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uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug Administration (FDA) has removed the clinical hold on the Company鈥檚 hemophilia B gene therapy program after determining the Company satisfactorily addressed all issues identied by the FDA related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B pivotal trial.

鈥淧atient safety is our top priority, and we are grateful to our advisors and the FDA for their help in resolving this clinical hold,鈥 stated Ricardo Dolmetsch, Ph.D., president of research and development at uniQure. 鈥淥ur comprehensive investigation showed that AMT-061 (etranacogene dezaparvovec) is very unlikely to have contributed to the HCC in our patient. We look forward to announcing top-line 52-week data from the HOPE-B pivotal trial later this quarter.鈥

uniQure previously announced on December 21, 2020, that the FDA placed the hemophilia B program on clinical hold following the diagnosis of HCC in one patient in the HOPE-B trial. The patient had multiple risk factors associated with HCC, including a twenty-ve-year history of hepatitis C (HCV), history of hepatitis B (HBV). Chronic infections with hepatitis B and C have beenassociated with approximately 80% of HCC cases.

Following a surgical resection of both tumor and adjacent liver tissue, multiple analyses conducted by an independent laboratory and reviewed by leading external experts in the eld show that AAV vector integration in the patient鈥檚 tissue sample was extremely rare and accounted for 0.027% of the cells in the sample. The integration events were distributed randomly across the genome, and there was no evidence of clonal expansion or any dominant integration event. Additionally, whole genome sequencing of the tumor conrmed that the tumor had genetic mutations that are characteristic of HCC and are independent of vector integration. Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver that may have predisposed this patient to HCC.

All patients in uniQure鈥檚 hemophilia B gene therapy program, including the 54 patients in HOPE-B, have had abdominal ultrasounds performed one year after dosing, and each will continue to be monitored by their care teams. Patients will continue聽to receive abdominal ultrasounds every six-months. 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 10 years ago.


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