A Multicenter, Randomized, Controlled, Open-label, Rater-blinded Study to Evaluate Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of ALXN1840 Versus Standard of Care in Pediatric Participants With Wilson Disease

This study is being conducted to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics of ALXN1840 versus standard of care in pediatric participants with Wilson disease (WD).

开始日期2021-09-13

申办/合作机构

Alexion Pharmaceuticals, Inc.

EUCTR2020-005832-31-DK

/ Completed临床2期

Efficacy of ALXN1840 on human hepatic copper uptake quantified with 64CuCl2 PET/CT-scan.

开始日期2021-04-13

申办/合作机构

Alexion Pharmaceuticals, Inc.

EUCTR2021-000102-25-DK

/ Completed临床2期IIT

Efficacy of ALXN1840 on human biliary copper excretion quantified with 64CuCl2 PET/MR-scan

开始日期2021-03-23

申办/合作机构

Århus Universitetshospital

100 项与四硫钼酸二胆碱相关的临床结果

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100 项与四硫钼酸二胆碱相关的转化医学

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100 项与四硫钼酸二胆碱相关的专利（医药）

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项与四硫钼酸二胆碱相关的文献（医药）

2025-12-01·JOURNAL OF HEPATOLOGY

Oral bis-choline tetrathiomolybdate rapidly improves copper balance in patients with Wilson disease

Letter

作者: Weiss, Karl Heinz ; Sandahl, Thomas Damgaard ; Askari, Fred ; Medici, Valentina ; Ala, Aftab

2025-05-26·JACS Au

Copper Chelation Induces Morphology Change in Mitochondria of Triple-Negative Breast Cancer

Article

作者: Wang, Ting ; Aghaei, Parisa ; Xiao, Zhen ; Burke, Peter J. ; Rao, Jianghong ; Lim, Irene ; Lee, ChiaHung

Recent studies implicate mitochondria playing a key role in the cellular response to copper depletion therapy; however, evidence has been indirect and downstream, and the initial target of chelation remains to be defined. Here, we show, using super-resolution voltage and structure imaging microscopy, that copper chelation directly affects mitochondria morphology (causing fragmentation of the filamentous network) and ultrastructure (causing internal cristae remodeling). When triple-negative breast cancer cells are treated with a mitochondria-targeting copper chelator, mitochondria undergo an irreversible change in morphology from tubular to spherical. This process can be prevented by the addition of exogenous copper during the treatment. We find that a tailor-designed chelating agent with positive charges to target mitochondrial electrostatics localizes inside the mitochondrial cristae in a voltage-dependent manner. On pharmacological induction of membrane potential collapse, the chelator is dispersed while the mitochondrial cristae structure is preserved. These results indicate that voltage-dependent localization/targeting of the copper chelator in mitochondrial cristae plays a key role in its cytotoxicity.

2024-12-01·EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Pharmacodynamic insights into maresin 1: Enhancing flap viability via the keap1/Nrf2 axis to control ROS-driven apoptosis and ferroptosis

Article

作者: Yang, Enhui ; Ma, Xianhui ; Fang, Pin ; Lai, Yingying ; Lu, Jingzhou ; Li, Guangyao ; Gao, Weiyang ; Yang, Ningning ; Cheng, Sheng ; Jiang, Renhao ; Lu, Keyu

Random flaps are widely used in tissue reconstruction, but the high incidence of flap necrosis after operation remains a significant challenge. Maresin 1 (MaR1), a mediator derived from docosahexaenoic acid, has been shown to have significant effects in resolving inflammation and promoting tissue regeneration. This study investigated the role of MaR1 in the survival of random flaps. Histological analysis, laser Doppler blood flow imaging, Masson trichrome staining, and survival area analysis were used to assess the viability of the flaps. Apoptosis, ferroptosis, oxidative stress, angiogenesis, and the underlying mechanisms were explored by examining the expression of specific molecules using immunofluorescence, western blotting, and other immunological and molecular biology techniques. The findings demonstrated that MaR1 could improve flap lifespan by significantly reducing oxidative stress, apoptosis, and ferroptosis, as well as by enhancing angiogenesis. The Keap1-Nrf2 pathway was upregulated by MaR1, which inhibited ROS-mediated apoptosis and ferroptosis. The protective effect of MaR1 on flap survival was abolished by ML385. Our findings indicate that MaR1 could be a novel therapeutic agent for enhancing flap treatment outcomes.

