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 Pharmaceuticals (Nasdaq: IONS) announced two-year DAWNZERA data showing sustained reductions in hereditary angioedema (HAE) attacks ahead of ACAAI presentations.
In the OASISplus open-label extension, where participants knew their treatment, attack rates fell 95% from baseline across four- and eight-week dosing groups (n=83); 84% remained after two years. In the switch cohort, attack rates fell 71% from baseline on prior preventive therapy (n=64), with 80% remaining. A post hoc analysis found patients receiving eight-week dosing at extension entry maintained a 95% reduction over two years. No new safety signals or serious treatment-emergent adverse events related to DAWNZERA were observed; most treatment-emergent events were mild or moderate.
Ionis Pharmaceuticals (IONS) announced that management will participate in fireside chats at four upcoming investor conferences in November and December 2026.
The schedule includes UBS Global Biopharma on November 16, Jefferies Global Healthcare on November 18, Citi’s Global Healthcare on December 1 and Piper Sandler’s 38th Annual Healthcare Conference on December 2. Live webcasts will be available on the Ionis website, with replays posted within 48 hours and archived for a limited time.
Ionis Pharmaceuticals (Nasdaq: IONS) reported third-quarter 2026 revenue of $243 million, while lowering its full-year TRYNGOLZA sales outlook. Revenue rose from $157 million a year earlier, and the operating loss narrowed to $130 million from $160 million. Nine-month revenue reached $757 million versus $740 million, while the operating loss widened to $350 million from $167 million.
Ionis cut TRYNGOLZA sales guidance to approximately $50 million from $100-110 million, citing a slower launch ramp. It reaffirmed total revenue guidance of $875-900 million and DAWNZERA sales of $110-120 million. Cash and short-term investments fell to $1.9 billion from $2.7 billion at year-end. ZANVASTRO's Alexander disease launch is underway. Partnered programs delivered two positive Phase 3 readouts, but eplontersen missed its overall primary endpoint in its heart disease study.
Ionis Pharmaceuticals (IONS) will host a live webcast on November 9, 2026, to discuss its third quarter financial results. The webcast begins at 9:00 a.m. Eastern Time and will also cover progress on key programs. A replay will be available for a limited time on the company's investor relations website.
Ionis Pharmaceuticals (IONS) partner Roche reported Phase 3 sefaxersen results showing a 43.1% proteinuria reduction versus placebo in IgA nephropathy. The reduction in urine protein, an indicator of kidney damage, was statistically significant at week 37 (95% confidence interval: 32.5–52.0; P<0.0001). The ongoing IMAgINATION trial enrolled 459 adults receiving optimized standard of care.
Effects were consistent across all prespecified subgroups. Among patients with blood in their urine at baseline, more achieved resolution with sefaxersen than placebo. Treatment was well tolerated, with no new safety signals. The blinded study continues to assess kidney function at week 105. Roche plans to share the data with health authorities. Ionis is eligible for regulatory and sales milestone payments and tiered royalties on net sales under its licensing agreement with Roche.
Ionis Pharmaceuticals (IONS) ranked first in Science’s 2026 Top Employers survey of the biotechnology and pharmaceutical industries, up from second last year. The survey drew approximately 5,100 responses from Science readers and other invitees, who rated companies on 24 characteristics covering areas including leadership and work culture.
Ionis Pharmaceuticals (Nasdaq: IONS) announced that partner GSK received FDA approval for Hibsago to treat eligible adults with chronic hepatitis B. Hibsago (bepirovirsen) is approved with nucleos(t)ide analogue therapy, an antiviral treatment, in adults without cirrhosis who have received that therapy for at least six months, have suppressed viral DNA and surface antigen levels ≤3000 IU/ml confirmed by an FDA-authorized test.
Six-month treatment achieved 19% functional cure in pooled Phase 3 trials versus no participants receiving placebo; the rate was 26% in the ≤1000 IU/ml subgroup. Ionis is eligible for additional regulatory and sales milestones and 10-12% tiered royalties on net sales. Hibsago carries a boxed warning for liver flares during treatment and potentially life-threatening reductions in blood platelets. GSK anticipates regulatory decisions in additional geographies in the coming months.
Ionis Pharmaceuticals (Nasdaq: IONS) received a positive CHMP recommendation to extend TRYNGOLZA’s EU indication to adults with severe hypertriglyceridemia. The recommended use is alongside diet and other lipid-lowering therapies in adults with fasting triglycerides ≥880 mg/dL. European Commission approval remains pending; Ionis expects a final decision in the fourth quarter of 2026, following a recommendation earlier than previously anticipated.
The Phase 3 CORE and CORE2 studies met their primary endpoint, showing a statistically significant placebo-adjusted mean triglyceride reduction of up to 72% at six months, sustained through 12 months. A pooled analysis showed a statistically significant 85% reduction in adjudicated acute pancreatitis events over 12 months versus placebo.
Ionis Pharmaceuticals (IONS)/b) and partner Roche reported that investigational RNA‑targeted therapy met the primary endpoint in the Phase 3 IMAgINATION study in adults with primary IgA nephropathy (IgAN). At 37 weeks, sefaxersen achieved statistically significant and clinically meaningful reductions in proteinuria versus placebo, measured by 24‑hour urine protein‑to‑creatinine ratio (UPCR), a key marker of kidney damage. The once‑monthly subcutaneous therapy showed a safety and tolerability profile consistent with prior data, with no new safety signals.
The trial will remain blinded and continue to 105 weeks to assess change in kidney function via estimated glomerular filtration rate (eGFR). Sefaxersen is licensed to Roche, with Ionis having received upfront, license and development milestone payments and being eligible for further regulatory and sales milestones plus tiered royalties. Interim results will be presented at a future medical congress and shared with health authorities.
Ionis Pharmaceuticals (IONS) and Otsuka reported positive topline results from the Phase 3 FUSION trial of ulefnersen in people with FUS-ALS, a rare genetic form of ALS. The study met its primary endpoint, with ulefnersen showing a statistically significant improvement versus placebo on a joint rank analysis of time to death or permanent ventilation, time to rescue and change in ALSFRS-R score from baseline to Day 505 (p=0.0005), indicating potential to modify disease progression.
The trial also showed statistically significant benefits on key secondary endpoints, including serum neurofilament light chain and a composite of time to death, permanent ventilation, rescue or withdrawal due to disease progression. Ulefnersen, given by intrathecal injection, had a favorable safety and tolerability profile, with most adverse events mild or moderate. Otsuka plans urgent discussions with the FDA and other regulators on potential expedited submission pathways.