Kura Oncology and Kyowa Kirin Announce Submission of New Drug Application for Ziftomenib to FDA
Kura Oncology (KURA) and Kyowa Kirin have submitted a New Drug Application (NDA) to the FDA for ziftomenib, their oral menin inhibitor designed to treat adult patients with relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutation.
The submission was completed on March 31, 2025, with FDA expected to determine application completeness within 60 days. Ziftomenib has received Breakthrough Therapy, Fast Track, and Orphan Drug Designations. The company has requested Priority Review, which could result in a six-month FDA review period if granted.
This therapeutic candidate represents a potential first FDA-approved targeted therapy for NPM1-mutant AML patients, addressing an significant unmet medical need in this devastating disease.
Kura Oncology (KURA) e Kyowa Kirin hanno presentato una Nuova Domanda di Farmaco (NDA) alla FDA per ziftomenib, il loro inibitore della menina orale progettato per trattare pazienti adulti con leucemia mieloide acuta (AML) recidivante o refrattaria con mutazione NPM1.
La presentazione è stata completata il 31 marzo 2025, con la FDA che si aspetta di determinare la completezza della domanda entro 60 giorni. Ziftomenib ha ricevuto le designazioni di Breakthrough Therapy, Fast Track e Orphan Drug. L'azienda ha richiesto una Revisione Prioritaria, che potrebbe comportare un periodo di revisione di sei mesi da parte della FDA se concessa.
Questo candidato terapeutico rappresenta una potenziale prima terapia mirata approvata dalla FDA per i pazienti con AML mutante NPM1, affrontando un'importante necessità medica insoddisfatta in questa malattia devastante.
Kura Oncology (KURA) y Kyowa Kirin han presentado una Nueva Solicitud de Medicamento (NDA) a la FDA para ziftomenib, su inhibidor oral de menina diseñado para tratar a pacientes adultos con leucemia mieloide aguda (AML) en recaída o refractaria con mutación NPM1.
La presentación se completó el 31 de marzo de 2025, y se espera que la FDA determine la integridad de la solicitud en un plazo de 60 días. Ziftomenib ha recibido las designaciones de Breakthrough Therapy, Fast Track y Orphan Drug. La empresa ha solicitado una Revisión Prioritaria, lo que podría resultar en un período de revisión de seis meses por parte de la FDA si se concede.
Este candidato terapéutico representa una posible primera terapia dirigida aprobada por la FDA para pacientes con AML mutante NPM1, abordando una importante necesidad médica no satisfecha en esta devastadora enfermedad.
Kura Oncology (KURA)와 Kyowa Kirin은 ziftomenib에 대한 새로운 의약품 신청서(NDA)를 FDA에 제출했습니다. 이는 NPM1 변이를 가진 재발성 또는 난치성 성인 급성 골수성 백혈병(AML) 환자를 치료하기 위해 설계된 경구용 메닌 억제제입니다.
신청서는 2025년 3월 31일에 완료되었으며, FDA는 60일 이내에 신청서의 완전성을 결정할 것으로 예상됩니다. Ziftomenib은 Breakthrough Therapy, Fast Track, 그리고 Orphan Drug Designations를 받았습니다. 회사는 우선 심사를 요청했으며, 승인될 경우 FDA 심사 기간이 6개월로 단축될 수 있습니다.
이 치료 후보는 NPM1 변이 AML 환자를 위한 FDA 승인 목표 치료의 첫 번째 가능성을 나타내며, 이 파괴적인 질병에서 중요한 의료적 필요를 충족합니다.
Kura Oncology (KURA) et Kyowa Kirin ont soumis une Demande de Nouveau Médicament (NDA) à la FDA pour ziftomenib, leur inhibiteur oral de la ménine conçu pour traiter les patients adultes atteints de leucémie myéloïde aiguë (LMA) récurrente ou réfractaire avec mutation NPM1.
La soumission a été complétée le 31 mars 2025, et la FDA devrait déterminer la complétude de la demande dans un délai de 60 jours. Ziftomenib a reçu les désignations de Breakthrough Therapy, Fast Track et Orphan Drug. L'entreprise a demandé un Examen Prioritaire, ce qui pourrait entraîner une période d'examen de six mois par la FDA si accordé.
Ce candidat thérapeutique représente une première thérapie ciblée potentiellement approuvée par la FDA pour les patients atteints de LMA mutante NPM1, répondant à un besoin médical non satisfait dans cette maladie dévastatrice.
Kura Oncology (KURA) und Kyowa Kirin haben einen Antrag auf Zulassung eines neuen Arzneimittels (NDA) bei der FDA für ziftomenib eingereicht, ihren oralen Menin-Inhibitor, der zur Behandlung von Erwachsenen mit rezidivierender oder refraktärer akuter myeloischer Leukämie (AML) mit NPM1-Mutation entwickelt wurde.
Die Einreichung wurde am 31. März 2025 abgeschlossen, und die FDA wird voraussichtlich innerhalb von 60 Tagen über die Vollständigkeit des Antrags entscheiden. Ziftomenib hat die Bezeichnungen Breakthrough Therapy, Fast Track und Orphan Drug erhalten. Das Unternehmen hat eine Prioritätsprüfung beantragt, die, falls genehmigt, zu einem sechsmonatigen Prüfungszeitraum durch die FDA führen könnte.
Dieser therapeutische Kandidat stellt eine potenzielle erste von der FDA genehmigte zielgerichtete Therapie für Patienten mit NPM1-mutierter AML dar und deckt einen erheblichen ungedeckten medizinischen Bedarf in dieser verheerenden Krankheit ab.
