Ionis Pharmaceuticals (IONS) Jefferies London Healthcare Conference 2025 summary

Key achievements and product launches

• Launched two independently approved medicines in the U.S. this year: Tryngolza for FCS and DAWNZERA for hereditary angioedema, both off to strong starts.

• Announced groundbreaking phase III data for severe hypertriglyceridemia, showing unprecedented reductions in triglycerides and acute pancreatitis.

• Positive phase III data for a neurology drug targeting Alexander disease, with approval targeted for next year.

• Five phase III readouts and up to three FDA approvals expected next year, setting up for further growth.

Pipeline strategy and market positioning

• Focused on severe hypertriglyceridemia, initially targeting the highest-risk patients, then expanding to broader populations.

• Advancing next-generation therapies to enable less frequent dosing, aiming for once or twice yearly self-administration.

• Exploring combination therapies for broader cardiovascular risk populations, leveraging flexible formulation and delivery.

• Collaborating with AstraZeneca on TTR cardiomyopathy, combining silencer and depleter approaches for rapid and sustained benefit.

Clinical development and trial design

• Eplontersen phase III trial in TTR cardiomyopathy is the largest to date, with robust imaging and subgroup data.

• Combination subgroup elevated to a key secondary endpoint, aiming for meaningful benefit even if not statistically significant.

• Study size and design expected to provide greater statistical power and faster separation in endpoints compared to prior studies.