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.
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Overall safety profile of OPC1 to date is excellent
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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.
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To date, there have been no unexpected serious adverse events
(SAEs) related to the OPC1 cells.
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No concerning safety issues and no intraoperative complications
have been noted.
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No SCiStar study subjects had worsening of neurological function
post-injection.
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No adverse findings observed on follow-up MRI scans.
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Immunosuppression with tacrolimus (an immunosuppressive drug
utilized mainly after allogeneic organ transplant to lower the
risk of organ rejection) was well-tolerated.
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Majority of SCiStar subjects who received 10M or 20M OPC1 cells
exhibited robust motor recovery in upper extremities
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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.
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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.
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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.
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MRI scans consistent with durable engraftment through 1 year
post-injection
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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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