SS-31 (Elamipretide)
clinical trialsAlso known as: Elamipretide, MTP-131, Bendavia
**Mechanism of Action** SS-31 (Elamipretide) is a mitochondria-targeting tetrapeptide (D-Arg-dimethylTyr-Lys-Phe-NH₂) that selectively binds to cardiolipin, a phospholipid enriched in the inner mitochondrial membrane. This interaction stabilizes the cristae structure, optimizes electron transport chain supercomplex assembly, and restores ATP synthesis under oxidative stress. By reducing mitochondrial reactive oxygen species (ROS) production and preventing cytochrome c release, SS-31 mitigates apoptosis and preserves cellular bioenergetics. **Key Research Findings** Preclinical studies demonstrate SS-31 improves mitochondrial function in models of ischemia-reperfusion injury, heart failure, and neurodegenerative diseases. Phase II clinical trials (e.g., in primary mitochondrial myopathy and acute kidney injury) showed trends toward improved exercise tolerance and reduced cardiac remodeling, though primary endpoints were not uniformly met. A Phase III trial (MM-01) in Barth syndrome reported significant improvements in cardiac function and quality of life. Safety profiles are favorable, with mild injection-site reactions as the most common adverse event. **Clinical Relevance** SS-31 is under investigation for conditions with mitochondrial dysfunction, including Barth syndrome, heart failure, and age-related sarcopenia. Its ability to target cardiolipin offers a novel therapeutic approach distinct from general antioxidants. Ongoing trials aim to confirm efficacy in broader patient populations and optimize dosing regimens. For research purposes only — not medical advice.
Key data
C32H49N9O5Research & studies
Elamipretide is a mitochondrial cardiolipin binder developed by Stealth BioTherapeutics.; Approved in the USA in September 2025 for improving muscle strength in Barth syndrome patients weighing ≥30 kg.; First disease-specific treatment for Barth syndrome, an ultra-rare X-linked recessive disorder.; Also in phase III development for dry age-related macular degeneration and mitochondrial myopathies.
SS-31@Fer-1 improved H/R-induced cardiomyocyte viability and reduced LDH and CK-MB levels.; It maintained cellular iron homeostasis by increasing FTH, FTMT, FPN, and ABCB8 expression.; The compound enhanced mitochondrial integrity and function while reducing oxidative stress markers.; SS-31@Fer-1 suppressed ferroptosis via upregulation of GPX4 and Nrf2 and downregulation of ACSL4 and PTGS2.
Elamipretide has a unique structure enabling selective mitochondrial targeting and uptake in various cell types.; It binds cardiolipin in the inner mitochondrial membrane, stabilizing cristae structure and improving bioenergetics.; Preclinical studies show efficacy in models of heart failure, neurodegeneration, ischemia-reperfusion injury, and muscle disorders.; Clinical trials (e.g., PROGRESS-HF, TAZPOWER) highlight significant therapeutic potential, warranting further research on long-term safety and efficacy.
AREDS/AREDS2 supplementation slows disease progression but cannot reverse retinal damage.; Molecularly targeted therapies (Pegcetacoplan, Avacincaptad Pegol) reduce inflammation.; Gene therapy (HMR59) protects RPE cells, and mitochondria-targeted drugs (SS-31) mitigate oxidative stress.; AI aids in predicting disease progression and personalizing treatment.
Frequently asked questions
What is SS-31 (Elamipretide)?
**Mechanism of Action** SS-31 (Elamipretide) is a mitochondria-targeting tetrapeptide (D-Arg-dimethylTyr-Lys-Phe-NH₂) that selectively binds to cardiolipin, a phospholipid enriched in the inner mitochondrial membrane. This interaction stabilizes the cristae structure, optimizes electron transport chain supercomplex ass
How does SS-31 (Elamipretide) work?
Mitochondria-targeting tetrapeptide that binds cardiolipin on the inner membrane to stabilize cristae and restore ATP production.
What is the research status of SS-31 (Elamipretide)?
SS-31 (Elamipretide) is currently classified as clinical trials, with 213 research references on record. This is for research purposes only and is not medical advice.
What is the molecular weight of SS-31 (Elamipretide)?
SS-31 (Elamipretide) has a molecular weight of approximately 639.8 g/mol (formula C32H49N9O5).
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