Longevity & mitochondria

Elamipretide

Peptilex evidence grade ABCDEUpdated 19 Oct 2026 How grades work

Type: Mitochondria-targeted tetrapeptide
Main use or focus: Barth syndrome (US, accelerated approval)
Also known as: Forzinity, SS-31

Key studies from the book

10 patients; six-minute walking distance up by 96.1 meters.
168-week extension – Genetics in Medicine, 2024

All key studies, risks and open questions are in the book.

Research at a glance

Publications by typePubMed, all years

All papers439

Human studies189

Animal studies only155

Randomized trials17

Meta-analyses & systematic reviews2

Source: PubMed and ClinicalTrials.gov via the Peptilex study database

Registered trials by phaseClinicalTrials.gov · name in study title

ongoing / startingcompleted / other

Phase 11

Phase 29

Phase 34

Other1

Source: PubMed and ClinicalTrials.gov via the Peptilex study database

Trial starts per yearClinicalTrials.gov · by start date (planned starts included)

72016

2018

2020

22022

12024

2026

Source: PubMed and ClinicalTrials.gov via the Peptilex study database

Go deeper

Full profile in the book

What it is, how it acts, the key studies, risks and open questions.

Peptilex – The Peptide Encyclopedia

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An evidence grade (A–E) and the key studies for every peptide
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Registered trials

ClinicalTrials.gov · name in study title · ongoing first

Study	Phase	Status	Participants	Start
ReNEW:Phase 3 Study of Efficacy, Safety & Pharmacokinetics of Subcutaneous Injections of Elamipretide in Subjects With Dry Age-Related Macular Degeneration (Dry AMD) NCT06373731Stealth BioTherapeutics Inc.	Phase 3	Running	313	May 2024
Study of Healthy Aging and Physical Function With Elamipretide NCT07275424David Marcinek	Phase 2	Recruiting	30	Nov 2025
Study to Evaluate Efficacy and Safety of Elamipretide in Subjects With Primary Mitochondrial Disease From Nuclear DNA Mutations (nPMD) NCT05162768Stealth BioTherapeutics Inc.	Phase 3	Completed	102	Apr 2022
A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome NCT03098797Stealth BioTherapeutics Inc.	Phase 2/3	Completed	12	Jul 2017
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 NCT02914665Stealth BioTherapeutics Inc.	Phase 2	Completed	308	Oct 2016
ReCLAIM-2 Study to Evaluate Safety,Efficacy & Pharmacokinetics of Elamipretide in Subjects With AMD With Non-central GA NCT03891875Stealth BioTherapeutics Inc.	Phase 2	Completed	176	Mar 2019
Effect of Elamipretide on Left Ventricular Function in Subjects With Stable Heart Failure With Reduced Ejection Fraction NCT02788747Stealth BioTherapeutics Inc.	Phase 2	Completed	71	Jun 2016
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 NCT02814097Stealth BioTherapeutics Inc.	Phase 2	Completed	46	Sep 2016
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for the Treatment of Fuchs' Corneal Endothelial Dystrophy (FCED) NCT02653391Stealth BioTherapeutics Inc.	Phase 1/2	Completed	22	Feb 2016
FRDA Investigator Initiated Study (IIS) With Elamipretide NCT05168774Children's Hospital of Philadelphia	Phase 1/2	Completed	20	Mar 2022

Show 4 moreShow fewer

Study	Phase	Status	Participants	Start
A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy NCT02693119Stealth BioTherapeutics Inc.	Phase 2	Completed	12	Mar 2016
An Open-Label, Phase 1 Clinical Study to Evaluate the Safety and Tolerability of Subcutaneous Elamipretide in Subjects With Intermediate Age-Related Macular Degeneration NCT02848313Stealth BioTherapeutics Inc.	Phase 1	Completed	40	Oct 2016
A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension NCT03323749Stealth BioTherapeutics Inc.	Phase 3	Stopped early	218	Oct 2017
Open-Label Extension Trial to Characterize the Long-term Safety and Tolerability of Elamipretide in Subjects With Genetically Confirmed Primary Mitochondrial Myopathy (PMM) NCT02976038Stealth BioTherapeutics Inc.	Phase 2	Stopped early	28	Dec 2016

Showing 14 of 15 matching registered studies. See all on ClinicalTrials.gov.

Latest key papers

PubMed · newest first

2026
Therapeutic targeting of mitochondrial dysfunction in heart failure: a systematic review & meta-analysis of clinical outcomes
Meta-analysisMohammed O et al. · Frontiers in Cardiovascular MedicinePMID 42440741
2025
Initial Psychometric Evaluation of the Barth Syndrome Symptom Assessment (BTHS-SA) for Adolescents and Adults in a Phase 2 Clinical Study
Human RCTPhase 2Gwaltney C et al. · Orphanet Journal of Rare DiseasesPMID 40281531
2024
Genotype-specific effects of elamipretide in patients with primary mitochondrial myopathy: a post hoc analysis of the MMPOWER-3 trial
Human RCTPhase 3Karaa A et al. · Orphanet Journal of Rare DiseasesPMID 39574155
2024
Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy: Results From SEQUOIA-HCM
Human RCTPhase 3Sherrod CF 4th et al. · Journal of the American College of CardiologyPMID 39217569
2024
Long-term efficacy and safety of elamipretide in patients with Barth syndrome: 168-week open-label extension results of TAZPOWER
Human RCTThompson WR et al. · Genetics in MedicinePMID 38602181
2024
Elamipretide Topical Ophthalmic Solution for the Treatment of Subjects with Leber Hereditary Optic Neuropathy: A Randomized Trial
Human RCTPhase 2Karanjia R et al. · OphthalmologyPMID 37923251
2023
Efficacy and Safety of Elamipretide in Individuals With Primary Mitochondrial Myopathy: The MMPOWER-3 Randomized Clinical Trial
Human RCTPhase 3Karaa A et al. · NeurologyPMID 37268435
2023
Identifying responders to elamipretide in Barth syndrome: Hierarchical clustering for time series data
Human RCTVan den Eynde J et al. · Orphanet Journal of Rare DiseasesPMID 37041653

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