Novo Nordisk has shared positive results from a late-stage study of its investigational haemophilia A therapy Mim8 in patients aged one to 11 years.
The phase 3 FRONTIER3 trial initially evaluated a once-weekly dose of the candidate to prevent prolonged and spontaneous bleeding, before giving patients the option to move to once-monthly dosing after 26 weeks.
Approximately 1,125,000 people worldwide are affected by haemophilia, a rare inherited bleeding disorder that impairs the body’s ability to make blood clots.
Haemophilia A, which is caused by a missing or defective clotting factor VIII, accounts for up to 85% of global cases, and it is estimated that up to 30% of patients with this form of the disease have inhibitors, an immune system response to the clotting factors in replacement therapy that causes treatment to stop working.
Novo’s Mim8 is a subcutaneously-administered mimetic bi-specific antibody that is designed to replace the function of the missing clotting factor VIII.
The interim FRONTIER3 data presented at this year’s Annual Congress of the European Association for Haemophilia and Allied Disorders demonstrated that Mim8 was well tolerated and efficacious in patients with and without inhibitors.
For part one of the study, which assessed once-weekly dosing, the estimated mean annualised bleeding rate (ABR) for treated bleeds was 0.53 and the median ABR was zero, with 74.3% of patients experiencing zero treated bleeds. Novo added that all children with inhibitors reported zero treated bleeds.
The company also detailed that, at week 26, 98% of caregivers said they preferred Mim8 to prior treatment, and positive improvement trends were observed in mean physical function and quality of life scores compared to baseline.
Ludovic Helfgott, executive vice president for rare disease at Novo, 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.”
The readout builds on the previously reported positive phase 3 results for Mim8 in adults and adolescents aged 12 years and older.
Novo said data from the ongoing phase 3 FRONTIER programme will be disclosed at upcoming congresses, and that it expects to submit regulatory submissions for the therapy this year.