FREMONT, Calif., Jan. 24, 2017 /PRNewswire/ -- Asterias Biotherapeutics, Inc. (NYSE MKT: AST), a biotechnology company pioneering the field of regenerative medicine, today announced positive efficacy results from the company's ongoing SCiStar Phase 1/2a clinical trial that showed additional motor function improvement at 6-months and 9-months following administration of 10 million AST-OPC1 cells in AIS-A patients with complete cervical spinal cord injuries (SCI).
"Recovery of upper extremity motor function is critically important to patients with complete cervical spinal cord injuries, since this can dramatically improve quality of life and their ability to live independently," said Richard Fessler, M.D., Professor, Department of Neurosurgery at Rush University Medical Center and lead investigator in the SCiStar study. "Patients in this cohort are seeing what we believe are meaningful improvements in their ability to use their arms, hands and fingers at 6-months and 9-months following AST-OPC1 administration."
"The results to date in the 10 million cell cohort treated with AST-OPC1 cells show that the improvements in arm, hand and finger function observed very early in the study have been maintained and in most patients have even been further enhanced over time," said Steve Cartt, Chief Executive Officer of Asterias. "These results to date are quite encouraging, and we look forward to initiating discussions with the FDA in mid-2017 to begin to determine the most appropriate clinical and regulatory path forward for this innovative therapy. In addition, we anticipate reporting 12-month efficacy and safety data for this cohort, as well as 6-month efficacy and safety data for the currently-enrolling AIS-A 20 million cell and AIS-B 10 million cell cohorts, during the third quarter of 2017."
A total of six patients were enrolled and dosed in the AIS-A 10 million-cell cohort, with five of six patients having now completed their 6-month follow-up and three of six patients having completed their 9-month follow-up.
Improvements in upper extremity motor function are being measured using the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI) scale, widely used to quantify functional status of patients with spinal cord injuries. The latest results include the following highlights:
Improvements in Motor Function
- Upper Extremity Motor Score - For the five patients who have completed at least 6 months of follow-up, five of five patients saw their early improvements in motor score (UEMS) at 3 months maintained or further increased through their most recent data point (6 months or 9 months, depending on the most recent data available for each patient).
- Motor Level Improvement - For patients completing at least 6 months of follow up, as of the date of each patient's last follow-up visit, 100% (five of five) had achieved at least a one motor level improvement (using the ISNCSCI scale) over baseline on at least one side, and 40% (two of five) had achieved two motor levels over baseline on at least one side, with one of these patients achieving a two motor level improvement on both sides.
- Matched Historical Control Data - Asterias and key experts in the spinal cord injury field have developed a set of matched historical control data for both Upper Extremity Motor Score and Motor Level Improvement to clearly document expected spontaneous recovery in untreated patients for comparison to results seen in patients treated with AST-OPC1. The key results from this analysis, which show a meaningful difference in the motor function recovery seen to date in patients treated with the 10 million cell dose of AST-OPC1, will be presented during today's conference call.
- The trial results to date continue to reveal a positive safety profile for AST-OPC1. There have been no serious adverse events related to AST-OPC1 and data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.
In September 2016, Asterias reported positive early efficacy data for AST-OPC1 from the AIS-A patients that had been dosed with 10 million AST-OPC1 cells in the SCiStar study. Today's data show that the improvements in motor function seen at 3 months post-implantation of AST-OPC1 for these patients appear to be maintained, and in some cases improved even further, on continued follow up. Furthermore, these results suggest that the observed gains exceed the expected rates of spontaneous recovery in a closely matched population of historical controls.
Each year in the U.S. more than 17,000 people suffer a severe, debilitating spinal cord injury. 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 lower healthcare costs, significant improvements in quality of life, increased ability to engage in activities of daily living, and increased independence.
Conference Call and Webcast Today at 5:00 p.m. ET (2:00 p.m. PT) to Discuss the Results
Asterias will host a conference call and webcast today, January 24, 2017 at 5:00 p.m. Eastern / 2:00 p.m. Pacific to discuss the results. Company management will be joined by Richard G. Fessler, MD, PhD, Professor, Department of Neurosurgery at Rush University Medical Center and SCiStar lead investigator, and John Steeves, Ph.D., Co-Chair of the Spinal Cord Outcomes Partnership Endeavor (SCOPE) and Professor of International Collaboration on Repair Discoveries (ICORD).
For both "listen-only" participants and those participants who wish to take part in the question-and-answer portion of the call, the dial-in number in the U.S./Canada is 888-737-3705. For international participants outside the U.S./Canada, the dial-in number is 913-312-0951. For all callers, refer to Conference ID 8558670. To access the live webcast, go to http://asteriasbiotherapeutics.com/inv_events_presentations.php.
A replay of the conference call will be available for one month beginning about two hours after the conclusion of the live call, by calling toll-free (from U.S./Canada) 888-203-1112; international callers dial 719-457-0820. Use the Conference ID 8558670. Additionally, the archived webcast will be available at http://asteriasbiotherapeutics.com/inv_events_presentations.php.
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 that have enrolled and dosed patients in the study include the Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) in Los Angeles, Rush University Medical Center in Chicago and Santa Clara Valley Medical Center in San Jose.
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 immunotherapy and pluripotent stem cell 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) and completing a successful end-of-Phase 2 meeting with the FDA. The company is currently focused on streamlining and modernizing the manufacturing process for AST-VAC1 in advance of a planned initiation of a Phase 2b study designed to set the registration pathway. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic 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 the second quarter of 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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SOURCE Asterias Biotherapeutics, Inc.