Agios Pharmaceuticals, Inc.’s topline results from the Phase II portion of its Phase II/III RISE UP trial of Pyrukynd (mitapivat) in sickle cell disease (SCD) show potential to significantly expand the addressable market for the drug, albeit in a space that has grown increasingly competitive in the past few years. They also indicate how rapidly the company has developed its pipeline since selling off its oncology business to Les Laboratoires Servier in 2020.
Agios announced 26 June the results from the 79 patients enrolled into the first portion of the Phase II/III study, showing that 46.2% of patients receiving Pyrukynd at 50mg twice daily and 50% of those receiving it at 100mg twice daily achieved a hemoglobin response, compared with 3