Quizartinib Hydrochloride

– Expanding clinical experience and safety profile of ziftomenib support its evaluation in combination with approved FLT3 inhibitors in frontline AML – – FLT3 mutations occur in approximately 30% of newly diagnosed adult patients with AML and up to 50% of adult patients with NPM1-m AML, making FLT3 one of the most common genetic alterations in AML – – Ziftomenib clinical trials are now active in multiple frontline settings that include up to 50% of incident patients with AML in the U.S. – SAN DIEGO and TOKYO , Oct. 01, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today announced dosing of the first patient in a cohort of the KOMET-007 clinical trial (NCT05735184). This cohort evaluates ziftomenib, a once-daily, investigational oral menin inhibitor, combined with cytarabine and daunorubicin (7+3) as well as quizartinib, for patients with newly diagnosed acute myeloid leukemia (AML). Despite recent advances, including regulatory approvals of FLT3 inhibitors such as quizartinib, patients with FLT3/NPM1 co-mutations face a high risk of relapse and limited durable treatment options. Ziftomenib is the only menin inhibitor to have received Breakthrough Therapy Designation by the Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory NPM1-mutated AML. “Patients with FLT3/NPM1 co-mutated AML, a significant subset of newly diagnosed cases, face high relapse rates and limited durable treatment options,” said Mollie Leoni , M.D., Chief Medical Officer of Kura Oncology . “Preclinical data demonstrate that ziftomenib synergizes with FLT3 inhibitors such as quizartinib, potentially enhancing activity without increasing toxicity. The KOMET-007 trial, alongside our recently launched KOMET-017 registrational trial combining ziftomenib with intensive and non-intensive chemotherapy, reflects our commitment to integrating menin inhibition across AML treatment regimens to improve patient outcomes.” “Initiation of the FLT3 inhibitor cohort in the KOMET-007 trial marks a pivotal advancement in addressing the urgent needs of patients with FLT3/NPM1 co-mutated AML,” said Takeyoshi Yamashita , Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin. “FLT3 mutations play a critical role in AML, driving aggressive leukemia cell proliferation, leading to poor prognosis, higher relapse rates, and shorter overall survival. Kyowa Kirin is proud to collaborate with Kura Oncology to advance this innovative menin inhibitor combination, aiming to improve outcomes for AML patients throughout the continuum of care.” The trial arm will evaluate safety, tolerability and activity of intensive chemotherapy and quizartinib in combination with ziftomenib in adult patients with newly diagnosed FLT3-ITD / NPM1 co-mutated AML. Primary and secondary endpoints include complete remission (CR) and composite complete remission (CRc). More information regarding this trial arm and the KOMET-007 trial is available at www.clinicaltrials.gov (identifier: NCT05735184). Ziftomenib is currently under clinical development, and its safety and efficacy have not been established by any regulatory authority. 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 of small molecule drug candidates is designed to target cancer signaling pathways and address high-need hematologic malignancies and solid tumors. Kura is developing ziftomenib, a menin inhibitor targeting certain genetic drivers of acute myeloid leukemias, and continues to pioneer advancements in both menin inhibition and farnesyl transferase inhibition to address mechanisms of adaptive and innate resistance in the treatment of solid tumors. For additional information, please visit the Kura website at https://kuraoncology.com/ and follow us on X and LinkedIn. About Kyowa KirinKyowa 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 StatementsThis 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, among other things, statements regarding the safety, tolerability, and therapeutic potential of ziftomenib, and expectations regarding trial enrollment. 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, 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 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. Kura Contact Investors and Media: Greg Mann 858-987-4046gmann@kuraoncology.com Kyowa Kirin Contacts Investors: Ryohei Kawai ir@kyowakirin.com Media, Global: Nobuyuki Manita media@kyowakirin.com Source: Kura Oncology, Inc.

