- Pfizer (PFE -0.4%) initiates a Phase 3 open-label lead-in study evaluating current factor IX prophylaxis replacement therapy in the usual care setting for patients with hemophilia B.
- The results will serve as the within-subject control group for those patients that enroll in the next part of the trial assessing gene therapy fidanacogene elaparvovec for the treatment of hemophilia B. The interventional portion of the trial will involve patients who have completed at least six months in the lead-in study.
- The primary endpoint of the 110-subject study is change in annualized bleed rate from baseline over a six-month treatment period. According to ClinicalTrials.gov, the estimated completion date is November 2020.
- The company inked a collaboration agreement with Spark Therapeutics (ONCE -3.2%) in December 2014 to develop the gene therapy and recently assumed full responsibility for the program.
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