Isis Pharmaceuticals Inc., a Carlsbad-based maker of gene therapy treatments, may seek to expand use of an experimental drug targeting triglycerides to fight heart disease, a move an analyst says could add $2 billion in sales.
The drug targets the APOC3 gene and is now aimed solely at a rare disease that’s linked to diabetes and pancreatitis and affects about 5,000 people. Two studies this week, however, have also tied the gene to lower risk of heart disease, an illness that kills 600,000 people yearly, a much larger target.
The research, done independently of Isis, was highlighted in the New England Journal of Medicine. It may open a new window of opportunity for Isis once the drug, called ISIS-APOCIIIRx and heading to final testing, is approved and may spark interest from potential acquirers, analysts said.
“We’re weighing how we want to take full advantage of this breakthrough, and as we do that, we have to consider the potential broader development of the follow on product,” Isis Chief Executive Officer Stanley Crooke said by telephone. “We have a great deal of licensing interest for this drug.”
In the New England Journal of Medicine studies, mutations in the APOC3 gene decreased the risk of a heart attack by about 40 percent. Adding value to the current drug could open the company up to a takeover bid, said Joshua Schimmer, a Piper Jaffray & Co. analyst in New York.
The company has “a super-deep pipeline, and none of it is adequately reflected in the stock price,” Schimmer said in a telephone interview. “At this valuation, it would be ripe for a hostile event.”
Isis (Nasdaq: ISIS), founded by Crooke in 1989, has grown to a $4.09 billion market capitalization and a pipeline of 32 products. On Thursday the stock rose 3.2 percent to $34.82. It has dropped 41 percent since reaching a record high of $59 on Feb. 21, compared with a 6.8 percent drop in the Nasdaq biotechnology index.
Normally, the APOC3 gene works by creating a protein that slows down removal of triglycerides from the blood to make sure the body has energy when it needs it. The mutations may allow them to be more quickly eliminated, the studies suggested.
Triglycerides are a form of fat created from excess calories the body doesn’t immediately use after eating. They gather in the bloodstream as a helpful way to store energy. Having levels that are too high, however, has long been linked to heart disease, though scientists have never definitively determined whether it was a cause or a signal.
The drug’s current path for FCS, meanwhile could generate as much as $2 billion in sales, Piper Jaffray’s Schimmer. If Isis expands the drug’s use to those at high risk of heart disease, it could double sales to $4 billion, he said. Heart disease is the leading cause of death in the United States, according to the Centers for Disease Control and Prevention.
Even though re-targeting the drug would make it available to a larger population “it costs a lot more money to get there,” Schimmer said in a telephone interview. “You need a big sales force to get there.
‘‘No matter which path, though, you’ve got a blockbuster therapeutic,’’ he said.
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