CARLSBAD, Calif.– Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today provided an update on the clinical advancement of OPC1, its investigational allogeneic oligodendrocyte progenitor cell (OPC) transplant therapy for the treatment of spinal cord injury (SCI). Following feedback received from an interaction held with the U.S. Food and Drug Administration (FDA) last week under the FDA’s Regenerative Medicine Advanced Therapy (RMAT) program, Lineage intends to submit an amendment to its Investigational New Drug application (IND) for OPC1 to support a Phase 1 clinical study to evaluate the safety and performance of Neurgain Technologies Inc.’s Parenchymal Spinal Delivery System (“Neurgain PSD system”) to deliver OPC1 cells to the spinal cord. In February, the Company entered into an exclusive option and license agreement with Neurgain to evaluate its novel PSD system in both preclinical and clinical settings. The IND amendment is expected to be submitted to the FDA in the fourth quarter of 2021. The data from the Phase 1 clinical study is intended to validate the Neurgain PSD system for use in a late-stage clinical study, expected to begin in 2022 following the completion of the Phase 1 study.
“It is a privilege to report that our novel OPC1 program will be returning to clinical testing earlier than anticipated. There currently are few opportunities for SCI patients to participate in clinical trials, so we are excited to re-engage with these patients and their advocacy community as part of our efforts to improve outcomes for individuals with this debilitating condition, for which there are no FDA-approved treatments,” stated Brian M. Culley, Lineage’s CEO. “In the past 18 months, we have significantly increased the purity and production scale of the OPC1 cells utilized in a prior clinical study. This improved production process has been transferred to our in-house Current Good Manufacturing Practice (cGMP) suite and will support production of clinical study material for later-stage clinical work. In parallel, we are finalizing plans to test the safety of the Neurgain PSD system to deliver OPC1 in SCI patients. We believe this device can improve the ease and precision of delivering our cells to the spinal parenchyma. As an added benefit, based on feedback from the FDA, in addition to patients with subacute SCI, we anticipate that patients with chronic SCI also will be eligible for enrollment in this study. Gaining additional OPC1 safety and device performance data across a broader range of patients and injury types will be more informative to the program and support further product and device development. Our recent accomplishments in areas of production and delivery contributed real-world feasibility to the promising clinical results previously reported with this program, in which OPC1 demonstrated improvements to quality of life and motor function for certain SCI patients. Importantly, we are working to be in a position to initiate a late-stage clinical study in SCI next year.”
The Neurgain PSD system has been designed to allow for the administration of cells to the spinal cord without stopping the patient’s ventilator during the procedure. Elimination of the need to stop respiration during surgery is expected to reduce the complexity, risk, and variability of administering cells to the area of injury. The Neurgain PSD system has been designed to provide delivery of cells with accurate anatomical positioning and dosing, is more compact than existing devices and is attached directly to the patient during the procedure. This innovative delivery system is expected to provide a significant improvement in usability and provide more flexibility to the surgeon when compared to the methods and tools utilized to deliver OPC1 cells in the completed Phase 1/2a SCiStar study of OPC1 for the treatment of cervical SCI. Neurgain Technologies, Inc. is a medical device company that is developing technologies developed by neurosurgeons at the University of California San Diego.
Lineage plans to evaluate the safety and performance of the Neurgain PSD system to deliver OPC1 to the spinal cord in both the preclinical and clinical setting. If results of these studies are positive, Lineage may exercise its option to enter into a pre-negotiated license and commercialization agreement with Neurgain. Pursuant to that agreement, Lineage may integrate the Neurgain PSD system into a late-stage clinical trial and, if approved, commercial use of OPC1 for the treatment of patients with spinal cord injury. There currently are no FDA approved treatments for spinal cord injury.
