Announcement • May 14
Geron Corporation Presents First Real-World Evidence Study of Rytelo (Imetelstat) in Lower-Risk Myelodysplastic Syndromes
Geron Corporation announced that the first real-world evidence study of RYTELO (imetelstat) in patients with lower-risk myelodysplastic syndromes (LR-MDS) will be presented at the European Hematology Association (EHA) 2026 Congress. The retrospective and prospective investigator-sponsored study, conducted at the Moffitt Cancer Center, reported safety and clinical efficacy of imetelstat in advanced, heavily transfusion dependent patients with LR-MDS, including patients with extensive prior therapies and after luspatercept failure. The data evaluated imetelstat in 40 patients with lower-risk MDS treated at Moffitt Cancer Center in a real-world setting following FDA approval. Patients had advanced, heavily transfusion-dependent disease and extensive prior treatment exposure, including prior luspatercept, erythropoiesis-stimulating agents (ESAs), hypomethylating agents and lenalidomide. With 14-month follow-up, the analysis reported a red blood cell (RBC) transfusion independence rate of 37.5% lasting at least eight weeks with several responses ongoing and identified potential predictors of response. The safety profile was generally consistent with the known safety profile of imetelstat. Cytopenias were the most common Grade 3/4 adverse event. Exploratory analyses also suggested a trend toward improved responses when imetelstat was used within the first three lines of therapy. Geron will also have additional presentations at the EHA 2026 Congress and the American Society of Clinical Oncology (ASCO) 2026 Annual Meeting, including abstracts related to its ongoing myelofibrosis clinical programs, including an updated overall survival analysis in patients with myelofibrosis treated with imetelstat in the Phase 2 IMbark trial compared with real-world data. EHA 2026 presentations include real-world outcomes of imetelstat in heavily pretreated LR-MDS patients, an updated overall survival analysis in relapsed or refractory myelofibrosis from IMBark versus real-world data, and a Phase 1/1B trial of imetelstat and azacitidine with or without venetoclax in relapsed acute myeloid leukemia (IMAGINE Trial). ASCO 2026 presentations include an updated overall survival analysis in relapsed/refractory myelofibrosis versus real-world data and an updated protocol for IMproveMF, a Phase 1b trial of imetelstat plus ruxolitinib in patients with intermediate-1/2 or high-risk myelofibrosis. RYTELO is an oligonucleotide telomerase inhibitor approved in the U.S. for adult patients with LR-MDS with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to, lost response to, or are ineligible for ESAs, administered as an intravenous infusion over two hours every four weeks. In the European Union, RYTELO is approved as a monotherapy for adult patients with transfusion-dependent anemia due to very low, low or intermediate risk non-del 5q MDS with unsatisfactory response to or ineligibility for erythropoietin-based therapy. RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity, preventing uncontrolled cell division in abnormal bone marrow cells. It is the first and only telomerase inhibitor approved by both the FDA and the European Commission, developed and exclusively owned by Geron. RYTELO can cause thrombocytopenia, with Grade 3/4 decreased platelets occurring in 65% of MDS patients, and neutropenia, with Grade 3/4 decreased neutrophils in 72% of patients. Infusion-related reactions occurred in 8% of patients, with the most common being headache. Patients should be premedicated with diphenhydramine and hydrocortisone at least 30 minutes prior to infusion. Based on animal findings, RYTELO can cause embryo-fetal harm; females of reproductive potential should use effective contraception during treatment and for one week after the last dose. Serious adverse reactions occurred in 32% of patients, including sepsis (4.2%), fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%). Fatal adverse reactions occurred in 0.8% of patients. Most common adverse reactions included decreased platelets, decreased white blood cells, decreased neutrophils, elevated liver enzymes, fatigue, arthralgia/myalgia, COVID-19 infections, and headache.
