Ionis Pharmaceuticals Inc 8-K, filed Apr 30, 2025
Ionis Pharmaceuticals Inc (IONS)
8-K
2025-04-30
For: 2025-04-30
View Original
Added on
April 07, 2026
About this 8-K
Ionis Pharmaceuticals Inc filed this 8-K on April 30, 2025.
Items reported
- Item 2.02: Financial results
- Item 9.01: Financial statements and exhibits
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
FORM 8-K
CURRENT REPORT
PURSUANT TO SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of report (Date of earliest event reported): April 30, 2025
(Exact Name of Registrant as Specified in Charter)
(State or Other Jurisdiction of Incorporation)
|
(Commission File No.)
|
(IRS Employer Identification No.)
|
(Address of Principal Executive Offices and Zip Code)
Registrant’s telephone number, including area code: (760 ) 931-9200
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following
provisions:
|
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)
|
|
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))
|
Securities registered pursuant to Section 12(b) of the Act:
|
Title of each class
|
Trading symbol
|
Name of each exchange on which registered
|
||
|
|
|
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (Section 230.405 of this chapter) or
Rule 12b-2 of the Securities Exchange Act of 1934 (Section 240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised
financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
| Item 2.02 |
Results of Operations and Financial Condition.
|
On April 30, 2025, Ionis Pharmaceuticals, Inc. (the “Company”) issued a press release announcing the
Company’s financial results for the quarter ended March 31, 2025. In addition to disclosing results that are determined in accordance with Generally Accepted Accounting Principles (“GAAP”),
the Company also discloses pro forma or non-GAAP results of operations, which are adjusted from GAAP to exclude non-cash compensation expense related to equity awards and the related tax effects. The Company is
presenting pro forma information excluding non-cash compensation expense and the related tax effects because the Company believes it better enables financial statement users to assess and compare its historical performance and project its future
operating results and cash flows. A copy of the release is furnished with this report as an exhibit pursuant to “Item 2.02. Results of Operations and Financial Condition” of Form 8-K in accordance with SEC Release Nos. 33-8216 and 34-47583.
The information in this Current Report on Form 8-K and the Exhibit attached hereto shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange
Act of 1934 (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933 or
the Exchange Act, regardless of any general incorporation language in such filing.
| Item 9.01. |
Financial Statements and Exhibits.
|
(d) Exhibits.
|
Exhibit No.
|
Description
|
|
Press Release dated April 30, 2025.
|
|
|
104
|
Cover Page Interactive Data File (embedded within the Inline XBRL document).
|
SIGNATURE
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its
behalf by the undersigned, thereunto duly authorized.
|
Ionis Pharmaceuticals, Inc.
|
||
|
Dated: April 30, 2025
|
By:
|
/s/ Patrick R. O’Neil |
|
Patrick R. O’Neil
|
||
|
Executive Vice President, Chief Legal Officer and General Counsel
|
Exhibit 99.1
Ionis reports first quarter 2025 financial results
- Encouraging start to first
independent launch with TRYNGOLZATM -
- On track for second
independent launch with donidalorsen PDUFA August 21, 2025 -
- Increasing 2025 financial
guidance by more than 20% -
CARLSBAD, Calif., April 30, 2025 – Ionis
Pharmaceuticals, Inc. (Nasdaq: IONS) (the “Company”) today reported financial results for the first quarter ended March 31, 2025.
“With an encouraging start to the TRYNGOLZA launch for familial chylomicronemia syndrome, the first of four independent launches expected over the next two years,
Ionis’ new chapter as a fully integrated, commercial-stage biotechnology company is well underway,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “We look forward to continued momentum this year, including our second independent
launch for donidalorsen in hereditary angioedema and Phase 3 results for olezarsen for severe hypertriglyceridemia in the third quarter. We also continue to advance our next wave of wholly owned neurology medicines, including ION582 for Angelman
syndrome, which is on track to start Phase 3 development shortly. Our advancing pipeline of transformational medicines, together with strong commercial and financial execution, position Ionis to deliver increasing value for all stakeholders.”
