Ionis cuts 2026 TRYNGOLZA outlook to about $50M
TRYNGOLZA’s slower-than-anticipated launch ramp is reflected in a lower sales target, while broader 2026 revenue, operating loss and cash guidance remains unchanged.
Sentiment and the balance of points
Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.
Form Type
8-K
Rhea-AI Filing Summary
Ionis Pharmaceuticals reported third-quarter 2026 revenue of $243 million, compared with $157 million a year earlier, and a GAAP net loss of $110 million versus $129 million. Nine-month GAAP net loss was $317 million, compared with $152 million in 2025.
Ionis lowered full-year TRYNGOLZA product-sales guidance to approximately $50 million from $100–110 million, citing a more gradual launch ramp than anticipated; U.S. net product sales were $9 million for the quarter and $41 million for nine months. DAWNZERA guidance remains $110–120 million, and 2026 total revenue guidance of $875–900 million, non-GAAP operating loss guidance of $425–475 million, and cash, cash equivalents and short-term investments guidance above $1.6 billion were reaffirmed.
Cash, cash equivalents and short-term investments were $1.884 billion at September 30, 2026, compared with $2.677 billion at December 31, 2025; Ionis attributed the decrease primarily to repayment of 0% convertible notes on April 1, 2026 and cash used to fund operations. ZANVASTRO’s U.S. launch is underway, with first patients dosed.
2 points · 0 major0 major · 3 points
How this balance works
Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.
It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.
Rhea-AI Sentiment measures something else, the tone of the wording.
Hollow bars mark forward-looking points. How the balance works
Positive
- Moderate pointQ3 revenue increased 55%, excluding the one-time second-quarter 2025 sapablursen upfront payment.
- Moderate pointThird-quarter GAAP net loss narrowed to $110 million from $129 million.
Negative
- Moderate point. Forward-looking: it has not happened yet and may not happen.TRYNGOLZA 2026 sales guidance fell to approximately $50 million from $100–110 million.
- Moderate pointNine-month GAAP net loss rose to $317 million from $152 million.
- Moderate pointNine-month GAAP operating loss rose to $350 million from $167 million.
Filing Explained
Ionis’s non-GAAP measures exclude non-cash equity-award compensation and related tax effects; for the quarter, non-GAAP operating loss was lower than GAAP loss, and non-GAAP net loss was lower than GAAP net loss.
8-K Event Classification
2 items: 2.02, 9.01
2 items
Item 2.02
Results of Operations and Financial Condition
Financial
Disclosure of earnings results, typically an earnings press release or preliminary financials.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, or exhibit attachments filed with this report.
Key Figures
Total revenue: $243 million
GAAP net loss: $110 million loss
GAAP net loss: $317 million loss
+5 more
8 metrics
Total revenue
$243 million
Three months ended September 30, 2026; $157 million in the same 2025 period.
GAAP net loss
$110 million loss
Three months ended September 30, 2026; $129 million loss in the same 2025 period.
GAAP net loss
$317 million loss
Nine months ended September 30, 2026; $152 million loss in the same 2025 period.
GAAP operating loss
$350 million loss
Nine months ended September 30, 2026; $167 million loss in the same 2025 period.
TRYNGOLZA product-sales guidance
Approximately $50 million
Full year 2026; previous guidance was $100–110 million.
TRYNGOLZA U.S. net product sales
$9 million
Three months ended September 30, 2026; $41 million for the nine months ended September 30, 2026.
DAWNZERA product-sales guidance
$110–120 million
Full year 2026 guidance.
Cash, cash equivalents and short-term investments
$1.884 billion
As of September 30, 2026; $2.677 billion as of December 31, 2025.
Key Terms
non-GAAP, collaborative agreement revenue, open-label extension study, proteinuria, +1 more
5 terms
non-GAAP financial
"operating expenses on a non-GAAP basis"
Non-GAAP refers to financial measures that companies use to show their earnings or performance without including certain expenses or income that are often added back to give a different picture. It matters because it can make a company's results look better or more favorable, but it may also hide important costs, so investors need to look at both GAAP (official rules) and non-GAAP numbers to get a full understanding.
