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| Share Name | Share Symbol | Market | Type |
|---|---|---|---|
| Ionis Pharmaceuticals Inc | NASDAQ:IONS | NASDAQ | Common Stock |
| Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
|---|---|---|---|---|---|---|---|---|---|---|
| 0.00 | 0.00% | 37.01 | 36.90 | 37.72 | 0 | 00:00:00 |
|
|
|
|
|
(Commission File No.)
|
(IRS Employer Identification No.)
|
|
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))
|
|
Title of each class
|
Trading symbol
|
Name of each exchange on which registered
|
||
|
|
“
|
|
|
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 November 6, 2024.
|
|
| 104 |
Cover Page Interactive Data File (embedded within the Inline XBRL document).
|
|
Ionis Pharmaceuticals, Inc.
|
||
|
Dated: November 6, 2024
|
By:
|
/s/ Patrick R. O’Neil
|
|
Patrick R. O’Neil
|
||
|
Executive Vice President, Chief Legal Officer and General Counsel
|
||
|
Three months
ended
September 30,
|
Nine months
ended
September 30,
|
|||||||||||||||
|
2024
|
2023
|
2024
|
2023
|
|||||||||||||
|
(amounts in millions)
|
||||||||||||||||
|
Total revenue
|
$
|
134
|
$
|
144
|
$
|
479
|
$
|
463
|
||||||||
|
Operating expenses
|
$
|
282
|
$
|
287
|
$
|
843
|
$
|
811
|
||||||||
|
Operating expenses on a non-GAAP basis
|
$
|
250
|
$
|
261
|
$
|
749
|
$
|
732
|
||||||||
|
Loss from operations
|
$
|
(148
|
)
|
$
|
(143
|
)
|
$
|
(364
|
)
|
$
|
(348
|
)
|
||||
|
Loss from operations on a non-GAAP basis
|
$
|
(116
|
)
|
$
|
(117
|
)
|
$
|
(270
|
)
|
$
|
(269
|
)
|
||||
| (1) |
Reconciliation of GAAP to non-GAAP basis contained later in this release.
|
| • |
Revenue for the three and nine months ended September 30, 2024 decreased by 7% and increased by 3% compared to the same periods last year, respectively. Ionis continued to
generate revenue from diverse sources, including a new source of royalty revenue with the launch of WAINUA in the U.S in the first quarter
|
| • |
Operating expenses for the three and nine months ended September 30, 2024 increased as planned compared to the same periods last year, excluding certain one-time costs in 2023,
reflecting continued investments in late-stage development, including WAINUA for ATTR cardiomyopathy (ATTR-CM) and olezarsen for severe hypertriglyceridemia (sHTG), and commercialization efforts for WAINUA, olezarsen and donidalorsen
|
| • |
Reaffirmed 2024 P&L financial guidance, increased cash guidance to $2.2 billion reflecting proceeds from equity offering
|
| • |
WAINUA (WAINZUA in Europe) for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis (ATTRv-PN) achieved multiple commercial and regulatory
milestones:
|
| o |
Generated sales of $23 million and $44 million resulting in royalty revenue of $5 million and $10 million in the three and nine months ended September 30, 2024, respectively
|
| o |
Received positive Committee for Medicinal Products for Human Use (CHMP) opinion from European Medicines Agency (EMA) for the treatment of hereditary transthyretin-mediated
amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
