This study is conducted in Japan of vonicog alfa (rVWF) used to treat pediatric participants with Von Willebrand Disease (vWD).
The main aim of the study is to evaluate adverse drug reaction and effectiveness of vonicog alfa (rVWF).
During the study, pediatric participants with vWD will be administered with rVWF under routine normal practice. The investigators will evaluate adverse events due to rVWF for 1 year from the start of drug administration.
The study sponsor will not be involved in how the participants are administered but will be recorded what happens during the study.

开始日期2026-02-19

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

NCT07129343

/ Recruiting临床3期

A Clinical Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in the Treatment of Bleeding Episodes in Chinese Subjects Diagnosed With Von Willebrand Disease

The main aim of this study is to find out if VONVENDI is safe for adult Chinese participants with VWD. The study will also check how well VONVENDI helps control bleeding with or without product ADVATE in the participants who may need elective surgery or dental procedures. In addition, the study will also examine how VONVENDI is processed by the body (known as pharmacokinetic [PK]) and how the drug helps the body respond or improve a condition (pharmacodynamic [PD]).
Participants will receive an initial dose of VONVENDI of 40 to 80 international units per kilogram (IU/kg) of body weight. If a participant's baseline factor VIII (FVIII) level is not high enough to help stop bleeding, VONVENDI will be given along with 30 to 45 IU/kg of ADVATE rFVIII.
Participants will be in the study for approximately 14 months. During the study, participants will be followed up at clinics or over telephone calls.

开始日期2025-10-13

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

ChiCTR2500108310

/ Suspended临床3期IIT

A Clinical Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Recombinant von Willebrand Factor (rVWF) With or Without ADVATE in the Treatment of Bleeding Episodes in Chinese Subjects Diagnosed With von Willebrand Disease

开始日期2025-03-21

申办/合作机构

苏州大学附属第一医院

100 项与伏尼凝血素α 相关的临床结果

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100 项与伏尼凝血素α 相关的转化医学

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100 项与伏尼凝血素α 相关的专利（医药）

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项与伏尼凝血素α 相关的文献（医药）

2026-04-01·Hamostaseologie

Use of Vonicog Alpha and Acquired von Willebrand Syndrome, a New Approach: A Case Report

Article

作者: Blandinières, Adeline ; Combe, Sophie ; Chanson, Noémie ; Lavenu-Bombled, Cécile ; Lambotte, Olivier

Abstract:

Therapeutic management of acquired von Willebrand syndrome (AVWS) can be challenging, particularly in cases of AVWS associated with monoclonal IgM such as Waldenström macroglobulinemia (WM) where several therapeutic options may be ineffective. Here, we describe the case of an 88-year-old patient who developed AVWS during follow-up for WM. The presence of a severe bleeding symptomatology not controlled by several therapies (plasma-derived von Willebrand factor, plasmapheresis) led us to introduce a supplementation with recombinant von Willebrand factor, vonicog α (Veyvondi, Takeda, Japan), starting at a dose of 50 IU/kg/d. This supplementation allowed clinical (no further bleeding) and biological (hemoglobin level, von Willebrand factor parameters) improvements. Because of the persistence of bleeding risk factors, the treatment was maintained at a prophylactic dose (20 UI/kg three times a week), without recurrence of bleeding events for a period of 9 months.

2026-02-10·Blood Advances

Modeling response to high-purity von Willebrand factor: toward tailored dosing during surgery in von Willebrand disease

Article

作者: Daniel, Mélanie Y. ; Dupont, Annabelle ; Zawadzki, Christophe ; Jeanpierre, Emmanuelle ; Marichez, Catherine ; Rauch, Antoine ; Denis, Cécile V. ; Goudemand, Jenny ; Lenting, Peter J. ; Paris, Camille ; Susen, Sophie ; Delavenne, Xavier ; Pradines, Bénédicte

Abstract:

In surgical settings, managing von Willebrand disease (VWD) often requires von Willebrand factor (VWF) concentrate administration, and close monitoring of factor VIII (FVIII) and VWF to balance bleeding control with the risk of excessive factor elevation. This management is further complicated by VWD-related variability and the pharmacokinetic (PK) impact of VWF products, containing or not exogenous FVIII. We hypothesized that building a PK model based on high-purity VWF concentrates may clarify the VWF-FVIII relationship by eliminating exogenous FVIII interference. Our aim was to characterize the PK/pharmacodynamic (PD) relationship and variability of a high-purity VWF concentrate in patients with VWD undergoing surgery. A retrospective analysis was conducted on patients treated perioperatively with a high-purity plasma-derived VWF concentrate (Wilfactin, Laboratoire Français du Fractionnement et des Biotechnologies, France), nearly devoid of FVIII, from January 2016 to December 2018. Data were analyzed using a population PK/PD modeling approach. VWF antigen (VWF:Ag) was prioritized as it better reflects VWF-FVIII binding capacity rather than VWF activity, which measures platelet interaction. Covariates influencing VWF:Ag and FVIII:C PK/PD were analyzed. Seventy-four patients undergoing 99 surgeries were included. The model revealed substantial variability in VWF:Ag and FVIII:C volume of distribution, clearance, and baseline levels. Covariates such as age, bodyweight, VWD type, blood group, and VWF propeptide significantly affected PK/PD parameters. Interindividual variability in PK parameters (eg, clearance, volume), ranged from 25.3% to 73.8%. High interindividual PK/PD variability was observed with high-purity VWF concentrate. This highlights the need to develop a robust dose-adjustment model to optimize factor concentrate utilization, avoid unnecessary waste, and reduce the frequency of laboratory monitoring.

