colorectal cancer is registered.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Pucotenlimab","url":"https://clinicaltrials.gov/search?intr=HX008"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma","colorectal"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["lepu-biopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05652894","nct06976190","nct07479485","nct07586124"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"HX008","modality":"Anti-PD-1 monoclonal antibody, intravenous","mechanism":"Blocks PD-1 on T cells so tumour cells cannot switch them off through PD-L1.","approvals":[{"region":"CN","year":2022,"indication":"Previously treated MSI-high or dMMR advanced solid tumours; unresectable or metastatic melanoma after prior therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"purinostat","kind":"drug","name":"Purinostat","aka":["Purinostat Mesylate"],"tldr":"Purinostat is an experimental investigational agent whose form is not stated in the registry from Chengdu Zenitar Biomedical Technology in phase 3 trials for diffuse large B-cell lymphoma and multiple myeloma, with its target not yet stated publicly.","summary":"Purinostat is an investigational agent whose form is not stated in the registry developed by Chengdu Zenitar Biomedical Technology. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: The subjects in the experimental group will receive treatment with Purinostat Mesylate for injection. The dosage is 11.2 mg/m2. Each administration cycle consists of intravenous infusion on days 1, 4, 8, and 11. The administration cycle las. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07011056 (A Phase III Clinical Study of Purinostat Mesylate for Injection in Patients With Diffuse Large B-cell Lymphoma), in diffuse large B-cell lymphoma and multiple myeloma. The largest, NCT07011056, plans to enrol 390 participants with primary completion expected 2029-06-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Purinostat","url":"https://clinicaltrials.gov/search?intr=Purinostat"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl","multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07011056"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"puxitatug-samrotecan","kind":"drug","name":"Puxitatug samrotecan","aka":[],"tldr":"Puxitatug samrotecan is a B7-H4 ADC targeting a checkpoint-like protein enriched in breast, ovarian, and endometrial cancers.","summary":"Puxitatug samrotecan is AstraZeneca's anti-B7-H4 IgG1 antibody carrying the topoisomerase I inhibitor AZ14170133 at a drug-to-antibody ratio of about 8 through a cleavable linker; the released payload traps topoisomerase I on DNA and crosses membranes to kill neighbouring cells. B7-H4 is a checkpoint-like protein enriched in breast, ovarian and endometrial cancers with minimal normal expression, and it is inversely correlated with PD-L1, offering a route into 'cold' tumours that immunotherapy fails to reach. The drug is in phase 2 dose optimisation on an every-3-weeks schedule in endometrial, ovarian and triple-negative breast cancer. Nausea, neutropenia and fatigue are the reported toxicities. Whether B7-H4 expression predicts response remains open. It targets a protein common in gynaecological and breast cancers that immunotherapy tends to miss.","status":"phase-2","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Puxitatug samrotecan","url":"https://clinicaltrials.gov/search?intr=AZD8205"}],"tags":[],"related":[],"cancers":["tnbc","ovarian","endometrial"],"sections":[],"technologies":["adc"],"targets":["b7h4"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07044336","nct05123482"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"AZD8205","modality":"ADC","payload":"TOP1 inhibitor (AZ14170133), DAR ~8","linker":"Cleavable","mechanism":"Anti-B7-H4 IgG1 with TOP1 inhibitor.","approvals":[],"mechanismSteps":["Antibody binds B7-H4 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","TOP1 inhibitor is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"Phase 2 dose-optimisation; every 3 weeks","source":"https://clinicaltrials.gov/study/NCT05123482"},"toxicity":[{"event":"Nausea"},{"event":"Neutropenia"},{"event":"Fatigue"}],"access":[{"country":"US","reimbursement":"Investigational","asOf":"2026-09-06"}],"regulatoryEvents":[]},{"id":"pyrotinib","kind":"drug","name":"Pyrotinib","aka":[],"tldr":"Pyrotinib is an irreversible pan-ErbB kinase inhibitor pill (EGFR, HER2, HER4) from Jiangsu Hengrui, approved in China since 2018 but not in the US or EU. It is the standard HER2 pill there, given with capecitabine after trastuzumab, and the comparator that new Chinese HER2 ADCs are beating; diarrhoea affects nearly every patient.","summary":"Pyrotinib is an irreversible pan-ErbB tyrosine kinase inhibitor covering EGFR, HER2 and HER4, developed by Jiangsu Hengrui. It is China's HER2 pill, widely used there with capecitabine for HER2-positive advanced breast cancer and in the first line with trastuzumab and chemotherapy. PHOEBE (versus lapatinib plus capecitabine) showed PFS of 12.5 versus 6.8 months, and PHILA (first line with trastuzumab plus docetaxel) showed PFS of 24.3 versus 10.4 months. It is approved in China (2018, with 2020s expansions) but not in the US or EU, and diarrhoea is near universal (95% any grade, 31% grade 3 or higher) and drives dose management. It is the control arm of HORIZON-Breast01, where the ADC trastuzumab rezetecan beat pyrotinib plus capecitabine (PFS 30.6 versus 8.3 months). For a newcomer, pyrotinib is the standard HER2 pill in China and the benchmark for the new Chinese HER2 ADCs.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Pyrotinib"}],"tags":[],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["her2","egfr"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["horizon-breast01","nct06015048","nct05482568","nct03980054"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Airuini","modality":"Small-molecule irreversible pan-HER kinase inhibitor","mechanism":"Irreversible pan-ErbB TKI (EGFR, HER2, HER4).","approvals":[{"region":"China","year":2018,"indication":"HER2+ advanced breast cancer with capecitabine"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"400 mg once daily with capecitabine","monitoring":"Diarrhoea"},"toxicity":[{"event":"Diarrhoea","anyGradePct":95,"grade3PlusPct":31},{"event":"Hand-foot syndrome","anyGradePct":60}],"access":[],"regulatoryEvents":[]},{"id":"ql1706","kind":"drug","name":"QL1706","aka":["Iparomlimab and Tuvonralimab，PSB205"],"tldr":"QL1706 is an experimental small-molecule drug from Qilu Pharmaceutical in phase 3 trials for non-small-cell lung cancer and colorectal cancer, with its target not yet stated publicly.","summary":"QL1706 (PSB205) is a small-molecule drug developed by Qilu Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: QL1706(5mg/kg Q3W IV) concomitantly with Platinum-based chemotherapy. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05487391 (A Study of QL1706 Combined With Platinum-containing Chemotherapy in Adjuvant Treatment of Stage II-IIIB Non-small Cell Lung Cancer After Complete Surgical Resection), NCT06686576 (A Study of Neoadjuvant QL1706 in Participants With Untreated dMMR/MSI-H Resectable Colon Cancer) and NCT05690945 (A Study of QL1706 in Combination With Chemotherapy in PD-L1-Negative Non-small Cell Lung Cancer), in non-small-cell lung cancer and colorectal cancer. The largest, NCT05487391, plans to enrol 632 participants with primary completion expected 2027-08-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of QL1706","url":"https://clinicaltrials.gov/search?intr=PSB205"},{"label":"Sponsor page","url":"https://www.qilu-pharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05487391","nct06686576","nct05690945","nct07256782"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PSB205","modality":"bifunctional antibody mixture (iparomlimab and tuvonralimab; INN stem -mab)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ql2107","kind":"drug","name":"QL2107","aka":[],"tldr":"QL2107 is an experimental investigational agent whose form is not stated in the registry from Qilu Pharmaceutical in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"QL2107 is an investigational agent whose form is not stated in the registry developed by Qilu Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: 200mg on day 1 of each 21-day cycle of the study. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06754644 (A Study to Evaluate Efficacy, and Safety of QL2107 Plus Chemo and Compare With Keytruda in Participants With IV nqNSCLC), in non-small-cell lung cancer. The largest, NCT06754644, plans to enrol 808 participants with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of QL2107","url":"https://clinicaltrials.gov/search?intr=QL2107"},{"label":"Sponsor page","url":"https://www.qilu-pharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06754644","nct06911827","nct07256782"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"qlc5508","kind":"drug","name":"QLC5508","aka":[],"tldr":"QLC5508 is an experimental antibody-drug conjugate from Qilu Pharmaceutical in phase 3 trials for oesophageal cancer, prostate cancer and non-small-cell lung cancer, aimed at B7-H3.","summary":"QLC5508 (MHB088C) is an antibody-drug conjugate developed by Qilu Pharmaceutical and Minghui Pharmaceutical (Hangzhou). Its target is B7-H3. ClinicalTrials.gov describes the intervention as: B7H3 ADC; QLC5508 will be administered by injection at the dose and dosing frequency specified in the protocol. It is the investigational product in 6 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07463573 (QLC5508 vs. Chemotherapy in Pretreated Advanced or Metastatic Oesophageal Squamous Cell Carcinoma) and NCT06954246 (A Study of MHB088C Injection Versus Treatment of Physician's Choice in Subjects With Relapsed Small Cell Lung Cancer), in oesophageal cancer, prostate cancer and non-small-cell lung cancer. The largest, NCT07102004, plans to enrol 515 participants with primary completion was scheduled for 2026-07 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of QLC5508","url":"https://clinicaltrials.gov/search?intr=MHB088C"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["esophageal","prostate","nsclc"],"sections":[],"technologies":[],"targets":["b7h3"],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07463573","nct06954246","nct07198633","nct07102004","nct07256782"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"MHB088C","modality":"ADC","mechanism":"Antibody-drug conjugate directed at B7-H3, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"qlh12016","kind":"drug","name":"QLH12016","aka":[],"tldr":"QLH12016 is an experimental protein degrader from Qilu Pharmaceutical in phase 2 trials for prostate cancer, aimed at Androgen receptor.","summary":"QLH12016 is a protein degrader developed by Qilu Pharmaceutical. Its target is Androgen receptor. ClinicalTrials.gov describes the intervention as: An oral androgen receptor PROTAC; QLH12016 will be administered orally at the dose and dosing frequency specified in the protocol. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in prostate cancer. The largest, NCT07198633, plans to enrol 212 participants with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of QLH12016","url":"https://clinicaltrials.gov/search?intr=QLH12016"},{"label":"Sponsor page","url":"https://www.qilu-pharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":["androgen-receptor"],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07198633"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"protein degrader","mechanism":"Protein degrader directed at Androgen receptor, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"qls31905","kind":"drug","name":"QLS31905","aka":[],"tldr":"QLS31905 is an experimental investigational agent whose form is not stated in the registry from Qilu Pharmaceutical in phase 3 trials for pancreatic ductal adenocarcinoma, with its target not yet stated publicly.","summary":"QLS31905 is an investigational agent whose form is not stated in the registry developed by Qilu Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: QLS31905 will be administered as an IV infusion. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07079228 (A Study to Compare QLS31905 and Chemotherapy With Placebo and Chemotherapy in Participants With Pancreatic Cancer), in pancreatic ductal adenocarcinoma. The largest, NCT07079228, plans to enrol 602 participants with primary completion expected 2028-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of QLS31905","url":"https://clinicaltrials.gov/search?intr=QLS31905"},{"label":"Sponsor page","url":"https://www.qilu-pharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07079228"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"qls32015","kind":"drug","name":"QLS32015","aka":[],"tldr":"QLS32015 is an experimental investigational agent whose form is not stated in the registry from Qilu Pharmaceutical in phase 3 trials for multiple myeloma, with its target not yet stated publicly.","summary":"QLS32015 is an investigational agent whose form is not stated in the registry developed by Qilu Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: QLS32015 will be administered subcutaneously. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07138209 (A Study Comparing QLS32015 Monotherapy Versus Pomalidomide, Dexamethasone (Pd) or Selinexor, Dexamethasone (Sd) in Participants With Relapsed or Refractory Multiple Myeloma), in multiple myeloma. The largest, NCT07138209, plans to enrol 228 participants with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of QLS32015","url":"https://clinicaltrials.gov/search?intr=QLS32015"},{"label":"Sponsor page","url":"https://www.qilu-pharma.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["qilu-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07138209","nct07018050"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"quemliclustat","kind":"drug","name":"Quemliclustat","aka":[],"tldr":"Quemliclustat is an experimental investigational agent whose form is not stated in the registry from Arcus Biosciences in phase 3 trials for pancreatic ductal adenocarcinoma and non-small-cell lung cancer, with its target not yet stated publicly.","summary":"Quemliclustat is an investigational agent whose form is not stated in the registry developed by Arcus Biosciences and Gilead Sciences. The sponsor describes its target as adenosine axis, which OnCo does not yet have a target page for. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06608927 (Study of Quemliclustat and Chemotherapy Versus Placebo and Chemotherapy in Patients With Metastatic Pancreatic Ductal Adenocarcinoma), in pancreatic ductal adenocarcinoma and non-small-cell lung cancer. The largest, NCT06608927, plans to enrol 610 participants with primary completion expected 2030-11-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Quemliclustat","url":"https://clinicaltrials.gov/search?intr=Quemliclustat"},{"label":"Sponsor page","url":"https://www.arcusbio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic","nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["arcus-biosciences","gilead"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06608927","nct05676931","nct05329766"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"quizartinib","kind":"drug","name":"Quizartinib","aka":[],"tldr":"Quizartinib is a more selective FLT3 blocker that, added to chemotherapy, roughly doubled median survival in the highest-risk FLT3 subtype.","summary":"QuANTUM-First (n=539, FLT3-ITD, age 18-75): median OS 31.9 vs 15.1 months (HR 0.776), including patients not transplanted; benefit sustained in the maintenance phase analysis. Approved July 2023 with a boxed warning for QT prolongation and a REMS. Earlier rejected (2019) in the relapsed setting after QuANTUM-R despite a positive OS result. Approved in Japan 2019.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Quizartinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Quizartinib"}],"tags":[],"related":["flt3-itd"],"cancers":["aml","aml-flt3"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["flt3"],"drugs":[],"companies":["daiichi-sankyo"],"institutions":[],"pathways":[],"terms":[],"trials":["quantum-first","nct06824168","nct03793478"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vanflyta","modality":"Small-molecule kinase inhibitor (FLT3-ITD, type II)","mechanism":"Type II FLT3 inhibitor binding the inactive DFG-out conformation; potent against ITD, inactive against TKD resistance mutations (D835).","approvals":[{"region":"US","year":2023,"indication":"Newly diagnosed FLT3-ITD AML with 7+3, consolidation, and maintenance"},{"region":"Japan","year":2019,"indication":"Relapsed/refractory FLT3-ITD AML"}],"mechanismSteps":["Quizartinib locks FLT3-ITD in its inactive conformation","Downstream STAT5 and MAPK signalling is silenced","FLT3-ITD blasts undergo apoptosis while normal progenitors are spared","Continued for up to 36 cycles of maintenance after consolidation or transplant"],"dosing":{"route":"Oral","schedule":"35.4 mg once daily on days 8-21 of induction and consolidation; 26.5 mg daily for 2 weeks then 53 mg daily as maintenance for up to 36 cycles","monitoring":"ECG at baseline and regularly (boxed warning: QT prolongation, torsades); electrolytes; REMS","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Vanflyta"},"toxicity":[{"event":"QT prolongation","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Vanflyta","note":"Boxed warning; grade 3 QTc >500 ms in ~3%"},{"event":"Febrile neutropenia","grade3PlusPct":43},{"event":"Neutropenia","grade3PlusPct":20,"note":"Prolonged count recovery during consolidation"},{"event":"Hypokalaemia","anyGradePct":32}],"access":[],"regulatoryEvents":[{"date":"2019-06-14","type":"crl","region":"US","note":"Complete response letter for relapsed/refractory indication (QuANTUM-R)"},{"date":"2023-07-20","type":"approval","region":"US","note":"Frontline FLT3-ITD AML (QuANTUM-First)"}]},{"id":"radar-mrd","kind":"drug","name":"RaDaR","aka":[],"tldr":"NeoGenomics' personalised blood test for tiny amounts of leftover cancer, tracking up to 48 mutations from the patient's own tumour.","summary":"RaDaR was developed by Inivata (Cambridge, UK), acquired by NeoGenomics in 2021, and received FDA Breakthrough Device designation in 2021 for detecting residual disease and recurrence. Tracking dozens of variants at once lets it reach very low tumour fractions (parts per hundred thousand in validation studies) and it has been used in head and neck, breast and lung cancer cohorts, including the TRACERx-associated work in lung cancer. It is offered as a laboratory-developed test and competes with Signatera, Guardant Reveal and Oncodetect in a market that Medicare coverage decisions largely shape.","status":"established","asOf":"2026-09-10","links":[{"label":"NeoGenomics: RaDaR (page moved; nearest live section)","url":"https://neogenomics.com/test-menu/"}],"tags":["test"],"related":["signatera","guardant-reveal"],"cancers":["head-and-neck","nsclc","breast-hr-positive","tnbc"],"sections":[],"technologies":["mrd-testing","liquid-biopsy"],"targets":[],"drugs":[],"companies":["neogenomics"],"institutions":[],"pathways":[],"terms":["mrd","ctdna"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: as with other MRD tests, detection of tumour DNA in blood predicts relapse months before scans; how to act on it is still being defined in trials."],"brand":"RaDaR","modality":"Tumour-informed ctDNA minimal residual disease test (up to 48 variants)","mechanism":"Whole-exome sequencing of the tumour selects up to 48 patient-specific variants, which are tracked in plasma by deep amplicon sequencing with error suppression.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"radioactive-iodine","kind":"drug","name":"Radioactive iodine (I-131)","aka":[],"tldr":"The original targeted radiotherapy: thyroid cells soak up iodine, so radioactive iodine destroys leftover thyroid tissue and metastases while sparing everything else.","summary":"In use since 1946 for differentiated thyroid cancer; the archetype of theranostics (I-123 or I-131 scans image the same uptake). Roles: remnant ablation after thyroidectomy (now omitted in low-risk disease after ESTIMABL2, IoN, HiLo), adjuvant treatment of intermediate/high-risk disease, and treatment of iodine-avid metastases. Given after TSH stimulation (withdrawal or recombinant TSH). Salivary damage, secondary malignancy at high cumulative doses, and refractoriness in dedifferentiated tumours are the limits.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Iodine-131","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Radioactive%20iodine"},{"label":"UniProt Q92911: sodium/iodide cotransporter (SLC5A5, NIS), the uptake route of iodide into thyroid cells","url":"https://www.uniprot.org/uniprotkb/Q92911/entry"}],"tags":[],"related":[],"cancers":["thyroid"],"sections":[],"technologies":["radioiodine-therapy","radioligand-therapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["theranostics","rai-refractory","low-risk-dtc","radioiodine-term"],"trials":["hilo","estimabl2","ion-trial"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Correction 23 Sept 2026: the record linked the somatostatin receptor 2 target, which belongs to the SSTR2 radioligands (Lutathera, RYZ101) and not to radioiodine. I-131 enters thyroid cells through the sodium-iodide symporter (SLC5A5, NIS), which has no target page in OnCo yet, so the record now names no target; the mechanism text says which transporter it uses. Found by the open drug engine, where the wrong link showed I-131 as an approved SSTR2 radioligand."],"brand":"Sodium iodide I-131","modality":"Radiopharmaceutical (beta/gamma emitter, natural uptake)","mechanism":"Sodium-iodide symporter (NIS) concentrates iodide in thyroid follicular cells; I-131 beta particles (mean path ~0.8 mm) irradiate the cell and neighbours; gamma emission enables imaging.","approvals":[{"region":"US","year":1951,"indication":"Hyperthyroidism and thyroid carcinoma (first radiopharmaceutical approval)"}],"mechanismSteps":["TSH stimulation upregulates the sodium-iodide symporter on thyroid cells","Oral I-131 is absorbed and concentrated in thyroid tissue and iodine-avid metastases","Beta decay deposits radiation within ~1 mm, killing the cells over weeks","Gamma emission allows a post-therapy scan to map uptake"],"dosing":{"route":"Oral capsule or solution","schedule":"1.1 GBq (30 mCi) for remnant ablation (HiLo); 3.7-7.4 GBq for adjuvant or metastatic treatment; low-iodine diet and TSH stimulation beforehand","monitoring":"Post-therapy whole-body scan; thyroglobulin; salivary and marrow function","source":"https://www.thyroid.org/professionals/ata-professional-guidelines/"},"toxicity":[{"event":"Sialadenitis / xerostomia","source":"https://www.thyroid.org/professionals/ata-professional-guidelines/","note":"Dose-dependent; common after repeated high-activity treatment"}],"access":[],"regulatoryEvents":[{"date":"1946","type":"approval","region":"US","note":"First therapeutic use in thyroid cancer (Seidlin); formal approval followed"}]},{"id":"radium-223","kind":"drug","name":"Radium-223 dichloride","aka":[],"tldr":"Radium-223 was the first alpha-emitting drug ever approved (2013). It homes to bone like calcium and treats prostate cancer that has spread only to bone.","summary":"Radium-223 is a calcium mimetic incorporated into bone matrix at sites of metastatic bone turnover, where each decay chain delivers four alpha emissions over a range of a few cells, killing tumour while largely sparing marrow. It is given intravenously to men with symptomatic castration-resistant prostate cancer whose spread is confined to bone without visceral disease. ALSYMPCA showed overall survival of 14.9 versus 11.3 months (HR 0.70) and fewer skeletal events, and it became the first alpha-emitting drug ever approved (2013). ERA 223 showed excess fractures when combined with abiraterone, now contraindicated without bone protection, while PEACE III (2024) with enzalutamide plus bone-protecting agents was positive for rPFS and overall survival. How to sequence it with 177Lu-PSMA therapy is unresolved. Simply put, it is a radioactive calcium look-alike that homes to bone metastases.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Radium-223","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Radium-223%20dichloride"},{"label":"NICE TA412: radium-223 dichloride for treating hormone-relapsed prostate cancer with bone metastases","url":"https://www.nice.org.uk/guidance/ta412"},{"label":"PEACE-3 final overall survival (Annals of Oncology 2026)","url":"https://doi.org/10.1016/j.annonc.2026.02.009"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["targeted-alpha-therapy"],"targets":[],"drugs":[],"companies":["bayer"],"institutions":[],"pathways":[],"terms":["alpha-vs-beta","prostate-crpc-sequencing","prostate-uk-drug-approvals"],"trials":["alsympca"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NICE TA412 recommends radium-223 dichloride for hormone-relapsed prostate cancer with symptomatic bone metastases and no known visceral metastases, only after docetaxel or where docetaxel is contraindicated or unsuitable, with the patient access scheme discount. Two trials define the combination rules: ERA 223 (abiraterone, no mandatory bone protection) produced fractures in 29 against 11 percent with no benefit, and PEACE-3 (enzalutamide, bone-protecting agents mandatory from March 2018) produced median overall survival 38.2 against 32.6 months, hazard ratio 0.76."],"brand":"Xofigo","modality":"Targeted alpha therapy (bone-seeking)","mechanism":"Calcium mimetic incorporated into bone matrix at metastases; four alpha emissions per decay chain.","approvals":[{"region":"US","year":2013,"indication":"Symptomatic bone-metastatic CRPC without visceral disease"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"radotinib","kind":"drug","name":"Radotinib","aka":["Supect"],"tldr":"Radotinib is an oral kinase inhibitor from Il-Yang Pharm. Co., Ltd., in registered phase 3 trials for chronic myeloid leukaemia.","summary":"Radotinib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 3 trial sponsored by Il-Yang Pharm. Co., Ltd., in chronic myeloid leukaemia. A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Radotinib","url":"https://clinicaltrials.gov/search?intr=Radotinib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["cml","cml-chronic-phase"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["il-yang-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03722420","nct03459534"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral kinase inhibitor","mechanism":"A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"raloxifene","kind":"drug","name":"Raloxifene","aka":[],"tldr":"Raloxifene (Evista) is an osteoporosis tablet that also lowers the chance of developing invasive breast cancer in postmenopausal women at higher risk, with fewer womb-cancer problems than tamoxifen.","summary":"Raloxifene was approved in December 1997 for treatment and prevention of postmenopausal osteoporosis and in September 2007 for reduction in the risk of invasive breast cancer in postmenopausal women with osteoporosis or at high risk of breast cancer, on the MORE, CORE and RUTH trials and the head-to-head STAR trial (NSABP P-2), in which raloxifene was about as effective as tamoxifen against invasive breast cancer with fewer uterine cancers and thromboembolic events but less protection against non-invasive disease. It does not treat breast cancer or reduce recurrence. Venous thromboembolism and fatal stroke in women with coronary disease carry a boxed warning.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Raloxifene","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=raloxifene"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/raloxifenehydrochloride"}],"tags":["nci-list","prevention"],"related":["tamoxifen"],"cancers":["breast-hr-positive"],"sections":[],"technologies":["chemoprevention","endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06062810"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Evista","modality":"Selective oestrogen receptor modulator (benzothiophene)","mechanism":"Oestrogen receptor agonist in bone and antagonist in breast and uterus; lowers the incidence of ER-positive invasive breast cancer.","approvals":[{"region":"US","year":1997,"indication":"Treatment and prevention of postmenopausal osteoporosis"},{"region":"US","year":2007,"indication":"Reduction in risk of invasive breast cancer in postmenopausal women with osteoporosis or at high risk"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"raltitrexed","kind":"drug","name":"Raltitrexed","aka":["ZD1694"],"tldr":"Raltitrexed is a three-weekly intravenous antifolate used in Europe, Canada and Australia for advanced bowel cancer, and with cisplatin for mesothelioma, in patients who cannot take fluorouracil.","summary":"Raltitrexed (Tomudex) was developed by ICI and Zeneca as a direct thymidylate synthase inhibitor that does not need the metabolic activation fluorouracil needs. It is approved in the United Kingdom, much of Europe, Canada and Australia for palliative treatment of advanced colorectal cancer and, in several countries, with cisplatin for malignant pleural mesothelioma on the strength of a European phase 3 trial. It was never approved in the United States after early deaths in a comparative trial. Its main use today is as a fluorouracil substitute in patients with cardiac toxicity from fluoropyrimidines.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Raltitrexed","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Raltitrexed"},{"label":"ChEMBL CHEMBL225071","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL225071"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["colorectal","mesothelioma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["tyms"],"drugs":["fluorouracil","pemetrexed"],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00002893","nct05701436","nct05766605"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tomudex","modality":"Antifolate thymidylate synthase inhibitor, intravenous every three weeks","mechanism":"A quinazoline antifolate taken up by the reduced folate carrier and polyglutamated inside cells, where it inhibits thymidylate synthase directly and so starves DNA synthesis of thymidine.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"raludotatug-deruxtecan","kind":"drug","name":"Raludotatug deruxtecan","aka":[],"tldr":"Raludotatug deruxtecan is a CDH6 ADC in phase 3 for platinum-resistant ovarian cancer.","summary":"Raludotatug deruxtecan is an anti-CDH6 IgG1 antibody linked through a cleavable GGFG linker to DXd, a topoisomerase I inhibitor that traps the enzyme on DNA and collapses replication forks; the payload crosses membranes, giving a bystander effect. CDH6 is a cadherin overexpressed in ovarian and renal cell carcinoma with little normal-tissue expression. Developed by Daiichi Sankyo with Merck, it produced an objective response rate around 46 percent in phase 1 in heavily pretreated ovarian cancer, and the REJOICE-Ovarian01 phase 2/3 trial is comparing it with chemotherapy in platinum-resistant disease at 5.6 mg/kg every 3 weeks. Nausea, fatigue, neutropenia and anaemia are the main toxicities, with interstitial lung disease monitored as for other DXd conjugates. Whether the response rate holds in a randomised trial is the open question for platinum-resistant ovarian cancer.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Raludotatug deruxtecan","url":"https://clinicaltrials.gov/search?intr=R-DXd"}],"tags":[],"related":[],"cancers":["ovarian","rcc"],"sections":[],"technologies":["adc"],"targets":["cdh6"],"drugs":[],"companies":["daiichi-sankyo","merck"],"institutions":[],"pathways":[],"terms":["ggfg"],"trials":["nct06843447","nct06864169","nct06780085","nct06660654"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"R-DXd, DS-6000","modality":"ADC","payload":"DXd","linker":"GGFG cleavable","mechanism":"Anti-CDH6 IgG1 with DXd.","approvals":[],"mechanismSteps":["Antibody binds CDH6 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","DXd is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"5.6 mg/kg every 3 weeks (phase 2/3 dose)","monitoring":"ILD, blood counts","source":"https://clinicaltrials.gov/study/NCT06161025"},"toxicity":[{"event":"Nausea"},{"event":"Fatigue"},{"event":"Neutropenia"},{"event":"Anaemia"}],"access":[{"country":"US","reimbursement":"Investigational (REJOICE-Ovarian01)","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-10","type":"filing","region":"US","note":"Merck co-development of R-DXd under the Daiichi Sankyo alliance","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"ramucirumab","kind":"drug","name":"Ramucirumab","aka":[],"tldr":"An antibody that blocks the VEGF receptor, approved in liver cancer only for patients with a high AFP blood level, the first biomarker-selected HCC drug.","summary":"Ramucirumab is a fully human IgG1 antibody that binds the extracellular domain of VEGFR2, blocking VEGF-A, C and D signalling at the receptor rather than the ligand. It is approved in second-line gastric cancer alone or with paclitaxel (RAINBOW, 2014: OS 9.6 versus 7.4 months), in NSCLC with docetaxel (REVEL) and with erlotinib in EGFR-mutant disease, in colorectal cancer with FOLFIRI (RAISE) and in HCC. In HCC, REACH-2 showed overall survival of 8.5 versus 7.3 months in sorafenib-pretreated patients with AFP of 400 ng/mL or more (HR 0.71), after REACH failed in unselected patients, making it the first biomarker-selected HCC drug. Hypertension (25%) and proteinuria are the main toxicities. Its gastric role is being eroded by trastuzumab deruxtecan (DESTINY-Gastric04) and Claudin drugs. For a newcomer, it is the VEGF-receptor antibody given every 2 weeks in several cancers.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Ramucirumab","links":[{"label":"FDA label","url":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/125477s040lbl.pdf"},{"label":"NICE TA403: ramucirumab for previously treated locally advanced or metastatic non-small-cell lung cancer (not recommended)","url":"https://www.nice.org.uk/guidance/ta403"}],"tags":[],"related":[],"cancers":["hcc","gastric","nsclc","colorectal","lung-cancer"],"sections":[],"technologies":["monoclonal-antibody","antiangiogenic"],"targets":["vegf"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":["vegf-angiogenesis"],"terms":["afp"],"trials":["nct02411448","nct06448754","nct07098338","nct06996782","nct06038578","nct05999994","nct06771622","nct05190445","nct06445972","nct06123494","rainbow","destiny-gastric04","raise","lung-map-s1800a"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In stomach cancer: RAINBOW (with paclitaxel) and REGARD (alone) established ramucirumab as second-line treatment of gastric and gastro-oesophageal junction cancer in 2014, given at 8 mg/kg on days 1 and 15 with paclitaxel 80 mg/m2 on days 1, 8 and 15 of a 28-day cycle; it is held for uncontrolled hypertension or proteinuria above 2 g in 24 hours. It is now the comparator arm that trastuzumab deruxtecan (DESTINY-Gastric04) and the CLDN18.2 ADC (CLARITY-Gastric01) have beaten.","In colorectal cancer RAISE showed median overall survival 13.3 against 11.7 months when ramucirumab was added to second-line FOLFIRI after progression on first-line bevacizumab, oxaliplatin and a fluoropyrimidine. It has no NICE recommendation for colorectal cancer and is not routinely funded in England."],"brand":"Cyramza","modality":"Monoclonal antibody (anti-VEGFR2)","mechanism":"Fully human IgG1 binding VEGFR2 ectodomain, blocking VEGF-A/C/D signalling.","approvals":[{"region":"US","year":2014,"indication":"Gastric cancer; NSCLC"},{"region":"US","year":2019,"indication":"HCC with AFP ≥400 ng/mL after sorafenib"},{"region":"England (NICE)","year":2016,"indication":"Locally advanced or metastatic non-small-cell lung cancer progressing after platinum-based chemotherapy, with docetaxel: not recommended","note":"TA403, published 24 August 2016, does not recommend ramucirumab with docetaxel within its marketing authorisation."}],"mechanismSteps":[],"dosing":{"route":"Intravenous","schedule":"8 mg/kg every 2 weeks"},"toxicity":[{"event":"Hypertension","anyGradePct":25,"grade3PlusPct":13,"note":"REACH-2"},{"event":"Proteinuria","anyGradePct":20,"grade3PlusPct":2,"note":"REACH-2"}],"access":[],"regulatoryEvents":[]},{"id":"rasburicase","kind":"drug","name":"Rasburicase","aka":["Fasturtec","Recombinant urate oxidase"],"tldr":"Rasburicase (Elitek) is an enzyme infusion that rapidly dissolves the uric acid released when large numbers of leukaemia or lymphoma cells die at the start of treatment, protecting the kidneys from tumour lysis syndrome.","summary":"Rasburicase was approved by the FDA in July 2002 for initial management of plasma uric acid in children (adults added 2009) with leukaemia, lymphoma or solid tumours receiving therapy expected to cause tumour lysis, on randomised trials against allopurinol in which uric acid normalised within four hours in almost all patients; the EU authorised Fasturtec in 2001. It is standard for high-risk tumour lysis syndrome (Burkitt lymphoma, high-count ALL and AML, venetoclax ramp-up in CLL with bulky disease), often as a single dose rather than the labelled five days. Haemolysis and methaemoglobinaemia in G6PD deficiency, anaphylaxis and interference with uric acid assays (samples must be chilled) carry boxed warnings.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Rasburicase","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=rasburicase"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/rasburicase"},{"label":"EPAR (Fasturtec)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/fasturtec"}],"tags":["nci-list","supportive"],"related":[],"cancers":["all-leukemia","aml","dlbcl","cll"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00199043","nct00057811","nct01200485"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Elitek","modality":"Recombinant enzyme (urate oxidase)","supportive":true,"mechanism":"Recombinant Aspergillus urate oxidase that converts uric acid to allantoin, which is five to ten times more soluble and readily excreted, lowering plasma uric acid within hours.","approvals":[{"region":"US","year":2002,"indication":"Initial management of plasma uric acid in paediatric patients with leukaemia, lymphoma or solid tumours at risk of tumour lysis (adults added 2009)"},{"region":"EU","year":2001,"indication":"Treatment and prophylaxis of acute hyperuricaemia in haematological malignancy with high tumour burden (Fasturtec)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ravulizumab","kind":"drug","name":"Ravulizumab","aka":["Ravulizumab-cwvz","ALXN1210"],"tldr":"Ravulizumab is an eight-weekly infusion that blocks the complement system; it treats paroxysmal nocturnal haemoglobinuria, a clonal bone marrow disorder managed by haematologists alongside aplastic anaemia and myelodysplasia.","summary":"Ravulizumab is a re-engineered, longer-acting successor to eculizumab that blocks complement C5. The United States approved it in 2018 for paroxysmal nocturnal haemoglobinuria (PNH), an acquired clonal disorder of blood stem cells in which complement destroys red cells, and then for atypical haemolytic uraemic syndrome and generalised myasthenia gravis. PNH is not a cancer but arises from the same stem-cell compartment as myelodysplastic syndromes and often follows aplastic anaemia, so it sits within the haematology and bone marrow failure workload of cancer centres. Complement blockade is also being tested for transplant-associated thrombotic microangiopathy after stem cell transplantation. Meningococcal infection risk requires vaccination before treatment.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Ravulizumab","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ravulizumab"},{"label":"Drugs@FDA BLA 761108","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761108"}],"tags":["gap-fill","supportive-care"],"related":[],"cancers":["mds"],"sections":[],"technologies":["supportive-care"],"targets":[],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ultomiris","modality":"Long-acting anti-complement C5 monoclonal antibody","mechanism":"Binds complement protein C5 and prevents its cleavage into C5a and C5b, so the membrane attack complex cannot form and complement-driven destruction of blood cells stops.","approvals":[{"region":"US","year":2018,"indication":"Paroxysmal nocturnal haemoglobinuria","note":"Later extended to atypical haemolytic uraemic syndrome and generalised myasthenia gravis"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rc118-adc","kind":"drug","name":"RC118-ADC","aka":["RC118 for injection"],"tldr":"RC118-ADC is an experimental antibody-drug conjugate from RemeGen in phase 2 trials, aimed at Claudin 18.2.","summary":"RC118-ADC (RC118) is an antibody-drug conjugate developed by RemeGen. Its target is Claudin 18.2 (the sponsor names Claudin 18.2). The sponsor states: An antibody-drug conjugate combining a Claudin 18.2-targeting antibody with a microtubule inhibitor payload. ClinicalTrials.gov describes the intervention as: RC118 for injection is a novel antibody-drug conjugate, with a Claudin 18.2-targeting antibody and a microtube inhibitor. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT05205850, plans to enrol 135 participants with primary completion was scheduled for 2025-09-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of RC118-ADC","url":"https://clinicaltrials.gov/search?intr=RC118"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":["remegen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06038396","nct05205850"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"RC118","modality":"ADC","mechanism":"An antibody-drug conjugate combining a Claudin 18.2-targeting antibody with a microtubule inhibitor payload.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rc148","kind":"drug","name":"RC148","aka":["RC148 plus Platinum-Based Chemotherapy","RC148 for injection"],"tldr":"RC148 is an experimental investigational agent whose form is not stated in the registry from RemeGen in phase 3 trials for non-small-cell lung cancer and HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"RC148 is an investigational agent whose form is not stated in the registry developed by RemeGen. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: 20mg/kg, intravenous infusion, once every 2 weeks. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07416474 (RC148 Plus Platinum-Based Chemotherapy vs Tislelizumab Plus Platinum-Based Chemotherapy for First-Line Squamous Non-Small Cell Lung Cancer (sqNSCLC)), in non-small-cell lung cancer and HR-positive / HER2-negative breast cancer. The largest, NCT07416474, plans to enrol 574 participants with primary completion expected 2027-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of RC148","url":"https://clinicaltrials.gov/search?intr=RC148"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["remegen"],"institutions":[],"pathways":[],"terms":[],"trials":["sqnsclc","nct07462143","nct06038396","nct06642545"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody (PD-1 x VEGF, per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"recombinant-human-endostatin","kind":"drug","name":"Recombinant human endostatin","aka":["Endostar","rh-endostatin"],"tldr":"Endostar is Simcere's recombinant endostatin, approved in China in 2005 with chemotherapy for advanced non-small-cell lung cancer, one of the first angiogenesis inhibitors approved anywhere.","summary":"Recombinant human endostatin (Endostar) was developed in China and is marketed by Simcere in Nanjing. The NMPA (then the SFDA) approved it in September 2005 in combination with vinorelbine and cisplatin for advanced non-small-cell lung cancer, a year after bevacizumab's US approval. It remains widely used in China and is being tested with immunotherapy.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Endostar","url":"https://clinicaltrials.gov/search?intr=Endostar"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["simcere"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00576914","nct01124253","nct02579564"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"modality":"Recombinant angiogenesis inhibitor protein","mechanism":"A recombinant form of endostatin, a fragment of collagen XVIII that stops the growth of new blood vessels a tumour needs.","approvals":[{"region":"CN","year":2005,"indication":"Advanced non-small-cell lung cancer, with vinorelbine and cisplatin"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"regn7075","kind":"drug","name":"REGN7075","aka":["EGFRxCD28 bispecific immunotherapy","marlotamig"],"tldr":"REGN7075 is a bispecific antibody from Regeneron Pharmaceuticals, in registered phase 2 trials for non-small-cell lung cancer, small-cell lung cancer.","summary":"REGN7075 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Regeneron Pharmaceuticals, in non-small-cell lung cancer, small-cell lung cancer. A bispecific antibody, as described in the registry record: one molecule binds two different targets, most often a tumour antigen and an immune cell or a second tumour pathway. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of REGN7075","url":"https://clinicaltrials.gov/search?intr=REGN7075"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc","sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["regeneron"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06465329","nct07154290","nct04626635"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"REGN7075","modality":"Bispecific antibody","mechanism":"A bispecific antibody, as described in the registry record: one molecule binds two different targets, most often a tumour antigen and an immune cell or a second tumour pathway.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"regorafenib","kind":"drug","name":"Regorafenib","aka":[],"tldr":"A sorafenib successor that became the first second-line drug proven to prolong life in liver cancer (2017).","summary":"Regorafenib is an oral multi-kinase inhibitor of VEGFR1-3, TIE2, PDGFR, FGFR, KIT, RET and RAF, a successor to sorafenib with broader targets. It is approved for previously treated metastatic colorectal cancer (CORRECT, 2012), GIST after imatinib and sunitinib (GRID, 2013) and hepatocellular carcinoma after sorafenib (2017). RESORCE showed overall survival of 10.6 versus 7.8 months versus placebo after sorafenib progression (HR 0.63), the first second-line survival benefit in liver cancer; sequencing sorafenib then regorafenib gave a median 26 months from sorafenib start. Hand-foot skin reaction (53%, 13% grade 3 or higher), hypertension and fatigue are common at 160 mg, so start-low escalation (ReDOS in colorectal cancer) is widely used. Its colorectal benefit is small, and fruquintinib now competes there. For a newcomer, it is a broad anti-angiogenic pill used late in three cancers.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Regorafenib","links":[{"label":"FDA label","url":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/203085s013lbl.pdf"},{"label":"NICE TA866: regorafenib for previously treated metastatic colorectal cancer","url":"https://www.nice.org.uk/guidance/ta866"},{"label":"EMA Stivarga","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/stivarga"}],"tags":[],"related":[],"cancers":["hcc","hcc-advanced","colorectal","sarcoma","gist-imatinib-resistant"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf","kit","ret"],"drugs":[],"companies":["bayer"],"institutions":[],"pathways":[],"terms":[],"trials":["resorce","nct06199973","nct05425940","nct04701476","nct02955940","nct07310173","nct06047379","nct06246643","correct","concur","imblaze370"],"people":[],"bottlenecks":[],"keyPapers":["paper-grid-regorafenib-gist-lancet-2013"],"journals":[],"dependsOn":[],"notes":["In bowel cancer: CORRECT (2013) gave OS 6.4 versus 5.0 months in refractory metastatic disease, a modest benefit with significant side effects, so it is now usually placed after trifluridine/tipiracil. ReDOS (start at 80 mg and escalate weekly) improves tolerability; hepatotoxicity carries a boxed warning, with liver function checked every 2 weeks for the first 2 months."],"brand":"Stivarga","modality":"Small-molecule multi-kinase inhibitor","mechanism":"Oral inhibitor of VEGFR1-3, TIE2, PDGFR, FGFR, KIT, RET and RAF.","approvals":[{"region":"US","year":2012,"indication":"Metastatic colorectal cancer, previously treated"},{"region":"US","year":2013,"indication":"GIST after imatinib and sunitinib"},{"region":"US","year":2017,"indication":"HCC previously treated with sorafenib"},{"region":"US","year":2012,"indication":"Metastatic colorectal cancer after fluoropyrimidine, oxaliplatin, irinotecan, anti-VEGF and, if RAS wild-type, anti-EGFR therapy","note":"Approved September 2012 on CORRECT."},{"region":"EU","year":2013,"indication":"Metastatic colorectal cancer after available therapies","note":"Stivarga marketing authorisation issued 26 August 2013."},{"region":"England (NICE)","year":2023,"indication":"Metastatic colorectal cancer after previous treatment or when those treatments are unsuitable","note":"TA866, published 8 February 2023, recommends it within its marketing authorisation subject to the commercial arrangement, a decade after the FDA approval."}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"160 mg daily for 3 weeks of each 4-week cycle","modifications":"Start-low (80-120 mg) escalation strategies (ReDOS in CRC) improve tolerability"},"toxicity":[{"event":"Hand-foot skin reaction","anyGradePct":53,"grade3PlusPct":13,"note":"RESORCE"},{"event":"Hypertension","anyGradePct":31,"grade3PlusPct":15,"note":"RESORCE"},{"event":"Fatigue","anyGradePct":40,"grade3PlusPct":9,"note":"RESORCE"}],"access":[],"regulatoryEvents":[]},{"id":"relacorilant","kind":"drug","name":"Relacorilant","aka":[],"tldr":"A first-in-class drug that blocks cortisol signalling in tumour cells, approved in 2026 for platinum-resistant ovarian cancer with chemotherapy.","summary":"Relacorilant is a selective glucocorticoid receptor antagonist that blocks cortisol signalling inside tumour cells, where glucocorticoid receptor activation is a mechanism of chemotherapy resistance, so it restores sensitivity to the taxane given alongside it. Corcept's ROSELLA phase 3 trial showed that relacorilant plus nab-paclitaxel improved both progression-free and overall survival versus nab-paclitaxel alone, and it was approved in Q1 2026 for platinum-resistant ovarian, fallopian tube or primary peritoneal cancer, the first drug in its class. It is taken as 150 mg the day before, the day of and the day after each weekly nab-paclitaxel dose. Whether the approach extends to other chemotherapy-resistant cancers is open. For a newcomer: a drug that removes a stress-hormone shield so chemotherapy works again.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Relacorilant"}],"tags":[],"related":[],"cancers":["ovarian","platinum-resistant-ovarian-cancer","advanced-adrenocortical-carcinoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["corcept"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07259317","nct06906341"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lifyorli","modality":"Small-molecule glucocorticoid receptor antagonist","mechanism":"Selective GR antagonist restoring chemosensitivity.","approvals":[{"region":"US","year":2026,"indication":"Platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer with nab-paclitaxel"}],"mechanismSteps":["Cortisol binds glucocorticoid receptors in tumour cells and switches on anti-apoptotic genes","Relacorilant blocks the glucocorticoid receptor selectively (no progesterone receptor effect)","Pro-survival signalling induced by stress hormones is removed","Tumour cells regain sensitivity to nab-paclitaxel"],"dosing":{"route":"Oral","schedule":"150 mg once daily the day before, day of, and day after each weekly nab-paclitaxel dose (ROSELLA schedule)","monitoring":"Electrolytes, blood pressure, neuropathy","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Neutropenia"},{"event":"Anaemia"},{"event":"Fatigue"},{"event":"Nausea"},{"event":"Peripheral neuropathy"},{"event":"Hypokalaemia"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2025-06","type":"filing","region":"US","note":"NDA submitted (ROSELLA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q1","type":"approval","region":"US","note":"Platinum-resistant ovarian, fallopian tube, or primary peritoneal cancer with nab-paclitaxel","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"relatlimab-nivolumab","kind":"drug","name":"Relatlimab + nivolumab","aka":[],"tldr":"Opdualag combines relatlimab, the first drug targeting the LAG-3 immune brake, with nivolumab for melanoma.","summary":"Opdualag is a fixed-dose combination of relatlimab, an antibody against the LAG-3 checkpoint, and nivolumab, an anti-PD-1 antibody, given as one infusion of nivolumab 480 mg with relatlimab 160 mg every 4 weeks. Because LAG-3 and PD-1 sit on the same exhausted T cells, releasing both brakes at once restores T-cell function more fully than PD-1 blockade alone. In RELATIVITY-047, in untreated advanced melanoma, progression-free survival was 10.1 versus 4.6 months compared with nivolumab alone, with less toxicity than ipilimumab-nivolumab. It was approved in 2022 in the US for unresectable or metastatic melanoma in patients aged 12 and over, the first LAG-3 therapy, and in the EU for PD-L1-negative melanoma. Fatigue, rash, thyroid dysfunction and adrenal insufficiency occur, and myocarditis is rare but monitored. Adjuvant and other tumour trials are ongoing.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Nivolumab/relatlimab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Relatlimab"}],"tags":[],"related":[],"cancers":["melanoma","advanced-melanoma"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["lag3","pd1"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06246916","nct05625399","nct06101134","nct05970497"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Opdualag","modality":"Fixed-dose bispecific combination (anti-LAG-3 + anti-PD-1)","mechanism":"Anti-LAG-3 plus anti-PD-1 in one infusion.","approvals":[{"region":"US","year":2022,"indication":"Unresectable or metastatic melanoma, age ≥12"},{"region":"EU","year":2022,"indication":"Melanoma, PD-L1 <1% restriction"}],"mechanismSteps":["Relatlimab binds LAG-3 and nivolumab binds PD-1 on the same exhausted T cells","Two inhibitory checkpoints are released simultaneously","T cells regain proliferation and cytotoxic function","Tumour is infiltrated and killed; less toxicity than CTLA-4 combination"],"dosing":{"route":"IV infusion","schedule":"Nivolumab 480 mg + relatlimab 160 mg every 4 weeks (≥40 kg)","modifications":"As for nivolumab; hold for grade 2 immune-mediated events","monitoring":"Thyroid, LFTs, creatinine, glucose; adrenal function; myocarditis awareness","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Fatigue"},{"event":"Musculoskeletal pain"},{"event":"Rash"},{"event":"Pruritus"},{"event":"Diarrhoea"},{"event":"Hypothyroidism/thyroiditis"},{"event":"Adrenal insufficiency"},{"event":"Myocarditis (rare, monitor troponin)"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.bmsaccesssupport.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for untreated advanced melanoma, PD-L1 <1% (2024)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2022-03-18","type":"approval","region":"US","note":"Unresectable or metastatic melanoma, age ≥12 (RELATIVITY-047): first LAG-3 therapy","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-09","type":"approval","region":"EU","note":"EMA approval, PD-L1 <1% melanoma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"relmacabtagene-autoleucel","kind":"drug","name":"Relmacabtagene autoleucel","aka":[],"tldr":"Relma-cel was the first CAR-T therapy developed and made in China to be approved, for large B-cell lymphoma that has come back after two treatments.","summary":"Approved by the NMPA in September 2021 for relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, three months after the Fosun Kite import of axicabtagene ciloleucel became China's first CAR-T approval; relapsed or refractory follicular lymphoma followed in 2022. The pivotal RELIANCE study (59 patients) reported an objective response rate of 75.9% and complete responses in 51.7%, with low rates of severe cytokine release syndrome. JW Therapeutics was formed in 2016 as a joint venture between Juno Therapeutics and WuXi AppTec, and the product is the Chinese sibling of lisocabtagene maraleucel.","status":"approved","asOf":"2026-09-10","links":[{"label":"JW Therapeutics","url":"https://www.jwtherapeutics.com/en/"},{"label":"RELIANCE (Cancer Med 2021)","url":"https://doi.org/10.1002/cam4.3686"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["dlbcl","follicular-lymphoma"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":[],"companies":["jw-therapeutics","wuxi-apptec"],"institutions":[],"pathways":[],"terms":[],"trials":["reliance","nct06093841","nct06479356"],"people":[],"bottlenecks":[],"keyPapers":["paper-ying-cancer-med"],"journals":[],"dependsOn":[],"notes":[],"brand":"Carteyva","code":"relma-cel, JWCAR029","modality":"Cell therapy (autologous CD19 CAR-T, 4-1BB)","mechanism":"Autologous T cells transduced with a CD19-directed CAR (4-1BB co-stimulatory domain) derived from the JCAR017 construct, manufactured in Shanghai by JW Therapeutics.","approvals":[{"region":"China","year":2021,"indication":"Relapsed or refractory large B-cell lymphoma after two or more lines"},{"region":"China","year":2022,"indication":"Relapsed or refractory follicular lymphoma after two or more lines"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"relugolix","kind":"drug","name":"Relugolix","aka":[],"tldr":"Relugolix is the first hormone-suppressing pill for prostate cancer, working within days and wearing off quickly when stopped.","summary":"Relugolix is an oral, competitive GnRH receptor antagonist that suppresses testosterone within days without the initial testosterone flare seen with GnRH agonists such as leuprolide. It is used for advanced prostate cancer wherever medical castration is needed, and its effect wears off quickly when stopped, which suits intermittent therapy or men with a short planned course. HERO (2020) showed sustained castration in 96.7% versus 88.8% for leuprolide, faster onset and recovery, and 54% fewer major cardiovascular events. Drug interactions through P-glycoprotein and reliance on daily adherence are practical considerations against the convenience of a depot injection. Whether the cardiovascular signal is a true class advantage of antagonists is still under study. In short, relugolix is the first hormone-suppressing pill for prostate cancer.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Relugolix","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Relugolix"},{"label":"NICE TA995: relugolix for treating hormone-sensitive prostate cancer","url":"https://www.nice.org.uk/guidance/ta995"}],"tags":[],"related":[],"cancers":["prostate"],"sections":[],"technologies":["androgen-deprivation"],"targets":[],"drugs":[],"companies":["sumitomo-pharma"],"institutions":[],"pathways":["ar-signaling"],"terms":["prostate-adt-burden","prostate-uk-drug-approvals"],"trials":["nct05862272"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NICE TA995 recommends relugolix within its marketing authorisation for advanced hormone-sensitive prostate cancer, alongside radiotherapy for high-risk localised or locally advanced hormone-sensitive disease, and as neoadjuvant treatment before radiotherapy. The committee's reasoning was that clinical trial evidence suggests relugolix reduces testosterone to castrate levels more reliably in the long term and reduces the risk of serious cardiovascular events compared with leuprolide, and that an indirect comparison, though uncertain, suggests it works as well as other forms of androgen deprivation."],"brand":"Orgovyx","modality":"Oral GnRH antagonist","mechanism":"Competitive GnRH receptor antagonist; no testosterone flare.","approvals":[{"region":"US","year":2020,"indication":"Advanced prostate cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rem-422","kind":"drug","name":"REM-422","aka":[],"tldr":"REM-422 is a small-molecule inhibitor from Remix Therapeutics, in registered phase 2 trials for salivary gland cancers.","summary":"REM-422 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Remix Therapeutics, in salivary gland cancers. Described in the registry record as a small molecule mrna inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of REM-422","url":"https://clinicaltrials.gov/search?intr=REM-422"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["salivary-gland","adenoid-cystic-carcinoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["remix-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06118086"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"REM-422","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a small molecule mrna inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"remestemcel-l","kind":"drug","name":"Remestemcel-L","aka":[],"tldr":"Donor bone marrow stromal cells given to children whose acute graft-versus-host disease no longer responds to steroids, the first mesenchymal stromal cell therapy approved in the United States.","summary":"Remestemcel-L is a suspension of culture-expanded mesenchymal stromal cells from the bone marrow of healthy adult donors. The cells dampen the T-cell attack on skin, gut and liver that defines acute graft-versus-host disease after an allogeneic stem cell transplant. The FDA approved it in December 2024 for steroid-refractory acute graft-versus-host disease in children aged two months and older, on a single-arm trial in which most children responded within a month. It is given intravenously twice a week for four weeks and is the first mesenchymal stromal cell product approved in the United States.","status":"approved","asOf":"2026-09-24","links":[{"label":"MSB-GVHD001 (Biol Blood Marrow Transplant 2020)","url":"https://doi.org/10.1016/j.bbmt.2020.01.018"},{"label":"Ryoncil label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=a4e0918f-7444-4694-adeb-d38d98345659"},{"label":"FDA approval announcement (December 2024)","url":"https://www.fda.gov/news-events/press-announcements/fda-approves-first-mesenchymal-stromal-cell-therapy-treat-steroid-refractory-acute-graft-versus-host"}],"tags":[],"related":[],"cancers":[],"sections":["cell-therapy","supportive-care"],"technologies":["gvhd-ruxolitinib-steroid-refractory"],"targets":[],"drugs":[],"companies":["mesoblast"],"institutions":[],"pathways":[],"terms":["gvhd"],"trials":["msb-gvhd001"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ryoncil","modality":"Allogeneic bone marrow-derived mesenchymal stromal cell therapy","supportive":true,"mechanism":"Mesenchymal stromal cells secrete anti-inflammatory factors and suppress activated T cells, reducing tissue damage in acute graft-versus-host disease.","approvals":[{"region":"US","year":2024,"indication":"Steroid-refractory acute graft-versus-host disease in children aged two months and older"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"repotrectinib","kind":"drug","name":"Repotrectinib","aka":[],"tldr":"A ROS1 and NTRK pill that also works after other ROS1 drugs fail, with dizziness as its signature side effect.","summary":"Repotrectinib is a compact macrocyclic ROS1 and TRK inhibitor whose small size lets it bind kinases carrying solvent-front mutations such as ROS1 G2032R, the usual escape route from crizotinib and entrectinib. It is used in ROS1-positive non-small-cell lung cancer both in patients who have never had a ROS1 drug and in those whose disease has progressed on one, and in NTRK-fusion solid tumours of any type. In TRIDENT-1 the response rate was 79% in TKI-naive and 38% in TKI-pretreated ROS1-positive NSCLC. It was approved in November 2023 for ROS1 and June 2024 for NTRK fusions (tumour-agnostic). Dizziness and other neurological effects from TRK inhibition are its signature toxicity, and zidesamtinib (2026) offers a TRK-sparing alternative with fewer neurologic effects. In short, it is the ROS1 pill that still works after other ROS1 drugs fail.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Repotrectinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Repotrectinib"}],"tags":[],"related":["ros1-fusion"],"cancers":["nsclc","ros1-positive-nsclc","ntrk-fusion-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["ros1","ntrk"],"drugs":["zidesamtinib"],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04094610","nct03093116","nct06140836"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Augtyro","modality":"Small-molecule kinase inhibitor (ROS1 / NTRK)","mechanism":"Compact macrocyclic ROS1/TRK inhibitor active against solvent-front mutations.","approvals":[{"region":"US","year":2023,"indication":"ROS1+ locally advanced or metastatic NSCLC"},{"region":"US","year":2024,"indication":"NTRK-fusion solid tumours (tumour-agnostic)"},{"region":"EU","year":2025,"indication":"ROS1+ NSCLC; NTRK+ solid tumours; 13 Jan 2025 (conditional)","note":"Conditional marketing authorisation"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2024-06-13","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 2.3 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-repotrectinib-adult-and-pediatric-patients-ntrk-gene-fusion-positive","indication":"Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic where surgical resection is likely results in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy"}]},{"id":"resminostat","kind":"drug","name":"Resminostat","aka":["resminostat","resminostat mesilate","Kinselby","4SC-201"],"tldr":"Resminostat is an oral drug of the HDAC inhibitor class, proposed for advanced mycosis fungoides and Sézary syndrome, two cutaneous T-cell lymphomas. Europe's medicines committee said no to it in May 2025, so it is not authorised.","summary":"Resminostat (Kinselby, applicant 4SC AG, orphan designation) was proposed for the treatment of patients with advanced-stage mycosis fungoides and Sézary syndrome. The CHMP adopted its opinion on 22 May 2025 and the opinion status on the EMA product page is negative, so there is no EU marketing authorisation. The active substance is resminostat mesilate; the EMA lists the therapeutic area as mycosis fungoides. The corpus's approved HDAC inhibitors in cutaneous T-cell lymphoma are romidepsin and vorinostat (target page `hdac`).","asOf":"2026-09-22","links":[{"label":"EMA: Kinselby (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/kinselby"}],"tags":["ema-register"],"related":["romidepsin"],"cancers":["cutaneous-t-cell-lymphoma"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["hdac"],"drugs":[],"companies":["4sc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02953301","nct00943449","nct01037478"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo content wave 5 (EMA medicines register and EPAR pages)","editedOn":"2026-09-22"},"brand":"Kinselby","modality":"Small-molecule HDAC inhibitor","mechanism":"Oral histone deacetylase inhibitor proposed for advanced-stage mycosis fungoides and Sézary syndrome; the EMA's committee adopted a negative opinion on the marketing application (EMA product page).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2025-05-22","type":"crl","region":"EU","note":"CHMP negative opinion on Kinselby (resminostat) for advanced mycosis fungoides and Sézary syndrome","source":"https://www.ema.europa.eu/en/medicines/human/EPAR/kinselby"}]},{"id":"resolution-ctdx-first","kind":"drug","name":"Resolution ctDx FIRST","aka":[],"tldr":"A blood test approved with adagrasib to find the KRAS G12C mutation in lung cancer when a tissue biopsy is not possible.","summary":"Agilent's Resolution ctDx FIRST was approved by the FDA in December 2022 alongside adagrasib as a liquid biopsy companion diagnostic for KRAS G12C-mutant non-small-cell lung cancer, with tumour-profiling claims for other lung cancer genes. It is the second liquid comprehensive genomic profiling test with a companion claim after Guardant360 CDx and FoundationOne Liquid CDx. As with all plasma tests, a negative result should be followed by tissue testing.","status":"approved","asOf":"2026-09-10","links":[{"label":"Agilent: Resolution ctDx FIRST","url":"https://www.agilent.com/en/product/liquid-biopsy/resolution-ctdx-first"}],"tags":["test"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":["liquid-biopsy","companion-diagnostic"],"targets":["kras"],"drugs":["adagrasib"],"companies":["agilent"],"institutions":[],"pathways":[],"terms":["ctdna"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: KRAS G12C detected in blood makes adagrasib or sotorasib an option after first-line treatment."],"brand":"Resolution ctDx FIRST","modality":"Liquid biopsy companion diagnostic test (NGS, cfDNA)","mechanism":"Targeted NGS of plasma cell-free DNA covering lung cancer driver genes, with a companion claim for KRAS G12C.","approvals":[{"region":"US","year":2022,"indication":"Companion diagnostic for adagrasib in KRAS G12C-mutant NSCLC (plasma)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"retifanlimab","kind":"drug","name":"Retifanlimab","aka":[],"tldr":"Retifanlimab is a PD-1 antibody approved for Merkel cell carcinoma and, with chemotherapy, as the first immunotherapy standard for advanced anal cancer.","summary":"Retifanlimab is an IgG4 monoclonal antibody that blocks PD-1 binding to PD-L1 and PD-L2, restoring T-cell activity against tumours. It is used in metastatic or recurrent locally advanced Merkel cell carcinoma and in advanced anal squamous cell carcinoma. POD1UM-201 showed a 52% response rate in chemotherapy-naive metastatic Merkel cell carcinoma, supporting accelerated US approval in 2023 and EU approval in 2024. POD1UM-303/InterAACT-2 (Lancet 2025) randomised patients with first-line advanced anal SCC to carboplatin-paclitaxel with or without retifanlimab, and adding the antibody improved both progression-free and overall survival, leading to approval in 2025 for the combination and for monotherapy after platinum. It is the first immunotherapy standard for anal cancer. Retifanlimab shows that adding checkpoint blockade to chemotherapy can lengthen survival in a virus-driven cancer.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Retifanlimab","links":[{"label":"POD1UM-303 (Lancet 2025)","url":"https://doi.org/10.1016/S0140-6736(25)00631-2"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=retifanlimab"}],"tags":["gap-fill"],"related":[],"cancers":["merkel-cell-carcinoma","anal","metastatic-anal-cancer","skin-cancer"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["incyte"],"institutions":[],"pathways":["pd1-checkpoint"],"terms":[],"trials":["nct05287113","nct06072612","pod1um-201"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zynyz","modality":"Anti-PD-1 monoclonal antibody (IgG4)","mechanism":"Blocks PD-1 binding to PD-L1/PD-L2, restoring T-cell activity.","approvals":[{"region":"US","year":2023,"indication":"Metastatic or recurrent locally advanced Merkel cell carcinoma"},{"region":"US","year":2025,"indication":"Locally recurrent or metastatic anal SCC with carboplatin-paclitaxel; monotherapy after platinum"},{"region":"EU","year":2024,"indication":"Merkel cell carcinoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2023-03-22","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-retifanlimab-dlwr-metastatic-or-recurrent-locally-advanced-merkel","indication":"Adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC)"},{"date":"2025-12-17","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2023 converted to traditional approval 2.7 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-retifanlimab-dlwr-metastatic-or-recurrent-locally-advanced-merkel","indication":"Adult patients with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC)"}]},{"id":"retlirafusp-alfa","kind":"drug","name":"Retlirafusp alfa","aka":[],"tldr":"Retlirafusp alfa is Hengrui's PD-L1 and TGF-beta bifunctional antibody, in phase 3 trials with chemotherapy in gastric cancer after the Western class leader bintrafusp alfa failed.","summary":"SHR-1701 combines PD-L1 blockade with a TGF-beta trap in a single molecule. Hengrui and Suncadia are running phase 3 trials with capecitabine and oxaliplatin (CAPOX) in first-line advanced gastric and gastro-oesophageal junction cancer, and further studies in cervical and other cancers, after Merck KGaA's bintrafusp alfa was discontinued in 2023 when its lung and biliary trials failed.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Retlirafusp alfa","url":"https://clinicaltrials.gov/search?intr=SHR-1701"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric"],"sections":[],"technologies":["checkpoint-inhibitor","bispecific-antibody"],"targets":["pdl1"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04950322","nct07175220","nct05300269","nct07102901","nct05671822"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"SHR-1701","modality":"Bifunctional fusion protein (anti-PD-L1 antibody fused to a TGF-beta receptor II trap), intravenous","mechanism":"One arm blocks PD-L1 while the fused receptor domain soaks up TGF-beta in the tumour, attacking two immunosuppressive signals at once; the class was pioneered by bintrafusp alfa.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"revumenib","kind":"drug","name":"Revumenib","aka":[],"tldr":"Revumenib (Revuforj) is the first menin inhibitor (2024), for acute leukaemias with KMT2A rearrangements or NPM1 mutations.","summary":"Revumenib is a small-molecule menin inhibitor that disrupts the menin-KMT2A interaction, releasing the differentiation block that keeps KMT2A-rearranged and NPM1-mutant leukaemia cells immature. It was approved in 2024, the first menin inhibitor, for relapsed or refractory KMT2A-rearranged acute leukaemia, on AUGMENT-101 (CR/CRh about 23% in heavily pretreated patients), and in 2025 for relapsed or refractory NPM1-mutant AML. Dosing is 270 mg twice daily for patients of 40 kg or more, reduced to 160 mg with strong CYP3A4 inhibitors. Differentiation syndrome and QT prolongation are the key risks. Syndax developed it and ziftomenib (Kura/Kyowa Kirin) was approved in 2025; acquired MEN1 mutations cause resistance, and front-line combinations with venetoclax and azacitidine are being tested. For a newcomer: a pill that makes leukaemia cells grow up instead of multiplying.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Revumenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Revumenib"}],"tags":[],"related":["npm1-mutation"],"cancers":["aml","all-leukemia","aml-npm1-kmt2a"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["menin"],"drugs":[],"companies":["syndax"],"institutions":[],"pathways":["menin-kmt2a"],"terms":[],"trials":["nct07211958"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Revuforj","modality":"Small-molecule menin inhibitor","mechanism":"Disrupts menin-KMT2A interaction, releasing differentiation block.","approvals":[{"region":"US","year":2024,"indication":"Relapsed/refractory KMT2A-rearranged acute leukaemia, age ≥1"},{"region":"US","year":2025,"indication":"Relapsed/refractory NPM1-mutant AML"}],"mechanismSteps":["Revumenib binds menin in the nucleus","Menin can no longer scaffold KMT2A fusion proteins onto chromatin","HOXA9 and MEIS1 leukaemia programmes switch off","Blasts differentiate into mature myeloid cells (differentiation syndrome risk)","Leukaemic clone shrinks"],"dosing":{"route":"Oral","schedule":"270 mg twice daily (≥40 kg) or 160 mg BID with strong CYP3A4 inhibitors; weight-based below 40 kg","modifications":"Hold for QTc >500 ms or grade 3 differentiation syndrome; steroids for differentiation syndrome","monitoring":"ECG before and during treatment; electrolytes; signs of differentiation syndrome","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Differentiation syndrome","note":"AUGMENT-101: ~27% any grade; boxed warning"},{"event":"QTc prolongation"},{"event":"Nausea"},{"event":"Febrile neutropenia"},{"event":"Haemorrhage"},{"event":"Infections"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2022-12","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-11-15","type":"approval","region":"US","note":"Relapsed/refractory KMT2A-rearranged acute leukaemia, age ≥1: first menin inhibitor","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-10","type":"approval","region":"US","note":"Relapsed/refractory NPM1-mutant AML","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"rezvilutamide","kind":"drug","name":"Rezvilutamide","aka":[],"tldr":"Rezvilutamide is Hengrui's androgen receptor blocker, approved in China in 2022 for high-volume metastatic hormone-sensitive prostate cancer on the CHART trial, where it improved survival over bicalutamide.","summary":"Jiangsu Hengrui's rezvilutamide (SHR3680) received NMPA approval in June 2022 for high-volume metastatic hormone-sensitive prostate cancer with androgen deprivation, after the CHART phase 3 trial showed longer radiographic progression-free and overall survival than bicalutamide. Further phase 3 trials in non-metastatic and castration-resistant disease are registered. It is not approved outside China.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Rezvilutamide","url":"https://clinicaltrials.gov/search?intr=SHR3680"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":["endocrine-therapy"],"targets":["androgen-receptor"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07241416","nct07230106","nct07407283"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"SHR3680","modality":"Oral androgen receptor antagonist","mechanism":"A second-generation antagonist of the androgen receptor, the class of enzalutamide, apalutamide and darolutamide, blocking androgen signalling in prostate cancer cells.","approvals":[{"region":"CN","year":2022,"indication":"High-volume metastatic hormone-sensitive prostate cancer with androgen deprivation therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ribociclib","kind":"drug","name":"Ribociclib","aka":["Ribociclib Succinate and Letrozole","Kisqali Femara Co-Pack"],"tldr":"Ribociclib is the CDK4/6 inhibitor with the most consistent survival benefit, approved for a broad population of early breast cancer patients since 2024.","summary":"Ribociclib is a CDK4-biased CDK4/6 inhibitor given for 21 days of a 28-day cycle with an aromatase inhibitor or fulvestrant, at 600 mg daily in advanced disease or 400 mg daily for 3 years in the adjuvant setting. Its survival data are the most consistent in the class: MONALEESA-2, -3 and -7 each showed an overall survival benefit in HR-positive, HER2-negative advanced breast cancer, and NATALEE extended it to stage II-III early disease, including node-negative tumours, with an invasive disease-free survival HR of 0.75 (4-year iDFS 88.5% versus 83.6%), leading to adjuvant approval in September 2024. Novartis markets it. Neutropenia is near-universal (94%), and QT and liver enzymes need monitoring. Whether the adjuvant benefit matures into a survival gain remains open. For a newcomer: the CDK4/6 pill with the broadest proven benefit in hormone-driven breast cancer.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Ribociclib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ribociclib"}],"tags":[],"related":[],"cancers":["breast-hr-positive","hr-positive-early-high-risk"],"sections":[],"technologies":["cdk46-inhibitor"],"targets":["cdk4-6"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["natalee","nct06726148","nct05867251","nct05358249","nct04417621","nct05768139","nct06065748","nct07002177","nct05563220","nct07085767","nct06757634","nct04862663","nct05573555","nct06380751","nct07647328","nct07405801","nct05827081","nct06760637","nct07054190"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Kisqali","modality":"Small-molecule CDK4/6 inhibitor","mechanism":"CDK4-biased CDK4/6 inhibitor.","approvals":[{"region":"US","year":2017,"indication":"HR+/HER2- advanced breast cancer"},{"region":"US","year":2024,"indication":"Adjuvant stage II-III HR+/HER2- breast cancer at high risk of recurrence"}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of CDK4 and CDK6 (CDK4-biased)","Phosphorylation of downstream substrates stops","G1 arrest in ER-driven cells; combined endocrine blockade removes the cyclin D1 drive","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"600 mg once daily (advanced) or 400 mg once daily (adjuvant, 3 years) for 21 days then 7 off, with aromatase inhibitor or fulvestrant","modifications":"Reduce to 400 then 200 mg for QTc >480 ms, hepatotoxicity, neutropenia","monitoring":"ECG at baseline, day 14 cycle 1, start of cycle 2; LFTs every 2 weeks for 2 cycles; blood counts; avoid strong CYP3A inhibitors and tamoxifen","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8"},"toxicity":[{"event":"Neutrophils decreased","anyGradePct":94,"grade3PlusPct":45,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"NATALEE / MONALEESA-2"},{"event":"Leukocytes decreased","anyGradePct":95,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"NATALEE / MONALEESA-2"},{"event":"Lymphocytes decreased","anyGradePct":97,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"NATALEE / MONALEESA-2"},{"event":"Infections","anyGradePct":37,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"NATALEE / MONALEESA-2"},{"event":"ALT/AST increased (grade 3-4)","grade3PlusPct":8,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"8-11% across trials"},{"event":"Nausea","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"NATALEE / MONALEESA-2"},{"event":"Fatigue","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"NATALEE / MONALEESA-2"},{"event":"Diarrhoea","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"NATALEE / MONALEESA-2"},{"event":"QT prolongation","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=aaeaef94-f3f5-4367-8ea2-b181d7be2da8","note":"Concentration-dependent; ECG monitoring required"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.novartis.com/us-en/patient-support","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with aromatase inhibitor (TA496), fulvestrant (TA687), and adjuvant (2025)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-03-13","type":"approval","region":"US","note":"With aromatase inhibitor, HR+/HER2- advanced breast cancer (MONALEESA-2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2018-07-18","type":"approval","region":"US","note":"Pre/perimenopausal women and with fulvestrant (MONALEESA-7/-3)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-09-17","type":"approval","region":"US","note":"Adjuvant stage II-III HR+/HER2- early breast cancer at high risk (NATALEE)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"rilvegostomig","kind":"drug","name":"Rilvegostomig","aka":[],"tldr":"Rilvegostomig is an experimental bispecific antibody from AstraZeneca in phase 2 trials for non-small-cell lung cancer, aimed at PD-1 and TIGIT.","summary":"Rilvegostomig (AZD2936) is a bispecific antibody developed by AstraZeneca. Its targets are PD-1 and TIGIT (the sponsor names TIGIT x PD-1). The sponsor states: Rilvegostomig (AZD2936) is an anti-TIGIT/anti-PD-1 bispecific antibody given intravenously as monotherapy to enhance anti-tumour immune response in advanced/metastatic NSCLC with PD-L1 expression. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT04995523, plans to enrol 212 participants (actual) with primary completion was scheduled for 2026-08-28 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Rilvegostomig","url":"https://clinicaltrials.gov/search?intr=Rilvegostomig"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["pd1","tigit"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06692738","nct06564844","nct06989112","nct06921785","nct06357533","nct06627647","nct05775159","nct07098338","nct06996782","nct05123482","nct07115043","nct05489211","nct07720284","nct05702229","nct06764875","nct06467357","nct06109779","nct07431281","nct07069712","nct06868277","nct07221253","nct04995523"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AZD2936","modality":"bispecific antibody","mechanism":"Rilvegostomig (AZD2936) is an anti-TIGIT/anti-PD-1 bispecific antibody given intravenously as monotherapy to enhance anti-tumour immune response in advanced/metastatic NSCLC with PD-L1 expression.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rinatabart-sesutecan","kind":"drug","name":"Rinatabart sesutecan","aka":[],"tldr":"Rinatabart sesutecan is a next-generation folate-receptor ADC with a topoisomerase payload that responds in both ovarian and endometrial cancer regardless of receptor level.","summary":"Genmab (via ProfoundBio, 2024). RAINFOL-01: ORR 55.6% in ovarian cancer at 120 mg/m² and 50% in heavily pretreated endometrial cancer at 100 mg/m², the latter earning Breakthrough Therapy designation. Phase 3 RAINFOL-02 versus chemotherapy in platinum-resistant ovarian cancer is enrolling. Neutropenia and anaemia are dose-limiting; ocular toxicity is minimal compared with mirvetuximab.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT05579366 (RAINFOL-01 (Rina-S))","url":"https://clinicaltrials.gov/study/NCT05579366"}],"tags":[],"related":[],"cancers":["ovarian","endometrial"],"sections":[],"technologies":["adc","topoisomerase-inhibitors"],"targets":["folr1"],"drugs":[],"companies":["genmab"],"institutions":[],"pathways":[],"terms":[],"trials":["rainfol-01","nct07604766","nct07166094","nct06619236","nct07225270","nct07564141","nct07288177"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"Rina-S, PRO1184","modality":"ADC","payload":"Exatecan (TOP1 inhibitor), DAR 8","linker":"Hydrophilic cleavable (ProfoundBio)","mechanism":"Anti-FRα IgG1 with a hydrophilic linker carrying exatecan; bystander killing extends activity to FRα-low tumours.","approvals":[],"mechanismSteps":["Binds folate receptor alpha on the tumour cell","Internalised and trafficked to lysosomes","Linker cleaved; exatecan released and diffuses to neighbours","Topoisomerase I trapped; replication collapses in dividing cells"],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2025","type":"designation","region":"US","note":"Breakthrough Therapy designation, advanced endometrial cancer after PD-(L)1 and platinum","source":"https://www.cancernetwork.com/view/rina-s-earns-fda-breakthrough-therapy-designation-in-endometrial-cancer"}]},{"id":"rindopepimut","kind":"drug","name":"Rindopepimut","aka":[],"tldr":"Rindopepimut is a peptide vaccine against EGFRvIII, a mutant protein found only on some glioblastomas. After a phase 2 that beat historical controls, the 745-patient double-blind phase 3 ACT IV found no benefit in 2016, and tumours in both arms had lost EGFRvIII at recurrence, a lesson in antigen escape.","summary":"EGFRvIII peptide conjugated to KLH with GM-CSF. Phase 2 (ACT III) suggested prolonged survival versus historical controls; the double-blind phase 3 ACT IV (n=745, EGFRvIII-positive newly diagnosed glioblastoma with minimal residual disease) showed no OS benefit (20.1 vs 20.0 months). Loss of EGFRvIII expression at recurrence in both arms illustrated antigen escape. Celldex discontinued the programme.","status":"negative","asOf":"2026-09-06","links":[{"label":"ClinicalTrials.gov NCT01480479 (ACT IV)","url":"https://clinicaltrials.gov/study/NCT01480479"}],"tags":[],"related":[],"cancers":["glioblastoma"],"sections":[],"technologies":["shared-antigen-vaccine"],"targets":["egfr"],"drugs":[],"companies":["celldex"],"institutions":[],"pathways":[],"terms":["egfrviii"],"trials":["act-iv"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"CDX-110","modality":"Peptide vaccine (EGFRvIII)","mechanism":"Induces humoral and cellular immunity against the EGFRvIII neoepitope.