Kura Oncology, Inc. and Kyowa Kirin Co., Ltd. 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.

Kura Oncology公司和Kyowa Kirin公司今日宣布，美国食品药品监督管理局（FDA）已接受Kura的新药申请（NDA），寻求全面批准ziftomenib用于治疗携带核磷蛋白1（NPM1）突变的复发或难治性（R/R）急性髓系白血病（AML）成年患者。

The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 30, 2025..

该申请已获得优先审查，并被分配了《处方药使用者费用法案》（PDUFA）的目标行动日期为2025年11月30日。

“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.

“FDA接受我们的新药申请对Kura和协和麒麟来说是一个重要的里程碑，更重要的是，对于患有这种AML基因亚型的患者而言，他们面临着一种侵袭性强且治疗选择有限的疾病，”Kura Oncology公司总裁兼首席执行官Troy Wilson博士说道。

“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.”.

“这一成就反映了ziftomenib临床数据的强大优势，也体现了我们团队的非凡承诺。我们与协和麒麟的合作伙伴一起，期待在审评过程中继续与FDA密切合作，并为这款有望对患者及其家庭生活产生重要影响的治疗方案的预期上市做好准备。”

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.

新药申请（NDA）基于KOMET-001注册试验的2期结果，该试验针对复发/难治性NPM1突变型（NPM1-m）急性髓系白血病（AML）（临床试验编号：NCT #04067336）。KOMET-001试验达到了其主要终点，即完全缓解（CR）加上部分血液学恢复的完全缓解（CRh），并且主要终点具有统计学显著性。

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..

Ziftomenib耐受性良好，骨髓抑制有限，与ziftomenib相关的停药率为3%。Ziftomenib的安全性和耐受性与之前的报告一致，其获益风险比非常令人鼓舞。

“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.

“成人R/R NPM1-m AML患者面临显著不良的预后，凸显了对创新治疗方案的迫切需求，以改善其治疗结果，”协和麒麟执行副总裁兼首席医学官山下武义博士表示。“这项新药申请的受理是我们通过全面的临床试验不断探索和评估AML各种治疗策略过程中的关键一步。”

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.”.

我们位于协和麒麟和Kura的专职团队全力致力于不懈努力，确保一旦获得批准，能尽快将ziftomenib提供给AML患者。我们认识到这项工作的重要性，并对可能对受这种棘手疾病影响的患者生活产生有意义的影响感到兴奋。"

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.

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 是唯一获得 FDA 授予的突破性疗法资格（BTD）用于治疗复发/难治性 NPM1 突变型急性髓系白血病（AML）的在研疗法。除 BTD 外，ziftomenib 还获得了快速通道和孤儿药资格。

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..

KOMET-001试验中关于ziftomenib在R/R NPM1-m AML患者中的完整数据分析已被选为2025年美国临床肿瘤学会（ASCO）年会的口头报告，时间定于6月2日星期一，并计划在2025年欧洲血液学协会（EHA）大会上进行再次展示。

About NPM1-Mutant AML

关于NPM1突变型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.

急性髓系白血病是成人中最常见的急性白血病，当骨髓产生异常的髓母细胞（白细胞）、红细胞或血小板时开始发病。尽管有许多可用的急性髓系白血病治疗方法，但患者的预后仍然较差，存在很高的未满足需求。Menin通路被认为是该疾病多种基因改变的驱动因素，其中NPM1突变是最常见的之一，约占急性髓系白血病病例的30%。

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.

虽然NPM1突变型急性髓系白血病患者对一线治疗的反应率较高，但复发率也很高，生存结局较差，在复发/难治性情况下的12个月总体生存率仅为30%。此外，NPM1突变常伴随其他疾病相关基因的共突变，包括FLT3、DNMT3A和IDH1/2，预后受这些共突变的存在影响显著。

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..

成人NPM1-m AML患者伴有特定共突变和/或复发/难治性疾病预后较差，二线治疗的中位总生存期仅为约7.8个月，三线为5.3个月，四线之后为3.5个月。目前尚无FDA批准的针对NPM1-m AML的疗法。

About Ziftomenib

关于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.

Ziftomenib 是一种强效且选择性的口服在研 menin 抑制剂，目前正被开发用于治疗具有高度未满足需求的基因定义的 AML 患者。2024 年 4 月，基于 Kura 的 KOMET-001 临床试验数据，ziftomenib 获得了 FDA 针对治疗携带 NPM1 突变的成人 R/R AML 患者的突破性疗法认定（BTD）。

Additional information about clinical trials for ziftomenib can be found at www.kuraoncology.com/clinical-trials/#ziftomenib..

有关 ziftomenib 的临床试验的更多信息，请访问 www.kuraoncology.com/clinical-trials/#ziftomenib。

About Kura Oncology

关于Kura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 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.

Kura Oncology是一家处于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。公司的研发管线包括旨在靶向癌症信号通路的小分子候选药物。2024年11月，Kura Oncology与协和麒麟达成了一项全球战略合作协议，共同开发和商业化用于治疗AML及其他血液恶性肿瘤的menin抑制剂——齐福替尼（ziftomenib）。

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.

KOMET-001 是一项针对 ziftomenib 治疗 R/R NPM1-m AML 的 2 期注册指导试验，其受试者招募已经完成。2025 年第二季度，各公司宣布已提交 ziftomenib 用于治疗成人 R/R NPM1-m AML 的新药申请（NDA）。Kura 和协和麒麟正在开展一系列临床试验，以评估 ziftomenib 联合当前护理标准在初诊及复发/难治性 NPM1-m 和 KMT2A 重排 AML 中的效果。

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.

KO-2806，一种下一代法呢基转移酶抑制剂 (FTI)，正在一项一期剂量递增试验 (FIT-001) 中作为单药疗法及与靶向疗法联用进行评估，用于治疗多种实体瘤患者。Tipifarnib，一种强效且选择性的 FTI，目前正处于一期/二期试验 (KURRENT-HN) 中，与 alpelisib 联合用于治疗依赖 PIK3CA 的头颈部鳞状细胞癌患者。

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.

协和麒麟致力于发现并提供具有改变生命价值的新药物和治疗方法。作为一家总部位于日本的全球专业制药公司，协和麒麟在药物发现和生物技术创新领域已投资超过70年，目前正致力于开发下一代抗体、细胞和基因疗法，这些技术有望帮助那些存在高度未满足医疗需求的患者，例如骨骼与矿物质疾病、难治性血液病/血液肿瘤以及罕见病患者。

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..

对协和麒麟价值观、可持续增长以及让人们微笑的共同承诺将全球的协和麒麟团结在一起。您可以访问 www.kyowakirin.com 了解更多关于协和麒麟的业务信息。