Drug Type: Protein Biologic
Conditions: ALS, Duchenne muscular dystrophy
Mechanism Type: Improving muscle function
Mechanism: ACE-031 is a monoclonal antibody that binds and inhibits myostatin. It acts as a decoy receptor for myostatin and other proteins that limit muscle growth. Myostatin blockage has been considered as a therapeutic approach for ALS by improving muscle size and strength.
U.S. Status for ALS: Discontinued
 Myostatin inhibition slows muscle atrophy in rodent models of amyotrophic lateral sclerosis. Holzbaur, EL et al. Neurobiol Dis. 2006 Sep;23(3):697-707.
 Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy. ClinicalTrials.gov, 31 Jan 2013. Accessed 11 Mar 2016 from https://clinicaltrials.gov/ct2/show/NCT01099761.
 UPDATE: ACE-031 clinical trials in Duchenne MD Wahl, M. Quest Magazine Online, 2 May 2013. Accessed 9 Mar 2016 from http://quest.mda.org/news/update-ace-031-clinical-trials-duchenne-md.
Last updated March 11th, 2016