uniQure melaporkan data 36 bulan AMT-130 Fase I/II pada penyakit Huntington

Pembacaan topline 36 bulan menunjukkan dosis tinggi AMT-130 memenuhi titik akhir primer yang telah ditentukan sebelumnya (cUHDRS) dan titik akhir sekunder utama (TFC) dibandingkan dengan kontrol eksternal yang dicocokkan dengan kecenderungan, dengan perlambatan progresi penyakit yang signifikan secara statistik dan kadar CSF NfL di bawah baseline. Data ini mendukung keterlibatan berkelanjutan dengan FDA mengenai jalur persetujuan yang dipercepat dan menempatkan AMT-130 sebagai kandidat terapi pertama yang memodifikasi penyakit untuk HD.

Fakta utama

Diajukan: 13 Jan 2026, 12.05
Peristiwa: Hasil topline
Arah: Positif
Perusahaan: QUREuniQure
Aset: AMT-130
Sumber: SEC 8-K
Keyakinan: Bersumber
tindak lanjut: 36 bulan
perlambatan TFC: 60% (p=0.033)
penurunan NfL: -8.2%
perlambatan cUHDRS: 75% (p=.003)

Kutipan sumber

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