uniQure stock price target doubled to $60 by Mizuho on Huntington's therapy data

uniQure (NASDAQ:QURE) has received significant analyst upgrades, including Mizuho doubling its price target to $60 and Leerink Partners raising theirs to $68, following better-than-expected three-year registrational data for its AMT-130 gene therapy in Huntington’s disease. The Phase 1/2 trial achieved statistical significance on both primary and key secondary endpoints, suggesting a path to full regulatory approval rather than accelerated, which has driven a 220%+ stock surge in the past week. Capitalizing on this positive momentum and market confidence, uniQure has launched a $200 million public offering of ordinary shares to fund its pipeline, with a Biologics License Application for AMT-130 anticipated by early 2026.

Analysis

UniQure (QURE) has experienced a significant re-rating following the release of better-than-expected three-year registrational data for its Huntington's disease gene therapy, AMT-130. The stock surged over 220% in the past week, driven by the Phase 1/2 trial achieving statistical significance on its primary endpoint (cUHDRS) and, crucially, also on the key secondary endpoint for Total Functional Capacity (TFC). This unexpected success on the TFC endpoint shifts the outlook from a potential accelerated approval to a more direct path for full regulatory approval, a scenario described by a key opinion leader as "game-changing." This clinical de-risking has prompted a wave of analyst upgrades, with Mizuho doubling its price target to $60, Leerink Partners raising its target to $68, and Stifel increasing its target to $65 while projecting an 80% probability of approval and over $1 billion in U.S. sales by 2031. Capitalizing on this momentum, uniQure has launched a $200 million public offering to fund operations ahead of its planned Biologics License Application (BLA) submission in early 2026.

Key Decisions for Investors

• Consider the recent trial data a major de-risking event for uniQure's lead asset, as the unexpected success on the TFC secondary endpoint materially increases the probability of a full regulatory approval and supports the bullish analyst consensus projecting blockbuster sales potential.
• Monitor the pricing and market absorption of the recently announced $200 million public offering, as the resulting share dilution, following a rapid 220% price surge, may create short-term volatility and a potentially more favorable entry point.
• Recognize that the primary value-inflection catalyst remains the Biologics License Application submission anticipated in early 2026 and subsequent FDA review; the investment thesis requires a long-term horizon and tolerance for regulatory risk leading up to this milestone.