A Randomized Phase II Trial of ASTX727 With Standard Duration Versus Shorter Duration of Venetoclax in Genomically Heterogenous AML Among Adults Aged 60 or Older and Less Fit for Intensive Therapy: A MyeloMATCH Substudy

This phase II MyeloMATCH treatment trial compares ASTX727 with standard duration versus shorter duration of venetoclax for the treatment of newly diagnosed acute myeloid leukemia (AML). ASTX727 is a combination of decitabine and cedazuridine. 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. 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. 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. Shorter duration venetoclax may be as effective as standard duration venetoclax when given with ASTX727 for the treatment of newly diagnosed AML.

开始日期2027-04-18

申办/合作机构

National Cancer Institute

NCT06917911

/ 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2027-02-02

申办/合作机构

National Cancer Institute

NCT07738887

/ Not yet recruiting临床2期IIT

Epcoritamab Plus Venetoclax Plus Ibrutinib in Patients With Chronic Lymphocytic Leukemia With TP53 Alterations

This is a phase 2, open-label, multicenter study evaluating the efficacy and safety of a fixed-duration combination of epcoritamab, venetoclax, and ibrutinib (EVI) in previously untreated patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have TP53 alterations.
Patients with TP53 abnormalities, including TP53 mutation and/or 17p deletion, have a poorer prognosis and are less likely to benefit from conventional chemoimmunotherapy. Targeted therapies such as ibrutinib and venetoclax have improved outcomes in this population, but many patients eventually experience disease progression. This study investigates whether adding epcoritamab, a CD3×CD20 bispecific antibody, to the combination of ibrutinib and venetoclax can improve the depth and durability of treatment responses while using a fixed-duration treatment approach.
Participants will receive sequential treatment with ibrutinib alone, followed by ibrutinib plus venetoclax, and then the triple combination of epcoritamab, venetoclax, and ibrutinib. The study will evaluate the effectiveness of this regimen, including the achievement of deep remission, as well as its safety and tolerability in this high-risk patient population.

开始日期2026-12-31

申办/合作机构

Le LYSARC

[+3]

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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5,465

项与维奈克拉相关的文献（医药）

2027-01-01·CLINICA CHIMICA ACTA

BCL-2 in leukemia: Enhancing clinical utility

Review

作者: Khan, Abida ; Khalid, Mohammad ; Bakht, Md Afroz ; Rehman, Zia Ur ; Singla, Neelam ; Ali, Md Imtiaz ; Kamal, Mohammad Azhar ; Imran, Mohd

B-cell lymphoma 2 (BCL-2) is an important anti-apoptotic protein in the mitochondrial cell death pathway and is biologically significant for leukemic cell survival, apoptosis resistance, drug resistance, and disease persistence. BCL-2 is clinically relevant in hematopathology and laboratory medicine, as it is a central regulator of apoptosis in several leukemia subtypes. This review evaluates BCL-2 primarily as a prognostic, treatment-stratifying, and venetoclax-related predictive biomarker in leukemia, while critically examining its more limited and context-dependent diagnostic contribution. We summarize existing data on the association of BCL-2 expression and functional dependency with leukemia biology, cytogenetic and molecular risk factors and survival. We also explore its use in distinguishing leukemic populations from non-malignant hematopoietic populations, primarily in conjunction with morphology, immunophenotyping, cytogenetics, molecular testing, and other markers of apoptosis. Laboratory considerations for clinical implementation are addressed, such as specimen selection, inter-platform variability, antibody and gating strategies, immunohistochemistry, flow cytometry, transcript-based assessment, functional apoptotic profiling, and preanalytical, analytical, and interpretative variability. We also discuss the clinical relevance of BCL-2 in venetoclax-based therapy, where dependence on BCL-2 may be more informative than expression. Overall, BCL-2 is better supported as prognostic and treatment-stratifying marker than a diagnostic marker in leukemia. To move BCL-2 assessment into routine laboratory practice, it will be necessary to establish harmonized thresholds for BCL-2 assays and to validate the analytical workflow and result in prospective studies. This review outlines the potential and current challenges of BCL-2 as an emerging biomarker in human leukemia within a laboratory medicine framework.

2026-12-01·Blood Research

The role of m6A RNA methyltransferase METTL3 in drug resistance mechanisms in acute myeloid leukemia

Review

作者: Prajapati, Suresh ; Patel, Mansi ; Gupta, Reeshu ; Jyotishi, Charmi

Abstract:

This review examines the role of METTL3, a core RNA methyltransferase, in therapeutic resistance in acute myeloid leukemia (AML) and discusses emerging strategies to address this challenge. METTL3 regulates N 6 -methyladenosine (m 6 A) modifications on transcripts involved in key cellular processes, including apoptosis (BCL2, MCL1), metabolism (PGC-1α, CSRP1), proliferation (MYC), autophagy (FOXO3), and bone marrow microenvironmental interactions (ITGA4, AKT1). These modifications enhance the stability and translation of resistance-associated genes, supporting leukemic cell survival under treatment pressure. Pharmacological targeting of METTL3 has shown efficacy in preclinical AML models. Inhibitors such as STM2457, METTL3-directed PROTACs, and rational drug combinations with agents including venetoclax, anthracyclines, and ATRA, have reversed resistance phenotypes and impaired leukemic cell fitness. Beyond canonical resistance mechanisms, METTL3 also regulates noncoding RNAs, autophagy, and metabolic–epigenetic crosstalk, including histone lactylation, linking epitranscriptomic regulation to broader resistance pathways. By integrating molecular, cellular, and microenvironmental evidence, this review underscores METTL3 as a central driver of drug resistance and a promising therapeutic target in relapsed or refractory AML. Unlike previous summaries, it highlights the convergence of METTL3-mediated m 6 A modifications with noncoding RNA regulation, autophagy, and niche adaptation, and critically evaluates emerging therapeutic approaches, including catalytic inhibitors, PROTACs, and natural compounds.

2026-12-01·Blood Research

Novel treatment strategies in acute myeloid leukemia

Review

作者: Simkhada, Shailesh ; Joshi, Utsav ; Dhakal, Prajwal

Acute myeloid leukemia (AML) is a molecularly diverse hematologic malignancy, with clinical outcomes driven by patient fitness, disease biology, and the depth of remission. Over the past decade, the increasing use of molecular studies has enabled genotype-directed therapy and accelerated the incorporation of targeted agents into both intensive and lower-intensity treatment regimens. FLT3 inhibitors are now well established across a wide disease spectrum, including frontline settings, relapsed/refractory disease, and post-allogeneic hematopoietic cell transplantation maintenance, with emerging evidence supporting measurable residual disease (MRD)-adapted maintenance approaches. IDH1/2 inhibitors offer therapy for specific molecular subsets, particularly in older or unfit patients, and are increasingly being evaluated in doublet and triplet combinations. Venetoclax-based regimens have become a cornerstone for older or unfit patients with AML and are being optimized through response-adapted dosing and careful partner selection to mitigate cytopenias while deepening remission. Menin inhibitors represent a major therapeutic advance for KMT2A-rearranged and NPM1-mutated AML and are rapidly moving into combination and earlier-line treatment strategies. In parallel, immune and cellular therapies, including antibody-drug conjugates, immune checkpoint modulation, bispecific engagers, and chimeric antigen receptor T-cell and chimeric antigen receptor natural killer constructs, are under investigation, although durable, practice-changing benefits outside select settings remain limited to date. This review synthesizes pivotal clinical data and translational principles informing emerging targeted, immune-based, and cellular strategies in AML, with an emphasis on remission depth, MRD-driven decision-making, and relapse prevention.

2,926

项与维奈克拉相关的新闻（医药）

2026-09-01

·GlobeNewswire-Health Care

NetraMark Strengthens Board of Directors with Appointment of Veteran Pharmaceutical Executive Dr. Keith Fournier

