Novo Nordisk has reported positive results from the ongoing Phase III FRONTIER4 extension trial, assessing the long-term safety and efficacy of investigational denecimig (Mim8) in patients with haemophilia A.
The open-label trial is evaluating denecimig as a subcutaneous prophylactic treatment in children, adolescents, and adults with or without inhibitors, with dosing regimens that include once-weekly, once-every-two-weeks, and once-monthly administration.
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The interim analysis included 426 participants aged one year and above, comprising 365 adults and adolescents with a median follow-up of 0.50 years and 61 children with a median follow-up of 0.33 years.
Safety assessment indicated that injection-site reactions occurred in 2% of injections for children and 1.8% for adolescents and adults, with all cases reported as mild and transient. No clinical evidence of neutralising antibodies was observed.
Estimated mean annualised bleeding rates (ABRs) were 0.75 for adults and adolescents and 0.37 for children, regardless of inhibitor status or dosing schedule.
Zero treated bleeds were reported in 71% of adults and adolescents, and 89% of children on denecimig prophylaxis.
Exploratory patient-reported outcomes suggested maintained improvements in joint pain and reduced treatment burden across all dosing frequencies.
Among 185 participants, 94.1% found the denecimig pen-injector easy or very easy to use, while 89.7% described the injection process as quick or very quick.
Additional post hoc analyses from other trials indicated that denecimig increased thrombin generation to normal ranges in adolescents and adults without excessive response.
Novo Nordisk submitted denecimig to the US Food and Drug Administration (FDA) through a biologics licence application in September 2025.
Novo Nordisk chief scientific officer and research and development executive vice-president Dr Martin Holst Lange said: “The positive safety and efficacy findings from FRONTIER4 reinforce denecimig’s potential as a preventive treatment option for children, adolescents, and adults with haemophilia A, regardless of their inhibitor status or the dosing frequency used.”
Last month, Novo Nordisk reported results from its Phase II study of zenagamtide, an investigational once-weekly subcutaneous treatment, showing statistically significant reductions in blood sugar and body weight among adults with type 2 diabetes compared to placebo.
