Pfizer Announces Positive Phase 3 Results for Fidanacogene Elaparvovec in Hemophilia B

Intelligent Finance APP learned that Pfizer (PFE.US) announced on Thursday that the company's investigational gene therapy fidanacogene elaparvovec for hemophilia B reached the primary endpoint in the phase 3 trial named BENEGENE-2, which involved adult male patients with moderate to severe forms of the disease.

According to reports, the company disclosed trial data from 40 subjects, indicating superiority in the mean adjusted annual bleed rate (ABR) for all bleeds. The data showed that a single dose of fidanacogene elaparvovec resulted in a 71% reduction in ABR (p<0.0001), a 78% reduction in ABR post-treatment (p=0.0001), and a 92% reduction in the annualized infusion rate (p<0.0001). The company added that the safety profile of the therapy was consistent with phase 1/2 data, and the drug was well-tolerated. The company also noted that there were 14 serious adverse events in 7 patients (16%), of which 2 were treatment-related.

Pfizer licensed fidanacogene elaparvovec (also known as SPK-9001) from Spark Therapeutics in 2014. Swiss pharmaceutical giant Roche (RHHBY.US) acquired Spark for $4.8 billion in 2019.