Ionis Pharmaceuticals (IONS) investor relations material

Strategic evolution and portfolio transition

• Transitioned from early-stage R&D to a fully integrated commercial biotech, focusing on expanding technology platforms and building a wholly owned pipeline in cardiometabolic and neurology areas.

• Launched first two wholly owned products, TRYNGOLZA for FCS and DAWNZERA for HAE, with plans for two more independent launches in 2024, including severe hypertriglyceridemia (sHTG) and zilganersen for Alexander disease.

• Aims for cash flow breakeven by 2028, with consistent revenue growth and profitability thereafter.

Key upcoming catalysts and financial outlook

• Anticipates FDA approval for TRYNGOLZA in sHTG (PDUFA June 30) and zilganersen in September, with Angelman program phase III data expected next year.

• Revenue guidance for TRYNGOLZA is $100–110 million in 2024, with initial pricing headwinds offset by expected volume growth in the second half.

• Raised peak U.S. sales estimate for TRYNGOLZA to over $3 billion, driven by new $40,000 WAC price and robust demand research.

• Partnered pipeline, including SPINRAZA and upcoming HBV drug with GSK, acts as a revenue accelerator alongside wholly owned assets.

Commercial strategy and market access

• Field team of ~200 is fully trained and prepared for sHTG launch, leveraging experience from FCS launch.

• Implemented $40,000 WAC price for both FCS and sHTG to optimize payer access and embed in 2027 budget cycles.

• Expects rapid acceleration in TRYNGOLZA volume post-sHTG approval, with significant revenue growth projected for 2025.