Pharmaceutical industry leader brings more than 20 years of global drug development, regulatory strategy and portfolio leadership experience across AbbVie, Merck KGaA, AstraZeneca and AbivaxTORONTO, Sept. 01, 2026 (GLOBE NEWSWIRE) -- NetraMark Holdings Inc. (the "Company" or "NetraMark") (TSX: AIAI) (OTCQB: AINMF) (Frankfurt: PF0), a clinical intelligence company transforming clinical trials with AI-powered precision analytics in the pharmaceutical industry, announced today the appointment of Dr. Keith M. Fournier, PhD, RAC, to its Board of Directors. Dr. Fournier is a highly experienced pharmaceutical executive with more than two decades of leadership across drug development, global regulatory strategy and cross-functional portfolio management. His career has spanned the development lifecycle from Investigational New Drug (IND) activities through global registration and lifecycle management, with senior positions at several of the world’s leading pharmaceutical organizations. Dr. Fournier currently serves as Senior Vice President, Head of Regulatory Affairs, at Abivax where he leads the company’s regulatory organization and strategy supporting advancement of its pipeline. Prior to Abivax, he served as Vice President, Global Regulatory Affairs at EMD Serono, the healthcare business of Merck KGaA, where he led approximately 75 regulatory leaders and project managers responsible for regulatory strategies across a broad global portfolio. Dr. Fournier also contributed to overall global regulatory organizational strategy as a member of the Regulatory, Quality and Safety leadership team. In addition, Dr. Fournier previously held a series of senior leadership positions at AbbVie. As an Asset Strategy Leader within AbbVie’s oncology organization, he led cross-functional strategy for VENCLEXTA® (venetoclax) programs across hematologic and solid tumors. He previously served as Senior Director and Therapeutic Area Lead for Global Regulatory Affairs in Immunology and as Global Regulatory Lead for HUMIRA®. Prior thereto, Dr. Fournier also spent five years at MedImmune, an AstraZeneca company, where he served as a Global Regulatory Lead across the respiratory, inflammation and autoimmune portfolio, including late-stage development programs in systemic lupus erythematosus, scleroderma and myositis. “Keith brings the type of pharmaceutical industry experience that we believe can add significant value to NetraMark as we continue expanding our relationships with drug developers,” said George Achilleos, CEO of NetraMark. “He has spent more than 20 years inside some of the world’s most sophisticated pharmaceutical organizations making decisions around regulatory strategy, clinical development and the advancement of important therapeutic assets. His understanding of how pharmaceutical companies evaluate clinical evidence, make development decisions and interact with regulators provides an extremely valuable perspective for our Board.” Achilleos continued, “As NetraMark works with pharmaceutical and biotechnology companies to seek to extract greater value from clinical trial data and inform subsequent clinical development strategies, we believe it is increasingly important that our Board combines expertise in artificial intelligence with executives who deeply understand how drugs actually move through the development and regulatory process. Keith significantly strengthens that capability.” Dr. Fournier commented, “Clinical development continues to face a fundamental challenge in understanding heterogeneous patient populations and identifying the factors that influence treatment response. NetraMark is approaching this problem with a differentiated technology designed specifically around the realities of clinical trial data. I am excited to join the Board and contribute my experience in drug development and regulatory strategy as the Company works to expand the application of NetraAI across the pharmaceutical industry.” Dr. Fournier earned his PhD in Pharmacology from the University of Pennsylvania School of Medicine and holds a Bachelor of Science in Biology and Bachelor of Arts in Economics, cum laude, from Duke University. He has also authored multiple peer-reviewed scientific publications. For more information on NetraMark and NetraAI, visit www.netramark.com. About NetraAI NetraAI is engineered to include focus mechanisms that separate small datasets into explainable and unexplainable subsets. Unexplainable subsets are collections of patients that can lead to suboptimal overfit models and inaccurate insights due to poor correlations with the variables involved. NetraAI uses explainable subsets to derive insights and hypotheses (including factors that influence treatment and placebo responses and adverse events), potentially increasing the likelihood of a clinical trial's success. Many other AI methods lack these focus mechanisms and assign every patient to a class, often leading to "overfitting," which drowns out critical information that could have been used to improve a trial's chance of success. About NetraMark NetraMark is focused on being a leader in the development of Generative Artificial Intelligence (Gen AI)/Machine Learning (ML) solutions that are targeted at the biotechnology and pharmaceutical industries. The Company's product offering uses a novel topology-based algorithm that has the ability to parse patient data sets into subsets of people that are strongly related according to several variables simultaneously. This allows NetraMark to use a variety of ML methods, depending on the character and size of the data, to transform the data into powerfully intelligent data that activates traditional AI/ML methods. The result is that NetraMark can work with much smaller datasets often prevalent in clinical trials, and accurately segment diseases into different types, as well as help to classify patients for sensitivity to drugs and/or efficacy of treatment. For further details on the Company please see the Company's publicly available documents filed on the System for Electronic Document Analysis and Retrieval+ (SEDAR+). Forward-Looking Statements This press release contains "forward-looking information" within the meaning of applicable Canadian securities legislation including statements regarding the potential impact and perspective that Dr. Fournier can bring to the Company's business and ability to engage with target markets, the Company’s anticipated business plans and strategies, and the potential for our technology to extract greater value from clinical trial data and inform subsequent clinical development strategies along with the potential expansion of the application of NetraAI across the pharmaceutical industry, which are based upon NetraMark’s current internal expectations, estimates, projections, assumptions and beliefs, and views of future events. Forward-looking information can be identified by the use of forward-looking terminology such as “expect”, “likely”, “may”, “will”, “should”, “intend”, “anticipate”, “potential”, “proposed”, “estimate” and other similar words, including negative and grammatical variations thereof, or statements that certain events or conditions “may”, “would” or “will” happen, or by discussions of strategy. Forward-looking information includes estimates, plans, expectations, opinions, forecasts, projections, targets, guidance, or other statements that are not statements of fact. The forward-looking statements are expectations only and are subject to known and unknown risks, uncertainties and other important factors that could cause actual results of the Company or industry results to differ materially from future results, performance or achievements. Any forward-looking information speaks only as of the date on which it is made, and, except as required by law, NetraMark does not undertake any obligation to update or revise any forward-looking information, whether as a result of new information, future events, or otherwise. New factors emerge from time to time, and it is not possible for NetraMark to predict all such factors. When considering these forward-looking statements, readers should keep in mind the risk factors and other cautionary statements as set out in the materials we file with applicable Canadian securities regulatory authorities on SEDAR+ at www.sedarplus.com including our Annual Information Form for the year ended September 30, 2025. These risk factors and other factors could cause actual events or results to differ materially from those described in any forward-looking information. The Toronto Stock Exchange does not accept responsibility for the adequacy or accuracy of this release. Contact Information: Swapan Kakumanu - CFO | swapan@netramark.com | 403-681-2549Adam Peeler – Investor Relations | adam.peeler@loderockadvisors.com | 416-427-1235 Media Contact:netramark@ampublicrelations.com

