~Preliminary Results Indicate Significant
Improvement in Kidney Function in Patients Switched from agalsidase
alfa (Replagal®) to pegunigalsidase alfa (PRX-102)~
Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX),
a biopharmaceutical company focused on the development and
commercialization of recombinant therapeutic proteins expressed
through its proprietary plant cell-based expression system,
ProCellEx®, today announced that positive preliminary data from the
BRIDGE study of pegunigalsidase alfa (PRX-102) for the treatment of
Fabry disease will be presented today at the 1st Canadian Symposium
on Lysosomal Diseases 2018. The symposium will take place
October 5-6, 2018 at the OLT Gouverneur Hotel in Sherbrooke,
Quebec. An oral presentation titled, “Pegunigalsidase Alfa-a
Novel Enzyme Replacement Therapy for the Treatment of Fabry
Disease: Preliminary Results from the Phase III Bridge Study,” will
be presented by Dr. Michael L. West, Professor, Division of
Nephrology, Department of Medicine, Dalhousie University, Halifax,
Nova Scotia, today, Friday, October 5 at 10:15 am ET. The
slides from Dr. West’s presentation will be available on the
Company’s website after the presentation.
The BRIDGE study is an open label 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 2 years and
on a stable dose for at least 6 months. Patients are screened
and evaluated over 3 months while continuing on agalsidase alfa.
Following the screening period, each patient was enrolled and
switched from Replagal treatment to receive intravenous (IV)
infusions of PRX-102 1 mg/kg every two weeks for 12 months.
Patients can receive PRX-102 infusions at a home care setup
based on the infusion tolerability. The Company anticipates
completing patient enrollment in the BRIDGE trial in the fourth
quarter of 2018.
As previously announced by the Company, preliminary
data from the first sixteen patients enrolled in the trial
demonstrated an improvement in kidney function, in both male and
female Fabry patients, when switched from agalsidase alfa to
pegunigalsidase alfa. Based on available historical serum
creatinine and study 3 month screening period values for
approximately 2 years while treated with agalsidase alfa before
switching to pegunigalsidase alfa treatment, the annualized
estimated glomerular filtration rate (eGFR) slope for patients on
Replagal was (negative) – -6.8ml/min/1.73m2. The mean eGFR
slope for the same patients following six months of treatment with
pegunigalsidase alfa was changed to be (positive) –
+3.7ml/min/1.73m2. These results are statistically
significant with a p-value of 0.015. Baseline characteristics
of these patients were: mean estimated glomerular filtration rate
(eGFR) 75.40 and 86.03 mL/min/1.73m2 for males and females, with
annualized eGFR slope of -8.0 and -5.1 mL/min/1.73m2/year,
respectively. This improvement in kidney function (e.g.,
eGFR) over time may potentially result in the delay or prevention
of kidney failure in these populations.
The enzyme has been well tolerated in the study,
with all adverse events being transient in nature without sequelae.
Most of the patients who are eligible for home care therapy
per country regulation are being treated under a home care
arrangement in which certain of the scheduled infusions are
performed at the patients’ home, and the first patient who
concluded the study has opted to enroll in a long-term extension
study and continues to be treated with PRX-102.
“These results are very encouraging and show that
pegunigalsidase alfa (PRX-102) has the potential to provide a
better enzyme replacement therapy for Fabry patients. I am
excited to be a part of this trial, and I look forward to
continuing to work with Protalix as pegunigalsidase alfa progresses
through its potential approval and availability for the treatment
of Fabry patients,” said Dr. Michael West, Professor, Division of
Nephrology, Department of Medicine, Dalhousie University, Halifax,
Nova Scotia.
In addition, in vitro analysis of PRX-102 in both human plasma
and in lysosomal-like conditions shows significantly longer
stability of enzyme activity compared to both
commercially-available enzyme replacement therapies (ERTs).
In lysosomal-like conditions, approximately 84% of PRX-102’s
activity was shown to have been preserved after 10 days compared to
approximately 1% remaining enzyme activity in each of the
commercially available ERTs. These results were statistically
significant with a p-value of 0.01 (Kizhner T., et al (2015)
Molecular Genetics and Metabolism 114: 259–267).
PRX-102 is the Company’s plant cell-expressed recombinant,
PEGylated, cross-linked α-galactosidase-A for Fabry disease.
In pre-clinical and clinical studies, PRX-102 demonstrated
higher stability in plasma, a longer half-life and higher exposure
in Fabry patients, and a reduction in Gb3 in kidney biopsies in
treatment naïve Fabry patients.
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’s unique expression
system presents a proprietary method for developing recombinant
proteins in a cost-effective, industrial-scale manner.
Protalix’s first product manufactured by ProCellEx, taliglucerase
alfa, was approved for marketing by the U.S. Food and Drug
Administration (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 includes the following product
candidates: pegunigalsidase alfa, a modified version of the
recombinant human alpha-GAL-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 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: 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; inability or
unwillingness of medical investigators and institutional review
boards to follow our clinical protocols; and lack of sufficient
funding to finance clinical trials; the risk that the results of
the clinical trials of our product candidates will not support our
claims of superiority, 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 Chiesi Farmaceutici and any other collaborator,
distributor or partner; risks related to the amount and sufficiency
of our cash and cash equivalents; risks related to the ultimate
purchase by Fundação Oswaldo Cruz of alfataliglicerase
pursuant to the stated purchase intentions of the Brazilian
Ministry of Health of the stated amounts, if at all; risks
related to the successful conclusion of our negotiations with
the Brazilian Ministry of Health regarding the purchase
of alfataliglicerase generally; risks related to our
commercialization efforts for alfataliglicerase in Brazil;
risks relating to the compliance by Fundação Oswaldo
Cruz with its purchase obligations and related milestones
under our supply and technology transfer agreement; risks related
to the amount and sufficiency of our cash and cash equivalents;
risks related to the amount of our future revenues, operations and
expenditures; the risk that despite the FDA’s grant of fast track
designation for pegunigalsidase alfa for the treatment of Fabry
disease, we may not experience a faster development process, review
or approval compared to applications considered for approval under
conventional FDA procedures; risks related to the FDA’s
ability to withdraw the fast track designation at any time; 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 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
Marcy Nanus, Managing Director Solebury Trout 646-378-2927
mnanus@soleburytrout.com
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