Phase 3 FRONTIER3 interim analysis data presented at EAHAD 2025 showed that 74.3% of children on once-weekly Mim8 prophylaxis experienced zero treated bleeds
February 7, 2025 – Clinical Trials, Drug Discovery, Other, Pharmaceutical – European Association for Haemophilia and Allied Disorders, Novo Nordisk, clinical trials, haemophilia a
7 February 2025 — Bagsværd, Denmark — Novo Nordisk today announced interim results from the phase 3 FRONTIER3 trial of 70 children (aged 1-11 years old) with haemophilia A with and without inhibitors. The trial initially assessed once-weekly prophylaxis treatment (regular treatment to prevent prolonged and spontaneous bleeding) with investigational Mim8 before giving participants the option to change to once-monthly dosing after 26 weeks. The data were presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD 2025) in Milan, Italy.
The data showed that Mim8 was well-tolerated and efficacious in children with haemophilia A with and without inhibitors. In part one of the FRONTIER3 study, participants with haemophilia A received once-weekly doses of Mim8 administered under the skin for 26 weeks. In part two, participants had the option to continue with once-weekly dosing or move to once-monthly dosing for the remaining 26 weeks. The interim analysis reports on results at the completion of part one of the study, with some preliminary data shared from part two, which continued following this analysis.
For part one of the study in children on once-weekly prophylaxis, the estimated mean (average) annualised bleeding rate (ABR) for treated bleeds was 0.53. The median (middle or central value in the data set) ABR was zero; 74.3% of participants had zero treated bleeds. All children with haemophilia A with inhibitors (n=14) reported zero treated bleeds. After completing the initial 26 weeks of the study, 45% of participants chose to move to once-monthly Mim8, and the rest (55%) remained on the once-weekly dose.
“Managing haemophilia A in young children can be a complex balancing act of delivering ongoing care, minimising time out of school, and ensuring their physical, emotional and social wellbeing is optimised,” said Professor Johnny Mahlangu, lead investigator and Director of the Haemophilia Comprehensive Care Centre at Charlotte Maxeke Johannesburg Hospital at University of the Witwatersrand in Johannesburg, South Africa. “The FRONTIER3 interim analysis data are encouraging for families with young children and indicate that Mim8 could offer an efficacious, convenient, flexible dosing option for children, helping to reduce the treatment burden so families can live more normal lives.”
Patient- and caregiver-reported outcomes data from part one of the FRONTIER3 trial with once-weekly Mim8 were also presented, which indicated that Mim8 may reduce treatment burden and improve physical function and quality of life (QoL) in children living with haemophilia A with or without inhibitors. At week 26, 98% of caregivers said they preferred Mim8 to prior treatment, of which 73% said they ‘very strongly’ preferred Mim8 to prior treatment. Additionally, at week 26, positive improvement trends were observed in the mean physical function score (a measure of a child’s ability to perform everyday physical activities such as running) and mean QoL total score, compared to baseline.
“We see the daily challenges faced by children living with haemophilia A and their caregivers. The FRONTIER3 data represent another step forward in our ambition to provide treatment options that place equal focus on safety and efficacy without requiring a compromise on treatment administration and quality of life,” said Ludovic Helfgott, executive vice president for Rare Disease at Novo Nordisk. “Mim8 is designed with the aim to offer treatment flexibility based on individual lifestyles, so it is encouraging to see that patients and caregivers in these analyses prefer Mim8 over their previous treatment. At Novo Nordisk, our commitment to the rare bleeding disorders community compels us to reduce limitations in the lives of children with haemophilia and their families.”
Novo Nordisk expects Mim8 regulatory submission during 2025. Data from the ongoing phase 3 FRONTIER programme will be disclosed at upcoming congresses and in publications in 2025 and 2026.
About Novo Nordisk
Novo Nordisk is a leading global healthcare company founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases, built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 76,300 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com
