-Cohort of SCiSTAR trial patients with complete cervical spinal cord injuries reaches the targeted 6-12 month efficacy endpoint within 3 months-
-Dose response based on number of AST-OPC1 cells administered appears to be emerging-
-Asterias to Host Conference Call Today at 4:30pm ET to Discuss Efficacy Data Presented at the 55th Annual Scientific Meeting of the International Spinal Cord Society-
FREMONT, Calif. – Sept. 14, 2016 – Asterias Biotherapeutics, Inc. (NYSE MKT: AST), today presented positive interim efficacy data from the 10 million cell cohort in the Company’s ongoing AST-OPC1 SCiSTAR Phase 1/2a multicenter clinical study in complete cervical spinal cord injury patients. While early in the study, with only 4 of the 5 patients in the cohort having reached 90 days after dosing, all patients have shown at least one motor level of improvement so far and the efficacy target of 2 of 5 patients in the cohort achieving two motor levels of improvement on at least one side of their body has already been achieved. Patient improvements are being measured by the ISNCSCI neurological classification scale widely used to quantify functional status of patients with spinal cord injuries. As suggested by existing research, patients with complete cervical spinal cord injuries that show two motor levels of improvement on at least one side may regain the ability to perform daily activities such as feeding, dressing and bathing.
The efficacy target was recommended by the independent Spinal Cord Outcomes Partnership Endeavor (SCOPE) based on recently published data (Steeves et al., Topics in Spinal Cord Injury Rehabilitation, 2012). SCOPE is affiliated with the American Spinal Injury Association.
“I am very encouraged by this first look at efficacy data for AST-OPC1 in complete cervical spinal cord injury patients,” commented Shekar N. Kurpad, MD, PhD, Professor of Neurosurgery, Director, Spinal Cord Injury Center Medical College of Wisconsin. “As a clinical investigator in the trial, I am looking forward to continuing the evaluation of this promising new treatment in these patients, for whom effective new therapeutic options are desperately needed. I am hopeful that we are able to see these early, positive efficacy trends continue as the study progresses.”
“The results to date in the 10 million cell cohort, while still early, demonstrate meaningful improvement in motor function, particularly in the use of a patient’s hands, fingers and arms, which is critically important for a patient’s quality of life and ability to function independently,” said Steve Cartt, Chief Executive Officer of Asterias. “We are quite encouraged by this first look at efficacy results and look forward to reporting six-month efficacy data as planned in January 2017. We have also just recently been cleared to begin enrolling a new cohort and administering to these new patients a much higher dose of 20 million cells. We look forward to begin evaluating efficacy results in this higher-dose cohort in the coming months as well.”
The SCiStar study is funded in part by a $14.3 million grant from the California Institute for Regenerative Medicine (CIRM).
The data were presented by Edward Wirth, MD, PhD, Chief Medical Officer of Asterias, at the 55th Annual Scientific Meeting of the International Spinal Cord Society (ISCoS) in Vienna, Austria and included the following highlights:
Efficacy and Safety Results: Cohort 2 dosed with 10 million cells
- 5 out of 5 patients dosed with 10 million cells have exhibited improved upper extremity motor scores (UEMS) relative to baseline.
- At Day 90 of follow up, 4 of 4 patients dosed have improved one motor level on at least one side, 2 of 4 patients have improved two motor levels on at least one side, and 1 patient has improved two motor levels on both sides.
- The average UEMS improvement at Day 90 for the 4 patients that have reached this follow up was 9.5 points.
- The results to date from Cohort 2 show no serious adverse events related to AST-OPC1, the injection procedure, or immunosuppression with low-dose tacrolimus. In addition, data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.
Efficacy and Safety Results: Cohort 1 dosed with 2 million cells
- 3 out of 3 patients have exhibited improved upper extremity motor scores (UEMS) relative to baseline.
- The average UEMS improvement for the 3 patients was 5.0 points at Day 90, and they continued to improve an average of 7.0 points at 1 year.
- Despite the very low dose used in this safety cohort, at 1 year of follow up 1 patient in Cohort 1 has improved one motor level on one side and 2 patients have improved one motor level on both sides.
- The 12-month results from Cohort 1 show no serious adverse events related to AST-OPC1, the injection procedure, or immunosuppression with low-dose tacrolimus. In addition, data from the study indicate that AST-OPC1 can be safely administered to patients in the subacute period after severe cervical spinal cord injury.
The results from Cohort 2 dosed with 10 million cells compared to those from Cohort 1 dosed with 2 million cells may also begin to support a dose response as patients dosed in the earlier safety cohort with 2 million cells saw an average upper extremity motor score (arms, hands, fingers) increase of 5.0 at Day 90, while the 10 million cell cohort has experienced an average increase of 9.5 at Day 90. The company expects to have data evaluating the efficacy results after implantation of 20 million AST-OPC1 cells in complete cervical spinal cord injury patients later in 2017.
For additional information on the presentation data presented, please see the ISCoS conference presentation on the company’s website at: http://asteriasbiotherapeutics.com/wp-content/uploads/2016/09/Wirth-ISCoS-14SEP2016-talk-FINAL.pdf.
Data evaluating the efficacy results six months after implantation of 10 million AST-OPC1 cells in complete cervical spinal cord injury patients will be available in January 2017 and will focus on improvement in physical functioning of the upper extremities (fingers, hands and arms) of each treated patient utilizing scoring on the International Standards for Neurological Classification of Spinal Cord Injury (ISNCSCI scale).
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 functional capabilities of spinal cord injury patients can result in significant improvements in daily living, less required care by an attendant and lower cost of care for patients.
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 12 to accommodate the expanded patient enrollment. The five patients in the second 10 million cell cohort were treated at Medical College of Wisconsin in Milwaukee, Shepherd Medical Center in Atlanta, University of Southern California (USC) in Los Angeles and Santa Clara Valley Medical Center in Santa Clara.
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 the administration of the cells, with AST-OPC1 itself, or the immunosuppressive regimen. 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.
Conference Call Information
Asterias’ management will host a conference call and webcast today, September 14, 2016, at 4:30 p.m. Eastern/1:30 p.m. Pacific, to discuss the data. Joining the Asterias management team on the call is Charles Liu, MD, PhD, director of the USC Neurorestoration Center. USC Keck School of Medicine, which is one of the trail sites, recently provided an update on one of the patients participating in the study. 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 888-224-1005. For international participants outside the U.S./Canada, the dial-in number is 913-312-0863. For all callers, refer to Conference ID 7322708. To access the live webcast, go to http://asteriasbiotherapeutics.com/events-presentations/.
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 7322708. Additionally, the archived webcast will be available at http://asteriasbiotherapeutics.com/events-presentations/.
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a leading biotechnology company in the emerging 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 oncology and neurology. 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 being evaluated by Asterias for further development after demonstrating promise in a Phase 2 study in Acute Myeloid Leukemia (AML) and completing a successful end-of-Phase 2 meeting with the FDA. 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 1/2 clinical trial of AST-VAC2 in non-small cell lung cancer in 2017. Additional information about Asterias can be found at www.asteriasbiotherapeutics.com.
Forward Looking Statements
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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