Novo Nordisk to present 13 abstracts at ASH 2024 including phase 2/3 HIBISCUS trial of etavopivat

Novo Nordisk announced the presentation of 13 abstracts, three of which will be presented in oral sessions, at the upcoming 66^th 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.

In an oral presentation on 7 December, 52-week results from the phase 2 part of the ongoing phase 2/3 HIBISCUS trial of etavopivat will be presented. These results have determined the dose for the phase 3 part of the trial, as well as examined the safety profile and efficacy of etavopivat, including the incidence of vaso-occlusive crises (severe pain caused when blood vessels are blocked and deprive tissues of oxygen¹) in patients with sickle cell disease. The results from HIBISCUS have been chosen to 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.

From Novo Nordisk’s haemophilia portfolio, there are two oral presentations of note: first on the efficacy and safety results of an interim analysis from FRONTIER4, a phase 3 open-label, multi-centre extension study of Mim8 in people living with haemophilia A with and without inhibitors (HA/HAwI). Additionally, an oral presentation of data from the phase 3 explorer7 study assessing the efficacy of concizumab in people with haemophilia 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 haemophilia, where we continue to advance research to address the unmet needs of patients.”

Summary of presentations
Accepted data at the 66^th ASH annual meeting include 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 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 haemoglobin-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 is associated with the likelihood of wanting to participate in a clinical trial: findings from the global LISTEN survey (1135-P)
• Motivators and barriers for people with sickle cell disease participating in clinical trials: United States findings from the LISTEN survey (1135-P)
• Noninvasive, accessible smartphone app for at-home hemoglobin monitoring in sickle cell disease (2248-P)

Haemophilia

• 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).