FREMONT, Calif., Feb. 4, 2016 /PRNewswire/ -- Asterias
Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company
focused on the emerging field of regenerative medicine, today
announced receipt of Orphan Drug Designation from the U.S. Food and
Drug Administration (FDA) for its product development candidate,
AST-OPC1, for the treatment of acute spinal cord injury.
Orphan Drug Designation is granted by the FDA Office of Orphan
Products Development to products that treat rare diseases. The FDA
defines rare diseases as those affecting fewer than 200,000 people
in the United States. Orphan Drug
Designation may provide the sponsor certain benefits and
incentives, including a period of marketing exclusivity for the
first marketing application if regulatory approval is received for
the designated indication, potential tax credits for certain
activities and waiver of certain administrative fees.
About AST-OPC1
AST-OPC1, an oligodendrocyte progenitor population derived from
human embryonic stem cells, has been shown in animals or 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 hind limb
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 level
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 spinal cord injuries, which represents the first targeted
population for registration trials. This trial, known as the
SCiStar trial, will test three escalating doses of AST-OPC1
administered at 14-30 days post-injury in patients with
neurologically complete cervical spinal cord injuries.
Asterias has concluded recruitment of the initial safety cohort of
the trial, in which three patients were administered a low dose of
2 million AST-OPC1 cells. The results of this cohort continue
to support a robust safety profile for AST-OPC1. Additionally
at 3 months post-injection the first patient in this cohort had
demonstrated neurological improvement progressing from a complete
ASIA Impairment Scale (AIS) A
injury to an incomplete AIS C injury. Recruitment is
currently underway in the second cohort of the study, in which five
patients will be administered a dose of 10 million AST-OPC1 cells,
the first of two dose cohorts designed to bracket the predicted
optimal dose range of AST-OPC1 based on the preclinical
studies.
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a leading biotechnology
company in the emerging field of regenerative medicine. The
company's proprietary, industry leading platforms are based on its
pluripotent stem cell and dendritic cell immunotherapy
technologies. Asterias is focused on developing therapies to treat
conditions in several medical areas where there is high unmet
medical need and inadequate available therapies. 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) has demonstrated
promise in a Phase 2 study in acute myelogenous leukemia. AST-VAC2
(antigen-presenting allogeneic dendritic cells) represents a second
generation, allogeneic approach to dendritic cell vaccines.
Additional information about Asterias can be found at
www.asteriasbiotherapeutics.com.
FORWARD-LOOKING STATEMENTS
Statements pertaining to future financial and/or operating 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's 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.