Key BioTime Patents Upheld by European Patent Office
EPO Rules Patents for OpRegen® are Valid and
Remain in Force as Granted
OpRegen® is in Development to Treat Dry-AMD
ALAMEDA, Calif.--(BUSINESS WIRE)--Mar. 28, 2017--
BioTime, Inc. (NYSE MKT:BTX), a clinical stage biotechnology company
developing and commercializing products addressing degenerative disease,
today announced the successful defense of two key patents from challenge
before the European Patent Office (EPO) Opposition division. A large
pharmaceutical company and an anonymous filer challenged the EPO’s
previous grant of two European patents (EP2554661 and EP2147094), which
cover the proprietary directed differentiation methods to produce
pluripotent stem cell-derived cell replacement therapies being developed
to treat retinal degenerative diseases such as age-related macular
degeneration (“AMD”). BioTime addressed the challenges at the EPO
headquarters in Munich, Germany during public oral hearings, which took
place March 16 -17, 2017.
“The EPO's decision to uphold these patents protecting OpRegen
reinforces BioTime’s ability to defend its intellectual property and
patent portfolios, which is one of the largest in the pluripotent cell
therapy industry,” said Stephana Patton, General Counsel of BioTime,
Inc. “The patents in question are a key component of the intellectual
property (IP) assets our subsidiary, Cell Cure Neurosciences Ltd.,
secured through license from Hadasit Medical Research Services and
Development Ltd. They have broad and significant claims describing
directed differentiation, which we believe are critical for the
development and commercialization of pluripotent cell-based therapeutics
aimed at the dry form of AMD. BioTime’s IP estate remains a cornerstone
in our efforts to advance the development and commercialization of
pluripotent cell-based therapies for patients in need.”
OpRegen is an investigational therapy in which retinal pigment
epithelial (RPE) cells are transplanted into the subretinal space, where
they are intended to replace missing RPE cells. The ongoing trial is a
Phase I/IIa dose-escalation study evaluating the safety and efficacy of
three different dose regimens of OpRegen in patients with the advanced
form of dry-AMD accompanied by geographic atrophy. Data recently
presented from the first patient cohort indicate that, at the first
dose, OpRegen caused no serious adverse events, and retinal imaging
suggests the presence and survival of transplanted cells in the
subretinal space for up to one year. Data from the cohort were presented
at the International Symposium on Ocular Pharmacology and Therapeutics (ISOPT)
in Rome, on December 2, 2016 and at Bascom Palmer Eye Institute’s annual Angiogenesis
meeting in Miami, Florida on February 11, 2017. Additional data will
be presented at the Annual Meeting of the Association for Research in
Vision and Ophthalmology (ARVO)
in Baltimore, Maryland on May 8, 2017. OpRegen has received Fast Track
designation from the Food and Drug Administration (FDA) for treatment of
the advanced form of dry age-related macular degeneration (dry-AMD).
Details of the trial and about a patient’s eligibility are available at https://clinicaltrials.gov/
with the following Identifier: NCT02286089 (dry-AMD).
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.
About OpRegen®
OpRegen 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. 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 either 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 BioTime
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
stem cells and cell/drug delivery platform technologies. 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 or
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and Google+.
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Source: BioTime, Inc.
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