Welcome to our dedicated page for Ionis Pharmaceuticals news (Ticker: IONS), a resource for investors and traders seeking the latest updates and insights on Ionis Pharmaceuticals stock.
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 (IONS) reported that partner Novartis’ Phase 3 Lp(a)HORIZON trial of pelacarsen in 8,323 patients with elevated Lp(a) and established cardiovascular disease did not meet its primary endpoint of reducing major cardiovascular events versus placebo.
The primary composite endpoint covered cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and urgent coronary revascularization requiring hospitalization in patients with Lp(a) ≥70 mg/dL and a ≥90 mg/dL subpopulation. Despite the lack of cardiovascular risk reduction, pelacarsen again achieved substantially lower Lp(a) levels, consistent with earlier studies, and demonstrated an acceptable safety profile. Ionis discovered pelacarsen and conducted early development before Novartis licensed global development and commercialization rights in 2019.
Ionis’ chief executive officer said the outcome clarifies whether Lp(a) lowering can reduce cardiovascular risk in optimally treated patients and will inform future care. The company highlighted its continued focus on independent commercialization of its marketed medicines TRYNGOLZA, DAWNZERA and ZANVASTRO and on advancing its broader RNA-targeted pipeline.
Ionis Pharmaceuticals (IONS) received FDA approval for ZANVASTRO™ (zilganersen) on September 3, 2026, as the first and only disease‑modifying treatment for Alexander disease (AxD) in both pediatric and adult patients.
ZANVASTRO is an RNA‑targeted therapy that reduces glial fibrillary acidic protein (GFAP) and is given as a 50 mg intrathecal injection every 12 weeks. In a global Phase 1‑3 study (N=54; ages 1.5–53), the pivotal 50 mg cohort met its primary endpoint in patients ≥5 years, showing statistically significant and clinically meaningful stabilization of gait speed on the 10‑Meter Walk Test at Week 61 versus control (least square mean difference 33.3%, p=0.041). Patients aged 2–4 years showed improved gross motor function on the GMFM‑88 versus control.
The safety profile was favorable, with most adverse events mild or moderate and serious treatment‑emergent events less frequent than control, though aseptic meningitis and CSF white blood cell increases were observed. The approval also triggered a Rare Pediatric Disease Priority Review Voucher for Ionis. ZANVASTRO will be available in the U.S. in the coming weeks, while Recordati holds rights outside the U.S., with regulatory filings in Europe and Japan expected in 2027.
Ionis Pharmaceuticals (IONS) will participate in fireside chats at two upcoming investor conferences in September 2026.
Management is scheduled for the 21st Annual Wells Fargo Healthcare Conference on Thursday, September 10, 2026, and the Morgan Stanley 24th Annual Global Healthcare Conference on Wednesday, September 16, 2026. Live webcasts and subsequent replays will be available via the Investors & Media section of the Ionis website.
Ionis (Nasdaq: IONS) will present new data on managing severe hypertriglyceridemia (sHTG) at the European Society of Cardiology Congress 2026 in Munich, August 28–31. Key sessions feature one-year results from the open-label extension of TRYNGOLZA (olezarsen), recently approved in the U.S. to reduce triglycerides and acute pancreatitis risk in adults with sHTG, and Phase 1, 12‑month data for ION775, a long-acting investigational siRNA targeting APOC3 with potential for semiannual dosing. Additional presentations with AstraZeneca cover Phase 3 CARDIO-TTRansform data for eplontersen in transthyretin-mediated amyloid cardiomyopathy, including subgroup and meta-analyses. TRYNGOLZA’s European sHTG indication extension is under EMA review, with Sobi holding EU commercialization rights.
Ionis Pharmaceuticals (Nasdaq: IONS) announced that Frank Bennett, Ph.D., executive vice president and chief scientific officer and a founding scientist of the company, will retire effective February 2027. He joined Ionis in 1989 and has led research on antisense oligonucleotides across multiple therapeutic areas.
