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Ziftomenib is an inhibitor of the protein-protein interaction between menin and mixed-lineage leukemia 1 (MLL1).1 It decreases the viability of OCI-AML-3 and MV4-11 acute myeloid leukemia (AML) cells in a concentration-dependent manner. Ziftomenib selectively reduces the viability of p53R175H-expressing MOLM-13 leukemia cells and TP53-/- MOLM-13 cells over wild-type p53-expressing MOLM-13 cells. Ziftomenib (75 mg/kg) increases survival in a patient-derived xenograft (PDX) mouse model of AML. Formulations containing ziftomenib have been used in the treatment of AML.
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