collaborative agreement revenue financial
"Collaborative agreement revenue"
open-label extension study medical
"CORE and CORE2 open-label extension study"
An open-label extension study is a follow-on clinical trial where participants continue receiving a treatment and both patients and researchers know what is being given (no blind or placebo). It gathers longer-term safety, tolerability and effectiveness information beyond the initial trial, like turning a short test drive into a longer lease to see how the product performs over time. Investors watch these studies for evidence that a treatment is safe and durable, which can affect regulatory approval, market adoption and sales forecasts.
proteinuria medical
"reduction in proteinuria relative to placebo at week 37"
Proteinuria is when abnormal amounts of protein are found in a person's urine. It can be a sign that the kidneys aren't working properly, since healthy kidneys usually prevent most proteins from passing into urine. Detecting proteinuria helps doctors identify and monitor kidney problems early.
primary endpoint medical
"The study met its primary endpoint"
The primary endpoint is the single main result a clinical study is designed to measure to decide if a treatment works, like the finish line in a race that tells you who won. Investors care because meeting or missing this goal drives regulatory decisions, future sales expectations and stock value — it turns trial data into a clear yes-or-no signal about a drug’s commercial prospects.
Earnings Snapshot
Total revenue: $243 million · Guidance included
Third quarter 2026
Total revenue
$243 million
Increased 55% year over year, excluding the one-time sapablursen upfront payment recognized in the second quarter of 2025.
GAAP net loss
$110 million loss
Compared with a $129 million loss in the third quarter of 2025.
GAAP operating loss
$130 million loss
Compared with a $160 million loss in the third quarter of 2025.
Guidance
Ionis reaffirmed 2026 total revenue guidance of $875–900 million, non-GAAP operating loss guidance of $425–475 million, and cash, cash equivalents and short-term investments guidance above $1.6 billion. TRYNGOLZA product-sales guidance was lowered to approximately $50 million from $100–110 million; DAWNZERA product-sales guidance remains $110–120 million.
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
How did IONS perform financially in the third quarter of 2026?
IONS reported $243 million in third-quarter revenue and a $110 million GAAP net loss. A year earlier, revenue was $157 million and GAAP net loss was $129 million.
AI-generated analysis. How Rhea-AI works. Not financial advice.
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of report (Date of earliest event reported): November 9, 2026
(Exact Name of Registrant as Specified in Charter)
(State or Other Jurisdiction of Incorporation)
| | | |
| (Commission File No.) | (IRS Employer Identification No.) |
(Address of Principal Executive Offices and Zip Code)
Registrant’s telephone number, including area
code: (760 ) 931-9200
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | | Trading symbol | | Name of each exchange on which registered |
| | “ |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (Section 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (Section 240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 2.02
Results of Operations and Financial Condition.
On November 9, 2026, Ionis Pharmaceuticals, Inc. (the “Company”) issued a press release announcing the Company’s financial results for the quarter ended September 30, 2026. In addition to disclosing results that are determined in accordance with Generally Accepted Accounting Principles (“GAAP”), the Company also discloses pro forma or non-GAAP results of operations, which are adjusted from GAAP to exclude non-cash compensation expense related to equity awards and the related tax effects. The Company is presenting pro forma information excluding non-cash compensation expense related to equity awards and the related tax effects because the Company believes it better enables financial statement users to assess and compare its historical performance and project its future operating results and cash flows. A copy of the release is furnished with this report as an exhibit pursuant to “Item 2.02. Results of Operations and Financial Condition” of Form 8-K in accordance with SEC Release Nos. 33-8216 and 34-47583.
The information in this Current Report on Form 8-K and the Exhibit attached hereto shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933 or the Exchange Act, regardless of any general incorporation language in such filing.
Item 9.01
Financial Statements and Exhibits.
(d)
Exhibits.
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Exhibit No.
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Description
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99.1
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Press Release dated November 9, 2026.
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104
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Cover Page Interactive Data File (embedded within the Inline XBRL document).
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SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.
| Ionis Pharmaceuticals, Inc. | ||
| Dated: November 9, 2026 | By: |
/s/ Patrick R. O’Neil |
| Patrick R. O’Neil | ||
| Executive Vice President, Chief Legal Officer and General Counsel | ||
Exhibit 99.1
Ionis reports third quarter 2026 financial results and highlights progress on key programs
– ZANVASTROTM (zilganersen) first approved medicine for Alexander disease; first independent launch underway from leading neurology pipeline –
– TRYNGOLZA® (olezarsen) 2026 product sales guidance lowered as sHTG launch ramps more gradually than expected reflecting early dynamics of establishing a new treatment paradigm –
– DAWNZERA® (donidalorsen) launch momentum continues; reaffirming 2026 product revenue guidance –
– Reaffirming 2026 total revenue, operating loss and cash guidance –
CARLSBAD, Calif., November 9, 2026 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results and provided key updates for the third quarter ended September 30, 2026.