高管变更

2026-09-01

·BioPlus

下一代ADC拼什么？8类新载荷的机制与进展

作者：琉璃韵前言当TOP1抑制剂和微管抑制剂成为ADC开发的主流选择，下一轮差异化竞争正在从“更换靶点”进一步转向“更换药理机制”。新型载荷的核心价值，不只是提高细胞毒性，而是借助抗体的选择性递送，为过去受限于全身暴露、靶点相关毒性或耐药问题的小分子机制重新建立治疗窗口。因此，下一代载荷创新并不等同于寻找“毒性更强”的分子，而是筛选那些适合被ADC重新递送的药理机制。结合目前公开项目，新型载荷的探索已延伸至蛋白脂质化、PI3K/mTOR与RAS信号、DNA损伤应答、RNA剪接与翻译以及凋亡调控等多个方向。以下选取八类代表性机制进行梳理：八类新型载荷 01｜NMT抑制剂：从蛋白脂质化切入ADC N-肉豆蔻酰转移酶（NMT）催化蛋白N-肉豆蔻酰化。人体主要表达NMT1和NMT2，这一翻译后修饰参与多类蛋白的膜定位、稳定性与功能调控。抑制NMT可同时扰动肿瘤细胞依赖的多个蛋白网络，因此，NMT抑制剂（NMTi）为ADC提供了区别于TOP1抑制剂和微管抑制剂的新型作用机制。 Myricx Bio围绕NMTi载荷布局了多项ADC，包括HER2靶向的MYX2449、TROP2靶向的MYX2468和B7-H3靶向的MYX2470。公开资料显示，这些项目分别采用trastuzumab、sacituzumab和ifinatamab来源的抗体骨架。跨多个靶点的布局说明，Myricx并非只在验证单一候选药物，而是在验证NMTi载荷与不同抗体组合的适配性。在HER2阳性的NCI-N87胃癌和BT-474乳腺癌模型中，MYX2449显示出细胞杀伤活性；在体内模型中，MYX2449、MYX2468和MYX2470也分别在相应靶点相关模型中表现出肿瘤生长抑制。 MYX2449不仅可以杀伤HER2阳性的靶细胞，而且能够产生一定程度的旁观者效应，对邻近的HER2阴性细胞产生影响。NMTi作为payload可能有助于应对实体瘤中的抗原异质性。 2026年7月6日，Novartis宣布拟收购Myricx Bio。交易总价值最高15亿美元，包括11亿美元现金首付款及最高4亿美元潜在里程碑付款。Novartis在公告中明确将Myricx的两项领先ADC资产及NMTi载荷平台列为交易重点，反映出大型药企对新机制载荷及其平台化潜力的持续关注。映恩生物近期半年报首次明确，DUP10为一类新型NMT抑制剂。不过，DUP10目前仍处于全球IND申报准备阶段，具体结构、定量活性及毒理数据尚未公开。 02｜PI3K/mTOR抑制剂：以靶向递送重塑治疗窗 PI3K/AKT/mTOR通路广泛参与肿瘤细胞增殖、存活、代谢和蛋白合成，但该通路同样承担正常组织的生理功能，系统性抑制可能引发高血糖等机制相关毒性。将PI3K/mTOR抑制剂设计为ADC载荷，核心目的不是简单增强小分子效力，而是利用抗体介导的肿瘤选择性递送，改变其组织分布并降低非靶组织暴露。在公开的HCC-1954、HN5和BR156等CDX/PDX模型中，TROP2-PI3K/mTORi ADC显示出肿瘤生长抑制，部分模型观察到持续性肿瘤回缩。 03｜Gαq/11抑制剂：让ADC精准匹配肿瘤驱动通路 DYP688通过靶向PMEL17递送Gαq/11抑制剂SDZ475，用于探索GNAQ/GNA11突变黑色素瘤的精准治疗。ADC与PMEL17结合并内化后释放SDZ475，抑制异常激活的Gαq/11信号，并进一步影响PKC/MAPK、Rho/Rac、β-catenin和YAP等下游通路。与广谱细胞毒载荷相比，DYP688体现了“肿瘤抗原+驱动通路”的双重生物学匹配。 2026年发表的Ⅰ期研究显示，在66例可评估患者中， ORR为19.7%，47例患者（71.2%）出现不同程度的靶病灶缩小，mPFS为7.2个月；TRAE≥3级发生率为7.6%。 04｜pan-RAS抑制剂：重新设计适合ADC递送的小分子 RAS通路是肿瘤领域最重要的驱动机制之一。尽管近年来KRAS抑制剂取得快速进展，但系统性RAS抑制仍面临一个核心问题：如何在维持持续RAS通路抑制的同时，降低正常组织暴露。 Zymeworks的RASi-ADC开发首先解决的并不是“RAS是否值得抑制”，而是“什么样的RAS抑制剂适合成为ADC payload”。 Zymeworks针对ADC应用筛选了超过170个新型RASi分子，并从药效强度、疏水性及payload属性等维度进行优化。最终形成的RASi payload被设计用于高DAR偶联，并进一步拓展至PTK7、Ly6E和CLDN18.2等不同ADC靶点。在临床前实验中，RASi ADC显示出较低的正常组织payload暴露；与此同时，肿瘤内DUSP6等RAS通路药效学指标显示出持续抑制。因此，RASi ADC的核心价值并非简单提高RAS抑制剂的药效，而是通过抗体介导的递送，实现“更集中、更持续的肿瘤内RAS抑制” 。 05｜ATR抑制剂：从DNA损伤应答寻找TOP1之外的路径 ATR是DNA损伤应答和复制压力调控的关键激酶。存在DNA修复缺陷或高复制压力的肿瘤细胞，往往更依赖ATR维持复制叉稳定和细胞存活；抑制ATR可削弱复制压力应答，并可能诱导复制灾难。这为ATRi作为ADC载荷提供了明确的生物学基础。 