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).

圣地亚哥，2025年2月5日（环球通讯社）--Kura Oncology，Inc.（纳斯达克：Kura，“Kura”）和Kyowa Kirin Co.，Ltd.（东京证交所：4151，“Kyowa Kirin”）今天宣布，KOMET-001（一种高选择性，每日一次的口服研究性menin抑制剂）治疗复发/难治性（R/R）NPM1突变体（NPM1-m）急性髓细胞白血病（AML）患者的2期注册指导试验）的阳性结果。

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..

KOMET-001的Topline数据已在2025年第二季度即将举行的医学会议上提交，Kura有望在2025年第二季度向美国食品和药物管理局（FDA）提交ziftomenib的新药申请（NDA）。。

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.

这些公司于2024年宣布联合合作将ziftomenib商业化，并宣布他们计划启动一项单一方案，其中包含两项独立的，随机的，双盲的，安慰剂对照的注册3期试验，以评估ziftomenib联合强化和非强化联合方案治疗新诊断的NPM1-m和KMT2A重排（KMT2A-r）AML患者，与FDA成功互动后。

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..

每个前线试验设计都包括两个主要终点，以支持潜在的美国加速批准和完全批准。这些公司计划在2025年下半年启动两项前线3期试验，并预计2025年将为ziftomenib AML计划和Kura的管道计划提供多项临床数据。。

“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.

Kura Oncology总裁兼首席执行官特洛伊·威尔逊（TroyWilson）博士说：“我们很高兴报告NPM1-m急性髓细胞白血病患者的积极治疗结果，这突显了我们的ziftomenib计划有可能改变这些患者的治疗前景的坚实基础。我们感谢我们的团队以及我们在协和麒麟的合作伙伴的承诺和奉献精神。”。

“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.”.

“我们相信，我们的KOMET-001试验取得的这一成就使Kura和Kyowa Kirin能够实现ziftomenib的商业化，从我们下个季度在R/R NPM1-m AML中可能提交的第一份NDA开始。此外，我们相信FDA对KOMET-017协议的积极互动，包括在两项试验中加速批准的机会，为我们将ziftomenib定位为潜在的一线治疗方法，以解决多达50%的AML患者铺平了道路。”。

Positive KOMET-001 Ziftomenib Monotherapy Trial in R/R NPM1-m AML

R/R NPM1-m AML中KOMET-001 Ziftomenib单药治疗试验阳性

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.

Kura和Kyowa Kirin今天宣布了KOMET-001的阳性结果，KOMET-001是ziftomenib的2期注册指导试验，用于R/R NPM1-m AML患者。KOMET-001试验达到了CR加CR的主要终点，部分血液学恢复（CRh），主要终点具有统计学意义。

The benefit-risk profile for ziftomenib is highly encouraging, and safety and tolerability were consistent with previous reports..

ziftomenib的益处-风险概况非常令人鼓舞，安全性和耐受性与之前的报道一致。。

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.

KOMET-001注册指导试验旨在评估ziftomenib的临床活性，安全性和耐受性证据，ziftomenib是唯一接受FDA突破性治疗指定（BTD）治疗R/R NPM1突变AML的研究性治疗。KOMET-001试验的全部结果将在2025年第二季度的未来医学会议上公布。

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..

在BTD部分促进了FDA的成功互动后，Kura宣布正在向FDA提交ziftomenib的NDA，以在2025年第二季度治疗R/R NPM1突变型AML患者。。

Positive FDA Feedback for Upcoming Frontline Combination Trial Designs

FDA对即将进行的前线联合试验设计的积极反馈

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.

。在成功结束与FDA的第一阶段会议后，这些公司宣布将着手启动KOMET-017试验的计划，该试验包括两项独立的，全球的，随机的，双盲的，安慰剂对照的3期试验，以评估ziftomenib联合强化和非强化联合方案治疗新诊断的NPM1-m和/或KMT2A-r AML患者。

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..

FDA的积极反馈，以及2024年美国血液学会年会上提交的KOMET-007试验数据，强化了Kura和Kyowa Kirin在一线治疗方案连续性中评估ziftomenib患者的承诺。。

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.

注册KOMET-017-IC（强化联合）试验将评估ziftomenib与诱导化疗（7+3）在新诊断的NPM1-m和KMT2A-r AML患者中的联合应用。。

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..

