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Raptor Pharmaceuticals Doses First Patient in Phase 2b Clinical Trial of DR Cysteamine for Cystinosis

NOVATO, Calif., June 1 /PRNewswire-FirstCall/ -- Raptor Pharmaceuticals Corp. ("Raptor" or the "Company") (OTC Bulletin Board: RPTP), today announced that it has dosed the first patient in its Phase 2b clinical trial, conducted in collaboration with the University of California, San Diego ("UCSD"), to evaluate Raptor's proprietary delayed-release cysteamine bitartrate ("DR Cysteamine") capsules in nephropathic cystinosis ("cystinosis"), a rare genetic lysosomal storage disease. Under Raptor's open Investigational New Drug application ("IND"), UCSD is performing the Raptor-sponsored trial at its General Clinical Research Center.

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The Phase 2b clinical trial will enroll up to six cystinosis patients with a history of good compliance using the currently available immediate-release form of cysteamine bitartrate, the only drug cleared for marketing by the U.S. Food and Drug Administration ("FDA") and European Medicines Agency ("EMEA") to treat cystinosis. The clinical trial will evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of DR Cysteamine in patients. Data from the study are expected in the third quarter of 2009. Raptor plans to follow the Phase 2b clinical study with a pivotal, Phase 3 clinical study in cystinosis patients anticipated to commence in the fourth quarter of 2009.

DR Cysteamine is Raptor's proprietary enteric-coated micro-bead formulation of cysteamine bitartrate contained in a gelatin capsule and designed for increased absorption, less frequent dosing and improved tolerability relative to the current standard of care. Published studies have shown that fewer than 25% of patients taking immediate-release cysteamine bitartrate are fully-compliant due to the drug's strict every-six hour dosing schedule and dose-limiting side effects, including gastrointestinal ("GI") problems such as stomach aches, nausea and vomiting.

Patrice Rioux, M.D., Ph.D., Raptor's Chief Medical Officer stated, "We are pleased that enrollment is now underway in our Phase 2b clinical trial in cystinosis. This is an underserved patient population and there is a compelling need for an improved treatment option. Many patients are unable to take adequate levels of the drug to halt the progression of their disease, which gradually destroys the organs in the body. We believe DR Cysteamine could have positive effects on the long-term health of cystinosis patients as well as the quality of life for patients, their families and caregivers."

Bruce Barshop, M.D., Ph.D., Professor of Clinical Pediatrics at UCSD and principal investigator for the cystinosis clinical trial, commented, "The initiation of this cystinosis trial offers us an opportunity to evaluate the potential for an improved treatment invented here at UCSD and further developed by Raptor. We're pleased to collaborate with Raptor in this effort to advance the standard of care in cystinosis."

About DR Cysteamine

DR Cysteamine is an enteric-coated micro-bead formulation of cysteamine bitartrate. 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 the University of California, San Diego for use of cysteamine and DR Cysteamine for the treatment of non-alcoholic steatohepatitis ("NASH"), a progressive liver disease believed to affect 2-5% of the U.S. population. In October, 2008 Raptor announced the initiation of a Phase 2a clinical study of cysteamine bitartrate in NASH patients, in collaboration with UCSD. In November, 2008 Raptor announced a collaboration with Centre Hospitalier Universitaire d'Angers, in France, to study DR Cysteamine in Huntington's Disease, a rare, genetic neurological disease.

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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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: that Raptor will be able to fully enroll and complete the clinical trial in cystinosis patients; that data will be available in the third quarter of 2009; that Raptor will start a Phase 3 clinical trial in cystinosis patients in the fourth quarter of 2009, if at all; that DR Cysteamine will increase absorption, require less frequent dosing or improve tolerability relative to the current standard of care; that DR Cysteamine will have a positive effect on cystinosis patients and increase their quality of life; and that any of the Company's clinical and preclinical drug candidates will result in approved therapeutics. 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 at raising funds to continue its development programs; 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; and that Raptor's products may not work as well as hoped or worse, that the Company's products may harm recipients. 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 April 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:

    Karl Cahill, Investor Relations
    (858) 531-6100

    The Ruth Group
    Sara Ephraim Pellegrino (investors) / Janine McCargo (media)
    (646) 536-7002 / (646) 536-7033 /

SOURCE Raptor Pharmaceuticals Corp.
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