Ionis Pharmaceuticals Reports Strong 2025 Performance and Outlines Ambitious 2026 Pipeline Milestones
核心洞察
TRYNGOLZA (olezarsen) exceeded expectations with $105 million in preliminary 2025 U.S. net sales as the first FDA-approved treatment for familial chylomicronemia syndrome (搜索).
Ionis increased peak sales guidance for olezarsen to over $2 billion from $1 billion based on positive Phase 3 data showing up to 72% reduction in fasting triglycerides (搜索).
The company anticipates five Phase 3 readouts and four NDA submissions in 2026, including potential launches of olezarsen for severe hypertriglyceridemia (搜索) and zilganersen for Alexander disease (搜索).
Ionis Pharmaceuticals (搜索) announced strong commercial performance for 2025 and outlined an ambitious pipeline of regulatory milestones for 2026, positioning the RNA-targeted therapy pioneer for continued growth as it transitions into a fully commercial biotechnology company.
TRYNGOLZA Exceeds Commercial Expectations
TRYNGOLZA (olezarsen) generated $105 million in preliminary U.S. net product sales in 2025, outperforming expectations as the first FDA-approved treatment for familial chylomicronemia syndrome (搜索) (FCS). The strong commercial performance prompted Ionis to increase its annual peak net sales guidance for olezarsen to over $2 billion from the previous $1 billion estimate.
"2025 was a defining year for Ionis, as we successfully executed our first two independent launches as a commercial stage biotech company," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "We expect 2026 to be another transformative year, poised for two additional independent launches of breakthrough therapies."
The company has submitted a supplemental New Drug Application (sNDA) for olezarsen to treat severe hypertriglyceridemia (搜索) (sHTG), following receipt of Breakthrough Therapy Designation. In pivotal Phase 3 CORE and CORE2 studies, olezarsen demonstrated a highly statistically significant placebo-adjusted reduction of up to 72% in fasting triglycerides (搜索) and an 85% reduction in acute pancreatitis events with favorable safety and tolerability.
Expanding Neurology Portfolio
Ionis is preparing for the potential approval and launch of zilganersen for Alexander disease (搜索) (AxD), which would mark the first and only investigational medicine to demonstrate clinically meaningful and disease-modifying impact for this rare neurological condition. The company plans to submit an NDA in Q1 2026 and launch later in the year, having received Breakthrough Therapy Designation. A U.S. Expanded Access Program is currently underway.
The company continues to advance its Phase 3 REVEAL study of ION582 for Angelman syndrome (搜索), expecting to complete enrollment in 2026 with data anticipated in 2027. Multiple Phase 2 data readouts from the neurology pipeline are also planned.
DAWNZERA Shows Early Momentum
DAWNZERA (donidalorsen), the first RNA-targeted therapy for hereditary angioedema (搜索) (HAE), is showing positive early commercial momentum with prescriptions written across all patient segments and a growing number of repeat prescribers. The company expects European Medicines Agency approval and launch in Q1 2026.
Partnered Programs Advance
Ionis-discovered bepirovirsen, partnered with GSK for chronic hepatitis B (搜索), demonstrated positive topline results from pivotal Phase 3 B-Well 1 and B-Well 2 studies, showing a statistically significant and clinically meaningful functional cure rate. Regulatory submissions to health authorities worldwide are planned for 2026.
The Phase 3 Lp(a) HORIZON cardiovascular outcomes study of pelacarsen, an Ionis-discovered medicine partnered with Novartis for lipoprotein(a)-driven cardiovascular disease (搜索), is expected to report results in H1 2026, with a planned NDA submission to follow.
Additional Phase 3 readouts are anticipated from the CARDIO-TTRansform study of eplontersen for transthyretin-mediated amyloid cardiomyopathy (搜索) (partnered with AstraZeneca), the IMAGINATION study of sefaxersen for IgA nephropathy (搜索) (partnered with Roche), and the FUSION study of ulefnersen for Fused in Sarcoma Amyotrophic Lateral Sclerosis (搜索) (partnered with Otsuka (搜索)).
Financial Outlook
Monia indicated that with strong execution and multiple key catalysts expected, Ionis is well-positioned to deliver accelerating revenue growth to achieve cash flow breakeven in 2028. The company anticipates a total of five Phase 3 readouts and four NDA submissions in 2026.
The robust pipeline progress reflects Ionis' three-decade focus on RNA-targeted medicines, with current marketed medicines and a leading pipeline spanning neurology, cardiometabolic disease, and other areas of high patient need.