|
| o |
Approved in UK by the Medicines and Healthcare products Regulatory Agency (MHRA) with an accelerated National Institute for Health and Care Excellence (NICE) recommendation;
earning $30 million from AstraZeneca
|
| o |
Launch underway in Canada, following approval and reimbursement from Health Canada
|
| • |
SPINRAZA®
(nusinersen) for the treatment of spinal muscular atrophy (SMA) generated global sales of $381 million and $1.2 billion resulting in royalty revenue of $57 million and $152 million in the three and nine months ended September 30, 2024,
respectively
|
| o |
Positive Phase 2/3 DEVOTE study data presented from higher dose nusinersen; global regulatory applications planned
|
| • |
QALSODY®
(tofersen) granted marketing approval in China for the treatment of SOD1-ALS
|
| • |
Olezarsen positioned to potentially treat two patient populations with urgent unmet need, familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG):
|
| o |
NDA for patients with FCS under FDA Priority Review with a PDUFA date of December 19, 2024
|
| o |
Marketing authorization application (MAA) under regulatory review
by the EMA
|
| o |
Ongoing pivotal development program for sHTG on track for data in H2:2025
|
| • |
Donidalorsen Phase 3 data position it to potentially launch next year as the first RNA-targeted prophylactic treatment for people with hereditary angioedema (HAE):
|
| o |
FDA accepted the NDA for patients with HAE with a PDUFA date of August 21, 2025; Otsuka, Europe and Asia Pacific partner, preparing to submit MAA
|
| o |
Presented positive Phase 2 open label extension (OLE) study data in patients treated up to three years with every four weeks or every eight weeks dosing
|
| • |
Zilganersen Phase 3 study fully enrolled as a potential treatment for Alexander disease; on track for data in 2025
|
| o |
Granted Fast Track designation by the FDA
|
| • |
ION582 achieved important clinical and regulatory milestones enabling initiation of Phase 3 development in Angelman syndrome (AS) in H1:2025:
|
| o |
Presented positive Phase 1/2 data in patients with AS at Angelman Syndrome Foundation (ASF) Family Conference
|
| o |
Completed positive End-of-Phase 2 discussion with FDA, included alignment on Phase 3 design
|
| • |
Reported positive data from the Phase 2 study of IONIS-FB-LRx in patients with immunoglobulin A nephropathy (IgAN); Roche continues to advance IONIS-FB-LRx in
the Phase 3 IMAGINATION study
|
| • |
Sapablursen for the treatment of polycythemia vera granted orphan drug designation by FDA; enrollment complete in Phase 2 IMPRSSION study with data expected in 2025
|
| • |
IONIS-MAPTRx (BIIB080) enrollment complete in Phase 2 CELIA study in patients with early Alzheimer’s disease (AD); data expected in 2026
|
| • |
Initiated first in human studies with multiple medicines from neurological disease pipeline:
|
| o |
Phase 1/2 Orbit study of ION356 (PLP1) in patients with Pelizaeus-Merzbacher disease (PMD)
|
| o |