“In 2026, we continue to advance innovative medicines for people with serious diseases, underscored by three approvals for groundbreaking first-in-class medicines, two of which are Ionis-owned,” said Brett P. Monia, Ph.D., chief executive officer of Ionis. “TRYNGOLZA, our first launch in a large indication, represents a new treatment paradigm for the underserved sHTG population. While initial sales progress has been more gradual than anticipated, we are encouraged by the underlying launch fundamentals. Importantly, we have identified clear opportunities to improve performance, and we remain confident in TRYNGOLZA's long-term growth potential. ZANVASTRO for people with Alexander disease marks our first independent neurology launch and lays the foundation for future launches from our leading neurology pipeline. In addition, our partnered pipeline continues to advance, with the recent approval of HIBSAGO for chronic hepatitis B and two additional positive Phase 3 outcomes creating near-term opportunities for additional potential approvals and revenue growth.”
Third Quarter 2026 Summary Financial Results(1):
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| September 30, | September 30, | |||||||||||||||
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2026
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2025
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2026
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2025
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(amounts in millions)
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||||||||||||||||
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Total revenue
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$ | 243 | $ | 157 | $ | 757 | $ | 740 | ||||||||
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Operating expenses
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$ | 373 | $ | 317 | $ | 1,107 | $ | 907 | ||||||||
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Operating expenses on a non-GAAP basis
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$ | 330 | $ | 286 | $ | 975 | $ | 816 | ||||||||
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Loss from operations
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$ | (130 | ) | $ | (160 | ) | $ | (350 | ) | $ | (167 | ) | ||||
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Loss from operations on a non-GAAP basis
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$ | (87 | ) | $ | (129 | ) | $ | (218 | ) | $ | (76 | ) | ||||
(1)
Reconciliation of GAAP to non-GAAP basis contained later in this release.
1
Third Quarter 2026 Financial Highlights
●
Revenue in the third quarter and nine months ended September 30, 2026 increased by 55% and 64% respectively, compared to the same periods last year, excluding the one-time sapablursen upfront payment recognized in the second quarter of 2025, driven by increased commercial revenue and substantial R&D revenue from multiple partnerships
●
Operating expenses for the third quarter and nine months ended September 30, 2026 were in line with expectations and increased year over year primarily from investments related to the commercialization efforts for TRYNGOLZA, DAWNZERA and ZANVASTRO
●
Ended the third quarter of 2026 with cash and short-term investments of $1.9 billion
Third Quarter 2026 Financial Results
“In the third quarter, we generated higher quarter-over-quarter commercial revenue and substantial R&D revenue from partnered medicines while continuing to invest in our innovative wholly owned pipeline. Our commercial revenue growth was driven by DAWNZERA, which continues to gain momentum, supporting our confidence in achieving full-year DAWNZERA product sales guidance. These results keep us on track to achieve our 2026 total revenue, operating loss and cash guidance,” said Elizabeth L. Hougen, chief financial officer of Ionis. “We are lowering our 2026 TRYNGOLZA product sales guidance to reflect a more gradual ramp than we anticipated. We remain confident in TRYNGOLZA’s potential to deliver sustained, long-term revenue growth and multi-billion-dollar peak sales. As we execute on our independent launches and advance the many additional opportunities across our rich pipeline, we remain committed to achieving our goal of sustained positive cash flow and long-term value creation.”
