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Insight Genetics Wins $1.5M Grant to Begin Clinical Testing for ALK Mutation CDx on Qiagen Platform

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Insight Genetics has been awarded a contract from the National Cancer Institute worth nearly $1.5 million to begin clinical testing of its qPCR-based assay for ALK mutations in lung cancer.

The test, which is based on intellectual property licensed from St. Jude Children's Research Hospital and being developed on a Qiagen real-time PCR system, is on track to soon be offered as a research-use-only assay, and as a CE-marked kit in the second quarter of next year, the company said this week.

Assuming Insight is successful in these endeavors, it then plans to carry out clinical studies in collaboration with an unnamed pharmaceutical partner in order to pursue US Food and Drug Administration approval of the assay as a companion diagnostic to an ALK inhibitor.

Insight's latest award is a two-year Phase II Small Business Innovation Research grant that follows the successful completion of a Phase I SBIR grant awarded to the Nashville, Tenn.-based company in November 2010.

The company's assay, called Insight ALK Screen, detects cancer-causing fusions and mutations of anaplastic lymphoma kinase, which have been demonstrated to have a pathogenic role in many cancers, including non-small cell lung cancer, diffuse large B-cell lymphoma, and anaplastic large cell lymphoma.

Approximately 5 percent to 10 percent of lung cancers are caused by ALK mutations. Pfizer recently won FDA approval for its drug Xalkori (crizotinib) to treat ALK-positive non-small cell lung cancer patients. Several other pharma companies, including Ariad, Novartis, Synta, and Xcovery, are also developing ALK-inhibitor therapies.

The current diagnostic standard for ALK mutations is Abbott's Vysis AK Break Apart FISH assay, which the FDA cleared in August 2011. According to Insight, however, this test has low sensitivity and is difficult to interpret, making it difficult for physicians to select the proper patients for ALK inhibitor treatments. In addition, as reported by PCR Insider sister publication Pharmacogenomics Reporter, results from a recent study suggest that FISH testing may miss some patients with rare types of ALK fusions that don't fit Abbot's FDA-approved testing criteria.

As part of its Phase I project, Insight demonstrated that its assay, which is based on real-time PCR, is "agnostic with regard to the 5' fusion partner of ALK, highly sensitive in cell line dilution studies, and highly specific" in formalin-fixed paraffin-embedded specimens from NSCLC patients, the company wrote in its recently published grant abstract.

In Phase II, Insight plans to demonstrate clinical utility of ALK Screen by testing a large cohort of clinical specimens "to unequivocally demonstrate statistical significance in sound patient selection for ALK inhibitor therapy," according to the grant's abstract.

If successful, Insight will then co-develop the product as an in vitro diagnostic with partner Qiagen in order to commercialize it as an FDA-approved companion diagnostic.

In January, Qiagen said that it had entered into a worldwide co-development and licensing deal with Insight for intellectual property related to ALK biomarkers.

This week, in an email to PCR Insider, Insight's vice president of R&D David Hout noted that Qiagen is "the sole development partner for the Insight ALK Screen," and that the companies are developing the assay on Qiagen's Rotor-Gene Q real-time PCR system with high-resolution melt analysis.

Although Insight declined to detail exactly when the partners might begin clinical studies for ALK Screen, Hout noted that "studies are underway to offer the assay soon as a RUO and CE-marked kit in Q2 of 2013. Clinical studies for an FDA companion diagnostic are an extension of the RUO/CE studies, which will be carried out in collaboration with a pharmaceutical partner."

Insight is developing another assay called ALK Resistance that will use allele-specific PCR to detect resistance mutations discovered by the inhibition of ALK in order to help physicians monitor cancer patients for mutations and upregulation in the ALK gene in order to make more effective treatment decisions.

In December 2010 Insight inked an exclusive worldwide license with St. Jude for the resistance mutations, discovered in the laboratory of St. Jude researcher Stephan Morris. At the time, company officials said that the company was developing this assay on Life Technologies' ABI 7500 Fast Dx real-time PCR system (PCR Insider, 12/23/2010).

Insight said last October that it had also netted a $193,000 grant from NCI to further develop ALK Resistance. The company did not provide an update on the progress of this test.

Hout noted that the IP it originally licensed from St. Jude for resistance mutations is separate from discoveries made at the institution related to the ALK Screen assay, and that Insight has licensed additional IP for that test.