SAN DIEGO, Sept. 21 /PRNewswire-FirstCall/ -- Ceregene announced today
that patient treatment is underway in its Phase I study of CERE-120 to treat
Parkinson's disease. The study is being conducted at the University of
California, San Francisco Medical Center and Rush University Medical Center in
Chicago. CERE-120 is a novel gene therapy product that delivers the neurturin
(NTN) gene via an adeno-associated virus (AAV) type2 vector delivery system.
The naturally occurring NTN gene encodes the NTN protein that maintains
survival of dopamine-producing nerve cells that are required for normal bodily
movement and are the nerves that degenerate in Parkinson's disease patients.
Neurturin is a member of the same protein family as GDNF and they have similar
pharmacological properties. GDNF has previously been tested in Parkinson's
disease patients. Ceregene owns exclusive technology and product rights to
"CERE-120 has the potential to significantly slow or even halt the
progression of symptoms of this devastating disease," said Jeffrey M. Ostrove,
Ph.D., president and chief executive officer of Ceregene. "Numerous studies
in animal models, including the most widely accepted animal models of
Parkinson's disease, consistently demonstrated that this NTN gene therapy
product may be able to improve symptoms as well as retard the progression of
the disease. These studies also demonstrate that CERE-120 is also safe and
well tolerated in animals at doses hundreds of times higher than the
equivalent doses being tested in humans. We are pleased that human clinical
studies of our CERE-120 product are now under way."
This study is being led by William J. Marks, Jr., M.D., a neurologist at
the University of California, San Francisco (UCSF). Dr. Philip Starr, a
neurosurgeon at UCSF, performs the surgery. UCSF investigators also include
neurologist Jill Ostrem, M.D. and neurosurgeon Paul Larson, M.D. In addition,
neurologist Leo Verhagen, M.D, Ph.D. and neurosurgeon Roy Bakay, M.D. are
conducting the study at Rush University Medical Center in Chicago.
This Phase I study involves in vivo (inside the body) gene therapy
delivery of NTN. Ceregene's in vivo gene therapies utilize a non-replicating
viral vector to deliver therapeutic genes to the nervous system, where they
express proteins with the ability to prevent neurodegeneration and restore
normal nerve function. The adeno-associated viral (AAV) vector, which has
never been causally associated with disease in humans, is an important gene
delivery tool that has demonstrated ability to efficiently and stably express
genetic information in non-dividing target cells, such as the neurons
degenerating in Parkinson's disease, and to potentially achieve long-term,
therapeutic benefit. The goal of this study is to determine the safety and
efficacy of this treatment. Efficacy will be measured by standardized tests
for Parkinson's patients, as well as brain imaging studies.
"Parkinson's disease represents a major unmet medical need, in that
current therapies are only useful for a limited time, for eventually their
side effects become debilitating," stated Raymond T. Bartus, Ph.D., Ceregene's
senior vice president and chief operating officer. "Moreover, no currently
available treatment is able to slow the progression of the disease, as we hope
will be possible with CERE-120, based on our animal studies." Dr. Bartus
further stated, "We are pleased that we have been able to develop two
different clinical products and have now initiated clinical programs for two
serious neurodegenerative diseases within a one-year timeframe." Ceregene
also has an active clinical program in Alzheimer's disease (CERE-110 or
AAV-NGF) and has recently completed enrollment in a Phase I clinical study at
Rush University Medical Center in Chicago.
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 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 closed a $32M Series B financing in August 2004 that was co-led
by Alta Partners and MPM Capital.
Contact: Jeffrey M. Ostrove, Ph.D.
Chief Executive Officer
SOURCE Ceregene, Inc.