Novo Nordisk Presents Promising Data on Sickle Cell Disease and Hemophilia Treatments at ASH 2024
Key Insights
Novo Nordisk presented Phase 2 results from the HIBISCUS trial, showing etavopivat reduced vaso-occlusive crises in sickle cell disease (search) patients.
Interim Phase 3 data from the FRONTIER4 trial highlighted the safety and efficacy of Mim8 prophylaxis in hemophilia (search) A patients with or without inhibitors.
Phase 3 explorer7 study results demonstrated the efficacy of concizumab in hemophilia (search) A or B patients with inhibitors, regardless of target joints at baseline.
Novo Nordisk announced the presentation of 13 abstracts at the 66th Annual Meeting and Exposition of the American Society of Hematology (ASH) in San Diego, California, from December 7-10, 2024. The data includes new findings from clinical trials evaluating potential treatments for sickle cell disease (search) and hemophilia (search).
Etavopivat Shows Promise in Sickle Cell Disease
Among the presentations, the 52-week results from the Phase 2 part of the Phase 2/3 HIBISCUS trial of etavopivat were presented. Etavopivat is an investigational, oral, small-molecule activator of erythrocyte pyruvate kinase (PKR). The HIBISCUS trial aimed to determine the dose for the Phase 3 part of the trial and examine the safety and efficacy of etavopivat, including the incidence of vaso-occlusive crises (VOCs) in patients with sickle cell disease (search). The results from HIBISCUS were highlighted in the ASH Annual Meeting Press Program session.
According to Novo Nordisk, etavopivat-mediated activation of PKR lowers levels of 2,3-diphosphoglycerate (2,3-DPG) and raises adenosine triphosphate (ATP) levels, which has the potential to improve oxygen bind to haemoglobin (i.e. increase oxygen affinity), reduce haemolysis and decrease VOCs by improving red blood cell health.
Hemophilia A Treatment Advances
Novo Nordisk also presented data from its hemophilia (search) portfolio, including an oral presentation on the efficacy and safety results of an interim analysis from FRONTIER4, a Phase 3 open-label, multi-center extension study of Mim8 in people living with hemophilia A with and without inhibitors (HA/HAwI). Mim8 is an investigational Factor VIIIa (search) (FVIIIa) mimetic bispecific antibody designed with the potential to deliver sustained haemostasis for once-monthly, once every two weeks or once-weekly prophylaxis for people living with haemophilia A with and without inhibitors. Administered under the skin, Mim8 bridges Factor IXa (search) and Factor X, replacing Factor VIII and restoring the body’s thrombin generation capacity, helping blood to clot.
Concizumab Data in Hemophilia A and B
Additionally, data from the Phase 3 explorer7 study assessing the efficacy of concizumab in people with hemophilia (search) A or B with inhibitors (HAwI/HBwI), with or without recurring bleeds into the same joint (or target joints) at baseline, was presented. Concizumab is an anti-tissue factor pathway inhibitor (TFPI (search)) monoclonal antibody designed to block TFPI, a protein in the body that stops blood from clotting. By blocking TFPI, concizumab encourages the production of thrombin, which helps to clot the blood and prevent bleeding.
Martin Holst Lange, executive vice president and head of Development at Novo Nordisk, stated, "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 (search), a first for Novo Nordisk. Sickle cell disease affects approximately eight million people worldwide with big consequences for the individual patient, yet there are few treatment options available. Our sickle cell research builds on our legacy in haemophilia, where we continue to advance research to address the unmet needs of patients."