Ionis Pharmaceuticals Reports Positive Study Results for Zilganersen

The latest update is out from Ionis Pharmaceuticals ( (IONS) ).

On September 22, 2025, Ionis Pharmaceuticals announced positive results from a pivotal study of zilganersen, an investigational medicine for Alexander disease (AxD), a rare neurological condition. The study showed statistically significant stabilization in gait speed and favorable safety, marking the first time a treatment has demonstrated a disease-modifying impact in AxD. Ionis plans to submit a new drug application to the FDA in early 2026, potentially transforming the treatment landscape for AxD and reinforcing Ionis’ position in addressing severe neurological diseases.

The most recent analyst rating on (IONS) stock is a Buy with a $95.00 price target. To see the full list of analyst forecasts on Ionis Pharmaceuticals stock, see the IONS Stock Forecast page.

Spark’s Take on IONS Stock

According to Spark, TipRanks’ AI Analyst, IONS is a Neutral.

Ionis Pharmaceuticals’ overall stock score is driven by strong technical momentum and positive corporate events, including FDA approvals and promising study results. However, financial performance concerns, particularly around profitability and cash flow, moderate the score.

To see Spark’s full report on IONS stock, click here.

More about Ionis Pharmaceuticals

Ionis Pharmaceuticals is a pioneer in RNA-targeted medicines with a focus on neurology, cardiometabolic disease, and other areas of high patient need. The company is known for its innovative approaches in RNA therapies and gene editing, driven by a deep understanding of disease biology and advanced technology.

Average Trading Volume: 2,458,937

Technical Sentiment Signal: Buy

Current Market Cap: $9.72B

Find detailed analytics on IONS stock on TipRanks’ Stock Analysis page.