Kura Oncology reports positive data for AML treatment ziftomenib

SAN DIEGO/TOKYO - Kura Oncology, Inc. (NASDAQ:KURA) and Kyowa Kirin Co., Ltd. (TSE:4151), a pharmaceutical company with an $11.67 billion market cap and "FAIR" financial health according to InvestingPro, reported positive updated clinical data from their KOMET-007 trial of ziftomenib combined with standard chemotherapy for newly diagnosed acute myeloid leukemia (AML) patients.

The data, presented Thursday at the European Hematology Association 2025 Congress in Milan, showed high response rates in patients with specific genetic mutations. Among 71 response-evaluable patients, 92% achieved composite complete remission, with 93% effectiveness in NPM1-mutant patients and 89% in KMT2A-rearranged patients. Kyowa Kirin, currently trading near analysts’ price targets and showing strong 9% revenue growth, appears positioned to capitalize on these positive results.

The trial also demonstrated high rates of measurable residual disease negativity: 71% in NPM1-mutant patients and 88% in KMT2A-rearranged patients. Most patients remained alive and on study, with 96% of NPM1-mutant and 88% of KMT2A-rearranged patients continuing treatment.

Ziftomenib’s safety profile was consistent with previous reports. The most common treatment-related adverse events of grade 3 or higher included febrile neutropenia (15%), decreased platelet count (15%), anemia (11%), and decreased neutrophil count (11%).

The companies reported that the combination therapy did not delay blood count recovery compared to standard intensive chemotherapy regimens.

Ziftomenib, an oral menin inhibitor, previously received Breakthrough Therapy Designation from the FDA for relapsed/refractory NPM1-mutant AML. The FDA is currently reviewing a New Drug Application for this indication with a target action date of November 30, 2025. With Kyowa Kirin’s P/E ratio at 29.68 and strong financial metrics, InvestingPro subscribers can access detailed valuation analysis and 12 additional ProTips about the company’s growth potential.

The companies plan to begin randomized Phase 3 studies in the second half of 2025 to further evaluate ziftomenib in combination with both intensive and non-intensive chemotherapy regimens.

Kura Oncology will host a virtual investor event on June 18 to discuss these results and their broader development plans for ziftomenib. Investors tracking Kyowa Kirin should note the company’s next earnings report is scheduled for August 7, 2025, approximately 56 days away.

This article is based on a press release statement from the companies.

In other recent news, Kura Oncology and Kyowa Kirin have submitted a New Drug Application to the U.S. Food and Drug Administration for ziftomenib, a treatment for acute myeloid leukemia with an NPM1 mutation. This submission, made on March 31, 2025, follows the drug’s receipt of Breakthrough Therapy, Fast Track, and Orphan Drug Designations from the FDA. Kura Oncology is awaiting the FDA’s decision on a Priority Review, which could expedite the review process to six months. The KOMET-001 trial, evaluating ziftomenib, met its primary endpoint of complete remission plus partial hematological recovery, showing statistical significance. The trial results, which demonstrated the drug’s tolerability and low discontinuation rate, will be presented at the 2025 American Society of Clinical Oncology Annual Meeting. Kura Oncology plans to host a virtual investor event to discuss these findings further. The company is also exploring the drug’s use in combination with other treatments for newly diagnosed and relapsed/refractory AML patients.

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