项与四硫钼酸二胆碱相关的新闻（医药）

2026-07-10

·醉心科学

醉心资讯 NO.202606 | 产业市场

关注醉心科学，聚焦干细胞领域研究进展醉心资讯以干细胞与再生医学领域综合信息跟踪和报道为主，围绕干细胞和再生医学领域创新研究主题开展编译报道和分析研究。 “战略与政策”栏目编译报道干细胞与再生医学国内外重要战略、政策、举措和项目部署方面的信息综述；“研究进展”栏目报道领域国内外科学技术前沿研究动态信息；“产业市场”栏目报道领域产品与服务应用及产业市场发展动态信息。产业市场产业发展1.跃赛生物干细胞疗法UX-DA003获美国FDA临床试验许可2.‌Made Scientific与Pluristyx达成战略合作打通下一代iPSC细胞疗法全链条开发制造路径3.‌CCRM、OmniaBio和Avectas合作推进细胞疗法生产自动化升级4.AcCELLerated Biologic与Forever Labs就推进干细胞储存和再生医学解决方案达成战略合作伙伴关系5.全球首款实体瘤CAR-T疗法在我国上市6.UpDoc推出首个获得FDA批准的临床人工智能平台市场动态7.FesariusTherapeutics完成2000万美元A轮融资推进水凝胶真皮再生模板覆盖全美医院系统8.xCures完成4600万美元B轮融资推进分散医疗记录转化为可用于决策的数据9.Syntax Bio扩大A轮融资规模用于推进Cellgorithm™平台10.西湖生物完成超亿元融资加速全球首创红细胞药物临床开发 01 跃赛生物干细胞疗法UX-DA003获美国FDA临床试验许可 2026年6月19日，上海跃赛生物科技有限公司（以下简称“跃赛生物”）宣布，UX-DA003（异体iPSC来源中脑多巴胺能神经前体细胞注射液）正式获得美国食品药品监督管理局（FDA）的临床试验默示许可（IND批件）。这是继2026年6月3日该产品获中国国家药品监督管理局（NMPA）临床试验默示许可后，跃赛生物在国际化征程中斩获的又一关键里程碑。至此，跃赛生物的“自体+异体”双路径战略在帕金森病（PD）领域已实现中美两地监管的全面贯通，标志着中国原研细胞疗法正式进军全球医药市场。 UX-DA003的顺利获批，核心依托于跃赛生物自主研发的高通量谱系示踪技术（SISBAR）平台和高效定向分化技术平台，在这两大技术平台的支撑下，UX-DA003的药物纯度远高于国际同行，移植后功能性细胞-多巴胺能神经元的获得率从国际平均水平的2-15%提高到了50-60%（数据均为已发表的经国际同行评议和审核的公开数据），跃赛还建立了行业最科学和精准的PD细胞治疗药物质控技术体系。UX-DA003的高度纯度，使得UX-DA003的使用剂量仅为国际平均水平的1/5-1/2，最大限度减少了细胞药物移植带来的周围脑组织压迫，安全性更高。UX-DA003含有更少的杂质细胞，移植后6个月增殖细胞的比例仅为0.23%（国际水平在0.5-11%），最大程度避免了脱靶细胞释放的其他神经递质带来的潜在副作用，以及脱靶本身过度增殖带来的长期致瘤性与安全性风险。 UX-DA003使用了跃赛生物完全自主研发的iPSCs种子细胞。跃赛通过其建立的GMP级iPSCs构建平台，构建了遗传背景清晰、分化潜能优异、符合中美标准可全球商业化报药的iPSC种子细胞库，并已通过中国食品药品检定研究院（NIFDC）及美国FDA的严格质量检测与认定。原文标题：跃赛生物UX-DA003获美国FDA临床试验许可全球化布局再下一城原文链接：https://mp.weixin.qq.com/s/SE_8lLOgvQs6_4fHjKyaGg 02 Made Scientific与Pluristyx达成战略合作打通下一代iPSC细胞疗法全链条开发制造路径 2026年6月4日，美国临床及商业化阶段细胞疗法CDMO头部企业Made Scientific，与iPSC平台技术服务商Pluristyx联合宣布达成深度战略合作伙伴关系，双方将把Pluristyx的临床级iPSC技术体系全面整合进Made Scientific的端到端CDMO服务中，为全球细胞与基因疗法开发者提供从细胞系构建到商业化供应的一站式解决方案。根据合作协议，Pluristyx将成为Made Scientific的iPSC及其衍生细胞的核心供应与开发合作伙伴，向其开放符合FDA、欧盟EMA附录I等全球监管标准的科研级与GMP级iPSC细胞系，以及iNK、iMSC等衍生细胞类型，覆盖从研发、临床阶段到商业化生产的全流程需求。依托双方技术与能力的深度整合，疗法开发者无需额外花费数月时间完成起始材料溯源重检、多供应商资质审核、多份监管文件协调等冗余工作，可直接从项目启动首日就开展工艺开发与GMP生产，大幅压缩IND申报周期，显著降低iPSC疗法从概念落地到进入临床的综合成本。同时双方还将联合提供iPSC基因编辑、定向分化、定制化GMP细胞库构建等延伸服务，通过单一责任主体的协同工作流，消除传统定制化iPSC项目中多环节交接带来的风险与进度延误。双方高管表示，此次合作直击当前iPSC疗法产业化的核心痛点，将为下一代自体及异基因细胞疗法的高效合规落地提供标准化的“即插即用”支撑平台。