Acceleron Pharma Inc. reported positive early data from its midstage trial of ACE-083 in patients with Charcot-Marie-Tooth disease.
CMT is a common inherited neurologic disease characterized by muscle weakness in the lower legs and arms. There is no approved therapy in the U.S. for CMT, which affects an estimated 125,000 people in the U.S.
In the phase 2 dose-escalation study, 18 CMT patients received ACE-083 in 150-milligram, 200-mg, or 240-mg doses.
Acceleron said ACE-083 increased patients' total muscle volume by 12.6% to 14.2%, with increases in viable and functional muscle volume of 15.8% to 19.6%. Treatment also reduced patients' total fat fraction by 1.7% to 3.5%.
Additionally, ACE-083 showed an encouraging safety profile, with the most common adverse events being mild to moderate.
The Cambridge, Mass.-based biopharmaceutical company plans to evaluate ACE-083 against placebo in the trial's second portion, which starts in the third quarter of 2018. Early data from the study, which will treat about 40 CMT patients over six months, is expected by the end of 2019.
ACE-083 is an injectable Acceleron is also developing to treat facioscapulohumeral muscular dystrophy, another genetic disorder with no approved cure. The U.S. Food and Drug Administration already granted ACE-083 fast track and orphan drug status.