Novo Nordisk’s bispecific antibody Mim8 prevented bleeding events and caused no adverse safety events in the Phase III FRONTIER3 trial. The company plans to file for approval this year, hoping to compete in the hemophilia A space with Roche’s blockbuster Hemlibra.
Novo Nordisk announced positive results Phase III results Friday for its investigational Mim8 bispecific antibody for hemophilia A. The readout, which follows a similar win in adults and adolescents last year, potentially puts Novo on track to compete in a space currently dominated by Roche’s Hemlibra.
Novo plans to submit for approval of Mim8 in both the U.S. and EU this year.
The FRONTIER3 trial enrolled 70 children, ages 1 to 11, with hemophilia A with and without inhibitors for 26 weeks. The study hit its primary endpoints of safety and tolerability, with no major adverse events reported.
As for efficacy, none of the 14 children with inhibitors experienced bleeding events requiring treatment with coagulation factors. Among all participants, the annualized rate of treated bleeds came to 0.53, with 74.3% experiencing no bleeds that required treatment during the trial. Novo also reported improvements in kids’ physical functioning such as running, as well as other quality of life metrics, compared to baseline. After 26 weeks, 98% of caregivers said they preferred the new Mim8 treatment to whatever treatment the kids received before.
“The FRONTIER3 interim analysis data are encouraging for families with young children and indicate that Mim8 could offer an efficacious, convenient, flexible dosing option for children, helping to reduce the treatment burden so families can live more normal lives,” Johnny Mahlangu, the study’s principal investigator, said in a statement.
Friday’s announcement is the latest in the series of positive results for Mim8, which also improved annualized bleed rate in the related Phase III FRONTIERS2 trial in adults and adolescents. That study also showed that the bispecific antibody could be administered prophylactically weekly and monthly. And in the earlier Phase I/II trial FRONTIERS1, Mim8 generated clinically meaningful amounts of thrombin, a clotting factor severely reduced in hemophilia A.
Novo is currently running another Phase III trial, FRONTIER4, to further investigate the safety of prophylactically administering Mim8. An early analysis of interim data from that trial showed no adverse effects.
While positive, it remains unclear whether Novo’s data package for Mim8 will be enough to compete with Roche’s Hemlibra, which is approved for both adults and children and has been entrenched in the market since 2017.
Also in the running to seize a piece of the hemophilia A market—which is forecast to exceed $28 million in the U.S. by 2034—is Pfizer. Last July, the pharma reported positive Phase III results in adult patients for its gene therapy candidate giroctocogene fitelparvovec, which met the trial’s primary and secondry endpoints, showing superiority to Factor VIII infusions. Pfizer currently markets Beqvez for hemophilia B.
Clarification (Feb. 7): This article has been updated to clarify that the FRONTIER3 trial enrolled a total of 70 children, ages 1 to 11, with hemophilia A with and without inhibitors.
