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Elamipretide (SS-31)

Mitochondria-targeting tetrapeptide

Also known as SS-31, MTP-131, Bendavia

A cardiolipin-binding peptide with real phase 3 programmes and a mixed record. Genuinely contested rather than merely unproven.

Guide updated Sep 2, 2026Literature checked Sep 2, 20261 curated study39 literature recordsBaseline editorial

Overview

In shortA small peptide that collects inside mitochondria, your cells' power plants. It has been through more real clinical development than almost anything else here outside the approved drugs. The record is mixed in an instructive way: some trials missed their main target while showing signals on secondary ones, which is the pattern that generates the longest arguments.

Elamipretide is a small peptide that accumulates in the inner mitochondrial membrane and binds cardiolipin. It has been through more real clinical development than almost anything else in this catalogue outside the approved drugs, across primary mitochondrial myopathy, Barth syndrome, dry age-related macular degeneration and heart failure.

The record is mixed in an instructive way. Some trials missed primary endpoints while showing signals on secondary ones, which is the pattern that generates the longest arguments and the least resolution.

Mechanism

In shortThere is a fat molecule found only in the inner membrane of mitochondria that is essential to how they generate energy, and it is easily damaged. This peptide binds it and is proposed to hold the structure together, making energy production more efficient and cutting damaging by-products. The mechanism is unusually well worked out for a compound at this stage.

Cardiolipin is a phospholipid unique to the inner mitochondrial membrane and essential to the structure of the electron transport chain supercomplexes. It is prone to peroxidation, and damaged cardiolipin degrades mitochondrial efficiency and promotes cytochrome c release.

Elamipretide binds cardiolipin and is proposed to stabilise cristae architecture, improve electron transport efficiency and reduce reactive oxygen species production. The mechanism is unusually well characterised for a compound at this stage.

Evidence

In shortContested. A late-stage trial in a mitochondrial muscle disease missed its main target. Work in a rarer condition where the reasoning is strongest has been more encouraging, in very small numbers. The honest reading is that a well-understood mechanism has so far produced inconsistent results, which is the ordinary history of pharmacology rather than a scandal.

Contested. A phase 3 trial in primary mitochondrial myopathy did not meet its primary endpoint. Work in Barth syndrome, an ultra-rare cardiolipin disorder where the mechanistic rationale is strongest, has produced more encouraging results in very small numbers.

The honest reading is that a well-characterised mechanism has so far produced inconsistent clinical results, which is the ordinary history of pharmacology rather than a scandal. Whether the failures reflect the wrong population, the wrong endpoint or no effect is not resolved by the published data.

Safety

In shortTrial use has generally been tolerated, with injection site reactions the most common effect. Because the trials are small and the populations specific, rare events are poorly characterised. There is no long-term safety data in healthy people, and no reason to assume something targeting mitochondria behaves the same in healthy ones as in damaged ones.

Trial exposure has generally been tolerated, with injection site reactions the most common effect. Because the trials are small and the populations specific, rare events are poorly characterised.

There is no long-term safety data in healthy people, and no reason to assume a mitochondrial-targeting agent behaves the same in functioning mitochondria as in dysfunctional ones.

Regulatory status

In shortInvestigational, with submissions made for rare conditions and a complicated review history. Not approved for general use. Material sold under the name SS-31 outside a trial is a research chemical.

Investigational. Regulatory submissions have been made for rare indications with a complicated review history. Not approved for general use. Material sold under the name SS-31 outside a trial is a research chemical.

The literature

1 study indexed. Findings and limitations get equal weight, on purpose.

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Everything above is free to read. What an account opens is the evidence underneath it: 1 study with their limitations, and the regimen each trial actually reported.

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The wider literature

18 indexed papers and 21 registered trials mentioning Elamipretide, straight from PubMed and ClinicalTrials.gov. 21 of 21 administer it; the rest measure it. These have not been read by us. Unlike the studies above, they carry no findings and no limitations written by anyone here, because writing either would mean claiming a reading nobody has done. They are a starting point for yours.

