Ionis Pharmaceuticals (IONS) investor relations material

Strategic Vision and Growth Outlook

• Focused on becoming a fully integrated, commercial-stage biotech, prioritizing wholly owned pipeline and independent launches in neurology and cardiometabolic diseases, with a steady cadence of independent and partnered medicine launches targeting both rare and prevalent diseases.

• Achieved or exceeded all key milestones set six years ago, including building a robust pipeline, commercial organization, and expanding RNA-targeted drug discovery.

• Advancing a high-value, innovative pipeline with multiple late-stage and recently approved medicines, including TRYNGOLZA, DAWNZERA, and Olezarsen.

• Ongoing investment in technology platforms, including RNA-targeted medicines, siRNA, and delivery innovations to maintain leadership and expand therapeutic reach.

• Clear path to sustained positive cash flow by 2028, supported by strong financial resources (~$2B cash), disciplined expense management, and efficient capital structure.

Key Product and Clinical Developments

• TRYNGOLZA (olezarsen) approved for FCS, showing substantial triglyceride and pancreatitis reduction, strong early commercial uptake, and approved in the EU.

• Olazarsen demonstrated up to 72% mean placebo-adjusted triglyceride reduction and 85% reduction in acute pancreatitis in SHTG, with sNDA filing planned by year-end and launch expected in the second half of next year.

• DAWNZERA (donidalorsen) launched in the US for HAE, offering first RNA-targeted prophylactic with strong efficacy, tolerability, and convenient dosing; EU launch anticipated in 2026.

• Zilganersen pivotal data for Alexander disease showed disease stabilization and robust safety, with NDA filing and first neurology launch planned for next year.

• ION582 for Angelman syndrome showed positive long-term data and broad Phase III enrollment, targeting both pediatric and adult populations, with full enrollment expected in 2026.

Commercial and Financial Strategy

• Commercial infrastructure built to support current and future launches, with omnichannel education reaching over 30,000 HCPs and strong payer dynamics.

• FCS launch generated $19M in Q2 revenue, with full-year guidance of $75–80M; Donzara launch off to an encouraging start.

• Olazarsen positioned as a potential blockbuster, targeting a million high-risk SHTG patients initially, with pricing and access strategy under development.

• Financial discipline maintained, with $2B in cash, clear path to sustained positive cash flow by 2028, and peak annual revenue potential exceeding $5B from multiple products and royalties.

• Multiple blockbuster opportunities in the pipeline, with independent and partner launches expected to generate over $5B in combined annual peak revenue.