Strong Phase III results for Pfizer's hemophilia B gene therapy candidate

Slightly earlier than expected, US pharma giant Pfizer (NYSE: PFE) last week announced positive top-line results from the Phase III BENEGENE-2 study evaluating fidanacogene elaparvovec, an investigational gene therapy, for the treatment of adult males with moderately severe to severe hemophilia B.

The BENEGENE-2 study met its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion versus prophylaxis regimen with Factor IX (FIX), administered as part of usual care. The results demonstrated superiority with a mean ABR for all bleeds of 1.3 for the 12 months from week 12 to month 15 compared to an ABR of 4.43 during the lead-in pre-treatment period of at least six months, resulting in a 71% reduction in ABR (p<0.0001) after a single dose of 5e11 vg/kg of fidanacogene elaparvovec.

Key secondary endpoints demonstrated a 78% reduction in treated ABR (p=0.0001) and a 92% reduction in annualized infusion rate (p<0.0001). Mean FIX activity was 27% at 15 months by one-stage SynthASil assay and 25% at 24 months. The mean steady-state FIX:C was significantly higher than the pre-specified threshold of 5% (p<0.0001).