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Karonudib is a first-in-class small molecule inhibitor targeting the DNA repair enzyme MutT homolog 1 (MTH1). By antagonizing MTH1, karonudib induces toxic oxidative DNA damage selectively in cancer cells and activated T cells, leading to cell death. This mechanism exploits the high oxidative stress and reliance on MTH1 for survival in malignant cells while sparing normal tissues. Karonudib has demonstrated potent anti-tumor activity in preclinical models of various cancers—including hepatocellular carcinoma, B-cell lymphoma, osteosarcoma, melanoma—and shows promise as an immunomodulatory agent by inhibiting proliferation of activated T cells. It is being developed primarily for hematological malignancies and solid tumors but also investigated for autoimmune diseases such as autoimmune hepatitis[1][2][3][4][5].
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