Ionis Pharmaceuticals Announces Positive Topline Results for HAE Treatment

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announced positive topline results for the Phase 3 OASIS-HAE study of donidalorsen in patients with hereditary angioedema (HAE). The study met its primary endpoint, showing a statistically significant reduction in the rate of HAE attacks in patients treated with donidalorsen every 4 weeks or every 8 weeks. The treatment demonstrated a favorable safety and tolerability profile with no serious adverse events reported. Ionis is preparing to submit a New Drug Application to the U.S. FDA and data will be presented at an upcoming medical congress.

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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.