Kura Oncology and Kyowa Kirin Announce Publication of Pivotal Ziftomenib Data in Relapsed/Refractory NPM1-Mutated AML in the Journal of Clinical Oncology
Kura Oncology (NASDAQ:KURA) and Kyowa Kirin announced the publication of pivotal KOMET-001 trial results for ziftomenib in the Journal of Clinical Oncology. The trial, evaluating this oral menin inhibitor in relapsed/refractory NPM1-mutated AML patients, met its primary endpoint with a CR/CRh rate of 22%, significantly exceeding the 12% historical standard.
Key findings include a 33% overall response rate, median overall survival of 6.6 months, and 61% MRD-negative rate among CR/CRh responders. The drug showed consistent efficacy across subgroups and demonstrated a favorable safety profile. The FDA has set a PDUFA date of November 30, 2025 for priority review.
[ "Met primary endpoint with CR/CRh rate of 22%, significantly higher than 12% historical standard", "61% of CR/CRh responders achieved MRD-negative status", "Favorable safety profile with low discontinuation rate (3%)", "Consistent efficacy across patient subgroups regardless of prior treatments", "FDA granted Priority Review with PDUFA date set for November 30, 2025" ]Kura Oncology (NASDAQ:KURA) e Kyowa Kirin hanno annunciato la pubblicazione dei risultati chiave dello studio KOMET-001 per ziftomenib nel Journal of Clinical Oncology. Lo studio, che valuta questo inibitore orale della menina in pazienti con AML recidivante o refractory e con mutazione NPM1, ha raggiunto il suo endpoint primario con un tasso CR/CRh del 22%, superando significativamente lo standard storico del 12%.
I principali risultati includono una risposta globale del 33%, una sopravvivenza globale mediana di 6,6 mesi e un tasso MRD-negativo del 61% tra i responder CR/CRh. Il farmaco ha mostrato efficacia coerente tra i sottogruppi e un profilo di sicurezza favorevole. La FDA ha fissato una data PDUFA del 30 novembre 2025 per una revisione prioritaria.
Kura Oncology (NASDAQ:KURA) y Kyowa Kirin anunciaron la publicación de los resultados principales del ensayo KOMET-001 para ziftomenib en el Journal of Clinical Oncology. El ensayo, que evalúa este inhibidor oral de menina en pacientes con AML mutado en NPM1 y en recaída/refractaria, cumplió su objetivo primario con una tasa CR/CRh del 22%, superando ampliamente el 12% del estándar histórico.
Entre los hallazgos clave se destacan una tasa de respuesta global del 33%, una mediana de supervivencia global de 6,6 meses y un 61% de MRD-negativo entre los respondedores CR/CRh. El fármaco mostró eficacia consistente en subgrupos y un perfil de seguridad favorable. La FDA ha establecido una fecha PDUFA de 30 de noviembre de 2025 para una revisión prioritaria.
Kura Oncology (NASDAQ:KURA)와 Kyowa Kirin은 Journal of Clinical Oncology에 지프트로늄의 중요한 KOMET-001 시험 결과를 발표했습니다. 이 구강형 멘인 억제제의 재발/난치성 NPM1 변이 AML 환자를 대상으로 한 이 임상은 주요 1차 평가변수인 CR/CRh 비율 22%를 달성했으며, 역사적 표준인 12%를 크게 상회했습니다.
핵심 발견으로는 전반 반응률 33%, 중간 전체 생존 6.6개월, CR/CRh 반응자 중 MRD 음성 비율 61%이 포함됩니다. 약물은 하위군 간 일관된 효능을 보였고 안전성 프로파일도 우수했습니다. FDA는 우선 심사를 위한 PDUFA 예정일 2025년 11월 30일를 정했습니다.
Kura Oncology (NASDAQ:KURA) et Kyowa Kirin ont annoncé la publication des résultats pivotants de l’essai KOMET-001 pour ziftomenib dans le Journal of Clinical Oncology. L’essai, évaluant cet inhibiteur oral de la menine chez des patients atteints d’AML réfractaire/relapé avec mutation NPM1, a atteint son objectif principal avec un taux CR/CRh de 22%, dépassant largement le standard historique de 12%.
Parmi les résultats clés figurent un taux de réponse global de 33%, une survie globale médiane de 6,6 mois et un taux MRD-négatif de 61% parmi les répondants CR/CRh. Le médicament a montré une efficacité cohérente dans les sous-groupes et un profil de sécurité favorable. La FDA a fixé une date PDUFA au 30 novembre 2025 pour un examen prioritaire.
