Ionis Pharmaceuticals (IONS) investor relations material

Key achievements and product launches

• Launched two independent 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 for 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.

Cardiovascular disease strategy and pipeline

• Focused on severe hypertriglyceridemia, targeting high-risk patients first, then expanding to broader populations.

• Advancing next-gen treatments to enable less frequent dosing, aiming for twice-yearly or yearly self-administration.

• Exploring combination therapies for patients with mildly elevated triglycerides and high cardiovascular risk.

• Collaborating with AstraZeneca on TTR cardiomyopathy, combining silencer and depleter approaches.

ATTR and TTR cardiomyopathy developments

• Prioritizing cardiomyopathy over polyneuropathy for initial depleter-silencer combination trials.

• Emphasizing self-administration as a key differentiator, especially as treatment expands beyond centers of excellence.

• Upcoming phase III data for Eplontersen in TTR cardiomyopathy, with the largest study to date and robust imaging endpoints.

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