Antisense specialist Isis Pharmaceuticals ($ISIS) has made it all the way to mid-stage trials with ISIS-APOCIIIRx, targeting patients with a rare ailment that leads to dangerously high triglycerides. But that candidate is looking all the more promising in light of two major studies linking its target to reduced rates of heart attack, handing the biotech an added potential blockbuster indication.
Isis' drug inhibits the protein APOC3, which is tied for triglyceride metabolism in the blood. The company is in the midst of Phase II development in familial chylomicronemia syndrome (FCS), which the National Institutes of Health says affects about one in a million people, but the results of two New England Journal of Medicine studies may spur it to consider widening its plans.
Looking at patients with mutations that blunted their APOC3 genes, one group of NIH-funded researchers found a 40% reduction in triglyceride levels and, most promising, a 40% lower risk of heart disease. A second, unrelated study in Denmark produced "eerily consistent" results, one investigator told The New York Times. To date, cardio drugs have mostly targeted LDL, or "bad," cholesterol in hopes of improving heart function--including the new crop of expected-blockbuster PCSK9-blocking therapies--and the potential APOC3 pathway presents the biggest advance in years, researchers said.
Now back to Isis. The biotech is suddenly sitting on a mid-stage candidate with activity against the industry's latest en-vogue target. Piper Jaffray analyst Joshua Schimmer told Bloomberg that an FCS approval alone could be worth $2 billion in sales, and the company could double that if it swung for a wider indication.
CEO Stanley Crooke said he's keeping his options open and his phone lines clear.
"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," Crooke told Bloomberg. "We have a great deal of licensing interest for this drug."
But, thanks to its wide pipeline of gene-muting therapies and chronically underperforming stock, an acquirer might come along and force its hand, Schimmer said.
"At this valuation, it would be ripe for a hostile event," the analyst told Bloomberg.
- read the Bloomberg story
- here's the NYT's piece on the APOC3 studies