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Ionis Pharmaceuticals (IONS) investor relations material
Ionis Pharmaceuticals 44th Annual J.P. Morgan Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Strategic evolution, positioning, and commercial progress
Transitioned from R&D to a fully integrated commercial-stage biotech, launching first two independent products, Tringulza and Donzera, in 2025.
Four approved medicines and six positive Phase III readouts, with 11 more in late-stage development.
Focused on delivering first- or best-in-class medicines in cardiometabolic and neurological diseases, with a deep late-stage pipeline and proven technology platform.
Strong financial foundation and disciplined expense management support ongoing investments and future launches.
Clear path to accelerating revenue growth, sustained positive cash flow, and substantial value creation, targeting cash flow break-even by 2028.
Product launches and pipeline highlights
Tringulza (Olezarsen) launched for FCS, showing strong efficacy, safety, and patient adherence, with $105 million in 2025 U.S. sales and EU launch underway.
Donzera approved for HAE prophylaxis, addressing unmet needs in efficacy, tolerability, and convenience, with a successful U.S. launch and European approval expected soon.
Olezarsen demonstrated up to 72% triglyceride reduction and 85% reduction in acute pancreatitis events in SHTG, with a supplemental NDA filed and launch expected in 2026.
Upgraded Olezarsen peak revenue guidance to over $2 billion based on strong market enthusiasm and clinical data.
Wainua and Qalsody continue to perform well, with Wainua showing strong uptake in hATTR polyneuropathy and anticipation for cardiomyopathy data.
Neurology and partner programs
Six wholly owned neurological medicines in clinical development, including Zolexarsen/Zilganersen for Alexander disease and ION582 for Angelman syndrome, both with breakthrough therapy designations.
Zolexarsen/Zilganersen showed unprecedented disease-modifying impact in Alexander disease, with NDA submission and launch planned for 2026.
ION582 for Angelman syndrome demonstrated consistent clinical improvements, with pivotal Phase III data expected in 2027.
Partner programs advancing, including Eplontersen for ATTR cardiomyopathy and Pelacarsen for Lp(a) cardiovascular disease, both with major Phase III data readouts in 2025.
Bepirovirsen for chronic HBV expected to be approved and launched by 2027, with additional partner launches planned.
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