DCx Biotherapeutics在AACR 2026公布了PD-L1-ATRi和TROP2-ATRi ADC的临床前研究。在携带TOP1 R364H等耐药相关改变的模型中，ATRi ADC仍保留体外活性，而Dato-DXd活性下降。表明ATRi载荷可能为TOP1抑制剂载荷不敏感或耐药场景提供差异化选择。 06｜RNA剪接抑制剂：从DNA和微管走向RNA加工 Akari Therapeutics开发的PH1代表另一类载荷思路：通过干扰剪接体（spliceosome）功能，引发广泛的异常RNA剪接，进而破坏关键蛋白的正常表达并产生细胞毒效应。该机制将ADC载荷的作用层级从微管和DNA进一步拓展至RNA加工。在公开的多药耐药（MDR）转运蛋白高表达模型中，PH1的体外活性未见明显下降，而MMAE的杀伤能力明显减弱。表明PH1可能在部分由药物外排泵介导的耐药背景中保留活性。 07｜MCL-1抑制剂：定向解除血液肿瘤的抗凋亡屏障 Servier开发的S227928是一款靶向CD74、偶联MCL-1抑制剂的ADC。MCL-1是BCL-2家族的重要抗凋亡蛋白，在AML等血液肿瘤中具有重要作用；MCL-1上调也与部分肿瘤对venetoclax耐药相关。 S227928与CD74结合并内化后，在细胞内释放MCL-1抑制剂S64315。解除MCL-1对BIM等促凋亡蛋白的抑制后，可促进BAX/BAK介导的线粒体凋亡。其开发逻辑是利用ADC提高MCL-1抑制剂在目标细胞中的相对暴露，以探索更可控的治疗窗，而不是将MCL-1抑制等同于传统广谱细胞毒机制。 08｜mRNA翻译抑制剂：切断肿瘤细胞的蛋白供应映恩生物开发的DUP5是一类新型mRNA翻译抑制载荷，DUP5选择性抑制真核翻译起始因子eIF4A，干扰帽依赖性（cap-dependent）蛋白翻译，从而下调多种致癌蛋白并诱导细胞周期阻滞与凋亡。 DUP5已被用于构建针对HER2、EGFR、GPC3和BCMA等多种靶点的ADC，相关ADC在多类实体瘤模型中显示抗肿瘤活性，并在部分对TOP1抑制剂载荷相对不敏感的模型中保留活性。其关键差异化假设，是通过抑制蛋白翻译绕开既有TOP1载荷的作用机制。 2026年8月27日，映恩生物与Genentech签订全球合作及许可协议，双方将基于映恩生物的DUPAC新型载荷平台，为Genentech选定的肿瘤靶点开发下一代ADC。映恩生物将获得4500万美元首付款，并有资格就全部合作项目获得合计超过10亿美元的开发、监管及商业化里程碑付款，以及获批产品净销售额的分层特许权使用费。需要强调的是，本次授权对象是DUPAC平台及合作产生的ADC，并非DUP5单一载荷；但DUP5与DUP9、DUP10同属DUPAC，DUP5的临床前数据构成该平台技术验证的重要组成部分。新型payload真正试图解决什么问题？新型载荷的竞争并非单一维度的“毒性升级”，而是在针对传统ADC与系统性小分子的不同瓶颈建立解决方案。小结 ADC正在从“抗体+经典细胞毒素”的组合，逐步演变为“靶向递送平台+机制型药物”的组合。TOP1抑制剂和微管抑制剂仍是当前最成熟的载荷类别，但NMT、PI3K/mTOR、Gαq/11、RAS、ATR、RNA剪接、MCL-1和mRNA翻译等机制的进入，正在持续拓宽ADC能够承载的药理空间。从研发角度看，真正值得关注的并不是新载荷数量本身，而是三个更具体的问题：载荷能否与抗体、连接子和DAR形成稳定可开发的整体；选择性递送能否在人体中实质性改变暴露—效应关系；新机制能否在既有载荷不敏感或耐药人群中提供可验证的临床获益。只有同时跨过这三道门槛，新型payload才可能从“机制新颖”走向“临床成立”。欢迎大家加入BioPlus Partners微信交流群。入群申请联系人：Lisa 邮箱：lisa.li@bp-bioplus.com 电话：18662346610（同微信）入群申请，请先添加Lisa微信，并分享名片入群画像：Biotech/药厂研发/BD、投资机构、临床医生等入群福利：不定时行业资料包分享参考资料 1. https://www.adcreview.com/wp-content/uploads/2023/10/ADC-world-Oct-2023-WADC-poster.pdf 2. 2026AACR poster 3. Carlino, M.S., Kapiteijn, E., Piperno-Neumann, S. et al. An anti-PMEL antibody−drug conjugate with a Gq/11 inhibitor payload in GNAQ/GNA11-mutant melanomas: a phase 1 trial. Nat Med (2026). 4. https://ir.zymeworks.com/static-files/f8d8a220-063a-4e40-a4a5-3ecc36145f58 5. 2026AACR poster 6. https://investor.akaritx.com/static-files/d0829da9-d8de-420a-9173-3ad62e1fdb86 7. 2024 ASH poster 8. 映恩生物管网资料

