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Ionis Pharmaceuticals (NASDAQ: IONS) posts $268M Q2 revenue and loss

(Moderate)
(Neutral)
Form Type
8-K

Rhea-AI Filing Summary

Ionis Pharmaceuticals reported Q2 2026 total revenue of $268 million, down from $452 million a year earlier, as prior-year results included a $280 million upfront payment related to a global license of sapablursen. Commercial revenue grew to $119 million, up from $103 million, driven mainly by DAWNZERA product sales and royalties from SPINRAZA and WAINUA. Product sales reached $31 million, compared with $19 million in Q2 2025.

Operating expenses rose to $370 million from $312 million, reflecting investment in launches for TRYNGOLZA and DAWNZERA and launch preparations for zilganersen. Ionis posted a GAAP net loss of $115 million versus net income of $124 million a year earlier; on a non-GAAP basis, net loss was $70 million versus non-GAAP net income of $154 million. Cash, cash equivalents and short-term investments were $2,055 million at June 30, 2026, down from $2,677 million at December 31, 2025, primarily after repayment of 0% convertible notes.

The company highlighted U.S. approval and early launch momentum for TRYNGOLZA in severe hypertriglyceridemia, DAWNZERA’s growth in hereditary angioedema, completion of enrollment in the Phase 3 REVEAL Angelman syndrome trial, and stated it remains on track to achieve its 2026 financial guidance and a goal of cashflow breakeven in 2028.

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Filing Explained

The filing marks eplontersen’s CARDIO-TTRansform milestone as not achieved while several pipeline decisions and data events remain pending.

The filing records that eplontersen’s CARDIO-TTRansform data milestone was “Not Achieved”; this leaves that program milestone incomplete while other listed regulatory and clinical events remain pending or achieved.

Form 8-K reports specified material events, and this Item 2.02 filing furnishes the results release as Exhibit 99.1 rather than treating that release as filed under the Exchange Act.

The named pending resolution points are U.S. approval of zilganersen, Lp(a) HORIZON data for pelacarsen, and regulatory decisions for bepirovirsen, each shown as pending in the milestone tables.

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, and exhibit attachments filed with this report.
Total revenue Q2 2026 $268 million Three months ended June 30, 2026; total revenue versus $452 million in Q2 2025.
Commercial revenue Q2 2026 $119 million Commercial revenue for three months ended June 30, 2026; up from $103 million in 2025.
Product sales Q2 2026 $31 million Net product sales for three months ended June 30, 2026; versus $19 million in 2025.
GAAP net income (loss) Q2 2026 $(115 million) Net loss for three months ended June 30, 2026; versus $124 million net income in 2025.
Non-GAAP net income (loss) Q2 2026 $(70 million) Non-GAAP net loss for three months ended June 30, 2026; versus $154 million non-GAAP net income in 2025.
Non-GAAP operating expenses Q2 2026 $325 million Operating expenses on a non-GAAP basis for three months ended June 30, 2026.
Cash, cash equivalents and short-term investments $2,055 million Balance at June 30, 2026; down from $2,677 million at December 31, 2025.
0% convertible senior notes due April 2026 $432 million Current portion outstanding at December 31, 2025; repaid by June 30, 2026.
non-GAAP financial
"the Company also discloses pro forma or non-GAAP results of operations"
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.
severe hypertriglyceridemia medical
"severe hypertriglyceridemia (sHTG: TG ≥500 mg/dL)"
An extreme elevation of triglycerides, the fat particles carried in the blood, that can overwhelm the body’s ability to process them and sharply raise the risk of acute pancreatitis and other complications. Think of blood as a river and triglycerides as oil — when levels get too high the flow and nearby organs can be damaged. Investors care because it drives demand for treatments, affects clinical trial safety and regulatory review, and can influence healthcare costs, reimbursement and company valuations.
hereditary angioedema medical
"DAWNZERA was approved by the U.S. Food and Drug Administration for prophylaxis to prevent attacks of hereditary angioedema"
A rare inherited disorder that causes sudden, painful swelling under the skin or in internal tissues, including the airway, because a natural blood‑control protein is missing or not working. Attacks can be unpredictable and sometimes life‑threatening, so people often need ongoing medication or emergency treatment. For investors, hereditary angioedema represents a niche but stable market for specialized therapies, diagnostics, and emergency care solutions.
convertible senior notes financial
"0% convertible senior notes due April 2026, net"
Convertible senior notes are a type of loan that a company issues to investors, which can be turned into company shares later on. They are called "senior" because they are paid back before other debts if the company runs into trouble. This allows investors to earn interest like a loan but also have the chance to own part of the company if its value rises.
sale of future royalties financial
"Liability related to sale of future royalties, net"
Total revenue Q2 2026 $268 million $452 million in Q2 2025
Commercial revenue Q2 2026 $119 million $103 million in Q2 2025
GAAP net income (loss) Q2 2026 $(115 million) $124 million net income in Q2 2025
Non-GAAP net income (loss) Q2 2026 $(70 million) $154 million non-GAAP net income in Q2 2025
Guidance

