Protalix BioTherapeutics Treats First Gaucher Patient in Phase I Study With PRX-112, an Orally-Administered Enzyme Treatment of
02 April 2013 - 1:30PM
Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced
today that the first patient has been treated in the Company's
phase I clinical trial of PRX-112, or Oral GCD, the Company's
orally-administered enzyme 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. Protalix expects
the phase I trial to be completed during the third quarter of
2013.
"With Oral GCD, we are using the natural characteristics of
plant cells to deliver active enzyme into the patient's blood
stream," said Professor Ari Zimran, M.D., Director of the Gaucher
Clinic in Shaare Zedek Medical Center, Jerusalem, Israel and lead
clinical investigator. "We believe oral delivery of GCD has
the potential to improve patients' quality of life without
compromising the efficacy or safety of the treatment."
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 receive 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.
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 Einat Brill Almon, the Company's Senior Vice President,
Product Development, added: "We successfully demonstrated the
ability of plant cells to act as an oral delivery mechanism for a
number of therapeutic proteins in our animal studies. We
believe that the results of our clinical trial of Oral GCD will
provide additional support for our belief that this oral delivery
mechanism can be developed for other proteins used to treat other
indications."
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,
taliglucerase alfa, was approved for marketing by the U.S. Food and
Drug Administration (FDA) in May 2012, by Israel's Ministry of
Health in September 2012 and by the Brazilian National Health
Surveillance Agency (ANVISA) in March 2013. It also has been
approved in Uruguay. Marketing applications for taliglucerase
alfa have been filed in additional territories as
well. Protalix has partnered with Pfizer Inc. for the
worldwide development and commercialization of taliglucerase alfa,
excluding Israel, where Protalix retains full
rights. 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.
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strictly historical, all such statements are forward-looking, and
are made pursuant to the safe-harbor provisions of the Private
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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 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
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including without limitation, clinical trial services; delays in
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inherent risks and uncertainties in developing drug platforms and
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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
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