A Randomized Phase II Trial of ASTX727 and Venetoclax Compared With ASTX727, Venetoclax, and Enasidenib for Newly Diagnosed Older Adults With IDH2 Mutant Acute Myeloid Leukemia: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial studies how well ASTX727 and venetoclax plus enasidenib works compared to ASTX727 and venetoclax alone for the treatment of older patients with newly diagnosed acute myeloid leukemia (AML) or younger patients who are considered unfit for standard treatment, and who have an abnormal change (mutation) in the IDH2 gene. This gene mutation can cause AML to grow and spread. This trial is being done to see if adding enasidenib to the usual treatment can help more patients with the IDH2 gene get rid of AML. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Enasidenib works by stopping the growth and spread of tumor cells that have the IDH2 mutation. Giving ASTX727 and venetoclax plus enasidenib may work better in treating AML patients with the IDH2 mutation.

开始日期2025-12-27

申办/合作机构

National Cancer Institute

NCT06917911

/ Not yet recruiting临床2期IIT

Phase II Study of Cytarabine + Daunorubicin (7 + 3) + Gemtuzumab Ozogamicin vs. Cytarabine + Daunorubicin (7 + 3) + Venetoclax for the Treatment of Newly Diagnosed Core Binding Factor Acute Myeloid Leukemia (CBF-AML) in Younger Adults: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial compares the effect of venetoclax to gemtuzumab ozogamicin, when given with cytarabine and daunorubicin ("7+3" regimen), for the treatment of patients with core binding factor acute myeloid leukemia (CBF-AML). Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Gemtuzumab ozogamicin is a monoclonal antibody, called gemtuzumab, linked to an antitumor antibiotic drug, called ozogamicin. Gemtuzumab is a form of targeted therapy because it attaches to specific molecules (receptors) on the surface of cancer cells, known as CD33 receptors, and delivers ozogamicin to kill them. Chemotherapy drugs, such as cytarabine and daunorubicin work in different ways to stop the growth of cancer cells either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving venetoclax with cytarabine and daunorubicin may have fewer side effects and be as effective or better than the combination with gemtuzumab ozogamicin in treating patients with core binding factor AML.

开始日期2025-11-20

申办/合作机构

National Cancer Institute

NCT06954987

/ Not yet recruiting临床2期IIT

A Two Cohort, Randomized, Double-Blind, Placebo-Controlled, Phase II Multi-Center Signal-Finding Study of Venetoclax Combined With Reduced-Intensity Conditioning Followed by Allogeneic Hematopoietic Cell Transplantation Then Venetoclax Maintenance in Adult Patients With Acute Myeloid Leukemia in First Complete Remission: A MyeloMATCH Sub-Study

This phase II MyeloMATCH treatment trial compares the effect of adding venetoclax or placebo to reduced intensity conditioning chemotherapy with fludarabine and busulfan or melphalan, with or without total body irradiation, followed by hematopoietic stem cell transplant and either venetoclax or placebo maintenance therapy after transplant, for the treatment of patients with acute myeloid leukemia (AML). Venetoclax is in a class of medications called B-cell lymphoma-2 (BCL-2) inhibitors. It may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Giving chemotherapy and total body irradiation before a donor stem cell transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When the healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets and may help destroy any remaining cancer cells. Adding venetoclax to conditioning therapy before, and giving it as maintenance therapy after, a hematopoietic stem cell transplant may be a more effective treatment option than the usual approach in patients with AML.

开始日期2025-09-18

申办/合作机构

National Cancer Institute

100 项与维奈克拉相关的临床结果

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100 项与维奈克拉相关的转化医学

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100 项与维奈克拉相关的专利（医药）

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5,144

项与维奈克拉相关的文献（医药）

2025-12-31·Hematology

Identification of t(X;1)(q28;q21) generating a novel GATAD2B::MTCP1 gene fusion in CMML and its persistence during progression to AML

Article

作者: Chen, Yu ; Zhu, Yi-yan ; Liu, Yi-zi ; Hou, Chun-xiao ; Zhang, Zhi-yu ; Chen, Su-ning ; Wang, Qian ; Zhang, Feng-hong

OBJECTIVE:

Hematological malignancies often involve chromosomal translocations and fusion genes that drive disease progression. While MTCP1 is well-known in T-cell prolymphocytic leukemia (T-PLL), its role in myeloid neoplasms is less understood. This report presents the first identification of the t(X;1)(q28;q21) translocation leading to the GATAD2B::MTCP1 fusion in acute myeloid leukemia (AML) transformed from chronic myelomonocytic leukemia (CMML).

METHODS:

The karyotypes were described according to the International System for Human Cytogenetic Nomenclature 2009. We performed targeted next-generation sequencing (NGS) on a panel of 172 genes commonly mutated in hematological malignancies (Supplemental Table 1), using an Illumina platform. RNA sequencing was conducted on total RNA extracted from bone marrow, also using the Illumina platform. The GATAD2B::MTCP1 fusion gene was confirmed by reverse transcription-polymerase chain reaction (RT-PCR) and Sanger sequencing, with specific primers for the fusion transcript (GATAD2B-F: CCTCTTTTTTTCGACGCC; MTCP1-R: ACTGAGCACAACACTTACGC).

