Enrollment complete in Ceregene's CERE-120 Phase 2b trial for Parkinson's

Dec 1 2011

Ceregene, Inc. reported today that it has completed enrollment in its Phase 2b trial of CERE-120, a gene therapy product expressing the neurturin (NRTN) gene for the treatment of Parkinson's disease.  This Phase 2b study enrolled 51 patients at 11 medical centers throughout the United States.  CERE-120 is comprised of an AAV vector intended to provide constant, sustained expression of neurturin (NRTN), a neurotrophic factor shown to rescue dying dopaminergic neurons, restoring their function and protecting them from cell death.

Ceregene also reported that it has been chosen to present its work on CERE-120 at Windhover's annual Therapeutic Area Partnership conference as one of the "Top 10 Neuroscience Projects to Watch".  Raymond T. Bartus, Ph.D., Ceregene's executive vice president and chief scientific officer will present at the Boston conference on Thursday December 1st at 1:30 pm.

"Safely completing enrollment in our current Phase 2b CERE-120 Parkinson's study is an important milestone in Ceregene's history, as this study builds on the results of an earlier Phase 2 trial and is powered to demonstrate statistical significance of clinical benefit for CERE-120," stated Jeffrey M. Ostrove, Ph.D., Ceregene's president and chief executive officer.  "We have been fortunate to work with many of the world's leading Parkinson's disease experts in both the clinical trial design and implementation," noted Dr. Ostrove.

"The protocol and dosing scheme developed for the current study leveraged the unique and important insight we gained from the results of our previous clinical studies with CERE-120," noted Dr. Bartus. "Among other things, we observed a fundamental deficiency in the degenerating neurons of Parkinson's disease, which limits how effectively material, like NRTN, is transported from the terminals of these neurons to their cell bodies.  This provided the key insight required to recognize the need to inject CERE-120 directly into the substantia nigra, thus reducing dependency on retrograde transport."  Additionally, because of the safety record thus far established with CERE-120, the current Phase 2b study injected a 4-fold higher CERE-120 dose into the putamen, compared to prior studies.  "These dosing changes are intended to help assure that the entire degenerating neuron is exposed to sufficient neurotrophic factor, stimulating a more robust neurotrophic response, which in turn, should further enhance and accelerate neuronal repair and clinical benefit," continued Dr. Bartus. 

Ceregene's previous Phase 2a clinical study of CERE-120 demonstrated "proof of concept", showing efficacy on a number of secondary clinical motor and quality of endpoints, with no measurement similarly favoring the sham control treatment.  While that study missed the primary endpoint at 12 months post treatment, a statistically significant improvement from CERE-120 was seen on this measure at 15 and 18 months, post treatmentUPDRS off" score which is a measure of motor function commonly used in clinical trials of Parkinson's disease.  Ceregene expects to report data from the new Phase 2b study near the end of the first quarter of 2013.

SOURCE Ceregene, Inc.