Agios begins AG-348 Phase 1 study to treat PK deficiency

Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the fields of cancer metabolism and inborn errors of metabolism (IEM), today 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 (PK-R), a metabolic enzyme, which, when mutated, leads to pyruvate kinase (PK) deficiency, a rare, inherited hemolytic anemia. There is substantial unmet need among patients with PK deficiency as there is no therapy available to treat the underlying disease. AG-348 is wholly owned by Agios, with the company maintaining full worldwide development and commercialization rights.

"Today's announcement marks the initiation of our first clinical program in IEMs and our third as a company," said David Schenkein, M.D., chief executive officer of Agios. "Agios believes in the potential of small molecules to become disease-modifying therapeutics for rare genetic disorders by targeting the specific metabolic defects driving these diseases. We expect that the results from this healthy volunteer study will enable us to move AG-348 rapidly into a study in patients with PK deficiency."

"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," said Scott Biller, chief scientific officer of Agios. "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."