Acceleron Announces Plans to Initiate a Phase 2 Trial of ACE-083 in Charcot-Marie-Tooth Neuromuscular Disease


Acceleron announced that in the second half of 2017 it plans to initiate a Phase 2 clinical trial of ACE-083, the Company’s locally acting muscle agent, for the treatment of patients with Charcot-Marie-Tooth disease (CMT).

This drug whose purpose is to both reinforce and raise locally the volume of the leg muscle has been already tested in facioscapulohumeral muscular dystrophy (FSHD).

“We are pleased to expand our clinical development program for ACE-083 into a second neuromuscular disease” said Matthew Sherman, Executive Vice President and Chief Medical Officer at Acceleron. “We believe ACE-083 can strengthen the targeted leg muscles in CMT patients and thereby improve their ability to walk and avoid falls”.

ACE-083 works by binding to and inhibiting select proteins in the TGF-beta protein superfamily that negatively regulate (reduce) muscle growth, such as activins and myostatin (GDF8). This is believed to increase muscle mass and strength in the muscle where the drug is administered. Untreated muscles or other organs are not affected, reducing the potential for systemic side effects.

We will run point from that promising clinical study, although some of the aspects need clarification, in particular those relating to the selection of the patients and the type of CMT.

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