Cambridge, Mass. - Jan. 6, 2011 - 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 it has been awarded a $1.5 million grant from the Muscular Dystrophy Association to support clinical studies of ACE-031 in Duchenne Muscular Dystrophy (DMD), a disabling neuromuscular disease in which patients experience a progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic designed to build muscle and increase strength by blocking proteins that inhibit muscle growth and strength.
"We are honored to be recognized and supported by the Muscular Dystrophy Association as we work collectively to develop promising therapies for the treatment of patients with Duchenne Muscular Dystrophy," said John Knopf, PhD, CEO of Acceleron. "This award along with our collaboration with Shire brings together the resources of several committed groups to evaluate the therapeutic potential of ACE-031 in DMD."
"Having invested more than $164 million on DMD-related research, MDA is eager to help Acceleron advance the clinical trial work needed to determine optimal doses of ACE-031 in terms of safety and pharmacodynamic activity," said R. Rodney Howell, M.D., Chairman of the MDA Board of Directors.
The MDA grant, awarded through the Association's Venture Philanthropy Program (MVP), will support ongoing clinical studies of ACE-031 in boys with DMD. The new investment will enable Acceleron to gather safe dosing and pharmcodynamic activity information vital to future studies of ACE-031. For more information on these ongoing studies, visit clinicaltrials.gov and query study identifiers NCT01099761 and NCT01239758.
"MDA is pleased to support Acceleron's efforts in the fight against Duchenne Muscular Dystrophy," said Valerie Cwik, M.D., MDA Executive Vice President - Research and Medical Director. "Our strategic investment should help Acceleron expedite the next phase of ACE-031 human clinical trials."
About Muscular Dystrophy Association
MDA is the nonprofit health agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education. Often credited with pioneering fields of neuromuscular disease research and clinical care, the Association is the world's leading non-governmental funder of neuromuscular disease research. MDA's current 3-year investment in DMD-related research exceeds $29.8 million, and its historical investment in DMD-related research is $164.4 million.
MDA Venture Philanthropy (MVP) is the drug development arm of MDA's Translational Research Program that is exclusively focused on identifying and funding the commercialization of treatments and cures for neuromuscular diseases.
MDA is the first nonprofit organization to receive a Lifetime Achievement Award from the American Medical Association "for significant and lasting contributions to the health and welfare of humanity." Thanks largely to the unparalleled quality of medical services available through its 200 hospital-affiliated clinics, substantial gains have been made in both DMD lifespan and also quality of life for boys and men living with DMD. For more information, go to www.mda.org.
ACE-031 is an investigational protein therapeutic designed to build muscle and increase 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 interferes with proteins, such as GDF-8 (myostatin), that normally limit the growth and regeneration of muscle by binding to and activating endogenous ActRIIB. Recent preclinical and clinical studies with ACE-031 suggest that blocking signaling through ActRIIB may be a way to increase muscle mass and improve muscle strength and function. In a range of animal models of muscle disease, including models of muscular dystrophy, muscle loss related to corticosteroid treatment, androgen deprivation or advanced age, ACE-031 increased muscle mass, strength and physical function. Unlike the mutation-specific RNA-based therapeutics in clinical development for Duchenne Muscular Dystrophy (DMD), ACE-031 could potentially benefit patients with DMD, irrespective of the underlying genetic mutation. ACE-031 is being developed in collaboration with Shire.
About Acceleron Pharma
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-β superfamily of proteins. 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 further information on Acceleron Pharma, please visit www.acceleronpharma.com
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