Registered trials (21)

  1. An Intermediate Size Expanded Access Protocol of Elamipretide

    Stealth BioTherapeutics Inc.NCT04689360

    expanded_accessavailable

  2. Clinical Trial in Patients With Barth Syndrome- 4TAZPower

    Stealth BioTherapeutics Inc.2026NCT07531251

    interventionalphase4recruitingn=48

  3. Study of Healthy Aging and Physical Function With Elamipretide

    David Marcinek2025NCT07275424

    interventionalphase2recruitingn=30

  4. ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD)

    Stealth BioTherapeutics Inc.2024NCT06373731

    interventionalphase3active not recruitingn=313

  5. Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD)

    Stealth BioTherapeutics Inc.2022NCT05162768

    interventionalphase3completedn=102

  6. FRDA Investigator Initiated Study (IIS) With Elamipretide

    Children's Hospital of Philadelphia2022NCT05168774

    interventionalphase1phase2completedn=20

  7. ReCLAIM-2 Study to Evaluate Safety,Efficacy & Pharmacokinetics of Elamipretide in Subjects With AMD With Non-central GA

    Stealth BioTherapeutics Inc.2019NCT03891875

    interventionalphase2completedn=176

  8. A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

    Stealth BioTherapeutics Inc.2017NCT03098797

    interventionalphase2phase3completedn=12

  9. A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension

    Stealth BioTherapeutics Inc.2017NCT03323749

    interventionalphase3terminatedn=218

  10. Safety, Tolerability, Efficacy of MTP-131 for Treatment of Mitochondrial Disease in Subjects From the MMPOWER Study

    Stealth BioTherapeutics Inc.2016NCT02805790

    interventionalphase2completedn=30

  11. A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy

    Stealth BioTherapeutics Inc.2016NCT02693119

    interventionalphase2completedn=12

  12. Effect of Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Reduced Ejection Fraction

    Stealth BioTherapeutics Inc.2016NCT02788747

    interventionalphase2completedn=71

  13. A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED)

    Stealth BioTherapeutics Inc.2016NCT02653391

    interventionalphase1phase2completedn=22

  14. A Phase 2 Study to Evaluate the Cardiac and Renal Effects of Short Term Treatment With Elamipretide in Patients Hospitalized With Congestion Due to Heart Failure

    Stealth BioTherapeutics Inc.2016NCT02914665

    interventionalphase2completedn=308

  15. A Study to Evaluate the Effects of 4 Weeks Treatment With Subcutaneous Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Preserved Ejection Fraction

    Stealth BioTherapeutics Inc.2016NCT02814097

    interventionalphase2completedn=46

  16. Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM)

    Stealth BioTherapeutics Inc.2016NCT02976038

    interventionalphase2terminatedn=28

  17. An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration

    Stealth BioTherapeutics Inc.2016NCT02848313

    interventionalphase1completedn=40

  18. Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy

    Stealth BioTherapeutics Inc.2015NCT02367014

    interventionalphase1phase2completedn=36

  19. A Phase 2 Study to Evaluate the Impact of MTP-131 (Bendavia™) on Skeletal Muscle Function in Elderly

    Stealth BioTherapeutics Inc.2015NCT02245620

    interventionalphase2completedn=41

  20. Evaluation of Myocardial Effects of MTP-131 for Reducing Reperfusion Injury in Patients With Acute Coronary Events

    Stealth BioTherapeutics Inc.2012NCT01572909

    interventionalphase2completedn=300

  21. Effects of Intravenous Bendavia™ on Reperfusion Injury in Patients Undergoing Angioplasty of the Renal Artery

    Stealth BioTherapeutics Inc.2012NCT01755858

    interventionalphase1phase2terminatedn=16

Papers (18)

  1. Elamipretide Hydrochloride.

    American journal of health-system pharmacy : AJHP : official journal of the American Society of Health-System Pharmacists2026PMID 41206662

    journal article

  2. Elamipretide: First Approval.

    Shirley MDrugs2026PMID 41335372

    journal articlereview

  3. Safety and Efficacy of Approved and Unapproved Peptide Therapies for Musculoskeletal Injuries and Athletic Performance.

