SAN DIEGO, Calif., April 13 // -- Ceregene, Inc., a biopharmaceutical company, confirmed today that enrollment is well under way in its randomized, controlled, double-blind, Phase 2 clinical trial evaluating CERE-120, a gene therapy product in development for the treatment of Parkinson's disease. This trial follows positive results from a Phase 1 trial of CERE-120 in 12 patients in which preliminary data showed the treatment to be well tolerated and to provide a 35 - 40 percent (p<0.001) reduction in Parkinson's symptoms at six through 12 months post treatment. The complete data set from the Phase 1 trial will be presented at the American Academy of Neurological Surgeons Meeting on Monday, April 16, 2007.
The Phase 2 clinical trial is now enrolling 51 patients with advanced Parkinson's disease at nine medical centers in the United States, with two-thirds of the patients enrolling in the active treatment group and one-third in the control group. Patients will receive CERE-120 via stereotactic neurosurgery to deliver the drug into their putamen-a region of the brain affected by the degeneration of neurons in Parkinson's disease. Patients will be followed for 12 months for safety and efficacy.
"Patients in the advanced stage of Parkinson's disease begin to run out of effective therapeutic options, and CERE-120 may have the ability to alleviate this great unmet medical need-especially given its potential to offer measurable and lasting benefit after just a single treatment," said William J. Marks Jr., M.D., principal investigator for the Phase 1 trial and associate professor of Neurology at the University of California, San Francisco (UCSF). "I am encouraged by the preliminary data from the Phase 1 clinical trial of CERE-120, including its excellent safety profile, and am pleased to be involved in further investigating the therapy in this controlled Phase 2 trial with the hope that it may someday provide benefit to patients with Parkinson's disease."
"The Michael J. Fox Foundation for Parkinson's Research funded a portion of our Phase 1 clinical trial, and upon seeing the results, chose to contribute funding for the Phase 2 trial as well. We are grateful for this vote of confidence in CERE-120," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "We are encouraged by the preclinical and early clinical data from CERE-120 and believe that this therapy may not only have the ability to improve the symptoms of Parkinson's disease, but also to treat the underlying disease and slow its progression."
"At least one million Americans currently suffer from Parkinson's disease, a number expected to rise with the aging of the baby boomer generation. The Michael J. Fox Foundation is dedicated to driving development of new treatments that can slow or stop this disease, and we're hopeful that the work being done by Ceregene could play a part in this goal," stated Deborah Brooks, president and co-founder of the Michael J. Fox Foundation for Parkinson's Research.
"We are pleased with the excellent institutions who have chosen to participate as clinical sites for our Phase 2 trial of CERE-120, including Baylor College of Medicine, Cleveland Clinic, Duke University, Mount Sinai College of Medicine in New York City, Oregon Health Sciences University, Rush University Medical Center, University of Alabama at Birmingham, University of California, San Francisco, and University of Pennsylvania," stated Raymond T. Bartus, Ph.D., Ceregene's senior vice president of clinical and preclinical R&D and chief operating officer. "The trial, which has been powered to achieve statistical significance, is expected to complete enrollment in the third quarter of this year." Contacts at the medical centers involved in this study can be found through a link on the Ceregene web site: http://www.ceregene.com.
CERE-120 is composed of an adeno-associated virus (AAV) vector carrying the gene for neurturin (NTN), a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and functioning normally. NTN is a member of the same protein family as glial cell-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties, and both have been shown to benefit the midbrain dopamine neurons that degenerate in Parkinson's disease and are responsible for the major motor impairments. CERE-120 is delivered by stereotactic injection to the affected area of the brain, providing stable, long-lasting expression of NTN in a highly targeted fashion. Ceregene owns exclusive technology and product rights to CERE-120.
About Parkinson's Disease
Parkinson's disease is a progressive movement disorder that affects as many as one million people in the United States. It is caused by a reduction in dopamine containing nerve cells of the midbrain. Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson's patients experiencing a reduction of dopamine-producing nerve cells exhibit the progressive inability to initiate and control physical movements. There is currently no cure for Parkinson's disease.
Ceregene, Inc. is a San Diego-based biotechnology company focused on the development of gene therapies for neurodegenerative disorders. Ceregene is in the clinic with CERE-110, an AAV2 based vector expressing nerve growth factor that is being tested as a treatment for Alzheimer's disease, and with CERE-120 for Parkinson's disease. CERE-130 is in preclinical development for ALS. Ceregene was launched in January 2001 and is a former subsidiary of Cell Genesys, Inc. (Nasdaq: CEGE), which is headquartered in South San Francisco, CA. Ceregene's investors include Alta Partners, MPM Capital, Investor Growth Capital and Cell Genesys, as well as Hamilton BioVentures and California Technology Partners.
About The Michael J. Fox Foundation
Founded in 2000, The Michael J. Fox Foundation for Parkinson's Research is dedicated to ensuring the development of a cure for Parkinson's disease within this decade through an aggressively funded research agenda. The Foundation has funded more than $90 million in research to date, either directly or through partnerships.