Ionis reports first quarter 2026 financial results and highlights progress on key programs

Ionis reports first quarter 2026 financial results and highlights progress on key programs CARLSBAD, Calif.--( BUSINESS WIRE)--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.”

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

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

Recent Highlights – Partnered Medicines

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

DAWNZERA sales, net

Total product sales, net

Royalty revenue:

SPINRAZA royalties

WAINUA royalties

Other royalties

Total royalty revenue

Other commercial revenue

Total commercial revenue

108

Research and development revenue:

Collaborative agreement revenue

120

WAINUA joint development revenue

Total research and development revenue

138

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.

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

Guidance

New

Guidance

Total Revenue

$800-825 million

$875-900 million

TRYNGOLZA product sales, net

$100-110 million

DAWNZERA product sales, net

$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

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.

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).

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 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

Royalty revenue

Other commercial revenue

Total commercial revenue

108

Research and development revenue:

Collaborative agreement revenue

120

WAINUA joint development revenue

Total research and development revenue

138

Total revenue

246

132

Expenses:

Cost of sales

Research, development and patent

210

201

Selling, general and administrative

151

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

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

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

Excluding compensation expense related to equity awards (1)

Non-GAAP cost of sales

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.

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.

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

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

Long-term deferred contract revenue

Total stockholders’ equity

491

489

Total liabilities and stockholders’ equity

$3,450

$3,524

Key 2026 Value Driving Events (1)

New Product Launches

Program

Indication

Location

DAWNZERA

HAE

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-MAPT Rx/ 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.