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.<\/p>\n
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.<\/p>\n
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.<\/p>\n
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.<\/p>\n
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.<\/p>\n
Zero treated bleeds were reported in 71% of adults and adolescents, and 89% of children on denecimig prophylaxis.<\/p>\n
Exploratory patient-reported outcomes suggested maintained improvements in joint pain and reduced treatment burden across all dosing frequencies.<\/p>\n
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.<\/p>\n
Additional post hoc analyses from other trials indicated that denecimig increased thrombin generation to normal ranges in adolescents and adults without excessive response.<\/p>\n
Novo Nordisk submitted denecimig to the US Food and Drug Administration (FDA) through a biologics licence application in September 2025.<\/p>\n
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.”<\/p>\n