CMT Blog

Acceleron Pharma Inc.’s ACE-083 drug trials are over.

According to a company press release, the drug “did not achieve statistically significant improvements in functional endpoints relative to placebo” with CMT patients, so it will no longer be developed.

Jacob Bell at BioPharma Dive takes a deeper look. 🤿

According to researchers Barry Miller et al., ACE-083 is “a locally acting follistatin‐based therapeutic that binds myostatin and other muscle regulators.” Studies showed it to increase muscle mass and force in neuromuscular disease mouse models.” (PubMed)

Unfortunately, it does not work on people well enough to improve quality of life for CMT patients and others experiencing muscle atrophy. While the drug does increase muscle volume, it doesn’t increase it much. 😔