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Exiqon Plans to Launch Two miRNA Diagnostics in 2014

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By Doug Macron

Exiqon said it could have two microRNA-based diagnostic tests on the market by 2014 as it provided new details last month on its trimmed-down pipeline.

However, the company, which maintains three diagnostic projects in total, noted that those plans remain contingent on finding partners to handle late-stage development and marketing responsibilities.

Exiqon also reported its financial results for the fourth quarter of 2010, reporting an increase in revenues and a sharp decline in operating costs, which helped lower losses. It also said it expects to conclude an ongoing intellectual property dispute with Santaris Pharma before the end of 2011.

The first of Exiqon's two diagnostic candidates is designed to use miRNA expression signatures to determine stage II colon cancer patients at risk for disease recurrence and for whom adjuvant therapy may be warranted.

Late last year, the company published data in Clinical & Experimental Metastasis showing that high levels of miR-21 were associated with a 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.

It also found that of those 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. By comparison, roughly 25 percent of all stage II colorectal cancers will recur, and the disease's average mortality rate is five years.

The study was based on in situ hybridization, and Exiqon said it is now working to translate the findings to its real-time qPCR platform, with the goal of launching a test in 2014, “provided we find a partner for commercialization.”

Last August, Exiqon had predicted it would find that partner before the end of 2010.

Next in the pipeline is a blood-based test for the early detection of colon cancer, which Exiqon expects could allow doctors to limit invasive colonoscopy procedures to those at high risk for the disease.

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 firm said it is now developing the miRNA signature that will be used to identify people at risk for the disease in a “large prospective clinical trial,” expected to begin this year. The test, which will also be based on qPCR, could be commercially available in 2014, but, again, with the caveat that the company finds a partner.

Pipeline Dreams

Long interested in miRNA diagnostics, Exiqon made its most significant investment in the space in 2007 when it paid $45 million to buy California-based Oncotech, a provider of molecular oncology testing services (GSN 11/29/2007).

Although the acquisition was supposed to give Exiqon the resources to launch its first miRNA cancer diagnostic before the end of 2008, developing such a test proved more challenging than the firm may have expected. By the end of 2009, Exiqon decided to divest Oncotech and only commercialize diagnostics with partners (GSN 12/17/2009).

Those plans ran aground, however, when Exiqon could not find a buyer. Exiqon CEO Lars Kongsbak told Gene Silencing News last June that the Medicare administrative contractor for California had decided to no longer reimburse for Oncotech's existing products, hamstringing the company's ability to compete with its peers. As a result, Exiqon shut down Oncotech (GSN 6/10/2010).

Meanwhile, Exiqon had been quietly shedding a number of miRNA diagnostics programs from its pipeline, including tests to detect drug resistance in patients with stage II, III, and IV colon cancer and with stage II, III, and IV lung cancer; and a test for evaluating the risk of stage I and II endometrial cancer recurrence.

In its 2010 annual report, Exiqon said that it expects to carry its diagnostic programs from proof of principle and through the miRNA signature-identification and assay-development stages. A partner would be required to handle both clinical validation, which “is often costly and will require regulatory skills that Exiqon … does not currently posses,” as well as commercialization.

Exiqon's final miRNA diagnostic, a test for identifying the source of cancer of unknown primary origin, is currently ready for clinical validation, the company said, adding that it is “investigating the potential for commercializing parts of this product through a third party.”

The Financials

For the three-month period ended Dec. 31, Exiqon said revenues rose 42 percent to DKK27.8 million ($5.2 million) from DKK19.6 million the year before, an increase driven by growth in sales from its life science research products operations.

Operating costs in the quarter shrank 75 percent to DKK23.8 million, in part reflecting the effect of a 2009 restructuring of Exiqon's life science division. Research and development spending dropped 90 percent to DKK8.2 million, while selling, general, and administrative costs were down 6 percent to DKK15.4 million.

Exiqon's net loss for the fourth quarter was sharply down at DKK8.6 million, or DKK0.25 a share, from DKK89.9 million, or DKK2.97 a share.

Looking ahead, Exiqon said it expects it could break even in 2011, and that this guidance does not include the potential impact of arbitration proceedings ongoing in the Danish courts with Santaris Pharma.

According to Exiqon, last September it initiated the proceedings after becoming concerned about a “potential breach” by Santaris of the company's co-ownership arrangement for the LNA technology.

Santaris was established when Cureon, a company Exiqon founded to develop LNA-based therapeutics, merged with Panteco. Exiqon said that under a licensing deal with Santaris, it holds all rights to the LNA technology outside of therapeutics, yet Santaris may be “commercializing products for the conduct of research.”

“We have a slightly different view of how we read [the companies' licensing] agreement and [arbitration] is the best way to figure out how we draw the line,” Kongsbak told Gene Silencing News this week. He added that the dispute specifically centers around the way Santaris is using raw materials, but didn't provide additional information.

Exiqon said it is seeking damages of DKK98.6 million, and that Santaris is seeking counter-damages of DKK100 million. All arbitration proceedings are expected to conclude in 2011, Exiqon said.

Officials from Santaris were not available for comment.


Have topics you'd like to see covered in Gene Silencing News? Contact the editor
at dmacron [at] genomeweb [.] com.

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