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Investing.com - uniQure BV (NASDAQ:QURE) shares are trading higher following positive data from its pivotal Phase 1/2 study of gene therapy AMT-130 in Huntington’s disease, which included three-year follow-up results from all treated patients in the first two cohorts. The stock has demonstrated remarkable momentum, surging over 220% in the past week and approaching its 52-week high of $51.21, according to InvestingPro data.
The high-dose cohort data, which will serve as the primary efficacy analysis for the Biologics License Application (BLA) filing in the first quarter of 2026 under an accelerated approval pathway, showed a statistically significant 75% mean slowing of disease progression in the composite Unified Huntington’s Disease Rating Scale compared to propensity score-matched natural history data.
Additional positive findings included a statistically significant 60% slowing of disease progression as measured by total functional capacity, and mean cerebrospinal fluid neurofilament light protein levels, a biomarker of neurodegeneration, remaining below baseline.
The safety profile was consistent with prior releases, with no new treatment-related serious adverse events observed since December 2022, according to Goldman Sachs, which maintained its Neutral rating and $56.00 price target on the stock.
uniQure plans to engage in a pre-BLA meeting with the FDA in the fourth quarter, with Goldman Sachs modeling potential peak global sales of $2.5 billion with a 90% probability of success, assuming approximately 25% cumulative penetration into the estimated 20,000 diagnosed, symptomatic patients in the US.
In other recent news, uniQure N.V. announced a $200 million public offering of its ordinary shares and pre-funded warrants, with Leerink Partners, Stifel, Van Lanschot Kempen, and Guggenheim Securities acting as bookrunning managers. This development is accompanied by a significant boost in analyst sentiment following the company’s Phase I/II study of AMT-130 for Huntington’s disease. Leerink Partners raised its price target for uniQure to $68 from $48, maintaining an Outperform rating, citing "impressive results" from the study. Similarly, Stifel increased its price target to $65 from $30, highlighting a potential market opportunity exceeding $1 billion in U.S. sales by 2031. TD Cowen reiterated a Buy rating after the study met its primary endpoint, demonstrating a 75% slowing of disease progression. The study’s results showed a statistically significant disease slowing of 75% at 36 months and a 60% slowing in disease progression in secondary measures. These developments underscore the positive momentum for uniQure in advancing its Huntington’s disease treatment.
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