Simoctocog alfa

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

Octapharma 将在 2024 年 2 月 6 日至 9 日于德国法兰克福举行的欧洲血友病及相关疾病协会 (EAHAD) 第 17 届年会上公布临床和科学数据，以推进对出血性疾病的理解和治疗 Octapharma 将公布 wilate ® 和 Nuwiq ® 的最新临床和科研成果 顶尖专家将在一场卫星研讨会、一次口头报告和六个海报展示中介绍有关血管性血友病 (VWD) 和 A 型血友病的新发现 展示内容将包括 wilate ® 对血管性血友病 (VWD) 患者的预防疗效和安全性的新数据 瑞士拉亨 2024年1月19日 /美通社/ -- 在 EAHAD 2024 上展示 wilate ® WIL-31 是 VWD 领域最大规模的前瞻性预防研究，其中包含按需进行的磨合研究，用于患者个体内的比较。该研究表明，wilate ® 预防疗法在降低各种类型 VWD 儿童和成人患者的出血率方面疗效卓著，而且适用于所有已知的出血部位。研究结果为 VWD 患者采用常规预防疗法提供了有力证据。 "WIL-31 研究的新数据为在所有类型的 VWD 患者中使用 wilate ® 预防疗法提供了有力证据。这些研究结果促使美国批准 wilate ® 预防用途，为这些患者提供了更多的治疗选择。"– IBU Haematology 高级副总裁兼负责人 Larisa Belyanskaya。 Octapharma 的 卫星研讨会 "转变 VWD 预防范式：聚焦 WIL-31 研究"将深入探讨 WIL-31 研究的发现： Jan Astermark（瑞典）将探讨相较于 A 型血友病，VWD 预防性治疗不足的问题 Robert F. Sidonio Jr. （美国）将阐述 WIL-31 的研究结果何以对 VWD 预防性治疗的现状造成冲击 将以 WIL-31 研究中的三个互动病例介绍 wilate ® 预防疗法的疗效： 由来自亚特兰大的 Robert F. Sidonio Jr. 介绍儿童 VWD 病患 由来自萨格勒布的 Ana Boban 介绍经常流鼻血的成人 由来自德布勒森的 Csongor Kiss 介绍经期大量出血的女性 卫星研讨会将于 欧洲中部时间 2 月 7 日（星期三） 17:30-18:45 在 Panorama Room 2 举行。 Ana Boban 将于 欧洲中部时间 2 月 9 日（星期五） 08:30-10:00 在 SLAM 第 6 分会场 发表题为"使用血浆来源的血管性血友病因子/凝血因子 VIII 浓缩剂进行常规预防，可有效减少血管性血友病儿童和成人患者的鼻出血" (OR04) 的口头 SLAM 报告，阐述预防 VWD 患者频繁流鼻血的重要性以及 wilate ® 在这种情况下的预防效果。 两个海报展示提供对 WIL-31 研究结果的更多看法： PO213 ：在 WIL-31 研究中，在使用血浆来源的血管性血友病因子/凝血因子 VIII 浓缩剂进行预防治疗期间，凝血因子 VIII 和血管性血友病因子没有蓄积 PO194 ：在 WIL-31 研究中，在使用血浆来源的血管性血友病因子/凝血因子 VIII 浓缩剂进行预防治疗期间，使用抑制剂治疗血管性血友病 在 EAHAD 2024 上展示 Nuwiq ® 除了在凝血级联中的作用外，新的证据表明 FVIII 还会影响血小板和内皮细胞的功能。使用重组 FVIII (rFVIII) 浓缩剂进行替代治疗已成为治疗 A 型血友病患者的主要方法，但目前尚不清楚 rFVIII 浓缩剂的改性对血小板和内皮细胞功能的影响是否存在差异。