Kura Oncology and Kyowa Kirin Report Positive Combination Data for Ziftomenib at American Society of Hematology Annual Meeting
Kura Oncology and Kyowa Kirin announced positive interim data from the Phase 1a portion of KOMET-007 trial, evaluating ziftomenib in combination with standard treatments for acute myeloid leukemia (AML). The study showed impressive results with a 100% complete remission rate in NPM1-m and 83% in KMT2A-r first-line adverse risk AML patients when combined with 7+3 therapy.
Key highlights include 100% survival rate in first-line NPM1-m and 96% in KMT2A-r AML patients, with median follow-up of 31 and 19 weeks respectively. The drug demonstrated promising activity in relapsed/refractory cases with venetoclax/azacitidine combination. Ziftomenib was well-tolerated across all dose levels, with manageable side effects.
Based on these positive results, the companies plan to initiate KOMET-017, a global Phase 3 study, in mid-2025.
Kura Oncology e Kyowa Kirin hanno annunciato dati intermedi positivi dalla fase 1a del trial KOMET-007, che valuta ziftomenib in combinazione con trattamenti standard per la leucemia mieloide acuta (AML). Lo studio ha mostrato risultati impressionanti con un tasso di remissione completa del 100% nei pazienti con AML a rischio avverso di prima linea NPM1-m e dell'83% nei pazienti KMT2A-r quando combinato con la terapia 7+3.
I punti salienti includono un tasso di sopravvivenza del 100% nei pazienti NPM1-m di prima linea e del 96% nei pazienti KMT2A-r, con un follow-up mediano rispettivamente di 31 e 19 settimane. Il farmaco ha dimostrato un'attività promettente nei casi di recidiva/riferimento con la combinazione di venetoclax/azacitidina. Ziftomenib è stato ben tollerato a tutti i livelli di dosaggio, con effetti collaterali gestibili.
In base a questi risultati positivi, le aziende pianificano di avviare il KOMET-017, uno studio globale di fase 3, a metà del 2025.
Kura Oncology y Kyowa Kirin anunciaron datos interinos positivos de la fase 1a del ensayo KOMET-007, que evalúa ziftomenib en combinación con tratamientos estándar para la leucemia mieloide aguda (LMA). El estudio mostró resultados impresionantes con una tasa de remisión completa del 100% en pacientes con LMA de riesgo adverso de primera línea NPM1-m y del 83% en pacientes KMT2A-r cuando se combina con la terapia 7+3.
Los aspectos más destacados incluyen una tasa de supervivencia del 100% en pacientes NPM1-m de primera línea y del 96% en pacientes KMT2A-r, con un seguimiento mediano de 31 y 19 semanas respectivamente. El fármaco mostró una actividad prometedora en casos de recaída/refractarios con la combinación de venetoclax/azacitidina. Ziftomenib fue bien tolerado en todos los niveles de dosis, con efectos secundarios manejables.
Con base en estos resultados positivos, las empresas planean iniciar KOMET-017, un estudio global de fase 3, a mediados de 2025.
Kura Oncology와 Kyowa Kirin은 급성 골수성 백혈병(AML)을 위한 표준 치료와 함께 ziftomenib를 평가하는 KOMET-007 시험의 1a상 부분에서 긍정적인 중간 데이터를 발표했습니다. 이 연구는 NPM1-m 환자의 경우 100% 완전 관해율, KMT2A-r의 첫 번째 선 악성 AML 환자의 경우 7+3 요법과 결합 시 83%의 인상적인 결과를 보여주었습니다.
주요 하이라이트는 첫 번째 선 NPM1-m 환자의 생존율이 100%이고 KMT2A-r AML 환자는 각각 31주와 19주의 중앙 추적 조사에서 96%입니다. 이 약물은 venetoclax/azacitidine 조합으로 재발/불응성 사례에서 유망한 활성을 보여주었습니다. Ziftomenib은 모든 용량 수준에서 잘 견뎌졌으며, 부작용은 관리 가능합니다.
