The US Food and Drug Administration (FDA) has granted clearance to Asterias Biotherapeutics for the initiation of a Phase I/IIa clinical trial of its product, AST-OPC1, to treat patients with complete cervical spinal cord injury.
The approved Phase I/IIa trial follows the completion of the Phase I clinical study of the AST-OPC1, which is a group of cells derived from human embryonic stem cells (hESCs) that contains oligodendrocyte progenitor cells (OPCs).
The new trial is designed to evaluate the safety and activity of escalating doses of AST-OPC1 in these patients.
Asterias president and CEO Pedro Lichtinger: “We would like to acknowledge the scientists, clinical investigators, and FDA for working with us to develop AST-OPC1.
“The new trial is designed to evaluate the safety and activity of escalating doses of AST-OPC1 in these patients.”
“We are especially enthusiastic about working with our new partner, CIRM, in executing this clinical trial.
“The FDA clearance provides Asterias with imminent access to the previously announced $14.3m CIRM grant, which provides non-dilutive funding to support both the clinical trial and other product development activities for AST-OPC1.”
The FDA clearance of the Phase I/IIa trial is based on results from the Phase I trial, which met its primary endpoints of safety and feasibility when administered to five patients with neurologically complete, thoracic spinal cord injury.
During the Phase I trial, these five patients were given a low dose of two million AST-OPC1 cells and have been followed to date for two to three years.
In the new open-label, single-arm Phase I/IIa trial, three escalating doses of AST-OPC1 will be evaluated in 13 patients with subacute, C5-C7, neurologically complete cervical spinal cord injury.
Patients participating in the trial have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs.
During the trial, AST-OPC1 will be administered 14 to 30 days post-injury and patients will be followed by neurological exams to evaluate the safety and activity of the product.
The company intends to initiate patient enrolment in the Phase I/IIa trial during the first quarter of 2015.