CAMBRIDGE, Mass.--(BUSINESS WIRE)--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.”
About 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 the late stages of erythropoiesis. ACE-536 affects the red blood cell production by a distinct mechanism from currently available erythropoiesis stimulating agents and has the potential to treat anemia in diseases such as Myelodysplastic syndromes (MDS) and beta-thalassemia that are poorly managed with current therapy. In MDS, a subset of patients has a defect in the late stage maturation process of red blood cell precursors. ACE-536 specifically targets and positively affects late stage red blood cell maturation and therefore ACE-536 may be particularly effective in correcting the anemia in these MDS patients.
About Acceleron
Acceleron is a privately-held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel protein therapeutics for orphan diseases and cancer. Acceleron’s scientific approach takes advantage of its unique insight to discover first-in-class therapies based on the TGF-β protein superfamily. Acceleron utilizes proven biotherapeutic technologies and capitalizes on the company’s internal GMP manufacturing capability to advance its therapeutic programs rapidly and efficiently. The investors in Acceleron include Advanced Technology Ventures, Alkermes, Bessemer Ventures, Celgene, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For further information on Acceleron, please visit www.acceleronpharma.com.