Research Title：Adult and adolescent hemophilia patients treated with Marstacimab: a patient Experience Registry (AMBER) Protocol Number：94250855 Protocol Version Number：version 0.3 Date：15-November-2024 Leading site：Institution of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences Active Pharmaceutical Ingredient：a human IgG1 monoclonal antibody that targets the tissue factor pathway inhibitor (TFPI) Drug Name Generic name: marstacimab-hncq Purpose of the Study Primary Objectives:To quantify patient preferences for subcutaneous versus intravenous (IV) injection using the Marstacimab-Patient Preference Questionnaire (M-PPQ) after 1 month of marstacimab for routine prophylaxis treatment.
To assess the treatment burden using the Hemophilia Treatment Experience Measure (Hemo-TEM) in hemophilia patients after 6 months of marstacimab for routine prophylaxis treatment.
Exploratory Objectives:
To evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.
To assess changes in joint status scores between baseline and the final visit after 6 months of subcutaneous marstacimab injections in hemophilia patients, using the Hemophilia Early Arthropathy Detection with Ultrasound in China (HEAD-US-C).
To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months

开始日期2026-02-15

申办/合作机构-

NCT07161687

/ Active, not recruitingN/A

HYMPAVZI S.C. INJECTION 150 MG PEN SPECIAL INVESTIGATION

A study to evaluate the safety of Hympavzi under the actual use in patients with congenital hemophilia who do not have inhibitors.

开始日期2025-10-30

申办/合作机构

Pfizer Inc.

NCT06703606

/ Recruiting临床1期

A PHASE 1B OPEN-LABEL NON-RANDOMIZED STUDY TO ASSESS THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF MARSTACIMAB TREATMENT FOLLOWING THE DISCONTINUATION OF EMICIZUMAB THERAPY IN ADOLESCENT AND ADULT PARTICIPANTS WITH SEVERE HEMOPHILIA A WITHOUT INHIBITORS.

The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors. Hemophilia A is rare bleeding disorder where the blood doesn't clot normally. This causes a person to bleed a lot, even from a small cut.
These patients who are on emicizumab medicine for routine prophylaxis for at least 6 months, and desire to switch to marstacimab medicine. Inhibitors are antibodies that the immune system develops because it sees the infused clotting factor as a foreign substance that needs to be destroyed. Antibodies are proteins that eat up the activated factor before it has time to stop the bleeding. Prophylaxis are preventive medicines.
This study is seeking for participants:
* with severe Hemophilia A withouth inhibitors who are on emicizumab treatment for at least 6 months.
* must be 12 to less than 75 years old
* must have a body weight of at least 35 kilograms. The results from this study will serve as a guide to doctors and their hemophilia A patients who will change their medicines in the real-world clinical setting. Patients who can take part in the study will receive marstacimab medicine as weekly injections under the skin of 150 milligrams for 4 months. Study treatment with marstacimab will be initiated no earlier than 14 days after last dose of emicizumab. The study can last up to 6 months. The sponsor will provide marstacimab. Patients will continue their usual treatment with the infused clotting factor for their bleeds when taking part in the study. Roll-over into an optional study treatment extension period will be available to participants who wish to continue prophylaxis with marstacimab in countries where it is not commercially available.

开始日期2025-05-13

申办/合作机构

Pfizer Inc.

100 项与马塔西单抗相关的临床结果

登录后查看更多信息

100 项与马塔西单抗相关的转化医学

登录后查看更多信息

100 项与马塔西单抗相关的专利（医药）

登录后查看更多信息

项与马塔西单抗相关的文献（医药）

2026-12-31·mAbs

Discovery and optimization of marstacimab, a human monoclonal antibody targeting tissue factor pathway inhibitor for the treatment of hemophilia A and B

Article

作者: Narula, Jatin ; Jin, Macy ; Apgar, James R. ; Jasuja, Reema ; Holsti, Matthew ; Juo, Zong Sean ; Stahl, Mark ; Lin, Laura ; Webster, Robert ; Rakhe, Swapnil ; Tam, Amy ; Bloom, Laird ; Mosyak, Lidia ; Hett, Sunita ; Hurst, Susan ; Joyce, Alison ; Pittman, Debra D.

