Acceleron begins dosing in Phase II trial of ACE-083 to treat CMT


Acceleron Pharma has started dosing patients in a Phase II clinical trial of its therapeutic candidate ACE-083 for the treatment of Charcot-Marie-Tooth disease (CMT) patients.

CMT is a neurological disorder characterised by muscle weakness, and ACE-083 is a locally acting muscle agent based on follistatin protein being developed to selectively enhance growth and strength in target muscles.

The two-part Phase II trial will assess the therapeutic candidate in patients with weakness in a lower leg muscle called tibialis anterior (TA) that is involved in raising the foot at the ankle.

Acceleron executive vice-president and chief medical officer Matthew Sherman said: “We designed our clinical development strategy for ACE-083 to explore its activity in diseases with weakness in specific muscles due to an underlying neurological or muscle disorder.

"We designed our clinical development strategy for ACE-083 to explore its activity in diseases with weakness in specific muscles due to an underlying neurological or muscle disorder."

“With Phase II trials now underway in both CMT and facioscapulohumeral muscular dystrophy, we will be able to evaluate ACE-083’s effect on both muscle strength and function across a range of neuromuscular diseases.”

In the first open-label, dose-escalation part of the trial, safety and increases in muscle volume will be assessed in approximately 18 patients, following administration of ACE-083 injection into the TA muscle once every three weeks over a period of three months.

The trial’s second randomised, double-blind, placebo-controlled part will evaluate an optimal dose level selected in the first part in up to 24 subjects over three months.

During this part, patients will be monitored for muscle volume, strength, function and safety.