Menlo Park, Calif., March 5, 2015 – Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a leading biotechnology company in the emerging field of regenerative medicine, announced today that Atlanta-based Shepherd Center, one of the nation’s top rehabilitation hospitals for spinal cord injury and brain injury, has commenced enrollment for the Phase 1/2a clinical trial of AST-OPC1 (oligodendrocyte progenitor cells) in newly injured patients with sensory and motor complete cervical spinal cord injury (SCI).
The Phase 1/2a trial follows the successful completion of the Phase 1 trial of AST-OPC1, which met its primary endpoints of safety and feasibility when administered to five patients with neurologically complete, thoracic SCI. Shepherd Center was a site in the Phase 1 study and enrolled two of the five subjects in that study. Asterias intends to initiate enrollment for the Phase 1/2a trial at up to seven additional sites in the coming months.
“The Phase 1 study generated a strong package of data regarding the safety of AST-OPC1,” said Donald Peck Leslie, M.D., medical director of Shepherd Center and principal investigator for this study site. “With the Phase 1/2a clinical trial, we are excited to begin testing AST-OPC1 at the doses and in the population where it has the maximum potential to bring benefit to patients.”
Richard G. Fessler, MD, PhD, professor of neurological surgery at Rush University Medical Center and principal investigator for the Phase 1 clinical trial, said, “There are currently no FDA-approved therapeutics or devices for the more than 12,000 individuals who sustain an SCI each year in the United States alone, or for the approximately 1.3 million Americans who are estimated to be living with an SCI. If AST-OPC1 could deliver even modest improvements in motor or sensory function, it would result in significant improvements in quality of life.”
The Phase 1/2a clinical trial is designed to assess safety and activity of escalating doses of AST-OPC1 for complete cervical SCI, the first targeted indication for AST-OPC1. The trial will be an open-label, single-arm study testing three escalating doses of AST-OPC1 in patients with sub-acute, C-5 to C-7, neurologically complete cervical SCI. These individuals have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs. AST-OPC1 will be administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging methods to assess the safety and activity of the product.
The initiation of this trial is in line with the recently announced initiative to accelerate the current timelines for the AST-OPC1 clinical program by approximately six months. In addition, the company plans to seek FDA concurrence to increase the robustness of the proof of concept in the Phase 1/2a clinical trial by expanding enrollment from 13 patients to up to 40 patients. The company believes these changes will increase the statistical confidence of the safety and efficacy readouts, reduce the risks of the AST-OPC1 program and position the product for potential accelerated regulatory approvals. 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.
“The initiation of our Phase 1/2a trial is a significant achievement for Asterias, putting us on track to achieve the accelerated timelines that we recently announced and moving us closer to realizing the value of AST-OPC1,” said Pedro Lichtinger, President and CEO of Asterias. “Individuals with SCI have severe disabilities that can significantly shorten projected lifespan, impact quality of life and result in lifetime costs of care of $3 million to $4 million. AST-OPC1 has been shown to have multiple reparative functions that address the complex pathologies observed at the spinal cord injury site and improve function in animal models. We look forward to testing the safety and activity of AST-OPC1 in this Phase 1/2a trial in our target population for first approval, complete cervical spinal cord injury.”
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. (NYSE MKT: AST) is a leading biotechnology company in the emerging field of regenerative medicine. The Company’s core technologies center on pluripotent stem cells, which are characterized by the ability to become all cell types in the human body. Asterias is focused on developing therapies based on pluripotent stem cells to treat diseases or serious injuries in several medical areas where there is high unmet medical need and without adequate available therapies. Asterias’ two therapeutic programs, AST-OPC1 (oligodendrocyte progenitor cells) for spinal cord injuries and AST-VAC2 (antigen-presenting allogeneic dendritic cells) for lung cancer, are based on the Company’s proprietary technology platforms of Pluripotent Stem Cells and Allogeneic Dendritic Cell Immunotherapy, respectively. AST-OPC1 is currently in a Phase 1/2a clinical trial. Additional information about Asterias can be found at www.asteriasbiotherapeutics.com.
About Shepherd Center
Shepherd Center, located in Atlanta, Ga., is a private, not-for-profit hospital specializing in medical treatment, research and rehabilitation for people with spinal cord injury or brain injury. Founded in 1975, Shepherd Center is ranked by U.S. News & World Report among the top 10 rehabilitation hospitals in the nation and is a 152-bed facility. Last year Shepherd Center had 965 admissions to its inpatient programs and 571 to its day patient programs. In addition, Shepherd Center sees more than 6,600 people annually on an outpatient basis. For more information, visit Shepherd Center online at www.shepherd.org
Statements pertaining to future financial and/or operating 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.