Novo's monthly haemophilia A therapy challenges Roche’s Hemlibra

A recent and crucial study has demonstrated that Novo Nordisk’s factor VIIIa mimetic bispecific antibody, Mim8, substantially reduced bleeding episodes in patients with haemophilia A compared to on-demand treatments and previous prophylactic regimens. These findings indicate that Mim8 could emerge as a strong competitor to Roche’s Hemlibra (emicizumab), which is administered once weekly. Martin Holst Lange, Executive Vice President for Development at Novo Nordisk, highlighted the advantages of weekly or monthly dosing schedules, offering flexibility and convenience for patients with differing needs.

The pivotal Phase IIIa FRONTIER 2 trial, which included a 26-week main phase followed by an extension of the same duration, evaluated the efficacy of once-weekly and once-monthly Mim8 against no prophylaxis and previous coagulation factor prophylaxis. The trial involved 254 haemophilia A patients aged 12 and older, including those with and without inhibitors. The primary endpoints focused on the number of treated bleeds comparing Mim8 with no prophylaxis and prior prophylactic treatments during the main study period.

The findings, announced on Monday, revealed that the once-weekly and once-monthly Mim8 regimens significantly reduced treated bleeds by 97% and 99%, respectively, compared to no prophylaxis. Moreover, 86% of patients on the weekly regimen and 95% on the monthly regimen experienced zero treated bleeds, in stark contrast to none of the patients without prophylaxis achieving this outcome. Analysts from Jefferies noted that the ability to prevent at least 80% of patients from requiring treatment for bleeding could be crucial in challenging Hemlibra’s market position.

Further analysis within the patient group that had previously received prophylaxis showed that Mim8 offered superior reductions in treated bleeds—48% for the weekly regimen and 43% for the monthly regimen—compared to earlier prophylactic treatments. Additionally, 66% of patients on the weekly regimen and 65% on the monthly regimen had no treated bleeds during the trial.

Regarding safety and tolerability, the drug’s profile was consistent with earlier studies, with no deaths or thromboembolic events reported. Novo Nordisk plans to present detailed data from the study at upcoming medical congresses. The company’s FRONTIER programme is assessing Mim8 in four advanced-stage studies for haemophilia A, with regulatory submissions anticipated by the end of the year. These submissions will include data from the Phase III FRONTIER 3 study alongside the results from FRONTIER 2.

In the current landscape of haemophilia A treatments, Mim8 could potentially join a range of available therapies, such as Roche’s Hemlibra, several recombinant coagulation factors, Sanofi and Sobi’s Altuviiio (efanesoctocog alfa), and BioMarin’s gene therapy Roctavian (valoctocogene roxaparvovec). The promising results from the FRONTIER 2 trial position Mim8 as a formidable new option for managing haemophilia A, offering patients more choices in their treatment regimens.