Plain-English peptide research. Affiliate links are disclosed.

SS-31

Also cited in the literature as Elamipretide

A four-amino-acid peptide aimed at the energy-producing parts of cells. It reached late-stage trials in rare mitochondrial diseases with mixed results.

Regulatory status: Not licensed as a medicine in the United Kingdom. It holds no MHRA marketing authorisation, has no approved indication, and is not approved for human or animal use.

Illustration of cells, mitochondria and surrounding fibres

Cell & tissue research

Research stage: Phase 3 results published in rare disease groups

Cells
Animal studies
Human trials

The research pathway — not every compound has reached human trials. Illustrations are simplified, not study results.

What it is

SS-31, developed as elamipretide, is a laboratory-made peptide of four amino acids designed to concentrate in mitochondria, the structures that generate energy inside cells.

How researchers think it works

Laboratory studies reported that it bound to cardiolipin, a fat found in mitochondrial membranes, and that treated cells handled energy production differently under stress.

What has research seen in people?

This one reached late-stage trials in people with rare mitochondrial conditions. Results were mixed: some measures moved, the main pre-specified endpoints were not consistently met.

Trials in heart failure and an eye condition were also published or reported.

Injection-site reactions were commonly reported, and no regulator had approved it.

Where this material is listed

For laboratory research use only. Not a medicine and not for human or animal use.

Veritide does not sell or supply anything. This links to a third-party UK laboratory supplier; we may earn a commission if you buy through it, at no extra cost to you. Materials are sold for laboratory research use only and are not licensed medicines.

What studies have found

  • Phase 2 and phase 3 trials studied people with primary mitochondrial myopathy and with Barth syndrome. Published results were mixed: some measures improved and the main pre-specified endpoints were not consistently met.
  • Trials in heart failure and in a form of macular degeneration were also published or reported.
  • Injection-site reactions were commonly reported.

What we still do not know

  • The clearest results came from very small groups with rare inherited conditions, which limits what can be read across to anyone else.
  • Several trials missed their primary endpoints, and regulators had not approved it at the time of writing.

Sources

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