Ionis Pharmaceuticals (NASDAQ: IONS) lifts 2026 guidance after strong Q1 revenue

Rhea-AI Filing Summary

Ionis Pharmaceuticals reported strong first quarter 2026 results and raised its full-year outlook. Total revenue rose to $246 million from $132 million a year earlier, driven by higher product sales of TRYNGOLZA and DAWNZERA and increased research and development revenue, including about $95 million of partnership milestones.

Despite the growth, Ionis recorded a GAAP net loss of $93 million, improving from a $147 million loss last year. Cash, cash equivalents and short-term investments were $1.9 billion as of March 31, 2026, with an additional $633 million held in escrow for 0% convertible notes maturing in April 2026.

Reflecting its momentum, Ionis increased 2026 total revenue guidance to $875–900 million and introduced product sales targets of $100–110 million for TRYNGOLZA and $110–120 million for DAWNZERA. The company also narrowed its expected 2026 non-GAAP operating loss to $425–475 million and reaffirmed a year-end liquidity goal of more than $1.6 billion.

Positive

• Strong top-line growth: Total revenue increased to $246 million from $132 million year over year, with commercial revenue and collaboration milestones both contributing.
• Upgraded 2026 outlook: Ionis raised 2026 total revenue guidance to $875–900 million and reduced expected non-GAAP operating loss to $425–475 million, reflecting confidence in product launches and partnered programs.

Negative

• Business still operating at a loss: Ionis reported a Q1 2026 GAAP net loss of $93 million and non-GAAP net loss of $50 million, highlighting ongoing dependence on external funding and partnerships.
• Cash balance declined: Cash, cash equivalents and short-term investments fell to $1.919 billion from $2.677 billion at December 31, 2025, alongside sizeable convertible note obligations.

Insights

Biotech earnings and guidance analyst

Ionis delivered strong revenue growth and raised 2026 guidance, though it remains loss-making.

Ionis nearly doubled revenue to $246 million in Q1 2026 versus $132 million a year earlier, with commercial revenue up on TRYNGOLZA and DAWNZERA and collaboration revenue boosted by about $95 million in milestones. This mix shows both product traction and ongoing partner support.

GAAP net loss improved to $93 million from $147 million, while non-GAAP net loss narrowed to $50 million, mainly by excluding $43 million of non-cash equity compensation. Cash and short-term investments were $1.919 billion plus a $633 million escrow for April 2026 note repayment, indicating substantial resources but also significant obligations.

The company lifted 2026 revenue guidance to $875–900 million and reduced expected non-GAAP operating loss to $425–475 million, supported by Priority Review for TRYNGOLZA in sHTG and strong first-quarter R&D revenue. Subsequent data readouts from Phase 3 programs like pelacarsen Lp(a) HORIZON and eplontersen CARDIO-TTRansform later in 2026 may further influence the outlook.

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, and exhibit attachments filed with this report.

Key Figures

Q1 2026 total revenue: $246 million Q1 2026 product sales: $43 million TRYNGOLZA Q1 2026 sales: $27 million +5 more

8 metrics

Q1 2026 total revenue $246 million Three months ended March 31, 2026; vs. $132 million in 2025

Q1 2026 product sales $43 million Net product sales; up from $6 million in Q1 2025

TRYNGOLZA Q1 2026 sales $27 million Net TRYNGOLZA sales for quarter; $6 million in Q1 2025

DAWNZERA Q1 2026 sales $16 million Net DAWNZERA sales; no comparable prior-year sales

Q1 2026 GAAP net loss $93 million Net loss for three months ended March 31, 2026; $147 million in 2025

Q1 2026 non-GAAP net loss $50 million Excludes $43 million equity-compensation and related tax effects

Cash and investments $1.919 billion Cash, cash equivalents and short-term investments as of March 31, 2026

2026 revenue guidance $875–900 million Full-year 2026 total revenue outlook, raised from $800–825 million

Key Terms

non-GAAP, Priority Review, familial chylomicronemia syndrome, hereditary angioedema, +2 more

6 terms

non-GAAP financial

"non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net loss were adjusted"

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.

