US Stocks Movement: uniQure Plunges on September 29th Due to Unimpressive 48-Month Data, Primary Endpoint Not Significant, and Safety Concerns

uniQure September 29 Major Drop Explained

Keywords: 48-month data underwhelming, primary endpoint not significant, safety concerns

1. On September 29, the company released four-year data for AMT-130: among 12 high-dose patients, 48-month cUHDRS disease progression slowed by 44%, but the primary endpoint did not reach statistical significance (p=0.144), which is lower than the 36-month data, weakening the certainty of sustained efficacy.

2. On September 29, the updated external control data had significant missing data and survivor bias; although post-hoc analysis restored statistical significance, this result was not a pre-specified primary analysis. Additionally, five patients experienced serious central nervous system inflammation, but all have recovered.

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