Asterias Biotherapeutics, Inc. (NYSE American: AST), a
biotechnology company dedicated to developing cell-based
therapeutics to treat neurological conditions associated with
demyelination and cellular immunotherapies to treat cancer, today
provided additional data from the Company’s ongoing Phase 1/2a
SCiStar study designed to evaluate the safety and potential
efficacy of OPC1 in the treatment of severe cervical spinal cord
injury. All 6 subjects from Cohort 2 in the SCiStar study have now
completed a 24-month follow-up as part of the study’s long-term
follow-up protocol and each subject either retained the motor
function recovery seen through 12 months or saw further motor
function recovery from 12 to 24 months.
“While the primary endpoint for the SCiStar
trial was 12 months, we are further encouraged by this additional
follow-up data that shows both durable engraftment and motor
function recovery being maintained or improved upon at 24 months,”
commented Ed Wirth, Chief Medical Officer. “We believe the
primary goal of SCiStar, which was to observe the safety of OPC1 in
cervical spinal cord injury patients and to accumulate data related
to important factors such as optimal dosing levels, the
immunosuppression regimen, engraftment of the cells, and rates of
motor recovery observed among different study subpopulations, have
been successfully achieved and increases our confidence as we
prepare to meet the FDA later this year to discuss the next trial
design.”
Below are a summary of key findings at 24 months
for the Cohort 2 subjects:
- Positive Safety Profile – MRI scans at 24
months post-injection of OPC1 has shown no evidence of adverse
changes in any of the Cohort 2 subjects treated with OPC1.
Additionally, Asterias has dosed 25 subjects with OPC1 in the
SCiStar study and a total of 30 subjects including the five
subjects from a previous Phase 1 safety trial in thoracic spinal
cord injury who have been followed for as long as seven
years. To date, there have been no serious adverse events
(SAEs) related to the OPC1 cells.
- Cell Engraftment – 100% (6/6) of Cohort 2
subjects have magnetic resonance imaging (MRI) scans at 24 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. The MRI
results provide supportive evidence that OPC1 cells have durably
engrafted at the injury site and helped to prevent
cavitation. Cavitation is a destructive process that occurs
within the spinal cord following spinal cord injuries, and
typically results in permanent loss of motor and sensory function.
Additionally, a patient with cavitation can develop a condition
known as syringomyelia, which results in additional neurological
and functional damage to the patient and can result in chronic
pain.
- Improved Motor Function – At 24 months, 100%
(6/6) of Cohort 2 subjects have recovered at least one motor level
on at least one side. At 24 months, 83% (5/6) of these subjects
have recovered two or more motor levels on at least one side,
compared to 67% (4/6) at 12 months after administration of the OPC1
cells. At 24 months, the average improvement in upper
extremity motor function for Cohort 2 subjects was 13.0 points,
compared to 12.3 points at 12 months after administration of the
OPC1 cells. At 24 months, one subject had recovered two motor
levels bilaterally and another subject had recovered three motor
levels on one side.**One subject had recently underwent an elective
surgery that restrained his ability to fully use one of the muscle
groups evaluated during his 24 month follow-up and thus this muscle
group was rated as not testable. The subject had otherwise
retained the motor function recovery seen through 12 months for all
muscle groups that could be evaluated during the 24 month
follow-up. For purposes of determining the Cohort’s average
improvement in upper extremity motor function at 24 months this
subject’s 12 month score was used for the muscle group that was not
testable.
OPC1 Therapeutic Platform
OPC1, an oligodendrocyte progenitor cell
population derived from human embryonic stem cells, has been shown
in preclinical testing in animals and in vitro to have three
potentially reparative functions that address the complex
pathologies observed in demyelination disorders, such as spinal
cord injury and multiple neurodegenerative diseases, including
multiple sclerosis and white matter stroke. These potential
reparative functions of OPC1 include the production of neurotrophic
factors, the stimulation of vascularization, and the induction of
remyelination of denuded axons, all of which are critical for
survival and regrowth of—and conduction of nerve impulses
through—axons at the injury site.
Each year in the United States, more than 17,000
people suffer a severe, debilitating spinal cord injury. As of
2016, the National Spinal Cord Injury Statistical Center reported
that approximately 4,500 of these new spinal cord injuries annually
in the U.S. are AIS-A, AIS-B, or AIS-C patients with C-4 to C-7
spinal cord injuries (https://www.nscisc.uab.edu/). These injuries
can be devastating to quality of life and ability to function
independently. Lifetime healthcare costs for these patients can
often approach $5 million. Improvements in arm, hand, and finger
functional capabilities in these patients can result in
meaningfully lower healthcare costs, significant improvements in
quality of life, greater ability to engage in activities of daily
living, and increased independence.
