ACT Files Investigational New Animal Drug (INAD) Application with FDA to Treat 10 Different Disease Indications Using Pluripo...
September 19 2013 - 9:48AM
Business Wire
Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in
the field of regenerative medicine, announced today that it has
filed an Investigational New Animal Drug (INAD) application with
the Food and Drug Administration (FDA) to test its proprietary
“off-the-shelf” mesenchymal stem cells (MSC) in a range of
different disease indications.
The studies will evaluate the safety and efficacy of ACT’s
pluripotent stem cell-derived MSCs in ten spontaneous disease
models in dogs, which are similar to various human inflammatory and
immune-mediated diseases. While these companion animal studies
represent research intended for ultimate licensure of veterinary
therapeutics, ACT anticipates that the trial results may be
relevant to its path to clinical trials in human patients and may
provide a more robust assessment of safety and therapeutic
endpoints than what can be obtained from inbred rodent models. The
INAD is directed to ten canine disorders corresponding to
hepatitis, glomerulonephritis, osteoarthritis, Crohn’s disease,
inflammatory bowel disease, spinal cord/disc disease,
meningoencephalitis, hemolytic anemia, pancreatitis and sepsis.
“This INAD filing extends our ongoing work with world experts in
veterinary and regenerative medicine ,” said Robert Lanza, M.D.,
chief scientific officer of ACT. “Preliminary studies in rodents
have shown that our hESC-derived MSCs possess unique biological and
therapeutic properties, and can dramatically impact a spectrum of
diseases ranging from lupus to multiple sclerosis. In addition to
veterinary applications, these large animal studies may help inform
and optimize subsequent human clinical trials that the company may
pursue. We believe the etiology and system-wide effects of
naturally-occurring diseases in large animals, such as dogs,
provide an excellent model of human conditions.”
ACT’s proprietary MSCs have been extensively characterized both
in vivo and in vitro, and their effects have been shown to be
superior in animals to those of their bone marrow (BM)-derived MSCs
counterparts. ACT’s proprietary MSCs are immunoprivileged and
therefore, may be used “off the shelf” in a wide range of clinical
indications. In addition to increased efficacy, hESC-MSCs can be
expanded to large numbers in vitro (approximately 30,000 times
greater yields per unit than adult BM sources). Since MSCs are
derived from an inexhaustible starting stem cell line, the cost and
regulatory burden of requiring many donors is thereby removed, as
is the case with the adult sources of MSCs currently in the clinic.
Having a single, donor-less source of pluripotent stem cells for
manufacturing MSCs also removes the batch-to-batch variability in
potency and the risk of communicable diseases that comes with
relying on bone marrow or other adult tissue donors in the current
MSC setting.
“The preclinical studies that Dr. Lanza and our team have
carried out can now be translated into companion animal studies
and, hopefully, human clinical studies in the not-too-distant
future,” said Gary Rabin, Chairman and CEO of ACT. “In addition to
advancing our corporate strategy to apply our cutting edge stem
cell technologies to bring human therapies to market, this step
into companion animals also elucidates new potential market
opportunities for our company. The veterinary market is a large and
exciting market in which we may have a significant opportunity to
apply our leadership in regenerative medicine. The development of
these therapies may permit veterinarians and pet owners to manage
pets’ medical needs safely and effectively, and could result in
longer and improved quality of life for pets.”
About Advanced Cell Technology, Inc.
Advanced Cell Technology, Inc. is a biotechnology company
applying cellular technology in the field of regenerative medicine.
For more information, visit http://www.advancedcell.com.
Forward-Looking Statements
Statements in this news release regarding future financial and
operating results, the relevance and applicability of clinical
trials in animals to studying the effect of products in humans,
future growth in animal and human research and development
programs, potential new applications of and expanded indications
covering our technology, the effects of donorless sources of stem
cells on potency and the risk of communicable diseases in the
manufacturing context, the existence and size of potential or
existing market opportunities for the company, the effect of the
company’s products on the medical needs and quality of life of
pets, and any other statements about the future expectations,
beliefs, goals, plans, or prospects expressed by management
constitute forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. Any statements
that are not statements of historical fact (including statements
containing the words “will,” “believes,” “plans,” “anticipates,”
“expects,” “estimates,” and similar expressions) should also be
considered to be forward-looking statements. There are a number of
important factors that could cause actual results or events to
differ materially from those indicated by such forward-looking
statements, including: limited operating history, need for and
limited sources of future capital, failures or delays in obtaining
regulatory approval of products, risks inherent in the development
and commercialization of potential products, reliance on new and
unproven technology in the development of products, protection of
our intellectual property, and economic conditions generally.
Additional information on potential factors that could affect our
results and other risks and uncertainties are detailed from time to
time in the company’s periodic reports, including the Quarterly
Report on Form 10-Q for the three months ended June 30, 2013.
Forward-looking statements are based on the beliefs, opinions, and
expectations of the company’s management at the time they are made,
and the company does not assume any obligation to update its
forward-looking statements if those beliefs, opinions,
expectations, or other circumstances should change. Forward-looking
statements are based on the beliefs, opinions, and expectations of
the company’s management at the time they are made, and the company
does not assume any obligation to update its forward-looking
statements if those beliefs, opinions, expectations, or other
circumstances should change. There can be no assurance that the
Company’s clinical trials will be successful.