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Small Pharma Firm Thinks Big in Partnerships,Treatments

Despite some market uncertainty for one of its top drugs, Kynamro, Carlsbad-based Isis Pharmaceuticals Inc. has positioned itself as a drug discovery frontrunner with its proprietary “antisense” therapies.

The company, which operates with a relatively lean 345 employees, has a broad pipeline of first-in-class drugs in development — 18 drugs in clinical development and five more in pre-clinical study. It leaves commercialization to the big pharma firms that it partners with, said Isis founder and Chief Executive Officer Stanley T. Crooke.

These partnerships are a key part of enabling Isis to do big things.

This year, Isis forged three separate agreements with Weston, Mass.-based Biogen Idec. The most recent was a $30 million upfront deal to begin developing three new drugs — with potential future payments that could make the deal worth $600 million. And it received $25 million upfront from British AstraZeneca PLC to develop an antisense cancer-fighting drug, with further payments promised if the Isis meets certain milestones in its clinical trials.

Drawing Investors

Since its 1989 launch, Isis has raised nearly $3 billion in venture capital and partnerships with companies such as GlaxoSmithKline PLC, Pfizer Inc. and Novartis AG. The publicly traded company is today worth about $1 billion.

Isis holds about 1,500 patents, many of them relating to antisense therapy — engineered RNA molecules that work to deactivate faulty genes. The company’s volume of intellectual property allows it control of the antisense therapy space for the foreseeable future, Crooke said.

“Isis is clearly the leader in RNA-based therapies, and as a result, they have had remarkable success in creating a breadth of partnership arrangements,” said Pratik Shah, a partner at La Jolla venture capital firm Thomas, McNerney and Partners.

Shah and his colleagues helped fund a company that branched out from Isis in 2007 called Altair Inc., but it folded in 2011 because its asthma medication performed poorly in clinical trials. Shah is no longer involved with Isis, but said he is following its trajectory closely.

The Market Test

“Only when these drugs start to really hit the market and generate significant sales is when I think the true underlying value and potential of their technology will be realized,” Shah said. “It’s taken a lot more time and money than I think anyone had expected, but there’s no doubt you can tackle unique disease targets with this technology.”

Development has been costly and time-consuming, as evidenced by the lukewarm performance of Isis’ biggest investment so far. According to regulatory filings, ten years of research and $350 million from Boston-based Genzyme Corp. have been invested in Kynamro, a drug that treats a form of high cholesterol.

In mid-December, a European Union regulatory agency did not recommend Kynamro’s approval due to possible side effects such as liver damage; a decision Isis’ development partner hopes to soon have re-examined

FDA Outlook

In the U.S. the drug is faring better. In October, a Food and Drug Administration panel recommended the drug receive U.S. approval.

“We have high hopes it will be approved here in the U.S.” Crooke said. “That’s (the panel’s recommendation) further validation of what we’ve created, and it’d be nice to have some real product revenue,” he said.

The FDA is expected to make a decision on whether to approve Kynamro by January 29.

Crooke said that the Isis’ strength is its ability to outpace larger companies in its ability to formulate new therapies. Crooke said he has no intention to develop Isis into a commercial entity.

“I’ve run large R&D companies, and smaller is better,” he said. “We believe we bring the most value by staying small, and focusing on what the patient and industry needs — innovation in science and technology that’s more efficient than traditional approaches.”

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