Ionis Pharmaceuticals announces 2024 expected milestones

Ionis Pharmaceuticals announced highlights from the Company’s 2023 achievements and previewed a number of important milestones expected in 2024. In 2023, Ionis made significant progress in advancing a steady cadence of medicines for people with serious diseases, including the U.S. approval of WAINUA for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis and the accelerated approval of QALSODY for SOD-1 amyotrophic lateral sclerosis. 2024 Anticipated Highlights Include: Continued progress with WAINUA, Ionis’ first co-commercialized medicine in collaboration with AstraZeneca in the U.S.: U.S. launch for ATTRv-PN in January; European Medicines Agency approval decision; additional country approvals; Additional regulatory filings for ATTRv-PN; Continuing the fully enrolled, landmark CARDIO-TTRansform trial in ATTR-cardiomyopathy, with data expected as early as 2025. Advancing olezarsen, our first anticipated independent launch: Detailed Phase 3 FCS results presentation; U.S. Food and Drug Administration regulatory filing in FCS, potential approval decision and U.S. launch; EMA filing for FCS; Completion of enrollment in severe hypertriglyceridemia Phase 3 studies, with data expected in 2025. Pivotal results and potential regulatory filing for investigational donidalorsen, our second anticipated independent launch: Phase 3 OASIS-HAE in first quarter and OASIS-Plus switch results by mid-year; Potential U.S. regulatory filing in hereditary angioedema. Advancing additional independent investigational medicines: Ionis expects to have six independent neurology programs in clinical development by year-end 2024, including new programs in rare pediatric diseases and severe dementias, which currently have no approved therapies; Phase 2 results for ION224 in nonalcoholic steatohepatitis anticipated. Continued progress with key partnered programs, including: QALSODY: EU SOD1-ALS CHMP and approval decision; ION582: Phase 1/2 clinical results in Angelman syndrome; ION541: Phase 2 results in ALS; IONIS-FB-LRx: Phase 2 results in IGA nephropathy and geographic atrophy, a serious eye disease.