Phase III BELIEVE study of luspatercept achieved primary and all key secondary endpoints for patients with transfusion-dependent Beta-Thalassemia. -Celgene + Acceleron Pharma.

Celgene Corporation and Acceleron Pharma Inc. announced results from a phase III, randomized, double-blind, multi-center clinical study (BELIEVE). Luspatercept achieved a highly statistically significant improvement in the primary endpoint of erythroid response, which was defined as at least a 33 percent reduction from baseline in red blood cell (RBC) transfusion burden with a reduction of at least 2 units during the protocol-defined period of 12 consecutive weeks, from week 13 to week 24, compared to placebo.

BELIEVE evaluated the efficacy and safety of luspatercept plus best supportive care versus placebo plus best supportive care in adults with transfusion-dependent beta-thalassemia. In addition to achieving the primary endpoint of the study, luspatercept also met all key secondary endpoints of demonstrating statistically significant improvements in RBC transfusion burden from baseline of at least a 33 percent reduction during the period from week 37 to week 48, at least a 50 percent reduction during the period from week 13 to week 24, at least a 50 percent reduction during the period from week 37 to week 48, and a mean change in transfusion burden from week 13 to week 24. Adverse events observed in the study were generally consistent with previously reported data.