FREMONT, Calif., Aug. 30, 2017 (GLOBE NEWSWIRE) -- Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, today announced the publication of a manuscript in the peer-reviewed journal Stem Cells Translational Medicine describing new efficacy and safety data from a preclinical study of AST-OPC1 that were submitted to the U.S. Food and Drug Administration (FDA) as part of an Investigational New Drug Application. AST-OPC1 is currently being investigated in the company's ongoing SCiStar Phase 1/2a clinical trial in patients with severe cervical spinal cord injury (SCI).
The preclinical results showed that AST-OPC1 cells improved locomotor performance after cervical spinal cord injury and that this functional improvement was associated with reduced parenchymal cavitation and increased sparing of myelinated axons within the injury site. In addition, AST-OPC1 cells exhibited limited migration within the cervical spinal cord and did not cause any adverse clinical observations, toxicities, allodynia or tumors.
“The preclinical results support the potential efficacy and general safety of AST-OPC1 for patients with severe cervical spinal cord injuries, and indicate minimal risk of the transplanted cells reaching unintended locations,” said Dr. Casey C. Case, Senior Vice President Research and Preclinical Development. “In addition, the results indicate that AST-OPC1, when administered during the subacute phase of spinal cord injuries, can act through multiple repair pathways that are relevant to helping patients with these injuries, including trophic factor signaling, cavity reduction, and stimulation of axon outgrowth and myelination.”
The preclinical results are supportive of the results to date observed in the SCiStar study. Asterias has enrolled more than twenty patients in the SCiStar study and more than twenty-five patients have been administered AST-OPC1 after including patients from a previous Phase 1 safety trial. In June 2017, Asterias reported 9-month data from the SCiStar study’s second dose cohort (AIS-A patients dosed 10 million cells). Subjects in this cohort have shown meaningful improvements in arm, hand and finger function through 9-months. Results to date from the study have also continued to support the safety of AST-OPC1.
The publication, titled "Human Embryonic Stem Cell-Derived Oligodendrocyte Progenitor Cells: Preclinical Efficacy and Safety in Cervical Spinal Cord Injury," appeared online ahead of the print edition of Stem Cells Translational Medicine. The efficacy and safety testing was conducted in nude rats subjected to cervical SCI.
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 four cohorts of this study. Results to date have continued to support the safety of AST-OPC1. 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.