Immune Globulin Intravenous (Human) (Octapharma)

Octapharma to present new clinical and scientific findings for wilate® and Nuwiq® at the upcoming ISTH 2024 Congress New data and clinical study developments to be showcased in seven oral presentations, two posters and two Supported Symposia LACHEN, Switzerland, June 10, 2024 /PRNewswire/ -- The most recent developments from Octapharma's Haematology portfolio will be presented at the 32nd Congress of the International Society on Thrombosis and Haemostasis (ISTH) on June 22–26, 2024, in Bangkok, Thailand. The data will be featured in seven oral presentations, two poster presentations and two Supported Symposia during this key international meeting. Octapharma is a proud Silver Supporter of the ISTH 2024 Congress. Octapharma will also present the offer in modern solutions for acute bleeding and coagulation management in critical and intensive care. Bleeding disorders, such as haemophilia A and von Willebrand disease (VWD), carry the risk of recurrent and prolonged bleeds, which can not only be life-threatening but also take a heavy toll on patients' quality of life and mental health. The presentations at ISTH 2024 reflect Octapharma's long-term commitment to improving patient care and addressing unmet clinical needs. "Octapharma is excited to present our latest findings and study results from across our haematology and critical care portfolios at ISTH 2024." said Olaf Walter, Board Member at Octapharma. "Bleeding and coagulation disorder patients continue to face many challenges in their day-to-day care and bleeding management. Here at Octapharma we remain dedicated to frontline research towards improving patients' lives." Oral Presentations Although prophylaxis is the standard of care for severe haemophilia A, it remains underutilised in VWD. WIL-31, the largest study in VWD prophylaxis, demonstrated the safety and efficacy of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate (wilate®) in patients with VWD. The lead investigator, Dr Robert F. Sidonio Jr., will present sub-analyses of data from the study which explore differences in bleed sites across VWD types and age groups. Prophylaxis was well-tolerated across all age groups in the 22 VWD type 3 patients who were included in the analysis population. MOTIVATE is an ongoing, international, investigator-initiated study supported through funding from Octapharma AG, evaluating the management of haemophilia A patients with inhibitors. Dr Carmen Escuriola-Ettingshausen, one of the study's coordinating investigators, will present an interim analysis of the study. Haemophilic arthropathy is a serious complication of haemophilia A and early diagnosis is critical to minimise its long-term impact. A proof-of-concept study has identified a signature of several circulating small non-coding microRNAs, which may be used as biomarkers to assist in the diagnosis of haemophilic arthropathy. Future work will investigate microRNAs associated with earlier stages of the condition in a sub-study of the PROVE clinical trial. Altered microvascular endothelial functionality has been observed in haemophilia A patients. Indeed, impaired tubulogenesis, migration and permeability of endothelial cells from haemophilia A patients has been observed, compared to those from healthy patients. New data will be presented showing that Nuwiq® can bind to endothelial cell surfaces, improving their functionality. FVIII physically interacts with platelets following vascular injury to ensure efficient clot formation. Different FVIII modifications may impact platelet binding and downstream signalling. Data will be presented showing that Nuwiq® exhibits significantly higher binding to platelets compared to comparator products, and that these differences in platelet binding may impact the efficacy of different rFVIII products in the treatment of haemophilia A. The oral presentations are scheduled at the following times: Sunday June 23 OC 27.5: Impact of differential binding of recombinant factor VIII concentrates to platelets on platelet clotting functionality. Presenting author: Viola Vogel. 15:45–16:00 ICT, Room 210 A-D Monday June 24 OC 35.1: Factor VIII regulates extracellular matrix proteins to stimulate angiogenesis and vessel stability. Presenting author: Alessia Cucci. 9:30–9:45 ICT, Room 110 A-C OC 36.4: miRNA expression profiles as a potential biomarker of joint and bone health in haemophilia A. Presenting author: Yesim Dargaud. 10:15–10:30 ICT, Room 111 A-C OC 36.5: Factor VIII is a regulator of angiogenesis and a promoter of endothelial barrier stability. Presenting author: Antonia Follenzi. 10:30–10:45 ICT, Room 111 A-C Tuesday June 25 OC 63.4: Bleeding Sites in von Willebrand Disease Patients on Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F Sidonio Jr. 15:30–15:45 ICT, Ballroom B3 OC 61.4: Immune tolerance induction (ITI) strategies in haemophilia A patients with inhibitors – Interim analysis from the MOTIVATE study. Presenting author: Carmen Escuriola-Ettingshausen. 15:45–16:00 ICT, Ballroom B1 Wednesday June 26 OC 73.5: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Type 3 Patients with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F Sidonio Jr. 11:15–11:30 ICT, Ballroom B4 Poster Presentations Poster presentations are scheduled at the following times: Sunday June 23 PB 0251 - Indirect comparison of prophylaxis efficacy between simoctocog alfa and efanesoctocog alfa in severe hemophilia A and their cost in the United States. Presenting author: Craig M. Kessler. 