Abstract
Summary
This is a summary of the results from a clinical study of treatment for people with severe hemophilia A or moderately severe to severe hemophilia B without inhibitors. Because severe hemophilia A and B predominantly affect men and boys, this study only included men and boys aged 12 to 74 years. The study was published in Blood.
People with hemophilia either have low amounts of clotting factors or are missing certain clotting factors in their blood. There are medicines that people with hemophilia can take to replace the missing clotting factor. These medicines must be injected into a vein and are usually given more than once a week. Marstacimab is an antibody that works by attaching to a protein in the blood called tissue factor pathway inhibitor (or TFPI). TFPI works separately from clotting factors. Marstacimab helps the balance between blood flowing freely and clotting. Marstacimab is given by a simple injection under the skin (not in a vein or muscle) and can be taken less often than clotting factors. Marstacimab may offer a new way to help people with hemophilia.
The study showed that men with hemophilia had fewer treated bleed events during 1 year with marstacimab treatment compared to previous factor replacement therapy. The researchers reported that marstacimab was safe and most side effects were mild to moderate.
These studies showed that marstacimab could help prevent bleeding in men with hemophilia A or B, without inhibitors.
Footnotes
Acknowledgements
Pfizer thanks all the people who took part in the study, their caregivers and supporters, and all study staff. Medical writing assistance was provided by Marion James, PhD and Jake Evans, PhD, Engage Scientific Solutions Ltd, and was funded by Pfizer Inc.
Ethics approval and consent to participate
Individuals were enrolled into the study following institutional review board or ethics committee approvals at each investigational site in accordance with Good Clinical Practice and the Declaration of Helsinki. All individuals provided written informed consent before enrolling in the research study.
Author contributions
Davide Matino, Andrew Palladino, Carrie Turich Taylor, Eunhee Hwang, Sangeeta Raje, Satyaprakash Nayak, Regina McDonald, Suchitra S. Acharya, Johnny Mahlangu, Victor Jimenez-Yuste, Nirmalkumar Choraria, Renchi Yang, Chi Kong Li, Murtadha Al-Khabori, Yasser Wali, Javier Morales Adrian, Young-Shil Park, O. Bülent Zülfikar, and John Teeter: conceptualization, writing – review and editing
Funding
This BASIS study was sponsored by Pfizer Inc.
Conflict of interest
Chi Kong Li has received honorarium from Amgen. Davide Matino has received research funding from Bayer, Pfizer, Novo Nordisk, Sanofi, Spark, Octapharma, and Roche; has received honoraria from Bayer, Pfizer, Novo Nordisk, Sanofi, Sobi, and Octapharma. Javier Morales Adrian has no disclosures to declare. Johnny Mahlangu has received research funding from BioMarin, CSL Behring, Novo Nordisk, Pfizer, Roche, Sanofi, and Spark; is a member of scientific advisory committees for BioMarin, Novo Nordisk, Roche, Takeda, Regeneron, Sanofi, and Spark; is a member of speakers’ bureaus for Novo Nordisk, Pfizer, Roche, Sanofi, Takeda. Murtadha Al-Khabori has no disclosures to declare. Nirmalkumar Choraria has no disclosures to declare. O. Bülent Zülfikar has received research funding from Sobi, Pfizer; serves on the speakers’ bureaus for Sanofi, Takeda, Pfizer, Roche, Genveon, Novo Nordisk, and CSL Behring. Renchi Yang has received honoraria or speaker fees from Novo Nordisk, Pfizer, Sanofi, and Takeda. Suchitra S. Acharya has received honoraria from Bayer and Pfizer. Victor Jiménez-Yuste has received grants or contracts from F. Hoffmann-La Roche Ltd, Novo Nordisk, SOBI, Takeda, Grifols, Bayer, Pfizer, Octapharma, and CSL Behring; received consulting fees and payment or honoraria for lectures, presentations, speakers’ bureau, manuscript writing, or educational events from F. Hoffmann-La Roche Ltd, Novo Nordisk, Sanofi, SOBI, Takeda, Grifols, Bayer, Pfizer, Spark Therapeutics, BioMarin, Octapharma, and CSL Behring. Yasser Wali has research grants from Pfizer, Novo Nordisk, and Novartis. Young-Shil Park reports research support or participation as a principal investigator from Pfizer, BioMarin Pharmaceutical Inc., CSL Behring, Novo Nordisk, Sanofi, Takeda, and Chugai. Andrew Palladino, Carrie Turich Taylor, Sangeeta Raje, and Satyaprakash Nayak are employees and stockholders of Pfizer. Eunhee Hwang, Regina McDonald, and John Teeter were employees and stockholders of Pfizer at the time of study conduct.
Availability of data and material
Upon request, and subject to review, Pfizer will provide the data that support the findings of this study. Subject to certain criteria, conditions, and exceptions, Pfizer may also provide access to the related individual de-identified participant data.