InvestigationalHuman evidence

Elamipretide (SS-31)

Also referred to as SS-31, MTP-131, Bendavia

A genuinely investigational compound with registered clinical trials and a development history that includes missed primary endpoints. A useful case study in conflicting evidence.

Molecule class: Cardiolipin-binding tetrapeptide
Category: Mitochondrial
Last evidence review: 2026-08-23

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Human studies

Animal / in vitro

Graded claims

—

Participants

Mechanism

What is being studied

Binds cardiolipin in the inner mitochondrial membrane, proposed to stabilise cristae architecture and improve electron-transport efficiency.

Evidence

Claims, graded individually

Each statement below carries its own evidence level, its own confidence rating, and its own account of what remains unestablished. A compound does not have a single grade.

Mitochondrial Research

XInsufficient / conflictingLow confidenceHuman evidence

Elamipretide has been studied in registered trials in mitochondrial myopathy and heart failure, with inconsistent results across programmes.

What is not established

Several programmes did not meet primary endpoints. This is a conflicting-evidence record, not a positive one.

Supporting sources (1)

Randomised controlled trialHumanIdentifier pending verification

Elamipretide (SS-31) in mitochondrial disease and heart-failure programmes

Multiple · Various cardiology and neurology journals · 2021

Population: Adults with primary mitochondrial myopathy or heart failure, by programme
Endpoint: Six-minute walk distance, fatigue scales, cardiac measures by programme
Route: Subcutaneous
Quantity as reported: Reported in registered trial protocols
Frequency: Daily in most programmes
Duration: 24 weeks

Result. Results across programmes have been mixed. Several endpoints did not reach statistical significance, and development history includes missed primary endpoints.

Adverse events. Injection-site reactions were common in the trials.

Limitations. This is an important example of conflicting evidence: positive early signals were not consistently confirmed in larger trials.

Search PubMed for this literature →

Last evidence review: 2026-08-23

Human evidence

1 human study catalogued

Human and non-human evidence are kept strictly separate. They are never pooled, averaged, or presented as a single body of support.

Randomised controlled trialHumanIdentifier pending verification

Elamipretide (SS-31) in mitochondrial disease and heart-failure programmes

Multiple · Various cardiology and neurology journals · 2021

Population: Adults with primary mitochondrial myopathy or heart failure, by programme
Endpoint: Six-minute walk distance, fatigue scales, cardiac measures by programme
Route: Subcutaneous
Quantity as reported: Reported in registered trial protocols
Frequency: Daily in most programmes
Duration: 24 weeks

Result. Results across programmes have been mixed. Several endpoints did not reach statistical significance, and development history includes missed primary endpoints.

Adverse events. Injection-site reactions were common in the trials.

Limitations. This is an important example of conflicting evidence: positive early signals were not consistently confirmed in larger trials.

Search PubMed for this literature →

Pharmacology

Reported parameters

Half-life

2 hours

Reported in the low single-digit hours in early-phase work.

Visualise this half-life →

Routes studied

Subcutaneous
Intravenous
Topical ophthalmic

Evidence from one route does not transfer to another. Topical, oral, and injected administration can produce entirely different exposure from the same quantity.

Regulatory status

Investigational. Development history includes trials that did not meet primary endpoints.

Storage as documented

No approved storage labelling exists.

Safety

Signals reported in the literature

This lists what has been reported. It is not a complete safety profile, and for several compounds in this catalogue no systematic safety surveillance exists at all.

Injection-site reactions were common in the trials
Long-term safety not established

Limitations

What the evidence cannot tell you

Given as much prominence as the findings, because for most compounds in this category it is the more important half.

Results across programmes have been inconsistent
Positive early signals were not consistently confirmed in larger trials

Reference

Evidence grading key

AStrong

Multiple high-quality human trials, or strong systematic-review or meta-analytic evidence.

BModerate

Human controlled evidence exists, but replication or sample size is limited.

CPreliminary

Small human studies, observational evidence, or early-phase clinical work.

DPreclinical

Predominantly animal or in-vitro evidence. No adequate human outcome data.

EMechanistic

Biological plausibility or mechanistic reasoning without adequate outcome evidence.

XInsufficient / conflicting

Evidence is absent, irreconcilable, or too weak to support any directional statement.

Other compounds studied in the same research areas

MOTS-c

An interesting endogenous signalling peptide with genuine mechanistic literature and essentially no human outcome evidence.

NAD+ and precursors

Included because it appears constantly alongside peptides. Oral precursors reliably raise blood NAD+. Downstream functional benefits have been inconsistent.