Ionis Pharmaceuticals (IONS) investor relations material

Key pipeline and regulatory milestones

• Achieved two FDA approvals: TRYNGOLZA for severe hypertriglyceridemia and ZANVASTRO for Alexander disease, with a third approval for chronic HBV expected in October in partnership with GSK.

• Completed enrollment in the phase III Angelman syndrome trial, with data expected in the second half of the year.

• Advanced multiple phase III programs, including salanersen for SMA and sapablursen for polycythemia vera, with ongoing mid-stage pipeline progress.

• Reported first-ever proof of concept for the tau program with Biogen, showing cognitive and functional improvements.

• Faced setbacks in the CARDIO-TTRansform and pelacarsen phase III trials but identified promising monotherapy data for TTR cardiomyopathy.

Commercial launches and market strategy

• TRYNGOLZA launch for severe hypertriglyceridemia is progressing well, with strong physician and payer enthusiasm and a blockbuster sales target of $3+ billion in the U.S.

• Label expansion from FCS to severe hypertriglyceridemia increased the addressable market to over 3 million patients, with pricing adjusted accordingly.

• Early adoption is focused on high-risk patients, but scripts are also being written for lower-risk groups.

• Emphasizing disease and drug awareness, patient support, and payer coverage as keys to sustained launch success.

• DAWNZERA for HAE is gaining traction in a competitive market, with differentiating features in convenience and tolerability.

Product differentiation and competitive landscape

• TRYNGOLZA offers dosing flexibility and requires no monitoring, resonating well with healthcare providers.

• Safety profile for TRYNGOLZA is positive, with manageable and transient increases in liver fat.

• First-mover advantage is being leveraged ahead of a competitor expected to enter the market next year; both drugs are expected to have similar efficacy.

• Next-generation program ION775 aims for once-yearly dosing, with phase II underway and phase III planned, potentially complementing or succeeding TRYNGOLZA.