Meet Some of the Physicians
and Cervical Spinal Cord
Injury Patients in the
AST-OPC1 Clinical Trial

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July 17, 2017
Asterias Biotherapeutics Completes Enrollment and Dosing of SCiStar Study's AIS-A 20 Million Cell Cohort
Twenty patients have been enrolled in SCiStar study
20 million cell dose escalation cohort to inform future trial design
Dosing fifth patient in this cohort triggers $1.5 million grant payment

FREMONT, Calif., July 17, 2017 /PRNewswire/ -- Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company pioneering the field of regenerative medicine, today announced completion of enrollment and dosing of the AIS-A 20 million cell cohort in the company's ongoing SCiStar Phase 1/2a clinical study of AST-OPC1 in complete cervical spinal cord injury (SCI). In this cohort, five patients with AIS-A grade SCIs were administered 20 million AST-OPC1 cells. The 20 million AST-OPC1 cell dose is the highest to be investigated in the SCiStar Phase 1/2a clinical trial. The company expects to report top-line six-month results from this cohort in January 2018.

"Completing enrollment and dosing of the first cohort of patients receiving 20 million AST-OPC1 cells marks another important milestone for our SCiStar study," said Dr. Edward Wirth, Chief Medical Officer of Asterias. "The results from this cohort will help us evaluate what is the optimal dose for a future, larger clinical study. We look forward to evaluating the data from our enrolled cohorts as we begin to design the next study for AST-OPC1."

Enrolling and dosing the fifth patient in this cohort triggers the final $1.5 million grant payment from the California Institute for Regenerative Medicine (CIRM) under the existing $14.3 million Strategic Partnerships Award grant awarded to Asterias. Asterias expects to receive this grant payment in the third quarter of 2017.

"On behalf of Asterias, its employees and the spinal cord injury community, I'd like to thank CIRM for its support of AST-OPC1 and the SCiStar study," stated Mike Mulroy, President and CEO of Asterias. "We look forward to continuing our relationship with CIRM as we advance this important program."

Asterias has now completed enrollment and dosing in four of the five planned SCiStar study cohorts and enrolled twenty patients in the SCiStar study. Twenty-five patients have been administered AST-OPC1 after including patients from a previous Phase 1 safety trial and results-to-date continue to support the safety of AST-OPC1, with no serious adverse events related to AST-OPC1 or its administration. In June 2017, Asterias reported 9 month data from the AIS-A 10 million cell cohort that showed improvements in arm, hand and finger function observed at 3-months and 6-months following administration of AST-OPC1 were confirmed and in some patients further increased at 9-months. The company intends to complete enrollment of the entire SCiStar study later this year, with multiple safety and efficacy readouts anticipated during the remainder of 2017 and 2018.

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 subacute, C-4 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 have retained some minimal sensory function below their injury site. AST-OPC1 is being administered 21 to 42 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 centers in the U.S. and the company plans to increase this to up to 12 sites to accommodate the expanded patient enrollment. Clinical sites involved in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical 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.

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, using Identifier NCT02302157, and at the SCiStar Study Website (

About AST-OPC1

AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells originally isolated in 1998, 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 7-14 days post-injury. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with cervical spine injuries in the current SCiStar study, which represents the first targeted population for registration trials.  Asterias has completed enrollment in the first four cohorts of this study. Results to date have continued to support the safety of AST-OPC1, with no serious adverse events related to AST-OPC1 or its administration.  Additionally, Asterias has recently reported results suggesting reduced cavitation and improved motor function in patients administered AST-OPC1 in the SCiStar trial.

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 which 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    


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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