Data Confirm 90% Knockdown of Factor VII at 1 mg/kg With Duration of Effect up to 28 DaysBOTHELL, WA, May 19, 2009 (MARKETWIRE via COMTEX) -- 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(R) 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.
CONTACT
Matthew D. Haines
Senior Director, Investor Relations and Corporate Communications
(212) 209-3874
Email Contact
McKinney|Chicago (Media)
Alan Zachary
(312) 944-6784 x 316 or
(708) 707-6834
Email Contact
SOURCE: MDRNA, Inc.