Ceregene Announces Promising Phase 1 Results From Gene Therapy Trial for
Parkinson's Disease
Michael J. Fox Foundation Awards $1.9 Million for Phase 2 Efficacy Study
SAN DIEGO, October 10, 2006 /PRNewswire/ -- Ceregene, Inc. announced today
that CERE-120, a gene therapy product in development for the treatment of
Parkinson's disease, was well tolerated and appeared to reduce symptoms by
approximately 40% (p<0.001), as measured by the Unified Parkinson's Disease
Rating Scale (UPDRS) motor "off" score, in an open-label Phase 1 study in 12
patients with advanced disease. Initial results of the study were presented
by William J. Marks Jr., M.D., principal investigator of the study and
associate professor of Neurology at the University of California, San
Francisco (UCSF) today at the American Neurological Association annual
meeting in Chicago.
The study was supported in part by a grant from The Michael J. Fox
Foundation for Parkinson's Research. Based on the initial results, the
Foundation today announced plans to partially fund a Phase 2 study with a
$1.9 million grant
"We were encouraged by the results of the Phase 1 trial," said Deborah W.
Brooks, president and CEO of The Michael J. Fox Foundation. "Based on these
and on the intriguing efficacy observations, we're eager to continue to
support research in Phase 2 that will more definitively assess the potential
of CERE-120 to treat PD."
CERE-120 is comprised of an adeno-associated virus (AAV) vector carrying the
gene for neurturin (NTN), a naturally occurring protein, whose role is to
keep dopamine-secreting neurons alive and functioning normally. All 12
patients enrolled in the study underwent stereotactic neurosurgery to
deposit CERE-120 into their putamen. The putamen is a region of the brain
that undergoes degeneration and reduced dopamine production in Parkinson's
disease patients and this has been closely linked to the major motor
deficits in these patients. All patients entered in the trial were judged to
have inadequate control of their disease with standard levodopa therapy and
were otherwise potential candidates for additional treatment interventions
such as deep brain stimulation (DBS) surgery.
CERE-120 was delivered at 2 different doses, with patients receiving the low
dose demonstrating approximately 40% improvement in UPDRS motor "off" scores
by 9 months and patients receiving the 4-fold higher dose showing a similar
effect 3 months sooner. Patients also demonstrated a 50% reduction in hours
of "off" time (i.e., time when normal Parkinson's medication was ineffective
and symptoms were troubling to the patient) and a doubling of good quality
"on" time without dyskinesias (i.e., time when a patient is functioning
well) according to self-reported diaries.
NTN (neurturin) is a member of the same protein family as glial cell-derived
neurotrophic factor (GDNF) and the two molecules have similar
pharmacological properties. GDNF has previously been tested in Parkinson's
disease patients. Ceregene owns exclusive technology and product rights to
CERE-120.
"Targeted delivery of the trophic factor neurturin is a compelling approach
to treating Parkinson's disease," said Dr. Marks. "The safety data and
preliminary efficacy data that we have seen in this Phase 1 study are
encouraging. Clearly, a larger-scale study is warranted."
According to Dr. Marks, existing treatments for Parkinson's disease treat
symptoms only, and for only a limited period of time. "Patients with
Parkinson's disease urgently need therapeutic approaches that not only
improve symptoms and function, but also have the ability to modify the
underlying disease itself in a favorable manner," he said.
In addition to Dr. Marks, the study was authored by: Jill Ostrem, M.D., UCSF
neurologist; Philip Starr, M.D., Ph.D. and Paul Larson, M.D., who conducted
the neurosurgery at UCSF; neurologist Leo Verhagen, M.D. with neurosurgeon
Roy Bakay, M.D. at Rush University Medical Center in Chicago; and Raymond T.
Bartus, Ph.D., who led the clinical and preclinical development of CERE-120
at Ceregene.
"The planned Phase 2 trial will be a randomized controlled trial involving
approximately 50 patients, and is designed to test if the efficacy we have
seen in our initial Phase 1 trial will hold up in a controlled study,"
stated Jeffrey M. Ostrove, Ph.D., president and CEO of Ceregene.
Eight medical centers will participate in the Phase 2 study: , Duke
University, Oregon Health Sciences University, University of Alabama at
Birmingham, University of Pennsylvania and Mount Sinai College of Medicine.
UCSF and Rush will also be participating.
"The Phase 1 data reported today affirms that the functioning of CERE-120
closely resembled its performance in preclinical studies both in terms of
its overall safety as well as its possible efficacy," noted Raymond T.
Bartus, Ph.D., Ceregene's chief operating officer. "The development of
growth factors as a treatment for neurodegenerative diseases has been
hampered by the difficulty of delivering them specifically to the targeted
areas that need their neuroprotective properties. We believe our programs
increasingly demonstrate that gene transfer may represent a safe and
effective means of solving this age-old problem," said Raymond Bartus.
About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the
development of gene therapies for neurodegenerative disorders. Ceregene is
in the clinic with CERE-110, an AAV2 based vector expressing nerve growth
factor that is being tested as a treatment for Alzheimer's disease, and with
CERE-120 for Parkinson's disease. CERE-130 is in late preclinical
development for ALS. Ceregene was launched in January 2001 and is a former
subsidiary of Cell Genesys, Inc. , which is headquartered in South San
Francisco, CA. Ceregene's investors include Alta Partners, MPM Capital and
Cell Genesys, as well as Hamilton BioVentures and California Technology
Partners.
About The Michael J. Fox Foundation
Founded in 2000, The Michael J. Fox Foundation for Parkinson's Research is
dedicated to ensuring the development of a cure for Parkinson's disease
within this decade through an aggressively funded research agenda. The
Foundation has funded approximately $80 million in research to date, either
directly or through partnerships.

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