uniQure reports AMT-130 Phase I/II data: 80% slowing of Huntington's progression at 36 months in high-dose patients

uniQure N.V.

At 36 months, 15 high-dose patients showed 80% slowing on cUHDRS (nominal p=0.005) and 67% on TFC (p=0.011) vs the updated ENROLL-HD external control.
At 48 months (n=12 high dose), cUHDRS slowing was 44% (p=0.144, not significant) and TFC 61% (p=0.008); a post-hoc prior-control analysis showed 53.5% and 68.3%.
FDA said 36-month data from 12 high-dose patients could support the accelerated-approval BLA, which was submitted before today's topline results were available.
Five high-dose patients (17%) had treatment-related serious adverse events from CNS inflammation, all resolved; one low-dose patient suicide ~5 years post-treatment was assessed as unrelated.
uniQure hosts an investor call today at 8:30 a.m. ET; AMT-130 holds FDA Breakthrough Therapy, RMAT and Fast Track designations.

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