Full Press Release Details
presentS UPDATED DATA FROM OPREGEN PHASE i/IIA CLINICAL STUDY AT THE ASSOCIATION FOR RESEARCH IN VISION AND OPHTHALMOLOGY
with OpRegen Continues to be Well Tolerated with Signs of Structural Improvement in the Retina Observed in Some
CA - May 2, 2019 - 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
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.
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.
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.
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.
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.
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.
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.