Raptor to Support Phase 2a Clinical Trial at UCSD
NOVATO, Calif., July 16 /CNW/ -- Raptor Pharmaceuticals Corp. ("Raptor"
or the "Company") (OTC Bulletin Board: RPTP), today announced the execution of
a collaboration agreement (the "Agreement") with the University of California,
San Diego ("UCSD") to include a Phase 2a clinical trial to evaluate a
delayed-release preparation of cysteamine bitartrate ("Cysteamine") in
adolescents diagnosed with Non-Alcoholic Steatohepatitis ("NASH"). NASH is a
progressive form of liver disease that accounts for approximately 10% of newly
diagnosed cases of chronic liver disease, and ranks as one of the leading
causes of cirrhosis of the liver in the U.S.
Under the terms of the Agreement, clinical researchers from UCSD will
perform the Phase 2a study at the University's General Clinical Research
Center, and Raptor will provide funding and clinical supply of Cysteamine. In
March 2008, Raptor acquired an exclusive, worldwide license to certain
intellectual property and development rights from UCSD surrounding the use of
Cysteamine as a potential treatment for NASH.
A rapid-release form of Cysteamine is currently approved for sale by the
U.S. Food and Drug Administration ("FDA") for the treatment of nephropathic
cystinosis, a rare, genetic lysosomal storage disease. Raptor is currently
developing DR Cysteamine, a proprietary, delayed-release formulation of
Cysteamine, designed to improve bioavailability and potentially reduce side
effects associated with the presently marketed form.
Ted Daley, President of Raptor's clinical division stated, "We look
forward to working with UCSD, one of the leading research universities in the
nation, under this collaboration agreement. The University's clinical
researchers have specific expertise in treating NASH patients, and have also
worked extensively with Cysteamine. We are fortunate to work with this team to
investigate our drug candidate's potential in this widespread liver disease.
NASH is a particularly significant unmet medical need with no current
treatment options beyond diet and exercise. If this initial trial shows
promising results, we will continue the development through an appropriate
dosage form of our proprietary DR Cysteamine formulation for NASH patients."
NASH is believed to affect 2% to 5% of the U.S. population. While
commonly diagnosed in overweight adults and children with and without
insulin-resistant diabetes and abnormal serum lipid profiles, NASH can also
occur in persons of average weight. Though most people with NASH feel healthy
and show no outward signs of liver disease, NASH causes liver scarring and
potentially leads to cirrhosis and liver failure. In more severe cases, the
progressive nature of NASH may require liver transplantation in affected
Ranjan Dohil, Professor of Pediatrics at UCSD, commented, "The
opportunity for DR Cysteamine in NASH could become a potentially significant
breakthrough for these patients. Our collaboration with Raptor allows us to
run this clinical trial by leveraging the University's resources and
scientific capabilities with Raptor's instrumental support in funding,
providing drug supply, and offering formulation expertise."
About Cysteamine and DR Cysteamine
Cysteamine is approved for sale by the FDA and European Medicines Agency
to treat nephropathic cystinosis, a rare, genetic lysosomal storage disease.
DR Cysteamine is designed as an improved, enterically coated, oral formulation
of Cysteamine. Compared to the currently marketed formulation, DR Cysteamine
could allow less frequent dosing, improving compliance and potentially
reducing gastrointestinal side effects, such as nausea and vomiting.
Raptor obtained an exclusive, worldwide license to DR Cysteamine, as well
as orphan drug designation from the FDA for DR Cysteamine for the treatment of
nephropathic cystinosis, through its December 2007 merger of Encode
Pharmaceuticals. In March 2008, Raptor acquired an exclusive, worldwide
license to the intellectual property and development rights for use of
Cysteamine and DR Cysteamine for the treatment of NASH. The Company plans to
initiate a Phase 2a clinical trial in collaboration with the University of
California, San Diego ("UCSD") to evaluate Cysteamine in patients with NASH.
