Raptor Pharmaceutical Announces Publication of Phase 3 Results in the Clinical Journal of the American Society of Nephrology

Raptor Pharmaceutical Corp.

"A Randomized Controlled Crossover Trial with a Delayed Release Cysteamine Bitartrate in Nephropathic Cystinosis: Effectiveness on WBC Cystine Levels and Comparison of Safety" in the Clinical Journal of the American Society of Nephrology ("CJASN"). The CJASN is one of the most respected peer reviewed scientific journals among kidney related publications. The article describes findings from Raptor's open-label, randomized, controlled, crossover Phase 3 clinical trial, referred to as RP103-03, the top-line results of which were reported by Raptor in July 2011, and presented by the clinical investigators as a late breaking poster at the American Society of Nephrology ("ASN") – Kidney Week 2011 in Philadelphia last November. The Phase 3 clinical trial enrolled 43 patients and was powered to show that Raptor's delayed-release formulation of cysteamine bitartrate, referred to as RP103, taken every 12 hours, was non-inferior to the currently-available immediate release cysteamine bitartrate treatment, Cystagon®, taken every 6 hours, for maintenance of white blood cell ("WBC") cystine levels in patients who were already well controlled on Cystagon®. The clinical trial also showed that RP103 met its primary endpoint at a lower total daily dose than Cystagon®.