Ionis Pharmaceuticals (IONS) TD Cowen Genetic Medicines & RNA Summit summary

Wainua launch and TTR polyneuropathy

• Wainua launched in late January after December approval, reporting $5 million in partial quarter revenue and strong initial uptake.

• High receptivity from clinicians and patients, with self-administration via autoinjector seen as a key differentiator.

• Focus is on newly diagnosed patients, as over 80% of those with ATTR polyneuropathy are not currently treated.

• Some patient switches from other approved treatments are occurring, but emphasis remains on new diagnoses.

TTR cardiomyopathy clinical development

• Study design for TTR cardiomyopathy is the largest to date, powered for cardiac hospitalizations and mortality as primary endpoints.

• Patient demographics have shifted to earlier-stage diagnoses, making direct comparisons to older studies difficult.

• Combination and monotherapy arms are balanced, with low drop-in rates for tafamidis during the study.

• Imaging and secondary endpoints will provide additional insights beyond mortality, addressing evolving patient needs.

• Relative risk reduction standards of 15%-25% are considered meaningful, but the changing landscape requires nuanced interpretation.

Angelman syndrome program

• Phase II data for ION582 in Angelman syndrome will be presented in July, showing 60-70% of patients benefited across multiple parameters.

• Study included a broad patient population and leveraged robust natural history data for comparison.

• Consistent improvements observed across various assessment instruments, supporting confidence in results.

• Plans are underway for a phase III trial, with regulatory discussions to finalize endpoints and design.

• FDA will be consulted first, followed by EMA, with efforts to align before opening sites in each territory.