Elamipretide (SS-31, MTP-131, formerly Bendavia) is a synthetic, mitochondria-targeting tetrapeptide (sequence D-Arg-Dmt-Lys-Phe-NH2) that binds cardiolipin on the inner mitochondrial membrane and is proposed to stabilise cristae structure and support mitochondrial bioenergetics. It was investigated clinically by Stealth BioTherapeutics across several indications, including primary mitochondrial myopathy, Barth syndrome, heart failure, and dry age-related macular degeneration (geographic atrophy). The trial record is mixed, with several pivotal studies missing their primary endpoints. In September 2025 elamipretide (brand name Forzinity) received accelerated FDA approval in the United States solely for the ultra-rare Barth syndrome; for all other investigated indications it remains investigational and it is not approved as a medicine outside the United States.
Elamipretide is a cell-permeable tetrapeptide with alternating aromatic and basic residues that selectively concentrates on cardiolipin — a phospholipid found almost exclusively in the inner mitochondrial membrane that is important for cristae curvature and the organisation of the respiratory-chain complexes. By binding cardiolipin, elamipretide is proposed to stabilise cristae architecture, support electron transport and ATP production, and reduce the formation of reactive oxygen species. These mechanistic models derive largely from cell and animal models and biophysical work; the extent to which they explain clinical efficacy in humans is, given the mixed trial results, a matter of ongoing research.
I peptidi arrivano come polvere secca. Una volta disciolti in un liquido (ricostituzione), questo calcolatore risponde a una sola domanda: quanta sostanza c'è in un millilitro di soluzione dopo?
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