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

Walid Abi-Saab, Chief medical officer of uniQure

“Four years after a single administration, ifezuntirgene inilparvovec continues to show meaningful slowing of disease progression, further strengthening our conviction in its benefit for people living with Huntington’s disease.”

Walid Abi-Saab, Chief medical officer of uniQure

“At 48 months, Total Functional Capacity (TFC), the primary measure of our confirmatory study, demonstrated consistent slowing of functional decline, with the absolute treatment benefit maintained in Month 48.”

Walid Abi-Saab, Chief medical officer of uniQure

“Furthermore, the observed differences between the high and low doses on both composite Unified Huntington’s Disease Rating Scale (cUHDRS) and TFC are consistent with a dose-dependent treatment effect.”

Walid Abi-Saab, Chief medical officer of uniQure

“The updated 36-month analysis, which now includes three additional high-dose patients, showed a substantial effect on cUHDRS and TFC, further reinforcing the data included in our license applications.”

Walid Abi-Saab, Chief medical officer of uniQure

“We believe these data are clinically meaningful for Huntington’s disease patients, and we look forward to presenting them at a future scientific meeting.”

Victor Sung, Professor of neurology at the University of Alabama at Birmingham and director of the UAB Huntington’s Disease Clinic

“Huntington’s disease does not slow on its own; the biology is one of inevitable progressive decline.”

Victor Sung, Professor of neurology at the University of Alabama at Birmingham and director of the UAB Huntington’s Disease Clinic

“What I find particularly notable in the expanded data is the consistently meaningful treatment effect at 36 months, and the apparent stability of the functional capacity benefit through Month 48.”

Victor Sung, Professor of neurology at the University of Alabama at Birmingham and director of the UAB Huntington’s Disease Clinic

“We see this even as the rate of decline in the updated comparator dataset slowed, an anticipated shift which appears to reflect some attrition in the longitudinal external control data, and which does not reflect the typical clinical presentation of accelerating decline over time.”

Victor Sung, Professor of neurology at the University of Alabama at Birmingham and director of the UAB Huntington’s Disease Clinic

“Total Functional Capacity tracks things that matter the most to patients and families – ability to work, perform household chores and handle daily self-care activities.”

Victor Sung, Professor of neurology at the University of Alabama at Birmingham and director of the UAB Huntington’s Disease Clinic

“Seeing that treatment difference maintained at four years is meaningful for people living with this relentlessly progressive degenerative disease.”