Ionis’ Zilganersen Stabilizes Walking and Motor Skills in Alexander Disease Study
Ionis drug zilganersen showed promise for rare brain disease (Alexander disease), improving symptoms; FDA decision due Sept 2026.
Ionis drug zilganersen showed promise for rare brain disease (Alexander disease), improving symptoms; FDA decision due Sept 2026.
GSK and Ionis announce breakthrough results for bepirovirsen, a potential first-in-class treatment offering functional cure for chronic hepatitis B in just six months.
JPMorgan analyst upgrades Ionis Pharmaceuticals to Overweight, raising price target to $80 from $49, citing strong drug launches and path to profitability.
ION582, a new medicine for Angelman Syndrome, received FDA Breakthrough status! It aims to fix the root cause, showing early promise in improving communication, thinking, and movement for this severe neurological condition.
Ionis's olezarsen (Tryngolza) shows "unprecedented" results for severe hypertriglyceridemia, cutting triglycerides by up to 72% and acute pancreatitis events by 85% in Phase 3 trials—a first! Favorable safety. sNDA planned by year-end.
The FDA accepted non-invasive Liver Stiffness Measurement as a surrogate endpoint for MASH clinical trials, replacing biopsies. This key decision accelerates MASH drug development by enabling safer monitoring, speeding recruitment, and predicting patient outcomes.