Full Press Release Details
Subsidiary Asterias Biotherapeutics, Inc. Announces a $14.3 Million
Strategic Partnership Award from the California Institute for
Regenerative Medicine
Supports Phase 1/2a Dose Escalation Clinical Trial of AST-OPC1 in
Cervical Spinal Cord Injury
Provides for the Expansion of AST-OPC1 Clinical Trial Originally
Initiated by Geron Corporation into Target Population for Pivotal
MENLO PARK, Calif. & ALAMEDA, Calif.--(BUSINESS WIRE)--May 30,
2014--Asterias Biotherapeutics, Inc. ("Asterias"), a subsidiary of
BioTime, Inc. (NYSE MKT: BTX), today announced that it has been awarded
a $14.3 million Strategic Partnership III grant entitled "A Phase 1/2a
Dose Escalation Study of AST-OPC1 in Patients with Cervical Sensorimotor
Complete Cervical Spinal Cord Injury" from the California Institute for
Regenerative Medicine ("CIRM"). The award provides funding for Asterias
to reinitiate clinical development of AST-OPC1 in subjects with spinal
cord injury and to expand clinical testing of escalating doses in the
target population intended for future pivotal trials.
AST-OPC1 is a population of cells derived from human embryonic stem
cells (hESCs) that contains oligodendrocyte progenitor cells (OPCs).
OPCs and their mature derivatives called oligodendrocytes provide
critical functional support for nerve cells in the spinal cord and
brain. Asterias recently presented the results from phase 1 clinical
trial testing of a low dose of AST-OPC1 in patients with
neurologically-complete thoracic spinal cord injury. The results showed
that AST-OPC1 was successfully delivered to the injured spinal cord
site. Patients followed 2-3 years after AST-OPC1 administration showed
no evidence of serious adverse events associated with the cells in
detailed follow-up assessments including frequent neurological exams and
MRIs. Immune monitoring of subjects through one year
post-transplantation showed no evidence of antibody-based or cellular
immune responses to AST-OPC1. In four of the five subjects, serial MRI
scans performed throughout the 2-3 year follow-up period indicate that
reduced spinal cord cavitation may have occurred and that AST-OPC1 may
have had some positive effects in reducing spinal cord tissue
deterioration. There was no unexpected neurological degeneration or
improvement in the five subjects in the trial as evaluated by the
International Standards for Neurological Classification of Spinal Cord
Injury (ISNCSCI) exam.
The Strategic Partnership III grant from CIRM will provide funding to
Asterias to support the next clinical trial of AST-OPC1 in subjects with
spinal cord injury, and for Asterias' product development efforts to
refine and scale manufacturing methods to support later-stage trials and
eventually commercialization. CIRM funding will be conditional on FDA
approval for the trial, completion of a definitive agreement between
Asterias and CIRM, and Asterias' continued progress toward the
achievement of certain pre-defined project milestones.
"These programs help bring together the most rigorous scientific
research with companies that know how to do clinical trials and move
therapies through the regulatory process. It's a partnership with a
simple goal, get the most promising therapies to patients as quickly as
possible," said Jonathan Thomas, Ph.D., J.D., Chair, Governing Board of
CIRM. "If this therapy can achieve even very modest improvements for
patients, it could have an enormous impact on the quality of their life,
and the lives of their families."
"We are preparing to initiate the dose escalation Phase 1/2a clinical
trial of AST-OPC1 in patients with cervical injuries in 6-9 months
subject to FDA clearance," stated Edward Wirth III M.D., Ph.D.,
Asterias' Chief Translational Officer. "Achievements in this
CIRM-supported program could also help accelerate further development of
AST-OPC1 in other neurodegenerative diseases such as stroke and multiple
sclerosis. We are currently evaluating the function of AST-OPC1 in
nonclinical models of these diseases."
"This award provides significant non-dilutive funding to accelerate the
development of AST-OPC1 for patients with spinal cord injury. Given the
lack of any approved therapies for spinal cord injury and the high level
of disability, substantial costs of care, and shorter life expectancy of
injured individuals, AST-OPC1 has the potential to address a substantial
unmet medical need in this condition," stated Katharine Spink, Ph.D.,
Asterias' Chief Operating Officer. "We look forward to working in
partnership with CIRM to bring this important therapy to patients."
More information about AST-OPC1 and Asterias is available on Asterias'
website, www.asteriasbiotherapeutics.com and in the company's
amended S-1 filed with the Securities and Exchange Commission.
About Spinal Cord Injury and AST-OPC1
Over 12,000 individuals suffer a spinal cord injury (SCI) each year in
the United States alone, and approximately 1.3 million Americans are
estimated to be living with a spinal cord injury. Traumatic SCI most
commonly impacts individuals in their 20s and 30s, resulting in a high
level of permanent disability in young and previously healthy
individuals. Individuals with SCI not only have impaired limb function,
but suffer from a wide range of additional disabilities which can each
significantly impact quality of life, and can even be life threatening
in some instances. According to the National Spinal Cord Injury
Statistical Center, the life expectancy of an individual suffering a
cervical spinal cord injury at age 20 is 20-25 years lower than that of
a similarly aged individual with no SCI.
In addition to its dramatic impact on quality of life for patients and
their families, SCI is responsible for tremendous costs to society. At
one year post injury, only 11.8% of SCI patients are employed, and fewer
than 35% are employed even at more than twenty years post-injury.
Additionally, many patients require help with activities of daily living
such as feeding and bathing. As a result, the lifetime cost of care for
SCI patients are enormous; a recent paper estimated lifetime costs of
care for a patient sustaining a cervical SCI at age 25 at $4.2 million.
Overall, it has been estimated that SCIs cost the U.S. over $14.5
billion per year in direct medical costs and disability support, plus an
additional $5.5 billion in lost productivity.
There are currently no approved therapies for the treatment of SCI, and
the complex pathology of the injury is unlikely to be addressed by a
traditional small molecule or protein therapeutic. AST-OPC1, an