Eunice S. Wang, MD, Chief of Leukemia at Roswell Park Comprehensive Cancer Center, discusses long-term results of ziftomenib in combination with venetoclax and azacitidine in patients with acute myeloid leukemia (AML).
AML is a cancer that affects the blood and bone marrow. The signs and symptoms of AML vary but may include easy bruising, bone pain or tenderness, fatigue, fever, frequent nosebleeds, bleeding from the gums, shortness of breath, and/or weight loss. There are many potential causes of AML such as certain blood disorders, inherited syndromes, environmental exposures, and drug exposures; however, most people who develop AML have no identifiable risk factor.
Long-term results from the phase 1/2 KOMET-007 clinical trial (NCT05735184) evaluating ziftomenib in combination with venetoclax and azacitidine (intensive chemotherapy), 7+3, in newly diagnosed NPM1-m or KMT2A-r AML were presented at the European Hematology Association 2026 Congress.
Ziftomenib is a once-daily, oral menin inhibitor approved by the US Food and Drug Administration (FDA) for adult patients with relapsed or refractory AML with a susceptible NPM1 mutation who have no effective alternative treatment options.
The data presented included 64 response-evaluable patients with R/R NPM1-m AML, 27 of whom were treated in phase 1a dose escalation and 37 of whom were treated in phase 1b expansion, as of the January 16, 2026 data cutoff date. Patients had received 1 to 8 prior lines of therapy, and 37 patients had prior venetoclax exposure.
Overall, nearly two-thirds of all patients experienced clinically meaningful, deep, and durable responses. Rapid responses were also observed, with a median time to composite complete remission (CRc) of 3.9 weeks.
In the venetoclax-naïve population, a 70% CRc rate was observed with 75% central measurable residual disease (MRD) negativity. The objective response rate was 87% with a median duration of CRc response of 9.2 months. Median overall survival was not reached after median follow-up of 10.7 months.
In the venetoclax-experienced population, a 24% CRc rate was observed with 60% central MRD negativity. The objective response rate was 48% with a median duration of CRc response of 8.6 months. The median overall survival was 7.4 months after a median follow-up of 9.9 months.
Overall, the combination therapy was well tolerated, with a safety profile consistent with that reported for venetoclax and azacitidine alone. 3% of patients experienced differentiation syndrome that resolved with protocol-specified mitigation and there was one case of ziftomenib-related QTc. Median time to neutrophil and platelet recovery were similar to venetoclax-based regimens alone.
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Results were also published in Blood.
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