Phase 3 Randomized, Double-blind, Placebo-controlled Studies Assessing Ziftomenib in Combination With Either Standard of Care Nonintensive (Venetoclax+Azacitidine) or Intensive (7+3) Therapy in Patients With Untreated NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia

Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with eligible genetic alterations. Ziftomenib is a type of therapy known to target the menin pathway in cancer cells.
This protocol has 2 separate studies that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) AML treatments in patients with certain genetic mutations who have not received any treatment for their AML. In the first study, the Nonintensive Therapy Study, older patients or those with serious medical problems will receive the SOC therapies venetoclax (ven) and azacitidine (aza), plus either ziftomenib or a placebo. In the second study, the Intensive Therapy Study, medically fit patients will receive (a) the SOC therapies cytarabine and daunorubicin, plus either ziftomenib or a placebo during a first treatment phase called induction, (b) cytarabine plus either ziftomenib or a placebo during a second treatment phase called consolidation, and (c) ziftomenib or a placebo during a third treatment phase called maintenance.
The physician will determine which study is the appropriate treatment for the patient, but neither the patient nor their physician will know whether the patient has been assigned to receive ziftomenib or a placebo. This design is called "double-blinded".

开始日期2025-09-01

申办/合作机构

Kura Oncology, Inc.

NCT06769490

/ Not yet recruiting临床1期

Phase 1 Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia

The goal of this all-oral combination is to deliver safe and effective therapy for the largest portion of AML subtypes (NPM1mt, KMT2Ar, NUP98r
40-45%).

开始日期2025-06-30

申办/合作机构-

NCT06930352

/ Not yet recruiting临床2期IIT

Frontline Ziftomenib in NPM1-Mutated or KMT2A-Rearranged Acute Myeloid Leukemia in Patients Not Eligible for Intensive Induction or Other Therapy

This phase II trial tests how well ziftomenib works in treating patients with NPM1 mutated or KMT2A rearranged acute myeloid leukemia (AML) and are not eligible to receive standard therapy. AML is often due to genetic changes in the cancer cells, including mutations in the NPM1 gene and rearrangements involving the KMT2A gene. These mutations result in activation of the menin pathway. Menin is a type of protein in the body that helps to regulate some of the naturally occurring processes in the body, but can also be involved in some types of cancers. Ziftomenib blocks this menin pathway and may prevent the cancer cells from continuing to grow. Giving ziftomenib may kill more cancer cells in patients with NPM1 mutated or KMT2A rearranged AML that are not eligible to receive standard therapy.

开始日期2025-06-01

申办/合作机构

Ohio State University Comprehensive Cancer Center

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100 项与 Ziftomenib 相关的临床结果

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100 项与 Ziftomenib 相关的转化医学

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100 项与 Ziftomenib 相关的专利（医药）

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项与 Ziftomenib 相关的文献（医药）

2025-09-01·CRITICAL REVIEWS IN ONCOLOGY HEMATOLOGY

Menin inhibitors in KMT2A-rearranged and NPM1-mutated acute leukemia: A scoping review of safety and efficacy

Review

作者: Kalfah, Anas ; Dali, Sara Al ; Mohamed, Shehab F ; Al-Mashdali, Abdulrahman F

BACKGROUND:

Menin inhibitors represent a novel therapeutic approach targeting the Menin-KMT2A interaction in acute leukemias with KMT2A rearrangements or NPM1 mutations. This scoping review synthesizes current clinical evidence for emerging Menin inhibitors in development.

METHODS:

We systematically analyzed clinical trials, conference proceedings, and regulatory documents regarding Menin inhibitors in clinical development through December 2024. Primary outcomes included response rates, minimal residual disease (MRD) status, and safety profiles.

RESULTS:

Thirteen clinical trials investigating six Menin inhibitors (Revumenib, Ziftomenib, Bleximenib, BMF-219, DS-1594, and Enzomenib) were identified. Revumenib demonstrated consistent efficacy across five pivotal trials, achieving MRD-negative rates of 70-90 % in both KMT2A-rearranged and NPM1-mutated leukemias, leading to FDA approval in November 2024. Ziftomenib showed particular efficacy in NPM1-mutated cases but exhibited a higher incidence of differentiation syndrome (30 %) in KMT2A-rearranged patients. Bleximenib reported a 93 % overall response rate, pending MRD validation. Early resistance emergence, primarily through MEN1 mutations, was observed across trials, emerging as early as two treatment cycles.

