Ionis Pharmaceuticals, Inc. announced highlights from the Company's 2025 achievements and provided additional updates on key milestones expected in 2026. The company will provide a business update at the 44th Annual J.P. Morgan Healthcare Conference on January 13 at 8:15am PT; the presentation is available on the Ionis website here. Preliminary TRYNGOLZA®?
(olezarsen) 2025 Full Year U.S. Net Sales; TRYNGOLZA outperformed expectations as the first U.S. Food and Drug Administration (FDA)-approved treatment for familial chylomicronemia syndrome (FCS), generating $105 million in preliminary U.S. net product sales in 2025 Anticipated Highlights: The company's Owned Programs: Potential approval and launch of olezarsen as the new standard of care for severe hypertriglyceridemia (sHTG); Submitted U.S. 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; Increasing annual olezarsen peak net sales guidance to > $2 billion from > $1 billion based on strong product profile and positive Phase 3 data; Continued early positive momentum with the launch of DAWNZERA?? (donidalorsen), the first RNA-targeted therapy for hereditary angioedema (HAE); Pres prescriptions written for all patient segments; growing number of repeat prescribers; expect European Medicines Agency (EMA) approval and launch in first quarter 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; U.S. Expanded Access Program (EAP) underway; Planned NDA submission in first quarter 2026 and launch later this year; received Breakthrough Therapy Designation; 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) expected to complete enrollment in 2026 with data expected in 2027; Multiple Phase 2 data readouts from neurology pipeline. Potential approval and launch of bepirovirsen, an Ionis-discovered investigational medicine for chronic hepatitis B (CHB) (GSK).
Results expected in first quarter 2026; Planned NDA submission; Results from Phase 3 CARDIO-TTRansform study of eplontersen, Ionis' co-commercialized investigational medicine with AstraZeneca for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) Results expected in H2 2026; Planned sNDA submission. Additional Phase 3 readouts from partnered programs: Results from IMAGINATION study of sefaxersen for IgA nephropathy (IgAN) (Roche). Results from FUSION study of ulefnersen for Fused in Sarcoma (FUS) Amyotrophic Lateral Sclerosis (ALS) (Otsuka); Multiple Phase 2 data readouts, including IONIS-MAPTRx (BIIB080) in Alzheimer's disease (AD) (Biogen) and new Phase 3 clinical trial initiations.
