Iniulat ng uniQure ang 36-buwang datos ng Phase I/II ng AMT-130 sa Huntington's disease

The 36-month topline readout shows high-dose AMT-130 met the pre-specified primary endpoint (cUHDRS) and key secondary endpoint (TFC) versus propensity-matched external controls, with statistically significant slowing of disease progression and CSF NfL levels below baseline. The data support continued engagement with FDA on a potential accelerated-approval pathway and position AMT-130 as the first disease-modifying therapy candidate for HD.

Mahahalagang fact

Na-file: Ene 13, 2026, 12:05 PM
Kaganapan: Topline na resulta
Direksyon: Positibo
Kumpanya: QUREuniQure
Asset: AMT-130
Pinagmulan: SEC 8-K
Kumpiyansa: May source
Pagsubaybay: 36 buwan
Pagbagal ng TFC: 60% (p=0.033)
Pagbawas ng NfL: -8.2%
Pagbagal ng cUHDRS: 75% (p=.003)

Sipi mula sa source

EX-99.1 2 qure-20260113xex99d1.htm EX-99.1 Sophia – Huntington’s Disease Community Advocate Leadership in Gene Therapy January 2026 Exhibit 99.1 LEADERSHIP IN GENE THERAPY JANUARY 2026 | 2 Disclaimer This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the “Securities Act”), and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to,” “may,” “plan,” “potential,” “predict,” “project,” “should,” "will,” “would” and similar expressions and the negatives of those terms. Forward-looking st

SEC 8-K