Strong Overall Revenue Growth
Total 2025 revenue of $944M, up 34% year-over-year, driven by growing contributions from marketed medicines and R&D collaborations.
TRYNGOLZA Launch Momentum
TRYNGOLZA generated $108M in 2025 product sales with $50M in Q4, a 56% quarter-over-quarter increase; December was the strongest month of 2025 and payer mix is ~60% commercial / 40% government.
DAWNZERA Early Commercial Traction
DAWNZERA achieved $8M in 2025 product sales from initial launch months; free trial program saw strong participation with 100% conversion to paid therapy to date and reaffirmed peak sales potential >$500M.
Olezarsen Pivotal Outcomes — Breakthrough Data
Olezarsen Phase III (CORE/CORE2) showed up to 72% mean placebo-adjusted fasting triglyceride reductions at 6 months and an 85% reduction in adjudicated acute pancreatitis events; number needed to treat (NNT) of 4 in the highest-risk subgroup; received Breakthrough Therapy designation and sNDA submitted with launch-readiness targeted by June.
Zilganersen Phase III Success and NDA Submission
Zilganersen delivered positive Phase III results demonstrating the first disease-modifying benefit in Alexander disease; NDA submitted in January with anticipated approval and launch in the second half of 2026 and planned expanded access program underway.
Partner Pipeline Progress and Bepirovirsen Readout
Partnered programs produced notable readouts: Bepirovirsen Phase III reported unprecedented functional cure rates for chronic HBV; GSK preparing global regulatory submissions and plans commercialization later this year; multiple partner Phase III readouts expected in 2026 (pelacarsen midyear, CARDIO-TTRansform H2 2026).
Diversified Revenue Streams and Royalties
Commercial products generated $436M (46% of revenue); R&D collaborations generated $508M (54%); royalty revenues increased 11% to $285M, supported by SPINRAZA and growing WAINUA royalties.
Financial Position and Outlook
Projected year-end cash and investments of approximately $1.6B, guidance reflecting ~20% like-for-like revenue increase for 2026 after adjusting for a one-time $280M license fee, and a public goal to achieve cash flow breakeven by 2028.
Commercial Readiness and Field Expansion
Full field organization in place with ~200 field team members hired, trained and deployed; commercial preparations in progress for two more independent launches (sHTG olezarsen and zilganersen) in 2026.