Protalix BioTherapeutics Receives Approval to Initiate Phase I Study in Gaucher Patients With PRX-112, an Orally-Administered C
07 Mars 2013 - 2:45PM
Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced
today that it has received approval from the Israeli Ministry of
Health to initiate a phase I clinical trial of PRX-112, or Oral
GCD, the Company's orally-administered product candidate for the
treatment of Gaucher disease. Oral GCD is a plant cell expressed
form of the glucocerebrosidase enzyme (GCD) that is naturally
encapsulated within carrot cells and administered orally. The
Company anticipates initiating this trial in two Israeli medical
centers during the next month.
"The majority of currently diagnosed patients with Gaucher
disease are treated with bi-weekly intravenous infusions of enzyme
replacement therapy. Having a safe and effective orally
available enzyme agent would potentially improve patients' quality
of life without compromising on the benefits of enzyme therapy,"
commented Dr. David Aviezer, Protalix BioTherapeutics' President
and Chief Executive Officer. "Our clinical trial programs for
IV administered plant cell-expressed GCD have already demonstrated
that the enzyme is safe and effective in treating Gaucher
disease. Now, in evaluating Oral GCD, our main goal is to
achieve therapeutic levels of the enzyme in the bloodstream after
oral consumption by patients."
The phase I clinical trial is an open label safety and
pharmacokinetic study designed to assess the delivery of prGCD
after oral administration of Oral GCD in 12 Gaucher
patients. Subjects will receive 250mL of re-suspended carrot
cells in a single oral administration during the first cohort of
the trial and three consecutive daily administrations during the
second cohort of the trial. The Company expects the phase I
trial to be completed during the second quarter of 2013.
Pre-clinical studies of oral GCD demonstrate the stability of
the enzyme in the carrot cell and the capacity of the cell's
cellulose wall to protect the enzyme against degradation in the
digestive tract in an in-vitro model of the stomach and
intestines. Additionally, both rats and pigs fed with PRX 112,
lyophilized carrot cells expressing GCD, have demonstrated enzyme
levels in the plasma and accumulation of the active enzyme in
target organs such as the spleen and liver.
Dr. Yoseph Shaaltiel, Protalix BioTherapeutics' Executive Vice
President, Research & Development added: "Our animal studies
have already demonstrated the capacity of plant cells to serve as a
vehicle for oral administration of other therapeutic proteins.
These proteins include for example, our plant cell-expressed
antiTNF fusion protein, a plant cell version of etanercept
(Enbrel®). Given the results of our preclinical studies, we
believe our Oral GCD clinical study will support our belief that we
have developed a wider platform for oral protein delivery."
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 by Israel's
Ministry of Health in September 2012. Protalix has partnered
with Pfizer Inc. for the worldwide development and
commercialization of ELELYSO™, 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
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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 the clinical trials of our product candidates will not support
our claims of 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; 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 U.S. Food and Drug Administration, 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
Marcy Nanus
The Trout Group, LLC
646-378-2927
mnanus@troutgroup.com
Media Contact
Kari Watson
MacDougall Biomedical Communications
781-235-3060
kwatson@macbiocom.com