Uygar Turancigil

Uygar Turancigil questioned why the FDA is now requiring a sham study, specifically asking if there were objections to the natural history database or statistical plan, and whether CEO Matt Kapusta remains committed to advancing AMT-130 even with a sham study requirement.

Answer

CEO Matt Kapusta noted that the FDA previously indicated openness to using Phase I/II data with an external control and has not criticized the Enroll-HD database, finding the current stance puzzling given its robustness. He acknowledged sham-controlled studies are gold standard for bias reduction. Matt Kapusta affirmed his commitment to AMT-130, stating that if a sham study is feasible and ethical, and supported by the patient community, uniQure has a moral obligation to pursue it.