Recent Highlights - Wholly Owned Medicines
●
TRYNGOLZA® (olezarsen), the first FDA-approved treatment to reduce triglycerides and acute pancreatitis risk in adults with severe hypertriglyceridemia (sHTG) as an adjunct to diet
o
Generated U.S. net product sales of $9 million and $41 million in the third quarter and nine months ended September 30, 2026, respectively
■
Full year 2026 product sales guidance lowered to approximately $50 million, reflecting a more gradual launch ramp than anticipated as Ionis establishes a new treatment paradigm for people with sHTG
o
FCS launch outside the U.S. underway; received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) for sHTG (sHTG fasting triglyceride levels of ≥880 mg/dL [≥10 mmol/L]), paving the way to bring TRYNGOLZA to sHTG patients across Europe with a final approval decision expected in the fourth quarter of 2026
o
Presented positive results from the CORE and CORE2 open-label extension study (CORE-OLE) at the European Society of Cardiology (ESC) Congress in August 2026
●
DAWNZERA® (donidalorsen), the first and only RNA-targeted prophylactic therapy for hereditary angioedema (HAE) in patients 12 years of age and older
o
Generated U.S. net product sales of $37 million and $79 million in the third quarter and nine months ended September 30, 2026, respectively
o
On track to achieve full year 2026 product sales guidance of $110-120 million, representing a continuing driver of year-over-year revenue growth
2
o
Launch outside the U.S. underway, with new market approvals in Europe and Canada
●
ZANVASTROTM (zilganersen), the first and only disease-modifying treatment approved for children and adults with Alexander disease (AxD)
o
Launch underway with first patients dosed with commercial drug
o
Commercializing outside the U.S. with Recordati, assuming approval
●
Obudanersen, a potential first-in-class treatment for Angelman syndrome (AS)
o
Completed enrollment in the Phase 3 REVEAL study, with data expected in the second half of 2027
o
Initiated Phase 3 CHAMPION study in people with AS due to paternal uniparental disomy (UPD), or imprinting center defects (abbreviated as either ID or ICD)
●
ION775, an investigational siRNA for sHTG with the potential for semiannual or less frequent dosing, entered a Phase 2 study in people with sHTG and moderately elevated triglycerides (HTG), based on Phase 1 results showing robust triglyceride lowering with potential for extended dosing intervals
o
Presented and published positive results from the Phase 1 study at ESC and in the Journal of Clinical Lipidology in August 2026
Recent Highlights – Partnered Medicines
●
SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $461 million in the third quarter of 2026, an increase of 23% compared to the same period in 2025, resulting in royalty revenue of $69 million
●
WAINUA® (eplontersen) (WAINZUA in EU) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated global sales of $68 million in the third quarter of 2026, resulting in royalty revenue of $7 million
o
Global WAINUA ATTRv-PN launches continuing with additional submissions in progress to further expand global WAINUA access
o
Presented and published Phase 3 CARDIO-TTRansform study results of eplontersen for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) at ESC and in the New England Journal of Medicine in August 2026. Although the study missed the primary composite endpoint in the overall population, eplontersen demonstrated nominal significance in the pre-specified monotherapy population and a favorable safety and tolerability profile
●
HIBSAGOTM (bepirovirsen), first-in-class treatment for chronic hepatitis B (CHB)
o
Approved in the U.S., Japan and Canada
o
Under regulatory review in additional countries, including the EU and China
●
Sefaxersen, first RNA-targeted potential treatment for Immunoglobulin A Nephropathy (IgAN)
o
Presented positive results from the Phase 3 IMAgINATION study at ASN in October 2026. The study met its primary endpoint, demonstrating a clinically meaningful 43.1% reduction in proteinuria relative to placebo at week 37 (p<0.0001), with a safety profile consistent with previous data. Results will be shared with health authorities
3
●
Ulefnersen Phase 3 study in patients with Fused in Sarcoma (FUS) Amyotrophic Lateral Sclerosis (ALS) met its primary endpoint (p=0.0005) and demonstrated a favorable safety and tolerability profile; data to be shared with health authorities
●
Sapablursen, a potential treatment for polycythemia vera, received Orphan Drug Designations in the U.S., EU and Japan
Revenue
Ionis’ revenue was comprised of the following:
| Three months ended | Nine months ended | |||||||||||||||
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2026
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2025
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2026
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2025
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Revenue:
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(amounts in millions)
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|||||||||||||||
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Commercial revenue:
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||||||||||||||||
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Product sales, net:
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||||||||||||||||
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TRYNGOLZA sales, net
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$ | 9 | $ | 32 | $ | 41 | $ | 57 | ||||||||
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DAWNZERA sales, net
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37 | 1 | 79 | 1 | ||||||||||||
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ZANVASTRO sales, net
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2 | - | 2 | - | ||||||||||||
