Phase 2 HIBISCUS study suggests etavopivat may reduce sickle cell vaso-occlusive crises

Novo Nordisk has announced notable findings from its phase 2 HIBISCUS study on etavopivat, an experimental treatment for sickle cell disease. These results, shared at the 66th Annual Meeting of the American Society of Hematology in San Diego, highlight a promising future for the drug.

Etavopivat, currently under investigation, aims to alleviate the challenges associated with sickle cell disease, a condition marked by the irregular shape of red blood cells that impairs their ability to transport oxygen, leading to severe pain and potential organ damage. The phase 2 study intended to determine the appropriate dosage for phase 3, involved patients who were administered 200 mg or 400 mg of etavopivat or a placebo.

The study yielded encouraging data, showing a potential reduction in vaso-occlusive crises (VOCs) and improvement in hemoglobin levels over time. Specifically, annualized VOC rates after 52 weeks were 1.07 in the 200 mg group and 1.06 in the 400 mg group, compared to 1.97 in the placebo group. The median time to the first VOC was considerably longer for those treated with etavopivat (33.6 weeks) compared to those on placebo (16.9 weeks). At the 24-week mark, the hemoglobin response rate, defined as an increase of more than 1 g/dL from baseline, was 38% for the 200 mg group and 25% for the 400 mg group, while the placebo group saw a mere 10.5% increase.

Moreover, patient-reported outcomes indicated improvements in fatigue levels among those taking etavopivat. In the per-protocol population, which adhered strictly to the study guidelines, the annualized VOC rates and hemoglobin response rates were even more favorable. However, there were some adverse effects noted, including increased hepatic enzyme levels and a cerebrovascular accident in the 200 mg group, as well as decreased hemoglobin levels in the 400 mg group.

Dr. Julie Kanter of the University of Alabama at Birmingham emphasized the need for new treatments for sickle cell disease, given the historical underfunding and limited treatment options for this condition. She expressed optimism about the potential impact of etavopivat on patients' lives.

Novo Nordisk, with its extensive experience in hematology and rare diseases, aims to revolutionize the care standard for sickle cell disease. The company hopes that etavopivat will not just alleviate symptoms but also alter the disease's progression. Martin Holst Lange, the company's executive vice president, highlighted this ambition, noting that while further studies are necessary, the HIBISCUS findings are a promising step forward.

Sickle cell disease affects millions globally and poses significant health challenges. The HIBISCUS trial represents a critical effort to address this by investigating the safety and efficacy of etavopivat over a 52-week period, with the potential for long-term open-label extension.

Etavopivat works by activating erythrocyte pyruvate kinase, which could increase the oxygen affinity of red blood cells, reduce hemolysis, and decrease the frequency of VOCs. The phase 3 component of the HIBISCUS trial will further explore these outcomes in a larger patient population.

Novo Nordisk, a global healthcare leader, continues to leverage its heritage in diabetes and other chronic diseases to address the unmet needs in sickle cell disease. The findings from the HIBISCUS study, coupled with ongoing research, aim to bring transformative solutions to those affected by this debilitating condition.