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Ziftomenib + venetoclax is an investigational combination therapy for acute myeloid leukemia (AML), particularly for patients with nucleophosmin 1 (NPM1)-mutated or lysine methyltransferase 2A (KMT2A)-rearranged AML. Ziftomenib is a potent, selective small molecule inhibitor of the menin-KMT2A (MLL, KMT2A) protein-protein interaction, a key driver in these AML subtypes, while venetoclax is a BCL-2 inhibitor that induces apoptosis in hematologic malignancies. In preclinical models, combining ziftomenib with venetoclax demonstrates synergistic lethality and improved response in AML. Clinical trials are ongoing to assess the safety, tolerability, and efficacy of this combination in both relapsed/refractory (R/R) and newly diagnosed AML patients[1][2][3][4].
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