We report the discovery and optimization of marstacimab, a novel monoclonal antibody targeting tissue factor pathway inhibitor (TFPI), for the treatment of hemophilia A and B. Hybridoma and phage display approaches identified antibodies that blocked the TFPI: coagulation factor Xa (FXa) interaction. Antibodies bound TFPI with nanomolar affinity to multiple epitopes, including one that covered the entire FXa binding surface and others that partially overlapped the interface from different sides of the TFPI-K2 domain. Several antibodies reduced bleeding in a hemophilia A mouse injury model for up to 96 h following a single dose. Rabbit pharmacokinetic studies indicated that antibodies with ≤~1 nM TFPI-binding affinity were cleared rapidly from circulation, whereas TFPI-23 (5.72 nM) had a longer plasma residence time. Pharmacokinetic-pharmacodynamic modeling indicated that this intermediate affinity allowed circulating concentrations of antibodies such as TFPI-23 to maintain concentrations required for 50% residual activity; in contrast, higher-affinity antibodies quickly decreased to concentrations that would not effectively neutralize TFPI. The end-to-end process leading to final candidate selection incorporated rigorous assessment of biophysical properties, including thermal stability, aggregation propensity, viscosity, predicted immunogenicity, stable cell line productivity, and nonspecific binding. An optimized derivative of TFPI-23, TFPI-106 (PF-06741086, known as marstacimab), had the functional and biophysical properties with other characteristics suitable for clinical development and was selected as the final candidate. TFPI-106 elicited enhanced hemostasis in hemophilic plasma, shortened clotting time, and enhanced thrombin generation velocity. Marstacimab is now approved for patients with hemophilia A or B with or without inhibitors.

2026-06-01·Therapeutic Advances in Hematology

A plain language summary of the BASIS study looking at people living with severe hemophilia A or B with inhibitors and being treated with marstacimab

作者: Mahlangu, Johnny ; Raje, Sangeeta ; Matino, Davide ; Palladino, Andrew ; Gould, Travis ; Agathon, Delphine ; Acharya, Suchitra S. ; Taylor, Carrie Turich ; Sun, Pengling

Summary:

What is this summary about? This summary describes a clinical study looking at how well marstacimab works in people living with hemophilia with inhibitors. The study was published in the medical journal Blood . Hemophilia is caused by not having enough blood clotting factors, which are proteins found in the blood that help form a clot and stop bleeding. People living with hemophilia either don’t make these proteins, have low amounts of these proteins, or these proteins do not work well. Hemophilia is usually inherited from biological parents, meaning it is passed on to their children through genes. In some cases, hemophilia happens even if no one in the family has it. There are some treatments to replace the missing clotting factor. This type of treatment is called factor replacement therapy and contains the missing clotting factor proteins. Some people make antibodies against the replacement clotting factors, so the replacement clotting factors do not work as well as they need to for the blood to clot. This is called hemophilia with inhibitors.People who have hemophilia with inhibitors need to take medicines called bypassing agents instead of replacement clotting factors. These medicines aim to activate the body’s blood clotting system, but do not always work well. They also need to be injected into a vein, known as intravenous infusion, or IV infusion for short. IV infusions are taken several times a week.Different options to IV factor replacement therapy for people living with hemophilia A or hemophilia B are called non-factor replacement therapy. A protein in the blood called Tissue Factor Pathway Inhibitor, or TFPI, can also prevent blood clotting. Marstacimab is a type of non-factor replacement therapy that attaches to TFPI and stops its activity. For people living with hemophilia, marstacimab can help blood to form a clot when needed. Marstacimab is injected under the skin. This can be an easier way to receive medicine than an injection into a vein. Marstacimab offers a new way to treat people living with hemophilia.What were the results of the study?The study showed that people living with hemophilia with inhibitors who were treated with marstacimab had fewer bleeding events over 1 year compared to previous therapy with bypassing agents. Most side effects with marstacimab treatment were minor or could be easily managed. One person stopped taking marstacimab treatment because they developed a skin rash that got better over time.What do the results mean?The study showed that marstacimab could help prevent bleeding in people living with hemophilia A or B with inhibitors.

2026-05-01·Therapeutic Advances in Hematology

Effect of marstacimab in young people living with severe hemophilia A or moderate to severe hemophilia B without inhibitors: A plain language summary from the BASIS study

作者: Patel, Priya

Summary:

