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Protalix BioTherapeutics Announces Completion of the Treatment Period for its Phase III BRIGHT Clinical Trial of Pegunigalsidase Alfa (PRX-102) for the Proposed Treatment of Fabry Disease
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Aug
24
CARMIEL, "Completion of the BRIGHT study marks another important key milestone in our pursuit of an alternative dose and regimen of pegunigalsidase alfa for the proposed treatment of Fabry disease," said "Completion of the treatment period of the BRIGHT study represents another important milestone in the effort to advance this development program, and we are grateful to all of the patients and clinicians who have been dedicated to moving this program forward in spite of the challenges of COVID-19," said About the Phase III BRIGHT Study The BRIGHT study is a phase III, open label, switch over study to assess the safety, efficacy and pharmacokinetics of pegunigalsidase alfa (PRX–102) 2 mg/kg administered by intravenous infusion every four weeks for 52 weeks in patients with Fabry disease currently treated with enzyme replacement therapy (ERT): agalsidase alfa or agalsidase beta. About Fabry Disease Fabry disease is an X-linked inherited disease that results from deficient activity of the lysosomal α–Galactosidase–A enzyme resulting in progressive accumulation of abnormal deposits of a fatty substance called globotriaosylceramide (Gb3) in blood vessel walls throughout a person's body. Fabry disease occurs in one person per 40,000 to 60,000. People living with Fabry inherit a deficiency of the α–Galactosidase–A enzyme, which is normally responsible for the breakdown of Gb3. The abnormal storage of Gb3 increases with time, accumulating primarily in blood and in blood vessel walls. The ultimate consequences of Gb3 deposition range from episodes of pain and impaired peripheral sensation to end-organ failure – particularly of the kidneys, but also of the heart and the cerebrovascular system. About Pegunigalsidase Alfa Pegunigalsidase alfa (PRX–102) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant α-Galactosidase-A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX–102 has been observed to have a circulatory half-life of approximately 80 hours. The Company designed PRX–102 to potentially address the continued unmet clinical need in Fabry patients. About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the About Based in About 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," "plan," "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: that the FDA might not grant marketing approval for PRX–102 by the PDUFA date or at all and, if approved, whether PRX–102 will may have significant limitations on its use or be commercially successful; risk that the FDA will request additional data or other conditions of the Biologics License Application (BLA) filing for Accelerated Approval of PRX–102; failure or delay in the commencement or completion of our preclinical and 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 monitor patients adequately during or after treatment; and inability or unwillingness of medical investigators and institutional review boards to follow our clinical protocols; risks associated with the novel coronavirus disease (COVID–19) outbreak, which may adversely impact our business, preclinical studies and clinical trials; the risk that the results of the clinical trials of our product candidates will not support our claims of safety or efficacy, that our product candidates will not have the desired effects or will be associated with undesirable side effects or other unexpected characteristics; risks related to our ability to maintain and manage our relationship with any collaborator, distributor or partner; risks related to the amount and sufficiency of our cash and cash equivalents; risks relating to our ability to make scheduled payments of the principal of, to pay interest on or to refinance our outstanding notes or any other indebtedness; our dependence on performance by third party providers of services and supplies, including without limitation, clinical trial services; delays in our preparation and filing of applications for regulatory approval; delays in the approval or potential rejection of any applications we file with the FDA or other health regulatory authorities, and other risks relating to the review process; our ability to identify suitable product candidates and to complete preclinical studies of such product candidates; 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 and institutions; potential product liability risks, and risks of securing adequate levels of product liability and other necessary insurance coverage; and other factors described in our filings with the Investor Contact Media Contact View original content to download multimedia:http://www.prnewswire.com/news-releases/protalix-biotherapeutics-announces-completion-of-the-treatment-period-for-its-phase-iii-bright-clinical-trial-of-pegunigalsidase-alfa-prx-102-for-the-proposed-treatment-of-fabry-disease-301116942.html SOURCE |