Ceregene Reports Disappointing Top-Line Data From Parkinson's Disease Phase 2b Study
Ceregene, Inc. today announced the top-line data from its double-blind, randomized, controlled Phase 2b clinical study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson's disease. The trial did not demonstrate statistically significant efficacy on the primary endpoint (UPDRS-motor off). However, one of the "key secondary endpoints" (Diary-off score), as defined and prespecified in the Statistical Analysis Plan, did produce statistically significant benefit. The trial also provided further evidence for the safety of CERE-120 and the dosing methods employed. A marked placebo effect was observed in this trial in that both the sham-surgery-control patients and the CERE-120 treated patients showed significant improvement following their surgery.
Fifty-one (51) patients with moderately advanced Parkinson's disease who could not be satisfactorily controlled with conventional Parkinson's medication were enrolled in the study at 11 leading clinical sites throughout the U.S. Approximately half of the patients received CERE-120 while the other half received sham (placebo) surgery as a control. Patients were monitored for 15-24 months to assess safety and changes in Parkinson's disease symptoms, using multiple endpoints such as the Unified Parkinson's Disease Rating Scale (UPDRS), Daily Diaries