Albumin (Human) (Grifols)

Grifols plans to file for regulatory approval for the AdFIrst (BT524) in both Europe and the US in Q4 of this year. Image Credit: Kittyfly / Shutterstock. Spanish pharmaceutical company Grifols has reported positive Phase III trial data for its fibrinogen concentrate, AdFIrst (BT524) as a treatment for reducing blood loss in patients with acquired fibrinogen deficiency (AFD). The company plans to seek regulatory approval for the therapy in both Europe and the US in Q4 of this year. The active control Phase III trial (2017-001163-20) evaluated the safety and efficacy of AdFIrst compared to the standard of care (cryoprecipitate or fresh frozen plasma) in patients with AFD. The trial met its primary endpoint by demonstrating non-inferiority to the standard of care in reducing intraoperative blood loss during a major spinal or abdominal surgery. Mean blood loss volume was 1,444mL and 1,735mL in the treatment and standard of care groups, respectively. AdFIrst (adjusted fibrinogen replacement strategy) was developed as a fibrinogen alternative to standard fibrinogen therapies for AFD such as cryoprecipitate, a plasma extract, and fresh frozen plasma. Grifols added BT524 as part of its portfolio following the acquisition of Biotest in 2022 . See Also: FDA grants fast track status to Edgewise’s Duchenne treatment AFD can be caused by a variety of reasons including trauma, medication, malignancy, and underlying clotting disorders like disseminated intravascular coagulopathy (DIC). AFD results in a low fibrinogen level, which plays an important role in blood clotting. Grifols has experienced turmoil in recent months. Last month, Gotham City Research released a report accusing the company of artificially manipulating its debt and earnings through transactions with a company related to the Grifols family, who own controlling interest in Grifols. The allegation plunged the company’s stock by more than 40%. The stock has not recovered in value to what it was worth before the allegations were made. Following the news, Grifols has changed its management to “separate its management from ownership”. The latest development involved Raimon Grifols and Víctor Grifols Deu stepping down from their roles as chief corporate officer and chief operating officer, respectively. Other therapies Grifols is currently developing include Albutein (albumen therapy) and emphysema treatment. In July 2023, the company completed the enrolment of patients in its Phase III PRECIOSA trial (NCT03451292) of long-term Albutein (albumin-human injection) therapy to increase the survival time in patients with decompensated cirrhosis. Topline data from the study is expected in Q4 of this year.

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