Ionis Pharmaceuticals announced positive pivotal trial results for zilganersen, its antisense oligonucleotide therapy targeting Alexander disease, a rare neurodegenerative disorder. The phase 3 study met its primary endpoint with statistically significant improvements in mobility measures, demonstrating disease-modifying potential. Ionis plans to submit an NDA in early 2026, highlighting advancement in a therapeutic area with no prior approved treatments. This success spotlights the promise of RNA-targeted therapies for devastating genetic neurological conditions.