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Ziftomenib in combination with intensive chemotherapy in newly diagnosed NPM1m or KMT2Ar AML: Updated KOMET-007 results
Mutations in NPM1 and KMT2A genes drive leukemogenesis in ~35–40% of patients with AML.1 Ziftomenib is a potent, selective, oral menin inhibitor under investigation in clinical trials as a monotherapy and in combination regimens for patients with NPM1-mutated (NPM1m) or KMT2A-rearranged (KMT2Ar) AML.1 During the European Hematology Association (EHA) 2025 Congress, Harry Erba presented updated results from the ongoing phase Ia/b KOMET-007 trial (NCT05735184) of ziftomenib at the RP2D of 600 mg QD in combination with intensive induction chemotherapy (7+3), in patients with newly diagnosed NPM1m (n = 49) or KMT2Ar (n = 33) AML (N = 82).1 The primary endpoints were AEs, DLTs in phase Ia, and CR.1
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Key learnings |
Robust clinical responses were demonstrated in both newly diagnosed NPM1m (n = 44; cCR, 93%) and KMT2Ar (n = 27; cCR, 89%) response-evaluable patients, with cCR MRD negativity observed in 68% and 83% of patients, respectively. |
At median follow ups of 25 and 16 weeks, respectively, 96% of patients with NPM1m AML and 88% of patients with KMT2Ar AML remained alive and continued on study. |
Grade ≥3 TRAEs of interest were observed in 35% of all patients; the most common were febrile neutropenia (15%), decreased platelet count (15%), anemia (11%), and decreased neutrophil count (11%). Differentiation syndrome, reported in one patient with KMT2Ar AML, was successfully brought under control. |
These tolerability and clinical activity data support the continued investigation of ziftomenib as a combination therapy in patients with newly diagnosed NPM1m and KMT2Ar AML in the upcoming phase III KOMET-017 trial. |
Abbreviations: AE, adverse event; AML, acute myeloid leukemia; CR, complete remission; cCR, composite CR; DLT, dose-limiting toxicity; EHA, European Hematology Association; KMT2Ar, KMT2A-rearranged; MRD, measurable residual disease; NPM1m, NPM1-mutated; QD, once daily; RP2D, recommended phase II dose; TRAE, treatment-related adverse event.
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When treating a patient newly diagnosed with IDH1-mutated AML who is ineligible for intensive chemotherapy, which initial treatment approach would you most likely consider?
