Raptor Pharmaceutical Reports Positive Interim Phase 2a Clinical Data in Non-Alcoholic Steatohepatitis (NASH)

NOVATO, California, October 12 /PRNewswire/ -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq: RPTPD), announced positive findings from the completed treatment phase of its open-label Phase 2a clinical trial of delayed-release cysteamine bitartrate ("DR Cysteamine") in adolescent patients with non-alcoholic steatohepatitis ("NASH"), a progressive form of liver disease believed to affect 2% to 5% of the U.S. population. At the completion of the initial six-month treatment phase, the study achieved the primary endpoint: mean blood levels of alanine aminotransferase ("ALT"), a common biomarker for NASH, were reduced by over 50%. Additionally, over half of the study participants had achieved normalized ALT levels by the end of the treatment phase.

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There are no currently approved drug therapies for NASH, and patients are limited to lifestyle changes such as diet, exercise and weight reduction to manage the disease. DR Cysteamine represents an important potential treatment option for patients with NASH. Although NASH is most common in insulin-resistant obese adults with diabetes and abnormal serum lipid profiles, its prevalence is increasing among juveniles as obesity rates rise within this patient population. Although most patients are asymptomatic and feel healthy, NASH causes decreased liver function and can lead to cirrhosis, liver failure and end-stage liver disease.

Under a collaboration agreement between Raptor and the University of California, San Diego ("UC San Diego"), the open-label Phase 2a trial of a prototype formulation of DR Cysteamine is being conducted at UC San Diego's General Clinical Research Center and entails six months of treatment followed by a six-month post-treatment monitoring period. Eligible patients with baseline ALT and aspartate aminotransferase ("AST") measurements at least twice that of normal levels were enrolled to receive twice-daily, escalating oral doses of up to 1,000 mg of DR Cysteamine. The trial currently has enrolled eleven NASH patients between 11-18 years old. No major adverse events were reported during the six-month treatment phase. Trial subjects continue to be monitored during the six-month post-treatment period currently underway. Full results are being submitted for peer review by Raptor and UC San Diego, and are expected to be presented in 2010.

Joel Lavine, M.D., Ph.D., pediatric gastroenterologist at UC San Diego and principal investigator for the NASH study, stated, "We were encouraged by the results of this study. The degree of ALT and AST reductions are indicative of likely improvements in severity of fatty liver damage. The trial results are consistent with ALT and AST reductions normally seen in patients that achieve at least 10% weight loss, even though study participants did not show a significant change in body mass index. DR Cysteamine appears to be a promising candidate for NASH and we look forward to further analyzing these patients during the post-treatment phase."

Raptor's chief medical officer, Patrice Rioux, M.D., Ph.D., said, "These interim results have established proof-of-concept and support further clinical development of DR Cysteamine in NASH. This is an area of significant unmet need, especially with growing numbers of obese children diagnosed with the disorder. While the clinical hurdle is usually high for studies in children and adolescents, we are satisfied with the long-term safety demonstrated in this age group by the currently-marketed immediate-release cysteamine bitartrate formulation. This safety track record, coupled with our interim Phase 2a efficacy data, gives us a great sense of encouragement as we advance DR Cysteamine through the clinic."

Under a license with UC San Diego, Raptor is developing DR Cysteamine for cystinosis, NASH and other potential therapeutic indications. Cysteamine is known to be a scavenger of reactive oxygen species and potent antioxidant, most likely through its ability to increase intracellular glutathione levels. Cysteamine has also demonstrated potential efficacy in preclinical and clinical studies in Huntington's Disease, Batten Disease and other indications.

About Raptor Pharmaceutical Corp.

Raptor Pharmaceutical Corp. (Nasdaq: RPTPD) ("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 designed to potentially treat nephropathic cystinosis, non-alcoholic steatohepatitis ("NASH"), Huntington's Disease ("HD"), aldehyde dehydrogenase ("ALDH2") deficiency, and a non-opioid solution designed to potentially treat chronic pain and thrombotic disorder.

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, DR 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 DR 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 the joint proxy statement/prospectus on Form S-4 filed with the SEC on August 19, 2009; Raptor's annual report on Form 10-K filed with the SEC on March 27, 2009; Raptor's quarterly report on Form 10-Q filed with the SEC on August 11, 2009; Raptor's wholly-owned subsidiary's, Raptor Pharmaceuticals Corp. ("RPC") Registration Statement on Form S-1, as amended, that was declared effective on August 7, 2008; RPC's 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 RPC's quarterly report on Form 10-Q filed with the SEC on July 15, 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 Raptor's reports filed with the SEC. Raptor expressly disclaims any intent or obligation to update any forward-looking statements.

    
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