Novo Nordisk has presented new long-term clinical data demonstrating the safety and efficacy of its investigational hemophilia A therapy, denecimig (Mim8), during the International Society on Thrombosis and Haemostasis (ISTH) Congress in Paris. The findings, from the ongoing Phase 3 FRONTIER4 extension study, showed sustained bleed protection and a favorable safety profile in children, adolescents and adults with hemophilia A, regardless of inhibitor status or dosing schedule.

The interim analysis included 426 participants aged one year and older receiving denecimig prophylaxis through once-weekly, once-every-two-weeks or once-monthly dosing regimens. The study reported low rates of mild and transient injection-site reactions, with no clinical evidence of neutralizing antibodies.

Efficacy outcomes remained consistent with previous FRONTIER studies. Estimated annualized bleeding rates were 0.75 among adults and adolescents and 0.37 among children. Approximately 71 percent of adults and adolescents and 89 percent of children experienced no treated bleeding episodes while receiving denecimig.

Patient-reported outcomes also indicated sustained improvements in joint pain and reduced treatment burden across all dosing frequencies. Most participants reported that the denecimig pen injector was easy to use and quick to prepare and administer.

Additional analyses from the Phase 3 FRONTIER2 and FRONTIER5 studies showed that denecimig restored thrombin generation to normal reference levels in adolescents and adults without triggering excessive clotting activity, further supporting its clinical profile.

Novo Nordisk has already submitted a Biologics License Application (BLA) for denecimig to the US Food and Drug Administration, with regulatory review ongoing.

The company also presented first-time results from the Phase 3 explorer10 study evaluating concizumab prophylaxis in children under 12 years of age with hemophilia A or B with inhibitors. The study demonstrated an estimated 82 percent reduction in annualized bleeding rates compared with previous on-demand treatment. While adverse events were common, most were mild, and injection-site reactions remained infrequent.

Denecimig is an investigational bispecific antibody designed to mimic the function of Factor VIII, offering prophylactic treatment for hemophilia A with or without inhibitors. Concizumab, marketed as Alhemo in several countries for specific patient populations, is being investigated for expanded use in younger children.

The new data add to the growing body of evidence supporting innovative prophylactic therapies aimed at improving long-term disease management and reducing treatment burden for people living with hemophilia.