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Total product sales, net
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48 | 33 | 122 | 58 | ||||||||||||
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Royalty revenue:
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SPINRAZA royalties
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69 | 56 | 167 | 158 | ||||||||||||
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WAINUA royalties
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7 | 13 | 34 | 33 | ||||||||||||
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Other royalties
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5 | 7 | 14 | 19 | ||||||||||||
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Total royalty revenue
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81 | 76 | 215 | 210 | ||||||||||||
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Other commercial revenue
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7 | 7 | 26 | 26 | ||||||||||||
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Total commercial revenue
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136 | 116 | 363 | 294 | ||||||||||||
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Research and development revenue:
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||||||||||||||||
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Collaborative agreement revenue
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103 | 31 | 356 | 414 | ||||||||||||
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WAINUA joint development revenue
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4 | 10 | 38 | 32 | ||||||||||||
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Total research and development revenue
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107 | 41 | 394 | 446 | ||||||||||||
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Total revenue
|
$ | 243 | $ | 157 | $ | 757 | $ | 740 | ||||||||
Commercial revenue for the third quarter and nine months ended September 30, 2026 increased 18% and 23%, respectively, compared to the same periods in 2025. This increase was primarily driven by DAWNZERA product sales. Research and development revenue was also higher in the third quarter and nine months ended September 30, 2026, compared to the same periods in 2025, driven by multiple payments for programs advancing under the Company’s R&D collaborations, and excluding the $280 million upfront payment for the global license of sapablursen to Ono Pharmaceutical Co., Ltd. the Company received in the second quarter of 2025.
Operating Expenses
Operating expenses for the third quarter and nine months ended September 30, 2026 increased year over year, in line with expectations, primarily from investments related to the commercialization efforts for TRYNGOLZA, DAWNZERA and ZANVASTRO, with full-year expenses remaining on track for low-teens percentage growth year-over-year.
4
Balance Sheet
As of September 30, 2026, Ionis’ cash, cash equivalents and short-term investments decreased to $1.9 billion, compared to $2.7 billion on December 31, 2025, primarily due to repayment of the 0% convertible notes on April 1, 2026 and cash used to fund operations.
2026 Financial Guidance
Ionis reaffirms its 2026 total revenue, including DAWNZERA product sales, operating loss and cash guidance based on its year-to-date results and fourth quarter outlook. Following the first full quarter of the TRYNGOLZA launch as the first and only treatment to reduce triglycerides and acute pancreatitis in people living with severe hypertriglyceridemia, Ionis is lowering TRYNGOLZA 2026 product sales guidance to reflect a more gradual-than-anticipated near-term launch ramp, while remaining confident in TRYNGOLZA’s long-term growth potential.
| Full Year 2026 Guidance | Previous Guidance | New Guidance |
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Total Revenue
|
$875-900 million | Unchanged |
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TRYNGOLZA product sales, net
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$100-110 million | ~$50 million |
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DAWNZERA product sales, net
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$110-120 million | Unchanged |
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Operating loss on a non-GAAP basis
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$425-475 million | Unchanged |
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Cash, cash equivalents and short-term investments
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>$1.6 billion | Unchanged |
Webcast and Other Updates
Management will host a conference call and webcast to discuss Ionis’ third quarter 2026 results at 9:00 a.m. Eastern time on Monday, November 9, 2026. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address. To access the Company’s third quarter 2026 earnings slides click here.
Ionis’ Marketed Medicines
TRYNGOLZA® (olezarsen): TRYNGOLZA was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG ≥500 mg/dL) and as an adjunct to diet to reduce TG in adults with familial chylomicronemia syndrome (FCS). For more information about TRYNGOLZA, including the full U.S. Prescribing Information, visit tryngolza.com.
DAWNZERA® (donidalorsen): DAWNZERA was approved by the U.S. Food and Drug Administration for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. For more information about DAWNZERA, including the full U.S. Prescribing Information, visit dawnzera.com.
ZANVASTROTM (zilganersen): ZANVASTRO was approved by the U.S. Food and Drug Administration for the treatment of Alexander disease (AxD) in pediatric and adult patients. For more information about ZANVASTRO, including the full U.S. Prescribing Information, visit zanvastro.com.
