QURE Soars on FDA Alignment for Speedy Approval of Huntington's Drug

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]