Acute myeloid leukaemia with 11q23 abnormality

Marks the First-Ever Global Pediatric Trial for Menin Inhibitors The PedAL (Pediatric Acute Leukemia) Master Trial is at the heart of LLS's Dare to Dream Project to transform treatment and care for kids with blood cancer WASHINGTON, April 21, 2025 /PRNewswire/ -- The Leukemia & Lymphoma Society (LLS) announced today the first pediatric patient has received treatment in a new subtrial of its Pediatric Acute Leukemia (PedAL) Master Clinical Trial (NCT04726241). PedAL is a first-of-its-kind integrated worldwide master trial that tests multiple targeted drugs simultaneously to accelerate treatments for leukemia, the most common childhood cancer. Infants and children with specific types of relapsed/refractory leukemia (KMT2A-rearranged, NUP98-rearranged, or NPM1-mutant acute leukemia) are now eligible to enroll in the subtrial, which is investigating a combination of chemotherapy and a menin inhibitor. This subtrial involves pediatric patients with some of the most common and hardest-to-treat leukemias. In fact, 80% of infants with acute lymphoblastic leukemia have a genetic rearrangement of the KMT2A gene. About 40% of infants with acute myeloid leukemia have one of the three specific types of leukemia that the trial's treatment targets1. Menin inhibitors are a new and promising class of drugs that block the interaction between other proteins and the menin protein. Research has shown that the menin protein is responsible for driving progression of certain types of leukemia. The new subtrial is open in North America at Children's Hospital of Philadelphia, Cincinnati Children's Hospital, St. Jude Children's Research Hospital, Memorial Sloan Kettering Cancer Center, Lurie Children's Hospital of Chicago and SickKids - The Hospital for Sick Children in Toronto. Global trial sites are open in Spain, the Netherlands, France and Austria. "It is rare for a trial to be so ambitious and forward thinking about the needs of children while studies in adults are still ongoing. The new subtrial is evaluating the safety, efficacy and dosing for the treatment of certain acute leukemias in pediatric patients," says E. Anders Kolb, M.D. President and Chief Executive Officer of The Leukemia & Lymphoma Society. "Historically, cancer treatments have been evaluated in adults first and if successful, then children. This has resulted in a nearly decade-long delay between when therapies are first available for adults compared to children." The new subtrial brings LLS one step closer to achieving its bold goal: By 2040, LLS will enable patients with blood cancer to gain more than one million years of life. New trial expands on PedAL, builds on success of LLS's Dare to Dream Project PedAL launched in 2022 with one treatment subtrial investigating venetoclax. That trial is now open at approximately 80 clinical sites in 19 countries across the globe. Enrollment in the treatment trials begins with the robust PedAL screening trial (NCT04726241), open in 179 sites in the United States, Canada, Australia and New Zealand. To date, the screening trial has enrolled almost 450 children to identify their unique tumor biology and help match them to any targeted treatment clinical trial they are eligible for, whether in or outside of PedAL. Because blood cancers are more common in adults, there is a larger incentive for new treatments to be developed in that population, and progress for pediatric acute leukemia has fallen behind. PedAL is changing this paradigm. A delay in research and the development of therapies for children with aggressive forms of cancer threatens their survival. Today, leukemia is the second leading cause of cancer deaths in children. And for children who survive, 80% develop life-altering and chronic health issues from their treatment. "More children are being diagnosed with leukemia today than ever before," says Kim Schuetz, an oncology nurse, an LLS Dare to Dream Ambassador and mother to Austin, who was diagnosed with acute lymphoblastic leukemia in 2011. "We need to study more treatment options for these children. Through Dare to Dream, we're striving to transform care, so they don't experience a laundry list of life-long health issues and the possibility of developing secondary cancers because of treatment from standard therapies, such as harmful chemotherapies." LLS has supported the development of menin inhibitors over two decades In the early 2000s, LLS provided funding to a group of bright scientific investigators, which led them to discover the menin protein inside cancer cells and its role in the devastating effects of certain forms of leukemia. Over the next several years, LLS provided more than $6 million through its venture philanthropy program, the Therapy Acceleration Program (TAP), to scientists at the University of Michigan. They discovered small molecule inhibitors of the interaction of two proteins, menin and KMT2A/MLL, that, when fused together, drive progression of AML. With TAP funding, the scientists demonstrated the activity of their compounds in the laboratory setting. To facilitate translation of the scientists' progress in the laboratory to the clinic, LLS introduced the scientists to a clinical-stage biopharmaceutical company. Helping children with blood cancer survive and thrive Dare to Dream is transforming treatment and care for kids with blood cancer, so they not only survive but thrive after