Ionis stated it is on track to achieve its 2026 financial guidance and its goal of cashflow breakeven in 2028.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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FAQ

What were Ionis Pharmaceuticals (IONS) Q2 2026 revenue and net income?

Ionis reported Q2 2026 total revenue of $268 million and a GAAP net loss of $115 million, compared with $452 million of revenue and $124 million of net income in Q2 2025.

How did Ionis (IONS) commercial revenue perform in Q2 2026?

Commercial revenue in Q2 2026 was $119 million, up from $103 million a year earlier. Growth was driven mainly by DAWNZERA product sales and royalties from SPINRAZA, WAINUA and other partnered medicines.

What non-GAAP results did Ionis (IONS) report for Q2 2026?

Ionis reported non-GAAP operating expenses of $325 million and a non-GAAP net loss of $70 million in Q2 2026, compared with non-GAAP operating expenses of $282 million and non-GAAP net income of $154 million in Q2 2025.

What is Ionis (IONS) cash position as of June 30, 2026?

As of June 30, 2026, Ionis held $2,055 million in cash, cash equivalents and short-term investments, down from $2,677 million at December 31, 2025, primarily due to repayment of its 0% convertible senior notes due April 2026.

Which product sales contributed to Ionis (IONS) Q2 2026 revenue?

Q2 2026 net product sales totaled $31 million, including $5 million from TRYNGOLZA and $26 million from DAWNZERA. This compared with $19 million of total product sales in the prior-year quarter.

What key pipeline and regulatory milestones did Ionis (IONS) highlight?

Ionis highlighted U.S. approval and early launch momentum for TRYNGOLZA, DAWNZERA’s growth in hereditary angioedema, completed enrollment of the Phase 3 REVEAL Angelman syndrome trial, and upcoming milestones including potential approval of zilganersen and pelacarsen Lp(a) HORIZON outcomes data.

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):  July 29, 2026

IONIS PHARMACEUTICALS, INC.
(Exact Name of Registrant as Specified in Charter)
 
Delaware
(State or Other Jurisdiction of Incorporation)
 
000-19125
 
33-0336973
(Commission File No.)
 
(IRS Employer Identification No.)

2855 Gazelle Court
Carlsbad, CA 92010
(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
Common Stock, $.001 Par Value
 
IONS
 
The Nasdaq Stock Market, LLC

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 July 29, 2026, Ionis Pharmaceuticals, Inc. (the “Company”) issued a press release announcing the Company’s financial results for the quarter ended June 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.
 
Exhibit No.
Description
99.1
Press Release dated July 29, 2026.
   
104
Cover Page Interactive Data File (embedded within the Inline XBRL document).


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:  July 29, 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 second quarter 2026 financial results and highlights progress on key programs

- TRYNGOLZA® (olezarsen) demonstrating early sHTG launch momentum, underscoring multi-billion-dollar revenue potential in large patient population –
 
- Completed enrollment in Phase 3 REVEAL trial in Angelman syndrome -
 
- On track to achieve 2026 financial guidance -
 
CARLSBAD, Calif., July 29, 2026 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results and provided key updates for the second quarter ended June 30, 2026.
 