5
For more information about SPINRAZA, WAINUA, HIBSAGO and QALSODY, visit spinraza.com, wainua.com, hibsago.com, qalsody.com, respectively. QALSODY is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients treated with QALSODY. Continued approval may be contingent upon verification of clinical benefit in confirmatory trial(s).
About Ionis Pharmaceuticals, Inc.
For more than three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis currently has marketed medicines and a leading pipeline in neurology, cardiometabolic disease and select areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram.
Ionis Forward-looking Statements
This press release includes forward-looking statements regarding Ionis’ business, financial guidance and the therapeutic and commercial potential of our commercial medicines, additional medicines in development, technologies and our expectations regarding development and regulatory milestones. Any statement describing Ionis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2025, and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company.
In this press release, unless the context requires otherwise, “Ionis,” “Company,” “we,” “our” and “us” all refer to Ionis Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. TRYNGOLZA® is a registered trademark of Ionis Pharmaceuticals, Inc. DAWNZERA® is a registered trademark of Ionis Pharmaceuticals, Inc. ZANVASTROTM is a trademark of Ionis Pharmaceuticals, Inc. AKCEATM is a trademark of Akcea Therapeutics, Inc. TEGSEDITM is a trademark of Akcea Therapeutics, Inc. WAYLIVRATM is a trademark of Akcea Therapeutics, Inc. SPINRAZA® and QALSODY® are registered trademarks of Biogen. WAINUA® is a registered trademark of the AstraZeneca group of companies. HIBSAGOTM is a trademark of the GSK group of companies.
Ionis Investor Contact:
D. Wade Walke, Ph.D.
IR@ionis.com
760-603-2331
Ionis Media Contact:
Hayley Soffer
media@ionis.com
760-603-4679
6
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
| Three months ended | Nine months ended | |||||||||||||||
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| 2026 | 2025 | 2026 | 2025 | |||||||||||||
| (unaudited) | (unaudited) | (unaudited) | (unaudited) | |||||||||||||
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Revenue:
|
||||||||||||||||
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Commercial revenue:
|
||||||||||||||||
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Product sales, net
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$ | 48 | $ | 33 | $ | 122 | $ | 58 | ||||||||
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Royalty revenue
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81 | 76 | 215 | 210 | ||||||||||||
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Other commercial revenue
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7 | 7 | 26 | 26 | ||||||||||||
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Total commercial revenue
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136 | 116 | 363 | 294 | ||||||||||||
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Research and development revenue:
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||||||||||||||||
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Collaborative agreement revenue
|
103 | 31 | 356 | 414 | ||||||||||||
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WAINUA joint development revenue
|
4 | 10 | 38 | 32 | ||||||||||||
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Total research and development revenue
|
107 | 41 | 394 | 446 | ||||||||||||
|
Total revenue
|
243 | 157 | 757 | 740 | ||||||||||||
|
Expenses:
|
||||||||||||||||
|
Cost of sales
|
3 | 2 | 9 | 8 | ||||||||||||
|
Research, development and patent
|
216 | 218 | 643 | 636 | ||||||||||||
|
Selling, general and administrative
|
154 | 97 | 455 | 263 | ||||||||||||
|
Total operating expenses
|
373 | 317 | 1,107 | 907 | ||||||||||||
|
Loss from operations
|
(130 | ) | (160 | ) | (350 | ) | (167 | ) | ||||||||
|
Other income (expense):
|
||||||||||||||||
|
Interest expense related to the sale of future royalties
|
(4 | ) | (18 | ) | (39 | ) | (55 | ) | ||||||||
|
Other income, net
|
24 | 49 | 73 | 70 | ||||||||||||
|
Loss before income tax benefit (expense)
|
(110 | ) | (129 | ) | (316 | ) | (152 | ) | ||||||||
|
Income tax benefit (expense)
|
- | - | (1 | ) | - | |||||||||||
|
Net loss
|
$ | (110 | ) | $ | (129 | ) | $ | (317 | ) | $ | (152 | ) | ||||
|
Basic and diluted net loss per share
|
$ | (0.66 | ) | $ | (0.80 | ) | $ | (1.92 | ) | $ | (0.95 | ) | ||||
|
Shares used in computing basic and diluted net loss per share
|
166 | 160 | 166 | 159 | ||||||||||||
7
IONIS PHARMACEUTICALS, INC.
