Ionis reports second quarter 2025 financial results and highlights progress on key programs

Published

- TRYNGOLZATM delivers $19 million in net product sales in the second quarter 2025 -

- Donidalorsen approval in hereditary angioedema (HAE) anticipated next month; Ionis’ second independent launch -

- Phase 3 data from the pivotal CORE and CORE2 studies in severe hypertriglyceridemia (sHTG) expected in September 2025 -

- Increasing 2025 financial guidance based on strong performance and improved outlook -

CARLSBAD, Calif.--(BUSINESS WIRE)-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results and provided key updates for the second quarter ended June 30, 2025.

“During the second quarter, we continued to build momentum across our business,” said Brett P. Monia, Ph.D., chief executive officer of Ionis. “Our strong performance included excellent commercial execution, resulting in a substantial increase in TRYNGOLZA revenues, our first independently launched medicine. We expect additional advancements in the second half, including Ionis’ second independent launch with donidalorsen for hereditary angioedema, anticipated next month, and important Phase 3 results for olezarsen in severe hypertriglyceridemia and zilganersen in Alexander disease. We believe these four programs collectively represent multi-billion-dollar revenue potential and a transformational opportunity for Ionis and for patients.”

Second Quarter 2025 Summary Financial Results(1):

 

Three months ended

Six months ended

 

June 30,

June 30,

 

2025

2024

2025

2024

 

(amounts in millions)

Total revenue

$452

$225

$584

$345

Operating expenses

$312

$291

$591

$560

Operating expenses on a non-GAAP basis

$282

$260

$532

$498

Income (loss) from operations

$140

($66)

($7)

($215)

Income (loss) from operations on a non-GAAP basis

$170

($35)

$52

($153)

(1)

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

Recent Financial Highlights

  • Revenue doubled in the second quarter of 2025 and increased nearly 70% in the first half compared to the same period last year, driven by the continued successful launch of TRYNGOLZA and increased royalty and R&D revenues
  • Operating expenses increased by single digits in the second quarter and first half of 2025, compared to the same periods last year, primarily due to investments related to commercialization efforts for TRYNGOLZA, donidalorsen and WAINUA
  • Increased 2025 financial guidance reflects an improved outlook for the full year, strong overall revenue performance experienced year-to-date, including the early strength in TRYNGOLZA revenues:

 

Full Year 2025 Guidance

 

Previous

Guidance

New

Guidance

Total Revenue

 

$725-750 million

$825-850 million

TRYNGOLZA product sales, net

 

Not provided

$75-80 million

Operating loss on a non-GAAP basis

 

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 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, cardiology, and other 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 Statement

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 and technologies. 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, 2024, 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. 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 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,

 

2025

2024

2025

2024

 

(unaudited)

Revenue:

 

 

 

 

Commercial revenue:

 

 

 

 

Product sales, net

$19

$-

$26

$-

Royalty revenue

70

64

134

113

Other commercial revenue

14

8

19

19

Total commercial revenue

103

72

179

132

Research and development revenue:

 

 

 

 

Collaborative agreement revenue

337

141

382

191

WAINUA joint development revenue

12

12

23

22

Total research and development revenue

349

153

405

213

Total revenue

452

225

584

345

Expenses:

 

 

 

 

Cost of sales

4

4

6

6

Research, development and patent

217

222

418

436

Selling, general and administrative

91

65

167

118

Total operating expenses

312

291

591

560

Income (loss) from operations

140

(66)

(7)

(215)

 

 

 

 

 

Other income (expense):

 

 

 

 

Interest expense related to the sale of future royalties

(19)

(18)

(37)

(36)

Other income, net

3

18

21

42

Income (loss) before income tax benefit (expense)

124

(66)

(23)

(209)

 

 

 

 

 

Income tax benefit (expense)

-

-

-

-

 

 

 

 

 

Net income (loss)

$124

($66)

($23)

($209)

Basic net income (loss) per share

$0.78

($0.45)

($0.15)

($1.43)

Diluted net income (loss) per share

$0.70

($0.45)

($0.15)

($1.43)

Shares used in computing basic net income (loss) per share

159

146

159

146

Shares used in computing diluted net income (loss) per share

182

146

159

146

 

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,

 

2025

2024

2025

2024

 

(unaudited)

As reported research, development and patent expenses according to GAAP

$217

$222

$418

$436

Excluding compensation expense related to equity awards

(20)

(23)

(40)

(45)

Non-GAAP research, development and patent expenses

$197

$199

$378

$391

 

 

 

 

 

 

