Reblozyl study evaluating the treatment of anemia associated with beta thalassemia was presented at ASH meeting.- BMS + Acceleron Pharma

Bristol-Myers Squibb Company and Acceleron Pharma announced data evaluating the erythroid maturation agent (EMA) Reblozyl (luspatercept-aamt) in patients with anemia associated with a range of serious and rare blood diseases were presented at the 2019 ASH Annual Meeting in Orlando, Fla. Data from investigational uses of luspatercept-aamt included the BELIEVE study in adult patients with anemia associated with beta thalassemia who require regular RBC transfusions.

Updated Results from the BELIEVE Study : The companies also presented at ASH an updated analysis of the pivotal phase III BELIEVE study evaluating luspatercept-aamt in adult patients with beta thalassemia who require regular RBC transfusions. In the study, 336 patients were randomized 2:1, with 224 patients receiving luspatercept-aamt. As of the clinical cutoff (January 7 2019), 45.1% (101/224) of luspatercept-aamt patients and 2.7% (3/112) of placebo patients had achieved at least a 33% reduction in RBC transfusion burden over any 24-week period. Among the luspatercept-aamt patients who had a response, 73.3% (74/101) had at least 2 separate responses, with 59.4% (60/101) having 3 or more responses. The median duration of clinical benefit (defined as the time from first response of at least a 33% reduction in RBC transfusion over any 24-week interval to discontinuation due to any cause) for patients who responded to luspatercept-aamt was 76.3 weeks (24-128.1).

The most commonly observed AEs in the safety population (n=332) included in luspatercept-aamt and placebo patients, respectively, bone pain (20.2% vs. 8.3%), arthralgia (21.1% vs. 14.7%) and dizziness (12.1% vs. 4.6%). These AEs typically occurred early on, and incidence decreased over time.