Acceleron Pharma, a clinical stage biopharmaceutical company located in Cambridge MA, has initiated a Phase 2 clinical study of ACE-083, an investigational drug, in patients with Charcot-Marie-Tooth (CMT) disease. The Phase 2 clinical study is designed to evaluate ACE-083, a locally acting protein therapeutic, in up to 42 adult CMT patients with muscle weakness affecting ankle dorsiflexion (the ability to raise the foot at the ankle).

ACE-083 is an investigational drug that is based on a naturally-occurring protein and works to enhance the body’s own promoters of muscle growth. ACE-083 can be customized to patients’ needs by administering the drug into target muscle(s) with the aim of increasing muscle size and strength. Acceleron is developing ACE-083 for neuromuscular diseases in which patients experience weakness and atrophy in specific muscles, such as CMT patients with foot drop.

Acceleron has studied ACE-083 in both animals and healthy human subjects prior to initiating this Phase 2 study in CMT. In disease-specific animal studies, including a CMT mouse model, ACE-083 increased muscle mass and force in the specific muscles targeted. In a Phase 1 clinical study in healthy volunteers, ACE-083 treatment resulted in significant increases in muscle size, with an average increase of 8.9% in the tibialis anterior (lower leg) and 14.5% in the rectus femoris (thigh) muscle volume at the highest dose levels. No serious side effects or treatment-limiting toxicities were observed in the study.

The Acceleron study in CMT will assess whether ACE-083 can safely increase muscle size and strength of the tibialis anterior muscle. The study is designed to determine whether ACE-083 can improve ankle dorsiflexion strength and alleviate foot drop, which in turn may improve measures of function and quality of life in these patients. This study is actively enrolling and will be conducted across 10 sites in the United States.

Learn more about the Phase 2 study in CMT here: