- 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 -
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 patients.”
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 cohort.
“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.
About OpRegen®
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.
About BioTime
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,
LinkedIn, Facebook, YouTube, and Google+.
To receive ongoing BioTime corporate communications, please
click on the following link to join the Company’s email alert list:
http://news.biotimeinc.com.
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.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20161020005519/en/
Investor Contact:EVC Group, Inc.Matt Haines,
917-733-9297mhaines@evcgroup.comorMedia Contact:Gotham
Communications, LLCBill Douglass,
646-504-0890bill@gothamcomm.com
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