Agios has announced top line data from the RISE UP Phase III trial assessing mitapivat, an oral pyruvate kinase (PK) activator, in subjects aged 16 years or above with sickle cell disease.
The global, randomised, double-blind, placebo-controlled trial is designed to evaluate the drug’s impact on haemolysis and multiple disease parameters over a 52-week double-blind period.
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Primary endpoint of haemoglobin response was achieved and mitapivat demonstrated a statistically significant improvement versus placebo.
Although mitapivat decreased the annualised rate of sickle cell pain crises (SCPCs), this reduction did not attain statistical significance.
Additionally, the therapy significantly improved two key secondary endpoints, indirect bilirubin levels, a marker of haemolysis and average haemoglobin concentration. However, the trial did not meet the key secondary endpoint of fatigue improvement measured by the Promis Fatigue 13a score.
Based on a post hoc analysis, subjects achieving haemoglobin response in the mitapivat arm experienced clinically significant benefits, such as reduced fatigue, reduced SCPC rates, and fewer hospitalisations for SCPCs.
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By GlobalDataThe drug’s safety profile was consistent with previous mitapivat trials in sickle cell disease.
RISE UP enrolled 207 participants representing the worldwide sickle cell disease population and randomised them into 2:1 ratio to mitapivat twice daily or placebo.
The 52-week period was completed by 87% of participants on mitapivat and 81.2% on placebo. Most of them entered a 216-week open-label extension period.
Mitapivat’s safety profile showed similar rates of adverse events compared to placebo, with serious adverse events lower in the mitapivat arm. Deaths were not deemed related to treatment.
Agios chief medical officer and R&D head Sarah Gheuens said: “We plan to engage with the FDA to discuss these findings and our goal of bringing this innovative medicine to patients with sickle cell disease.”
The company plans to apply for potential regulatory approval in the US.
