New England Journal of Medicine publishes results from pivotal phase III BELIEVE trial of Reblozyl in adult patients With beta thalassemia- BMS

Bristol-Myers Squibb Company and Acceleron Pharma Inc. announced that the New England Journal of Medicine (NEJM) has published results from BELIEVE, the pivotal Phase III study evaluating the safety and efficacy of Reblozyl (luspatercept-aamt) for the treatment of anemia in adults with beta thalassemia who require regular red blood cell (RBC) transfusions.

Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb said. “Patients with beta thalassemia experience chronic anemia, often requiring life-long treatment with red blood cell transfusions, which are associated with multiple medical complications. The ability of Reblozyl to reduce the frequency and burden of regular red blood cell transfusions is clinically meaningful for these patients.”

In the BELIEVE trial, a significantly higher proportion of patients achieved a greater than 33% reduction in RBC transfusion burden from baseline (with a reduction of at least two units) during weeks 13-24 when treated with Reblozyl compared to placebo, meeting the study’s primary endpoint. The study also met all key secondary endpoints with a significantly greater proportion of patients receiving Reblozyl compared to placebo achieving a greater than 33% reduction in RBC transfusion burden during weeks 37-48 and a greater than 50% reduction during weeks 13-24 or weeks 37-48. The most common adverse events (AEs) of any grade in greater than 5% of the Reblozyl treatment group compared to placebo were bone pain (19.7% vs. 8.3%), arthralgia (19.3% vs. 11.9%), dizziness (11.2% vs. 4.6%), hypertension (8.1% vs. 2.8%) and hyperuricemia (7.2% vs 0%). Grade >3 treatment emergent AEs were reported in a greater proportion of patients receiving Reblozyl compared to placebo (29.1% vs. 15.6%). The most common Grade >3 AEs reported with Reblozyl were anemia (3.1% vs. 0%), increased liver iron concentration (2.7% vs. 0.9%) and hyperuricemia (2.7% vs. 0%) compared to placebo, respectively. Serious adverse events were reported in 15.2% of patients receiving Reblozyl and 5.5% of patients receiving placebo.