Agios halts development of anemia drug

Agios Pharmaceuticals Inc. said Thursday it has halted development of an experimental drug to treat a form of anemia after regulators, concerned about a safety issue surfacing in an early-stage clinical study, put a hold on clinical testing of the compound.

Agios disclosed for the first time Thursday that the Food and Drug Administration had ordered it to suspend testing of the drug after a healthy volunteer in a British clinical trial was diagnosed with drug-induced cholestatic hepatitis, the obstruction of bile secretion. Agios said the patient is being monitored and no other adverse events have been reported. Nonetheless, the company has stopped the British study and a separate palability study with US volunteers.


The surprise disclosure, which came after financial markets closed, sent the company’s shares down 19.4 percent to $45.06 in after-market trading on the Nasdaq exchange.

Agios, founded in 2008, has been one of the most closely followed companies in the Cambridge biotech cluster. It is developing a portfolio of medicines in the fields of cancer metabolism and rare genetic metabolic disorders. The drug it pulled from its pipeline seeks to treat pyruvate kinase deficiency, or PK deficiency, a rare inherited disease that destroys red blood cells.

The company said it is moving forward with another experimental drug that has the potential to treat PK deficiency in a different way. That drug candidate is continuing to be tested in a mid-stage clinical trial

“We share the FDA’s commitment to patient safety and believe this is the right decision to ultimately help people with PK deficiency,” Agios chief executive David Schenkein said in a statement.

Robert Weisman can be reached at robert.weisman@globe.com. Follow him on Twitter @GlobeRobW.