On May 28, 2025 Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, reported presentations on RYTELO (imetelstat) at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting and the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress (Press release, Geron, MAY 28, 2025, View Source [SID1234653440]). Together, the presentations reinforce the potential benefits of the first-in-class oligonucleotide telomerase inhibitor RYTELO for a range of patients with lower-risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia and showcase the progress Geron is making with the ongoing IMpactMF and IMproveMF trials of imetelstat in myelofibrosis (MF).
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"Transfusion independence is an important goal for LR-MDS patients, but one that historically has not been achievable for many," said Rami S. Komrokji, M.D., Vice Chair, Malignant Hematology Department, Moffitt Cancer Center. "While imetelstat is already playing a vital role in LR-MDS, these new analyses being presented at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) reinforce its potential to give a broad range of patients more time without transfusions and should also give clinicians confidence to add it to their toolkit as a second-line option for eligible patients."
Presentations at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) include:
New pooled, post-hoc analyses expand on the pivotal IMerge results across challenging LR-MDS subpopulations
Analysis of patients with ring sideroblast negative (RS-) disease, showing that these difficult-to-treat patients appeared to experience clinical benefit with RYTELO, including ≥8-week, ≥24-week and ≥1-year red blood cell transfusion independence (RBC-TI), duration of RBC-TI, and hemoglobin rise in patients who achieved RBC-TI, consistent with prior findings from the overall Phase 3 IMerge population.
Analysis showing clinical benefit with RYTELO regardless of baseline serum erythropoietin (sEPO) level, supporting the use of RYTELO in the frontline setting for LR-MDS patients ineligible for erythropoiesis-stimulating agents (ESAs), and in the second-line setting after ESAs regardless of sEPO.
Analysis showing clinical benefit with RYTELO in ESA-ineligible or relapsed/refractory patients regardless of prior treatment with luspatercept or lenalidomide. Patients who had prior treatment with a hypomethylating agent (HMA) showed modest clinical activity with RYTELO.
Patient-centric outcome measures offering deeper insight on RYTELO impact in new post-hoc analyses from Phase 3 IMerge trial
Analysis showing that certain RYTELO-treated patients experienced sustained improvements in health-related quality-of-life (QOL) compared with placebo, as measured by certain categories in the patient-reported QOL in Myelodysplasia Scale (QUALMS) instrument.
Exploratory analysis suggesting that certain patients treated with RYTELO experienced a longer mean duration of time without transfusion reliance or relapse (TWiTR), compared with placebo.
New updates on ongoing trials in MF
Recently updated preliminary data from the dose escalation portion of the Phase 1/1b IMproveMF trial showing the combination of imetelstat and ruxolitinib was generally well-tolerated, with no dose-limiting toxicities observed as of the data cutoff date, and encouraging early dose-dependent efficacy data suggesting the potential of the combination for patients with intermediate-1 (INT-1), intermediate-2 (INT-2) or high-risk (HR) MF.
Trial-in-progress (TiP) update on the Phase 3 IMpactMF trial investigating imetelstat in relapsed/refractory MF, reporting that the trial has met approximately 80% of its enrollment target as of February 2025.
"At ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper), we’re excited to present new analyses on how RYTELO can deliver meaningful benefit across a range of LR-MDS patients who otherwise may have had limited options, whether due to their ring sideroblast status, baseline EPO level, or their treatment history," said Faye Feller, M.D., Executive Vice President and Chief Medical Officer of Geron. "We believe the depth and breadth of our data reinforce the potential of RYTELO to address critical unmet needs in LR-MDS and, combined with the advances we are making with our myelofibrosis program, underscore our confidence in telomerase inhibition as a potentially transformative clinical strategy and our commitment to exploring its full potential."
See below for a full schedule of presentations at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper).
ASCO 2025 Presentations
Presentation Title
Author
Abstract Number
Presentation Details
IMproveMF update: phase 1/1B trial of imetelstat (IME)+ruxolitinib (RUX) in patients (pts) with intermediate (INT)-1, INT-2, or high-risk (HR) myelofibrosis (MF)
John O. Mascarenhas, M.D.
#6515
Rapid Oral, Fri. May 30, 1:00-2:30 PM CDT
Effect of prior treatment (tx) on the clinical activity of imetelstat (IME) in transfusion-
dependent (TD) patients (pts) with erythropoiesis-stimulating agent (ESA), relapsed or
refractory (R/R)/ineligible lower-risk myelodysplastic syndromes (LR-MDS)
Rami S. Komrokji, M.D.
#6569
Poster, Sun. June 1, 9:00 AM-12:00 PM CDT
IMpactMF, randomized, open-label, phase 3 trial of imetelstat (IME) versus best available therapy (BAT) in patients (pts) with intermediate-2 (INT-2) or high-risk (HR) myelofibrosis (MF) relapsed or refractory (R/R) to Janus kinase inhibitors (JAKi)
John O. Mascarenhas, M.D.
#TPS6588
Poster, Sun. June 1, 9:00 AM-12:00 PM CDT
EHA 2025 Presentations
Presentation Title
Author
Abstract Number
Presentation Details
Increased Duration of Time Without Transfusion Reliance (TWiTR) For Patients with LR-MDS Treated with Imetelstat Versus Placebo in the IMerge Trial
Mikkael A. Sekeres, M.D.
#PF646
Poster, Fri. June 13, 18:30-19:30 CEST
IMproveMF Update: Phase 1/1B Trial of Imetelstat + Ruxolitinib in Patients with INT-1, INT-2, or High-Risk MF
John O. Mascarenhas, M.D.
#PF830
Poster, Fri. June 13, 18:30-19:30 CEST
IMpactMF: Randomized, Open-Label, Phase 3 Trial of Imetelstat vs Best Available Therapy in Patients with INT-2 or High-Risk MF Relapsed/Refractory to JAK Inhibitors
John O. Mascarenhas, M.D.
#PF841
Poster, Fri. June 13, 18:30-19:30 CEST
Health-related Quality of Life Outcomes in Patients with Lower-Risk Myelodysplastic Syndromes Treated with Imetelstat in the IMerge Trial
María Díez-Campelo, M.D., Ph.D.
#PS1639
Poster, Sat. June 14, 18:30-19:30 CEST
Outcomes of Imetelstat Therapy in RS-Negative LR-MDS from the Pooled IMerge Study Populations
Valeria Santini, M.D.
#PS1622
Poster, Sat. June 14, 18:30-19:30 CEST
Outcomes with Imetelstat by Serum Erythropoietin Levels in Patients with LR-MDS Who Were Treatment Naïve or Who Had Prior Treatment with Erythropoiesis-Stimulating Agents
Rami S. Komrokji, M.D.
#PS1640
Poster, Sat. June 14, 18:30-19:30 CEST
Characterization and Management of Transient Cytopenias Associated with Imetelstat in LR-MDS from the IMerge Trial
Amer M. Zeidan, M.D.
#PB2760
Publication-only
Please see the full presentations for important qualifications and limitations on these post-hoc analyses.
About RYTELO (imetelstat)
RYTELO is an oligonucleotide telomerase inhibitor approved in the U.S. for the treatment of adult patients with low-to-intermediate-1 risk myelodysplastic syndromes (LR-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). It is indicated to be administered as an intravenous infusion over two hours every four weeks.
In addition, RYTELO is approved in the European Union as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.
RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration and the European Commission.