According to Ionis, Dr. Bennett contributed to the discovery of more than 50 drug candidates, with seven receiving regulatory approval worldwide, including SPINRAZA for spinal muscular atrophy and QALSODY for a genetic form of ALS. He will serve as a strategic consultant after retirement, and his responsibilities will transition to Eric Swayze, Ph.D., executive vice president, head of research.
Ionis (Nasdaq: IONS) reported second quarter 2026 revenue of $268 million and a loss from operations of $102 million, with net loss of $115 million. According to Ionis, revenue rose 56% year over year excluding a one-time sapablursen upfront payment in 2025, driven by higher commercial and R&D revenue.
Commercial revenue grew to $119 million, up 15%, led by DAWNZERA® net sales of $26 million and TRYNGOLZA® net sales of $5 million. For the first half of 2026, product sales reached $74 million. Ionis ended the quarter with $2.1 billion in cash and short-term investments and reaffirmed that it remains on track to meet its 2026 financial guidance and to reach cashflow breakeven in 2028.
On the pipeline, Ionis highlighted TRYNGOLZA’s early sHTG launch, DAWNZERA’s ongoing global rollout, NDA Priority Review for zilganersen in Alexander disease (PDUFA September 22, 2026), and bepirovirsen’s Priority Review in chronic hepatitis B (PDUFA October 26, 2026). The Phase 3 CARDIO‑TTRansform trial of eplontersen in ATTR‑CM did not meet its primary endpoint in the overall population.
Ionis (Nasdaq: IONS) will host a live webcast on Wednesday, July 29, 2026 at 8:30 a.m. Eastern Time to discuss its second quarter 2026 financial results and provide updates on key programs. The webcast and a limited-time replay will be available via the company’s investor relations website.
Ionis (Nasdaq: IONS) reported that partner Biogen presented full Phase 2 CELIA data at AAIC 2026 for diranersen, an investigational antisense therapy targeting tau in early Alzheimer’s disease. According to Ionis, diranersen showed efficacy across all studied doses at 18 months on multiple prespecified cognitive and composite endpoints.
The 60 mg every-six-months regimen (n=60) versus placebo (n=115) slowed clinical decline by 26% on CDR-SB, 42% on ADAS-Cog13, 50% on MMSE, 30% on modified iADRS, and 23% on ADCOMS, with most differences nominally statistically significant. Other dose regimens also showed slowing of decline, though the study’s primary endpoint—a dose response on CDR-SB at 18 months—was not met.
Diranersen produced mean 50–65% reductions in CSF total tau and reduced brain tau on PET across all doses, making it, per Ionis, the first tau-directed therapy in Phase 2 to lower both CSF tau and PET tau pathology. The drug was generally well tolerated, without anticipated amyloid-related imaging abnormalities, and more than 90% of completers entered the extension. Based on Phase 1b and Phase 2 data, Biogen plans to advance diranersen into confirmatory Phase 3 development.
Ionis Pharmaceuticals (Nasdaq: IONS) has dosed the first participant in ASCEND, an open-label Phase 1-2 study of ION337, an investigational RNA-targeted medicine for Dravet syndrome, a rare, severe childhood-onset neurological disorder. ASCEND will evaluate safety and tolerability in children aged 2–12 years with a clinical diagnosis of Dravet syndrome.
The study includes a 6‑month single ascending dose part, followed by a 24‑month multiple ascending dose part with intrathecal dosing of ION337 every 6 months, plus a 7‑month safety follow‑up. ION337 is Ionis’ first wholly owned medicine developed with its next‑generation N‑Methylacetamide (NMA) antisense technology, designed to enhance potency and support infrequent dosing.
Ionis (Nasdaq: IONS) and AstraZeneca reported that the Phase 3 CARDIO-TTRansform trial of eplontersen in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet its primary endpoint of CV mortality and recurrent CV events versus placebo.
A prespecified eplontersen monotherapy subgroup showed a nominally significant hazard ratio of 0.71, while no effect was seen in patients on stabilizers at baseline. Eplontersen achieved large, sustained TTR reductions and a favorable safety profile. Full data are planned for ESC Congress August 2026.