QURE Shares Surge 3.54% as Two-Day Rally Hits 13.34% on AMT-130 Trial Progress and FDA Designations

uniQure (QURE) shares surged 3.54% on Tuesday, extending a two-day rally that lifted the stock by 13.34% in the past two trading sessions. The biotech firm’s share price reached its highest level since October 2025, with an intraday gain of 6.70%, driven by renewed investor confidence in its gene therapy pipeline and regulatory progress.

The recent momentum stems from positive Phase I/II clinical trial results for AMT-130, uniQure’s investigational gene therapy for Huntington’s disease. The trial demonstrated a 75% statistically significant slowing of disease progression in high-dose patients over 36 months, with favorable trends in motor and cognitive function metrics. These findings, combined with a favorable safety profile and no new serious adverse events reported since 2022, have reinforced the therapy’s potential to become the first disease-modifying treatment for HD.

Regulatory tailwinds further bolster the stock’s case. AMT-130 holds Breakthrough Therapy and RMAT designations from the FDA, expediting its path to approval. The company plans to submit a Biologics License Application in early 2026, with a potential U.S. launch later in the year. The FDA’s acceptance of the trial’s external control methodology, which leverages the Enroll-HD dataset, has reduced the need for large-scale Phase III trials, accelerating timelines.

uniQure’s financial fortification has also alleviated near-term risks. A $300 million equity offering and $175 million term loan in September 2025 have strengthened its balance sheet, enabling regulatory submissions and manufacturing scale-up. The capital raise, priced at $475 per share, provides flexibility to navigate the BLA process without diluting existing shareholders. This financial resilience has been cited by analysts as a key factor in the stock’s recent outperformance.

Analyst sentiment has shifted sharply in uniQure’s favor. Guggenheim, RBC Capital, and Cantor Fitzgerald have raised price targets to $95, $55, and $80, respectively, reflecting confidence in AMT-130’s regulatory and commercial potential. Market dynamics also favor the stock, as AMT-130’s one-time dosing model and addressable $2 billion HD market position it as a high-conviction play in a therapeutic area with unmet needs. Strategic experience from its hemophilia B gene therapy approval in 2023 further underlines its regulatory and commercial capabilities.