2026-02-01·JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Prevention of hemorrhage after implantation of mechanical circulatory support with a purified von Willebrand factor concentrate: results of the early terminated randomized controlled trial

Article

作者: Dambrin, Camille ; Fiore, Mathieu ; Smadja, David M ; Lenting, Peter J ; Flecher, Erwan ; Jeanpierre, Emmanuelle ; Rauch, Antoine ; Barandon, Laurent ; Bouchot, Olivier ; Nedelec, Fabienne ; Repessé, Yohann ; Vincentelli, André ; Moussa, Mouhamed D ; Rozec, Bertrand ; Voisin, Sophie ; Laporte, Silvy ; Boissier, Elodie ; De Maistre, Emmanuel ; Labreuche, Julien ; Ajzenberg, Nadine ; Lebreton, Guillaume ; Susen, Sophie

BACKGROUND:

Bleeding complications are frequent under left ventricular assist device (LVAD) support. LVAD-associated high shear stress induces increased proteolysis of circulating von Willebrand factor (VWF), which may contribute to this high bleeding rate.

OBJECTIVES:

To assess if a prophylactic administration of a VWF concentrate could reduce bleeding rate in LVAD patients METHODS: In this multicenter open-label randomized controlled study, we investigated the efficacy, safety, and pharmacokinetic of a plasma-derived VWF concentrate (WILFACTIN, LFB), administered prophylactically twice-weekly at 50 IU.kg^-1 for 3 months after LVAD implantation, compared with standard of care. The efficacy end points were the difference in the incidence rate of any, clinically significant or major bleeding postrandomization. Bleeding and thrombotic events were reviewed at weeks 1 and 2 and then every 2 weeks. The study ended prematurely due to deficient recruitment and drug supply constraints, leading to prioritization of prescriptions for von Willebrand disease patients. We present the available data.

RESULTS:

Twenty-nine adult patients (166 planned) were randomized and analyzed. VWF prophylaxis resulted in a nonsignificant 55% reduction in bleeding incidence rate compared with standard of care (risk ratio, 0.45; 95% CI, 0.18-1.04; P = .06). Accordingly, there was a similar reduction of clinically relevant and major bleeding in VWF arm. Severe adverse events were similar postrandomization between VWF arm and control arm including deaths (2 and 4, respectively) and thrombotic events (2 in each arm). In pharmacokinetic analysis, a rapid degradation of WILFACTIN occurred within 24 hours.

CONCLUSION:

The lack of observed therapeutic efficacy of VWF prophylaxis in LVAD setting is likely due to a short-lived prohemostatic effect due to rapid degradation of VWF concentrate.

项与伏尼凝血素α 相关的新闻（医药）

2026-08-11

·FierceBiotech

After tasting IPO success, Hemab’s CEO seeks ‘flavor of a multi-product coagulation company’

Hemab’s Kendall Square office in Cambridge, Massachusetts, gives visitors a rare view above the Charles River and downtown Boston. It’s an enviable backdrop for realizing the company’s vision of becoming the premier clotting biopharma. The view isn’t the only thing keeping spirits high at the newly public company, which completed an upsized $301.5 million IPO in May to fund its pipeline of therapies aimed at helping patients living with bleeding disorders. In addition to underwriters helping lift the final IPO proceeds to $346.7 million, the biotech's stock had tripled in value to $53.48 by August 12, up from a May debut of $18. The company ended June with $457.5 million in the bank, which Hemab expects to pave a cash runway into 2029. The biopharma has modestly expanded its workforce since the IPO, and now employs 80 people. With the post-IPO quiet period having come to an end, CEO Benny Sørensen, M.D., Ph.D., was free to sit down with Fierce to discuss Hemab’s future as a publicly listed company. Sørensen called the Nasdaq listing both a “natural step” and a “dream come true.” The IPO was a result of years of hard work and an expected part of the plan, but he was pleased with the scale of the response. The company, which has dual headquarters in Copenhagen, Denmark and Cambridge, has raised several rounds of funding over the years. This included a $55 million series A in 2021, a $135 million series B in 2023 and a $157 million series C last year. “It was clear that to advance the company, build out the portfolio, and be serious about this mission of building the ultimate coagulation company, the natural