Topline Results: Phase 3 Energize Study of Pyrukynd in Thalassemia
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There are no approved oral treatments for non-transfusion-dependent
thalassemia (NTDT)
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Thalassemia is a group of genetic disorders impacting a- and/or B-globin genes, resulting in an
imbalance of globin production ¹,2
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Excess globin chains precipitate and are toxic to red blood cells (RBCs), directly leading to in effective erythropoiesis
and hemolysis²
Thalassemic RBCs lack sufficient levels of ATP to meet the increased energy demands
associated with degradation of globin chain precipitates and cellular oxidative stress
responses 3,4
Although patients with non-transfusion-dependent thalassemia (NTDT) do not require regular
blood transfusions for survival, they can experience chronic anemia and serious
complications 1,2
Treatment options for NTDT are supportive only, highlighting an unmet need for disease-modifying therapies 5
Mitapivat is an investigational, first-in-class, oral, small-molecule allosteric activator of pyruvate
kinase (PK) in RBCs, a key enzyme that regulates ATP production6
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PYRUKYND® is approved in the U.S., EU, and Great Britain for adult PK deficiency and is under investigation for pediatric PK deficiency, thalassemia, and sickle cell disease.
ATP, adenosine triphosphate; NTDT, non-transfusion-dependent thalassemia; PK, pyruvate kinase; RBC, red blood cell; 1. Taher AT et al. Lancet 2018;391:155-67; 2. Galanello R et al. Orphanet J Rare Dis
2010;5:11; 3. Khandros E et al. Blood 2012;119:5265-75; 4. Shaeffer JR. J Biol Chem 1988;263:13663-9; 5. Musallam KM et al. Haematologica 2021;106:2489-92; 6. Kung C et al. Blood 2017;130:1347-56View entire presentation