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Breaking the Von Willebrand Ceiling in Hemophilia

The first transatlantic flight. Breaking the sound barrier. Running a 4-minute mile. All notable “firsts” that were thought to be near impossible before they were achieved. Recently, our researchers achieved another notable first for people living with hemophilia A.

Data presented at an international congress earlier this year showed that Bioverativ’s investigational therapy for hemophilia A, BIVV001, had broken through the von Willebrand ceiling for the very first time. For decades, scientists had been working to overcome the von Willebrand factor ceiling, which limits the time - or half-life - that a factor VIII replacement therapy can stay in the body to provide protection from bleeding. Importantly, this accomplishment has the potential to transform the treatment paradigm for people with hemophilia A.

Early Results Show Promise

Early interim results from the ongoing EXTEN-A clinical trial are promising, showing that BIVV001 stays nearly twice as long in the body. In the study, BIVV001 demonstrated an unprecedented half-life of 37 hours with high factor VIII activity at seven days (>5%) following a single low dose of 25 IUs/kg (n= 4 patients). With BIVV001, Bioverativ is exploring the possibility of weekly dosing while maintaining protection from all types of bleeds.

Factor activity levels are important in hemophilia A as they refer to the amount of factor VIII in a person’s plasma, and they determine a person’s ability to clot properly. As a reference, participants in this study have severe hemophilia A which means they have a factor activity level of <1%. Moderate hemophilia A is characterized by factor levels of 1-5%, and mild hemophilia A is from 6-49%.1

Providing Complete Protection in Hemophilia

For people living with hemophilia, effective treatment means protection from all bleeds – the ones you see and the ones you don’t. People with hemophilia A are deficient in a protein in their blood called factor VIII; this means their blood does not clot properly which can result in bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages

“Bioverativ introduced the first extended half-life factor therapies in 2014 which represented the first major advancements in the treatment of hemophilia in nearly 20 years,” said Rob Peters, PhD, Senior Vice President of Research. “Now, with BIVV001, we are building on the proven biology of our factor therapies with the goal of taking another huge step forward for patients and once again changing the treatment paradigm. At Bioverativ, our research and development teams are committed to enabling protection for people with hemophilia, striving each day to improve the standard of care for patients.”

The Joy of Discovery

As the lead scientist behind BIVV001, Ekta Seth Chhabra thinks about hemophilia every day. She has spent much of the last decade pursuing a deep understanding of the biology of the disease to engineer solutions that could have a meaningful impact on the lives of people with this rare blood disorder.

“It has been exciting and gratifying to see the early clinical results for BIVV001,” says Dr. Chhabra. “Over the course of five years, we tested more than 500 different molecules to find a viable solution and then spent another two years in post-research development before we were ready to begin testing the new drug candidate in people.”

Bioverativ continues to test BIVV001 in more patients and at higher doses, and expects additional data to be available later this year. It’s important to note that this is an investigational treatment, and its safety and efficacy has not been fully evaluated by any regulatory authority.

1 National Hemophilia Foundation, Hemophilia A. Available at https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A. Accessed May 17, 2018