Naxos Disease

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM) is a devastating genetic cardiac disease affecting approximately 60,000 individuals in the United States LX2020 preclinical studies demonstrated proof-of-concept with treatment restoring desmosomal integrity, reducing arrhythmias, and extending survival in a clinically relevant murine model Second cardiac gene therapy IND clearance bolsters LEXEO’s clinical stage pipeline LEXEO Therapeutics (LEXEO), a clinical-stage gene therapy company advancing adeno-associated virus (AAV)-based gene therapy candidates for genetically defined cardiovascular diseases and a genetically defined sub-group of Alzheimer’s disease, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for LX2020. LX2020 is an AAVrh10-based gene therapy candidate designed to intravenously deliver a functional PKP2 gene to cardiac muscle for the treatment of Arrhythmogenic Cardiomyopathy (ACM) caused by variants in the PKP2 gene (PKP2-ACM). “This IND clearance marks an important step in advancing a potential one-time treatment for individuals with PKP2-ACM, who are in need of more effective options for this devastating disease. Current clinical management strategies are only marginally effective and primarily focus on symptom management,” said Eric Adler, M.D., Chief Scientific Officer of LEXEO. “LX2020 seeks to address the underlying cause of this disease by delivering a functional PKP2 gene to halt progression and reverse the disease phenotype. We look forward to initiating the Phase 1/2 clinical trial and rapidly advancing the program through the clinic.” LX2020 is designed to intravenously deliver a functional PKP2 gene to cardiac muscle to increase PKP2 protein levels in the cardiac desmosome, a cell structure critical for cell adhesion. The program is based on collaborative research led by Dr. Farah Sheikh at the University of California, San Diego School of Medicine, which utilized a genetic mouse model of PKP2-ACM that displays the disease phenotype. In preclinical studies, LX2020 treatment demonstrated restoration of desmosomal and cell-cell junction integrity, leading to improvements in cardiac tissue composition and integrity, alleviation of arrhythmias as well as improvements in right and left ventricular function resulting in increased survival. The planned Phase 1/2 trial, HEROIC-PKP2, is a first in human, 52-week open-label, dose-escalating, multicenter trial to determine the safety and tolerability of LX2020 in adult subjects with PKP2-ACM. Preliminary efficacy measures will evaluate myocardial protein expression, biomarkers measuring cardiac structure and function, and arrhythmia burden. LX2020 will be administered as a one-time intravenous infusion to patients in two ascending-dose cohorts of three patients each, with the potential for cohort expansion. Long-term safety and efficacy will be evaluated for an additional four years following completion of the initial trial. About PKP2 Arrhythmogenic Cardiomyopathy PKP2-ACM is a genetic heart disease primarily characterized by myocardial cell loss and the replacement of heart muscle with fibrotic tissue and fatty deposits with a prevalence of approximately 60,000 individuals in the United States. ACM can result from variants in several desmosomal genes, with variants in the PKP2 gene accounting for a majority of cases. These genetic variants impair the structure and function of cardiac desmosomes, which are membrane protein complexes engaged in cell-to-cell adhesion, as well as the structural integrity of the ventricular myocardium. Lack of functioning cardiac desmosomes can lead to myocardial cell death and fibrosis, heart dysfunction, rhythm abnormalities, and sudden death. Currently, there are no approved treatments to slow, prevent, or reverse disease progression in patients with PKP2-ACM. Current standard-of-care treatments include antiarrhythmics, implantable cardioverter-defibrillators (ICDs), and ablation procedures, none of which address the underlying cause of myocardial dysfunction and ACM. About LEXEO Therapeutics LEXEO Therapeutics is a New York City-based, clinical-stage gene therapy company focused on addressing some of the most devastating genetically defined cardiovascular diseases and a genetically defined sub-group of Alzheimer’s disease. LEXEO’s foundational science stems from partnerships and exclusive licenses with leading academic laboratories at Weill Cornell Medicine and the University of California, San Diego. LEXEO is advancing a deep and diverse pipeline of AAV-based gene therapy candidates for diseases affecting both larger-rare and prevalent patient populations and is led by pioneers and experts with decades of collective experience in genetic medicines, rare disease drug development, manufacturing, and commercialization. For more information, please visit www.lexeotx.com or LinkedIn. The content above comes from the network. if any infringement, please contact us to modify.

