Alrizomadlin (APG-115) Wins FDAs Orphan Drug Designation

Release Date: 16-Sep-2021

US FDA has granted an orphan drug designation (ODD) to alrizomadlin (APG-115), an MDM2-p53 inhibitor, for the treatment of stage 2b to 4 melanoma. Alrizomadlin in combination with a PD-1 blockade advances antitumor activity by activating adaptive antitumor activity. This drug holds 5 other ODDs in gastric cancer, acute myeloid leukemia, soft tissue sarcoma, and retinoblastoma.


In Phase 1/2 study the estimated enrollment is 203 patients and an estimated completion date is October 2022. The primary end points of the study are maximum tolerated dose, recommended phase 2 dose, and overall response rate (ORR). In the melanoma cohort, the ORR was 17.4% and the disease control rate (DCR) was 55.2%. Two PRs were pending confirmation at the time of data cutoff. There were 3 melanoma subgroups assessed in the study, including 8 patients with uveal melanoma, 5 with muscosal melanoma, 16 with cutaneous melanoma, and 3 with an unknown primary melanoma type.


Treatment-related adverse events (TRAEs) involved were diarrhea (21.4%), neutropenia (15.4%), nausea (63.1%), thrombocytopenia (36.9%), vomiting (33.3%), fatigue (31.0%), decreased appetite (27.4%), and anemia (11.9%).Grade andge;3 TRAEs include thrombocytopenia (20.2%), neutropenia (14.2%), and anemia (8.3%).

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