Kura Oncology Completes Enrollment in Registration-Directed Trial of Ziftomenib in NPM1-Mutant AML

Company Overview - Kura Oncology, Inc. is a clinical-stage biopharmaceutical company focused on precision medicines for cancer treatment [7] - The company's lead investigational drug, ziftomenib (KO-539), is a menin inhibitor targeting relapsed or refractory NPM1-mutant acute myeloid leukemia (AML) [5][7] Clinical Trial Progress - Kura has completed enrollment of 85 patients in the Phase 2 portion of the KOMET-001 trial in under 16 months [1] - Topline data from the trial is expected to be reported in early 2025 [1][2] - The trial aims to assess the clinical activity, safety, and tolerability of ziftomenib, with a primary endpoint of complete response [2] Breakthrough Therapy Designation - Ziftomenib has received Breakthrough Therapy Designation from the FDA, which facilitates expedited review for its potential as an innovative treatment for R/R NPM1-mutant AML [2][5] - This designation underscores the urgent need for effective treatment options in AML, particularly for patients with NPM1 mutations, which account for approximately 30% of new AML cases annually [3][4] Market Need and Patient Prognosis - NPM1-mutant AML has a high unmet medical need, with no approved targeted therapies currently available [3][4] - Patients with R/R NPM1-mutant AML have poor survival outcomes, with only 30% overall survival at 12 months in the R/R setting [4] - The median overall survival for patients with NPM1-m AML is approximately 7.8 months in the second line, 5.3 months in the third line, and 3.5 months following the fourth line of treatment [4] Ziftomenib's Clinical Profile - In the Phase 1 study, ziftomenib demonstrated a favorable safety profile and a 35% complete remission rate at the recommended Phase 2 dose of 600 mg [5] - The drug is designed to be taken once daily and targets the menin-KMT2A/MLL protein-protein interaction [5][6]