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Luca Issi from RBC Capital set a price target of $11 for UniQure (NASDAQ:QURE), indicating a potential upside of approximately 7.32%.The FDA's decision on insufficient trial data for marketing application has significantly impacted UniQure's stock price.Despite recent challenges, UniQure's market capitalization of approximately $646.84 million and a trading volume of 16,764,186 shares reflect ongoing investor interest.UniQure (NASDAQ:QURE) is a biotechnology company focused on developing gene therapies for ...

Core Viewpoint - Rosen Law Firm is reminding investors who purchased ordinary shares of uniQure N.V. during the specified class period of the upcoming lead plaintiff deadline for a class action lawsuit [1]. Group 1: Class Action Details - Investors who bought uniQure shares between September 24, 2025, and October 31, 2025, may be eligible for compensation without any out-of-pocket fees through a contingency fee arrangement [2]. - A class action lawsuit has already been filed, and interested parties must move the Court to serve as lead plaintiff by April 13, 2026 [3]. - The Rosen Law Firm emphasizes the importance of selecting qualified legal counsel with a successful track record in securities class actions [4]. Group 2: Case Allegations - The lawsuit alleges that uniQure misrepresented and/or failed to disclose critical information regarding the design of its Pivotal Study for a drug candidate targeting Huntington's Disease, including the lack of full FDA approval [5]. - It is claimed that uniQure downplayed the likelihood of needing to delay its Biologics License Application (BLA) timeline due to the necessity of additional studies [5]. - The lawsuit asserts that the defendants' statements regarding uniQure's business and prospects lacked a reasonable basis, leading to investor damages when the true information became public [5].

Core Viewpoint - uniQure BV is a biotechnology company focused on gene therapies for genetic disorders, facing a competitive landscape and recent downgrade by RBC Capital from "Outperform" to "Sector Perform" [1][5] Financial Performance - The company reported a quarterly loss of $0.56 per share, which was better than the Zacks Consensus Estimate of a $0.93 loss, resulting in an earnings surprise of approximately 39.91% [2][5] - Compared to the previous year's loss of $1.50 per share, this represents a notable improvement [2] Revenue Performance - uniQure reported revenues of $5.57 million for the quarter ending December 2025, missing the Zacks Consensus Estimate by 21.2% [3][5] - This revenue figure shows a slight increase from $5.22 million reported a year ago, but the company has consistently failed to meet consensus revenue estimates over the past four quarters [3] Stock Performance - The current stock price for QURE is $10.55, reflecting a significant decrease of 32.50% from its previous high [4] - The stock has fluctuated between $8.96 and $10.58 today, with a 52-week high of $71.50 and a low of $7.76 [4] - The company's market capitalization is approximately $649.9 million, with a trading volume of 13,733,434 shares on the NASDAQ exchange [4]

Core Viewpoint - A securities fraud lawsuit has been filed against uniQure N.V. following significant declines in its stock price due to insufficient clinical data for its AMT-130 gene therapy for Huntington's disease [1][2]. Group 1: Stock Performance - Shares of uniQure fell over 35% on March 2, 2026, after the U.S. FDA indicated that the clinical data for AMT-130 was inadequate for a marketing application [2]. - The decline on March 2 followed a previous drop of up to 66% on November 3, 2025, when the company disclosed prior FDA feedback regarding its clinical data [2]. Group 2: Legal Action - A securities class action has been filed in the U.S. District Court for the Southern District of New York, alleging that uniQure and certain executives misled investors about the clinical results and regulatory prospects from September 24, 2025, to October 31, 2025 [2]. - Investors who purchased uniQure common stock during this period and experienced losses may be eligible to participate in the lawsuit [3]. Group 3: Next Steps for Investors - The deadline to seek appointment as lead plaintiff in the class action is April 13, 2026, and a class has not yet been certified [4]. - Investors are encouraged to contact Block & Leviton for more information on how to proceed if they have lost money on their investment [4]. Group 4: Whistleblower Information - Individuals with non-public information about uniQure N.V. are encouraged to assist in the investigation or file a report with the SEC under the whistleblower program, potentially receiving rewards of up to 30% of any successful recovery [5]. Group 5: About Block & Leviton - Block & Leviton is recognized as a leading securities class action firm, having recovered billions for defrauded investors and representing many top institutional investors [6].

