IONIS PHARMACEUTICALS INC Q3 FY2025 earnings call

Key Points

• Brett Monia highlighted the third quarter as a watershed moment with progress in advancing medicines for serious diseases, including Tringolza's strong launch and Donzara's approval for Hereditary Angioedema (HAE). Positive results for olazarsen in severe hypertriglyceridemia (SHTG) and zilgarnirsen in Alexander disease were noted, paving the way for additional independent launches next year.
• Richard Geary discussed olazarsen's phase III results showing significant triglyceride reductions and acute pancreatitis event reductions, zilgarnirsen's phase III results for Alexander disease with disease-modifying effects, and ION 582's data from the HALO study showing communication and functional domain improvements.
• Kyle Jenne detailed Tringolza's strong momentum, preparations for olazarsen's launch including targeting HCPs and engaging payers, Donzara's successful launch execution with early positive feedback, and zilgarnirsen's commercial plans for Alexander disease.
• Beth Hougen reported strong revenue growth, increased 2025 financial guidance, with total revenue expected $875M-$900M, TRINGOZA sales $85M-$95M, operating loss $275M-$300M, and a cash balance over $2.1B.

In the third quarter, Ionis Pharmaceuticals, Inc. generated $157 million in revenue, a 17% year-over-year increase. For the first nine months, revenue totaled $740 million, a 55% increase compared to the prior year. Tringolza, the company's first independent launch, earned $32 million in product sales in the third quarter, representing nearly a 70% increase over the second quarter. Royalty revenues increased by approximately 13% to $76 million in the third quarter, driven by contributions from SPINRAZA and WAINUA. Tringolza's revenue contribution is significant due to its strong launch momentum and addressing unmet need in familial chylomicronemia syndrome (FCS).

Forward-Looking Statements

• Ionis Pharmaceuticals is raising its 2025 financial guidance. It now expects total revenue between $875 million and $900 million, an increase of $50 million from prior guidance. TRINGOZA product sales are anticipated to be between $85 million and $95 million for the full year. The company expects an operating loss between $275 million and $300 million for the full year and to end the year with a cash balance of more than $2.1 billion.

Risks

• Forward-looking statements are subject to certain risks and uncertainties. Actual results may differ materially from those projected. Investors are encouraged to consult the risk factors contained in the company's filings for additional detail.

Q: Jessica Fye asked about the launch curve shape for olazarsen in SHTG.

A: Kyle Jenne responded that they target ~20,000 HCPs covering ~360,000 SHTG patients, expecting strong uptake based on interest and market learnings.

Q: Gena Wang asked about olazarsen's core and core two data and peak revenue potential for Donzara, Alassane, and Alexander disease.

A: Brett Monia stated the AP data is groundbreaking and they look forward to presenting detailed data, while Kyle Jenne mentioned peak sales for Donzara over $500M, olazarsen over $1B, and Alexander disease over $100M.

Q: Mike Ulz asked about olazarsen's pricing.

A: Kyle Jenne replied pricing work is ongoing, with additional information expected next year based on research on patient exposure and market analysis.

Q: Yaron Werber asked about AP event reduction in SHTG and HAE prevalence.

A: Brett Monia noted AP data is groundbreaking with rapid protection and durability, and Kyle Jenne stated they work off the 7,000 estimated HAE patients in the US.

Q: Gary Nachman asked about Trungosa's additional FCS patients and olazarsen's filing priority.

A: Kyle Jenne discussed FCS patient identification through disease education and payer policies, and Brett Monia mentioned olazarsen's filing is on track with standard review but potential for priority review.

Q: Jason Gerberry asked about Cardio Transform and NNT in SHTG.

A: Brett Monia and Kyle Jenne discussed Cardio Transform data expectations and NNT's value in payer decision-making.

Q: Yanan Zhu asked about Donzara's early prescriptions and Winua's revenue bump.

A: Kyle Jenne reported Donzara's launch is going well with positive HCP feedback, and Waynua's revenue bump is from new patient identification in a growing market.

Q: Luca Issi asked about olazarsen's peak revenue assumptions and dosing filing.

A: Kyle Jenne stated greater than $1B in peak sales is expected with ongoing market research, and Brett Monia mentioned filing on both olazarsen doses for dosing flexibility.

Q: Jay Olson asked about capital allocation priorities.

A: Beth Hougen replied capital allocation is prioritized for internal growth to support pipeline and launch execution.

Q: Mitchell Kapoor asked about olazarsen's trial dropout reasons.

A: Richard Geary responded dropout rates were lower than expected with varied reasons like personal issues, and the drug has a favorable safety and tolerability profile