RESULTS:

The GATAD2B::MTCP1 fusion results from a breakpoint on 1q21 within GATAD2B exon 1 and Xq28 within MTCP1 exon 2. The patient with the GATAD2B::MTCP1 fusion exhibited disease progression from CMML to AML. Despite achieving initial remission with venetoclax-based therapy and allo-HSCT, the patient relapsed and died.

CONCLUSIONS:

We propose that the GATAD2B::MTCP1 fusion upregulates MTCP1 expression rather than generating a fusion protein, thereby contributing to transformation and relapse in AML. Further investigations are needed to elucidate the precise role of this fusion event in myeloid malignancies.

2025-12-31·CANCER BIOLOGY & THERAPY

Targeting the IKZF1/BCL-2 axis as a novel therapeutic strategy for treating acute T-cell lymphoblastic leukemia

Article

作者: Li, Jun ; Ye, Chunmei ; Li, Hui ; Li, Juan

OBJECTIVES:

Acute T-cell lymphoblastic leukemia (T-ALL) is a severe hematologic malignancy with limited treatment options and poor long-term survival. This study explores the role of IKZF1 in regulating BCL-2 expression in T-ALL.

METHODS:

CUT&Tag and CUT&Run assays were employed to assess IKZF1 binding to the BCL-2 promoter. IKZF1 overexpression and knockdown experiments were performed in T-ALL cell lines. The effects of CX-4945 and venetoclax, alone and in combination, were evaluated in vitro and in vivo T-ALL models.

RESULTS:

CUT&Tag sequencing identified IKZF1 binding to the BCL-2 promoter, establishing it as a transcriptional repressor. Functional assays demonstrated that IKZF1 overexpression reduced BCL-2 mRNA levels and increased repressive histone marks at the BCL-2 promoter, while IKZF1 knockdown led to elevated BCL-2 expression. CX-4945, a CK2 inhibitor, could reduced BCL-2 levels in T-ALL cells. Notably, knockdown of IKZF1 partially rescued the CX-4945-induced repression of BCL-2. These results underscore the CK2-IKZF1 signaling axis as a key regulator of BCL-2 expression. In vitro, CX-4945 enhanced the cytotoxicity of venetoclax, with the combination showing significant synergistic effects and increased apoptosis in T-ALL cell lines. In vivo studies with cell line-derived xenograft (CDX) and patient-derived xenograft (PDX) models demonstrated that CX-4945 and venetoclax combined therapy provided superior therapeutic efficacy, reducing tumor burden and prolonging survival compared to single-agent treatments.

CONCLUSIONS:

IKZF1 represses BCL-2 in T-ALL, and targeting the CK2-IKZF1 axis with CX-4945 and venetoclax offers a promising therapeutic strategy, showing enhanced efficacy and potential as a novel treatment approach for T-ALL.

2025-12-01·Blood Research

Relative efficacy of systemic treatments for patients with relapsed/refractory chronic lymphocytic leukemia: a network meta-analysis according to 17p deletion/TP53 mutations

Article

作者: Lim, Joo Han ; Kim, Jinchul ; Lee, Moon Hee ; Cho, Jinhyun

Abstract:

Purpose:

This network meta-analysis aimed to evaluate the relative efficacy of systemic treatments in patients with relapsed/refractory chronic lymphocytic leukemia (R/R CLL), focusing on key genetic mutations, specifically the 17p deletion and TP53 mutations.

Methods:

We conducted a systematic literature review to identify all publicly available randomized controlled trials (RCTs) using PubMed, EMBASE, the Cochrane database, and meeting abstracts published through December 2023. A Bayesian network meta-analysis was performed to estimate the hazard ratios (HRs) for progression-free survival (PFS) with 95% confidence intervals (CIs) and to determine the ranking of the included regimens.

Results:

Twelve trials involving 4,437 patients and 13 treatment options were included in the meta-analysis. Venetoclax plus rituximab and zanubrutinib emerged as the most effective treatments for the overall R/R CLL population, showing the lowest PFS HR (HR 0.62, 95% CI 0.32–1.20 and HR 0.65, 95% CI 0.49–0.86, respectively) versus ibrutinib, and were ranked as the best agent (surface under the cumulative ranking curve [SUCRA] value of both 90%, respectively) among the included drugs. In the 17p deletion/TP53 mutation subgroup, zanubrutinib demonstrated the most favorable efficacy (HR 0.52, 95% CI 0.31–0.88 versus ibrutinib) with the highest SUCRA value (97%). In patients without these mutations, venetoclax plus rituximab was the most effective (HR 0.49, 95% CI 0.26–0.94 versus ibrutinib) with a SUCRA value of 94%.

Conclusion:

Our findings highlight the superior efficacy of venetoclax plus rituximab and zanubrutinib for treating R/R CLL and confirm that the role of each regimen may vary depending on the clinically significant mutations.