“With the approval of TRYNGOLZA in late June, Ionis is bringing the first and only treatment to reduce triglycerides and acute pancreatitis to people living with severe hypertriglyceridemia. We are encouraged by the early launch momentum and look forward to accelerating growth from TRYNGOLZA and our other wholly owned medicines in the quarters and years to come,” said Brett P. Monia, Ph.D., chief executive officer of Ionis. “In the second half of this year, we expect multiple important milestones, including approval of zilganersen for Alexander disease, positioning us for our first independent launch from our leading neurology portfolio. We also expect results from the landmark pelacarsen Lp(a) HORIZON cardiovascular outcomes trial and the global launch of bepirovirsen for chronic hepatitis B. With our advancing pipeline and growing commercial momentum, Ionis is on track to deliver accelerating value to patients and all Ionis stakeholders.”

Second Quarter 2026 Summary Financial Results(1):

    
Three months ended
June 30,
     
Six months ended
June 30,
  
   
2026
   
2025
   
2026
   
2025
 
   
(amounts in millions)
 
Total revenue
 
$
268
   
$
452
   
$
514
   
$
584
 
Operating expenses
 
$
370
   
$
312
   
$
734
   
$
591
 
Operating expenses on a non-GAAP basis
 
$
325
   
$
282
   
$
645
   
$
532
 
Income (loss) from operations
 
(102
)
 
$
140
   
(220
)
 
(7
)
Income (loss) from operations on a non-GAAP basis
 
(57
)
 
$
170
   
(131
)
 
$
52
 

  (1)
Reconciliation of GAAP to non-GAAP basis contained later in this release.

1

Second Quarter 2026 Financial Highlights
 
Revenue in the second quarter and first half of 2026 increased by 56% and 69% respectively, compared to the same periods last year, excluding the one-time sapablursen upfront payment recognized in the second quarter of 2025, driven by continued commercial success and substantial R&D revenue from multiple partnerships
 
Operating expenses for the second quarter and first half of 2026 were in line with expectations and increased year over year primarily from investments related to the commercialization efforts for TRYNGOLZA and DAWNZERA and launch preparations for zilganersen in Alexander disease
 
Ended the second quarter of 2026 with cash and short-term investments of $2.1 billion as of June 30, 2026, enabling continued investments to drive value in Ionis’ wholly owned portfolio
 
Second Quarter 2026 Financial Results
 
“Our performance in the first half of this year reflects the strength and resilience of our business. We delivered significantly increased commercial revenue from our independent launches, substantial R&D revenue from multiple partnered medicines and invested in our advancing wholly owned pipeline,” said Elizabeth L. Hougen, chief financial officer of Ionis. “Even with the outcome of the CARDIO-TTRansform study of eplontersen in ATTR-CM, our first-half execution and positive outlook for the second half of the year keep us on track to achieve our 2026 financial guidance. We also remain on track to achieve our goal of cashflow breakeven in 2028 and deliver substantial growth 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 $5 million and $32 million in the second quarter and first half of 2026, respectively
 

Demonstrated continued strong demand in FCS, offset by the reduced net price effective April 1, 2026
 

On track to achieve full year 2026 product sales of $100-110 million, in line with revenues generated in 2025
 

o
sHTG U.S. launch demonstrating early momentum following June 2026 approval
 

o
FCS launch outside the U.S. underway; sHTG marketing application under review in the European Union (EU) with potential launch in 2027
 

o
Results from the CORE and CORE2 open-label extension study (CORE-OLE) will be presented 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 $26 million and $42 million in the second quarter and first half of 2026, respectively; second quarter net product sales increased 63% compared to the first quarter of 2026
 

o
On track to achieve full year 2026 product sales of $110-120 million, representing a continuing driver of year-over-year revenue growth
 

o
Launch outside the U.S. underway, with new market approvals in the EU
 
2

Zilganersen on track to launch this year as the first and only medicine to demonstrate clinically meaningful and disease-modifying benefit in children and adults with Alexander disease (AxD), assuming approval
 

o
New Drug Application (NDA) under FDA Priority Review with PDUFA target action date of September 22, 2026
 

o
Entered a license agreement with Recordati to develop and commercialize zilganersen outside the U.S.
 
