UniQure's Stock Is Trading Higher As FDA Lifts Clinical Hold On Its Hemophilia B Gene Therapy Program

The FDA has removed the clinical hold on uniQure NV's QURE hemophilia B gene therapy program after determining the company had satisfactorily addressed all identified issues related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B pivotal trial.
Last month, a comprehensive investigation into the case of HCC diagnosed in one patient in the HOPE-B trial was concluded.
The investigation found that uniQure's hemophilia B gene therapy candidate etranacogene dezaparvovec did not cause liver cancer in the patient.
Following surgical resection of both tumor and adjacent liver tissue, multiple analyses conducted by an independent laboratory and reviewed by external experts showed that AAV vector integration in the patient's tissue sample was extremely rare and accounted for 0.027% of the cells in the sample.
The trial was put on clinical last year in December, after submitting a safety report relating to a possibly related severe adverse event associated with a preliminary diagnosis of hepatocellular carcinoma in one patient.
All patients in uniQure's hemophilia B gene therapy program, including the 54 patients in HOPE-B, have had abdominal ultrasounds performed one year after dosing. Each will continue to be monitored.
Price Action: QURE shares are up 10% at $35.62 in premarket trading on the last check Monday.