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SS-31 (Elamipretide)
Research Use
Also known as Bendavia, MTP-131
Function
Primary
Mitochondrial dysfunction · Primary mitochondrial myopathy (orphan)
Secondary
Heart failure · Dry AMD
Mechanism
Aromatic-cationic tetrapeptide that selectively binds cardiolipin on the inner mitochondrial membrane — restores cristae structure, ATP synthesis, and reduces ROS.
Evidence
Clinical RCT (Limited)
Research
Phase 3 trials in Barth syndrome and primary mitochondrial myopathy
Dosing
Range
40 mg/day (SQ, trials)
Frequency
Daily
Route
Subcutaneous
Cycling
Continuous daily dosing in trials.
Benefits
- ·Improved mitochondrial bioenergetics
- ·Restored cristae morphology
- ·Improved 6-min walk in some trials
Risks
- ·Injection-site reactions
- ·Mixed late-stage trial results
- ·Unknown chronic safety
Contraindications
- ·Pregnancy
- ·Known hypersensitivity
Regulatory
Investigational (Stealth BioTherapeutics) — orphan & fast-track designations
Functional Groups
Cited Works
- Szeto HH. Cardiolipin-targeted peptide SS-31 (Elamipretide). Br J Pharmacol. 2014;171:2029.source