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Magnite (NASDAQ:MGNI) Shares Gap Up - Here's What Happened
The 12-patient study showed a 61% slowing at three years, but analysts said updated control data may understate AMT-130's effect.
On Tuesday, UniQure reported its gene therapy AMT-130 slowed Huntington's disease progression by 44% at four years, though shares plummeted 40% to trade just below $24 following the disclosure.
While the therapy showed a 44% slowdown, the result lacked statistical significance; UniQure noted that 53% of 48-month reference data was missing, likely understating the treatment effect.
Analysts at Stifel and Leerink Partners noted three-year data from 15 high-dose patients appeared robust, showing 80% and 67% slowing of disease progression compared to external controls.
The Food and Drug Administration is reviewing UniQure's marketing application for AMT-130, with a verdict expected in about eight months as the company reported a $135 million net loss this year.
Despite mixed results, the Netherlands-based company maintains AMT-130 demonstrates a "meaningful" effect on Huntington's disease, noting rare disease communities often accept greater risks for potential treatment options.