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Investing.com - RBC Capital has lowered its price target on uniQure BV (NASDAQ:QURE) to $45.00 from $65.00 while maintaining an Outperform rating following a regulatory setback for the company’s Huntington’s disease treatment. The news triggered a steep 51% decline in uniQure’s share price over the past week, though the stock remains up over 500% from a year ago, according to InvestingPro data.
The FDA has changed its position on uniQure’s Huntington’s disease therapy, indicating that the company’s three-year data may not be adequate for approval despite showing statistical significance (p=0.003) compared to external controls in a pre-specified analysis.
According to RBC Capital, the FDA is now arguing that the trial was hypothesis-generating and the analysis was not pre-specified from the beginning, while also citing other limitations including no clear dose response in the data.
RBC Capital suggests recent FDA leadership changes may have contributed to this regulatory shift, noting that Nicole Verdun, whom they believe supported accelerated approval, left the agency shortly after a Type B meeting where the statistical analysis plan was discussed.
The investment firm has reduced its probability of success for the therapy from 75% to 50% but maintains its Outperform rating, noting that there are currently no disease-modifying therapies for Huntington’s disease and expecting potential responses from patient advocacy groups and the scientific community.
In other recent news, uniQure has faced a significant regulatory setback concerning its Huntington’s disease treatment, AMT-130. The U.S. Food and Drug Administration (FDA) has indicated that data from the company’s Phase 1/2 studies may no longer be sufficient to support a Biologics License Application, reversing previous communications. In response to this development, H.C. Wainwright has lowered its price target for uniQure from $110 to $70 while maintaining a Buy rating. Despite this challenge, uniQure has successfully completed a public offering, raising approximately $345 million to support its gene therapy pipeline. This offering included 6,736,841 ordinary shares sold at $47.50 each, along with pre-funded warrants to purchase additional shares. Prior to the completion of this offering, the company had announced a pricing of $300 million for the underwritten public offering of its shares. The recent financial moves reflect uniQure’s continued efforts to advance its gene therapy initiatives despite regulatory hurdles.
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