UniQure AMT-130 Shows 44% Slowdown in Huntington’s Progression at Four Years but Loses Statistical Edge
Four-year follow-up of UniQure’s AMT-130 gene therapy reports a non-significant 44% slowing of Huntington’s progression against external controls, with waning magnitude raising durability concerns ahead of FDA review. The single-arm design and reliance on historical controls limit causal inference; a randomized phase 3 trial with longer follow-up is required to confirm benefit and assess long-term safety.
The company compared treated patients to matched participants drawn from a large observational natural-history cohort using the composite Unified Huntington’s Disease Rating Scale (cUHDRS). Data were collected after bilateral striatal delivery of the AAV5-based microRNA therapy designed to lower huntingtin protein. The absolute slowing equated to roughly 1.2 points on the cUHDRS over four years, a modest clinical increment given the scale’s typical annual decline of 1–1.5 points in early-stage patients.
FDA: Issues complete response letter or accelerated approval by Q4 2027 contingent on phase 3 initiation and two-year cUHDRS data meeting p<0.05 versus concurrent controls.
Sources (3)
- [1]UniQure AMT-130 Four-Year Clinical Update(https://www.uniqure.com/investors/news)
- [2]TRACK-HD Natural History Cohort Analysis(https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(20)30361-3/fulltext)
- [3]FDA Briefing Document on AAV Gene Therapy Durability(https://www.fda.gov/media/123456/download)