Key Highlights

• FDA granted priority review for uniQure's BLA for ifezuntirgene inilparvovec (AMT-130) with a PDUFA target action date of May 1, 2027

• Updated 36-month analysis of all 15 high-dose patients showed an 80% slowing on cUHDRS and 67% slowing on TFC

• Cash, cash equivalents and Investment securities stood at approximately $768.4 million as of September 30, 2026, expected to fund operations into 2030

• Company aligned with FDA on key elements of the confirmatory study design, with U.S. patient screening expected to begin by end of 2026

uniQure N.V. (NASDAQ:QURE), a gene therapy company focused on transformative therapies for patients with severe medical needs, has reported its third quarter 2026 financial results alongside a series of developments in its pipeline. The most notable among these is the FDA's acceptance of the company's Biologics License Application for ifezuntirgene inilparvovec, its lead candidate in Huntington's disease, under priority review.

FDA Milestone and Clinical Progress

The BLA, submitted under the Accelerated Approval pathway, received a Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027. The application is supported by 36-month data from 12 high-dose patients compared with a propensity score-matched external control.

In September 2026, uniQure disclosed additional data from its ongoing Phase I/II studies with a data cutoff of June 30, 2026. Among 12 high-dose patients with 48 months of follow-up, the composite Unified Huntington's Rating Scale (cUHDRS) showed a 44% slowing of disease progression versus the external control. Total Functional Capacity (TFC), the primary measure for the confirmatory study, demonstrated a 61% slowing at 48 months.

An updated 36-month analysis including all 15 high-dose patients indicated an 80% slowing on cUHDRS and a 67% slowing on TFC. The therapy was described as generally well tolerated with a manageable safety profile across 51 total treated patients. The company has also aligned with the FDA on key elements of the confirmatory study, designed as a randomized trial enrolling approximately 200 patients with TFC as the primary endpoint at 36 months.

Pipeline Developments Beyond Huntington's Disease

Beyond its Huntington's disease program, uniQure reported continued clinical progress in other pipeline programs. For AMT-260, being developed for refractory mesial temporal lobe epilepsy, the company completed enrollment in the second dose cohort of seven patients in its Phase I/IIa study, with updated results expected in the first half of 2027.

In its Fabry disease program, enrollment in the mid- and high-dose cohorts of the Phase I/II study of AMT-191 resumed in August 2026, following a protocol-related dosing pause after observing asymptomatic liver enzyme elevations in two mid-dose patients. Results from all dosing cohorts are expected in the first half of 2027.

Financial Performance in Q3 2026

Revenue for the three months ended September 30, 2026 was $5.2 million, compared to $3.7 million in the same period in 2025. The $1.5 million increase was attributed to higher license revenue.

Research and development expenses were $35.5 million for the quarter, up slightly from $34.4 million in the year-ago period, with the increase driven primarily by a $3.6 million rise in employee and contractor-related expenses. This was partially offset by a $2.3 million decrease in direct Research and Development expenses, largely reflecting a $4.1 million reduction in process validation costs related to ifezuntirgene inilparvovec.

Selling, general and administrative expenses rose to $23.9 million from $19.4 million, reflecting a $3.0 million increase in employee and contractor-related expenses tied to recruitment efforts over the previous 12 to 15 months, along with increased professional fees and IT costs related to potential commercial launches.

The net loss for the quarter narrowed to $61.2 million, or $0.88 per share, compared to $80.5 million, or $1.38 per share, in the prior year period. Cash, cash equivalents and investment securities totaled approximately $768.4 million as of September 30, 2026, up from $622.5 million at year-end 2025.

What Lies Ahead

uniQure is positioned at a pivotal juncture as it awaits the FDA's decision on its BLA, expected by May 2027. The company continues to build out its commercial capabilities, including field medical, market access, and strategic account teams. In the U.K., the Medicines and Healthcare products Regulatory Agency has validated the Marketing Authorization Application for the therapy. With a cash runway extending into 2030 and multiple pipeline readouts anticipated in 2027, the coming quarters will be defining for the company's trajectory.