P Palthera
Signalling Peptides

SS-31

Elamipretide / MTP-131 / Bendavia

SS-31 (elamipretide, MTP-131) is a mitochondria-targeted synthetic developed by Szeto and Schiller. It concentrates in the inner mitochondrial membrane and interacts with cardiolipin to support electron transport and reduce reactive oxygen species. It has been studied in multiple late-phase clinical trials (Barth syndrome, primary mitochondrial myopathy, age-related macular degeneration, heart failure) but is not FDA-approved at the time of writing.

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Signalling Peptides
Classification
Mitochondria-targeted tetrapeptide (Szeto-Schiller-31 / elamipretide)
Research stage
Multiple Phase II–III clinical trials (Barth syndrome, primary mitochondrial myopathy, dry AMD, heart failure); not FDA-approved at time of writing
Sequence
D-Arg-2',6'-dimethyltyrosine-Lys-Phe-NH2
Molecular weight
639.8 Da

Snapshot

Key takeaways

A three-bullet snapshot before reading the full dossier.

  1. 01

    Mitochondria-targeted that accumulates in the inner mitochondrial membrane via interaction with cardiolipin.

  2. 02

    Researched across Barth syndrome, primary mitochondrial myopathy, dry AMD, and heart failure clinical programmes.

  3. 03

    Investigational — not FDA-approved at the time of writing.

Dossier overview

4

research areas

3

references

3

handling notes

01

Mechanism of action

Elamipretide binds cardiolipin in the inner mitochondrial membrane, supporting cristae structure, electron-transport chain efficiency, and reduced reactive oxygen species generation. Downstream effects in research models include improved ATP production and reduced mitochondrial dysfunction markers.

02

Research applications

  • Mitochondrial disease research (Barth syndrome, mitochondrial myopathy)
  • Age-related macular degeneration clinical research
  • Heart failure with reduced ejection fraction clinical research
  • Neurodegenerative and oxidative stress models

Evidence at a glance

What's behind this profile

3 citations · 2019–2021

Animal
2

Studies in rodents or other animal models.

Review
1

Narrative or systematic reviews; no primary data.

Publication years

  1. 19
  2. 20
  3. 21
20192021

Counts are derived from the cited studies below. A study covering both in vivo and in vitro work is counted by its primary model. Sample size is reported in 0 of 3 citations. Findings remain model-specific and are not extrapolated to therapeutic use.

03

Study references

Each profile cites a minimum of two peer-reviewed sources, with model type and reported sample size where the source provides it. Findings are model-specific and must not be extrapolated to therapeutic use.

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

2019

Zhao W et al. · Journal of Neuroinflammation

Model
In vivo — mouse model of LPS-induced cognitive impairment
Sample
Not reported in abstract

SS-31 was associated with reversal of LPS-induced learning and memory deficits via mitochondrial-dysfunction reduction and enhanced BDNF signalling in hippocampus.

PMID 31747905 DOI 10.1186/s12974-019-1627-9

Mitochondrial targeted therapy with elamipretide (MTP-131) as an adjunct to TNF inhibition for traumatic optic neuropathy

2020

Tse BC et al. · Experimental Eye Research

Model
In vivo — mouse sonication-induced traumatic optic neuropathy model
Sample
Not reported in abstract

MTP-131 combined with etanercept was associated with improved retinal ganglion cell survival following optic-nerve trauma, without synergistic effects.

PMID 32758490 DOI 10.1016/j.exer.2020.108178

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

2021

Nashine S · Cells

Model
Narrative review of AMD therapeutic candidates
Sample
N/A (review)

Reviewed SS-31 / elamipretide among therapeutic candidates targeting mitochondrial dysfunction and oxidative stress in AMD clinical research.

PMID 34572131 DOI 10.3390/cells10092483

Evidence caveats

  • Investigational compound. Multiple Phase II/III trials have produced mixed primary-endpoint results (e.g. dry AMD ReCLAIM-2 missed primary endpoint).
  • Published primary mechanism work is concentrated in preclinical models; the clinical evidence base is still consolidating.

04

Storage and handling

Trial material follows the clinical trial protocol cold-chain. Research-grade material must be stored under controlled laboratory conditions per protocol.

  • Trial material follows the clinical trial protocol cold-chain.
  • Research-grade material requires controlled laboratory storage and batch traceability.
  • Investigational — not for unsupervised use.