Ionis Pharmaceuticals Inc

Latest Results (14.05.2026):

Ionis Pharmaceuticals Inc reported third quarter 2025 results: total revenue of $157 million (up 17% year-over-year), a GAAP net loss of $129 million (non-GAAP net loss $98 million), and an improved nine-month performance driven in part by a $280 million upfront licensing payment; the company raised 2025 guidance and highlighted multiple commercial launches and pivotal clinical wins.

Revenue and mix

Total revenue for Q3 2025 was $157 million vs. $134 million in Q3 2024 (+17%). Commercial revenue was $116 million (including TRYNGOLZA product sales of $32 million and total royalty revenue of $76 million). Nine‑month revenue was $740 million vs. $479 million (+55%), helped by a $280 million upfront license payment received in Q2 2025.

Profitability and expenses

Q3 2025 operating expenses (GAAP) were $317 million and $286 million on a non‑GAAP basis (adjusted to exclude equity‑award compensation). GAAP loss from operations was $160 million (non‑GAAP $129 million). Q3 GAAP net loss was $129 million (non‑GAAP net loss $98 million). Year‑to‑date results show material improvement versus 2024, driven by the upfront payment noted above.

Cash and balance sheet

As of the report, cash, cash equivalents and short‑term investments totaled $2.24 billion (compared with $2.30 billion a year earlier). Convertible notes and liability related to sale of future royalties remain on the balance sheet.

TRYNGOLZA commercial traction

TRYNGOLZA (olezarsen) generated $32 million in net product sales in Q3 2025 (its third full quarter), a nearly 70% increase over the prior quarter, and $57 million year‑to‑date. The EU approval was achieved and Sobi expects an EU launch in Q4 2025.

DAWNZERA launch

DAWNZERA (donidalorsen) received FDA approval on August 21, 2025; the U.S. launch is underway and described as off to an encouraging start. The product is under regulatory review in the EU.

Olezarsen (sHTG) pivotal results and regulatory path

Olezarsen showed highly significant Phase 3 CORE/CORE2 results in severe hypertriglyceridemia: up to a 72% placebo‑adjusted reduction in fasting triglycerides and an 85% reduction in acute pancreatitis events, with favorable safety. The sNDA filing is on track by year‑end 2025 and approval is anticipated in Q4 2026; detailed data to be presented at AHA on November 8, 2025.

Zilganersen and other neurology programs

Zilganersen achieved positive pivotal results in Alexander disease (statistically significant stabilization in gait speed); NDA submission is planned for Q1 2026 with potential approval in 2026. ION582 received Breakthrough Therapy designation for Angelman syndrome; Phase 3 enrollment expected to complete in 2026.

Partnered product royalties

Royalties continued to be a significant revenue driver: SPINRAZA royalties were $56 million in Q3 2025 (global SPINRAZA sales $374 million), and WAINUA royalties were $13 million in Q3 2025 (WAINUA sales $59 million in the quarter).

Guidance and outlook

Management increased 2025 financial guidance, citing strong year‑to‑date performance and fourth‑quarter momentum—notably TRYNGOLZA growth—and reiterated multiple near‑term value‑driving milestones including additional launches and regulatory filings into 2026.