Raptor Pharmaceuticals Announces Phase 2a Clinical Trial Initiation in Non-Alcoholic Steatohepatitis (NASH)



    Commences Patient Dosing of Delayed-ReleaseCysteamine in Collaboration
with UC, San Diego
    

    
    NOVATO, Calif., Oct. 20 /PRNewswire-FirstCall/ -- Raptor Pharmaceuticals
Corp. ("Raptor" or the "Company") (OTC Bulletin Board:   RPTP), today announced
the initiation of patient dosing in a Phase 2a clinical trial, in
collaboration with the University of California, San Diego ("UCSD"), to
evaluate cysteamine bitartrate ("cysteamine") in adolescents diagnosed with
Non-alcoholic Steatohepatitis ("NASH").  NASH, a progressive form of liver
disease that accounts for approximately 10% of newly diagnosed cases of
chronic liver disease, is one of the leading causes of cirrhosis in the U.S.
    

    (Logo:  http://www.newscom.com/cgi-bin/prnh/20071022/NYM074LOGO )

    
    Under a previously announced collaboration agreement between Raptor and
UCSD to evaluate the safety and potential efficacy of cysteamine in NASH
patients, UCSD is performing the Phase 2a open-label clinical study at UCSD's
General Clinical Research Center while Raptor is providing funding and
clinical supplies of cysteamine. The trial is expected to enroll 12 adolescent
NASH patients who have been managing the disease through diet and exercise.
Positive data could provide the rationale for later stage clinical studies of
cysteamine in NASH patients.
    

    
    Cysteamine is currently cleared for market by the U.S. Food and Drug
Administration ("FDA") and European Medicines Agency ("EMEA") to treat
nephropathic cystinosis ("cystinosis"), a rare and genetic lysosomal storage
disease. Raptor is developing cysteamine and a delayed release form of
cysteamine ("DR Cysteamine") for a number of new potential therapeutic
indications, including NASH, under licenses acquired from UCSD. Cysteamine has
demonstrated potential efficacy in preclinical and clinical studies in NASH,
Huntington's Disease, Batten Disease and other indications.
    

    
    Ted Daley, President of Raptor's clinical division stated, "We are
pleased that dosing is now underway in the Phase 2a clinical trial in NASH, a
disease that may have a severe impact on the long-term health of a large
number of people.  Cysteamine represents an exciting potential treatment for
NASH patients currently limited to diet and exercise for disease management.
The NASH program is part of our strategy to leverage cysteamine's known safety
profile and potential efficacy in several indications where it has already
shown promise in clinical and preclinical studies."
    

    
    Joel Lavine, M.D., Ph.D., pediatric gastroenterologist at UCSD and
principal investigator for the NASH study, commented, "The initiation of
patient dosing in the NASH trial offers the opportunity to evaluate a
potential new treatment for this disorder. We're pleased to collaborate with
Raptor for this study as we work to find a treatment for NASH."
    

    
    NASH is believed to affect 2% to 5% of the U.S. population. While
commonly diagnosed in insulin-resistant obese adults with diabetes and
abnormal serum lipid profiles, NASH can also occur in children, elderly and
non-diabetic persons of average weight. Though most people with NASH feel
healthy and show no outward signs of liver disease, NASH causes decreased
liver function and potentially leads to cirrhosis, liver failure and end-stage
liver disease. In more severe cases, the progressive nature of NASH may
require liver transplantation in affected patients.
    

    About Cysteamine and DR Cysteamine
    
    Cysteamine is cleared for market by the FDA and EMEA to treat cystinosis,
a rare and genetic lysosomal storage disease. The drug's potential to
accumulate in the liver may create a targeted treatment effect for patients
with NASH. DR Cysteamine is designed as an enterically coated, oral
formulation of cysteamine.
    

    
    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 acquisition of Encode
Pharmaceuticals. In March 2008, Raptor acquired an exclusive worldwide license
to intellectual property from UCSD for the use of cysteamine and DR Cysteamine
for the treatment of NASH.
    

    About Raptor Pharmaceuticals Corp.

    
    Raptor Pharmaceuticals Corp. ("Raptor") is a publicly traded
biotechnology company dedicated to speeding the delivery of new treatment
options to patients by enhancing existing therapeutics through the application
of highly specialized drug targeting platforms and formulation expertise.
Raptor focuses on underserved patient populations where it can have the
greatest impact.  Raptor is involved in clinical development of its
proprietary Delayed-Release Cysteamine ("DR Cysteamine") product candidate in
nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's
Disease and Batten Disease, and its Convivia(TM) product candidate in aldehyde
dehydrogenase ("ALDH2") deficiency.  In its preclinical programs Raptor
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 for the treatment of NASH, cystinosis and
other indications; Raptor's ability to formulate and manufacture DR Cysteamine
in clinical quantities to support clinical trials; and cysteamine's ability to
benefit NASH patients.  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 S-1 that was declared effective on August 7,
2008; 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 forward-looking
statements.
    

    For more information, please contact:
    

    
    The Ruth Group
    Sara Ephraim (investors)
    (646) 536-7002
    sephraim@theruthgroup.com
    

    
    Janine McCargo (media)
    (646) 536-7033
    jmccargo@theruthgroup.com



    




For further information:

For further information: Sara Ephraim (investors), +1-646-536-7002,
sephraim@theruthgroup.com, or Janine McCargo (media), +1-646-536-7033,
jmccargo@theruthgroup.com Web Site: http://www.raptorpharma.com

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