Core Viewpoint - A class action lawsuit has been filed against uniQure N.V. for securities fraud, alleging material misstatements regarding its Huntington's disease gene therapy drug, AMT-130, during the class period from September 24, 2025, to October 31, 2025 [1]. Summary by Relevant Sections Lawsuit Details - The lawsuit was filed by Kessler Topaz Meltzer & Check, LLP on behalf of investors who purchased uniQure ordinary shares during the specified class period [1]. - The deadline for investors to seek lead plaintiff status is April 13, 2026 [1]. Allegations - The key allegations include misleading statements about the Phase I/II clinical trials of AMT-130 and the timeline for the Biologics License Application (BLA) submission to the FDA [1]. - Specific misrepresentations include the lack of full FDA approval for the study design and the downplaying of the likelihood of delays in the BLA timeline due to additional studies required [1]. Impact on Share Price - On November 3, 2025, uniQure's share price dropped by $33.40, or over 49%, from $67.69 to $34.29, following revelations about the inadequacy of the data for BLA submission [1]. Investor Actions - Investors are encouraged to file for lead plaintiff status by the deadline or contact KTMC for a free case evaluation [1]. - The lead plaintiff will represent the class in directing the litigation and selecting counsel [1].

UniQure Stock Drops as Bad News From FDA Gets Even Worse - Barron'sSkip to Main ContentThis copy is for your personal, non-commercial use only. Distribution and use of this material are governed by our Subscriber Agreement and by copyright law. For non-personal use or to order multiple copies, please contact Dow Jones Reprints at 1-800-843-0008 or visit www.djreprints.com.# UniQure Stock Drops 36% After Earnings. Bad News From the FDA Gets Even Worse.By [Nate Wolf]ShareResize---ReprintsIn this article[QURE] ...

Summary of the Conference Call Company and Industry - **Company**: uniQure - **Industry**: Biotechnology, specifically focusing on gene therapies for neurodegenerative diseases such as Huntington's disease and epilepsy Key Points and Arguments Huntington's Disease and AMT-130 - **Phase 3 Data**: The pivotal Phase 1/2 trial for AMT-130 showed a 75% slowing of disease progression at three years, measured by the composite Unified Huntington's Disease Rating Scale (UHDRS) [10] - **Total Functional Capacity**: A 60% decline in disease progression was observed, which is significant for FDA approval [10] - **Neurofilament Light**: A decrease from baseline was noted, indicating reduced neurodegeneration, contrasting with the expected annual increase of 10%-15% [10] - **Patient Cohorts**: Data was pooled from 17 patients, with 12 having over three years of follow-up [3] - **FDA Interactions**: The FDA granted RMAT designation in early 2024 based on two-year data showing signals of slowing progression [16] - **BLA Submission**: The FDA expressed concerns about the Phase 1/2 study being hypothesis-generating and not pre-specifying statistical analyses, which affected the potential for BLA submission [28] - **Next Steps**: A Type B meeting with the FDA is planned to discuss Phase 3 study design and the use of external controls [53] Safety Profile - **Safety of AMT-130**: The procedure is reported to be remarkably safe, with no significant adverse events attributed to AMT-130 since December 2022 [72] - **Neuroinflammation**: Potential neuroinflammation is manageable with immune suppression, which will now be administered perioperatively [73] Regulatory Landscape - **International Engagement**: uniQure is engaging with regulators outside the U.S. for potential expedited reviews and expanded access pathways [75][78] - **Patient Advocacy**: Strong support from patient advocacy groups has been noted, with significant community involvement in lobbying for the therapy [86] Other Programs - **AMT-260 (Temporal Lobe Epilepsy)**: Early data showed a 92% reduction in seizure frequency in a case study, with ongoing trials expected to provide more data soon [87] - **AMT-191 (Fabry Disease)**: The program is in a Phase I/II study with 11 patients off enzyme replacement therapy, showing promising results [100][102] Financial Position and Partnerships - **Funding Strategy**: The company has a runway into the second half of 2029 and is disciplined in investment decisions, focusing on data that supports further investments [104][107] - **Partnerships**: Future partnerships will depend on data from existing programs and the Phase III strategy for Huntington's disease [107] Additional Important Information - **Ethical Considerations**: The company is considering the ethical implications of sham-controlled studies in slow-progressing diseases like Huntington's [61] - **Long-term Data**: The company plans to update the statistical analysis plan to include four-year assessments, which will provide further insights into treatment durability [66]

