Ceregene Reports Data From Parkinson's Disease Phase 2b Study

        Ceregene Reports Data From Parkinson's Disease Phase 2b Study

PR Newswire

SAN DIEGO, April 19, 2013

SAN DIEGO, April 19, 2013 /PRNewswire/ -- Ceregene, Inc. today announced the
top-line data from its double-blind, randomized, controlled Phase 2b clinical
study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver
the neurotrophic factor neurturin, for Parkinson's disease. The trial did
not demonstrate statistically significant efficacy on the primary endpoint
(UPDRS-motor off). However, one of the "key secondary endpoints" (Diary-off
score), as defined and prespecified in the Statistical Analysis Plan, did
produce statistically significant benefit. The trial also provided further
evidence for the safety of CERE-120 and the dosing methods employed. A marked
placebo effect was observed in this trial in that both the
sham-surgery-control patients and the CERE-120 treated patients showed
significant improvement following their surgery.

Fifty-one (51) patients with moderately advanced Parkinson's disease who could
not be satisfactorily controlled with conventional Parkinson's medication were
enrolled in the study at 11 leading clinical sites throughout the U.S.
Approximately half of the patients received CERE-120 while the other half
received sham (placebo) surgery as a control. Patients were monitored for
15-24 months to assess safety and changes in Parkinson's disease symptoms,
using multiple endpoints such as the Unified Parkinson's Disease Rating Scale
(UPDRS), Daily Diaries that assess motor function throughout the day, and
PDQ-39 (a measure of quality of life), among others. Ceregene continues to
analyze the data from this trial to gain as much information as possible.

Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene,
Inc. stated, "We are disappointed that we did not achieve broader statistical
significance in this small clinical trial, perhaps due in part to the marked
placebo effect noted above. That said, we at Ceregene want to acknowledge the
courage of all of the patients enrolled in this very important study and their
family members. We also would like to acknowledge the Michael J. Fox
Foundation for Parkinson's Research for their long-term support as we
continued the development of this novel neurotrophic factor-based treatment
with the potential to improve symptoms and also slow disease progression."

Raymond T. Bartus, Ph.D., executive vice president and chief scientific
officer of Ceregene stated: "While we did not achieve the degree of efficacy
we had hoped for in this trial, we are proud that our efforts have helped to
establish that gene therapy can provide the enabling technology to safely
deliver stable, long-term bioactive protein to targeted sites deep in the
human brain and that the Parkinson's disease brain is able to show a positive
response to neurotrophic factor stimulation. Hopefully, the information and
insight we achieved and shared with the biomedical community will aid in the
continuing effort to develop more effective therapies for many of these tragic
and dehumanizing neurodegenerative diseases."

C. Warren Olanow, M.D., Chairman Emeritus of the Department of Neurology and
Professor of Neuroscience at the Mount Sinai School of Medicine in New York
and a clinical advisor to Ceregene noted, "This was an extremely
well-conceived and designed study. It is unfortunate for patients that broader
benefits from this extremely promising therapy could not be demonstrated in
this clinical trial. These results illustrate how difficult it is to establish
clinical efficacy with entirely novel therapeutic approaches in complicated
neurological diseases. The Ceregene team, its scientific advisors and the
participating clinical sites are to be congratulated for that effort and the
flawless execution of this difficult scientific

Ceregene established a leadership position in the fields of gene therapy and
neurotrophic factors for the treatment of neurodegenerative diseases. A total
of over 100 patients have been safely dosed in two clinical programs: CERE-120
(AAV-NRTN) for Parkinson's disease and CERE-110 (AAV-NGF) for Alzheimer's
disease. A randomized, controlled Phase 2 study of CERE-110 for Alzheimer's is
continuing. It is fully enrolled and financially supported in large part by a
grant from the NIH, with top line data expected by late 2014. In addition to
the Parkinson's and Alzheimer's programs, Ceregene has conducted extensive
preclinical work with CERE-120 for Huntington's disease, as well as another
gene therapy/neurotrophic factor product (AAV-NT4) for blinding ocular
diseases (such as Retinitis Pigmentosa, macular degeneration, diabetic
retinopathy and glaucoma) and yet another (AAV-IGF1) for Lou Gehrig's disease
(ALS). Ceregene is currently looking at strategic alternatives to advance its
AAV gene therapy platform.

About CERE-120 and its Application to Treating Parkinson's Disease
CERE-120 is composed of a harmless 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
function. Neurturin is a member of the same protein family as glial cell
line-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. Degeneration of
these neurons is responsible for the major motor impairments of Parkinson's
disease. CERE-120 is 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, as well as the cell bodies for these same neurons,
located in a different area of the brain, called the substantia nigra. Once
CERE-120 is delivered to the brain, it provides stable, controlled and highly
targeted neurturin expression for years following a single injection,
confirmed in both animal and human studies.

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.

About Ceregene
Ceregene, Inc. is a San Diego-based biotechnology company focused on the
development of nervous system growth factors (neurotrophic factors) as
treatments for neurodegenerative and retinal disorders using gene transfer for
their delivery. The company has established a leadership position in the
fields of gene therapy and neurotrophic factors, having launched 6 separate
clinical trials in Parkinson's and Alzheimer's disease, enrolling a total of
nearly 200 patients, over 100 of whom have been administered the gene therapy
products, some several years ago, with no safety serious issues. Additionally,
the company has published 2 dozen peer-reviewed scientific papers describing
novel nonclinical and clinical findings. Ceregene's clinical program for
Alzheimer's disease involves CERE-110, an AAV2-based vector expressing nerve
growth factor (NGF). A fully enrolled multi-center, controlled Phase 2 study
with CERE-110 is ongoing, conducted in collaboration with the Alzheimer's
Disease Cooperative Study and partially funded by a grant from the National
Institutes of Health (NIH). 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).

About The Michael J. Fox Foundation for Parkinson's Research
As the world's largest private funder of Parkinson's research, The Michael J.
Fox Foundation is dedicated to accelerating a cure for Parkinson's disease and
improved therapies for those living with the condition today. The Foundation
pursues its goals through an aggressively funded, highly targeted research
program coupled with active global engagement of scientists, Parkinson's
patients, business leaders, clinical trial participants, donors and
volunteers. In addition to funding more than $325 million in research to
date, the Foundation has fundamentally altered the trajectory of progress
toward a cure. Operating at the hub of worldwide Parkinson's research, the
Foundation forges groundbreaking collaborations with industry leaders,
academic scientists and government research funders; increases the flow of
participants into Parkinson's disease clinical trials with its online tool,
Fox Trial Finder; promotes Parkinson's awareness through high-profile
advocacy, events and outreach; and coordinates the grassroots involvement of
thousands of Team Fox members around the world.

SOURCE Ceregene, Inc.

Website: http://www.ceregene.com
Contact: Jeffrey Ostrove, Ph.D., President and CEO, Ceregene, Inc.,
+1-858-458-8808, jostrove@ceregene.com
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