Haemophilia drug could reduce treated bleeding episodes

A next-generation FVIIIa mimetic bispecific antibody has demonstrated statistically significant and superior reduction of treated bleeding episodes in haemophilia A, new headline data from a pivotal Phase IIIa trial show. Mim8 was evaluated in participants aged 12 years or older as both a once-weekly and once-monthly treatment versus no prophylaxis treatment and prior coagulation factor prophylaxis treatment.

Mim8 replaces the missing factor FVIII, “effectively restores the body’s thrombin generation capacity, helping blood to clot”, Novo Nordisk AS explained.

Significant reductions of treated bleeding episodes in haemophilia A

In the FRONTIER 2 trial, treatment once-weekly and once-monthly with Mim8 provided a 97 percent and 99 percent reduction in treated bleeds in patients with no prior prophylaxis treatment, respectively. This was compared to individuals not given prophylaxis treatment, according to Novo Nordisk.

Furthermore, the company explained that 86 percent of people treated with once-weekly Mim8 and 95 percent of those treated with once-monthly Mim8 experienced zero treated bleeds. This was compared to zero percent of individuals not given prophylaxis therapy.

In patients with prior coagulation factor prophylaxis, once-weekly and once-monthly Mim8 demonstrated superior reductions of 48 percent and 43 percent in treated bleeds, respectively. This was compared to prior coagulation factor prophylaxis.

These statistics were reported in the intra-patient analysis, Novo Nordisk shared.

Another of these findings were that 66 percent of those given once-weekly Mim8 and 65 percent of people treated with once-monthly Mim8 experienced zero treated bleeds.

Potential of Mim8

The safety profile of Mim8 was in line with previous trials. No deaths or thromboembolic events were observed, the study found.

“These data [from the FRONTIER 2 trial] demonstrate the ability of Mim8 to prevent bleeding episodes effectively and safely in people with haemophilia A, regardless of their dosing frequency,” commented Martin Holst Lange, Executive Vice President for Development at Novo Nordisk.

The company plans to submit filings for regulatory approval of the antibody treatment towards the end of the year.

Data from the Phase III FRONTIER programme will be shared in future conferences and publications in 2024 and 2025, Novo Nordisk added.