原文标题：Made Scientific and Pluristyx Launch Integrated iPSC Development & Manufacturing Partnership to Advance Next-Generation iPSC-derived Cell Therapies 原文链接：https://www.prnewswire.com/news-releases/made-scientific-and-pluristyx-launch-integrated-ipsc-development--manufacturing-partnership-to-advance-next-generation-ipsc-derived-cell-therapies-302791712.html 03 CCRM、OmniaBio和Avectas合作推进细胞疗法生产自动化升级 2026年6月15日，加拿大再生医学商业化中心（CCRM）及其旗下专注于细胞与基因疗法的合同研发生产组织（CDMO）——加拿大最大的细胞与基因疗法CDMO公司OmniaBio，宣布与爱尔兰细胞疗法先进制造公司Avectas达成一项合作协议，旨在评估并整合其自动化、集成化、可扩展的细胞疗法生产平台‌。此次合作将探索该平台如何与CCRM的全球枢纽网络以及OmniaBio推动的标准化、自动化工作流程形成互补。合作将聚焦于解决当前细胞疗法制造在规模化扩产、自动化实施与成本控制方面的核心瓶颈，加速突破性疗法的可及性。OmniaBio在汉密尔顿运营着加拿大最大的CGT生产设施，并致力于通过整合人工智能、自动化等先进技术来革新治疗产品的生产。此次与Avectas的平台评估，是CCRM全球“中心-辐射”制造战略的关键一环，旨在支持先进疗法的区域化生产，最终降低治疗成本、惠及更广泛患者。原文标题：CCRM, OmniaBio and Avectas Partner to Improve Cell Therapy Manufacturing 原文链接：https://www.prnewswire.com/news-releases/ccrm-omniabio-and-avectas-partner-to-improve-cell-therapy-manufacturing-302799943.html 04 AcCELLerated Biologic与Forever Labs就推进干细胞储存和再生医学解决方案达成战略合作伙伴关系 2026年6月9日，再生医学领域的美国领军企业AcCELLerated Biologics宣布与成人干细胞冷冻及储存服务企业Forever Labs达成深度战略合作，双方将整合各自技术与服务优势，面向全美临床医师与患者推出一体化自体生物制剂解决方案，覆盖干细胞采集、长期低温存储到后续个性化治疗应用的全流程。本次合作将AcCELLerated Biologics在富血小板血浆（PRP）、脂肪来源自体生物制剂等循证医疗产品领域的成熟经验，与Forever Labs的微创干细胞采集、合规低温存储技术深度结合，依托其在美国境内符合ISO认证、cGMP标准的存储设施，帮助患者在年轻状态下留存健康自体干细胞，为后续对抗衰老相关疾病、延长健康寿命储备医疗资源。合作将率先在佛罗里达州落地，依托当地最新出台的干细胞监管新政搭建合规服务体系，后续将逐步向全美推广医师培训、联合服务包等配套项目，推动个性化再生医疗服务的可及性提升。原文标题：AcCELLerated Biologics Announces Strategic Partnership with Forever Labs to Advance Stem Cell Storage and Regenerative Medicine Solutions 原文链接：https://www.prnewswire.com/news-releases/accellerated-biologics-announces-strategic-partnership-with-forever-labs-to-advance-stem-cell-storage-and-regenerative-medicine-solutions-302795562.html 05 全球首款实体瘤CAR-T疗法在我国上市 2026年6月22日，专注于开发创新CAR-T细胞疗法的生物制药公司科济药业收到国家药品监督管理局（NMPA）通知，批准其自主研发的Claudin18.2自体人源化CAR-T细胞治疗产品——恺力美®（舒瑞基奥仑赛注射液）新药上市申请，用于治疗Claudin18.2阳性、人表皮生长因子受体2（HER2）阴性，至少二线治疗失败的晚期胃/食管胃结合部腺癌。恺力美®是全球首款获批用于实体瘤治疗的CAR-T细胞治疗产品。6月23日，科济药业在投资人交流会中公布了恺力美的价格，为99万元/人份。 Claudin18.2是一种高选择性标记蛋白，在正常健康组织中表达十分有限，仅在已分化的胃粘膜上皮细胞中表达，而在胃癌等多种恶性肿瘤中呈现高表达。恺力美®是一款靶向Claudin18.2的自体CAR-T细胞产品，它是经基因修饰以表达由人源化Claudin18.2特异性单链单抗（hu8E5-2I）、CD8α铰链区、CD28跨膜区、CD28胞内信号域（CD28 ICD）及CD3ζ胞内信号区组成的CAR构建体。尽本公司所知，我们在全球范围内率先成功识别、验证并报告实体瘤相关抗原Claudin18.2作为CAR-T细胞疗法的有效靶点。为了进一步解决CAR-T细胞疗法治疗实体瘤肿瘤微环境的挑战，本公司针对恺力美®自主研发了一种受专利保护的创新性清淋预处理方案，特点是在传统方案（环磷酰胺和氟达拉滨）基础上加入低剂量的白蛋白结合型紫杉醇以增强CAR-T细胞的渗透和抗肿瘤效果。