{"entity":{"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":[]},"route":"/drugs/imetelstat/","neighbours":{"cancer":[{"id":"mds-lower-risk","kind":"cancer","name":"Lower-risk myelodysplastic syndromes","route":"/cancers/mds-lower-risk/"},{"id":"mds","kind":"cancer","name":"Myelodysplastic syndromes / neoplasms (MDS)","route":"/cancers/mds/"},{"id":"myeloproliferative-neoplasms","kind":"cancer","name":"Myeloproliferative neoplasms (PV, ET, myelofibrosis)","route":"/cancers/myeloproliferative-neoplasms/"},{"id":"primary-myelofibrosis","kind":"cancer","name":"Primary myelofibrosis","route":"/cancers/primary-myelofibrosis/"}],"technology":[{"id":"antisense-sirna","kind":"technology","name":"Oligonucleotide therapeutics","route":"/technologies/antisense-sirna/"},{"id":"transfusion-support","kind":"technology","name":"Transfusion support and anaemia management","route":"/technologies/transfusion-support/"}],"company":[{"id":"geron","kind":"company","name":"Geron Corporation","route":"/companies/geron/"}],"trial":[{"id":"imerge","kind":"trial","name":"IMerge","route":"/trials/imerge/"},{"id":"impactmf","kind":"trial","name":"IMpactMF","route":"/trials/impactmf/"},{"id":"nct05583552","kind":"trial","name":"Study to Evaluate Imetelstat in Patients With High-Risk MDS or AML Failing HMA-based Therapy","route":"/trials/nct05583552/"}],"paper":[{"id":"paper-commands-luspatercept-mds-lancet-2023","kind":"paper","name":"COMMANDS: luspatercept versus epoetin alfa as first treatment for anaemia in lower-risk MDS needing transfusions","route":"/key-papers/paper-commands-luspatercept-mds-lancet-2023/"},{"id":"paper-imerge-imetelstat-mds-lancet-2024","kind":"paper","name":"IMerge: imetelstat, a telomerase inhibitor, for transfusion-dependent lower-risk MDS after erythropoietin has failed","route":"/key-papers/paper-imerge-imetelstat-mds-lancet-2024/"}],"term":[{"id":"anaemia","kind":"term","name":"Anaemia","route":"/terms/anaemia/"},{"id":"hallmarks-synthesis","kind":"term","name":"Hallmarks of cancer as a synthesis of the theories","route":"/terms/hallmarks-synthesis/"},{"id":"ipss-m-ipss-r","kind":"term","name":"IPSS-R and IPSS-M (myelodysplastic syndrome risk scores)","route":"/terms/ipss-m-ipss-r/"},{"id":"sf3b1-mutation","kind":"term","name":"SF3B1 mutation","route":"/terms/sf3b1-mutation/"}]}}