vWF

STOCKHOLM, April 23, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) presents clinical data aimed at updating the global haemophilia community attending this year's WFH 2025 Comprehensive Care Summit in Dubai from the 23 – 25 April. New and updated outcomes and analyses will be presented from the XTEND phase 3clinical program testing the effectiveness of Altuvoct® (efanesoctocog alfa) treatment for haemophilia A, including updates on patients' joint health and surgical outcomes in patients. "At WFH 2025 CCS, Sobi will present highlights from the XTEND phase 3 program and discuss the new treatment paradigm with FVIII levels in the non-haemophilia range. Sharing these clinical outcomes with the wider haemophilia community will help to ensure the latest treatment approaches are widely known and understood," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. Key data to be presented at WFH 2025 Comprehensive Care Summit, Dubai, UAE Altuvoct® (efanesoctocog alfa) About efanesoctocog alfa Efanesoctocog alfa [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is a novel and significant part of the week factor VIII therapy. It has the potential to deliver near-normal factor activity levels for significant parts of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. It was approved as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the US in February 2023. About the Sobi and Sanofi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, referred to as ALTUVIIIO™ in the USA and ALTOVUCT in Europe. An investigational factor VIII therapy with the potential to provide high sustained factor activity levels with once-weekly dosing for people with haemophilia A. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. Sobi® Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East and Asia. In 2022, revenue amounted to SEK 18.8 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. Contact For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Developed by Sanofi, CABLIVI is the first FDA-approved nanobody therapy for this condition. Increasing awareness, improved diagnostic rates, and rising demand for targeted therapies in hematology drive the market growth. With its niche indication and high treatment costs, CABLIVI holds a strong position in the rare disease therapeutics segment. LAS VEGAS, March 19, 2025 /PRNewswire/ -- DelveInsight's " CABLIVI Market Size, Forecast, and Market Insight Report" highlights the details around CABLIVI, a von Willebrand factor (vWF)-directed antibody fragment. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of CABLIVI. 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]. Ablynx NV's CABLIVI (caplacizumab-yhdp) Overview CABLIVI is an antibody fragment that targets von Willebrand factor (vWF) and is used to treat adult patients with acquired thrombotic thrombocytopenic purpura (aTTP) in combination with plasma exchange and immunosuppressive therapy. It works by binding to the A1-domain of vWF, preventing its interaction with platelets, which helps reduce vWF-mediated platelet adhesion and consumption. CABLIVI is approved in both the US and EU for treating aTTP. In 2024, CABLIVI sales reached €249 million, reflecting a 9.7% increase, driven by a rise in the number of patients identified for suitable treatment in the US, as well as new launches in Europe and other regions. The recommended CABLIVI dosing schedule is as follows: Day 1: Administer an 11 mg intravenous bolus injection at least 15 minutes before plasma exchange, followed by an 11 mg subcutaneous injection after the exchange on the same day. During Plasma Exchange Therapy: Administer 11 mg subcutaneously once daily after each plasma exchange session. After Plasma Exchange Therapy: Continue with 11 mg subcutaneous injections once daily for 30 days after the final plasma exchange. If signs of persistent underlying disease, such as low ADAMTS13 activity, are still present, treatment can be extended for up to 28 additional days. CABLIVI treatment should be discontinued if the patient experiences more than two recurrences of aTTP while on the medication. Learn more about CABLIVI projected market size for thrombocytopenia @ CABLIVI Market Potential Thrombocytopenia is a medical condition where the platelet count in the blood drops below the normal level (<150,000/µL). Common causes include heparin-induced thrombocytopenia, chemotherapy-induced thrombocytopenia, thrombocytopenia linked to chronic liver disease, immune thrombocytopenia (ITP), and thrombotic thrombocytopenic purpura (TTP). Among the 7MM, the United States reported the highest number of thrombocytopenia cases, accounting for about 40% of the total cases in 2023, according to DelveInsight. Treatment for thrombocytopenia varies based on its cause and severity, with the primary goal of preventing bleeding-related complications and improving patient outcomes. Approved therapies for thrombocytopenia in the US include MULPLETA, DOPTELET, TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. Additionally, danaparoid, argatroban, and lepirudin are approved for treating thrombosis in patients with heparin-induced thrombocytopenia. For severe thrombocytopenia related to chronic liver disease, treatment options include platelet transfusion, splenic artery embolization, splenectomy, and transjugular intrahepatic portosystemic stent shunt (TIPSS) placement. The US holds approximately 60% of the thrombocytopenia market share, surpassing the combined market size of the EU4, the UK, and Japan. Market growth is expected to be driven by an increasing patient population, the introduction of new therapies, and deeper market penetration within the 7MM. Discover more about the