Skip to Main Content

Agios Pharmaceuticals on Friday reported positive results from the first clinical trial of its lead pipeline drug in patients with two different types of thalassemia, an inherited blood disorder.

Overall, 12 of the 13 patients treated with the Agios drug called mitapivat achieved meaningful hemoglobin responses in the Phase 2 study. The results are still preliminary but set up a pivotal Phase 3 studies starting next year, the company said.

Unlock this article by subscribing to STAT+ and enjoy your first 30 days free!

GET STARTED