Long an R&D company that partnered off assets, RNAi biotech Ionis Pharmaceuticals shifted in 2025 to bring two medicines to market alone. Analysts are already impressed—and there’s more to come in 2026.
Investors were not exactly expecting Ionis’ GSK-partnered antisense oligonucleotide to achieve a functional cure in chronic hepatitis B. CEO Brett Monia, however, was not surprised.
“I think we made our point. I don’t think it’s seeping in well enough yet, but it will,” Monia told BioSpace on the sidelines of the J.P. Morgan Healthcare Conference in January.
Kicking off a catalyst-rich year for Ionis, partner GSK on January 7 revealed that bepirovirsen achieved a “statistically significant and clinically meaningful functional cure rate” in a Phase 3 trial. GSK did not reveal the specific figures, only saying that the trial met its primary endpoint. The Big Pharma partner will release more details from the trial at the European Association for the Study of the Liver meeting this spring, while a new drug application will be filed in the first quarter.
Monia explained that the trial measured a functional cure rate of about 15–20%, while existing treatments achieve maybe 1–3%.
And last month’s bepirovirsen readout just scratches the surface of what Ionis has in store for 2026. Last year, the RNA interference biotech launched two products independently—Dawnzera for the prevention of hereditary angioedema attacks and Tryngolza for familial chylomicronemia syndrome (FCS).
“Ionis has been historically an R&D organization. Now we built the commercial capabilities to support the launches that occurred,” Monia said.
Tryngolza took home approximately $102 million in 2025 while Ionis has yet to report sales for Dawnzera. But the launch of the former has impressed analysts so far. William Blair in January predicted that Tryngolza could reach $2 billion in sales in 2034.
Ionis also had two successful Phase 3 readouts last year, including one for Tryngolza in severe hypertriglyceridemia (sHTG). Monia said the data showed “remarkable reductions” in triglycerides. The trial, which could help support bringing the treatment into a larger disease population, helped underpin an FDA application at the end of the year.
The initial launch success in FCS is “a sign of the company’s impressive salesforce in cardiometabolic disease and auspicious for a potential launch of Tryngolza in sHTG,” William Blair wrote in a January note.
While Ionis has now launched medicines independently, the potential sHTG indication would be the first time the company has forayed into more common diseases.
In this space, Ionis is up against Arrowhead Pharmaceuticals, another mid-sized biotech that is rising as an independent commercial company. Plozasiran was approved for FCS in November 2025 and a Phase 3 readout for sHTG is expected in the third quarter.
With the FCS approval, Monia said Ionis priced the drug for a rare indication with a cost of $595,000. The CEO promised the price would come down as it moves into a more common indication.
“Let’s face the facts: the FCS market—we pretty much created it, and we’re doing extremely well. There’s not much left for a competitor to come in,” Monia said.
The specific sHTG price has not been announced, although William Blair put the wholesale acquisition cost between $10,000 to $20,000 per year—much lower than Arrowhead’s planned $60,000 cost for the rival medicine.
Ionis also has zilganersen, which showed a disease-modifying benefit in a Phase 3 trial of patients with the rare neurodegenerative condition Alexander disease last year. Monia said that children with the devastating disorder typically do not survive past age four. Ionis plans to file an approval application for zilganersen this quarter.
Elsewhere, Ionis’ partnered pipeline is advancing as well, with four Phase 3 readouts expected this year. In addition to GSK, the company has partnerships with AstraZeneca, Roche and Novartis, as well as a handful of smaller companies.
“It seems like [2025] was the greatest year in our history, based on all of the achievements we made and how we transformed the company,” Monia said. “And I expect to make the same claim in December of this year.”
Ionis will report earnings and provide company highlights on February 25.
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