Michael J. Fox Foundation Awards $1.9 Million for Phase 2 Efficacy Study
SAN DIEGO, Oct. 10 /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: Baylor
College of Medicine, 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. (NASDAQ: CEGE), 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.
Media Contacts: Ceregene; UCSF
Jeffrey M. Ostrove; Ceregene, Inc. Carol Hyman; UCSF
858-458-8808 415-476-2557
jostrove@ceregene.com chyman@pubaff.ucsf.edu
SOURCE Ceregene, Inc.
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Related links:
http://www.ceregene.com
CONTACT:
Jeffrey M. Ostrove of Ceregene, Inc.,
+1-858-458-8808, or jostrove@ceregene.com; or Carol Hyman of
UCSF, +1-415-476-2557, or chyman@pubaff.ucsf.edu
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