Full Press Release Details
Subsidiary, Asterias Biotherapeutics, and Cancer Research UK and Cancer
Research Technology Partner for Clinical Trial of Immunotherapy Vaccine
ALAMEDA, Calif.--(BUSINESS WIRE)--September 11, 2014--BioTime, Inc.
(NYSE MKT: BTX) announced that its subsidiary Asterias Biotherapeutics,
Inc. (OTCBB: ASTY) has reached an agreement with Cancer Research
UK and Cancer Research Technology (CRT), the charity's development and
commercialization arm, to conduct a clinical trial of Asterias'
novel immunotherapy treatment AST-VAC2 in subjects with non-small cell
AST-VAC2 is a non-patient specific (allogeneic) cancer vaccine designed
to stimulate patients' immune systems to attack telomerase, a protein
that is expressed in over 95 percent of cancers but is rarely expressed
in normal adult cells.
The vaccine was developed following successful early phase clinical
trials of a similar, patient specific (autologous) Asterias vaccine,
called AST-VAC1, which was derived from patients' blood cells and tested
in prostate cancer and acute myeloid leukemia.
Unlike AST-VAC1, and other autologous (patient specific) vaccines that
are developed from a patient's own cells, AST-VAC2 is derived from human
embryonic stem cells (hESCs), meaning it can be produced on a large
scale and stored ready for use, rather than having to produce a specific
version of the drug for each patient.
The trial of AST-VAC2 will evaluate the safety and toxicity of the
vaccine, feasibility, stimulation of patient immune responses to
telomerase and AST-VAC2, and clinical outcome after AST-VAC2
administration in patients with resected early-stage lung cancer and in
patients with advanced forms of the disease.
Pedro Lichtinger, Asterias' chief executive officer, said: "The Asterias
collaboration with Cancer Research UK's Drug Development Office and CRT
represents a major step in advancing our proprietary dendritic cell
platform for the potential benefit of patients.
"AST-VAC2 is based on a specific mode of action that is complementary
and potentially synergistic to other immune therapies. We are delighted
to partner with Cancer Research UK to advance this important platform
through Phase 1/2 clinical trials. Cancer Research UK's Drug Development
Office has the global recognition of having the quality, capability and
track record of successfully advancing development programs. We are
excited about the possibility of favorably impacting the lives of
patients across multiple cancers and are proud to be working with Cancer
Under the agreement, Asterias will complete development of the
manufacturing process for AST-VAC2. Cancer Research UK will then produce
the vaccine and conduct the phase 1/2 clinical trial in the United
Kingdom. On completion of the clinical trial, Asterias will have an
exclusive first option to acquire a license to the data from the trial
on pre-agreed terms including an upfront payment, milestones and
royalties on sales of products. If Asterias declines this option, CRT
will then have an option to obtain a license to Asterias' intellectual
property to continue the development and commercialization of AST-VAC2
and related products in exchange for a revenue share to Asterias of
development and partnering proceeds.
Dr. Jane Lebkowski, president of research and development at Asterias,
said: "The use of human embryonic stem cells to derive allogeneic
dendritic cells for cancer immunotherapy has the potential to
dramatically improve the scalability, consistency, and feasibility of
cellular cancer vaccines. We believe this collaboration will enable the
acceleration of clinical studies of AST-VAC2 and the collection of
important proof-of-concept data for the entire human embryonic stem
cell-derived dendritic cell immunotherapy platform."
Lung cancer is the leading cause of cancer death in the United States.
More than 150,000 people in the US die from lung cancer annually. In the
US five year survival rates remain only 25-50 percent for early stage
disease and less than 20 percent for advanced disease. NSCLC is much
more common than other types of lung cancer, and accounts for 85 percent
of all lung cancer cases.
Lung cancer is the leading cause of cancer death in the UK, with more
than 35,000 people dying from the disease each year. It has one of the
lowest survival rates of any cancer and less than 10 per cent of people
survive more than five years.
About Cancer Immunotherapy and Cancer Vaccines
Vaccines have the potential to act synergistically with immune
checkpoint inhibitors by using the checkpoint inhibitor to activate, and
the vaccine to direct, the immune response to cancer cells. One
promising cancer vaccine approach uses dendritic cells, which normally
serve this training function - known as antigen presentation - in the
human immune system. However, first generation dendritic cell vaccines,
such as Dendreon's Provenge (sipuleucel-T) and Asterias' AST-VAC1, must
be manufactured on a patient-by-patient (autologous) basis, which can
result in higher costs of manufacture, variability in product
performance, and delayed availability of such autologous products.
Asterias' AST-VAC2 product represents a scalable platform for low cost,
consistent manufacturing of a dendritic cell vaccine with on demand
AST-VAC2 is an immunotherapeutic product candidate consisting of human
embryonic stem cell derived- mature dendritic cells (hESC-DCs) that are
engineered to express telomerase. AST-VAC2 is designed as an
"off-the-shelf" vaccine platform to stimulate an immune response against
tumor cells expressing telomerase. Telomerase is expressed in most human
cancers, and plays an important role in the prolonged proliferative
lifespan of cancer cells. In contrast, telomerase expression is rare and
transient in most normal adult tissues, making telomerase an attractive
target for cancer immunotherapy.
Previous studies of a patient specific (autologous), peripheral
blood-derived, dendritic cell vaccine targeting telomerase (AST-VAC1) in
prostate cancer and acute myelogenous leukemia (AML) provided promising
data supporting the safety and feasibility of dendritic cell-based
telomerase immunotherapy, and showed telomerase-specific immune
responses in 55% of AML and 95% of prostate cancer patients.
Additionally, in the AST-VAC1 prostate cancer trials, reductions in PSA
velocity and circulating tumor cells which correlated with patient
immune responses were observed. The use of human embryonic stem cells,
as opposed to patient blood, as the starting material for production of
AST-VAC2 provides a scalable system for the production of a large number