Ionis Pharmaceuticals — Apr 30, 2025 release

Recent Financial Highlights   • Revenue increased 10% in the first quarter of 2025 compared to the same period last year, driven by higher commercial revenue including new TRYNGOLZA product revenue following approval in late December and higher SPINRAZA and WAINUA royalty revenue   1 • Operating expenses increased slightly in the first quarter of 2025 driven by commercialization efforts for TRYNGOLZA , donidalorsen and WAINUA   • Increased 2025 financial guidance reflects recent successful licensing transactions:     Full Year 2025 Guidance Previous Guidance New Guidance Revenue >$600 million $725-750 million Operating loss on a non-GAAP basis <$495 million <$375 million Cash, cash equivalents and short-term investments ~$1.7 billion ~$1.9 billion Recent Highlights - Marketed Medicines   • TRYNGOLZA TM (olezarsen) , the first-ever treatment for adults living with familial chylomicronemia syndrome (FCS) as an adjunct to diet , generated net product sales of over $6 million in its first full quarter following approval in the U.S. on December 19, 2024   o Commercialization rights in countries outside of the U.S., Canada and China licensed to Sobi   o EU approval decision anticipated in H2:2025   • WAINUA TM (eplontersen) (WAINZUA in EU) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) achieved commercial and regulatory milestones:   o Generated sales of $39 million resulting in royalty revenue of $9 million in the first quarter of 2025   o Launch underway in numerous countries, including the EU following approval by the European Commission (EC);