UniQure (QURE) came out with a quarterly loss of $1.50 per share versus the Zacks Consensus Estimate of a loss of $0.44. This compares to loss of $1.53 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -240.91%. A quarter ago, it was expected that this human gene therapy company would post a loss of $1.12 per share when it actually produced a loss of $0.91, delivering a surprise of 18.75%.Over the last four quarters, the company ...

Financial Performance - The company reported net losses for the years ended December 31, 2024 and 2023, with significant losses in previous years and expects to incur losses in the future[16] - The company reported net losses for the years ended December 31, 2024 and 2023, and anticipates continued losses in the future, indicating a risk of never achieving profitability[16] - The company has incurred significant losses in previous years and will need to raise additional funding to advance product development, which may not be available on acceptable terms[18] Product Development and Clinical Trials - The lead product candidate, AMT-130, is critical for the treatment of Huntington's disease, and any failure in its clinical development could adversely affect the company's business[15] - The company may face delays in clinical trials and challenges in demonstrating the safety and efficacy of its product candidates, which could impact future operations[15] - The company may face delays in clinical trials and challenges in demonstrating safety and efficacy of its product candidates, which could materially harm its business[15] - The company has encountered risks associated with the marketing approval process for gene therapies, which may affect the commercialization of its product candidates[15] Regulatory and Manufacturing Risks - The company has encountered risks related to the manufacturing of gene therapies, which are complex and expensive, potentially leading to delays in development or commercialization[15] - The company is dependent on third-party manufacturers for its product candidates, which may not perform satisfactorily or comply with regulatory requirements[15] - The company relies on third parties for manufacturing its product candidates, which may not perform satisfactorily or comply with regulations[15] - The company plans to leverage specialized regulatory pathways, such as the FDA's accelerated approval pathway, but may struggle to maintain the benefits associated with these designations[15] - The company may leverage specialized regulatory pathways for its product candidates, but there is no guarantee of obtaining or maintaining the associated benefits[15] Market and Competitive Landscape - The market price of the company's ordinary shares has been volatile and may fluctuate substantially in the future[18] - The company faces substantial competition in the industry, which may impact its ability to successfully commercialize its products[18] - The company faces substantial competition and risks that competitors may develop or commercialize competing products more successfully[18] Business Development and Strategic Initiatives - The company’s business development initiatives, including acquisitions and partnerships, may not achieve their intended benefits, posing additional risks[15] - The Lexington Transaction may not yield expected benefits and could introduce additional risks related to manufacturing and supply requirements[15]

~ Announced alignment with the U.S. Food and Drug Administration (FDA) on key elements of the Accelerated Approval pathway for AMT-130 in Huntington's disease; Initiated preparations for a potential Biologics License Application (BLA) submission ~ ~ Completed patient enrollment in the third cohort of the Phase I/II study of AMT-130 ~ Exhibit 99.1 uniQure Announces 2024 Financial Results and Highlights Recent Company Progress ~ Initiated dosing of the Phase I/II study of AMT-260 in mesial temporal lobe epile ...

Core Insights - uniQure has made significant clinical and operational progress in 2024, particularly with AMT-130 for Huntington's disease, including alignment with the FDA on the Accelerated Approval pathway [2][5] - The company has completed patient enrollment in various Phase I/II studies, including AMT-130, AMT-260, AMT-191, and AMT-162, and has received favorable recommendations from Independent Data Monitoring Committees [1][2] - Financially, uniQure reported a cash position of approximately $367.5 million as of December 31, 2024, with net proceeds from a recent financing expected to fund operations into the second half of 2027 [1][7] Clinical Developments - The FDA granted RMAT designation for AMT-130, indicating its potential to address unmet medical needs in Huntington's disease [2] - The company has initiated preparations for a Biologics License Application (BLA) submission for AMT-130, with the FDA agreeing that ongoing Phase I/II study data may serve as the primary basis for this submission [2][5] - Enrollment in the Phase I/II studies of AMT-191 for Fabry disease and AMT-162 for SOD1-ALS is progressing, with the second cohorts expected to begin dosing soon [1][2] Financial Performance - Revenue for the year ended December 31, 2024, was $27.1 million, an increase from $15.8 million in 2023, driven by higher license and collaboration revenues [8][20] - Research and development expenses decreased to $143.8 million in 2024 from $214.9 million in 2023, reflecting cost-cutting measures [10] - Selling, general, and administrative expenses also decreased to $52.7 million in 2024 from $74.6 million in 2023, indicating improved operational efficiency [11] Strategic Actions - The company completed the sale of its Lexington manufacturing facility and underwent a restructuring that reduced its workforce by approximately 65%, significantly lowering cash burn by about $70 million per year [2][7] - A public offering of 5.1 million ordinary shares at $17.00 per share was completed, providing additional funding for operations [7][8] - Upcoming investor events are scheduled, including participation in healthcare conferences in March and April 2025 [4][6]

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 - Shares of uniQure have surged significantly due to positive news regarding its Huntington's disease candidate AMT-130 and potential FDA accelerated approval [1][2][3] Company Developments - uniQure's stock increased by 152% in the first 12 days of December, driven by the FDA's willingness to review an application for accelerated approval based on ongoing phase 1/2 clinical trials [2][3] - The FDA's consideration of accelerated approval is significant as Huntington's disease progresses slowly, with trials initiated in 2019 expected to conclude in 2029 [4] FDA Interaction - The FDA has not yet started reviewing the application for AMT-130's accelerated approval, with a meeting scheduled for the first half of 2025 to discuss the path forward [5] - The phase 1/2 trials included a control group receiving a sham procedure, which complicates the interpretation of results as many patients may not show measurable symptom worsening within 12 months [6][8] Market Potential - Huntington's disease affects approximately 70,000 individuals in the U.S. and Europe, presenting a larger market opportunity compared to hemophilia B, for which uniQure's first therapy Hemgenix was approved [11] - AMT-130 has shown promising results, significantly lowering harmful protein fragments and slowing disease progression by 80% compared to external controls [12] Financial Outlook - Sales estimates for AMT-130 suggest potential annual revenues exceeding $1 billion at peak sales, with uniQure holding $435 million in cash as of September [14] - The current market cap of uniQure is $734 million, which could increase significantly if AMT-130 receives FDA approval and achieves substantial sales [15]

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]