Phase 1/2 HERO study of ION269 (APP), for the potential treatment of Alzheimer’s disease (AD), which is initially being evaluated in patients with Down syndrome (DS) who have a
genetic risk for developing AD
|
| o |
Phase 1/2 ATTUNE study of ION440 (MECP2) in patients with MECP2 duplication syndrome
|
|
Three months ended
|
Nine months ended
|
|||||||||||||||
|
September 30,
|
September 30,
|
|||||||||||||||
|
2024
|
2023
|
2024
|
2023
|
|||||||||||||
|
Revenue:
|
(amounts in millions)
|
|||||||||||||||
|
Commercial revenue:
|
||||||||||||||||
|
SPINRAZA royalties
|
$
|
57
|
$
|
67
|
$
|
152
|
$
|
179
|
||||||||
|
WAINUA royalties
|
5
|
-
|
10
|
-
|
||||||||||||
|
Other commercial revenue:
|
||||||||||||||||
|
TEGSEDI and WAYLIVRA revenue, net
|
9
|
8
|
26
|
25
|
||||||||||||
|
Licensing and other royalty revenue
|
5
|
9
|
19
|
26
|
||||||||||||
|
Total commercial revenue
|
76
|
84
|
207
|
230
|
||||||||||||
|
Research and development revenue:
|
||||||||||||||||
|
Amortization from upfront payments
|
28
|
18
|
105
|
47
|
||||||||||||
|
Milestone payments
|
16
|
16
|
76
|
90
|
||||||||||||
|
License fees
|
-
|
5
|
38
|
25
|
||||||||||||
|
Other services
|
1
|
5
|
18
|
11
|
||||||||||||
|
Collaborative agreement revenue
|
45
|
44
|
237
|
173
|
||||||||||||
|
WAINUA joint development revenue
|
13
|
16
|
35
|
60
|
||||||||||||
|
Total research and development revenue
|
58
|
60
|
272
|
233
|
||||||||||||
|
Total revenue
|
$
|
134
|
$
|
144
|
$
|
479
|
$
|
463
|
||||||||
|
Three months ended
|
Nine months ended
|
|||||||||||||||
|
September 30,
|
September 30,
|
|||||||||||||||
|
2024
|
2023
|
2024
|
2023
|
|||||||||||||
|
(unaudited)
|
||||||||||||||||
|
Revenue:
|
||||||||||||||||
|
Commercial revenue:
|
||||||||||||||||
|
SPINRAZA royalties
|
$
|
57
|
$
|
67
|
$
|
152
|
$
|
179
|
||||||||
|
WAINUA royalties
|
5
|
-
|
10
|
-
|
||||||||||||
|
Other commercial revenue
|
14
|
17
|
45
|
51
|
||||||||||||
|
Total commercial revenue
|
76
|
84
|
207
|
230
|
||||||||||||
|
Research and development revenue:
|
||||||||||||||||
|
Collaborative agreement revenue
|
45
|
44
|
237
|
173
|
||||||||||||
|
WAINUA joint development revenue
|
13
|
16
|
35
|
60
|
||||||||||||
|
Total research and development revenue
|
58
|
60
|
272
|
233
|
||||||||||||
|
Total revenue
|
134
|
144
|
479
|
463
|
||||||||||||
|
Expenses:
|
||||||||||||||||
|
Cost of sales
|
1
|
2
|
7
|
6
|
||||||||||||
|
Research, development and patent
|
220
|
215
|
656
|
643
|
||||||||||||
|
Selling, general and administrative
|
61
|
70
|
180
|
162
|
||||||||||||
|
Total operating expenses
|
282
|
287
|
843
|
811
|
||||||||||||
|
Loss from operations
|
(148
|
)
|
(143
|
)
|
(364
|
)
|
(348
|
)
|
||||||||
|
Other income (expense):
|
||||||||||||||||
|
Interest expense related to the sale of future royalties
|
(19
|
)
|
(18
|
)
|
(55
|
)
|
(51
|
)
|
||||||||
|
Other income, net
|
23
|
20
|
66
|
68
|
||||||||||||
|
Loss before income tax benefit (expense)
|
(144
|
)
|
(141
|
)
|
(353
|
)
|
(331
|
)
|
||||||||
|
Income tax benefit (expense)
|
4
|
(6
|
)
|
3
|
(26
|
)
|
||||||||||
|
Net loss
|
$
|
(140
|
)
|
$
|
(147
|
)
|
$
|
(350
|
)
|
$
|
(357
|
)
|
||||
|
Basic and diluted net loss per share
|
$
|
(0.95
|
)
|
$
|
(1.03
|
)
|
$
|
(2.38
|
)
|
$
|
(2.50
|
)
|
||||
|
Shares used in computing basic and diluted net loss per share
|
149
|
143
|
147
|
143
|
||||||||||||
|
Three months ended
September 30,
|
Nine months ended
September 30,
|
|||||||||||||||
|
2024
|
2023
|
2024
|
2023
|
|||||||||||||
|
(unaudited)
|
||||||||||||||||
|
As reported research, development and patent expenses according to GAAP
|
$
|
220
|
$
|
215
|
$
|
656
|
$
|
643
|
||||||||
|
Excluding compensation expense related to equity awards
|
(22
|
)
|
(19
|
)
|
(67
|
)
|
(58
|
)
|
||||||||
|
Non-GAAP research, development and patent expenses
|
$
|
198
|
$
|
196
|
$
|
589
|
$
|
585
|
||||||||
|
As reported selling, general and administrative expenses according to GAAP
|
$
|
61
|
$
|
70
|
$
|
180
|
$
|
162
|
||||||||
|
Excluding compensation expense related to equity awards
|
(10
|
)
|
(7
|
)
|
(26
|
)
|
(22
|
)
|
||||||||
|
Non-GAAP selling, general and administrative expenses
|
$
|
51
|
$
|
63
|
$
|
154
|
$
|
140
|
||||||||
|
As reported operating expenses according to GAAP
|
$
|
282
|
$
|
287
|
$
|
843
|
$
|
811
|
||||||||
|
Excluding compensation expense related to equity awards
|
(32
|
)
|
(26
|
)
|
(94
|
)
|
(79
|
)
|
||||||||
|
Non-GAAP operating expenses
|
$
|
250
|
$
|
261
|
$
|
749
|
$
|
732
|
||||||||
|
As reported loss from operations according to GAAP
|
$
|
(148
|
)
|
$
|
(143
|
)
|
$
|
(364
|
)
|
$
|
(348
|
)
|
||||
|
Excluding compensation expense related to equity awards
|
(32
|
)
|
(26
|
)
|
(94
|
)
|
(79
|
)
|
||||||||
|
Non-GAAP loss from operations
|
$
|
(116
|
)
|
$
|
(117
|
)
|
$
|
(270
|
)
|
$
|
(269
|
)
|
||||
|
As reported net loss according to GAAP
|
$
|
(140
|
)
|
$
|
(147
|
)
|
$
|
(350
|
)
|
$
|
(357
|
)
|
||||
|
Excluding compensation expense related to equity awards and related tax effects
|
(32
|
)
|
(26
|
)
|
(94
|
)
|
(79
|
)
|
||||||||
|
Non-GAAP net loss
|
$
|
(108
|
)
|
$
|
(121
|
)
|
$
|
(256
|
)
|
$
|
(278
|
)
|
||||
|
September 30,
|
December 31,
|
|||||||
|
2024
|
2023
|
|||||||
|
(unaudited)
|
||||||||
|
Assets:
|
||||||||
|
Cash, cash equivalents and short-term investments
|
$
|
2,483
|
$
|
2,331
|
||||
|
Contracts receivable
|
18
|
98
|
||||||
|
Other current assets
|
213
|
213
|
||||||
|
Property, plant and equipment, net
|
83
|
71
|
||||||
|
Right-of-use assets
|
164
|
172
|
||||||
|
Other assets
|
120
|
105
|
||||||
|
Total assets
|
$
|
3,081
|
$
|
2,990
|
||||
|
Liabilities and stockholders’ equity:
|
||||||||
|
Current portion of deferred contract revenue
|
$
|
76
|
$
|
151
|
||||
|
0.125% convertible senior notes, net – short-term
|
44
|
44
|
||||||
|
Other current liabilities
|
184
|
253
|
||||||
|
1.75% convertible senior notes, net
|
564
|
562
|
||||||
|
0% convertible senior notes, net
|
628
|
625
|
||||||
|
Liability related to sale of future royalties, net
|
538
|
514
|
||||||
|
Long-term lease liabilities
|
168
|
171
|
||||||
|
