CAMBRIDGE, Mass.– August 19, 2010 - Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the United States Food and Drug Administration (FDA) granted orphan designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients experience a progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.
“In the past two weeks, the FDA has granted both orphan status and Fast Track designation to ACE-031 in recognition of the drug’s potential to address the enormous unmet medical need for therapeutics to treat DMD patients”, said Matthew Sherman, M.D., Chief Medical Officer at Acceleron. “We will continue to work collaboratively with clinical investigators, health authorities and patient advocacy groups around the world to develop ACE-031.”
Orphan drug designation is granted by the FDA Office of Orphan Products Development to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. Under the Orphan Drug Act, the FDA may provide grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following drug approval by the FDA. The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website at www.fda.gov
About ACE-031
ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling through a cell surface receptor called activin receptor type IIB (ActRIIB). ACE-031 is a recombinant fusion protein that is produced by joining a portion of the human ActRIIB receptor to a portion of a human antibody. This creates a freely circulating, decoy version of ActRIIB which removes proteins, such as GDF-8 (myostatin), that limit the growth and regeneration of muscle. Animal studies with ACE-031 suggest that blocking signaling through ActRIIB leads to increased muscle mass and improved physical function in a range of animal models of muscle disease, including models of muscular dystrophy. ACE-031 is currently being studied in a phase 2 clinical trial of patients with Duchenne Muscular Dystrophy (DMD) who are also receiving corticosteroid therapy.
About Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy (DMD) is a debilitating and fatal genetic disorder characterized by the progressive loss of muscle strength and function. It primarily affects boys and occurs in approximately 1 in every 3,500 live male births. DMD is caused by genetic mutations that result in the absence or a defect in dystrophin, a protein necessary to maintain the structural integrity of muscle fibers. This condition leads to deterioration of and damage to skeletal muscles, which eventually become infiltrated by non-functional scar and fatty tissue. As a result, patients experience a relentless decline in muscle strength that impairs their ability to walk, breathe and live independently. Many patients spend the majority of their lives confined to wheelchairs and eventually lose all upper body function. Few patients survive beyond their late-20s when their heart and respiratory muscles weaken and eventually fail.
About Acceleron
Acceleron is a privately held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel biotherapeutics that modulate the growth of red blood cells, bone, muscle, fat and the vasculature to treat musculoskeletal, metabolic and cancer-related diseases. Acceleron’s scientific approach takes advantage of its unique insight into the regenerative powers of the TGF-ß superfamily of proteins. ACE-011 is in Phase 2 clinical trials in patients with anemia. ACE-031 is being studied in a Phase 2 clinical trial in patients with Duchenne Muscular Dystrophy and ACE-041 is being studied in a Phase 1 clinical trial in patients with advanced cancer. In addition, the company is developing several other new product candidates that increase muscle mass, control angiogenesis, inhibit fat accumulation and increase bone mass and strength. Acceleron utilizes proven biotherapeutic technologies and capitalizes on the company’s internal GMP manufacturing capability to rapidly and efficiently advance its therapeutic programs. 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 more information, visit www.acceleronpharma.com
CONTACT:
Acceleron Pharma: Steven Ertel, 617-649-9234 Vice President, Corporate Development
Paul Kidwell (Media) Suda Communications LLC 617-296-3854
Paul Kidwell 617.296.3854: Office 617.680.1088: Mobile
