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“While the primary endpoint for the SCiStar trial was 12 months, we are further encouraged by this additional follow-up data that shows both durable engraftment and motor function recovery being maintained or improved upon at 24 months,” commented
Below are a summary of key findings at 24 months for the Cohort 2 subjects:
- Positive Safety Profile – MRI scans at 24 months post-injection of OPC1 has shown no evidence of adverse changes in any of the Cohort 2 subjects treated with OPC1. Additionally, Asterias has dosed 25 subjects with OPC1 in the SCiStar study and a total of 30 subjects including the five subjects from a previous Phase 1 safety trial in thoracic spinal cord injury who have been followed for as long as seven years. To date, there have been no serious adverse events (SAEs) related to the OPC1 cells.
- Cell Engraftment – 100% (6/6) of Cohort 2 subjects have magnetic resonance imaging (MRI) scans at 24 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. The MRI results provide supportive evidence that OPC1 cells have durably engrafted at the injury site and helped to prevent cavitation.
Cavitationis a destructive process that occurs within the spinal cord following spinal cord injuries, and typically results in permanent loss of motor and sensory function. Additionally, a patient with cavitation can develop a condition known as syringomyelia, which results in additional neurological and functional damage to the patient and can result in chronic pain.
- Improved Motor Function – At 24 months, 100% (6/6) of Cohort 2 subjects have recovered at least one motor level on at least one side. At 24 months, 83% (5/6) of these subjects have recovered two or more motor levels on at least one side, compared to 67% (4/6) at 12 months after administration of the OPC1 cells. At 24 months, the average improvement in upper extremity motor function for Cohort 2 subjects was 13.0 points, compared to 12.3 points at 12 months after administration of the OPC1 cells. At 24 months, one subject had recovered two motor levels bilaterally and another subject had recovered three motor levels on one side.*
*One subject had recently underwent an elective surgery that restrained his ability to fully use one of the muscle groups evaluated during his 24 month follow-up and thus this muscle group was rated as not testable. The subject had otherwise retained the motor function recovery seen through 12 months for all muscle groups that could be evaluated during the 24 month follow-up. For purposes of determining the Cohort’s average improvement in upper extremity motor function at 24 months this subject’s 12 month score was used for the muscle group that was not testable.
OPC1 Therapeutic Platform
OPC1, an oligodendrocyte progenitor cell population derived from human embryonic stem cells, has been shown in preclinical testing in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed in demyelination disorders, such as spinal cord injury and multiple neurodegenerative diseases, including multiple sclerosis and white matter stroke. These potential reparative functions of OPC1 include the production of neurotrophic factors, the stimulation of vascularization, and the induction of remyelination of denuded axons, all of which are critical for survival and regrowth of—and conduction of nerve impulses through—axons at the injury site.
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Anticipated Data Readouts for the SCiStar Study
Asterias has completed enrollment and dosing in all five of its planned SCiStar study cohorts. The Company intends to report 12 month results for the entire SCiStar study in the first quarter of 2019.
OPC1, an oligodendrocyte progenitor cell population derived from human embryonic stem cells, has been shown in preclinical testing in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed in demyelination disorders, such as spinal cord injuries, and multiple neurodegenerative diseases, including multiple sclerosis and white matter stroke. These potential reparative functions of OPC1 include the production of neurotrophic factors, the stimulation of vascularization, and the induction of remyelination of denuded axons, all of which are critical for survival and regrowth of—and conduction of nerve impulses through—axons at the injury site.
About the SCiStar Trial
The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of OPC1 administered 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) 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. OPC1 is administered 21 to 42 days post-injury. Subjects will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.
The SCiStar study consists of five cohorts:
|Cohort||Injury Type; OPC1 Dose||# of Subjects|
|Cohort 1||AIS-A; 2M OPC1 cells
(low dose for initial safety evaluation)
|Cohort 2||AIS-A; 10M OPC1 cells||6|
|Cohort 3||AIS-A; 20M OPC1 cells*||6|
|Cohort 4||AIS-B; 10M OPC1 cells||6|
|Cohort 5||AIS-B; 20M OPC1 cells*||4|
|*One subject from Cohort 3 and one subject from Cohort 5 were administered 10 million cells.|
Asterias has received a Strategic Partnerships Award grant from the
Statements pertaining to future financial and/or operating and/or clinical research results, future growth in research, technology, clinical development, and potential opportunities for Asterias, along with other statements about the future expectations, beliefs, goals, plans, or prospects expressed by management constitute forward-looking statements. Any statements that are not historical fact (including, but not limited to statements that contain words such as "will," "believes," "plans," "anticipates," "expects," "estimates") should also be considered to be forward-looking statements. Forward-looking statements involve risks and uncertainties, including, without limitation, risks inherent in the development and/or commercialization of potential products, uncertainty in the results of clinical trials or regulatory approvals, need and ability to obtain future capital, and maintenance of intellectual property rights. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the businesses of Asterias, particularly those mentioned in the cautionary statements found in Asterias' filings with the