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rinzimetostat","kind":"drug","name":"Rinzimetostat","aka":[],"tldr":"Rinzimetostat is an experimental small-molecule drug from ORIC Pharmaceuticals in phase 3 trials for prostate cancer, with its target not yet stated publicly.","summary":"Rinzimetostat (ORIC-944) is a small-molecule drug developed by ORIC Pharmaceuticals. The sponsor describes its target as PRC2 (EED subunit), which OnCo does not yet have a target page for. The sponsor states: A potent and selective allosteric inhibitor of Polycomb Repressive Complex 2 (PRC2) acting via the EED subunit, intended to provide advantages over EZH2-targeted approaches. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07723248 (A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1)), in prostate cancer. The largest, NCT07723248, plans to enrol 600 participants with primary completion expected 2028-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Rinzimetostat","url":"https://clinicaltrials.gov/search?intr=ORIC-944"},{"label":"Sponsor pipeline page","url":"https://oricpharma.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["oric-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["himalayas-1"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ORIC-944","modality":"small molecule","mechanism":"A potent and selective allosteric inhibitor of Polycomb Repressive Complex 2 (PRC2) acting via the EED subunit, intended to provide advantages over EZH2-targeted approaches.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ripretinib","kind":"drug","name":"Ripretinib","aka":[],"tldr":"A fourth-line GIST drug that locks KIT in an off state regardless of which resistance mutation the tumour has acquired.","summary":"INVICTUS: PFS 6.3 vs 1.0 months vs placebo (HR 0.15), OS HR 0.36; approved May 2020 after ≥3 TKIs. INTRIGUE (second line vs sunitinib) not superior overall but superior in KIT exon 11 + 17/18 mutations, prompting INSIGHT (ctDNA-selected phase 3, positive on PFS 2025-26 per interim reports). Deciphera/Ono.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Ripretinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ripretinib"}],"tags":[],"related":[],"cancers":["sarcoma","gist-imatinib-resistant"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["kit"],"drugs":[],"companies":["deciphera"],"institutions":[],"pathways":[],"terms":[],"trials":["invictus","insight-gist","nct03673501"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Qinlock","modality":"Small-molecule kinase inhibitor (KIT/PDGFRA switch-control)","mechanism":"Binds the switch pocket and activation loop of KIT/PDGFRA, inhibiting primary and secondary mutations (exons 9, 11, 13, 14, 17, 18).","approvals":[{"region":"US","year":2020,"indication":"Advanced GIST after ≥3 prior kinase inhibitors including imatinib"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"150 mg once daily; 150 mg twice daily on progression","monitoring":"Skin exams (new cutaneous SCC), hypertension, cardiac function, palmar-plantar erythrodysaesthesia"},"toxicity":[{"event":"Alopecia","anyGradePct":52,"note":"INVICTUS"},{"event":"Palmar-plantar erythrodysaesthesia","anyGradePct":21},{"event":"Hypertension","grade3PlusPct":4},{"event":"Cutaneous squamous cell carcinoma","anyGradePct":4.7}],"access":[],"regulatoryEvents":[{"date":"2020-05-15","type":"approval","region":"US","note":"INVICTUS"},{"date":"2022-05","type":"label-change","region":"US","note":"Label updated with INTRIGUE data"}]},{"id":"rituximab","kind":"drug","name":"Rituximab","aka":["Rituximab and Hyaluronidase Human","Rituxan Hycela"],"tldr":"Rituximab was the first antibody ever approved for cancer (1997). It made chemoimmunotherapy the CLL standard for a decade, and biosimilars keep it cheap and everywhere.","summary":"Approved in follicular lymphoma (1997), DLBCL (2006, R-CHOP), CLL (2010, with fludarabine-cyclophosphamide: FCR, the first regimen to show OS benefit in CLL). Superseded in CLL by targeted agents (CLL13, CLL14, ELEVATE-TN) but still used with venetoclax in relapse (MURANO: venetoclax-rituximab 24 months) and in resource-limited settings. Biosimilars (Truxima, Ruxience, Riabni) since 2018.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Rituximab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Rituximab"},{"label":"Hiraga et al., Blood 2009: CD20-negative transformation after rituximab-containing chemotherapy (19 rebiopsied patients)","url":"https://doi.org/10.1182/blood-2008-08-175208"}],"tags":[],"related":["cd20-expression"],"cancers":["cll","dlbcl","marginal-zone-lymphoma","nodular-lymphocyte-predominant-hodgkin-lymphoma"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd20"],"drugs":[],"companies":["roche-genentech","hetero","biogen","halozyme","mabion"],"institutions":[],"pathways":["complement-in-cancer"],"terms":["adcc","lymphoma-bio-lineage-antigen-cost","lymphoma-bio-antigen-escape"],"trials":["nct06097364","nct04442022","nct04529772","nct06717347","nct04384484","nct04002297","nct04712097","nct06149286","nct06508658","nct06191744","nct06911502","nct06230224","nct04224493","nct04680052","nct06091865","nct06091254","nct04404283","nct05100862","nct06363994","nct05888493","nct05409066","nct05371093","nct05952024","nct06601504","nct04663347","nct06090539","nct04542824","nct07189065","nct05951959","nct05533775","nct05201248","nct02180711","nct04844866","nct06119685","nct05283720","nct03671018","nct04669171","nct06564038","nct07029737","nct03930953","nct06943872","nct05947851","nct05624554","nct06084936","nct04075292","nct06846671","nct05023980","nct02970318","nct06082102","nct04965493","nct05406401","nct04623541","nct06890884","nct05615974","nct04494503","nct04980222","nct07015242","nct06425302","nct07520006","nct06970743","nct02972840"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: nothing is tested before the first dose, because CD20 is on essentially every mature B-cell lymphoma, and that is also why the treatment empties the normal B-cell compartment. The two consequences worth naming are hepatitis B reactivation, which is screened for, and low immunoglobulin with a blunted vaccine response, which can persist for years. At relapse the question changes: of 19 rebiopsied patients in one series, 5 had become CD20-negative, with lower CD20 messenger RNA in the negative cells from the same patient (Hiraga 2009)."],"brand":"Rituxan / MabThera (and biosimilars)","modality":"Monoclonal antibody (anti-CD20, chimeric)","mechanism":"Chimeric type I anti-CD20 IgG1: CDC, ADCC, and modest direct signalling.","approvals":[{"region":"US","year":1997,"indication":"Relapsed follicular lymphoma; first antibody approved for cancer"},{"region":"US","year":2010,"indication":"CLL with fludarabine and cyclophosphamide"}],"mechanismSteps":["Binds CD20 and clusters it into lipid rafts","Complement is fixed (CDC) and NK cells recruited (ADCC)","B cells and CLL cells are lysed; normal B cells recover after 6-12 months"],"dosing":{"route":"IV or subcutaneous (Rituxan Hycela)","schedule":"CLL: 375 mg/m² cycle 1 then 500 mg/m² cycles 2-6 with chemotherapy or venetoclax","monitoring":"Infusion reactions, HBV reactivation (boxed), PML (boxed), late-onset neutropenia","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rituxan"},"toxicity":[{"event":"Infusion-related reactions (first infusion)","anyGradePct":77,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Rituxan","note":"Historic lymphoma data; lower with premedication"},{"event":"Hepatitis B reactivation","note":"Boxed warning; screen all patients"},{"event":"Late-onset neutropenia","anyGradePct":8}],"access":[],"regulatoryEvents":[]},{"id":"rivoceranib","kind":"drug","name":"Rivoceranib (apatinib)","aka":["Apatinib"],"tldr":"Apatinib, sold as rivoceranib outside China, was the first pill of its kind approved for stomach cancer and is now the partner of camrelizumab in first-line liver cancer.","summary":"Hengrui's apatinib was approved by the CFDA in October 2014 for advanced gastric or gastro-oesophageal junction adenocarcinoma after two or more lines, the first oral VEGFR2 inhibitor approved for gastric cancer anywhere, and in December 2020 for hepatocellular carcinoma after prior systemic therapy. Combined with camrelizumab it beat sorafenib in first-line HCC (CARES-310, Lancet 2023) and was approved in China in January 2023; the same combination has drawn three complete response letters from the FDA over manufacturing inspections and trial conduct rather than efficacy. Elevar Therapeutics (HLB) holds ex-China rights.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Apatinib","links":[{"label":"Jiangsu Hengrui Pharmaceuticals","url":"https://www.hengrui.com/en/"},{"label":"CARES-310 (Lancet 2023)","url":"https://doi.org/10.1016/S0140-6736(23)00961-3"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":["camrelizumab-rivoceranib","camrelizumab"],"cancers":["gastric","hcc"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":["vegf-angiogenesis"],"terms":[],"trials":["cares-310","nct04639180","nct05320692","nct05934331","nct04342910"],"people":[],"bottlenecks":[],"keyPapers":["paper-qin-lancet"],"journals":[],"dependsOn":[],"notes":[],"brand":"Aitan","code":"apatinib, YN968D1","modality":"Small-molecule VEGFR2 TKI","mechanism":"Selective ATP-competitive inhibitor of VEGFR2 (KDR) with weaker activity on RET, KIT and SRC; blocks tumour angiogenesis.","approvals":[{"region":"China","year":2014,"indication":"Advanced gastric or gastro-oesophageal junction adenocarcinoma after two or more lines"},{"region":"China","year":2020,"indication":"Advanced hepatocellular carcinoma after prior systemic therapy"},{"region":"China","year":2023,"indication":"First-line unresectable hepatocellular carcinoma with camrelizumab (CARES-310)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rnk05047","kind":"drug","name":"RNK05047","aka":[],"tldr":"RNK05047 is a targeted protein degrader from Ranok Therapeutics (Hangzhou) Co., Ltd., in registered phase 2 trials for metastatic cancer.","summary":"RNK05047 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Ranok Therapeutics (Hangzhou) Co., Ltd., in metastatic cancer. A targeted protein degrader, as described in the registry record: it tags a cancer-driving protein for destruction by the cell's own disposal system. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of RNK05047","url":"https://clinicaltrials.gov/search?intr=RNK05047"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct05487170"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"RNK05047","modality":"Targeted protein degrader","mechanism":"A targeted protein degrader, as described in the registry record: it tags a cancer-driving protein for destruction by the cell's own disposal system.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ro7771950","kind":"drug","name":"RO7771950","aka":["ZN-A-1041"],"tldr":"RO7771950 is an experimental small-molecule drug from Hoffmann-La Roche in phase 3 trials for HER2-positive breast cancer and HR-positive / HER2-negative breast cancer, aimed at HER2.","summary":"RO7771950 (A-1041, RG6596) is a small-molecule drug developed by Hoffmann-La Roche. Its target is HER2 (the sponsor names HER2). The sponsor states: A blood-brain-barrier-penetrant HER2-targeted inhibitor, studied orally in combination with trastuzumab and capecitabine in HER2-positive breast cancer, including patients with CNS metastases. ClinicalTrials.gov describes the intervention as: Participants will receive one of two doses of RO7771950 orally (PO) twice a day (BID). It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07413939 (RO7771950 Versus Tucatinib in Combination With Trastuzumab and Capecitabine in People With Locally Advanced or Metastatic Breast Cancer That is Human Epidermal Growth Factor Receptor 2 (HER2)-Positive), in HER2-positive breast cancer and HR-positive / HER2-negative breast cancer. The largest, NCT07413939, plans to enrol 650 participants with primary completion expected 2029-05-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of RO7771950","url":"https://clinicaltrials.gov/search?intr=A-1041"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-her2-positive","breast-hr-positive"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07413939","nct05593094"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"A-1041, RG6596","modality":"small molecule","mechanism":"A blood-brain-barrier-penetrant HER2-targeted inhibitor, studied orally in combination with trastuzumab and capecitabine in HER2-positive breast cancer, including patients with CNS metastases.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rocbrutinib","kind":"drug","name":"Rocbrutinib","aka":[],"tldr":"Rocbrutinib is an experimental small-molecule drug from Guangzhou Lupeng Pharmaceutical in phase 3 trials for mantle cell lymphoma, chronic lymphocytic leukaemia and diffuse large B-cell lymphoma, with its target not yet stated publicly.","summary":"Rocbrutinib (NWP-775, LP-168) is a small-molecule drug developed by Guangzhou Lupeng Pharmaceutical and Newave Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: 200mg qd PO. The treatment will continue until progressive disease, unacceptable toxicity, etc. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07377578 (A Study of Rocbrutinib Versus Investigator's Choice of BTK Inhibitors in Patients With Relapsed or Refractory Mantle Cell Lymphoma) and NCT07342478 (ROCKET-CLL Global Phase 3 Study: Rocbrutinib vs Pirtobrutinib in cBTKi-Pretreated R/R CLL/SLL), in mantle cell lymphoma, chronic lymphocytic leukaemia and diffuse large B-cell lymphoma. The largest, NCT07377578, plans to enrol 394 participants with primary completion expected 2031-05-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Rocbrutinib","url":"https://clinicaltrials.gov/search?intr=NWP-775"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["mantle-cell-lymphoma","cll","dlbcl"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07377578","nct07342478","nct07189065","nct05716087","nct07609862"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"NWP-775, LP-168","modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"roginolisib","kind":"drug","name":"Roginolisib","aka":["IOA-244"],"tldr":"Roginolisib is an oral pi3k inhibitor from iOnctura, in registered phase 2 trials for non-small-cell lung cancer, small-cell lung cancer.","summary":"Roginolisib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by iOnctura, in non-small-cell lung cancer, small-cell lung cancer. A phosphoinositide 3-kinase inhibitor: the name stem -lisib marks a small molecule that blocks PI3K signalling. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of roginolisib","url":"https://clinicaltrials.gov/search?intr=roginolisib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc","sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["ionctura"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06879717","nct06717126"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral PI3K inhibitor","mechanism":"A phosphoinositide 3-kinase inhibitor: the name stem -lisib marks a small molecule that blocks PI3K signalling.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rolapitant","kind":"drug","name":"Rolapitant","aka":["Varuby"],"tldr":"Rolapitant (Varubi) is a long-lasting anti-sickness tablet taken once before chemotherapy, alongside a 5-HT3 blocker and dexamethasone, to prevent nausea and vomiting in the days after treatment.","summary":"Rolapitant was approved by the FDA in September 2015 in combination with other antiemetics for prevention of delayed nausea and vomiting with initial and repeat courses of emetogenic chemotherapy including highly emetogenic regimens, on three phase 3 trials (two in cisplatin-based and one in moderately emetogenic chemotherapy) in which complete response in the delayed phase was higher than with granisetron and dexamethasone alone, a statistically significant difference. An intravenous emulsion approved in 2017 was withdrawn after anaphylaxis reports. Its long half-life and lack of CYP3A4 inhibition avoid the dexamethasone dose adjustment required with aprepitant, but it inhibits CYP2D6 and BCRP, so thioridazine is contraindicated and irinotecan and methotrexate exposure may rise. Marketed in the US by TerSera.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Rolapitant","links":[{"label":"Study 3 (Lancet Oncology 2015)","url":"https://doi.org/10.1016/S1470-2045(15)00034-0"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=8670c3cf-82d9-466d-be49-de33d8c8742b"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/rolapitanthydrochloride"}],"tags":["nci-list","supportive"],"related":["aprepitant","netupitant-palonosetron"],"cancers":[],"sections":[],"technologies":["antiemetic-therapy"],"targets":[],"drugs":[],"companies":["tersera-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["ts-p04834"],"people":[],"bottlenecks":[],"keyPapers":["paper-rolapitant-study-3-schwartzberg-lancet-oncol-2015"],"journals":[],"dependsOn":[],"notes":[],"brand":"Varubi","modality":"Neurokinin-1 receptor antagonist (antiemetic)","supportive":true,"mechanism":"Selective, competitive substance P/NK1 receptor antagonist with a half-life of about seven days, so one oral dose covers the delayed phase; unlike aprepitant it does not inhibit CYP3A4.","approvals":[{"region":"US","year":2015,"indication":"Prevention of delayed nausea and vomiting with emetogenic chemotherapy, in combination with other antiemetics"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"romidepsin","kind":"drug","name":"Romidepsin","aka":[],"tldr":"Romidepsin is an epigenetic drug for T-cell lymphomas of the skin and lymph nodes; its US lymph-node indication was withdrawn when a confirmatory trial failed.","summary":"Romidepsin is a cyclic peptide inhibitor of class I histone deacetylases 1 and 2; it induces histone hyperacetylation, cell-cycle arrest and apoptosis, and is particularly active in T-cell lymphomas of follicular helper type that carry TET2 or DNMT3A mutations. It was approved in the US in 2009 for cutaneous T-cell lymphoma after at least one systemic therapy and in 2011, under accelerated approval, for peripheral T-cell lymphoma. The Ro-CHOP trial (2021) failed to improve progression-free survival when romidepsin was added to CHOP, and the PTCL indication was voluntarily withdrawn in 2021. The CTCL indication remains, and use with azacitidine in TFH lymphoma is an active area. Nausea, fatigue, cytopenias and QT prolongation are the main toxicities. Romidepsin is an epigenetic drug that works for skin T-cell lymphoma but could not prove itself in a randomised trial in nodal disease.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Romidepsin","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=romidepsin"}],"tags":["gap-fill"],"related":[],"cancers":["peripheral-t-cell-lymphoma","cutaneous-t-cell-lymphoma"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04747236","nct00106431","nct00112463"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Istodax","modality":"Class I HDAC inhibitor (cyclic peptide)","mechanism":"Inhibits histone deacetylases 1/2, inducing histone hyperacetylation, cell-cycle arrest and apoptosis; activity in T-cell lymphomas including TFH type with TET2/DNMT3A mutations.","approvals":[{"region":"US","year":2009,"indication":"Cutaneous T-cell lymphoma after ≥1 systemic therapy"},{"region":"US","year":2011,"indication":"Peripheral T-cell lymphoma after ≥1 therapy","note":"Withdrawn 2021 after Ro-CHOP"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2011-06-16","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Peripheral T-cell lymphoma in patients who have received at least one prior therapy"},{"date":"2020-03-13","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Formulation: Treatment of peripheral T-cell lymphoma in adults who have received at least one prior therapy"},{"date":"2021-07-30","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 10.1 years after its accelerated approval.","indication":"Peripheral T-cell lymphoma in patients who have received at least one prior therapy"},{"date":"2021-12-08","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 1.7 years after its accelerated approval.","indication":"Formulation: Treatment of peripheral T-cell lymphoma in adults who have received at least one prior therapy"}]},{"id":"romiplostim","kind":"drug","name":"Romiplostim","aka":["AMG 531"],"tldr":"Romiplostim is a weekly injection that tells the bone marrow to make more platelets; it is approved for immune thrombocytopenia and for radiation injury, and is being tested for the low platelets that chemotherapy causes.","summary":"Romiplostim is an engineered peptibody that mimics thrombopoietin. The United States approved it in 2008 for adults with immune thrombocytopenia (ITP) who had not responded to steroids, immunoglobulins or splenectomy, later extended to children and, separately, to increase survival after acute exposure to myelosuppressive doses of radiation (haematopoietic syndrome of acute radiation syndrome). It is not approved for chemotherapy-induced thrombocytopenia, the commonest reason platelets fall in cancer care, but randomised trials in that setting are under way and haematologists use it off label when platelet counts would otherwise delay treatment.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Romiplostim","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=romiplostim"},{"label":"Drugs@FDA BLA 125268","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=125268"}],"tags":["gap-fill","supportive-care"],"related":[],"cancers":["aml","mds"],"sections":[],"technologies":["supportive-care"],"targets":["mpl"],"drugs":["eltrombopag"],"companies":["amgen"],"institutions":[],"pathways":[],"terms":["thrombocytopenia"],"trials":["nct03937154"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Nplate","modality":"Thrombopoietin receptor agonist (peptibody), weekly injection","supportive":true,"mechanism":"A peptide fused to an antibody Fc fragment that binds and activates the thrombopoietin receptor MPL, increasing platelet production by megakaryocytes.","approvals":[{"region":"US","year":2008,"indication":"Immune thrombocytopenia with insufficient response to corticosteroids, immunoglobulins or splenectomy","note":"Later extended to children and to the haematopoietic syndrome of acute radiation syndrome"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rondecabtagene-autoleucel","kind":"drug","name":"Rondecabtagene autoleucel","aka":[],"tldr":"Rondecabtagene autoleucel is an experimental CAR-T cell therapy from Lyell Immunopharma in phase 3 trials for diffuse large B-cell lymphoma and hodgkin lymphoma, aimed at CD19 and CD20.","summary":"Rondecabtagene autoleucel (LYL314) is a CAR-T cell therapy developed by Lyell Immunopharma. Its targets are CD19 and CD20 (the sponsor names CD19/CD20). The sponsor states: An autologous, dual-targeting CD19/CD20 CAR T-cell product using an OR-gated CAR construct that fully activates on recognition of either antigen, enriched for CD62L-positive naive/central memory T cells for enhanced proliferation and persistence. ClinicalTrials.gov describes the intervention as: An autologous, dual-targeting CD19/20 CAR T-cell candidate. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07188558 (A Study to Investigate Ronde-cel Versus Investigator's Choice CD19 CAR T-Cell Therapy), in diffuse large B-cell lymphoma and hodgkin lymphoma. The largest, NCT07188558, plans to enrol 400 participants with primary completion expected 2029-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Rondecabtagene autoleucel","url":"https://clinicaltrials.gov/search?intr=LYL314"},{"label":"Sponsor pipeline page","url":"http://www.lyell.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl","hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":["cd19","cd20"],"drugs":[],"companies":["lyell-immunopharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07188558","nct05826535"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"LYL314","modality":"CAR-T cell therapy","mechanism":"An autologous, dual-targeting CD19/CD20 CAR T-cell product using an OR-gated CAR construct that fully activates on recognition of either antigen, enriched for CD62L-positive naive/central memory T cells for enhanced proliferation and persistence.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ropeginterferon-alfa-2b","kind":"drug","name":"Ropeginterferon alfa-2b","aka":[],"tldr":"Ropeginterferon alfa-2b is a long-acting interferon for polycythaemia vera that, unlike hydroxyurea, can shrink the mutant clone over years.","summary":"Ropeginterferon alfa-2b is a mono-pegylated, long-acting interferon alfa given every two weeks. Type I interferon signalling preferentially suppresses the JAK2-mutant clone in polycythaemia vera, with antiproliferative and immunomodulatory effects that hydroxyurea does not share. In PROUD-PV and its CONTINUATION-PV extension, complete haematological response and JAK2 allele-burden reduction were superior to hydroxyurea by 3 to 6 years, and Low-PV showed benefit in low-risk patients who would otherwise receive phlebotomy alone. It was the first interferon approved specifically for PV, in the EU in 2019 and the US in 2021. Depression, autoimmunity and flu-like symptoms need monitoring, and whether clone reduction means fewer transformations is still being followed. In short, this is the PV drug that can shrink the mutant clone over years rather than just control blood counts.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Ropeginterferon_alfa-2b","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ropeginterferon"}],"tags":["gap-fill"],"related":[],"cancers":["myeloproliferative-neoplasms","polycythaemia-vera","essential-thrombocythaemia"],"sections":[],"technologies":["cytokine-therapy"],"targets":["jak2"],"drugs":[],"companies":["aop-health"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06468033","nct05481151","nct05485948","nct05482971","nct04285086","nct04655092"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Besremi","modality":"Long-acting mono-pegylated interferon alfa","mechanism":"Type I interferon signalling suppresses the JAK2-mutant clone preferentially, with immunomodulatory and antiproliferative effects; every-2-week dosing.","approvals":[{"region":"EU","year":2019,"indication":"Polycythaemia vera without symptomatic splenomegaly"},{"region":"US","year":2021,"indication":"Polycythaemia vera"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rovalpituzumab-tesirine","kind":"drug","name":"Rovalpituzumab tesirine","aka":[],"tldr":"Rovalpituzumab tesirine (Rova-T) was the first drug against DLL3 in small-cell lung cancer. AbbVie bought it for $5.8B and abandoned it after two failed phase 3 trials. The target later worked with a different weapon.","summary":"A DLL3-directed ADC with a PBD dimer payload (DAR ~2). Phase 2 TRINITY showed modest response rates with high toxicity (effusions, oedema, photosensitivity). Phase 3 TAHOE (second line vs topotecan) was stopped for shorter survival in the Rova-T arm; MERU (first-line maintenance) failed. AbbVie ended development in 2019 after the 2016 Stemcentrx acquisition. Tarlatamab, a DLL3×CD3 T-cell engager, later improved survival in the same setting (DeLLphi-304, 2025).\n\nLesson: target validation and modality are separable. DLL3 was the right address; a PBD payload with a narrow window in a frail population was the wrong weapon.","status":"withdrawn","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Rovalpituzumab_tesirine","links":[{"label":"TAHOE phase 3 (J Thorac Oncol 2021)","url":"https://www.jto.org/article/S1556-0864(21)02264-5/fulltext"}],"tags":["failure","lesson:wrong-drug"],"related":[],"cancers":["sclc"],"sections":[],"technologies":["adc"],"targets":["dll3"],"drugs":["tarlatamab"],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03033511","nct03061812","nct02674568"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"Rova-T, SC16LD6.5","modality":"ADC","payload":"PBD dimer (SC-DR002)","linker":"Cleavable dipeptide","mechanism":"Humanised anti-DLL3 with PBD dimer (SC-DR002) via cleavable linker.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rph-051","kind":"drug","name":"RPH-051","aka":[],"tldr":"RPH-051 is an experimental monoclonal antibody from R-Pharm in phase 3 trials for HR-positive / HER2-negative breast cancer and HER2-positive breast cancer, aimed at HER2.","summary":"RPH-051 is a monoclonal antibody developed by R-Pharm. Its target is HER2 (the sponsor names HER2). The sponsor states: A pertuzumab biosimilar candidate, a HER2-targeted monoclonal antibody given with trastuzumab and docetaxel, being tested for non-inferiority and comparability of safety/immunogenicity against reference product Perjeta. ClinicalTrials.gov describes the intervention as: RPH-051: concentrate for solution for infusion, 30 mg/mL 14 mL of the liquid concentrate is diluted with 250 mL of 0.9% sodium chloride solution. The nominal concentration of the prepared solution is 3.0 mg/mL for the loading dose and 1.6 m. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07386938 (A Study of the Efficacy, Safety and Pharmacokinetics of RPH-051 and Perjeta® in Combination With Trastuzumab and Docetaxel as the 1st Line Therapy in Patients With HER2-positive Breast Cancer), in HR-positive / HER2-negative breast cancer and HER2-positive breast cancer. The largest, NCT07386938, plans to enrol 246 participants with primary completion was scheduled for 2025-10-15 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of RPH-051","url":"https://clinicaltrials.gov/search?intr=RPH-051"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","breast-her2-positive"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07386938"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"A pertuzumab biosimilar candidate, a HER2-targeted monoclonal antibody given with trastuzumab and docetaxel, being tested for non-inferiority and comparability of safety/immunogenicity against reference product Perjeta.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rucaparib","kind":"drug","name":"Rucaparib","aka":[],"tldr":"A PARP inhibitor for BRCA-mutated ovarian and prostate cancer whose original maker went bankrupt; still available, with a narrowed label.","summary":"Approved 2016 (BRCA-mutated ovarian cancer after ≥2 chemotherapies; later restricted after OS concerns in ARIEL4), 2018 as recurrent-disease maintenance (ARIEL3; later restricted to BRCA-mutated), and 2020 for BRCA-mutated mCRPC (TRITON2). ATHENA-MONO showed first-line maintenance PFS benefit in all comers. Clovis Oncology filed for bankruptcy in December 2022; pharma& now markets it.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Rucaparib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Rucaparib"},{"label":"TRITON3 (New England Journal of Medicine 2023)","url":"https://doi.org/10.1056/NEJMoa2214676"},{"label":"NICE TA887: olaparib for previously treated BRCA mutation-positive hormone-relapsed metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta887"}],"tags":[],"related":["brca-somatic"],"cancers":["ovarian","prostate","high-grade-serous-ovarian-cancer","platinum-sensitive-ovarian-cancer","pancreatic","brca-palb2-pdac"],"sections":[],"technologies":["parp-inhibitor"],"targets":["parp","brca"],"drugs":[],"companies":["pharmaand"],"institutions":[],"pathways":[],"terms":["hrd","prostate-hrr-eligibility"],"trials":["athena-mono"],"people":[],"bottlenecks":[],"keyPapers":["paper-reiss-rucaparib-maintenance-brca-palb2-pancreatic-jco-2021"],"journals":[],"dependsOn":[],"notes":["Pancreatic ductal adenocarcinoma: a single-arm phase 2 of maintenance rucaparib in platinum-sensitive patients with germline or somatic BRCA1, BRCA2 or PALB2 variants gave 6-month progression-free survival of 59.5% and a 41.7% response rate, including responses in germline PALB2 and somatic BRCA2; there is no pancreatic approval (Reiss 2021).","TRITON3 is the trial that defines who rucaparib helps in prostate cancer, and it is a gene-specific answer: imaging-based progression-free survival 11.2 against 6.4 months in the BRCA subgroup (hazard ratio 0.50, 95 percent confidence interval 0.36 to 0.69) and 8.1 against 6.8 months in the ATM subgroup (0.95, 0.59 to 1.52). There is no NICE appraisal of rucaparib in prostate cancer; in England the PARP inhibitor route for BRCA-mutated hormone-relapsed disease is olaparib under TA887."],"brand":"Rubraca","modality":"Small-molecule PARP inhibitor","mechanism":"PARP1/2/3 inhibitor and trapper; synthetic lethality with homologous recombination deficiency.","approvals":[{"region":"US","year":2016,"indication":"BRCA-mutated advanced ovarian cancer after ≥2 chemotherapies (later narrowed)"},{"region":"US","year":2018,"indication":"Maintenance in recurrent ovarian cancer after platinum response (now BRCA-mutated only)"},{"region":"US","year":2020,"indication":"BRCA-mutated mCRPC after ARPI and taxane"}],"mechanismSteps":["Enters the cell and binds PARP1 at single-strand DNA breaks","Traps PARP on DNA, blocking base-excision repair","Replication forks collapse into double-strand breaks","BRCA-deficient cells cannot repair them and die"],"dosing":{"route":"Oral","schedule":"600 mg twice daily continuously","modifications":"Dose reductions to 500, 400, 300 mg for anaemia, nausea, ALT rise","monitoring":"CBC monthly; creatinine rises are usually benign (transporter inhibition)"},"toxicity":[{"event":"Nausea","anyGradePct":75,"grade3PlusPct":4,"note":"ARIEL3"},{"event":"Anaemia","anyGradePct":37,"grade3PlusPct":19},{"event":"Fatigue","anyGradePct":69,"grade3PlusPct":7}],"access":[],"regulatoryEvents":[{"date":"2016-12-19","type":"accelerated-approval","region":"US","note":"Accelerated approval, gBRCA/sBRCA ovarian, ≥2 prior lines","indication":"Deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer treated with 2 or more chemotherapies 2"},{"date":"2018-04-06","type":"conversion","region":"US","note":"Recurrent ovarian maintenance (ARIEL3)","indication":"Deleterious BRCA mutation (germline and/or somatic) associated advanced ovarian cancer treated with 2 or more chemotherapies 2"},{"date":"2020-05-15","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-retifanlimab-dlwr-metastatic-or-recurrent-locally-advanced-merkel","indication":"Treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy"},{"date":"2022-06","type":"label-change","region":"US","note":"Later-line treatment indication withdrawn after ARIEL4 OS imbalance"},{"date":"2022-12-11","type":"withdrawal","region":"US","note":"Clovis Oncology bankruptcy; asset sold to pharma&"},{"date":"2025-12-17","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 5.6 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-retifanlimab-dlwr-metastatic-or-recurrent-locally-advanced-merkel","indication":"Treatment of adult patients with a deleterious BRCA mutation (germline and/or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor-directed therapy and a taxane-based chemotherapy"}]},{"id":"rusfertide","kind":"drug","name":"Rusfertide","aka":[],"tldr":"Rusfertide is the first drug to control polycythaemia vera's excess red cells by restricting iron, sparing patients repeated blood removal.","summary":"Rusfertide is an injectable hepcidin mimetic peptide that induces degradation of ferroportin, restricting iron supply to red-cell production and so holding haematocrit in range without repeated phlebotomy. It is intended for polycythaemia vera patients whose erythrocytosis persists despite standard therapy and who would otherwise depend on blood removal. In the VERIFY phase 3 trial (2025), significantly more patients on rusfertide plus standard therapy were phlebotomy-free versus placebo, with improved symptoms. The FDA approved it in August 2026 for erythrocytosis in adults with PV, the first drug to work through iron restriction in this disease; it was developed by Protagonist with Takeda. Long-term safety and whether this route reduces thrombosis as phlebotomy does remain open. In short, rusfertide treats the excess red cells by starving them of iron rather than draining them.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Rusfertide","links":[{"label":"FDA novel approvals 2026","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2026"}],"tags":["gap-fill"],"related":[],"cancers":["myeloproliferative-neoplasms","polycythaemia-vera"],"sections":[],"technologies":["peptide-drug-conjugate"],"targets":[],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["verify","nct07648030","nct06033586"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mimrylo","modality":"Hepcidin mimetic peptide","mechanism":"Injectable hepcidin mimetic that degrades ferroportin, restricting iron availability to erythropoiesis and controlling haematocrit without phlebotomy.","approvals":[{"region":"US","year":2026,"indication":"Erythrocytosis in adults with polycythaemia vera"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ruxolitinib","kind":"drug","name":"Ruxolitinib","aka":[],"tldr":"Ruxolitinib was the first JAK inhibitor: it shrinks the spleen and relieves symptoms in myelofibrosis and controls blood counts in polycythaemia vera, without eliminating the disease clone.","summary":"COMFORT-I/II (2011) showed spleen volume and symptom benefit versus placebo/best available therapy with a later-demonstrated survival advantage; RESPONSE (2014) established it in hydroxyurea-resistant PV. Also approved in acute and chronic graft-versus-host disease (REACH2/3). Anaemia, thrombocytopenia, infections and a discontinuation syndrome are the main issues.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Ruxolitinib","links":[{"label":"COMFORT-I (NEJM 2012)","url":"https://doi.org/10.1056/NEJMoa1110557"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ruxolitinib"}],"tags":["gap-fill"],"related":[],"cancers":["myeloproliferative-neoplasms","polycythaemia-vera","primary-myelofibrosis","all-ph-like"],"sections":[],"technologies":["kinase-inhibitors","allogeneic-hsct"],"targets":["jak2","jak1"],"drugs":[],"companies":["incyte","novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02955940","nct04562389","nct04509700","nct04817007","nct03669965"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Jakafi / Jakavi","modality":"Small-molecule JAK1/JAK2 inhibitor","mechanism":"ATP-competitive inhibition of JAK1 and JAK2, dampening JAK-STAT signalling regardless of JAK2/CALR/MPL driver.","approvals":[{"region":"US","year":2011,"indication":"Intermediate/high-risk myelofibrosis"},{"region":"US","year":2014,"indication":"Polycythaemia vera after hydroxyurea"},{"region":"US","year":2019,"indication":"Steroid-refractory acute GVHD"},{"region":"US","year":2021,"indication":"Chronic GVHD after failure of 1-2 lines"},{"region":"EU","year":2012,"indication":"Myelofibrosis"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rv001v","kind":"drug","name":"RV001V","aka":[],"tldr":"RV001V is a cancer vaccine from RhoVac APS, in registered phase 2 trials for prostate cancer.","summary":"RV001V is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by RhoVac APS, in prostate cancer. A cancer vaccine, as described in the registry record: it trains the immune system against tumour antigens. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of RV001V","url":"https://clinicaltrials.gov/search?intr=RV001V"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["prostate"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["rhovac"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04114825"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"RV001V","modality":"Cancer vaccine","mechanism":"A cancer vaccine, as described in the registry record: it trains the immune system against tumour antigens.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"rvu120","kind":"drug","name":"RVU120","aka":["SEL120"],"tldr":"RVU120 is a small-molecule inhibitor from Ryvu Therapeutics SA, in registered phase 2 trials for acute myeloid leukaemia.","summary":"RVU120 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Ryvu Therapeutics SA, in acute myeloid leukaemia. Described in the registry record as a selective inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of RVU120","url":"https://clinicaltrials.gov/search?intr=RVU120"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["aml"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["ryvu-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06191263"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"RVU120","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a selective inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sabatolimab","kind":"drug","name":"Sabatolimab","aka":["MBG453"],"tldr":"Sabatolimab is a monoclonal antibody from Novartis Pharmaceuticals, in registered phase 2 trials for myelodysplastic syndromes / neoplasms.","summary":"Sabatolimab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Novartis Pharmaceuticals, in myelodysplastic syndromes / neoplasms. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Sabatolimab","url":"https://clinicaltrials.gov/search?intr=Sabatolimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["mds","mds-higher-risk"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05201066"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sacituzumab-govitecan","kind":"drug","name":"Sacituzumab govitecan","aka":[],"tldr":"The first TROP2-targeted ADC. It delivers a strong chemotherapy directly to breast and bladder cancer cells and is now a first-line option in triple-negative breast cancer.","summary":"Approved 2020 (accelerated) and 2021 (full) for pretreated metastatic TNBC (ASCENT: OS 12.1 vs 6.7 months), 2023 for HR+/HER2- breast cancer (TROPiCS-02), and urothelial cancer (later withdrawn in the US after TROPiCS-04). In 2026 the FDA approved it in first-line metastatic TNBC as monotherapy for patients not eligible for PD-1 inhibitors (ASCENT-03) and in combination with pembrolizumab for PD-L1-positive disease (ASCENT-04). Hydrolysable linker releases SN-38 in the tumour microenvironment, giving bystander killing. Neutropenia and diarrhoea are the key toxicities; UGT1A1*28 homozygotes are at higher risk.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Sacituzumab_govitecan","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Sacituzumab%20govitecan"},{"label":"NICE TA819: sacituzumab govitecan for unresectable triple-negative advanced breast cancer after 2 or more therapies (17 August 2022)","url":"https://www.nice.org.uk/guidance/ta819"},{"label":"Trodelvy label (openFDA): first-line and later-line TNBC indications, ASCENT-03 study section","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22TRODELVY%22"},{"label":"EMA Trodelvy product page: EU therapeutic indications","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/trodelvy"}],"tags":[],"related":["pd-l1-cps"],"cancers":["tnbc","breast-hr-positive","urothelial","tnbc-metastatic"],"sections":[],"technologies":["adc","topoisomerase-inhibitors"],"targets":["trop2"],"drugs":[],"companies":["gilead"],"institutions":[],"pathways":[],"terms":["bystander-effect","payload","cl2a"],"trials":["ascent","ascent-03","ascent-04","nct05089734","nct06801834","nct06486441","nct05633667","nct05186974","nct06926920","nct03547973","nct05840211","nct05119907","nct05609968","nct05101096","nct04454437","nct03964727","nct04639986","nct05327530","nct06827613","diamond"],"people":[],"bottlenecks":[],"keyPapers":["paper-bardia-ascent-trop2-biomarker-ann-oncol-2021","paper-keynote-355-nejm-2022"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer biomarkers: no TROP2 test is required; benefit in ASCENT was seen at high and medium Trop-2 H-score and regardless of germline BRCA (Bardia 2021). The first-line combination with pembrolizumab (ASCENT-04) is gated by 22C3 CPS 10 or more, the KEYNOTE-355 threshold (Cortes 2022); monotherapy first line (ASCENT-03) is for patients not candidates for PD-1 inhibitors.","Triple-negative breast cancer, UK status: NICE TA819 covers third line and beyond. The first-line PD-L1-negative indication (ASCENT-03) is a NICE appraisal awaiting development (GID-TA11489, expected publication to be confirmed); no NICE appraisal of sacituzumab govitecan with pembrolizumab (ASCENT-04) was listed on 24 September 2026. The UK ASCENT sites were Colchester, Coventry, Durham, Guildford, Hereford, the Royal Free, the Christie, Nottingham, Plymouth, Taunton and Wakefield.","Label toxicity, first line with pembrolizumab (ASCENT-04, 221 patients): decreased neutrophil count and haemoglobin 86 percent each, diarrhoea 72 percent, nausea 68 percent, alopecia 52 percent (Trodelvy label, adverse reactions)."],"brand":"Trodelvy","code":"IMMU-132","modality":"ADC","payload":"SN-38 (topoisomerase-I inhibitor), DAR ~7.6","linker":"CL2A, pH-sensitive cleavable","mechanism":"Humanised anti-TROP2 IgG1 (hRS7) internalised; SN-38 released by linker hydrolysis inside and around tumour cells.","approvals":[{"region":"US","year":2020,"indication":"Metastatic TNBC, ≥2 prior lines (accelerated; full 2021)"},{"region":"US","year":2023,"indication":"HR+/HER2- metastatic breast cancer after endocrine therapy and ≥2 chemotherapies"},{"region":"US","year":2026,"indication":"First-line metastatic TNBC: monotherapy (PD-1 ineligible) or with pembrolizumab (PD-L1 CPS ≥10)"},{"region":"EU","year":2021,"indication":"mTNBC ≥2 lines; HR+ 2023"},{"region":"EU","year":2021,"indication":"Unresectable or metastatic TNBC after two or more systemic therapies, at least one for advanced disease (ASCENT); marketing authorisation issued 22 November 2021","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/trodelvy"},{"region":"EU","year":2026,"indication":"First-line unresectable locally advanced or metastatic TNBC in patients who are not candidates for PD-1 or PD-L1 inhibitor therapy (ASCENT-03); listed in the EMA therapeutic indications read on 24 September 2026","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/trodelvy"},{"region":"UK","year":2022,"indication":"Unresectable triple-negative locally advanced or metastatic breast cancer after two or more systemic therapies, at least one for advanced disease; NICE TA819 (17 August 2022) recommends within the marketing authorisation with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta819"}],"mechanismSteps":["Antibody binds TROP2 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","SN-38 is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"10 mg/kg on days 1 and 8 of each 21-day cycle","modifications":"Hold and reduce for grade 3-4 neutropenia or diarrhoea; G-CSF prophylaxis permitted","monitoring":"Blood counts each dose; UGT1A1*28 homozygotes have earlier and more frequent neutropenia; anti-emetic premedication","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d"},"toxicity":[{"event":"Neutropenia","anyGradePct":78,"grade3PlusPct":49,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Anaemia","anyGradePct":94,"grade3PlusPct":9,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Fatigue","anyGradePct":65,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Diarrhoea","anyGradePct":59,"grade3PlusPct":11,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Nausea","anyGradePct":57,"grade3PlusPct":3.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Alopecia","anyGradePct":47,"grade3PlusPct":0,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Constipation","anyGradePct":37,"grade3PlusPct":0.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Vomiting","anyGradePct":33,"grade3PlusPct":1.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Decreased appetite","anyGradePct":28,"grade3PlusPct":1.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Rash","anyGradePct":12,"grade3PlusPct":0.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=57a597d2-03f0-472e-b148-016d7169169d","note":"ASCENT, Trodelvy arm, n=258"},{"event":"Neutropenia (grade 3 or higher)","grade3PlusPct":51,"source":"https://doi.org/10.1056/NEJMoa2028485","note":"ASCENT, sacituzumab arm; 33 percent with chemotherapy"},{"event":"Diarrhoea (grade 3 or higher)","grade3PlusPct":10,"source":"https://doi.org/10.1056/NEJMoa2028485","note":"ASCENT; below 1 percent with chemotherapy"},{"event":"Febrile neutropenia (grade 3 or higher)","grade3PlusPct":6,"source":"https://doi.org/10.1056/NEJMoa2028485","note":"ASCENT; 2 percent with chemotherapy"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.gileadadvancingaccess.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for pretreated metastatic TNBC (TA819) and HR+/HER2- breast cancer after endocrine therapy and chemotherapy","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"},{"country":"EU","reimbursement":"EMA approved; national reimbursement varies","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2016-02-05","type":"designation","region":"US","note":"Breakthrough Therapy designation for pretreated metastatic TNBC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-04-22","type":"accelerated-approval","region":"US","note":"Accelerated approval, metastatic TNBC after ≥2 prior therapies","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adults with metastatic TNBC following at least 2 prior therapies for metastatic disease"},{"date":"2021-04-07","type":"conversion","region":"US","note":"Full approval in metastatic TNBC (ASCENT); accelerated approval in urothelial cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adults with metastatic TNBC following at least 2 prior therapies for metastatic disease"},{"date":"2021-04-13","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sacituzumab-govitecan-advanced-urothelial-cancer","indication":"Treatment of adult patients with locally advanced or metastatic urothelial cancer (mUC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor"},{"date":"2021-11-22","type":"approval","region":"EU","note":"EMA approval, pretreated metastatic TNBC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-02-03","type":"approval","region":"US","note":"HR+/HER2- metastatic breast cancer after endocrine therapy and ≥2 chemotherapies (TROPiCS-02)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-11","type":"withdrawal","region":"US","note":"Urothelial cancer indication voluntarily withdrawn after TROPiCS-04","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-11-22","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 3.6 years after its accelerated approval.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sacituzumab-govitecan-advanced-urothelial-cancer","indication":"Treatment of adult patients with locally advanced or metastatic urothelial cancer (mUC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor"},{"date":"2026-06-24","type":"approval","region":"US","note":"First-line metastatic TNBC: monotherapy (PD-1 ineligible, ASCENT-03) and with pembrolizumab (CPS ≥10, ASCENT-04)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-sacituzumab-govitecan-hziy-monotherapy-and-combination-pembrolizumab-first-line"}]},{"id":"sacituzumab-tirumotecan","kind":"drug","name":"Sacituzumab tirumotecan","aka":[],"tldr":"Sacituzumab tirumotecan is Kelun-Biotech's TROP2 ADC, approved in China in 2024 for pretreated triple-negative breast cancer and then EGFR-mutant lung cancer. Merck holds rights outside Greater China and runs the TroFuse programme of more than ten phase 3 trials across breast, lung, endometrial and cervical cancer; in the US it holds a priority voucher but no approval yet.","summary":"Kelun-Biotech's sac-TMT was approved by China's NMPA in 2024 for pretreated TNBC (the first TROP2 ADC approval in China) and later for EGFR-mutant NSCLC. Merck holds ex-Greater-China rights and runs the TroFuse programme of >10 phase 3 trials, including first-line TNBC (positive on primary endpoint per 2026 reports), HR+ breast, NSCLC, endometrial, and cervical cancer, many combined with pembrolizumab. FDA Breakthrough Therapy designation (EGFR-mutant NSCLC, 2024) and a Commissioner's National Priority Voucher (July 2026).","status":"approved","asOf":"2026-09-04","links":[{"label":"Frontiers review (2026)","url":"https://www.frontiersin.org/journals/oncology-reviews/articles/10.3389/or.2026.1781533/full"}],"tags":[],"related":[],"cancers":["tnbc","nsclc","breast-hr-positive","endometrial","cervical"],"sections":[],"technologies":["adc","topoisomerase-inhibitors"],"targets":["trop2"],"drugs":[],"companies":["kelun-biotech","merck"],"institutions":[],"pathways":[],"terms":["t030","hydrophilic-next-gen"],"trials":["nct06670196","nct06305754","nct06448312","nct07216703","nct07318558","nct06824467","nct06356311","nct06170788","nct06459180","nct06422143","nct06711900","nct06312137","nct06952504","nct06132958","nct05870319","nct06074588","nct04152499","nct05351788","nct05816252","nct07329322","nct06428409","nct06483334","nct06393374","nct06469944","nct07252739","nct06312176","nct07419295","nct06966700","nct07286149","nct05319730","nct06637423","nct07324629","nct06780111","nct06445972","nct06788912","nct06841354"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"sac-TMT, MK-2870, SKB264","modality":"ADC","payload":"T030 (belotecan-derived TOP1 inhibitor), DAR ~7.4","linker":"Sulfonyl pyrimidine (CL2A-like), pH-sensitive and enzyme-cleavable","mechanism":"Anti-TROP2 IgG1 with a stable, irreversibly-conjugated linker and a belotecan-derived TOP1 payload with reported lower efflux-pump susceptibility.","approvals":[{"region":"China","year":2024,"indication":"Pretreated advanced TNBC; later EGFR-mutant NSCLC after TKI"}],"mechanismSteps":["Antibody binds TROP2 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","T030 (belotecan-derived TOP1 inhibitor) is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"5 mg/kg every 2 weeks (China label, TNBC); phase 3 trials use 4 mg/kg every 2 weeks in combination regimens","monitoring":"Blood counts; stomatitis and ocular surface care","source":"https://www.frontiersin.org/journals/oncology-reviews/articles/10.3389/or.2026.1781533/full"},"toxicity":[{"event":"Neutropenia","note":"Most common grade ≥3 event in OptiTROP-Breast01; rates per publication"},{"event":"Anaemia"},{"event":"Stomatitis"},{"event":"Nausea"},{"event":"Alopecia"}],"access":[{"country":"CN","reimbursement":"NMPA approved 2024; National Reimbursement Drug List inclusion from 2025","asOf":"2026-09-06"},{"country":"US","reimbursement":"Investigational; not yet approved","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2022-05","type":"filing","region":"China","note":"Merck licenses ex-Greater-China rights (deal expanded December 2022)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-11","type":"approval","region":"China","note":"NMPA approval, pretreated advanced TNBC (OptiTROP-Breast01); first TROP2 ADC approved in China","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-12-03","type":"designation","region":"US","note":"Breakthrough Therapy designation, EGFR-mutant NSCLC after TKI","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-03","type":"approval","region":"China","note":"EGFR-mutant NSCLC after TKI failure","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-07","type":"designation","region":"US","note":"Commissioner's National Priority Voucher (CNPV) awarded","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"safusidenib","kind":"drug","name":"Safusidenib","aka":["DS-1001b"],"tldr":"Safusidenib is an experimental small molecule inhibitor from Nuvation Bio in phase 3 trials for glioma & glioblastoma, aimed at IDH1 / IDH2.","summary":"Safusidenib (AB-218) is a small molecule inhibitor developed by Nuvation Bio. Its target is IDH1 / IDH2 (the sponsor names IDH1-mutant glioma). The sponsor states: novel, oral, potent, brain-penetrant, targeted inhibitor of mutant IDH1. ClinicalTrials.gov describes the intervention as: safusidenib administered continuously as dosed single agent orally on Days 1 to 28 of a 28-day cycle. Subjects may continue treatment with agent safusidenib until disease progression or development of other unacceptable toxicity. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05303519 (SIGMA (Safusidenib in IDH1 Mutant Glioma Maintenance)), in glioma & glioblastoma. The largest, NCT05303519, plans to enrol 365 participants with primary completion expected 2028-12-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Safusidenib","url":"https://clinicaltrials.gov/search?intr=AB-218"},{"label":"Sponsor page","url":"https://www.nuvationbio.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["glioblastoma","idh-mutant-astrocytoma","oligodendroglioma"],"sections":[],"technologies":[],"targets":["idh"],"drugs":[],"companies":["nuvation-bio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05303519","nct04458272"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AB-218","modality":"small molecule inhibitor","mechanism":"novel, oral, potent, brain-penetrant, targeted inhibitor of mutant IDH1.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"samarium-153-lexidronam","kind":"drug","name":"Samarium-153 lexidronam","aka":["153Sm-EDTMP","Samarium Sm 153 lexidronam pentasodium"],"tldr":"Quadramet (samarium-153 lexidronam) is a radioactive bone-seeking injection that lodges in bone metastases and irradiates them from inside, approved in 1997 to relieve pain from prostate, breast and other cancers spread to bone. Most patients get relief within one to two weeks, but marrow suppression follows at three to five weeks, and radium-223 and PSMA radioligands have largely replaced it.","summary":"Samarium-153 lexidronam was approved by the FDA in March 1997 and authorised in the EU in February 1998 for relief of pain in patients with confirmed osteoblastic metastatic bone lesions that take up technetium-labelled bisphosphonate on bone scan, on placebo-controlled trials in which most patients had pain relief within one to two weeks lasting for months. It and strontium-89 (Metastron) were the bone-pain radiopharmaceuticals of the 1990s; radium-223 (Xofigo), which extends survival in prostate cancer, and PSMA radioligands have since taken most of their role. A single intravenous dose is given; transient marrow suppression, with nadir at three to five weeks, is the main toxicity, and a pain flare can occur early. Distributed by Lantheus in the US and Curium in Europe.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Samarium_(153Sm)_lexidronam","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/quadramet"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=samarium"}],"tags":["ema-list","supportive"],"related":["radium-223"],"cancers":["prostate","breast-hr-positive","nsclc"],"sections":[],"technologies":["radioligand-therapy","palliative-care"],"targets":[],"drugs":[],"companies":["lantheus","curium"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00551525","nct00450619","nct01886105"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Quadramet","modality":"Bone-seeking therapeutic radiopharmaceutical (beta emitter)","supportive":true,"mechanism":"Samarium-153 chelated to the tetraphosphonate EDTMP concentrates in areas of high bone turnover around osteoblastic metastases; beta emission (1.9 day half-life) irradiates the metastases and relieves pain, with gamma emission allowing imaging.","approvals":[{"region":"US","year":1997,"indication":"Relief of pain in patients with confirmed osteoblastic metastatic bone lesions that enhance on radionuclide bone scan"},{"region":"EU","year":1998,"indication":"Relief of bone pain from multiple painful osteoblastic skeletal metastases (Quadramet)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"samuraciclib","kind":"drug","name":"Samuraciclib","aka":["ICEC0942","CT7001"],"tldr":"Samuraciclib is an oral cdk inhibitor from Pfizer, in registered phase 2 trials for metastatic cancer, HR-positive / HER2-negative breast cancer.","summary":"Samuraciclib is listed on ClinicalTrials.gov as an intervention in 2 registered phase 2 trials sponsored by Pfizer and Hoffmann-La Roche, in metastatic cancer, HR-positive / HER2-negative breast cancer. A cyclin-dependent kinase inhibitor: the name stem -ciclib marks a small molecule that stops the cell-cycle kinases tumour cells rely on to divide. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Samuraciclib","url":"https://clinicaltrials.gov/search?intr=Samuraciclib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer","breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["pfizer","flatiron-health"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06125522","nct04802759"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral CDK inhibitor","mechanism":"A cyclin-dependent kinase inhibitor: the name stem -ciclib marks a small molecule that stops the cell-cycle kinases tumour cells rely on to divide.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sapanisertib","kind":"drug","name":"Sapanisertib","aka":[],"tldr":"Sapanisertib is an experimental small-molecule drug from Faeth Therapeutics in phase 2 trials for HR-positive / HER2-negative breast cancer and endometrial cancer, aimed at mTOR.","summary":"Sapanisertib (FTH-003, MLN0128) is a small-molecule drug developed by Faeth Therapeutics. Its target is mTOR. ClinicalTrials.gov describes the intervention as: Sapanisertib is a small molecule inhibitor of the mammalian mTOR serine/threonine kinase. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in HR-positive / HER2-negative breast cancer and endometrial cancer. The largest, NCT06463028, plans to enrol 40 participants with primary completion expected 2028-09. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Sapanisertib","url":"https://clinicaltrials.gov/search?intr=FTH-003"},{"label":"Sponsor page","url":"https://www.faeththerapeutics.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","endometrial"],"sections":[],"technologies":[],"targets":["mtor"],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06463028","nct07558733"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"FTH-003, MLN0128","modality":"small molecule","mechanism":"Small-molecule drug directed at mTOR, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sargramostim","kind":"drug","name":"Sargramostim","aka":["rhu GM-CSF"],"tldr":"Sargramostim is a lab-made version of GM-CSF, the signal that tells bone marrow to make white cells; approved in 1991, it shortens the dangerous low-count period after leukaemia chemotherapy and stem cell transplants.","summary":"Sargramostim, recombinant human GM-CSF made in yeast, was approved by the FDA in March 1991 for myeloid reconstitution after autologous bone marrow transplantation in lymphoma and leukaemia. Its label grew to include neutrophil recovery after induction chemotherapy for acute myeloid leukaemia in older adults, stem cell mobilisation, allogeneic transplant and graft failure, and in 2018 the haematopoietic syndrome of acute radiation syndrome. Unlike G-CSF it also stimulates monocytes and dendritic cells, which is why GM-CSF is built into talimogene laherparepvec and several cancer vaccines. Fever, bone pain and fluid retention are the usual side effects.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Sargramostim","links":[{"label":"Drugs@FDA BLA103362","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=103362"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=sargramostim"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Sargramostim"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["aml","dlbcl"],"sections":[],"technologies":["cytokine-therapy"],"targets":["csf2ra"],"drugs":["filgrastim"],"companies":["partner-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Leukine","modality":"Recombinant granulocyte-macrophage colony-stimulating factor, injection","supportive":true,"mechanism":"Activates the GM-CSF receptor on myeloid progenitors, speeding recovery of neutrophils, monocytes and dendritic cells after chemotherapy or transplant.","approvals":[{"region":"US","year":1991,"indication":"Myeloid reconstitution after autologous bone marrow transplant; later AML post-induction, stem cell mobilisation, allogeneic transplant, graft failure and acute radiation syndrome"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"saruparib","kind":"drug","name":"Saruparib","aka":[],"tldr":"Saruparib is an experimental small-molecule drug from AstraZeneca in phase 3 trials for prostate cancer, ovarian cancer and non-small-cell lung cancer, aimed at PARP.","summary":"Saruparib (AZD5305) is a small-molecule drug developed by AstraZeneca. Its target is PARP (the sponsor names PARP (poly(ADP-ribose) polymerase)). The sponsor states: Saruparib (AZD5305) is an oral PARP inhibitor, studied added to androgen deprivation therapy (with or without abiraterone) in patients with BRCA-mutated prostate cancer. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06952803 (A Study of Metastases Free Survival With Saruparib vs Placebo Added to a Standard RT/ADT in Men With High-risk Prostate Cancer With a BRCA Mutation) and NCT06120491 (Saruparib (AZD5305) vs Placebo in Men With Metastatic Castration-Sensitive Prostate Cancer Receiving Physician's Choice New Hormonal Agents), in prostate cancer, ovarian cancer, non-small-cell lung cancer and endometrial cancer. The largest, NCT06120491, plans to enrol 1898 participants (actual) with primary completion expected 2027-09-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Saruparib","url":"https://clinicaltrials.gov/search?intr=AZD5305"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["prostate","ovarian","nsclc","endometrial"],"sections":[],"technologies":[],"targets":["parp"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06952803","nct06120491","nct05797168","nct05367440","nct05123482","nct07336446","nct04644068","nct07446855","nct06380751"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AZD5305","modality":"small molecule","mechanism":"Saruparib (AZD5305) is an oral PARP inhibitor, studied added to androgen deprivation therapy (with or without abiraterone) in patients with BRCA-mutated prostate cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sasanlimab","kind":"drug","name":"Sasanlimab","aka":[],"tldr":"Sasanlimab is an experimental monoclonal antibody from Pfizer in phase 3 trials for bladder & urothelial cancer, aimed at PD-1 and PD-L1.","summary":"Sasanlimab (PF-06801591) is a monoclonal antibody developed by Pfizer. Its targets are PD-1 and PD-L1 (the sponsor names PD-1). The sponsor states: A monoclonal antibody that blocks the interaction between PD-1 and its ligands PD-L1/PD-L2. ClinicalTrials.gov describes the intervention as: A monoclonal antibody (mAb) that blocks the interaction between PD-1 and PD-L1/PD-L2. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04165317 (A Study of Sasanlimab in People With Non-muscle Invasive Bladder Cancer), in bladder & urothelial cancer. The largest, NCT04165317, plans to enrol 1068 participants (actual) with primary completion was scheduled for 2024-12-02 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Sasanlimab","url":"https://clinicaltrials.gov/search?intr=Sasanlimab"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":[],"targets":["pd1","pdl1"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04165317"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PF-06801591","modality":"monoclonal antibody","mechanism":"A monoclonal antibody that blocks the interaction between PD-1 and its ligands PD-L1/PD-L2.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"satricabtagene-autoleucel","kind":"drug","name":"Satricabtagene autoleucel","aka":[],"tldr":"Satricabtagene autoleucel (satri-cel) is the first CAR-T therapy approved for a solid tumour (gastric cancer), in China.","summary":"Satricabtagene autoleucel (satri-cel) is an autologous CAR-T built from a humanised scFv against Claudin 18.2, a tight-junction protein normally confined to gastric mucosa and exposed on a large share of gastric and pancreatic adenocarcinomas; lymphodepletion uses a nab-paclitaxel-containing regimen. CARsgen developed it. In the randomised phase 2 CT041-ST-01 trial in China (Lancet 2025), satri-cel improved progression-free survival versus physician's choice in pretreated CLDN18.2-positive gastric or gastro-oesophageal junction cancer (median 3.25 versus 1.77 months; the abstract reports no overall survival figures), and NMPA approval followed in 2025-26 per company reports; the phase 2 dose was 2.5 x 10^8 CAR-positive T cells. Cytokine release syndrome and cytopenias occur, and gastric mucosal injury is an expected on-target effect. US trials are ongoing, and durability and use in pancreatic cancer remain open. For a newcomer: the first CAR-T approved anywhere for a solid tumour.","status":"approved","asOf":"2026-09-24","links":[{"label":"CT041-ST-01 (Lancet 2025)","url":"https://doi.org/10.1016/S0140-6736(25)00860-8"}],"tags":[],"related":[],"cancers":["gastric","pancreatic"],"sections":[],"technologies":["car-t"],"targets":["cldn18-2"],"drugs":[],"companies":["carsgen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04581473"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"satri-cel, CT041","modality":"CAR-T (Claudin 18.2)","mechanism":"Humanised CLDN18.2 scFv CAR-T; lymphodepletion with nab-paclitaxel-containing regimen.","approvals":[{"region":"China","year":2025,"indication":"CLDN18.2+ advanced gastric/GEJ adenocarcinoma, previously treated"}],"mechanismSteps":["Patient's T cells are collected by leukapheresis","Cells are engineered to express a CAR against Claudin 18.2 and expanded","Patient receives lymphodepleting chemotherapy","CAR-T cells are infused, home to tumour, and expand","CAR binds Claudin 18.2; T cell kills the tumour cell and proliferates","Memory CAR-T cells persist and patrol"],"dosing":{"route":"Single IV infusion after lymphodepletion","schedule":"2.5 × 10⁸ CAR+ T cells (phase 2 dose); lymphodepletion includes nab-paclitaxel, fludarabine, cyclophosphamide","monitoring":"CRS, gastric mucosal injury (on-target), cytopenias","source":"https://www.carsgen.com"},"toxicity":[{"event":"Cytokine release syndrome"},{"event":"Gastric mucosal injury / nausea"},{"event":"Cytopenias"},{"event":"Infections"}],"access":[{"country":"CN","reimbursement":"NMPA approved 2025-26; self-pay","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2022-01","type":"designation","region":"US","note":"RMAT designation, CLDN18.2+ gastric cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-09","type":"approval","region":"China","note":"First CAR-T approved for a solid tumour (CLDN18.2+ gastric/GEJ adenocarcinoma)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"savolitinib","kind":"drug","name":"Savolitinib","aka":[],"tldr":"Savolitinib is a Chinese-discovered pill that blocks the MET growth signal, approved in China for lung cancers with a MET exon 14 mutation and being tested worldwide with osimertinib.","summary":"Discovered by Hutchmed in Shanghai and partnered with AstraZeneca since 2011, savolitinib received conditional NMPA approval in June 2021 for MET exon 14 skipping NSCLC after platinum chemotherapy or in patients unfit for it, the first selective MET inhibitor approved in China; full approval and first-line use followed. In EGFR-mutant NSCLC with MET-driven resistance to osimertinib, the SAVANNAH and SACHI trials support the savolitinib plus osimertinib combination, with a Chinese approval of the combination in 2025 and the global SAFFRON phase 3 continuing. Also studied in papillary renal cell carcinoma and MET-amplified gastric cancer.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Savolitinib","links":[{"label":"Hutchmed: Orpathys","url":"https://www.hutch-med.com/"},{"label":"AstraZeneca and Hutchmed","url":"https://www.astrazeneca.com/media-centre/press-releases.html"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["nsclc","rcc","gastric","papillary-rcc","met-altered-nsclc","egfr-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["met","egfr"],"drugs":[],"companies":["hutchmed","astrazeneca"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct05009836","nct05043090","nct04606771","nct03944772","nct03091192","nct03778229","nct05261399","nct05374603"],"people":["su-weiguo"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Orpathys","code":"HMPL-504, AZD6094, volitinib","modality":"Small-molecule selective MET TKI","mechanism":"Highly selective ATP-competitive inhibitor of MET kinase, active against MET exon 14 skipping mutations and MET amplification.","approvals":[{"region":"China","year":2021,"indication":"MET exon 14 skipping NSCLC after platinum chemotherapy or unfit for chemotherapy (conditional; later full)"},{"region":"China","year":2025,"indication":"With osimertinib in EGFR-mutant NSCLC with MET amplification after EGFR TKI (SACHI)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sctb14","kind":"drug","name":"SCTB14","aka":[],"tldr":"SCTB14 is an experimental investigational agent whose form is not stated in the registry from Sinocelltech in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"SCTB14 is an investigational agent whose form is not stated in the registry developed by Sinocelltech. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SCTB14 is administered at selected dose by intravenous infusion on Day 1 of each 3-week cycle. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07362459 (A Study to Evaluate the Safety and Tolerability of SCTB14 as First-Line Therapy in Non-Small Cell Lung Cancer), in non-small-cell lung cancer. The largest, NCT07362459, plans to enrol 246 participants with primary completion expected 2027-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SCTB14","url":"https://clinicaltrials.gov/search?intr=SCTB14"},{"label":"Sponsor page","url":"https://www.sinocelltech.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sinocelltech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07362459"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sctb35","kind":"drug","name":"SCTB35","aka":[],"tldr":"SCTB35 is an experimental investigational agent whose form is not stated in the registry from Sinocelltech in phase 3 trials for diffuse large B-cell lymphoma and follicular lymphoma, with its target not yet stated publicly.","summary":"SCTB35 is an investigational agent whose form is not stated in the registry developed by Sinocelltech. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SCTB35 will be subcutaneously administered at a dose as specified. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07570823 (A Study to Evaluate the Safety and Efficacy of SCTB35 in Combination With Gemcitabine and Oxaliplatin in Patients With Relapsed or Refractory Diffuse Large B-Cell Lymphoma) and NCT07562022 (A Study to Evaluate the Safety and Efficacy of SCTB35 in Combination With Lenalidomide in Patients With Relapsed or Refractory Follicular Lymphoma), in diffuse large B-cell lymphoma and follicular lymphoma. The largest, NCT07570823, plans to enrol 101 participants with primary completion expected 2027-11. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SCTB35","url":"https://clinicaltrials.gov/search?intr=SCTB35"},{"label":"Sponsor page","url":"https://www.sinocelltech.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl","follicular-lymphoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sinocelltech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07570823","nct07562022"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sctb41","kind":"drug","name":"SCTB41","aka":[],"tldr":"SCTB41 is an experimental investigational agent whose form is not stated in the registry from Sinocelltech in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"SCTB41 is an investigational agent whose form is not stated in the registry developed by Sinocelltech. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SCTB41 of different doses,IV,every 3 weeks. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07662395 (SCTB41 Combined With Chemotherapy in Advanced Squamous Non-Small Cell Lung Cancer), in non-small-cell lung cancer. The largest, NCT06600022, plans to enrol 441 participants with primary completion expected 2027-04-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SCTB41","url":"https://clinicaltrials.gov/search?intr=SCTB41"},{"label":"Sponsor page","url":"https://www.sinocelltech.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sinocelltech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07662395","nct06600022"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sctc21c","kind":"drug","name":"SCTC21C","aka":[],"tldr":"SCTC21C is an experimental investigational agent whose form is not stated in the registry from Sinocelltech in phase 3 trials for multiple myeloma, with its target not yet stated publicly.","summary":"SCTC21C is an investigational agent whose form is not stated in the registry developed by Sinocelltech. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Pharmaceutical form: Solution for infusion； Route of administration: Subcutaneous. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07297329 (A Study to Evaluate the Safety and Efficacy of SCTC21C in Combination With Bortezomib, Lenalidomide and Dexamethasone in Patients With Newly Diagnosed Multiple Myeloma Not Eligible for Transplant), in multiple myeloma. The largest, NCT07297329, plans to enrol 292 participants with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SCTC21C","url":"https://clinicaltrials.gov/search?intr=SCTC21C"},{"label":"Sponsor page","url":"https://www.sinocelltech.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sinocelltech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07297329"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"selicrelumab","kind":"drug","name":"Selicrelumab","aka":[],"tldr":"Selicrelumab is a monoclonal antibody from Hoffmann-La Roche, in registered phase 2 trials for metastatic cancer.","summary":"Selicrelumab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Hoffmann-La Roche, in metastatic cancer. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Selicrelumab","url":"https://clinicaltrials.gov/search?intr=Selicrelumab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["flatiron-health"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03424005"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"selinexor","kind":"drug","name":"Selinexor","aka":[],"tldr":"Selinexor is a first-in-class pill that traps tumour-suppressor proteins inside the nucleus; approved in myeloma, it failed its endometrial cancer test in 2026.","summary":"Approved 2019 (penta-refractory myeloma with dexamethasone), 2020 (DLBCL; with bortezomib in myeloma, BOSTON). In endometrial cancer, SIENDO (2022) missed its primary endpoint but a TP53-wild-type subgroup showed a large PFS difference; the confirmatory XPORT-EC-042 missed its primary endpoint in July 2026. Nausea, fatigue, weight loss, and thrombocytopenia limit tolerability.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Selinexor","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Selinexor"}],"tags":[],"related":[],"cancers":["endometrial","multiple-myeloma","dlbcl","myeloma-relapsed-refractory"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["tp53"],"drugs":[],"companies":["karyopharm","antengene"],"institutions":[],"pathways":[],"terms":[],"trials":["xport-ec-042","nct04442022","nct06158841","nct02227251","nct02343042","nct04414475","nct04782687","nct04562389"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Xpovio","code":"KPT-330","modality":"Small-molecule XPO1 (nuclear export) inhibitor","mechanism":"Covalent inhibitor of exportin-1 (XPO1/CRM1), retaining p53, RB, FOXO, and other tumour suppressors in the nucleus and reducing oncoprotein translation.","approvals":[{"region":"US","year":2019,"indication":"Relapsed/refractory multiple myeloma (≥4 lines) with dexamethasone"},{"region":"US","year":2020,"indication":"Relapsed/refractory DLBCL; myeloma with bortezomib after ≥1 line"},{"region":"EU","year":2021,"indication":"EU brand Nexpovio","note":"Conditional marketing authorisation"}],"mechanismSteps":["Binds XPO1 in the nuclear pore complex","Tumour suppressors (p53, p21, RB) accumulate in the nucleus","Oncogene mRNAs (MYC, cyclin D) are not exported","Cells with intact p53 arrest or die"],"dosing":{"route":"Oral","schedule":"80 mg weekly with dexamethasone (myeloma); 60 mg weekly (endometrial trials)","monitoring":"Weekly CBC, sodium, weight; antiemetic prophylaxis"},"toxicity":[{"event":"Nausea","anyGradePct":84,"grade3PlusPct":10,"note":"SIENDO selinexor arm"},{"event":"Thrombocytopenia","anyGradePct":43,"grade3PlusPct":9},{"event":"Fatigue","anyGradePct":49,"grade3PlusPct":9}],"access":[],"regulatoryEvents":[{"date":"2019-07-03","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with dexamethasone for adults with relapsed/refractory multiple myeloma after at least 4 prior therapies and refractory to at least 2 proteasome inhibitors, 2 immunomodulatory agents, and an anti-CD38 monoclonal antibody"},{"date":"2020-06-22","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://wayback.archive-it.org/7993/20201222062817/https:/www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selinexor-relapsedrefractory-diffuse-large-b-cell-lymphoma","indication":"Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma (FL), after at least 2 lines of systemic therapy."},{"date":"2020-12-18","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2019 converted to traditional approval 1.5 years after it was granted.","indication":"In combination with dexamethasone for adults with relapsed/refractory multiple myeloma after at least 4 prior therapies and refractory to at least 2 proteasome inhibitors, 2 immunomodulatory agents, and an anti-CD38 monoclonal antibody"},{"date":"2026-04-30","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 5.9 years after its accelerated approval.","source":"https://wayback.archive-it.org/7993/20201222062817/https:/www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selinexor-relapsedrefractory-diffuse-large-b-cell-lymphoma","indication":"Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from follicular lymphoma (FL), after at least 2 lines of systemic therapy."},{"date":"2026-07-30","type":"filing","region":"US","note":"XPORT-EC-042 misses primary endpoint; endometrial development deprioritised","source":"https://cancerletter.com/clinical-roundup/20260807_8a/"}]},{"id":"selpercatinib","kind":"drug","name":"Selpercatinib","aka":[],"tldr":"Selpercatinib is a selective RET inhibitor approved for any tumour with a RET fusion, with a further label update in July 2026.","summary":"Selpercatinib is a selective RET kinase inhibitor, designed to avoid the off-target VEGFR effects of older multikinase drugs, taken as 160 mg twice daily. LIBRETTO-001 showed a response rate of about 84% in treatment-naive RET-fusion NSCLC, LIBRETTO-431 beat chemo-immunotherapy first line, and LIBRETTO-531 beat cabozantinib or vandetanib in RET-mutant medullary thyroid cancer. It was approved in 2020 for RET-fusion NSCLC and thyroid cancer and RET-mutant MTC, gained a tumour-agnostic RET-fusion indication in 2022, and a July 2026 FDA action is listed among that month's oncology approvals. Hypertension, raised transaminases and QT prolongation are the main adverse events. Acquired resistance through RET solvent-front mutations and bypass pathways is the current limit. For a newcomer: a pill that works wherever a RET fusion drives the cancer, regardless of organ.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Selpercatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Selpercatinib"},{"label":"NICE TA911: selpercatinib for untreated RET fusion-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta911"},{"label":"NICE TA1042: selpercatinib for previously treated RET fusion-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1042"}],"tags":[],"related":["ret-fusion"],"cancers":["thyroid","nsclc","medullary-thyroid-cancer","papillary-thyroid-cancer","ret-fusion-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["ret"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":["cancer-drugs-fund"],"trials":["nct03157128","nct04280081","nct03899792","nct04819100","libretto-431"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Retevmo","modality":"Small-molecule kinase inhibitor (RET)","mechanism":"Highly selective RET TKI.","approvals":[{"region":"US","year":2020,"indication":"RET-fusion NSCLC and thyroid; RET-mutant MTC"},{"region":"US","year":2022,"indication":"RET-fusion solid tumours (tumour-agnostic)"},{"region":"US","year":2026,"indication":"Label update (July 2026)"},{"region":"EU","year":2021,"indication":"EU brand Retsevmo"},{"region":"England (NICE)","year":2023,"indication":"Untreated RET fusion-positive advanced non-small-cell lung cancer","note":"TA911, published 26 July 2023, recommends selpercatinib with managed access only, under a managed access agreement."},{"region":"England (NICE)","year":2025,"indication":"Previously treated RET fusion-positive advanced non-small-cell lung cancer not previously treated with a RET inhibitor","note":"TA1042, published 19 February 2025, routine commissioning subject to the commercial arrangement."}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of RET fusion and mutant proteins","Phosphorylation of downstream substrates stops","Highly selective; spares VEGFR, avoiding multikinase toxicity","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"160 mg twice daily (≥50 kg); 120 mg BID (<50 kg); paediatric by BSA","modifications":"Reduce for hepatotoxicity, QTc, hypertension","monitoring":"LFTs every 2 weeks for 3 months; blood pressure; ECG; TSH; growth plates in children","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Oedema"},{"event":"Diarrhoea"},{"event":"Fatigue"},{"event":"Dry mouth"},{"event":"Hypertension"},{"event":"AST/ALT increased"},{"event":"QT prolongation"},{"event":"Hypersensitivity"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.lillycares.