A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension
Stealth BioTherapeutics Inc.218 participants9 Oct 2017 to 10 Feb 2020
Primary Mitochondrial Myopathy
In PepBank now:147 peptides, 6 blends and 13 other compounds97,577 study results from 17,383 studies8,473 clinical trialsUpdated
Comparing 2 records · Data version 308560
Swipe sideways to see every peptide.
| Peptide | Synthetic analog Elamipretide (SS-31) | ||
|---|---|---|---|
| Identity | |||
| Kind | Synthetic analog | Small molecule | |
| Area | Longevity and mitochondria | Longevity and mitochondria | |
| Evidence | |||
| Amount of evidence | 1,784 | 1,513 (plus 2 on natural levels, listed separately) | |
| Evidence level | Has results from studies in people | Has results from studies in people | |
| Safety | |||
| Safety results | 35 (29 in people) | 64 (35 in people) | |
| Status | |||
| Approval | |||
| Sport (WADA) | |||
| Registered trials | 30 · 3 active | 19 · 8 active14 more mention it but do not test it | |
Shared outcomes
From the regulatory data of each peptide.
The FDA label for Forzinity says: “To improve muscle strength in adult and pediatric patients with Barth syndrome weighing at least 30 kg” No central EU authorisation (EMA).
Outcome groups with study results for at least two of these peptides. Counts include only results from studies that gave the peptide. Bar length is the number of results, on one scale. The bars show how much was studied, not how large an effect was. A peptide with more studies can look stronger here without being stronger. For results, see the randomized results above.
| Outcome group | Elamipretide (SS-31) | NMN (nicotinamide mononucleotide) |
|---|---|---|
| Mitochondrial functionWhole body | 4795258216 |
PepBank is a research reference. Talk to a doctor before you use any peptide.
Its most advanced trial is in phase 2. The FDA, the EMA and the other sources we use show no approval (as of 21 Oct 2026).
| Receptor activity and mechanismRespiratory | 883850 | 2138135655 |
|---|
| Inflammation markersWhole body | 17312548 | 131391361 |
|---|
| NeuroprotectionNervous system and cognition | 17814335 | 1028715 |
|---|
| Heart functionCardiovascular | 120208614 | 561523 |
|---|
| Adverse eventsWhole body | 292621 | 4328132 |
|---|
| Exercise and physical functionWhole body | 6220357 | 33258 |
|---|
| Kidney functionKidney and urinary | 130611410 | 454365 |
|---|
| Sex hormones and fertilityReproductive and sexual health | 37928 | 16513530 |
|---|
| Vision and retinaEye | 412714 | 835 |
|---|
| Oxidative stressSkin and wound | 914645 | 372611 |
|---|
| PharmacokineticsPharmacology | 8233 | 32248 |
|---|
For each outcome that two or more peptides have results for: the best result of a single randomized trial, on the same measure (the same kind of number, the same unit). When a trial has several dose arms, the cell shows the highest dose arm. The trials differ in people, length and dose, so a gap between two cells is not proof that one peptide works better.
No outcome has a randomized-trial result on the same measure for two or more of these peptides yet.
The other peptides have no randomized result on the same measure, so these results cannot be set side by side.
| Outcome | Elamipretide (SS-31) | NMN (nicotinamide mononucleotide) |
|---|---|---|
| Exercise and physical functionMusculoskeletalCompared as: difference from placebo, in m | −3.2 m 6MWT least squares mean difference Dose studied: 40 mg/d24 weeksn = 2182023 | No comparable result 20 randomized-trial results measure it in another way or another unit. |
| Vision and retinaEyeCompared as: difference from placebo, in % | −43% Reduction in progression of total EZ loss · Shown as a change. The study reports a reduction in progression of total ez loss of this size. Dose studied: 40 mg daily48 weeksn = 1762025 | |
| Kidney functionKidney and urinaryCompared as: value in the arm that got the peptide, in % | −6% Change in fractional hypoxia Placebo: 47% Dose studied: 0.05 mg/kg per hourbefore and during PTRAn = 142017 | |
| Adverse eventsEyeCompared as: value in the arm that got the peptide, in % | 86% Adverse event rate Placebo: 71% Dose studied: 40 mg daily48 weeksn = 1762025 |
−3.2 m
6MWT least squares mean difference
Dose studied: 40 mg/d24 weeksn = 2182023
No comparable result
20 randomized-trial results measure it in another way or another unit.
No comparable result 2 randomized-trial results measure it in another way or another unit. |
No comparable result
3 randomized-trial results measure it in another way or another unit.
No comparable result 15 randomized-trial results measure it in another way or another unit. |
−43%
Reduction in progression of total EZ loss · Shown as a change. The study reports a reduction in progression of total ez loss of this size.
Dose studied: 40 mg daily48 weeksn = 1762025
No comparable result
2 randomized-trial results measure it in another way or another unit.
−6%
Change in fractional hypoxia
Placebo: 47%
Dose studied: 0.05 mg/kg per hourbefore and during PTRAn = 142017
No comparable result
3 randomized-trial results measure it in another way or another unit.
86%
Adverse event rate
Placebo: 71%
Dose studied: 40 mg daily48 weeksn = 1762025
No comparable result
15 randomized-trial results measure it in another way or another unit.
Registered trials, ranked by phase (late phases first), then posted results and enrollment. Status is the registry status. Study results come from published studies. Results posted at ClinicalTrials.gov are shown on each trial page.
Top 3 of 30 trials, ranked by phase, then posted results and enrollment: 1 ongoing · 1 completed · 1 stopped early · 2 with posted results · source ClinicalTrials.gov (opens in a new tab)
Stealth BioTherapeutics Inc.218 participants9 Oct 2017 to 10 Feb 2020
Primary Mitochondrial Myopathy
Stealth BioTherapeutics Inc.12 participants5 Jul 2017 to 11 Oct 2021
Barth Syndrome
Stealth BioTherapeutics Inc.313 participants30 May 2024 to est. Sept 2027
Age Related Macular Degeneration (ARMD)
Top 3 of 19 trials, ranked by phase, then posted results and enrollment: 1 ongoing · 1 completed · source ClinicalTrials.gov (opens in a new tab)
First Affiliated Hospital, Sun Yat-Sen University20 participants1 Jun 2021 to est. 31 Jul 2022
Hypertension
Christopher Kai Shun Leung138 participants19 May 2025 to est. 31 Dec 2026
Glaucoma
Zhejiang Chinese Medical University22 participants1 May 2024 to 6 Apr 2025
Binge Drinking · Liver Injury · Nutritional Supplementation · Hepatic Steatosis
14 more mention it but do not test it. They only measure it or name it in their text.