Full Press Release Details
Subsidiary Cell Cure Neurosciences Receives FDA Authorization to
Initiate Phase I/IIa Trial of Embryonic Stem Cell-Derived OpRegen
for the Treatment of the Dry Form of Age-Related Macular Degeneration
cleared for Phase I/IIa dose escalation trial in patients with the dry
form of age-related macular degeneration (AMD) called geographic
approved therapy exists for dry-AMD, the leading cause of visual
impairment in an aging population in the US and other developed
be the first preparation of xeno-free human embryonic stem
cell-derived RPE cells evaluated for transplant therapy of dry-AMD
ALAMEDA, Calif. & JERUSALEM--(BUSINESS WIRE)--November 3, 2014--BioTime,
Inc. (NYSE MKT:BTX) and Cell Cure Neurosciences Ltd. (Cell Cure) today
announced that the U.S. Food and Drug Administration (FDA) has cleared
Cell Cure's Investigational New Drug (IND) application to initiate a
Phase I/IIa clinical trial of OpRegen in
patients with the severe form of age-related macular degeneration (AMD)
called geographic atrophy (GA). AMD is the leading cause of blindness in
the aging US population and many other developed countries around the
world. 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 of the disease
occurring in approximately 90% of all patients with AMD.
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
About the OpRegen Clinical Trial
Cell Cure's 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 FDA's 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 Cure's 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 Cure's 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
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