The Michael J. Fox Foundation Awards $2.5 Million to Support Ceregene's New Phase 2 Neurturin Clinical Study for Parkinson's Disease

Enrollment Completed in the New Phase 1 CERE-120 Study

Jun 22, 2010, 12:30 ET from Ceregene

SAN DIEGO, June 22 /PRNewswire/ -- The Michael J. Fox Foundation for Parkinson's Research (MJFF) will provide $2.5 million to support Ceregene Inc.'s new Phase 2 study of CERE-120. This novel gene therapy product delivers the gene for neurturin, a potent neurotrophic (growth) factor that has shown potential to improve the status of degenerating neurons in both preclinical and clinical studies. CERE-120 has the potential to improve motor function as well as slow Parkinson's disease (PD) progression. The competitive funding was awarded as part of the MJFF 2010 LEAPS (Linked Efforts to Accelerate Parkinson's Solutions) program on neurotrophic factors.

"Ceregene is enthusiastic about the continued support from The Michael J. Fox Foundation and this award will allow us to enroll the appropriate number of Parkinson's patients to statistically power our upcoming controlled Phase 2 clinical study," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc. "This funding marks the fourth award from The Michael J. Fox Foundation for Ceregene and we are truly grateful for their support," noted Dr. Ostrove.

The principal investigators of this award are Ceregene's executive vice president and chief scientific officer Raymond T. Bartus, Ph.D. and Joao Siffert, M.D., Ceregene's chief medical officer. "We have made significant progress researching and implementing important refinements to improve the delivery and bioactivity of CERE-120, based on insight gained from our past clinical trials in PD patients. We now look forward to testing these improvements in a controlled clinical trial in PD patients to more clearly establish the safety and improved efficacy of CERE-120," stated Dr. Bartus.

"With the enrollment in the Phase 1 portion of this study complete, we now look forward to initiating the controlled Phase 2 portion. The CERE-120 clinical development continues to receive strong support from leading movement disorder centers and The Michael J. Fox Foundation," said Dr. Siffert.

MJFF funding complements Ceregene's own multi-million dollar investment in the study. Patient enrollment for  the new Phase 2 study is expected to begin later this summer at 10 major medical centers throughout the US including: Baylor College of Medicine, Houston, TX; Columbia University Medical Center in New York, NY; Duke University, Durham, NC; Emory University Hospital, Atlanta, GA; Mount Sinai Medical Center New York, NY; University of Alabama at Birmingham, AL; University of California, San Francisco, CA; University of Pennsylvania, Philadelphia, PA; Rush University Medical Center, Chicago, IL and Stanford School of Medicine, Palo Alto, CA.

About CERE-120 and its Potential for 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. The revised dosing regimen being employed in the current Phase 1 trial administered CERE-120 to both the substantia nigra as well as the putamen, thereby accounting for deficiencies in the ability of these degenerating neurons to efficiently transport neurturin from its terminals to its cell bodies. Once CERE-120 is delivered to the brain, it provides stable, long-lasting expression of neurturin in a highly targeted fashion. As has been announced, the previous Phase 2 trial of CERE-120 involved 58 patients and did not demonstrate a difference between CERE-120 and control in the primary endpoint at 12 months. However, CERE-120 did show improvement on several secondary endpoints at 12 months, and importantly at 18 months CERE-120 demonstrated a statistically significant treatment effect on the primary endpoint. Equally important, on no measure did the sham-surgery control group perform better than the CERE-120-treated group. Based on those findings, and insight gained from analyses of post-mortem brain tissue from two CERE-120 treated patients, the company revised the dosing regimen which is reflected in the current Phase 1 trial, which has completed enrollment, as well as the planned Phase 2 trial.

About Ceregene

Ceregene, Inc. is a San Diego-based biotechnology company focused on the delivery of neurotrophic (or nervous system growth) 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 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-140 is in preclinical development for several blinding, ocular disorders. Ceregene was launched in January 2001. The company's investors include Alta Partners, MPM Capital, Investor Growth Capital and BioSante Pharmaceuticals (Nasdaq: BPAX) which acquired its position following its merger with Cell Genesys, Inc. in October 2009, as well as Hamilton BioVentures and California Technology Partners.

About The Michael J. Fox Foundation

Founded in 2000, 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 almost $184 million in research to date.

SOURCE Ceregene