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Research Monograph

SS-31 (Elamipretide / Bendavia / MTP-131)

Also Known AsElamipretide · Bendavia · MTP-131 · D-Arg-Dmt-Lys-Phe-NH2

Molecular Weight639.8 g/mol
Discovered2004
Citations5 peer-reviewed
Research Areas5 domains
Background

Designed by Hazel Szeto and Peter Bhatt at Weill Cornell Medical College as a cell-permeable, mitochondria-targeted tetrapeptide. The alternating aromatic-cationic motif enables selective concentration in the inner mitochondrial membrane at >1000-fold relative to extracellular concentration.

Amino Acid SequenceD-Arg-Dmt-Lys-Phe-NH2
Mechanisms of Action

How SS-31 Works

01

Cardiolipin Interaction

Selectively binds cardiolipin on the inner mitochondrial membrane via electrostatic and hydrophobic interactions. This stabilizes cardiolipin's interaction with cytochrome c, maintaining its electron carrier function and preventing conversion to a peroxidase that generates reactive oxygen species.

cardiolipincytochrome cinner mitochondrial membrane
02

Electron Transport Chain Optimization

By stabilizing the cardiolipin–cytochrome c interaction, SS-31 improves electron transfer efficiency through Complex III and Complex IV, reducing electron leak at Complex I and Complex III and thereby lowering mitochondrial superoxide production at the source.

Complex IComplex IIIComplex IVcytochrome c
03

Mitochondrial Permeability Transition Inhibition

Prevents opening of the mitochondrial permeability transition pore (mPTP) under stress conditions, preserving membrane potential (ΔΨm) and preventing release of cytochrome c and apoptosis-inducing factor into the cytosol.

mPTPΔΨmcytochrome c release
04

Mitochondrial Dynamics

Preclinical evidence suggests modulation of mitochondrial fission/fusion balance by preserving cristae structure and supporting OPA1-mediated fusion, which maintains functional mitochondrial networks under metabolic stress.

OPA1DRP1cristae structure
Evidence-Graded Research Areas

Published Research

Strong
Moderate
Emerging
Preclinical

Cardiac Ischemia-Reperfusion Injury

Strong

Phase I/II clinical trials (EMBRACE STEMI, NCT01572909) demonstrated reduced infarct size when administered during percutaneous coronary intervention. Preclinical models show preserved left ventricular function and reduced myocardial fibrosis following ischemia-reperfusion. Mechanisms include prevention of cardiolipin peroxidation and mPTP opening.

Barth Syndrome (TAFAZZIN Deficiency)

Moderate

Phase II TAZPOWER trial (NCT03098797) in Barth syndrome patients showed improvements in six-minute walk test distance and cardiac stroke volume. Barth syndrome features pathological cardiolipin remodeling due to TAFAZZIN mutations, making it a direct target for SS-31's mechanism of action.

Age-Related Mitochondrial Dysfunction

Moderate

Preclinical studies in aged mice demonstrate reversal of age-related decline in skeletal muscle mitochondrial energetics, improved exercise tolerance, and restoration of redox balance. SS-31 treatment in aged animals restores Complex I-linked and Complex II-linked respiration to levels approaching young controls.

Renal Ischemia-Reperfusion

Preclinical

Animal models of acute kidney injury show protection of mitochondrial structure and function, reduced tubular necrosis, and preservation of GFR when SS-31 is administered before or shortly after ischemic insult.

Neurodegenerative Disease Models

Preclinical

Preclinical data in MPTP-induced parkinsonism and amyloid-beta toxicity models show protection of mitochondrial function and reduced neuronal death. The ability to cross the blood-brain barrier and concentrate in neuronal mitochondria supports further investigation.

Safety Profile

SS-31 has been administered in multiple Phase I and Phase II clinical trials with a generally favorable safety profile. The most commonly reported adverse events include injection site reactions and transient changes in taste perception. No dose-limiting toxicities were observed in the EMBRACE STEMI trial at doses up to 0.25 mg/kg/hr. Preclinical toxicology studies showed no mutagenicity or organ toxicity at tested doses.

Handling & Storage

Reconstitute with bacteriostatic water. SS-31 is a small, stable tetrapeptide with good aqueous solubility. Store lyophilized powder at -20°C. Reconstituted solution should be stored at 2-8°C and used within 28 days. Protect from prolonged light exposure.

References & Citations

Peer-Reviewed Literature

  1. 1

    First-in-class cardiolipin-protective compound as a therapeutic agent to restore mitochondrial bioenergetics

    Szeto HH.

    British Journal of Pharmacology, 2014PubMed 24641310DOI
  2. 2

    The mitochondrial-targeted compound SS-31 re-energizes ischemic mitochondria by interacting with cardiolipin

    Birk AV, Liu S, Soong Y, et al.

    Journal of the American Society of Nephrology, 2013PubMed 23813219DOI
  3. 3

    Mitochondrial-targeted peptide rapidly improves mitochondrial energetics and skeletal muscle performance in aged mice

    Siegel MP, Kruse SE, Percival JM, et al.

    Aging Cell, 2013PubMed 23530891DOI
  4. 4

    EMBRACE STEMI study: a Phase 2a trial to evaluate the safety, tolerability, and efficacy of intravenous MTP-131 on reperfusion injury in patients undergoing primary percutaneous coronary intervention

    Gibson CM, Giugliano RP, Kloner RA, et al.

    European Heart Journal, 2016PubMed 26273011DOI
  5. 5

    A phase 2/3 randomized clinical trial followed by an open-label extension to evaluate the effectiveness of elamipretide in Barth syndrome, a genetic disorder of mitochondrial cardiolipin metabolism

    Reid Thompson W, Hornby B, Manuel R, et al.

    Genetics in Medicine, 2021PubMed 33495528DOI
Frequently Asked Questions

SS-31 FAQ

What is SS-31?

SS-31 (also known as Elamipretide or Bendavia) is a synthetic mitochondria-targeted tetrapeptide with the sequence D-Arg-Dmt-Lys-Phe-NH2. It selectively concentrates in the inner mitochondrial membrane where it binds cardiolipin, stabilizing electron transport chain function and reducing oxidative stress at the source.

How does SS-31 target mitochondria?

SS-31 uses an alternating aromatic-cationic motif (positively charged and aromatic residues) that exploits the large negative membrane potential across the inner mitochondrial membrane. This drives its accumulation >1000-fold inside mitochondria without requiring any active transport or targeting sequence.

Has SS-31 been studied in human clinical trials?

Yes. SS-31 (as elamipretide) has been evaluated in multiple clinical trials including EMBRACE STEMI (cardiac ischemia-reperfusion), TAZPOWER (Barth syndrome), and studies in primary mitochondrial myopathy and age-related macular degeneration. Results have shown generally favorable safety and preliminary efficacy signals.

How should SS-31 be stored?

Lyophilized SS-31 should be stored at -20°C for long-term stability. Reconstituted solution should be kept at 2-8°C and used within 28 days. Protect from prolonged exposure to light.

Available for Research

SS-31 Products

All compounds 99%+ purity, verified by Janoshik Analytical. GMP-manufactured lyophilized powder.

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Disclaimer: This monograph is provided for educational and research purposes only. G26x Peptides products are sold exclusively as research chemicals. They are not intended for human consumption, therapeutic use, or as dietary supplements. All research should be conducted in compliance with applicable laws and institutional review board protocols. Information presented here is sourced from published peer-reviewed literature and does not constitute medical advice.