Ionis Pharmaceuticals — Jan 12, 2026 release

Supplemental New Drug Application (sNDA), following receipt of Breakthrough Therapy Designation ■ Olezarsen achieved a highly statistically significant placebo-adjusted reduction of up to 72% in fasting triglycerides and an 85% reduction in acute pancreatitis events with favorable safety and tolerability in pivotal Phase 3 CORE and CORE2 studies o Increasing annual olezarsen peak net sales guidance to >$2 billion from >$1 billion based on strong product profile and positive Phase 3 data 1 • Continued early positive momentum with the launch of DAWNZERA™ (donidalorsen), the first RNA-targeted therapy for hereditary angioedema (HAE) o Prescriptions written for all patient segments; growing number of repeat prescribers o Expect European Medicines Agency (EMA) approval and launch in Q1 2026 • Potential approval and launch of zilganersen for Alexander disease (AxD), the first and only investigational medicine to demonstrate clinically meaningful and disease-modifying impact o U.S. Expanded Access Program (EAP) underway o Planned NDA submission in Q1 2026 and launch later this year; received Breakthrough Therapy Designation o First anticipated launch from Ionis owned neurology portfolio • Continued progress of Phase 3 REVEAL study of ION582, a promising investigational medicine for Angelman syndrome (AS) o Expect to complete enrollment in 2026 with data expected in 2027 • Multiple Phase 2 data readouts from neurology pipel