Business Description
Raptor Pharmaceutical Corp was initially incorporated in Nevada on July 29, 1997 as Axonyx Inc. In October 2006, Axonyx Inc. and its then wholly-owned subsidiary completed a reverse merger, business combination with TorreyPines Therapeutics, Inc., reincorporated in Delaware and changed the corporate name to 'TorreyPines Therapeutics, Inc.'. On September 28, 2009, the Company name was again changed from TorreyPines Therapeutics, Inc., to Raptor Pharmaceutical Corp. The Company researches, produces, and delivers medicines that improve life for patients with severe, rare disorders. Its product portfolio includes both candidates from its proprietary drug targeting platforms and in-licensed and acquired product candidates. Its current pipeline includes three clinical development programs, which it is actively developing. The Company also has two other clinical-stage product candidates, one of which it is seeking additional business development partners in Asia, but are not internally developing, and it has three pre-clinical product candidates for which it is seeking development partners. The Company's three active clinical development programs are based on an existing therapeutic that it is reformulating for potential improvement in safety and/or efficacy and for application in new disease indications. These clinical development programs include DR Cysteamine, or RP103, for the potential treatment of nephropathic cystinosis, or cystinosis, a rare genetic disorder; and RP103 for the potential treatment of Huntington's Disease, or HD, an inherited neurodegenerative disorder; RP103 is its proprietary delayed-release formulation of cysteamine bitartrate microbeads in capsules, which may require less frequent dosing and reduce gastro-intestinal side effects compared to the current standard of care; RP104, for the potential treatment of non-alcoholic steatohepatitis, or NASH, a metabolic disorder of the liver. RP104 is proprietary delayed-release formulation of cysteamine bitartrate in tablets. Its other clinical-stage product candidates include: Convivia for the potential management of acetaldehyde toxicity due to alcohol consumption by individuals with aldehyde dehydrogenase, or ALDH2 deficiency, an inherited metabolic disorder; and Tezampanel, a glutamate receptor antagonist as a potential anti-platelet agent, and NGX 426. Its preclinical platforms consist of targeted therapeutics for the potential treatment of multiple indications, including liver diseases, neurodegenerative diseases and breast cancer. It is seeking development partners for these programs. These preclinical programs include its receptor-associated protein, or RAP, platform consists of: HepTide for the potential treatment of primary liver cancer and other liver diseases; and NeuroTrans to potentially deliver therapeutics across the blood-brain barrier for treatment of a variety of neurological diseases. Its mesoderm development protein, or Mesd, platform consists of Wnt
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