Summary of uniQure FY Conference Call - March 02, 2026 Company Overview - **Company**: uniQure (NasdaqGS:QURE) - **Focus**: Gene therapies for neurodegenerative diseases, particularly Huntington's disease and epilepsy Key Points on Huntington's Disease Program (AMT-130) - **Phase 3 Data**: The pivotal phase 1/2 trial showed a 75% slowing of disease progression at three years, measured by the composite Unified Huntington's Disease Rating Scale (UHDRS) [9] - **Total Functional Capacity**: A 60% decline in disease progression was observed, which is significant for FDA approval [9] - **Neurofilament Light**: A decrease from baseline was noted, contrasting with the expected 10%-15% annual increase in neurodegeneration markers [9] - **FDA Interactions**: The FDA granted RMAT designation in early 2024 based on initial data, but later expressed concerns about the Phase 1/2 study being hypothesis-generating and not suitable for BLA submission [15][20] - **Next Steps**: A Type B meeting with the FDA is planned to discuss phase 3 study design and the use of external controls [44][46] - **Patient Community Support**: Strong advocacy from patient groups has been noted, with significant engagement in policy discussions and petitions to the FDA [82][84] Key Points on AMT-260 (Temporal Lobe Epilepsy Program) - **Initial Data**: A case study showed a 92% reduction in seizure frequency in the first patient treated, with no significant adverse events reported [84] - **Current Status**: Six patients have been dosed in the first cohort, with data expected in the second quarter of 2026 [85] - **Future Goals**: Aiming for a 50% or higher reduction in seizures to support progression to pivotal trials [96] Key Points on AMT-191 (Fabry Disease Program) - **Study Progress**: A Phase I/II study is ongoing with 11 patients treated, all of whom are off enzyme replacement therapy [99] - **Outcomes**: Dose-dependent increases in the deficient enzyme have been observed, with ongoing monitoring for long-term outcomes [99] Financial and Strategic Considerations - **Funding Position**: The company has a strong balance sheet with a runway into the second half of 2029, allowing for continued investment in promising programs [104] - **Partnership Strategy**: The company remains open to partnerships but emphasizes the need for data to support investment decisions [105] Safety Profile - **AMT-130 Administration**: The procedure is reported to be safe and well-tolerated, with most adverse events being procedure-related rather than drug-related [64] Regulatory Landscape - **International Engagement**: The company is engaging with regulators outside the U.S. for potential expedited reviews and expanded access pathways [71][74] Conclusion - **Commitment to Patients**: The company is focused on advancing its therapies while ensuring ethical considerations in study designs, particularly for slow-progressing diseases like Huntington's [46][54]

uniQure N.V. (QURE) shares tumbled 36.58 percent to $9.91, down $5.72 on Monday, after the company reported a full-year net loss and disclosed FDA feedback indicating that current Phase 1/2 data for AMT-130 in Huntington's disease are insufficient to support a marketing application.The stock is currently trading at $9.91, compared with a previous close of $15.63. It opened at $9.19 and has traded between $8.96 and $10.28 during the session on the Nasdaq. Trading volume stands at 8.53 million shares, well a ...