Recent Updates — IONS
Partner Roche announced late-breaking oral presentation data from the ongoing Phase 3 IMAgINATION study, demonstrating that investigational antisense drug sefaxersen achieved a statistically significant and clinically meaningful 43.1% reduction in proteinuria relative to placebo at 37 weeks (P<0.0001). The treatment was well tolerated with no new safety signals. Ionis Pharmaceuticals is an RNA-targeted biotechnology company developing medicines for serious diseases.
The FDA has approved Hibsago (bepirovirsen) for the treatment of chronic hepatitis B in adults without cirrhosis who have suppressed HBV DNA and surface antigen levels ≤3000 IU/ml. GSK licensed bepirovirsen from Ionis in 2019 under a collaborative agreement that entitles Ionis to tiered royalties of 10-12% on net sales, along with potential milestone payments. The approval follows Phase 3 B-Well trials demonstrating functional cure rates of 19%. Ionis is a biotechnology company focused on discovering and developing RNA-targeted medicines for serious diseases.
Ionis Pharmaceuticals and Roche announced positive prespecified interim results from the Phase 3 IMAgINATION study evaluating sefaxersen in adults with primary IgA nephropathy. The study met its primary endpoint, demonstrating statistically significant and clinically meaningful reductions in proteinuria compared to placebo at 37 weeks, measured by the urine protein-to-creatinine ratio. Sefaxersen is a once-monthly subcutaneous injection targeting complement factor B, with a safety profile consistent with prior data and no new signals identified. The study will continue blinded to assess kidney function changes over two years via estimated glomerular filtration rate at week 105. Ionis Pharmaceuticals operates in the biotechnology industry, developing RNA-targeted medicines for serious diseases.
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.