Reconciliation of GAAP to Non-GAAP Basis:
Condensed Consolidated Operating Expenses, Loss From Operations, and Net Loss
(In Millions)
|
Three months ended
September 30, |
Nine months ended
September 30,
|
|||||||||||||||
| 2026 | 2025 |
2026
|
2025
| |||||||||||||
|
(unaudited)
|
||||||||||||||||
|
As reported cost of sales according to GAAP
|
$ | 3 | $ | 2 | $ | 9 | $ | 8 | ||||||||
|
Excluding compensation expense related to equity awards (1)
|
- | - | - | (1 | ) | |||||||||||
|
Non-GAAP cost of sales
|
$ | 3 | $ | 2 | $ | 9 | $ | 7 | ||||||||
|
As reported research, development and patent expenses according to GAAP
|
$ | 216 | $ | 218 | $ | 643 | $ | 636 | ||||||||
|
Excluding compensation expense related to equity awards
|
(24 | ) | (21 | ) | (74 | ) | (61 | ) | ||||||||
|
Non-GAAP research, development and patent expenses
|
$ | 192 | $ | 197 | $ | 569 | $ | 575 | ||||||||
|
As reported selling, general and administrative expenses according to GAAP
|
$ | 154 | $ | 97 | $ | 455 | $ | 263 | ||||||||
|
Excluding compensation expense related to equity awards
|
(19 | ) | (10 | ) | (58 | ) | (29 | ) | ||||||||
|
Non-GAAP selling, general and administrative expenses
|
$ | 135 | $ | 87 | $ | 397 | $ | 234 | ||||||||
|
As reported operating expenses according to GAAP
|
$ | 373 | $ | 317 | $ | 1,107 | $ | 907 | ||||||||
|
Excluding compensation expense related to equity awards
|
(43 | ) | (31 | ) | (132 | ) | (91 | ) | ||||||||
|
Non-GAAP operating expenses
|
$ | 330 | $ | 286 | $ | 975 | $ | 816 | ||||||||
|
As reported loss from operations according to GAAP
|
$ | (130 | ) | $ | (160 | ) | $ | (350 | ) | $ | (167 | ) | ||||
|
Excluding compensation expense related to equity awards
|
(43 | ) | (31 | ) | (132 | ) | (91 | ) | ||||||||
|
Non-GAAP loss from operations
|
$ | (87 | ) | $ | (129 | ) | $ | (218 | ) | $ | (76 | ) | ||||
|
As reported net loss according to GAAP
|
$ | (110 | ) | $ | (129 | ) | $ | (317 | ) | $ | (152 | ) | ||||
|
Excluding compensation expense related to equity awards and related tax effects
|
(43 | ) | (31 | ) | (132 | ) | (91 | ) | ||||||||
|
Non-GAAP net loss
|
$ | (67 | ) | $ | (98 | ) | $ | (185 | ) | $ | (61 | ) | ||||
| (1) | Amounts appear as zero due to rounding in millions. |
8
Reconciliation of GAAP to Non-GAAP Basis
As illustrated in the Selected Financial Information in this press release, non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net loss were adjusted from GAAP to exclude compensation expense related to equity awards and the related tax effects. Compensation expense related to equity awards are non-cash. These measures are provided as supplementary information and are not a substitute for financial measures calculated in accordance with GAAP. Ionis reports these non-GAAP results to better enable financial statement users to assess and compare its historical performance and project its future operating results and cash flows. Further, the presentation of Ionis’ non-GAAP results is consistent with how Ionis’ management internally evaluates the performance of its operations.