treatment. PedAL is an integral part of LLS's Dare to Dream Project along with support services, advocacy efforts and funding for blood cancer research: Research: With 35 active academic research grants totaling more than $31 million in funding commitments to pediatric and adolescent and young adult research, LLS-funded investigators are conducting groundbreaking research including exploring the origins of pediatric blood cancers and investigating increased incidence and better treatment options for leukemia in children with Down Syndrome. Advocacy: To ensure that more kids with blood cancer have better access to care, LLS advocates for meaningful changes at the state and federal level. A recent victory was the reauthorization of the Childhood Cancer Survivorship, Treatment, Access and Research (STAR) Act, which expanded childhood cancer research and created plans to enhance the tracking and reporting of pediatric cancer cases and treatment outcomes. Education & Support Services: LLS continues to expand its free patient education and personalized support services for pediatric patients and families to provide guidance, information and comfort during treatment and beyond. "The successes of the Dare to Dream Project, now and in the future, positions us well to reach our bold goal of enabling blood cancer patients to gain more than one million years of life by 2040," says Gwen Nichols, M.D., Chief Medical Officer of LLS. "Advances in treatment and care, spearheaded by Dare to Dream, will give even more children a chance to grow and thrive into adulthood." People can donate to LLS at any time. From May 12-16, all donations made to LLS will go directly to The Dare to Dream Project. Interested in donating to The Dare to Dream Project? Visit this link for more information. About The Leukemia & Lymphoma Society The Leukemia & Lymphoma Society® (LLS) is the global leader in the fight against blood cancer. The LLS mission: Cure blood cancer and improve the quality of life of all patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care. Founded in 1949, LLS has regions throughout the United States and Canada. To learn more, visit . Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET. For additional information, visit lls.org/lls-newsnetwork. Follow us on Facebook, X, Instagram, LinkedIn and TikTok. About The Dare to Dream Project Dare to Dream exists to transform treatment and care for kids with blood cancer. Because kids are different and need to be treated differently, Dare to Dream funds groundbreaking research and the LLS PedAL Master Clinical Trial as well as expands LLS support services and drives advocacy efforts to help all kids with blood cancers get accessible, affordable, quality healthcare. LLS proudly and gratefully acknowledges all those contributing to the LLS mission, including corporate partners Walgreens, Valvoline™ Global Operations, The Wawa Foundation, and Subaru of America, Inc. and its retailers who have each pledged over $1 million to Dare to Dream. LLS also recognizes Sarah Asma and the Moore family; The Don & Lorraine Freeberg Foundation; The Harry T. Mangurian Jr. Foundation; Norcross Foundation, Inc.; Joan and Paul Rubschlager; and The Bobby Zahurak Pediatric AML Research and Patient Support Fund for their outstanding contributions to Dare to Dream. For more information about Dare to Dream, including resources for patients and their families, trial details, and eligibility, visit: lls.org/dare-to-dream. About LLS PedAL As part of LLS's Dare to Dream Project, the Pediatric Acute Leukemia Master Trial (PedAL) is the first-of-its-kind global master clinical trial for pediatric acute leukemia patients that will fundamentally change how children are treated. Prior to enrolling in therapeutic trials, patients enroll in the PedAL Screening Trial (NCT04726241) to identify the unique tumor biology of each child's cancer and help them to match with the most promising treatment. The Screening Trial is currently open at multiple sites in the United States, Canada, Australia, and New Zealand. At this time, two PedAL therapeutic trials are open and actively enrolling patients in the U.S., Canada, Australia, New Zealand, Japan, and Europe, with more therapeutic trials planned for global execution. To learn more about PedAL, visit lls.org/dare-to-dream. PedAL would not be possible without the support of major foundation donors such as Gateway for Cancer Research, which pledged $1.5 million over three years to support genomic sequencing and flow cytometry, which is being conducted through the PedAL screening trial, and the Lisa Dean Moseley Foundation, which has committed $1.25 million over five years to support the PedAL Principal Investigators Fellowship Program, whose members are leading the PedAL screening trial and therapeutic trials. Media Contact: Sandra Salviejo Senior Director, Communications [email protected] Reference: 1 Juul-Dam KL et al. European Journal of Medical Genetics 2023 SOURCE The Leukemia & Lymphoma Society (LLS) WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

