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Milademetan is an orally available small molecule inhibitor of the MDM2-p53 interaction. By binding to MDM2 (murine double minute 2), it prevents MDM2 from interacting with and inhibiting the tumor suppressor protein p53. This reactivates p53 function, leading to cell cycle arrest and apoptosis in cancer cells with wild-type TP53. Milademetan has demonstrated antitumor activity in preclinical models and clinical trials, particularly in tumors characterized by MDM2 amplification such as dedifferentiated liposarcoma (DDLPS) and other solid tumors. It is being developed for use in various cancers where restoration of p53 activity may be therapeutically beneficial.
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