Syndax Pharmaceuticals announced positive results from the BEAT AML trial of revumenib combined with venetoclax and azacitidine (ven/aza) in newly diagnosed older adults with mutant NPM1 (mNPM1) or KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML). The combination therapy demonstrated a high complete remission rate (67%), overall response rate (88%), and 100% measurable residual disease (MRD) negativity among responders. These findings are particularly important for older AML patients ineligible for intensive chemotherapy, who historically face poor prognoses.
This news offers significant potential advancements in AML treatment, specifically for the targeted patient population. The high CR, ORR, and MRD negativity rates observed with the revumenib combination suggest a substantial improvement over existing venetoclax/azacitidine regimens. These data could lead to a new standard of care, potentially extending survival and improving quality of life for these patients.
The Phase 1b BEAT AML trial enrolled 43 patients, the majority with mNPM1 AML. Revumenib was well-tolerated at both tested dose levels in combination with ven/aza. Most common adverse events were generally low grade and manageable. Early survival analysis showed a median overall survival of 15.5 months, notably higher than historical data for similar patient groups treated with ven/aza alone. Importantly, a high proportion of patients achieved MRD negativity, a key predictor of long-term remission.
These promising results support the ongoing Phase 3 EVOLVE-2 trial evaluating the revumenib/ven/aza combination in newly diagnosed mNPM1 AML patients unfit for intensive chemotherapy. Positive results from EVOLVE-2 could lead to regulatory approval and widespread adoption of this combination therapy, potentially transforming the treatment landscape for this patient population. This development also strengthens revumenib’s position as a potential cornerstone therapy in menin-dependent leukemias.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
