ALAMEDA, Calif. & JERUSALEM--(BUSINESS WIRE)--BioTime, Inc. (NYSE MKT: BTX) and its subsidiary Cell Cure Neurosciences Ltd. (Cell Cure) today provided an update on Cell Cures product development and partnering activities.
On February 16, 2015, Cell Cure opened the clinical trial of OpRegen titled Phase I/IIa Dose Escalation Safety and Efficacy Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelium Cells Transplanted Subretinally in Patients with Advanced Dry-Form Age-Related Macular Degeneration with Geographic Atrophy at Hadassah University Medical Center in Jerusalem, Israel. Patient enrollment is expected to begin shortly. OpRegen consists of animal product-free retinal pigment epithelial (RPE) cells with high purity and potency.
On October 31, 2014, the United States Food and Drug Administration (FDA) cleared Cell Cure's Investigational New Drug (IND) application to initiate the clinical trial of OpRegen in patients with the severe form of age-related macular degeneration (AMD) with geographic atrophy (GA). While treatment options exist for the treatment of the wet form of AMD, it amounts to only about 10% of the disease prevalence. There is currently no FDA-approved therapy for the dry form occurring in approximately 90% of those afflicted with AMD. Cell Cure intends to transplant OpRegen as a single dose into the subretinal space of patients eyes in order to test the safety and efficacy of the product in this leading cause of blindness.
The Phase I/IIa clinical trial, will evaluate three different dose regimens of OpRegen. Following transplantation, the patients will be followed for 12 months at specified intervals, to evaluate the safety and tolerability of the product. Following the initial 12 month period, patients will continue to be monitored at longer intervals for an additional period of time. A secondary objective of the clinical trial will be to examine the ability of transplanted OpRegen to engraft, survive, and moderate disease progression in the patients. In addition to thorough characterization of visual function, a battery of ophthalmic imaging modalities will be used to quantify structural changes and rate of GA expansion.
Cell Cure also announced today that the option granted to Teva Pharmaceutical Industries Ltd. (Teva) under a Research and Exclusive Option Agreement of October 7, 2010 to license-in rights to its OpRegen product has expired without having been exercised by Teva. Cell Cure will therefore be continuing the clinical development of OpRegen on its own and pursuing discussions with other potential strategic partners, including those that have already indicated interest in participating in development and commercialization of the product.
Cell Cure also announced that US patent No. 8,956,866 relating to a proprietary method of manufacturing RPE cells (the active ingredient of OpRegen) is expected to issue on February 17, 2015. This patent combined with other patents and patent applications in the BioTime family of companies provides significant patent protection for this novel therapeutic modality for AMD.
The large markets currently associated with therapies for the wet form of AMD combined with the elegance of RPE replacement therapy for the larger unmet needs associated with the dry form, highlights why Cell Cure has prioritized the development of this product, said Dr. Charles Irving, CEO of Cell Cure. We look forward to initiation of the trials and providing updates in the coming months.
About Age-Related Macular Degeneration
Age-related macular degeneration (AMD) is one of the major diseases of aging and is the leading eye disease responsible for visual impairment of older persons in the US, Europe and Australia. AMD affects the macula, which is the part of the retina responsible for sharp, central vision that is important for facial recognition, reading and driving. There are two forms of AMD. The dry form (dry-AMD) advances slowly and painlessly but may progress to geographic atrophy (GA) in which RPE cells and photoreceptors degenerate and are lost. Once the atrophy involves the fovea (the center of the macula), patients lose their central vision and may develop legal blindness. There are about 1.6 million new cases of dry-AMD in the US annually, and as yet there is no effective treatment for this condition. About 10% of patients with dry-AMD develop wet (or neovascular) AMD, the second main form of this disease, which usually manifests acutely and can lead to severe visual loss in a matter of weeks. Wet-AMD can be treated with currently-marketed VEGF inhibitors. However, such products typically require frequent repeated injections in the eye, and patients often continue to suffer from continued progression of the underlying dry-AMD disease process. Current estimated annual sales of VEGF inhibitors for the treatment of the wet form of AMD are estimated to be in excess of $5 billion worldwide. The root cause of the larger problem of dry-AMD is believed to be the dysfunction of RPE cells. One of the most exciting therapeutic approaches to dry-AMD is the transplantation of healthy, young RPE cells to support and replace the patients old degenerating RPE cells, which may prevent progression of the atrophy as well as the development of wet-AMD. Pluripotent stem cells, such as hESCs, can provide an unlimited source for the derivation of such healthy RPE cells for transplantation.
About OpRegen
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Cell Cure Neurosciences Ltd. Provides Update on its Product Development and Partnering Activities
