# Asciminib

Source: https://onco.cc/drugs/asciminib/  
OnCo record `asciminib` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

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.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-07
- 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: US 2021: CML after ≥2 prior TKIs; T315I CML; US 2024: Newly diagnosed Ph+ CML in chronic phase (ASC4FIRST)
- Dosing: Oral; CML: 80 mg once daily or 40 mg twice daily; 200 mg twice daily for T315I
- Toxicity (grade 3+): Thrombocytopenia 17%

## Notes

- Approved indications are in chronic myeloid leukaemia; Ph+ ALL use is investigational.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Asciminib
- FDA label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/search.cfm?query=Asciminib

## Connected records

- biomarkers: [BCR::ABL1 T315I](https://onco.cc/biomarkers/bcr-abl1-t315i/), [BCR::ABL1 transcript (Philadelphia chromosome, quantitative PCR)](https://onco.cc/biomarkers/bcr-abl1-transcript/)
- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Chronic myeloid leukaemia (CML)](https://onco.cc/cancers/cml/), [Chronic myeloid leukaemia, accelerated and blast phase](https://onco.cc/cancers/cml-advanced-phase/), [Chronic myeloid leukaemia, chronic phase](https://onco.cc/cancers/cml-chronic-phase/), [Philadelphia chromosome-positive acute lymphoblastic leukaemia in children (Ph-positive ALL)](https://onco.cc/cancers/all-paediatric-ph-positive/)
- technologies: [Small-molecule kinase inhibitors](https://onco.cc/technologies/kinase-inhibitors/)
- targets: [ABL1](https://onco.cc/targets/abl1/), [BCR::ABL1 (Philadelphia chromosome)](https://onco.cc/targets/bcr-abl/)
- companies: [Novartis](https://onco.cc/companies/novartis/)
- terms: [BCR::ABL1 kinase domain mutations (T315I and others)](https://onco.cc/terms/abl1-kinase-domain-mutations/), [Molecular response (MMR, MR4, treatment-free remission)](https://onco.cc/terms/molecular-response/), [On-target resistance mutations (gatekeeper, solvent-front, compound)](https://onco.cc/terms/gatekeeper-mutation/), [Ph-positive ALL](https://onco.cc/terms/ph-positive-all/), [Philadelphia chromosome (Ph+, BCR::ABL1)](https://onco.cc/terms/philadelphia-chromosome/), [Sokal and ELTS risk scores (chronic myeloid leukaemia)](https://onco.cc/terms/sokal-elts-scores/)
- trials: [A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP](https://onco.cc/trials/nct04971226/), [A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)](https://onco.cc/trials/nct05456191/), [Asciminib Monotherapy, With Dose Escalation, for 2nd and 1st Line Chronic Myelogenous Leukemia](https://onco.cc/trials/nct05384587/), [Open-label Study of Asciminib for CML-CP or CML-AP Patients With T315I Mutation Who Are Resistant, Intolerant or Ineligible to Ponatinib.](https://onco.cc/trials/nct06514534/), [Safety and Efficacy of Asciminib in Pediatrics and Young Adults With Relapse/Refractory (r/r) Philadelphia Positive (Ph+) or ABL-class Ph-like Acute Lymphoblastic Leukemia (ALL)](https://onco.cc/trials/nct07387926/), [Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia](https://onco.cc/trials/nct04925479/), [Study to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP](https://onco.cc/trials/nct07354074/)
- pathways: [BCR::ABL1 (Philadelphia chromosome)](https://onco.cc/pathways/bcr-abl1-signalling/), [Chronic myeloid leukaemia (KEGG map)](https://onco.cc/pathways/cml-signalling/), [Resistance routes: how a blocked pathway comes back](https://onco.cc/pathways/resistance-routes-map/)
- key papers: [IRIS: imatinib versus interferon plus cytarabine as first treatment for chronic myeloid leukaemia](https://onco.cc/key-papers/paper-iris-imatinib-nejm-2003/)

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