Acceleron’s ACE-083 Therapy for Facioscapulohumeral Muscular Dystrophy (FSHD) Begins Phase 2 Trial

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by Alice Melão |

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muscles, DMD patients, metabolic changes

Acceleron Pharma said the first patient has been dosed in a Phase 2 clinical trial of ACE-083, its proprietary lead candidate drug ACE-083 to treat facioscapulohumeral muscular dystrophy (FSHD) — a genetic disease affecting 19,000 Americans for which no approved therapy yet exists.

“ACE-083 has generated a strong data set in both preclinical and clinical trials to date,” Matthew Sherman, Acceleron’s chief medical officer, said in  a press release. “Our understanding of its novel mechanism of action suggests that ACE-083 may become an important new treatment for FSHD patients with muscle weakness that negatively affects their strength and functional abilities.”

The new drug binds to and inhibits the activity of proteins such as activins and myostatin, which are known to negatively regulate muscle growth. Because of this mechanism of action, ACE-083 is believed to increase muscle mass and muscle strength in sites where it is given. Yet other organs or untreated muscles are not affected, leading to improved safety and reduced toxicity.

Results from a Phase 1 study (NCT02257489) in healthy volunteers showed that ACE-083 is dose-dependent and increases muscle volume by 14.5 percent — the best result reported so far.

For this reason, Acceleron has initiated a two-part placebo-controlled Phase 2 trial (NCT02927080) in FSHD patients with muscle weakness in the tibialis anterior or biceps. The study will determine ACE-083’s safety and tolerability, as well as the best dose for therapeutic use. Acceleron exports to have results of the first part of this trial ready by late 2017.

Later this year, Acceleron will initiate a second Phase 2 clinical trial (NCT03124459) with ACE-083, but this time for treating patients with Charcot-Marie-Tooth disease. This treatment may benefit patients who suffer from foot drop and reduced mobility due to muscle weakness.

“In the first quarter, we made significant progress across our entire pipeline,” Acceleron President and CEO Habib Dable said in a press release, adding that the Cambridge, Massachusetts-based company reported $25.4 million in net losses for the first quarter ending March 31, 2017.