ASH 2024 | Results of the Phase II HIBISCUS trial investigating etavopivat in sickle cell disease

This year I’m presenting the Phase II results of the HIBISCUS study. It’s a clinical trial which is the largest double-blind clinical trial in sickle cell disease to date and it is evaluating the effects of a novel activator, a pyruvate kinase activator named etavopivat. In this trial, 60 patients with sickle cell disease of all genotypes have been randomized in this Phase II in three-arm cohorts with the majority of the patients receiving as a standard of care hydroxyurea, which is the oldest drug that we have for this disease. In the 12-week follow-up analysis, after the last patient was enrolled, was the analysis that helped determine the optimal dose of etavopivat that is going to be used in the Phase III clinical trial. The 52-week efficacy analysis showed us a 46% reduction of the painful vaso-occlusive crisis per year and showed an improvement in hemoglobin levels and in the hemolytic markers of these patients, which is very important. Also, the groups that were in the investigational product of etavopivat had double the interval time between the first VOC episode than the others who were in the placebo arm. Also, they had a positive effect in quality of life by reducing the hemolysis and had these good responses to the VOC crisis. And that was measured by a fatigue score that was improved too. We didn’t have any major serious adverse events and the investigational product showed that it was well tolerated by most of the patients. The adverse events that were reported were mild to moderate and were resolved without the need of any additional medical treatment. So now we have the recruitment of the HIBISCUS 2 Phase III clinical trial which is ongoing and we look forward to its completion and the results.