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April 17, 2014
08:04 EDTAGIOAgios initiates AG-348 Phase 1 study
Agios Pharmaceuticals announced dose administration of AG-348 in a Phase 1 study in healthy volunteers. AG-348 is an orally available, potent, selective small molecule activator of pyruvate kinase-R, a metabolic enzyme, which, when mutated, leads to pyruvate kinase deficiency, a rare, inherited hemolytic anemia. AG-348 is wholly owned by Agios, with the company maintaining full worldwide development and commercialization rights. The company said, “Preclinical studies have demonstrated that AG-348 activates a broad spectrum of PK-R mutant proteins, and corrects the metabolic defects found in patient-derived blood samples. These data support the hypothesis that drug intervention with AG-348 will restore glycolytic pathway flux and normalize red blood cell metabolism. Because AG-348 directly targets the underlying disease, it has the potential to be an important treatment option for patients with PK deficiency.”
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