Imetelstat confers durable transfusion independence in lower-risk myelodysplastic syndrome
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Imetelstat conferred a “highly statistically significant and clinically meaningful benefit” vs. placebo in enabling certain patients with myelodysplastic syndrome to achieve transfusion independence, according to the agent’s manufacturer.
Imetelstat (Geron Corporation) targets telomerase to inhibit uncontrolled malignant stem cell and progenitor cell proliferation in myeloid hematologic malignancies, ultimately leading to apoptosis of malignant cells and potential disease-modifying activity.
The double-blind, randomized, phase3 IMerge study evaluated imetelstat among patients with lower risk, transfusion-dependent myelodysplastic syndrome who relapsed after, were refractory to or ineligible for treatment with an erythropoiesis stimulating agent, had not been treated with a hypomethylating agent or lenalidomide and did not have 5q deletion.
Researchers assigned patients in a 2:1 ratio to imetelstat (n = 118) or placebo (n = 60).
Eight-week transfusion independence served as the primary efficacy endpoint, with 24-week transfusion independence as a key secondary endpoint.
Median time on treatment was 8 months for the imetelstat group and 7 months for the placebo group at data cutoff in October.
Results showed 8-week transfusion independence rates of 39.8% in the imetelstat group vs. 15% in the placebo group (P < .001) and 24-week rates of 28% vs. 3.3% (P < .001). Median duration of transfusion independence among 8-week responders approached 1 year for the imetelstat group vs. about 13 weeks for the placebo group. Those who achieved 24-week transfusion independence with imetelstat maintained it for a median 1.5 years.
Researchers observed a statistically significant improvement in 8-week transfusion independence across disease subtypes with imetelstat, which exhibited no new safety signals. The most common nonhematologic treatment-emergent adverse events in the imetelstat group included asthenia, COVID-19, peripheral edema, headache, diarrhea and alanine aminotransferase increase. The most common hematologic treatment-emergent adverse events in the imetelstat vs. placebo group included grade 3/grade 4 thrombocytopenia (61.9% vs. 8.5%) and neutropenia (67.8% vs. 3.4%).
“Today is a great day for [patients with lower-risk myelodysplastic syndrome] who are living with the burden of transfusions. The results from the IMerge phase 3 study were resoundingly positive, presenting compelling durability of transfusion independence, delivering on the promise of imetelstat and telomerase inhibition for these patients,” John A. Scarlett, MD, Geron chairman and CEO, said in a press release. “This milestone is the first of many upcoming catalysts for Geron, with planned U.S. and EU regulatory submissions in 2023, as well as preparations for a potential U.S. commercial launch. In addition, in 2024, we expect an interim analysis of the IMpactMF phase 3 trial of imetelstat in relapsed/refractory myelofibrosis.”
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