Albumin (Human) (Grifols)

PRECIOSA clinical trial is designed to evaluate efficacy and safety of Grifols Albutein® plus standard medical treatment to increase survival time in patients with decompensated cirrhosis awaiting transplant Albumin’s antioxidant and anti-inflammatory properties have the potential to mitigate the complications associated with decompensated cirrhosis Grifols continues innovating across its plasma-protein franchise and is committed to applying its ever-growing expertise in a range of therapeutic areas to help treat pressing societal healthcare challenges BARCELONA, Spain, July 18, 2023 (GLOBE NEWSWIRE) -- Grifols (MCE: GRF, MCE: GRF.P NASDAQ: GRFS), one of the world’s leading producers of plasma-derived medicines, today announced it has completed enrollment in PRECIOSA (NCT03451292), its phase 3 clinical trial designed to determine the potential of long-term albumin treatment with Grifols Albutein® to increase survival time in patients with decompensated cirrhosis and ascites until a suitable transplant is available. Cirrhosis, a condition in which the liver is permanently scarred and can lead to liver failure, is the leading cause of liver-related deaths globally,1 with more than 1.32 million deaths reported in 2017.2 In the U.S. alone, researchers estimate that about 1 in 400 adults have cirrhosis,3 including those who have progressed into decompensated cirrhosis once complications such as ascites appear. Ascites is a buildup of fluid in the abdomen and signals that a patient’s risk for poor outcomes, including death, have significantly increased. Albumin, the most abundant protein in plasma, has antioxidant and anti-inflammatory properties with the potential to mitigate the complications associated with decompensated cirrhosis and its progression into the next stage of the disease, acute-on-chronic liver failure (ACLF). Treatment with albumin has the potential to reduce the high one-year mortality rates observed in decompensated cirrhosis. Over 400 patients with decompensated cirrhosis with ascites are participating in this multi-center, randomized (1:1), controlled, parallel-group, open-label study in 69 sites across North America and Europe. It will evaluate the efficacy and safety of long-term Albutein® administration (dosed every 10 ± 2 days for up to 12 months) plus standard medical treatment. “There is great potential for albumin to improve the survival prospects of patients suffering from decompensated cirrhosis until they can get a liver transplant, a large unmet need given the limited availability of livers for patients,” said Sandra Camprubi, Grifols Senior Director Clinical Operations. “We look forward to providing topline data from this study in the fourth quarter of 2024 and evaluating next regulatory steps to provide patients with a much-needed treatment.” Grifols is committed to strengthening its innovation pipeline, which consists of multiple plasma and non-plasma programs across various clinical stages, all dedicated to potential treatments to help patients live longer, better-quality lives. About Decompensated Cirrhosis and Ascites Chronic liver disease is an all too common and growing problem