QURE

In June 2026, the U.S. FDA aligned with uniQure on accepting a three-year analysis from the Phase I/II study of AMT-130 as the primary basis for a Biologics License Application (BLA) for accelerated approval in Huntington's disease, also indicating that a sham control is no longer required for the confirmatory study. The company successfully completed an upsized public offering in June 2026, raising $259 million and extending its projected cash runway into 2030 with $810.3 million in cash. In September 2026, uniQure announced the submission of a BLA for AMT-130 for Huntington's disease to the U.S. FDA, requesting priority review, along with a parallel Marketing Authorisation Application (MAA) to the U.K. MHRA. The company expects to present four-year data from the ongoing Phase I/II studies of AMT-130 in September 2026. uniQure reported a net loss of $53.5 million for Q1 2026 and $81.1 million for Q2 2026. Preliminary six-month data from the first, low-dose cohort in the Phase I/IIa trial of AMT-260 for refractory mesial temporal lobe epilepsy showed biological signals of therapeutic activity and a favorable safety profile. The development of AMT-162 for ALS (SOD1) was discontinued. Over the last six months, uniQure's stock price increased by 178.95%.