San Diego, CA–April 28, 2010— Ceregene, Inc., a biopharmaceutical company, announced today that
enrollment is proceeding in a new Phase 1/2 clinical study evaluating CERE-120, a gene therapy
product which delivers the neurotrophic factor neurturin, to dying neurons in Parkinson’s disease
patients. This new clinical study follows a completed Phase 2 trial and builds on experience gained in
that trial, by enhancing the dosing regimen and optimizing the duration of patient follow up.
The new study was initiated in the fourth quarter of 2009 and is proceeding as planned. The ongoing
Phase 1/2 trial will enroll six patients with advanced Parkinson’s disease. The first four patients have
been treated safely and two additional patients will be treated over the next two months. This Phase 1
open label portion of the trial is being conducted by Drs. Mark Stacy and Dennis Turner at Duke
University School of Medicine, Drs. Michele Tagliati and Ron Alterman at Mount Sinai Medical
Center, New York, and Drs. Stewart Factor and Nicholas Boulis at Emory University Hospital.
Ceregene expects to initiate the next phase of this trial (a sham-surgery-controlled, double-blind Phase
2 portion) beginning during the third quarter of this year. The Phase 2 portion will include 10 leading
US neurological medical centers.
The first Phase 2 clinical trial of CERE-120 initially reported in November 2008, did not meet its predesignated
primary endpoint (UPDRS or Unified Parkinson’s Disease Rating Scale- Motor Off, at 12
months) although modest benefit was seen in improving motor performance in Parkinson’s disease
patients, based on several secondary endpoints. Even greater improvement was seen following an
analysis of all patients who were assessed under blinded conditions at 15 to 18 months post-treatment,
including on the primary measure (UPDRS motor off [p=0.025] at 18 months). Importantly, new
scientific insight regarding degenerating dopamine neurons in the brains of advanced Parkinson’s
disease gained from two CERE-120-treated patients who died of unrelated causes, combined with the
clinical results, led to important changes in CERE-120 dosing that may significantly enhance its
bioactivity and response to treatment. This revised dosing paradigm now targets both the terminals (or
nerve endings) of the degenerating neurons in a site in the brain called the putamen, as well as the cell
bodies of these neurons, located in another region called the substantia nigra.
“We are pleased to report on progress being made in the development of CERE-120,” stated Jeffrey M.
Ostrove, Ph.D., president and chief executive officer of Ceregene. “We continue to strongly believe
that CERE-120 has the potential to improve the symptoms of Parkinson’s disease while also delaying
further disease progression, and may therefore represent a significant advancement in the treatment of
patients for whom existing treatments inevitably fail as their disease progresses.”
“We are optimistic that the improved dosing method used in the current trial will assure that adequate
neurturin protein is expressed throughout the degenerating nigrostriatal system and significantly
enhance the biological effects and clinical benefit of CERE-120,” stated Raymond T. Bartus, Ph.D.,
Ceregene’s executive vice president and chief scientific officer.
“We are pleased that we have observed no safety issues with the revised CERE-120 dosing procedure,
to date, and that our investigators have been able to implement the dosing protocol with no apparent
complications or difficulty,” added Joao Siffert, M.D., vice president and chief medical officer at
Ceregene. “We look forward to completing the Phase 1 safety portion and proceeding to the Phase 2
effectiveness portion of developing CERE-120.”
About CERE-120 and its Potential for Treating Parkinson’s Disease
CERE-120 is composed of an 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 normal function. Neurturin 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. Degeneration of these neurons is responsible for the major motor impairments of Parkinson’s
disease. CERE-120 has been 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. The cell bodies for these
same neurons are located in a different area of the brain, called the substantia nigra, and the amended
dosing regimen being employed in the ongoing Phase 1 trial calls for administration of CERE-120 to
both the substantia nigra as well as the putamen. Once CERE-120 is delivered to the brain, it provides
stable, long-lasting expression of neurturin in a highly targeted fashion. As has been announced, the
previous Phase 2 trial of CERE-120 involved 58 patients and did not demonstrate a difference between
CERE-120 and control in the primary endpoint at 12 months. However, CERE-120 did show
improvement on several secondary endpoints at 12 months, and importantly at 18 months CERE-120
demonstrated a statistically significant treatment effect on the primary endpoint. Based on those
findings, and insight gained from analyses of post-mortem brain tissue from two CERE-120 treated
patients, the company revised the dosing regimen which is reflected in the current Phase 1 trial, as well
as the planned Phase 2 trial.
About Parkinson’s Disease
PD 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 dopaminecontaining
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 delivery of nervous
system growth (neurotrophic) factors for the treatment of neurodegenerative and retinal disorders using
gene delivery. Ceregene’s clinical programs include CERE-110, an AAV2 based vector expressing
nerve growth factor currently in a multi-center, controlled Phase 2 study for the treatment of
Alzheimer’s disease, and CERE-120 (AAV2-Neurturin) for Parkinson’s disease. 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 BioSante Pharmaceuticals (Nasdaq:BPAX)
which acquired its position following its merger with Cell Genesys, Inc. in October 2009, as well as
Hamilton BioVentures and California Technology Partners.
