Ionis Pharmaceuticals Inc.
Ionis Pharmaceuticals Announces Positive Results for Zilganersen in Alexander Disease
Summary
Ionis Pharmaceuticals, Inc. announced additional positive results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive, and often fatal neurological condition. The study met its primary endpoint, showing statistically significant and clinically meaningful stabilization of gait speed in individuals aged 5 years and older. New data also suggest potential improvements in gross motor function in younger children (2-4 years of age). Key secondary endpoints consistently favored zilganersen over control, with more patients reporting improvement or no change and fewer reporting worsening. Zilganersen demonstrated a favorable safety and tolerability profile. The results will be presented at the 2026 American Academy of Neurology annual meeting, and the U.S. Food and Drug Administration has set a Prescription Drug User Fee Act action date of September 22, 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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