Lydia Unknown's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q3 2024
Question
An analyst identified as Lydia, on for Salveen Richter, asked about the Wilson's disease program, specifically when to expect data from the additional cohort and what results would provide confidence for the regulatory and commercial outlook.
Answer
CEO Emil Kakkis projected that data from the fourth Wilson's disease cohort would take most of the next year to collect. He stated the goal is to see a consistent and profound reduction in the need for standard-of-care treatment in the majority of patients, which he believes is necessary to justify a Phase 3 investment and ensure the gene therapy is commercially viable.