SAN DIEGO, April 19, 2013 /PRNewswire/ -- 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 that assess motor function throughout the day, and PDQ-39 (a measure of quality of life), among others. Ceregene continues to analyze the data from this trial to gain as much information as possible. Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene, Inc. stated, "We are disappointed that we did not achieve broader statistical significance in this small clinical trial, perhaps due in part to the marked placebo effect noted above. That said, we at Ceregene want to acknowledge the courage of all of the patients enrolled in this very important study and their family members. We also would like to acknowledge the Michael J. Fox Foundation for Parkinson's Research for their long-term support as we continued the development of this novel neurotrophic factor-based treatment with the potential to improve symptoms and also slow disease progression." Raymond T. Bartus, Ph.D., executive vice president and chief scientific officer of Ceregene stated: "While we did not achieve the degree of efficacy we had hoped for in this trial, we are proud that our efforts have helped to establish that gene therapy can provide the enabling technology to safely deliver stable, long-term bioactive protein to targeted sites deep in the human brain and that the Parkinson's disease brain is able to show a positive response to neurotrophic factor stimulation. Hopefully, the information and insight we achieved and shared with the biomedical community will aid in the continuing effort to develop more effective therapies for many of these tragic and dehumanizing neurodegenerative diseases." C. Warren Olanow, M.D., Chairman Emeritus of the Department of Neurology and Professor of Neuroscience at the Mount Sinai School of Medicine in New York and a clinical advisor to Ceregene noted, "This was an extremely well-conceived and designed study. It is unfortunate for patients that broader benefits from this extremely promising therapy could not be demonstrated in this clinical trial. These results illustrate how difficult it is to establish clinical efficacy with entirely novel therapeutic approaches in complicated neurological diseases. The Ceregene team, its scientific advisors and the participating clinical sites are to be congratulated for that effort and the flawless execution of this difficult scientific study." Ceregene established a leadership position in the fields of gene therapy and neurotrophic factors for the treatment of neurodegenerative diseases. A total of over 100 patients have been safely dosed in two clinical programs: CERE-120 (AAV-NRTN) for Parkinson's disease and CERE-110 (AAV-NGF) for Alzheimer's disease. A randomized, controlled Phase 2 study of CERE-110 for Alzheimer's is continuing. It is fully enrolled and financially supported in large part by a grant from the NIH, with top line data expected by late 2014. In addition to the Parkinson's and Alzheimer's programs, Ceregene has conducted extensive preclinical work with CERE-120 for Huntington's disease, as well as another gene therapy/neurotrophic factor product (AAV-NT4) for blinding ocular diseases (such as Retinitis Pigmentosa, macular degeneration, diabetic retinopathy and glaucoma) and yet another (AAV-IGF1) for Lou Gehrig's disease (ALS). Ceregene is currently looking at strategic alternatives to advance its AAV gene therapy platform.