Raptor Pharmaceuticals to Collaborate with Centre Hospitalier Universitaire d'Angers for Phase II Clinical Trial in Huntington...(understanding of cysteamine's effects in...),Health

The primary endpoint of the Phase II trial will be evaluated using the Unified Hungtington's Disease Rating Scale ("UHDRS"). The secondary endpoint will measure brain-derived neurotrophic factor ("BDNF") levels, known to be deficient in Huntington's patients. A preclinical study published by Drs. Sandrine Humbert and Frederic Saudou from the Curie Institute demonstrated a potential mechanism for cysteamine in an in vivo preclinical Huntington's model that showed increased brain and blood levels of BDNF. The Curie Institute will participate in the clinical trial by determining the BDNF levels in the patients.

About Huntington's Disease

Huntington's is a rare, progressive, and hereditary neurological disease that often leads to death within 15 to 20 years after diagnosis. The disease is thought to affect as many as 20,000 patients in the U.S. There is no currently available drug that targets the defective gene believed to cause Huntington's, which results in the degeneration of certain nerve cells in the brain. The disease is characterized by uncontrollable movements and mood swings or depression, followed by dementia.

About Cysteamine and DR Cysteamine

Cysteamine is approved for sale by the FDA and European Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), a rare, genetic lysosomal storage disease. DR Cysteamine is designed as an enterically coated, proprietary oral formulation of cysteamine to potentially reduce dosing frequency and gastrointestinal side effects.

Raptor obtained an exclusive, worldwide license from the University of California, San Diego ("UCSD") for the development of cysteamine and DR Cysteamine for a number of potential indications including Huntington's, cystinosis, non-alcoholic steatohepatitis ("NASH") and Batten Disease. Raptor is
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