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MI-519-64 is an orally active small-molecule inhibitor of the MDM2 (Mouse double minute 2 homolog) protein, originally developed at the University of Michigan and licensed by Ascenta Therapeutics. It is designed to disrupt the interaction between MDM2 and the p53 tumor suppressor protein. In many cancers, MDM2 is overexpressed or amplified, leading to the degradation and inactivation of wild-type p53. By blocking this interaction, MI-519-64 restores p53's natural tumor suppression function, potentially triggering cell cycle arrest and apoptosis in cancer cells. In 2010, Ascenta Therapeutics licensed MI-519-64 to Sanofi as part of a global collaboration to develop MDM2 inhibitors for the treatment of various neoplasms.
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