in the developed world, with worldwide prevalence rates of around 20%.4 One such condition is cirrhosis, in which the liver is permanently scarred and, in many cases, can lead to liver failure. Decompensated cirrhosis is defined by the complications that can occur in a patient with cirrhosis, which include ascites, variceal bleeding, hepatic encephalopathy, and bacterial infections. These complications are associated with worse survival (2-4 years) compared with compensated cirrhosis (10-15 years).5 About Grifols Grifols is a global healthcare company founded in Barcelona in 1909 committed to improving the health and well-being of people around the world. A leader in essential plasma-derived medicines and transfusion medicine, the company develops, produces and provides innovative healthcare services and solutions in more than 110 countries. Patient needs and Grifols’ ever-growing knowledge of many chronic, rare and prevalent conditions, at times life-threatening, drive the company’s innovation in both plasma and other biopharmaceuticals to enhance quality of life. Grifols is focused on treating conditions across a broad range of therapeutic areas: immunology, hepatology and intensive care, pulmonology, hematology, neurology and infectious diseases. A pioneer in the plasma industry, Grifols continues to grow its network of donation centers, the world’s largest with over 390 across North America, Europe, Africa and the Middle East and China. As a recognized leader in transfusion medicine, Grifols offers a comprehensive portfolio of solutions designed to enhance safety from donation to transfusion, in addition to clinical diagnostic technologies. It provides high-quality biological supplies for life-science research, clinical trials, and for manufacturing pharmaceutical and diagnostic products. The company also supplies tools, information and services that enable hospitals, pharmacies and healthcare professionals to efficiently deliver expert medical care. Grifols, with more than 24,000 employees in more than 30 countries and regions, is committed to a sustainable business model that sets the standard for continuous innovation, quality, safety and ethical leadership. In 2022, Grifols’ economic impact in its core countries of operation was EUR 9.6 billion. The company also generated 193,000 jobs, including indirect and induced. The company’s class A shares are listed on the Spanish Stock Exchange, where they are part of the Ibex-35 (MCE:GRF). Grifols non-voting class B shares are listed on the Mercado Continuo (MCE:GRF.P) and on the U.S. NASDAQ through ADRs (NASDAQ:GRFS). For more information, please visit . MEDIA CONTACT: Media Press Office media@grifols.com Tel. +34 93 571 00 02 INVESTORS: Investors Relations Department & Sustainability inversores@grifols.com - investors@grifols.com Tel. +34 93 571 02 21 LEGAL DISCLAIMER The facts and figures contained in this report that do not refer to historical data are “future projections and assumptions”. Words and expressions such as “believe”, “hope”, “anticipate”, “predict”, “expect”, “intend”, “should”, “will seek to achieve”, “it is estimated”, “future” and similar expressions, insofar as they