next step was a public offering to access those capital markets,” Sørensen explained. Fortunate that the company’s data and progress aligned with a wide-open IPO window, Sørensen said he encountered excitement for Hemab’s vision and progress when the company set out on its roadshow. “We had good interest from very high-quality investors,” he said. “Not just a few, but multiple—positioning us for building a book that is highly concentrated with top-notch investors. It felt like a dream come true.” Sørensen said Hemab had already achieved its initial goal of having five projects in development by 2025. The company recently shared data on three of those programs, including its bispecific antibody sutacimig, which is in phase 2 studies for patients with the rare inherited bleeding disorders Glanzmann thrombasthenia (GT) and factor VII deficiency (FVIID). At the International Society on Thrombosis and Haemostasis Congress last month, the company unveiled phase 2 data from a long-term extension study of sutacimig in GT that it said demonstrated the therapy's “potential to shift people ... from reactive, IV-dependent treatment to subcutaneous prophylaxis.” Hemab is gearing up for a phase 3 trial later this year. This summer also saw Hemab read out data for its phase 2-stage antibody HMB-002. The subcutaneous prophylactic injection targets the lifelong inherited bleeding disorder von Willebrand disease (VWD), and has been tied to a 2.4-fold or greater peak increase in von Willebrand factor, a glycoprotein that helps blood clot. “The hardworking may be allowed to be lucky too,” said Sørensen as he reflected on the findings. “The HMB-002 data were much better than anyone had hoped for or thought they would be.” Last month, Hemab unveiled HMB-003, a non-hormonal direct plasmin inhibitor designed to reduce bleeding across multiple settings, beginning with heavy menstrual bleeding. The asset is set to enter the clinic later this year. The fatty acid-conjugated peptide resembles Wegovy or Mounjaro in its delivery platform, but is tailored for women with heavy menstrual bleeding, Sørensen said. “Taboo and stigma hold on to this, and [heavy bleeding] generates iron deficiency and anemia,” the CEO explained. “On average, a woman with heavy menstrual bleeding is losing 3.6 weeks from work every year.” Sørensen emphasized the company’s commitment to women’s health, pointing out that Hemab has made an effort to ensure women are well represented in its trials. A total of 86% of the participants in the phase 1 study of HMB-001 were female, according to the biotech, with women accounting for half of the patients enrolled in the phase 2 trial for sutacimig in GT. “There's a recognition that this is a completely underappreciated, unmet need,” Sørensen said. “It requires modern treatment options to empower women to control this.” Sørensen has dedicated much of his career to hematology and coagulation. Before joining Hemab as CEO in 2021, his previous roles included senior director of clinical research at Alnylam Pharmaceuticals, where he led development of Qfitlia and other hematology products, as well as global medical director at Baxter Healthcare, where he helped develop the hemophilia therapy Adynovate and the VWD drug Vonvendi, which were acquired by Takeda in the 2019 Shire buyout. Before joining the biotech industry, he was the director of the hemostasis research unit and honorary lecturer at Guy’s and St. Thomas’ Hospital and King’s College London School of Medicine. Sørensen said two of Hemab's existing five pipeline programs have yet to be unveiled, and he already has his sights set on launching two commercial products, advancing two new late-stage programs and entering two other drugs into phase 1/2 by 2030. This will give Hemab “the flavor of a multi-product coagulation company,” he explained. The CEO is hopeful about the future for patients living with bleeding disorders. Sørensen pointed to the success of Genentech’s clotting medicine, which transformed hemophilia “from being a debilitating disease to being put on Hemlibra today.” These medical advances mean “you are not going to die from hemophilia,” he pointed out. Still, there is plenty of unmet need in the bleeding disorder space. “We left so many behind, and now we're on the mission of trying to close the gap,” Sørensen said.

