BioCentury
ARTICLE | Clinical News

Agios falls after revealing PKR activator data

June 10, 2016 1:26 AM UTC

Agios Pharmaceuticals Inc. (NASDAQ:AGIO) sank $9.87 (15%) to $56.22 on Thursday after the company said data from a Phase I study showed proof-of-mechanism for AG-519, but disclosed safety data for the small molecule activator of pyruvate kinase R that may have worried investors. The compound is one of two Agios programs for pyruvate kinase deficiency (PKD).

One volunteer among 16 in the trial's first two multiple-ascending dose cohorts experienced grade 2 thrombocytopenia on day 14 after receiving 375 mg of AG-519 twice daily, Agios said. The subject's platelet levels started to recover within five days of the last dose, and had returned to normal within seven days. ...