Protalix BioTherapeutics Discloses Three New Compounds in Development
20 Juni 2013 - 8:55PM
- Oral PRX-106 for immune mediated disorders -
PRX-110 for Cystic Fibrosis (CF) - PRX-107 for emphysema due to
hereditary alpha1-antitrypsin deficiency
Protalix BioTherapeutics, Inc. (NYSE:PLX) (TASE:PLX), today held an
Analyst Event in which included the disclosure of new data
regarding three new compounds in development, oral PRX-106 for
immune mediated disorders, PRX-110 for Cystic Fibrosis (CF), and
PRX-107 for emphysema from heredity alpha1-antitrypsin (AAT)
deficiency.
"Our validated plant-cell based platform, ProCellEx®, has the
capacity to generate a diverse array of protein therapeutics, each
with highly unique characteristics. With this next wave of
compounds and oral administration modalities, we are building a
strong pipeline and potentially treatment advances to patients,"
said Dr. David Aviezer, Protalix BioTherapeutics' President and
Chief Executive Officer.
PRX-106 is the Company's proprietary plant cell recombinant
Anti-TNF fusion protein being developed as an orally-administered
treatment for immune mediated disorders. In preclinical studies,
oral PRX-106 alleviated immune-mediated hepatitis and reduced
interferon gamma levels in a concanavalin A (ConA) mouse model.
Additionally, oral administration of PRX-106 alleviated
immune mediated colitis, a well established model for Crohn's
disease, promoting serum levels of anti-inflammatory IL-10 and
regulatory T-cells. The Company is conducting additional
preclinical studies for oral PRX-106 in additional attractive
indications.
PRX-110 is the Company's proprietary plant cell recombinant
human Deoxyribonuclease 1 (DNase 1) under development for the
treatment of CF, to be administered by inhalation. PRX-110
works by cleaving extracellular DNA and thinning the thick mucus
that accumulates in CF patients' lungs. In preclinical trials,
PRX-110 demonstrated improved enzyme kinetics, less sensitivity to
inhibition by actin and improved ex-vivo efficacy when compared to
Pulmozyme®, the only approved form of recombinant DNase 1
manufactured in Chinese hamster ovary (CHO) cells. The Company
held a pre-Investigational New Drug (IND) meeting with the U.S.
Food and Drug Administration (FDA) in 2012, and plans to file an
IND with the FDA following the completion of toxicology studies,
which is expected to occur by year end.
PRX-107 is the Company's proprietary plant cell recombinant
human Alpha1-antitrypsin (AAT) under development for the treatment
of emphysema due to hereditary AAT deficiency, to be administered
by inhalation. PRX-107 is a protein that works by regulating
the AAT-dependent inflammatory response in the
lungs. Currently, there is no approved recombinant form of
AAT. Plasma derived-forms of AAT are available, but are
associated with limitations, including inadequate supply, the
potential for adventitious agents and poor absorption. In
preclinical studies, PRX-107 demonstrated the ability to rescue
elastase induced lung damage in rats and as effective as a
plasma-derived reagent. The Company plans to hold a pre-IND meeting
with the FDA in the second half of 2013 to discuss next steps for
this compound.
Additional information on these compounds can be found in the
slides presented at the Company's Analyst Day on June 20,
2013. These are available under the presentation tab on the
Company's website.
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, by Israel's Ministry of
Health in September 2012, by the Brazilian National Health
Surveillance Agency (ANVISA) in March 2013, by the Mexican Federal
Commission for the Protection against Sanitary Risk (COFEPRIS) in
April 2013, 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 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 produced and
encapsulated within 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; PRX-110 for
the treatment of Cystic Fibrosis; PRX-107 for the treatment of
emphysema due to hereditary alpha1-antitrypsin deficiency; 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 studies 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 the clinical trials; the risk
that the results of our clinical trials will not support the
applicable claims of safety or efficacy, that our product
candidates 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;
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; 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; risks related to the potential infringement of a third
party's patents or other intellectual property rights; the
uncertainty of obtaining patents covering our products and
processes and in successfully enforcing our intellectual property
rights against third parties; risks of securing adequate
levels of product liability and clinical trial 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
Media Contact
Kari Watson
MacDougall Biomedical Communications
781-235-3060
kwatson@macbiocom.com