Raptor Pharmaceuticals Announces Completion of Patient Enrollment in Phase 2a Clinical Trial in Non-Alcoholic Steatohepatitis (NASH)



    
    Study Evaluating Cysteamine in Collaboration with UC San Diego

    
    NOVATO, Calif., Feb. 5 /PRNewswire-FirstCall/ -- Raptor Pharmaceuticals
Corp. ("Raptor" or the "Company") (OTC Bulletin Board:   RPTP), today announced
the completion of patient enrollment in its Phase 2a open-label clinical trial
to evaluate cysteamine bitartrate ("cysteamine") in patients  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.
NASH patients are currently limited to managing their disease through weight
control, diet and exercise.
    

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

    
    NASH is believed to affect 2% to 5% of the U.S. population. While
commonly diagnosed in insulin-resistant obese adults, 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.
    

    
    Under a collaboration agreement between the University of California, San
Diego ("UC San Diego") and Raptor, UC San Diego is conducting the Phase 2a
clinical trial at UC San Diego's General Clinical Research Center. In the
six-month, open-label clinical trial, UC San Diego enrolled a total of 12
adolescent NASH patients receiving a twice daily oral dose of cysteamine and
will measure possible reduction of blood alanine aminotransferase ("ALT") and
aspartate aminotransferase ("AST") levels as a biomarker of cysteamine's
efficacy for potentially treating NASH. Positive Phase 2a data could lead to
later stage clinical trials of cysteamine in NASH patients.
    

    
    Cysteamine, currently cleared for market by the U.S. Food and Drug
Administration ("FDA") and European Medicines Agency ("EMEA") to treat
nephropathic cystinosis ("cystinosis"), has demonstrated potential efficacy in
preclinical and clinical studies in NASH, Huntington's Disease, Batten Disease
and other indications. Under a license with UC San Diego, Raptor is developing
cysteamine and a delayed-release form of cysteamine ("DR Cysteamine") for NASH
and a number of new potential therapeutic indications. Preliminary data
suggests that cysteamine is readily taken up by the liver, and is a precursor
of the potent liver anti-oxidant glutathione ("GSH"). Raptor's UC San Diego
collaborators believe that increasing GSH has the potential to reverse
NASH-related liver damage.
    

    
    Ted Daley, President of Raptor's clinical division stated, "Raptor's
strategy is to leverage cysteamine's known safety profile for the treatment of
cystinosis and potential efficacy in several other indications. In this case,
cysteamine represents an exciting potential treatment for NASH, a disease that
may have a severe impact on the long-term health of a growing worldwide
patient population among both adults and adolescents. Positive data from the
Phase 2a NASH trial may enable us to advance to later stage clinical trials in
NASH and may provide further insight on how to maximize the potential value of
cysteamine for NASH and other potential indications."
    

    
    Joel Lavine, M.D., Ph.D., pediatric gastroenterologist at UC San Diego
and principal investigator for the NASH study, commented, "We are encouraged
that the Phase 2a trial has reached full enrollment and look forward to
completing patient dosing and evaluating cysteamine's clinical results in
adolescents with NASH. There is currently no FDA-approved, effective treatment
option for their disease. UC San Diego and Raptor understand the need to
deliver a treatment solution that augments disease management through diet and
exercise and we are dedicated to working together to find a solution for these
patients."
    

    About Raptor Pharmaceuticals Corp.

    
    Raptor Pharmaceuticals Corp. ("Raptor") is dedicated to speeding the
delivery of new treatment options to patients by working to improve 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 potential impact. Raptor currently
has product candidates in clinical development to treat nephropathic
cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease
("HD"), and aldehyde dehydrogenase ("ALDH2") deficiency.
    

    
    Raptor's preclinical programs are based upon bioengineered novel drug
candidates and drug-targeting platforms derived from the human
receptor-associated protein ("RAP") and related proteins that are designed to
target cancer, neurodegenerative disorders and infectious diseases.
    
    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
and UC San Diego's ability to complete the clinical trial in NASH patients,
cysteamine's ability to treat NASH, ALT and AST as a biomarker to determine
the efficacy of a treatment for NASH, Raptor's ability to further develop
cysteamine in NASH and other indications. These statements are only
predictions and involve known and unknown risks, uncertainties and other
factors, which may cause the Company's actual results to be materially
different from these forward-looking statements. Factors which may
significantly change or prevent the Company's forward looking statements from
fruition include that Raptor may be unsuccessful in developing any products or
acquiring products; that Raptor's technology may not be validated as it
progresses further and its methods may not be accepted by the scientific
community; that Raptor is unable to retain or attract key employees whose
knowledge is essential to the development of its products; that unforeseen
scientific difficulties develop with the Company's process; that Raptor's
patents are not sufficient to protect essential aspects of its technology;
that competitors may invent better technology; that Raptor's products may not
work as well as hoped or worse, that the Company's products may harm
recipients; and that Raptor may not be able to raise sufficient funds for
development or working capital. As well, Raptor's products may never develop
into useful products and even if they do, they may not be approved for sale to
the public. Raptor cautions 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 the Company's filings from time to time with the Securities
and Exchange Commission (the "SEC"), which Raptor strongly urges you to read
and consider, including its Registration Statement on Form S-1, as amended,
that was declared effective on August 7, 2008; its annual report on Form 10-K
filed with the SEC on October 30, 2008, as amended by that Form 10-K/A filed
with the SEC on December 23, 2008; and its Form 10-Q filed with the SEC on
January 13, 2009, 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 Raptor or to persons acting on its behalf are
expressly qualified in their entirety by the cautionary statements set forth
in the Company's reports filed with the SEC. Raptor expressly disclaims any
intent or obligation to update any forward-looking statements.
    

    

    
    For more information, please contact:
    

    
    The Ruth Group
    Sara Ephraim (investors) / Janine McCargo (media)
    (646) 536-7002 / (646) 536-7033
    sephraim@theruthgroup.com / 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, both of The Ruth Group for Raptor Pharmaceuticals
Corp. Web Site: http://www.raptorpharma.com/

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RAPTOR PHARMACEUTICAL CORP.

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