Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs, today provided an update on OPC1, the Company’s oligodendrocyte progenitor cell (OPC) therapy for the treatment of acute spinal cord injury (SCI). The OPC1 program was obtained through Lineage’s acquisition of Asterias Biopharmaceuticals in March 2019, and manufacturing has been completely transferred to the Company’s current Good Manufacturing Practice (cGMP) facility in Israel, where key process improvements have been developed and implemented. Lineage also has strengthened its patent position in order to protect the OPC1 processes, product and composition, and methods of use. Lineage intends to meet with the U.S. Food and Drug Administration (FDA) to discuss further development of the OPC1 program by the end of 2020.
“We have worked diligently over the past year to transition all manufacturing activities for the OPC1 program to our in-house cGMP facility, where our experienced cell therapy production team could develop and deploy much-needed improvements and modernization to the production and analytical processes,” stated Brian M. Culley, Lineage CEO. “This work has achieved significantly better efficiency and improved quality control, which we expect will enable a consistent supply of material to support a late-stage clinical trial of OPC1. With these necessary steps now completed, our focus turns to developing a “thaw-and-inject” formulation and superior delivery tools, to enable an easier surgical procedure and facilitate faster enrollment in the next clinical trial. We also are evaluating ways to return OPC1 to the clinic sooner than originally planned, reflecting our view of compelling clinical data which continues to read out from the 25-patient phase 1/2a SCiStar study. Our approach with OPC1 replicates our development strategy for OpRegen, our cell therapy program for dry AMD, in which we seek not only to provide cell-based regenerative benefits, but also commercially relevant solutions with competitive advantages in areas of scale-up, production costs, and delivery techniques. By analyzing every piece of the overall therapeutic landscape, we seek to position ourselves as the clear leader in the emerging field of cell therapy transplant medicine.”
Key OPC1 Program Milestones Achieved Since the Asterias Acquisition Include:
- Technology transfer has been completed and cell production processes have been established at Lineage’s cGMP cell therapy facility;
- The production process was improved to achieve greater efficiency and higher quality control, becoming compatible with larger-scale manufacturing, and Biologics Review Application (BLA) readiness;
- Multiple OPC1 batches have been successfully manufactured; GMP production is planned to begin in early 2021;
- New master and working pluripotent cell banks have been produced to supply consistent clinical and commercial batches of OPC1;
- New in-process controls and release testing have been introduced to the production and release process and have been updated to be more compatible with current and expected future FDA BLA review processes;
- Issuance of two new patents in 2019 related to methods for utilizing pluripotent stem cell-derived OPCs for the treatment of SCI and reducing cavitation in patients with acute SCI. Additional patent applications are pending which may further protect the processes, product and composition, and methods of use of OPC1.
Key 2020 Milestone and Development Plans for the OPC1 Program Include:
- Meeting with the FDA to discuss manufacturing improvements and the design of a late-stage comparative clinical trial;
- Introducing a new “thaw-and-inject” formulation to enable easier preparation and administration of cells to the spinal cord, avoiding the need for washing and other dose preparation steps one day prior to treatment and improving commercial positioning;
- Identifying a novel delivery system, to make the surgical procedure easier, faster, and more compatible with a thaw and inject formulation of OPC1, improving overall end-user experience for the surgeon.