Pfizer’s hympavzi shows positive topline Phase 3 results in hemophilia A or B

Pfizer (PFE) announced positive topline results from the Phase 3 BASIS study evaluating hympavzi – marstacimab – for adults and adolescents living with hemophilia A or B with inhibitors. The study met the primary endpoint and key secondary bleeding endpoints demonstrating the superiority of once-weekly subcutaneous hympavzi in improving key bleeding outcomes compared to on-demand treatment in a patient population where less burdensome treatment approaches are needed. Of the more than 800,000 people in the world living with hemophilia A or hemophilia B, approximately 20% of people with hemophilia A and 3% of people with hemophilia B are unable to continue taking factor replacement therapies because they develop inhibitors to Factors VIII and IX and these therapies no longer prevent or stop bleeding episodes. Hympavzi was superior to on-demand treatment with a 93% reduction in ABR over 12 months. Hympavzi was generally well-tolerated, consistent with the non-inhibitor cohort of the BASIS study and Phase 1/2 results. No deaths or thromboembolic events were reported. Additional data will be presented at upcoming medical meetings. Pfizer plans to discuss these data with regulatory authorities, with the goal of initiating regulatory filings for hympavzi for the treatment of patients living with hemophilia with inhibitors.