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
Ministry of Health.
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
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®
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® is anticipated 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.
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). BioTime directly and indirectly through its
subsidiaries owns approximately 62.5% of Cell Cure. Cell Cure’s second
largest shareholder is HBL- Hadasit Bio-Holdings, (TASE: HDST, OTC:
HADSY) followed by Teva Pharmaceuticals Industries Ltd. (NYSE: TEVA).
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. Additional information about Cell
Cure can be found on the web at www.cellcureneurosciences.com.
A video
of a presentation by Cell Cure’s CEO Dr. Charles Irving is available on
BioTime’s website.
About BioTime
BioTime is a biotechnology company engaged in research and product
development in the field of regenerative medicine. Regenerative medicine
refers to therapies based on stem cell technology that are designed to
rebuild cell and tissue function lost due to degenerative disease or
injury. BioTime’s focus is on pluripotent stem cell technology based on
human embryonic stem (“hES”) cells and induced pluripotent stem (“iPS”)
cells. hES and iPS cells provide a means of manufacturing every cell
type in the human body and therefore show considerable promise for the
development of a number of new therapeutic products. BioTime’s
therapeutic and research products include a wide array of proprietary PureStem®
progenitors, HyStem®
hydrogels, culture media, and differentiation kits. BioTime is
developing Renevia™ (a HyStem® product) as a
biocompatible, implantable hyaluronan and collagen-based matrix for cell
delivery in human clinical applications, and is planning to initiate a
pivotal clinical trial around Renevia™, in 2014. In addition,
BioTime has developed Hextend®,
a blood plasma volume expander for use in surgery, emergency trauma
treatment and other applications. Hextend® is
manufactured and distributed in the U.S. by Hospira, Inc. and in South
Korea by CJ HealthCare Corporation, under exclusive licensing agreements.
BioTime is also developing stem cell and other products for research,
therapeutic, and diagnostic use through its subsidiaries:
-
Asterias
Biotherapeutics, Inc. is developing pluripotent stem-cell based
therapies in neurology and oncology, including AST-OPC1
oligodendrocyte progenitor cells in spinal cord injury, multiple
sclerosis and stroke, and AST-VAC2, an allogeneic dendritic cell-based
cancer vaccine. Asterias trades publicly on the NYSE MKT under the
symbol AST.
-
BioTime Asia, Ltd., a Hong Kong company,
may offer and sell products for research use for BioTime’s ESI BIO
Division.
-
Cell
Cure Neurosciences Ltd. is an Israel-based biotechnology company
focused on developing stem cell-based therapies for retinal and
neurological disorders, including the development of retinal pigment
epithelial cells for the treatment of macular degeneration, and
treatments for multiple sclerosis.
-
ESI
BIO is the research and product marketing division of BioTime,
providing stem cell researchers with products and technologies to
enable them to translate their work into the clinic, including PureStem®
progenitors and HyStem® hydrogels.
-
LifeMap
Sciences, Inc. markets, sells, and distributes GeneCards®,
the leading human gene database, as part of an integrated database
suite that also includes the LifeMap
Discovery® database of embryonic development, stem
cell research, and regenerative medicine, and MalaCards,
the human disease database.
-
LifeMap
Solutions, Inc. is a subsidiary of LifeMap Sciences focused on
developing mobile health (mHealth) products.
-
OncoCyte
Corporation is developing products and technologies to diagnose and
treat cancer, including PanC-Dx™, with four clinical studies
currently underway.
-
OrthoCyte
Corporation is developing therapies to treat orthopedic disorders,
diseases and injuries.
-
ReCyte
Therapeutics, Inc. is developing therapies to treat a variety of
cardiovascular and related ischemic disorders, as well as products for
research using cell reprogramming technology.
BioTime stock is traded on the NYSE MKT, ticker BTX. For more
information, please visit www.biotimeinc.com
or connect with the company on Twitter,
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Facebook,
YouTube,
and Google+.
Forward-Looking Statements
Statements pertaining to future financial and/or operating results,
future growth in research, technology, clinical development, and
potential opportunities for BioTime and its subsidiaries, along with
other statements about the future expectations, beliefs, goals, plans,
or prospects expressed by management constitute forward-looking
statements. Any statements that are not historical fact (including, but
not limited to statements that contain words such as “will,” “believes,”
“plans,” “anticipates,” “expects,” “estimates”) should also be
considered to be forward-looking statements. Forward-looking statements
involve risks and uncertainties, including, without limitation, risks
inherent in the development and/or commercialization of potential
products, uncertainty in the results of clinical trials or regulatory
approvals, need and ability to obtain future capital, and maintenance of
intellectual property rights. Actual results may differ materially from
the results anticipated in these forward-looking statements and as such
should be evaluated together with the many uncertainties that affect the
business of BioTime and its subsidiaries, particularly those mentioned
in the cautionary statements found in BioTime's Securities and Exchange
Commission filings. BioTime disclaims any intent or obligation to update
these forward-looking statements.
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Copyright Business Wire 2014