1,406

项与维奈克拉相关的新闻（医药）

16 小时之前

·GlobeNewswire-Health Care

Aptose Provides Clinical Update for the Tuspetinib-based Triple Drug Frontline Therapy in Newly Diagnosed AML Patients from the Phase 1/2 TUSCANY Trial

Aptose is developing TUS+VEN+AZA as a one-of-a-kind safe and mutation agnostic frontline triple drug therapy for newly diagnosed AML patients First two dose cohorts of TUS+VEN+AZA triplet demonstrate safety, complete remissions, and MRD negativity across patients with diverse mutations, including TP53-mutated/CK AML and FLT3-wildtype AML patients SAN DIEGO and TORONTO, May 05, 2025 (GLOBE NEWSWIRE) -- Aptose Biosciences Inc. (“Aptose” or the “Company”) (TSX: APS; OTC: APTOF), a clinical-stage precision oncology company, today reported updated and new data from Aptose’s Phase 1/2 TUSCANY trial in newly diagnosed acute myeloid leukemia (AML) patients dosed with a 40 mg or 80 mg dose of tuspetinib (TUS) in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet). The TUS+VEN+AZA triplet is being developed as a safe and mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy. Earlier this year, Aptose announced the initiation of the TUSCANY trial and dosing in newly diagnosed AML patients at an initial dose of 40 mg TUS in the first cohort of four patients. The second cohort of patients is now receiving 80 mg TUS. Data from the first two cohorts, with a 40 mg or 80 mg dose of tuspetinib in the TUS+VEN+AZA combination, reveal promising clinical safety and antileukemic activity. To date, four newly diagnosed AML patients received the initial dose of TUS (40 mg) as part of the (TUS+VEN+AZA) combination. Notably, a patient with biallelic TP53 mutations and a complex karyotype (TP53-mutated/CK) and FLT3-wildtype achieved a complete remission (CR) and the clinical site reported no measurable residual disease (MRD-negative status) in this patient.One FLT3-wildtype patient having an IDH-2 mutation achieved a CR and MRD-negative status.Another FLT3-wildtype patient achieved a CRi during Cycle 1 and MRD-negative status.The first three patients continue on treatment, while a fourth patient did not respond at this 40 mg dose level of TUS and was discontinued.The 40 mg dose of the TUS+VEN+AZA triplet remains safe, and no dose-limiting toxicities (DLTs) have been reported. To date, three newly diagnosed AML patients having diverse mutation profiles have received the 80 mg of TUS, as part of the TUS+VEN+AZA combination. The 80 mg TUS dose has been considered the optimal dose that has demonstrated safety and consistent blood exposure levels that exert potent antileukemic activity. All patients achieved blast reductions in Cycle 1 that met the criteria for complete remissions (CR or CRi) and continue on treatment. Notably, another TP53-mutated/CK and FLT3-wildtype patient achieved blast reductions that met CRi criteria in Cycle 1 and is now receiving additional therapy in Cycle 2.The second patient, having FLT3-wildtype status, achieved a CR.The third patient, having FLT3-ITD and NPM1 mutations and entering the trial with a 75% bone marrow blast count, achieved a CRi. All three patients are early in their course of treatment and are expected to show further improvements in their disease status as they are all continuing with treatment, and MRD status will be monitored as the patients move through their courses of therapy.The 80 mg dose of TUS, as part of the TUS+VEN+AZA triplet, continues to show favorable safety with no dose-limiting toxicities (DLTs) having been reported. “The treatment paradigm for AML is shifting to triplet combination therapy,” said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. “We have always maintained that tuspetinib, with its notable safety profile and ability to treat the larger, difficult-to-treat AML populations with high-risk mutations, could be an ideal drug for a triplet combination therapy in the frontline setting. With the majority of patients already achieving complete responses -- including early responses in patients with adverse mutations -- the clinical findings to date are bearing that out.” William G. Rice, Ph.D., Chairman, President and Chief Executive Officer of Aptose, will review the data at a presentation today, Monday, May 5th, 2025, 3:00 p.m. EDT, at the 2025 Bloom Burton & Co. Healthcare Investor Conference. The presentation and webcast can be accessed here and will be available on the Aptose website. TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study Tuspetinib based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated. Earlier APTIVATE trials of TUS as a single agent and in combination as TUS+VEN demonstrated favorable safety and broad activity in diverse relapsed or refractory (R/R) AML populations that went beyond the more prognostically favorable NPM1 and IDH mutant subgroups. Responses to TUS were also observed in those with prior-VEN and prior-FLT3 inhibitor (FLT3i) therapies, those with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes. The TUSCANY triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS will be administered in 28-day cycles, beginning at 40mg once daily, with dose escalations planned after a safety review of each dose level. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025. Data will be released as it becomes available. More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here). About Aptose Aptose Biosciences is a clinical-stage biotechnology company committed to developing precision medicines addressing unmet medical needs in oncology, with an initial focus on hematology. The Company’s lead clinical-stage, oral kinase inhibitor tuspetinib (TUS) has demonstrated activity as a monotherapy and in combination therapy in patients with relapsed or refractory acute myeloid leukemia (AML) and is being developed as a frontline triplet therapy in newly diagnosed AML. For more information, please visit www.aptose.com. Forward Looking Statements This press release may contain forward-looking statements within the meaning of Canadian and U.S. securities laws, including, but not limited to, statements relating to the therapeutic potential and safety profile of tuspetinib (including the triplet therapy) and its clinical development, the anticipated enrollment rate in the TUSCANY trial and the timing thereof, as well as statements relating to the Company’s plans, objectives, expectations and intentions and other statements including words such as “continue”, “expect”, “intend”, “will”, “should”, “would”, “may”, and other similar expressions. Such statements reflect our current views with respect to future events and are subject to risks and uncertainties and are necessarily based upon a number of estimates and assumptions that, while considered reasonable by us are inherently subject to significant business, economic, competitive, political and social uncertainties and contingencies. Many factors could cause our actual results, performance or achievements to be materially different from any future results, performance or achievements described in this press release. Such factors could include, among others: our ability to obtain the capital required for research and operations and to continue as a going concern; the inherent risks in early stage drug development including demonstrating efficacy; development time/cost and the regulatory approval process; the progress of our clinical trials; our ability to find and enter into agreements with potential partners; our ability to attract and retain key personnel; changing market conditions; inability of new manufacturers to produce acceptable batches of GMP in sufficient quantities; unexpected manufacturing defects; and other risks detailed from time-to-time in our ongoing quarterly filings, annual information forms, annual reports and annual filings with Canadian securities regulators and the United States Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should the assumptions set out in the section entitled "Risk Factors" in our filings with Canadian securities regulators and the United States Securities and Exchange Commission underlying those forward-looking statements prove incorrect, actual results may vary materially from those described herein. These forward-looking statements are made as of the date of this press release and we do not intend, and do not assume any obligation, to update these forward-looking statements, except as required by law. We cannot assure you that such statements will prove to be accurate as actual results and future events could differ materially from those anticipated in such statements. Investors are cautioned that forward-looking statements are not guarantees of future performance and accordingly investors are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty therein. For further information, please contact: Aptose Biosciences Inc. Susan Pietropaolo Corporate Communications & Investor Relations 201-923-2049 spietropaolo@aptose.com

