Syndax’s Revumenib Shows Positive Phase 2 Results in AML Treatment

Syndax Pharmaceuticals recently shared positive data from the Phase 2 AUGMENT-101 trial, examining the efficacy of revumenib, a menin inhibitor, for treating relapsed or refractory mutant NPM1 (mNPM1) acute myeloid leukemia (AML). The drug achieved a 23% complete remission rate among patients, including those with partial hematological recovery, demonstrating promise in a cohort with limited treatment options. Of those who achieved remission, the median duration of response was 4.7 months, with 64% reaching minimal residual disease (MRD)-negative status, indicating a potential for deep, molecular remissions. The overall response rate in this population was 47%, with several patients advancing to stem cell transplantation.

The safety profile of revumenib was generally manageable, with adverse events like QTc prolongation and febrile neutropenia, though these events were consistent with prior data. No patients experienced Grade 5 treatment-related adverse events, and only a small percentage discontinued the treatment due to side effects. These findings highlight revumenib’s potential to provide therapeutic benefit with a tolerable safety profile, offering hope for patients with refractory AML, especially those who have failed prior treatments, including venetoclax.

CEO Michael Metzger expressed optimism about these results, underscoring revumenib’s potential as a new option for treating AML with specific genetic markers. The drug’s impact could extend to about 40% of AML patients with mNPM1 or KMT2A mutations. With anticipated FDA approval for revumenib in other leukemia indications on the horizon, Syndax is poised to make a significant impact in the AML treatment landscape, providing alternatives where few effective therapies exist.