uniQure N.V. (NASDAQ:QURE) stock is trading lower on Tuesday after the company shared mixed long-term data from ongoing Phase 1/2 clinical studies evaluating ifezuntirgene inilparvovec for Huntington’s disease.
36-Month Clinical Results And BLA Pathway
In the 36-month analysis, high-dose patients demonstrated an 80% slowing of disease progression on the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) and a 67% slowing on Total Functional Capacity (TFC).
cUHDRS is a specialized scoring system used to track Huntington’s disease progression.
Mean cerebrospinal neurofilament light protein levels fell 6% below baseline in 14 evaluated high-dose patients.
Following a June 2026 Type B meeting, the U.S. Food and Drug Administration confirmed that 36-month data from 12 high-dose participants would be acceptable as the primary foundation for a Biologics License Application (BLA) under accelerated approval.
uniQure submitted the BLA before generating these latest topline trial results.
48-Month Findings And Dose Analysis
At 48 months, high-dose recipients showed a 61% slowing in TFC progression and a 44% slowing in cUHDRS progression, although the cUHDRS outcome was not statistically significant.
Data missingness in the external control group reached 53% at 48 months.
uniQure noted that faster-progressing control patients dropped out early, likely understating both disease progression in controls and the overall treatment effect.
A post-hoc sensitivity analysis using an earlier control dataset demonstrated statistically significant slowing of 53.5% in cUHDRS and 68.3% in TFC.
Comparative results between high and low doses indicated a clear dose-dependent therapeutic effect across all primary measures.
Safety Profile And Adverse Events
Ifezuntirgene inilparvovec maintained a generally manageable safety profile across both treatment groups. Most reported adverse events related directly to administration procedures, and all fully resolved.
Investigators reported one suicide in a low-dose participant five years post-treatment, assessing it as unrelated to the gene therapy, given elevated suicide rates in the Huntington’s patient population.
QURE Price Action: uniQure shares were down 35.62% at $25.18 at the time of publication on Tuesday, according to Benzinga Pro data.
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