San Diego, CA and Cambridge, MA - June 21, 2007 –Ceregene, Inc. and Genzyme Corporation (Nasdaq: GENZ) today announced that they have entered into a partnership for the development and commercialization of CERE-120, Ceregene’s proprietary lead program for the treatment of Parkinson’s disease.
Under the terms of the agreement, Genzyme will pay Ceregene a $25 million up-front payment in exchange for certain partnership-related expenses. Ceregene will also be entitled to development-related milestone payments of up to $125 million and 50 percent reimbursement of the worldwide Phase 3 development costs. Genzyme will gain marketing rights in all markets outside of the US and Canada, and Genzyme will pay Ceregene tiered royalties based on sales in markets where it has rights. Ceregene will retain exclusive rights to CERE-120 in the US and Canada.
“We are pleased to be partnering CERE-120 with Genzyme—a company with a strong international presence and marketing expertise that will enable us to maximize the worldwide commercialization of CERE-120,” stated Jeffrey M. Ostrove, Ph.D., president and chief executive officer of Ceregene. “CERE-120, which is currently undergoing a controlled Phase 2 trial in the United States that is being partially funded by the Michael J. Fox Foundation, has demonstrated strong clinical and preclinical data to date and may have the ability to both improve Parkinson’s disease symptoms and slow the disease progression. The resources provided by our new partnership with Genzyme, together with the greater than $27 million initial closing of our Series C financing, which was led by Investor Growth Capital and closed in the first quarter, provides the funding we currently need to advance CERE-120 toward commercialization.”
“We are very enthusiastic about the potential that CERE-120 may have to improve the lives of people with Parkinson’s disease, which currently affects at least one million Americans,” said David Meeker, president, Lysosomal Storage Disorders unit, Genzyme Corporation. “We look forward to helping further the development of this exciting approach, building on our long-term commitment to gene therapy and neurodegenerative diseases. This program – which is focused on preserving neuronal function – will complement our existing Parkinson’s clinical trial that is currently underway.”
“We look forward to working with Genzyme to accelerate the development of CERE-120 in Europe and other areas outside North America,” stated Raymond T. Bartus, Ph.D., Ceregene’s senior vice president of clinical and preclinical R&D and chief operating officer, as well as chairman of the partnership’s joint steering committee. “Strong synergies and complementary skills and experience exist between Ceregene and Genzyme. Their deep infrastructure in developing and commercializing innovative therapeutic products and their experience working with international regulatory agencies will be invaluable as we expand the development of CERE-120.”
In a complementary program, Genzyme is conducting a separate Phase 1-2 clinical trial of a gene therapy for Parkinson's disease designed to restore the therapeutic effectiveness of levo-Dopa by enhancing the brain’s ability to convert it into dopamine. In addition, Genzyme has an extensive gene therapy portfolio that includes two additional ongoing clinical trials.
“These data add to the accumulating evidence from our studies in both humans and animals that neurotrophic factors can be delivered safely and effectively using gene transfer, thus providing the potential to restore function and slow further degeneration in many different neurodegenerative diseases,” added Raymond T. Bartus, Ph.D., Ceregene’s senior vice president of clinical and preclinical R&D and chief operating officer. “These results in Alzheimer’s patients, wherein we target degenerating cholinergic neurons, builds upon on the research of the past 30 years, showing that improving the function of these neurons can significantly reduce the memory loss in this disease”.
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.
About CERE-120 Clinical Trials
Ceregene is currently conducting a Phase 2 clinical trial, which is in the process of enrolling 51 patients with advanced Parkinson’s disease at nine medical centers in the United States. In a Phase 1 trial, CERE-120 demonstrated 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 the study. The 12-month results were presented at the American Association of Neurological Surgeons (AANS) annual meeting in April 2007.
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 amyotrophic lateral sclerosis (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’s investors include Alta Partners, MPM Capital, Investor Growth Capital and Cell Genesys, as well as Hamilton BioVentures and California Technology Partners.
One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 9,000 employees in locations spanning the globe and 2006 revenues of $3.2 billion. Genzyme has been selected by FORTUNE as one of the “100 Best Companies to Work for” in the United States.
With many established products and services helping patients in nearly 90 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as immune disease, infectious disease, and other areas of unmet medical need.