Syndax Pharmaceuticals Inc (NASDAQ:SNDX) announced topline data from the AUGMENT-101 trial of revumenib in adult and pediatric patients with relapsed/refractory (R/R) KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL).
The AUGMENT-101 trial met its primary endpoint with a complete remission (CR) or a CR with partial hematological recovery (CRh) rate of 23% (13/57) among the 57 efficacy evaluable patients in the pooled KMT2Ar acute leukemia cohort.
Related: Syndax Pharmaceuticals Set for Potential Breakthrough with Revumenib: Analyst Calls It ‘Potentially Best-In-Class’ In Competitive Landscape.
The CR/CRh rate in patients with KMT2Ar AML was 24.5% (12/49).
The CR/CRh responses in 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% (6/13) remaining in response.
Minimal residual disease (MRD) status was assessed in 10 of the 13 patients who achieved a CR/CRh, 70% (7/10) of whom were MRD negative.
In the efficacy-evaluable patients, the overall response rate was 63%. 14 (39%) patients who achieved an overall response underwent a hematopoietic stem cell transplant (HSCT), eight of whom did not achieve a CR or CRh before the transplant.
Half (7/14) of the patients who had an HSCT received post-transplant maintenance with revumenib, and three additional patients (3/14; 21%) were in follow-up and are eligible to restart revumenib as post-transplant maintenance.
Based on the Independent Data Monitoring Committee (IDMC) recommendation, the company is stopping the trial to further accrual in the KMT2Ar cohorts.
Price Action: SNDX shares are down 9.09% at $13.20 on the last check Monday.
