Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial
Karaa A, Bertini E, Carelli V, et al. (MMPOWER-3 Trial Investigators) (2023). Neurology.
Summary
A phase 3, randomised, double-blind, placebo-controlled trial of 24 weeks of daily subcutaneous elamipretide in 218 adults with primary mitochondrial myopathy. The trial did not meet either primary endpoint: the difference versus placebo in six-minute walk distance was about -3 metres and the difference in the fatigue score was not significant. Elamipretide was well tolerated, with mostly mild to moderate adverse events. The authors state this provides Class I evidence that the drug does not improve walking distance or fatigue at 24 weeks in this population. Several authors report payments from the sponsor.