BioTime Initiates Dosing in Phase I/IIa Clinical Study of OpRegen for Treatment of Dry-AMD Utilizing Orbit Subretinal Deliver...
July 11 2019 - 8:00AM
Business Wire
BioTime, Inc. (NYSE American and TASE: BTX), a clinical-stage
biotechnology company developing cellular therapies for unmet
medical needs, today announced that it has dosed its first patient
with the Orbit Subretinal Delivery System (Orbit SDS) as well as
with a new Thaw-and-Inject formulation of OpRegen®, the Company’s
retinal pigment epithelium (RPE) transplant therapy, in its ongoing
Phase I/IIa clinical study for the treatment of dry age-related
macular degeneration, a leading cause of adult blindness in the
developed world.
“We are excited to be evaluating the FDA-cleared Orbit SDS in
the next six patients of our Phase I/IIa study,” stated Brian M.
Culley, Chief Executive Officer of BioTime. “We believe that
precise administration of OpRegen cells to the back of the eye
utilizing the Orbit SDS will lead to better dose control and an
overall safer procedure, with an increased likelihood of positive
clinical outcomes. In preparation for potential future
commercialization, we also are introducing our new Thaw-and-Inject
formulation, enabling rapid off-the-shelf administration of RPE
cells upon thawing, which we believe will streamline the use of
OpRegen by retinal surgeons.”
The primary objective of the Phase I/IIa study is to evaluate
the safety and tolerability of OpRegen as assessed by the incidence
and frequency of treatment emergent adverse events (AEs). Secondary
objectives are to evaluate the preliminary efficacy of OpRegen
treatment by assessing the changes in ophthalmological parameters
measured by various methods of primary clinical relevance.
Additionally, for the patients in Cohort 4 that receive subretinal
delivery of OpRegen utilizing the Orbit SDS, objectives will
include the evaluation of the safety of delivery of OpRegen using
the Orbit SDS.
About the Phase I/IIa Clinical Study
This is a Phase I/IIa open-label, dose escalation safety and
efficacy study of human embryonic stem cell-derived retinal pigment
epithelium cells transplanted subretinally in patients with
advanced dry age-related macular degeneration with geographic
atrophy. The study will enroll approximately 24 subjects, divided
into 4 cohorts. The first 2 cohorts, each consisting of 3 legally
blind subjects with best corrected visual acuity (BCVA) of 20/200
or less, received a single subretinal implantation of OpRegen. The
third cohort included 6 subjects with BCVA of 20/200 or less, who
received a single subretinal implantation of OpRegen. Staggered
intervals within and between cohorts are applied to ensure subject
safety and welfare. The fourth cohort will include approximately 12
subjects with BCVA between 20/64 and 20/250, who will receive a
single subretinal implantation of OpRegen. Cohort 4 includes two
formulations of OpRegen; the first 3 subjects were treated with an
ophthalmic Balanced Salt Solution Plus (BSS Plus) version of
OpRegen. Remaining subjects will be treated with an “off-the-shelf”
or “thaw and inject” (TAI) formulation. OpRegen TAI can be shipped
directly to sites and used upon thawing, which removes the
complications and logistics of having to use a dose preparation
facility. Staggered intervals will be applied between at least the
first two subjects of each delivery modality to ensure subject
safety and welfare.
About OpRegen®
OpRegen is a retinal pigment epithelium (RPE) transplant therapy
in Phase I/IIa development for the treatment of dry-AMD, a leading
cause of adult blindness in the developed world. OpRegen consists
of a suspension of RPE cells delivered subretinally as an
intraocular injection. RPE cells are essential components of the
back lining of the retina and function to help nourish the retina
including photoreceptors. OpRegen has been granted Fast Track
designation from the U.S. Food and Drug Administration. OpRegen is
a registered trademark of Cell Cure Neurosciences Ltd., a
majority-owned subsidiary of BioTime, Inc.
About BioTime, Inc.
