IONIS PHARMACEUTICALS INC Q1 FY2025 earnings call

• Completed strategic licensing transactions, substantially increasing 2025 financial guidance.
• TRYNGOLZA had an encouraging debut in Q1 with over $6 million in net product sales, driven by patient uptake from expanded access, pent-up demand, and new patient identification.
• Donidalorsen for HAE is on track for anticipated approval in August, with preparations for launch including patient support programs and field team hiring.
• Olezarsen for sHTG is making progress, with ESSENCE study data expected in Q2 and pivotal study data in Q3, aiming for an sNDA submission by year-end.
• Broadened partnership with Sobi for olezarsen commercialization outside the U.S.

In the first quarter, Ionis earned $132 million in revenue, a 10% year-over-year increase. Over half of the revenue came from commercial products, which grew 28% YOY. TRYNGOLZA, the first independent launch, generated over $6 million in net product sales in its first full quarter. SPINRAZA royalties were $48 million, up 25% YOY, and WAINUA royalties were $9 million. The remainder of revenue came from R&D collaborations.

• Increased 2025 revenue guidance to $725-$750 million, driven by strong Q1 results and licensing transactions.
• Projected operating expenses to increase in the high single-digit percentage range, with R&D expenses steady. Non-GAAP operating loss guidance improved by nearly 25% to less than $375 million.
• Expect $1.9 billion in cash by year-end and sustained revenue growth beyond 2025, with late-stage programs having multibillion-dollar peak sales potential.

• Uncertainties from recent FDA changes and new tariff policies.
• Monitoring of supply chain and clinical trial impacts from tariffs and FDA changes, with contingencies being built.

Q: Curious about overlap of physician prescribers for FCS and sHTG and AP reduction targeting A: Kyle Jenne discussed overlap of cardiologists, endocrinologists, etc., treating both FCS and sHTG patients, and Brett Monia addressed AP data powering and targeting Q: On donidalorsen launch preparation and macro dynamics A: Kyle Jenne talked about donidalorsen launch preparation and patient switching, Brett Monia discussed FDA and tariff impacts with no significant current impact Q: On FCS launch patient estimate and price erosion A: Unidentified company representative and Kyle Jenne discussed confidence in FCS patient estimate and timing of sHTG commercialization Q: On TRYNGOLZA channel stocking, revenue guidance sources, and manufacturing exposure to tariffs A: Kyle Jenne and Beth Hougen addressed channel stocking as demand-driven, revenue guidance from commercial performance and licensing, and manufacturing with supply chain redundancies Q: On TRYNGOLZA patient conversion and Angelman Phase III start A: Kyle Jenne and Brett Monia discussed TRYNGOLZA patient conversion and Angelman Phase III study on track for Q2 start Q: On WAINUA Part D impact and TRYNGOLZA patient growth A: Kyle Jenne discussed WAINUA Part D impact and TRYNGOLZA patient identification efforts Q: On TTR cardiomyopathy pricing and strategy A: Kyle Jenne discussed TTR cardiomyopathy pricing strategy and WAINUA's market evolution Q: On olezarsen core trials AP data analysis A: Eugene Schneider and Brett Monia discussed AP data analysis in olezarsen trials Q: On TRYNGOLZA patient distribution and olezarsen data timing A: Kyle Jenne and Brett Monia discussed TRYNGOLZA patient distribution and olezarsen data timing and AP readouts