TLDR
- uniQure (QURE) stock surged 150% after announcing positive 36-month results for AMT-130 Huntington’s disease treatment
- The therapy showed 75% disease slowing on primary endpoint with statistical significance (p=0.003)
- Treatment met key secondary endpoint with 60% slowing on Total Functional Capacity measure
- Stifel reiterated Buy rating with $30 price target following the strong data release
- Company plans to submit FDA application in Q1 2026 for potential U.S. launch later that year
uniQure stock exploded higher Wednesday morning, climbing 150% after the gene therapy company released three-year data for its experimental Huntington’s disease treatment. The results exceeded Wall Street expectations and sent shares soaring.
The company’s AMT-130 therapy met its primary goal in the pivotal Phase I/II study. The treatment showed a 75% disease slowing effect at 36 months when measured against external controls.
This result carried statistical weight with a p-value of 0.003. The measurement used the composite Unified Huntington’s Disease Rating Scale, a standard assessment tool for the progressive brain disorder.
AMT-130 also hit a key secondary target. The therapy demonstrated 60% slowing of disease progression when measured by Total Functional Capacity.
This secondary endpoint surprised analysts. Stifel noted the TFC results could help with FDA approval considerations down the road.
The treatment showed promise across other measures too. AMT-130 delivered an 88% slowing in the Symbol Digit Modalities Test and 113% slowing in the Stroop Word Reading Test.
Biomarker data supported the clinical findings. Cerebrospinal fluid neurofilament light protein levels stayed 8.2% below baseline at the 36-month mark. These proteins indicate brain cell damage when elevated.
Safety Profile Remains Manageable
The company reported no new drug-related serious side effects since December 2022. Most adverse events related to how the treatment gets administered rather than the therapy itself.
AMT-130 uses gene therapy to deliver instructions directly to brain cells. The one-time treatment aims to reduce production of the toxic huntingtin protein that causes Huntington’s disease.
Dr. Sarah Tabrizi from University College London praised the results. She leads the Huntington’s Disease Center there and called the statistical effects on both primary and secondary measures “thrilling.”
Regulatory Timeline Takes Shape
uniQure plans to file its FDA application in the first quarter of 2026. The Biologics License Application would seek approval for commercial use in the United States.
If approved, the company expects to launch AMT-130 later in 2026. The treatment already holds Breakthrough Therapy status and Regenerative Medicine Advanced Therapy designation from regulators.
Wall Street analysts remained bullish after the data release. Stifel kept its Buy rating and $30 price target on the stock, which closed Tuesday at $13.66.
The firm believes these results strengthen uniQure’s case for fast-track FDA approval. However, analysts noted some study limitations including small patient numbers and reliance on external control comparisons.
uniQure recently secured $175 million in financing from Hercules Capital. This loan facility provides runway for the potential commercial launch of AMT-130 while refinancing existing debt with better terms extending to October 2030.
