BioMarin Pharmaceutical has dosed the first patient in the global GENEr8-1 Phase 3 study with the 6e13 vg/kg dose for valoctocogene roxaparvovec (formerly BMN 270), an investigational gene therapy for the treatment of patients with severe hemophilia A.
This is the first of two Phase 3 studies in the global Phase 3 program to dose a first patient.
“We are thrilled to have enrolled the first patient in this potential registration-enabling study for gene therapy in severe hemophilia A,” said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. “I am proud of the effort that BioMarin has made to move this breakthrough therapy rapidly into Phase 3 bringing this one-time treatment one step closer to patients.”
The global Phase 3 program includes two studies with valoctocogene roxaparvovec, one with the 6e13 vg/kg dose (GENEr8-1) and one with the 4e13 vg/kg dose (GENEr8-2). Both Phase 3 GENEr8 studies will be open-label single-arm studies to evaluate the efficacy and safety of valoctocogene roxaparvovec. GENEr8-2 will enroll the first patient at the start of 2018. The primary endpoint in both studies will be based on the FVIII activity level achieved following valoctocogene roxaparvovec, and the secondary endpoints will measure annualized FVIII replacement therapy use rate and annualized bleed rate.
BioMarin will also begin a Phase 1/2 Study with the 6e13kg/vg dose and with approximately 10 patients who are AAV5 positive. The first patient is expected to enroll in the first half of 2018.