Full Press Release Details
Ionis reports second quarter 2025 financial results and highlights progress on key programs
- 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., July 30, 2025 - 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):
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| 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 | $ | 26 0 | $ | 532 | $ | 498 | ||||||||
| Income (loss) from operations | $ | 140 | $ | (66 | ) | $ | (7 | ) | $ | (215 | ) | |||||
| Income (loss) from operations on a non-GAAP basis | $ | 170 | $ | (35 | ) | $ | 52 | $ | (153 | ) |
Recent Financial Highlights
| 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 | <$375 million | $300-325 million |
| Cash, cash equivalents and short-term investments | ~$1.9 billion | ~$2.0 billion |
Second Quarter 2025 Financial Results
"For the second time this year, we are significantly raising our 2025 financial guidance - this time driven by an improved outlook for the year and strong revenue
performance to date, which includes the early launch excellence with TRYNGOLZA. In addition to strong commercial performance, our second quarter results included the substantial revenue we earned
from licensing sapablursen, a medicine outside our core areas of focus. We are in a strong financial position, with a commitment to drive operating leverage as we continue executing on our
strategic priorities," said Elizabeth L. Hougen, chief financial officer, Ionis. "Moving forward, the three additional independent launches anticipated over the next eighteen months, including donidalorsen for hereditary angioedema, olezarsen in
severe hypertriglyceridemia and zilganersen in Alexander disease, position Ionis to deliver substantial and growing product revenue. This product revenue, coupled with anticipated increasing
royalty revenue from multiple partner launches, along with disciplined investment, position Ionis to achieve sustained growth and positive cash flow in the next few years."
Recent Highlights - Wholly Owned Medicines
Recent Highlights - Partnered Medicines
Ionis' revenue was comprised of the following:
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| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| Revenue: | (amounts in millions) | |||||||||||||||
| Commercial revenue: | ||||||||||||||||
| Product sales, net: | ||||||||||||||||
| TRYNGOLZA sales, net | $ | 19 | $ | - | $ | 26 | $ | - | ||||||||
| Total product sales, net | 19 | - | 26 | - | ||||||||||||
| Royalty revenue: | ||||||||||||||||
| SPINRAZA royalties | 54 | 57 | 102 | 95 | ||||||||||||
| WAINUA royalties | 10 | 4 | 20 | 5 | ||||||||||||
| Other royalties | 6 | 3 | 12 | 13 | ||||||||||||
| Total royalty revenue | 70 | 64 | 134 | 113 | ||||||||||||
| Other commercial revenue: | ||||||||||||||||
| TEGSEDI and WAYLIVRA revenue, net | 14 | 8 | 19 | 17 | ||||||||||||
| Other revenue | - | - | - | 2 | ||||||||||||
| 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 |
Commercial revenue for the second quarter and first half of 2025 increased 43% and 36% respectively, compared to the same periods in 2024. This increase was driven by
TRYNGOLZA product sales. Higher royalty revenue also contributed to the year over year increase.
The remainder of the Company's revenue came from programs under its R&D collaborations, including a $280 million upfront payment for the global license of
sapablursen to Ono Pharmaceutical Co., Ltd., reflecting the value that Ionis' pipeline and technology continues to generate.
SG&A expenses increased as anticipated for the second quarter and first half of 2025, compared to the same
periods in 2024, primarily due to the launches of TRYNGOLZA and WAINUA, and advancing launch preparation activities for donidalorsen. This increase was partially offset by a decrease in R&D expenses as several late-stage studies ended. Overall,
this led to a modest year-over-year increase in total operating expenses.
As of June 30, 2025, Ionis' cash, cash equivalents and short-term investments were $2.3
billion, consistent with December 31, 2024. Ionis received $280 million from the global license of sapablursen in the second quarter of 2025. Ionis' working capital decreased over the same period primarily due to the reclassification of the Company's
0% convertible notes as a current liability.
Webcast and Other Updates
Management will host a conference call and webcast to discuss Ionis' second quarter 2025 results at 11:30 a.m. Eastern time on Wednesday, July 30, 2025. 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 2025 earnings slides click here.
Ionis will be initiating a quiet period starting July 31, 2025, as the Company plans to announce the topline results from both the CORE and CORE2 studies simultaneously.
The quiet period will be lifted upon the data announcement, expected in September.
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
IMPORTANT SAFETY INFORMATION
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.
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 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.
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
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 Investor Contact:
D. Wade Walke, Ph.D.
Ionis Media Contact:
IONIS PHARMACEUTICALS, INC.
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
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| (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)
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| (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 | $ | 26 0 | $ | 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.