uniQure N.V. (NASDAQ: QURE) shares more than doubled Wednesday. The Lexington, Mass.-based company a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced positive topline data from the pivotal Phase I/II study of AMT-130 for the treatment of Huntington’s disease.
The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) at 36 months compared to a propensity score-matched external control. The study also met a key secondary endpoint by achieving statistically significant slowing of disease progression as measured by Total Functional Capacity (TFC) at 36 months compared to a propensity score-matched external control.
“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,” stated Sarah Tabrizi, M.D., FRCP, FRS, FMedSci, Ph.D., professor of clinical neurology, director of the University College London Huntington’s Disease Center and joint head of the department of neurodegenerative disease. “I believe these groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in
Huntington’s disease, where an urgent need persists. These data indicate that AMT-130 has the potential to meaningfully slow disease progression – offering long-awaited hope to individuals and families impacted by this devastating disease.”
QURE shares began Wednesday higher by $26.26, or 192.2%, to $39.92.