Obudanersen, a potential treatment for Angelman syndrome, completed enrollment in the Phase 3 REVEAL study, with data anticipated in the second half of 2027
 
ION775, a potential treatment for sHTG, 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
Results from the Phase 1 study of ION775 will be presented at ESC in August 2026
 
Recent Highlights – Partnered Medicines
 
SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $402 million in the second quarter of 2026, resulting in royalty revenue of $54 million
 
WAINUA® (eplontersen) (WAINZUA in EU) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated global sales of $70 million in the second quarter of 2026, resulting in royalty revenue of $16 million
 

o
Global WAINUA ATTRv-PN launches continuing with additional submissions in progress to further expand global WAINUA access
 

o
Phase 3 CARDIO-TTRansform study of eplontersen for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) missed the primary composite endpoint in the overall population, demonstrated nominal significance in the pre-specified monotherapy population, and a favorable safety and tolerability profile
 

o
Results from the CARDIO-TTRansform study will be presented at the ESC Congress in August 2026
 
Bepirovirsen, a potential first-in-class treatment of chronic hepatitis B (CHB), on track for global launch this year
 

o
Under regulatory review in multiple countries, including the U.S., EU, Japan, and China
 

o
Granted Priority Review in the U.S. and PDUFA target action date of October 26, 2026
 

o
Positive data from the Phase 3 B-Well studies showing unprecedented functional cure rates presented at the European Association for the Study of the Liver (EASL) Congress 2026
 
Salanersen, a potential treatment for SMA, entered Phase 3 development and granted Breakthrough Therapy Designation by the FDA, based on positive interim Phase 1 results demonstrating potential to achieve high efficacy with annual dosing
 
Sapablursen, a potential treatment for polycythemia vera, entered Phase 3 development based on positive Phase 2 data
 
Diranersen (IONIS-MAPTRx / BIIB080), a potential treatment for Alzheimer’s disease, demonstrated benefit in measures of cognition with favorable safety and tolerability in the Phase 2 CELIA study; Biogen plans to advance diranersen into Phase 3 development
 
3

Revenue
 
Ionis’ revenue was comprised of the following:

   
Three months ended
June 30,
   
Six months ended
June 30,
 
   
2026
   
2025
   
2026
   
2025
 
Revenue:
 
(amounts in millions)
 
Commercial revenue:
                       
Product sales, net:
                       
TRYNGOLZA sales, net
 
$
5
   
$
19
   
$
32
   
$
26
 
DAWNZERA sales, net
   
26
     
-
     
42
     
-
 
Total product sales, net
   
31
     
19
     
74
     
26
 
Royalty revenue:
                               
SPINRAZA royalties
   
53
     
54
     
97
     
102
 
WAINUA royalties
   
16
     
10
     
27
     
20
 
Other royalties
   
7
     
6
     
10
     
12
 
Total royalty revenue
   
76
     
70
     
134
     
134
 
Other commercial revenue
   
12
     
14
     
18
     
19
 
Total commercial revenue
   
119
     
103
     
226
     
179
 
Research and development revenue:
                               
Collaborative agreement revenue
   
133
     
337
     
254
     
382
 
WAINUA joint development revenue
   
16
     
12
     
34
     
23
 
Total research and development revenue
   
149
     
349
     
288
     
405
 
Total revenue
 
$
268
   
$
452
   
$
514
   
$
584
 

Commercial revenue for the second quarter and first half of 2026 increased 15% and 27%, 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 second quarter and first half of 2026, compared to the same periods in 2025, driven by multiple payments for programs advancing under its 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 second quarter and first half of 2026 increased year over year, in line with expectations, primarily from investments related to the commercialization efforts for TRYNGOLZA and DAWNZERA and launch preparations for zilganersen in Alexander disease, with full-year expenses remaining on track for low-teens percentage growth year-over-year.

Balance Sheet
 
As of June 30, 2026, Ionis’ cash, cash equivalents and short-term investments decreased to $2.1 billion, compared to $2.7 billion on December 31, 2025, primarily due to repayment of the 0% convertible notes on April 1, 2026.
 
4

Webcast and Other Updates

Management will host a conference call and webcast to discuss Ionis’ second quarter 2026 results at 8:30 a.m. Eastern time on Wednesday, July 29, 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 second 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.

WAINUA® (eplontersen): WAINUA was approved by the U.S. Food and Drug Administration for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. For more information about WAINUA, including the full U.S. Prescribing Information, visit wainua.com.

