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REGENXBIO REPORTS POSITIVE BIOMARKER DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202 GENE THERAPY
Positive biomarker data in patient aged 1-3 add to consistent, robust microdystrophin and transduction levels across all treated ages Patient aged 3 years at
About this update from Regenxbio Inc.
[{"type":"text","content":"Positive biomarker data in patient aged 1-3 add to consistent, robust microdystrophin and transduction levels across all treated ages Patient aged 3 years at dosing had expression level at 122.3% compared to controlWith a differentiated novel construct and proactive short course immune modulation regimen, RGX-202 continues to demonstrate encouraging safety profile with no SAEs or AESIs Phase III portion of AFFINITY DUCHENNE® trial enrolling ambulatory patients aged 1 and above, on track for BLA submission mid-2026ROCKVILLE, Md., March 19, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated investigational gene therapy for Duchenne muscular dystrophy (Duchenne). Results were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.\n\"RGX-202 is the only next generation gene therapy for Duchenne in a pivotal phase trial. The new data from the age 1-3 cohort builds on the favorable safety and efficacy profile seen in ages 4 and older and reinforces the potential of RGX-202 to serve a wide age range of patients,\" said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. \"The consistent, robust microdystrophin levels seen across the age range as well as the functional improvements previously reported support RGX-202's potential to alter the course of this devastating disease. We look forward to sharing additional Phase I/II functional data in the first half of 2025. We also continue to rapidly advance the pivotal trial towards completing enrollment this year and BLA submission mid-2026.\"\n\"Patients with Duchenne continue to be in need of treatment options that could meaningfully impact the course of disease,\" said Carolina Tesi-Rocha, M.D., Stanford Children's Hospital. \"The microdystrophin expression and biomarker data presented represent key indicators of potential therapeutic effect. Combined with the safety and functional data to date, I am highly encouraged by the profile of RGX-202.\"\nAFFINITY DUCHENNE Phase I/II Interim Data Updates\nBiomarker DataNew biomarker data from two patients who received the pivotal dose of RGX-202 were presented at MDA and continue to support consistent, robust expression and transduction of RGX-202 m...