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Cell Cure Neurosciences Initiates Phase I/IIa Trial and Granted Key US Patent for Retinal Pigment Epithelial (RPE) Stem Cells

Feb. 17, 2015

Hadasit Bio-Holdings Portfolio Company Cell Cure Neurosciences Initiates Phase I/IIa Trial of Lead Candidate OpRegen® for Treatment of Dry-AMD


Company Granted Key US Patent for Retinal Pigment Epithelial (RPE) Stem Cells


Jerusalem, Israel February 17, 2015 – Cell Cure NeuroSciences Ltd., a portfolio company of HBL Hadasit Bio-Holdings Ltd. (TASE: HDST, OTC: HADSY), today announced that it has received the necessary approvals and has initiated its previously announced Phase I / IIa clinical study of lead product candidate, OpRegen®, a unique cell therapy to for the treatment of dry age related macular degeneration (Dry-AMD). The trial will be conducted at Hadassah Ein Kerem Medical Center in Jerusalem, Israel, and is expected to begin enrolling patients immediately.


The goal of this trial is to prove safety and efficacy of OpRegen® in patients with Dry-AMD with geographic atrophy, and to examine the long-term effectiveness of the product in slowing disease progression and maintaining patient vision. The trial is a dose-escalation study, in which patients will receive a single transplant of one of three dose regimens of OpRegen® into the subretinal space of the eye. Following transplantation, patients will be followed for 12 months at specified intervals to assess the safety and tolerability of the product. At the conclusion of the 12 months, patients will continue to be monitored at longer intervals for an extended period. Cell Cure expects to enroll 15 patients, with no control group, with the last patient expected to be treated in the third quarter of 2016 and results of the initial 12 month follow-up available in the fourth quarter of 2017.


Tami Kfir, CEO of HBL, said, "We are thrilled with the progress that Cell Cure has made during the last several months, culminating in the initiation of this clinical trial of OpRegen® as a potential treatment of Dry-AMD, the most severe form of the disease. This trial represents the first preparation of xeno-free human embryonic stem cell-derived RPE cells evaluated for transplant therapy of dry-AMD. We believe that great potential exists in alternatives to animal cell-based treatments such as OpRegen®, and look forward to monitoring the progress of the trial as it moves forward.”


In addition, the Company announced that it has been awarded U.S. patent number 8,956,866, titled: "RETINAL PIGMENT EPITHELIAL CELLS DIFFERENTIATED FROM EMBRYONIC STEM CELLS WITH NICOTINAMIDE AND ACTIVIN A". The patent refers to the method for sorting of pluripotent cells (stem cells from the fetus) grunt pigment epithelial cells (RPE cells), and is fundamental to the technology developed by Cell Cure.


Ms. Kfir added, “This recognition by the U.S. Patent and Trademark Office is an important validation of Cell Cure’s technology, which we believe represents a potential breakthrough in the use of embryonic stem cells to develop novel therapies targeting areas with a high unmet medical need, such as dry AMD.”


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


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