thrombocytopenia market in detail @ Thrombocytopenia Market Report Emerging Competitors of CABLIVI The potential therapies that can mark a significant change in the thrombocytopenia treatment landscape and give tough competition to CABLIVI include Rilzabrutinib (Sanofi), Ianalumab (Novartis), Mezagitamab (Takeda), PF-06835375 (Pfizer), Povetacicept (Vertex), Cevidoplenib (Genosco/Oscotec), and Efgartigimod (ARGX-113) (Argenx), among others. In December 2024, Sanofi shared encouraging Phase III results at ASH for rilzabrutinib, its investigational oral BTK inhibitor, in patients with ITP. In June 2024, Takeda reported late-breaking Phase IIb data on Mezagitamab, highlighting its potential to revolutionize the treatment of Primary ITP. In Novartis' Q2 2024 update, the company forecasted key trial results: VAYHIT1 (NCT05653349) for first-line therapy, expected in 2026, and VAYHIT2 (NCT05653219) for second-line therapy, anticipated in 2025. Novartis also aims to file for ianalumabin approval in both first- and second-line ITP treatment by 2027 or later. To know more about the number of competing drugs in development, visit @ CABLIVI Market Positioning Compared to Other Drugs Key Milestones of CABLIVI In June 2022, at the 27th Annual European HematologyAssociation (EHA) Congress, Sanofi presented long-term safety and efficacy results from the post-HERCULES trial (NCT02878603), a three-year prospective follow-up study of patients with acquired thrombotic thrombocytopenic purpura (aTTP) who completed the Phase 3 HERCULES trial. In February 2019, the FDA approved CABLIVI (caplacizumab-yhdp) injection as the first therapy specifically indicated for treating adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), a rare and life-threatening blood clotting disorder. It is to be used in combination with plasma exchange and immunosuppressive therapy. In September 2018, the European Commission approved CABLIVI (caplacizumab) for marketing, authorizing its use in treating adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), a rare blood-clotting disorder. CABLIVI is the first therapy specifically designed for aTTP treatment. In July 2017, Ablynx announced a research collaboration and global exclusive licensing agreement with Sanofi, aiming to develop and commercialize Nanobody-based therapies for treating various immune-mediated inflammatory diseases. Discover how CABLIVI is shaping the thrombocytopenia treatment landscape @ CABLIVI FDA Approval CABLIVI Market Dynamics The primary driver of CABLIVI's market growth is the increasing recognition and diagnosis of aTTP, coupled with improved access to specialized treatments. Growing awareness among healthcare providers and advancements in diagnostic capabilities have facilitated early and accurate diagnosis, thereby boosting the demand for targeted therapies like CABLIVI. The drug's ability to prevent microthrombi formation, shorten the duration of plasma exchange, and reduce the risk of relapses has positioned it as a preferred option in the treatment landscape for aTTP. Moreover, Sanofi's robust commercial infrastructure and strategic marketing efforts have further contributed to market penetration and adoption. Despite its therapeutic benefits, CABLIVI faces challenges related to its high cost and reimbursement complexities. The specialized nature of aTTP treatment, combined with the requirement for hospital-based administration and close monitoring, adds to the overall cost burden on healthcare systems and patients. Additionally, competition from alternative therapies, including plasma exchange and immunosuppressive treatments, may limit its market expansion. Regulatory hurdles and the need for long-term safety data could also impact the drug's market trajectory. The CABLIVI market is expected to grow steadily, driven by ongoing clinical research and potential label expansions into related thrombotic disorders. Increased investments in rare disease treatments and orphan drug incentives are likely to support market growth. Moreover, the rise in strategic collaborations and partnerships to improve patient access and streamline reimbursement processes could enhance market uptake. However, balancing pricing pressures with broader patient access will remain a key factor in sustaining long-term market performance. Dive deeper to get more insight into CABLIVI's strengths & weaknesses relative to competitors @ CABLIVI Market Drug Report Table of Contents Related Reports Acquired Thrombotic Thrombocytopenic Purpura Pipeline Acquired Thrombotic Thrombocytopenic Purpura Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acquired thrombotic thrombocytopenic purpura companies, including Sanofi, TargED Biopharmaceuticals, Takeda, Genentech, Alexion Pharmaceuticals, among others. Thrombocytopenia Market Thrombocytopenia 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 thrombocytopenia companies including Sanofi, Principia Biopharma, Baxalta, Takeda, Argenx, Millennium Pharmaceuticals, Biotest, GC Pharma, Genosco (Subsidiary of Oscotec), Rigel Pharmaceuticals, Kissei Pharmaceutical, Shionogi & Co., Ltd, Amgen, Novartis, Zenyaku Kogyo, among others. Thrombocytopenia Pipeline Thrombocytopenia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key thrombocytopenia companies, including Takeda, Argenx, Keros Therapeutics, Principia Biopharma, Momenta Pharmaceuticals, Veralox Therapeutics, Novartis Pharmaceuticals, Pfizer, HUTCHMED, Principia Biopharma, Genosco, UCB, among others. Immune Thrombocytopenia Market Immune Thrombocytopenia 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 immune thrombocytopenia companies including Rigel Pharmaceuticals, Kissei Pharmaceutical, Sobi (Dova Pharmaceuticals), Amgen, Argenx, 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

