Study record · Elamipretide

MMPOWER-3 Phase III primary mitochondrial myopathy trial

Neither co-primary endpoint was significantly improved versus placebo.

Key result

Neither co-primary endpoint was significantly improved versus placebo.

Study designRandomized double-blind placebo-controlled trial

PhasePhase III

PopulationPrimary mitochondrial myopathy

Sample size218

RouteSubcutaneous

Regimen / exposure40 mg once daily

Duration24 weeks

ComparatorPlacebo

Primary endpoint / question6-minute walk distance and Primary Mitochondrial Myopathy Symptom Assessment fatigue

Safety resultInjection-site reactions were common; no evidence of efficacy on co-primary endpoints.

Key limitationsLarge definitive PMM trial was negative; should override impressions from smaller exploratory signals for this indication.

Registry / identifierPMID 37268435 / NCT03323749