Elamipretide (MTP-131) is a mitochondria-targeted antioxidant tetrapeptide (D-Arg-dimethylTyr-Lys-Phe-NH2) that reduces toxic reactive oxygen species (ROS) production and stabilizes cardiolipin, with blood-brain-barrier permeability. It is a potential therapeutic agent in the rare disease of Barnes syndrome.

METHODS: Rat insulinoma cells, INS1, were treated with Elamipretide (1-10 μM) for 20 h. Cellular mitochondria were detected by Immunofluorescence.
RESULTS: Elamipretide prevented mitochondrial breaks induced by nutrient overload, and at the same time had no effect on the mitochondrial morphology of healthy INS1 cells. [1]
METHODS: Immortalized human trabecular meshwork cells iHTM and glaucomatous human trabecular cell line GTM3 cells were pretreated with Elamipretide (0.01-1 μM) for 1 h, followed by incubation with H2O2 (200 μM) for 24 h, and LDH release was detected by calorimetric assay.
RESULTS: Incubation with H2O2 resulted in a significant increase in LDH release in iHTM and GTM3 cells, and pretreatment with MTP-131 dose-dependently inhibited LDH release. [2]

METHODS: To investigate the effects on cognitive sequelae of inflammation and oxidative stress, Elamipretide (5 mg/kg) was administered intraperitoneally to LPS-injected C57BL/6 mice once daily for three days.
RESULTS: LPS-induced memory impairment could be attenuated by the mitochondria-targeted antioxidant Elamipretide. [3]
METHODS: To test the phenotypic effects on aging in mice, Elamipretide (3 mg/kg) was injected subcutaneously into C57BL/6 mice five times a week for ten months.
RESULTS: Elamipretide enhanced healthy aging and cardiac function in both male and female mice, although the specific effects on function varied by sex. In females, the treatment improved cognition and maintained body composition, while in males, rotisserie performance was preserved. [4]