Ionis Pharmaceuticals (IONS) investor relations material

Introduction and purpose

• FDA approved TRYNGOLZA (olezarsen) as the first and only therapy to reduce triglycerides and prevent acute pancreatitis in adults with severe hypertriglyceridemia (sHTG, TG ≥500 mg/dL), as an adjunct to diet.

• TRYNGOLZA is self-administered monthly via autoinjector, available in 50 mg and 80 mg doses, and addresses a significant unmet need for over 3 million sHTG patients in the U.S.

Details of approval or decision

• Approved for both 50 mg and 80 mg doses, administered once monthly, with flat pricing at $40,000 WAC effective April 1.

• Label includes acute pancreatitis risk reduction, a first for any therapy in this indication, and is indicated as adjunct to diet for sHTG and familial chylomicronemia syndrome (FCS).

• Approval based on positive Phase 3 CORE and CORE2 studies showing significant triglyceride and pancreatitis event reduction.

Impact on industry and stakeholders

• TRYNGOLZA represents a new treatment paradigm and first-mover advantage, expected to become a multi-billion dollar medicine with projected peak sales over $3 billion and 2026 guidance of $100–110 million.

• Early launch targets high-risk sHTG patients, with a specialized field team and broad payer engagement for access, and full suite of patient support services including nutrition, injection training, insurance, and financial assistance.

• Broad payer access anticipated, with coverage expected for all patients above 500 mg/dL.