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DURHAM, N.C., April 23, 2025 /PRNewswire/ -- Baebies has received Breakthrough Device Designation from the U.S. Food and Drug Administration (FDA) for its Anti-Factor Xa test on the FINDER® platform —the first point-of-care heparin monitoring assay. Designed for patients receiving unfractionated or low molecular weight heparin, the test provides results in under 15 minutes from just 50 µL of whole blood. This designation recognizes the test's potential to bring heparin monitoring closer to the patient, enabling faster and more effective dose management in critical care settings. Continue Reading Baebies FINDER Anti-Factor Xa | Cartridge Protocol | FDA Breakthrough Device FINDER Multifunctional Testing - One Platform, Multiple Types of Tests, Delivering Rapid Results for Near-Patient Use FINDER in Action: Simple 4-Step Workflow "This designation provides priority FDA review and dedicated regulatory guidance, accelerating our efforts to bring the test to market," said Richard West, Founder and CEO of Baebies. "It positions FINDER® to expand rapidly into the multi-billion-dollar coagulation testing space." Heparin dosing requires tight control to prevent both bleeding and clotting complications. In high-risk settings such as extracorporeal membrane oxygenation (ECMO), mortality rates are often around 50%, and bleeding occurs in over a third of patients. Anti-Factor Xa tests—the preferred method for guiding therapy—are confined to central labs and often take over an hour, delaying time-sensitive dosing. "It is rewarding to see our long-standing, NIH-funded collaboration with Baebies achieve FDA breakthrough," said Dr. Sitaram Emani, Associate Professor of Surgery at Harvard Medical School. "I look forward to the test supporting timely anticoagulation decisions and advancing heparin management." Unlike existing tests, the FINDER Anti-Factor Xa Test requires no centrifugation and can be run directly at the point of care or in a central lab, delivering results in minutes. It addresses a life-threatening condition, offers significant advantages over existing methods, introduces breakthrough technology where no cleared alternatives exist, and serves the best interest of patients—meeting the FDA Breakthrough Device criteria. "We're growing beyond our pediatric roots to serve broader populations by bringing multifunctional diagnostics to patients of all ages, wherever care is delivered," said Vamsee Pamula, PhD, Founder and President of Baebies . Baebies is a diagnostics company headquartered in Research Triangle Park, North Carolina. With over 22 million tests delivered globally, Baebies is a technology leader that pioneered digital microfluidics (DMF). Its multifunctional FINDER® platform supports chemistry, immunoassay, molecular, and coagulation testing from small-volume samples. Baebies was awarded the ADLM Disruptive Technology Award. Driven by its mission—Any Test, Anywhere, Everyone™—Baebies is shaping a future where diagnostic testing is accessible everywhere, delivering better healthcare for everyone—across every age and setting. Media Contact: Baebies Communications Team [email protected] 919-328-8389 SOURCE Baebies Inc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

