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 Cure’s 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 patient’s 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®
Cell Cure's OpRegen® consists of RPE cells that are produced using a proprietary process that drives the differentiation of human embryonic stem cells into high purity RPE cells. OpRegen is also “xeno-free," meaning that no animal products were used either in the derivation and expansion of the human embryonic stem cells or in the directed differentiation process. The avoidance of the use of animal products eliminates some safety concerns. OpRegen is formulated as a suspension of RPE cells. Preclinical studies in mice have shown that following a single subretinal injection of OpRegen, the cells can rapidly organize into their natural monolayer structure and survive throughout the lifetime of the animal. OpRegen is designed to be an “off-the-shelf” allogeneic product provided to retinal surgeons in a final formulation ready for transplantation. Unlike treatments that require multiple, frequent injections into the eye, such as currently-marketed products like Lucentis and Eylea for wet-AMD, it is expected that OpRegen would be administered in a single procedure.
About Cell Cure Neurosciences Ltd.
Established in 2005, Cell Cure is located in Jerusalem, Israel on the campus of Hadassah Medical Center. Cell Cure's mission is to become a leading supplier of human cell-based therapies for the treatment of retinal and neural degenerative diseases. Its technology platform is based on the manufacture of diverse cell products sourced from clinical-grade (GMP-compatible) human embryonic stem cells. Its current focus is the development of retinal pigment epithelial (RPE) cells for the treatment of age-related macular degeneration. Cell Cure's major shareholders include BioTime, Inc. (NYSE MKT: BTX), HBL Hadasit Bio-Holdings Ltd., Teva Pharmaceuticals Industries Ltd., and Asterias Biotherapeutics,Inc. Additional information about Cell Cure can be found on the web at www.cellcureneurosciences.com.
About BioTime
BioTime is a biotechnology company engaged in research and product development in the field of regenerative medicine. Regenerative medicine refers to therapies based on stem cell technology that are designed to rebuild cell and tissue function lost due to degenerative disease or injury. BioTime’s focus is on pluripotent stem cell technology based on human embryonic stem (“hES”) cells and induced pluripotent stem (“iPS”) cells. hES and iPS cells provide a means of manufacturing every cell type in the human body and therefore show considerable promise for the development of a number of new therapeutic products. BioTime’s therapeutic and research products include a wide array of proprietary PureStem® progenitors, HyStem® hydrogels, culture media, and differentiation kits. Renevia™ (a HyStem product), is now in a pivotal trial in Europe as a biocompatible, implantable hyaluronan and collagen-based matrix for cell delivery in the treatment of HIV-related lipoatrophy. In addition, BioTime has developed Hextend®, a blood plasma volume expander for use in surgery, emergency trauma treatment and other applications. Hextend is manufactured and distributed in the U.S. by Hospira, Inc. and in South Korea by CJ HealthCare Corporation, under exclusive licensing agreements.
BioTime is also developing stem cell and other products for research, therapeutic, and diagnostic use through its subsidiaries:
- Asterias Biotherapeutics, Inc. is developing pluripotent stem-cell based therapies in neurology and oncology, including AST-OPC1 oligodendrocyte progenitor cells in spinal cord injury, multiple sclerosis and stroke, and AST-VAC2, an allogeneic dendritic cell-based cancer vaccine. Asterias Series A common stock is traded on the NYSE MKT under the symbol AST.
- BioTime Asia, Ltd., a Hong Kong company, may offer and sell products for research use for BioTime’s ESI BIO Division.
- Cell Cure Neurosciences Ltd. is an Israel-based biotechnology company focused on developing stem cell-based therapies for retinal and neurological disorders. OpRegen® is currently in a Phase I/IIa clinical trial for the treatment of the dry-form of age-related macular degeneration.
- ESI BIO is the research and product marketing division of BioTime, providing stem cell researchers with products and technologies to enable them to translate their work into the clinic, including PureStem® progenitors and HyStem® hydrogels.
- LifeMap Sciences, Inc. markets, sells, and distributes GeneCards®, the leading human gene database, as part of an integrated database suite that also includes the LifeMap Discovery® database of embryonic development, stem cell research, and regenerative medicine, and MalaCards, the human disease database.
- LifeMap Solutions, Inc. is a subsidiary of LifeMap Sciences focused on developing mobile health (mHealth) products.
- OncoCyte Corporation is developing products and technologies to diagnose and treat cancer, including PanC-Dx™, with four clinical studies currently underway.
- OrthoCyte Corporation is developing therapies to treat orthopedic disorders, diseases and injuries.
- ReCyte Therapeutics, Inc. is developing therapies to treat a variety of cardiovascular and related ischemic disorders, as well as products for research using cell reprogramming technology.
BioTime common stock is traded on the NYSE MKT under the symbol BTX. For more information, please visit www.biotimeinc.com or connect with the company on Twitter, LinkedIn, Facebook, YouTube, and Google+.
Forward-Looking Statements
Statements pertaining to future financial and/or operating results, future growth in research, technology, clinical development, and potential opportunities for BioTime and its subsidiaries, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as “will,” “believes,” “plans,” “anticipates,” “expects,” “estimates”) should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of BioTime and its subsidiaries, particularly those mentioned in the cautionary statements found in BioTime's Securities and Exchange Commission filings. BioTime disclaims any intent or obligation to update these forward-looking statements.
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