Kura Oncology’s ziftomenib meets primary endpoint in AML study

Kura Oncology (KURA) and Kyowa Kirin announced the Journal of Clinical Oncology published the full results from the pivotal KOMET-001 clinical trial evaluating ziftomenib, an investigational, once-daily, oral menin inhibitor, in adult patients with relapsed/refractory NPM1-mutated acute myeloid leukemia, or AML.There is currently no approved therapy to specifically target NPM1-m AML. Ziftomenib is currently under priority review by the FDA for treatment of R/R NPM1-m AML. The publication includes data from patients with R/R NPM1-m AML in the phase 2 portion of the trial. The KOMET-001 phase 2 trial met its primary endpoint with a complete remission with full or partial hematologic recovery rate of 22%, significantly higher than the 12% historical standard-of-care response rate for patients with R/R NPM1-m AML. 61% of evaluable CR/CRh responders were negative for measurable residual disease. Overall response rate was 33%. Median overall survival was 6.6 months. Ziftomenib was well tolerated with a safety profile consistent with previously disclosed data. Three patients discontinued treatment because of ziftomenib-related adverse events. These findings formed part of the data set used for the NDA for ziftomenib as a potential treatment for patients with R/R NPM1-m AML. The FDA target action date is November 30.