uniQure CEO Defends Huntington’s Therapy Data, Sees Opportunity Ahead of FDA Decision

uniQure N.V. (QURE) shares rose 17.5% in a month, outperforming the industry and S&P 500. The rally was driven by FDA acceptance of its Huntington's disease drug application, ifezuntirgene inilparvovec, for review. Despite weaker Q3 2026 results, investors focus on the drug's potential. QURE's cash position is strong, with $768.4M as of Sept. 30, 2026, expected to fund operations through 2030.

uniQure (QURE) is preparing for a potential FDA panel review of its gene therapy AMT-130 for Huntington's disease after receiving a priority review nod. The company presented 48-month data showing treated patients had less functional decline than untreated ones, though control group limitations were noted. uniQure plans a confirmatory study and is preparing for a potential launch, with $780 million in cash to fund operations into 2030.

uniQure's AMT-130 was accepted for FDA Priority Review with a decision goal of May 1, 2027. The U.K. validated its marketing application. 48-month data showed slowed Huntington’s disease progression. Q3 2026 revenue rose 40.4% to $5.2 million, below estimates, with net loss narrowing to $61.2 million. Cash reserves were $768.4 million.

uniQure N.V. reported that the FDA accepted its AMT-130 BLA with priority review for Huntington's disease, citing 36-month data showing a 60% slowing in TFC decline. The company plans a focused commercial launch targeting 15-30 priority institutions. Management expects the confirmatory study to be underway by the 2027 PDUFA date and cash runway to extend into 2030. SG&A expenses increased by $4.5 million year-over-year.

uniQure (QURE) reported progress in its Q3 2026 earnings call. The company's AMT-130 therapy for Huntington's disease showed a 75% slowing of disease progression in trials. The FDA is reviewing the Biologics License Application (BLA) with a PDUFA date of May 1, 2027. uniQure is preparing for a potential U.S. launch in 2027 and advancing its pipeline, including studies for AMT-260 and AMT-191. The company is also building its commercial teams ahead of the planned launch.

UniQURE (QURE) reported a Q3 net loss of $61.22 million, narrower than last year's profit of $80.53 million. Revenue rose to $5.19 million, and expenses fell. The company advanced clinical trials for Huntington's disease, epilepsy, and Fabry disease. Cash reserves total $768.4 million. QURE shares are down 3.06%.