uniQure (QUREN) Q3 2026 Earnings Report

The call conveyed strong positive momentum, led by FDA priority review and MHRA validation for AMT-130, statistically significant disease-progression slowing, progress toward the confirmatory study, commercial preparation and a cash runway into 2030. The company also reported higher operating expenses, mixed early AMT-260 responses, a prior AMT-191 enrollment pause and remaining uncertainty around regulatory review, access and launch capacity. Highlights significantly outweighed the lowlights.

uniQure expects the AMT-130 confirmatory study to be a global randomized study with a 2:1 ratio of AMT-130 high dose to standard of care, approximately 200 patients, and TFC at 36-month as the primary endpoint; patient screening is expected to begin by the end of this year, with the study well underway by the May 1, 2027 PDUFA date, and standard-of-care participants offered AMT-130 in a separate open-label study after 36-month. The company expects updated AMT-260 and AMT-191 data in the first half of 2027, its core commercial team fully in place by early next year, and a potential U.S. launch in 2027; it expects to be north of 15 treatment centers at launch, although whether that could be 25 or 30 has not been defined, with center capacity ranging from 2 a week to one a month. Cash, cash equivalents and investment securities are expected to fund operations into 2030, and uniQure expects to provide a day 120 safety update.