Asterias Biotherapeutics Announces Data Monitoring Committee Unanimously Recommends Continuation Of Scistar Phase I/IIa Clinical Trial Of AST-OPC1 For Cervical Spinal Cord Injury
Published: Apr 25, 2017
FREMONT, Calif., April 25, 2017 /PRNewswire/ -- Asterias Biotherapeutics, Inc. (NYSE MKT: AST) today announced that following a regularly scheduled interim review of safety data from its SCiStar Phase 1/2a clinical trial of AST-OPC1 for acute spinal cord injury, the study's Data Monitoring Committee (DMC) recommended continuation of enrollment for the 10 million cell and 20 million cell dose cohorts in the study, as planned.
"The DMC's recommendation to continue our SCiStar study without modification reaffirms the committee's previous safety review of AST-OPC1 in August 2016, and confirms initial safety data from the high dose cohorts (10 million and 20 million AST-OPC1 cells) in the study," said Dr. Edward Wirth, Chief Medical Officer of Asterias Biotherapeutics. "Based on their review of all the available study data, the DMC has concluded that AST-OPC1 is safe for acute spinal cord injury patients and that the SCiStar study is being conducted according to the highest clinical research standards. This is another important step for the SCiStar study as we continue clinical development of AST-OPC1."
The DMC reviewed all of the accumulated safety data to date. The review included the safety data from six complete cervical injury (AIS-A) patients dosed with 10 million AST-OPC1 cells in Cohort 2, each of whom have completed at least six months of follow-up, as well as initial safety data from the currently enrolling Cohorts 3 and 4. Cohort 3 is enrolling AIS-A patients dosed with the highest dose of 20 million cells; Cohort 4 is testing 10 million cells in patients with less severe AIS-B incomplete cervical spinal cord injuries. In addition, the DMC reviewed the ongoing long-term safety data from the study's initial cohort of three patients dosed with 2 million cells, all of whom completed 12 months of follow-up in 2016.
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 safeguarding the interest of study participants.
Asterias previously reported on positive early efficacy data from Cohort 2 (AIS-A; 10 million cells) of the SCiStar study. Patients from this cohort showed improvement in upper extremity motor function at 3-months following administration of AST-OPC1 and maintained or further increased this improvement at 6-months and 9-months. The results suggest a meaningful and favorable difference to date in recovery of arm, hand and finger function in patients treated with the 10 million cell dose of AST-OPC1 as 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 additional efficacy and safety data for Cohort 2, as well as for the currently-enrolling Cohorts 3 and 4, later this year.
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 sub-acute, C-5 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 retain some minimal sensory function below their injury site. AST-OPC1 is being administered 14 to 30 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.
AST-OPC1, an oligodendrocyte progenitor population derived from human embryonic stem cells, 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. They also received low levels of immunosuppression for the next 60 days. Delivery of AST-OPC1 was successful in all five subjects with no serious adverse events associated with AST-OPC1. No evidence of rejection of AST-OPC1 was observed in detailed immune response monitoring of all patients. In four of the five patients, serial MRI scans indicated that reduced spinal cord cavitation may have occurred. Based on the results of this study, Asterias received clearance from FDA to progress testing of AST-OPC1 to patients with complete cervical spine injuries, which represents the first targeted population for registration trials.
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 www.asteriasbiotherapeutics.com.
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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