Kura Oncology Completes Enrollment in Registration-Directed Trial of Ziftomenib in NPM1-Mutant AML
Kura Oncology has completed enrollment of 85 patients in its Phase 2 KOMET-001 clinical trial, targeting relapsed or refractory (R/R) NPM1-mutant acute myeloid leukemia (AML), within 16 months.
The trial focuses on assessing the clinical activity, safety, and tolerability of their menin inhibitor, ziftomenib (KO-539). Topline data is expected in early 2025.
Notably, ziftomenib has received Breakthrough Therapy Designation from the FDA, which aims to expedite the review process for new drug applications due to its potential efficacy in treating R/R NPM1-mutant AML.
This milestone underscores the urgent need for effective treatment options in AML and highlights ziftomenib's potential to become a best-in-class treatment.
- Enrollment of 85 patients completed in fewer than 16 months.
- Topline data expected in early 2025.
- Breakthrough Therapy Designation from FDA for ziftomenib.
- Potential to expedite New Drug Application review.
- Evidence of clinical activity and safety in ongoing studies.
- Favorable safety profile and encouraging clinical activity of ziftomenib.
- Potential to transform the standard of care for AML patients.
- Topline data release not expected until early 2025, creating an extended period of uncertainty.
- No currently approved targeted therapy for NPM1-mutant AML highlights the urgent unmet need.
Insights
The completion of enrollment for Kura Oncology's ziftomenib in the KOMET-001 trial is a noteworthy event in the field of hematologic malignancies. This Phase 2 trial focuses on relapsed or refractory (R/R) NPM1-mutant acute myeloid leukemia (AML), a specific subset of AML that accounts for approximately 30% of all new AML cases. The quick enrollment, within 16 months, underscores the significant demand for effective treatments in this space. The Breakthrough Therapy Designation granted by the FDA further emphasizes the drug's potential, which promises to expedite the review process. This could hasten the availability of ziftomenib to patients, offering a much-needed targeted therapy for those with limited options. Early 2025 data will be important in confirming the efficacy and safety, potentially setting a new standard of care in treating NPM1-mutant AML.
From a financial perspective, the completion of patient enrollment and the Breakthrough Therapy Designation by the FDA are significant milestones for Kura Oncology. These developments potentially position ziftomenib as a revenue-generating product sooner than anticipated. The rapid enrollment indicates robust interest and unmet need in the market, which could translate into substantial market uptake upon approval. Investors should note that the announcement of topline data in early 2025 will be a key catalyst for the stock. If the data is positive, it could significantly boost the company's valuation. However, it's also important to consider the inherent risks of clinical trials and the approval process, as any negative outcomes could adversely affect the stock price. Stakeholders should monitor forthcoming data closely to better gauge the financial prospects.
The trial's completion of enrollment and the Breakthrough Therapy Designation highlight ziftomenib's potential as a game-changer in treating R/R NPM1-mutant AML. This designation means the FDA recognizes the preliminary clinical evidence indicating the drug might offer substantial improvement over existing therapies. For retail investors, understanding the significance of these milestones is essential as it implies a shorter path to market, potentially reducing the time and cost associated with bringing the drug to patients. The primary endpoint of complete response will be critical to watch, as achieving this metric could lead to expedited regulatory approval, increased market trust and potential early revenue streams. However, investors should be aware of the typical risks involved in drug development, including the possibility of unforeseen safety issues or lower-than-expected efficacy.
– Company enrolls 85 patients with R/R NPM1-mutant AML in fewer than 16 months –
– Topline data expected in early 2025 –
– Breakthrough Therapy Designation to enable expedited review by FDA –
SAN DIEGO, May 14, 2024 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced that it has completed enrollment of 85 patients in the Phase 2 portion of KOMET-001, a registration-directed clinical trial of the Company’s menin inhibitor, ziftomenib (KO-539), in patients with relapsed or refractory (R/R) NPM1-mutant acute myeloid leukemia (AML). The Company expects to report topline data from the trial in early 2025.
“We are thrilled to announce this critical milestone, which brings us one step closer to delivering ziftomenib as a potentially best-in-class treatment for patients with genetically defined acute leukemias,” said Troy Wilson, Ph.D., J.D., President and Chief Executive Officer of Kura Oncology. “Our confidence is supported by our recently announced Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA), which recognizes ziftomenib’s potential as an innovative medicine for patients with R/R NPM1-mutant AML and is intended to expedite review as we prepare for submission of a New Drug Application. We are grateful for the KOMET-001 investigators, patients and their families, and we look forward to sharing topline data from this pivotal study early next year.”
Kura announced the first patients dosed in the Phase 2 portion of KOMET-001 in February 2023. The registration-directed study is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib in patients with R/R NPM1-mutant AML, with a primary endpoint of complete response. The study has completed enrollment of the 85 patients necessary to support the primary endpoint analysis.
“The rapid enrollment of this study reflects the urgent need for more effective treatment options in AML as well as the potential for ziftomenib to address this need,” said Eunice Wang, M.D., Chief of the Leukemia Service at Roswell Park Comprehensive Cancer Center and principal investigator of the trial. “NPM1-mutant AML represents approximately
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 novel, once-daily, oral investigational drug candidate targeting the menin-KMT2A/MLL protein-protein interaction for treatment of genetically defined AML patients with high unmet need. In the KOMET-001 Phase 1 study, ziftomenib demonstrated an encouraging safety profile and tolerability with reported events most often consistent with features and manifestations of underlying disease. Clinical activity of ziftomenib as a monotherapy was optimal at the 600 mg daily dose and a
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, a once-daily, oral drug candidate targeting the menin-KMT2A protein-protein interaction, has received Breakthrough Therapy Designation for the treatment of R/R NPM1-mutant AML. Kura has completed enrollment in a Phase 2 registration-directed trial of ziftomenib in NPM1-mutant R/R AML (KOMET-001) and expects to report topline data in early 2025. The Company is also conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in NPM1-mutant and KMT2A-rearranged newly diagnosed and R/R AML. Tipifarnib, a potent and selective farnesyl transferase inhibitor (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). Kura is also evaluating KO-2806, a next-generation FTI, in a Phase 1 dose-escalation trial as a monotherapy and in combination with targeted therapies (FIT-001). For additional information, please visit Kura’s website at www.kuraoncology.com and follow us on X and LinkedIn.
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, potential benefits of combining ziftomenib with appropriate standards of care, and progress and expected timing of the ziftomenib program and clinical trials. 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, 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
Investors:
Pete De Spain
Executive Vice President, Investor Relations &
Corporate Communications
(858) 500-8833
pete@kuraoncology.com
Media:
Alexandra Weingarten
Associate Director, Corporate Communications &
Investor Relations
(858) 500-8822
alexandra@kuraoncology.com
1 Issa G, et al. Blood Adv 2023;7(6):933-42.
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