Kura Oncology and Kyowa Kirin Report Positive Pivotal Ziftomenib Monotherapy Data at 2025 ASCO Annual Meeting

Core Insights - Kura Oncology and Kyowa Kirin announced positive pivotal results from the KOMET-001 Phase 2 trial of ziftomenib for relapsed/refractory NPM1-mutant AML patients, with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% [1][3][2] - The trial demonstrated consistent efficacy across various pre-specified subgroups, indicating the potential of ziftomenib as a treatment option for heavily pre-treated patients [1][4][2] - Ziftomenib has a favorable safety profile, with limited myelosuppression and a low discontinuation rate due to treatment-related adverse events [1][5][7] Company Overview - Kura Oncology is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment, with ziftomenib being the first investigational therapy to receive Breakthrough Therapy Designation from the FDA for R/R NPM1-m AML [9] - The company has a strategic collaboration with Kyowa Kirin to develop and commercialize ziftomenib, with ongoing clinical trials evaluating its efficacy in combination with current standards of care [9][10] Clinical Trial Details - The KOMET-001 Phase 2 trial included 92 adult patients with a median age of 69, with 33% having received three or more prior lines of therapy [2][3] - Among the patients achieving CR/CRh, 63% were minimal residual disease (MRD)-negative, highlighting the treatment's potential effectiveness [3][4] Safety and Tolerability - The safety profile of ziftomenib showed that treatment-related adverse events led to discontinuation in only 3% of patients, with differentiation syndrome being the most common adverse event [5][6][7] - The absence of clinically significant QTc prolongation and effective management of differentiation syndrome further support ziftomenib's favorable benefit-risk profile [7][6] Future Outlook - The FDA has set a PDUFA target action date of November 30, 2025, for the New Drug Application (NDA) of ziftomenib, indicating a potential first approval for a once-daily oral menin inhibitor for this patient population [1][2] - Kura Oncology is hosting a virtual investor event to discuss the trial results and future plans for ziftomenib [8]