Bis-choline tetrathiomolybdate

本期快讯摘要 临床方面，Arrowhead Pharmaceuticals Redemplo在严重高甘油三酯血症治疗中展现卓越疗效，阿斯利康冲击其2030年800亿美元销售目标；公司动态方面，AI drug discovery AI制药企业与药企合作中数据归属成关键谈判焦点，Crystalys Therapeutics Crystalys完成1.3亿美元B轮融资；FDA获批方面，Revolution Medicines 用于治疗既往治疗失败的转移性胰腺癌，Monopar 启动ALXN1840治疗威尔逊病的NDA滚动申报；监管政策方面，美国NIH加入特朗普AI国家计划。本文多维度解读全球生物医药产业新变化，助力读者高效洞察行业趋势。 01 专题板块临床试验 4 条 01Arrowhead Pharmaceuticals 阶段3试验数据亮眼，Redemplo（plozasiran）在严重高甘油三酯血症治疗中展现卓越疗效 Arrowhead Pharmaceuticals 在其针对严重高甘油三酯血症（sHTG）的两项 III 期临床试验 SHASTA-3 和 SHASTA-4 中取得了积极结果，Redemplo（plozasiran）在约 750 名患者中实现了所有主要和次要预设终点。试验数据显示，与安慰剂相比，Redemplo 显著降低了 78% 的胰腺炎发生率，其中高风险患者（甘油三酯水平高于 880 mg/dL 且有急性胰腺炎病史）实现 100% 的胰腺炎减少。此外，在第 12 个月时，患者的甘油三酯中位数下降了 79% 和 81%，远超安慰剂组的 27%。Arrowhead 表示计划于今年内向 FDA 提交 Redemplo 治疗 sHTG 的上市申请，并在其他地区跟进。 Arrowhead PharmaceuticalsRedemploplozasiran严重高甘油三酯血症siRNA 02阿斯利康Wainua III期临床失利，2030年800亿美元销售目标承压 阿斯利康（AstraZeneca）在罕见病药物Wainua的III期临床试验中遭遇失败，该药物原本被寄予厚望用于扩展其在转甲状腺素蛋白淀粉样变性心肌病（ATTR-CM）的适应症。Wainua于2023年12月获得FDA批准用于治疗ATTR多发性神经病（polyneuropathy），但其2025年销售额仅为2.93亿美元，远低于此前预测的50亿美元峰值。此次失败削弱了公司2030年实现800亿美元销售目标的路径，需依赖SERENA-4和AVANZAR两项高风险临床试验的成功。其中，SERENA-4评估口服SERD camizestrant在HR阳性乳腺癌一线治疗中的表现，而AVANZAR则测试抗体药物偶联物（ADC）Datroway在非小细胞肺癌一线治疗中的疗效。Leerink分析师认为这两项试验的成功概率分别为20%和50%。若失败，阿斯利康的销售预期将面临进一步下调。 阿斯利康WainuaATTR-CM临床试验 03LB Pharmaceuticals宣布加速其精神分裂症3期NOVA-2试验顶线数据披露 LB Pharmaceuticals近日宣布，基于患者入组速度超出预期，其3期临床试验NOVA-2的顶线结果将提前至2027年上半年披露，并计划于2027年下半年与FDA召开pre-NDA会议。NOVA-2是一项多中心、随机、双盲、安慰剂对照试验，评估LB-102在精神分裂症患者中的疗效和安全性，计划在美国25个中心入组约460名患者。LB-102是一种新型口服小分子药物，为阿米舒必利的甲基化衍生物，具有D2、D3和5HT-7受体强效选择性拮抗作用，潜在治疗精神分裂症、双相情感障碍和重度抑郁症（MDD）等神经精神疾病。此前的2期试验中，LB-102在精神分裂症急性期治疗中展现出快速起效、持续疗效及优于现有药物的安全性，尤其在减少运动障碍（EPS）和胃肠道副作用方面表现突出。该药物若成功获批，有望成为美国首个苯甲酰胺类抗精神病药物，为精神疾病治疗提供新选择。 LB PharmaceuticalsLB-102精神分裂症3期临床试验 04Healx 开展 HLX-4310 一期/二期临床试验，探索骨肉瘤转移复发预防新策略 Healx 宣布其临床阶段候选药物 HLX-4310 在针对复发性、难治性肉瘤（以骨肉瘤为重点）的 Phase 1/2 临床试验中已给药第一例患者。HLX-4310 是一种口服联合疗法，包含一种 I 类选择性 HDAC 抑制剂和一种 mTOR 抑制剂，旨在通过靶向微转移肿瘤的存活机制，降低癌症复发风险。骨肉瘤是一种侵袭性强的骨癌，主要影响儿童、青少年和年轻人，转移性骨肉瘤患者的五年生存率低于 30%，近 40 年来治疗进展有限。Healx 基于其 AI 驱动的药物发现平台，提出了一种新的治疗范式，即通过干预癌症复发的生物学机制，而非仅针对原发肿瘤。此次试验由美国国家儿科癌症基金会（NPCF）旗下 Sunshine Project™ 支持，汇集了多个顶尖儿科癌症中心，旨在加速该疗法的临床转化。该临床试验的启动标志着 Healx 在罕见肿瘤领域“预防转移复发”战略的重要进展，具有潜在的广泛癌症治疗应用前景。 HealxHLX-4310骨肉瘤AI drug discovery 02 专题板块公司动态 3 条 01AI制药企业与药企合作中数据归属成关键谈判焦点 在药物研发日益复杂的背景下，AI制药企业与大型药企之间的合作中，数据的生成、使用和归属问题已成为交易谈判中的关键难点。AI模型需要大量高质量数据来优化药物靶点筛选，而药企则希望对合作中产生的数据保持严格控制。