Prion Diseases

Younger patients in the U.S. can now benefit from Grifols Fibrin Sealant (FS) and its positive effect on surgical outcomes, including a fast time to hemostasisGrifols surgical bleeding management treatments form part of an increasingly robust portfolio of innovative therapeutics enhancing the health and well-being of patients BARCELONA, Spain, Oct. 29, 2024 (GLOBE NEWSWIRE) -- Grifols (MCE:GRF, MCE:GRF.P, NASDAQ:GRFS), a global healthcare company and leading manufacturer of plasma-derived medicines, today announced that its plasma-protein based fibrin sealant (FS) for controlling surgical bleeding has received approval from the United States Food and Drug Administration (FDA) for pediatric patients. The U.S. indication for children and adolescents extends the availability of FS, which is already approved for this patient segment in Europe, in addition to adults. During surgery Grifols FS promotes hemostasis and tissue sealing, resulting in reduced blood loss and potentially fewer complications. Grifols FS biosurgery treatment is commercialized as VISTASEAL™ in the U.S. and Canada, and VERASEAL™ in Europe and elsewhere. Both brands are marketed and distributed by Johnson & Johnson MedTech, as part of a strategic collaboration between the two companies announced in 2019. Grifols FS combines two plasma proteins, fibrinogen and thrombin, and is applied with Johnson & Johnson MedTech’s airless spray technology to rapidly form clots. The FS solution is now available in 18 countries. In early 2023, Grifols announced that it had met all primary and secondary endpoints of its phase 3b study evaluating the administration of Grifols FS to pediatric patients, defined as those not having reached 18 years of age. Researchers conducted a global prospective, randomized, active-controlled, single-blind, parallel group clinical trial designed to evaluate the safety and efficacy of the FS as an adjunct to hemostasis during surgery in pediatric patients (compared with an active control). The study included a total of 178 patients enrolled and treated across 18 recruitment centers. A greater than 95% efficacy rate was achieved in both treatment arms, with hemostasis within four minutes of application. In addition, Grifols FS demonstrated a good safety and tolerability profile, as the distribution of adverse events was comparable between arms. “Developing innovative biosurgery solutions reflects Grifols’ ability to provide patients with more medicines across many therapeutic areas to enhance their well-being,” said Joerg Schuettrumpf, Grifols Chief Scientific Innovation Officer. It’s estimated that between roughly one-third and two-thirds of open surgeries experience disruptive bleeding,1 while challenging and uncontrollable bleeding during surgery is associated with high mortality rates.1,2 About VISTASEAL VISTASEAL is a single-use product that uses a combination of human fibrinogen and human thrombin to assist with mild to moderate bleeding control when standard surgical techniques such as suture or cautery are ineffective. The sealant is applied in a thin layer over the bleeding tissue in order to generate a cross-linked fibrin clot to achieve hemostasis. VISTASEAL can be utilized in high-risk patients when there are concerns regarding coagulopathy, antiplatelets, anticoagulants, and friable tissue. VISTASEAL™ Fibrin Sealant (Human) IMPORTANT SAFETY INFORMATION INDICATIONVISTASEAL™, a fibrin sealant, is indicated as an adjunct to hemostasis for mild to moderate bleeding in patients undergoing surgery when control of bleeding by standard surgical techniques (such as suture, ligature, and cautery) is ineffective or impractical. VISTASEAL™ is effective in heparinized patients. CONTRAINDICATIONSDo not inject directly into the circulatory system. Do not use for the treatment of severe or brisk arterial bleeding.Do not use in patients with history of anaphylaxis or severe systemic reactions to human blood products.Do not use VISTASEAL™ for spraying unless the minimum recommended distance from the applicator tip to the bleeding site can be achieved. WARNINGS AND PRECAUTIONS Thromboembolic events may occur if VISTASEAL™ is administered intravascularly. Hypersensitivity reactions can occur. May carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. ADVERSE REACTIONSThe most common adverse reactions (reported in >1% of patients) were procedural pain, and nausea. For complete indications, contraindications, warnings, precautions, and adverse reactions, please reference full package insert.MEDIA CONTACTS: Grifols Press Office media@grifols.comTel. +34 93 571 00 02 SpainDuomo ComunicaciónTel.: +34 91 311 92 89 – +34 91 311 92 90Raquel Lumbreras (Tel. +34 659 572 185)Raquel_lumbreras@duomocomunicacion.comBorja Gómez (Tel. + 34 659 572 185)Borja_gomez@duomocomunicacion.com InvestorsInvestors Relations & Sustainabilityinversores@grifols.com - investors@grifols.comTel. +34 93 571 02 21 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. 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 23,000 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. 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 about Grifols, please visit grifols.com LEGAL DISCLAIMERThe 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, in so far 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 Corral M, Ferko N, Hollmann S, Broder MS, Chang E. Health and economic outcomes associated with uncontrolled surgical bleeding: a retrospective analysis of the Premier Perspectives Database. Clinicoecon Outcomes Res. 2015;7:409-421. doi:10.2147/CEOR.S863692 Marietta M, Facchini L, Pedrazzi P, Busani S, Torelli G. Pathophysiology of bleeding in surgery. Transplant Proc. 2006;38(3):812-814. doi:10.1016/j.transproceed.2006.01.047

A protein called alpha-synuclein that accumulates and ravages the brains of some people with Parkinson’s disease is the target of several experimental drugs. Until recently, the only way to detect it was by opening someone’s brain when they died. That’s starting to change. Amprion, a 30-person biotech based in San Diego, has developed a way to detect small amounts of alpha-synuclein from spinal fluid. A study published last year in The Lancet Neurology suggests the method could help confirm suspected diagnoses and even predict Parkinson’s before common symptoms arise. And now Amprion is scaling up to commercialize the test. CEO and co-founder Russell Lebovitz told Endpoints News in an exclusive interview that Amprion has raised $6 million in the first portion of its Series B, which will help it hire 15 more employees and expand its salesforce and operations throughout the US. The round was led by Formation Venture Engineering, with participation from Eli Lilly. Lilly’s interest is notable because the pharma company helped pioneer the use of diagnostics to detect amyloid in the brains of people with Alzheimer’s disease. The drugmaker is also developing drugs for Parkinson’s, and the company’s scientific leaders have mentioned alpha-synuclein as an area of interest for future neurodegenerative research at the company. “They have recognized, as have a lot of the drug companies, that this is going to be important in the future,” Lebovitz said. The technology underlying Amprion’s test was forged in the early 2000s in the aftermath of the mad cow disease crisis. Claudio Soto, a biochemist and professor of neurology at the UT Health Houston McGovern Medical School in Houston, developed a way to detect prions, the misfolded proteins that cause bovine spongiform encephalopathy in cattle and Creutzfeldt-Jakob disease in humans. The infectious proteins cause natural versions of the proteins to misfold, leaving tainted and toxic prions in their wake as they spread through the brain. That natural amplification formed the basis of Soto’s test. He added highly purified and properly folded proteins to spinal fluid or blood from a patient suspected of having the disease. If prions were present in the sample, they would quickly convert the normal proteins into diseased ones. He compared the method to PCR, the technique used to make many copies of DNA for easier sequencing. Soto formed a startup with Lebovitz in 2007 that aimed to refine the technique, now dubbed a seed amplification assay. The mad cow crisis abated, but the duo believed the technique would be valuable to help detect other misfolded proteins, such as amyloid and tau in Alzheimer’s and alpha-synuclein in Parkinson’s. The first ten years were slow going. The test originally took nearly two weeks to run, but they got it down to 20 hours. And a fluorescent dye that only lights up in the presence of misfolded proteins made the results easy to interpret. The tests are currently run out of its CLIA-certified lab in San Diego. The company is developing a simplified kit of the test that other labs can run, and hopes to submit it to the FDA in about 18 months. By 2019, Amprion had developed an alpha-synuclein test for Parkinson’s that it believed would be scalable. The company raised a $13.7 million Series A over the next three years and began offering the test to doctors at major medical centers and research institutions in 2023, Lebovitz said. The study published last year revealed that the test had a sensitivity of about 88%, meaning that about 12 in 100 people with Parkinson’s had a false negative result. Among the 1,100 healthy volunteers, the test had a 97% specificity, meaning about 3 in 100 people without the disease had a false positive. The test performed more poorly for certain subsets of Parkinson’s disease, including those with mutations in the LRRK2 gene linked to the disease. Drugmakers and regulators alike are increasingly turning to biomarkers to decide which patients to include in a clinical trial and how to assess whether the drug is working. In August, the FDA issued a letter of support encouraging “further study and use of” seed amplification assays for alpha-synuclein. Amprion has worked on similar