Novo Nordisk's Mim8 Shows Superior ABR Reduction in Haemophilia A

BAGSVAERD, Denmark, June 23, 2024 — Novo Nordisk has unveiled findings from the phase 3 FRONTIER2 trial, which studied 254 participants aged 12 and above with haemophilia A, with and without inhibitors. The trial evaluated both once-weekly and once-monthly prophylactic treatments using the investigational drug Mim8. These findings were presented at the International Society of Thrombosis and Haemostasis Annual Congress (ISTH 2024) in Bangkok, Thailand.

For participants with no prior prophylactic treatment, up to 95% experienced no treated bleeds while on Mim8. In contrast, those with previous coagulation factor prophylaxis also showed significant improvements. According to Dr. Maria Elisa Mancuso, the lead investigator of the trial and a Senior Consultant in Haematology at IRCCS Humanitas Research Hospital in Milan, Italy, Mim8 prophylaxis demonstrated superiority over both on-demand treatment and prior clotting factor prophylaxis in reducing the number of treated bleeding episodes.

Mim8 is a next-generation Factor VIIIa (FVIIIa) mimetic bispecific antibody. It aims to provide sustained haemostasis and is currently being developed for both once-weekly and once-monthly prophylactic treatment in haemophilia A patients, with and without inhibitors. Administered subcutaneously, Mim8 functions by bridging Factor IXa/X (FIXa/FX), effectively replacing the missing FVIII and restoring the body's ability to generate thrombin, thus aiding in blood clotting.

The FRONTIER2 trial revealed that Mim8 was well-tolerated, with no significant safety concerns, such as thromboembolic events or serious adverse events, aligning with previous data. Martin Holst Lange, Executive Vice President of Development at Novo Nordisk, expressed optimism regarding the efficacy and safety data, highlighting the potential for Mim8 to offer patients zero bleeds and flexible dosing to accommodate their lifestyles. Novo Nordisk plans to discuss these findings with regulatory authorities, aiming for the first regulatory approval of Mim8 by the end of 2024. Further data from the ongoing phase 3 FRONTIER program will be shared in upcoming congresses and publications through 2025.

Haemophilia is a rare genetic disorder that affects the body's ability to form blood clots, necessary to stop bleeding. Approximately 1,125,000 people globally are affected by haemophilia, with haemophilia A comprising 80-85% of these cases. The disorder often presents differently in males and females due to its x-linked recessive nature, with about 88% of diagnosed individuals being male. Haemophilia A is caused by a deficiency or defect in clotting Factor VIII (FVIII). Some patients develop inhibitors, an immune response to replacement therapy that makes treatment ineffective. It is estimated that up to 30% of people with severe haemophilia A develop these inhibitors.

The FRONTIER2 trial, part of the larger FRONTIER phase 3 clinical development program, investigates the efficacy and safety of Mim8 as a prophylactic treatment for haemophilia A patients. The trial consists of a 52-week period comparing once-weekly and once-monthly Mim8 with no prophylaxis and prior coagulation factor prophylaxis. Participants were randomized into five treatment groups, which included those who previously received on-demand or prophylactic treatments before enrolling in the trial.

Novo Nordisk, established in 1923 and headquartered in Denmark, focuses on transforming the treatment of serious chronic diseases. The company employs approximately 66,000 people across 80 countries and markets its products in about 170 countries.