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HDM201 + venetoclax is a combination therapy of HDM201 (also known as siremadlin), a small molecule MDM2 inhibitor, and venetoclax, a selective BCL-2 inhibitor. HDM201 (siremadlin) restores p53 tumor suppressor activity by antagonizing the MDM2-p53 protein interaction, leading to p53 stabilization and apoptosis in TP53 wild-type cancer cells. Venetoclax induces apoptosis by inhibiting the anti-apoptotic protein BCL-2. This combination is being studied primarily for antineoplastic effects in acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS), leveraging dual pro-apoptotic mechanisms. The regimen is under early-phase clinical investigation and does not have a unified brand name. HDM201 was developed by Novartis and venetoclax by AbbVie/Genentech.
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