Kura Oncology and Kyowa Kirin announced positive topline results from the KOMET-001 Phase 2 trial of ziftomenib, an oral menin inhibitor, in patients with relapsed/refractory NPM1-mutant acute myeloid leukemia (AML). The trial met its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh), with a statistically significant result and an encouraging safety profile. An NDA submission is planned for Q2 2025.
This positive outcome is crucial for AML patients, particularly those with the NPM1 mutation, who face a high risk of relapse and limited treatment options. The achievement of the primary endpoint suggests ziftomenib could offer a new therapeutic avenue for this patient population, potentially improving remission rates and overall survival. The upcoming NDA submission signifies a significant step towards bringing this potential treatment to market.
The KOMET-001 trial achieved its primary endpoint of CR/CRh, demonstrating the efficacy of ziftomenib as a monotherapy in this difficult-to-treat patient population. Furthermore, the companies received positive FDA feedback on the design of the upcoming KOMET-017 Phase 3 trial, which will evaluate ziftomenib in combination with standard therapies in both newly diagnosed and relapsed/refractory NPM1-mutant and KMT2A-rearranged AML patients. This trial incorporates dual primary endpoints (MRD-negative CR and event-free survival for the intensive arm; CR and overall survival for the non-intensive arm) designed to support potential accelerated and full approvals in the US. The companies also announced additional data presentations anticipated throughout 2025 related to ziftomenib and other pipeline programs focusing on other cancers and treatment combinations.
The positive KOMET-001 results and planned KOMET-017 trial position ziftomenib as a potential game-changer in the AML treatment landscape. If approved, ziftomenib could become a key therapy for patients with NPM1-mutant and KMT2A-rearranged AML, both in the relapsed/refractory and newly diagnosed settings. This could significantly improve patient outcomes and address a substantial unmet medical need in this challenging disease. The upcoming NDA submission and initiation of the Phase 3 trial will be critical milestones to watch, as they will further define the role of ziftomenib in the future of AML treatment.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
