OPC1 is an oligodendrocyte progenitor cell therapy that is currently in a Phase I/IIa 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.
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.
OPC1 has been tested in two clinical trials to date; a five patient Phase 1 safety trial and a 25 patient Phase I/IIa 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: 95% (21/22 patients)
- Two or more levels 32% (7/22 patients)
- Cell Engraftment – 95% (21/22)
- 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 I/IIa study, please see the press release here (originally issued by Asterias).
We are now in the process of analyzing the data from the SciStar trial to inform us as to how to proceed with further discussions with the FDA. Additionally, we are leveraging our manufacturing capabilities and process development expertise learned from OpRegen to the support of OPC1 development.