Kura Oncology and Kyowa Kirin Announce First Patient Dosed in Pivotal Phase 3 KOMET-017 Trial of Ziftomenib for Frontline Acute Myeloid Leukemia (AML)
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.
Kura Oncology (NASDAQ:KURA) e Kyowa Kirin (TSE:4151) hanno annunciato la somministrazione del primo farmaco al primo paziente nel loro studio pivotale di fase 3 KOMET-017 per ziftomenib, un inibitore della menina in fase di sperimentazione, destinato al trattamento di prima linea della leucemia mieloide acuta (AML).
Lo studio comprende due studi globali indipendenti che valutano ziftomenib in combinazione con regimi sia intensivi sia non intensivi per pazienti con AML recentemente diagnosticata con mutazione NPM1 o riarrangiamento KMT2A. Lo studio di chemioterapia intensiva valuterà la CR negativa a MRD e l'EF (Event-Free Survival) come endpoint primari doppi, mentre lo studio non intensivo esaminerà la CR e la sopravvivenza globale. I studi sono progettati per supportare potenziali approvazioni accelerate e complete negli Stati Uniti.
Lo studio recluterà pazienti in 200 centri in tutto il mondo e mira a coinvolgere circa metà dei pazienti con AML recentemente diagnosticata. Questo programma è notevole poiché è l’unico inibitore della menina attivamente impegnato in studi registrativi sia in contesti di chemioterapia intensiva sia non intensiva.
Kura Oncology (NASDAQ:KURA) y Kyowa Kirin (TSE:4151) han anunciado el inicio de la administración al primer paciente en su ensayo pivotal de fase 3 KOMET-017 para ziftomenib, un inhibidor de menina en investigación, destinado al tratamiento de primera línea de la leucemia mieloide aguda (LMA).
El ensayo consta de dos estudios globales independientes que evalúan ziftomenib en combinación con regímenes intensivos y no intensivos para pacientes con LMA recientemente diagnosticada con mutación NPM1 o reordenamiento KMT2A. El ensayo de quimioterapia intensiva evaluará CR negativa MRD y EFS como endpoints duales primarios, mientras que el ensayo no intensivo evaluará CR y supervivencia global. Los estudios están diseñados para respaldar posibles aprobaciones aceleradas y completas en EE. UU.
El ensayo reclutará pacientes en 200 sitios en todo el mundo y apunta a cubrir aproximadamente la mitad de los pacientes con LMA recién diagnosticada. Este programa es notable por ser el único inhibidor de menina que persigue activamente ensayos registrables en entornos de quimioterapia tanto intensiva como no intensiva.
Kura Oncology (NASDAQ:KURA)와 Kyowa Kirin (TSE:4151)은 ziftomenib에 대한 3상 KOMET-017의 첫 환자 투여를 발표했습니다. ziftomenib은 전임상에서 개발 중인 멘인 억제제로, 1차 치료로서의 급성 골수성 백혈병(AML) 치료를 목표로 합니다.
이 임상은 전 세계적으로 독립적으로 두 개의 연구로 구성되며, 집중 요법과 비집중 요법 모두를 병용한 ziftomenib를 평가합니다. 새로 진단된 NPM1 돌연변이 또는 KMT2A 재배열 AML 환자들에 대한 연구입니다. 집중 화학요법 연구는 MIS(잔류 질환 음성 CR)와 EFS를 이중 주요 종단점으로 평가하고, 비집중 연구는 CR 및 overall 생존율을 평가합니다. 이 연구들은 미국의 가속 및 전체 승인을 지원하기 위해 설계되었습니다.
전 세계 200개 사이트에서 환자를 모집하며, 새로 진단된 AML 환자의 거의 절반에 도달하는 것을 목표로 합니다. 이 프로그램은 집중 치료 및 비집중 화학요법 환경 모두에서 등록 가능 연구를 적극적으로 추진하는 유일한 멘인 억제제라는 점에서 주목할 만합니다.
Kura Oncology (NASDAQ:KURA) et Kyowa Kirin (TSE:4151) ont annoncé l’administration du premier patient dans leur essai pivot de phase 3 KOMET-017 pour le ziftomenib, un inhibiteur de la menine en développement, destiné au traitement de première ligne de la leucémie myéloïde aiguë (LMA).
