San Diego, CA–October 19, 2010— 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 on-line
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
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
“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
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, p=0.025). Importantly, new scientific insight regarding degenerating
dopamine neurons in the brains of advanced Parkinson’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.
About CERE-120 and its Potential for Treating Parkinson’s Disease
CERE-120 is composed of an adeno-associated virus (AAV) vector carrying the gene for neurturin, a
naturally occurring protein known to repair damaged and dying dopamine-secreting neurons, keeping
them alive and restoring normal function. Neurturin is a member of the same protein family as glial
cell-derived neurotrophic factor (GDNF). The two molecules have similar pharmacological properties,
and both have been shown to benefit the dopamine neurons in the substantia nigra that degenerate in
Parkinson’s disease. Degeneration of these neurons is responsible for the major motor impairments of
Parkinson’s disease. CERE-120 has been delivered by stereotactic injection to the terminal fields, i.e.,
the ends of the degenerating neurons, located in an area of the brain called the putamen. The cell
bodies for these same neurons are located in a different area of the brain, called the substantia nigra,
and the amended dosing regimen being employed in the ongoing Phase 2b trial calls for administration
of CERE-120 to both the substantia nigra as well as the putamen. Once CERE-120 is delivered to the
brain, it provides stable, long-lasting expression of neurturin in a highly targeted fashion.
About Parkinson’s Disease
Parkinson’s disease is a progressive movement disorder that affects a million people in the United
States. Its main symptoms, stiffness, tremors and slowed movements and gait, are caused by a loss of
dopamine-containing nerve cells in the substantia nigra, which project their axons to the putamen.
Dopamine is a neurotransmitter involved in controlling movement and coordination, so Parkinson’s
patients exhibit a progressive inability to initiate and control physical movements. There is currently
no treatment that can reverse the degeneration of these neurons, let alone cure Parkinson’s disease.
Ceregene, Inc. is a San Diego-based biotechnology company focused on the delivery of nervous
system growth (neurotrophic) factors for the treatment of neurodegenerative and retinal disorders using
gene delivery. Ceregene’s clinical programs include CERE-110, an AAV2 based vector expressing
nerve growth factor (NGF) currently in a multi-center, controlled Phase 2 study for the treatment of
Alzheimer’s disease (in collaboration with the ADCS), and CERE-120 (AAV2-Neurturin) for
Parkinson’s disease. Ceregene was launched in January 2001. The company’s investors include Alta
Partners, MPM Capital, Hamilton BioVentures, Investor Growth Capital, California Technology
Partners and BioSante Pharmaceuticals (Nasdaq:BPAX) which acquired its position following its
merger with Cell Genesys, Inc. in October 2009.
About The Michael J. Fox Foundation
The Michael J. Fox Foundation for Parkinson’s Research is dedicated to finding a cure for Parkinson’s
disease through an aggressively funded research agenda and to ensuring the development of improved
therapies for those living with Parkinson’s today. The Foundation has funded over $205 million in
research to date.