CONCLUSIONS:

Menin inhibitors demonstrate promising clinical activity in molecularly defined leukemias, with Revumenib establishing proof-of-concept for this therapeutic approach. However, challenges remain, including resistance development, optimal timing of therapy initiation, and determination of effective combination strategies. Larger randomized trials with extended follow-up are needed to establish long-term efficacy and safety profiles. The rapid clinical development of multiple agents in this class suggests an expanding role for Menin inhibitors in leukemia treatment paradigms.

2025-02-01·CTS-Clinical and Translational Science

Pharmacokinetics and ADME Characterization After Oral and Intravenous Administration of [¹⁴C]‐Ziftomenib in Healthy Male Participants

Article

作者: Mitra, Amitava ; El‐Shahat, Taha ; Leoni, Mollie ; Dale, Stephen ; Tabachri, Marilyn ; Ahsan, Julie Mackey

ABSTRACT:

Ziftomenib, a potent, selective inhibitor that binds menin at the lysine methyltransferase 2 interaction site, has demonstrated promising clinical activity with manageable toxicity in heavily pretreated patients with acute myeloid leukemia (AML) and nucleophosmin 1 mutations. This phase 1, open‐label study characterized the absorption, metabolism, excretion, and bioavailability of ziftomenib in healthy men and comprised two parts. In part A, a single oral dose of ziftomenib 400 mg (containing 250 μCi [14C]‐ziftomenib) was given to evaluate routes and rates of elimination, total radioactivity, and other pharmacokinetic parameters. In part B, a single oral dose of ziftomenib 400 mg followed by an intravenous dose of ziftomenib < 100 μg (containing 1 μCi [14C]‐ziftomenib) was administered to evaluate absolute bioavailability (both n = 8 patients). A median tmax of 3.5 h and an elimination t1/2 of 61.5 h demonstrated rapid ziftomenib absorption and enabled once‐daily dosing. Total radioactivity recovery was 89.7% in feces and 0.5% in urine over 480 h. Absolute bioavailability of 12.9% was observed. Ziftomenib was primarily metabolized by oxidation, N‐demethylation, and N‐dealkylation, with 19 metabolites recovered in plasma. All metabolites were considered minor (< 10% of total drug‐related exposure). Ziftomenib was the most abundant plasma component (> 10% of total drug‐related exposure). In conclusion, ziftomenib underwent limited metabolism following absorption and was primarily excreted as unchanged parent drug in feces. Ziftomenib was well tolerated with no new safety concerns in healthy men. Considering the pharmacokinetic profile and manageable safety outcomes, these findings support further clinical investigation of ziftomenib as treatment for AML.

2024-10-01·LANCET ONCOLOGY

Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial

Article

作者: Nie, Kun ; Patnaik, Mrinal M ; Pettit, Kristen ; Peterlin, Pierre ; Issa, Ghayas C ; Savona, Michael R ; Erba, Harry P ; Salamero, Olga ; Baer, Maria R ; Soifer, Harris S ; Shah, Mithun Vinod ; Walter, Roland B ; Kremyanskaya, Marina ; Rodríguez-Arbolí, Eduardo ; Berthon, Celine ; Adès, Lionel ; Grembecka, Jolanta ; Tabachri, Marilyn ; DeBotton, Stephane ; Leoni, Mollie ; Linhares, Brian M ; Cierpicki, Tomasz ; Altman, Jessica K ; Ray, Joshua ; Wang, Eunice S ; Mitra, Amitava ; Montesinos, Pau ; Foran, James M ; Heiblig, Mael ; Corum, Daniel ; Clegg, Bradley ; Fathi, Amir T ; Schiller, Gary ; Dale, Stephen ; Ahsan, Julie Mackey ; Papayannidis, Cristina

BACKGROUND:

Ziftomenib (KO-539) is an oral selective menin inhibitor with known preclinical activity in menin-dependent acute myeloid leukaemia models. The primary objective of this study was to determine the recommended phase 2 dose in patients with relapsed or refractory acute myeloid leukaemia based on safety, pharmacokinetics, pharmacodynamics, and preliminary activity.

