Global biotech BioMarin Pharmaceutical Inc. (BMRN) has announced updated results of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. People living with hemophilia A lack a specific protein to help their blood clot and are at risk for painful and potentially life-threatening bleeds from even modest injuries.
The four-year update and three-year update demonstrated that all subjects remain off prophylactic Factor VIII treatment since receiving their single dose of valoctocogene roxaparvovec.
Cumulative mean annualized bleed rates (ABR) remained less than one (1) in both cohorts and below pre-treatment baseline levels. The mean ABR in year four for the 6e13 vg/kg cohort was 1.3, and the mean ABR in year three for the 4e13 vg/kg cohort was 0.5. Over the past year, six of the seven participants in the 6e13 vg/kg cohort and five of the six participants in the 4e13 vg/kg cohort remain free of spontaneous bleeds. Factor VIII remain in a range to provide hemostatic efficacy.
“This additional data is an important step toward a potential first treatment of its kind for this devastating disease,” said Professor John Pasi, M.B., chief investigator for the study. “Each year of data increases our knowledge of safety and efficacy and contributes to the growing body of scientific data on gene therapies in general and hemophilia A in particular.”
Overall, the safety profile remained consistent with previously reported data with no delayed-onset treatment related events, BioMarin said.
The data has been submitted as a late-breaking abstract to the World Federation of Hemophilia (WFH) Virtual Summit, which will take place on June 14-19, 2020.
The Food and Drug Administration (FDA) is currently reviewing the biologics license application, under Priority Review, for valoctocogene roxaparvovec with a PDUFA action date of August 21, 2020. The FDA has already granted valoctocogene roxaparvovec Breakthrough Therapy designation and Orphan Drug status.
Meanwhile the European Medicines Agency (EMA) has validated the company’s Marketing Authorization Application (MAA) for the gene therapy. However, BMRN expects the review procedure to be extended by at least three months due to COVID-19 delays. It now expects an opinion from the Committee for Medicinal Products for Human Use (CHMP) in late 2020/early 2021.
Approximately 1 in 10,000 people have Hemophilia A. Additionally, people with severe hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. These individuals with the most severe form of the disease make up 50% of the hemophilia A population.
Shares in BioMarin have soared 26% year-to-date, and the Street has a bullish take on the stock’s prospects. Fifteen analysts have published recent buy ratings vs 2 hold ratings, adding up to a Strong Buy consensus. Meanwhile the average analyst price target indicates 12% further upside potential lies ahead. (See BioMarin stock analysis on TipRanks).
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