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KAT-101 is a small molecule derivative of 3-bromopyruvic acid under development for the treatment of various cancers, including metastatic hepatocellular carcinoma (HCC), bladder cancer, melanoma, pancreatic cancer, leukemia, fibrolamellar hepatocellular carcinoma (FLC), non-small-cell lung cancer (NSCLC), acute myeloid leukemia (AML), and uterine cancer[1][3][4][8]. The drug acts by targeting glyceraldehyde 3-phosphate dehydrogenase (GAPDH) to disrupt both glycolysis and mitochondrial oxidative phosphorylation in cancer cells. This dual inhibition leads to depletion of adenosine triphosphate (ATP) levels within tumor cells, thereby limiting their energy supply and inducing apoptosis while preventing proliferation[1][3]. Clinical trials are ongoing to evaluate its safety and efficacy in HCC using oral and intratumoral administration routes[2][4][5].
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