First Quarter 2025 Summary Financial Results(1):
|
Three months
ended
March 31,
|
||||||||
|
2025
|
2024
|
|||||||
|
(amounts in millions)
|
||||||||
|
Total revenue
|
$
|
132
|
$
|
119
|
||||
|
Operating expenses
|
$
|
278
|
$
|
269
|
||||
|
Operating expenses on a non-GAAP basis
|
$
|
249
|
$
|
238
|
||||
|
Loss from operations
|
$
|
(146
|
)
|
$
|
(150
|
)
|
||
|
Loss from operations on a non-GAAP basis
|
$
|
(117
|
)
|
$
|
(119
|
)
|
||
| (1) |
Reconciliation of GAAP to non-GAAP basis contained later in this release.
|
Recent Financial Highlights
| • |
Revenue increased 10% in the first quarter of 2025 compared to the same period last year, driven by higher commercial revenue including new TRYNGOLZA product revenue following approval in late
December and higher SPINRAZA and WAINUA royalty revenue
|
1
| • |
Operating expenses increased slightly in the first quarter of 2025 driven by commercialization efforts for TRYNGOLZA, donidalorsen and WAINUA
|
| • |
Increased 2025 financial guidance reflects recent successful licensing transactions:
|
|
Full Year 2025 Guidance
|
Previous
Guidance
|
New
Guidance
|
|
Revenue
|
>$600 million
|
$725-750 million
|
|
Operating loss on a non-GAAP basis
|
<$495 million
|
<$375 million
|
|
Cash, cash equivalents and short-term investments
|
~$1.7 billion
|
~$1.9 billion
|
Recent Highlights - Marketed Medicines
| • |
TRYNGOLZATM (olezarsen), the first-ever treatment for adults living with familial chylomicronemia syndrome (FCS) as an adjunct to
diet, generated net product sales of over $6 million in its first full quarter following approval in the U.S. on December 19, 2024
|
| o |
Commercialization rights in countries outside of the U.S., Canada and China licensed to Sobi
|
| o |
EU approval decision anticipated in H2:2025
|
| • |
WAINUATM (eplontersen) (WAINZUA in EU) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) achieved commercial and regulatory
milestones:
|
| o |
Generated sales of $39 million resulting in royalty revenue of $9 million in the first quarter of 2025
|
| o |
Launch underway in numerous countries, including the EU following approval by the European Commission (EC); additional global submissions in progress to expand WAINUA access
|
| • |
SPINRAZA® (nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $424 million resulting in royalty revenue of $48 million in the first quarter of 2025
|
| o |
Higher dose nusinersen under review for marketing approval in U.S. (PDUFA date of September 22, 2025) and EU
|
Recent Highlights - Late-Stage Wholly Owned Pipeline
| • |
Olezarsen on track for topline Phase 3 data from supportive ESSENCE study in Q2:2025 and pivotal CORE and CORE2 studies in patients with severe hypertriglyceridemia (sHTG) in Q3:2025, positioning olezarsen to potentially treat this second, more prevalent patient population with high unmet need
|
| o |
Published Phase 3 study design and baseline characteristics for CORE, CORE2 and ESSENCE studies in the American Heart Journal, highlighting the potential to generate robust and meaningful data
|
| • |
Donidalorsen on track for launch this year as the first RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE), assuming approval:
|
| o |
Under review for marketing approval in U.S. (PDUFA date of August 21, 2025) and EU
|
| o |
Presented positive Phase 2 open label extension (OLE) study data demonstrating an overall sustained mean reduction in HAE attack rates of 96% in patients treated up to 196 weeks with every four
weeks or every eight weeks dosing
|
2
| o |
Published positive patient-reported outcomes (PROs) from Phase 3 OASIS-HAE study in Allergy showing donidalorsen significantly improved quality of life (QoL) and other PROs compared to placebo
|
| • |
ION582 on track to initiate Phase 3 development in Angelman syndrome (AS) in Q2:2025
|
Recent Highlights - Partnered Pipeline
| • |
Pelacarsen Phase 3 Lp(a) HORIZON study design and baseline characteristics published in the American Heart Journal
highlighting the potential to generate robust and meaningful data
|
| • |
Licensed sapablursen global development and commercialization rights to Ono Pharmaceutical Co. generating $280 million in an upfront payment, with the potential to earn up to $660 million in
additional payments plus royalties in the mid-teen percentage range on annual net sales
|
| • |
IONIS-MAPTRx (BIIB080)
received FDA Fast Track designation for the treatment of Alzheimer’s disease
|
First Quarter 2025 Financial Results
“We are increasing our 2025 financial guidance including raising revenue guidance by more than 20 percent due to our strong first quarter results and recent successful
licensing transactions. We are also substantially improving our operating loss and cash guidance and now expect to end the year with approximately $1.9 billion in cash. Our strong financial
position and commitment to drive operating leverage position Ionis to advance our strategic priorities and successfully navigate the dynamic macroeconomic environment,” said Elizabeth L. Hougen, chief financial officer, Ionis. “Our first quarter
results reflected encouraging early performance in the TRYNGOLZA launch, adding product revenue to our P&L for the first time. Moving forward, the three additional independent launches anticipated over the next couple of years position Ionis to
deliver substantial and growing product revenue. This product revenue coupled with anticipated increasing royalty revenue from multiple partner launches and disciplined investment, position Ionis to achieve sustained positive cash flow.”
