Kura Oncology and Kyowa Kirin Announce Positive Ziftomenib Monotherapy Registrational Trial and Positive FDA Feedback for Upcoming Frontline Combination Trial Designs

Core Insights - Kura Oncology and Kyowa Kirin announced positive topline results from the KOMET-001 trial for ziftomenib in R/R NPM1-mutant AML, achieving the primary endpoint of complete response (CR) plus CR with partial hematological recovery (CRh) [1][4][5] - The companies are on track to submit a New Drug Application (NDA) for ziftomenib to the FDA in the second quarter of 2025 [1][5] - Two Phase 3 trials, KOMET-017-IC and KOMET-017-NIC, are expected to initiate in the second half of 2025 to evaluate ziftomenib in combination with different chemotherapy regimens [2][8] Company Developments - Kura and Kyowa Kirin's collaboration aims to commercialize ziftomenib, with plans for two registrational Phase 3 trials to support potential accelerated approval [2][8] - The KOMET-001 trial is the only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the FDA for R/R NPM1-mutant AML [5][16] - Kura's management expressed confidence in ziftomenib's potential to transform treatment for AML patients, particularly given the positive FDA interactions [3][11] Clinical Trial Insights - The KOMET-017-IC trial will assess ziftomenib combined with intensive chemotherapy, while the KOMET-017-NIC trial will evaluate ziftomenib with venetoclax and azacitidine for patients unfit for intensive chemotherapy [9][10] - Both trials will have dual-primary endpoints to support potential U.S. accelerated approval and full approval [7][10] - The focus on achieving minimum residual disease (MRD) negative CR as a primary endpoint is seen as innovative and may expedite therapy availability [12][13] Market Context - AML is a challenging blood cancer with a high relapse rate, affecting approximately 20,000 Americans annually, with significant unmet medical needs in the treatment landscape [15] - The 5-year survival rate for AML is reported at 31.9%, highlighting the urgency for effective therapies [11][15] - Ziftomenib is positioned to address a substantial portion of AML patients, particularly those with NPM1 mutations [3][11]