Published: 2009-09-15 14:12:59 CEST
Exiqon A/S - Press release
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. 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.

Additional information:
Lars Kongsbak, CEO, tel. +45 4566 0888 (cell: +45 4090 2101) 

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
www.tacerebio.com. 

See the full press release in the attached PDF.


press_release_tacere_090915_final.pdf