BioTimes Subsidiary Cell Cure Neurosciences Receives FDA Authorization to Initiate Phase I/IIa Trial of Embryonic …

OpRegen consists of animal product-free retinal pigment epithelial (RPE) cells with high purity and potency that were derived from human embryonic stem cells (hESCs). Cell Cure will conduct the trial in Israel where OpRegen will be transplanted as a single dose into the subretinal space of the eye to test the safety and efficacy of the product. Patient enrollment is expected to begin in 2014 following approval of the trial by the Israel Ministry of Health.

About the OpRegenClinical Trial

Cell Cures Phase I/IIa clinical trial is a dose escalation safety and preliminary efficacy study of hESC-derived Retinal Pigment Epithelial (RPE) cells transplanted subretinally in patients with advanced dry-form AMD called geographic atrophy. The open-label, single center, nonrandomized trial will evaluate three different dose regimens of 50,000 to 500,000 cells. A total of 15 patients will be enrolled. The patients will be 55 years of age and older, with non-neovascular (dry-AMD) who have funduscopic findings of GA in the macula with absence of additional concomitant ocular disorders. The eye most affected by the disease will be treated with the contralateral eye being the control. Following transplantation, the patients will be followed for 12 months at specified intervals, to evaluate the safety and tolerability of OpRegen. A secondary objective of the clinical trial will be to examine the ability of transplanted OpRegen to engraft, survive, and induce changes in visual acuity. In addition to thorough characterization of visual function, a battery of defined ophthalmic imaging modalities will be used to quantify structural changes and rate of GA expansion. The study will be performed at Hadassah Ein Kerem Medical Center in Jerusalem, Israel.

The FDAs acceptance of our IND for the Phase I/IIa trial of OpRegen is a significant milestone for our company, and in the broader development of therapies based on human embryonic stem cells for the treatment of major diseases, said Benjamin Reubinoff, MD, PhD, Chief Scientific Officer of Cell Cure and Chairman of Obstetrics and Gynecology and Director of the Hadassah Human Embryonic Stem Cell Research Center at Hadassah Medical Center, Jerusalem, Israel. We look forward to initiating this first-of-its-kind study, and to continuing the clinical development of OpRegen.

Cell Cures Phase I/IIa study of OpRegen has been designed to provide preliminary, objective functional and structural data on the ability of hESC-RPE cell transplantation to slow the progression of geographic atrophy, in addition to safety data, added Prof. Eyal Banin, Head of the Center for Retinal and Macular Degenerations at the Department of Ophthalmology of Hadassah University Medical Center, Jerusalem, Israel who together with Prof. Reubinoff helped develop this novel treatment over the last decade. We are truly excited that this unique, hESC-based therapy will finally be tested in patients with dry-AMD which severely impacts the quality of life of the elderly, and for which no approved therapy yet exists, Dr. Banin stated.

Information about the trial will be made available at ClinicalTrials.gov website of the National Institutes of Health http://www.clinicaltrials.gov/ct2/home. Additional information will be made available on Cell Cures website at http://www.cellcureneurosciences.com/.

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. The yearly economic loss to the gross domestic product in the United States from dry-AMD has been estimated to be $24.4 billion. The market opportunity for a treatment for GA has been estimated at over $5 billion globally. 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 annual sales of VEGF inhibitors for the treatment of the wet form of AMD are estimated to be about $7 billion worldwide.

The root cause of the larger problem of dry-AMD is believed to be the dysfunction of RPE cells. Therefore, one of the most exciting new therapeutic strategies for dry-AMD is the transplantation of healthy young RPE cells to support and replace those lost with age. Pluripotent stem cells, such as hESCs, can potentially provide a means of manufacturing such healthy RPE cells on an industrial scale.

About OpRegen

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BioTimes Subsidiary Cell Cure Neurosciences Receives FDA Authorization to Initiate Phase I/IIa Trial of Embryonic ...