Raptor Pharmaceuticals Corp. is a development-stage biotechnology company with their corporate headquarters in Novato, California. They engage in the acquisition, development, and commercialization of pharmaceutical products in the United States. As an OTCBB-traded enterprise, they dedicate their efforts to speeding the delivery of new treatment options to patients. They do this by working to improve existing therapeutics through the application of highly specialized drug targeting platforms and formulation expertise.

The Company focuses on underserved patient populations. Their DR Cysteamine is an investigational, delayed-release (DR) oral formulation of cysteamine bitartrate. This is for the potential treatment of nephropathic cystinosis (cystinosis), which is a rare lysosomal storage disease. Raptor’s DR Cysteamine formulation may require less frequent dosing and reduce gastrointestinal side effects.

The company is also developing DR Cysteamine for the potential treatment of Non-alcoholic Steatohepatitis (NASH), Huntington’s Disease, and Batten Disease. Additionally, Raptor is in clinical development of Convivia™ for the potential treatment of aldehyde dehydrogenase (ALDH2) deficiency.

Raptor Pharmaceuticals Corp. bases their preclinical programs on bioengineered novel drug candidates and drug-targeting platforms derived from the human receptor-associated protein (RAP) and related proteins. The design of these is to target cancer, neurodegenerative disorders, and infectious diseases. Their preclinical programs include HepTide™, WntTide™ and NeuroTrans™.

Last month, Raptor Pharmaceuticals announced the completion of patient enrollment in their Phase 2a open-label clinical trial to evaluate cysteamine bitartrate (cysteamine) in patients diagnosed with non-alcoholic steatohepatitis (NASH). NASH patients currently can only manage their disease through weight control, diet, and exercise. NASH is a progressive form of liver disease and is one of the leading causes of cirrhosis in the U.S. It accounts for approximately 10 percent of newly diagnosed cases of chronic liver disease.

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