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Exiqon Preps miRNA Diagnostics for 2014 Launch, Still Needs to Find Partners


By Doug Macron

Exiqon last month provided an update on its pipeline of cancer-focused microRNA-based diagnostics, saying that it could have three tests, including ones that measure miRNA expression using biofluids, on the market within two years.

The company also released its fourth-quarter 2011 financial results, posting a small profit amid a roughly 8 percent rise in revenues.

Denmark-based Exiqon has maintained an interest in the miRNA diagnostic space for years, and notably paid $45 million in 2007 to acquire California-based molecular oncology testing services provider Oncotech as part of a bid to introduce its first test in 2008 (GSN 11/29/2007).

Developing miRNA diagnostics, however, proved more challenging than expected, and two years later Exiqon divested Oncotech as it put more attention on its research products business (GSN 12/17/2009).

At the time, the company said that although it would continue working on a trimmed down suite of miRNA tests, it would only commercialize diagnostics with the help of partners.

Remaining in Exiqon's pipeline is a diagnostic that uses tumor tissue to identify stage II colon cancer patients at significantly high risk for disease recurrence and for whom adjuvant chemotherapy may be appropriate.

In 2010, the company reported that high levels of miR-21 were associated with short disease-free survival, and that probes Exiqon developed with its locked nucleic acid technology were able to detect the miRNA in cancer tissue collected from 130 patients with the disease.

The data also indicated that among patients with the highest miR-21 levels, only 73 percent survived two years after surgery. The two-year survival rate for patients with the lowest miRNA levels was 98 percent.

The company said in its 2011 annual report last month that it had completed an independent clinical validation study of more than 500 patients, with data expected to be published in the first half of this year.

“Provided the product development is successful, we expect our colon cancer stage II recurrence test to be commercially available by 2014,” it noted.

Also on deck is Exiqon's blood-based test for the early detection of colorectal cancer, which the company hopes could provide doctors with a diagnostic option that is less invasive than colonoscopy.

In late 2010, Exiqon announced that it and academic collaborators had profiled 730 individual miRNAs in blood plasma from 50 stage II colorectal cancer patients and 50 matched controls, as well as 378 individual miRNAs in 120 stage II cancer patients, 50 stage III cancer patients, and 170 matched controls.

The company said that it completed the analytic validation of the samples in the fourth quarter of last year, and expected the results to be published by the summer. Pending positive results, the early colorectal cancer-detection test could reach the market by 2014, as well.

Joining these two colon-cancer initiatives is a recently launched program in melanoma.

According to Exiqon, it is working with the New York University Cancer Institute to identify blood-borne miRNAs that can be used to diagnose the disease and predict the risk of its recurrence with “high accuracy at the time of initial diagnosis.”

This collaboration yielded a signature of five miRNAs that, when added to standard clinical examination methods, improved prognostic performance significantly (GSN 6/16/2011). In addition, four of the five non-coding RNAs — miR-222, miR-182, miR-339-3p, and miR-338-3p — were statistically significant predictors of melanoma recurrence.

Exiqon said last month that it is working with NYU to “find sponsoring to clinically validate [these] initial results in an independent cohort of patient samples.” Assuming it meets this goal and the data are positive, the melanoma test could be ready for commercialization by 2014, it added.

In its annual report, Exiqon reiterated its reliance on partners to commercialize its miRNA diagnostics, noting that alliances will allow it to share the “risks and costs of commercializing our tests with reputable commercial partners who have the necessary insight and understanding of the diagnostic markets … to successfully market and sell new products.”

The company did not, however, provide any guidance on when such arrangements may be formed.

Fourth Quarter

For the three-month period ended Dec. 31, Exiqon reported a profit of DKK825,000 ($146,581), compared with a year-ago loss of DKK8.9 million ($1.58 million).

Contributing to the profit was a rise in revenues to DKK 30.1 million from DKK 27.8 million, which the company said reflects a rise in use of its MiRcury LNA RT-PCR platform for miRNA research.

Operating expenses in the quarter, meantime, fell to DKK 10.6 million from DKK 12.4 million.

Looking ahead, Exiqon said that it expects to reach profitability this year on anticipated 2012 total revenues of DKK 130 million.

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