Analyst Conference Summary

biotechnology

conference date: April 29, 2026 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2026 (Q1, first quarter 2026)

Forward-looking statements

Overview: Revenue expanding very rapidly, but still operating at a loss.

Basic data (GAAP):

Revenue was $246 million, up 21% sequentially from $203 million, and up 86% from $132 million year-earlier.

Net income was negative $118 million, up sequentially from negative $229 million, and up from negative $146 million year-earlier.

EPS (diluted) was negative $0.56, up sequentially from negative $1.41, and up from negative $0.93 year-earlier,

Guidance:

Increased 2026 guidance. Revenue $875 to $900 million. Operating loss $425 to $475 million. Cash ending at over $1.6 billion.

Conference Highlights:

CEO Brett Monia said "Ionis’ strong performance in the first quarter of 2026 underscores the strength of our commercial and R and 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. 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."

Dawnzera launch is going well. Largely a switch market.

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

In Tryngolza for SHTG (severe hypertriglyceridemia) the Phase 3 CORE data, and from 2 other Phase 3 trials, were positive. Showed pancreatitis reduction. Ionis submitted the sNDA in Q4 2025 with PDUFA 6/30/2026. Estimated U.S. sHTG patients over 1 million. Will adjust price for this much larger population. Already has full field team in place in Q1 2026. Increased 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.

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.

Eplontersen, partnered, for ATTR-CM could launch in 2027.

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. Sales ramp will mainly be in 2026.

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, 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.

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 Q4 2025 Sapablursen for polycythemia vera (PV) had positive Phase 2 results, presented at American Society of Hematology (ASH) conference. Ono is advancing sapablursen into 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 $50 million, sequentially up from negative $186 million, and up from negative $118 million year-earlier. No non-GAAP EPS given, but about negative $0.30.

Cash ended at $1.9 billion, down sequentially from $2.7 billion. Paid off $633 million of 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 $364 million, consisting of $3 million for cost of goods sold; $210 million for R&D and $151 million for selling, general and administrative. Operating income was negative $118 million. Other income net was negative $25 million. Income tax $0 million.

Q&A selective summary:

Increased Tryngolza guidance? Phase 3 data and demand research. Payor research and price setting. Granting of Priority Review. Updated FCS treatment guidelines singly out Tryngolza.

Tryngolza 2026 trajectory? Strong Q1 FCS demand. June 30 PDUFA, interest of patients, including those who've had accute pancreatitis. But expect more steady growth rather than a bolus. Payers will wait for the final label before they make coverage decisions. Unprededented triglyceride lowering.

Discontinuations for long term extension study are very low.

SHTG competition? Looking forward to seeing competitor data. Our Phase 3 data is incredibly compelling, creating a very high bar for competitors. It is a large market, over 3 million potential patients.

Dawnzera competition? Evolving. In US 75% of patients are on a prophylactic treatment, so it is a switch market. Patient and HCP feedback has been very positive. Most start on a four week dosing schedule. Over time many will progress to an eight-week schedule.

We also expect some large collaboration milestone payments in 2027.

Wainua q/q decline? We saw strong demand in Q1, including new patient starts. In January there can be pressure from reauthorizations, so we expect return to growth in Q2.

Future generic competition in ATTR-CR market? Not surprising, in product sales guidance. Believer silencer class will be prefered to stabilizers and utilized robustly.

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