Kura Oncology and Kyowa Kirin Announce First Patient Dosed in Pivotal Phase 3 KOMET-017 Trial of Ziftomenib for Frontline Acute Myeloid Leukemia (AML)
Rhea-AI Summary
Kura Oncology (NASDAQ:KURA) and Kyowa Kirin (TSE:4151) have announced the dosing of the first patient in their pivotal Phase 3 KOMET-017 trial for ziftomenib, an investigational menin inhibitor for frontline Acute Myeloid Leukemia (AML) treatment.
The trial consists of two independent global studies evaluating ziftomenib in combination with both intensive and non-intensive regimens for patients with newly diagnosed NPM1-mutated or KMT2A-rearranged AML. The intensive chemotherapy trial will assess MRD negative CR and EFS as dual-primary endpoints, while the non-intensive trial will evaluate CR and overall survival. The studies are designed to support potential U.S. accelerated and full approval.
The trial will enroll patients across 200 sites worldwide and targets nearly half of newly diagnosed AML patients. This program is notable as the only menin inhibitor actively pursuing registrational trials across both intensive and non-intensive chemotherapy settings.
Positive
- First patient dosed in pivotal Phase 3 trial, marking significant advancement toward potential approval
- Trial design approved by FDA with innovative endpoints that could enable faster approval pathway
- Broad market potential targeting nearly 50% of newly diagnosed AML patients
- Only menin inhibitor program pursuing both intensive and non-intensive chemotherapy settings
Negative
- Complex dual-trial structure could extend development timeline
- Large trial scope across 200 sites may present operational challenges
- Faces competition in developing AML treatments
Insights
Kura and Kyowa Kirin's ziftomenib enters pivotal Phase 3 trials for frontline AML, potentially transforming treatment for this aggressive blood cancer.
The dosing of the first patient in the pivotal Phase 3 KOMET-017 trials represents a significant clinical milestone for ziftomenib, Kura Oncology's investigational menin inhibitor. This development is particularly noteworthy as it advances the compound into frontline treatment for newly diagnosed acute myeloid leukemia (AML) with NPM1 mutations or KMT2A rearrangements, which together represent nearly half of all AML cases.
The trial design is comprehensive, consisting of two independent randomized studies testing ziftomenib in both intensive chemotherapy (with cytarabine/daunorubicin) and non-intensive regimens (with venetoclax/azacitidine). This dual-track approach makes it the only menin inhibitor program actively pursuing registration in both treatment settings, significantly broadening its potential clinical application.
Most importantly, the FDA has permitted the use of surrogate endpoints for potential accelerated approval – MRD negative complete response for the intensive arm and complete response for the non-intensive arm – which could substantially accelerate the regulatory timeline. The inclusion of robust survival endpoints (event-free survival and overall survival) further positions these trials for eventual full approval.
For context, AML remains one of the most challenging hematologic malignancies, with poor outcomes despite current therapies. Targeting patients with specific genetic alterations (NPM1-m and KMT2A-r) with a menin inhibitor is scientifically rational given menin's critical role in these leukemic pathways. The planned enrollment at up to 200 sites worldwide underscores the global interest in this approach and increases the probability of successful execution of this pivotal program.
– KOMET-017-IC trial of intensive chemotherapy combination will assess MRD negative CR and EFS as dual-primary endpoints to support potential U.S. accelerated and full approval –
– KOMET-017-NIC trial of venetoclax / azacitidine combination will assess CR and OS as dual-primary endpoints to support potential U.S. accelerated and full approval –
SAN DIEGO and TOKYO, Sept. 29, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today announced that the first patient has been dosed under the KOMET-017 clinical trial protocol (NCT07007312), comprising two independent, global, randomized double-blind, placebo-controlled Phase 3 trials to evaluate ziftomenib, Kura Oncology’s investigational menin inhibitor, in combination with both intensive and non-intensive combination regimens in patients with newly diagnosed NPM1-mutated (NPM1-m) or KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML).
