Ionis Pharmaceuticals reported its fourth quarter and full-year 2024 financial results, highlighting the launch of TRYNGOLZA, its first independent medicine for familial chylomicronemia syndrome. The company is preparing for the potential launch of donidalorsen for hereditary angioedema in August 2025 and expects additional product launches in the coming years. WAINUA, approved for hereditary transthyretin-mediated amyloidosis, showed strong sequential growth, contributing to revenue exceeding 2024 guidance.

Revenue for 2024 totaled $705 million, compared to $788 million in 2023. Operating expenses increased slightly to $1.18 billion due to commercialization efforts, resulting in a net operating loss of $475 million. The company ended 2024 with $2.3 billion in cash, exceeding revised guidance.

TRYNGOLZA launched in the U.S. in December 2024 as the first treatment for familial chylomicronemia syndrome. WAINUA generated $85 million in sales, with royalty revenue of $20 million, and expanded to multiple countries, including the UK. SPINRAZA, used for spinal muscular atrophy, generated global sales of $1.6 billion, contributing $216 million in royalty revenue. QALSODY, treating SOD1-ALS, reached $32 million in sales and received marketing approval in China and Japan.

Ionis continues advancing its late-stage pipeline. Olezarsen is on track for Phase 3 data in severe hypertriglyceridemia in the second half of 2025. Donidalorsen, under regulatory review in the U.S. and EU, showed a 96% reduction in hereditary angioedema attacks in a Phase 2 extension study. ION582 is set to begin Phase 3 development for Angelman syndrome in early 2025. Ulefnersen remains in Phase 3 development for FUS-ALS under a licensing agreement with Otsuka.