uniQure’s (QURE) stock experienced a dramatic surge, jumping more than 111% to reach $15.43 in midday trading. This spike came after the company announced it had reached a significant agreement with the U.S. Food and Drug Administration (FDA) concerning the Accelerated Approval pathway for its gene therapy candidate, AMT-130, aimed at treating Huntington’s disease.
Walid Abi-Saab, M.D., uniQure’s chief medical officer, expressed enthusiasm about this development, emphasizing the collaborative and productive discussions with the FDA’s Center for Biologics Evaluation and Research (CBER). The agreement signifies a pivotal moment for the Huntington’s disease community, offering hope for a faster route to providing a potentially life-altering treatment. This pathway allows uniQure to bypass the need for additional pre-submission studies by leveraging data from ongoing Phase I/II studies, which will be compared against a natural history external control for the Biologics License Application (BLA).
This strategic alignment with the FDA not only accelerates the path to market for AMT-130 but also underscores the robustness of the clinical data uniQure has gathered. The company has already started preparations for the BLA, with plans to further engage with the FDA in the first half of 2025 to refine their statistical analysis and meet technical requirements.
Adding to the positive momentum, Raymond James upgraded QURE’s rating from Outperform to Strong Buy, reflecting a bullish outlook on the stock’s future performance based on this regulatory progress. This upgrade and the stock’s significant jump highlight investor confidence in uniQure’s direction, particularly its potential to bring a new therapy to market for Huntington’s disease, a condition currently without a cure.
This development marks a critical step forward in gene therapy, potentially setting a precedent for how such treatments could be expedited through regulatory processes to reach patients in need sooner.
Price Action: At last check, QURE was up 111.45% to $15.43. The ticker is up 128% year to date and 116% year over year.
Leave a Reply