uniQure N.V. (NASDAQ: QURE) Long Term Investor Alert: Investigation of potential Wrongdoing

An investigation on behalf of current long-term investors in shares of uniQure N.V. (NASDAQ: QURE) concerning potential breaches of fiduciary duties by certain directors and officers of uniQure N.V. was announced.

Investors who are current long term investors in uniQure N.V. (NASDAQ: QURE) shares, have certain options and should contact the Shareholders Foundation at mail@shareholdersfoundation.com or call +1(858) 779 - 1554.

The investigation by a law firm for current long term investors in NASDAQ: QURE stocks follows a lawsuit filed against uniQure N.V. over alleged securities laws violations. The investigation on behalf of current long term investors in NASDAQ: QURE stocks, concerns whether certain uniQure N.V. directors are liable in connection with the allegations made in that lawsuit.

Amsterdam, Netherlands based uniQure N.V. develops treatments for patients suffering from rare and other devastating diseases in the United States. uniQure N.V. (NASDAQ: QURE) reported that its annual Total Revenue rose from $15.84 million in 2023 to $27.11 million in 2024, and that its Net Loss increased from $308.47 million in 2023 to $239.55 million in 2024.

On September 24, 2025, uniQure N.V. 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, Defendant Kapusta touted the study results and asserted that "we believe these findings provide compelling and clinically meaningful evidence of AMT-130 disease modifying potential."
Additionally, Walid Abi-Saab, 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.

Capitalizing on the substantial increase in the value of uniQure ordinary shares, the Company publicly offered more than 5.7 million uniQure ordinary shares, and more than 500,000 pre-funded warrants to purchase ordinary shares, over the next several days after the release of the Pivotal Study results (the "September 2025 Offering"). Despite the fact that AMT-130's future remained uncertain pending uniQure's discussion of the Pivotal Study results with the FDA, in the prospectus supplement to the September 2025 Offering, uniQure explained that it was engaging in the September 2025 Offering in order to "fund our commercialization readiness activities" and "the potential commercial launch of AMT-130 and related commercialization activities." Through the September 2025 Offering, uniQure generated approximately $345 million in proceeds (before expenses).

On November 3, 2025, uniQure revealed 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." Although the Company "plan[ned] to urgently interact with the FDA to find a path forward for the timely accelerated approval of AMT-130," uniQure admitted that "the timing of the BLA submission for AMT-130 is now unclear."

Shares of uniQure N.V. (NASDAQ: QURE) declined from $71.50 per share on October 28, 2025, to as low as $19.29 per share on December 15, 2025.

According to that complaint filed in the U.S. District Court for the Southern District of New York the plaintiff alleges that, the Defendants misrepresented and/or 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, that the 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 that as a result, Defendants' statements about the Company's business, operations, and prospects lacked a reasonable basis.

Those who purchased shares of uniQure N.V. (NASDAQ: QURE) have certain options and should contact the Shareholders Foundation.

Contact:
Michael Daniels
Shareholders Foundation, Inc.
3111 Camino Del Rio North
Suite 423
San Diego, CA 92108
Tel: +1-(858)-779-1554
E-Mail: mail@shareholdersfoundation.com

About Shareholders Foundation, Inc.
The Shareholders Foundation, Inc. is a professional portfolio monitoring and settlement claim filing service, and an investor advocacy group, which does research related to shareholder issues and informs investors of securities lawsuits, settlements, judgments, and other legal related news to the stock/financial market. Shareholders Foundation, Inc. is in contact with a large number of shareholders and offers help, support, and assistance for every shareholder. The Shareholders Foundation, Inc. is not a law firm. Referenced cases, investigations, and/or settlements are not filed/initiated/reached and/or are not related to Shareholders Foundation. The information is provided as a public service. It is not intended as legal advice and should not be relied upon.

This release was published on openPR.