relate to the Grifols group, are used to identify future projections and assumptions. These expressions reflect the assumptions, hypotheses, expectations and predictions of the management team at the time of writing this report, and these are subject to a number of factors that mean that the actual results may be materially different. The future results of the Grifols group could be affected by events relating to its own activities, such as a shortage of supplies of raw materials for the manufacture of its products, the appearance of competitor products on the market, or changes to the regulatory framework of the markets in which it operates, among others. At the date of compiling this report, the Grifols group has adopted the necessary measures to mitigate the potential impact of these events. Grifols, S.A. does not accept any obligation to publicly report, revise or update future projections or assumptions to adapt them to events or circumstances subsequent to the date of writing this report, except where expressly required by the applicable legislation. This document does not constitute an offer or invitation to buy or subscribe shares in accordance with the provisions of the following Spanish legislation: Royal Legislative Decree 4/2015, of 23 October, approving recast text of Securities Market Law; Royal Decree Law 5/2005, of 11 March and/or Royal Decree 1310/2005, of 4 November, and any regulations developing this legislation. In addition, this document does not constitute an offer of purchase, sale or exchange, or a request for an offer of purchase, sale or exchange of securities, or a request for any vote or approval in any other jurisdiction. The information included in this document has not been verified nor reviewed by the external auditors of the Grifols group. 1 GBD 2017 Causes of Death Collaborators. Global, regional, and national age-sex-specific mortality for 282 causes of death in 195 countries and territories, 1980-2017: a systematic analysis for the Global Burden of Disease Study 2017. Lancet. 2018;392(10159):1736-1788. doi:10.1016/S0140-6736(18)32203-7 2 GBD 2017 Cirrhosis Collaborators. The global, regional, and national burden of cirrhosis by cause in 195 countries and territories, 1990-2017: a systematic analysis for the Global Burden of Disease Study 2017. Lancet Gastroenterol Hepatol. 2020;5(3):245-266. doi:10.1016/S2468-1253(19)30349-8 3 Scaglione S, Kliethermes S, Cao G, et al. The Epidemiology of Cirrhosis in the United States: A Population-based Study. J Clin Gastroenterol. 2015;49(8):690-696. doi:10.1097/MCG.0000000000000208 4 Moon AM, Singal AG, Tapper EB. Contemporary epidemiology of chronic liver disease and cirrhosis. Clin Gastroenterol Hepatol. 2020;18(12):2650-2666. doi: 10.1016/j.cgh.2019.07.060 5 López-Sánchez GN, Dóminguez-Pérez M, Uribe M, Nuño-Lámbarri N. The fibrogenic process and the unleashing of acute-on-chronic liver failure. Clin Mol Hepatol. 2020;26(1):7-15. doi:10.3350/cmh.2019.0011; Fanali G, di Masi A, Trezza V, Marino M, Fasano M, Ascenzi P. Human serum albumin: from bench to bedside. Mol Aspects Med. 2012;33(3):209-290. doi:10.1016/j.mam.2011.12.002

