Core Insights - The Independent Data Monitoring Committee (IDMC) has recommended proceeding with the enrollment of the second cohort in the Phase I/IIa trial of AMT-191 after reviewing safety data from the first cohort [1][2] - The company expects to initiate enrollment of the second dose cohort in the first quarter of 2025 [1] Company Overview - uniQure N.V. is a leading gene therapy company focused on developing transformative therapies for patients with severe medical needs [1][6] - The company has a pipeline of proprietary gene therapies targeting various severe diseases, including Huntington's disease, refractory temporal lobe epilepsy, ALS, and Fabry disease [6] Clinical Trial Details - The Phase I/IIa clinical trial of AMT-191 is a multi-center, open-label study in the U.S. involving two dosing cohorts of up to six adult male patients each [3] - Patients will receive intravenous infusions of AMT-191 while continuing their regular enzyme replacement therapy for a period of 24 months [3] - The trial aims to explore safety, tolerability, and early signs of efficacy by measuring the expression of the lysosomal enzyme α-galactosidase A (aGLA-A) [3] Disease Context - Fabry disease is an X-linked genetic disorder caused by a deficiency of the GLA enzyme, leading to lipid accumulation in various cell types, including kidney and heart cells [2][5] - The prevalence of Fabry disease is estimated to be between one in 40,000 and one in 117,000 individuals [5] - Current standard care involves bi-weekly infusions of enzyme replacement therapy, which has limited effectiveness due to poor cross-correction [5] Regulatory Status - AMT-191 has received Orphan Drug status and Fast Track designation from the U.S. Food and Drug Administration [4]

Core Insights - The Independent Data Monitoring Committee (IDMC) has recommended proceeding with dose escalation for AMT-162 after reviewing 28-day safety data from the first cohort of a Phase I/II clinical trial for SOD1-ALS [1][2] - Enrollment for the second dose cohort is expected to begin in the first quarter of 2025 [1][2] Company Overview - uniQure N.V. is a gene therapy company focused on developing transformative therapies for severe medical conditions, including SOD1-ALS [1][6] - The company has previously achieved significant milestones, including the approval of gene therapy for hemophilia B, and is advancing a pipeline for various severe diseases [6] Clinical Trial Details - The EPISOD1 trial is a multi-center, open-label study involving three dose-escalating cohorts, each with up to four patients, assessing the safety and tolerability of AMT-162 [3] - The trial will measure neurofilament light chain and SOD1 protein as exploratory efficacy indicators [3] Treatment Mechanism - AMT-162 is an AAVrh10-based gene therapy designed to reduce the expression of the mutated SOD1 protein, which is toxic to motor neurons [2] - The therapy aims to provide a one-time, intrathecal administration approach to potentially slow or halt the progression of SOD1-ALS [2] Disease Context - SOD1-ALS is a rare and fatal neurodegenerative disease affecting motor neurons, with an estimated 170,000 individuals diagnosed globally, of which 2% have SOD1 mutations [5] - The average life expectancy for ALS patients is three to five years from symptom onset [5] Regulatory Status - AMT-162 has received Orphan Drug status and Fast Track designation from the U.S. Food and Drug Administration [4]

Offering Details - uniQure announced the pricing of its underwritten public offering of 4,411,764 ordinary shares at $17.00 per share, with expected gross proceeds of approximately $75 million before deductions [1] - The company granted underwriters a 30-day option to purchase up to 661,764 additional ordinary shares at the public offering price, less underwriting discounts and commissions [1] - The offering is expected to close on or about January 10, 2025, subject to customary closing conditions [1] Underwriters and Prospectus - Leerink Partners, Stifel, and Guggenheim Securities are acting as the bookrunning managers, while Chardan and H.C. Wainwright & Co. are the lead managers for the offering [2] - The securities are offered pursuant to uniQure's automatically effective shelf registration statement filed with the SEC on January 7, 2025 [3] - A preliminary prospectus supplement and accompanying prospectus were filed with the SEC, and the final versions will be filed and made available through the underwriters [3] Company Overview - uniQure is a leading gene therapy company focused on developing transformative therapies for severe medical needs [5] - The company has achieved a historic milestone with the approval of its gene therapy for hemophilia B, representing a major advancement in genomic medicine [5] - uniQure is advancing a pipeline of proprietary gene therapies for Huntington's disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe diseases [5]

Core Viewpoint - uniQure N.V. has initiated an underwritten public offering of its ordinary shares and pre-funded warrants, with the potential for underwriters to purchase an additional 15% of shares at the public offering price [1][2]. Company Overview - uniQure is a leading gene therapy company focused on developing transformative therapies for patients with severe medical needs, including a gene therapy for hemophilia B, which marks a significant milestone in genomic medicine [5]. Offering Details - The offering is subject to market conditions, and there is no assurance regarding its completion or the actual size and terms [1]. - The securities are being offered under an automatically effective shelf registration statement filed with the SEC on January 7, 2025, with a preliminary prospectus supplement to be filed [3]. Underwriter Information - Leerink Partners is acting as the bookrunning manager for the proposed offering [2].

