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 and Kyowa Kirin Announce Publication of Pivotal Ziftomenib Data in Relapsed/Refractory NPM1-Mutated AML in the Journal of Clinical Oncology
09-25-2025
– 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 –
“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
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
The KOMET-001 phase 2 trial met its primary endpoint with a complete remission with full or partial hematologic recovery (CR/CRh) rate of 22% (95% CI, 14 to 32; P=0.0058), which was significantly higher than the 12% historical standard-of-care response rate for patients with R/R NPM1-m AML. One additional response of CRh occurred after the primary analysis data cutoff resulting in a cumulative CR/CRh rate of 23% (95% CI, 15 to 33). 61% of evaluable CR/CRh responders were negative for measurable residual disease (MRD). Overall response rate (ORR) was 33% (95% CI, 23 to 43), with a median duration of overall response of 4.6 months (95% CI, 2.8 to 7.4).
Median overall survival (OS) was 6.6 months (95% CI, 3.6 to 8.6). Among ORR responders, median OS was 18.4 months (95% CI, 8.6 to not estimable) vs. 3.5 months (95% CI, 2.7 to 4.2) among non-responders. Two responders received subsequent allogeneic stem cell transplant and both resumed ziftomenib maintenance after transplant. At the time of data cutoff, nine patients (two after transplantation) remained on ziftomenib treatment. Prespecified subgroup analyses showed comparable CR/CRh rates regardless of lines of therapy, prior venetoclax exposure, or presence of co-mutations, including FLT3m or IDH1/2m.
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 (26%), anemia (20%), and thrombocytopenia (20%). Differentiation syndrome occurred in 25% of patients (15% grade 3; no grade 4-5) and was manageable with protocol-defined mitigation. Three patients (3%) discontinued treatment because of ziftomenib-related adverse events.
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
“The publication of the investigational ziftomenib data adds important scientific context for clinicians and patients,” said
The publication is now available on the
Ziftomenib is currently under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.
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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.
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gmann@kuraoncology.com
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Source: Kura Oncology, Inc.