Novo Nordisk's Mim8 Shows Superior Bleeding Reduction in Haemophilia A Trial

Bagsværd, Denmark, 13 May 2024 – Novo Nordisk has unveiled the main findings from its pivotal FRONTIER 2 trial, a phase 3a study lasting 26 weeks. This open-label, randomised, controlled, multi-arm trial involved 254 participants and sought to evaluate the effectiveness and safety of once-weekly and once-monthly subcutaneous Mim8. The study compared these regimens against no prophylaxis and prior coagulation factor prophylaxis treatments in individuals aged 12 and above with haemophilia A, both with and without inhibitors.

The trial reached its co-primary goals by showing a statistically significant reduction in treated bleeding episodes with both weekly and monthly Mim8 compared to no prophylaxis and previous coagulation factor prophylaxis treatments. Among participants with no prior prophylaxis, weekly and monthly Mim8 reduced treated bleeds by 97% and 99%, respectively, compared to those without prophylaxis. Additionally, 86% of those on weekly Mim8 and 95% on monthly Mim8 experienced no treated bleeds, while none of the participants receiving no prophylaxis reported zero treated bleeds.

In an intra-patient analysis of those with prior coagulation factor prophylaxis, weekly and monthly Mim8 reduced treated bleeds by 48% and 43%, respectively, compared to their previous treatments. Furthermore, 66% of participants on weekly Mim8 and 65% on monthly Mim8 experienced no treated bleeds.

Mim8 was found to have a safety profile in line with previous trials, with no deaths or thromboembolic events reported.

Martin Holst Lange, executive vice president for Development at Novo Nordisk, expressed satisfaction with the results. He highlighted the significance of Mim8 in effectively and safely preventing bleeding episodes in haemophilia A patients, accommodating different dosing needs by offering both weekly and monthly options.

Novo Nordisk plans to seek regulatory approval for Mim8 by the end of 2024. Findings from the FRONTIER programme, including FRONTIER 2, will be presented at upcoming conferences and published in 2024 and 2025.

Haemophilia is a rare inherited bleeding disorder hindering the body's ability to form blood clots, essential for stopping bleeding. It affects approximately 1,125,000 people worldwide, with haemophilia A constituting 80-85% of all cases. Due to its x-linked recessive inheritance, it predominantly affects males, who comprise about 88% of diagnosed cases globally. Haemophilia A results from a deficiency or defect in clotting Factor VIII (FVIII). Some individuals with haemophilia develop inhibitors that render replacement therapies ineffective, affecting up to 30% of those with haemophilia A.

Mim8 is a next-generation FVIIIa mimetic bispecific antibody designed for sustained haemostasis, suitable for once-weekly or once-monthly prophylaxis in haemophilia A patients, with or without inhibitors. Administered subcutaneously, Mim8 bridges Factor IXa/X (FIXa/FX) upon activation, thereby replacing the missing FVIII, which helps restore thrombin generation and blood clotting.

The FRONTIER clinical development programme evaluates Mim8 as a preventative treatment for haemophilia A patients. The phase 3 programme includes several studies:

- FRONTIER 2: A 52-week efficacy and safety trial with a 26-52-week run-in period comparing weekly and monthly Mim8 to no prophylaxis and prior prophylaxis treatments in individuals aged 12 and up.
- FRONTIER 3: A 52-week safety and efficacy trial in pediatric patients aged 1-11 years, with an option to switch from weekly to monthly Mim8 after 26 weeks.
- FRONTIER 4: An open-label extension study collecting long-term safety data following participation in earlier FRONTIER trials.
- FRONTIER 5: A 26-week trial investigating the pharmacokinetics, pharmacodynamics, and safety of switching from emizicumab to Mim8 in adults and adolescents.

Novo Nordisk, a leading global healthcare company, was founded in 1923 and is headquartered in Denmark. The company aims to combat chronic diseases through scientific advancements, improved access to medicines, and efforts toward prevention and cure. Novo Nordisk employs around 66,000 people in 80 countries and markets its products in about 170 countries.