Red blood cells, platelets and fibrin fibers are clustered together in a clot (shown above). Patients with hemophilia have deficient levels of a clotting factor needed to stop bleeding.
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Pursuing Scientific Breakthroughs for Hematological Diseases

Red blood cells, platelets and fibrin fibers are clustered together in a clot (shown above). Patients with hemophilia have deficient levels of a clotting factor needed to stop bleeding.

In our efforts to change patients’ lives, Pfizer focuses on delivering breakthrough science to areas of unmet patient needs. One such area is in hematology. With internal expertise in gene therapy and antibody-based therapies, combined with strategic collaborations, Pfizer is working to provide treatment options for diseases like hemophilia A and B and sickle cell disease (SCD). For example, marstacimab, our anti-tissue factor pathway inhibitor, is a novel monoclonal antibody antagonist and a potential therapy for patients with eitherhemophilia A or B, including those with inhibitors, that may help reduce bleeding episodes by promoting coagulation activity.

Following positive Phase 1 / 2 clinical trial results, we have begun dosing patients in our Phase 3 trial with fidanacogene elaparvovec, our investigational gene therapy for the treatment of hemophilia B. We are also enrolling patients in our Phase 3 hemophilia A gene therapy lead-in trial, which we developed through Phase 2 in partnership withSangamo.

Pfizer is committed to building on our broad hematology expertise; although our investigational candidate for SCD, rivipansel, failed to meet its Phase 3 endpoints, we are continuing to pursue discovery research to identify potential compounds to treat SCD. Additionally, we have conducted a non-interventional studyto better understand both the patient experience with and biomarkers of SCD. Through partnerships with patients, advocacy communities and fellow scientists, our hope is that research will enable options for patients with hematological diseases using multiple modalities.

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