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REGENXBIO Announces Expansion of AFFINITY DUCHENNE® Trial to Include a New Cohort of Younger Patients
Company to begin enrollment of patients aged 1-3 yearsExpects data from younger cohort to be part of pivotal plans and BLA filing for broad labelEnd-of-Phase
About this update from Regenxbio Inc.
[{"type":"text","content":"Company to begin enrollment of patients aged 1-3 yearsExpects data from younger cohort to be part of pivotal plans and BLA filing for broad labelEnd-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 designRemains on track to initiate pivotal trial in late Q3 to early Q4 2024The next potential therapy to become available for Duchenne patientsROCKVILLE, Md., June 24, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial to evaluate the safety and efficacy of RGX-202 in boys with Duchenne muscular dystrophy (Duchenne).\nRGX-202 is an investigational one-time AAV Therapeutic targeted to deliver a novel microdystrophin, representing the next wave of innovative design in Duchenne gene therapy. RGX-202 is the only gene therapy approved or in development for Duchenne that incorporates the C-Terminal domain, making the RGX-202 transgene the closest to the naturally occurring dystrophin gene.\nWith this announcement, REGENXBIO is enrolling ambulatory boys with Duchenne aged 1 to 11 in the AFFINITY DUCHENNE trial. The new cohort is expected to enroll up to five patients aged 1-3 to receive RGX-202 at the pivotal dose level (2x1014 genome copies (GC)/kg body weight).\nAdditionally, REGENXBIO has confirmed an end-of-Phase II (EOP2) meeting is scheduled with the FDA at the end of July. This meeting is expected to finalize the AFFINITY DUCHENNE pivotal trial design, with the goal of continuing to expedite the development of RGX-202. The Company anticipates that all patients enrolled at dose level 2 (n=12) will be included in its pivotal trial data set. The Company plans to use RGX-202 microdystrophin expression as a surrogate endpoint to support a Biologics License Application (BLA) submission using the accelerated approval pathway with the potential to receive a broad label. REGENXBIO has recently confirmed with the FDA that the pathway can be used given the ongoing high unmet need for differentiated treatment options in the Duchenne community. RGX-202 is the most advanced gene therapy enrolling in active clinical trials and is anticipated to be ...