Syndax announces ‘positive’ results from AUGMENT-101 trial of revumenib

Syndax Pharmaceuticals announced positive topline data from the protocol-defined pooled analysis of the pivotal AUGMENT-101 trial of revumenib, a first-in-class menin inhibitor, in adult and pediatric patients with relapsed/refractory KMT2A-rearranged acute myeloid leukemia and acute lymphoid leukemia. The AUGMENT-101 trial met its primary endpoint at the protocol-defined interim analysis stage with a complete remission or a CR with partial hematological recovery rate of 23% among the 57 efficacy evaluable patients in the pooled KMT2Ar acute leukemia cohort. The CR/CRh rate in patients with KMT2Ar AML was 24.5%. The CR/CRh responses in both the overall population and the AML subset were durable with a 6.4-month median duration as of the July 24, 2023 data cut-off, with 46% remaining in response. Minimal residual disease status was assessed in 10 of the 13 patients who achieved a CR/CRh, 70% of whom were MRD negative. In the efficacy-evaluable patients, the overall response rate1 was 63%. A total of 14 patients who achieved an overall response underwent hematopoietic stem cell transplant, eight of whom did not achieve a CR or CRh prior to transplant. Half of the patients who had an HSCT received post-transplant maintenance with revumenib and three additional patients were in follow-up and are eligible to restart revumenib as post-transplant maintenance. Based on the Independent Data Monitoring Committee recommendation, the company is stopping the trial to further accrual in the KMT2Ar cohorts. Syndax continues to expect to submit an NDA for revumenib for the treatment of R/R KMT2Ar acute leukemia to the U.S. FDA by year-end.