Novo Nordisk’s haemophilia A therapy Mim8 stopped bleeding completely in 74.3% of children in a Phase III trial.

The FRONTIER3 trial (NCT05306418) achieved its co-primary endpoints, demonstrating a statistically significant and superior reduction of treated bleeding episodes with both once-weekly and once-monthly Mim8 versus no prophylaxis treatment and prior coagulation factor prophylaxis treatment in 70 children aged one to 11 years. The drug has also been evaluated in adults and adolescents.

Following the positive data, Novo Nordisk expects to make its Mim8 regulatory submission in 2025.

Haemophilia A is a rare blood disorder caused by missing or defective factor VIII (FVIII), a clotting protein, meaning a patient’s blood struggles to clot.

Mim8 is a bispecific monoclonal antibody that activates coagulation factor IX and factor X, in paediatric patients aged between one and 11 years, in two parts. In the first part, patients received once-weekly doses of Mim8 administered under the skin for 26 weeks. In part two, patients could continue with once-weekly dosing or move to once-monthly dosing for the remaining 26 weeks. Patients were able to stay on inhibitors during the FRONTIER3 study.

During part one, the mean average annualised bleeding rate (ABR) for treated bleeds was 0.53 while the median was 0. In total, 74.3% of patients suffered no treated bleeds while all children with haemophilia A with inhibitors 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 remained on the once-weekly dose. Parents and guardians of patients in the trial stated that they saw an improvement in the child’s quality of life (QoL), with 73% saying they ‘very strongly’ preferred Mim8 to prior treatment.

Ludovic Helfgott, executive vice president for rare disease at Novo Nordisk said: “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.”

In May 2024, the Danish pharma-giant announced data from the FRONTIER2 trial, a pivotal Phase IIIa study (NCT05053139), which showed that 86% of patients treated weekly, as well as 95% of those treated monthly, experienced no treated bleeds. This was in contrast to 0% of those without prophylaxis. The FRONTIER 2 trial evaluated Mim8 in adolescents and adults.

FRONTIER1 (NCT04204408) was a Phase I/II trial, which evaluated the safety tolerability pharmacokinetics and pharmacodynamics of Mim8.

Novo Nordisk not the only company in the game

The current standard of care (SOC) for paediatric haemophilia A patients is Factor VIII replacement, as well as Roche’s Hemlibra (emicizumab), which is a bispecific monoclonal antibody that restores the hemostatic process by bridging IXa and factor X. GlobalData predicts sales of Hemlibra to reach $5.53bn in 2030 while Novo Nordisk’s therapy is set to reach $926m in the same year.

GlobalData is the parent company of Clinical Trials Arena.

Another big name in the game is Sanofi announced data from a Phase III trial of its haemophilia A drug ALTUVIIIO (efanesoctocog alfa) in paediatric patients in March 2023. Sanofi’s trial (NCT04759131) reported a median ABR of 0 and a mean ABR of 0.61. The therapy is approved for adults and children in the US and Europe. GlobalData predicts sales of Sanofi’s therapy to reach £3.1bn in 2030.