First participant with Von Willebrand Disease dosed with HMB-002, a novel subcutaneous therapy designed to increase endogenous von Willebrand Factor and Factor VIII levels COPENHAGEN, Denmark and CAMBRIDGE, MA, USA I February 27, 2025 I Hemab Therapeutics, a clinical-stage biotechnology company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders, today announced the first participant with Von Willebrand Disease (VWD) has been dosed in Velora Pioneer, a Phase 1/2 clinical trial investigating HMB-002, a potential first-in-class subcutaneous therapy for VWD. “We are excited to advance the clinical development of HMB-002 for Von Willebrand Disease,” said Catherine Rea, MD, PhD, Vice President of Clinical Development at Hemab. “HMB-002 is the first fixed-dose subcutaneously administered antibody-based preventative therapy aiming to transform daily life for patients.” HMB-002 is a monovalent antibody uniquely designed to increase levels of von Willebrand Factor (VWF) and Factor VIII and provide a long-acting, subcutaneous prophylactic treatment for individuals with all types of VWD. Preclinical data for this investigational therapy were first presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), showcasing its potential to target the underlying patho-etiology of VWD by stabilizing VWF and promoting sustained hemostatic correction. “Von Willebrand Disease is characterized by a deficiency in VWF activity and sometimes associated with lowered levels of FVIII – both essential to control severe bleeding,” said Dr. Priyanka Raheja, the Royal London Hospital, Barts Health NHS Trust. “HMB-002 functions by increasing VWF and FVIII and could offer a complete disease correction for some and substantial modification for bleeding tendency for others. I am excited to be a contributing investigator on the Phase 1/2 trial.” The Phase 1/2 study is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of HMB-002 in individuals with VWD. The initial part of the study will evaluate single ascending doses of HMB-002 in a controlled setting. The first participant was dosed at Richmond Pharmacology in London under the supervision of Principal Investigator, Dr. Ulrike Lorch. The study is actively enrolling participants, with interim data anticipated later this year. “The initiation of the HMB-002 clinical program establishes Hemab as a multi-asset clinical-stage biotech and underscores our unwavering commitment to reimagine blood clotting therapies for people living with a variety of underserved bleeding disorders,” said Benny Sorensen, MD, PhD, CEO of Hemab. “Von Willebrand Disease has been documented for about 100 years. However, the current standard of care is sparse, ancient, and inconvenient. It’s time to leapfrog treatment into the 21 st century, and we believe HMB-002 can become a new back-bone therapy for all types of Von Willebrand Disease.” In addition to Velora Pioneer, Hemab is also conducting Velora Discover , a prospective natural history study collecting data on bleeds, treatments, and quality-of-life elements in people with VWD. Hemab is partnered with Haemnet, a specialist research and communications consultancy in the bleeding disorders community, to conduct VWD 360, the largest-ever natural history study in all types of VWD based on people’s detailed reported lived experience. Recent initial results were shared at EAHAD 2025. About Von Willebrand Disease Von Willebrand Disease (VWD) is the most common inherited bleeding disorder, characterized by quantitative or qualitative defects in Von Willebrand Factor (VWF), often resulting in frequent mucocutaneous bleeding events and heavy menstrual bleeding in women. The severity of bleeding ranges from low-volume events to potentially life-threatening hemorrhages. Chronic blood loss frequently leads to iron deficiency anemia, exacerbating the disease burden and reducing quality of life, particularly for those with clinically understated subtypes. Despite its prevalence, current treatment options for VWD primarily focus on managing symptoms rather than addressing the underlying defect in VWF production or function. About HMB-002 HMB-002 is a monovalent human antibody developed as the first-in-class prophylactic treatment for Von Willebrand Disease targeting the underlying root cause of the disease, a condition driven by a deficiency or defect in Von Willebrand Factor (VWF), a key regulator of hemostasis. By specifically targeting the C-terminal CK domain of VWF, which is distinct from regions critical to its essential interactions, HMB-002 shields the protein from degradation, boosting endogenous levels without compromising its function. Preclinical data suggest strong potential for meaningful therapeutic benefit. For more information, please visit clinicaltrials.gov (NCT06610201). About Hemab Therapeutics Hemab is a multiple clinical-asset biotech company developing novel prophylactic therapeutics for serious, underserved bleeding and thrombotic disorders. Based in Cambridge, MA, and Copenhagen, Denmark, Hemab is progressing a pipeline of innovative therapeutic solutions, leveraging a variety of cutting-edge technologies and approaches to transform the treatment paradigm for patients with high unmet need. The company’s strategic guidance, Hemab 1-2-5 TM , targets building a pipeline of multiple development programs to deliver long-awaited innovation for patients with high unmet need blood-clotting disorders like Glanzmann thrombasthenia, Factor VII Deficiency, Von Willebrand Disease, and others. Learn more at hemab.com . Follow us on LinkedIn , Facebook , Instagram , and X . SOURCE: Hemab Therapeutics