What is this summary about?This summary describes a study that looked at how well the study treatment (marstacimab) works in people living with hemophilia. The summary describes how well marstacimab reduced bleeding episodes in young people (adolescents) 12 to 17 years old.Hemophilia is a condition that causes people to bleed more easily because their blood is unable to clot properly. Clotting is when blood turns from liquid into gel to stop bleeding. Hemophilia is caused by not having enough blood clotting factors, which are proteins found in the blood that help in forming a clot and stop bleeding. People living with hemophilia either don’t make these proteins, have low amounts of these proteins, or their proteins do not work well. There are different types of hemophilia, depending on which clotting factor is affected. For example, people living with hemophilia A lack clotting factor 8, and people living with hemophilia B lack clotting factor 9.Some treatments for hemophilia work by replacing clotting factors. These treatments are called factor replacement therapy, and are given directly into a vein (called intravenous, or IV for short). They are given frequently, sometimes even more than once a week. In some people living with hemophilia, the body develops antibodies called inhibitors that block replacement clotting factors from working properly. Not all people living with hemophilia produce these antibodies – those who don’t are classified as having hemophilia without inhibitors.A different way to treat people living with hemophilia is by using treatment called non-factor replacement therapy. One type of non-factor treatment called marstacimab is given as a regular preventive treatment (called prophylaxis). It works without replacing the missing clotting factors but brings blood clotting and bleeding back to a normal balance.Marstacimab is given once a week as a flat-dose (not based on body weight) injection under the skin (called subcutaneous) using an auto-injector device. This injection is sometimes called a shot and can be easier than injecting directly into a vein. In some cases, marstacimab can be taken less often than clotting factors. Marstacimab offers a new way to treat people living with hemophilia A or B without inhibitors.What were the results of the study?In this study, young people treated with marstacimab for 1 year had on average fewer bleeding episodes than on previous clotting factor replacement treatment. Marstacimab was well tolerated in young people (similar to adults), which means that most side effects that people had were mild to moderate and resolved by the end of the study.What do the results mean?This study showed that a once weekly injection of marstacimab could help reduce bleeding episodes in young people living with hemophilia A or B, without inhibitors.

