-- Provides Direction for Late-Stage Development of AST-VAC1 in Acute
Myeloid Leukemia --
BioTime, Inc. (NYSE MKT:BTX), a clinical-stage regenerative medicine
company with a focus on pluripotent stem cell technology, today
announced that its subsidiary, Asterias Biotherapeutics (“Asterias”),
has successfully completed an End-of-Phase II meeting with the U.S. Food
and Drug Administration (FDA) for AST-VAC1, its investigational therapy
targeting acute myeloid leukemia (AML).
During the meeting, the FDA indicated general agreement with Asterias'
proposed development plan for registration of AST-VAC1 through a single
Phase 3 trial to support an accelerated development pathway and
Biologics License Application (BLA) filing. In this study, Asterias will
assess the impact of AST-VAC1 compared to placebo on the duration of
relapse-free-survival as the primary endpoint, and on overall survival
as the secondary endpoint in patients who have achieved complete
remission using standard therapies. The proposed trial will include AML
patients 60 years and older, along with younger individuals who are at
high risk for relapse and are not candidates for allogeneic bone marrow
transplantation. Pending positive results, this trial could be the basis
for accelerated approval of AST-VAC1. Asterias currently plans to submit
a request for a Special Protocol Assessment (SPA) to the FDA to confirm
the primary endpoint and other design elements of this pivotal Phase 3
trial.
About Acute Myeloid Leukemia
Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow.
AML is the most common type of acute leukemia and has the potential for
rapid progression, if untreated. In AML, the bone marrow produces an
excess number of immature cells known as blasts. In AML, these blasts
fail to mature into normal red and white blood cells. Instead, the
blasts proliferate and accumulate in the bone marrow and peripheral
blood, leading to deficiencies in normal mature cells. These
deficiencies, often referred to as cytopenias, can induce several
adverse effects including anemias and susceptibility to infections.
Current treatment strategies for AML are associated with significant
morbidities and in most instances, AML leads to death.
Approximately 20,500 new cases of AML are diagnosed in the U.S.
annually. AML remains a high unmet clinical need, particularly in
patients over the age of 60 years who face poor outcomes and have
limited therapeutic options. Treatment and prognosis in AML is strongly
influenced by a patient's age and tumor profile. Successful treatment
and survival of advanced age patients or those with a high risk profile
is very poor, with a four year relapse-free survival of 10% – 20% (Rolig
et al, 2011). Detailed characterizations of genetic abnormalities
associated with AML have elucidated their high number and relative
complexity, making development of targeted therapeutics to these
mutations very challenging. For this reason, broad immunotherapy
approaches such as autologous cell vaccines are particularly promising.
About AST-VAC1
AST-VAC1 is a cancer immunotherapy, consisting of autologous mature
antigen-presenting dendritic cells pulsed with a messenger RNA for the
protein component of human telomerase (hTERT) and a portion of a
lysosomal targeting signal (LAMP). hTERT is a common protein in tumor
cells and is responsible for the increased proliferative lifespan of
cancer cells. In AST-VAC1, the dendritic cells present telomerase to the
immune system to induce T cells to target and kill hTERT-expressing
tumor cells. The LAMP signal allows AST-VAC1 to stimulate both cytotoxic
and helper T cell responses to telomerase, critical elements to induce
and maintain immune responses that kill tumor cells. In June 2015,
Asterias announced long-term follow-up results from a Phase 2 clinical
trial in AML patients receiving AST-VAC1. In this trial, twenty-one
patients received at least three injections of AST-VAC1, including 19
patients in complete remission (CR). AST-VAC1 showed a strong safety
profile in this trial and in a previous study in prostate cancer
patients. Eleven of the 19 patients (58 percent) in the trial remain in
CR with a median duration of follow-up of 52 months from first
vaccination. Four of the seven patients who were at least 60 years old
at the time of immunotherapy with AST-VAC1 remained relapse-free 52 to
59 months from first vaccination.
Because of the widespread expression of telomerase in the majority of
cancers, AST-VAC1 is a platform immunotherapeutic that could be used
alone or in conjunction with other therapeutics such as immune
checkpoint inhibitors to target immune-based destruction of tumors.
