Ionis Pharmaceuticals
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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.
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44th Annual J.P. Morgan Healthcare Conference summary13 Jan, 2026

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.

Olezarsen's NNT for high-risk SHTG patients?
What drives DAWNZERA's encouraging launch?
What makes Zilganersen's results unprecedented?
What drives the $2B+ peak revenue for SHTG olezarsen?
Eplontersen combo data: Market impact vs. stabilizers?
Pelacarsen Lp(a): Minimum efficacy for launch?
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Ionis Pharmaceuticals Inc. discovers and develops RNA-targeted therapeutics. It applies antisense technology platforms to develop treatments for a range of genetic and rare diseases. The company is headquartered in Carlsbad, California, and its shares are listed on the NASDAQ.

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