临床2期IPO高管变更并购临床1期

2026-07-10

·肽集圈

2026年6月全球生物制药TOP交易汇总：M&A、授权合作与AI驱动交易全景

2026年6月，全球生物制药行业交投活跃，并购（M&A）与授权合作（Licensing）双线开花。艾伯维（AbbVie）以109亿美元收购Apogee Therapeutics领跑月度榜单，GSK以106亿美元拿下Nuvalent紧随其后。mRNA疗法、小分子降解剂及AI驱动的药物发现成为资本最热追逐方向。🚀 一、TOP M&A：巨头疯狂"扫货"1️⃣ AbbVie $10.9B → Apogee Therapeutics（抗体）核心资产： Zumilokibart（抗IL-13/IL-22双靶点抗体），用于特应性皮炎（AD）、哮喘及嗜酸细胞性食管炎（EoE）看点： Apogee管线与AbbVie自免旗舰Skyrizi（IL-23抑制剂）形成完美互补，强化皮肤科/呼吸免疫霸主地位意义： AbbVie 2026年首笔收购，标志其后Humira时代自免管线"换血"加速2️⃣ GSK $10.6B → Nuvalent（小分子）核心资产： Zidesamtinib（NVL-655），一款高选择性ALK/ROS1/TRK多靶点抑制剂，用于NSCLC 看点： GSK近年来在肺癌领域持续加码（与翰森合作risvutatug rezetecan ADC），此次收购补强口服小分子肿瘤管线背景： GSK今年已连续完成多笔交易（Sirna Bio反义寡核苷酸、Frontier siRNA、RAPT mAb等），全面铺开技术平台3️⃣ Servier $未披露 → Edgewise Therapeutics（小分子）核心资产： Sevasemten（EDG-7500）——肌球蛋白抑制剂，用于杜氏肌营养不良（DMD）及心力衰竭看点：继3月$25亿收购Day One后，Servier继续深耕神经肌肉罕见病额外资产： Edgewise还有心血管管线（肌节调节剂）4️⃣ Incyte $1.25B → Star Therapeutics（抗体）核心资产： VGA039——抗von Willebrand因子（vWF）单抗，用于血管性血友病（VWD），III期进行中看点：获FDA快速通道+孤儿药+罕见儿科疾病+突破性疗法四项认定；对标武田Vonvendi 里程碑： Star可获最高$7.5亿销售里程碑5️⃣ Ipsen $1.75B → Kartos Therapeutics（小分子）核心资产： Navtemadlin（MDM2抑制剂），用于骨髓纤维化（Myelofibrosis），III期进行中看点： Kartos可获$4.5亿首付款，里程碑最高可达$13亿（含销售） Ipsen第二笔： €2亿收购苏黎世Memo Therapeutics（BK多瘤病毒肾病mAb）6️⃣ J&J 未披露金额 → Firefly Bio（降解剂-抗体偶联平台）核心平台：降解剂抗体偶联物（Degrader Antibody Conjugate, DAC）——与ADC不同，载荷为靶向蛋白降解剂而非化疗药物看点：用于KRAS驱动肿瘤，是J&J 2026年首笔收购，标志其正式入局靶向蛋白降解7️⃣ Biogen $1B → RayThera（免疫）核心资产：免疫介导疾病候选分子（具体未详述），即将进入临床背景：继$5.6B收购Apellis（肾脏病）后，Biogen持续扩张免疫/肾脏管线8️⃣ Zymeworks $929M → Theravance Biopharma（吸入制剂）核心资产： Revefenacin——长效毒蕈碱拮抗剂（LAMA），用于COPD，已获批上市看点： Zymeworks借此从肿瘤双抗平台跨界呼吸疾病9️⃣ Thalia Therapeutics £3.675M → Sanmirna Therapeutics（miRNA）核心资产： microRNA抑制剂，用于急性髓系白血病（AML），I期进行中看点：英国Biotech加码RNA疗法🔟 Lilly 未披露 → 4E Therapeutics（神经科学）核心资产： MNK抑制剂，用于慢性疼痛（非阿片类），IND-enabling阶段看点：礼来"去阿片化"镇痛策略的重要拼图🧬 二、授权合作（Licensing）：mRNA与AI强势崛起📌 mRNA疗法热度不减合作方合作内容金额日本新药（Nippon Shinyaku）× Elixirgen mRNA疗法EXG-7001治疗DMD 未披露 Alnylam × Inceptive Nucleics AI辅助发现siRNA（利用Inceptive基础模型）最高$20亿（含$3000万首付） Lilly × Ascidia Therapeutics RNA外显子编辑器（靶向肾脏疾病）最高$19亿📌 抗体与AI持续发酵合作方合作内容金额 BI × Immunai（纽约） AI发现T细胞靶点（IO+自免） $1500万+延期 BI × Combotope（哥本哈根） SMART-Phage平台发现肿瘤糖基化抗体未披露 TrueLab（中国）× Bionyra（法国）抗TL1A mAb + TL1A/IL-23p19双抗（炎症疾病）最高$9.85亿+股权 Insilico Medicine × SK Biopharmaceuticals（韩国） AI平台发现神经免疫候选分子最高$25亿+（$1800万首付）📌 小分子/蛋白降解合作方合作内容金额 Roche × Nurix Therapeutics BTK降解剂Bexobrutideg（CLL）最高$30亿（$7亿首付） Travere × Everest Medicines（中国） BTK抑制剂Civorebrutinib（罕见肾病）最高$11.4亿（$1.125亿首付）📊 三、2026年6月交易全景数据维度关键数据月度总交易额（估算） >500亿美元（含M&A+授权里程碑）最大单笔 AbbVie收购Apogee（$109亿）最热技术 mRNA疗法、AI药物发现、蛋白降解（DAC/PROTAC）、双抗/多抗最活跃MNC GSK（4笔）、AbbVie、Lilly、BI、J&J 最活跃国家美国（买方/卖方均占主导）、瑞士、德国、英国、中国（授权出方）主要治疗领域肿瘤、自免/炎症、神经科学、呼吸/COPD、罕见病💡 四、一句话洞察 "2026年6月证明：MNC的'管线焦虑'已转化为实际行动——109亿买抗体、106亿买小分子，AI和mRNA不再是'未来概念'而是'当下刚需'。中国Biotech（Everest、TrueLab）持续作为创新输出方参与全球价值链。"

