mitochonic acid 5

Mitochonic acid 5 (MA-5) is an orally active, synthetic small molecule derived from the plant hormone indole-3-acetic acid. Developed by researchers at Tohoku University, MA-5 is designed to enhance mitochondrial function and ATP production. It physically binds to the mitochondrial inner membrane protein mitofilin (MIC60), facilitating ATP synthase oligomerization and supercomplex formation. This mechanism stabilizes mitochondrial cristae, boosts ATP production, and reduces reactive oxygen species (ROS) generation under stress conditions, independent of the electron transport chain. MA-5 is under clinical development for the treatment of mitochondrial diseases (such as MELAS, Leigh syndrome, Leber's hereditary optic neuropathy, and Kearns-Sayre syndrome) and associated hearing loss, and has shown preclinical efficacy in models of amyotrophic lateral sclerosis (ALS), Barth syndrome, and acute kidney injury.

Other names

4-(2,4-difluorophenyl)-2-(1H-indol-3-yl)-4-oxobutanoic acidmitochonic acid-5