Novo Nordisk Reports Positive Mim8 Results in Late-Stage Haemophilia A Trial

Novo Nordisk has revealed promising outcomes from a phase 3 study of its haemophilia A treatment, Mim8, targeting adults and adolescents aged 12 and above. Haemophilia A, a rare inherited bleeding disorder, results from a missing or defective clotting factor VIII and constitutes up to 85% of the global haemophilia cases, which total approximately 1,125,000.

The FRONTIER 2 trial has been investigating the safety and effectiveness of once-weekly and once-monthly doses of Mim8, a subcutaneous therapy designed to prevent prolonged and spontaneous bleeding. The study includes patients both with and without inhibitors. Inhibitors are immune system responses to the clotting factors in replacement therapy that can render treatment ineffective, affecting up to 30% of haemophilia A patients.

At the International Society of Thrombosis and Haemostasis Annual Congress this year, Novo Nordisk presented the trial results showing that Mim8 significantly reduced the estimated mean annualised bleeding rate (ABR) of treated bleeds. For participants who had no prior prophylaxis treatment, there was a 97.1% reduction with once-weekly Mim8 and a 98.7% reduction with once-monthly Mim8. Additionally, zero bleeds were reported in 85.7% of those on the once-weekly regimen and in 95% of individuals receiving the once-monthly dose.

For participants previously on coagulation factor prophylaxis, Mim8 also demonstrated substantial benefits. The estimated mean ABR was reduced by 48% with once-weekly treatment and by 42.6% with once-monthly treatment. Furthermore, the study showed a superior reduction in treated bleeds compared to prior coagulation factor prophylaxis. Zero bleeds were observed in 66.3% of patients treated weekly and in 65.3% of those on the monthly regimen.

Novo Nordisk is planning to seek the first regulatory approval for Mim8 by the end of this year. Martin Holst Lange, executive vice president of development at Novo Nordisk, expressed great optimism about the findings, noting that the data is "very encouraging." He emphasized that Mim8 could offer a significant proportion of patients the possibility of zero bleeds along with dosing flexibility to accommodate their lifestyles and needs.