Marstacimab reduces bleeding in hemophilia A or B
Click Here to Manage Email Alerts
Marstacimab reduced annualized bleeding rate compared with prophylaxis and on-demand IV regimens for patients with hemophilia A or B, according to the agent’s manufacturer.
Marstacimab (PF-06741086, Pfizer) is an investigational anti-tissue factor pathway inhibitor. It targets the Kunitz 2 domain of tissue factor pathway inhibitor, a natural anticoagulation protein that prevents formation of blood clots.
Marstacimab reduced annualized bleeding rate compared with prophylaxis and on-demand IV regimens for patients with hemophilia A or B.
The FDA granted fast track designation to the agent for routine prophylaxis to prevent or reduce frequency of bleeding episodes among individuals with hemophilia A or hemophilia B with inhibitors.
The phase 3 BASIS study included adolescents or adults aged 12 to 74 years with severe hemophilia A or moderately severe to severe hemophilia B, with or without inhibitors.
The analysis included 116 study participants. Some received marstacimab during a 12-month period. Treatment consisted of a 300 mg subcutaneous loading dose of marstacimab, followed by 150 mg subcutaneously weekly, with potential for dose escalation to 300 mg once weekly.
Other study participants received a prophylaxis and on-demand IV regimen with Factor VIII or Factor IX, administered as part of usual care in the 6-month lead-in period.
Among patients treated with on-demand factor replacement IV therapy during the lead-in period, marstacimab conferred a 92% reduction in bleeds (P < .0001).
Researchers also reported a benefit with marstacimab compared with prophylaxis, noting a 35% reduction in annualized bleeding rate (P = .0376).
Treatment generally appeared well tolerated, according to a Pfizer-issued press release.
Investigators reported no deaths. No patients enrolled in trials of marstacimab have experienced thromboembolic events or events of consumptive coagulopathy.
Complete results of the BASIS study will be presented at a scientific conference.
“Despite significant treatment advances in recent years, many people living with hemophilia unfortunately continue to experience bleeding episodes and are required to manage their disease with frequent intravenous infusions,” Chris Boshoff, MD, PhD, chief development officer for oncology and rare diseases with Pfizer Global Product Development, said in the release. “These results support the potential for marstacimab to become the first once-weekly non-factor treatment for people with hemophilia B and a treatment option that helps address the diverse needs of patients with hemophilia A or B without inhibitors. These needs include preventing excessive or potentially life-threatening bleeds, while at the same time reducing the burden of treatment with once-weekly, subcutaneous administration.”
Read more about
Click Here to Manage Email Alerts