UniQure Plunges As FDA Slaps Clinical Hold On Hemophilia B Gene Therapy Study On Possible Liver Cancer Link

Uniqure NV QURE shares are moving sharply to the downside after the gene therapy company announced a setback to its clinical study.

What Happened: Lexington, Massachusetts-based uniQure said the FDA has placed its Hemophilia B gene therapy program, including the late-stage HOPE-B study, on clinical hold.

The negative FDA action follows the submission of a safety report in mid-December, which included a serious adverse event of hepatocellular carcinoma – a form of liver cancer – that occurred in a patient in the HOPE B study, who was treated in October 2019. The trial is evaluating uniQure's investigational gene therapy etranacogene dezaparvovec for Hemophilia B.

The company clarified that the patient has multiple risk factors associated with HCC, including a 25-year history of hepatitis C, hepatitis B, evidence of non-alcoholic fatty liver disease and advanced age.

No other cases of HCC have been reported in clinical trials conducted in more than 100 patients in Hemophilia B and other indications, with some patients dosed more than 10 years ago, uniQure said.

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Why It's Important: Etranacogene dezaparvovec, a AAV5-based gene therapy, is the most advanced assets in uniQure's pipeline. At the ASH conference in early December, in a late-breaking presentation of the initial date, the company said the HOPE-B pivotal study met the primary endpoint of mean FIXX activity of 37% of normal at 26 weeks.

What's Next: uniQure said it's working closely with the FDA and its advisors to conduct a thorough investigation into the cause of this event. It expects the review to be completed in early 2021.

"All patients in our hemophilia B gene therapy program, including the 54 patients in HOPE-B, will continue to be monitored by their care teams while we gather additional information as rapidly as possible," said Matt Kapusta, CEO of uniQure.

The company also said it doesn't see any impact from the clinical hold on the regulatory submission timeline for the Hemophilia B program.

In premarket trading Monday, uniQure shares were slumping 16.19% to $38.51.