According to an article from the National Hemophilia Foundation, CSL Behring and uniQure have recently provided an update on their phase III HOPE-B trial. This study is evaluating etranacogene dezaparvovec, which is an investigational gene therapy for hemophilia B. The update was a positive one, as the two companies announced that the primary endpoint had been met.

Trial Update

First, let’s take a look at this trial as a whole. Titled the HOPE-B trial, this phase III study is investigating etranacogene dezaparvovec as a treatment for patients with moderately severe to severe hemophilia B. 54 participants have been dosed as of now, with all but one completing 18 months of follow-up.

The investigational gene therapy is being compared to baseline factor IX prophylactic replacement therapy in order to establish non-inferiority. This means that researchers are testing to see if etranacogene dezaparvovec is more efficacious than its comparator. In fact, the recent update from the two companies was to announce that this primary endpoint of non-inferiority in terms of annualized bleeding rate (ABR) was met.

Previously announced results include:

• ABR was 1.51 after 18 months of follow-up
  • This can be compared to an ABR of 4.19 during the six-month lead-in period
• Durable and sustained FIX expression after 18 months
• The gene therapy was generally well-tolerated
  • 80% of adverse events (AEs) were mild

These results, along with the new update, are very positive for medical professionals and hemophilia patients alike. Hopefully, data will continue to be positive, and hemophilia B patients will have a new, viable treatment option.

About Hemophilia B

Hemophilia B falls under the larger category of hemophilia, and it is characterized by a deficiency of the factor IX (FIX) gene. This stops blood from clotting properly, leading to prolonged bleeding. Affected individuals experience symptoms like unprovoked and spontaneous bleeds, easy bruising, nosebleeds, extending bleeding, blood in the stool, and bleeding in the urinary and/or gastrointestinal tract. It’s important to be aware of this condition if one is undergoing surgery or procedures, as they could experience prolonged bleeding. In fact, it is necessary to seek treatment in general, as untreated hemophilia B could lead to serious complications and even fatality.