Agios shares plunge as it drops development of AG-519

US cancer metabolism specialist Agios Pharmaceuticals (Nasdaq: AGIO) saw its shares drop 20% to $44.74 today, after it announced that development of its second pyruvate kinase-R (PKR) activator, AG-519, was to be discontinued following feedback from the US Food and Drug Administration.

The company said it has withdrawn its Investigational New Drug (IND) application following a verbal notification of a clinical hold from the FDA, due to a case of cholestatic hepatitis in a patient taking the drug in a Phase I trial. These decisions do not affect the company’s ongoing global Phase II study (DRIVE PK) for AG-348, a novel, first-in-class activator of both wild-type (normal) and mutated PKR enzymes. Agios is advancing AG-348 into pivotal development as the first potential disease-modifying treatment for pyruvate kinase (PK) deficiency.

“We share the FDA’s commitment to patient safety and believe this is the right decision to ultimately help people with PK deficiency,” said David Schenkein, chief executive officer at Agios. “As the lead compound in our PKR program, AG-348 has demonstrated clear proof of concept with robust, rapid and sustained increases in hemoglobin in patients with PK deficiency. Based on our clinical experience with DRIVE PK, we are developing a registration path for AG-348 in adult PK deficiency patients and plan to discuss this strategy with regulators,” Dr Schenkein added.