2026-08-31

·BioSpace

ORYZON Announces Notice of Allowance for U.S. Patent Application Covering Iadademstat Combination With Gilteritinib

Iadademstat is under clinical development in acute myeloid leukemia (AML), including in combination with gilteritinib in the Phase Ib FRIDA trial in patients with FLT3-mutated relapsed/refractory (R/R) AML FRIDA data presented at EHA-2026 showed a high composite complete remission (CRc) rate of 67% in heavily pretreated FLT3-mutated R/R AML patients, together with a favorable safety profile MADRID and CAMBRIDGE, Mass., Aug. 31, 2026 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and a global leader in epigenetics, today announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for U.S. patent application No. US 18/554,241, entitled “Combinations of LSD1 inhibitors for treating myeloid cancers”. The allowed claims cover combinations of iadademstat, or certain other LSD1 inhibitors, with gilteritinib and their use for the treatment of myeloid cancers, including acute myeloid leukemia (AML). Once granted, the U.S. patent is expected to expire in 2042, excluding any potential patent term adjustment or patent term extension. A corresponding patent has recently also been granted in Taiwan, with additional patent applications pending in other jurisdictions. “This U.S. Notice of Allowance further strengthens the intellectual property protection around iadademstat and its use in combination with gilteritinib in AML,” said Neus Virgili, Oryzon’s Chief IP Officer. “Together with our broader portfolio of patents covering iadademstat combinations with other AML therapies, this allowance reinforces the long-term protection of our iadademstat franchise.” Iadademstat is currently being evaluated in seven ongoing oncology clinical trials, including the Phase Ib FRIDA study in combination with gilteritinib in FLT3-mutated relapsed/refractory AML and the Phase Ib ALICE-2 study in combination with venetoclax and azacitidine in first-line AML. Updated positive clinical data from both studies were presented at the European Hematology Association (EHA) 2026 Annual Congress in June. In FRIDA, iadademstat in combination with gilteritinib achieved a composite complete remission (CRc) rate of 67% at the dose level selected for expansion in a heavily pretreated population with relapsed/refractory FLT3-mutated AML, together with a favorable safety profile. These results are particularly encouraging when compared with contemporary real-world data for gilteritinib monotherapy in a broadly comparable population, which reported a CR/CRi rate of 28% and a median overall survival of 7.1 months. In ALICE-2, iadademstat in combination with venetoclax and azacitidine achieved a 100% overall response rate, an 89% CRc rate and a 78% complete remission rate. These findings compare favorably with historical outcomes for venetoclax plus azacitidine, where approximately 36% of patients failed to achieve a clinical response. Additional, more mature data from both studies are expected to be presented by year-end. In addition to this patent family covering combinations with gilteritinib, Oryzon has patent protection covering combinations of iadademstat with venetoclax, azacitidine and other AML therapies, including granted patents in the United States and other major markets. About Oryzon Founded in 2000 and headquartered in Barcelona, Spain, Oryzon (ISIN: ES0167733015) is a clinical-stage biopharmaceutical company and a European leader in epigenetics, with a strong focus on personalized medicine for central nervous system (CNS) disorders and oncology. Oryzon’s team comprises highly experienced pharmaceutical professionals based in Barcelona, Boston, and New Jersey. The Company has an advanced clinical portfolio built around two LSD1 inhibitors: iadademstat, its oncology/hematology program, with several ongoing Phase I and II studies and which has demonstrated strong preliminary clinical activity in acute myeloid leukemia, including a 100% overall response rate (ORR) in first-line AML; and vafidemstat, its lead CNS program, which is Phase III–ready in borderline personality disorder (BPD). In addition, Oryzon is advancing a broader epigenetics pipeline targeting other mechanisms, including HDAC6, for which the Company has nominated ORY-4001 as a clinical candidate for potential development in Charcot–Marie–Tooth disease (CMT), amyotrophic lateral sclerosis (ALS), and other neurological disorders. The Company also operates a robust platform for biomarker identification and target validation across malignant and neurological diseases. For more information, visit About Iadademstat Iadademstat (ORY-1001) is an oral, highly selective inhibitor of the epigenetic enzyme LSD1, with potent differentiating effect in hematologic cancers. Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L acute myeloid leukemia (AML) patients (ALICE trial). Iadademstat is currently being evaluated in combination with azacitidine and venetoclax in 1L AML in the ALICE-2 trial, an investigator-initiated study (IIS) led by OHSU, and in combination with gilteritinib in the company-sponsored Phase Ib FRIDA trial in relapsed/refractory FLT3-mutant AML, with highly encouraging preliminary safety and efficacy data in both trials reported at EHA-2026: 100% overall response rate (ORR) and 89% composite complete remission rate (CRc), with 78% strict CR in 1L AML, and 67% CRc in R/R Flt3-mut AML. Subject to positive final data in the ALICE-2 study, the company has announced plans to conduct a potentially registrational study in 1L AML. Additional studies in hematologic malignancies include an IIS in myelodysplastic syndrome (MDS) and National Cancer Institute (NCI)-sponsored trials in myeloproliferative neoplasms and 1L AML conducted under the Cooperative Research and Development Agreement (CRADA) between Oryzon and the NCI. Beyond hematological cancers, iadademstat is being evaluated in extensive stage small cell lung cancer (ED-SCLC) in a Phase I/II randomized trial in 1L in combination with immune checkpoint inhibition (ICI) sponsored by NCI and led by the Memorial Sloan Kettering Cancer Center, and an IIS trial in combination with ICI and radiotherapy. Oryzon has also expanded iadademstat into non-oncological hematology indications, with ongoing trials in sickle cell disease and essential thrombocythemia. Iadademstat has orphan drug designation for AML in the US and EU and for SCLC in the US. FORWARD-LOOKING STATEMENTS This communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives, and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees, or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward‐looking statements, whether as a result of new information, future events, or otherwise. This document does not constitute an offer or invitation to purchase or subscribe shares in accordance with the provisions of Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017, and/or the restated text of the Securities Market Law, approved by Law 6/2023 of 17 March, and its implementing regulations. Nothing in this document constitutes investment advice. In addition, this document does not constitute an offer of purchase, sale or exchange, nor a request for an offer of purchase, sale or exchange of securities, nor a request for any vote or approval in any jurisdiction. The shares of Oryzon Genomics, S.A. may not be offered or sold in the United States of America except pursuant to an effective registration statement under the Securities Act of 1933 or pursuant to a valid exemption from registration. Spain Oryzon IR & Media, Europe & US Patricia Cobo/Mario Cordera Emili Torrell Sandya von der Weid Atrevia Chief BD Officer LifeSci Advisors, LLC +34 91 564 07 25 +34 673 33 97 65 +34 93 515 1313 +41 78 680 05 38 pcobo@atrevia.com mcordera@atrevia.com etorrell@oryzon.com svonderweid@lifesciadvisors.com