METHODS:

KOMET-001 is a multicentre, open-label, multi-cohort, phase 1/2 clinical trial of ziftomenib in adults with relapsed or refractory acute myeloid leukaemia. Results of the phase 1 study, conducted at 22 hospitals in France, Italy, Spain, and the USA, are presented here and comprise the dose-escalation (phase 1a) and dose-validation and expansion (phase 1b) phases. Eligible patients were aged 18 years or older, had relapsed or refractory acute myeloid leukaemia, and had an Eastern Cooperative Oncology Group performance status of 2 or less. For phase 1a, patients (all molecular subtypes) received ziftomenib (50-1000 mg) orally once daily in 28-day cycles. For phase 1b, patients with NPM1 mutations or with KMT2A rearrangements were randomly assigned (1:1) using third-party interactive response technology to two parallel dose cohorts (200 mg and 600 mg ziftomenib). Primary endpoints were maximum tolerated dose or recommended phase 2 dose in phase 1a, and safety, remission rates, and pharmacokinetics supporting recommended phase 2 dose determination in phase 1b. Analyses were performed in all patients who received at least one dose of ziftomenib (modified intention-to-treat population). Phase 1a/1b is complete. This trial is registered with ClinicalTrials.gov, NCT04067336, and the EU Clinical Trials register, EudraCT 2019-001545-41.

FINDINGS:

From Sept 12, 2019, to Aug 19, 2022, 83 patients received 50-1000 mg ziftomenib (39 [47%] were male and 44 [53%] were female). Median follow-up was 22·3 months (IQR 15·4-30·2). Of 83 patients, the most common grade 3 or worse treatment-emergent adverse events were anaemia (20 [24%]), febrile neutropenia (18 [22%]), pneumonia (16 [19%]), differentiation syndrome (12 [15%]), thrombocytopenia (11 [13%]), and sepsis (ten [12%]). Overall, 68 of 83 patients had serious adverse events, with two reported treatment-related deaths (one differentiation syndrome and one cardiac arrest). Differentiation syndrome rate and severity influenced the decision to halt enrolment of patients with KMT2A rearrangements. In Phase 1b, no responses were reported in patients treated at the 200 mg dose level. At the recommended phase 2 dose of 600 mg, nine (25%) of 36 patients with KMT2A rearrangement or NPM1 mutation had complete remission or complete remission with partial haematologic recovery. Seven (35%) of 20 patients with NPM1 mutation treated at the recommended phase 2 dose had a complete remission.

INTERPRETATION:

Ziftomenib showed promising clinical activity with manageable toxicity in heavily pretreated patients with relapsed or refractory acute myeloid leukaemia. Phase 2 assessment of ziftomenib combination therapy in the upfront and relapsed or refractory setting is ongoing.

FUNDING:

Kura Oncology.

181

项与 Ziftomenib 相关的新闻（医药）

2025-06-12

·癌度

《癌度早报2025年6月12日》

癌度临床试验病友交流群（点击）一、新药快讯1、2025年最值得关注的三大癌症的前沿疗法2025年美国临床肿瘤学年会上，有三款创新疗法表现卓越，成为肿瘤学年会的关注焦点。第一款是名为Lifileucel的细胞疗法，这是一种肿瘤浸润淋巴细胞疗法，一次性治疗让五分之一的人活过5年。第二款药是DLL3的塔拉妥单抗，该药治疗小细胞肺癌的客观缓解率达到40%，中位总生存时间达到了14.3个月，第三款是代号为KO-539的口服药，英文名称Ziftomenib，该药对于NPM1突变的急性髓性白血病疗效数据很好，完全缓解率达到了35%，缓解持续时间较长。这三种疗法代表了肿瘤个体化治疗的方向。2、国产小分子癌症新药匹米替尼申报上市2025年6月10日，中国国家药品监督管理局官网显示，国产CSF-1R抑制剂匹米替尼申报上市，用于需要系统性治疗的腱鞘巨细胞瘤患者，腱鞘巨细胞瘤生长在关节内部和周围，主要是年轻人和中年人患病，该药基于MANEUVER研究的最新数据申报上市，给药方式是每日一次口服。3、卵巢癌新药PHST001获得快速通道资格，有望打破免疫治疗困局美国食品药品监督管理局FDA近日授予新药PHST001快速通道资格，第一个适应症是晚期铂类耐药性卵巢癌患者，另外一个适应症是该药联合化疗用于铂类敏感性卵巢癌患者。该药是一种抗CD24的单抗药物，可以增强先天免疫识别能力，在实体肿瘤患者发挥抗肿瘤活性，目前该药处于临床试验阶段。二、抗癌前沿1、TACE联合PD-1和仑伐替尼治疗肝内胆管癌获得突破肝内胆管癌是第二大原发性肝癌，最近一项研究表明，经动脉化疗栓塞TACE，联合PD-1抑制剂与仑伐替尼，这种三联疗法在不可切除肝内胆管癌产生积极疗效，中位生存时间达到了25个月，疾病控制率高达96.6%，中位无进展生存时间达到了9.5个月，有5.1%的患者达到了完全缓解，50.8%的患者达到了部分缓解。这一三联疗法的总体安全性可控，耐受性良好。