370

项与马塔西单抗相关的新闻（医药）

2026-08-28

·海百生物

靶点介绍04 | 组织因子（Tissue Factor）深度研究（下）：凝血之始，肿瘤之靶

靶点介绍靶向治疗的黄金时代： TF-ADC与抗TFPI抗体的临床竞速中篇，我们解析了TF作为信号受体驱动肿瘤进展的分子网络。当靶点的生物学被充分阐明，临床转化的逻辑便自然浮现：同一个TF通路，可以导向两种截然不同的治疗策略，在肿瘤侧阻断其表达，在凝血侧解除其抑制。本文将聚焦这两条已进入收获期的抗体赛道，剖析从分子设计到临床验证的完整路径。双轨并行：同一个靶点，两种治疗逻辑基于对TF通路生物学功能的深入理解，靶向治疗沿着两条截然不同的抗体路径展开。子赛道A：TF-ADC（肿瘤治疗）利用肿瘤细胞表面TF的高表达，将细胞毒性载荷精准递送至肿瘤部位。正常组织中TF主要定位于血管外周，不与血流直接接触，这一表达差异为ADC提供了安全窗口。核心逻辑是“抗体导航、载荷杀伤”。子赛道B：抗TFPI抗体（血友病治疗）通过抑制TF凝血途径的天然“刹车”TFPI，增强血友病患者残存的凝血能力。核心逻辑是“功能再平衡”。两条赛道共享对TF通路分子机制的深刻认知，却在治疗目标上截然相反：一个要消灭表达TF的肿瘤细胞，一个要恢复被TFPI过度抑制的凝血功能。这种“同靶异治”的范式在药物开发史上并不多见，也体现了基础研究的深度如何决定临床转化的广度。 TF-ADC：从First-in-class到治疗窗口的瓶颈 TF-ADC的成败取决于抗体表位、内吞效率和连接子-载荷系统三个要素的协同。Tisotumab vedotin（TIVDAK）作为全球首个获批的TF-ADC，其设计体现了对每个要素的精密考量。抗体表位：抗体组分hTF011的表位位于TF胞外域C端FnIII结构域，该位置是TF-FVIIa相互作用的关键区域，抗体结合后可竞争性抑制FVIIa结合，同时诱导强烈的受体介导内吞。人源化过程中，框架区经过回复突变优化，以恢复亲和力并降低免疫原性。连接子-载荷系统：连接子采用蛋白酶可裂解的缬氨酸-瓜氨酸（Val-Cit）二肽，在血浆中稳定，在溶酶体组织蛋白酶B环境中高效裂解。载荷为微管抑制剂MMAE，药物抗体比（DAR）约为4。图1 Tisotumab vedotin作用机制 TIVDAK的获批基于InnovaTV 204和InnovaTV 301两项关键试验。 InnovaTV 204为单臂II期试验，在101例既往接受过治疗的复发或转移性宫颈癌患者中取得24%的客观缓解率，中位缓解持续时间为8.3个月。基于这一数据，2021年FDA授予加速批准。 InnovaTV 301为III期验证性试验，2024年发表于NEJM。研究纳入502例患者，结果显示TIVDAK较研究者选择的化疗显著改善总生存期：中位OS为11.5个月 vs 9.5个月，死亡风险降低30%。无进展生存期和客观缓解率亦显著优于化疗。图2 InnovaTV 301试验总生存期曲线。然而，MMAE的脱靶毒性限制了TIVDAK的治疗窗口。眼部毒性（结膜炎、干眼、角膜炎，发生率约40%）和周围神经病变（发生率约30%）是最主要的剂量限制性毒性，约14.8%的患者因毒性停药。这一毒性谱与MMAE作为微管抑制剂的系统性暴露特性有关。Val-Cit连接子在循环中虽相对稳定，但并非完全不可裂解，游离MMAE的释放可导致非靶向组织的微管破坏。眼部毒性的机制尤为特殊：MMAE通过泪液排泄至结膜，直接损伤角膜缘干细胞。这一经验为新一代TF-ADC指明了方向：需要肿瘤选择性更高、治疗窗口更宽的载荷，以及更稳定的连接子系统来降低系统性暴露。抗TFPI抗体：亲和力悖论与再平衡策略 TFPI由三个Kunitz型蛋白酶抑制域构成，是外源性凝血途径的天然反馈抑制物。血友病A和B分别由FVIII和FIX的遗传性缺失导致，传统替代疗法面临抑制物产生和频繁静脉输注的瓶颈。抗TFPI抗体提供了一种全新的“再平衡”策略：通过解除TFPI对残存凝血能力的抑制，使血友病患者在不依赖外源性凝血因子的情况下恢复止血功能。图3 Marstacimab与TFPI-K2域的相互作用 Marstacimab（Hympavzi，辉瑞）的开发历程体现了抗体工程的关键权衡。其研发团队通过杂交瘤和噬菌体展示技术筛选抗体库，发现一个反直觉的规律：过高亲和力的抗体（Kd < 1 nM）因与TFPI形成过于稳定的复合物，反而被快速清除；中等亲和力（Kd约5.72 nM）的抗体具有更长的血浆驻留时间。这提示治疗性抗体的开发不是“越强越好”，而需要与靶点的生物学特性相匹配。Marstacimab采用全人源IgG1，Fc段经过优化以降低效应功能，避免对表达TFPI的内皮细胞产生不必要的细胞毒性。III期BASIS试验中，年化出血率降低91.6%，2024年10月获FDA批准，成为首个抗TFPI产品。 Concizumab（Alhemo，诺和诺德）针对“伴有抑制物”的最棘手人群，皮下注射每日一次，2024年获FDA批准。KN057（armocibart，康宁杰瑞）是中国首个自主研发的抗TFPI单抗，定位覆盖所有类型血友病的“泛用型”治疗，目前处于III期临床。下一代竞速：平台之战新一代TF-ADC围绕载荷升级、连接子优化和抗体工程三个维度展开，本质上是抗体工程平台的竞速。载荷升级：从MMAE到TOP1i 载荷升级是核心突破口。XNW28012（信诺维）是首个携带TOP1抑制剂载荷的TF-ADC。与MMAE不同，TOP1i通过诱导DNA双链断裂杀伤肿瘤细胞，对缓慢增殖的实体瘤可能更有效，且不易产生与微管抑制剂交叉的耐药。截至2025年11月，在313例患者的I/II期研究中，胰腺癌一线治疗ORR达47.6%。 ADCE-T02（Adcendo）采用exatecan-based载荷，抗体经过Fc沉默改造，2026年5月与MSD达成合作，计划联合Keytruda探索“ADC+PD-1”的协同策略。连接子优化：定点偶联实现DAR均一传统随机偶联产生DAR分布不均的混合物，导致药代动力学波动和脱靶毒性。SYS6051（石药集团）采用工程化半胱氨酸定点偶联，实现均一的DAR值（约2），2026年在中美两地获批临床。 STRO-004（Sutro Biopharma）采用非天然氨基酸定点偶联技术，在抗体特定位置引入对乙酰苯丙氨酸，通过肟键与载荷连接，实现精确的DAR控制。其DAR8 exatecan设计在临床前模型中显示出较第一代TF-ADC显著拓宽的治疗窗口。图4 新一代ADC技术平台：均一化双载荷设计结语：格局与展望 TF-ADC赛道正处于从“First-in-class”到“Best-in-class”的演进期。未来3至5年的关键趋势包括：TOP1i载荷取代微管抑制剂成为主流；位点特异性偶联技术提升均一性；以及“ADC+IO”联合策略的验证。胰腺癌可能成为突破性适应症，TF在PDAC中的高表达率（>90%）和当前治疗的匮乏，使其成为理想的“首发战场”。图5 ADC临床试验全球增长趋势（2006—2022）抗TFPI抗体赛道已进入商业化竞争阶段。Marstacimab的血栓风险（2025年报告一例长期用药患者术后发生致死性血栓性卒中）提示，TFPI抑制的治疗窗口需要更精细的个体化管理。未来的方向包括Fc段改造以延长半衰期、双特异性抗体协同增强凝血，以及口服小分子TFPI抑制剂的开发。从19世纪中叶的组织提取物，到2026年多个抗体药物竞速临床，组织因子的故事印证了药物开发的核心逻辑：深刻的靶点认知是起点，精妙的分子设计是桥梁，而未被满足的临床需求是终点。TF这个百年靶点，正在迎来属于它的黄金时代！