Ionis Pharmaceuticals (IONS) investor relations material

Executive summary

• Entered 2026 with strong momentum, driven by successful launches of TRYNGOLZA and DAWNZERA, which generated $27M and $16M in Q1 2026 U.S. net sales, respectively.

• Robust late-stage pipeline with two additional independent launches (olezarsen for sHTG and zilganersen for Alexander disease) on track for 2026, and multiple regulatory milestones including PDUFA dates for both.

• Partnered pipeline advancing, with bepirovirsen for hepatitis B and major cardiovascular outcomes trials expected to drive future royalties.

• Positioned for continued growth with anticipated launches through 2028 and expanding global access.

Financial highlights

• Q1 2026 total revenue was $246.1M, up 87% year-over-year, driven by commercial growth and milestone payments.

• Commercial revenue rose 42% year-over-year, led by TRYNGOLZA ($27M) and DAWNZERA ($16M, up 125% sequentially).

• Net loss for Q1 2026 was $92.5M (GAAP), improved from $146.9M in Q1 2025; non-GAAP net loss was $50M.

• Operating expenses increased to $364M (GAAP), mainly due to commercialization and launch readiness.

• Ended Q1 with $1.9B in cash and short-term investments after repaying $633M in convertible notes.

Outlook and guidance

• 2026 total revenue guidance raised to $875–$900M, $75M above prior guidance, with a greater share from commercial revenue.

• TRYNGOLZA and DAWNZERA full-year sales expected at $100–$110M and $110–$120M, respectively.

• Non-GAAP operating loss projected at $425–$475M, a $75M improvement over previous guidance.

• Annual peak net sales guidance for olezarsen increased to over $3B for sHTG.

• Year-end cash balance expected to exceed $1.6B; on track for cash flow breakeven in 2028.