– KOMET-001 registrational trial in R/R NPM1-mutant AML achieved primary endpoint – – Alignment reached with FDA and EMA on key aspects of the KOMET-017 protocol including use of MRD-negative CR endpoint for potential U.S. accelerated approval pathway in frontline intensive chemotherapy trial – – Topline MRD-negative CR results from KOMET-017-IC Phase 3 trial anticipated in 2028 – – Multiple data presentations for ziftomenib and pipeline programs expected throughout 2025 – – $727.4 million in cash, together with anticipated collaboration agreement payments, 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 , Feb. 26, 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 fourth quarter and full year 2024 financial results and provided a corporate update. “We are very pleased the KOMET-001 registrational trial achieved its primary endpoint. We look forward to sharing topline data at an upcoming medical conference and expect to submit a New Drug Application (NDA) in relapsed/refractory (R/R) NPM1-mutant acute myeloid leukemia (AML) next quarter,” said Troy Wilson , Ph.D., J.D., President and Chief Executive Officer of Kura Oncology . “In parallel, we are advancing ziftomenib into registrational studies in the frontline (1L) setting. Approximately half of patients with newly diagnosed NPM1-mutated (NPM1-m) AML and 80% of patients with KMT2A-rearranged (KMT2A-r) AML will die from the disease within five years. Given this unmet need, we are pleased to have reached alignment with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) on key aspects of our Phase 3 trials, including the use of minimum residual disease (MRD)-negative complete response (CR) as a primary endpoint for potential accelerated approval in the U.S. , with topline results anticipated in 2028. Our KOMET-017 trial is breaking new ground, and we and our partners at Kyowa Kirin are working as rapidly as possible to bring ziftomenib to AML patients worldwide.” Recent Highlights Positive topline results from registration-directed trial of ziftomenib in R/R NPM1-m AML – Kura and Kyowa Kirin announced positive topline results from KOMET-001, the Phase 2 registration-directed trial of ziftomenib in patients with R/R NPM1-m AML. The KOMET-001 trial achieved its primary endpoint, consistent with the targeted 20-30% CR/CR with partial hematological recovery (CRh) rate, and data have been submitted for presentation at ASCO. The benefit-risk profile for ziftomenib is highly encouraging, and safety and tolerability were consistent with previous reports. Facilitated by the Breakthrough Therapy Designation status of ziftomenib in R/R NPM1-m AML, the Company completed its pre-NDA meeting with FDA and anticipates submitting an NDA in the second quarter of 2025. Positive feedback from FDA for 1L combination trial designs – Earlier this month, Kura and Kyowa Kirin announced alignment with FDA on the KOMET-017 global trial protocol evaluating ziftomenib in combination with both intensive and non-intensive combination regimens in patients with newly diagnosed NPM1-m and/or KMT2A-r AML. This includes alignment on potential pathways for accelerated approval in the U.S. in both the KOMET-017-IC (intensive chemotherapy) and KOMET-017-NIC (non-intensive chemotherapy) trials by allowing the trials to use MRD-negative CR and CR, as primary endpoints, respectively. The companies also gained alignment with the EMA on the KOMET-017 protocol, and expect to initiate the KOMET-017 Phase 3 trials in the second half of 2025. Positive clinical data for Phase 1 trial of ziftomenib in combination with standards of care – In December 2024 , Kura Oncology and Kyowa Kirin announced encouraging clinical data from KOMET-007, a Phase 1 trial of ziftomenib in combination with standards of care, including cytarabine/daunorubicin (7+3) and venetoclax/azacitidine in patients with NPM1-m and KMT2A-r AML. Among response-evaluable patients enrolled in the 7+3 combination cohort for patients with 1L NPM1-m or KMT2A-r adverse risk AML, 91% achieved a CR (100% for NPM1-m, 83% for KMT2A-r patients). Ziftomenib was generally well tolerated in combination at all dose levels evaluated across all cohorts in the Phase 1a dose-escalation portion of the trial. The positive results from KOMET-007 reported at ASH reinforce the companies’ commitment to evaluating ziftomenib across the continuum of 1L AML treatment options. Global strategic collaboration with Kyowa Kirin to develop and commercialize ziftomenib in acute leukemias – In November 2024 , Kura Oncology and Kyowa Kirin announced they entered into a global strategic collaboration to develop and commercialize ziftomenib (Kyowa Agreement), funding the expansive AML development program through U.S. commercialization in 1L combinations. Under the partnership, Kura retains leadership and key strategic rights to ziftomenib in the U.S. and preserves strategic flexibility, while enabling development and commercialization of ziftomenib across the continuum of care in acute leukemias, including both fit and unfit 1L indications, post-transplant maintenance setting and combinations with targeted therapies. Preclinical data supporting opportunity for ziftomenib in treatment of gastrointestinal stromal tumors (GIST) – In October 2024 , Kura reported preclinical data presented 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 GIST patients. 