Full Press Release Details
Protalix BioTherapeutics Presents Key
of Pegunigalsidase Alfa for the Treatment of Fabry Disease
at the 16th Annual WORLDSymposium 2020
CARMIEL, Israel, February 10, 2020 /PRNewswire/ -- Protalix
BioTherapeutics, Inc. (NYSE American: PLX) (TASE: 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, announced today that one-year interim data from the ongoing Phase III BRIDGE clinical trial of the Company's
pegunigalsidase alfa (PRX-102) candidate for the treatment of Fabry disease will be presented via a poster presentation at the
16th Annual WORLDSymposium 2020 in Florida.
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 David Warnock, M.D., University of Alabama at Birmingham, a principal investigator in the Company's BALANCE trial.
The Company has already announced the schedule of the presentations.
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
Charles University in Prague, Czech Republic, a principal investigator in the BRIDGE study, suggest that there exists a
potential benefit of pegunigalsidase alfa on renal function for Fabry patients previously treated with agalsidase alfa.
"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-na ve male and female Fabry patients will be presented in a poster presentation by Prof. Derralynn
Hughes of University College London (UK), a principal investigator in the Company's Phase III
clinical trial of PRX-102.
"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
"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.
Fabry disease is an X-linked inherited disease that results
from deficient activity of the lysosomal -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 -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 -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 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 . Protalix was 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. Protalix's unique expression system represents a new method
for developing recombinant proteins in an industrial-scale manner.
Protalix's first product manufactured by ProCellEx, taliglucerase
alfa, was approved for marketing by the FDA in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix
has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where
Protalix retains full rights.
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 -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 the United States and outside the
United States, for the development and commercialization of pegunigalsidase alfa.
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 FDA will not accept an application for accelerated
approval of PRX-102 with the data generated to date or will request additional data or other conditions of our submission of any
application for accelerated approval of PRX-102; 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; the risk