快速通道免疫疗法临床研究

2025-06-12

·ir.kuraoncology.com

Kura Oncology and Kyowa Kirin Report Positive Updated Combination Data for Ziftomenib in Newly Diagnosed AML at 2025 European Hematology Association Congress

– Encouraging clinical activity with deep responses demonstrated in the KOMET-007 trial with the combination of 600 mg ziftomenib with 7+3 in newly diagnosed patients with NPM1-m and KMT2A-r AML – – 93% (41/44) and 89% (24/27) CRc observed in NPM1-m and KMT2A-r AML response-evaluable patients, respectively – – 71% (24/34) and 88% (14/16) CR measurable residual disease (MRD)-negativity observed among responding NPM1-m and KMT2A-r AML patients, respectively – – 96% (47/49) of NPM1-m and 88% (29/33) KMT2A-r AML patients remain alive and continue on-study – – Combination was well tolerated with no additive myelosuppression – – KOMET-017-IC (intensive chemotherapy) and NIC (non-intensive chemotherapy) randomized phase 3 studies (NCT07007312) expected to start in 2H 2025 – – Kura Oncology to host virtual investor event to discuss results and broader ziftomenib development plan on June 18, 2025 at 4:30pm ET / 1:30pm PT – SAN DIEGO and TOKYO , June 12, 2025 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA, “Kura”) and Kyowa Kirin Co., Ltd. (TSE: 4151, “Kyowa Kirin”) today provided positive updated clinical data from KOMET-007, a Phase 1a/1b trial of ziftomenib, a highly selective oral investigational menin inhibitor, in combination with standards of care in patients with newly diagnosed NPM1-mutant (NPM1-m) and KMT2A-rearranged (KMT2A-r) acute myeloid leukemia (AML). The data for the combination with cytarabine/daunorubicin (7+3) were presented as an oral presentation at the European Hematology Association 2025 Congress (EHA2025) being held in Milan, Italy from June 12-15, 2025 . “The findings presented at EHA2025 underscore the potential of ziftomenib in combination with 7+3 as an early intervention in the frontline setting of AML and could offer a meaningful opportunity to improve patient outcomes,” said Harry Erba , M.D., Ph.D., Director of the Leukemia Program at the Duke Cancer Institute . “The high rates of complete remission and MRD negativity across the 7+3 cohorts are particularly encouraging. The continued rapid enrollment in the Phase 1b portion of this study underscores the urgency and enthusiasm for further evaluating this combination approach.” “We remain very encouraged by the updated clinical activity, safety and tolerability data from the KOMET-007 study evaluating ziftomenib with 7+3 in newly diagnosed AML patients with NPM1 mutations or KMT2A rearrangements,” said Mollie Leoni , M.D., Chief Medical Officer of Kura Oncology . “These updated data reinforce the combination potential of ziftomenib in the frontline setting, strengthening our confidence in its ability to provide a valuable treatment option for a significant portion of the AML population. We and our partners at Kyowa Kirin are working in earnest to prepare for the KOMET-017-IC and NIC pivotal Phase 3 studies, which will enable us to test ziftomenib-based combinations and their potential, if approved, to transform care for AML patients worldwide.” In the ongoing study, ziftomenib dosed once daily at 600 mg in combination with 7+3 continued to demonstrate robust and evolving clinical activity in patients with newly diagnosed AML. Among 71 response-evaluable patients, 92% (65/71) achieved a composite complete remission (CRc) (93% for NPM1-m, 89% for KMT2A-r patients) and 80% (57/71) achieved a complete remission (CR) (84% for NPM1-m, 74% for KMT2A-r patients) at the time of data cutoff. A rate of CR minimal residual disease (CR-MRD) negativity of 71% for NPM1-m with a median time to MRD negativity of 4.7 weeks and a rate of CR-MRD negativity of 88% for KMT2A-r patients with a median time to MRD negativity of 4.4 weeks were observed. Ziftomenib did not delay time to neutrophil and platelet count recovery, which was comparable to intensive chemotherapy regimens. Median follow-up times for the two populations were 24.9 weeks (range 4.3-47.1) in NPM1-m patients and 15.7 weeks (range 1.1-40.3) in KMT2A-r patients. Among response-evaluable NPM1-m patients, neither a median duration of CR nor a median overall survival (OS) had been