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. Sixty percent of patients develop resistance to imatinib within two years and ziftomenib has the potential to delay the onset of resistance to, or overcome resistance in patients pre-treated with, imatinib. In August 2024 , Kura announced clearance by the FDA of an IND application and the Company remains on track to initiate the KOMET-015 trial evaluating ziftomenib plus imatinib in patients with advanced GIST, in the first half of 2025. Clinical and preclinical data support combinations of farnesyl transferase inhibitors with targeted therapies – Despite multiple advances, innate and adaptive resistance remain a challenge for many classes of targeted therapies in cancer. A growing body of clinical and preclinical data demonstrates the potential of farnesyl transferase inhibitors as companion therapeutic agents to augment the antitumor activities of various targeted therapies and overcome resistance in combination. Enrollment in the FIT-001 trial evaluating our next-generation farnesyl transferase inhibitor KO-2806 continues to progress and the dose-escalation portion of the KURRENT-HN trial with tipifarnib is now complete. Kura expects to present the first clinical data for KO-2806 as a monotherapy and in combination, as well as clinical data from the KURRENT-HN trial, in the second half of 2025. Financial Results Collaboration revenue from our Kyowa Kirin partnership for the fourth quarter and full year 2024 was $53.9 million , compared to no revenue in 2023. Research and development (R&D) expenses for the fourth quarter of 2024 were $52.3 million , compared to $32.5 million for the fourth quarter of 2023. R&D expenses for the full year 2024 were $170.0 million , compared to $115.2 million for the prior year. General and administrative (G&A) expenses for the fourth quarter of 2024 were $24.1 million , compared to $14.2 million for the fourth quarter of 2023. G&A expenses for the full year 2024 were $77.1 million , compared to $50.6 million for the prior year. Net loss for the fourth quarter of 2024 was $19.2 million , compared to a net loss of $42.8 million for the fourth quarter of 2023. Net loss for the full year 2024 was $174.0 million , compared to a net loss of $152.6 million for the prior year. Net loss for the fourth quarter and full year 2024 included non-cash, share-based compensation expense of $8.6 million and $33.9 million , respectively. This compares to $7.2 million and $28.1 million for the same periods in 2023. As of December 31, 2024 , Kura had cash, cash equivalents and short-term investments of $727.4 million , including the upfront payment of $330.0 million from Kyowa Kirin, compared to $424.0 million as of December 31, 2023 . Based on our current plans, we believe our cash, cash equivalents and short-term investments as of December 31, 2024 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 1L combination setting. Forecasted Milestones Submit an NDA for ziftomenib in R/R NPM1-m AML in the second quarter of 2025. Present topline data from KOMET-001 Phase 2 registration-directed trial in R/R NPM1-m AML 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) at a medical meeting in the second quarter of 2025. Initiate the KOMET-015 trial evaluating ziftomenib and imatinib in patients with advanced GIST in the first 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. Present preliminary clinical data from the KOMET-007 Phase 1b expansion cohort evaluating ziftomenib with venetoclax and azacitidine at a medical meeting 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 expansion cohorts of KO-2806 and cabozantinib in patients with advanced renal cell carcinoma in the first half of 2025. Present data from the Phase 1 monotherapy dose escalation of KO-2806 in patients with RAS mutations in the second half of 2025. Present data from the Phase 1 trial evaluating KO-2806 and cabozantinib in patients with renal cell carcinoma in the second half of 2025. Present data from the dose escalation portion of 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, February 26, 2025 , to discuss the financial results for the fourth quarter and full year 2024 and to provide a corporate update. The live call may be accessed by dialing (800) 579-2543 for domestic callers and (785) 424-1789 for international callers and entering the conference ID: KURAQ4. A live webcast and archived replay of the event will be available here or online from the investor relations section of the company 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 targeting 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 a global strategic collaboration agreement with Kyowa Kirin Co., Ltd. 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 the companies anticipate submission of an NDA to the FDA in the second quarter of 2025. Kura Oncology and Kyowa Kirin are also 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-r AML. KO-2806, a next-generation farnesyl transferase inhibitor, is being evaluated in a Phase 1 dose-escalation trial as a monotherapy and in combination with targeted therapies. Tipifarnib, a potent and selective farnesyl transferase inhibitor, is currently in a Phase 1/2 trial 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, tipifarnib and KO-2806; plans, trial designs and expected timing of clinical trials; the expected timing and presentation of results and data from clinical trials; the anticipated timing of submission of an NDA for ziftomenib; the potential for U.S. accelerated approval and full approval of product candidates; and the success and impact of interactions with the FDA; the potential for menin inhibitors to shift the treatment paradigm for GIST; 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, 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. 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.

