Exiqon announces launch of first molecular diagnostic test for colon cancer recurrence based on miRNA

Exiqon A/S, NASDAQ OMX Copenhagen (“EXQ”), today announced the launch of its first molecular diagnostic test based on miRNA. The miRNA-based prognostic test will help identify stage II colon cancer patients who may be at significantly higher risk for recurrence and for whom adjuvant chemotherapy may be warranted.

Annually, colon cancer affects about 108,000 people in the U.S. (1.2 million colorectal worldwide) with 25% of them diagnosed as stage II. Generally, stage II patients are not treated with adjuvant chemotherapy, yet up to 25% of them will recur. The new miRNA test identifies patients who are at increased risk for recurrence and thus helps guide physicians in making critical treatment decisions.

The test has been completed and commercialization will begin in January 2009. The test will be marketed to colon cancer surgeons, medical oncologists and pathologists across the U.S. and internationally through Exiqon Diagnostics CLIA laboratory. Efforts will initially focus on establishing early adopters of the test to drive uptake en route to gaining support from payers in the U.S. market.

The successful completion of this first miRNA test for commercialization through Exiqon Diagnostics CLIA laboratory marks an important milestone in realizing the synergies from a successful integration of Oncotech Inc. following the acquisition by Exiqon earlier this year.

Exiqon’s new prognostic test for colon cancer recurrence based on miRNA is the first in a series of new in situ-based molecular diagnostic tests enabled by Exiqon’s proprietary LNA(tm) technology that can now be marketed through Exiqon Diagnostics.

“With the timely completion of our first miRNA based diagnostic test following the acquisition of Oncotech, we have managed an important first step towards realizing the full potential of our recent acquisition. Now we have the product development capabilities in place to commercialize through our CLIA laboratory a truly remarkable pipeline of new diagnostic and prognostic tests based on miRNA which will improve patient care and cancer treatment selection,” states Lars Kongsbak, Ph.D., president and chief executive officer of Exiqon.

On track towards realizing our strategy. Next steps:

MicroRNA’s have recently been found to play a fundamental role in the regulation of cancer. With its proprietary LNA(tm) technology, Exiqon has developed novel methods to accurately measure these important RNA molecules in small amounts of tissue. Over the next few years, Exiqon will be introducing a series of proprietary prognostic, predictive and drug response tests based on miRNA (miRNA tests). These new miRNA tests will be designed to address key questions in the treatment selection for and management of cancer patients. Initially focus will be within lung, colorectal, ovarian and endometrial cancers. Exiqon is also developing an exquisitely sensitive propriety miRNA-based “Cancer of Unknown Primary” (CUP) test which can identify the origin of an unidentified cancer from a small sample of the metastasised tumor.

Exiqon plans to introduce two to three proprietary miRNA-based tests each year over the next few years to address the unmet diagnostic need for better treatment selection in high incidence cancers: lung, colorectal, ovarian and endometrial. These new miRNA-based tests, along with the Oncotech EDR(r) Assay, will have the potential of helping guide treatment decisions in over 171,000 lung, 118,000 colorectal, and 53,000 ovarian/endometrial cancer patients yearly in the U.S. alone.

The miRNA-based tests can be rapidly developed and validated due, in part, to Exiqon’s extensive tumor and tissue repositories comprising a total of 150,000 samples, as well as in collaborations with key institutions such as MD Anderson, National Cancer Institute, and leading university hospitals in Denmark (e.g. Rigshospitalet). Initially, Exiqon will launch these tests through its CLIA-certified laboratory in the U.S. and, in the future, make them available to other laboratories and pathologists as fully approved in vitro diagnostic kits.

An overview of planned proprietary product offerings by Exiqon Diagnostic including the pipeline of new miRNA based molecular diagnostic tests is depicted below.

The figure shows the current and planned proprietary product offering by Exiqon Diagnostics. EDR, Extreme Drug Resistance; FFPE, Formalin Fixed Paraffin Embedded; CUP, Cancer of Unknown Primary. 1) American Cancer Society, Cancer Facts & Figures 2008, 2) American Cancer Society, Global Cancer Facts & Figures, 2007. Stage distribution is based on numbers published by National Cancer Data Base.

The introduction of a new molecular diagnostic in oncology typically requires a year or more to establish the adoption and broad clinical validation that is required for adequate reimbursement.

“We believe our proprietary miRNA tests will be of significant value to the healthcare system. These assays are anticipated to help physicians make more informed and individualized treatment decisions that will benefit patients and reduce downstream healthcare costs,” says Erik Holmlin, Ph.D., Exiqon’s chief commercial officer.

As part of the overall diagnostic strategy, a number of complimentary, non-proprietary molecular diagnostic tests will be added to Exiqon Diagnostics’ menu. These additional assays will help address the need of cancer treating physicians who require a variety of tests to make optimal treatment decisions for each patient.

Notably, Exiqon has had significant progress in detection of various plasma-based miRNA profiles within cancer. Due to the significant potential and market need for monitoring of miRNA in plasma, Exiqon Pharma Services will be offering miRNA profiles in plasma performed on the basis of our LNA(tm) technology to selected customers from January 2009.

“I’m amazed how miRNAs keep revealing new opportunities within diagnostics and disease monitoring, and the recent discoveries related to miRNAs in blood may be the beginning of a new era in diagnostics. Exiqon has developed a number of proprietary extraction and detection technologies for identification of miRNA in blood and is currently in the process of identifying a partner to take on this new opportunity,” says Lars Kongsbak, Ph.D. president & chief executive officer of Exiqon.

Exiqon maintains its long-term financial goal of reaching profitability by 2011 with its current cash position. A financial guidance for 2009 will be available in March 2009 following announcement of year end results for 2008.