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.