HEMLIBRA has strong market potential due to its unique, subcutaneous prophylactic treatment for Hemophilia A, including patients with and without factor VIII inhibitors. With increasing global diagnosis rates and expanding access in emerging markets, HEMLIBRA is poised for continued growth. LAS VEGAS, April 21, 2025 /PRNewswire/ -- DelveInsight's " HEMLIBRA Market Size, Forecast, and Market Insight Report" highlights the details around HEMLIBRA, a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of HEMLIBRA. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Chugai/Genentech/Roche's HEMLIBRA (emicizumab) Overview HEMLIBRA is a bispecific antibody that targets both factor IXa and factor X, two key proteins in the body's natural blood clotting process. Bridging these proteins it helps restore clotting function in individuals with hemophilia A. HEMLIBRA is used as a preventive treatment and is administered as a ready-to-use subcutaneous injection. It can be given once a week, every two weeks, or every four weeks—following an initial four-week period of weekly doses. Developed by Chugai Pharmaceutical Co., Ltd., HEMLIBRA is co-developed internationally by Chugai, Roche, and Genentech. In the U.S., Genentech markets the drug under the name HEMLIBRA (emicizumab-kxwh), with "kxwh" assigned per FDA guidance on naming biological products. HEMLIBRA can be prescribed in various dosage regimens. The initial loading dose is 3 mg/kg administered via subcutaneous injection once weekly for the first four weeks. This is followed by one of the following maintenance dosing options: 1.5 mg/kg once every week, 3 mg/kg once every two weeks, or 6 mg/kg once every four weeks The choice of maintenance dose should be guided by the healthcare provider's judgment, taking into account the potential for improved patient adherence with certain regimens. The prophylactic use of bypassing agents should be stopped the day before initiating HEMLIBRA prophylaxis. Factor VIII (FVIII) prophylaxis may be continued during the first week of HEMLIBRA treatment. Learn more about HEMLIBRA projected market size for hemophilia A @ HEMLIBRA Market Potential Hemophilia A is marked by bleeding episodes, though in mild and moderate cases, these initial episodes are often less intense, which can lead to delays in diagnosis as patients may not seek medical attention promptly. Moreover, early detection is frequently hindered by the lack of access to adequate diagnostic facilities. In 2023, the number of treated prevalent Hemophilia A cases across the 7MM was estimated at around 45,000, with projections indicating growth throughout the forecast period, according to DelveInsight. Among hemophilia A patients—whether or not they have inhibitors—those without inhibitors represent a larger portion of the population. The standard treatment approach focuses on replacing the missing clotting factor, FVIII, to support normal clot formation and minimize or prevent bleeding episodes. Initially, FVIII replacement was sourced from whole blood and plasma, but advances in biotechnology led to the development of recombinant FVIII (rFVIII) therapies, significantly enhancing disease management by offering more precise and efficient options. More recently, innovative therapies using novel mechanisms to prevent bleeding are expanding the treatment arsenal. Several approved therapies for hemophilia A include ALTUVIIIO (Sanofi/Sobi), ROCTAVIAN (BioMarin), OBIZUR (Takeda), ALHEMO (Novo Nordisk), HEMLIBRA (Chugai/Genentech/Roche), ESPEROCT (Novo Nordisk), JIVI (Bayer), WILATE (Octapharma), and ADYNOVATE (Takeda), among others. Notably, ROCTAVIAN, a gene therapy developed by BioMarin, represents a major