SS-31

SS-31, also known as elamipretide, is a short peptide that concentrates in the inner mitochondrial membrane and binds cardiolipin, a phospholipid whose organisation affects the efficiency of the electron transport chain.

It is among the more seriously developed compounds in this index, with a genuine clinical programme in primary mitochondrial myopathy, Barth syndrome and ophthalmic conditions. Those trials have produced mixed results, including failures to meet primary endpoints, and any account of this compound that reports only the mechanistic promise without the trial outcomes is incomplete.

Primary mitochondrial myopathy, Barth syndrome, heart failure and ophthalmic disease, in a real clinical programme with mixed results.

Injection-site reactions are the most commonly reported effect in the trial literature. The programme is the source for its safety profile, and it is more informative than most in this index.

Supplied as a solution for subcutaneous injection in the trial setting, refrigerated.