Zilganersen

Ionis Pharmaceuticals has licensed sapablursen, an RNA-targeted medicine for polycythemia vera, to Ono Pharmaceutical in a deal worth up to $940 million including $280 million upfront.

Ionis Pharmaceuticals is set to enter a new chapter in 2025, focusing on advancing treatments for serious neurological diseases and cardiovascular conditions. The company has outlined plans for Phase 3 trials for ION582 for Angelman syndrome, zilganersen for Alexander disease, and ION464 for multiple system atrophy. Additionally, Ionis has received FDA approval for TRYNGOLZA™ (olezarsen) to reduce triglycerides in adults with familial chylomicronemia syndrome and for WAINUA™ (eplontersen) for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis.

The FDA has approved Ionis Pharmaceuticals' Tryngolza (olezarsen) as the first treatment for adults with familial chylomicronemia syndrome (FCS).

Ionis Pharmaceuticals reports positive progress with WAINUA launch and regulatory approvals in the UK and Europe for ATTRv-PN treatment.

Ionis Pharmaceuticals reports positive progress across its pipeline, highlighted by the U.S. launch of WAINUA and advancements in regulatory approvals.