Novo hemophilia A drug meets phase 3 goals

May 14, 2024

Novo Nordisk announced positive results from a phase 3a study evaluating the efficacy and safety of its subcutaneous FVIIIa mimetic bispecific antibody, Mim8, in patients with hemophilia A.

The 26-week FRONTIER 2 trial included 254 participants aged 12 and older. The trial met its co-primary endpoints, showing that both once-weekly and once-monthly dosing regimens of Mim8 achieved a statistically significant reduction in treated bleeds.

In patients with no prior prophylaxis, once-weekly and once-monthly Mim8 reduced treated bleeds by 97% and 99%, respectively. Additionally, 86% of patients on the weekly regimen and 95% on the monthly regimen experienced zero treated bleeds, a stark contrast to the 0% in the no prophylaxis group.

In patients previously on coagulation factor prophylaxis, Mim8 also demonstrated superior results. Weekly and monthly Mim8 reduced treated bleeds by 48% and 43%, respectively. Furthermore, 66% of patients on weekly Mim8 and 65% on monthly Mim8 experienced zero treated bleeds, showcasing the effectiveness of the treatment compared to previous prophylaxis methods.

Hemophilia A is a rare, inherited bleeding disorder characterized by the body's inability to produce sufficient amounts of clotting Factor VIII (FVIII), a protein essential for blood clotting. This deficiency leads to prolonged bleeding episodes, which can occur spontaneously or after injuries or surgeries. The disorder can vary in severity, with severe cases experiencing frequent bleeding episodes and mild cases having bleeding problems primarily during surgeries or injuries. Treatment often involves regular infusions of FVIII to prevent or control bleeding.

Mim8 (denecimig) is a new type of antibody that mimics Factor VIII by working with other clotting factors (FIX and FX) on the surface of platelets.

Novo Nordisk says it aims to submit Mim8 for the first regulatory approval towards the end of 2024.