TNFRSF11B

LACHEN, Switzerland, Nov. 27, 2023 /PRNewswire/ -- New clinical data from Octapharma's haematology portfolio will be presented during the scientific program at the 65th American Society of Hematology (ASH) Meeting and Exposition, which is being held on December 9–12, 2023, in San Diego, California. New data from the prospective clinical trial using wilate® for prophylaxis in patients with von Willebrand disease (VWD) will be presented. Furthermore, the design of a new phase IV clinical trial of bone and joint health in people with haemophilia A will be presented, in addition to updates from research collaborations between Octapharma and academic institutions. "We are excited to present new data at ASH 2023 highlighting our ongoing commitment to understand the mechanisms underlying bleeding disorders and to improve the standards of care for patients. We are delighted to support clinical and scientific research that advances the care of people with bleeding disorders." – Olaf Walter, Board Member and Head of International Business Units at Octapharma. The WIL-31 study investigated the efficacy and safety of prophylaxis with wilate® in adults and children of any gender with all types of VWD. WIL-31 is the largest prospective prophylaxis study in VWD and the only one with a prospective on-demand run-in study as an intra-individual comparator. The results of the study showed that wilate® prophylaxis is highly effective at reducing the number of bleeds in children and adults and provides compelling evidence to support the use of regular prophylaxis in patients with severe VWD. Data from sub-analyses of the study will be presented at ASH 2023, focusing on the effectiveness of prophylaxis in patients with different types of bleeding and in different subsets of patients. Women with bleeding disorders such as VWD are at an increased risk for heavy menstrual bleeding, but they are underdiagnosed and undertreated. A sub-analysis of WIL-31 will be presented, demonstrating that wilate® prophylaxis was effective in reducing the incidence of heavy menstrual bleeding. An additional sub-analysis focused on the efficacy and safety of wilate® prophylaxis in children and adolescents will also be presented. People with haemophilia A often suffer from joint health issues and reduced bone strength. Prophylaxis with factor VIII replacement therapy has been associated with reductions in joint bleeding rates, joint deterioration, and degenerative joint disease. In contrast, there is a shortage of data on the long-term effects of prophylaxis with the non-factor therapy emicizumab. The new phase IV clinical trial PROVE aims to assess the long-term effects of prophylaxis with Nuwiq® or emicizumab on joint and bone health. The study design will be presented for the first time at ASH 2023. In addition to clinical studies, Octapharma is proud to support scientific investigations with academic research partners that further our understanding of different aspects of bleeding disorders. Results from various avenues of scientific investigation