BioMarin Pharmaceuticals Inc. (NASDAQ:BMRN) has provided an update to its ongoing phase 1/2 valoctocogene roxaparvovec study results. The study was evaluating the investigational gene therapy, valoctocogene roxaparvovec, in treating severe hemophilia.
BioMarin submit valoctocogene roxaparvovec study result to WFH summit
The company has submitted the results as an abstract to the expected World Federation of Haemophilia Virtual Summit that will take place between June 14 and June 19, 2020. The three-year 4e13 vg/kg and four-year 6e13 vg/kg cohorts updates demonstrated that participants in both cohorts were off prophylactic Factor Eight treatment after receiving the single dose valoctogene roxaparvovec. Results indicate that aggregate average annualized bleed rates (ABR) were below one in the two cohorts and less than the pre-treatment start point levels.
The ABR mean in three-year 4e13 vg/kg cohort was around 0.5 while that in four-year 6e13 vg/kg was 1.3. In the last one year, 6 out of 7 participants in the four-year cohort and 5 out of 6 participants in the three-year cohort remained free of spontaneous bleeding. Equally, Factor 8 activity levels dropped proportionate with the observations in recent years and were within range to offer hemostatic efficacy.
Data from the study helps understand gene therapy in hemophilia
Professor John Pasi of Barts and the London School of Medicine and Dentistry was the lead investigator in the Phase 1/2 valoctocogene roxaparvovec study and also the principal investigator for the third phase study. Pasi was delighted to be part of the pioneering research team observing patients in the study to understand gene therapy studies in hemophilia A. He added that every year data has been increasing knowledge ion efficacy and safety helped in contributing to growing research in gene therapy.
BioMarin’s Worldwide Research and Development President Hank Fuchs said that the company was proud to advance knowledge on gene therapy’s potential to transform lives. Fuchs added that they were grateful to support the hemophilia community and are working with authorities to deliver treatment for hemophilia patients.