13:45–14:45 ICT, Exhibition Hall Tuesday June 25 PB 1102 - Covalently linked complexes of coagulation factor VIII and von Willebrand factor fragment display increased stability and half-life in animal models and retain their function. Presenting author: Barbara Solecka-Witulska. 13:45–14:45 ICT, Exhibition Hall Supported Symposia Two Supported Symposia during the Congress will share new clinical and scientific data, providing updates on ongoing and new studies in the fields of haemophilia A and VWD. Learn About Tomorrow's Advances Today – Breakthrough in All-Round Bleed Protection Sunday June 23, 12:15–13:30 ICT, Room 208 Chair: Guy Young, Children's Hospital Los Angeles and Keck School of Medicine, Los Angeles, California, USA Octapharma's Symposium on haemophilia A this year is dedicated to cutting-edge research committed to bleed protection. Speakers will present the first results from the MOTIVATE study interim analysis and introduce the PROVE study investigating bone and joint health. This symposium will also explore the critical role of FVIII binding to platelets and endothelial cells in haemophilia A. Next Level Insights on Prophylaxis in von Willebrand Disease – Are You Addressing Your Patient's Needs? Monday June 24, 12:15–13:30 ICT, Room 210 Chair: Craig Kessler, Georgetown University Medical Center, Washington DC, USA This symposium will explore the critical role of prophylaxis in severe VWD. Topics presented will include the importance of prophylaxis and how to identify patients who may benefit from prophylactic treatment. Key lessons from the WIL-31 VWD prophylaxis study will be shared as well as the important role for prophylaxis in the management of heavy menstrual bleeding in women with VWD. Optimising care during pregnancy will also be discussed and the VIP and EMPOWER studies will be introduced. Both symposia will be open to congress participants in Bangkok and will be live streamed for participants not directly attending the Congress. An opportunity to ask questions will be available at both symposia. Larisa Belyanskaya, SVP and Head of IBU Haematology at Octapharma commented: "We are excited to present new insights from the WIL-31 data, and will take a deep dive into the importance of prophylaxis for specific patient groups, as well as discussing important clinical and scientific questions with the goal of improving the quality of life for people with von Willebrand disease and haemophilia A." About Nuwiq® Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein1. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor1. Nuwiq® treatment has been assessed in nine1 2 3 completed clinical trials which included 201 previously treated patients (190 individuals)1 and 108 previously untreated patients2 with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations4. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups4. About wilate® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production1. No albumin is added as a stabiliser5. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma1. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma1. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres6. wilate® is available in 500 IU and 1000 IU presentations. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)6. About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs nearly 12,000 employees worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Hematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 195 plasma donation centers across Europe and the US. "Octapharma press releases are specifically for health specialist/medical media and are not for consumer press." [1] Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. [2]Liesner RJ et al. Thromb Haemost 2021; 121:1400–8. [3] Octapharma AG, data on file. [4] Nuwiq® Summary of Product Characteristics [5] Stadler M et al. Biologicals 2006; 34:281-8. [6] wilate® Summary of Product Characteristics. Logo: SOURCE Octapharma AG

DUBLIN--( BUSINESS WIRE )--The "Sepsis - Pipeline Insight, 2024" clinical trials has been added to ResearchAndMarkets.com's offering. This report provides comprehensive insights about 30+ companies and 35+ pipeline drugs in Sepsis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. The report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Sepsis pipeline landscape is provided which includes the disease overview and Sepsis treatment guidelines. The assessment part of the report embraces, in depth Sepsis commercial assessment and clinical assessment of the pipeline products under development In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Sepsis collaborations, licensing, mergers and acquisition, funding, designations and other product related details. Report Highlights The companies and academics are working to assess challenges and seek opportunities that could influence Sepsis R&D. The therapies under development are focused on novel approaches to treat/improve Sepsis. Sepsis Emerging Drugs Chapters This segment of the Sepsis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases. Sepsis