1213269-98-7

EPI-743 (Vatiquinone; α-Tocotrienol quinone; PTC-743; NCT04378075) is a potent cellular oxidative stress protectant, inhibits ferroptosis in cells, which could be used for the study for mitochondrial diseases. EPI-743 is a synthetic analog of vitamin E with oral activity, targets repletion of reduced intracellular glutathione[1][2].

Vatiquinone (EPI-743)a metabolite of a-tocotrienolis an orally available and highly potent analog of coenzyme Q10 th at modulates the activity of oxidoreductases including AD(P)H:quinone oxidoreductase 1resulting in increased cellular GSH concentration and improvement in redox status. Vatiquinone is an agent designated to tre at mitochondrial diseases.

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[1] Kouga T, et al. Japanese Leigh syndrome case treated with EPI-743. Brain Dev. 2018 Feb;40(2):145-149. Brain Dev. 2018 Feb;40(2):145-149. DOI:10.1016/j.braindev.2017.08.005
[2] Avula S, et al. Treatment of Mitochondrial Disorders. Curr Treat Options Neurol. 2014. 16:292.
[3] Kahn-Kirby AH, et al. Targeting ferroptosis: A novel therapeutic strategy for the treatment of mitochondrial disease-related epilepsy. PLoS One. 2019 Mar 28;14(3):e0214250. DOI:10.1371/journal.pone.0214250