이러한 긍정적인 결과를 바탕으로, 두 회사는 2025년 중반에 글로벌 3상 연구인 KOMET-017을 시작할 계획입니다.
Kura Oncology et Kyowa Kirin ont annoncé des données intermédiaires positives de la phase 1a de l'essai KOMET-007, évaluant ziftomenib en combinaison avec des traitements standards pour la leucémie myéloïde aiguë (LMA). L'étude a montré des résultats impressionnants avec un taux de rémission complète de 100 % chez les patients NPM1-m et de 83 % chez les patients KMT2A-r à risque défavorable en première ligne lorsqu'il est combiné avec la thérapie 7+3.
Les points clés incluent un taux de survie de 100 % chez les patients NPM1-m de première ligne et de 96 % chez les patients KMT2A-r, avec un suivi médian de 31 et 19 semaines respectivement. Le médicament a montré une activité prometteuse dans les cas de rechute/réfractaires avec la combinaison venetoclax/azacitidine. Ziftomenib a été bien toléré à tous les niveaux de dose, avec des effets secondaires gérables.
Sur la base de ces résultats positifs, les entreprises prévoient de lancer KOMET-017, une étude mondiale de phase 3, à la mi-2025.
Kura Oncology und Kyowa Kirin haben positive Zwischenresultate aus dem Phase-1a-Teil der KOMET-007-Studie angekündigt, die ziftomenib in Kombination mit Standardbehandlungen für akute myeloische Leukämie (AML) bewertet. Die Studie zeigte beeindruckende Ergebnisse mit einer Remissionsrate von 100 % bei NPM1-m und 83 % bei KMT2A-r AML-Patienten im ersten Behandlungslinienrisiko, wenn es mit der 7+3-Therapie kombiniert wird.
Wichtige Erkenntnisse umfassen eine Überlebensrate von 100 % bei NPM1-m-Patienten der ersten Linie und 96 % bei KMT2A-r-AML-Patienten, mit einer medianen Nachbeobachtungszeit von 31 und 19 Wochen. Das Medikament zeigte vielversprechende Aktivität bei rückfälligen/refraktären Fällen mit der Kombination von Venetoclax/Azacitidin. Ziftomenib wurde in allen Dosisstufen gut vertragen, mit handhabbaren Nebenwirkungen.
Basierend auf diesen positiven Ergebnissen planen die Unternehmen, Mitte 2025 mit der globalen Phase-3-Studie KOMET-017 zu beginnen.
- 100% complete remission rate in NPM1-m and 83% in KMT2A-r first-line AML patients
- High survival rates: 100% in NPM1-m and 96% in KMT2A-r AML patients
- 76% MRD negativity in NPM1-m and 75% in KMT2A-r patients
- 68% overall response rate in R/R NPM1-m population with ven/aza combination
- Lower response rates in previously venetoclax-exposed patients (50% ORR)
- Only 30% response rate in KMT2A-r patients with ven/aza combination
Insights
The interim analysis from KOMET-007 demonstrates exceptional efficacy with ziftomenib combinations. The 100% CR rate in NPM1-m and 83% CR rate in KMT2A-r first-line AML patients are remarkable outcomes in this difficult-to-treat population. The high MRD negativity rates (
The safety profile is particularly encouraging, with manageable differentiation syndrome rates of just
The relapsed/refractory data showing activity in venetoclax-experienced patients is clinically significant, as these patients typically have treatment options. The planned Phase 3 KOMET-017 study's dual-arm design targeting both intensive and non-intensive therapy pathways could position ziftomenib as a standard of care across the AML treatment spectrum.
