A New Era in Hemophilia A Management with Clinical Insights on Mim8 – NEJM Group
NEJM Group shared a post on LinkedIn about a recent article by Alok Srivastava, published in NEJM, adding:
“In a new editorial, Alok Srivastava describes the scientific foundations and implications of the results of a randomized, controlled trial of Mim8, a new mimetic of activated factor VIII (factor VIIIa), to treat hemophilia A.
Hemophilia A is caused by inherited deficiency of clotting factor VIII and manifests predominantly with recurrent musculoskeletal bleeding. Its treatment was transformed approximately a decade ago through the introduction of a bispecific humanized monoclonal antibody, emicizumab.
This drug mimics factor VIIIa in that it increases the catalytic activity of factor IXa in activating factor X. (Factor IXa, on its own, is inefficient in activating factor X.) Factor Xa promotes thrombin generation and thus enhances clinical hemostasis in patients with hemophilia A (seen in figure).
The demonstration that emicizumab is effective in persons with hemophilia A with or without inhibitors (neutralizing antibodies to factor VIII) resulted in a shift from clotting factor concentrate – based therapy including bypass agents (which bypass the factor VIII – dependent step of the clotting cascade) for the 20 to 30% of patients in whom inhibitors develop after exposure to clotting factor concentrates. Indeed, emicizumab set a new benchmark of efficacy in the treatment of hemophilia A.
Its subcutaneous delivery, once a week to once a month, which provided stable plasma levels for sustained hemostasis without the peaks and troughs that occur with clotting factor concentrates, was sufficient to prevent all spontaneous bleeding in 60 to 70% of patients. Subsequent real-world data have confirmed these results.
In NEJM, Mancuso et al. present data from FRONTIER2, a phase 3a trial of Mim8, a fully human monoclonal antibody and a mimetic of factor VIIIa, to treat hemophilia A. The results support the primary objective: to confirm the efficacy of Mim8 for bleeding prevention.”
Title: A Second Factor VIIIa Mimetic for Hemophilia A
Author: Alok Srivastava
Title: Mim8 Bispecific Antibody Prophylaxis in Hemophilia A with or without Inhibitors
Authors: Maria Elisa Mancuso, Anthony K.C. Chan, Chandrakala Shanmukhaiah, Chuhl Joo Lyu, Joanna Zdziarska, Johnny Mahlangu, Lize F.D. van Vulpen, Pratima Chowdary, Renchi Yang, Steven R. Lentz, Tadashi Matsushita, Wan Hui Ong Clausen, Ilgiz Rakhmatullin, Johannes Oldenburg
Read the Full Article on NEJM
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