UniQure tumbles after FDA says brain disorder therapy data insufficient, seeks new trial

uniQure

02/03/2026 12:47

** U.S.-listed shares of Dutch firm UniQure UQ1.F, QURE.O fall 43.4% to $8.85 premarket

** UniQure says the U.S. health regulator informed the company that data from an early- to mid-stage trial of its gene therapy for a brain disorder would not support a marketing application for the treatment

** Co is developing a gene therapy, AMT-130, for Huntington's disease, a rare condition that affects motor function; the therapy is delivered directly to the brain by inserting a micro-catheter

** Co says received final minutes from a meeting held on January 30, in which the FDA concluded results from the studies — when compared to an external control — cannot serve as primary evidence of effectiveness required for approval

     ** The FDA recommended uniQure to run a new study that randomly assigns patients to get either the gene therapy or a sham surgery

** Stock has fallen 63% in 2025

 (Reporting by Siddhi Mahatole)

 ((siddhi.mahatole@thomsonreuters.com))