This data release represents a significant value inflection point for Kura Oncology. The exceptional efficacy in first-line AML, particularly the 100% complete remission rate in NPM1-mutant patients, positions ziftomenib as a potential blockbuster therapy. The market opportunity is substantial, as NPM1 mutations occur in approximately
The partnership with Kyowa Kirin adds strategic value, bringing expertise in hematological malignancies and global commercialization capabilities. The planned Phase 3 KOMET-017 study in mid-2025 provides a clear regulatory pathway. The strong safety profile and compatibility with standard treatments could drive broad adoption if approved.
For a company with an
– Interim analysis from Phase 1a portion of KOMET-007 shows
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– Promising clinical activity in R/R NPM1-m and KMT2A-r AML with ven/aza, including ven-experienced patients –
– Ziftomenib generally well tolerated in combination with standards of care at all dose levels studied –
– Kura Oncology to host virtual investor event today at 8:00 a.m. ET –
SAN DIEGO and TOKYO, Dec. 09, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) provided encouraging clinical data from KOMET-007, a Phase 1 dose-escalation trial of ziftomenib, a highly selective oral investigational menin inhibitor, in combination with standards of care, including cytarabine/daunorubicin (7+3) and venetoclax/azacitidine (ven/aza), in patients with NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML).
These data were presented at the 2024 American Society of Hematology (ASH) Annual Meeting. An oral presentation highlighting ziftomenib combined with 7+3 in newly diagnosed (1L) NPM1-m and KMT2A-r adverse riski AML, and a poster featuring ziftomenib in combination with ven/aza in relapsed/refractory (R/R) NPM1-m and KMT2A-r AML are available in the Posters and Presentations section on Kura’s website.
Ziftomenib was generally well tolerated in combination at all dose levels evaluated across all cohorts in the Phase 1a dose-escalation portion of the study. No dose-limiting toxicities, evidence of ziftomenib-associated QTc prolongation, drug-drug interactions or additive myelosuppression were observed. In the 7+3 combination cohorts, on-target differentiation syndrome (DS) occurred in
Among the response-evaluable patients enrolled in the 7+3 combination cohort for patients with 1L NPM1-m or KMT2A-r adverse riski AML,
A total of 54 patients were enrolled in the combination cohort with ven/aza in R/R NPM1-m or KMT2A-r AML. The NPM1-m population achieved an overall response rate (ORR) of
“The findings presented at ASH underscore the potential of ziftomenib in combination with standards of care as an early intervention in the frontline setting of AML and could offer a meaningful opportunity to improve patient outcomes,” said Amer Zeidan, MBBS, MHS, chief of the Division of Hematologic Malignancies, director of Hematology Early Therapeutics Research at Yale Cancer Center, and lead investigator of the KOMET-007 trial. “The high rates of complete remission and MRD negativity across the 7+3 cohorts are particularly encouraging. The rapid enrollment in the Phase 1a portion of this study underscores the urgency and enthusiasm for further evaluating this combination approach.”
Kura and Kyowa Kirin recently announced plans for KOMET-017, a global, pivotal Phase 3 study evaluating ziftomenib in combination with standards of care for adults with newly diagnosed KMT2A-r or NPM1-m AML. The trial includes two independently powered, randomized, double-blind, placebo-controlled studies: a non-intensive therapy arm testing ziftomenib with ven/aza, and an intensive therapy arm testing ziftomenib with 7+3. The positive results from KOMET-007 reported at ASH reinforce Kura’s and Kyowa Kirin’s commitment to evaluating ziftomenib for patients across the continuum of frontline treatment options. The KOMET-017 study is expected to initiate in mid-2025.
“Starting patients on therapy early is essential to improving outcomes in AML,” said Mollie Leoni, M.D., Executive Vice President, Clinical Development at Kura Oncology. “The updated KOMET-007 data underscore 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. Together, the KOMET-007 Phase 1b trial and the KOMET-017 pivotal Phase 3 study will allow us to further explore this approach and the potential to transform care if approved for AML patients worldwide.”
