SAN DIEGO, Nov. 14 /PRNewswire-FirstCall/ -- Ceregene, Inc. today announced that the company has completed enrollment of 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. The trial enrolled 58 patients with advanced Parkinson's disease across nine medical centers in the United States. The company expects to announce results from the trial in late 2008. CERE-120 is designed to deliver the neurturin gene, a nervous system growth factor with the ability to maintain the growth and normal function of dopamine-producing neurons which deteriorate in Parkinson's disease.
The Phase 2 trial, which was supported in part by The Michael J. Fox Foundation for Parkinson's Research, was prompted by positive results from a Phase 1 study of CERE-120 which demonstrated a 36 percent (p<0.001) reduction in Parkinson's symptoms at 12 months after administration, as measured by the Unified Parkinson's Disease Rating Scale (UPDRS) motor "off" score ("motor off" meaning patients were off Parkinson's medication at evaluation time). CERE-120 was also shown to be well tolerated in the Phase 1 study. The Phase 2 study was designed to confirm these findings in a controlled trial by randomly assigning patients to receive either CERE-120 or a control treatment at a 2 to 1 ratio and is powered at 90 percent to demonstrate clinical efficacy. The patients in this Phase 2 study will be followed for 12 months for safety and efficacy.
"Completing full enrollment of this multi-center controlled trial represents an important milestone in CERE-120's development as well as in the development of Ceregene as an emerging biotechnology company," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "We are encouraged by the positive results from our Phase 1 trial and carefully designed this Phase 2 study as a controlled trial to confirm those results. With positive results from the Phase 2 study, we would expect to initiate a Phase 3 trial for CERE-120 in 2009."
"CERE-120 represents a truly revolutionary future treatment option for Parkinson's disease patients, given that a single treatment is not only intended to improve the symptoms of the disease, but also slow disease progression. We appreciate the courage of the patients and the efforts of the physicians who have participated in our Phase 2 trial and hope that CERE-120 may someday provide a meaningful difference in the lives of Parkinson's disease patients. We thank all trial participants for enrolling this study safely and within the projected time frame," stated Raymond T. Bartus, Ph.D., Ceregene's senior vice president of clinical and preclinical R&D and chief operating officer.
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. Genzyme Corporation (Nasdaq: GENZ) has licensed the ex-North American rights for the development and commercialization of CERE-120 from Ceregene, a deal which was announced in June 2007.
About Parkinson's Disease
Parkinson's disease is a progressive movement disorder that affects at least 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 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 1/2 studies for the treatment of Alzheimer's disease, and CERE-120 (AAV2-Neurturin) which recently completed enrollment of a controlled Phase 2 study for Parkinson's disease. CERE-130 and CERE-140 are in preclinical development for ALS (Lou Gehrig's disease) and ocular disorders, respectively. Ceregene was launched in January 2001 and is a former subsidiary of Cell Genesys, Inc. (Nasdaq: CEGE). The company's investors include Alta Partners, MPM Capital, Investor Growth Capital and Cell Genesys as well as Hamilton BioVentures and California Technology Partners.
|SOURCE Ceregene, Inc.|
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