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“We believe the primary goals of the SCiStar Study, which were to observe the safety of OPC1 in cervical spinal cord injury patients as well as other important metrics including related to the optimal timing of OPC1 injection, tolerability of the immunosuppression regimen, engraftment of OPC1 cells, and rates of motor recovery observed among different study subpopulations, have all been successfully achieved,” stated Dr. Wirth. “We now are in the process of analyzing the full data set from the SCiStar Study to inform how best to proceed with this promising program. We expect to propose a clinical plan to the
“We appreciate the support of the
The SCiStar Study is an open-label, single-arm trial testing three sequential escalating doses of OPC1 administered 21 to 42 days post-injury, at up to 20 million OPC1 cells in 25 subjects with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) acute spinal cord injuries (SCI). These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A subjects have lost all motor and sensory function below their injury site, while AIS-B subjects have lost all motor function but may have retained some minimal sensory function below their injury site. The primary endpoint in the SCiStar study was safety as assessed by the frequency and severity of adverse events related to OPC1, the injection procedure, and immunosuppression with short-term, low-dose tacrolimus. Secondary outcome measures included neurological functions as measured by upper extremity motor scores and motor level on International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) examinations at 30, 60, 90, 180, 270, and 365 days after injection of OPC1.
Below are a summary of key findings from the SCiStar Study. A copy of Dr. Wirth’s presentation will be available on the Events section of BioTime’s website concurrent with his presentation at ASNTR.
- Overall safety profile of OPC1 to date is excellent
- Magnetic resonance imaging (MRI) scans at 12 months post-injection of OPC1 has shown no evidence of adverse changes in any of the 25 SCiStar study subjects treated with OPC1.
- To date, there have been no unexpected serious adverse events (SAEs) related to the OPC1 cells.
- No concerning safety issues and no intraoperative complications have been noted.
- No SCiStar study subjects had worsening of neurological function post-injection.
- No adverse findings observed on follow-up MRI scans.
- Immunosuppression with tacrolimus (an immunosuppressive drug utilized mainly after allogeneic organ transplant to lower the risk of organ rejection) was well-tolerated.
- Majority of SCiStar subjects who received 10M or 20M OPC1 cells exhibited robust motor recovery in upper extremities
- Three subjects (Cohort 1) received a sub-therapeutic dose of 2M cells to evaluate the initial safety of injecting OPC1 into lesions in the cervical spinal cord. All other subjects (Cohorts 2-5) received 10M or 20M cells.
- At 12 months, 95% (21/22) of SCiStar study subjects in Cohorts 2-5 recovered at least one motor level on at least one side and 32% (7/22) of these subjects recovered two or more motor levels on at least one side. The average improvement in upper extremity motor score as measured by the ISNCSCI scale for these subjects was 8.9 points.
- Notably, no SCiStar study subjects saw decreased motor function following administration of OPC1 and subjects either retained for 12 months the motor function recovery seen through 6 months or experienced further motor function recovery from 6 to 12 months.
- MRI scans consistent with durable engraftment through 1 year post-injection
- All three SCiStar study subjects in Cohort 1 and 95% (21/22) of SCiStar study subjects in Cohorts 2 to5 have MRI scans at 12 months consistent with the formation of a tissue matrix at the injury site, which is encouraging evidence that OPC1 cells have engrafted at the injury site and helped to prevent cavitation, a destructive process that occurs within the spinal cord following spinal cord injuries, and typically results in permanent loss of motor and sensory function.
OPC1 is an oligodendrocyte progenitor cell (OPC) therapy currently being tested in a Phase I/IIa clinical trial known as SCiStar for the treatment of acute spinal cord injuries. 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 control and other body functions. The clinical development of the OPC1 program has been partially funded by a
BioTime Inc. IR
Ioana C. Hone
Solebury Trout IR
Gitanjali Jain Ogawa