Agios's mitapivat data in sickle cell disappoints at ASH

 

• An update on Agios Pharmaceuticals’ (AGIO -15.3%) experimental sickle cell disease medicine, mitapivat, has potentially missed some investors’ expectations after it was shown that six of 11 patients hit a key benchmark to show improvement of their disease in Phase 1 study. Data were presented at the American Society of Hematology.
• According to J.P. Morgan analyst, investors were looking for ~60%-70% of patients to show 1.0 gram/deciliter or more improvement of their hemoglobin levels, but the company reported 55% of patients showed such a degree of improvement.
• Mitapivat was associated with decreases in hemolytic markers such as bilirubin, lactate dehydrogenase and reticulocytes. One patient discontinued the study due to a pre-existing pulmonary embolism.
• One previously reported vaso-occlusive crisis (VOC) occurred during drug taper and was viewed as possibly drug- related. Two other VOCs occurred during 28-day safety follow-up after drug exposure but were not attributed to mitapivat.
• Agios plans to start mitapivat Phase 3 trials in sickle cell disease and thalassemia in 2021.
• Last week the company announced that primary endpoint of a Phase 3 trial in pyruvate kinase deficiency who do not receive regular transfusions, was met.
• Also the company expects topline data from a single arm trial, ACTIVATE-T evaluating mitapivat in patients who receive regular transfusions, in Q1 2021.
• https://seekingalpha.com/news/3642553-agioss-mitapivat-data-in-sickle-cell-disappoints-ash-presentation