2026-06-23

·今日头条

融资66亿元：这家Biotech再次冲刺纳斯达克

2026年5月，在生物医药领域，有5家公司以IPO的方式赴美上市，1家公司宣布以借壳形式上市。一、IPO上市今年5月，共有5家生物医药公司以IPO形式上市，合计融资规模达 9.87亿美元，其中 Hemab、 Seaport、 Odyssey三家公司的IPO规模均超2亿美元。 1、 Hemab Therapeutics(NASDAQ:COAG) 5月1日， Hemab Therapeutics正式登陆纳斯达克，发行价为18美元，发行规模为1675万股，募集资金 3.015亿美元，发行规模较最初计划上调27%。 Hemab Therapeutics 上市首日开盘28美元，收盘涨幅达88.89% ，市值逼近16亿美元。 Hemab Therapeutics成立于2020年，是一家来自丹麦的生物医药公司，由诺和诺德旗下投资机构Novo Holdings的风投团队Novo Seeds出资创立，主要致力于研发治疗带有出血症状的罕见血液病药物。公司拥有诺和诺德及另一家丹麦药企Genmab的抗体技术平台专利授权许可，基于授权许可，Hemab有两款在研药物： sutacimig ：一款双特异性抗体，其作用机制为：一条臂结合并稳定内源性凝血因子Ⅶa，另一条臂结合活化血小板表面的TLT-1受体，促进内源性凝血因子Ⅶa在体内富集并直接募集至活化血小板表面，助力止血栓形成。该药为全球首创的格兰茨曼血小板无力症预防性在研药物。目前， sutacimig 正在用于两种罕见凝血障碍：格兰茨曼血小板无力症（Glanzmann thrombasthenia）和Ⅶ因子缺乏症。公司计划于今年启动sutacimig在格兰茨曼血小板无力症的Ⅲ期临床试验，并预计在2026年底或2027年初披露其在Ⅶ因子缺乏症中的中期试验数据。格兰茨曼血小板无力症（GT）为严重出血性疾病，以反复、致残性且可危及生命的出血发作为特征。国际GT360自然史研究揭示疾病沉重负担：在117例受试者中，88%报告近一周内至少发生1次出血，65%在过去六个月内因出血入院。出血事件显著影响患者心理健康与生活质量：超80%存在误工/缺课，超50%社交活动受限，超50%出行受限。截至目前，该病尚无有效预防性治疗手段。今年3月5日，美国食品药品监督管理局（FDA）授予sutacimig突破性疗法认定（BTD），用于格兰茨曼血小板无力症（GT）患者的出血预防。此外， sutacimig还获得了FDA授予的快速通道与孤儿药资质。 HMB‑002 ：一款用于 Von Willebrand Disease （血管性假血友病）的预防性抗体疗法，目前正处于Ⅰ/Ⅱ期临床研究的阶段，下一批数据预计将于今年年底或明年年初公布。 Hemab的管理层与 RNAi疗法龙头企业Alnylam 颇具渊源，公司CEO Benny Sorensen曾在Alnylam工作，领导了靶向抗凝血酶药物 fitusiran 的研发。在此之前，他是百特医疗公司（Baxter）的全球医学总监，负责推进Adynovate®和Vonvendi®的关键开发工作。在进入行业之前，他曾是盖伊医院和圣托马斯医院及伦敦国王学院医学院的血栓研究部门主任。首席医疗官 Kate Madigan博士拥有超过20年的临床血液学经验，曾担任Syndax Pharmaceuticals的首席医疗官，期间她领导了Revuforj和Niktimvo在FDA的获批工作，亦曾担任Alnylam Pharmaceuticals高级医学总监以及Biogen的罕见病创新部门总监等职务。董事会主席John Maraganore则是 Alnylam的创始人之一，并担任了近20年的 Alnylam公司的CEO 。在一级市场， Hemab Therapeutics 受到资本追捧，成立五年时间，已经完成近 3.5亿美元（约合23.48亿元）的融资： 2021年7月，公司完成超额认购5500万美元的A轮融资，本轮融资由RA Capital Management、Novo Holdings和HealthCap领投； 2023年2月，公司完成1.35亿美元的B轮融资，本轮融资由Access Biotechnology领投，Deep Track、Avoro Ventures、Invus、Rock Springs、Novo Holdings、RA Capital等机构跟投； 2025年10月，公司完成1.57亿美元C轮融资。本轮融资由Sofinnova Partners领投，Avoro Capital、RA Capital、Novo Holdings、Access Biotechnology、Deep Track、HealthCap、Invus、Avoro Ventures、Maj Invest Equity、Rock Springs Capital等机构跟投。 2、 Seaport Therapeutics(NASDAQ:SPTX) 5月1日，Seaport Therapeutics在纳斯达克交易所上市，发行价为每股18美元，发行1416万股，融资规模达2.55亿美元。 Seaport Therapeutics由PureTech Health孵化成立于2023年，是一家中枢神经系统（CNS）新药研发公司，曾入选Endpoints 11评出的“Biopharma's Most Exciting Startups in 2024”企业名单，以及 Fierce Biotech’s 2025 Fierce 15名单。 PureTech Health曾创立CNS药企Karuna Therapeutics，后于2023年底被百时美施贵宝以140亿美元收购。 PureTech通过 “Hub-and-Spoke”模式运营，发现、开发或收购有前景的药物项目，然后将其拆分成个体初创公司（ Hub-and-Spoke：创新药企的新模式）。 2017 年， PureTech从莫纳什大学Chris Porter教授获得Glyph技术平台的授权。 Glyph 平台是一种药物递送平台，改变了药物进入体循环的正常路径，绕过肝脏，从肠道进入通常处理膳食脂肪的淋巴管，提高口服生物利用度，避免首过代谢，减少肝酶升高或肝毒性及其他副作用。