Protalix BioTherapeutics to Conduct Phase III Clinical Trial for PRX-102 for the Treatment of Fabry Disease Following a Succe...
16 November 2015 - 11:00PM
Clear Path for Biologics License Application
(BLA) Submission
One Short-Term Safety and Efficacy Study Required
to Support Full Approval
In Parallel, Protalix to Conduct Phase III
Head-to-Head Superiority Trial Comparing PRX-102 Versus
Fabrazyme®
Protalix BioTherapeutics, Inc. (NYSE MKT:PLX) (TASE:PLX), announced
today that it recently held an End-of-Phase II meeting with the
U.S. Food and Drug Administration (FDA) to discuss the Company's
proposed BLA plan for PRX-102 for the treatment of Fabry disease.
Official FDA meeting minutes indicate the FDA's acceptance of the
Company's path forward for a phase III clinical trial to support a
full BLA approval.
The phase III clinical trial will be a randomized, multi-center,
placebo-controlled, safety and efficacy study in treatment-naïve
Fabry patients evaluating the 1 mg/kg dose of PRX-102. The
Company anticipates a small sample size of patients will be needed
to achieve statistical significance with a study duration of
approximately six months. The primary endpoint will be
Gastrointestinal Symptoms, with key secondary endpoints including
renal function.
In the official FDA meeting minutes, the FDA noted that the
Company reported interim analysis results from its phase I/II
clinical trial of PRX-102 that preliminarily show a favorable trend
in the severity and frequency of abdominal pain and frequency of
diarrhea after six months of treatment with PRX-102. According
to the FDA, during a recent ERT (enzyme replacement therapy)
shortage, patients who reduced or discontinued ERT dosing developed
worsening of GI signs and symptoms within a few weeks to
months.
In addition to the phase III clinical trial described above, the
Company and the FDA also agreed to a phase III head-to-head
superiority trial comparing PRX-102 versus Fabrazyme, which the
Company plans to commence in early 2016. The primary endpoint
for this head-to-head trial will be an improvement in
eGFR. The trial will enroll patients who are currently treated
with Fabrazyme; such patients will be treated with 1mg/kg of
PRX-102 for a two-year period. Interim results from this
head-to-head trial will also provide supportive safety data for the
BLA submission.
"We are very pleased with the outcome of the FDA meeting and
appreciate the valuable guidance the agency has provided us
regarding the phase III clinical program required to support a full
BLA approval," said Moshe Manor, Protalix's President and Chief
Executive Officer. "We are in a strong financial position and
believe we are well capitalized to run both phase III trials
through an anticipated BLA filling."
Guidance from the official FDA minutes, suggests no additional
non-clinical studies are required to support a BLA for PRX-102. The
Company plans to submit a request for a Special Protocol Assessment
(SPA) to the FDA later this year, and to commence both
Phase III trials in early 2016. The Company will provide more
detailed information on the design of the Phase III clinical trials
after completion of the SPA process.
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(R). 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
anti-inflammatory treatment; 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: slower than expected rates of
patient recruitment; unforeseen safety issues; determination of
dosing issues; lack of effectiveness during clinical trials;
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
646-378-2952
mnanus@troutgroup.com
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