San Diego, CA - November 4, 2008 – Ceregene, Inc., a biopharmaceutical company, today announced that the University of California, San Diego (UCSD) has received a $5.4 million grant from the National Institute of Aging at the National Institutes of Health (NIH) to support a Phase 2 clinical study of Ceregene’s CERE-110, a gene therapy product designed to deliver nerve growth factor (NGF) for the treatment of Alzheimer’s disease. A Phase 1 study of CERE-110 indicated that a single administration of the therapy was well tolerated, though the study was too small to make a definitive statement about treatment efficacy.
Ceregene anticipates initiating the Phase 2 study, which is expected to enroll 50 patients with mild to moderate Alzheimer’s disease at multiple clinical trial sites in the U.S. early next year. The study will be double-blinded and will evaluate the treatment arm versus an appropriate control arm with respect to safety measures and cognitive function and quality of life at two years.
“We are pleased to be involved in evaluating CERE-110 which we believe may be a promising therapy for Alzheimer’s disease,” stated Paul Aisen, M.D., principal investigator of the planned Phase 2 trial and director of the Alzheimer’s Disease Cooperative Study at University of California, San Diego, a preeminent research consortium for testing new treatments for Alzheimer’s disease. “Over five million people in the U.S. are currently living with Alzheimer’s disease—a disease which is devastating to patients and their families—and we are in need of more effective treatment options for these patients.”
“The safety profile that we have observed from CERE-110 in the Phase 1 study is very encouraging, and we are delighted that this NIH grant will enable us to further evaluate this product candidate. We are especially grateful for this funding given that it was based on peer review, and we look forward to using it to advance this product. The development of CERE-110 was led by Raymond T. Bartus, Ph.D., Ceregene’s executive vice president and chief scientific officer and is a continuation of the pioneering work of Mark Tuszynski, M.D., Ph.D., Ceregene’s co-founder and a professor of Neurosciences at UCSD,” stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. “Similar to the other therapies in our pipeline which target Parkinson’s disease, ocular disorders and ALS (Lou Gehrig’s disease), CERE-110 may have the ability to not only treat the symptoms of Alzheimer’s disease but to protect dying neurons and potentially result in the slowing of disease progression.”
CERE-110 is composed of an adeno-associated viral (AAV) vector carrying the gene for NGF, a naturally occurring protein that maintains survival of nerve cells in the brain. CERE-110 is surgically injected into the Nucleus Basalis of Meynert (NBM), a brain region where cholinergic cell degeneration occurs in Alzheimer's disease. The cholinergic system is important in memory and cognitive function, and a restoration in the function of this system may improve memory in individuals with Alzheimer’s disease. Delivery of NGF using an AAV vector should have the potential to induce sustained expression of NGF, resulting in long-lasting restoration of the function.
The Phase 1 study was conducted at Rush University Medical Center, Chicago and UCSD. The open-label study involved 10 subjects with mild-to-moderate Alzheimer’s disease. The administration of CERE-110 was generally safe and well tolerated. The 10 participants underwent cognitive testing, measures of activities of daily living, MRI scans and PET (positron emission tomography) scans. Increases in brain metabolism were observed in several cortical regions at six months and 12 months (p<0.05) in four subjects as compared to other severity-matched individuals with Alzheimer’s disease suggesting a potential reversal of patterns typically observed in Alzheimer’s disease.
About Alzheimer’s Disease
Alzheimer’s disease is a progressive disorder of the brain that gradually affects one’s memory and ability to learn, reason, communicate and carry out daily activities. There are now more than five million people in the United States living with Alzheimer’s disease, and there is currently no cure.
Ceregene, Inc. is a San Diego-based biotechnology company focused on the delivery of nervous system growth factors for the treatment of neurodegenerative disorders using gene delivery. Ceregene’s clinical programs include CERE-110, an AAV2 based vector expressing nerve growth factor that is currently in Phase 1 studies for the treatment of Alzheimer’s disease, and CERE-120 (AAV2-Neurturin) which has completed enrollment of a controlled Phase 2 study for Parkinson’s disease. CERE-135 and CERE-140 are in preclinical development for ALS (Lou Gehrig’s disease) and ocular disorders, respectively. Ceregene was launched in January 2001. The company’s investors include Alta Partners, MPM Capital, Investor Growth Capital and Cell Genesys, Inc. (Nasdaq: CEGE) as well as Hamilton BioVentures and California Technology Partners.