Bispecific antibody successfully reduced bleeds in haemophilia A

Woman with a nose bleed

Novo Nordisk has announced results from the Phase III FRONTIER2 trial of 254 adults and adolescents aged 12 years and over with haemophilia A, with and without inhibitors.

The trial assessed both once-weekly and once-monthly prophylactic treatment with the investigational treatment Mim8.

The data were presented at the International Society of Thrombosis and Haemostasis Annual Congress (ISTH 2024) in Bangkok, Thailand.

In the trial population with no prior prophylaxis treatment:

  • Mim8 demonstrated 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.
Restoring thrombin generation capacity

FRONTIER2 lead investigator Dr Maria Elisa Mancuso, Senior Consultant in Haematology, Centre for Thrombosis and Haemorrhagic Diseases, IRCCS Humanitas Research Hospital, Milan, Italy, said: “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.

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.

A similar bispecific antibody, emicizumab, manufactured by Genentech, was first granted Food and Drug Administration (FDA) approval for haemophilia A in 2017.

Diana Spencer, Senior Digital Content Editor, DDW

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