Study
| Phase I dose-escalation and expansion study [NCT03013998] |
| First-line treatment for patients aged 60 years and older with newly diagnosed NPM1-mutated or KMT2A-rearranged AML |
| Azacitidine + Venetoclax + Revumenib at two dose levels (113 mg or 163 mg) in combination with azoles (n=43) |
Efficacy
| ORR: 88.4% (NPM1m: 85%; KMT2Ar: 100%) |
| CR: 67.4% (NPM1m: 65%; KMT2Ar: 78%) Median time to response: 28 days mOS: 15.5 mos (NPM1m: 15.5 mos, KMT2Ar: 18.0 mos) |
Safety
| Grade >=3 AE: QTc prolongation (44%), hypokalemia (44%), nausea (60%), constipation (53%) Differentiation syndrome: 19% |
J Clin Oncol 2025;43:2606-2615
http://doi.org/10.1200/JCO-25-00914
Reviewed by Elvin Chalabiyev, MD on Sep 3, 2025
