Alex Nackenoff's questions to Agios Pharmaceuticals Inc (AGIO) leadership •
Question
Alex Nackenoff from Raymond James asked if the RISE UP study in sickle cell disease needs to succeed on both primary endpoints for a regulatory filing, or if success on one, like hemoglobin response, plus a key secondary endpoint like fatigue, could be sufficient for approval.
Answer
CMO Dr. Sarah Gheuens explained that while the trial targets both endpoints, hitting one primary allows for alpha to be transferred to test secondary endpoints, such as fatigue, to demonstrate clinical benefit. She emphasized their 'hemoglobin plus' strategy, noting regulators will make the final benefit-risk assessment. CEO Brian Goff added this comprehensive design was planned from the outset.