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Exiqon Pushes Back Launch of First miRNA Dx, Takes NY State Path to Commercialization

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Exiqon President and CEO Lars Kongsbak this week said that the company would not launch its first microRNA-based diagnostic, a test to predict colon cancer recurrence, before the end of the year as previously announced. Instead, the test is slated for introduction sometime early in 2009.
 
He said the company has yet to submit the test for regulatory approval, but expects to do so before the end of the month. He also noted that Exiqon will seek that approval from the New York State Department of Health, even though commercialization of the test will be handled by its California-based subsidiary Exiqon Diagostics, formerly Oncotech.
 
Kongsbak said clearance by New York’s health department is “considered the most complicated approval to obtain because [that agency] has the most sophisticated and advanced system” to assess products. Therefore, if the company wins approval there, “it will be less complicated to get approval from other states in the US,” he added.
 
Exiqon isn’t the only miRNA diagnostic company taking this tack. This summer, Rosetta Genomics obtained approval by the agency for its first miRNA test, which differentiates squamous from non-squamous non-small cell lung cancer (see RNAi News, 7/24/2008). The company launched that test this week [see related story, this issue).
 
And while Rosetta was initially expecting to market its diagnostic through its New York-based partner Columbia University Medical Center, the company said a few months ago that this and future tests would be commercialized by its own recently acquired Clinical Laboratory Improvement Amendments-certified laboratory Parkway Clinical Laboratories, which is located in Pennsylvania (see RNAi News, 8/21/2008).
 
Kongsbak said that the NY approval would be sufficient for Exiqon to begin selling its colon cancer diagnostic throughout the US, but noted that it may be necessary to gain clearance from other states’ regulatory agencies. But given the stringent requirements of the New York State Department of Health, these approvals are likely to be easily obtained, he added.
 
But ultimately, state regulators may not be the issue for miRNA diagnostics developers. Last year, the US Food and Drug Administration indicated that it may begin regulating so-called home-brew diagnostics available through clinical laboratories. However, it is not yet clear how this oversight will impact miRNA tests.
 
Kongsbak told RNAi News this week that he expects the FDA will eventually begin regulating miRNA diagnostics, in part because of other companies that “have pushed the limit of CLIA labs to where the authorities want to get more [robust] control system in place. If that comes, we’ll certainly respond to that and make sure we obey the rules.”
 
‘All About Personalized Medicine’
 
Although its colon cancer recurrence test is to be Exiqon’s first miRNA diagnostic, the company has a number of others under development. Kongsbak said Exiqon intends to limit its diagnostic portfolio to cancer-treatment selection, and noted that one area of development will be ovarian cancer. He declined to disclose other indications.
 
“So they may be related to drug sensitivity, drug resistance, drug metabolism, [or they] may be predictive tests like the [colon cancer] recurrence test,” he said. “We’d like to provide the products [physicians] need to make the final decision on which treatments to give each patient. It’s all about personalized medicine.”
 

“I’m of the opinion that if you want to succeed and be number one, it’s very important that you stay focused in your business. … I would rather be number one in a well-defined area than be number ten in a broad spectrum of different applications.”

As a result, Exiqon will pass on a variety of potentially lucrative opportunities within the miRNA diagnostics space, including early detection of cancer — an area that peer Rosetta Genomics recently said it intends to enter (see related story, this issue).
 
“I’m of the opinion that if you want to succeed and be number one, it’s very important that you stay focused in your business,” Kongsbak explained. “If we try to do too many things, we will dilute our resources — human resources, as well as financial resources. I would rather be number one in a well-defined area than be number 10 in a broad spectrum of different applications.”
 
In line with that approach, Kongsbak said, Exiqon will also limit its diagnostic activities to oncology despite the increasing body of evidence linking miRNAs to a variety of other conditions including cardiovascular disease.
 
“We can see there are opportunities there,” but Exiqon simply does not have the resources to pursue them all, Kongsbak said. Even within cancer, the company expects its reach will be limited, he added.
 
“We … plan on soon releasing information on the types of cancer we will focus on because we’re not doing every cancer out there,” he said. “It’s simply too complicated to focus on all cancers.”
 
Kongsbak declined to elaborate in detail on which cancers that Exiqon will pursue, although he indicated that ovarian cancer would be one.
 
‘Outsourcing Profiling’
 
As Exiqon advances its diagnostics operations, which Kongsbak said would be a key factor in helping the company reach its goal of profitability in 2011, it is also looking to leverage its miRNA expertise in other areas.
 
According to Kongsbak, in 2009 the company plans to begin offering miRNA profiling in blood and serum to both academic and industry customers.
 
“There is no doubt that big universities have a significant need for outsourcing profiling,” he said. At the same time, pharmaceutical firms are increasingly interested in exploring the potential of miRNAs as biomarkers for monitoring disease progression or for patient stratification in clinical trials, he added.
 
“There are many applications of microRNAs that pharma companies see now,” Kongsbak said. “They’d like to team up with companies that can offer these services, but also can offer the adequate technologies for that,” and Exiqon aims to be that provider.