CARLSBAD, Calif. — Stroll inside Ionis Pharmaceuticals’ Southern California headquarters, past the giant hanging glass sculpture of an RNA molecule, and you can’t miss it on the back wall: In bright red, a digital countdown clock ticks off the days, hours, minutes, and seconds until a moment the biotech is betting will be transformational. It’s now down to just six days.
Ionis is expecting a Food and Drug Administration decision by Dec. 19 on whether to approve olezarsen, a therapy designed to improve the body’s ability to break down fats. While the biotech is seeking initial approval for a rare genetic disease, familial chylomicronemia syndrome, it sees an opportunity to treat a broader swath of patients with sky-high triglycerides. Peak sales could reach $2 billion in 2035, according to Wolfe Research, a sell-side research firm.
This isn’t just a drug approval, but a major strategic shift. For more than 30 years, Ionis developed all of its medicines in partnership with large pharmaceutical companies. This time, it is standing alone.
“The business model was to partner everything,” said CEO Brett Monia, who took the reins in 2020 and implemented the new strategy. “We would give our drugs, drugs that we conceived, discovered, and developed, and we would license them to every company — Biogen, GSK, AstraZeneca, oh, you name it,” he told STAT. “And I recognized that not only does that limit the value to the company, but it limits the value to patients.” Big Pharma, he added, doesn’t move with the urgency of a nimble biotech like Ionis, which can delay the arrival of new treatments on the market.
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