Novo Nordisk advances Forma’s sickle cell treatment to Phase 3

Novo Nordisk’s experimental drug, etavopivat, designed for daily use in treating sickle cell disease, has shown promising results in reducing pain crises in a yearlong Phase 2 study. This Danish pharmaceutical giant, renowned for its treatments in diabetes and obesity, intends to initiate a Phase 3 trial for etavopivat in December, as indicated in a federal clinical trials database. Novo Nordisk had acquired the drug following its $1.1 billion acquisition of Forma Therapeutics in 2022. The company has not provided further comments on the future plans for etavopivat.

The Phase 2 HIBISCUS trial involved 60 participants who were administered either 200 mg of etavopivat, 400 mg of the drug, or a placebo daily for a year. Participants were allowed to continue any other standard treatments with consistent dosing. The results were significant, with both low and high doses of etavopivat reducing the frequency of vaso-occlusive crises, a painful complication of sickle cell disease, by approximately 46% compared to the placebo group.

Patients who were given the low and high doses of etavopivat experienced an annual rate of 1.06 and 1.07 pain episodes, respectively, in contrast to those on the placebo, who had an annual rate of 1.97. Etavopivat functions by activating pyruvate kinase, an enzyme in red blood cells that helps convert sugar into energy. The aim is that by increasing the activity of this enzyme, it will result in modifications in the red blood cells, enhancing their oxygen-binding capacity and preventing the sickling process characteristic of sickle cell disease.

The study indicated that adverse events were mostly mild to moderate and resolved without intervention. However, there were some serious adverse events. One participant on the low dose experienced increased liver enzymes, and one on the high dose experienced a decrease in hemoglobin, both potentially linked to the drug. Additionally, three participants taking the 400 mg dose reported insomnia.

These findings offer hope for a new treatment option for those suffering from sickle cell disease, a condition that currently has limited therapeutic options. The upcoming Phase 3 trial will be crucial in determining the efficacy and safety of etavopivat in a larger patient population, potentially paving the way for its approval and widespread use.
 

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