SS-31 (elamipretide)

SS-31, generic name elamipretide, is a tetrapeptide designed to concentrate inside mitochondria and bind cardiolipin on the inner membrane. It is the most clinically tested mitochondrial-targeted peptide, with phase 2 and phase 3 trials in rare mitochondrial disease, heart failure and eye disease. It is not approved for any indication, and regulators have declined or delayed approval for Barth syndrome.

1. 1

   Binds cardiolipin, the lipid that anchors the energy-producing machinery of the inner mitochondrial membrane.

2. 2

   Stabilises the electron transport chain, improving ATP output in damaged mitochondria.

3. 3

   Reduces reactive oxygen species leaking from the chain.

4. 4

   Restores mitochondrial inner-membrane architecture in laboratory models.

5. 5

   Because it acts on the organelle rather than a receptor, its effects are broad rather than tissue-specific — both the appeal and the problem.

• Improved walking distance in some mitochondrial myopathy trials, though the largest trial missed its primary endpoint
• Improved cardiac measures in small heart-failure studies
• Lower oxidative-stress markers
• No consistent, confirmed clinical benefit yet

1. 01Elamipretide clinical trials — PubMed search
2. 02SS-31 cardiolipin and mitochondrial function — PubMed search

References link to published literature, regulator pages or literature indexes. Where a link points to a search, it collects the peer-reviewed work on that molecule rather than a single study.