reached. Among response-evaluable KMT2A-r patients, a median duration of CR was determined to be 25.6 weeks (95% CI, range 8.3-NE), and a median OS had not been reached. Notably, 96% (47/49) of NPM1-m patients and 88% (29/33) of KMT2A-r patients remained alive and on study. The safety population included 82 newly diagnosed adult patients with NPM1-m or KMT2A-r AML from the pooled Phase 1a/1b portions of the trial at the 600 mg QD dose of ziftomenib. The safety profile observed with ziftomenib was consistent with previously reported data. Ziftomenib-related adverse events (TRAEs) of ≥ Grade 3 (Gr3), which occurred in more than 10% of patients were febrile neutropenia (15%), decreased platelet count (15%), anemia (11%) and decreased neutrophil count (11%). One case of differentiation syndrome (KMT2A-r, Gr3) was successfully managed by protocol-specified mitigation strategies. Two cases of investigator-assessed QTc prolongation (both KMT2A-r, Gr3) were reported; both patients were on other medications (posaconazole and/or piperacillin/tazobactam), which have been identified as potentially causing QT prolongation at the time of QT assessment. No dose-limiting toxicities, drug-drug interactions, clinically meaningful ziftomenib-associated QTc prolongation or additive myelosuppression were observed. “Despite the availability of approved therapies for AML, up to 70% of patients who initially achieve a complete response relapse within three years – highlighting a substantial unmet need,” said Takeyoshi Yamashita , Ph.D., Executive Vice President and Chief Medical Officer of Kyowa Kirin . “The data presented at EHA2025 suggest a favorable safety, tolerability, and efficacy profile for ziftomenib. We are encouraged by its potential as a future first-line treatment option and are committed to advancing the KOMET-017 Phase 3 trial, expected to begin later this year, to further evaluate its value in AML care.” The EHA2025 oral presentation highlighting ziftomenib combined with 7+3 in newly diagnosed (1L) NPM1-m and KMT2A-r AML, and an encore presentation of results from the KOMET-001 registrational trial of ziftomenib in relapsed/refractory (R/R) NPM1-m AML (also presented during EHA2025) are available in the Posters and Presentations section of the Kura website. The KOMET-017 protocol consists of 2 separate Phase 3 studies, which will investigate the benefits and risks of adding ziftomenib to standards of care treatments in patients newly diagnosed NPM1-m or KMT2A-r AML and which is registered at www.clinicaltrials.gov as NCT07007312. Virtual Investor Event Kura will host a virtual investor event featuring company management and investigators from the KOMET-007 trial of ziftomenib in combination with 7+3 in patients with NPM1-m and KMT2A-r AML at 4:30pm ET / 1:30pm PT on Wednesday, June 18, 2025. Those who would like to participate may access the live webcast here, or register in advance for the teleconference here. The event can also be accessed on the Investors section of Kura’s website at www.kuraoncology.com. An archived replay will be available shortly after the conclusion of the live event. 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 (BTD) from the U.S. Food and Drug Administration (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 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 the FDA’s acceptance of a New Drug Application for ziftomenib for the treatment of adult patients with R/R NPM1-m AML and assignment of a Prescription Drug User Fee Act target action date of November 30, 2025. 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. Ziftomenib is also being evaluated in a Phase 1 dose-escalation trial (KOMET-015) in combination with imatinib for treatment of patients with advanced GIST. 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 the Kura 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; potential benefits of combining ziftomenib with intensive chemotherapy and other standards of care for AML; progress of the ziftomenib program and clinical trials, including the KOMET-017-IC pivotal Phase 3 study; and the potential for ziftomenib to obtain regulatory approval. 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. 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.