About Accelerated Approval
The FDA instituted its Accelerated Approval Program to allow for earlier
approval of drugs that treat serious conditions, and that fill an unmet
medical need based on a surrogate endpoint. A surrogate endpoint is a
marker, such as a laboratory measurement, radiographic image, physical
sign or other measure that is thought to predict clinical benefit, but
is not itself a measure of clinical benefit. The use of a surrogate
endpoint can considerably shorten the time required prior to receiving
FDA approval.
Drug companies are still required to conduct studies to confirm the
anticipated clinical benefit. These studies are known as phase 4
confirmatory trials. If the confirmatory trial shows that the drug
actually provides a clinical benefit, then the FDA grants traditional
approval for the drug. If the confirmatory trial does not show that the
drug provides clinical benefit, FDA has regulatory procedures in place
that could lead to removing the drug from the market.
About Asterias Biotherapeutics
Asterias Biotherapeutics, Inc. is a leading biotechnology company in the
emerging field of regenerative medicine. The company’s proprietary
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 myeloid 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 www.asteriasbiotherapeutics.com.
About BioTime
BioTime, Inc., a pioneer in regenerative medicine, is a clinical-stage
biotechnology company. BioTime and its subsidiaries are leveraging their
industry-leading experience in pluripotent stem cell technology and a
broad intellectual property portfolio to facilitate the development and
use of cell-based therapies and gene marker-based molecular diagnostics
for major diseases and degenerative conditions for which there presently
are no cures. The lead clinical programs of BioTime and its subsidiaries
include OpRegen®, currently in a Phase I/IIa trial for
the treatment of the dry form of age-related macular degeneration;
AST-OPC1, currently in a Phase I/IIa trial for spinal cord injuries; Renevia™,
currently in a pivotal trial in Europe as an injectable matrix for the
engraftment of transplanted cells to treat HIV-related lipoatrophy; and
cancer diagnostics, nearing the completion of initial clinical studies
for the detection of lung, bladder, and breast cancers. AST-VAC1 has
demonstrated promise in a Phase 2 study in acute myeloid leukemia.
AST-VAC2, a cancer vaccine, is in the pre-clinical trial stage.
BioTime’s subsidiaries include the publicly traded Asterias
Biotherapeutics, Inc, developing pluripotent stem cell-based therapies
in neurology and oncology, including AST-OPC1 and AST-VAC2; Cell Cure
Neurosciences Ltd., developing stem cell-based therapies for retinal and
neurological disorders, including OpRegen®;
publicly traded OncoCyte Corporation, developing cancer diagnostics;
LifeMap Sciences, Inc., developing and marketing an integrated online
database resource for biomedical and stem cell research; LifeMap
Solutions, Inc., a subsidiary of LifeMap Sciences, developing mobile
health (mHealth) products; OrthoCyte Corporation, developing therapies
to treat orthopedic disorders, diseases, and injuries; ReCyte
Therapeutics, Inc., developing therapies to treat a variety of
cardiovascular and related ischemic disorders; and Ascendance
Biotechnology, Inc. which manufactures and sells proprietary products
and services that assay new drug candidates for potential toxicity,
including HepatoPac® and HepatoMune®,
and other products for use as research tools.
BioTime common stock is traded on the NYSE MKT and TASE under the symbol
BTX. For more information, please visit www.biotimeinc.com or
connect with the company on Twitter, LinkedIn, Facebook, YouTube,
and Google+.
Forward-Looking Statements
Statements pertaining to future financial and/or operating results,
future growth in research, technology, clinical development, and
potential opportunities for BioTime and its subsidiaries, 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 or diagnostic tests, 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 business of BioTime and its
subsidiaries, particularly those mentioned in the cautionary statements
found in Securities and Exchange Commission filings of BioTime and its
subsidiaries Asterias Biotherapeutics, Inc. and OncoCyte Corporation.
BioTime disclaims any intent or obligation to update these
forward-looking statements.
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