Ceregene, Inc., a biopharmaceutical company, announced today that enrollment has begun in a new double-blind sham surgery-controlled Phase 2b study evaluating CERE-120 in Parkinson's disease patients. CERE-120 is a gene therapy product that delivers the neurotrophic factor neurturin to degenerating or dying dopamine neurons. Data from the first Phase 2 clinical trial of CERE-120 are being published later this year in Lancet Neurology (to appear online October 21, 2010).
The launch of Phase 2 follows a fully enrolled Phase 1 portion that demonstrated early safety using an enhanced CERE-120 dosing regimen targeting the substantia nigra and putamen, two brain areas especially affected by Parkinson's disease. This study is enrolling at 11 major medical centers throughout the United States and includes investigators Drs. Mark Stacy and Dennis Turner at Duke University School of Medicine, Drs. Catherine Cho and Ron Alterman at Mount Sinai Medical Center, New York, and Drs. Stewart Factor and Nicholas Boulis at Emory University Hospital, who were all involved in treating the patients in the Phase 1 portion of this study.
Additional clinical sites include Columbia University Medical Center, NYC, NY; Beth Israel Medical Center, NYC, NY; University of Pennsylvania Hospital, Philadelphia, PA; University of Alabama, Birmingham, AL; Rush University Medical Center, Chicago, IL; Baylor College of Medicine, Houston, TX; University of California, San Francisco, CA and Stanford University School of Medicine, Palo Alto, CA.
Details on the clinical study can be found at www.ceregene.com or www.clinicaltrials.gov/ct2/show/NCT00985517.
This Phase 2b clinical study is partially funded by a $2.5M LEAPS (Linked Efforts to Accelerate Parkinson's Solutions) award from The Michael J. Fox Foundation for Parkinson's Research (www.michaeljfox.org).
"We are proud to play a key role in this potentially high-impact project," said Todd Sherer, Ph.D., vice president of research programs at The Michael J. Fox Foundation. "The Foundation continues to believe that trophic factors hold enormous potential to become transformative therapeutics for people with Parkinson's."
The first Phase 2 clinical trial of CERE-120, completed in November 2008, did not meet its pre-designated primary endpoint (Unified Parkinson's Disease Rating Scale- Motor Off at 12 months) although a clinically modest benefit was seen in improving motor performance and quality of life in Parkinson's disease patients, based on statistically significant differences in several secondary endpoints. Even greater improvement was seen following an analysis of all patients who were assessed under blinded conditions at 15 to 18 months post-treatment, including on the primary measure (UPDRS motor off at 18 months,s disease patients was gained from CERE-120-treated patients who died of unrelated causes, combined with the clinical results, led to important changes in CERE-120 dosing intended to significantly enhance its bioactivity and response to treatment. This revised dosing paradigm now targets both the terminals (or nerve endings) of the degenerating neurons in a site in the brain called the putamen, as well as the cell bodies of these neurons, located in another brain region called the substantia nigra.
"We are pleased to initiate a new controlled Phase 2b study that is statistically powered to demonstrate the efficacy of CERE-120," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "We continue to strongly believe that CERE-120 has the potential to improve the symptoms of Parkinson's disease while also delaying further disease progression, and may therefore represent a significant advancement in the treatment of patients for whom existing treatments inevitably fail as their disease progresses."
"This new, improved dosing approach we have developed to account for functional changes in Parkinson's disease neurons should assure that adequate neurturin protein is expressed throughout the degenerating nigrostriatal system. Our nonclinical studies suggest this may significantly enhance the biological effects and therefore the clinical benefit of CERE-120," stated Raymond T. Bartus, Ph.D., Ceregene's executive vice president and chief scientific officer.
"The revised CERE-120 dosing procedure appears safe, to date, and our investigators have been able to implement the dosing protocol with no surgical complications," added Joao Siffert, M.D., vice president and chief medical officer at Ceregene.
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