临床3期临床结果临床1期临床2期突破性疗法

2025-06-03

·药明康德

疗效长达8小时！FDA受理老花眼眼药水上市申请；“癌症之王”总生存率达65%！癌症疫苗最新进展公布……

疗效长达8小时！FDA受理老花眼眼药水上市申请Tenpoint Therapeutics今日宣布，美国FDA已受理其为Brimochol PF滴眼液所提交的新药申请（NDA），用于治疗老花眼。该申请的PDUFA日期为2026年1月28日。该NDA的提交得到了关键性3期临床试验BRIO-I和BRIO-II研究中积极数据的支持。之前公布的BRIO-II数据显示，相较于对照组，Brimochol PF在所有时间点（最长至8小时）显著改善患者的近视力（p<0.008）。Brimochol PF还在所有时间点显示出具有临床意义和统计学显著的瞳孔缩小效果。瞳孔缩小是缩瞳药物类别疗法的关键作用机制，通过形成针孔效应，矫正近视力丧失并增加景深。Brimochol PF结合了卡巴胆碱和溴莫尼定两种获批药物。缩瞳剂卡巴胆碱在缩小瞳孔的同时，可能刺激控制晶状体形状的睫状体肌肉的收缩，改变晶状体的形状，导致它的屈光特征发生变化和引起远视力下降。经常接受治疗的患者还会出现头痛、近视移位、眼睛发红等副作用。溴莫尼定是一种α2受体激动剂，它可以起到抑制睫状体肌肉收缩和减少红眼的作用，还可以提高卡巴胆碱的半衰期，让它的效果可以持续8-12个小时，这意味着早上滴一次眼药水效果可能持续一整天。疾病进展或死亡风险降低近半！罗氏组合疗法3期试验结果积极罗氏（Roche）日前公布3期IMforte研究的积极结果，该试验旨在评估PD-L1抑制剂Tecentriq（atezolizumab）联合小分子疗法Zepzelca（lurbinectedin）作为广泛期小细胞肺癌（ES-SCLC）一线诱导治疗后维持疗法的作用。分析显示，与Tecentriq单药维持治疗相比，该联合方案使疾病进展或死亡风险降低46%，死亡风险降低27%。根据新闻稿，这是在ES-SCLC一线维持治疗中，在无进展生存期（PFS）与总生存期（OS）上均显示出具有临床意义改善的首项3期研究。在IMforte研究中，患者在接受约三个月、四个周期的Tecentriq联合化疗（卡铂和依托泊苷）后被随机分配进入维持治疗阶段。分析显示，Tecentriq与Zepzelca联合治疗组患者自随机化后的中位总生存期为13.2个月，而Tecentriq单药组为10.6个月（HR=0.73；95% CI：0.57–0.95；p=0.0174）；独立评估下的中位无进展生存期分别为5.4个月与2.1个月（HR=0.54；95% CI：0.43–0.67；p<0.0001）。安全性方面未观察到新的不良信号，结果与两款药物既有安全性概况一致。“癌症之王”总生存率达65%！癌症疫苗最新进展公布日前，OSE Immunotherapeutics公布其癌症疫苗Tedopi的最新临床进展。在2期TEDOPaM研究中，Tedopi联合FOLFIRI化疗作为维持治疗在晚期或转移性胰腺导管腺癌（PDAC）患者中展现出积极疗效。该研究纳入了107名HLA-A2阳性的患者，所有患者在接受八个周期的FOLFIRINOX诱导化疗后无疾病进展，随后被随机分入接受FOLFIRI单药（A组）或Tedopi联合FOLFIRI（B组）治疗。研究主要终点为B组患者的一年总生存率。结果显示，Tedopi联合FOLFIRI治疗组12个月的总生存率为65%，达到了预设的统计学显著性目标。此外，该方案耐受性良好，未观察到新的安全性信号，且有两例患者出现完全缓解（CR），进一步支持了Tedopi作为“现货型”新表位癌症疫苗用于PDAC治疗的潜力。突破性小分子获FDA授予优先审评资格Kura Oncology与Kyowa Kirin近日宣布，美国FDA已接受其在研疗法ziftomenib用于治疗复发/难治性（R/R）NPM1突变（NPM1-m）急性髓系白血病（AML）成人患者的NDA，并授予优先审评资格。该申请的PDUFA日期为2025年11月30日。根据新闻稿，若获批准，ziftomenib将成为首个用于NPM1突变R/R AML治疗的menin抑制剂。此次NDA主要基于2期注册性KOMET-001研究的结果。该研究达到主要终点，即完全缓解与部分血液学恢复的完全缓解（CRh）总率，且达到统计学显著性。Ziftomenib在治疗过程中耐受性良好，骨髓抑制作用有限，因药物相关原因停药的比例仅为3%。Ziftomenib是一种针对menin与KMT2A/MLL蛋白复合体之间相互作用的候选药物。该疗法已获得FDA授予的孤儿药资格和突破性疗法认定，用于治疗AML。参考资料：[1] Tenpoint Therapeutics Ltd. Announces FDA Acceptance of New Drug Application for BRIMOCHOL™ PF for the Treatment of Presbyopia. Retrieved June 3, 2025 form https://cdn.prod.website-files.com/64b01a75c3f993c7c15191dd/683e898a70e25f0e10cf6159_PR%20NDA%20Acceptance%203June%20FINAL.pdf[2] Roche’s Tecentriq combined with lurbinectedin shows significant survival benefit in extensive-stage small cell lung cancer. Retrieved June 3, 2025 from https://www.roche.com/media/releases/med-cor-2025-06-03[3] OSE Immunotherapeutics Provides Clinical Updates on Neo-Epitope Based Cancer Vaccine Tedopi® in Pancreatic Cancer and Non-Small Cell Lung Cancer. Retrieved June 3, 2025 from https://www.globenewswire.com/news-release/2025/06/02/3092136/0/en/OSE-Immunotherapeutics-Provides-Clinical-Updates-on-Neo-Epitope-Based-Cancer-Vaccine-Tedopi-in-Pancreatic-Cancer-and-Non-Small-Cell-Lung-Cancer.html[4] Kura Oncology and Kyowa Kirin Announce FDA Acceptance and Priority Review of New Drug Application for Ziftomenib in Adults with Relapsed or Refractory NPM1-Mutant AML. Retrieved June 3, 2025 from https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-announce-fda-acceptance-and免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床3期临床结果疫苗免疫疗法加速审批

