MIM8 PROPHYLAXIS IN ADULTS AND ADOLESCENTS WITH HAEMOPHILIA A WITH OR WITHOUT INHIBITORS: IN-TERIM RESULTS FROM THE FRONTIER4 LONG-TERM SAFETY AND EFFICACY STUDY
8 affiliations
- 1Institute of Hematology and Transfusion Medicine, Poland
- 2Novo Nordisk, Spain
- 3University Clinic Bon, Germany
- 4IRCCS Humanitas Research Hospital, Italy
- 5McMaster University, Canada
- 6Novo Nordisk, Denmark
- 7Nagoya University Hospital, Japan
- 8Novo Nordisk, Rio de Janeiro, Brazil
Abstract
Background: Mim8 (denecimig) is a next-generation, FVIIIa mimetic intended for subcutaneous prophylaxis of haemophilia A with or without FVIII inhibitors (HAwI/HA). Previous FRONTIER studies found Mim8 efficacious, well-tolerated, and without safety concerns. Aims: Investigate long-term safety and efficacy across Mim8 dosing frequencies in adolescents and adults with HAwI/HA. Methods: Ongoing FRONTIER4 (NCT05685238) enrolled participants from completed Mim8 phase-2/3 studies. Participants aged ≥12 years received Mim8 once-every-week (QW), once-every-two-weeks (Q2W) or once-every-month (QM). Primary endpoints were number of adverse events (AEs), serious AEs, and unintended medical events. Secondary endpoints included injec-tion site reactions (ISRs), anti-Mim8 antibodies, treated bleeding episodes, and annualised bleeding rates (ABRs). Bleeds were analysed using a negative binomial regression model. The study followed Good Clinical Practice and the Declaration of Helsinki, was approved by local eth-ics committees, and participants provided written informed consent. Results: Of 365 participants, 24.4% were aged 12-17 years, 11% had HAwI, and 87.1% severe haemophilia. Mean (SD) age was 33 (16) years and 98.6% were male. Participants entered FRON-TIER4 with a 12-month median ABR of 0.58 and mean (SD) Mim8 exposure of 1.17 (0.30) years. Over mean (SD) treatment analysis period of 0.57 years, no thromboembolic events were re-ported (Table 1). Of the serious AEs, 1 fatal outcome was unlikely related to Mim8. Of 6138 injec-tions, 1.8% resulted in ISRs and 0.4% in pain and/or burning. All ISRs were mild and transient. Anti-Mim8 antibody incidence was 4.7% with no clinical evidence of neutralising effect. Across QW, Q2W and QM, estimated mean ABRs (95%CI) were 0.91 (0.66-1.27), 0.55 (0.34-0.87) and 0.74 (0.54-1.02), respectively, and participants with zero treated-bleed rates (95%CI) were 70.4% (61.9-77.9), 80.8% (71.7-88.0), and 70.9% (63.5-77.5), respectively. ABRs across bleed subtypes were low, and most participants had zero treated bleeds (Figure 1). ABRs (95%CI) for HA and HAwI participants were 0.88 (0.71-1.08) and 0.15 (0.05-0.45), respectively, across dosing frequencies. Conclusion: Interim analyses from FRONTIER4 supports long-term safety and efficacy of Mim8 prophylaxis in adults and adolescents with consistently low ABRs across dosing frequen-cies and inhibitor status.
