Shares of uniQure N.V. (QURE) are down by 42% following the regulatory update and results. The company reported a net loss as operating expenses increased with continued investment in its gene therapy pipeline.
For the full year 2025, uniQure reported revenues of $16.1 million, a decline from $27.1 million in 2024.
The company posted a net loss of $199.0 million, or $3.46 basic and diluted loss per ordinary share, narrowing from a $239.6 million net loss for the same period in 2024, or $4.92 basic and diluted loss per ordinary share.
Separately, uniQure announced that it received final meeting minutes from the U.S. FDA regarding a Type A meeting held on January 30, 2026, to discuss AMT-130, its investigational gene therapy for Huntington's disease. The FDA stated that Phase 1/2 data compared to an external control are not sufficient to support a marketing application and strongly recommended a prospective, randomized, double-blind, sham surgery- controlled study.
uniQure intends to request a Type B meeting in the second quarter of 2026 to further discuss Phase 3 development considerations.
The company also provided update across its pipeline. Enrollment was completed in the first cohort of Phase 1/2a study of AMT-260 in refractory mesial temporal lobe epilepsy, with additional data expected in the first half of 2026.
In Fabry disease, updated Phase 1/2 results from AMT-191 showed durable, dose-dependent increases in a-Gal A enzyme activity, with some patients successfully withdrawn from enzyme replacement therapy.
Meanwhile, enrolment in AMT-162 for SOD1 amyotrophic lateral sclerosis (ALS) remains on voluntary pause following an Independent data in September 2025. This included one serious adverse event of dose-limiting toxicities determined to be related to AMT-162. The company continues to collect and evaluate data from the five patients treated in Phase 1/2 EPISOD1 study.
As of December 31, 2025, uniQure reported cash, cash equivalents and current investment securities of approximately $622.5 million, expected to fund operations into the second half of 2029.
QURE has traded between $7.76 and $71.50 over the past year. The stock is currently trading at $9.17, down 42%.
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