• Stem cell research has long held tremendous promise for curing some of humanity’s most severe ailments, including cancer and severe paralysis. Though the FDA has yet to approve any form of stem cell therapy, there are some very promising candidates. Part of the reason there are no approved stem cell therapies is because the safety requirements imposed by the FDA for cell therapies have been incredibly daunting, even by pharmaceutical standards. Pre-clinical and safety studies alone can take years to complete. However, if a company were able to successfully develop a cell therapy that earned FDA approval, the spoils of victory would be tremendous.

    In searching for promising companies within this space, we came across Asterias Biotherapeutics. Asterias (NYSE MKT: AST) which owns technology platforms that will specifically target cancer and neurological conditions. What caught our attention is the fact that Asterias has completed their pre-clinical work and initial safety studies for one of its indications (cervical spinal cord injury) and is currently conducting a Phase 1/2a study, called the SCiStar study, with some highly encouraging efficacy results to date. One of its cancer immunotherapy programs also has positive safety and efficacy data in AML and a second cancer immunotherapy program is expected to initiate testing in lung cancer later this year.

    Asterias has developed three initial cell therapy programs: AST-OPC1, a cell therapy being investigated to treat severe spinal cord injuries; and two cancer immunotherapy programs, AST-VAC1 and AST-VAC2. The company uses an embryonic stem cell line that has been around since the 1990s for its AST-OPC1 and AST-VAC2 programs, and this source material can supply an entire lifecycle of product, which eliminates variability and ensures consistency.

    Their current programs are also foundational, meaning that if any of these programs show promise moving forward, the underlying technology supporting their current programs can also be used to develop therapies for other indications such as Multiple Sclerosis, Alzheimer’s, stroke, ALS, and other forms of cancer.

    Additionally, Asterias may have some near-term catalysts. By October of this year, Asterias expects to receive its full year efficacy results from its first set of patients treated in the AST-OPC1 SCiStar study for spinal cord injury. So far, through the first 9 months, the data for these patients has been promising. If the positive results persist through the first full year, then Asterias will be the first company, of which we’re aware, to show meaningful results at 12 months for patients with severe cervical spinal cord injuries. The company also has indicated that their AST-VAC2 program could enter the clinic as soon as this year, which would likely have initial data readouts beginning in 2018.


    The spinal cord is made of soft tissue that extends from the base of the brain to a little above the waist. Along the spine are nerve cells and nerve groups called “tracts,” which connect to different parts of the body. These tracts carry messages from the brain to the rest of the body, whether it involves moving muscles or sensing temperature, pain, etc. The tracts do this by using nerve fibers that send electric signals to each other through their axons surrounded by a myelin sheath, which coats the axon and allows it to send the electric signal. Paralysis occurs when the nerve fibers that run through the injured area are damaged, specifically when the axons disconnect or when the myelin sheath becomes bare, disconnecting the brain from muscles in the body and preventing signals from being transmitted.

    Asterias offers an innovative approach to treating spinal cord injury by using a specific cell type created from pluripotent stem cells. Pluripotent stem calls can be developed into any type of cell in the human body. In the case of spinal cord injury, the company creates a cell type called oligodendrocyte progenitor cells (OPCs) from their pluripotent stem cell technology to supplement the body’s internal supply of OPCs, the cells that create myelin sheaths around nerve axons. The company’s AST-OPC1 product is believed to have several benefits that help patients suffering from spinal cord injuries, including preventing an injury cavity from being formed post injury, regenerating nerve tissue, and remyelinating nerve axons that have lost their myelin sheath after a spinal cord injury due to the body’s own OPCs in the spinal cord dying off after the injury.

    Asterias is currently conducting a Phase 1/2a clinical study involving administration of AST-OPC1 cells into patients with subacute, C-4 through C-7 spinal cord injuries, meaning these patients have lost all or almost all motor function (and in some cases all sensory function) below their neck. Recent results have been very encouraging. In a Q1 press release in 2017, Asterias reported that patients with severe paralysis of the upper limbs were able to have significant improvement in their ability to use their arms, hands, and fingers nine months after administration of AST-OPC1.

