- 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.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results and provided key updates for the second quarter ended June 30, 2026.
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“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
Six months ended
June 30,
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.
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
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
sHTG U.S. launch demonstrating early momentum following June 2026 approval
FCS launch outside the U.S. underway; sHTG marketing application under review in the European Union (EU) with potential launch in 2027
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
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
On track to achieve full year 2026 product sales of $110-120 million, representing a continuing driver of year-over-year revenue growth
Launch outside the U.S. underway, with new market approvals in the EU
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
New Drug Application (NDA) under FDA Priority Review with PDUFA target action date of September 22, 2026
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
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
Global WAINUA ATTRv-PN launches continuing with additional submissions in progress to further expand global WAINUA access
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
Results from the CARDIO-TTRansform study will be presented at ESC in August 2026
Bepirovirsen, a potential first-in-class treatment of chronic hepatitis B (CHB), on track for global launch this year
Under regulatory review in multiple countries, including the U.S., EU, Japan, and China
Granted Priority Review in the U.S. and PDUFA target action date of October 26, 2026
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-MAPT
Rx
/ 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
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.
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
and
, 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 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 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. 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
June 30,
Six months ended
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
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.
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,
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
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.
Contacts
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