Kura Oncology (NASDAQ:KURA) und Kyowa Kirin gaben die Veröffentlichung der wichtigsten KOMET-001-Studienergebnisse für ziftomenib im Journal of Clinical Oncology bekannt. Die Studie bewertet diesen oralen Menin-Inhibitor bei relapsiertem/refraktärem AML mit NPM1-Mutation und erreichte den primären Endpunkt mit einer CR/CRh-Rate von 22%, deutlich über dem historischen Standard von 12%.
Wesentliche Ergebnisse umfassen eine gesamte Ansprechrate von 33%, eine mediane Gesamtüberleben von 6,6 Monaten und eine MRD-negative-Rate von 61% unter CR/CRh-Respondern. Das Medikament zeigte konsistente Wirksamkeit über Untergruppen hinweg und ein günstiges Sicherheitsprofil. Die FDA hat einen PDUFA-Termin am 30. November 2025 für eine Prioritätsprüfung festgelegt.
Kura Oncology (NASDAQ:KURA) و Kyowa Kirin أعلنا عن نشر نتائج تجربة KOMET-001 الحاسمة لـ ziftomenib في مجلة Journal of Clinical Oncology. التجربة التي تقوّم مثبِّط المينين الفموي في مرضى AML المتكرر/المقاوم مع طفرة NPM1، حققت الهدف الأول بمعدل CR/CRh 22%، وهو أعلى بكثير من المعيار التاريخي البالغ 12%.
من النتائج الأساسية: معدل استجابة إجمالية قدره 33%، ومتوسط البقاء على قيد الحياة الإجمالي 6.6 أشهر، ومعدل MRD سلبي 61% بين المستجيبين CR/CRh. الدواء أظهر فاعلية ثابتة عبر المجموعات الفرعية وبروفيل أمان متوازن. أصدرت FDA تاريخ PDUFA> للمراجعة الأولية يوم 30 نوفمبر 2025.
Kura Oncology（纳斯达克股票代码：KURA） 与 Kyowa Kirin 公布了 ziftomenib 在 Journal of Clinical Oncology 上的关键 KOMET-001 试验结果。该试验评估这款口服的孟宁抑制剂在复发/难治性 NPM1 突变的 AML 患者中的应用，达到了主要终点，CR/CRh 比率为22%，显著高于历史标准的12%。
主要发现包括：总体应答率33%、中位总体生存期6.6个月、以及在 CR/CRh 反应者中< b>61% MRD 阈值阴性。该药在各亚组中均显示出一致的疗效，安全性也令人乐观。FDA 已将优先审评日期定为 2025 年 11 月 30 日的 PDUFA 日期。
- None.
- Median duration of overall response limited to 4.6 months
- 25% of patients experienced differentiation syndrome
- Significant adverse events included febrile neutropenia (26%), anemia (20%), and thrombocytopenia (20%)
Insights
Ziftomenib shows promising efficacy and safety in treating NPM1-mutated AML, with FDA decision expected by November 2025.
The KOMET-001 trial results for ziftomenib represent a significant advancement in treating relapsed/refractory NPM1-mutated acute myeloid leukemia (AML), a condition with poor survival outcomes after relapse. The trial met its primary endpoint with a 22% complete remission rate, significantly exceeding the 12% historical standard-of-care response rate. The overall response rate reached 33%, with 61% of responders achieving measurable residual disease negativity—indicating deep molecular responses.
Particularly noteworthy is ziftomenib's consistent efficacy across subgroups regardless of prior treatments, including venetoclax exposure, previous stem cell transplantation, or presence of co-mutations like FLT3 or IDH1/2. This suggests broad applicability within the NPM1-mutated population.
The survival data further validates ziftomenib's potential clinical impact. Overall survival reached 6.6 months for all patients, but jumped dramatically to 18.4 months in responders versus just 3.5 months in non-responders. This five-fold survival increase in responders demonstrates meaningful clinical benefit.
The safety profile appears favorable for a late-line AML therapy. While common hematologic toxicities occurred (febrile neutropenia, anemia, thrombocytopenia), the absence of clinically significant QTc prolongation and low myelosuppression rates are advantages over many existing AML therapies. The 3% discontinuation rate due to treatment-related adverse events is remarkably low for this patient population. The manageable differentiation syndrome, seen in 25% of patients, aligns with expectations for this drug class.