L’essai comprend deux études mondiales indépendantes évaluant le ziftomenib en association avec des régimes à la fois intensifs et non intensifs chez des patients atteints de LMA récemment diagnostiquée avec mutation NPM1 ou réarrangement KMT2A. L’étude de chimiothérapie intensive évaluera une CR négative MRD et une EFS comme endpoints primaires doubles, tandis que l’étude non intensive évaluera la CR et la survie globale. Les études sont conçues pour soutenir des approbations potentielles accélérées et complètes aux États‑Unis.
L’essai recrutera des patients sur 200 sites dans le monde et vise à atteindre près de la moitié des patients atteints de LMA récemment diagnostiquée. Ce programme est notable car il est le seul inhibiteur de menine activement engagé dans des essais enregistrables dans des contextes de chimiothérapie à la fois intensifs et non intensifs.
Kura Oncology (NASDAQ:KURA) und Kyowa Kirin (TSE:4151) haben die Verabreichung des ersten Patienten in ihrer pivotalen Phase-3-Studie KOMET-017 für Ziftomenib angekündigt, einen in der Forschung befindlichen Menin-Inhibitor zur Behandlung der ersten Linie bei akuter myeloischer Leukämie (AML).
Die Studie besteht aus zwei unabhängigen globalen Studien, die Ziftomenib in Kombination mit sowohl intensiven als auch nicht-intensiven Behandlungsregimen bei Patienten mit neu diagnostizierter NPM1-mutierter oder KMT2A-Rearrangement-AML evaluieren. Die intensive Chemotherapie-Studie wird MRD-negative CR und EFS als doppelte Primendatenpunkte beurteilen, während die nicht-intensive Studie CR und Gesamtüberleben bewertet. Die Studien sind darauf ausgelegt, potenzielle beschleunigte und vollständige Zulassungen in den USA zu unterstützen.
Die Studie wird Patienten an 200 Standorten weltweit einschließen und zielt darauf ab, fast die Hälfte der neu diagnostizierten AML-Patienten zu erreichen. Dieses Programm ist bemerkenswert, da es der einzige Menin-Inhibitor ist, der aktiv Registrationsstudien in sowohl intensiven als auch nicht-intensiven Chemotherapie-Settings verfolgt.
Kura Oncology (NASDAQ:KURA) و Kyowa Kirin (TSE:4151) أعلنوا عن بدء إعطاء الجرعة لأول مريض في تجربتهما الحاسمة في المرحلة الثالثة KOMET-017 لدواء ziftomenib، وهو مثبِّط للـ menin قيد البحث، لعلاج سرطان الدم النخاعي الحاد كعلاج خط أول.
تتكون التجربة من دراستين عالميتين مستقلّتين تقيمان ziftomenib بالاقتران مع أنظمة علاجية مركّزة وغير مركّزة للمرضى الذين يعانون من AML مُشخّصة حديثاً مع طفرة NPM1 أو إعادة ترتيب KMT2A. ستقيّم دراسة الكيمياء الحرارية المركّزة CR سلبية لـ MRD وEFS كنقاط نهاية أساسية مزدوجة، بينما ستقيّم الدراسة غير المركزة CR والبقاء على قيد الحياة بشكل عام. صممت الدراسات لدعم احتمالات الموافقات الأمريكية السريعة والكاملة.
ستقوم الدراسة بتجنيد مرضى في 200 مركزاً حول العالم وتستهدف نحو نصف مرضى AML المُشخّصين حديثاً. هذا البرنامج ملحوظ كونه مثبِّط menin الوحيد الذي يسعى بنشاط لإجراء تجارب تسجيلية في بيئات العلاج الكيميائي العالية والاستراحة (غير العالية) على حد سواء.
Kura Oncology (NASDAQ:KURA) 与 Kyowa Kirin (TSE:4151) 已宣布在他们的关键性 III 期 KOMET-017 试验中为 ziftomenib 给第一位患者用药，该药物是正在研究中的 Menin 抑制剂，用于一线急性髓系性白血病（AML）治疗。
该试验由两项独立的全球研究组成，评估 ziftomenib 与强化和非强化方案联合使用的效果，针对 新诊断的 NPM1 突变或 KMT2A 重排的 AML 患者。强化化疗研究将以 MRD 阴性 CR 和 EFS 作为双主端点评估，而非强化研究将评估 CR 与总生存期。研究旨在支持美国的加速批准与全面批准的潜在可能性。
该试验将于全球 200 个地点 招募患者，目标覆盖新诊断 AML 患者的近一半。该计划的突出之处在于，它是唯一在强化与非强化化疗环境中都在积极推进注册性试验的 menin 抑制剂。
- 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
- 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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