Novo Nordisk has announced that 13 abstracts from its research will be presented at the 66th Annual Meeting and Exposition of the American Society of Hematology (ASH) in San Diego, California, from December 7 to 10, 2024. Of these, three will be featured in oral sessions, showcasing the company’s advancements in treatments for rare blood disorders, including sickle cell disease and hemophilia.
One of the key oral presentations on December 7 will focus on the 52-week results from the phase 2 portion of the ongoing phase 2/3 HIBISCUS trial, investigating the investigational drug etavopivat in patients with sickle cell disease. These findings provide crucial data on the optimal dose for the phase 3 trial and examine the drug’s safety and efficacy, particularly in reducing vaso-occlusive crises—painful episodes that occur when blood vessels become blocked and deprive tissues of oxygen. The results will be highlighted in the ASH Annual Meeting Press Program during the session titled “Reading Up on the Classics: Treating Not-So-Benign Hematology Conditions” on December 7 at 08:30 PST.
Novo Nordisk’s hemophilia portfolio will also be prominently featured, with two important oral presentations. The first will present the interim analysis results from the phase 3 FRONTIER4 study of Mim8, an investigational therapy for people with hemophilia A, both with and without inhibitors. The second presentation will provide data from the phase 3 explorer7 study, evaluating the efficacy of concizumab in individuals with hemophilia A or B, with or without inhibitors, and with or without recurring joint bleeds at baseline.
Martin Holst Lange, executive vice president and head of Development at Novo Nordisk, commented on the significance of these findings: “There is a significant unmet need for novel treatment options that have the potential to transform care for people with rare blood disorders globally. I am particularly excited that we at the ASH congress will present new data from our pipeline in sickle cell disease, a first for Novo Nordisk. Sickle cell disease affects approximately eight million people worldwide, and there are few treatment options available. Our sickle cell research builds on our legacy in hemophilia, where we continue to advance research to address the unmet needs of patients.”
Summary of Presentations at ASH 2024
Novo Nordisk will present a variety of data in both poster and oral sessions. The accepted abstracts include preliminary data that may be updated before final publication in the journal Blood following the congress. Below are the details of the presentations:
Sickle Cell Disease:
- Etavopivat Reduces Incidence of Vaso-Occlusive Crises in Patients with Sickle Cell Disease: HIBISCUS Trial Phase 2 Results Through 52 Weeks (179-O)
- Etavopivat Increases Arterial Hemoglobin-Oxygen Saturation During Moderate and Severe Hypoxia: A Mechanistic Phase 1 Trial in Healthy Volunteers (2461-P)
- Large-Scale Analysis of the Real-World Association Between Fetal Hemoglobin and Vaso-Occlusive Crises in Sickle Cell Disease (1124-P)
- Characterizing People with Sickle Cell Disease Who Share Attitudes Regarding Clinical Trial Participation: Findings from the Global LISTEN Survey (1124-P)
- How People with Sickle Cell Disease Rate Motivators Associated with the Likelihood of Participating in Clinical Trials: Findings from the Global LISTEN Survey (1135-P)
- Motivators and Barriers for People with Sickle Cell Disease Participating in Clinical Trials: U.S. Findings from the LISTEN Survey (1135-P)
- Noninvasive, Accessible Smartphone App for At-Home Hemoglobin Monitoring in Sickle Cell Disease (2248-P)
Hemophilia:
- Safety and Efficacy of Mim8 Prophylaxis Administered Once Every Two Weeks for Patients with Hemophilia A with or Without Inhibitors: Interim Analysis of the FRONTIER4 Open-Label Extension Study (718-O)
- Mim8 Prophylaxis Beyond Bleeding: Investigating Multifaceted, Patient-Reported Outcomes for Hemophilia A in the FRONTIER2 Study (1212-P)
- Annualized Bleeding Rates in Patients with Hemophilia A or B and Inhibitors, With and Without Target Joints at Baseline: Results from the Concizumab Phase 3 Explorer7 Study (715-O)
- Burden of Treatment on People with Hemophilia: Global Real-World Data (5077-P)
- Physical and Psychological Burden on People with Hemophilia: Global Real-World Data (2318-P)
- Unmet Needs of Patients with Hemophilia A/B with or Without Inhibitors: Real-World End-of-Study Results from the Explorer6 Non-Interventional Study (2585-P)
These presentations reflect Novo Nordisk’s continued dedication to addressing the unmet needs of patients with blood disorders. By advancing research in sickle cell disease and hemophilia, the company aims to improve patient outcomes and contribute to the global conversation on transforming care for these conditions.
