Performance of Novo’s monthly haemophilia A therapy sets up challenge to Roche’s Hemlibra

2024-05-14

临床3期临床结果基因疗法临床2期

A pivotal study showed that both weekly and monthly regimens of Novo Nordisk’s factor VIIIa mimetic bispecific antibody Mim8 significantly decreased bleeding episodes compared with on-demand and previous prophylactic treatment in patients with haemophilia A. The results suggest that Mim8 could be a strong challenger to Roche’s Hemlibra (emicizumab), which is given once weekly.

“Given the differing needs of people living with haemophilia A, a convenient once-weekly or once-monthly dosing provides optionality and flexibility,” remarked Martin Holst Lange, EVP for development at Novo Nordisk.

The Phase IIIa FRONTIER 2 trial — comprising a 26-week main segment and an equally long extension portion — compared once-weekly and once-monthly Mim8 against no prophylaxis, and against prior coagulation factor prophylaxis in 254 patients with haemophilia A aged 12 years and above, with or without inhibitors. The study’s co-primary endpoints were the number of treated bleeds for Mim8 versus no prophylaxis and prior prophylaxis during the main phase.

Main goals met

The headline results announced Monday showed that once-weekly and once-monthly Mim8 demonstrated significant 97% and 99% reductions in treated bleeds, respectively, compared with no prophylaxis. Additionally, 86% and 95% of patients receiving once-weekly and once-monthly Mim8 regimens, respectively, had zero treated bleeds versus none of the patients who did not receive prophylaxis.

Jefferies analysts suggested that preventing at least 80% of patients from needing to be treated for bleeding is a key determiner of whether Mim8 poses a threat to Hemlibra. Thus, the FRONTIER 2 results “may challenge Roche’s incumbent,” the analysts said.

In an intra-patient analysis of patients who had received prophylaxis earlier, once-weekly and once-monthly Mim8 showed superior reductions of 48% and 43% in treated bleeds, respectively, compared with prior prophylactic treatment. Furthermore, 66% of patients on weekly and 65% on monthly Mim8 had no treated bleeds.

Marketing applications this year

Meanwhile, the drug’s safety and tolerability were consistent with previous studies, with no reports of deaths or thromboembolic events. The company noted that detailed data will be presented at upcoming medical congresses.

Novo Nordisk’s FRONTIER programme is investigating Mim8 across four late-stage studies in haemophilia A. The company plans to file regulatory submissions towards the end of this year, which alongside FRONTIER 2, will also include data from the Phase III FRONTIER 3 study.