Kura Oncology and Kyowa Kirin Report Positive Updated Combination Data for Ziftomenib in Newly Diagnosed AML at 2025 European Hematology Association Congress

Rhea-AI Summary

Kura Oncology and Kyowa Kirin reported positive Phase 1a/1b trial results for ziftomenib combined with 7+3 chemotherapy in newly diagnosed AML patients. The KOMET-007 trial showed impressive complete remission rates of 93% in NPM1-mutant and 89% in KMT2A-rearranged AML patients. Notable MRD-negativity rates were achieved: 71% in NPM1-m and 88% in KMT2A-r patients. The treatment demonstrated favorable safety with no additive myelosuppression. High survival rates were observed with 96% of NPM1-m and 88% of KMT2A-r patients remaining alive and on study. Phase 3 studies (KOMET-017-IC and NIC) are planned to start in second half of 2025. The data suggests ziftomenib's potential as a frontline treatment option for AML patients.

Positive

• High complete remission rates: 93% for NPM1-m and 89% for KMT2A-r AML patients
• Strong MRD-negativity rates: 71% for NPM1-m and 88% for KMT2A-r patients
• Excellent survival rates with 96% NPM1-m and 88% KMT2A-r patients remaining alive
• Well-tolerated safety profile with no dose-limiting toxicities or drug-drug interactions
• Rapid progression to Phase 3 trials planned for 2H 2025

Negative

• Median duration of CR for KMT2A-r patients was relatively short at 25.6 weeks
• Some Grade 3 adverse events reported including febrile neutropenia (15%) and decreased platelet count (15%)
• Two cases of QTc prolongation reported in KMT2A-r patients

Insights

Oncology Research Specialist

Kura Oncology's ziftomenib shows impressive 89-93% response rates in AML patients with specific mutations, demonstrating strong potential as first-line treatment.

The clinical data presented at EHA2025 for ziftomenib in combination with standard chemotherapy (7+3) represents a significant advancement in the treatment landscape for acute myeloid leukemia (AML). The results are particularly impressive for a targeted population with specific genetic markers - NPM1 mutations and KMT2A rearrangements, which collectively represent approximately 30-40% of AML cases.

The efficacy metrics are remarkably strong: 93% and 89% composite complete remission rates in NPM1-mutant and KMT2A-rearranged patients, respectively. Even more compelling is the depth of these responses, with 71% and 88% of responding patients achieving measurable residual disease (MRD) negativity. In the context of AML treatment, MRD negativity is a critical predictor of long-term outcomes and potential cure.

The high survival rates - 96% of NPM1-mutant and 88% of KMT2A-rearranged patients remaining alive and on study - are especially noteworthy given that median follow-up times were 24.9 and 15.7 weeks, respectively. The median duration of complete remission and overall survival had not been reached for NPM1-mutant patients, suggesting durable responses.

From a safety perspective, the absence of additive myelosuppression is particularly important. Traditional AML therapies often cause profound bone marrow suppression, leading to life-threatening complications. The fact that ziftomenib didn't delay neutrophil and platelet recovery compared to standard intensive chemotherapy suggests it could be safely integrated into existing treatment protocols without exacerbating toxicity.

The advancement to Phase 3 trials (KOMET-017-IC and NIC) expected in the second half of 2025 represents a critical milestone in the drug's development pathway. If these larger randomized studies confirm the current findings, ziftomenib could potentially transform the standard of care for genetically-defined AML subtypes, addressing the high relapse rates (up to 70%) currently observed even after initial complete responses with existing therapies.

– Encouraging clinical activity with deep responses demonstrated in the KOMET-007 trial with the combination of 600 mg ziftomenib with 7+3 in newly diagnosed patients with NPM1-m and KMT2A-r AML –

– 93% (41/44) and 89% (24/27) CRc observed in NPM1-m and KMT2A-r AML response-evaluable patients, respectively –

– 71% (24/34) and 88% (14/16) CR measurable residual disease (MRD)-negativity observed among responding NPM1-m and KMT2A-r AML patients, respectively –

– 96% (47/49) of NPM1-m and 88% (29/33) KMT2A-r AML patients remain alive and continue on-study –

– Combination was well tolerated with no additive myelosuppression –

– KOMET-017-IC (intensive chemotherapy) and NIC (non-intensive chemotherapy) randomized phase 3 studies (NCT07007312) expected to start in 2H 2025 –

– Kura Oncology to host virtual investor event to discuss results and broader ziftomenib development plan on June 18, 2025 at 4:30pm ET / 1:30pm PT –

SAN DIEGO and TOKYO, June 12, 2025 (GLOBE NEWSWIRE) --  Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today provided positive updated clinical data from KOMET-007, a Phase 1a/1b trial of ziftomenib, a highly selective oral investigational menin inhibitor, in combination with standards of care in patients with newly diagnosed NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML). The data for the combination with cytarabine/daunorubicin (7+3) were presented as an oral presentation at the European Hematology Association 2025 Congress (EHA2025) being held in Milan, Italy from June 12-15, 2025.

“The findings presented at EHA2025 underscore the potential of ziftomenib in combination with 7+3 as an early intervention in the frontline setting of AML and could offer a meaningful opportunity to improve patient outcomes,” said Harry Erba, M.D., Ph.D., Director of the Leukemia Program at the Duke Cancer Institute. “The high rates of complete remission and MRD negativity across the 7+3 cohorts are particularly encouraging. The continued rapid enrollment in the Phase 1b portion of this study underscores the urgency and enthusiasm for further evaluating this combination approach.”

