Ionis Pharmaceuticals Inc.
Ionis Pharmaceuticals' Eplontersen Phase 3 Trial Fails to Meet Primary Endpoint
Summary
Ionis Pharmaceuticals, Inc. announced on July 9, 2026, that its CARDIO-TTRansform Phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet the primary efficacy endpoint of the composite outcome of cardiovascular (CV) mortality and recurrent CV events up to Week 140 compared with placebo. In a prespecified subgroup analysis of patients treated with eplontersen monotherapy, a nominally significant hazard ratio of 0.71 was observed on the composite outcome of CV mortality and recurrent CV events. However, in patients who were on stabilizer therapy at baseline, no treatment effect was observed. Multiple secondary, imaging, and biomarker analyses favored eplontersen versus placebo, with large and sustained reductions in transthyretin observed. Eplontersen was well tolerated, with a favorable safety profile consistent with previous results. Ionis and AstraZeneca will continue to analyze the full data set, and results will be shared with the scientific community at the European Society of Cardiology Congress in August 2026.
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About Ionis Pharmaceuticals Inc.
Ionis Pharmaceuticals Inc. is a biotechnology company focused on discovering and developing RNA-targeted therapeutics. The primary mission of Ionis is to harness its expertise in antisense oligonucleotide technology to address complex genetic disorders. Ionis is pioneering in the field of RNA-targeted drug discovery, aiming to treat a wide range of diseases, including neurological, cardiovascular, metabolic, and cancer-related conditions. Notably, the company has a robust pipeline of drugs in various stages of development and partnerships with leading pharmaceutical companies to expand the reach and impact of its therapeutic discoveries. As an innovative entity in the biotechnology sector, Ionis Pharmaceuticals plays a significant role in advancing medical treatments that target the underlying genetic causes of diseases, thereby offering new hope and potential solutions to patients worldwide. Its advancements contribute substantially to the growth and evolution of personalized medicine, emphasizing the importance of genetic research in modern healthcare.
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