Analyst Conference Summary

biotechnology

conference date: February 25, 2026 @ 8:30 AM Pacific Time
for quarter ending: December 31, 2025 (Q4, fourth quarter 2025)

Forward-looking statements

Overview:

Basic data (GAAP):

Revenue was $ million, up % sequentially from $157 million, and up % from $ million year-earlier.

Net income was negative $ million, down sequentially from negative $129 million, and up from negative $ million year-earlier.

EPS (diluted) was negative $, down sequentially from negative $0.80, and up from negative $ year-earlier,

Guidance:

Conference Highlights:

CEO Brett Monia said ""

2026 convertible debt will be refinanced before its maturity date. Well positioned to achieve significant revenue growth and sustained positive cash flow in the next few years.

Tryngolza (Olezarsen) for familial chylomicronemia syndrome (FCS) had $32 million Q3 2025 sales, stronger than expected. Payer coverage is good and continues to grow. Most potential patients are undiagnosed; estimated U.S. total is just 3,000. EU CHMP issued a positive opinion in Q2 2025, approved in Q3 2025, with partner Sobi to launch in Q4 2025. Licensed global rights ex U.S., Canada, and China to Sobi.

In SHTG (severe hypertriglyceridemia) the Phase 3 CORE data, and from 2 other Phase 3 trials, were positive. Ionis plans to submit the sNDA by end of 2025. Approval possible in late 2026. Estimated U.S. sHTG patients over 1 million.

Zilganersen for Alexander disease Phase 3 study reported positive results in Q3 2025. Could launch in 2026.

Wainua revenue continues to grow. Believes royalties could add meaningfully to revenue in the growing ATTRv-PN market. ATTR Cardiomyopathy trial continues, with enrollment completed in Q3 2023, but data not expected until later in 2025. Launches for PN are underway in several nations. Received EU approval for PN in Q2 2025. [But competing drug for ATTR-CM now on market from Alnylam, in addition to older drugs like Tafamidis.]

ION582 for Angelman syndrome reported positive Phase 2 data in Q3 2025. The Phase 3 study is expectd to fully enroll in 2026.

Donidalorsen (now Dawnzera) for hereditary angioedema (HAE) was approved by the FDA in August 2025. Otsuka is planning to submit the European MAA. HAE has over 20,000 patients in the US and EU. Independent commercial launch is off to a good start. Believes peak sales could be $500 million per year. Sales ramp will mainly be in 2026.

Spinraza (nusinersen) for SMA higher dose now has a PDUFA of April 3, 2026 after receiving a CRL earlier in 2025. Also under EU review. Salanersen for SMA had postive Phase 1 results, Biogen will take into registrational studies. Salanersen royalty rates would be higher than those for Spinraza, and of course would extend the patent coverage period.

Opemalirsen (ION532) to reduce APOL1 for APOL1-mediated kidney disease began a Phase 2b study in Q2 2025, triggering a $30 million milestone payment.

Non-GAAP numbers: net income negative $ million, sequentially up from negative $98 million, and up from negative $ million year-earlier. No non-GAAP EPS given.

Cash ended at $ billion, down sequentially from $2.2 billion.

Ionis has a pipeline of about 45 potential drugs, with 13 in clinical development, and 9 in Phase 3 trials. A growing number are wholly-owned.

GAAP Operating expense was $ million, consisting of $ million for cost of goods sold; $ million for R&D and $ million for selling, general and administrative. Operating income was negative $ million. Other income net was $ million. Income tax $ million.

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