Priority Review regulatory

"Olezarsen sNDA accepted by the FDA for Priority Review; sHTG launch preparations on track"

Priority review is a regulatory fast-track that shortens the time an agency spends evaluating a drug, vaccine or medical device application so a decision comes sooner than normal. For investors, it matters because a faster review is like an express lane to market: it can speed revenue potential and reduce regulatory uncertainty, but it does not guarantee approval and still requires the product to meet safety and effectiveness standards.

familial chylomicronemia syndrome medical

"TRYNGOLZA was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome"

A rare inherited disorder in which the body cannot clear large fat particles from the bloodstream after meals, causing extremely high blood-fat levels and recurrent inflammation of the pancreas, severe abdominal pain, and other complications. It matters to investors because there are few effective treatments, so drugmakers, gene-therapy developers, and diagnostic firms pursuing solutions can see outsized commercial value, regulatory incentives, and volatile stock moves if clinical trials or approvals succeed or fail — like a tiny market where each customer can be highly profitable.

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 and 1.75% convertible senior notes due 2028"

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.

CARDIO-TTRansform medical

"eplontersen CARDIO-TTRansform cardiovascular outcomes trials later this year"

Offering Details

earnings_snapshot

Offering

Offering Type earnings_snapshot

Understanding 8-K Material Events →

false000087401500008740152026-04-292026-04-29

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):  April 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 April 29, 2026, Ionis Pharmaceuticals, Inc. (the “Company”) issued a press release announcing the Company’s financial results for the quarter ended March 31, 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 April 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:  April 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 first quarter 2026 financial results and highlights progress on key programs

- TRYNGOLZA^® (olezarsen) showed increasing demand in FCS driven by strong launch execution –

- Olezarsen sNDA accepted by the FDA for Priority Review; sHTG launch preparations on track –

– Increasing annual TRYNGOLZA peak net sales guidance to >$3B for sHTG –

- Zilganersen NDA for Alexander disease accepted for Priority Review, paving way for Ionis’ first independent launch from leading neurology pipeline –

- Strong first-quarter performance and outlook for the year supports improved 2026 financial guidance -

CARLSBAD, Calif., April 29, 2026 – Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results and provided key updates for the first quarter ended March 31, 2026.

“Ionis’ strong performance in the first quarter of 2026 underscores the strength of our commercial and R&D engines. Our independent launches are increasingly contributing to revenue, driven by strong commercial execution, and we are on track for two additional groundbreaking independent launches in 2026 — olezarsen for severe hypertriglyceridemia, our first medicine for a broad patient population, and zilganersen for Alexander disease, the first launch from our leading neurology pipeline,” said Brett P. Monia, Ph.D., chief executive officer of Ionis. “In addition, we look forward to multiple key value-driving events this year, including results from pivotal Phase 3 partnered programs. These include presentation of positive bepirovirsen data in chronic hepatitis B next month at EASL, as well as results from the landmark pelacarsen Lp(a) HORIZON and eplontersen CARDIO-TTRansform cardiovascular outcomes trials later this year.”

1

First Quarter 2026 Summary Financial Results⁽¹⁾:

		Three months ended March 31,
		2026				2025
		(amounts in millions)
Total revenue		$	246			$	132
Operating expenses		$	364			$	278
Operating expenses on a non-GAAP basis		$	321			$	249
Loss from operations		$	(118	)		$	(146	)
Loss from operations on a non-GAAP basis		$	(75	)		$	(117	)

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

First Quarter 2026 Financial Highlights

• Revenue increased 87% in the first quarter of 2026 compared to the same period last year, driven by continued commercial success. In addition, Ionis earned substantial R&D revenue, including $95 million in payments from both clinical and regulatory milestones from multiple partnerships

