Kura Oncology and Kyowa Kirin Announce Pivotal Monotherapy Data for Ziftomenib in Oral Presentation at the 2025 ASCO Annual Meeting
- Achieved primary endpoint with statistical significance in the KOMET-001 trial
- Only therapy with Breakthrough Therapy Designation from FDA for R/R NPM1-m AML
- Well-tolerated with limited myelosuppression and low discontinuation rate (3%)
- Addresses an unmet need with no current FDA-approved therapies for NPM1-m AML (30% of AML cases)
- None.
Insights
Kura's ziftomenib trial met primary endpoint with statistical significance, showing promising efficacy in difficult-to-treat NPM1-mutant AML.
The KOMET-001 registration-directed trial data represents a significant milestone for Kura Oncology and Kyowa Kirin's ziftomenib development program. The trial achieved its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh) with statistical significance - the gold standard for efficacy in AML clinical trials.
What makes these results particularly noteworthy is that ziftomenib is the only investigational therapy to receive FDA Breakthrough Therapy Designation for relapsed/refractory NPM1-mutant AML - a designation reserved for treatments demonstrating substantial improvement over available therapies. NPM1 mutations occur in approximately 30% of AML cases, representing a significant patient population currently lacking FDA-approved targeted therapies.
The safety profile appears highly favorable, with limited myelosuppression and only 3% treatment-related discontinuations. This is particularly important in AML, where treatment toxicity often limits therapeutic options, especially in relapsed/refractory settings. The tolerability profile could potentially allow for longer treatment duration and combination approaches.
The selection for oral presentation at ASCO (rather than a poster) further validates the clinical significance of these findings. The companies' plans for commercialization and the upcoming investor event suggest confidence in the data and regulatory pathway forward. For a company focused on targeted oncology therapies, achieving positive pivotal trial results represents a critical value inflection point on the path to potential regulatory submission.
– Results from KOMET-001 registration-directed trial of ziftomenib in R/R NPM1-m AML patients selected for oral presentation on Monday, June 2nd –
– Encore presentation planned at EHA 2025 Congress –
– Kura Oncology to host virtual investor event at 7:30pm ET / 4:30pm PT on June 2nd to discuss the trial results –
SAN DIEGO and TOKYO, May 22, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today announced that an abstract highlighting the full data analyses from the KOMET-001 registration-directed trial of ziftomenib, a once-daily, oral investigational menin inhibitor, has been accepted for oral presentation at the upcoming 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, to be held in Chicago, IL from May 30 - June 3, 2025.
The KOMET-001 registration-directed trial (NCT #04067336) is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib, the only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for treatment of relapsed/refractory (R/R) NPM1-mutant (NPM1-m) acute myeloid leukemia (AML). NPM1 mutations are among the most common, representing approximately
“These data highlight ziftomenib’s potential use as a treatment option for R/R NPM1-mutant AML,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “We are encouraged by the safety and tolerability profile as well as the clinical efficacy observed for this subset of AML patients, and together, Kura and Kyowa Kirin are committed to advancing ziftomenib toward commercialization. We look forward to sharing a more comprehensive dataset at the ASCO Annual Meeting in the coming weeks.”
In addition to the oral presentation, a trial-in-progress abstract for the KOMET-015 trial has been accepted for poster presentation on May 31, 2025. Session titles and information for both abstracts are listed below and are now available on the ASCO.org website. Updated data from the published abstract for KOMET-001 will be disclosed during the oral presentation.
Ziftomenib in Relapsed/Refractory (R/R) NPM1-Mutant Acute Myeloid Leukemia (AML): Phase 1b/2 Clinical Activity and Safety Results from the Pivotal KOMET-001 Study (#6506)
Session: Hematologic Malignancies – Leukemia, Myelodysplastic Syndromes, and Allotransplant
Session Date and Time: Monday, June 2, 2025; 3:00PM - 6:00PM CDT
Presentation Time: 4:36PM - 4:48PM CDT
Location: McCormick Place, S100a
Phase 1a/1b Study of the Safety, Pharmacokinetics, and Antitumor Activity of Ziftomenib in Combination with Imatinib in Patients with Advanced Gastrointestinal Stromal Tumors (GIST) After Imatinib Failure
Session: Poster Session – Sarcoma
Session Date and Time: Saturday, May 31, 2025; 9:00AM - 12:00PM CDT
Location: McCormick Place, Hall A – Posters and Exhibits
Copies of the presentations will be available on Kura's website at www.kuraoncology.com/pipeline/publications/ following presentation at the meeting.
