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The following is an excerpt from a press release from uniQure. Read the update in its entirety聽here.


Today uniQure announced a clinical hold by the U.S. Food and Drug Administration (FDA) in its聽phase III HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients.

Per uniQure鈥檚 statement:

鈥淭he clinical hold was initiated following the submission of a safety report in mid-December relating to a possibly related serious adverse event associated with a preliminary diagnosis of hepatocellular carcinoma (HCC), a form of liver cancer, in one patient in the HOPE-B trial that was treated with etranacogene dezaparvovec (AMT-061) in October 2019. The patient has multiple risk factors associated with HCC, including a twenty-five-year history of hepatitis C (HCV), hepatitis B virus (HBV), evidence of non-alcoholic fatty liver disease and advanced age. Chronic infections with hepatitis B and C have been associated with approximately 80% of HCC cases.

The liver lesion was detected during a routine abdominal ultrasound conducted as part of the required study assessments in patients at one-year post dosing. A full surgical resection of the lesion is scheduled this week that will allow for confirmation of the diagnosis. 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.”

Additionally, uniQure management along with Dr. Steven Pipe, principal investigator in the pivotal, Phase III HOPE-B trial, and Dr. Graham Foster, professor of hepatology at Queen Mary University of London, UK, will host a conference call today, Monday, December 21, 2020, at 8:30 a.m. ET. The conference call may be accessed by dialing (877) 870鈥9135 for domestic callers and +44 020 719 283 38 for international callers. The passcode is 5892217. Please specify to the operator that you would like to join the 鈥渦niQure Conference call.鈥 The webcast may also be accessed through the Investors section of the uniQure website at http://uniqure.com/investors-newsroom/overview.php. Following the live webcast, a replay of the call will be archived for several weeks.

Patients who are enrolled in the clinical trial will continue to be under close clinical observation聽according to study protocol.

Patient dosing is complete in each of uniQure鈥檚 three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients.

Read the full notice from uniQure聽here.

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As has been our past practice (and consistent with the principles articulated at the聽January 2020 Safety Summit), HFA and NHF commit to keeping the bleeding disorders community informed as we gather further information. Community members are encouraged to submit your questions and concerns to our joint Safety Inbox here.


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