Neurochem Inc announced that its Investigational New Drug (IND) application for Cerebril, the Company's lead drug candidate for the prevention of recurrent Hemorrhagic Stroke due to Cerebral Amyloid Angiopathy (CAA), has been cleared by the U.S. Food and Drug Administration (FDA). The application was filed last September 23, 2002 and its clearance means that the Company may now proceed with the initiation of its Phase II clinical trial for patients suffering from this life-threatening disease.
"The IND approval for Cerebril is an important development adding to the significant clinical milestones achieved by Neurochem this year," said Dr. Louis R. Lamontagne, President and CEO of Neurochem. "Our strong pipeline of drug candidates includes Fibrillex in a pivotal Phase II/III clinical trial, Alzhemed and Cerebril both in Phase II, and an exciting preclinical program for Diabetes Type 2 and Epileptic seizures and convulsions arising from Traumatic Brain Injury."
The Phase II clinical trial will be initiated in four U.S. centers this Fall, and will investigate the safety, tolerability and pharmacokinetic profile of Cerebril in CAA patients who have had lobar cerebral hemorrhage. The study has been designed as a multicenter, randomized, double-blind, placebo-controlled and parallel design study. CAA patients with lobar cerebral hemorrhage will be randomized to receive placebo or Cerebril (3 different dose levels) for a period of twelve weeks.
Neurochem previously investigated the safety, tolerability and pharmacokinetic profiles of Cerebril in healthy volunteers (young and old) in four Phase I clinical trials. In all four studies, Cerebril was shown to be safe and well tolerated at the anticipated therapeutic dose in both young and elderly volunteers with a good pharmacokinetic profile.
Cerebril is a small organic molecule that has been designed to modify the course of Hemorrhagic Stroke due to CAA by interfering with the association between glycosaminoglycans (GAGs) and AB amyloid protein in the blood vessels of the brain of patients with CAA. As part of a "disease modifying" class of drug candidates, Cerebril is expected to act at two levels: to prevent and stop the formation and deposition of amyloid fibrils and in binding to AB, to inhibit the inflammatory response associated with amyloid build-up in Hemorrhagic Stroke due to CAA.