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A single-arm, open-label, multicenter trial evaluated the efficacy of revumenib in 104 adult and pediatric patients. The median age of the patients was 37 years, with 83% diagnosed with acute myeloid leukemia, 15% with acute lymphoblastic leukemia, and 2% with mixed-phenotype acute leukemia. Patients had received a median of 2 lines of prior therapy, and 44% had undergone stem cell transplantation.
The primary efficacy endpoint was the complete remission rate plus the complete remission rate with partial hematologic recovery (CR+CRh). The results showed that the CR+CRh rate was 21.2%, with a median duration of response of 6.4 months. The complete remission (CR) rate was 12.5%, with a median duration of 4.3 months. The complete remission with partial hematologic recovery (CRh) rate was 8.7%, with a median duration of 6.4 months. The median time to achieve CR or CRh was 1.9 months. Among patients who were transfusion-dependent at baseline, 14% achieved transfusion independence; among patients who were transfusion-independent at baseline, 48% maintained transfusion independence.
Another cohort of the same trial evaluated the efficacy of revumenib in 65 patients. The median age of the patients was 65 years, and 50.8% were aged 65 years or older.
The results showed that the CR+CRh rate was 23.1%, with a median duration of response of 4.5 months. The CR rate was 18.5%, with a median duration of 3.7 months. The CRh rate was 4.6%, with observed durations of response of 1.8, 2.0, and 4.5 months, respectively. The median time to achieve CR or CRh was 2.8 months. Among patients who were transfusion-dependent at baseline, 17% achieved transfusion independence; among patients who were transfusion-independent at baseline, 68% maintained transfusion independence.
FDA，2025.10
