uniQure N.V. on Wednesday released topline data from the pivotal Phase 1/2 study of AMT-130 for Huntington’s disease.

Huntington’s disease is an inherited condition in which nerve cells in the brain break down over time. The disease affects a person’s movements, thinking ability and mental health.

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.

Also Read: uniQure’s Investigational Gene Therapy Shows Promising Efficacy With Manageable Safety Risks For Fabry Disease

Data

Topline 36-month efficacy results for patients receiving high-dose AMT-130 (data cutoff as of June 30, 2025):

A statistically significant 75% slowing of disease progression as measured by cUHDRS (p=0.003), which met the primary endpoint of the study.

The mean change in cUHDRS from baseline for treated patients was -0.38, compared to -1.52 for patients in the propensity score-matched external control.

A statistically significant 60% slowing of disease progression as measured by TFC (p=0.033), which met a key secondary endpoint of the study. Treated patients had a mean change in TFC from baseline of -0.36 compared to a change of -0.88 for patients in the propensity score-matched external control.

Favorable trends in other secondary endpoint measures of motor and cognitive function, including Symbol Digit Modalities Test (SDMT), Stroop Word Reading Test (SWRT) and Total Motor Score (TMS).

An 88% slowing of disease progression as measured by SDMT (p=0.057), with a mean change in SDMT from baseline of -0.44 compared to a change of -3.73 for patients in the propensity score-matched external control.

A 113% slowing of disease progression as measured by SWRT (p=0.0021), with a mean change in SWRT from baseline of 0.88 compared to a change of -6.98 for patients in the propensity score-matched external control.

A 59% slowing of disease progression as measured by TMS (p=0.1741), with a mean change in TMS from baseline of 2.01 compared to a change of 4.88 for patients in the propensity score-matched external control.

A mean reduction from baseline in cerebrospinal neurofilament light protein (CSF NfL) of -8.2%. CSF NfL is a well-characterized, supportive biomarker of neurodegeneration.

AMT-130 was generally well-tolerated, with a manageable safety profile at both doses.

The most common adverse events in the treatment groups were related to the administration procedure, which all resolved.

Financing

Concurrently, uniQure raised $175 million via a non-dilutive senior secured term loan facility with Hercules Capital, Inc. ( HTGC ) to enhance the company’s financial flexibility to fund the potential commercial launch of AMT-130.

QURE Price Action: uniQure shares were up 189.82% at $39.59 at the time of publication on Wednesday. The stock is trading at a new 52-week high, according to Benzinga Pro data.

Read Next:

Trump’s ‘Gold Card’ Visa Price Slashed By 80%, Expert Calls It ‘Admission Of Failure’