Novo Nordisk’s Mim8 shows promise in Hemophilia A treatment

DENMARK – Novo Nordisk has released promising Phase III data for its new treatment, Mim8, a bispecific antibody mimicking factor VIIIa, which could compete with Roche’s leading drug for haemophilia A, Hemlibra.

The latest findings come from the FRONTIER3 trial involving children aged 1 to 11 years, revealing strong efficacy and patient satisfaction, potentially paving the way for Mim8 to challenge Hemlibra’s dominance. Regulatory applications for Mim8 are expected later this year.

The FRONTIER3 trial, which enrolled 70 children with haemophilia A (both with and without inhibitors), was conducted in two parts. In the first phase, all participants received once-weekly Mim8 prophylaxis for 26 weeks.

In the second phase, patients could choose to continue with once-weekly dosing or switch to a once-monthly regimen for another 26 weeks.

Remarkably, the results showed that 74.3% of children receiving weekly doses had no treated bleeds, including all 14 children with inhibitors.

The mean annualised bleeding rate (ABR) was estimated at 0.53, with a median of zero, highlighting Mim8’s effectiveness. After the initial period, 45% of patients opted for the more convenient monthly dosing.

“Mim8 is designed with the aim to offer treatment flexibility based on individual lifestyles,” said Ludovic Helfgott, Novo’s executive vice president for rare diseases.

Patient and caregiver feedback strongly supported the treatment, with 98% expressing a preference for Mim8 over prior therapies, and 73% stating they “very strongly” preferred it. Improvements were also reported in physical function and quality of life.

Safety remained a key focus during the study. Mim8 demonstrated a consistent safety profile, with no major concerns or signs of neutralizing anti-drug antibodies.

Additionally, less than 1% of injections caused site reactions, making it a reliable option for young patients.

The FRONTIER3 findings build on earlier results from the Phase IIIa FRONTIER2 trial, which compared Mim8 to no prophylaxis and previous coagulation factor treatments in 254 patients aged 12 years and older.

That study showed that Mim8 reduced treated bleeds by 99% compared to no prophylaxis after 26 weeks. Furthermore, 95% of patients on Mim8 experienced zero bleeds, while none in the control group achieved this outcome.

Despite these successes, Novo faces an uphill battle in displacing Hemlibra from its leading position. According to key opinion leader Benjamin Samelson-Jones, Mim8’s once-monthly dosing and safety profile could attract new patients.

However, its modest advantage over Hemlibra, combined with high patient satisfaction with existing treatments, may limit Mim8’s market share to 10-20%, while Hemlibra could retain 60-70%.