Pfizer hemophilia therapy succeeds in late-stage trial

Pfizer (NYSE:PFE) announced Thursday that a late-stage trial for Hympavzi, its once-weekly injectable for bleeding disorder hemophilia, succeeded in a group of patients with antibodies that neutralize factor replacement therapies.

Citing topline data from its Phase 3 BASIS study, the New York-based pharma giant said Hympavzi led to a statistically significant and clinically relevant decline in annualized bleeding rate compared to on-demand therapy over 12 months, reaching the trial’s main goal.

The open-label trial enrolled adolescent and adult patients with hemophilia A and hemophilia B, and the readout focused on 48 hemophilia patients with inhibitors, which develop in about 20% and 3% with hemophilia A and hemophilia B, respectively.

Hemopavzi also outperformed the comparator in terms of all bleeding-related secondary endpoints, including spontaneous bleeds, joint bleeds, and total bleeds, the company said. As for safety, the experimental therapy was well tolerated, with no deaths or thromboembolic events.

Pfizer (NYSE:PFE) plans to share the results with healthcare authorities, targeting regulatory filings for the Hympavzi in hemophilia patients with inhibitors.