100 项与 Ziftomenib 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
NPM1突变急性髓系白血病	申请上市	美国	Kura Oncology, Inc.	2025-04-08
急性髓性白血病	临床3期	-	Kura Oncology, Inc.	2025-09-01
急性淋巴细胞白血病	临床2期	美国	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	比利时	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	加拿大	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	法国	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	德国	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	意大利	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	波兰	Kura Oncology, Inc.	2019-09-12
急性淋巴细胞白血病	临床2期	西班牙	Kura Oncology, Inc.	2019-09-12

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04067336 (KOMET-001, ASCO2025) 人工标引	临床1/2期	NPM1突变急性髓系白血病 NPM1 Mutation	112	Ziftomenib 600 mg QDZiftomenib 600 mg QD (Phase 1b/2)	餘艱獵積艱壓窪淵齋繭(襯觸構簾鬱鏇願醖鹽淵) = 鑰鹹壓壓製憲鏇鑰繭艱鬱積網鹹壓醖簾餘積鑰 (廠鬱夢繭築鑰艱襯觸鹽, 17 ~ 34) 更多	积极	2025-05-30
				Ziftomenib 600 mg QDZiftomenib 600 mg QD (Phase 2)	餘艱獵積艱壓窪淵齋繭(襯觸構簾鬱鏇願醖鹽淵) = 鹹壓簾淵繭蓋壓築淵願鬱積網鹹壓醖簾餘積鑰 (廠鬱夢繭築鑰艱襯觸鹽, 15 ~ 33)
NCT04067336 (KOMET-001, Company_Website) 人工标引	临床1/2期	急性髓性白血病 NPM1 Mutation	-	Ziftomenib	願夢醖鏇顧築願醖鑰鬱(鑰齋選繭鏇淵鏇繭鏇蓋) = The KOMET-001 trial achieved its primary endpoint of CR plus CR with partial hematological recovery (CRh) and the primary endpoint was statistically significant. 鏇鑰製壓夢鹹淵網選選 (憲壓獵憲夢簾鑰製鑰膚 ) 达到更多	积极	2025-02-07
KOMET-007 (GlobeNewswire) 人工标引	临床1期	NPM1突变急性髓系白血病 \| KMT2A易位的急性白血病一线 NPM1 Mutation \| KMT2A Rearrangement	54	ziftomenib +venetoclax/azacitidine	襯齋淵繭構簾膚蓋顧廠(構蓋餘膚選遞選夢餘齋) = 膚壓壓壓繭糧鏇顧構淵鑰艱遞鏇觸構壓齋遞醖 (餘簾簾築築顧簾積顧膚 ) 更多	积极	2024-12-09
