uniQure Faces Securities Class Action Over AMT-130 Clinical Trial Design

Hagens Berman Scrutinizing Alleged AMT-130 Clinical Trial Misstatements; April 13 Lead Plaintiff Deadline

Mar. 17, 2026 at 12:05am by Ben Kaplan

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Investor rights law firm Hagens Berman has filed a securities class action lawsuit against uniQure N.V. (NASDAQ: QURE) and certain of its executives, alleging they made inaccurate and misleading statements regarding the regulatory path for its lead gene therapy candidate, AMT-130, specifically concerning the FDA's acceptance of a "pivotal" study design that omitted a traditional placebo control.

Why it matters

The lawsuit alleges that uniQure downplayed the likelihood that, despite purportedly highly successful results from the AMT-130 Pivotal Study, the company would have to delay its Biologics License Application (BLA) timeline to perform additional studies to supplement its BLA submission. This could have significant implications for uniQure's ability to bring AMT-130 to market and generate revenue from the treatment.

The details

The complaint alleges that throughout the Class Period, which runs from September 24, 2025 to October 31, 2025, the defendants misrepresented and failed to disclose that 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. The truth was allegedly revealed on November 3, 2025, when uniQure disclosed that the FDA "currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to [the ENROLL-HD] 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." As a result, uniQure allegedly stated that "the timing of the BLA submission for AMT-130 is now unclear," but that uniQure "plans to urgently interact with the FDA to find a path forward for the timely accelerated approval of AMT-130."

  • The Class Period runs from September 24, 2025 to October 31, 2025.
  • The Lead Plaintiff Deadline in the litigation is April 13, 2026.

The players

uniQure N.V.

A biopharmaceutical company that develops treatments for patients suffering from rare and other devastating diseases. Its leading drug candidate is AMT-130, a novel gene therapy being developed to slow the progression of Huntington's disease.

Hagens Berman

An investor rights law firm that has filed the securities class action lawsuit against uniQure and is investigating the alleged claims that the company violated federal securities laws.

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What they’re saying

“The core of our investigation is the potential delta between what uniQure told investors about its interactions with the FDA and the details revealed in the recent Type A meeting minutes.”

— Reed Kathrein, Hagens Berman partner leading the firm's investigation (PR Newswire)

What’s next

The Lead Plaintiff Deadline in the litigation is April 13, 2026. Hagens Berman is urging investors who purchased QURE securities during the Class Period and suffered significant losses to contact the firm to discuss their rights.

The takeaway

This case highlights the importance of transparency and accurate disclosure from biopharmaceutical companies regarding the regulatory approval process for their drug candidates. Investors will be closely watching how uniQure navigates its interactions with the FDA and the potential impact on the timeline for AMT-130's approval and commercialization.