Protalix BioTherapeutics Receives FDA IND Clearance to Initiate a Phase I/II Study of Fabry Disease Patients With PRX-102, a Mo
August 13 2012 - 7:30AM
Protalix BioTherapeutics, Inc. (NYSE-MKT:PLX) (TASE:PLX), announced
today that it has received clearance of its Investigational New
Drug (IND) application from the U.S. Food and Drug Administration
(FDA) to initiate clinical trials of PRX-102. The Company plans to
commence enrollment of Fabry disease patients for a phase I/II
trial in the fourth quarter of 2012.
PRX-102 is a proprietary plant cell-expressed, chemically
modified, recombinant alpha-galactosidase-A in development as a
long-term enzyme replacement therapy (ERT) for the treatment of
Fabry disease. The phase I/II clinical trial is designed as a
multi-center, open label, dose ranging study to evaluate the
safety, tolerability, pharmacokinetics and efficacy of PRX-102 in
adult Fabry patients.
"We are very excited to begin the clinical development of
PRX-102, which we believe may prove to present an important
improvement to the well being of patients with Fabry disease, a
rare, genetic lysosomal storage disorder affecting approximately
8,000 people globally," said Dr. David Aviezer, Protalix's
President and Chief Executive Officer. "We have designed PRX-102 as
a potentially improved version of the currently marketed enzyme
replacement therapies for Fabry disease given its potential to be a
more stable, potent and specific enzyme. This enzyme is
expressed through ProCellEx®, our proprietary, plant cell-based
protein expression system. We are also excited that another
biotherapeutic protein evolving from our ProcellEx platform
technology is anticipated to enter clinical development
shortly."
Eighteen adult Fabry patients will be enrolled in one of three
dosing groups. Each patient will receive intravenous infusions
of PRX-102 every two weeks for 12 weeks, and will be infused
sequentially and stepwise in order to evaluate
safety. Exploratory efficacy parameters will be evaluated as a
preliminary assessment. Following the end of the trial, the Company
intends to offer patients the option to continue to receive PRX-102
in an open-label extension study.
About Protalix
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,
ELELYSO™ (taliglucerase alfa), was approved for marketing by the
U.S. Food and Drug Administration on May 1, 2012 and is partnered
with Pfizer Inc. for worldwide development and commercialization,
excluding Israel, where Protalix retains full
rights. Marketing applications for taliglucerase alfa have
been filed in additional territories as well. Protalix's
development pipeline also includes the following product
candidates: PRX-102, a modified version of the recombinant human
alpha-GAL-A protein for the treatment of Fabry disease; PRX-105, a
pegylated recombinant human acetylcholinesterase in development for
several therapeutic and prophylactic indications, a biodefense
program and an organophosphate-based pesticide treatment program;
an orally-delivered glucocerebrosidase enzyme that is naturally
encased in carrot cells, also for the treatment of Gaucher disease;
pr-antiTNF, a similar plant cell version of etanercept (Enbrel®)
for the treatment of certain immune diseases such as rheumatoid
arthritis, juvenile idiopathic arthritis, ankylosing spondylitis,
psoriatic arthritis and plaque psoriasis; and others.
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
"anticipate," "believe," "estimate," "expect," "plan" 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. Factors that might cause material differences
include, among others: failure or delay in the commencement or
completion of our clinical trials which may be caused by several
factors, including: unforeseen safety issues; determination of
dosing issues; lack of effectiveness during clinical trials; slower
than expected rates of patient recruitment; 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 the clinical trials; the risk that the results
of our clinical trial of PRX-102 will not support our claims of
safety or efficacy, that PRX-102 will not have the desired effects
or includes undesirable side effects or other unexpected
characteristics; 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; 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 clinical trial insurance coverage; and other
factors described in our filings with the U.S. Securities and
Exchange Commission. The statements in this release are valid
only as of the date hereof and we disclaim any obligation to update
this information.
CONTACT: Investor Contact
Tricia Swanson
The Trout Group, LLC
646-378-2953
tswanson@troutgroup.com
Media Contact
Jennifer Conrad or Kari Watson
MacDougall Biomedical Communications
781-235-3060
jconrad@macbiocom.com
kwatson@macbiocom.com