ACE-083 Trials Show no Benefit

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. 😔

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

This site uses Akismet to reduce spam. Learn how your comment data is processed.

%d bloggers like this: