Neurosyphilis

The contract will provide an initial commitment of $19.6 million and up to $135.2 million over a six-year period, supporting drug manufacturing and phase 1 trials for the two programs GigaGen’s recombinant polyclonals are part of Grifols’ robust innovation strategy and commitment to delivering the next generation of antibody drugs for patients and healthcare professionals SAN CARLOS, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- GigaGen Inc., a biotechnology company advancing transformative antibody drugs for immunodeficiencies, infectious diseases and checkpoint-resistant cancers, and a subsidiary of Grifols, announced today that it has been awarded a contract by the Biomedical Advanced Research and Development Authority (BARDA), part of the Administration for Strategic Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS). The award, valued at up to $135.2 million, is to develop a recombinant polyclonal antibody therapy for botulinum neurotoxins (BoNT) and a second biothreat of interest to the agency that will be determined at a later time. “We are thrilled to have been awarded this BARDA contract and are proud to continue our collaboration with the U.S. government to advance innovative therapies against naturally emerging and intentional biological threats,” said Carter Keller, senior vice president of Grifols and head of GigaGen. “GigaGen aims to change the way infectious diseases are treated with the world’s only recombinant polyclonal antibody therapeutic platform. Building on our successful collaboration with the Department of Defense (DOD), this project demonstrates the versatility of our recombinant polyclonal antibody platform, which is ideally suited for rapid responses to imminent biological threats.” The BARDA project builds upon GigaGen’s contract awarded by the U.S. DOD Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND) in 2022. The DOD project demonstrated the utility of GigaGen’s first-in-class recombinant human polyclonal antibody discovery platform against biological threats, focusing on two BoNT variants. Following success in this program that included in vivo neutralization of the two botulinum neurotoxins, the BARDA project will support the manufacturing and initial clinical development of a drug product that targets all seven BoNT variants. BoNT, one of the most toxic biological substances, is produced by the bacterium Clostridium botulinum and can cause progressive muscle paralysis from the head to the rest of the body, which can be fatal if left untreated. GigaGen recently received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to initiate a Phase 1 clinical trial to evaluate the company’s first recombinant polyclonal antibody therapeutic for the treatment of hepatitis B virus (HBV) infection, GIGA-2339. The company anticipates initiating the trial in Q4 2024. This project has been funded in whole or in part with federal funds from the Department of Health and Human Services; Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority, under contract number 75A50124C00049. About GigaGen’s platformGigaGen’s next-generation recombinant polyclonal platform offers a novel way to develop synthetic polyclonal antibody therapeutics in the laboratory, which are potentially more powerful than what a natural immune response can provide. Using high-throughput, single-cell genomic and protein engineering technology, GigaGen creates cell lines that express recombinant human antibodies against a diversity of infectious disease antigens. The polyclonal cell bank can then be used to continuously manufacture recombinant polyclonal products against the pathogen of interest at existing manufacturing facilities. GigaGen has demonstrated that its products are hundreds of times more potent than plasma-derived antibody therapies. They replicate and enhance the natural antibody diversity, encompassing thousands of antibodies. This offers a significant advantage over monoclonal antibody therapies, as they can address the vast diversity of circulating pathogen variants and help prevent immune escape upon pathogen mutation. About GrifolsGrifols 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. The company 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. 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). GigaGen is advancing transformative antibody drugs for immunodeficiencies, infectious diseases and checkpoint resistant cancers by leveraging industry-leading, single-cell technologies. Its novel technology platforms uniquely capture and recreate complete immune repertoires as functional antibody libraries. This approach has enabled the creation of first-in-class recombinant polyclonal antibody therapies for the treatment of infectious diseases. In addition, GigaGen’s lead oncology asset, GIGA-564, is an anti-CTLA-4 monoclonal antibody that has demonstrated improved anti-tumor efficacy and reduced toxicities in preclinical models through a unique mechanism of action. For more information, please visit www.grifols.com or www.gigagen.com. Media Contacts:Michael RubensteinLifeSci Communicationsmrubenstein@lifescicomms.comTel: +1- 561-289-7981 Grifols Press Officemedia@grifols.comTel. +34 93 571 00 02 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.

Results from the PARADIGM clinical trial have revealed that PrimeC, a treatment being developed by NeuroSense Therapeutics, was able to significantly slow down disease progression in ALS patients. The phase 2b clinical trial recruited 68 people living with ALS across Canada, Italy and Israel, who were followed for up to 18 months. ALS patients treated with PrimeC showed an improvement of 36% in the rate of decline  after 12 months, as compared to those who took a placebo. In addition, patients treated with PrimeC had a 43% higher survival rate during the same time period. "These exciting long-term results demonstrate how study participants experienced more slowing of progression over time with PrimeC as measured against ALSFRS-R, which is the current gold-standard scale used in ALS drug development," said Merit Cudkowicz, chair of neurology and Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. Underscoring the success of the trial, all participants requested to continue taking PrimeC after the study was completed. Based on these positive results, NeuroSense Therapeutics is planning to start a phase 3 clinical study of PrimeC in the US and Europe. ALS is a neurodegenerative disease that causes progressive paralysis and eventually death within a few years from receiving a diagnosis. There is currently no cure for the condition and only a handful of approved treatments that can help patients manage the disease. "The need for new treatments for people living with ALS has never been greater. PrimeC has great potential based on its mode of action and the phase 2 trial results and warrants further evaluation in a phase 3 trial in an expeditious manner," said Cudkowicz. googletag.cmd.push(function () { googletag.display('text-ad1'); }); PrimeC is NeuroSense Therapeutics’ lead drug candidate. The treatment consists of an oral formulation with extended release that contains two FDA-approved drugs: ciprofloxacin and celecoxib. The drug combination is designed to target multiple mechanisms of ALS, including the degeneration of neurons, inflammation, iron accumulation and impaired RNA regulation. "The promising results from the 12-month PARADIGM study highlight the significant potential of PrimeC as a disease-modifying drug for ALS,” said Vivian Drory, Head of the ALS clinic at Tel-Aviv Sourasky Medical Center. “These findings underscore the importance of early intervention, which can lead to more substantial benefits, and provide valuable insights that will inform the design of the company's phase 3 study, increasing the likelihood of success."

