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REGENXBIO Announces Completion of Pivotal Enrollment and Initiates Commercial Production in Duchenne Gene Therapy Program
Patients treated with RGX-202 demonstrate consistent, robust microdystrophin expression and functional improvement compared to natural history in Phase I/II
About this update from Regenxbio Inc.
[{"type":"text","content":"\n \n Patients treated with RGX-202 demonstrate consistent, robust microdystrophin expression and functional improvement compared to natural history in Phase I/II portion of AFFINITY DUCHENNE® trial supporting potential approval via the accelerated approval pathway\n \n \n REGENXBIO continues to enroll patients in the confirmatory trial\n \n \n First batches intended for commercial supply manufactured at in-house Manufacturing Innovation Center\n \n \n Capacity to produce up to 2,500 RGX-202 doses per year\n \n \n \n \n Topline pivotal data now expected in early Q2 2026 and BLA submission in mid-2026\n \n \n \n ROCKVILLE, Md., Oct. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the completion of enrollment in the AFFINITY DUCHENNE® pivotal trial of RGX-202, an investigational gene therapy for the treatment of Duchenne muscular dystrophy, as well as the successful production of the first batches intended for commercial supply.\n \"The Duchenne community urgently needs new treatment options that provide durable, safe outcomes and can meaningfully change the course of this degenerative disease. Completing this pivotal trial milestone and manufacturing in-house our first doses intended for commercial use bring us even closer to delivering RGX-202 as a potential best-in-class gene therapy for Duchenne patients with limited options,\" said Curran Simpson, President and Chief Executive Officer, REGENXBIO. \"The differentiated therapeutic approach behind RGX-202, including our industry-leading product purity levels and novel construct with the C-Terminal domain, has resulted in the positive safety and efficacy profile, with consistent functional benefit seen in Phase I/II. With these highly encouraging results, we are committed to expanding our commercial supply and sharing topline pivotal data in early Q2 of next year.\"\n REGENXBIO continues enrolling ambulatory participants aged 1 year and above in the confirmatory trial.\n \n AFFINITY DUCHENNE® TRIAL\n The pivotal portion of the multi-center, open-label Phase I/II/III AFFINITY DUCHENNE trial completed enrollment of 30 participants in October 2025. To support accelerated approval, the primary pivotal endpoint is the proportion of participants whose RGX-202 microdystrophin expression is ≥10% at Week 12. Secondary endpoints include change from baseline on timed ...