▎WuXiEdited by Kant Content Team

Recently, the American Society of Hematology (ASH) released abstracts of some studies to be presented at the 2024 annual meeting. Among them,Novo Nordisk's investigational therapy etavopivat reduced the incidence of vaso-occlusive crises (VOC) by nearly 50% in patients with sickle cell anemia (SCD) in a Phase 2 clinical trial.Information from ClinicalTrials.gov shows that the Phase 3 clinical trial of this small-molecule oral therapy is expected to commence by the end of this year.

Etavopivat is a once-daily selective pyruvate kinase-R (PKR) agonist. It can reduce the content of anaerobic glycolysis product 2,3-DPG in red blood cells by activating the natural PKR within red blood cells, enabling the cells to carry more oxygen, increase adenosine triphosphate (ATP) production, and reduce hemolysis and the formation of sickle cells.Novo Nordisk Invested Approximately $1.1 Billion in 2022AcquisitionForma Therapeutics Acquires This Therapy.

The results of the Phase 2 clinical trial, published in the abstract, showed that 60 patients with SCD received low-dose (200 mg), high-dose (400 mg) etavopivat, or placebo treatment.In the intention-to-treat population, the annualized VOC rate was 1.07 in the low-dose group, representing a 45% reduction compared to the placebo group (1.97), and 1.06 in the high-dose group, representing a 46% reduction compared to the placebo group.

Moreover, in the intention-to-treat population, the average hemoglobin levels increased, and all hemolysis biomarkers improved after 24 weeks of treatment in both dose groups. The abstract stated that etavopivat treatment for SCD achieved clinical proof of concept based on the overall data.

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