Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B

Summary

Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently than factor replacement products and will work in the presence of inhibitors. The potential for once weekly (QW) subcutaneous (SC) administration provides for treatment options in the absence of reliable vascular access, increased convenience and may enable better compliance. Combined, these qualities should result in a reduction of bleeding episodes.

Timeline

Start

2020-03-09

Primary completion

2025-04-29

Completion

2025-04-29

Outcome

Met primary endpoint

release “the pivotal Phase 3 BASIS clinical trial ( NCT03938792 ) evaluating marstacimab has met its primary endpoints” pfizer.com ↗

release “NCT03938792 ) evaluating HYMPAVZI™ (marstacimab) for adults and adolescents living with hemophilia A or B with inhibitors. The study met the primary endpoint” pfizer.com ↗

Publications

• Matino D, Acharya SS, Taylor CT, Sun P, Agathon D, Raje S, Gould T, Palladino A, Mahlangu J, Lissitchkov T, Todorova M, Chan A, Carcao M, Sun J, Yang R, Wu R, Jin C, Zeng X, Boban A, Bilic E, Frenzel L, Fung Chan GC, Kong Li C, Apte S, Choraria N, Chistolini A, Marchesini E, Peyvandi F, Fujii T, Matsushita T, Kaneda M, Lyu CJ, Park YS, Kim S, Martinez LV, Adrian JM, Wali Y, Al Khabori M, Kurtov I, Alzahrani H, Zaher G, Micic D, Miljic P, Kostic G, Vucic M, Djurdjevic P, Hidalgo OB, Porras JG, Jimenez-Yuste V, Calvo Villas JM, Lopez Fernandez MF, Albayrak C, Okan V, Balkan C, Sahin F, Antmen A, Unal E, Visweshwar N, Sharathkumar A, Kruse-Jarres R. Efficacy and safety of marstacimab prophylaxis in hemophilia A/B with inhibitors: results from the phase 3 BASIS trial. Blood. 2026 Feb 26;147(9):920-931. doi: 10.1182/blood.2025031065.
• Matino D, Palladino A, Taylor CT, Hwang E, Raje S, Nayak S, McDonald R, Acharya SS, Mahlangu J, Jimenez-Yuste V, Choraria N, Yang R, Li CK, Al-Khabori M, Wali Y, Morales Adrian J, Park YS, Zulfikar OB, Teeter J. Marstacimab prophylaxis in hemophilia A/B without inhibitors: results from the phase 3 BASIS trial. Blood. 2025 Oct 2;146(14):1654-1663. doi: 10.1182/blood.2024027468.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Marstacimab	Monoclonal antibody	150 mg	Subcutaneous
Subject	Marstacimab	Monoclonal antibody	300 mg	Subcutaneous

Indications