点击蓝字 关注我们 2025年9月10日至14日，第28届中国临床肿瘤学会（CSCO）学术年会将在山东济南隆重召开。本届年会以“规范诊疗，创新引领“为主题，汇聚全球肿瘤领域知名专家学者，分享前沿研究成果与临床经验，促进学术交流与合作。血液肿瘤学领域将汇聚国内外顶尖专家，围绕淋巴瘤、骨髓瘤、白血病等核心疾病，呈现多场覆盖创新药物研发、诊疗规范优化、细胞治疗进展及多学科协作的学术盛宴。会议期间，《肿瘤瞭望-血液时讯》特邀哈尔滨血液病肿瘤研究所马军教授深入解析FLT3突变急性髓系白血病治疗的前沿脉络，助力广大同道把握最新学术动态。 Q1 您于本次CSCO大会上分享了《血液淋巴系统疾病先进治疗药物现状及展望》的精彩讲座。能否请您分享AML领域的一些先进治疗药物进展和发展趋势？ 马军教授 急性髓系白血病（AML）作为老年人群高发的血液系统疾病，60%～70%的病例发生于60岁以上。目前临床中，儿童AML患者的临床治愈率已达80%～90%，60岁以下的青中年患者低危人群临床治愈率可超过60%，中高危人群经异基因造血干细胞移植亦能获得50%以上的临床治愈率。 然而，对于老年AML患者，特别是65岁以上的高龄患者，其治疗领域仍荆棘丛生，面临着巨大的挑战与诸多尚未满足的临床需求，包括新药研发进程相对迟缓、部分药物价格高昂导致可及性较低等。为推动老年AML治疗领域取得实质性进步，仍需深入开展临床试验，并借助更多真实世界研究来积累证据、优化方案。 近年来，AML的新型治疗手段如雨后春笋般不断涌现，为患者带来了新的希望。在靶向治疗领域，FLT3抑制剂、IDH抑制剂、Menin抑制剂、XPO1抑制剂、PLK4抑制剂等新型药物持续拓展；针对白血病细胞表面抗原的疗法也日益丰富，包括单克隆抗体、双特异性抗体、CAR - T/CAR - NK细胞疗法等；此外，抗CD33等抗体药物偶联物（ADC）等也在AML治疗中展现出潜在价值与应用前景。 尽管如此，与淋巴瘤领域50余种治疗药物、骨髓瘤领域30余种治疗药物相比，AML治疗药物的数量总体上仍较为有限，研发进程需进一步加快，特别是针对老年和unfit患者，探索新的治疗策略以延长生存期、争取实现临床治愈，已成为当前研究的重点方向。 在AML的联合治疗方面，针对FLT3突变型AML，去甲基化药物（HMA）+维奈克拉+FLT3抑制剂的三联治疗方案正逐渐成为新的治疗趋势，尤其适用于unfit患者。其中，我国即将上市的第二代FLT3抑制剂奎扎替尼，相较于第一代药物显示出更优疗效，可显著延长患者的中位总生存期（OS），且安全性良好。同样，在IDH突变型AML中，类似三联治疗方案也取得了显著的疗效改善。但需注意，在实际临床应用中，应进一步结合患者的个体情况进行综合分析，以实现个体化精准治疗。 此外，鉴于AML治疗难度较大，维持治疗在疾病整体管理过程中占据着举足轻重的地位。未来，维持治疗方案有望逐步走向规范化。例如，在造血干细胞移植后，针对FLT3突变型患者应用FLT3抑制剂进行维持治疗，可实现有效降低疾病复发风险，延长患者的生存期等核心治疗目标。 Q2 携带FLT3突变的AML患者通常面临欠佳的生存和治疗挑战。在FLT3突变AML领域中，近年来发生了哪些重要的治疗变化和进展？您如何看待奎扎替尼等FLT3抑制剂在目前的治疗格局中的地位和应用前景？ 马军教授 FLT3抑制剂是FLT3突变AML治疗的一大重要进展，包括从一代到奎扎替尼等二代抑制剂。在复发/难治性AML患者中，FLT3抑制剂单药治疗缓解率优于传统化疗。在一线治疗中，研究显示奎扎替尼联合“3+7”诱导方案及巩固化疗后维持治疗可显著提升疗效。 在2025年美国国家综合癌症网络（NCCN）指南中，已将奎扎替尼和米哚妥林联合标准7+3化疗纳入一线治疗FLT3突变阳性AML的推荐。目前，国内尚无FLT3抑制剂获批用于一线治疗。但随着相关临床研究及临床实践中的证据日益丰硕，将其纳入我国一线治疗方案指日可待。 