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for RET-fusion NSCLC and thyroid cancers (TA911, TA742, TA760)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2020-05-08","type":"accelerated-approval","region":"US","note":"Accelerated approval, RET-fusion NSCLC and thyroid cancer, RET-mutant MTC (LIBRETTO-001)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy *"},{"date":"2020-05-08","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/ongoing-cancer-accelerated-approvals#endnote2","indication":"Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) *"},{"date":"2020-05-08","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adult patients with metastatic RET fusion-positive Non-Small Cell Lung Cancer (NSCLC)"},{"date":"2022-09-21","type":"accelerated-approval","region":"US","note":"RET-fusion solid tumours (tumour-agnostic, accelerated); full approval in NSCLC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with locally advanced or metastatic solid tumors with a RET gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options *"},{"date":"2022-09-21","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 2.4 years after it was granted.","indication":"Adult patients with metastatic RET fusion-positive Non-Small Cell Lung Cancer (NSCLC)"},{"date":"2024-04-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Formulation: Adult and pediatric patients 12 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy"},{"date":"2024-04-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Formulation: Adult and pediatric patients 12 years of age and older with advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)"},{"date":"2024-04-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Formulation: Adult patients with locally advanced or metastatic solid tumors with a RET gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options"},{"date":"2024-05-29","type":"approval","region":"US","note":"Paediatric ≥2 years","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-06-12","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 4.1 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/ongoing-cancer-accelerated-approvals#endnote2","indication":"Adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) *"},{"date":"2024-06-12","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 0.2 years after it was granted.","indication":"Formulation: Adult and pediatric patients 12 years of age and older with advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate)"},{"date":"2024-09-27","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2020 converted to traditional approval 4.4 years after it was granted.","indication":"Adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy *"},{"date":"2024-09-27","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 0.5 years after it was granted.","indication":"Formulation: Adult and pediatric patients 12 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy"},{"date":"2026-07-14","type":"conversion","region":"US","note":"Traditional approval, locally advanced or metastatic RET fusion-positive solid tumours","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-traditional-approval-selpercatinib-locally-advanced-or-metastatic-ret-fusion-positive","indication":"Adult patients with locally advanced or metastatic solid tumors with a RET gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options *"},{"date":"2026-07-14","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 2.3 years after it was granted.","indication":"Formulation: Adult patients with locally advanced or metastatic solid tumors with a RET gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options"}]},{"id":"selumetinib","kind":"drug","name":"Selumetinib","aka":[],"tldr":"Selumetinib (Koselugo) is the first medicine for children and adults with neurofibromatosis type 1 whose plexiform neurofibromas, benign but disfiguring and painful nerve tumours, cannot be removed by surgery.","summary":"Selumetinib was approved by the FDA in April 2020 for children aged 2 and over with NF1 and symptomatic, inoperable plexiform neurofibromas, on the SPRINT phase 2 stratum 1 trial in which two-thirds of 50 children had a confirmed partial response with improvements in pain and function; approval was extended to adults (KOMET trial) and, with a granule formulation, to children from 1 year in 2025. The EU granted conditional authorisation in 2021. Plexiform neurofibromas are benign tumours, but the drug is an oncology-class MEK inhibitor developed with Merck & Co. Rash, paronychia, diarrhoea, raised creatine kinase, reduced ejection fraction and ocular toxicity are class effects and require monitoring. Mirdametinib (Gomekli, 2025) is the second MEK inhibitor for the same condition.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Selumetinib","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=selumetinib"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/selumetinibsulfate"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/koselugo"}],"tags":["nci-list"],"related":["trametinib"],"cancers":[],"sections":[],"technologies":["kinase-inhibitors"],"targets":["mek"],"drugs":[],"companies":["astrazeneca","merck"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct01933932"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["NF1 plexiform neurofibroma is a benign tumour with no cancer record; not linked to a cancer id."],"brand":"Koselugo","modality":"Small-molecule allosteric MEK1/2 inhibitor","mechanism":"Non-ATP-competitive inhibitor of MEK1 and MEK2; in NF1-deficient cells with unrestrained RAS activity it lowers ERK signalling and shrinks plexiform neurofibromas.","approvals":[{"region":"US","year":2020,"indication":"NF1 with symptomatic, inoperable plexiform neurofibromas, children 2 and over (extended to adults and to age 1 and over in 2025)"},{"region":"EU","year":2021,"indication":"Symptomatic, inoperable plexiform neurofibromas in NF1, children 3 and over (conditional)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"imp4927","kind":"drug","name":"Senaparib (IMP4297)","aka":["senaparib","IMP4297","IMP4927","Sepalna"],"tldr":"Senaparib is an oral PARP inhibitor from IMPACT Therapeutics tested as maintenance treatment after first-line chemotherapy for advanced ovarian cancer. On 17 September 2026 the EMA's medicines committee recommended EU approval under the name Sepalna; the European Commission's decision is pending.","summary":"Senaparib (IMP4297; the registry's spelling of the code is IMP4927) is a small-molecule PARP inhibitor developed by IMPACT Therapeutics. The phase 3 study NCT04169997 (FLAMES, 404 participants) tested it as monotherapy maintenance after first-line chemotherapy in advanced ovarian cancer. On 17 September 2026 the CHMP adopted a positive opinion recommending a marketing authorisation for Sepalna (applicant SFL Pharmaceuticals Deutschland GmbH) as monotherapy for the maintenance treatment of adults with advanced (FIGO stage III and IV) high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response after first-line platinum-based chemotherapy. The register gives the benefit as longer progression-free survival than placebo and lists anaemia, leukopenia, neutropenia, thrombocytopenia, nausea, raised transaminases and fatigue first among the most common side effects. The status stays at phase 3 until the European Commission decides. No efficacy figures are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of IMP4927","url":"https://clinicaltrials.gov/search?intr=IMP4927"},{"label":"Sponsor page","url":"https://www.impacttherapeutics.com"},{"label":"EMA: Sepalna (senaparib), CHMP opinion 17 September 2026","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/sepalna"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":["parp"],"drugs":[],"companies":["impact-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04169997"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Mechanism, indication wording, applicant and opinion date read from the EMA register page for Sepalna on 24 September 2026 (PR 74 review); before that the record said the target was not stated."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"brand":"Sepalna","modality":"small molecule (oral PARP inhibitor)","mechanism":"Inhibitor of poly(ADP-ribose) polymerase (PARP), ATC code L01XK07, that blocks the repair of damaged DNA in cancer cells and causes them to die (EMA summary of opinion, 17 September 2026).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"serabelisib","kind":"drug","name":"Serabelisib","aka":[],"tldr":"Serabelisib is an experimental small-molecule drug from Faeth Therapeutics in phase 2 trials for HR-positive / HER2-negative breast cancer and endometrial cancer, aimed at PIK3CA / PI3K-alpha.","summary":"Serabelisib (FTH-001, MLN1117) is a small-molecule drug developed by Faeth Therapeutics. Its target is PIK3CA / PI3K-alpha. ClinicalTrials.gov describes the intervention as: Serabelisib is a selective, small molecule inhibitor of PI3Kα. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in HR-positive / HER2-negative breast cancer and endometrial cancer. The largest, NCT06463028, plans to enrol 40 participants with primary completion expected 2028-09. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Serabelisib","url":"https://clinicaltrials.gov/search?intr=FTH-001"},{"label":"Sponsor page","url":"https://www.faeththerapeutics.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","endometrial"],"sections":[],"technologies":[],"targets":["pik3ca"],"drugs":[],"companies":["takeda","celcuity"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06463028","nct07558733"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"FTH-001, MLN1117","modality":"small molecule","mechanism":"Small-molecule drug directed at PIK3CA / PI3K-alpha, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"serplulimab","kind":"drug","name":"Serplulimab","aka":[],"tldr":"Serplulimab is a Chinese PD-1 antibody with the largest survival gain of any first-line small-cell lung cancer immunotherapy trial, approved in China, Europe, and the UK but not yet the US.","summary":"Serplulimab is a humanised IgG4 antibody against PD-1, given at 4.5 mg/kg every 3 weeks with carboplatin-etoposide and then as maintenance for up to 2 years. In ASTRUM-005 it produced OS 15.4 versus 10.9 months (HR 0.63) in extensive-stage SCLC, the largest survival gain among the first-line chemo-immunotherapy trials. It is approved in China (2022, the first PD-1 antibody for ES-SCLC), the EU (2025), the UK, India and Korea; Henlius develops it with Accord in Europe. In the US a bridging trial versus atezolizumab (ASTRIDE) has fully enrolled and the FDA decision is pending. Immune-related adverse events occurred in 37% of patients, with hypothyroidism in 12% and pneumonitis in 4%. It is a PD-1 drug whose data match or exceed the established Western options, but whose reach still depends on regulators outside China.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Serplulimab"},{"label":"NICE TA1167: serplulimab with carboplatin and etoposide for untreated extensive-stage small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta1167"}],"tags":[],"related":[],"cancers":["sclc","extensive-stage-sclc","lung-cancer"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["henlius"],"institutions":[],"pathways":[],"terms":["limited-extensive-stage"],"trials":["astrum-005","nct05353257","nct04547166","nct05468489","nct07253142","nct07269782","nct06848699","nct06349980","nct05787613"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Hansizhuang / Hetronifly","code":"HLX10","modality":"Monoclonal antibody (anti-PD-1)","mechanism":"Humanised IgG4 anti-PD-1.","approvals":[{"region":"China","year":2022,"indication":"First-line ES-SCLC with chemotherapy"},{"region":"EU","year":2025,"indication":"First-line ES-SCLC with carboplatin-etoposide"},{"region":"England (NICE)","year":2026,"indication":"Untreated extensive-stage small-cell lung cancer, with carboplatin and etoposide","note":"TA1167, published 18 June 2026 (ASTRUM-005); must be funded in England within 90 days of publication."}],"mechanismSteps":["Binds PD-1 on exhausted T cells","Blocks PD-L1/PD-L2 engagement","Restores cytotoxic T-cell function against antigens released by chemotherapy"],"dosing":{"route":"Intravenous","schedule":"4.5 mg/kg every 3 weeks with carboplatin-etoposide, then maintenance until progression or 2 years"},"toxicity":[{"event":"Immune-related adverse events (any)","anyGradePct":37},{"event":"Hypothyroidism","anyGradePct":12},{"event":"Pneumonitis","anyGradePct":4}],"access":[],"regulatoryEvents":[{"date":"2025-02","type":"approval","region":"EU","note":"European Commission approval for ES-SCLC","source":"https://www.henlius.com/en/NewsDetails-5903-26.html"},{"date":"2025","type":"approval","region":"UK","note":"MHRA approval","source":"https://www.henlius.com/en/NewsDetails-5324-26.html"}]},{"id":"setidegrasib","kind":"drug","name":"Setidegrasib","aka":[],"tldr":"Setidegrasib is an experimental investigational agent whose form is not stated in the registry from Astellas Pharma Global Development in phase 3 trials for pancreatic ductal adenocarcinoma and non-small-cell lung cancer, aimed at KRAS.","summary":"Setidegrasib (ASP3082) is an investigational agent whose form is not stated in the registry developed by Astellas Pharma Global Development. Its target is KRAS (the sponsor names KRAS G12D). The sponsor states: Setidegrasib (ASP3082) is designed to stop the action of abnormal proteins produced by the KRAS G12D mutation; given as a weekly intravenous infusion combined with mFOLFIRINOX or NALIRIFOX chemotherapy in KRAS G12D-mutated metastatic pancreatic cancer, and versus docetaxel in KRAS G12D-mutated NSCLC. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07409272 (A Study to Evaluate the Effectiveness and Safety of Setidegrasib, Given With Either mFOLFIRINOX or NALIRIFOX Chemotherapies, in People With Pancreatic Cancer) and NCT07566052 (A Study to Compare Setidegrasib (ASP3082) With Docetaxel, in People With Non-small Cell Lung Cancer With a KRAS G12D Mutation), in pancreatic ductal adenocarcinoma and non-small-cell lung cancer. The largest, NCT07409272, plans to enrol 614 participants with primary completion expected 2029-08-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Setidegrasib","url":"https://clinicaltrials.gov/search?intr=ASP3082"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic","nsclc"],"sections":[],"technologies":[],"targets":["kras"],"drugs":[],"companies":["astellas"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07409272","nct07566052"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["23 Sept 2026: a reviewer reports ASP3082 as a KRAS G12D-selective protein degrader that recruits the VHL E3 ligase, per its discovery paper. That paper is not in the corpus and could not be checked when this note was written, so the modality stays as the registry states it and no E3 ligase is recorded; neither this record nor the open drug engine names cereblon for it (the engine files it outside the degrader format until a sourced modality is added). Add the discovery paper as a link and set the modality to a degrader to move it into the target x E3 ligase grid."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ASP3082","modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Setidegrasib (ASP3082) is designed to stop the action of abnormal proteins produced by the KRAS G12D mutation; given as a weekly intravenous infusion combined with mFOLFIRINOX or NALIRIFOX chemotherapy in KRAS G12D-mutated metastatic pancreatic cancer, and versus docetaxel in KRAS G12D-mutated NSCLC.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"setmelanotide","kind":"drug","name":"Setmelanotide","aka":["RM-493"],"tldr":"Setmelanotide is a daily injection that switches back on the brain's fullness signal. It is approved for rare genetic causes of obesity and, since March 2026 in the United States, for the continuous weight gain that follows damage to the hypothalamus from a tumour such as craniopharyngioma or from its treatment.","summary":"Setmelanotide is Rhythm Pharmaceuticals' melanocortin-4 receptor agonist. The FDA approved it in 2020 for chronic weight management in obesity due to POMC, PCSK1 or LEPR deficiency, later for Bardet-Biedl syndrome, and on 19 March 2026 for acquired hypothalamic obesity in adults and children aged 4 years and older (Drugs@FDA supplement 9; label revised March 2026). It is injected under the skin once daily and reduces hunger and weight in patients whose hypothalamic satiety pathway is broken upstream of the receptor.\n\nHypothalamic obesity after craniopharyngioma or other hypothalamic injury is an acquired lesion of the same pathway and resists diet and conventional drugs. After a phase 2 study showed weight loss, the phase 3 TRANSCEND trial (NCT05774756; Miller, NEJM 2026) randomised 120 participants aged 4 to 66 and found a least-squares mean body-mass index change of minus 16.5 percent with setmelanotide against plus 3.3 percent with placebo at 52 weeks, with less hunger, which supported the label expansion. The craniopharyngioma page names it, with GLP-1 agonists and oxytocin, among the treatments for hypothalamic obesity.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Setmelanotide","links":[{"label":"TRANSCEND (NEJM 2026)","url":"https://doi.org/10.1056/NEJMoa2512275"},{"label":"Imcivree label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=70c3ccf7-4df0-4c75-ba07-fede9970c8d9"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Setmelanotide"}],"tags":["subtype-drugs-wave"],"related":[],"cancers":["craniopharyngioma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["rhythm-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["transcend"],"people":[],"bottlenecks":[],"keyPapers":["paper-transcend-miller-nejm-2026"],"journals":[],"dependsOn":[],"notes":[],"brand":"Imcivree","modality":"Subcutaneous melanocortin-4 receptor agonist","mechanism":"Activates the melanocortin-4 receptor downstream of the leptin-POMC pathway in the hypothalamus, restoring the satiety signal that is lost when the hypothalamus is damaged by a craniopharyngioma or its treatment.","approvals":[{"region":"US","year":2020,"indication":"Chronic weight management in obesity due to POMC, PCSK1 or LEPR deficiency"},{"region":"US","year":2026,"indication":"Acquired hypothalamic obesity in adults and children aged 4 years and older","note":"Approved 19 March 2026 (Drugs@FDA supplement 9) on the TRANSCEND trial"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sevabertinib","kind":"drug","name":"Sevabertinib","aka":[],"tldr":"Sevabertinib is an oral HER2 inhibitor for lung cancers with HER2 mutations, approved in November 2025 as an alternative to Enhertu and zongertinib.","summary":"Bayer's reversible HER2 TKI, derived from a Broad Institute alliance. SOHO-01: ORR ~64% in previously treated HER2-mutant NSCLC; accelerated approval 20 November 2025 for HER2 tyrosine-kinase-domain mutations after prior systemic therapy. First-line sNDA under priority review (2026). Diarrhoea and rash are the main toxicities; less ILD than T-DXd.","status":"approved","asOf":"2026-09-06","links":[{"label":"FDA approval (Bayer)","url":"https://www.bayer.com/en/us/news-stories/hyrnuo"},{"label":"OncLive report","url":"https://www.onclive.com/view/fda-grants-accelerated-approval-to-sevabertinib-in-her2-mutated-nonsquamous-nsclc"}],"tags":[],"related":[],"cancers":["nsclc","her2-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["her2"],"drugs":["zongertinib","trastuzumab-deruxtecan"],"companies":["bayer"],"institutions":[],"pathways":[],"terms":[],"trials":["soho-01","nct06452277"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Hyrnuo","code":"BAY 2927088","modality":"Small-molecule kinase inhibitor (HER2)","mechanism":"Reversible, HER2-mutant-selective TKI sparing wild-type EGFR.","approvals":[{"region":"US","year":2025,"indication":"HER2 TKD-mutant non-squamous NSCLC after prior systemic therapy (accelerated)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2025-11-19","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 0.8 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sevabertinib-non-squamous-non-small-cell-lung-cancer","indication":"Treatment of adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy"},{"date":"2026-09-09","type":"accelerated-approval","region":"US","note":"FDA grants accelerated approval to sevabertinib for locally advanced or metastatic non-squamous non-small cell lung cancer The confirmatory requirement was still open 0.0 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sevabertinib-locally-advanced-or-metastatic-non-squamous-non-small","indication":"Treatment of adult patients with locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-authorized test"}]},{"id":"sg301","kind":"drug","name":"SG301","aka":[],"tldr":"SG301 is an experimental investigational agent whose form is not stated in the registry from Hangzhou Sumgen Biotech in phase 3 trials for multiple myeloma, aimed at CD38.","summary":"SG301 is an investigational agent whose form is not stated in the registry developed by Hangzhou Sumgen Biotech. Its target is CD38 (the sponsor names CD38). The sponsor states: Humanized anti-CD38 monoclonal antibody; a subcutaneous formulation (SG301 SC) has also been cleared to enter clinical trials. ClinicalTrials.gov describes the intervention as: Dosage form: solution for infusion Route of administration: intravenous Frequency: weekly intervals (QW) for 8 weeks, then every 2 weeks (Q2W) thereafter. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06508983 (A Clinical Study Comparing SG301 Plus Pomalidomide and Dexamethasone to Placebo Plus Pomalidomide and Dexamethasone in Relapsed or Refractory Multiple Myeloma Patients), in multiple myeloma. The largest, NCT06508983, plans to enrol 360 participants with primary completion expected 2027-03-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SG301","url":"https://clinicaltrials.gov/search?intr=SG301"},{"label":"Sponsor page","url":"https://www.sumgenbio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":["cd38"],"drugs":[],"companies":["hangzhou-sumgen-biotech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06508983"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody (humanised anti-CD38, per the sponsor's description; form not stated in the registry record)","mechanism":"Humanized anti-CD38 monoclonal antibody; a subcutaneous formulation (SG301 SC) has also been cleared to enter clinical trials.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sgm-101","kind":"drug","name":"SGM-101","aka":[],"tldr":"SGM-101 is an experimental monoclonal antibody from Surgimab in phase 3 trials for colorectal cancer, aimed at CEACAM5.","summary":"SGM-101 is a monoclonal antibody developed by Surgimab. Its target is CEACAM5 (the sponsor names CEA (carcinoembryonic antigen)). The sponsor states: A fluorochrome-labelled anti-CEA monoclonal antibody used as an intraoperative near-infrared imaging agent to delineate primary and recurrent tumour and metastases during surgery. ClinicalTrials.gov describes the intervention as: A fluorochrome-labeled anti-carcinoembryonic antigen (CEA) monoclonal antibody intraoperative imaging agent. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT03659448 (Performance of SGM-101 for the Delineation of Primary and Recurrent Tumour and Metastases in Patients Undergoing Surgery for Colorectal Cancer), in colorectal cancer. The largest, NCT03659448, plans to enrol 300 participants with primary completion was scheduled for 2024-11 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SGM-101","url":"https://clinicaltrials.gov/search?intr=SGM-101"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["ceacam5"],"drugs":[],"companies":["surgimab"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03659448"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"A fluorochrome-labelled anti-CEA monoclonal antibody used as an intraoperative near-infrared imaging agent to delineate primary and recurrent tumour and metastases during surgery.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"shield","kind":"drug","name":"Shield","aka":[],"tldr":"The first FDA-approved blood test for colorectal cancer screening (2024), now optionally reporting other cancers too.","summary":"Shield is Guardant Health's blood-based colorectal cancer screening test: it reads cell-free DNA for methylation patterns, fragmentation signatures and mutations that indicate a tumour. It was the first FDA-approved blood test for colorectal cancer screening (2024), for average-risk adults aged 45 and over with Medicare coverage. In the ECLIPSE study it detected 83% of colorectal cancers with 90% specificity, but only 13% of advanced adenomas, the precancerous lesions that colonoscopy removes to prevent cancer. Its value therefore lies in reaching people who decline colonoscopy or stool tests, and any positive result must be followed by colonoscopy. In 2025 a Shield MCD option added multi-cancer detection with Breakthrough designation. For a newcomer: a convenient blood test for bowel cancer that catches cancers well but polyps poorly.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA premarket approval P230009: Shield blood-based colorectal cancer screening test","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P230009"}],"tags":[],"related":[],"cancers":["colorectal"],"sections":[],"technologies":["mced","liquid-biopsy"],"targets":[],"drugs":[],"companies":["guardant-health"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Blood-based colorectal cancer screening test","mechanism":"cfDNA methylation, fragmentomics, and mutations.","approvals":[{"region":"US","year":2024,"indication":"Colorectal cancer screening, average-risk adults ≥45"}],"mechanismSteps":["Cell-free DNA is extracted from plasma","Methylation, fragmentomic, and mutation signals are measured","Classifier reports colorectal cancer signal detected or not","Positive results are followed by colonoscopy"],"toxicity":[],"access":[{"country":"US","listPrice":"$1,495 list price","reimbursement":"Medicare covers Shield every 3 years for average-risk adults 45-85 (2024 NCD); commercial coverage expanding","source":"https://guardanthealth.com/shield","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-05-23","type":"advisory-committee","region":"US","note":"FDA advisory committee votes favourably","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-07-29","type":"approval","region":"US","note":"PMA approval: first blood test for primary colorectal cancer screening (ECLIPSE)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-08","type":"approval","region":"US","note":"Medicare coverage under the blood-based CRC screening NCD","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-06","type":"designation","region":"US","note":"Breakthrough Device designation for Shield multi-cancer (MCD)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"shr-1501","kind":"drug","name":"SHR-1501","aka":[],"tldr":"SHR-1501 is an experimental investigational agent whose form is not stated in the registry from Suzhou Suncadia Biopharmaceuticals in phase 3 trials for bladder & urothelial cancer, with its target not yet stated publicly.","summary":"SHR-1501 is an investigational agent whose form is not stated in the registry developed by Suzhou Suncadia Biopharmaceuticals and Shanghai Hengrui Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SHR-1501 single agent dose escalation, and in combination of BCG or SHR-1316. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07424287 (A Study of Intravesical SHR-1501 Combination With BCG Versus Investigator-selected Chemotherapy in Subjects With BCG-unresponsive NMIBC) and NCT07726992 (A Study of SHR-1501 Combination With BCG Versus Placebo Combined With BCG in Subjects With Papillary-Only NMIBC), in bladder & urothelial cancer. The largest, NCT07726992, plans to enrol 750 participants with primary completion expected 2029-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SHR-1501","url":"https://clinicaltrials.gov/search?intr=SHR-1501"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07424287","nct07726992","nct07073534"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"shr-8068","kind":"drug","name":"SHR-8068","aka":[],"tldr":"SHR-8068 is an experimental investigational agent whose form is not stated in the registry from Suzhou Suncadia Biopharmaceuticals in phase 3 trials for biliary tract cancer, non-small-cell lung cancer and cervical cancer, with its target not yet stated publicly.","summary":"SHR-8068 is an investigational agent whose form is not stated in the registry developed by Suzhou Suncadia Biopharmaceuticals and Shanghai Hengrui Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SHR-8068: injection, 50mg（10mL）/ bottle, intravenous drip. It is the investigational product in 10 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07229625 (A Phase III Study of SHR-8068 in Combination With Adebrelimab and Platinum-Containing Chemotherapy Versus Durvalumab in Combination With Platinum-Containing Chemotherapy as First-line Treatment for Advanced Biliary Tract Cancer (BTC)), in biliary tract cancer, non-small-cell lung cancer, cervical cancer, gastric & gastro-oesophageal junction cancer and bladder & urothelial cancer. The largest, NCT06703177, plans to enrol 876 participants with primary completion was scheduled for 2026-01-01 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SHR-8068","url":"https://clinicaltrials.gov/search?intr=SHR-8068"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cholangiocarcinoma","nsclc","cervical","gastric","urothelial","colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07229625","nct07454720","nct06589778","nct07071714","nct07239596","nct06754930","nct06859775","nct06465563","nct06247956","nct06778031","nct06639347","nct07110571","nct07028281","nct07241767","nct06703177"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody (anti-CTLA-4, per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"shr-a1912","kind":"drug","name":"SHR-A1912","aka":[],"tldr":"SHR-A1912 is an experimental investigational agent whose form is not stated in the registry from Suzhou Suncadia Biopharmaceuticals in phase 3 trials for diffuse large B-cell lymphoma, with its target not yet stated publicly.","summary":"SHR-A1912 is an investigational agent whose form is not stated in the registry developed by Suzhou Suncadia Biopharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06929624 (A Phase 3 Clinical Study of SHR-A1912 Combined With R-GemOx Versus R-GemOx in Diffuse Large B-cell Lymphoma), in diffuse large B-cell lymphoma. The largest, NCT06929624, plans to enrol 280 participants with primary completion expected 2027-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SHR-A1912","url":"https://clinicaltrials.gov/search?intr=SHR-A1912"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06929624"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"shr-a1921","kind":"drug","name":"SHR-A1921","aka":[],"tldr":"SHR-A1921 is an experimental investigational agent whose form is not stated in the registry from Suzhou Suncadia Biopharmaceuticals in phase 3 trials for ovarian cancer, with its target not yet stated publicly.","summary":"SHR-A1921 is an investigational agent whose form is not stated in the registry developed by Suzhou Suncadia Biopharmaceuticals and Shanghai Hengrui Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Drug: SHR-A1921 administered as an IV infusion Drug: Adebrelimab administered as an IV infusion Drug: Carboplatin administered as an IV infusion Drug: Cisplatin administered as an IV infusion Drug: Bevacizumab administered as an IV infusion. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06394492 (SHR-A1921 for Injection in Patients With Platinum-Resistant Recurrent Epithelial Ovarian Cancer), in ovarian cancer. The largest, NCT06394492, plans to enrol 440 participants with primary completion was scheduled for 2026-05-31 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SHR-A1921","url":"https://clinicaltrials.gov/search?intr=SHR-A1921"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06394492","nct06474455","nct05765032"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"shr-a2009","kind":"drug","name":"SHR-A2009","aka":[],"tldr":"SHR-A2009 is an experimental investigational agent whose form is not stated in the registry from Shanghai Hengrui Pharmaceutical in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"SHR-A2009 is an investigational agent whose form is not stated in the registry developed by Shanghai Hengrui Pharmaceutical and Suzhou Suncadia Biopharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SHR-A2009 monotherapy ，SHR-A2009 will be administered intravenously. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06671379 (A Study of SHR-A2009 Versus Platinum-based Chemotherapy in EGFR-mutated, Advanced or Metastatic NSCLC), in non-small-cell lung cancer. The largest, NCT06671379, plans to enrol 498 participants (actual) with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SHR-A2009","url":"https://clinicaltrials.gov/search?intr=SHR-A2009"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06671379","nct07183189","nct06222879","nct06474455"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"shr-a2102","kind":"drug","name":"SHR-A2102","aka":[],"tldr":"SHR-A2102 is an experimental investigational agent whose form is not stated in the registry from Shanghai Hengrui Pharmaceutical in phase 3 trials for cervical cancer, non-small-cell lung cancer and head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"SHR-A2102 is an investigational agent whose form is not stated in the registry developed by Shanghai Hengrui Pharmaceutical and Suzhou Suncadia Biopharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SHR-A2102: injection, 80mg/ bottle, intravenous drip. It is the investigational product in 9 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07418749 (A Study of SHR-A2102 Versus Investigator's Choice of Chemotherapy in Patients With Platinum-based Chemotherapy and PD-(L)1 Inhibitor Treatment Failed Recurrent or Metastatic Cervical Cancer), in cervical cancer, non-small-cell lung cancer, head and neck squamous cell carcinoma and bladder & urothelial cancer. The largest, NCT06895928, plans to enrol 400 participants with primary completion expected 2028-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SHR-A2102","url":"https://clinicaltrials.gov/search?intr=SHR-A2102"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cervical","nsclc","head-and-neck","urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07418749","nct06589778","nct07059221","nct06512051","nct07229729","nct07175220","nct06895928","nct06639347","nct06915142","nct07393542","nct06417554","nct06879145","nct06474468","nct06654440"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["23 Sept 2026: target and payload are not on any record OnCo reads (no paper is linked here and the registry states neither), so the open drug engine lists SHR-A2102 as not placed rather than guessing; add the target and payload with a source to put it in the grid."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"si-b001","kind":"drug","name":"SI-B001","aka":[],"tldr":"SI-B001 is an experimental investigational agent whose form is not stated in the registry from Sichuan Baili Pharmaceutical in phase 3 trials for non-small-cell lung cancer and head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"SI-B001 is an investigational agent whose form is not stated in the registry developed by Sichuan Baili Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SI-B001 is administered by intravenous drip once weekly (QW). 120 min ± 10 min after the first intravenous drip, if the infusion reaction is tolerable during the first dose, the subsequent infusion can be completed within 60-120 min (unless. It is the investigational product in 7 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05020769 (SI-B001 Combined With Osimertinib Mesylate Tablets in the Treatment of Recurrent Metastatic Non-small Cell Lung Cancer), in non-small-cell lung cancer and head and neck squamous cell carcinoma. The largest, NCT05949606, plans to enrol 95 participants (actual) with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SI-B001","url":"https://clinicaltrials.gov/search?intr=SI-B001"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["systimmune"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05020769","nct06668961","nct05949606","nct05020457","nct05044897","nct05054439","nct05668858"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody (SI-B001 is izalontamab, EGFR x HER3; INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"signatera","kind":"drug","name":"Signatera","aka":[],"tldr":"Signatera is Natera's tumour-informed blood test that tracks 16 mutations from each patient's own tumour to detect residual or returning cancer after surgery. Medicare covers it in colorectal, breast, bladder, lung and ovarian cancer and for immunotherapy monitoring, and in 2026 it selected the bladder cancer patients for the first approval based on circulating tumour DNA.","summary":"Signatera is Natera's tumour-informed MRD test and tracks 16 patient-specific variants. Medicare-covered in colorectal, breast, bladder, lung, ovarian, and immunotherapy monitoring. Used in interventional trials (CIRCULATE-US, ZEST in TNBC with niraparib, negative for feasibility reasons; IMvigor011 used Signatera to select ctDNA+ bladder patients for atezolizumab, leading to the first ctDNA-based approval in 2026).","status":"established","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov NCT04660344 (IMvigor011)","url":"https://clinicaltrials.gov/study/NCT04660344"}],"tags":[],"related":[],"cancers":["colorectal","urothelial","tnbc","nsclc","pancreatic"],"sections":[],"technologies":["mrd-testing","liquid-biopsy"],"targets":[],"drugs":[],"companies":["natera"],"institutions":[],"pathways":[],"terms":[],"trials":["imvigor011","dynamic-iii","altair"],"people":[],"bottlenecks":[],"keyPapers":["paper-magbanua-ispy2-ctdna-neoadjuvant-ann-oncol-2021","paper-lee-ctdna-adjuvant-benefit-localized-pancreatic-ann-oncol-2019","paper-groot-kras-ctdna-clinical-test-resected-pancreatic-ccr-2019","paper-reinert-ctdna-ultradeep-sequencing-colorectal-jama-oncol-2019","paper-henriksen-ctdna-stage-iii-colorectal-improve-it-ccr-2022","paper-galaxy-signatera-nat-med-2023"],"journals":[],"dependsOn":[],"notes":["Pancreatic ductal adenocarcinoma: tumour-informed residual disease testing after resection is a research readout here; the published pancreatic series used bespoke KRAS droplet PCR or SafeSeqS rather than a commercial assay, and found every ctDNA-positive patient recurred (Lee 2019, Groot 2019).","Colorectal cancer: this is the disease with the deepest tumour-informed residual disease evidence. Week-4 positivity carried a recurrence hazard ratio of 10 in 1,039 prospectively followed patients (Kotani 2023), and serial testing separates patients who clear during adjuvant chemotherapy, who do not relapse, from those who do not (Henriksen 2022). No approval turns on the result; the randomised evidence is for de-escalation in stage II (DYNAMIC), not escalation."],"modality":"Tumour-informed ctDNA MRD test","mechanism":"WES of tumour → bespoke 16-plex PCR/NGS assay on plasma at very high depth.","approvals":[],"mechanismSteps":["Tumour tissue and normal DNA are sequenced (whole exome)","16 patient-specific clonal variants are chosen","A bespoke multiplex PCR assay is built","Plasma is tested at high depth at each visit","Detection of ≥2 variants calls ctDNA positive, months before imaging"],"toxicity":[],"access":[{"country":"US","listPrice":"~$3,500 per test (Medicare rate, approximate)","reimbursement":"Medicare covers colorectal (stage II-III and IV), muscle-invasive bladder, breast (neoadjuvant/adjuvant), ovarian, NSCLC and pan-cancer immunotherapy monitoring under MolDX; commercial coverage growing","source":"https://www.natera.com/oncology/signatera-advanced-cancer-detection/","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2019-05","type":"designation","region":"US","note":"FDA Breakthrough Device designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-03","type":"approval","region":"US","note":"Medicare coverage (MolDX) for stage II-III colorectal cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-10","type":"approval","region":"US","note":"Medicare coverage for immunotherapy response monitoring, pan-cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q2","type":"label-change","region":"US","note":"Used to select ctDNA-positive patients in the first ctDNA-guided drug approval (IMvigor011, atezolizumab)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"silevertinib","kind":"drug","name":"Silevertinib","aka":["silevertinib (BDTX-1535)"],"tldr":"Silevertinib is an experimental small-molecule drug from Black Diamond Therapeutics in phase 2 trials for non-small-cell lung cancer, aimed at EGFR.","summary":"Silevertinib (BDTX-1535) is a small-molecule drug developed by Black Diamond Therapeutics. Its target is EGFR (the sponsor names EGFR). The sponsor states: A first and best-in-class 4th generation, brain-penetrant, irreversible EGFR inhibitor designed to address classical and non-classical EGFR driver and resistance mutations in NSCLC and glioblastoma. ClinicalTrials.gov describes the intervention as: Silevertinib (BDTX-1535) is a 4th generation irreversible brain penetrant EGFR MasterKey inhibitor, which targets a family of oncogenic EGFR classical and non-classical driver and resistance mutations in NSCLC. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT05256290, plans to enrol 200 participants with primary completion was scheduled for 2025-11-03 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Silevertinib","url":"https://clinicaltrials.gov/search?intr=BDTX-1535"},{"label":"Sponsor pipeline page","url":"https://blackdiamondtherapeutics.com/pipeline-programs/silevertinib-nsclc"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["egfr"],"drugs":[],"companies":["black-diamond-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05256290","nct07326566"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"BDTX-1535","modality":"small molecule","mechanism":"A first and best-in-class 4th generation, brain-penetrant, irreversible EGFR inhibitor designed to address classical and non-classical EGFR driver and resistance mutations in NSCLC and glioblastoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"siltuximab","kind":"drug","name":"Siltuximab","aka":[],"tldr":"Siltuximab (Sylvant) is an antibody that neutralises the inflammatory messenger interleukin-6. It is the only approved treatment for multicentric Castleman disease, a rare lymph node disorder, in people without HIV or HHV-8 infection.","summary":"Siltuximab was approved by the FDA in April 2014 and by the EU in the same year for multicentric Castleman disease in HIV-negative and HHV-8-negative patients, on a randomised placebo-controlled phase 2 trial of 79 patients in which durable tumour and symptomatic responses occurred only in the siltuximab arm. Castleman disease is a lymphoproliferative disorder rather than a cancer, but it is managed by haematologist-oncologists and the NCI lists siltuximab among cancer drugs. Infusion is every three weeks indefinitely in responders. Because IL-6 blockade suppresses C-reactive protein and fever, infections may be masked; hypertriglyceridaemia and neutropenia occur. Tocilizumab, which blocks the IL-6 receptor, is used in the same disease off label.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Siltuximab","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=siltuximab"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/siltuximab"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/sylvant"}],"tags":["nci-list"],"related":["tocilizumab"],"cancers":[],"sections":[],"technologies":["monoclonal-antibody"],"targets":["il6"],"drugs":[],"companies":["recordati","johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07570173","nct06679829","nct03315026"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Multicentric Castleman disease is a lymphoproliferative disorder with no cancer record."],"brand":"Sylvant","modality":"Monoclonal antibody (anti-IL-6, chimeric IgG1)","mechanism":"Binds interleukin-6 directly and prevents it engaging soluble and membrane-bound IL-6 receptors, cutting the cytokine drive behind Castleman disease.","approvals":[{"region":"US","year":2014,"indication":"Multicentric Castleman disease in HIV-negative and HHV-8-negative patients"},{"region":"EU","year":2014,"indication":"Multicentric Castleman disease in HIV-negative and HHV-8-negative adults"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sim0270","kind":"drug","name":"SIM0270","aka":[],"tldr":"SIM0270 is an experimental investigational agent whose form is not stated in the registry from Jiangsu Simcere Pharmaceutical in phase 3 trials for HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"SIM0270 is an investigational agent whose form is not stated in the registry developed by Jiangsu Simcere Pharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06680921 (A Study of SIM0270 Combined With Everolimus vs. Treatment of Physician's Choice in Patients With ER+/HER2- Advanced Breast Cancer (SIMRISE)), in HR-positive / HER2-negative breast cancer. The largest, NCT06680921, plans to enrol 482 participants (actual) with primary completion expected 2028-08-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SIM0270","url":"https://clinicaltrials.gov/search?intr=SIM0270"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["simcere"],"institutions":[],"pathways":[],"terms":[],"trials":["simrise"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"simplescreen-crc","kind":"drug","name":"SimpleScreen CRC","aka":[],"tldr":"The second FDA-approved blood test for bowel cancer screening, from Freenome, sold by Abbott from late 2026.","summary":"Freenome's blood test was approved by the FDA on 24 July 2026 for average-risk adults aged 45 and older, based on the PREEMPT CRC study of more than 48,000 participants, in which sensitivity for colorectal cancer was 79.2%, specificity for advanced neoplasia 91.5%, and sensitivity for advanced precancerous lesions 12.5% (JAMA 2025). Abbott holds US commercial rights. Like Shield, the test is intended for people who decline stool tests or colonoscopy: guidelines still rank it below colonoscopy and FIT because it misses most precancerous polyps and therefore prevents fewer cancers.","status":"approved","asOf":"2026-09-24","links":[{"label":"PREEMPT CRC (JAMA 2025)","url":"https://doi.org/10.1001/jama.2025.7515"},{"label":"FDA approval (MedTech Dive)","url":"https://www.medtechdive.com/news/freenome-secures-fda-nod-for-blood-based-colorectal-cancer-test/826290/"},{"label":"Freenome","url":"https://www.freenome.com"}],"tags":["test"],"related":["shield","cologuard-plus"],"cancers":["colorectal"],"sections":[],"technologies":["colorectal-screening","liquid-biopsy","methylation-profiling"],"targets":[],"drugs":[],"companies":["freenome","abbott"],"institutions":[],"pathways":[],"terms":["cfdna","screening","sensitivity-specificity"],"trials":["preempt-crc"],"people":[],"bottlenecks":[],"keyPapers":["paper-preempt-crc-shaukat-jama-2025"],"journals":[],"dependsOn":[],"notes":["What a result means: a positive result needs a colonoscopy; a negative result is reassuring for cancer but says little about polyps, so the test should be repeated on schedule."],"brand":"SimpleScreen","modality":"Blood-based colorectal cancer screening test (cfDNA methylation and protein)","mechanism":"Multiomics plasma assay combining cell-free DNA methylation with protein biomarkers and machine learning to classify colorectal cancer signal.","approvals":[{"region":"US","year":2026,"indication":"Colorectal cancer screening in average-risk adults aged 45 and older"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sintilimab","kind":"drug","name":"Sintilimab","aka":[],"tldr":"Sintilimab is Innovent's PD-1 antibody, approved in China since 2018 for Hodgkin lymphoma and then, on the ORIENT trials, for first-line lung, liver, oesophageal and stomach cancer. In 2022 the FDA rejected its lung cancer application because a China-only trial against chemotherapy did not fit US practice, defining the agency's stance on single-country data.","summary":"First approved by the NMPA in December 2018 for relapsed or refractory classical Hodgkin lymphoma, then added by the ORIENT programme: first-line non-squamous NSCLC with pemetrexed-platinum (ORIENT-11), squamous NSCLC (ORIENT-12), first-line hepatocellular carcinoma with the bevacizumab biosimilar IBI305 (ORIENT-32), first-line oesophageal squamous cell carcinoma (ORIENT-15) and first-line gastric or gastro-oesophageal junction adenocarcinoma (ORIENT-16). Co-commercialised in China with Eli Lilly. In March 2022 the FDA issued a complete response letter for the ORIENT-11 indication after an advisory committee voted 14 to 1 that a single-country trial with a chemotherapy comparator did not fit US practice, the case that defined the agency's stance on China-only data. Listed on the National Reimbursement Drug List since 2019 after a steep negotiated price cut.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Sintilimab","links":[{"label":"Innovent: Tyvyt","url":"https://www.innoventbio.com/"},{"label":"ORIENT-11 (J Thorac Oncol 2020)","url":"https://doi.org/10.1016/j.jtho.2020.07.014"},{"label":"ORIENT-32 (Lancet Oncol 2021)","url":"https://doi.org/10.1016/S1470-2045(21)00252-7"},{"label":"ORIENT-16 (JAMA 2023)","url":"https://doi.org/10.1001/jama.2023.19918"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["nsclc","hcc","esophageal","gastric","hodgkin-lymphoma"],"sections":[],"technologies":["checkpoint-inhibitor","monoclonal-antibody"],"targets":["pd1"],"drugs":[],"companies":["innovent","eli-lilly"],"institutions":[],"pathways":["pd1-checkpoint"],"terms":[],"trials":["orient-11","orient-32","orient-16","nct06584032","nct05116462","nct06497985","nct07521852","nct05632939","nct06558227","nct06530251","nct05890742","nct06220318"],"people":["yu-michael"],"bottlenecks":[],"keyPapers":["paper-xu-jama","paper-ren-lancet-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Tyvyt","code":"IBI308","modality":"Monoclonal antibody (anti-PD-1, IgG4)","mechanism":"Fully human IgG4 antibody that blocks PD-1 binding to PD-L1 and PD-L2, restoring T-cell activity against the tumour.","approvals":[{"region":"China","year":2018,"indication":"Relapsed or refractory classical Hodgkin lymphoma after two or more lines"},{"region":"China","year":2021,"indication":"First-line non-squamous NSCLC with pemetrexed and platinum (ORIENT-11); first-line squamous NSCLC with gemcitabine and platinum (ORIENT-12); first-line unresectable hepatocellular carcinoma with IBI305 (ORIENT-32)"},{"region":"China","year":2022,"indication":"First-line oesophageal squamous cell carcinoma with chemotherapy (ORIENT-15)"},{"region":"China","year":2023,"indication":"First-line gastric or gastro-oesophageal junction adenocarcinoma with chemotherapy (ORIENT-16)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2022-03","type":"crl","region":"US","note":"Complete response letter for ORIENT-11 (first-line non-squamous NSCLC); ODAC voted 14 to 1 that additional data were needed for US applicability","source":"https://web.archive.org/web/20250302091714/https://www.fda.gov/advisory-committees/advisory-committee-calendar/february-10-2022-meeting-oncologic-drugs-advisory-committee-meeting-announcement-02102022"}]},{"id":"sipuleucel-t","kind":"drug","name":"Sipuleucel-T","aka":[],"tldr":"Sipuleucel-T was the first therapeutic cancer vaccine approved (2010): it lengthened survival in metastatic prostate cancer by about four months without shrinking tumours or lowering PSA.","summary":"IMPACT (NEJM 2010): OS 25.8 vs 21.7 months in asymptomatic/minimally symptomatic mCRPC. Cost, logistics (three leukaphereses) and absence of PSA/radiographic response limited uptake; Dendreon went bankrupt in 2014. Remains NCCN-listed for low-volume mCRPC and a proof of principle for cancer vaccines.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Sipuleucel-T","links":[{"label":"IMPACT (NEJM 2010)","url":"https://doi.org/10.1056/NEJMoa1001294"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=sipuleucel"},{"label":"IMPACT (New England Journal of Medicine 2010)","url":"https://doi.org/10.1056/NEJMoa1001294"},{"label":"NICE TA332: sipuleucel-T for asymptomatic or minimally symptomatic metastatic hormone-relapsed prostate cancer, guidance withdrawn","url":"https://www.nice.org.uk/guidance/ta332"}],"tags":["gap-fill"],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["shared-antigen-vaccine"],"targets":[],"drugs":[],"companies":["dendreon"],"institutions":[],"pathways":[],"terms":["prostate-uk-drug-approvals"],"trials":["nct06134232","impact-sipuleucel-t"],"people":[],"bottlenecks":[],"keyPapers":["paper-kantoff-n-engl-j-med"],"journals":[],"dependsOn":[],"notes":["Sipuleucel-T was the first therapeutic cancer vaccine approved anywhere, on the strength of IMPACT: median overall survival 25.8 against 21.7 months, hazard ratio 0.78 (95 percent confidence interval 0.61 to 0.98, p=0.03), with no difference in time to objective disease progression. It is not available in England: NICE TA332 has been withdrawn because the marketing authorisation was withdrawn. Dendreon, which made it, filed for bankruptcy in 2014; the manufacturing burden of a per-patient autologous product was never recovered in sales."],"brand":"Provenge","modality":"Autologous cellular immunotherapy (antigen-presenting cell vaccine)","mechanism":"Patient's leukapheresed APCs are cultured with PA2024 (prostatic acid phosphatase fused to GM-CSF) and reinfused to prime PAP-specific T cells.","approvals":[{"region":"US","year":2010,"indication":"Asymptomatic or minimally symptomatic metastatic castration-resistant prostate cancer"},{"region":"EU","year":2013,"indication":"Same (withdrawn 2015 for commercial reasons)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sirolimus","kind":"drug","name":"Sirolimus","aka":["Rapamycin"],"tldr":"Sirolimus is an anti-rejection tablet taken after an organ transplant. Switching to it from a calcineurin inhibitor roughly halves the number of new skin squamous cell carcinomas in people who have already had one, at the cost of more side effects and a quarter of people stopping it.","summary":"Sirolimus, first isolated from a soil bacterium on Rapa Nui and originally an antifungal, was approved by the FDA in 1999 to prevent rejection of a kidney transplant. It is not a cancer drug and has no cancer indication; it belongs to this family because of what switching to it does to skin cancer risk.\n\nTUMORAPA randomised 120 kidney transplant recipients who had already had one cutaneous squamous cell carcinoma either to switch from a calcineurin inhibitor to sirolimus or to carry on. New squamous cell carcinomas occurred in 22 per cent against 39 per cent, a relative risk of 0.56 (95 per cent confidence interval 0.32 to 0.98), with the median time to the next cancer moving from 7 to 15 months and graft function stable in both arms. The cost was 60 serious adverse events against 14 and 23 per cent stopping the drug, and switching rapidly caused twice as many serious events as switching gradually. That trade is why the switch is discussed rather than made automatically.\n\nSirolimus is also the drug the immunosuppression is cross-tapered to before a transplant recipient is given PD-1 blockade for advanced disease, which is how the Dana-Farber study avoided rejection. Two related records exist in the corpus: temsirolimus, its intravenous ester, and albumin-bound sirolimus, which is licensed for perivascular epithelioid cell tumour.","status":"approved","asOf":"2026-09-25","wikipedia":"https://en.wikipedia.org/wiki/Sirolimus","links":[{"label":"Euvrard et al., sirolimus and secondary skin-cancer prevention in kidney transplantation, TUMORAPA (New England Journal of Medicine 2012)","url":"https://doi.org/10.1056/NEJMoa1204166"},{"label":"DailyMed: Rapamune (sirolimus) prescribing information","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=RAPAMUNE"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Sirolimus"}],"tags":["skin"],"related":["temsirolimus","sirolimus-albumin-bound","cemiplimab"],"cancers":["cutaneous-scc","skin-cancer","advanced-cutaneous-scc"],"sections":[],"technologies":[],"targets":["mtor"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":["skin-cancer-after-organ-transplant"],"trials":["tumorapa","cemiplimab-kidney-transplant-cscc"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Rapamune","modality":"Oral mammalian target of rapamycin (mTOR) inhibitor, used as an immunosuppressant","mechanism":"Binds FKBP12; the complex inhibits mTOR complex 1, blocking the interleukin-2 signal that drives lymphocyte proliferation. The same inhibition slows tumour cell growth and angiogenesis, which is why an anti-rejection drug has an antitumour effect.","approvals":[{"region":"US","year":1999,"indication":"Prophylaxis of organ rejection in kidney transplant recipients"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sirolimus-albumin-bound","kind":"drug","name":"Sirolimus protein-bound particles","aka":["nab-sirolimus","ABI-009","Nanoparticle albumin-bound rapamycin"],"tldr":"Fyarro is an infusion of the transplant drug sirolimus packaged in albumin particles. It is the first approved treatment for malignant PEComa, a rare soft-tissue tumour driven by loss of the TSC genes.","summary":"Sirolimus protein-bound particles were approved by the FDA in November 2021 for adults with locally advanced unresectable or metastatic malignant perivascular epithelioid cell tumour (PEComa), on the AMPECT single-arm trial in which 39% of 31 patients responded, with responses concentrated in tumours carrying TSC2 inactivation and several lasting over two years. The albumin formulation allows higher intratumoural drug exposure than oral rapalogues. It is being studied in other TSC1/2-altered solid tumours (PRECISION 1). Stomatitis, myelosuppression, hyperglycaemia, hyperlipidaemia and pneumonitis are the mTOR-class effects.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Sirolimus","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=sirolimus%20protein-bound"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/sirolimus-protein-bound-particles"}],"tags":["nci-list"],"related":["everolimus","temsirolimus"],"cancers":["sarcoma","pecoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["mtor"],"drugs":[],"companies":["aadi-bioscience"],"institutions":[],"pathways":["pi3k-akt-mtor"],"terms":[],"trials":["nct05997017","nct05103358"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Fyarro","modality":"Albumin-bound mTOR inhibitor (intravenous)","mechanism":"Sirolimus bound to albumin nanoparticles; binds FKBP12 and allosterically inhibits mTORC1, the kinase left unrestrained by the TSC1/TSC2 loss that drives PEComa.","approvals":[{"region":"US","year":2021,"indication":"Locally advanced unresectable or metastatic malignant PEComa"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sitneprotafib","kind":"drug","name":"Sitneprotafib","aka":[],"tldr":"Sitneprotafib is an experimental small-molecule drug from Allist Pharmaceuticals in phase 3 trials for non-small-cell lung cancer, colorectal cancer and pancreatic ductal adenocarcinoma, with its target not yet stated publicly.","summary":"Sitneprotafib (JAB-3312) is a small-molecule drug developed by Allist Pharmaceuticals. The sponsor describes its target as SHP2, which OnCo does not yet have a target page for. The sponsor states: JAB-3312 (sitneprotafib) is a second-generation allosteric SHP2 inhibitor developed by Jacobio, described as having more potent anti-tumour activity than earlier SHP2 inhibitors; combined with the KRAS G12C inhibitor glecirasib (JAB-21822) in a registrational phase 3 trial for first-line KRAS G12C-mutated NSCLC. ClinicalTrials.gov describes the intervention as: JAB-3312 administered orally as a tablet or capsule. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06416410 (JAB-21822 Combined With JAB-3312 Compared SOC in the First Line for Treatment of Advanced Non-small Cell Lung Cancer With KRAS p.G12C Mutation), in non-small-cell lung cancer, colorectal cancer and pancreatic ductal adenocarcinoma. The largest, NCT06416410, plans to enrol 392 participants with primary completion expected 2029-09-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Sitneprotafib","url":"https://clinicaltrials.gov/search?intr=Sitneprotafib"},{"label":"Sponsor pipeline page","url":"https://www.jacobiopharma.com/en/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","colorectal","pancreatic"],"sections":[],"technologies":[],"targets":["shp2"],"drugs":[],"companies":["allist"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06416410","nct05288205"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"JAB-3312","modality":"small molecule","mechanism":"JAB-3312 (sitneprotafib) is a second-generation allosteric SHP2 inhibitor developed by Jacobio, described as having more potent anti-tumour activity than earlier SHP2 inhibitors; combined with the KRAS G12C inhibitor glecirasib (JAB-21822) in a registrational phase 3 trial for first-line KRAS G12C-mutated NSCLC.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"skb105","kind":"drug","name":"SKB105","aka":[],"tldr":"SKB105 is an antibody-drug conjugate from Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd., in registered phase 2 trials for metastatic cancer.","summary":"SKB105 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Sichuan Kelun-Biotech Biopharmaceutical Co., Ltd., in metastatic cancer. An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of SKB105","url":"https://clinicaltrials.gov/search?intr=SKB105"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["kelun-biotech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07380386"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"SKB105","modality":"Antibody-drug conjugate","mechanism":"An antibody-drug conjugate, as described in the registry record: an antibody against a tumour surface protein carries a cytotoxic payload into the cell.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"socazolimab","kind":"drug","name":"Socazolimab","aka":[],"tldr":"Socazolimab is Lee's Pharmaceutical's PD-L1 antibody, in phase 3 trials in China for extensive-stage small cell lung cancer and recurrent cervical cancer.","summary":"Socazolimab (ZKAB001), licensed by Lee's Pharmaceutical from Sorrento, is a fully human PD-L1 antibody. Its phase 3 programme in China includes first-line extensive-stage small cell lung cancer with chemotherapy and recurrent or metastatic cervical cancer; earlier trials included osteosarcoma. OnCo records its Chinese regulatory status only where a source states it.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Socazolimab","url":"https://clinicaltrials.gov/search?intr=ZKAB001"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cervical","sclc"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pdl1"],"drugs":[],"companies":["lees-pharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06459687"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"ZKAB001","modality":"Anti-PD-L1 monoclonal antibody, intravenous","mechanism":"Binds PD-L1 on tumour and immune cells so it cannot engage PD-1 on T cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sodium-thiosulfate","kind":"drug","name":"Sodium thiosulfate (otoprotectant)","aka":[],"tldr":"An old antidote proven in two paediatric trials to halve permanent hearing loss from cisplatin, and approved in 2022 as the first drug to prevent a chemotherapy side effect in children.","summary":"SIOPEL-6 (NEJM 2018, hepatoblastoma) and ACCL0431 (Lancet Oncol 2017) showed hearing loss reduced from ~63% to ~33% without compromising survival in localised disease (a survival signal in disseminated disease in ACCL0431 keeps use to localised tumours). Approved FDA September 2022 (ages ≥1 month), EMA 2023.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Sodium_thiosulfate_(medical_use)","links":[{"label":"SIOPEL-6 (NEJM 2018)","url":"https://doi.org/10.1056/NEJMoa1801109"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Pedmark"}],"tags":["gap-fill","supportive"],"related":[],"cancers":["hepatoblastoma","medulloblastoma","osteosarcoma","neuroblastoma"],"sections":[],"technologies":["platinum","survivorship-care-plan"],"targets":[],"drugs":[],"companies":["fennec-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05382338","nct00716976","nct00652132"],"people":[],"bottlenecks":[],"keyPapers":["paper-brock-n-engl-j-med"],"journals":[],"dependsOn":[],"notes":[],"brand":"Pedmark","modality":"Small-molecule chemoprotectant","supportive":true,"mechanism":"Thiol that inactivates circulating cisplatin and scavenges reactive oxygen species in the cochlea when given 6 hours after cisplatin, after tumour exposure is complete.","approvals":[{"region":"US","year":2022,"indication":"Reduce risk of ototoxicity from cisplatin in patients ≥1 month with localised, non-metastatic solid tumours"},{"region":"EU","year":2023,"indication":"Same"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sofetabart-mipitecan","kind":"drug","name":"Sofetabart Mipitecan","aka":["Sofe-M"],"tldr":"Sofetabart Mipitecan is an experimental antibody-drug conjugate from Eli Lilly and in phase 3 trials for ovarian cancer, aimed at Folate receptor alpha.","summary":"Sofetabart Mipitecan (LY4170156) is an antibody-drug conjugate developed by Eli Lilly and. Its target is Folate receptor alpha (the sponsor names folate receptor alpha). The sponsor states: An antibody-drug conjugate targeting folate receptor alpha that delivers a topoisomerase inhibitor payload to cancer cells. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07213804 (A Three-Part Phase 3 Study of Sofetabart Mipitecan in Participants With Platinum-Resistant (Part A) and Platinum-Sensitive (Parts B and C) Ovarian Cancer), in ovarian cancer. The largest, NCT07213804, plans to enrol 1630 participants with primary completion expected 2028-11. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Sofetabart Mipitecan","url":"https://clinicaltrials.gov/search?intr=LY4170156"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":["folr1"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07213804"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"LY4170156","modality":"ADC","mechanism":"An antibody-drug conjugate targeting folate receptor alpha that delivers a topoisomerase inhibitor payload to cancer cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"octreotide-lanreotide","kind":"drug","name":"Somatostatin analogues (octreotide, lanreotide)","aka":["Octreotide","Lanreotide","Octreotide LAR","Somatostatin analogue"],"tldr":"Monthly injections of a synthetic hormone that both quiets tumour hormone symptoms and slows tumour growth, the first treatment for most neuroendocrine tumours.","summary":"Octreotide (1988) controls carcinoid syndrome; PROMID (2009) showed octreotide LAR delays progression in midgut NETs (TTP 14.3 vs 6.0 months); CLARINET (2014) showed lanreotide improves PFS across enteropancreatic NETs (median not reached vs 18 months, HR 0.47). Standard first-line antiproliferative therapy for SSTR-positive grade 1-2 disease; also premedication against carcinoid crisis.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Octreotide","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Somatostatin%20analogues"}],"tags":[],"related":[],"cancers":["neuroendocrine","small-intestinal-net","pancreatic-net","lung-net","thymoma"],"sections":[],"technologies":[],"targets":["sstr2"],"drugs":[],"companies":["novartis","ipsen"],"institutions":[],"pathways":[],"terms":["carcinoid-syndrome","prrt-term"],"trials":["promid","clarinet","netter-1"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Sandostatin LAR, Somatuline Depot","modality":"Peptide hormone analogue (SSTR2 agonist)","mechanism":"SSTR2/5 agonism suppresses hormone secretion and proliferation; PROMID and CLARINET established the antiproliferative effect.","approvals":[{"region":"US","year":1988,"indication":"Carcinoid syndrome symptoms (octreotide)"},{"region":"US","year":2014,"indication":"Unresectable GEP-NETs to improve PFS (lanreotide)"}],"mechanismSteps":["Binds SSTR2 on tumour cell","Inhibits adenylyl cyclase and hormone exocytosis","Cell-cycle arrest via SHP-1/SHP-2 phosphatases","Symptom relief within days; growth slowing over months"],"dosing":{"route":"Deep subcutaneous or intramuscular depot","schedule":"Octreotide LAR 30 mg or lanreotide 120 mg every 4 weeks; short-acting octreotide for breakthrough symptoms","monitoring":"Gallstones, glucose, vitamin B12"},"toxicity":[{"event":"Diarrhoea/steatorrhoea","anyGradePct":26,"note":"CLARINET"},{"event":"Cholelithiasis","anyGradePct":10},{"event":"Hyperglycaemia","anyGradePct":5}],"access":[],"regulatoryEvents":[]},{"id":"cmg901","kind":"drug","name":"Sonesitatug vedotin","aka":[],"tldr":"Sonesitatug vedotin is a Claudin 18.2 ADC in phase 3 for gastric cancer, licensed by AstraZeneca from KYM Biosciences.","summary":"Sonesitatug vedotin (CMG901) is an anti-Claudin 18.2 antibody carrying the microtubule inhibitor MMAE through a cleavable linker; Claudin 18.2 is a tight-junction protein normally buried in gastric mucosa but exposed on gastric and some pancreatic cancer cells, giving the ADC a tumour-selective address. AstraZeneca licensed global rights from KYM Biosciences in 2023. Phase 1 produced an objective response rate around 33 percent in CLDN18.2-positive gastric cancer, and the CLARITY-Gastric01 phase 3 trial is testing it at 2.2 mg/kg every 3 weeks in second-line gastric cancer. Nausea, vomiting, neutropenia and decreased appetite are the main toxicities, the gut effects reflecting on-target binding to normal stomach lining. It competes with XNW27011 and other CLDN18.2 ADCs, so randomised data will decide the field. It delivers a cell poison to a protein hidden in healthy tissue.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Sonesitatug vedotin","url":"https://clinicaltrials.gov/search?intr=CMG901"}],"tags":[],"related":[],"cancers":["gastric","pancreatic"],"sections":[],"technologies":["adc"],"targets":["cldn18-2"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06219941","nct07431281","nct07069712"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"CMG901, AZD0901","modality":"ADC","payload":"MMAE","linker":"Cleavable","mechanism":"Anti-CLDN18.2 with MMAE.","approvals":[],"mechanismSteps":["Antibody binds Claudin 18.2 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","MMAE is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"2.2 mg/kg every 3 weeks (phase 3 dose)","source":"https://clinicaltrials.gov/study/NCT06346392"},"toxicity":[{"event":"Nausea"},{"event":"Vomiting"},{"event":"Neutropenia"},{"event":"Decreased appetite"}],"access":[{"country":"US","reimbursement":"Investigational (CLARITY-Gastric01)","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-02","type":"filing","region":"US","note":"AstraZeneca licenses global rights from KYM Biosciences","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"sonidegib","kind":"drug","name":"Sonidegib","aka":[],"tldr":"Sonidegib (Odomzo) is the second hedgehog inhibitor for locally advanced basal cell carcinoma, similar in effect and side effects to vismodegib.","summary":"Sonidegib is an oral smoothened (SMO) antagonist that blocks hedgehog signalling, the pathway driven by PTCH1 or SMO mutations in nearly all basal cell carcinomas. It has a long half-life and high tissue distribution. In the BOLT trial (2015), 56% of patients with locally advanced BCC responded to the 200 mg dose, with responses that remained durable at 42 months of follow-up. It was approved in the US and EU in 2015 for locally advanced BCC that has recurred after surgery or radiation or in patients who are not candidates for those treatments, but not for metastatic disease. Its adverse effects are those of the class: muscle spasms, taste disturbance, hair loss and raised creatine kinase, which often lead to dose interruption. For a newcomer, sonidegib is the second hedgehog pill for advanced basal cell carcinoma and behaves much like vismodegib.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Sonidegib","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=sonidegib"}],"tags":["gap-fill"],"related":[],"cancers":["basal-cell-carcinoma","medulloblastoma-shh","locally-advanced-bcc","skin-cancer"],"sections":[],"technologies":["kinase-inhibitors","hedgehog-inhibitors"],"targets":["smoothened"],"drugs":[],"companies":["novartis","sun-pharma"],"institutions":[],"pathways":[],"terms":["hedgehog-inhibitor-tolerability"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Odomzo","modality":"Small-molecule smoothened inhibitor","mechanism":"SMO antagonist blocking hedgehog signalling; long half-life and high tissue distribution.","approvals":[{"region":"US","year":2015,"indication":"Locally advanced BCC recurrent after surgery or radiation, or not candidates"},{"region":"EU","year":2015,"indication":"Same"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sonrotoclax","kind":"drug","name":"Sonrotoclax","aka":[],"tldr":"Sonrotoclax is a more potent, shorter-acting successor to venetoclax. It was approved for mantle cell lymphoma in May 2026 and is in late-stage trials with zanubrutinib for CLL.","summary":"Approved in China (late 2025) for relapsed MCL and CLL/SLL after BTK inhibitor, and in the US on 13 May 2026 (accelerated) for relapsed/refractory MCL after ≥2 lines including a BTK inhibitor (BGB-11417-201: ORR 52%, median DOR 15.8 months). In treatment-naive CLL, sonrotoclax + zanubrutinib (BGB-11417-101) produced uMRD in nearly all patients by 96 weeks; phase 3 CELESTIAL-TNCLL (vs venetoclax-obinutuzumab) completes 2026. Shorter half-life allows faster ramp-up and less overlapping myelosuppression.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Sonrotoclax"}],"tags":[],"related":["venetoclax"],"cancers":["cll","dlbcl"],"sections":[],"technologies":["kinase-inhibitors","bcl2-inhibitors"],"targets":["bcl2"],"drugs":[],"companies":["beone"],"institutions":[],"pathways":["apoptosis-bcl2"],"terms":["tumor-lysis-syndrome"],"trials":["celestial-tncll","nct06742996","nct06697184","nct05471843","nct04973605","nct06943872","nct04771130","nct07277231","nct05479994","nct06634589","nct06637501","nct05952037"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Beqalzi","code":"BGB-11417","modality":"Small-molecule BCL-2 inhibitor (second generation)","mechanism":"Selective BH3-mimetic BCL-2 inhibitor with higher potency and shorter half-life than venetoclax; active against venetoclax-resistant G101V BCL2 in preclinical models.","approvals":[{"region":"China","year":2025,"indication":"Relapsed/refractory MCL and CLL/SLL after BTK inhibitor"},{"region":"US","year":2026,"indication":"Relapsed/refractory mantle cell lymphoma after ≥2 lines including a BTK inhibitor (accelerated)"}],"mechanismSteps":["Sonrotoclax occupies the BH3 groove of BCL-2","Sequestered BIM/BAX are released","Mitochondrial outer membrane permeabilises; caspases activate","Rapid apoptosis of CLL/MCL cells (TLS precautions during 4-week ramp)"],"dosing":{"route":"Oral","schedule":"4-week ramp-up then 320 mg once daily until progression (MCL label); CLL trials use 160-320 mg with zanubrutinib lead-in","monitoring":"TLS during ramp-up, neutropenia, infections","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sonrotoclax-relapsed-or-refractory-mantle-cell-lymphoma"},"toxicity":[{"event":"Neutropenia","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sonrotoclax-relapsed-or-refractory-mantle-cell-lymphoma","note":"Most common grade ≥3 event in MCL and CLL studies"},{"event":"Tumour lysis syndrome","note":"Mitigated by 4-week ramp"},{"event":"Diarrhoea, nausea","note":"Mostly low grade"}],"access":[],"regulatoryEvents":[{"date":"2026-05-13","type":"accelerated-approval","region":"US","note":"Accelerated approval in MCL; priority review, Project Orbis The confirmatory requirement was still open 0.3 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sonrotoclax-relapsed-or-refractory-mantle-cell-lymphoma","indication":"Treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after at least two lines of systemic therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor."}]},{"id":"soquelitinib","kind":"drug","name":"Soquelitinib","aka":[],"tldr":"Soquelitinib is an experimental small-molecule drug from Corvus Pharmaceuticals in phase 3 trials for peripheral T-cell lymphomas, with its target not yet stated publicly.","summary":"Soquelitinib (CPI-818) is a small-molecule drug developed by Corvus Pharmaceuticals. The sponsor describes its target as ITK (interleukin-2-inducible T-cell kinase), which OnCo does not yet have a target page for. The sponsor states: An oral, selective ITK inhibitor that promotes Th1 helper cell development while suppressing Th2 and Th17 cells, and can shift differentiation toward regulatory T cells. ClinicalTrials.gov describes the intervention as: Soquelitinib 200 mg tablets will be taken by mouth two times a day. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06561048 (Soquelitinib vs Standard of Care in Participants With Relapsed/Refractory Peripheral T-cell Lymphoma Not Otherwise Specified, Follicular Helper T-cell Lymphomas, or Systemic Anaplastic Large-cell Lymphoma), in peripheral T-cell lymphomas. The largest, NCT06561048, plans to enrol 150 participants with primary completion expected 2027-11. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Soquelitinib","url":"https://clinicaltrials.gov/search?intr=CPI-818"},{"label":"Sponsor pipeline page","url":"https://corvuspharma.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["peripheral-t-cell-lymphoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["corvus-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06561048"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CPI-818","modality":"small molecule","mechanism":"An oral, selective ITK inhibitor that promotes Th1 helper cell development while suppressing Th2 and Th17 cells, and can shift differentiation toward regulatory T cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sorafenib","kind":"drug","name":"Sorafenib","aka":[],"tldr":"The first drug ever to extend life in advanced liver cancer (2007), now mostly a comparator arm that newer combinations are measured against.","summary":"SHARP: OS 10.7 vs 7.9 months versus placebo (HR 0.69). Standard first-line therapy for a decade until REFLECT (lenvatinib non-inferior) and IMbrave150 (atezolizumab-bevacizumab superior). Also approved in RCC and radioiodine-refractory thyroid cancer. Hand-foot skin reaction and diarrhoea dominate toxicity.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Sorafenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Sorafenib"}],"tags":[],"related":[],"cancers":["hcc","hcc-advanced","rcc","thyroid","papillary-thyroid-cancer","follicular-thyroid-cancer"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf","braf"],"drugs":[],"companies":["bayer","natco"],"institutions":[],"pathways":[],"terms":[],"trials":["sharp","nct03755791","nct05822752"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Nexavar","modality":"Small-molecule multi-kinase inhibitor (VEGFR, PDGFR, RAF)","mechanism":"Oral inhibitor of VEGFR1-3, PDGFR-β, KIT, FLT3 and RAF kinases; anti-angiogenic and anti-proliferative.","approvals":[{"region":"US","year":2005,"indication":"Advanced RCC"},{"region":"US","year":2007,"indication":"Unresectable HCC"},{"region":"US","year":2013,"indication":"Radioiodine-refractory differentiated thyroid cancer"}],"mechanismSteps":["Enters tumour and endothelial cells","Blocks VEGFR/PDGFR signalling in vessels, cutting blood supply","Inhibits RAF-MEK-ERK in tumour cells","Tumour growth slows; rarely shrinks"],"dosing":{"route":"Oral","schedule":"400 mg twice daily continuously","modifications":"Dose reduction to 400 mg daily for grade 2-3 hand-foot skin reaction","monitoring":"Blood pressure, skin, liver function"},"toxicity":[{"event":"Diarrhoea","anyGradePct":39,"grade3PlusPct":8,"note":"SHARP"},{"event":"Hand-foot skin reaction","anyGradePct":21,"grade3PlusPct":8,"note":"SHARP"},{"event":"Fatigue","anyGradePct":22,"grade3PlusPct":4,"note":"SHARP"}],"access":[],"regulatoryEvents":[{"date":"2007-11-16","type":"approval","region":"US","note":"Unresectable HCC on SHARP"}]},{"id":"sotevtamab","kind":"drug","name":"Sotevtamab","aka":["AB-16B5"],"tldr":"Sotevtamab is a monoclonal antibody from Alethia Biotherapeutics, in registered phase 2 trials for colorectal cancer.","summary":"Sotevtamab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Alethia Biotherapeutics, in colorectal cancer. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Sotevtamab","url":"https://clinicaltrials.gov/search?intr=Sotevtamab"},{"label":"ClinicalTrials.gov NCT06225843","url":"https://clinicaltrials.gov/study/NCT06225843"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["alethia-biotherapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06225843"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Sotevtamab (AB-16B5) is described in its registry record as a fully humanised IgG2 monoclonal antibody against tumour-associated secreted clusterin, given as an inhibitor of the epithelial-to-mesenchymal transition; it is being tested with FOLFOX before resection of colorectal liver metastases. Clusterin has no target record in the corpus yet."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sotorasib","kind":"drug","name":"Sotorasib","aka":[],"tldr":"Sotorasib (Lumakras) was the first drug to hit KRAS, approved in 2021 after four decades of failure.","summary":"Sotorasib is a covalent inhibitor that binds cysteine 12 in the switch-II pocket of mutant KRAS, locking KRAS G12C in its inactive GDP-bound state; it was the first drug to hit KRAS, approved in 2021 after four decades of failure. CodeBreaK 100 gave a 37% response rate in previously treated NSCLC, CodeBreaK 200 showed a progression-free survival advantage over docetaxel, and CodeBreaK 300 with panitumumab in colorectal cancer gave PFS 5.6 versus 2.2 months (HR 0.49), leading to approval in January 2025. The label dose is 960 mg daily, with 240 mg an optional lower dose in NSCLC; hepatotoxicity (25%, 12% grade 3 or higher) is the main concern. Full approval in NSCLC still awaits confirmatory data, and resistance emerges faster than with EGFR or ALK drugs. For a newcomer: proof that the 'undruggable' KRAS could be drugged, with modest but real benefit.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Sotorasib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Sotorasib"},{"label":"Strickler et al., CodeBreaK 100 pancreatic cohort (NEJM 2023)","url":"https://doi.org/10.1056/NEJMoa2208470"},{"label":"Lumakras label (openFDA): indications","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22LUMAKRAS%22"},{"label":"FDA oncology approval notifications","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications"},{"label":"NICE TA781: sotorasib for previously treated KRAS G12C mutation-positive advanced non-small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta781"}],"tags":[],"related":["kras-g12c"],"cancers":["nsclc","colorectal","kras-g12c-colorectal","kras-g12c-nsclc","kras-g12c-pdac","pancreatic","lung-cancer"],"sections":[],"technologies":["kras-inhibitors"],"targets":["kras"],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":["cancer-drugs-fund"],"trials":["codebreak-300","nct05920356","nct06252649","nct07563738","nct05074810","nct07172919","codebreak-100","codebreak-200"],"people":[],"bottlenecks":[],"keyPapers":["paper-codebreak-300-nejm-2023","paper-codebreak-100-sotorasib-kras-g12c-pancreatic-nejm-2023","paper-schirripa-kras-g12c-metastatic-colorectal-clin-colorectal-cancer-2020"],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: the Lumakras label (effective 22 January 2025) covers KRAS G12C lung cancer and, with panitumumab, colorectal cancer, not pancreatic cancer; the EMA and NICE (TA781, lung cancer) likewise. The CodeBreaK 100 pancreatic cohort (38 patients: response 21 percent, median overall survival 6.9 months) is the basis of the NCCN listing for KRAS G12C pancreatic cancer after first-line therapy; daraxonrasib, approved for all previously treated metastatic pancreatic adenocarcinoma, covers G12C as well.","Pancreatic ductal adenocarcinoma: CodeBreaK 100 gave 8 confirmed responses among 38 previously treated KRAS G12C patients (21%), with progression-free survival 4.0 months and overall survival 6.9 months; G12C is only 1 to 2% of pancreatic cancers (Strickler 2023).","Colorectal cancer biomarkers: KRAS G12C, about 3% of colorectal cancers, and only in combination with panitumumab, because blocking G12C alone triggers adaptive EGFR reactivation in this tissue (CodeBreaK 300). G12C patients present more often with lung and liver metastases and have shorter survival than other KRAS-mutant patients (Schirripa 2020)."],"brand":"Lumakras","modality":"Small-molecule inhibitor (KRAS G12C)","mechanism":"Covalent binder to cysteine-12 in the switch-II pocket, locking KRAS G12C in the inactive GDP state.","approvals":[{"region":"US","year":2021,"indication":"KRAS G12C NSCLC, previously treated (accelerated)"},{"region":"US","year":2025,"indication":"KRAS G12C colorectal cancer with panitumumab"},{"region":"EU","year":2022,"indication":"EU brand Lumykras","note":"Conditional marketing authorisation"},{"region":"US","year":2025,"indication":"KRAS G12C-mutated metastatic colorectal cancer after fluoropyrimidine, oxaliplatin and irinotecan chemotherapy, with panitumumab","note":"Approved 16 January 2025 on CodeBreaK 300. CodeBreaK 301 is testing the first-line combination. No NICE recommendation for colorectal cancer at September 2026."},{"region":"England (NICE)","year":2022,"indication":"KRAS G12C mutation-positive locally advanced or metastatic non-small-cell lung cancer that has progressed on, or after intolerance of, platinum-based chemotherapy or PD-1/PD-L1 immunotherapy","note":"TA781, published 30 March 2022, recommends sotorasib for use within the Cancer Drugs Fund only, under a managed access agreement, because the clinical evidence was uncertain."}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of KRAS G12C","Phosphorylation of downstream substrates stops","Covalent bond to the mutant cysteine locks KRAS in the inactive GDP-bound state","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"960 mg once daily (240 mg daily is the label-optional lower dose in NSCLC); with panitumumab in colorectal cancer","modifications":"Hold for grade ≥3 hepatotoxicity; discontinue for ILD","monitoring":"LFTs every 3 weeks for 3 months then monthly; respiratory symptoms","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1"},"toxicity":[{"event":"Diarrhoea","anyGradePct":42,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1","note":"CodeBreaK 100"},{"event":"Musculoskeletal pain","anyGradePct":35,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1","note":"CodeBreaK 100"},{"event":"Nausea","anyGradePct":26,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1","note":"CodeBreaK 100"},{"event":"Fatigue","anyGradePct":26,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1","note":"CodeBreaK 100"},{"event":"Hepatotoxicity","anyGradePct":25,"grade3PlusPct":12,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1","note":"CodeBreaK 100"},{"event":"Cough","anyGradePct":20,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1","note":"CodeBreaK 100"},{"event":"Vomiting","anyGradePct":17,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1","note":"CodeBreaK 100"},{"event":"Interstitial lung disease","anyGradePct":2.2,"grade3PlusPct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c80a362c-7ac3-4894-a076-0691e68ef8c1","note":"CodeBreaK 100"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.amgensupportplus.