Demonstrates RNAi-Mediated Knockdown of Previously "Non-Druggable" Targets in Liver and Bladder CancersBOTHELL, WA, Oct 12, 2009 (MARKETWIRE via COMTEX) -- MDRNA, Inc. (NASDAQ: MRNA), a leading RNAi-based drug discovery and
development company, today presented new in vivo data demonstrating
continued progress in the advancement of the Company's oncology
program. J. Michael French, President and CEO, reported that MDRNA's
UsiRNAs, delivered by the Company's DiLA2 platform, down-regulated a
previously "non-druggable" target with subsequent reductions in tumor
growth in models of liver and bladder cancer via both systemic and
local delivery.
In a presentation to BioPartnering Europe in London today, Mr. French
said that MDRNA has demonstrated successful delivery of a UsiRNA
targeting survivin, a protein involved in mitotic progression and
inhibition of apoptosis, via intravenous administration using its
DiLA2 liposome formulation in two liver cancer models. Knockdown ( >
60%) of survivin mRNA in a rodent orthotopic model was noted as early
as 24 hours after a second (of six) dose and this was associated with
an approximate 65% decrease in tumor weight at study termination;
this decrease was comparable to tumor weight reduction with
Avastin(R) (bevacizumab)-treated mice as a positive control. A
similar level of survivin mRNA knockdown was noted in subcutaneously
implanted liver tumors following intravenous administration of the
UsiRNA/DiLA2 liposomes.
Data from an orthotopic bladder cancer model were also presented, in
which localized application (intravesical dosing) of the survivin
UsiRNA to a bladder tumor was performed using a DiLA2 liposome
formulation. Again, the UsiRNA was highly active in providing gene
silencing, demonstrating > 90% inhibition of survivin mRNA which was
dose-dependent and sustained over at least a three week period. At
study termination there was also a dose-dependent decrease in
bioluminescence of up to approximately 90% in UsiRNA-treated mice
which is a clear indication of reduced tumor growth.
Mr. French said, "We have always maintained that our RNAi discovery
engine can generate novel compounds with broad therapeutic
applicability. These data are a powerful indicator of the value and
strength of that drug discovery platform and represents a significant
step in the advancement of our product pipeline. Moreover, we now
have evidence illustrating the potential role of RNAi-based
therapeutics in down-regulating typically 'non-druggable' targets."
The presentation given by Mr. French is posted on the Company website
(www.mdrnainc.com).
About UsiRNAs
A UsiRNA is a duplex siRNA containing at least one Unlocked
Nucleobase Analog (UNA). In a UsiRNA, UNAs are non-nucleotide
monomers and synthesized much like RNA in the construction of a
double-stranded oligonucelotide for use as an RNAi-based therapeutic.
In the case of the UsiRNA, UNA is substituted for specific
nucleotides in both the guide and passenger strands. UsiRNAs are
fully recognized by the cellular RNAi machinery, as demonstrated by
their potent activity. MDRNA has also shown that substitution of UNA
for specific RNA increases stability to nucleases, substantially
reduces cytokine induction, and reduces passenger and guide
strand-mediated offtarget 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 diseases. 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.
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 through the development of
RNAi-based compounds and drug delivery technologies that together
provide superior therapeutic options for patients. Over the past
decade, we have developed substantial capabilities in molecular
biology, cellular biology, lipid chemistry, peptide chemistry,
pharmacology and bioinformatics, which we are applying to a wide
range of RNAi technologies and delivery approaches. These
capabilities plus the in-licensing of key RNAi-related intellectual
property have rapidly enabled us to become a leading RNAi-based
therapeutics company with a pre-clinical pipeline in oncology.
Through our capabilities, expertise and know-how, we are
incorporating multiple RNAi technologies as well as peptide- and
lipid-based delivery approaches into a single integrated drug
discovery platform that will be the engine for our clinical pipeline
as well as a versatile platform for establishing broad therapeutic
partnerships with biotechnology and pharmaceutical companies. We are
also investing in new technologies that we expect to lead to safer
and more effective RNAi-based therapeutics while aggressively
building upon our broad and extensive intellectual property estate.
By combining broad expertise in siRNA science with proven delivery
platforms and a strong IP position, MDRNA is well positioned as a
leading RNAi-based drug discovery and development company. Additional
information about MDRNA, Inc. is available at
http://www.mdrnainc.com.
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.
Contact information:
MDRNA, Inc.:
Peter Garcia
Chief Financial Officer
(425) 908-3603
pgarcia@mdrnainc.com
Westwicke Partners (Investors):
Stefan Loren, Ph.D.
(443) 213-0507
sloren@westwicke.com
John Woolford
(443) 213-0506
john.woolford@westwicke.com
McKinney|Chicago (Media):
Alan Zachary
(312) 944-6784 x 316
(708) 707-6834
azachary@mckinneychicago.com
SOURCE: MDRNA Inc.
mailto:pgarcia@mdrnainc.com
mailto:sloren@westwicke.com
mailto:john.woolford@westwicke.com
mailto:azachary@mckinneychicago.com