While it wouldn’t be fair to describe hemophilia B as the red-headed stepchild of the bleeding disorder community, it is far rarer than the more common hemophilia A, and bleeds tend to last longer.
Worse, the majority of people with hemophilia B—some 60%—have a severe form of the disease where their body has less than 1% of the factor IX clotting protein.
So any new development in treating and preventing hemophilia B bleeds has to be welcome news. To date, factor replacement infusions have been pretty much the only option. But that may be changing soon.
UniQure is a biotech company in the Netherlands working to develop gene therapies for a variety of conditions.
This past June, they announced findings from the first part of a Phase I/II clinical trial for a unique gene therapy for patients with hemophilia B.
The first part of the trial treated five patients with severe hemophilia B with a low dose of AMT060, UniQure’s unique (pun definitely intended) adeno-associated virus technology.
The basic idea is that the patient is infused with a single dose of the small virus—the virus doesn’t cause disease, but it does trigger an immune response in the body, which then delivers a functioning factor IX gene directly into the liver.
The hope is that this will, in turn, jump start long-term protein production, which in turn would reduce bleeds.
Speaking at the 21st Congress of the European Hematology Association (EHA) in Copenhagen, lead investigator Dr. Frank Leebeek of the Erasmus University Medical Center in Rotterdam announced that six months after the patients in the trial received one low dose of AMT060, their median level of functioning factor IX level rose by 5.4%.
That may not sound like much, but it was enough to decrease their bleed activity to mild/moderate, and that’s a huge deal.
Of course, there’s still a long way to go before this therapy works its way into the marketplace.
Wider Phase III trials will be needed to see if the results transfer to a bigger group, and UniQure is awaiting follow-up results from the second part of the trial, which saw five patients receive a much higher dose of AMT060. And that’s before we even get into the Byzantium approval process required for approval in the States.
But in the bigger fight against bleeding disorders, this has to count as one of the most exciting developments in years!
Keep those fingers and toes crossed…