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended via Cancer Drugs Fund for KRAS G12C NSCLC after platinum (TA781); later terminated then re-appraised","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2020-12","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2021-05-28","type":"accelerated-approval","region":"US","note":"Accelerated approval, KRAS G12C NSCLC after ≥1 therapy: first KRAS inhibitor The confirmatory requirement was still open 5.3 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy."},{"date":"2023-12","type":"crl","region":"US","note":"FDA declines full approval based on CodeBreaK 200; postmarketing dose study required","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-01-16","type":"approval","region":"US","note":"KRAS G12C colorectal cancer with panitumumab (CodeBreaK 300)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"spartalizumab","kind":"drug","name":"Spartalizumab","aka":["PDR001"],"tldr":"Spartalizumab is a monoclonal antibody from Novartis Pharmaceuticals, in registered phase 2 trials for myelodysplastic syndromes / neoplasms.","summary":"Spartalizumab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Novartis Pharmaceuticals, in myelodysplastic syndromes / neoplasms. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Spartalizumab","url":"https://clinicaltrials.gov/search?intr=Spartalizumab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["mds"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05201066"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"spevatamig","kind":"drug","name":"Spevatamig","aka":["Spevatamig (PT886)"],"tldr":"Spevatamig is an experimental bispecific antibody from Phanes Therapeutics in phase 2 trials for gastric & gastro-oesophageal junction cancer, pancreatic ductal adenocarcinoma and biliary tract cancer, aimed at Claudin 18.2 and CD47.","summary":"Spevatamig (PT886) is a bispecific antibody developed by Phanes Therapeutics. Its targets are Claudin 18.2 and CD47. ClinicalTrials.gov describes the intervention as: Spevatamig (PT886) monotherapy, a novel bispecific antibody that targets Claudin 18.2 and CD47. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in gastric & gastro-oesophageal junction cancer, pancreatic ductal adenocarcinoma and biliary tract cancer. The largest, NCT05482893, plans to enrol 258 participants with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Spevatamig","url":"https://clinicaltrials.gov/search?intr=PT886"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric","pancreatic","cholangiocarcinoma"],"sections":[],"technologies":[],"targets":["cldn18-2","cd47"],"drugs":[],"companies":["phanes-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05482893"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"PT886","modality":"bispecific antibody","mechanism":"Bispecific antibody directed at Claudin 18.2 and CD47, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sph4336","kind":"drug","name":"SPH4336","aka":[],"tldr":"SPH4336 is an experimental small-molecule drug from Shanghai Pharmaceuticals in phase 3 trials for HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"SPH4336 is a small-molecule drug developed by Shanghai Pharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SPH4336 Tablets ：Administered by oral; Letrozole tablets：Administered by oral; Fulvestrant injection：Administered by intravenous infusion; Exemestane：Administered by oral. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05744687 (Phase II/III Study of SPH4336 Combined With Letrozole vs Placebo Combined With Letrozole in First-line Treatment of Breast Cancer) and NCT05860465 (Safety and Efficacy of SPH4336 in Combination With Endocrine Therapy in the Treatment of Locally Advanced or Metastatic Breast Cancer), in HR-positive / HER2-negative breast cancer. The largest, NCT05744687, plans to enrol 374 participants with primary completion was scheduled for 2026-05-31 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SPH4336","url":"https://clinicaltrials.gov/search?intr=SPH4336"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["shanghai-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05744687","nct05860465","nct05944224","nct05872347"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ssgj-612","kind":"drug","name":"SSGJ-612","aka":[],"tldr":"SSGJ-612 is an experimental monoclonal antibody from Shenyang Sunshine Pharmaceutical in phase 2 trials, aimed at HER2.","summary":"SSGJ-612 is a monoclonal antibody developed by Shenyang Sunshine Pharmaceutical. Its target is HER2. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT07646626, plans to enrol 150 participants with primary completion expected 2027-06-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SSGJ-612","url":"https://clinicaltrials.gov/search?intr=SSGJ-612"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["3sbio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07646626"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at HER2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ssgj-705","kind":"drug","name":"SSGJ-705","aka":[],"tldr":"SSGJ-705 is an experimental monoclonal antibody from Shenyang Sunshine Pharmaceutical in phase 2 trials, aimed at PD-1 and HER2.","summary":"SSGJ-705 is a monoclonal antibody developed by Shenyang Sunshine Pharmaceutical. Its targets are PD-1 and HER2. ClinicalTrials.gov describes the intervention as: anti-PD-1 and anti-HER2 bispecifc antibody. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials. The largest, NCT07022002, plans to enrol 340 participants with primary completion expected 2026-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SSGJ-705","url":"https://clinicaltrials.gov/search?intr=SSGJ-705"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":["pd1","her2"],"drugs":[],"companies":["3sbio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07646626","nct07022002"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"Monoclonal antibody directed at PD-1 and HER2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ssgj-706","kind":"drug","name":"SSGJ-706","aka":[],"tldr":"SSGJ-706 is an experimental bispecific antibody from Shenyang Sunshine Pharmaceutical in phase 2 trials for non-small-cell lung cancer, aimed at PD-1 and PD-L1.","summary":"SSGJ-706 is a bispecific antibody developed by Shenyang Sunshine Pharmaceutical. Its targets are PD-1 and PD-L1. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT07171606, plans to enrol 240 participants with primary completion was scheduled for 2026-08-31 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SSGJ-706","url":"https://clinicaltrials.gov/search?intr=SSGJ-706"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["pd1","pdl1"],"drugs":[],"companies":["3sbio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07171606"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"Bispecific antibody directed at PD-1 and PD-L1, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sti-6129","kind":"drug","name":"STI-6129","aka":[],"tldr":"STI-6129 is an experimental antibody-drug conjugate from Zhejiang ACEA Pharmaceutical in phase 2 trials for multiple myeloma, aimed at CD38.","summary":"STI-6129 is an antibody-drug conjugate developed by Zhejiang ACEA Pharmaceutical. Its target is CD38 (the sponsor names CD38). The sponsor states: Anti-CD38 human antibody covalently bound to a duostatin tubulin inhibitor payload. ClinicalTrials.gov describes the intervention as: Anti-CD38 A2 human antibody drug conjugate (ADC) containing an antibody covalently bound to a duostatin tubulin inhibitor. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in multiple myeloma. The largest, NCT05565807, plans to enrol 84 participants with primary completion expected 2026-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of STI-6129","url":"https://clinicaltrials.gov/search?intr=STI-6129"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":[],"targets":["cd38"],"drugs":[],"companies":["sorrento-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05565807"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Anti-CD38 human antibody covalently bound to a duostatin tubulin inhibitor payload.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"stp938","kind":"drug","name":"STP938","aka":[],"tldr":"STP938 is a small-molecule inhibitor from Step Pharma, SAS, in registered phase 2 trials for non-Hodgkin lymphoma.","summary":"STP938 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Step Pharma, SAS, in non-Hodgkin lymphoma. Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of STP938","url":"https://clinicaltrials.gov/search?intr=STP938"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["non-hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["step-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05463263"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"STP938","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a small-molecule inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"streptozocin","kind":"drug","name":"Streptozocin","aka":["Streptozotocin"],"tldr":"Streptozocin is a chemotherapy drug that seeks out pancreatic islet cells, approved in 1982 for the rare tumours that arise from them; the same selectivity makes it the standard way to induce diabetes in laboratory animals.","summary":"Streptozocin, an antibiotic from Streptomyces achromogenes, carries a glucose group that lets islet cells take it up through GLUT2. The FDA approved it in 1982 for metastatic islet cell carcinoma of the pancreas, where it produced responses alone and with fluorouracil or doxorubicin in the Eastern Cooperative Oncology Group trials of the 1980s. Kidney toxicity is dose-limiting and nausea is severe. Streptozocin-based chemotherapy remains a guideline option for progressive pancreatic neuroendocrine tumours, though capecitabine with temozolomide and the targeted drugs everolimus and sunitinib are used more often, and it is the standard tool for creating diabetic animal models.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Streptozotocin","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=streptozocin"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Streptozotocin"},{"label":"ChEMBL CHEMBL1201294","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1201294"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["neuroendocrine","pancreatic","pancreatic-net"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":["fluorouracil","doxorubicin","temozolomide"],"companies":["nobelpharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00602082","nct00004688"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zanosar","modality":"Nitrosourea cytotoxic with glucose moiety, intravenous","mechanism":"A glucose-linked nitrosourea taken up by the GLUT2 transporter, which is why it homes to pancreatic islet cells; it methylates DNA and depletes NAD, killing the cells.","approvals":[{"region":"US","year":1982,"indication":"Metastatic islet cell carcinoma of the pancreas"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sugemalimab","kind":"drug","name":"Sugemalimab","aka":[],"tldr":"Sugemalimab is CStone's PD-L1 antibody, approved in China for first-line lung cancer with chemotherapy and after chemoradiotherapy in stage III disease, and the first China-developed PD-L1 antibody to win European approval.","summary":"GEMSTONE-302 (479 patients, squamous and non-squamous metastatic NSCLC) gave a progression-free survival hazard ratio of 0.50 versus placebo plus platinum chemotherapy, the basis of the NMPA approval in December 2021. GEMSTONE-301 added unresectable stage III NSCLC after concurrent or sequential chemoradiotherapy in 2022, and relapsed or refractory extranodal NK/T-cell lymphoma followed. The European Commission approved sugemalimab with platinum chemotherapy for first-line metastatic NSCLC without EGFR, ALK, ROS1 or RET alterations in July 2024, making it the first anti-PD-L1 antibody from a Chinese company approved in the EU.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Sugemalimab","links":[{"label":"CStone Pharmaceuticals","url":"https://www.cstonepharma.com/en/"},{"label":"GEMSTONE-302 (Lancet Oncol 2022)","url":"https://doi.org/10.1016/S1470-2045(21)00650-1"},{"label":"EMA: Cejemly","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/cejemly"}],"tags":["china"],"related":[],"cancers":["nsclc","peripheral-t-cell-lymphoma","lung-cancer","stage-iii-unresectable-nsclc"],"sections":[],"technologies":["checkpoint-inhibitor","monoclonal-antibody"],"targets":["pdl1"],"drugs":[],"companies":["cstone"],"institutions":[],"pathways":["pd1-checkpoint"],"terms":[],"trials":["gemstone-302","nct06617416","gemstone-301"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cejemly","code":"CS1001","modality":"Monoclonal antibody (anti-PD-L1, fully human IgG4)","mechanism":"Fully human anti-PD-L1 IgG4 antibody generated in the OmniRat platform; blocks PD-L1 binding to PD-1 and B7-1.","approvals":[{"region":"China","year":2021,"indication":"First-line metastatic squamous and non-squamous NSCLC with platinum chemotherapy (GEMSTONE-302)"},{"region":"China","year":2022,"indication":"Unresectable stage III NSCLC without progression after chemoradiotherapy (GEMSTONE-301)"},{"region":"EU","year":2024,"indication":"First-line metastatic NSCLC with platinum chemotherapy, no sensitising EGFR, ALK, ROS1 or RET alterations"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sunitinib","kind":"drug","name":"Sunitinib","aka":[],"tldr":"Sunitinib is an anti-angiogenic pill approved for pancreatic neuroendocrine tumours, kidney cancer and GIST.","summary":"Sunitinib is an oral inhibitor of VEGFR1-3, PDGFR, KIT, FLT3 and RET that blocks tumour blood-vessel growth and, in GIST, the KIT driver itself. It is approved for advanced renal cell carcinoma and imatinib-resistant GIST (2006), progressive pancreatic neuroendocrine tumours (2011, 37.5 mg daily continuously) and, little used, as adjuvant therapy in high-risk RCC (S-TRAC, 2017). The pancreatic NET phase 3 (Raymond 2011) showed PFS of 11.4 versus 5.5 months and was stopped early for benefit. In kidney cancer it has been largely displaced by immunotherapy combinations and is the control arm they beat in CheckMate 214, KEYNOTE-426, CheckMate 9ER and CLEAR, while it is still standard second line in GIST. Fatigue, diarrhoea, hand-foot syndrome, hypertension and hypothyroidism are common. For a newcomer, sunitinib defined first-line kidney cancer treatment before immunotherapy.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Sunitinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Sunitinib"}],"tags":[],"related":[],"cancers":["neuroendocrine","rcc","sarcoma","clear-cell-rcc","chromophobe-rcc","thymic-carcinoma","metastatic-ppgl"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf","kit"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05043090","nct05024214","nct03091192","nct03673501","checkmate-214","keynote-426","checkmate-9er","clear"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In kidney cancer: Motzer 2007 showed PFS 11 versus 5 months against interferon alfa and OS 26.4 versus 21.8 months, and sunitinib then served as the control arm of CheckMate 214, KEYNOTE-426, CheckMate 9ER and CLEAR; it is now mainly used in favourable-risk disease, where immunotherapy doublets show no OS gain, and in GIST, and has been generic since 2021. Resistance runs through alternative angiogenic pathways (FGF, MET, AXL)."],"brand":"Sutent","modality":"Small-molecule multi-kinase inhibitor (VEGFR, PDGFR, KIT)","mechanism":"Oral inhibitor of VEGFR1-3, PDGFR, KIT, FLT3, RET.","approvals":[{"region":"US","year":2006,"indication":"Advanced RCC; GIST after imatinib"},{"region":"US","year":2011,"indication":"Progressive pancreatic NETs"},{"region":"US","year":2017,"indication":"Adjuvant RCC at high risk (S-TRAC), little used"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"37.5 mg daily continuously (pNET)","monitoring":"Blood pressure, thyroid, cardiac function"},"toxicity":[{"event":"Diarrhoea","anyGradePct":59},{"event":"Nausea","anyGradePct":45},{"event":"Hypertension","anyGradePct":26,"grade3PlusPct":10},{"event":"Hand-foot syndrome","anyGradePct":23,"grade3PlusPct":6}],"access":[],"regulatoryEvents":[{"date":"2006-01-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Advanced renal cell carcinoma"},{"date":"2007-02-02","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2006 converted to traditional approval 1.0 year after it was granted.","indication":"Advanced renal cell carcinoma"}]},{"id":"sunvozertinib","kind":"drug","name":"Sunvozertinib","aka":[],"tldr":"An oral drug for EGFR exon 20 insertion lung cancer that succeeded where mobocertinib failed; approved in China (2023) and the US (2025).","summary":"Sunvozertinib is a covalent EGFR tyrosine kinase inhibitor selective for exon 20 insertions and other uncommon EGFR mutations, with wild-type sparing and CNS penetration. Developed by Dizal, it is used in locally advanced or metastatic NSCLC with EGFR exon 20 insertions after platinum chemotherapy. The WU-KONG1B study reported response rates of about 45 to 50% after platinum, including intracranial responses, and supported approval in China in 2023 and FDA accelerated approval in July 2025. The WU-KONG28 phase 3 trial versus platinum-pemetrexed in the first line has reported positive progression-free survival. Diarrhoea, rash and raised creatine phosphokinase are the main side effects. Sunvozertinib succeeded where mobocertinib failed and gives exon 20 patients an oral option alongside amivantamab.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Sunvozertinib","links":[{"label":"FDA novel approvals 2025","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025"}],"tags":["gap-fill"],"related":[],"cancers":["nsclc","egfr-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr"],"drugs":[],"companies":["dizal"],"institutions":[],"pathways":[],"terms":["egfr-exon20-insertion"],"trials":["nct07182682","nct05668988","nct05712902","nct03974022","nct07079475","nct06295432","nct06276283"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zegfrovy","code":"DZD9008","modality":"Small-molecule EGFR exon 20 insertion TKI (irreversible)","mechanism":"Covalent inhibitor selective for EGFR exon 20 insertions and other uncommon mutations with wild-type sparing and CNS penetration.","approvals":[{"region":"CN","year":2023,"indication":"EGFR exon 20 insertion NSCLC after platinum"},{"region":"US","year":2025,"indication":"Locally advanced/metastatic NSCLC with EGFR exon 20 insertions after platinum"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2025-07-02","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.2 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-sunvozertinib-metastatic-non-small-cell-lung-cancer-egfr-exon-20","indication":"Treatment of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test"}]},{"id":"surufatinib","kind":"drug","name":"Surufatinib","aka":["sulfatinib","Sulanda"],"tldr":"Surufatinib is HUTCHMED's angio-immuno kinase inhibitor, approved in China in 2020 for non-pancreatic and in 2021 for pancreatic neuroendocrine tumours.","summary":"Surufatinib (Sulanda) was discovered and developed by HUTCHMED in Shanghai. The NMPA approved it in December 2020 for unresectable locally advanced or metastatic, well-differentiated, non-pancreatic neuroendocrine tumours (SANET-ep trial) and in June 2021 for pancreatic neuroendocrine tumours (SANET-p trial). A US application was withdrawn in 2022 after the FDA asked for a multi-regional trial.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of surufatinib","url":"https://clinicaltrials.gov/search?intr=surufatinib"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["neuroendocrine"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["vegf","csf1r"],"drugs":[],"companies":["hutchmed"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"HMPL-012","modality":"Oral VEGFR, FGFR1 and CSF1R tyrosine kinase inhibitor","mechanism":"Blocks the VEGF and FGF receptors that build tumour blood vessels and the CSF1 receptor on tumour-associated macrophages, cutting the tumour's blood supply and its immune shelter at once.","approvals":[{"region":"CN","year":2020,"indication":"Advanced non-pancreatic neuroendocrine tumours; pancreatic neuroendocrine tumours from 2021"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"hypericin-sgx301","kind":"drug","name":"Synthetic hypericin","aka":["Hypericin","SGX301","Hypericin ointment"],"tldr":"HyBryte is an ointment containing synthetic hypericin, the active pigment of St John's wort. Rubbed on patches of cutaneous T-cell lymphoma and switched on with ordinary visible light, it kills the lymphoma cells in the skin without ultraviolet light.","summary":"Synthetic hypericin (SGX301, HyBryte) is Soligenix's photodynamic therapy for early-stage cutaneous T-cell lymphoma (mycosis fungoides). The ointment is applied to lesions and, after a delay, the area is exposed to visible fluorescent light twice a week. Because activation uses visible rather than ultraviolet light, the treatment avoids the skin-cancer and ageing risks of long-term PUVA and narrow-band UVB.\n\nThe phase 3 FLASH trial reported in 2020 that significantly more patients had a treatment response after the first 6-week cycle than with placebo ointment, and responses increased with longer treatment. The FDA asked for a second confirmatory study before approval, and the confirmatory FLASH2 trial is under way. The cutaneous T-cell lymphoma page names it as the skin-directed treatment closest to approval.","status":"phase-3","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Hypericin","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Hypericin"}],"tags":["subtype-drugs-wave"],"related":[],"cancers":["cutaneous-t-cell-lymphoma"],"sections":[],"technologies":["photodynamic-therapy-lasers"],"targets":[],"drugs":[],"companies":["soligenix"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02448381","nct06470451","nct06149247"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"HyBryte","code":"SGX301","modality":"Topical photosensitiser activated by visible light","mechanism":"Hypericin, the pigment of St John's wort made synthetically, is applied to skin lesions and taken up by malignant T cells; visible light then activates it to generate reactive oxygen that kills the cells, without the ultraviolet exposure of PUVA.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sys6010","kind":"drug","name":"SYS6010","aka":[],"tldr":"SYS6010 is an experimental investigational agent whose form is not stated in the registry from CSPC Megalith Biopharmaceutical in phase 3 trials for non-small-cell lung cancer and head and neck squamous cell carcinoma, with its target not yet stated publicly.","summary":"SYS6010 is an investigational agent whose form is not stated in the registry developed by CSPC Megalith Biopharmaceutical and CSPC ZhongQi Pharmaceutical Technology. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: SYS6010will be administrated on a 14-day cycle. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06927986 (Phase III Trial of SYS6010 Versus Platinum-based Chemotherapy for EGFR-mutated NSCLC（SYNSTAR01）), in non-small-cell lung cancer and head and neck squamous cell carcinoma. The largest, NCT07241936, plans to enrol 444 participants with primary completion expected 2027-10-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SYS6010","url":"https://clinicaltrials.gov/search?intr=SYS6010"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06927986","nct07254585","nct07241936"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC), per the sponsor's pipeline page; form not stated in the registry record","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"sys6023","kind":"drug","name":"SYS6023","aka":[],"tldr":"SYS6023 is an experimental antibody-drug conjugate from CSPC Megalith Biopharmaceutical in phase 2 trials for HR-positive / HER2-negative breast cancer, aimed at HER3.","summary":"SYS6023 is an antibody-drug conjugate developed by CSPC Megalith Biopharmaceutical. Its target is HER3 (the sponsor names HER3). The sponsor states: Antibody-drug conjugate targeting HER3, administered by intravenous infusion. ClinicalTrials.gov describes the intervention as: SYS6023 is a novel antibody-drug conjugate (ADC) targeting HER3, administered via intravenous infusion. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in HR-positive / HER2-negative breast cancer. The largest, NCT07597629, plans to enrol 36 participants with primary completion expected 2027-11-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of SYS6023","url":"https://clinicaltrials.gov/search?intr=SYS6023"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":["her3"],"drugs":[],"companies":["cspc"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07597629"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Antibody-drug conjugate targeting HER3, administered by intravenous infusion.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"t3011","kind":"drug","name":"T3011","aka":[],"tldr":"T3011 is an experimental small-molecule drug from ImmVira Pharma in phase 2 trials for melanoma, head and neck squamous cell carcinoma and sarcomas, aimed at PD-1 and PD-L1.","summary":"T3011 is a small-molecule drug developed by ImmVira Pharma and Shanghai Pharmaceuticals. Its targets are PD-1 and PD-L1. ClinicalTrials.gov describes the intervention as: T3011 will be administered up to 4mL as an intratumoral injection given Q2W. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in melanoma, head and neck squamous cell carcinoma, sarcomas, non-small-cell lung cancer and bladder & urothelial cancer. The largest, NCT06971614, plans to enrol 160 participants with primary completion expected 2028-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of T3011","url":"https://clinicaltrials.gov/search?intr=T3011"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma","head-and-neck","sarcoma","nsclc","urothelial"],"sections":[],"technologies":[],"targets":["pd1","pdl1"],"drugs":[],"companies":["immvira-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"oncolytic virus (intratumoral oncolytic HSV-1, per the sponsor's pipeline page; registered as a drug on ClinicalTrials.gov)","mechanism":"Small-molecule drug directed at PD-1 and PD-L1, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tabelecleucel","kind":"drug","name":"Tabelecleucel","aka":["Ebvallo","ATA129","tab-cel"],"tldr":"Tabelecleucel is a bank of donor T cells trained against Epstein-Barr virus, approved in Europe in 2022 as the first off-the-shelf T-cell therapy, for EBV-driven lymphoma after transplant when rituximab has failed.","summary":"Atara Biotherapeutics built tabelecleucel from EBV-specific T-cell lines of healthy donors, selected for each patient by shared HLA alleles, so no manufacturing from the patient's own cells is needed. The European Commission approved it in December 2022 as Ebvallo for relapsed or refractory EBV-positive post-transplant lymphoproliferative disease after at least one prior therapy, the first allogeneic T-cell immunotherapy approved anywhere; the FDA issued a complete response letter in 2024 over manufacturing questions. Pierre Fabre markets it and runs trials in other EBV-associated diseases.","status":"approved","asOf":"2026-09-16","wikipedia":"https://en.wikipedia.org/wiki/Tabelecleucel","links":[{"label":"ClinicalTrials.gov: trials of Tabelecleucel","url":"https://clinicaltrials.gov/search?intr=Tabelecleucel"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["post-transplant-lymphoproliferative-disorder"],"sections":[],"technologies":["virus-specific-t-cells"],"targets":[],"drugs":[],"companies":["atara-biotherapeutics","pierre-fabre"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03394365","nct04554914"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"modality":"Cell therapy: allogeneic off-the-shelf Epstein-Barr virus-specific T cells","mechanism":"T cells from healthy donors expanded against Epstein-Barr virus antigens and matched to the patient by HLA restriction; infused, they kill EBV-infected lymphoma cells.","approvals":[{"region":"EU","year":2022,"indication":"Relapsed or refractory EBV-positive post-transplant lymphoproliferative disease after at least one prior therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tac01-cldn18-2","kind":"drug","name":"TAC01-CLDN18.2","aka":[],"tldr":"TAC01-CLDN18.2 is an experimental TCR-T cell therapy from Triumvira Immunologics in phase 2 trials, aimed at Claudin 18.2.","summary":"TAC01-CLDN18.2 (TAC01) is a TCR-T cell therapy developed by Triumvira Immunologics. Its target is Claudin 18.2 (the sponsor names Claudin 18.2 (CLDN18.2)). The sponsor states: A genetically engineered autologous T-cell therapy using a T-cell receptor Antigen Coupler (TAC) that directs T cells to CLDN18.2-expressing tumour cells and, once engaged, activates the T cells via the endogenous T-cell receptor. ClinicalTrials.gov describes the intervention as: TAC01-CLDN18.2 preceded by lymphodepletion with fludarabine or clofarabine, cyclophosphamide, and nab-paclitaxel. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial. The largest, NCT05862324, plans to enrol 113 participants with primary completion expected 2027-08-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TAC01-CLDN18.2","url":"https://clinicaltrials.gov/search?intr=TAC01"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":["tcr-t"],"targets":["cldn18-2"],"drugs":[],"companies":["triumvira-immunologics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05862324"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"TAC01","modality":"TCR-T cell therapy","mechanism":"A genetically engineered autologous T-cell therapy using a T-cell receptor Antigen Coupler (TAC) that directs T cells to CLDN18.2-expressing tumour cells and, once engaged, activates the T cells via the endogenous T-cell receptor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tacquell","kind":"drug","name":"Tacquell (autologous melanoma-derived tumour-infiltrating lymphocytes)","aka":["Autologous melanoma-derived tumor infiltrating lymphocytes, ex vivo-expanded"],"tldr":"Tacquell is a tumour-infiltrating lymphocyte therapy for melanoma developed by the Netherlands Cancer Institute, the academic group whose randomised trial first showed TIL therapy could beat ipilimumab. Europe's medicines committee gave it a negative opinion in June 2026, so it is not authorised.","summary":"Tacquell (EMA product number EMEA/H/C/006563; marketing authorisation applicant Netherlands Cancer Institute) is described in the EMA register as autologous melanoma-derived tumour-infiltrating lymphocytes, ex vivo expanded, for the treatment of melanoma. The CHMP adopted its opinion on 25 June 2026 and the opinion status on the product page is negative, so there is no EU marketing authorisation. It is an academic TIL product; the linked key paper is the Netherlands Cancer Institute's randomised phase 3 trial of TIL therapy against ipilimumab (NCT02278887). The only other TIL product with a regulatory history in the corpus is lifileucel (Amtagvi), whose EU application was withdrawn in 2025.","asOf":"2026-09-22","links":[{"label":"EMA: Tacquell (EPAR)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/tacquell"}],"tags":["ema-register"],"related":["lifileucel","ipilimumab"],"cancers":["melanoma","advanced-melanoma"],"sections":[],"technologies":["til-therapy"],"targets":[],"drugs":[],"companies":[],"institutions":["nki"],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-rohaas-til-vs-ipilimumab-nejm-2022"],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo content wave 5 (EMA medicines register and EPAR pages)","editedOn":"2026-09-22"},"brand":"Tacquell","modality":"TIL cell therapy (autologous tumour-infiltrating lymphocytes, ex vivo expanded)","mechanism":"A patient's own tumour-infiltrating lymphocytes are taken from a melanoma deposit, expanded outside the body and returned; the EMA's committee adopted a negative opinion on the application for melanoma (EMA product page).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2026-06-25","type":"crl","region":"EU","note":"CHMP negative opinion on Tacquell (autologous TIL) for melanoma; applicant Netherlands Cancer Institute","source":"https://www.ema.europa.eu/en/medicines/human/EPAR/tacquell"}]},{"id":"tafasitamab","kind":"drug","name":"Tafasitamab","aka":[],"tldr":"A CD19 antibody given with lenalidomide for lymphoma patients who cannot have a transplant; in 2026 it showed the first frontline gain over R-CHOP in high-risk disease.","summary":"L-MIND (single arm, with lenalidomide): ORR 60%, CR 43%, median DOR ~44 months; accelerated approval July 2020 for transplant-ineligible R/R DLBCL. frontMIND phase 3 (Lancet 2026): tafasitamab + lenalidomide + R-CHOP vs placebo + R-CHOP in IPI 3-5 DLBCL; 2-year PFS 71.1% vs 62.9%, HR 0.75; OS immature (HR 0.85). Also approved 2025 with lenalidomide-rituximab in follicular lymphoma (inMIND).","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Tafasitamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tafasitamab"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd19"],"drugs":["lenalidomide"],"companies":["incyte","morphosys"],"institutions":[],"pathways":[],"terms":["adcc"],"trials":["l-mind","frontmind","nct05429268","nct04680052","nct05222555","nct03930953","nct06465433"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Monjuvi","modality":"Monoclonal antibody (anti-CD19, Fc-enhanced)","mechanism":"Fc-engineered (XmAb) anti-CD19 antibody with enhanced ADCC/ADCP; lenalidomide augments NK activity.","approvals":[{"region":"US","year":2020,"indication":"R/R DLBCL with lenalidomide, transplant-ineligible (accelerated)"},{"region":"US","year":2025,"indication":"R/R follicular lymphoma with lenalidomide and rituximab"},{"region":"EU","year":2021,"indication":"EU brand Minjuvi","note":"Conditional marketing authorisation"}],"mechanismSteps":[],"dosing":{"route":"IV","schedule":"12 mg/kg days 1, 8, 15, 22 of cycles 1-3, then days 1 and 15 from cycle 4; lenalidomide 25 mg days 1-21 for up to 12 cycles, tafasitamab until progression","monitoring":"Cytopenias, infections, infusion reactions"},"toxicity":[{"event":"Neutropenia","grade3PlusPct":48,"note":"L-MIND, with lenalidomide"},{"event":"Thrombocytopenia","grade3PlusPct":17},{"event":"Febrile neutropenia","grade3PlusPct":12}],"access":[],"regulatoryEvents":[{"date":"2020-07-31","type":"accelerated-approval","region":"US","note":"L-MIND accelerated approval The confirmatory requirement was still open 6.1 years later, when the FDA's table was read.","indication":"In combination with lenalidomide for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) not otherwise specified, including DLBCL arising from low grade lymphoma, and who are not eligible for autologous stem cell transplant (ASCT)."},{"date":"2026-03","type":"filing","region":"US","note":"sBLA for frontline high-risk DLBCL (frontMIND) planned H1 2026","source":"https://www.onclive.com/view/frontmind-results-tafasitamab-based-combo-yields-significant-pfs-benefit-over-r-chop-in-frontline-high-risk-dlbcl"}]},{"id":"tagraxofusp","kind":"drug","name":"Tagraxofusp","aka":[],"tldr":"Tagraxofusp is a fusion of the growth factor IL-3 with a bacterial toxin that homes to CD123 on a rare, aggressive blood cancer.","summary":"Approved 2018 for blastic plasmacytoid dendritic cell neoplasm (BPDCN), the first CD123-directed therapy (CR/CRc 57% in untreated patients). Capillary leak syndrome is the boxed warning. Under study in CD123+ AML and CMML, usually in combination. Now joined in BPDCN by the CD123 ADC pivekimab sunirine (2026).","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Tagraxofusp","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tagraxofusp"}],"tags":[],"related":[],"cancers":["aml"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["cd123"],"drugs":[],"companies":["menarini"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06456463"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Elzonris","modality":"Recombinant cytotoxin (IL-3 fused to diphtheria toxin)","mechanism":"IL-3 domain binds CD123 (IL-3Rα); internalised diphtheria toxin fragment inhibits protein synthesis (ADP-ribosylation of EF-2).","approvals":[{"region":"US","year":2018,"indication":"Blastic plasmacytoid dendritic cell neoplasm, adults and children ≥2"}],"mechanismSteps":["IL-3 moiety binds CD123 on the blast","Receptor-mediated endocytosis internalises the fusion protein","Diphtheria toxin catalytic domain escapes to the cytosol","Elongation factor 2 is inactivated; protein synthesis stops; the cell dies"],"dosing":{"route":"IV over 15 minutes","schedule":"12 µg/kg daily on days 1-5 of a 21-day cycle; first cycle inpatient","monitoring":"Capillary leak syndrome (boxed warning): albumin, weight, oedema; hepatic enzymes","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Elzonris"},"toxicity":[{"event":"Capillary leak syndrome","anyGradePct":55,"grade3PlusPct":9,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Elzonris","note":"Boxed warning"},{"event":"Hypoalbuminaemia","anyGradePct":51},{"event":"Transaminase elevation","anyGradePct":60}],"access":[],"regulatoryEvents":[]},{"id":"tak-928","kind":"drug","name":"TAK-928","aka":[],"tldr":"TAK-928 is an experimental bispecific antibody from Takeda in phase 3 trials for non-small-cell lung cancer, aimed at PD-1 and CD25 (IL-2 receptor alpha).","summary":"TAK-928 is a bispecific antibody developed by Takeda. Its targets are PD-1 and CD25 (IL-2 receptor alpha) (the sponsor names PD-1 x IL-2 receptor (CD25)). The sponsor states: Described as a first-in-class bispecific monoclonal antibody comprised of an IL-2 mutein fused with a recombinant anti-PD-1 antibody, intended to simultaneously block PD-(L)1 signalling and deliver IL-2 stimulation to tumour-specific CD8+ T cells co-expressing PD-1 and CD25, to reverse T-cell exhaustion. ClinicalTrials.gov describes the intervention as: TAK-928 will be administered by IV infusion. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07217301 (A Study Comparing TAK-928 With Docetaxel in Adults With Non-Small Cell Lung Cancer), in non-small-cell lung cancer. The largest, NCT07217301, plans to enrol 600 participants with primary completion expected 2028-11-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TAK-928","url":"https://clinicaltrials.gov/search?intr=TAK-928"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["pd1","cd25"],"drugs":[],"companies":["takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07217301"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"Described as a first-in-class bispecific monoclonal antibody comprised of an IL-2 mutein fused with a recombinant anti-PD-1 antibody, intended to simultaneously block PD-(L)1 signalling and deliver IL-2 stimulation to tumour-specific CD8+ T cells co-expressing PD-1 and CD25, to reverse T-cell exhaustion.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"talazoparib","kind":"drug","name":"Talazoparib","aka":[],"tldr":"Talazoparib is a PARP inhibitor that traps PARP on DNA about 100 times more strongly than olaparib, which is why it works at a 1 mg daily dose. It is approved for germline BRCA-mutant HER2-negative breast cancer and, with enzalutamide, for HRR-mutant castration-resistant prostate cancer; anaemia is its dominant side effect.","summary":"Talazoparib is a PARP inhibitor with about 100 times greater PARP trapping potency than olaparib, which lets it work at a 1 mg daily dose. It was approved in 2018 for germline BRCA-mutant HER2-negative locally advanced or metastatic breast cancer on EMBRACA, and in 2023 with enzalutamide for HRR-mutant metastatic castration-resistant prostate cancer on TALAPRO-2, where an overall survival benefit was reported in 2024-25; the prostate dose is 0.5 mg daily. Pfizer markets it. Anaemia is the dominant toxicity, with fatigue, nausea, neutropenia and thrombocytopenia also reported. TALAPRO-2 enrolled patients regardless of HRR status but the US label was limited to HRR-mutant disease, so use in unselected prostate cancer remains contested. For a newcomer: the most potent PARP trapper, used in BRCA breast cancer and alongside hormone therapy in prostate cancer.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Talazoparib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Talazoparib"},{"label":"NICE TA952: talazoparib for HER2-negative advanced breast cancer with germline BRCA mutations (21 February 2024)","url":"https://www.nice.org.uk/guidance/ta952"},{"label":"Talzenna label (openFDA): EMBRACA study section","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22TALZENNA%22"},{"label":"NICE TA1130: talazoparib with enzalutamide for untreated hormone-relapsed metastatic prostate cancer","url":"https://www.nice.org.uk/guidance/ta1130"}],"tags":[],"related":["brca-germline","brca-somatic"],"cancers":["tnbc","breast-hr-positive","prostate","prostate-mcrpc","tnbc-metastatic"],"sections":[],"technologies":["parp-inhibitor"],"targets":["parp","brca"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":["prostate-hrr-eligibility","prostate-uk-drug-approvals"],"trials":["nct04821622"],"people":[],"bottlenecks":[],"keyPapers":["paper-couch-tnbc-germline-17-genes-jco-2015"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: 44 percent of EMBRACA patients had triple-negative disease and randomisation was stratified by it (Talzenna label). Final overall survival did not differ (hazard ratio 0.85, medians 19.3 versus 19.5 months); 32.6 percent of the chemotherapy arm later received a PARP inhibitor. Six UK sites took part.","NICE TA1130 recommends talazoparib with enzalutamide for untreated hormone-relapsed metastatic prostate cancer only when chemotherapy is not clinically indicated and abiraterone with prednisolone is not tolerated or is clinically precluded, with the companies' commercial arrangements. It must be funded in England within 90 days of publication."],"brand":"Talzenna","modality":"Small-molecule PARP inhibitor","mechanism":"PARP inhibitor with ~100x greater trapping than olaparib.","approvals":[{"region":"US","year":2018,"indication":"gBRCA HER2- locally advanced/metastatic breast cancer"},{"region":"US","year":2023,"indication":"HRR-mutant mCRPC with enzalutamide"},{"region":"EU","year":2019,"indication":"Germline BRCA1/2-mutated HER2-negative locally advanced or metastatic breast cancer (EMBRACA); marketing authorisation issued 20 June 2019","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/talzenna"},{"region":"UK","year":2024,"indication":"HER2-negative locally advanced or metastatic breast cancer with germline BRCA1 or BRCA2 mutations after an anthracycline or a taxane; NICE TA952 (21 February 2024) recommends within the marketing authorisation with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta952"}],"mechanismSteps":["Talazoparib traps PARP1 on DNA about 100-fold more potently than olaparib","Trapped PARP-DNA complexes block replication","Double-strand breaks accumulate in HR-deficient tumour cells","Synthetic lethality kills the tumour; anaemia is the dose-limiting toxicity"],"dosing":{"route":"Oral","schedule":"1 mg once daily (breast); 0.5 mg once daily with enzalutamide (prostate)","modifications":"0.75 mg for moderate renal impairment; hold for grade ≥3 cytopenias","monitoring":"Blood counts monthly","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Anaemia","note":"EMBRACA: 53% any grade, 39% grade 3-4"},{"event":"Fatigue"},{"event":"Nausea"},{"event":"Neutropenia"},{"event":"Thrombocytopenia"},{"event":"Headache"},{"event":"Alopecia"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.pfizeroncologytogether.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for gBRCA HER2-negative advanced breast cancer (TA952) and with enzalutamide in HRR-mutant mCRPC (2024)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2018-10-16","type":"approval","region":"US","note":"gBRCA HER2-negative locally advanced/metastatic breast cancer (EMBRACA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-06-20","type":"approval","region":"US","note":"HRR-mutant mCRPC with enzalutamide (TALAPRO-2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"talc-sclerosant","kind":"drug","name":"Talc (intrapleural)","aka":["Talc slurry","Talc poudrage"],"tldr":"Sterile talc is puffed or instilled into the chest cavity after fluid is drained to make the lung stick to the chest wall, stopping cancer-related fluid (malignant pleural effusion) from building up again.","summary":"Sclerosol intrapleural aerosol was approved by the FDA in December 1997 and sterile talc powder in 2003 to decrease the recurrence of malignant pleural effusion in symptomatic patients after drainage; talc has been the most effective pleurodesis agent in comparative trials and meta-analyses for decades. Poudrage at thoracoscopy and bedside slurry through a chest drain give similar success (TAPPS trial), and indwelling pleural catheters, with or without talc instilled through them (IPC-PLUS), are the main alternative for patients with trapped lung or short prognosis. Graded, large-particle talc is used to avoid the acute respiratory distress syndrome reported with small-particle preparations. Fever and chest pain after the procedure are expected.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Pleurodesis","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=talc"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/talc"}],"tags":["nci-list","supportive"],"related":[],"cancers":["nsclc","breast-hr-positive","mesothelioma","ovarian"],"sections":[],"technologies":["palliative-care"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct00002622","nct00002872","nct00821860"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Sclerosol / Sterile Talc Powder / Steritalc","modality":"Sclerosing agent (intrapleural powder or aerosol)","supportive":true,"mechanism":"Sterile asbestos-free magnesium silicate that provokes an inflammatory pleuritis and fibrosis, sealing the visceral and parietal pleura together so that fluid can no longer accumulate.","approvals":[{"region":"US","year":1997,"indication":"Prevention of recurrence of malignant pleural effusion after drainage (Sclerosol aerosol; sterile powder 2003)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"taletrectinib","kind":"drug","name":"Taletrectinib","aka":[],"tldr":"A ROS1 lung-cancer pill approved in 2025 that works in the brain and after crizotinib, with fewer dizziness-type side effects than repotrectinib.","summary":"Taletrectinib is a selective, CNS-penetrant ROS1 tyrosine kinase inhibitor active against G2032R and other solvent-front mutations that cause resistance to crizotinib. It has limited TRK inhibition, which means fewer of the dizziness and neurological effects seen with repotrectinib. In the TRUST-I (China) and TRUST-II (global) studies, response rates were about 85 to 90% in TKI-naive patients and 50 to 60% after crizotinib, with intracranial responses and activity against G2032R. China approved it in January 2025 and the FDA in June 2025 for locally advanced or metastatic ROS1-positive NSCLC, developed by Nuvation Bio; hepatotoxicity and gastrointestinal effects are the main toxicities. Sequencing against crizotinib, entrectinib and repotrectinib is not settled. In short, taletrectinib is a ROS1 pill that works in the brain and after resistance, with fewer neurological side effects.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Taletrectinib","links":[{"label":"FDA novel approvals 2025","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025"}],"tags":["gap-fill"],"related":["ros1-fusion"],"cancers":["nsclc","ros1-positive-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["ros1"],"drugs":[],"companies":["nuvation-bio","innovent"],"institutions":[],"pathways":[],"terms":["gene-fusion"],"trials":["nct07154706","nct06564324","nct04919811"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ibtrozi","modality":"Small-molecule ROS1 TKI (next generation, CNS-penetrant)","mechanism":"Selective ROS1 inhibitor active against G2032R and other solvent-front mutations with limited TRK inhibition (fewer neurologic effects).","approvals":[{"region":"CN","year":2025,"indication":"ROS1-positive NSCLC"},{"region":"US","year":2025,"indication":"Locally advanced or metastatic ROS1-positive NSCLC"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"talicabtagene-autoleucel","kind":"drug","name":"Talicabtagene autoleucel","aka":["tali-cel","actalycabtagene autoleucel (earlier name)","HCAR19"],"tldr":"India's first home-grown CAR-T cell therapy, approved in 2023 for relapsed leukaemia and lymphoma, made in Mumbai for roughly a tenth of what the same kind of treatment costs in the US.","summary":"Talicabtagene autoleucel (NexCAR19) is an autologous CD19-directed CAR-T therapy with a humanised binder developed at IIT Bombay (Rahul Purwar) with Tata Memorial Centre and manufactured by ImmunoACT in Mumbai. The open-label phase 1/2 study enrolled 64 patients (14 in phase 1, 50 in phase 2) with relapsed or refractory B-cell lymphoma or B-cell acute lymphoblastic leukaemia at six Indian centres; among 51 evaluable patients the overall response rate was 73% (37 of 51; 95% CI 59-83), grade 3 or worse neutropenia occurred in 96%, and there were two treatment-related deaths. The DCGI granted market authorisation in 2023, the first CAR-T approval in India and one of the first in any lower-middle-income country.\n\nThe product's significance is economic as much as clinical: ImmunoACT reports more than 600 patients treated at over 130 centres, a 98% manufacturing success rate and about 20 days from apheresis to infusion, at a price reported to be around a tenth of that of US products. The company's real-world series and a 2026 distribution deal with Cipla for Africa make it the test case for whether autologous cell therapy can reach middle-income countries at scale.","status":"approved","asOf":"2026-09-10","links":[{"label":"ImmunoACT: NexCAR19","url":"https://immunoact.com"},{"label":"Phase 1/2 study (Lancet Haematol 2025)","url":"https://doi.org/10.1016/S2352-3026(24)00377-6"},{"label":"Commentary: CAR T-cell therapy in LMICs (Lancet Haematol 2025)","url":"https://doi.org/10.1016/S2352-3026(25)00040-7"}],"tags":[],"related":["tisagenlecleucel","idea-reg-lmic-public-cart-manufacturing"],"cancers":["dlbcl","all-leukemia"],"sections":[],"technologies":["car-t","point-of-care-cell-manufacturing"],"targets":["cd19"],"drugs":[],"companies":["immunoact","cipla"],"institutions":["tata-memorial","actrec","iit-bombay"],"pathways":[],"terms":["crs","icans"],"trials":["talicel-phase-1-2"],"people":["purwar-rahul","jain-hasmukh","narula-gaurav"],"bottlenecks":["b-manufacturing-cell-therapy","b-global-access","b-drug-pricing"],"keyPapers":["paper-jain-talicabtagene-lancet-haem-2025"],"journals":[],"dependsOn":[],"notes":[],"brand":"NexCAR19","code":"NexCAR19","modality":"CAR-T (CD19, humanised)","mechanism":"Autologous T cells transduced with a lentiviral humanised anti-CD19 scFv CAR with 4-1BB costimulation and CD3-zeta signalling; expand in vivo and kill CD19-positive B cells after lymphodepletion.","approvals":[{"region":"IN","year":2023,"indication":"Relapsed or refractory B-cell lymphoma and B-cell acute lymphoblastic leukaemia","note":"DCGI market authorisation; first CAR-T approved in India"}],"mechanismSteps":["Leukapheresis collects the patient's T cells at an authorised centre","Cells are shipped to Mumbai, transduced with the humanised CD19 CAR and expanded (about 20 days end to end)","Lymphodepleting chemotherapy makes room for the infused cells","CAR-T cells find CD19 on lymphoma or leukaemia cells and kill them; cytokine release and neurotoxicity are watched for","B-cell aplasia persists while the cells remain active"],"dosing":{"route":"Single IV infusion after lymphodepletion","schedule":"Phase 2 dose at least 5 x 10^6 CAR-T cells per kg (up to 2 x 10^9 cells)","monitoring":"Cytokine release syndrome, neurotoxicity, prolonged cytopenias, infection, haemophagocytic lymphohistiocytosis-like syndrome","source":"https://doi.org/10.1016/S2352-3026(24)00377-6"},"toxicity":[{"event":"Neutropenia (grade 3 or worse)","grade3PlusPct":96,"source":"https://doi.org/10.1016/S2352-3026(24)00377-6","note":"Phase 1/2, 64 patients"},{"event":"Thrombocytopenia (grade 3 or worse)","grade3PlusPct":65,"source":"https://doi.org/10.1016/S2352-3026(24)00377-6"},{"event":"Anaemia (grade 3 or worse)","grade3PlusPct":61,"source":"https://doi.org/10.1016/S2352-3026(24)00377-6"}],"access":[{"country":"IN","listPrice":"Priced by the developer at a small fraction of US CAR-T prices; no figure is published on the linked page","reimbursement":"Public insurance cover not established; ask the treating centre about state and charitable funding","generic":false,"source":"https://immunoact.com","asOf":"2026-09-10"}],"regulatoryEvents":[{"date":"2023","type":"approval","region":"IN","note":"CDSCO/DCGI market authorisation; the developer's page gives the year only","source":"https://immunoact.com"}]},{"id":"talimogene-laherparepvec","kind":"drug","name":"Talimogene laherparepvec","aka":[],"tldr":"Talimogene laherparepvec (T-VEC, Imlygic) was the first approved oncolytic virus (2015), injected into melanoma skin lesions.","summary":"Talimogene laherparepvec (T-VEC) is a herpes simplex virus type 1 with ICP34.5 and ICP47 deleted, so it replicates in tumour cells but not healthy ones, and it expresses GM-CSF to attract antigen-presenting cells. It was the first approved oncolytic virus (2015), injected into unresectable melanoma lesions at 10^6 PFU/mL initially, then 10^8 PFU/mL 3 weeks later and every 2 weeks. In OPTiM the durable response rate was 16% versus 2% for GM-CSF alone. Uptake has been modest because responses concentrate in injected lesions, and the MASTERKEY-265 combination with pembrolizumab did not improve progression-free or overall survival. Amgen markets it; its legacy is proving the principle that later viruses build on. For a newcomer: the first cancer-killing virus approved, useful mainly for accessible skin lesions.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Talimogene_laherparepvec","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Talimogene%20laherparepvec"}],"tags":[],"related":[],"cancers":["melanoma","advanced-melanoma","stage-iii-melanoma"],"sections":[],"technologies":["oncolytic-virus"],"targets":[],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02263508","nct00769704","nct01368276"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Imlygic","code":"T-VEC","modality":"Oncolytic virus (HSV-1)","mechanism":"HSV-1 with ICP34.5 and ICP47 deleted, expressing GM-CSF.","approvals":[{"region":"US","year":2015,"indication":"Unresectable melanoma with injectable lesions"}],"mechanismSteps":["HSV-1 with ICP34.5 and ICP47 deleted is injected into melanoma lesions","Replicates in tumour cells and lyses them","GM-CSF expression attracts antigen-presenting cells","Local and some distant lesions regress"],"dosing":{"route":"Intratumoural injection","schedule":"10⁶ PFU/mL initial dose, then 10⁸ PFU/mL 3 weeks later and every 2 weeks; volume by lesion size (up to 4 mL total)","monitoring":"Herpetic infection, injection-site complications, immune-mediated events","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Fatigue"},{"event":"Chills"},{"event":"Pyrexia"},{"event":"Nausea"},{"event":"Influenza-like illness"},{"event":"Injection-site pain"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for unresectable melanoma when systemic immunotherapy is unsuitable (TA410)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2015-10-27","type":"approval","region":"US","note":"Unresectable melanoma with injectable lesions (OPTiM): first oncolytic virus approved in the US","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2015-12","type":"approval","region":"EU","note":"EMA approval","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"talquetamab","kind":"drug","name":"Talquetamab","aka":[],"tldr":"Talquetamab is the first drug against GPRC5D, a second myeloma target used after BCMA therapies stop working; taste and skin side effects are its signature.","summary":"Talquetamab is a humanised IgG4 GPRC5DxCD3 bispecific antibody and the first drug against GPRC5D, a receptor expressed on plasma cells and on keratinised tissue. In MonumenTAL-1 the ORR was about 73% at both the weekly and biweekly doses, including about 65% in patients after prior T-cell redirection, showing that a second target works after BCMA therapy stops. Accelerated approval followed in August 2023 after at least 4 prior lines. Dysgeusia (72%), skin toxicity (67%) and nail disorders (54%) are frequent, cytokine release syndrome was grade 3 or higher in only 2%, and infection rates are lower than with BCMA agents. Combinations with teclistamab (RedirecTT-1, ORR about 80% with extramedullary disease) and daratumumab (TRIMM-2) are in phase 3 (MonumenTAL-3, -5 and -6). It opened a second target after BCMA in myeloma, at the cost of taste and skin effects.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Talquetamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Talquetamab"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-relapsed-refractory"],"sections":[],"technologies":["t-cell-engager"],"targets":["gprc5d","cd3"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["monumental-1","nct06208150","nct05552222","nct05455320","nct06550895","nct04586426","nct06577025","nct06500884"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Talvey","modality":"Bispecific T-cell engager (GPRC5D×CD3)","mechanism":"Humanised IgG4 GPRC5D×CD3 bispecific; GPRC5D is expressed on plasma cells and keratinised tissue.","approvals":[{"region":"US","year":2023,"indication":"Relapsed/refractory myeloma after ≥4 lines (accelerated)"}],"mechanismSteps":[],"dosing":{"route":"Subcutaneous","schedule":"Step-up then 0.4 mg/kg weekly or 0.8 mg/kg every 2 weeks","monitoring":"CRS, dysgeusia and weight loss, skin/nail toxicity, infections (lower than BCMA agents)"},"toxicity":[{"event":"Dysgeusia","anyGradePct":72,"note":"MonumenTAL-1"},{"event":"Skin toxicity","anyGradePct":67},{"event":"Nail disorders","anyGradePct":54},{"event":"Cytokine release syndrome","anyGradePct":77,"grade3PlusPct":2}],"access":[],"regulatoryEvents":[{"date":"2023-08-09","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 3.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-talquetamab-tgvs-relapsed-or-refractory-multiple-myeloma","indication":"Adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody"}]},{"id":"tamoxifen","kind":"drug","name":"Tamoxifen","aka":[],"tldr":"The original targeted cancer drug (1977): a pill that blocks oestrogen's effect on breast cancer and halves recurrence, still essential for premenopausal women.","summary":"Selective oestrogen receptor modulator: antagonist in breast, partial agonist in bone and endometrium. Five years reduces 15-year breast cancer mortality by about a third (EBCTCG); ten years is better than five (ATLAS, aTTom). Standard for premenopausal women (alone or with OFS), for men, and for chemoprevention. Endometrial cancer and thromboembolism are the serious rare harms; CYP2D6 activation to endoxifen.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Tamoxifen","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tamoxifen"}],"tags":[],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":["endocrine-therapy","chemoprevention"],"targets":["estrogen-receptor"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["soft-text","dbcg-82bc","nct05768139","nct06979596","nct05774951","nct04906395","nct07287098","nct06492616","nct02747004","nct05514054"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Nolvadex","modality":"Small-molecule SERM","mechanism":"Competitive ER antagonist in breast tissue; tissue-selective co-regulator recruitment.","approvals":[{"region":"US","year":1977,"indication":"Advanced breast cancer"},{"region":"US","year":1998,"indication":"Risk reduction in high-risk women"}],"mechanismSteps":["Tamoxifen is converted by CYP2D6 to endoxifen","Endoxifen binds the ER ligand pocket","Co-repressors rather than co-activators are recruited in breast cells","ER-driven genes (cyclin D1, PGR) are silenced","Proliferation stops; bone and endometrium see partial agonism"],"dosing":{"route":"Oral","schedule":"20 mg once daily for 5-10 years","modifications":"Low-dose 5 mg for DCIS/prevention (TAM-01)","monitoring":"Gynaecologic symptoms; avoid strong CYP2D6 inhibitors"},"toxicity":[{"event":"Hot flushes","anyGradePct":41},{"event":"Endometrial cancer (10-year cumulative)","anyGradePct":3},{"event":"Venous thromboembolism","anyGradePct":2},{"event":"Cataract","anyGradePct":5}],"access":[],"regulatoryEvents":[]},{"id":"tar-210","kind":"drug","name":"TAR-210","aka":[],"tldr":"TAR-210 is an experimental small molecule (intravesical drug-releasing system) from Janssen Research & Development in phase 3 trials for bladder & urothelial cancer, aimed at FGFR2.","summary":"TAR-210 (JNJ-42756493) is a small molecule (intravesical drug-releasing system) developed by Janssen Research & Development. Its target is FGFR2 (the sponsor names FGFR (fibroblast growth factor receptor)). The sponsor states: An intravesical system that provides approximately two years of continuous local bladder delivery of erdafitinib, an FGFR inhibitor, for patients with high-risk non-muscle-invasive bladder cancer harbouring susceptible FGFR alterations. ClinicalTrials.gov describes the intervention as: TAR-210 will be administered intravesically. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06919965 (A Study to Evaluate TAR-210 Versus Intravesical Chemotherapy Treatment in Participants With High Risk Non-Muscle-Invasive Bladder Cancer), in bladder & urothelial cancer. The largest, NCT06919965, plans to enrol 220 participants with primary completion expected 2028-04-06. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TAR-210","url":"https://clinicaltrials.gov/search?intr=JNJ-42756493"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":[],"targets":["fgfr2"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06919965","nct06319820"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"JNJ-42756493","modality":"small molecule (intravesical drug-releasing system)","mechanism":"An intravesical system that provides approximately two years of continuous local bladder delivery of erdafitinib, an FGFR inhibitor, for patients with high-risk non-muscle-invasive bladder cancer harbouring susceptible FGFR alterations.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tara-002","kind":"drug","name":"TARA-002","aka":[],"tldr":"TARA-002 is an experimental cell therapy whose type is not stated from Protara Therapeutics in phase 3 trials for bladder & urothelial cancer, with its target not yet stated publicly.","summary":"TARA-002 is a cell therapy whose type is not stated developed by Protara Therapeutics. The sponsor describes its target as non-muscle invasive bladder cancer and lymphatic malformations, which OnCo does not yet have a target page for. The sponsor states: TARA-002 is based on OK-432, a broad immunopotentiator that is approved in Japan and Taiwan for other indications. ClinicalTrials.gov describes the intervention as: All participants will receive 6 weekly instillations of TARA-002 at the established RP2D (First Treatment Period). Participants who are eligible for reinduction after the First Treatment Period will receive 6 additional weekly doses of TARA. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07480356 (Efficacy and Safety of Intravesical TARA-002 Compared With Investigator's Choice of Intravesical Chemotherapy in Participants With BCG-naïve High-grade Non-muscle Invasive Bladder Cancer), in bladder & urothelial cancer. The largest, NCT07480356, plans to enrol 284 participants with primary completion expected 2028-04. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TARA-002","url":"https://clinicaltrials.gov/search?intr=TARA-002"},{"label":"Sponsor page","url":"https://www.protaratx.com/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["protara-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07480356","nct05951179"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"cell therapy (type not stated)","mechanism":"TARA-002 is based on OK-432, a broad immunopotentiator that is approved in Japan and Taiwan for other indications.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tarlatamab","kind":"drug","name":"Tarlatamab","aka":[],"tldr":"The first T-cell engager to improve survival in a common solid tumour, small-cell lung cancer.","summary":"Accelerated approval May 2024 (DeLLphi-301, ORR 40%); DeLLphi-304 phase 3 (2025) showed OS 13.6 vs 8.3 months versus chemotherapy in second-line SCLC, converting to full approval. First-line combination with PD-L1 (DeLLphi-305) and limited-stage trials ongoing. CRS in ~50% (mostly grade 1-2); step-up dosing with monitoring.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Tarlatamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tarlatamab"},{"label":"NICE TA1091: tarlatamab for extensive-stage small-cell lung cancer after 2 or more treatments (not recommended)","url":"https://www.nice.org.uk/guidance/ta1091"}],"tags":[],"related":[],"cancers":["sclc","extensive-stage-sclc","limited-stage-sclc","lung-cancer"],"sections":[],"technologies":["t-cell-engager"],"targets":["dll3","cd3"],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":["crs"],"trials":["dellphi-304","nct07005128","nct06117774","nct07778316","nct06502977","nct05060016","nct06745323","dellphi-305"],"people":[],"bottlenecks":[],"keyPapers":["paper-dellphi-301-nejm-2023"],"journals":[],"dependsOn":[],"notes":[],"brand":"Imdelltra","modality":"Bispecific T-cell engager (DLL3×CD3)","mechanism":"Half-life-extended BiTE binding DLL3 on tumour and CD3 on T cells.","approvals":[{"region":"US","year":2024,"indication":"Extensive-stage SCLC after platinum chemotherapy"},{"region":"EU","year":2026,"indication":"ES-SCLC after platinum-based chemotherapy; 29 May 2026"},{"region":"England (NICE)","year":2025,"indication":"Extensive-stage small-cell lung cancer progressing after two or more lines of treatment including platinum-based chemotherapy: not recommended","note":"TA1091, published 20 August 2025, says tarlatamab should not be used in the NHS in England for this indication."}],"mechanismSteps":["One arm binds DLL3 on the tumour cell","The other arm binds CD3 on any passing T cell","An artificial immune synapse forms, independent of MHC","The T cell releases perforin and granzymes into the tumour cell","Tumour cell dies; cytokines are released (source of CRS)"],"dosing":{"route":"IV infusion over 1 hour","schedule":"Step-up: 1 mg on cycle 1 day 1, then 10 mg on day 8 and every 2 weeks thereafter","modifications":"Hold for grade 2 CRS/ICANS until resolved; dexamethasone premedication and IV fluids for first two doses","monitoring":"Inpatient monitoring 22-24 h after first two doses; temperature, blood pressure, neurology; ICE score","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":55,"grade3PlusPct":1.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Haemoglobin decreased","anyGradePct":58,"grade3PlusPct":5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Fatigue","anyGradePct":51,"grade3PlusPct":10,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Dysgeusia","anyGradePct":36,"grade3PlusPct":0,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Pyrexia","anyGradePct":36,"grade3PlusPct":0,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Decreased appetite","anyGradePct":34,"grade3PlusPct":2.7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Musculoskeletal pain","anyGradePct":30,"grade3PlusPct":1.1,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Constipation","anyGradePct":30,"grade3PlusPct":0.5,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Nausea","anyGradePct":22,"grade3PlusPct":1.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"DeLLphi-301, n=187"},{"event":"Neurologic toxicity incl. ICANS","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=1e7b6163-5d83-42ea-82c9-cf7620cdc782","note":"Boxed warning; ICANS in a minority, mostly grade 1-2"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.amgensupportplus.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: appraisal for ES-SCLC after platinum in progress (2026)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2022-11","type":"designation","region":"US","note":"Breakthrough Therapy designation, ES-SCLC after platinum","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-05-16","type":"accelerated-approval","region":"US","note":"Accelerated approval, ES-SCLC after platinum (DeLLphi-301)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy"},{"date":"2025-06","type":"filing","region":"US","note":"Phase 3 DeLLphi-304 OS benefit presented; conversion to full approval pursued","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-11-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2024 converted to traditional approval 1.5 years after it was granted.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-tarlatamab-dlle-extensive-stage-small-cell-lung-cancer","indication":"Adult patients with extensive stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy"},{"date":"2025-12","type":"approval","region":"US","note":"Full approval based on DeLLphi-304","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"tasonermin","kind":"drug","name":"Tasonermin","aka":["Recombinant TNF-alpha-1a","rTNF-alpha"],"tldr":"Beromun is a recombinant form of the immune protein TNF-alpha, pumped with the chemotherapy melphalan through the isolated blood supply of an arm or leg to shrink soft-tissue sarcomas so that the limb can be saved rather than amputated.","summary":"Tasonermin was authorised by the European Commission in April 1999 as an adjunct to surgery for irresectable soft-tissue sarcoma of the limbs, with melphalan via mild hyperthermic isolated limb perfusion, to prevent or delay amputation or for palliation, on multicentre European studies in which the majority of patients achieved limb salvage. Systemic TNF-alpha is far too toxic to give intravenously, so the drug exists only for this surgical technique performed in a small number of specialist centres. It is not approved in the US. Regional toxicity (limb oedema, skin reactions, neuropathy) and, if leakage into the systemic circulation occurs, shock are the main risks; the same approach is used for in-transit melanoma.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Tasonermin","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/beromun"}],"tags":["ema-list"],"related":["melphalan"],"cancers":["sarcoma","melanoma"],"sections":[],"technologies":["isolated-limb-perfusion","cytokine-therapy"],"targets":[],"drugs":[],"companies":["boehringer-ingelheim"],"institutions":[],"pathways":[],"terms":["limb-salvage-term"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Beromun","modality":"Recombinant tumour necrosis factor alpha for isolated limb perfusion","mechanism":"Recombinant human TNF-alpha delivered with melphalan under mild hyperthermia to an isolated limb circulation; disrupts tumour vasculature and increases melphalan penetration, causing haemorrhagic necrosis of the sarcoma.","approvals":[{"region":"EU","year":1999,"indication":"Irresectable soft-tissue sarcoma of the limbs with melphalan via isolated limb perfusion, as an adjunct to surgery or for palliation"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tasquinimod","kind":"drug","name":"Tasquinimod","aka":["ABR-215050"],"tldr":"An oral drug aimed at the tissue around the tumour rather than the cancer cells. It held the cancer back on scans and, if anything, shortened life.","summary":"Tasquinimod is an oral quinoline-3-carboxamide that acts on the tumour microenvironment: it binds S100A9, has anti-angiogenic and immunomodulatory effects, and was intended to work where drugs aimed at the androgen receptor had stopped working.\n\nA randomised phase 2 trial improved progression-free survival, and 10TASQ10 was designed to confirm it. It assigned 1,245 chemotherapy-naive men with metastatic castration-resistant prostate cancer and bone metastases 2:1 to tasquinimod (832) or placebo (413) at 241 sites in 37 countries. Median radiographic progression-free survival by central review was 7.0 months (95 percent confidence interval 5.8 to 8.2) with tasquinimod and 4.4 months (3.5 to 5.5) with placebo, hazard ratio 0.64 (0.54 to 0.75, p<0.001).\n\nMedian overall survival went the other way: 21.3 months (19.5 to 23.0) with tasquinimod and 24.0 months (21.4 to 26.9) with placebo, hazard ratio 1.10 (0.94 to 1.28, p=0.25). Grade 3 or worse adverse events were more frequent with tasquinimod (42.8 against 33.6 percent), the commonest being anaemia, fatigue and cancer pain. Active Biotech and Ipsen stopped the programme in prostate cancer.","status":"withdrawn","asOf":"2026-09-25","links":[{"label":"10TASQ10 (Journal of Clinical Oncology 2016)","url":"https://doi.org/10.1200/JCO.2016.66.9697"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["antiangiogenic"],"targets":[],"drugs":[],"companies":["active-biotech","ipsen"],"institutions":[],"pathways":[],"terms":["castration-resistance"],"trials":["tasquinimod-10tasq10"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ABR-215050","modality":"small molecule","mechanism":"Oral quinoline-3-carboxamide binding S100A9, with anti-angiogenic and immunomodulatory effects on the tumour microenvironment.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tazemetostat","kind":"drug","name":"Tazemetostat","aka":[],"tldr":"Tazemetostat was the first EZH2 inhibitor, approved for epithelioid sarcoma and follicular lymphoma in 2020 and withdrawn worldwide in 2026 after secondary blood cancers.","summary":"Accelerated approvals in 2020 (epithelioid sarcoma; relapsed EZH2-mutant or option-less FL). Modest response rates (15% and ~35-69%) with good tolerability; SYMPHONY-1 combined it with lenalidomide-rituximab. Withdrawn worldwide in March 2026 over secondary haematologic malignancies (T-ALL, MDS), ending the EZH2 class in lymphoma for now.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Tazemetostat","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Tazemetostat"},{"label":"Morin et al., Nat Genet 2010: somatic EZH2 Tyr641 mutations in follicular and germinal-centre diffuse large B-cell lymphoma","url":"https://doi.org/10.1038/ng.518"}],"tags":["gap-fill"],"related":[],"cancers":["follicular-lymphoma","sarcoma"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["ezh2"],"drugs":[],"companies":["ipsen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04224493","nct05372354","nct07407283"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: the EZH2 mutations this drug was built for are gain-of-function changes at a single tyrosine in the SET domain, Tyr641 in the original numbering and Tyr646 now, which make the enzyme better at writing the trimethyl mark and worse at making the first methylation, so H3K27me3 accumulates and the germinal-centre exit stays shut. They occur in 7.2% of follicular lymphomas and 21.7% of germinal-centre diffuse large B-cell lymphomas and are absent from the activated B-cell-like subtype (Morin 2010). This is one of only two genotype-selected drug choices in B-cell lymphoma."],"brand":"Tazverik","modality":"Small-molecule EZH2 inhibitor","mechanism":"SAM-competitive inhibitor of EZH2 methyltransferase (wild-type and mutant), reducing H3K27me3 and de-repressing differentiation genes.","approvals":[{"region":"US","year":2020,"indication":"Epithelioid sarcoma; relapsed FL (EZH2-mutant after 2 lines; wild-type without alternatives)","note":"Withdrawn March 2026"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2020-01-23","type":"accelerated-approval","region":"US","note":"Accelerated approval, epithelioid sarcoma","indication":"Treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection"},{"date":"2020-06-18","type":"accelerated-approval","region":"US","note":"Accelerated approval, follicular lymphoma","indication":"Treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least 2 prior systemic therapies. Treatment of adult patients with R/R FL who have no satisfactory alternative treatment options"},{"date":"2026-03","type":"withdrawal","region":"Global","note":"Withdrawn over secondary haematologic malignancies"},{"date":"2026-06-22","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 6.4 years after its accelerated approval.","source":"https://wayback.archive-it.org/7993/20201222063544/https:/www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-tazemetostat-advanced-epithelioid-sarcoma","indication":"Treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection"},{"date":"2026-06-22","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 6.0 years after its accelerated approval.","source":"https://wayback.archive-it.org/7993/20201222062826/https:/www.fda.gov/drugs/fda-granted-accelerated-approval-tazemetostat-follicular-lymphoma","indication":"Treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) whose tumors are positive for an EZH2 mutation as detected by an FDA-approved test and who have received at least 2 prior systemic therapies. Treatment of adult patients with R/R FL who have no satisfactory alternative treatment options"}]},{"id":"tbi-1301","kind":"drug","name":"TBI-1301","aka":[],"tldr":"TBI-1301 is an experimental investigational agent whose form is not stated in the registry from Takara Bio in phase 3 trials for sarcomas, with its target not yet stated publicly.","summary":"TBI-1301 is an investigational agent whose form is not stated in the registry developed by Takara Bio. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Split dose of TBI-1301 is administered intravenously for 2 days following cyclophosphamide/fludarabine pre-treatment. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07174427 (Multi-centre Study of TBI-1301 (INN: Mipetresgene Autoleucel; Mip-cel) in Patients With NY-ESO-1 Positive Synovial Sarcoma), in sarcomas. The largest, NCT07174427, plans to enrol 5 participants with primary completion expected 2032-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TBI-1301","url":"https://clinicaltrials.gov/search?intr=TBI-1301"},{"label":"Sponsor page","url":"https://www.takara-bio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["sarcoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["takara-bio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07174427"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"TCR-T cell therapy (per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tbi-1501","kind":"drug","name":"TBI-1501","aka":[],"tldr":"TBI-1501 is an experimental CAR-T cell therapy from Takara Bio in phase 2 trials for acute lymphoblastic leukaemia, aimed at CD19.","summary":"TBI-1501 is a CAR-T cell therapy developed by Takara Bio. Its target is CD19. ClinicalTrials.gov describes the intervention as: Phase-I portion: Cyclophosphamide is administered for conditioning medication of TBI1501, that is CD19-CAR-T cells, (cohort -1: 3×10\\^5 cells/kg, cohort 1: 1×10\\^6 cells/kg, cohort 2: 3×10\\^6 cells/kg). Phase-II portion: Recommended dose of. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in acute lymphoblastic leukaemia. The largest, NCT03155191, plans to enrol 21 participants with primary completion expected 2035-03-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TBI-1501","url":"https://clinicaltrials.gov/search?intr=TBI-1501"},{"label":"Sponsor page","url":"https://www.takara-bio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["all-leukemia"],"sections":[],"technologies":[],"targets":["cd19"],"drugs":[],"companies":["takara-bio"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03155191"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"CAR-T cell therapy directed at CD19, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tc011","kind":"drug","name":"TC011","aka":[],"tldr":"TC011 is an experimental CAR-T cell therapy from TICAROS in phase 2 trials for diffuse large B-cell lymphoma and follicular lymphoma, aimed at CD19.","summary":"TC011 is a CAR-T cell therapy developed by TICAROS. Its target is CD19 (the sponsor names CD19). The sponsor states: TC011 is an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, given after fludarabine/cyclophosphamide lymphodepleting chemotherapy, for relapsed/refractory large B-cell non-Hodgkin lymphoma. ClinicalTrials.gov describes the intervention as: Anti-CD19 Chimeric Antigen Receptor T cell. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in diffuse large B-cell lymphoma and follicular lymphoma. The largest, NCT07473167, plans to enrol 98 participants with primary completion expected 2028-02-11. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TC011","url":"https://clinicaltrials.gov/search?intr=TC011"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl","follicular-lymphoma"],"sections":[],"technologies":[],"targets":["cd19"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07473167"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"CAR-T cell therapy","mechanism":"TC011 is an anti-CD19 chimeric antigen receptor (CAR) T-cell therapy, given after fludarabine/cyclophosphamide lymphodepleting chemotherapy, for relapsed/refractory large B-cell non-Hodgkin lymphoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tebentafusp","kind":"drug","name":"Tebentafusp","aka":[],"tldr":"The first drug to improve survival in metastatic uveal melanoma, and the first TCR-based bispecific.","summary":"Tebentafusp is an ImmTAC: a soluble, affinity-enhanced T-cell receptor that recognises a gp100 peptide presented on HLA-A*02:01, fused to an anti-CD3 single-chain antibody that recruits any T cell to the melanoma cell. Because gp100 is a melanocyte-lineage protein, the drug also hits skin melanocytes, so rash and pruritus are expected. It is given weekly after step-up doses of 20, 30 and 68 micrograms, with inpatient monitoring for the first three doses because of cytokine release syndrome and hypotension. Approved in 2022 in the US and EU for HLA-A*02:01-positive metastatic uveal melanoma, it was the first drug to improve survival in that disease (overall survival 21.7 versus 16.0 months) and the first TCR-based bispecific approved anywhere. It is being tested in cutaneous melanoma after PD-1 therapy (TEBE-AM), and Immunocore's PRAME-directed brenetafusp follows the same design.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Tebentafusp","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tebentafusp"}],"tags":[],"related":["hla-a-02-01"],"cancers":["melanoma"],"sections":[],"technologies":["t-cell-engager","tcr-t"],"targets":["gp100","cd3","hla-a"],"drugs":[],"companies":["immunocore"],"institutions":[],"pathways":[],"terms":["step-up-dosing"],"trials":["nct05549297","nct04262466"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Kimmtrak","modality":"ImmTAC (TCR×CD3 bispecific)","mechanism":"Soluble affinity-enhanced TCR against gp100 peptide-HLA fused to anti-CD3 scFv.","approvals":[{"region":"US","year":2022,"indication":"HLA-A*02:01+ metastatic uveal melanoma"}],"mechanismSteps":["Soluble high-affinity TCR binds gp100 peptide presented on HLA-A*02:01 on melanoma cells","Fused anti-CD3 scFv recruits any T cell","Immune synapse forms; T cell releases cytotoxic granules","Melanocytic tumour cells are killed; skin melanocytes cause rash","Weekly redosing maintains pressure"],"dosing":{"route":"IV infusion","schedule":"20 µg day 1, 30 µg day 8, 68 µg day 15, then 68 µg weekly","modifications":"Hold for grade ≥3 CRS; skin reactions managed with antihistamines and steroids","monitoring":"Inpatient monitoring for first three doses; blood pressure, temperature, LFTs","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Cytokine release syndrome"},{"event":"Rash"},{"event":"Pyrexia"},{"event":"Pruritus"},{"event":"Fatigue"},{"event":"Nausea"},{"event":"Hypotension"},{"event":"Transaminase increase"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.kimmtrak.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for HLA-A*02:01+ metastatic uveal melanoma (TA867)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-02","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-01-25","type":"approval","region":"US","note":"HLA-A*02:01+ unresectable or metastatic uveal melanoma: first TCR therapeutic approved","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-04","type":"approval","region":"EU","note":"EMA approval","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"technetium-sulfur-colloid","kind":"drug","name":"Technetium Tc-99m sulfur colloid","aka":["Tc-99m sulphur colloid"],"tldr":"Technetium sulfur colloid is the radioactive tracer injected around a breast cancer or melanoma before surgery so the surgeon can find the first lymph node the tumour drains to and remove only that node instead of the whole basin.","summary":"Technetium-99m sulfur colloid has been used for liver, spleen and bone marrow scintigraphy since the 1970s, and the FDA label was extended to lymphatic mapping to localise lymph nodes draining a primary breast tumour or melanoma. Sentinel node biopsy guided by the tracer, often with blue dye, replaced routine axillary dissection in breast cancer and elective node dissection in melanoma after the NSABP B-32 and MSLT-I trials, sparing most patients lymphoedema. Tilmanocept (Lymphoseek), a purpose-designed mannose receptor agent, is the newer alternative.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Technetium_(99mTc)_sulfur_colloid","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Technetium_(99mTc)_sulfur_colloid"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=technetium%20tc%2099m%20sulfur%20colloid"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["breast-hr-positive","melanoma"],"sections":[],"technologies":["sentinel-node"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07764653","nct07121595","nct01276054"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Technetium Tc 99m Sulfur Colloid Kit","modality":"Radiopharmaceutical for lymphatic mapping and liver, spleen and marrow imaging","mechanism":"Colloidal particles labelled with technetium-99m are taken up by macrophages; injected near a tumour they drain to and lodge in the first (sentinel) lymph nodes, which a gamma probe then finds during surgery.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tilmanocept-tc99m","kind":"drug","name":"Technetium-99m tilmanocept","aka":[],"tldr":"The purpose-built tracer for sentinel lymph node mapping in breast cancer, melanoma and oral cancer, replacing off-label sulfur colloid.","summary":"Technetium-99m tilmanocept is a mannosylated dextran labelled with technetium-99m that binds CD206, the mannose receptor on macrophages in lymph nodes. Because it is small it drains rapidly from the injection site, and because it binds a receptor it stays in the sentinel node rather than passing through to second-echelon nodes, which simplifies surgery. It was the first agent purpose-built for sentinel lymph node mapping, replacing off-label sulfur colloid. The FDA approved it in March 2013 for breast cancer and melanoma, in 2014 for sentinel node localisation in oral cavity squamous cell carcinoma, where mapping showed 97.8% sensitivity, and extended it to children in 2017; the EU approved it in 2014. It is detected with a handheld gamma counter in theatre. Tilmanocept finds the first node a cancer would spread to so that only that node needs removal.