SS-31 (Elamipretide)
A synthetic tetrapeptide targeting cardiolipin on the inner mitochondrial membrane; in clinical trials for mitochondrial disease.
Overview
SS-31, known clinically as elamipretide, is a synthetic aromatic-cationic tetrapeptide developed in the Szeto-Schiller program to target the inner mitochondrial membrane. It is among the most extensively studied mitochondrial peptides, with a large body of preclinical work and ongoing clinical trials in mitochondrial diseases such as Barth syndrome.
Mechanism
SS-31 binds cardiolipin on the inner mitochondrial membrane, stabilizing the interaction between cardiolipin and cytochrome c and supporting normal cristae structure and electron-transport efficiency. The result is reduced mitochondrial reactive oxygen species and improved ATP production under stress. It acts by optimizing membrane organization rather than by functioning as a classical antioxidant scavenger.
Dosing
Clinical protocols have used daily subcutaneous injection of elamipretide, and animal-model research uses species-specific parenteral dosing. There is no approved indication, and all doses are protocol-defined.
- Daily subcutaneous administration in clinical trials
- Animal models use systemic injection over days to weeks
- Ongoing trials target specific mitochondrial conditions
Expected Effects
- Improved mitochondrial respiration and reduced oxidative stress in preclinical models
- Cardiac and skeletal-muscle functional improvements in animal disease models
- Early clinical signals reported in cardiomyopathy trials in Barth syndrome
Side Effects & Tolerability
In clinical trials elamipretide has been generally well tolerated, with injection-site reactions among the more common events. Longer-term safety continues to be evaluated in the ongoing clinical program.
Storage
Lyophilized peptide should be stored cold and desiccated, protected from light and moisture. Reconstituted solutions are typically aliquoted and refrigerated or frozen, avoiding repeated freeze-thaw cycles.