“We remain very encouraged by the updated clinical activity, safety and tolerability data from the KOMET-007 study evaluating ziftomenib with 7+3 in newly diagnosed AML patients with NPM1 mutations or KMT2A rearrangements,” said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. “These updated data reinforce the combination potential of ziftomenib in the frontline setting, strengthening our confidence in its ability to provide a valuable treatment option for a significant portion of the AML population. We and our partners at Kyowa Kirin are working in earnest to prepare for the KOMET-017-IC and NIC pivotal Phase 3 studies, which will enable us to test ziftomenib-based combinations and their potential, if approved, to transform care for AML patients worldwide.”

In the ongoing study, ziftomenib dosed once daily at 600 mg in combination with 7+3 continued to demonstrate robust and evolving clinical activity in patients with newly diagnosed AML. Among 71 response-evaluable patients, 92% (65/71) achieved a composite complete remission (CRc) (93% for NPM1-m, 89% for KMT2A-r patients) and 80% (57/71) achieved a complete remission (CR) (84% for NPM1-m, 74% for KMT2A-r patients) at the time of data cutoff. A rate of CR minimal residual disease (CR-MRD) negativity of 71% for NPM1-m with a median time to MRD negativity of 4.7 weeks and a rate of CR-MRD negativity of 88% for KMT2A-r patients with a median time to MRD negativity of 4.4 weeks were observed. Ziftomenib did not delay time to neutrophil and platelet count recovery, which was comparable to intensive chemotherapy regimens.

Median follow-up times for the two populations were 24.9 weeks (range 4.3-47.1) in NPM1-m patients and 15.7 weeks (range 1.1-40.3) in KMT2A-r patients. Among response-evaluable NPM1-m patients, neither a median duration of CR nor a median overall survival (OS) had been reached. Among response-evaluable KMT2A-r patients, a median duration of CR was determined to be 25.6 weeks (95% CI, range 8.3-NE), and a median OS had not been reached. Notably, 96% (47/49) of NPM1-m patients and 88% (29/33) of KMT2A-r patients remained alive and on study.

The safety population included 82 newly diagnosed adult patients with NPM1-m or KMT2A-r AML from the pooled Phase 1a/1b portions of the trial at the 600 mg QD dose of ziftomenib. The safety profile observed with ziftomenib was consistent with previously reported data. Ziftomenib-related adverse events (TRAEs) of ≥ Grade 3 (Gr3), which occurred in more than 10% of patients were febrile neutropenia (15%), decreased platelet count (15%), anemia (11%) and decreased neutrophil count (11%). One case of differentiation syndrome (KMT2A-r, Gr3) was successfully managed by protocol-specified mitigation strategies. Two cases of investigator-assessed QTc prolongation (both KMT2A-r, Gr3) were reported; both patients were on other medications (posaconazole and/or piperacillin/tazobactam), which have been identified as potentially causing QT prolongation at the time of QT assessment. No dose-limiting toxicities, drug-drug interactions, clinically meaningful ziftomenib-associated QTc prolongation or additive myelosuppression were observed.

“Despite the availability of approved therapies for AML, up to 70% of patients who initially achieve a complete response relapse within three years – highlighting a substantial unmet need,” said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. “The data presented at EHA2025 suggest a favorable safety, tolerability, and efficacy profile for ziftomenib. We are encouraged by its potential as a future first-line treatment option and are committed to advancing the KOMET-017 Phase 3 trial, expected to begin later this year, to further evaluate its value in AML care.”

The EHA2025 oral presentation highlighting ziftomenib combined with 7+3 in newly diagnosed (1L) NPM1-m and KMT2A-r AML, and an encore presentation of results from the KOMET-001 registrational trial of ziftomenib in relapsed/refractory (R/R) NPM1-m AML (also presented during EHA2025) are available in the Posters and Presentations section of the Kura website. The KOMET-017 protocol consists of 2 separate Phase 3 studies, which will investigate the benefits and risks of adding ziftomenib to standards of care treatments in patients newly diagnosed NPM1-m or KMT2A-r AML and which is registered at www.clinicaltrials.gov as NCT07007312.

Virtual Investor Event

Kura will host a virtual investor event featuring company management and investigators from the KOMET-007 trial of ziftomenib in combination with 7+3 in patients with NPM1-m and KMT2A-r AML at 4:30pm ET / 1:30pm PT on Wednesday, June 18, 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 Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (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 in the second quarter of 2025, the companies announced the FDA’s acceptance of a New Drug Application for ziftomenib for the treatment of adult patients with R/R NPM1-m AML and assignment of a Prescription Drug User Fee Act target action date of November 30, 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 Phase 1 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 the Kura 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; potential benefits of combining ziftomenib with intensive chemotherapy and other standards of care for AML; progress of the ziftomenib program and clinical trials, including the KOMET-017-IC pivotal Phase 3 study; and the potential for ziftomenib to obtain regulatory approval. 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 complete remission rates for ziftomenib in the KOMET-007 trial for AML patients?

The trial showed 93% complete remission rates for NPM1-mutant AML patients and 89% for KMT2A-rearranged AML patients when combined with 7+3 chemotherapy.

What are the main side effects reported for ziftomenib in the KURA clinical trial?

The main Grade 3 adverse events included febrile neutropenia (15%), decreased platelet count (15%), anemia (11%), and decreased neutrophil count (11%).

When will KURA start Phase 3 trials for ziftomenib?

Kura Oncology plans to start the KOMET-017-IC and NIC Phase 3 studies in the second half of 2025.

What is the survival rate for patients treated with ziftomenib in the KOMET-007 trial?

The trial showed 96% of NPM1-mutant patients and 88% of KMT2A-rearranged patients remained alive and on study.

What is the MRD-negativity rate for ziftomenib in AML treatment?

The trial achieved 71% MRD-negativity in NPM1-mutant patients and 88% MRD-negativity in KMT2A-rearranged patients.