uniQure Narrows Third-Quarter 2026 Net Loss to $61.2 Million

The Tip Desk · November 4, 2026 · 4 min read

The gene therapy company’s third-quarter 2026 revenue rose to $5.2 million from $3.7 million a year earlier, while its net loss narrowed to $61.2 million, or $0.88 a share. Cash, cash equivalents and investment securities reached $768.4 million.

uniQure N.V. (QURE) reported third-quarter 2026 revenue of $5.2 million, compared with $3.7 million a year earlier. Net loss narrowed to $61.2 million, or $0.88 a share, from $80.5 million, or $1.38 a share.

The gene therapy company, which is advancing treatments for severe medical needs, said the U.S. Food and Drug Administration accepted its Biologics License Application for ifezuntirgene inilparvovec in Huntington’s disease and granted Priority Review, with a Prescription Drug User Fee Act target action date of May 1, 2027.

The company had said in its second-quarter update that it expected to submit the BLA in the third quarter of 2026 after a June Type B meeting in which the FDA confirmed that an accelerated-approval pathway based on existing clinical data was reasonable. The application is supported by 36-month data from 12 high-dose patients compared with a propensity score-matched external control.

In September, the company reported additional Phase I/II data through June 30, 2026. Among 12 high-dose patients with 48 months of follow-up, the composite Unified Huntington’s Rating Scale showed a 44% slowing of disease progression versus an updated external control (non-significant p=0.144). Total Functional Capacity, the planned primary endpoint for the confirmatory study, showed a 61% slowing (nominal p=0.008) at 48 months. An updated 36-month analysis including all 15 high-dose patients showed an 80% slowing on cUHDRS (nominal p=0.005) and a 67% slowing on TFC (nominal p=0.011). The company said the therapy was generally well tolerated across 51 treated patients.

uniQure and the FDA aligned on a confirmatory trial of approximately 200 patients randomized 2:1 to treatment or standard of care, with TFC as the primary endpoint at 36 months. The company plans to begin U.S. patient screening by the end of 2026. In the third quarter, the U.K. MHRA validated the Marketing Authorization Application submitted earlier in the period.

Chief executive Matthew Kapusta said the company is building field medical, market access and strategic account teams and engaging U.S. Huntington’s disease treatment centers as it prepares for a potential launch.

Revenue for the three months ended September 30, 2026 was $5.2 million, compared with $3.7 million a year earlier. The company attributed the $1.5 million increase to higher license revenue.

Research and development expenses were $35.5 million, versus $34.4 million in the year-ago quarter. The company said the $1.1 million increase reflected a $3.6 million rise in employee and contractor costs including share-based compensation, partly offset by a $2.3 million drop in direct R&D, including a $4.1 million reduction in ifezuntirgene inilparvovec process-validation expenses.

Selling, general and administrative expenses were $23.9 million, versus $19.4 million a year earlier. The company said the $4.5 million increase was driven mainly by a $3.0 million rise in employee and contractor costs from 12-to-15-month recruitment, plus $0.7 million each in professional fees and information-technology costs to support a potential commercial launch of ifezuntirgene inilparvovec.

Net loss narrowed to $61.2 million, or $0.88 a share, from $80.5 million, or $1.38 a share, a year earlier. Non-operating items, net, improved to a $7.6 million expense from $20.9 million, which the company said reflected a $6.3 million reduction in pre-funded warrant expenses and a $6.8 million improvement in net interest income.

Cash, cash equivalents and investment securities were $768.4 million as of September 30, 2026, versus $622.5 million as of December 31, 2025. The company said those resources are expected to fund projected operating expenses into 2030.

In the pipeline, uniQure completed enrollment in the second dose cohort of seven patients in the Phase I/IIa study of AMT-260 for refractory mesial temporal lobe epilepsy and expects updated results in the first half of 2027. Enrollment in the mid- and high-dose cohorts of the AMT-191 Phase I/II study in Fabry disease resumed in August 2026. Dosing had previously been paused following asymptomatic liver-enzyme elevations in two mid-dose patients that resolved with immunosuppression. The company expects updated results from all AMT-191 dosing cohorts in the first half of 2027.