January 15, 2018
Acceleron Pharma announced positive preliminary results for the first two cohorts in Part 1 of the Phase II clinical trial with ACE-083 in patients with facioscapulohumeral dystrophy (FSHD), a rare genetic muscle disorder that results in progressive focal muscle loss and weakness. The company plans to initiate Part 2 of the ACE-083 FSHD Phase II trial during the second quarter of 2018. Part 1 is an open-label, dose-escalation study of ACE-083 designed to evaluate safety as well as changes in total muscle volume in up to 36 patients with FSHD. Preliminary results include data from 23 patients evaluable for magnetic resonance imaging (MRI) among two different cohorts (11 patients with tibialis anterior weakness and 12 patients with biceps brachii weakness). Each patient received ACE-083 (150mg or 200mg) as a unilateral intramuscular injection once every three weeks for 12 weeks. Total muscle volume changes were measured by MRI relative to baseline at three weeks after the last injection of ACE-083. Based on overlap in dosing on a milligram per gram muscle analysis, dose cohorts were pooled for the analyses of each muscle. Strength and function tests are being explored in Part 1 to assist with the design of the randomized, double-blind, placebo-controlled Part 2 of the study.