BioTime, Inc. (NYSE American and TASE: BTX), a clinical-stage
biotechnology company developing new cellular therapies, announced
today that Edward D. Wirth, III, M.D., Ph.D., Chief Medical Officer
of BioTime, will present at the 26th Annual American Society for
Neural Therapy and Repair (ASNTR) Annual Conference on April 26th,
2019 at 10:30am EDT as part of Session 6: “Spinal Cord Injury”. Dr.
Wirth’s presentation is entitled “Top-line 12-month Results from
the SCiStar Study - A Phase 1/2a Trial of Human Embryonic Stem
Cell-Derived Oligodendrocyte Progenitor Cells (OPC1) in Patients
with Subacute Cervical Spinal Cord Injury”. ASNTR will be held
April 25 – 27, 2019 at the Sheraton Sand Key Resort in Clearwater
Beach, Florida.
“We believe the primary goals of the SCiStar Study, which were
to observe the safety of OPC1 in cervical spinal cord injury
patients as well as other important metrics including related to
the optimal timing of OPC1 injection, tolerability of the
immunosuppression regimen, engraftment of OPC1 cells, and rates of
motor recovery observed among different study subpopulations, have
all been successfully achieved,” stated Dr. Wirth. “We now are in
the process of analyzing the full data set from the SCiStar Study
to inform how best to proceed with this promising program. We
expect to propose a clinical plan to the U.S. Food and Drug
Administration later this year and expect to share the outcome of
those discussions when they are available.”
“We appreciate the support of the California Institute for
Regenerative Medicine, the world’s largest institution dedicated to
bringing the future of cellular medicine closer to reality, whose
generous grant funding to date of $14.3 million has helped advance
the clinical development of our OPC1 program and generate these
encouraging clinical results in patients with traumatic spinal cord
injuries,” stated Brian M. Culley, Chief Executive Officer of
BioTime. “We look forward to continuing our partnership with CIRM
and will support their mission to accelerate stem cell treatments
to patients with unmet medical needs and fast-track the development
of the most promising stem cell technologies.”
The SCiStar Study is an open-label, single-arm trial testing
three sequential escalating doses of OPC1 administered 21 to 42
days post-injury, at up to 20 million OPC1 cells in 25 subjects
with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7)
acute spinal cord injuries (SCI). These individuals have
essentially lost all movement below their injury site and
experience severe paralysis of the upper and lower limbs. AIS-A
subjects have lost all motor and sensory function below their
injury site, while AIS-B subjects have lost all motor function but
may have retained some minimal sensory function below their injury
site. The primary endpoint in the SCiStar study was safety as
assessed by the frequency and severity of adverse events related to
OPC1, the injection procedure, and immunosuppression with
short-term, low-dose tacrolimus. Secondary outcome measures
included neurological functions as measured by upper extremity
motor scores and motor level on International Standards for
Neurological Classification of Spinal Cord Injury (ISNCSCI)
examinations at 30, 60, 90, 180, 270, and 365 days after injection
of OPC1.
Below are a summary of key findings from the SCiStar Study. A
copy of Dr. Wirth’s presentation will be available on the Events
section of BioTime’s website concurrent with his presentation at
ASNTR.
- Overall safety profile of OPC1 to
date is excellent
- Magnetic resonance imaging (MRI) scans
at 12 months post-injection of OPC1 has shown no evidence of
adverse changes in any of the 25 SCiStar study subjects treated
with OPC1.
- To date, there have been no unexpected
serious adverse events (SAEs) related to the OPC1 cells.
- No concerning safety issues and no
intraoperative complications have been noted.
- No SCiStar study subjects had worsening
of neurological function post-injection.
- No adverse findings observed on
follow-up MRI scans.
- Immunosuppression with tacrolimus (an
immunosuppressive drug utilized mainly after allogeneic organ
transplant to lower the risk of organ rejection) was
well-tolerated.
- Majority of SCiStar subjects who
received 10M or 20M OPC1 cells exhibited robust motor recovery in
upper extremities
- Three subjects (Cohort 1) received a
sub-therapeutic dose of 2M cells to evaluate the initial safety of
injecting OPC1 into lesions in the cervical spinal cord. All other
subjects (Cohorts 2-5) received 10M or 20M cells.
- At 12 months, 95% (21/22) of SCiStar
study subjects in Cohorts 2-5 recovered at least one motor level on
at least one side and 32% (7/22) of these subjects recovered two or
more motor levels on at least one side. The average improvement in
upper extremity motor score as measured by the ISNCSCI scale for
these subjects was 8.9 points.
- Notably, no SCiStar study subjects saw
decreased motor function following administration of OPC1 and
subjects either retained for 12 months the motor function recovery
seen through 6 months or experienced further motor function
recovery from 6 to 12 months.
- MRI scans consistent with durable
engraftment through 1 year post-injection
- All three SCiStar study subjects in
Cohort 1 and 95% (21/22) of SCiStar study subjects in Cohorts 2 to5
have MRI scans at 12 months consistent with the formation of a
tissue matrix at the injury site, which is encouraging evidence
that OPC1 cells have engrafted at the injury site and helped to
prevent cavitation, a destructive process that occurs within the
spinal cord following spinal cord injuries, and typically results
in permanent loss of motor and sensory function.
About OPC1
OPC1 is an oligodendrocyte progenitor cell (OPC) therapy
currently being tested in a Phase I/IIa clinical trial known as
SCiStar for the treatment of acute spinal cord injuries. OPCs are
naturally-occurring precursors to the cells which provide
electrical insulation for nerve axons in the form of a myelin
sheath. SCI occurs when the spinal cord is subjected to a severe
crush or contusion injury and typically results in severe
functional impairment, including limb paralysis, aberrant pain
signaling, and loss of bladder control and other body functions.
The clinical development of the OPC1 program has been partially
funded by a $14.3 million grant from the California Institute for
Regenerative Medicine. OPC1 has received Regenerative Medicine
Advanced Therapy (RMAT) designation for the treatment of acute SCI
and has been granted Orphan Drug designation from the U.S. Food and
Drug Administration (FDA).
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 timing
of when we propose a clinical plan to the U.S. Food and Drug
Administration and the sharing of the outcome of those discussions
when they are available, and that MRI results are supportive
evidence showing that OPC1 cells have durably engrafted to help
prevent cavitation at the injury site. 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190426005120/en/
BioTime Inc. IRIoana C. Hone(ir@biotimeinc.com)(510)
871-4188
Solebury Trout IRGitanjali Jain
Ogawa(Gogawa@troutgroup.com)(646) 378-2949
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