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000-19125
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33-0336973 |
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(Commission File No.)
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(IRS Employer Identification No.)
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| ☐ |
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)
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| ☐ |
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))
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| ☐ |
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
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Trading symbol
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Name of each exchange on which registered
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||
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Common Stock, $.001 Par Value
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“IONS”
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The Nasdaq Stock Market, LLC
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Emerging growth company
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☐
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| Item 2.02 |
Results of Operations and Financial Condition.
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| Item 9.01. |
Financial Statements and Exhibits.
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Exhibit No.
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Description
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|
Press Release dated April 29, 2026.
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|
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104
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Cover Page Interactive Data File (embedded within the Inline XBRL document).
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Ionis Pharmaceuticals, Inc.
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||
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Dated: April 29, 2026
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By:
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/s/ Patrick R. O’Neil
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Patrick R. O’Neil
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||
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Executive Vice President, Chief Legal Officer and General Counsel
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||
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Three months
ended
March 31,
|
||||||||
|
2026
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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.
|
| • |
Revenue increased 87% in the first quarter of 2026 compared to the same period last year, driven by continued commercial success. In addition, Ionis earned substantial R&D revenue, including $95 million in payments from both clinical
and regulatory milestones from multiple partnerships
|
| • |
Operating expenses for the quarter ended March 31, 2026 were in line with expectations and increased year over year primarily from investments related to the commercialization efforts for TRYNGOLZA and DAWNZERA as well as launch
preparations for olezarsen in sHTG and zilganersen in Alexander disease
|
| • |
Cash and short-term investments were $1.9 billion as of March 31, 2026. The change in cash and short-term investments from year end 2025 was primarily related to the $633 million the Company used for the maturity of the 0% convertible
notes due on April 1, 2026
|
| • |
Increasing annual olezarsen peak net sales guidance to >$3 billion from >$2 billion to reflect increasing confidence in the sHTG market opportunity for olezarsen
|
| • |
TRYNGOLZA® (olezarsen), the first FDA-approved treatment for adults living with familial chylomicronemia syndrome (FCS) as an adjunct to diet
|
| o |
Generated U.S. net product sales of $27 million in the first quarter of 2026, reflecting continued strong demand, offset by a decrease in net price
|
| o |
Launch initiated in the European Union (EU) by Sobi
|
| • |
Olezarsen on track to launch this year as a transformational medicine for severe hypertriglyceridemia (sHTG), assuming approval
|
| o |
sNDA accepted by the FDA for Priority Review for the treatment of sHTG with a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026
|
| o |
The European Medicines Agency (EMA) accepted an indication extension application in March for the treatment of adult patients with sHTG
|
| • |
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 $16 million in the first quarter of 2026, an increase of 125% versus the fourth quarter of 2025
|
| o |
Launch initiated in the EU by Otsuka
|
| o |
Positive one-year results from OASISplus open-label extension cohort published in the Journal of Asthma and Allergy
|
| • |
