Note: The below is an edited version of a press release issued by BioMarin Pharmaceutical Inc. The original release can be read in its entirety here.
BioMarin Pharmaceutical announced today positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A. Nine patients with severe hemophilia A received a single dose of BMN 270. Six of the seven patients treated with the highest dose had Factor VIII levels above 50% as of each patient’s most recent reading (between 12 and 28 weeks post-treatment). Two patients with Factor VIII levels above 200% had no unexpected events or need for medical intervention. No clinically relevant sustained rises in ALT levels or other markers of liver toxicity were observed.