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ASTERIAS BIOTHERAPEUTICS, INC. filed this Form DEFM14A on 02/04/2019
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In late July 2018, Asterias provided a clinical trial update on the AST-OPC1 SCiStar study that highlighted, among other things, the following:


Positive Safety Profile – Asterias has dosed 25 subjects with AST-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. The results-to-date continue to support the safety of AST-OPC1.


Cell Engraftment - 92% (11/12) of Cohort 3 and 4 subjects have magnetic resonance imaging (“MRI”) scans at twelve months consistent with the formation of a tissue matrix at the injury site, which is encouraging evidence that AST-OPC1 cells have engrafted at the injury site and helped to prevent cavitation. The 12-month MRI results-to-date for 94% (17/18) of the Cohort 2-4 subjects provide supportive evidence that AST-OPC1 cells have durably engrafted at the injury site and helped to prevent cavitation. In addition, 100% (4/4) of Cohort 5 subjects had MRI scans at six months consistent with the formation of a tissue matrix at the injury site. Cavitation is 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 twelve months, 94% (17/18) of Cohort 2-4 subjects recovered at least one motor level on at least one side and 33% (6/18) of these subjects recovered two or more motor levels on at least one side. Asterias expects to report the 12-month top-line readout for the entire study early in the first quarter of 2019.


In September 2018, Asterias announced that the FDA has accepted Asterias’ request to meet to discuss proposed next steps for the OPC1 clinical development program. The meeting with the FDA under OPC1’s RMAT designation took place in November 2018. In October 2018, Asterias announced a positive outcome from an independent Data Review Panel’s review of the data generated by patients enrolled in the SCiStar study.


Asterias expects to have the 12-month top-line readout for the entire study late in the first quarter of 2019.


The SCiStar study followed a previous Phase 1 clinical trial, in which five subjects with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site 7-14 days post-injury. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with the administration of the cells, with AST-OPC1 itself, or the immunosuppressive regimen. All five subjects have completed at least six years of follow-up. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all subjects. In four of the five subjects, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. There have been five minor adverse events possibly related to AST-OPC1 such as transient fever and nerve pain. There have been no unexpected neurological changes to date.


Regenerative Medicine Advanced Therapy Designation and Orphan Drug Designation


In September 2017, the FDA granted Asterias’ request for AST-OPC1 to be designated a Regenerative Medicine Advanced Therapy (“RMAT”) under the 21st Century Cures Act. The RMAT designation is intended to facilitate expedited development, review and approval for important new regenerative medicine therapies for which preliminary clinical evidence indicates the potential to address a serious or life-threatening disease or condition. In addition to providing an avenue for increased and earlier interactions with the FDA, RMAT-designated products may be eligible for priority review and accelerated approval. Asterias expects to have discussions with the FDA sometime in 2018 on the development of AST-OPC1 using the RMAT designation.


In February 2016, Asterias announced that the FDA had granted Asterias’ application for Orphan Drug Designation of AST-OPC1 for the treatment of acute spinal cord injury.


Manufacturing and Process Development


Asterias is in the process of establishing additional cGMP master and working cell banks of undifferentiated hES cells of the H1 cell line for future clinical development and commercial use. Asterias is also updating the manufacturing process for AST-OPC1 to improve yields and support a scalable process for eventually manufacturing on a larger scale. Asterias will need to successfully complete the update of the manufacturing process in order to manufacture additional clinical-grade lots of AST-OPC1 to use for future clinical development.



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