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Tilmanocept","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Lymphoseek"}],"tags":["gap-fill","diagnostic"],"related":[],"cancers":["breast-hr-positive","melanoma","head-and-neck"],"sections":[],"technologies":["sentinel-node","spect"],"targets":[],"drugs":[],"companies":["navidea"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00671918","nct01106040","nct00911326"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lymphoseek","modality":"Radiopharmaceutical lymphatic mapping agent","mechanism":"Mannosylated dextran that binds CD206 (mannose receptor) on macrophages in sentinel nodes, giving rapid uptake and low pass-through to second-echelon nodes.","approvals":[{"region":"US","year":2013,"indication":"Lymphatic mapping with a handheld gamma counter in breast cancer and melanoma"},{"region":"US","year":2014,"indication":"Sentinel lymph node localisation in oral cavity squamous cell carcinoma"},{"region":"EU","year":2014,"indication":"Sentinel node imaging in breast cancer, melanoma, oral SCC"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"teclistamab","kind":"drug","name":"Teclistamab","aka":[],"tldr":"Teclistamab was the first off-the-shelf bispecific for multiple myeloma, and is now approved after just one prior line of therapy.","summary":"Teclistamab is a humanised IgG4 DuoBody bispecific that binds BCMA on myeloma cells with one arm and CD3 on T cells with the other, forming an artificial immune synapse that kills the tumour cell independent of MHC. It was the first off-the-shelf bispecific for myeloma, given subcutaneously after step-up doses of 0.06 and 0.3 mg/kg, then 1.5 mg/kg weekly, with less frequent dosing in sustained responders. Accelerated approval in 2022 rested on MajesTEC-1 in heavily pretreated myeloma (objective response rate 63 percent), and in the first quarter of 2026 the label expanded to relapsed or refractory myeloma after at least one prior therapy, with daratumumab, based on MajesTEC-3. Cytokine release syndrome occurred in 72 percent of MajesTEC-1 patients but was severe in under 1 percent, and serious infections affected 30 to 54 percent across trials, so infection prophylaxis is essential.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Teclistamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Teclistamab"}],"tags":[],"related":[],"cancers":["multiple-myeloma","myeloma-relapsed-refractory"],"sections":[],"technologies":["t-cell-engager"],"targets":["bcma","cd3"],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05572515","nct06208150","nct05552222","nct04586426","nct06577025"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tecvayli","modality":"Bispecific T-cell engager (BCMA×CD3)","mechanism":"Humanised IgG4 BCMA×CD3 DuoBody.","approvals":[{"region":"US","year":2022,"indication":"Relapsed/refractory myeloma after ≥4 lines"},{"region":"US","year":2026,"indication":"Relapsed/refractory myeloma after ≥1 prior therapy"},{"region":"EU","year":2022,"indication":"R/R myeloma ≥3 lines (conditional, Aug 2022)","note":"Conditional marketing authorisation"}],"mechanismSteps":["One arm binds BCMA on the tumour cell","The other arm binds CD3 on any passing T cell","An artificial immune synapse forms, independent of MHC","The T cell releases perforin and granzymes into the tumour cell","Tumour cell dies; cytokines are released (source of CRS)"],"dosing":{"route":"Subcutaneous injection","schedule":"Step-up 0.06 then 0.3 mg/kg, then 1.5 mg/kg weekly; may move to 3 mg/kg every 2 weeks after ≥6 months of response, then every 4 weeks","modifications":"Hold for grade ≥2 CRS/ICANS; IVIG for hypogammaglobulinaemia; antimicrobial prophylaxis","monitoring":"48-hour hospitalisation after each step-up dose; infections, IgG levels, blood counts","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e"},"toxicity":[{"event":"Pyrexia","anyGradePct":76,"grade3PlusPct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"MajesTEC-1"},{"event":"Cytokine release syndrome","anyGradePct":72,"grade3PlusPct":0.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"MajesTEC-1"},{"event":"Neutrophils decreased","anyGradePct":88,"grade3PlusPct":70,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"MajesTEC-1"},{"event":"Musculoskeletal pain","anyGradePct":44,"grade3PlusPct":4.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"MajesTEC-1"},{"event":"Injection site reaction","anyGradePct":37,"grade3PlusPct":0.6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"MajesTEC-1"},{"event":"Fatigue","anyGradePct":33,"grade3PlusPct":2.4,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"MajesTEC-1"},{"event":"Pneumonia","anyGradePct":24,"grade3PlusPct":15,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"MajesTEC-1"},{"event":"Neurologic toxicity","anyGradePct":60,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"ICANS 6%"},{"event":"Serious infections","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=54e0f974-ccee-44ea-9254-40e9883cee1e","note":"30-54% across trials; fatal 4-5%"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.janssencarepath.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for relapsed/refractory myeloma after ≥3 therapies (TA1006)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-06","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-08-24","type":"approval","region":"EU","note":"Conditional approval: first BCMA bispecific worldwide","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-10-25","type":"accelerated-approval","region":"US","note":"Accelerated approval, relapsed/refractory myeloma after ≥4 lines (MajesTEC-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody"},{"date":"2024-02-20","type":"label-change","region":"US","note":"Biweekly dosing added for responders","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q1","type":"approval","region":"US","note":"Relapsed/refractory myeloma after ≥1 prior line, with daratumumab (MajesTEC-3)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-03-05","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2022 converted to traditional approval 3.4 years after it was granted.","indication":"Adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent and an anti-CD38 monoclonal antibody"}]},{"id":"tegafur-gimeracil-oteracil","kind":"drug","name":"Tegafur, gimeracil and oteracil (S-1)","aka":["S-1","TS-1"],"tldr":"Teysuno (S-1) is an oral chemotherapy combining a fluorouracil pro-drug with two protectors. It is a standard in Japan and approved in Europe for stomach cancer with cisplatin and for bowel cancer when other fluoropyrimidines cause hand-foot syndrome or heart problems.","summary":"S-1 has been a mainstay of gastric, pancreatic, colorectal and lung cancer treatment in Japan since 1999 (ACTS-GC established adjuvant S-1 in gastric cancer). The European Commission authorised Teysuno in March 2011 for advanced gastric cancer with cisplatin, on the FLAGS trial in which S-1 plus cisplatin was non-inferior to fluorouracil plus cisplatin with less toxicity, and in 2022 for metastatic colorectal cancer as monotherapy or with oxaliplatin or irinotecan, with or without bevacizumab, in patients who cannot continue another fluoropyrimidine because of hand-foot syndrome or cardiovascular toxicity. Because Western patients metabolise tegafur faster through CYP2A6 polymorphisms, doses differ from Japan. It is not approved in the US, where a trial of S-1 versus fluorouracil failed to show superiority. Diarrhoea, neutropenia and stomatitis are the main toxicities.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Tegafur/gimeracil/oteracil","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/teysuno"},{"label":"Uesaka et al., JASPAC 01 (Lancet 2016)","url":"https://doi.org/10.1016/S0140-6736(16)30583-9"},{"label":"Ueno et al., GEST (JCO 2013)","url":"https://doi.org/10.1200/JCO.2012.43.3680"}],"tags":["ema-list"],"related":["fluorouracil","capecitabine"],"cancers":["gastric","colorectal","pancreatic","resectable-pdac","metastatic-pdac"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["taiho"],"institutions":[],"pathways":[],"terms":["prodrug"],"trials":["jaspac-01","nct07262567","nct00660894","nct03448549"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer: S-1 is the adjuvant standard in Japan on JASPAC 01 (five-year survival 44.1 against 24.4 percent with gemcitabine, hazard ratio 0.57) and non-inferior to gemcitabine in advanced disease in GEST (median 9.7 against 8.8 months, hazard ratio 0.96; the gemcitabine plus S-1 arm was not superior). The EU product Teysuno (marketing authorisation 14 March 2011) is licensed for gastric and colorectal cancer only, and Western patients metabolise tegafur differently, so the drug is not used for pancreatic cancer in Europe or the United States."],"brand":"Teysuno","modality":"Oral fluoropyrimidine combination (antimetabolite)","mechanism":"Tegafur is a fluorouracil prodrug; gimeracil inhibits dihydropyrimidine dehydrogenase to sustain fluorouracil levels, and oteracil blocks phosphorylation of fluorouracil in the gut to reduce diarrhoea.","approvals":[{"region":"JP","year":1999,"indication":"Gastric cancer; later colorectal, head and neck, lung, pancreatic, biliary and breast cancer (TS-1)"},{"region":"EU","year":2011,"indication":"Advanced gastric cancer with cisplatin; metastatic colorectal cancer intolerant of other fluoropyrimidines added 2022 (Teysuno)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"telisotuzumab-adizutecan","kind":"drug","name":"Telisotuzumab adizutecan","aka":[],"tldr":"Telisotuzumab adizutecan is an experimental antibody-drug conjugate from AbbVie in phase 3 trials for colorectal cancer and non-small-cell lung cancer, with its target not yet stated publicly.","summary":"Telisotuzumab adizutecan (ABBV-400) is an antibody-drug conjugate developed by AbbVie. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 8 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07525206 (A Study to Access Intravenous (IV) Telisotuzumab Adizutecan in Combination With IV Bevacizumab Compared to Standard of Care IV Bevacizumabin Combination With Oral Trifluridine and Tipiracil in Adult Participants With Refractory Metastatic Colorectal Cancer), NCT06614192 (A Study Assessing Adverse Events and Disease Activity of Intravenously (IV) Infused Telisotuzumab Adizutecan in Adult Participants With c-Met Protein Above Cutoff Level Above Refractory Metastatic Colorectal Cancer) and NCT07155187 (A Study to Assess Adverse Events and Change in Disease Activity of Intravenous (IV) Telisotuzumab Adizutecan, Monotherapy or in Combination With Osimertinib, Compared to Standard of Care in Adult Participants With Locally Advanced or Metastatic EGFR-Mutated Non-Squamous Non-Small Cell Lung Cancer), in colorectal cancer and non-small-cell lung cancer. The largest, NCT07005102, plans to enrol 854 participants with primary completion expected 2031-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Telisotuzumab adizutecan","url":"https://clinicaltrials.gov/search?intr=ABBV-400"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal","nsclc","met-altered-nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07525206","nct06614192","nct07155187","nct07005102","nct07023289","nct06107413","nct06820463","nct07196644","nct06772623"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["23 Sept 2026: the payload class comes from the INN stem alone; no phase 1 paper is linked on this record, so the specific payload is left unstated rather than copied from memory."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"ABBV-400","modality":"ADC","payload":"Topoisomerase I inhibitor class, read from the INN stem -tecan of adizutecan (WHO INN stems for ADC payloads); the specific payload and DAR are not stated on any record OnCo reads","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"telisotuzumab-vedotin","kind":"drug","name":"Telisotuzumab vedotin","aka":[],"tldr":"Telisotuzumab vedotin (Emrelis) is the first c-MET-directed ADC, approved in 2025 for lung cancer with high c-MET protein.","summary":"Telisotuzumab vedotin (Emrelis) is an anti-c-MET antibody linked to the microtubule inhibitor MMAE through a cleavable mc-vc-PABC linker; it needs no MET mutation, only high c-MET protein on the tumour surface, which it uses as a delivery address. It received accelerated approval in May 2025 for previously treated non-squamous NSCLC with high c-MET overexpression on the strength of LUMINOSITY (objective response rate around 35 percent), and is given at 1.9 mg/kg every 2 weeks. It is the first c-MET-directed ADC to reach approval, after Breakthrough Therapy designation in 2022. Peripheral neuropathy, fatigue, decreased appetite, peripheral oedema and nausea are the main toxicities, and interstitial lung disease and ocular symptoms are monitored. The confirmatory TeliMET NSCLC-01 trial is ongoing. It treats lung cancers that overproduce c-MET protein by using that protein as a doorway.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Telisotuzumab%20vedotin"}],"tags":[],"related":["met-overexpression"],"cancers":["nsclc","met-altered-nsclc"],"sections":[],"technologies":["adc"],"targets":["met"],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":["mc-vc-pabc"],"trials":["luminosity","nct06568939"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Emrelis","modality":"ADC","payload":"MMAE","linker":"mc-vc-PABC","mechanism":"Anti-c-MET antibody with MMAE.","approvals":[{"region":"US","year":2025,"indication":"c-MET-high non-squamous NSCLC, previously treated (accelerated)"}],"mechanismSteps":["Antibody binds c-MET on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","MMAE is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"1.9 mg/kg every 2 weeks","monitoring":"Peripheral neuropathy, ocular symptoms, ILD","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Peripheral neuropathy"},{"event":"Fatigue"},{"event":"Decreased appetite"},{"event":"Peripheral oedema"},{"event":"Nausea"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2022-01","type":"designation","region":"US","note":"Breakthrough Therapy designation, c-MET overexpressing NSCLC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-05-14","type":"accelerated-approval","region":"US","note":"Accelerated approval, previously treated c-MET-high non-squamous NSCLC (LUMINOSITY) The confirmatory requirement was still open 1.3 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) with high c-Met protein overexpression [≥50% of tumor cells with strong (3+) staining], as determined by an FDA-approved test, who have received a prior systemic therapy."}]},{"id":"telotristat-ethyl","kind":"drug","name":"Telotristat ethyl","aka":["LX1032","Telotristat etiprate"],"tldr":"Telotristat ethyl (Xermelo) is a tablet that cuts the serotonin that neuroendocrine tumours pour out, easing the constant diarrhoea of carcinoid syndrome when somatostatin analogue injections are not enough.","summary":"Telotristat ethyl was approved by the FDA in February 2017 and authorised in the EU in September 2017 for carcinoid syndrome diarrhoea in combination with somatostatin analogue therapy in adults inadequately controlled by that therapy, on the TELESTAR phase 3 trial (135 patients) in which the 250 mg three-times-daily dose reduced daily bowel movements more than placebo, a statistically significant difference, and lowered urinary 5-HIAA. It is the first oral drug for carcinoid syndrome and the first tryptophan hydroxylase inhibitor approved for any indication. Nausea, headache, raised liver enzymes, depression and constipation are the main adverse effects. Marketed by TerSera in the US and SERB in Europe.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Telotristat_ethyl","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/xermelo"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=telotristat"}],"tags":["ema-list","supportive"],"related":["octreotide-lanreotide"],"cancers":["neuroendocrine","small-intestinal-net"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["tersera-therapeutics","serb-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03790111","nct04543955"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Xermelo","modality":"Oral tryptophan hydroxylase inhibitor","supportive":true,"mechanism":"Prodrug of telotristat, which inhibits tryptophan hydroxylase, the rate-limiting enzyme of peripheral serotonin synthesis in neuroendocrine tumour cells, lowering urinary 5-HIAA and bowel frequency without crossing into the brain.","approvals":[{"region":"US","year":2017,"indication":"Carcinoid syndrome diarrhoea with somatostatin analogue therapy in adults inadequately controlled by SSA alone"},{"region":"EU","year":2017,"indication":"Same (Xermelo)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"temoporfin","kind":"drug","name":"Temoporfin","aka":["mTHPC","meta-tetra(hydroxyphenyl)chlorin"],"tldr":"Foscan is a light-activated drug used in Europe to shrink advanced head and neck cancers that have failed other treatments and cannot be operated on, irradiated or given chemotherapy, to relieve symptoms.","summary":"Temoporfin was authorised by the European Commission in October 2001 for palliative treatment of advanced head and neck squamous cell carcinoma failing prior therapies and unsuitable for radiotherapy, surgery or systemic chemotherapy, on open-label phase 2 studies in which a proportion of patients achieved local tumour responses and symptom relief. It is a second-generation photosensitiser with a shorter photosensitivity period than porfimer sodium (Photofrin), the first-generation agent approved in the US for oesophageal and lung cancer, but patients must still avoid bright light for about two weeks. The FDA did not approve it. Pain at the treated site, local swelling and skin photosensitivity are the main adverse effects. Marketed by biolitec.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Temoporfin","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/foscan"}],"tags":["ema-list"],"related":[],"cancers":["head-and-neck"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["biolitec"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01415986","nct01086488","nct03003065"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Foscan","modality":"Intravenous photosensitiser for photodynamic therapy","mechanism":"Chlorin photosensitiser that accumulates in tumour tissue and, on illumination with 652 nm laser light four days after injection, generates singlet oxygen that causes tumour necrosis and vascular shutdown.","approvals":[{"region":"EU","year":2001,"indication":"Palliative treatment of advanced head and neck squamous cell carcinoma failing prior therapy and unsuitable for radiotherapy, surgery or chemotherapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"temozolomide","kind":"drug","name":"Temozolomide","aka":[],"tldr":"The only chemotherapy proven to extend life in glioblastoma, given during and after radiation. It works best when the tumour has switched off a repair gene called MGMT.","summary":"Stupp/EORTC 26981-NCIC (2005): adding concurrent and adjuvant temozolomide to radiotherapy raised median OS from 12.1 to 14.6 months and 2-year survival from 10% to 27%. Benefit concentrates in MGMT-promoter-methylated tumours (median OS ~23 months vs ~13 months unmethylated). Also standard with radiotherapy in grade 3 astrocytoma (CATNON) and, with PCV as an alternative, in oligodendroglioma. Oral, well tolerated; lymphopenia and hypermutation at recurrence are the costs.","status":"standard-of-care","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Temozolomide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Temozolomide"}],"tags":[],"related":[],"cancers":["glioblastoma","idh-mutant-astrocytoma","paediatric-high-grade-glioma"],"sections":[],"technologies":["cytotoxic-chemotherapy","imrt-igrt"],"targets":[],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":["mgmt"],"trials":["eortc-26981","catnon","eortc-22033","nct06703398","nct03709680","nct07326566","nct07310784","nct04478279","nct06595186","nct04752813","nct07569042","nct06703255","nct04485949","nct06413706","nct05765812","nct05440786","nct04587830","nct04121455","nct07297212","nct07492680","nct06012695","nct05902169","nct05417594","nct07015242","nct04443010","nct07195591","nct06556563","nct04910022","nct03491683","nct04919226","nct05768919","nct05664243","nct03862430"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Temodar","modality":"Oral alkylating chemotherapy","mechanism":"Prodrug of MTIC; methylates O6-guanine; cytotoxicity depends on unrepaired lesions when MGMT is silenced.","approvals":[{"region":"US","year":1999,"indication":"Refractory anaplastic astrocytoma"},{"region":"US","year":2005,"indication":"Newly diagnosed glioblastoma with radiotherapy"},{"region":"EU","year":1999,"indication":"Temodal; malignant glioma (recurrent 1999; newly diagnosed glioblastoma with radiotherapy 2005); 26 Jan 1999"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"1999-08-11","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adults with refractory anaplastic astrocytoma"},{"date":"2005-03-15","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 1999 converted to traditional approval 5.6 years after it was granted.","indication":"Adults with refractory anaplastic astrocytoma"}]},{"id":"tempus-xt-cdx","kind":"drug","name":"Tempus xT CDx","aka":[],"tldr":"Tempus's tumour-and-normal gene panel, FDA-approved in 2023 as a companion test for EGFR antibodies in bowel cancer.","summary":"The FDA approved Tempus xT CDx in 2023 as a 648-gene tumour-normal panel with a companion diagnostic claim for KRAS and NRAS wild-type colorectal cancer (cetuximab and panitumumab eligibility) and with tumour-profiling claims across solid tumours. The matched-normal design distinguishes inherited variants from tumour-acquired ones and reduces false positives. Tempus pairs the test with its multimodal database and trial-matching software, which is the company's commercial thesis: the test is the entry point to data.","status":"approved","asOf":"2026-09-10","links":[{"label":"Tempus: xT CDx","url":"https://www.tempus.com/oncology/genomic-profiling/xt-cdx/"}],"tags":["test"],"related":[],"cancers":["colorectal","pancreatic"],"sections":[],"technologies":["cgp","companion-diagnostic"],"targets":["kras","egfr"],"drugs":["cetuximab","panitumumab"],"companies":["tempus"],"institutions":[],"pathways":[],"terms":["ngs","germline-vs-somatic"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-sepulveda-molecular-biomarkers-colorectal-guideline-jco-2017"],"journals":[],"dependsOn":[],"notes":["What a result means: a RAS mutation rules out EGFR antibodies in bowel cancer; other findings feed treatment and trial options."],"brand":"xT CDx","modality":"Tissue NGS companion diagnostic test (648 genes, tumour-normal)","mechanism":"Hybrid-capture sequencing of 648 genes in tumour tissue with matched normal blood or saliva to separate somatic from germline variants; reports MSI status and companion claims.","approvals":[{"region":"US","year":2023,"indication":"Companion diagnostic for cetuximab and panitumumab (KRAS/NRAS wild-type colorectal cancer) with tumour-profiling claims"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"temsirolimus","kind":"drug","name":"Temsirolimus","aka":[],"tldr":"A weekly infusion that was the first drug to extend survival in poor-risk kidney cancer (2007), and in 2024 the first targeted drug to improve outcomes in childhood rhabdomyosarcoma.","summary":"Global ARCC trial (NEJM 2007): OS 10.9 vs 7.3 months versus interferon in poor-risk metastatic RCC; approved 2007. Displaced in RCC by VEGF TKIs and immunotherapy. Approved in EU for relapsed mantle cell lymphoma (2009). COG ARST1431 (2024) showed improved event-free survival when added to VAC/VI in intermediate-risk rhabdomyosarcoma.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Temsirolimus","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=temsirolimus"}],"tags":["gap-fill"],"related":[],"cancers":["rcc","rhabdomyosarcoma","mantle-cell-lymphoma"],"sections":[],"technologies":["kinase-inhibitors","pi3k-akt-mtor-inhibitors"],"targets":[],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":["pi3k-akt-mtor","hif-vhl"],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Torisel","modality":"Intravenous mTOR inhibitor (rapamycin analogue)","mechanism":"Binds FKBP12 and allosterically inhibits mTORC1, reducing HIF-1α translation, cell growth and angiogenesis.","approvals":[{"region":"US","year":2007,"indication":"Advanced renal cell carcinoma"},{"region":"EU","year":2007,"indication":"Advanced RCC (poor risk); relapsed/refractory mantle cell lymphoma added 2009"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tenalisib","kind":"drug","name":"Tenalisib","aka":["RP6530"],"tldr":"Tenalisib is an oral pi3k inhibitor from Rhizen Pharmaceuticals SA, in registered phase 2 trials for triple-negative breast cancer.","summary":"Tenalisib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Rhizen Pharmaceuticals SA, in triple-negative breast cancer. A phosphoinositide 3-kinase inhibitor: the name stem -lisib marks a small molecule that blocks PI3K signalling. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Tenalisib","url":"https://clinicaltrials.gov/search?intr=Tenalisib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["tnbc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["rhizen-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06189209"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral PI3K inhibitor","mechanism":"A phosphoinositide 3-kinase inhibitor: the name stem -lisib marks a small molecule that blocks PI3K signalling.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"teniposide","kind":"drug","name":"Teniposide","aka":["VM-26"],"tldr":"Teniposide is a close relative of etoposide approved in the United States in 1992 for children whose acute lymphoblastic leukaemia has come back after induction, and used in Europe for childhood brain tumours and neuroblastoma.","summary":"Teniposide was synthesised alongside etoposide at Sandoz in the 1960s and is roughly ten times more potent in vitro. The FDA approved it in 1992, in combination with other agents, for induction in children with refractory acute lymphoblastic leukaemia, based on Children's Cancer Group and St Jude experience. It has been used in Europe in neuroblastoma, small cell lung cancer and lymphomas. Hypersensitivity reactions to its polysorbate vehicle and the class risk of secondary acute myeloid leukaemia limited it, and etoposide became the standard podophyllotoxin. Bristol-Myers Squibb discontinued Vumon in most markets.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Teniposide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Teniposide"},{"label":"ChEMBL CHEMBL1200604","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200604"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["all-leukemia","neuroblastoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["top2a"],"drugs":["etoposide"],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07583511","nct07188441","nct01700946"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vumon","modality":"Podophyllotoxin topoisomerase II inhibitor, intravenous","mechanism":"Stabilises the complex between topoisomerase II and DNA so the double-strand breaks the enzyme makes are not resealed.","approvals":[{"region":"US","year":1992,"indication":"Refractory childhood acute lymphoblastic leukaemia, in combination induction"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tepotinib","kind":"drug","name":"Tepotinib","aka":["MSC2156119","EMD 1214063"],"tldr":"Tepotinib is a once-daily targeted pill for lung cancers driven by a MET exon 14 skipping mutation, and the MET inhibitor NICE funds in England.","summary":"Tepotinib is an oral MET inhibitor for advanced non-small cell lung cancer with MET exon 14 skipping alterations, which can be detected in tumour tissue or in circulating tumour DNA. The VISION trial showed durable responses in about half of patients whether or not they had been treated before. Japan approved it first in 2020, the FDA in 2021 and the EU in 2022. NICE recommended it in 2022 within its licence with a commercial arrangement. Peripheral oedema is the main side effect.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Tepotinib","links":[{"label":"VISION (NEJM 2020)","url":"https://doi.org/10.1056/NEJMoa2004407"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=tepotinib"},{"label":"NICE TA789: tepotinib for treating advanced non-small-cell lung cancer with MET gene alterations","url":"https://www.nice.org.uk/guidance/ta789"}],"tags":["gap-fill"],"related":["met-ex14"],"cancers":["nsclc","met-altered-nsclc","lung-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["met"],"drugs":["capmatinib"],"companies":["merck-kgaa"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02864992"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tepmetko","modality":"Small-molecule MET tyrosine kinase inhibitor","mechanism":"A highly selective, once-daily MET kinase inhibitor that blocks signalling from MET receptors kept active by exon 14 skipping mutations.","approvals":[{"region":"US","year":2021,"indication":"Metastatic NSCLC with MET exon 14 skipping alterations"},{"region":"EU","year":2022,"indication":"Advanced NSCLC with MET exon 14 skipping alterations after immunotherapy or platinum chemotherapy"},{"region":"JP","year":2020,"indication":"Unresectable advanced or recurrent NSCLC with MET exon 14 skipping"},{"region":"England (NICE)","year":2022,"indication":"Advanced non-small-cell lung cancer with MET exon 14 skipping alterations","note":"TA789, published 18 May 2022, subject to the commercial arrangement (VISION)."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2021-02-03","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition (MET) exon 14 skipping alterations."},{"date":"2024-02-15","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2021 converted to traditional approval 3.0 years after it was granted.","indication":"Treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition (MET) exon 14 skipping alterations."}]},{"id":"tersolisib","kind":"drug","name":"Tersolisib","aka":[],"tldr":"Tersolisib is an experimental small-molecule drug from Eli Lilly and in phase 3 trials for HR-positive / HER2-negative breast cancer, aimed at PIK3CA / PI3K-alpha.","summary":"Tersolisib (STX-478, LY4064809) is a small-molecule drug developed by Eli Lilly and. Its target is PIK3CA / PI3K-alpha (the sponsor names mutant-selective PI3Kalpha (PIK3CA)). The sponsor states: Tersolisib (STX-478/LY4064809) is a mutant-selective PI3Kalpha inhibitor studied as monotherapy and in combination with fulvestrant, with or without CDK4/6-targeted endocrine therapy, in PIK3CA-mutated advanced solid tumours and HR-positive breast cancer. ClinicalTrials.gov describes the intervention as: STX-478 is a mutant-selective PI3Kα inhibitor. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07174336 (A Study of Tersolisib (LY4064809/STX-478) With Other Anti-Cancer Treatments in Participants With Advanced Breast Cancer With a Genetic Change (PIK3CA)), in HR-positive / HER2-negative breast cancer. The largest, NCT05768139, plans to enrol 880 participants with primary completion expected 2030-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Tersolisib","url":"https://clinicaltrials.gov/search?intr=STX-478"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":["pik3ca"],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["pik3ca-2"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"STX-478, LY4064809","modality":"small molecule","mechanism":"Tersolisib (STX-478/LY4064809) is a mutant-selective PI3Kalpha inhibitor studied as monotherapy and in combination with fulvestrant, with or without CDK4/6-targeted endocrine therapy, in PIK3CA-mutated advanced solid tumours and HR-positive breast cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tgrx-326","kind":"drug","name":"TGRX-326","aka":["TGRX-326 QD (once a day)"],"tldr":"TGRX-326 is an experimental small-molecule drug from Shenzhen TargetRx in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"TGRX-326 is a small-molecule drug developed by Shenzhen TargetRx. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: Subjects will be treated with the investigational drug TGRX-326 at 60 mg once day in 21-day cycles. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06082635 (TGRX-326 Chinese Phase III for Advanced Non-small Cell Lung Cancer (NSCLC)), in non-small-cell lung cancer. The largest, NCT06082635, plans to enrol 321 participants (actual) with primary completion was scheduled for 2026-04-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TGRX-326","url":"https://clinicaltrials.gov/search?intr=TGRX-326"},{"label":"Sponsor page","url":"https://www.tjrbiosciences.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["shenzhen-targetrx"],"institutions":[],"pathways":[],"terms":[],"trials":["nsclc-3","nct05955391"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"thalidomide","kind":"drug","name":"Thalidomide","aka":[],"tldr":"Thalidomide is the drug behind the 1960s birth-defect tragedy, rehabilitated as the first immunomodulatory myeloma drug and the parent of lenalidomide and pomalidomide.","summary":"Singhal (NEJM 1999) showed activity in refractory myeloma; approved 2006 with dexamethasone for newly diagnosed disease under the strict REMS-type distribution programme first built for its leprosy indication (1998). Still used in low-cost regimens worldwide (thalidomide-based induction/maintenance, e.g. in Myeloma XI and LMIC settings). Neuropathy, thrombosis and sedation limit it.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Thalidomide","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Thalidomide"}],"tags":["gap-fill","historic"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["protac-degrader","antiangiogenic"],"targets":["ikzf1"],"drugs":[],"companies":["bms"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Thalomid","modality":"Oral cereblon modulator (first IMiD)","mechanism":"Binds cereblon, altering E3 ligase substrate specificity (IKZF1/3 degradation), with anti-angiogenic and immunomodulatory effects; the mechanism explained decades after its teratogenic disaster.","approvals":[{"region":"US","year":1998,"indication":"Erythema nodosum leprosum"},{"region":"US","year":2006,"indication":"Newly diagnosed multiple myeloma with dexamethasone"},{"region":"EU","year":2008,"indication":"Untreated multiple myeloma ≥65 or transplant-ineligible, with melphalan-prednisone"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2006-05-25","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Newly diagnosed multiple myeloma"},{"date":"2014-06-19","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2006 converted to traditional approval 8.1 years after it was granted.","indication":"Newly diagnosed multiple myeloma"}]},{"id":"theo-260","kind":"drug","name":"THEO-260","aka":[],"tldr":"THEO-260 is an oncolytic virus from Theolytics Limited, in registered phase 2 trials for ovarian cancer.","summary":"THEO-260 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Theolytics Limited, in ovarian cancer. An oncolytic virus, as described in the registry record: a virus engineered to infect and burst tumour cells and alert the immune system. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of THEO-260","url":"https://clinicaltrials.gov/search?intr=THEO-260"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["theolytics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06618235"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"THEO-260","modality":"Oncolytic virus","mechanism":"An oncolytic virus, as described in the registry record: a virus engineered to infect and burst tumour cells and alert the immune system.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"therascreen-cdx","kind":"drug","name":"therascreen companion diagnostic kits (KRAS, EGFR, PIK3CA, FGFR, BRAF)","aka":[],"tldr":"A family of quick single-gene tests that decide who can have several bowel, lung, breast and bladder cancer drugs.","summary":"Qiagen's therascreen kits were among the first PCR companion diagnostics: therascreen KRAS RGQ (PMA P110030, 6 July 2012) identifies KRAS wild-type colorectal cancers eligible for cetuximab and panitumumab; therascreen EGFR RGQ (2013) was the companion for afatinib; therascreen PIK3CA RGQ (2019) selects alpelisib for PIK3CA-mutant, hormone-receptor-positive breast cancer from tissue or plasma; therascreen FGFR RGQ RT-PCR (2019) detects FGFR3 mutations and FGFR2/3 fusions for erdafitinib in urothelial cancer; therascreen BRAF V600E RGQ (2020) is the companion for encorafenib with cetuximab in colorectal cancer. Single-gene PCR is cheap and fast but is being displaced by panels where many genes must be checked from one small biopsy.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P110030","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P110030"},{"label":"Qiagen therascreen portfolio","url":"https://www.qiagen.com/us/product-categories/diagnostics-and-clinical-research/oncology"}],"tags":["test"],"related":[],"cancers":["colorectal","nsclc","breast-hr-positive","urothelial"],"sections":[],"technologies":["companion-diagnostic"],"targets":["kras","egfr","pik3ca","fgfr2","braf"],"drugs":["cetuximab","panitumumab","afatinib","alpelisib","erdafitinib","encorafenib"],"companies":["qiagen"],"institutions":[],"pathways":[],"terms":["companion-diagnostic-term"],"trials":["nct07617805"],"people":[],"bottlenecks":[],"keyPapers":["paper-karapetis-kras-cetuximab-colorectal-nejm-2008","paper-douillard-prime-panitumumab-ras-nejm-2013"],"journals":[],"dependsOn":[],"notes":["What a result means: for bowel cancer, a KRAS or NRAS mutation means the EGFR antibodies will not work; for breast cancer, a PIK3CA mutation opens the door to a PI3K inhibitor.","Colorectal cancer: the RAS companion diagnostic family. The threshold the assays must reach is extended RAS, KRAS and NRAS exons 2, 3 and 4, because 17% of KRAS exon 2 wild-type patients carry another RAS mutation and gain nothing from an EGFR antibody (Douillard 2013, Sepulveda 2017)."],"brand":"therascreen","modality":"Real-time PCR companion diagnostic test family","mechanism":"Scorpions and ARMS allele-specific real-time PCR kits run on the Rotor-Gene Q, each detecting a defined set of hotspot mutations or fusions from tumour tissue (and plasma for PIK3CA).","approvals":[{"region":"US","year":2012,"indication":"therascreen KRAS: cetuximab and panitumumab eligibility in metastatic colorectal cancer"},{"region":"US","year":2013,"indication":"therascreen EGFR: afatinib in EGFR-mutant NSCLC"},{"region":"US","year":2019,"indication":"therascreen PIK3CA (tissue and plasma): alpelisib in breast cancer; therascreen FGFR: erdafitinib in urothelial cancer"},{"region":"US","year":2020,"indication":"therascreen BRAF V600E: encorafenib plus cetuximab in colorectal cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"thioguanine","kind":"drug","name":"Thioguanine","aka":["6-TG","Tioguanine"],"tldr":"Thioguanine (Tabloid) is an oral chemotherapy tablet used in some regimens to induce and consolidate remission in acute myeloid leukaemia; long-term use is avoided because it damages the liver.","summary":"Thioguanine, a sibling of mercaptopurine from the Elion-Hitchings programme, was approved in 1966 for remission induction and consolidation of acute non-lymphocytic leukaemias. It appears in historical AML regimens such as DAT and TAD and in some childhood ALL protocols, but the label advises against maintenance or long-term use because of hepatic sinusoidal obstruction syndrome and nodular regenerative hyperplasia; the UK ALL97 trial confirmed excess liver toxicity when it replaced mercaptopurine in maintenance. Myelosuppression is dose-limiting, and TPMT-deficient patients need dose reduction.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Tioguanine","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=thioguanine"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/thioguanine"}],"tags":["nci-list","generic"],"related":["mercaptopurine"],"cancers":["aml"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["aall1231","aall1331","nct02521493"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tabloid","modality":"Thiopurine antimetabolite","mechanism":"Guanine analogue converted to thioguanine nucleotides incorporated into DNA, causing strand breaks and cell death; inactivated by TPMT.","approvals":[{"region":"US","year":1966,"indication":"Remission induction and consolidation of acute non-lymphocytic leukaemias"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"thiotepa","kind":"drug","name":"Thiotepa","aka":["Thioplex (historic)","Triethylenethiophosphoramide"],"tldr":"Thiotepa is an old chemotherapy with a modern job: at high doses it prepares patients for stem cell transplants, and it is still instilled into the bladder or body cavities for superficial bladder cancer and malignant effusions.","summary":"Thiotepa was first approved in 1959 for adenocarcinoma of the breast or ovary, superficial papillary carcinoma of the urinary bladder and control of malignant intracavitary effusions. Tepadina (approved 2017 in the US, 2010 in the EU) added conditioning before allogeneic haematopoietic stem cell transplantation, initially for children with class 3 beta-thalassaemia in the US and, in the EU, for haematological and solid tumours in adults and children. In practice thiotepa-based regimens (TBC, thiotepa-busulfan-fludarabine) are standard before autologous transplant in primary CNS lymphoma and in cord blood and haploidentical transplantation. Profound myelosuppression, mucositis and skin toxicity from sweat excretion (patients bathe frequently) are the main issues.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Thiotepa","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=thiotepa"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/thiotepa"},{"label":"EPAR (Tepadina)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/tepadina"}],"tags":["nci-list"],"related":[],"cancers":["urothelial","breast-hr-positive","ovarian","primary-cns-lymphoma"],"sections":[],"technologies":["cytotoxic-chemotherapy","allogeneic-hsct","autologous-stem-cell-transplant","bcg-and-intravesical-therapy"],"targets":[],"drugs":[],"companies":["adienne"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tepadina","modality":"Alkylating agent (ethylenimine)","mechanism":"Polyfunctional alkylating agent related to nitrogen mustard; releases ethyleneimine radicals that cross-link DNA. Crosses the blood-brain barrier, which underlies its use in CNS lymphoma conditioning.","approvals":[{"region":"US","year":1959,"indication":"Breast and ovarian adenocarcinoma; superficial papillary bladder carcinoma (intravesical); malignant effusions"},{"region":"US","year":2017,"indication":"Conditioning before allogeneic HSCT for class 3 beta-thalassaemia in children (Tepadina)"},{"region":"EU","year":2010,"indication":"Conditioning before haematopoietic progenitor cell transplantation for haematological diseases and solid tumours (Tepadina)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"thyrotropin-alfa","kind":"drug","name":"Thyrotropin alfa","aka":["Recombinant human TSH","rhTSH"],"tldr":"Thyrogen is an injected version of thyroid-stimulating hormone that lets people with thyroid cancer have radioiodine treatment and follow-up blood tests without stopping their thyroid hormone tablets and suffering weeks of hypothyroidism.","summary":"Thyrotropin alfa was approved by the FDA in November 1998 as an adjunctive diagnostic tool for serum thyroglobulin testing with or without radioiodine imaging in the follow-up of well-differentiated thyroid cancer, and in 2007 for pre-therapeutic stimulation before radioiodine remnant ablation after thyroidectomy in patients without distant metastases; the EU authorised Thyrogen in 2000 with the same uses. The HiLo and ESTIMABL trials showed that rhTSH-stimulated low-dose (1.1 GBq) radioiodine ablation is as effective as high-dose ablation after thyroid hormone withdrawal, sparing patients hypothyroid symptoms and reducing radiation exposure. Nausea and headache are the main adverse effects; transient tumour swelling can occur with CNS or spinal metastases. Marketed by Sanofi (Genzyme).","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Thyrotropin_alfa","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/thyrogen"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=thyrotropin%20alfa"}],"tags":["ema-list","supportive"],"related":[],"cancers":["thyroid"],"sections":[],"technologies":["radioiodine-therapy"],"targets":[],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":["radioiodine-term"],"trials":["nct04964284","nct04971473","nct00295763"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Thyrogen","modality":"Recombinant human thyroid-stimulating hormone","mechanism":"Recombinant TSH stimulates iodine uptake and thyroglobulin release from thyroid remnants and differentiated thyroid cancer cells, allowing radioiodine ablation and thyroglobulin testing without stopping levothyroxine.","approvals":[{"region":"US","year":1998,"indication":"Adjunctive diagnostic tool for thyroglobulin testing in well-differentiated thyroid cancer; pre-therapeutic stimulation for radioiodine remnant ablation added 2007"},{"region":"EU","year":2000,"indication":"Thyroglobulin testing and radioiodine imaging in thyroid cancer follow-up; pre-therapeutic stimulation for radioiodine ablation"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tilatamig-samrotecan","kind":"drug","name":"Tilatamig samrotecan","aka":[],"tldr":"AstraZeneca's EGFR×c-MET bispecific ADC, the most advanced in the most crowded next-generation ADC target pair.","summary":"Tilatamig samrotecan is AstraZeneca's bispecific antibody-drug conjugate that binds both EGFR and c-MET and delivers the topoisomerase I inhibitor AZ14170133 through a cleavable linker. Its low-affinity EGFR arm and high-affinity c-MET arm mean avidity biases delivery toward tumour cells co-expressing both receptors and spares normal tissue that expresses EGFR alone. It is in phase 2 in EGFR-mutant NSCLC after osimertinib and in EGFR-wild-type NSCLC, per AstraZeneca's 2026 pipeline, and is also being explored in head and neck cancer. Stomatitis, nausea, fatigue and neutropenia are the reported toxicities, and dose optimisation on an every-3-weeks schedule is under way. It is the most advanced of roughly 24 c-MET×EGFR ADC candidates, so its results will shape a crowded field. It is a two-address ADC meant to hit lung cancers carrying both EGFR and c-MET while sparing normal cells.","status":"phase-2","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Tilatamig samrotecan","url":"https://clinicaltrials.gov/search?intr=AZD9592"}],"tags":[],"related":[],"cancers":["nsclc","head-and-neck"],"sections":[],"technologies":["bispecific-adc"],"targets":["egfr","met"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05647122"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"AZD9592","modality":"Bispecific ADC","payload":"AZ14170133 (TOP1 inhibitor)","linker":"Cleavable","mechanism":"Bispecific EGFR/c-MET IgG with TOP1 payload; avidity-driven tumour selectivity.","approvals":[],"mechanismSteps":["Antibody binds EGFR and c-MET (bispecific) on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","TOP1 inhibitor is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"Phase 2 dose-optimisation; every 3 weeks","source":"https://clinicaltrials.gov/study/NCT05647122"},"toxicity":[{"event":"Stomatitis"},{"event":"Nausea"},{"event":"Fatigue"},{"event":"Neutropenia"}],"access":[{"country":"US","reimbursement":"Investigational","asOf":"2026-09-06"}],"regulatoryEvents":[]},{"id":"tilt-123","kind":"drug","name":"TILT-123","aka":["Ad5/3-E2F-d24-hTNFa-IRES-hIL2 (TILT-123)"],"tldr":"TILT-123 is an oncolytic virus from TILT Biotherapeutics Ltd., in registered phase 2 trials for ovarian cancer.","summary":"TILT-123 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by TILT Biotherapeutics Ltd., in ovarian cancer. An oncolytic virus, as described in the registry record: a virus engineered to infect and burst tumour cells and alert the immune system. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of TILT-123","url":"https://clinicaltrials.gov/search?intr=TILT-123"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["tilt-biotherapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05271318"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"TILT-123","modality":"Oncolytic virus","mechanism":"An oncolytic virus, as described in the registry record: a virus engineered to infect and burst tumour cells and alert the immune system.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tinengotinib","kind":"drug","name":"Tinengotinib","aka":[],"tldr":"A next-generation FGFR inhibitor designed to work after pemigatinib or futibatinib stop working, now in a global phase 3.","summary":"Tinengotinib is a type I multi-kinase inhibitor active against FGFR1-3, including the FGFR2 kinase-domain resistance mutations (N550, V565) that emerge after pemigatinib or futibatinib, plus VEGFR, Aurora and JAK kinases. It is aimed at FGFR-altered cholangiocarcinoma that has progressed on a prior FGFR inhibitor, a group with no approved targeted option. A phase 1/2 study in heavily pretreated patients, including after prior FGFR inhibitors, showed disease control with median PFS of about 5 to 6 months. The global phase 3 FIRST-308 trial randomises FGFR inhibitor-refractory patients to tinengotinib versus FOLFOX or FOLFIRI, primary endpoint PFS; the first US patient was dosed in 2025. Whether its broad kinase profile adds toxicity without benefit over selective FGFR2 inhibitors is open. For a newcomer, it is a drug for bile duct cancer that has outgrown the existing FGFR pills.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT05948475: tinengotinib versus physician's choice in FGFR-altered cholangiocarcinoma (phase 3)","url":"https://clinicaltrials.gov/study/NCT05948475"},{"label":"ClinicalTrials.gov NCT05253053: TT-00420 (tinengotinib) monotherapy and combinations (phase 1/2)","url":"https://clinicaltrials.gov/study/NCT05253053"}],"tags":[],"related":[],"cancers":["cholangiocarcinoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["fgfr2"],"drugs":[],"companies":["transthera"],"institutions":[],"pathways":[],"terms":["fgfr2-fusion"],"trials":["first-308","nct07052253","nct07498478","nct05948475"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"TT-00420","modality":"Small-molecule multi-kinase inhibitor (FGFR1-3, VEGFR, Aurora, JAK)","mechanism":"Type I inhibitor active against FGFR2 kinase-domain resistance mutations (N550, V565) plus VEGFR and Aurora kinases.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tiragolumab","kind":"drug","name":"Tiragolumab","aka":[],"tldr":"An immune-brake blocker that looked excellent in a phase 2 lung cancer trial and then failed every phase 3.","summary":"CITYSCAPE (phase 2, 2020) showed tiragolumab plus atezolizumab roughly doubled response rate in PD-L1-high NSCLC. Phase 3 SKYSCRAPER-01 (PD-L1-high NSCLC) missed PFS and OS; SKYSCRAPER-02 (extensive-stage SCLC) was negative; SKYSCRAPER-06 (non-squamous NSCLC) was worse than control. Roche discontinued most of the programme in 2024 and 2025. Other anti-TIGIT antibodies (domvanalimab, ociperlimab) showed no clearer benefit.\n\nLesson: a small randomised phase 2 with a surrogate endpoint in a selected population can mislead; redundancy between checkpoints means blocking a second one does not necessarily add to PD-1/PD-L1 blockade.","status":"negative","asOf":"2026-09-06","links":[{"label":"Roche SKYSCRAPER-01 final analysis (2024)","url":"https://www.roche.com/media/releases/med-cor-2024-11-26"}],"tags":["failure","lesson:phase-2-mirage"],"related":["tigit-plus-pd1-caution"],"cancers":["nsclc","sclc"],"sections":[],"technologies":["checkpoint-inhibitor","tigit-blockade"],"targets":["tigit","pdl1"],"drugs":["atezolizumab"],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05904886","nct05009069","nct05805501","nct04524871","nct05645692","nct04665856"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"RG6058, MTIG7192A","modality":"Monoclonal antibody (anti-TIGIT)","mechanism":"IgG1 anti-TIGIT with intact Fc; blocks TIGIT-PVR interaction on T and NK cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tisagenlecleucel","kind":"drug","name":"Tisagenlecleucel","aka":[],"tldr":"Tisagenlecleucel was the first CAR-T therapy ever approved (2017), for children and young adults whose leukaemia had come back after everything else.","summary":"ELIANA (n=75 infused, age ≤25): ORR 81% within 3 months, 12-month OS 76%, 5-year EFS ~44% in responders without further therapy (2023 update). Approved 30 August 2017 for relapsed/refractory B-ALL up to age 25; later for DLBCL and follicular lymphoma. 4-1BB costimulation gives long persistence; CD19-negative relapse is the main failure mode. Emily Whitehead, treated in 2012, remains in remission.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Tisagenlecleucel","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tisagenlecleucel"}],"tags":[],"related":[],"cancers":["all-leukemia","all-paediatric-relapsed","dlbcl"],"sections":[],"technologies":["car-t"],"targets":["cd19"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":["crs","icans"],"trials":["eliana","nct04094311","nct05888493","nct03876769"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Kymriah","code":"CTL019","modality":"CAR-T (CD19)","mechanism":"Autologous T cells transduced with a lentiviral CD19 scFv-4-1BB-CD3ζ CAR; expand in vivo and kill CD19+ blasts.","approvals":[{"region":"US","year":2017,"indication":"Relapsed/refractory B-ALL, patients up to 25 years"},{"region":"US","year":2018,"indication":"Relapsed/refractory DLBCL"},{"region":"US","year":2022,"indication":"Relapsed/refractory follicular lymphoma"}],"mechanismSteps":["Leukapheresis collects the patient's T cells","Lentivirus inserts the CD19 CAR gene; cells expand for ~3 weeks","Lymphodepleting fludarabine/cyclophosphamide makes room","Infused CAR-T cells find CD19 on blasts and kill them, expanding a thousand-fold (CRS risk)","Persisting CAR-T cells cause B-cell aplasia, a marker of ongoing activity"],"dosing":{"route":"Single IV infusion","schedule":"0.2-5.0 × 10^6 CAR+ cells/kg (≤50 kg) or 0.1-2.5 × 10^8 (>50 kg) after lymphodepletion","monitoring":"CRS and ICANS (boxed warning; tocilizumab on site), prolonged cytopenias, hypogammaglobulinaemia, secondary T-cell malignancy (class warning 2024)","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Kymriah"},"toxicity":[{"event":"Cytokine release syndrome","anyGradePct":77,"grade3PlusPct":46,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Kymriah","note":"ELIANA, Penn grading"},{"event":"Neurotoxicity (ICANS)","anyGradePct":40,"grade3PlusPct":13},{"event":"Prolonged cytopenias","grade3PlusPct":32},{"event":"B-cell aplasia / hypogammaglobulinaemia","note":"Expected; IVIG replacement"}],"access":[],"regulatoryEvents":[{"date":"2017-08-30","type":"approval","region":"US","note":"First CAR-T approval in history"},{"date":"2022-05-27","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 4.3 years later, when the FDA's table was read.","indication":"Treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy"},{"date":"2024-04-18","type":"label-change","region":"US","note":"Class boxed warning for T-cell malignancies"},{"date":"2025-06-27","type":"label-change","region":"US","note":"REMS requirements removed for approved CAR-T products"}]},{"id":"tislelizumab","kind":"drug","name":"Tislelizumab","aka":[],"tldr":"A Chinese-developed PD-1 blocker, engineered to avoid a side-channel that may blunt other PD-1 drugs, now approved in the US and EU for oesophageal and stomach cancer.","summary":"Humanised IgG4 with an Fc engineered to minimise FcγR binding (reducing macrophage-mediated T-cell clearance). US approvals: second-line ESCC (March 2024), first-line HER2-negative gastric/GEJ with chemotherapy (December 2024, RATIONALE-305), first-line ESCC PD-L1 ≥1% with chemotherapy (March 2025, RATIONALE-306). Also the PD-1 partner in HERIZON-GEA-01 with zanidatamab. Approved in China across many indications since 2019.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Tislelizumab","links":[{"label":"FDA approval, first-line ESCC","url":"https://www.onclive.com/view/fda-approves-first-line-tislelizumab-plus-chemotherapy-for-unresectable-or-metastatic-escc"}],"tags":[],"related":[],"cancers":["esophageal","gastric","nsclc","hcc","recurrent-metastatic-nasopharyngeal-carcinoma"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["beone"],"institutions":[],"pathways":[],"terms":["irae","fc-effector"],"trials":["rationale-306","herizon-gea-01","rationale-302","nct07502300","nct04379635","nct07170995","nct05840016","nct06030258","nct05651022","nct05431270","nct05609370","nct05635708","nct05661955","nct07244705","nct06427941","nct05632939","nct07700667","nct07554521","nct07331155","nct06745908","nct04699188","nct07315750","nct04170283","nct03967977","nct07554456","nct06499350","nct07390383","nct06825494","nct07019675","nct07669415","nct03736889"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tevimbra","code":"BGB-A317","modality":"Monoclonal antibody (anti-PD-1)","mechanism":"PD-1 blockade with reduced Fcγ receptor engagement.","approvals":[{"region":"China","year":2019,"indication":"Classical Hodgkin lymphoma (first); subsequently urothelial, NSCLC, HCC, ESCC, gastric, nasopharyngeal"},{"region":"US","year":2024,"indication":"Second-line ESCC after chemotherapy; first-line HER2-negative gastric/GEJ with chemotherapy (PD-L1 ≥1)"},{"region":"US","year":2025,"indication":"First-line ESCC, PD-L1 ≥1%, with platinum chemotherapy"}],"mechanismSteps":["Tislelizumab binds PD-1 on exhausted T cells","PD-L1 on the tumour can no longer switch them off","Its engineered tail avoids being grabbed by macrophages, which may otherwise strip the drug off T cells","T cells resume attacking the tumour"],"dosing":{"route":"Intravenous","schedule":"200 mg every 3 weeks","modifications":"Withhold or discontinue for immune-related adverse events per label","monitoring":"Thyroid, liver, glucose; immune-related toxicity","source":"https://www.drugs.com/history/tevimbra.html"},"toxicity":[{"event":"Immune-related adverse events (thyroiditis, pneumonitis, colitis, hepatitis)","note":"Class effects of PD-1 blockade"},{"event":"Rash, fatigue"}],"access":[],"regulatoryEvents":[{"date":"2026-08-25","type":"approval","region":"US","note":"FDA approves zanidatamab-hrii and tislelizumab-jsgr for HER2-positive gastric, gastroesophageal junction, or esophageal adenocarcinoma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-zanidatamab-hrii-and-tislelizumab-jsgr-her2-positive-gastric-gastroesophageal-junction"},{"date":"2024-03","type":"approval","region":"US","note":"Second-line ESCC (RATIONALE-302)"},{"date":"2024-12","type":"approval","region":"US","note":"First-line gastric/GEJ (RATIONALE-305)"},{"date":"2025-03-04","type":"approval","region":"US","note":"First-line ESCC PD-L1 ≥1% (RATIONALE-306)"}]},{"id":"tisotumab-vedotin","kind":"drug","name":"Tisotumab vedotin","aka":[],"tldr":"Tisotumab vedotin is an ADC against tissue factor, the first to show a survival benefit in recurrent cervical cancer.","summary":"Tisotumab vedotin is a fully human anti-tissue-factor IgG1 antibody joined to the microtubule inhibitor MMAE through a cleavable mc-vc-PABC linker; tissue factor is abundant on cervical cancer cells, and the released payload kills the target cell and its neighbours. It is given at 2 mg/kg every 3 weeks for recurrent or metastatic cervical cancer after chemotherapy. Accelerated approval in 2021 followed innovaTV 204, and full approval in 2024 followed innovaTV 301, in which overall survival was 11.5 versus 9.5 months against chemotherapy in second- and third-line disease, the first survival benefit in this setting. Conjunctival disorders affected 37 percent, so eye exams, steroid and vasoconstrictor drops and cold packs during infusion are required; epistaxis (26 percent) and haemorrhage (21 percent) reflect the tissue-factor target.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Tisotumab_vedotin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tisotumab%20vedotin"}],"tags":[],"related":[],"cancers":["cervical","recurrent-metastatic-cervical-cancer"],"sections":[],"technologies":["adc"],"targets":["tissue-factor"],"drugs":[],"companies":["pfizer","genmab"],"institutions":[],"pathways":[],"terms":["mc-vc-pabc"],"trials":["nct06459180","nct03485209"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tivdak","modality":"ADC","payload":"MMAE","linker":"mc-vc-PABC","mechanism":"Human anti-tissue-factor IgG1 with MMAE.","approvals":[{"region":"US","year":2021,"indication":"Recurrent/metastatic cervical cancer after chemotherapy (full 2024)"},{"region":"EU","year":2025,"indication":"Recurrent/metastatic cervical after systemic therapy; 28 Mar 2025"}],"mechanismSteps":["Antibody binds Tissue factor on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","MMAE is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion over 30 min","schedule":"2 mg/kg (max 200 mg) every 3 weeks","modifications":"Hold for grade 2 conjunctival changes; discontinue for grade ≥3 corneal events","monitoring":"Ophthalmic exam before first 9 cycles; steroid, vasoconstrictor, and lubricating eye drops; cold packs during infusion","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1"},"toxicity":[{"event":"Peripheral neuropathy","anyGradePct":38,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Conjunctival disorders","anyGradePct":37,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Nausea","anyGradePct":33,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Fatigue","anyGradePct":28,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Epistaxis","anyGradePct":26,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Alopecia","anyGradePct":24,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Decreased appetite","anyGradePct":24,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Haemorrhage","anyGradePct":21,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Dry eye","anyGradePct":21,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"},{"event":"Corneal complications","grade3PlusPct":3.2,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=c9fe3f32-4219-466e-acb9-3f609b4f4df1","note":"innovaTV 301, n=250"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: appraisal for recurrent cervical cancer (2025-26)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2021-09-20","type":"accelerated-approval","region":"US","note":"Accelerated approval, recurrent or metastatic cervical cancer after chemotherapy (innovaTV 204)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy"},{"date":"2024-04-29","type":"conversion","region":"US","note":"Full approval with OS benefit (innovaTV 301)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy"}]},{"id":"tivozanib","kind":"drug","name":"Tivozanib","aka":[],"tldr":"Tivozanib is a VEGF-receptor pill that hits VEGFR1 to 3 with little off-target kinase activity, so it is better tolerated than other anti-angiogenic pills, though hypertension and fatigue remain common. It is approved for kidney cancer after two or more prior treatments, and its TiNivo-2 trial showed that restarting immunotherapy after failure adds nothing.","summary":"Tivozanib is a VEGFR1-3 inhibitor with low off-target kinase activity, which explains its relatively favourable tolerability among anti-angiogenic pills. In TIVO-3 (2019) it gave PFS 5.6 versus 3.9 months against sorafenib in third line and beyond, leading to FDA approval in March 2021 for advanced RCC after at least 2 prior systemic therapies. TiNivo-2 (2024 to 2026) then tested adding nivolumab after prior immunotherapy: PFS was 5.7 versus 7.4 months (HR 1.10), so rechallenge gave no benefit, and with CONTACT-03 this settled the IO-rechallenge question in RCC for now. Hypertension (44%) and fatigue (43%) are the main adverse effects. AVEO developed it and was acquired by LG Chem in 2023. It is a well-tolerated later-line VEGFR pill whose own trial showed that restarting immunotherapy after failure does not help.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Tivozanib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tivozanib"}],"tags":[],"related":[],"cancers":["rcc"],"sections":[],"technologies":["kinase-inhibitors","antiangiogenic"],"targets":["vegf"],"drugs":[],"companies":["aveo"],"institutions":[],"pathways":[],"terms":[],"trials":["tinivo-2"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Fotivda","modality":"Small-molecule kinase inhibitor (VEGFR)","mechanism":"Selective VEGFR1-3 inhibitor with low off-target kinase activity.","approvals":[{"region":"US","year":2021,"indication":"Relapsed/refractory advanced RCC after ≥2 prior systemic therapies"},{"region":"EU","year":2017,"indication":"EU first (Aug 2017)"}],"mechanismSteps":["Oral dosing 3 weeks on / 1 off","Potent VEGFR blockade with minimal PDGFR/KIT inhibition","Less hand-foot syndrome and diarrhoea than multi-targeted TKIs"],"dosing":{"route":"Oral","schedule":"1.34 mg daily, 21 days on / 7 days off","monitoring":"Blood pressure, thyroid, liver enzymes"},"toxicity":[{"event":"Hypertension","anyGradePct":44},{"event":"Fatigue","anyGradePct":43},{"event":"Diarrhoea","anyGradePct":33},{"event":"Hand-foot syndrome","anyGradePct":15}],"access":[],"regulatoryEvents":[]},{"id":"tk216","kind":"drug","name":"TK216","aka":["TK-216"],"tldr":"TK216 was an attempt to hit the fusion protein that causes Ewing sarcoma directly, something long thought impossible for a transcription factor. In early trials with vincristine it produced a few responses but not enough to carry the drug forward.","summary":"TK216 was Oncternal Therapeutics' first-in-class small molecule aimed at EWS-FLI1, the fusion transcription factor present in about 85 percent of Ewing sarcomas. It was given as a continuous intravenous infusion, alone and with vincristine, in a phase 1/2 trial in relapsed or refractory Ewing sarcoma. A minority of heavily pretreated patients had objective responses, including some complete responses, but the response rate did not meet the threshold for a registrational study and the sponsor did not continue development.\n\nThe Ewing sarcoma page names it with IGF-1R antibodies and PARP inhibitors among the targeted approaches that have not yet succeeded, while lurbinectedin and combinations remain in trials.","status":"phase-2","asOf":"2026-09-17","links":[],"tags":["subtype-drugs-wave"],"related":["vincristine","lurbinectedin"],"cancers":["ewing-sarcoma"],"sections":[],"technologies":[],"targets":["ewsr1-fli1"],"drugs":[],"companies":["oncternal-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"TK216","modality":"Small-molecule EWS-FLI1 inhibitor","mechanism":"Designed from the YK-4-279 series to bind the EWS-FLI1 fusion oncoprotein and prevent its interaction with RNA helicase A, disabling the transcription factor that drives Ewing sarcoma; it also disrupts microtubules.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tng260","kind":"drug","name":"TNG260","aka":[],"tldr":"TNG260 is a small-molecule inhibitor from Tango Therapeutics, Inc., in registered phase 2 trials for non-small-cell lung cancer, small-cell lung cancer.","summary":"TNG260 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Tango Therapeutics, Inc., in non-small-cell lung cancer, small-cell lung cancer. Described in the registry record as a corest inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of TNG260","url":"https://clinicaltrials.gov/search?intr=TNG260"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc","sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["tango-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05887492"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"TNG260","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a corest inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tng456","kind":"drug","name":"TNG456","aka":[],"tldr":"TNG456 is an experimental small-molecule drug from Tango Therapeutics in phase 2 trials for non-small-cell lung cancer and glioma & glioblastoma, aimed at PRMT5 (MTAP-deleted cancers).","summary":"TNG456 is a small-molecule drug developed by Tango Therapeutics. Its target is PRMT5 (MTAP-deleted cancers) (the sponsor names PRMT5). The sponsor states: A selective PRMT5 inhibitor being developed for cancers with MTAP loss/deletion, including glioblastoma. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer and glioma & glioblastoma. The largest, NCT06810544, plans to enrol 191 participants with primary completion expected 2027-03-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TNG456","url":"https://clinicaltrials.gov/search?intr=TNG456"},{"label":"Sponsor pipeline page","url":"https://tangotx.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","glioblastoma"],"sections":[],"technologies":[],"targets":["prmt5-mtap"],"drugs":[],"companies":["tango-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06810544"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"A selective PRMT5 inhibitor being developed for cancers with MTAP loss/deletion, including glioblastoma.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tng462","kind":"drug","name":"TNG462","aka":[],"tldr":"TNG462 is an experimental small-molecule drug from Tango Therapeutics in phase 2 trials for pancreatic ductal adenocarcinoma and non-small-cell lung cancer, aimed at PRMT5 (MTAP-deleted cancers).","summary":"TNG462 is a small-molecule drug developed by Tango Therapeutics and Servier Bio-Innovation. Its target is PRMT5 (MTAP-deleted cancers) (the sponsor names PRMT5). The sponsor states: A selective PRMT5 inhibitor administered orally, developed for tumours with an MTAP homozygous deletion. ClinicalTrials.gov describes the intervention as: TNG462, a selective PRMT5 inhibitor, will be administered orally. It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in pancreatic ductal adenocarcinoma and non-small-cell lung cancer. The largest, NCT05732831, plans to enrol 225 participants with primary completion expected 2027-06. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TNG462","url":"https://clinicaltrials.gov/search?intr=TNG462"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic","nsclc"],"sections":[],"technologies":[],"targets":["prmt5-mtap"],"drugs":[],"companies":["tango-therapeutics","servier"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06922591","nct06188702","nct05732831"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"A selective PRMT5 inhibitor administered orally, developed for tumours with an MTAP homozygous deletion.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tno155","kind":"drug","name":"TNO155","aka":[],"tldr":"TNO155 is a small-molecule inhibitor from Novartis Pharmaceuticals, in registered phase 2 trials for non-small-cell lung cancer, small-cell lung cancer.","summary":"TNO155 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Novartis Pharmaceuticals, in non-small-cell lung cancer, small-cell lung cancer. Described in the registry record as a shp2 inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of TNO155","url":"https://clinicaltrials.gov/search?intr=TNO155"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc","sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07468071","nct04699188"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"TNO155","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a shp2 inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tocilizumab","kind":"drug","name":"Tocilizumab","aka":["RoActemra","Tofidence","Tyenne"],"tldr":"Tocilizumab (Actemra) is a rheumatoid arthritis antibody that has become the rescue drug for cytokine release syndrome, the fever and blood-pressure crash that CAR-T cells and bispecific antibodies can trigger. Every CAR-T centre must have it on hand.","summary":"Tocilizumab was approved by the FDA in 2010 for rheumatoid arthritis and in August 2017, on the same day as the first CAR-T approval (tisagenlecleucel), for severe or life-threatening CAR-T-cell-induced cytokine release syndrome in patients aged 2 and over, on a retrospective analysis of pooled CAR-T trials in which about two-thirds of patients had CRS resolve within 14 days of one or two doses. The CAR-T REMS programmes require two doses to be available on site for every patient, and it is used with the same logic for CRS from bispecific T-cell engagers, though not on label. Because it does not cross the blood-brain barrier it does not treat neurotoxicity (ICANS), for which corticosteroids are used. The EU authorised RoActemra in 2009; biosimilars (Tofidence, Tyenne) followed in 2023 to 2024. Serious infection carries a boxed warning in chronic use.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Tocilizumab","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=tocilizumab"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/tocilizumab"}],"tags":["nci-list","supportive"],"related":["siltuximab","tisagenlecleucel"],"cancers":[],"sections":[],"technologies":["car-t","t-cell-engager","monoclonal-antibody"],"targets":["il6"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07589634","nct04077723","nct05583617","nct05535244","nct04246086","nct05207670","nct03075696","nct03671018","nct04524871","nct06084936","nct03533283","nct04980222"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Actemra","modality":"Monoclonal antibody (anti-IL-6 receptor, humanised IgG1)","supportive":true,"mechanism":"Binds soluble and membrane-bound IL-6 receptors and blocks IL-6 signalling, the central mediator of cytokine release syndrome after T-cell-redirecting therapy.","approvals":[{"region":"US","year":2010,"indication":"Rheumatoid arthritis (first approval)"},{"region":"US","year":2017,"indication":"Severe or life-threatening CAR-T-cell-induced cytokine release syndrome, age 2 and over"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"topotecan","kind":"drug","name":"Topotecan","aka":[],"tldr":"Topotecan is the long-standing second-line chemotherapy for relapsed small-cell lung cancer, and now the comparator that new drugs must beat.","summary":"Topotecan is a topoisomerase-I poison that stabilises the enzyme-DNA cleavage complex so replication forks break the DNA. Approved 1996 (IV) and 2007 (oral) for relapsed SCLC after platinum, it is given on days 1 to 5 of a 21-day cycle and also has roles in ovarian and cervical cancer. Activity is modest, with ORR around 20% and median OS around 6 to 8 months, and myelosuppression is heavy: grade 4 neutropenia in 70% of patients, so weekly blood counts and dose reductions are routine. It was the control arm in DeLLphi-304, where tarlatamab beat it with OS 13.6 versus 8.3 months, and is the comparator again in IDeate-Lung02; in ATLANTIS, lurbinectedin plus doxorubicin did not outperform it. Topotecan defines the floor for relapsed small-cell lung cancer, and new agents are judged by how far they rise above it.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Topotecan","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Topotecan"},{"label":"NICE TA184: topotecan for the treatment of relapsed small-cell lung cancer","url":"https://www.nice.org.uk/guidance/ta184"}],"tags":[],"related":[],"cancers":["sclc","ovarian","cervical","extensive-stage-sclc"],"sections":[],"technologies":["topoisomerase-inhibitors","cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["gsk","novartis"],"institutions":[],"pathways":[],"terms":[],"trials":["dellphi-304","atlantis","ideate-lung02","nct06828354","nct06801834","nct06459180","nct07604766","nct06496048","nct06619236","nct06128837","nct06500026","nct03709680","nct06449209","nct07099898","nct06679634","nct07145333","nct07286266","lurbinectedin-basket-sclc"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Hycamtin","modality":"Cytotoxic (topoisomerase-I inhibitor)","mechanism":"Topoisomerase-I poison; stabilises the cleavage complex causing replication-associated DNA breaks.","approvals":[{"region":"US","year":1996,"indication":"Relapsed SCLC (IV)"},{"region":"US","year":2007,"indication":"Relapsed SCLC (oral)"},{"region":"England (NICE)","year":2009,"indication":"Relapsed small-cell lung cancer, oral topotecan, only where re-treatment with the first-line regimen is inappropriate and cyclophosphamide, doxorubicin and vincristine is contraindicated","note":"TA184, published 25 November 2009. It remains the only NICE recommendation for a cytotoxic drug in relapsed small-cell lung cancer."}],"mechanismSteps":["Enters cell as active lactone","Traps TOP1 on DNA","Replication fork collision produces double-strand breaks","Apoptosis in S-phase cells"],"dosing":{"route":"Intravenous or oral","schedule":"1.5 mg/m² IV days 1-5 every 21 days, or 2.3 mg/m² oral days 1-5","modifications":"Frequent dose reduction for neutropenia; renal dose adjustment","monitoring":"Weekly blood counts"},"toxicity":[{"event":"Neutropenia (grade 4)","grade3PlusPct":70},{"event":"Thrombocytopenia (grade 4)","grade3PlusPct":27},{"event":"Anaemia","anyGradePct":90},{"event":"Diarrhoea (oral)","anyGradePct":30}],"access":[],"regulatoryEvents":[]},{"id":"toremifene","kind":"drug","name":"Toremifene","aka":[],"tldr":"Toremifene (Fareston) is a tamoxifen-like tablet for postmenopausal women with hormone-sensitive breast cancer that has spread.","summary":"Toremifene was approved by the FDA in May 1997 for metastatic breast cancer in postmenopausal women with oestrogen receptor-positive or unknown tumours, on three randomised trials (North American, Eastern European and Nordic) that showed response rates and time to progression similar to tamoxifen; the EU authorised Fareston in 1996 for first-line hormonal treatment of hormone-dependent metastatic breast cancer. It never displaced tamoxifen and aromatase inhibitors then moved ahead of both. QT prolongation carries a boxed warning; hot flushes, sweating and vaginal discharge are the common effects, with thromboembolism and endometrial changes as class risks.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Toremifene","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=toremifene"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/toremifene"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/fareston"}],"tags":["nci-list"],"related":["tamoxifen"],"cancers":["breast-hr-positive"],"sections":[],"technologies":["endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["kyowa-kirin","gtx"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00129142","nct03351062","nct02132390"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Fareston","modality":"Selective oestrogen receptor modulator (triphenylethylene)","mechanism":"Chlorinated tamoxifen analogue that binds the oestrogen receptor and acts as an antagonist in breast tissue with partial agonist effects elsewhere.","approvals":[{"region":"US","year":1997,"indication":"Metastatic breast cancer in postmenopausal women with ER-positive or unknown tumours"},{"region":"EU","year":1996,"indication":"First-line hormonal treatment of hormone-dependent metastatic breast cancer in postmenopausal women"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"toripalimab","kind":"drug","name":"Toripalimab","aka":[],"tldr":"A Chinese-developed PD-1 blocker that became the first immunotherapy approved in the US for nasopharyngeal cancer.","summary":"Approved by the FDA 27 October 2023 with gemcitabine-cisplatin for first-line recurrent or metastatic NPC and as monotherapy after platinum (JUPITER-02, POLARIS-02); EU approval 2024 for NPC and oesophageal squamous cell carcinoma. Broadly approved in China across lung, oesophageal, urothelial, and other cancers. Junshi Biosciences; Coherus in the US.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Toripalimab","links":[{"label":"FDA approval coverage","url":"https://www.onclive.com/view/fda-approves-toripalimab-for-recurrent-or-metastatic-nasopharyngeal-carcinoma"}],"tags":[],"related":[],"cancers":["head-and-neck","esophageal","nasopharyngeal","recurrent-metastatic-nasopharyngeal-carcinoma","locoregionally-advanced-nasopharyngeal-carcinoma","lung-cancer","resectable-nsclc"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["junshi","coherus"],"institutions":[],"pathways":[],"terms":["irae"],"trials":["jupiter-02","nct07258979","nct06679985","nct07169994","nct06623136","nct05980481","nct05302284","nct06155383","nct06227117","nct06389006","nct07111832","nct05518045","nct06038396","nct06868199","nct07392736","nct05934331","nct06178159","nct06117566","nct06823427","nct06592326","nct06682780","nct07314723","nct05615974","nct04785196","nct06975293","nct06079112","nct06088004","nct07281976","nct06642545","neotorch"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Loqtorzi","modality":"Monoclonal antibody (anti-PD-1)","mechanism":"Humanised IgG4 anti-PD-1 with a distinct epitope and slow off-rate.","approvals":[{"region":"US","year":2023,"indication":"Recurrent/metastatic nasopharyngeal carcinoma, first line with chemotherapy and later lines"},{"region":"China","year":2018,"indication":"Melanoma (first of many indications)"}],"mechanismSteps":["Binds PD-1 on T cells and blocks PD-L1/PD-L2 engagement","Restores cytotoxic activity against EBV-antigen-bearing NPC cells","Chemotherapy releases antigen and depletes suppressive cells, complementing the effect"],"dosing":{"route":"Intravenous","schedule":"240 mg every 3 weeks with gemcitabine-cisplatin for up to 6 cycles, then maintenance","monitoring":"Immune-related adverse events; thyroid function","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761240s000lbl.pdf"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2023-10-27","type":"approval","region":"US","note":"First FDA approval for nasopharyngeal carcinoma"}]},{"id":"tovecimig","kind":"drug","name":"Tovecimig","aka":[],"tldr":"Tovecimig is an experimental bispecific antibody from Compass Therapeutics in phase 3 trials for biliary tract cancer and bladder & urothelial cancer, aimed at VEGF / VEGFR.","summary":"Tovecimig (CTX-009) is a bispecific antibody developed by Compass Therapeutics. Its target is VEGF / VEGFR (the sponsor names DLL4 x VEGF-A). The sponsor states: Simultaneously targets Delta-like ligand 4 (DLL4) and VEGF-A to inhibit tumour angiogenesis. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05506943 (A Study of CTX-009 in Combination With Paclitaxel in Adult Patients With Unresectable Advanced, Metastatic or Recurrent Biliary Tract Cancers (COMPANION-002)), in biliary tract cancer and bladder & urothelial cancer. The largest, NCT05506943, plans to enrol 168 participants (actual) with primary completion expected 2027-08-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Tovecimig","url":"https://clinicaltrials.gov/search?intr=Tovecimig"},{"label":"Sponsor pipeline page","url":"https://www.compasstherapeutics.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cholangiocarcinoma","urothelial"],"sections":[],"technologies":[],"targets":["vegf"],"drugs":[],"companies":["compass-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["companion-002"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CTX-009","modality":"bispecific antibody","mechanism":"Simultaneously targets Delta-like ligand 4 (DLL4) and VEGF-A to inhibit tumour angiogenesis.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tovorafenib","kind":"drug","name":"Tovorafenib","aka":[],"tldr":"Tovorafenib is a pill for the most common childhood brain tumour, low-grade glioma driven by BRAF changes, approved in 2024.","summary":"FDA accelerated approval 23 April 2024 for relapsed/refractory paediatric low-grade glioma (age ≥6 months) with BRAF fusion/rearrangement or V600 mutation (FIREFLY-1: ORR ~51% by RANO-HGG). European conditional approval April 2026 (Ipsen, regardless of BRAF alteration type). Type II RAF inhibitor active against KIAA1549-BRAF fusions where type I inhibitors cause paradoxical activation. Phase 3 LOGGIPY-2 in first line versus chemotherapy. Day One Biopharmaceuticals.","status":"approved","asOf":"2026-09-06","links":[{"label":"FDA approval","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-tovorafenib-patients-relapsed-or-refractory-braf-altered-pediatric"}],"tags":[],"related":["braf-fusion"],"cancers":["glioblastoma","paediatric-low-grade-glioma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf"],"drugs":[],"companies":["day-one-biopharmaceuticals","ipsen"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct05566795"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ojemda","modality":"Small-molecule type II RAF inhibitor","mechanism":"Pan-RAF type II inhibitor binding the DFG-out conformation; blocks monomeric and dimeric BRAF signalling.","approvals":[{"region":"US","year":2024,"indication":"Relapsed/refractory BRAF-altered paediatric low-grade glioma (accelerated)"},{"region":"EU","year":2026,"indication":"Relapsed/refractory paediatric low-grade glioma (conditional)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2024-04-23","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 2.4 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-tovorafenib-patients-relapsed-or-refractory-braf-altered-pediatric","indication":"Patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation."}]},{"id":"tq-b3234","kind":"drug","name":"TQ-B3234","aka":[],"tldr":"TQ-B3234 is an experimental small-molecule drug from Chia Tai Tianqing Pharmaceutical in phase 3 trials for rare cancers of childhood, with its target not yet stated publicly.","summary":"TQ-B3234 is a small-molecule drug developed by Chia Tai Tianqing Pharmaceutical. The sponsor describes its target as MEK1/2 (mitogen-activated protein kinase 1/2), which OnCo does not yet have a target page for. The sponsor states: A selective MEK1/2 inhibitor, dosed as an oral capsule, that inhibits the MEK protein (an upstream regulator of the ERK pathway) and is being tested against placebo for symptomatic, inoperable plexiform neurofibromas in neurofibromatosis type 1. ClinicalTrials.gov describes the intervention as: TQ-B3234 is an antitumor molecular targeted drug, a selective mitogen-activated protein kinase 1 and 2 (MEK1/2) inhibitor. It primarily inhibits the mitogen-activated protein kinase (MEK) protein (an upstream regulator of the extracellular. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07407803 (Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas), in rare cancers of childhood. The largest, NCT07407803, plans to enrol 177 participants with primary completion expected 2027-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQ-B3234","url":"https://clinicaltrials.gov/search?intr=TQ-B3234"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["rare-childhood-cancers"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07407803"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"A selective MEK1/2 inhibitor, dosed as an oral capsule, that inhibits the MEK protein (an upstream regulator of the ERK pathway) and is being tested against placebo for symptomatic, inoperable plexiform neurofibromas in neurofibromatosis type 1.