– KOMET-001 registrational trial in R/R NPM1-m AML achieved primary CR/CRh endpoint; topline data submitted for presentation at an upcoming medical meeting – – NDA submission for ziftomenib on track for 2Q 2025 – – Registrational KOMET-017-IC trial of intensive chemotherapy combination will assess MRD negative CR and EFS as dual-primary endpoints to support potential U.S. accelerated approval and full approval – – Registrational KOMET-017-NIC trial of venetoclax / azacitidine combination will assess CR and OS as dual-primary endpoints to support potential U.S. accelerated approval and full approval – – KOMET-017-IC and NIC Phase 3 trials expected to initiate in 2H 2025 – – Multiple clinical data presentations for ziftomenib and pipeline programs anticipated throughout 2025 – – Kura management to host virtual investor event today at 4:30 p.m. ET – SAN DIEGO , Feb. 05, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today announced positive topline results from KOMET-001, the Phase 2 registration-directed trial of ziftomenib, a highly selective, once-daily, oral investigational menin inhibitor, in patients with relapsed/refractory (R/R) NPM1-mutant (NPM1-m) acute myeloid leukemia (AML). Topline data for KOMET-001 has been submitted for presentation at an upcoming medical conference in the second quarter of 2025, and Kura is on track to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ziftomenib in the second quarter of 2025. The companies, which announced their joint collaboration to commercialize ziftomenib in 2024, also announced they plan to initiate a single protocol containing two independently powered, randomized, double-blind, placebo-controlled, registrational Phase 3 trials to evaluate ziftomenib in combination with both intensive and non-intensive combination regimens in patients with newly diagnosed NPM1-m and KMT2A-rearranged (KMT2A-r) AML, following successful interactions with the FDA. Each frontline trial design includes dual-primary endpoints to support potential U.S. accelerated approval and full approval. The companies plan to initiate the two frontline Phase 3 trials in the second half of 2025 and anticipate multiple clinical data presentations for the ziftomenib AML program as well as Kura’s pipeline programs in 2025. “We are excited to report positive topline results in R/R NPM1-m AML patients, underscoring the strong foundation for our ziftomenib program to potentially transform the treatment landscape for these patients. We appreciate the commitment and dedication from our team as well as our partners at Kyowa Kirin,” said Troy Wilson , Ph.D., J.D., President and Chief Executive Officer of Kura Oncology . “We believe this achievement for our KOMET-001 trial positions Kura and Kyowa Kirin to deliver on its path to commercialization of ziftomenib, beginning with our potential first NDA submission in R/R NPM1-m AML next quarter. Furthermore, we believe the positive FDA interactions for the KOMET-017 protocol, including the opportunity for accelerated approval in both trials, pave the way for us to position ziftomenib as a potential frontline therapy to address up to 50% of patients with AML.” Positive KOMET-001 Ziftomenib Monotherapy Trial in R/R NPM1-m AML Kura and Kyowa Kirin today announced positive topline results from KOMET-001, a Phase 2 registration-directed trial of ziftomenib, in patients with R/R NPM1-m AML. The KOMET-001 trial achieved its primary endpoint of CR plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. The benefit-risk profile for ziftomenib is highly encouraging, and safety and tolerability were consistent with previous reports. 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. Full results from the KOMET-001 trial will be presented at a future medical meeting in the second quarter of 2025. After successful FDA interactions in part facilitated by BTD, Kura announced that it is on track to submit an NDA to the FDA for ziftomenib for the treatment of patients with R/R NPM1-mutant AML in the second quarter of 2025. Positive FDA Feedback for Upcoming Frontline Combination Trial Designs Kura and Kyowa Kirin recently announced plans for KOMET-017, a global protocol evaluating ziftomenib in combination with standards of care for adults with newly diagnosed NPM1-m or KMT2A-r AML. Following successful End-of-Phase 1 meetings with the FDA, the companies announced they will proceed with plans to initiate the KOMET-017 trial, comprising of two independent, global, randomized, double-blind, placebo-controlled Phase 3 trials to evaluate ziftomenib in combination with both