Ionis Pharmaceuticals, Inc.

What they do

Ionis discovers and develops RNA-targeted medicines and now commercializes several of them. It independently sells TRYNGOLZA (olezarsen) and DAWNZERA (donidalorsen) in the U.S., while partners Biogen (SPINRAZA, QALSODY), AstraZeneca (WAINUA ex-U.S.), Sobi (Europe), Otsuka (DAWNZERA ex-U.S.) and PTC (Latin America) handle other regions or products. Revenue comes from product sales, royalties and collaboration/R&D payments.

Revenue drivers

• TRYNGOLZA (olezarsen) — Wholly owned, independently commercialized in the U.S. for FCS and, since June 2026, sHTG; generated $5M in Q2 2026 and $32M in H1 2026, with full-year 2026 guidance of $100-110M.
• DAWNZERA (donidalorsen) — Wholly owned, independently commercialized in the U.S. for HAE prophylaxis; second independent U.S. launch, with European/Asia-Pacific rights licensed to Otsuka.
• Partnered products and royalties — WAINUA with AstraZeneca (co-developed, co-commercialized in U.S.), SPINRAZA and QALSODY with Biogen, plus TEGSEDI and WAYLIVRA through Sobi and PTC; these provide product and royalty revenue.
• Collaboration and R&D revenue — Substantial R&D revenue from multiple partnerships; Q2 2026 total revenue of $268M and H1 2026 of $514M reflect these partnered programs.

Recent performance

Q2 2026 total revenue was $268M and H1 2026 revenue was $514M, up 56% and 69% respectively versus prior-year periods excluding a 2025 one-time sapablursen upfront payment. Q2 2026 GAAP operating loss was $102M versus $140M income in Q2 2025, and H1 2026 operating loss was $220M versus $7M loss. TRYNGOLZA U.S. net product sales were $5M in Q2 2026 and $32M in H1 2026, showing early sHTG launch momentum. The company ended Q2 2026 with $2.1B in cash and short-term investments. Full-year 2025 revenue was $943.7M with a net loss of $381.4M.

Strategy

Ionis is transitioning to a fully integrated commercial-stage company, independently launching TRYNGOLZA and DAWNZERA in the U.S. while expanding indications. It plans to independently launch zilganersen for Alexander disease in 2026, following an NDA submitted in January 2026. The company is investing in commercialization and launch preparations and advancing wholly owned and partnered Phase 3 programs. Management targets cash flow breakeven in 2028 and aims for growing product and royalty revenue.

Risks

• Commercial launch execution — Independent launches of TRYNGOLZA and DAWNZERA require building commercial infrastructure, and TRYNGOLZA faces a reduced net price effective April 1, 2026.
• Clinical and regulatory setbacks — The CARDIO-TTRansform study of eplontersen in ATTR-CM had an outcome that management referenced as a notable event, illustrating pipeline binary risk.
• Partner dependence — A large share of revenue comes from partners including Biogen, AstraZeneca, Otsuka, Sobi and PTC, so their commercial performance and decisions affect Ionis.
• Persistent losses and cash use — Ionis reported net losses each year 2022-2025 and operating cash outflows of $268.6M in 2025, with $1.33B long-term debt at year-end 2025.

Outlook

Management says it is on track to achieve 2026 financial guidance and expects multiple second-half milestones. These include potential approval and launch of zilganersen for Alexander disease, results from the pelacarsen Lp(a) HORIZON cardiovascular outcomes trial, and the global launch of bepirovirsen for chronic hepatitis B. Ionis maintains a goal of cash flow breakeven in 2028 and expects TRYNGOLZA full-year 2026 product sales of $100-110M.