Positive efficacy data across all disease
parameters
Positive safety data with low level of antibody
formation
End of Phase II meeting with FDA scheduled before
year end
Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced
today positive interim data from the Company's phase I/II clinical
trial of 1mg/kg 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 are very pleased with the positive results from the 1mg
cohort for PRX-102," commented Mr. Moshe Manor, Protalix's
President and Chief Executive Officer. "The efficacy results for
the 1mg/kg cohort appear even more robust than those previously
announced for the 0.2mg cohort, while maintaining a favorable
safety profile with a very low level of antibody formation."
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 adult 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, with a six-month
efficacy follow up period.
This interim analysis includes 6 patients enrolled in the 1mg/kg
dose group at six months of treatment. The interim safety analysis
includes 18 patients enrolled in three dose cohorts of 0.2mg/kg,
1mg/kg and 2mg/kg.
Interim Efficacy Results
Based on an analysis of kidney biopsies with randomized blinded
scoring (n=4), PRX-102 demonstrated a reduction in renal
peritubular capillary Gb3 of 86% using a quantitative Barisoni
Lipid Inclusion Scoring System (BLISS).
Reductions of plasma Lyso-Gb3 and plasma Gb3 concentrations were
also observed. Males (n=4) demonstrated a -67.5 ng/mL and a -5.3
µg/mL change, Females (n=2) demonstrated a -9.2 ng/mL mean change
in Lyso-Gb3 and a -0.23 µg/mL mean change in plasma Gb3,
respectively.
Furthermore, all patients had stable cardiac function after only
six months, as measured by left ventricular mass (LVM), left
ventricular mass index (LVMI) and ejection fraction (EF). Stable
kidney function was also observed, as measured by estimated
glomerular filtration rate (eGFR) and urine protein.
Safety Results
The safety analysis for adverse events represents a total of 15
patient years. PRX-102 was well tolerated, with the majority of
adverse events being mild and moderate. Only one of the patients
evaluated for safety experienced hypersensitivity, and only three
patients, or approximately 19%, developed antibodies.
"The data presented from the 1mg cohort of PRX-102 continues to
be very encouraging," said Dr. Derralynn Hughes of the Lysosomal
Storage Disease Unit, Institute of Immunity and Transplantation,
Royal Free London NHS Foundation Trust, London, UK, and a principal
investigator in the PRX-102 clinical trial. "Significant
improvement in Gb3 levels with stability in renal function and
cardiac parameters were observed after a relatively short period of
time with low antibody formation."
Raphael Schiffmann, M.D., M.H.Sc., an investigator with the
Institute of Metabolic Disease, Baylor Research Institute, Dallas,
TX, stated, "The data presented from the 1mg/kg cohort of PRX-102
continues to be very promising with a significant potential for
improvement over the currently approved enzyme replacement
therapies for Fabry disease. As PRX-102 has a different chemical
structure with a different PK profile, which is probably the cause
for the very low formation of antibodies, it has the potential for
reduced immunogenicity as demonstrated in this interim report.
Renal disease represents one of the main causes of morbidity and
mortality among Fabry patients, and may not be adequately
controlled by current standard of care. As a principal investigator
in the ongoing clinical study, I am very encouraged when eGFR
levels are kept stable."
Enrollment in the phase I/II clinical trial of PRX-102 was
completed in early February 2015. All patients that completed the
trial opted to continue to receive PRX-102 in an open-label
extension study. The Company expects to report longer term data of
the first 0.2mg/kg cohort by the end of this month and to report
full top-line results from all dosing cohorts in the fourth quarter
of 2015. The Company scheduled an End of Phase II meeting with the
Food and Drug Administration before year-end to discuss the design
of the pivotal phase III trial, which the Company expects to start
in early 2016.
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 Israel's Ministry of Health, by the
Brazilian National Health Surveillance Agency (ANVISA) and by the
regulatory authorities of other countries. 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 and 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-106, an
orally-delivered antiTNF; PRX-110 for the treatment of Cystic
Fibrosis; and others.
About Baylor Research Institute
Established in 1984 in Dallas, Texas, Baylor Research Institute
(BRI) promotes and supports research to bring innovative treatments
from the laboratory workbench to the patient bedside. To achieve
this bench-to-bedside concept, BRI focuses on basic science,
clinical trials, healthcare effectiveness and quality of care
research. Today, BRI is conducting more than 930 active research
protocols with 400 research investigators, spanning more than 22
medical specialties, and has research and development projects in
areas ranging from human immunology and orphan metabolic diseases
to diabetes, cardio-vascular disease and many other unmet medical
needs.
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
Marcy Nanus
The Trout Group, LLC
646-378-2927
mnanus@troutgroup.com
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