UniQure's hemophilia B gene therapy hit with FDA clinical hold

 

• UniQure (NASDAQ:QURE) drops 19% in premarket in reaction to the FDA clinical hold on its hemophilia B gene therapy program, including the pivotal, Phase 3 HOPE-B study. Patient dosing is complete in each of uniQure's three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients.
• The hold comes after the submission of a safety report related to a possibly related serious adverse event associated with a preliminary diagnosis of hepatocellular carcinoma, a form of liver cancer, in one patient in the HOPE-B trial that was treated with etranacogene dezaparvovec (AMT-061) in October 2019.
• FDA investigation is expected to complete by early 2021.
• uniQure management along with trial investigators hosted a conference call today, at 8:30 a.m. ET.
• https://seekingalpha.com/news/3646104-uniqures-hemophilia-b-gene-therapy-hit-fda-clinical-hold-stock-down-19