AMT-130

July 29, 2026: uniQure N.V. (NASDAQ: QURE) reported Q2 2026 results and provided regulatory/clinical updates across its gene therapy pipeline.

AMT-130 (Huntington’s disease): Following a June 2026 FDA Type B meeting, July 2026 minutes confirm alignment that an accelerated approval BLA based on existing clinical data is “reasonable.” FDA requested alignment on confirmatory study design before BLA submission, including considering a randomized standard-of-care control instead of a sham procedure, and indicated the confirmatory study should be well underway (potentially fully enrolled) at the time of accelerated approval. uniQure expects U.S. BLA submission in Q3 2026 and a U.K. MHRA submission also in Q3 2026 (after a March 2026 pre-submission meeting). Topline four-year Phase I/II data are expected in September 2026, including four-year follow-up on 24 patients across low- and high-dose cohorts.

AMT-260 (refractory mesial temporal lobe epilepsy): Preliminary 6-month data (low-dose cohort; n=6) showed 3/6 patients with 79%–100% reductions in disabling seizures during months 4–6; others ranged from 33% decrease to 36% increase. No treatment/procedure-related SAEs as of a June 19, 2026 presentation; AEs were mild/moderate; no immunosuppression required. Enrollment of the second dose cohort expected to complete in Q3 2026; updated results expected in 1H 2027.

AMT-191 (Fabry disease): Phase I/II data (cutoff March 15, 2026) showed dose-dependent α-Gal A activity increases across 11 patients; lyso-Gb3 stable; all 11 withdrawn from ERT. Additional mid/high-dose dosing remains paused due to dose-limiting asymptomatic liver enzyme elevations in two mid-dose patients (resolved by end of May 2026 with protocol immunosuppression).

Financing: Closed a $259M follow-on offering in June 2026; cash/investments were $810.3M at June 30, 2026, with runway projected into 2030.