uniQure Q4 2025 Earnings Amid AMT-130 Regulatory Setback
uniQure reports Q4 2025 results as investors await clarity on AMT-130 Huntington's disease gene therapy following FDA's November indication that Phase I/II data unlikely to support biologics license application.
uniQure N.V. reported fourth-quarter 2025 results before the market opened Monday, with investors seeking updates on the regulatory fate of AMT-130, the company's Huntington's disease gene therapy, following an unexpected setback with the Food and Drug Administration. Analysts expected the Amsterdam-based biotech to post a loss of 92 cents per share on revenue of $5.29 million for the quarter.
The company demonstrated groundbreaking 3-year data from its Phase I/II study of AMT-130, which showed a statistically significant 75% slowing of disease progression. The treatment slowed disease progression by 75% after three years in a pivotal Phase I/II trial, results that initially sent shares soaring more than 240% in September.
In November, uniQure announced it was no longer aligned with the FDA on how to advance AMT-130, with the agency indicating that data from the Phase I/II trials would be "unlikely" to provide primary evidence for a biologics license application. The reversal came despite previous breakthrough therapy and regenerative medicine designations for the drug.
Investors are watching closely for any updates on the Type A meeting uniQure scheduled with the FDA to discuss the biologics license application data package for AMT-130. Type A meetings are urgent, high-priority discussions reserved for stalled programs, typically held within 30 days, and official minutes usually arrive shortly thereafter.
Efficacy, durability, and safety are not in question; rather, the debate centers on when confirmatory evidence must be generated. AMT-130 could generate more than $1.5 billion US peak sales for Huntington's disease if approved.
The company had planned to file a biologics license application in early 2026, but that timeline now appears uncertain. Ten of 14 analysts covering the stock rate it a Buy, with a mean price target of $53.14—implying more than 210% upside from the current price near $17. EPS estimates have edged up 0.8% over the past 60 days, while revenue estimates have declined 2.91% over the same period.
That would represent a sequential improvement from the third quarter, when uniQure reported a loss of $1.38 per share on revenue of $3.7 million, missing analyst expectations on both metrics. The modest estimate adjustments reflect ongoing uncertainty about uniQure's commercial timeline, particularly for AMT-130.