关于海百海百生物（HBind）成立于2016年5月，位于武汉市东湖高新区，是一家以“加速药物研发、守护人类健康”为使命，致力于创新生物药、抗体等各类靶向结合分子的开发和应用的高新技术企业，为全球创新药企和科研机构提供从抗原设计制备、抗体开发、筛选评价、工程化改造和生产的一站式抗体服务。团队现已成功完成900+项目，形成多种属全平台的抗体等靶向结合分子开发能力。涵盖人、鼠、兔、羊驼等15个物种，包含流式单B、噬菌体、酵母、细胞、mRNA展示、杂交瘤共六大技术平台。【往期精选】靶点介绍02 | Nectin-4深度研究（上）：一个靶点的起源、沉默与命运转折靶点介绍02 | Nectin-4深度研究（中）：从靶点验证到临床突破，竞争格局如何演变？靶点介绍02 | Nectin-4深度研究（下）：未来方向与关键问题靶点介绍03 | 靶点5T4的三十七年研究历程（上）：从癌胚抗原到多技术路线临床探索靶点介绍03 | 靶点5T4的三十七年研究历程（下）：从临床转化困境到未来突破方向【参考文献】 [1] BOC SCIENCES. Tivdak: FDA's first approved tissue factor-targeted antibody-drug conjugate[EB/OL]. (2021-11-11)[2026-08-21]. [2] VERGOTE I B, GONZALEZ MARTIN A, FUJIWARA K, et al. Tisotumab vedotin as second- or third-line therapy for recurrent cervical cancer[J]. N Engl J Med, 2024, 391(16): 1500-1510. [3] JIN M, HETT S, PATEL S, et al. Discovery and optimization of marstacimab, a human monoclonal antibody targeting tissue factor pathway inhibitor for the treatment of hemophilia A and B[J]. mAbs, 2026, 18(1): 2685362. [4] WEN M, YU A, PARK Y, et al. Homogeneous antibody-drug conjugates with dual payloads: potential, methods and considerations[J]. Int J Mol Sci, 2025, 26(13): 6969. [5] ZHOU W, XU Z, LIU S, et al. Landscape of clinical drug development of ADCs used for the pharmacotherapy of cancers: an overview of clinical trial registry data from 2002 to 2022[J]. BMC Cancer, 2024, 24: 898. [6] COLEMAN R L, LORUSSO D, GENNIGENS C, et al. Efficacy and safety of tisotumab vedotin in previously treated recurrent or metastatic cervical cancer (innovaTV 204/GOG-3023/ENGOT-cx6): a multicentre, open-label, single-arm, phase 2 study[J]. Lancet Oncol, 2021, 22(5): 609-619. [7] DE BONO J S, CONCIN N, HONG D S, et al. Tisotumab vedotin in patients with advanced or metastatic solid tumours (InnovaTV 201): a first-in-human, multicentre, phase 1-2 trial[J]. Lancet Oncol, 2019, 20(3): 383-393. [8] CHAUVIN C, STROH M, HONG D S, et al. Tisotumab vedotin antibody-drug conjugate targeting tissue factor: dose selection and clinical pharmacology strategies[J]. Clin Pharmacol Ther, 2023, 113(5): 1014-1024. [9] DRAGO J Z, MODI S, CHANDARLAPATY S. Unlocking the potential of antibody-drug conjugates for cancer therapy[J]. Nat Rev Clin Oncol, 2021, 18(6): 327-344. [10] FU Z, LI S, HAN S, et al. Antibody drug conjugate: the "biological missile" for targeted cancer therapy[J]. Signal Transduct Target Ther, 2022, 7(1): 93. [11] TARANTINO P, RICCIUTI B, PRADHAN S M, et al. Optimizing the safety of antibody-drug conjugates for cancer therapy[J]. Nat Rev Clin Oncol, 2023, 20(8): 557-573. [12] MATINO D, ACHARYA S S, PALLADINO A, et al. Efficacy and safety of the anti-tissue factor pathway inhibitor marstacimab in participants with severe hemophilia without inhibitors: results from the phase 3 BASIS trial[J]. Blood, 2023, 142(Supplement 1): 285. [13] PANOWSKI S, BHAKTA S, RAAB H, et al. Site-specific antibody drug conjugates for cancer therapy[J]. mAbs, 2014, 6(1): 34-45. [14] DUMONTET C, REICHERT J M, SENTER P D, et al. Antibody-drug conjugates come of age in oncology[J]. Nat Rev Drug Discov, 2023, 22(8): 641-661. [15] BROZE G J, GIRARD T J. Tissue factor pathway inhibitor: structure-function and implications for hemophilia therapy[J]. Thromb Res, 2024, 235: S1-S4.

