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Raptor Pharmaceuticals Doses First Patient in Phase 2b Clinical Trial of DR Cysteamine for Cystinosis
Date:6/1/2009

mine 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 ann
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SOURCE Raptor Pharmaceuticals Corp.
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