本公司围绕恺力美®进行全球专利布局，覆盖靶点、适应症、给药剂量和预处理方案等。恺力美®的临床疗效得到了国际顶级医学期刊的权威认可，其确证性随机对照研究结果发表于The Lancet期刊。临床数据显示，对于治疗选择极其有限、预后极差的晚期末线胃/食管胃结合部癌患者，恺力美®展现出较现有治疗显著的疗效获益和良好的安全性，为晚期胃癌患者带来了全新的治疗选择。这一重大突破不仅确立了实体瘤CAR-T治疗的新标准，更为后续推进早线治疗、联合治疗方案探索，以及拓展应用于胰腺癌、胆道癌等其他Claudin18.2阳性实体瘤奠定了坚实的科学基础。原文标题：科济药业全球首款治疗实体瘤的CAR-T细胞治疗产品恺力美®获批上市原文链接：https://mp.weixin.qq.com/s/SUslqNnTFJvC1mrt-wZhbQ 06 UpDoc推出首个获得FDA批准的临床人工智能平台 2026年6月25日，专注于开发医生级智能体的企业级临床AI公司UpDoc宣布，其首个采用面向患者的大型语言模型的医疗软件即设备（SaMD）已获得FDA批准。结合在全美四家领先医疗机构的初步部署，该公司重新定义并推进了快速发展的新兴医疗服务领域——临床AI。临床AI是指能够提供以往需要执业临床医生才能提供的医疗服务的AI。在“AI医生”竞相涌入市场且缺乏严格审查的当下，UpDoc作为首个获得FDA批准的智能体临床AI平台横空出世，旨在辅助医生，而非取代医生。这是一款专为临床应用而设计的操作系统，直接嵌入医疗服务提供者的工作流程中。该平台的核心是UpDoc临床智能，这是一种专有的编排和安全架构，它整合了临床指南、患者的长期病史、医生管理和电子病历数据，从而能够大规模地安全协调和执行真实的临床诊疗。该平台能够自动完成以往需要医生直接参与的干预措施，例如调整药物、开具化验单、协调医疗团队等。原文标题：UpDoc Debuts First FDA-Cleared Clinical AI Platform Built for Real-Time Patient Care Delivery and Intelligent Care Coordination 原文链接：https://www.prnewswire.com/news-releases/updoc-debuts-first-fda-cleared-clinical-ai-platform-built-for-real-time-patient-care-delivery-and-intelligent-care-coordination-302810065.html 07 FesariusTherapeutics完成2000万美元A轮融资推进水凝胶真皮再生模板覆盖全美医院系统 2026年6月10日，致力于革新皮肤修复技术的商业化阶段医疗科技公司Fesarius Therapeutics宣布完成超额认购的2000万美元A轮融资。本轮由Jefferson Life Sciences领投，强生创新（JJDC）、纽约州风险投资机构NY Ventures及美国整形外科医师协会（ASPS）首次以机构投资者身份跟投。公司核心产品DermiSphere™为美国市场首款水凝胶真皮再生模板（hDRT），已于2025年获FDA批准用于多种创面管理。该产品采用新型双密度胶原微结构，旨在加速细胞浸润和血管化，促进自然愈合。此轮资金将用于扩大直销团队以覆盖全美医院系统，推进“突破性设备认定”及拓宽报销路径，并启动OneStep手术临床试验。该术式将DermiSphere™与皮移植在同一次手术中完成，有望免除当前标准疗法所需的二次手术，减少患者康复时间、疼痛及总医疗成本。美国真皮再生市场规模达16亿美元，但近30年缺乏实质创新，Fesarius Therapeutics的临床和商业动能正加速获得顶级战略投资者认可。原文标题：FesariusTherapeutics Closes Oversubscribed $20M Series A, Led by Jefferson Life Sciences, and Backed by NY Ventures, and the American Society of Plastic Surgeons 原文链接：https://www.prnewswire.com/news-releases/fesariustherapeutics-closes-oversubscribed-20m-series-a-led-by-jefferson-life-sciences-and-backed-by-ny-ventures-and-the-american-society-of-plastic-surgeons-302795892.html 08 xCures完成4600万美元B轮融资推进分散医疗记录转化为可用于决策的数据 2026年6月24日，专注于改善医疗效果的创新型医疗数据公司xCures宣布完成4600万美元B轮融资，本轮融资由Innovius Capital领投，iGrow、GKCC、Spring Mountain Capital及现有投资者跟投。此次融资使xCures的总融资额超过7600万美元，并将用于支持其临床清晰层（clinical clear layer）的持续扩展，以将分散的医疗记录转化为可用于决策的临床数据。患者信息分散在数千个实验室、医院、影像中心和电子病历系统中，通常以非结构化文档的形式存在，难以在临床工作流程中使用。