January 13, 2010 07:30 ET

MDRNA Reports Potent Anti-Tumor Activity Against Multiple Targets in Liver and Bladder Cancer

Announces Additional Early Collaborative Effort With Major International Pharmaceutical Company

BOTHELL, WA--(Marketwire - January 13, 2010) - MDRNA, Inc. (NASDAQ: MRNA), a leading RNAi-based drug discovery and development company, reported today in vivo data for bladder and liver cancer demonstrating further advancement of the Company's oncology programs. The Company reported a reduction in tumor growth in both liver and bladder cancers by targeting genes key to tumor progression, via both systemic and local delivery with the Company's proprietary UsiRNAs delivered by its novel DiLA2 platform. In addition, MDRNA disclosed the establishment of an early collaborative effort with a major international pharmaceutical company, its second such effort.

In a presentation at the 2010 OneMedForum in San Francisco today, Mr. J. Michael French, President & CEO of MDRNA, stated that the Company has demonstrated potent anti-tumor activity with a UsiRNA targeting PLK1 (Polo-like Kinase 1), a protein involved in cell mitosis and tumor progression. Data from local (intravesical) application of a PLK1 UsiRNA in a DiLA2 liposome formulation in a mouse orthotopic bladder cancer model demonstrated a PLK1 UsiRNA dose-dependent decrease in bioluminescence in a mouse model of orthotopic bladder cancer, with greater than 90% reduction at a dose of 1 mg/kg. Decreased bioluminescence is generally considered to be a clear indication of reduced tumor growth. This study was conducted in conjunction with the Company's collaborators at the Vancouver Prostate Centre.

The PLK1 UsiRNA has also demonstrated activity in models of orthotopic liver cancer and subcutaneous liver tumors, in which the UsiRNA was delivered by systemic administration of a DiLA2 formulation. MRNA-046, a DiLA2-formulated survivin UsiRNA, has been previously reported to have potent RNAi and anti-tumor activity in bladder and liver cancer. Effective delivery (per RNAi activity and tolerability) of a UsiRNA with DiLA2 liposomes has also been previously reported by MDRNA in non-human primates.

"The data we're reporting regarding the potent activity of a PLK1 UsiRNA further validates the strength and speed at which our proprietary RNAi-based drug discovery platform can generate novel compounds," stated Mr. French. "We have demonstrated that UsiRNAs against multiple non-cancer and cancer targets are highly active in rodents and non-human primates, and can be delivered with DiLA2 liposomes by intravenous or local routes. In our oncology programs, we have now demonstrated significant knockdown of both survivin and PLK1, which are high profile targets implicated in apoptosis and proliferation, respectively. Both of these targets have been difficult to inhibit by traditional small molecule and monoclonal antibody approaches. The ability for a single RNAi drug product to target both an apoptotic and proliferation pathway will provide for a potentially more efficacious therapeutic compound against multiple cancers. Taken as a whole, MDRNA's UsiRNA and delivery technologies represent a unique and proprietary approach to the use of RNAi-based therapeutics to treat human diseases."

The additional early collaborative effort with a major international pharmaceutical company will utilize the broad capabilities of MDRNA's proprietary discovery engine for RNAi therapeutics and its world-class research team. The collaboration will focus on in vivo delivery of siRNAs using MDRNA's DILA2 liposome platform. As part of the collaboration, research teams at MDRNA and the pharmaceutical company will examine safety and efficacy of systemic delivery of siRNA in animal models. Financial details of the collaboration were not disclosed.

"Today we also disclosed the establishment of a second early collaborative effort today," continued Mr. French. "Between the collaboration we disclosed in September 2009 and this new effort, we believe we are on track to complete a major R&D collaboration well before our goal of mid 2010."

The MDRNA presentation given at the OneMedForum will be webcast live today, January 13 at 11:15am PST. Access to the MDRNA audio webcast is available live via the following link: and will be archived for replay for 3 months.

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

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.

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