• The United States Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for EV68-228-N, a human monoclonal IgG1 against the capsid of enterovirus D68 (EV-D68) designed as an intravenous therapeutic for the treatment of acute flaccid myelitis (AFM) • The Phase 1 study is ongoing, and the first patient has been dosed at the Vanderbilt University Medical Center (VUMC) LONDON, UK I June 28, 2024 I KBio, one of the leading developers of new biological treatments derived from a plant-based production system, today announced that the United States Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for EV68-228-N, a human monoclonal IgG1 against the capsid of enterovirus D68 (EV-D68) designed as an intravenous therapeutic for the treatment of acute flaccid myelitis (AFM). EV68-228-N is being manufactured by KBio using its proprietary Rapid Protein Production Platform (RP3), which allows for the production of biologics using Nicotiana benthamiana . KBio’s Drug Master File (DMF) was reviewed by the FDA as part of this IND submission. The Phase 1 placebo-controlled double-blinded study is evaluating the safety and pharmacokinetics of single ascending doses of EV68-228-N following intravenous administration in healthy adult volunteers. The first patient has been dosed at the Vanderbilt University Medical Center (VUMC). Dr. Matthew Vogt discovered EV68-228 as a postdoctoral fellow in the Crowe laboratory at the Vanderbilt University Medical Center. In partnership with Dr. Vogt and Dr. C. Buddy Creech, the study’s principal investigator and director of the Vanderbilt Vaccine Research Program, KBio and ZabBio successfully transitioned the antibody from research and discovery into this first-in-human study by leveraging the RP3 platform and the combined decades of regulatory expertise in developing plant-produced biopharmaceuticals at KBio and ZabBio. “We’re pleased that the FDA has granted IND clearance for EV68-228-N in AFM, which is a rare and serious neurologic condition that can cause symptoms such as muscle weakness and progressive paralysis. AFM cases have been rising since 2014, and there are no approved disease-specific treatments,” said Patrick Doyle, chief executive officer at KBio. “Using our proprietary plant-based RP3 platform, we’re able to manufacture a monoclonal antibody that is directed against enterovirus, the most common cause of AFM. This novel platform was developed to produce high-quality biologics in an unprecedented 8-10 weeks, which allows for cost-effective and efficient production. We’re looking forward to continued enrollment in the study and providing a sense of renewed hope for these patients.” The trial will include 36 healthy volunteers ages 18 to 49. Six will receive a placebo (control group) and 30 (in groups of 10) will receive either a 3, 10 or 30 mg/kg dose of EV68-228-N intravenously. As part of the safety evaluation, scientists will monitor the first two participants in each group receiving the experimental treatment for at least 72 hours before others receive the infusion. Researchers will then monitor and evaluate participants during nine subsequent in-person examinations during the next 120 days. The trial is being sponsored by the National Institute of Allergy and Infectious Diseases (NIAID), which is part of the National Institutes of Health (NIH). It is being conducted in collaboration with multiple academic centers through the NIAID-funded Infectious Diseases Clinical Research Consortium (IDCRC), which encompasses the Institute’s long-standing Vaccine and Treatment Evaluation Units (VTEU). Monoclonal antibodies have expansive applications across diverse therapeutic areas. However, limitations exist in manufacturing these potentially life-saving medicines. Leveraging an eco-friendly production system that offers unprecedented speed and scalability, KBio develops monoclonal antibody therapeutics and vaccines with the potential to address a broad range of indications and public health challenges. About KBio KBio aims to create a new generation of biologics using its plant-based platform, Rapid Protein Production Platform (RP3), which has demonstrated the ability to create drug candidates at a fraction of the time and cost of conventional platforms. The Company is focused on discovering and developing monoclonal antibodies targeting validated pathways for immunological diseases and due to the speed and accuracy of its antibody production system, is positioned well to develop fast follower therapies against validated drug targets. KBio’s plant-based platform also has unique capabilities for addressing public health challenges where rapid development and large-scale production of vaccines and other bioactives are critical. Through a third-party partner, the Company is working with several health organizations that are funding its public health-specific pipeline. Formed in December 2021, KBio is a subsidiary of BAT with operations in the UK and U.S. For more information, please visit www.kbio.com . About Infectious Disease Clinical Research Consortium (IDCRC) The IDCRC , consisting of the Vaccine Treatment and Evaluation Units (VTEUs) and the IDCRC Leadership Group (UM1AI148684), was formed in 2019 to support the planning and implementation of infectious diseases clinical research that efficiently addresses the scientific priorities of NIAID. The consortium includes infectious diseases leaders and clinical researchers from Emory University, University of Maryland School of Medicine, Baylor College of Medicine, Cincinnati Children’s Medical Center and University of Cincinnati, FHI360, Fred Hutchinson Cancer Research Center, Johns Hopkins University, Kaiser Permanente Washington Health Research Institute, New York University, Saint Louis University, Vanderbilt University Medical Center, University of Alabama at Birmingham, University of Rochester, University of Washington, and NIAID. View other IDCRC studies . SOURCE: KBio