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb2102","kind":"drug","name":"TQB2102","aka":[],"tldr":"TQB2102 is an experimental antibody-drug conjugate from Chia Tai Tianqing Pharmaceutical Nanjing Shunxin Pharmaceutical in phase 3 trials for HR-positive / HER2-negative breast cancer, HER2-positive breast cancer and biliary tract cancer, aimed at HER2.","summary":"TQB2102 is an antibody-drug conjugate developed by Chia Tai Tianqing Pharmaceutical Nanjing Shunxin Pharmaceutical and Chia Tai Tianqing Pharmaceutical. Its target is HER2 (the sponsor names HER2). The sponsor states: Described on the study page as a next-generation HER2 antibody-drug conjugate carrying the small-molecule toxin TQ22723. ClinicalTrials.gov describes the intervention as: TQB2102 is a next-generation HER2 Antibody-Drug Conjugate drug proposed for patients with HER2 positive Recurrent/Metastatic Breast Cancer. It is the investigational product in 8 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07003074 (A Clinical Study of TQB2102 Versus Docetaxel Plus Trastuzumab and Pertuzumab in the Treatment of HER2 Positive Recurrent or Metastatic Breast Cancer), NCT06561607 (A Clinical Trial of TQB2102 for Injection in the Treatment of HER2 Low-Expressing Recurrent/Metastatic Breast Cancer) and NCT07008976 (Clinical Trial of TQB2102 for Injection Versus Trastuzumab Emtansine for Injection in HER2-positive Advanced Breast Cancer), in HR-positive / HER2-negative breast cancer, HER2-positive breast cancer, biliary tract cancer and non-small-cell lung cancer. The largest, NCT07003074, plans to enrol 642 participants with primary completion expected 2029-07. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQB2102","url":"https://clinicaltrials.gov/search?intr=TQB2102"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive","breast-her2-positive","cholangiocarcinoma","nsclc"],"sections":[],"technologies":[],"targets":["her2"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07003074","nct06561607","nct07008976","nct07043725","nct06496490","nct07512583","nct06431490","nct06452706","nct06198751"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Described on the study page as a next-generation HER2 antibody-drug conjugate carrying the small-molecule toxin TQ22723.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb2450","kind":"drug","name":"TQB2450","aka":[],"tldr":"TQB2450 is an experimental monoclonal antibody from Chia Tai Tianqing Pharmaceutical in phase 3 trials for renal cell carcinoma and non-small-cell lung cancer, aimed at PD-L1 and PD-1.","summary":"TQB2450 is a monoclonal antibody developed by Chia Tai Tianqing Pharmaceutical. Its targets are PD-L1 and PD-1 (the sponsor names PD-L1). The sponsor states: TQB2450 is a humanised monoclonal antibody targeting programmed death ligand-1 (PD-L1), which prevents PD-L1 binding to PD-1 and B7.1 receptors on T cells, restoring T-cell activity and enhancing immune response. ClinicalTrials.gov describes the intervention as: TQB2450 is a humanized monoclonal antibody targeting programmed death ligand-1 (PD-L1), which prevents PD-L1 from binding to PD-1 and B7.1 receptors on T cell surface, restores T cell activity, thus enhancing immune response and has potenti. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT04523272 (A Study of TQB2450 Injection Combined With Anlotinib Hydrochloride Capsule Versus Sunitinib in Subjects With Advanced Renal Cancer), in renal cell carcinoma and non-small-cell lung cancer. The largest, NCT04523272, plans to enrol 528 participants (actual) with primary completion was scheduled for 2025-06 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQB2450","url":"https://clinicaltrials.gov/search?intr=TQB2450"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["rcc","nsclc"],"sections":[],"technologies":[],"targets":["pdl1","pd1"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04523272","nct05913089"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody","mechanism":"TQB2450 is a humanised monoclonal antibody targeting programmed death ligand-1 (PD-L1), which prevents PD-L1 binding to PD-1 and B7.1 receptors on T cells, restoring T-cell activity and enhancing immune response.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb2825","kind":"drug","name":"TQB2825","aka":[],"tldr":"TQB2825 is an experimental bispecific antibody from Chia Tai Tianqing Pharmaceutical in phase 2 trials for diffuse large B-cell lymphoma, aimed at CD20 and CD3.","summary":"TQB2825 is a bispecific antibody developed by Chia Tai Tianqing Pharmaceutical and Shanghai Chia Tai Tianqing Pharmaceutical Technology Development. Its targets are CD20 and CD3 (the sponsor names CD3 x CD20). ClinicalTrials.gov describes the intervention as: Drug: TQB2825 Injection + Gemcitabine Hydrochloride for Injection + Oxaliplatin for Injection; Other Name: Gemcitabine Hydrochloride for Injection, Zefei; Oxaliplatin for Injection, Aihen TQB2825 injection is Cluster of Differentiation 3 (C. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in diffuse large B-cell lymphoma. The largest, NCT06829771, plans to enrol 90 participants with primary completion was scheduled for 2026-05 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQB2825","url":"https://clinicaltrials.gov/search?intr=TQB2825"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":[],"targets":["cd20","cd3"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06829771","nct06854445"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"Bispecific antibody directed at CD20 and CD3, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb2868","kind":"drug","name":"TQB2868","aka":[],"tldr":"TQB2868 is a bispecific antibody from Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd., in registered phase 2 trials for pancreatic ductal adenocarcinoma.","summary":"TQB2868 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Chia Tai Tianqing Pharmaceutical Group Nanjing Shunxin Pharmaceutical Co., Ltd., in pancreatic ductal adenocarcinoma. A bispecific antibody, as described in the registry record: one molecule binds two different targets, most often a tumour antigen and an immune cell or a second tumour pathway. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of TQB2868","url":"https://clinicaltrials.gov/search?intr=TQB2868"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06767813"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"TQB2868","modality":"Bispecific antibody","mechanism":"A bispecific antibody, as described in the registry record: one molecule binds two different targets, most often a tumour antigen and an immune cell or a second tumour pathway.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb2922","kind":"drug","name":"TQB2922","aka":[],"tldr":"TQB2922 is an experimental bispecific antibody from Chia Tai Tianqing Pharmaceutical Nanjing Shunxin Pharmaceutical in phase 2 trials for colorectal cancer, aimed at EGFR and MET.","summary":"TQB2922 is a bispecific antibody developed by Chia Tai Tianqing Pharmaceutical Nanjing Shunxin Pharmaceutical. Its targets are EGFR and MET (the sponsor names EGFR x c-Met). The sponsor states: TQB2922 is an anti-EGFR/c-Met bispecific IgG1 antibody that blocks EGFR and c-Met signalling by binding both receptors on tumour cells and engages antibody-dependent cellular cytotoxicity and phagocytosis via NK cells and macrophages. ClinicalTrials.gov describes the intervention as: TQB2922 is an anti-EGFR/c-Met bispecific antibody, subtype Immunoglobulin G1 (IgG1). TQB2922 blocks the activation of EGFR and c-Met signalling pathway by binding to EGFR and c-Met on the surface of tumour cells, thus preventing tumour grow. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in colorectal cancer. The largest, NCT07044908, plans to enrol 72 participants with primary completion was scheduled for 2026-07 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQB2922","url":"https://clinicaltrials.gov/search?intr=TQB2922"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["egfr","met"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07044908"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"TQB2922 is an anti-EGFR/c-Met bispecific IgG1 antibody that blocks EGFR and c-Met signalling by binding both receptors on tumour cells and engages antibody-dependent cellular cytotoxicity and phagocytosis via NK cells and macrophages.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb2930","kind":"drug","name":"TQB2930","aka":[],"tldr":"TQB2930 is an experimental bispecific antibody from Chia Tai Tianqing Pharmaceutical in phase 2 trials for HR-positive / HER2-negative breast cancer, aimed at HER2 and EGFR.","summary":"TQB2930 is a bispecific antibody developed by Chia Tai Tianqing Pharmaceutical and Chia Tai Tianqing Pharmaceutical Nanjing Shunxin Pharmaceutical. Its targets are HER2 and EGFR (the sponsor names HER2). The sponsor states: TQB2930 is a HER2 bispecific antibody given by intravenous infusion, being studied as monotherapy or combination therapy in HER2-positive recurrent/metastatic breast cancer. ClinicalTrials.gov describes the intervention as: TQB2930 for injection is a HER2 bispecific antibody. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in HR-positive / HER2-negative breast cancer. The largest, NCT07512583, plans to enrol 178 participants with primary completion expected 2028-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQB2930","url":"https://clinicaltrials.gov/search?intr=TQB2930"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":["her2","egfr"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06202261","nct07512583","nct07047365"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"bispecific antibody","mechanism":"TQB2930 is a HER2 bispecific antibody given by intravenous infusion, being studied as monotherapy or combination therapy in HER2-positive recurrent/metastatic breast cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb3454","kind":"drug","name":"TQB3454","aka":[],"tldr":"TQB3454 is an experimental small-molecule drug from Chia Tai Tianqing Pharmaceutical in phase 3 trials for biliary tract cancer, aimed at IDH1 / IDH2.","summary":"TQB3454 is a small-molecule drug developed by Chia Tai Tianqing Pharmaceutical. Its target is IDH1 / IDH2 (the sponsor names IDH1 (mutant)). The sponsor states: Selective inhibitor of mutant IDH1 enzyme, given as an oral tablet, being tested against placebo in advanced biliary tract cancer patients with IDH1 mutations. ClinicalTrials.gov describes the intervention as: TQB3454 is a selective IDH1 mutant enzyme inhibitor. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT05987358 (A Clinical Study of TQB3454 Tablets in the Treatment of Advanced Biliary Carcinoma), in biliary tract cancer. The largest, NCT05987358, plans to enrol 165 participants with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQB3454","url":"https://clinicaltrials.gov/search?intr=TQB3454"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["cholangiocarcinoma"],"sections":[],"technologies":[],"targets":["idh"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05987358"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Selective inhibitor of mutant IDH1 enzyme, given as an oral tablet, being tested against placebo in advanced biliary tract cancer patients with IDH1 mutations.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb3616","kind":"drug","name":"TQB3616","aka":[],"tldr":"TQB3616 is an experimental small-molecule drug from Chia Tai Tianqing Pharmaceutical in phase 3 trials for HR-positive / HER2-negative breast cancer, aimed at CDK4/6.","summary":"TQB3616 is a small-molecule drug developed by Chia Tai Tianqing Pharmaceutical. Its target is CDK4/6 (the sponsor names CDK4/6). The sponsor states: TQB3616 is a novel oral cyclin-dependent kinase (CDK) 4/6 inhibitor used in the treatment of various malignant solid tumours. ClinicalTrials.gov describes the intervention as: TQB3616 capsule is a Cyclin-dependent kinase 4/6 (CDK4/6) inhibitor. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05780567 (Clinical Study on Adjuvant Therapy of TQB3616 Combined With Endocrine Therapy Compared With Placebo Combined With Endocrine Therapy in Patients With Breast Cancer) and NCT05365178 (To Evaluate the Efficacy and Safety of TQB3616 in Combination With Fulvestrant Versus Placebo in Combination With Fulvestrant in Previously Untreated Hormone-receptor (HR)-Positive, Human Epidermal Growth Factor Receptor 2 (HER2)-Negative Advanced Breast Cancer), in HR-positive / HER2-negative breast cancer. The largest, NCT05780567, plans to enrol 1946 participants with primary completion expected 2026-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQB3616","url":"https://clinicaltrials.gov/search?intr=TQB3616"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":["cdk4-6"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05780567","nct05365178","nct06202261","nct06702618"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"TQB3616 is a novel oral cyclin-dependent kinase (CDK) 4/6 inhibitor used in the treatment of various malignant solid tumours.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb3702","kind":"drug","name":"TQB3702","aka":[],"tldr":"TQB3702 is a small-molecule inhibitor from Chia Tai Tianqing Pharmaceutical Group Co., Ltd., in registered phase 2 trials for non-Hodgkin lymphoma.","summary":"TQB3702 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Chia Tai Tianqing Pharmaceutical Group Co., Ltd., in non-Hodgkin lymphoma. Described in the registry record as a tyrosine kinase inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of TQB3702","url":"https://clinicaltrials.gov/search?intr=TQB3702"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["non-hodgkin-lymphoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06566586"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"TQB3702","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a tyrosine kinase inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb3909","kind":"drug","name":"TQB3909","aka":[],"tldr":"TQB3909 is a small-molecule inhibitor from Chia Tai Tianqing Pharmaceutical Group Co., Ltd., in registered phase 2 trials for metastatic cancer.","summary":"TQB3909 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Chia Tai Tianqing Pharmaceutical Group Co., Ltd., in metastatic cancer. Described in the registry record as a tqb3909 is a protein inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of TQB3909","url":"https://clinicaltrials.gov/search?intr=TQB3909"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07011186"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"TQB3909","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a tqb3909 is a protein inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tqb6411","kind":"drug","name":"TQB6411","aka":[],"tldr":"TQB6411 is an experimental antibody-drug conjugate from Chia Tai Tianqing Pharmaceutical in phase 2 trials for non-small-cell lung cancer and oesophageal cancer, aimed at EGFR and MET.","summary":"TQB6411 is an antibody-drug conjugate developed by Chia Tai Tianqing Pharmaceutical. Its targets are EGFR and MET. ClinicalTrials.gov describes the intervention as: TQB6411 for Injection is administered every 21 days as a treatment cycle. TQB6411 for injection is administered every 28 days as a treatment cycle. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in non-small-cell lung cancer and oesophageal cancer. The largest, NCT07367529, plans to enrol 465 participants with primary completion expected 2028-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TQB6411","url":"https://clinicaltrials.gov/search?intr=TQB6411"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","esophageal"],"sections":[],"technologies":[],"targets":["egfr","met"],"drugs":[],"companies":["sino-biopharm"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07367516","nct07367529"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Antibody-drug conjugate directed at EGFR and MET, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trabectedin","kind":"drug","name":"Trabectedin","aka":[],"tldr":"A chemotherapy derived from a sea squirt, used with liposomal doxorubicin in relapsed ovarian cancer in Europe and for sarcomas.","summary":"Trabectedin is a cytotoxic derived from a sea squirt that binds the DNA minor groove, bends the DNA, blocks transcription-coupled repair and modulates tumour-associated macrophages. In ovarian cancer, OVA-301 showed a PFS benefit with pegylated liposomal doxorubicin in partially platinum-sensitive relapse; the EU approved the combination in 2009, the US never did for this indication, and INOVATYON found no OS advantage over platinum. In sarcoma it is approved in the EU (2007) after anthracycline or ifosfamide and in the US (2015) for liposarcoma and leiomyosarcoma after anthracycline. Grade 3 to 4 neutropenia and ALT elevation are the main toxicities, so liver function is monitored. It is a niche chemotherapy with a distinctive mechanism, most valuable in specific sarcoma subtypes.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Trabectedin","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Trabectedin"}],"tags":[],"related":[],"cancers":["ovarian","sarcoma","leiomyosarcoma","liposarcoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["johnson-johnson"],"institutions":[],"pathways":[],"terms":["sarcoma-histotype-tailoring"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In sarcoma: ET743-SAR-3007 gave PFS 4.2 versus 1.5 months against dacarbazine (HR 0.55) with no OS difference, and US approval followed in October 2015 for liposarcoma and leiomyosarcoma; it is particularly active in myxoid liposarcoma (FUS-DDIT3), where it displaces the FUS-CHOP oncoprotein. Given as a 24-hour infusion of 1.5 mg/m2 every 3 weeks with dexamethasone premedication through a central line; grade 3 or higher ALT elevation in 26 percent and neutropenia in 37 percent, with rare rhabdomyolysis."],"brand":"Yondelis","modality":"Cytotoxic (DNA minor-groove binder)","mechanism":"Binds the DNA minor groove, bends DNA, blocks transcription-coupled repair, and modulates tumour-associated macrophages.","approvals":[{"region":"EU","year":2009,"indication":"Relapsed platinum-sensitive ovarian cancer with PLD"},{"region":"US","year":2015,"indication":"Liposarcoma and leiomyosarcoma after anthracycline"},{"region":"EU","year":2007,"indication":"Advanced soft-tissue sarcoma after anthracycline/ifosfamide"}],"mechanismSteps":[],"dosing":{"route":"Intravenous (central line)","schedule":"1.1 mg/m² with PLD every 3 weeks (ovarian, EU); 1.5 mg/m² 24-hour infusion every 3 weeks (sarcoma)","monitoring":"Liver function, CPK, CBC; dexamethasone premedication"},"toxicity":[{"event":"Neutropenia grade 3-4","grade3PlusPct":63,"note":"OVA-301 combination arm"},{"event":"ALT increase grade 3-4","grade3PlusPct":31}],"access":[],"regulatoryEvents":[]},{"id":"trabedersen","kind":"drug","name":"Trabedersen","aka":[],"tldr":"Trabedersen is an experimental investigational agent whose form is not stated in the registry from Oncotelic in phase 3 trials for pancreatic ductal adenocarcinoma, with its target not yet stated publicly.","summary":"Trabedersen (OT-101) is an investigational agent whose form is not stated in the registry developed by Oncotelic. The sponsor describes its target as TGF-beta2, which OnCo does not yet have a target page for. The sponsor states: An antisense oligodeoxynucleotide complementary to TGF-beta2 mRNA; by suppressing TGF-beta2, which cancers overexpress to cloak themselves from the immune system, it is intended to let innate or therapeutic immunity attack and eliminate the tumour. ClinicalTrials.gov describes the intervention as: OT-101: antisense oligodeoxynucleotide complementary to the messenger ribonucleic acid (mRNA) of the human TGF-β2 gene. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06079346 (A Study of OT-101 With mFOLFIRINOX in Patients With Advanced and Unresectable or Metastatic Pancreatic Cancer), in pancreatic ductal adenocarcinoma. The largest, NCT06079346, plans to enrol 455 participants with primary completion was scheduled for 2026-06-01 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Trabedersen","url":"https://clinicaltrials.gov/search?intr=Trabedersen"},{"label":"Sponsor pipeline page","url":"https://www.oncotelic.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["isarna-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06079346"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"OT-101","modality":"antisense oligonucleotide (per the sponsor's description; form not stated in the registry record)","mechanism":"An antisense oligodeoxynucleotide complementary to TGF-beta2 mRNA; by suppressing TGF-beta2, which cancers overexpress to cloak themselves from the immune system, it is intended to let innate or therapeutic immunity attack and eliminate the tumour.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trametinib","kind":"drug","name":"Trametinib","aka":[],"tldr":"Trametinib (Mekinist) blocks MEK, the protein one step below BRAF in the growth-signal chain. Paired with dabrafenib it treats BRAF-mutant melanoma, lung, thyroid and other cancers, including brain tumours in children.","summary":"Trametinib was approved by the FDA in May 2013 as a single agent for BRAF V600E or V600K metastatic melanoma (METRIC: progression-free survival superior to chemotherapy) and in January 2014 in combination with dabrafenib, the pairing that became standard after COMBI-d and COMBI-v. All later indications are with dabrafenib: adjuvant stage III melanoma (COMBI-AD), BRAF V600E metastatic NSCLC, anaplastic thyroid cancer, tumour-agnostic BRAF V600E solid tumours and paediatric low-grade glioma with an oral solution (2023). The EU authorised Mekinist in 2014. Rash, diarrhoea, reduced ejection fraction, retinal vein occlusion and central serous retinopathy are the MEK-class toxicities; pyrexia dominates the combination.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Trametinib","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=trametinib"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/trametinib"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/mekinist"}],"tags":["nci-list"],"related":["dabrafenib","dabrafenib-trametinib","braf-v600e"],"cancers":["melanoma","nsclc","thyroid","anaplastic-thyroid-cancer","low-grade-serous-ovarian-cancer","paediatric-low-grade-glioma","paediatric-high-grade-glioma","braf-v600e-nsclc","braf-v600-melanoma","stage-iii-melanoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["mek"],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["combi-ad","nct04940052","nct05372354","nct05358249","nct04417621","nct03899155","nct05874414"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Mekinist","modality":"Small-molecule allosteric MEK1/2 inhibitor","mechanism":"Reversible, non-ATP-competitive inhibitor of MEK1 and MEK2 kinase activity and activation, blocking ERK phosphorylation downstream of BRAF.","approvals":[{"region":"US","year":2013,"indication":"BRAF V600E/K unresectable or metastatic melanoma (single agent)"},{"region":"US","year":2014,"indication":"With dabrafenib: metastatic melanoma; later adjuvant melanoma, NSCLC, anaplastic thyroid cancer, tumour-agnostic BRAF V600E solid tumours, paediatric low-grade glioma"},{"region":"EU","year":2014,"indication":"BRAF V600 melanoma alone or with dabrafenib; later NSCLC and adjuvant melanoma"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2014-01-08","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"In combination with dabrafenib for unresectable or metastatic melanoma with BRAF V600E or V600K mutations, as detected by an FDA-approved test"},{"date":"2015-11-20","type":"conversion","region":"US","note":"Confirmed: the accelerated approval of 2014 converted to traditional approval 1.9 years after it was granted.","indication":"In combination with dabrafenib for unresectable or metastatic melanoma with BRAF V600E or V600K mutations, as detected by an FDA-approved test"},{"date":"2022-06-22","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 4.2 years later, when the FDA's table was read.","source":"https://www.fda.gov#endnote2","indication":"In combination with dabrafenib for adult and pediatric patients 6 years of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options *"}]},{"id":"transpara","kind":"drug","name":"Transpara","aka":[],"tldr":"The breast screening AI tested in Sweden's MASAI trial, where AI-supported reading found more cancers and nearly halved the radiologists' workload.","summary":"Transpara (ScreenPoint Medical, Nijmegen) is CE-marked and FDA 510(k)-cleared for 2D mammography (2018) and digital breast tomosynthesis (2020). It was the software used in the MASAI randomised trial of 80,000 women in Sweden: the interim analysis (Lancet Oncology 2023) reported a cancer detection rate of 6.1 per 1,000 with AI-supported screening versus 5.1 per 1,000 with standard double reading, similar recall rates, and a 44.3% reduction in screen-reading workload. Transpara is deployed in screening programmes in Sweden, Denmark, Germany and elsewhere, and its use as one of two readers is the model the NHS is evaluating in the EDITH trial.","status":"approved","asOf":"2026-09-10","links":[{"label":"MASAI interim analysis (Lancet Oncol 2023)","url":"https://doi.org/10.1016/S1470-2045(23)00298-X"}],"tags":["test"],"related":[],"cancers":["breast-hr-positive","breast-her2-positive","tnbc"],"sections":[],"technologies":["radiology-ai-screening","mammography"],"targets":[],"drugs":[],"companies":["screenpoint-medical"],"institutions":[],"pathways":[],"terms":[],"trials":["masai"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: the AI does not give a diagnosis; a high score prompts closer radiologist review or recall for further imaging, and a low score can let a programme use one human reader instead of two."],"brand":"Transpara","modality":"AI mammography detection and decision-support software","mechanism":"Deep-learning system scores each mammogram or tomosynthesis exam from 1 to 10 for cancer likelihood and marks suspicious regions, usable as a concurrent reader, second reader or triage tool.","approvals":[{"region":"EU","year":2017,"indication":"CE mark, mammography decision support"},{"region":"US","year":2018,"indication":"510(k) clearance, 2D mammography"},{"region":"US","year":2020,"indication":"510(k) clearance, digital breast tomosynthesis"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trastuzumab","kind":"drug","name":"Trastuzumab","aka":["Trastuzumab and Hyaluronidase-oysk","Herceptin Hylecta"],"tldr":"The first targeted antibody for a solid tumour (1998), which turned HER2-positive breast cancer from the worst subtype into one of the most treatable.","summary":"Trastuzumab is a humanised IgG1 antibody that binds domain IV of HER2, blocking receptor signalling and recruiting immune cells through ADCC. It was the first targeted antibody for a solid tumour, approved for HER2-positive metastatic breast cancer in 1998, as adjuvant therapy in 2006 (HERA, NSABP B-31/N9831, about a 37% reduction in death) and for HER2-positive gastric cancer in 2010. Dual blockade with pertuzumab (CLEOPATRA, APHINITY) and the subcutaneous Phesgo built on it, and it is the antibody backbone of the conjugates T-DM1 and T-DXd. Adjuvant treatment lasts 1 year, and cardiomyopathy requires LVEF monitoring. Biosimilars since 2017 have cut cost worldwide, and whether shorter adjuvant courses are safe for lower-risk patients remains debated. For a newcomer: the drug that turned the worst breast cancer subtype into one of the most treatable.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Trastuzumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Trastuzumab"}],"tags":[],"related":["tucatinib-triplet-brain-mets","her2-ihc-3-plus","her2-ish-amplified"],"cancers":["breast-her2-positive","gastric","gastric-her2-positive","salivary-duct-carcinoma","her2-positive-early-breast-cancer","endometrial-p53-abnormal","colorectal","gallbladder","biliary-tract-cancer"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["her2"],"drugs":[],"companies":["roche-genentech","nippon-kayaku","halozyme"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07518173","nct06435429","nct07196774","nct06891833","nct04430738","nct05081609","nct06324357","nct07102381","nct06589830","nct05458674","nct05980481","nct06731478","nct03379428","nct07497386","nct05785741","nct06057610","nct06764875","nct04208178","nct06771622","nct04873362","nct07047365","nct00781612","nct07007559","nct06686394","nct02320435","nct05417594","nct07315750","nct04379596","nct07069712","nct07679360","nct04721977","nct04579380","mountaineer","nct07140393"],"people":[],"bottlenecks":[],"keyPapers":["paper-sartore-bianchi-heracles-trastuzumab-lapatinib-lancet-oncol-2016","paper-toga-trastuzumab-gastric-lancet-2010","paper-valtorta-her2-scoring-colorectal-heracles-mod-pathol-2015","paper-bertotti-xenopatients-her2-cetuximab-resistant-colorectal-cancer-discov-2011","paper-javle-mypathway-her2-biliary-lancet-oncol-2021"],"journals":[],"dependsOn":[],"notes":["Colorectal cancer biomarkers: HER2 positivity by the HERACLES criteria, intense membranous staining in more than 50% of cells corresponding to homogeneous amplification, in a RAS wild-type tumour. That is about 5% of RAS wild-type patients, so roughly 20 must be screened to find one (Valtorta 2015, Sartore-Bianchi 2016). The combination with lapatinib gave 8 responses in 27 patients and has since been superseded by tucatinib plus trastuzumab and trastuzumab deruxtecan.","Biliary tract cancer: with pertuzumab, objective response rate 23% (9 of 39) in previously treated HER2-positive disease in the MyPathway basket (Javle 2021); off-label."],"brand":"Herceptin (and biosimilars, Phesgo with pertuzumab)","modality":"Monoclonal antibody (anti-HER2)","mechanism":"Humanised IgG1 binding HER2 ECD4; signal inhibition and ADCC.","approvals":[{"region":"US","year":1998,"indication":"HER2+ metastatic breast cancer"},{"region":"US","year":2006,"indication":"Adjuvant HER2+ breast cancer"},{"region":"US","year":2010,"indication":"HER2+ gastric cancer"}],"mechanismSteps":["Antibody binds domain IV of HER2","HER2 signalling and shedding are inhibited; receptor internalisation increases","Fc engages NK cells: antibody-dependent cellular cytotoxicity","HER2-amplified cells stop proliferating and are cleared","With pertuzumab, dimerisation with HER3 is also blocked"],"dosing":{"route":"IV infusion (subcutaneous Herceptin Hylecta / Phesgo with pertuzumab available)","schedule":"8 mg/kg loading then 6 mg/kg every 3 weeks (or 4 then 2 mg/kg weekly); 1 year adjuvant","modifications":"Hold for LVEF drop ≥16 points or below normal","monitoring":"LVEF at baseline and every 3 months during therapy; infusion reactions","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Cardiomyopathy / LVEF decline","note":"Adjuvant trials: ~2-4% symptomatic heart failure with anthracyclines"},{"event":"Infusion reactions"},{"event":"Fever"},{"event":"Nausea"},{"event":"Diarrhoea"},{"event":"Pulmonary toxicity (rare)"}],"access":[{"country":"US","reimbursement":"Medicare Part B; five biosimilars available since 2019, roughly 15-35% below reference price","assistance":"https://www.genentech-access.com","generic":true,"source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: standard of care; biosimilars widely used","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"1998-09-25","type":"approval","region":"US","note":"HER2+ metastatic breast cancer: first targeted antibody for a solid tumour","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2006-11-16","type":"approval","region":"US","note":"Adjuvant HER2+ early breast cancer (HERA, NSABP B-31/N9831)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2010-10-20","type":"approval","region":"US","note":"HER2+ metastatic gastric cancer (ToGA)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2017-12-01","type":"approval","region":"US","note":"First trastuzumab biosimilar (Ogivri)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2019-02-28","type":"approval","region":"US","note":"Subcutaneous trastuzumab-hyaluronidase (Herceptin Hylecta)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-06-29","type":"approval","region":"US","note":"Phesgo (pertuzumab, trastuzumab, hyaluronidase) subcutaneous","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-06-24","type":"approval","region":"US","note":"FDA approves palbociclib with trastuzumab, with or without pertuzumab, and endocrine therapy for the maintenance treatment of HR-positive, HER2-positive metastatic breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-palbociclib-trastuzumab-or-without-pertuzumab-and-endocrine-therapy-maintenance"}]},{"id":"trastuzumab-biosimilars","kind":"drug","name":"Trastuzumab biosimilars","aka":[],"tldr":"Near-identical copies of Herceptin, approved since 2017, that cut the price of HER2 treatment and widened access worldwide.","summary":"Six US-approved biosimilars (first: Ogivri, December 2017) with equivalence shown on pCR or ORR endpoints; WHO prequalification (2019) enabled low- and middle-income access. Biosimilar uptake exceeds 80% of trastuzumab volume in the US and EU; they are the trastuzumab component of many chemotherapy-antibody regimens and control arms.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Biosimilar","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Trastuzumab%20biosimilars"}],"tags":[],"related":[],"cancers":["breast-her2-positive","gastric"],"sections":[],"technologies":["monoclonal-antibody"],"targets":["her2"],"drugs":[],"companies":["organon"],"institutions":[],"pathways":[],"terms":["biosimilar"],"trials":["nct01275677","nct00769379","nct02003209"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ogivri, Herzuma, Kanjinti, Trazimera, Ontruzant, Hercessi","modality":"Biosimilar monoclonal antibody (anti-HER2)","mechanism":"Same amino acid sequence and mechanism as trastuzumab; analytical and clinical equivalence demonstrated.","approvals":[{"region":"US","year":2017,"indication":"First trastuzumab biosimilar (Ogivri); all trastuzumab indications"},{"region":"EU","year":2017,"indication":"Ontruzant, first EU trastuzumab biosimilar"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trastuzumab-brengitecan","kind":"drug","name":"Trastuzumab brengitecan","aka":[],"tldr":"SystImmune's HER2 ADC, sharing its payload with iza-bren, now in a 1,450-patient trial to replace Kadcyla after surgery.","summary":"Trastuzumab brengitecan is SystImmune's HER2 antibody-drug conjugate: an anti-HER2 antibody joined through a cleavable linker to the payload Ed-04, which can cross into neighbouring cells (bystander effect) and is shared with the company's iza-bren. Phase 1 data (ESMO 2025; Lancet-family 2026) showed responses in both HER2-positive and HER2-low breast cancer. A phase 3 trial of about 1,450 patients is enrolling in China, comparing it with T-DM1 in patients with residual disease after neoadjuvant therapy (NCT06830889). That is the same setting DESTINY-Breast05 has just redefined with T-DXd (3-year invasive disease-free survival 92.4% versus 83.7%, HR 0.47), so the trial's relevance depends on whether T-DM1 remains a fair comparator. For a newcomer, it is a Chinese HER2 ADC trying to replace Kadcyla after surgery.","status":"phase-3","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov NCT04622319 (DESTINY-Breast05)","url":"https://clinicaltrials.gov/study/NCT04622319"}],"tags":[],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":["adc","topoisomerase-inhibitors"],"targets":["her2"],"drugs":[],"companies":["systimmune"],"institutions":[],"pathways":[],"terms":[],"trials":["destiny-breast05","nct07518173","nct06830889","nct06316531","nct07178795","nct06891833","nct06114511","nct06445400","nct07264816"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"BL-M07D1, T-Bren","modality":"ADC","payload":"Ed-04 (camptothecin-derived TOP1 inhibitor), DAR ~8","linker":"Cathepsin B-cleavable","mechanism":"Anti-HER2 antibody with cleavable linker and Ed-04 payload (bystander-capable).","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2025-02","type":"filing","region":"China","note":"Phase 3 vs T-DM1 in residual disease registered (NCT06830889)","source":"https://clinicaltrials.gov/study/NCT06830889"}]},{"id":"trastuzumab-deruxtecan","kind":"drug","name":"Trastuzumab deruxtecan","aka":[],"tldr":"Trastuzumab deruxtecan (Enhertu) is the most successful ADC ever. It redefined HER2 by working in tumours with only tiny amounts of the protein, and in 2026 moved into early-stage breast cancer.","summary":"Approved in HER2+ metastatic breast cancer (DESTINY-Breast03: beat T-DM1), HER2-low (DESTINY-Breast04, 2022) and HER2-ultralow (DESTINY-Breast06, 2025) HR+ breast cancer, HER2+ gastric, HER2-mutant NSCLC, and tumour-agnostically for HER2 IHC 3+ solid tumours (2024). DESTINY-Breast09 (with pertuzumab) established it in first-line HER2+ metastatic disease. In Q2 2026 the FDA approved two early-stage HER2+ indications (neoadjuvant DESTINY-Breast11; post-neoadjuvant DESTINY-Breast05). Interstitial lung disease (~10-15%, ~1% fatal) requires monitoring. Membrane-permeable payload gives a strong bystander effect.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Trastuzumab_deruxtecan","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Trastuzumab%20deruxtecan"},{"label":"NICE TA992: trastuzumab deruxtecan for HER2-low metastatic or unresectable breast cancer after chemotherapy, not recommended (29 July 2024)","url":"https://www.nice.org.uk/guidance/ta992"},{"label":"DESTINY-Breast04 long-term survival analysis (Nature Medicine 2025)","url":"https://doi.org/10.1038/s41591-025-03981-4"},{"label":"Enhertu label (openFDA): HER2-low and HER2-ultralow indications, DESTINY-Breast04 study section","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22ENHERTU%22"},{"label":"FDA oncology approval notifications","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications"}],"tags":[],"related":["her2-ihc-3-plus","her2-ihc-2-plus","her2-ihc-1-plus","her2-ihc-0","her2-low-ihc","her2-ultralow","her2-ish-amplified","her2-mutation"],"cancers":["breast-her2-positive","breast-hr-positive","gastric","gastric-her2-positive","nsclc","colorectal","oesophageal-adenocarcinoma","salivary-duct-carcinoma","her2-amplified-colorectal","her2-low-metastatic-breast-cancer","her2-positive-early-breast-cancer","her2-positive-breast-brain-metastases","her2-mutant-nsclc","advanced-small-bowel-adenocarcinoma","tnbc","tnbc-metastatic","gallbladder","biliary-tract-cancer"],"sections":[],"technologies":["adc","topoisomerase-inhibitors"],"targets":["her2"],"drugs":[],"companies":["daiichi-sankyo","astrazeneca"],"institutions":[],"pathways":[],"terms":["her2-low","ild","bystander-effect","ggfg","her2-testing-in-biliary-cancer"],"trials":["destiny-breast03","destiny-breast04","destiny-breast06","destiny-breast09","destiny-breast11","nct06899126","nct06989112","nct06819007","nct06174987","nct05048797","nct07022483","nct04639219","nct04644068","nct06324357","nct05246514","nct05824975","nct06525298","nct06731478","nct07497386","nct06764875","nct07060807","nct04538742","nct06467357","nct05417594","nct04379596","nct07069712","nct05950945","nct06271837","nct03742102","nct06832202","destiny-crc01","destiny-pantumor02"],"people":[],"bottlenecks":[],"keyPapers":["paper-destiny-breast06-nejm-2024","paper-destiny-gastric01-nejm-2020","paper-destiny-lung01-nejm-2022","paper-destiny-breast04-nejm-2022","paper-schettini-her2-low-features-npj-breast-cancer-2021","paper-boissiere-michot-her2-ultralow-tnbc-virchows-arch-2026","paper-valtorta-her2-scoring-colorectal-heracles-mod-pathol-2015","paper-ohba-herb-trastuzumab-deruxtecan-biliary-jco-2024","paper-oh-destiny-pantumor02-biliary-pancreatic-esmo-open-2026"],"journals":[],"dependsOn":[],"notes":["Triple-negative breast cancer: the HER2-low label (IHC 1+ or 2+/ISH-negative) rests on DESTINY-Breast04, where 63 of 557 patients were hormone receptor-negative (Modi 2022); about a third of TNBC is HER2-low (Schettini 2021, Denkert 2021). Ultralow TNBC (37.6% of untreated tumours; Boissiere-Michot 2026) is outside the label because DESTINY-Breast06 was hormone receptor-positive only.","Triple-negative breast cancer: DESTINY-Breast04 enrolled 63 hormone-receptor-negative patients (11.3 percent) alongside 494 hormone-receptor-positive; in that exploratory cohort median progression-free survival was 8.5 versus 2.9 months (hazard ratio 0.46) and the long-term analysis reported that overall survival also favoured trastuzumab deruxtecan in hormone-receptor-negative disease. The FDA HER2-low indication after chemotherapy is not restricted by hormone receptor status; the HER2-ultralow indication (DESTINY-Breast06) is hormone-receptor-positive only, so an ultralow triple-negative tumour has no licensed route to the drug. In England the HER2-low indication is not commissioned (NICE TA992, not recommended), which is the largest access gap between the UK and the United States in this disease.","Colorectal cancer biomarkers: the conjugate's colorectal trials selected on HER2 immunohistochemistry 3+ or 2+ with in situ hybridisation amplification under the colorectal-specific criteria; unlike in breast cancer there is no HER2-low colorectal indication, and the DESTINY-CRC02 dose comparison was run in the amplified population (Valtorta 2015).","Biliary tract cancer: confirmed response rate 36.4% in 22 HER2-positive and 12.5% in 8 HER2-low patients in HERB, with interstitial lung disease in 25% including two deaths (Ohba 2024); 22.0% by investigator in the 41-patient DESTINY-PanTumor02 biliary cohort and 56.3% in centrally confirmed IHC 3+ tumours, with lung disease in 17.1% (Oh 2026). The tumour-agnostic IHC 3+ indication is the on-label route in gallbladder cancer."],"brand":"Enhertu","code":"DS-8201, T-DXd","modality":"ADC","payload":"DXd (exatecan derivative, TOP1 inhibitor), DAR ~8","linker":"Tetrapeptide GGFG, protease-cleavable","mechanism":"Trastuzumab backbone; DXd released by lysosomal cathepsins; TOP1 inhibition and bystander diffusion.","approvals":[{"region":"US","year":2019,"indication":"HER2+ metastatic breast cancer, ≥2 prior anti-HER2 regimens"},{"region":"US","year":2022,"indication":"HER2-low metastatic breast cancer; HER2-mutant NSCLC"},{"region":"US","year":2024,"indication":"HER2 IHC3+ solid tumours (tumour-agnostic)"},{"region":"US","year":2025,"indication":"HER2-low/ultralow HR+ breast cancer after endocrine therapy (DESTINY-Breast06)"},{"region":"US","year":2026,"indication":"Early-stage HER2+ breast cancer (neoadjuvant and post-neoadjuvant)"},{"region":"EU","year":2023,"indication":"Unresectable or metastatic HER2-low breast cancer after chemotherapy in the metastatic setting or recurrence within 6 months of adjuvant chemotherapy (DESTINY-Breast04), hormone-receptor-negative disease included","note":"https://www.ema.europa.eu/en/medicines/human/EPAR/enhertu"},{"region":"UK","year":2024,"indication":"HER2-low metastatic or unresectable breast cancer after chemotherapy: NICE TA992 (29 July 2024) does not recommend trastuzumab deruxtecan, the cost per quality-adjusted life year being above the range NICE accepts; a rapid review (GID-TA12551) is in development","note":"https://www.nice.org.uk/guidance/ta992"},{"region":"US","year":2024,"indication":"Unresectable or metastatic HER2-positive (IHC 3+) solid tumours after previous systemic treatment, including colorectal cancer","note":"Tumour-agnostic accelerated approval on 5 April 2024; the colorectal evidence is DESTINY-CRC01 and DESTINY-CRC02 at 5.4 mg/kg. No NICE recommendation for colorectal cancer at September 2026."}],"mechanismSteps":["Antibody binds HER2 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","DXd is released inside the cell","Topoisomerase-I is trapped on DNA; replication forks collapse into double-strand breaks","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"5.4 mg/kg every 3 weeks (breast, NSCLC, HER2 IHC3+ tumours); 6.4 mg/kg every 3 weeks (gastric)","modifications":"Permanently discontinue for grade ≥2 ILD/pneumonitis; hold for grade 1 until resolved; reduce for neutropenia and LVEF decrease","monitoring":"Baseline and periodic LVEF; prompt CT and steroids for any respiratory symptoms; blood counts","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6"},"toxicity":[{"event":"Nausea","grade3PlusPct":7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"DESTINY-Breast03/04; all-grade rates ≥20% per label"},{"event":"Fatigue","grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"DESTINY-Breast03/04; all-grade rates ≥20% per label"},{"event":"Neutropenia","grade3PlusPct":18,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"DESTINY-Breast03/04; all-grade rates ≥20% per label"},{"event":"Anaemia","grade3PlusPct":7,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"DESTINY-Breast03/04; all-grade rates ≥20% per label"},{"event":"Interstitial lung disease / pneumonitis","anyGradePct":12,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"0.9% fatal; pooled breast studies at 5.4 mg/kg"},{"event":"Vomiting","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"DESTINY-Breast03/04; all-grade rates ≥20% per label"},{"event":"Alopecia","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"DESTINY-Breast03/04; all-grade rates ≥20% per label"},{"event":"Constipation","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"DESTINY-Breast03/04; all-grade rates ≥20% per label"},{"event":"Decreased appetite","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=7e67e73e-ddf4-4e4d-8b50-09d7514910b6","note":"DESTINY-Breast03/04; all-grade rates ≥20% per label"},{"event":"Drug-related interstitial lung disease or pneumonitis (adjudicated)","anyGradePct":12.1,"source":"https://doi.org/10.1056/NEJMoa2203690","note":"DESTINY-Breast04; 0.8 percent grade 5"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.enhertu4u.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended in HER2+ breast cancer after ≥1 anti-HER2 regimen (TA862) and HER2-low breast cancer (TA1090); HER2-mutant NSCLC via CDF","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"},{"country":"JP","reimbursement":"NHI listed; approved in HER2+ breast and gastric cancer since 2020","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2017-08","type":"designation","region":"US","note":"Breakthrough Therapy designation, HER2+ breast cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2019-12-20","type":"accelerated-approval","region":"US","note":"Accelerated approval, HER2+ metastatic breast cancer after ≥2 anti-HER2 regimens (DESTINY-Breast01)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting"},{"date":"2021-01-15","type":"approval","region":"US","note":"HER2+ gastric/GEJ adenocarcinoma (DESTINY-Gastric01)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-05-04","type":"conversion","region":"US","note":"Second-line HER2+ metastatic breast cancer (DESTINY-Breast03)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting"},{"date":"2022-08-05","type":"approval","region":"US","note":"HER2-low metastatic breast cancer (DESTINY-Breast04); first HER2-low indication","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2022-08-11","type":"accelerated-approval","region":"US","note":"HER2-mutant NSCLC (accelerated) The confirmatory requirement was still open 4.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with unresectable or metastatic NSCLC whose tumors have an activating HER2 (ERBB2) mutation, as detected by an FDA-approved test, and who have received a prior systemic therapy"},{"date":"2024-04-05","type":"accelerated-approval","region":"US","note":"HER2 IHC3+ solid tumours, tumour-agnostic (accelerated) The confirmatory requirement was still open 2.5 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Adult patients with unresectable or metastatic HER2-positive (IHC 3+) solid tumors who have received prior systemic treatment and have no satisfactory alternative treatment options."},{"date":"2025-01-27","type":"approval","region":"US","note":"HER2-low and HER2-ultralow HR+ breast cancer after endocrine therapy (DESTINY-Breast06)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q2","type":"approval","region":"US","note":"Early-stage HER2+ breast cancer: neoadjuvant (DESTINY-Breast11) and post-neoadjuvant residual disease (DESTINY-Breast05)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"trastuzumab-duocarmazine","kind":"drug","name":"Trastuzumab duocarmazine","aka":[],"tldr":"A HER2 ADC with a DNA-alkylating payload that beat chemotherapy in a phase 3 trial yet never reached the market, because eye and lung toxicity and a stronger rival arrived first.","summary":"Byondis' duocarmycin-payload HER2 ADC improved PFS over physician's choice in pretreated HER2+ metastatic breast cancer (TULIP, 2021; PFS 7.0 vs 4.9 months) but with frequent ocular toxicity (~78%) and ILD. The FDA issued a complete response letter in 2023; the EMA application was withdrawn in 2024. Trastuzumab deruxtecan's DESTINY-Breast03 result made its niche disappear.\n\nLesson: statistical significance is not enough when a competitor redefines the standard; payload toxicity profile decides whether an ADC survives.","status":"withdrawn","asOf":"2026-09-06","links":[{"label":"TULIP (Ann Oncol 2021 abstract)","url":"https://www.annalsofoncology.org/article/S0923-7534(21)04434-8/fulltext"}],"tags":["failure","lesson:toxicity"],"related":[],"cancers":["breast-her2-positive"],"sections":[],"technologies":["adc"],"targets":["her2"],"drugs":["trastuzumab-deruxtecan"],"companies":["byondis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03262935","nct04205630","i-spy-2-2"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"SYD985","modality":"ADC","payload":"seco-DUBA (duocarmycin)","linker":"Val-Cit cleavable","mechanism":"Trastuzumab with seco-DUBA duocarmycin via cleavable Val-Cit linker; DNA alkylation.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trastuzumab-emtansine","kind":"drug","name":"Trastuzumab emtansine","aka":[],"tldr":"Trastuzumab emtansine (Kadcyla, T-DM1) was the first ADC for a solid tumour (2013). It is still standard after surgery for HER2+ breast cancer patients whose tumour did not fully respond to pre-surgery treatment.","summary":"Approved 2013 for pretreated HER2+ metastatic breast cancer (EMILIA) and 2019 for residual disease after neoadjuvant therapy (KATHERINE: invasive DFS HR 0.50, with OS benefit). Non-cleavable linker means no bystander effect, which explains its inferiority to T-DXd in DESTINY-Breast03 and its failure in HER2-low disease. Being displaced post-neoadjuvantly by T-DXd (DESTINY-Breast05).","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Trastuzumab_emtansine","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Trastuzumab%20emtansine"}],"tags":[],"related":["her2-ihc-3-plus"],"cancers":["breast-her2-positive","her2-positive-early-breast-cancer"],"sections":[],"technologies":["adc"],"targets":["her2"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["smcc"],"trials":["nct06313086","nct06830889","nct06316531","nct05081609","nct06324357","nct04931342","nct04873362","nct00781612","nct06126640"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Kadcyla","code":"T-DM1","modality":"ADC","payload":"DM1 (maytansinoid, tubulin inhibitor), DAR ~3.5","linker":"SMCC, non-cleavable","mechanism":"Trastuzumab with lysine-conjugated DM1; released as Lys-MCC-DM1 after lysosomal degradation, cell-impermeable.","approvals":[{"region":"US","year":2013,"indication":"HER2+ metastatic breast cancer after trastuzumab and taxane"},{"region":"US","year":2019,"indication":"Adjuvant HER2+ breast cancer with residual disease after neoadjuvant therapy"}],"mechanismSteps":["Antibody binds HER2 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","DM1 (lysine-MCC-DM1, non-permeable) is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis"],"dosing":{"route":"IV infusion","schedule":"3.6 mg/kg every 3 weeks; 14 cycles in the adjuvant setting","modifications":"Reduce to 3 then 2.4 mg/kg for thrombocytopenia, hepatotoxicity, neuropathy; discontinue for LVEF <40%","monitoring":"LFTs and platelets before each dose; LVEF every 3 months","source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e"},"toxicity":[{"event":"Fatigue","anyGradePct":50,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"},{"event":"Nausea","anyGradePct":42,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"},{"event":"Transaminases increased","anyGradePct":32,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"},{"event":"Musculoskeletal pain","anyGradePct":30,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"},{"event":"Haemorrhage","anyGradePct":29,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"},{"event":"Thrombocytopenia","anyGradePct":29,"grade3PlusPct":6,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"},{"event":"Headache","anyGradePct":28,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"},{"event":"Peripheral neuropathy","anyGradePct":28,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"},{"event":"Left ventricular dysfunction","anyGradePct":3,"source":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=23f3c1f4-0fc8-4804-a9e3-04cf25dd302e","note":"KATHERINE (adjuvant)"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","assistance":"https://www.genentech-access.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for residual invasive disease after neoadjuvant therapy (TA632) and HER2+ metastatic disease (TA458)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2013-02-22","type":"approval","region":"US","note":"HER2+ metastatic breast cancer after trastuzumab and taxane (EMILIA); first ADC for a solid tumour","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2019-05-03","type":"approval","region":"US","note":"Adjuvant treatment of HER2+ early breast cancer with residual disease (KATHERINE)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q2","type":"label-change","region":"US","note":"Displaced post-neoadjuvantly by T-DXd approval (DESTINY-Breast05)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"trastuzumab-pamirtecan","kind":"drug","name":"Trastuzumab pamirtecan","aka":["BNT323"],"tldr":"Trastuzumab pamirtecan is DualityBio and BioNTech's HER2 antibody-drug conjugate, in phase 3 trials in breast cancers with high and with modest HER2 expression and in HER2-expressing endometrial cancer.","summary":"DualityBio's DB-1303, licensed to BioNTech as BNT323, is a HER2-directed antibody-drug conjugate with a topoisomerase I inhibitor payload. Phase 3 trials are testing it in metastatic breast cancer with high and with modest HER2 expression against standard chemotherapy or other HER2 conjugates and in advanced HER2-expressing endometrial cancer, where it received US breakthrough designation. It is one of several conjugates trying to improve on trastuzumab deruxtecan's payload chemistry and tolerability.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Trastuzumab pamirtecan","url":"https://clinicaltrials.gov/search?intr=DB-1303"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-her2-positive","endometrial"],"sections":[],"technologies":["adc"],"targets":["her2"],"drugs":[],"companies":["dualitybio","biontech"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06265428","nct06018337","nct06340568","nct06827236","nct05150691"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"DB-1303","modality":"Antibody-drug conjugate (anti-HER2 antibody, topoisomerase I inhibitor payload)","mechanism":"A HER2 antibody carrying a topoisomerase I inhibitor payload through a cleavable linker, the same design family as trastuzumab deruxtecan, with a bystander effect on neighbouring cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trastuzumab-rezetecan","kind":"drug","name":"Trastuzumab rezetecan","aka":[],"tldr":"Hengrui's HER2 ADC, approved in China for lung cancer and showing Enhertu-scale results in breast cancer, part of a wave of Chinese ADCs heading for global trials.","summary":"NMPA approval May 2025 for HER2-mutant NSCLC (HORIZON-Lung: ORR 74.5%, PFS 11.5 months). HORIZON-Breast01 (HER2+ breast after trastuzumab/taxane): PFS 30.6 vs 8.3 months vs pyrotinib + capecitabine (HR 0.22). Also active in HER2-low breast cancer and as neoadjuvant therapy; global phase 1 (HORIZON-X) reported 2026. ILD ~3-5%.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Trastuzumab%20rezetecan"}],"tags":[],"related":[],"cancers":["breast-her2-positive","nsclc"],"sections":[],"technologies":["adc","topoisomerase-inhibitors"],"targets":["her2"],"drugs":[],"companies":["hengrui"],"institutions":[],"pathways":[],"terms":[],"trials":["horizon-breast01","nct06199973","nct06828354","nct06430437","nct07196774","nct07676162","nct07051486","nct06840002","nct06859775","nct06222879","nct06015048","nct07497386","nct05814354","nct07102901","nct06057610","nct07111832","nct06778031","nct05482568","nct07110571","nct06126640","nct07679360","nct06413745","nct07140393","nct06123494","nct07231211","nct05671822"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"SHR-A1811","modality":"ADC","payload":"Rezetecan (SHR9265, camptothecin-derived TOP1 inhibitor), DAR ~6","linker":"Cleavable tetrapeptide","mechanism":"Trastuzumab-based antibody, cleavable linker, membrane-permeable TOP1 payload with bystander effect.","approvals":[{"region":"China","year":2025,"indication":"HER2-mutant NSCLC after ≥1 systemic therapy (May 2025)"}],"mechanismSteps":[],"dosing":{"route":"Intravenous","schedule":"4.8 mg/kg every 3 weeks (breast); 4.8-6.4 mg/kg studied"},"toxicity":[{"event":"Neutropenia","grade3PlusPct":40},{"event":"Nausea","anyGradePct":60},{"event":"Interstitial lung disease","anyGradePct":4}],"access":[],"regulatoryEvents":[{"date":"2025-05","type":"approval","region":"China","note":"HER2-mutant NSCLC","source":"https://www.precisionmedicineonline.com/precision-oncology/nmpa-approves-hengrui-pharmas-trastuzumab-rezetecan-her2-mutant-nsclc"}]},{"id":"tremelimumab","kind":"drug","name":"Tremelimumab","aka":[],"tldr":"Tremelimumab is AstraZeneca's CTLA-4 antibody, given as a single priming dose with durvalumab and chemotherapy in lung and liver cancer.","summary":"Tremelimumab is a human IgG2 antibody against CTLA-4, the checkpoint that restrains T-cell priming; a limited number of priming doses alongside a PD-L1 antibody keeps toxicity lower than continuous CTLA-4 blockade. It is used with durvalumab and platinum chemotherapy in first-line metastatic NSCLC without EGFR or ALK alterations, and with durvalumab in unresectable hepatocellular carcinoma. POSEIDON showed tremelimumab plus durvalumab plus chemotherapy improved overall survival versus chemotherapy (HR 0.77), with the largest benefit in STK11/KEAP1/KRAS-mutant and PD-L1-negative disease. Approved November 2022 (NSCLC) and October 2022 (HCC with durvalumab, HIMALAYA). Whether the CTLA-4 component justifies its extra immune toxicity remains debated. For a newcomer, it is the second checkpoint antibody in AstraZeneca's combinations, given briefly to kick-start the immune response.","status":"approved","asOf":"2026-09-06","wikipedia":"https://en.wikipedia.org/wiki/Tremelimumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tremelimumab"}],"tags":[],"related":[],"cancers":["nsclc","hcc","hcc-advanced","limited-stage-sclc"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["ctla4"],"drugs":["durvalumab"],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06921785","nct03164616","nct05883644","nct05557838","nct07081633","nct01843374","nct02519348","nct03682068","nct06008093","nct02516241","nct02453282","nct03837899","nct02542293","nct04960709"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Imjudo","modality":"Monoclonal antibody (anti-CTLA-4)","mechanism":"Human IgG2 anti-CTLA-4; limited priming doses reduce toxicity.","approvals":[{"region":"US","year":2022,"indication":"Metastatic NSCLC without EGFR/ALK, with durvalumab and platinum chemotherapy; unresectable HCC with durvalumab"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"treosulfan","kind":"drug","name":"Treosulfan","aka":[],"tldr":"A conditioning chemotherapy given before donor stem-cell transplant in AML and MDS; it was long used in Europe and reached the US in 2025.","summary":"Treosulfan is a busulfan analogue that converts non-enzymatically to epoxides that alkylate DNA. It is myeloablative but carries lower hepatic and neurological toxicity than busulfan, which makes it suitable for conditioning older or comorbid patients before allogeneic stem-cell transplant. In the MC-FludT.14/L trial (Lancet Haematology 2020), treosulfan-fludarabine improved event-free and overall survival versus reduced-intensity busulfan-fludarabine in older or comorbid patients with AML or MDS. It was approved in the EU in 2019 for conditioning in adults and children and reached the US in 2025 as a preparative regimen for allogeneic transplant in AML and MDS with fludarabine. It has also been used in paediatric transplant and historically in ovarian cancer in Europe. The core idea is a gentler way to clear the marrow so a donor graft can take hold.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Treosulfan","links":[{"label":"FDA novel approvals 2025","url":"https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025"}],"tags":["gap-fill","supportive"],"related":[],"cancers":["aml","mds"],"sections":[],"technologies":["allogeneic-hsct","cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["medac"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00822393","euro-ewing-2012","nct01423500"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Grafapex","modality":"Alkylating conditioning agent (busulfan analogue)","mechanism":"Prodrug converting non-enzymatically to epoxides that alkylate DNA; myeloablative with lower hepatic and neurotoxicity than busulfan.","approvals":[{"region":"EU","year":2019,"indication":"Conditioning before allogeneic HSCT (with fludarabine) in adults and children"},{"region":"US","year":2025,"indication":"Preparative regimen for allogeneic HSCT in AML/MDS with fludarabine"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tretinoin-atra","kind":"drug","name":"Tretinoin (all-trans retinoic acid, ATRA)","aka":["All-trans retinoic acid","ATRA","Retinoic acid"],"tldr":"The vitamin-A derivative that made acute promyelocytic leukaemia the first cancer cured by differentiation therapy rather than by killing cells.","summary":"Tretinoin (all-trans retinoic acid, ATRA) binds the RARA portion of the PML-RARA fusion protein at pharmacological doses, releasing co-repressors and forcing malignant promyelocytes to mature into neutrophils rather than killing them. The Shanghai group led by Huang reported complete remissions with ATRA alone in 1988, and the drug was approved in the US in 1995 for induction of remission in acute promyelocytic leukaemia with t(15;17). Combined first with chemotherapy and later with arsenic trioxide, APL became the most curable adult leukaemia. Differentiation syndrome, with capillary leak, fever and pulmonary infiltrates, is the characteristic danger and is treated with dexamethasone. ATRA is the founding example of differentiation therapy: a vitamin A derivative that cures a leukaemia by teaching its cells to grow up.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Tretinoin","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=tretinoin%20capsules"}],"tags":["gap-fill"],"related":[],"cancers":["aml","apl"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vesanoid","modality":"Oral retinoid (differentiation agent)","mechanism":"Binds the RARA moiety of PML-RARA at pharmacologic doses, releasing co-repressors and forcing terminal differentiation of promyelocytes.","approvals":[{"region":"US","year":1995,"indication":"Induction of remission in APL (t(15;17)/PML-RARA)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tri-611","kind":"drug","name":"TRI-611","aka":[],"tldr":"TRI-611 is an experimental protein degrader from TRIANA Biomedicines in phase 2 trials for non-small-cell lung cancer, aimed at ALK.","summary":"TRI-611 is a protein degrader developed by TRIANA Biomedicines. Its target is ALK (the sponsor names ALK). The sponsor states: TRI-611 is an oral, brain-penetrant molecular glue degrader that brings ALK fusion proteins and cereblon together through a mechanism independent of the kinase active site, driving degradation of ALK fusion proteins in ALK-positive NSCLC. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT07491497, plans to enrol 160 participants with primary completion expected 2029-05-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TRI-611","url":"https://clinicaltrials.gov/search?intr=TRI-611"},{"label":"Sponsor pipeline page","url":"https://www.trianabio.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["alk"],"drugs":[],"companies":["triana-biomedicines"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07491497"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"protein degrader","mechanism":"TRI-611 is an oral, brain-penetrant molecular glue degrader that brings ALK fusion proteins and cereblon together through a mechanism independent of the kinase active site, driving degradation of ALK fusion proteins in ALK-positive NSCLC.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trifluridine-tipiracil","kind":"drug","name":"Trifluridine/tipiracil","aka":[],"tldr":"An oral chemotherapy pill for bowel cancer that has stopped responding to everything else; with bevacizumab it extends life by about three months.","summary":"Trifluridine is incorporated into DNA; tipiracil blocks its breakdown. RECOURSE (2015) showed OS 7.1 vs 5.3 months alone; SUNLIGHT (2023) showed 10.8 vs 7.5 months with bevacizumab. Also approved in gastric cancer (TAGS). Neutropenia is the main toxicity; works even after 5-FU resistance because of a different mechanism.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Trifluridine/tipiracil","links":[{"label":"FDA label","url":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/207981s012lbl.pdf"},{"label":"NICE TA405: trifluridine-tipiracil for previously treated metastatic colorectal cancer","url":"https://www.nice.org.uk/guidance/ta405"},{"label":"NICE TA1008: trifluridine-tipiracil with bevacizumab after 2 systemic treatments","url":"https://www.nice.org.uk/guidance/ta1008"},{"label":"EMA Lonsurf","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/lonsurf"}],"tags":[],"related":[],"cancers":["colorectal","gastric"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["servier","takeda"],"institutions":[],"pathways":[],"terms":[],"trials":["sunlight","circulate-japan","nct06199973","nct07361003","nct06873763","nct04701476","nct05919264","nct06992258","nct05405595","nct07280377","nct04626635","recourse","altair"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Lonsurf","code":"TAS-102","modality":"Cytotoxic chemotherapy (oral nucleoside)","mechanism":"Trifluridine misincorporation into DNA causes strand dysfunction; tipiracil inhibits thymidine phosphorylase to maintain exposure.","approvals":[{"region":"US","year":2015,"indication":"Refractory metastatic colorectal cancer"},{"region":"US","year":2019,"indication":"Refractory metastatic gastric/GEJ cancer (TAGS)"},{"region":"US","year":2023,"indication":"Refractory mCRC with bevacizumab"},{"region":"EU","year":2016,"indication":"Metastatic colorectal cancer after available therapies","note":"Lonsurf marketing authorisation issued 25 April 2016."},{"region":"England (NICE)","year":2016,"indication":"Previously treated metastatic colorectal cancer","note":"TA405, published 24 August 2016, recommends it within its marketing authorisation with the patient access scheme discount."},{"region":"England (NICE)","year":2024,"indication":"Metastatic colorectal cancer after two lines of treatment, with bevacizumab","note":"TA1008, published 25 September 2024, recommends the combination within its marketing authorisation subject to the commercial arrangement."}],"mechanismSteps":["Tipiracil blocks the enzyme that would destroy trifluridine, keeping blood levels up","Trifluridine is taken up by dividing cancer cells and converted to its active form","It is built into DNA in place of thymidine","The corrupted DNA cannot function and the cell dies"],"dosing":{"route":"Oral","schedule":"35 mg/m² twice daily on days 1-5 and 8-12 of a 28-day cycle","modifications":"Delay for neutrophils <1,500 or platelets <75,000","monitoring":"Complete blood count before each cycle and on day 15","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/207981s012lbl.pdf"},"toxicity":[{"event":"Neutropenia","note":"Most common grade 3-4 event; febrile neutropenia uncommon"},{"event":"Anaemia"},{"event":"Nausea, fatigue"}],"access":[],"regulatoryEvents":[{"date":"2015-09","type":"approval","region":"US","note":"RECOURSE"},{"date":"2023-08","type":"approval","region":"US","note":"SUNLIGHT combination"}]},{"id":"trilaciclib","kind":"drug","name":"Trilaciclib","aka":["G1T28"],"tldr":"Trilaciclib is given as an infusion just before chemotherapy for small cell lung cancer to put the bone marrow's stem cells briefly to sleep, so fewer are killed and patients need fewer transfusions and growth factor injections.","summary":"G1 Therapeutics developed trilaciclib as a myeloprotectant rather than an anticancer drug. In three randomised trials in extensive-stage small cell lung cancer it cut the duration and incidence of severe neutropenia, red cell transfusions and growth factor use without reducing tumour responses, because small cell lung cancer lacks functional retinoblastoma protein and is not paused by CDK4/6 inhibition. The FDA approved it in February 2021 to decrease chemotherapy-induced myelosuppression in adults receiving platinum with etoposide or topotecan for extensive-stage small cell lung cancer. A trial in triple-negative breast cancer did not confirm a survival advantage, and Pharmacosmos acquired the drug in 2024.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Trilaciclib","links":[{"label":"Drugs@FDA NDA214200","url":"https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=214200"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=trilaciclib"},{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Trilaciclib"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["sclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["cdk4-6"],"drugs":["etoposide","topotecan"],"companies":["g1-therapeutics","pharmacosmos"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07473128"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Cosela","modality":"Intravenous CDK4/6 inhibitor given before chemotherapy to protect bone marrow","supportive":true,"mechanism":"A short-acting CDK4/6 inhibitor that pauses haematopoietic stem and progenitor cells in G1 for the hours chemotherapy is present, so the cytotoxic drugs kill fewer of them.","approvals":[{"region":"US","year":2021,"indication":"To decrease chemotherapy-induced myelosuppression in adults receiving platinum-etoposide or topotecan for extensive-stage small cell lung cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"triptorelin","kind":"drug","name":"Triptorelin","aka":["Decapeptyl","Triptodur (paediatric)"],"tldr":"Triptorelin (Trelstar) is a one-, three- or six-monthly injection that switches off testosterone production for men with advanced prostate cancer.","summary":"Triptorelin pamoate was approved by the FDA in June 2000 (3.75 mg monthly), with 11.25 mg three-monthly (2001) and 22.5 mg six-monthly (2010) depots following, for palliative treatment of advanced prostate cancer; the pivotal randomised trial of 277 men showed castrate testosterone suppression comparable to leuprolide. As Decapeptyl it has been used in Europe since the 1980s for prostate cancer, endometriosis and, in premenopausal breast cancer, for ovarian function suppression in the SOFT and TEXT trials that established the combination with exemestane. Hot flushes, loss of bone density, metabolic effects and initial flare (covered with an antiandrogen) are shared with the class.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Triptorelin","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=triptorelin"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/triptorelin-pamoate"}],"tags":["nci-list"],"related":["leuprolide","goserelin"],"cancers":["prostate","breast-hr-positive"],"sections":[],"technologies":["androgen-deprivation","endocrine-therapy"],"targets":["androgen-receptor"],"drugs":[],"companies":["verity-pharma","ipsen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct00066703","nct01020448","nct00412022"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Trelstar","modality":"GnRH agonist depot","mechanism":"Synthetic decapeptide GnRH agonist; continuous stimulation desensitises pituitary GnRH receptors, suppressing LH and testosterone to castrate levels after an initial surge.","approvals":[{"region":"US","year":2000,"indication":"Palliative treatment of advanced prostate cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trk-950","kind":"drug","name":"TRK-950","aka":[],"tldr":"TRK-950 is an experimental investigational agent whose form is not stated in the registry from Toray Industries in phase 2 trials for gastric & gastro-oesophageal junction cancer and melanoma, aimed at NTRK.","summary":"TRK-950 is an investigational agent whose form is not stated in the registry developed by Toray Industries. Its target is NTRK. ClinicalTrials.gov describes the intervention as: 5 mg/kg or 10 mg/kg IV infusion over 60 minutes on Day 1, 8, 15 and 21 of each 28 day cycle. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in gastric & gastro-oesophageal junction cancer and melanoma. The largest, NCT06038578, plans to enrol 146 participants with primary completion was scheduled for 2026-06-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TRK-950","url":"https://clinicaltrials.gov/search?intr=TRK-950"},{"label":"Sponsor page","url":"https://www.toray.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gastric","melanoma"],"sections":[],"technologies":[],"targets":["ntrk"],"drugs":[],"companies":["toray-industries"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05423262","nct06038578"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"monoclonal antibody (per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Investigational agent whose form is not stated in the registry directed at NTRK, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"trusight-oncology-comprehensive","kind":"drug","name":"TruSight Oncology Comprehensive","aka":[],"tldr":"A large gene panel hospitals can run themselves, approved by the FDA in 2024 as a companion diagnostic for the tumour-agnostic drug larotrectinib.","summary":"Illumina's TruSight Oncology Comprehensive is a kit-based, distributable comprehensive genomic profiling test, CE-IVD marked in Europe in 2022 and approved by the FDA in August 2024 with a companion claim for NTRK fusions (larotrectinib) alongside tumour-profiling claims. Unlike send-out services such as FoundationOne CDx, the test is run locally, which shortens turnaround and keeps data in the hospital. It reports tumour mutational burden and microsatellite status, so it also serves the tissue-agnostic pembrolizumab indications, and Illumina has filed additional companion claims.","status":"approved","asOf":"2026-09-10","links":[{"label":"Illumina: TruSight Oncology Comprehensive (page moved; nearest live section)","url":"https://www.illumina.com/products/by-type/clinical-research-products/"}],"tags":["test"],"related":[],"cancers":["pancreatic","colorectal"],"sections":[],"technologies":["cgp","companion-diagnostic"],"targets":["ntrk"],"drugs":["larotrectinib"],"companies":["illumina"],"institutions":[],"pathways":[],"terms":["ngs","tmb","msi"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: the report lists every actionable alteration found; the companion claim is currently for NTRK fusions, while the rest of the findings guide off-label or trial options."],"brand":"TSO Comprehensive","modality":"Distributable tissue NGS companion diagnostic test (500+ genes, DNA and RNA)","mechanism":"Hybrid-capture DNA and RNA sequencing of more than 500 genes on Illumina NextSeq instruments run in the hospital laboratory, reporting variants, fusions, tumour mutational burden and microsatellite instability.","approvals":[{"region":"EU","year":2022,"indication":"CE-IVD comprehensive genomic profiling"},{"region":"US","year":2024,"indication":"Companion diagnostic for larotrectinib (NTRK fusions) with tumour-profiling claims"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tst001","kind":"drug","name":"TST001","aka":[],"tldr":"TST001 is a monoclonal antibody from Transcenta Therapeutics (Hangzhou) Co., Ltd., in registered phase 2 trials for metastatic cancer.","summary":"TST001 is listed on ClinicalTrials.gov as an intervention in 2 registered phase 2 trials sponsored by Transcenta Therapeutics (Hangzhou) Co., Ltd., in metastatic cancer. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of TST001","url":"https://clinicaltrials.gov/search?intr=TST001"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["transcenta"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04396821","nct04495296"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"TST001","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tst003","kind":"drug","name":"TST003","aka":[],"tldr":"TST003 is a monoclonal antibody from Transcenta Therapeutics (Hangzhou) Co., Ltd., in registered phase 2 trials for colorectal cancer.","summary":"TST003 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Transcenta Therapeutics (Hangzhou) Co., Ltd., in colorectal cancer. A monoclonal antibody, as described in the registry record. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of TST003","url":"https://clinicaltrials.gov/search?intr=TST003"},{"label":"ClinicalTrials.gov NCT05731271","url":"https://clinicaltrials.gov/study/NCT05731271"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["colorectal"],"sections":[],"technologies":[],"targets":["grem1"],"drugs":[],"companies":["transcenta"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05731271"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["TST003 is described in its first-in-human registry record as an intravenous humanised anti-GREM1 monoclonal antibody; gremlin-1 is a BMP antagonist that keeps intestinal and tumour cells in a stem-like state."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"TST003","modality":"Monoclonal antibody","mechanism":"A monoclonal antibody, as described in the registry record.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tucatinib","kind":"drug","name":"Tucatinib","aka":[],"tldr":"A HER2-selective pill that works in the brain, for HER2-positive breast cancer with brain metastases.","summary":"Tucatinib is a HER2-selective reversible tyrosine kinase inhibitor that spares EGFR, so it causes less rash and diarrhoea than lapatinib or neratinib, and it penetrates the central nervous system. Taken as 300 mg twice daily, it was approved in 2020 with trastuzumab and capecitabine for HER2-positive metastatic breast cancer after HER2CLIMB, which showed an overall survival benefit that held in patients with active brain metastases, a group usually excluded from trials. In 2023 it was approved with trastuzumab for HER2-positive, RAS-wild-type metastatic colorectal cancer after MOUNTAINEER. Pfizer (via Seagen) markets it. Its place after trastuzumab deruxtecan, which also has brain activity, is the current sequencing question. For a newcomer: the HER2 pill chosen when the cancer has reached the brain.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Tucatinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Tucatinib"},{"label":"FDA oncology approval notifications","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancerhematologic-malignancies-approval-notifications"}],"tags":[],"related":["tucatinib-triplet-brain-mets"],"cancers":["breast-her2-positive","colorectal","her2-amplified-colorectal","secondary-brain-tumours","her2-positive-breast-brain-metastases"],"sections":[],"technologies":["kinase-inhibitors","her2-tyrosine-kinase-inhibitors"],"targets":["her2"],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04430738","nct05458674","nct04538742","nct06157892","nct06686394","nct05230810","nct05190445","nct04721977","nct04579380","mountaineer","mountaineer-03"],"people":[],"bottlenecks":[],"keyPapers":["paper-valtorta-her2-scoring-colorectal-heracles-mod-pathol-2015","paper-richman-her2-amplification-quasar-focus-piccolo-j-pathol-2016"],"journals":[],"dependsOn":[],"notes":["Colorectal cancer biomarkers: eligibility is HER2 amplification or immunohistochemistry 3+ by the colorectal-specific criteria in a RAS wild-type tumour, about 5% of the RAS wild-type population (Valtorta 2015, Richman 2016)."],"brand":"Tukysa","modality":"Small-molecule kinase inhibitor (HER2)","mechanism":"Highly HER2-selective reversible TKI sparing EGFR.","approvals":[{"region":"US","year":2020,"indication":"HER2+ metastatic breast cancer including brain metastases, with trastuzumab and capecitabine"},{"region":"US","year":2023,"indication":"HER2+ RAS-wild-type mCRC with trastuzumab"},{"region":"US","year":2023,"indication":"HER2-positive, RAS wild-type unresectable or metastatic colorectal cancer that has progressed after fluoropyrimidine, oxaliplatin and irinotecan, with trastuzumab","note":"Accelerated approval on 19 January 2023 on the MOUNTAINEER cohorts; MOUNTAINEER-03 is the confirmatory trial. No NICE recommendation for colorectal cancer at September 2026."}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of HER2 kinase domain","Phosphorylation of downstream substrates stops","EGFR-sparing selectivity; brain-penetrant, so intracranial metastases respond","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"300 mg twice daily with trastuzumab and capecitabine (breast) or trastuzumab (colorectal)","modifications":"Reduce to 250 then 200 mg for hepatotoxicity or diarrhoea","monitoring":"LFTs every 3 weeks; diarrhoea management","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Diarrhoea","note":"HER2CLIMB: 81% any grade, 13% grade 3"},{"event":"Palmar-plantar erythrodysaesthesia"},{"event":"Nausea"},{"event":"Fatigue"},{"event":"Hepatotoxicity"},{"event":"Stomatitis"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.pfizeroncologytogether.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended with trastuzumab and capecitabine for HER2+ breast cancer after ≥2 anti-HER2 regimens (TA786)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2020-04-17","type":"approval","region":"US","note":"HER2+ metastatic breast cancer including brain metastases, with trastuzumab and capecitabine (HER2CLIMB)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2023-01-19","type":"accelerated-approval","region":"US","note":"HER2+ RAS wild-type metastatic colorectal cancer with trastuzumab (MOUNTAINEER, accelerated) The confirmatory requirement was still open 3.7 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with trastuzumab for the treatment of adult patients with RAS wild-type, HER2-positive, unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy"}]},{"id":"tucidinostat","kind":"drug","name":"Tucidinostat (chidamide)","aka":["Chidamide"],"tldr":"Chidamide, approved in 2014, was the first epigenetic cancer drug invented in China and the first oral drug of its kind anywhere, used for T-cell lymphoma and, with hormone therapy, for breast cancer.","summary":"Chipscreen Biosciences' chidamide was approved by the CFDA in December 2014 for relapsed or refractory peripheral T-cell lymphoma, the first China-origin small molecule in a new chemical class and the first oral subtype-selective HDAC inhibitor approved anywhere. The ACE trial (Lancet Oncology 2019) added HR-positive advanced breast cancer with exemestane after endocrine therapy, approved in 2019. Licensed to HUYA Bioscience as HBI-8000, it was approved in Japan in 2021 for adult T-cell leukaemia-lymphoma and is being tested with PD-1 antibodies in melanoma.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Chidamide","links":[{"label":"Chipscreen Biosciences","url":"https://www.chipscreen.com/en/"},{"label":"ACE trial (Lancet Oncol 2019)","url":"https://doi.org/10.1016/S1470-2045(19)30164-0"},{"label":"NMPA (National Medical Products Administration)","url":"https://www.nmpa.gov.cn"}],"tags":["china"],"related":[],"cancers":["peripheral-t-cell-lymphoma","breast-hr-positive"],"sections":[],"technologies":["epigenetic-drugs"],"targets":[],"drugs":[],"companies":["chipscreen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06497985","nct06776952","nct05833724","nct04668690"],"people":[],"bottlenecks":[],"keyPapers":["paper-jiang-lancet-oncol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Epidaza","code":"chidamide, HBI-8000, CS055","modality":"Small-molecule oral subtype-selective HDAC inhibitor","mechanism":"Benzamide-class inhibitor of class I HDACs 1, 2 and 3 and class IIb HDAC10, relaxing chromatin, restoring tumour suppressor expression and enhancing NK and T-cell activity.","approvals":[{"region":"China","year":2014,"indication":"Relapsed or refractory peripheral T-cell lymphoma"},{"region":"China","year":2019,"indication":"HR-positive, HER2-negative advanced breast cancer with exemestane after endocrine therapy (ACE)"},{"region":"Japan","year":2021,"indication":"Relapsed or refractory adult T-cell leukaemia-lymphoma (HBI-8000)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tunlametinib","kind":"drug","name":"Tunlametinib","aka":[],"tldr":"Tunlametinib is a Chinese MEK inhibitor approved in 2024 for advanced melanoma with an NRAS mutation, a group with no targeted therapy elsewhere in the world, and in phase 3 trials with vemurafenib for BRAF-mutant disease and for colorectal cancer.","summary":"Shanghai Kechow Pharma's tunlametinib (HL-085) received NMPA approval in 2024 for advanced NRAS-mutant melanoma after prior immunotherapy, a first for that molecular group. NRAS mutations are common in the acral and mucosal melanomas that predominate in East Asia. Phase 3 trials test tunlametinib with vemurafenib in BRAF V600-mutant melanoma and in combinations for metastatic colorectal cancer.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Tunlametinib","url":"https://clinicaltrials.gov/search?intr=HL-085"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["melanoma","colorectal"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["mek"],"drugs":[],"companies":["shanghai-kechow-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06008106","nct06008119"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"HL-085","modality":"Oral MEK1/2 inhibitor","mechanism":"An allosteric inhibitor of MEK1 and MEK2 that shuts off the MAPK pathway downstream of RAS and RAF, the route NRAS-mutant melanoma depends on.","approvals":[{"region":"CN","year":2024,"indication":"Advanced NRAS-mutant melanoma after prior immunotherapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tusamitamab-ravtansine","kind":"drug","name":"Tusamitamab ravtansine","aka":[],"tldr":"Tusamitamab ravtansine was Sanofi's ADC against the classic CEA tumour marker, stopped for futility in lung cancer in 2023.","summary":"A CEACAM5-directed ADC with a DM4 maytansinoid payload. Phase 3 CARMEN-LC03 (vs docetaxel in CEACAM5-high non-squamous NSCLC) was stopped in December 2023 when an interim analysis showed the PFS endpoint would not be met; OS was not improved. Sanofi discontinued the whole programme.