Bioverativ Presents Preliminary Phase 1/2a Data on BIVV001, the First Factor VIII Therapy to Break Through the VWF Half-Life Ceiling in Hemophilia A
EXTEN-A is an ongoing Phase 1/2a, open-label, multicenter study to evaluate the safety and pharmacokinetic (PK) of BIVV001 in both a low-dose and high-dose cohort of subjects aged 18-65 years with severe hemophilia A. In the data presented at the WFH World Congress, four adult males from the low-dose cohort received a single dose of rFVIII (25 IU/kg) followed, after a washout period, by a single low dose of BIVV001 (25 IU/kg). Primary endpoints include occurrence of adverse events and development of inhibitors. Secondary endpoints related to pharmacokinetic parameters were also presented. Key findings included (cut-off 20 April 2018):
- A single, low dose of BIVV001 extended the half-life of factor VIII to 37 hours, a substantial increase over the 13 hours seen with rFVIII
- Average factor VIII activity for the four subjects was 13.0% at five days and 5.6% at seven days post infusion with a single low dose of BIVV001
- BIVV001 was generally well tolerated, with no development of inhibitors
BIVV001(rFVIIIFc-VWF-XTEN) is a novel and investigational recombinant factor VIII therapy that is designed to extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. BIVV001 builds on the company’s innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to potentially extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. BIVV001 was granted orphan drug designation by the Food and Drug Administration in August 2017.
About Hemophilia A
Hemophilia is a rare, genetic disorder in which the ability of a person's blood to clot is impaired. Hemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. The World Federation of Hemophilia estimates that approximately 150,000 people are currently diagnosed with hemophilia A worldwide.3
People with hemophilia A experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. Prophylactic injections of factor VIII can temporarily replace the clotting factor that is needed to control bleeding and prevent new bleeding episodes.4 The World Federation of Hemophilia (WFH) recommends prophylaxis as the optimal therapy as it can prevent bleedings and joint destruction.5
About Bioverativ, a Sanofi company
Bioverativ, a Sanofi company, is dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development, and commercialization of innovative therapies. Bioverativ is committed to actively working with the blood disorders community, and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. For more information, visit www.bioverativ.com or follow @bioverativ on Twitter.
1 Ghosh, K. and Shetty, S. (2012), Bone health in persons with haemophilia: a review. Eur J Haematol, 89: 95-102. doi:10.1111/j.1600-0609.2012.01803.x
2 National Hemophilia Foundation, Hemophilia A. Available at https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Hemophilia-A . Accessed May 17, 2018
3 World Federation of Hemophilia, Annual Global Survey 2016, published in October 2017. Available at: http://www.wfh.org/en/data-collection
4 World Federation of Hemophilia. About Bleeding Disorders – Frequently Asked Questions. Available at: http://www.wfh.org/en/page.aspx?pid=637#Difference_A_B. Accessed on: June 17, 2016
5 Guideline for the management of hemophilia, World Federation of Hemophilia, 2nd edition, http://www1.wfh.org/publications/files/pdf-1472.pdf. Accessed on December 2015
Marianne McMorrow, +1 781-663-4376
Source: Bioverativ Inc.