|
|
|
|
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(Commission File No.)
|
(IRS Employer Identification No.)
|
|
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
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|
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))
|
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Title of each class
|
Trading symbol
|
Name of each exchange on which registered
|
||
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|
“
|
|
|
Emerging growth company
|
|
| Item 2.02 |
Results of Operations and Financial Condition.
|
| Item 9.01. |
Financial Statements and Exhibits.
|
|
Exhibit No.
|
Description
|
|
Press Release dated July 29, 2026.
|
|
|
104
|
Cover Page Interactive Data File (embedded within the Inline XBRL document).
|
|
Ionis Pharmaceuticals, Inc.
|
||
|
Dated: July 29, 2026
|
By:
|
/s/ Patrick R. O’Neil
|
|
Patrick R. O’Neil
|
||
|
Executive Vice President, Chief Legal Officer and General Counsel
|
||
|
Three months ended
June 30,
|
Six months ended
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.
|
| • |
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
|
| • |
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
|
| o |
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
|
| o |
sHTG U.S. launch demonstrating early momentum following June 2026 approval
|
| o |
FCS launch outside the U.S. underway; sHTG marketing application under review in the European Union (EU) with potential launch in 2027
|
| o |
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
|
| o |
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
|
| o |
On track to achieve full year 2026 product sales of $110-120 million, representing a continuing driver of year-over-year revenue growth
|
| o |
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
|
| o |
New Drug Application (NDA) under FDA Priority Review with PDUFA target action date of September 22, 2026
|
| o |
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
|
| o |
Results from the Phase 1 study of ION775 will be presented at ESC in August 2026
|
| • |
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
|
| o |
Global WAINUA ATTRv-PN launches continuing with additional submissions in progress to further expand global WAINUA access
|
| o |
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
|
| o |
Results from the CARDIO-TTRansform study will be presented at the ESC Congress in August 2026
|
| • |
Bepirovirsen, a potential first-in-class treatment of chronic hepatitis B (CHB), on track for global launch this year
|
| o |
Under regulatory review in multiple countries, including the U.S., EU, Japan, and China
|
| o |
Granted Priority Review in the U.S. and PDUFA target action date of October 26, 2026
|
| o |
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-MAPTRx / 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
|
|
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
|
||||||||
|
Three months ended
|
Six months ended
|
|||||||||||||||
|
June 30,
|
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
|
||||||||||||
|
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.
|
|
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
|
||||
|
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.
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| • |
Indicates that the milestone is anticipated in 2026.
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