PLAINSBORO, N.J., Dec. 3, 2024
PLAINSBORO, N.J., Dec. 3, 2024 /PRNewswire/ -- Novo Nordisk today announced the presentation of 13 abstracts at the upcoming 66th Annual Meeting and Exposition of the American Society of Hematology (ASH), which will take place from 7 to 10 December 2024 in San Diego, California.
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New results from the phase 2/3 HIBISCUS trial of investigational etavopivat in patients with sickle cell disease will be presented to determine the dose for the phase 3 part of the trial, as well as examine the safety and efficacy of etavopivat. This includes the incidence of vaso-occlusive crises, which occur when sickled red blood cells block blood flow, causing extreme pain. The HIBISCUS data will be highlighted in the ASH Annual Meeting Press Program session: Reading Up on the Classics: Treating Not-So-Benign Hematology Conditions on 7 December at 08:30 PST.
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There are two oral presentations of note from Novo Nordisk's investigational hemophilia portfolio. The first explores 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). Additionally, new data from the phase 3 explorer7 study assesses the efficacy of concizumab in people with hemophilia A or B with inhibitors (HAwI/HBwI), with or without recurring bleeds into the same joint (or target joints) at baseline.
"There is a significant unmet need for novel treatment options that have the potential to transform care for people with rare blood disorders globally," said Martin Holst Lange, executive vice president and head of Development at Novo Nordisk. "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 with big consequences for the individual patient, yet 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 presentationsAccepted data at the 66th ASH annual meeting includes the following poster and oral presentations. Accepted abstracts include preliminary data that may be subject to change in final manuscripts which will be published in the journal Blood following the congress. Dates and times of the presentations can be found on the ASH website.
Sickle cell diseaseInvestigational etavopivat
General sickle cell disease
HemophiliaInvestigational Mim8
Investigational concizumab
General hemophilia
About sickle cell diseaseSickle cell disease (SCD) is a debilitating, life-threatening group of rare, inherited red blood cell disorders, caused by a mutation in the hemoglobin gene.1 This mutation causes red blood cells to become stiff and half-moon or 'sickle' shaped.1 Sickle cells are less effective at carrying oxygen, don't last as long as healthy cells and risk getting stuck in blood vessels, leading to blockages known as vaso-occlusion.2,3-7 Sickle cell disease is characterized by acute and chronic pain, anemia and fatigue alongside vaso-occlusive crises (VOCs), which can require hospitalization and can lead to complications including organ damage.4,7 Globally, there are almost 8 million people living with sickle cell disease.8
About etavopivatEtavopivat is an investigational, oral, small-molecule activator of erythrocyte pyruvate kinase (PKR) in development for the treatment of sickle cell disease and other hemoglobinopathies.9 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 increase oxygen affinity, reduce hemolysis and decrease vaso-occlusive crises (VOCs).9,10
About hemophiliaHemophilia is a rare inherited bleeding disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding.11 It is estimated to affect approximately 1,125,000 people worldwide.12 There are different types of hemophilia, which are characterized by the type of clotting factor protein that is defective or missing.11 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).11 Hemophilia is often treated by replacing the missing clotting factor via intravenous infusions, also known as replacement therapy.13 However, sometimes the body can produce inhibitors as an immune response to the clotting factors in the therapy, which means replacement therapy does not work and limits overall treatment options.14
About Mim8Mim8 is a FVIIIa mimetic bispecific antibody being investigated for once-weekly, once biweekly, and once-monthly prophylaxis for people living with hemophilia A, with and without inhibitors.15 Administered subcutaneously, Mim8 is designed to bridge Factor IXa/X (FIXa/FX) together upon activation, thereby replacing missing FVIII, which helps to restore thrombin generation capacity, and blood clotting.15,16 The use of Mim8 in people living with HA/HAwI is investigational and not approved by any regulatory authority.
About concizumabConcizumab is an investigational tissue factor pathway inhibitor (TFPI) antagonist designed to block a protein in the body that stops blood from clotting.17 By blocking TFPI, concizumab encourages the production of thrombin, which helps to clot the blood and prevent bleeding.17
About Novo NordiskNovo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat serious chronic diseases, built upon our heritage in diabetes. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 72,000 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Instagram, X, LinkedIn, and YouTube.
Contacts for further information
Media:
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Liz Skrbkova (US)+1 609 917 [email protected]
Investors:
Jacob Martin Wiborg Rode+45 3075 [email protected]
David Heiberg Landsted +45 3077 6915 [email protected]
Sina Meyer+45 3079 [email protected]
Frederik Taylor Pitter+1 609 613 [email protected]
Ida Schaap Melvold
+45 3077 [email protected]
References
Novo Nordisk is a registered trademark of Novo Nordisk A/S. © 2024 Novo Nordisk All rights reserved. US24NNG00067 December 2024
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SOURCE Novo Nordisk
