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Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients

Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for
Oral GCD in Gaucher Disease Patients

CARMIEL, Israel, Oct. 14, 2013 (GLOBE NEWSWIRE) -- Protalix BioTherapeutics,
Inc. (NYSE MKT:PLX) (TASE:PLX), announced today initial positive results from
its phase I clinical trial of oral glucocerebrosidase (GCD), or Oral GCD
(PRX-112), in patients with Gaucher disease. In the trial, Oral GCD was
well-tolerated, and active enzyme was detected in patients' blood
circulation.Oral GCD is an orally available form of the plant cell-expressed
enzyme, glucocerebrosidase or GCD, which is the same active substance as the
Company's approved enzyme replacement therapy, ELELYSO™.Oral GCD is an active
form of human glucocerebrosidase which is naturally encapsulated within the
carrot cells in which it is produced.

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 Gaucher patients.Patients received one of three doses of re-suspended
carrot cells in a single oral administration during the first segment of the
trial and three consecutive daily administrations during the second segment of
the trial.The study is being conducted in two sites; at Shaare Zedek Medical
Center, Jerusalem, Israel, and at the Rambam Medical Center, Haifa, Israel.

Overall, oral GCD was found to be safe and well tolerated in all 12 patients
across all of the three doses tested. There were no serious adverse reactions
reported and no patient discontinued the study prematurely. Presence of an
enzyme was detected in patients' blood circulation and the enzyme demonstrated
biological activity. In addition, some of the patients who suffered from
thrombocytopenia and had low platelet counts demonstrated a meaningful
improvement in platelet count.Accordingly, the trial has been extended to
enroll and evaluate additional Gaucher patients with low platelet counts. The
amended study is expected to conclude during the fourth quarter of 2013.
Additional data from the phase I study will be presented at the 10^th Annual
WORLD Symposium, February 10-13, 2014 in San Diego, CA.

"Oral administration of proteins has been a long time challenge for the
biopharmaceutical industry due to the early degradation of proteins in the
digestive track.The results of this phase I trial provide compelling proof of
concept data for the oral delivery of enzymes using plant cells as a delivery
platform," commented Prof. Ari Zimran, Associate Professor and Director of the
Gaucher Clinic at Shaare Zedek and Principal Investigator. "Oral delivery of
GCD has the potential to dramatically change the way Gaucher patients are
treated—not only improving patients' quality of life by eliminating the need
for bi-weekly infusions, but perhaps also having clinical benefit due to the
steady maintenance of enzyme levels in patients' blood circulation."

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 theU.S. Food and Drug Administration (FDA) inMay 2012,
byIsrael'sMinistry of HealthinSeptember 2012, by theBrazilian National
Health Surveillance Agency(ANVISA) inMarch 2013, by theMexican Federal
Commission for the Protection against Sanitary Risk (COFEPRIS) inApril 2013,
and by the regulatory authorities of other countries. Marketing applications
for taliglucerase alfa have been filed in additional territories as well.
Protalix has partnered withPfizer Inc.for the worldwide development and
commercialization of taliglucerase alfa, excludingIsraelandBrazil, 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 produced and encapsulated within 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; PRX-110 for
the treatment of Cystic Fibrosis; PRX-107 for the treatment of emphysema due
to hereditary alpha1-antitrypsin deficiency; 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. 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 preclinical
studies and 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 trials will not support the applicable claims of safety or
efficacy, that our product candidates will not have the desired effects or
will include 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 theFDA 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 other
factors described in our filings with theU.S. Securities and Exchange
Commission. These forward-looking statements are based on current information
that may change and you are cautioned not to place undue reliance on these
forward-looking statements. The statements in this release are valid only as
of the date hereof and we disclaim any obligation to update this information.
All forward-looking statements are qualified in their entirety by this
cautionary statement.

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
 
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