UniQure achieves planned enrollment in HOPE-B trial of AMT-061

UniQure N.V. (NASDAQ:QURE) announces that the planned enrollment of 56 patients has been achieved in the HOPE-B pivotal trial of etranacogene dezaparvovec (AMT-061), an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B.

Due to the high level of interest in the study from both patients and study investigators, uniQure expects to over-enroll up to six additional patients before the end of September.

The study’s primary endpoint is the assessment of Factor IX activity 26 weeks after dosing.

Patients enrolled in the HOPE-B trial will be tested for the presence of pre-existing neutralizing antibodies to AAV5 but will not be excluded from the trial based on their titers.

https://seekingalpha.com/news/3496320-uniqure-achieves-planned-enrollment-hope-b-trial-amtminus-061