Ionis Pharmaceuticals (IONS) Oppenheimer 36th Annual Healthcare Life Sciences Conference summary

Recent regulatory milestones and product launches

• FDA accepted the supplemental NDA for olezarsen in sHTG with priority review and a PDUFA date of June 30, setting up a potential launch in late June or early July.

• Two independent product launches in 2025, TRYNGOLZA for FCS and DAWNZERA for HAE prophylaxis, both off to strong starts.

• DAWNZERA approved in the US and Europe, offering a novel, convenient prophylactic treatment for HAE, with strong uptake among patients switching from existing therapies.

• TRYNGOLZA achieved $108 million in first-year revenue, exceeding expectations, and is poised for expansion into the larger sHTG market.

• Commercial readiness for sHTG launch includes a 200-person field team and established supply chain.

Pipeline progress and future outlook

• Six positive phase III readouts and four approved medicines in the past 2.5 years, with 11 medicines in late-stage development.

• Three product approvals and launches expected in 2026, including olezarsen for sHTG, zilganersen for Alexander disease, and bepirovirsen for chronic HBV.

• Additional launches anticipated in 2027, including pelacarsen for Lp(a) and eplontersen for ATTR cardiomyopathy.

• Four independent launches by year-end, with four key partner launches expected by the end of next year.

• Five phase III data readouts expected in 2026, leading to four NDA submissions and three product launches.

Strategic focus and commercial strategy

• Focused on cardiometabolic and neurological diseases, targeting both rare and prevalent conditions.

• Initial sHTG launch will prioritize high-risk patients with a history of acute pancreatitis or triglycerides above 880, expanding to broader populations over time.

• Commercial strategy leverages established reputation in triglyceride management and strong relationships with healthcare providers.

• Financial guidance will be updated to reflect the accelerated sHTG launch due to priority review.

• Projected to achieve cash flow break-even by 2028, with over $4 billion in potential annual peak product revenue from owned medicines and $2 billion from partnered products.