uniQure N.V. (NASDAQ:QURE) on Friday announced updated preliminary safety and exploratory efficacy data from 11 patients in its Phase 1/2a trial of AMT-191.The updated data was presented at the WORLDSymposium in San Diego, California.Trial DataAMT-191 is an investigational AAV gene therapy under development for Fabry disease, a rare X-linked lysosomal disorder that results in excessive deposition of lipids in the tissues.As of the January 8, 2026, study data cutoff date, all 11 patients in the three dose co ...

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]

Apis Capital Advisors’ Apis Flagship Fund released its Q4 2025 investor letter. A copy of the letter can be downloaded here. Apis Flagship Fund delivered strong performance in the fourth quarter of 2025, with the Fund up 10.0% net for the quarter and gaining 55.1% for the full year, significantly outperforming the MSCI ACWI global benchmark by approximately 7.0% in Q4 and 33.0% for the year. Long positions drove the majority of gains as they generated 11.9% gross contribution, while shorts added a further 0 ...

Apis Capital Advisors’ Apis Flagship Fund released its Q4 2025 investor letter. A copy of the letter can be downloaded here. Apis Flagship Fund delivered strong performance in the fourth quarter of 2025, with the Fund up 10.0% net for the quarter and gaining 55.1% for the full year, significantly outperforming the MSCI ACWI global benchmark by approximately 7.0% in Q4 and 33.0% for the year. Long positions drove the majority of gains as they generated 11.9% gross contribution, while shorts added a further 0 ...

Group 1 - Biotech stocks experienced significant gains in after-hours trading, with several companies showing strong percentage increases despite no new company-specific news [1][2][3][4] - Coherus Oncology, Inc. (CHRS) rose by 9.09% to close at $1.73, adding $0.14 in extended trading [1] - Sensei Biotherapeutics, Inc. (SNSE) surged 10.06% to finish at $12.47, gaining $1.14 [1] - Cue Biopharma, Inc. (CUE) advanced 9.37% to $0.40, increasing by $0.034 [1] - Alpha Tau Medical Ltd. (DRTS) increased by 3.35% to $7.41, up $0.24, with recent focus on new leadership [2] - Cingulate Inc. (CING) edged up 1.49% to close at $5.45, gaining $0.08 [3] - uniQure N.V. (QURE) posted a notable 9.96% jump to $24.95, adding $2.26 in after-hours trading [3] - IO Biotech, Inc. (IOBT) gained 4.19% to $0.50, up $0.020, while exploring strategic alternatives including mergers and asset sales [4]

Bragar Eagel & Squire, P.C. Litigation Partner Brandon Walker Encourages Investors Who Suffered Losses In uniQure (QURE) To Contact Him Directly To Discuss Their Options If you purchased or acquired stock in uniQure and would like to discuss your legal rights, call Bragar Eagel & Squire partner Brandon Walker or Marion Passmore directly at (212) 355-4648. Click here to participate in the action. NEW YORK, Jan. 22, 2026 (GLOBE NEWSWIRE) -- What’s Happening: Bragar Eagel & Squire, P.C., a nationally recogniz ...

Core Insights - The Alger Small Cap Focus Fund's fourth-quarter 2025 investor letter highlights a strong performance in the US equity market, with the S&P 500 increasing by 2.7% due to better-than-expected corporate earnings and a supportive macroeconomic environment [1] - The letter notes a divergence in market performance, particularly regarding AI investments, which face skepticism due to various challenges [1] - The Fund's Class A shares outperformed the Russell 2000 Growth Index, with contributions from the Utilities and Financials sectors, while Consumer Discretionary and Information Technology sectors negatively impacted performance [1] Company Insights - UniQure N.V. (NASDAQ:QURE) is a biotechnology company focused on developing treatments for rare diseases, specifically AMT-130 for Huntington's disease, which currently lacks approved disease-modifying treatments [2][3] - The stock of UniQure N.V. experienced a one-month return of -8.57% but gained 62.56% over the past 52 weeks, closing at $22.84 with a market capitalization of $1.42 billion on November 18, 2025 [2] - The company's shares were volatile in 2025, initially surging after promising Phase 1/2 data but later facing regulatory uncertainty due to FDA feedback regarding the adequacy of external-control data for accelerated approval [3]

Core Viewpoint - Kessler Topaz Meltzer & Check, LLP is investigating potential violations of federal securities laws on behalf of investors in uniQure N.V. following significant stock price decline due to FDA feedback on its gene therapy AMT-130 [1][4]. Group 1: Company Developments - On November 3, 2025, uniQure announced that the FDA indicated the data for its investigational gene therapy AMT-130 did not provide sufficient evidence to support its Biologics License Application (BLA) [2]. - The FDA no longer agrees that data from the Phase I/II studies of AMT-130 may be adequate for BLA submission, leading to uncertainty regarding the timing of the BLA submission [2]. Group 2: Market Reaction - Following the FDA's announcement, uniQure's stock price plummeted over 50%, dropping from $67.69 on October 31, 2025, to $34.29 on November 3, 2025 [3].

Group 1 - UniQure (NASDAQ:QURE) is considered a promising stock to buy under $50, with recent price target adjustments from analysts [1][2] - Stifel lowered its price target for UniQure to $40 from $50 while maintaining a Buy rating, following a comprehensive review of the biotech sector [1] - Mizuho reduced its price target for UniQure to $33 from $60, maintaining an Outperform rating, due to regulatory concerns regarding the Phase I/II studies for AMT-130 [2][3] Group 2 - The FDA indicated that current data for AMT-130 likely lacks the primary evidence required for a Biologics License Application (BLA), leading to a reduced probability of success and a delayed market launch by one year [2][3] - Despite these challenges, there is optimism about AMT-130's long-term potential, with a follow-up Type A meeting with the FDA scheduled for Q1 2026 to establish a new path forward [3]

Core Viewpoint - uniQure N.V. is advancing its investigational gene therapy AMT-130 for Huntington's disease and has scheduled a Type A meeting with the FDA to discuss the Biologics License Application (BLA) data package for accelerated approval [1][2] Company Overview - uniQure is a leading gene therapy company focused on delivering transformative therapies for patients with severe medical needs, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [3] - The company has achieved a significant milestone with the approval of its gene therapy for hemophilia B, marking a historic achievement in genomic medicine [3] Regulatory Engagement - The CEO of uniQure expressed optimism about the upcoming discussion with the FDA, highlighting the urgent need for disease-modifying therapies in the Huntington's disease community [2] - The company plans to provide a regulatory update following the receipt of official meeting minutes from the FDA [2]