Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company focused on the emerging field of regenerative medicine, today announced that its Data Monitoring Committee has reviewed the initial safety data from all three patients in the first cohort and has recommended dose escalation to the second cohort. Recruitment for the second cohort will begin immediately, with a planned enrollment of five patients who will each receive 10 million cells of AST-OPC1 (oligodendrocyte progenitor cells).
The SCiStar Study is a Phase 1/2a clinical trial evaluating the safety and activity of escalating doses of AST-OPC1 in newly injured patients with sensory and motor complete cervical spinal cord injury (SCI). The three patients in the first cohort were administered a low dose of 2 million AST-OPC1 cells and have completed at least 30 days of follow up for safety. The results of the trial continue to support a positive safety profile for AST-OPC1, with no serious adverse events observed to date in any of the three treated patients.
The first patient in this cohort was dosed at Shepherd Center in Atlanta and has demonstrated additional neurological improvement at 3-months post-injection. This patient had previously progressed from a complete ASIA Impairment Scale (AIS) A injury to an incomplete AIS B injury, and improved further to an AIS C injury at the 3-month assessment. The principal investigator at Shepherd Center, Dr. Donald Peck Leslie, said, "This progress in the first patient is very encouraging and is observed in fewer than 5 percent of our AIS A patients at this stage of their recovery."
The second and third patients received AST-OPC1 at Rush University Medical Center, and have completed safety follow up for 2 months and 1 month, respectively. AST-OPC1 was administered to both patients by the lead neurosurgeon for the clinical trial, Dr. Richard Fessler, Professor of Neurological Surgery at Rush.
Dr. Fessler stated, "I am very encouraged by the excellent safety data that we have observed in our two patients to date and I look forward to the dose escalation phase of the study."
Dr. Edward Wirth, Chief Medical Officer of Asterias, said "We are pleased with the confirmation of safety in the first dose cohort and are excited to begin recruitment of patients who will receive the higher dose of 10 million AST-OPC1 cells, which we believe corresponds to the doses that showed efficacy in animal studies."
The open-label, single-arm trial is being conducted at three centers currently and will include up to twelve centers in the United States. "We are encouraged by the demonstrated safety and the performance improvement in this initial low-dose cohort of AST-OPC1," commented Pedro Lichtinger, President and CEO of Asterias. "The escalation of dosing in this Phase 1/2a trial is a significant advancement for our AST-OPC1 development program in that we will now begin to study the product at the doses that we believe have the most likely potential to bring benefit to patients."
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