Novo Nordisk has shared positive results from a late-stage study of its haemophilia A therapy, Mim8, which the company hopes to submit for regulatory approval towards the end of this year.
Haemophilia A, a rare inherited bleeding disorder caused by a missing or defective clotting factor VIII, accounts for 80 to 85% of the 1,125,000 global haemophilia cases.
Up to 30% of haemophilia A patients have inhibitors, an immune system response to the clotting factors in replacement therapy that causes treatment to stop working.
Administered subcutaneously, Mim8 is designed to replace the function of the missing clotting factor VIII.
The phase 3a FRONTIER 2 trial has been evaluating the efficacy and safety of a once-weekly and once-monthly dose of the candidate against both no prophylaxis and prior coagulation factor prophylaxis treatment in patients aged 12 years and older with or without inhibitors.
The trial achieved its co-primary endpoints by demonstrating a statistically significant and superior reduction of treated bleeding episodes with both once-weekly and once-monthly Mim8 versus no prophylaxis and prior coagulation factor prophylaxis treatment.
In patients with no prior prophylaxis treatment, once-weekly and once-monthly Mim8 demonstrated reductions of 97% and 99% in treated bleeds, respectively, compared to no prophylaxis treatment.
In addition, 86% of patients with no prior prophylaxis treatment who received once-weekly Mim8 and 95% of those treated with once-monthly Mim8 experienced zero treated bleeds, compared to 0% of those treated with no prophylaxis.
In the intra-patient analysis of patients with prior coagulation factor prophylaxis, once-weekly and once-monthly Mim8 demonstrated reductions of 48% and 43% in treated bleeds, respectively, compared to those who received only prior coagulation factor prophylaxis. Additionally, 66% of patients treated with once-weekly Mim8 and 65% of those receiving once-monthly Mim8 experienced zero treated bleeds.
Martin Holst Lange, executive vice president for development at Novo Nordisk, said: “This data demonstrates the ability of Mim8 to prevent bleeding episodes effectively and safely in people with haemophilia A, regardless of their dosing frequency.
“Given the differing needs of people living with haemophilia A, a convenient once-weekly or once-monthly dosing provides optionality and flexibility for people living with haemophilia A with or without inhibitors.”