\n\nLesson: a tubulin-inhibitor payload at DAR ~4 with a non-permeable release mechanism could not match the TOP1-payload ADC bar set in lung cancer; CEACAM5 itself remains under study with T-cell engagers.","status":"withdrawn","asOf":"2026-09-06","links":[{"label":"Sanofi press release, CARMEN-LC03 (Dec 2023) (page moved; nearest live section)","url":"https://www.sanofi.com/en/media-room/press-releases/2023/"}],"tags":["failure","lesson:wrong-drug"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":["adc"],"targets":["ceacam5"],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04154956","nct04524689","nct04659603"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"SAR408701","modality":"ADC","payload":"DM4 (ravtansine)","linker":"SPDB disulfide","mechanism":"Humanised anti-CEACAM5 IgG1 with DM4 via cleavable SPDB linker.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tuspetinib","kind":"drug","name":"Tuspetinib","aka":["HM43239"],"tldr":"Tuspetinib is an oral kinase inhibitor from Aptose Biosciences Inc., in registered phase 2 trials for myelodysplastic syndromes / neoplasms, chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms.","summary":"Tuspetinib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Aptose Biosciences Inc., in myelodysplastic syndromes / neoplasms, chronic myelomonocytic leukaemia and MDS/MPN overlap neoplasms. A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Tuspetinib","url":"https://clinicaltrials.gov/search?intr=Tuspetinib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["mds","cmml"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["aptose"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03850574"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral kinase inhibitor","mechanism":"A kinase inhibitor: the name stem -tinib marks a small molecule that blocks a tyrosine kinase driving tumour growth. The registry record does not state which kinase unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ty-9591","kind":"drug","name":"TY-9591","aka":["TY-9591 Tablets"],"tldr":"TY-9591 is an experimental small-molecule drug from TYK Medicines in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"TY-9591 is a small-molecule drug developed by TYK Medicines. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: The dose of TY-9591 is 160 mg once daily. A cycle of treatment is defined as 21 days of once daily treatment. Number of Cycles: as long as patients are continuing to show clinical benefit, as judged by the Investigator, and in the absence o. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05382728 (Phase III Study of TY-9591 in Patients With Locally Advanced or Metastatic Non-small Cell Lung Cancer (FLETEO)), in non-small-cell lung cancer. The largest, NCT05382728, plans to enrol 680 participants with primary completion was scheduled for 2025-05 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TY-9591","url":"https://clinicaltrials.gov/search?intr=TY-9591"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["tyk-medicines"],"institutions":[],"pathways":[],"terms":[],"trials":["fleteo","nct05948813"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"tyra-300","kind":"drug","name":"TYRA-300","aka":[],"tldr":"TYRA-300 is an experimental small-molecule drug from Tyra Biosciences in phase 2 trials for bladder & urothelial cancer, aimed at FGFR2.","summary":"TYRA-300 is a small-molecule drug developed by Tyra Biosciences. Its target is FGFR2 (the sponsor names FGFR3). The sponsor states: TYRA-300 is an oral, FGFR3-selective tyrosine kinase inhibitor that targets tumours containing activating FGFR3 gene alterations. ClinicalTrials.gov describes the intervention as: TYRA-300 is an oral, novel potent FGFR 3-selective tyrosine kinase inhibitor that targets tumours that contain activating gene alterations of FGFR3. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in bladder & urothelial cancer. The largest, NCT05544552, plans to enrol 310 participants with primary completion expected 2026-11. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of TYRA-300","url":"https://clinicaltrials.gov/search?intr=TYRA-300"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":[],"targets":["fgfr2"],"drugs":[],"companies":["tyra-biosciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06995677","nct05544552"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"TYRA-300 is an oral, FGFR3-selective tyrosine kinase inhibitor that targets tumours containing activating FGFR3 gene alterations.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ubamatamab","kind":"drug","name":"Ubamatamab","aka":["REGN4018"],"tldr":"Ubamatamab (REGN4018) is Regeneron's investigational MUC16×CD3 bispecific antibody, in phase 1/2 and phase 2 trials for ovarian and endometrial cancer.","summary":"Ubamatamab (REGN4018) is a bispecific antibody that binds MUC16 on tumour cells and CD3 on T cells and is designed to redirect T-cell killing towards MUC16-expressing cells. The first-in-human study NCT03564340 tests ubamatamab alone or with cemiplimab in recurrent ovarian cancer and other MUC16-positive cancers (phase 1/2); NCT06787612 is a multi-arm phase 2 platform study of ubamatamab combinations in platinum-resistant ovarian cancer; NCT04590326 combines it with the MUC16×CD28 costimulatory bispecific REGN5668; and NCT07154290 tests it with marlotamig in non-small-cell lung cancer. No results are recorded here; the registry entries and the cited abstracts are the sources.","status":"phase-2","asOf":"2026-09-21","links":[{"label":"ClinicalTrials.gov: trials of Ubamatamab","url":"https://clinicaltrials.gov/search?intr=Ubamatamab"},{"label":"Ubamatamab phase 1/2 study design (JCO 2024, TPS5632)","url":"https://doi.org/10.1200/JCO.2024.42.16_suppl.TPS5632"},{"label":"Ubamatamab translational study (Zhu et al., Clin Transl Sci 2024)","url":"https://doi.org/10.1111/cts.70082"},{"label":"ClinicalTrials.gov NCT03564340","url":"https://clinicaltrials.gov/study/NCT03564340"},{"label":"ClinicalTrials.gov NCT06787612","url":"https://clinicaltrials.gov/study/NCT06787612"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["ovarian","endometrial"],"sections":[],"technologies":["t-cell-engager"],"targets":["muc16","cd3"],"drugs":[],"companies":["regeneron"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04590326","nct03564340","nct06787612","nct07154290"],"people":[],"bottlenecks":[],"keyPapers":["paper-zhu-clin-transl-sci"],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo maintainers (issue #46)","editedOn":"2026-09-21","note":"Ingested from ClinicalTrials.gov v2 (registry-only) on 2026-09-16; mechanism and targets checked on 2026-09-21 against the NCT03564340 official title, the JCO 2024 TPS5632 study-design abstract and Zhu et al., Clin Transl Sci 2024."},"modality":"Bispecific T-cell engager (MUC16×CD3)","mechanism":"Bispecific antibody that binds MUC16 on tumour cells and CD3 on T cells, bringing them together to promote T-cell-mediated killing of MUC16-expressing cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ucart22","kind":"drug","name":"UCART22","aka":[],"tldr":"UCART22 is an experimental allogeneic CAR T-cell therapy from Cellectis S.A. in phase 2 trials for acute lymphoblastic leukaemia, aimed at CD22.","summary":"UCART22 is an allogeneic CAR T-cell therapy developed by Cellectis S.A.. Its target is CD22. The sponsor states: Gene-edited, donor-derived T cells engineered to recognise and attack CD22-positive leukaemia cells. ClinicalTrials.gov describes the intervention as: Allogeneic engineered T-cells expressing anti-CD22 Chimeric Antigen Receptor given following a lymphodepleting regimen. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in acute lymphoblastic leukaemia. The largest, NCT04150497, plans to enrol 52 participants with primary completion was scheduled for 2026-06-30 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of UCART22","url":"https://clinicaltrials.gov/search?intr=UCART22"},{"label":"Sponsor page","url":"https://www.cellectis.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["all-leukemia"],"sections":[],"technologies":[],"targets":["cd22"],"drugs":[],"companies":["cellectis"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04150497"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"allogeneic CAR T-cell therapy","mechanism":"Gene-edited, donor-derived T cells engineered to recognise and attack CD22-positive leukaemia cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ugn-103","kind":"drug","name":"UGN-103","aka":["UGN-103 (mitomycin) for intravesical solution"],"tldr":"UGN-103 is an experimental chemotherapy (mitomycin) delivered via hydrogel from UroGen Pharma in phase 3 trials for bladder & urothelial cancer, with its target not yet stated publicly.","summary":"UGN-103 is a chemotherapy (mitomycin) delivered via hydrogel developed by UroGen Pharma. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. The sponsor states: Combines UroGen's RTGel technology with medac's proprietary formulation of mitomycin (75 mg dosage strength) for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer. ClinicalTrials.gov describes the intervention as: UGN-103 consists of mitomycin and sterile hydrogel (a proprietary thermally responsive gel) that is used to reconstitute mitomycin before instillation. The reverse thermal properties of UGN-103 allow for local administration of mitomycin as. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06331299 (A Phase 3 Study of UGN-103 for Treatment of Patients With Low-grade Intermediate-risk Non-muscle Invasive Bladder Cancer), in bladder & urothelial cancer. The largest, NCT06331299, plans to enrol 99 participants (actual) with primary completion was scheduled for 2025-09-11 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of UGN-103","url":"https://clinicaltrials.gov/search?intr=UGN-103"},{"label":"Sponsor page","url":"https://www.urogen.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["urogen-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06331299"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"chemotherapy (mitomycin) delivered via hydrogel","mechanism":"Combines UroGen's RTGel technology with medac's proprietary formulation of mitomycin (75 mg dosage strength) for recurrent low-grade intermediate-risk non-muscle invasive bladder cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ugn-104","kind":"drug","name":"UGN-104","aka":["UGN-104 (mitomycin) for pyelocalyceal solution"],"tldr":"UGN-104 is an experimental chemotherapy (mitomycin) delivered via hydrogel from UroGen Pharma in phase 3 trials for bladder & urothelial cancer, with its target not yet stated publicly.","summary":"UGN-104 is a chemotherapy (mitomycin) delivered via hydrogel developed by UroGen Pharma. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. The sponsor states: Combines RTGel technology with medac's mitomycin formulation for kidney-sparing treatment of low-grade upper tract urothelial cancer; currently in a Phase 3 study. ClinicalTrials.gov describes the intervention as: UGN-104 consists of mitomycin and sterile hydrogel (a proprietary thermally responsive gel) that is used to reconstitute mitomycin before instillation. The reverse thermal properties of UGN-104 allow for local administration of mitomycin as. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06774131 (A Phase 3 Single-arm Study of UGN-104 for the Treatment of Low-grade Upper Tract Urothelial Cancer), in bladder & urothelial cancer. The largest, NCT06774131, plans to enrol 70 participants with primary completion expected 2027-03. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of UGN-104","url":"https://clinicaltrials.gov/search?intr=UGN-104"},{"label":"Sponsor page","url":"https://www.urogen.com/pipeline"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["urogen-pharma"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06774131"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"chemotherapy (mitomycin) delivered via hydrogel","mechanism":"Combines RTGel technology with medac's mitomycin formulation for kidney-sparing treatment of low-grade upper tract urothelial cancer; currently in a Phase 3 study.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"uliledlimab","kind":"drug","name":"Uliledlimab","aka":[],"tldr":"Uliledlimab is an experimental investigational agent whose form is not stated in the registry from TJ Biopharma in phase 3 trials for non-small-cell lung cancer, with its target not yet stated publicly.","summary":"Uliledlimab is an investigational agent whose form is not stated in the registry developed by TJ Biopharma. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: 30 mg/kg, administered on Days 1 and 8 of Cycle 1 (C1D1 and C1D8), then once every 3 weeks (Q3W) starting from C2D1,. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT06984588 (A Study to Compare Uliledlimab Combined With Toripalimab, Toripalimab Monotherapy, and Pembrolizumab Monotherapy in Patients With Previously Untreated Locally Advanced Unresectable or Metastatic PD-L1- and CD73- Selected Non-Small Cell Lung Cancer), in non-small-cell lung cancer. The largest, NCT06984588, plans to enrol 450 participants with primary completion expected 2028-08-05. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Uliledlimab","url":"https://clinicaltrials.gov/search?intr=Uliledlimab"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["i-mab"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06984588"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody (INN stem -mab; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"umbralisib","kind":"drug","name":"Umbralisib","aka":[],"tldr":"A PI3K inhibitor for marginal zone and follicular lymphoma approved in 2021 and withdrawn in 2022 after the UNITY-CLL trial suggested more deaths.","summary":"Umbralisib is an oral inhibitor of PI3K-delta and casein kinase 1 epsilon, designed to retain the B-cell activity of the class while reducing its immune-mediated toxicities such as colitis and hepatitis. In UNITY-NHL, response rates were around 45 to 50% in relapsed marginal zone and follicular lymphoma, and the FDA granted accelerated approval in February 2021. The UNITY-CLL trial, which combined umbralisib with ublituximab, suggested a possible overall-survival detriment in the experimental arm. TG Therapeutics withdrew the drug in 2022 and the FDA revoked the approval. The episode contributed to the FDA's broader 2022 review of PI3K inhibitors in blood cancers. Umbralisib illustrates why accelerated approvals depend on confirmatory data: a good response rate did not translate into patients living longer.","status":"withdrawn","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Umbralisib","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Umbralisib"}],"tags":["gap-fill"],"related":[],"cancers":["follicular-lymphoma","cll"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["pik3ca"],"drugs":[],"companies":["tg-therapeutics"],"institutions":[],"pathways":["pi3k-akt-mtor"],"terms":[],"trials":["nct03801525","nct02793583","nct04624633"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Ukoniq","modality":"Oral PI3Kδ / CK1ε inhibitor","mechanism":"PI3K delta inhibition with casein kinase 1 epsilon inhibition, intended to reduce immune-mediated toxicity of the class.","approvals":[{"region":"US","year":2021,"indication":"Relapsed marginal zone lymphoma and follicular lymphoma","note":"Withdrawn 2022"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2021-02-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one prior anti-CD20- based regimen"},{"date":"2021-02-05","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adult patients with relapsed or refractory follicular lymphoma (FL) who have received at least three prior lines of systemic therapy"},{"date":"2022-05-31","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 1.3 years after its accelerated approval.","indication":"Adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one prior anti-CD20- based regimen"},{"date":"2022-05-31","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 1.3 years after its accelerated approval.","indication":"Adult patients with relapsed or refractory follicular lymphoma (FL) who have received at least three prior lines of systemic therapy"}]},{"id":"upifitamab-rilsodotin","kind":"drug","name":"Upifitamab rilsodotin","aka":["XMT-1536","UpRi"],"tldr":"Upifitamab rilsodotin is an antibody-drug conjugate from Mersana Therapeutics, in registered phase 2 trials for ovarian cancer, non-small-cell lung cancer.","summary":"Upifitamab rilsodotin is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Mersana Therapeutics, in ovarian cancer, non-small-cell lung cancer. An antibody-drug conjugate: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. The registry record does not state the target or payload; the trial entries below carry the sponsor's description. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Upifitamab rilsodotin","url":"https://clinicaltrials.gov/search?intr=Upifitamab%20rilsodotin"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian","nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["mersana"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03319628"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Antibody-drug conjugate","mechanism":"An antibody-drug conjugate: an antibody against a tumour surface protein carries a cytotoxic payload into the cell. The registry record does not state the target or payload; the trial entries below carry the sponsor's description.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"uracil-mustard","kind":"drug","name":"Uracil mustard","aka":[],"tldr":"Uracil mustard was an oral alkylating agent approved in 1962 for chronic lymphocytic leukaemia and lymphomas, one of the early nitrogen mustard tablets, and long since discontinued.","summary":"Uracil mustard joined chlorambucil, melphalan and cyclophosphamide as the oral nitrogen mustards of the early 1960s and was approved in the United States in 1962 for chronic lymphocytic leukaemia, lymphomas including lymphosarcoma and reticulum cell sarcoma in the terminology of the day, and thrombocythaemia. Its results were similar to chlorambucil without clear advantages, and it was discontinued. It appears here for completeness of the alkylating agent story that began with the wartime nitrogen mustard studies.","status":"historic","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Uramustine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Uramustine"},{"label":"ChEMBL CHEMBL1731","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1731"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["cll"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":["chlorambucil"],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Uramustine","modality":"Oral nitrogen mustard alkylating agent","mechanism":"A nitrogen mustard attached to uracil that cross-links DNA strands; the uracil was intended to carry it into nucleic-acid-hungry cells.","approvals":[{"region":"US","year":1962,"indication":"Chronic lymphocytic leukaemia, lymphomas and essential thrombocythaemia (historic label)","note":"Discontinued"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"uridine-triacetate","kind":"drug","name":"Uridine triacetate","aka":["Xuriden (hereditary orotic aciduria formulation)"],"tldr":"Uridine triacetate (Vistogard) is an oral antidote for fluorouracil or capecitabine overdose, or for dangerously early severe toxicity from these drugs, and must be started within four days of the chemotherapy.","summary":"Uridine triacetate was approved by the FDA in December 2015 for emergency treatment of adults and children after a fluorouracil or capecitabine overdose regardless of symptoms, or with early-onset severe or life-threatening cardiac, neurological, gastrointestinal or haematological toxicity within 96 hours of the end of administration, on two open-label studies in which 96% of 135 treated patients survived compared with historical mortality of around 84% after overdose. The 96-hour window means it must be given before toxicity is fully manifest; it is not indicated for routine expected toxicity. Patients with DPD deficiency are a key group. Diarrhoea, nausea and vomiting are the main adverse effects. Developed by Wellstat and marketed by BTG, now SERB.","status":"approved","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Uridine_triacetate","links":[{"label":"Vistogard Studies 1 and 2 (Cancer 2017)","url":"https://doi.org/10.1002/cncr.30321"},{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=269f7363-63ed-444e-85f1-f0009e44818b"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/uridinetriacetate"}],"tags":["nci-list","supportive"],"related":["fluorouracil","capecitabine"],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["serb-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":["vistogard-studies-1-2"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vistogard","modality":"Pyrimidine analogue antidote (oral)","supportive":true,"mechanism":"Acetylated prodrug of uridine; delivered uridine is converted to uridine triphosphate, which competes with fluorouracil metabolites for incorporation into RNA and restores normal pyrimidine pools.","approvals":[{"region":"US","year":2015,"indication":"Emergency treatment after fluorouracil or capecitabine overdose or early-onset severe toxicity within 96 hours"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"urovysion","kind":"drug","name":"UroVysion Bladder Cancer Kit","aka":[],"tldr":"A urine test that looks for chromosome changes in shed bladder cells, approved for people with blood in the urine and for follow-up after bladder cancer.","summary":"UroVysion (Abbott Molecular) was approved by the FDA in 2001 for monitoring recurrence in patients with previously diagnosed bladder cancer and on 24 January 2005 (PMA P030052) for the initial work-up of haematuria. It is more sensitive than urine cytology, particularly for high-grade disease, but less specific, and an 'anticipatory positive' result can precede visible recurrence by months. Guidelines regard urine markers, including UroVysion, as adjuncts to cystoscopy rather than replacements; its main clinical use is resolving atypical cytology and assessing response to BCG.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/UroVysion","links":[{"label":"FDA PMA P030052","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P030052"}],"tags":["test"],"related":["cxbladder"],"cancers":["urothelial"],"sections":[],"technologies":["cytogenetics-fish","cystoscopy-turbt"],"targets":[],"drugs":[],"companies":["abbott"],"institutions":[],"pathways":[],"terms":["fish"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["What a result means: abnormal chromosomes in urine cells raise the suspicion of bladder cancer and usually lead to cystoscopy and biopsy; a normal result does not fully exclude low-grade tumours."],"brand":"UroVysion","modality":"Urine FISH cytogenetic test (bladder cancer)","mechanism":"Multicolour fluorescence in situ hybridisation on urine cells for aneuploidy of chromosomes 3, 7 and 17 and loss of the 9p21 locus (CDKN2A).","approvals":[{"region":"US","year":2001,"indication":"Monitoring for recurrence in patients with previously diagnosed bladder cancer"},{"region":"US","year":2005,"indication":"Aid to diagnosis of bladder cancer in patients with haematuria"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"utidelone","kind":"drug","name":"Utidelone","aka":["Youheng"],"tldr":"Utidelone is Biostar Pharmaceuticals' epothilone chemotherapy, approved in China in 2021 with capecitabine for advanced breast cancer after anthracyclines and taxanes.","summary":"Utidelone is a genetically engineered epothilone analogue produced by fermentation, developed by Biostar Pharmaceuticals in Chengdu. The NMPA approved it in March 2021 in combination with capecitabine for recurrent or metastatic breast cancer previously treated with an anthracycline and a taxane, after a phase 3 trial showed longer progression-free and overall survival than capecitabine alone. An oral form is in later trials.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of utidelone","url":"https://clinicaltrials.gov/search?intr=utidelone"}],"tags":["china","nmpa-approved"],"related":[],"cancers":[],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["tubulin"],"drugs":[],"companies":["biostar-pharmaceuticals"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"UTD1","modality":"Epothilone analogue, microtubule-stabilising cytotoxic chemotherapy","mechanism":"Binds tubulin and locks microtubules in place so that dividing cells cannot complete mitosis; it works in cells that have become resistant to taxanes.","approvals":[{"region":"CN","year":2021,"indication":"Recurrent or metastatic breast cancer after anthracycline and taxane, with capecitabine"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"utomilumab","kind":"drug","name":"Utomilumab","aka":[],"tldr":"Utomilumab is a monoclonal antibody from Pfizer, in registered phase 3 trials for ovarian cancer.","summary":"Utomilumab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 3 trial sponsored by Pfizer, in ovarian cancer. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Utomilumab","url":"https://clinicaltrials.gov/search?intr=Utomilumab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05059522"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vactosertib","kind":"drug","name":"Vactosertib","aka":["TEW-7197"],"tldr":"Vactosertib is an oral serine/threonine kinase inhibitor from MedPacto, Inc., in registered phase 2 trials for osteosarcoma.","summary":"Vactosertib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by MedPacto, Inc., in osteosarcoma. A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Vactosertib","url":"https://clinicaltrials.gov/search?intr=Vactosertib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["osteosarcoma"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["medpacto"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05588648"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral serine/threonine kinase inhibitor","mechanism":"A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"valrubicin","kind":"drug","name":"Valrubicin","aka":[],"tldr":"Valrubicin (Valstar) is a chemotherapy washed into the bladder for carcinoma in situ that has not responded to BCG, in patients who cannot yet have their bladder removed.","summary":"Valrubicin was approved in September 1998 for intravesical therapy of BCG-refractory carcinoma in situ of the urinary bladder in patients for whom immediate cystectomy would carry unacceptable morbidity or mortality. In the pivotal experience, 230 patients received the drug and about one in five with refractory CIS had a complete response, mostly not durable, so it is a bridge rather than a cure; the label stresses that delaying cystectomy risks progression. Newer options for BCG-unresponsive disease (pembrolizumab, nadofaragene firadenovec, nogapendekin alfa inbakicept, TAR-200) have reduced its use. Irritative bladder symptoms and red-tinged urine are expected.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Valrubicin","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=valrubicin"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/valrubicin"}],"tags":["nci-list"],"related":["nadofaragene-firadenovec","nogapendekin-alfa"],"cancers":["urothelial"],"sections":[],"technologies":["bcg-and-intravesical-therapy","topoisomerase-inhibitors"],"targets":[],"drugs":[],"companies":["endo"],"institutions":[],"pathways":[],"terms":[],"trials":["nct01310803"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Valstar","modality":"Anthracycline (intravesical)","mechanism":"Semi-synthetic anthracycline analogue of doxorubicin instilled into the bladder; inhibits nucleoside incorporation into nucleic acids and topoisomerase II, arresting cells in G2.","approvals":[{"region":"US","year":1998,"indication":"Intravesical therapy of BCG-refractory carcinoma in situ of the bladder when immediate cystectomy is not acceptable"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vandetanib","kind":"drug","name":"Vandetanib","aka":[],"tldr":"Vandetanib was the first drug approved for medullary thyroid cancer (2011), now largely replaced by RET-selective selpercatinib.","summary":"Vandetanib is a multi-kinase inhibitor of RET, VEGFR2 and EGFR, given at 300 mg once daily. It was the first drug approved for medullary thyroid cancer (April 2011), for symptomatic or progressive disease, after the ZETA trial showed a PFS hazard ratio of 0.46 versus placebo. Approval came with a REMS programme because QT prolongation requires ECG and electrolyte monitoring. LIBRETTO-531 then showed the RET-selective selpercatinib was superior first line to vandetanib or cabozantinib (PFS HR 0.28, 12-month PFS 86.8% versus 65.7%) with fewer grade 3 or higher adverse events (52.8% versus 76.4%), so vandetanib is now largely replaced. Its remaining role is in patients who cannot access or tolerate selective RET inhibitors. For a newcomer, it is the original medullary thyroid cancer pill, superseded by a drug built specifically for RET.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Vandetanib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Vandetanib"}],"tags":[],"related":[],"cancers":["thyroid","medullary-thyroid-cancer"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["ret","vegf","egfr"],"drugs":[],"companies":["sanofi"],"institutions":[],"pathways":[],"terms":[],"trials":["libretto-531"],"people":[],"bottlenecks":[],"keyPapers":["paper-zeta-vandetanib-mtc-wells-jco-2012"],"journals":[],"dependsOn":[],"notes":[],"brand":"Caprelsa","modality":"Small-molecule kinase inhibitor (RET, VEGFR, EGFR)","mechanism":"Multikinase inhibitor of RET, VEGFR2, and EGFR.","approvals":[{"region":"US","year":2011,"indication":"Symptomatic or progressive medullary thyroid cancer"}],"mechanismSteps":["Blocks RET kinase, the driver in hereditary and most sporadic MTC","Blocks VEGFR2, reducing tumour blood supply","Off-target EGFR and hERG inhibition cause rash, diarrhoea, and QT prolongation"],"dosing":{"route":"Oral","schedule":"300 mg once daily","monitoring":"ECG and electrolytes (QT prolongation REMS)","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/022405s015lbl.pdf"},"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2011-04-06","type":"approval","region":"US","note":"First approval for medullary thyroid cancer"}]},{"id":"varegacestat","kind":"drug","name":"Varegacestat","aka":[],"tldr":"Varegacestat is an experimental small-molecule drug from Immunome in phase 3 trials for desmoid tumour, with its target not yet stated publicly.","summary":"Varegacestat (AL102) is a small-molecule drug developed by Immunome. The sponsor describes its target as gamma secretase / Notch signalling, which OnCo does not yet have a target page for. The sponsor states: AL102 (varegacestat) is a small-molecule inhibitor of gamma secretase-mediated Notch signalling. ClinicalTrials.gov describes the intervention as: AL102 is an inhibitor of gamma secretase-mediated Notch signaling. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT04871282 (A Study of AL102 in Patients With Progressing Desmoid Tumours), in desmoid tumour. The largest, NCT04871282, plans to enrol 198 participants (actual) with primary completion was scheduled for 2025-12-03 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Varegacestat","url":"https://clinicaltrials.gov/search?intr=Varegacestat"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["desmoid-tumour"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["immunome"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04871282"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"AL102","modality":"small molecule","mechanism":"AL102 (varegacestat) is a small-molecule inhibitor of gamma secretase-mediated Notch signalling.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"varlitinib","kind":"drug","name":"Varlitinib","aka":[],"tldr":"A pill that blocked the HER family of growth receptors, tested with capecitabine as second-line treatment for bile duct and gallbladder cancer. It did not beat capecitabine alone in the TreeTopp trial and development stopped.","summary":"Varlitinib is an oral reversible inhibitor of EGFR, HER2 and HER4 developed by ASLAN Pharmaceuticals for biliary tract cancer on the rationale that EGFR and HER2 are overexpressed in a quarter to a half of these tumours. The global double-blind TreeTopp phase 2 randomised 127 patients with advanced biliary tract cancer after one gemcitabine-containing line to capecitabine with varlitinib 300 mg twice daily or placebo. Objective response was 9.4 versus 4.8 percent (p 0.42), median progression-free survival 2.83 versus 2.79 months (hazard ratio 0.90) and overall survival 7.8 versus 7.5 months (hazard ratio 1.11); grade 3 or worse adverse events occurred in 66 versus 59 percent. A subgroup analysis suggested a progression-free survival signal in women and in gallbladder cancer (2.9 versus 1.6 months, hazard ratio 0.55, 95 percent confidence interval 0.26 to 1.19), which was hypothesis-generating only. The planned phase 3 part was not pursued and varlitinib is not in development for biliary cancer. It stands as one of several HER-directed small molecules that failed in unselected biliary populations, in contrast to HER2-selected antibody therapy.","status":"historic","asOf":"2026-09-24","links":[{"label":"TreeTopp, ESMO Open 2022","url":"https://doi.org/10.1016/j.esmoop.2021.100314"},{"label":"ClinicalTrials.gov NCT03093870","url":"https://clinicaltrials.gov/study/NCT03093870"}],"tags":["failure"],"related":[],"cancers":["gallbladder","cholangiocarcinoma"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["egfr","her2"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["treetopp"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ASLAN001, ARRY-334543","modality":"Small-molecule reversible pan-HER kinase inhibitor (EGFR, HER2, HER4)","mechanism":"ATP-competitive reversible inhibition of EGFR, HER2 and HER4 kinases.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"varnimcabtagene-autoleucel","kind":"drug","name":"Varnimcabtagene autoleucel","aka":["var-cel","IMN-003A","ARI-0001"],"tldr":"India's second approved CAR-T therapy, a Bengaluru-made version of a Spanish hospital's academic CD19 cell therapy, for lymphoma that has come back after other treatments.","summary":"Varnimcabtagene autoleucel (var-cel) is the international nonproprietary name of ARI-0001, the CD19 CAR-T developed at Hospital Clinic de Barcelona and approved in Spain under the hospital exemption; Immuneel Therapeutics licensed it for India as IMN-003A and manufactures it at Narayana Health City in Bengaluru. Immuneel's phase 2 IMAGINE study, described as the first industry-sponsored CAR-T trial in India, treated adults with relapsed or refractory B-cell malignancies and was reported at ASH 2023 and in Hematological Oncology. On the strength of it Qartemi received CDSCO market authorisation in 2024 for relapsed or refractory B-cell lymphoma.\n\nIn Spain the same cell product has reported an 84.4% complete response with undetectable measurable residual disease by day 28 in 32 adults with relapsed B-ALL (CART19-BE-02, Lancet Haematology 2026), 100% response in follicular lymphoma and 89% in mantle cell lymphoma (HemaSphere 2025-26). Qartemi and NexCAR19 together give India two locally made CD19 CAR-Ts competing on price, something no other lower-middle-income country has.","status":"approved","asOf":"2026-09-10","links":[{"label":"Immuneel Therapeutics","url":"https://www.immuneel.com"},{"label":"IMAGINE study cytokine analysis (ASH 2023)","url":"https://doi.org/10.1182/blood-2023-181585"},{"label":"IMAGINE B-NHL subanalysis (Hematol Oncol 2023)","url":"https://doi.org/10.1002/hon.3165_632"},{"label":"ARI-0001 academic CAR-T review (Br J Haematol 2023)","url":"https://doi.org/10.1111/bjh.19170"}],"tags":[],"related":["talicabtagene-autoleucel","tisagenlecleucel"],"cancers":["dlbcl","all-leukemia"],"sections":[],"technologies":["car-t","point-of-care-cell-manufacturing"],"targets":["cd19"],"drugs":[],"companies":["immuneel"],"institutions":["narayana-health"],"pathways":[],"terms":["crs","icans"],"trials":["imagine-varnimcabtagene"],"people":[],"bottlenecks":["b-manufacturing-cell-therapy","b-global-access"],"keyPapers":["paper-martinez-cibrian-br-j-haematol"],"journals":[],"dependsOn":[],"notes":[],"brand":"Qartemi","code":"IMN-003A","modality":"CAR-T (CD19, academic origin)","mechanism":"Autologous T cells transduced with a lentiviral anti-CD19 (A3B1 scFv) CAR with 4-1BB and CD3-zeta domains; academic point-of-care design from Hospital Clinic de Barcelona.","approvals":[{"region":"IN","year":2024,"indication":"Relapsed or refractory B-cell lymphoma","note":"CDSCO market authorisation as announced by Immuneel; ARI-0001 is separately approved in Spain under the hospital exemption"}],"mechanismSteps":["Leukapheresis at the treating centre","Lentiviral transduction and expansion in Bengaluru","Lymphodepletion, then infusion (fractionated dosing was used in the Spanish programme)","CAR-T cells kill CD19-positive lymphoma cells","Monitoring for cytokine release syndrome and neurotoxicity"],"toxicity":[],"access":[{"country":"IN","reimbursement":"Public insurance cover not established; ask the treating centre","generic":false,"source":"https://www.immuneel.com","asOf":"2026-09-10"}],"regulatoryEvents":[]},{"id":"vb15010","kind":"drug","name":"VB15010","aka":[],"tldr":"VB15010 is a small-molecule inhibitor from Zhejiang Yangli Pharmaceutical Technology Co., Ltd., in registered phase 2 trials for ovarian cancer, prostate cancer, pancreatic ductal adenocarcinoma.","summary":"VB15010 is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Zhejiang Yangli Pharmaceutical Technology Co., Ltd., in ovarian cancer, prostate cancer, pancreatic ductal adenocarcinoma, biliary tract cancer, colorectal cancer. Described in the registry record as a oral parp1 inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of VB15010","url":"https://clinicaltrials.gov/search?intr=VB15010"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian","prostate","pancreatic","cholangiocarcinoma","colorectal"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06819215"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"VB15010","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a oral parp1 inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vc004","kind":"drug","name":"VC004","aka":[],"tldr":"VC004 is an experimental small-molecule drug from Jiangsu vcare pharmaceutical technology co. in phase 3 trials, with its target not yet stated publicly.","summary":"VC004 is a small-molecule drug developed by Jiangsu vcare pharmaceutical technology co.. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06658353 (Phase III Clinical Study of VC004 in Patients With Localized Advanced/ Metastatic Solid Tumours). The largest, NCT06658353, plans to enrol 54 participants with primary completion expected 2026-12-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of VC004","url":"https://clinicaltrials.gov/search?intr=VC004"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct06658353"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vebreltinib","kind":"drug","name":"Vebreltinib","aka":["Bozitinib","APL-101","CBT-101"],"tldr":"Vebreltinib is a Chinese MET inhibitor approved in 2023 for lung cancers with a MET exon 14 skipping mutation, and the first drug tested in a phase 3 trial for glioblastomas carrying a PTPRZ1-MET fusion.","summary":"Beijing Pearl Biotechnology's vebreltinib (PLB1001, also developed as APL-101 by Apollomics) was approved by China's NMPA in November 2023 for locally advanced or metastatic non-small cell lung cancer with MET exon 14 skipping. Because it crosses the blood-brain barrier, it was also taken into a randomised phase 3 trial in secondary glioblastoma with PTPRZ1-MET fusions, a rare molecular subgroup identified by Chinese neuro-oncology groups, and a further trial in MET-altered lung cancer is registered.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Vebreltinib","url":"https://clinicaltrials.gov/search?intr=PLB1001"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","glioblastoma","sclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["met"],"drugs":[],"companies":["apollomics","avistone-biotechnology"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06105619","nct05989542","nct06343064","nct03175224","nct06574347"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"PLB1001","modality":"Oral selective MET tyrosine kinase inhibitor","mechanism":"A selective, brain-penetrant inhibitor of the MET kinase; tumours with MET exon 14 skipping mutations, MET amplification or PTPRZ1-MET fusions depend on that signal.","approvals":[{"region":"CN","year":2023,"indication":"Locally advanced or metastatic non-small cell lung cancer with MET exon 14 skipping"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"veliparib","kind":"drug","name":"Veliparib","aka":[],"tldr":"Veliparib is a PARP inhibitor that AbbVie tested with chemotherapy in breast cancer. It added nothing to carboplatin before surgery (BrighTNess) and lengthened progression-free but not overall survival with carboplatin and paclitaxel in inherited BRCA advanced disease (BROCADE3), so it was never licensed.","summary":"Veliparib inhibits the catalytic activity of PARP1 and PARP2 but traps PARP on DNA far less than olaparib or talazoparib, which is why it could be combined with full-dose chemotherapy where the other PARP inhibitors cannot. That property was tested in two phase 3 breast cancer trials. In BrighTNess (634 patients, stage II to III triple-negative disease) paclitaxel with carboplatin and veliparib gave a pathological complete response in 53 percent against 58 percent with paclitaxel and carboplatin and 31 percent with paclitaxel alone, so the gain came from carboplatin; at 4.5 years the event-free survival hazard ratio for the veliparib arm against carboplatin alone was 1.12. In BROCADE3 (509 patients with a germline BRCA1 or BRCA2 mutation and HER2-negative advanced breast cancer, up to two prior chemotherapy lines) adding veliparib to carboplatin and paclitaxel improved median progression-free survival from 12.6 to 14.5 months (hazard ratio 0.71, p 0.0016) but final overall survival was 32.4 versus 28.2 months (hazard ratio 0.92, p 0.43), with grade 3 or worse neutropenia in 81 versus 84 percent and thrombocytopenia in 40 versus 28 percent. AbbVie did not file for approval in breast cancer; veliparib remains an investigational agent and the two trials are the reference for why weak PARP trapping with chemotherapy did not translate into survival.","status":"negative","asOf":"2026-09-24","wikipedia":"https://en.wikipedia.org/wiki/Veliparib","links":[{"label":"BrighTNess primary results (Lancet Oncology 2018)","url":"https://doi.org/10.1016/S1470-2045(18)30111-6"},{"label":"BrighTNess 4-year follow-up (Annals of Oncology 2022)","url":"https://doi.org/10.1016/j.annonc.2022.01.009"},{"label":"BROCADE3 primary results (Lancet Oncology 2020)","url":"https://doi.org/10.1016/S1470-2045(20)30447-2"},{"label":"BROCADE3 final overall survival (European Journal of Cancer 2024)","url":"https://doi.org/10.1016/j.ejca.2024.113580"}],"tags":[],"related":[],"cancers":["tnbc","tnbc-early","tnbc-metastatic","breast-hr-positive"],"sections":[],"technologies":["parp-inhibitor"],"targets":["parp","brca"],"drugs":[],"companies":["abbvie"],"institutions":[],"pathways":[],"terms":[],"trials":["brightness","brocade3"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ABT-888","modality":"Small-molecule PARP inhibitor (weak PARP trapper)","mechanism":"Catalytic inhibition of PARP1 and PARP2 with minimal PARP-DNA trapping; sensitises to platinum and alkylating chemotherapy.","approvals":[],"mechanismSteps":[],"toxicity":[{"event":"Neutropenia (grade 3 or worse), with carboplatin and paclitaxel","grade3PlusPct":81,"source":"https://doi.org/10.1016/S1470-2045(20)30447-2","note":"BROCADE3 veliparib arm; 84 percent in the placebo arm"},{"event":"Thrombocytopenia (grade 3 or worse)","grade3PlusPct":40,"source":"https://doi.org/10.1016/S1470-2045(20)30447-2","note":"BROCADE3 veliparib arm; 28 percent with placebo"}],"access":[],"regulatoryEvents":[]},{"id":"velzatinib","kind":"drug","name":"Velzatinib","aka":[],"tldr":"Velzatinib is an experimental investigational agent whose form is not stated in the registry from GlaxoSmithKline in phase 3 trials for gastrointestinal stromal tumour, with its target not yet stated publicly.","summary":"Velzatinib (GSK6042981) is an investigational agent whose form is not stated in the registry developed by GlaxoSmithKline. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07585266 (A Study to Investigate Velzatinib Compared With Imatinib in Adult Participants With Previously Untreated Metastatic and/or Unresectable Gastrointestinal Stromal Tumours (StrateGIST Frontline)), in gastrointestinal stromal tumour. The largest, NCT07585266, plans to enrol 800 participants with primary completion expected 2032-08-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Velzatinib","url":"https://clinicaltrials.gov/search?intr=GSK6042981"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["gist"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["gsk"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07585266","nct07689201"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"GSK6042981","modality":"not stated (registered as a drug or biological on ClinicalTrials.gov)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vemurafenib","kind":"drug","name":"Vemurafenib","aka":[],"tldr":"Vemurafenib was the first BRAF inhibitor (2011); it shrank melanomas in weeks and proved that a single mutation could be drugged in a solid tumour.","summary":"BRIM-3: OS benefit versus dacarbazine in BRAF V600E melanoma; approved August 2011 alongside ipilimumab in the same year that transformed the disease. Now used with cobimetinib (coBRIM) or displaced by encorafenib-binimetinib and dabrafenib-trametinib. Also approved for Erdheim-Chester disease. Photosensitivity, arthralgia, and cutaneous squamous cell carcinomas (from paradoxical MAPK activation in RAS-mutant keratinocytes) are characteristic.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Vemurafenib","links":[{"label":"BRIM-3, NEJM 2011","url":"https://www.nejm.org/doi/full/10.1056/NEJMoa1103782"}],"tags":[],"related":["braf-v600e"],"cancers":["melanoma","braf-v600-melanoma","advanced-melanoma","colorectal","braf-v600e-colorectal"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["braf"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["columbus","nct04302025","swog-s1406"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["In colorectal cancer vemurafenib alone is almost inactive because BRAF blockade causes feedback activation of EGFR. SWOG S1406 added it to irinotecan and cetuximab in previously treated BRAF V600E disease and improved progression-free survival (hazard ratio 0.50, p=0.001) and response (17 against 4 percent), the proof of principle that BEACON CRC and BREAKWATER turned into approved encorafenib-based regimens."],"brand":"Zelboraf","modality":"Small-molecule kinase inhibitor (BRAF V600)","mechanism":"ATP-competitive inhibitor of BRAF V600 monomers; paradoxically activates wild-type RAF dimers, hence MEK partnering.","approvals":[{"region":"US","year":2011,"indication":"Unresectable or metastatic BRAF V600E melanoma"},{"region":"US","year":2017,"indication":"Erdheim-Chester disease with BRAF V600 mutation"}],"mechanismSteps":["Vemurafenib enters the cell and binds the ATP pocket of mutant BRAF V600E","MEK and ERK phosphorylation fall within hours","Proliferation stops and apoptosis follows in BRAF-addicted melanoma cells","In RAS-active normal cells, drug-bound BRAF dimerises with CRAF and paradoxically boosts signalling, causing skin squamous lesions","Resistance emerges via NRAS mutation, BRAF splice variants, or MEK mutations, restoring ERK output"],"dosing":{"route":"Oral","schedule":"960 mg twice daily continuously","modifications":"Dose reduction for grade 2-3 rash, arthralgia, photosensitivity, QT prolongation","monitoring":"Dermatology exams for squamous cell carcinoma; ECG; liver enzymes","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/202429s019lbl.pdf"},"toxicity":[{"event":"Cutaneous squamous cell carcinoma / keratoacanthoma","anyGradePct":24,"source":"https://www.nejm.org/doi/full/10.1056/NEJMoa1103782","note":"BRIM-3 vemurafenib arm"},{"event":"Arthralgia","anyGradePct":53,"source":"https://www.nejm.org/doi/full/10.1056/NEJMoa1103782"},{"event":"Photosensitivity","anyGradePct":33,"source":"https://www.nejm.org/doi/full/10.1056/NEJMoa1103782"}],"access":[],"regulatoryEvents":[{"date":"2011-08-17","type":"approval","region":"US","note":"Approved with the cobas BRAF V600 companion test, the first melanoma targeted therapy"},{"date":"2015-11-10","type":"approval","region":"US","note":"Cobimetinib approved in combination (coBRIM)"}]},{"id":"venetoclax","kind":"drug","name":"Venetoclax","aka":[],"tldr":"A pill that removes the survival shield from leukaemia cells, enabling chemotherapy-free, time-limited treatment for CLL.","summary":"Venetoclax is a BH3-mimetic that selectively inhibits BCL-2, removing the survival shield that lets leukaemia cells ignore apoptotic signals. In CLL it enables chemotherapy-free, time-limited treatment: fixed-duration venetoclax with obinutuzumab (CLL14) or with ibrutinib, following approval in 2016 for CLL with 17p deletion. In AML it is combined with azacitidine, decitabine or low-dose cytarabine for patients unfit for intensive chemotherapy (VIALE-A, approved 2018). AbbVie and Genentech co-develop it, and tumour lysis syndrome is the key early risk, managed with a 5-week ramp-up from 20 to 400 mg daily in CLL and a 3-day ramp in AML. Open questions include MRD-guided treatment duration, use in fit AML patients and resistance through BCL-2 mutations or MCL-1. For a newcomer: a pill that reopens the cell's self-destruct switch.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Venetoclax","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Venetoclax"},{"label":"Blombery et al., Cancer Discov 2019: the recurrent BCL2 Gly101Val mutation confers resistance to venetoclax","url":"https://doi.org/10.1158/2159-8290.CD-18-1119"}],"tags":[],"related":[],"cancers":["cll","aml","aml-older-unfit"],"sections":[],"technologies":["kinase-inhibitors","bcl2-inhibitors"],"targets":["bcl2"],"drugs":[],"companies":["abbvie","roche-genentech"],"institutions":[],"pathways":[],"terms":["lymphoma-bio-antigen-escape"],"trials":["nct03844048","nct03539744","nct03314181","nct04068597","nct05951959","nct07387471","nct06115135","nct04988555","nct02899052","nct06382168","nct05673057","nct07007312","nct06943872","nct04588922","nct05947851","nct02966756","nct07277231","nct06428019","nct06846671","nct04229979","nct04256317","nct07743112","nct05211570","nct07154264","nct04581512","nct05520567","nct04965493","nct07581002","nct07024706","nct04086264","nct05963074","nct05057494","nct04895436","nct07255872","nct07520006","nct06456463","nct06524375"],"people":["peter-hillmen"],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Lymphoma biology: the escape mutation is in the drug's own binding groove. BCL2 G101V lowers affinity for venetoclax about 180-fold and was found at progression in 7 of 15 paired patients and in none at study entry, first detectable 19 to 42 months into treatment and months before clinical progression (Blombery 2019). The reason venetoclax has not repeated its chronic lymphocytic leukaemia result in lymphoma is different: those cells lean on MCL1 and BCL-xL as well as on BCL-2."],"brand":"Venclexta","modality":"Small-molecule BCL-2 inhibitor","mechanism":"BH3-mimetic selective BCL-2 inhibitor.","approvals":[{"region":"US","year":2016,"indication":"CLL with 17p deletion"},{"region":"US","year":2018,"indication":"AML with azacitidine/decitabine/LDAC in unfit patients"}],"mechanismSteps":["Venetoclax occupies the BH3-binding groove of BCL-2","Pro-apoptotic BIM and BAX/BAK are released","Mitochondrial outer membrane is permeabilised","Cytochrome c triggers caspase activation","Leukaemic cells die within hours (tumour lysis risk)"],"dosing":{"route":"Oral","schedule":"CLL: 5-week ramp-up 20 → 50 → 100 → 200 → 400 mg daily, then 400 mg daily (fixed 12 months with obinutuzumab); AML: 3-day ramp to 400 mg daily with azacitidine (100 mg with posaconazole)","modifications":"TLS prophylaxis (hydration, allopurinol; hospitalisation for high tumour burden); reduce with CYP3A inhibitors","monitoring":"Chemistry and uric acid at each ramp step; blood counts","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Neutropenia","note":"CLL14: ~53% grade 3-4 with obinutuzumab"},{"event":"Diarrhoea"},{"event":"Nausea"},{"event":"Anaemia"},{"event":"Upper respiratory infection"},{"event":"Tumour lysis syndrome","note":"Prevented by ramp-up; rare with prophylaxis"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.venclexta.com/support","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended in CLL (TA487, TA561, TA663) and AML with azacitidine (TA765)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2016-04-11","type":"accelerated-approval","region":"US","note":"CLL with 17p deletion after ≥1 therapy: first BCL-2 inhibitor","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Chronic lymphocytic leukemia with 17P deletion as detected by an FDA-approved test, after at least one prior therapy"},{"date":"2018-06-08","type":"conversion","region":"US","note":"CLL/SLL with rituximab (MURANO)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Chronic lymphocytic leukemia with 17P deletion as detected by an FDA-approved test, after at least one prior therapy"},{"date":"2018-11-21","type":"accelerated-approval","region":"US","note":"Newly diagnosed AML in unfit patients with azacitidine/decitabine/LDAC (accelerated)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with azacitidine or decitabine or low-dose cytarabine for newly-diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy"},{"date":"2019-05-15","type":"approval","region":"US","note":"First-line CLL with obinutuzumab (CLL14)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2020-10-16","type":"conversion","region":"US","note":"Full approval in AML (VIALE-A)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with azacitidine or decitabine or low-dose cytarabine for newly-diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy"}]},{"id":"ventana-mmr-rxdx","kind":"drug","name":"VENTANA MMR RxDx Panel","aka":[],"tldr":"A four-stain test that shows whether a womb cancer has lost its DNA spell-checker, which makes immunotherapy likely to work.","summary":"Approved by the FDA in August 2021 together with dostarlimab for mismatch-repair-deficient (dMMR) recurrent or advanced endometrial cancer, the first immunohistochemistry companion diagnostic for mismatch-repair status. MMR immunohistochemistry is cheap, fast and available in every pathology laboratory, and is the recommended first-line test in endometrial and colorectal cancer, where it also serves as universal screening for Lynch syndrome. Where results are equivocal, PCR or NGS-based microsatellite-instability testing is used. Immunotherapy responses in dMMR tumours are among the most durable in oncology, so the stain has outsized consequences for a patient.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA: dostarlimab accelerated approval (dMMR endometrial cancer) (archived copy)","url":"https://web.archive.org/web/20260217063458/https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-dostarlimab-gxly-dmmr-endometrial-cancer"}],"tags":["test"],"related":[],"cancers":["endometrial","colorectal","pancreatic"],"sections":[],"technologies":["msi-mmr-testing","companion-diagnostic","histopathology-ihc"],"targets":[],"drugs":["dostarlimab","pembrolizumab"],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["msi","mss-pmmr","lynch-syndrome","endometrial-molecular-classes"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-luchini-msi-dmmr-pancreatic-systematic-review-gut-2021","paper-moreira-lynch-syndrome-identification-jama-2012","paper-sepulveda-molecular-biomarkers-colorectal-guideline-jco-2017"],"journals":[],"dependsOn":[],"notes":["What a result means: loss of one or more MMR proteins (dMMR) means checkpoint immunotherapy is an option and that inherited Lynch syndrome should be considered; intact staining (pMMR) means other treatments come first.","Colorectal cancer: the four-protein mismatch repair immunohistochemistry panel is the guideline-recommended first test on every colorectal cancer, both to find Lynch syndrome and to select immunotherapy (Moreira 2012, Sepulveda 2017)."],"brand":"VENTANA MMR RxDx","modality":"Mismatch-repair immunohistochemistry companion diagnostic assay","mechanism":"Four immunohistochemical stains (MLH1, PMS2, MSH2, MSH6) read together; loss of nuclear staining for any protein classifies the tumour as mismatch-repair deficient.","approvals":[{"region":"US","year":2021,"indication":"Companion diagnostic for dostarlimab in dMMR endometrial cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ventana-pd-l1-sp142","kind":"drug","name":"VENTANA PD-L1 (SP142) Assay","aka":[],"tldr":"The PD-L1 stain that decides who can have atezolizumab, scored on immune cells rather than tumour cells in breast cancer.","summary":"Approved 18 May 2016 (PMA P160002) as the first PD-L1 assay linked to atezolizumab, in urothelial carcinoma, and subsequently in non-small-cell lung cancer and, in 2019, as the companion diagnostic for atezolizumab plus nab-paclitaxel in triple-negative breast cancer using an immune-cell threshold of 1% (IMpassion130). SP142 stains fewer tumour cells than 22C3 or SP263, and the Blueprint comparison studies showed it is not interchangeable with them, which is why each drug label names its own assay. The breast cancer indication for atezolizumab was later withdrawn in the US, but the assay remains in use in Europe and for lung cancer.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P160002","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P160002"}],"tags":["test"],"related":["dako-pd-l1-22c3-pharmdx","ventana-pd-l1-sp263"],"cancers":["urothelial","nsclc","tnbc","tnbc-metastatic"],"sections":[],"technologies":["companion-diagnostic","histopathology-ihc"],"targets":["pdl1"],"drugs":["atezolizumab"],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["pd-l1-testing","ihc"],"trials":["impassion130"],"people":[],"bottlenecks":[],"keyPapers":["paper-rugo-pd-l1-assay-comparison-impassion130-jnci-2021","paper-sigurjonsdottir-sp142-22c3-tnbc-bcr-2023"],"journals":[],"dependsOn":[],"notes":["What a result means: 'PD-L1 positive' on this assay refers to immune cells in the tumour; a different assay with a different cut-off may give a different answer, so ask which test was used."],"brand":"VENTANA PD-L1 (SP142)","modality":"PD-L1 immunohistochemistry companion diagnostic assay","mechanism":"Rabbit monoclonal antibody SP142 with OptiView amplification on the BenchMark stainer; scored on tumour cells and on tumour-infiltrating immune cells (IC), the latter being the basis of the breast cancer claim.","approvals":[{"region":"US","year":2016,"indication":"PD-L1 expression to inform atezolizumab treatment in urothelial carcinoma (later NSCLC)"},{"region":"US","year":2019,"indication":"Companion diagnostic for atezolizumab in PD-L1 IC ≥1% triple-negative breast cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ventana-pd-l1-sp263","kind":"drug","name":"VENTANA PD-L1 (SP263) Assay","aka":[],"tldr":"The VENTANA SP263 assay is an immunohistochemistry stain that measures PD-L1 on tumour cells. Approved in 2017 alongside durvalumab in bladder cancer, it became the companion test in 2021 for adjuvant atezolizumab in resected lung cancer with PD-L1 on at least 1% of tumour cells, and it agrees closely with 22C3, so laboratories often validate it as a single platform.","summary":"Approved 1 May 2017 (PMA P160046) alongside durvalumab in urothelial carcinoma as a complementary test, SP263 became a companion diagnostic in 2021 for adjuvant atezolizumab in resected stage II-IIIA non-small-cell lung cancer with PD-L1 on at least 1% of tumour cells (IMpower010). In Europe the CE-IVD version carries claims for pembrolizumab, nivolumab and durvalumab across lung, bladder and head and neck cancers, and SP263 is the assay most often shown to be concordant with 22C3 in harmonisation studies. Laboratories frequently validate SP263 as a single platform for several drugs.","status":"approved","asOf":"2026-09-10","links":[{"label":"FDA PMA P160046","url":"https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfpma/pma.cfm?id=P160046"}],"tags":["test"],"related":[],"cancers":["urothelial","nsclc","tnbc"],"sections":[],"technologies":["companion-diagnostic","histopathology-ihc"],"targets":["pdl1"],"drugs":["durvalumab","atezolizumab"],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["pd-l1-testing","ihc"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":["paper-rugo-pd-l1-assay-comparison-impassion130-jnci-2021"],"journals":[],"dependsOn":[],"notes":["What a result means: the percentage of tumour cells that stain; higher scores generally predict more benefit from immunotherapy, and thresholds differ by drug and cancer."],"brand":"VENTANA PD-L1 (SP263)","modality":"PD-L1 immunohistochemistry companion diagnostic assay","mechanism":"Rabbit monoclonal antibody SP263 on the BenchMark ULTRA stainer, scored as the percentage of tumour cells with membrane staining (tumour-cell score).","approvals":[{"region":"US","year":2017,"indication":"Complementary PD-L1 assay for durvalumab in urothelial carcinoma"},{"region":"US","year":2021,"indication":"Companion diagnostic for adjuvant atezolizumab in PD-L1 TC ≥1% resected NSCLC"},{"region":"EU","year":2017,"indication":"CE-IVD with claims for several PD-1/PD-L1 antibodies"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vepdegestrant","kind":"drug","name":"Vepdegestrant","aka":[],"tldr":"Vepdegestrant is the first PROTAC ever approved (2026): a pill that tags the oestrogen receptor for destruction, for breast cancers with ESR1 mutations.","summary":"Vepdegestrant is a PROTAC: a heterobifunctional molecule with one end binding oestrogen receptor alpha and the other recruiting the cereblon E3 ligase, tagging the receptor for proteasomal destruction rather than simply blocking it. Taken as 200 mg once daily, it was approved in Q2 2026 for ESR1-mutated ER-positive, HER2-negative advanced breast cancer after endocrine therapy, the first PROTAC ever approved (Arvinas/Pfizer). In VERITAC-2 it improved progression-free survival versus fulvestrant to 5.0 versus 2.1 months (HR 0.57) in ESR1-mutant disease after CDK4/6 and endocrine therapy, but showed no benefit in the overall population, so the label is restricted to ESR1-mutant tumours. The absolute gain is modest, and combinations with CDK4/6 inhibitors are the next test. For a newcomer: a pill that dismantles the oestrogen receptor instead of just blocking it.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Vepdegestrant"}],"tags":[],"related":[],"cancers":["breast-hr-positive","hr-positive-metastatic-post-cdk46"],"sections":[],"technologies":["protac-degrader","endocrine-therapy"],"targets":["estrogen-receptor"],"drugs":[],"companies":["arvinas","pfizer"],"institutions":[],"pathways":[],"terms":[],"trials":["veritac-2","nct04606446","nct06206837","nct05548127","nct05909397","nct05573555"],"people":[],"bottlenecks":[],"keyPapers":["paper-protac-concept-sakamoto-pnas-2001"],"journals":[],"dependsOn":[],"notes":[],"brand":"Veppanu","code":"ARV-471","modality":"PROTAC oestrogen receptor degrader","mechanism":"Heterobifunctional molecule recruiting cereblon E3 ligase to ERα for proteasomal degradation.","approvals":[{"region":"US","year":2026,"indication":"ESR1-mutated ER+/HER2- advanced or metastatic breast cancer after endocrine therapy"}],"mechanismSteps":["Oral heterobifunctional molecule enters the tumour cell","One end binds ERα, the other recruits the cereblon E3 ubiquitin ligase","ERα is ubiquitinated and destroyed by the proteasome","The drug is released and repeats the cycle (catalytic)","ER-driven transcription stops even with ligand-independent ESR1 mutations"],"dosing":{"route":"Oral","schedule":"200 mg once daily","modifications":"Reduce for grade 3 toxicity","monitoring":"LFTs; lipids","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Fatigue"},{"event":"Nausea"},{"event":"Arthralgia"},{"event":"Hot flush"},{"event":"Transaminase increase"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","assistance":"https://www.pfizeroncologytogether.com","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-02","type":"designation","region":"US","note":"Fast Track designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-06","type":"filing","region":"US","note":"NDA submitted (VERITAC-2)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-Q2","type":"approval","region":"US","note":"ESR1-mutated ER+/HER2- advanced breast cancer after endocrine therapy: first PROTAC approved","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"vepugratinib","kind":"drug","name":"Vepugratinib","aka":[],"tldr":"Vepugratinib is an experimental small-molecule drug from Eli Lilly and in phase 3 trials for bladder & urothelial cancer, with its target not yet stated publicly.","summary":"Vepugratinib is a small-molecule drug developed by Eli Lilly and. The sponsor describes its target as FGFR3, which OnCo does not yet have a target page for. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT07218380 (A Study of Vepugratinib (LY3866288) in Participants With Cancer in the Urinary Tract), in bladder & urothelial cancer. The largest, NCT07218380, plans to enrol 450 participants with primary completion expected 2029-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Vepugratinib","url":"https://clinicaltrials.gov/search?intr=Vepugratinib"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07218380"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vibostolimab","kind":"drug","name":"Vibostolimab","aka":["MK-7684"],"tldr":"Vibostolimab is a monoclonal antibody from Merck Sharp & Dohme LLC, in registered phase 2 trials for non-small-cell lung cancer.","summary":"Vibostolimab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Merck Sharp & Dohme LLC, in non-small-cell lung cancer. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Vibostolimab","url":"https://clinicaltrials.gov/search?intr=Vibostolimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04165070"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vimseltinib","kind":"drug","name":"Vimseltinib","aka":[],"tldr":"Vimseltinib is a pill approved in February 2025 for tenosynovial giant cell tumour, a benign but destructive joint tumour, offering an alternative to repeated surgery.","summary":"Vimseltinib is a switch-control kinase inhibitor of CSF1R. Tenosynovial giant cell tumour is driven by CSF1 overexpression from a COL6A3-CSF1 translocation, which recruits CSF1R-positive macrophages to form the tumour mass, so blocking the receptor disperses the bulk of the lesion. Taken twice weekly, it was tested in the phase 3 MOTION trial, where the ORR at week 25 was 40% versus 0% on placebo, with improvements in range of motion, pain, stiffness and function (Lancet 2024). The FDA approved it on 14 February 2025 for symptomatic TGCT where surgery would worsen function or cause severe morbidity. No liver toxicity signal of the kind that restricted pexidartinib, the first CSF1R inhibitor, was seen; periorbital oedema was the main effect; Deciphera and Ono develop it. It is a pill for a benign but destructive joint tumour that shrinks it by dispersing the immune cells it is made of.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Vimseltinib"}],"tags":[],"related":[],"cancers":["sarcoma","tenosynovial-giant-cell-tumour"],"sections":[],"technologies":["kinase-inhibitors"],"targets":[],"drugs":[],"companies":["deciphera"],"institutions":[],"pathways":[],"terms":[],"trials":["motion","nct03069469"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Romvimza","modality":"Small-molecule kinase inhibitor (CSF1R)","mechanism":"Switch-control inhibitor of CSF1R; TGCT is driven by CSF1 overexpression (COL6A3-CSF1 translocation) recruiting CSF1R+ macrophages.","approvals":[{"region":"US","year":2025,"indication":"Symptomatic tenosynovial giant cell tumour where surgery would worsen function or cause severe morbidity"},{"region":"EU","year":2025,"indication":"Symptomatic TGCT where surgery has been exhausted or would cause unacceptable morbidity; 17 Sep 2025"}],"mechanismSteps":[],"dosing":{"route":"Oral","schedule":"30 mg twice weekly","monitoring":"Liver enzymes, CPK, cholesterol, periorbital oedema"},"toxicity":[{"event":"Periorbital oedema","anyGradePct":45,"note":"MOTION"},{"event":"Fatigue","anyGradePct":33},{"event":"Increased CPK","anyGradePct":28},{"event":"Pruritus","anyGradePct":28}],"access":[],"regulatoryEvents":[{"date":"2025-02-14","type":"approval","region":"US","note":"MOTION","source":"https://www.cancernetwork.com/view/fda-approves-vimseltinib-in-tenosynovial-giant-cell-tumor"}]},{"id":"vinblastine","kind":"drug","name":"Vinblastine","aka":[],"tldr":"Vinblastine is the vinca alkaloid in ABVD, the standard Hodgkin lymphoma regimen, and was formerly in the cisplatin combinations that first cured testicular cancer.","summary":"Vinblastine is a vinca alkaloid that binds tubulin and blocks mitotic spindle formation. It is more myelosuppressive and less neurotoxic than vincristine, which is why the two are chosen for different regimens. Approved in the US in 1965, it is the V in ABVD, the standard chemotherapy for Hodgkin lymphoma, and was in PVB, the cisplatin combination that first cured metastatic testicular cancer. It also features in MVAC for bladder cancer, in treatment of Kaposi sarcoma and Langerhans cell histiocytosis, and with carboplatin for low-grade glioma in children. It is a vesicant that causes severe tissue injury if it leaks from the vein, and myelosuppression is the dose-limiting toxicity. Sixty years on, vinblastine is still curing patients within well-tested combinations.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Vinblastine","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=vinblastine"}],"tags":["gap-fill","generic","who-essential"],"related":[],"cancers":["hodgkin-lymphoma","testicular","urothelial","kaposi-sarcoma","lch-single-system","lch-multisystem","early-stage-classical-hodgkin-lymphoma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03407144","nct02979522","nct06960577"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Velban","modality":"Vinca alkaloid (microtubule inhibitor)","mechanism":"Tubulin-binding antimitotic; more myelosuppressive and less neurotoxic than vincristine.","approvals":[{"region":"US","year":1965,"indication":"Hodgkin lymphoma, testicular cancer, Kaposi sarcoma, choriocarcinoma, breast cancer and others"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vincristine","kind":"drug","name":"Vincristine","aka":[],"tldr":"A plant-derived chemotherapy from the Madagascar periwinkle that has been in almost every childhood leukaemia and lymphoma regimen since the 1960s.","summary":"Approved 1963. Component of CHOP, ABVD-era regimens, ALL induction/maintenance, VAC/VDC (rhabdomyosarcoma, Ewing), Wilms tumour (VA/DD-4A), neuroblastoma and medulloblastoma regimens, EMA-CO. Fatal if given intrathecally; vincristine shortages (2019) disrupted paediatric care. Liposomal vincristine (Marqibo, 2012) for Ph-negative ALL was discontinued 2022.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Vincristine","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=vincristine"}],"tags":["gap-fill","generic","who-essential"],"related":[],"cancers":["all-leukemia","dlbcl","hodgkin-lymphoma","rhabdomyosarcoma","ewing-sarcoma","wilms-tumor","neuroblastoma","medulloblastoma","retinoblastoma","oligodendroglioma"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["rtog-9402","eortc-26951","nct06097364","nct04529772","nct06717347","nct06191744","nct06911502","nct06091865","nct06091254","nct05888493","nct03407144","nct04663347","nct04542824","nct05201248","nct07730515","nct06564038","nct04623541","nct04980222","nct07387926","nct06947967","nct06425302"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Oncovin / Marqibo","modality":"Vinca alkaloid (microtubule inhibitor)","mechanism":"Binds β-tubulin and prevents microtubule polymerisation, arresting mitosis; dose-limited by peripheral neuropathy.","approvals":[{"region":"US","year":1963,"indication":"Acute leukaemia; Hodgkin and non-Hodgkin lymphoma, rhabdomyosarcoma, neuroblastoma, Wilms tumour"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[{"date":"2012-08-09","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint, with a confirmatory trial required.","indication":"Adults with Philadelphia (PH) chromosome negative (-) ALL in second relapse or greater relapsed or whose disease has progressed following 2 or greater treatment lines of anti-leukemia therapies"},{"date":"2022-05-02","type":"withdrawal","region":"US","note":"Withdrawn: the indication came off the label 9.7 years after its accelerated approval.","indication":"Adults with Philadelphia (PH) chromosome negative (-) ALL in second relapse or greater relapsed or whose disease has progressed following 2 or greater treatment lines of anti-leukemia therapies"}]},{"id":"vindesine","kind":"drug","name":"Vindesine","aka":["Desacetylvinblastine amide","DVA"],"tldr":"Vindesine is a vinca alkaloid, a semi-synthetic relative of vinblastine, used in Europe and elsewhere for acute lymphoblastic leukaemia and some solid tumours; it was never marketed in the United States.","summary":"Vindesine is made from vinblastine and shares the class mechanism of the vinca alkaloids, binding tubulin so microtubules cannot form. It has been used in acute lymphoblastic leukaemia in children and adults, in non-small cell lung cancer combinations in the 1980s and 1990s, and in melanoma and breast cancer, mostly in European protocols. Peripheral neuropathy is its dose-limiting toxicity. It remains a licensed medicine in several European countries but has largely given way to vincristine and vinorelbine.","status":"established","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Vindesine","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Vindesine"},{"label":"ChEMBL CHEMBL1200937","url":"https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1200937"}],"tags":["gap-fill","chembl-universe"],"related":[],"cancers":["all-leukemia","nsclc"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":["tubulin"],"drugs":["vinblastine","vincristine","vinorelbine"],"companies":["eli-lilly"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04221035","nct03206671","nct01117441"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Eldisine","modality":"Vinca alkaloid, intravenous","mechanism":"Binds tubulin and blocks microtubule assembly, arresting dividing cells in metaphase.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vinflunine","kind":"drug","name":"Vinflunine","aka":[],"tldr":"Vinflunine (Javlor) is a chemotherapy infusion used in Europe as a second treatment for advanced bladder cancer after platinum chemotherapy has stopped working.","summary":"Vinflunine was authorised by the European Commission in September 2009 as monotherapy for advanced or metastatic transitional cell carcinoma of the urothelial tract after failure of a prior platinum-containing regimen, on a phase 3 trial of 370 patients against best supportive care in which median overall survival improved modestly, a statistically significant difference in the eligible population, making it the first drug with level 1 evidence in second-line urothelial cancer. It is not approved in the US, where the FDA declined the application. Pembrolizumab, enfortumab vedotin and erdafitinib have since displaced it in most guidelines, but it remains an option where immunotherapy is unsuitable. Neutropenia, constipation (prophylactic laxatives are recommended), fatigue and QT prolongation are the main adverse effects. Marketed by Pierre Fabre.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Vinflunine","links":[{"label":"EPAR (EMA)","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/javlor"}],"tags":["ema-list"],"related":["vinorelbine","vinblastine"],"cancers":["urothelial"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["pierre-fabre"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07419295"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Javlor","modality":"Fluorinated vinca alkaloid (microtubule inhibitor)","mechanism":"Third-generation vinca alkaloid that binds tubulin with lower affinity than vinorelbine, suppressing microtubule dynamics and causing mitotic arrest; less neurotoxic than earlier vincas.","approvals":[{"region":"EU","year":2009,"indication":"Advanced or metastatic transitional cell carcinoma of the urothelial tract after failure of a platinum-containing regimen"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vinorelbine","kind":"drug","name":"Vinorelbine","aka":[],"tldr":"Vinorelbine is a vinca chemotherapy for lung and breast cancer, available orally, and is now part of maintenance therapy that improved survival in childhood rhabdomyosarcoma.","summary":"Vinorelbine is a semi-synthetic vinca alkaloid that binds tubulin and blocks mitosis, with relative selectivity for mitotic over axonal microtubules and therefore less neuropathy than older vincas. It was approved in the EU in 1989 and in the US in 1994 for advanced NSCLC as a single agent or with cisplatin, and cisplatin-vinorelbine was the adjuvant regimen in the ANITA and JBR.10 trials that established post-operative chemotherapy in lung cancer. It is also used in metastatic breast cancer, mesothelioma and Hodgkin lymphoma salvage, and its oral formulation allows metronomic low-dose schedules. In children with high-risk rhabdomyosarcoma, maintenance vinorelbine with cyclophosphamide improved overall survival in the EpSSG RMS 2005 trial. Neutropenia is the main toxicity. Vinorelbine is an old chemotherapy that keeps finding new roles.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Vinorelbine","links":[{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=vinorelbine"}],"tags":["gap-fill","generic"],"related":[],"cancers":["nsclc","rhabdomyosarcoma","breast-hr-positive","mesothelioma","lung-cancer","resectable-nsclc"],"sections":[],"technologies":["cytotoxic-chemotherapy"],"targets":[],"drugs":[],"companies":["pierre-fabre"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06435429","nct06459180","nct02486718","nct06081959","nct06908304","nct06202261","nct05999994","nct06343948","nct07007559","nct04639986","jbr-10","anita","ialt","lace-pooled-analysis"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Navelbine","modality":"Semi-synthetic vinca alkaloid","mechanism":"Tubulin-binding antimitotic with relative selectivity for mitotic over axonal microtubules (less neuropathy).","approvals":[{"region":"US","year":1994,"indication":"Advanced NSCLC as single agent or with cisplatin"},{"region":"EU","year":1989,"indication":"NSCLC, metastatic breast cancer"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vismodegib","kind":"drug","name":"Vismodegib","aka":[],"tldr":"Vismodegib was the first hedgehog-pathway drug, for basal cell carcinomas too advanced for surgery; it shrinks most tumours but muscle cramps, taste loss and hair loss make long-term use hard.","summary":"ERIVANCE (NEJM 2012): 30% response in metastatic and 43% in locally advanced BCC; long-term follow-up ~50-60% response in locally advanced disease. Teratogenic (pregnancy prevention programme). Also used in SHH medulloblastoma (adults) and studied neoadjuvantly (VISMONEO). Resistance via SMO mutations.","status":"approved","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/Vismodegib","links":[{"label":"ERIVANCE (NEJM 2012)","url":"https://doi.org/10.1056/NEJMoa1113713"},{"label":"Label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=vismodegib"}],"tags":["gap-fill"],"related":[],"cancers":["basal-cell-carcinoma","medulloblastoma","medulloblastoma-shh","locally-advanced-bcc"],"sections":[],"technologies":["kinase-inhibitors","hedgehog-inhibitors"],"targets":["smoothened"],"drugs":[],"companies":["roche-genentech"],"institutions":[],"pathways":[],"terms":["hedgehog-inhibitor-tolerability"],"trials":["nct06344052","stevie","vismoneo"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Erivedge","modality":"Small-molecule smoothened (hedgehog) inhibitor","mechanism":"Binds and inhibits SMO, preventing GLI-mediated transcription downstream of PTCH1 loss.","approvals":[{"region":"US","year":2012,"indication":"Metastatic BCC or locally advanced BCC recurrent after surgery or not amenable to surgery/radiation"},{"region":"EU","year":2013,"indication":"Same"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vistusertib","kind":"drug","name":"Vistusertib","aka":[],"tldr":"Vistusertib is an oral serine/threonine kinase inhibitor from AstraZeneca, in registered phase 2 trials for non-small-cell lung cancer, small-cell lung cancer.","summary":"Vistusertib is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by AstraZeneca, in non-small-cell lung cancer, small-cell lung cancer. A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Vistusertib","url":"https://clinicaltrials.gov/search?intr=Vistusertib"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["nsclc","sclc"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":[],"trials":["nct03334617"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Oral serine/threonine kinase inhibitor","mechanism":"A serine/threonine kinase inhibitor: the name stem -sertib marks a small molecule that blocks a kinase in this family.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"visugromab","kind":"drug","name":"Visugromab","aka":[],"tldr":"Visugromab is CatalYm's antibody against GDF-15, a tumour-made signal that both blocks immune cells and causes cancer wasting; it is in a phase 3 trial with PD-1 blockade after durable responses in patients whose cancers had stopped responding to checkpoint inhibitors.","summary":"CatalYm, a Munich biotech, developed visugromab (CTL-002) after showing that GDF-15 secreted by tumours prevents T cells from entering them. In the GDFATHER trials it produced durable responses with nivolumab in patients with checkpoint-refractory cancers, and the same molecule is being studied for cancer-associated cachexia, since GDF-15 acts on the brainstem to suppress appetite. A phase 3 trial is registered.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Visugromab","url":"https://clinicaltrials.gov/search?intr=CTL-002"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":[],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":[],"drugs":[],"companies":["catalym"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07112196","nct07219459","nct07098988","nct07246863","nct04725474"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"CTL-002","modality":"Anti-GDF-15 monoclonal antibody, intravenous","mechanism":"Neutralises growth differentiation factor 15, a cytokine many tumours secrete that keeps T cells out of the tumour and drives cachexia; blocking it is meant to restore immune infiltration and reverse weight loss.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vorasidenib","kind":"drug","name":"Vorasidenib","aka":[],"tldr":"The first targeted therapy for low-grade brain tumours, delaying the need for radiation and chemotherapy by years.","summary":"Vorasidenib is a brain-penetrant dual inhibitor of mutant IDH1 and IDH2 that lowers the oncometabolite 2-hydroxyglutarate (2-HG), which drives the epigenetic block behind IDH-mutant glioma. In INDIGO it extended progression-free survival to 27.7 versus 11.1 months in grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery, and it was approved in August 2024 for patients aged 12 and over, the first targeted therapy for low-grade brain tumours. Servier markets it; the dose is 40 mg once daily (20 mg below 40 kg), and raised ALT and AST are the main laboratory findings. Its purpose is to delay radiotherapy and chemotherapy and their long-term cognitive cost by years; whether it improves overall survival, and its role in higher-grade IDH-mutant tumours, are open. For a newcomer: a pill that holds slow-growing brain tumours in check and postpones harsher treatment.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Vorasidenib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Vorasidenib"}],"tags":[],"related":["idh1-r132","idh2-mutation"],"cancers":["glioblastoma","idh-mutant-astrocytoma","oligodendroglioma"],"sections":[],"technologies":["epigenetic-drugs","idh-inhibitors"],"targets":["idh"],"drugs":[],"companies":["servier"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Voranigo","modality":"Small-molecule IDH1/2 inhibitor","mechanism":"Brain-penetrant dual IDH1/IDH2 inhibitor reducing 2-HG.","approvals":[{"region":"US","year":2024,"indication":"Grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery, age ≥12"},{"region":"EU","year":2025,"indication":"Grade 2 IDH-mutant glioma ≥12 yrs; 17 Sep 2025"}],"mechanismSteps":["Brain-penetrant drug crosses the blood-brain barrier","Binds mutant IDH1 and IDH2 enzymes","Production of the oncometabolite 2-hydroxyglutarate stops","Histone and DNA methylation blocks are lifted; glioma cells differentiate","Tumour growth slows for years, delaying radiation and chemotherapy"],"dosing":{"route":"Oral","schedule":"40 mg once daily (≥40 kg); 20 mg once daily (<40 kg)","modifications":"Hold for grade 2 hepatotoxicity; discontinue for grade 4","monitoring":"LFTs every 2 weeks for 2 months, then monthly for 2 years","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"ALT increased","note":"INDIGO: ~39% any grade, ~9.6% grade 3-4"},{"event":"AST increased"},{"event":"Fatigue"},{"event":"Headache"},{"event":"COVID-19"},{"event":"Musculoskeletal pain"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"},{"country":"UK","reimbursement":"NICE: recommended for grade 2 IDH-mutant glioma after surgery (2025)","source":"https://www.nice.org.uk/guidance/published?ngt=Technology%20appraisal%20guidance","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-03","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-08-06","type":"approval","region":"US","note":"Grade 2 IDH-mutant astrocytoma or oligodendroglioma after surgery, age ≥12 (INDIGO): first targeted therapy for low-grade glioma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-06","type":"approval","region":"EU","note":"EMA approval","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"vorinostat","kind":"drug","name":"Vorinostat","aka":["SAHA","Suberoylanilide hydroxamic acid"],"tldr":"Vorinostat (Zolinza) was the first drug of its kind, a capsule that loosens the chemical packaging of DNA. It treats the skin disease of cutaneous T-cell lymphoma after two other treatments have failed.","summary":"Vorinostat was approved by the FDA in October 2006 for cutaneous manifestations of cutaneous T-cell lymphoma in patients with progressive, persistent or recurrent disease on or after two systemic therapies, on two open-label studies (response rate about 30% in the pivotal single-arm study of 74 patients). It was the first histone deacetylase inhibitor approved for any cancer and remains a standard option in refractory mycosis fungoides and Sezary syndrome; a European application was withdrawn and it is not authorised in the EU. Fatigue, diarrhoea, thrombocytopenia, taste change and QT prolongation are the main adverse effects; thromboembolism is a class warning.