uniQure Announces Favorable Recommendation from Independent Data Monitoring Committee for its Phase I/II EPISOD1 Clinical Trial of AMT-162 for the Treatment of SOD1-ALS

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]