Ionis Pharmaceuticals, Inc.

Ionis partner Roche announces new Phase 3 data showing sefaxersen significantly reduced proteinuria in people with IgA nephropathy (IgAN)

Ionis partner Roche reported Phase 3 IMAgINATION data showing sefaxersen achieved a statistically significant 43.1% reduction in proteinuria versus placebo at 37 weeks in adults with IgA nephropathy (P<0.0001). Sefaxersen, a once-monthly subcutaneous antisense inhibitor of complement factor B licensed from Ionis, was well tolerated with no new safety signals, and effects were consistent across all prespecified subgroups. Ionis is eligible for regulatory and sales milestone payments plus tiered royalties on net sales, so the positive readout advances a royalty-bearing future revenue stream while the study continues to a week-105 eGFR endpoint.

•9d ago

Ionis ranked #1 on Science’s Top Employers list 

Ionis Pharmaceuticals rose from #2 to #1 on Science's annual Top Employers survey of the biotech and pharmaceutical industries. The rankings were based on roughly 5,100 survey responses rating companies across 24 characteristics including corporate image, leadership, and work culture. The recognition is a corporate-image milestone with no direct financial or pipeline impact, though it may support talent retention for the RNA-targeted medicines pioneer.

•10d ago

Ionis partner GSK announces Hibsago (bepirovirsen) approved in the U.S. for the treatment of chronic hepatitis B (CHB)

The FDA approved Hibsago (bepirovirsen), Ionis' partnered first-in-class antisense therapy for chronic hepatitis B, granted under priority review and Breakthrough Therapy Designation. Phase 3 B-Well data showed a 19% functional cure rate versus less than 1% for standard of care, rising to 26% in patients with HBsAg ≤1000 IU/ml. GSK licensed the drug from Ionis in 2019, and Ionis receives tiered royalties of 10-12% on net sales plus regulatory and sales milestones. The approval opens a U.S. market of roughly 1.7 million CHB patients with limited treatment options, with regulatory decisions in additional geographies expected in coming months.

•12d ago

TRYNGOLZA® (olezarsen) recommended for approval in the EU by CHMP for severe hypertriglyceridemia (sHTG)

Ionis announced that the EMA's CHMP has adopted a positive opinion recommending TRYNGOLZA (olezarsen) as an adjunct to diet, with other lipid-lowering therapies, for adults with severe hypertriglyceridemia in the EU. The recommendation came ahead of the previously anticipated timeline; if confirmed by the European Commission, TRYNGOLZA would be the first and only RNA-targeted therapy authorized in the EU for sHTG with triglycerides >=880 mg/dL, with a final EC decision expected in Q4 2026. The opinion is based on the pivotal Phase 3 CORE and CORE2 studies, which showed up to 72% placebo-adjusted triglyceride reduction at six months and an 85% reduction in adjudicated acute pancreatitis events over 12 months. Sobi holds exclusive rights to commercialize TRYNGOLZA outside the U.S. (excluding Canada and China), so EU revenue would flow to Ionis largely through royalties and milestones.

•16d ago

Ionis announces positive interim data from partner Roche’s Phase 3 IMAgINATION study in IgA nephropathy (IgAN)

Ionis and partner Roche announced positive prespecified interim results from the Phase 3 IMAgINATION study, with sefaxersen meeting its primary endpoint of statistically significant and clinically meaningful proteinuria reduction versus placebo at 37 weeks in IgA nephropathy. The safety profile was consistent with prior data with no new safety signals, and interim data will be presented at an upcoming medical meeting and shared with health authorities. Roche licensed sefaxersen from Ionis, which remains eligible for regulatory and sales milestone payments plus tiered royalties on net sales, so the readout supports the long-term royalty stream on the asset.

•1mo ago

Ionis announces positive topline results from Phase 3 FUSION study of ulefnersen marking significant milestone in advancing first potential disease modifying treatment for FUS-ALS

Ionis and Otsuka announced positive topline results from the Phase 3 FUSION study of ulefnersen in FUS-ALS, meeting the primary endpoint of functional impairment and survival with statistical significance (p=0.0005). Ulefnersen is the first treatment to target the underlying genetic cause of FUS-ALS and showed favorable safety, with most adverse events mild or moderate. Secondary endpoints including serum NfL also improved significantly. Otsuka plans to engage the FDA and other global health authorities on potential expedited submission pathways. Ionis licensed ulefnersen to Otsuka in 2024 and is eligible for regulatory and sales milestone payments plus tiered royalties on net sales.

