Kura Oncology and Kyowa Kirin Announce Submission of New Drug Application for Ziftomenib to FDA
Rhea-AI Summary
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.
Positive
- 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
Negative
- FDA acceptance of NDA still pending
- Priority Review status not yet confirmed
News Market Reaction – KURA
In the trading session that priced this news, KURA declined 2.43%, reflecting a moderate negative market reaction.
Data tracked by StockTitan Argus on the day of publication.
AI-generated analysis. How Rhea-AI works. Not financial advice.
– 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.
