Full Press Release Details
Subsidiary Cell Cure Neurosciences Ltd. Demonstrates the Safety and
in Preclinical Animal Studies
ALAMEDA, Calif. & JERUSALEM--(BUSINESS WIRE)--September 17,
2014--BioTime, Inc. (NYSE MKT: BTX), HBL Hadasit Bio-Holdings Ltd. (Tel
Aviv Stock Exchange: HDST) and Cell Cure Neurosciences Ltd. (Cell Cure)
today announced that Cell Cure has received the final results of a
series of extensive preclinical safety and efficacy studies of its
development-stage product designated OpRegen ,
which is intended for use in the treatment of dry form age-related
macular degeneration (dry-AMD). These studies were conducted by Cell
Cure and contract research organizations in preparation for an IND
filing with the Food and Drug Administration for a Phase 1/2a study in
patients with geographic atrophy, the severe stage of dry-AMD. OpRegen
consists of retinal pigment epithelial cells derived from human
embryonic stem cells using a proprietary directed differentiation
method. OpRegen is intended to be administered
into the subretinal space of patients as a suspension of cells to treat
this leading cause of blindness.
"We are very pleased with the safety data obtained in two animal species
that demonstrate that OpRegen cells
survive following transplantation for a long period of time and do not
result in teratoma formation or any other type of pathology. The
efficacy of the OpRegen cells was
evaluated in the Royal College of Surgery rat model of retinal
degeneration, which is a well-established animal model of retinal
degeneration which has been extensively used to evaluate various
potential cell therapies. The OpRegen cells were
found to remain therapeutically functional over long periods and to
maintain the animal's visual performance that would normally decay over
time in this disease model," 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.
"Furthermore, the protection of the animal's vision from decay was dose
dependent. As an additional indicator of therapeutic potential, the
number of cone photoreceptors, which are responsible for fine vision in
humans and are degenerating in the macula of dry-AMD patients, was found
to remain constant over an extended period in the animal model."
"We are very pleased with the progress that Cell Cure's team has made in
preparing for the company's FDA submission," said Charles S. Irving PhD,
Cell Cure's CEO. "We look forward to initiating the clinical trial that
will utilize for the first time high quality, xeno-free grade RPE cells
for the treatment of geographic atrophy, the severe stage of dry-AMD."
About Age-Related Macular Degeneration
Age-related macular degeneration (AMD) is one of the major diseases of
aging and is the leading cause of visual impairment in Americans 55
years of age and older. 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 until it reaches the
severe form called geographic atrophy (GA), which may result in legal
blindness. About 10% of patients with dry-AMD develop wet-AMD, which is
an acute disease and can lead to blindness in a matter of weeks. Wet-AMD
can be treated with currently-marketed angiogenesis inhibitors such as
Lucentis or Eylea, however, such products typically require frequent
injections, and patients often continue to suffer from the continued
progression of the underlying dry-AMD disease process. There is no
FDA-approved treatment for dry-AMD for which some seven million people
in the US have the intermediate form of the disease and have a high risk
for developing GA. The market opportunity for a treatment for GA has
been estimated at over $5 billion globally. Current estimated sales of
angiogenesis 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 degeneration of a particular
type of cell in the retina called "retinal pigment epithelial" (RPE)
cells. One of the most exciting therapeutic approaches to dry-AMD is the
transplantation of healthy, young RPE cells to replace the patient's old
degenerating RPE cells. One of the most promising sources of healthy RPE
cells is from pluripotent stem cells.
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 OpRegen transplanted
subretinally as a suspension of 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 provided to retinal surgeons in a final formulation
ready for transplantation. Unlike treatments that require multiple
injections into the eye, such as currently-marketed products like
Lucentis and Eylea for wet-AMD, it is expected that OpRegen
will be administered in a single procedure.
About Cell Cure Neurosciences Ltd.
Cell Cure Neurosciences Ltd. was established in 2005 as a subsidiary of
ES Cell International Pte. Ltd. (ESI), now a subsidiary of BioTime, Inc.
(NYSE MKT: BTX). Cell Cure's second largest shareholder is HBL Hadasit
Bio-Holdings, (TASE: HDST, OTC: HADSY). 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., HBL Hadasit Bio-Holdings Ltd.,
Teva Pharmaceuticals Industries Ltd. (NYSE: TEVA), and Asterias