•1mo ago

ZANVASTRO™ (zilganersen) approved by the FDA as the first and only disease modifying treatment for Alexander disease (AxD) in pediatric and adult patients

The FDA approved Ionis Pharmaceuticals' ZANVASTRO (zilganersen) as the first and only disease-modifying treatment for Alexander disease, an ultra-rare and often fatal neurological disorder, in pediatric and adult patients. Approval was based on the pivotal Phase 1-3 study of 54 patients, which met its primary endpoint with a statistically significant 33.3% least-square-mean benefit in gait speed versus control at Week 61 (p=0.041). Ionis also received a Rare Pediatric Disease Priority Review Voucher alongside the approval, and ZANVASTRO will be available in the U.S. in the coming weeks as the company's first independent launch from its neurology pipeline. Recordati holds exclusive ex-U.S. commercialization rights under a June 2026 license agreement, with regulatory submissions in Europe and Japan expected in 2027.

•1mo ago

Ionis to present new data strengthening leadership in the management of severe hypertriglyceridemia (sHTG) at the European Society of Cardiology Congress 2026

Ionis announced it will present new data at the ESC Congress 2026, including one-year results from an open-label extension study of its recently approved therapy TRYNGOLZA for severe hypertriglyceridemia. The data package will also feature Phase 1 results for ION775, a next-generation follow-on siRNA with potential for semiannual dosing, alongside detailed Phase 3 analyses for eplontersen in partnership with AstraZeneca. TRYNGOLZA secured FDA approval in June 2026 as the first treatment to reduce triglycerides and pancreatitis risk in sHTG patients, with a European indication extension currently under review.

•2mo ago

Ionis Partner Biogen Presents Phase 2 CELIA Data at AAIC Demonstrating Meaningful Clinical Outcomes and Robust Tau Reduction with Diranersen in Early Alzheimer’s Disease

Partner Biogen presented Phase 2 CELIA data at AAIC showing diranersen achieved robust tau reduction of 50–65% in CSF and slowed clinical decline by 26% on CDR-SB at the 60 mg dose in early Alzheimer's patients. Although the study did not meet its primary endpoint of dose response on CDR-SB, the strong biomarker and secondary efficacy signals provided Phase 2 proof of concept, prompting Biogen to advance the program into confirmatory Phase 3 development. The therapy was generally well tolerated with mostly mild or moderate adverse events, and Biogen holds the exclusive worldwide license to the Ionis-discovered asset.

•3mo ago

Ionis doses first participant in Phase 1-2 ASCEND study of ION337 in Dravet syndrome

Ionis Pharmaceuticals announced the dosing of the first participant in the Phase 1-2 ASCEND study of ION337 for Dravet syndrome. The open-label study will evaluate the safety and tolerability of ION337 in children aged 2 to 12, with dosing potentially occurring every six months using the company's NMA technology. ION337 has received Fast Track designation from the FDA and is Ionis' first wholly owned medicine developed with its advanced NMA platform.

•3mo ago

Ionis completes enrollment in pivotal cohort of the Phase 3 REVEAL study evaluating obudanersen in Angelman syndrome

Ionis completed enrollment in the pivotal cohort of its Phase 3 REVEAL study evaluating obudanersen in Angelman syndrome, randomizing 136 pediatric participants aged 2 to 18. Topline data from the study is anticipated in the second half of 2027, while the company expects to complete enrollment in an adult cohort by the third quarter of 2026. The company also plans to advance obudanersen into a separate Phase 3 CHAMPION study for patients with uniparental disomy or imprinting defect genotypes before the end of 2026.

•3mo ago

Ionis announces license agreement with Recordati for zilganersen in Alexander disease (AxD) in all countries outside the U.S.

Ionis granted Recordati exclusive rights to develop and commercialize zilganersen for Alexander disease outside the U.S., receiving $30 million upfront plus potential milestones and tiered royalties. Ionis retains sole commercial rights in the U.S., where the drug is under FDA review with a PDUFA target date of September 22, 2026. The agreement leverages Recordati's rare disease expertise for global markets while Ionis prepares for its first independent neurology launch in the U.S.