目前，Seaport Therapeutics有三款在研药物： SPT-300 ：一款异孕烷酮的口服前物，目前正在进行针对伴有或不伴有焦虑困扰的重度抑郁症的临床2b期试验。孕烯醇酮是一种内源性神经类固醇，具有经临床验证的快速抗抑郁和抗焦虑活性，虽然在一系列神经精神疾病中具有改善作用，但是它仅被批准为静脉注射，限制了其临床使用范围； SPT-320 ：一款新型阿戈美拉汀前药，目前正在用于治疗广泛性焦虑症，有可能成为数十年来治疗GAD的首个新机制。利用Glyph平台技术，SPT-320绕过肝脏的首过代谢，有潜力降低其有效剂量、减少肝脏暴露，降低对肝功能监测的需求，而这正是限制阿戈美拉汀使用的瓶颈； SPT-348 ：一款非致幻性神经塑性剂的前药，正在开发中用于治疗情绪和其他神经精神疾病。此外，Seaport还有多个临床前项目，其中包括Seaport之前披露的口服 CBD项目。公司CEO Daphne Zohar之前担任PureTech Health的创始人兼首席执行官并共同创立了Karuna Therapeutics，她从16岁时开始创业，PureTech Health自创立以来，目前已开发出了28种疗法。另一位联合创始人是Karuna Therapeutics前首席执行官、前首席科学官Steven Paul，此前亦曾担任礼来的研发总裁，负责Zyprexa、Cymbalta、xanomeline等CNS药物的开发。 2024年4月， Seaport Therapeutics宣布完成1亿美元的A轮融资，本轮融资由ARCH Venture、Sofinova Investments、Third Rock和PureTech Health共同领投。 2024年10月， Seaport Therapeutics宣布完成2.25亿美元的B轮融资，本轮融资由General Atlantic领投，T.Rowe Price Associates、Foresite Capital、Invus、Goldman Sachs Alternatives、ARCH Venture、Sofinnova、Third Rock Ventures、PureTech Health跟投。 3、Odyssey Therapeutics(NASDAQ:ODTX) 5月8日， Odyssey Therapeutics 在纳斯达克交易所上市，发行价为18美元，发行1550万股，募资2.79亿美元，累计融资规模达9.79亿美元（约合66.21亿元）。 Odyssey Therapeutics是一家专注于发现、开发和商业化下一代免疫调节剂和肿瘤药物的生物技术公司，曾入选BioSpace评选出 NextGen Bio“Class of 2023”榜单。 Odyssey最早于2025年1月秘密递交IPO申请，但随后生物科技IPO市场急剧转冷，2025全年仅9家生物制药公司完成IPO，募资总额16亿美元，为近五年来最低。2025年6月，Odyssey正式撤回IPO申请。目前，Odyssey正在构建一个将人工智能和机器学习相结合用于分子设计的发现引擎，一个包含针对多种氨基酸、分子胶和天然产物的专有共价库的化学平台，以及用于新靶点发现的功能基因组学平台。管线方面，Odyssey正在迅速推进免疫学和肿瘤学项目，重点关注那些经过有力验证但尚无市场化解决方案的靶点，包括小分子和蛋白质疗法在内的8个项目： OD-001: 一款口服小分子RIPK2支架抑制剂，针对溃疡性结肠炎和克罗恩病开发，目前正处于2期临床试验阶段。RIPK2是先天性免疫系统的关键信号蛋白，能对消化道细菌副产物做出反应，与传统生物制剂相比，靶向RIPK2的覆盖面更广；与JAK抑制剂相比，选择性更高，有望规避JAK类药物常见的系统性副作用风险。在试验设计上，公司采取了单药与联合用药双线推进策略：单药治疗2期顶线数据预计2026年上半年公布；联合武田Entyvio的2a期研究预计2027年年中公布联用诱导结果。 OD-002 ：一款口服小分子SLC15A4抑制剂，针对系统性红斑狼疮、干燥综合征、皮肌炎等B细胞介导疾病开发，目前处于临床前阶段。OD-002通过选择性阻断TLR7/8/9介导的IRF5活化，靶向上游免疫反应通路，有望减少现有疗法的免疫抑制风险。目前，全球尚无获批的SLC15A4靶向药物，OD-002一旦成功，将具备首创新药（First-in-class）的先发优势。此外，公司还布局了IRAK4支架抑制剂（针对特应性皮炎、化脓性汗腺炎）、TNFR2激动剂（针对系统性红斑狼疮、斑秃）、TSLP/IL-33双特异性拮抗剂（针对哮喘、COPD）等多条管线，形成了从2期临床到早期发现的完整梯队。在一级市场，Odyssey Therapeutics受到资本追捧，自2021年末成立以来筹集的总资本达到7亿美元之多： 2021年12月， Odyssey Therapeutics完成了2.18亿美元的A轮融资。A轮融资由 OrbiMed Advisors与SR One共同领投，Foresite Capital、Woodline Partners、Logos Capital、HBM Healthcare、Colt Ventures、Creacion Ventures等机构跟投。 2022年10月， Odyssey Therapeutics完成了1.68亿美元的B轮融资。B轮融资由 General Catalyst领投，Fidelity，GreatPoint Ventures，Walleye Capital，The Healthcare Innovation Investment Fund LLC（与SVB Securities LLC相关的投资基金）等机构跟投。 2024年12月， Odyssey Therapeutics 宣布完成 1.01亿美元的C轮融资。本轮融资 Ascenta Capital领投，OrbiMed、 GSK旗下的SR One 、Foresite Capital、HBM Healthcare、Fidelity、T.Rowe Price Associates、Catalio Capital 、Alexandria Venture等机构跟投。 2025年9月， Odyssey Therapeutics宣布完成2.13亿美元D轮融资。