uniQure Faces FDA Setback on AMT-130 as Investors Navigate Legal and Regulatory Uncertainty

uniQure N.V. disclosed that the U.S. Food and Drug Administration stated it cannot agree that data from the Phase I/II studies of AMT-130, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application. On November 3, 2025, the company acknowledged that the FDA no longer agrees that the 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 Biologics License Application submission.

Following this announcement on November 3, 2025, uniQure's share price fell $33.40, or more than 49%, from $67.69 on October 31, 2025, to close at $34.29. The stock experienced a significant price spike on March 20, 2026, climbing over 12.9% in one of its most volatile sessions recently, with an intraday high of $17.72.

The company held a pre-BLA meeting with the FDA in October 2025 and a Type A meeting in January 2026 to discuss the regulatory path forward. Following receipt of final meeting minutes from the Type A meeting, the FDA strongly recommended conducting a randomized controlled trial. The company is evaluating Phase III development considerations and plans to request a follow-up Type B meeting in the second quarter of 2026.

In September 2025, uniQure announced positive topline data from the pivotal Phase I/II study for AMT-130 for the treatment of Huntington's disease. Topline 36-month efficacy results for 12 patients receiving high-dose AMT-130 included a statistically significant 75% slowing in disease progression measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS), which met the primary endpoint compared to a propensity score-matched external control (p=0.003). A key secondary endpoint of Total Functional Capacity (TFC) demonstrated a statistically significant 60% slowing of disease progression compared to a propensity score-matched external control (p=0.033). A mean reduction from baseline in cerebrospinal neurofilament light protein (NfL) of -8.2% was observed at 36 months in the high-dose of AMT-130 of the Phase I/II studies.

AMT-130 was generally well-tolerated across both doses, with a manageable safety profile and no new drug-related serious adverse events observed since December 2022. The most common adverse events in the treatment groups were related to the administration procedure. In the fourth quarter of 2025, dosing was completed in a fourth cohort of six patients receiving high-dose AMT-130 to evaluate safety and efficacy in patients with lower baseline striatal volumes compared to previous cohorts in the U.S. Phase I/II study.

On February 23, 2026, the company presented new analyses at the 21st Annual Huntington's Disease Therapeutics Conference in Palm Springs, California showing that propensity score methodology using clinical covariates with TRACK-HD/TRACK-ON and PREDICT-HD datasets effectively substitutes for baseline striatal volume in predicting Huntington's disease progression.

Investors who purchased shares of uniQure between September 24, 2025, and October 31, 2025, have until April 13, 2026, to file lead plaintiff applications in a securities class action lawsuit. The lawsuit, filed in the United States District Court for the Southern District of New York, alleges that uniQure and certain executives failed to disclose material information during the class period, violating federal securities laws. The case is identified as Scocco v. uniQure N.V., et al., Case No. 1:26-cv-01124.

Multiple class-action suits allege that the company and its leadership misrepresented aspects of its Pivotal Study and underestimated potential regulatory delays for its Biologics License Application. Several law firms are seeking investors to serve as lead plaintiffs in the ongoing securities litigation.

Beyond Huntington's disease, uniQure completed enrollment of the first cohort in the Phase I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy, with additional clinical data expected in the first half of 2026. The company presented updated Phase I/II data from AMT-191 in Fabry disease showing durable, dose-dependent increases in α-Gal A enzyme activity. All 11 patients across three dose cohorts exhibited elevated α-galactosidase A (α-Gal A) activity, with the highest dose cohort demonstrating activity ranging from 27.7- to 223.7-fold above normal levels.

uniQure reported cash, cash equivalents and current investment securities of approximately $622.5 million as of December 31, 2025, expected to fund operations into the second half of 2029.