Analyst Conference Summary

biotechnology

conference date: October 29, 2025 @ 8:30 AM Pacific Time
for quarter ending: September 30, 2025 (Q3, third quarter 2025)

Forward-looking statements

Overview:

Basic data (GAAP):

Revenue was $452 million, up 242% sequentially from $132 million, and up 100% from $ million year-earlier.

Net income was $ million, up sequentially from $140 million, and up from negative $ million year-earlier.

EPS (diluted) was $, up sequentially from $0.70, 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.

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 are underway in several nations. Received EU approval for PN in Q2 2025. [But competing drug for ATTR-CM now on market from Alnylam]

Tryngolza (Olezarsen) for familial chylomicronemia syndrome (FCS) approved by FDA on December 19, 2024. Is the first Ionis independent commercial drug launch, with $19 million Q2 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, approval likely in H2 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, should be available in September 2025. If positive would submit the sNDA by end of 2025. Estimated U.S. sHTG patients over 1 million.

ION582 for Angelman syndrome started Phase 3 in Q2 2025. Data from the Phase 1/2 trial were positive.

Donidalorsen for hereditary angioedema (HAE) submitted an NDA in the US. PDUFA date is August 21, 2025. Otsuka is planning to submit the European MAA. HAE has over 20,000 patients in the US and EU. Preparing for independent commercial launch.

Zilganersen for Alexander disease Phase 3 study is fully enrolled, data expected in H2 2025.

Spinraza (nusinersen) for SMA higher dose had a PDUFA of September 22, 2025 but received a CRL. 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 $154 million, and up from negative $ million year-earlier. No non-GAAP EPS given.

Cash ended at $ billion, up sequentially from $2.3 billion. About $ billion in long-term debt (convertible notes).

Ionis continues to develop technologies that allow RNA therapies to almost any part of the body, including inhaled agents.

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 $ million. Other expense net was $ million. Income tax $0 million.

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