With FDA priority review underway and a decision expected by November 30, 2025, ziftomenib represents a potential targeted therapy addressing a significant unmet need in NPM1-mutated AML.
– KOMET-001 in R/R NPM1-m AML met its primary endpoint with ziftomenib monotherapy demonstrating significant clinical benefit and deep responses –
– Consistent activity across pre-specified subgroups, regardless of prior HSCT, prior venetoclax, lines of therapy, or FLT3/IDH co-mutations –
– Favorable safety and tolerability profile, including a lack of clinically significant QTc prolongation or myelosuppression –
– Prescription Drug User Fee Act (PDUFA) target action date for FDA Priority Review of ziftomenib in adults with R/R NPM1-m AML set for November 30, 2025 –
SAN DIEGO and TOKYO, Sept. 25, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today announced the Journal of Clinical Oncology published the full results from the pivotal KOMET-001 clinical trial (NCT04067336) evaluating ziftomenib, an investigational, once-daily, oral menin inhibitor, in adult patients with relapsed/refractory (R/R) NPM1-mutated (NPM1-m) acute myeloid leukemia (AML). Although newly diagnosed patients with NPM1-m AML have high response rates to approved standard of care, relapses are common and survival outcomes are poor. There is currently no approved therapy to specifically target NPM1-m AML. Ziftomenib is currently under priority review by the Food and Drug Administration (FDA) for treatment of R/R NPM1-m AML.
“Relapsed or refractory NPM1-mutated AML remains very challenging to treat, particularly after venetoclax-based therapy or transplant,” said Eunice Wang, M.D., Chief of Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center, Buffalo, NY. “The manuscript describes deep responses, signals of clinical activity across relevant subgroups and a generally manageable tolerability profile, which is important in treatment of late line AML patients where accumulated toxicity can limit treatment options. The benefit-risk profile of ziftomenib is highly encouraging and, if replicated in additional treatment settings, has potential to be transformative for a large population of patients with menin pathway-driven AML.”
“Publication in ASCO’s Journal of Clinical Oncology is an important advancement for adult patients with NPM1-m AML,” said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. “In addition to evidence of monotherapy activity, the safety and tolerability profile of ziftomenib from this trial is encouraging, marked by the absence of clinically meaningful QTc prolongation as well as low rates of both myelosuppression and treatment discontinuation. No clinically meaningful drug-drug interactions were observed, including with commonly used supportive-care medications, which may simplify co-administration in a polypharmacy setting. We continue to conduct studies in earlier line settings and in combination with multiple therapeutic agents in close collaboration with investigators, study teams and our partner Kyowa Kirin.”
Summary of the published data
The publication, entitled “Ziftomenib in Relapsed or Refractory NPM1-Mutated AML”, includes positive data from 92 adult patients with R/R NPM1-m AML in the phase 2 portion of the clinical trial as of the primary data cutoff date of October 28, 2024.
The KOMET-001 phase 2 trial met its primary endpoint with a complete remission with full or partial hematologic recovery (CR/CRh) rate of
Median overall survival (OS) was 6.6 months (
Ziftomenib was well tolerated with a safety profile consistent with previously disclosed data. The most common grade ≥3 treatment-emergent adverse events were febrile neutropenia (
These findings formed part of the data set used for the New Drug Application for ziftomenib as a potential treatment for patients with R/R NPM1-m AML. The FDA target action date is November 30, 2025. There is currently no FDA-approved treatment for patients with R/R NPM1-m AML.
“The publication of the investigational ziftomenib data adds important scientific context for clinicians and patients,” said Takeyoshi Yamashita, Ph. D., Executive Vice President and Chief Medical Officer, Kyowa Kirin. “Together with Kura Oncology, we are committed to rigorous, globally coordinated evidence generation to support the benefit-risk profile of menin inhibition across the treatment landscape. Our shared goal is to advance development rapidly and generate the evidence needed to deliver ziftomenib to appropriate patients in need.”
The publication is now available on the Journal of Clinical Oncology website and in the Scientific Manuscripts section on Kura’s website.
Ziftomenib is currently under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.
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.
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 efficacy, safety and therapeutic potential of ziftomenib. 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, 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 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
What were the key results of KURA's KOMET-001 trial for ziftomenib in NPM1-mutated AML?
What is the FDA PDUFA date for Kura Oncology's ziftomenib?
What were the main side effects reported in the KURA ziftomenib trial?
How effective was ziftomenib in different patient subgroups in the KURA trial?
What was the survival benefit for responders to KURA's ziftomenib?
