On July 29, 2026 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company") reported financial results and provided key updates for the second quarter ended June 30, 2026.
"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."
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Second Quarter 2026 Summary Financial Results(1):
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.
1
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
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
2
•
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
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
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
3
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.
4
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 View Source and View Source, 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).
(Press release, Ionis Pharmaceuticals, JUL 29, 2026, View Source [SID1234669498])