Phase II study of CERE 120 (Ceregene) for Parkinsons fails to reach primary endpoint

Top-line data from a double-blind, randomized, controlled Phase IIb clinical study of CERE 120 (AAV-neurturin), from Ceregene, for Parkinson's disease failed to demonstrate statistically significant efficacy on its primary endpoint. Fifty-one patients with moderately advanced Parkinson's disease which could not be controlled with conventional medication were enrolled. Approximately half received CERE 120 while the other half received sham (placebo) surgery.

Patients were monitored for 15-24 months to assess safety and changes in disease symptoms, using multiple endpoints such as the Unified Parkinson's Disease Rating Scale (UPDRS), Daily Diaries and PDQ-39 (a measure of quality of life). Results show the trial didn't reach statistically significant efficacy on the primary endpoint (UPDRS-motor off) but one secondary endpoint (Diary-off score) did produce statistically significant benefit. The trial also provided further evidence for the safety of CERE 120 and the dosing methods employed.