Novo Nordisk A/S (NVO), announced topline results from HIBISCUS, a pivotal phase 3 trial of once-daily oral Etavopivat, in adults and adolescents with sickle cell disease. The trial met both co-primary endpoints and showed greater haemoglobin response than placebo.
Sickle cell disease is driven by the polymerisation of mutated sickle haemoglobin in red blood cells, and affects around 8 million people worldwide.
Etavopivat is a first-in-class oral, once-daily pyruvate kinase-R (PKR) activator developed to treat sickle cell disease. Trial Details
The HIBISCUS trial was a randomised, double-blinded, 52-week efficacy and safety trial investigating Etavopivat 400 mg versus placebo in 385 people aged 12 years or older with SCD. Participants were allowed to receive standard-of-care treatment throughout the trial.
Key Findings
The results demonstrated that Etavopivat met both co-primary endpoints, achieving superior reductions in vaso-occlusive crises (VOCs) and superior improvement in haemoglobin (Hb) response compared with placebo.
In the trial, people treated with Etavopivat demonstrated a superior 27% reduction in the annualised rate of VOCs compared with placebo.
Haemoglobin response was superior with Etavopivat, with 48.7% of people on treatment achieving an increase of more than 1g/dL after 24 weeks, versus 7.2% on placebo.
The time to first VOC was significantly prolonged with Etavopivat, with a median of 38.4 weeks versus 20.9 weeks with placebo.
In the trial, Etavopivat appeared to be well tolerated, with a topline safety profile consistent with previous Etavopivat trials.
What's Ahead for Etavopivat?
Novo Nordisk plans to submit for the first regulatory approval of Etavopivat in the second half of 2026.
The U.S. Food and Drug Administration (FDA) has granted Etavopivat Fast Track, Rare Pediatric Disease and Orphan Drug designations.
NVO has traded between $35.13 and $81.44 over the last year. The stock closed Friday's trade at $40.52, down 1%.
In the pre-market, NVO is down 0.64% at $40.24.
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