LAWRENCEVILLE, N.J.,
May 21, 2015 /PRNewswire/
-- Celsion Corporation (NASDAQ: CLSN), a fully-integrated
oncology company focused on the development of a portfolio of
innovative cancer treatments, including directed chemotherapies,
immunotherapies and RNA- or DNA-based therapies for the treatment
of cancer and other difficult-to-treat diseases, today reported
data from a preclinical study confirming that the Company's
TheraSilence™ technology platform can safely and effectively
deliver RNA to the lungs in non-human primates. TheraSilence™
is designed to enable the delivery of synthetically-generated mRNA,
inhibitory RNA (RNAi) such as small inhibitory RNAs (siRNAs),
microRNAs, microRNA mimics, and related molecules that can regulate
protein expression at the transcript level by exploiting endogenous
cell mechanisms.
"The findings from this study are consistent with the data
observed in earlier preclinical studies and underscore the
differentiating and widespread potential of our TheraSilence™ RNA
delivery platform," stated Dr. Khursheed
Anwer, Celsion's Executive Vice President and Chief
Scientific Officer. "Development for RNA therapeutics has been
significantly limited due to the delivery challenges associated
with nucleic acid-based therapies, with development efforts in the
space focused primarily on diseases of the liver. Our
chemically-flexible approach offers the opportunity to
significantly expand the development of RNA therapies to include
major unmet medical needs affecting the lung, such as lung cancer,
asthma and pulmonary hypertension."
The study was designed to evaluate the expression profile and
safety of RNA formulated with Celsion's TheraSilence™ delivery
system in non-human primates. Three primates were dosed in
the study, receiving either TheraSilence-formulated RNA
intravenously at one of two doses, or intravenous RNA formulated
with a liver-directed formulation used as a positive control.
Following the treatment, an analysis for safety and RNA expression
was performed.
In the study, TheraSilence-formulated signaling RNA resulted in
preferential expression in the lungs, with expression in the liver
at less than 15% of expression levels observed in the lungs.
Expression levels in tissues other than the lung, spleen and liver
were very low or at background levels. The
TheraSilence-formulated RNA was well-tolerated at both dose levels
as determined by safety analysis including complete blood cell
count, blood chemistry, histopathology, interferon response and
compliment activation. A liver-directed delivery system, used as a
positive control for the study, yielded preferential expression in
liver and spleen, with only background expression levels observed
in the lung.
These data build on previous preclinical studies indicating the
preferential delivery of RNA to the lung using the TheraSilence™
RNA delivery system. A murine study evidenced that the delivery of
TheraSilence-formulated siRNA molecules targeting anti-vascular
endothelial receptor 2 (VEGF2), a protein that is critical for the
growth of new blood vessels in tumors, significantly inhibit lung
tumor growth. Additionally, delivery of a TheraSilence-formulated
anti-micro RNA molecule into rats with experimentally induced
pulmonary arterial hypertension appeared to normalize vascular
remodeling that occurs in the lung and help restore cardiac
function that is compromised as a result of the disease.
"The data highlights the great potential of our TheraSilence™
technology platform to provide unique treatment options for lung
diseases that are not addressable by conventional drugs," said
Michael H. Tardugno, the Company's
Chairman, President and Chief Executive
Officer. "TheraSilence™ has significant potential in a wide
variety of indications, including those that fall outside our core
focus of oncology. Our strategy is to seek to maximize the
value of this platform in the near-term by pursuing collaborations
and licensing agreements, while focusing internal development
efforts on our two clinical stage candidates, ThermoDox™ and GEN-1,
our DNA-based immunotherapy for the localized treatment of ovarian
and brain cancers."
About TheraSilence™
TheraSilence™ is a technology platform for the delivery of
synthetically-generated messenger RNA, inhibitory RNA (RNAi) such
as small inhibitory RNAs (siRNAs), microRNAs, microRNA mimics, and
related molecules that can regulate protein expression at the
transcript level by exploiting endogenous cell mechanisms.
Inhibitory RNA based therapies have potential for targeting
virtually any disease related gene with a high degree of
specificity and thus eliminating so called "non-drugable" target
classes. The technology addresses the primary obstacle to nucleic
acid-based therapeutics, which is the efficient delivery to target
cells. Celsion's proprietary, novel structures tare able to
interact with the RNAi molecules forming protective nanoparticles
that can be readily taken up into cells. In addition, these systems
are chemically flexible and amenable to attachment of
tissue-targeted ligands, in vivo stabilizing agents and other
functional moieties which can tailor a formulation for a particular
application and delivery modality. These features can provide high
specificity for RNAi delivery to select tissue, enhance stability
and reduce in vivo toxicity.
About Celsion Corporation
Celsion is a fully-integrated oncology company focused on
developing a portfolio of innovative cancer treatments, including
directed chemotherapies, immunotherapies and RNA- or DNA-based
therapies. The Company's lead program is ThermoDox®, a proprietary
heat-activated liposomal encapsulation of doxorubicin, currently in
Phase III development for the treatment of primary liver cancer and
in Phase II development for the treatment of recurrent chest wall
breast cancer. The pipeline also includes GEN-1, a DNA-based
immunotherapy for the localized treatment of ovarian and brain
cancers. Celsion has three platform technologies for the
development of novel nucleic acid-based immunotherapies and other
anti-cancer DNA or RNA therapies, including TheraPlas™,
TheraSilence™ and RAST ™. For more information on Celsion,
visit our website: http://www.celsion.com.
Celsion wishes to inform readers that forward-looking
statements in this release are made pursuant to the "safe harbor"
provisions of the Private Securities Litigation Reform Act of
1995. Readers are cautioned that such forward-looking
statements involve risks and uncertainties including, without
limitation, unforeseen changes in the course of research and
development activities and in clinical trials; the uncertainties of
and difficulties in analyzing pre-clinical data, particularly in
small groups that are not statistically significant; we conducted a
small non-human primate study, which is not necessarily indicative
of any potential clinical results in human beings or any potential
results in a larger primate study; FDA and regulatory uncertainties
and risks; the significant expense, time, and risk of failure of
conducting clinical trials; the need for Celsion to evaluate its
future development plans; possible acquisitions or licenses of
other technologies, assets or businesses or the possible failure to
make such acquisitions or licenses; possible actions by customers,
suppliers, competitors, regulatory authorities; and other risks
detailed from time to time in the Celsion's periodic reports and
prospectuses filed with the Securities and Exchange
Commission. Celsion assumes no obligation to update or
supplement forward-looking statements that become untrue because of
subsequent events, new information or otherwise.
Celsion Investor Contact
Jeffrey W. Church
Sr. Vice President and CFO
609-482-2455
jchurch@celsion.com
To view the original version on PR Newswire,
visit:http://www.prnewswire.com/news-releases/celsion-announces-potent-durable-preclinical-lung-expression-data-for-its-novel-therasilence-rna-program-300086910.html
SOURCE Celsion Corporation