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“We believe the primary goal of SCiStar, which was to observe the safety of OPC1 in cervical spinal cord injury patients and to accumulate data related to important factors for the design of later-stage trials, such as optimal dosing levels, timing of OPC1 injection after SCI, the immunosuppression regimen, engraftment of the cells, and rates of motor recovery observed among different study subpopulations, have been successfully achieved,” commented
Below are a summary of key findings at 12 months for the SCiStar study subjects:
- Positive Safety Profile –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. Asterias has dosed 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 eight years. To date, there have been no unexpected serious adverse events (SAEs) related to the OPC1 cells.
- Cell Engraftment – All three SCiStar subjects in Cohort 1 and 95% (21/22) of SCiStar subjects in Cohorts 2-5 have magnetic resonance imaging (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. 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 12 months, 95% (21/22) of SCiStar study subjects who were administered either 10 million or 20 million cells of OPC1 (Cohorts 2-5) recovered at least one motor level on at least one side. At 12 months, 32% (7/22) of these subjects recovered two or more motor levels on at least one side. At 12 months, the average improvement in upper extremity motor score as measured by the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) scale for Cohort 2-5 subjects was 8.9 points. No subjects saw decreased motor function following administration of OPC1 and subjects consistently retained the motor function recovery seen through 6 months or saw further motor function recovery from 6 to 12 months.
- Results Excluding Certain SCiStar Study Subjects– Excluding those SCiStar subjects in Cohorts 2-5 that would either (i) not meet the eligibility criteria the Company proposed to
FDAduring its recent Type B meeting to discuss the next study for OPC1 or (ii) receive a modified post-surgical procedure to reduce potential cord compression issues, at 12 months 100% (17/17) of these SCiStar study subjects recovered at least one motor level on at least one side, 41% (7/17) of these subjects recovered two or more motor levels on at least one side, and the average improvement in upper extremity motor score as measured by the ISNCSCI scale for these subjects was 10.2 points.
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.
Each year in
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.
Improvements in upper extremity motor function are being measured using the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) scale, widely used to quantify functional status of patients with spinal cord injuries. Both subjects and physicians consistently report that improvements in upper extremity motor function are the most desirable functional improvement target in the quadriplegic population, since even relatively modest changes can potentially have a significant impact on functional independence, quality of life and cost of care. The SCiStar study is monitoring two separate ISNCSCI measurements of upper extremity motor function. The upper extremity motor score (UEMS), is a linear scale used to quantify motor function at each of five upper extremity muscle groups driving arm and hand function; these scores are also used to determine "motor levels", which define the level within the cord above which a subject has normal function. As suggested by existing research, patients with severe spinal cord injuries that show two motor levels of improvement on at least one side may regain the ability to perform daily activities such as feeding, dressing and bathing, which significantly reduces the overall level of daily assistance needed for the patient and associated healthcare costs.
Asterias has received a Strategic Partnerships Award grant from the
Additional information on the Phase 1/2a trial, including trial sites, can be found at www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar Study Website (www.SCiStar-study.com).
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
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