amplification assays for amyloid and tau, but these tests are still experimental. The company offers clinical tests for amyloid, tau and another brain biomarker called neurofilament using machines sold by Fujirebio. With the new funding, Leboviz said “we can hit every major medical center doing cognitive and motor disorders in the US.” He’s hoping to raise another $9 million in the Series B round this year to help the company open a lab in Europe. But Amprion’s efforts to scale up come at a challenging time. About two weeks ago, an investigation published in Science cast doubt on several research papers supporting the role of alpha-synuclein in Parkinson’s disease. More than 130 papers published by National Institutes of Health neuroscientist Eliezer Masliah had “suspect images,” including doctored and duplicated results, according to the report. Some of the questionable studies were cited as evidence underpinning the alpha-synuclein-targeting antibody prasinezumab, which is being developed by Prothena Biosciences and Roche. Lebovitz said that other scientists have consistently demonstrated alpha-synuclein’s role in Parkinson’s and other neurodegenerative diseases irrespective of Masliah’s studies. “None of Amprion’s data relies on any work ever performed or published by Eliezer Masliah,” Lebovitz said. “We have never met with Dr. Masliah, and he has never provided any advice or other counsel.”

TEANECK, N.J., Sept. 9, 2024 /PRNewswire/ -- GC Biopharma USA, Inc. announced the launch and distribution of its immunoglobulin (IG) product ALYGLO (immune globulin intravenous, human-stwk), the company's first 10% intravenous immunoglobulin therapy for the treatment of adult patients aged 17 years and older with primary humoral immunodeficiency (PI). ALYGLO is a treatment option for patients with PI. This rare and chronic disorder occurs when the body's ability to produce antibodies is impaired, affecting the immune system's ability to fight infections. ALYGLO, an immune globulin formulation, has met the primary endpoints for both safety and efficacy as established by the FDA recommended guidelines in our Phase 3 clinical trials. Its novel manufacturing process may help improve patient safety by using Cation Exchange Chromatography (CEX) to remove coagulation factor XIa (FXIa) to undetectable levels. ALYGLO was approved by the U.S. Food and Drug Administration (FDA) on Dec. 15, 2023. Dr. Eun Chul Huh, President and CEO of GC Biopharma, said, "The US launch of ALYGLO by GC Biopharma USA is a historic milestone for the company and patients with primary immunodeficiency disease. It executes GC Biopharma's mission to improve the health and lives of individuals who suffer from rare and chronic diseases globally. GC Biopharma's commitment to the US further reflects its investment in R&D and a pipeline of future products to the US marketplace that will benefit patients and healthcare professionals." ALYGLO® is available to patients and healthcare professionals through a limited distribution network of top national Specialty Pharmacy providers. These partnerships provide a national footprint, delivering high-quality patient care for the primary immune deficiency population. ALYGLO is also available through specialty distributors throughout the United States. "ALYGLO expands patients' treatment options and improves access within the US marketplace," stated Dr. Lisa Betts, Chief Operating Officer for GC Biopharma USA, Inc. "GC Biopharma is committed to providing life-saving therapies to people with rare diseases. We are excited to offer the primary immune deficiency population another immune globulin therapeutic option." Please see Important Safety Information for ALYGLO on the following pages and refer to the full Prescribing Information (PI) or visit Alyglo.com For any medical or scientific questions regarding ALYGLO, call the GC Biopharma USA medical department at 1-833-426-6426. About ALYGLO ALYGLO is a liquid solution containing 10% immunoglobulin G (100 mg/mL) for intravenous infusion, manufactured from pooled human plasma from US donors. The manufacturing process includes multiple steps to reduce the risk of virus transmission. These include fractionation, solvent/detergent treatment, and nanofiltration. ALYGLO also uses its novel Cation Exchange Chromatography (CEX) in the manufacturing process for removing FXIa to undetectable levels. About Primary Humoral Immunodeficiency (PI) refers to a group of disorders where the body's ability to produce antibodies is impaired, affecting the immune system's ability to fight infections. In PI, there's a deficiency or absence of B cells or plasma cells, which are key in producing antibodies, particularly immunoglobulin G. This leads to increased susceptibility to infection, especially bacterial infections affecting the respiratory and gastrointestinal tracts. The PI indication for ALYGLO includes, but is not limited to, congenital agammaglobulinemia, common variable immunodeficiency (CVID), Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. About GC Biopharma Corp. GC Biopharma Corp. (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers lifesaving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma is a leading global plasma protein and vaccine product manufacturer dedicated to quality healthcare solutions for over half a century. About GC Biopharma USA, Inc. GC Biopharma USA, Inc., headquartered in Teaneck, NJ, established its sales, marketing, and business operations in 2018 to serve customers and patients throughout the US. Our foundation is built on the expertise of our parent company, GC Biopharma's, expertise—a leading biopharmaceutical company delivering plasma therapies and vaccines worldwide. With GC Biopharma USA, Inc., GC Biopharma further intends to extend its footprint, bringing its expertise and legacy to the United States. Forward-Looking Statements This press release may contain forward-looking statements that express the current beliefs and expectations of GC Biopharma USA management within the meaning of the Private Securities Litigation Reform Act of 1995. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. All statements contained in this press release other than statements of historical fact should be considered forward-looking statements and do not represent any guarantee by GC Biopharma USA or its management of future performance and involve known and unknown risks, uncertainties, and other factors. When used herein, words including "anticipate," "believe," "can," "continue," "could," "designed," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "possible," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon the Company's current expectations and various assumptions. The Company believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. The Company may not realize its expectations, and its beliefs may not prove correct. GC Biopharma USA undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. INDICATION ALYGLO™ is indicated for the treatment of primary humoral immunodeficiency (PI) in adults aged 17 years and older. This includes, but is not limited to, congenital agammaglobulinemia, common variable immunodeficiency (CVID), Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. IMPORTANT SAFETY INFORMATION Contraindications: ALYGLO is contraindicated in patients who have a history of anaphylactic or severe systemic reaction to the administration of human immune globulin and in IgA-deficient patients with antibodies to IgA and a history of hypersensitivity. Hypersensitivity: In case of hypersensitivity, discontinue infusion immediately and institute appropriate treatment. Epinephrine should be available for immediate treatment of severe acute hypersensitivity reactions. Hyperproteinemia, Increased Serum Viscosity, and Hyponatremia: Hyperproteinemia, increased serum viscosity, and hyponatremia may occur. Aseptic Meningitis Syndrome (AMS): Aseptic meningitis syndrome (AMS) may occur, especially with high doses or rapid infusion. AMS usually begins within several hours to 2 days following ALYGLO treatment. Discontinuation of treatment has resulted in remission of AMS within several days without sequelae. Hemolysis: Delayed hemolytic anemia due to enhanced red blood cell (RBC) sequestration and acute hemolysis consistent with intravascular hemolysis have been reported. Cases of severe hemolysis-related renal dysfunction/failure or disseminated intravascular coagulation have occurred following infusion of IGIV. Closely monitor patients for clinical signs and symptoms of hemolysis, particularly patients with risk factors. Transfusion-Related Acute Lung Injury: Noncardiogenic pulmonary edema (transfusion-related acute lung injury [TRALI]) may occur. TRALI is characterized by severe respiratory distress, pulmonary edema, hypoxemia, normal left ventricular function, and fever. Patients with TRALI may be managed using oxygen therapy with adequate ventilator support. Monitor patients for pulmonary adverse reactions. Transmissible Infectious Agents: Because ALYGLO is made from human blood, it may carry a risk of transmitting infectious agents (eg, viruses, the variant Creutzfeldt-Jakob disease [vCJD] agent and, theoretically, the Creutzfeldt-Jakob disease [CJD] agent). Interference with Laboratory Tests: After infusion of immunoglobulin, the transitory rise of the various passively transferred antibodies in the patient's blood may yield positive serological testing results, with the potential for a misleading interpretation. Adverse reactions (observed in ≥ 5% of study subjects) were headache, nausea/vomiting, fatigue, nasal/sinus congestion, rash, arthralgia, diarrhea, muscle pain/aches, infusion site pain/swelling, abdominal pain/discomfort, cough, and dizziness. It is recommended that ALYGLO be administered separately from other drugs or medications. ©2024 GC Biopharma USA, Inc. All rights reserved. ALYGLO is a trademark of Green Cross Corporation. SOURCE GC Biopharma USA Inc.