ION582

Ionis Pharmaceuticals is moving forward with Phase 3 trials for ION582, an antisense therapy designed to unsilence the paternal UBE3A gene in Angelman syndrome patients, after Biogen declined to license the drug.

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 is initiating a Phase 3 trial (REVEAL) for ION582, a potential treatment for Angelman Syndrome, following positive Phase 2 results and FDA agreement.

Ionis Pharmaceuticals has finalized the design for a Phase III clinical trial, named REVEAL, to evaluate ION582 as a potential treatment for Angelman syndrome.