xCures临床清晰度引擎（Clinical Clarity Engine）整合了三大功能：基于自动化患者病史构建的决策就绪清单，且以循证数据为支撑。它为医疗服务提供者、价值医疗机构、诊断实验室和消费者健康组织提供了他们长期以来所需要的清晰度。迄今为止，xCures已处理了来自全美超过55万家医疗机构的3亿多份医疗记录，为美国数百万患者的临床决策提供支持。原文标题：xCures Closes $46 Million Series B Financing to Transform Fragmented Medical Records into Decision-ready Data through its Clinical Clarity Engine 原文链接：https://www.prnewswire.com/news-releases/xcures-closes-46-million-series-b-financing-to-transform-fragmented-medical-records-into-decision-ready-data-through-its-clinical-clarity-engine-302809282.html 09 Syntax Bio扩大A轮融资规模用于推进Cellgorithm™平台 2026年6月21日，致力于研发下一代细胞疗法的合成生物学公司Syntax Bio宣布扩大A轮融资规模。此次融资使Syntax Bio的A轮融资总额达到1440万美元，总融资额超过2500万美元。此轮融资在原有投资机构 Astellas Venture Management、Illumina Ventures、DCVC Bio、Civilization Ventures、EGB Capital、Mansueto Office 和 Portal Innovations 的支持基础上，新增了 Draper Associates、Allegis Capital、LongGame、Mayo Clinic、伊利诺伊州商业和经济机会部、Illinois Ventures、Exit Fund、Sigma Group 和 Walder Ventures 等投资机构。募集资金将用于进一步推进公司专有的Cellgorithm™平台，并资助针对1型糖尿病的胰岛β细胞疗法的临床前概念验证研究。Cellgorithm™平台采用基于CRISPR的系统来编程和加速干细胞分化，从而变革再生医学的发现、开发和生产方式。 Syntax Bio同时宣布了公司高管团队、董事会和科学顾问委员会的领导层任命。联合创始人Ryan Clarke博士已从首席技术官转任首席科学官，他将继续领导公司的科学战略和干细胞疗法创新。另一位联合创始人Nikolas Balanis博士晋升为首席技术官，将负责公司合成生物学平台和人工智能驱动的计算生物学能力的持续发展。原文标题：Syntax Bio Announces Expanded Series A and Leadership Appointments 原文链接：https://www.businesswire.com/news/home/20260621148785/en/Syntax-Bio-Announces-Expanded-Series-A-and-Leadership-Appointments 10 西湖生物完成超亿元融资加速全球首创红细胞药物临床开发 2026年6月18日讯，西湖生物医药科技有限公司（以下简称“西湖生物”）宣布完成新一轮超亿元融资。本轮融资由北京市石景山区现代创新产业发展基金有限公司与瑞力合成生物基金联合领投，知名产业投资人参与跟投，倚锋资本、华方资本、红杉中国等老股东持续追加投资。本轮融资款项将主要用于推进公司红细胞药物平台在肿瘤、自身免疫等治疗领域的管线开发，加速核心产品的临床验证与注册申报，并进一步加强研发、生产和产业化能力建设。西湖生物成立于2019年，是西湖大学首家由科研成果转化设立的生物科技创新企业，专注于工程化红细胞药物研发与产业化。公司聚焦红细胞与免疫系统之间的天然互作关系，利用红细胞归巢脾脏并参与免疫调控的生物学特性，开发新一代免疫红细胞药物。围绕这一方向，公司建立了全球首个成体红细胞–药物偶联技术平台REDx，可支持抗体、核酸、小分子、酶等多类型治疗载荷与红细胞稳定偶联，并已布局肿瘤、自身免疫、代谢性疾病及核酸递送等多个创新管线。公司有2条核心管线：（1）全球首个红细胞-抗PD-1抗体偶联药物WTX-212 在研究者发起的首次人体临床研究中，WTX-212在PD-1/PD-L1耐药实体瘤患者中显示出良好的安全性和显著抗肿瘤活性，实现全球首例红细胞药物治疗实体瘤患者完全缓解，并观察到持续生存获益。WTX-212计划于2026年递交中美IND申请，并已启动异体红细胞版本的临床探索。（2）红细胞-mRNA-LNP偶联药物WTX-101 WTX-101通过利用红细胞与脾脏免疫系统的天然互作机制，重塑mRNA药物在体内的分布与递送路径，实现对脾脏及髓系免疫细胞的高效靶向递送，并降低传统LNP系统相关肝脏暴露和系统性炎症反应风险。基于该平台，公司已实现体内CAR-免疫细胞生成，为肿瘤和自身免疫疾病治疗提供新的技术路径。原文标题：西湖生物完成超亿元融资，加速全球首创红细胞药物临床开发原文链接：https://mp.weixin.qq.com/s/ni5DidgfFzVA01mArVhFEg 点击关注关注醉心科学聚焦干细胞领域研究进展合作交流扫码填写表单选择合作意向系列沙龙扫码填写表单获得参会名额

