Today, Raptor Pharmaceuticals announced it has secured exclusive technology rights to intellectual property regarding compounds related to the potential treatment of Parkinson’s Disease. Université Laval, located in Quebec, Canada, entered into an agreement with Raptor to license the use of crysteamine and related compounds to potentially modify the progression of Parkinson’s Disease.
Researchers at Laval reported that administration of cystamine (an oxidized form of cysteamine) in an animal model of Parkinson’s Disease showed signs of preventing neuron loss and rescuing neurons undergoing a degenerative process. Signs of restoration and partial reversal of behavioral impairments were also observed. Raptor Pharmaceuticals’ proprietary delayed release oral medication, RP103, is currently being investigated in several indications. RP103 is an enteric coated, microbead formulation of cysteamine bitartrate.
Dr. Patrice P. Rioux, Raptor’s Chief Medical Officer, as quoted in the company press release, stated, “The agreement with Laval extends our existing portfolio in neurodegenerative diseases. We are currently conducting a Phase 2/3 clinical trial in France of Delayed-release Cysteamine Bitartrate capsules (RP103), for the potential treatment of Huntington’s Disease. The researchers at Laval have compiled a solid preclinical foundation which, when combined with the existing safety profile of cysteamine, may enable us to more rapidly advance this program for the potential treatment of Parkinson’s Disease in to a Phase 2 clinical trial. We plan to seek grant support to help fund the clinical development of RP103 in Parkinson’s, as we have done with our Huntington’s and NASH programs.”
Dr. Francesca Cicchetti, Professor at the Faculty of Medicine at Université Laval, said, “Treatments for Parkinson’s Disease that provide neuroprotective/neurorestorative effects remain an urgent unmet clinical need. Our preclinical results obtained in various animal models of parkinsonism substantiate the need for more research with cysteamine for this indication. We hope to see the study of this potential cysteamine treatment move to clinical trial.”
In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego for the development of RP103 and other forms of cysteamine for the potential treatment of Huntington’s Disease currently in a Phase 2/3 clinical trial in France, non-alcoholic steatohepatitis (“NASH”) currently in a Phase 2b clinical trial in the U.S., and for the development of RP103 for the potential treatment of nephropathic cystinosis, which Raptor has recently filed for marketing approval in the U.S. and E.U.
The U.S. Food and Drug Administration (“FDA”) has accepted for filing Raptor’s New Drug Application (“NDA”) for RP103 for the potential treatment of nephropathic cystinosis and assigned the user fee goal date of January 30, 2013. Raptor’s E.U. marketing application of RP103 for the potential treatment of nephropathic cystinosis is under review by the EMA, and Raptor expects a decision in the first half of calendar 2013. Raptor has licensed issued patents related to the potential treatment of Huntington’s Disease and other neurodegenerative diseases with cysteamine and related compounds from Niigata University and Weizmann Institute and patent applications for the potential treatment of malaria and other parasitic diseases from McGill University. Raptor has been granted orphan product designation for RP103 for the potential treatment of nephropathic cystinosis by the European Medicines Agency and FDA and for the potential treatment of Huntington’s Disease by the FDA.
Raptor Pharmaceuticals is a NASDAQ-listed biopharmaceutical company with a product pipeline that may advance therapeutic options for 8 different indications and includes potential for product launch within the next 12 months. The company’s mission is to research, produce, and deliver medicines that improve life for patients with severe, rare diseases. Within its clinical development pipeline, Raptor is developing its proprietary delayed-release form of cysteamine to target genetic and metabolic disorders, including Parkisons Disease, Huntingtons Disease, and cystinoses, among other conditions.
For more information visit www.raptorpharma.com
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