STAT Plus: Agios drug demonstrates strong response in patients with inherited blood disorder
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.
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