Ionis Pharmaceuticals, Inc. develops RNA-targeted medicines and reports news around marketed medicines and a pipeline in neurology, cardiometabolic disease and other high-need areas. Company updates commonly address commercial performance for TRYNGOLZA, financial results, R&D revenue from partnerships, and progress across antisense oligonucleotide programs.
Clinical and regulatory news also covers programs such as olezarsen for severe hypertriglyceridemia, zilganersen for Alexander disease, and partner-developed bepirovirsen for chronic hepatitis B. Governance and investor-relations releases include annual meeting materials, board composition changes, and healthcare conference presentations.
Ionis (Nasdaq: IONS) announced management will present fireside chats at three investor conferences in May–June 2026: Bank of America Health Care (May 12), RBC Capital Markets Global Healthcare (May 19), and Goldman Sachs Global Healthcare (June 10).
Live webcasts and replays will be available on the Investors & Media section of the Ionis website; replays posted within 48 hours and archived for a limited time.
Ionis (Nasdaq: IONS) reported Q1 2026 revenue of $246M versus $132M a year earlier (an 87% increase). Q1 product sales: TRYNGOLZA $27M, DAWNZERA $16M; R&D revenue $138M including approximately $95M in milestone payments. Operating expenses were $364M and loss from operations was $(118)M. Cash and short-term investments were $1.9B after using a $633M escrow for convertible-note maturity. Company raised 2026 revenue guidance to $875–900M and set TRYNGOLZA/DAWNZERA sales targets of $100–110M and $110–120M, respectively.
Ionis (Nasdaq: IONS) partner GSK announced the FDA accepted the NDA for bepirovirsen for Priority Review, granted Breakthrough Therapy designation, and set a PDUFA date of October 26, 2026.
Regulatory action follows Phase 3 B-Well 1 and 2 trials reporting statistically significant, clinically meaningful functional cure rates versus standard of care; data to be presented at EASL 2026. Bepirovirsen is also under review with EMA, NMPA, and MHLW, and carries tiered royalties of 10–12% to Ionis under the GSK license.
Ionis (Nasdaq: IONS) reported positive pivotal study results for zilganersen in Alexander disease, with the trial meeting its primary endpoint and a PDUFA date set for September 22, 2026.
Key findings: 10MWT gait stabilization +33.3% versus control (p=0.041), GMFM-88 +22.9 points in ages 2–4 (nominal p=0.034), plasma GFAP −33.6% (nominal p=0.003). Safety was described as favorable with fewer serious TEAEs versus control.
Ionis (Nasdaq: IONS) will host a live webcast to discuss its first quarter 2026 financial results on Wednesday, April 29 at 8:30 a.m. ET.
According to Ionis, the webcast will highlight progress on key programs and will be available live and as a limited-time replay at the company investor events page.
Ionis (Nasdaq: IONS) said the FDA accepted the New Drug Application for zilganersen for Alexander disease and granted Priority Review with a PDUFA target date of September 22, 2026. The pivotal study showed a 33.3% least-square-mean gait-speed improvement (10MWT) at week 61 (p=0.0412) with favorable safety. Zilganersen previously received Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations. New pivotal-study data will be presented at the 2026 AAN annual meeting.
Ionis (Nasdaq: IONS) announced board changes effective June 4, 2026: B. Lynne Parshall and Joseph Wender will retire at term end, and Peter N. Reikes will rejoin the board.
Parshall and Wender are long-tenured directors; Reikes returns with recent roles at ARPA-H and the FDA and extensive healthcare finance experience.
Ionis (Nasdaq: IONS) will present new data supporting DAWNZERA (donidalorsen) for hereditary angioedema (HAE) at the 2026 AAAAI Annual Meeting in Philadelphia. Presentations include new analyses from the Phase 3 OASIS-HAE and OASISplus studies and one-year adolescent results.
Ionis will present eight poster presentations on Feb 27 and Mar 1, 2026, covering switching benefit, adherence, safety and efficacy in adolescents, patient-reported outcomes, predictors of disease control, unmet needs, and severity assessment.
Ionis (Nasdaq: IONS) announced the FDA has accepted the sNDA for olezarsen in severe hypertriglyceridemia (sHTG) and granted Priority Review, with a PDUFA target action date of June 30, 2026. The filing is supported by Phase 3 CORE and CORE2 results.
In those studies, olezarsen showed up to a 72% placebo-adjusted triglyceride reduction, an 85% reduction in acute pancreatitis events, and nearly 90% of treated patients reached triglycerides <500 mg/dL; safety and tolerability were described as favorable.
Ionis (Nasdaq: IONS) reported 2025 total revenue of $944 million (up from $705M in 2024), driven by commercial launches and a $280 million sapablursen upfront. TRYNGOLZA first-year net sales were $108 million. Cash and short-term investments were $2.7 billion at year-end.
2026 guidance: $800–$825M revenue, non-GAAP operating loss $500–$550M, and cash ~$1.6B.