Analyst Conference Summary

biotechnology

conference date: July 30, 2025 @ 8:30 AM Pacific Time
for quarter ending: June 30, 2025 (Q2, second quarter 2025)

Forward-looking statements

Overview: Revenue jumped, showed GAAP profits.

Basic data (GAAP):

Revenue was $452 million, up 242% sequentially from $132 million, and up 100% from $225 million year-earlier.

Net income was $140 million, up sequentially from negative $147 million, and up from negative $66 million year-earlier.

EPS (diluted) was $0.70, up sequentially from negative $0.93, and up from negative $0.45 year-earlier,

Guidance:

Increased total 2025 expected revenue to between $825 and $850 million, including Tryngolza sales between $75 and $80 million. Expects operating loss, non-GAAP, between $300 and $325 million. End of year cash and equivalents $2 billion.

Conference Highlights:

CEO Brett Monia said "During the second quarter, we continued to build momentum across our business. Our strong performance included excellent commercial execution, resulting in a substantial increase in Tryngolza revenues, our first independently launched medicine. We expect additional advancements in the second half, including Ionis' second independent launch with donidalorsen for hereditary angioedema, anticipated next month, and important Phase 3 results for olezarsen in severe hypertriglyceridemia and zilganersen in Alexander disease. We believe these four programs collectively represent multi-billion-dollar revenue potential and a transformational opportunity for Ionis and for patients"

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.

Q2 2025 profits mainly due to $337 million from R&D partnerships.

Q2 2025 had substantial revenue from licensing sapablursen.

In Q2 2025 the first patient was dosed in the Phase 3 REVEAL study of ION582, for the treatment of people living with Angelman Syndrome (AS), a serious and rare neurodevelopmental disorder.

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 has a PDUFA of September 22, 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.

Q2 2025 revenue included a $280 million upfront payment for the global license of sapablursen to Ono Pharmaceutical.

Non-GAAP numbers: net income negative $154 million, sequentially up from negative $118 million, and up from negative $35 million year-earlier. No non-GAAP EPS given.

Cash ended at $2.3 billion, up sequentially from $2.1 billion. About $1.2 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 $312 million, consisting of $4 million for cost of goods sold; $217 million for R&D and $91 million for selling, general and administrative. Operating income was $140 million. Other expense net was $16 million. Income tax $0 million.

Q&A selective summary:

Tryngolza launch, finding patients? FCS has early momentum. Product profile is strong. Reduces risk of acute pancreatitis. Talking to 3000 physicians. Reimbursement access going very well. Continues to identify patients. About 3,000 patients.

SHTG triglyceride lowering in later Phase 3 trials should be about the same as with the first Phase 3 trial. Physicians see the need for treatment. Anything north of 50% is a big win. Physicans believe this will help with acute pancreatitis. Could observe a favorable trend for AP.

Everything looks to be in place for the Donidalorsen launch, on approval.

We don't know what the AP rate is in sHTG, this is the first study of that.

Pricing with sHTG? First mover advantage is important in both FCS and sHTG. We are still working on the pricing dynamic, will work with the payer community. Price will be announced upon the sHTG approval.

Donidalorsen competition? Patients on prophylactic treatments are not satisfied and are willing to switch to an improved therapy. Self administration and 4 or 8 week dosing schedule are advantages. So we should be in good shape for the competition.

In Europe, triglyceride lowering should be suffient for sHTG approval. But AP lowering would help with pricing and reimbursement. We expect Sobi to do well with the issue there.

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