Activin receptor fusion proteins can be used in treatment of beta-talassemia

Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc. (NASDAQ:XLRN) presented  interim data from two studies of candidates from the companies' joint development program in beta-thalassemia. at the 19th European Hematology Association (EHA) annual congress. Companies’ products sotatercept and ACE-536 demonstrated good activity in patients with both transfusion dependent and non-transfusion dependent beta-thalassemia. Significant increase in hemoglobin concentration and reduced iron overload and transfusion burden were observed. Several patients had left the trial due to adverse effects like arrhythmia, bone pain and phlebitis.

ACE-536 is a modified type IIB activin receptor fusion protein. Sotatercept is an activin receptor type IIA fusion protein. Both   act as  ligand traps for members in the Transforming Growth Factor-Beta (TGF-β) superfamily involved in the late stages of erythropoiesis (red blood cell production). and regulate  late-stage erythrocyte (red blood cell) precursor cell differentiation and maturation.