Protalix BioTherapeutics Presents Key Clinical Data of Pegunigalsidase Alfa for the Treatment of Fabry Disease at the 16th Annual WORLDSymposium™ 2020
CARMIEL,
The Company will also deliver additional data via a poster presentation on the Phase I/II dose-ranging studies of pegunigalsidase alfa for the treatment of Fabry disease, and both a poster and oral presentation on the design of the pivotal Phase III BALANCE clinical trial of pegunigalsidase alfa for the treatment of Fabry disease by
The Company's BRIDGE clinical trial is a Phase III open-label single arm switch-over study evaluating the safety and efficacy of pegunigalsidase alfa, 1 mg/kg infused every two weeks, in up to 22 Fabry patients currently treated with agalsidase alfa (Replagal®) for at least two years and on a stable dose for at least six months.
The data to be presented via a poster by Dr. Ales Linhart of
"In addition to suggesting a positive safety and tolerability profile of pegunigalsidase alfa, these interim data indicate amelioration of the course of the disease," said Dr. Linhart. "While these results must be confirmed by the long-term data, all of the progressing patients and two-thirds of those in the 'fast progressing' group, to date, achieved the proposed therapeutic goals after switching to pegunigalsidase alfa, demonstrating substantial improvement in disease progression rate."
In previously announced interim data, pegunigalsidase alfa was found to be well tolerated in the study, with all adverse events being transient in nature without sequelae. As of today, the majority of patients who completed the study rolled over to a long-term extension study and continues to receive treatment. The BRIDGE study final results are expected by mid-2020.
Data on the Phase I/II dose ranging studies designed to evaluate the safety, efficacy and pharmacokinetics of pegunigalsidase alfa administered intravenously every other week in adult symptomatic, treatment-naïve male and female Fabry patients will be presented in a poster presentation by Prof. Derralynn Hughes of University College London (
"The results of this study demonstrate that pegunigalsidase alfa reaches the affected tissue and reduces the kidney Gb3 inclusions burden and the Lyso-Gb3 levels in circulation," said Prof. Hughes. "Further, the high correlation found between the two Fabry disease biomarkers, reduction of kidney Gb3 inclusions and reduction of plasma Lyso-Gb3 over six months of treatment, gives additional support to the potential effectiveness of pegunigalsidase alfa in treating Fabry disease."
An oral presentation, to be given by Dr. Warnock, describes the design and methods of the study protocol and the baseline characteristics for approximately 75 patients enrolled at 29 U.S. and European study sites.
The Company's Phase III BALANCE clinical trial is a fully enrolled, randomized, double blind, head-to-head active control study which aims to demonstrate pegunigalsidase alfa's superiority in kidney function over 24 months of treatment as compared to agalsidase beta (Fabrazyme®). The study enrolled adult Fabry patients that were previously treated with agalsidase beta with deteriorating renal function, where it is aimed to demonstrate clinical benefit on renal function post-switch to pegunigalsidase alfa versus patients remaining under agalsidase beta.
"The BALANCE study is designed to show pegunigalsidase alfa's solid potential to demonstrate clinical benefit in Fabry patients," said Dr. Warnock. "An improved Fabry treatment remains a significant unmet need for this underserved population, and we look forward to the completion of our study to help address this situation."
Copies of the oral presentation and the posters will be made available on the Company's website under the Presentations tab in the Investors section.
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. Fabry patients 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 and, accordingly, Gb3 accumulates, primarily in the blood and in the 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 pegunigalsidase alfa to potentially address the continued unmet clinical need in Fabry patients of continuous disease progression, infusion reactions and immunogenicity.
About
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®. Protalix was the first company to gain U.S. Food and Drug Administration (
Protalix's first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the
Protalix's development pipeline consists of proprietary, potentially clinically superior versions of recombinant therapeutic proteins that target established pharmaceutical markets, including the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human α‑Galactosidase‑A protein for the treatment of Fabry disease; OPRX-106, an orally-delivered anti-inflammatory treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in
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: failure or delay in the commencement or completion of our preclinical and clinical trials which may be caused by several factors, including: risks that the
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