Stage (next event)
Phase 3 to be Initiated
Catalyst Info & Data Links
TITLE: Mitapivat (pyruvate kinase R (PKR) activator) for Sickle Cell Disease Phase 3 to be Initiated
Clinical Trial (NCT03559699): A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Clinical Trial ( NCT03548220): A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
WHAT IS THE NEXT CATALYST EVENT?
Phase 3 To be Initiated
WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?
MECHANISM OF ACTION
Mitapivat is an investigational, wholly owned, first-in-class, novel, oral activator of both wild-type (normal) and mutated pyruvate kinase-R (PKR) enzymes.
Mutations in PKR cause deficiencies in red blood cell glycolysis, which lead to a disease known as PK deficiency. Mitapivat is currently being studied in two registration-enabling studies for this rare disease.
Additionally, Agios has expanded the clinical application of mitapivat into thalassemia and sickle cell disease where it has the potential to provide therapeutic benefit by activating wild-type PKR. Based on preliminary clinical data, proof-of-concept has been established for mitapivat as a potential treatment for both of these hemolytic anemias. Mitapivat is currently being evaluated in a Phase 2 clinical trial for thalassemia. Agios is also collaborating with the National Institutes of Health (NIH) to evaluate mitapivat in sickle cell disease.
Updated by MV
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