May 04, 2010 08:30 ET

MDRNA, Inc. Announces Substantial Knockdown of a Liver Target via Systemic Delivery With Microgram Dosing Using Its Proprietary UsiRNA/DiLA2 Technology

Announces Early Collaborative Effort Bringing Total of On-Going Research Collaborations to Five

BOTHELL, WA--(Marketwire - May 4, 2010) - MDRNA, Inc. (NASDAQ: MRNA), a leading RNAi-based drug discovery and development company, today reported substantial advances in the activity of UsiRNA constructs delivered by its DiLA2 delivery technology resulting in 50% knockdown of Factor VII (FVII) protein at a 80 microgram per kilogram dose via systemic delivery. The Company also announced an additional early collaborative effort with a RNA-based therapeutics company focused on microRNA (miRNA)-directed oncology therapies. This adds to MDRNA's existing on-going collaborations and represents the fifth early effort announced in the last nine months. This was reported today by J. Michael French, President and CEO of MDRNA, at the 2010 BIO International Conference in Chicago, Illinois.

Effective delivery is a well recognized hurdle in the development of RNAi-based therapeutics, and MDRNA has been a leader in this area with its proprietary Di-Alkylated Amino Acid (DiLA2) delivery systems. The versatility of the DiLA2 system provides for a rapid and scientifically robust process for improving the delivery characteristics of its novel formulations to meet the specific requirements of a particular therapeutic application, i.e., administration via systemic versus local delivery or cellular uptake in liver hepatocytes versus lung epithelial cells. In addition, the team at MDRNA is constantly focused on the long term commercial needs of each DiLA2 formulation such as scale-up, manufacturing and cost-of-goods. In continuing to support on-going early collaborative efforts and the advancement of the DiLA2 delivery system, researchers at MDRNA identified novel DiLA2 formulations and process procedures that have improved the systemic delivery of UsiRNA at least five-fold. Using a mouse model and a UsiRNA targeting FVII mRNA in liver (hepatocytes), a single systemically delivered dose of 0.08 mg/kg (80 micrograms per kilogram) provided 50% inhibition of FVII protein activity. 

"These results represent rapid advancement in our DiLA2 technology and demonstrate the tremendous potential of this platform for systemic delivery," stated Barry Polisky, Ph.D., Chief Scientific Officer of MDRNA, Inc. "Given the modular nature of DiLA2, the extensive chemical space for this platform, and our robust research efforts into manufacturing procedures, we expect significant near-term advances in the development of our proprietary delivery formulations for RNAi-based therapeutics."

The new collaborative effort announced will utilize the broad capabilities of MDRNA's proprietary discovery engine for RNAi therapeutics and its world-class research team and will focus on MDRNA's proprietary DiLA2 technology for delivery of proprietary miRNAs in experimental oncology models. Other details of the collaborations were not disclosed.

"The level of interest in our technology and the work of our world-class research team is a clear demonstration of the progress MDRNA has made in the past year. With a total of five on-going early collaborative efforts, our team is gaining tremendous experience in working with large international pharmaceutical companies," said Michael French, President and CEO of MDRNA. "In addition, our team is receiving significant exposure within the larger RNAi research community which we see as a huge opportunity for the company. We expect to be able to transition at least two of these early collaborative efforts into major R&D collaborations this year."

About MDRNA's Technology

MDRNA has a broad intellectual property estate that encompasses four key RNAi technology platforms: siRNA constructs, chemistry, nucleic acid delivery, and gene targets. The MDRNA-owned siRNA constructs and chemistry include its proprietary UsiRNA construct, which is a duplex siRNA chemically modified with non-nucleotide acyclic monomers (UNAs), and is distinct from the standard siRNA construct used by others in the industry. UsiRNAs are fully recognized by the RNAi machinery and provide for potent RNAi activity while specific placement of UNAs in a duplex siRNA minimizes potential off-target effects by the guide strand and reduces undesired passenger strand activity. Furthermore, UsiRNAs escape the surveillance mechanisms associated with cytokine induction, and provide protection from nuclease degradation. 

The MDRNA delivery platforms include DiLA2 and nanoparticle forming peptides. DiLA2 is an MDRNA proprietary delivery platform of novel synthetic di-alklylated amino acid compounds used to make liposomal delivery formulations. The DiLA2 platform enables MDRNA to tailor the charge, linker and acyl chains of amino acids in order to configure liposomes for delivery to target tissues 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. The MDRNA peptide nanoparticle platform includes exclusively in-licensed and developed IP surrounding the use of peptides for nanoparticle formulations that increase cellular uptake and endosomal release of siRNAs. MDRNA is currently biopanning its patented phage display library to identify additional peptides for targeted delivery, cellular uptake and endosomal release of siRNA. 

MDRNA owns or controls 17 issued or allowed patents; and has 36 pending patent applications, 125 pending foreign patent applications and 7 PCT applications.

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, amino acid 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 liposomal-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; (v) the ability of MDRNA and/or a partner to develop and commercialize products that can compete favorably with those of competitors; and (vi) the failure of the stockholders of MDRNA to approve the merger with Cequent, the failure of either party to meet any of the other conditions to closing the merger, contractual restrictions on the conduct of our business included in the merger agreement, and any impact on our relationships with third parties as a result of the announcement of the proposed merger. 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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