Preliminary results from the ongoing phase 2 clinical trial of ACE-083 in FSHD patients were presented today at the American Academy of Neurology 70th Annual Meeting in Los Angeles, California.The oral presentation by Jeffery Statland, MD, of the University of Kansas Medical Center, included preliminary findings from cohorts 1 and 2 in the open-label, dose escalation Part 1 of the trial.
The presentation was selected for the “Best of” Session in clinical trial updates in neuromuscular disorders. This designation was given to only 24 abstracts out of some 3,000!
Developed by Cambridge-based biotech Acceleron Pharma, ACE-083 consists of a modified form of human follistatin that binds GDF8 (myostatin) plus other molecules that suppress skeletal muscle growth. The drug, which is injected directly into affected muscles, is intended to help FSHD muscles grow by removing a natural “brake system” that keeps normal muscles from excessive growth. In FSHD, muscles are struggling to overcome the disease process, and the idea is that by taking off the “brakes”, they would be able to regenerate better. (See related story.)
The trial results appear to bear out this prediction, with increases in muscle volume of 15-20 percent reported at the higher dose (200 mg) and a decline of the fat-to-muscle ratio especially in the tibialis anterior (shin muscle). As muscle degenerates, it is replaced by fat, so a lowering of the fat-to-muscle ratio indicates that the proportion of muscle relative to fat had increased after treatment.
ACE-083 is currently being evaluated in two Phase 2 trials: one in FSHD and one in Charcot-Marie-Tooth (CMT) disease. The final Part 1 results from both Phase 2 trials are expected in the second of half of 2018.
The randomized, double-blind, placebo controlled Part 2 of the FSHD study has been initiated, with results expected in the second half of 2019. Part 2 of the CMT trial is expected to be initiated by the end of 2018.
Reference: Preliminary Results from a Phase 2 Study to Evaluate ACE-083, a Local Muscle Therapeutic, in Patients with Facioscapulohumeral Muscular Dystrophy. 2018 American Academy of Neurology . April 21-27, 2018.
Download the full presentation here.
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