FREMONT, Calif., May 24, 2016 /PRNewswire/ -- Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company with three clinical-stage development programs focused on the emerging field of regenerative medicine, today announced new positive long-term follow-up results from its Phase 1 clinical trial assessing the safety of AST-OPC1 (oligodendrocyte progenitor cells) in patients with spinal cord injury. The results showed that in four of the five patients, serial MRI scans performed throughout the 4 to 5 year follow-up period indicate that reduced spinal cord cavitation occurred and that AST-OPC1 appeared to have positive long-term effects on reducing spinal cord tissue deterioration. The company also filed a new patent application with the United States Patent and Trademark Office (USPTO) that included this new safety data.
In the Phase 1 clinical trial, five patients with neurologically complete thoracic spinal cord injury, as classified by the American Spinal Injury Association Impairment Scale (AIS), were administered a relatively low dose of two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. The aim of this first human clinical study was to evaluate the safety of AST-OPC1 when administered at a low dose. The patients also received low-level immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five patients with no serious adverse events associated with the administration of the cells, with AST-OPC1 itself, or with 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 occurred. Cavitation is a destructive process which takes place within the spinal cord following spinal cord injuries and typically results in permanent loss of a patient's motor function. The patients in the study have now been followed for 4 to 5 years through numerous clinical visits, MRIs, and neurological assessments.
"These new follow-up results are very encouraging and provide important further support for expansion of the ongoing Phase 1/2a clinical study in patients with complete cervical spinal cord injuries announced just last week," said Steve Cartt, President and Chief Executive Officer. "We are continuing to enroll patients in the second dose cohort of the current Phase 1/2a trial. Patients in this cohort are receiving a significantly higher dose of 10 million cells, which we believe corresponds to the doses that showed efficacy in animal studies."
Asterias believes that this data provides important confirmation of the favorable long-term safety profile of AST-OPC1.
"This new long term follow-up data continues to support the general safety of AST-OPC1 and indicate minimal risk of the transplanted cells having unintended effects," stated Dr. Edward Wirth III, Chief Medical Officer. "In detailed immune response monitoring of patients, the results are consistent with long-term cell engraftment, immune system tolerability, and an absence of adverse effects. In short, AST-OPC1 does not appear to present any immunological or other long-term safety issues when administered to patients suffering from spinal cord injuries."
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 the survival and regrowth of axons at the injury site, as well as for the conduction of nerve impulses through those axons. 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.
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 Phase 1/2a trial, known as the SCiStar trial, is in progress and 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 three months post-injection, the first patient in this cohort had demonstrated neurological improvement progressing from a complete AIS A injury to an incomplete AIS C injury. Enrollment is currently in progress in the second cohort of the study, in which five patients will be administered a dose of 10 million AST-OPC1 cells. This cohort is the first of two dose cohorts receiving doses in the predicted efficacious range based on preclinical animal studies. In addition, Asterias has also recently been granted FDA clearance to expand patient enrollment in this 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.
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-VAC1 (antigen-presenting autologous dendritic cells) demonstrated promise in a Phase 2 study in acute myelogenous leukemia (AML) and completed a successful end-of-Phase 2 meeting with the FDA in advance of initiating planning for a single pivotal Phase 3 AML study. 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. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. Additional information about Asterias can be found at www.asteriasbiotherapeutics.com.
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' 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.