intensive and non-intensive combination regimens in patients with newly diagnosed NPM1-m and/or KMT2A-r AML. The positive feedback from the FDA, along with data from the KOMET-007 trial presented at the 2024 American Society of Hematology Annual Meeting, reinforces Kura’s and Kyowa Kirin’s commitment to evaluating ziftomenib in patients across the continuum of frontline treatment options. The registrational KOMET-017-IC (Intensive Combination) trial will evaluate the combination of ziftomenib with induction chemotherapy (7+3) in newly diagnosed NPM1-m and KMT2A-r AML patients. Patients will be randomized to receive ziftomenib or placebo, in combination with standard induction, consolidation chemotherapy and post consolidation maintenance. The KOMET-017-IC trial will assess minimum residual disease (MRD) negative complete response (CR) and event-free survival (EFS) as dual-primary endpoints to support potential U.S. accelerated approval and full approval, respectively and is anticipated to be initiated in the second half of 2025. The registrational KOMET-017-NIC (Non-Intensive Combination) trial will evaluate the combination of ziftomenib with venetoclax plus azacitidine in newly diagnosed NPM1-m patients unfit to receive intensive chemotherapy. The KOMET-017-NIC trial will assess CR and overall survival (OS) as dual-primary endpoints to support potential U.S. accelerated approval and full approval, respectively. Patients will be randomized to receive ziftomenib or placebo, in combination with venetoclax and azacitidine. The KOMET-017-NIC trial is anticipated to be initiated in the second half of 2025. “Even with approved therapies, up to 70% of patients who achieve a first CR will see their AML return within 3 years. The 5-year survival rate for AML is 31.9% and as low as 11.2% for patients aged older than 65 years,” said Mollie Leoni , M.D., Chief Medical Officer of Kura Oncology . “Given this urgent need, we are pleased with the outcome of these FDA interactions and look forward to initiating our Phase 3 trials to establish the benefit-risk profile of ziftomenib in both the intensive and non-intensive chemotherapy settings. We were particularly pleased by the FDA’s willingness to allow the trials to use MRD negative CR and CR as primary endpoints for accelerated approval in the two populations. In so doing, the KOMET-017 protocol is breaking new ground, which may help deliver ziftomenib more quickly to patients living with this devastating disease.” “Starting patients on combination therapy early is essential to improving outcomes in AML,” said Takeyoshi Yamashita , Ph.D., Senior Managing Executive Officer and Chief Medical Officer of Kyowa Kirin . “The data from the completed KOMET-001 trial and FDA feedback on the planned KOMET-017 protocol strengthens our confidence these trials may offer valuable treatment options for patients throughout the continuum of treatment. We remain committed to working with our colleagues at Kura to bring ziftomenib as rapidly as possible to AML patients worldwide.” MRD is a term describing small numbers of leukemic cells, which are still detectable during or after treatment, even when a patient has achieved CR by standard criteria. Remaining leukemia cells in the body can become active and start to multiply, resulting in a relapse of the disease, which may be fatal for patients. Achieving MRD negativity, which may be associated with longer remissions and improved survival, means that a treatment has reduced the number of leukemic cells to below the limit of detection by the most sensitive analytical methods. “We carefully designed KOMET-017 to allow patients to go on the same protocol but on one of the two sub-studies based on whether they are fit or unfit for intensive chemotherapy, and this approach is intended to be very patient centric, facilitate rapid enrollment and offer operational advantages to the study sites,” said Amer Zeidan , MBBS, MHS, chief of the Division of Hematologic Malignancies , director of Hematology Early Therapeutics Research at Yale Cancer Center , and the lead investigator of the KOMET-017 trial. “Further, we designed KOMET-017 to allow for a potential accelerated approval based on endpoints that have been widely accepted as surrogates for meaningful clinical benefit in these patient populations,” Dr Zeidan added. “The association between MRD negativity and improved survival in patients with NPM1-mutated AML is well established in the literature. AML experts around the world recommend monitoring MRD in patients to guide treatment decisions. The best opportunity to achieve long-lasting remission and extend survival is to achieve MRD negativity with the first attempt at treatment. Therefore, using MRD negative CR as an approvable endpoint in AML is very innovative and could allow faster availability of therapies to our patients,” Dr Zeidan concluded. 