    Mendias CL, et al.Sports medicine (Auckland, N.Z.)2026PMID 41966639

    journal articlereview

  4. Beyond the injection: delivery systems reshaping retinal disease management.

    Rowe LW, et al.Expert opinion on pharmacotherapy2025PMID 40319468

    journal articlereview

  5. SS-31@Fer-1 Alleviates ferroptosis in hypoxia/reoxygenation cardiomyocytes via mitochondrial targeting.

    Zheng H, et al.Biomedicine & pharmacotherapy = Biomedecine & pharmacotherapie2025PMID 39848110

    journal article

  6. Elamipretide in the Management of Barth Syndrome: Current Evidence and a Case Report.

    Jacob N, et al.Molecular genetics and metabolism2025PMID 40816230

    journal articlecase reportsreview

  7. Elamipretide: A Review of Its Structure, Mechanism of Action, and Therapeutic Potential.

    Tung C, et al.International journal of molecular sciences2025PMID 39940712

    journal articlereview

  8. YTHDF1-mediated mitochondrial dysfunction and allergic airway inflammation by interaction with β-catenin/TCF4 signaling.

    Xie C, et al.International immunopharmacology2025PMID 40633209

    journal article

  9. Mitochondrial Dysfunction: The Silent Catalyst of Kidney Disease Progression.

    Pavlović N, et al.Cells2025PMID 40497970

    journal articlereviewresearch support, non-u.s. gov't

  10. Advances in Management of Mitochondrial Myopathies.

    Bangeas A, et al.International journal of molecular sciences2025PMID 40508218

    journal articlereview

  11. Telomere length in offspring is determined by mitochondrial-nuclear communication at fertilization.

    Winstanley YE, et al.Nature communications2025PMID 40087268

    journal article

  12. ALCAT1-mediated abnormal cardiolipin remodelling promotes mitochondrial injury in podocytes in diabetic kidney disease.

    Hao Y, et al.Cell communication and signaling : CCS2024PMID 38200543

    video-audio mediajournal articleresearch support, non-u.s. gov't

  13. Podocyte senescence: from molecular mechanisms to therapeutics.

    Zhao Q, et al.Renal failure2024PMID 39248407

    journal articlereview

  14. Cardiomyopathy in Duchenne Muscular Dystrophy and the Potential for Mitochondrial Therapeutics to Improve Treatment Response.

    Gandhi S, et al.Cells2024PMID 39056750

    journal articlereviewresearch support, non-u.s. gov't

  15. Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial.

    Karaa A, et al.Neurology2023PMID 37268435

    randomized controlled trialclinical trial, phase iiijournal articleresearch support, non-u.s. gov't

  16. Potential Therapeutic Candidates for Age-Related Macular Degeneration (AMD).

    Nashine SCells2021PMID 34572131

    journal articlereview

  17. Elamipretide (SS-31) improves mitochondrial dysfunction, synaptic and memory impairment induced by lipopolysaccharide in mice.

    Zhao W, et al.Journal of neuroinflammation2019PMID 31747905

    journal article

  18. Mitochondria-targeted therapies for acute kidney injury.

    Tábara LC, et al.Expert reviews in molecular medicine2014PMID 25104110

    journal articleresearch support, non-u.s. gov'treview

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For Research & Educational Discussion Only. Not Medical Advice

This guide is educational reference material compiled from published literature. It is not medical advice, not a recommendation, and not a substitute for a clinician who knows your history. Nothing here should be used to make a decision about your own health without one.

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