    Take a look at this company video – we think it’s pretty amazing.

    AST-OPC1 has received $14.3 million of non-dilutive funding from the California Institute for Regenerative Medicine (CIRM) for their Phase 1/2a clinical trial. We also believe that the company may be able to receive another non-dilutive grant from CIRM for its next clinical trial for AST-OPC1.

    AST-OPC1 cells have the potential mechanisms of action to also help patients suffering from stroke, MS, Alzheimer’s, and ALS, meaning these cells may prove to be a reliable, multi-faceted solution to a wide range of unsolved problems in medicine.

    In July, Asterias announced completion of enrollment and dosing of two additional cohorts of patients in the AST-OPC1 study. The enrollment of these cohorts has added 10 patients (for a total of 16) that have now been administered potentially efficacious doses of AST-OPC1 cells and the company expects to report top-line six-month results from these cohorts in January 2018.

    Completion of this milestone also triggered the final $1.5 million grant payment from CIRM under the existing $14.3 million Strategic Partnerships Award grant awarded to Asterias. Asterias expects to receive this grant payment in the third quarter of 2017.

    The company intends to complete enrollment of the entire SCiStar study later this year, with multiple safety and efficacy readouts anticipated during the remainder of 2017 and 2018.


    Asterias intends on using immunotherapy as a means for treating cancer. Immunotherapy treats a disease by stimulating the immune system to attack cancer cells. Asterias’ goal is to utilize the process by creating vaccines that target telomerase, a protein that is found exclusively in over 95% of cancer cells. These vaccines use dendritic cells, which show the immune system what certain cancerous antigens look like so that the immune system can attack and kill the cancerous cells. Asterias’ vaccines also use the LAMP sequence which optimizes the dendritic cells’ function and optimizes immune responses to telomerase. These three key approaches are what give Asterias’ vaccines a differentiated approach to triggering immune responses to cancer cells. Additionally, since telomerase is not normally expressed in normal adult cells, immunotherapies targeting telomerase (including Asterias’ AST-VAC1 program) have shown a strong safety profile in numerous clinical trials.

    Asterias is developing two vaccines at the moment: AST-VAC1 and AST-VAC2. AST-VAC1 is a patient-specific vaccine that is created by using cells sourced from the patient, whereas AST-VAC2 is not specific to a patient and is manufactured from Asterias’ own pluripotent stem cell platform.

    The AST-VAC vaccines are also subject to scalable manufacturing, so a single manufacturing process can produce multiple doses. Also, because AST-VAC2 is non-patient specific, it can be mass produced, which cuts down on costs and time relative to other patient-specific therapeutic options.

    A Phase 2 trial of AST-VAC1 in Acute Myeloid Leukemia (AML) showed a significant improvement in relapse free survival rates for high risk AML patients. The company views this as proof of concept data for its cancer immunotherapy technology and AST-VAC2 program that will be entering the clinic later this year.

    Asterias will be testing AST-VAC2 in a Phase 1/2a clinical study in non-small cell lung cancer, funded by Cancer Research-UK (CRUK), expected to have its first patient dosed later in 2017. If this study yields positive results, then it will show that immunotherapy from pluripotent stem cells can be used to target non-small cell lung cancer, which accounts for 80-85% of lung cancer, and other forms of cancer. With nearly 222,500 new cases of lung cancer in the U.S. in 2017 alone and 1 out of 4 cancer deaths stemming from lung cancer, it is clear that any treatment option that is safe and that works can be a lucrative find.

    We believe Asterias could become a leader in cell therapy. With the pre-clinical work behind them, and promising efficacy results to date in two of its programs, the company has already achieved a number of significant milestones to move its programs forward. There are potentially more milestones in 2017, so it appears to us as if the company may be at an inflection point. We like the fact that Asterias has received non-dilutive grant funding, and is positioned to potentially receive more of the same. We find Asterias to be a compelling investment opportunity with considerable upside potential upon continued validation of their clinical studies and non-dilutive funding from third parties.

    Disclaimers & Disclosures: For a full list of disclaimers and disclosures, please visit:

    Contact: [email protected]


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