2026-06-30

·BioSpace

Monopar Therapeutics Receives FDA Rare Pediatric Disease Designation for ALXN1840 for the Treatment of Wilson Disease

WILMETTE, Ill., June 30, 2026 (GLOBE NEWSWIRE) -- Monopar Therapeutics Inc. (“Monopar” or the “Company”) (Nasdaq: MNPR), a clinical-stage biopharmaceutical company developing innovative treatments for patients with unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to ALXN1840 (tiomolibdate choline, TMC), the Company’s late-stage candidate for the treatment of Wilson disease. The FDA grants RPD designation to therapies intended to treat serious or life-threatening diseases that primarily affect children from birth to 18 years of age. The designation provides the Company with the potential at the time of NDA approval to receive a pediatric Priority Review Voucher (PRV), which can be used to obtain priority review of a subsequent marketing application or sold or transferred to another sponsor. Priority review can reduce the FDA’s target review time by several months. “Receiving Rare Pediatric Disease designation for ALXN1840 underscores the serious impact that Wilson disease has on patients and reinforces the urgency of bringing forward a new treatment option,” said Chandler Robinson, M.D., Chief Executive Officer of Monopar. About Wilson Disease Wilson disease is a rare genetic disorder that affects approximately 1 in 30,000 people worldwide. It is caused by mutations in the ATP7B gene, which impairs the body’s ability to excrete copper. It is characterized by toxic accumulation of copper in the liver, brain, and other organs, leading to progressive and potentially fatal outcomes if untreated. About ALXN1840 ALXN1840 (tiomolibdate choline, TMC) is a novel first-in-class albumin tripartite complex (ATC) activator under investigation for the treatment of Wilson disease. ALXN1840 rapidly mobilizes and tightly sequesters excess copper in ATCs, suppressing its redox reactivity, limiting oxidative damage, and blocking transport across the blood–brain barrier. Clinical data demonstrate that ALXN1840 improves copper balance by increasing fecal copper excretion. In the Phase 3 pivotal trial, ALXN1840 met the primary endpoint by demonstrating rapid and sustained copper mobilization significantly greater than standard of care over 48 weeks in both previously treated and untreated patients. Durable clinical improvement and a favorable safety and tolerability pro observed across 645 patient-years of follow-up in 266 patients. ALXN1840 is an oral tablet with a once-a-day dosing regimen. About Monopar Therapeutics Inc. Monopar Therapeutics is a clinical-stage biopharmaceutical company with late-stage ALXN1840 for Wilson disease, and radiopharmaceutical programs including MNPR-101-Zr (Phase 1) for imaging advanced cancers along with MNPR-101-Lu (Phase 1a) and MNPR-101-Ac (late preclinical) for the treatment of advanced cancers. For more information, visit: . Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. An example of a forward-looking statement includes the statement concerning: that the designation (RPD) provides the Company with the potential at the time of (ALXN1840) NDA approval to receive a pediatric Priority Review Voucher (PRV). The forward-looking statements involve risks and uncertainties including, but not limited to: uncertainties related to whether the ALXN1840 marketing application will receive marketing approval and, if approved, whether Monopar will be awarded a Priority Review Voucher; whether, if awarded, the Priority Review Voucher can be used to obtain priority review of a subsequent marketing application or sold or transferred to another sponsor; the continued authorization and availability of the Rare Pediatric Disease Priority Review Voucher program; uncertainties related to the regulatory process that Monopar intends to initiate related to ALXN1840 and the outcome thereof; the rate of market acceptance and competitiveness in terms of pricing, efficacy and safety, of any products for which Monopar receives marketing approval, and Monopar’s ability to competitively market any such products as compared to larger pharmaceutical firms; Monopar’s ability to raise sufficient funds in order for the Company to support continued preclinical, clinical, regulatory, precommercial and commercial development of its programs and to make contractual milestone payments, as well as its ability to further raise additional funds in the future to support any existing or future product candidate programs through completion of clinical trials, the approval processes and, if applicable, commercialization; and the significant general risks and uncertainties surrounding the research, development, regulatory approval, and commercialization of imaging agents and therapeutics. Actual results may differ materially from those expressed or implied by such forward-looking statements. Risks are described more fully in Monopar’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Monopar undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Any forward-looking statements contained in this press release represent Monopar’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. CONTACT: Monopar Therapeutics Inc. Investor Relations Quan Vu Chief Financial Officer vu@monopartx.com Follow Monopar on social media for updates: X: @MonoparTx LinkedIn: Monopar Therapeutics Source: Monopar Therapeutics Inc.

