SS-31 / Elamipretide: from early signals to negative phase 3 results

This Research Library page summarizes independently published research on Elamipretide / SS-31. For each study, the research model, key finding, and main limitation are stated.

Study 1

Evidence level: Human research - phase 1/2

Topic: Safety, tolerability, and exercise outcomes.

Key finding: The study described dose- and time-dependent signals, but was small and exploratory.

Main limitation: No definitive evidence of sustained clinical efficacy.

Source: Original publication via PubMed

Study 2

Evidence level: Human research - phase 3 RCT

Topic: Primary mitochondrial myopathy.

Key finding: The primary endpoints for walking distance and fatigue were not met significantly.

Main limitation: An important negative/neutral result that should be mentioned in a balanced presentation.

Source: Original publication via PubMed

Study 3

Evidence level: Human research - phase 2/3 crossover

Topic: Mitochondrial dysfunction in Barth syndrome.

Key finding: The blinded phase showed no significant difference in the primary endpoints; later signals were reported during the extension.

Main limitation: Rare disease, small population, and open-label extension with a greater risk of bias.

Source: Original publication via PubMed

Scientific scope: Research on a molecule, research model, or pharmaceutical formulation does not constitute evidence of the efficacy, safety, purity, or suitability of a commercial Peptidera product. This page does not contain treatment or usage advice.

Header image: illustrative visualization, not a research result.