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Presentation Details
| From Efficacy to Experience: 52-Week Patient-Reported Outcomes with Mim8 in Adolescents and Adults with Hemophilia A with or without Inhibitors (FRONTIER2) Cédric Hermans1, Sanjay Ahuja2, Hazza Alzahrani3, Cihan Ay4, Peter Kampmann5, Samya Obaji6, Sophie Susen7, Ciprian Ionut Tomuleasa8, Lize van Vulpen9, Amalie Rhode Høgh Nielsen10, Ilgiz Rakhmatullin10, Emily Waters11, Carmen Escuriola-Ettingshausen12, Arjun Menon11. 1Division of Hematology, Cliniques universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium.2Department of Pediatric Hematology and Oncology, Rainbow Babies and Children’s Hospital, Cleveland, OH, USA.3Department of Hematology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia.4Department of Medicine I, Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria.5Department of Hematology, Rigshospitalet, Copenhagen, Denmark.6Department of Haematology, Cardiff and Vale University Health Board, Cardiff, United Kingdom.7Hemostasis and Transfusion Department, University of Lille, Lille University Hospital, Lille, France.8Department of Hematology, “Ion Chiricuta” Institute of Oncology, Cluj-Napoca, Rumania.9Center for Benign Haematology, Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands.10Novo Nordisk A/S, Søborg, Denmark.11Novo Nordisk, Inc., Plainsboro, NJ, USA.12Haemophilia Centre Rhein Main (HZRM), Frankfurt, Germany |
Abstract
Background: Mim8 is a bispecific antibody, activated factor VIII mimetic in development for subcutaneous prophylaxis (PPX) in hemophilia A (HA) with/without inhibitors. In the phase 3 FRONTIER2 study (NCT05053139) once weekly (QW) and once monthly (QM) Mim8 dosages significantly reduced annualized bleeding rate for treated bleeds and improved patient-reported outcomes (PROs) versus continued on-demand treatment or previous clotting factor concentrate (CFC) PPX at 26 weeks. Objectives: Assess PROs with Mim8 PPX over 52 weeks. Methods: FRONTIER2 is an open-label, randomized trial in participants aged ≥12 years with HA, with or without inhibitors. Participants previously receiving on-demand treatment were randomized to Mim8 PPX QW (Arm 2a) or QM (Arm 2b) for 52 weeks or continued on-demand treatment (Arm 1) for 26 weeks then switch to Mim8 QW or QM for another 26 weeks. Participants on prior CFC PPX were randomized to Mim8 QW (Arm 3) or QM (Arm 4) for 52 weeks. PROs assessed were Hemophilia Treatment Experience Measure (Hemo-TEM; lower score indicates lower burden, ≥8-point reduction defined as clinically meaningful improvement), physical functioning domain of the Pediatric Quality of Life Inventory (PedsQL; higher score indicates better functioning), Joint Pain Rating Scale (JPRS; lower score indicates less pain), Patient Global Impression of Change (PGI-C) for pain and physical function and Hemophilia Patient Preference Questionnaire (HPPQ). Changes were calculated from the start of Mim8 PPX (Week 0 for Arms 2a/2b/3/4; Week 26 for Arm 1). Results: QW and QM dosing produced similar PRO outcomes; therefore, results were pooled by previous treatment (Arm 1, Arms 2a/2b, Arms 3/4). This report includes 27 patients from China absent from the prior 26-week abstract. Mean changes from baseline Hemo-TEM total scores were –16.0 in Arm 1 (n=13), –12.7 in Arms 2a/2b (n=29), and –9.7 in Arms 3/4 (n=182). Clinically meaningful improvement was achieved by 8/13 (62%), 16/29 (55%), and 84/182 (46%), respectively. A “fairly strong” or “very strong” preference for Mim8 over previous treatment was reported by 15/17 (88%) of participants in Arm 1, 39/40 (98%) in Arms 2a/2b, and 175/192 (91%) in Arms 3/4, as assessed by HPPQ. Mean PedsQL physical function score changes were +13.0 in Arm 1 (n=13), +17.6 in Arms 2a/2b (n=29), and +4.0 in Arms 3/4 (n=167). Improvements in PGI-C physical function were reported by 17/17 (100%) of participants in Arm 1, 36/40 (90%) in Arms 2a/2b, and 138/192 (72%) in Arms 3/4. Mean changes in JPRS joint pain scores were –1.8 in Arm 1 (n=13), –1.5 in Arms 2a/2b (n=29), and –0.4 in Arms 3/4 (n=184). Improvements in PGI-C pain intensity were reported by 15/17 (88%) of participants in Arm 1, 38/40 (95%) in Arms 2a/2b, and 115/192 (60%) in Arms 3/4. Conclusions: Over 52 weeks, Mim8 QW and QM PPX provided consistent improvements in all PROs in adults and adolescents with HA, with or without inhibitors. These results support the long-term benefit of Mim8 in HA treatment. Previously presented at ASH 2025, Orlando, Florida, 6–9 December 2025.
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No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.