“More than half of AML patients with an NPM1 mutation will relapse with poor survival outcomesii, making it a significant area of unmet medical need in the frontline setting,” said Takeyoshi Yamashita, Ph.D., Senior Managing Executive Officer and Chief Medical Officer of Kyowa Kirin. “The data observed to date represent the potential of ziftomenib to help address the treatment gap and improve upon current standards of care. Leveraging our hemato-oncology expertise and commitment to patients, we are committed to rapidly advancing the clinical development of ziftomenib.”
Virtual Investor Event
Kura will host a webcast and conference call featuring Kura Oncology management and Key Opinion Leaders Amir T. Fathi, MD, Associate Professor of Medicine at Harvard Medical School and Director of the Leukemia Program at Massachusetts General Cancer Center, and Amer Zeidan, MBBS, MHS, interim chief of the Division of Hematologic Malignancies, Director of Hematology Early Therapeutics Research at Yale Cancer Center. The live call may be accessed by dialing (800) 715-9871 for domestic callers and (646) 307-1963 for international callers and entering the conference ID: 4326549. A live webcast will be available here and in the Investors section of Kura’s website, with an archived replay available shortly after the event.
About Ziftomenib
Ziftomenib is a selective and oral menin inhibitor currently in development for the treatment of genetically defined AML patients with high unmet need. In April 2024, ziftomenib received Breakthrough Therapy Designation (BTD) by the FDA for the treatment of R/R NPM1-mutant AML based on data from Kura’s ongoing KOMET-001 clinical trial. Additional information about clinical trials for ziftomenib can be found at kuraoncology.com/clinical-trials/#ziftomenib.
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 that target cancer signaling pathways. Ziftomenib is being investigated as a once-daily, oral drug candidate targeting the menin-KMT2A protein-protein interaction, has received BTD for the treatment of R/R NPM1-mutant AML. Kura has completed enrollment in a Phase 2 registration-directed trial of ziftomenib in R/R NPM1-mutant AML (KOMET-001). The Company is also conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-mutant and KMT2A-rearranged AML. Kura is evaluating KO-2806, a next-generation farnesyl transferase inhibitor (FTI), in a Phase 1 dose-escalation trial as a monotherapy and in combination with targeted therapies (FIT-001). Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial in combination with alpelisib for patients with PIK3CA-dependent head and neck squamous cell carcinoma (KURRENT-HN). For additional information, please visit Kura’s website at www.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 pursuit of a broad ziftomenib development program including frontline indications and combinations with targeted therapies; the efficacy, safety and therapeutic potential of ziftomenib; potential benefits of combining ziftomenib with appropriate standards of care, including chemotherapies; and progress and expected timing of the ziftomenib program and clinical trials, including the timing of initiation of the pivotal Phase 3 frontline study. 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, applications 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 (SEC), including the Company’s Form 10-Q for the quarter ended September 30, 2024 filed with the SEC on November 7, 2024, 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.
Amer Zeidan has consulted and received honoraria from Kura. Opinions expressed are his own and do not necessarily represent those of his employer.
Kura Contacts
Investors:
Pete De Spain
Executive Vice President, Investor Relations &
Corporate Communications
(858) 500-8833
pete@kuraoncology.com
Media:
Cassidy McClain
Vice President
Inizio Evoke Comms
(619) 849-6009
cassidy.mcclain@inizioevoke.com
Kyowa Kirin Contacts
Wataru Suzuki
Corporate Communications Department – Global
media@kyowakirin.com
Lauren Walrath
Vice President, Public Affairs – North America
lauren.walrath.g4@kyowakirin.com
i Age ≥ 60 years and/or treatment-related AML and/or adverse risk cytogenetics per European LeukemiaNet (ELN)
ii Prata PH, Bally C, Prebet T, et al. NPM1 mutation is not associated with prolonged complete remission in acute myeloid leukemia patients treated with hypomethylating agents. Haematologica. 2018;103(10):e455-e457.
FAQ
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