Pfizer’s Growing Hematology Pipeline Matures With New Hemophilia, Sickle Cell Data

Multiple Modalities Advance Across Non-Malignant Diseases

The big pharma is building on its hematology legacy beyond coagulation factors with marstacimab and gene therapies for hemophilia, plus oral and injectable medicines for sickle cell disease.

3D rendering of red blood cells
• Source: Shutterstock

Pfizer Inc. has a long history in non-malignant hematology after bringing forward multiple coagulation factor replacement therapies for hemophilia, but the company has a renewed and growing presence in the space after investing in gene therapies for hemophilia, buying sickle cell drug developer Global Blood Therapeutics, Inc. and advancing additional treatments for both diseases.

The company presented results at the American Society of Hematology (ASH) meeting on 9 December from the Phase III BASIS clinical trial of its anti-tissue factor pathway inhibitor (TFPI) inhibitor marstacimab in hemophilia A and B as well as Phase II results from a Phase II/III trial of its sickle hemoglobin (HbS) polymerization inhibitor GBT021601 (GBT601)

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