Drugs / Imetelstat
last change Jun 2026 re-read 1 minute ago

Imetelstat

also known as Imetelstat Sodium · GRN163L

Oligonucleotide (other) targets TERT via inhibition

Developed for
myelofibrosis · myelodysplastic syndrome · plasma cell myeloma · lymphoma · lymphoproliferative syndrome · neuroblastoma · acquired polycythemia vera · acute myeloid leukemia
+26 more · anaplastic astrocytoma · anaplastic ependymoma · brainstem neoplasm · breast cancer · breast neoplasm · cancer · central nervous system neoplasm · childhood acute myeloid leukemia · childhood malignant neoplasm · childhood myelodysplastic syndrome · ependymoma · essential thrombocythemia · Ewing sarcoma/peripheral primitive neuroectodermal tumor · female breast carcinoma · giant cell glioblastoma · gliosarcoma · hepatoblastoma · juvenile myelomonocytic leukemia · lung cancer · myelodysplastic/myeloproliferative neoplasm · non-small cell lung carcinoma · oligodendroglioma · osteosarcoma · primary myelofibrosis · rhabdomyosarcoma · small intestine cancer
Investigated by
Geron Corporation · National Cancer Institute (NCI) · Children's Oncology Group · Australasian Leukaemia and Lymphoma Group · Breast Cancer Research Foundation · GCP-Service International West GmbH
+9 more · German Myelodysplastic Syndrome Study Group · Groupe Francophone des Myelodysplasies · Indiana University · M.D. Anderson Cancer Center · NCIC Clinical Trials Group · Pediatric Brain Tumor Consortium · QIMR Berghofer Medical Research Institute · Saint-Louis Hospital, Paris, France · Universitätsklinikum Leipzig

Regulatory milestones approvals, filings & regulatory actions · 2 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved EU (EMA) Rytelo 2025-03-07 europa.eu
Approved US (FDA) RYTELO 2024-06-06 fda.gov

Trials 25 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 112 trials
Phase 1 NCT06247787 Feb 2025 → Mar 2028 expected childhood acute myeloid leukemia, childhood myelodysplastic syndrome, juvenile myelomonocytic leukemia Children's Oncology Group Recruiting No outcome recorded
Phase 1 NCT05371964 May 2022 → Feb 2027 expected myelofibrosis Geron Corporation Recruiting No outcome recorded
Phase 1 NCT01916187 Jul → Nov 2013 neuroblastoma NCIC Clinical Trials Group Withdrawn No outcome recorded
Phase 1 NCT01568632 Mar → Oct 2012 cancer, lymphoma National Cancer Institute (NCI) Withdrawn No outcome recorded
Phase 1 NCT01273090 May 2011 → Sep 2013 central nervous system neoplasm, childhood malignant neoplasm, lymphoma, lymphoproliferative syndrome +1 Children's Oncology Group Completed No outcome recorded
Phase 1 NCT01265927 Jan 2011 → Aug 2012 breast neoplasm Indiana University Completed No outcome recorded
Phase 1 NCT00732056 Jul 2008 → Sep 2011 breast cancer Geron Corporation Completed No outcome recorded
Phase 1 NCT00718601 Jul 2008 → Oct 2011 plasma cell myeloma Geron Corporation Completed No outcome recorded
Phase 1 NCT00594126 Nov 2007 → Jul 2011 plasma cell myeloma Geron Corporation Completed No outcome recorded
Phase 1 NCT00510445 Jul 2007 → Apr 2011 lung cancer Geron Corporation Completed No outcome recorded
Phase 210 trials
Phase 2 NCT07549451 Oct 2026 → May 2027 expected myelodysplastic/myeloproliferative neoplasm M.D. Anderson Cancer Center Not yet recruiting No outcome recorded
Phase 2 NCT05583552 Jun 2023 → Mar 2025 overdue acute myeloid leukemia, myelodysplastic syndrome GCP-Service International West GmbH Active not recruiting No outcome recorded
Phase 2 NCT02426086 Aug 2015 → Apr 2018 myelofibrosis Geron Corporation Completed No outcome recorded
Phase 2 NCT02011126 Jun 2014 → Mar 2016 Ewing sarcoma/peripheral primitive neuroectodermal tumor, hepatoblastoma, neuroblastoma, osteosarcoma +1 Children's Oncology Group Withdrawn No outcome recorded
Phase 2 NCT01836549 Mar 2013 → Apr 2016 anaplastic astrocytoma, anaplastic ependymoma, brainstem neoplasm, ependymoma +3 Pediatric Brain Tumor Consortium Terminated No outcome recorded
Phase 2 NCT01731951 Oct 2012 → May 2018 myelofibrosis, primary myelofibrosis Geron Corporation Completed No outcome recorded
Phase 2 NCT01243073 Dec 2010 → Oct 2013 acquired polycythemia vera, essential thrombocythemia Geron Corporation Completed No outcome recorded
Phase 2 NCT01242930 Nov 2010 → Nov 2014 plasma cell myeloma Geron Corporation Completed No outcome recorded
Phase 2 NCT01256762 Nov 2010 → Oct 2012 female breast carcinoma Geron Corporation Completed No outcome recorded
Phase 2 NCT01137968 May 2010 → Sep 2013 non-small cell lung carcinoma Geron Corporation Completed No outcome recorded
Phase 2/31 trial
Phase 2/3 NCT02598661 Jan 2016 → Oct 2023 overdue myelodysplastic syndrome Geron Corporation Active not recruiting No outcome recorded
Phase 31 trial
Phase 3 NCT04576156 Apr 2021 → Jun 2028 expected myelofibrosis Geron Corporation Active not recruiting No outcome recorded
Phase not stated1 trial
NCT05937568 myelodysplastic syndrome Geron Corporation Approved for marketing No outcome recorded
Also used as a comparator or background therapy in 1 trial
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT07320235 Comparator Jan 2026 → Jun 2030 expected acute myeloid leukemia Douglas Tremblay Recruiting No outcome recorded