2022年8月26日至29日，第七十届欧洲心脏病学会年会（ESC Congress 2022）在西班牙巴塞罗那隆重召开。会上，欧洲心脏病学会（ESC）联合欧洲呼吸学会（ERS）一起重磅发布了《2022 ESC/ERS 肺高血压诊断和治疗指南》（以下简称《指南》），为全球肺高血压的诊疗提供了最佳的方案推荐以及最新的证据支持，供医生专家和病人家庭借鉴与参考。值得关注的是，新版《指南》在针对肺动脉高压（PAH）的治疗策略介绍中，特别新增了关于肺动脉去神经术（PADN技术）的介绍和分析，肯定了PADN技术在肺动脉高压治疗领域业已呈现出的重要学术价值和临床潜力，也反映了该技术为肺动脉高压患者的疾病治愈和健康管理所带来的积极意义。除此之外， PADN技术在国际肺高血压治疗领域还在持续受到更多的关注，包括即将在今年9月召开的2022年美国经导管心血管治疗学研讨会（TCT 2022），帕母医疗将对全球首次公布PADN-CFDA注册临床研究结果，让我们拭目以待！《指南》在针对肺动脉高压（PAH）的介入治疗章节中指出：正常人体的肺动脉分叉处分布有【压力】/【牵张】感受器，这些感受器通过负反馈机制调节肺动脉压力而产生血管收缩和血管重构，而促进PAH发生的交感神经兴奋正与肺动脉血管收缩和血管重构存在关联。动物实验证明局部损伤或干预肺动脉交感神经的完整性和功能后，哺乳动物的肺动脉压力可得到显著变化。人体试验方面，已有小型多中心临床试验结果表明肺动脉高压患者接受PADN治疗的安全性，并且该治疗使患者的活动耐量显著提高，具体表现为肺血管阻力降低和6分钟步行距离增加。PADN治疗肺动脉高压极具潜力！PADN治疗原理探索1980年，发表在CHEST杂志的一项临床研究，探讨了肺动脉球囊扩张诱导的肺动脉高压原理，结果表明肺动脉球囊扩张将刺激肺动脉分叉处或附近的【压力】/【牵张】感受器和/或交感神经神经纤维，并通过肾上腺素能神经介导，造成肺动脉压力升高。随后，2004年发表于Circulation的一项研究进一步明确了肺动脉高压患者交感神经活动增加的假设，首次报告了晚期肺动脉高压患者中交感神经活性增加的直接证据，并提出肺动脉高压患者中的交感神经过度激活可能与疾病严重程度相关。2010年发表于AJRCCM杂志的一项研究探讨了肺动脉高压患者肌肉中交感神经系统活动（MSNA）对患者预后中的意义，并提示交感神经系统激活是肺动脉高压临床恶化的独立预测因子。在上述临床研究的基础上，可以进一步明确人类肺动脉血管主要受交感神经调控，而交感神经密度从肺动脉近端到远端逐渐降低，肺动脉的牵张刺激会激活肺动脉交感神经，增加肺动脉压力和肺循环阻力。基于此机制，陈绍良院长于2012年正式提出PADN技术，并在20只实验犬身上开展了首次PADN动物实验。研究结果显示，经过PADN处理后，平均肺动脉压(mPAP)、收缩期PAP和舒张期PAP均恢复到基线水平。无不良后果或并发症，如急性血栓形成、PA穿孔或死亡。这个最初的动物实验表明，反射的传入神经纤维和传出神经纤维位于主PA的分叉处。虽然该研究缺乏组织学成分来评估PADN治疗消融神经纤维的可行性，但该动物实验表明了PADN治疗PAH的有效性和安全性。  临床研究陆续展开，PADN临床应用安全有效在动物实验获得预期结果后，陈绍良院长团队陆续开展了PADN1-Phase1、PADN1-Phase2、PADN5等一系列PADN临床研究，探索PADN在WHO Group1、Group2和Group4 肺高血压（PH）患者中的安全性与有效性。PADN-1 Phase1研究探讨了PADN术在药物治疗无反应的特发性肺动脉高压（IPAH）患者中的的安全性和有效性。该研究共纳入21例PAH患者，其中13例患者行PADN手术，为手术组；8例患者未进行手术干预，为对照组。三个月随访结果显示，手术组患者平均肺动脉压（PAP）由55±5 mmHg下降到36±5 mmHg（P＜0.01）；六分钟步行距离（6WMT）从324±21 m增加到491±38 m（P＜0.006）；三尖瓣偏移指数（Tei）由 0.7±0.04下降至0.50±0.04（P＜0.001）。该研究首次报道了PADN对药物治疗无反应的IPAH患者心脏功能和血流动力学状态的积极作用。PADN-1 Phase2研究旨在分析PADN对于不同病因的PH患者血流动力学、心脏功能和临床症状的影响。该研究是一项前瞻性研究，共纳入2012年4月至2014年4月期间，连续66例静息mPAP≥25 mmHg且接受PADN的患者。该研究结果提示，PADN治疗PH安全可行，且PADN手术可显著改善患者血流动力学状态、心脏功能、运动能力，且PADN治疗后一年的PH相关事件和死亡率更低。未来需要开展进一步临床研究，明确PADN在PH患者中的疗效。PADN-5研究以术后6个月6分钟步行距离改善作为主要有效终点，是全球首个证实肺动脉消融对心衰患者疗效的临床随机对照研究。共纳入通过右心导管检测确诊的CpcPH患者98 例，其中假手术组患者50例，在抗心衰药物及西地那非治疗基础上接受PADN假手术，PADN治疗组患者共48例，接受PADN联合抗心衰药物治疗。研究结果显示，两组间除右房内径的改变无显著统计学差异外，PADN手术组其他指标均较西地那非药物组改善明显，证实了CpcPH患者可从PADN治疗中获益，且该获益在射血分数下降的患者及射血分数保留的患者中均存在。PADN治疗的PAH患者的长期随访研究纳入了从2012年3月至2018年10月期间接受PADN治疗的120名肺动脉高压患者。根据病因，将患者分为68名IPAH与52名结缔组织疾病相关肺动脉高压（CTD-PAH）。平均随访6.6年，共23名患者死亡，其中IPAH患者15例，CTD-PAH患者8 例，死亡风险比是0.675。PADN术后6MWD净增加中位数为43米。接受PADN患者血流动力学指标有着显著改善。REVEAL评分≥7和基线WHO功能等级是长期死亡率的独立因素。长期随访数据表明PADN显著提高了IPAH和CTD-PAH患者的长期生存率。   多中心RCT临床研究陆续开展，填补肺动脉高压诊疗领域空白基于多项临床研究结果，PADN在肺动脉高压患者治疗中的安全性与有效性得到了初步证实，进一步开展多中心RCT临床研究势在必行。如今，由陈绍良教授团队领衔开展的PADN-CFDA研究已全部完成， 临床研究结果即将于TCT 2022期间在主会场进行公布，该研究作为一项单盲、随机、假手术对照临床研究，旨在评估PADN手术治疗PAH的安全性和有效性。目前披露的初步临床研究数据表现非常积极，PADN为肺动脉高压临床治疗提供了全新有效的治疗方法，相信PADN-CFDA最终研究结果的发布将为广大术者带来更多惊喜，填补PADN技术在RCT研究领域的空白。此外，由全球心血管领域泰斗Gregg W. Stone教授担任PI的PADN国际多中心临床试验也即将启动，以期为PADN技术提供更多循证医学支持，同时为肺动脉高压治疗提供新的解决方案。帕母医疗始终致力于为患者提供以临床价值为驱动的突破性疗法，专注硬核创新，为本土自主创新医疗器械代言。此次，PADN作为帕母医疗原创探索成果，收录于ESC指南，既是对帕母医疗企业使命的认可，也再一次阐明帕母医疗在行业中的贡献与价值！2022年9月1日19:00-20:40CDQI肺血管病中心指南巡讲：ESC/ERS肺动脉高压诊治指南第一期想要联系动脉网报道的企业，请点击文末左下方“阅读原文”填写表单，我们的工作人员将征求企业意见后，尽快为您服务。近期推荐声明：动脉网所刊载内容之知识产权为动脉网及相关权利人专属所有或持有。未经许可，禁止进行转载、摘编、复制及建立镜像等任何使用。动脉网，未来医疗服务平台