KOMET-017-IC试验将评估最小残留病（MRD）阴性完全缓解（CR）和无事件生存（EFS）作为双重主要终点，分别支持潜在的美国加速批准和完全批准，预计将于2025年下半年启动。。

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.

注册KOMET-017-NIC（非强化联合）试验将评估ziftomenib与venetoclax加阿扎胞苷联合治疗新诊断的NPM1-m患者不适合接受强化化疗。KOMET-017-NIC试验将评估CR和总生存期（OS）作为双重主要终点，以支持潜在的美国。

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..

加速批准和完全批准。。KOMET-017-NIC试验预计将于2025年下半年启动。。

“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.

Kura肿瘤学首席医学官Mollie Leoni医学博士说：“即使采用批准的治疗方法，高达70%的首次CR患者将在3年内恢复AML。AML的5年生存率为31.9%，65岁以上患者的5年生存率低至11.2%。”。“鉴于这一迫切需求，我们对这些FDA相互作用的结果感到满意，并期待启动我们的3期临床试验，以确定ziftomenib在强化和非强化化疗环境中的益处-风险概况。

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.”.

我们特别高兴的是，FDA愿意允许试验使用MRD阴性CR和CR作为两个人群加速批准的主要终点。通过这样做，KOMET-017协议正在开辟新的领域，这可能有助于更快地向患有这种毁灭性疾病的患者提供ziftomenib。”。

“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.

Kyowa Kirin高级管理执行官兼首席医疗官Takeyoshi Yamashita博士说：“尽早开始联合治疗对于改善AML的预后至关重要。”来自已完成的KOMET-001试验的数据和FDA对计划中的KOMET-017方案的反馈增强了我们的信心，这些试验可能会在整个治疗过程中为患者提供有价值的治疗选择。

We remain committed to working with our colleagues at Kura to bring ziftomenib as rapidly as possible to AML patients worldwide.”.

我们仍然致力于与Kura的同事合作，尽快将ziftomenib带给全世界的AML患者。”。

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.

MRD是一个描述少量白血病细胞的术语，即使患者按照标准达到CR，在治疗期间或之后仍然可以检测到。体内剩余的白血病细胞可能变得活跃并开始繁殖，导致疾病复发，这可能对患者致命。

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..

实现MRD阴性可能与更长的缓解期和改善的生存期有关，这意味着一种治疗已经将白血病细胞的数量减少到最敏感的分析方法检测限以下。。

“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.

“我们精心设计了KOMET-017，允许患者按照相同的方案进行，但根据他们是否适合强化化疗进行两个子研究之一，这种方法旨在以患者为中心，促进快速注册，并为研究地点提供操作优势，”血液恶性肿瘤科科长，耶鲁大学癌症中心血液学早期治疗研究主任，KOMET-017试验首席研究员Amer Zeidan，MBBS，MHS说。

“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.

Zeidan博士补充道：“此外，我们设计了KOMET-017，以允许基于终点的潜在加速批准，这些终点已被广泛接受为这些患者群体中有意义的临床益处的替代指标。”。“文献中已经充分证实了MRD阴性与NPM1突变AML患者生存率提高之间的关联。

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..

世界各地的反洗钱专家建议监测患者的MRD，以指导治疗决策。实现长期缓解和延长生存期的最佳机会是在首次尝试治疗时达到MRD阴性。因此，使用MRD阴性CR作为AML的可批准终点是非常创新的，可以更快地为我们的患者提供治疗，”Zeidan博士总结道。。

2025 Anticipated Clinical Data Highlights

2025年预期临床数据亮点

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:

Kura和Kyowa Kirin预计将在2025年提供其ziftomenib AML计划的多项临床数据更新，Kura预计将在2025年提供其KO-2806和tipifarnib计划的更新，如下所示：

About AML

关于反洗钱

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.

AML主要影响成年人，是最难治疗的血癌之一。急性髓细胞白血病始于骨髓，可以迅速转移到血液和身体的其他部位，包括淋巴结、脾脏和中枢神经系统。每年大约有20000名美国人被诊断出患有AML，在大约35%的病例中发现了NPM1基因突变或KMT2A重排。

Relapse in AML is common, and despite available treatments, nearly 11,000 Americans will die from the disease each year..

AML复发很常见，尽管有可用的治疗方法，但每年仍有近11000名美国人死于该疾病。。

About Ziftomenib

关于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..

