Tipifarnib

– New Drug Application based on positive results from the Phase 2 KOMET-001 trial – – FDA assigns a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025 – – Potential first approval of a menin inhibitor for the treatment of adult patients with relapsed or refractory AML with an NPM1 mutation – SAN DIEGO and TOKYO , June 01, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today announced the U.S. Food and Drug Administration (FDA) has accepted Kura’s New Drug Application (NDA) seeking full approval for ziftomenib as a treatment for adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025 . “The FDA’s acceptance of our New Drug Application marks a significant milestone for Kura and Kyowa Kirin and, more importantly, for patients living with this genetic subset of AML, who face an aggressive form of the disease with few treatment options,” said Troy Wilson , Ph.D., J.D., President and Chief Executive Officer of Kura Oncology . “This achievement reflects the strength of the clinical data for ziftomenib as well as the incredible commitment of our teams. Along with our partners at Kyowa Kirin , we look forward to continuing to work closely with the FDA throughout the review process and to prepare for the anticipated launch of this treatment, which holds potential to meaningfully impact the lives of patients and their families.” The NDA is based on results from the Phase 2 KOMET-001 registrational trial in R/R NPM1-mutant (NPM1-m) AML (NCT #04067336). The KOMET-001 trial achieved its primary endpoint of complete remission (CR) plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. Ziftomenib was well‑tolerated with limited myelosuppression and 3% ziftomenib-related discontinuations. The safety and tolerability of ziftomenib were consistent with previous reports, and the benefit-risk profile for ziftomenib is highly encouraging. “Adult R/R NPM1-m AML patients face a significantly poor prognosis, highlighting the urgent need for innovative treatment options that can improve their outcomes,” said Takeyoshi Yamashita , Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin . “The acceptance of this NDA is a crucial step in our ongoing efforts to explore and evaluate various therapeutic strategies for AML through our comprehensive clinical trials. Our dedicated teams at Kyowa Kirin and Kura are fully committed to working tirelessly to ensure that, once approved, ziftomenib is made available to AML patients as quickly as possible. We recognize the importance of this endeavor and are excited about the possibility of making a meaningful impact on the lives of those affected by this challenging disease.” The KOMET-001 registration-directed trial is designed to assess evidence of clinical activity, safety and tolerability of ziftomenib, the only investigational therapy to receive Breakthrough Therapy Designation (BTD) from the FDA for treatment of R/R NPM1-mutant AML. In addition to BTD, ziftomenib has received Fast Track and Orphan Drug Designations. The full data analyses from the KOMET-001 trial of ziftomenib in R/R NPM1-m AML patients have been selected for oral presentation on Monday, June 2nd at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, and an encore presentation is planned at the 2025 European Hematology Association (EHA) Congress . 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 30% of AML cases. While patients with NPM1-m AML have high response rates to frontline therapy, relapse rates are high and survival outcomes are poor, with only 30% overall survival at 12 months in the R/R setting. Additionally, NPM1 mutations frequently occur with co-mutations in other disease-associated genes, including FLT3, DNMT3A, and IDH1/2, with prognosis heavily influenced by the presence of such co-occurring mutations. Adult patients with NPM1-m AML and select co-mutations and/or R/R disease have a poor prognosis, with median overall survival of only approximately 7.8 months in 2nd line, 5.3 months in 3rd line, and 3.5 months following the 4th line1. There are currently no FDA-approved therapies targeting NPM1-m AML. 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 BTD from the FDA for the treatment of adult patients with R/R AML with an 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. In November 2024, Kura Oncology entered into a global strategic collaboration agreement with Kyowa Kirin to develop and commercialize ziftomenib, a menin inhibitor, for AML and other hematologic malignancies. Enrollment in KOMET-001, 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, 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 efficacy, safety and therapeutic potential of ziftomenib; the potential for ziftomenib to obtain FDA approval for the treatment of patients with NPM1-m AML, and the anticipated timing of such FDA approval; and the potential launch of ziftomenib. Factors that may cause actual results to differ materially from those indicated by these forward-looking statements include the risk that Kura may not be able to successfully demonstrate the safety and/or efficacy of its product candidates, including ziftomenib; the risk that Kura may not obtain approval to market its product candidates, including ziftomenib, or that such approval may be delayed; 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. Kura Contacts Investors: Patti Bank Managing Director(415) 513-1284patti.bank@icrhealthcare.com Media:media@kuraoncology.com Kyowa Kirin Contacts Investors: Ryohei Kawai ir@kyowakirin.com Media, Global: Wataru Suzuki media@kyowakirin.com Source: Kura Oncology, Inc.