will be presented at ASH 2023. In PUPs with haemophilia A, the development of inhibitors to FVIII is the most significant treatment complication, and there is a need to better understand the mechanisms underlying inhibitor development. The NuProtect study with Nuwiq® was the largest prospective study of a single FVIII product in PUPs. Researchers from University College London, UK, will present new data from a sub-study of the NuProtect study, revealing differences in gene expression profiles between PUPs who developed inhibitors and those who did not. In collaboration with the ETH Zurich and Balgrist Campus Zurich, Switzerland, the effect of FVIII on bone remodelling has been evaluated in vivo, with the results indicating that FVIII and/or von Willebrand can further inhibit the formation of osteoclasts, cells that play a key role in bone remodelling. Platelets and endothelial cells play key roles in haemostasis. Research performed in collaborations with the ETH Zurich, Switzerland, and the University of Eastern Piedmont, Italy, revealed significant differences in the binding of different recombinant FVIII products to platelets and endothelial cells. "We are excited to present additional data from the WIL-31 study, the largest prospective study in VWD, that further supports the use of prophylaxis for children, adolescents and women with von Willebrand disease. We are also proud to introduce the upcoming PROVE study, which will help us understand how best to protect people with haemophilia A from debilitating joint damage." – Larisa Belyanskaya, Head of IBU Haematology. The posters will be presented at the following times: Saturday December 9, 17:30-19:30 (PST) Poster 1255: Design of a Comparative Study (PROVE) To Assess the Long-Term Effects of Prophylaxis With Simoctocog Alfa or Emicizumab on Joint and Bone Health in Hemophilia A Patients. Presenting author: Robert Klamroth Poster 1227: Transcriptomic profiling to understand inhibitor development in previously untreated patients with severe hemophilia A. Presenting author: Paul Batty Poster 1244: Impact of recombinant factor VIII and platelet interaction on platelet functionality and hemophilia A treatment. Presenting author: Fabrizio A. Pennacchio Monday December 11, 18:00-20:00 (PST) Poster 3977: Factor VIII is an endothelial factor that promotes vessel stability. Presenting author: Antonia Follenzi Poster 3979: Inter-donor variability in Osteoclastogenesis and responses to Osteoprotegerin (OPG), von Willebrand Factor (vWF), and Factor VIII (FVIII). Presenting author: Joyce Kimenai Poster 3992: Efficacy and Safety of Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Children and Adolescents with von Willebrand Disease – A Sub-Analysis of Data from the WIL-31 Study. Presenting author: Robert F. Sidonio, Jr Poster 3997: Efficacy of Regular Prophylaxis with a Plasma-derived von Willebrand Factor/Factor VIII Concentrate in Reducing Heavy Menstrual Bleeding in Females with von Willebrand Disease. Presenting author: Robert F. Sidonio, Jr 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 more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Haematology 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 190 plasma donation centres across Europe and the US. Octapharma has 40 years of experience in patient care. Octapharma press releases are specifically for health specialist/medical media and are not for consumer press. Logo -

