Tony [Analyst]'s questions to Regenxbio Inc (RGNX) leadership • Q3 2024
Question
Tony [Analyst], on behalf of Gena Wang, asked about the RGX-202 Duchenne program, focusing on durability in the 1-3 year old cohort, biomarker requirements for accelerated approval, and the expected magnitude of functional change in upcoming data.
Answer
Dr. Steve Pakola, Chief Medical Officer, addressed durability by citing the 12+ year durability of the AAV8 vector in liver-directed hemophilia, expressing confidence for muscle tissue. CEO Curran Simpson added that no additional biomarker data has been requested by regulators and that existing benchmarks, such as those from the ELEVIDYS review, provide a good precedent for assessing the upcoming functional data, especially given RGX-202's high microdystrophin expression levels.