Astellas、Bayer等药企的高管在BIO国际大会上表示，数据来源（如使用哪家化学实验室、数据生成地点等）正成为交易条款中的核心考量。Xaira Therapeutics等AI驱动的生物技术公司则希望通过合作获取新的数据集以提升平台能力。Lilly、Merck、BMS等头部药企也正在积极布局AI药物发现技术，并通过数据共享机制（如Lilly的TuneLab）与AI企业建立合作关系。此类数据驱动型合作模式的兴起，标志着AI制药正从概念走向实际落地，同时也对合作双方的法律与商业策略提出更高要求。 AI drug discovery数据归属LillyAstellasBayer 02Crystalys完成1.3亿美元B轮融资，加速推进痛风药物dotinurad的后期临床试验 Crystalys Therapeutics近日宣布完成1.3亿美元的B轮融资，由Frazier Life Sciences领投，Wellington Management、Cormorant Asset Management等多家知名机构参与。该融资将用于支持dotinurad的全球后期临床试验及商业化准备工作。dotinurad是一种靶向尿酸转运蛋白URAT1的小分子药物，已被日本批准用于痛风治疗，并在中国、菲律宾和泰国获得上市许可。Crystalys于2024年从Fortress Bio子公司获得该药在美国和欧洲的开发及商业化权利。目前，dotinurad正在进行两项全球性试验，分别针对严重痛风患者和尿酸水平过高的患者，与传统药物allopurinol进行24周的头对头比较，预计明年将公布结果。此外，公司还启动了一项II期临床试验，针对无法耐受allopurinol或对其反应不佳的患者。 Crystalys TherapeuticsdotinuradURAT1痛风 03Vertex以高溢价竞购Crinetics，成唯一竞标方 Vertex Pharmaceuticals已确认为唯一竞标方，参与对Crinetics Pharmaceuticals的高溢价收购。此次交易预计将成为Vertex历史上规模最大的一笔并购。分析师指出，Vertex在竞购过程中表现出的强烈兴趣和高溢价策略，可能引发投资者对其估值合理性的进一步质疑。Crinetics的核心资产包括其在内分泌疾病领域的多肽类药物管线，其中最引人关注的是其针对肢端肥大症的药物，目前处于临床后期阶段。该交易不仅反映了Vertex在内分泌疾病领域的战略扩张意图，也凸显了多肽药物在特定适应症中的研发价值。若交易最终完成，Vertex将进一步增强其在罕见病药物市场的竞争力，并可能推动相关多肽药物的商业化进程。 VertexCrinetics多肽收购 03 专题板块FDA 获批 2 条 01Revolution Medicines 向 FDA 提交 Daraxonrasib 新药申请，用于治疗既往治疗失败的转移性胰腺癌 Revolution Medicines 向美国 FDA 提交了 Daraxonrasib 的新药申请（NDA），用于治疗既往接受过治疗的转移性胰腺导管腺癌（PDAC）。该申请基于全球随机 III 期 RASolute 302 临床试验结果，试验显示 Daraxonrasib 在总生存期（OS）和无进展生存期（PFS）方面均取得显著改善。与传统化疗相比，Daraxonrasib 展现出可控的安全性，并显著延迟了患者癌症相关疼痛、总体健康状况和生活质量的恶化。该药物已获得 FDA 突破性疗法认定和孤儿药认定，并入选 FDA 委员会的国家优先凭证试点计划，以加速其审查。Daraxonrasib 是一款口服 RAS(ON) 多选择性抑制剂，旨在通过阻断 RAS 蛋白与其下游效应物的相互作用来抑制 RAS 信号传导，适用于多种 RAS 突变驱动的癌症。 Revolution MedicinesDaraxonrasibRAS(ON) 抑制剂胰腺癌 02Monopar启动ALXN1840治疗威尔逊病的NDA滚动申报 Monopar Therapeutics宣布已启动其候选药物ALXN1840（tiomolibdate choline, TMC）治疗威尔逊病的新药申请（NDA）滚动提交。ALXN1840是一种基于白蛋白三元复合物（ATC）机制的新型铜螯合剂，已在关键III期临床试验中达到主要终点，显示出比标准治疗更显著的铜动员效果，且在266例患者、累计645患者年的随访中表现出良好的耐受性和安全性。该药物已获得FDA的快速通道、孤儿药及罕见儿科疾病认定（RPD），若获批，将成为美国数十年来首个针对威尔逊病的全新作用机制疗法。此次NDA滚动提交标志着该药物向商业化迈出关键一步，具有重要的临床和商业价值。 MonoparALXN1840威尔逊病NDA 04 专题板块监管政策 1 条 01美国NIH加入特朗普AI国家计划，推动生物医学研究智能化转型 美国卫生与公共服务部（HHS）宣布将支持一项由总统特朗普发起的50亿美元AI驱动的Genesis Mission，重点推动生物医学研究的智能化发展，特别是慢性病、儿童癌症和药物发现领域。NIH将牵头实施“Bio Genesis Mission”，目标是构建一个智能化、未来导向的生物医学研究生态系统，结合AI和高性能计算，加速从科学发现到临床应用的转化进程。该计划还将推动自主实验室和AI模型的建设，以提升生物系统研究的可预测性。NIH表示将在未来十年内将科学发现到患者应用的时间缩短一半，并已规划超过12亿美元用于相关挑战领域。此外，美国国家科学基金会（NSF）也宣布投资4亿美元建立20个AI驱动的自主实验室节点，推动科研数据基础设施的升级。该举措与FDA和NIH近期推动减少动物实验、采用新方法学（NAMs）的政策方向一致，具有重要的行业导向意义。 NIHAI drug discovery监管政策自主实验室

