Ionis Pharmaceuticals investor relations material

Ionis Pharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of RNA-targeted therapeutics for the treatment of patients with severe, rare diseases by leveraging its proprietary antisense technology platforms in the United States. The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA/volanesorsen an injection for the treatment of familial chylomicronemia syndrome and familial partial lipodystrophy. It also develops SPINRAZA that is in Phase 3 long-term open-label extension study; TEGSEDI, which is under Phase 3 open-label extension study; IONIS ANGPTL3-LRx that is in Phase 3 clinical trial to treat hypertriglyceridemia; AKCEA-TTR-LRx to treat transthyretin amyloidosis; IONIS APO(a)-LRx to prevent cardiovascular disease caused by elevated Lp(a).