IMerge
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.
Overview
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.
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