NEWYORK, NY — The Michael J. Fox Foundation has signed on to fund the long-term, open-label analysis of data from Ceregene, Inc.’s Phase 2 trial of CERE-120, a gene therapy approach to deliver trophic factor neurturin to the brains of Parkinson’s patients. The funding will allow Ceregene to collect and analyze data from trial enrollees for another 48 months. While the study will be unblinded, the goal is to gather as much data on safety and efficacy as possible in an open-label setting, while looking for suggestions of a longer-term neuroprotective effect.
“Our Foundation remains hopeful that neurotrophic factors can yield new and better treatments for PD,” said Katie Hood, MJFF CEO. “Extending the observation and analysis of Phase 2 trial enrollees represents a major opportunity to amass new data and learn from patients’ experience.”
The double-blind, controlled clinical trial of CERE-120, which concluded in November 2008, failed to meet its primary endpoint — improved Unified Parkinson’s Disease Rating Scale (UPDRS) motor-off scores after 12 months. However, Ceregene recently reported that an analysis of multiple secondary endpoints suggest modest improvement following CERE-120 and that a revised dosing paradigm will be employed in an additional trial to start later this year. MJFF’s ongoing partnership will allow Ceregene to continue collecting and analyzing data from Phase 2 trial enrollees for another 48 months.
The Michael J. Fox Foundation supported both the Phase 1 and Phase 2 trials of CERE-120 with leadership funding from the family of MJFF Board member Daniel E. Spitzer, MD, and The Pioneer Fund, respectively. The Phase 1 trial in 12 patients demonstrated that CERE-120 was safe and well-tolerated.