SS-31

Overview

SS-31 (elamipretide) is a mitochondria-targeted tetrapeptide that concentrates in the inner mitochondrial membrane to improve energy production and reduce oxidative stress. It is in clinical trials for mitochondrial myopathy, heart failure, and age-related mitochondrial dysfunction.

Mechanism of Action

SS-31 selectively binds to cardiolipin in the inner mitochondrial membrane, stabilizing the electron transport chain and preventing cytochrome c peroxidase activity. This improves mitochondrial ATP production, reduces reactive oxygen species (ROS) generation, and prevents mitochondria-mediated apoptosis.

Research Summary

Phase 2 and 3 clinical trials are ongoing for Barth syndrome, primary mitochondrial myopathy, and heart failure with reduced ejection fraction. The TAZPOWER trial showed improvements in cardiac function. The compound received Fast Track designation from the FDA for Barth syndrome. Results in heart failure have been mixed.

Dosing Information

Reported Side Effects

• Injection site reactions
• Headache
• Nausea
• Diarrhea

Research References

• Elamipretide (SS-31) for mitochondrial diseases: a randomized, double-blind, placebo-controlled trial (2021)
• SS-31 peptide targets the inner mitochondrial membrane and inhibits oxidative stress (2004)
• Cardiolipin-targeting peptide SS-31 improves mitochondrial function and cardiac performance (2014)