Pfizer Touts Positive Results From Late-Stage Hemophilia B Gene Therapy Trial

Pfizer Inc PFE announced topline results from the Phase 3 BENEGENE-2 study of fidanacogene elaparvovec, an investigational gene therapy, for severe hemophilia B.
The 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.
Key secondary endpoints demonstrated a 78% reduction in treated ABR and a 92% reduction in annualized infusion rate.
Fidanacogene elaparvovec was generally well-tolerated
Pfizer currently has three Phase 3 programs investigating gene therapy for hemophilia B, hemophilia A, and Duchenne muscular dystrophy.
Related: Pfizer Resumes Dosing In Phase 3 Hemophilia Gene Therapy Trial Six Months After FDA Lifts Clinical Hold.
Pfizer licensed SPK-9001 (fidanacogene elaparvovec), from Spark Therapeutics under a December 2014 agreement.
Price Action: PFE shares are up 0.35% at $50.98 during the premarket session on the last check Thursday.
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