Revenue
Ionis’ revenue was comprised of the following:
|
Three months ended
|
||||||||
|
March 31,
|
||||||||
|
2025
|
2024
|
|||||||
|
Revenue
|
(amounts in millions)
|
|||||||
|
Commercial revenue:
|
||||||||
|
Product sales, net:
|
||||||||
|
TRYNGOLZA sales, net
|
$
|
6
|
$
|
-
|
||||
|
Total product sales, net
|
6
|
-
|
||||||
|
Royalty revenue:
|
||||||||
|
SPINRAZA royalties
|
48
|
38
|
||||||
|
WAINUA royalties
|
9
|
1
|
||||||
|
Other royalties
|
7
|
10
|
||||||
|
Total royalty revenue
|
64
|
49
|
||||||
|
Other commercial revenue:
|
||||||||
|
TEGSEDI and WAYLIVRA revenue, net
|
6
|
9
|
||||||
|
Other revenue
|
-
|
1
|
||||||
|
Total other commercial revenue
|
6
|
10
|
||||||
|
Total commercial revenue
|
76
|
59
|
||||||
|
Research and development revenue:
|
||||||||
|
Collaborative agreement revenue
|
46
|
49
|
||||||
|
WAINUA joint development revenue
|
10
|
11
|
||||||
|
Total research and development revenue
|
56
|
60
|
||||||
|
Total revenue
|
$
|
132
|
$
|
119
|
||||
3
Commercial revenue for the first quarter of 2025 increased 28% compared to the same period in 2024, driven in part by revenue from U.S. product sales from the launch
of TRYNGOLZA. Higher royalty revenues from SPINRAZA, WAINUA and QALSODY also contributed to the year over year increase.
The remainder of the Company’s revenue came from programs under its R&D collaborations, reflecting the value that Ionis’ pipeline and technology continues to
generate.
Operating Expenses
SG&A expenses increased in the first quarter of 2025 compared to the same period in 2024 primarily due to the launches of WAINUA and TRYNGOLZA, 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 slight increase in total operating expenses.
Balance Sheet
As of March 31, 2025, Ionis’ cash, cash equivalents and short-term investments were $2.1 billion, compared to $2.3 billion at December 31,
2024. Ionis’ working capital decreased over the same period primarily due to the Company’s lower cash and short-term investments balance. Ionis generated $280 million from the global license of sapablursen in the second quarter.
Webcast
Management will host a conference call and webcast to discuss Ionis’ first quarter 2025 results at 11:30 a.m. Eastern time on Wednesday, April 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 first quarter 2025 earnings slides click here.
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
syndrome (FCS).
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
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.
4
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.
ADVERSE REACTIONS
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.
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 six marketed medicines and a leading
pipeline in neurology, cardiology 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.
5
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 Pharmaceuticals® is a registered trademark of Ionis Pharmaceuticals, Inc. TRYNGOLZATM is a trademark of Ionis Pharmaceuticals, Inc. Akcea
Therapeutics® is a registered trademark of Akcea Therapeutics, Inc. TEGSEDI® is a registered trademark of Akcea Therapeutics, Inc. WAYLIVRA® is a registered trademark of Akcea Therapeutics, Inc. SPINRAZA®
and QALSODY® are registered trademarks of Biogen. WAINUATM is a registered trademark of the AstraZeneca group of companies.
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
6
SELECTED FINANCIAL INFORMATION
Condensed Consolidated Statements of Operations
(In Millions, Except Per Share Data)
|
Three months ended
|
||||||||
|
March 31,
|
||||||||
|
2025
|
2024
|
|||||||
|
(unaudited)
|
||||||||
|
Revenue:
|
||||||||
|
Commercial revenue:
|
||||||||
|
Product sales, net
|
$
|
6
|
$
|
-
|
||||
|
Royalty revenue
|
64
|
49
|
||||||
|
Other commercial revenue
|
6
|
10
|
||||||
|
Total commercial revenue
|
76
|
59
|
||||||
|
Research and development revenue:
|
||||||||
|
Collaborative agreement revenue
|
46
|
49
|
||||||
|
WAINUA joint development revenue
|
10
|
11
|
||||||
|
Total research and development revenue
|
56
|
60
|
||||||
|
Total revenue
|
132
|
119
|
||||||
|
Expenses:
|
||||||||
|
Cost of sales
|
1
|
2
|
||||||
|
Research, development and patent
|
201
|
214
|
||||||
|
Selling, general and administrative
|
76
|
53
|
||||||
|
Total operating expenses
|
278
|
269
|
||||||
|
Loss from operations
|
(146
|
)
|
(150
|
)
|
||||
|
Other income (expense):
|
||||||||
|
Interest expense related to the sale of future royalties
|
(19
|
)
|
(18
|
)
|
||||
|
Other income, net
|
18
|
25
|
||||||
|
Loss before income tax expense
|
(147
|
)
|
(143
|
)
|
||||
|
Income tax expense
|
-
|
-
|
||||||
|
Net loss
|
$
|
(147
|
)
|
$
|
(143
|
)
|
||
|
Basic and diluted net loss per share
|
$
|
(0.93
|
)
|
$
|
(0.98
|
)
|
||
|
Shares used in computing basic and diluted net loss per share
|
159
|
146
|
||||||
7
IONIS PHARMACEUTICALS, INC.
