MMPOWER-3 in primary mitochondrial myopathy
Neurology / PMC
TL;DR
In the 218-person MMPOWER-3 phase 3 trial, elamipretide was well tolerated but did not improve walking distance or fatigue versus placebo in primary mitochondrial myopathy.
Phase 3 · randomized · double-blind · placebo-controlled · 218 participants
What was studied
The question.
Whether daily elamipretide could improve exercise capacity and fatigue in people with genetically confirmed primary mitochondrial myopathy.
How it was conducted
The study design.
Participants were randomized 1:1 to 40 mg subcutaneous elamipretide or placebo daily for 24 weeks. The two primary endpoints were the six-minute walk test and the Primary Mitochondrial Myopathy Symptom Assessment fatigue score.
What it found
Did it actually work?
- The trial did not meet either primary efficacy endpoint.
- Walking distance and fatigue outcomes were not significantly different from placebo at 24 weeks.
- Elamipretide was generally well tolerated, with most adverse events mild to moderate.
Why it matters
How to read this result.
It is strong human evidence showing that mitochondrial-targeted biology does not automatically translate into broad clinical benefit across every mitochondrial disorder. It also helps define where elamipretide evidence is strongest and where it is not.
Keep in mind
What the study does not prove.
- Primary mitochondrial myopathy is genetically heterogeneous.
- These results are not the same question as the later Barth syndrome indication for which Forzinity received accelerated approval.
Original source
Want to read the original?
We summarize the study here first. The original source is there when you want the full methods, statistics, tables and author discussion.
Open EU trial results