Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases today announced that it has initiated a Phase 1 clinical study of ACE-536. ACE-536 is a ligand trap that increases red blood cells and hemoglobin by inhibiting members of the TGF-beta superfamily involved in late stages of erythropoiesis. ACE-536 is being developed for the treatment of anemia in a global collaboration with Celgene Corporation. The Phase 1 clinical trial is a multiple-dose, dose-escalating study designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of ACE-536. Acceleron will receive a $7.5 million payment from Celgene for achieving this milestone event.
"ACE-536 has the potential to make a significant impact on the treatment of anemia, specifically, in diseases such as Myelodysplastic syndromes and beta-thalassemia in which the severe anemia isn't well managed by currently available therapies. By affecting production of red blood cells via a mechanism distinct from erythropoiesis stimulating agents, ACE-536 may become an option for patients for whom current therapies are ineffective," said John Knopf, Ph.D., Chief Executive Officer of Acceleron. "Acceleron's leadership in the biology of the TGF-beta superfamily proteins and our related expertise in discovering, developing and manufacturing potent biologic modulators of these proteins has enabled incredible productivity achievements in a short period of time. ACE-536 is our fourth internally discovered, developed and manufactured drug to enter human clinical trials in the past five years."