“The dosing of the first patient under the KOMET-017 protocol is a major milestone in the pursuit of improved treatments for patients with newly diagnosed AML,” said Amer Zeidan, M.B.B.S., M.H.S., Chief of the Division of Hematologic Malignancies, Director of Hematology Early Therapeutics Research at Yale Cancer Center and lead investigator of KOMET-017. “AML remains one of the most aggressive and difficult-to-treat blood cancers, with many patients relapsing despite currently available therapies. Ziftomenib, which in my opinion has the potential to be the best-in-class menin inhibitor, has demonstrated promising safety and activity in early phase clinical trials of NPM1-m and KMT2A-r AML both as monotherapy and in combination with multiple standards of care. These two randomized Phase 3 trials offer the potential to confirm benefit across frontline populations that account for nearly half of newly diagnosed AML patients and where safe, tolerable and effective options are urgently needed.”
“This is a pivotal moment for Kura, Kyowa Kirin, and patients with AML,” said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. “To our knowledge, KOMET-017 is the only menin inhibitor program actively pursuing registrational trials across both intensive and non-intensive chemotherapy settings, underscoring the potential to address a broad spectrum of patients with AML. The opportunity to advance to the frontline AML setting offers the potential to reach patients earlier in their disease course, when the possibility to meaningfully change the trajectory of the disease is greatest. The willingness of the FDA to allow the trials to use MRD negative CR and CR as primary endpoints for accelerated approval is groundbreaking and could potentially enable us to deliver ziftomenib more quickly to patients in need. We are committed to driving the KOMET-017 program forward with the goal of transforming care for patients who continue to face a devastating prognosis.”
“The initiation of the KOMET-017 trial represents a significant step forward in expanding treatment options for newly diagnosed AML patients with NPM1-m and KMT2A-r,” said Takeyoshi Yamashita, Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. “AML remains a disease with poor prognosis, and developing safe and effective therapies is an urgent need. Ziftomenib's innovative mechanism of action, combined with its potential efficacy alongside both intensive and non-intensive therapies, holds promise to improve patients’ quality of life and extend survival. Kyowa Kirin is committed to collaborating closely with Kura Oncology to bring life-changing value to patients living with AML.”
Each frontline trial design includes dual-primary endpoints to support potential U.S. accelerated approval and full approval. The intensive chemotherapy Phase 3 trial of ziftomenib in combination with standard induction cytarabine / daunorubicin (7+3) will assess minimal residual disease (MRD) negative complete response (CR) and event-free survival (EFS) as dual-primary endpoints. The non-intensive chemotherapy Phase 3 trial of ziftomenib in combination with venetoclax / azacitidine will assess CR and overall survival (OS) as dual-primary endpoints. The trial is intended to serve as a registrational study and builds on encouraging clinical data previously reported with ziftomenib in genetically defined subsets of AML. The trial is expected to enroll patients at up to 200 sites worldwide, reflecting strong global interest in advancing ziftomenib for patients in need. More information regarding the KOMET-017 trial is available at www.clinicaltrials.gov (identifier: NCT07007312).
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 of small molecule drug candidates is designed to target cancer signaling pathways and address high-need hematologic malignancies and solid tumors. Kura is developing ziftomenib, a menin inhibitor targeting certain genetic drivers of acute myeloid leukemias, and continues to pioneer advancements in both menin inhibition and farnesyl transferase inhibition to address mechanisms of adaptive and innate resistance in the treatment of solid tumors. 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.
Amer Zeidan, M.D., has consulted for and received honoraria from Kura Oncology and Kyowa Kirin. The opinions expressed represent his personal views and not necessarily those of his employer.
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, among other things, statements regarding the therapeutic potential of ziftomenib in combination with intensive and non-intensive combination regimens in patients in the frontline AML setting; the potential for U.S. accelerated approval and full approval; and expectations regarding trial enrollment. 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, 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 Kura faces, please refer to Kura’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 Contact
Investors and Media:
Greg Mann
858-987-4046
gmann@kuraoncology.com
Kyowa Kirin Contacts
Investors:
Ryohei Kawai
ir@kyowakirin.com
Media, Global:
Nobuyuki Manita
media@kyowakirin.com
FAQ
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