Raptor Pharmaceutical Obtains Tissue Fibrotic Disease Intellectual Property Related To Cysteamine

Daryl Okamura, M.D., Principal Investigator at SCRI and Assistant Professor of Pediatrics at the University of Washington School of Medicine, said, "Kidney dysfunction is a recognized risk factor for poor outcome in a variety of disease states, yet there are few therapies to stop the relentless progression of CKD and alleviate the ongoing oxidative stress within the kidney. We believe that our preclinical results support further development with cysteamine for this indication. We hope to see the study of this potential cysteamine treatment move into a clinical trial."

About Chronic Kidney Disease

CKD affects approximately 26 million Americans and is characterized by the progressive loss of renal function over a protracted period of time. If the progression of CKD is not halted, CKD can develop into end stage renal disease, or chronic renal failure where the kidney no longer functions and the patient requires dialysis or a kidney transplant. There is no cure for CKD. The goals of treatment are to slow disease progression, treat the underlying causes, treat complications of the disease and when necessary, replace lost kidney function. The most common causes of CKD are diabetes mellitus, hypertension and glomerulonephritis. CKD is also caused by genetic disorders, including nephropathic cystinosis.

About Cysteamine and RP103

RP103 is Raptor's proprietary delayed release oral medication currently being investigated in several indications. RP103 is an enteric coated, microbead formulation of cysteamine bitartrate. In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development of RP103 and other forms of cysteamine for the potential treatment of: Huntington's Disease, currently in a Phase 2/3 clinical trial in France; non-alcoholic steatohepatitis ("NASH"), currently in a Phase 2b clinical trial in the U.S.; and for the development of RP103 for the potential treatment of nephropathic cystinosis, which Raptor has recently filed for marketing approval in the U.S. and E.U. The U.S. Food and Drug Administration ("FDA") has accepted for filing Raptor's New Drug Application ("NDA") for RP103 for the potential treatment of nephropathic cystinosis and assigned the user fee goal date of January 30, 2013. Raptor's E.U. marketing application of RP103 for the potential treatment of nephropathic cystinosis is under review by the EMA, and Raptor expects a decision in the first half of calendar 2013. Raptor has licensed issued patents related to the potential treatment of Huntington's Disease and other neurodegenerative diseases with cysteamine and related compounds from Niigata University and Weizmann Institute and patent applications for the use of cysteamine and related compounds for the potential treatment of Parkinson's Disease from Laval University, for the use of cysteamine and related compounds for the potential treatment of malaria and other parasitic diseases from McGill University and for the potential treatment of tissue fibrosis from the Seattle Children's Research Institute. Raptor has been granted orphan product designation for RP103 for the potential treatment of nephropathic cystinosis by the EMA and the FDA, and for the potential treatment of Huntington's Disease by the FDA.

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