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Investing.com - H.C. Wainwright has reiterated a Buy rating and $70.00 price target on uniQure BV (NASDAQ:QURE) following the company’s presentation of initial Phase 1/2a data for its Fabry disease gene therapy. The stock, currently trading at $17.95, has shown remarkable momentum with a 231% return over the past year and is approaching its 52-week high of $19.18, according to InvestingPro data.
The data for AMT-191, presented at the International Congress of Inborn Errors of Metabolism in Kyoto, Japan, on September 5, demonstrated robust enzyme activity increases, successful discontinuation of enzyme replacement therapy, and a manageable safety profile.
H.C. Wainwright noted that while uniQure’s lead value driver remains AMT-130 for Huntington’s disease, which is advancing toward a Biologics License Application filing in the first quarter of 2026, the AMT-191 data provides important proof-of-concept for the therapy.
The research firm specifically highlighted AMT-191’s potential as a best-in-class, one-time treatment option for Fabry disease patients who currently have limited alternatives beyond enzyme replacement therapy.
H.C. Wainwright has designated uniQure as a "Top Pick" for the second half of 2025, maintaining its $70 price target for the stock. This aligns with the broader analyst consensus, which remains strongly bullish on the company. Subscribers to InvestingPro can access 12 additional key insights about uniQure’s financial health and market position.
In other recent news, uniQure N.V. reported its second-quarter revenue, which fell short of analyst expectations. The company posted $5.3 million in revenue, slightly below the consensus estimate of $5.4 million, marking a significant drop from $11.1 million in the same period last year. This decline was largely attributed to a $7.1 million decrease in collaboration revenue and a $2.1 million reduction in contract manufacturing of HEMGENIX for CSL Behring. Despite the revenue miss, uniQure’s loss per share was $0.69, better than the anticipated $0.89 loss. Additionally, the company announced positive initial data from its Phase I/IIa trial of AMT-191, an investigational gene therapy for Fabry disease, showing substantial increases in enzyme activity in all patients. In a separate development, Mizuho upgraded uniQure’s stock rating from Neutral to Outperform, raising its price target to $30.00 due to the potential of the company’s Huntington’s disease therapy, AMT-130. Mizuho believes this therapy could become a leading treatment in the Huntington’s disease field, potentially reaching a broader patient population than previously estimated. These updates reflect ongoing developments in uniQure’s clinical pipeline and market outlook.
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