On September 26, 2017 (GLOBE NEWSWIRE) — Asterias Biotherapeutics, Inc. (NYSE MKT:AST), a biotechnology company pioneering the field of regenerative medicine, reported that the Medicines and Healthcare Products Regulatory Agency (MHRA) and the NHS Research Ethics Committee (REC) have provided the necessary approvals to initiate the first-in-human (FIH) clinical trial of AST-VAC2 in the United Kingdom (UK) (Press release, BioTime, SEP 26, 2017, View Source [SID1234520633]). The trial, which is being sponsored and managed by Cancer Research UK, will examine the safety, tolerability, immunogenicity and activity of AST-VAC2 in non-small cell lung cancer (NSCLC) patients and is expected to be initiated later this year.

AST-VAC2 is a “first-in-class” allogeneic cancer immunotherapy that is composed of mature dendritic cells which are designed to kill tumor cells by stimulating immune responses to telomerase, a tumor antigen expressed by over 85% of malignant tumor cells. AST-VAC2 is available for “on demand” patient use because it is produced from allogeneic pluripotent stem cells that can be manufactured in scale and then cryopreserved. The AST-VAC2 to be used in this trial has been manufactured by Cancer Research UK’s Biotherapeutics Development Unit.

AST-VAC2 is a platform cancer immunotherapy that could be investigated as a potential therapeutic for many cancer indications and for targeting of many antigens. The results from the clinical trial sponsored by Cancer Research UK could be used to support advanced clinical studies in one or more of the following areas:

Non-small cell lung cancer
Other indications showing high levels of telomerase activity and susceptibility to immunotherapy
In combination with check point or immune pathway inhibitors
In combination with additional antigens, including those arising from the exciting new field of tumor neoantigens
“The recently announced acquisition of Kite Pharma by Gilead for $11.9 billion provides strong validation for the cell therapy industry generally and especially in oncology,” said Mike Mulroy, President and Chief Executive Officer. “With its potential as a ready-to-administer, off-the-shelf cancer immunotherapy, AST-VAC2 represents an exciting opportunity for Asterias in the rapidly evolving immuno-oncology sector and the approvals received from MHRA and REC to commence clinical testing represent an important milestone in the development of AST-VAC2.”

The clinical trial will administer AST-VAC2 in up to twenty-four patients in two cohorts. In the first cohort, up to 12 patients with advanced non-small cell lung cancer and a specific immunological marker called HLA-A2 will receive AST-VAC2, and will be followed for safety, immune responses to telomerase, and overall clinical survival. The second cohort will evaluate AST-VAC2 in up to 12 patients with the HLA-A2 marker who have had successful resection of their tumor with no evidence of metastasis and each patient will be followed for safety, immune responses to telomerase, overall clinical survival and time to relapse. Both cohorts will have a control group consisting of patients that meet all inclusion/exclusion criteria for the study except those patients do not have the HLA-A2 marker.

“The design of the trial will allow us to assess many features of AST-VAC2 and how to best position its use in future trials,” said Jane Lebkowski, Chief Scientific Officer. “We will be testing immune responses invoked by AST-VAC2 in the settings of advanced disease and resected disease and perform intermediate assessments of immune response during the course of AST-VAC2 dosing. Clinical outcome and immune response data will help confirm whether AST-VAC2 is most beneficial for patients in an active or minimal residual disease setting and inform determination of the optimal dosing regimen for future trials. The trial will also have a concurrent control group to provide real-time assessment of the safety and activity of the product. We are very excited to begin the clinical development of AST-VAC2 which could become a cornerstone agent in the immunotherapy of cancer.”

“The AST-VAC2 study is an exciting step towards improving our tools in cancer immunotherapy,” said Professor Christian Ottensmeier, the study’s principal investigator. “Not only does the Asterias approach already have a track record in Acute Myeloid Leukemia (AML), but an ‘off the shelf’ dendritic cell vaccine opens the path towards making dendritic cell vaccination easily deliverable in the clinic. We are very excited to be working towards opening this study in the second half of 2017.”

The partnership between Asterias and Cancer Research UK is being conducted under Cancer Research UK’s Clinical Development Partnerships (CDP) scheme, which allows the first clinical trial of AST-VAC2 to be initiated without significant Asterias resources being allocated to the trial and the manufacturing of the product. On completion of the clinical trial, Asterias will have an exclusive first option to acquire the data from the trial.

About AST-VAC2

AST-VAC2 is an innovative immunotherapy product that contains mature dendritic cells derived from pluripotent stem cells. These non-patient specific (allogeneic) AST-VAC2 cells are engineered to express a modified form of telomerase, a protein widely expressed in tumor cells, but rarely found in normal cells. The modified form of telomerase invokes enhanced stimulation of immune responses to the protein. Similar to an earlier, Asterias-sponsored, hematological cancer program which provided proof-of-concept data, the AST-VAC2 dendritic cells instruct the immune system to generate responses against telomerase which will target tumor cells. AST-VAC2 is based on a specific mode of action that is complementary to and potentially synergistic with other immune therapies such as checkpoint inhibitors or other immune pathway inhibitors.