Uniqure stock (NASDAQ: QURE) surged during intraday trading Tuesday after the company announced encouraging interim results for AMT-130 in Huntington’s disease trials.
Gene therapy company uniQure has released interim data from its Phase I/II trials of AMT-130, a potential treatment for Huntington’s disease. The data indicates a significant slowing in disease progression and neurofilament light protein (NfL) levels reduction, a neurodegeneration marker, in cerebrospinal fluid (CSF) after 24 months.
The company conducted the trials in the United States and Europe and involved 29 patients who received either a high or low AMT-130 dose, with a control group undergoing imitation surgery. The findings revealed that the high-dose cohort demonstrated an 80% reduction in the rate of disease progression compared to a propensity score-weighted external control group. Although the low-dose group showed a 30% reduction, this result did not reach statistical significance.
Moreover, the treatment resulted in an 11% decrease in CSF NfL levels from baseline, contrasting with the typical 26% increase observed in untreated patients over the same period.
These findings have positioned uniQure to engage in discussions with the FDA regarding potentially expedited clinical development. The company has received the first-ever Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for Huntington’s disease, potentially facilitating a faster review and approval process.
AMT-130 is administered in a one-time procedure and has demonstrated a favorable safety profile, with no new serious adverse events related to the treatment reported. The next steps include completing enrollment for a third cohort to study AMT-130 in conjunction with immunosuppression and presenting further interim analysis in mid-2025.
Huntington’s disease is a hereditary neurodegenerative disorder with no current therapies available to delay or slow its progression. uniQure’s interim findings offer a glimmer of hope to approximately 70,000 individuals affected by the disease in the U.S. and Europe.
Uniqure Stock (NASDAQ: QURE) Movement
QURE stock soared 76.46% on Tuesday, closing at $6.67, marking a 50.06% increase for the week. The trading volume was 51,495,472 shares, significantly higher than the average daily volume of 1.40 million.
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