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Ziftomenib in relapsed/refractory NPM1m AML: KOMET-001 efficacy and safety results
NPM1 mutations drive leukemogenesis in ~30% of patients with acute myeloid leukemia (AML).1 Ziftomenib is a potent, highly selective, oral menin inhibitor that has shown promising activity and tolerability as a monotherapy and in combination with standard chemotherapy for patients with relapsed/refractory (R/R) NPM1-mutated (NPM1m) and KMT2A-rearranged (KMT2Ar) AML.1 During the European Hematology Association (EHA) 2025 Congress, Amir Fathi presented results from the ongoing phase Ib/II KOMET-001 trial (NCT04067336) of ziftomenib at the recommended phase II dose of 600 mg once daily, in patients with R/R NPM1m AML (N = 112).1 The phase II (n = 92) primary endpoint was complete remission with full or partial hematologic recovery.1
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Key learnings |
Median OS was 6.1 months (95% CI, 3.8–8.4); 16.4 months (9.6–20.4) in responders and 3.5 months (2.5–4.0) in non-responders. 24/112 patients remained on-study, with nine patients on-treatment. |
The phase II primary endpoint was met, with a CR/CRh rate of 23% (p = 0.0058 vs historical control rate) and MRD negativity observed in 63% of CR/CRh responders. |
Grade ≥3 TRAEs were reported in 40% of phase II patients, including Grade ≥3 differentiation syndrome in 15% (no patients had Grade 4–5 differentiation syndrome). Of all patients, 3% discontinued treatment due to TRAEs. |
Ziftomenib was well tolerated, with clinically meaningful MRD-negative responses achieved in this heavily pre-treated population. Results support ziftomenib monotherapy as a potential treatment option for R/R NPM1m AML. |
Abbreviations: AML, acute myeloid leukemia; CR, complete remission; CRh, complete remission with partial hematologic recovery; EHA, European Hematology Association; KMT2Ar, KMT2A-rearranged; MRD, measurable residual disease; NPM1m, NPM1-mutated; OS, overall survival; R/R, relapsed/refractory; TRAE, treatment-related adverse event.
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