Core Viewpoint - Kessler Topaz Meltzer & Check, LLP has filed a securities fraud class action lawsuit against uniQure N.V. on behalf of investors who purchased shares during a specified class period, alleging that the company made materially false and misleading statements regarding its drug AMT-130 and its regulatory approval process [1][11]. Company Overview - uniQure N.V. is a biotechnology company focused on developing gene therapies for rare diseases, including Huntington's disease (HD) [3]. - The company's leading drug candidate, AMT-130, aims to slow the progression of HD, a fatal genetic disorder with no existing cure [4]. Clinical Trials and FDA Interaction - uniQure completed patient enrollment for two ongoing Phase I/II clinical trials for AMT-130, known as the Pivotal Study, in March 2022 [4]. - The FDA had previously agreed that the Pivotal Study would not include a placebo comparator, allowing results to be compared to an external historical dataset, ENROLL-HD, for potential BLA submission [5]. Investor Communication and Stock Performance - On September 24, 2025, uniQure announced topline results from the Pivotal Study, leading to a significant increase in share price from $13.66 to $47.50, a nearly 250% rise [8]. - By October 29, 2025, shares were trading above $70.00, prompting the company to offer over 5.7 million shares to capitalize on the increased valuation, generating approximately $345 million [9]. Disclosure of Regulatory Challenges - On November 3, 2025, uniQure disclosed that the FDA no longer agreed that the data from the Pivotal Study would be adequate for BLA submission, leading to a sharp decline in share price by over 49% [10]. Allegations in the Lawsuit - The lawsuit alleges that uniQure misrepresented the approval status of the Pivotal Study design and downplayed the likelihood of delays in the BLA timeline, resulting in misleading statements about the company's business and prospects [11].

Core Viewpoint - A class action lawsuit has been filed against uniQure N.V. on behalf of investors who acquired its securities during the specified class period, alleging misrepresentation and failure to disclose critical information regarding the company's drug development process [1][3]. Company Overview - uniQure N.V. is a biotechnology company based in Amsterdam, focusing on gene therapies for severe diseases, including Huntington's disease [2]. Legal Allegations - The lawsuit claims that uniQure misrepresented the approval status of its Pivotal Study for the drug AMT-130 and downplayed the likelihood of delays in its Biologics License Application (BLA) timeline due to the need for additional studies [3]. Stock Impact - Following the disclosure that the FDA did not agree with the use of Phase I/II data for BLA submission, uniQure's stock price plummeted over 49%, dropping from $67.69 on October 31, 2025, to $34.29 on November 3, 2025 [4].

Core Viewpoint - Faruqi & Faruqi, LLP is investigating potential claims against uniQure N.V. (NASDAQ: QURE) and reminds investors of the April 13, 2026 deadline to seek the role of lead plaintiff in a federal securities class action filed against the company [1] Summary by Relevant Sections Company Overview - uniQure N.V. is facing allegations of violating federal securities laws by making false or misleading statements regarding its Pivotal Study and the approval status of its BLA submission for AMT-130 [1] Legal Proceedings - The complaint alleges that the design of uniQure's Pivotal Study was not fully approved by the FDA, and that the company downplayed the likelihood of needing to delay its BLA timeline for additional studies [1] - On November 3, 2025, uniQure disclosed that the FDA no longer agreed that data from the Phase I/II AMT-130 studies would support a BLA submission, leading to a significant drop in share price [1] Financial Impact - Following the disclosure, uniQure's share price fell by $33.40, or over 49%, from $67.69 on October 31, 2025, to $34.29 on November 3, 2025 [1]

Core Viewpoint - Rosen Law Firm has initiated a class action lawsuit on behalf of shareholders of uniQure N.V. (NASDAQ: QURE) due to allegations of misleading investors regarding the company's business operations and the approval status of its pivotal study for a drug candidate targeting Huntington's Disease [1] Group 1: Allegations Against uniQure N.V. - The lawsuit claims that uniQure misrepresented the approval status of its pivotal study by the U.S. Food and Drug Administration (FDA) [1] - It is alleged that the company downplayed the likelihood of needing to delay its Biologics License Application (BLA) timeline to conduct additional studies [1] - The lawsuit asserts that the statements made by uniQure regarding its business, operations, and prospects lacked a reasonable basis, leading to investor damages when the truth was revealed [1] Group 2: Class Action Participation - Shareholders who wish to serve as lead plaintiffs must file their motions by April 13, 2026 [1] - Participation in the class action is not mandatory for recovery; shareholders can remain absent class members if they choose [1] - All legal representation is provided on a contingency fee basis, meaning shareholders incur no fees or expenses unless a recovery is achieved [1]

