MDRNA, Inc. Demonstrates The Potential For Greater Efficacy In Cancer With An UsiRNA Combination Approach

Main Category: Cancer / Oncology
Article Date: 16 Mar 2010 - 4:00 PDT


MDRNA, Inc. (NASDAQ: MRNA), a leading RNAi-based drug discovery and development company, announced enhanced efficacy for tumor reduction when two UsiRNA were combined within a single formulation. The UsiRNAs targeted two proteins survivin, a protein involved in cell division and inhibition of apoptosis, and PLK1 (Polo-like Kinase 1), a protein involved in cell mitosis and tumor progression. Both UsiRNA were encapsulated in the Company's proprietary DiLA2-based formulation, and delivered directly to the bladder (intravesical) in an orthotopic cancer model. At an equivalent total dose, tumor bioluminescence with a combination approach was significantly lower (~30%), when compared to a single UsiRNA. Additionally, the Company also announced another early collaborative effort with a major international pharmaceutical company bringing the total number of early collaborative efforts to three.

"Oncology drug products that simultaneously target multiple disease genes provide a greater potential therapeutic impact for a variety of cancers," stated Barry Polisky, PhD, Chief Scientific Officer at MDRNA, Inc. "These studies demonstrate the versatility of our DiLA2 platform to readily formulate two UsiRNAs into a single formulation and to efficiently deliver these UsiRNAs to target cells. We believe that a multi-target approach is likely to be required for treatment of many cancers and thus plays a central role in MDRNA's oncology pipeline."

The early collaborative effort with AstraZeneca Investment (China) Company, Ltd. will utilize the broad capabilities of MDRNA's proprietary discovery engine for RNAi therapeutics and its world-class research team. This latest collaboration will focus on MDRNA's proprietary delivery system for systemic delivery in hepatocellular carcinoma (HCC). Financial details of the collaboration were not disclosed.

"Today we announced an early collaborative effort with AstraZeneca's Innovation Center in China," stated Michael French, President and CEO at MDRNA, Inc. "This marks the third collaboration we have announced in the past nine months and we continue to believe we are well on our way to meeting our goal of completing two major collaborations by year-end."

About MDRNA Technology

UsiRNAs are MDRNA's proprietary constructs in which Unlocked Nucleobase Analogs (UNA) strategically replace ribonucleotides, and are distinct from standard siRNAs. Placement of UNA in the passenger strand specifically blocks the activity of this strand to function in RNAi, thus decreasing off-target activity. Placement of UNA within the guide strand confers greater specificity upon RNAi by inhibiting micro-RNA-like effects. UsiRNAs have been demonstrated to be highly active models of metabolic disorders and cancer, and function by RNAi to cleave their mRNA target.

DiLA2 delivery platform is MDRNA's proprietary platform for creating novel 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 to target tissue of interest. In vivo studies have demonstrated effective delivery in models of cancer, metabolic disorders, and other diseases. DiLA2 based delivery is well tolerated in systemic and local administration, and in rodent and non-human primates. MDRNA is also utilizing condensing peptides to form peptide-siRNA nanoparticles to further increase the delivery efficiency of its DiLA2 delivery systems.

Source
MDRNA, Inc.

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