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) for AxD accepted by FDA for Priority Review with PDUFA target action date of September 22, 2026
|
| o |
Expanded access program (EAP) in U.S. underway
|
| o |
Positive additional results from the pivotal study presented at the American Academy of Neurology 2026 annual meeting
|
| • |
SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $374
million in the first quarter of 2026, resulting in royalty revenue of $44 million
|
| o |
SPINRAZA high dose regimen approved and launched in the U.S. and EU
|
| • |
WAINUA® (eplontersen) (WAINZUA in EU) for the treatment of adults
with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) generated global sales of $51 million in the first quarter of 2026, resulting in royalty revenue of $11 million
|
| o |
Launches underway in numerous regions, including the EU and China; submissions in progress to expand WAINUA access globally
|
| o |
Phase 3 CARDIO-TTRansform study design and baseline characteristics to be presented at the Annual Congress of the Heart Failure Association of the ESC 2026
|
| • |
Bepirovirsen, a potential first-in-class medicine for chronic hepatitis B (CHB), achieved the primary endpoint demonstrating a statistically significant and clinically meaningful functional cure rate in the B-Well 1 and B-Well 2 Phase 3
studies
|
| o |
GSK to present the positive Phase 3 data at the European Association for the Study of the Liver (EASL) Congress 2026
|
| o |
On track for a 2026 launch with global regulatory filings underway, assuming approval
|
| ■ |
NDA filing accepted by FDA for Priority Review with PDUFA date of October 26, 2026; granted Breakthrough Therapy designation
|
| ■ |
Accepted for regulatory review in EU, Japan, and China
|
|
Three months ended
|
||||||||
|
March 31,
|
||||||||
|
2026
|
2025
|
|||||||
|
Revenue
|
(amounts in millions)
|
|||||||
|
Commercial revenue:
|
||||||||
|
Product sales, net:
|
||||||||
|
TRYNGOLZA sales, net
|
$
|
27
|
$
|
6
|
||||
|
DAWNZERA sales, net
|
16
|
-
|
||||||
|
Total product sales, net
|
43
|
6
|
||||||
|
Royalty revenue:
|
||||||||
|
SPINRAZA royalties
|
44
|
48
|
||||||
|
WAINUA royalties
|
11
|
9
|
||||||
|
Other royalties
|
3
|
7
|
||||||
|
Total royalty revenue
|
58
|
64
|
||||||
|
Other commercial revenue
|
7
|
6
|
||||||
|
Total commercial revenue
|
108
|
76
|
||||||
|
Research and development revenue:
|
||||||||
|
Collaborative agreement revenue
|
120
|
46
|
||||||
|
WAINUA joint development revenue
|
18
|
10
|
||||||
|
Total research and development revenue
|
138
|
56
|
||||||
|
Total revenue
|
$
|
246
|
$
|
132
|
||||
|
Full Year 2026 Guidance
|
Previous
Guidance
|
New
Guidance
|
|
Total Revenue
|
$800-825 million
|
$875-900 million
|
|
TRYNGOLZA product sales, net
|
NA
|
$100-110 million
|
|
DAWNZERA product sales, net
|
NA
|
$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
|
>$1.6 billion
|
|
Three months ended
|
||||||||
|
March 31,
|
||||||||
|
2026
|
2025
|
|||||||
|
(unaudited)
|
||||||||
|
Revenue:
|
||||||||
|
Commercial revenue:
|
||||||||
|
Product sales, net
|
$
|
43
|
$
|
6
|
||||
|
Royalty revenue
|
58
|
64
|
||||||
|
Other commercial revenue
|
7
|
6
|
||||||
|
Total commercial revenue
|
108
|
76
|
||||||
|
Research and development revenue:
|
||||||||
|
Collaborative agreement revenue
|
120
|
46
|
||||||
|
WAINUA joint development revenue
|
18
|
10
|
||||||
|
Total research and development revenue
|
138
|
56
|
||||||
|
Total revenue
|
246
|
132
|
||||||
|
Expenses:
|
||||||||
|
Cost of sales
|
3
|
1
|
||||||
|
Research, development and patent
|
210
|
201
|
||||||
|
Selling, general and administrative
|
151
|
76
|
||||||
|
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
|
42
|
18
|
||||||
|
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
|
||||||
|
Three months ended
March 31,
|
||||||||
|
2026
|
2025
|
|||||||
|
(unaudited)
|
||||||||
|
As reported cost of sales according to GAAP
|
$
|
3
|
$
|
1
|
||||
|
Excluding compensation expense related to equity awards (1)
|
-
|
-
|
||||||
|
Non-GAAP cost of sales
|
$
|
3
|
$
|
1
|
||||
|
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.
|
|
March 31,
|
December 31,
|
|||||||
|
2026
|
2025
|
|||||||
|
(unaudited)
|
||||||||
|
Assets:
|
||||||||
|
Cash, cash equivalents and short-term investments
|
$
|
1,919
|
$
|
2,677
|
||||
|
Escrow deposits
|
633
|
-
|
||||||
|
Contracts receivable
|
74
|
66
|
||||||
|
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
|
28
|
28
|
||||||
|
Long-term deferred contract revenue
|
76
|
92
|
||||||
|
Total stockholders’ equity
|
491
|
489
|
||||||
|
Total liabilities and stockholders’ equity
|
$
|
3,450
|
$
|
3,524
|
||||
|
New Product Launches
|
|||||
|
Program
|
Indication
|
Location
|
|||
|
DAWNZERA
|
HAE
|
EU
|
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 2 Clinical Events
|
||||||
|
Program
|
Indication
|
Event
|
||||
|
IONIS-MAPTRx/ 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.
|
| • |
Indicates that the milestone is anticipated in 2026.
|