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IMerge showed that imetelstat, a drug that blocks telomerase in the abnormal blood-forming cells, freed many people with lower-risk myelodysplastic syndromes from transfusions for months or years after erythropoietin had failed.
IMerge enrolled patients with very low-, low- or intermediate-1 risk myelodysplastic syndromes who needed regular red cell transfusions, had relapsed after or were refractory to erythropoiesis-stimulating agents, and did not have a del(5q) abnormality. The phase 3 part randomised them two to one to imetelstat, an oligonucleotide that inhibits telomerase, or placebo, with red cell transfusion independence for at least eight weeks as the primary endpoint.
Imetelstat produced eight-week and 24-week transfusion independence in significantly more patients than placebo, with reductions in the mutant clone burden in some. Cytopenias were the main toxicity. The drug was approved in the United States in June 2024 for this population.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
289 enrolled.
Rate difference 25% (95% CI 9.9 to 36.9); median follow-up 19.5 months
SourceShares Uwe Platzbecker, Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Shares Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Shares Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Shares Myelodysplastic syndromes / neoplasms (MDS) and the tag subtype-trials.
Shares Geron Corporation, Imetelstat, Oligonucleotide therapeutics, Myelodysplastic syndromes / neoplasms (MDS).
Shares Geron Corporation, Imetelstat, Oligonucleotide therapeutics.
Shares Uwe Platzbecker, Imetelstat, Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS).
Shares Uwe Platzbecker, Myelodysplastic syndromes / neoplasms (MDS).