NOVATO, Calif., Nov. 8, 2010 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), today announced that the first patient has been dosed in its Phase 2 clinical trial of DR Cysteamine for the potential treatment of Huntington's Disease. The Phase 2 clinical trial is being conducted under a previously announced collaboration agreement with The Centre Hospitalier Universitaire d'Angers ("CHU d'Angers"), which has commenced enrolling patients at eight clinical sites throughout France.
"Dosing of the first patient marks a key milestone in the clinical development of DR Cysteamine in patients with Huntington's Disease," said Dr. Christophe Verny, Lead Investigator for the Phase 2 clinical trial. "At present, the treatment for Huntington's Disease is limited to therapies that can help to minimize some of the symptoms, but does not target the root cause of the disease in an effort to slow its progression. It is our hope that this Phase 2 clinical trial will advance the preclinical work published by Drs. Sandrine Humbert and Frédéric Saudou from the Curie Institute in France, the results of which supported that cysteamine increased brain and blood levels of brain-derived neurotrophic factor ("BDNF"), a growth factor known to be deficient in Huntington's Disease patients through its inhibition of transglutaminase, a key regulating enzyme in BDNF production, contributing by that mechanism to slowing the progression of the disease."
The Phase 2 clinical trial will enroll 96 patients for an 18-month, placebo-controlled study. This will be followed by an open-label extension study with all patients on the placebo capsules rolling onto DR Cysteamine and all other patients continuing on DR Cysteamine for up to an additional 18 months. The primary end point of the clinical trial is based upon the Unified Huntington's Disease Rating Scale ("UHDRS"). Under the collaboration agreement with CHU d'Angers, Raptor will supply DR Cysteamine and placebo capsules for the clinical trial and open-label extension study in exchange for commercial rights to the clinical trial results. Clinical expenses of the study are covered by a grant from the French government (PHRC 2004-03bis CYST-HD).