Street View: Tough path ahead for uniQure after FDA setback on Huntington's therapy

uniQure

03/03/2026 14:20

** Shares of drug developer uniQure UQ1.F, QURE.N fall 12.86% to $9.15 in premarket trading

** The U.S. FDA on Monday informed the company that data from the early- to mid-stage trial of its gene therapy for Huntington's disease was insufficient, and would not support marketing application for the treatment

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

** At least four analysts cut PTs on the company

COSTLIER, LONGER LATE-STAGE PATH

** Mizuho ("neutral," PT: $12) says such a study would be difficult to execute and potentially unethical, and thinks the FDA is unlikely to reverse course despite a 2Q26 meeting

** Cantor ("neutral," PT: $9) says the new study "will inevitably set the company back a few years" and adds QURE may  lose its first-mover competitive advantage as other therapies are also entering late-stage trials, especially with PTC's PTCT.O votoplam, an oral option that avoids neurosurgery

** Barclays ("equal weight," PT: $31) calls its a "major setback" for QURE, adding a late-stage study would likely be costly and a data readout would be multiple years away

** Wells Fargo ("equal weight," PT: $15) says a required late-stage study for the Hutington's program reflects increased time to market and development risk; near-term focus shifts to 2Q26 epilepsy trial results, with details awaited to gauge its value

 (Reporting by Kunal Das in Bengaluru)

 ((Kunal.Das2@thomsonreuters.com;))