ION775

July 29, 2026: Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) reported 2Q26 results and highlighted multiple near-term catalysts across its wholly owned and partnered pipeline. TRYNGOLZA (olezarsen) saw early U.S. launch momentum following FDA approval in late June 2026 for severe hypertriglyceridemia (sHTG) (to reduce triglycerides and acute pancreatitis risk); EU sHTG marketing application is under review with a potential 2027 launch. CORE/CORE2 open-label extension (CORE-OLE) data and Phase 1 ION775 data are slated for presentation at the ESC Congress (Aug 2026). DAWNZERA (donidalorsen) continues its U.S. launch for hereditary angioedema prophylaxis with EU market approvals underway.

Regulatory upcoming: zilganersen (Alexander disease) is under FDA Priority Review with a PDUFA target action date of Sept. 22, 2026; Ionis signed a license agreement with Recordati for ex-U.S. development/commercialization. Bepirovirsen (chronic hepatitis B) is under regulatory review in multiple countries; U.S. Priority Review with PDUFA Oct. 26, 2026 and a global launch targeted this year.

Clinical updates: obudanersen (Angelman syndrome) completed enrollment in Phase 3 REVEAL; data expected 2H27. Partnered eplontersen (ATTR-CM) Phase 3 CARDIO-TTRansform missed the primary composite endpoint overall (nominal significance in monotherapy subgroup); results to be presented at ESC Aug 2026.