Acceleron Pharma's ACE-083 Fails In Phase 2 Study For FSHD

(RTTNews) - Acceleron Pharma Inc. (XLRN) on Monday said its investigational therapy ACE-083 for the treatment of facioscapulohumeral muscular dystrophy (FSHD) did not achieve functional secondary endpoints in the Phase 2 trial.

Although ACE-083 showed a significant increase in mean total muscle volume, which was the primary endpoint of the trial, the secondary endpoint of functional tests was failed. As a result, the company is not planning to continue with further study of ACE-083 for FSHD.

FSHD is a genetic muscle disorder that starts usually before the age of 20. It first affects the muscles of the face, shoulder blades and upper arms by weakening them, and gradually spreads to other muscles.

ACE-083 is also being explored as a potential treatment for Charcot-Marie-Tooth disease—a neuromuscular disorder, and a phase II trial in this indication is underway. Topline results from this trial are expected in the first quarter of the next year.

XLRN closed Monday's trading at $44.65, up 1.71%. In after-hours, the stock was down 6.83% to $41.60.