BioMarin highlights new results for valoctocogene roxaparvovec at ASH
BioMarin Pharmaceutical announced updates on valoctocogene roxaparvovec -- formerly BMN 270, an investigational gene therapy treatment for severe hemophilia at ASH. With the 4e13 vg/kg dose, the three patients with the longest follow-up have Factor VIII activity levels that are in or near to the normal range with both median and mean values of 49%. Median annualized bleed and factor VIII use rates for the 4e13 vg/kg cohort were zero after Week 4 and when their Factor VIII activity rose above 5%. Mean annualized bleed and factor VIII use rates for the 4e13vg/kg cohort were 0.6 and 2.0, respectively. With the 6e13 vg/kg dose, at 78 weeks post infusion, the median and mean Factor VIII levels of the 6e13 vg/kg cohort were 90 and 89%, respectively. Median annualized bleed and factor VIII use rates for the 6e13 vg/kg were zero after Week 4. Mean annualized bleed and factor VIII use rates for the 6e13 vg/kg cohort were 0.5 and 6.1, respectively. The company also announced that the New England Journal of Medicine published an independent, peer-reviewed article on the ongoing Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy, in men with severe hemophilia A. The article assessed the safety and efficacy of valoctocogene roxaparvovec at the 6e13 dose, after 52 weeks. As presented at ASH on December 10, the objective of this study was to determine the comparative pharmacodynamics of valoctocogene roxaparvovec when given as a single intravenous bolus injection to cynomolgus monkeys with varying baseline anti-AAV5 total antibody levels and transduction inhibition titers. The results demonstrated no evidence for decreased FVIII expression in animals with non-antibody based transduction inhibition, while baseline AAV5 antibody positive animals had a range of FVIII expression.