临床结果

2025-05-04

·健识局

三甲医院院长被砍；明星董事长卸任；两家药企垄断遭重罚

5月1日，津药药业、江苏联环药业因涉嫌垄断地塞米松磷酸钠原料药，被天津市市场监督管理委员会行政处罚，合计被罚没超1.3亿元。此前4月23日，仙琚制药在年报中披露，涉嫌地塞米松磷酸钠原料药价格垄断，收到天津市市场监督管理委员会行政处罚告知书，罚没款预计为 1.95 亿元。值得一提的是，津药药业已经不是第一次垄断，津药药业及其下属公司天津天药此前已经因药品垄断被处罚3次，三次没收和罚款合计超亿元。更多消息，健识局整理如下：重磅政策一览1.涉嫌垄断，两家药企被罚超1.3亿元5月1日，津药药业、江苏联环药业公告：公司因对地塞米松磷酸钠原料药实施的垄断行为，收到天津市市场监督管理委员会下发的《行政处罚决定书》、《行政处罚告知书》。地塞米松磷酸钠注射剂是一款激素类抗炎药，为国家医保药品目录甲类药品，也是第九批国采采购金额最大的品种。据媒体报道，近两三年来，这款原本廉价的临床常用药品价格经历过暴涨和暴跌。公告显示，津药药业、江苏联环药业与具有竞争关系的其他经营者，就地塞米松磷酸钠原料药达成并实施“固定或者变更商品价格”垄断协议。津药药业被没收违法所得4276.44万元、并处2023年度销售额8%的罚款约1.32亿元，结合宽大情形，减免罚款额的80%，减免后实际罚款约2642.8万元。以上罚没款合计超过6919万元。江苏联环药业被没收违法所得约1790万元，并处2023年度销售额8%的罚款约6162.7万元，结合宽大情形，减免罚款额的30%，减免后实际罚款约4314万元。合计被罚没约6163万元。医药卫生事件1.吴以芳辞任复星医药董事长4 月 30 日，复星医药发布公告，收到吴以芳的书面辞职函，辞任自2025年4月29日生效。吴以芳是复星医药老将，在复星医药工作逾二十年，曾任万邦生化副总经理、总裁、董事长兼首席执行官。2004 年万邦生化加入复星医药集团，吴以芳进入复星医药，2016年至今任复星医药首席执行官。2020年，吴以芳就任复星医药董事长职务。吴以芳的职位由陈玉卿接任。陈玉卿2010年加入复星医药，从人力资源岗位起步，任总裁助理兼人力资源部总经理、联席首席执行官等职。2.三甲医院院长被砍伤4月30日，经济观察报报道，南京鼓楼医院院长于成功在4月28日被人砍伤，目前已脱离危险。一位知情人士表示，行凶者长期尾随于成功，4月28日夜间，行凶者携带凶器尾随于成功至家中，将于成功及其妻子砍成重伤。目前未发现于成功接诊过该行凶者，行凶者也并非传闻中与于成功家人有情感纠纷。南京鼓楼医院又名南京大学医学院附属鼓楼医院，是一家知名三甲医院，建于1892年，是中国最早的西医院之一，有床位4300余张，在岗职工6200余人。于成功生于1965年，1997年博士毕业后进入南京鼓楼医院工作至今，是二级教授、博士生导师，擅长炎症性肠病诊治。3.葫芦娃药业被ST4月28日，葫芦娃药业发布公告称，因2024年度财报被出具保留意见审计报告且内控存在重大缺陷，公司股票自4月30日起被实施退市风险警示，股票简称变更为“ST葫芦娃”。葫芦娃药业财务问题由来已久。去年，海南证监局出具责令改正的决定，要求葫芦娃药业重述其2023年度财报。审计机构表示，截至审计报告日，葫芦娃药业尚未提供与上述重述事项相关的完整财务资料，以及相关原始资料，因此出具保留意见审计报告。近期葫芦娃药业人事变动频繁，公司总经理张铭芮入职不到两个月，就宣布辞职。2024年财报显示，公司实现营业收入14.14亿元，同比下降21.26%；归母净利润为-2.74亿元，同比下降2629.23%。一周药械盘点1.百济神州的BCL2抑制剂申报上市4月28日，CDE官网公示，百济神州的BCL2抑制剂索托克拉片的上市申请获得受理，并纳入优先审评，适用于治疗既往接受过治疗的慢性淋巴细胞白血病（CLL）/ 小淋巴细胞淋巴瘤（SLL）成人患者。目前，全球只有艾伯维/罗氏的维奈克拉一款BCL2抑制剂获批上市，2023年全球销售额超过20亿美元。根据百济神州介绍，与维奈克拉相比，索托克拉在临床前研究和肿瘤模型中均显示出更高的效力和靶点选择性，并有可能克服耐药性。除了百济神州之外，亚盛医药的BCL2抑制剂也已经递交上市申请；诺诚健华的同类品种处于III期临床阶段。艾伯维/罗氏在研的Bcl-xl/Bcl-2抑制剂Navitoclax也已进入III期临床。2.强生FcRn单抗获批上市4月30日，强生宣布尼卡利单抗获得FDA批准上市，用于治疗自身抗体阳性的12岁及以上青少年和成人全身型重症肌无力患者。尼卡利单抗是一款靶向FcRn的IgG1型单克隆抗体。2020年8月，强生以65亿美元价格收购Momenta，获得了这款药物。此前，全球已有两款FcRn单抗获得FDA批准上市，分别是Argenx/再鼎的艾加莫德α和优时比的罗泽利昔珠单抗。3.诺华重磅核药Pluvicto在华申报新适应症4月28日，CDE官网显示，诺华的镥[177Lu]特昔维匹肽在华申报新适应症。根据临床试验及注册申报进展，推测此次申报的适应症为前列腺癌。镥[177Lu]特昔维匹肽是诺华收购Endocyte获得的一款放射配体疗法，由靶向PSMA的小分子化合物与治疗性放射性核素镥177偶联而成，2022年3月首次获得FDA批准上市。据诺华财报，2024年该药物的全球销售额已达13.92亿美元。撰稿 | 李傲编辑 | 江芸贾亭运营 | 雾纪插图 | 视觉中国三个月闭店14000家，疯狂时代终结迈瑞医疗增长神话崩塌，狂奔时代结束胰岛素市场发生重大变化，通化东宝上市以来首次亏损

