SS-31

Clinical trial dosing: 40 mg/day subcutaneous injection (Barth syndrome TAZPOWER trial). Heart failure trials tested 4 mg and 40 mg daily subcutaneous. IV infusion studied at 0.01-0.25 mg/kg/hour over 4 hours. Well-tolerated across a wide dose range. Research context dosing typically ranges from 0.5-5 mg/day subcutaneous. Injection site reactions are the most common side effect.

Selectively binds to cardiolipin, a phospholipid found exclusively in the inner mitochondrial membrane. This stabilizes mitochondrial cristae structure and improves membrane integrity, optimizing the organization of electron transport chain complexes.

By stabilizing cardiolipin-dependent supercomplex assembly, enhances electron transport chain efficiency and oxidative phosphorylation (ATP production), reducing electron leak.

Reduces mitochondrial ROS production by improving electron transport efficiency and preventing electron leak at complexes I and III, rather than acting as a direct free radical scavenger.

Electrostatically attracted to the highly negative potential of the inner mitochondrial membrane due to its positive charges, concentrating at the site of action and restoring membrane potential in dysfunctional mitochondria.

TAZPOWER trial showed significant improvements in 6-minute walk test (+95.9 m) and symptom scores at 36 weeks. FDA advisory committee endorsed efficacy in 2024. FDA approved for Barth syndrome treatment in 2025.

Restores mitochondrial bioenergetics in cells with mitochondrial dysfunction, improving ATP production and reducing oxidative stress across multiple tissue types.

Studied in heart failure trials (phase 2). Improves cardiac mitochondrial morphology and defective mitophagy. Tested at 4 mg and 40 mg daily subcutaneous doses in heart failure patients.

Optimizes ATP production by improving electron transport chain efficiency, potentially benefiting age-related mitochondrial decline and exercise performance.

Preclinical studies suggest neuroprotective effects through mitochondrial stabilization in neuronal cells, with potential applications in neurodegenerative diseases.

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