On September 4, 2026, Ionis Pharmaceuticals announced that its partner Novartis reported the pelacarsen Phase 3 Lp(a)HORIZON study did not meet its primary endpoint of reducing cardiovascular events compared to placebo. The trial included 8,323 patients with elevated lipoprotein (a) and established cardiovascular disease. Although pelacarsen achieved substantially lower Lp(a) levels and demonstrated an acceptable safety profile, it failed to translate these biomarker reductions into clinical risk reduction. Ionis discovered and conducted early development of pelacarsen before Novartis licensed the rights in February 2019. The company operates in the biotechnology industry, focusing on RNA-targeted medicines for serious diseases.

On September 3, 2026, the U.S. FDA approved ZANVASTRO (zilganersen) for the treatment of Alexander disease in pediatric and adult patients. This marks the first and only disease-modifying treatment for this ultra-rare neurological disorder. The approval is based on pivotal study results demonstrating statistically significant stabilization of gait speed compared to control at Week 61. Ionis received a Priority Review Voucher with the approval. ZANVASTRO will be available in the U.S. in the coming weeks, while Recordati holds exclusive rights for commercialization outside the U.S. under a June 2026 license agreement. Ionis Pharmaceuticals is a biotechnology company focused on developing RNA-targeted medicines.

Ionis Pharmaceuticals reported second quarter 2026 financial results, posting total revenue of $268 million compared to $452 million in the prior year period. The decline was primarily driven by a significant drop in research and development revenue from collaborative agreements, which fell to $133 million from $337 million, excluding a one-time $280 million upfront payment received in Q2 2025 for sapablursen. Commercial revenue increased to $119 million from $103 million, supported by TRYNGOLZA net sales of $5 million and DAWNZERA net sales of $26 million. The company reported a net loss of $115 million, or $0.69 per diluted share, compared to net income of $124 million in the prior year. Operating expenses rose to $370 million due to commercialization investments for TRYNGOLZA and DAWNZERA. Ionis maintains a cash position of $2.1 billion and remains on track to achieve its 2026 financial guidance and cash flow breakeven by 2028. The company operates in the biotechnology industry, specializing in RNA-targeted medicines for neurology and cardiometabolic diseases.

Ionis Pharmaceuticals, Inc. is reaffirming its previously announced financial guidance for the fiscal year ending December 31, 2026, which remains unchanged from the guidance provided on April 29, 026. Ionis Pharmaceuticals, Inc. operates in the biotechnology industry and develops medicines for human therapeutics.

Ionis Pharmaceuticals, Inc. announced that its CARDIO-TTRansform Phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) failed to meet its primary efficacy endpoint of composite cardiovascular mortality and recurrent cardiovascular clinical events up to Week 140 compared to placebo. While a subgroup analysis of monotherapy patients showed a hazard ratio of 0.71, no treatment effect was observed in patients receiving stabilizer therapy at baseline. The company and AstraZeneca will share full data set results at the European Society of Cardiology Congress in August 2026. Ionis Pharmaceuticals, Inc. is a biotechnology company that develops RNA-targeted therapeutics.

Ionis Pharmaceuticals, Inc. announced that the U.S. FDA has approved TRYNGOLZA (olezarsen) as an adjunct to diet to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia. The approval was based on Phase 3 CORE and CORE2 studies, which showed TRYNGOLZA demonstrated rapid and consistent triglyceride control, lowering fasting triglyceride levels by up to 72% compared to placebo at six months and sustaining those reductions at 12 months. TRYNGOLZA is available in 50 mg or 80 mg monthly self-administered autoinjector doses. Ionis Pharmaceuticals, Inc. is a biotechnology company that develops and commercializes medicines for genetic diseases.