Agios will not advance tebapivat in sickle cell disease after a 59-patient Phase 2 trial failed to establish a differentiated hemoglobin response. Response rates ranged from 29.4% to 47.1% across three doses versus 33.3%…
Agios Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease (SCD) and granted Priority Review, establishing a PDUFA target…
Beam Therapeutics announced updated biomarker data from the Phase 1/2 BEACON trial of ristoglogene autogetemcel (risto-cel), reinforcing its position as the leading base-edited cell therapy program in sickle cell disease ahead of a planned…
Agios Pharmaceuticals submitted a supplemental NDA to the FDA seeking accelerated approval of mitapivat for sickle cell disease, positioning the oral PKR activator in a highly competitive emerging therapeutic class.
