ASH Clinical News ACN_4.6_Full_Issue_web | Page 43

PK DEFICIENCY
CLINICAL TRIALS
NOW ENROLLING
For adult patients with PK deficiency who are not regularly
transfused.
For adult patients with PK deficiency who receive regular
blood transfusions.
Do you have a patient with
PK DEFICIENCY?
Two Phase 3 studies of AG-348 are now enrolling
adult patients with pyruvate kinase (PK) deficiency.
This investigational drug is an orally available, potent,
small molecule activator of pyruvate kinase-R (PKR),
the defective enzyme in PK deficiency.
For study criteria, visit activateclinicaltrials.com
or clinicaltrials.gov.
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The safety and efficacy of AG-348 have not been established. There
is no guarantee that AG-348 will receive health authority approval or
become commercially available for the use being investigated.
©2018 Agios Pharmaceuticals, Inc. All rights reserved. PKD-US-0050