Acceleron Pharma commences Phase 2 clinical trial of ACE-031 protein therapeutic in DMD patients

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 initiation of a Phase 2 clinical trial with ACE-031 in patients with Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease. ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling of a cell surface receptor called the activin receptor type IIB (ActRIIB).

“We are enthusiastic about the therapeutic potential for ACE-031 in DMD based on its compelling preclinical and clinical activity”

"We are enthusiastic about the therapeutic potential for ACE-031 in DMD based on its compelling preclinical and clinical activity," said Matthew Sherman, M.D., Chief Medical Officer at Acceleron. "DMD patients suffer from progressive muscle weakness that gradually impairs their ability to walk, breathe voluntarily and live independently, and treatment with ACE-031 has the potential to increase muscle strength and slow the progression of the disease."

"In preclinical studies in the mdx mouse model of DMD, ACE-031 has shown potent effects on increasing muscle mass," said Jasbir Seehra, PhD, Chief Scientific Officer and co-founder of Acceleron. "These studies have also demonstrated improvements in muscle strength and function, which we hope will translate directly to the clinical setting in patients with DMD."