2026-08-20

·寻找那棵摇钱树

白话药企 | 神州细胞（688520）：靠“造八因子”起家，正把双抗三抗变成第二张牌

神州细胞，这家公司说白了就是一群顶尖生物工程高手，先靠一款“国产重组凝血八因子”安佳因养活自己、打通工厂和渠道，现在想告诉市场：我们不只会做蛋白药，还能做PD-1/VEGF双抗、PD-1/VEGF/TGFβ三抗、自免和血液瘤抗体，是从“单品救命”往“平台型创新药企”转型的典型样本。一、安佳因：曾是现金奶牛，现在降价出清、靠“扩面+出海”续命安佳因（SCT800）是国产首个自研重组八因子，2021年上市，治甲型血友病。巅峰是2024年卖18.9亿，国内rhFVIII份额35.5%排第一；但2025年受医保控费+集采+多次降价拖累，销售额掉到9.97亿，直接把公司拖回亏损。但它不是没戏了：一是血友病中国诊断率、预防治疗渗透率都低，市场本身在变大；二是安佳因已覆盖成人/儿童/围手术期全场景，2025年12月新增围术期适应症，用血场景变多；三是公司建成100亿IU年产能，成本比进口药低一大截，已签超15国合作，巴基斯坦、印尼获批，泰国、菲律宾、越南在注册；四是罗氏艾美赛珠、辉瑞马塔西单抗、赛诺菲RNA疗法虽新，但都没进医保且价格高昂，安佳因在“临时替代+医保可及”这块还是刚需。国海判断降价影响逐步出清，后续看销量提升弥补单价下滑。二、第二曲线：双抗三抗+自免血液瘤，管线排得满但离赚钱还早公司真正想讲的新故事是“IO双抗到三抗”： • SCTB14（PD-1/VEGF双抗）： 2025年11月启动一线驱动基因阴性、TPS≥10%非小细胞肺癌3期，头对头帕博利珠单抗，预计2027年7月读出主要PFS终点。 2期数据不错（一线NSCLC目标剂量ORR 90.3%、6月PFS率89%），赛道里康方依沃西已证明双抗疗效优于单药K药，三生制药同类双抗卖给辉瑞、荣昌RC148卖给艾伯维，首付款5–12.5亿美元，神州这款分子具备BD出海想象空间。 • SCTB41（PD-1/VEGF/TGFβRII三抗）： 2期单药在≥2线实体瘤uORR 22%、2线NSCLC mPFS 8.3个月，再加SCTB39-1/39G（PD-L1/CTLA-4/TIGIT）两款三抗，国内研发进度靠前；三抗出海先例已有（先声CD3/BCMA/GPRC5D授权艾伯维，首付10.55亿美元；泽璟ZG006三抗同样授权艾伯维）。 • 自免+血液瘤补刀： SCT650C（IL-17A单抗）银屑病3期进行中，维持期24周给药一次，比司库奇尤4周一针依从性更强； SCTC21C（CD38单抗）一线多发性骨髓瘤和轻链淀粉样变均进入3期，国内同靶点在研品种稀缺； SCTB35（CD20/CD3 TCE）复发淋巴瘤处于3期、系统性红斑狼疮处于2期，走“肿瘤TCE技术反哺自免疾病”路线。三、底子与隐患：工程能力真硬，但钱紧、单品依赖、临床兑现是关键正面看，谢良志（MIT化工博士、前默克研发人员）+王阳的专业班底，把CHO细胞表达、蛋白纯化、规模化放大、全流程质控完整跑通，所以既能落地100亿IU八因子大产能，又能同步推进双抗、三抗管线开发—— 这是它和单纯引进外部管线（License-in）药企最核心的区别。 2026年5月递表港股，中信证券担任保荐人，募资计划投入早期新药研发、临床推进与商业化推广，核心逻辑是“A股阶段烧钱压力大，赴港上市补充长期资金”。反面看三大风险点： ①营收高度依赖安佳因，2025年安佳因仍占总营收63.9%，单品价格、销量波动会直接造成业绩大幅跳水； ②25年末资产负债率96.85%、经营现金流转负，26年一季末有息负债约31亿元，经营现金流承压； ③SCTB14要到2027年下半年才读出三期关键数据，SCT650C、CD38单抗、CD20/CD3 TCE等品种距离商业化还有数年，中间一旦出现临床进度延期、海外BD合作不及预期，估值支撑逻辑会明显弱化。总而言之，神州细胞是“八因子生产平台进化为多特异性抗体创新平台”的典型尝试，安佳因守住业绩基本盘、SCTB14等创新管线打开长期成长天花板。当下最真实的现状是——管线临床数据亮眼、持续研发投入持续失血，本次港股IPO将决定公司能否撑到多款新药上市兑现收益。