临床结果临床1期临床2期孤儿药临床3期

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 GK	2025-03-27
难治性套细胞淋巴瘤	日本	AbbVie GK	2025-03-27
难治性慢性淋巴细胞白血病	日本	AbbVie GK	2019-09-20
复发性慢性淋巴细胞白血病	日本	AbbVie GK	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	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	Genentech, Inc.	2022-10-01
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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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03054896 (CRC-043 \| phase 2, CTgov)	临床2期	里氏综合症	69	Etoposide+Cyclophosphamide+Pegfilgrastim+Prednisone+Venetoclax+Doxorubicin+Rituximab+Vincristine (VR-EPOCH (Cohort 1))	膚艱艱憲廠獵構選鏇襯 = 鬱鑰選鹽蓋壓壓觸鑰製遞夢壓製鹹糧遞膚繭獵 (醖鑰糧餘鹽窪鹹艱膚廠, 觸齋範繭憲遞襯糧廠築 ~ 遞糧窪淵簾積餘憲簾願) 更多	-	2026-07-10
NCT03054896 (CRC-043 \| phase 2, CTgov)	临床2期	里氏综合症	69	cyclophosphamide+prednisone+Venetoclax+doxorubicin+Vincristine Sulfate+rituximab (VR-CHOP (Cohort 2))	膚艱艱憲廠獵構選鏇襯 = 選鬱願構夢鏇壓選繭鹹遞夢壓製鹹糧遞膚繭獵 (醖鑰糧餘鹽窪鹹艱膚廠, 鑰積遞餘獵膚觸遞餘鹽 ~ 蓋齋艱選網鹽選艱壓鏇) 更多	-	2026-07-10
NCT04285567 (CRISTALLO, Pubmed \| Blood)	临床3期	慢性淋巴细胞白血病一线	166	Venetoclax-obinutuzumab (VenO)	網範衊艱顧願範艱簾廠(鹽窪網遞夢獵範憲膚艱) = 鏇壓積窪範餘鹹鏇膚鑰鏇網襯顧觸衊夢鑰築顧 (遞艱壓壓鑰獵遞夢鬱鑰 ) 更多	积极	2026-06-11
NCT04285567 (CRISTALLO, Pubmed \| Blood)	临床3期	慢性淋巴细胞白血病一线	166	Fludarabine, cyclophosphamide, and rituximab (FCR) or bendamustine-rituximab (BR)	網範衊艱顧願範艱簾廠(鹽窪網遞夢獵範憲膚艱) = 鏇鬱鑰選積襯鏇顧醖憲鏇網襯顧觸衊夢鑰築顧 (遞艱壓壓鑰獵遞夢鬱鑰 ) 更多	积极	2026-06-11
