Treatment with OpRegen®
Continues to be Well Tolerated with Signs of Structural Improvement in
the Retina Observed in Some Patients
ALAMEDA, Calif.–(BUSINESS WIRE)–lt;a href="https://twitter.com/search?q=%24BTX&src=ctag" target="_blank"gt;$BTXlt;/agt; lt;a href="https://twitter.com/hashtag/AMD?src=hash" target="_blank"gt;#AMDlt;/agt;–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.
Contacts
BioTime Inc. IR
Ioana C. Hone
(ir@biotimeinc.com)
(510)
871-4188
Solebury Trout IR
Gitanjali Jain Ogawa
(Gogawa@troutgroup.com)
(646)
378-2949