uniQure Advances AMT-130 Filing as Loss Widens
Cash reached $810.3 million after a June offering extended the gene-therapy developer’s runway to 2030.
uniQure N.V. (QURE), a gene-therapy developer, said its second-quarter loss more than doubled as it prepared for a potential AMT-130 launch and advanced the Huntington’s disease treatment toward a U.S. regulatory submission.
The quarter marked a shift in AMT-130’s regulatory trajectory. After the Food and Drug Administration said in the first quarter that existing Phase I/II data using an external control were insufficient as primary evidence, July meeting minutes confirmed alignment that an accelerated-approval application based on the existing clinical results was reasonable. uniQure now expects to submit the biologics license application in the third quarter of 2026.
Revenue rose about 11% to $5.8 million from $5.3 million a year earlier, driven by higher license revenue. The net loss widened to $81.1 million, or $1.22 a share, from $37.7 million, or 69 cents a share. Net non-operating items swung to a $27.0 million expense from a $6.6 million gain.
Research-and-development spending declined to $34.0 million as lower facility, personnel and contingent-consideration costs outweighed increased direct spending on AMT-260, AMT-162 and AMT-191. Spending on lead program AMT-130 decreased. Selling, general and administrative expense rose 29% to $17.4 million, largely because employee and contractor costs increased as uniQure staffed for a potential launch.
The company also recorded $8.0 million of other expense, up from $2.2 million a year earlier, primarily because costs associated with supplying HEMGENIX to CSL Behring increased by $6.0 million.
Beyond AMT-130, early AMT-260 efficacy data showed three of six low-dose patients had 79% to 100% reductions in disabling seizures during follow-up months four through six. Outcomes for the other three ranged from a 33% reduction to a 36% increase, and no treatment- or procedure-related serious adverse events were reported. Enrollment of the second dose cohort is now expected to finish in the third quarter, later than the previous midyear target.
uniQure paused dosing in AMT-191’s mid- and high-dose cohorts after asymptomatic liver-enzyme elevations in two mid-dose patients were confirmed as dose-limiting toxicities. The elevations resolved by the end of May following immunosuppression. All 11 treated patients had withdrawn from enzyme-replacement therapy, and follow-up extended beyond 18 months, with no additional high-dose serious adverse events beyond the two reported in September 2025.
The June stock offering raised $259 million after underwriters fully exercised their option, exceeding the roughly $225 million initially priced. uniQure projects its cash will fund operations into 2030, while the AMT-130 confirmatory study must be aligned with the FDA before the application is submitted and could need to be fully enrolled by accelerated approval.