Gaucher Disease

CARMIEL, Israel, Oct. 25, 2024 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE American:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx® plant cell–based protein expression system, today announced that data from the phase I clinical trial of PRX-115, the Company's recombinant PEGylated uricase product candidate in development for the treatment of uncontrolled gout, will be presented in a late-breaking poster at the American College of Rheumatology (ACR) Convergence 2024, being held November 14-19, 2024 at the Walter E. Washington Convention Center in Washington, D.C. Details of the presentation are as follows: ACR Convergence 2024 Title: Prolonged Plasma Urate-Lowering After a Single Intravenous Administration of PRX-115, a Novel PEGylated Uricase, in Participants with Elevated Urate Levels Session: Late-Breaking Posters (L01-L14) Session Date/Time: Monday, November 18, 2024, 10:30 AM – 12:30 PM Eastern Standard Time Presenting Author: Orit Cohen Barak, Ph.D. (Protalix Ltd.) Abstract Number: L05 The accepted abstract can be accessed on the ACR Convergence 2024 website at . A copy of the poster will be made available on the Protalix website. About Protalix BioTherapeutics, Inc. Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx. It is the first company to gain U.S. Food and Drug Administration (FDA) approval of a protein produced through plant cell-based in suspension expression system. This unique expression system represents a new method for developing recombinant proteins in an industrial-scale manner. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights to taliglucerase alfa for the treatment of Gaucher disease, Protalix's first product manufactured through ProCellEx, excluding in Brazil, where Protalix retains full rights. Protalix's second product, Elfabrio®, was approved by both the FDA and the European Medicines Agency in May 2023. Protalix has partnered with Chiesi Farmaceutici S.p.A. for the global development and commercialization of Elfabrio. Protalix's development pipeline consists of proprietary versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: PRX–115, a plant cell-expressed recombinant PEGylated uricase for the treatment of uncontrolled gout; PRX–119, a plant cell-expressed long action DNase I for the treatment of NETs–related diseases; and others. Forward-Looking Statements To the extent that statements in this press release are not strictly historical, all such statements are forward-looking, and are made pursuant to the safe-harbor provisions of the Private Securities Litigation Reform Act of 1995. The terms "expect," "anticipate," "believe," "estimate," "project," "may," "plan," "will," "would," "should" and "intend," and other words or phrases of similar import are intended to identify forward-looking statements. These forward-looking statements are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes. Drug discovery and development involve a high degree of risk and the final results of a clinical trial may be different than the preliminary findings for the clinical trial. Factors that might cause material differences include, among others: failure or delay in the commencement or completion of clinical trials which may be caused by several factors, including: slower than expected rates of patient recruitment; unforeseen safety issues; determination of dosing issues; lack of effectiveness during clinical trials; inability to satisfactorily demonstrate non-inferiority to approved therapies; inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; inability to monitor patients adequately during or after treatment; and/or lack of sufficient funding to finance our clinical trials; the risk that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; our dependence on performance by third-party providers of services and supplies, including without limitation, clinical trial services; the inherent risks and uncertainties in developing drug platforms and products of the type we are developing; the impact of development of competing therapies and/or technologies by other companies; potential product liability risks, and risks of securing adequate levels of related insurance coverage; the possible disruption of our operations due to terrorist activities and armed conflict, including as a result of the disruption of the operations of certain regulatory authorities and of certain of our suppliers, collaborative partners, licensees, clinical trial sites, distributors and customers; and other factors described in our filings with the U.S. Securities and Exchange Commission. The statements in this press release are valid only as of the date hereof and we disclaim any obligation to update this information, except as may be required by law. Investor Contact Mike Moyer, Managing Director LifeSci Advisors +1-617-308-4306 [email protected] Logo - SOURCE Protalix BioTherapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Data highlight potential of FLT201 to set new standard of care for Gaucher disease Dramatic and durable reductions observed in lyso-Gb1, an established biomarker of clinical response, in patients with persistently high levels despite years of prior treatment on currently approved therapies Substantial improvements in bone marrow burden demonstrate FLT201 penetrating deeper tissues that currently approved therapies poorly address Favorable safety and tolerability continue to be seen in all patients dosed On track to initiate Phase 3 trial of FLT201 in 2025 Oct. 23, 2024 -- Spur Therapeutics today announced positive new data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, an adeno-associated virus (AAV) gene therapy candidate for Gaucher disease type 1, showing durable reductions in glucosylsphingosine (lyso-Gb1), one of the best predictors of clinical response in Gaucher disease, and bone marrow burden, as well as improvements or maintenance of blood counts and organ volume in patients treated with a single infusion of FLT201. FLT201 continues to demonstrate a favorable safety and tolerability profile in all patients treated in the study. These data are being showcased in an oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress taking place in Rome, Italy. “Gaucher disease is a debilitating chronic disorder, and despite treatment with currently approved therapies, many patients continue to have serious symptoms,” said Pamela Foulds, M.D., Spur’s Chief Medical Officer. “Consistent with what is seen in real-world practice, patients entered our trial with different unmet needs. All of them had bone disease, which current therapies poorly address. Most had high levels of harmful substrate accumulation. Some also had an enlarged spleen, low platelets, pain or fatigue. What is exciting is that after a single infusion of FLT201, patients improved in areas their disease was poorly controlled and remained well-controlled in areas their disease was controlled. These data underscore FLT201’s potential to set a new standard of care for Gaucher disease type 1.” Today’s oral presentation at ESGCT during the session titled “AAV Vectors as Tools in Gene Therapy of Rare Diseases - Recent Development to Improve Efficacy and Safety” will include updated data on safety and tolerability, as well as biomarker and efficacy endpoints from the ongoing Phase 1/2 GALILEO-1 study, a first-in-human, international, multicenter dose-finding study in adults with Gaucher disease Type 1. Dosing has been completed in the trial. A total of six patients were treated with a single infusion of FLT201 at a dose of 4.5e11 vg/kg and have been followed for between seven and 15 months after dosing. All six patients are included in the safety analysis; one patient with detectable pre-existing neutralizing antibodies (NAbs) to the AAVS3 capsid below the protocol cut-off has been excluded from the efficacy analysis. The data as of September 27, 2024 demonstrated: Favorable safety and tolerability, with no infusion reactions or dose limiting toxicities. All treatment-related adverse events were mild to moderate in nature Durable reductions, ranging from 42% to 96%, in lyso-Gb1 levels in patients who entered the trial with persistently high levels despite years of treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT). Low lyso-Gb1 levels maintained for more than a year as of the cut-off date after the withdrawal of prior therapy in the one patient who entered the trial with well-controlled levels. Lyso-Gb1 levels in the blood are highly correlated with substrate levels in disease-affected tissues and one of the best predictors of clinical response in Gaucher disease. Substantial improvements in bone marrow burden (BMB) seen in all five patients, including those who entered the trial with severe bone involvement. Notably, BMB is correlated with increased fractures, necrosis, bone pain and joint replacements. Improvement or maintenance of spleen and liver volume. Maintenance of hemoglobin levels, an established endpoint for Gaucher disease clinical trials, was observed beyond a year post withdrawal of treatment with ERT or SRT. Improvement or maintenance of platelet counts was also seen post withdrawal of treatment with ERT or SRT. “FLT201 exemplifies our focus on developing the next generation of gene therapies, optimizing each component of our gene therapy candidates with the aim of changing the course of disease and changing people’s lives,” said Michael Parini, Spur’s Chief Executive Officer. “Our more stable GCase85 enzyme, combined with our potent and proprietary capsid, is leading to improvements in bone, pain and fatigue, all of which are key areas of ongoing need in Gaucher disease. These data highlight FLT201’s potential to provide better outcomes than current standard of care while dramatically reducing the treatment burden for patients, and we are actively engaging with regulators to advance FLT201 into a Phase 3 trial.” Spur expects to report additional data from the Phase 1/2 GALILEO-1 trial in the first half of 2025 and initiate a Phase 3 trial for FLT201 in adults with Gaucher disease type 1 in 2025. FLT201 is an adeno-associated virus (AAV) gene therapy candidate that is currently being investigated in the Phase 1/2 GALILEO-1 clinical trial in adults with Gaucher disease. FLT201 leverages Spur’s proprietary and potent AAVS3 capsid to deliver GCase85, a rationally engineered longer-acting version of the enzyme deficient in people with Gaucher disease, with the goal of stopping disease progression, reducing or eliminating symptoms, and allowing patients to come off current lifelong treatments. Preclinical and clinical data for FLT201 have shown robust and durable expression and a substantial reduction in the toxic buildup of substrate that results from the enzyme deficiency. For more information about the GALILEO-1 trial, please visit clinicaltrials.gov (NCT05324943). Gaucher disease is caused by a mutation in the GBA1 gene that results in abnormally low levels of glucocerebrosidase (GCase), an enzyme needed to metabolize a certain type of lipid. As a result, harmful substrates glucosylceramide (Gb-1) and glucosylsphingosine (lyso-Gb1) build up in cells, which then accumulate in tissues and organs throughout the body, causing inflammation and dysfunction. Despite treatment with currently approved therapies, many people with Gaucher disease continue to experience debilitating symptoms, including enlarged organs, fatigue, bone pain and reduced lung function. Gaucher disease affects approximately 18,000 people in the United States, United Kingdom, France, Germany, Spain, Italy and Israel. Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions. By optimizing every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realize outsized clinical results. Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and a potential first-in-class gene therapy candidate for adrenomyeloneuropathy, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of Parkinson’s, dementia, and cardiovascular disease. Expanding our impact, and advancing the practice of genetic medicine. The content above comes from the network. if any infringement, please contact us to modify.

