Ionis Pharmaceuticals (NASDAQ:IONS) stock climbed 5% following the announcement of encouraging topline results from a pivotal study of zilganersen in patients with Alexander disease, a rare and often fatal neurological disorder with no approved disease-modifying treatments.
The company reported that zilganersen 50 mg demonstrated statistically significant stabilization in gait speed compared to control, with a mean difference of 33.3% (p=0.0412) at week 61. The therapy also showed consistent benefits across key secondary endpoints, suggesting potential to slow disease progression.
This is the first time an investigational therapy has shown a positive disease-modifying effect in Alexander disease, which usually begins in early childhood and impacts essential functions such as walking, speaking, and swallowing.
“These unprecedented results highlight the potential of zilganersen to create new possibilities for people living with Alexander disease,” said Holly Kordasiewicz, senior vice president of neurology at Ionis.
The drug demonstrated a favorable safety profile, with most adverse events being mild or moderate. The occurrence of serious adverse events was numerically lower in the zilganersen group compared to the control arm.
Ionis plans to submit a new drug application to the U.S. Food and Drug Administration in the first quarter of 2026 and is exploring the possibility of initiating an Expanded Access Program in the U.S. Detailed data will be presented at an upcoming medical conference.
Ionis Pharmaceuticals stock price
This content is for informational purposes only and does not constitute financial, investment, or other professional advice. It should not be considered a recommendation to buy or sell any securities or financial instruments. All investments involve risk, including the potential loss of principal. Past performance is not indicative of future results. You should conduct your own research and consult with a qualified financial advisor before making any investment decisions.