- Current Readout Includes New Six Month Data for
Cohort 5 and Aggregate Trial Data -
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 AST-OPC1 in the
treatment of severe cervical spinal cord injury.
The SCiStar study is an open-label, single-arm
trial testing three escalating doses of AST-OPC1 in 25 subjects
with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7)
spinal cord injury. These individuals have lost essentially all
movement below their injury site and experience severe paralysis of
the upper and lower limbs. The SCiStar study consists of five
cohorts:
Cohort |
Injury Type; AST-OPC1 Dose |
# of Subjects |
Cohort 1 |
AIS-A; 2M AST-OPC1 cells (low dose for initial safety
evaluation) |
3 |
Cohort 2 |
AIS-A; 10M AST-OPC1 cells |
6 |
Cohort 3 |
AIS-A; 20M AST-OPC1 cells* |
6 |
Cohort 4 |
AIS-B; 10M AST-OPC1 cells |
6 |
Cohort 5 |
AIS-B; 20M AST-OPC1 cells* |
4 |
Total |
|
25 |
*One
subject from Cohort 3 and one subject from Cohort 5 were
administered 10 million cells. |
Each subject in the SCiStar study has now
completed a six month follow-up and the updated results for the
SCiStar study have shown the following:
- Positive Safety Profile - Asterias has dosed
25 subjects with AST-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 AST-OPC1 cells.
- Cell Engraftment - 100% (4/4) of Cohort 5
subjects have magnetic resonance imaging (MRI) scans at six months
consistent with the formation of a tissue matrix at the injury
site, which is encouraging evidence that AST-OPC1 cells have
engrafted at the injury site and helped to prevent cavitation.
Together with the previously reported results from Cohorts
2-4, the MRI results-to-date for 95% (21/22) of the Cohort 2-5
subjects provide supportive evidence that AST-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 - 100% (4/4) of Cohort
5 subjects have recovered at least one motor level on at least one
side through six months, with two subjects having recovered one
motor level bilaterally. At six months, 86% (19/22) of Cohort
2-5 subjects recovered at least one motor level on at least one
side and 18% (4/22) of these subjects recovered two or more motor
levels on at least one side. More detailed information on
improved motor function is provided in table format further
below.
“The results from the SCiStar study remain
encouraging as the six-month follow-up data continued to
demonstrate a positive safety profile and show that the AST-OPC1
cells are successfully engrafting in patients,” stated Ed Wirth,
Chief Medical Officer. “We are pleased that 19 of the 22
subjects dosed in Cohorts 2 through 5 have recovered at least one
motor level on at least one side through six months of follow-up
and four of these subjects have recovered two motor levels during
this same period. We expect to release the top-line 12-month
data for the study in the first quarter of 2019 and are actively
evaluating the design of a randomized controlled study for
OPC1.”
“The results are in-line with the data we reported earlier in
the year for cohorts 3 and 4 and the overall body of evidence to
date supports the primary safety objective for the study,”
commented Michael Mulroy, Chief Executive Officer. “There
remain no serious or unexpected adverse events related to OPC1 and
we believe the durable engraftment of the OPC1 cells, as evidenced
in the earlier results as well, is an important prerequisite to
seeing sustained clinical benefits. We expect to report the
12 month data on cohorts 3 and 4 later this quarter and the full
trial results in early 2019 and begin to have more formalized
discussions with FDA in the fourth quarter of 2018 on the next
clinical study design for OPC1. We are also in early
discussions with the California Institute for Regenerative Medicine
(CIRM) regarding a possible grant to partially fund the next
clinical study.”
AST-OPC1 Therapeutic
Platform
AST-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 AST-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.
Updated clinical data from the SCiStar study is
set forth in the tables below:
Safety
Asterias has dosed 25 subjects with AST-OPC1 in
the SCiStar study and a total of 30 subjects including the five
subjects from the previous Phase 1 safety trial. The
results-to-date, which include subjects from the Phase 1 safety
trial who have been followed for as long as seven years, continue
to support the safety of AST-OPC1. In particular, there have
been no serious, unexpected, adverse events related to AST-OPC1,
the injection procedure, or the drug used for immunosuppression in
any of the 30 subjects. Additionally, long-term follow up in
the Phase 1 safety trial with annual MRI scans through five years
post-injection of AST-OPC1 has shown no evidence of adverse changes
in any of the subjects treated with AST-OPC1.
