Novo Nordisk's Mim8 Effective and Safe for Children with Hemophilia A

In a recent announcement on February 7, 2025, Novo Nordisk revealed interim findings from the Phase 3 FRONTIER3 trial, focusing on children with haemophilia A both with and without inhibitors. This significant study involved 70 children aged between 1 and 11 years. Initially, the trial explored the efficacy of once-weekly prophylaxis using the investigational drug Mim8, with an option for participants to switch to a monthly regimen after 26 weeks. These promising results were shared at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD 2025) in Milan, Italy.

The interim results demonstrated that Mim8 was both effective and well-tolerated in the young participants. During the first part of the trial, the children received weekly subcutaneous injections of Mim8 over a 26-week period. As the study progressed into its second phase, participants were given the choice to continue with the weekly doses or switch to a monthly schedule for the next 26 weeks. The interim analysis focused on the findings from the end of the first part, with some preliminary insights from the second part also shared.

Notably, for those on the weekly regimen, the average annualised bleeding rate (ABR) for treated episodes was calculated at 0.53, with the median ABR being zero. Impressively, 74.3% of the children experienced no treated bleeds. More specifically, all participants with haemophilia A with inhibitors reported zero treated bleeds. Upon completing the initial segment of the study, 45% opted to transition to the monthly dosing schedule, while 55% continued with the weekly treatment.

Professor Johnny Mahlangu, the lead investigator and a respected figure in haemophilia research, highlighted the potential benefits of Mim8. He noted that the drug could simplify the management of haemophilia A in young children by reducing the treatment burden and facilitating a more normal lifestyle for families. These interim results suggest that Mim8 offers a flexible and effective dosing alternative that could significantly enhance the quality of life for children living with this condition and their families.

The study also presented data from patient and caregiver feedback, which underscored the potential of Mim8 to lessen the treatment burden and bolster physical function and quality of life in affected children. By the 26th week, a significant 98% of caregivers expressed a preference for Mim8 over previous treatments, with a substantial 73% indicating a strong preference. Additionally, there were noticeable improvements in both physical function and overall quality of life scores compared to the baseline.

Ludovic Helfgott, Executive Vice President for Rare Disease at Novo Nordisk, reinforced the company’s commitment to advancing treatment options that prioritize both safety and efficacy without compromising the quality of life. He expressed satisfaction that patients and caregivers have shown a clear preference for Mim8, reflecting its potential as a flexible treatment tailored to individual needs.

Looking ahead, Novo Nordisk anticipates submitting Mim8 for regulatory approval within the year. Further data from the ongoing Phase 3 FRONTIER program are expected to be presented at upcoming conferences and published throughout 2025 and 2026.

Haemophilia is a rare genetic disorder affecting the blood’s ability to clot, with haemophilia A being caused by a deficiency or defect in clotting Factor VIII. Novo Nordisk’s investigational drug, Mim8, aims to address this by mimicking Factor VIIIa through a bispecific antibody, thereby restoring normal clotting function. While Mim8 is still under investigation, the FRONTIER3 trial represents a significant step forward in the search for more effective treatments for haemophilia A.