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imetelstat

Generic: IMETELSTAT SODIUM·Brand: RYTELO, Rytelo

imetelstat: developed by 1 company · 6 clinical trials tracked · 1 FDA decision.

imetelstat evidence translation

What we can confirm

1 FDA record · 6 tracked trials

What it means

Source-backed records connect imetelstat to GERN; that link alone does not prove the drug works.

What to watch

Watch active trials and any change in FDA or trial status.
BioSniper AIPage evidence connected

Prepared research question

How could imetelstat → GERN affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence6 indications · 1 study sponsors

Related indications

Myelofibrosis; Multiple Myeloma; Myelodysplastic SyndromesAdult patients with low- to intermediate-1 risk myelodysplastic syndromes (lower-risk MDS) with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs)Acute Myeloid LeukemiaMultiple MyelomaMyelodysplastic SyndromesMyelofibrosis
Development Pipeline (2)
CompanyPhase
GERON CORP (GERN)
Myelofibrosis; Multiple Myeloma; Myelodysplastic Syndromes
Phase 3
GERON CORP (GERN)
Adult patients with low- to intermediate-1 risk myelodysplas…
Approved
Clinical Trials (6)

A Phase 1 Study of GRN163L (Imetelstat) in Combination With Fludarabine and Cytarabine for Patients With Acute Myeloid Leukemia That is in Second or Greater Relapse or That is Refractory to Relapse Therapy; Myelodysplastic Syndrome or Juvenile Myelomonocytic Leukemia in First or Greater Relapse or is Refractory to Relapse Therapy

NCT06247787·Phase 1
Recruiting

Primary endpoint: Dose limiting toxicities of imetelstat administered in combination with fludarabine and cytarabine

Interventionalopen36 targetStarted 2025-02Completion 2028-0319 sites · United StatesChildren's Oncology Group

A Phase II Study Evaluating the Efficacy and Safety of Imetelstat in Patients With HR Myelodysplastic Syndromes or AML Failing HMA-based Therapy

NCT05583552·Phase 2·HWH
Active, not recruiting

Primary endpoint: Overall Hematological Response Rate of Participants After Treatment With Imetelstat

Interventionalopen46 targetStarted 2023-06Completion 2026-0611 sites · Australia, France, GermanyGCP-Service International West GmbH

An Open Label, Phase 1/1b Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of Imetelstat in Combination With Ruxolitinib in Patients With Myelofibrosis

NCT05371964·Phase 1·GERN
Active, not recruiting

Primary endpoint: Part 1: Incidence, Type, and Severity of Adverse Events, Including Dose-limiting Toxicity (DLT) During the DLT Observation Period and/or Study Treatment

Interventionalopen30 enrolledStarted 2022-05Completion 2028-086 sites · United StatesGeron Corporation

A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients With Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK) Inhibitor

NCT04576156·Phase 3·GERN
Active, not recruiting

Primary endpoint: Overall survival (OS)

InterventionalRandomizedopen327 enrolledStarted 2021-04Completion 2028-0650 sites · Argentina, Australia, Austria, Belgium…Geron Corporation

A Study to Evaluate Imetelstat (GRN163L) in Transfusion-Dependent Subjects With IPSS Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) That is Relapsed/Refractory to Erythropoiesis-Stimulating Agent (ESA) Treatment

NCT02598661·Phase 2/Phase 3·GERN
Active, not recruiting

Primary endpoint: Phase 2: Percentage of Participants Without Any Red Blood Cell (RBC) Transfusion During Any Consecutive 8-Weeks Period (All Participants)

InterventionalRandomizeddouble289 enrolledStarted 2016-01Completion 2026-1050 sites · Belgium, Canada, Czechia, France…Geron Corporation

A Phase II Trial to Determine the Effect of Imetelstat (GRN163L) on Patients With Previously Treated Multiple Myeloma

NCT01242930·Phase 2·GERN
Completed

Primary endpoint: Rate of Improvement in Response

Interventionalopen13 enrolledStarted 2010-11Completion 2014-112 sites · United StatesGeron Corporation
FDA Decisions1 records
Approval1. INDICATIONS AND USAGE RYTELO is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA). RYTELO is an oligonucleotide telomerase inhibitor indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndrom(GERN)FDA ↗
NDA217779Type 1 - New Molecular EntityOrphan DrugStandardIntravenousPowder

Frequently asked questions

Who is developing imetelstat?

imetelstat is being developed by GERON CORP.

How many clinical trials involve imetelstat?

BioSniper tracks 6 clinical trials involving imetelstat.

What is imetelstat's latest FDA decision?

The most recent tracked FDA decision for imetelstat is Approval for 1. INDICATIONS AND USAGE RYTELO is indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESA). RYTELO is an oligonucleotide telomerase inhibitor indicated for the treatment of adult patients with low- to intermediate-1 risk myelodysplastic syndrom, dated June 6, 2024.