MT-514

MT-514 is a small-molecule mitochondrial uncoupler developed by Mito BioPharm as an investigational candidate for the treatment of non-alcoholic steatohepatitis (NASH), type 2 diabetes, and various cancers (including hepatocellular carcinoma, metastatic colon cancer, metastatic pancreatic cancer, acute lymphocytic leukemia, and acute myeloid leukemia). Developed using Mito BioPharm's proprietary 'miunins' platform, MT-514 acts as a mitochondrial modulator that mimics the physiological uncoupling actions of uncoupling protein 1 (UCP1) and uncoupling proteins 2 and 3 (UCP2/3). By facilitating proton leak across the inner mitochondrial membrane, MT-514 dissipates the proton gradient, thereby increasing oxygen consumption and cellular energy expenditure while reducing ATP synthesis. This metabolic reprogramming activates AMP-activated protein kinase (AMPK) and enhances lipid oxidation, addressing ectopic lipid accumulation in metabolic diseases and restricting energy availability in cancer cells. MT-514 has been selected as an IND lead compound, demonstrating favorable pharmacology, ADME, pharmacokinetic/pharmacodynamic (PK/PD), and toxicological profiles in preclinical studies.