2026-08-17

·Pharmaceutical Technology

EMA validates Pfizer and Valneva’s Lyme disease vaccine

In the VALOR clinical trial, PF-07307405 showed efficacy of over 70% in preventing Lyme disease. Credit: MrWinn / Shutterstock.com. The European Medicines Agency (EMA) has validated the marketing authorisation application (MAA) from Pfizer and Valneva for their Lyme disease vaccine candidate, PF-07307405. With this validation, the agency will begin its formal assessment of the application. The submission is supported by efficacy and safety data from the Phase III VALOR clinical trial, which enrolled 9,437 participants aged five years and above across the US, Canada, and Europe. In the study, PF-07307405 showed efficacy of more than 70% in preventing Lyme disease. The vaccine candidate was found to be well tolerated with no identified safety concerns, according to the companies. Pfizer senior vice-president and chief vaccines officer Annaliesa Anderson said: “I’m proud of the progress we have made with our Lyme disease vaccine candidate and excited about the opportunity to potentially bring the first vaccine of its kind to Europe. I look forward to collaborating with the EMA as this vaccine candidate advances through the review cycle.” “Lyme disease remains the most common vector-borne illness in Europe, affecting more than 100,000 people each year. When left untreated, it can lead to serious and potentially long-lasting complications involving the skin, joints, nervous system, and heart. “We believe vaccination has the potential to provide an important new layer of protection against this debilitating disease and help people continue to enjoy the outdoors with greater confidence.” Pfizer and Valneva began collaborating on PF-07307405 in April 2020, with the former holding exclusive manufacturing and commercialisation rights if regulatory approval is achieved. The vaccine is a 6-valent outer surface protein A (OspA)-based protein subunit, targeting six Borrelia OspA serotypes found in North America and Europe. Currently, no approved human vaccines exist for Lyme disease. In June 2026, Pfizer received approval from the US Food and Drug Administration for an expanded indication of Hympavzi (marstacimab-hncq) to treat haemophilia A or B.

临床结果疫苗临床3期临床1期

100 项与马塔西单抗相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11261	-	-	-

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
血友病A	美国	Pfizer Inc.	2024-10-11
血友病B	美国	Pfizer Inc.	2024-10-11

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
中面发育不全，轻度至重度	临床3期	美国	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	日本	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	阿根廷	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	澳大利亚	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	奥地利	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	巴西	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	加拿大	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	捷克	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	丹麦	辉瑞投资有限公司	2022-12-09
中面发育不全，轻度至重度	临床3期	法国	辉瑞投资有限公司	2022-12-09

