BioMarin doses first patient in in a Phase 1/2 study evaluating valoctocogene
BioMarin Pharmaceutical announced that it has dosed the first patient in a Phase 1/2 study evaluating its investigational gene therapy, valoctocogene roxaparvovec, in severe hemophilia A patients with pre-existing AAV5 antibodies. The study is an open-label, single-arm, titer-escalation trial evaluating the safety and efficacy of valoctocogene roxaparvovec in AAV5+ hemophilia A patients. Patients with pre-existing AAV5 antibodies will be sequentially enrolled into two titer cohorts that will encompass the range of observed AAV5 antibody titer levels generally observed in the hemophilia population and be treated with the 6e13 vg/kg dose. The primary endpoint will evaluate safety of valoctocogene roxaparvovec in this population. Secondary endpoints include assessment of FVIII activity level, frequency of required FVIII replacement therapy, and the number of bleeding episodes requiring treatment after therapy.