A pivotal time for an RNA pioneer

It’s a pivotal year for RNA therapeutics pioneer Ionis Pharmaceuticals.  After transitioning from a partnering model to a full-ownership strategy following the 2024 approval of Tryngolza, the company is expecting an FDA decision to expand the drug into severe hypertriglyceridemia as its first non-rare-disease indication for a wholly owned product. Meanwhile, potentially game-changing phase 3 readouts are expected from Ionis’ Novartis-partnered lipoprotein(a) candidate and AstraZeneca-partnered ATTR cardiomyopathy program. In this week’s episode of "The Top Line," Fierce’s Angus Liu interviews Ionis CEO Brett Monia, Ph.D. He discusses the science of antisense oligonucleotides, the significance of the two closely watched upcoming clinical readouts, and what it takes to turn a 30-year-old discovery platform into a full-fledged commercial biotech. To learn more about the topics in this episode:  Ionis slashes Tryngolza's price tag by 93% ahead of anticipated label expansion  'It’s going to be huge': The 'diabolical' molecule poised to become biotech's next gold rush  AstraZeneca sets sights on 25+ blockbusters by 2030 to fuel $80B revenue ambition  As questions swirl around ATTR competition, Alnylam plots path to market leadership for Amvuttra  Ionis exec shares method to the Madness after 2026 Drug Name Tournament win  Ionis plots FDA filing after rare neurological disease candidate posts phase 3 win  See omnystudio.com/listener for privacy information.