Magnetic Resonance Imaging (MRI)
Data
The MRI results-to-date for 95% (21/22) of the
subjects in Cohorts 2-5 are consistent with formation of a tissue
matrix at the injury site, which is supportive evidence showing
that AST-OPC1 cells have durably engrafted at the injury site and
helped to prevent cavitation. Published data
indicates that about 50% of all subjects with spinal cord injuries
develop an injury cavity within six months following the spinal
cord injury. Meanwhile, for subjects with the severe
contusive spinal cord injuries that would meet the criteria for
inclusion in the SCiStar study, the percentage of subjects that
typically develop an injury cavity would be closer to
80%.
Upper Extremity Motor
Recovery
Improvements in upper extremity motor function
are being measured using the International Standards for
Neurological Classification of Spinal Cord Injury (ISNCSCI) scale,
widely used to quantify functional status of patients with spinal
cord injuries. Both patients and physicians consistently report
that improvements in upper extremity motor function are the most
desirable functional improvement target in the quadriplegic
population, since even relatively modest changes can potentially
have a significant impact on functional independence, quality of
life and cost of care. The SCiStar study is monitoring two separate
ISNCSCI measurements of upper extremity motor function. The upper
extremity motor score (UEMS), is a scale used to quantify motor
function at each of five upper extremity muscle groups driving arm
and hand function; these scores are also used to determine "motor
levels," which define the level within the cord above which a
subject has normal function. As suggested by existing
research, patients with severe spinal cord injuries that show two
motor levels of improvement on at least one side may regain the
ability to perform daily activities such as feeding, dressing and
bathing, which significantly reduces the overall level of daily
assistance needed for the patient and associated healthcare
costs.
One-Motor Level Recovery
Cohort |
Subjects recovering atleast one motor level onat least one side at
6months |
Subjects recovering atleast one motor level onat least one side at
12months |
Cohort 2 |
6/6 |
6/6 |
Cohort 3 |
4/6 |
TBD |
Cohort 4 |
5/6 |
TBD |
Cohort 5 |
4/4 |
TBD |
Cohorts 2-4 |
19/22 |
TBD |
Two-Motor Level Recovery
Cohort |
Subjects recovering atleast two motor levelson at least one side at
6months |
Subjects recovering atleast two motor levelson at least one side
at12 months |
Cohort 2 |
2/6 |
4/6 |
Cohort 3 |
1/6 |
TBD |
Cohort 4 |
1/6 |
TBD |
Cohort 5 |
0/4 |
TBD |
Cohorts 2-4 |
4/22 |
TBD |
Upper Extremity Motor Score
Cohort |
Average UEMSimprovement at 6months |
Average UEMSimprovement at 12months |
Cohort 2 |
9.7 |
12.3 |
Cohort 3 |
6.0 |
TBD |
Cohort 4 |
5.5 |
TBD |
Cohort 5 |
5.8 |
TBD |
Cohorts 2-5 |
6.8 |
TBD |
Anticipated 2018-19 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 the following data readouts later this
year or early in 2019:
- 12 month update for Cohorts 3 and 4 in the third quarter of
2018.
- 24 month update for Cohort 2 in the third or fourth quarter of
2018.
- 12 month update for the entire SCiStar study, including Cohort
5, in the first quarter of 2019.
The Company will provide an overview of the
results on its second quarter 2018 operating results conference
call, scheduled for August 9, 2018.
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 three clinical-stage
programs which have the potential to address areas of very high
unmet medical need in the fields of neurology and oncology.
AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase
1/2a dose escalation clinical trial in spinal cord injury. AST-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
AST-VAC2 in non-small cell lung cancer. AST-VAC1
(antigen-presenting autologous dendritic cells) is an autologous
cancer immunotherapy with promising efficacy and safety data from
an earlier Phase 2 study in Acute Myeloid Leukemia (AML).
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 AST-OPC1
AST-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 AST-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 AST-OPC1
administered at up to 20 million AST-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. AST-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.
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.
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 AST-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 STATEMENTS
Statements 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," or “possible”) 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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