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Form 8-K · Current Report · Filed 2025-09-22

Ionis Pharmaceuticals Announces Positive Topline Results for Zilganersen in Alexander Disease

Ionis Pharmaceuticals, Inc. announced positive topline results from the pivotal study of zilganersen in children and adults with Alexander disease (AxD), a rare and progressive neurological condition. Zilganersen demonstrated statistically significant and clinically meaningful stabilization in gait speed and favorable safety and tolerability. The company plans to submit a new drug application to the FDA in Q1 2026.

Filer
Ionis Pharmaceuticals Inc. IONS · NASDAQ
Filed
Accession
0001140361-25-035634
CIK
0000874015
Signal
High impactHealthcare

Items reported

Item 7.01

Regulation FD Disclosure

Regulation FD Disclosure
Item 8.01

Other Events

Regulatory Investigation
Item 9.01

Financial Statements and Exhibits

Exhibits Furnished

Market reaction

Close before filing

$61.01

Next session

+2.6%

Since filing

-15.1%

Latest close

$51.77

What happened, not what will happen — closing prices around the filing date.

Precedent

Previous filings from IONS reporting the same item, with the next session's move:

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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