高管变更带量采购

2025-05-01

·ir.kuraoncology.com

Kura Oncology Reports First Quarter 2025 Financial Results

– NDA submitted in 1Q 2025 for ziftomenib for the treatment of adult patients with relapsed or refractory AML with an NPM1 mutation – – Data from Phase 1b/2 registration-directed trial of ziftomenib selected for oral presentation at ASCO Annual Meeting – – $45.0 million milestone payment earned for NDA submission under collaboration agreement with Kyowa Kirin – – First patients dosed in Phase 1 trial of ziftomenib plus imatinib in GIST – – $703.2 million in pro forma cash, together with anticipated collaboration agreement payments, expected 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 , May 01, 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 first quarter 2025 financial results and provided a corporate update. “In the first quarter of 2025, we achieved a significant milestone with the submission of our first NDA for ziftomenib,” said Troy Wilson , Ph.D., J.D., President and Chief Executive Officer of Kura Oncology . “We are committed to working with FDA to support its review and are strategically advancing our pre-commercial activities to prepare for potential approval for the treatment of adult patients with relapsed or refractory NPM1-mutant AML. Beyond the monotherapy setting, we look forward to sharing data on the combination of ziftomenib with intensive and non-intensive standards of care, while gearing up for two Phase 3 studies in the frontline setting. With a strong pipeline, multiple clinical data readouts expected this year, and a solid financial foundation, we are well-positioned to drive progress across our programs.” Recent Highlights Submission of New Drug Application for ziftomenib to FDA – Kura and Kyowa Kirin Co., Ltd. (Kyowa Kirin) announced submission of the New Drug Application (NDA) for ziftomenib for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation to the U.S. Food and Drug Administration (FDA) on March 31, 2025 . From the time of submission, the FDA has a 60-day filing review period, and the Company expects to receive notification from the FDA on this preliminary evaluation in the second quarter of 2025. If Priority Review is granted, it would provide a target FDA review period of six months after NDA acceptance. Abstracts accepted for presentation at ASCO and EHA – In February 2025 , Kura and Kyowa Kirin announced positive topline results from the KOMET-001, the Phase 2 registration-directed trial of ziftomenib in patients with R/R NPM1-mutant (NPM1-m) AML. These data have been accepted for oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago and accepted for an encore presentation at the European Hematology Association (EHA) Congress in Milan, Italy . In addition, preliminary clinical data from the Phase 1b expansion cohort evaluating ziftomenib in combination with intensive (7+3) in the frontline setting has been accepted for an oral presentation at EHA. Abstract titles and presentation details can be found on the ASCO and EHA meeting sites at the lift of the respective embargos. Submission of the NDA for ziftomenib has triggered a $45 million milestone payment obligation from Kyowa Kirin – As a result of the NDA submission for ziftomenib, Kura has earned a $45 million milestone payment under the global strategic collaboration agreement between Kura and Kyowa Kirin to develop and commercialize ziftomenib in acute leukemias (Kyowa Agreement). Under the terms of the Kyowa Agreement, Kura received an upfront payment of $330 million in December 2024 and accounting for this $45 million milestone payment, Kura expects to receive up to $375 million in additional, near-term milestone payments. First patients dosed in KOMET-015 trial in GIST – Earlier this week, Kura announced the first patients have been dosed in its KOMET-015 Phase 1 clinical trial of ziftomenib in patients with advanced gastrointestinal stromal tumors (GIST) after imatinib failure. In October 2024 , Kura presented preclinical data 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 the treatment of GIST. 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. Preclinical data for KO-2806 presented in oral minisymposium session at AACR Annual Meeting – Last month, Kura presented preclinical data for KO-2806, the Company’s next-generation farnesyl transferase inhibitor (FTI), in combination with cabozantinib for the treatment of clear cell renal cell carcinoma at the American Association for Cancer Research (AACR) Annual Meeting in Chicago . These data add to a growing body of preclinical evidence demonstrating the potential of FTIs as companion therapeutic agents to augment the antitumor activities of and to overcome resistance to various targeted therapies. Financial Results Collaboration revenue from our Kyowa Kirin partnership for the first quarter of 2025 was $14.1 million , compared to no revenue for the first quarter of 2024. Research and development expenses for the first quarter of 2025 were $56.0 million , compared to $36.3 million for the first quarter of 2024. General and administrative expenses for the first quarter of 2025 were $22.8 million , compared to $18.2 million for the first quarter of 2024. Net loss for the first quarter of 2025 was $57.4 million , compared to a net loss of $49.5 million for the first quarter of 2024. Net loss for the first quarter of 2025 included non-cash, share-based compensation expense of $7.8 million . This compares to $8.5 million for the same period in 2024. As of March 31, 2025 , Kura had cash, cash equivalents and short-term investments of $658.2 million , compared to $727.4 million as of December 31, 2024 . As adjusted for the $45 million NDA submission milestone payment earned under our collaboration agreement with Kyowa Kirin, Kura had, on a pro forma basis, $703.2 million in cash, cash equivalents and short-term investments as of March 31, 2025 . Based on our current plans, we believe our cash, cash equivalents and short-term investments as of March 31, 2025 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 frontline combination setting. Forecasted Milestones Present data from the KOMET-001 Phase 1b/2 registration-directed trial in R/R NPM1-m AML at ASCO and EHA 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) in the frontline setting at EHA in the second quarter of 2025. Present preliminary clinical data from the KOMET-007 Phase 1b expansion cohort evaluating ziftomenib with venetoclax and azacitidine in the frontline setting at a medical meeting in the second 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. Nominate a development candidate for next-generation menin inhibitor program in diabetes in mid-2025. Initiate one or more FIT-001 expansion cohorts of KO-2806 and cabozantinib in patients with advanced renal cell carcinoma in the second half of 2025. Present data from the FIT-001 Phase 1 trial evaluating KO-2806 and cabozantinib in patients with renal cell carcinoma in the second half of 2025. Present data from the FIT-001 Phase 1 monotherapy dose escalation of KO-2806 in patients with RAS mutations in the second half of 2025. Present data from the 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, May 1, 2025 , to discuss the financial results for the first quarter of 2025 and to provide a corporate update. The live call may be accessed by dialing (800) 245-3047 for domestic callers and (203) 518-9765 for international callers and entering the conference ID: KURAQ1. A live webcast and archived replay of the event will be available here or online from the investor relations section of the Company’s 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 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 from the 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 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. KO-2806, a next-generation 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 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, KO-2806 and tipifarnib; the expected timing of clinical trials; the expected timing and presentation of results and data from clinical trials; the anticipated timing of FDA’s notification on its preliminary evaluation of the NDA for ziftomenib; the potential duration of FDA’s review of the NDA; the potential FDA approval of product candidates; the success and impact of interactions with the FDA; 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, 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. Contacts 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.

