Research Shows Improved Arm, Hand and Finger Function in People with Spinal Cord Injury

Asterias Biotherapeutics Inc., a California-based biotechnology company pioneering the field of regenerative medicine, announced on June 13 that new nine-month follow-up data show improved arm, hand and finger function in study participants with complete (AIS-A) cervical spinal cord injury (SCI) who received the company's treatment called AST-OPC1 (oligodendrocyte progenitor cells derived from federally approved stem cell lines). The study's eligibility criteria limit participation to people who are newly injured because AST-OPC1 must be administered between 14 and 30 days following injury.

In addition, the study – called SCiStar – shows that three of the six patients who received a 10-million-cell dose of AST-OPC1 in the Phase 1/2a clinical trial have now recovered two levels of motor function on at least one side. And previously announced improvements in arm, hand and finger function at three months and six months following administration of AST-OPC1 have been confirmed and further increased at nine months. The study is being conducted at six U.S. facilities, including Shepherd Center, and the company plans to increase this to up to 12 sites to accommodate expanded patient enrollment.

“The new efficacy results show that previously reported meaningful improvements in arm, hand and finger function in the 10-million-cell cohort treated with AST-OPC1 cells have been maintained and in some patients have been further enhanced even nine months following dosing,” said Edward Wirth III, M.D., chief medical officer at Asterias. “Gains in motor function, such as the improvements observed in the SCiStar study to date, have been shown to increase a patient’s ability to function independently following complete cervical spinal cord injuries. We are increasingly encouraged by these continued positive results, which are remarkable compared with spontaneous recovery rates observed in a closely matched untreated patient population.”

Jane S. Lebkowski, Ph.D., Asterias’ president of research and development and its chief scientific officer, presented the nine-month efficacy and safety data on the AIS-A 10-million-cell cohort on June 13 during the International Society for Stem Cell Research (ISSCR) 2017 Annual Meeting held in Boston, Mass. Her slide presentation is available here. 

Nine-Month Follow-up Results

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 research participants and physicians consistently report that improvements in upper-extremity motor function are the most desirable functional improvement target in people with quadriplegia, as even relatively modest changes can 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 spinal cord above which a research participant has normal function. As suggested by existing research, patients with severe spinal cord injury who 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. This ability significantly reduces the overall level of daily assistance needed for the patient and associated healthcare costs.

Six patients were enrolled and dosed in the AIS-A 10-million-cell cohort, with five of six patients completing a nine-month follow-up visit. The results include the following highlights:

Motor Level Improvement – Additional motor level improvement was seen in the AIS-A 10-million-cell cohort at nine months.

• Three of six patients (50 percent) achieved two motor levels of improvement over baseline on at least one side as of their latest follow-up visit through nine months. This compares to two of six patients (33 percent) who had improved two motor levels on at least one side through three and six months of follow-up.
• In addition, all six patients (100 percent) achieved at least one motor level of improvement on at least one side as of their latest follow-up through nine months.

Upper-Extremity Motor Score – Additional improvement in the average UEMS score for this cohort was observed at nine months. The average UEMS improvement at nine months was 11.2 points, compared to 9.7 points at six months.

Matched Historical Control Data – The nine-month results show a meaningful improvement in the motor function recovery in the AIS-A patients receiving 10 million AST-OPC1 cells compared to a historical control group of 62 closely matched patients from the EMSCI database. In the historical matched control, 29 percent of patients recovered two motor levels on at least one side 12 months after baseline compared to the 50 percent of the AIS-A patients receiving AST-OPC1 who have recovered two motor levels on at least one side nine months after baseline.

Safety – The trial results to date continue to indicate 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.

As previously reported, Asterias will report 12-month efficacy and safety data from the AIS-A 10-million-cell cohort sometime late in the third quarter of 2017 after the 12-month results are collected for the entire cohort.

Each year in the United States, more than 17,000 people sustain 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.

About the SCiStar Trial

The SCiStar trial 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 as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical 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 is being administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.

The study is being conducted at six U.S. centers, and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study are the Medical College of Wisconsin in Milwaukee, Shepherd Center in Atlanta, University of Southern California (USC) jointly with Rancho Los Amigos National Rehabilitation Center in Los Angeles, Indiana University, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose, jointly with Stanford University.

All clinical trial participants at Shepherd Center must be patients who are admitted to Shepherd Center for rehabilitation. Also, clinical trial eligibility requirements apply. Medical professionals are invited to promptly refer patients for assessment to determine whether they are appropriate for admission to Shepherd Center. Contact Shepherd Center Admissions at 800-SHEPHERD (800-743-7437) or admissions@shepherd.org.

Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provides $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for AST-OPC1.

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 at www.SCiStar-study.com.

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, has been shown in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed at the injury 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 hindlimb and forelimb locomotor function, dramatic reductions in injury-related cavitation and significant preservation of myelinated axons traversing the injury site.

In a previous Phase 1 clinical trial, five patients with neurologically complete, thoracic spinal cord injury were administered two million AST-OPC1 cells at the spinal cord injury site between seven and 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 AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spinal cord injuries, which represents the first targeted population for registration trials.

About Asterias Biotherapeutics

Asterias Biotherapeutics Inc. is a biotechnology company pioneering the field of regenerative medicine. The company's proprietary cell therapy programs are based on its pluripotent stem cell and immunotherapy platform technologies. Asterias is presently focused on advancing three clinical-stage programs that have the potential to address areas of very high unmet medical need in the fields of neurology and oncology. AST-OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. AST-VAC1 (antigen-presenting autologous dendritic cells) is undergoing continuing development by Asterias based on promising efficacy and safety data from a Phase 2 study in acute myeloid leukemia (AML), with current efforts focused on streamlining and modernizing the manufacturing process. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic cancer immunotherapy. The company's research partner, Cancer Research UK, plans to begin a Phase 1/2a clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at www.asteriasbiotherapeutics.com.

FORWARD-LOOKING STATEMENTS

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 Securities and Exchange Commission. Asterias disclaims any intent or obligation to update these forward-looking statements.

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