IONIS PHARMACEUTICALS INC Q3 FY2024 earnings call

• Pipeline progress: Olezarsen is near its FDA action date for familial chylomicronemia syndrome (FCS), with Phase 3 data for severe hypertriglyceridemia (sHTG) planned for the second half of 2025. Donidalorsen's NDA was accepted by the FDA with a PDUFA date of August 21, 2025. ION582 for Angelman syndrome is on track for Phase 3 development in the first half of 2025. - Launches: Ionis is set to launch Olezarsen for FCS in 2024 and donidalorsen for hereditary angioedema (HAE) in 2025. WAINUA and QALSODY have been successfully launched, with WAINUA showing strong growth in the U.S. - Clinical data: Olezarsen demonstrated substantial triglyceride reductions and reduced acute pancreatitis events in Phase 3. Donidalorsen showed over 90% reduction in HAE attack rates. WAINUA's CARDIO-TTRansform trial is ongoing with data expected in the second half of 2026.

In the third quarter of 2024, Ionis reported revenues of $134 million, with year-to-date revenues reaching $479 million. SPINRAZA remained the primary source of commercial revenue, contributing $57 million in royalties for the third quarter and $152 million year-to-date. WAINUA product revenue showed accelerating growth, with $23 million in the third quarter and $44 million year-to-date. R&D revenue was in line with the prior year. Non-GAAP operating expenses increased due to higher sales and marketing investments in preparation for upcoming launches, with sales and marketing expenses contributing to the overall increase in SG&A expenses.

• 2024 revenue guidance: Ionis is on track to meet revenue guidance of over $575 million, including approximately $175 million from non-cash amortization of partner payments. - Cash projection: Ending 2024 with an expected $2.2 billion in cash after recent equity offering. - Future plans: Continued investment in go-to-market preparations for Olezarsen and donidalorsen launches, and pre-commercialization activities for ION582 and Zilganersen.

• Regulatory uncertainties: Delays or issues in FDA approvals for pipeline products. - Competition: Potential entry of competitors affecting market share. - Trial risks: Unforeseen challenges in Phase 3 studies impacting timelines and outcomes.

Q: On olezarsen for FCS, how soon will you be able to launch post approval and pricing?

A: Ready to launch in 2024, with ultra-rare pricing to be communicated upon approval.

Q: On Phase 3 Angelman study, any interim looks and FDA agreement?

A: No interim looks planned, strong relationship with FDA on study design.

Q: On FCS patient population and competition?

A: Identified patients through medical affairs and sales teams, expecting 9-month lead over competitors.

Q: On APOC3 program and Angelman study endpoint?

A: Lilly's APOC3 program discontinuation noted; Angelman study uses expressive communication as primary endpoint as preferred by FDA.

Q: On sales operation for launches?

A: Sales teams in place for Olezarsen launch, hiring ongoing for donidalorsen launch.

Q: On pelacarsen readout and timing?

A: Novartis expects Phase 3 data next year based on blinded event rates.

Q: On Angelman study ex-U.S. and ION269?

A: Ex-U.S. partnerships early, ION269 in down syndrome due to APP gene triplication in down syndrome patients.