uniQure reports updated AMT-130 Huntington's data; 48-month cUHDRS result not significant
uniQure presented additional data from its ongoing Phase I/II studies of AMT-130 for Huntington's disease, comparing treated patients with updated external controls. The 36-month analysis of all 15 high-dose patients showed 80% slowing on cUHDRS (nominal p=0.005) and 67% on TFC (nominal p=0.011), and the company says the BLA submission was based on 36-month data. The 48-month primary cUHDRS result showed 44% slowing but did not reach statistical significance (p=0.144), and the company notes that missing data and survivor bias in the updated control may understate the treatment effect.
Key points
- 36-month analysis of 15 high-dose patients: 80% slowing of cUHDRS decline (nominal p=0.005) and 67% slowing of TFC decline (nominal p=0.011)
- 48-month analysis of 12 high-dose patients: 44% slowing on cUHDRS (p=0.144, not significant) and 61% slowing on TFC (nominal p=0.008)
- Data missingness in the updated Enroll-HD B control reached 53% at Month 48; data cutoff was June 30, 2026
- Company says the BLA was submitted on 36-month data, which the FDA said at a June 2026 Type B meeting would be acceptable under the accelerated approval pathway
The filing
Written by AI on Nov 9, 2026, 8:38 AM ET, from this filing only. Written later: this filing was briefed after the fact, when past filings were added to the site, so the brief may be read with hindsight; the filing itself is the record of what was known on September 29, 2026.
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