75% Disease Slowing: uniQure's AMT-130 Nears UK Submission for Huntington's
QURE has more than doubled off its 52-week low of $8.73.
Summary
uniQure announced a successful pre-submission meeting with the UK MHRA for AMT-130 in Huntington's disease, with plans to submit a Marketing Authorization Application (MAA) in Q3 2026. This follows the FDA's previous rejection of the BLA pathway for AMT-130, requiring a new trial, as disclosed in the recent 10-K. The MAA will be supported by three-year Phase I/II data showing a statistically significant 75% slowing of disease progression at the high dose. Additionally, the company has secured a Type B meeting with the U.S. FDA in Q2 2026 to discuss a potential Phase III trial design. This news provides a significant positive development and a clear regulatory path in the UK, alongside renewed engagement with the FDA, after a prior setback for the lead candidate. Traders will be watching for the MAA submission in Q3 and the outcome of the upcoming FDA meeting.
At the time of this announcement, QURE was trading at $17.75 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $1B. The 52-week trading range was $8.73 to $71.50. This news item was assessed with positive market sentiment and an importance score of 8 out of 10. Source: GlobeNewswire.