Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced
today that new clinical data on ELELYSO™ (taliglucerase alfa) will
be presented at the 9th Annual Meeting of the Lysosomal Disease
Network: WORLD Symposium 2013 being held February 13-15 in Orlando,
Florida. ELELYSO, the Company's first commercial product, is the
first FDA-approved plant cell-based enzyme replacement therapy for
Gaucher disease.
"The data to be presented at the conference further reinforce
the use of ELELYSO as a treatment option for Gaucher patients,
including naïve Gaucher patients, and patients who were previously
treated with imiglucerase (Cerezyme®)," stated Professor Ari
Zimran, M.D., Director of the Gaucher Clinic in Shaare Zedek
Medical Center, Jerusalem, Israel and lead clinical investigator.
"With the approval of ELELYSO in Israel, I am pleased to be able to
provide a new treatment option to my patients."
Gregory Pastores, M.D., Professor of Neurology and Pediatrics
and Director of the Neurogenetics Laboratory at the New York
University School of Medicine, is presenting long-term data from
the Company's multi-center, open-label switchover extension trial
of ELELYSO for the treatment of Gaucher disease. The Company's
original switchover trial was a nine-month trial in which patients
with stable disease were switched from treatment via intravenous
infusions of imiglucerase (Cerezyme®) to intravenous infusions of
ELELYSO every two weeks at an equivalent dose to the patient's
previous imiglucerase dose. Patients who participated in the
switchover trial were given the option to continue treatment with
ELELYSO in the Company's switchover extension trial.
Twenty-five adult patients completed the switchover trial, of
which 19 elected to continue treatment with ELELYSO through the
long-term extension trial. Five of the six patients who did not
enroll in the extension trial continued nonetheless to receive
ELELYSO through the Company's various compassionate use programs.
One patient was unable to comply with the study protocol and
therefore was not eligible to participate in the extension
trial.
A 24 month interim analysis of the switchover trial demonstrates
that all patients remained stable with regard to all key disease
parameters, spleen volume, liver volume, platelet count and
hemoglobin concentration, as well as the chitotriosidase activity
biomarker after switching to ELELYSO from imiglucerase. The safety
analysis presented for the 24-month switchover treatment duration
demonstrates that ELELYSO was well tolerated, and no drug related
serious adverse events were reported. One patient developed
neutralizing IgG antibodies that were determined to be positive in
an in vitro assay, and were determined to be negative in a
cell-based assay. Four of the 19 patients enrolled in the
extension trial discontinued treatment; one switched to the ELELYSO
compassionate use program, one enrolled in another clinical trial,
one was unable to comply with the study protocol and one was not
pleased with that individual's personal results. In
conclusion, the data demonstrates that ELELYSO has a
well-established safety profile and is an effective alternative
treatment for adult Gaucher patients treated previously with
Cerezyme.
These results will also be presented during the poster sessions,
which will take place on Wednesday, February 13 from 4:30-6:30 PM
ET and on Thursday, February 14 from 4:30-6:00 PM ET.
Professor Ari Zimran is presenting a poster describing long-term
safety and efficacy data from the Company's double-blind, follow-on
extension study of ELELYSO for the treatment of Gaucher disease in
adult naïve patients. Eligible patients who completed
treatment in the Company's pivotal nine-month phase III clinical
trial were offered the opportunity to participate in the extension
study and continue to receive ELELYSO at the same dose they
received in the pivotal trial for an additional 30 months in a
blinded manner. Accordingly, the extension trial included two
treatment groups; one treated with a 60 U/kg dose and the other
with a 30 U/kg dose. The primary endpoint of the extension
trial was the percent change from baseline in spleen
volume. Major secondary endpoints included percentage change
from baseline in hemoglobin concentration, liver volume, platelet
count and chitotriosidase activity. Twenty-six patients
enrolled in the extension trial which was performed in centers
throughout Europe, Israel, North America, South America and South
Africa.
