On Tuesday, Stifel, a financial services company, indicated a positive outlook for uniQure (NASDAQ: NASDAQ:QURE) following the biotechnology firm's announcement of reaching an agreement with the U.S. Food and Drug Administration (FDA) on key aspects of an accelerated approval process for its gene therapy, AMT-130.
UniQure revealed that they could use an external control and data from the ongoing phase 1/2 study for AMT-130, a treatment for Huntington's disease.
The FDA has agreed that uniQure can employ the composite Unified Huntington's Disease Rating Scale (cUHDRS) as the endpoint for their study. Earlier this year, a two-year analysis showed a statistically significant difference when compared to natural history, which is considered a favorable development for the company.
"In our view, this is a big win for the stock in the backdrop of low expectations that an accelerated path could actually materialize, primarily in-light of the discordant/confusing mHTT KD data," said Stifel analysts.
Although there are questions remaining about the specifics of the approval process, such as the time point for comparison with natural history and the details of the Statistical Analysis Plan (SAP), Stifel anticipates further updates as uniQure continues its discussions with the FDA throughout the first half of 2025.
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