SAN DIEGO, Nov. 26 /PRNewswire/ -- Ceregene, Inc., a biopharmaceutical
company, today reported clinical data from a double-blind, controlled Phase 2
trial of CERE-120 in 58 patients with advanced Parkinson's disease. The trial
did not demonstrate an appreciable difference between patients treated with
CERE-120 versus those in the control group. Both groups showed an approximate
7 point improvement in the protocol-defined primary endpoint (Unified
Parkinson's Disease Rating Scale- motor off score at 12 months), relative to a
mean at baseline of approximately 39 points. Both groups had a substantial
number of patients who demonstrated a meaningful clinical improvement from
baseline. CERE-120 appeared to be safe and well tolerated.
"On behalf of everyone at Ceregene, I would like to personally express my
gratitude to all the patients, physicians and staff who participated in this
study. We share their disappointment. We also offer our heartfelt
appreciation to the many talented and dedicated employees at Ceregene who
continue to work extremely hard on this and Ceregene's other programs," stated
Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene.
"We continue to believe in the multiple opportunities in our pipeline that
target a range of serious neurodegenerative and ocular diseases."
"The medical research community has for several decades attempted to use
the unique healing properties of neurotrophic factors to treat serious
neurodegenerative diseases, and Ceregene's use of gene therapy as an essential
enabling technology to translate this idea into an effective human therapeutic
has consistently generated strong empirical support," stated Raymond T.
Bartus, Ph.D., Ceregene's executive vice president and chief scientific
officer. "Thus, we are stunned by the results of this trial and will continue
to analyze the data in order to gain greater insight into the factors that may
have contributed to this negative outcome, not only to build upon this insight
for our Parkinson's program, but also to help assure continued successful
development of our product candidates for other diseases."
About Phase 2 Trial of CERE-120
Ceregene's Phase 2 trial was a double-blind, controlled clinical trial
that completed enrollment of 58 patients with advanced Parkinson's disease in
November 2007. This study was launched after successful execution of an
extensive nonclinical program and preliminary evidence of safety and efficacy
in advanced Parkinson's patients via an open-label Phase 1 trial in 12
patients. Patients in the Phase 2 trial were enrolled across nine leading
academic medical centers in the United States, with two thirds of patients
receiving CERE-120 and one third enrolled into a control group. Patients
received a single administration of CERE-120 via stereotactic neurosurgery to
deliver the drug into the putamen region of the brain and were followed for 12
months for safety and efficacy.
CERE-120 is composed of an adeno-associated virus (AAV) vector carrying
the gene for neurturin (NTN), a naturally occurring protein known to repair
damaged and dying dopamine-secreting neurons, keeping them alive and restoring
normal function. NTN 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 midbrain
dopamine neurons that degenerate in Parkinson's disease and are responsible
for the major motor impairments. CERE-120 is delivered by stereotactic
injection to the affected area of the brain, providing stable, long-lasting
expression of NTN in a highly targeted fashion. Genzyme Corporation has
licensed the ex-North American rights for the development and
commercialization of CERE-120 from Ceregene, an agreement that was announced
in June 2007. Ceregene gratefully acknowledges the financial support received
from the Michael J. Fox Foundation for Parkinson's Research to help defray
some of the costs of the CERE-120 Phase 1 and Phase 2 clinical trials.
About Parkinson's Disease
Parkinson's disease is a progressive movement disorder that affects a
million people in the United States. Its main symptoms are caused by a
reduction in dopamine containing nerve cells of the midbrain. 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 cure for Parkinson's disease.
Ceregene, Inc. is a San Diego-based biotechnology company focused on the
delivery of nervous system growth factors for the treatment of
neurodegenerative disorders using gene delivery. Ceregene's clinical programs
include CERE-110, an AAV2 based vector expressing nerve growth factor that is
about to enter a Phase 2 study for the treatment of Alzheimer's disease, and
CERE-120 (AAV2-Neurturin). CERE-135 and CERE-140 are in preclinical
development for ALS (Lou Gehrig's disease) and ocular disorders, respectively.
Ceregene was launched in January 2001. The company's investors include Alta
Partners, MPM Capital, Investor Growth Capital and Cell Genesys, Inc.
(Nasdaq: CEGE) as well as Hamilton BioVentures and California Technology
SOURCE Ceregene, Inc.