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AZD3632 is an orally administered investigational small molecule developed by AstraZeneca for the treatment of advanced hematologic malignancies, specifically for patients with relapsed or refractory acute leukemia, higher-risk myelodysplastic syndromes (MDS), and acute myeloid leukemia (AML) that harbor genotypes such as KMT2A rearrangement (KMT2Ar), NPM1 mutation (NPM1m), or other mutations associated with HOX gene overexpression. The drug is being studied as a monotherapy and in combination with anticancer agents like posaconazole. Its mechanism of action likely targets genetic pathways involving HOX gene overexpression, but the precise molecular target is not disclosed in public sources. It is currently in first-in-human, phase 1/2 clinical evaluation for safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy in this genetically defined subset of hematologic cancers[1][3][4][5].
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