advancement—it offers a one-time treatment by introducing functional FVIII genes into the patient's cells, potentially restoring normal clotting ability and reducing the need for regular injections. With a wholesale acquisition cost of USD 2.9 million, BioMarin has already facilitated reimbursement in Europe and administered treatment to a patient in Germany in Q2 2023 at a discounted price of approximately USD 900,000. Looking ahead, the hemophilia A treatment landscape is expected to evolve significantly from 2024 to 2034, fueled by the introduction of new therapies currently in development. According to DelveInsight, the hemophilia A market across the 7MM is projected to grow from USD 11.1 billion in 2023 at a robust CAGR through 2034. Nonetheless, factors like delayed diagnosis, limited awareness, and high treatment costs may pose barriers to market growth. Discover more about the hemophilia A market in detail @ Hemophilia A Market Report Emerging Competitors of HEMLIBRA Emerging therapies such as SPK-8011 (Roche [Spark Therapeutics]), fitusiran (Sanofi), marstacimab (Pfizer), Mim8 (Novo Nordisk), and others are being developed to provide safe and effective treatment options. Companies like GeneVentiv Therapeutics are also working on advancements like Adeno-associated Virus (AAV)-based universal gene therapy for hemophilia (GENV-HEM [AAV8.Fva]). Fitusiran is a subcutaneously administered small interfering RNA (siRNA) therapy under development for the prophylactic treatment of hemophilia A and B, regardless of the presence of inhibitors. Its mechanism involves reducing levels of antithrombin—a protein that inhibits clot formation—to enhance thrombin generation, restore hemostatic balance, and prevent bleeding episodes. In January 2018, Sanofi and Alnylam Pharmaceuticals restructured their RNAi therapeutics collaboration to better align and accelerate the development and commercialization of select treatments for rare genetic diseases. Through this revised agreement, Sanofi obtained global rights to develop and commercialize fitusiran. Marstacimab (PF-06741086) is a monoclonal IgG1 antibody that targets the Kunitz 2 domain of the Tissue Factor Pathway Inhibitor (TFPI). It is being developed to prevent or reduce bleeding events in patients with severe hemophilia A or B (defined as <1% activity of Factor VIII or IX), whether or not they have inhibitors. The FDA granted Fast Track Designation to marstacimab in September 2019 for its potential use in patients with inhibitors. In December 2023, Pfizer announced that the FDA had accepted its Biologics License Application (BLA) for marstacimab, supported by data from the Phase III BASIS study involving patients without FVIII or FIX inhibitors. BBM-H803 is Belief BioMed's first gene therapy candidate for hemophilia A and its second product to gain Investigational New Drug (IND) approval from China's National Medical Products Administration (NMPA). In December 2022, the U.S. FDA granted Orphan Drug Designation to BBM-H803. To know more about the number of competing drugs in development, visit @ HEMLIBRA Market Positioning Compared to Other Drugs Key Milestones of HEMLIBRA In June 2022, Chugai received approval in Japan for acquired Hemophilia A based on a domestic Phase III AGEHA study. In October 2018, HEMLIBRA was approved by the US FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors (FVIII). However, this therapy, which was co-developed by Genentech, Chugai, and Roche, was