Positive results from BELIEVE study of luspatercept for the treatment of adults with beta-thalassemia-associated anemia. Celgene + Acceleron.

Celgene Corporation and Acceleron Pharma Inc.announced results from a pivotal, phase III trial (BELIEVE) evaluating the safety and efficacy of luspatercept for the treatment of adults with beta-thalassemia-associated anemia who require regular red blood cell (RBC) transfusions. The data were presented by Maria Domenica Cappellini, M.D. in an oral session of the 60th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, CA (Abstract #163).

�Currently, the standard of care to help patients with beta-thalassemia manage their anemia is regular, lifelong red blood cell transfusions, which over time can result in iron overload and life-threatening co-morbidities,� said Professor Cappellini, M.D., Professor of Medicine, University of Milan - Fondazione IRCCS. �These findings from the BELIEVE study are exciting because they suggest that luspatercept may help patients reduce their dependence on red blood cell transfusions.�

BELIEVE met the primary endpoint of erythroid response, defined as a greater than 33% reduction in RBC transfusion burden (with a reduction of more than 2 units of RBC) during weeks 13�24 compared to the baseline 12-week interval prior to randomization. The study also included secondary endpoints that evaluated the impact of treatment on RBC transfusion burden. Mean change in transfusion burden from baseline to weeks 13-24 (luspatercept vs. placebo) was -1.35 RBC units. Grade 3 or higher treatment-emergent adverse events (TEAEs) were reported in 29.1% (65/223) of patients receiving luspatercept and 15.6% (17/109) of patients receiving placebo. Serious adverse events were reported in 15.2% (34/223) of patients receiving luspatercept and 5.5% (6/109) of patients receiving placebo. A TEAE of acute cholecystitis resulted in death in one placebo-treated patient (0.9%). No luspatercept-treated patients died due to TEAEs. All grades of thromboembolic events, including DVT, PE, portal vein thrombosis, ischemic stroke, thrombophlebitis, and superficial phlebitis were reported in 8 of 223 (3.6%) luspatercept-treated versus 1 of 109 (0.9%) placebo-treated patients.