The trial did not demonstrate an appreciable difference between patients treated with CERE-120 versus those in the control group. Both groups showed an approximate seven point improvement in the protocol-defined primary endpoint, relative to a mean at baseline of approximately 39 points.
Both groups had a substantial number of patients who demonstrated a meaningful clinical improvement from baseline. CERE-120 appeared to be safe and well tolerated, the company said.
Patients in the Phase II trial were enrolled across nine leading academic medical centers in the US, with two thirds of patients receiving CERE-120 and one third enrolled into a control group. Patients received a single administration of CERE-120 via stereotactic neurosurgery to deliver the drug into the putamen region of the brain and were followed for 12 months for safety and efficacy.
Raymond Bartus, chief scientific officer of Ceregene, said: The medical research community has for several decades attempted to use the unique healing properties of neurotrophic factors to treat serious neurodegenerative diseases, and Ceregene’s use of gene therapy as an essential enabling technology to translate this idea into an effective human therapeutic has consistently generated strong empirical support.
Thus, we are stunned by the results of this trial and will continue to analyze the data in order to gain greater insight into the factors that may have contributed to this negative outcome, not only to build upon this insight for our Parkinson’s program, but also to help assure continued successful development of our product candidates for other diseases.