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Exiqon Announces Achievement of Primary Milestones in RNAi Project for Tacere Therapeutics

Exiqon A/S (NASDAQ OMX Copenhagen: "EXQ") today announced the successful completion of several key milestones in a custom-designed RNAi project for Tacere Therapeutics.

San Jose and Copenhagen (PRWEB) September 18, 2009 -- Exiqon A/S (NASDAQ OMX Copenhagen: "EXQ") today announced the successful completion of several key milestones in a custom-designed RNAi project for Tacere Therapeutics. This project resulted from a collaboration between Tacere and Pfizer Inc. to develop and commercialize the TT-033 family of compounds to provide a potential single-administration treatment for the Hepatitis C virus (HCV).

Exiqon was selected to complete a series of customized assays, aimed at the detection of therapeutic shRNA molecules currently under development by Pfizer and Tacere, using Exiqon's proprietary LNA™ technology platform. The assays will be used to assess critical performance points for the RNAi therapeutic in a pre-clinical setting.

"The ability to quantify the levels and assess the biodistribution of therapeutic RNAi are critical issues for those in the field who are developing the technology as a new therapeutic modality," says David Suhy, PhD, Director of Research & Development for Tacere. "Unfortunately, the use of traditional quantitative assays to obtain these data has been hampered by the exceptionally small size of the RNA. The combination of the unique properties of LNA-enhanced oligonucleotides and the demonstrated scientific expertise of the Exiqon team made them an attractive partner for this project."

The nature of the specimens posed a significant challenge for this kind of assay, according to members of the Exiqon development team. "Unlike many detection assays of this kind, which are designed for use within a single cell type per experiment (e.g. cell-pellet assays), this project required the use of animal tissue specimens, which adds a layer of complexity, due to the presence of heterogeneous cellular populations within tissues," says Adam Baker, Director of Diagnostic Product Development for Exiqon. "In addition, these assays were designed and developed for use in Formalin-fixed, Paraffin-embedded (FFPE) tissue samples." FFPE sample preparation, which is an important preservation method used mainly in medical histology, provides several key benefits to researchers and clinicians. FFPE specimens may be stored indefinitely at room temperature, enabling their use in certain diagnostic applications, even decades after fixation. Formalin, however, which is used to fix the tissue, causes cross-linkage between nucleic acids and proteins, leading to RNA modification and degradation, which can impede the development of nucleic acid detection assays in these specimens.

The development of these customized assays represents a new breakthrough in the detection and quantification of RNAi species in tissue samples, according to Exiqon Chief Commercial Officer Erik Holmlin. "Until now, researchers have had to choose, within any particular tissue sample, whether to determine how much of their target is present, or where it has been localized in the tissue," Holmlin explains. "Through the application of LNA™ technology in this novel approach to assay design, we are able to deliver both pieces of information on the same tissue sample, adding a new depth of perspective into the biology taking place. We believe this has the potential to dramatically improve the quality of information obtained from these types of experiments, and reduce development times for new therapies."

Financial terms of the project were not disclosed.

About Exiqon
Exiqon is a biotech company with activities in three business areas where the company's technologies provide a competitive advantage: Sale of diagnostic tests (Exiqon Diagnostics), sale of innovative research products for miRNA research (Exiqon Life Sciences), and in contract research together with pharmaceutical companies (Exiqon Pharma Services). Exiqon is dedicated to personalizing the treatment selection for cancer patients. The aim is to optimize the use of existing medicine and avoid unnecessary and non-effective treatment. By using molecular diagnostic tests that analyze the genetic profile of each patient's tumor, treatment selection can be optimized for individuals. Exiqon is uniquely positioned to develop such new diagnostic tests. Exiqon already markets diagnostic tests that based on fresh tumor tissue enable doctors to test whether their patients are resistant to one or more of the chemotherapies offered to treat these patients and help them select an efficacious treatment. Exiqon's new molecular diagnostic products are based on the LNA™ technology that enables testing on fixed tissue. Launch of the first molecular diagnostic product was announced in December 2008. A number of new products will follow in the years ahead. Using the LNA™ technology is what has allowed Exiqon to establish a position for itself as one of the market's leading providers of research products for gene expression analysis. These research products are used by university scientists and in the pharmaceutical industry around the world to make groundbreaking discoveries about the correlation between gene activity and the development various diseases. Exiqon is also collaborating with pharmaceutical companies in their effort to develop new medicines based on biomarkers (Personalized Medicine). Exiqon has more than 170 employees and is listed on the NASDAQ OMX in Copenhagen. Exiqon is financed until expected breakeven in 2011.

About Tacere Therapeutics
Tacere is an innovative biotechnology company focused on developing therapeutics to treat serious infectious diseases using its proprietary knowledge in the development of RNAi therapeutics. Tacere is located in San Jose, California, USA and its lead therapeutic compounds are RNAi drugs for the treatment of Hepatitis C. For additional information, please visit

Forward-looking statements:
This announcement contains forward-looking statements regarding Exiqon's potential future development and financial performance and other statements which are not historical facts. Such statements are made on the basis of assumptions and expectations which, to the best of Exiqon's knowledge, are reasonable and well-founded at this time, but which may prove to be erroneous. Exiqon's operations are characterized by the fact that its actual results may deviate significantly from that described herein as anticipated, believed, estimated or expected.


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