Buy Rating for uniQure Driven by Promising AMT-130 Advancements and Strategic FDA Alignment

William Blair analyst Sami Corwin has maintained their bullish stance on QURE stock, giving a Buy rating today.

Sami Corwin has given his Buy rating due to a combination of factors related to uniQure’s promising advancements in their AMT-130 program for Huntington’s disease. The company has successfully aligned with the FDA on key components necessary for their Biologics License Application (BLA) submission, which is anticipated in early 2026. This alignment includes agreement on the use of the composite Unified Huntington’s Disease Rating Scale (cUHDRS) as an intermediate clinical endpoint, which is crucial for accelerated approval.
Additionally, the primary efficacy analysis will focus on three-year data from patients receiving a high dose of AMT-130, with results expected in the third quarter of 2025. UniQure’s strategic use of the ENROLL-HD dataset as an external control for their primary analysis, due to its larger sample size and lower attrition rate, further strengthens their position. These developments, along with the potential for full approval and discussions on post-marketing requirements, underpin Corwin’s positive outlook on the stock.

In another report released today, H.C. Wainwright also reiterated a Buy rating on the stock with a $70.00 price target.