• Operating expenses for the quarter ended March 31, 2026 were in line with expectations and increased year over year primarily from investments related to the commercialization efforts for TRYNGOLZA and DAWNZERA as well as launch preparations for olezarsen in sHTG and zilganersen in Alexander disease

• Cash and short-term investments were $1.9 billion as of March 31, 2026.  The change in cash and short-term investments from year end 2025 was primarily related to the $633 million the Company used for the maturity of the 0% convertible notes due on April 1, 2026

• Increasing annual olezarsen peak net sales guidance to >$3 billion from >$2 billion to reflect increasing confidence in the sHTG market opportunity for olezarsen

First Quarter 2026 Financial Results

“Ionis entered 2026 with strong momentum. We continued this momentum with the first quarter financial results reflecting increased commercial revenue from our independent launches and robust R&D revenue when compared to the same period last year,” said Elizabeth L. Hougen, chief financial officer of Ionis. “Based on our strong year-to-date revenue performance, accelerating momentum and positive outlook for the rest of the year, we are improving our 2026 financial guidance. The strong performance we expect in 2026 will support substantial growth and long-term value creation and our goal of reaching cash-flow breakeven in 2028.”

Recent Highlights - Wholly Owned Medicines

• TRYNGOLZA^® (olezarsen), the first FDA-approved treatment for adults living with familial chylomicronemia syndrome (FCS) as an adjunct to diet

o Generated U.S. net product sales of $27 million in the first quarter of 2026, reflecting continued strong demand, offset by a decrease in net price

2

o Launch initiated in the European Union (EU) by Sobi

• Olezarsen on track to launch this year as a transformational medicine for severe hypertriglyceridemia (sHTG), assuming approval

o sNDA accepted by the FDA for Priority Review for the treatment of sHTG with a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026

o The European Medicines Agency (EMA) accepted an indication extension application in March for the treatment of adult patients with sHTG

• 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 $16 million in the first quarter of 2026, an increase of 125% versus the fourth quarter of 2025

o Launch initiated in the EU by Otsuka

o Positive one-year results from OASISplus open-label extension cohort published in the Journal of Asthma and Allergy

• 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) for AxD accepted by FDA for Priority Review with PDUFA target action date of September 22, 2026

o Expanded access program (EAP) in U.S. underway

o Positive additional results from the pivotal study presented at the American Academy of Neurology 2026 annual meeting

Recent Highlights – Partnered Medicines

• SPINRAZA^® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $374 million in the first quarter of 2026, resulting in royalty revenue of $44 million

o SPINRAZA high dose regimen approved and launched in the U.S. and EU

• WAINUA^® (eplontersen) (WAINZUA in EU) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated global sales of $51 million in the first quarter of 2026, resulting in royalty revenue of $11 million

o Launches underway in numerous regions, including the EU and China; submissions in progress to expand WAINUA access globally

o Phase 3 CARDIO-TTRansform study design and baseline characteristics to be presented at the Annual Congress of the Heart Failure Association of the ESC 2026

• Bepirovirsen, a potential first-in-class medicine for chronic hepatitis B (CHB), achieved the primary endpoint demonstrating a statistically significant and clinically meaningful functional cure rate in the B-Well 1 and B-Well 2 Phase 3 studies

o GSK to present the positive Phase 3 data at the European Association for the Study of the Liver (EASL) Congress 2026

3

o On track for a 2026 launch with global regulatory filings underway, assuming approval

■ NDA filing accepted by FDA for Priority Review with PDUFA date of October 26, 2026; granted Breakthrough Therapy designation

■ Accepted for regulatory review in EU, Japan, and China

4

Revenue

Ionis’ revenue was comprised of the following:

