Elamipretide Review 2025 - PMC
/PMC/2025
Why It Matters
This caught my attention because mitochondrial dysfunction shows up everywhere — fatigue, muscle weakness, cognitive decline. If elamipretide actually improves mitochondrial efficiency in humans, it could address root causes rather than symptoms. That said, clinical data remains mixed: some trials showed benefits in Barth syndrome and primary mitochondrial disease, but heart failure trials have been disappointing. Not ready for prime time, but worth watching.
Key Findings
- Elamipretide binds cardiolipin with high affinity, stabilizing the inner mitochondrial membrane and improving cristae structure where ATP production occurs
- In Barth syndrome patients (genetic cardiolipin deficiency), treatment improved 6-minute walk distance by 55 meters at 12 weeks — clinically meaningful for this rare disease
- Phase 3 trial in heart failure with preserved ejection fraction (LVEF >40%) failed primary endpoint, showing no improvement in 6-minute walk distance despite earlier promising biomarker data
- Preclinical models showed 30-50% reductions in reactive oxygen species and improved respiratory chain efficiency, but translating this to functional human outcomes has proven difficult
- Current clinical development focuses on rare mitochondrial diseases and specific neuromuscular conditions where mitochondrial defects are well-defined, not general 'anti-aging' use
Read the Paper↗PMC11816484