▎药明康德内容团队报道12月20日，迈威生物宣布与Binnopharm Group就三款生物类似药在俄罗斯和欧亚经济联盟国家地区内的开发、生产和销售达成战略合作协议，分别为阿达木单抗生物类似药9MW0113，地舒单抗生物类似药9MW0321（预防肿瘤骨转移）和9MW0311（治疗骨质疏松）。其中，9MW0113（君迈康）是迈威生物与君实生物合作开发的一款重组人源抗TNF-α单克隆抗体注射液，为修美乐（阿达木单抗）的生物类似药。该产品已于2022年3月在中国获批上市，目前已获批用于类风湿关节炎、强直性脊柱炎、银屑病、克罗恩病、葡萄膜炎、多关节型幼年特发性关节炎、儿童斑块状银屑病和儿童克罗恩病等8项适应症。9MW0321是Xgeva（安加维）的生物类似药，是一款重组全人源抗RANKL单克隆抗体注射液。它可通过与RANKL结合，抑制OPG/RANKL/RANK信号传导通路的激活，从而达到抑制肿瘤生长和减少骨破坏的目的，拟用于预防实体瘤骨转移患者发生骨相关事件。9MW0311是Prolia（普罗力）的生物类似药，拟用于治疗绝经后妇女的骨质疏松症。这也是一款重组全人源抗RANKL单克隆抗体注射液，其作用机制在于：与RANKL结合后，阻止RANK活化，抑制破骨细胞的形成、活化和存活，减少骨吸收，从而消除全身性骨质疏松或局部骨质溶解等症状。迈威生物联合创始人、董事、首席执行官刘大涛博士表示：“很高兴迈威生物能与 Binnopharm Group达成在俄罗斯和欧亚经济联盟国家地区的战略合作，这是我们该地区的第一个战略合作伙伴，该区域也是生物类似药和创新药的重要市场。我们的三款生物类似药将在该区域商业化和本土化，这代表了我们高品质的产品和高可靠性的临床数据得到了认可。”Binnopharm Group董事、首席执行官Rustem Muratov先生表示：“与迈威生物的伙伴关系是我们在俄罗斯和欧亚经济联盟地区进一步发展生物技术业务战略的一个组成部分。本项目的目标不仅是在俄罗斯和欧亚经济联盟引进迈威生物的产品，还要与迈威生物等创新公司建立合作。此次合作对于Binnopharm Group以及我们在俄罗斯和欧亚经济联盟国家的患者来说也是一个重要的里程碑，他们将获得优质且可负担起的药物，提升生活品质。”参考资料：[1]海外拓展 │ 迈威生物与 Binnopharm Group 达成重要战略合作.Retrieved Dec 20, 2022. From https://mp.weixin.qq.com/s/-XeodNI0hmX1fAVQZcl5Qg本文由药明康德内容团队根据公开资料整理编辑，欢迎个人转发至朋友圈。转发授权请在「医药观澜」微信公众号留言联系我们。其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

骨重建是一个复杂的过程，需要在多个水平(时间、空间和数量)精确耦合成骨细胞和骨吸收破骨细胞活动。这种成骨细胞/破骨细胞偶联的破坏会导致骨质疏松。 骨重建是一个复杂的过程，需要在多个水平(时间、空间和数量)精确耦合成骨细胞和骨吸收破骨细胞活动。这种成骨细胞/破骨细胞偶联的破坏会导致骨质疏松。成骨细胞协调破骨细胞分化和骨吸收活性，破骨细胞主要通过产生破骨细胞分化因子、核因子-kB配体受体激活剂(RANKL)及其诱骗受体骨保护素(OPG)。 骨质疏松症的发生是由于骨重建的失调，这个过程需要骨形成成骨细胞和骨吸收破骨细胞。目前主要的骨质疏松治疗方法抑制破骨细胞介导的骨吸收，但由于成骨细胞介导的骨形成同时减少，所以治疗效果有限。 图片来源: https://doi-org.libproxy1.nus.edu.sg/10.1016/j.omtn.2022.07.008. 近日，来自马萨诸塞大学陈医学院的研究者们在Molecular Therapy: Nucleic Acids杂志上发表了题为“AAV-mediated delivery of osteoblast/osteoclast-regulating miRNAs for osteoporosis therapy”的文章，该研究揭示了骨靶向rAAV介导的miR-214-3p或miR-34a-5p的调节是一种很有前途的治疗骨质疏松症的新方法，同时限制了对非骨骼组织的不良反应。 在本研究中，研究者开发了一种针对骨质疏松症的基因治疗策略，通过靶向两个microRNAs(MiRNAs)-miR-214-3p和miR-34a-5p，同时促进骨形成和抑制骨吸收。研究者使用系统递送的针对骨骼的重组腺相关病毒(RAAV)载体来调节这些miRNAs的表达。RAAV介导的miR-214-3p在骨骼中的过表达或miR-34a-5p的抑制导致成年小鼠的骨丢失，类似于骨质疏松。 相反，rAAV介导的miR-214-3p的抑制或miR-34a-5p的过表达通过增加成骨细胞介导的骨形成和减少破骨细胞介导的骨吸收来逆转绝经后和老年性骨质疏松症小鼠模型中的骨丢失。值得注意的是，这些小鼠在非骨骼组织中没有表现出任何明显的病理表型。在机制上，抑制miR-214-3p上调成骨细胞中激活转录因子4和破骨细胞中的phatase和tensin同源物，而过表达miR34a-5p下调成骨细胞中的Notch1和TGFb诱导的破骨细胞中的同源盒2。 携带miR-214-3p TUD或miR-34a-5p的AAV9载体对健康小鼠的影响 图片来源: https://doi-org.libproxy1.nus.edu.sg/10.1016/j.omtn.2022.07.008. 综上所述，骨靶向rAAV9介导的基因治疗为探索低骨量下人类骨骼疾病的miRNA治疗提供了新的机会。此外，AAV基因治疗的潜力不仅限于骨质疏松症，特别是罕见的骨骼疾病，AAV运送有效载荷纠正基因突变的能力提供了直接解决这些疾病背后的遗传缺陷的唯一方法之一。（生物谷 Bioon.com） 参考文献 Aijaz Ahmad John et al. AAV-mediated delivery of osteoblast/osteoclast-regulating miRNAs for osteoporosis therapy. Mol Ther Nucleic Acids. 2022 Jul 11;29:296-311. doi: 10.1016/j.omtn.2022.07.008.