临床1期临床结果临床2期突破性疗法财报

100 项与维奈克拉相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10679	维奈克拉	L01XX52	DB11581

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
复发性套细胞淋巴瘤	日本	AbbVie LLC	2025-03-27
难治性套细胞淋巴瘤	日本	AbbVie LLC	2025-03-27
难治性慢性淋巴细胞白血病	日本	AbbVie LLC	2019-09-20
复发性慢性淋巴细胞白血病	日本	AbbVie LLC	2019-09-20
小淋巴细胞淋巴瘤	美国	AbbVie, Inc.	2018-06-08
成人急性髓性白血病	欧盟	AbbVie Deutschland GmbH & Co. KG	2016-12-04
成人急性髓性白血病	冰岛	AbbVie Deutschland GmbH & Co. KG	2016-12-04
成人急性髓性白血病	列支敦士登	AbbVie Deutschland GmbH & Co. KG	2016-12-04
成人急性髓性白血病	挪威	AbbVie Deutschland GmbH & Co. KG	2016-12-04
急性髓性白血病	加拿大	AbbVie AS	2016-10-31
慢性淋巴细胞白血病	美国	AbbVie, Inc.	2016-04-11

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
肿瘤溶解综合征	临床3期	美国	AbbVie, Inc.	2024-08-05
肿瘤溶解综合征	临床3期	澳大利亚	AbbVie, Inc.	2024-08-05
肿瘤溶解综合征	临床3期	法国	AbbVie, Inc.	2024-08-05
肿瘤溶解综合征	临床3期	希腊	AbbVie, Inc.	2024-08-05
肿瘤溶解综合征	临床3期	波多黎各	AbbVie, Inc.	2024-08-05
肿瘤溶解综合征	临床3期	塞尔维亚	AbbVie, Inc.	2024-08-05
肿瘤溶解综合征	临床3期	西班牙	AbbVie, Inc.	2024-08-05
肿瘤溶解综合征	临床3期	中国台湾	AbbVie, Inc.	2024-08-05
肿瘤溶解综合征	临床3期	英国	AbbVie, Inc.	2024-08-05
复发性急性髓细胞白血病	临床3期	美国	AbbVie, Inc.	2022-10-01

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临床结果

适应症

分期

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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


