Sanofi Genzyme reported positive data from their recently completed phase 1/2 EXTEN-A study of their investigational factor replacement product for hemophilia A. BIVV001 is a recombinant factor VIII therapy (rFVIII) designed for the prevention of bleeding episodes in hemophilia A patients via once weekly prophylactic infusions. The data was included in an oral presentation at the XXVII Congress of the International Society on Thrombosis and Haemostasis, July 6-10, 2019 in Melbourne, Australia.
EXTEN-A was a multicenter trial designed to evaluate the safety, tolerability, and pharmacokinetics of BIVV001 in two different size doses in severe hemophilia A patients between ages 18-65 years. Enrolled individuals were placed in one of two groups, depending on the size dose they received.
Patients who received the larger dose of the therapy achieved a factor VIII (FVIII) half-life of 43 hours, representing a significant increase from the 13 hour half-life reached in response to treatment with an existing rFVIII product. Average FVIII activity for participants in the larger dose group was 38% at five days post-BIVV001 administration and 17% at seven days – these represent therapeutically significant increases from less than 1% FVIII levels that are normally found in individuals with severe hemophilia A. In the smaller dose group, patients achieved a FVIII half-life of 38 hours, signifying a notable improvement on the nine hour half-life achieved with an existing FVIII product. The company also reported that the therapy was generally well tolerated with no inhibitor development and no adverse events.
Overall, a single, prophylactic dose of BIVV001 resulted in sustained FVIII activity levels across trial participants who benefited from an extended half-life 3-to-4-fold higher than conventional rFVIII.
“These data reinforce the potential for BIVV001 to address an area of significant unmet need for people with hemophilia A; optimal protection from bleeds with longer prophylactic dosing intervals,” said Mouhamed Gueye, PharmD, MBA, Head of Global Medical Affairs, Rare Blood Disorders, Sanofi Genzyme. “Factor replacement therapy is a foundational component of hemophilia care and these early clinical results support the continued development of BIVV001.”
Source: Sanofi Genzyme press release dated July 7, 2019
