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MDRNA, Inc. Reports Effective Local Delivery of a UsiRNA in an In Vivo Model of Bladder Cancer
Improved Performance of the DiLA2 siRNA Delivery Platform With Proprietary Peptide Nanoparticles
| Source: MDRNA, Inc.
BOTHELL, WA--(Marketwire - June 8, 2009) - MDRNA, Inc. (NASDAQ : MRNA ) announced today in
vivo data from a bladder cancer model demonstrating effective localized
delivery of a UsiRNA to a solid tumor, thus further expanding the delivery
capabilities of the DiLA2 Platform. In addition, the Company reported in
vivo data demonstrating the ability of its proprietary peptide-based
nanoparticle technology to significantly improve siRNA delivery efficiency.
The integration of the peptide nanoparticle technology with the DiLA2
Platform resulted in 85% knockdown of ApoB messenger RNA while decreasing
the overall amount of the DiLA2 delivery vehicle by 45%. The data are being
presented today at the RNA Interference Summit in San Francisco by Roger
Adami, Ph.D., Associate Director, Molecular Pharmaceutics, MDRNA, Inc.
Dr. Adami will also present data demonstrating knockdown of additional
hepatocyte targets in rodent models. The DiLA2 liposomes showed 90%
knockdown of DGAT2 in mice following a single 2 mg/kg administration and a
75% knockdown of PCSK9 with a single 2 mg/kg dose. This highly efficient
delivery to hepatocytes provides the basis for MDRNA's development pipeline
in oncology.
"The data reported today demonstrate the breadth and versatility of the
DiLA2 delivery system," stated Barry Polisky, Ph.D., Chief Scientific
Officer. "We believe that the DiLA2 Platform will enable effective
therapeutic applications of siRNAs in oncology and various other disease
indications. Additionally, peptide-siRNA particles combined with the DiLA2
Platform will provide improved delivery efficiency of RNAi-based
therapeutics. During 2009, we intend to expand the capabilities of the
DiLA2 delivery system to target multiple oncology indications beyond our
initial internal program in liver cancer."
About UsiRNAs
UsiRNAs are duplex siRNAs that are modified with non-nucleotide acyclic
monomers, termed unlocked nucleobase analogs (UNA), in which the bond
between two adjacent carbon atoms of ribose is removed. UsiRNAs are fully
recognized by the RNAi machinery and provide for potent RNAi activity.
Placement of UNA within a UsiRNA minimizes the potential for off-target
effects by the guide strand as well as undesired activity of the passenger
strand. Further, the change in sugar structure renders this unlocked
nucleobase analog conformationally flexible. The flexibility of the monomer
escapes the body's surveillance mechanisms associated with cytokine
induction, as well as providing protection from nuclease degradation.
About the DiLA2 Platform
The DiLA2 Platform is MDRNA's proprietary platform for creating novel
liposomal delivery systems from amino acids. The platform enables MDRNA to
tailor the charge, linker and acyl chains of amino acids in order to
optimize the liposome for delivery to the target tissue of interest and is
designed to permit attachment of various peptides and other targeting
molecules to improve a variety of delivery characteristics. In addition,
MDRNA is utilizing peptides for nanoparticle formulations to increase
cellular uptake and endosomal release.
About MDRNA, Inc.
MDRNA is a biotechnology company focused on the development and
commercialization of therapeutic products based on RNA interference (RNAi).
Our goal is to improve human health by combining novel RNAi-based compounds
and proprietary peptide- and liposomal-based drug delivery technologies to
provide superior therapeutic options. Our multi-disciplinary portfolio of
capabilities includes molecular biology, cellular biology, formulation
expertise, peptide and alkylated amino acid chemistry, pharmacology,
toxicology and bioinformatics. We are applying this expertise to a single,
integrated drug discovery platform that will be the engine for our clinical
pipeline and a versatile platform for establishing broad therapeutic
partnerships. We are also building on new technologies, such as UsiRNAs
that incorporate the non-nucleotide moiety Unlocked Nucleobase Analog (UNA)
within the siRNA molecule, that we expect to lead to safer and more
effective RNAi-based therapeutics. By combining broad expertise in siRNA
science with proven delivery platforms and a strong and growing IP
position, MDRNA is well positioned as a leading RNAi therapeutics company
and value-added collaborator for our research partners. Additional
information about MDRNA, Inc. is available at http://www.mdrnainc.com.
Forward-Looking Statement
Statements made in this news release may be forward-looking statements
within the meaning of Federal Securities laws that are subject to certain
risks and uncertainties and involve factors that may cause actual results
to differ materially from those projected or suggested. Factors that could
cause actual results to differ materially from those in forward-looking
statements include, but are not limited to: (i) the ability of MDRNA to
obtain additional funding; (ii) the ability of MDRNA to attract and/or
maintain manufacturing, research, development and commercialization
partners; (iii) the ability of MDRNA and/or a partner to successfully
complete product research and development, including preclinical and
clinical studies and commercialization; (iv) the ability of MDRNA and/or a
partner to obtain required governmental approvals; and (v) the ability of
MDRNA and/or a partner to develop and commercialize products that can
compete favorably with those of competitors. Additional factors that could
cause actual results to differ materially from those projected or suggested
in any forward-looking statements are contained in MDRNA's most recent
periodic reports on Form 10-K and Form 10-Q that are filed with the
Securities and Exchange Commission. MDRNA assumes no obligation to update
and supplement forward-looking statements because of subsequent events.