Treatment with OpRegen®
Continues to be Well Tolerated with Signs of Structural
Improvement in the Retina Observed in Some Patients
BioTime, Inc. (NYSE American and TASE: BTX), a clinical-stage
biotechnology company developing new cellular therapies, announced
that updated results from a Phase I/IIa clinical study of its lead
product candidate, OpRegen®, a retinal pigment epithelium (RPE)
cell transplant therapy currently in development for the treatment
of dry age-related macular degeneration (AMD) with geographic
atrophy (GA), will be presented today at the 2019 Association for
Research in Vision and Ophthalmology Annual Meeting (ARVO 2019) in
Vancouver, BC, Canada. Data from the study demonstrate that
treatment with OpRegen continues to be well tolerated and in some
patients, signs of structural improvement in the treated areas of
the retina have been observed. Of note, early data from Cohort 4
patients with earlier-stage dry-AMD and smaller areas of GA remain
encouraging, with indications of the continued presence of the
transplanted OpRegen cells and improvements in visual acuity.
Data presented at ARVO 2019 showed that both the surgical
procedure and the OpRegen cells were generally well tolerated with
no unexpected adverse events or treatment-related systemic serious
adverse events reported in the first fifteen patients enrolled to
date. The most common and expected ocular adverse events were the
formation of mild epiretinal membranes (ERM). One instance of
retinal detachment occurred in a patient who was legally blind
prior to treatment. The event was not able to be assigned as
related to treatment, procedure, or to the combination, and the
patient continued in the study following successful surgical
repair. One instance of a severe ERM required surgical removal,
which was successful, and the subject continues to demonstrate
improved visual acuity from baseline following OpRegen
administration.
Imaging of several patients from Cohorts 1, 2 and 3, and of
particular interest, those from Cohort 4 (n=3) with better baseline
vision, demonstrated sustained structural improvement within the
retina and evidence of the continued presence of the transplanted
OpRegen cells. Within the area of the OpRegen cell transplant,
signs of a reduction and change in drusen material, as well as
improvements or possible restorations of the ellipsoid zone and RPE
layers, have persisted. The photoreceptor layer and ellipsoid zone
assumed a more regular structural appearance in areas of the
transition zone where OpRegen was administered, suggesting
potential structural restoration of the retina in areas receiving
the RPE cells. This is of particular importance because in dry-AMD
the structure of the retina can be impacted by the formation of
excess drusen and ultimately death of RPE cells and photoreceptors,
which are critical to sight. Other changes observed following
OpRegen treatment persisted through the last time point examined
(up to 3 years) and included subretinal pigmentation and
hyper-reflective areas seen on optical coherence tomography (OCT).
Additionally, asymmetrical, reduced growth of GA in the treated
areas receiving OpRegen was observed in some subjects. These
observations are being independently evaluated by the Doheny Eye
Institute and Doheny Image Reading & Research Lab (DIRRL), Los
Angeles, CA.
The Best Corrected Visual Acuity (BCVA) and areas of GA
continued to remain largely stable in the treated eyes. Notably,
the visual acuity of the first three Cohort 4 patients with better
baseline vision have all seen improvements from baseline levels and
will be followed for longer periods of time. Overall, OpRegen
appears well-tolerated with preliminary evidence of improved
structural changes and potential improvement in visual acuity
following treatment observed in some patients.
Eyal Banin, MD, PhD, Professor of Ophthalmology, Director,
Center for Retinal and Macular Degenerations, Department of
Ophthalmology at Hadassah-Hebrew University Medical Center, the
presenting author and one of the investigators participating in the
study, presented data from the Phase I/IIa clinical study. A copy
of Dr. Banin’s presentation will be available on the Events section
of BioTime’s website concurrent with his presentation at ARVO
2019.
About OpRegen®
OpRegen is a RPE transplant therapy in Phase I/IIa development
for the treatment of dry AMD, the 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.
About BioTime, Inc.
BioTime is a clinical-stage biotechnology company developing new
cellular therapies for degenerative retinal diseases, neurological
conditions associated with demyelination, and aiding the body in
detecting and combating cancer. 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, the 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.
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 the
potential improvement in visual acuity following treatment observed
in some patients. 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, without limitation, risk and
uncertainties related to: BioTime’s ability to raise additional
capital when and as needed, to advance its product candidates;
BioTime’s ability to develop and commercialize product candidates;
the failure or delay in starting, conducting and completing
clinical trials or obtaining FDA or foreign regulatory approval for
BioTime’s product candidates in a timely manner; the therapeutic
potential of BioTime’s product candidates, and the disease
indications for which BioTime intends to develop its product
candidates; BioTime’s ability to conduct and design successful
clinical trials, to enroll a sufficient number of patients, to meet
established clinical endpoints, to avoid undesirable side effects
and other safety concerns, and to demonstrate sufficient efficacy
of its product candidates; developments by BioTime competitors that
make BioTime’s product candidates less competitive or obsolete;
BioTime’s ability to manufacture its product candidates for
clinical development and, if approved, for commercialization, and
the timing and costs of such manufacture; the performance of third
parties in connection with the development and manufacture of
BioTime’s product candidates, including third parties conducting
clinical trials as well as third-party suppliers and manufacturers;
the potential of BioTime’s cell therapy platform, and BioTime’s
plans to apply its platform to research, develop and commercialize
our product candidates; BioTime’s ability, and the ability of its
licensors, to obtain, maintain, defend and enforce intellectual
property rights protecting BioTime’s product candidates, and
BioTime’s ability to develop and commercialize its product
candidates without infringing the proprietary rights of third
parties; BioTime’s ability to recruit and retain key personnel; and
BioTime’s ability to successfully integrate the operations of
Asterias into BioTime. 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. For a detailed description
of BioTime’s risks and uncertainties, you are encouraged to review
its documents filed with the SEC including its recent filings on
Form 8-K, Form 10-K and Form 10-Q. 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190502005299/en/
BioTime Inc. IRIoana C. Hone(ir@biotimeinc.com)(510)
871-4188
Solebury Trout IRGitanjali Jain
Ogawa(Gogawa@troutgroup.com)(646) 378-2949
Brooklyn ImmunoTherapeut... (AMEX:BTX)
Historical Stock Chart
From Jun 2024 to Jul 2024
Brooklyn ImmunoTherapeut... (AMEX:BTX)
Historical Stock Chart
From Jul 2023 to Jul 2024