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AVN-944 is an orally available, synthetic small molecule with potential antineoplastic activity. It acts as a potent, selective, noncompetitive inhibitor of inosine monophosphate dehydrogenase (IMPDH), an enzyme involved in the de novo synthesis of guanosine triphosphate (GTP), which is essential for DNA and RNA synthesis. By inhibiting IMPDH—particularly the isoforms IMPDH1 and IMPDH2—AVN-944 deprives cancer cells of GTP, disrupting nucleic acid synthesis and cell proliferation while inducing apoptosis. The drug has demonstrated antiproliferative effects in various cancer cell lines and animal models and appears to have a selective effect on cancer cells compared to normal cells. AVN-944 has been investigated primarily for advanced hematologic malignancies such as leukemia, lymphoma, multiple myeloma, as well as solid tumors including pancreatic cancer[1][2][3][4][5][6][8]. The drug was developed by Avalon Pharmaceuticals.
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