Sebelipase alfa: Additional Phase III data

Additional data from the double-blind, international Phase III ARISE trial in 66 patients ages >=4 with LAL deficiency showed that 1 mg/kg IV Kanuma every 2 weeks led to ALT normalization, the primary endpoint, in 31% of patients vs. 7% of patients receiving placebo (p=0.03). On secondary endpoints, Kanuma reduced mean LDL-C from baseline to week 20 by 28.4% vs. 6.2% for placebo (p<0.001), non-HDL-C by 28% vs. 6.9% for placebo (p<0.001) and triglycerides by 25.5% vs. 11.1% for placebo (p=0.041). Kanuma also significantly increased mean HDL-C from baseline to week 20 by 19.6% vs. a reduction of 0.3% for placebo (p<0.001). Additionally, 42% of patients receiving Kanuma achieved AST normalization vs. 3% of patients receiving placebo (p<0.001) and Kanuma reduced mean hepatic fat content as assessed by multi-echo gradient echo (MEGE) MRI from baseline to week 20 by 32% vs. 4.2% for placebo (p<0.001). Furthermore, Kanuma reduced mean ALT levels from baseline to week 20 by 58 U/L vs. 7 U/L for placebo (p<0.001). Data were published in The New England Journal of Medicine. ...