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Ela3.0
Elamipretide Research
Mostly mechanism / observational
22 peer-reviewed studies
What the evidence says
Mostly mechanism / observational
Most Elamipretide studies are mechanism or observational rather than RCTs that measure a clinical effect — keep findings provisional.
Most evidence is from high-quality meta-analyses and randomised trials published 2016–2026 with a typical study size of 19 participants.
Based on 22 studies · 1 meta-analysis · 14 RCTs · 58 total participants
Confidence
Moderate confidence
By outcome
Mitochondrial disease & myopathy
Mostly mechanism / observational13 studies
Cardiac & reperfusion
Mostly mechanism / observational7 studies
Energy & fatigueA mitochondria-targeted (cardiolipin-binding) peptide that improved subjective fatigue and showed an open-label signal in Barth syndrome; failed pivotal myopathy and infarct-size endpoints. · Not established
Mostly mechanism / observational5 studies
Safety profile
Mostly mechanism / observational3 studies
Vision & eye health
Too few graded studies1 study
Active research area
15 studies in the last 5 years · Latest meta-analysis: 2022
201620212026
1RCT2020
Karaa A, Haas R, Goldstein A, Vockley J, Cohen BH · J Cachexia Sarcopenia Muscle (2020)
In plain English: Furthermore, we offer a comprehensive outlook on the expansive prospects of SS-31's future development and application.
Du X, Zeng Q, Luo Y, He L, Zhao Y, Li N, Han C, Zhang G, Liu W. · Mitochondrion (2024)
Its notable attributes encompass the mitigation of oxidative stress, the suppression of inflammatory processes, the maintenance of mitochondrial dynamics, and the prevention of cellular apoptosis.
As such, SS-31 may emerge as a viable choice for the treatment of mitochondrial dysfunction-related ailments in the foreseeable future.
Furthermore, we offer a comprehensive outlook on the expansive prospects of SS-31's future development and application.
In plain English: A comprehensive understanding of mitochondrial redox processes during gestational diabetes offers unique potential for improving pregnancy outcomes and may contribute to reducing the intergenerational inheritance of metabolic diseases.
Burzynska-Pedziwiatr I, Wozniak LA, Bukowiecka-Matusiak M. · Free radical biology & medicine (2026)
This structure identifies placental mitochondria as potential therapeutic targets.
Preclinical studies on mitochondrial antioxidants (SS-31, MitoQ), uncoupling factors, and biogenesis-supporting substances have shown great potential in restoring mitochondrial integrity and reducing oxidative stress.
However, there is still no clinical confirmation of their effectiveness during pregnancy.
In plain English: Administration of MTP-131 was not associated with a significant reduction in the primary endpoint, infarct size by CK-MB AUC over 72 h, nor with improvement in prespecified MRI, angiographic, electrocardiographic, or clinical outcomes.
Gibson CM, Giugliano RP, Kloner RA, Bode C, Tendera M, Jánosi A, et al. · Eur Heart J (2016)
Multicentre, randomized, double-blind Phase 2a trial of IV MTP-131 (elamipretide) vs placebo during primary PCI for first anterior STEMI
FAILED its primary endpoint: no reduction in infarct size (CK-MB AUC over 72 h)
No improvement in any prespecified imaging, angiographic, ECG, or clinical outcome
Gwaltney C, Shields A, Love E, Ollis S, Stokes J, Mazar I, Arenson E, Aiudi A, Wirth RJ, Houts C. · Orphanet journal of rare diseases (2025)
Results Among the N = 12 white males (M age = 19.5, SD = 7.7) participating in the TAZPOWER trial, overall symptoms were rated as mild (n = 5, 41.7%), moderate (n = 5, 41.7%), severe (n = 1, 8.3%), or very severe (n = 1, 8.3%).
The BTHS-SA may be a useful tool to evaluate treatment benefits in this underserved population.
In plain English: Herein we evaluate the emerging role of PLSCR3 as a potentially druggable mitochondrial target, supported by recent genetic, biochemical, and in vivo evidence, and discuss translational strategies that may bridge the gap between experimental promise and clinical application.
Patel PS, Pabla NS, Bajwa A. · Seminars in nephrology (2026)
This review synthesizes current mitochondrial-directed approaches for AKI, with a particular emphasis on the mechanistic role of PLSCR3 in maintaining mitochondrial homeostasis and injury responses.
Despite encouraging data, mitochondrial therapies face several translational hurdles, including limited bioavailability, challenges in establishing effective dosing regimens, incomplete mechanistic understanding, and variability in efficacy across different experimental models.
Moreover, concerns regarding cost, accessibility, and long-term safety remain unresolved, contributing to inconsistent outcomes in clinical trials.
In plain English: While short-term studies indicate that mitoAOXs are generally well tolerated, there is currently limited evidence to support the use of mitoAOXs in the management of glycaemic control and cardiovascular health.
Mason SA, Wadley GD, Keske MA, Parker L. · Diabetes Obes Metab (2022)
Systematic review and meta-analysis of 19 RCTs (n=884) of mitochondrial-targeted antioxidants — Elamipretide, MitoQ and MitoTEMPO — searched through June 2021
Pooled mitoAOXs significantly improved brachial flow-mediated dilation (3 trials; SMD 1.19, 95% CI 0.28-2.16) but with very-low evidence certainty; no significant effect on any glycaemic, cardiovascular or oxidative-stress outcome
Subcutaneous elamipretide specifically increased mild-to-moderate injection-site events; no serious treatment-emergent adverse events across mitoAOXs
In plain English: For the 6-minute walk test, the least-squares mean difference between elamipretide-treated patients and natural-history controls was 79.7 m (P = 0.0004) at week 64.
Hornby B, Thompson WR, Almuqbil M, Manuel R, Abbruscato A, Carr J, et al. · Orphanet J Rare Dis (2022)
Phase 3 observational, retrospective study comparing 8 TAZPOWER open-label-extension patients with 19 untreated natural-history controls via propensity scoring
Reported significant 6MWT and muscle-strength advantages for elamipretide and increased LV stroke volume
Designed to bolster the open-label efficacy signal because the randomized trial portion was not conclusive