2025 Anticipated Clinical Data Highlights Kura and Kyowa Kirin expect to present multiple clinical data updates from their ziftomenib AML program, and Kura expects to present updates from its KO-2806 and tipifarnib programs, in 2025 as follows: Topline data from the KOMET-001 trial of ziftomenib monotherapy in R/R NPM1-m AML (2Q 2025) KOMET-007 Phase 1b data of ziftomenib in combination with 7+3 in newly diagnosed NPM1-m AML and KMT2A-r AML (2Q 2025) FIT-001 Phase 1 data of KO-2806 monotherapy in HRAS-mutant and KRAS-mutated solid tumors (2H 2025) FIT-001 Phase 1 data of KO-2806 in combination with cabozantinib in renal cell carcinoma (RCC) (2H 2025) KURRENT-HN Phase 1 data of tipifarnib in combination with alpelisib in PIK3CA-dependent head and neck squamous cell carcinoma (HNSCC) (2H 2025) KOMET-007 Phase 1b data of ziftomenib in combination with venetoclax / azacitidine in NPM1-m AML (2H 2025) Virtual Investor Event Kura will host a webcast and conference call featuring company management today at 4:30 p.m. ET . Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The link to the live webcast will also be available on the Investors section of Kura’s website, with an archived replay available shortly after the event. About AML AML primarily affects adults and is one of the most difficult-to-treat blood cancers. AML starts in the bone marrow and can quickly move to the blood and other parts of the body including the lymph nodes, spleen and central nervous system. Approximately 20,000 Americans are diagnosed with AML each year, with an NPM1 genetic mutation or KMT2A rearrangement found in approximately 35% of cases. Relapse in AML is common, and despite available treatments, nearly 11,000 Americans will die from the disease each year. About Ziftomenib Ziftomenib is a selective and oral menin inhibitor currently in development for the treatment of genetically defined AML patients with high unmet need. In April 2024, ziftomenib received BTD by the FDA for the treatment of R/R NPM1-mutant AML based on data from Kura’s KOMET-001 clinical trial. Additional information about clinical trials for ziftomenib can be found at 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 that 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 relapsed/refractory (R/R) NPM1-m AML. In November 2024 , Kura entered a global strategic collaboration agreement with Kyowa Kirin Co., Ltd. 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-mutant AML has been completed, and the companies anticipate submission of a New Drug Application in the second quarter of 2025. Kura and Kyowa Kirin are also conducting a series of clinical trials to evaluate ziftomenib in combination with current standards of care in newly diagnosed and R/R NPM1-mutant and KMT2A-rearranged AML. KO-2806, a next-generation farnesyl transferase inhibitor, is being evaluated in a Phase 1 dose-escalation trial as a monotherapy and in combination with targeted therapies. Tipifarnib, a potent and selective FTI, is currently in a Phase 1/2 trial 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. 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 therapeutic potential and potential success of ziftomenib, KO-2806 and tipifarnib; plans, trial designs and expected timing of clinical trials; the expected timing and presentation of data from clinical trials; the anticipated timing of submission of a New Drug Application for ziftomenib; the potential for U.S. accelerated approval and full approval of product candidates; and the success and impact of interactions with the FDA. 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 that 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. Amer Zeidan has consulted for and received honoraria from Kura. Opinions expressed are his own and do not necessarily represent those of his employer. Kura Contacts Investors: Patti Bank Managing Director(415) 513-1284patti.bank@icrhealthcare.com Media: Alexandra Weingarten Associate Director, Corporate Communications & Investor Relations(858) 500-8822alexandra@kuraoncology.com Kyowa Kirin Contacts Wataru Suzuki Corporate Communications Department – Globalmedia@kyowakirin.com Lisa Popyk Senior Director, Corporate Communications – Kyowa Kirin North America lisa.popyk.3z@kyowakirin.com Source: Kura Oncology, Inc.