For more information about SPINRAZA and QALSODY, visit https://www.spinraza.com/ and https://www.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.

5

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 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.

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
   
Six months ended
 
   
June 30,
   
June 30,
 
   
2026
   
2025
   
2026
   
2025
 
   
(unaudited)
 
Revenue:
                       
Commercial revenue:
                       
Product sales, net
 
$
31
   
$
19
   
$
74
   
$
26
 
Royalty revenue
   
76
     
70
     
134
     
134
 
Other commercial revenue
   
12
     
14
     
18
     
19
 
Total commercial revenue
   
119
     
103
     
226
     
179
 
Research and development revenue:
                               
Collaborative agreement revenue
   
133
     
337
     
254
     
382
 
WAINUA joint development revenue
   
16
     
12
     
34
     
23
 
Total research and development revenue
   
149
     
349
     
288
     
405
 
Total revenue
   
268
     
452
     
514
     
584
 
Expenses:
                               
Cost of sales
   
3
     
4
     
6
     
6
 
Research, development and patent
   
217
     
217
     
427
     
418
 
Selling, general and administrative
   
150
     
91
     
301
     
167
 
Total operating expenses
   
370
     
312
     
734
     
591
 
Income (loss) from operations
   
(102
)
   
140
     
(220
)
   
(7
)
                                 
Other income (expense):
                               
Interest expense related to the sale of future royalties
   
(18
)
   
(19
)
   
(35
)
   
(37
)
Other income, net
   
6
     
3
     
49
     
21
 
Income (loss) before income tax benefit (expense)
   
(114
)
   
124
     
(206
)
   
(23
)
                                 
Income tax benefit (expense)
   
(1
)
   
-
     
(1
)
   
-
 
                                 
Net income (loss)
 
(115
)
 
$
124
   
(207
)
 
(23
)
Basic net income (loss) per share
 
(0.69
)
 
$
0.78
   
(1.25
)
 
(0.15
)
Diluted net income (loss) per share
 
(0.69
)
 
$
0.70
   
(1.25
)
 
(0.15
)
Shares used in computing basic net income (loss) per share
   
165
     
159
     
165
     
159
 
Shares used in computing diluted net income (loss) per share
   
165
     
182
     
165
     
159
 

7

IONIS PHARMACEUTICALS, INC.
 
Reconciliation of GAAP to Non-GAAP Basis:
Condensed Consolidated Operating Expenses, Income (Loss) From Operations, and Net Income (Loss)
(In Millions)

   
Three months ended
June 30,
   
Six months ended
June 30,
 
   
2026
   
2025
   
2026
   
2025
 
   
(unaudited)
 
As reported cost of sales according to GAAP
 
$
3
   
$
4
   
$
6
   
$
6
 
Excluding compensation expense related to equity awards (1)
   
-
     
-
     
-
     
-
 
Non-GAAP cost of sales
 
$
3
   
$
4
   
$
6
   
$
6
 
                                 
As reported research, development and patent expenses according to GAAP
 
$
217
   
$
217
   
$
427
   
$
418
 
Excluding compensation expense related to equity awards
   
(25
)
   
(20
)
   
(50
)
   
(40
)
Non-GAAP research, development and patent expenses
 
$
192
   
$
197
   
$
377
   
$
378
 
                                 
As reported selling, general and administrative expenses according to GAAP
 
$
150
   
$
91
   
$
301
   
$
167
 
Excluding compensation expense related to equity awards
   
(20
)
   
(10
)
   
(39
)
   
(19
)
Non-GAAP selling, general and administrative expenses
 
$
130
   
$
81
   
$
262
   
$
148
 
                                 
As reported operating expenses according to GAAP
 
$
370
   
$
312
   
$
734
   
$
591
 
Excluding compensation expense related to equity awards
   
(45
)
   
(30
)
   
(89
)
   
(59
)
Non-GAAP operating expenses
 
$
325
   
$
282
   
$
645
   
$
532
 
                                 
As reported income (loss) from operations according to GAAP
 
(102
)
 
$
140
   
(220
)
 
(7
)
Excluding compensation expense related to equity awards
   
(45
)
   
(30
)
   
(89
)
   
(59
)
Non-GAAP income (loss) from operations
 
(57
)
 
