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: Rapid revenue growth on small base, but still operating at a loss.

Basic data (GAAP):

Revenue was $157 million, up 19% sequentially from $132 million, and up 17% from $134 million year-earlier.

Net income was negative $129 million, down sequentially from $140 million, and up from negative $140 million year-earlier.

EPS (diluted) was negative $0.80, down sequentially from $0.70, and up from negative $0.95 year-earlier,

Guidance:

Increased 2025 guidance to revenue between $875 and $900 million. Includes Tryngolza sales of $85 to $95 million. Operating loss, non-GAAP, $275 to $300 million. Cash and equivalents to end at $2.1 billion. Expects to reach cash flow breakeven in 2028.

Conference Highlights:

CEO Brett Monia said "The third quarter was a watershed moment for Ionis, as we made important progress advancing our Ionis-owned medicines. With two independent launches now underway, and two more anticipated in 2026, we are delivering on our goal to bring a steady cadence of new medicines to people in need. Last month, we announced groundbreaking, positive topline Phase 3 results for olezarsen in severe hypertriglyceridemia and for zilganersen in Alexander disease, with regulatory filings planned in the coming months. Our approved and late-stage portfolio continues to deliver, positioning Ionis for substantial growth."

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) 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, 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 $98 million, sequentially up from negative $154 million, and up from negative $108 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.2 billion, down sequentially from $2.3 billion. Reduced long-term debt (convertible notes) to $0 in Q3 2025, but increased current notes liabilities to $1.2 billion.

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 $317 million, consisting of $2 million for cost of goods sold; $218 million for R&D and $97 million for selling, general and administrative. Operating income was negative $160 million. Other income net was $31 million. Income tax $0 million.

Q&A selective summary:

Shape of launch for sHTG? Pent up demand? We already are getting inqueries from physicians. 3,000 HCPs are first targets of the sales force, but will expand to 20,000. Many current patients are on standard of care but not getting to goal. We expect strong uptake based on the interest and what we have learned about the market so far.

Anything concerning about data outside of topline results; acute pancreatitis? Detailed data will be released at American Heart Association on November 8. We see nothing to be concerned about. We believe protection against acute pancreatitis is very good.

Olezarsen peak revenue? Greater than $1 billion per year.

Alexander peak revenue? Greater than $100 million per year.

Final pricing for Olezarsen (Tryngolza)? Will not be decided for sHTG until 2026, will announce upon approval.

Generally, the higher the triglycerides, the higher the incidence of AP. Our study shows triglycerides are driving the events, and lowering triglycerides lowers the event rate.

For Dawnzera we have not seen at 11,000 number, we are using 7,000 potential patients.

FCS patient, prescriber details? We've been IDing patients. Working with highest prescribing patients. Physicians are looking for these patients, test for them. Payers are streamlining the process. Disease education is approving.

The goal is to prevent the first AP attack, that is the key value for payers.

We are seeing switches from all the approved treatments to Dawnzera.

We expect Wainua revenue to continue to grow. AstraZeneca is doing a good job with it.

External capital development? Plenty of cash to execute on our launches. Focus is on internal growth through our pipeline.

sHTG dropout rate? Dropout rate was very low, about half our original expectation. Very well tolerated medicine.

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