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MDRNA, Inc. Reports Positive In Vivo Data Demonstrating That Its UsiRNAs Are Highly Potent Against Metabolic and Oncology Targets
UsiRNAs Work Via the RNAi Mechanism of Action but With Minimal Off-Target Activity
| Source: MDRNA, Inc.
BOTHELL, WA--(Marketwire - August 3, 2009) - MDRNA, Inc. (NASDAQ : MRNA ) announced today
positive data on its proprietary UsiRNA technology, demonstrating that its
UsiRNAs are highly potent against metabolic disease and cancer targets in
rodent models. Further, the data establish that the knockdown of these
targets is achieved via an RNAi-mediated process. Of particular importance
is that the data demonstrate that strategic placement of UNA (unlocked
nucleobase analogs) results in the ability to manipulate, either increasing
or decreasing, strand-specific activity thus minimizing off-target activity
and providing the ability to impart improved drug-like characteristics to
the UsiRNAs. The data are being presented today by Michael V. Templin,
Ph.D., Vice President, Discovery Research and Pharmaceutical Development of
MDRNA, at the IBC Drug Discovery & Development Week, Oligonucleotide
Therapeutics: From Concept to Implementation, in Boston, Massachusetts.
"UNA-modified siRNAs are novel and proprietary constructs that have high
potency in target mRNA reduction. In addition, we have recently determined
that strategic placement of UNA in the siRNA reduces micro-RNA-like
off-target activity. This observation is distinct from, and in addition to,
our previous work in which we have demonstrated that UNA residues have a
dramatic effect on reduction of cytokine induction by siRNA," stated Barry
Polisky, Ph.D., Chief Scientific Officer of MDRNA. "We are encouraged by
these significant results that demonstrate the versatility of UNA and
UsiRNAs for improving the safety and specificity of RNAi, and reinforce our
confidence in our UsiRNA platform."
"Repeated positive results from multiple in vivo studies in rodents using
our UsiRNAs and DiLA2 delivery platform has led us to the initiation of
preclinical safety and efficacy studies in non-human primates," stated J.
Michael French, President and Chief Executive Officer. "We are one of a
handful of companies to have initiated non-human primate studies with a
liposomal-based delivery system and we look forward to reporting positive
data this quarter."
About Unlocked Nucleobase Analogs and UsiRNAs
Unlocked Nucleobase Analogs (UNA) are substitutes for nucleotides that make
up conventional siRNAs. UNAs have an open structure in the place of the
ribose portion, making them more flexible than common nucleotides. UsiRNAs
are a duplex siRNAs containing at least one UNA. UsiRNAs are fully
recognized by the cellular RNAi machinery, as demonstrated by their potent
activity. MDRNA has also shown that inclusion of UNA increases stability to
nucleases, substantially reduces cytokine induction, and reduces passenger
and guide strand-mediated off-target effects. The high potency, and
improved drug-like properties, associated with UsiRNAs provide the
potential to greatly enhance RNAi-based therapeutics.
About the DiLA2 Delivery Platform
The DiLA2 Delivery Platform is MDRNA's proprietary platform for creating
novel liposomal delivery systems based on di-alkylated amino acids (DiLA2).
The DiLA2 Platform enables MDRNA to tailor the charge, linker length, and
acyl chain characteristics to improve delivery of the liposomes to target
tissue of interest. In vivo studies have demonstrated effective delivery in
models of metabolic disease, cancer, and other targets. DiLA2-based
liposomes are well tolerated for repeat dose, and systemic and local
administration. MDRNA is also utilizing condensing peptides to form
peptide-siRNA nanoparticles to further increase the delivery efficiency of
its DiLA2 delivery systems. In addition, the platform is designed to permit
attachment of peptides and other targeting molecules for delivery to a
variety of tissues, and thus provide for a diverse therapeutic portfolio.
MDRNA Forward-Looking Statements
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.