Clinical Trial ProgressRegenXBio has initiated a pivotal trial, a multicenter, open-label Phase 3 portion of the AFFINITY DUCHENNE trial, with a focus on microdystrophin expression as a key endpoint for potential accelerated approval.
Market OpportunityThe potential to dose younger patients broadens the addressable market and opens a niche for first therapy approval for certain ages.
Safety And TolerabilityRGX-202 shows a clean safety profile with no severe adverse events, which should alleviate safety concerns ahead of the pivotal trial.