与其他 rFVIII 浓缩剂相比，Nuwiq ® 在体外试验中与活化血小板的结合程度更高，而在另一项研究中，Nuwiq ® 比其他 rFVIII 浓缩剂对内皮细胞功能的影响更大： PO027 ：重组因子 VIII 浓缩剂与血小板的不同结合对血小板功能的影响 PO024 ：研究凝血因子 VIII 在内皮细胞功能中的作用 对于 A 型血友病等罕见疾病，无法在临床研究中直接比较治疗方法。在这种情况下，可以采用间接治疗比较方法来比较不同治疗方法的效果。其中匹配调整间接比较法 (MAIC) 是一种行之有效的间接比较方法。Nuwiq ® 以药代动力学为指导的个性化预防治疗与其他治疗方法的疗效已采用 MAIC 进行比较： PO041 ：在 A 型血友病患者中，对使用 simoctocog alfa 的个性化预防与使用 efanesoctocog alfa 的标准预防进行匹配调整的间接比较 PO042 ：在 A 型血友病患者中，对使用 simoctocog alfa 的个性化预防与使用 emicizumab 的标准预防进行匹配调整的间接比较 "Octapharma 的核心价值观是全力改善患者的生活，支持临床和科学研究项目是我们实现这一承诺的必要手段。我们很高兴能在 EAHAD 2024 上展示这些研究成果。"—Octapharma 董事会成员兼国际业务部负责人 Olaf Walter。 关于 Octapharma Octapharma 公司总部位于瑞士拉亨，是全球最大的人类蛋白质生产商之一，主要开发和生产源于人类血浆和人类细胞系的人类蛋白。 Octapharma 在全球拥有 11,000 多名员工，通过免疫疗法、血液学和重症监护三大治疗领域的产品，为 118 个国家\/地区的患者提供治疗支持。 Octapharma 在奥地利、法国、德国和瑞典拥有七个研发基地和五个先进生产设施，并在欧洲和美国设立 190 多个血浆捐赠中心。Octapharma 在患者护理方面积累了 40 年的丰富经验。 关于 Nuwiq ® Nuwiq ® (simoctocog alfa) 是第 4 代重组因子 VIII (rFVIII) 蛋白，源自人类细胞系，未经化学修饰或与任何其他蛋白融合 1。在培养过程中不含人源性或动物源性添加剂。不含非人类蛋白抗原表位，与血管性血友病因子具有高度亲和性 1。Nuwiq ® 治疗已在九项 1-3 已完成的临床试验中进行了评估，包括 201 名接受过治疗（190 人） 1 、108 名未接受过治疗 2 的重症 A 型血友病患者。Nuwiq ® 提供 250 IU、500 IU、1,000 IU、1,500 IU、2,000 IU、2,500 IU、3,000 IU 和 4,000 IU 八种规格 4。Nuwiq ® 已获准用于治疗和预防各年龄组 A 型血友病（先天性 FVIII 缺乏）患者的出血 4。 关于 wilate ® wilate ® 是一种高纯度人类血管性血友病因子\/凝血因子 VIII (VWF\/FVIII) 浓缩剂，在生产过程中采用双重病毒灭活步骤 5。未添加白蛋白作为稳定剂 5。经过纯化过程，VWF 与 FVIII 的比率达到与普通血浆近似的 1:1 5。wilate ® 含有一种 VWF 三重结构以及与普通人血浆类似的大分子量多聚体含量 5。wilate ®提供 500 IU 和 1000 IU 两种规格。 6wilate ® 在仅使用去氨加压素 (DDAVP) 无效或存在使用禁忌时，用于预防和治疗血管性血友病 (VWD) 患者的大出血或手术出血，同时还用于治疗和预防 A 型血友病（先天性 FVIII 缺乏）患者的出血 6。美国预防性治疗的适应症详见更新后的处方信息： wilate 完整处方信息 (wilateusa.com) Octapharma 新闻稿专供健康专家\/医疗媒体使用，不面向消费者媒体。 参考文献： 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. 联系方式： Ivana Spotakova +41 79 3474607 Ivana.Spotakova@octapharma. com