奎扎替尼是第二代FLT3抑制剂，药代动力学显示与其他FLT3抑制剂相比，其对FLT3-ITD的抑制具有最低的IC50，因此更具特异性。QuANTUM-First研究表明，相对于单纯化疗，奎扎替尼联合强化疗（包括诱导期、巩固期以及单药维持治疗）在FLT3-ITD AML患者中具有显著优势——中位OS倍增至32个月（vs. 15个月）。 基于该项研究取得的积极成果，奎扎替尼于2023年获美国FDA批准治疗带有FLT3-ITD突变阳性的新确诊AML成人患者，用于诱导、巩固和维持治疗。目前，奎扎替尼已经在中国申报上市，适应证为初诊的FLT3-ITD阳性AML。未来，随着其获批及临床应用的逐步普及，将切实落地“一切创新为了患者”的核心理念，有望实现FLT3突变AML患者预后的进一步改善。 Q3 当今靶向治疗时代下，欧洲白血病网络（ELN）指南将没有其他高危遗传学的FLT3-ITD突变归类为中危，极大地简化了治疗决策，然而其临床管理仍存一定争议。请问您如何考量FLT3-ITD突变AML患者的一线及维持治疗策略？此外，FLT3野生型AML有哪些值得期待的治疗进展？ 马军教授 对于FLT3-ITD突变AML患者，一线治疗应根据患者年龄、体能状态（ECOG评分）、合并症等多方面因素进行综合考量，进而根据个体具体情况，在强化治疗方案与低强度治疗方案中做出恰当选择。 在2025年NCCN指南中，强调了将FLT3抑制剂整合进标准治疗的重要性，建议适合强化化疗的患者的标准方案为“7+3”诱导化疗联合FLT3抑制剂；而对于不适合强化化疗的unfit患者，维奈克拉联合阿扎胞苷是目前的基础方案，并推荐添加FLT3抑制剂形成三联方案，以改善FLT3-ITD突变治疗响应欠佳的情况。 在维持治疗方面，MORPHO、RATIFY等研究显示，移植采用酪氨酸激酶抑制剂（TKI）进行维持治疗可显著改善患者的预后结局。我国新版CSCO指南中则建议，FLT3-ITD阳性患者在异基因造血干细胞移植后，可以选择FLT3抑制剂进行维持治疗；经过诱导和巩固治疗后，中高危组患者可用去甲基化药物进行维持治疗，直至疾病进展（I级推荐）。 需要特别指出的是，老年患者或unfit患者在AML患者群体中占比超过半数，因此，针对这一群体的治疗优化成为重点方向，其中微小残留病灶（MRD）监测以及平衡治疗效果与药物毒性是关键所在。目前，在该领域中，阿扎胞苷、维奈克拉与奎扎替尼联合的三联疗法，已展现出积极的临床应用前景与价值。 此外，FLT3野生型（FLT3-WT）AML患者群体的治疗策略进展同样不容忽视。QUIWI研究结果显示，在随访时间39.4个月时，FLT3-WT AML患者采用奎扎替尼联合化疗方案后，中位无事件生存期（EFS）显著延长（18.8个月 vs. 9.9个月；P=0.045），OS也获得显著改善（未达到 vs. 29.3个月；P=0.009）。在此基础上，全球多中心、III期、双盲、随机、安慰剂对照的QuANTUM-Wild研究（NCT06578247）进一步开展，旨在评估奎扎替尼联合标准诱导/巩固化疗及维持治疗在新诊断FLT3-ITD阴性（FLT3-ITD neg）AML患者中的疗效与安全性。该研究目前正处于全球患者入组阶段，其结果有望为奎扎替尼成为该类患者新的标准治疗提供关键证据。 马军 教授 哈尔滨血液病肿瘤研究所 主任医师，教授，博士生导师 哈尔滨血液病肿瘤研究所所长 中国临床肿瘤学会（CSCO）监事会监事长 亚洲临床肿瘤学会副主任委员 国家卫生健康委能力建设与继续教育中心淋巴瘤专科建设项目专家组组长 白血病·淋巴瘤杂志 总编辑 原中国临床肿瘤学会（CSCO）主任委员 原中华医学会血液学分会副主任委员 （来源：《肿瘤瞭望–血液时讯》编辑部） 声 明 凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。