FRONTIER1: a partially randomized phase 2 study assessing the safety, pharmacokinetics, and pharmacodynamics of Mim8, a factor VIIIa mimetic

Lentz, Steven R.; Chowdary, Pratima; Gil, Lidia; Lopez-Jaime, Francisco J.; Mahlangu, Johnny; Matytsina, Irina; Nielsen, Anne Louise; Windyga, Jerzy

doi:10.1016/j.jtha.2023.12.016

Journal of Thrombosis and Haemostasis

2024

DOI: 10.1016/j.jtha.2023.12.016

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FRONTIER1: a partially randomized phase 2 study assessing the safety, pharmacokinetics, and pharmacodynamics of Mim8, a factor VIIIa mimetic

Steven R. Lentz,

Pratima Chowdary,

Lidia Gil

et al.

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2024

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Cited by 6 publications

References 12 publications

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Advances in Development of Drug Treatment for Hemophilia with Inhibitors

Wichaiyo

2024

ACS Pharmacol. Transl. Sci.

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Patients with hemophilia A and B who have inhibitors face limited treatment options, because replacement therapy with clotting factor VIII or IX concentrates is ineffective, particularly for patients with high-titer inhibitors. Current mainstay therapies include immune tolerance induction (through frequent injections of clotting factor VIII or IX concentrates) to eradicate inhibitors and bypassing agents (such as recombinant activated clotting factor VII and activated prothrombin complex concentrates) for the prevention and treatment of bleeding episodes. The use of these agents typically requires intravenous injections and sometimes hospitalization, which can be burdensome for patients. More recently, emicizumab, a bispecific antibody that mimics the function of activated clotting factor VIII, has demonstrated favorable efficacy for prophylaxis in patients with hemophilia A and inhibitors, representing a promising new therapeutic strategy. Ongoing research aims to discover and develop easy-to-use nonfactor agents for managing hemophilia with inhibitors. This review summarizes the current understanding of the pathophysiology of inhibitor development in hemophilia, outlines existing treatment options, and discusses advancements in novel therapeutic biologics, including a recombinant activated clotting factor VII variant (marzeptacog alfa), a new bispecific antibody (Mim8), antitissue factor pathway inhibitor antibodies (concizumab and marstacimab), and small interfering RNA targeting antithrombin (fitusiran). All of these agents are administered subcutaneously, with some offering the convenience of less frequent dosing (e.g., weekly or monthly). These potential drug candidates may provide significant benefits for the prophylaxis or treatment of bleeding disorders in patients with hemophilia and inhibitors.

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Advances in Development of Drug Treatment for Hemophilia with Inhibitors

Wichaiyo

2024

ACS Pharmacol. Transl. Sci.

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Strategies for Performing Factor Assays in the Presence of Emicizumab or Other Novel/Emerging Hemostatic Agents

Kershaw

2024

Semin Thromb Hemost

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For several decades, therapeutic options for inherited deficiencies of factor VIII or IX (hemophilia A or B, respectively) have largely been the replacement of the missing clotting factor with plasma-derived or recombinant products. Hemostasis laboratories use standard activated partial thromboplastin time (aPTT)-based clotting or chromogenic assays to monitor plasma factor levels to guide therapy. The emergence in the past 10 years of extended half-life replacement products and other novel therapies for hemophilia has led to a reappraisal of assay suitability, with studies of product measurement showing some existing assay types or reagents to be unsuitable for some products. The hemostasis laboratory must adapt to the changing landscape by adding new assays or modifying existing assays to ensure accurate results for product measurement. These strategies include switching from a chromogenic assay to a clotting assay, or vice versa, changing an aPTT reagent brand, or introducing product specific calibrators. This article evaluates the effects of some of the newer treatment options on the laboratory testing of factor levels and related assays.

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Emicizumab and unmet needs of patients with hemophilia a who are managed with replacement therapies

Mahlangu

2024

Expert Review of Hematology

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FRONTIER1: a partially randomized phase 2 study assessing the safety, pharmacokinetics, and pharmacodynamics of Mim8, a factor VIIIa mimetic

Cited by 6 publications

References 12 publications

Advances in Development of Drug Treatment for Hemophilia with Inhibitors

Advances in Development of Drug Treatment for Hemophilia with Inhibitors

Strategies for Performing Factor Assays in the Presence of Emicizumab or Other Novel/Emerging Hemostatic Agents

Emicizumab and unmet needs of patients with hemophilia a who are managed with replacement therapies

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