Asterias Biotherapeutics, a biotechnology company in the emerging field of regenerative medicine, announced that Atlanta-based Shepherd Center, a rehabilitation hospital for spinal cord injury and brain injury, has commenced enrollment for the phase I/IIa clinical trial of AST-OPC1 (oligodendrocyte progenitor cells) in newly injured patients with sensory and motor complete cervical spinal cord injury (SCI).
The phase I/IIa trial follows the successful completion of the phase I trial of AST-OPC1, which met its primary endpoints of safety and feasibility when administered to five patients with neurologically complete, thoracic SCI. Shepherd Center was a site in the phase I study and enrolled two of the five subjects in that study. Asterias intends to initiate enrollment for the phase I/IIa trial at up to seven additional sites in the coming months.
"The phase I study generated a strong package of data regarding the safety of AST-OPC1," said Donald Peck Leslie, M.D., medical director of Shepherd Center and Principal Investigator for this study site.
Richard G. Fessler, M.D., Ph.D., professor of neurological surgery at Rush University Medical Center and Principal Investigator for the phase I clinical trial, said, "There currently are no FDA-approved therapeutics or devices for the more than 12,000 individuals who sustain an SCI each year in the U.S. alone, or for the approximately 1.3 million Americans who are estimated to be living with an SCI. If AST-OPC1 could deliver even modest improvements in motor or sensory function, it would result in significant improvements in quality of life."
The phase I/IIa clinical trial is designed to assess safety and activity of escalating doses of AST-OPC1 for complete cervical SCI, the first targeted indication for AST-OPC1. The trial will be an open-label, single-arm study testing three escalating doses of AST-OPC1 in patients with sub-acute, C-5 to C-7, neurologically complete cervical SCI. These individuals have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs. AST-OPC1 will be administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging methods to assess the safety and activity of the product.
The initiation of this trial is in line with the recently announced initiative to accelerate the current timelines for the AST-OPC1 clinical program by approximately six months. In addition, the company plans to seek FDA concurrence to increase the robustness of the proof-of-concept in the phase I/IIa clinical trial by expanding enrollment from 13 patients to up to 40 patients. The company believes these changes will increase the statistical confidence of the safety and efficacy readouts, reduce the risks of the AST-OPC1 program and position the product for potential accelerated regulatory approvals.
Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the phase I/IIa clinical trial and other product development activities for AST-OPC1.
"The initiation of our phase I/IIa trial is a significant achievement for Asterias, putting us on track to achieve the accelerated timelines that we recently announced and moving us closer to realizing the value of AST-OPC1," said Pedro Lichtinger, president and CEO of Asterias. "Individuals with SCI have severe disabilities that can significantly shorten projected lifespan, impact quality of life and result in lifetime costs of care of $3 million to $4 million. AST-OPC1 has been shown to have multiple reparative functions that address the complex pathologies observed at the spinal cord injury site and improve function in animal models."