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; PDUFA date set for June 30, enabling a potential launch by early July.

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

• DAWNZERA approved in the US and Europe, offering a novel, convenient prophylactic treatment for HAE, with positive physician and patient reception.

• 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 this year: olezarsen for sHTG, zilganersen for Alexander disease, and bepirovirsen for chronic HBV.

Commercial strategy and market focus

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

• Initial sHTG launch will target high-risk patients with a history of acute pancreatitis or triglycerides above 880 mg/dL.

• Expansion to broader sHTG populations planned after initial focus on highest-risk groups.

• Revenue guidance will be updated at the end of Q1 to reflect earlier market entry due to priority review.

• Commercial model prioritizes US launches, with ex-US distribution through partners.

Pipeline innovation and future outlook

• Robust cardiometabolic pipeline includes follow-on programs for olezarsen and targeted delivery medicines.

• TRYNGOLZA's first-year revenue exceeded $108 million, with peak sales estimates for TRYNGOLZA and sHTG raised to over $2 billion.

• Neurology pipeline features 12 clinical-stage medicines, including zilganersen for Alexander disease and obinutersen for Angelman syndrome.

• NDA for zilganersen submitted; launch expected in the second half of the year, with global access planned via partnerships.

• Financial objectives include cash flow break-even by 2028 and over $6 billion in potential annual revenue from owned and partnered products.