- First potential FDA-approved targeted therapy for NPM1-mutant AML
- Multiple FDA designations secured (Breakthrough, Fast Track, Orphan Drug)
- Potential for expedited 6-month review if Priority Review is granted
- FDA acceptance of NDA still pending
- Priority Review status not yet confirmed
Insights
Kura Oncology's NDA submission for ziftomenib represents a critical regulatory milestone that significantly derisks their lead asset's path to commercialization. This submission targets NPM1-mutated acute myeloid leukemia (AML), where currently no FDA-approved targeted therapies exist, positioning ziftomenib to potentially capture an untapped market segment if approved.
The drug's impressive regulatory designation trifecta (Breakthrough Therapy, Fast Track, and Orphan Drug) signals FDA recognition of both ziftomenib's potential clinical importance and the high unmet need in this patient population. Their request for Priority Review, if granted, could accelerate time-to-market by shortening the standard review period to six months versus the typical ten-month timeline.
For a company of Kura's size (
The ziftomenib NDA submission represents a significant advancement for NPM1-mutated AML patients. As a highly selective, oral menin inhibitor, ziftomenib targets a specific genetic driver in approximately
The triple regulatory designations (Breakthrough, Fast Track, Orphan) reflect compelling clinical data demonstrating meaningful benefit in this difficult-to-treat population. Mechanistically, menin inhibition disrupts the interaction between menin and MLL1, which is crucial for leukemic cell survival in NPM1-mutated AML.
The oral administration route provides important advantages over traditional chemotherapy, potentially enabling outpatient treatment with reduced hospitalization requirements. If approved, ziftomenib would be pioneering as the first FDA-approved targeted therapy specifically for NPM1-mutated AML patients. This represents a textbook example of precision oncology - matching a targeted therapeutic to a specific genetic alteration - and could significantly improve outcomes for this genetically-defined patient population currently lacking personalized treatment options.
– Submission seeks approval for the treatment of adult patients with relapsed or refractory AML with a NPM1 mutation –
SAN DIEGO and TOKYO, April 08, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”), and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”), today announced Kura submitted a New Drug Application (NDA) for ziftomenib, a highly selective, once-daily, oral, investigational menin inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM 1) mutation to the U.S. Food and Drug Administration (FDA) on March 31, 2025.
Ziftomenib has received Breakthrough Therapy, Fast Track, and Orphan Drug Designations. The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review; Kura expects to receive notification from the FDA on this preliminary evaluation in the second quarter of 2025. Priority Review was requested, which, if granted, would provide a target FDA review period of six months after NDA acceptance.
“This NDA submission brings us one step closer to our goal of advancing ziftomenib to market as a new therapeutic option for adult patients with R/R NPM1-m AML, a devastating disease for which there are currently no FDA-approved targeted therapy options,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “We look forward to working closely with the FDA throughout the review process and are optimistic about the potential of ziftomenib to impact patients with NPM1-mutant AML. We extend our gratitude to the team at Kura, our dedicated investigators, study site teams, and most importantly, to the patients who participated in our clinical trials, and their families and caregivers, who all helped make this possible. We appreciate the support and cooperation we enjoy with our partner Kyowa Kirin, and we look forward with confidence to the continued progress of this program and our collaboration.”
About NPM1-Mutant AML
AML is the most common acute leukemia in adults and begins when the bone marrow makes abnormal myeloblasts (white blood cells), red blood cells, or platelets. Despite the many available treatments for AML, prognosis for patients remains poor and a high unmet need remains. The menin pathway is considered a driver for multiple genetic alterations of the disease, of which NPM1 mutations are among the most common, representing approximately
About Ziftomenib
Ziftomenib is a potent and selective, oral, investigational 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) from the FDA for the treatment of adult patients with R/R AML with a NPM1 mutation based on data from Kura’s KOMET-001 clinical trial. Additional information about clinical trials for ziftomenib can be found at www.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, designed to target cancer signaling pathways. Ziftomenib, a once-daily, oral menin inhibitor, is the first and only investigational therapy to receive BTD from the FDA for the treatment of R/R NPM1-m AML. In November 2024, Kura entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib for AML and other hematologic malignancies. Enrollment in 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 submission of an NDA for ziftomenib for the treatment of adult patients with R/R NPM1-m AML. 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. 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 Kura’s 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, we have invested in drug discovery and biotechnology innovation for more than 70 years and are 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 our values, to sustainable growth, and to making people smile unites us across the globe. You can learn more about the business of Kyowa Kirin at: https://www.kyowakirin.com.
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; the anticipated timing of the FDA’s notification on its preliminary evaluation of the NDA for ziftomenib; future interactions with the FDA related to the NDA; and the continued progress of Kura’s ziftomenib program and our collaboration with Kyowa Kirin. 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 the FDA, 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.
Contacts: Kura Oncology
Investors:
Patti Bank
Managing Director
(415) 513-1284
patti.bank@icrhealthcare.com
Media:
Alexandra Weingarten
Associate Director, Corporate Communications
(858) 500-8822
alexandra@kuraoncology.com
Contacts: Kyowa Kirin
Investors:
Ryohei Kawai
ir@kyowakirin.com
Media, Global:
Wataru Suzuki,
media@kyowakirin.com
Media, US:
Lisa Popyk
lisa.popyk.3z@kyowakirin.com
1 Issa G, et al. Blood Adv 2023;7(6):933-42.
FAQ
When did Kura Oncology (KURA) submit the NDA for ziftomenib to the FDA?
What designations has ziftomenib received from the FDA for KURA's AML treatment?
What is the target patient population for KURA's ziftomenib treatment?
What is the expected FDA review timeline for KURA's ziftomenib NDA?
Is there any current FDA-approved targeted therapy for NPM1-mutant AML?
