Novo Nordisk to Present New Hemophilia Research

Novo Nordisk will present new data from clinical studies in hemophilia A and B at the 2025 ISTH Congress, set to take place on June 21-25. The presentations will feature results from multiple phase 3 trials evaluating investigational therapies and examining various clinical, treatment, and patient-reported outcomes.

Hemophilia is a rare genetic bleeding disorder that prevents proper clot formation due to deficiencies in clotting factors VIII (hemophilia A) or IX (hemophilia B), reports Novo Nordisk. The condition affects an estimated 1.1 million people worldwide and about one in every 10,000 people in Mexico, according to the National Epidemiological Surveillance System of the Ministry of Health. It is often managed with intravenous replacement therapies, though immune responses can sometimes reduce their effectiveness.

Among the highlights, Novo Nordisk will share data from the phase 3 FRONTIER5 study, which examines Mim8, an investigational mimetic therapy intended to simulate clotting factor function. This trial specifically evaluates the safety and preferences of individuals with hemophilia A, both with and without inhibitors, who switched from emicizumab to Mim8.

The company will also present findings from the explorer7 and explorer8 trials. These open-label, multicenter phase 3 studies assess the use of prophylactic concizumab compared to on-demand treatment. Researchers will analyze outcomes including joint and non-joint bleeds and annualized bleeding rates in both hemophilia A and B patients.

In addition to clinical metrics, presentations will address treatment satisfaction among physicians, patient preferences in administration, and global real-world data related to joint bleeding episodes and diagnostic trends.

“Our research is rooted in a deep understanding of the hemophilia community,” says Stephanie Seremetis, Chief Medical Officer and Corporate Vice President for Rare Disease, Novo Nordisk. “Through this latest research, we are honored to build on our long-standing legacy in rare blood disorders to support patients who face the challenges of this complex condition.”