		Three months ended
		March 31,
		2026				2025
Revenue		(amounts in millions)
Commercial revenue:
Product sales, net:
TRYNGOLZA sales, net		$	27			$	6
DAWNZERA sales, net			16				-
Total product sales, net			43				6
Royalty revenue:
SPINRAZA royalties			44				48
WAINUA royalties			11				9
Other royalties			3				7
Total royalty revenue			58				64
Other commercial revenue			7				6
Total commercial revenue			108				76
Research and development revenue:
Collaborative agreement revenue			120				46
WAINUA joint development revenue			18				10
Total research and development revenue			138				56
Total revenue		$	246			$	132

Commercial revenue for the first quarter ended March 31, 2026, increased 42%, compared to the same period in 2025. This increase was primarily driven by TRYNGOLZA and DAWNZERA product sales. Higher research and development revenue also contributed to the year-over-year revenue increase including approximately $95 million in milestone payments from multiple partnerships.

Operating Expenses

Operating expenses for the first quarter ended March 31, 2026, were driven from investments primarily related to commercialization efforts for TRYNGOLZA and DAWNZERA as well as launch preparations for olezarsen in sHTG and zilganersen in Alexander disease.

Balance Sheet

As of March 31, 2026, Ionis’ cash, cash equivalents and short-term investments decreased to $1.9 billion, compared to $2.7 billion on December 31, 2025. At March 31, 2026, Ionis had an escrow deposit of $633 million, which the Company used for the maturity of its 0% convertible notes due on April 1, 2026.

5

2026 Financial Guidance

Ionis improved its 2026 financial guidance to reflect the strong revenue performance experienced year-to-date and the Company’s outlook for the balance of 2026. Overall, the Company increased total revenue and decreased operating loss both by $75 million. The improvements were driven by Priority Review for TRYNGOLZA, strong first quarter R&D revenue and the anticipated continued success of the Company’s ongoing commercial launches.

Full Year 2026 Guidance	Previous Guidance	New Guidance
Total Revenue	$800-825 million	$875-900 million
TRYNGOLZA product sales, net	NA	$100-110 million
DAWNZERA product sales, net	NA	$110-120 million
Operating loss on a non-GAAP basis	$500-550 million	$425-475 million
Cash, cash equivalents and short-term investments	>$1.6 billion	>$1.6 billion

Webcast and Other Updates

Management will host a conference call and webcast to discuss Ionis’ first quarter 2026 results at 8:30 a.m. Eastern time on Wednesday, April 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 first quarter 2026 earnings slides click here.

Ionis’ Marketed Medicines

INDICATION for TRYNGOLZA^® (olezarsen)

TRYNGOLZA^® (olezarsen) was approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred.

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions

Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur.

ADVERSE REACTIONS

The most common adverse reactions (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count and arthralgia.

Please see full Prescribing Information for TRYNGOLZA.

6

INDICATION for DAWNZERA^TM (donidalorsen)

DAWNZERA™ (donidalorsen) 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.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

DAWNZERA is contraindicated in patients with a history of serious hypersensitivity reactions, including anaphylaxis, to donidalorsen or any of the excipients in DAWNZERA.

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions

Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with DAWNZERA. If signs and symptoms of serious hypersensitivity reactions occur, discontinue DAWNZERA and institute appropriate therapy.

ADVERSE REACTIONS

Most common adverse reactions (incidence ≥ 5%) are injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort.

Please see full Prescribing Information for DAWNZERA.

INDICATION for WAINUA^® (eplontersen)

WAINUA injection, for subcutaneous use, 45 mg is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

IMPORTANT SAFETY INFORMATION for WAINUA^® (eplontersen)

WARNINGS AND PRECAUTIONS

Reduced Serum Vitamin A Levels and Recommended Supplementation WAINUA leads to a decrease in serum vitamin A levels. Supplement with recommended daily allowance of vitamin A. Refer patient to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

ADVERSE REACTIONS

Most common adverse reactions (≥9% in WAINUA-treated patients) were vitamin A decreased (15%) and vomiting (9%).

Please see link to U.S. Full Prescribing Information for WAINUA.

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).

