Biologic could reduce treatment burden for children with haemophilia A

Interim results from a Phase III trial have demonstrated promising efficiency of Novo Nordisk’s Factor VIIIa (FVIIIa) mimetic bispecific antibody in children living with haemophilia A with and without inhibitors. The FRONTIER3 trial evaluating Mim8 enrolled a total of 70 children aged between one and 11 years old.

Key outcomes from the FRONTIER3 trial

Part one of the study involved participants receiving once-weekly doses of the treatment.

In this initial part of the study, findings showed a median annualised bleeding rate (ABR) of zero; 74.3 percent of participants had zero treated bleeds.

Furthermore, the estimated mean annualised bleeding rate for treated bleeds, was 0.53, Novo Nordisk reported.

“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,” commented 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.

“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,” stated Ludovic Helfgott, Executive Vice President for Rare Disease at Novo Nordisk.

The interim data from FRONTIER3 were presented at the 2025 Congress of the European Association for Haemophilia and Allied Disorders (EAHAD).

Advancing clinical development of Mim8

Earlier published results from the FRONTIER2 trial, part of Novo Nordisk’s Phase III programme, found that Mim8 provided “a 97 percent and 99 percent reduction in treated bleeds in patients with no prior prophylaxis treatment, respectively. This was compared to individuals not given prophylaxis treatment”, according to Novo Nordisk.

Regulatory submission for Mim8 is anticipated in 2025. Data from the ongoing Phase III FRONTIER programme will be announced in 2025 and 2026, shared Novo Nordisk.