Acceleron: First patient treated in Phase 2 ACE-083 trial
Acceleron Pharma announced that the first patient has been treated in a Phase 2 clinical trial of ACE-083, the company's locally acting muscle agent, for the treatment of patients with facioscapulohumeral muscular dystrophy. "We are excited to start our first Phase 2 trial for ACE-083," said Matthew Sherman, M.D., Chief Medical Officer of Acceleron. "ACE-083 has generated a strong data set in both preclinical and clinical trials to date. Our understanding of its novel mechanism of action suggests that ACE-083 may become an important new treatment for FSHD patients with muscle weakness that negatively affects their strength and functional abilities." The two-part Phase 2 clinical trial is designed to evaluate ACE-083 in FSHD patients with muscle weakness in either the tibialis anterior, a muscle in the lower leg involved in foot flexion, or the biceps. Part 1 is an open-label, dose-escalation study of ACE-083 administered by intramuscular injection once every three weeks in up to 36 patients to evaluate safety and increases in muscle volume over a three-month treatment period. Part 2 is a randomized, double-blind, placebo-controlled study using the optimal dose level selected in Part 1. Up to 40 patients will be randomized to receive either placebo or ACE-083 administered by intramuscular injection once every three weeks and evaluated for increases in muscle volume, strength, function and safety over a three-month treatment period.