FREMONT, Calif., July 10, 2017 /PRNewswire/ -- Asterias
Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company
pioneering the field of regenerative medicine, today announced that
the U.S. Food and Drug Administration (FDA) has accepted the
company's amendment to the clinical research protocol for its
ongoing AST-OPC1 SCiStar Phase 1/2a clinical trial in motor
complete cervical spinal cord injury (SCI). The amendment expands
the eligibility criteria to include patients with a C-4 spinal cord
injury and extends the dosing window from 14 to 30 days to 21 to 42
days post-injury.
"The FDA's decision to allow Asterias to enroll qualified
patients with C-4 level injuries is the result of the data
supporting the safety of both AST-OPC1 and the procedure to inject
the cells, and means that the second most common cervical spinal
cord injury population can now be eligible to receive AST-OPC1,"
said Dr. Edward Wirth, Chief Medical
Officer of Asterias. "The overall changes to the study protocol
will enhance our ability to enroll qualified patient candidates for
our current SCiStar study and we also expect the changes to help
enrollment rates in a future, larger clinical study."
The protocol amendment will expand patient eligibility and
enable study investigators to administer AST-OPC1 to patients with
injuries at one vertebral level higher than the trial's previous
C-5 limitation, to the fourth cervical vertebra down, known as C-4,
near the middle of the neck. A C-4 cervical level injury, the
second most common level of SCI in the SCiStar study's targeted
patient population, generally means that the injured person is
paralyzed from the neck down and requires round-the-clock care. The
lifetime direct costs of care for a patient suffering a high
cervical spinal cord injury, such as a C-4 spinal cord injury, can
approach $5 million. As
suggested by existing research, if these patients can show two
motor levels of improvement on at least one side they may regain
the ability to perform daily activities such as feeding, dressing
and bathing, which increases their quality of life and independence
and significantly reduces the overall level of required daily
assistance and associated healthcare costs for these patients.
In addition, the amendment to inclusion parameters will also
expand the dosing window to 21 to 42 days after a patient's spinal
cord injury occurs, providing study investigators more time to
screen patients to determine if they are eligible to participate in
the SCiStar study. The expansion of the dosing window is supported
by recent preclinical research that indicated AST-OPC1 cells can
durably engraft at a patient's injury site when administered up to
two months after the date of injury.
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 as many as 35 patients with subacute,
C-4 to C-7, motor complete (AIS-A or AIS-B) cervical SCI. These
individuals have essentially lost all movement below their injury
site and experience severe paralysis of the upper and lower limbs.
AIS-A patients have lost all motor and sensory function below their
injury site, while AIS-B patients have lost all motor function but
may have retained some minimal sensory function below their injury
site. AST-OPC1 is being administered 21 to 42 days post-injury.
Patients will be followed by neurological exams and imaging
procedures to assess the safety and activity of the product.
The study is being conducted at six centers in the U.S. and the
company plans to increase this to up to 12 sites to accommodate the
expanded patient enrollment. Clinical sites involved in the study
include the Medical College of
Wisconsin in Milwaukee,
Shepherd Medical Center in Atlanta, University of
Southern California (USC)
jointly with Rancho Los Amigos National Rehabilitation Center in
Los Angeles, Indiana University, Rush
University Medical Center in Chicago and Santa Clara Valley Medical Center
in San Jose jointly with
Stanford University.
Asterias has received a Strategic Partnerships Award grant from
the California Institute for Regenerative Medicine, which provides
$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).
About AST-OPC1
AST-OPC1, an oligodendrocyte progenitor population derived from
human embryonic stem cells originally isolated in 1998, has been
shown in animals and in vitro to have three potentially reparative
functions that address the complex pathologies observed at the
injury site of a spinal cord injury. These activities of AST-OPC1
include production of neurotrophic factors, stimulation of
vascularization, and induction of remyelination of denuded axons,
all of which are critical for survival, regrowth and conduction of
nerve impulses through axons at the injury site. In preclinical
animal testing, AST-OPC1 administration led to remyelination of
axons, improved hindlimb and forelimb locomotor function, dramatic
reductions in injury-related cavitation and significant
preservation of myelinated axons traversing the injury site.
In a previous Phase 1 clinical trial, five patients with
neurologically complete, thoracic spinal cord injury were
administered two million AST-OPC1 cells at the spinal cord injury
site 7-14 days post-injury. Based on the results of this study,
Asterias received clearance from FDA to progress testing of
AST-OPC1 to patients with cervical spine injuries in the current
SCiStar study, which represents the first targeted population for
registration trials. Asterias has completed enrollment in the
first two cohorts of this study. Results to date have continued to
support the safety of AST-OPC1, with no serious adverse events
related to AST-OPC1 or its administration. Additionally,
Asterias has recently reported results suggesting reduced
cavitation and improved motor function in patients administered
AST-OPC1 in the SCiStar trial.
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a biotechnology company
pioneering the field of regenerative medicine. The company's
proprietary cell therapy programs are based on its pluripotent stem
cell and immunotherapy platform technologies. 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-VAC1 (antigen-presenting
autologous dendritic cells) is undergoing continuing development by
Asterias based on promising efficacy and safety data from a Phase 2
study in Acute Myeloid Leukemia (AML), with current efforts focused
on streamlining and modernizing the manufacturing process. AST-VAC2
(antigen-presenting allogeneic dendritic cells) represents a second
generation, allogeneic cancer immunotherapy. The company's research
partner, Cancer Research UK, plans to begin a Phase 1/2a clinical
trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional
information about Asterias can be found at
www.asteriasbiotherapeutics.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") 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.
View original
content:http://www.prnewswire.com/news-releases/asterias-biotherapeutics-receives-fda-clearance-to-enroll-c-4-patients-in-scistar-study-300485009.html
SOURCE Asterias Biotherapeutics, Inc.