Acceleron Pharma Inc, a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases, today 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), one of the most common inherited neurologic diseases leading to focal muscle weakness.
“We are pleased to expand our clinical development program for ACE-083 into a second neuromuscular disease,” said Matthew Sherman,XLRN_CMT_Webinar_FINAL_4.3.2017 M.D., 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. We look forward to hosting the educational webinar during which we will describe CMT and our enthusiasm for the potential of ACE-083 to address the substantial unmet medical needs of patients.”
CMT Phase 2 Trial Design
The two-part Phase 2 clinical trial is designed to evaluate ACE-083 in CMT patients with muscle weakness in the tibialis anterior (TA), a muscle in the lower leg involved in foot dorsiflexion (raising the foot at the ankle). Part 1 is an open-label, dose-escalation phase of the study, with ACE-083 administered by injection into the TA muscle once every 3 weeks in up to 18 patients to evaluate safety and increases in muscle volume over a 3-month treatment period. Part 2 is a randomized, double-blind, placebo-controlled phase using the optimal dose level selected in Part 1. A total of 24 patients will be randomized in Part 2 to receive either placebo or ACE-083 and will be evaluated for increases in muscle volume, strength, function and safety over a 3-month treatment period.
Read Acceleron’s Webinar Presentation here
ACE-083 is a therapeutic candidate that acts as a ligand trap for members in the transforming growth factor-beta (TGF-β) superfamily involved in the regulation of muscle mass and strength. ACE-083 has been designed to increase muscle mass and strength selectively in the muscles into which the drug is administered. Acceleron is developing ACE-083 for diseases such as facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth disease in which improved muscle strength in target muscles may provide a clinical benefit.
Acceleron is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of innovative therapeutics to treat serious and rare diseases. Its pioneering research platform leverages the powerful biology behind the body’s ability to rebuild and repair its own cells and tissues. This approach to drug discovery has generated four therapeutic candidates that are currently in clinical trials.