Novo Nordisk has bolstered the case for its near-approval hemophilia A drug candidate Mim8, linking the prospect to reductions in treated bleeds in children with the hereditary bleeding disorder.
Mim8, a factor VIIIa mimetic bispecific antibody created using Genmab technology, has already shown its promise in adults with hemophilia A. Novo plans to file for approval in the U.S. and the EU this year. Through the FRONTIER3 trial, Novo has generated evidence that the molecule may have a future in the treatment of children with hemophilia A.
Investigators enrolled 70 children aged 1 to 11 years to receive once-weekly doses of Mim8 for 26 weeks. Fourteen of the children had hemophilia A with inhibitors, which prevent standard factor replacement treatments from stopping bleeding.
The average annualized rate of treated bleeds (ABR) was 0.53, and 74.3% of participants had no treated bleeds. All 14 of the children with hemophilia A with inhibitors had zero treated bleeds. However, comparing the results to data on drugs such as Roche’s Hemlibra is complicated by differences between the trials. Roche excluded kids without inhibitors and reported a treated ABR of 0.3 after a median of 57.6 weeks.
Novo’s primary endpoint looked at safety. No patients suffered major safety concerns such as deaths, thromboembolic events or severe treatment-emergent adverse events. Similarly, investigators saw no clinical evidence of neutralizing anti-drug antibodies. Less than 1% of injections were reported to have caused site reactions.
The secondary endpoints included measures of physical function and quality of life. Novo found 98% of caregivers preferred Mim8 to the prior treatment—73% strongly preferred the study drug—and children showed signs of improved physical function and quality of life after 26 weeks. The kids have now moved into part two of the trial, with 45% switching to monthly dosing and the rest staying on weekly jabs.