uniQure Faces Lawsuit After Stock Plunge
A securities fraud class action lawsuit has been filed against gene therapy firm uniQure N.V. The suit follows an FDA approval delay for one of its products, which allegedly contributed to a 49% decline in the company's stock price.
The core of the uniQure lawsuit revolves around the company's gene therapy candidate, AMT-130, for Huntington's disease. The suit alleges that uniQure made misleadingly positive statements about the likelihood of accelerated FDA approval based on its Phase I/II clinical trials. These statements, made between September and October 2025, contributed to a nearly 250% surge in the company's stock price. The dramatic stock decline followed uniQure's November 2025 announcement that the FDA had reversed its earlier stance. Regulators stated that the existing trial data, which compared AMT-130 patients to an external control group from a natural history study, would likely be insufficient to support a Biologics License Application (BLA). This was a significant shift from a December 2024 agreement where the FDA had indicated this pathway could be acceptable. From a technical perspective, AMT-130 is an AAV5 vector-based therapy designed to silence the huntingtin gene using a proprietary microRNA (miQURE). Prior to the setback, uniQure had expressed confidence in its manufacturing and controls (CMC), with the FDA agreeing in early 2025 that the process validation could leverage experience from the company's previously developed approved therapy, HEMGENIX. The regulatory issue appears centered on the clinical trial design and statistical analysis plan, not the product's manufacturing process itself. Despite the clinical trial showing a 75% slowing of disease progression at 36 months, the FDA's reversal has thrown the BLA submission timeline into question. The lawsuit alleges that uniQure failed to disclose that its study design was not fully approved by the FDA. There is no cure or disease-modifying treatment currently approved for Huntington's, a fatal neurodegenerative disorder. Financially, uniQure reported revenues of $3.7 million in the third quarter of 2025 with a net loss of $80.5 million. The company's cash, cash equivalents, and investment securities stood at $694.2 million as of September 30, 2025, following a public offering that raised approximately $323.7 million in net proceeds. This financial runway will be critical as the company navigates the now-extended regulatory process. Looking ahead, uniQure has scheduled a high-priority "Type A" meeting with the FDA to discuss the path forward for AMT-130. This urgent meeting is specifically for stalled development programs and aims to clarify what additional data or analysis would be required to support an application for accelerated approval. The outcome of this meeting will be a critical determinant of the company's future direction and the timeline for potentially bringing AMT-130 to market.