本轮融资的投资人包括 Affinity Asset、Dimension Capital、Jeito Capital、Lightspeed Ventures、TPG Life Sciences Innovations和Wedbush Healthcare Partners等机构。二、反向并购上市（ Reverse Merger ） 5月，有一家公司采用反向并购形式上市。 1、InMed(NASDAQ:INM) 5月19日，美股上市公司 InMed 宣布与 Mentari Therapeutics 达成最终合并协议。合并后公司的预计股权价值约为4.214亿美元，原Mentari股东将持有合并后公司约98.5%的绝对控股权益，原InMed股东仅持有约1.51%的股权。在合并的同时，Mentari Therapeutics已获得超额认购的2.9亿美元的私募融资。本轮融资由Paragon的创始投资方Fairmount领投，参与方包括Commodore Capital、Deep Track、Janus Henderson Investors、a16z Bio+Health、Venrock Healthcare、Wellington Management、Blackstone Multi-Asset Investing、BB Biotech、Farallon Capital、RTW Investments、Vivo Capital和Perceptive Advisors等知名机构。 Mentari Therapeutics 是一家专注于开发偏头痛创新疗法的公司，也是 Paragon Therapeutics孵化设立的第八家上市公司。 Paragon Therapeutics由投资机构Fairmount于2021年创立，是一家生物制品工程和研发公司，采取Hub-and-Spoke模式，聚焦慢病尤其是自免疾病，针对成熟靶点开发迭代的长效抗体药物，技术上也多采用专利过期的YTE改造策略。 Paragon 分拆孵化公司的效率很高，多以反向并购的方式上市，目前已有七家Biotech上市公司，合计市值达 190亿美元：第一家上市公司Apogee Therapeutics(NASDAQ:APGE)成立于2022年2月，2023年7月完成3亿美元的IPO，聚焦于超长半衰期的自免药物研发，目前市值约 51亿美元；第二家上市公司Spyre Therapeutics (NASDAQ:SYRE) 成立于2023年，聚焦炎症性肠病领域，2023年11月完成借壳Aeglea(NASDAQ:AGLE)上市，目前市值约 65亿美元；第三家上市公司Oruka Therapeutics(NASDAQ:ORKA)自2024年4月对外披露，9月完成借壳ARCA biopharma (NASDAQ:ABIO) 上市，聚焦自免皮肤疾病，开发长效IL-23p19抗体、IL-17A/F抗体等，目前市值约 38亿美元；第四家上市公司 Jade Biosciences(NASDAQ:JBIO)于2024年10月，与 Aerovate(NASDAQ:AVTE)合并上市，聚焦于 IgA肾病领域，目前市值约 13亿美元；第五家上市公司 Crescent Biopharma(NASDAQ:CBIO) 于2024年10月，与GlycoMimetics（NASDAQ:GLYC）合并上市，聚焦于 PD-L1xVEGF双抗药物，目前市值约 5亿美元；第六家上市公司 Damora Therapeutics （NASDAQ:DMRA）于2025年11月，与 Galecto(NASDAQ:GLTO)合并上市，聚焦于 mutCALR单抗药物，目前市值约 15亿美元。第七家上市公司 Korsana Biosciences于2026年4月宣布与 Cyclerion （NASDAQ：CYCN）合并上市，聚焦于中枢神经系统药物的研发，预计合并后市值为4.2亿美元。在偏头痛领域，以CGRP抑制剂为代表的新疗法已显著改善了治疗现状，但仍有40%-50%的患者存在用药无效、疗效衰减或耐受性差等问题，临床替代疗法缺口巨大。公司布局了两款差异化、互补性极强的首创潜力抗体药物，构建了覆盖CGRP和PACAP两条核心致病通路的研发管线： MT-001 ：一款靶向PACAP通路的单克隆抗体。PACAP是近年偏头痛病理机制领域的全新验证靶点，作用机制完全独立于传统CGRP通路，可精准覆盖对CGRP抑制剂无应答的难治性偏头痛患者。 MT-001已于2026年中完成临床试验申请筹备，即将开展首次人体临床试验，预计2028年发布2a期概念性验证数据，有望成为难治性偏头痛治疗的全新突破口。 MT-002 ：一款同时靶向CGRP+PACAP的双特异性抗体，具备同类首创（First-in-class）潜力。该药物可同时阻断两条偏头痛致病核心通路，实现双重机制抑制，有望大幅提升治疗有效率、降低复发概率，特别适配中重度、反复发作的顽固性偏头痛患者。目前MT-002已完成临床前非人灵长类动物验证，安全性与靶点结合活性表现优异，预计2027年完成健康受试者一期临床数据读出，研发进度处于全球第一梯队。当前全球偏头痛药物市场仍由CGRP抑制剂主导，安进/诺华的Aimovig、梯瓦的Ajovy、礼来的Emgality和灵北的Vyepti等产品占据了大部分市场份额。然而，随着CGRP靶点药物专利陆续到期，以及大量患者对现有疗法应答不足，市场对全新机制药物的需求日益迫切。本文内容只供做参考之用，并不构成投资建议，任何人依据本文所做的投资决策所造成的一切后果自负。点击关注新康界，获取更多热点分析/行研报告/蓝皮书。【免责声明】 1.“新康界”部分文章信息来源于网络转载是出于传递更多信息之目的，并不意味着赞同其观点或证实其内容的真实性。如对内容有疑议，请及时与我司联系。 2.“新康界”致力于提供合理、准确、完整的资讯信息，但不保证信息的合理性、准确性和完整性，且不对因信息的不合理、不准确或遗漏导致的任何损失或损害承担责任。 3.“新康界”所有信息仅供参考，不做任何商业交易及或医疗服务的根据，如自行使用“新康界”内容发生偏差，我司不承担任何责任，包括但不限于法律责任，赔偿责任。 4.本声明未涉及的问题参见国家有关法律法规，当本声明与国家法律法规冲突时，以国家法律法规为准。

