SAN DIEGO, Aug. 5 /PRNewswire/ -- Ceregene, Inc. today announced that the Michael J. Fox Foundation will provide partial funds for long-term follow-up testing of patients enrolled in the company's Phase 2 trial of CERE-120 for Parkinson's disease. The funding will enable Ceregene to collect and analyze more extensive data for up to 48 months from patients with advanced Parkinson's disease who were enrolled in the double-blind, controlled trial which ended in November 2008.
As was previously announced, the Phase 2 trial of CERE-120 involving 52 patients, failed to demonstrate a difference in the primary endpoint between patients in the CERE-120 versus control group. It was subsequently announced, however, that CERE-120 suggested improvement on several secondary endpoints at 12 months, and at 18 months, a statistically significant treatment effect on the primary endpoint emerged, while several additional secondary endpoints also showed improvement. In contrast, on no measure did sham patients perform better than CERE-120 patients, at either 12 or 18 months. Based on those findings, and insight gained from analyses of post-mortem brain tissue from two CERE-120 treated patients, the company has revised the dosing regimen and expects to initiate a new trial of CERE-120 in the near future.
"We are pleased to be able to gather unique, long-term data from both CERE-120 treated and control patients and thus gain more information about the potential long-term effects of CERE-120 on Parkinson's disease," stated Raymond T. Bartus, Ph.D., Ceregene's executive vice president and chief scientific officer. "We very much appreciate the support the MJFF has provided to enable us to expand the testing of these patients and further enrich our data base. Our conviction about CERE-120's potential to treat Parkinson's disease remains strong. We learned a significant amount from the initial Phase 2 trial which enabled us to design a protocol that should maximize the potential for the greatest treatment effect in our upcoming Phase 2 trial."
About Phase 2 Trial of CERE-120
Ceregene's Phase 2 trial was a double-blind, controlled clinical trial that completed enrollment of 58 patients with advanced Parkinson's disease in October 2007. Patients in the Phase 2 trial were enrolled across nine leading academic medical centers in the United States, with two thirds of patients receiving CERE-120 and one third enrolled into a control group. Patients received a single administration of CERE-120 via stereotactic neurosurgery to deliver the drug into the putamen region of the brain and were followed for a minimum of 12 months for safety and efficacy, with over half the subjects followed for 15 to 18 months under blinded conditions, allowing longer-term analyses of the therapeutic effects of CERE-120.
About CERE-120 and its Application to Treating Parkinson's Disease
CERE-120 is composed of an adeno-associated virus (AAV) vector carrying the gene for neurturin, a naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping them alive and restoring normal function. Neurturin 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. Degeneration of these neurons is responsible for the major motor impairments of Parkinson's disease. CERE-120 has been delivered by stereotactic injection to the terminal fields (i.e., the ends of the degenerating neurons), located in an area of the brain called the putamen. The cell bodies for these same neurons are located in a different area of the brain, called the substantia nigra. Once CERE-120 is delivered to the brain, it provides stable, long-lasting expression of neurturin in a highly targeted fashion. Genzyme Corporation (Nasdaq: GENZ) has licensed the ex-North American rights for the development and commercialization of CERE-120 from Ceregene, an agreement that was announced in June 2007.
About Parkinson's Disease
Parkinson's disease is a progressive movement disorder that affects a million people in the United States. Its main symptoms, stiffness, tremors and slowed movements and gait, are caused by a loss of dopamine-containing nerve cells in the substantia nigra, which project their axons to the putamen. Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson's patients exhibit a progressive inability to initiate and control physical movements. There is currently no treatment that can reverse the degeneration of these neurons, let alone cure Parkinson's disease.
About The Michael J. Fox Foundation
The Michael J. Fox Foundation is dedicated to ensuring the development of better treatments, and ultimately a cure, for Parkinson's disease through an aggressively funded research agenda. MJFF has funded $149 million in research to date.
Ceregene, Inc. is a San Diego-based biotechnology company focused on the delivery of nervous system growth (neurotrophic) factors for the treatment of neurodegenerative and retinal disorders using gene delivery. Ceregene's clinical programs include CERE-110, an AAV2 based vector expressing nerve growth factor that is currently in a multi-center, controlled Phase 2 study for the treatment of Alzheimer's disease, and CERE-120 (AAV2-Neurturin) 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.
SOURCE Ceregene, Inc.