LAWRENCEVILLE, N.J.,
May 15, 2015 /PRNewswire/ -- Celsion
Corporation (NASDAQ:CLSN), a fully-integrated oncology company
focused on the development of a portfolio of innovative cancer
treatments, today reported the publication of an abstract in the
prestigious Journal of Controlled Release summarizing
findings from a preclinical study confirming effective delivery of
RNA to lung cells. In the study, the Company's TheraSilence™
technology platform safely and effectively delivered an inhibitor
of microRNA-145 (miR-145) in a well-established model of severe
occlusive pulmonary arterial hypertension (PAH). Increased
expression of miR-145 has been linked to the development and
progression of PAH in humans. Treatment was associated with
significant delivery of miR-145 inhibitor in the lung, inhibition
of miR-145 levels and reversal of the pulmonary hypertension
associated with the advanced stages of the disease leading to a
normalization of cardiovascular function. This preclinical
program was conducted in collaboration with Dr. William Gerthoffer at the University of South
Alabama.
"There is a clear unmet medical need for novel agents that can
treat PAH," said Dr. William
Gerthoffer, Professor and Chair, Biochemistry &
Molecular Biology, University of South
Alabama. "The lung directed nature of TheraSilence™ platform
has provided us with a delivery vector to approach this devastating
disease. We have been very encouraged by the positive results seen
in this study, which shows that we may be able to significantly
improve pulmonary function by inhibiting miR-145 and believe that
this approach has tremendous potential as a novel therapeutic."
Celsion's TheraSilence™ RNA delivery platform is designed to
enable the preferential delivery to the lungs of
synthetically-generated mRNA, inhibitory RNA (RNAi) such as small
inhibitory RNAs (siRNAs), microRNAs, microRNA mimics, microRNA
inhibitors and related molecules that can regulate protein
expression at the transcript level by exploiting endogenous cell
mechanisms. Delivery of a TheraSilence-formulated
anti-miR-145 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
findings from this study are consistent with the data observed in
earlier preclinical studies. 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 data highlights the differentiating and widespread
potential of the TheraSilence™ RNA delivery platform to enable lung
specific delivery of RNA and related molecules and 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."
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,
microRNA inhibitors 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 safe and efficient delivery
to target cells. Celsion's proprietary, novel structures are 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 PAH
Pulmonary arterial hypertension (PAH) is characterized by high
blood pressure in the arteries of the lungs. Progressive,
symptomatic, and ultimately fatal disorder results from a
proliferation of the smooth muscle cells that line the arteries in
the lung. As the arteries narrow the arterial pressure is
increased and blood flow from the heart to the lungs is
decreased. To compensate, the heart must pump harder,
causing the walls on the right side of the heart to thicken and
weaken. These changes make it difficult for the heart to push
blood through the arteries and into the lungs. Ultimately the heart
becomes so weak that it cannot pump enough blood to the lungs which
can ultimately result in heart failure. Currently, there are
no effective treatments for PAH, only treatments that improve the
management of symptoms.
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
Celsion Media Contacts
Harriet Shelare
Director, Communications
hshelare@celsion.com
Bill Berry
Berry & Company
212-253-8881
bberry@berrypr.com
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SOURCE Celsion Corporation