Protalix BioTherapeutics Reports Positive Preliminary Data from the BRIDGE Study of pegunigalsidase alfa for the Treatment of...
September 21 2018 - 10:57AM
Preliminary Results Indicate 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 positive preliminary data from the
BRIDGE study of pegunigalsidase alfa (PRX-102) for the treatment of
Fabry disease. Additional data will be released at the 1st
Canadian Symposium on Lysosomal Diseases 2018.
The BRIDGE study is an open label switch over
study evaluating the safety and efficacy of PRX-102 in patients
with Fabry disease currently treated with agalsidase alfa 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 agalsidase alfa 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.
Preliminary data from the first sixteen patients enrolled in the
trial demonstrated an improvement in kidney function when switched
from agalsidase alfa (Replagal®) to pegunigalsidase alfa (PRX-102).
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 (PRX-102) was changed to be (positive) of +3.7ml/min/1.73m2,
these results were statistically significant. Baseline
characteristic 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.
The BRIDGE study is an open-label, single arm switch-over study
to assess the safety and efficacy of pegunigalsidase alfa, 1 mg/kg
infused every two weeks, in 22 Fabry patients currently treated
with Replagal. Protalix anticipates completing patient
enrollment in the BRIDGE trial in the fourth quarter of 2018.
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 disease 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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