Significantly Improved PK Characteristics
Result in:
Higher Active Enzyme Quantities, Induced
Immune Tolerance
Meaningful Clinical Benefit Demonstrated
Across All Key Disease Parameters
Reversal in eGFR Slope Achieved
Suggesting Improvement in Kidney Function
Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced
today positive long term data from the 0.2mg, or lowest dose, of
the Company's phase I/II dose ranging clinical trial of PRX-102 for
the treatment of Fabry disease. PRX-102 is a recombinant plant
cell expressed, chemically modified version of the human
alpha-Galactosidase-A enzyme.
"We continue to see excellent results from PRX-102 and are very
pleased with what we see in our lowest dose of the study," said
Moshe Manor, Protalix's President and Chief Executive
officer. "Now that we have viewed results from 12 months of
the study, we believe that PRX-102 has a significant potential to
improve the condition of Fabry patients, particularly compared to
the currently available enzyme replacement therapies."
The phase I/II clinical trial of PRX-102 for the treatment of
Fabry disease is an open-label, dose-ranging study treating up to
18 naïve male and female patients. The three dose cohorts
include dosage groups of 0.2 mg/kg, 1mg/kg and 2mg/kg with
intravenous infusions of PRX-102 every two weeks. The data
presented herein is from patients in the 0.2mg/kg dose group
following 12 months of treatment.
"The results to date from the 12 month interim report of the
0.2mg/kg of the Phase I/II clinical trial are promising on both
efficacy and safety," commented Prof. David G Warnock, Professor of
Nephrology at UAB Hospital, Birmingham, Alabama, USA, and a member
of the Medical Advisory Board for PRX-102. "I am looking
forward for the further development of PRX-102 that could
potentially be beneficial to the Fabry patients' community."
The Company has scheduled an end of phase II meeting with the
U.S. Food and Drug Administration to be held during the month of
November to discuss the design of the pivotal phase III trial which
the Company expects to start in early 2016.
Clinical Data on Kidney Functions
Among the leading causes for death of Fabry patients include
renal failures. On average, patients that participated in the
0.2mg/kg cohort of the PRX-102 trial exhibited stability in kidney
function with favorable trends shown, as measured by estimated
Glomerular filtration rate (eGFR).
In a typical Fabry patient, the eGFR deteriorates over time,
showing that kidney function is worsening. After dosing with
0.2mg/kg of PRX-102 for 12 months, a majority of the patients (4/6)
experienced a stabilization or improvement in kidney function; a
reversal of the decline shown by annualized eGFR slope was
observed.
Detailed Clinical Data on Other Biomarkers and
Scales
Lyso-Gb3 is a sensitive and reliable biomarker of Fabry disease.
It is used as a biomarker as it dramatically increases and
accumulates in the plasma of Fabry patients. Throughout the
study, continuous and durable reduction of up to 61.8% of plasma
lyso-Gb3 levels from base line was observed in patients in the
0.2mg/kg cohort. This represents a meaningful positive outcome
of PRX-102 treatment.
In addition, Fabry patients of the 0.2mg/kg cohort of the
PRX-102 trial showed a continuous reduction and durable improvement
in Mainz Severity Score Index (MSSI), a tool for disease status
evaluation of a variety of signs and symptoms of Fabry disease
including cardiovascular, renal and neurological.
Pharmacokinetics
PRX-102 unique and enhanced PK properties resulted in long
half-life, high AUC and measured levels of enzyme found throughout
the entire two weeks infusion intervals in all patients of the
0.2mg/kg cohort of the trial, potentially contributing to an immune
tolerance phenomenon. This resulted in low incidence of antibody
formation with low titers in general, and moreover, in antibody
positive patients it resulted in a transient and reversible shift
of overall drug availability. Mean values for Cmax, and AUC were
found to have briefly shifted at three and six months in antibody
positive patients, where at 12 months, PK parameters returned to
the high AUC levels observed at baseline, demonstrating that the
antibody presence and its impact was transient, leading to full
active dose availability for effective treatment.
In addition, PRX-102 continues to be well tolerated with a
favorable safety profile, with the majority of adverse events being
mild and moderate in severity with a very low rate of antibody
formation.
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 product manufactured by ProCellEx,
taliglucerase alfa, was approved for marketing by the U.S.
Food and Drug Administration (FDA) in May 2012 and,
subsequently, by the regulatory authorities of other countries.
Protalix has licensed to Pfizer Inc. the worldwide development and
commercialization rights for taliglucerase alfa,
excluding Brazil, where Protalix retains full
rights. Protalix's development pipeline 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-112, an orally-delivered glucocerebrosidase enzyme that is
produced and encapsulated within carrot cells, for the treatment of
Gaucher disease; PRX-106, an orally-delivered treatment for the
treatment of Inflammatory Bowel Disease; PRX-110 for the treatment
of Cystic Fibrosis; 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 preclinical 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 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 FDA or other health
regulatory authorities, and other risks relating to the review
process; 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 other
necessary 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
Alan Lada
The Trout Group, LLC
646-378-2952
alada@troutgroup.com
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