Exiqon Reports Positive Results on Early Detection of Colorectal Cancer in Blood

Copenhagen, Denmark – Exiqon A/S (NASDAQ OMX: “EXQ”), a leading supplier of research products for qPCR analysis and biomarker development, today announced positive results in the company’s development of a diagnostic test for early detection of colorectal cancer in blood. The results were revealed during an oral presentation at the annual American Association for Cancer Research (AACR) meeting in Florida, USA, today.

In a multicenter study, Exiqon has demonstrated the ability of the company’s proprietary miRCURY LNA™ Universal RT microRNA PCR platform to robustly detect miRNA biomarkers associated with the presence of colorectal cancer based on

less than 0.2 ml blood.

“The results we present today represent the preliminary culmination of many years of technology development which now allows Exiqon to monitor minute amounts of miRNA biomarkers in blood based on a standard blood sample format

and handling protocol. We are excited about this promising potential for our first diagnostic test based on standard PCR analysis of blood samples taken under standard testing procedures”, says Dr. Adam Baker, Vice President of

Exiqon Diagnostics.

The objective for the first phase of the early detection of colorectal cancer program was to identify candidate miRNA biomarkers in plasma samples from stage II/III colorectal cancer patients and age- and gender-matched colonoscopy-verified healthy controls. A genome wide screen in blood plasma profiled 730 individual miRNAs from 50 stage II cancers and 50 matched controls. The results were used to develop a candidate panel of miRNAs detectable in less than 0.1 ml plasma.

In the second phase of the program, the candidate panel of miRNAs was profiled in a study set of 227 stage II/III colorectal cancer patients and matched controls. The results demonstrated that using a simple miRNA signature derived

from plasma obtained under standard clinical conditions it is possible to detect colorectal cancer. In the study, including samples from patients with proven colorectal cancer and matched healthy subjects from five Danish hospitals, the biomarker signature had a sensitivity and specificity for cancer of 75% and 80%, respectively, easily fulfilling the requirement for a

commercial test.

Exiqon has begun the next phase which includes final assay development and validation of the biomarker signature in a larger study cohort of more than 3,000 patient/control samples. Exiqon plans to publish the results of the validation study before year end.

The goal of Exiqon’s early detection of colorectal cancer program is to develop, validate, and market, through a partner, a simple, safe, sensitive, and specific blood-based screening method that, by implementation into nation-wide screening programs will result in earlier detection of colorectal cancer (down-staging) and therefore reduced mortality due to colorectal cancer.

In the western world, 600.000 patients are diagnosed with colorectal cancer every year and it is the second most deadly cancer. Today, guidelines worldwide recommend that every person older than 50 years is screened regularly for early

detection of colorectal cancer. The only reliable screening test offered today is colonoscopy. However, this test is both time consuming and inconvenient, and compliance is therefore very low. As a result, an unmet need for a simple blood

based test exists. The addressable market for such a convenient test is in excess of USD 3 billion.

For an abstract of Exiqon’s presentation given at the AACR conference today:

“Discovery of a miRNA-based RT-qPCR signature able to detect early stage colorectal cancer in blood plasma”, please see the attached PDF or follow the below link for an online version (abstract 2816):

http://www.abstractsonline.com/plan/ViewAbstract.aspx?mID=2734&sKey=3143326d-b0b1-4408-9077-09ac88372faa&cKey=74343b2b-06d3-41b2-95cd-8c8b3114c641&mKey=%7b507D311A-B6EC-436A-BD67-6D14ED39622C%7d

Additional information:

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

Adam Baker, Vice President, Diagnostics and Pharma Alliances, tel. +45 4566 0986 (cell: +45 4090 2153)

www.exiqon.com/investor/portal.

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