uniQure (QURE): Focus shifts to commercial amidst regulatory clarity in Huntington’s disease

研报英文原文证据摘录

uniQure (QURE): Focus shifts to commercial amidst regulatory clarity in Huntington’s disease

Goldman Sachs uniQure (QURE)

approval. To support study recruitment and retention, management noted that in the

earlier part of the study, enrollment will focus on geographies where AMT-130 would

not yet be commercially available. Further, enrolled patients will be able to receive

AMT-130 at the end of the three-year study regardless of eligibility criteria (as long as it

is safe).

Ex-US, QURE recently announced that following a meeting with the UK’s MHRA (~7-8K

HD patients), QURE plans to submit a Marketing Authorization Application for AMT-130

in 3Q. On the potential UK launch, QURE noted that several centers of excellence are

involved in the ongoing Ph1/2 study and are thus prepared to administer AMT-130.

Additionally, management noted that review timelines are dependent on the number

and complexity of the questions that arise. Outside of the UK, QURE is engaging with

additional EU regulators, focusing on markets with named-patient and early-access

programs (e.g. Germany, France, and Italy).

Monitoring earlier stage pipeline progress

We continue to monitor additional pipeline gene therapy assets:

n AMT-260 in refractory temporal lobe epilepsy (rTLE), where QURE recently

presented initial six-month follow-up data on the first, low dose cohort (n=6) in its

ongoing Ph1/2a trial of an investigational gene therapy candidate, AMT-260, for the

treatment of refractory mesial temporal lobe epilepsy (MTLE) at the Epilepsy

Foundation Pipeline Conference (June 19). As of the May 29, 2026 data cutoff date,

three of six patients in the first, low-dose cohort (1x1012 gc/mL) achieved

meaningful reductions in disabling seizures during months four through six of