BioMarin Shares 5-Year Data From Open-Label Trial Of Hemophilia Gene Therapy

BioMarin Pharmaceutical Inc BMRN has announced new data from open-label Phase 1/2 trial of valoctocogene roxaparvovec, an investigational gene therapy candidate for severe hemophilia A.

• Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.  
• Five-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, show a sustained treatment benefit of valoctocogene roxaparvovec. 
• All participants in both cohorts remain off prophylactic Factor VIII treatment. 
• The 6e13 vg/kg dose cohort of the Phase 1/2 study (N=7), with a mean follow-up of 266.1 weeks (5.1 years), showed that a single dose after week 4 reduced the mean Annualized Bleed Rate (ABR) by 95% to 0.8. 
• In year 5, 86% (6 of 7) of study participants in the 6e13 vg/kg dose cohort had no treated bleeds.
• The 4e13 vg/kg dose cohort of the Phase 1/2 study (N=6), with a mean follow-up of 218.6 weeks (4.2 years), showed that a single dose reduced mean ABR by 92% to 1.0. 
• In year 4, 50% (3/6) of study participants in the 4e13 vg/kg dose cohort had no treated bleeds.
• In the 6e13 vg/kg and 4e13 vg/kg dose cohorts reduced infusions per year by 96% from 135.6 to 5.2, and by 95% from 142.8 to 7.8, respectively.
• Price Action: BMRN shares are up 0.54% at $79.73 during the market session on the last check Wednesday.