Anticipated Data Readouts for the
SCiStar Study
Asterias has completed enrollment and dosing in
all five of its planned SCiStar study cohorts. The Company intends
to report 12 month results for the entire SCiStar study in the
first quarter of 2019.
About Asterias
Biotherapeutics
Asterias Biotherapeutics, Inc. is a
biotechnology company dedicated to developing cell-based
therapeutics to treat neurological conditions associated with
demyelination and cellular immunotherapies to treat cancer.
Asterias is presently focused on advancing two clinical-stage
programs which have the potential to address areas of very high
unmet medical need in the fields of neurology and oncology. OPC1
(oligodendrocyte progenitor cells) is currently in a Phase 1/2a
dose escalation clinical trial in spinal cord injury. VAC2
(antigen-presenting allogeneic dendritic cells) is an allogeneic
cancer immunotherapy. The Company's research partner, Cancer
Research UK, has commenced a first-in-human clinical trial of VAC2
in non-small cell lung cancer. Asterias is also sponsoring
pre-clinical work in two conditions with a demyelinating component:
Multiple Sclerosis and White Matter Stroke, and is evaluating other
cancer indications where its immunotherapy platform could provide
therapeutic benefit. Additional information about Asterias can be
found at www.asteriasbiotherapeutics.com.
About OPC1
OPC1, an oligodendrocyte progenitor cell
population derived from human embryonic stem cells, has been shown
in preclinical testing in animals and in vitro to have three
potentially reparative functions that address the complex
pathologies observed in demyelination disorders, such as spinal
cord injuries, and multiple neurodegenerative diseases, including
multiple sclerosis and white matter stroke. These potential
reparative functions of OPC1 include the production of neurotrophic
factors, the stimulation of vascularization, and the induction of
remyelination of denuded axons, all of which are critical for
survival and regrowth of—and conduction of nerve impulses
through—axons at the injury site.
About the SCiStar Trial
The SCiStar trial is an open-label, single-arm
trial testing three sequential escalating doses of OPC1
administered 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) 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. OPC1 is administered 21 to 42 days post-injury.
Subjects will be followed by neurological exams and imaging
procedures to assess the safety and activity of the product.
The SCiStar study consists of five cohorts:
|
|
Cohort |
Injury Type; OPC1 Dose |
# of Subjects |
|
|
Cohort 1 |
AIS-A; 2M OPC1 cells (low dose for initial safety evaluation) |
3 |
|
|
Cohort 2 |
AIS-A; 10M OPC1 cells |
6 |
|
|
Cohort 3 |
AIS-A; 20M OPC1 cells* |
6 |
|
|
Cohort 4 |
AIS-B; 10M OPC1 cells |
6 |
|
|
Cohort 5 |
AIS-B; 20M OPC1 cells* |
4 |
|
|
Total |
|
25 |
*One subject from Cohort 3 and one subject from Cohort
5 were administered 10 million cells. |
Asterias has received a Strategic Partnerships
Award grant from the California Institute for Regenerative
Medicine, which provided $14.3 million of non-dilutive funding for
the Phase 1/2a clinical trial and other product development
activities for OPC1.
Additional information on the Phase 1/2a trial,
including trial sites, can be found at www.clinicaltrials.gov,
using Identifier NCT02302157, and at the SCiStar Study Website
(www.SCiStar-study.com).
FORWARD-LOOKING
STATEMENTSStatements pertaining to future financial and/or
operating and/or clinical research results, future growth in
research, technology, clinical development, and potential
opportunities for Asterias, along with other statements about the
future expectations, beliefs, goals, plans, or prospects expressed
by management constitute forward-looking statements. Any statements
that are not historical fact (including, but not limited to
statements that contain words such as "will," "believes," "plans,"
"anticipates," "expects," "estimates") should also be considered to
be forward-looking statements. Forward-looking statements involve
risks and uncertainties, including, without limitation, risks
inherent in the development and/or commercialization of potential
products, uncertainty in the results of clinical trials or
regulatory approvals, need and ability to obtain future capital,
and maintenance of intellectual property rights. Actual results may
differ materially from the results anticipated in these
forward-looking statements and as such should be evaluated together
with the many uncertainties that affect the businesses of Asterias,
particularly those mentioned in the cautionary statements found in
Asterias' filings with the Securities and Exchange Commission.
Asterias disclaims any intent or obligation to update these
forward-looking statements.
Contacts:Investor
Relations(510) 456-3892InvestorRelations@asteriasbio.comorEVC
Group, Inc.Michael Polyviou/Todd Kehrli(732)
232-6914mpolyviou@evcgroup.com; tkehrli@evcgroup.com
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