Kura Oncology and Kyowa Kirin have published full results from a pivotal phase 2 trial evaluating ziftomenib, an investigational, once-daily, oral menin inhibitor, in adult patients with relapsed/refractory NPM1 mutated (NPM1-m) acute myeloid leukemia (AML).
Survival outcome for NPM1-m AML is poor in cases of relapse, and there is no treatment currently available to specifically target the condition. Kyowa Kirin inked a potential $1.16 billion deal with Kura last year to develop ziftomenib, which is now currently under FDA priority review. The application has a November 30, 2025 PDUFA date.
In the KOMET-001 phase 2 clinical trial, ziftomenib monotherapy met the primary endpoint. The 22% complete remission with full or partial hematologic recovery was significantly higher than the historical standard of care response rate. The drug also showed consistent activity across all the subgroups tested and had a favorable safety and tolerability profile with no clinically-significant QTc prolongation and low rates of myelosuppression and treatment discontinuation.