- Price
- $44.12
- Fair value
- $84.00
- Discount
- 47.5%
- Uncertainty
- High
- LSEG rating
- 1.96
- Moat
- Narrow
- Next earnings
- 2026-10-29
- Next earnings is estimate
- Yes
- Company name
- Ionis Pharmaceuticals
- Sector
- Biotechnology
- About
- Ionis Pharmaceuticals discovers and develops RNA-targeted medicines, mostly antisense drugs that stop the production of a disease-causing protein. It sells three medicines on its own in the US: TRYNGOLZA (olezarsen) for very high triglycerides, DAWNZERA (donidalorsen) for hereditary angioedema, and ZANVASTRO (zilganersen) for Alexander disease, approved on September 3, 2026. It also earns royalties and milestone payments from partners that sell or develop its drugs, such as Biogen (SPINRAZA for spinal muscular atrophy), AstraZeneca (WAINUA for a hereditary nerve disease), GSK (bepirovirsen for chronic hepatitis B), Novartis and Roche. In Q2 2026, revenue was $268 million, and royalty revenue was $76 million. Ionis still loses money, because it spends on research and on new launches, and it targets cash flow breakeven in 2028.
- Thesis
- The market lowered its view of Ionis's pipeline after two large partnered heart trials failed in 2026. On July 9, 2026, AstraZeneca's eplontersen trial in transthyretin amyloid cardiomyopathy missed its primary endpoint. On September 4, 2026, Novartis said that pelacarsen lowered Lp(a) but did not reduce cardiovascular events in the HORIZON trial. Both trials were possible sources of large royalties. Investors now value Ionis mostly on its own launches, which are still small: TRYNGOLZA sales were $5 million in Q2 2026 after a price cut, and Ionis still has an operating loss. The market doubts that TRYNGOLZA can reach the multi-billion-dollar potential that management sees in severe hypertriglyceridemia, which the FDA approved in June 2026. For the gap to close, four things must be true. TRYNGOLZA sales must grow fast from Q3 2026 in severe hypertriglyceridemia, with the EU decision for this use in 2027. DAWNZERA must reach its 2026 guidance of $110 million to $120 million of sales. GSK must win approval for bepirovirsen in chronic hepatitis B, with an FDA target date of October 26, 2026, and Roche must file sefaxersen in IgA nephropathy after the positive Phase 3 interim data of September 2026. Ionis must stay on its path to cash flow breakeven in 2028. The bepirovirsen decision and the Q3 2026 report, expected at the end of October 2026, give the first evidence. If the launches grow, the gap can close over the next four to eight quarters. If TRYNGOLZA grows slowly, the market can keep a low value on the pipeline.
- Risks
- Ionis's value depends on new launches. TRYNGOLZA sales were only $5 million in Q2 2026, and its growth in severe hypertriglyceridemia is not yet proven. Other drugs that lower triglycerides can compete, and payers can limit use.
Clinical trials can fail. Two partnered Phase 3 heart trials, of eplontersen and pelacarsen, missed their primary endpoints in 2026. Results for obudanersen in Angelman syndrome are due in the second half of 2027, and other pipeline drugs also depend on trial results.
Ionis still loses money: its loss from operations was $220 million in the first half of 2026. It targets cash flow breakeven in 2028. If launches are slow, it can need more capital.
Partners decide on development and sales of many Ionis drugs. GSK's bepirovirsen needs approvals, with an FDA target date of October 26, 2026, and Roche's sefaxersen still needs its full two-year results. A partner can delay or stop a program.
Ionis sold part of its SPINRAZA and pelacarsen royalties to Royalty Pharma, so it keeps less of these royalties. SPINRAZA also faces competition from other treatments for spinal muscular atrophy.
- Balance sheet
- Cash: $2.1B
Debt: $1.3B
Net cash: $710M
Debt due within 12 months: $0.0M
Last debt due: 2030
Ionis has net cash. At June 30, 2026, it had $2,055 million of cash, cash equivalents and short-term investments, down from $2,677 million at December 31, 2025, mainly because it repaid its 0% convertible notes on April 1, 2026. Debt is the principal of two convertible note issues: $770.0 million of 0% notes due 2030 and $575.0 million of 1.75% notes due 2028. Ionis can settle conversions in cash, shares or both. Debt does not include a $562.6 million liability for the sale of future SPINRAZA and pelacarsen royalties to Royalty Pharma in 2023, which Ionis pays from those royalties and records at an effective interest rate of 12.0%. Ionis has no EBITDA to compare with debt, because it had an operating loss of $220 million in the first half of 2026. It targets cash flow breakeven in 2028. No debt is due within 12 months of June 30, 2026, and the first notes are due in 2028.
As of 2026-06-30.
- Checkpoints
- Oct 26, 2026: FDA decision on GSK's bepirovirsen in chronic hepatitis B. Pending. Watch: FDA approval on time passes; a rejection or a long delay fails.
Oct 29, 2026 (estimate): Q3 2026 results: first quarter of the TRYNGOLZA sHTG launch. Pending. Watch: Fast growth in TRYNGOLZA sales after the June approval and DAWNZERA on track for $110 million to $120 million pass; flat TRYNGOLZA sales or a cut to 2026 guidance fail.
Feb 2027 (estimate): Full-year 2026 results and 2027 outlook. Pending. Watch: A 2027 outlook with strong growth in product sales and a clear path to cash flow breakeven in 2028 passes; a later breakeven or slow launch growth fails.
- Recent events
- 2026-09-23: Ionis and Roche said that sefaxersen met its primary endpoint at a prespecified interim analysis of the Phase 3 IMAgINATION study in IgA nephropathy, with a significant reduction in proteinuria at 37 weeks. The study continues to measure kidney function at week 105.
2026-09-04: Novartis said that its Phase 3 Lp(a)HORIZON trial of pelacarsen did not meet its primary endpoint of fewer cardiovascular events, although pelacarsen lowered Lp(a) as in earlier studies. Ionis discovered pelacarsen and earns royalties from Novartis, and it sold part of these royalties to Royalty Pharma in 2023.
2026-09-03: The FDA approved ZANVASTRO (zilganersen) as the first disease-modifying treatment for Alexander disease, an ultra-rare neurological disorder. Ionis received a Priority Review Voucher with the approval. This is its first independent launch in neurology.
2026-07-29: Ionis reported Q2 2026 results. Revenue was $268 million, and the loss from operations was $102 million. TRYNGOLZA sales were $5 million, after a lower net price from April 1, 2026, and DAWNZERA sales were $26 million. Cash and short-term investments were $2.1 billion. Ionis said it is on track for its 2026 guidance and for cash flow breakeven in 2028.
2026-07-09: Ionis and AstraZeneca said that the Phase 3 CARDIO-TTRansform trial of eplontersen in transthyretin amyloid cardiomyopathy missed its primary endpoint of cardiovascular death and recurrent cardiovascular events. A prespecified subgroup on eplontersen alone showed a nominally significant result.
2026-06-24: The FDA approved TRYNGOLZA to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia. This is Ionis's first independent launch in a common condition.
2026-05-28: GSK reported that bepirovirsen gave a functional cure rate of 19% in chronic hepatitis B in the pivotal B-Well studies, against 0% with standard of care. In Q2 2026, Ionis and GSK amended their agreement, with higher milestones and royalty terms that exclude China, Hong Kong and Macau.