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Hemophilia A at ASH 2019
Advances in Hemophilia A Treatment: A Peek Ahead to ASH 2019

Released: December 03, 2019

Expiration: December 01, 2020

Cindy A. Leissinger
Cindy A. Leissinger, MD
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Although recombinant factor VIII (FVIII) concentrates have been the cornerstone of hemophilia A treatment for almost 2 decades, replacement therapy can impose a significant burden on patients. Efforts continue to find safer, more tolerable, and more effective agents—many of which will have new data presented at the 2019 ASH ASH meeting, where my colleagues and I will present in an upcoming symposium in Orlando.

Break Through the VWF-Imposed Half-life Ceiling
Frequent administration is required for prophylaxis with standard half-life FVIII replacement therapies. And between infusions, low FVIII activity is associated with increased risk of bleeding. BIVV001 is a next-generation FVIII therapy that circulates independently of endogenous von Willebrand factor (VWF), thus breaking the half-life ceiling imposed by VWF. The safety, tolerability, and pharmacokinetics of repeat dosing with BIVV001 were evaluated in patients with severe hemophilia A in a recent phase I trial.

Four weekly infusions of BIVV001 50 IU/kg (n = 10) and 65 IU/kg (n = 14) were well tolerated with no identified safety concerns. With weekly dosing, FVIII trough levels appeared to be higher than those achieved with existing extended half-life products. By breaking through the half-life ceiling imposed by VWF, BIVV001 may better protect against bleeds than do standard FVIII therapies in patients with severe hemophilia A.

Next-Generation FVIII Mimetic
Recently, the FVIII mimetic emicizumab was approved by the FDA for patients with hemophilia A with or without inhibitors. Emicizumab is a bispecific antibody that offers a subcutaneous prophylactic treatment option with potential to significantly reduce treatment burden in patients with hemophilia.

And another promising FVIII mimetic is in development and being presented at the meeting is Mim8, a highly potent, subcutaneous humanized bispecific antibody in preclinical and early-stage development for patients with hemophilia A with or without inhibitors. It has approximately 15 times greater potency than emicizumab and a terminal half-life of 14 days (range: 10-17 days). The high potency of Mim8 allows for small volume administration in pen devices.

Of course, there are numerous promising factor and nonfactor approaches, such as anti-TFPI antibodies and siRNA, which aim to rebalance the coagulation pathway, and multiple gene therapies, which aim to provide cure or near-cure for hemophilia. The 2019 ASH meeting promises plenty of information on the latest advances in these alternative approaches for the care of our patients with hemophilia.

Your Thoughts?
These data suggest promising strides toward reducing the burden of disease associated with severe hemophilia A. We will discuss these and other new and emerging approaches—and how they may help you improve outcomes in your patients with hemophilia A—at our upcoming CME-certified symposium at the ASH annual meeting in Orlando.

What hemophilia studies are you most looking forward to? Please tell us your thoughts in the comment box below. Then join me and my colleagues, Michael Callaghan, MD; Stacy E. Croteau, MD, MMS; and Guy A. Young, MD, for expert guidance and practical discussions on advances in hemophilia. If you cannot be there in person, you can register to attend a live simulcast from your home or office.

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