Ionis Pharmaceuticals (IONS) Goldman Sachs 45th Annual Global Healthcare Conference summary

Pipeline progress and late-stage programs

• Nine drugs in phase III development for 11 indications, expected to expand to at least 10 by year-end.

• Recent positive phase III readouts and approvals for WAINUA, olezarsen, donidalorsen, and pelacarsen.

• WAINUA approved for hereditary ATTR polyneuropathy, launched with AstraZeneca, with global expansion underway.

• Olezarsen showed strong phase III results in FCS, NDA submitted, sHTG trial ongoing.

• Donidalorsen phase III data positive, U.S. approval submission planned this year.

Commercialization and market strategy

• WAINUA co-commercialized with AstraZeneca; Ionis leads patient education, AstraZeneca leads U.S. sales.

• Early WAINUA launch metrics are meeting or exceeding expectations, with strong patient and provider resonance.

• Global expansion for WAINUA expected, with approvals anticipated in Europe, Canada, and other territories this year.

• Medicare Part D redesign caps out-of-pocket costs, providing a tailwind for WAINUA.

Clinical trial design and competitive positioning

• TTR cardiomyopathy phase III trial is the largest to date, designed for robust data and demographic shifts.

• Trial balanced between monotherapy and tafamidis baseline patients, with event rates tracking as projected.

• No significant confounding from tafamidis use; minimal drop-ins during the study.

• Base case for trial readout is 2026, but early readout scenarios are being considered.