Asterias' AST-OPC1 Clinical Trial Will Continue as Planned Following Recommendation of Data Monitoring Committee
FREMONT, Calif., Sept. 25, 2017 (GLOBE NEWSWIRE) -- Asterias Biotherapeutics, Inc. (NYSE American:AST), a biotechnology company pioneering the field of regenerative medicine, today announced that the ongoing SCiStar Phase 1/2a study of AST-OPC1 in acute spinal cord injury will continue as planned, based on the recommendation of an independent Data Monitoring Committee (DMC) after it completed a regularly scheduled review of the accumulated safety data to date from the study.
"The DMC's recommendation to continue our SCiStar study without modification reaffirms the committee's previous safety reviews of AST-OPC1, and confirms the safety data to date from the high dose 10 and 20 million cell cohorts in the study," said Dr. Edward Wirth, Chief Medical Officer of Asterias Biotherapeutics.
As specified in the SCiStar study protocol, the DMC meets on a regular basis to review data from the ongoing trial. The DMC is comprised of an independent group of medical and scientific experts and is responsible for reviewing and evaluating patient safety and efficacy data for the purpose of safeguarding the interest of study participants.
Asterias has enrolled over twenty-two subjects in the SCiStar study and more than twenty-seven subjects have been administered AST-OPC1 after including subjects from a previous Phase 1 safety trial. The results-to-date continue to support the safety of AST-OPC1.
Asterias previously reported 9-month data from the study's second dose cohort (AIS-A patients dosed 10 million cells). Subjects in this cohort showed a meaningful and favorable difference at 9 months in the recovery of arm, hand and finger function compared to the level of expected rates of spontaneous recovery shown from historical control data of a closely matched patient population. Asterias expects to report 12-month efficacy and safety data for the study's second dose cohort later this year, and 6- and 12-month efficacy and safety data for Cohort 3 (AIS-A subjects dosed 20 million cells) and Cohort 4 (AIS-B subjects dosed 10 million cells) in 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 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 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 nine centers in the U.S. 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, Santa Clara Valley Medical Center in San Jose jointly with Stanford University, Thomas Jefferson University Hospital in partnership with Magee Rehabilitation Hospital in Philadelphia, UC San Diego Health in San Diego, California, and Washington University School of Medicine in partnership with Barnes-Jewish Hospital in St. Louis, MO.
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 (www.SCiStar-study.com).
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. Additionally, Asterias has recently reported results suggesting reduced cavitation and improved motor function in patients administered AST-OPC1 in the SCiStar trial. |
