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Santaris Inks LNA Rx Deal with Shire, Leveraging Technology in Areas Outside of Core Focus


By Doug Macron

Santaris Pharma announced this week that it has signed a deal to use its locked nucleic acid technology to develop drug candidates against rare genetic disorders for British biopharmaceutical firm Shire.

The deal, which calls for Shire to handle the majority of the drugs’ development, provides Santaris with a way to realize additional value from its technology in areas outside of its core expertise as it advances its own therapeutic programs, Santaris CSO Henrik Orum told RNAi News.

And while those internal programs include the phase I microRNA-targeting hepatitis C treatment SPC3649, Orum indicated that the company isn’t likely to advance additional miRNA drugs into the clinic in the near term as it takes a measured approach to selecting future clinical candidates.

A pioneer in the miRNA space, having been the first to move a drug targeting the non-coding RNAs into human testing with SPC3649 (see RNAi News, 5/29/2008), Santaris has “quite a number” of miRNA research and development efforts currently underway, he said.

Yet the relationships between miRNA function and dysfunction and disease are not as clear-cut as, for example, they are with messenger RNA, Orum explained. “So it’s not as easy at this point to choose targets for clinical development in the microRNA world as it is in the messenger RNA world, and that obviously dictates how we address the field.”

When it comes to the in-house drug-development process, Santaris favors a de-risking approach that involves judging potential targets against a set of criteria in order to minimize the risks associated with clinical studies, he said.

“Obviously, we want to recruit patients effectively and quickly, we want clinical endpoints to be measurable and readily achievable in a reasonable timespan, et cetera,” he noted. “If a microRNA satisfies that list of criteria, which also includes third-party IP assessment … it has as good of a chance to become an in-house development candidate as a messenger RNA.”

But at this stage, “the messenger RNA targets are more likely to qualify on that set of objective parameters than a microRNA,” Orum said. “That’s just how it is today; it’s not a decision to do either/or. Basically, we let this set of criteria determine what we’re doing.”

And Santaris’ pipeline bears this out. According to the company’s quarterly report for the period ended June 30, of the company’s ten most-advanced drug candidates, only one, SPC3649, targets a miRNA. Santaris lists a miRNA-directed oncology program in the earliest stages of preclinical development, but does not provide any details on the targets themselves.

Still, Santaris has no plans to give up on the lead in the space. Orum noted that the company is currently looking at “a number of different microRNAs from a drug-discovery perspective.” At the same time, it is preparing to release data from the recently completed phase I study of SPC3649 and is planning to begin a second phase I trial, this time evaluating multiple doses of the drug, before the end of the year, he added.

Playing with Shire

Under the terms of the deal, Santaris will use its LNA drug platform to identify and select drug candidates against certain targets provided by Shire.

Although a Shire spokeswoman said that details about the targets are not being publicly disclosed for competitive reasons, the company said that they would be related to “rare diseases,” building off its existing position in enzyme-replacement therapies.

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"With this novel platform technology Shire has the potential to move into a wider range of genetic orphan diseases,” Sylvie Gregoire, president of Shire’s human genetic therapies business, said in a statement. “We will look to develop treatments for people with a variety of rare life-altering and life-threatening conditions that, to date, could not be effectively addressed by enzyme replacement therapy."

Once Santaris has developed the LNA oligos, Shire will take over all responsibilities for the drugs’ development and commercialization.

In exchange, Santaris said it will receive “initial early-stage payments” of $6.5 million, which covers technology access fees, exclusivity for three predefined targets, and initial discovery funding. Santaris also stands to be paid an additional $13.5 million upon the successful completion of certain initial studies.

The term of the arrangement is two years, although Shire has the option to extend the deal for an additional two years. Further terms were not disclosed.

"We are delighted that the increasing interest in the LNA drug platform and our drug-discovery engine so quickly has materialized in our fourth strategic alliance,” Santaris President and CEO Soren Tulstrup said in a statement. The deal with Shire “follows only seven months after the announcement of our new strategic alliance with Wyeth Pharmaceuticals.”

In January, Wyeth acquired the rights to use Santaris’ LNA technology to develop drugs targeted mRNA (see RNAi News, 1/15/2009).

In 2007, Santaris struck a similar arrangement with GlaxoSmithKline (see RNAi News, 12/20/2007). Like the Wyeth arrangement, the GlaxoSmithKline deal did not expressly cover miRNAs. However, the British pharmaceuticals giant did take an option to develop and commercialize SPC3649.

Orum told RNAi News this week that that Santaris is currently in discussions with GlaxoSmithKline about the option, which has not yet been exercised, but he declined to elaborate.

In 2006, Santaris forged its first strategic alliance with Enzon to develop LNA-based cancer drugs. This arrangement does not include miRNA targets.

Going to California

Orum also confirmed this week that Denmark-based Santaris continues to move forward with a planned US subsidiary, and said that an announcement on the effort is expected to be made in “the very near future.”

As reported by RNAi News, the company began setting up a San Diego, Calif.-based subsidiary earlier this year in order to strengthen its position in the US (see RNAi News, 4/16/2009). The company’s newly appointed Chief Development Officer Arthur Levin, who was formerly senior vice president of drug development at antisense shop Isis Pharmaceuticals, is expected to lead the new unit.

Orum declined to provide additional details about the subsidiary ahead of the planned announcement.

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