First Round of Patient Enrollment Completed in Clinical Trial of Investigational Product to Treat Newly Injured People with Cervical Spinal Cord Injury
Researchers confirm safety of injection procedure and expect to increase dose in second round of patient enrollment in the study.
Asterias Biotherapeutics Inc., a biotechnology company focused on the emerging field of regenerative medicine, announced today that it has completed the first round of patient enrollment in a Phase 1/2a clinical trial of escalating doses of an investigational product to treat newly injured people with cervical spinal cord injury.
The first patient in this safety cohort was treated at Shepherd Center in early June. The second and third patients were successfully dosed at Chicago-based Rush University Medical Center. Asterias officials said the results of the clinical trial – called SCi-STAR – studying the investigational product, called AST-OPC1, continue to support a robust safety profile with no serious adverse events observed in any of the three treated patients to date. These patients received the initial low dose of 2 million cells, intended to test the safety of the injection procedure used to treat these patients.
The patient treated at Shepherd Center has completed the two-month post-injection assessment and has progressed from a complete ASIA Impairment Scale (AIS) A injury to an incomplete AIS B injury. The principal investigator at Shepherd Center, Medical Director Donald Peck Leslie, M.D., said, “This progress in the first patient is very encouraging and is observed in less than 5 percent of our AIS A patients at this stage of their recovery.”
The study’s lead neurosurgeon, Richard Fessler, M.D., from Rush University Medical Center, performed the AST-OPC1 injections in the second and third patients. “The injection procedure went very smoothly for both patients, and there were no complications,” Dr. Fessler said. “Both patients recovered quickly from the injection surgery and were able to resume their rehabilitation programs soon afterward.”
Asterias expects to begin enrollment of a second group of study participants following Data Monitoring Committee review of the 30-day, post-injection safety data from all three patients. The second cohort will enroll five patients who will receive 10 million AST-OPC1 cells.
“The safety data in this first cohort now paves the way for testing the higher doses of AST-OPC1 (10-20 million cells) that we believe correspond most closely to the doses that showed the greatest efficacy in animal studies,” said Edward Wirth, M.D., Ph.D., chief medical officer of Asterias.
The open-label, single-arm study is being conducted at three medical facilities, including Shepherd Center, and will include up to 12 centers in the United States. Enrollment in the trial has begun to accelerate, with seven weeks elapsed between dosing the first and second patients, and only three weeks between dosing the second and third patients.
“We are encouraged by this performance improvement and are confident in meeting our disclosed timelines,” said Pedro Lichtinger, president and CEO of Asterias. “We expect to provide updates as identified milestones are reached or when major events occur.”
The SCi-STAR trial will test three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in 13 patients with sub-acute, C-5 to C-7, neurologically complete cervical spinal cord injury. 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. Additional information on the Phase 1/2a study, including trial sites, can be found at www.clinicaltrials.gov, using Identifier NCT02302157, and at the SCiStar study website (www.scistarstudy.com).
Upon achievement of initial safety data from the first two cohorts of this study, Asterias plans to seek concurrence from the U.S. Food and Drug Administration 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. Asterias believes this change 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.
More than 12,000 people sustain a spinal cord injury each year, but there are no FDA-approved therapeutics or devices that could potentially restore some function in individuals who have recently sustained a spinal cord injury.
All clinical trial participants must be patients who are admitted to Shepherd Center. Also, clinical trial eligibility requirements apply. Medical professionals are invited to 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 firstname.lastname@example.org.
For more information about Asterias, visit www.asteriasbiotherapeutics.com.
Shepherd Center, located in Atlanta, Georgia, is a private, not-for-profit hospital specializing in medical treatment, research and rehabilitation for people with spinal cord injury, brain injury, multiple sclerosis, spine and chronic pain, and other neuromuscular conditions. Founded in 1975, Shepherd Center is ranked by U.S. News & World Report among the top 10 rehabilitation hospitals in the nation. In its more than four decades, Shepherd Center has grown from a six-bed rehabilitation unit to a world-renowned, 152-bed hospital that treats more than 740 inpatients, nearly 280 day program patients and more than 7,100 outpatients each year in more than 46,000 visits.