NCT04070768 (BTCRC-AML17-113, CTgov)	临床1期	急性髓性白血病 \| 复发性急性髓细胞白血病	18	Venetoclax+Gemtuzumab Ozogamicin	鑰範壓膚簾醖糧蓋製繭 = 壓襯夢鑰鹽簾選築鏇積築蓋獵選襯繭壓顧窪築 (膚艱齋積鬱範廠窪壓夢, 醖願網築選獵遞鏇窪夢 ~ 襯膚糧壓餘觸鬱鹽網範)	-	2025-05-04
NCT04487106 (CTgov)	临床2期	复发性急性髓细胞白血病 \| 复发性急性白血病 \| 慢性粒单核细胞白血病 ... 更多	21	Trametinib+Venetoclax+Azacitidine (Cohort A (Newly Diagnosed AML Patients))	鏇衊獵夢網艱廠憲築壓 = 壓範選願鹹積餘廠築繭獵遞鏇蓋選壓鏇範艱蓋 (壓鬱襯廠鹹願齋遞範蓋, 鏇選鏇範鑰壓壓鬱餘壓 ~ 鹹淵鏇鹹鏇艱範遞選艱) 更多	-	2025-04-22
				Trametinib+Venetoclax+Azacitidine (Cohort B (Relapsed/Refractory AML or Higher-risk MDS or CMML Patients))	鏇衊獵夢網艱廠憲築壓 = 願夢構鹹顧衊餘網憲遞獵遞鏇蓋選壓鏇範艱蓋 (壓鬱襯廠鹹願齋遞範蓋, 廠簾繭蓋簾襯獵構築選 ~ 製襯積壓鬱鬱憲鏇鏇獵) 更多
NCT05211336 (CTgov)	临床1期	难治性弥漫性大B细胞淋巴瘤活化B细胞型 \| 弥漫性大B细胞淋巴瘤 \| 原发性中枢神经系统淋巴瘤	14	Lenalidomide+Nivolumab+Ibrutinib+Prednisone+Venetoclax+Obinutuzumab (Lenalidomide+Nivolumab Followed by Venetoclax, Ibrutinib,Prednisone, Obinutuzumab, Lenalidomide-Nivo)	艱衊網繭淵積蓋範繭獵 = 繭網廠選遞鹽襯鹹襯選衊鏇夢範遞網鬱積願窪 (窪鬱積顧選範構膚鏇願, 鑰糧淵鹽壓鑰願顧蓋淵 ~ 醖願廠壓鏇窪觸襯積積) 更多	-	2025-04-20
				Lenalidomide+VIPOR+Ibrutinib+Prednisone+Venetoclax+Obinutuzumab (Cohort 1, Arm 2: Experimental- VIPOR (Venetoclax, Ibrutinib, Prednisone, Obinutuzumab, Lenalidomide))	艱衊網繭淵積蓋範繭獵 = 壓繭膚糧壓夢獵構製觸衊鏇夢範遞網鬱積願窪 (窪鬱積顧選範構膚鏇願, 鏇醖觸範壓鹽衊糧鑰齋 ~ 鏇選憲膚觸繭壓網廠齋) 更多
NCT04285567 (CRISTALLO, CTgov)	临床3期	慢性淋巴细胞白血病	166	VEN (Arm A: VEN+G)	艱顧艱願齋選選壓餘襯 = 鹽願遞鏇範築繭構鏇顧顧餘壓構糧憲範範選鏇 (鏇選壓憲鏇鬱廠艱糧觸, 範選範鬱鹽淵鏇艱艱鹹 ~ 窪顧窪構鏇壓選鹽顧齋) 更多	-	2025-04-17
				bendamustine+fludarabine+cyclophosphamide+rituximab (Arm B: FCR/BR)	艱顧艱願齋選選壓餘襯 = 鏇鹹構鏇襯鹹獵窪齋觸顧餘壓構糧憲範範選鏇 (鏇選壓憲鏇鬱廠艱糧觸, 構網餘鏇衊艱願鑰網鏇 ~ 築積鹽鑰膚簾鑰窪糧選) 更多
