Ceregene Presents Long Term Follow-Up Data From Phase 1 Trial ofCERE-120 Demonstrating Improved Motor Function in Parkinson'sPatientsEncouraging Results Prompted Phase 2 Study Now Underway at Nine Centers
SAN DIEGO, Calif., April 16, 2007 /PRNewswire/ -- Ceregene, Inc., a biopharmaceutical company, today presented long term follow-up data from a Phase 1 clinical trial of CERE-120, a gene therapy product in development for the treatment of Parkinson's disease, demonstrating a 36 percent (p<0.001) reduction in Parkinson's symptoms at 12 months after administration, as measured by the Unified Parkinson's Disease Rating Scale (UPDRS) motor "off" score ("motor off" meaning patients were off Parkinson's medication at evaluation time). CERE-120 was also shown to be well tolerated in this study. The 12-month results were presented today by Philip Starr, M.D., Ph.D., associate professor of neurosurgery at the University of California, San Francisco at the American Association of Neurological Surgeons (AANS) annual meeting in Washington D.C. These data were also highlighted by the AANS in a specific CERE-120 press release issued by the Association.
The Phase 1 trial was an open-label study conducted in 12 patients with advanced Parkinson's disease at two clinical trial sites -- University of California, San Francisco and Rush University Medical Center in Chicago. All 12 patients enrolled in the study underwent stereotactic neurosurgery to deliver CERE-120 into their putamen -- a region of the brain affected by the degeneration of neurons in Parkinson's disease.
CERE-120 was delivered at two different doses, and patients receiving the low dose demonstrated approximately 36 percent improvement in UPDRS motor "off" scores by nine months and patients receiving the four-fold higher dose showed a similar effect three months sooner. This statistically significant, 36 percent improvement was maintained in both groups at the 12-month mark, the final follow-up time point in the study. Patients also demonstrated a 50 percent 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.
This Phase 1 clinical trial was partially supported by a grant from The Michael J. Fox Foundation for Parkinson's Research. Based on the results of the Phase 1 study, the Foundation has provided a $1.9 million grant, which will provide partial funding for Ceregene's ongoing Phase 2 trial.
"We are pleased with the results of this early study which suggests that the majority of patients treated with CERE-120 may have exhibited significant and stable improvement for a full year after receiving a single administration of CERE-120," stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. "Each of our products, much like CERE-120, is aimed at unmet neurodegenerative diseases including Parkinson's disease, Alzheimer's disease and Amyotrophic Lateral Sclerosis (AML or Lou Gehrig's disease) with the goal of not only improving the symptoms but also preventing progression of the disease."
"Through stereotactic surgery, we are able to administer CERE-120 in a highly targeted fashion to one area of the brain that Parkinson's disease affects, and we were able to do this safely," stated Dr. Starr.
"Given the encouraging data from the Phase 1 trial of CERE-120 in Parkinson's disease, we are conducting a follow-on Phase 2 clinical trial that is currently enrolling patients at nine clinical trial sites in the United States," said Raymond T. Bartus, Ph.D., Ceregene's senior vice president of clinical and preclinical R&D and chief operating officer. "The data from the Phase 1 trial are reflective of the impressive results we gathered from preclinical studies, which demonstrated the ability of CERE-120 to stimulate the survival and improve the function of key neuronal cells affected by Parkinson's disease, as well as an excellent safety profile over a wide range of CERE-120 dose levels."
About Phase 2 Trial of CERE-120 Currently Underway
A double-blind, controlled Phase 2 clinical trial is now enrolling 51 patients with advanced Parkinson's disease at nine medical centers in the United States, with two thirds of patients being enrolled in the active treatment group and one third in the control group. Patients are receiving CERE-120 via stereotactic neurosurgery to deliver the drug into the putamen region of the brain. Patients will be 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 functioning normally. 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. Ceregene owns exclusive technology and product rights to CERE-120.
About Parkinson's disease
Parkinson's disease is a progressive movement disorder that affects at least one million people in the United States. It is 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 experiencing a reduction of dopamine-producing nerve cells exhibit the 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 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. , which is headquartered in South San Francisco, CA. Ceregene's investors include Alta Partners, MPM Capital, Investor Growth 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 more than $90 million in research to date, either directly or through partnerships.
Posted: April 2007