Ziftomenib Development and Clinical Trials - Ziftomenib is a targeted menin inhibitor being developed for relapsed/refractory and newly diagnosed acute myeloid leukemia (AML)[7] - An NDA for ziftomenib has been granted Priority Review, with a PDUFA target action date of November 30, 2025[7] - Kura Oncology and Kyowa Kirin are collaborating to investigate ziftomenib across the AML continuum, targeting up to 50% of patients for whom the Menin-KMT2A pathway is a disease driver[59] - KOMET-017 are Phase 3 clinical trials evaluating ziftomenib in combination with intensive chemotherapy (7+3) or non-intensive therapy (Venetoclax + Azacitidine) in newly diagnosed AML patients[62] KOMET-007 Clinical Trial Results - In the KOMET-007 study, the combination of ziftomenib 600 mg QD with 7+3 chemotherapy showed a safety profile consistent with previous reports for newly diagnosed AML patients treated with 7+3 alone[56] - The KOMET-007 study demonstrated robust clinical activity in newly diagnosed NPM1-m and KMT2A-r AML patients, with a composite complete remission (CRc) rate of 93% for NPM1-m and 89% for KMT2A-r patients[56] - Composite complete remission with measurable residual disease (CRc MRD) negativity was achieved in 68% of NPM1-m patients at a median of 4.7 weeks and 83% of KMT2A-r patients at a median of 4.1 weeks[56] - 96% (47/49) of NPM1-m and 88% (29/33) of KMT2A-r patients remained alive and continued on-study, with median follow-up times of 25 and 16 weeks, respectively[56] AML Market and Opportunity - An estimated 22,000 new cases of AML are diagnosed each year in the United States[16] - Up to 50% of AML cases may be menin-dependent, including those driven by NPM1m and KMT2Ar mutations[19] - The potential peak sales for menin inhibitors in first-line AML is estimated to be greater than $7 billion per year in the U.S[73]