May 18 2015
Celsion Corporation, 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.