Uniqure's Hemophilia B Gene Therapy Unlikely To Be Cause Of Liver Cancer Reported In One Patient

Uniqure NV QURE has reported results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial of etranacogene dezaparvovec, its hemophilia B gene therapy candidate.

What Happened: The trial was put on clinical last year in December, following the submission of a safety report in mid-December relating to a possibly related severe adverse event associated with a preliminary diagnosis of hepatocellular carcinoma in one patient.

The results of the investigation found that it is implausible the HCC was caused by etranacogene dezaparvovec.

Multiple analyses conducted by an independent laboratory and reviewed by external experts in the field show that AAV vector integration in the patient's tissue sample was extremely rare and accounted for 0.027% of the cells in the sample.

Why It Matters: The whole-genome sequencing of the tumor confirmed that the patient had several genetic mutations characteristic of HCC and independent of vector integration.

Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver consistent with several risk factors that predispose this patient to HCC.

The external lab analyzed more than 220,000 cells from the tissue sample and identified 60 cells with random integration events that have no known association with the development of HCC," stated Ricardo Dolmetsch, uniQure president of research and development.

See also: Best Private Health Insurance

The company has shared the data with the FDA and is prepared to have further communications regarding the clinical hold status in the second quarter of 2021.

All patients in uniQure's hemophilia B gene therapy program, including the 54 patients in HOPE-B, have now had abdominal ultrasounds performed one year after dosing. Each will continue to be monitored by their care teams.

No other cases of HCC have been reported in uniQure clinical trials conducted in more than 100 patients in hemophilia B and other indications, with some patients dosed more than ten years ago.

Price Action: QURE shares are trading 9.47% higher at $32.01 in the premarket session on the last check Monday.