uniQure Announces Additional Data from Ongoing Phase I/II Studies of ifezuntirgene inilparvovec(AMT-130) in Huntington’s Disease Showing Continued Slowing of Disease Progression

~ In 12 high-dose patients at 48 months, both cUHDRS and TFC continued to demonstrate meaningful slowing of disease progression and clear dose-dependent response; the primary endpoint of cUHDRS showed 44% slowing of disease progression (non-significant p=0.144) and TFC showed a 61% slowing of disease progression (nominal p=0.008) ~

~ Updated data reflecting all 15 high-dose patients showed substantial treatment effect on both cUHDRS and TFC at 36 months, the timepoint that is the regulatory anchor for the submitted BLA and the confirmatory study; the new analysis demonstrated 80% slowing of disease progression based on cUHDRS (nominal p=0.005) and 67% based on TFC (nominal p=0.011) ~

~ Treatment effect at 48 months likely understated by substantial missing data and survivor bias in updated external control; post hoc analysis using prior external control showed slowing of disease of 54% on cUHDRS (nominal p=0.041) and 68% on TFC (nominal p=0.001) at 48 months ~

~ These positive data are meaningful for Huntington's disease patients who currently have no approved disease-modifying treatment options ~