CAP-003 achieved best-in-class GCase increases in NHPs exceeding levels needed for normalizing activity in patients Brainwide RNA expression greater than 200-fold higher than AAV9, with significant detargeting of liver and DRGs with proprietary engineered capsid Company on track to file IND in first half of 2025 THOUSAND OAKS, CA, USA I October 7, 2024 I Capsida Biotherapeutics (“Capsida”) presented new IND-enabling data on its intravenously (IV) delivered gene therapy for Parkinson’s disease associated with GBA mutations (PD-GBA), CAP-003, that demonstrate its substantial therapeutic potential. Capsida’s next-generation therapy, using a proprietary engineered capsid, demonstrates best-in-class potential to effectively and safely treat PD-GBA. CAP-003 achieves unprecedented levels of GCase protein and activity while simultaneously detargeting the liver and dorsal root ganglion (DRGs). These data were presented in a late-breaking poster presentation at the Society for Neuroscience “Neuroscience 2024” annual meeting being held October 5-9, 2024 in Chicago, IL. Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of PD patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase activity deficit in patients 1 . Other potential treatments for PD-GBA have been limited by their inability to cross the blood-brain barrier and supplement GCase enzyme activity in sufficient quantities to overcome the deficit in patients and impact the disease. In an attempt to overcome these challenges, they have utilized invasive direct brain or cerebrospinal fluid administration, with limited results, but significant burden for patients. Results from Capsida’s IND-enabling non-human primate (NHP) studies show brainwide RNA expression more than 200-fold greater than IV administered AAV9 and substantial increases in GCase protein and enzyme activity compared to untreated animals. A single IV infusion of CAP-003 resulted in up to six-fold increase in GCase protein compared to untreated animals in relevant brain regions. Across doses and brain regions, including the substantia nigra, the levels of GCase activity following infusion of CAP-003 was increased by up to approximately 250% compared to untreated animals. These data support the potential for meaningful clinical benefit in patients. Increases of 30% are expected to normalize GCase activity in patients with PD-GBA. NHPs dosed with CAP-003 IV have 19- and 17-fold less biodistribution to the liver and DRG, respectively, compared to IV administered wild-type AAV9. CAP-003 is well tolerated with no notable safety findings in NHPs. “Capsida’s wholly owned novel gene therapy offers the potential to normalize GCase activity in patients with a single IV infusion safely, enabling the potential for long-term disease modification and substantial slowing of disease progression,” said Peter Anastasiou, Capsida’s Chief Executive Officer. “These data give us confidence that we are on track to enter the clinic with CAP-003 in the first half of 2025.” Capsida to Present at Cell & Gene Meeting on the Mesa In addition, Capsida will present these data as part of a corporate presentation at the Cell & Gene Meeting on the Mesa taking place October 7-9, 2024, in Phoenix, AZ. Mr. Anastasiou will present a corporate update on Tuesday, October 8, 2024 at 3:45 PM local time. The Capsida corporate presentation can be accessed by visiting the News section of the Company’s website at www.capsida.com . About Capsida Biotherapeutics Capsida Biotherapeutics is a fully integrated gene therapy company with a central nervous system (CNS) pipeline consisting of disease modifying and potentially curative treatments for rare and more common diseases across all ages. Capsida’s intravenously (IV) administered gene therapies utilize proprietary engineered capsids that enable high transduction levels to desired tissues and cells, while limiting tropism to non-target organs, such as the liver. Capsida has three wholly owned programs, including potential best-in-class treatments for genetic epilepsy due to STXBP1 mutations and Parkinson’s disease associated with GBA mutations, both of which are in IND-enabling studies and on track to enter the clinic in the first half of 2025. In addition to its wholly owned programs, the Company has validating CNS partnerships with AbbVie, Lilly, CRISPR Therapeutics, and the AbbVie partnership was expanded to include ophthalmology disorders. Capsida was founded in 2019 by lead investors Versant Ventures and Westlake Village BioPartners and originated from groundbreaking research in the laboratory of Viviana Gradinaru, Ph.D., a neuroscience professor at Caltech. Visit us at www.capsida.com . 1 Gegg, M. E, et al (2012). Annals of neurology, 72(3), 455-463. SOURCE: Capsida Biotherapeutics