•4mo ago

TRYNGOLZA® (olezarsen) approved by the FDA as the first and only treatment to reduce triglycerides and the risk of acute pancreatitis in patients with severe hypertriglyceridemia (sHTG)

The FDA approved TRYNGOLZA (olezarsen) as the first and only treatment indicated to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia. Clinical data from the Phase 3 CORE and CORE2 studies demonstrated triglyceride reductions of up to 72% and a reduction in acute pancreatitis events of up to 91% compared to placebo. This approval marks Ionis' first independent commercial launch in a prevalent condition affecting more than 3 million people in the U.S., with product availability expected in July 2026.

•4mo ago

Ionis announces appointment of Ludwig Hantson, Ph.D. to Board of Directors

Ionis appointed Ludwig Hantson, Ph.D., former CEO of Alexion, to its Board of Directors effective immediately. The company is preparing for a steady cadence of new commercial launches, including two additional independent launches this year, as it scales into a fully integrated biotechnology company. This appointment coincides with the recent addition of Peter Reikes to the board and the retirement of long-serving directors Joseph Wender and Lynne Parshall.

•4mo ago

Ionis to present new analyses from CORE and CORE2 pivotal studies supporting olezarsen for severe hypertriglyceridemia (sHTG) at upcoming congresses

Ionis will present new analyses from its pivotal Phase 3 CORE and CORE2 studies for olezarsen at three major medical conferences in June. The presentations cover insights into patients with diabetes, pancreatitis risk across triglyceride levels, and efficacy in patients with versus without baseline fibrate use. The FDA is currently reviewing the marketing application for olezarsen under Priority Review, with a target action date of June 30, 2026.

•4mo ago

Ionis partner GSK announces bepirovirsen achieves unprecedented functional cure rates with potential to redefine treatment for chronic hepatitis B

GSK announced pivotal Phase 3 data for bepirovirsen, an investigational therapy for chronic hepatitis B licensed from Ionis. The pooled results from the B-Well 1 and B-Well 2 trials showed a 19% functional cure rate in the overall population and 26% in patients with lower viral activity, compared to 0% in the placebo group. The data met the primary endpoints with high statistical significance (p<0.001). In an exploratory analysis, 49% of recipients achieved a surface antigen level of ≤100 IU/mL one year after treatment ended, a marker linked to improved long-term outcomes. Bepirovirsen is under priority review by the FDA with Breakthrough and Fast Track Designations. GSK anticipates regulatory decisions in Q3 2026. Ionis is eligible for additional regulatory and sales milestone payments plus tiered royalties of 10-12% on net sales.

•5mo ago

Ionis partner Biogen announces topline results from Phase 2 CELIA study of diranersen (BIIB080): first study to show reduction in tau pathology and cognitive benefit in patients with early Alzheimer’s disease

Biogen, Ionis' partner, announced topline results from the Phase 2 CELIA study of diranersen, an investigational antisense oligonucleotide (ASO) therapy targeting tau in early Alzheimer’s disease. The study demonstrated robust reductions in tau pathology across all doses and cognitive benefit, particularly at the lowest dose of 60 mg every 24 weeks. Despite not meeting the primary endpoint, Biogen plans to advance diranersen to registrational development, indicating strong confidence in the drug's potential. The safety and tolerability profile of diranersen was generally consistent with the Phase 1b study, with a higher incidence of serious adverse events at the highest dose.

•5mo ago

Ionis partner GSK announces bepirovirsen accepted for Priority Review and granted Breakthrough Therapy Designation by U.S. FDA as a potential first-in-class medicine for chronic hepatitis B

Partner GSK announced that the FDA accepted the NDA for bepirovirsen for Priority Review and granted it Breakthrough Therapy designation, setting a PDUFA target action date of October 26, 2026. The submission is supported by Phase 3 B-Well trials showing statistically significant and clinically meaningful functional cure rates, positioning bepirovirsen as a potential first-in-class medicine for chronic hepatitis B. Ionis is eligible to receive tiered royalties of 10-12% on net sales of bepirovirsen, which is also under regulatory review in Europe, China, and Japan.

•6mo ago