Novo Nordisk to present phase 3 trials across hemophilia portfolio, reinforcing commitment to research in rare blood disorders, at ISTH 2025

• Key presentations include two updates from a phase 3 trial evaluating investigational treatment with Mim8 (denecimig) and five assessing treatment outcomes with concizumab in hemophilia
  
  
• A phase 3 trial analysis from FRONTIER5 will evaluate the safety of switching directly from emicizumab to Mim8 (denecimig) in people living with hemophilia A/B
  
  
• Findings from explorer7 and explorer8 phase 3 trials will assess data including non-joint bleeds, annualized bleeding rates and additional studies including thrombin generation with concizumab in hemophilia A/B

PLAINSBORO, N.J., June 6, 2025 /PRNewswire/ -- Novo Nordisk today announced that new hemophilia data will be presented at the upcoming International Society on Thrombosis and Haemostasis (ISTH) Congress, June 21-25. Key data presentations across both hemophilia A and B (HA and HB), with and without inhibitors, will share insights on clotting, thrombin generation, bleeding episodes and medication impact, patient administration preferences, physician treatment satisfaction, and global real-world diagnosis and treatment data for joint bleeds.

"At Novo Nordisk, we believe understanding the whole person and their journey is essential to addressing the unmet needs in people with hemophilia. 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," said Stephanie Seremetis, Chief Medical Officer and CVP for Rare Disease at 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."

New phase 3 data will be presented on Mim8, an investigational mimetic therapy designed to replicate the function of missing clotting factors.¹ Analysis from the phase 3 FRONTIER5 trial will assess the safety and preferences of people with HA, with and without inhibitors, who switch from emicizumab to Mim8.

In addition, new data from two prospective, multicenter, open label, phase 3 trials (explorer7 and explorer8) will investigate joint bleeds, non-joint bleeds, and annualized bleeding rate with preventive concizumab being investigated versus on-demand treatment across both HA and HB, with and without inhibitors. 

Summary of presentations
Accepted data at the 33^rd ISTH Congress includes the following poster and oral presentations. Additional information can be found on the ISTH website.

Full details of Novo Nordisk abstracts to be presented:

About hemophilia
Hemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.² It is estimated to affect approximately 1,125,000 people worldwide.³ There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing. Hemophilia A is caused by a missing or defective clotting Factor VIII (FVIII), and hemophilia B is caused by a missing or defective clotting Factor IX (FIX).² Hemophilia is often treated by replacing the missing clotting factor via intravenous infusions, also known as replacement therapy. However, sometimes the body can produce inhibitors as an immune response to the clotting factor replacement therapy. When this happens, the therapy may not work and can limit treatment options.⁵ 

About Novo Nordisk
Novo Nordisk is a leading global healthcare company that's been making innovative medicines to help people with diabetes lead longer, healthier lives for more than 100 years. This heritage has given us experience and capabilities that also enable us to drive change to help people defeat other serious chronic diseases such as obesity, rare blood, and endocrine disorders. We remain steadfast in our conviction that the formula for lasting success is to stay focused, think long-term, and do business in a financially, socially, and environmentally responsible way. With a U.S. presence spanning 40 years, Novo Nordisk U.S. is headquartered in New Jersey and employs over 10,000 people throughout the country across 12 manufacturing, R&D, and corporate locations in eight states plus Washington DC. For more information, visit novonordisk-us.com, Facebook, Instagram, and X.

Contacts for further information

References

1. Østergaard H, Lund J, Greisen PJ, et al. A factor VIIIa-mimetic bispecific antibody, Mim8, ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021;138(14):1258-1268.
2. MedlinePlus. Hemophilia. Accessed May 2025. Available at https://medlineplus.gov/genetics/condition/hemophilia/.
3. Iorio A, Stonebraker JS, Chambost H, et al.; Data and demographics committee of the World Federation of Hemophilia. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med. 2019;171(8):540–546.
4. Centers for Disease Control and Prevention (CDC). Treatment of hemophilia. Accessed May 2025. Available at https://www.cdc.gov/hemophilia/treatment/index.html.
5. Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26 Suppl 6:1-158.

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