As reported selling, general and administrative expenses according to GAAP

$91

$65

$167

$118

Excluding compensation expense related to equity awards

(10)

(8)

(19)

(17)

Non-GAAP selling, general and administrative expenses

$81

$57

$148

$101

 

 

 

 

 

 

As reported operating expenses according to GAAP

$312

$291

$591

$560

Excluding compensation expense related to equity awards

(30)

(31)

(59)

(62)

Non-GAAP operating expenses

$282

$260

$532

$498

 

 

 

 

 

 

As reported income (loss) from operations according to GAAP

$140

($66)

($7)

($215)

Excluding compensation expense related to equity awards

(30)

(31)

(59)

(62)

Non-GAAP income (loss) from operations

$170

($35)

$52

($153)

 

 

 

 

 

 

As reported net income (loss) according to GAAP

$124

($66)

($23)

($209)

Excluding compensation expense related to equity awards and related tax effects

(30)

(31)

(59)

(62)

Non-GAAP net income (loss)

$154

($35)

$36

($147)

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.

IONIS PHARMACEUTICALS, INC.

Condensed Consolidated Balance Sheets

(In Millions)

 

 

 

 

 

 

 

June 30,

 

December 31,

 

 

2025

 

2024

 

 

(unaudited)

 

 

Assets:

 

 

 

 

Cash, cash equivalents and short-term investments

 

$2,290

 

$2,298

Contracts receivable

 

53

 

92

Other current assets

 

235

 

230

Property, plant and equipment, net

 

112

 

94

Right-of-use assets

 

164

 

162

Other assets

 

131

 

127

Total assets

 

$2,985

 

$3,003

 

 

 

 

 

Liabilities and stockholders’ equity:

 

 

 

 

Current portion of deferred contract revenue

 

$76

 

$79

0% convertible senior notes, net – current

 

630

 

-

Other current liabilities

 

191

 

229

1.75% convertible senior notes, net

 

566

 

565

0% convertible senior notes, net

 

-

 

629

Liability related to sale of future royalties, net

 

541

 

542

Long-term lease liabilities

 

164

 

162

Long-term obligations, less current portion

 

60

 

52

Long-term deferred contract revenue

 

125

 

157

Total stockholders’ equity

 

632

 

588

Total liabilities and stockholders’ equity

 

$2,985

 

$3,003

Key 2025 and 2026 Value Driving Events(1)

New Product Launches

Program

Indication

2025

2026

Donidalorsen (U.S.)

HAE

•

 

TRYNGOLZA (U.S.)

FCS

Achieved

 

WAINZUA (EU)

ATTRv-PN

Achieved

 

Olezarsen (U.S.)

sHTG

 

•

Zilganersen (U.S.)

Alexander disease

 

•

Regulatory Actions

Program

Indication

Regulatory Action

2025

2026

Donidalorsen

HAE

U.S. approval decision

•

 

EU approval decision

 

•

TRYNGOLZA

FCS

EU approval decision

•

 

Olezarsen

sHTG

U.S. submission

•

 

U.S. approval decision

 

•

Zilganersen

Alexander disease

U.S. submission

 

•

U.S. approval decision

 

•

Nusinersen

(higher dose)

SMA

U.S. and EU submissions

Achieved

 

U.S. approval decision

•

 

WAINZUA

ATTRv-PN

EU approval decision

Achieved

 

Pelacarsen

Lp(a)- CVD

U.S. submission

 

•

Bepirovirsen

HBV

Regulatory submission(s)

 

•

Regulatory decision(s)

 

•

Key Phase 3 Clinical Events

Program

Indication

Event

2025

2026

Olezarsen

sHTG

CORE, CORE2 data

•

 

Essence data

Achieved

 

Zilganersen

Alexander disease

Phase 3 data

•

 

ION582

Angelman syndrome

Phase 3 study start

Achieved

 

Phase 3 enrollment completion

 

•

Pelacarsen

Lp(a)-CVD

Lp(a) HORIZON data

 

•

Bepirovirsen

HBV

B-Well data

 

•

Eplontersen

ATTR-CM

CARDIO-TTRansform data

 

•

Sefaxersen

IgAN

IMAGINATION data

 

•

Ulefnersen

FUS-ALS

FUSION data

 

•

(1)

Timing expectations based on current assumptions and subject to change.

  • Indicates that the milestone is anticipated in the respective year. 

 

Ionis Investor Contact: D. Wade Walke, Ph.D. IR@ionis.com 760-603-233

Ionis Media Contact: Hayley Soffer media@ionis.com 760-603-4679

Source: Ionis Pharmaceuticals, Inc.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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