Raptor Pharmaceutical Corp., a biopharmaceutical company, focuses on developing and commercializing life-altering therapeutics that treat debilitating and fatal diseases. Clinical Development Programs The company focuses on developing its product candidate, RP103 (a delayed and extended release formulation of cysteamine bitartrate), as a treveeatment for nephropathic cystinosis or cystinosis, a rare genetic disorder. Cystinosis patients are at risk of experiencing life-threatening metabolic disorders, including kidney failure, severe gastrointestinal dysfunction, and rickets as a result of an accumulation of the amino acid, cystine, in cells. In addition to cystinosis, the company is testing RP103 for the treatment of non-alcoholic steatohepatitis (NASH), a metabolic liver disorder and Huntington's disease (HD), a neurodegenerative disorder. The company has completed Phase 3 clinical trial designed to evaluate RP103 as a potential treatment for cystinosis; and conducting a Phase 2/3 clinical trial of RP103 designed to investigate potential mechanism of cysteamine in HD patients, as well as a Phase 2b juvenile clinical trial evaluating the safety and efficacy of RP103 as a potential treatment of NASH. The company’s three active clinical development programs utilize the same active pharmaceutical ingredient cysteamine bitartrate. Its proprietary extended and delayed-release formulation, RP103, is a capsule containing enteric coated micro-beads of cysteamine bitartrate. Other Clinical-Stage Product Candidate The company is developing Convivia, its proprietary oral formulation of 4-methylpyrazole for the potential management of acetaldehyde toxicity due to alcohol consumption by individuals with aldehyde dehydrogenase (ALDH2) deficiency, an inherited metabolic disorder. It is developing Convivia to lower systemic acetaldehyde levels and reduce symptoms associated with alcohol intake by ALDH2-deficient individuals. The company completed a Phase 2a clinical trial of Convivia taken concomitantly with alcohol, at a clinical research center in Honolulu, Hawaii. Preclinical Product Candidates The company’s preclinical platforms consist of targeted therapeutics for the treatment of multiple indications, including liver diseases, neurodegenerative diseases, and breast cancer. These preclinical programs include its receptor-associated protein platform that consists of HepTide for the treatment of primary liver cancer and other liver diseases, and NeuroTrans to deliver therapeutics across the blood-brain barrier for treatment of various neurological diseases; and its mesoderm development protein platform that consists of WntTide for the treatment of breast cancer. Agreements The company has a worldwide license to delayed-release cysteamine bitartrate from the University of California, San Diego, which is the basis for its proprietary formulation of cysteamine. The company also has a licensing agreement with Yeda Research and Development Company Limited, for patents originating from Weizmann Institute of Technology and Niigata University, related to use of transglutaminase inhibitors to treat neurological diseases. Significant Events In November 2013, Raptor Pharmaceuticals Corp. and DaVita Clinical Research have announced a collaboration to screen blood samples from patients with end-stage renal disease, or ESRD, in an effort to identify patients with unrecognized late-onset nephropathic cystinosis.
raptor pharmaceutical corp
9 Commercial Boulevard
Novato, CA 94949
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