Data From BioTime's Phase I/IIa OpRegen® Trial to be Presented at the 2017 American Academy of Ophthalmology (AAO) Annual Meeting
ALAMEDA, Calif.--(BUSINESS WIRE)--
BioTime, Inc. (NYSE MKT:BTX), a clinical-stage biotechnology company
developing and commercializing products addressing degenerative
diseases, today announced that an abstract related to the Company's
retinal tissue implant has been accepted for a paper presentation at the
American Academy of Ophthalmology (AAO) annual meeting being held in New
Orleans, Louisiana, November 11-14.
The abstract accepted for paper presentation is titled, "Phase 1 and
2a Study of Human Embryonic Stem Cell-Derived Retinal Pigment Epithelial
Cells Transplanted Subretinally in Advanced Dry-Form AMD Patients." The
data will be presented by Dr. Oscar Cuzzani MD PhD, BioTime's Vice
President of Clinical Development. With more than 30 years of experience
as a physician and in clinical development, Dr. Cuzzani has a wealth of
experience practicing retinal surgery, and has held senior level
positions working at some of the best research organizations including
the National Eye institute and the Bascom Palmer Eye Institute.
"The clinical work that our team, along with the principal investigators
and other leading ophthalmologists, is conducting is of great importance
for the millions of patients suffering from this debilitating disease
and leading cause of blindness among the elderly population," commented
Adi Mohanty, co-Chief Executive Officer. "This is a significant
opportunity to broaden awareness among clinicians about the significant
progress of the OpRegen program."
The Company announced data from the Phase I/IIa clinical trial of
OpRegen at the Association for Research in Vision and Ophthalmology
(ARVO) annual meeting in May. The presentation reported new clinical
trial data with two patients that were treated in cohort 2, where they
received a dose of 200,000 cells. Imaging analysis suggests the
transplanted OpRegen cells remained in place (engrafted) in an area of
the scar that was completely depleted of retinal pigment epithelium
(RPE) because of the advanced stages of the disease. Cell engraftment
appears evident in four of the five patients treated thus far. There was
also possible evidence of a biological response with some areas
appearing to show structural improvement (a thickening of the area of
the neural retina above the scar) without any signs of retinal edema, a
fluid build-up that can further compromise vision.
OpRegen is now in a Phase I/IIa dose finding study to evaluate its
safety and efficacy in patients who have an advanced stage of the
disease, and has received fast track designation by the FDA.
OpRegen®, which is being studied for the treatment of the dry form of
age-related macular degeneration (AMD), consists of a suspension of
Retinal Pigment Epithelial (RPE) cells that are delivered subretinally
during a simple intraocular injection. RPE cells are essential
components of the back lining of the retina, and function to help
nourish the retina including photoreceptors. A proprietary process that
drives the differentiation of human pluripotent stem cells is used to
generate high purity OpRegen® RPE cells. OpRegen® RPE cells are also
"xeno-free," meaning that no animal products are used at any point in
the derivation and production process. The avoidance of the use of
animal products eliminates some potential safety concerns. Preclinical
studies in rats have shown that following a single subretinal injection
of OpRegen®, the cells can rapidly organize into its natural monolayer
structure in the subretinal space and survive throughout the lifetime of
the animal. OpRegen® is designed to be an "off-the-shelf" allogeneic
(non-patient specific) product. Unlike treatments that require multiple,
frequent injections into the eye, it is expected that OpRegen® would be
administered in a single procedure. OpRegen® was granted Fast Track
designation from FDA, which allows more frequent interactions with the
agency, and eligibility for accelerated approval and priority review.
OpRegen® is a registered trademark of Cell Cure Neurosciences Ltd., a
majority-owned subsidiary of BioTime, Inc.
About Dry Age-Related Macular Degeneration (Dry-AMD)
Macular degeneration affects approximately 11 million people in the U.S.
and is the leading cause of blindness in people over the age of 60.
Approximately 90 percent of these patients suffer from the dry form, for
which there are no FDA-approved therapies. In dry-AMD, there is a loss
or dysfunction of the layer of retinal pigment epithelial (RPE) cells
generally in the region of the eye called the macula, which is the part
of the retina responsible for sharp, central vision that is important
for facial recognition, reading and driving. These RPE cells support the
light detecting photoreceptor cells that are so critical to vision. When
we look at something, the photoreceptors (rods and cones) detect the
light and send the information to the brain allowing us to perceive our
surroundings. The age-dependent loss of the RPE cells therefore leads to
degeneration of nearby photoreceptors and this can lead to severe vision
loss or even legal blindness. Generally, the damage caused by the "dry"
form is not as severe or rapid as that of the "wet" form. However, in
the advanced stage of dry macular degeneration widespread loss of RPE
and photoreceptors in the macular area, called geographic atrophy, leads
to severe vision loss. While therapeutics are available to treat the wet
form of AMD, there are currently no FDA-approved therapies for dry-AMD.
BioTime is a clinical-stage biotechnology company focused on developing
and commercializing products addressing degenerative diseases. Our
clinical programs are based on two platform technologies: pluripotent
cells and cell/drug delivery. The foundation of our core therapeutic
technology platform is pluripotent cells that are capable of becoming
any of the cell types in the human body. The foundation of our cell
delivery platform is its HyStem® cell and drug delivery matrix
technology. The Company's current clinical programs are targeting three
primary sectors, aesthetics, ophthalmology and cell/drug delivery. We
also have significant equity holdings in two publicly traded companies,
Asterias Biotherapeutics, Inc. ("Asterias") and OncoCyte Corporation
("OncoCyte"), which we founded and which, until recently, were our
majority-owned consolidated subsidiaries.
BioTime common stock is traded on the NYSE MKT and TASE under the symbol
BTX. For more information, please visit www.biotimeinc.com
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Certain statements contained in this release are "forward-looking
statements" within the meaning of the Private Securities Litigation
Reform Act of 1995. Statements pertaining to future financial and/or
operating results, future growth in research, technology, clinical
development, and potential opportunities for BioTime, Inc. 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, Inc. and its
subsidiaries, particularly those mentioned in the cautionary statements
found in more detail in the "Risk Factors" section of its Annual Reports
on Form 10-K and Quarterly Reports on Form 10-Q filed with the SEC
(copies of which may be obtained at www.sec.gov).
Subsequent events and developments may cause these forward-looking
statements to change. BioTime specifically disclaims any obligation or
intention to update or revise these forward-looking statements as a
result of changed events or circumstances that occur after the date of
this release, except as required by applicable law.
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Source: BioTime, Inc.