Evidence & citations 7 cited values

Every value below carries the sentence it was read from. 28 sources stand behind the page.

FieldValueCited text
Known as Imetelstat “Part A Safety Run-In phase: Imetelstat in combination with Azacitidine” NCT07320235
21

NCT06247787

NCT01916187

NCT02426086

NCT00310895

NCT02598661

NCT00594126

NCT01137968

NCT01836549

NCT04576156

NCT00510445

NCT05937568

NCT01568632

NCT02011126

NCT00718601

NCT01273090

NCT01243073

NCT07549451

NCT01242930

NCT01256762

NCT01731951

NCT05371964

Known as GRN163L “A Phase II Trial to Evaluate the Activity of Imetelstat (GRN163L) in Patients with Essential Thrombocythemia or Polycythemia Vera Who Require Cytoreduction and Have Failed or...” NCT01243073
7

“Imetelstat Sodium (GRN163L)” NCT00718601

“Imetelstat Sodium (GRN163L)” NCT00510445

“Imetelstat Sodium (GRN163L)” NCT00594126

“Imetelstat Sodium (GRN163L)” NCT00310895

NCT01265927

NCT00732056

NCT00124189

Known as Imetelstat Sodium Imetelstat Sodium (GRN163L) is a telomerase template antagonist with in vitro and in vivo activity in a variety of tumor model systems.” NCT00124189
8

“To evaluate the safety and tolerability of imetelstat (imetelstat sodium) in myelofibrosis (MF).” NCT01731951

Imetelstat sodium will be administered as intravenous (IV) every 28 days.” NCT05371964

Imetelstat sodium IV infusion.” NCT02598661

Imetelstat Sodium (GRN163L)” NCT00594126

Imetelstat Sodium (GRN163L)” NCT00310895

Imetelstat Sodium (GRN163L)” NCT00718601

“Drug: Imetelstat SodiumNCT02011126

NCT05583552

Action Inhibit Inhibition of telomerase may result in antineoplastic effects.” NCT00310895
Modality Oligonucleotide (other) “Imetelstat, a 13-mer oligonucleotide that is covalently modified with lipid extensions” PMID 26332546 Sep 2015
Route Intravenous “Weekly intravenous infusion” NCT00124189
Target TERT “Target inhibition was demonstrated by reduction of telomerase activity and human telomerase reverse transcriptase level” PMID 34138638 Jun 2021