Asterias Biotherapeutics (AST) Announces Positive Interim Data for AST-OPC1

September 14, 2016 7:05 AM EDT

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Asterias Biotherapeutics, Inc. (NYSE: AST) presented positive interim efficacy data from the 10 million cell cohort in the Company's ongoing AST-OPC1 SCiSTAR Phase 1/2a multicenter clinical study in complete cervical spinal cord injury patients. While early in the study, with only 4 of the 5 patients in the cohort having reached 90 days after dosing, all patients have shown at least one motor level of improvement so far and the efficacy target of 2 of 5 patients in the cohort achieving two motor levels of improvement on at least one side of their body has already been achieved. Patient improvements are being measured by the ISNCSCI neurological classification scale widely used to quantify functional status of patients with spinal cord injuries. As suggested by existing research, patients with complete cervical 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.

The efficacy target was recommended by the independent Spinal Cord Outcomes Partnership Endeavor (SCOPE) based on recently published data (Steeves et al., Topics in Spinal Cord Injury Rehabilitation, 2012). SCOPE is affiliated with the American Spinal Injury Association.

"I am very encouraged by this first look at efficacy data for AST-OPC1 in complete cervical spinal cord injury patients," commented Shekar N. Kurpad, MD, PhD, Professor of Neurosurgery, Director, Spinal Cord Injury Center Medical College of Wisconsin. "As a clinical investigator in the trial, I am looking forward to continuing the evaluation of this promising new treatment in these patients, for whom effective new therapeutic options are desperately needed. I am hopeful that we are able to see these early, positive efficacy trends continue as the study progresses."

"The results to date in the 10 million cell cohort, while still early, demonstrate meaningful improvement in motor function, particularly in the use of a patient's hands, fingers and arms, which is critically important for a patient's quality of life and ability to function independently," said Steve Cartt, Chief Executive Officer of Asterias. "We are quite encouraged by this first look at efficacy results and look forward to reporting six-month efficacy data as planned in January 2017. We have also just recently been cleared to begin enrolling a new cohort and administering to these new patients a much higher dose of 20 million cells. We look forward to begin evaluating efficacy results in this higher-dose cohort in the coming months as well."

The SCiSTAR study is funded in part by a $14.3 million grant from the California Institute for Regenerative Medicine (CIRM).

The data were presented by Edward Wirth, MD, PhD, Chief Medical Officer of Asterias, at the 55th Annual Scientific Meeting of the International Spinal Cord Society (ISCoS) in Vienna, Austria and included the following highlights:

Efficacy and Safety Results: Cohort 2 dosed with 10 million cells

  • 5 out of 5 patients dosed with 10 million cells have exhibited improved upper extremity motor scores (UEMS) relative to baseline.
  • At Day 90 of follow up, 4 of 4 patients dosed have improved one motor level on at least one side, 2 of 4 patients have improved two motor levels on at least one side, and 1 patient has improved two motor levels on both sides.
  • The average UEMS improvement at Day 90 for the 4 patients that have reached this follow up was 9.5 points.
  • The results to date from Cohort 2 show no serious adverse events related to AST-OPC1, the injection procedure, or immunosuppression with low-dose tacrolimus. In addition, data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.

Efficacy and Safety Results: Cohort 1 dosed with 2 million cells

  • 3 out of 3 patients have exhibited improved upper extremity motor scores (UEMS) relative to baseline.
  • The average UEMS improvement for the 3 patients was 5.0 points at Day 90, and they continued to improve an average of 7.0 points at 1 year.
  • Despite the very low dose used in this safety cohort, at 1 year of follow up 1 patient in Cohort 1 has improved one motor level on one side and 2 patients have improved one motor level on both sides.
  • The 12-month results from Cohort 1 show no serious adverse events related to AST-OPC1, the injection procedure, or immunosuppression with low-dose tacrolimus. In addition, data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.

The results from Cohort 2 dosed with 10 million cells compared to those from Cohort 1 dosed with 2 million cells may also begin to support a dose response as patients dosed in the earlier safety cohort with 2 million cells saw an average upper extremity motor score (arms, hands, fingers) increase of 5.0 at Day 90, while the 10 million cell cohort has experienced an average increase of 9.5 at Day 90. The company expects to have data evaluating the efficacy results after implantation of 20 million AST-OPC1 cells in complete cervical spinal cord injury patients later in 2017.

For additional information on the presentation data presented, please see the ISCoS conference presentation on the company's website at: http://asteriasbiotherapeutics.com/wp-content/uploads/2016/09/Wirth-ISCoS-14SEP2016-talk-FINAL.pdf.

Data evaluating the efficacy results six months after implantation of 10 million AST-OPC1 cells in complete cervical spinal cord injury patients will be available in January 2017 and will focus on improvement in physical functioning of the upper extremities (fingers, hands and arms) of each treated patient utilizing scoring on the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI scale).

Each year in the U.S. more than 17,000 people suffer a severe, debilitating spinal cord injury. These injuries can be devastating to quality of life and ability to function independently. Lifetime healthcare costs for these patients can often approach $5 million. Improvements in functional capabilities of spinal cord injury patients can result in significant improvements in daily living, less required care by an attendant and lower cost of care for patients.



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