Elamipretide (SS-31) is a first-in-class mitochondria-targeted tetrapeptide that binds to cardiolipin in the inner mitochondrial membrane. It was granted FDA Breakthrough Therapy designation for Barth syndrome and is the first cardiolipin-directed therapeutic to reach late-stage clinical trials. It stabilizes mitochondrial structure and improves ATP production.
Mechanism of Action
Selectively accumulates in mitochondria via its dimethyltyrosine residue and binds to cardiolipin, a phospholipid essential for inner mitochondrial membrane integrity. This binding prevents cardiolipin peroxidation, stabilizes respiratory supercomplexes, maintains cristae structure, and improves electron transport chain efficiency — ultimately enhancing ATP synthesis and reducing reactive oxygen species production.
Benefits
First cardiolipin-directed mitochondrial therapeutic in clinical development[1]
Improves exercise capacity and muscle function in primary mitochondrial myopathy[1]
FDA Breakthrough Therapy designation for Barth syndrome[1]
Potential applications in age-related mitochondrial dysfunction and heart failure[1]
Not medical advice - research-reported information only
This content is for informational purposes only and does not constitute medical advice.
SS-31 (Elamipretide) - Dosing in Published Research
Reported routes: Subcutaneous injection
Investigational dosing in trials: 40-80 mg SC daily. Phase 3 trials ongoing for Barth syndrome and primary mitochondrial myopathy. Not commercially available outside clinical trials as of 2026.
The dosing information above is sourced from published research literature and clinical trials. These are not recommendations. Individual responses vary. Always consult a healthcare provider before considering any peptide-based therapy.
Elamipretide did not meet primary endpoint in MMPOWER-3 for mitochondrial myopathy, but post-hoc analyses suggested benefit in specific genetic subgroups and biomarkers
References
1.Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.. Neurology, 2023. "Elamipretide did not meet primary endpoint in MMPOWER-3 for mitochondrial myopathy, but post-hoc analyses suggested benefit in specific genetic subgroups and biomarkers" [PMID: 37268435]
Recommended Resources
The Peptide Protocols Handbook
Evidence-based reference guide covering mechanisms, research, and clinical applications.
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SS-31 (Elamipretide) FAQ
Medical Disclaimer
This content is for informational and educational purposes only and does not constitute medical advice, diagnosis, or treatment recommendations.
Always consult a qualified healthcare provider before starting, stopping, or modifying any treatment. Do not disregard professional medical advice based on information found on this site.
No claims of therapeutic efficacy are made for substances that are not FDA-approved for the discussed indications. Research citations reflect published findings and do not imply endorsement.