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
- Review
- 1
Studies in rodents or other animal models.
Narrative or systematic reviews; no primary data.
Publication years
- 19
- 20
- 21
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.
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.
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.
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.