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A new drug combo is turning heads in AML treatment. Adding ziftomenib to venetoclax and azacitidine produced a 64% overall response rate and 61% MRD negativity in patients with relapsed or refractory NPM1-mutated acute myeloid leukemia — with no dose-limiting toxicities. Researchers say the results support moving this combo into larger, earlier-stage trials.
A phase 1 clinical trial is offering fresh hope for patients with one of the toughest-to-treat blood cancers. The KOMET-007 study tested ziftomenib — a menin inhibitor — combined with venetoclax and azacitidine in 67 patients with relapsed or refractory (R/R) NPM1-mutated acute myeloid leukemia (AML). Results published in Blood show the triple combination delivered meaningful responses even in heavily pretreated patients.
The safety profile was reassuring too. No dose-limiting toxicities were observed, and the most notable side effect — pruritus (itching) — was generally manageable with gabapentin. Two patients developed differentiation syndrome but were able to continue treatment.
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Why it matters: These results are especially striking in venetoclax-naïve patients, suggesting the combo could be even more powerful earlier in treatment. A phase 3 trial (KOMET-017) is already underway to test this in newly diagnosed AML — potentially reshaping the standard of care.
