Uniqure stock soars over 247% today after breakthrough Huntington’s disease drug results

Qure stock: Shares of UniQure NV skyrocketed to more than 247% on Wednesday after the gene therapy company revealed encouraging topline data from its pivotal Phase I/II study of AMT-130 in patients with Huntington’s disease, as per a report. The results signal a potential breakthrough for a devastating condition that currently has no cure.

UniQure stock surges after Huntington’s disease breakthrough

The study’s high-dose treatment arm met its primary endpoint, showing a statistically significant 75% slowing of disease progression at 36 months on the composite Unified Huntington’s Disease Rating Scale (cUHDRS), compared with a matched external control group, as per an Investing.com report.

UniQure also reported that AMT-130 met a key secondary endpoint, slowing disease progression by 60% on Total Functional Capacity, another widely used clinical measure, according to the report. Favorable trends appeared across additional assessments, including an 88% slowing in the Symbol Digit Modalities Test and a 113% slowing in the Stroop Word Reading Test, as per the Investing.com report. Importantly, cerebrospinal fluid neurofilament light protein levels, a biomarker of neurodegeneration, were 8.2% below baseline at 36 months, as per the Investing.com report.


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The therapy continued to show a manageable safety profile, with no new drug-related serious adverse events reported since December 2022, as per the report. Most side effects were linked to the procedure used to administer the treatment, as per the Investing.com report.

Sarah Tabrizi, professor of clinical neurology and director of the University College London Huntington’s Disease Center, said that, "I am thrilled that this pivotal study of AMT-130 showed statistically significant effects on both cUHDRS and TFC at 36 months, supported by mean CSF NfL remaining below baseline," as quoted in the report.

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Next Steps: When could AMT-130 reach US market?

Looking ahead, uniQure said it intends to file for a Biologics License Application (BLA) in the first quarter of 2026, aiming for a potential US launch later that year, pending FDA approval, as per the Investing.com report. The therapy has already earned Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations from the agency, reflecting its potential to transform the standard of care for Huntington’s disease, as per the Investing.com report.

FAQs

Why did UniQure’s stock jump so much?
Because their gene therapy AMT-130 showed groundbreaking results in slowing Huntington’s disease progression, as per the Investing.com report.

Were there any side effects?
Most side effects were related to the procedure itself, and no new serious drug-related issues have been reported since 2022, as per the Investing.com report.