The European Patent Office (EPO) has issued a Notice of Allowance for a key patent to Raptor Pharmaceutical Corp. covering the use of enteric-coated, delayed-release (DR) oral formulations of cysteamine bitartrate, including Raptor's proprietary microbead formulation, RP103, as well as other formulations of cystamine and cysteamine.

The patent titled as  "Enterically Coated Cystamine, Cysteamine and Derivatives Thereof" issued on March 14, 2012 covering the use of DR cysteamine for administration to patients for the potential treatment of cystinosis, including nephropathic cystinosisas. The issued patent is for 20 years and to expire on January 26, 2027

Raptor holds exclusive, worldwide licenses to this and other related patent applications, which are owned by the Regents of the University of California, and are based on work performed at the University of California, San Diego (UCSD). In 2011, counterpart patents were granted in the US directed to DR cysteamine compositions, including RP103, and methods of use for any indication, including nephropathic cystinosis.

"Having submitted a Marketing Authorization Application for RP103 for the potential treatment of nephropathic cystinosis to the European Medicines Agency, this notice of allowance gives the Company key additional IP protection in Europe," commented Ted Daley, president of Raptor.

Patent application 07 762 690.1 covers the use of a composition of cysteamine or cystamine, regardless of the specific formulation, that provides increased delivery to the small intestine with pharmacokinetic benefits that allow for twice daily dosing in the potential treatment of cystinosis, including nephropathic cystinosis. Raptor believes that its proprietary formulation of cysteamine may provide significant therapeutic and compliance advantages compared to the presently marketed, immediate-release cysteamine bitartrate for the potential treatment of nephropathic cystinosis. In July 2011, Raptor announced that its phase 3 clinical trial of RP103 (Cysteamine Bitartrate Delayed-release capsules) met the sole primary endpoint and there were no unexpected serious safety concerns attributable to RP103 experienced by patients in the trial. The trial was conducted at three clinical sites in the U.S. and five clinical sites in Europe. In March, 2012 Raptor filed a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA), as well as a New Drug Application (NDA) with the US Food and Drug Administration (FDA), for RP103 for the potential treatment of nephropathic cystinosis.

Nephropathic cystinosis, an orphan disease, is estimated to effect a population of 2,000 patients worldwide, including 500 patients in the US and 800 patients in Europe. Cystinosis patients have inherited a defective cystine transporter gene, which results in body-wide cellular toxicity resulting from the abnormal buildup of the amino acid cystine in the lysosomes. Cystinosis is usually diagnosed in the first year of life and requires lifelong therapy. Cystine crystals accumulate in various tissues and organs, including the kidneys, brain, liver, thyroid, pancreas, muscles and eyes. Left untreated, the disease is fatal by the first decade of life. RP103 reduces cellular toxicity by continuously removing cystine from the lysosome.

RP103 is Raptor's proprietary delayed and extended release oral medication designed to potentially treat the underlying metabolic cause of cystinosis. RP103 is an enteric coated, microbead formulation of cysteamine bitartrate that has been formulated to be sprinkled onto food for administration to patients too young to take oral capsules. Raptor has been granted orphan product designation for RP103 by the EMA and FDA.

In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development of RP103 for nephropathic cystinosis and for cysteamine for other potential indications including Huntington's Disease, currently in a Phase 2/3 clinical trial in France, and non-alcoholic steatohepatitis ("NASH") currently in a Phase 2b clinical trial in the US.

Raptor Pharmaceutical Corp. seeks to research, produce, and deliver medicines that improve life for patients with severe, rare disorders. Raptor currently has product candidates in clinical development designed to potentially treat nephropathic cystinosis, non-alcoholic Steatohepatitis (NASH), Huntington's Disease (HD), aldehyde dehydrogenase deficiency (ALDH2), and thrombotic disorder.