FREMONT, Calif., Aug. 31, 2016 /PRNewswire/ -- Asterias
Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company
focused on the emerging field of regenerative medicine, today
announced that its Data Monitoring Committee (DMC) has reviewed the
safety data from the initial cohort of three patients dosed with 2
million cells, and a subsequent five patients in the second cohort
dosed with 10 million cells, and has cleared the company to now
begin dosing a third cohort of 5-8 complete cervical injury
patients (AIS-A patients) with the highest dose of 20 million
cells. Concurrently, the study is also proceeding with
enrolling the first cohort of 5-8 sensory incomplete cervical
spinal cord injury patients (AIS-B patients), each of whom will be
administered 10 million cells.
The SCiStar study is an ongoing Phase 1/2a clinical trial funded
in part by a $14.3 million grant from
the California Institute for Regenerative Medicine (CIRM) and is
designed to evaluate the safety and efficacy of escalating doses of
AST-OPC1 in newly injured patients with sensory and motor complete
cervical spinal cord injury (SCI), as well as newly injured
patients with sensory incomplete SCI. These patients are
commonly referred to as AIS-A and AIS-B patients,
respectively. The results of the ongoing trial continue to
support a positive safety profile for AST-OPC1. There have
been no serious or unexpected adverse events related to AST-OPC1,
the administration procedure or the accompanying short course of
low-dose immunosuppression in any of the patients treated with
AST-OPC1, including five patients in an earlier Phase 1 trial with
neurologically complete thoracic SCI.
"The positive safety data in the previous phase 1 study and in
the ongoing phase 1/2a study gives us the confidence to now proceed
to administration of 20 million cells, which based on our
significant pre-clinical research is likely well within the dosing
range where we would expect to see clinically meaningful
improvement in these patients," said Dr. Edward Wirth, Chief Medical Officer of Asterias
Biotherapeutics.
The data set evaluating the efficacy results six months after
implantation of 10 million AST-OPC1 cells in complete cervical
spinal cord injury patients will be available in January 2017 and will focus on improvement in
physical functioning of the upper extremities (fingers, hands and
arms) of each treated patient utilizing scoring on the
International Standards for Neurological Classification of Spinal
Cord Injury (ISNCSCI scale). The published literature and
opinion leaders in the spinal cord injury field indicate that a two
motor level improvement in functioning utilizing this validated
scale is clinically meaningful for these patients and should be the
key measure in the evaluation of new therapies.
"According to the published scientific literature as well as key
opinion leaders that we speak to, a two motor level improvement in
physical functioning on the ISNCSCI scale can translate into a
significant improvement in quality of life, reduced need for daily
care and increased ability to live independently for patients with
cervical spinal cord injuries. We look forward to announcing the
six month follow up data from this 10 million cell cohort in
January 2017," commented Steve Cartt, President & Chief Executive
Officer.
About the SCiStar Trial
The SCiStar trial is testing three sequential escalating doses
of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as
many as 35 patients with sub-acute, C-5 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 retain some
minimal sensory function below their injury site. AST-OPC1 is
being administered 14 to 30 days post-injury. Patients will be
followed by neurological exams and imaging procedures to assess the
safety and activity of the product. 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).
Asterias previously announced that it had been granted FDA
clearance to expand patient enrollment in the Phase 1/2a clinical
trial from 13 patients to up to 35 patients, based on the continued
favorable safety profile observed in the ongoing clinical study.
The Company believes that this change will increase the statistical
confidence of the safety and efficacy readouts, reduce the risks of
the AST-OPC1 program and position the product for potential
accelerated regulatory approvals. 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.
More than 17,000 people sustain a spinal cord injury each year,
but there are no FDA-approved therapeutics or devices that could
potentially restore some function in individuals who have recently
sustained a spinal cord injury.
About AST-OPC1
AST-OPC1, an oligodendrocyte progenitor population derived from
human embryonic stem cells, 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. They also received low levels of
immunosuppression for the next 60 days. Delivery of AST-OPC1
was successful in all five subjects with no serious adverse events
associated with the administration of the cells, with AST-OPC1
itself, or the immunosuppressive regimen. No evidence of
rejection of AST-OPC1 was observed in detailed immune response
monitoring of all patients. In four of the five patients,
serial MRI scans indicated that reduced spinal cord cavitation may
have occurred. Based on the results of this study, Asterias
received approval from FDA to progress testing of AST-OPC1 to
patients with complete cervical spine injuries, which represents
the first targeted population for registration trials.
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a leading biotechnology
company in the emerging field of regenerative medicine. The
company's proprietary cell therapy programs are based on its
immunotherapy and pluripotent stem cell 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 oncology and neurology.
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 being evaluated
by Asterias for further development after demonstrating promise in
a Phase 2 study in Acute Myeloid Leukemia (AML) and completing a
successful end-of-Phase 2 meeting with the FDA. AST-VAC2
(antigen-presenting allogeneic dendritic cells) represents a second
generation, allogeneic immunotherapy. The company's research
partner, Cancer Research UK, plans to begin a Phase 1/2 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.
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SOURCE Asterias Biotherapeutics, Inc.