Core Insights - The combination of ziftomenib with venetoclax and azacitidine shows promising clinical activity in treating acute myeloid leukemia (AML) with NPM1 mutations, achieving an 86% composite complete remission (CRc) rate in newly diagnosed patients and a 65% overall response rate (ORR) in relapsed/refractory cases [1][2][5] - Ziftomenib has a favorable safety profile, with low rates of myelosuppression and manageable side effects, supporting its potential integration into treatment regimens for AML [4][7][8] - Ongoing registrational trials for ziftomenib are expected to further establish its role in both front-line and relapsed/refractory settings for AML [2][8][13] Summary by Category Clinical Efficacy - In newly diagnosed NPM1-m AML, 86% of patients achieved CRc, with 68% of responders attaining molecular minimal residual disease (MRD) negativity [1][3] - In relapsed/refractory NPM1-m AML, the ORR was 65%, and in venetoclax-naïve patients, it increased to 83% [2][11] - For KMT2A-r AML, the ORR was 41%, with 70% in venetoclax-naïve patients [1][11] Safety Profile - The triplet combination of ziftomenib, venetoclax, and azacitidine was well tolerated, with low rates of ziftomenib-related myelosuppression [4][7] - Adverse events included one case of grade 2 differentiation syndrome and one case of grade 3 QTc prolongation, both managed without treatment discontinuation [4][7] Ongoing Development - Kura Oncology is conducting registrational trials for ziftomenib in both intensive chemotherapy-eligible and -ineligible patients [1][2] - The company is also activating sites for pivotal trials, indicating confidence in the drug's potential as a foundational treatment for AML [8][13]