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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.

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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.

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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 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 /

SOURCE Raptor Pharmaceuticals Corp.
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