uniQure slides 2.9% as class action lawsuits mount over FDA rejection
Gene therapy company faces legal scrutiny after November regulatory setback crushed shares 49%
uniQure N.V. shares declined 2.9% on Wednesday as multiple law firms announced class action lawsuits against the gene therapy company, alleging investors were misled about regulatory hurdles for its Huntington's disease treatment.
The stock fell to approximately $24.00, extending losses for the Netherlands-based biotech whose market capitalization has contracted to $1.62 billion. Holzer & Holzer, LLC announced it is encouraging investors with significant losses to contact the firm, joining at least five other law firms that have filed similar complaints in the U.S. District Court for the Southern District of New York.
The lawsuits target investors who purchased uniQure ordinary shares between September 24, 2025, and October 31, 2025, seeking to recover damages following a devastating regulatory setback. According to complaints filed by several law firms, the company and certain executives made materially false or misleading statements regarding its lead gene therapy candidate, AMT-130.
At the heart of the allegations is uniQure's disclosure on November 3, 2025, that the U.S. Food and Drug Administration "currently 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 BLA submission." BusinessWire reported this revelation prompted a staggering 49% single-day decline, with uniQure shares plunging $33.40 to $34.29 on November 3, wiping out more than $1.6 billion in market value.
The complaints allege that uniQure failed to disclose that the design of its Pivotal Study for AMT-130, including comparison to the ENROLL-HD external historical data set, had not received full FDA approval. Furthermore, plaintiffs claim executives downplayed the likelihood that the company would need to delay its Biologics License Application timeline to conduct additional studies.
AMT-130 represents uniQure's most advanced clinical candidate, a gene therapy designed to treat Huntington's disease, a fatal neurodegenerative disorder. The company's proprietary adeno-associated virus platform targets genetic disorders and severe diseases, with AMT-130 considered the linchpin of its commercial prospects.
Despite the regulatory challenges, analysts have maintained a relatively bullish outlook on the stock. The average analyst target price stands at $53.67, according to market data, with 10 analysts rating the shares a buy and three recommending hold. That target implies more than 120% upside from current levels, reflecting optimism that uniQure can navigate the FDA's requirements and ultimately bring AMT-130 to market.
However, the company's financials reveal the high-risk nature of gene therapy development. uniQure reported trailing twelve-month revenue of just $15.8 million with a price-to-sales ratio of 103x, indicating investors are paying heavily for future growth that remains uncertain. The company posted an operating margin of negative 13.3% and earnings per share of negative $4.47.
The legal challenges come at a precarious time for uniQure, which has seen its shares swing dramatically between a 52-week high of $71.50 and a low of $7.76. Institutional investors own 95.7% of outstanding shares, meaning the class period potentially affected sophisticated market participants who conducted extensive due diligence.
Investors who purchased uniQure shares during the class period have until April 13, 2026, to apply to the court to be appointed as lead plaintiff in the consolidated litigation. Several law firms including Bragar Eagel & Squire, P.C., Robbins LLP, Kessler Topaz Meltzer & Check, LLP, and Kahn Swick & Foti, LLC have all announced investigations or filings related to the alleged securities violations.
The lawsuits allege that throughout the class period, defendants made positive statements about the company's business, operations, and prospects that lacked a reasonable basis in light of the undisclosed FDA concerns. The legal proceedings will likely focus on what company officials knew about the regulatory issues and when they became aware of potential problems with the AMT-130 study design.