Pfizer Reports P-III Study (BENEGENE-2) Results of Fidanacogene Elaparvovec for Hemophilia B
- The (BENEGENE-2) study evaluating fidanacogene elaparvovec (5e11vg/kg, IV) vs prophylaxis regimen with Factor IX in 45 adult males
- The study met its 1EPs of non-inferiority & superiority in ABR of total bleeds post-fidanacogene elaparvovec, superiority with a mean ABR for all bleeds of 1.3 for 12mos. from 12wk. to 15mos. vs ABR of 4.43 during the lead-in pre-treatment period of 6mos., 71% reduction in ABR after a single dose
- In the 2EPs, 78% reduction in ABR & 92% in annualized infusion rate; 27% mean FIX activity @15mos. by one-stage SynthASil assay & 25% @24mos., mean steady-state FIX: C was higher over the pre-specified threshold, was well-tolerated. The safety profile was consistent with P-I/II trial, SAEs were reported in 16% of patients
Ref: Business Wire | Image: Pfizer
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