Novo Nordisk announced that it will present new phase 3 clinical trial data for its hemophilia treatment portfolio at the upcoming International Society on Thrombosis and Haemostasis (ISTH) Congress, scheduled for June 21-25, 2025. The presentations will focus on two investigational therapies: Mim8 (denecimig) and concizumab, representing significant advances in treating both hemophilia A and B.
Key Phase 3 Trial Updates
The pharmaceutical company will present two updates from phase 3 trials evaluating Mim8, an investigational mimetic therapy designed to replicate the function of missing clotting factors. The FRONTIER5 phase 3 trial analysis will specifically assess the safety and patient preferences when switching directly from emicizumab to Mim8 in people with hemophilia A, both with and without inhibitors.
Additionally, Novo Nordisk will share findings from two prospective, multicenter, open-label phase 3 trials known as explorer7 and explorer8. These studies investigate the effectiveness of preventive concizumab treatment versus on-demand treatment across both hemophilia A and B patients, with and without inhibitors.
Clinical Endpoints and Outcomes
The explorer7 and explorer8 trials will present data on several critical clinical endpoints, including joint bleeds, non-joint bleeds, and annualized bleeding rates. The studies will also include additional research on thrombin generation with concizumab treatment in hemophilia A and B patients.
The data presentations will encompass insights on clotting mechanisms, thrombin generation, bleeding episodes and medication impact, patient administration preferences, physician treatment satisfaction, and global real-world diagnosis and treatment data for joint bleeds.
Addressing Unmet Medical Needs
"At Novo Nordisk, we believe understanding the whole person and their journey is essential to addressing the unmet needs in people with hemophilia," said Stephanie Seremetis, Chief Medical Officer and CVP for Rare Disease at Novo Nordisk. "Our research is rooted in a deep understanding of the hemophilia community, aiming to drive critical advancements across rare blood disorders in order to help address these unmet needs."
Seremetis emphasized the company's commitment to supporting patients facing the challenges of this complex condition, stating, "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."
Disease Background and Treatment Challenges
Hemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process essential for stopping bleeding. The condition affects approximately 1,125,000 people worldwide. Hemophilia A results from missing or defective clotting Factor VIII (FVIII), while hemophilia B is caused by missing or defective clotting Factor IX (FIX).
Current treatment typically involves replacing the missing clotting factor through intravenous infusions, known as replacement therapy. However, the body can sometimes produce inhibitors as an immune response to clotting factor replacement therapy, which can render the treatment ineffective and limit available treatment options.
Conference Presentations
The accepted data at the 33rd ISTH Congress will include both poster and oral presentations covering Novo Nordisk's hemophilia research portfolio. The presentations will provide comprehensive insights into treatment outcomes across both hemophilia A and B populations, including patients with and without inhibitors.
