Agios’ genetically driven disease pipeline also includes the PKR activator AG-946, which is being tested in a Phase I healthy volunteer study with plans to initiate future studies in patients with hereditary spherocytosis, anemia associated with low-risk myelodysplastic syndromes and additional hereditary hemolytic anemias.

Other inhibitors of pyruvate kinase (PK) and PK isozymes M1/M2 (PKM2) also are in development potentially for the treatment of anemia or chronic kidney disease, retinal indications, diabetic retinopathy and nephropathy, hereditary myopathies and neurologic indications, including amyotrophic lateral sclerosis.

The company also is developing a drug against phenylalanine hydroxylase (PAH) for the treatment of phenylketonuria and multiple candidates targeting BCAT-II in methylmalonic acidemia, propionic acidemia and multiple rare inborn errors of metabolism.

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