CEREGENE ANNOUNCES RESULTS FROM PARKINSON'S TREATMENT TRIAL

Ceregene has announced that CERE-120, a gene therapy product in development for the treatment of Parkinson's disease, was well-tolerated and appeared to reduce symptoms by approximately 40 percent, as measured by the Unified Parkinson's Disease Rating Scale motor "off" score, in an open-label Phase I study in 12 patients with advanced disease.

The study was supported in part by a grant from The Michael J. Fox Foundation for Parkinson's Research. Based on the initial results, the foundation announced plans to partially fund a Phase II study with a $1.9 million grant

CERE-120 consists of an adeno-associated virus vector carrying the gene for neurturin, a naturally occurring protein, whose role is to keep dopamine-secreting neurons functioning normally. All 12 patients enrolled in the study underwent stereotactic neurosurgery to deposit CERE-120 into their putamen. The putamen is a region of the brain that undergoes degeneration and reduced dopamine production in Parkinson's disease patients and this has been closely linked to the major motor deficits in these patients. All patients entered in the trial were judged to have inadequate control of their disease with standard levodopa therapy and were otherwise potential candidates for additional treatment interventions such as deep brain stimulation surgery.

CERE-120 was delivered at two different doses, with patients receiving the low dose demonstrating approximately 40 percent improvement in UPDRS motor "off" scores at nine months and patients receiving the four-fold higher dose showing a similar effect three months sooner.