Ziftomenib是一种选择性口服menin抑制剂，目前正在开发中，用于治疗未满足需求的基因定义的AML患者。2024年4月，ziftomenib根据Kura的KOMET-001临床试验数据，接受了FDA的BTD治疗R/R NPM1突变型AML。有关ziftomenib临床试验的更多信息，请访问kuraoncology.com/clinical-trials/ziftomenib。。

About Kura Oncology

关于库拉肿瘤学

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.

Kura Oncology是一家临床阶段的生物制药公司，致力于实现精准药物治疗癌症的承诺。该公司的管道由靶向癌症信号传导途径的小分子候选药物组成。Ziftomenib是一种每日一次的口服menin抑制剂，是第一种也是唯一一种接受FDA BTD治疗复发/难治性（R/R）NPM1-m AML的研究性疗法。

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.

2024年11月，Kura与Kyowa Kirin Co.，Ltd.签订了全球战略合作协议，开发用于AML和其他血液系统恶性肿瘤的ziftomenib并将其商业化。ziftomenib在R/R NPM1突变型AML中的第二阶段注册指导试验已经完成，这些公司预计在2025年第二季度提交新药申请。

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.

Kura和Kyowa Kirin也正在进行一系列临床试验，以评估ziftomenib与新诊断和R/R NPM1突变体和KMT2A重排AML的当前护理标准相结合。下一代法尼基转移酶抑制剂KO-2806正在1期剂量递增试验中作为单一疗法和靶向疗法进行评估。

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..

Tipifarnib是一种有效且选择性的FTI，目前正在与alpelisib联合进行1/2期试验，用于PIK3CA依赖性头颈部鳞状细胞癌患者。有关更多信息，请访问Kura的网站https://kuraoncology.com/并在X和LinkedIn上关注我们。。

About Kyowa Kirin

关于协和麒麟

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.

协和麒麟旨在发现并提供具有改变生命价值的新药和治疗方法。作为一家总部位于日本的全球专业制药公司，协和麒麟投资于药物发现和生物技术创新已有70多年的历史，目前正在致力于设计下一代抗体以及细胞和基因疗法，这些疗法有可能帮助骨矿、难治性血液病/血液肿瘤学和罕见病等高度未满足医疗需求的患者。

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..

共同致力于共同遵守麒麟的价值观，实现可持续发展，并让人们微笑，这使全球的麒麟团结在一起。你可以在www.kyowakirin.com上了解更多关于麒麟的业务。。

Forward-Looking Statements

前瞻性声明

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.

本新闻稿包含某些涉及风险和不确定性的前瞻性陈述，这些风险和不确定性可能导致实际结果与历史结果或此类前瞻性陈述所表达或暗示的任何未来结果存在重大差异。这些前瞻性声明包括关于ziftomenib，KO-2806和tipifarnib的治疗潜力和潜在成功的声明；临床试验的计划，试验设计和预期时间；临床试验数据的预期时间和呈现；ziftomenib新药申请提交的预期时间；美国的潜力。

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.

加速批准和全面批准候选产品；以及与FDA互动的成功和影响。可能导致实际结果产生重大差异的因素包括：在早期研究或临床试验中看起来有希望的化合物在后来的临床前研究或临床试验中没有显示出安全性和/或有效性的风险，Kura可能无法获得批准以销售其候选产品的风险，与进行临床试验，监管备案，应用以及与监管机构的其他互动相关的不确定性，与依赖第三方成功进行临床试验相关的风险，与依赖外部融资以满足资本要求相关的风险，以及与发现，开发和商业化安全有效用作人类治疗药物的过程相关的其他风险，以及围绕这些药物建立业务的努力。

You are urged to consider statements that include the words “may,” “will,” “would,” “could,” “should,” “believes,” “estimates,” “projects,” “promise,” “potential,” “.

敦促您考虑包含“可能”、“将”、“将”、“可能”、“应该”、“相信”、“估计”、“项目”、“承诺”、“潜力”等词语的陈述。

www.sec.gov

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.

。此类前瞻性声明仅在发布之日是最新的，Kura没有义务更新任何前瞻性声明，无论是由于新信息、未来事件还是其他原因。

Amer Zeidan has consulted for and received honoraria from Kura. Opinions expressed are his own and do not necessarily represent those of his employer.

Amer Zeidan曾为Kura提供咨询并获得酬金。表达的意见是他自己的，不一定代表雇主的意见。

Source: Kura Oncology, Inc.

资料来源：库拉肿瘤公司。

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