– NDA submitted in 1Q 2025 for ziftomenib for the treatment of adult patients with relapsed or refractory AML with an NPM1 mutation – – Data from Phase 1b/2 registration-directed trial of ziftomenib selected for oral presentation at ASCO Annual Meeting – – $45.0 million milestone payment earned for NDA submission under collaboration agreement with Kyowa Kirin – – First patients dosed in Phase 1 trial of ziftomenib plus imatinib in GIST – – $703.2 million in pro forma cash, together with anticipated collaboration agreement payments, expected to support ziftomenib commercialization through the frontline AML combination setting – – Management to host webcast and conference call today at 4:30 p.m. ET – SAN DIEGO , May 01, 2025 (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 reported first quarter 2025 financial results and provided a corporate update. “In the first quarter of 2025, we achieved a significant milestone with the submission of our first NDA for ziftomenib,” said Troy Wilson , Ph.D., J.D., President and Chief Executive Officer of Kura Oncology . “We are committed to working with FDA to support its review and are strategically advancing our pre-commercial activities to prepare for potential approval for the treatment of adult patients with relapsed or refractory NPM1-mutant AML. Beyond the monotherapy setting, we look forward to sharing data on the combination of ziftomenib with intensive and non-intensive standards of care, while gearing up for two Phase 3 studies in the frontline setting. With a strong pipeline, multiple clinical data readouts expected this year, and a solid financial foundation, we are well-positioned to drive progress across our programs.” Recent Highlights Submission of New Drug Application for ziftomenib to FDA – Kura and Kyowa Kirin Co., Ltd. (Kyowa Kirin) announced submission of the New Drug Application (NDA) for ziftomenib for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation to the U.S. Food and Drug Administration (FDA) on March 31, 2025 . From the time of submission, the FDA has a 60-day filing review period, and the Company expects to receive notification from the FDA on this preliminary evaluation in the second quarter of 2025. If Priority Review is granted, it would provide a target FDA review period of six months after NDA acceptance. Abstracts accepted for presentation at ASCO and EHA – In February 2025 , Kura and Kyowa Kirin announced positive topline results from the KOMET-001, the Phase 2 registration-directed trial of ziftomenib in patients with R/R NPM1-mutant (NPM1-m) AML. These data have been accepted for oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago and accepted for an encore presentation at the European Hematology Association (EHA) Congress in Milan, Italy . In addition, preliminary clinical data from the Phase 1b expansion cohort evaluating ziftomenib in combination with intensive (7+3) in the frontline setting has been accepted for an oral presentation at EHA. Abstract titles and presentation details can be found on the ASCO and EHA meeting sites at the lift of the respective embargos. Submission of the NDA for ziftomenib has triggered a $45 million milestone payment obligation from Kyowa Kirin – As a result of the NDA submission for ziftomenib, Kura has earned a $45 million milestone payment under the global strategic collaboration agreement between Kura and Kyowa Kirin to develop and commercialize ziftomenib in acute leukemias (Kyowa Agreement). Under the terms of the Kyowa Agreement, Kura received an upfront payment of $330 million in December 2024 and accounting for this $45 million milestone payment, Kura expects to receive up to $375 million in additional, near-term milestone payments. First patients dosed in KOMET-015 trial in GIST – Earlier this week, Kura announced the first patients have been dosed in its KOMET-015 Phase 1 clinical trial of ziftomenib in patients with advanced gastrointestinal stromal tumors (GIST) after imatinib failure. In October 2024 , Kura presented preclinical data at the 36th EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics in Barcelona , supporting the potential for ziftomenib in combination with KIT inhibitors for the treatment of GIST. The combination of ziftomenib and imatinib demonstrated robust and durable antitumor activity in imatinib-sensitive (1L) and imatinib-resistant (2L/3L) GIST patient-derived xenograft models. Preclinical data for KO-2806 presented in oral minisymposium session at AACR Annual Meeting – Last month, Kura presented preclinical data for KO-2806, the Company’s next-generation farnesyl transferase inhibitor (FTI), in combination with cabozantinib for the treatment of clear cell renal cell carcinoma at the American Association for Cancer Research (AACR) Annual Meeting in Chicago . These data add to a growing body of preclinical evidence demonstrating the potential of FTIs as companion therapeutic agents to augment the antitumor activities of and to overcome resistance