Milademetan, an oral MDM2 inhibitor, in well-differentiated/ dedifferentiated liposarcoma: results from a phase 1 study in patients with solid tumors or lymphomas

      Background: Patients with well-differentiated and/or dedifferentiated liposarcoma (WD/DD LPS) have few treatment options. The MDM2 gene encodes an E3 ubiquitin ligase that targets the p53 tumor suppressor for degradation; it is amplified in WD/DD LPS and is a diagnostic hallmark. Milademetan (DS-3032b, RAIN-32) is an oral MDM2 inhibitor with demonstrated preclinical antitumor activity.
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