Group 1 - A shareholder class action lawsuit has been filed against uniQure N.V. alleging false and misleading statements regarding its business and operations [1] - The lawsuit claims that the design of uniQure's Pivotal Study was not fully approved by the FDA and that the company downplayed the likelihood of delays in its Biologics License Application timeline [1] - The lawsuit specifically addresses the comparison of Pivotal Study results to the ENROLL-HD external historical data set [1] Group 2 - Shareholders who purchased uniQure shares between September 24, 2025, and October 31, 2025, and experienced significant losses are encouraged to discuss their legal rights [2] - The deadline to request to be appointed lead plaintiff in the case is April 13, 2026 [3] - Holzer & Holzer, LLC is a law firm specializing in shareholder class action and derivative litigation, having recovered hundreds of millions of dollars for shareholders [3]

Core Viewpoint - The article discusses a securities fraud class action lawsuit against uniQure N.V. due to a delay in FDA approval for its drug candidate AMT-130, which led to a significant stock price decline of 49% [1] Summary by Relevant Sections Lawsuit Details - The lawsuit alleges that uniQure and its executives failed to disclose material information during the class period from September 24, 2025, to October 31, 2025, violating federal securities laws [1] - The company had previously indicated a high likelihood of receiving accelerated FDA approval for AMT-130 after a planned Biologics License Application (BLA) submission in Q1 2026 [1] - On November 3, 2025, uniQure announced that the FDA no longer agreed that the data from Phase I/II studies were adequate for BLA submission, resulting in an unclear timeline for the submission [1] Stock Price Impact - Following the FDA announcement, uniQure's stock price fell from $67.69 per share on October 31, 2025, to $34.29 per share on November 3, 2025, a decline of $33.40 per share or over 49% [1] Legal Actions - Investors who suffered substantial losses have until April 13, 2026, to file lead plaintiff applications in the class action lawsuit [1] - The case is identified as Scocco v. uniQure N.V., et al., Case No. 1:26-cv-01124 [1]

Group 1 - A class action lawsuit has been filed against uniQure N.V. on behalf of investors who purchased shares between September 24, 2025, and October 31, 2025 [1] - The lawsuit alleges that uniQure misled investors regarding the likelihood of FDA approval for its leading drug candidate, AMT-130, by failing to disclose that the pivotal study design was not fully approved by the FDA [1] - Following the announcement on November 3, 2025, regarding FDA feedback, uniQure's share price dropped by $33.40, or over 49%, from $67.69 to $34.29 [1] Group 2 - Robbins LLP is also investigating a class action lawsuit against POMDoctor Ltd. for investors who acquired securities between October 9, 2025, and December 11, 2025 [1] - Another class action has been filed against Inovio Pharmaceuticals, Inc. for investors who purchased securities between October 10, 2023, and December 26, 2025 [1] - A class action lawsuit has been initiated against Masonite International Corporation for sellers of common stock between June 5, 2023, and February 8, 2024 [1]

Core Viewpoint - Kessler Topaz Meltzer & Check, LLP has filed a securities fraud class action lawsuit against uniQure N.V. on behalf of investors who purchased shares between September 24, 2025, and October 31, 2025, alleging that the company made materially false and misleading statements regarding its drug AMT-130 and its regulatory approval process [1][2]. Group 1: Lawsuit Details - The lawsuit is titled Scocco v. uniQure N.V., filed in the U.S. District Court for the Southern District of New York [1]. - Investors can move to serve as lead plaintiff by April 13, 2026, and the firm encourages those affected to contact them for more information [2]. Group 2: Company Background - uniQure N.V. is a biotechnology company focused on developing gene therapies for rare diseases, particularly Huntington's disease (HD) [1]. - The leading drug candidate, AMT-130, aims to slow the progression of HD, a fatal genetic disorder with no current cure [1]. Group 3: Clinical Trials and FDA Interaction - uniQure completed patient enrollment for two ongoing Phase I/II clinical trials for AMT-130 in March 2022 [1]. - The FDA had previously agreed that the Pivotal Study results could be compared to an external historical dataset (Enroll-HD) for the basis of a Biologics License Application (BLA) submission [1]. Group 4: Stock Performance and Offering - Following the announcement of positive topline results from the Pivotal Study on September 24, 2025, uniQure's stock price surged nearly 250%, from $13.66 to $47.50 per share [1]. - The company subsequently offered over 5.7 million shares and generated approximately $345 million in proceeds, despite uncertainties regarding AMT-130's future [2]. Group 5: Allegations of Misrepresentation - The complaint alleges that uniQure misrepresented the FDA's approval of the Pivotal Study design and downplayed the likelihood of delays in the BLA timeline [2]. - On November 3, 2025, uniQure disclosed that the FDA no longer agreed that the Pivotal Study data would suffice for a BLA submission, leading to a stock price drop of over 49% [2].