Core Viewpoint - uniQure N.V. (QURE) experienced a significant stock surge of 109.7% following the announcement of alignment with the FDA on an accelerated approval pathway for AMT-130, a gene therapy aimed at treating Huntington's disease [1] Company Developments - The FDA's Regenerative Medicine Advanced Therapy (RMAT) designation has been granted to AMT-130, which is designed to treat Huntington's disease [2] - An RMAT meeting in November 2024 confirmed that existing data from early to mid-stage studies of AMT-130 can serve as the primary basis for a regulatory filing, eliminating the need for an additional pre-submission study [3] - The FDA has accepted the use of cUHDRS as an intermediate clinical endpoint and reductions in neurofilament light (NfL) in cerebrospinal fluid (CSF) as supportive evidence for therapeutic benefit [4] - uniQure is preparing to submit a biologics license application to the FDA for AMT-130 and plans to engage in further discussions regarding statistical analysis and technical requirements in the first half of 2025 [5] Clinical Data and Efficacy - Updated interim data from July 2024 indicated a potential long-term clinical benefit and reduction of neurodegeneration markers in patients treated with AMT-130 [6][7] - Two phase I/II studies are being conducted, with 29 patients treated and a total of 21 patients having 24-month follow-up data available [8] - The treatment has shown a manageable safety profile, with no new drug-related serious adverse events reported [9] Future Plans and Pipeline - uniQure aims to complete patient enrollment in the third cohort of the U.S. phase I/II study soon, with safety data expected in the first half of 2025 [10] - An interim analysis comparing treated patients to external controls is planned for mid-2025 [11] - The company has additional candidates in early-stage development for conditions such as refractory temporal lobe epilepsy, amyotrophic lateral sclerosis, and Fabry disease [12] Market Position - uniQure markets Hemgenix, a gene therapy for hemophilia B, which received approvals in the U.S. and EU in 2022 and 2023, respectively [13] - The company currently holds a Zacks Rank 2 (Buy), indicating a favorable market position [14]

Core Viewpoint - Shares of uniQure (QURE) have increased by 5.2% recently, closing at $6.02, with a potential upside indicated by Wall Street analysts' price targets suggesting a mean estimate of $19.75, representing a 228.1% upside [1] Price Targets - The average of 12 short-term price targets ranges from a low of $7 to a high of $28, with a standard deviation of $8.05, indicating variability among estimates [2] - The lowest estimate suggests a 16.3% increase from the current price, while the highest estimate indicates a 365.1% upside [2] - A low standard deviation signifies greater agreement among analysts regarding price movement [2][7] Analyst Sentiment - Analysts show increasing optimism about QURE's earnings prospects, as evidenced by a strong consensus in revising EPS estimates higher, which correlates with potential stock price increases [4][9] - Over the past 30 days, five estimates have been revised upward, resulting in a 7.1% increase in the Zacks Consensus Estimate for the current year [10] Zacks Rank - QURE holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, indicating strong potential for upside [11] Conclusion on Price Targets - While consensus price targets may not reliably indicate the extent of potential gains, they can provide a directional guide for price movement [12]

uniQure (QURE) could be a solid choice for investors given its recent upgrade to a Zacks Rank #2 (Buy). This upgrade primarily reflects an upward trend in earnings estimates, which is one of the most powerful forces impacting stock prices.The sole determinant of the Zacks rating is a company's changing earnings picture. The Zacks Consensus Estimate -- the consensus of EPS estimates from the sell-side analysts covering the stock -- for the current and following years is tracked by the system.Since a changing ...

uniQure (QURE) closed the last trading session at $7.43, gaining 37.1% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $20 indicates a 169.2% upside potential.The average comprises 12 short-term price targets ranging from a low of $7 to a high of $28, with a standard deviation of $7.82. While the lowest estimate indicates a decline of 5.8% from the current price level, the most opt ...

UniQure (QURE) came out with a quarterly loss of $0.91 per share versus the Zacks Consensus Estimate of a loss of $1.12. This compares to loss of $1.88 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of 18.75%. A quarter ago, it was expected that this human gene therapy company would post a loss of $1.24 per share when it actually produced a loss of $1.16, delivering a surprise of 6.45%.Over the last four quarters, the company ha ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from _______ to _______ Commission file number: 001-36294 uniQure N.V. (Exact name of Registrant as specified in its charter) The Netherlands ...