$
170
   
(131
)
 
$
52
 
                                 
As reported net income (loss) according to GAAP
 
(115
)
 
$
124
   
(207
)
 
(23
)
Excluding compensation expense related to equity awards and related tax effects
   
(45
)
   
(30
)
   
(89
)
   
(59
)
Non-GAAP net income (loss)
 
(70
)
 
$
154
   
(118
)
 
$
36
 

  (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 income (loss) from operations, and non-GAAP net income (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)

   
June 30,
   
December 31,
 
   
2026
   
2025
 
   
(unaudited)
       
Assets:
           
Cash, cash equivalents and short-term investments
 
$
2,055
   
$
2,677
 
Contracts receivable
   
59
     
66
 
Other current assets
   
378
     
247
 
Property, plant and equipment, net
   
155
     
123
 
Right-of-use assets
   
232
     
239
 
Other assets
   
116
     
172
 
Total assets
 
$
2,995
   
$
3,524
 
                 
Liabilities and stockholders’ equity:
               
Current portion of deferred contract revenue
 
$
74
   
$
74
 
0% convertible senior notes due April 2026, net – current
   
-
     
432
 
Other current liabilities
   
242
     
277
 
0% convertible senior notes due 2030, net
   
753
     
751
 
1.75% convertible senior notes due 2028, net
   
569
     
568
 
Liability related to sale of future royalties, net
   
563
     
551
 
Long-term lease liabilities
   
262
     
262
 
Long-term obligations, less current portion
   
29
     
28
 
Long-term deferred contract revenue
   
63
     
92
 
Total stockholders’ equity
   
440
     
489
 
Total liabilities and stockholders’ equity
 
$
2,995
   
$
3,524
 

10

Key 2026 Value Driving Events(1)
 
 
New Product Launches
 
Program
 
Indication
 
Location
Status
 
DAWNZERA
 
HAE
 
EU
Achieved
 
Olezarsen
 
sHTG
 
U.S.
Achieved
 
Zilganersen
 
Alexander disease
 
U.S.
 
Bepirovirsen
 
CHB
 
U.S. & Japan

 
Regulatory Actions
 
Program
 
Indication
 
Regulatory Action
Status
 
Donidalorsen
 
HAE
 
EU approval decision
Achieved
 
Olezarsen
 
sHTG
 
U.S. approval decision
Achieved
 
EU approval decision
 •
 
Zilganersen
 
Alexander disease
 
U.S. submission
Achieved
 
U.S. approval decision
 
Nusinersen
(high dose)
 
SMA
 
EU approval decision
Achieved
 
U.S. approval decision
Achieved
 
Eplontersen
 
ATTR-CM
 
Regulatory submission(s)
 
Bepirovirsen
 
CHB
 
Regulatory submission(s)
Achieved
 
Regulatory decision(s)
 
Pelacarsen
 
Lp(a)- CVD
 
U.S. submission

 
Key Phase 3 Clinical Events
 
Program
 
Indication
 
Event
Status
 
Obudanersen
 
Angelman syndrome
 
REVEAL fully enrolled
Achieved
 
Bepirovirsen
 
HBV
 
B-Well data
Achieved
 
Pelacarsen
 
Lp(a)-CVD
 
Lp(a) HORIZON data
 
Eplontersen
 
ATTR-CM
 
CARDIO-TTRansform data
Not Achieved
 
Sefaxersen
 
IgAN
 
IMAGINATION data
 
Ulefnersen
 
FUS-ALS
 
FUSION data
 
Salanersen
 
SMA
 
Phase 3 initiation
Achieved
 
Sapablursen
 
Polycythemia Vera
 
Phase 3 initiation
Achieved

 
Key Phase 2 Clinical Events
 
Program
 
Indication
 
Event
Status
 
Diranersen
 
Alzheimer’s disease
 
Phase 2 CELIA data
Achieved
 
Tominersen
 
Huntington’s disease
 
Phase 2 data
Not Achieved
 
Tonlamarsen
 
Acute Severe Hypertension
 
Phase 2 data
Achieved

  (1)
Timing expectations based on current assumptions and subject to change.


Indicates that the milestone is anticipated in 2026.
 
#   #   #


11

Filing Exhibits & Attachments

4 documents