Imaging Analysis Suggests Cell Engraftment and
Likely Biological Response
BioTime, Inc. (NYSE MKT:BTX), a clinical-stage
biotechnology company developing and commercializing products
addressing degenerative diseases, today announced new data from the
Phase I/IIa clinical trial of OpRegen® in the advanced
form of dry age-related macular degeneration (dry-AMD). The interim
data were presented on May 8, 2017 at the Annual Meeting of the
Association for Research in Vision and Ophthalmology (ARVO) in
Baltimore, Maryland by Eyal Banin, M.D., Ph.D.
“We are encouraged by the results and look forward to the next
steps in moving this therapeutic through the clinic and addressing
the needs of the millions of patients with dry-AMD,” said Adi
Mohanty, Co-Chief Executive Officer.
The presentation at ARVO 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
occurred 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
thinned area of retina above the scar) without any signs of retinal
edema, a fluid build-up that can further compromise vision.
“I am excited to see that the second cohort is showing evidence
of cell survival and early signs suggesting a biological response,”
stated Dr. David Boyer, senior partner at Retina-Vitreous
Associates Medical Group in Los Angeles and a principal
investigator for the OpRegen trial. “I am looking forward to
treating patients in the U.S. under this trial protocol.”
Best corrected visual acuity (BCVA) remained stable in all
treated eyes and importantly showed no signs of deterioration,
which could be expected if no treatment was given. The data also
continues to show that the procedure is well tolerated in all
patients, including those patients with follow-up for more than one
year after treatment.
“We continue to be encouraged by the lack of serious adverse
events in the trial during both the surgical procedure required for
cell transplantation and long-term post-operative monitoring,”
stated Professor Eyal Banin, M.D., Ph.D., Center for Hereditary
Retinal and Macular Degeneration (CRMD) Department of Ophthalmology
Hadassah-Hebrew University Medical Center, Jerusalem, Israel. “Thus
far in the trial, we have not encountered any serious systemic side
effects or unexpected severe ocular adverse events.”
OpRegen is the lead product of BioTime’s ophthalmology
subsidiary Cell Cure Neurosciences Ltd., which has been conducting
the clinical trial in Israel and will soon be opening additional
trial sites in the United States. BioTime anticipates DSMB review
of cohort 2 by the end of the second quarter of 2017 and if
approved will then begin cohort 3 immediately after. Cohort 3 is
expected to enroll more quickly due to the reduced stagger
requirements between patients and the ability of new clinical trial
sites in the U.S. to enroll patients. The Company also expects to
share additional data from all patients before the end of the
year.
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.
More information about the data presented at ARVO is available
here.
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. 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
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 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 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. BioTime also
has significant equity holdings in two publicly traded companies,
Asterias Biotherapeutics, Inc. and OncoCyte Corporation, which
BioTime founded and which, until recently, were majority-owned
consolidated subsidiaries of BioTime.
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+.
Forward-Looking Statements
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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version on businesswire.com: http://www.businesswire.com/news/home/20170509005727/en/
Investor Contact:for BioTime, Inc.Brian Moore,
310-770-0389bmoore@evcgroup.comorMedia Contact:Gotham
Communications, LLCBill Douglass,
646-504-0890bill@gothamcomm.com
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