uniQure reported FY 2025 revenue of USD 16.1 million (down USD 11.0 million), with R&D expenses of USD 140.7 million (down USD 3.1 million) and SG&A expenses of USD 65.5 million (up USD 12.8 million). Net loss for FY 2025 was USD 199.0 million, or USD 3.46 per share. Cash, cash equivalents and current investment securities totaled USD 622.5 million as of December 31, 2025, and the company said this is expected to fund operations into the second half of 2029. On the business update, uniQure said it held a Type A meeting with the FDA in January 2026 on AMT-130 for Huntington’s disease after a pre-BLA meeting in October 2025; the FDA said Phase I/II data versus an external control are not sufficient as primary evidence of effectiveness and recommended a prospective, randomized, double-blind, sham surgery-controlled study. The company said it is evaluating Phase III considerations and plans to request a Type B meeting in Q2 2026. uniQure also said it completed enrollment of the first six-patient cohort in the Phase I/IIa AMT-260 study in refractory mesial temporal lobe epilepsy and expects updated data in Q2 2026, while updated Phase I/II AMT-191 Fabry data showed durable, dose-dependent increases in α-Gal A activity; additional dosing in mid- and high-dose cohorts has been paused pending evaluation of asymptomatic Grade 3 liver enzyme elevations in two mid-dose patients that were confirmed as dose-limiting toxicities. Enrollment for AMT-162 in SOD1 ALS remains on voluntary pause following an IDMC recommendation after a dose-limiting toxicity SAE reported in September 2025.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. uniQure NV published the original content used to generate this news brief via GlobeNewswire (Ref. ID: 202603020705PRIMZONEFULLFEED9663657) on March 02, 2026, and is solely responsible for the information contained therein.