Emerging Drugs Thymosin alpha 1: SciClone Pharmaceuticals Thymosin alpha 1, referred to as thymalfasin, is a synthetic 28-amino acid peptide with multiple biological activities primarily directed towards immune response enhancement. SciClone developed and launched Thymosin alpha 1, under the trade name Zadaxin, for the treatment of hepatitis B virus (HBV) infection and hepatitis C virus (HCV) infections. The drug is also being developed for the treatment of COVID 2019 infections. Scientific and clinical studies have shown that Thymosin alpha 1 helps to stimulate and direct the body's immune response to eradicate infectious diseases, such as HBV, HCV, bacterial, and fungal infections; to fight certain cancers such as melanoma and liver cancer; and to enhance response to vaccines. Clinical trials have shown that Thymosin alpha 1 improves survival in patients in intensive care units being treated for sepsis from severe bacterial infections. The therapy is being evaluated in Phase III stage to treat Sepsis. Currently, the drug is in Phase III stage of its development for the treatment of sepsis. Enibarcimab: AdrenoMed AG Enibarcimab (HAM8101; former name: Adrecizumab) a clinical-stage, first-in-class drug targeting loss of vascular integrity. The strong rationale for Enibarcimab is supported by the elegance of its mode of action, a monoclonal antibody that on binding to its target Adrenomedullin preserves its functionality as regulator of vascular integrity. Enibarcimab targets Adrenomedullin (ADM), a vasoprotective peptide hormone. ADM that remains in the bloodstream, however, has a different effect, one that helps mitigate sepsis: promoting stability of the endothelial barrier by restoring the cell junctions between endothelial cells that ordinarily regulate molecule transport and leakage. In health, levels of ADM in the bloodstream and extravascular space are in equilibrium so that endothelial barrier integrity is maintained and blood pressure remains normal. Currently, the drug is in Phase II stage of its development for the treatment of sepsis. M 6229: Matisse Pharmaceuticals Matisse's platform technology is based on the discovery that in many patients suffering from sepsis, proteins called histones are released by the innate immune system and dying cells into the blood stream, where they are toxic to other cells. Due to a self-enforcing cascade, people may die from organ failure within one or two days. Preclinical results have shown that by neutralizing the toxic histones with Matisse's product M6229, the negative cascade is terminated by neutralization of cationic histones by anionic M6229. Matisse claims to have identified an elegant solution for treating one of the major complications in sepsis by using a non-anticoagulant fraction of heparin called M6229 to neutralize toxic circulatory histones .Currently, the drug is in Phase I stage of its development for the treatment of sepsis. SNIPR 001: SNIPR Biome SNIPR001 is a novel, orally-administered antibiotic that is designed to precisely target difficult-to-treat bacterial infections. It is developed using SNIPR Biome's CRISPR-Guided VectorsT (CGVT Technology), which is designed to deliver CRISPR reagents into target bacterial cells. SNIPR001 is designed to target certain E. coli bacteria in the gut and thus prevent their translocation to the bloodstream, without affecting beneficial bacteria in the microbiome. It contains four CRISPR-armed phages that selectively target and eliminate E. coli strains that are resistant to fluoroquinolone, with demonstrated efficacy in animal disease models. Currently, the drug is in preclinical stage of its development for the treatment of sepsis. Sepsis: Pipeline Development Activities The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Sepsis therapeutic drugs key players involved in developing key drugs. Pipeline Development Activities The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Sepsis drugs. Sepsis: Therapeutic Assessment Phases Late stage products (Phase III) Mid-stage products (Phase II) Early-stage product (Phase I) along with the details of Pre-clinical and Discovery stage candidates Discontinued & Inactive candidates Route of Administration Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Parenteral Topical Molecule Type Products have been categorized under various Molecule types such as Recombinant fusion proteins Small molecule Monoclonal antibody Peptide Polymer Gene therapy Product Type Sepsis Report Insights Sepsis Pipeline Analysis Therapeutic Assessment Unmet Needs Impact of Drugs Sepsis Report Assessment Pipeline Product Profiles Therapeutic Assessment Pipeline Assessment Inactive drugs assessment Unmet Needs Key Players SciClone Pharmaceuticals AdrenoMed AG Matisse Pharmaceuticals SNIPR Biome Tianjin Chase Sun Pharmaceutical Beijing Scitech-Mq Pharmaceuticals Recce Pharmaceuticals Inotrem Octapharma Shaperon Shionogi Pharmazz Seres Therapeutics Key Products Thymosin alpha 1 Enibarcimab M 6229 SNIPR 001 Kukoamine B ST-1830 RECCE 327 Nangibotide OctaplasLG HY209 Cefiderocol Centhaquine SER 155 For more information about this clinical trials report visit https://www.researchandmarkets.com/r/ixi7k5 About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. 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