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03938792 (BASIS, ASH2025)	临床3期	血友病B \| 血友病A	17	Marstacimab	膚遞鏇繭膚鑰願餘簾憲(製願顧鹹顧網襯夢簾醖) = 鑰遞遞簾獵廠齋齋艱窪繭憲憲鹽鏇顧鏇憲鏇繭 (餘觸齋積願遞簾淵蓋鹽 ) 更多	积极	2025-12-06
				prior routine prophylaxis	膚遞鏇繭膚鑰願餘簾憲(製願顧鹹顧網襯夢簾醖) = 鬱鑰鹹鏇糧遞構壓鏇襯繭憲憲鹽鏇顧鏇憲鏇繭 (餘觸齋積願遞簾淵蓋鹽 ) 更多
NCT03938792 (BASIS, ASH2025)	临床3期	血友病B \| 血友病A	116	Marstacimab 300 mg SC	窪糧顧廠衊繭艱顧醖淵(衊構鏇鬱製齋顧蓋繭艱) = 積窪製繭襯鬱壓願鹽鹽網鹹艱襯觸糧觸齋蓋願 (獵淵餘築蓋憲製積鬱蓋, 5.52) 更多	积极	2025-12-06
NCT03938792 (BASIS, BusinessWire) 人工标引	临床3期	血友病B \| 血友病A	-	Hympavzi	憲顧網鬱觸襯網鬱顧願(顧簾構願憲餘衊鏇襯範) = 鹹鑰範齋鑰遞鏇糧淵顧鬱餘齋鹹鬱齋餘壓選衊 (壓願網窪窪構築積鏇蓋 ) 达到	积极	2025-06-26
				on-demand treatment	憲顧網鬱觸襯網鬱顧願(顧簾構願憲餘衊鏇襯範) = 醖膚襯衊選顧醖糧夢廠鬱餘齋鹹鬱齋餘壓選衊 (壓願網窪窪構築積鏇蓋 ) 达到
ASH2024	N/A	血友病B \| 血友病A	-	Marstacimab 150 mg SC QW	鏇齋憲夢醖憲餘繭製願(鑰淵糧選襯蓋糧獵淵願) = 蓋製網襯構鬱簾醖蓋顧簾範簾淵製糧鹹鹽鬱範 (艱網糧鹹膚遞鑰襯壓衊 )	-	2024-12-09
				Factor replacement therapy (OD or RP)	鏇齋憲夢醖憲餘繭製願(鑰淵糧選襯蓋糧獵淵願) = 艱顧襯夢鑰淵遞網艱艱簾範簾淵製糧鹹鹽鬱範 (艱網糧鹹膚遞鑰襯壓衊 )
NCT03938792 (FDA_CDER) 人工标引	临床3期	血友病B \| 血友病A	66	On-Demand Factor-Based Therapy	簾顧膚鏇網選糧願齋鹹(獵遞繭網遞製製鹹積窪) = 遞廠窪淵糧獵鹽夢觸壓鏇淵淵鏇鹽窪築鬱艱選 (顧齋網鬱醖鹹範憲壓膚, 31.03 ~ 46.54) 更多	积极	2024-10-11
				HYMPAVZI Prophylaxis	簾顧膚鏇網選糧願齋鹹(獵遞繭網遞製製鹹積窪) = 網製遞廠選鑰顧淵積鹹鏇淵淵鏇鹽窪築鬱艱選 (顧齋網鬱醖鹹範憲壓膚, 2.09 ~ 4.85) 更多
NCT05145127 (FDA_CDER) 人工标引	临床3期	血友病B \| 血友病A	-	On-Demand Factor-Based Therapy	夢夢壓醖糧鑰蓋觸膚獵(獵鏇齋繭獵襯鬱淵夢醖) = 獵壓構製築積鑰製夢鹽願鑰壓窪鏇繭簾餘鏇襯 (觸膚襯衊糧遞願鑰鏇淵, 5.09 ~ 10.61) 更多	积极	2024-10-11
				HYMPAVZI Prophylaxis	夢夢壓醖糧鑰蓋觸膚獵(獵鏇齋繭獵襯鬱淵夢醖) = 壓顧獵製願鹹鹽鬱網窪願鑰壓窪鏇繭簾餘鏇襯 (觸膚襯衊糧遞願鑰鏇淵, 3.40 ~ 6.77) 更多
NCT03938792 (BASIS, ASH2023) 人工标引	临床3期	血友病B \| 血友病A	128	Marstacimab (On Demand) (Observational Phase)	選糧餘網艱膚餘壓積淵(構獵願願觸憲艱壓衊積) = 廠製壓構襯繭襯齋窪願選顧糧顧襯顧繭製壓繭 (製繭窪壓鬱獵鑰鑰淵繭, 31.03 ~ 46.54) 更多	积极	2023-12-09
				Marstacimab (Routine Prophylaxis) (Observational Phase)	選糧餘網艱膚餘壓積淵(構獵願願觸憲艱壓衊積) = 餘夢襯製獵壓鹽襯遞齋選顧糧顧襯顧繭製壓繭 (製繭窪壓鬱獵鑰鑰淵繭, 5.09 ~ 10.61) 更多
NCT04878731 (CTgov)	临床1期	血友病	6	PF-06741086 (Marstacimab)	鏇鏇觸鏇窪選糧鹹夢鬱 = 簾製蓋壓鑰壓選築製窪鹹積襯餘構糧淵壓壓遞 (選鬱糧窪醖簾願窪鏇膚, 艱壓壓廠觸壓鏇顧廠蓋 ~ 衊憲壓繭醖鬱選壓網構) 更多	-	2023-07-07
NCT03363321 (Pubmed) 人工标引	临床2期	血友病A	18	Marstacimab	壓鹽顧觸膚醖壓淵艱願(鹹壓獵獵鏇衊鏇選積夢) = 積遞餘觸糧艱選窪觸範憲簾醖壓襯廠觸艱築鏇 (範壓襯淵窪廠醖醖衊鬱 )	积极	2022-10-11
NCT02974855 (CTgov)	临床2期	血友病	27	PF-06741086 (PF-06741086 300 mg SC QW Non-Inhibitor)	範獵觸餘鏇鹹醖製衊獵 = 廠餘窪簾鏇糧鹹鑰鬱壓膚鹹積築襯製衊積窪憲 (鬱壓簾製築簾遞壓鏇範, 餘夢選膚鹽簾鹽選廠壓 ~ 鬱夢築築簾積繭鹽積構) 更多	-	2019-12-04
				PF-06741086 (PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor)	範獵觸餘鏇鹹醖製衊獵 = 顧憲醖鬱獵蓋選鹽鑰鹹膚鹹積築襯製衊積窪憲 (鬱壓簾製築簾遞壓鏇範, 廠獵窪夢獵範範構獵艱 ~ 簾獵願淵夢範淵夢糧範) 更多