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MDRNA Reports Its Proprietary RNAi-Based Compounds Are Well Tolerated and Demonstrate High Potency Against Multiple Targets in Preclinical Studies
Data Confirm 90% Knockdown of Factor VII at 1 mg/kg With Duration of Effect up to 28 Days
| Source: MDRNA, Inc.
BOTHELL, WA--(Marketwire - May 19, 2009) - MDRNA, Inc. (NASDAQ : MRNA ) announced today
positive in vivo efficacy data showing that its proprietary UsiRNA
constructs are highly potent and highly specific against ApoB and Factor
VII message. The data are being presented today by Michael V. Templin,
Ph.D., Vice President, Discovery Research and Pharmaceutical Development of
MDRNA, at the TIDES Oligonucleotide and Peptide® Technology and Product
Development Conference in Las Vegas, Nevada.
"Data from recent in vivo studies using our UsiRNAs targeting ApoB message
confirm that RNA interference is the mechanism of action by which knockdown
occurs, giving us high confidence that our UsiRNAs work by a
sequence-specific mechanism," stated Barry Polisky, Ph.D., Chief Scientific
Officer of MDRNA. "Data from the Factor VII studies indicate that a siRNA
in our lead formulation achieved greater than 90% knockdown at 1 mg/kg with
duration of effect of up to 28 days. This level of inhibition and duration
of effect meets or exceeds published data for Factor VII."
Dr. Polisky added, "Data from rodent studies with our DiLA2 delivery system
also continue to show encouraging results, with potency, specificity and
duration of effect data now achieved for four independent liver targets,
including ApoB, PCSK9, DGAT2 and Factor VII. The absence of histological
changes in the liver with doses up to 9 mg/kg in rodents confirms that our
DiLA2 liposomes are well tolerated."
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 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 Delivery Platform
DiLA2 is MDRNA's proprietary platform technology 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. In
addition, the platform 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.
MDRNA 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.