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
uniQure reported additional data from the ongoing Phase I/II studies of ifezuntirgene inilparvovec (AMT-130) in Huntington's disease, with the 36-month analysis of 15 high-dose patients showing 80% slowing of disease progression on cUHDRS (nominal p=0.005) and 67% on TFC (nominal p=0.011) versus an updated external control.
Key figures
- Drug Name
- ifezuntirgene inilparvovec (AMT-130)
- Phase Of Trial
- Phase I/II
- TFC slowing 36mo
- 67% (nominal p=0.011)
- TFC slowing 48mo
- 61% (nominal p=0.008)
- External control n
- 1337
- C UHDRS slowing 36mo
- 80% (nominal p=0.005)
- C UHDRS slowing 48mo
- 44% (p=0.144, non-significant)
- Patients dosed total
- 51
- Treatment related SAEs
- 5 high-dose patients (17%), CNS inflammation, all resolved
- Control missingness 48mo
- 53%
- Post hoc 48mo TFC slowing
- 68.3% (nominal p=0.001)
- Post hoc 48mo c UHDRS slowing
- 53.5% (nominal p=0.041)
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