Kuan Hong

Kuan Hong asked for elaboration on what uniQure considers a 'too difficult or challenging' Phase III design, specifically regarding study length and sham surgery risks, and whether any additional evidence will be presented at the planned Type B meeting.

Answer

CEO Matt Kapusta explained that a 'too difficult' design involves the risks of sham surgery (anesthesia, drilling, patient uncertainty) for a potentially multi-year study, where patients might progress without benefit. He emphasized appealing to the FDA's flexibility to minimize patient burden. CMO Walid Abi-Saab clarified that the Type B meeting will solely focus on discussing Phase III design, with no additional analyses planned until the Statistical Analysis Plan is built, and the next data share is expected in Q4.