UniQure Reports Positive Topline Data From Phase I/II Study Of AMT-130 For Huntingtons Disease

uniQure B.V.

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(RTTNews) - Gene therapy company uniQure N.V. (QURE) announced Wednesday 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.

The Company believes that the consistently favorable results in functional, motor and cognitive endpoints at 36 months observed in the high dose group, compared to the variable trends observed in the low dose group, reflect a dose-dependent response to AMT-130.

AMT-130 continued to be generally well-tolerated with a manageable safety profile

AMT-130 has been granted Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA.