Up 152% in December, Is uniQure a Good Stock to Buy Now and Hold Through 2025?

Core Viewpoint - Shares of uniQure have surged significantly due to positive news regarding its Huntington's disease candidate AMT-130 and potential FDA accelerated approval [1][2][3] Company Developments - uniQure's stock increased by 152% in the first 12 days of December, driven by the FDA's willingness to review an application for accelerated approval based on ongoing phase 1/2 clinical trials [2][3] - The FDA's consideration of accelerated approval is significant as Huntington's disease progresses slowly, with trials initiated in 2019 expected to conclude in 2029 [4] FDA Interaction - The FDA has not yet started reviewing the application for AMT-130's accelerated approval, with a meeting scheduled for the first half of 2025 to discuss the path forward [5] - The phase 1/2 trials included a control group receiving a sham procedure, which complicates the interpretation of results as many patients may not show measurable symptom worsening within 12 months [6][8] Market Potential - Huntington's disease affects approximately 70,000 individuals in the U.S. and Europe, presenting a larger market opportunity compared to hemophilia B, for which uniQure's first therapy Hemgenix was approved [11] - AMT-130 has shown promising results, significantly lowering harmful protein fragments and slowing disease progression by 80% compared to external controls [12] Financial Outlook - Sales estimates for AMT-130 suggest potential annual revenues exceeding $1 billion at peak sales, with uniQure holding$435 million in cash as of September [14] - The current market cap of uniQure is $734 million, which could increase significantly if AMT-130 receives FDA approval and achieves substantial sales [15]