Acceleron Announces Topline Results from the Phase 2 Trial of ACE-083 in Patients with Charcot-Marie-Tooth Disease.
The two-part Phase 2 clinical trial was designed to evaluate ACE-083 in CMT patients with muscle weakness in the tibialis anterior (TA), a muscle in the lower leg involved in ankle dorsiflexion (raising the foot at the ankle). A total of 44 patients were randomized and treated with either placebo or ACE-083, and were evaluated for changes in muscle volume, fat fraction, strength, function, quality of life, and safety over a 6-month primary treatment period, followed by a 6-month open-label treatment period.
ACE-083 demonstrated a statistically significant increase in mean total muscle volume, the trial’s primary endpoint. However, the increase did not translate to statistically significant improvements in any of the functional or quality of life secondary endpoints when compared to placebo. As a result, Acceleron is discontinuing development of ACE-083.