Ionis Pharmaceuticals Reveals 'Unprecedented Results' From Rare Neurological Disease Trial

Core Insights - Ionis Pharmaceuticals released topline results from a pivotal study of zilganersen for Alexander disease, a rare neurological condition [1] - Zilganersen showed statistically significant stabilization in gait speed, with a mean difference of 33.3% compared to control at week 61 [2] - The drug demonstrated a positive disease-modifying impact, marking a first in AxD treatment [3] Efficacy and Safety - Zilganersen exhibited consistent benefits across key secondary endpoints, indicating slowed disease progression and stabilization [3] - Key secondary endpoints included various patient and clinician global impression scores, showing favorable trends [4] - The safety profile of zilganersen was favorable, with most adverse events being mild or moderate, and serious adverse events were numerically lower in the treatment group [4] Regulatory and Market Impact - Ionis plans to submit a new drug application to the FDA in Q1 2026 and is considering an Expanded Access Program in the U.S. [5] - Following the news, Ionis stock rose by 4.88% to $63.99 in premarket trading [5]