100 项与伏尼凝血素α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D08681	伏尼凝血素α	B02BD10	DB12872

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
1型血管性血友病	美国	Takeda Pharmaceuticals U.S.A., Inc.	2025-09-05
2型血管性血友病	美国	Takeda Pharmaceuticals U.S.A., Inc.	2025-09-05
冯·维勒布兰德病	欧盟	Baxalta Innovations GmbH	2018-08-31
冯·维勒布兰德病	冰岛	Baxalta Innovations GmbH	2018-08-31
冯·维勒布兰德病	列支敦士登	Baxalta Innovations GmbH	2018-08-31
冯·维勒布兰德病	挪威	Baxalta Innovations GmbH	2018-08-31
血管性血友病3型	美国	Takeda Pharmaceuticals U.S.A., Inc.	2015-12-08

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
出血	临床3期	美国	Baxalta, Inc.	2011-11-01
出血	临床3期	日本	Baxalta, Inc.	2011-11-01
出血	临床3期	澳大利亚	Baxalta, Inc.	2011-11-01
出血	临床3期	奥地利	Baxalta, Inc.	2011-11-01
出血	临床3期	比利时	Baxalta, Inc.	2011-11-01
出血	临床3期	保加利亚	Baxalta, Inc.	2011-11-01
出血	临床3期	加拿大	Baxalta, Inc.	2011-11-01
出血	临床3期	德国	Baxalta, Inc.	2011-11-01
出血	临床3期	印度	Baxalta, Inc.	2011-11-01
出血	临床3期	意大利	Baxalta, Inc.	2011-11-01

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04344860 (VWD-Woman, Pubmed \| Blood Adv)	临床3期	1型血管性血友病	20	Tranexamic acid + recombinant VWF	製簾窪鏇鹹鹽淵遞選簾(簾夢顧齋積製鏇鹽醖憲): P-Value = 0.49 更多	不佳	2025-12-09
				Recombinant VWF
ASH2025	N/A	冯·维勒布兰德病	9	Recombinant von willebrand factor (severe VWD)	獵鹽獵艱淵選衊齋蓋顧(鑰鏇構鏇醖襯糧築齋蓋) = There were no reported adverse events defined by the European Haemophilia Safety Surveillance program, including treatment-emergent side effects, hypersensitivity/allergic reactions, thrombotic events, or events of VWF inhibitor development. 鬱範繭衊襯衊範簾廠顧 (鏇築簾壓齋餘繭鹽簾構 )	积极	2025-12-06
NCT04344860 (VWD Woman Trial \| VWD-Woman, CTgov)	临床3期	冯·维勒布兰德病	20	Recombinant Von Willebrand factor+Tranexamic Acid Injection [Cyklokapron] (rVWF plus TA)	窪製壓窪衊遞窪衊繭鬱(襯遞憲鏇膚憲艱蓋觸齋) = 鹽壓廠餘觸廠顧構膚遞糧簾範憲範衊範醖蓋範 (鬱鹽蓋餘構壓積積鑰齋, 802.5) 更多	-	2025-10-31
				Recombinant Von Willebrand factor (rVWF alone)	窪製壓窪衊遞窪衊繭鬱(襯遞憲鏇膚憲艱蓋觸齋) = 窪淵憲選衊範鹽構廠繭糧簾範憲範衊範醖蓋範 (鬱鹽蓋餘構壓積積鑰齋, 355.08) 更多
ASH2024	N/A	冯·维勒布兰德病	-	Continuous prophylaxis with recombinant VWF	繭顧構築構襯遞淵糧鏇(簾繭鏇齋積鬱艱齋壓壓) = One participant reported 9 adverse events (AEs); all AEs were resolved and determined to be unrelated to rVWF treatment 淵觸膚鹽選鬱餘繭蓋選 (獵廠獵窪齋膚憲廠鹹鑰 ) 更多	-	2024-12-08
NCT02973087 (Pubmed \| Circulation \| Blood) 人工标引	临床3期	冯·维勒布兰德病	23	Recombinant von Willebrand factor (Prior On-Demand group)	鑰構獵顧憲餘蓋鹹鹹淵(醖淵鹹醖齋遞壓淵蓋憲) = 糧窪壓憲膚網壓衊壓簾膚窪獵鹹憲蓋餘鬱構壓 (鏇鑰蓋鑰觸鬱艱壓廠觸 )	积极	2022-04-19
				Recombinant von Willebrand factor (Switch group)	鑰構獵顧憲餘蓋鹹鹹淵(醖淵鹹醖齋遞壓淵蓋憲) = 膚餘餘鑰網憲觸積鹹製膚窪獵鹹憲蓋餘鬱構壓 (鏇鑰蓋鑰觸鬱艱壓廠觸 )
NCT02973087 (CTgov)	临床3期	冯·维勒布兰德病	29	VWF concentrate+rVWF (vonicog alpha)+ADVATE (rFVIII) (Prior On-demand Participants)	鑰鹹顧獵構壓鏇餘廠夢(齋鏇壓積襯襯範範獵觸) = 鑰醖襯齋獵築齋顧窪鬱蓋積蓋築齋蓋糧範鑰衊 (餘鏇製淵廠壓獵鹽構構, 鬱壓鏇夢選觸廠選鑰膚 ~ 築餘襯範構壓糧膚簾築) 更多	-	2021-08-06
				VWF concentrate+rVWF (vonicog alpha)+ADVATE (rFVIII) (Switch Participants)	鑰鹹顧獵構壓鏇餘廠夢(齋鏇壓積襯襯範範獵觸) = 積簾壓餘膚鑰窪窪鑰鬱蓋積蓋築齋蓋糧範鑰衊 (餘鏇製淵廠壓獵鹽構構, 製鬱齋憲網艱衊製醖觸 ~ 積網範鏇遞築鑰鏇網蓋) 更多
ISTH2020	N/A	冯·维勒布兰德病	8	Recombinant von Willebrand Factor (rVWF)	鹽壓壓廠網衊夢構積糧(餘製製遞淵餘膚壓構築) = 範壓鹽艱夢壓淵衊顧鏇夢簾衊積鏇構繭憲鏇範 (鬱壓鬱夢衊觸遞鹽構觸 ) 更多	-	2020-07-12
NCT01410227 (ISTH2019)	临床3期	血管性血友病3型	15	Recombinant von Willebrand factor (rVWF)	憲構鬱鏇繭築鑰繭遞製(蓋鬱糧顧鹹壓遞簾鹽艱) = 艱顧製餘製築餘願淵壓艱餘廠網鬱鏇網願蓋齋 (獵築醖鬱繭醖網艱廠鹹 ) 更多	-	2019-06-10