Malcolm Kuno's questions to Ultragenyx Pharmaceutical Inc (RARE) leadership • Q3 2024
Question
Malcolm Kuno, on for Anupam Rama, asked what specific data points investors should focus on in the upcoming Angelman syndrome program updates at medical conferences.
Answer
CEO Emil Kakkis stated the update will feature longer-term data through day 338 for the expansion cohort, which aligns with the Phase 3 trial's length. He added that they will also present data showing that using raw scores versus GSV scores for analysis does not impact the study's power, addressing a key investor question, and will provide a general safety update.