Raptor initiates dosing in DR Cysteamine Phase 2 clinical trial for Huntington's Disease

Nov 8 2010

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).

In addition to intellectual property licensed from the University of California, San Diego ("UCSD") related to its DR Cysteamine programs, Raptor holds exclusive worldwide licenses to intellectual property related to the potential treatment of Huntington's Disease from the Weizmann Institute of Science in Israel and Japan's Niigata University.

Huntington's Disease is a rare, progressive, and hereditary neurological disease that often leads to death within 15 to 20 years after diagnosis. The disease is thought to affect as many as 20,000-30,000 patients in the U.S. and a comparable number in Europe. There is no currently available drug that targets the defective gene believed to cause Huntington's Disease, which results in the degeneration of certain nerve cells in the brain. The disease is characterized by uncontrollable movements and mood swings or depression, followed by dementia. 

Grant Awarded to Raptor for Qualifying Therapeutic Discovery Project ("QTDP")

In July 2010, Raptor applied for the QTDP grants from the U.S. Government for five of its research programs including its cystinosis, Huntington's Disease and NASH (non-alcoholic steatohepatitis) clinical programs and its HepTide^TM and WntTide^TM preclinical cancer research programs. Raptor was granted an aggregate of $1 million for all five programs of which $827,000 was funded in October 2010 and the balance will be funded in October 2011. Raptor received the maximum allowable program award of $244,000 for each of its three clinical programs. 

New Issued Patent for NeuroTrans^TM

Additionally, a third Raptor patent will issue on November 9, 2010 adding to the NeuroTrans^TM portfolio.  US Patent 7,829,537 includes composition claims on conjugates of RAP-based peptides and therapeutic agents.  In July 2009, Raptor entered into a license and research collaboration agreement with Roche that includes an exclusive worldwide license to NeuroTrans^TM peptides for use in the delivery of diagnostic and therapeutic molecules across the blood-brain barrier.

SOURCE Raptor Pharmaceutical Corp.