Question 1

What is SS-31?

Accepted Answer

SS-31 (elamipretide, Bendavia, MTP-131) is a mitochondria-targeted tetrapeptide developed at Weill Cornell Medical College that selectively concentrates in the inner mitochondrial membrane, binding to cardiolipin — a unique phospholipid essential for electron transport chain complex organization and function. By stabilizing cardiolipin-protein interactions, SS-31 restores efficient mitochondrial electron transport, reduces ROS production, and improves ATP synthesis. SS-31 has undergone extensive clinical trials for heart failure (EMBRACE trial), mitochondrial myopathy (MMPOWER trials), and Barth syndrome. Its ability to directly target mitochondrial dysfunction addresses a fundamental mechanism underlying aging, neurodegeneration, cardiomyopathy, and metabolic disease.

Question 2

How does SS-31 work?

Accepted Answer

SS-31's alternating aromatic-cationic motif (Arg-Dmt-Lys-Phe) enables selective accumulation in the inner mitochondrial membrane (1000-5000 fold concentration over cytoplasm). It binds cardiolipin, stabilizing its interactions with cytochrome c and electron transport chain complexes (I, III, IV). This optimizes electron flow, reduces electron leak and superoxide generation, maintains mitochondrial membrane potential, and enhances ATP production efficiency.

Question 3

What are the research applications of SS-31?

Accepted Answer

Heart Failure

EMBRACE-STEMI trial showed reduced infarct size. PROGRESS-HF studied for chronic heart failure. Mixed clinical results but consistent improvement in mitochondrial biomarkers.

Mitochondrial Myopathy

MMPOWER trials for primary mitochondrial myopathy showed improvements in 6-minute walk test.

Barth Syndrome

Targets the cardiolipin deficiency central to this genetic mitochondrial disease.

Age-Related Diseases

Mitochondrial dysfunction underlies many aging pathologies — SS-31 addresses this root cause.

Question 4

What are the biological pathways affected by SS-31?

Accepted Answer

Cardiolipin stabilization in inner mitochondrial membrane. Electron transport chain optimization. Reduced ROS from Complex I and III. Maintained mitochondrial membrane potential (ΔΨm). Enhanced ATP synthase (Complex V) activity.

Question 5

What are the potential side effects of SS-31?

Accepted Answer

Generally well-tolerated in clinical trials. Injection site reactions. Transient headache. No serious adverse effects in Phase 1-3 studies. Long-term safety data limited.

Question 6

How should SS-31 be stored?

Accepted Answer

Supplied as lyophilized powder or solution for injection. Store at -20°C (powder) or 2-8°C (solution). The D-Arg modification provides protease resistance. Good aqueous solubility. Stable at physiological pH.

Question 7

What is the regulatory status of SS-31?

Accepted Answer

Investigational. Multiple Phase 2/3 trials completed (Stealth BioTherapeutics). Orphan drug designation for Barth syndrome. Not yet FDA-approved. Not WADA-prohibited.

Question 8

Where can I find research studies on SS-31?

Accepted Answer

Scientific studies on SS-31 can be found in peer-reviewed journals through databases like PubMed and Google Scholar.

Question 9

What quality standards should SS-31 meet for research?

Accepted Answer

Research-grade SS-31 should meet high purity standards, typically >95% purity, with proper analytical certification and storage requirements.

SS-31