Core Insights - uniQure N.V. announced updated preliminary safety and exploratory efficacy data from its Phase 1/2a trial of AMT-191 for Fabry disease, presented at the WORLDSymposium in San Diego, California [1] Trial Data - AMT-191 is an investigational AAV gene therapy for Fabry disease, a rare X-linked lysosomal disorder characterized by excessive lipid deposition in tissues [2] - As of January 8, 2026, all 11 patients in three dose cohorts (6×10^13 gc/kg, 4×10^13 gc/kg, and 2×10^13 gc/kg) showed elevated α-galactosidase A (α-Gal A) activity, which is crucial as deficiency leads to GB3 buildup and Fabry disease [2] Efficacy Observations - Dose-dependent increases in α-Gal A activity were noted, ranging from 0.34- to 82.2-fold at the lowest dose, 1.6- to 312.52-fold at the mid dose, and 27.7- to 223.7-fold at the highest dose [3] - These increases were durable, with follow-up periods ranging from over a year in the high-dose cohort to four months in the mid-dose cohort [3] Patient Outcomes - Six out of 11 patients were withdrawn from enzyme replacement therapy (ERT) after meeting criteria including elevated α-Gal A activity [4] Safety Profile - Stable plasma lyso-Gb3 levels were maintained across all dose cohorts, regardless of ERT status, indicating a manageable safety profile for AMT-191 [5] - No Serious Adverse Events (SAEs) related to AMT-191 were observed at the 4×10^13 gc/kg and 2×10^13 gc/kg doses, although two patients at the 4×10^13 gc/kg dose experienced asymptomatic Grade 3 liver enzyme elevations [5] Dose-Limiting Toxicity - The two liver enzyme elevation cases were confirmed as dose-limiting toxicity, leading to a pause in additional dosing in the mid- and high-dose cohorts pending further evaluation [6] - Both patients responded to corticosteroid therapy and are under follow-up [6] Additional Observations - At the 6×10^13 gc/kg dose, five previously reported SAEs occurred, including two unrelated (stroke, diplopia) and two related (chest pain, increased troponin) to AMT-191, along with one possibly related SAE (leptomeningeal enhancement) [7] - One patient at this dose experienced an asymptomatic Grade 3 liver enzyme elevation that resolved with corticosteroid therapy [7] Market Reaction - Following the announcement, uniQure shares increased by 6.10% to $26.01 [8]

Core Insights - The article discusses updated preliminary safety and exploratory efficacy data from uniQure's Phase I/IIa trial of AMT-191, a gene therapy for Fabry disease, presented at the WORLDSymposium [1][7] Group 1: Efficacy Data - All 11 patients in the trial exhibited elevated α-galactosidase A (α-Gal A) activity across three dosing cohorts [2] - Dose-dependent increases in α-Gal A activity were observed, ranging from 0.34- to 82.2-fold at the lowest dose, 1.6- to 312.52-fold at the mid dose, and 27.7- to 223.7-fold at the highest dose, with durability noted over follow-up periods [3] - Six out of 11 patients discontinued enzyme replacement therapy after meeting pre-specified criteria, including elevated α-Gal A activity, while stable plasma lyso-Gb3 levels were maintained post-dose across all cohorts [4] Group 2: Safety Profile - The safety profile of AMT-191 was manageable, with no serious adverse events (SAEs) related to the therapy observed at the lower doses [5] - Two patients at the mid dose experienced asymptomatic Grade 3 liver enzyme elevations, confirmed as dose-limiting toxicity, leading to a pause in additional dosing in mid- and high-dose cohorts [5][6] - At the highest dose, five SAEs were reported, including two unrelated to AMT-191, and one patient experienced an asymptomatic Grade 3 liver enzyme elevation that resolved with corticosteroid therapy [6] Group 3: Clinical Trial Overview - The Phase I/IIa trial is a multi-center, open-label study in the U.S. with three dosing cohorts, exploring safety, tolerability, and early efficacy signs [8] - Patients were not excluded based on pre-existing neutralizing antibodies to AAV5 and will be followed for 24 months [8] - AMT-191 has received Orphan Drug and Fast Track designations from the U.S. FDA, indicating its potential significance in treating Fabry disease [9] Group 4: Background on Fabry Disease - Fabry disease is an X-linked genetic lysosomal storage disorder caused by α-Gal A deficiency, leading to toxic accumulation of lyso-Gb3, which can damage various organs [10] - The current standard treatment involves bi-weekly enzyme replacement therapy, which has limited effectiveness due to poor cross-correction and substrate clearance [10] Group 5: Company Overview - uniQure is focused on advancing gene therapies for severe diseases, with a history of significant achievements in the field, including a gene therapy for hemophilia B [11] - The company is developing a pipeline of gene therapies for various conditions, including Fabry disease, and aims to deliver potentially curative treatments [11]