Bioverativ to Present New Data on Hemophilia and Other Blood Disorders at ASH

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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Bioverativ will present some of its most recent therapeutic advancements regarding hemophilia and other blood diseases at the 60th Annual Meeting of the American Society of Hematology (ASH) in San Diego, California, Dec. 1-4.

The company, which is part of Sanofi, has focused on the development and commercialization of innovative therapies for hemophilia and other rare blood disorders for the past 20 years.

Research highlights include an oral presentation on the Phase 1/2a trial for BIVV001 (rFVIIIFc-VWF-XTEN) (NCT03205163), still recruiting patients, and poster presentations confirming the long-term safety and efficacy of Eloctate and Alprolix.

“The new data we are presenting at ASH highlight our commitment to protection in hemophilia and our focus on advancing cutting-edge science that has the potential to make a meaningful, positive impact for people with rare blood disorders,” Tim Harris, PhD, executive vice president of research and development at Bioverativ, said in a press release.

BIV001 is the first investigational blood-clotting factor VIII replacement therapy designed to extend hemophilia A patients’ protection from bleeding episodes through a preventive regimen of one dose per week or longer.

Findings presented at the National Hemophilia Foundation Bleeding Disorders Conference demonstrated that a small dose (25 IU/kg) of BIVV001 prolonged the half-life of factor VIII to 37 hours, which is more than four times that of recombinant (made in the lab) factor VIII (rFVIII), and, at the same time, ensured its activity levels remained above 5% for seven days.

The ASH oral presentation will focus on preliminary data from the same study regarding patients receiving high doses of BIVV001.

Data from ASPIRE (NCT01454739), the long-term extension phase 3 study to address the safety and efficacy of Eloctate for hemophilia A carried out by Bioverativ and Sobi, and B-YOND (NCT01425723), the trial intended to assess the long-term effectiveness and safety of Alprolix for hemophilia B, will also be presented.

The company is also planning to present preclinical findings on BIVV003, a candidate gene-editing cell therapy based on modifying a small portion of the BCL11A gene in patients’ hematopoietic stem cells (blood cells’ precursors, also known as HSCs) to prevent the production of abnormal hemoglobin and treat sickle cell disease.

The investigational new drug (IND) application for BIVV003, which has been developed with Sangamo Therapeutics, has already been accepted by the U.S. Food and Drug Administration (FDA). Bioverativ already launched a Phase 1/2 trial (NCT03653247) to test its efficacy, safety and tolerability in patients with sickle cell disease.