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Vorinostat","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=vorinostat"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/vorinostat"}],"tags":["nci-list"],"related":["romidepsin","bexarotene"],"cancers":[],"sections":[],"technologies":["epigenetic-drugs"],"targets":["hdac"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Cutaneous T-cell lymphoma has no cancer record yet."],"brand":"Zolinza","modality":"Small-molecule pan-HDAC inhibitor (hydroxamate)","mechanism":"Inhibits class I (HDAC1, 2, 3) and class II (HDAC6) histone deacetylases at nanomolar concentrations, causing histone hyperacetylation, re-expression of silenced genes, cell-cycle arrest and apoptosis.","approvals":[{"region":"US","year":2006,"indication":"Cutaneous manifestations of CTCL with progressive, persistent or recurrent disease after two systemic therapies"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vorolanib","kind":"drug","name":"Vorolanib","aka":["CM082","Fuwei"],"tldr":"Vorolanib is Betta Pharmaceuticals' oral VEGFR and PDGFR inhibitor, approved in China in 2023 with everolimus for advanced kidney cancer after a first kinase inhibitor has failed.","summary":"Vorolanib (X-82, CM082) was developed by Betta Pharmaceuticals in Hangzhou, licensed from Tyrogenex. The NMPA approved it in June 2023 in combination with everolimus for advanced renal cell carcinoma in patients whose disease had progressed on a prior tyrosine kinase inhibitor, on the CONCEPT phase 3 trial, which compared the combination with everolimus alone. It is also in trials in lung cancer and, as an eye drop formulation, in retinal disease.","status":"approved","asOf":"2026-09-16","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Vorolanib"},{"label":"ClinicalTrials.gov: trials of vorolanib","url":"https://clinicaltrials.gov/search?intr=vorolanib"}],"tags":["china","nmpa-approved"],"related":[],"cancers":["rcc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["vegf"],"drugs":[],"companies":["betta"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07047001","nct04373369","nct06523049"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo China deep dive","editedOn":"2026-09-16"},"code":"X-82","modality":"Oral multi-target VEGFR and PDGFR tyrosine kinase inhibitor","mechanism":"Blocks the VEGF and PDGF receptors that build and stabilise tumour blood vessels; designed as a shorter-acting relative of sunitinib to limit off-target toxicity.","approvals":[{"region":"CN","year":2023,"indication":"Advanced renal cell carcinoma after a prior tyrosine kinase inhibitor, with everolimus (June 2023)"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vrn110755","kind":"drug","name":"VRN110755","aka":[],"tldr":"VRN110755 is an experimental small-molecule drug from Voronoi in phase 2 trials for non-small-cell lung cancer, aimed at EGFR.","summary":"VRN110755 is a small-molecule drug developed by Voronoi. Its target is EGFR (the sponsor names EGFR). The sponsor states: A highly selective oral EGFR inhibitor designed to target activating EGFR mutations and selected resistance mutations, including C797S, in EGFR-mutant NSCLC. ClinicalTrials.gov describes the intervention as: VRN110755 is an investigational highly selective oral EGFR inhibitor supplied as capsules for oral administration. The drug is designed to target activating EGFR mutations and selected resistance mutations, including C797S, in patients with. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in non-small-cell lung cancer. The largest, NCT07699328, plans to enrol 315 participants with primary completion expected 2029-01-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of VRN110755","url":"https://clinicaltrials.gov/search?intr=VRN110755"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["egfr"],"drugs":[],"companies":[],"institutions":[],"pathways":[],"terms":[],"trials":["nct07699328"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"A highly selective oral EGFR inhibitor designed to target activating EGFR mutations and selected resistance mutations, including C797S, in EGFR-mutant NSCLC.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vs-7375","kind":"drug","name":"VS-7375","aka":[],"tldr":"VS-7375 is an experimental small-molecule drug from Verastem in phase 2 trials for pancreatic ductal adenocarcinoma, non-small-cell lung cancer and colorectal cancer, aimed at KRAS.","summary":"VS-7375 is a small-molecule drug developed by Verastem. Its target is KRAS (the sponsor names KRAS G12D). The sponsor states: An oral KRAS G12D (ON/OFF) inhibitor. ClinicalTrials.gov describes the intervention as: VS-7375 is a highly selective oral, non-covalent, small molecule KRAS G12D (ON/OFF) inhibitor. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in pancreatic ductal adenocarcinoma, non-small-cell lung cancer and colorectal cancer. The largest, NCT07020221, plans to enrol 295 participants with primary completion expected 2028-12. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of VS-7375","url":"https://clinicaltrials.gov/search?intr=VS-7375"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic","nsclc","colorectal"],"sections":[],"technologies":[],"targets":["kras"],"drugs":[],"companies":["verastem"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07659782","nct07659795","nct07644559","nct07020221"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"An oral KRAS G12D (ON/OFF) inhibitor.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vudalimab","kind":"drug","name":"Vudalimab","aka":[],"tldr":"Vudalimab is a bispecific antibody from Xencor, Inc., in registered phase 2 trials for ovarian cancer, endometrial cancer, cervical cancer.","summary":"Vudalimab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Xencor, Inc., in ovarian cancer, endometrial cancer, cervical cancer, prostate cancer. A bispecific antibody, as described in the registry record: one molecule binds two different targets, most often a tumour antigen and an immune cell or a second tumour pathway. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Vudalimab","url":"https://clinicaltrials.gov/search?intr=Vudalimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["ovarian","endometrial","cervical","prostate"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["xencor"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05032040"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Bispecific antibody","mechanism":"A bispecific antibody, as described in the registry record: one molecule binds two different targets, most often a tumour antigen and an immune cell or a second tumour pathway.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"vusolimogene-oderparepvec","kind":"drug","name":"Vusolimogene oderparepvec","aka":[],"tldr":"Vusolimogene oderparepvec is an engineered herpes virus injected into melanoma tumours, approved in August 2026 with nivolumab after immunotherapy failure.","summary":"Vusolimogene oderparepvec (RP1) is an engineered herpes simplex virus type 1 that replicates selectively in tumour cells, expresses GM-CSF to recruit antigen-presenting cells and carries the GALV-GP R- fusogenic protein. It is injected into melanoma lesions at 1 x 10^6 PFU/mL for the first dose, then 1 x 10^7 PFU/mL every 2 weeks for up to 8 cycles, with nivolumab. In IGNYTE the combination produced a response rate of about 33% in melanoma that had failed anti-PD-1 therapy, including shrinkage of uninjected lesions, evidence of a systemic immune effect. The FDA issued a complete response letter in July 2025 and then granted accelerated approval on 6 August 2026; the randomised IGNYTE-3 trial must confirm the benefit. Replimune developed it. For a newcomer: a tumour-injected virus that helps immunotherapy work again in melanoma.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Vusolimogene%20oderparepvec"}],"tags":[],"related":[],"cancers":["melanoma","advanced-melanoma"],"sections":[],"technologies":["oncolytic-virus"],"targets":[],"drugs":["nivolumab"],"companies":["replimune","bms"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06264180","nct06898970"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Tudriqev","code":"RP1","modality":"Oncolytic virus (HSV-1)","mechanism":"Selective replication in tumour cells, cell fusion and lysis, GM-CSF-driven antigen presentation.","approvals":[{"region":"US","year":2026,"indication":"Unresectable/metastatic melanoma after anti-PD-1, with nivolumab (accelerated)"}],"mechanismSteps":["Engineered HSV-1 is injected into a tumour","Virus replicates selectively in tumour cells (defective antiviral responses)","Cells lyse and fuse (GALV-GP R- fusogen), releasing antigens","Encoded GM-CSF recruits dendritic cells; interferon response follows","Nivolumab sustains the systemic T-cell response against uninjected lesions"],"dosing":{"route":"Intratumoural injection","schedule":"1 × 10⁶ PFU/mL first dose, then 1 × 10⁷ PFU/mL every 2 weeks for up to 8 cycles (injectable lesions up to 10 mL), with nivolumab","monitoring":"Injection-site reactions, flu-like symptoms, herpetic infection precautions","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Fatigue"},{"event":"Chills"},{"event":"Pyrexia"},{"event":"Nausea"},{"event":"Influenza-like illness"},{"event":"Injection-site pain"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-11","type":"designation","region":"US","note":"Breakthrough Therapy designation, anti-PD-1-failed melanoma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-07-22","type":"crl","region":"US","note":"Complete response letter citing trial design and heterogeneity","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-08-06","type":"accelerated-approval","region":"US","note":"Accelerated approval with nivolumab for unresectable/metastatic melanoma after anti-PD-1 (IGNYTE) The confirmatory requirement was still open 0.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"In combination with nivolumab for treatment of adult patients with unresectable advanced cutaneous melanoma who experienced disease progression with a programmed death receptor-1 (PD-1)-blocking antibody-based regimen"}]},{"id":"wjb001","kind":"drug","name":"WJB001","aka":["Wee1 inhibitor+ Taxol/Abraxane","Wee1 inhibitor+ PARAPLATIN/Aqupla","Wee1 inhibitor+ PARAPLATIN+Taxol","Wee1 inhibitor+ Zejula"],"tldr":"WJB001 is a small-molecule inhibitor from Wigen Biomedicine Technology (Shanghai) Co., Ltd., in registered phase 2 trials for metastatic cancer.","summary":"WJB001 is listed on ClinicalTrials.gov as an intervention in 8 registered phase 2 trials sponsored by Wigen Biomedicine Technology (Shanghai) Co., Ltd., in metastatic cancer. Described in the registry record as a wee1 inhibitor; the trial entries below carry the sponsor's wording. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of WJB001","url":"https://clinicaltrials.gov/search?intr=WJB001"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["metastatic-cancer"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["wigen-biomedicine-technology-shanghai"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05773820","nct06953323","nct06953323","nct06953323","nct06953323","nct06953323","nct06953323","nct06953323"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"code":"WJB001","modality":"Small-molecule inhibitor","mechanism":"Described in the registry record as a wee1 inhibitor; the trial entries below carry the sponsor's wording.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"xaluritamig","kind":"drug","name":"Xaluritamig","aka":[],"tldr":"Xaluritamig is Amgen's T-cell engager for prostate cancer, now in two phase 3 trials, aiming to do for prostate cancer what tarlatamab did for small-cell lung cancer.","summary":"Phase 1 (ESMO 2024): PSA50 ~49% and confirmed responses in heavily pretreated mCRPC at high doses; CRS mostly grade 1-2 with step-up dosing. XALute (NCT06691984, n≈675, post-taxane, vs cabazitaxel or ARPI, OS primary; active, not recruiting) and XALience (with abiraterone, chemo-naive mCRPC).","status":"phase-3","asOf":"2026-09-06","links":[{"label":"ClinicalTrials.gov NCT06691984","url":"https://clinicaltrials.gov/study/NCT06691984"},{"label":"XALience trial in progress (ASCO 2026)","url":"https://ascopubs.org/doi/10.1200/JCO.2026.44.16_suppl.TPS5144"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":["t-cell-engager"],"targets":["steap1","cd3"],"drugs":[],"companies":["amgen"],"institutions":[],"pathways":[],"terms":["crs","psa50"],"trials":["xalute","nct07213674"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"AMG 509","modality":"Bispecific T-cell engager (STEAP1×CD3, XmAb 2+1)","mechanism":"Two STEAP1-binding domains and one CD3 arm (avidity for high-STEAP1 cells) redirect T cells.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"xevinapant","kind":"drug","name":"Xevinapant","aka":["Debio 1143","AT-406","SM-406"],"tldr":"Xevinapant was a tablet meant to make head and neck cancer cells easier to kill with chemoradiation by removing the proteins that stop them dying. A promising mid-stage trial was followed by a phase 3 that was stopped in 2024 because it was not working.","summary":"Xevinapant (Debio 1143) is an oral antagonist of the inhibitor-of-apoptosis proteins developed by Debiopharm and licensed to Merck KGaA in 2021. In a randomised phase 2 trial in locally advanced squamous cell carcinoma of the head and neck, adding it to cisplatin-based chemoradiation improved locoregional control and, on longer follow-up, survival, which made it the most anticipated radiosensitiser in the disease.\n\nThe confirmatory phase 3 TrilynX trial in unresected locally advanced disease was stopped for futility in 2024 after an interim analysis found it was unlikely to improve event-free survival, and the companion trial XRay Vision in resected high-risk disease was also halted; Merck KGaA returned the rights. The HPV-negative head and neck cancer page records it among the treatments that failed to improve chemoradiation.","status":"negative","asOf":"2026-09-17","wikipedia":"https://en.wikipedia.org/wiki/Xevinapant","links":[{"label":"Wikipedia","url":"https://en.wikipedia.org/wiki/Xevinapant"}],"tags":["subtype-drugs-wave"],"related":["cisplatin"],"cancers":["hpv-negative-head-and-neck-cancer"],"sections":[],"technologies":["radiosensitisers"],"targets":[],"drugs":[],"companies":["debiopharm","merck-kgaa"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"Debio 1143","modality":"Oral IAP antagonist (SMAC mimetic) radiosensitiser","mechanism":"Mimics the SMAC protein to block inhibitor-of-apoptosis proteins (XIAP, cIAP1 and cIAP2), restoring the cancer cell's ability to die after the DNA damage of radiotherapy and cisplatin.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"xnw27011","kind":"drug","name":"XNW27011","aka":[],"tldr":"XNW27011 is an experimental antibody-drug conjugate from Astellas Pharma Global Development in phase 2 trials for pancreatic ductal adenocarcinoma, non-small-cell lung cancer and ovarian cancer, aimed at Claudin 18.2.","summary":"XNW27011 is an antibody-drug conjugate developed by Astellas Pharma Global Development. Its target is Claudin 18.2 (the sponsor names Claudin 18.2 (CLDN18.2)). The sponsor states: XNW27011 is an antibody-drug conjugate targeting Claudin 18.2, a tight-junction transmembrane protein, carrying the topoisomerase I inhibitor payload YL0010014, given intravenously every three weeks. ClinicalTrials.gov describes the intervention as: Eligible patient(s) in each dose cohort will receive the assigned XNW27011 dose administration every 3 weeks (Q3W, cycle) until intolerable toxicity, progression of the disease without clinical benefit, or withdrawal of informed consent. An. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, in pancreatic ductal adenocarcinoma, non-small-cell lung cancer, ovarian cancer, colorectal cancer and biliary tract cancer. The largest, NCT06792435, plans to enrol 240 participants with primary completion expected 2026-12-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of XNW27011","url":"https://clinicaltrials.gov/search?intr=XNW27011"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["pancreatic","nsclc","ovarian","colorectal","cholangiocarcinoma","gastric"],"sections":[],"technologies":[],"targets":["cldn18-2"],"drugs":[],"companies":["astellas"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06792435"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","payload":"YL0010014 (topoisomerase I inhibitor), as stated in the registry record; DAR not stated","mechanism":"XNW27011 is an antibody-drug conjugate targeting Claudin 18.2, a tight-junction transmembrane protein, carrying the topoisomerase I inhibitor payload YL0010014, given intravenously every three weeks.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"xnw5004","kind":"drug","name":"XNW5004","aka":["EZH2i"],"tldr":"XNW5004 is an experimental small-molecule drug from Evopoint Biosciences in phase 2 trials for follicular lymphoma and prostate cancer, aimed at EZH2.","summary":"XNW5004 is a small-molecule drug developed by Evopoint Biosciences. Its target is EZH2. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in follicular lymphoma and prostate cancer. The largest, NCT06702995, plans to enrol 307 participants with primary completion was scheduled for 2026-05-13 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of XNW5004","url":"https://clinicaltrials.gov/search?intr=XNW5004"},{"label":"Sponsor page","url":"https://www.evopointbio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["follicular-lymphoma","prostate"],"sections":[],"technologies":[],"targets":["ezh2"],"drugs":[],"companies":["evopoint-biosciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07545603","nct07730515","nct06702995","nct06776952"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule (structure undisclosed or not yet in PubChem)","mechanism":"Small-molecule drug directed at EZH2, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"xzp-3287","kind":"drug","name":"XZP-3287","aka":[],"tldr":"XZP-3287 is an experimental small-molecule drug from Sihuan Pharmaceutical in phase 3 trials for HR-positive / HER2-negative breast cancer, with its target not yet stated publicly.","summary":"XZP-3287 is a small-molecule drug developed by Sihuan Pharmaceutical and Xuanzhu Biopharmaceutical. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: XZP-3287 360 mg orally Twice daily(Q12H) of every 28-day cycle Fulvestrant 500mg intramuscular injection on day 1 and day 15 for the first cycle and then on day 1 for every cycle (28-day) until progressive disease. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT05077449 (A Study of XZP-3287 in Combination With Fulvestrant in Patients With Advanced Breast Cancer), in HR-positive / HER2-negative breast cancer. The largest, NCT04539496, plans to enrol 402 participants (actual) with primary completion was scheduled for 2023-07-31 on the registry. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of XZP-3287","url":"https://clinicaltrials.gov/search?intr=XZP-3287"},{"label":"Sponsor page","url":"https://www.xuanzhubio.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["xuanzhu-biopharmaceutical"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05077449","nct04539496","nct05257395"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"yl201","kind":"drug","name":"YL201","aka":["Tam-Peli","Tambotatug Pelitecan"],"tldr":"YL201 is an experimental antibody-drug conjugate from MediLink Therapeutics (Suzhou) in phase 3 trials for nasopharyngeal carcinoma, non-small-cell lung cancer and small-cell lung cancer, aimed at B7-H3.","summary":"YL201 is an antibody-drug conjugate developed by MediLink Therapeutics (Suzhou). Its target is B7-H3 (the sponsor names B7-H3). ClinicalTrials.gov describes the intervention as: Patients will be treated with YL201 intravenous (IV) infusion once every 3 weeks on Day 1 or twice every 3 weeks on Day 1 and Day 8 during a 3-week (21-day) cycle. It is the investigational product in 9 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06629597 (A Phase III Study of YL201 in Recurrent or Metastatic Nasopharyngeal Carcinoma(TAISHAN-301)), NCT06612151 (A Phase III Study of YL201 in Relapsed Small Cell Lung Cancer（TAISHAN-302）) and NCT07739758 (A Study of YL201 in Combination With Serplulimab in Participants With Treatment-naïve Extensive-stage Small Cell Lung Cancer), in nasopharyngeal carcinoma, non-small-cell lung cancer, small-cell lung cancer and prostate cancer. The largest, NCT06057922, plans to enrol 990 participants with primary completion expected 2026-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of YL201","url":"https://clinicaltrials.gov/search?intr=YL201"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nasopharyngeal","nsclc","sclc","prostate"],"sections":[],"technologies":[],"targets":["b7h3"],"drugs":[],"companies":["medilink"],"institutions":[],"pathways":[],"terms":[],"trials":["taishan-301","nct06612151","nct07739758","nct07258979","nct07208773","nct06241846","nct05434234","nct07407933","nct06057922"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"Antibody-drug conjugate directed at B7-H3, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"yl202","kind":"drug","name":"YL202","aka":[],"tldr":"YL202 is an experimental antibody-drug conjugate (ADC) from MediLink Therapeutics (Suzhou) in phase 3 trials for non-small-cell lung cancer, HR-positive / HER2-negative breast cancer and colorectal cancer, aimed at HER3.","summary":"YL202 is an antibody-drug conjugate (ADC) developed by MediLink Therapeutics (Suzhou). Its target is HER3 (the sponsor names HER3). ClinicalTrials.gov describes the intervention as: YL202 will be intravenously infused over 60±10 min. It is the investigational product in 4 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT07416994 (Pivotal Study to Evaluate YL202 Versus Docetaxel in Patients With Locally Advanced or Metastatic EGFR Sensitive Mutation Non-Squamous Non-Small Cell Lung Cancer) and NCT07461454 (YL202 Versus Treatment of Physician's Choice in Patients With HR+/HER2- Breast Cancer), in non-small-cell lung cancer, HR-positive / HER2-negative breast cancer, colorectal cancer and cervical cancer. The largest, NCT07416994, plans to enrol 440 participants with primary completion expected 2028-08-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of YL202","url":"https://clinicaltrials.gov/search?intr=YL202"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","breast-hr-positive","colorectal","cervical"],"sections":[],"technologies":[],"targets":["her3"],"drugs":[],"companies":["medilink"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07416994","nct07461454","nct07169994","nct07202364","nct06107686","nct06439771"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"antibody-drug conjugate (ADC)","mechanism":"Antibody-drug conjugate (ADC) directed at HER3, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zalifrelimab","kind":"drug","name":"Zalifrelimab","aka":[],"tldr":"Zalifrelimab is a monoclonal antibody from Nelum Corp, in registered phase 2 trials for pancreatic ductal adenocarcinoma.","summary":"Zalifrelimab is listed on ClinicalTrials.gov as an intervention in 1 registered phase 2 trial sponsored by Nelum Corp and ImmunoGenesis, in pancreatic ductal adenocarcinoma. A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below. OnCo records the agent from the registry alone; approvals, results and the sponsor's own description will follow as they are published.","status":"phase-2","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Zalifrelimab","url":"https://clinicaltrials.gov/search?intr=Zalifrelimab"}],"tags":["pipeline","ctgov-ingest","registry-only"],"related":[],"cancers":["pancreatic"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["agenus"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04827953","nct06782555"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, registry-only)","editedOn":"2026-09-16"},"modality":"Monoclonal antibody","mechanism":"A monoclonal antibody: the international non-proprietary name stem -mab marks an antibody that binds one target. The registry record does not state the target unless named in the trial entries below.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zamtocabtagene-autoleucel","kind":"drug","name":"Zamtocabtagene autoleucel","aka":["zamtocabtagene autoleucel (MB-CART2019.1)"],"tldr":"Zamtocabtagene autoleucel is an experimental CAR-T cell therapy from Miltenyi Biomedicine in phase 2 trials for diffuse large B-cell lymphoma, primary CNS lymphoma and mantle cell lymphoma, aimed at CD19 and CD20.","summary":"Zamtocabtagene autoleucel (CART2019) is a CAR-T cell therapy developed by Miltenyi Biomedicine. Its targets are CD19 and CD20 (the sponsor names CD19-CD20). The sponsor states: tandem CAR-T approach targeting two B-cell antigens. ClinicalTrials.gov describes the intervention as: Tandem CD20-CD19-directed non-cryopreserved CAR-T cell therapy. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, in diffuse large B-cell lymphoma, primary CNS lymphoma and mantle cell lymphoma. The largest, NCT04792489, plans to enrol 315 participants with primary completion expected 2026-12-31. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-2","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Zamtocabtagene autoleucel","url":"https://clinicaltrials.gov/search?intr=CART2019"},{"label":"Sponsor page","url":"https://www.miltenyibiomedicine.com"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["dlbcl","primary-cns-lymphoma","mantle-cell-lymphoma"],"sections":[],"technologies":[],"targets":["cd19","cd20"],"drugs":[],"companies":["miltenyi-biomedicine"],"institutions":[],"pathways":[],"terms":[],"trials":["nct04792489","nct07288879","nct04844866","nct06508931"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CART2019","modality":"CAR-T cell therapy","mechanism":"tandem CAR-T approach targeting two B-cell antigens.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zanidatamab","kind":"drug","name":"Zanidatamab","aka":[],"tldr":"Zanidatamab (Ziihera) is an antibody that grabs HER2 at two different spots, approved for HER2+ bile duct cancer.","summary":"Accelerated approval November 2024 for HER2+ biliary tract cancer (HERIZON-BTC-01). HERIZON-GEA-01 phase 3 in first-line HER2+ gastro-oesophageal cancer with chemotherapy ± tislelizumab was positive (2025). Jazz (ex-Asia) and BeOne. Biparatopic binding drives receptor clustering and internalisation, the basis of the ADC zanidatamab zovodotin.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Zanidatamab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Zanidatamab"}],"tags":[],"related":["her2-ihc-3-plus"],"cancers":["cholangiocarcinoma","gastric","gallbladder","biliary-tract-cancer"],"sections":[],"technologies":["bispecific-antibody"],"targets":["her2"],"drugs":[],"companies":["jazz","zymeworks","beone"],"institutions":[],"pathways":[],"terms":["her2-testing-in-biliary-cancer"],"trials":["nct06435429","nct07102381","nct06695845"],"people":[],"bottlenecks":[],"keyPapers":["paper-harding-lancet-oncol","paper-angerilli-her2-ihc-cish-biliary-hum-pathol-2026"],"journals":[],"dependsOn":[],"notes":["Gallbladder cancer was the largest anatomical subgroup in HERIZON-BTC-01, which required ERBB2 amplification by central ISH and reported a 41.3% confirmed response rate in the 80 IHC 2+ or 3+ patients (Harding 2023); the FDA label restricts use to IHC 3+ on the Ventana PATHWAY 4B5 assay."],"brand":"Ziihera","modality":"Biparatopic bispecific antibody (HER2)","mechanism":"Binds ECD2 and ECD4 of HER2 simultaneously; clustering, internalisation, ADCC.","approvals":[{"region":"US","year":2024,"indication":"HER2+ (IHC 3+) unresectable/metastatic biliary tract cancer, previously treated","note":"Ziihera label (openFDA); 53% of HERIZON-BTC-01 patients had gallbladder cancer: https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22ZIIHERA%22"},{"region":"EU","year":2025,"indication":"HER2+ biliary tract after ≥1 line; 27 Jun 2025 (conditional)","note":"Conditional marketing authorisation"},{"region":"UK","year":2026,"indication":"HER2-positive (IHC 3+) unresectable locally advanced or metastatic biliary tract cancer after at least one line of systemic treatment; MHRA 19 February 2026, NICE TA1153 published 7 May 2026 with a commercial arrangement","note":"https://www.nice.org.uk/guidance/ta1153"}],"mechanismSteps":["Two arms bind two different HER2 epitopes (ECD2 and ECD4) on one or neighbouring receptors","HER2 receptors cluster and are internalised and degraded","HER2 signalling and dimerisation with HER3 stop","Fc engages immune effectors (ADCC, phagocytosis)","HER2-dependent tumour cells die"],"dosing":{"route":"IV infusion","schedule":"20 mg/kg every 2 weeks","modifications":"Hold for LVEF decline or grade 3 diarrhoea","monitoring":"LVEF at baseline and every 3 months; infusion reactions; diarrhoea","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Diarrhoea"},{"event":"Infusion-related reactions"},{"event":"Abdominal pain"},{"event":"Fatigue"},{"event":"Nausea"},{"event":"Left ventricular dysfunction"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2023-01","type":"designation","region":"US","note":"Breakthrough Therapy designation, HER2+ biliary tract cancer","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-11-20","type":"accelerated-approval","region":"US","note":"Accelerated approval, previously treated HER2+ (IHC3+) biliary tract cancer (HERIZON-BTC-01) The confirmatory requirement was still open 1.8 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test"},{"date":"2025-12","type":"filing","region":"US","note":"First-line HER2+ gastro-oesophageal adenocarcinoma (HERIZON-GEA-01) submission","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-08-25","type":"approval","region":"US","note":"First-line HER2-positive gastric, gastro-oesophageal junction or oesophageal adenocarcinoma with tislelizumab and fluoropyrimidine-platinum chemotherapy","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-zanidatamab-hrii-and-tislelizumab-jsgr-her2-positive-gastric-gastroesophageal-junction"}]},{"id":"zanidatamab-zovodotin","kind":"drug","name":"Zanidatamab zovodotin","aka":["ZW49"],"tldr":"Zanidatamab zovodotin joined the two-armed HER2 antibody zanidatamab to a cell-killing payload. It was tested in early trials in HER2-expressing cancers; the plain antibody went on to approval, the conjugate did not progress beyond phase 1.","summary":"Zanidatamab zovodotin (ZW49) was Zymeworks' antibody-drug conjugate built on zanidatamab, the biparatopic HER2 antibody now approved as Ziihera. Binding two HER2 epitopes was intended to make the receptor cluster and internalise faster, delivering more of the auristatin payload into HER2-expressing tumour cells, including those with lower HER2 expression.\n\nIt was given intravenously in a phase 1 dose-escalation study across HER2-expressing solid tumours, including breast and gastro-oesophageal cancers. The programme was not advanced into registrational trials; it is named on the HER2-positive breast cancer page as one of the next-generation and biparatopic HER2 conjugates that followed trastuzumab deruxtecan.","status":"phase-1","asOf":"2026-09-17","links":[{"label":"Zimmerman and Esteva, Cancers 2024: next-generation HER2-targeted antibody-drug conjugates (table of payloads and linkers)","url":"https://doi.org/10.3390/cancers16040800"},{"label":"ClinicalTrials.gov NCT03821233 (phase 1)","url":"https://clinicaltrials.gov/study/NCT03821233"}],"tags":["subtype-drugs-wave"],"related":["zanidatamab","trastuzumab-deruxtecan"],"cancers":["breast-her2-positive","gastric","gallbladder"],"sections":[],"technologies":["adc"],"targets":["her2"],"drugs":[],"companies":["zymeworks"],"institutions":[],"pathways":[],"terms":[],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["Payload and linker read from the ADC table in Zimmerman and Esteva, Cancers 2024 (zovodotin, cleavable vc linker, DAR 2 to 4 variable); Niquille et al., MAbs 2024, describe the payload as a cytotoxic tubulin inhibitor delivered by the biparatopic antibody. Listed on the gallbladder page as the conjugate built on zanidatamab; the phase 1 study (NCT03821233) enrolled HER2-expressing solid tumours and the programme did not advance."],"code":"ZW49","modality":"Biparatopic HER2 antibody-drug conjugate","payload":"Zovodotin (auristatin-class tubulin inhibitor), variable DAR (2 to 4)","linker":"Cleavable valine-citrulline (vc) linker","mechanism":"The biparatopic antibody zanidatamab, which binds two different HER2 domains at once, carried an auristatin payload; the dual binding clusters HER2 and drives the conjugate into the cell where the payload is released.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zanubrutinib","kind":"drug","name":"Zanubrutinib","aka":[],"tldr":"Zanubrutinib is the only BTK blocker to beat ibrutinib on both efficacy and safety in a head-to-head trial. It is now the most prescribed BTK inhibitor in CLL.","summary":"ALPINE (relapsed CLL): PFS superior to ibrutinib (HR 0.65) with less atrial fibrillation; SEQUOIA (frontline): PFS superior to bendamustine-rituximab, 5-year PFS 72% in del(17p); SEQUOIA arm D (zanubrutinib + venetoclax) uMRD ~60%. Approved in CLL January 2023. Also MCL, Waldenström, MZL, follicular lymphoma. BeOne is testing zanubrutinib + sonrotoclax (CELESTIAL-TNCLL) and the BTK degrader BGB-16673 as successors.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Zanubrutinib","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Zanubrutinib"}],"tags":[],"related":[],"cancers":["cll","dlbcl","marginal-zone-lymphoma","cll-treatment-naive"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["btk"],"drugs":[],"companies":["beone"],"institutions":[],"pathways":[],"terms":[],"trials":["alpine","sequoia","celestial-tncll","nct04002297","nct06742996","nct05100862","nct06697184","nct07277231","nct06634589","nct04170283","nct06375733","nct04662255","nct06637501","nct05952037"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Brukinsa","modality":"Small-molecule covalent BTK inhibitor (second generation)","mechanism":"Covalent BTK inhibitor with sustained near-complete BTK occupancy in lymph nodes and fewer off-target kinases than ibrutinib.","approvals":[{"region":"US","year":2019,"indication":"Relapsed MCL (accelerated)"},{"region":"US","year":2023,"indication":"CLL/SLL, all lines (SEQUOIA, ALPINE)"}],"mechanismSteps":["Twice-daily dosing keeps BTK occupancy >95% in nodes throughout the day","BCR signalling and CXCR4-mediated homing are blocked","CLL cells exit protective niches and die","Lower EGFR/ITK inhibition means fewer cardiac and bleeding events"],"dosing":{"route":"Oral","schedule":"160 mg twice daily or 320 mg once daily, continuously","monitoring":"Infections, cytopenias, bleeding, atrial fibrillation (lower than ibrutinib), hypertension","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Brukinsa"},"toxicity":[{"event":"Atrial fibrillation/flutter","anyGradePct":5.2,"source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=Brukinsa","note":"ALPINE vs 13.3% ibrutinib"},{"event":"Neutropenia","grade3PlusPct":16},{"event":"Hypertension","anyGradePct":23},{"event":"Upper respiratory infection","anyGradePct":27}],"access":[],"regulatoryEvents":[{"date":"2019-11-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 6.8 years later, when the FDA's table was read.","indication":"Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy."},{"date":"2021-09-14","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 5.0 years later, when the FDA's table was read.","indication":"Treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen"},{"date":"2023-01-19","type":"approval","region":"US","note":"CLL/SLL","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2024-03-07","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 2.5 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-zanubrutinib-relapsed-or-refractory-follicular-lymphoma","indication":"Adult patients with relapsed or refractory follicular lymphoma (FL), in combination with obinutuzumab, after two or more lines of systemic therapy"},{"date":"2025-06-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.3 years later, when the FDA's table was read.","indication":"Formulation: Treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy."},{"date":"2025-06-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.3 years later, when the FDA's table was read.","indication":"Formulation: Treatment of adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen."},{"date":"2025-06-10","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.3 years later, when the FDA's table was read.","indication":"Formulation: Treatment of adult patients with relapsed or refractory follicular lymphoma (FL), in combination with obinutuzumab, after two or more lines of systemic therapy."}]},{"id":"zanzalintinib","kind":"drug","name":"Zanzalintinib","aka":[],"tldr":"Zanzalintinib is an experimental small-molecule drug from Exelixis in phase 3 trials for neuroendocrine tumours, head and neck squamous cell carcinoma and renal cell carcinoma, aimed at VEGF / VEGFR and MET.","summary":"Zanzalintinib (XL092) is a small-molecule drug developed by Exelixis and Merck Sharp & Dohme. Its targets are VEGF / VEGFR and MET (the sponsor names MET/VEGFR/AXL/MER). The sponsor states: Exelixis describes zanzalintinib as a next-generation tyrosine kinase inhibitor (TKI) targeting MET, VEGFR, AXL and MER receptors. It is the investigational product in 6 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT06943755 (Zanzalintinib Versus Everolimus in Participants With Locally Advanced or Metastatic Neuroendocrine Tumours), NCT06082167 (Study of Zanzalintinib (XL092) + Pembrolizumab vs Pembrolizumab in Subjects With PD-L1 Positive Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma) and NCT07227402 (A Clinical Study of Belzutifan and Zanzalintinib in People With Recurrent Kidney Cancer Following Adjuvant Therapy (MK-6482-033)), in neuroendocrine tumours, head and neck squamous cell carcinoma and renal cell carcinoma. The largest, NCT07227402, plans to enrol 904 participants with primary completion expected 2032-02-27. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Zanzalintinib","url":"https://clinicaltrials.gov/search?intr=XL092"},{"label":"Sponsor pipeline page","url":"https://www.exelixis.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["neuroendocrine","head-and-neck","rcc"],"sections":[],"technologies":[],"targets":["vegf","met"],"drugs":[],"companies":["exelixis","merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06943755","nct06082167","nct07227402","nct07489495","nct05425940","nct07049926"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"XL092","modality":"small molecule","mechanism":"Exelixis describes zanzalintinib as a next-generation tyrosine kinase inhibitor (TKI) targeting MET, VEGFR, AXL and MER receptors.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zelenectide-pevedotin","kind":"drug","name":"Zelenectide pevedotin","aka":[],"tldr":"A tiny peptide instead of an antibody carries the same payload as Padcev to Nectin-4, with far less rash and neuropathy in early data, but its developer stepped back in 2026.","summary":"Duravelo-2 (randomised phase 2/3): encouraging responses and a differentiated safety profile (skin reactions ~4-fold lower, neuropathy half that of enfortumab vedotin), presented ASCO 2026. Bicycle Therapeutics deprioritised the programme in March 2026 after regulators indicated the trial design could not support registration.","status":"phase-2","asOf":"2026-09-07","links":[{"label":"ClinicalTrials.gov: trials of Zelenectide pevedotin","url":"https://clinicaltrials.gov/search?intr=BT8009"}],"tags":["lesson:trial-design-registration"],"related":[],"cancers":["urothelial"],"sections":[],"technologies":["peptide-drug-conjugate"],"targets":["nectin4"],"drugs":[],"companies":["bicycle-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06933329","nct06840483","nct06225596","nct04561362"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"BT8009","modality":"Bicycle toxin conjugate (Nectin-4, MMAE)","mechanism":"Bicyclic peptide binds Nectin-4; cleavable linker releases MMAE; rapid renal clearance limits systemic exposure.","approvals":[],"mechanismSteps":["Bicyclic peptide binds Nectin-4 with high affinity","Short plasma half-life; tumour penetration within hours","Cleavable linker releases MMAE inside the tumour","Bystander killing with less skin and nerve exposure than antibody ADCs"],"dosing":{"route":"Intravenous","schedule":"5 mg/m² weekly, 3 weeks on / 1 off (optimised dose), alone or with pembrolizumab"},"toxicity":[{"event":"Fatigue","anyGradePct":40},{"event":"Skin reactions","anyGradePct":13,"note":"Roughly one quarter the rate reported for enfortumab vedotin"},{"event":"Peripheral neuropathy","anyGradePct":25}],"access":[],"regulatoryEvents":[{"date":"2026-03","type":"withdrawal","region":"US","note":"Programme deprioritised after regulatory feedback on Duravelo-2 design","source":"https://www.oncologypipeline.com/apexonco/bicycle-finally-falls"}]},{"id":"zen-3694","kind":"drug","name":"ZEN-3694","aka":["ZEN003694","Zenith BET inhibitor"],"tldr":"ZEN-3694 is a BET inhibitor from Zenith Epigenetics being tested in NUT carcinoma, where the cancer's driving fusion is itself a BET protein, and in prostate and breast cancers.","summary":"BET inhibitors block the bromodomain proteins that switch on growth genes such as MYC. NUT carcinoma is the clearest use: its BRD4-NUT fusion is a BET protein, and inhibitors make the cells differentiate in the laboratory, with objective but short-lived responses in early trials of molibresib and birabresib. ZEN-3694 is the BET inhibitor now carrying that idea forward in National Cancer Institute trials with platinum chemotherapy and with abemaciclib in NUT carcinoma, and it has been studied with enzalutamide in castration-resistant prostate cancer and with talazoparib in triple-negative breast cancer. Thrombocytopenia and fatigue are the class side effects.","status":"phase-2","asOf":"2026-09-08","wikipedia":"https://en.wikipedia.org/wiki/BET_inhibitor","links":[{"label":"NCI trial with platinum chemotherapy, NCT05019716","url":"https://clinicaltrials.gov/study/NCT05019716"},{"label":"NCI trial with abemaciclib, NCT05372640","url":"https://clinicaltrials.gov/study/NCT05372640"}],"tags":["gap-fill","owner-request"],"related":[],"cancers":["nut-carcinoma","prostate","tnbc"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["brd4"],"drugs":[],"companies":["zenith-epigenetics"],"institutions":[],"pathways":[],"terms":[],"trials":["zen-3694-platinum-nut","zen-3694-abemaciclib-nut","nct04986423"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"modality":"Oral small-molecule BET bromodomain inhibitor","mechanism":"Binds the bromodomains of BRD2, BRD3 and BRD4 so they cannot read acetylated histones; in NUT carcinoma this displaces the BRD4-NUT fusion from chromatin and lets the cells differentiate, and in other cancers it lowers MYC and androgen receptor signalling.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zenocutuzumab","kind":"drug","name":"Zenocutuzumab","aka":[],"tldr":"Zenocutuzumab is the first drug for cancers driven by NRG1 gene fusions, working by blocking HER3 from receiving its growth signal.","summary":"Zenocutuzumab is a HER2xHER3 bispecific antibody that docks on HER2 and physically blocks neuregulin 1 (NRG1) from binding HER3, the 'dock and block' mechanism that shuts off the growth signal in tumours driven by NRG1 gene fusions. It received accelerated approval in December 2024 for NRG1-fusion NSCLC and pancreatic cancer on the eNRGy trial, and in Q2 2026 the label was expanded to NRG1-fusion cholangiocarcinoma. It is given intravenously at 750 mg every 2 weeks; diarrhoea, musculoskeletal pain, fatigue and infusion reactions are the main adverse events. NRG1 fusions are rare and easily missed by DNA-only panels, so RNA-based sequencing is needed to find eligible patients, and confirmatory data are still required. Merus developed it and Partner Therapeutics commercialises it. For a newcomer: the first drug for a rare fusion that can appear in several different cancers.","status":"approved","asOf":"2026-09-04","wikipedia":"https://en.wikipedia.org/wiki/Zenocutuzumab","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Zenocutuzumab"},{"label":"Bizengri label (openFDA): pancreatic indication and eNRGy study section","url":"https://api.fda.gov/drug/label.json?search=openfda.brand_name:%22BIZENGRI%22"},{"label":"FDA notice: accelerated approval of zenocutuzumab (4 December 2024)","url":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"label":"Schram et al., efficacy of zenocutuzumab in NRG1 fusion-positive cancer (NEJM 2025)","url":"https://doi.org/10.1056/NEJMoa2405008"}],"tags":[],"related":["nrg1-fusion"],"cancers":["nsclc","pancreatic","kras-wild-type-pdac","cholangiocarcinoma","metastatic-pdac"],"sections":[],"technologies":["bispecific-antibody"],"targets":["her2","her3"],"drugs":[],"companies":["merus"],"institutions":[],"pathways":[],"terms":[],"trials":["nct02912949"],"people":[],"bottlenecks":[],"keyPapers":["paper-enrgy-zenocutuzumab-nrg1-fusion-positive-cancer-nejm-2025","paper-heining-nrg1-fusions-kras-wild-type-pancreatic-cancer-discov-2018","paper-jones-nrg1-fusions-recurrent-actionable-kras-wild-type-pdac-ccr-2019"],"journals":[],"dependsOn":[],"notes":["Pancreatic cancer, regulators: the Bizengri label (effective 19 May 2026) indicates zenocutuzumab 750 mg every two weeks for advanced unresectable or metastatic pancreatic adenocarcinoma with an NRG1 gene fusion after prior systemic therapy, under accelerated approval granted on 4 December 2024 on the eNRGy study (30 pancreatic patients in the label cohort; 15 of 36 responses, 42 percent, in the NEJM 2025 report). The EMA product page did not exist on 24 September 2026 (the corpus records a marketing authorisation application under evaluation) and NICE had no appraisal, so the drug is available in Europe only through trials or named-patient routes. NRG1 fusions are found almost only in KRAS wild-type tumours and need RNA-based sequencing.","Pancreatic ductal adenocarcinoma biomarkers: eligibility is an NRG1 fusion, found in 0.3 to 1% of pancreatic cancers and 1.3% of KRAS wild-type tumours, and detected reliably only by RNA-based or intron-covering sequencing; eNRGy gave responses in 15 of 36 pancreatic patients, 42% (Schram 2025)."],"brand":"Bizengri","modality":"Bispecific antibody (HER2×HER3)","mechanism":"Docks on HER2 and blocks NRG1 binding to HER3 ('dock and block').","approvals":[{"region":"US","year":2024,"indication":"NRG1-fusion NSCLC and pancreatic adenocarcinoma"},{"region":"US","year":2026,"indication":"NRG1-fusion cholangiocarcinoma"}],"mechanismSteps":["One arm docks on HER2","The other arm blocks the NRG1-binding site on HER3","NRG1 fusion protein cannot activate HER2/HER3 heterodimers","PI3K/AKT signalling collapses in NRG1-fusion tumours","Tumour regresses"],"dosing":{"route":"IV infusion","schedule":"750 mg every 2 weeks","modifications":"Hold for infusion reactions and LVEF decline","monitoring":"LVEF; infusion reactions; embryo-fetal toxicity","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Diarrhoea"},{"event":"Musculoskeletal pain"},{"event":"Fatigue"},{"event":"Nausea"},{"event":"Infusion-related reactions"},{"event":"Dyspnoea"}],"access":[{"country":"US","reimbursement":"Medicare Part B (physician-administered); commercial plans per formulary","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-12-04","type":"accelerated-approval","region":"US","note":"Accelerated approval, NRG1-fusion NSCLC and pancreatic adenocarcinoma (eNRGy): first therapy for NRG1 fusions The confirmatory requirement was still open 1.8 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adults with advanced unresectable or metastatic pancreatic adenocarcinoma harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy."},{"date":"2024-12-04","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 1.8 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-zenocutuzumab-zbco-non-small-cell-lung-cancer-and-pancreatic","indication":"Treatment of adults with advanced unresectable or metastatic nonsmall cell lung cancer (NSCLC) harboring a neuregulin 1 (NRG1) gene fusion with disease progression on or after prior systemic therapy."},{"date":"2026-Q2","type":"approval","region":"US","note":"NRG1-fusion cholangiocarcinoma","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"}]},{"id":"zevorcabtagene-autoleucel","kind":"drug","name":"Zevorcabtagene autoleucel","aka":[],"tldr":"Zevor-cel is CARsgen's myeloma CAR-T, approved in China in 2024 for patients whose disease has come back after three or more treatments.","summary":"Approved by the NMPA in February 2024 for relapsed or refractory multiple myeloma after three or more lines including a proteasome inhibitor and an immunomodulatory drug, on the LUMMICAR STUDY 1 phase 1/2 trial run in China. CARsgen reported high response rates with deep remissions and mostly low-grade cytokine release syndrome; the full phase 2 report appeared in Experimental Hematology and Oncology in 2025. Commercialised in China with Huadong Medicine. CARsgen's second approval, satricabtagene autoleucel for Claudin 18.2-positive gastric cancer, followed in 2025.","status":"approved","asOf":"2026-09-10","links":[{"label":"CARsgen Therapeutics","url":"https://www.carsgen.com/en/"},{"label":"Phase 2 study of zevorcabtagene autoleucel (Exp Hematol Oncol 2025)","url":"https://doi.org/10.1186/s40164-025-00710-y"},{"label":"Zevorcabtagene autoleucel: first approval (Mol Diagn Ther 2024)","url":"https://doi.org/10.1007/s40291-024-00723-z"}],"tags":["china"],"related":[],"cancers":["multiple-myeloma"],"sections":[],"technologies":["car-t"],"targets":["bcma"],"drugs":[],"companies":["carsgen"],"institutions":[],"pathways":[],"terms":[],"trials":["lummicar-1","nct03915184"],"people":[],"bottlenecks":[],"keyPapers":["paper-chen-exp-hematol-oncol","paper-dhillon-mol-diagn-ther"],"journals":[],"dependsOn":[],"notes":[],"brand":"Saikaize","code":"zevor-cel, CT053","modality":"Cell therapy (autologous BCMA CAR-T, fully human binder)","mechanism":"Autologous T cells transduced with a fully human anti-BCMA CAR (4-1BB co-stimulation) developed by CARsgen from its Shanghai platform.","approvals":[{"region":"China","year":2024,"indication":"Relapsed or refractory multiple myeloma after three or more lines including a proteasome inhibitor and an immunomodulatory agent"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zg006","kind":"drug","name":"ZG006","aka":[],"tldr":"ZG006 is an experimental investigational agent whose form is not stated in the registry from Suzhou Zelgen Biopharmaceuticals in phase 3 trials for non-small-cell lung cancer and neuroendocrine tumours, with its target not yet stated publicly.","summary":"ZG006 is an investigational agent whose form is not stated in the registry developed by Suzhou Zelgen Biopharmaceuticals. Its molecular target is not stated in the ClinicalTrials.gov intervention record, and OnCo has not confirmed one from the sponsor's pipeline page. ClinicalTrials.gov describes the intervention as: ZG006 will be administered as an intravenous (IV) infusion. It is the investigational product in 6 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07189455 (Study Comparing ZG006 With Investigator-Selected Chemotherapy in Participants With Relapsed Small Cell Lung Cancer), in non-small-cell lung cancer and neuroendocrine tumours. The largest, NCT07189455, plans to enrol 420 participants with primary completion expected 2028-10. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ZG006","url":"https://clinicaltrials.gov/search?intr=ZG006"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","neuroendocrine"],"sections":[],"technologies":[],"targets":[],"drugs":[],"companies":["zelgen"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07189455","nct05978284","nct06283719","nct07038096","nct07258121","nct06440057"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"trispecific T-cell engager antibody (DLL3 x DLL3 x CD3, per the sponsor's pipeline page; form not stated in the registry record)","mechanism":"Mechanism not stated in the ClinicalTrials.gov record or on the sponsor's pipeline page.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zibotentan","kind":"drug","name":"Zibotentan","aka":["ZD4054"],"tldr":"A drug blocking a signal that prostate cancer uses to grow in bone. Three trials in prostate cancer found no survival benefit and it was dropped.","summary":"Endothelin-1 and the endothelin A receptor are implicated in the growth of prostate cancer in bone, and for most of the 2000s the endothelin axis was one of the most attractive non-hormonal targets in the disease. Zibotentan is a specific endothelin A receptor antagonist.\n\nThe ENTHUSE programme tested it in three settings. In ENTHUSE M1, 594 men with castration-resistant prostate cancer, bone metastases and no or mild pain were randomised 1:1 to zibotentan 10 mg daily or placebo with standard treatment. Median overall survival was 24.5 months with zibotentan against 22.5 with placebo, hazard ratio 0.87 (95.2 percent confidence interval 0.69 to 1.10, p=0.240), not significant, and no secondary endpoint reached significance either. Peripheral oedema (44 percent) and headache (31 percent) were the commonest adverse events, and cardiac failure of any grade occurred in 5.7 percent against 1.7 percent.\n\nThe other two ENTHUSE trials, in combination with docetaxel and in non-metastatic disease, were also negative, and AstraZeneca discontinued zibotentan in prostate cancer. Atrasentan, the other endothelin A antagonist, failed in SWOG S0421 in the same years. Both drugs have since been revived for kidney disease, where the target behaves differently.","status":"withdrawn","asOf":"2026-09-25","links":[{"label":"ENTHUSE M1 (Cancer 2012)","url":"https://doi.org/10.1002/cncr.27674"}],"tags":[],"related":[],"cancers":["prostate","prostate-mcrpc"],"sections":[],"technologies":[],"targets":["ednra"],"drugs":[],"companies":["astrazeneca"],"institutions":[],"pathways":[],"terms":["bone-metastases","castration-resistance"],"trials":["enthuse-m1-zibotentan","swog-s0421-atrasentan"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"ZD4054","modality":"small molecule","mechanism":"Specific endothelin A receptor antagonist, intended to block endothelin-1 signalling in bone metastases.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zidesamtinib","kind":"drug","name":"Zidesamtinib","aka":[],"tldr":"A ROS1 inhibitor approved in July 2026 that works after other ROS1 drugs fail and avoids their brain side effects.","summary":"Zidesamtinib is a ROS1-selective tyrosine kinase inhibitor engineered to spare TRK and penetrate the brain, so it avoids the dizziness and weight gain caused by the TRK activity of repotrectinib and entrectinib while covering the G2032R resistance mutation. It is Nuvalent's first approved drug, cleared in July 2026 for ROS1-positive NSCLC on the ARROS-1 trial, and it is taken once daily. Oedema, fatigue, constipation and raised ALT are the main adverse events reported. It is intended both for patients whose disease has progressed on earlier ROS1 inhibitors and as a better-tolerated option, though its first-line position against repotrectinib and the durability of response are still being defined. Nuvalent's ALK inhibitor neladalkib follows the same selective design. For a newcomer: a ROS1 pill built to work after others fail without the brain side effects.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Zidesamtinib"}],"tags":[],"related":[],"cancers":["nsclc","ros1-positive-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":[],"drugs":[],"companies":["nuvalent"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05118789"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Jideytro","modality":"Small-molecule kinase inhibitor (ROS1)","mechanism":"TRK-sparing, brain-penetrant ROS1-selective TKI.","approvals":[{"region":"US","year":2026,"indication":"ROS1+ NSCLC (July 2026)"}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of ROS1 fusions including G2032R","Phosphorylation of downstream substrates stops","TRK-sparing design avoids dizziness and weight gain","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"Once daily (label dose per July 2026 approval)","monitoring":"LFTs, oedema, neuro-cognitive symptoms (minimised by TRK sparing)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Oedema"},{"event":"Fatigue"},{"event":"Constipation"},{"event":"ALT increased"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-06","type":"designation","region":"US","note":"Breakthrough Therapy designation, ROS1+ NSCLC after prior ROS1 TKI","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-07-22","type":"approval","region":"US","note":"ROS1+ NSCLC after at least one prior ROS1 TKI (ARROS-1)","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-zidesamtinib-ros1-positive-non-small-cell-lung-cancer"}]},{"id":"ziftomenib","kind":"drug","name":"Ziftomenib","aka":[],"tldr":"Ziftomenib is the second menin inhibitor for leukaemia, approved in November 2025 as a once-daily pill for relapsed NPM1-mutated AML.","summary":"KOMET-001 (n=112 relapsed/refractory NPM1-mutated AML): CR 23%, ORR 33%, median OS 6.6 months. Approved 13 November 2025 (Kura Oncology / Kyowa Kirin). KOMET-007 combines ziftomenib with 7+3 and with venetoclax-azacitidine in newly diagnosed NPM1-mutated and KMT2A-rearranged AML, with high remission rates in early cohorts; KOMET-017 phase 3 registration studies are underway. Menin inhibition also being tested in KMT2Ar ALL.","status":"approved","asOf":"2026-09-07","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ziftomenib"}],"tags":[],"related":["revumenib","npm1-mutation"],"cancers":["aml","all-leukemia","aml-npm1-kmt2a"],"sections":[],"technologies":["epigenetic-drugs"],"targets":["menin","npm1","kmt2a"],"drugs":[],"companies":["kura-oncology"],"institutions":[],"pathways":[],"terms":["differentiation-syndrome"],"trials":["komet-001","nct07007312","nct07623616"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Komzifti","modality":"Small-molecule menin inhibitor","mechanism":"Displaces KMT2A/KMT2A-fusion complexes from menin, silencing HOXA9/MEIS1 and releasing the differentiation block in NPM1-mutant and KMT2A-rearranged blasts.","approvals":[{"region":"US","year":2025,"indication":"Relapsed/refractory NPM1-mutated AML with no satisfactory alternative"}],"mechanismSteps":["Menin scaffolds KMT2A (or its fusion) onto chromatin at HOX loci","Ziftomenib occupies the menin pocket and evicts the complex","HOXA9 and MEIS1 transcription falls within days","Blasts differentiate (differentiation syndrome risk) and the leukaemic clone shrinks","MEN1 resistance mutations at the binding site can emerge after months"],"dosing":{"route":"Oral","schedule":"600 mg once daily until progression","monitoring":"Differentiation syndrome (boxed warning), QT, counts","source":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Ziftomenib"},"toxicity":[{"event":"Differentiation syndrome","source":"https://ascopost.com/news/november-2025/fda-approves-ziftomenib-for-npm1-positive-aml/","note":"Boxed warning; mitigated by early steroids and hydroxyurea"},{"event":"Nausea, diarrhoea","note":"Common, mostly low grade"},{"event":"Cytopenias","note":"Common in relapsed AML"}],"access":[],"regulatoryEvents":[{"date":"2025-11-13","type":"approval","region":"US","note":"KOMET-001; priority review","source":"https://ascopost.com/news/november-2025/fda-approves-ziftomenib-for-npm1-positive-aml/"}]},{"id":"zilovertamab-vedotin","kind":"drug","name":"Zilovertamab vedotin","aka":[],"tldr":"Zilovertamab vedotin is a ROR1-directed ADC in phase 3 for large B-cell lymphoma.","summary":"Zilovertamab vedotin is an anti-ROR1 antibody carrying the microtubule inhibitor MMAE through a cleavable linker; ROR1 is an oncofetal receptor re-expressed on B-cell cancers including large B-cell lymphoma and largely absent from normal adult tissue, which makes it an attractive ADC address. Merck acquired the programme through VelosBio and is developing it chiefly in large B-cell lymphoma. The waveLINE-003 phase 3 trial combines it at 1.75 mg/kg every 3 weeks with rituximab, gemcitabine and oxaliplatin (R-GemOx) in relapsed DLBCL, and it is also being tested in first line. Neutropenia, peripheral neuropathy and fatigue are the expected MMAE-class toxicities. Whether ROR1 targeting adds to existing CD19- and CD20-directed options, including CAR-T and bispecifics, is the question phase 3 must answer. It is an ADC against a fetal-type receptor that lymphoma cells switch back on.","status":"phase-3","asOf":"2026-09-04","links":[{"label":"ClinicalTrials.gov: trials of Zilovertamab vedotin","url":"https://clinicaltrials.gov/search?intr=MK-2140"}],"tags":[],"related":[],"cancers":["dlbcl"],"sections":[],"technologies":["adc"],"targets":["ror1"],"drugs":[],"companies":["merck"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06717347","nct05458297","nct06395103","nct05562830","nct05406401","nct06890884"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"MK-2140","modality":"ADC","payload":"MMAE","linker":"Cleavable","mechanism":"Anti-ROR1 with MMAE.","approvals":[],"mechanismSteps":["Antibody binds ROR1 on the tumour cell surface","Receptor-ADC complex is internalised by endocytosis","Trafficked to the lysosome; linker is cleaved or antibody degraded","MMAE is released inside the cell","Microtubule assembly is blocked; mitosis arrests","Tumour cell dies by apoptosis","Membrane-permeable payload diffuses to kill neighbouring antigen-negative cells (bystander effect)"],"dosing":{"route":"IV infusion","schedule":"Phase 3 dose 1.75 mg/kg every 3 weeks with R-GemOx","source":"https://clinicaltrials.gov/study/NCT05139017"},"toxicity":[{"event":"Neutropenia"},{"event":"Peripheral neuropathy"},{"event":"Fatigue"}],"access":[{"country":"US","reimbursement":"Investigational","asOf":"2026-09-06"}],"regulatoryEvents":[]},{"id":"zimberelimab","kind":"drug","name":"Zimberelimab","aka":["AB122"],"tldr":"Zimberelimab is a PD-1 antibody approved in China in 2021 for relapsed classical Hodgkin lymphoma, and the checkpoint partner in Arcus and Gilead's Western trials of the TIGIT antibody domvanalimab.","summary":"Guangzhou Gloria Biosciences' zimberelimab (GLS-010) received NMPA approval in 2021 for relapsed or refractory classical Hodgkin lymphoma after at least two lines of therapy, with a later cervical cancer indication. Outside China, Arcus Biosciences licensed it as AB122 and uses it as the PD-1 backbone with domvanalimab in the STAR-121 and STAR-221 phase 3 trials in lung and gastro-oesophageal cancer. A phase 3 trial in cervical cancer is registered in China.","status":"approved","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Zimberelimab","url":"https://clinicaltrials.gov/search?intr=GLS-010"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["hodgkin-lymphoma","cervical"],"sections":[],"technologies":["checkpoint-inhibitor"],"targets":["pd1"],"drugs":[],"companies":["arcus-biosciences"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05798819","nct05633667","nct05676931","nct04736173","nct06727565","nct06155396","nct05909436","nct07710885","nct03547973","nct05329766"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"GLS-010","modality":"Anti-PD-1 monoclonal antibody, intravenous","mechanism":"Blocks PD-1 on T cells so tumour cells cannot switch them off through PD-L1.","approvals":[{"region":"CN","year":2021,"indication":"Relapsed or refractory classical Hodgkin lymphoma after two or more lines of therapy"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zipalertinib","kind":"drug","name":"Zipalertinib","aka":[],"tldr":"Zipalertinib is an experimental small-molecule drug from Taiho Oncology in phase 3 trials for non-small-cell lung cancer, aimed at EGFR.","summary":"Zipalertinib (CLN-081, TAS6417) is a small-molecule drug developed by Taiho Oncology. Its target is EGFR (the sponsor names EGFR exon 20 insertion (ex20ins) mutations). It is the investigational product in 3 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including phase 3 studies NCT05973773 (REZILIENT3 (REsearching ZIpaLertinib In Egfr Non-small Cell Lung Cancer Tumours)) and NCT07128199 (A Study to Assess Zipalertinib Versus Placebo in Participants With Early Stage NSCLC With Uncommon EGFR Mutations, Following Complete Tumour Resection), in non-small-cell lung cancer. The largest, NCT07128199, plans to enrol 360 participants with primary completion expected 2029-10-01. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Zipalertinib","url":"https://clinicaltrials.gov/search?intr=CLN-081"},{"label":"Sponsor pipeline page","url":"https://www.taihooncology.com/us/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc","egfr-mutant-nsclc"],"sections":[],"technologies":[],"targets":["egfr"],"drugs":[],"companies":["taiho-oncology"],"institutions":[],"pathways":[],"terms":[],"trials":["nct05973773","nct07128199","nct04036682"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"code":"CLN-081, TAS6417","modality":"small molecule","mechanism":"Small-molecule drug directed at EGFR, as stated in the ClinicalTrials.gov intervention record; the detailed mechanism is not stated.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"girentuximab-zr89","kind":"drug","name":"Zirconium-89 girentuximab","aka":["89Zr-DFO-girentuximab","Zircaix"],"tldr":"Zirconium-89 girentuximab is Telix's PET scan that tells whether a kidney mass is clear cell renal cell carcinoma without a biopsy; the phase 3 ZIRCON trial met its endpoints in 2023 and the product is under regulatory review.","summary":"Telix Pharmaceuticals' TLX250-CDx labels the anti-CAIX antibody girentuximab with zirconium-89. In the ZIRCON phase 3 trial of 300 patients with indeterminate renal masses, the scan identified clear cell renal cell carcinoma with high sensitivity and specificity against surgical histology. Telix filed with the FDA, which issued a refusal-to-file letter in 2024 over manufacturing information, and a resubmission followed. A further phase 3 trial is registered.","status":"phase-3","asOf":"2026-09-16","links":[{"label":"ClinicalTrials.gov: trials of Zirconium-89 girentuximab","url":"https://clinicaltrials.gov/search?intr=TLX250-CDx"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["rcc"],"sections":[],"technologies":["immuno-pet","pet"],"targets":[],"drugs":[],"companies":["telix"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06750419"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2, curated)","editedOn":"2026-09-16"},"code":"TLX250-CDx","modality":"PET imaging antibody labelled with zirconium-89","mechanism":"Girentuximab binds carbonic anhydrase IX, expressed by nearly all clear cell renal cell carcinomas but not by normal kidney; the zirconium-89 label shows the tumour on PET days after injection.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"ziv-aflibercept","kind":"drug","name":"Ziv-aflibercept","aka":["Aflibercept (oncology formulation)","VEGF Trap"],"tldr":"Ziv-aflibercept (Zaltrap) is a decoy receptor that soaks up the blood-vessel growth signal VEGF. It is added to the FOLFIRI chemotherapy combination for bowel cancer that has spread and progressed after oxaliplatin.","summary":"Ziv-aflibercept was approved by the FDA in August 2012 with FOLFIRI for metastatic colorectal cancer resistant to or progressing after an oxaliplatin-containing regimen, on the VELOUR trial (1,226 patients) in which adding the drug modestly improved overall survival and progression-free survival over FOLFIRI plus placebo, including in patients who had received bevacizumab. The EU authorised Zaltrap in 2013. Its role overlaps with bevacizumab continued beyond progression and with ramucirumab (RAISE), and cost has limited uptake. Haemorrhage, gastrointestinal perforation and compromised wound healing carry boxed warnings; hypertension and proteinuria are common. The same molecule is the ophthalmic drug Eylea.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Aflibercept","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=ziv-aflibercept"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/ziv-aflibercept"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/zaltrap"},{"label":"NICE TA307 (not recommended)","url":"https://www.nice.org.uk/guidance/ta307"}],"tags":["nci-list"],"related":["bevacizumab","ramucirumab","folfiri"],"cancers":["colorectal"],"sections":[],"technologies":["antiangiogenic"],"targets":["vegf"],"drugs":[],"companies":["sanofi","regeneron"],"institutions":[],"pathways":[],"terms":[],"trials":["velour","nct03172299","nct00390234","nct01882868"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zaltrap","modality":"Recombinant fusion protein (VEGF ligand trap)","mechanism":"Fusion of VEGFR1 and VEGFR2 extracellular domains with human IgG1 Fc; binds VEGF-A, VEGF-B and placental growth factor with sub-picomolar affinity, preventing receptor activation and angiogenesis.","approvals":[{"region":"US","year":2012,"indication":"Metastatic colorectal cancer resistant to or progressing after oxaliplatin, with FOLFIRI"},{"region":"EU","year":2013,"indication":"Metastatic colorectal cancer resistant to or progressing after oxaliplatin, with FOLFIRI"},{"region":"US","year":2012,"indication":"Metastatic colorectal cancer resistant to or progressing after an oxaliplatin-containing regimen, with FOLFIRI","note":"Approved August 2012 on VELOUR."},{"region":"EU","year":2013,"indication":"Metastatic colorectal cancer resistant to or progressing after an oxaliplatin-containing regimen, with FOLFIRI","note":"Zaltrap marketing authorisation issued 1 February 2013."},{"region":"England (NICE)","year":2014,"indication":"Not recommended","note":"TA307, published 25 March 2014: aflibercept with irinotecan and fluorouracil-based therapy is not recommended within its marketing authorisation; patients already receiving it could continue."}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zl-1310","kind":"drug","name":"ZL-1310","aka":[],"tldr":"ZL-1310 is an experimental antibody-drug conjugate from Zai Lab (Shanghai) in phase 3 trials for non-small-cell lung cancer, aimed at DLL3.","summary":"ZL-1310 is an antibody-drug conjugate developed by Zai Lab (Shanghai). Its target is DLL3 (the sponsor names DLL3). The sponsor states: ZL-1310 is a DLL3-targeting antibody-drug conjugate (ADC), given as a single agent versus investigator's choice therapy (topotecan or amrubicin) in relapsed small cell lung cancer. It is the investigational product in 2 recruiting or active industry-led phase 2 and phase 3 interventional cancer trials, including the phase 3 study NCT07218146 (A Study of ZL-1310 Versus Investigator's Choice of Therapy in Participants With Relapsed Small Cell Lung Cancer (DLLEVATE)), in non-small-cell lung cancer. The largest, NCT07218146, plans to enrol 480 participants with primary completion expected 2028-06-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of ZL-1310","url":"https://clinicaltrials.gov/search?intr=ZL-1310"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["nsclc"],"sections":[],"technologies":[],"targets":["dll3"],"drugs":[],"companies":["zai-lab"],"institutions":[],"pathways":[],"terms":[],"trials":["dllevate","nct06885281"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":["23 Sept 2026: the payload is not stated on any record OnCo reads, so the open drug engine shows ZL-1310 under DLL3 with the payload class not recorded rather than guessing."],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"ADC","mechanism":"ZL-1310 is a DLL3-targeting antibody-drug conjugate (ADC), given as a single agent versus investigator's choice therapy (topotecan or amrubicin) in relapsed small cell lung cancer.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zolbetuximab","kind":"drug","name":"Zolbetuximab","aka":[],"tldr":"Zolbetuximab is the first drug against Claudin 18.2, a protein exposed on stomach cancer cells. Added to chemotherapy it extends survival by two to three months; nausea is the price.","summary":"Chimeric IgG1 binding CLDN18.2, killing via ADCC and CDC. SPOTLIGHT and GLOW positive; FDA approval 18 October 2024 with a companion IHC assay (VENTANA CLDN18). Eligible patients are CLDN18.2 ≥75% moderate-to-strong membranous staining, HER2-negative. Nausea and vomiting occur in most patients in cycle 1 and need aggressive antiemetic prophylaxis and infusion-rate adjustment.","status":"approved","asOf":"2026-09-07","wikipedia":"https://en.wikipedia.org/wiki/Zolbetuximab","links":[{"label":"FDA label","url":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761365s000lbl.pdf"}],"tags":[],"related":["cldn18-2-expression"],"cancers":["gastric","gastric-cldn18-2-positive","oesophageal-adenocarcinoma"],"sections":[],"technologies":["monoclonal-antibody","companion-diagnostic"],"targets":["cldn18-2"],"drugs":[],"companies":["astellas"],"institutions":[],"pathways":[],"terms":["adcc"],"trials":["spotlight-glow","nct06901531","nct03816163","nct03505320","nct07431281"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Vyloy","code":"IMAB362","modality":"Monoclonal antibody (anti-Claudin 18.2)","mechanism":"Binds CLDN18.2 exposed on malignant gastric cells; Fc-mediated ADCC and complement-dependent cytotoxicity.","approvals":[{"region":"Japan","year":2024,"indication":"CLDN18.2+ HER2- gastric cancer (first approval, March 2024)"},{"region":"US","year":2024,"indication":"First-line CLDN18.2+ (≥75%), HER2- locally advanced/metastatic gastric or GEJ adenocarcinoma with fluoropyrimidine/platinum chemotherapy"},{"region":"EU","year":2024,"indication":"1L CLDN18.2+ HER2- gastric/GEJ; 19 Sep 2024"}],"mechanismSteps":["Normal stomach cells hide Claudin 18.2 inside tight junctions; cancer cells expose it on their surface","Zolbetuximab binds the exposed protein","Its IgG1 tail recruits NK cells (ADCC) and complement (CDC)","The coated tumour cells are killed; some normal gastric mucosa is hit too, causing nausea"],"dosing":{"route":"Intravenous","schedule":"800 mg/m² loading, then 600 mg/m² every 3 weeks or 400 mg/m² every 2 weeks with chemotherapy","modifications":"Slow or interrupt infusion for nausea/vomiting; premedicate with antiemetics","monitoring":"Nausea/vomiting in cycle 1; hypersensitivity","source":"https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761365s000lbl.pdf"},"toxicity":[{"event":"Nausea","note":"Most patients; worst in cycle 1"},{"event":"Vomiting","note":"Most patients; infusion-related"},{"event":"Decreased appetite"},{"event":"Hypersensitivity/infusion reactions"}],"access":[],"regulatoryEvents":[{"date":"2024-03","type":"approval","region":"Japan","note":"World-first approval"},{"date":"2024-10-18","type":"approval","region":"US","note":"SPOTLIGHT/GLOW; companion diagnostic VENTANA CLDN18 (43-14A)"}]},{"id":"zoldonrasib","kind":"drug","name":"Zoldonrasib","aka":[],"tldr":"The first drug aimed specifically at KRAS G12D, the single most common mutation in pancreatic cancer. Early combination data in 2026 showed half of previously treated patients responding.","summary":"Revolution Medicines' covalent tri-complex inhibitor of KRAS G12D(ON). Breakthrough Therapy designation in G12D NSCLC (2025). At ESMO GI 2026, zoldonrasib plus daraxonrasib in previously treated RAS G12D metastatic PDAC (n=60, cutoff 9 Feb 2026): ORR 50% and DCR 97% in second line, median PFS 9.6 months; grade ≥3 treatment-related events 35% (rash, anaemia, stomatitis). First-line combination with gemcitabine/nab-paclitaxel showed high response rates and ctDNA clearance. Phase 3 planning underway.","status":"phase-2","asOf":"2026-09-06","links":[{"label":"Revolution Medicines, ESMO GI 2026","url":"https://ir.revmed.com/news-releases/news-release-details/revolution-medicines-presents-phase-12-clinical-data-zoldonrasib"},{"label":"OncLive report","url":"https://www.onclive.com/view/zoldonrasib-combinations-show-compelling-antitumor-activity-in-ras-g12d-mutant-metastatic-pdac"}],"tags":[],"related":[],"cancers":["pancreatic","metastatic-pdac","nsclc","colorectal"],"sections":[],"technologies":["kras-inhibitors"],"targets":["kras"],"drugs":["daraxonrasib"],"companies":["revolution-medicines"],"institutions":[],"pathways":["ras-mapk"],"terms":[],"trials":["nct07805954","nct07621718","nct07777822","nct06445062","nct07397338","nct06922591"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"code":"RMC-9805","modality":"Small-molecule RAS(ON) G12D-selective inhibitor","mechanism":"Cyclophilin-A-mediated tri-complex that covalently engages the G12D mutant aspartate and blocks effector binding in the active state.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zoledronic-acid","kind":"drug","name":"Zoledronic acid","aka":["Reclast / Aclasta (osteoporosis formulation)","Zoledronate"],"tldr":"Zoledronic acid (Zometa) is a fifteen-minute infusion given every few weeks or months to strengthen bone and prevent fractures, spinal cord compression and the need for radiotherapy in people with myeloma or cancer that has spread to bone; it also treats dangerously high calcium caused by cancer.","summary":"Zoledronic acid was approved by the FDA in August 2001 for hypercalcaemia of malignancy (two randomised trials against pamidronate showed faster and more complete normalisation of calcium) and in 2002 for multiple myeloma and bone metastases from solid tumours in conjunction with antineoplastic therapy, on trials in breast cancer, prostate cancer, lung and other solid tumours that reduced skeletal-related events. The EU authorised Zometa in 2001. Every-12-week dosing was shown non-inferior to monthly (CALGB 70604), and adjuvant zoledronic acid reduces recurrence and death in postmenopausal early breast cancer (EBCTCG meta-analysis), an off-label but guideline-endorsed use. Osteonecrosis of the jaw, renal impairment (dose adjustment and creatinine monitoring) and acute-phase reactions after the first dose are the main risks. Denosumab is the alternative when renal function is poor.","status":"approved","asOf":"2026-09-10","wikipedia":"https://en.wikipedia.org/wiki/Zoledronic_acid","links":[{"label":"US label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?labeltype=all&query=zoledronic%20acid"},{"label":"NCI drug page","url":"https://www.cancer.gov/about-cancer/treatment/drugs/zoledronicacid"},{"label":"EPAR","url":"https://www.ema.europa.eu/en/medicines/human/EPAR/zometa"}],"tags":["nci-list","generic","supportive"],"related":["denosumab","pamidronate"],"cancers":["multiple-myeloma","breast-hr-positive","prostate","nsclc","rcc"],"sections":[],"technologies":["bone-modifying-agents"],"targets":[],"drugs":[],"companies":["novartis"],"institutions":[],"pathways":[],"terms":["bone-metastases"],"trials":[],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Zometa","modality":"Nitrogen-containing bisphosphonate (intravenous)","supportive":true,"mechanism":"Binds bone mineral and inhibits farnesyl pyrophosphate synthase in osteoclasts, blocking prenylation of signalling proteins and inducing osteoclast apoptosis; reduces bone resorption and calcium release.","approvals":[{"region":"US","year":2001,"indication":"Hypercalcaemia of malignancy"},{"region":"US","year":2002,"indication":"Multiple myeloma and bone metastases from solid tumours, with antineoplastic therapy"},{"region":"EU","year":2001,"indication":"Prevention of skeletal-related events in advanced malignancies involving bone; tumour-induced hypercalcaemia"}],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]},{"id":"zongertinib","kind":"drug","name":"Zongertinib","aka":[],"tldr":"Zongertinib was the first oral HER2 inhibitor for lung cancer with HER2 mutations, approved in 2025 and moved to first line in 2026.","summary":"Zongertinib is an irreversible HER2-selective tyrosine kinase inhibitor that spares EGFR, which limits the rash and diarrhoea seen with pan-HER inhibitors; it is taken as 120 mg once daily. Beamion LUNG-1 showed a response rate of about 71% in previously treated HER2-mutant NSCLC, leading to accelerated approval in August 2025, and in Q1 2026 it was approved as first-line therapy for HER2-mutant non-squamous NSCLC, the first oral HER2 inhibitor for this setting. Boehringer Ingelheim developed it. Diarrhoea, rash, raised liver enzymes, nausea and fatigue are the main adverse events. It competes with trastuzumab deruxtecan and with sevabertinib (Bayer, approved late 2025), and the best sequence between a pill and an antibody-drug conjugate is unresolved. For a newcomer: the first pill for lung cancers driven by HER2 mutations.","status":"approved","asOf":"2026-09-04","links":[{"label":"FDA label (DailyMed)","url":"https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Zongertinib"}],"tags":[],"related":["her2-mutation"],"cancers":["nsclc","her2-mutant-nsclc"],"sections":[],"technologies":["kinase-inhibitors"],"targets":["her2"],"drugs":[],"companies":["boehringer-ingelheim"],"institutions":[],"pathways":[],"terms":[],"trials":["nct07195695","nct06324357","nct06581432","nct07486817","nct06151574"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"brand":"Hernexeos","modality":"Small-molecule kinase inhibitor (HER2)","mechanism":"Irreversible HER2-selective TKI sparing EGFR.","approvals":[{"region":"US","year":2025,"indication":"HER2-mutant NSCLC, previously treated (accelerated)"},{"region":"US","year":2026,"indication":"First-line HER2-mutant non-squamous NSCLC"}],"mechanismSteps":["Oral drug is absorbed and reaches the tumour","Binds the ATP pocket (or allosteric site) of mutant HER2 (exon 20 insertions and others)","Phosphorylation of downstream substrates stops","Irreversible, EGFR-sparing binding limits diarrhoea and rash","Oncogene-addicted cells arrest and undergo apoptosis"],"dosing":{"route":"Oral","schedule":"120 mg once daily","modifications":"Reduce for hepatotoxicity, diarrhoea","monitoring":"LFTs, LVEF, respiratory symptoms","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},"toxicity":[{"event":"Diarrhoea"},{"event":"Rash"},{"event":"ALT/AST increased"},{"event":"Nausea"},{"event":"Fatigue"}],"access":[{"country":"US","reimbursement":"Medicare Part D (oral); commercial plans per formulary, often with prior authorisation","source":"https://www.cms.gov/medicare/payment/part-b-drugs/asp-pricing-files","asOf":"2026-09-06"}],"regulatoryEvents":[{"date":"2024-03","type":"designation","region":"US","note":"Breakthrough Therapy designation","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2025-08-08","type":"accelerated-approval","region":"US","note":"Accelerated approval, previously treated HER2-mutant non-squamous NSCLC (Beamion LUNG-1) The confirmatory requirement was still open 1.1 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications","indication":"Treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations, as detected by an FDA approved test, and who have received prior systemic therapy"},{"date":"2026-Q1","type":"approval","region":"US","note":"First-line HER2-mutant non-squamous NSCLC","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications"},{"date":"2026-02-26","type":"accelerated-approval","region":"US","note":"Accelerated approval on a surrogate endpoint; the confirmatory requirement was still open 0.6 years later, when the FDA's table was read.","source":"https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-zongertinib-unresectable-or-metastatic-non-squamous-non-small-cell","indication":"Treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) tyrosine kinase domain activating mutations, as detected by an FDA-authorized test"}]},{"id":"zovegalisib","kind":"drug","name":"Zovegalisib","aka":[],"tldr":"Zovegalisib is an experimental small-molecule drug from Relay Therapeutics in phase 3 trials for HR-positive / HER2-negative breast cancer, aimed at PIK3CA / PI3K-alpha.","summary":"Zovegalisib is a small-molecule drug developed by Relay Therapeutics. Its target is PIK3CA / PI3K-alpha (the sponsor names PI3Kα (mutant-selective)). The sponsor states: Described as the first allosteric, pan-mutant (including kinase-domain mutations such as H1047X and helical-domain mutations such as E542X/E545X) and isoform-selective PI3Kα inhibitor, designed using cryo-EM structural analysis and molecular dynamics simulations to differentiate mutant from wild-type PI3Kα. ClinicalTrials.gov describes the intervention as: 400 mg orally BID administered daily on a 28-day treatment cycle. It is the investigational product in 1 recruiting or active industry-led phase 2 and phase 3 interventional cancer trial, including the phase 3 study NCT06982521 (Phase 3 Study of RLY-2608 + Fulvestrant vs Capivasertib + Fulvestrant as Treatment for Locally Advanced or Metastatic PIK3CA-mutant HR+/HER2- Breast Cancer), in HR-positive / HER2-negative breast cancer. The largest, NCT06982521, plans to enrol 540 participants with primary completion expected 2028-04-30. Status reflects the highest phase registered on ClinicalTrials.gov; no efficacy results are recorded here.","status":"phase-3","asOf":"2026-09-11","links":[{"label":"ClinicalTrials.gov: trials of Zovegalisib","url":"https://clinicaltrials.gov/search?intr=Zovegalisib"},{"label":"Sponsor pipeline page","url":"https://relaytx.com/pipeline/"}],"tags":["pipeline","ctgov-ingest"],"related":[],"cancers":["breast-hr-positive"],"sections":[],"technologies":[],"targets":["pik3ca"],"drugs":[],"companies":["relay-therapeutics"],"institutions":[],"pathways":[],"terms":[],"trials":["nct06982521"],"people":[],"bottlenecks":[],"keyPapers":[],"journals":[],"dependsOn":[],"notes":[],"provenance":{"editedBy":"OnCo ingestion (ClinicalTrials.gov v2 + sponsor pipeline pages)","editedOn":"2026-09-11"},"modality":"small molecule","mechanism":"Described as the first allosteric, pan-mutant (including kinase-domain mutations such as H1047X and helical-domain mutations such as E542X/E545X) and isoform-selective PI3Kα inhibitor, designed using cryo-EM structural analysis and molecular dynamics simulations to differentiate mutant from wild-type PI3Kα.","approvals":[],"mechanismSteps":[],"toxicity":[],"access":[],"regulatoryEvents":[]}]