临床结果优先审批临床3期临床1期

2026-06-26

·ir.monopartx.com

Monopar Presents New Analyses of Phase 3 FoCus Data at EAN 2026 Showing Greater Neurologic and Global Clinical Benefit with ALXN1840 Versus Standard of Care in Wilson Disease

WILMETTE, Ill., June 26, 2026 (GLOBE NEWSWIRE) -- Monopar Therapeutics Inc. (“Monopar” or the “Company”) (Nasdaq: MNPR), a clinical-stage biopharmaceutical company developing innovative treatments for patients with unmet medical needs, today announced that new analyses from the Phase 3 FoCus randomized controlled clinical trial of ALXN1840 (tiomolibdate choline, TMC) will be presented at the 12th Congress of the European Academy of Neurology (EAN 2026), June 27–30, 2026, in Geneva, Switzerland. These analyses build upon the previously reported Phase 3 FoCus results demonstrating ALXN1840 met its primary endpoint of superior copper mobilization versus standard of care. In an encore poster presentation titled “Greater clinical benefit with tiomolibdate choline versus standard of care in neurologic Wilson disease patients in the Phase 3 FoCus Trial,” Aurélia Poujois, MD, PhD, Department of Neurology, Adolphe de Rothschild Foundation Hospital, Paris, France, will present results showing that ALXN1840 produced significant neurologic improvement over time and greater global clinical improvement compared to standard-of-care (SoC) therapy in Wilson disease (WD) patients with neurologic symptoms at baseline. ALXN1840 also demonstrated similar or better outcomes compared to SoC across a range of psychiatric and hepatic measures during the 48-week study. Key findings presented at EAN 2026: In the subset of patients with neurologic symptoms at baseline from the 2:1 randomized Phase 3 FoCus clinical trial (NCT03403205; n=207), ALXN1840 demonstrated improved outcomes compared to SoC across multiple clinical measures: Neurologic improvement on the rater-blinded, physician-assessed Unified Wilson Disease Rating Scale (UWDRS) Part III was significant and continued over time with ALXN1840 (p=0.006) but not with SoC (p=0.435). Global clinical improvement as assessed by the Clinical Global Impressions – Improvement (CGI-I) scale at Week 48 was significantly greater with ALXN1840 than with SoC (p<0.001). A greater proportion of patients treated with ALXN1840 achieved improvement on the rater-blinded UWDRS Part III at Week 48 compared with SoC, with consistent results observed across multiple improvement thresholds. ALXN1840 also produced similar or greater improvement than SoC at Week 48 across psychiatric and hepatic measures. Across Phase 2 and Phase 3 studies, ALXN1840 has demonstrated a well-characterized and favorable safety profile in 266 patients, with a median treatment duration of 2.58 years and maximum exposure of more than 8 years. Drug-related serious adverse events (SAEs) occurred in 4.9% of patients, including neurologic SAEs in less than 1% and no treatment-related deaths. “For Wilson disease patients with neurologic symptoms, meaningful improvement can be difficult to achieve with existing therapies, and some patients experience severe paradoxical worsening,” said Dr. Poujois. “These new analyses from the Phase 3 FoCus trial are encouraging because they show that ALXN1840 treatment was associated with continued neurologic improvement over time and greater global clinical benefit compared with standard of care.” The poster (link) is available on Monopar’s website. These findings further support Monopar’s planned New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA) for ALXN1840 in mid-2026. About Wilson Disease Wilson disease (WD) is a rare genetic disorder that affects approximately 1 in 30,000 people worldwide. It is caused by mutations in the ATP7B gene, which impairs the body’s ability to excrete copper. It is characterized by toxic accumulation of copper in the liver, brain, and other organs, leading to progressive and potentially fatal outcomes if untreated. About ALXN1840 ALXN1840 (tiomolibdate choline, TMC) is a novel first-in-class albumin tripartite complex (ATC) activator under investigation for the treatment of Wilson disease. ALXN1840 rapidly mobilizes and tightly sequesters excess copper in ATCs, suppressing its redox reactivity, limiting oxidative damage, and blocking transport across the blood–brain barrier. Clinical data demonstrate that ALXN1840 improves copper balance by increasing fecal copper excretion. In the Phase 3 pivotal trial, ALXN1840 met the primary endpoint by demonstrating rapid and sustained copper mobilization significantly greater than standard of care over 48 weeks in both previously treated and untreated patients. Durable clinical improvement and a favorable safety and tolerability profile were observed across 645 patient-years of follow-up in 266 patients. About Monopar Therapeutics Inc. Monopar Therapeutics is a clinical-stage biopharmaceutical company with late-stage ALXN1840 for Wilson disease, and radiopharmaceutical programs including MNPR-101-Zr (Phase 1) for imaging advanced cancers along with MNPR-101-Lu (Phase 1a) and MNPR-101-Ac (late preclinical) for the treatment of advanced cancers. For more information, visit: www.monopartx.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. An example of a forward-looking statement includes the statement concerning: that these findings (the Phase 3 FoCus data) further support Monopar’s planned New Drug Application (NDA) submission to the FDA for ALXN1840 in mid-2026. The forward-looking statements involve risks and uncertainties including, but not limited to: uncertainties related to the regulatory process that Monopar intends to initiate related to ALXN1840 and the outcome thereof; the rate of market acceptance and competitiveness in terms of pricing, efficacy and safety, of any products for which Monopar receives marketing approval, and Monopar’s ability to competitively market any such products as compared to larger pharmaceutical firms; Monopar’s ability to raise sufficient funds in order for the Company to support continued preclinical, clinical, regulatory, precommercial and commercial development of its programs and to make contractual milestone payments, as well as its ability to further raise additional funds in the future to support any existing or future product candidate programs through completion of clinical trials, the approval processes and, if applicable, commercialization; and the significant general risks and uncertainties surrounding the research, development, regulatory approval, and commercialization of imaging agents and therapeutics. Actual results may differ materially from those expressed or implied by such forward-looking statements. Risks are described more fully in Monopar’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Monopar undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Any forward-looking statements contained in this press release represent Monopar’s views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. CONTACT: Monopar Therapeutics Inc. Investor Relations Quan Vu Chief Financial Officer vu@monopartx.com Follow Monopar on social media for updates: X: @MonoparTx LinkedIn: Monopar Therapeutics Source: Monopar Therapeutics Inc. Released June 26, 2026

