– OpRegen at the first dose caused no serious adverse events in the
first patient cohort –
– Retinal imaging suggests presence and survival of the transplanted
cells in the subretinal space for up to one year –
ALAMEDA, Calif.–(BUSINESS WIRE)–BioTime, Inc. (NYSE MKT and TASE: BTX), a clinical stage biotechnology
company with a focus on pluripotent stem cell technologies, today
reported that data from the first patient cohort of the Phase I/IIa
clinical trial of OpRegen® in the advanced form of dry
age-related macular degeneration (dry-AMD) will be presented at the International
Symposium on Ocular Pharmacology and Therapeutics (ISOPT) on Friday,
December 2, 2016, in Rome, Italy.
The Phase I/IIa clinical trial, which is being conducted by BioTime’s
subsidiary, Cell Cure Neurosciences Ltd., is evaluating the safety of
three different dose regimens of OpRegen in the advanced form of dry-AMD
that is accompanied by geographic atrophy. The first Phase I/IIa patient
cohort received an initial targeted dose of 50,000 cells.
“The primary focus of this cohort is safety. OpRegen was successfully
administered with no serious adverse events,” commented Prof. Eyal
Banin, Director of the Center for Retinal and Macular Degenerations at
Hadassah Medical Center, where the trial is being conducted.
“Importantly, retinal imaging suggests that OpRegen RPE cells are able
to engraft. Imaging from the first patient, who just completed one-year
of post-treatment clinical assessment, may indicate that the graft can
survive for at least 12 months. We are encouraged by these data and our
continued progress in this important trial.”
“OpRegen RPE cells were created without the use of animal-derived
products through a proprietary directed differentiation process to
produce a highly purified population of RPE cells,” said Prof. Benjamin
Reubinoff, Chief Scientific Officer of Cell Cure Neurosciences Ltd. and
director of Hadassah’s Stem Cell Research Center. “We are pleased that
OpRegen did not cause serious adverse events in the first cohort of
The safety profile of the first cohort of subjects was thoroughly
assessed by the Data Safety Monitoring Board (DSMB), an independent
group of physicians and medical experts closely monitoring the clinical
trial, before it gave its recommendation that the company continue the
trial with the second cohort at a higher dose of 200,000 cells. The
company expects a similar review by the DSMB at the end of the second
“We are looking forward to the opportunity to present data from the
first patient cohort at ISOPT,” said Adi Mohanty, Co-Chief Executive
Officer of BioTime. “OpRegen’s progress in the clinic is gaining
momentum and we have already treated patients with 200,000 cell doses.
It is in these higher cell dose cohorts where we believe OpRegen has the
potential to demonstrate more meaningful clinical outcomes and we expect
to start reporting on these data in early 2017. Our goal is to develop a
treatment that can serve the millions of dry-AMD patients for whom there
are currently no FDA-approved therapies.”
Enrollment in the second cohort is expected to be completed in 2016.
Depending on the outcome of the DSMB’s review of the second patient
cohort, approval to begin administering the 500,000 cell dosage to the
third patient cohort could be provided by the end of the current year.
The first OpRegen clinical trial site in the United States is expected
to be selected in the near future.
OpRegen has received Fast Track designation from the FDA for treatment
of the advanced form of 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 the ISOPT Clinical Symposium
The ISOPT Clinical Symposium is an annual symposium focusing on clinical
drug treatments in ophthalmology via a pragmatic clinical angle.
Symposium participants are clinicians, clinical investigators, academy
based researchers and members of the industry. The ISOPT Clinical board
has two major missions: 1) To provide updates on current paradigms of
therapy for common ophthalmic diseases with profound risk / benefit
coverage, and 2) To assess expected therapeutic paradigm shifts in the
near future as reflected in current clinical research. The ISOPT
Clinical meeting will focus on case presentations as they demonstrate
current treatment options. This year’s symposium will take place
December 1-3, 2016, in Rome, Italy. For more information please go to http://www.isoptclinical.com/home.ehtml.
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.
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 rats have shown that following a single
subretinal injection of OpRegen, as a suspension of cells, the cells can
rapidly organize into its natural monolayer structure 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® is a registered trademark of Cell Cure Neurosciences
Ltd., a majority-owned subsidiary of BioTime, Inc.
BioTime, Inc. is a clinical-stage biotechnology company focused on
developing and commercializing novel therapies developed from what the
Company believes to be the world’s premier collection of pluripotent
cell assets. The foundation of BioTime’s core therapeutic technology
platform is pluripotent cells that are capable of becoming any of the
cell types in the human body. Pluripotent cells have potential
application in many areas of medicine with large unmet patient needs,
including various age-related degenerative diseases and degenerative
conditions for which there presently are no cures. Unlike
pharmaceuticals that require a molecular target, therapeutic strategies
based on the use of pluripotent cells are generally aimed at
regenerating or replacing affected cells and tissues, and therefore may
have broader applicability than pharmaceutical products.
In addition to the development of therapeutics, BioTime’s research and
other activities have resulted, over time, in the creation of other
subsidiaries that address other non-therapeutic market opportunities
such as cancer diagnostics, drug development and cell research products,
and mobile health software applications.
BioTime common stock is traded on the NYSE MKT and TASE under the symbol
BTX. For more information, please visit www.biotimeinc.com
or connect with the company on Twitter,
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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.