already FDA-approved in 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors In April 2018, the US FDA granted breakthrough therapy designation to Genentech's HEMLIBRA for people with hemophilia A without factor VIII inhibitors. In October 2014, the US FDA granted orphan drug designation to HEMLIBRA for the treatment of Hemophilia A. Discover how HEMLIBRA is shaping the hemophilia A treatment landscape @ HEMLIBRA Cost HEMLIBRA Market Dynamics HEMLIBRA developed by Roche/Genentech, has significantly reshaped the hemophilia A treatment landscape since its approval. Designed as a bispecific monoclonal antibody, HEMLIBRA mimics the function of factor VIII by bridging activated factor IX and factor X, offering prophylactic treatment for patients with hemophilia A with or without factor VIII inhibitors. Its subcutaneous administration and less frequent dosing schedule (weekly, biweekly, or monthly) offer considerable convenience compared to traditional intravenous factor replacement therapies, making it a preferred option for both patients and caregivers. The market dynamics for HEMLIBRA have been largely influenced by its clinical efficacy, improved patient compliance, and strong safety profile. Since its launch, it has rapidly gained market share, especially among inhibitor patients where treatment options were limited and complex. In non-inhibitor patients as well, the shift away from frequent IV infusions has led to strong adoption rates, allowing HEMLIBRA to cannibalize traditional recombinant factor VIII therapies. Furthermore, its entry has pressured pricing and market share dynamics across the hemophilia therapeutic space, triggering strategic shifts by legacy players like Takeda, Sanofi, and Bayer. However, HEMLIBRA's market leadership is being challenged by the evolving competitive landscape. The emergence of gene therapies, which offer the potential for long-term or even curative outcomes, introduces a new dimension to treatment decisions. While some gene therapies are still in early stages of adoption and face challenges related to durability, safety, and patient eligibility, they represent a looming threat to the long-term dominance of HEMLIBRA, particularly in younger, eligible patient populations. Going forward, the market trajectory for HEMLIBRA will depend on several factors: expanding label indications (such as pediatric use), long-term safety and efficacy data, continued pricing negotiations with payers, and how effectively Roche can defend its position against next-generation therapies. Additionally, with increasing emphasis on patient-centric outcomes and value-based care, HEMLIBRA's ability to demonstrate long-term cost-effectiveness versus both traditional and novel competitors will be key to sustaining its market share. Dive deeper to get more insight into HEMLIBRA's strengths & weaknesses relative to competitors @ HEMLIBRA Market Drug Report Table of Contents Related Reports Hemophilia A Market Hemophilia A Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia A companies, including BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical, among others. Hemophilia A Pipeline Hemophilia A Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including UniQure Biopharma B.V., CSL Behring, Pfizer, Spark Therapeutics, Genzyme, a Sanofi Company, Alnylam Pharmaceuticals, Novo Nordisk, ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, among others. Hemophilia B Market Hemophilia B Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key hemophilia B companies such as Gem Pharmaceuticals, Lytix Biopharma, Incyte Corporation, Daiichi Sankyo, NantPharma, Iovance Biotherapeutics, Agenus, Eli Lilly and Company, Adaptimmune, Aadi, AVEO Pharmaceuticals, Amgen, among others. Hemophilia B Pipeline Hemophilia B Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including Shanghai Vitalgen BioPharma, Belief BioMed, TiumBio, Jiangsu Gensciences, Centessa Pharmaceuticals, Takeda, Sanofi, GC Biopharma Corp, Equilibra Bioscience LLC, Regeneron Pharmaceuticals, ISU Abxis, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

-01-引言淋巴系统的运输维持健康的稳态，也是免疫监测所必需的，但淋巴生长通常与实体瘤的发展和扩散有关。肿瘤相关的淋巴管为淋巴结转移提供了必要的途径，淋巴管密度通常随着淋巴管增殖过程的进展而增加，称为淋巴管生成。人们最初认为肿瘤相关的淋巴重塑和生长只是扩大了区域转移的被动途径。然而，最近的临床数据未能证明立即完全淋巴结切除对总生存率的益处，并且在淋巴结清扫前给予的新辅助治疗对多种肿瘤类型具有效果。此外，新出现的临床前研究表明，肿瘤引流淋巴结是肿瘤特异性免疫的蓄水池，当其被转移抑制，会削弱系统免疫监视。这些数据共同表明，有必要重新思考区域疾病管理及其对免疫疗法患者结果的影响。这些研究表明，淋巴系统已成为宿主对癌症反应的关键功能组成部分，它积极调节液体、抗原、白细胞和肿瘤细胞向引流淋巴结的输送。因此，系统地了解淋巴功能在恶性肿瘤期间是如何协同作用的以辅助免疫监测，对于当前免疫治疗时代的癌症治疗至关重要。-02-一、淋巴系统的结构和稳态功能淋巴系统调节液体和细胞从外周组织单向缓慢流动，通过淋巴结（LNs）和胸导管流回血液，淋巴系统对于去除由毛细管滤液产生的间质液至关重要。淋巴毛细血管是钝端薄壁血管，是液体摄取和白细胞进入的部位。毛细血管中的淋巴内皮细胞（LECs）在叉指状皮瓣处形成特殊的不连续的内皮间连接，称为“Button”，由粘附物（血管内皮钙粘蛋白）和紧密连接分子（claudin-5，JAM-A， ESAM和occludin）组成，通过支架蛋白，如ZO1和β-连环蛋白，锚定在肌动蛋白细胞骨架上。锚定丝将LEC连接到细胞外基质（ECM），并在组织肿胀时打开LEC连接，从而形成淋巴液。淋巴液被转移到收集血管中，通过基底膜、肌肉细胞、连接和瓣膜运动将液体和白细胞定向输送进入LN包膜下窦。淋巴管平时处于稳定状态，在炎症的情况下，是将外周组织状态传达给引流淋巴管的关键通道。这些淋巴管协调单向流体、大分子和细胞摄取、运输和LN内的分选，从而直接调节免疫监测从启动到分辨和记忆的周期性阶段。淋巴管还表现出重要的组织特异性功能，例如淋巴管对于胃中的膳食脂质吸收、皮肤中的电解质清除、棕色脂肪组织中的产热和肺的膨胀是非常必要的。此外，淋巴管分泌因子通过调节干细胞周期来调节心脏、皮肤和骨骼中的组织再生。-03- 二、肿瘤相关淋巴管生成肿瘤微环境（TME）中淋巴管密度的增加是由淋巴管生成过程驱动的，TME中的淋巴血管生成足以增强LN转移。两种关键的淋巴管生成生长因子VEGFC和VEGFD通过VEGFR3及其共受体neuropilin-2发出信号，促进LEC增殖、迁移和存活以及LN转移。肿瘤表达的VEGFC驱动淋巴管生成和LN转移，同时也促进巨噬细胞的募集。在骨肉瘤小鼠模型中，表达VEGFC的巨噬细胞和肿瘤相关淋巴管生成呈正相关，抑制巨噬细胞集落刺激因子（CSF1）减少肿瘤相关巨噬细胞（TAMs）可有效抑制病理性淋巴管生成。因此，LECs和髓系细胞似乎参与双向串扰，这种串扰机制表明，局部炎症会随着时间的推移加剧淋巴重塑，这可能会前馈影响正在进行的先天和适应性免疫反应。此外，VEGFC或VEGFD驱动局部生长的程度可能取决于局部细胞因子环境中的其他因素。LECs表达多种细胞因子和趋化因子受体，通过这些受体感知炎症并对其作出反应，与炎症环境发生动态串扰。1型细胞因子如IL-1β、IL-12和肿瘤坏死因子（TNF）以及某些趋化因子，如CXCL12，具有促淋巴管生成作用；而2型细胞因子，如IL-4和IL-13，似乎是淋巴管生成的负调节因子。最后，对发育和病理性淋巴管生成的机制研究产生了新的治疗靶点。然而，尽管阻断VEGFC–VEGFD–VEGFR3信号传导的几种治疗方法显示出良好的安全性，但迄今为止在患者中仅观察到微弱的疗效。在没有持续刺激的情况下，成人组织中淋巴增生的代偿性信号机制和持续性可能是导致临床缺乏成功的原因。因此，淋巴管生长、维持和存活的额外机制，例如血管生成素-2、成纤维细胞生长因子2（FGF2）、胰岛素样生长因子（IGFs）和代谢途径，可能成为淋巴管靶向治疗的替代策略。-04-三、淋巴管生成与免疫治疗最早在十多年前就报道了第一批测试淋巴管生成对肿瘤相关炎症和免疫监测影响的实验。这些初步研究表明，VEGFC在B16-F10黑色素瘤中的过表达增强了LN转移，并诱导了几种免疫逃逸机制。这些数据似乎表明，扩张的淋巴管系统可能会限制正在进行的抗肿瘤免疫反应。然而，也有人提出淋巴管生成在免疫治疗中可能是有益的。在黑色素瘤和脑癌的小鼠模型中使用VEGFC诱导淋巴生长确实导致了对多种免疫治疗剂的增强反应，包括疫苗、过继性T细胞转移和免疫检查点阻断（ICB）。淋巴管生成与肿瘤免疫治疗之间似乎存在着矛盾。因此，与其说淋巴管是好是坏，不如说是淋巴管生长、转运及其表型和转录状态的调节决定了肿瘤免疫监测的最佳环境。与时间相关的变化限制了系统免疫监测和对免疫疗法的反应。现有证据表明：（1）肿瘤相关淋巴管是新生适应性免疫所必需的，可促进抗原传播；（2） 用VEGFC驱动肿瘤内淋巴生长可以促进抗原呈递，但其益处被局部炎症加剧、进行性免疫功能障碍和转移所抵消；（3） 淋巴管促进淋巴细胞从肿瘤微环境中再循环，并促进免疫消解；（4）淋巴转运，最终转移本身使LN微环境具有更强的抑制作用，从而损害系统免疫监测。基于这些证据，我们不能对肿瘤相关淋巴管生成作用的进行简单的二元化，这是一个与时间相关的框架，通过该框架，淋巴管系统的渐进激活和生长可以将TME内的平衡转向慢性炎症、驱动免疫消解的淋巴流出和转移。因此，淋巴管生成本身可能不是正确的靶点。相反，也许我们应该考虑如何开启淋巴管用于促进主动免疫监测的特定机制，或者将对淋巴转运何时何地起作用的机制理解作为当前临床免疫疗法的设计原则。-05-结语长期以来，肿瘤相关淋巴管系统一直被视为一组功能失调的血管，它们只允许肿瘤细胞从原发肿瘤被动迁移到引流LN。在十多年的时间里，这种观点发生了明显的变化。现在，淋巴管开始因其对组织稳态、再生和免疫监测的动态和积极贡献而受到重视。正在进行的机制研究开始转向淋巴管及其运输如何促进适应性免疫反应，增强主动免疫监视。未来的工作必须继续深入理解这些淋巴管的位点特异性功能，同时整合淋巴转运和外周组织的系统效应。这些努力不仅将提高我们对这一对人类健康至关重要的系统的基本理解，还将为治疗干预开辟新的途径，为癌症患者带来新的治疗手段。参考资料：1.Lymphatic vessels in the age of cancer immunotherapy. Nat Rev Cancer. 2024 Apr 11公众号内回复“ADC”或扫描下方图片中的二维码免费下载《抗体偶联药物：从基础到临床》的PDF格式电子书！公众号已建立“小药说药专业交流群”微信行业交流群以及读者交流群，扫描下方小编二维码加入，入行业群请主动告知姓名、工作单位和职务。