SS-31 (Elamipretide)
Also referenced as: Elamipretide, MTP-131, Bendavia
A cardiolipin-targeting mitochondrial peptide that has reached genuine clinical trials, with mixed outcomes.
- Evidence grade
- CEarly Human Research
Early Human Research
- Regulatory status
- Clinical Trials
- Human data
- Yes
Registered clinical trials exist
- Last reviewed
- September 1, 2026
Overview
SS-31, developed as elamipretide, concentrates in the inner mitochondrial membrane where it interacts with cardiolipin. Unlike most compounds in this library, it has been studied in properly registered clinical trials.
Results have been mixed. Some trials in primary mitochondrial myopathy and Barth syndrome reported signals on secondary endpoints while missing primary ones — a pattern worth understanding rather than glossing over.
Why It's Being Studied
- Primary mitochondrial myopathy and rare mitochondrial disease.
- Barth syndrome and cardiolipin-related cardiomyopathy.
- Heart failure, ischaemia-reperfusion injury and dry age-related macular degeneration.
Mechanism of Action
It concentrates inside mitochondria and stabilises a key membrane fat, helping the energy-producing machinery work more normally.
Elamipretide is a cell-permeable tetrapeptide (D-Arg-dimethylTyr-Lys-Phe) that accumulates in the inner mitochondrial membrane through electrostatic and hydrophobic interaction with cardiolipin. It is proposed to stabilise cardiolipin-cytochrome c interaction, preserving cristae architecture, electron transport efficiency and reducing reactive oxygen species production.
Research Areas & Routes Studied
- Mitochondrial myopathy
- Cardiomyopathy
- Heart failure
- Retinal disease
- Subcutaneous injection (clinical trials)
- Intravenous (clinical trials)
- Topical ophthalmic (trials)
Evidence Summary
Multiple registered human trials, including randomised studies in mitochondrial and cardiac disease, with mixed results on primary endpoints and no approval at last review.
Human clinical evidence
- Randomised trials in primary mitochondrial myopathy reported inconsistent results on six-minute walk distance and patient-reported outcomes.
- Barth syndrome research reported functional signals in small populations.
Clinical trials
- Multiple registered trials across cardiac, ophthalmic and mitochondrial indications.
Animal studies
- Extensive ischaemia-reperfusion and heart failure model data.
Reported Adverse Events in Research
- Injection-site reactions are the most commonly reported effect in trials.
- Gastrointestinal effects and headache reported.
Limitations of Current Evidence
- Primary endpoints have not been consistently met.
- Rare-disease populations are small, limiting statistical power.
Documented Research Protocols
Elamipretide (SS-31) — clinical trial protocols as registered
Summarise the administration structure used in registered trials in primary mitochondrial myopathy and related conditions.
View documented protocol
Why It Is Used
- Longevity
Energy and Mitochondrial Function
Fatigue that sleep does not fix is the entry point. Mitochondrial compounds are the most biologically interesting and least resolved category on this site.
Read the case study
Research References
- Randomised controlled trial
Elamipretide in primary mitochondrial myopathy: randomised trial
- Main finding
- Mixed results across functional and patient-reported endpoints.
- Regulatory document
Registered elamipretide trials