REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include a New Cohort of Younger Patients

• Company to begin enrollment of patients aged 1-3 years
  • Expects data from younger cohort to be part of pivotal plans and BLA filing for broad label
• End-of-Phase II meeting with FDA scheduled for late July to finalize pivotal program design
  • Based on recent commercial landscape, confirmed accelerated approval pathway remains available given ongoing unmet need and RGX-202 differentiated design
• Remains on track to initiate pivotal trial in late Q3 to early Q4 2024
  • The next potential therapy to become available for Duchenne patients

REGENXBIO announced they are expanding their AFFINITY DUCHENNE Trial, which delivers a micro-dystrophin via AAV8, to include a new cohort of younger patients. The trial, which has been enrolling ambulatory boys with Duchenne aged 4 to 11, is now expected to enroll up to five individuals aged 1-3 as well. These younger participants will receive RGX-202 at the pivotal dose level (dose level 2, at 2×1014 genome copies/kg body weight). REGENXBIO expects to share initial strength and functional assessment data for individuals already treated (at both dose levels) later this year.

Press Release here