In May 2008, Raptor received orphan drug designation from the FDA for DR
Cysteamine for the treatment of Huntington's Disease.
About Raptor Pharmaceuticals Corp.
Raptor Pharmaceuticals Corp. ("Raptor") is a development-stage
biopharmaceutical company leveraging novel drug-targeting platforms and
reformulated therapeutics to improve treatment outcomes among patients with
liver disorders, infectious diseases, cancer, and various orphan indications.
The Company's clinical division advances internally developed and in-licensed
clinical-stage product candidates towards marketing approval and
commercialization and is currently involved in clinical trials in patients
with aldehyde dehydrogenase ("ALDH2") deficiency as part of Raptor's
Convivia(TM) program and nephropathic cystinosis as part of Raptor's DR
Cysteamine program. Raptor's preclinical division bioengineers novel drug
candidates and drug-targeting platforms derived from the human receptor-
associated protein ("RAP") and related proteins to target cancer,
neurodegenerative disorders and infectious diseases. Raptor's preclinical
programs include HepTide(TM), WntTide(TM) and NeuroTrans(TM).
For additional information, please visit www.raptorpharma.com.
FORWARD LOOKING STATEMENTS
This document contains forward-looking statements as that term is defined
in the Private Securities Litigation Reform Act of 1995. These statements
relate to future events or our future results of operation or future financial
performance, including, but not limited to the following statements: Raptor's
ability to develop DR Cysteamine to improve bioavailability, reduce side
effects and reduce dosage frequency; Raptor's ability to develop DR Cysteamine
for the treatment of Cystinosis; Raptor's ability to develop an appropriate
dosage form of DR Cysteamine for NASH patients; and Raptor's ability to
formulate and manufacture DR Cysteamine in clinical quantities to support
clinical trials. These statements are only predictions and involve known and
unknown risks, uncertainties and other factors, which may cause our actual
results to be materially different from these forward-looking statements.
Factors which may significantly change or prevent our forward looking
statements from fruition include that we may be unsuccessful in developing any
products or acquiring products; that our technology may not be validated as we
progress further and our methods may not be accepted by the scientific
community; that we are unable to retain or attract key employees whose
knowledge is essential to the development of our products; that unforeseen
scientific difficulties develop with our process; that our patents are not
sufficient to protect essential aspects of our technology; that competitors
may invent better technology; that our products may not work as well as hoped
or worse, that our products may harm recipients; and that we may not be able
to raise sufficient funds for development or working capital when we require
it. As well, our products may never develop into useful products and even if
they do, they may not be approved for sale to the public. We caution readers
not to place undue reliance on any such forward-looking statements, which
speak only as of the date they were made. Certain of these risks,
uncertainties, and other factors are described in greater detail in our
filings from time to time with the Securities and Exchange Commission (the
"SEC"), which we strongly urge you to read and consider, including our
Registration Statement on Form SB-2, as amended, that was declared effective
on July 10, 2006; our annual report on Form 10-KSB filed with the SEC on
November 14, 2007; and our Form 10-QSB filed with the SEC on July 9, 2008, all
of which are available free of charge on the SEC's web site at
http://www.sec.gov. Subsequent written and oral forward-looking statements
attributable to us or to persons acting on our behalf are expressly qualified
in their entirety by the cautionary statements set forth in our reports filed
with the SEC. We expressly disclaim any intent or obligation to update any
For more information, please contact:
The Ruth Group
Sara Ephraim (investors) / Janine McCargo (media)
(646) 536-7002 / (646) 536-7033
email@example.com / firstname.lastname@example.org
For further information:
For further information: Investors, Sara Ephraim, +1-646-536-7002,
email@example.com, or Media, Janine McCargo, +1-646-536-7033,
firstname.lastname@example.org, both of The Ruth Group, for Raptor Pharmaceuticals
Corp. Web Site: http://www.raptorpharma.com