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Presentation Details
| Evaluating pen-injector handling and PROs in patients switching from emicizumab to Mim8 in FRONTIER5 Johnny Mahlangu1, Sanjay Ahuja2, Erin Cockrell3, Víctor Jiménez-Yuste4, Michele Schiavulli5, Amalie Rhode Høgh Nielsen6, Camila Martins Mazini Tavares6, Emily Waters7, Cédric Hermans8, Dan Johnson7. 1Department of Molecular Medicine and Haematology, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg, South Africa.2Indiana Hemophilia & Thrombosis Center, Innovative Hematology, Indianapolis, IN, USA.3Pediatric Hematology Oncology, Saint Joseph’s Children’s Hospital, Tampa, FL, USA.4Hematology Department, La Paz University Hospital, Hospital La Paz Institute for Health Research, Universidad Autónoma Madrid, Madrid, Spain.5Santobono-Pausilipon, Children’s Hospital A.O.R.N., Centro Regionale Pediatrico Malattie della coagulazione, Naples, Italy.6Novo Nordisk A/S, Søborg, Denmark.7Novo Nordisk Inc., Plainsboro, NJ, USA.8Division of Hematology, Cliniques universitaires Saint-Luc, Université catholique de Louvain (UCLouvain), Brussels, Belgium |
Abstract
Background: Mim8 (denecimig) is a next-generation, activated factor VIII mimetic, recombinant human bispecific IgG4 antibody in clinical development for subcutaneous prophylactic treatment of hemophilia A (HA) with/without inhibitors. FRONTIER5 (NCT05878938) is a phase 3b, open-label study evaluating the safety of switching directly from emicizumab to Mim8 prophylaxis using the Mim8 pen-injector (Figure 1). The pen-injector handling experience and patient preference were assessed as secondary endpoints, alongside other patient-reported outcomes (PROs). Objectives: This study investigated the pen-injector handling experience and preference of patients switching directly from emicizumab to Mim8 prophylaxis and, evaluated the change from baseline in participants’ treatment burden using PRO tools. Methods: The study enrolled male or female participants with HA of any severity with/without inhibitors, aged ≥12 years, and treated with emicizumab prophylaxis according to the label dose for ≥8 weeks before screening. Participants who switched directly from emicizumab to the Mim8 maintenance dose of the once-weekly (QW), once-every-2-weeks (Q2W), or once-monthly (QM) schedule were followed for 26 weeks, without a washout period or loading dose. Recorded PROs included the Hemophilia Device Handling and Preference Assessment (HDHPA) questionnaire (completed at Week 26), and the Hemophilia Treatment Experience Measure (Hemo-TEM), Pediatric Quality of Life Inventory (PedsQL) and Joint Pain Rating Scale (JPRS) (completed at baseline and Week 26). Results: Of 61 patients exposed to Mim8 (Table 1), 59 responded to the HDHPA questionnaire. Most participants, 98% (58/59), found the Mim8 pen-injector “very easy” or “easy” to use, and 95% found it “much easier” or “easier” compared with previous administration method. Most participants reported it “very easy” to inject the dose (83%). All participants (100%) were “extremely confident” or “very confident” in using the pen-injector correctly. Overall, 97% (57/59) preferred the pen-injector, with 97% of those participants reporting a “fairly strong” or “very strong” preference. Mean baseline PRO scores are reported in Table 1. Mean change from baseline [standard deviation] to Week 26 ranged from: –6.2 [9.6] to –3.5 [10.9] for Hemo-TEM scores, none of which were considered clinically relevant (≥8 points); –0.4 [1.4] to 0.6 [2.8] for JPRS; and –0.7 [8.7] to 3.6 [9.1] for PedsQL physical functioning. Conclusions: The HDHPA questionnaire results found the Mim8 pen-injector easy to use with an overall strong user preference over their previous injection system. At Week 26 of Mim8 prophylaxis, following a direct switch from emicizumab with no washout, there were no unexpected findings related to PROs. Previously presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress, Washington, DC, USA, 21-25 June 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.