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AZD-5991 is a potent and selective small molecule inhibitor of the anti-apoptotic protein Mcl-1 (myeloid cell leukemia 1), a member of the Bcl-2 family. It acts as a BH3 mimetic that binds directly to Mcl-1 with sub-nanomolar potency and induces apoptosis in cancer cells by activating the Bak-dependent mitochondrial apoptotic pathway. Preclinical studies have shown strong activity against hematological malignancies such as multiple myeloma (MM) and acute myeloid leukemia (AML), both as monotherapy and in combination with other agents like venetoclax or bortezomib. The drug has demonstrated tumor regression in animal models after single intravenous doses. Clinical development has focused on relapsed or refractory hematologic malignancies, particularly AML.
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