BOSTON, MASSACHUSETTS, Feb. 22, 2023 (GLOBE NEWSWIRE) -- BIOXYTRAN, INC. (OTCQB: BIXT) (the “Company”), a clinical stage biotechnology company developing oral and intravenous drugs to treat COVID-19, other viral diseases, and fibrosis announced today that Nature Reviews Drug Discovery journal published a peer-reviewed article that supports the rationale behind the development of ProLectin-I for fibrosis and ProLectin-M as an antiviral drug. The article published on 9 February, 2023, was titled “Targeting galectin-driven regulatory circuits in cancer and fibrosis“ was authored by Karina Marino, Alejandro Cagnoni, Diego Croci, and Gabriel Rabinovich. The review article independently supports Bioxytran’s drug development program for fibrosis and antiviral disease treatment. The theoretical basis of the drug design was inhibition of multiple galectins like those highlighted in the journal article. https://www.nature.com/articles/s41573-023-00636-2 The article highlighted 3 out of 5 polysaccharides that Dr. David Platt invented as therapies over his 30-year career in galectins. The article concluded that galectins shared a common pro-fibrotic function and that disease conditions like NASH, Idiopathic Pulmonary Fibrosis (IPF) and malignancies could benefit from targeting galectins as well as a diverse group of other disease states implicated by heightened galectin expression. Bioxtyran developed multiple galectin antagonists and the review article independently bolstered the scientific basis of its drug development. About ProLectin-M ProLectin-M is an oral galectin antagonist that prevents the entry of the SARS-CoV-2 virus into human cells. In recent clinical trials the drug achieved a 100% responders rate of negative PCR tests by day 7. In 3 days the drug achieved an 88% responders rate of negative PCR tests. The treated population experienced no viral rebounds during the 14-day observation period. The company is preparing for a phase 3 clinical trial in order to seek regulatory approval. About ProLectin-I ProLectin-I is an intravenous new chemical entity drug that is expected to treat Long COVID and Idiopathic Pulmonary Fibrosis (IPF). Long COVID is estimated to have 65 – 100 million cases worldwide.1, 2 The CDC believes it affects one in five people that contract COVID-19. According to Harvard University, the economic cost of Long COVID is $3.7 trillion in the just the United States.3 IPF affects approximately 3 million people worldwide.4 The disease primarily affects patients over the age of 50 and affects more men than women. 5 The top theory behind the pathogenesis of Long Covid is viral persistence6 or viral fragments. ProLectin-I binds to the ‘galectin fold’ of the spike protein thereby neutralizing a replication competent viruses’ ability to infect other cells, but it also binds to spike protein fragments thought to be the cause of ongoing inflammation. 7 The medical term for scar tissue is fibrin. The word fibrosis stems from the continued growth of fibrin. Once scar tissue forms in the lungs combined with an already suppressed immune system, scar tissue can start to spread quickly. The reason for scar tissue forming in the lungs can vary, but it can always be associated with the onset of lung damage. Combining lung damage with an impaired immune system leads to scar tissue forming in the lungs. Multiple galectin types are associated with fibrosis, and ProLectin-I is thought to bind to a few. About Bioxytran, Inc. Bioxytran, Inc. is a clinical stage biotechnology company developing novel therapies targeting the treatment of significant unmet medical needs in virology, degenerative disease, and hypoxia. The leading drug candidates, ProLectin-M (“PLM”) and ProLectin-I (“PLI”), are a new class of antiviral drugs designed to antagonize galectins implicated in viral, inflammatory, fibrotic, and malignant diseases. Bioxytran’s other development programs are for pulmonary fibrosis and stroke treatment. More information can be found at www.bioxytraninc.com Investor RelationsMichael Sheikh509-991-0245mike.sheikh@bioxytraninc.com Forward-Looking Statements This press release includes forward-looking statements as defined under federal law, including those related to the performance of technology described in this press release. These forward-looking statements are generally identified by the words “believe,” “expect,” “anticipate,” “estimate,” “intend,” “plan,” and similar expressions, although not all forward-looking statements contain these identifying words. Such statements are subject to significant risks, assumptions and uncertainties. Known material factors that could cause Bioxytran’s actual results to differ materially from the results contemplated by such forward-looking statements are described in the forward-looking statements and risk factors in the Company’s Annual Report on Form 10-K for the fiscal year ended December 31, 2021 and those risk factors set forth from time-to-time in other filings with the Securities and Exchange Commission. Bioxytran undertakes no obligation to correct or update any forward-looking statement, whether as a result of new information, future events, or otherwise, except to the extent required under federal securities laws. 1Davis, H.E., McCorkell, L., Vogel, J.M. et al. Long COVID: major findings, mechanisms and recommendations. Nat Rev Microbiol (2023). https://doi.org/10.1038/s41579-022-00846-22 Chen Chen, et al. Global Prevalence of Post-Coronavirus Disease 2019 (COVID-19) Condition or Long COVID: A Meta-Analysis and Systematic Review, The Journal of Infectious Diseases, Volume 226, Issue 9, 1 November 2022, Pages 1593–1607, https://doi.org/10.1093/infdis/jiac1363 Cutler, D.M. The Economic Cost of Long Covid: An Update. Harvard University, July 2022.4 Nalysnyk, L., et al. Incidence and Prevalence of Idiopathic Pulmonary Fibrosis: Review of the Literature. Eur. Respir. Rev. 2012;21(126):355-3615 Raghu, G., et al. An Official ATS/ERS/JRS/ALAT Statement: Idiopathic Pulmonary Fibrosis: Evidence-based Guidelines for Diagnosis and Management. Am J Respir Crit Care Med. 2011; 183:788–8246 Peluso, M.J. and Deeks, S.G. Early clues regarding the pathogenesis of long-COVID. Trends in Immunology, Volume 43, Issue 4, April 2022, Pages 268-270.7 Patterson BK, et al. (2022). Persistence of SARS CoV-2 S1 Protein in CD16+ Monocytes in Post-Acute Sequelae of COVID-19 (PASC) up to 15 Months Post-Infection. Front. Immunol. 12:746021. doi: 10.3389/fimmu.2021.746021