AST-VAC1 and AST-VAC2 are BioTime company Asterias Biotherapeutics’ (NYSE MKT: AST) telomerase-based dendritic cell immunotherapy products designed to target and destroy tumor cells. Telomerase is an unprecedented target, found to be abnormally expressed in approximately 95% of cancer types. AST-VAC2, Asterias’ next-generation telomerase-based cancer immunotherapy is advancing toward clinical development for the treatment of Non-Small Cell Lung Cancer (NSCLC). Asterias is working with Cancer Research UK (CRUK) to conduct the initial Phase I/IIa clinical trial of AST-VAC2 using CRUK’s non-dilutive funding.

AST-VAC1 is an autologous dendritic cell immunotherapy that uses a patient’s own cells as a cancer vaccine. Long-term follow-up data from a Phase II trial of AST-VAC1 in Acute Myelogenous Leukemia (AML) patients showed 58% of patients to be relapse-free after a median follow up period of 52 weeks. These findings were selected for oral presentation at the annual meeting of the American Society of Clinical Oncology (ASCO) in May 2015. This new data suggest that targeting telomerase through an autologous dendritic cell immunotherapy approach may prolong remission duration in patients with high-risk AML and warrant additional study in future trials.

In development by Asterias Biotherapeutics, AST-VAC2 is based on Asterias’ proprietary technology platform of allogeneic dendritic cell immunotherapy, using embryonic stem cells rather than the patient’s own cells. It is designed to stimulate patient immune responses to telomerase, which is abnormally expressed in over 95% of human cancers. The use of human embryonic stem cells as opposed to patient’s own cells, as the starting material for production of AST-VAC2 provides a scalable system for the production of a large number of vaccine doses in a single production lot, enabling reduced costs of manufacturing, “off-the-shelf” availability, and improved product consistency.


Positive New AST-VAC1 Data Leads to Development of AST-VAC2

The positive long-term findings with AST-VAC1 provide additional clinical rationale that support our focus on moving our AST-VAC2 telomerase-based allogeneic dendritic cell immunotherapy cancer vaccine development program into a Phase 1/2a clinical trial for lung cancer, as well as continuing development strategies for the AST-VAC1 program.

Phase I/IIa Trial

Asterias Biotherapeutics has partnered with Cancer Research UK (CRUK) to conduct a Phase I/IIa dose-escalation clinical trial for AST-VAC2 immunotherapy for Non-Small Cell Lung Cancer (NSCLC).

The trial will evaluate:

  • the safety and tolerability of the vaccine
  • the feasibility and stimulation of patient immune responses to telomerase and AST-VAC2
  • clinical outcomes after AST-VAC2 administration in patients with resected early-stage lung cancer as well as in patients with advanced forms of the disease

Under the terms of the deal with CRUK, Asterias will complete development of the manufacturing process for AST-VAC2. CRUK will then produce the vaccine and conduct the Phase I/II trial in the UK.

CRUK will provide personnel and funding for cGMP manufacturing and regulatory filings, as well as for actually conducting the trial.

The trial will be conducted as follows:

  • Five patients with resected NSCLC will receive six vaccinations of one million AST-VAC2 cells
  • 12 patients with resected NSCLC will receive six vaccinations of 10 million AST-VAC2 cells
  • 12 patients with resected NSCLC will receive six vaccinations of 10 million AST-VAC2 cells

The trial is anticipated to start in the first half of 2016.

On completion of clinical trial, Asterias will have an exclusive first option to acquire a license to the data from the trial on pre-agreed terms.

Following completion of the trial, Asterias’ management will evaluate developing its telomerase-based dendritic cell immunotherapy cancer vaccine program to address additional types of cancers.

Additional information about the trial may be found at

Scientific references for AST-VAC2 may be found here.

Unmet need and Size of Market

  • Non-Small Cell Lung Cancer (NSCLC) accounts for 85% of all lung cancers in the United States
  • The immune-oncology market is projected to reach $35 billion in annual revenue by the year 2024

Intellectual Property

Asterias Biotherapeutics’ products and technologies are protected by a strong portfolio of intellectual property developed internally and licensed from other entities.

About Asterias Biotherapeutics

BioTime subsidiary, Asterias Biotherapeutics, Inc. (NYSE MKT: AST) is a leading biotechnology company in the emerging field of regenerative medicine. The company’s proprietary, industry leading platforms are based on its pluripotent stem cell and dendritic cell immunotherapy technologies. Asterias is focused on developing therapies to treat conditions in several medical areas where there is high unmet medical need and inadequate available therapies. 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) has demonstrated promise in a Phase 2 study in acute myelogenous leukemia. AST-VAC2 (antigen-presenting allogeneic dendritic cells) represents a second generation, allogeneic approach to dendritic cell vaccines. Additional information about Asterias can be found at

Asterias is led by chief executive officer Stephen L. Cartt; president of R&D Jane Lebkowski, Ph.D.; chief operating officer and vice president Katherine Spink, Ph.D.; and chief medical officer Edward D. Wirth, III, M.D, Ph.D.

Asterias has significant financial support from the California Institute of Regenerative Medicine (CIRM).