AGIO

Mitapivat (pyruvate kinase R (PKR) activator)

Sickle Cell Disease

Stage (next event)

Expected Date

Phase 3 to be Initiated

H1 2020

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?

  • H1 2020 


PRIOR DATA/EVENTS

PRESS RELEASES

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.

  • Source 

Updated by MV

Prior Data (click to view full image)

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