MTX652

MTX652 is a potent, selective, and peripheral (non-CNS penetrant) small molecule inhibitor of Ubiquitin-specific protease 30 (USP30), a deubiquitylating enzyme (DUB) located on the outer mitochondrial membrane. Developed by Mission Therapeutics, MTX652 is designed to enhance mitophagy—the cellular process for identifying and degrading damaged or dysfunctional mitochondria—by preventing USP30 from removing ubiquitin chains that signal for mitochondrial clearance. By improving mitochondrial quality and cellular health, MTX652 aims to treat conditions driven by mitochondrial dysfunction. The drug has completed Phase I clinical trials demonstrating safety and tolerability and has received FDA approval to advance into Phase II trials for acute kidney injury (AKI). It is also being investigated for its potential in treating heart failure with preserved ejection fraction (HFpEF) and cardiomyopathy associated with Duchenne muscular dystrophy (DMD).

Other names

(S)-4-(5-cyclopropyl-2-methoxyphenyl)-N-(1-(3,4-difluorophenyl)ethyl)benzenesulfonamide