BioTime is a clinical-stage biotechnology company developing new
cellular therapies for unmet medical needs. BioTime’s programs are
based on its proprietary cell-based therapy platform and associated
development and manufacturing capabilities. With this platform
BioTime develops and manufactures specialized,
terminally-differentiated human cells from its pluripotent and
progenitor cell starting materials. These differentiated cells are
developed either to replace or support cells that are dysfunctional
or absent due to degenerative disease or traumatic injury or
administered as a means of helping the body mount an effective
immune response to cancer. BioTime’s clinical assets include (i)
OpRegen®, a retinal pigment epithelium transplant therapy in Phase
I/IIa development for the treatment of dry age-related macular
degeneration, a leading cause of blindness in the developed world;
(ii) OPC1, an oligodendrocyte progenitor cell therapy in Phase
I/IIa development for the treatment of acute spinal cord injuries;
and (iii) VAC2, an allogeneic cancer immunotherapy of
antigen-presenting dendritic cells currently in Phase I development
for the treatment of non-small cell lung cancer. For more
information, please visit www.biotimeinc.com or follow the Company
on Twitter @BioTimeBTX.
About the Orbit Biomedical Subretinal Delivery System
The Orbit Biomedical Surgical Delivery System (Orbit SDS) was
designed to help surgeons deliver therapies to the retina with
precision and accuracy. To deliver a therapy to the retina a
surgeon would traditionally perform a vitrectomy and remove the
gel-like substance (the vitreous) that fills the eye. Then the
therapy is injected into the eye through the retina, a process
called a retinotomy. The Orbit SDS, which received 510K clearance
from the U.S. Food and Drug Administration, is designed to avoid
the need for a vitrectomy and perforation of the retina. The goal
is to simplify surgical procedures to make the administration safe
and consistent to give the medicine the best chance of working. The
Orbit SDS was developed by Orbit Biomedical. In 2019 Orbit
Biomedical merged with Gyroscope Therapeutics creating the only
retinal gene therapy company to combine discovery research, drug
development, a manufacturing platform and surgical delivery
capabilities. In addition to developing the Orbit SDS technology
for its own medicines, Gyroscope also licenses the Orbit SDS
technology to other companies who are developing gene and cell
therapies to treat eye disease.
Forward-Looking Statements
BioTime cautions you that all statements, other than statements
of historical facts, contained in this press release, are
forward-looking statements. Forward-looking statements, in some
cases, can be identified by terms such as “believe,” “may,” “will,”
“estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,”
“could,” “plan,” “potential,” “predict,” “seek,” “should,” “would,”
“contemplate,” “project,” “target,” “tend to,” or the negative
version of these words and similar expressions. Such statements
include, but are not limited to, statements relating to BioTime’s
ability to advance its product candidates and BioTime’s
expectations regarding its delivery systems and formulations.
Forward-looking statements involve known and unknown risks,
uncertainties and other factors that may cause BioTime’s actual
results, performance or achievements to be materially different
from future results, performance or achievements expressed or
implied by the forward-looking statements in this press release,
including risks and uncertainties inherent in BioTime’s business
and other risks described in BioTime’s filings with the Securities
and Exchange Commission (SEC). BioTime’s forward-looking statements
are based upon its current expectations and involve assumptions
that may never materialize or may prove to be incorrect. All
forward-looking statements are expressly qualified in their
entirety by these cautionary statements. Further information
regarding these and other risks is included under the heading “Risk
Factors” in BioTime’s periodic reports filed with the SEC,
including BioTime’s Annual Report on Form 10-K filed with the SEC
on March 14, 2019 and its other reports, which are available from
the SEC’s website. You are cautioned not to place undue reliance on
forward-looking statements, which speak only as of the date on
which they were made. BioTime undertakes no obligation to update
such statements to reflect events that occur or circumstances that
exist after the date on which they were made, except as required by
law.
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version on businesswire.com: https://www.businesswire.com/news/home/20190711005165/en/
BioTime Inc. IR Ioana C. Hone ir@biotimeinc.com (510)
871-4188
Solebury Trout IR Gitanjali Jain Ogawa
Gogawa@troutgroup.com (646) 378-2949
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