MTX325

MTX325 is a potent, selective, central nervous system (CNS)-penetrant small molecule inhibitor of the mitochondrial deubiquitylating enzyme USP30. It is designed to improve mitochondrial quality and function by enhancing mitophagy—the process by which cells remove dysfunctional mitochondria. By inhibiting USP30, which acts as a negative regulator of mitophagy, MTX325 promotes the clearance of damaged mitochondria and protects dopamine-producing neurons in the brain. Preclinical studies have shown that MTX325 can protect against dopaminergic neuron loss and reduce pathological alpha-synuclein accumulation in mouse models of Parkinson’s disease. The drug is being developed as a potential disease-modifying therapy for Parkinson’s disease and may have applications in other conditions driven by mitochondrial dysfunction such as idiopathic pulmonary fibrosis, chronic kidney disease, and heart failure[1][3][4][5][6].

Other names

MTX115325MTX-115325MTX 115325