Long-term obligations, less current portion
|
43
|
42
|
||||||
|
Long-term deferred contract revenue
|
174
|
241
|
||||||
|
Total stockholders’ equity
|
662
|
387
|
||||||
|
Total liabilities and stockholders’ equity
|
$
|
3,081
|
$
|
2,990
|
||||
|
New Product Launches
|
||||
|
Program
|
|
Indication |
Achieved
|
|
|
WAINUA
|
ATTRv-PN
|
•
|
||
|
Olezarsen
|
FCS
|
|||
|
QALSODY (EU)
|
SOD1-ALS
|
•
|
||
|
|
Regulatory Actions |
|
Program
|
Indication
|
Regulatory Action
|
Achieved
|
|||
|
Eplontersen
|
ATTRv-PN
|
Additional OUS filings
|
•
|
|||
|
EMA approval decision
|
||||||
|
Additional OUS approval decision(s)
|
•
|
|||||
|
Olezarsen
|
FCS
|
FDA approval decision
|
||||
|
NDA filing
|
•
|
|||||
|
EU filing
|
•
|
|||||
|
Canada filing
|
||||||
|
Donidalorsen
|
HAE
|
NDA filing
|
•
|
|||
|
QALSODY
|
SOD1-ALS
|
EMA approval decision
|
•
|
|||
|
China approval
|
•
|
|
Key Phase 3 Clinical Data Events
|
|
Program
|
Indication
|
Event
|
Achieved
|
|||
|
Olezarsen
|
FCS
|
Balance study full data
|
•
|
|||
|
Donidalorsen
|
HAE
|
OASIS-HAE topline data
|
•
|
|||
|
Donidalorsen
|
HAE
|
OASIS-HAE full data
|
•
|
|||
|
Donidalorsen
|
HAE
|
OASIS-Plus: OLE + Switch data
|
•
|
|||
|
Nusinersen
|
SMA
|
DEVOTE study data (higher dose)
|
•
|
|
|
Key Phase 2 Clinical Data Events |
|
Program
|
Indication
|
Event
|
Achieved
|
|||
|
Donidalorsen
|
HAE
|
3-year Phase 2 OLE data
|
•
|
|||
|
IONIS-FB-LRx
|
IgAN
|
Phase 2 data
|
•
|
|||
|
IONIS-FB-LRx
|
GA
|
GOLDEN study data
|
--
|
|||
|
ION224 (DGAT2)
|
NASH
|
Phase 2 data
|
•
|
|||
|
ION582 (UBE3A)
|
Angelman syndrome
|
HALOS study data
|
•
|
|||
|
ION541 (ATXN2)
|
ALS
|
ALSpire study data
|
--
|
|||
|
Sapablursen (TMPRSS6)
|
Polycythemia vera
|
IMPRSSION study full enrollment
|
•
|
|||
|
IONIS-MAPTRx (Tau)
|
Alzheimer’s disease
|
CELIA study full enrollment
|
•
|
| (1) |
Timing expectations based on current assumptions and subject to change.
|
| • |
Milestone achieved
|
Document and Entity Information |
Nov. 06, 2024 |
|---|---|
| Cover [Abstract] | |
| Document Type | 8-K |
| Amendment Flag | false |
| Document Period End Date | Nov. 06, 2024 |
| Entity File Number | 000-19125 |
| Entity Registrant Name | IONIS PHARMACEUTICALS, INC. |
| Entity Central Index Key | 0000874015 |
| Entity Incorporation, State or Country Code | DE |
| Entity Tax Identification Number | 33-0336973 |
| Entity Address, Address Line One | 2855 Gazelle Court |
| Entity Address, City or Town | Carlsbad |
| Entity Address, State or Province | CA |
| Entity Address, Postal Zip Code | 92010 |
| City Area Code | 760 |
| Local Phone Number | 931-9200 |
| Title of 12(b) Security | Common Stock, $.001 Par Value |
| Trading Symbol | IONS |
| Security Exchange Name | NASDAQ |
| Entity Emerging Growth Company | false |
| Written Communications | false |
| Soliciting Material | false |
| Pre-commencement Tender Offer | false |
| Pre-commencement Issuer Tender Offer | false |
1 Year Ionis Pharmaceuticals Chart |
1 Month Ionis Pharmaceuticals Chart |
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