Related
- CONCIZUMAB PROPHYLAXIS IN PAEDIATRIC PARTICIPANTS WITH HAEMOPHILIA A/B WITH INHIBITORS IN THE PHASE 3 EXPLORER10 STUDY: EFFICACY, SAFETY AND PK/PD RESULTS FROM THE 32-WEEK CUT-OFF
- PHASE 3 STUDY INVESTIGATING THE EFFICACY AND SAFETY OF FITUSIRAN PROPHYLAXIS IN MALE PARTICIPANTS AGED 1 TO <12 YEARS WITH HEMOPHILIA A/B, WITH OR WITHOUT INHIBITORS: ATLAS-KIDS, A TRIAL IN PROGRESS IN BRAZIL
- CO002 | RETROSPECTIVE LONGITUDINAL ASSESSMENT OF JOINT HEALTH AND QUALITY OF LIFE IN ADOLESCENT AND ADULT PATIENTS AFFECTED BY MODERATE OR SEVERE HAEMOPHILIA A WITH OR WITHOUT INHIBITOR SWITCHING FROM INTRAVENOUS FVIII TREATMENT TO SUBCUTANEOUS PROPHYLAXIS WITH EMICIZUMAB
- Oral Health Status, Salivary Characteristics, and Cariogenic Bacterial Levels in Children and Adolescents with Haemophilia
- The use of triptans, opioids, and other rescue medications among patients with migraine prescribed calcitonin gene–related peptide inhibitors for migraine prophylaxis
BibTeX
@article{windyga2026mim8,
title = {{MIM8 PROPHYLAXIS IN ADULTS AND ADOLESCENTS WITH HAEMOPHILIA A WITH OR WITHOUT INHIBITORS: IN-TERIM RESULTS FROM THE FRONTIER4 LONG-TERM SAFETY AND EFFICACY STUDY}},
author = {Jerzy WINDYGA and Llenalia María García FERNÁNDEZ and Johannes OLDENBURG and Maria Elisa MANCUSO and Davide MATINO and Ilgiz RAKHMATULLIN and Tadashi MATSUSHITA and Vanessa Maira Lima CAMPOS},
journal = {Hematology, Transfusion and Cell Therapy},
year = {2026},
month = oct,
doi = {10.1016/j.htct.2026.108030},
url = {https://doi.org/10.1016/j.htct.2026.108030},
note = {CC BY},
}
RIS
TY - JOUR
TI - MIM8 PROPHYLAXIS IN ADULTS AND ADOLESCENTS WITH HAEMOPHILIA A WITH OR WITHOUT INHIBITORS: IN-TERIM RESULTS FROM THE FRONTIER4 LONG-TERM SAFETY AND EFFICACY STUDY
AU - Jerzy WINDYGA
AU - Llenalia María García FERNÁNDEZ
AU - Johannes OLDENBURG
AU - Maria Elisa MANCUSO
AU - Davide MATINO
AU - Ilgiz RAKHMATULLIN
AU - Tadashi MATSUSHITA
AU - Vanessa Maira Lima CAMPOS
PY - 2026
DA - 2026/10/23
JO - Hematology, Transfusion and Cell Therapy
DO - 10.1016/j.htct.2026.108030
UR - https://doi.org/10.1016/j.htct.2026.108030
AB - Background: Mim8 (denecimig) is a next-generation, FVIIIa mimetic intended for subcutaneous prophylaxis of haemophilia A with or without FVIII inhibitors (HAwI/HA). Previous FRONTIER studies found Mim8 efficacious, well-tolerated, and without safety concerns. Aims: Investigate long-term safety and efficacy across Mim8 dosing frequencies in adolescents and adults with HAwI/HA. Methods: Ongoing FRONTIER4 (NCT05685238) enrolled participants from completed Mim8 phase-2/3 studies. Participants aged ≥12 years received Mim8 once-every-week (QW), once-every-two-weeks (Q2W) or once-every-month (QM). Primary endpoints were number of adverse events (AEs), serious AEs, and unintended medical events. Secondary endpoints included injec-tion site reactions (ISRs), anti-Mim8 antibodies, treated bleeding episodes, and annualised bleeding rates (ABRs). Bleeds were analysed using a negative binomial regression model. The study followed Good Clinical Practice and the Declaration of Helsinki, was approved by local eth-ics committees, and participants provided written informed consent. Results: Of 365 participants, 24.4% were aged 12-17 years, 11% had HAwI, and 87.1% severe haemophilia. Mean (SD) age was 33 (16) years and 98.6% were male. Participants entered FRON-TIER4 with a 12-month median ABR of 0.58 and mean (SD) Mim8 exposure of 1.17 (0.30) years. Over mean (SD) treatment analysis period of 0.57 years, no thromboembolic events were re-ported (Table 1). Of the serious AEs, 1 fatal outcome was unlikely related to Mim8. Of 6138 injec-tions, 1.8% resulted in ISRs and 0.4% in pain and/or burning. All ISRs were mild and transient. Anti-Mim8 antibody incidence was 4.7% with no clinical evidence of neutralising effect. Across QW, Q2W and QM, estimated mean ABRs (95%CI) were 0.91 (0.66-1.27), 0.55 (0.34-0.87) and 0.74 (0.54-1.02), respectively, and participants with zero treated-bleed rates (95%CI) were 70.4% (61.9-77.9), 80.8% (71.7-88.0), and 70.9% (63.5-77.5), respectively. ABRs across bleed subtypes were low, and most participants had zero treated bleeds (Figure 1). ABRs (95%CI) for HA and HAwI participants were 0.88 (0.71-1.08) and 0.15 (0.05-0.45), respectively, across dosing frequencies. Conclusion: Interim analyses from FRONTIER4 supports long-term safety and efficacy of Mim8 prophylaxis in adults and adolescents with consistently low ABRs across dosing frequen-cies and inhibitor status.
SN - 2531-1379
SN - 2531-1387
ER -