UniQure (QURE) Stock Drop Looms as FDA Concern Over Huntington's Data Spurs Mizuho Downgrade and Delayed Launch

UniQure (NASDAQ:QURE) is one of the stocks that will double in 2026. On December 9, Mizuho analyst Uy Ear lowered the firm’s price target on uniQure to $33 from $60, while keeping an Outperform rating on the shares. The firm adjusted its financial model for AMT-130 after the company announced that the FDA communicated concerns that the current data submitted from the Phase I/II studies for Huntington’s disease are presently unlikely to serve as the primary evidence needed to support a BLA. Consequently, Mizuho reduced its view of AMT-130’s probability of success and delayed its expected launch by 1 year.

In other news, earlier in its Q3 2025 earnings report, UniQure detailed promising clinical data for its lead gene therapy candidate, AMT-130, alongside new regulatory challenges. The company reported $3.7 million in revenue, an increase from $2.3 million in Q3 2024. However, financial expenses rose, with R&D expenses reaching $34.4 million (up from $30.6 million in Q3 2024) and SG&A expenses totaling $19.4 million (up from $11.6 million in Q3 2024). The increase in R&D was primarily driven by costs associated with preparing BLA for AMT-130, while the rise in SG&A was due to increased employee-related expenses and professional fees.

UniQure also announced positive top-line data from its pivotal Phase I/II study of AMT-130 for Huntington’s disease, showing a statistically significant slowing of disease progression over 3 years. Specifically, the high dose demonstrated a 75% slowing of disease progression, meeting the study’s primary endpoint. The therapy also showed a 60% slowing of disease progression in the key secondary endpoint of total functional capacity at 3 years.

UniQure (NASDAQ:QURE) develops treatments for patients suffering from rare and other devastating diseases in the US.

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Disclosure: None. This article is originally published at Insider Monkey.