On April 29, 2026 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) (the "Company") reported financial results and provided key updates for the first quarter ended March 31, 2026.

"Ionis’ strong performance in the first quarter of 2026 underscores the strength of our commercial and R&D engines. Our independent launches are increasingly contributing to revenue, driven by strong commercial execution, and we are on track for two additional groundbreaking independent launches in 2026 — olezarsen for severe hypertriglyceridemia, our first medicine for a broad patient population, and zilganersen for Alexander disease, the first launch from our leading neurology pipeline," said Brett P. Monia, Ph.D., chief executive officer of Ionis. "In addition, we look forward to multiple key value-driving events this year, including results from pivotal Phase 3 partnered programs. These include presentation of positive bepirovirsen data in chronic hepatitis B next month at EASL, as well as results from the landmark pelacarsen Lp(a) HORIZON and eplontersen CARDIO-TTRansform cardiovascular outcomes trials later this year."

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First Quarter 2026 Summary Financial Results(1):

Three months
ended
March 31,

2026

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.

First Quarter 2026 Financial Highlights

•
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

First Quarter 2026 Financial Results

"Ionis entered 2026 with strong momentum. We continued this momentum with the first quarter financial results reflecting increased commercial revenue from our independent launches and robust R&D revenue when compared to the same period last year," said Elizabeth L. Hougen, chief financial officer of Ionis. "Based on our strong year-to-date revenue performance, accelerating momentum and positive outlook for the rest of the year, we are improving our 2026 financial guidance. The strong performance we expect in 2026 will support substantial growth and long-term value creation and our goal of reaching cash-flow breakeven in 2028."

Recent Highlights – Wholly Owned Medicines

•
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

2

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

Recent Highlights – Partnered Medicines

•
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

(Press release, Ionis Pharmaceuticals, APR 29, 2026, View Source [SID1234664895])