Agios Pharmaceuticals Inc. has started a phase I dosing study of its lead candidate for inborn errors of metabolism, AG-348. The experimental treatment for pyruvate kinase (PK) deficiency, a rare inherited metabolic disorder, could help rapidly advance the drug toward a proof-of-concept test in target patients.
The investigational therapy showcase's the company's core capability: exploring cellular metabolism to identify cellular targets, then designing small molecules with the potential to correct metabolic defects at the heart of disease.
Agios is developing AG-348 to be the first approved treatment for PK deficiency, a type of hemolytic anemia, a rare condition in which red blood cells are destroyed at an accelerated rate and removed from the bloodstream. Mild cases don't require treatment, but the deficiency can lead to severe anemia, fatigue and other symptoms and can require blood transfusions and even the removal of a patient's