General Updates | The potential of tebapivat in sickle cell disease

Yes, so tebapivat is a novel pyruvate kinase activator. So pyruvate kinase is the most important enzyme in glycolysis. It generates 50% of the red blood cells’ ATP. And we know that in sickle cell disease, pyruvate kinase activity and stability is impaired. And previous work have already shown that mitapivat, the first pyruvate kinase activator, works quite well in sickle cell disease. So with this novel agent, tebapivat, we looked into ex vivo activation of pyruvate kinase in sickle cell disease, and we found that tebapivat actually stabilizes pyruvate kinase and activates pyruvate kinase even better than mitapivat. And we saw an increase in ATP and a decrease in 2,3-DPG, which was accompanied by a reduced sickling of the red blood cells. And I think what was most important that we also showed that there was a lower red blood cell adhesion to laminin. And what we also looked into is if there were differential results on low-density and high-density red blood cells. And we actually found that tebapivat could activate pyruvate kinase in both red blood cell populations. And that the most prominent effect was seen in the higher-density blood cells. But yeah, in this study, we could show that tebapivat works quite well. And also what I said in high-density red blood cells, but also low-density red blood cells.

This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.