uniQure Faces Securities Fraud Class Action After FDA Setback and 49% Stock Plunge

A securities fraud class action lawsuit has been filed against uniQure N.V. (NASDAQ: QURE) on behalf of investors who purchased or acquired the company's ordinary shares between September 24, 2025 and October 31, 2025. The action, captioned Scocco v. uniQure N.V., et al., Case No. 1:26-cv-01124, is pending in the United States District Court for the Southern District of New York.

uniQure is a biotechnology company incorporated in The Netherlands with principal executive offices in Amsterdam, developing gene therapies for rare diseases, including Huntington's disease. The company's leading drug candidate is AMT-130, a novel gene therapy being developed to slow the progression of Huntington's disease, a usually fatal, inherited genetic disorder that causes nerve cells in the brain to break down, leading to problems with movement and thinking, as well as psychiatric issues. There is no existing cure or approved means for slowing the progression of the disease. Some drugs can address certain Huntington's disease symptoms, but do not halt its progression to a usually fatal outcome. AMT-130 is one of a very few drugs in testing intended to slow the progression of Huntington's disease.

In March 2022, uniQure completed patient enrollment for two, ongoing multi-center, dose-escalating Phase I/II clinical trials for AMT-130 called the Pivotal Phase I/II Study of AMT-130 in patients with Huntington's disease.

The lawsuit alleges that uniQure and certain of its executives are charged with failing to disclose material information during the Class Period, violating federal securities laws. The complaint alleges that uniQure failed to disclose that the design of its Pivotal Study for its leading drug candidate AMT-130 was not fully approved by the FDA, and that the company downplayed the likelihood it would have to delay its Biologics License Application (BLA) submission to perform additional studies.

According to the defendants, the U.S. Food and Drug Administration previously agreed that uniQure's Pivotal Study would not include any placebo comparator, but instead, the Pivotal Study results could be compared to an external historical data set, known as Enroll-HD or ENROLL-HD, and the analysis derived from such comparison potentially could serve as the basis for uniQure's Biologics License Application submission to the FDA for approval to use AMT-130 to treat patients with Huntington's disease.

The company's chief executive officer assured investors of the company's alignment with the FDA during calls with investors on June 2, 2025, and July 29, 2025.

The Class Period begins on September 24, 2025, when the company announced topline results of the Pivotal Study. The company emphasized that AMT-130 saw a "mean reduction from baseline in cerebrospinal neurofilament light protein" (CSF NfL)—which uniQure asserted was "a well-characterized, supportive biomarker of neurodegeneration." Accordingly, uniQure explained that "[e]levation in CSF NfL has been shown to be strongly associated with greater clinical severity of [HD]." Thus, based on the totality of the results and as compared to data from ENROLL-HD, investors were led to believe that AMT-130 was effective in slowing the neurodegeneration in patients with Huntington's disease and that uniQure would file for accelerated approval of a BLA for AMT-130 in the near-term.

During the related investor conference held that same day, the chief executive officer touted the study results and asserted that "we believe these findings provide compelling and clinically meaningful evidence of AMT-130 disease modifying potential." Additionally, the company's chief medical officer reminded investors that uniQure previously discussed the trial design with the FDA and that the FDA agreed that "cUHDRS could serve as an acceptable registrational, intermediate clinical endpoint for accelerated approval." Moreover, he stated that "[t]he FDA also agreed that ENROLL-HD . . . may be acceptable as the external control dataset for the primary analysis, with each dose matched the corresponding controls based on their baseline characteristics."

Thus, investors were led to believe that there was a high likelihood that AMT-130 would receive accelerated approval from the FDA after the company's planned BLA submission in the first quarter of 2026. The market acted accordingly and, in response to defendants' statements, the price of the company's ordinary shares jumped from a close of $13.66 per share on September 23, 2025, to close at $47.50 per share on September 24, 2025, a nearly 250% increase. By October 29, 2025, uniQure ordinary shares were trading above $70.00 per share.

During the Class Period, the company represented to investors that there was a high likelihood that its leading drug candidate, AMT-130, would receive accelerated approval from the U.S. Food and Drug Administration after the company's planned Biologics License Application submission in the first quarter of 2026.

However, on November 3, 2025, the company disclosed that "the FDA currently no longer agrees that the data from the Phase I/II studies of AMT-130 in comparison to an external control, as per the prespecified protocols and statistical analysis plans shared with the FDA in advance of the analyses, may be adequate to provide the primary evidence in support of a BLA submission" and as a result, "the timing of the BLA submission for AMT-130 is now unclear."

On this news, the price of uniQure's shares plummeted $33.40 per share, or more than 49%, from a close of $67.69 per share on October 31, 2025, to close at $34.29 per share on November 3, 2025.

Investors who purchased or otherwise acquired uniQure ordinary shares during the Class Period may, no later than April 13, 2026, move the Court to serve as lead plaintiff for the class. Shareholders who wish to serve as lead plaintiff for the class must submit their papers to the court by April 13, 2026.