Agios slumps on mixed Phase III trial of mitapivat in sickle cell disease

Cambridge, USA-based Agios Pharmaceuticals’ (Nasdaq: AGIO) shares closed down 51% at $22.34 yesterday, after the company announced top-line results from the 52-week double-blind period of the global RISE UP Phase III trial of mitapivat, an oral pyruvate kinase (PK) activator, in patients aged 16 years or older with sickle cell disease.

RISE UP was designed with two primary endpoints and five key secondary endpoints to evaluate objective measures of hemolysis improvement as well as additional parameters of sickle cell disease. This comprehensive design enabled a broad assessment of the potential benefits of mitapivat across multiple aspects of the disease. The trial met its primary endpoint of hemoglobin response, with mitapivat demonstrating a statistically significant improvement compared to placebo.