7

About Ionis Pharmaceuticals, Inc.

For 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^® is a registered trademark of Ionis Pharmaceuticals, Inc. TRYNGOLZA^® is a registered trademark of Ionis Pharmaceuticals, Inc. DAWNZERA^TM is a trademark of Ionis Pharmaceuticals, Inc. AKCEA^TM is a trademark of Akcea Therapeutics, Inc. TEGSEDI^TM is a trademark of Akcea Therapeutics, Inc. WAYLIVRA^TM 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

8

IONIS PHARMACEUTICALS, INC.

SELECTED FINANCIAL INFORMATION

Condensed Consolidated Statements of Operations

(In Millions, Except Per Share Data)

		Three months ended
		March 31,
		2026				2025
		(unaudited)
Revenue:
Commercial revenue:
Product sales, net		$	43			$	6
Royalty revenue			58				64
Other commercial revenue			7				6
Total commercial revenue			108				76
Research and development revenue:
Collaborative agreement revenue			120				46
WAINUA joint development revenue			18				10
Total research and development revenue			138				56
Total revenue			246				132
Expenses:
Cost of sales			3				1
Research, development and patent			210				201
Selling, general and administrative			151				76
Total operating expenses			364				278
Loss from operations			(118	)			(146	)
Other income (expense):
Interest expense related to the sale of future royalties			(17	)			(19	)
Other income, net			42				18
Loss before income tax expense			(93	)			(147	)
Income tax expense			-				-
Net loss		$	(93	)		$	(147	)
Basic and diluted net loss per share		$	(0.56	)		$	(0.93	)
Shares used in computing basic and diluted net loss per share			165				159

9

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 March 31,
		2026				2025
		(unaudited)
As reported cost of sales according to GAAP		$	3			$	1
Excluding compensation expense related to equity awards (1)			-				-
Non-GAAP cost of sales		$	3			$	1
As reported research, development and patent expenses according to GAAP		$	210			$	201
Excluding compensation expense related to equity awards			(25	)			(20	)
Non-GAAP research, development and patent expenses		$	185			$	181
As reported selling, general and administrative expenses according to GAAP		$	151			$	76
Excluding compensation expense related to equity awards			(18	)			(9	)
Non-GAAP selling, general and administrative expenses		$	133			$	67
As reported operating expenses according to GAAP		$	364			$	278
Excluding compensation expense related to equity awards			(43	)			(29	)
Non-GAAP operating expenses		$	321			$	249
As reported loss from operations according to GAAP		$	(118	)		$	(146	)
Excluding compensation expense related to equity awards			(43	)			(29	)
Non-GAAP loss from operations		$	(75	)		$	(117	)
As reported net loss according to GAAP		$	(93	)		$	(147	)
Excluding compensation expense related to equity awards and related tax effects			(43	)			(29	)
Non-GAAP net loss		$	(50	)		$	(118	)

(1) Amounts appear as zero due to rounding in millions.

10

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.

11

IONIS PHARMACEUTICALS, INC.

Condensed Consolidated Balance Sheets

(In Millions)

		March 31,				December 31,
		2026				2025
		(unaudited)
Assets:
Cash, cash equivalents and short-term investments		$	1,919			$	2,677
Escrow deposits			633				-
Contracts receivable			74				66
Other current assets			312				247
Property, plant and equipment, net			142				123
Right-of-use assets			235				239
Other assets			135				172
Total assets		$	3,450			$	3,524
Liabilities and stockholders’ equity:
Current portion of deferred contract revenue		$	69			$	74
0% convertible senior notes due April 2026 – current			433				432
Other current liabilities			215				277
0% convertible senior notes due 2030, net			752				751
1.75% convertible senior notes due 2028, net			569				568
Liability related to sale of future royalties, net			558				551
Long-term lease liabilities			259				262
Long-term obligations, less current portion			28				28
Long-term deferred contract revenue			76				92
Total stockholders’ equity			491				489
Total liabilities and stockholders’ equity		$	3,450			$	3,524

