Additional Relevant Details The primary efficacy analysis in the ongoing Phase 3 PALISADE trial will now be conducted on ages 4-17 years, which aligns with the Breakthrough Therapy Designation population, based on the company’s discussions with the FDA. The company expects to conduct separate analyses for older patients in PALISADE. Additionally, based on FDA feedback, the company anticipates that the safety database for a potential Biologics License Application (BLA) will include data from at least 600 patients treated with AR101.