OPC1 is an oligodendrocyte progenitor cell therapy that is currently in a Phase 1/2a multicenter clinical trial for acute spinal cord injuries (“SCI”), that has been partially funded by the California Institute for Regenerative Medicine. (“CIRM”).
Oligodendrocyte progenitor cells (OPCs) are naturally-occurring precursors to the cells which provide electrical insulation for nerve axons in the form of a myelin sheath. SCI occurs when the spinal cord is subjected to a severe crush or contusion injury and typically results in severe functional impairment, including limb paralysis, aberrant pain signaling, and loss of bladder and sexual function. Based on the multiple reparative properties associated with OPC1, we believe this candidate cell therapy product is ideally suited to treat neurological conditions such as SCI and other demyelination and demyelination disorders of the central nervous system.
OPC1 has received a regenerative medicine advanced therapy (RMAT) and Orphan Drug designations from the FDA, which include accelerated programs intended to facilitate the development and review process, including early interactions with FDA during clinical development and enhanced favorability for marketing approval.
Highlighting the progress made by Kris Boesen and Lucas Lindner, both of whom received Lineage’s OPC1 cell therapy following traumatic spinal cord injury (SCI).
There are approximately 18,000 new spinal cord injuries annually in the U.S. (NSCIC SCI Facts and Figures at a Glance (2019), and there are currently no drugs approved by the FDA specifically for the treatment of SCI.
Learn More About Spinal Cord Injury and Lineage’s OPC1 Cell Therapy
OPC1 has been tested in two clinical trials to date; a five patient Phase 1 safety trial and a 25 patient Phase 1/2a dose escalation trial (the “SCiStar trial”). The clinical development of OPC1 has been partially funded by a $14.3 million grant from the California Institute for Regenerative Medicine.
In January 2019, top line data was announced from the SciStar trial:
- Improved Motor Function
- At least one-level: 96%
- Two or more levels 32%
- Cell Engraftment – 96%
- Positive Safety Profile – To date, no serious adverse events (SAEs) related to the OPC1 cells (includes long-term follow up of Phase 1 safety study subjects)
For a more details about the Phase 1/2a study, please see the press release here (originally issued by Asterias).
In November 2019, we reported positive results from the ongoing SciStar study of OPC1, where the overall safety profile of OPC1 has remained excellent with robust motor recovery in upper extremities maintained through Year 2 patient follow-ups available to date. In July 2020, we reported that following the transfer of OPC1 manufacturing to our cGMP manufacturing facility in Israel, key process improvements have been developed and implemented. Lineage also strengthened its patent position in order to protect the OPC1 processes, product and composition, and methods of use. In December 2020, Lineage reported it has developed an enhanced differentiation process, leading to major improvements in production and quality of its OPC1 cell therapy drug product.
In February 2021, we announced that we had entered into an exclusive option and license agreement with Neurgain Technologies, Inc., a medical device company that is commercializing technology developed by neurosurgeons at the University of California San Diego. Under the terms of the agreement, the Company and Neurgain will collaborate on the clinical testing of Neurgain Technologies Inc.’s Parenchymal Spinal Delivery System (“Neurgain PSD system”), which is designed to allow for the administration of cells to the spinal cord without stopping the patient’s respiration. 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. Lineage will evaluate the Neurgain PDI system’s ability to safely and effectively deliver OPC1, Lineage’s allogeneic oligodendrocyte progenitor cell (OPC) transplant, to the spinal cord in both preclinical and clinical studies beginning this year.
In June 2021, Lineage provided an update on the clinical advancement of OPC1. 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’s PSD system to deliver OPC1 cells to the spinal cord. 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.
View our pipeline to see our other programs in development.