12

Key 2026 Value Driving Events⁽¹⁾

	New Product Launches
	Program		Indication	Location
	DAWNZERA		HAE	EU	Achieved
	Olezarsen		sHTG	U.S.	•
	Zilganersen		Alexander disease	U.S.	•
	Bepirovirsen		CHB	U.S. & Japan	•

	Regulatory Actions
	Program		Indication		Regulatory Action
	Donidalorsen		HAE		EU approval decision	Achieved
	Olezarsen		sHTG		U.S. approval decision	•
					EU submission	Achieved
	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		HBV		Regulatory submission(s)	Achieved
					Regulatory decision(s)	•
	Pelacarsen		Lp(a)- CVD		U.S. submission	•

	Key Phase 3 Clinical Events
	Program		Indication		Event
	Obudanersen		Angelman syndrome		Phase 3 enrollment completion	•
	Bepirovirsen		HBV		B-Well data	Achieved
	Pelacarsen		Lp(a)-CVD		Lp(a) HORIZON data	•
	Eplontersen		ATTR-CM		CARDIO-TTRansform data	•
	Sefaxersen		IgAN		IMAGINATION data	•
	Ulefnersen		FUS-ALS		FUSION data	•
	Salanersen		SMA		Phase 3 initiation	•
	Sapablursen		Polycythemia Vera		Phase 3 initiation	•

	Key Phase 2 Clinical Events
	Program		Indication		Event
	IONIS-MAPTRx/ BIIB080		Alzheimer’s disease		Phase 2 CELIA data	•
	Tominersen		Huntington’s disease		Phase 2 GENERATION HD2 data	•
	Tonlamarsen		Uncontrolled hypertension		Phase 2 data	Achieved

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

• Indicates that the milestone is anticipated in 2026.

#   #   #

13

FAQ

How did Ionis Pharmaceuticals (IONS) perform financially in Q1 2026?

Ionis generated $246 million in total revenue in Q1 2026, up from $132 million a year earlier. Commercial revenue rose on TRYNGOLZA and DAWNZERA sales, while research and development revenue benefited from about $95 million in milestone payments from multiple partnerships.

What were Ionis Pharmaceuticals’ Q1 2026 profits or losses?

Ionis reported a GAAP net loss of $93 million in Q1 2026, improving from a $147 million net loss in Q1 2025. On a non-GAAP basis, which excludes non-cash equity compensation and related taxes, net loss narrowed to $50 million from $118 million.

What 2026 financial guidance did Ionis Pharmaceuticals (IONS) provide?

Ionis raised 2026 total revenue guidance to $875–900 million, up from $800–825 million. It now expects a non-GAAP operating loss of $425–475 million, improved from $500–550 million, and continues to target year-end cash, cash equivalents and short-term investments above $1.6 billion.

How are TRYNGOLZA and DAWNZERA contributing to Ionis Pharmaceuticals’ results?

In Q1 2026, Ionis reported $27 million in TRYNGOLZA sales and $16 million in DAWNZERA sales, driving total product sales to $43 million from $6 million a year earlier. Updated 2026 guidance now targets TRYNGOLZA sales of $100–110 million and DAWNZERA sales of $110–120 million.

What is Ionis Pharmaceuticals’ cash position and debt structure as of March 31, 2026?

Ionis held $1.919 billion in cash, cash equivalents and short-term investments as of March 31, 2026, plus a $633 million escrow deposit. The balance sheet includes 0% convertible notes due 2026 and 2030 and 1.75% convertible notes due 2028, alongside a liability related to future royalty sales.

How does Ionis Pharmaceuticals use non-GAAP financial measures in its reporting?

Ionis provides non-GAAP operating expenses, loss from operations and net loss by excluding non-cash compensation expense related to equity awards and related tax effects. Management believes these measures help users assess historical performance and better understand projected operating results and cash flows.

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