Syndax presents ‘positive’ results from AUGMENT-101 trial of Revumenib

Syndax Pharmaceuticals announced the presentation of positive data from the pivotal AUGMENT-101 trial in pediatric patients with relapsed/refractory KMT2A-rearranged acute myeloid leukemia and acute lymphoid leukemia treated with revumenib, a first-in-class menin inhibitor. The pediatric data was featured in a Plenary Session titled “Pivotal Phase 2 Results of AUGMENT-101 for Revumenib in KMT2Ar Acute Leukemia: Pediatric Experience” at the 2024 American Society of Pediatric Hematology/Oncology Conference held April 3 – 6, 2024 in Seattle, Washington. “We are pleased to present positive results from pediatric patients treated with revumenib in the AUGMENT-101 pivotal trial demonstrating impressive activity and consistency with the adult population,” said Neil Gallagher, M.D., Ph.D., President, Head of Research and Development at Syndax. “As the only menin inhibitor with a formulation designed for the pediatric setting, we have an ongoing commitment to bringing this first- and best-in-class therapeutic agent to this important patient population in need of effective treatments.” The presented data include efficacy and safety findings for pediatric patients with R/R KMT2Ar acute leukemia from the pivotal Phase 2 portion of the AUGMENT-101 study. Of the 57 patients enrolled in AUGMENT-101 with central confirmation of their KMT2Ar status and sufficient follow-up to be included in the efficacy-evaluable population, 13 (23%) were less than 18 years old with a median age of 5 years. The pediatric patients were heavily pretreated with a median of 4 prior lines of therapy including 8 (62%) that received prior venetoclax, 2 (15%) that received CAR-T and 6 (46%) that received prior hematopoietic stem cell transplant. In this population, the complete remission or CR with partial hematological recovery rate was 23% (3/13; 95% CI: 5.0, 53.8), with a median time to CR or CRh of 2.3 months (95% CI: 1.0, 3.9). The overall response rate1 was 46% (6/13), and the composite response rate2 (CRc) was 39% (5/13). Sixty percent (3/5) of CRc patients achieved minimal residual disease negative status. The median overall survival was 6.9 months (95% CI, 2.3-not reached). Four (67%) of the 6 patients who achieved an overall response underwent HSCT, two of whom did not achieve a CR or CRh prior to transplant. Half (2/4) of the patients who underwent HSCT received post-transplant maintenance with revumenib and had been in remission for 6 and 9 months at the time of the July 24, 2023 data cutoff. Revumenib was well-tolerated, and the safety profile was consistent with the Company’s previously reported data. In the safety-evaluable patient population, Grade 3 or greater treatment-related adverse events that occurred in greater than 10% of patients included febrile neutropenia (13%; 3/23) and decreased neutrophil count (13%; 3/23). Grade 3 or greater differentiation syndrome was observed in 9% (2/23) of patients and Grade 3 QTc prolongation was observed in 4% (1/23) of patients. No treatment-related discontinuations or dose reductions due to adverse events occurred in the trial.