临床结果临床3期临床1期申请上市临床2期

100 项与四硫钼酸二胆碱相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
胆汁性肝硬化	申请上市	美国	AstraZeneca PLC	-
肝豆状核变性	临床3期	中国	Alexion Pharmaceuticals, Inc.	2018-02-15
肝豆状核变性	临床3期	比利时	Alexion Pharmaceuticals, Inc.	2018-02-15
肝豆状核变性	临床3期	俄罗斯	Alexion Pharmaceuticals, Inc.	2018-02-15
非转移性前列腺癌	临床2期	美国	Attenuon, LLC	2006-11-01
局部晚期黑色素瘤	临床2期	美国	Attenuon, LLC	2006-09-01
浆细胞白血病	临床2期	美国	Attenuon, LLC	2006-06-01
骨髓增生异常综合征	临床1期	美国	Attenuon, LLC	2006-12-30
肌萎缩侧索硬化	临床前	瑞典	Immune Disease Institute, Inc.	-

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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


Company_Website 人工标引	N/A	肝豆状核变性	-	ALXN1840 (tiomolybdate choline)	觸範網蓋膚網餘鏇艱糧(築壓獵夢膚鏇鹹積餘膚) = 構夢遞製糧襯遞夢觸簾衊繭憲網蓋構簾構鹹餘 (鏇遞願積願簾繭觸衊艱 ) 更多	积极	2025-05-13
NCT03403205 (FoCus, MDS2024)	临床3期	肝豆状核变性	214	TMC	鹹醖衊範夢鹽衊簾遞醖(簾鬱鬱鏇願鏇繭範簾構) = 顧襯製繭鑰餘鹹簾顧選壓願簾壓襯築襯鹹獵築 (構醖鹹願構積鏇窪積製 ) 更多	积极	2024-09-27
				SoC	鹹醖衊範夢鹽衊簾遞醖(簾鬱鬱鏇願鏇繭範簾構) = 衊糧網繭膚襯襯顧膚鹽壓願簾壓襯築襯鹹獵築 (構醖鹹願構積鏇窪積製 ) 更多
NCT04594252 (CTgov)	临床1期	-	17	ALXN1840 (Group 1: ALXN1840)	廠廠積鹹構鬱選糧淵積(築鏇製鏇壓餘網構衊鬱) = 網齋膚衊積憲積淵壓膚網夢鏇繭範膚夢醖製壓 (壓網襯廠顧鹹衊齋憲鏇, 0.37419) 更多	-	2024-08-19
				ALXN1840 (Group 2: ALXN1840)	廠廠積鹹構鬱選糧淵積(築鏇製鏇壓餘網構衊鬱) = 壓壓構鬱淵廠夢窪襯鏇網夢鏇繭範膚夢醖製壓 (壓網襯廠顧鹹衊齋憲鏇, 0.48176) 更多
NCT04610580 (CTgov)	临床1期	-	48	ALXN1840 (Crossover: ALXN1840 Test Formulation (Treatment A))	醖醖廠顧顧衊簾壓夢網(夢襯淵膚淵餘簾餘衊網) = 遞積顧鑰觸積製艱網鹹鑰襯夢簾積獵衊築遞糧 (齋襯夢選鏇顧襯顧夢鹹, 47.5) 更多	-	2024-01-16
				ALXN1840 (Crossover: ALXN1840 Reference Formulation (Treatment B))	醖醖廠顧顧衊簾壓夢網(夢襯淵膚淵餘簾餘衊網) = 繭鏇獵願艱積獵蓋壓膚鑰襯夢簾積獵衊築遞糧 (齋襯夢選鏇顧襯顧夢鹹, 30.8) 更多
NCT05641311 (CTgov)	临床1期	肝豆状核变性	24	ALXN1840 (Cohort 1: Japanese Participants)	衊糧遞鹹製衊鏇簾糧膚(網夢築壓醖繭膚築窪築) = 網齋繭夢觸獵餘窪鬱襯蓋鹹鏇願鹽糧鬱醖遞鹽 (積壓鏇構夢鏇齋鬱範網, 1427.41833) 更多	-	2023-11-07
				ALXN1840 (Cohort 2: Non-Japanese Participants)	衊糧遞鹹製衊鏇簾糧膚(網夢築壓醖繭膚築窪築) = 膚鏇鏇壓鹹製齋糧鏇壓蓋鹹鏇願鹽糧鬱醖遞鹽 (積壓鏇構夢鏇齋鬱範網, 3413.75213) 更多
NCT04573309 (CTgov)	临床2期	肝豆状核变性	9	ALXN1840 (Cohort 1)	醖憲鑰構餘獵夢鏇觸鬱(憲膚壓襯衊簾糧餘積糧) = 壓製壓觸願夢積鹽夢廠遞襯膚夢繭襯餘鏇膚淵 (鹽製積襯顧鹹鹽鑰遞廠, 0.26860) 更多	-	2023-10-27
				ALXN1840 (Cohort 2)	醖憲鑰構餘獵夢鏇觸鬱(憲膚壓襯衊簾糧餘積糧) = 鑰鹽廠網醖糧鏇選願衊遞襯膚夢繭襯餘鏇膚淵 (鹽製積襯顧鹹鹽鑰遞廠, NA) 更多
NCT04526210 (CTgov)	临床1期	肝豆状核变性	54	Bupropion HCl (Treatment A: Bupropion HCl)	糧壓醖構網繭顧鹽膚鏇(顧製製憲艱簾醖積繭構) = 繭簾壓範衊糧範願築廠齋觸襯淵選網齋願廠廠 (選鏇蓋糧顧憲壓淵廠製, 31.6) 更多	-	2023-08-31
				ALXN1840+Bupropion HCl (Treatment B: Bupropion HCl + ALXN1840)	糧壓醖構網繭顧鹽膚鏇(顧製製憲艱簾醖積繭構) = 築襯鑰艱鏇醖網餘鑰選齋觸襯淵選網齋願廠廠 (選鏇蓋糧顧憲壓淵廠製, 27.9) 更多
NCT04526197 (CTgov)	临床1期	肝豆状核变性	36	celecoxib (Treatment A)	願簾蓋獵齋衊獵夢顧構(壓鬱製鹹積範願鬱齋鏇) = 遞壓齋願選夢淵鏇糧鹽壓鬱艱膚艱鹹齋築糧願 (醖鑰艱遞鏇醖構鑰廠糧, 48.7) 更多	-	2023-08-21
				ALXN1840+celecoxib (Treatment B)	願簾蓋獵齋衊獵夢顧構(壓鬱製鹹積範願鬱齋鏇) = 選製鑰餘顧製積壓構廠壓鬱艱膚艱鹹齋築糧願 (醖鑰艱遞鏇醖構鑰廠糧, 49.4) 更多
NCT05303324 (CTgov)	临床1期	-	48	ALXN1840 (ALXN1840 Treatment A)	製膚壓獵範憲繭獵淵鏇(鹽選積夢餘衊遞鬱餘積) = 鹹廠鬱窪艱窪鑰餘範遞繭窪餘獵製憲壓鬱顧夢 (遞選築醖艱齋願衊夢窪, 78.098) 更多	-	2023-08-02
				ALXN1840 (ALXN1840 Treatment B)	製膚壓獵範憲繭獵淵鏇(鹽選積夢餘衊遞鬱餘積) = 製壓醖壓夢繭繭蓋選鹽繭窪餘獵製憲壓鬱顧夢 (遞選築醖艱齋願衊夢窪, 62.956) 更多
NCT05319912 (CTgov)	临床1期	-	18	ALXN1840 (Treatment A: ALXN1840 (Fasted))	鑰醖繭膚壓膚壓餘夢製(遞膚窪壓選齋齋鬱夢簾) = 製製餘襯積衊顧鑰觸簾製壓構醖壓顧齋鹹獵願 (壓膚製遞觸襯構膚憲淵, 5635) 更多	-	2023-08-02
				ALXN1840 (Treatment B: ALXN1840 (Fed))	鑰醖繭膚壓膚壓餘夢製(遞膚窪壓選齋齋鬱夢簾) = 觸餘廠糧獵窪製鏇襯膚製壓構醖壓顧齋鹹獵願 (壓膚製遞觸襯構膚憲淵, 4681) 更多