Acceleron Pharma Investigates ACE-083 as Treatment for CMT Patients

Acceleron Pharma has launched a Phase 2 clinical trial (NCT03124459) to investigate the safety and effectiveness of its product ACE-083 as a potential treatment for Charcot-Marie-Tooth disease (CMT). The first patient already has been dosed, the company announced.

“People diagnosed with CMT currently have no drug therapy options to address the major consequences of their disease, such as impaired walking and falls due to progressive muscle weakness in the lower leg,” Colin Quinn, MD, said in a press release. “ACE-083 has the potential to increase muscle growth and strength in the lower leg muscles we are targeting, and could improve patients’ ability to walk,” said Quinn, a researcher involved in the multi-center trial.

The Phase 2 study will enroll CMT patients with muscle weakness in the tibialis anterior (TA), a muscle in the lower leg that helps the foot raise at the ankle. U.S. trial sites, currently recruiting patients, include the University of Kansas Medical Center Neurology Department, the Hackensack (N.J.) University Medical Center and the University of Pennsylvania.

The study will have two parts. The first part will involve 18 patients, to whom researchers will administer ACE-083 injections into the TA muscle (once every three weeks) to assess muscle volume increase and the drug’s safety for three months.

The second part will include 24 patients who will be assigned to receive ACE-083 or a placebo. Researchers will assess treatment’s effectiveness through changes in muscle volume, strength and function, as well as the drug’s safety, for a period of three months.

“We are proud to have advanced ACE-083 into a second Phase 2 clinical trial,” said Matthew Sherman, MD, and Acceleron’s executive vice president and chief medical officer. “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 2 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,” he said.

ACE-083 is an investigational drug based on a natural protein, called follistatin, whose action contributes to muscle growth and strength.

CMT is a hereditary neurological disease that affects the peripheral motor and sensory nerves motor of the body. Motor nerves control muscle contractions, movement and other activities involving muscles, such as speaking, breathing and swallowing. Symptoms include muscle weakness, abnormal gait and foot deformities.