Protalix BioTherapeutics Completes Enrollment in the Phase III BRIGHT Clinical Trial of pegunigalsidase alfa (PRX 102) for th...
June 17 2019 - 07:00AM
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 the completion of enrollment in the
phase III BRIGHT clinical trial of pegunigalsidase alfa, or
PRX‑102, for the treatment of Fabry disease, via intravenous (IV)
infusions of 2 mg/kg administered every 4 weeks. PRX‑102 is
the Company’s plant cell-expressed recombinant, PEGylated,
cross-linked α‑galactosidase‑A drug candidate.
“We are grateful to the participants and our investigators for
their dedication and support of the study,” said Moshe Manor,
Protalix’s President and Chief Executive Officer. “Completion
of enrollment marks a key milestone for us. As Fabry disease
is a chronic illness with no cure that requires enzyme replacement
therapy infusions every 2 weeks under the current standard of care,
the possibility of doubling the time between infusions has the
potential to greatly enhance the quality of life for some portion
of these patients.”
The BRIGHT study is a 12 month, open-label switchover study to
assess the safety, efficacy and pharmacokinetics (PK) of
pegunigalsidase alfa 2 mg/kg administered every 4 weeks in up to 30
Fabry patients previously treated with an enzyme replacement
therapy (ERT): Fabrazyme® or Replagal®. To determine
eligibility for participation in the study, candidates were
screened to identify and select Fabry patients with stable kidney
disease. Patients that matched the criteria were enrolled in
the study and switched from their current treatment of intravenous
(IV) infusions every 2 weeks to 2 mg/kg of PRX‑102 every 4 weeks
for 12 months.
Patients participating in the study are being evaluated to,
among other disease parameters, determine if their kidney disease
has not further deteriorated while being treated with the four‑week
dosing regimen as measured by eGFR and Lyso Gb3, as well as other
parameters. In addition, participating patients are being
evaluated to assess the safety and tolerability of PRX‑102.
To date, substantially all patients that were enrolled in the
BRIGHT study remain on the 4‑week dosing regimen, and all of the
patients that completed the study opted, with the advice of the
treating physician, to continue treatment under the 4‑week dosing
regimen in a long-term extension study.
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 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; lack of sufficient
funding to finance clinical trials; 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 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 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
Alan Lada, Vice President Solebury Trout 617-221-8006
alada@soleburytrout.com
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