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.
Frequent intravitreal injections create a significant treatment burden due to limited drug half-life and chronic disease nature.; Emerging approaches include intravitreal sustained-release platforms and alternative delivery routes like suprachoroidal, subretinal, topical, and subcutaneous.; Specific innovations include the ranibizumab port delivery system, EYP-1901, KSI-301, OTX-TKI, and encapsulated cell therapy.; Challenges include overcoming the blood-retinal barrier, surgical complications, and ensuring patient adherence.
Elamipretide's mechanism of action involves binding to cardiolipin, modulating mitochondrial membrane electrostatic potentials, and assembling cardiolipin-dependent proteins.; Elamipretide improves mitochondrial function, structure, and bioenergetics in animal models of cardiac and skeletal muscle myopathies and ocular pathologies.; Recent clinical trials have been completed for Barth syndrome, primary mitochondrial myopathy, and age-related macular degeneration, with elamipretide showing continued promise as a potential therapy.
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 214 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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