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In IMerge, imetelstat, the first telomerase inhibitor to reach approval, freed about 40% of heavily transfused MDS patients from transfusions for eight weeks and 28% for six months, versus 15% and 3% with placebo.
IMerge randomised 178 patients with lower-risk, non-del(5q) MDS who were red-cell transfusion dependent and had relapsed after or were refractory to, or ineligible for, erythropoiesis-stimulating agents to intravenous imetelstat every four weeks or placebo (2:1). The primary endpoint was eight-week red-cell transfusion independence. This was achieved by 39.8% versus 15.0%; 24-week independence was 28.0% versus 3.3%, and responses lasted around a year with reductions in the SF3B1 mutant allele burden suggesting disease modification. Grade 3-4 thrombocytopenia and neutropenia were frequent but short-lived. Imetelstat was approved in 2024, the first drug in its class.
IMerge validated telomerase as a drug target in cancer, decades after its discovery, and gave a second-line option for MDS patients whose anaemia no longer responds to erythropoietin or luspatercept. The hint of clonal reduction is what makes the drug interesting beyond transfusion counts. Cytopenias require close monitoring in the first cycles.
Shares Pierre Fenaux, Luspatercept, Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS).
Shares Imetelstat, Luspatercept, Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS).
Shares Imetelstat, Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS).
Shares Luspatercept, Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS).
Shares Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS).
Shares Imetelstat, Luspatercept, Lower-risk myelodysplastic syndromes.
Shares Lower-risk myelodysplastic syndromes, Myelodysplastic syndromes / neoplasms (MDS).
Shares Luspatercept, Myelodysplastic syndromes / neoplasms (MDS).