Human evidence record · Elamipretide
MMPOWER-3 Phase III primary mitochondrial myopathy trial
Neither co-primary endpoint was significantly improved versus placebo.
Quick read
Human evidence record
What this study tells us
Participants were assigned to comparison groups by chance, usually with placebo and/or blinding to reduce bias. This is a strong design for testing cause and effect, but the population, duration and endpoints still limit what can be concluded.
Who was studied?
Primary mitochondrial myopathy
How many?
n=218
What happened?
Neither co-primary endpoint was significantly improved versus placebo.
What stops us overclaiming?
Large definitive PMM trial was negative; should override impressions from smaller exploratory signals for this indication.
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Key result
Neither co-primary endpoint was significantly improved versus placebo.
Study details
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