Ionis Pharmaceuticals (IONS) investor relations material

Executive summary

• Achieved FDA approval and strong early launch momentum for TRYNGOLZA in severe hypertriglyceridemia (sHTG) and familial chylomicronemia syndrome (FCS), with both 50mg and 80mg doses available within one week of approval and $32M in H1 2026 U.S. sales.

• DAWNZERA continues to gain share in the hereditary angioedema (HAE) prophylaxis market, with $42M in H1 2026 U.S. sales, driven by positive efficacy, safety, and patient-friendly features.

• Anticipated launch of zilganersen for Alexander disease remains on track, with commercial and regulatory preparations underway in the US and globally, and a PDUFA date in September 2026.

• Robust pipeline progress, including advancement of ION775 for sHTG, ION337 for Dravet syndrome, and positive developments in partnered programs such as bepirovirsen for hepatitis B.

• Maintains robust cash position to support ongoing investments in pipeline and commercialization.

Financial highlights

• Q2 2026 revenue: $268M; H1 2026 revenue: $514M, up 56% and 69% year-over-year, excluding prior year one-time payments.

• Commercial revenue up 15% in Q2 and 27% in H1 2026, mainly from DAWNZERA and TRYNGOLZA sales.

• DAWNZERA Q2 sales grew 63% sequentially to $26M; TRYNGOLZA generated $5M in Q2 and $32M in H1.

• Operating expenses increased due to commercialization and launch investments, with Q2 2026 operating loss at $(102)M GAAP, $(57)M non-GAAP; H1 2026 operating loss at $(220)M GAAP, $(131)M non-GAAP.

• Ended Q2 2026 with $2.1B in cash and short-term investments.

Outlook and guidance

• Full-year 2026 revenue guidance reaffirmed at $875M–$900M, with TRYNGOLZA sales expected at $100M–$110M and DAWNZERA at $110M–$120M.

• Non-GAAP operating loss projected at $425M–$475M for 2026, similar to 2025 after adjusting for prior one-time items.

• Year-end 2026 cash balance expected to exceed $1.6B; cash flow breakeven targeted for 2028.

• Anticipates multiple regulatory approvals and launches in H2 2026, including zilganersen and bepirovirsen.