Acceleron Pharma Starts Phase 2 clinical trials with ACE-031 in patients with Duchenne muscular dystrophy

‘In preclinical studies in the mdx mouse model of DMD, ACE-031 has demonstrated potent effects on gains in muscle mass,’ said Jasbir Seehra, Ph., Chief Scientific Officer and co-founder of Acceleron. ‘These studies have also shown improvements in muscle strength and function, which we hope will translate directly to the clinic in patients with DMD.’Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues, including muscles, bones, fat, red blood cells and vasculature, today announced the launch of a Phase 2 clinical study of ACE-031 in patients with Duchenne muscular dystrophy , a fatal neuromuscular disease. ACE-031 is an experimental therapeutic protein that builds muscle and increases strength by inhibiting the signaling of a cell surface receptor called activin receptor type IIB .

‘We are excited about the therapeutic potential for ACE-031 in DMD based on its compelling preclinical and clinical activities,’ said Matthew Sherman, MD, Chief Medical Officer of Acceleron. ‘The DMD patients suffer from progressive muscle weakness that gradually impairs their ability to walk, to breathe freely and live independently, and treatment with ACE-031 has the potential to increase muscle strength and slow the progression of the disease . ‘

ACE-031 is a protein Experimental Therapeutics that builds muscles and increases the strength of signaling by inhibiting through a receptor on the cell surface called activin receptor type IIB . ACE-031 is a fully human, recombinant fusion protein that is produced by combining part of the human ActRIIB receptor to a portion of a human antibody. This creates a freely circulating, version recall of ActRIIB which removes proteins, such as GDF-8 , that limit the growth and muscle regeneration. Recent studies with ACE-031 suggest that blocking signaling through ActRIIB may be an effective way to increase muscle mass and improve physical function. In a range of animal models of muscle diseases, including models of muscular dystrophy and muscle loss associated with corticosteroid therapy, androgen deprivation or advanced age, ACE-031 function of increased muscle mass, strength and fitness

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