Gene therapy demonstrated stable, long-lasting expression of neurturin.
Ceregene closed a Series D preferred financing in the amount of $11.5 million. The funds will be used for Ceregene’s currently enrolling Phase IIb study of CERE-120 in Parkinson disease.
The financing comes on the back of Ceregene receiving three grants totaling $733,437 under the Qualifying Therapeutic Discovery Program. These funds were awarded to support CERE-120 as well as the currently enrolling Phase II study of CERE-110 in Alzheimer disease and the firm’s ocular disease program.
The current venture round was co-led by Hamilton BioVentures and Alta Partners with participation from MPM Capital and Investor Growth Capital. Kerry Dance, Ph.D., managing director of Hamilton BioVentures, will join Ceregene’s board of directors.
“The closing of our Series D financing coupled with our Therapeutic Tax Credit grant and recent support from The Michael J. Fox Foundation should provide us with the resources to complete our Phase IIb Parkinson disease clinical study,” states Jeffrey M. Ostrove, Ph.D., president and CEO of Ceregene.
“We are encouraged by the data we obtained in our last Phase II study in Parkinson disease, which was recently published in the online version of Lancet Neurology, and expect to complete the new Phase IIb clinical study by the end of 2012.”
Ceregene, a San Diego-based biotechnology company, is focused on the delivery of nervous system growth factors for the treatment of neurodegenerative and retinal disorders using gene delivery.
“Because nervous system growth factors have the ability to restore dying neurons in the brain, keeping them alive and functioning better, these novel therapeutics should improve symptoms as well as have the ability to slow disease progression, making them unique among developing therapies,” states Dr. Dance.
CERE-120 is composed of an adeno-associated virus (AAV) vector carrying the gene for neurturin. This naturally occurring protein is reportedly known to repair damaged and dying dopamine-secreting neurons.
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 dopamine neurons in the substantia nigra that degenerate in Parkinson disease, Ceregene notes. Degeneration of these neurons is responsible for the major motor impairments of Parkinson disease.
In previous clinical trials, CERE-120 was delivered to the terminal fields, located in an area of the brain called the putamen, using stereotactic injections. The cell bodies for these same neurons are located in the substantia nigra.
The Phase IIb trial has been amended such that CERE-120 will be administered to both the substantia nigra as well as the putamen. Thus far, the drug has shown to provide stable, long-lasting expression of neurturin in a highly targeted fashion once delivered to the brain.
CERE-110, Ceregene’s candidate for Alzheimer disease, comprises an AAV vector carrying the gene for NGF. This protein maintains survival of nerve cells in the brain. CERE-110 is surgically injected into the nucleus basalis of meynert, a brain region where cholinergic cell degeneration occurs in Alzheimer disease.
The cholinergic system is important in memory and cognitive function. A restoration in the function of this system may improve memory in individuals with Alzheimer’s disease, Ceregene points out.
A Phase II randomized, controlled study is currently enrolling patients with mild-to-moderate Alzheimer disease in 10 centers across the U.S. This study is being conducted in collaboration with the ADCS (Alzheimer’s Disease Cooperative Study) and is also supported by a $5.4 million NIH grant.
Ceregene’s eye disease program consists of three preclinical compounds. CERE-140 is in development as a therapy for retinitis pigmentosa. It is made of an AAV vector carrying the gene for NT4. It is also being researched for the treatment of macular degeneration and glaucoma.