Novo Nordisk A/S: Mim8 demonstrated superior reductions in annualised bleeding rate (ABR) compared to on-demand and prior prophylaxis treatment in people with haemophilia A

Novo Nordisk announced results from the phase 3 FRONTIER2 trial of 254 adults and adolescents aged 12 years and over with haemophilia A, with and without inhibitors,  showing a superior reduction in the estimated mean annualised bleeding rate (ABR) of treated bleeds by 97.1% with once-weekly and 98.7% with once-monthly Mim8.

Once-weekly and once-monthly Mim8 prophylaxis demonstrated a superior reduction in treated bleeds, as evidenced by an estimated mean ABR of 0.45 and 0.20 bleeds per patient-year, respectively, compared to an estimated mean ABR of 15.75 for those who received no prior prophylaxis treatment.

Zero bleeds were observed in 85.7% of people treated with once-weekly Mim8 and 95.0% of those treated with once-monthly Mim8. In the trial population with prior coagulation factor prophylaxis: Mim8 demonstrated a superior reduction in the estimated mean ABR by 48% for once-weekly and 42.6% for once-monthly treatment.

Once-weekly and once-monthly Mim8 demonstrated a superior reduction in treated bleeds compared to prior coagulation factor prophylaxis. Estimated mean ABRs for treated bleeds were 2.51 bleeds per patient-year for once-weekly Mim8 (versus 4.83 on prior clotting factor prophylaxis) and 1.78 bleeds per patient-year for once-monthly Mim8 (versus 3.10 on prior clotting factor prophylaxis).

Zero bleeds were observed in 66.3% of participants treated with once-weekly Mim8 and 65.3% treated with once-monthly Mim8.

“Despite treatment advances, there are still many unmet needs for people living with haemophilia A,” said FRONTIER2 lead investigator Dr Maria Elisa Mancuso, Senior Consultant in Haematology, Centre for Thrombosis and Haemorrhagic Diseases, IRCCS Humanitas Research Hospital, Milan, Italy. “The FRONTIER2 data demonstrated superiority of Mim8 prophylaxis over both on demand treatment and prior clotting factor prophylaxis with respect to the number of treated bleeding episodes in people living with haemophilia A, regardless of their inhibitor status. It is also encouraging to see that in some subgroups in the study, up to 95% of people treated with Mim8 experienced zero bleeds.”

Mim8 is a next-generation Factor VIIIa (FVIIIa) mimetic bispecific antibody designed with the potential to deliver sustained haemostasis. It is currently in development for once-weekly or once-monthly prophylaxis treatment for people living with haemophilia A, with and without inhibitors. Administered subcutaneously, Mim8 bridges Factor IXa/X (FIXa/FX), thereby replacing missing FVIII.

This restores the body’s thrombin generation capacity, helping blood to clot.1 In this trial, Mim8 was well-tolerated, and no safety concerns (thromboembolic events or related serious adverse events) were observed, in line with previous findings.

“The FRONTIER2 efficacy and safety data is very encouraging,” said Martin Holst Lange, Executive Vice President of Development at Novo Nordisk. “With Mim8, we have the potential of offering a substantial proportion of patients the prospect of zero bleeds and convenient dosing flexibility to fit their lifestyles and needs. We look forward to discussing these data with regulatory authorities.”

Contingent on regulatory interactions, Novo Nordisk aims to submit Mim8 for the first regulatory approval towards the end of 2024. Data from the ongoing phase 3 FRONTIER programme will continue to be disclosed at upcoming congresses and in publications in 2024 and 2025.novononovo nordisk

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