At thirty-six months of treatment, both the primary and major
secondary efficacy endpoints were achieved. Mean spleen volume
decreased 62% and 47% in each of the 60 U/kg dose and 30 U/kg dose
groups, respectively; mean hemoglobin concentration increased by 3
g/dL, from 11.0 g/dL to 14.0 g/dL, in the 60 U/kg dose group and by
1.9 g/dL, from 12.4 g/dL to 14.3 g/dL, in the 30 U/kg dose group;
mean liver volume decreased 19% and 21% in each of the 60 U/kg dose
and 30 U/kg dose groups, respectively; mean platelet counts
increased by 62,972 /mm3, from 73,055 to 136,027 /mm3, in the 60
U/kg dose group and by 29,783 /mm3, from 64,900 to 94,683/mm3, in
the 30 U/kg dose group; and mean chitotriosidase activity decreased
83.0% and 73.5% for each of the 60 U/kg dose and 30 U/kg dose
groups, respectively.
The safety analysis presented for both treatment groups
demonstrates that ELELYSO was well tolerated, and no drug related
serious adverse events were reported. Two participants
developed neutralizing IgG antibodies that were determined to be
positive in an in vitro assay, and were determined to be
negative in a cell-based assay. In addition, one patient in
the 60 U/kg dose group experienced a hypersensitivity reaction
during month 10 of treatment. Treatment of this patient with
ELELYSO has been continued with premedication for an additional 44
months without any treatment related adverse events
reported. Three of the 26 participants enrolled in the
extension trial discontinued treatment; one switched into the
ELELYSO compassionate use program, one was unable to comply with
study protocol and one had a skin reaction during month 15.
The long-term safety and efficacy results from the naïve adult
patient extension study demonstrate that ELELYSO has a
well-established safety profile and is an effective long-term
treatment for Gaucher disease.
Professor Zimran will also present a poster entitled "A
Multicenter, Double-Blind, Randomized Safety and Efficacy Study of
Two Dose Levels of Taliglucerase Alfa in Pediatric Patients with
Gaucher Disease." These data were first announced by the
Company at the 10th Annual European Working Group on Gaucher
Disease Meeting in June 2012.
Copies of the posters are being posted on the investor relations
page of the Company's website, www.protalix.com. The content
of the Company's website is not intended to be incorporated by
reference into this press release or in any report or document we
file.
Safety Information for ELELYSO™
As with any intravenous protein product, allergic reactions,
some severe, were reported in the taliglucerase alfa clinical
trials. A definition of anaphylaxis (as defined by Sampson et
al 2006) was retrospectively applied to some of these reports. In
patients who have experienced anaphylaxis during infusion with
ELELYSO or with other ERT, caution should be exercised upon
retreatment; appropriate medical support should be readily
available.
Infusion reactions (including allergic reactions), defined as a
reaction occurring within 24 hours of the infusion, were the most
commonly observed reactions in patients treated with ELELYSO in
clinical studies. The most commonly observed symptoms of
infusion reactions were headache, chest pain or discomfort,
asthenia, fatigue, urticaria, erythema, increased blood pressure,
back pain and arthralgia, and flushing. Most of these
reactions were mild and did not require treatment intervention.
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 approved product manufactured by
ProCellEx, ELELYSO™ (taliglucerase alfa), an enzyme replacement
therapy for the treatment of Gaucher disease, was approved for
marketing by the U.S. Food and Drug Administration (FDA) in May
2012, and by Israel's Ministry of Health in September
2012. Additional marketing applications for taliglucerase alfa
have been filed in other countries. Protalix is partnered with
Pfizer Inc. for worldwide development and commercialization,
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.
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" 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; a lack of sufficient
funding to finance the clinical trials; the risk that the results
of our clinical trials will not support our claims of safety or
efficacy; that taliglucerase alfa 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; risks relating to the review process of
the FDA and other foreign regulatory bodies, including the risk
that regulatory authorities may find that the data from our
clinical trials and other studies is insufficient for regulatory
approval; risks relating to delays in the FDA's, or other foreign
regulatory authorities' approval of any applications we file or
refusals to approve such filings; the risk that applicable
regulatory authorities may refuse to approve the marketing and sale
of a drug product even after acceptance of an application we file
for the drug product; and other factors described in our filings
with the Securities and Exchange Commission. Companies in the
pharmaceutical and biotechnology industries have suffered
significant setbacks in advanced or late-stage clinical trials,
even after obtaining promising earlier trial results or in
preliminary findings for such clinical trials. 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