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HDM201 + cytarabine is an investigational combination therapy under clinical evaluation primarily for acute myeloid leukemia (AML). HDM201 (also known as NVP-HDM201) is a potent, selective, orally available small molecule inhibitor of MDM2 (mouse double minute 2 homolog), which activates the p53 tumor suppressor pathway by blocking the interaction between MDM2 and p53. Cytarabine is a well-established antimetabolite chemotherapeutic that inhibits DNA synthesis. The combination is designed to exploit synthetic lethality, with HDM201 reactivating p53-mediated apoptosis and cytarabine inducing DNA damage; together, they are hypothesized to synergistically induce cell death in p53 wild-type AML. Preclinical and early clinical studies focus on patients with relapsed or refractory AML, and/or specific genetic subtypes, including FLT3-ITD/TP53wt AML[1][3].
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