Bristol Myers Squibb has announced positive top-line results from an ongoing ex-U.S. Phase 2 registrational study (NCT05664737) evaluating Reblozyl® (luspatercept-aamt) versus placebo for the treatment of anemia in adults with alpha (α)-thalassemia.
The study enrolled both non–transfusion-dependent (NTD) and transfusion-dependent (TD) patients and met its respective primary endpoints in both cohorts. In NTD patients, Reblozyl demonstrated a statistically significant and clinically meaningful increase in hemoglobin levels. In TD patients, the therapy achieved a statistically significant and clinically meaningful reduction in red blood cell (RBC) transfusion burden. The study also met all key secondary endpoints, and safety findings were consistent with the known profile of Reblozyl in thalassemia.
The trial is the first registrational Phase 2 study specifically designed to address the needs of patients with alpha-thalassemia, particularly in China, where the company plans to discuss the data with the Center for Drug Evaluation. The results are also expected to be presented at an upcoming medical congress.
The Phase 2 trial is evaluating the efficacy and safety of luspatercept plus best supportive care compared with placebo in adults and adolescents with alpha-thalassemia across both TD and NTD cohorts. In the NTD cohort, the primary endpoint measured an increase from baseline of at least 1 gram per deciliter in mean hemoglobin levels over a continuous 12-week interval from Week 13 to Week 24 in the absence of RBC transfusion. In the TD cohort, the primary endpoint assessed a reduction of at least 50% in RBC transfusion burden from baseline, with a minimum reduction of two units during any continuous 12-week period between Week 13 and Week 48 compared with the 12-week interval prior to the first dose. The adolescent NTD and TD cohorts remain ongoing.
Reblozyl is a first-in-class therapy designed to promote late-stage red blood cell maturation, based on findings from animal models. It has been developed and commercialized under a global collaboration with Merck since November 2021. In the United States, Reblozyl is approved for the treatment of anemia in adult patients with beta-thalassemia who require regular red blood cell transfusions.
It is also indicated for anemia in adult patients with very low- to intermediate-risk myelodysplastic syndromes, including erythropoiesis-stimulating agent-naïve patients who may require regular transfusions, as well as patients with myelodysplastic syndromes with ring sideroblasts or myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis who have failed erythropoiesis-stimulating agents and require two or more red blood cell units over eight weeks.
Reblozyl is not indicated as a substitute for red blood cell transfusions in patients requiring immediate correction of anemia, and in the U.S. it is not approved for use in non-transfusion-dependent beta-thalassemia.
The positive Phase 2 results position Reblozyl as a potential new treatment option for adults with alpha-thalassemia, a condition with limited therapeutic choices. With regulatory discussions planned in China and further data presentation ahead, the findings mark an important step toward expanding treatment access in this underserved patient population.
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