Asterias Biotherapeutics, Inc. (AST)

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Asterias Announces Additional Motor Function Improvement at 6-months and 9-months Following Treatment with AST-OPC1 in Patients with Complete Cervical Spinal Cord Injuries



-Management to discuss results on conference call today at 5:00 p.m. Eastern / 2:00 p.m. Pacific-

-SCiStar lead investigator Richard Fessler, MD, and John Steeves, Ph.D., Co-Chair of the Spinal Cord Outcomes Partnership Endeavor (SCOPE), will join Asterias management on the call-

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Asterias Biotherapeutics, Inc.

Jan 24, 2017, 07:00 ET

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FREMONT, Calif., Jan. 24, 2017 /PRNewswire/ -- Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company pioneering the field of regenerative medicine, today announced positive efficacy results from the company's ongoing SCiStar Phase 1/2a clinical trial that showed additional motor function improvement at 6-months and 9-months following administration of 10 million AST-OPC1 cells in AIS-A patients with complete cervical spinal cord injuries (SCI).

"Recovery of upper extremity motor function is critically important to patients with complete cervical spinal cord injuries, since this can dramatically improve quality of life and their ability to live independently," said Richard Fessler, M.D., Professor, Department of Neurosurgery at Rush University Medical Center and lead investigator in the SCiStar study. "Patients in this cohort are seeing what we believe are meaningful improvements in their ability to use their arms, hands and fingers at 6-months and 9-months following AST-OPC1 administration."

"The results to date in the 10 million cell cohort treated with AST-OPC1 cells show that the improvements in arm, hand and finger function observed very early in the study have been maintained and in most patients have even been further enhanced over time," said Steve Cartt, Chief Executive Officer of Asterias. "These results to date are quite encouraging, and we look forward to initiating discussions with the FDA in mid-2017 to begin to determine the most appropriate clinical and regulatory path forward for this innovative therapy. In addition, we anticipate reporting 12-month efficacy and safety data for this cohort, as well as 6-month efficacy and safety data for the currently-enrolling AIS-A 20 million cell and AIS-B 10 million cell cohorts, during the third quarter of 2017."

A total of six patients were enrolled and dosed in the AIS-A 10 million-cell cohort, with five of six patients having now completed their 6-month follow-up and three of six patients having completed their 9-month follow-up.

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. The latest results include the following highlights:

Improvements in Motor Function

Upper Extremity Motor Score - For the five patients who have completed at least 6 months of follow-up, five of five patients saw their early improvements in motor score (UEMS) at 3 months maintained or further increased through their most recent data point (6 months or 9 months, depending on the most recent data available for each patient).
Motor Level Improvement - For patients completing at least 6 months of follow up, as of the date of each patient's last follow-up visit, 100% (five of five) had achieved at least a one motor level improvement (using the ISNCSCI scale) over baseline on at least one side, and 40% (two of five) had achieved two motor levels over baseline on at least one side, with one of these patients achieving a two motor level improvement on both sides.
Matched Historical Control Data - Asterias and key experts in the spinal cord injury field have developed a set of matched historical control data for both Upper Extremity Motor Score and Motor Level Improvement to clearly document expected spontaneous recovery in untreated patients for comparison to results seen in patients treated with AST-OPC1. The key results from this analysis, which show a meaningful difference in the motor function recovery seen to date in patients treated with the 10 million cell dose of AST-OPC1, will be presented during today's conference call.

Safety

The trial results to date continue to reveal a positive safety profile for AST-OPC1. There have been no serious adverse events related to AST-OPC1 and data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.

In September 2016, Asterias reported positive early efficacy data for AST-OPC1 from the AIS-A patients that had been dosed with 10 million AST-OPC1 cells in the SCiStar study. Today's data show that the improvements in motor function seen at 3 months post-implantation of AST-OPC1 for these patients appear to be maintained, and in some cases improved even further, on continued follow up. Furthermore, these results suggest that the observed gains exceed the expected rates of spontaneous recovery in a closely matched population of historical controls.

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 arm, hand and finger functional capabilities in these patients can result in lower healthcare costs, significant improvements in quality of life, increased ability to engage in activities of daily living, and increased independence.

Conference Call and Webcast Today at 5:00 p.m. ET (2:00 p.m. PT) to Discuss the Results

Asterias will host a conference call and webcast today, January 24, 2017 at 5:00 p.m. Eastern / 2:00 p.m. Pacific to discuss the results. Company management will be joined by Richard G. Fessler, MD, PhD, Professor, Department of Neurosurgery at Rush University Medical Center and SCiStar lead investigator, and John Steeves, Ph.D., Co-Chair of the Spinal Cord Outcomes Partnership Endeavor (SCOPE) and Professor of International Collaboration on Repair Discoveries (ICORD).