Key PointsShares of uniQure tumbled 48% to $20.50 during premarket hours Tuesday.The selloff was triggered by newly released 48-month data from the AMT-130 gene therapy trial.The treatment demonstrated a 44% reduction in Huntington’s disease progression at 48 months but failed to achieve statistical significance.The company maintains confidence in its FDA submission, which relies on robust 36-month outcomes from all 15 high-dose trial participants.Earlier this month, uniQure filed a Biologics License Application with the FDA seeking accelerated approval.Shares of uniQure plummeted 48% to $20.50 in premarket action Tuesday following the Dutch biotechnology firm’s announcement of updated clinical results for its experimental Huntington’s disease treatment.uniQure N.V., QUREThe investigational gene therapy, designated AMT-130, aims to decelerate the advancement of this devastating neurological condition by reducing concentrations of a harmful protein in the brain.According to a 48-month assessment of 12 patients receiving the high dose, AMT-130 demonstrated a 44% reduction in disease advancement. However, this outcome failed to reach the predetermined threshold for statistical significance.The disappointing results mark a dramatic reversal from June, when uniQure announced plans to pursue regulatory clearance for the treatment. Investor sentiment was decidedly positive at that time.Breaking Down the Latest Clinical FindingsResearchers at uniQure evaluated trial subjects against an external control cohort. As the study progressed, attrition rates increased within that control population.This created a comparison baseline composed predominantly of individuals whose condition was naturally advancing at a slower rate. According to the company, this compositional shift may have distorted the 48-month findings.The biotech highlighted an alternative 36-month evaluation as a more dependable reference point. This assessment encompassed all 15 participants in the high-dose arm and achieved statistical robustness.An additional 36-month review focusing solely on 12 high-dose subjects revealed an 80% slowdown in disease advancement. This calculation utilized information gathered through June 30, 2026.The company emphasized that participants demonstrated good tolerability across both dosage levels examined. Safety concerns were not identified as contributing factors to Tuesday’s market reaction.Regulatory Pathway and FDA EngagementAt the beginning of this month, uniQure submitted a Biologics License Application to the FDA. The application requests accelerated approval for AMT-130, scientifically known as ifezuntirgene inilparvovec.The regulatory submission was constructed around the 36-month dataset rather than the more recent 48-month analysis. The company noted that FDA officials confirmed during a June consultation that 36-month outcomes from 12 high-dose participants would constitute adequate evidence for the filing.The regulatory agency has previously expressed reservations about the program. In the prior year, the FDA determined that Phase I and II evidence was insufficient to warrant a submission and recommended conducting an additional trial as recently as March.Huntington’s disease is a hereditary condition with universal fatality. Currently, no approved therapies exist that alter the disease trajectory; available interventions only address symptomatic management.uniQure has dedicated nearly ten years to developing AMT-130. The program represents the cornerstone asset in the company’s development portfolio.In Tuesday’s announcement, uniQure emphasized that the update remains clinically significant for patients considering the absence of alternative disease-modifying therapies. Complete study results will be disclosed at an upcoming scientific conference.The sharp decline on Tuesday represents what is expected to be uniQure’s steepest single-session percentage loss since November, based on information from Dow Jones Market Data.The post uniQure (QURE) Stock Crashes 48% on Disappointing Huntington’s Trial Results appeared first on Blockonomi.