				Ziftomenib+cytarabinecytarabine/daunorubicindaunorubicin	襯齋淵繭構簾膚蓋顧廠(構蓋餘膚選遞選夢餘齋) = 選廠鬱憲築願夢鹹鏇鏇鑰艱遞鏇觸構壓齋遞醖 (餘簾簾築築顧簾積顧膚 ) 更多
ASH2024	N/A	-	-	Ziftomenib 200 mg	衊繭獵觸選憲憲遞膚鑰(夢積膚繭廠齋艱築網蓋) = 鑰膚鹽襯鑰鏇顧餘淵鏇夢顧憲鑰淵鹹艱鏇蓋繭 (簾簾襯構窪顧築觸襯網 ) 更多	-	2024-12-08
				Ziftomenib 400 mg	衊繭獵觸選憲憲遞膚鑰(夢積膚繭廠齋艱築網蓋) = 鬱艱壓觸獵鑰醖遞鹽願夢顧憲鑰淵鹹艱鏇蓋繭 (簾簾襯構窪顧築觸襯網 ) 更多
ASH2024	N/A	-	-	Ziftomenib 200 mg	選夢製窪獵淵壓艱窪糧(範窪製醖製範鹽餘憲築) = 齋壓衊憲構築顧遞窪範積繭淵顧鏇顧窪觸淵鏇 (鬱願網構簾範築繭遞膚 )	-	2024-12-07
				Ziftomenib 400 mg	選夢製窪獵淵壓艱窪糧(範窪製醖製範鹽餘憲築) = 觸鹹膚蓋鏇壓憲廠齋鑰積繭淵顧鏇顧窪觸淵鏇 (鬱願網構簾範築繭遞膚 )
NCT04067336 (KOMET-001, Pubmed \| Lancet Oncol) 人工标引	临床1/2期	急性髓性白血病 NPM1 Mutation \| KMT2A Rearrangement	83	Ziftomenib 50-1000 mg	構膚衊積製鹽衊鬱遞顧(構齋選壓憲遞醖製簾構) = the most common grade 3 or worse treatment-emergent adverse events were anaemia (20 [24%]), febrile neutropenia (18 [22%]), pneumonia (16 [19%]), differentiation syndrome (12 [15%]), thrombocytopenia (11 [13%]), and sepsis (ten [12%]). 壓觸遞廠夢繭鹹網範憲 (膚遞鹹築積獵齋網壓選 ) 更多	积极	2024-10-01
NCT04067336 (KOMET-001, SOHO2023)	临床1/2期	NPM1突变急性髓系白血病 FLT3 \| IDH1/2 \| NPM1	29	Ziftomenib 600mg	築積膚齋遞襯艱衊製膚(鹹製鹹淵窪範鏇遞廠窪) = developed in 1 of 29 patients, detected at C4D28; the patient maintained stable disease through cycle 7 築網遞膚膚願積窪蓋鑰 (範壓獵廠壓築遞鏇選鑰 ) 更多	积极	2023-09-01
NCT04067336 (KOMET-001, EHA2023)	临床1/2期	谱系不明的急性白血病 \| 复发性急性髓细胞白血病 \| 混合表型急性白血病 ... 更多	-	Ziftomenib	蓋簾膚衊製鬱製鑰醖顧(網醖膚鏇鏇鏇繭鹽淵襯) = 構繭鹽膚範廠觸簾淵餘鹽憲範糧膚鏇餘鑰網壓 (壓餘窪積範鏇願膚醖淵 ) 更多	-	2023-06-08
NCT04067336 (KOMET-001, ASH 12022 \| ASH 22022) 人工标引	临床1/2期	复发性急性髓细胞白血病	-	Ziftomenib 200 mg	糧窪觸築繭鬱鏇製鑰簾(獵積簾衊膚艱鹹齋糧憲) = 鹹構簾構築簾製夢襯糧壓壓顧廠餘遞鹹鏇獵簾 (築窪觸鹹範蓋膚憲選鑰, 0 ~ 26.5) 更多	积极	2022-11-15
				Ziftomenib 600 mg	糧窪觸築繭鬱鏇製鑰簾(獵積簾衊膚艱鹹齋糧憲) = 壓願夢膚衊鹹衊選鹹廠壓壓顧廠餘遞鹹鏇獵簾 (築窪觸鹹範蓋膚憲選鑰, 5.5 ~ 57.2) 更多