9
IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)
| September 30, | December 31, | |||||||
| 2026 | 2025 | |||||||
| (unaudited) | ||||||||
|
Assets:
|
||||||||
|
Cash, cash equivalents and short-term investments
|
$ | 1,884 | $ | 2,677 | ||||
|
Contracts receivable
|
88 | 66 | ||||||
|
Other current assets
|
389 | 247 | ||||||
|
Property, plant and equipment, net
|
162 | 123 | ||||||
|
Right-of-use assets
|
228 | 239 | ||||||
|
Other assets
|
162 | 172 | ||||||
|
Total assets
|
$ | 2,913 | $ | 3,524 | ||||
|
Liabilities and stockholders’ equity:
|
||||||||
|
Current portion of deferred contract revenue
|
$ | 70 | $ | 74 | ||||
|
0% convertible senior notes due April 2026, net – current
|
- | 432 | ||||||
|
Liability related to sale of future royalties, net – current
|
138 | 12 | ||||||
|
Other current liabilities
|
227 | 265 | ||||||
|
0% convertible senior notes due 2030, net
|
754 | 751 | ||||||
|
1.75% convertible senior notes due 2028, net
|
570 | 568 | ||||||
|
Liability related to sale of future royalties, net
|
428 | 551 | ||||||
|
Long-term lease liabilities
|
262 | 262 | ||||||
|
Long-term obligations, less current portion
|
21 | 28 | ||||||
|
Long-term deferred contract revenue
|
49 | 92 | ||||||
|
Total stockholders’ equity
|
394 | 489 | ||||||
|
Total liabilities and stockholders’ equity
|
$ | 2,913 | $ | 3,524 | ||||
10
Key 2026 and 2027 Value Driving Events(1)
|
New Product Launches
|
||||
|
Program
|
Indication
|
Location
|
2026
|
2027
|
|
DAWNZERA
|
HAE
|
EU
|
Achieved
|
|
|
TRYNGOLZA
|
sHTG
|
U.S.
|
Achieved
|
|
|
EU
|
•
|
|||
|
ZANVASTRO
|
Alexander disease
|
U.S.
|
Achieved
|
|
|
HIBSAGO
|
CHB
|
Japan
|
Achieved
|
|
|
U.S.
|
•
|
|||
|
EU
|
•
|
|||
|
China
|
•
|
|||
|
Sefaxersen
|
IgAN
|
U.S.
|
•
|
|
|
Ulefnersen
|
FUS-ALS
|
U.S.
|
•
|
|
| Regulatory Actions | ||||
|
Program
|
Indication
|
Regulatory Action
|
2026 | 2027 |
|
Donidalorsen
|
HAE
|
EU approval decision
|
Achieved
|
|
|
Olezarsen
|
sHTG
|
U.S. approval decision
|
Achieved
|
|
|
EU submission
|
Achieved
|
|||
|
EU approval decision
|
•
|
|||
|
Zilganersen
|
Alexander disease
|
U.S. submission
|
Achieved
|
|
|
U.S. approval decision
|
Achieved
|
|||
|
Nusinersen
(high dose) |
SMA
|
EU approval decision
|
Achieved
|
|
|
U.S. approval decision
|
Achieved
|
|||
|
Bepirovirsen
|
CHB
|
Regulatory submission(s)
|
Achieved
|
|
|
U.S. and Japan approval decisions
|
Achieved
|
|||
|
EU approval decision
|
• | |||
|
China approval decision
|
• | |||
|
Pelacarsen
|
Lp(a)- CVD
|
U.S. submission
|
Not Achieved | |
|
Sefaxersen
|
IgAN
|
U.S. submission
|
• | |
|
U.S. approval
|
• | |||
|
Ulefnersen
|
FUS-ALS
|
U.S. submission
|
• | |
|
U.S. approval
|
• | |||
11
|
Key Phase 3 Clinical Events
|
||||
|
Program
|
Indication
|
Event
|
2026
|
2027
|
|
Obudanersen
|
Angelman syndrome
|
REVEAL fully enrolled
|
Achieved
|
|
|
REVEAL data
|
•
|
|||
|
Bepirovirsen
|
CHB
|
B-Well data
|
Achieved
|
|
|
Pelacarsen
|
Lp(a)-CVD
|
Lp(a) HORIZON data
|
Not Achieved | |
|
Eplontersen
|
ATTR-CM
|
CARDIO-TTRansform data
|
Not Achieved
|
|
|
Sefaxersen
|
IgAN
|
IMAgINATION interim data
|
Achieved | |
|
IMAgINATION data
|
• | |||
|
Ulefnersen
|
FUS-ALS
|
FUSION data
|
Achieved | |
|
Salanersen
|
SMA
|
Phase 3 initiation
|
Achieved | |
|
Sapablursen
|
Polycythemia Vera
|
Phase 3 initiation
|
Achieved | |
|
Diranersen
|
Alzheimer’s disease
|
Phase 3 initiation
|
• | |
(1)
Timing expectations based on current assumptions and subject to change.
●
Indicates that the milestone is anticipated in the year indicated.
# # #
12