Full Press Release Details
Neurosciences Announces Preclinical Efficacy Data Demonstrating OpRegen
to be Presented at Association for Research in Vision and Ophthalmology
(ARVO) 2015 Annual Meeting
ALAMEDA, Calif. & JERUSALEM--(BUSINESS WIRE)--May 4, 2015--BioTime, Inc.
(NYSE MKT: BTX) and its subsidiary Cell Cure Neurosciences Ltd. (Cell
Cure) today announced that preclinical data demonstrating that Cell
Cure's product candidate, OpRegen , preserved
vision and retinal structure when transplanted into the leading animal
model of retinal disease, will be presented at the Association for
Research in Vision and Ophthalmology (ARVO) 2015 Annual Meeting taking
place May 3-7, 2015, in Denver, Colorado. OpRegen
consists of animal product-free retinal pigment epithelial (RPE) cells
with high purity and potency.
The preclinical study was conducted by a scientific team led by Trevor
J. McGill, Ph.D., Research Assistant Professor at the Casey Eye
Institute, Oregon Health and Science University. The abstract accepted
for paper presentation is titled, "Long Term Efficacy of Xeno-free
hESC-derived RPE Cells Following Transplantation into Royal College of
Surgeons Rats." The data that will be presented by Michael D. Andrews of
the Casey Eye Institute, Oregon Health and Science University, and lead
study investigator, during session 202, titled, "Retinal degeneration
and disease: experimental models," is scheduled for Monday, May 4, 2015
from 9:00 AM to 9:15 AM Mountain Time Zone. The abstract number is 1275.
The presentation abstract is available online at the ARVO website at http://www.arvo.org/Online_Planner/.
Cell Cure has received regulatory clearance from the U.S. Food and Drug
Administration (FDA) and the Israeli Ministry of Health to initiate a
Phase I/IIa dose escalation safety and efficacy clinical study of OpRegen
for geographic atrophy (GA), the severe stage of the dry form of
age-related macular degeneration (dry-AMD). Patient enrollment has
started at Hadassah University Medical Center in Jerusalem, Israel. The
trial consists of four cohorts and will evaluate three different dose
regimens. Details of the trial are available at https://clinicaltrials.gov/.
Cell Cure expects to report interim data from the cohorts in the coming
While treatment options exist for the wet form of AMD, which represents
about 10% of the disease prevalence, there is currently no FDA-approved
therapy for the dry form that occurs 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
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 such as Lucentis or Eylea. 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 about $8 billion worldwide. The root cause of the larger problem
of 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
human embryonic stem cells (hESCs), can provide an unlimited source for
the derivation of such healthy RPE cells for transplantation.
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
as a suspension of cells, the cells can rapidly organize into their
natural monolayer structure and survive throughout the lifetime of the
animal. OpRegen will be an "off-the-shelf"
allogeneic product. Unlike treatments that require multiple, frequent
injections into the eye, 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.
BioTime, Inc., a pioneer in regenerative medicine, is a clinical-stage
biotechnology company. BioTime and its subsidiaries are leveraging their
industry-leading experience in pluripotent stem cell technology and a
broad intellectual property portfolio to facilitate the development and