Pelacarsen (TQJ230) (AKCEA-APO(a)-LRx)

February 25, 2026: Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) reported Q4 and full-year 2025 results and outlined 2026 catalysts across its wholly owned and partnered pipeline. TRYNGOLZA (olezarsen) generated $50M Q4 and $108M FY2025 net product sales and is approved/launching in the EU for genetically confirmed familial chylomicronemia syndrome (FCS). Ionis submitted an sNDA for olezarsen in severe hypertriglyceridemia (sHTG) in the U.S.; the company is awaiting acceptance and plans a 2026 launch assuming approval; EU submission is also planned. DAWNZERA (donidalorsen) posted $7M Q4 net sales; it was approved in the EU in January and launched for routine prevention of hereditary angioedema (HAE) attacks (≥12 years), with one-year OASISplus open-label extension results published.

Zilganersen (Alexander disease): NDA submitted; U.S. approval decision anticipated in H2 2026; U.S. expanded access program underway.

Partnered programs: SPINRAZA (nusinersen) high-dose is under U.S. review with a PDUFA date of April 3, 2026 (EU approval achieved). Bepirovirsen (chronic hepatitis B) met the primary endpoint in Phase 3 B-Well 1 and 2 with a statistically significant, clinically meaningful functional cure rate; regulatory filings planned beginning Q1 2026 and EASL 2026 presentation planned (pending acceptance). Additional 2026 readouts/submissions highlighted include pelacarsen (Lp(a) HORIZON mid-2026), eplontersen CARDIO-TTRansform (2H 2026), and multiple Phase 2/3 milestones (e.g., obudanersen, sefaxersen, ulefnersen, BIIB080/IONIS-MAPT Rx, tominersen).