NCT03573024 (CTgov)	临床2期	急性髓性白血病	36	Venetoclax+Azacitidine	衊齋窪製襯廠製壓選獵 = 築糧網鹽鏇鬱觸積餘廠鏇築憲窪鹽艱鑰鹹鹽鑰 (簾齋築艱築鑰憲艱糧顧, 廠鏇製醖餘淵廠遞窪顧 ~ 鏇壓獵獵遞鹹夢齋蓋觸) 更多	-	2026-06-11
NCT05025423 (CTgov)	临床2期	套细胞淋巴瘤 \| 难治性套细胞淋巴瘤	7	Venetoclax Oral Tablet [Venclexta]	膚繭餘醖膚願獵範簾壓 = 繭窪齋餘鏇糧蓋壓獵鏇糧鬱鬱窪鬱製窪顧憲淵 (艱衊夢遞壓窪壓憲遞築, 積鑰淵網構襯鹹構製窪 ~ 鑰餘範廠築衊窪構淵顧) 更多	-	2026-06-09
NCT04657081 (ASCERTAIN-V, Pubmed \| N Engl J Med)	临床1/2期	急性髓性白血病	189	Oral Decitabine-Cedazuridine + Oral Venetoclax	獵鹹鬱鹽鬱餘餘糧鹹築(鏇網鑰憲構範遞觸鹽遞) = 鏇齋觸衊齋壓構膚觸鬱糧築網糧築憲構製製選 (遞網製餘簾膚鏇衊齋繭, 36 ~ 57) 更多	积极	2026-06-04
NCT05084027 (Pubmed \| Cancer)	临床2期	急性髓性白血病 \| 骨髓增生异常综合征	60	Venetoclax+Fludarabine+Melphalan	壓鹽憲壓繭網壓鏇顧鏇(蓋壓願淵鏇築窪鹽顧鏇) = 蓋選艱鏇鏇衊膚襯鹹齋範壓簾襯範醖鹹鹽網鬱 (餘齋選夢齋蓋範膚繭鏇, 64.8 ~ 86.8) 更多	积极	2026-06-01
ASCO2026	N/A	慢性粒单核细胞白血病	145	Venetoclax-based regimens	憲夢憲構襯鑰膚鏇簾醖(製築膚繭窪願鏇鏇齋窪) = 構選構糧醖齋選觸窪範遞範構獵簾簾壓積選顧 (醖廠憲齋鑰願廠構廠鏇, 9.4 ~ 34.9) 更多	积极	2026-05-29
ASCO2026	N/A	急性髓性白血病	55	Azacitidine	鹽憲繭夢顧積製夢網製(製鑰鹹膚築遞蓋鑰窪糧) = 餘簾願積構廠製襯鏇繭獵觸鹽鑰遞顧膚醖觸壓 (簾鹹糧築製齋餘糧醖鬱 ) 更多	积极	2026-05-29
ASCO2026	N/A	急性髓性白血病	55	Combinations of HMAs±LDAC	淵築鹹鹹遞網選構範範(餘餘鏇醖簾餘選鑰醖壓) = 願艱壓積蓋淵窪顧構遞簾齋遞繭醖網鬱積構廠 (網觸廠糧壓簾鏇壓顧憲, 17 ~ 35) 更多	积极	2026-05-29
ASCO2026	N/A	-	280	7+3	顧簾窪製醖醖網夢糧壓(獵醖鹽選衊製蓋鹽製淵) = 廠遞遞醖鑰顧網蓋鹹願夢鬱蓋選築醖獵艱壓淵 (製憲遞積遞壓鏇餘鏇憲 ) 更多	积极	2026-05-29
ASCO2026	N/A	-	280	azacitidine plus venetoclax	顧簾窪製醖醖網夢糧壓(獵醖鹽選衊製蓋鹽製淵) = 糧醖憲觸鹹齋願選鹹憲夢鬱蓋選築醖獵艱壓淵 (製憲遞積遞壓鏇餘鏇憲 ) 更多	积极	2026-05-29
ASCO2026	N/A	急性髓性白血病	16	HiDAC/mitoxantrone/venetoclax (HMV)	膚艱廠繭網觸構顧鏇顧(網觸窪觸鑰鬱衊鑰鹹獵) = 簾鏇鬱簾觸齋艱窪選齋壓構鹽選繭範構遞獵壓 (餘餘糧簾淵鹽憲積壓齋 ) 更多	积极	2026-05-29