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Raptor Pharmaceuticals Enters Collaboration Agreement With UC San Diego in Liver Disease

July 16, 2008

NOVATO, Calif., July 16 /PRNewswire-FirstCall/ — Raptor Pharmaceuticals Corp. (“Raptor” or the “Company”) (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 patients.

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 http://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 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  

Raptor Pharmaceuticals Corp.

CONTACT: Investors, Sara Ephraim, +1-646-536-7002,sephraim@theruthgroup.com, or Media, Janine McCargo, +1-646-536-7033,jmccargo@theruthgroup.com, both of The Ruth Group, for Raptor PharmaceuticalsCorp.

Web site: http://www.raptorpharma.com/




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