uniQure N.V. (QURE) SEC Filings & Earnings Calls

Clinical trial risk remains; data based on small sample (N=12 high-dose) and external control may have survivor bias

“Survivor bias and missingness in the updated external control likely understate disease progression and treatment effect at 48 months” · word for word

Regulatory approval not guaranteed; clinical trial data may not support approval

“BLA submission based on Phase I/II data; FDA review outcome uncertain”

AMT-191 program paused due to dose-limiting toxicities; liver enzyme elevations.

“Additional dosing in mid- and high-dose cohorts remains paused, pending further evaluation of asymptomatic liver enzyme elevations.”

Clinical trial risk; AMT-260 data based on small cohort with variable responses.

“Three of six patients achieved meaningful reductions; remaining three had variable changes.”

FDA regulatory reversal risk for AMT-130 accelerated approval

“In December 2025, we announced that in the final meeting minutes, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 were currently unlikely to provide the primary evidence to support a BLA submission.” · word for word

Dose-limiting toxicity in AMT-191 Fabry trial

“Per protocol, additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation following asymptomatic Grade 3 liver enzyme elevations observed in two patients in the mid-dose cohort, which were confirmed dose-limiting toxicity.” · word for word

Dependence on future financing for operations

“If adequate funds are not available to us on acceptable terms when we need them, we may be unable to pursue further development of our clinical product candidates.” · word for word

Foreign exchange volatility impact on financial results

“We recognized a net foreign currency loss of $1.7 million during the three months ended June 30, 2026, compared to a net gain of $18.6 million during the same period in 2025.” · word for word

Dilution from equity offering; 5.7M new shares issued.

“Underwriters exercised option in full for 741,758 additional shares.”

Small sample size (6 patients); variable responses; early stage data.

“Remaining three patients experienced variable changes...ranging from a 33% decrease to a 36% increase.”

Clinical trial risk; longer follow-up needed.

“Although these findings are based on a limited number of patients and require longer follow-up.” · word for word

Confirmatory study design not yet finalized; FDA seeks alignment prior to BLA submission.

“FDA seeks alignment with the Company on the confirmatory study design, including consideration of a concurrent control of standard of care.” · word for word

Accelerated approval pathway subject to confirmatory study feasibility and enrollment.

“FDA stated that the confirmatory study should be feasible to conduct within a reasonable timeline and be well underway.”

FDA regulatory setback for lead asset AMT-130

“In March 2026, following receipt of the final meeting minutes from the Type A meeting, we announced that the FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommend” · word for word

Clinical hold or dosing pause on AMT-191 due to dose-limiting toxicity

“Per protocol, additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation following asymptomatic Grade 3 liver enzyme elevations observed in two patients in the mid-dose cohort, which were confirmed dose-limiting toxicity.” · word for word

Discontinuation of AMT-162 program due to safety issue

“Following review of the preliminary efficacy and safety data generated from EPISOD1, we have decided to discontinue development of AMT-162.” · word for word

Significant milestone payment obligations for AMT-260

“As of March 31, 2026, our remaining commitment amounts include EUR 160.0 million ($184.0 million) in potential milestone payments associated with Phase III development and the approval of AMT-260 in the U.S. and European Union.” · word for word

Dependence on single partner for revenue (CSL Behring)

“We recognize license revenues from CSL Behring related to royalty payments owed on HEMGENIX® sales, when earned.” · word for word

FDA rejection of AMT-130 Phase I/II data as primary evidence for BLA; regulatory path uncertain in US.

“The FDA stated it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130.” · word for word

AMT-191 dosing paused due to dose-limiting toxicities (Grade 3 liver enzyme elevations) in mid-dose cohort.

“Additional dosing in the mid- and high-dose cohorts has been paused pending further evaluation of asymptomatic Grade 3 liver enzyme elevations reported in two patients from the mid-dose cohort, which were confirmed as dose-limiting toxicities.” · word for word

AMT-162 development discontinued after dose-limiting toxicity observed.

“Following review of the preliminary efficacy and safety data generated from EPISOD1, the decision was made to discontinue development of AMT-162.” · word for word

FDA rejection of AMT-130 external control data; requires new randomized controlled trial, significantly delaying and increasing cost of approval pathway.

“FDA stated it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommended the Company conduct a prospective, randomized, double-blind, sham surgery-controlled study.” · word for word

All pipeline assets are early-stage (Phase I/II); no approved products; single point of failure risk on AMT-130.

“AMT-130 for Huntington's disease; AMT-260 for refractory mesial temporal lobe epilepsy; AMT-191 for Fabry disease”

FDA regulatory setback for lead candidate AMT-130

“the FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for AMT-130. The FDA strongly recommended we conduct a prospective, randomized, double-blind, sham surgery-controlled study.” · word for word

Significant accumulated deficit and ongoing net losses

“We had a net loss of $199.0 million in the year ended December 31, 2025 and a net loss of $239.6 million and $308.5 million during the same periods in 2024 and 2023, respectively. As of December 31, 2025, we had an accumulated deficit of $1,328.9 million (December 31, 2024: $1,130.0 million).” · word for word

Dependence on AMT-130 for near-term value

“The increase of $24.5 million in external cost in 2025 compared to 2024 includes $19.4 million of costs incurred in relation to the preparation of the BLA submission for AMT-130 in the U. S.” · word for word

High debt and royalty financing costs

“We recognized non-cash interest expenses related to the Royalty Financing Agreement of $54.1 million in 2025, $50.9 million in 2024 and $26.9 million in 2023, respectively.” · word for word

FDA regulatory uncertainty for AMT-130 BLA submission; potential delay or additional trial requirement.

“FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an external control... may be adequate to provide the primary evidence in support of a BLA submission.” · word for word

Dose limiting toxicity in AMT-162 trial; enrollment paused.

“voluntarily paused enrollment in the study as a dose limiting toxicity, which resulted in a serious adverse event determined to be related to AMT-162, was observed in one patient in the second cohort.” · word for word

FDA no longer agrees Phase I/II data may be adequate for BLA submission

“based on discussions at a pre-BLA meeting held with the FDA, we believe that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence ” · word for word

Voluntary pause in EPISOD1 trial due to dose limiting toxicity

“we have voluntarily paused enrollment in EPISOD1, our Phase I/II clinical trial of AMT-162, upon the recommendation of the IDMC following a September 2025 review of available preliminary data related to the safety and efficacy of AMT-162 in the context of a dose limiting toxicity, which resulted in a SAE determined to be related to AMT-162, that was observed” · word for word

Dependence on single lead product candidate AMT-130

“We are dependent on the success of our lead product candidate, AMT-130, for the treatment of Huntington’s disease.” · word for word

Regulatory uncertainty due to FDA leadership changes and budget cuts

“the recent loss of FDA leadership and personnel, including the administration’s recent reduction in force and budget cuts, and new leadership at the U.S. Department of Health and Human Services and the FDA, including the Center for Drug Evaluation and Research and Center for Biologics Evaluation and Research, could lead to disruptions and delays in FDA guida” · word for word