AMT-130 Approval Will Transform Huntington's Treatment Pipeline

uniQure's analyst price targets have surged, with fair value estimates nearly doubling from around $35 to $66 per share. Analysts point to robust, statistically significant AMT-130 data in Huntington's disease, which supports higher revenue growth expectations and dramatically improved profitability assumptions.

Analyst Commentary

Recent Street research reflects a generally optimistic outlook for uniQure following updated, positive AMT-130 data in Huntington's disease. Analysts have revised price targets sharply upward and identified key drivers for potential future growth, while also highlighting areas to watch as the company advances its lead program. Below is a summary of prevailing bullish and bearish viewpoints from the latest coverage.

• Bullish analysts substantially increased their probability of success for AMT-130, noting the robust, statistically significant, and dose-dependent slowing of disease progression. This could support potential approval and rapid uptake in both the U.S. and Europe.
• The newly reported topline Phase 1/2 data are widely seen as a game-changer, with clear evidence that AMT-130 meaningfully impacts Huntington's progression across several functional and cognitive measures. This reduces regulatory risk and bolsters the outlook for accelerated approval.
• Updates revealed consistently favorable safety and efficacy data across multiple endpoints and geographies, establishing AMT-130 as a leading candidate and strengthening prospects for a biologics license application submission in early 2026.
• Strong cash reserves and an extended operational runway are seen as enabling uniQure to advance AMT-130 development and commercialization plans without near-term financing concerns.

• Bearish analysts caution that there may be some patients and prescribers who adopt a wait-and-see stance until AMT-130 receives formal approval. This presents uncertainty around near-term uptake and revenue generation.
• Some note that the absence of recent data releases can create short-term volatility and investor anxiety, a dynamic observed in temporary share price pullbacks.
• Ongoing discussions regarding study methodology and external control comparisons, such as changes in propensity score measurement, may introduce perceived regulatory complexities that merit monitoring as the program moves forward.
• Potential for competitive pressure, highlighted by comparisons to other clinical programs in Huntington's disease, suggests that consistent, compelling data will remain essential for uniQure to sustain its leading position.

What's in the News

• uniQure reports positive topline results from its pivotal Phase I/II study of AMT-130 in Huntington's disease. The high-dose AMT-130 demonstrated statistically significant slowing of disease progression (Key Developments).
• The company successfully completed a follow-on equity offering and raised approximately $300 million through ordinary shares and pre-funded warrants (Key Developments).
• Ongoing lock-up agreements will prevent members of uniQure's board and executive officers from selling or transferring certain shares, restricted stock, performance stock, and options during the period between September 25 and November 25, 2025 (Key Developments).
• uniQure continues to expand its pivotal clinical program for AMT-130, conducting multiple multi-center, dose-escalating trials to explore the therapy's safety and efficacy in Huntington's disease (Key Developments).

Valuation Changes

• Consensus Analyst Price Target (Fair Value) has risen significantly, nearly doubling from $34.94 to $66.50 per share.
• Discount Rate has declined modestly, falling from 7.12% to 6.62%.
• Revenue Growth expectations have increased, moving from 190.4% to 212.2%.
• Net Profit Margin outlook has expanded substantially, improving from 5.8% to 20.0%.
• Future P/E ratio forecast has decreased sharply, dropping from 141.06x to 61.90x. This suggests a more favorable earnings profile relative to price.