New targeted combination in people with relapsed acute myeloid leukemia shows promise
Medical Xpress
Last updated: June 5, 2026
New research highlights a promising investigational treatment for acute myeloid leukemia (AML) using the targeted therapy ziftomenib in combination with venetoclax and azacitidine. This three-drug regimen shows encouraging results for patients with relapsed or refractory NPM1-mutated AML, a particularly challenging subtype.
- A clinical trial evaluated adding ziftomenib, an oral targeted therapy, to venetoclax and azacitidine for patients with NPM1-mutated AML whose leukemia had returned or not responded to previous treatments.
- Among patients who had not previously received venetoclax, approximately 90% responded to the combination therapy, with 70% achieving complete or near-complete remission. Many of these responses were deep, indicating no detectable cancer cells.
- For patients who had prior venetoclax treatment, about half showed a response.
- Responses were rapid, occurring within an average of four weeks, and often lasted for several months. The treatment was generally well-tolerated with a favorable safety profile.
- These findings are from the ongoing KOMET-007 clinical trial. While ziftomenib is approved as a single agent for certain AML cases, its use in combination therapies is still investigational.
