Kura Oncology and Kyowa Kirin Report Positive Phase 2 Results for Ziftomenib in AML, Prepare for FDA Submission
Kura Oncology and Kyowa Kirin announced positive topline results from the KOMET-001 Phase 2 registration-directed trial of ziftomenib in patients with relapsed/refractory NPM1-mutant acute myeloid leukemia (AML)16.
The KOMET-001 trial achieved its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh), with statistical significance1.
Ziftomenib's safety and tolerability profile was consistent with previous reports1.
Kura plans to submit a New Drug Application (NDA) to the FDA for ziftomenib in the second quarter of 2025110.
Full results from the KOMET-001 trial will be presented at a medical meeting in the second quarter of 20251.
Ziftomenib is the only investigational therapy to receive Breakthrough Therapy Designation from the FDA for treatment of relapsed/refractory NPM1-mutant AML19.
Kura and Kyowa Kirin plan to initiate two frontline Phase 3 trials (KOMET-017-IC and KOMET-017-NIC) in the second half of 2025, evaluating ziftomenib in combination with intensive and non-intensive regimens1.
The companies formed a global strategic collaboration in 2024 to develop and commercialize ziftomenib, with Kyowa Kirin providing $330 million upfront and up to $1.1 billion in potential milestones4.
Specific data from the KOMET-001 trial were not disclosed in the announcement3.
Ziftomenib is being developed as a potential treatment for up to 50% of patients with AML1.
Sources:
1. https://www.globenewswire.com/news-release/2025/02/05/3021565/0/en/Kura-Oncology-and-Kyowa-Kirin-Announce-Positive-Ziftomenib-Monotherapy-Registrational-Trial-and-Positive-FDA-Feedback-for-Upcoming-Frontline-Combination-Trial-Designs.html
3. https://endpts.com/kuras-aml-drug-succeeds-in-advanced-patients-will-take-to-fda/
4. https://www.fiercebiotech.com/biotech/kyowa-kirin-fronts-330m-share-rights-kuras-leukemia-drug-ahead-push-fda
6. https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-announce-positive-ziftomenib
9. https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-announce-global-strategic