MA-5

MA-5 (Mitochonic Acid 5) is a first-in-class, orally bioavailable small molecule developed by Mitochonic Medical and Tohoku University for the treatment of mitochondrial diseases and other conditions associated with mitochondrial dysfunction. Chemically identified as an indole derivative, MA-5 functions by binding to the alpha subunit of mitochondrial F1F0-ATP synthase (ATP5A1). This binding facilitates the dimerization and oligomerization of ATP synthase, which in turn stabilizes the mitochondrial cristae and respiratory supercomplexes. The resulting enhancement in ATP production and reduction in reactive oxygen species (ROS) levels help preserve cellular viability under conditions of metabolic stress. MA-5 has demonstrated the ability to protect cells from death caused by mitochondrial DNA mutations and is currently being evaluated in Phase 1 clinical trials for mitochondrial diseases, including Leigh syndrome and MELAS. It has also shown preclinical efficacy in models of amyotrophic lateral sclerosis (ALS) and chronic kidney disease (CKD).

Other names

Mitochonic Acid 54-(2,4-difluorophenyl)-2-(1H-indol-3-yl)-4-oxobutanoic acid