WILMETTE, Ill., July 20, 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 the addition of seasoned commercial leaders to support the anticipated launch of the Company’s first commercial product, ALXN1840, pending U.S. Food and Drug Administration (FDA) approval. Nicole Sweeny, former Chief Commercial Officer of KalVista Pharmaceuticals, was elected to the Board of Directors at the Company’s Annual Meeting of Stockholders on June 22, 2026. In addition, Monopar has appointed Sharon Funk as Senior Vice President, Sales and Marketing, and Daniel Olmstead as Senior Vice President, Market Access, Distribution and Patient Services. ALXN1840 is a first-in-class Albumin Tripartite Complex (ATC) activator for the treatment of Wilson disease that has demonstrated clinical safety, efficacy and tolerability across a robust clinical development program. The Company plans to submit an FDA New Drug Application (NDA) for ALXN1840 in mid-2026. Nicole Sweeny ’s 20-plus years of commercial leadership and rare disease launch experience will provide valuable strategic guidance as Monopar prepares for the potential commercialization of ALXN1840. “I’m pleased to join the Monopar Board of Directors as the Company prepares for its next, important phase of growth,” said Ms. Sweeny. “Throughout my career, I’ve seen firsthand the importance of pairing scientific innovation with thoughtful commercial strategy and disciplined execution. I look forward to contributing my experience alongside my fellow directors and the management team as the Company advances its mission, navigates the opportunities ahead and works to create lasting value for patients and shareholders.” ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); Monopar also continued the build-out of its commercial organization with the appointments of Sharon Funk and Daniel Olmstead. Ms. Funk brings more than 20 years of biopharmaceutical commercial leadership experience, with a proven track record of successful scale up and product launches, most recently playing a key role in the commercialization of LUMRYZ at Avadel Pharmaceuticals, acquired by Alkermes Q1 2026. Mr. Olmstead brings more than 30 years of experience creating best-in-class market access, distribution and patient services strategies, most recently instrumental to the successful launches