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SEC Filings

DEFM14A
ASTERIAS BIOTHERAPEUTICS, INC. filed this Form DEFM14A on 02/04/2019
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Products Under Development

 

AST-OPC1

 

About AST-OPC1

 

Asterias’ AST-OPC1 product candidate is an oligodendrocyte progenitor cell population, derived from a current Good Manufacturing Practice (“cGMP”) master cell bank of undifferentiated hES cells that has been qualified for human use. These cells, which are stored frozen until ready for use, are produced under cGMP conditions and screened for adventitious agents.

 

Oligodendrocytes are nature’s neural insulating cells. Like the insulation covering an electrical wire, oligodendrocytes enable the conduction of electrical impulses along nerve fibers throughout the central and peripheral nervous system. They are also known to promote neural growth, as well as induce blood vessel formation around nerve axons.

 

AST-OPC1 cells have been shown to reproduce the natural functions of oligodendrocytes in animals, including three potentially reparative functions that address the complex pathologies observed at the site of a spinal cord injury. These activities of AST-OPC1 include production of neurotrophic factors, stimulation of vascularization, and induction of remyelination of denuded axons, all of which are critical for survival, regrowth, and conduction of nerve impulses through axons at the injury site. In preclinical animal testing, AST-OPC1 administration led to remyelination of axons, improved hind limb and forelimb locomotor function, dramatic reductions in injury-related cavitation, and significant preservation of myelinated axons traversing the injury site.

 

Phase 1/2a Study in Patients with Complete Cervical Spinal Cord Injuries

 

Based on the results of a previously completed Phase 1 safety trial of AST-OPC1 in thoracic spinal cord injury, Asterias obtained permission from the FDA in August 2014 to initiate a Phase 1/2a dose escalation trial in subjects with neurologically complete (AIS-A) cervical spinal cord injuries (the “SCiStar study”). In May 2016, Asterias was granted FDA clearance to expand the SCiStar study to enroll additional subjects to include two additional cohorts of subjects with motor complete, sensory incomplete (AIS-B) cervical spinal cord injury. The SCiStar study is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in twenty-five subjects with subacute, C-4 to C-7, motor complete (AIS-A or AIS-B) cervical 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 patients have lost all motor and sensory function below their injury site, while AIS-B patients have lost all motor function but may retain some minimal sensory function below their injury site. AST-OPC1 was 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.

 

Asterias completed enrollment of the entire SCiStar study in December 2017. The SCiStar study consists of five cohorts:

 

Cohort   Injury Type; AST-OPC1 Dose   # of Subjects
Cohort 1   AIS-A; 2M AST-OPC1 cells
(low dose for safety evaluation)
  3
Cohort 2   AIS-A; 10M AST-OPC1 cells   6
Cohort 3   AIS-A; 20M AST-OPC1 cells*   6
Cohort 4   AIS-B; 10M AST-OPC1 cells   6
Cohort 5   AIS-B; 20M AST-OPC1 cells*   4
Total       25

 

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