Protalix BioTherapeutics Announces Poster Presentation on Baseline Characteristics for Fabry Disease Patients Screened in the...
May 25 2018 - 7:00AM
GlobeNewswire /Protalix BioTherapeutics, Inc. (NYSE American:PLX)
(TASE:PLX), a biopharmaceutical company focused on the development
and commercialization of recombinant therapeutic proteins expressed
through its proprietary plant cell-based expression system,
ProCellEx®, today announced that a poster presentation on the
characteristics of Fabry disease patients screened in the phase III
BALANCE clinical trial of pegunigalsidase alfa, a PEGylated alfa
galactosidase alfa enzyme, or PRX-102, for the treatment of Fabry
disease was presented at the 55th ERA-EDTA Congress (European Renal
Association – European Dialysis and Transplant Association).
Dr. David Warnock, Director of the Division of Nephrology and
Professor of Medicine and Physiology at the University of Alabama
at Birmingham, presented a poster titled “Progression of
nephropathy in Fabry patients receiving enzyme replacement therapy
(ERT); relation to anti-drug antibodies (ADA) status and
proteinuria.” The poster highlights an analysis of key
baseline characteristics including, presence of anti-drug
antibodies (ADA) toward agalsidase beta (Fabrazyme®), cross
recognition of those ADAs to pegunigalsidase alfa, annualized eGFR
slope and proteinuria at screening for the first 37 patients (27
males, 10 females) screened for the BALANCE study as of January
2018.
The results demonstrate that 15 out of 27 screened male patients
were ADA positive and, of those, all 15 had neutralizing antibodies
to Fabrazyme and 14 out of the 15 also had neutralizing activity
toward pegunigalsidase alfa as part as ex-vivo immunogenicity
testing. However, titers measured in ADA against Fabrazyme
were higher by an average of more than double than those measured
against pegunigalsidase alfa. In addition, Fabrazyme was
inhibited by an average of 83.6% leaving only 16.4% of effective
enzyme, whereby pegunigalsidase alfa was inhibited by 61.6%,
leaving 38.4%, more than double, the effective enzyme. No
ADAs were present in the 10 female patients screened.
In addition, the results generated in a recent analysis of 60
patients screened for the BALANCE study to date, including the 37
patients analyzed for the poster presentation, have been
substantially in-line with the above analysis implying that the
remaining effective enzyme in blood samples was more than double
for pegunigalsidase alfa compared to Fabrazyme.
Based on an analysis of patients’ history, the average
annualized eGFR slope for the 15 male patients that tested positive
for ADA was -7.9 (SD=6.0) mL/min/1·73m2/year demonstrating that the
Fabry disease patients screened for the BALANCE study progressively
lost kidney function, despite being treated with Fabrazyme for 1 to
12 years. Regarding proteinuria, the average urine protein
creatinine ratio was 767 (SD=546) mg/gr for male patients.
“There is an unmet medical need in patients with Fabry disease
who continue to show progressive loss of renal function despite
years of treatment with agalsidase beta,” commented Dr. Warnock.
“The progressive loss of kidney function and proteinuria seen
in patients suffering from Fabry disease may be the result of
neutralizing antibodies. Given pegunigalsidase alfa is less
inhibited by preexisting neutralizing antibodies than Fabrazyme,
coupled with its significantly longer half-life, there is the
potential for pegunigalsidase alfa to control proteinuria and/or
stabilize renal function in patients who have not had an optimal
clinical response to agalsidase beta.”
The BALANCE study is a head-to-head blinded comparison study of
pegunigalsidase alfa to agalsidase beta (Fabrazyme®) with 1 mg/kg
every two weeks, with change in eGFR as the primary end-point.
In addition, the BALANCE study also measures a wide range of
clinical end points including pain, quality of life parameters,
biomarkers and others. The study is enrolling Fabry patients
who continue to progressively lose kidney function, despite
receiving standard enzyme replacement therapy.
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: pegunigalsidase alfa, a modified version of the
recombinant human alpha-GAL-A protein for the treatment of Fabry
disease; OPRX-106, an orally delivered anti-inflammatory treatment;
alidornase alfa for the treatment of Cystic Fibrosis; and
others. Protalix has entered into an ex-United States
partnership with Chiesi Farmaceutici S.p.A. for the development and
commercialization of pegunigalsidase alfa. Protalix maintains
full rights to pegunigalsidase alfa in the United States.
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 “expect,”
“anticipate, “believe,” “estimate,” “project,” “plan,” “should” 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: risks that data generated in ex vivo studies
will not be repeated in clinical trials; 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
superiority, 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; risks related to
the ultimate purchase by Fundação Oswaldo Cruz of
alfataliglicerase pursuant to the stated purchase intentions of
the Brazilian Ministry of Health of the stated amounts,
if at all; risks related to the successful conclusion of our
negotiations with the Brazilian Ministry of
Health regarding the purchase of alfataliglicerase generally;
risks related to our commercialization efforts for
alfataliglicerase in Brazil; risks relating to the compliance
by Fundação Oswaldo Cruz with its purchase obligations
and related milestones under our supply and technology transfer
agreement; risks related to the amount and sufficiency of our cash
and cash equivalents; risks related to the amount of our future
revenues, operations and expenditures; risks related to our ability
to maintain and manage our relationship with Chiesi Farmaceutici
and any other collaborator, distributor or partner; the risk that
despite the FDA’s grant of fast track designation for
pegunigalsidase alfa for the treatment of Fabry disease, we may not
experience a faster development process, review or approval
compared to applications considered for approval under conventional
FDA procedures; risks related to the FDA’s ability to withdraw the
fast track designation at any time; risks relating to our ability
to make scheduled payments of the principal of, to pay interest on
or to refinance our outstanding notes or any other indebtedness;
our dependence on performance by third party providers of services
and supplies, including without limitation, clinical trial
services; our ability to identify suitable product candidates and
to complete preclinical studies of such product candidates; 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 press release are valid only as of the date
hereof and we disclaim any obligation to update this information,
except as may be required by law.
Investor Contact
Marcy NanusSolebury Trout 646-378-2927
mnanus@soleburytrout.com
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