of novel therapies by Ardelyx and Akebia Therapeutics. “This is a pivotal time for Monopar as we advance ALXN1840 toward an NDA submission and prepare for a potential commercial launch,” said Susan Rodriguez, Chief Commercial and Strategy Officer of Monopar. “Sharon and Dan are highly experienced commercial leaders with a proven track record of successful launches of innovative therapies in the rare disease and specialty therapy space. I am also very pleased to welcome Nicole to the Monopar Board of Directors, who brings an invaluable commercial perspective to our Board and management team as we quickly advance our commercial readiness activities. These additions meaningfully deepen the commercial expertise at Monopar as we work to bring ALXN1840 to the Wilson disease community.” About Monopar Therapeutics Inc. Monopar is a clinical-stage biopharmaceutical company developing innovative treatments for patients with unmet medical needs. ALXN1840 is a first-in-class Albumin Tripartite Complex (ATC) activator for the treatment of Wilson disease in late-stage development. MNPR-101, a first-in-class targeted anti-uPAR (urokinase plasminogen activator receptor) antibody platform in Phase 1 development, is designed to enable targeted radiopharmaceutical imaging and therapy against aggressive solid tumors associated with multiple cancer types. For more information, visit and connect with us on X (formerly known as Twitter) and LinkedIn . 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. Examples of these forward-looking statements include statements concerning: Monopar’s plans to support the anticipated launch of the Company’s first commercial product, ALXN1840, pending U.S. Food and Drug Administration (FDA) approval; that the Company plans to submit an FDA New Drug Application (NDA) for ALXN1840 in mid-2026; that Monopar prepares for the potential commercialization of ALXN1840; that Monopar advances ALXN1840 toward an NDA submission and prepare[s] for a potential commercial launch; and that Monopar works to bring ALXN1840 to the Wilson disease community. The forward-looking statements involve risks and uncertainties including, but not limited to, the following: uncertainties related to the regulatory process that Monopar intends to initiate related to ALXN1840, including the submission of the NDA to the FDA, 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