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ATN-224 is an orally available small molecule copper chelator and a second-generation analog of ammonium tetrathiomolybdate. It acts primarily as an inhibitor of copper-zinc superoxide dismutase 1 (SOD1), leading to antiangiogenic and antitumor effects by reducing the secretion of angiogenic factors and inhibiting tumor growth. ATN-224 has been investigated in multiple clinical trials for advanced solid tumors, prostate cancer, multiple myeloma (in combination with bortezomib), Wilson's disease (as WTX-101), and hepatolenticular degeneration. The drug is well tolerated at oral doses up to 300 mg/day, with dose-limiting toxicities including fatigue, anemia, neutropenia, and sulfur eructation. It significantly reduces serum ceruloplasmin levels—a marker for copper depletion—and SOD1 activity in blood cells[2][5][6][8].
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