Dr Ball on Data From from the IMerge Trial in Low– to Intermediate-Risk MDS

Somedeb Ball, MBBS, assistant professor of medicine, Division of Hematology Oncology, Department of Medicine, Vanderbilt University Medical Center, discussed the efficacy of the telomerase inhibitor imetelstat as evaluated in the phase 2/3 IMerge/MDS3001 trial (NCT02598661 in the treatment of low– to intermediate-risk myelodysplastic syndromes (MDS), highlighting key takeaways from this study.

This double-blind, randomized trial evaluated the treatment with imetelstat vs placebo. Imetelstat has been explored in various oncological contexts, but the agents role in MDS has become particularly significant, Ball explains. Notably, on March 24, 2024, in a 12 to 2 vote, the FDA’s Oncologic Drugs Advisory Committee agreed that the benefits of imetelstat outweigh the risks in the treatment of transfusion-dependent anemia in adult patients with International Prognostic Scoring System (IPSS) low- to intermediate-1–risk MDS who have not responded to, no longer respond to, or are ineligible for treatment with erythropoiesis-stimulating agents (ESAs).

The IMerge trial evaluated participants who fit this criteria, as well as who had a baseline serum erythropoietin level exceeding 500 U/L, indicating probable resistance to erythropoietin-based therapies. Ball highlights the challenge in managing patients with MDS who do not respond to conventional ESAs, underscoring the utility of imetelstat.

Furthermore, the trial's outcomes revealed a notable efficacy of imetelstat in achieving transfusion independence compared to the placebo group, he continues. This was quantified by a significant increase in the hemoglobin levels, Ball explains.

These findings position imetelstat as a promising therapeutic option for this patient population, he continues. Additionally, Ball shares his hoped for an FDA approval of the agent in patients with low– to intermediate-risk MDS who are relapsed/refractory to other treatment options. However, luspatercept-aamt (Reblozyl) is approved by the FDA for the treatment of anemia without prior ESA use in these patients patients who may require regular red blood cell transfusions, Ball concludes.