Analyst Conference Summary

biotechnology

conference date: July 29, 2026 @ 5:30 AM Pacific Time
for quarter ending: June 30, 2026 (Q2, second quarter 2026)

Forward-looking statements

Overview: Pretty good, comparison warped by a single upfront payment in 2025. New SHTG revenue ramp barely begun.

Basic data (GAAP):

Revenue was $268 million, up 9% sequentially from $246 million, and down 41% from $ million year-earlier.

Net income was negative $115 million, up sequentially from negative $118 million, and down from $124 million year-earlier.

EPS (diluted) was negative $0.69, down sequentially from negative $0.56, and down from $0.70 year-earlier,

Guidance:

On track to achieve prior guidance. Revenue $875 to $900 million. Operating loss $425 to $475 million. Ending cash balance $1.6 billion. Believes can be cash-flow break even in 2028.

Conference Highlights:

CEO Brett Monia said "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. 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." Confident Tryngolza is well-positioned to become the first Ionis multi-billion dollar medicine.

Revenue in Q2 and H1 of 2026 increased by 56% and 69% y/y respectively excluding the one-time sapablursen upfront payment recognized in the second quarter of 2025,

In Tryngolza (olezarsen) for SHTG (severe hypertriglyceridemia) approved by the FDA in June 2026. The Phase 3 CORE data, and from 2 other Phase 3 trials, were positive. Showed pancreatitis reduction. Estimated U.S. sHTG patients over 1 million. Has adjusted price for this much larger population. Tryngolza (olezarsen) peak sales guidance to greater than $3 billion per year. The EU accepted an indication extension application in March. Sales for FCS should benefit from broader access. Changed annual wholesale acquisition price to $40,000, including both FCS and SHTG. About 1 million Americans have high-risk sHTG.

In Q2 2026 obudanersen for Angelman syndrome completed enrollment in its Phase 3 trial. Data due 2H 2027.

In Q2 2026 Wainua (eplontersen) Phase 3 data for ATTR-CM missed its primary endpoint.

Biogen will advance Diranersen (IONIS-MAPTRx / BIIB080), a potential treatment for Alzheimer’s disease, into Phase 3 development.

Dawnzera launch is going well. Largely a switch market. Estimated full year sales of $110 to $120 millon.

Increased operating costs in remainder of 2026 will be mainly from increased sales efforts, notably for Tryngolza for SHTG.

Pelacarsen for LP(a), partnered, could launch in 2027.

Zilganersen for Alexander disease Phase 3 study reported positive results in Q3 2025. NDA submitted in January 2026. Priority Review PDUFA 9/22/2026. Should launch in 2026. Licensed outside the US to Recordati.

In Q2 2026, ION775 for sHTG started a Phase 2 study. Phase 1 data will be presented in August 2026.

Donidalorsen (now Dawnzera) for hereditary angioedema (HAE) was launched in the EU in Q1 by Otsuka. It was approved by the FDA in August 2025. European approval was in January 2026. HAE has over 7,000 patients in the US. Independent commercial launch is off to a good start. Believes peak sales could be $500 million per year.

Spinraza (nusinersen) for SMA higher dose approved by FDA in Q1 2026. 2026 after receiving a CRL earlier in 2025. Approved in Q4 in EU. Salanersen for SMA had postive Phase 1 results, In Q2 2026 started Phase 3 development. Salanersen royalty rates would be higher than those for Spinraza, and of course would extend the patent coverage period.

In Q4 2025 Bepirovirsen for chronic hepatitis B achieved its primary endpoint and demonstrated a statistically significant and clinically meaningful functional cure rate in 2 Phase 3 studies. Global filings began in Q1 2026. Partner GSK to present Phase 3 data at European Association for the Study of the Liver Congress. The FDA PDUFA is 10/26/2026.

In Q2 2026 Sapablursen for polycythemia vera started Phase 3 development.

Possible Phase 3 readouts in 2026 include Pelacarsen for Lp(a), Eplantersen for ATTR-CM, Ulefnersen for FUS-ALS, and Sefaxerson for IgAN. All could launch in 2027, all are partnered.

Non-GAAP numbers: net income negative $70 million, sequentially down from negative $50 million, and down from $154 million year-earlier.

Cash ended at $2.1 billion, up sequentially from $1.9 billion. Debt $1.3 billion in convertible notes.

Ionis has a pipeline of about 45 potential drugs, with 12 in clinical development, and 8 in Phase 3 trials. A growing number are wholly-owned.

GAAP Operating expense was $370 million, consisting of $3 million for cost of goods sold; $217 million for R&D and $150 million for selling, general and administrative. Operating income was negative $102 million. Other income net was negative $12 million. Income tax $1 million.

Q&A selective summary:

Tryngolza early adoption? FCS launch helps SHTG launch. Early prescribers are prior treaters of FCS. But see other physicians as well. FCS numbers grew in Q2. Too early to discuss payers, except to say approval times seem good and discussions with payers are good. Currently mostly medical exceptions for coverage.

Reimbursement for different triglyceride levels? Coverage on label, >500 mg/dL.

Wainua? Demand continues to be strong. Challenge is mixed phenotype patients. Feedback good, including ability to self-administer with an auto-injector.

SHTG, are they on other drugs, switching, or naive? Seeing a mix. Most are high risk patients who have been on a therapy like statins. They are adding Tryngolza to their prior therapies. Acute pancreatitis on label helps. Adherence is good so far. Liver fat effects has not been a concern, partly because it returns to baseline with long-term treatment.

Competition? We believe Tryngolza is best in class, overall. Plus we have first mover advantage. We think peak product sales will be $3 billion per year or more.

The 2028 cash-flow break even goal is very, very important to us. We will work hard to achieve it.

ION775 for SHTG is really for a longer dosing schedule, it is not mainly about improving efficacy.

SHTG pent up demand or bolus? We believe it will be a gradual build. Will need to build up payer access, too, should be mostly in by 2027.

Dawnzera, switching from what? Some switching patients have an efficacy challenge, some tolerability, some duration of treatment or frequency issue. We are seeing patients from all the former therapies out there.

IN582 looks at least as potent as competitors, data readout next year.

ION337 data? We just started dosing, too early to discuss timeline. The community is excited.

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