Celgene, Acceleron on Results of Phase 3 Beta-Thalassemia Trial at ASH

Pivotal phase 3 data demonstrated treatment with investigational luspatercept resulted in significant reduction of transfusion burden compared to placebo

Regulatory submissions planned in the United States and Europe in the first half of 2019

Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced results from a pivotal, phase 3 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 60^th 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 ≥33% reduction in RBC transfusion burden (with a reduction of ≥ 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.

https://www.marketscreener.com/ACCELERON-PHARMA-INC-14297719/news/Celgene-Corporation-and-Acceleron-Pharma-Announce-Results-of-the-Phase-3-BELIEVE-Trial-Evaluating-27693110/