Securities Fraud Class Action Filed Against uniQure Over Huntington's Disease Gene Therapy

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

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.

uniQure is a biotechnology company developing gene therapies for rare diseases, including Huntington's disease. uniQure is incorporated in The Netherlands with its principal executive offices in Amsterdam, The Netherlands. The Company's leading drug candidate is AMT-130, a novel gene therapy being developed to slow the progression of HD, 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 HD 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 HD.

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 HD (the "Pivotal Study").

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 HD. 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. Notably, 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 HD 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.

The complaint alleges that, throughout the Class Period, Defendants made materially false and/or misleading statements, as well as failed to disclose material adverse facts, about uniQure's business and operations. Specifically, Defendants misrepresented and/or failed to disclose that: (1) the design of uniQure's Pivotal Study—including comparison of the Pivotal Study results to the ENROLL-HD external historical data set—was not fully approved by the FDA; (2) Defendants downplayed the likelihood that, despite purportedly highly successful results from the Pivotal Study, uniQure would have to delay its BLA timeline to perform additional studies to supplement its BLA submission; and (3) as a result, Defendants' statements about uniQure's business, operations, and prospects lacked a reasonable basis.