Meeting with Food and Drug Administration Remains on Track for End of 2018
FREMONT, Calif., Oct. 31, 2018 (GLOBE NEWSWIRE) — Asterias Biotherapeutics, Inc. (NYSE American: AST), a biotechnology company dedicated to developing cell-based therapeutics to treat neurological conditions associated with demyelination and cellular immunotherapies to treat cancer, today announced a positive outcome from an independent Data Review Panel’s review of the data generated by patients enrolled in the Company’s ongoing Phase 1/2a SCiStar study designed to evaluate the safety and efficacy of OPC1 in the treatment of severe cervical spinal cord injury. Based on a review of the data, the Panel recommended moving forward with the continued clinical development of OPC1. The next step in the development of OPC1 is a meeting with the Food and Drug Administration (FDA) later this year to discuss proposed next steps for the OPC1 clinical development program, including the trial design for a randomized controlled Phase 2 trial.
“We believe the the positive feedback we received from the Panel will strengthen our meeting with the Food and Drug Administration (FDA) later this year,” commented Ed Wirth, Chief Medical Officer of Asterias. “Assuming successful execution of the FDA meeting and obtainment of additional CIRM funding or alternative financing, we expect to enroll the first patient in the Phase 2 randomized controlled trial in the first half of 2020.”
Asterias worked with the California Institute for Regenerative Medicine (CIRM) to complete the review of the data under CIRM’s Clinical Advisory Panel process. The Panel comprised of outside medical and scientific experts that include James Guest, M.D., Ph.D., Professor of Clinical Neurological Surgery at the University of Miami, John Steeves, Ph.D., Co-Chair of the Spinal Cord Outcomes Partnership Endeavor (SCOPE) and Professor of International Collaboration on Repair Discoveries (ICORD) at the University of British Columbia, and Ann Parr, MD, PhD, Neurosurgeon and Director of Spinal Neurosurgery at the University of Minnesota. The Panel reviewed the most recent safety, engraftment and efficacy data from the SCiStar study including analysis of the data after removing a small subset of subjects that are likely to be excluded from next trial.
“From our review of the data, the combination of the safety of the cells and administration procedure, the level of motor recovery, and the compelling evidence of the engraftment of OPC1 cells is an unprecedented step forward for the program and spinal cord injury community,” commented Drs. Guest and Steeves.
The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of OPC1 administered at up to 20 million OPC1 cells in 25 subjects with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) spinal cord injuries. Asterias has completed enrollment and dosing in all five of its planned SCiStar study cohorts. The Company intends to report 12-month results for the entire SCiStar study in the first quarter of 2019. As previously announced, the Company has scheduled a Type B meeting with the FDA in accordance with the Regenerative Medicine Advanced Therapy (RMAT) designation under the 21st Century Cures Act.
OPC1 Therapeutic Platform
OPC1, an oligodendrocyte progenitor cell population derived from human embryonic stem cells, has been shown in preclinical testing in animals and in vitro to have three potentially reparative functions that address the complex pathologies observed in demyelination disorders, such as spinal cord injury and multiple neurodegenerative diseases, including multiple sclerosis and white matter stroke. These potential reparative functions of OPC1 include the production of neurotrophic factors, the stimulation of vascularization, and the induction of remyelination of denuded axons, all of which are critical for survival and regrowth of—and conduction of nerve impulses through—axons at the injury site.
Each year in the United States, more than 17,000 people suffer a severe, debilitating spinal cord injury. As of 2016, the National Spinal Cord Injury Statistical Center reported that approximately 4,500 of these new spinal cord injuries annually in the U. S. are AIS-A, AIS-B, or AIS-C patients with C-4 to C-7 spinal cord injuries (https://www.nscisc.uab.edu/). 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 meaningfully lower healthcare costs, significant improvements in quality of life, greater 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 OPC1 administered at up to 20 million OPC1 cells in 25 subjects with subacute motor complete (AIS-A or AIS-B) cervical (C-4 to C-7) SCI. These individuals have essentially lost all movement below their injury site and experience severe paralysis of the upper and lower limbs. AIS-A subjects have lost all motor and sensory function below their injury site, while AIS-B subjects have lost all motor function but may have retained some minimal sensory function below their injury site. OPC1 is administered 21 to 42 days post-injury. Subjects will be followed by neurological exams and imaging procedures to assess the safety and activity of the product.
Asterias has received a Strategic Partnerships Award grant from the California Institute for Regenerative Medicine, which provided $14.3 million of non-dilutive funding for the Phase 1/2a clinical trial and other product development activities for 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 (www.SCiStar-study.com).
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a biotechnology company dedicated to developing cell-based therapeutics to treat neurological conditions associated with demyelination and cellular immunotherapies to treat cancer. Asterias is presently focused on advancing two clinical-stage programs which have the potential to address areas of high unmet medical need in the fields of neurology and oncology. OPC1 (oligodendrocyte progenitor cells) is currently in a Phase 1/2a dose escalation clinical trial in spinal cord injury. VAC2 (antigen-presenting allogeneic dendritic cells) is an allogeneic cancer immunotherapy. The Company’s research partner, Cancer Research UK, has commenced a first-in-human clinical trial of VAC2 in non-small cell lung cancer. Asterias is also sponsoring pre-clinical work in two conditions with a demyelinating component: Multiple Sclerosis and White Matter Stroke, and is evaluating other cancer indications where its immunotherapy platform could provide therapeutic benefit. Additional information about Asterias can be found at www.asteriasbiotherapeutics.com.