NCT02942290 (Pubmed \| Blood)	临床1期	高危骨髓增生异常综合征	107	Venetoclax 400 mg	顧觸衊製鏇繭艱範窪鬱(膚襯網積衊窪餘衊醖艱) = 築鬱構築襯繭積製窪夢繭衊觸網鏇襯淵蓋築壓 (製鹹窪衊憲蓋築齋襯膚 ) 更多	积极	2025-03-13
NCT03236857 (Pubmed \| Pediatr Blood Cancer)	临床1期	复发性儿童急性淋巴细胞白血病	-	Venetoclax monotherapy	鹹鹹廠網餘廠夢鹽膚鹹(構糧窪顧願範繭醖鏇淵) = One fatal adverse event possibly related to venetoclax occurred 壓艱膚築觸顧範鏇獵夢 (蓋繭壓壓簾鹹餘獵糧艱 ) 更多	积极	2025-03-10
NCT03214562 (Pubmed \| Leukemia)	临床1/2期	难治性急性髓细胞白血病 wild-type TP53 status	138	FLAG-IDA + Venetoclax	遞選膚簾築壓選鏇衊餘(鬱餘鏇膚鹹構遞簾膚繭) = low 襯齋淵製憲餘遞壓淵艱 (獵築蓋艱衊醖齋廠鹹齋 ) 更多	积极	2025-02-25
NCT03836261 (NEWS \| Pubmed) 人工标引	临床3期	慢性淋巴细胞白血病一线	867	Acalabrutinib-Venetoclax	衊簾獵醖夢積廠範製遞(構壓鹽餘繭糧積鹹簾衊) = 壓壓鏇齋憲餘夢夢窪淵獵願製範鹹鑰獵膚醖蓋 (鏇襯鑰願網齋壓築網憲 ) 更多	积极	2025-02-21
				Acalabrutinib-Venetoclax-Obinutuzumab	衊簾獵醖夢積廠範製遞(構壓鹽餘繭糧積鹹簾衊) = 顧獵願築憲餘願選憲鏇獵願製範鹹鑰獵膚醖蓋 (鏇襯鑰願網齋壓築網憲 ) 更多
NCT03319901 (Pubmed \| Blood Adv)	临床1期	急性淋巴细胞白血病 hypodiploid TP53-mutated	19	Venetoclax + Mini-HCVD	艱壓鑰鑰築範鏇製衊獵(淵餘膚構顧選積淵憲積) = 淵範鬱憲窪襯網構構築願築獵選製繭鏇蓋壓淵 (顧範範襯鹹衊鑰製網鏇, 71 ~ 100) 更多	积极	2025-02-11
NCT04599634 (CTgov)	临床1期	滤泡性淋巴瘤 \| 套细胞淋巴瘤 \| 慢性淋巴细胞白血病 ... 更多	11	venetoclax+magrolimab+obinutuzumab (Arm 1, Cohort 1 Follicular Lymphoma Level 1)	範鬱糧鏇獵製觸選構壓 = 鹽餘艱夢願築觸齋餘製鏇淵顧製醖製壓獵鏇選 (窪憲憲鬱齋醖網憲膚蓋, 壓艱淵壓選顧觸遞襯蓋 ~ 鑰衊選構壓廠簾蓋簾膚) 更多	-	2025-02-04
				venetoclax+magrolimab+obinutuzumab (Arm 2, Cohort 2 Marginal Zone Lymphoma, Mantle Cell Lymphoma, & Chronic Lymphocytic Leukemia Level 1)	範鬱糧鏇獵製觸選構壓 = 憲襯遞鏇鬱憲築醖簾餘鏇淵顧製醖製壓獵鏇選 (窪憲憲鬱齋醖網憲膚蓋, 蓋簾簾選繭願膚鑰鏇積 ~ 獵積齋範構襯鏇獵選醖) 更多