to various targeted therapies. Financial Results Collaboration revenue from our Kyowa Kirin partnership for the first quarter of 2025 was $14.1 million , compared to no revenue for the first quarter of 2024. Research and development expenses for the first quarter of 2025 were $56.0 million , compared to $36.3 million for the first quarter of 2024. General and administrative expenses for the first quarter of 2025 were $22.8 million , compared to $18.2 million for the first quarter of 2024. Net loss for the first quarter of 2025 was $57.4 million , compared to a net loss of $49.5 million for the first quarter of 2024. Net loss for the first quarter of 2025 included non-cash, share-based compensation expense of $7.8 million . This compares to $8.5 million for the same period in 2024. As of March 31, 2025 , Kura had cash, cash equivalents and short-term investments of $658.2 million , compared to $727.4 million as of December 31, 2024 . As adjusted for the $45 million NDA submission milestone payment earned under our collaboration agreement with Kyowa Kirin, Kura had, on a pro forma basis, $703.2 million in cash, cash equivalents and short-term investments as of March 31, 2025 . Based on our current plans, we believe our cash, cash equivalents and short-term investments as of March 31, 2025 will be sufficient to enable us to fund our current operating expenses into 2027 and, combined with anticipated collaboration funding under the Kyowa Agreement, should support our ziftomenib AML program through commercialization in the frontline combination setting. Forecasted Milestones Present data from the KOMET-001 Phase 1b/2 registration-directed trial in R/R NPM1-m AML at ASCO and EHA in the second quarter of 2025. Present preliminary clinical data from the KOMET-007 Phase 1b expansion cohort evaluating ziftomenib with intensive chemotherapy (7+3) in the frontline setting at EHA in the second quarter of 2025. Present preliminary clinical data from the KOMET-007 Phase 1b expansion cohort evaluating ziftomenib with venetoclax and azacitidine in the frontline setting at a medical meeting in the second half of 2025. Initiate two independent Phase 3 registration-enabling trials in 1L intensive (KOMET-017-IC) and non-intensive (KOMET-017-NIC) AML in the second half of 2025. Nominate a development candidate for next-generation menin inhibitor program in diabetes in mid-2025. Initiate one or more FIT-001 expansion cohorts of KO-2806 and cabozantinib in patients with advanced renal cell carcinoma in the second half of 2025. Present data from the FIT-001 Phase 1 trial evaluating KO-2806 and cabozantinib in patients with renal cell carcinoma in the second half of 2025. Present data from the FIT-001 Phase 1 monotherapy dose escalation of KO-2806 in patients with RAS mutations in the second half of 2025. Present data from the KURRENT-HN trial evaluating tipifarnib and alpelisib in PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC) in the second half of 2025. Conference Call and Webcast Kura’s management will host a webcast and conference call at 4:30 p.m. ET / 1:30 p.m. PT today, May 1, 2025 , to discuss the financial results for the first quarter of 2025 and to provide a corporate update. The live call may be accessed by dialing (800) 245-3047 for domestic callers and (203) 518-9765 for international callers and entering the conference ID: KURAQ1. A live webcast and archived replay of the event will be available here or online from the investor relations section of the Company’s website at www.kuraoncology.com. 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 Breakthrough Therapy Designation from the FDA for the treatment of R/R NPM1-m AML. In November 2024 , Kura Oncology 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 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 HNSCC. 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 Kura’s product candidates, ziftomenib, KO-2806 and tipifarnib; the expected timing of clinical trials; the expected timing and presentation of results and data from clinical trials; the anticipated timing of FDA’s notification on its preliminary evaluation of the NDA for ziftomenib; the potential duration of FDA’s review of the NDA; the potential FDA approval of product candidates; the success and impact of interactions with the FDA; the strength of Kura’s balance sheet and the sufficiency of cash, cash equivalents and short-term investments to fund its current operating plan to 2027 and, combined with anticipated collaboration funding under the Kyowa Agreement, to support Kura’s ziftomenib AML program through commercialization in the 1L combination setting. 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, 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 Investors: Patti Bank Managing Director(415) 513-1284patti.bank@icrhealthcare.com Media: Alexandra Weingarten Associate Director, Corporate Communications (858) 500-8822alexandra@kuraoncology.com Source: Kura Oncology, Inc.