Reconciliation of GAAP to Non-GAAP Basis:
Condensed Consolidated Operating Expenses, Loss From Operations, and Net Loss
(In Millions)
|
Three months ended
March 31,
|
||||||||
|
2025
|
2024
|
|||||||
|
(unaudited)
|
||||||||
|
As reported research, development and patent expenses according to GAAP
|
$
|
201
|
$
|
214
|
||||
|
Excluding compensation expense related to equity awards
|
(20
|
)
|
(22
|
)
|
||||
|
Non-GAAP research, development and patent expenses
|
$
|
181
|
$
|
192
|
||||
|
As reported selling, general and administrative expenses according to GAAP
|
$
|
76
|
$
|
53
|
||||
|
Excluding compensation expense related to equity awards
|
(9
|
)
|
(9
|
)
|
||||
|
Non-GAAP selling, general and administrative expenses
|
$
|
67
|
$
|
44
|
||||
|
As reported operating expenses according to GAAP
|
$
|
278
|
$
|
269
|
||||
|
Excluding compensation expense related to equity awards
|
(29
|
)
|
(31
|
)
|
||||
|
Non-GAAP operating expenses
|
$
|
249
|
$
|
238
|
||||
|
As reported loss from operations according to GAAP
|
$
|
(146
|
)
|
$
|
(150
|
)
|
||
|
Excluding compensation expense related to equity awards
|
(29
|
)
|
(31
|
)
|
||||
|
Non-GAAP loss from operations
|
$
|
(117
|
)
|
$
|
(119
|
)
|
||
|
As reported net loss according to GAAP
|
$
|
(147
|
)
|
$
|
(143
|
)
|
||
|
Excluding compensation expense related to equity awards and related tax effects
|
(29
|
)
|
(31
|
)
|
||||
|
Non-GAAP net loss
|
$
|
(118
|
)
|
$
|
(112
|
)
|
||
Reconciliation of GAAP to Non-GAAP Basis
As illustrated in the Selected Financial Information in this press release, non-GAAP operating expenses, non-GAAP loss from operations, and non-GAAP net 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.
8
IONIS PHARMACEUTICALS, INC.
Condensed Consolidated Balance Sheets
(In Millions)
|
March 31,
|
December 31,
|
|||||||
|
2025
|
2024
|
|||||||
|
(unaudited)
|
||||||||
|
Assets:
|
||||||||
|
Cash, cash equivalents and short-term investments
|
$
|
2,145
|
$
|
2,298
|
||||
|
Contracts receivable
|
40
|
92
|
||||||
|
Other current assets
|
234
|
230
|
||||||
|
Property, plant and equipment, net
|
103
|
94
|
||||||
|
Right-of-use assets
|
159
|
162
|
||||||
|
Other assets
|
132
|
127
|
||||||
|
Total assets
|
$
|
2,813
|
$
|
3,003
|
||||
|
Liabilities and stockholders’ equity:
|
||||||||
|
Current portion of deferred contract revenue
|
$
|
81
|
$
|
79
|
||||
|
Other current liabilities
|
169
|
229
|
||||||
|
1.75% convertible senior notes, net
|
566
|
565
|
||||||
|
0% convertible senior notes, net
|
629
|
629
|
||||||
|
Liability related to sale of future royalties, net
|
536
|
542
|
||||||
|
Long-term lease liabilities
|
159
|
162
|
||||||
|
Long-term obligations, less current portion
|
56
|
52
|
||||||
|
Long-term deferred contract revenue
|
141
|
157
|
||||||
|
Total stockholders’ equity
|
476
|
588
|
||||||
|
Total liabilities and stockholders’ equity
|
$
|
2,813
|
$
|
3,003
|
||||
9
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 and Essence data
|
•
|
||||
|
Zilganersen
|
Alexander disease
|
Phase 3 data
|
•
|
||||
|
ION582
|
Angelman syndrome
|
Phase 3 study start
|
•
|
||||
|
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.
|
# # #
10