Virtual Investor Event
Kura will host a virtual investor event featuring company management and investigators from the KOMET-001 trial of ziftomenib in R/R NPM1-m AML at 7:30pm ET / 4:30pm PT on Monday, June 2, 2025. Those who would like to participate may access the live webcast here, or register in advance for the teleconference here.The event can also be accessed on the Investors section of Kura’s website at www.kuraoncology.com. An archived replay will be available shortly after the conclusion of the live event.
About Kura Oncology
Kura Oncology is a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer. The Company’s pipeline consists of small molecule drug candidates designed to target cancer signaling pathways. Ziftomenib, a once-daily, oral menin inhibitor, is the first and only investigational therapy to receive BTD from the FDA for the treatment of R/R NPM1-m AML. In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib for AML and other hematologic malignancies. Enrollment in KOMET-001, a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-m AML, has been completed, and the companies submitted a New Drug Application for ziftomenib for the treatment of adult patients with R/R NPM1-m AML in the first quarter of 2025. Kura and Kyowa Kirin are conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-m and KMT2A-rearranged AML. Ziftomenib is also being evaluated in a Phase1 dose-escalation trial (KOMET-015) in combination with imatinib for treatment of patients with advanced GIST. KO-2806, a next-generation farnesyl transferase inhibitor (FTI), is being evaluated in a Phase 1 dose-escalation trial (FIT-001) as a monotherapy and in combination with targeted therapies for patients with various solid tumors. Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial (KURRENT-HN) in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma. For additional information, please visit Kura’s website at https://kuraoncology.com/ and follow us on X and LinkedIn.
About Kyowa Kirin
Kyowa Kirin aims to discover and deliver novel medicines and treatments with life-changing value. As a Japan-based Global Specialty Pharmaceutical Company, Kyowa Kirin has invested in drug discovery and biotechnology innovation for more than 70 years and is currently working to engineer the next generation of antibodies and cell and gene therapies with the potential to help patients with high unmet medical needs, such as bone & mineral, intractable hematological diseases/hemato-oncology and rare diseases. A shared commitment to Kyowa Kirin’s values, to sustainable growth, and to making people smile unites Kyowa Kirin across the globe. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.
Kura Forward-Looking Statements
This news release contains certain forward-looking statements that involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. Such forward-looking statements include statements regarding, among other things, the efficacy, safety and therapeutic potential of ziftomenib; the advancement of ziftomenib toward commercialization as a monotherapy and expansion of its development across diverse settings; potential benefits of combining ziftomenib with intensive chemotherapy and the expected timing and presentation of results and data from clinical trials. Factors that may cause actual results to differ materially include the risk that compounds that appeared promising in early research or clinical trials do not demonstrate safety and/or efficacy in later preclinical studies or clinical trials, the risk that Kura may not obtain approval to market its product candidates, uncertainties associated with performing clinical trials, regulatory filings, and other interactions with regulatory bodies, risks associated with reliance on third parties to successfully conduct clinical trials, the risks associated with reliance on outside financing to meet capital requirements, the risk that the collaboration with Kyowa Kirin is unsuccessful, and other risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. You are urged to consider statements that include the words “may,” “will,” “would,” “could,” “should,” “believes,” “estimates,” “projects,” “promise,” “potential,” “expects,” “plans,” “anticipates,” “intends,” “continues,” “designed,” “goal,” or the negative of those words or other comparable words to be uncertain and forward-looking. For a further list and description of the risks and uncertainties the Company faces, please refer to the Company's periodic and other filings with the Securities and Exchange Commission, which are available at www.sec.gov. Such forward-looking statements are current only as of the date they are made, and Kura assumes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.
Kura Contacts
Investors:
Patti Bank
Managing Director
(415) 513-1284
patti.bank@icrhealthcare.com
Media:
media@kuraoncology.com
Kyowa Kirin Contacts
Investors:
Ryohei Kawai
ir@kyowakirin.com
Media, Global:
Wataru Suzuki,
media@kyowakirin.com
FAQ
What are the key results from KURA's KOMET-001 trial for ziftomenib?
What is the significance of ziftomenib's Breakthrough Therapy Designation for KURA stock?
What is the market opportunity for KURA's ziftomenib in AML treatment?
When will KURA present the full ziftomenib trial results?
