Elamipretide (also known as SS-31, MTP-131 and Bendavia)
is a small
mitochondrial
A mitochondrion () is an organelle found in the cells of most eukaryotes, such as animals, plants and fungi. Mitochondria have a double membrane structure and use aerobic respiration to generate adenosine triphosphate (ATP), which is used ...
ly-targeted
tetrapeptide
A tetrapeptide is a peptide, classified as an oligopeptide, since it only consists of four amino acids joined by peptide bonds. Many tetrapeptides are pharmacologically active, often showing affinity and specificity for a variety of receptors ...
(
D-Arg-dimethylTyr-Lys-Phe-NH
2) that appears to reduce the production of toxic reactive oxygen species and stabilize
cardiolipin
Cardiolipin (IUPAC name 1,3-bis(''sn''-3’-phosphatidyl)-''sn''-glycerol, "''sn''" designating stereospecific numbering) is an important component of the inner mitochondrial membrane, where it constitutes about 20% of the total lipid composition. ...
.
Stealth Peptides, a privately held company, was founded in 2006 to
develop intellectual property licensed from several universities including elamipretide; it subsequently changed its name to Stealth BioTherapeutics.
As of November 2017 Stealth had obtained an orphan designation in the US for use in
mitochondrial myopathy
Mitochondrial myopathies are types of myopathy, myopathies associated with mitochondrial disease. Adenosine triphosphate (Adenosine triphosphate, ATP), the chemical used to provide energy for the cell, cannot be produced sufficiently by oxidative ...
and had started a Phase III trial in that indication.
[ As of January 2020, trial expectations were not met.]
References
{{Reflist
Tetrapeptides
Experimental drugs